UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): June 19, 2019

 

 

ARCTURUS THERAPEUTICS HOLDINGS INC.

(Exact name of registrant as specified in its charter)

 

 

 

Delaware   001-38942   46-1981974

(State or other jurisdiction

of incorporation)

 

(Commission

File Number)

 

(I.R.S. Employer

Identification No.)

10628 Science Center Drive, Suite 250

San Diego, California 92121

(Address of principal executive offices)

Registrant’s telephone number, including area code: (858) 900-2660

Arcturus Therapeutics Ltd.

(Former name or former address, if changed since last report)

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company  ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading

Symbol(s)

 

Name of each exchange

on which registered

Common stock, par value $0.001 per share   ARCT   The NASDAQ Stock Market LLC

 

 

 


Item 7.01

Regulation FD Disclosure

Arcturus Therapeutics Holdings Inc. (the “ Company ”) has made available a presentation about the Company’s business (the “ Presentation ”), a copy of which is filed as Exhibit 99.1 to this Current Report on Form 8-K (the “ Report ”) and is hereby incorporated by reference.

The furnishing of the Presentation is not an admission as to the materiality of any information therein. The information contained in the Presentation is summary information that should be considered in the context of the Company’s filings with the Securities and Exchange Commission (the “ SEC ”) and other public announcements the Company may make by press release or otherwise from time to time. The Presentation speaks as of the date of this Report. While the Company may elect to update the Presentation in the future to reflect events and circumstances occurring or existing after the date of this Report, the Company specifically disclaims any obligation to do so.

The Presentation contains forward-looking statements, and as a result, investors should not place undue reliance on these forward-looking statements.

Cautionary Note Regarding Forward-Looking Statements

Certain statements in this communication are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, but are not limited to, statements about: expectations regarding our capitalization and resources; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials; our strategy and focus; the development and commercial potential of any of our product candidates; the timing and success of our development efforts; the success of any of our trials and our ability to achieve regulatory approval for any product candidate; the entry into or modification or termination of collaborative agreements; the date that an IND may be filed with the FDA; the potential market or success for the clinical development programs of the Company; and any statements other than statements of historical fact, including those related to the Company’s future cash, market or financial position. These forward-looking statements are subject to numerous assumptions, risks and uncertainties which change over time. Forward-looking statements speak only as of the date they are made and the Company assumes no duty to update forward-looking statements, except as required by law.

In addition to factors previously disclosed in the Company’s reports filed with the SEC and those identified elsewhere in this communication, the following factors, among others, could cause actual results to differ materially from forward-looking statements and historical performance: the availability and access, in general, of funds to fund operations and necessary capital expenditures, the strength of our intellectual property portfolio, the expected safety profiles of our product candidates, the proof of concept of our product candidates, the target opportunities for certain of our product candidates, our expected dates of submission of one or more Investigational New Drug Applications to the FDA, market opportunities with respect to certain diseases connected with our product candidates, manufacturing and formulation capabilities of the Company or our manufacturing partners with respect to our product candidates, as well as our management’s response to the preceding factors. The foregoing factors are in addition to the other factors set forth in the Company’s reports on Form 10-K, Form 8-K, and other documents on file with the SEC.

Other risks and uncertainties are more fully described under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018, filed with the SEC on March 18, 2019, and in other filings that the Company makes and will make with the SEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. The statements made in this Current Report on Form 8-K and the exhibit(s) attached hereto speak only as of the date stated herein, and subsequent events and developments may cause the Company’s expectations and beliefs to change. While the Company may elect to update these forward-looking statements publicly at some point in the future, the Company specifically disclaims any obligation to do so, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the Company’s views as of any date after the date stated herein.


Item 9.01

Financial Statements and Exhibits.

