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(Mark One)
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ý
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ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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For the fiscal year ended December 31, 2016
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Or
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o
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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For the transition period from to
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Delaware
(State or other jurisdiction of
incorporation or organization)
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20-8185347
(I.R.S. Employer
Identification No.)
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One Penn Plaza, 19th Floor
New York, NY
(Address of principal executive offices)
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10119
(Zip Code)
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Title of each class
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Name of each exchange on which registered
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Common Stock, $0.001 par value
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The NASDAQ Stock Market LLC
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Large accelerated filer
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Accelerated filer
o
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Non-accelerated filer
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(Do not check if a
smaller reporting company)
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Smaller reporting company
o
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the potential benefits of our updated business plan and strategy to potentially expand our product pipeline;
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our ability to in-license or acquire additional products, product candidates or technologies to treat ophthalmic diseases and the timing, costs, conduct and outcome of preclinical development or clinical trials we undertake for these newly acquired assets;
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our expectations related to our use of available cash;
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our estimates regarding expenses, future revenues, capital requirements and needs for additional financing;
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the timing, costs, conduct and outcome of our remaining Phase 3 clinical trial of Fovista® (pegpleranib) administered in combination with Eylea or Avastin for the treatment of wet age-related macular degeneration, or AMD, including statements regarding the timing and the availability of, and the costs to obtain, initial, top-line results from, and the completion of, such trial;
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the timing, costs, conduct and outcome of our clinical trials for Zimura® (avacincaptad pegol) for the treatment of patients with geographic atrophy, or GA, a form of dry AMD and, in combination with anti-VEGF drugs, for the treatment of wet AMD, including statements regarding the timing of the initiation of and completion of enrollment in such trials, and the costs to obtain and timing of receipt of initial results from, and the completion of, such trials;
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the timing of and our ability to obtain marketing approval of our product candidates, and the ability of our product candidates to meet existing or future regulatory standards;
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our ability to maintain a productive collaborative relationship with Novartis Pharma AG, including our ability to achieve remaining potential milestone payments under our agreement;
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the potential advantages of our product candidates;
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the rate and degree of potential market acceptance and clinical utility of our product candidates, if approved;
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our estimates regarding the potential market opportunity for our product candidates;
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the potential receipt of revenues from future sales of our product candidates, if approved;
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our sales, marketing and distribution capabilities and strategy;
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our ability to establish and maintain arrangements for the manufacture of our product candidates;
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our intellectual property position;
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the impact of existing and new governmental laws and regulations; and
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our competitive position.
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Zimura Phase 2a Wet AMD Study
. During the fourth quarter of 2015, we initiated an open‑label Phase 2a clinical trial to evaluate Zimura’s potential role when administered in combination with anti‑VEGF therapy for the treatment of wet AMD in patients who do not respond adequately to treatment with anti-VEGF monotherapy. We plan to enroll up to approximately 60 patients in this trial, and may include patients with PCV. Recruitment of patients in this study is ongoing.
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Zimura Phase 2/3 GA Study
. During the fourth quarter of 2015, we initiated a randomized, double‑masked, controlled Phase 2/3 clinical trial to evaluate the safety and efficacy of Zimura monotherapy in patients with GA. Recruitment of patients in this study is ongoing.
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mean change in visual acuity in ETDRS letters from baseline at 12 weeks;
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proportion of patients in each treatment group gaining 15 or more ETDRS letters from baseline at 12 weeks;
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proportion of patients in each treatment group gaining 15 or more ETDRS letters from baseline at 24 weeks; and
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mean change in area of choroidal neovascularization from baseline at 24 weeks.
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Arm
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# (%) of Patients
Gaining ≥ 15 letters
at Week 12
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# (%) of Patients
Gaining ≥ 15 letters
at Week 24
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1.5 mg Fovista + Lucentis
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48 (31.8)%
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59 (39.1)%
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0.3 mg Fovista + Lucentis
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31 (21.1)%
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49 (33.3)%
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0.5 mg Lucentis
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33 (22.4)%
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50 (34.0)%
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Monotherapy
Lucentis
N = 148
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0.3 mg Fovista +
Lucentis
N = 149
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1.5 mg Fovista +
Lucentis
N = 152
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Patients With One or More Systemic Serious Adverse Events
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11 (7.4)%
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13 (8.7)%
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9 (5.9)%
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MedDRA System Organ Class(1)
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Cardiac Disorders
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2 (1.4)%
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2 (1.3)%
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2 (1.3)%
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Gastrointestinal Disorders
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1 (0.7)%
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2 (1.3)%
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3 (2.0)%
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Infections
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1 (0.7)%
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2 (1.3)%
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0 (0.0)%
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Musculoskeletal Disorders
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1 (0.7)%
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0 (0.0)%
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2 (1.3)%
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Neoplasms
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3 (2.0)%
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3 (2.0)%
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1 (0.7)%
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Nervous System Disorders
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3 (2.0)%
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1 (0.7)%
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0 (0.0)%
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Respiratory Disorders
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0 (0.0)%
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3 (2.0)%
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2 (1.3)%
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Any Antiplatelet Trialists’ Collaboration (APTC) Event
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Non‑Fatal Myocardial Infarction
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0 (0.0)%
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0 (0.0)%
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0 (0.0)%
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Non‑Fatal Stroke
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2 (1.4)%
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1 (0.7)%
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0 (0.0)%
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Vascular Death
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1 (0.7)%
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0 (0.0)%
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0 (0.0)%
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(1)
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Data are listed only for system organ classes with three or more events.
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Monotherapy
Lucentis
N = 148
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0.3 mg Fovista +
Lucentis
N = 149
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1.5 mg Fovista +
Lucentis
N = 152
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Ocular Serious Adverse Events
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1 (0.7)%
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1 (0.7)%
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1 (0.7)%
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Corneal Erosion
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0 (0.0)%
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0 (0.0)%
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1 (0.7)%
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Uveitis
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0 (0.0)%
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1 (0.7)%
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0 (0.0)%
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Visual Acuity Reduced
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1 (0.7)%
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0 (0.0)%
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0 (0.0)%
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Monotherapy
Lucentis
N = 148
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0.3 mg Fovista +
Lucentis
N = 149
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1.5 mg Fovista +
Lucentis
N = 152
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Patients with One or More Adverse Events
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75 (50.7)%
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79 (53.0)%
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79 (52.0)%
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Conjunctival hemorrhage
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37 (25.0)%
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34 (22.8)%
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51 (33.6)%
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Punctate keratitis
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10 (6.8)%
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19 (12.8)%
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15 (9.9)%
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Eye pain
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8 (5.4)%
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10 (6.7)%
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13 (8.6)%
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Conjunctival hyperemia
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13 (8.8)%
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9 (6.0)%
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13 (8.6)%
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Subretinal fibrosis
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8 (5.4)%
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6 (4.0)%
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5 (3.3)%
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Intraocular pressure increase
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4 (2.7)%
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8 (5.4)%
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9 (5.9)%
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proportion of patients in each treatment group gaining 20 or more ETDRS letters from baseline at month 12;
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proportion of patients in each treatment group losing 5 or more ETDRS letters from baseline at month 12;
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proportion of patients in each treatment group achieving visual acuity of 20/25 or better at month 12; and
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proportion of patients with growth of choroidal neovascular lesion area from baseline to month 12 (as determined by fluorescein angiography).
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proportion of patients in each treatment group gaining 20 or more ETDRS letters from baseline at month 12;
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proportion of patients in each treatment group losing 5 or more ETDRS letters from baseline at month 12; and
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proportion of patients in each treatment group achieving visual acuity of 20/25 or better at month 12.
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In the first group, 310 and 312 patients, respectively, for OPH1002 and OPH1003 received intravitreal injections of 1.5 mg of Fovista following intravitreal injections of 0.5 mg of Lucentis.
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In the second group, which served as the control arm for each trial, 309 and 314 patients, respectively, for OPH1002 and OPH1003, received sham injections following intravitreal injections of 0.5 mg of Lucentis.
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1.5 mg of Fovista following intravitreal injections of 1.25 mg of Avastin; or
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1.5 mg of Fovista following intravitreal injections of 2.0 mg of Eylea.
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sham injections following intravitreal injections of 1.25 mg of Avastin; or
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sham injections following intravitreal injections of 2.0 mg of Eylea.
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OPH1002
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OPH1003
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OPH1002 + OPH1003
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1.5mg Fovista + 0.5mg Lucentis
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0.5mg Lucentis Monotherapy
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1.5mg Fovista + 0.5mg Lucentis
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0.5mg Lucentis Monotherapy
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1.5mg Fovista + 0.5mg Lucentis
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0.5mg Lucentis Monotherapy
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N = 310
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N = 309
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N = 312
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N = 314
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N = 622
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N = 623
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Patients With One or More Serious Systemic Adverse Event
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42 (13.55%)
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34 (11%)
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45 (14.42%)
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46 (14.65%)
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87 (13.99%)
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80 (12.84%)
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MedDRA System Organ Class (1)
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Infections and infestations
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11 (3.55%)
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10 (3.24%)
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9 (2.88%)
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8 (2.55%)
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20 (3.22%)
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18 (2.89%)
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Cardiac disorders
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11 (3.55%)
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5 (1.62%)
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5 (1.6%)
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9 (2.87%)
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16 (2.57%)
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14 (2.25%)
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Neoplasms benign, malignant and unspecified (incl. cysts and polyps)
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4 (1.29%)
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6 (1.94%)
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11 (3.53%)
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10 (3.18%)
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15 (2.41%)
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16 (2.57%)
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Injury, poisoning and procedural complications
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2 (0.65%)
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5 (1.62%)
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7 (2.24%)
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10 (3.18%)
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9 (1.45%)
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15 (2.41%)
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Respiratory, thoracic and mediastinal disorders
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5 (1.61%)
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3 (0.97%)
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4 (1.28%)
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2 (0.64%)
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9 (1.45%)
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5 (0.8%)
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Gastrointestinal disorders
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6 (1.94%)
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4 (1.29%)
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2 (0.64%)
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3 (0.96%)
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8 (1.29%)
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7 (1.12%)
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Nervous system disorders
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5 (1.61%)
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6 (1.94%)
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3 (0.96%)
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3 (0.96%)
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8 (1.29%)
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9 (1.44%)
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Vascular disorders
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0 (0%)
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6 (1.94%)
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3 (0.96%)
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1 (0.32%)
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3 (0.48%)
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7 (1.12%)
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OPH1002
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OPH1003
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OPH1002 + OPH1003
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1.5mg Fovista + 0.5mg Lucentis
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0.5mg Lucentis Monotherapy
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|
1.5mg Fovista + 0.5mg Lucentis
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|
0.5mg Lucentis Monotherapy
|
|
1.5mg Fovista + 0.5mg Lucentis
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|
0.5mg Lucentis Monotherapy
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N = 310
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N = 309
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N = 312
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N = 314
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N = 622
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N = 623
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Patients With One or More Arterial Thromboembolic Events
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Standardized MedDRA Query
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Myocardial Infarction
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3 (0.97%)
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3 (0.97%)
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0 (0%)
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4 (1.27%)
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3 (0.48%)
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7 (1.12%)
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Ischaemic CNS Vascular Conditions
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3 (0.97%)
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4 (1.29%)
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4 (1.28%)
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2 (0.64%)
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7 (1.13%)
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6 (0.96%)
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Haemorrhagic CNS Vascular Conditions
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2 (0.65%)
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2 (0.65%)
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1 (0.32%)
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1 (0.32%)
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3 (0.48%)
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3 (0.48%)
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|
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OPH1002
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OPH1003
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OPH1002 + OPH1003
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||||||
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1.5mg Fovista + 0.5mg Lucentis
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0.5mg Lucentis Monotherapy
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1.5mg Fovista + 0.5mg Lucentis
|
|
0.5mg Lucentis Monotherapy
|
|
1.5mg Fovista + 0.5mg Lucentis
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|
0.5mg Lucentis Monotherapy
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N = 310
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N = 309
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N = 312
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N = 314
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N = 622
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N = 623
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Patients With One or More Serious Ocular Adverse Event
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5 (1.61%)
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2 (0.65%)
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10 (3.21%)
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2 (0.64%)
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15 (2.41%)
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4 (0.64%)
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Eye Disorders
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3 (0.97%)
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2 (0.65%)
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5 (1.6%)
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1 (0.32%)
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8 (1.29%)
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3 (0.48%)
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Cataract
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1 (0.32%)
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0 (0%)
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0 (0%)
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0 (0%)
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1 (0.16%)
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0 (0%)
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Retinal detachment
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0 (0%)
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0 (0%)
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3 (0.96%)
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0 (0%)
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3 (0.48%)
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0 (0%)
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Macular hole
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0 (0%)
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1 (0.32%)
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2 (0.64%)
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0 (0%)
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2 (0.32%)
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1 (0.16%)
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Cataract Subcapsular
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1 (0.32%)
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0 (0%)
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0 (0%)
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0 (0%)
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1 (0.16%)
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0 (0%)
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Retinal pigment epithelial tear
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0 (0%)
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0 (0%)
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1 (0.32%)
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0 (0%)
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1 (0.16%)
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0 (0%)
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Retinal tear
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1 (0.32%)
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0 (0%)
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0 (0%)
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0 (0%)
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1 (0.16%)
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0 (0%)
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Vitreous hemorrhage
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1 (0.32%)
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0 (0%)
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0 (0%)
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1 (0.32%)
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1 (0.16%)
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|
1 (0.16%)
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Visual acuity reduced
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0 (0%)
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1 (0.32%)
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0 (0%)
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0 (0%)
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0 (0%)
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1 (0.16%)
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Infections and Infestations
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|
2 (0.65%)
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|
0 (0%)
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6 (1.92%)
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|
1 (0.32%)
|
|
8 (1.29%)
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|
1 (0.16%)
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Endophthalmitis
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|
2 (0.65%)
|
|
0 (0%)
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|
6 (1.92%)
|
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1 (0.32%)
|
|
8 (1.29%)
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|
1 (0.16%)
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Injury, Poisoning and Procedural Complications
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|
0 (0%)
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|
0 (0%)
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1 (0.32%)
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|
0 (0%)
|
|
1 (0.16%)
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|
0 (0%)
|
Cataract traumatic
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|
0 (0%)
|
|
0 (0%)
|
|
1 (0.32%)
|
|
0 (0%)
|
|
1 (0.16%)
|
|
0 (0%)
|
|
|
OPH1002
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|
OPH1003
|
|
OPH1002 + OPH1003
|
||||||
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1.5mg Fovista + 0.5mg Lucentis
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|
0.5mg Lucentis Monotherapy
|
|
1.5mg Fovista + 0.5mg Lucentis
|
|
0.5mg Lucentis Monotherapy
|
|
1.5mg Fovista + 0.5mg Lucentis
|
|
0.5mg Lucentis Monotherapy
|
|
|
N = 310
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|
N = 309
|
|
N = 312
|
|
N = 314
|
|
N = 622
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|
N = 623
|
|
Patients With One or More Ocular Adverse Event (1)
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|
154 (49.68%)
|
|
140 (45.31%)
|
|
199 (63.78%)
|
|
162 (51.59%)
|
|
353 (56.75%)
|
|
302 (48.48%)
|
Eye Disorders
|
|
129 (41.61%)
|
|
121 (39.16%)
|
|
175 (56.09%)
|
|
150 (47.77%)
|
|
304 (48.87%)
|
|
271 (43.5%)
|
Conjunctival haemorrhage
|
|
60 (19.35%)
|
|
56 (18.12%)
|
|
74 (23.72%)
|
|
50 (15.92%)
|
|
134 (21.54%)
|
|
106 (17.01%)
|
Punctate keratitis
|
|
15 (4.84%)
|
|
18 (5.83%)
|
|
35 (11.22%)
|
|
40 (12.74%)
|
|
50 (8.04%)
|
|
58 (9.31%)
|
Eye pain
|
|
23 (7.42%)
|
|
26 (8.41%)
|
|
22 (7.05%)
|
|
21 (6.69%)
|
|
45 (7.23%)
|
|
47 (7.54%)
|
Conjunctival hyperaemia
|
|
15 (4.84%)
|
|
13 (4.21%)
|
|
24 (7.69%)
|
|
24 (7.64%)
|
|
39 (6.27%)
|
|
37 (5.94%)
|
Vitreous floaters
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|
12 (3.87%)
|
|
6 (1.94%)
|
|
22 (7.05%)
|
|
12 (3.82%)
|
|
34 (5.47%)
|
|
18 (2.89%)
|
Eye irritation
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|
5 (1.61%)
|
|
12 (3.88%)
|
|
18 (5.77%)
|
|
15 (4.78%)
|
|
23 (3.7%)
|
|
27 (4.33%)
|
Keratitis
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|
2 (0.65%)
|
|
4 (1.29%)
|
|
18 (5.77%)
|
|
14 (4.46%)
|
|
20 (3.22%)
|
|
18 (2.89%)
|
Investigations
|
|
43 (13.87%)
|
|
30 (9.71%)
|
|
49 (15.71%)
|
|
25 (7.96%)
|
|
92 (14.79%)
|
|
55 (8.83%)
|
Intraocular pressure increased
|
|
43 (13.87%)
|
|
30 (9.71%)
|
|
49 (15.71%)
|
|
25 (7.96%)
|
|
92 (14.79%)
|
|
55 (8.83%)
|
•
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OPH1005 Fovista Anti-Fibrosis Study
. During the third quarter of 2014, we initiated an open-label Phase 2a clinical trial of 1.5 mg of Fovista administered in combination with either Lucentis, Eylea or Avastin, to study subretinal fibrosis in wet AMD patients. The trial did not have any pre-specified efficacy endpoints as it was uncontrolled, open-label Phase 2a study with broad patient eligibility criteria. We completed enrollment in this trial in May 2015 with a total of 101 patients enrolled. Patients were followed for up to a 24-month period prior to our determination in December 2016 to stop treating patients in the Fovista Expansion Studies.
