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Delaware
(State or other jurisdiction of
incorporation or organization)
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27-0072226
(I.R.S. Employer
Identification No.)
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128 Sidney Street
Cambridge, Massachusetts
(Address of principal executive offices)
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02139
(Zip Code)
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Title of 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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NASDAQ Global Market
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Large accelerated filer
ý
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Accelerated filer
o
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Non-accelerated filer
o
(Do not check if a
smaller reporting company)
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Smaller reporting company
o
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Page
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our ongoing and planned preclinical studies and clinical trials;
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clinical trial data and the timing of results of our ongoing clinical trials;
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our plans to develop and commercialize dalantercept and ACE-083, and our and Celgene's plans to develop and commercialize luspatercept and sotatercept;
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the potential benefits of strategic partnership agreements and our ability to enter into selective strategic partnership arrangements;
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the timing of, and our and Celgene's ability to, obtain and maintain regulatory approvals for our therapeutic candidates;
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the rate and degree of market acceptance and clinical utility of any approved therapeutic candidate, particularly in specific patient populations;
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our ability to quickly and efficiently identify and develop therapeutic candidates;
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our commercialization, marketing and manufacturing capabilities and strategy;
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our intellectual property position; and
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our estimates regarding our results of operations, financial condition, liquidity, capital requirements, prospects, growth and strategies.
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Luspatercept in Rare Blood Disorders
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◦
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Complete patient enrollment in the MEDALIST Phase 3 clinical trial in the second half of 2017.
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◦
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Evaluate and design a clinical and regulatory strategy for luspatercept in first-line lower risk MDS patients.
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◦
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Complete patient enrollment in the BELIEVE Phase 3 clinical trial in the second half of 2017.
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Initiate a Phase 2 trial in patients with non-transfusion dependent beta-thalassemia by the end of 2017.
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Initiate a Phase 2 trial in patients with myelofibrosis by the end of 2017.
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ACE-083 in Neuromuscular Disease
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◦
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Present initial topline results from the open label, dose-escalation stage of the Phase 2 study in FSHD in late 2017.
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Initiate a Phase 2 clinical trial in a second neuromuscular disease in 2017.
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Pipeline Expansion
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◦
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Initiate a Phase 1 healthy volunteer study with ACE-2494 in 2017.
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◦
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Host an investor and analyst research day to discuss ongoing preclinical research and potential future disease areas in 2017.
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Dalantercept in Advanced Renal Cell Carcinoma
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◦
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Present topline progression-free survival (PFS) results from Phase 2 DART study in advanced renal cell carcinoma patients in the second half of 2017.
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Recombinant erythropoietin and other erythropoiesis stimulating agents. Although these agents are not approved to treat anemia in MDS, current practice guidelines include the use of erythropoiesis stimulating agents and granulocyte colony stimulating factor agents (G-CSF) to treat patients with MDS. Additionally, Amgen is currently studying erythropoiesis stimulating agent, Aranesp® and Janssen Pharmaceuticals is studying erythropoiesis stimulating agent Eprex® in Phase 3 clinical trials in Europe for treatment of anemia in patients with lower risk MDS.
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Red blood cell transfusion and iron chelation therapy, including Exjade® and Jadenu
TM
, which is used to treat anemia and iron overload in patients with MDS.
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Immunomodulators, including Celgene's approved product, Revlimid® (lenalidomide), for the treatment of anemia of certain MDS patients.
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Eli Lilly and Company is studying TGF-beta receptor I kinase inhibitor, LY2157299 in a Phase 2 study in lower risk MDS patients with anemia.
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Other therapies in development, including: an oral form of the hypomethylating agent azacitidine, known as CC-486, being developed by Celgene to treat patients with transfusion dependent anemia and thrombocytopenia due to lower risk MDS, which is currently in Phase 3 clinical trials in the United States and Europe; an anti-cancer therapy being developed by Onconova to treat patients with MDS; a telemorase inhibitor, imetelstat, being studied by Geron and Janssen in a Phase 2/3 study in lower risk MDS patients; and a CD95 ligand inhibitor, APG101, being studied by Apogenix in a Phase 1 study in transfusion dependent, lower risk MDS patients.
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Red blood cell transfusions and iron chelation therapy, such as Novartis's oral iron chelating agents, Exjade® and Jadenu™.
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Fetal hemoglobin stimulating agents, such as hydroxyurea, which are primarily used to treat patients with anemia from sickle cell disease, are sometimes used to treat patients with beta-thalassemia.
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Hematopoietic stem cell transplant treatment is given to a small percentage of patients with beta-thalassemia, since it requires a sufficiently well-matched source of donor cells. Certain academic centers around the world are seeking to develop improvements to this approach.
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Other therapies in development, including gene therapy and genome editing are being developed by several different groups, including bluebird bio, Inc., Memorial Sloan Kettering Cancer Center, GlaxoSmithKline plc, and Sangamo BioSciences Inc. in collaboration with Biogen Idec.
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Nivolumab, an anti-PD1 monoclonal antibody developed by Bristol-Myers Squibb, cabozantinib, an inhibitor of VEGF and MET receptors developed by Exelixis, and lenvatinib, a multireceptor tyrosine kinase inhibitor, developed by Eisai, in combination with everolimus, were approved in 2015, 2016 and 2016, respectively, to treat patients with renal cell carcinoma. Additionally, nivolumab is being studied in combination with other treatments for renal cell carcinoma. Merck is also studying pembrolizumab, an anti-PD1 antibody, is also being studied as monotherapy and in combination therapy in renal cell carcinoma.
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Tracon is developing TRC105, an antibody to endoglin, which is a protein in the TGF-beta superfamily that is overexpressed on endothelial cells and plays a role in angiogenesis. TRC105 is currently being studied in Phase 1 and Phase 2 clinical trials for the treatment of multiple solid tumor types, including soft tissue sarcoma, renal cell carcinoma, glioblastoma, hepatocellular carcinoma and colorectal cancer, in combination with approved VEGF inhibitors.
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Other non-VEGF angiogenesis inhibitors in development, which also have the potential to be combined with VEGF pathway inhibitors or used independently of VEGF pathway inhibitors to inhibit angiogenesis. Amgen, Regeneron, MedImmune, and OncoMed Pharmaceuticals are each developing non-VEGF angiogenesis inhibitors.
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completion of preclinical laboratory tests, animal studies and formulation studies conducted according to Good Laboratory Practices, or GLPs, and other applicable regulations;
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submission to the FDA of an Investigational New Drug application or IND, which must become effective before human clinical trials may commence;
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completion of adequate and well-controlled human clinical trials in accordance with Good Clinical Practices, or GCPs, to establish that the biological product is "safe, pure and potent", which is analogous to the safety and efficacy approval standard for a chemical drug product for its intended use;
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submission to the FDA of a BLA;
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satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the product is produced to assess compliance with applicable current Good Manufacturing Practice requirements, or cGMPs; and
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FDA review of the BLA and issuance of a biologics license which is the approval necessary to market a therapeutic candidate.
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the scope, progress, results and costs of researching and developing our other therapeutic candidates, and conducting preclinical studies and clinical trials;
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the timing of, and the costs involved in, obtaining regulatory approvals for our other therapeutic candidates if clinical trials are successful;
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the cost of commercialization activities for our other therapeutic candidates, if any of these therapeutic candidates is approved for sale, including marketing, sales and distribution costs;
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the cost of manufacturing our other therapeutic candidates for clinical trials in preparation for regulatory approval and in preparation for commercialization;
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our ability to establish and maintain strategic partnerships, licensing or other arrangements and the financial terms of such agreements;
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the costs involved in preparing, filing, prosecuting, maintaining, defending and enforcing patent claims, including litigation costs and the outcome of such litigation; and
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the timing, receipt, and amount of sales of, or royalties on, our future products, if any.
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delays by us or our current or future partners in reaching a consensus with regulatory agencies on trial design;
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delays in reaching agreement on acceptable terms with prospective clinical research organizations, or CROs, and clinical trial sites;
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delays in obtaining required Institutional Review Board, or IRB, approval at each clinical trial site;
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delays in recruiting suitable patients to participate in clinical trials;
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imposition of a clinical hold by regulatory agencies for any reason, including safety or manufacturing concerns or after an inspection of clinical operations or trial sites;
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failure by CROs, other third parties or us or our current or future partners to adhere to clinical trial requirements;
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failure to perform in accordance with the FDA's good clinical practices, or GCP, or applicable regulatory guidelines in other countries;
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delays in the testing, validation, manufacturing and delivery of the therapeutic candidates to the clinical sites;
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delays caused by patients not completing participation in a trial or not returning for post-treatment follow-up;
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clinical trial sites or patients dropping out of a trial;
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occurrence of serious adverse events in clinical trials that are associated with the therapeutic candidates that are viewed to outweigh its potential benefits; or
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changes in regulatory requirements and guidance that require amending or submitting new clinical protocols.
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restrictions on the marketing or manufacturing of the product, withdrawal of the product from the market, or voluntary or mandatory product recalls;
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fines, warning letters, or holds on clinical trials;
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refusal by the FDA to approve pending applications or supplements to approved applications filed by us or our partners, 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; and
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injunctions or the imposition of civil or criminal penalties.
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Celgene has wide discretion in determining the efforts and resources that it will apply to its partnership with us. The timing and amount of any development milestones, and downstream commercial milestones and royalties that we may receive under such partnership will depend on, among other things, the efforts, allocation of resources and successful development and commercialization of these therapeutic candidates by Celgene.
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Celgene may develop and commercialize, either alone or with others, products that are similar to or competitive with the therapeutic candidates that are the subject of its partnerships with us. For example, Celgene is currently commercializing and/or developing certain of its existing products, including lenalidomide and azacitidine, for certain MDS patients for which luspatercept is also being developed.
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Celgene may terminate its partnership with us without cause and for circumstances outside of our control, which could make it difficult for us to attract new strategic partners or adversely affect how we are perceived in scientific and financial communities.
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Celgene may develop or commercialize our therapeutic candidates in such a way as to elicit litigation that could jeopardize or invalidate our intellectual property rights or expose us to potential liability.
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Celgene may not comply with all applicable regulatory requirements, or may fail to report safety data in accordance with all applicable regulatory requirements.
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If Celgene were to breach its arrangements with us, we may need to enforce our right to terminate the agreement in legal proceedings, which could be costly and cause delay in our ability to receive rights back to the relevant therapeutic candidates. If we were to terminate an agreement with Celgene due to Celgene's breach or Celgene terminated the agreement without cause, the development and commercialization of sotatercept and luspatercept could be delayed, curtailed or terminated because we may not have sufficient financial resources or capabilities to continue development and commercialization of these candidates on our own if we choose not to, or are unable to, enter into a new collaboration for these candidates.
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Celgene may enter into one or more transactions with third parties, including a merger, consolidation, reorganization, sale of substantial assets, sale of substantial stock or other change in control, which could divert the attention of its management and adversely affect Celgene's ability to retain and motivate key personnel who are important to the continued development of the programs under the strategic partnership with us. In addition, the third-party to any such transaction could determine to reprioritize Celgene's development programs such that Celgene ceases to diligently pursue the development of our programs and/or cause the respective partnership with us to terminate.