(d) Exhibits

 

Exhibit

Number

  

Description

99.1    Presentation of Arcturus Therapeutics Holdings Inc., dated June 2019


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    Arcturus Therapeutics Holdings Inc.
Date: June 19, 2019    
    By:  

/s/ Joseph E. Payne

    Name:   Joseph E. Payne
    Title:   Chief Executive Officer

SLIDE 0

Building the Next Generation of RNA Medicines ARCTURUS THERAPEUTICS June 2019 Exhibit 99.1


SLIDE 1

FORWARD LOOKING STATEMENTS ARCTURUS THERAPEUTICS This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future performances or achievements expressed or implied by the forward-looking statements. Each of these statements is based only on current information, assumptions and expectations that are inherently subject to change and involve a number of risks and uncertainties. Forward-looking statements include, but are not limited to, statements about: expectations regarding our capitalization and resources; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials; our strategy and focus; the development and commercial potential of any of our product candidates; the timing and success of our development efforts; the success of any of our trials and our ability to achieve regulatory approval for any product candidate; the entry into or modification or termination of collaborative agreements; the date that an IND may be filed with the FDA; the potential market or success for the clinical development programs of Arcturus; and any statements other than statements of historical fact, including those related to Arcturus’ future cash, market or financial position. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward looking statements. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing, and you should not place undue reliance on such forward-looking statements.  The forward-looking statements contained or implied in this press presentation are subject to other risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus’ Annual Report on Form 10-K for the fiscal year ended December 31, 2018, filed with the SEC on March 18, 2019 and in subsequent filings with, or submissions to, the SEC. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.   BUILDING INNOVATIVE RNA MEDICINES


SLIDE 2

Arcturus is an mRNA Medicines Drug Development Company Focused on Rare Diseases HQ: San Diego; Founded: 2013; Nasdaq: ARCT Outstanding Shares: 10.8 M; Employees: 85; Insider Ownership: 24% BUILDING INNOVATIVE RNA MEDICINES Broad and Strong Intellectual Property Portfolio 152 Patents & Patent Applications LUNAR® Delivery Technology RNA Drug Substance & Drug Product Process Manufacturing Investment Highlights ARCTURUS THERAPEUTICS LUNAR® Delivery Platform Validated by Multiple Strategic Partners More than $1 Billion in potential milestones and royalties Promising Preclinical Safety Data for LUNAR® Delivery and mRNA Drug Products


SLIDE 3

Arcturus mRNA Medicines BUILDING INNOVATIVE RNA MEDICINES Key Value Drivers: mRNA Medicines & Platform ARCTURUS THERAPEUTICS Arcturus LUNAR® Delivery Platform: Enabling Genetic Medicines Strategic Partners: More than $1 Billion in Potential Milestones & Royalties OTC Deficiency market potential $500M annual sales LUNAR-CF to treat Cystic Fibrosis (CF); Funded by the Class I CF market potential $900M annual sales LUNAR-OTC (ARCT-810) to treat Ornithine Transcarbamylase (OTC) Deficiency


SLIDE 4

LUNAR® Mechanism of Delivery LUNAR Associates with Cell Membrane Enters Cell Via Endocytosis Lipid Particle in Endosome pH-Mediated Disruption Rapid Biodegradation of Vehicle RNA in Cytosol RNA Processing and Translation Increased Acidity as Endosome Ages ARCTURUS THERAPEUTICS BUILDING INNOVATIVE RNA MEDICINES


SLIDE 5

BUILDING INNOVATIVE RNA MEDICINES Name Partner Indication Arcturus Chemistry Arcturus Delivery mRNA Process LUNAR-HBV Hepatitis B RNA LUNAR® Hepatocytes ARCT LUNAR-NASH NASH RNA LUNAR® Stellate Cells ARCT LUNAR-GSD3 Glycogen Storage Disease Type III mRNA LUNAR® Hepatocytes ARCT LUNAR-RARE Undisclosed Rare Disease mRNA LUNAR® Hepatocytes ARCT LUNAR-RPL RNA Replicon Large Pharma Infectious Disease Prophylactic Vaccines SGI’s Replicon RNA LUNAR® Undisclosed Arcturus Platform: Enabling Genetic Medicines Greater than $1 Billion in Potential Milestones & Royalties Enabling Different Types of RNA – Messenger RNA, Gene Editing RNA, Replicon RNA Multiple Cell Types Targeted 5 ARCTURUS THERAPEUTICS


SLIDE 6

LUNAR® Platform Substantial Preclinical Proof-of-Concept Demonstrated Vaccines Respiratory Ophthalmology Liver Cancer Inf. Disease Infectious Disease LUNAR-CF mRNA Intratracheal Inhalation Gene Editing RNA mRNA Animal Health mRNA Shared Antigens Ultragenyx LUNAR-GSD3 mRNA Takeda LUNAR-NASH LUNAR-OTC (ARCT-810) mRNA mRNA Intravitreal Sub-retinal Janssen siRNA triplicate Hepatocytes Stellate Cells LUNAR® Platform Preclinical Proof-of-Concept Demonstrated in Hepatocytes, Liver Stellate Cells, Bronchial Epithelial Cells (Lung), Photoreceptors (Eye), Infectious Diseases, Cancer Vaccines BUILDING INNOVATIVE RNA MEDICINES mRNA Neoantigens 6