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OPH1006 Fovista Treatment Burden Reduction Study
. During the fourth quarter of 2014, we initiated an open-label Phase 2a clinical trial of 1.5 mg of Fovista administered in combination with anti-VEGF drugs (Lucentis, Eylea or Avastin) to investigate a different treatment regimen of Fovista combination therapy. We completed enrollment in this trial in October 2015 with a total of 64 patients enrolled. Patients in this trial were to be followed for up to a 24-month period prior to our determination in December 2016 to stop treating patients in the Fovista Expansion Studies.
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OPH1007
Fovista in Combination with Avastin Discontinuous Regimen Study.
During the fourth quarter of 2015, we initiated a randomized, double-masked, controlled Phase 2b clinical trial to evaluate the safety and efficacy of a discontinuous, bimonthly regimen of 1.5 mg of Fovista administered in combination with Avastin during the maintenance phase of wet AMD treatment, compared to a discontinuous, bimonthly regimen of Avastin monotherapy. This trial was terminated prior to full enrollment.
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OPH1008 Fovista Imaging Study
. During the fourth quarter of 2015, we initiated an open-label Phase 2a clinical trial to investigate the role of multi-modal imaging in assessing anatomic responses to various wet AMD treatment regimens of 1.5 mg of Fovista administered in combination with anti-VEGF drugs (Lucentis, Eylea or Avastin). This trial was terminated prior to full enrollment.
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Geographic Atrophy.
With severe and progressive macular degeneration, a readily identifiable pattern of severe degeneration called GA, forms, which consequently leads to profound and irreversible vision loss. GA is readily diagnosed by macular visualization using standard diagnostic instruments utilized by ophthalmologists. GA appears as abrupt and deep levels of macular tissue loss. It has sharp margins of characteristic degeneration compared to surrounding macular tissue, resulting in progressive and chronic degeneration of the retina characterized by variable thinning and dysfunction of retinal tissue.
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Conversion to Wet AMD.
Dry AMD progresses to the wet form of the disease in approximately 10% ‑ 15% of patients, leading to more rapid and further visual loss.
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patents and patent applications owned by Ophthotech:
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patents covering the treatment of wet AMD with a combination of Fovista and an anti‑VEGF‑A antibody or binding fragment thereof (such as Avastin or Lucentis), or the use of Fovista in the manufacture of a medicine for the treatment of wet AMD when administered with an anti‑VEGF‑A antibody or binding fragment thereof, which have issued in the United States, the European Union, Japan and certain other jurisdictions, and which are expected to expire in 2024; and patent applications covering the treatment of wet AMD with a combination of Fovista and an anti‑VEGF‑A antibody or binding fragment thereof or the use of Fovista in the manufacture of a medicine for the treatment of wet
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patent applications covering the treatment of wet AMD with a combination of Fovista and Eylea, or the use of Fovista in the manufacture of a medicine for the treatment of wet AMD when administered with Eylea, which are pending in the United States, the European Union, Japan and certain other jurisdictions, and which, if granted, are expected to expire in 2030;
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patents covering co‑formulations of Fovista and an anti‑VEGF‑A antibody or binding fragment thereof, which have issued in the United States, Japan and certain other jurisdictions, and which are expected to expire in the United States in 2025 and elsewhere in 2024; and patent applications covering co‑formulations of Fovista and an anti‑VEGF‑A antibody or binding fragment thereof, which are pending in the European Union and certain other jurisdictions, and which, if granted, are expected to expire in 2024;
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patents covering methods for treating AMD with a combination of Fovista and Macugen, which have issued in the United States, the European Union, Japan and certain other jurisdictions, and which are expected to expire in 2024; and patent applications covering methods for treating AMD with a combination of Fovista and Macugen, which are pending in certain other jurisdictions, and which, if granted, are expected to expire in 2024;
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patent applications covering co‑formulations and other proprietary technology relating to Fovista, which are pending in the United States, the European Union, Japan and certain other jurisdictions, and which, if granted, are expected to expire in 2033;
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patent applications covering formulations and dosing regimens and other proprietary technology relating to Fovista, which are pending in the United States, the European Union, Japan and certain other jurisdictions, and which, if granted, are expected to expire in 2034;
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patent applications covering co‑formulations and other proprietary technology relating to Zimura, which are pending in the United States, the European Union, Japan and certain other jurisdictions, and which, if granted, are expected to expire in 2034; and
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patent applications covering dosing regimens and methods for treating AMD and other proprietary technology relating to Fovista, which are pending in the United States and under the Patent Cooperation Treaty system, and which, if granted, are expected to expire in 2035; and
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patents and patent applications in‑licensed from Archemix Corp., or Archemix:
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composition‑of‑matter patents covering Fovista, which have issued in the European Union, Japan and certain other jurisdictions, and which are expected to expire in 2018;
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composition‑of‑matter patents covering Zimura, which have issued in the United States, the European Union, Japan and certain other jurisdictions, and which are expected to expire in Japan in 2026 and elsewhere in 2025; and composition‑of‑matter patent applications covering Zimura, which are pending in certain other jurisdictions, and which, if granted, are expected to expire in 2025; and
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patents covering the treatment of certain complement mediated disorders with Zimura, Zimura for use in a method of treating certain complement mediated disorders or a composition comprising Zimura for treating certain complement mediated disorders, which have issued in the United States, the European Union, Japan and certain other jurisdictions, and which are expected to expire in Japan and the United States in 2026 and elsewhere in 2025.
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an exclusive, worldwide license under specified patent rights and know‑how owned or controlled by Nektar to make, have made, develop, use, import, offer for sale and sell particular products that are produced by linking the API in Fovista to a specified polyethylene glycol, or PEG, reagent by means of pegylation; and
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non‑exclusive sublicenses of certain other patent rights controlled by Nektar.
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completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA’s good laboratory practice, or GLP, regulations;
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submission to the FDA of an investigational new drug application, or IND, which must take effect before human clinical trials may begin;
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approval by an independent institutional review board, or IRB, representing each clinical site before each clinical trial may be initiated;
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performance of adequate and well‑controlled human clinical trials in accordance with good clinical practices, or GCP, to establish the safety and efficacy of the proposed drug product for each indication;
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preparation and submission to the FDA of an NDA requesting marketing for one or more proposed indications;
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review by an FDA advisory committee, where appropriate or if applicable;
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satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the product, or components thereof, are produced to assess compliance with current Good Manufacturing Practices, or cGMP, requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s identity, strength, quality and purity;
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satisfactory completion of FDA audits of clinical trial sites to assure compliance with GCPs and the integrity of the clinical data;
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payment of user fees and securing FDA approval of the NDA; and
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compliance with any post‑approval requirements, including the potential requirement to implement a Risk Evaluation and Mitigation Strategy, or REMS, and the potential requirement to conduct post‑approval studies.
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Phase 1.
The drug is initially introduced into a small number of healthy human subjects or, in certain indications, patients with the target disease or condition and tested for safety, dosage tolerance, absorption, metabolism,
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Phase 2.
The drug is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage.
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Phase 3.
These clinical trials are commonly referred to as “pivotal” studies, which denotes a study that presents the data that the FDA or other relevant regulatory agency will use to determine whether or not to approve a drug. The drug is administered to an expanded patient population, generally at geographically dispersed clinical trial sites, in well‑controlled clinical trials to generate enough data to statistically evaluate the efficacy and safety of the product for approval, identify adverse effects, establish the overall risk‑benefit profile of the product and to provide adequate information for the labeling of the product.
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Phase 4.
Post‑approval studies may be required to be conducted after initial marketing approval. These studies are used to gain additional experience from the treatment of patients in the intended therapeutic indication.
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restrictions on the marketing or manufacturing of the product, suspension of the approval, or complete withdrawal of the product from the market or product recalls;
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fines, warning letters or holds on post‑approval clinical trials;
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refusal of the FDA to approve pending NDAs or supplements to approved NDAs, or suspension or revocation of product license approvals;
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product seizure or detention, or refusal to permit the import or export of products; or
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injunctions or the imposition of civil or criminal penalties.
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the required patent information has not been filed;
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the listed patent has expired;
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the listed patent has not expired, but will expire on a particular date and approval is sought after patent expiration; or
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the listed patent is invalid, unenforceable or will not be infringed by the new product.
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the federal Anti‑Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, paying, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid;
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the federal civil and criminal false claims laws, including the civil False Claims Act, and civil monetary penalties laws, which prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false, fictitious or fraudulent or knowingly making, using or causing to made or used a false record or statement to avoid, decrease or conceal an obligation to pay money to the federal government;
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the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created additional federal criminal laws that prohibit, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;
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HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, and their respective implementing regulations, including the Final Omnibus Rule published in January 2013, which impose obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
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the federal false statements statute, which prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for healthcare benefits, items or services;
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the federal transparency requirements known as the federal Physician Payments Sunshine Act, under the Patient Protection and Affordable Care Act, as amended by the Health Care Education Reconciliation Act, or the Affordable Care Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies to report annually to the Centers for Medicare & Medicaid Services, or CMS, within the United States Department of Health and Human Services, information related to payments and other transfers of value made by that entity to physicians and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members; and
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analogous state and foreign laws and regulations, such as state anti‑kickback and false claims laws, which may apply to healthcare items or services that are reimbursed by non‑governmental third‑party payors, including private insurers.
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an annual, nondeductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government healthcare programs, although this fee would not apply to sales of certain products approved exclusively for orphan indications;
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expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to certain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing a manufacturer’s Medicaid rebate liability;
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expanded manufacturers’ rebate liability under the Medicaid Drug Rebate Program by increasing the minimum rebate for both branded and generic drugs and revising the definition of “average manufacturer price,” or AMP, for calculating and reporting Medicaid drug rebates on outpatient prescription drug prices and extending rebate liability to prescriptions for individuals enrolled in Medicare Advantage plans;
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addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;
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expanded the types of entities eligible for the 340B drug discount program;
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established the Medicare Part D coverage gap discount program by requiring manufacturers to provide a 50% point‑of‑sale‑discount off the negotiated price of applicable brand drugs to eligible beneficiaries during their coverage gap period as a condition for the manufacturers’ outpatient drugs to be covered under Medicare Part D;
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established a new Patient‑Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research;
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established the Independent Payment Advisory Board, or IPAB, which has authority to recommend certain changes to the Medicare program to reduce expenditures by the program that could result in reduced payments for prescription drugs. However, the IPAB implementation has been not been clearly defined. The Affordable Care Act provided that under certain circumstances, IPAB recommendations will become law unless Congress enacts legislation that will achieve the same or greater Medicare cost savings; and
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established the Center for Medicare and Medicaid Innovation within CMS to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending. Funding has been allocated to support the mission of the Center for Medicare and Medicaid Innovation from 2011 to 2019.
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exposure to known and unknown liabilities, including possible intellectual property infringement claims, violations of laws, tax liabilities and commercial disputes;
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incurrence of substantial debt, dilutive issuances of securities or depletion of cash to pay for acquisitions;
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higher than expected acquisition and integration costs;
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difficulty in combining the operations and personnel of any acquired businesses with our operations and personnel;
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inability to maintain uniform standards, controls, procedures and policies;
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restructuring charges related to eliminating redundancies or disposing of assets as part of any such combination;
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large write-offs and difficulties in assessing the relative percentages of in-process research and development expense that can be immediately written off as compare to the amount that must be amortized over the appropriate life of the asset;
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increased amortization expenses or, in the event that we write-down the value of acquired assets, impairment losses;
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impairment of relationships with key suppliers or customers of any acquired businesses due to changes in management and ownership;
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inability to retain personnel, key customers, distributors, vendors and other business partners integral to an in-licensed or acquired product candidate or technology;
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potential failure of the due diligence processes to identify significant problems, liabilities or other shortcomings or challenges of an acquired or licensed product candidate or technology, including, without limitation, problems, liabilities or other shortcomings or challenges with respect to intellectual property, product quality, revenue recognition or other accounting practices, partner disputes or issues and other legal and financial contingencies and known and unknown liabilities; and
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entry into indications or markets in which we have no or limited direct prior development or commercial experience and where competitors in such markets have stronger market positions.
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in‑license or acquire the rights to, and pursue the development of, other products, product candidates or technologies;
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complete the activities necessary to receive initial, top-line data from OPH1004 and wind down OPH1002, OPH1003 and the Fovista Expansion Studies and our Fovista contract manufacturing commitments;
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potentially undertake additional clinical development of Fovista in wet AMD if the initial, top-line data from OPH1004 is favorable or in other indications if we believe there is a sufficient scientific rationale to pursue such development;
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continue the clinical development of Zimura for the treatment of GA and in combination with anti-VEGF drugs for the treatment of wet AMD, or potentially in other indications if we believe there is a sufficient scientific rationale to pursue such development;
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complete our previously announced reduction in personnel;
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maintain, expand and protect our intellectual property portfolio;
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hire additional clinical, manufacturing, quality control, quality assurance and scientific personnel if we are successful in progressing the clinical development of any of our product candidates;
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seek marketing approval for any product candidates that successfully complete clinical trials; and
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expand our outsourced manufacturing activities, expand our commercial operations and establish sales, marketing and distribution capabilities, if we receive, or expect to receive, marketing approval for any of our product candidates.
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the extent to which we in‑license or acquire rights to, and undertake development of products, product candidates or technologies;
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the amount of any upfront, milestone payments and other financial obligations associated with the in-license or acquisition of other product candidates;
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the scope, progress, results and costs of preclinical development and/or clinical trials for any other product candidates that we may acquire or in-license and develop;
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the scope, progress, costs and results of the OPH1004 trial and any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Fovista in wet AMD or any other indication;
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the scope, costs and results of our Zimura clinical programs, including our Zimura Phase 2/3 GA trial and our Zimura Phase 2a wet AMD trial, as well as any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Zimura in any indication (including a potential second Phase 3 trial for GA);
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if OPH1004 is positive, the costs and timing of restarting the manufacturing of commercial supply for Fovista;
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the costs and timing of process development and manufacturing scale‑up and validation activities associated with Zimura;
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our ability to establish collaborations on favorable terms, if at all;
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the costs, timing and outcome of regulatory reviews of our product candidates;
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the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending intellectual property‑related claims;
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the timing, scope and cost of commercialization activities for any of our product candidates if we receive, or expect to receive, marketing approval for a product candidate; and
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subject to receipt of marketing approval, net revenue received from commercial sales of any of our product candidates, after milestone payments and royalty payments that we would be obligated to make.
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designing, conducting and completing clinical trials for our product candidates;
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obtaining favorable results from required clinical trials, including for each ophthalmic product candidate, favorable results from two adequate and well controlled pivotal clinical trials in the relevant indication;
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applying for and receiving marketing approvals from applicable regulatory authorities for the use of our product candidates;
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making arrangements with third‑party manufacturers, receiving regulatory approval of our manufacturing processes and our third‑party manufacturers’ facilities from applicable regulatory authorities and ensuring adequate supply of drug product;
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establishing sales, marketing and distribution capabilities, either internally or through collaborations or other arrangements, to effectively market and sell our product candidates;
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achieving acceptance of the product candidate, if and when approved, by patients, the medical community and third‑party payors;
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if our product candidates are approved, obtaining from governmental and third‑party payors adequate coverage and reimbursement for our product candidates and, to the extent applicable, associated injection procedures conducted by treating physicians;
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effectively competing with other therapies, including the existing standard of care, and other forms of drug delivery;
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maintaining a continued acceptable safety profile of the product candidate during development and following approval;
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obtaining and maintaining patent and trade secret protection and regulatory exclusivity;
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protecting and enforcing our rights in our intellectual property portfolio; and
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complying with all applicable regulatory requirements, including FDA Good Clinical Practices, or GCP, Good Manufacturing Practices, or GMP, and standards, rules and regulations governing promotional and other marketing activities.
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we may not be able to generate sufficient preclinical, toxicology, or other
in vivo
or
in vitro
data to support the initiation of clinical studies for any preclinical product candidates that we in-license or acquire;
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regulators or institutional review boards may not agree with our study design or may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site;
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we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective clinical research organizations or clinical trial sites;
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our contract research organizations or clinical trial sites may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;
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we, through our clinical trial sites, may not be able to locate and enroll a sufficient number of eligible patients to participate in our clinical trials as required by the FDA or similar regulatory authorities outside the United States;
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we may decide, or regulators or institutional review boards may require us, to suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements, including GCPs, or a finding that the participants are being exposed to unacceptable health risks;
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there may be changes in regulatory requirements and guidance or we may have changes in trial design that require amending or submitting new clinical protocols;
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there may be changes in the standard of care on which a clinical development plan was based, which may require new or additional trials;
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clinical trials of our product candidates may produce inconclusive or negative results, such as the results we observed in our pivotal OPH1002 and OPH1003 Fovista trials for the treatment of wet AMD, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs;
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the number of patients required for clinical trials of our product candidates to demonstrate statistically significant results may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate or participants may drop out of these clinical trials at a higher rate than we anticipate;
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the cost of clinical trials of our product candidates may be greater than we anticipate; and
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the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates, such as the anti‑VEGF drugs we need to use in combination with Fovista and Zimura in our wet AMD trials, may become insufficient or inadequate or we may face delays in the manufacture and supply of such materials as a result our decision to transfer manufacturing between contract manufacturers or on account of interruptions in our supply chain, including in relation to the packaging and distribution or import / export of clinical materials.
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be delayed in obtaining marketing approval for our product candidates;
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not obtain marketing approval at all;
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obtain approval for indications or patient populations that are not as broad as intended or desired;
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obtain approval with labeling that includes significant use limitations, distribution restrictions or safety warnings, including boxed warnings;
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be subject to additional post-marketing testing requirements; or
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have the product removed from the market after obtaining marketing approval.