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the efficacy and safety of the product, as demonstrated in clinical trials;
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the clinical indications for which the product is approved and the label approved by regulatory authorities for use with the product, including any warnings that may be required on the label;
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acceptance by physicians and patients of the product as a safe and effective treatment;
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decisions by healthcare organizations to utilize the product;
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the cost, safety and efficacy of treatment in relation to alternative treatments;
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the availability of adequate reimbursement and pricing by third party payers and government authorities;
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the continued projected growth of drug markets in our various indications;
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relative convenience and ease of administration;
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the prevalence and severity of adverse side effects; and
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the effectiveness of our and our current or future partners' sales and marketing efforts.
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a covered benefit under its health plan;
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safe, effective and medically necessary;
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appropriate for the specific patient;
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cost-effective; and
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neither experimental nor investigational.
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public and market perceptions of our future business prospects, including future revenue and profitability;
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the demand for any drug products for which we may obtain regulatory approval;
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our ability to set a price that we believe is fair for our products;
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our ability to obtain coverage and reimbursement approval for a product;
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our ability to generate revenues and achieve or maintain profitability; and
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the level of taxes that we are required to pay.
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regulators or institutional review boards 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 have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites;
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clinical trials of our therapeutic candidates may produce negative or inconclusive results, 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 therapeutic candidates 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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third-party contractors 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 might have to suspend or terminate clinical trials of our therapeutic candidates for various reasons, including a finding that the participants are being exposed to unacceptable health risks;
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regulators, institutional review boards, or the data safety monitoring board for such trials may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks;
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the cost of clinical trials of our therapeutic candidates may be greater than we anticipate;
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the supply or quality of our therapeutic candidates or other materials necessary to conduct clinical trials of our therapeutic candidates may be insufficient or inadequate; and
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our therapeutic candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators or institutional review boards to suspend or terminate the trials.
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be delayed in obtaining or be unable to obtain marketing approval for our therapeutic candidates;
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obtain approval for indications or patient populations that are not as broad as intended or desired;
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be required to provide a medication guide outlining the risks of such side effects for distribution to patients;
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obtain approval with labeling that includes significant use or distribution restrictions or safety warnings, including boxed warnings;
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suffer reputational harm;
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be sued and held liable for harm caused to patients;
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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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injury to our reputation;
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withdrawal of clinical trial participants;
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costs to defend the related litigations;
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a diversion of management's time and our resources;
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substantial monetary awards to trial participants or patients;
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product recalls, withdrawals, or labeling, marketing or promotional restrictions;
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loss of revenue;
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the inability to commercialize our therapeutic candidates; and
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a decline in our stock price.
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results of clinical trials of our therapeutic candidates, including sotatercept, luspatercept, dalantercept and ACE-083;
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the timing of the release of results of our clinical trials that are being conducted by Celgene;
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results of clinical trials of our competitors' products;
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regulatory actions with respect to our products or our competitors' products;
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actual or anticipated fluctuations in our financial condition and operating results;
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publication of research reports by securities analysts about us or our competitors or our industry;
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our failure or the failure of our competitors to meet analysts' projections or guidance that we or our competitors may give to the market;
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additions and departures of key personnel;
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strategic decisions by us or our competitors, such as acquisitions, divestitures, spin-offs, joint ventures, strategic investments or changes in business strategy;
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the passage of legislation or other regulatory developments affecting us or our industry;
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fluctuations in the valuation of companies perceived by investors to be comparable to us;
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sales of our common stock by us, our insiders or our other stockholders;
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speculation in the press or investment community;
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announcement or expectation of additional financing efforts;
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changes in accounting principles;
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terrorist acts, acts of war or periods of widespread civil unrest;
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natural disasters and other calamities;
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actual or perceived changes in current or future market conditions for biopharmaceutical stocks; and
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changes in general market and economic conditions.
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authorize "blank check" preferred stock, which could be issued by our board of directors without stockholder approval and may contain voting, liquidation, dividend and other rights superior to our common stock;
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create a classified board of directors whose members serve staggered three-year terms;
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specify that special meetings of our stockholders can be called only by our board of directors;
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prohibit stockholder action by written consent;
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establish an advance notice procedure for stockholder approvals to be brought before an annual meeting of our stockholders, including proposed nominations of persons for election to our board of directors;
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provide that our directors may be removed only for cause;
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provide that vacancies on our board of directors may be filled only by a majority of directors then in office, even though less than a quorum;
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specify that no stockholder is permitted to cumulate votes at any election of directors;
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expressly authorize our board of directors to modify, alter or repeal our amended and restated by-laws; and
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require supermajority votes of the holders of our common stock to amend specified provisions of our restated certificate of incorporation and amended and restated by-laws
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Common Stock Price
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||||||||||||||
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2016
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2015
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||||||||||||
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High
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Low
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High
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Low
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||||||||
First Quarter
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$
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48.05
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$
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22.67
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$
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43.00
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|
$
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35.11
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Second Quarter
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$
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37.67
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$
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25.49
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$
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37.90
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|
|
$
|
26.94
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Third Quarter
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$
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40.70
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$
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28.29
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|
|
$
|
36.31
|
|
|
$
|
20.00
|
|
Fourth Quarter
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$
|
41.69
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|
|
$
|
24.77
|
|
|
$
|
50.86
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|
|
$
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21.93
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|
(1)
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This performance graph shall not be deemed "soliciting material" or to be "filed" with the SEC for purposes of Section 18 of the Securities and Exchange Act of 1934, as amended, or otherwise subject to the liabilities under that Section, and shall not be deemed incorporated by reference into any filing of Acceleron Pharma Inc. under the Securities Act of 1933, as amended.
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(2)
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$100 invested on September 19, 2013 in stock, or on August 31, 2013 in each index, including reinvestment of dividends.
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•
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In December 2016, we issued
271,616
shares of common stock upon the cashless exercise of warrants to purchase
332,211
shares of common stock.
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Year Ended December 31,
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||||||||||||||||||
(in thousands, except per share data)
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2016
|
|
2015
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2014
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2013
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|
2012
|
||||||||||
Consolidated Statements of Operations Data:
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|
|
|
|
|
|
|
|
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||||||||||
Revenue:
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||
Collaboration revenue:
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||
License and milestone
|
$
|
15,550
|
|
|
$
|
1,184
|
|
|
$
|
1,673
|
|
|
$
|
43,948
|
|
|
$
|
9,696
|
|
Cost-sharing, net
|
12,221
|
|
|
16,913
|
|
|
12,959
|
|
|
13,282
|
|
|
5,558
|
|
|||||
Total revenue
|
27,771
|
|
|
18,097
|
|
|
14,632
|
|
|
57,230
|
|
|
15,254
|
|
|||||
Costs and expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Research and development
|
68,580
|
|
|
58,404
|
|
|
50,897
|
|
|
36,051
|
|
|
35,319
|
|
|||||
Litigation settlement
|
—
|
|
|
—
|
|
|
5,000
|
|
|
—
|
|
|
—
|
|
|||||
General and administrative
|
25,297
|
|
|
20,572
|
|
|
14,199
|
|
|
14,227
|
|
|
8,824
|
|
|||||
Total costs and expenses
|
93,877
|
|
|
78,976
|
|
|
70,096
|
|
|
50,278
|
|
|
44,143
|
|
|||||
(Loss) income from operations
|
(66,106
|
)
|
|
(60,879
|
)
|
|
(55,464
|
)
|
|
6,952
|
|
|
(28,889
|
)
|
|||||
Total other income (expense), net
|
9,116
|
|
|
(3,015
|
)
|
|
4,205
|
|
|
(28,850
|
)
|
|
(3,693
|
)
|
|||||
Loss before income taxes
|
(56,990
|
)
|
|
$
|
(63,894
|
)
|
|
(51,259
|
)
|
|
$
|
(21,898
|
)
|
|
(32,582
|
)
|
|||
Income tax provision
|
(24
|
)
|
|
—
|
|
|
$
|
—
|
|
|
—
|
|
|
—
|
|
||||
Net loss
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
|
$
|
(21,898
|
)
|
|
$
|
(32,582
|
)
|
Reconciliation of net loss to net loss applicable to common stockholders:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Net loss
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
|
$
|
(21,898
|
)
|
|
$
|
(32,582
|
)
|
Accretion of dividends, interest, redemption value and issuance costs on redeemable convertible preferred stock
|
—
|
|
|
—
|
|
|
—
|
|
|
(19,870
|
)
|
|
(27,061
|
)
|
|||||
Gain on extinguishment of redeemable convertible preferred stock
|
—
|
|
|
—
|
|
|
—
|
|
|
2,765
|
|
|
—
|
|
|||||
Net loss applicable to common stockholders-basic and diluted
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
|
$
|
(39,003
|
)
|
|
$
|
(59,643
|
)
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Net loss per share applicable to common stockholders-basic and diluted(1)
|
$
|
(1.52
|
)
|
|
$
|
(1.92
|
)
|
|
$
|
(1.63
|
)
|
|
$
|
(4.15
|
)
|
|
$
|
(24.84
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||
Weighted-average number of common shares used in computing net loss per share applicable to common stockholders-basic and diluted
|
37,430
|
|
|
33,303
|
|
|
31,515
|
|
|
9,407
|
|
|
2,401
|
|
|
As of December 31,
|
||||||||||||||||||
(in thousands)
|
2016
|
|
2015
|
|
2014
|
|
2013
|
|
2012
|
||||||||||
Consolidated Balance Sheet Data:
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||
Cash and cash equivalents
|
$
|
20,950
|
|
|
$
|
27,783
|
|
|
$
|
176,460
|
|
|
$
|
113,163
|
|
|
$
|
39,611
|
|
Short and long-term investments
|
213,432
|
|
|
108,198
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|||||
Total assets
|
247,647
|
|
|
146,337
|
|
|
186,296
|
|
|
123,732
|
|
|
49,212
|
|
|||||
Total current liabilities
|
16,149
|
|
|
14,491
|
|
|
9,253
|
|
|
18,162
|
|
|
38,802
|
|
|||||
Long-term deferred revenue
|
3,704
|
|
|
4,239
|
|
|
4,816
|
|
|
5,620
|
|
|
6,760
|
|
|||||
Long-term deferred rent
|
953
|
|
|
1,157
|
|
|
1,818
|
|
|
2,337
|
|
|
2,837
|
|
|||||
Long-term notes payable
|
—
|
|
|
—
|
|
|
—
|
|
|
9,048
|
|
|
16,525
|
|
|||||
Warrants to purchase redeemable convertible preferred stock
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
1,422
|
|
|||||
Warrants to purchase common stock
|
1,244
|
|
|
17,187
|
|
|
14,124
|
|
|
30,753
|
|
|
5,229
|
|
|||||
Redeemable convertible preferred stock
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
268,610
|
|
|||||
Total stockholder's equity (deficit)
|
225,597
|
|
|
109,263
|
|
|
156,285
|
|
|
57,812
|
|
|
(290,973
|
)
|
(1)
|
See Note 2 within the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K for a description of the method used to calculate basic and diluted net (loss) income per common share.
|
•
|
conduct clinical trials for dalantercept, ACE-083 and ACE-2494;
|
•
|
continue our preclinical studies and potential clinical development efforts of our existing and new preclinical therapeutic candidates;
|
•
|
continue research activities for the discovery of new therapeutic candidates;
|
•
|
manufacture therapeutic candidates for our preclinical studies and clinical trials;
|
•
|
seek regulatory approval for our therapeutic candidates; and
|
•
|
operate as a public company.