SLIDE 7

Target Opportunities for LUNAR® Delivery Platform Exceed $100 Billion in Potential Value Transcription Factors Immuno-oncology (non-vaccine) Vaccines Respiratory Ophthalmology CNS Liver Cancer Inf. Disease Oral Intracellular Targets Gene Editing Immune System Transcription Factors Transcription Factors Transcription Factors Transcription Factors Transcription Factors mRNA Intracellular Targets Intracellular Targets Intracellular Targets Intracellular Targets Gene Editing Infectious Disease (non-vaccine) Gene Editing Gene Editing Transcription Factors Gene Editing Gene Editing Gene Editing mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA mRNA geRNA DNA Kidney Transcription Factors Intracellular Targets Gene Editing mRNA geRNA DNA Spleen Transcription Factors Intracellular Targets Gene Editing mRNA geRNA DNA Topical Transcription Factors Intracellular Targets Gene Editing mRNA geRNA DNA BUILDING INNOVATIVE RNA MEDICINES


SLIDE 8

Arcturus Pipeline of mRNA Medicines Name Indication IND Date Route of Administration Target Organ Target Cells Prevalence Worldwide LUNAR-OTC (ARCT-810) Ornithine Transcarbamylase (OTC) Deficiency Q1 2020 Intravenous (i.v.) Liver Hepatocytes > 10,000 LUNAR-CF Cystic Fibrosis H2 2020 Nebulized Aerosol to Lung Lung Bronchial Epithelial Cells > 70,000 LUNAR-GSD3 (UX053) Glycogen Storage Disease Type III 2020 Intravenous (i.v.) Liver Hepatocytes BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Pipeline programs focus on messenger RNA (mRNA) drug products for rare diseases LUNAR-OTC (ARCT-810, intravenous mRNA medicine): Investigational New Drug (IND) Filing Target Q1 2020 LUNAR-CF is funded by the Cystic Fibrosis (CF) Foundation – IND Target H2 2020 LUNAR-GSD3 (UX053) is a licensed program, partnered with Ultragenyx – IND Target 2020


SLIDE 9

OTC Deficiency Market Opportunity LUNAR-OTC Aims to Restore Enzyme Function Expression of OTC enzyme in liver has potential to restore normal urea cycle activity to detoxify ammonia, preventing neurological damage and removing need for liver transplantation Unmet Medical Need Present standard of care involves a strict diet (low protein, high fluid intake) plus ammonia scavengers (sodium phenylbutyrate) Present standard of care does not effectively prevent spikes of ammonia. OTC Deficiency patients are typically referred for liver transplant. Ornithine Transcarbamylase (OTC) Deficiency: The most common urea cycle disorder The urea cycle converts neurotoxic ammonia to water-soluble urea that can be excreted in urine Deficiency in OTC causes elevated blood ammonia, which can lead to neurological damage, coma, and death 10,000 worldwide prevalence BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS


SLIDE 10

LUNAR-OTC 10 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Disease Normalization Following Single and Repeat Dosing in OTC Mouse Model Survival of OTC-deficient Mice on High Protein Diet Weekly LUNAR-OTC Treatment


SLIDE 11

LUNAR-OTC 11 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS Exceeds Therapeutic Target of 10% Enzyme Replacement at all Doses in OTC-Deficient Mouse Model LUNAR-OTC treatment increases OTC expression in mouse periportal hepatocytes (main site of ureagenesis) Therapeutic Target = 10% OTCD impacts ureagenesis (ammonia detoxification) The main site of ureagenesis is the periportal region of the liver* Establishing 10% of natural enzyme levels is expected to be therapeutically significant *Li, L. et al. PGC-1α Promotes Ureagenesis in Mouse Periportal Hepatocytes through SIRT3 and SIRT5 in Response to Glucagon. Scientific Reports. 6:24156 | DOI: 10.1038/srep24156, April 2016