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efficacy and potential advantages compared to alternative treatments, including the existing standard of care;
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any restrictions in the label on the use of our products in combination with other medications;
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any restrictions in the label on the use of our products by a subgroup of patients;
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restrictions in the label on of any for our combination therapy product candidates, such as Fovista or Zimura, limiting their use in combination with particular standard of care drugs, such as a particular anti‑VEGF drug;
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our and any commercialization partner’s ability to offer our products at competitive prices, particularly in light of the cost of any of our combination therapy product candidates in addition to the cost of the underlying standard of care drug;
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availability of third‑party coverage and adequate reimbursement, particularly by Medicare given the target market for AMD indications for persons over age 55;
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increasing reimbursement pressures on treating physicians due to the formation of accountable care organizations and the shift away from traditional fee‑for‑service reimbursement models to reimbursement based on quality of care and patient outcomes;
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willingness of the target patient population to try new therapies and of physicians to prescribe these therapies, particularly in light of the existing available standard of care;
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prevalence and severity of any side effects; and
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whether competing products or other alternatives are more convenient or easier to administer, including whether co‑formulated alternatives, alternatives that can be co‑administered in a single syringe or alternatives that offer a less invasive method of administration than intravitreal injection come to market.
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our inability to recruit and retain adequate numbers of effective sales and marketing personnel;
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the inability of sales personnel to obtain access to adequate numbers of physicians who may prescribe our products;
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the lack of complementary products to be offered by our sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and
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unforeseen costs and expenses associated with creating an independent sales and marketing organization.
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decreased demand for any product candidates or products that we may develop or in‑license;
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injury to our reputation and significant negative media attention;
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withdrawal of clinical trial participants;
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significant costs to defend the related litigation;
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substantial monetary awards to trial participants or patients;
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loss of revenue;
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reduced time and attention of our management to pursue our business strategy; and
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the inability to commercialize any products that we may develop or in‑license.
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collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations and may not perform their obligations as expected;
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collaborators may deemphasize or not pursue development and commercialization of our product candidates or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus, changes in product candidate priorities or available funding or changes in priorities as a result of a merger, acquisition or other corporate restructuring or transaction;
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collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;
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collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;
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we could grant exclusive rights to our collaborators, which would prevent us from collaborating with others;
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disagreements or disputes with collaborators, including disagreements or disputes over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of products or product candidates, might lead to additional responsibilities for us with respect to product candidates or might result in litigation or arbitration, any of which would divert management attention and resources, be time‑consuming and be expensive;
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collaborators with marketing and distribution rights to one or more products may not commit sufficient resources to the marketing and distribution of such product or products;
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collaborators may not properly maintain or defend our intellectual property rights, may infringe the intellectual property rights of third parties, may misappropriate our trade secrets or may use our proprietary information in
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collaborations may be terminated for the convenience of the collaborator, our breach of the terms of the collaboration or other reasons and, if terminated, we may need to raise additional capital to pursue further development or commercialization of the applicable product candidates.
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our product candidates may compete with other product candidates and products for access to a limited number of suitable manufacturing facilities that operate under current good manufacturing practices, or cGMP, regulations;
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reliance on the third party for regulatory compliance, quality assurance and quality control;
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the possible breach of the manufacturing agreement by the third party;
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the possible breach of our supply obligations to Novartis;
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the possible misappropriation of our proprietary information, including our trade secrets and know‑how; and
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the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us.
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Under the terms of the agreement with OSI Pharmaceuticals under which we acquired certain rights to develop and commercialize Fovista, if we or our commercialization or collaborative partners fail to meet certain obligations, OSI Pharmaceuticals may terminate the agreement as to such countries with respect to which such failure has occurred, and upon such termination we will be obligated to grant, assign and transfer to OSI Pharmaceuticals specified rights and licenses related to our anti‑PDGF aptamer technology and other related assets, and if we are manufacturing such anti‑PDGF products at the time of such termination, may be obligated to provide transitional supply to OSI Pharmaceuticals of covered anti‑PDGF products, for such countries.
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•
|
Under the terms of the amended license, manufacturing and supply agreement with Nektar, pursuant to which we obtained, among other licenses, an exclusive, worldwide license to make, develop, use, import, offer for sale and sell certain products that incorporate a specified PEG reagent linked with the active ingredient in Fovista, if we fail to use commercially reasonable efforts to achieve the first commercial sale of Fovista in the United States by June 30, 2018, we and Nektar may agree in good faith to extend such date in specified circumstances. If such date is not extended, Nektar may either terminate our license or convert our license for such country to a non‑exclusive license. In addition, if we fail to use commercially reasonable efforts to develop Fovista and file and seek approval of new drug applications on a schedule permitting us to make first commercial sales of Fovista in specified countries by June 30, 2019, do not make such first commercial sales of Fovista by such date, or thereafter fail to use commercially reasonable efforts to continue to commercialize and market Fovista in such countries, we would be in material breach of the agreement and Nektar would have the right to terminate the agreement.
|
•
|
restrictions on such products, manufacturers or manufacturing processes;
|
•
|
restrictions on the labeling or marketing of a product;
|
•
|
restrictions on distribution or use of a product;
|
•
|
requirements to conduct post‑marketing studies or clinical trials;
|
•
|
warning letters or untitled letters;
|
•
|
withdrawal of the products from the market;
|
•
|
refusal to approve pending applications or supplements to approved applications that we submit;
|
•
|
recall of products;
|
•
|
damage to relationships with any potential collaborators;
|
•
|
unfavorable press coverage and damage to our reputation;
|
•
|
fines, restitution or disgorgement of profits or revenues;
|
•
|
suspension or withdrawal of marketing approvals;
|
•
|
refusal to permit the import or export of our products;
|
•
|
product seizure;
|
•
|
injunctions or the imposition of civil or criminal penalties; and
|
•
|
litigation involving patients using our products.
|
•
|
the federal Anti‑Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation or arranging of, any good or service, for which payment may be made under a federal healthcare program such as Medicare and Medicaid;
|
•
|
the federal False Claims Act imposes criminal and civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to be presented, false or fraudulent claims for payment by a federal healthcare program or making a false statement or record material to payment of a false claim or avoiding, decreasing or concealing an obligation to pay money to the federal government, with potential liability including mandatory treble damages and significant per‑claim penalties, currently set at $5,500 to $11,000 per false claim;
|
•
|
the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;
|
•
|
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and its implementing regulations, also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
|
•
|
the federal Physician Payments Sunshine Act requires applicable manufacturers of covered drugs to report payments and other transfers of value to physicians and teaching hospitals, with data collection beginning in August 2013; and
|
•
|
analogous state and foreign laws and regulations, such as state anti‑kickback and false claims laws and transparency statutes, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non‑governmental third-party payors, including private insurers.
|
•
|
an annual, non‑deductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic agents;
|
•
|
an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program;
|
•
|
a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;
|
•
|
expansion of healthcare fraud and abuse laws, including the civil False Claims Act and the federal Anti‑Kickback Statute, new government investigative powers and enhanced penalties for noncompliance;
|
•
|
a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% point‑of‑sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D;
|
•
|
extension of manufacturers’ Medicaid rebate liability to individuals enrolled in Medicaid managed care organizations;
|
•
|
expansion of eligibility criteria for Medicaid programs;
|
•
|
expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program;
|
•
|
new requirements to report certain financial arrangements with physicians and teaching hospitals;
|
•
|
a new requirement to annually report drug samples that manufacturers and distributors provide to physicians;
|
•
|
a new Patient‑Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research;
|
•
|
a new Independent Payment Advisory Board, or IPAB, which has authority to recommend certain changes to the Medicare program to reduce expenditures by the program that could result in reduced payments for prescription drugs; and
|
•
|
establishment of the Center for Medicare and Medicaid Innovation within CMS to test innovative payment and service delivery models.
|
•
|
provide for a classified board of directors such that only one of three classes of directors is elected each year;
|
•
|
allow the authorized number of our directors to be changed only by resolution of our board of directors;
|
•
|
limit the manner in which stockholders can remove directors from the board of directors;
|
•
|
provide for advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors;
|
•
|
require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent;
|
•
|
limit who may call stockholder meetings;
|
•
|
authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a “poison pill” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and
|
•
|
require the approval of the holders of at least 75% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our certificate of incorporation or by‑laws.
|
•
|
the success of products or technologies that compete with our product candidates, including results of clinical trials of product candidates of our competitors;
|
•
|
results of clinical trials for our product candidates and the timing of the receipt of such results;
|
•
|
the results of our efforts to in‑license or acquire the rights to other products, product candidates and technologies for the treatment of ophthalmic diseases;
|
•
|
political, regulatory or legal developments in the United States and other countries;
|
•
|
developments or disputes concerning patent applications, issued patents or other proprietary rights;
|
•
|
the recruitment or departure of key personnel;
|
•
|
the level of expenses related to any of our product candidates or clinical development programs;
|
•
|
actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;
|
•
|
variations in our financial results or those of companies that are perceived to be similar to us;
|
•
|
changes in the structure of healthcare payment systems;
|
•
|
market conditions in the pharmaceutical and biotechnology sectors;
|
•
|
general economic, industry and market conditions; and
|
•
|
the other factors described in this “Risk Factors” section.
|
|
|
Year ended December 31, 2016
|
|
Year ended December 31, 2015
|
||||||||||||
|
|
High
|
|
Low
|
|
High
|
|
Low
|
||||||||
Quarter ended March 31,
|
|
$
|
74.07
|
|
|
$
|
40.40
|
|
|
$
|
58.29
|
|
|
$
|
44.30
|
|
Quarter ended June 30,
|
|
$
|
58.45
|
|
|
$
|
41.84
|
|
|
$
|
53.17
|
|
|
$
|
44.55
|
|
Quarter ended September 30,
|
|
$
|
65.03
|
|
|
$
|
46.13
|
|
|
$
|
72.51
|
|
|
$
|
35.72
|
|
Quarter ended December 31,
|
|
$
|
45.30
|
|
|
$
|
4.82
|
|
|
$
|
80.00
|
|
|
$
|
37.45
|
|
|
|
|
9/25/2013
|
|
9/30/2013
|
|
12/31/2013
|
|
3/31/2014
|
|
6/30/2014
|
|
9/30/2014
|
|
12/31/2014
|
|
3/31/2015
|
||||||||||||||||
Ophthotech Corporation
|
|
$
|
100.00
|
|
|
$
|
135.05
|
|
|
$
|
147.05
|
|
|
$
|
162.16
|
|
|
$
|
192.32
|
|
|
$
|
176.95
|
|
|
$
|
203.95
|
|
|
$
|
211.50
|
|
NASDAQ Composite
|
|
100.00
|
|
|
105.46
|
|
|
117.13
|
|
|
118.23
|
|
|
124.11
|
|
|
126.29
|
|
|
133.19
|
|
|
137.78
|
|
||||||||
NASDAQ Biotechnology
|
|
100.00
|
|
|
108.52
|
|
|
118.51
|
|
|
122.37
|
|
|
133.81
|
|
|
147.15
|
|
|
156.52
|
|
|
171.61
|
|
|
|
6/30/2015
|
|
9/30/2015
|
|
12/31/2015
|
|
3/31/2016
|
|
6/30/2016
|
|
9/30/2016
|
|
12/31/2016
|
||||||||||||||
Ophthotech Corporation
|
|
$
|
236.64
|
|
|
$
|
184.18
|
|
|
$
|
356.95
|
|
|
$
|
192.14
|
|
|
$
|
231.95
|
|
|
$
|
209.68
|
|
|
$
|
21.95
|
|
NASDAQ Composite
|
|
140.65
|
|
|
130.11
|
|
|
141.32
|
|
|
138.82
|
|
|
138.21
|
|
|
151.71
|
|
|
153.66
|
|
|||||||
NASDAQ Biotechnology
|
|
181.93
|
|
|
149.90
|
|
|
166.10
|
|
|
135.14
|
|
|
132.26
|
|
|
145.41
|
|
|
134.22
|
|
•
|
approximately
$136.9 million
to fund certain costs of our Phase 3 clinical program for Fovista administered in combination with anti-VEGF drugs for the treatment of wet AMD, which costs consists of external research and development expenses and clinical development related employee expenses; and
|
•
|
approximately
$38.7 million
for working capital and other general corporate purposes.
|
|
Years ended December 31,
|
||||||||||||||||||
|
2016
|
|
2015
|
|
2014
|
|
2013
|
|
2012
|
||||||||||
|
(in thousands, except per share data)
|
||||||||||||||||||
Statements of Operations Data:
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||
Collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
||||||||||
Research and development
|
196,295
|
|
|
131,012
|
|
|
88,385
|
|
|
33,215
|
|
|
6,792
|
|
|||||
General and administrative
|
50,178
|
|
|
44,021
|
|
|
33,387
|
|
|
14,210
|
|
|
6,889
|
|
|||||
Total operating expenses
|
246,473
|
|
|
175,033
|
|
|
121,772
|
|
|
47,425
|
|
|
13,681
|
|
|||||
Loss from operations
|
(195,564
|
)
|
|
(123,528
|
)
|
|
(80,513
|
)
|
|
(47,425
|
)
|
|
(13,681
|
)
|
|||||
Interest income (expense)
|
1,704
|
|
|
971
|
|
|
217
|
|
|
(1,454
|
)
|
|
(507
|
)
|
|||||
Loss on extinguishment of debt
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,091
|
)
|
|
—
|
|
|||||
Other income (expense)
|
34
|
|
|
53
|
|
|
—
|
|
|
(1,175
|
)
|
|
(374
|
)
|
|||||
Loss before income tax (benefit) provision
|
(193,826
|
)
|
|
(122,504
|
)
|
|
(80,296
|
)
|
|
(51,145
|
)
|
|
(14,562
|
)
|
|||||
Income tax (benefit) provision
|
(406
|
)
|
|
(16,787
|
)
|
|
36,476
|
|
|
—
|
|
|
—
|
|
|||||
Net loss
|
(193,420
|
)
|
|
(105,717
|
)
|
|
(116,772
|
)
|
|
(51,145
|
)
|
|
(14,562
|
)
|
|||||
Add: accretion of preferred stock dividends
|
—
|
|
|
—
|
|
|
—
|
|
|
(5,891
|
)
|
|
(7,063
|
)
|
|||||
Net loss attributable to common stockholders
|
$
|
(193,420
|
)
|
|
$
|
(105,717
|
)
|
|
$
|
(116,772
|
)
|
|
$
|
(57,036
|
)
|
|
$
|
(21,625
|
)
|
Net loss per common share:
|
|
|
|
|
|
|
|
|
|
||||||||||
Basic and diluted
|
$
|
(5.45
|
)
|
|
$
|
(3.06
|
)
|
|
$
|
(3.51
|
)
|
|
$
|
(6.34
|
)
|
|
$
|
(14.89
|
)
|
Weighted average common shares outstanding:
|
|
|
|
|
|
|
|
|
|
||||||||||
Basic and diluted
|
35,486
|
|
|
34,580
|
|
|
33,258
|
|
|
9,003
|
|
|
1,452
|
|
|
As of December 31,
|
||||||||||||||||||
|
2016
|
|
2015
|
|
2014
|
|
2013
|
|
2012
|
||||||||||
|
(in thousands)
|
||||||||||||||||||
Balance sheets data:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||
Cash, cash equivalents, and marketable securities
|
$
|
289,278
|
|
|
$
|
391,890
|
|
|
$
|
463,560
|
|
|
$
|
210,596
|
|
|
$
|
4,304
|
|
Total assets
|
$
|
299,630
|
|
|
$
|
428,851
|
|
|
$
|
479,786
|
|
|
$
|
217,682
|
|
|
$
|
4,879
|
|
Deferred revenue
|
$
|
209,976
|
|
|
$
|
213,066
|
|
|
$
|
209,624
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Royalty purchase liability
|
$
|
125,000
|
|
|
$
|
125,000
|
|
|
$
|
125,000
|
|
|
$
|
41,667
|
|
|
$
|
—
|
|
Total liabilities
|
$
|
394,248
|
|
|
$
|
368,904
|
|
|
$
|
351,249
|
|
|
$
|
47,962
|
|
|
$
|
14,410
|
|
Additional paid-in capital
|
$
|
504,517
|
|
|
$
|
465,927
|
|
|
$
|
428,390
|
|
|
$
|
352,739
|
|
|
$
|
—
|
|
Accumulated deficit
|
$
|
(598,959
|
)
|
|
$
|
(405,539
|
)
|
|
$
|
(299,822
|
)
|
|
$
|
(183,050
|
)
|
|
$
|
(126,471
|
)
|
Total stockholders’ equity (deficit)
|
$
|
(94,618
|
)
|
|
$
|
59,947
|
|
|
$
|
128,537
|
|
|
$
|
169,720
|
|
|
$
|
(123,470
|
)
|
•
|
Zimura Phase 2a Wet AMD Study
. During the fourth quarter of 2015, we initiated an open‑label Phase 2a clinical trial to evaluate Zimura’s potential role when administered in combination with anti‑VEGF therapy for the treatment of wet AMD in patients who do not respond adequately to treatment with anti-VEGF monotherapy. We plan to enroll up to approximately 60 patients in this trial, and may include patients with PCV. Recruitment of patients in this study is ongoing.
|
•
|
Zimura Phase 2/3 GA Study
. During the fourth quarter of 2015, we initiated a randomized, double‑masked, controlled Phase 2/3 clinical trial to evaluate the safety and efficacy of Zimura monotherapy in patients with GA. Recruitment of patients in this study is ongoing.
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
|
(in thousands)
|
||||||||||
License revenue
|
$
|
22,937
|
|
|
$
|
38,083
|
|
|
$
|
38,373
|
|
Research and development activity revenue
|
9,741
|
|
|
8,378
|
|
|
2,000
|
|
|||
API transfer revenue
|
18,212
|
|
|
5,020
|
|
|
883
|
|
|||
Joint operating committee revenue
|
19
|
|
|
24
|
|
|
3
|
|
|||
Total collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
•
|
external research and development expenses incurred under arrangements with third parties, such as contract research organizations, or CROs, and other vendors and contract manufacturing organizations, or CMOs, for the production of API and drug product; and
|
•
|
employee-related expenses, including salaries, benefits and share-based compensation expense.