|
•
|
direct employee-related expenses, including salaries, benefits, travel and stock-based compensation expense of our research and development personnel;
|
•
|
expenses incurred under agreements with clinical research organizations, or CROs, and investigative sites that will conduct our clinical trials;
|
•
|
the cost of acquiring and manufacturing preclinical and clinical study materials and developing manufacturing processes;
|
•
|
allocated facilities, depreciation, and other expenses, which include rent and maintenance of facilities, insurance and other supplies;
|
•
|
expenses associated with obtaining and maintaining patents; and
|
•
|
costs associated with preclinical activities and regulatory compliance.
|
•
|
the scope, rate of progress, and expense of our ongoing, as well as any additional, clinical trials and other research and development activities;
|
•
|
future clinical trial results;
|
•
|
potential changes in government regulation; and
|
•
|
the timing and receipt of any regulatory approvals.
|
|
Year ended December 31,
|
||||||||||
(in thousands)
|
2016
|
|
2015
|
|
2014
|
||||||
Luspatercept(1)
|
6,945
|
|
|
9,744
|
|
|
7,944
|
|
|||
Dalantercept
|
6,757
|
|
|
8,864
|
|
|
7,526
|
|
|||
ACE-083
|
4,973
|
|
|
3,293
|
|
|
5,111
|
|
|||
ACE-2494
|
1,045
|
|
|
—
|
|
|
—
|
|
|||
Total direct research and development expenses
|
19,720
|
|
|
21,901
|
|
|
20,581
|
|
|||
Other expenses(2)
|
48,860
|
|
|
36,503
|
|
|
30,316
|
|
|||
Total research and development expenses
|
$
|
68,580
|
|
|
$
|
58,404
|
|
|
$
|
50,897
|
|
(1)
|
Expenses associated with luspatercept are reimbursed 100% by Celgene. These reimbursements are recorded as revenue and are presented as cost-sharing, net.
|
(2)
|
Other expenses include unallocated employee and contractor-related expenses, facility expenses, lab supplies and miscellaneous expenses.
|
|
Year Ended
December 31, |
|
|
||||||||
|
Increase
(Decrease) |
||||||||||
(in thousands)
|
2016
|
|
2015
|
|
|||||||
Revenue:
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue:
|
|
|
|
|
|
|
|
|
|||
License and milestone
|
$
|
15,550
|
|
|
$
|
1,184
|
|
|
$
|
14,366
|
|
Cost-sharing, net
|
12,221
|
|
|
16,913
|
|
|
(4,692
|
)
|
|||
Total revenue (all amounts are with a related party)
|
27,771
|
|
|
18,097
|
|
|
9,674
|
|
|||
Costs and expenses:
|
|
|
|
|
|
||||||
Research and development
|
68,580
|
|
|
58,404
|
|
|
10,176
|
|
|||
General and administrative
|
25,297
|
|
|
20,572
|
|
|
4,725
|
|
|||
Total costs and expenses
|
93,877
|
|
|
78,976
|
|
|
14,901
|
|
|||
Loss from operations
|
(66,106
|
)
|
|
(60,879
|
)
|
|
(5,227
|
)
|
|||
Other income (expense), net
|
9,116
|
|
|
(3,015
|
)
|
|
12,131
|
|
|||
Loss before income taxes
|
(56,990
|
)
|
|
(63,894
|
)
|
|
6,904
|
|
|||
Income tax provision
|
(24
|
)
|
|
—
|
|
|
(24
|
)
|
|||
Net loss
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
6,880
|
|
|
Year Ended
December 31,
|
|
|
||||||||
|
Increase
(Decrease)
|
||||||||||
(in thousands)
|
2015
|
|
2014
|
|
|||||||
Revenue:
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue:
|
|
|
|
|
|
|
|
|
|||
License and milestone
|
$
|
1,184
|
|
|
$
|
1,673
|
|
|
$
|
(489
|
)
|
Cost-sharing, net
|
16,913
|
|
|
12,959
|
|
|
3,954
|
|
|||
Total revenue (all amounts are with a related party)
|
18,097
|
|
|
14,632
|
|
|
3,465
|
|
|||
Costs and expenses:
|
|
|
|
|
|
|
|||||
Research and development
|
58,404
|
|
|
50,897
|
|
|
7,507
|
|
|||
Litigation settlement
|
—
|
|
|
5,000
|
|
|
(5,000
|
)
|
|||
General and administrative
|
20,572
|
|
|
14,199
|
|
|
6,373
|
|
|||
Total costs and expenses
|
78,976
|
|
|
70,096
|
|
|
8,880
|
|
|||
Loss from operations
|
(60,879
|
)
|
|
(55,464
|
)
|
|
(5,415
|
)
|
|||
Other (expense) income, net
|
(3,015
|
)
|
|
4,205
|
|
|
(7,220
|
)
|
|||
Net loss
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
|
$
|
(12,635
|
)
|
|
Year Ended December 31,
|
||||||||||
(in thousands)
|
2016
|
|
2015
|
|
2014
|
||||||
Net cash (used in) provided by:
|
|
|
|
|
|
|
|
||||
Operating activities
|
$
|
(44,695
|
)
|
|
$
|
(44,211
|
)
|
|
$
|
(53,220
|
)
|
Investing activities
|
(108,805
|
)
|
|
(108,971
|
)
|
|
(514
|
)
|
|||
Financing activities
|
146,667
|
|
|
4,505
|
|
|
117,031
|
|
|||
Net (decrease) increase in cash and cash equivalents
|
$
|
(6,833
|
)
|
|
$
|
(148,677
|
)
|
|
$
|
63,297
|
|
•
|
the achievement of milestones under our agreement with Celgene;
|
•
|
the terms and timing of any other collaborative, licensing and other arrangements that we may establish;
|
•
|
the initiation, progress, timing and completion of preclinical studies and clinical trials for our therapeutic candidates and potential therapeutic candidates;
|
•
|
the number and characteristics of therapeutic candidates that we pursue;
|
•
|
the progress, costs and results of our clinical trials;
|
•
|
the outcome, timing and cost of regulatory approvals;
|
•
|
delays that may be caused by changing regulatory requirements;
|
•
|
the cost and timing of hiring new employees to support our continued growth;
|
•
|
the costs involved in filing and prosecuting patent applications and enforcing and defending patent claims;
|
•
|
the costs and timing of procuring clinical and commercial supplies of our therapeutic candidates;
|
•
|
the extent to which we acquire or invest in businesses, products or technologies; and
|
•
|
the costs involved in defending and prosecuting litigation regarding in-licensed intellectual property.
|
(in thousands)
|
Total
|
|
2017
|
|
2018 through 2019
|
|
2020 through 2021
|
|
After 2021
|
||||||||||
Operating lease obligations(1)
|
$
|
9,584
|
|
|
$
|
4,547
|
|
|
$
|
4,217
|
|
|
$
|
821
|
|
|
$
|
—
|
|
Total
|
$
|
9,584
|
|
|
$
|
4,547
|
|
|
$
|
4,217
|
|
|
$
|
821
|
|
|
$
|
—
|
|
(1)
|
We lease office and lab space at 128 Sidney Street and 149 Sidney Street in Cambridge, Massachusetts under noncancelable operating leases that expire in September 2018. We also lease space at 125 Sidney Street under a noncancelable operating lease that expires in March 2021. Our leasehold improvements are being amortized over 2-10 years which represent the shorter of their useful life or remaining lease term.
|
•
|
Under our license agreement with the Beth Israel Deaconess Medical Center, or BIDMC, in respect of BIDMC's joint interest in patent rights related to the treatment of renal cell cancer by combination therapy with dalantercept and VEGF-receptor tyrosine kinase inhibitors, we agreed to pay BIDMC specified development and sales milestone payments aggregating up to $1.0 million. In addition, we are required to pay BIDMC royalties in the low single digits on worldwide net product sales of drug labeled for treatment regimens that are claimed in the licensed patents.
|
•
|
Under our license agreement with the Ludwig Institute for Cancer Research, or LICR, in respect of patent rights relating to the first cloning of the type I activin receptors, as well as the treatment of pancreatic tumors with dalantercept, we agreed to pay LICR specified development and sales milestone payments aggregating up to $1.6 million relating to the development and commercialization of dalantercept. In addition, we are required to pay LICR royalties in the low single-digits on worldwide net product sales of dalantercept, with royalty obligations continuing at a 50% reduced rate for a period of time after patent expiration. If we sublicense the LICR patent rights, we will owe LICR a percentage of sublicensing revenue, excluding payments based on the level of sales, profits or other levels of commercialization.
|
•
|
Under our two license agreements with the Salk Institute for Biological Studies, or Salk, relating to the first cloning of the type II activin receptors, if we sublicense the Salk patent rights, we will owe Salk a percentage of sublicensing revenue, excluding payments based on sales. Under one agreement, we agreed to pay Salk specified development milestone payments totaling up to $2.0 million for sotatercept. Under the other agreement, we agreed to pay Salk specified development milestone payments of up to $0.7 million for luspatercept. In addition, under both agreements, we are required to pay Salk royalties in the low single-digits on worldwide net product sales by us or our sublicensees under the licensed patent rights of products claimed in the licensed patents, or products derived from use of the
|
•
|
In May 2014, we executed a collaboration agreement with a research technology company. We paid an upfront and research fee of
$0.3 million
upon execution of the agreement. We also received an option to obtain a commercial license to the molecules developed during the collaboration, which, if exercised, would obligate us to pay royalty and milestone payments.
|
|
|
/s/ Ernst & Young LLP
|
(a)
|
The following documents are filed as part of this report:
|
(1)
|
Financial Statements.
|
(2)
|
Financial Statement Schedules.
|
(3)
|
Exhibits.
|
(b)
|
The list of Exhibits filed as a part of this Annual Report on Form 10-K is set forth on the Exhibit Index immediately preceding such Exhibits and is incorporated herein by this reference.
|
(c)
|
None.