SLIDE 12

Arcturus Safety Profile 12 BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS External Validation Multiple strategic partnerships over many years confirms the positive safety profile of Arcturus LUNAR® and mRNA Arcturus is committed to developing safe mRNA products 15 studies over several years with strategic partners Over $3 Million invested to date   Top Safety Concern for RNA Medicines is Delivery Arcturus LUNAR® Delivery Technology is well tolerated in non-human primates (NHPs) @ 15 mg/kg single dose of non-coding RNA @ 3 mg/kg x eight (8) weekly doses of non-coding RNA (total of 24 mg/kg over 2 months)   Arcturus mRNA chemistry shows promising efficacy and tolerability data Efficacy of OTC mRNA in mouse model @ 0.1 – 1 mg/kg Well tolerated in mouse @ 7 mg/kg single dose   IND-enabling toxicology studies at higher doses will provide Maximum Tolerated Dose (MTD)


SLIDE 13

LUNAR-CF Aims to Restore CFTR Function An mRNA replacement therapy has the potential to deliver a new copy of CFTR into the lungs of CF-patients, independent of any genotype A functional CFTR protein can restore chloride channel efflux in the airways, reducing mucus accumulation, tissue scarring and minimizing the progressive respiratory dysfunction observed in CF-patients Cystic Fibrosis: The most common rare disease in the United States Caused by genetic mutations in the CFTR gene, resulting in aberrant flux of ions in and out of cells, causing thick mucus buildup in lung airways Chronic airway obstruction leads to infection and inflammation, which causes permanent tissue scarring and respiratory failure 70,000 worldwide prevalence Cystic Fibrosis Market Opportunity Unmet Medical Need No CFTR functional corrector is approved for treatment of all patients Present standard of care does not effectively prevent long-term effects of mucus accumulation. CF patients with late-stage loss of respiratory function require lung transplant BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS


SLIDE 14

LUNAR® Targeting Lung NEBULIZATION AND DELIVERY TO LUNG Functional Delivery of LUNAR®-mRNA into Lung Epithelial Cells LUNAR-Luc mRNA LUNAR® Delivery into Bronchial Epithelial Cells (BECs) Nebulization TdTomato/FoxJ1/Dapi BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS


SLIDE 15

Drug Substance: mRNA Design INTERNAL RNA DEVELOPMENT PLATFORMS Proprietary mRNA Optimization Platform Demonstrates Sustained Activity Upon Repeat Dosing in NHPs Weekly Dosing in Non-Human Primates Arcturus’ proprietary mRNA optimization platform Optimized conditions mRNA sequence Chemistry Process optimization Improved protein expression and duration Improved functional activity Sustained hEPO activity in NHPs upon repeat dosing BUILDING INNOVATIVE RNA MEDICINES ARCTURUS THERAPEUTICS


SLIDE 16

Arcturus mRNA Manufacturing Arcturus’ Internal mRNA Production: Up to 30 g in Less Than One Week ARCTURUS THERAPEUTICS Features Benefits Optimized IVT Method Reduced Cost; Higher Purity Improved Capping Reaction Reduced Cost of Goods Proprietary Purification Process Higher Purity in a Shorter Time Efficient Entire Process Less Than One Week Scalable to > 1Kg Access Large Patient Populations Adaptable Can Utilize a Variety of Modifications BUILDING INNOVATIVE RNA MEDICINES DNA Template Production IVT and Capping Reaction Purification Process Buffer Exchange & Concentration


SLIDE 17

BUILDING INNOVATIVE RNA MEDICINES Drug Product: LUNAR® Formulation & Production PROPRIETARY REPRODUCIBLE & SCALABLE PRODUCTION PROCESS LUNAR® Has Been Successfully Scaled From Milligram to Multigram Batch Sizes LUNAR® Reproducibility LUNAR® Scalability Particle Size % Encapsulated RNA % Encapsulated RNA Particle Size ARCTURUS THERAPEUTICS


SLIDE 18

Management Team ARCTURUS THERAPEUTICS Joseph E. Payne, MSc Founder, President & CEO Andrew Sassine, MBA CFO Dr. Pad Chivukula Founder, CSO & COO Kevin Skol, MBA Sr. VP of Business Development & Alliance Management Dr. Suezanne Parker VP of Translational Biology BUILDING INNOVATIVE RNA MEDICINES


SLIDE 19