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
|
(in thousands)
|
||||||||||
Fovista
|
$
|
129,661
|
|
|
$
|
86,906
|
|
|
$
|
66,095
|
|
Zimura
|
7,400
|
|
|
7,644
|
|
|
4,377
|
|
|||
Personnel-related
|
26,700
|
|
|
15,830
|
|
|
9,514
|
|
|||
Share-based compensation
|
21,380
|
|
|
16,608
|
|
|
7,594
|
|
|||
Other
|
11,154
|
|
|
4,024
|
|
|
805
|
|
|||
|
$
|
196,295
|
|
|
$
|
131,012
|
|
|
$
|
88,385
|
|
•
|
the scope, rate of progress and expense of our research and development activities;
|
•
|
the potential benefits of our product candidates over other therapies;
|
•
|
clinical trial results;
|
•
|
the terms and timing of regulatory approvals;
|
•
|
our ability, together with any commercialization partner's ability, to market, commercialize and achieve market acceptance for any of our product candidates; and
|
•
|
our ability to successfully file, prosecute, defend and enforce patent claims and other intellectual property rights, together with associated expenses.
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
|
(in thousands)
|
||||||||||
License revenue
|
$
|
22,937
|
|
|
$
|
38,083
|
|
|
$
|
38,373
|
|
Research and development activity revenue
|
9,741
|
|
|
8,378
|
|
|
2,000
|
|
|||
API transfer revenue
|
18,212
|
|
|
5,020
|
|
|
883
|
|
|||
Joint operating committee revenue
|
19
|
|
|
24
|
|
|
3
|
|
|||
Total collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
|
Years ended December 31,
|
||||
|
2016
|
|
2015
|
|
2014
|
Expected common stock price volatility
|
71%
|
|
72%
|
|
82%
|
Risk-free interest rate
|
1.14% - 2.37%
|
|
1.35% - 2.24%
|
|
1.61% - 2.13%
|
Expected term of options (years)
|
6.1
|
|
6.2
|
|
6.2
|
Expected dividend yield
|
—
|
|
—
|
|
—
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
|
(in thousands)
|
||||||||||
Research and development
|
$
|
21,380
|
|
|
$
|
16,608
|
|
|
$
|
7,594
|
|
General and administrative
|
10,280
|
|
|
8,152
|
|
|
5,446
|
|
|||
Total
|
$
|
31,660
|
|
|
$
|
24,760
|
|
|
$
|
13,040
|
|
|
Years ended December 31,
|
|
|
||||||||
|
2016
|
|
2015
|
|
Increase
(Decrease)
|
||||||
|
(in thousands)
|
|
|
||||||||
Statements of Operations Data:
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
(596
|
)
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|||
Research and development
|
196,295
|
|
|
131,012
|
|
|
65,283
|
|
|||
General and administrative
|
50,178
|
|
|
44,021
|
|
|
6,157
|
|
|||
Total operating expenses
|
246,473
|
|
|
175,033
|
|
|
71,440
|
|
|||
Loss from operations
|
(195,564
|
)
|
|
(123,528
|
)
|
|
72,036
|
|
|||
Interest income
|
1,704
|
|
|
971
|
|
|
733
|
|
|||
Other income
|
34
|
|
|
53
|
|
|
(19
|
)
|
|||
Loss before income tax (benefit) provision
|
(193,826
|
)
|
|
(122,504
|
)
|
|
71,322
|
|
|||
Income tax (benefit) provision
|
(406
|
)
|
|
(16,787
|
)
|
|
(16,381
|
)
|
|||
Net loss
|
$
|
(193,420
|
)
|
|
$
|
(105,717
|
)
|
|
$
|
87,703
|
|
|
Years ended December 31,
|
|
|
||||||||
|
2015
|
|
2014
|
|
Increase
(Decrease)
|
||||||
|
(in thousands)
|
|
|
||||||||
Statements of Operations Data:
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue
|
$
|
51,505
|
|
|
$
|
41,259
|
|
|
$
|
10,246
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|||
Research and development
|
131,012
|
|
|
88,385
|
|
|
42,627
|
|
|||
General and administrative
|
44,021
|
|
|
33,387
|
|
|
10,634
|
|
|||
Total operating expenses
|
175,033
|
|
|
121,772
|
|
|
53,261
|
|
|||
Loss from operations
|
(123,528
|
)
|
|
(80,513
|
)
|
|
43,015
|
|
|||
Interest income
|
971
|
|
|
217
|
|
|
754
|
|
|||
Other income
|
53
|
|
|
—
|
|
|
53
|
|
|||
Loss before income tax provision
|
(122,504
|
)
|
|
(80,296
|
)
|
|
42,208
|
|
|||
Income tax (benefit) provision
|
(16,787
|
)
|
|
36,476
|
|
|
(53,263
|
)
|
|||
Net loss
|
$
|
(105,717
|
)
|
|
$
|
(116,772
|
)
|
|
$
|
(11,055
|
)
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
|
(in thousands)
|
||||||||||
Net cash (used in) provided by:
|
|
|
|
|
|
||||||
Operating Activities
|
$
|
(108,596
|
)
|
|
$
|
(78,531
|
)
|
|
$
|
111,088
|
|
Investing Activities
|
13,731
|
|
|
247,803
|
|
|
(427,817
|
)
|
|||
Financing Activities
|
6,934
|
|
|
12,775
|
|
|
145,947
|
|
|||
Net change in cash and cash equivalents
|
$
|
(87,931
|
)
|
|
$
|
182,047
|
|
|
$
|
(170,782
|
)
|
•
|
in‑license or acquire the rights to, and pursue the development of, other products, product candidates or technologies;
|
•
|
complete the activities necessary to receive initial, top-line data from OPH1004 and wind down OPH1002, OPH1003 and the Fovista Expansion Studies and our Fovista contract manufacturing commitments;
|
•
|
potentially undertake additional clinical development of Fovista in wet AMD if the initial, top-line data from OPH1004 is favorable or in other indications if we believe there is a sufficient scientific rationale to pursue such development;
|
•
|
continue the clinical development of Zimura for the treatment of GA and in combination with anti-VEGF drugs for the treatment of wet AMD, or potentially in other indications if we believe there is a sufficient scientific rationale to pursue such development;
|
•
|
complete our previously announced reduction in personnel;
|
•
|
maintain, expand and protect our intellectual property portfolio;
|
•
|
hire additional clinical, manufacturing, quality control, quality assurance and scientific personnel if we are successful in progressing the clinical development of any of our product candidates;
|
•
|
seek marketing approval for any product candidates that successfully complete clinical trials; and
|
•
|
expand our outsourced manufacturing activities, expand our commercial operations and establish sales, marketing and distribution capabilities, if we receive, or expect to receive, marketing approval for any of our product candidates.
|
•
|
the extent to which we in‑license or acquire rights to, and undertake development of products, product candidates or technologies;
|
•
|
the amount of any upfront, milestone payments and other financial obligations associated with the in-license or acquisition of other product candidates;
|
•
|
the scope, progress, results and costs of preclinical development and/or clinical trials for any other product candidates that we may acquire or in-license and develop;
|
•
|
the scope, progress, costs and results of the OPH1004 trial and any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Fovista in wet AMD or any other indication;
|
•
|
the scope, costs and results of our Zimura clinical programs, including our Zimura Phase 2/3 GA trial and our Zimura Phase 2a wet AMD trial, as well as any additional clinical trials we undertake to obtain data sufficient to seek marketing approval for Zimura in any indication (including a potential second Phase 3 trial for GA);
|
•
|
if OPH1004 is positive, the costs and timing of restarting the manufacturing of commercial supply for Fovista;
|
•
|
the costs and timing of process development and manufacturing scale‑up and validation activities associated with Zimura;
|
•
|
our ability to establish collaborations on favorable terms, if at all;
|
•
|
the costs, timing and outcome of regulatory reviews of our product candidates;
|
•
|
the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending intellectual property‑related claims;
|
•
|
the timing, scope and cost of commercialization activities for any of our product candidates if we receive, or expect to receive, marketing approval for a product candidate; and
|
•
|
subject to receipt of marketing approval, net revenue received from commercial sales of any of our product candidates, after milestone payments and royalty payments that we would be obligated to make.
|
|
Payments Due by Period
|
||||||||||||||||||
|
Total
|
|
Less than
1 year
|
|
1 - 3 years
|
|
3 - 5 years
|
|
More than
5 years
|
||||||||||
|
(in thousands)
|
||||||||||||||||||
Operating Leases (1)
|
$
|
4,565
|
|
|
$
|
4,316
|
|
|
$
|
249
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Purchase Obligations (2)
|
44,263
|
|
|
44,263
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|||||
Total (3)
|
$
|
48,828
|
|
|
$
|
48,579
|
|
|
$
|
249
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
(1)
|
In January 2017, we terminated our office leases in New York, NY and Princeton, NJ and made aggregate termination payments of approximately $2.1 million. The table above includes these termination payments, as well as our continuing rent obligations through February 2018.
|
(2)
|
Purchase obligations represent our commitments under binding forecasts, and purchase orders (inclusive of cancellation fees), including those provided under our agreement with Nektar and our clinical and commercial supply agreements with Agilent. The actual amounts incurred will be determined based on the amount of goods purchased and the pricing then in effect under the applicable arrangement
|
(3)
|
This table does not include (a) any milestone payments which may become payable to third parties under license agreements as the timing and likelihood of such payments are not known with certainty, (b) any royalty payments to third parties as the amounts, timing and likelihood of such payments are not known, (c) anticipated expenditures under supply agreements for periods for which we are not yet bound under binding purchase orders, (d) contracts that are entered into in the ordinary course of business that are not material in the aggregate in any period presented above and (e) our royalty purchase liability of $125.0 million as of
December 31, 2016
, due to the fact that the royalty payment period, if any, is not known.
|
•
|
Under our divestiture agreement with OSI (Eyetech), Inc., which agreement is now held by OSI
|
•
|
Under a license agreement with Archemix Corp., or Archemix, with respect to pharmaceutical products comprised of or derived from any anti-PDGF aptamer, we are obligated to make future payments to Archemix of up to an aggregate of $14.0 million if we achieve specified clinical and regulatory milestones with respect to Fovista, up to an aggregate of $3.0 million if we achieve specified commercial milestones with respect to Fovista and, for each other anti-PDGF aptamer product that we may develop under the agreement, up to an aggregate of approximately $18.8 million if we achieve specified clinical and regulatory milestones and up to an aggregate of $3.0 million if we achieve specified commercial milestones. No royalties are payable to Archemix under this license agreement.
|
•
|
Under a license agreement with Archemix with respect to pharmaceutical products comprised of or derived from anti- C5 aptamers, for each anti-C5 aptamer product that we may develop under the agreement, including Zimura, we are obligated to make future payments to Archemix of up to an aggregate of $57.5 million if we achieve specified development, clinical and regulatory milestones, and up to an aggregate of $22.5 million if we achieve specified commercial milestones. We are also obligated to pay Archemix a double-digit percentage of specified non-royalty payments we may receive from any sublicensee of our rights under this license agreement. No royalties are payable to Archemix under this license agreement.
|
•
|
Under a license, manufacturing and supply agreement with Nektar Therapeutics, or Nektar, for specified pegylation reagents used to manufacture Fovista, we are obligated to make future payments to Nektar of up to an aggregate of $6.5 million if we achieve specified clinical and regulatory milestones, and an additional payment of $3.0 million if we achieve a specified commercial milestone with respect to Fovista. We are obligated to pay Nektar tiered royalties at low to mid-single-digit percentages of net sales of any licensed product we successfully commercialize, with the royalty percentage determined by our level of licensed product sales, the extent of patent coverage for the licensed product and whether we have granted a third party commercialization rights to the licensed product. In June 2014, we paid Nektar $19.8 million in connection with our entry into the Novartis Agreement.
|
|
|
Page
|
|
||
|
||
|
||
|
||
|
||
|
||
|
|
|
OPHTHOTECH CORPORATION
|
||
|
|
By:
|
|
/s/ DAVID R. GUYER
|
|
|
|
|
David R. Guyer, M.D.
Chief Executive Officer
|
Signature
|
|
Title
|
|
Date
|
|
|
|
|
|
/s/ DAVID R. GUYER
|
|
Chief Executive Officer and Chairman of the Board of Directors (principal executive officer)
|
|
February 28, 2017
|
David R. Guyer, M.D.
|
|
|
|
|
|
|
|
|
|
/s/ GLENN P. SBLENDORIO
|
|
President and Chief Financial Officer (principal financial and accounting officer)
|
|
February 28, 2017
|
Glenn P. Sblendorio
|
|
|
|
|
|
|
|
|
|
/s/ AXEL BOLTE
|
|
Director
|
|
February 28, 2017
|
Axel Bolte
|
|
|
|
|
|
|
|
|
|
/s/ THOMAS DYRBERG
|
|
Director
|
|
February 28, 2017
|
Thomas Dyrberg, M.D., D.M.Sc.
|
|
|
|
|
|
|
|
|
|
/s/ DAVID E. REDLICK
|
|
Director
|
|
February 28, 2017
|
David E. Redlick
|
|
|
|
|
|
|
|
|
|
/s/ MICHAEL ROSS
|
|
Director
|
|
February 28, 2017
|
Michael Ross, Ph.D.