|
|
|
/s/ Ernst & Young LLP
|
|
December 31,
|
||||||
|
2016
|
|
2015
|
||||
Assets
|
|
|
|
||||
Current assets:
|
|
|
|
||||
Cash and cash equivalents
|
$
|
20,950
|
|
|
$
|
27,783
|
|
Short-term investments
|
118,740
|
|
|
77,064
|
|
||
Collaboration receivables (all amounts are with a related party)
|
3,234
|
|
|
3,628
|
|
||
Prepaid expenses and other current assets
|
3,862
|
|
|
2,458
|
|
||
Total current assets
|
146,786
|
|
|
110,933
|
|
||
Property and equipment, net
|
5,201
|
|
|
3,106
|
|
||
Long-term investments
|
94,692
|
|
|
31,134
|
|
||
Restricted cash
|
946
|
|
|
796
|
|
||
Other assets
|
22
|
|
|
368
|
|
||
Total assets
|
$
|
247,647
|
|
|
$
|
146,337
|
|
Liabilities and stockholders' equity
|
|
|
|
||||
Current liabilities:
|
|
|
|
||||
Accounts payable
|
$
|
1,590
|
|
|
$
|
875
|
|
Accrued expenses
|
13,249
|
|
|
12,400
|
|
||
Deferred revenue
|
541
|
|
|
555
|
|
||
Deferred rent
|
769
|
|
|
661
|
|
||
Total current liabilities
|
16,149
|
|
|
14,491
|
|
||
Deferred revenue, net of current portion
|
3,704
|
|
|
4,239
|
|
||
Deferred rent, net of current portion
|
953
|
|
|
1,157
|
|
||
Warrants to purchase common stock
|
1,244
|
|
|
17,187
|
|
||
Total liabilities
|
22,050
|
|
|
37,074
|
|
||
Commitments and contingencies (Note 8)
|
—
|
|
|
—
|
|
||
Stockholders' equity:
|
|
|
|
||||
Undesignated preferred stock, $0.001 par value: 25,000,000 shares authorized and no shares issued or outstanding
|
—
|
|
|
—
|
|
||
Common stock, $0.001 par value: 175,000,000 shares authorized; 38,251,826 and 33,313,355, shares issued and outstanding at December 31, 2016 and 2015, respectively
|
39
|
|
|
34
|
|
||
Additional paid-in capital
|
590,474
|
|
|
416,926
|
|
||
Accumulated deficit
|
(364,491
|
)
|
|
(307,477
|
)
|
||
Accumulated other comprehensive loss
|
(425
|
)
|
|
(220
|
)
|
||
Total stockholders' equity
|
225,597
|
|
|
109,263
|
|
||
Total liabilities and stockholders' equity
|
$
|
247,647
|
|
|
$
|
146,337
|
|
|
Year Ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Revenue:
|
|
|
|
|
|
|
|||||
Collaboration revenue:
|
|
|
|
|
|
|
|||||
License and milestone
|
$
|
15,550
|
|
|
$
|
1,184
|
|
|
$
|
1,673
|
|
Cost-sharing, net
|
12,221
|
|
|
16,913
|
|
|
12,959
|
|
|||
Total revenue (all amounts are with a related party)
|
27,771
|
|
|
18,097
|
|
|
14,632
|
|
|||
Costs and expenses:
|
|
|
|
|
|
|
|||||
Research and development
|
68,580
|
|
|
58,404
|
|
|
50,897
|
|
|||
Litigation settlement
|
—
|
|
|
—
|
|
|
5,000
|
|
|||
General and administrative
|
25,297
|
|
|
20,572
|
|
|
14,199
|
|
|||
Total costs and expenses
|
93,877
|
|
|
78,976
|
|
|
70,096
|
|
|||
Loss from operations
|
(66,106
|
)
|
|
(60,879
|
)
|
|
(55,464
|
)
|
|||
Other income (expense):
|
|
|
|
|
|
|
|
||||
Other income (expense), net
|
7,262
|
|
|
(3,527
|
)
|
|
5,044
|
|
|||
Interest income
|
1,854
|
|
|
512
|
|
|
83
|
|
|||
Interest expense
|
—
|
|
|
—
|
|
|
(922
|
)
|
|||
Total other income (expense), net
|
9,116
|
|
|
(3,015
|
)
|
|
4,205
|
|
|||
Loss before income taxes
|
(56,990
|
)
|
|
(63,894
|
)
|
|
(51,259
|
)
|
|||
Income tax provision
|
(24
|
)
|
|
—
|
|
|
—
|
|
|||
Net loss
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
Other comprehensive loss:
|
|
|
|
|
|
||||||
Net unrealized holding losses on short-term and long-term investments during the period
|
(205
|
)
|
|
(220
|
)
|
|
—
|
|
|||
Comprehensive loss
|
$
|
(57,219
|
)
|
|
$
|
(64,114
|
)
|
|
$
|
(51,259
|
)
|
|
|
|
|
|
|
||||||
Net loss per share - basic and diluted
|
$
|
(1.52
|
)
|
|
$
|
(1.92
|
)
|
|
$
|
(1.63
|
)
|
|
|
|
|
|
|
||||||
Weighted-average number of common shares used in computing net loss per share -basic and diluted
|
37,430
|
|
|
33,303
|
|
|
31,515
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
|
Common Stock
|
|
|
|
|
|
|
|
|
|||||||||||||
|
Number of
Shares
|
|
$0.001 Par
Value
|
|
Additional
Paid-In Capital
|
|
Accumulated
Deficit
|
|
Comprehensive Income (Loss)
|
|
Total
Stockholders'
Equity (Deficit)
|
|||||||||||
Balance at December 31, 2013
|
28,348,630
|
|
|
$
|
29
|
|
|
$
|
250,107
|
|
|
$
|
(192,324
|
)
|
|
$
|
—
|
|
|
$
|
57,812
|
|
Issuance of common stock, net of expenses of $554
|
2,760,000
|
|
|
3
|
|
|
129,171
|
|
|
—
|
|
|
—
|
|
|
129,174
|
|
|||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
4,778
|
|
|
—
|
|
|
—
|
|
|
4,778
|
|
|||||
Exercise of stock options
|
853,507
|
|
|
1
|
|
|
3,207
|
|
|
—
|
|
|
—
|
|
|
3,208
|
|
|||||
Net exercise of warrants to purchase common stock
|
303,204
|
|
|
—
|
|
|
7,422
|
|
|
—
|
|
|
—
|
|
|
7,422
|
|
|||||
Exercise of warrants to purchase common stock
|
166,684
|
|
|
—
|
|
|
5,150
|
|
|
—
|
|
|
—
|
|
|
5,150
|
|
|||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(51,259
|
)
|
|
—
|
|
|
(51,259
|
)
|
|||||
Balance at December 31, 2014
|
32,432,025
|
|
|
33
|
|
|
399,835
|
|
|
(243,583
|
)
|
|
—
|
|
|
156,285
|
|
|||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
12,075
|
|
|
—
|
|
|
—
|
|
|
12,075
|
|
|||||
Exercise of stock options
|
837,361
|
|
|
1
|
|
|
3,962
|
|
|
—
|
|
|
—
|
|
|
3,963
|
|
|||||
Issuance of common stock related to ESPP
|
23,787
|
|
|
—
|
|
|
589
|
|
|
—
|
|
|
—
|
|
|
589
|
|
|||||
Net exercise of warrants to purchase common stock
|
20,182
|
|
|
—
|
|
|
465
|
|
|
—
|
|
|
—
|
|
|
465
|
|
|||||
Unrealized loss on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(220
|
)
|
|
(220
|
)
|
|||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(63,894
|
)
|
|
—
|
|
|
(63,894
|
)
|
|||||
Balance at December 31, 2015
|
33,313,355
|
|
|
34
|
|
|
416,926
|
|
|
(307,477
|
)
|
|
(220
|
)
|
|
109,263
|
|
|||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
18,557
|
|
|
—
|
|
|
—
|
|
|
18,557
|
|
|||||
Issuance of common stock, net of expenses of $665
|
3,750,000
|
|
|
4
|
|
|
140,340
|
|
|
—
|
|
|
—
|
|
|
140,344
|
|
|||||
Exercise of stock options
|
885,075
|
|
|
1
|
|
|
5,311
|
|
|
—
|
|
|
—
|
|
|
5,312
|
|
|||||
Issuance of common stock related to ESPP
|
30,671
|
|
|
—
|
|
|
658
|
|
|
—
|
|
|
—
|
|
|
658
|
|
|||||
Net exercise of warrants to purchase common stock
|
272,725
|
|
|
—
|
|
|
8,682
|
|
|
—
|
|
|
—
|
|
|
8,682
|
|
|||||
Unrealized loss on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(205
|
)
|
|
(205
|
)
|
|||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(57,014
|
)
|
|
—
|
|
|
(57,014
|
)
|
|||||
Balance at December 31, 2016
|
38,251,826
|
|
|
$
|
39
|
|
|
$
|
590,474
|
|
|
$
|
(364,491
|
)
|
|
$
|
(425
|
)
|
|
$
|
225,597
|
|
|
Year Ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Operating Activities
|
|
|
|
|
|
|
|||||
Net loss
|
$
|
(57,014
|
)
|
|
$
|
(63,894
|
)
|
|
$
|
(51,259
|
)
|
Adjustments to reconcile net loss to net cash used in operating activities:
|
|
|
|
|
|
|
|||||
Depreciation and amortization
|
1,676
|
|
|
1,176
|
|
|
1,118
|
|
|||
Loss on disposition of property and equipment
|
6
|
|
|
34
|
|
|
25
|
|
|||
Stock-based compensation
|
18,557
|
|
|
12,075
|
|
|
4,778
|
|
|||
Accretion of deferred interest
|
—
|
|
|
—
|
|
|
(536
|
)
|
|||
Amortization of deferred debt issuance costs
|
—
|
|
|
—
|
|
|
36
|
|
|||
Change in fair value of warrants
|
(7,262
|
)
|
|
3,528
|
|
|
(5,037
|
)
|
|||
Net amortization of premium on investments
|
(14
|
)
|
|
(406
|
)
|
|
—
|
|
|||
Changes in assets and liabilities:
|
|
|
|
|
|
|
|
||||
Prepaid expenses and other current assets
|
(1,411
|
)
|
|
7
|
|
|
(255
|
)
|
|||
Collaboration receivables
|
394
|
|
|
(261
|
)
|
|
249
|
|
|||
Accounts payable
|
644
|
|
|
151
|
|
|
(161
|
)
|
|||
Accrued expenses
|
524
|
|
|
4,976
|
|
|
(17
|
)
|
|||
Deferred revenue
|
(549
|
)
|
|
(1,184
|
)
|
|
(1,673
|
)
|
|||
Deferred rent
|
(96
|
)
|
|
(519
|
)
|
|
(499
|
)
|
|||
Restricted cash
|
(150
|
)
|
|
106
|
|
|
11
|
|
|||
Net cash used in operating activities
|
(44,695
|
)
|
|
(44,211
|
)
|
|
(53,220
|
)
|
|||
Investing Activities
|
|
|
|
|
|
|
|
||||
Purchase of investments
|
(218,314
|
)
|
|
(134,697
|
)
|
|
—
|
|
|||
Proceeds from sales and maturities of investments
|
112,889
|
|
|
26,685
|
|
|
—
|
|
|||
Purchases of property and equipment
|
(3,380
|
)
|
|
(959
|
)
|
|
(514
|
)
|
|||
Net cash used in investing activities
|
(108,805
|
)
|
|
(108,971
|
)
|
|
(514
|
)
|
|||
Financing Activities
|
|
|
|
|
|
|
|||||
Proceeds from issuance of common stock from public offering, net of issuance costs
|
140,697
|
|
|
(47
|
)
|
|
129,174
|
|
|||
Payments of long-term debt
|
—
|
|
|
—
|
|
|
(16,331
|
)
|
|||
Proceeds from issuances of common stock related to employee stock purchase plan
|
658
|
|
|
589
|
|
|
—
|
|
|||
Proceeds from exercise of stock options and warrants to purchase common stock
|
5,312
|
|
|
3,963
|
|
|
4,188
|
|
|||
Net cash provided by financing activities
|
146,667
|
|
|
4,505
|
|
|
117,031
|
|
|||
Net (decrease) increase in cash and cash equivalents
|
(6,833
|
)
|
|
(148,677
|
)
|
|
63,297
|
|
|||
Cash and cash equivalents at beginning of year
|
27,783
|
|
|
176,460
|
|
|
113,163
|
|
|||
Cash and cash equivalents at end of year
|
$
|
20,950
|
|
|
$
|
27,783
|
|
|
$
|
176,460
|
|
Supplemental Disclosure of Cash Flow Information:
|
|
|
|
|
|
|
|
||||
Cash paid for interest
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
1,574
|
|
Supplemental Disclosure of Non-Cash Investing and Financing Activities:
|
|
|
|
|
|
|
|
||||
Reclassification of warrant liability to additional paid-in capital
|
$
|
8,682
|
|
|
$
|
465
|
|
|
$
|
11,592
|
|
Capitalized follow-on public offering costs included in accrued expenses
|
$
|
—
|
|
|
$
|
306
|
|
|
$
|
—
|
|
Purchase of property and equipment included in accounts payable and accrued expenses
|
$
|
397
|
|
|
$
|
270
|
|
|
$
|
11
|
|
•
|
Level 1—Quoted market prices in active markets for identical assets or liabilities.