|
|
|
|
|
|
|
|
|
|
/s/ IAN SMITH
|
|
Director
|
|
February 28, 2017
|
Ian Smith
|
|
|
|
|
|
||
|
||
|
||
|
||
|
||
|
||
|
|
December 31, 2016
|
|
December 31, 2015
|
||||
Assets
|
|
|
|
|
|
||
Current assets
|
|
|
|
|
|
||
Cash and cash equivalents
|
$
|
133,930
|
|
|
$
|
221,861
|
|
Available for sale securities
|
155,348
|
|
|
74,731
|
|
||
Due from Novartis Pharma AG
|
3,531
|
|
|
4,389
|
|
||
Prepaid expenses and other current assets
|
3,078
|
|
|
5,504
|
|
||
Total current assets
|
295,887
|
|
|
306,485
|
|
||
Available for sale securities
|
—
|
|
|
95,298
|
|
||
Property and equipment, net
|
3,281
|
|
|
3,466
|
|
||
Deferred tax assets
|
—
|
|
|
23,113
|
|
||
Other assets
|
462
|
|
|
489
|
|
||
Total assets
|
$
|
299,630
|
|
|
$
|
428,851
|
|
Liabilities and Stockholders' Equity (Deficit)
|
|
|
|
|
|
||
Current liabilities
|
|
|
|
|
|
||
Accrued research and development expenses
|
$
|
47,240
|
|
|
$
|
18,820
|
|
Accounts payable and accrued expenses
|
12,032
|
|
|
12,018
|
|
||
Deferred revenue
|
6,646
|
|
|
6,667
|
|
||
Total current liabilities
|
65,918
|
|
|
37,505
|
|
||
Deferred revenue, long-term
|
203,330
|
|
|
206,399
|
|
||
Royalty purchase liability
|
125,000
|
|
|
125,000
|
|
||
Total liabilities
|
394,248
|
|
|
368,904
|
|
||
Stockholders' equity (deficit)
|
|
|
|
|
|
||
Preferred stock—$0.001 par value, 5,000,000 shares authorized, no shares issued or outstanding
|
$
|
—
|
|
|
$
|
—
|
|
Common stock—$0.001 par value, 200,000,000 shares authorized, 35,733,276 and 35,196,567 shares issued and outstanding at December 31, 2016 and December 31, 2015, respectively
|
36
|
|
|
35
|
|
||
Additional paid-in capital
|
504,517
|
|
|
465,924
|
|
||
Accumulated deficit
|
(598,959
|
)
|
|
(405,539
|
)
|
||
Accumulated other comprehensive loss
|
(212
|
)
|
|
(473
|
)
|
||
Total stockholders' equity (deficit)
|
(94,618
|
)
|
|
59,947
|
|
||
Total liabilities and stockholders' equity (deficit)
|
$
|
299,630
|
|
|
$
|
428,851
|
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|||
Research and development
|
196,295
|
|
|
131,012
|
|
|
88,385
|
|
|||
General and administrative
|
50,178
|
|
|
44,021
|
|
|
33,387
|
|
|||
Total operating expenses
|
246,473
|
|
|
175,033
|
|
|
121,772
|
|
|||
Loss from operations
|
(195,564
|
)
|
|
(123,528
|
)
|
|
(80,513
|
)
|
|||
Interest income
|
1,704
|
|
|
971
|
|
|
217
|
|
|||
Other income
|
34
|
|
|
53
|
|
|
—
|
|
|||
Loss before income tax (benefit) provision
|
(193,826
|
)
|
|
(122,504
|
)
|
|
(80,296
|
)
|
|||
Income tax (benefit) provision
|
(406
|
)
|
|
(16,787
|
)
|
|
36,476
|
|
|||
Net loss
|
(193,420
|
)
|
|
(105,717
|
)
|
|
(116,772
|
)
|
|||
Net loss per common share:
|
|
|
|
|
|
|
|
|
|||
Basic and diluted
|
$
|
(5.45
|
)
|
|
$
|
(3.06
|
)
|
|
$
|
(3.51
|
)
|
Weighted average common shares outstanding:
|
|
|
|
|
|
||||||
Basic and diluted
|
35,486
|
|
|
34,580
|
|
|
33,258
|
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Net loss
|
$
|
(193,420
|
)
|
|
$
|
(105,717
|
)
|
|
$
|
(116,772
|
)
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|||
Unrealized gain (loss) on available for sale securities, net of tax
|
261
|
|
|
(408
|
)
|
|
(65
|
)
|
|||
Other comprehensive income (loss)
|
261
|
|
|
(408
|
)
|
|
(65
|
)
|
|||
Comprehensive loss
|
$
|
(193,159
|
)
|
|
$
|
(106,125
|
)
|
|
$
|
(116,837
|
)
|
|
Junior Series A
Preferred Stock
|
|
Common Stock
|
|
Additional
paid-in
capital
|
|
Accumulated
Deficit
|
|
Accumulated
Other
Comprehensive
Income (Loss)
|
|
Total
|
||||||||||||||||||
|
Shares
|
|
Amount
|
|
Shares
|
|
Amount
|
|
|
|
|
||||||||||||||||||
Balance at December 31, 2013
|
—
|
|
|
$
|
—
|
|
|
31,413
|
|
|
$
|
31
|
|
|
$
|
352,739
|
|
|
$
|
(183,050
|
)
|
|
$
|
—
|
|
|
$
|
169,720
|
|
Issuance of common stock under employee stock compensation plans and warrants
|
—
|
|
|
—
|
|
|
682
|
|
|
1
|
|
|
2,948
|
|
|
—
|
|
|
—
|
|
|
2,949
|
|
||||||
Issuance from follow-on public offering, net
|
—
|
|
|
—
|
|
|
1,900
|
|
|
2
|
|
|
55,407
|
|
|
—
|
|
|
—
|
|
|
55,409
|
|
||||||
Share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
13,040
|
|
|
—
|
|
|
—
|
|
|
13,040
|
|
||||||
Excess tax benefit from share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
4,256
|
|
|
—
|
|
|
—
|
|
|
4,256
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(116,772
|
)
|
|
—
|
|
|
(116,772
|
)
|
||||||
Unrealized loss on available for sale securities, net of tax
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(65
|
)
|
|
(65
|
)
|
||||||
Balance at December 31, 2014
|
—
|
|
|
$
|
—
|
|
|
33,995
|
|
|
$
|
34
|
|
|
$
|
428,390
|
|
|
$
|
(299,822
|
)
|
|
$
|
(65
|
)
|
|
$
|
128,537
|
|
Issuance of common stock under employee stock compensation plans and warrants
|
—
|
|
|
—
|
|
|
1,202
|
|
|
1
|
|
|
11,472
|
|
|
—
|
|
|
—
|
|
|
11,473
|
|
||||||
Share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
24,760
|
|
|
—
|
|
|
—
|
|
|
24,760
|
|
||||||
Excess tax benefit from share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
1,302
|
|
|
—
|
|
|
—
|
|
|
1,302
|
|
||||||
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
—
|
|
|
(105,717
|
)
|
|
—
|
|
|
(105,717
|
)
|
||||||
Unrealized loss on available for sale securities, net of tax
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(408
|
)
|
|
(408
|
)
|
||||||
Balance at December 31, 2015
|
—
|
|
|
$
|
—
|
|
|
35,197
|
|
|
$
|
35
|
|
|
$
|
465,924
|
|
|
$
|
(405,539
|
)
|
|
$
|
(473
|
)
|
|
$
|
59,947
|
|
Issuance of common stock under employee stock compensation plans and warrants
|
—
|
|
|
—
|
|
|
536
|
|
|
1
|
|
|
6,933
|
|
|
—
|
|
|
—
|
|
|
6,934
|
|
||||||
Share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
31,660
|
|
|
—
|
|
|
—
|
|
|
31,660
|
|
||||||
Excess tax benefit from share-based compensation
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(193,420
|
)
|
|
—
|
|
|
(193,420
|
)
|
||||||
Unrealized gain on available for sale securities, net of tax
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
261
|
|
|
261
|
|
||||||
Balance at December 31, 2016
|
—
|
|
|
$
|
—
|
|
|
35,733
|
|
|
$
|
36
|
|
|
$
|
504,517
|
|
|
$
|
(598,959
|
)
|
|
$
|
(212
|
)
|
|
$
|
(94,618
|
)
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Operating Activities
|
|
|
|
|
|
|
|
|
|||
Net loss
|
$
|
(193,420
|
)
|
|
$
|
(105,717
|
)
|
|
$
|
(116,772
|
)
|
Adjustments to reconcile net loss to net cash (used in) provided by operating activities
|
|
|
|
|
|
|
|
|
|||
Depreciation
|
757
|
|
|
698
|
|
|
127
|
|
|||
Amortization of premium and discounts on investment securities
|
595
|
|
|
2,846
|
|
|
2,024
|
|
|||
Gain on sale of marketable securities
|
—
|
|
|
(57
|
)
|
|
—
|
|
|||
Deferred income taxes
|
22,954
|
|
|
(17,341
|
)
|
|
(214
|
)
|
|||
Share-based compensation
|
31,660
|
|
|
24,760
|
|
|
13,040
|
|
|||
Excess tax benefits from share-based compensation
|
—
|
|
|
(1,302
|
)
|
|
(4,256
|
)
|
|||
Changes in operating assets and liabilities:
|
|
|
|
|
|
|
|
|
|||
Due from Novartis Pharma AG
|
858
|
|
|
(3,429
|
)
|
|
(960
|
)
|
|||
Prepaid expense and other current assets
|
2,426
|
|
|
3,308
|
|
|
(2,008
|
)
|
|||
Accrued interest receivable
|
203
|
|
|
155
|
|
|
280
|
|
|||
Other assets
|
27
|
|
|
(107
|
)
|
|
(127
|
)
|
|||
Accrued research and development expenses
|
28,420
|
|
|
10,902
|
|
|
5,433
|
|
|||
Accounts payable and accrued expenses
|
14
|
|
|
3,311
|
|
|
4,897
|
|
|||
Deferred revenue
|
(3,090
|
)
|
|
3,442
|
|
|
209,624
|
|
|||
Net cash (used in) provided by operating activities
|
(108,596
|
)
|
|
(78,531
|
)
|
|
111,088
|
|
|||
Investing Activities
|
|
|
|
|
|
|
|
|
|||
Purchase of marketable securities
|
(72,197
|
)
|
|
(411,565
|
)
|
|
(597,762
|
)
|
|||
Sale of marketable securities
|
—
|
|
|
395,977
|
|
|
—
|
|
|||
Maturities of marketable securities
|
86,500
|
|
|
266,000
|
|
|
171,600
|
|
|||
Purchase of property and equipment
|
(572
|
)
|
|
(2,615
|
)
|
|
(1,655
|
)
|
|||
Proceeds from sale of assets
|
—
|
|
|
6
|
|
|
—
|
|
|||
Net cash provided by (used in) investing activities
|
13,731
|
|
|
247,803
|
|
|
(427,817
|
)
|
|||
Financing Activities
|
|
|
|
|
|
|
|
|
|||
Proceeds from stock option/warrant exercises
|
6,934
|
|
|
11,473
|
|
|
2,949
|
|
|||
Proceeds from follow-on public offering, net
|
—
|
|
|
—
|
|
|
55,409
|
|
|||
Excess tax benefits from share-based compensation
|
—
|
|
|
1,302
|
|
|
4,256
|
|
|||
Proceeds from royalty purchase agreement
|
—
|
|
|
—
|
|
|
83,333
|
|
|||
Net cash provided by financing activities
|
6,934
|
|
|
12,775
|
|
|
145,947
|
|
|||
Net change in cash and cash equivalents
|
(87,931
|
)
|
|
182,047
|
|
|
(170,782
|
)
|
|||
Cash and cash equivalents
|
|
|
|
|
|
|
|
|
|||
Beginning of period
|
221,861
|
|
|
39,814
|
|
|
210,596
|
|
|||
End of period
|
$
|
133,930
|
|
|
$
|
221,861
|
|
|
$
|
39,814
|
|
Supplemental disclosure of cash paid
|
|
|
|
|
|
|
|
|
|||
Income taxes paid (received), net
|
$
|
(26,998
|
)
|
|
$
|
399
|
|
|
$
|
40,159
|
|
Supplemental disclosures of non-cash information related to investing activities
|
|
|
|
|
|
|
|
|
|||
Change in unrealized gain (loss) on available for sale securities, net of tax
|
$
|
261
|
|
|
$
|
(408
|
)
|
|
$
|
(65
|
)
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
License revenue
|
$
|
22,937
|
|
|
$
|
38,083
|
|
|
$
|
38,373
|
|
Research and development activity revenue
|
9,741
|
|
|
8,378
|
|
|
2,000
|
|
|||
API transfer revenue
|
18,212
|
|
|
5,020
|
|
|
883
|
|
|||
Joint operating committee revenue
|
19
|
|
|
24
|
|
|
3
|
|
|||
Total collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
•
|
external research and development expenses incurred under arrangements with third parties, such as contract research organizations ("CROs") and other vendors and contract manufacturing organizations ("CMOs") for the production of drug substance and drug product; and
|
•
|
employee-related expenses, including salaries, benefits and share-based compensation expense.
|
|
Years ended December 31,
|
||||
|
2016
|
|
2015
|
|
2014
|
Expected common stock price volatility
|
71%
|
|
72%
|
|
82%
|
Risk-free interest rate
|
1.14% - 2.37%
|
|
1.35% - 2.24%
|
|
1.61% - 2.13%
|
Expected term of options (years)
|
6.1
|
|
6.2
|
|
6.2
|
Expected dividend yield
|
—
|
|
—
|
|
—
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Research and development
|
$
|
21,380
|
|
|
$
|
16,608
|
|
|
$
|
7,594
|
|
General and administrative
|
10,280
|
|
|
8,152
|
|
|
5,446
|
|
|||
Total
|
$
|
31,660
|
|
|
$
|
24,760
|
|
|
$
|
13,040
|
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Basic and diluted net loss per common share calculation:
|
|
|
|
|
|
|
|
|
|||
Net loss
|
$
|
(193,420
|
)
|
|
$
|
(105,717
|
)
|
|
$
|
(116,772
|
)
|
Weighted average common shares outstanding
|
35,486
|
|
|
34,580
|
|
|
33,258
|
|
|||
Net loss per common share—basic and diluted
|
$
|
(5.45
|
)
|
|
$
|
(3.06
|
)
|
|
$
|
(3.51
|
)
|
|
Years ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Stock options outstanding
|
3,359
|
|
|
3,009
|
|
|
3,680
|
|
Restricted stock units
|
721
|
|
|
288
|
|
|
37
|
|
Warrants
|
—
|
|
|
—
|
|
|
14
|
|
Total
|
4,080
|
|
|
3,297
|
|
|
3,731
|
|
|
As of December 31, 2016
|
||||||||||||||
|
Cost
|
|
Unrealized Gains
|
|
Unrealized Losses
|
|
Fair Value
|
||||||||
U.S. Treasury securities
|
$
|
120,288
|
|
|
$
|
6
|
|
|
$
|
(33
|
)
|
|
$
|
120,261
|
|
Corporate debt securities
|
35,114
|
|
|
—
|
|
|
(27
|
)
|
|
35,087
|
|
||||
Total
|
$
|
155,402
|
|
|
$
|
6
|
|
|
$
|
(60
|
)
|
|
$
|
155,348
|
|
|
As of December 31, 2015
|
||||||||||||||
|
Cost
|
|
Unrealized Gains
|
|
Unrealized Losses
|
|
Fair Value
|
||||||||
U.S. Treasury securities
|
$
|
130,507
|
|
|
$
|
—
|
|
|
$
|
(311
|
)
|
|
$
|
130,196
|
|
Corporate debt securities
|
39,995
|
|
|
—
|
|
|
(162
|
)
|
|
39,833
|
|
||||
Total
|
$
|
170,502
|
|
|
$
|
—
|
|
|
$
|
(473
|
)
|
|
$
|
170,029
|
|
|
Years ended December 31,
|
||||||
|
2016
|
|
2015
|
||||
Beginning balance
|
$
|
(473
|
)
|
|
$
|
(65
|
)
|
Current period changes in fair value before reclassifications, net of tax
|
261
|
|
|
(351
|
)
|
||
Amounts reclassified from accumulated other comprehensive income (loss), net of tax
|
—
|
|
|
(57
|
)
|
||
Total other comprehensive income (loss)
|
261
|
|
|
(408
|
)
|
||
Ending balance
|
$
|
(212
|
)
|
|
$
|
(473
|
)
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
License revenue
|
$
|
22,937
|
|
|
$
|
38,083
|
|
|
$
|
38,373
|
|
Research and development activity revenue
|
9,741
|
|
|
8,378
|
|
|
2,000
|
|
|||
API transfer revenue
|
18,212
|
|
|
5,020
|
|
|
883
|
|
|||
Joint operating committee revenue
|
19
|
|
|
24
|
|
|
3
|
|
|||
Total collaboration revenue
|
$
|
50,909
|
|
|
$
|
51,505
|
|
|
$
|
41,259
|
|
|
Useful Life
(Years)
|
|
December 31,
2016 |
|
December 31,
2015 |
||||
Manufacturing and clinical equipment
|
7 - 10
|
|
$
|
617
|
|
|
$
|
617
|
|
Computer, software and other office equipment
|
5
|
|
1,711
|
|
|
944
|
|
||
Furniture and fixtures
|
7
|
|
774
|
|
|
738
|
|
||
Leasehold improvements
|
3 - 5
|
|
1,835
|
|
|
1,551
|
|
||
Construction-in-progress
|
|
|
—
|
|
|
515
|
|
||
|
|
|
4,937
|
|
|
4,365
|
|
||
Accumulated depreciation
|
|
|
(1,656
|
)
|
|
(899
|
)
|
||
Property and equipment, net
|
|
|
$
|
3,281
|
|
|
$
|
3,466
|
|
|
Years ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Percent of pre-tax income:
|
|
|
|
|
|
|
|
|
U.S. federal statutory income tax rate
|
35.0
|
%
|
|
35.0
|
%
|
|
35.0
|
%
|
State taxes, net of federal benefit
|
2.8
|
%
|
|
7.4
|
%
|
|
6.8
|
%
|
Permanent items
|
(1.4
|
)%
|
|
(0.5
|
)%
|
|
2.3
|
%
|
Impact of state rate changes
|
(11.0
|
)%
|
|
0.9
|
%
|
|
—
|
%
|
Research and development credit
|
1.9
|
%
|
|
—
|
%
|
|
—
|
%
|
Other
|
1.1
|
%
|
|
—
|
%
|
|
—
|
%
|
Change in valuation allowance
|
(28.2
|
)%
|
|
(29.1
|
)%
|
|
(89.5
|
)%
|
Effective income tax rate
|
0.2
|
%
|
|
13.7
|
%
|
|
(45.4
|
)%
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Current:
|
|
|
|
|
|
|
|
|
|||
Federal
|
$
|
(23,393
|
)
|
|
$
|
136
|
|
|
$
|
29,505
|
|
State
|
21
|
|
|
91
|
|
|
11,440
|
|
|||
Deferred:
|
|
|
|
|
|
|
|
|
|||
Federal
|
22,966
|
|
|
(17,014
|
)
|
|
(4,469
|
)
|
|||
State
|
—
|
|
|
—
|
|
|
—
|
|
|||
Income tax (benefit) expense
|
$
|
(406
|
)
|
|
$
|
(16,787
|
)
|
|
$
|
36,476
|
|
|
As of December 31,
|
||||||
|
2016
|
|
2015
|
||||
Deferred tax assets (liabilities)
|
|
|
|
|
|
||
Deferred revenue
|
$
|
125,634
|
|
|
$
|
142,675
|
|
License and technology payments
|
10,532
|
|
|
13,150
|
|
||
Share-based compensation
|
16,494
|
|
|
11,427
|
|
||
Accrued expenses
|
530
|
|
|
743
|
|
||
Depreciation
|
(651
|
)
|
|
(617
|
)
|
||
Federal and state net operating loss carryforwards
|
75,177
|
|
|
26,065
|
|
||
Excess tax benefits related to share-based compensation
|
—
|
|
|
1,630
|
|
||
Research and development credits
|
3,720
|
|
|
—
|
|
||
Other
|
2,155
|
|
|
5
|
|
||
Deferred income tax assets
|
233,591
|
|
|
195,078
|
|
||
Valuation allowance
|
(233,591
|
)
|
|
(171,965
|
)
|
||
Net deferred tax assets
|
$
|
—
|
|
|
$
|
23,113
|
|
Opening balance
|
$
|
300
|
|
Gross amount of increases in unrecognized tax benefits during the period - current year provisions
|
—
|
|
|
Gross amount of increases in unrecognized tax benefits during the period - prior year provisions
|
3,828
|
|
|
Gross amount of increases in unrecognized tax benefits during the period - other
|
—
|
|
|
Decreases due to settlement with tax authorities during the period
|
—
|
|
|
Reduction of unrecognized tax benefits due to expiration of the state of limitations during the period
|
—
|
|
|
Closing Balance
|
$
|
4,128
|
|
2017
|
$
|
4,316
|
|
2018
|
249
|
|
|
Total
|
$
|
4,565
|
|
•
|
Under the Company's divestiture agreement with OSI (Eyetech), Inc., which agreement is now held by OSI Pharmaceuticals, LLC., or OSI Pharmaceuticals, a subsidiary of Astellas US, LLC, for rights to particular anti-PDGF aptamers, including Fovista, the Company is obligated to pay to OSI Pharmaceuticals future one-time payments of
$12.0 million
in the aggregate upon marketing approval in the United States and the European Union of a covered anti-PDGF product. The Company is also obligated to pay to OSI Pharmaceuticals a royalty at a low single-digit percentage of net sales of any covered anti-PDGF product the Company successfully commercializes.
|
•
|
Under a license agreement with Archemix Corp., or Archemix, with respect to pharmaceutical products comprised of or derived from any anti-PDGF aptamer, the Company is obligated to make future payments to Archemix of up to an aggregate of
$14.0 million
if the Company achieves specified clinical and regulatory milestones with respect to Fovista, up to an aggregate of
$3.0 million
if the Company achieves specified commercial milestones with respect to Fovista and, for each other anti-PDGF aptamer product that it may develop under the agreement, up to an aggregate of approximately
$18.8 million
if the Company achieves specified clinical and regulatory milestones and up to an aggregate of
$3.0 million
if the Company achieves specified commercial milestones.
No
royalties are payable to Archemix under this license agreement.
|
•
|
Under a license agreement with Archemix with respect to pharmaceutical products comprised of or derived from anti-C5 aptamers, for each anti-C5 aptamer product that the Company may develop under the agreement, including Zimura, the Company is obligated to make future payments to Archemix of up to an aggregate of
$57.5 million
if the Company achieves specified development, clinical and regulatory milestones, and up to an aggregate of
$22.5 million
if the Company achieves specified commercial milestones. The Company is also obligated to pay Archemix a double-digit percentage of specified non-royalty payments the Company may receive from any sublicensee of the Company's rights under this license agreement.
No
royalties are payable to Archemix under this license agreement.
|
•
|
Under a license, manufacturing and supply agreement with Nektar Therapeutics, or Nektar, for specified pegylation reagents used to manufacture Fovista, the Company is obligated to make future payments to Nektar of up to an aggregate of
$6.5 million
if the Company achieves specified clinical and regulatory milestones, and an additional payment of
$3.0 million
if the Company achieves a specified commercial milestone with respect to Fovista. The Company is obligated to pay Nektar tiered royalties at low to mid-single-digit percentages of net sales of any licensed product the Company successfully commercializes, with the royalty percentage determined by the Company's level of licensed product sales, the extent of patent coverage for the licensed product and whether the Company has granted a third-party commercialization rights to the licensed product. In June 2014, the Company paid Nektar
$19.8 million
in connection with its entry into the Novartis Agreement.
|
•
|
Under the Novo Agreement, with respect to Fovista, the Company will be obligated to pay Novo A/S a mid-single-digit percentage royalty based on worldwide sales of Fovista. See "Note 6—Financing Agreement with Novo A/S" above for further information about Novo Agreement.
|
•
|
Under an option agreement with AVEO Pharmaceuticals relating to tivozanib, the Company was obligated to make milestone payments of
$2.0 million
upon the submission of an Investigational New Drug Application to the FDA and
$6.0 million
upon the earlier of demonstration of proof of concept in humans and a specified date in January 2017, subject to any exercise by the Company of its right to terminate the option agreement. In January 2017, the Company provided notice of termination of the Agreement, effective April 3, 2017.
|
|
Years ended December 31,
|
|||||||||||||||||||
|
2016
|
|
2015
|
|
2014
|
|||||||||||||||
|
Common
Stock
Options
|
|
Weighted
Average
Exercise
Price
|
|
Common
Stock
Options
|
|
Weighted
Average
Exercise
Price
|
|
Common
Stock
Options
|
|
Weighted
Average
Exercise
Price
|
|||||||||
Outstanding, December 31, 2015
|
3,009
|
|
|
$
|
30.43
|
|
|
3,680
|
|
|
$
|
21.03
|
|
|
2,708
|
|
|
$
|
9.41
|
|
Granted
|
972
|
|
|
$
|
60.40
|
|
|
764
|
|
|
$
|
47.31
|
|
|
1,744
|
|
|
$
|
33.92
|
|
Exercised
|
(428
|
)
|
|
$
|
15.73
|
|
|
(1,133
|
)
|
|
$
|
10.31
|
|
|
(621
|
)
|
|
$
|
4.75
|
|
Expired or forfeited
|
(194
|
)
|
|
$
|
48.63
|
|
|
(302
|
)
|
|
$
|
34.09
|
|
|
(151
|
)
|
|
$
|
28.50
|
|
Outstanding, December 31, 2016
|
3,359
|
|
|
$
|
39.92
|
|
|
3,009
|
|
|
$
|
30.43
|
|
|
3,680
|
|
|
$
|
21.03
|
|
|
Years ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Options exercisable at December 31, 2016
|
1,531
|
|
|
955
|
|
|
993
|
|
Weighted average grant date fair value (per share) of options granted during the period
|
38.18
|
|
|
31.33
|
|
|
24.41
|
|
|
|
|
December 31, 2016
|
||||||||||||
|
|
|
Options Outstanding
|
|
Options Exercisable
|
||||||||||
Range of Exercise Prices
|
Total
Options
Outstanding
|
|
Weighted
Average
Remaining
Life (Years)
|
|
Weighted
Average
Exercise
Price
|
|
Number
Exercisable
|
|
Weighted
Average
Exercise
Price
|
||||||
$0.12-$10.03
|
193
|
|
|
5.3
|
|
$
|
6.84
|
|
|
137
|
|
|
$
|
5.52
|
|
$10.04-$20.00
|
273
|
|
|
6.5
|
|
$
|
13.44
|
|
|
170
|
|
|
$
|
13.48
|
|
$20.01-$30.00
|
154
|
|
|
6.8
|
|
$
|
25.56
|
|
|
114
|
|
|
$
|
25.53
|
|
$30.01-$40.00
|
1,087
|
|
|
6.7
|
|
$
|
32.70
|
|
|
758
|
|
|
$
|
32.97
|
|
$40.01-$55.00
|
1,125
|
|
|
8.6
|
|
$
|
46.22
|
|
|
319
|
|
|
$
|
45.49
|
|
$55.01-$73.22
|
527
|
|
|
9.0
|
|
$
|
71.33
|
|
|
33
|
|
|
$
|
69.08
|
|
|
3,359
|
|
|
7.6
|
|
$
|
39.92
|
|
|
1,531
|
|
|
$
|
31.19
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Aggregate Intrinsic Value
|
254
|
|
|
|
|
|
|
|
254
|
|
|
|
|
|
Years ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Cash proceeds from options exercised
|
$
|
6,934
|
|
|
$
|
11,473
|
|
|
$
|
2,949
|
|
Aggregate intrinsic value of options exercised
|
$
|
14,439
|
|
|
$
|
49,255
|
|
|
$
|
21,646
|
|
|
Restricted
Stock
Units
|
|
Weighted Average
Grant-Date
Fair Value
|
|||
Outstanding, December 31, 2015
|
288
|
|
|
$
|
44.54
|
|
Awarded
|
583
|
|
|
$
|
58.19
|
|
Vested
|
(109
|
)
|
|
$
|
43.88
|
|
Forfeited
|
(41
|
)
|
|
$
|
50.60
|
|
Outstanding, December 31, 2016
|
721
|
|
|
$
|
55.33
|
|
•
|
Level 1—inputs to the valuation methodology are quoted prices (unadjusted) for an identical asset or liability in an active market. The Company's Level 1 assets consist of investments in money market funds and U.S. Treasury securities.
|
•
|
Level 2—inputs to the valuation methodology include quoted prices for a similar asset or liability in an active market or model-derived valuations in which all significant inputs are observable for substantially the full term of the asset or liability. The Company's Level 2 assets consist of investments in investment-grade corporate debt securities.
|
•
|
Level 3—inputs to the valuation methodology are unobservable and significant to the fair value measurement of the asset or liability. The Company does not hold any assets that are measured using Level 3 inputs.
|
|
Fair Value Measurement Using
|
||||||||||
|
Quoted prices in
active markets for
identical assets
(Level 1)
|
|
Significant other
observable inputs
(Level 2)
|
|
Significant
unobservable
inputs
(Level 3)
|
||||||
Assets
|
|
|
|
|
|
|
|
|
|||
Investments in money market funds*
|
$
|
108,096
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Investments in U.S. Treasury securities
|
$
|
120,261
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Investments in Corporate debt securities
|
$
|
—
|
|
|
$
|
35,087
|
|
|
$
|
—
|
|
|
Fair Value Measurement Using
|
||||||||||
|
Quoted prices in
active markets for
identical assets
(Level 1)
|
|
Significant other
observable inputs
(Level 2)
|
|
Significant
unobservable
inputs
(Level 3)
|
||||||
Assets
|
|
|
|
|
|
|
|
|
|||
Investments in money market funds*
|
$
|
196,188
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Investments in U.S. Treasury securities*
|
$
|
150,387
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Investments in Corporate debt securities
|
$
|
—
|
|
|
$
|
39,833
|
|
|
$
|
—
|
|
|
*
|
Investments in money market funds, U.S. Treasury securities and corporate debt securities with maturities less than 90 days are reflected in cash and cash equivalents in the accompanying Balance Sheets.
|
|
2016
|
||||||||||||||
|
March 31
|
|
June 30
|
|
September 30
|
|
December 31
|
||||||||
Collaboration revenue
|
$
|
15,721
|
|
|
$
|
28,198
|
|
|
$
|
1,668
|
|
|
$
|
5,322
|
|
Research and development expenses
|
37,770
|
|
|
48,262
|
|
|
50,854
|
|
|
59,409
|
|
||||
General and administrative expenses
|
14,696
|
|
|
10,489
|
|
|
12,024
|
|
|
12,968
|
|
||||
Loss from operations
|
(36,745
|
)
|
|
(30,553
|
)
|
|
(61,210
|
)
|
|
(67,055
|
)
|
||||
Net loss attributable to common stockholders
|
$
|
(36,301
|
)
|
|
$
|
(29,945
|
)
|
|
$
|
(60,891
|
)
|
|
$
|
(66,283
|
)
|
Basic and diluted loss per common share
|
$
|
(1.03
|
)
|
|
$
|
(0.85
|
)
|
|
$
|
(1.71
|
)
|
|
$
|
(1.86
|
)
|
|
2015
|
||||||||||||||
|
March 31
|
|
June 30
|
|
September 30
|
|
December 31
|
||||||||
Collaboration revenue
|
$
|
41,678
|
|
|
$
|
1,597
|
|
|
$
|
3,448
|
|
|
$
|
4,782
|
|
Research and development expenses
|
24,557
|
|
|
32,059
|
|
|
40,479
|
|
|
33,917
|
|
||||
General and administrative expenses
|
9,584
|
|
|
11,959
|
|
|
10,412
|
|
|
12,066
|
|
||||
Income (loss) from operations
|
7,537
|
|
|
(42,421
|
)
|
|
(47,443
|
)
|
|
(41,201
|
)
|
||||
Net income (loss) attributable to common stockholders
|
$
|
6,636
|
|
|
$
|
(37,131
|
)
|
|
$
|
(39,573
|
)
|
|
$
|
(35,649
|
)
|
Basic earnings (loss) per common share
|
$
|
0.19
|
|
|
$
|
(1.08
|
)
|
|
$
|
(1.14
|
)
|
|
$
|
(1.02
|
)
|
Diluted earnings (loss) per common share
|
$
|
0.19
|
|
|
$
|
(1.08
|
)
|
|
$
|
(1.14
|
)
|
|
$
|
(1.02
|
)
|
|
|
|
|
|
|
Incorporated by Reference
|
|
|
|
|
||||
Exhibit
Number
|
|
Description of Exhibit
|
|
Form
|
|
File
Number
|
|
Date of
Filing
|
|
Exhibit
Number
|
|
Filed
Herewith
|
||
3.1
|
|
|
Restated Certificate of Incorporation of the Registrant
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
3.3
|
|
|
|
3.2
|
|
|
Amended and Restated Bylaws of the Registrant
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
3.4
|
|
|
|
4.1
|
|
|
Specimen Stock Certificate evidencing the shares of common stock
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
4.1
|
|
|
|
10.1
|
|
+
|
Amended and Restated 2007 Stock Incentive Plan, as amended
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.1
|
|
|
|
10.2
|
|
+
|
Form of Incentive Stock Option Agreement under Amended and Restated 2007 Stock Incentive Plan
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.2
|
|
|
|
10.3
|
|
+
|
Form of Nonstatutory Stock Option Agreement under 2007
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.3
|
|
|
|
10.4
|
|
+
|
2013 Stock Incentive Plan
|
|
10-K
|
|
|
|
3/2/2015
|
|
10.4
|
|
|
|
10.5
|
|
+
|
Amendment No. 1 to Stock Incentive Plan, adopted June 4, 2015
|
|
10-Q
|
|
|
|
8/10/2015
|
|
10.1
|
|
|
|
10.6
|
|
+
|
Form of Incentive Stock Option Agreement under 2013 Stock Incentive Plan
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
10.5
|
|
|
|
10.7
|
|
+
|
Form of Nonqualified Stock Option Agreement under 2013 Stock Incentive Plan
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
10.6
|
|
|
|
10.8
|
|
+
|
Form of Restricted Stock Unit Agreement under 2013 Stock Incentive Plan
|
|
10-K
|
|
|
|
3/2/2015
|
|
10.7
|
|
|
|
10.9
|
|
|
Lease Agreement, dated as of September 30, 2007, between the Registrant and One Penn Plaza LLC, as the same has been supplemented by agreement dated March 12, 2013 and amended by the Amendment of Lease, dated as of August 30, 2013, Second Amendment to Lease, entered into on January 7, 2014, Third Amendment of Lease, dated as of April 18, 2014, and the Fourth Amendment of Lease, dated as of December 22, 2014
|
|
10-K
|
|
|
|
3/2/2015
|
|
10.8
|
|
|
|
10.10
|
|
|
Sublease Agreement, dated April 7, 2015, by and between Otsuka America Pharmaceutical, Inc. and the Registrant
|
|
10-Q
|
|
|
|
8/10/2015
|
|
10.2
|
|
|
|
|
|
|
|
|
|
Incorporated by Reference
|
|
|
|
|
||||
Exhibit
Number
|
|
Description of Exhibit
|
|
Form
|
|
File
Number
|
|
Date of
Filing
|
|
Exhibit
Number
|
|
Filed
Herewith
|
||
10.11
|
|
|
Lease Agreement with Carnegie 214 Associates Limited Partnership, dated as of October 25, 2013
|
|
S-1
|
|
333-193681
|
|
1/31/2014
|
|
10.8
|
|
|
|
10.12
|
|
|
Office Lease Agreement, dated as of August 22, 2013, by and between the Registrant and PSN Partners, L.P.
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
10.17
|
|
|
|
10.13
|
|
†
|
Divestiture Agreement, dated as of July 27, 2007, by and between the Registrant and (OSI) Eyetech, Inc.
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.9
|
|
|
|
10.14
|
|
†
|
License, Manufacturing and Supply Agreement, dated as of September 30, 2006, by and between Nektar Therapeutics AL, Corporation and (OSI) Eyetech, Inc., as the same was assigned to the Registrant on July 27, 2007 and amended by Amendment No. 1 thereto, dated as of April 5, 2012, and supplemented by a letter agreement, dated as of June 20, 2013
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.10
|
|
|
|
10.15
|
|
†
|
Amendment No. 2 to, Scope of Work #1 for, and Amendment No. 3 to License, Manufacturing and Supply Agreement, dated as of September 30, 2006, by and between Nektar Therapeutics AL, Corporation and (OSI) Eyetech, Inc., as the same was assigned to the Registrant on July 27, 2007 and amended by Amendment No. 1 thereto, dated as of April 5, 2012, and supplemented by a letter agreement, dated as of June 20, 2013.