|
•
|
Level 2—Inputs other than Level 1 inputs that are either directly or indirectly observable, such as quoted market prices, interest rates, and yield curves.
|
•
|
Level 3—Unobservable inputs developed using estimates of assumptions developed by the Company, which reflect those that a market participant would use.
|
|
December 31, 2016
|
||||||||||||||
|
Quoted Prices
in Active Markets for Identical Items (Level 1) |
|
Significant Other
Observable Inputs (Level 2) |
|
Significant
Unobservable Inputs (Level 3) |
|
Total
|
||||||||
Assets:
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds
|
$
|
19,818
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
19,818
|
|
Corporate obligations
|
—
|
|
|
88,492
|
|
|
—
|
|
|
88,492
|
|
||||
U.S. Treasury securities
|
—
|
|
|
33,968
|
|
|
—
|
|
|
33,968
|
|
||||
Certificates of deposit
|
—
|
|
|
23,373
|
|
|
—
|
|
|
23,373
|
|
||||
Mortgage and other asset backed securities
|
—
|
|
|
67,599
|
|
|
—
|
|
|
67,599
|
|
||||
Restricted cash
|
946
|
|
|
—
|
|
|
—
|
|
|
946
|
|
||||
Total assets
|
$
|
20,764
|
|
|
$
|
213,432
|
|
|
$
|
—
|
|
|
$
|
234,196
|
|
Liabilities:
|
|
|
|
|
|
|
|
|
|
|
|
||||
Warrants to purchase common stock
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
1,244
|
|
|
$
|
1,244
|
|
Total liabilities
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
1,244
|
|
|
$
|
1,244
|
|
|
December 31, 2015
|
||||||||||||||
|
Quoted Prices
in Active Markets for Identical Items (Level 1) |
|
Significant Other
Observable Inputs (Level 2) |
|
Significant
Unobservable Inputs (Level 3) |
|
Total
|
||||||||
Assets:
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds
|
$
|
24,811
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
24,811
|
|
Corporate obligations
|
—
|
|
|
67,706
|
|
|
—
|
|
|
67,706
|
|
||||
U.S. Treasury securities
|
—
|
|
|
10,991
|
|
|
—
|
|
|
10,991
|
|
||||
Certificates of deposit
|
—
|
|
|
16,776
|
|
|
—
|
|
|
16,776
|
|
||||
Mortgage and other asset backed securities
|
—
|
|
|
13,228
|
|
|
—
|
|
|
13,228
|
|
||||
Restricted cash
|
796
|
|
|
—
|
|
|
—
|
|
|
796
|
|
||||
Total assets
|
$
|
25,607
|
|
|
$
|
108,701
|
|
|
$
|
—
|
|
|
$
|
134,308
|
|
Liabilities:
|
|
|
|
|
|
|
|
|
|
|
|
||||
Warrants to purchase common stock
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
17,187
|
|
|
$
|
17,187
|
|
Total liabilities
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
17,187
|
|
|
$
|
17,187
|
|
|
Year Ended
December 31,
|
||||||
|
2016
|
|
2015
|
||||
Beginning balance
|
$
|
17,187
|
|
|
$
|
14,124
|
|
Change in fair value
|
(7,262
|
)
|
|
3,528
|
|
||
Exercises
|
(8,681
|
)
|
|
(465
|
)
|
||
Ending balance
|
$
|
1,244
|
|
|
$
|
17,187
|
|
Asset
|
Estimated Useful Life
|
Computer equipment and software
|
3 years
|
Office and laboratory equipment
|
3 years
|
Leasehold improvements
|
Shorter of the useful life or remaining lease term
|
|
Year Ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Outstanding stock options
|
3,316
|
|
|
3,191
|
|
|
3,230
|
|
Common stock warrants
|
64
|
|
|
398
|
|
|
422
|
|
Shares issuable under employee stock purchase plan
|
23
|
|
|
15
|
|
|
14
|
|
Restricted stock units
|
732
|
|
|
521
|
|
|
—
|
|
|
4,135
|
|
|
4,125
|
|
|
3,666
|
|
|
December 31,
|
||||||
|
2016
|
|
2015
|
||||
Computer equipment and software
|
$
|
1,260
|
|
|
$
|
1,163
|
|
Office equipment
|
519
|
|
|
260
|
|
||
Laboratory equipment
|
13,204
|
|
|
11,458
|
|
||
Leasehold improvements
|
11,126
|
|
|
9,990
|
|
||
Construction in progress
|
849
|
|
|
390
|
|
||
Total property and equipment
|
26,958
|
|
|
23,261
|
|
||
Accumulated depreciation and amortization
|
(21,757
|
)
|
|
(20,155
|
)
|
||
Property and equipment, net
|
$
|
5,201
|
|
|
$
|
3,106
|
|
|
December 31, 2016
|
||||||||||||||
|
Amortized Cost
|
|
Gross Unrealized Gains
|
|
Gross Unrealized Losses
|
|
Estimated Fair Value
|
||||||||
Cash and cash equivalents due in 90 days or less
|
$
|
20,950
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
20,950
|
|
Available-for-sale securities:
|
|
|
|
|
|
|
|
||||||||
Corporate obligations due in one year or less
|
45,839
|
|
|
1
|
|
|
(58
|
)
|
|
45,782
|
|
||||
Corporate obligations due in more than one year
|
42,895
|
|
|
—
|
|
|
(185
|
)
|
|
42,710
|
|
||||
U.S. Treasury securities due in one year or less
|
22,490
|
|
|
—
|
|
|
(10
|
)
|
|
22,480
|
|
||||
U.S. Treasury securities due in more than one year
|
11,541
|
|
|
—
|
|
|
(53
|
)
|
|
11,488
|
|
||||
Certificates of deposit due in one year or less
|
13,562
|
|
|
—
|
|
|
—
|
|
|
13,562
|
|
||||
Certificates of deposit due in more than one year
|
9,811
|
|
|
—
|
|
|
—
|
|
|
9,811
|
|
||||
Mortgage and other asset backed securities due in one year or less
|
36,948
|
|
|
—
|
|
|
(32
|
)
|
|
36,916
|
|
||||
Mortgage and other asset backed securities due in more than one year
|
30,771
|
|
|
—
|
|
|
(88
|
)
|
|
30,683
|
|
||||
Total available-for-sale securities
|
$
|
213,857
|
|
|
$
|
1
|
|
|
$
|
(426
|
)
|
|
$
|
213,432
|
|
Total cash, cash equivalents and available-for-sale securities
|
$
|
234,807
|
|
|
$
|
1
|
|
|
$
|
(426
|
)
|
|
$
|
234,382
|
|
|
December 31, 2015
|
||||||||||||||
|
Amortized Cost
|
|
Gross Unrealized Gains
|
|
Gross Unrealized Losses
|
|
Estimated Fair Value
|
||||||||
Cash and cash equivalents due in 90 days or less
|
$
|
27,783
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
27,783
|
|
Available-for-sale securities:
|
|
|
|
|
|
|
|
||||||||
Corporate obligations due in one year or less
|
53,243
|
|
|
—
|
|
|
(81
|
)
|
|
53,162
|
|
||||
Corporate obligations due in more than one year
|
14,112
|
|
|
—
|
|
|
(72
|
)
|
|
14,040
|
|
||||
U.S. Treasury securities due in one year or less
|
6,016
|
|
|
—
|
|
|
(4
|
)
|
|
6,012
|
|
||||
U.S. Treasury securities due in more than one year
|
4,995
|
|
|
—
|
|
|
(15
|
)
|
|
4,980
|
|
||||
Certificates of deposit due in one year or less
|
11,890
|
|
|
—
|
|
|
—
|
|
|
11,890
|
|
||||
Certificates of deposit due in more than one year
|
4,886
|
|
|
—
|
|
|
—
|
|
|
4,886
|
|
||||
Mortgage and other asset backed securities due in one year or less
|
6,010
|
|
|
—
|
|
|
(10
|
)
|
|
6,000
|
|
||||
Mortgage and other asset backed securities due in more than one year
|
7,266
|
|
|
—
|
|
|
(38
|
)
|
|
7,228
|
|
||||
Total available-for-sale securities
|
$
|
108,418
|
|
|
$
|
—
|
|
|
$
|
(220
|
)
|
|
$
|
108,198
|
|
Total cash, cash equivalents and available-for-sale securities
|
$
|
136,201
|
|
|
$
|
—
|
|
|
$
|
(220
|
)
|
|
$
|
135,981
|
|
|
December 31,
|
||||||
|
2016
|
|
2015
|
||||
Research and development related
|
$
|
5,238
|
|
|
$
|
4,938
|
|
Employee compensation
|
4,840
|
|
|
3,551
|
|
||
Professional services
|
739
|
|
|
1,198
|
|
||
Other
|
2,432
|
|
|
2,713
|
|
||
|
$
|
13,249
|
|
|
$
|
12,400
|
|
|
Warrants as of
|
|
|
|
|
|
|
|
|
||||||
|
|
|
|
|
Weighted-
Average
Exercise
|
|
|
|
Balance Sheet
Classification
|
||||||
|
December 31, 2016
|
|
December 31, 2015
|
|
Price Per
Share
|
|
Expiration
|
|
December 31, 2016
|
|
December 31, 2015
|
||||
Warrants to purchase common stock
|
61
|
|
|
393
|
|
|
$
|
5.88
|
|
|
June 10, 2020 - July 9, 2020
|
|
Liability(2)
|
|
Liability(2)
|
Warrants to purchase common stock
|
4
|
|
|
5
|
|
|
4.00 - 7.40
|
|
|
March 28, 2017 - December 31, 2017
|
|
Equity(1) (3)
|
|
Equity(1) (3)
|
|
All warrants
|
65
|
|
|
398
|
|
|
$
|
5.91
|
|
|
|
|
|
|
|
(1)
|
In March 2016, the warrant holders exercised warrants to purchase
1,317
shares of Common Stock on a net basis, resulting in the issuance of
1,109
shares of Common Stock.
|
(2)
|
In December 2016, the warrant holders exercised warrants to purchase
332,211
shares of Common Stock on a net basis, resulting in the issuance of
271,616
shares of Common Stock.
|
(3)
|
Warrants to purchase common stock were issued in connection with various debt financing transactions that were consummated in periods prior to December 31, 2012. See discussion below for further details.