|
|
10-Q
|
|
|
|
5/11/2015
|
|
10.1
|
|
|
|
10.16
|
|
†
|
Amended and Restated Exclusive License Agreement, dated as of September 12, 2011, by and between the Registrant and Archemix Corp., as amended by Amendment No. 1 thereto dated December 20, 2011 and supplemented by a letter agreement, dated as of April 30, 2012
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.11
|
|
|
|
|
|
|
|
|
|
Incorporated by Reference
|
|
|
|
|
||||
Exhibit
Number
|
|
Description of Exhibit
|
|
Form
|
|
File
Number
|
|
Date of
Filing
|
|
Exhibit
Number
|
|
Filed
Herewith
|
||
10.17
|
|
†
|
Amended and Restated Exclusive License Agreement, dated as of September 12, 2011, by and between the Registrant and Archemix Corp., as amended by Amendment No. 1 thereto, dated as of December 20, 2011
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.12
|
|
|
|
10.18
|
|
†
|
Purchase and Sale Agreement, dated as of May 23, 2013, by and between the Registrant and Novo A/S
|
|
S-1
|
|
333-190643
|
|
8/15/2013
|
|
10.13
|
|
|
|
10.19
|
|
†
|
Amendment No. 1 to the Purchase and Sale Agreement, dated as of May 23, 2013, by and between the Registrant and Novo A/S
|
|
10-K
|
|
|
|
3/2/2015
|
|
10.24
|
|
|
|
10.20
|
|
†
|
Licensing and Commercialization Agreement by and between the Registrant and Novartis Pharma AG dated May 19, 2014
|
|
10-Q
|
|
|
|
8/6/2014
|
|
10.2
|
|
|
|
10.21
|
|
†
|
Clinical Manufacturing and Supply Agreement by and between the Registrant and Agilent Technologies, Inc. dated May 2, 2014
|
|
10-Q
|
|
|
|
8/6/2014
|
|
10.1
|
|
|
|
10.22
|
|
†
|
Amendment No. 1 to Clinical Manufacturing and Supply Agreement by and between the Registrant and Agilent Technologies, Inc. dated September 3, 2015
|
|
10-Q
|
|
|
|
11/5/2015
|
|
10.1
|
|
|
|
10.23
|
|
†
|
Commercial Manufacturing and Supply Agreement by and between the Registrant and Agilent Technologies, Inc. dated September 3, 2015
|
|
10-Q
|
|
|
|
11/5/2015
|
|
10.2
|
|
|
|
10.24
|
|
+
|
Offer of Employment between the Registrant and David Guyer
|
|
S-1/A
|
|
333-190643
|
|
9/9/2013
|
|
10.14
|
|
|
|
10.25
|
|
+
|
Letter Agreement between the Registrant and David R. Guyer dated February 26, 2015, amending the Offer of Employment between the Registrant and David R. Guyer dated April 26, 2013
|
|
10-Q
|
|
|
|
5/11/2015
|
|
10.2
|
|
|
|
10.26
|
|
+
|
Third Amended and Restated Employment Agreement between the Registrant and Samir C. Patel, dated May 1, 2015
|
|
10-Q
|
|
|
|
5/11/2015
|
|
10.3
|
|
|
|
|
|
|
|
|
|
Incorporated by Reference
|
|
|
|
|
||||
Exhibit
Number
|
|
Description of Exhibit
|
|
Form
|
|
File
Number
|
|
Date of
Filing
|
|
Exhibit
Number
|
|
Filed
Herewith
|
||
10.27
|
|
+
|
Separation and Release of Claims Agreement between the Registrant and Samir C. Patel dated January 12, 2017
|
|
|
|
|
|
|
|
|
|
Yes
|
|
10.28
|
|
+
|
Consulting Agreement between the Registrant and Samir C. Patel dated January 12, 2017
|
|
|
|
|
|
|
|
|
|
Yes
|
|
10.29
|
|
+
|
Letter Agreement between the Registrant and Glenn P. Sblendorio, dated January 4, 2016
|
|
10-Q
|
|
|
|
5/9/2015
|
|
10.1
|
|
|
|
10.30
|
|
+
|
Letter Agreement between the Registrant and Glenn P. Sblendorio, dated January 4, 2016
|
|
10-Q
|
|
|
|
5/9/2015
|
|
10.2
|
|
|
|
10.31
|
|
+
|
Offer of Employment between the Registrant and Henric Bjarke, dated July 15, 2015
|
|
10-K
|
|
|
|
2/26/2016
|
|
10.31
|
|
|
|
10.32
|
|
+
|
Letter Agreement between the Registrant and Henric Bjarke, dated July 15, 2015
|
|
10-K
|
|
|
|
2/26/2016
|
|
10.32
|
|
|
|
10.33
|
|
+
|
Nonstatutory Stock Option Agreement between the Registrant and Henric Bjarke, dated August 31, 2015
|
|
10-K
|
|
|
|
2/26/2016
|
|
10.33
|
|
|
|
10.34
|
|
+
|
Offer of Employment between the Registrant and Barbara A. Wood, dated October 21, 2013, revised October 22, 2013
|
|
10-K
|
|
|
|
2/26/2016
|
|
10.34
|
|
|
|
10.35
|
|
+
|
Letter Agreement between the Registrant and Barbara A. Wood, dated February 20, 2015
|
|
10-Q
|
|
|
|
5/11/2015
|
|
10.6
|
|
|
|
10.36
|
|
+
|
Form of Indemnification Agreement between the Registrant and each Director and Executive Officer
|
|
10-Q
|
|
|
|
8/5/2016
|
|
10.2
|
|
|
|
10.37
|
|
+
|
2016 Employee Stock Purchase Plan
|
|
S-8
|
|
333-211916
|
|
6/8/2016
|
|
99.1
|
|
|
|
10.38
|
|
*
|
Clinical and Commercial Services Agreement Between the Registrant and Ajinimoto Althea, Inc. dated October 31, 2016
|
|
|
|
|
|
|
|
|
|
Yes
|
|
23.1
|
|
|
Consent of Ernst & Young LLP
|
|
|
|
|
|
|
|
|
|
Yes
|
|
31.1
|
|
|
Certification of principal executive officer pursuant to Rule 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended.
|
|
|
|
|
|
|
|
|
|
|
Yes
|
31.2
|
|
|
Certification of principal financial officer pursuant to Rule 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended.
|
|
|
|
|
|
|
|
|
|
|
Yes
|
32.1
|
|
|
Certification of principal executive officer pursuant to 18 U.S.C. §1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.
|
|
|
|
|
|
|
|
|
|
|
Yes
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Incorporated by Reference
|
|
|
|
|
|||
Exhibit
Number
|
|
Description of Exhibit
|
|
Form
|
|
File
Number
|
|
Date of
Filing
|
|
Exhibit
Number
|
|
Filed
Herewith
|
|
32.2
|
|
|
Certification of principal financial officer pursuant to 18 U.S.C. §1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.INS
|
|
|
XBRL Instance Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.SCH
|
|
|
XBRL Taxonomy Extension Schema Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.CAL
|
|
|
XBRL Taxonomy Calculation Linkbase Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.DEF
|
|
|
XBRL Taxonomy Extension Definition Linkbase Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.LAB
|
|
|
XBRL Taxonomy Label Linkbase Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
101.PRE
|
|
|
XBRL Taxonomy Presentation Linkbase Document.
|
|
|
|
|
|
|
|
|
|
Yes
|
|
|
†
|
|
Confidential treatment has been granted as to certain portions, which portions have been omitted and separately filed with the Securities and Exchange Commission.
|
*
|
|
Confidential treatment has been requested as to certain portions, which portions have been omitted and separately filed with the Securities and Exchange Commission.
|
+
|
|
Management contract or compensatory plan or arrangement filed in response to Item 15(a)(3) of the Instructions to the Annual Report on Form 10-K.
|
1.
|
Separation Date; Post-Employment Consulting Arrangement
–
(a) Executive’s effective date of separation from employment with the Company will be January 13, 2017 (the “ Separation Date ”). As of the Separation Date, the Employment Agreement will terminate and be of no further force or effect; provided, however, that Sections 7, 8 and 9 thereof shall, as amended by this Agreement, remain in full force and effect. Executive hereby resigns, as of the Separation Date, from his employment with the Company, as an officer of the Company and as a member of the Company’s Board of Directors (the “ Board ”). Executive agrees to execute and deliver any documents reasonably necessary to effectuate such resignations, as requested by the Company. Executive shall be paid (x) in accordance with the Company’s regular payroll practices, all unpaid base salary earned through the Separation Date, reimbursement of all unreimbursed business expenses incurred through the Separation Date and any accrued but unused vacation time in accordance with Company policy to which Executive was entitled through the Separation Date and (y) as of the date on which any other Company employees who are eligible to receive an annual bonus for 2016 receive such bonuses, any earned but unpaid annual bonus for 2016 (together, the “ Accrued Benefits ”). As of the Separation Date, all salary payments from the Company will cease and any benefits Executive had as of the Separation Date under Company-provided benefit plans, programs, or practices will terminate, except as required by federal or state law or as otherwise specifically set forth in this Agreement. (b) Upon the Separation Date, the Company and Executive shall enter into the Consulting Agreement. During the Consultation Period (as such term is defined in the Consulting Agreement), any outstanding and unvested equity awards previously granted to Executive by the Company will continue to vest and be exercisable in accordance with the applicable equity plans and award agreements. |
2.
|
Severance Benefits
– On the earlier to occur of the end of the Consultation Period (as such term is defined in the Consulting Agreement) and Executive’s “separation from service” (as defined in
|
a.
|
Salary Continuation
– Commencing on the Company’s first regularly scheduled payroll date that follows the Additional Release Effective Date (as defined in the Additional Release) (the “
Payment Commencement Date
”), the Company will, for a twelve (12) month period (the “
Severance Period
”), provide Executive with severance pay in the form of twelve equal monthly installments of $52,083.33, less all applicable taxes and withholdings.
|
b.
|
Pro-Rata Bonus
–
On the Payment Commencement Date, the Company shall provide Executive with a pro-rata bonus payment of $14,469.18, which is equivalent to (x) sixty-five percent (65%) of Executive’s annualized base salary as of the Separation Date, multiplied by (y) a fraction, the numerator of which is the number of days during calendar year 2017 during which Executive remained employed by the Company and the denominator of which is 365.
|
c.
|
Group Health Insurance
– Should Executive be eligible for and timely elect to continue receiving group health and/or dental insurance coverage under the law known as COBRA, the Company shall, until the earliest of (x) the last day of the Severance Period, (y) the date that Executive is no longer eligible for COBRA continuation coverage, and (z) the end of the calendar month in which Executive becomes eligible to receive group health insurance coverage under another employer’s benefit plan (the “
COBRA Contribution Period
”),
pay on Executive’s behalf the employer share of premium for such coverage at the same rates as from time to time in effect for the Company’s active workforce. Should Executive cease during the Severance Period to be eligible to continue receiving group health and/or dental insurance coverage under COBRA for reasons other than becoming enrolled in another group health plan (or should Executive have exhausted his COBRA coverage prior to the commencement of the Severance Period), the Company shall provide Executive with an additional monthly payment in an amount equal to the monthly employer premium paid during the final month of his COBRA continuation coverage until the earlier of (x) the last day of the Severance Period or (y) the end of the calendar month in which Executive becomes eligible to receive group health or dental insurance coverage under another employer’s benefit plan(s), as applicable. For the avoidance of doubt, (i) the Company’s assistance with health coverage costs shall in all events not extend beyond the Severance Period and (ii) to the extent Executive wishes to remain enrolled in COBRA following the expiration of the COBRA Contribution Period, all premium costs after
the COBRA Contribution Period shall be paid by Executive on a monthly basis during the elected period of health insurance coverage under COBRA for as long as, and to the extent that, he remains eligible for and wishes to remain enrolled in COBRA continuation coverage.
|
d.
|
Equity Exercise
– Executive may exercise any stock options outstanding and vested as of the expiration or termination of the Consultation Period during the applicable period following the expiration or termination of the Consultation Period specified in, and otherwise in accordance with the terms of, the applicable option award agreements and equity plans under which any such outstanding stock options were awarded; provided further, however, that in no event may any stock option be exercised beyond the earlier of (x) the original maximum term specified in the applicable option award agreement, and (y) ten (10) years from the original grant date of such stock option.
|
3.
|
Release of Claims
– In exchange for the consideration set forth in this Agreement, which Executive acknowledges he would not otherwise be entitled to receive, Executive hereby fully, forever, irrevocably and unconditionally releases, remises and discharges the Company, its affiliates, subsidiaries, parent companies, predecessors, and successors, and all of their respective past and present officers, directors, stockholders, partners, members, employees, agents, representatives, plan administrators, attorneys, insurers and fiduciaries (each in their individual and corporate capacities) (collectively, the “
Released Parties
”) from any and all claims, charges, complaints, demands, actions, causes of action, suits, rights, debts, sums of money, costs, accounts, reckonings, covenants, contracts, agreements, promises, doings, omissions, damages, executions, obligations, liabilities, and expenses (including attorneys’ fees and costs), of every kind and nature that Executive ever had or now has against any or all of the Released Parties up to the date on which he signs this Agreement, whether known or unknown, including, but not limited to, any and all claims arising out of or relating to Executive’s employment with, provision of consulting or other services to, separation or termination from, and/or ownership of securities of the Company, including, but not limited to, all claims under Title VII of the Civil Rights Act, the Americans With Disabilities Act, the Age Discrimination in Employment Act, the Genetic Information Nondiscrimination Act, the Family and Medical Leave Act, the Worker Adjustment and Retraining Notification Act, the Rehabilitation Act, Executive Order 11246, Executive Order 11141, the Fair Credit Reporting Act, and the Employee Retirement Income Security Act, all as amended; all claims arising out of the New York Human Rights Law, N.Y. Exec. Law § 290
et seq.
, the New York City Human Rights Law, N.Y.C. Admin. Code § 8-101
et seq.
, N.Y. Civ. Rights Law § 40-c
et seq.
(New York anti-discrimination law), N.Y. Lab. Law § 194
et seq.
(New York equal pay law), N.Y. Lab. Law § 740 (New York whistleblower protection law), and N.Y. Lab. Law § 201-c (New York adoption leave law), all as amended; all claims arising out of the New Jersey Law Against Discrimination, N.J. Stat. Ann. § 10:5-1
et seq.
, the New Jersey Family Leave Act, N.J. Stat. Ann. § 34:11B-1
et seq.
, the New Jersey Conscientious Employee Protection Act, N.J. Stat. Ann. § 34:19-1
et seq.
, N.J. Stat. Ann. § 34:11-56.1
et seq.
(New Jersey equal pay law), and the New Jersey “mini-WARN” Act
(
N.J.S.A. 34:21-1, et seq.), all as amended; all common law claims including, but not limited to, actions in defamation, intentional infliction of emotional distress, misrepresentation, fraud, wrongful discharge, and breach of contract (including, without limitation, all claims arising out of or related to the Employment Agreement);
|
4.
|
Non-Solicitation and Non-Competition Obligations
– Executive acknowledges and reaffirms his non-competition and non-solicitation obligations as set forth in Section 7 of the Employment Agreement (the “
Restrictive Covenant Obligations
”), which Restrictive Covenant Obligations survive his termination of employment and remain in full force and effect; provided, however, that in consideration of this Agreement and the Consulting Agreement, Executive acknowledges and agrees that (a) the duration of the Restrictive Covenant Obligations is amended hereby such that such Restrictive Covenant Obligations shall remain in effect during the Consultation Period and for a period of one (1) year thereafter, and (b) the definition of “Competitive” in Section 7(a)(ii) of the Employment Agreement is amended hereby such that a business will deemed “Competitive” with the Company if it satisfies such definition set forth in Section 7(a)(ii) as of the Separation Date or any date until and ending upon the termination of the Consultation Period.
|
5.
|
Non-Disclosure Obligations
– Executive acknowledges and reaffirms his obligation, except as otherwise permitted by Section 9 below, to keep confidential and not to use or disclose any and all non-public information concerning the Company or any of its subsidiaries that he acquired during the course of his employment with the Company, or may acquire during his service under the Consulting Agreement, including, but not limited to, any non-public information concerning the Company’s or any of its subsidiaries’ business, operations, products, programs, affairs, performance, personnel, technology, science, intellectual property, plans, strategies, approaches, prospects, financial condition or development related matters. Executive also acknowledges his continuing obligations with respect to confidential information and developments as set forth in the Invention and Non-Disclosure Agreement he executed on November 30, 2009 and that is referenced in Section 8 of the Employment Agreement (the “
NDA
”), which, as amended by the last sentence of this Section 5, survives his separation from employment with the Company and remains in full force and effect. Further, in consideration of this Agreement and the Consulting Agreement, Executive acknowledges and agrees that the NDA is amended hereby to apply to his Services for the Company during the Consultation Period, and all references in the NDA to “Employee” are amended hereby to refer as well to “Consultant” and all references to
|
6.
|
Non-Disparagement
– Executive understands and agrees that, except as otherwise permitted by Section 9 below, he will not, in public or private, make any false, disparaging, negative, critical, adverse, derogatory or defamatory statements, whether orally or in writing, including online (including, without limitation, on any social media, networking, or employer review site) or otherwise, to any person or entity, including, but not limited to, any media outlet, industry group, key opinion leader, financial institution, research analyst or current or former employee, board member, consultant, shareholder, client or customer of the Company or any of its subsidiaries, regarding the Company, any of its subsidiaries or any of the other Released Parties, or regarding the Company’s or any of its subsidiaries’ business, operations, products, programs, affairs, performance, personnel, technology, science, intellectual property, plans, strategies, approaches, prospects, financial condition or development related matters. For the avoidance of doubt, the foregoing shall not prevent Executive from stating or repeating factual information with respect to the Company or its assets which is otherwise publicly available. The Company agrees that its Board members and its named executive officers (as determined pursuant to Item 402(a)(3) of Regulation S-K) will not, in public or private, make any false, disparaging, negative, critical, adverse, derogatory or defamatory statements, whether orally or in writing, including online (including, without limitation, on any social media, networking, or employer review site) or otherwise, to any person or entity, including, but not limited to, any media outlet, industry group, key opinion leader, financial institution, research analyst or current or former employee, board member, consultant, shareholder, client or customer of the Company or any of its subsidiaries, regarding Executive; provided, however, that nothing in this Section 6 shall restrict or otherwise limit such Board members or named executive officers from disclosing events or circumstances in such manner as they or the Company deem necessary to comply with or satisfy their or the Company’s disclosure, reporting or other obligations under applicable law.
|
7.
|
Return of Company Property
– Executive confirms that he will, except as specifically instructed otherwise by the Company’s Chief Executive Officer, return to the Company no later than January 19, 2017 all property of the Company, tangible or intangible, including but not limited to keys, files, records (and copies thereof), equipment (including, but not limited to, computer hardware, software and printers, wireless handheld devices, cellular phones, tablets, etc.), Company identification and any other Company-owned property in his possession or control and will leave intact all electronic Company documents, including but not limited to those that he developed or helped to develop during his employment or while performing services under the Consulting Agreement. Executive further agrees that he will, except as specifically instructed otherwise by the Company’s Chief Executive Officer, cancel no later than January 19, 2017 all accounts for his benefit, if any, in the Company’s name, including but not limited to, credit cards, telephone charge cards, cellular phone and/or wireless data accounts and computer accounts. Executive will provide a written and signed certification to the Company no later than January 19, 2017, that he has returned all property and cancelled all accounts as required in compliance with this Section 7.
|
8.
|
Confidentiality
– Executive understands and agrees that, except as otherwise permitted by Section 9 below, the contents of the negotiations and discussions resulting in this Agreement shall be maintained as confidential by Executive and his agents and representatives and shall not be disclosed except as otherwise agreed to in writing by the Company and except to his immediate
|
9.
|
Scope of Disclosure Restrictions
– Nothing in this Agreement prohibits Executive or the Company from communicating with government agencies about possible violations of federal, state, or local laws or otherwise providing information to government agencies, filing a complaint with government agencies, or participating in government agency investigations or proceedings. Executive is not required to notify the Company of any such communications; provided, however, that nothing herein authorizes the disclosure of information Executive obtained through a communication that was subject to the attorney-client privilege. Further, notwithstanding Executive’s confidentiality and nondisclosure obligations, Executive is hereby advised as follows pursuant to the Defend Trade Secrets Act: “An individual shall not be held criminally or civilly liable under any Federal or State trade secret law for the disclosure of a trade secret that (A) is made (i) in confidence to a Federal, State, or local government official, either directly or indirectly, or to an attorney; and (ii) solely for the purpose of reporting or investigating a suspected violation of law; or (B) is made in a complaint or other document filed in a lawsuit or other proceeding, if such filing is made under seal. An individual who files a lawsuit for retaliation by an employer for reporting a suspected violation of law may disclose the trade secret to the attorney of the individual and use the trade secret information in the court proceeding, if the individual (A) files any document containing the trade secret under seal; and (B) does not disclose the trade secret, except pursuant to court order.”