|
2017
|
$
|
4,547
|
|
2018
|
3,577
|
|
|
2019
|
640
|
|
|
2020
|
656
|
|
|
2021
|
165
|
|
|
Total
|
$
|
9,585
|
|
|
December 31, 2016
|
|
Outstanding stock options to purchase common stock
|
3,316
|
|
Outstanding restricted stock units
|
732
|
|
Shares available for future issuance under stock option plans
|
1,997
|
|
Warrants to purchase common stock
|
64
|
|
Shares available for future issuance under the employee stock purchase plan
|
221
|
|
Additional shares reserved for unissued, but designated, Preferred Stock
|
25,000
|
|
Total shares of authorized common stock reserved for future issuance
|
31,330
|
|
•
|
$18.8 million
for research and development services
|
•
|
$2.9 million
for the sotatercept joint development committee
|
•
|
$3.7 million
for the ACE 536 joint development committee
|
•
|
$2.8 million
for the manufacturing services
|
|
Year Ended December 31,
|
||||||||||
|
2016
|
|
2015
|
|
2014
|
||||||
Research and development
|
$
|
8,171
|
|
|
$
|
4,852
|
|
|
$
|
2,065
|
|
General and administrative
|
10,386
|
|
|
7,223
|
|
|
2,713
|
|
|||
|
$
|
18,557
|
|
|
$
|
12,075
|
|
|
$
|
4,778
|
|
|
Year Ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Expected volatility
|
65.1
|
%
|
|
67.0
|
%
|
|
70.9
|
%
|
Expected term (in years)
|
6.0
|
|
|
6.0
|
|
|
6.0
|
|
Risk-free interest rate
|
1.69
|
%
|
|
1.67
|
%
|
|
1.82
|
%
|
Expected dividend yield
|
—
|
%
|
|
—
|
%
|
|
—
|
%
|
|
Number
of Grants |
|
Weighted-
Average Exercise Price Per Share |
|
Weighted-
Average Contractual Life
(in years)
|
|
Aggregate
Intrinsic Value(1) |
|||||
Outstanding at December 31, 2015
|
3,191
|
|
|
$
|
18.85
|
|
|
|
|
|
||
Granted
|
1,046
|
|
|
$
|
31.13
|
|
|
|
|
|
|
|
Exercised
|
(885
|
)
|
|
$
|
6.00
|
|
|
|
|
|
|
|
Canceled or forfeited
|
(36
|
)
|
|
$
|
36.45
|
|
|
|
|
|
|
|
Outstanding at December 31, 2016
|
3,316
|
|
|
$
|
25.96
|
|
|
7.03
|
|
$
|
18,562,282
|
|
Exercisable at December 31, 2016
|
1,779
|
|
|
$
|
19.36
|
|
|
5.43
|
|
$
|
18,421,393
|
|
Vested and expected to vest at December 31, 2016(2)
|
3,258
|
|
|
$
|
25.83
|
|
|
6.99
|
|
$
|
18,560,293
|
|
(1)
|
The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the estimated fair value of the common stock for the options that were in the money at
December 31, 2016
and
2015
.
|
(2)
|
This represents the number of vested options at
December 31, 2016
, plus the number of unvested options expected to vest at
December 31, 2016
, based on the unvested options outstanding at
December 31, 2016
, adjusted for the estimated forfeiture rate.
|
|
Number
of Grants |
|
Weighted-
Average Grant Date Fair Value |
|||
Unvested balance at December 31, 2015
|
521
|
|
|
$
|
31.57
|
|
Granted
|
221
|
|
|
$
|
31.56
|
|
Vested
|
—
|
|
|
$
|
—
|
|
Forfeited
|
(10
|
)
|
|
$
|
33.94
|
|
Unvested balance at December 31, 2016
|
732
|
|
|
$
|
31.55
|
|
|
Year Ended December 31,
|
||||||
|
2016
|
|
2015
|
||||
Deferred tax assets:
|
|
|
|
|
|||
U.S. and state net operating loss carryforwards
|
$
|
109,429
|
|
|
$
|
90,732
|
|
Research and development credits
|
9,717
|
|
|
7,864
|
|
||
Deferred revenue
|
1,666
|
|
|
1,883
|
|
||
Accruals and other temporary differences
|
15,725
|
|
|
9,319
|
|
||
Total deferred tax assets
|
136,537
|
|
|
109,798
|
|
||
Less valuation allowance
|
(136,537
|
)
|
|
(109,798
|
)
|
||
Net deferred tax assets
|
$
|
—
|
|
|
$
|
—
|
|
|
Year Ended December 31,
|
|||||||
|
2016
|
|
2015
|
|
2014
|
|||
Federal income tax expense at statutory rate
|
34.0
|
%
|
|
34.0
|
%
|
|
34.0
|
%
|
State income tax, net of federal benefit
|
5.9
|
%
|
|
4.6
|
%
|
|
4.1
|
%
|
Permanent differences
|
4.0
|
%
|
|
(8.3
|
)%
|
|
2.0
|
%
|
Research and development credit
|
3.1
|
%
|
|
1.2
|
%
|
|
1.6
|
%
|
Other
|
(0.1
|
)%
|
|
—
|
%
|
|
4.6
|
%
|
Change in valuation allowance
|
(46.9
|
)%
|
|
(31.5
|
)%
|
|
(46.3
|
)%
|
Effective income tax rate
|
—
|
%
|
|
—
|
%
|
|
—
|
%
|
|
For the Three Months Ended(1)
|
||||||||||||||
|
March 31
|
|
June 30
|
|
September 30
|
|
December 31
|
||||||||
|
(in thousands except per share data)
|
||||||||||||||
2016
|
|
|
|
|
|
|
|
||||||||
Total revenue
|
$
|
18,201
|
|
|
$
|
3,195
|
|
|
$
|
3,005
|
|
|
$
|
3,369
|
|
Total costs and expenses
|
22,157
|
|
|
22,850
|
|
|
23,513
|
|
|
25,355
|
|
||||
Loss from operations
|
(3,956
|
)
|
|
(19,655
|
)
|
|
(20,508
|
)
|
|
(21,986
|
)
|
||||
Net income (loss)
|
5,061
|
|
|
(22,016
|
)
|
|
(20,770
|
)
|
|
(19,288
|
)
|
||||
Basic net income (loss) per share
|
$
|
0.14
|
|
|
$
|
(0.59
|
)
|
|
$
|
(0.55
|
)
|
|
$
|
(0.51
|
)
|
Diluted net income (loss) per share
|
$
|
0.13
|
|
|
$
|
(0.59
|
)
|
|
$
|
(0.55
|
)
|
|
$
|
(0.51
|
)
|
2015
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total revenue
|
$
|
4,420
|
|
|
$
|
5,717
|
|
|
$
|
4,155
|
|
|
$
|
3,804
|
|
Total costs and expenses
|
19,479
|
|
|
18,811
|
|
|
18,768
|
|
|
21,919
|
|
||||
Loss from operations
|
(15,059
|
)
|
|
(13,094
|
)
|
|
(14,613
|
)
|
|
(18,115
|
)
|
||||
Net loss
|
(14,574
|
)
|
|
(10,383
|
)
|
|
(11,858
|
)
|
|
(27,082
|
)
|
||||
Basic net loss per share
|
$
|
(0.45
|
)
|
|
$
|
(0.32
|
)
|
|
$
|
(0.36
|
)
|
|
$
|
(0.81
|
)
|
Diluted net loss per share
|
$
|
(0.45
|
)
|
|
$
|
(0.32
|
)
|
|
$
|
(0.36
|
)
|
|
$
|
(0.81
|
)
|
(1)
|
The amounts were computed independently for each quarter, and the sum of the quarters may not total the annual amounts.
|
Exhibit
Number
|
|
Exhibit Description
|
|
Filed
with this
Report
|
|
Incorporated by
Reference
herein from
Form or
Schedule
|
|
Filing Date
|
|
SEC File/Reg.
Number
|
3.1
|
|
Restated Certificate of Incorporation
|
|
|
|
Form 8-K (Exhibit 3.1)
|
|
9/24/2013
|
|
001-36065
|
3.2
|
|
Amended and Restated By-laws
|
|
|
|
Form 8-K (Exhibit 3.2)
|
|
9/24/2013
|
|
001-36065
|
3.2.1
|
|
Amendment No. 1 to Amended and Restated Bylaws of Acceleron Pharma Inc.
|
|
|
|
Form 8-K (Exhibit 3.1)
|
|
9/12/2016
|
|
001-36065
|
4.1
|
|
Form of Common Stock Certificate
|
|
|
|
Form S-1 (Exhibit 4.1)
|
|
8/7/2013
|
|
333-190417
|
4.2
|
|
Form of Amended and Restated Registration Rights Agreement
|
|
|
|
Form S-1 (Exhibit 4.2)
|
|
8/7/2013
|
|
333-190417
|
4.3
|
|
Form of Warrant to Purchase Stock, issued to Series E Investors as of June 10, 2010 and July 9, 2010
|
|
|
|
Form S-1 (Exhibit 4.3)
|
|
8/7/2013
|
|
333-190417
|
4.4
|
|
Form of Common Stock Warrant Certificate, issued to General Electric Capital Corporation as of March 28, 2007
|
|
|
|
Form S-1 (Exhibit 4.4)
|
|
8/7/2013
|
|
333-190417
|
10.1*
|
|
Form of Director Indemnification Agreement
|
|
|
|
Form S-1 (Exhibit 10.1)
|
|
8/7/2013
|
|
333-190417
|
10.2+
|
|
Collaboration, License and Option Agreement between Acceleron Pharma Inc. and Celgene Corporation, dated as of February 20, 2008, and amended as of August 2, 2011
|
|
|
|
Form 10-Q (Exhibit 10.1)
|
|
8/4/2016
|
|
001-36065
|
10.3
|
|
Amended and Restated License Agreement between Acceleron Pharma Inc. and Ludwig Institute for Cancer Research Ltd., dated as of August 6, 2010
|
|
|
|
Form S-1 (Exhibit 10.7)
|
|
8/7/2013
|
|
333-190417
|
10.4+
|
|
Exclusive License Agreement between Beth Israel Deaconess Medical Center and Acceleron Pharma Inc., dated as of June 21, 2012
|
|
|
|
Form 10-Q (Exhibit 10.3)
|
|
8/4/2016
|
|
001-36065
|
10.4.1+
|
|
Second Amendment to Exclusive License Agreement between Beth Israel Deaconess Medical Center and Acceleron Pharma Inc., dated as of November 7, 2016
|
|
X
|
|
|
|
|
|
|
10.5+
|
|
Collaboration, License and Option Agreement between Acceleron Pharma Inc. and Celgene Corporation, dated as of August 2, 2011
|
|
|
|
Form S-1/A (Exhibit 10.9)
|
|
9/6/2013
|
|
333-190417
|
10.6
|
|
Amended and Restated License Agreement (ActRIIA) between Salk Institute for Biological Studies and Acceleron Pharma Inc., dated as of August 10, 2010
|
|
|
|
Form S-1 (Exhibit 10.10)
|
|
8/7/2013
|
|
333-190417
|
10.7
|
|
Amended and Restated License Agreement (ActRIIB)between Salk Institute for Biological Studies and Acceleron Pharma Inc., dated as of August 11, 2010
|
|
|
|
Form S-1 (Exhibit 10.11)
|
|
8/7/2013
|
|
333-190417
|
10.7.1
|
|
Amendment to Amended and Restated License Agreement (ActRIIB) between Salk Institute for Biological Studies and Acceleron Pharma Inc., dated as of July 25, 2014
|
|
|
|
Form 10-Q (Exhibit 10.1)
|
|
8/12/2014
|
|
001-36065
|
10.8
|
|
Indenture of Lease between Massachusetts Institute of Technology and Acceleron Pharma Inc., dated as of May 20, 2008
|
|
|
|
Form S-1 (Exhibit 10.12)
|
|
8/7/2013
|
|
333-190417
|
10.9*
|
|
Employment Agreement, by and between Habib J. Dable and Acceleron Pharma Inc., dated as of September 23, 2016
|
|
|
|
Form 8-K
(Exhibit 10.1)
|
|
9/27/2016
|
|
001-36065
|
10.10*
|
|
Amended and Restated Employment Agreement between John Knopf and Acceleron Pharma Inc., dated as of August 26, 2013
|
|
|
|
Form 10-K
(Exhibit 10.13)
|
|
3/2/2015
|
|
001-36065
|
10.10.1*
|
|
Consulting Agreement, by and between John L. Knopf, Ph.D. and Acceleron Pharma Inc., dated as of November 3, 2016
|
|
|
|
Form 8-K
(Exhibit 10.1) |
|
11/3/2016
|
|
001-36065
|
Exhibit
Number
|
|
Exhibit Description
|
|
Filed
with this
Report
|
|
Incorporated by
Reference
herein from
Form or
Schedule
|
|
Filing Date
|
|
SEC File/Reg.
Number
|
10.11*
|
|
Amended and Restated Employment Agreement between Matthew L. Sherman and Acceleron Pharma Inc., dated as of August 26, 2013
|
|
|
|
Form 10-K
(Exhibit 10.14)
|
|
3/2/2015
|
|
001-36065
|
10.12*
|
|
Amended and Restated Employment Agreement between John D. Quisel and Acceleron Pharma Inc., dated as of August 26, 2013
|
|
|
|
Form 10-K
(Exhibit 10.15)
|
|
3/2/2015
|
|
001-36065
|
10.13*
|
|
Amended and Restated Employment Agreement between Kevin F. McLaughlin and Acceleron Pharma Inc. dated as of January 31, 2014
|
|
|
|
Form 10-K
(Exhibit 10.16)
|
|
3/2/2015
|
|
001-36065
|
10.13.1*
|
|
First Amendment to Amended and Restated Employment Agreement, by and between Kevin F. McLaughlin and Acceleron Pharma Inc., dated as of September 9, 2015
|
|
|
|
Form 8-K
(Exhibit 10.2)
|
|
9/11/2015
|
|
001-36065
|
10.14*
|
|
Amended and Restated Employment Agreement between Steven D. Ertel and Acceleron Pharma Inc. dated as of January 31, 2014
|
|
|
|
Form 10-K
(Exhibit 10.17)
|
|
3/2/2015
|
|
001-36065
|
10.14.1*
|
|
First Amendment to Amended and Restated Employment Agreement, by and between Steven D. Ertel and Acceleron Pharma Inc., dated as of September 9, 2015
|
|
|
|
Form 8-K
(Exhibit 10.1)
|
|
9/11/2015
|
|
001-36065
|
10.15*
|
|
Form of Acceleron Pharma Inc. Cash Incentive Plan
|
|
|
|
Form S-1/A (Exhibit 10.14)
|
|
8/19/2013
|
|
333-190417
|
10.16*
|
|
Acceleron Pharma Inc. Short-Term Incentive Compensation Plan
|
|
|
|
Form 8-K
(Exhibit 10.1) |
|
6/6/2016
|
|
001-36065
|
10.17*
|
|
Employee Stock Purchase Plan
|
|
|
|
Form S-1/A (Exhibit 10.20)
|
|
9/6/2013
|
|
333-190417
|
10.18*
|
|
Acceleron Pharma Inc. 2003 Stock Option and Restricted Stock Plan
|
|
|
|
Form S-1 (Exhibit 10.15)
|
|
8/7/2013
|
|
333-190417
|
10.19*
|
|
Acceleron Pharma Inc. 2013 Equity Incentive Plan
|
|
|
|
Form S-8 (Exhibit 4.4)
|
|
12/12/2013
|
|
333-192789
|
10.20*
|
|
Form of Non-Statutory Stock Option Agreement under the 2013 Equity Incentive Plan
|
|
X
|
|
|
|
|
|
|
10.21*
|
|
Form of Incentive Stock Option Agreement under the 2013 Equity Incentive Plan
|
|
|
|
Form S-1 (Exhibit 10.22)
|
|
1/9/2014
|
|
333-193252
|
10.22*
|
|
Form of Restricted Stock Unit Award Agreement under the 2013 Equity Incentive Plan
|
|
X
|
|
|
|
|
|
|
10.23*
|
|
Form of Restricted Stock Unit Award Agreement under the 2013 Equity Incentive Plan (Executives)
|
|
X
|
|
|
|
|
|
|
21.1
|
|
List of Subsidiaries
|
|
|
|
Form 10-K
(Exhibit 21.1) |
|
2/25/2016
|
|
001-36065
|
23.1
|
|
Consent of Ernst & Young LLP, Independent Registered Public Accounting Firm
|
|
X
|
|
|
|
|
|
|
31.1
|
|
Certification of Principal Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
|
|
X
|
|
|
|
|
|
|
31.2
|
|
Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
|
|
X
|
|
|
|
|
|
|
32.1
|
|
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002
|
|
X
|
|
|
|
|
|
|
Exhibit
Number
|
|
Exhibit Description
|
|
Filed
with this
Report
|
|
Incorporated by
Reference
herein from
Form or
Schedule
|
|
Filing Date
|
|
SEC File/Reg.
Number
|
101
|
|
The following financial information from the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2016 formatted in XBRL (eXtensible Business Reporting Language): (i) Consolidated Balance Sheets as of December 31, 2016 and December 31, 2015, (ii) Consolidated Statements of Operations and Comprehensive Loss for the years ended December 31, 2016, 2015 and 2014, (iii) Consolidated Statements of Stockholders' Equity for the years ended December 31, 2016, 2015 and 2014, (iv) Consolidated Statements of Cash Flows for the years ended December 31, 2016, 2015 and 2014, and (v) Notes to Consolidated Financial Statements
|
|
X
|
|
|
|
|
|
|
+
|
Confidential treatment has been granted by, or is being requested from, the Securities and Exchange Commission as to certain portions of this Exhibit, which portions have been omitted and filed separately with the Securities and Exchange Commission as part of an application for confidential treatment pursuant to the Securities Act of 1933, as amended, and the Securities Exchange Act of 1934, as amended, as applicable.
|
*
|
Management contract or compensatory plan or arrangement.
|
|
ACCELERON PHARMA INC.
|
||
Date: March 1, 2017
|
By:
|
|
/s/ HABIB J. DABLE
|
|
|
|
Habib J. Dable
Chief Executive Officer, President and Director
|
Signature
|
|
Title
|
|
Date
|
|
|
|
|
|
/s/ HABIB J. DABLE
|
|
Chief Executive Officer, President and Director (Principal Executive Officer)
|
|
March 1, 2017
|
Habib J. Dable
|
|
|
||
|
|
|
|
|
/s/ KEVIN F. MCLAUGHLIN
|
|
Senior Vice President, Chief Financial Officer and Treasurer (Principal Financial Officer and Principal Accounting Officer)
|
|
March 1, 2017
|
Kevin F. McLaughlin
|
|
|
||
|
|
|
|
|
/s/ FRANCOIS NADER, M.D.
|
|
Chair of the Board of Directors
|
|
March 1, 2017
|
Francois Nader, M.D.
|
|
|
||
|
|
|
|
|
/s/ JEAN M. GEORGE
|
|
Director
|
|
March 1, 2017
|
Jean M. George
|
|
|
||
|
|
|
|
|
/s/ GEORGE GOLUMBESKI, PH.D.
|
|
Director
|
|
March 1, 2017
|
George Golumbeski, Ph.D.
|
|
|
||
|
|
|
|
|
/s/ TERRENCE C. KEARNEY
|
|
Director
|
|
March 1, 2017
|
Terrence C. Kearney
|
|
|
||
|
|
|
|
|
/s/ TOM MANIATIS, PH.D.
|
|
Director
|
|
March 1, 2017
|
Tom Maniatis, Ph.D.
|
|
|
||
|
|
|
|
|
/s/ THOMAS MCCOURT
|
|
Director
|
|
March 1, 2017
|
Thomas McCourt
|
|
|
||
|
|
|
|
|
/s/ RICHARD F. POPS
|
|
Director
|
|
March 1, 2017
|
Richard F. Pops
|
|
|
||
|
|
|
|
|
/s/ JOSEPH S. ZAKRZEWSKI
|
|
Director
|
|
March 1, 2017
|
Joseph S. Zakrzewski
|
|
|
1.
|
Section 4.3 of the Agreement is deleted in its entirety and replaced with the following:
|
Pre-Market Milestones
|
Payments
|
Due Date
|
|
|
|
Enrollment of first patient in the first clinical trial (Clinical Trials.gov Identifier: NCT01727336) initiated after the Effective Date with an ALK1 Product for a Patented Use:
|
$[***]
|
November 15, 2015 (Paid October 29, 2015)
|
|
|
|
Enrollment of first patient in the first Phase 3 Clinical Trial with an ALK1 Product for a Patented Use:
|
$[***]
|
(1)
|
|
|
|
Post-Market Milestones
|
Payments
|
Due Date
|
|
|
|
First commercial sale of a Patented Product:
|
$[***]
|
(2)
|
(1)
|
The first $[***] of the milestone shall be due within forty-five (45) days after the date of achievement of the milestone by Licensee, a Licensee Affiliate or a Sublicensee, and the remaining $[***] of the milestone shall be due within forty-five (45) days after the one-year anniversary of achievement of the milestone by Licensee, a Licensee Affiliate or a Sublicensee.
|
(2)
|
Payment shall be due within forty-five (45) days after the date of achievement by Licensee, a Licensee Affiliate or a Sublicensee of the milestone."
|
ACCELERON PHARMA INC.
|
|
BETH ISRAEL DEACONESS MEDICAL CENTER
|
||
|
|
|
||
BY:
/s/John Knopf, Ph.D.
Name: John Knopf, Ph.D.
TITLE:
CEO
DATE:
11/15/16
|
|
BY:
/s/Vikas P. Sukhatme, M.D., Sc.D.
Name: Vikas P. Sukhatme, M.D., Sc.D.