|
10.
|
Cooperation
– Executive agrees that, to the extent permitted by law, he shall, during the Consultation Period and for the longer of eighteen (18) months thereafter or the duration of any matter in which he and/or the Company are defendants or responding parties that is brought or is pending during such eighteen (18) month period, reasonably cooperate with the Company in the investigation, defense or prosecution of any claims or actions which already have been brought, are currently pending, or which may be brought in the future against the Company by a third party or by or on behalf of the Company against any third party, whether before a state or federal court, any state or federal government agency, or a mediator or arbitrator. Executive’s reasonable cooperation in connection with such claims or actions shall include, but not be limited to, being available to meet with the Company’s counsel, at reasonable times and locations designated by the Company, to investigate or prepare the Company’s claims or defenses, to prepare for trial or discovery or an administrative hearing, mediation, arbitration or other proceeding, to provide any relevant information in his possession, and to act as a witness when requested by the Company. The Company will reimburse Executive for all reasonable and documented out of pocket costs that he incurs to comply with this paragraph. Executive further agrees that,
to the extent permitted by law, he will notify the Company promptly in the event that he is served with a subpoena (other than a subpoena issued by a government agency), or
in the event that he is asked to provide a third party (other than a government agency) with information concerning any actual or potential complaint or claim against the Company.
|
11.
|
Business Expenses; Final Compensation; Legal Fees
– Executive acknowledges that he has been reimbursed by the Company for all business expenses incurred in conjunction with the performance of his employment and that no other reimbursements are owed to him. Executive further acknowledges that he has received all compensation due to him from the Company, including, but not limited to, all wages, bonuses and accrued, unused vacation time, and that, other than pursuant to the Consulting Agreement, he is not eligible or entitled to receive any additional payments or consideration from the Company beyond that provided for in Section 2 of
|
12.
|
Amendment and Waiver
– This Agreement and the Additional Release shall be binding upon the Parties and may not be modified in any manner, except by an instrument in writing of concurrent or subsequent date signed by duly authorized representatives of the Parties. This Agreement and the Additional Release are binding upon and shall inure to the benefit of the Parties and their respective agents, assigns, heirs, executors/administrators/personal representatives, and successors. No delay or omission by the Company in exercising any right under this Agreement or the Additional Release shall operate as a waiver of that or any other right. A waiver or consent given by the Company on any one occasion shall be effective only in that instance and shall not be construed as a bar to or waiver of any right on any other occasion.
|
13.
|
Validity
– Should any provision of this Agreement or the Additional Release be declared or be determined by any arbitrator acting pursuant to Section 18 below or court of competent jurisdiction to be illegal or invalid, the validity of the remaining parts, terms or provisions shall not be affected thereby and said illegal or invalid part, term or provision shall be deemed not to be a part of this Agreement or the Additional Release.
|
14.
|
Nature of Agreement
–
Both Parties understand and agree that this Agreement is a separation agreement and does not constitute an admission of liability or wrongdoing on the part of the Company or Executive.
|
15.
|
Time for Consideration and Revocation
–
Executive acknowledges that he was initially presented with this Agreement on January 2, 2017 (the “
Receipt Date
”). Executive understands that this Agreement shall be of no force or effect unless he signs and returns this Agreement on or before January 24, 2017, and does not revoke his acceptance in the subsequent seven (7) day period (the day immediately following expiration of such revocation period, the “
Effective Date
”). Executive further understands that he will not be eligible to receive the Severance Benefits unless he timely signs, returns, and does not revoke the Additional Release.
|
16.
|
Acknowledgments
–
Executive acknowledges that he has been given at least twenty-one (21) days from the Receipt Date to consider this Agreement and the Additional Release (the “
Consideration Period
”), and that the Company is hereby advising him to consult with an attorney of his own choosing prior to signing this Agreement and the Additional Release. Executive further acknowledges and agrees that any changes made to this Agreement or any exhibits or attachments hereto following his initial receipt of this Agreement on the Receipt Date, whether material or immaterial, shall not re-start or affect in any manner the Consideration Period. Executive understands that he may revoke this Agreement and the Additional Release for a period of seven (7) days after he signs each respective agreement by notifying the Company in writing, and that neither agreement shall be effective or enforceable until the expiration of each respective seven (7) day revocation period. In the event the Executive executed this Agreement within less than twenty-one (21) days after the Receipt Date, he acknowledges that such decision was entirely voluntary and that he had the opportunity to consider this Agreement until the end of the twenty-one (21) day period. Executive understands and agrees that by entering into this
|
17.
|
Voluntary Assent
–
Executive affirms that no other promises or agreements of any kind have been made to or with Executive by any person or entity whatsoever to cause him to sign this Agreement or the Additional Release, and that he fully understands the meaning and intent of this Agreement and the Additional Release and that he has been represented by counsel of his own choosing. Executive further states and represents that he has carefully read this Agreement and the Additional Release, understands the contents herein, freely and voluntarily assents to all of the terms and conditions hereof, and signs his name of his own free act.
|
18.
|
Arbitration
– Any dispute between Executive and the Company relating in any way to this Agreement (including the Additional Release), the Consulting Agreement, Executive’s ownership of any securities of the Company or Executive’s relationship with the Company (including without limitation, Executive’s service to the Company as a consultant or the termination thereof, or Executive’s employment with the Company or separation therefrom), including, but not limited to (i) claims alleging unlawful discrimination, harassment, or retaliation on any basis protected by any applicable federal, state, or local law, (ii) claims for wages, bonuses, severance, employee benefits or other compensation, whether pursuant to contract or federal or state wage and hour laws, (iii) common law claims, including, but not limited to, tort claims, wrongful discharge claims, contract claims, defamation claims and unfair business practices claims, and (iv) any claim under any statute, law, or ordinance not expressly set forth above (“
Arbitrable Claims
”) shall be resolved by binding arbitration before a neutral arbitrator as provided in this Section 18 (the “
Arbitration
”). For the avoidance of doubt, (1) either Party shall be entitled to elect to seek from a court injunctive relief, specific performance or a declaration of rights with respect to any claim relating to any dispute that could result in irreparable harm or loss to such Party and (2) Arbitrable Claims shall not include any claim for which a Party seeks from a court such injunctive relief, specific performance or a declaration of rights pursuant to clause (1) immediately above. Further, and for the avoidance of doubt, nothing herein prevents Executive from filing a charge with, cooperating with, or participating in any proceeding or investigation before the EEOC or a state fair employment practices agency (except that Executive acknowledges that he may not recover any monetary benefits in connection with any such charge, proceeding or investigation, and further waives any rights or claims to any payment, benefit, attorneys’ fees or other remedial relief in connection with any such charge, proceeding or investigation); provided, however, that nothing in the immediately preceding parenthetical constitutes a waiver of any of Executive’s rights or claims in connection with any arbitration proceeding hereunder. The Arbitration shall take place in New York, New York, unless otherwise agreed by Executive and the Company. The Arbitration shall be administered by the American Arbitration Association (“
AAA
”) under its Employment Arbitration Rules and Mediation Procedures (the “
Rules
”), shall be final and binding upon Executive and the Company, and shall be the exclusive remedy for all claims covered by this arbitration provision. The Arbitration shall be presided over by a single arbitrator, who shall be selected by Executive and the Company in accordance with the AAA Rules and shall be experienced in employment law and/or securities law, as may be applicable. The arbitrator shall render an award and written opinion in the form typically rendered in employment arbitrations within the time provided in the Rules. The Company shall pay any filing fee and the fees and costs of the arbitrator; provided, however, that if Executive is the party initiating the Arbitration, Executive will pay an amount equivalent to the
|
19.
|
Governing Law
– This Agreement and the Additional Release shall be interpreted and construed by the laws of the State of New York, without regard to conflict of laws provisions. Each of the Company and Executive hereby irrevocably submits to and acknowledges and recognizes the exclusive jurisdiction and venue of the courts of the State of New York located in New York County, or if appropriate, the United States District Court for the Southern District of New York (which courts, for purposes of this Agreement and the Additional Release, are the only courts of competent jurisdiction), over any Exempted Action or claim that is not an Arbitrable Claim, and waives any objection to laying venue in any such action or proceeding in such courts, waives any objection that such courts are an inconvenient forum or do not have jurisdiction over either party, and agrees that service of process upon such party in any such action or proceeding shall be effective if such process is given as a notice in accordance with the terms of this Agreement.
|
20.
|
Entire Agreement
– This Agreement, including all exhibits and attachments hereto, contains and constitutes the entire understanding and agreement between the Parties hereto with respect to Executive’s separation from the Company, severance benefits and the settlement of claims against the Company, and cancel all previous oral and written negotiations, agreements, commitments and writings in connection therewith; provided, however, that nothing in this Section 20 shall modify, cancel or supersede Executive’s obligations set forth in Sections 4 and 5 above.
|
21.
|
Tax Acknowledgement
– In connection with the Severance Benefits provided to Executive pursuant to this Agreement and the Additional Release, the Company shall withhold and remit to the tax authorities the amounts required under applicable law, and Executive shall be responsible for all applicable taxes owed by him with respect to such Severance Benefits under applicable law. Executive acknowledges that he is not relying upon the advice or representation of the Company with respect to the tax treatment of any of the Severance Benefits set forth in this Agreement.
|
22.
|
Counterparts
– This Agreement may be executed in several counterparts, each of which shall be deemed to be an original, but all of which together will constitute one and the same Agreement. Facsimile and PDF signatures shall be deemed to be of equal force and effect as originals.
|
Samir Patel, M.D.
____________
/s/ Samir C. Patel
____________
|
OPHTHOTECH CORPORATION
By:
__/s/ David R. Guyer
_________________
Name: _ David R. Guyer ____________________ Title: _ CEO & Chairman of the Board _______ |
|
|
Confidential Materials omitted and filed separately with the
Securities and Exchange Commission. Double asterisks denote omissions.
|
|
Exhibit 10.38
|
|
|
CONFIDENTIAL
|
1.
|
DEFINITIONS.
|
CONFIDENTIAL
|
2
|
|
CONFIDENTIAL
|
3
|
|
CONFIDENTIAL
|
4
|
|
CONFIDENTIAL
|
5
|
|
2.
|
VALIDATION AND NON-PRODUCTION SERVICES.
|
CONFIDENTIAL
|
6
|
|
CONFIDENTIAL
|
7
|
|
CONFIDENTIAL
|
8
|
|
CONFIDENTIAL
|
9
|
|
4.
|
PRODUCTION – FORECASTS AND ORDERS
|
CONFIDENTIAL
|
10
|
|
5.
|
PRODUCTION – RELEASE AND ACCEPTANCE
|
CONFIDENTIAL
|
11
|
|
CONFIDENTIAL
|
12
|
|
CONFIDENTIAL
|
13
|
|
CONFIDENTIAL
|
14
|
|
CONFIDENTIAL
|
15
|
|
6.
|
PRODUCTION – DELIVERY & STORAGE
|
CONFIDENTIAL
|
16
|
|
7.
|
PAYMENT TERMS FOR PRODUCTION.
|
CONFIDENTIAL
|
17
|
|
8.
|
TERM AND TERMINATION.
|
CONFIDENTIAL
|
18
|
|
CONFIDENTIAL
|
19
|
|
9.
|
PRODUCTION RECORDS, AUDIT AND COMPLIANCE.
|
CONFIDENTIAL
|
20
|
|
CONFIDENTIAL
|
21
|
|
10.
|
CLIENT PRODUCT RECALLS.
|
11.
|
FORCE MAJEURE; SAFETY STOCK; RISK MANAGEMENT.
|
CONFIDENTIAL
|
22
|
|
12.
|
CHANGES IN PRODUCTION AND CLIENT PRODUCT.
|
13.
|
CONFIDENTIALITY.
|
CONFIDENTIAL
|
23
|
|
CONFIDENTIAL
|
24
|
|
14.
|
INVENTIONS; INTELLECTUAL PROPERTY.
|
CONFIDENTIAL
|
25
|
|
CONFIDENTIAL
|
26
|
|
15.
|
REPRESENTATIONS, WARRANTIES AND COVENANTS.
|
CONFIDENTIAL
|
27
|
|
CONFIDENTIAL
|
28
|
|
16.
|
LIMITATION OF LIABILITY.
|
17.
|
INDEMNIFICATION.
|
CONFIDENTIAL
|
29
|
|
18.
|
INSURANCE.
|
CONFIDENTIAL
|
30
|
|
19.
|
GENERAL PROVISIONS.
|
CONFIDENTIAL
|
31
|
|
CONFIDENTIAL
|
32
|
|
CONFIDENTIAL
|
33
|
|
OPHTHOTECH CORPORATION
|
AJINOMOTO ALTHEA, INC.
|
||
|
|
|
|
By:
|
/s/ Glenn Sblendorio
|
By:
|
/s/ J. David Enloe Jr.
|
|
|
|
|
Name:
|
Glenn Sblendorio
|
Name:
|
J. David Enloe Jr.
|
|
|
|
|
Title:
|
EVP & CFO
|
Title:
|
President & CEO
|
CONFIDENTIAL
|
34
|
|
•
|
Ophthotech Purchase Order number 01-0415, dated July 1, 2015
|
CONFIDENTIAL
|
35
|
|
1.
|
Ophthotech PO #000787 dated September 13, 2016 for Fovista Price Per Unit Agreement for Rota 150 Filling Line for 2017 Production of [**] Lots of Fovista
|
CONFIDENTIAL
|
36
|
|
Note:
|
The Specifications appearing below are current as of September 30, 2016. Any changes to these Specifications will be handled through the change control process in accordance with this Agreement and the Quality Agreement and approved by Ophthotech. Those approved changes will replace the Specifications set forth herein as of the time of this Agreement’s execution.
|
CONFIDENTIAL
|
37
|
|
CONFIDENTIAL
|
38
|
|
•
|
Stage 1 Bulk Compound formulation pre filtration –
Actual yield is based on (volume of product * concentration divided by theoretical yield) * 100%. Theoretical yield is based on Bulk Compound used to formulate solution, water content purity and concentration/volume.
|
•
|
Stage 2 Filtered drug product solution
– Actual yield is based on weight of product in bag (total minus tare) divided by theoretical * 100%. Theoretical yield = actual yield from stage 1 minus known filter loss.
|
•
|
Stage 3 Filling yield
– Number of vials filled during Production ( including available for testing, retains, and visual inspection) * (average mass per vial) divided by (mass of filtered product in bag – (mass of prime, calibration, and weight check vials + mass of hold up)) x 100%
|
•
|
Stage 4 Acceptable vials
– Number of final acceptable vials divided by theoretical yield x 100%. Theoretical yield = actual vials from Stage 3 minus vials taken for stability and testing.
|
CONFIDENTIAL
|
39
|
|
CONFIDENTIAL
|
40
|
|
(1)
|
Registration Statement (Form S-8 No. 333-211916) pertaining to the 2016 Employee Stock Purchase Plan of Ophthotech Corporation effective June 8, 2016,
|
(2)
|
Registration Statement (Form S-8 No. 333-208893) pertaining to the 2013 Stock Incentive Plan and Inducement Stock Option Grant of Ophthotech Corporation, effective January 6, 2016,
|
(3)
|
Registration Statement (Form S-8 No. 333-202438) pertaining to the 2013 Stock Incentive Plan and inducement stock options of Ophthotech Corporation, effective March 2, 2015,
|
(4)
|
Registration Statement (Form S-8 No. 333-193694) pertaining to the 2013 Stock Incentive Plan of Ophthotech Corporation, effective January 31, 2014,
|
(5)
|
Registration Statement (Form S-8 No. 333-191767) pertaining to the 2013 Stock Incentive Plan and Amended and Restated 2007 Stock Incentive Plan of Ophthotech Corporation, effective October 16, 2013,
|
|
1.
|
I have reviewed this Annual Report on Form 10-K for the fiscal year ended December 31, 2016 of Ophthotech Corporation;
|
2.
|
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
|
3.
|
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
|
4.
|
The registrant's other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
|
a.
|
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
|
b.
|
Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
|
c.
|
Evaluated the effectiveness of the registrant's disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
|
d.
|
Disclosed in this report any change in the registrant's internal control over financial reporting that occurred during the registrant's most recent fiscal quarter (the registrant's fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant's internal control over financial reporting; and
|
5.
|
The registrant's other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant's auditors and the audit committee of the registrant's board of directors (or persons performing the equivalent functions):
|
a.
|
All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant's ability to record, process, summarize and report financial information; and
|
b.
|
Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant's internal control over financial reporting.
|
Date: February 28, 2017
|
|
By:
|
|
/s/ DAVID R. GUYER
|
|
|
|
|
David R. Guyer, M.D.
|
|
|
|
|
Chief Executive Officer
|
|
|
|
|
(Principal Executive Officer)
|
|
Date: February 28, 2017
|
|
By:
|
|
/s/ GLENN P. SBLENDORIO
|
|
|
|
|
Glenn P. Sblendorio
President and Chief Financial Officer
(Principal Financial Officer)
|
|
Date: February 28, 2017
|
|
By:
|
|
/s/DAVID R. GUYER
|
|
|
|
|
David R. Guyer, M.D.
Chief Executive Officer
(Principal Executive Officer) |
|
Date: February 28, 2017
|
|
By:
|
|
/s/ GLENN P. SBLENDORIO
|
|
|
|
|
Glenn P. Sblendorio
President and Chief Financial Officer
(Principal Financial Officer)
|
|