TITLE:
Chief Academic Officer
DATE:
November 7, 2016
|
Name:
|
[
●]
|
Number of Shares of Stock subject to Stock Option:
|
[
●]
|
Exercise Price Per Share:
|
$[
●]
|
Date of Grant:
|
[
●]
|
Vesting Start Date:
|
[
●]
|
(a)
|
“
Beneficiary
” means, in the event of the Optionee’s death, the beneficiary named in the written designation (in form acceptable to the Administrator) most recently filed with the Administrator by the Optionee prior to the Optionee’s death and not subsequently revoked, or, if there is no such designated beneficiary, the executor or administrator of the Optionee’s estate. An effective beneficiary designation will be treated as having been revoked only upon receipt by the Administrator, prior to the Optionee’s death, of an instrument of revocation in form acceptable to the Administrator.
|
(b)
|
“
Employment
” means, unless otherwise determined by the Administrator, the Participant’s employment with the Company or its Affiliates. Unless otherwise determined by the Administrator, the Participant’s Employment will terminate at such time as the Participant ceases to be an employee of the Company or its Affiliates.
|
(c)
|
“
Option Holder
” means the Optionee or, if as of the relevant time the Stock Option has passed to a Beneficiary, the Beneficiary.
|
(a)
|
Vesting
. As used herein with respect to the Stock Option or any portion thereof, the term “vest” means to become exercisable and the term “vested” as applied to any outstanding Stock Option means that the Stock Option is then exercisable, subject in each case to the terms of the Plan. Unless earlier terminated, forfeited, relinquished or expired, the Stock Option will vest as to [●]. Notwithstanding the foregoing, Shares subject to the Stock Option shall not vest on any vesting date unless the Optionee has remained in continuous Employment from the Date of Grant through such vesting date.
|
(b)
|
Exercise of the Stock Option
. No portion of the Stock Option may be exercised until such portion vests. Each election to exercise any vested portion of the Stock Option will be subject to the terms and conditions of the Plan and shall be in writing, signed by the Option Holder (or in such other form as is acceptable to the Administrator). Each such written exercise election must be received by the Company at its principal office or by such other party as the Administrator may prescribe and be accompanied by payment in full as provided in the Plan. The exercise price may be paid (i) by cash or check acceptable to the Administrator, (ii) to the extent permitted by the Administrator, through a broker-assisted cashless exercise program acceptable to the Administrator, (iii) by such other means, if any, as may be acceptable to the Administrator, or (iv) by any combination of the foregoing permissible forms of payment. In the event that the Stock Option is exercised by a person other than the Optionee, the Company will be under no obligation to deliver Shares hereunder unless and until it is satisfied as to the authority of the Option Holder to exercise the Stock Option and compliance with applicable securities laws. The latest date on which the Stock Option or any portion thereof may be exercised will be the 10th anniversary of the Date of Grant (the “
Final Exercise Date
”);
provided
,
however
, if at such time the Optionee is prohibited by applicable law or written Company policy applicable to similarly situated employees from engaging in any open-market sales of Stock, the Final Exercise Date will be automatically extended to thirty (30) days following the date the Optionee is no longer prohibited from engaging in such open-market sales. If the Stock Option is not exercised by the Final Exercise Date the Stock Option or any remaining portion thereof will thereupon immediately terminate.
|
(c)
|
Treatment of the Stock Option Upon Cessation of Employment
. If the Optionee’s Employment ceases, the Stock Option, to the extent not already vested will be immediately forfeited, and any vested portion of the Stock Option that is then outstanding will be treated as follows:
|
(i)
|
Subject to clauses (ii) and (iii) below and Section 4 of this Agreement, the Stock Option, to the extent vested immediately prior to the cessation of the Optionee’s Employment, will remain exercisable until the earlier of (A) the date that is three months following the date of such cessation of Employment, or (B) the Final Exercise Date, and except to the extent previously exercised as permitted by this Section 3(c)(i) will thereupon immediately terminate.
|
(ii)
|
Subject to clauses (iii) below and Section 4 of this Agreement, the Stock Option, to the extent vested immediately prior to the cessation of the Optionee’s Employment due to death, will remain exercisable until the earlier of (A) the first
|
(iii)
|
If the Optionee’s Employment is terminated by the Company or its Affiliates in connection with an act or failure to act constituting Cause (as the Administrator, in its sole discretion, may determine), or such termination occurs in circumstances that in the determination of the Administrator would have entitled the Company and its subsidiaries to terminate the Optionee’s Employment for Cause, the Stock Option (whether or not vested) will immediately terminate and be forfeited upon such termination.
|
(a)
|
The Administrator may cancel, rescind, withhold or otherwise limit or restrict the Stock Option at any time if the Optionee is not in compliance with all applicable provisions of this Agreement and the Plan.
|
(b)
|
By accepting the Stock Option, the Optionee expressly acknowledges and agrees that his or her rights, and those of any permitted transferee of the Stock Option, under the Stock Option, including to any Stock acquired under the Stock Option or proceeds from the disposition thereof, are subject to Section 6(a)(5) of the Plan (including any successor provision). Nothing in the preceding sentence shall be construed as limiting the general application of Section 8 of this Agreement.
|
Name:
|
[●]
|
Number of Restricted Stock Units subject to Award:
|
[●]
|
Date of Grant:
|
[●]
|
(a)
|
Time-Based Vesting
. This Award shall vest with respect to [●]. Notwithstanding the foregoing, no Restricted Stock Units shall vest on any vesting date unless the Participant has remained in continuous Employment from the Date of Grant through such vesting date.
|
(b)
|
Termination of Employment
. Automatically and immediately upon the cessation of the Participant’s Employment (i) the unvested portion of this Award shall terminate and be forfeited for no consideration, and (ii) the vested portion of this Award, if any, shall terminate and be forfeited for no consideration if the Participant’s Employment is terminated by the Company or its Affiliates in connection with an act or failure to act constituting Cause (as the Administrator, in its sole discretion, may determine), or if such termination occurs in circumstances that in the determination of the Administrator would have entitled the Company or its Affiliates to terminate the Participant’s Employment for Cause.
|
(a)
|
No shares of Stock will be delivered pursuant to this Award unless and until the Participant shall have remitted to the Company in cash or by check an amount sufficient to satisfy any federal, state or local withholding tax requirements or tax payments, or shall have made other arrangements satisfactory to the Administrator with respect to such taxes.
|
(b)
|
The Participant acknowledges and agrees that the minimum federal, state and local tax withholding due in connection with the vesting and settlement of the Restricted Stock Units (or portion thereof) may, in the Administrator’s sole discretion, be satisfied by the Company, pursuant to such procedures as it may specify from time to time, withholding a number of shares of Stock otherwise deliverable upon settlement of the Restricted Stock
|
(c)
|
The Participant authorizes the Company and its subsidiaries to withhold any amounts due in respect of any required tax withholdings or payments from any amounts otherwise owed to the Participant, but nothing in this sentence shall be construed as relieving the Participant of any liability for satisfying his or her obligation under the preceding provisions of this Section 8.
|
Name:
|
[●]
|
Number of Restricted Stock Units subject to Award:
|
[●]
|
Date of Grant:
|
[●]
|
(a)
|
Vesting Conditions
. Except as provided in Section 2(b) and Section 2(c) below, this Award shall vest:
|
(i)
|
on [●] (the “
Initial Vesting Date
”) as to any Restricted Stock Unit that became an Earned Unit on or prior to the Initial Vesting Date, and
|
(ii)
|
following the Initial Vesting Date, as follows: (A) on each subsequent “Determination Date” (as such term is defined in
Schedule A
) as to any Restricted Stock Unit that is then determined in accordance with Schedule A to have become an Earned Unit, and (B) on [●] with respect to any remaining unvested Restricted Stock Units;
|
(b)
|
Termination of Employment
. Automatically and immediately upon the termination of the Participant’s Employment for any reason, all outstanding Restricted Stock Units, if any,
|
(i)
|
any Restricted Stock Units that had already vested but had not yet been settled pursuant to Section 3 below at the time of such termination of Employment shall remain outstanding until settlement in the ordinary course pursuant to Section 3 below; provided, that this Section 2(b)(i) shall not apply if the termination of the Participant’s Employment was a termination by the Company or its Affiliates in connection with an act or failure to act constituting Cause (as the Administrator, in its sole discretion, may determine), or occurred in circumstances that in the determination of the Administrator would have entitled the Company or its Affiliates to terminate the Participant’s Employment for Cause (“
Cause Circumstances
”); and
|
(ii)
|
if the Participant’s Employment is terminated by the Company or its Affiliates without Cause and in the absence of any Cause Circumstances or is terminated by the Participant for Good Reason (as such term is defined in the Participant’s employment agreement with the Company, to the extent such agreement remains in effect), and if such termination occurs on or prior to the Initial Vesting Date, any Restricted Stock Units previously determined to have become Earned Units shall vest immediately upon such termination.
|
(c)
|
Change in Control
. Notwithstanding the foregoing,
|
(i)
|
if a Change in Control occurs and this Award is not continued or assumed (including by the grant of a substitute award) in connection with the Change in Control, all then outstanding and unvested Restricted Stock Units shall vest immediately upon the Change in Control, and
|
(ii)
|
if a Change in Control occurs and this Award is continued or assumed (including by the grant of a substitute award) in connection with the Change in Control, and if within one year following the date of the Change in Control the Participant’s Employment is terminated, without Cause and in the absence of Cause Circumstances or is terminated by the Participant for Good Reason, all unvested Restricted Stock Units that are outstanding immediately prior to such termination shall vest immediately upon such termination. In any case where this Section 2(c)(ii) applies, references in this Agreement to “Restricted Stock Unit” shall include any continued, assumed or substitute award referred to in the preceding sentence.
|
(a)
|
No shares of Stock will be delivered pursuant to this Award unless and until the Participant shall have remitted to the Company in cash or by check an amount sufficient to satisfy any federal, state or local withholding tax requirements or tax payments, or shall have made other arrangements satisfactory to the Administrator with respect to such taxes.
|
(b)
|
The Participant acknowledges and agrees that the minimum federal, state and local tax withholding due in connection with the vesting and settlement of the Restricted Stock Units (or portion thereof) may, in the Administrator’s sole discretion, be satisfied by the Company, pursuant to such procedures as it may specify from time to time, withholding a number of shares of Stock otherwise deliverable upon settlement of the Restricted Stock Units (or portion thereof) having an aggregate fair market value sufficient to satisfy all or part, as determined by the Administrator, of such federal, state and local withholding tax requirements. In addition, the Administrator hereby reserves the discretion to use any one or more methods permitted by the Plan to satisfy the Participant’s obligations with respect to the federal, state and local withholding tax requirements attributable to the Restricted Stock Units, or portion thereof, being settled.
|
(c)
|
The Participant authorizes the Company and its subsidiaries to withhold any amounts due in respect of any required tax withholdings or payments from any amounts otherwise owed to the Participant, but nothing in this sentence shall be construed as relieving the Participant of any liability for satisfying his or her obligation under the preceding provisions of this Section 8.
|
(1)
|
Registration Statement (Form S-8 No. 333-192789) pertaining to the Acceleron Pharma Inc. 2003 Stock Option and Restricted Stock Plan and the Acceleron Pharma Inc. 2013 Equity Incentive Plan,
|
(2)
|
Registration Statement (Form S-8 No. 333-198259) pertaining to the Acceleron Pharma Inc. 2013 Employee Stock Purchase Plan and the Acceleron Pharma Inc. 2013 Equity Incentive Plan,
|
(3)
|
Registration Statement (Form S-8 No. 333-203354) pertaining to the Acceleron Pharma Inc. 2013 Equity Incentive Plan,
|
(4)
|
Registration Statement (Form S-3 No. 333-208845) of Acceleron Pharma Inc., and
|
(5)
|
Registration Statement (Form S-8 No. 333-211867) pertaining to the Acceleron Pharma Inc. 2013 Equity Incentive Plan;
|
|
|
/s/ Ernst & Young LLP
|
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;
|
March 1, 2017
|
|
/s/ Habib J. Dable
|
Date
|
Habib J. Dable
|
|
|
Chief Executive Officer and President
(Principal Executive Officer)
|
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;
|
March 1, 2017
|
|
/s/ Kevin F. McLaughlin
|
Date
|
Kevin F. McLaughlin
|
|
|
Senior Vice President, Chief Financial Officer and Treasurer (Principal Financial Officer)
|
Date: March 1, 2017
|
By:
|
/s/ Habib J. Dable
|
|
|
Habib J. Dable
|
|
|
President and Chief Executive Officer
(Principal Executive Officer)
|
|
|
|
|
|
|
Date: March 1, 2017
|
By:
|
/s/ Kevin F. McLaughlin
|
|
|
Kevin F. McLaughlin
|
|
|
Senior Vice President, Chief Financial Officer and Treasurer (Principal Financial Officer)
|
|
|
|