Abeona Therapeutics(ABEO) - 2019 Q4 - Annual Report

Part I Business Abeona Therapeutics develops gene and cell therapies for rare genetic diseases, advancing lead programs and leveraging proprietary AIM™ capsid technology Our Pipeline Abeona's pipeline features lead programs EB-101, ABO-102, and ABO-101, alongside early-stage candidates leveraging the AIM™ capsid platform - EB-101 for RDEB: A pivotal Phase III clinical trial (VIITAL™ Study) was initiated in the first quarter of 2020. The primary endpoint is the proportion of treated wounds with >50% healing at three months[28] - ABO-102 for MPS IIIA: Ongoing Phase I/II trial data shows evidence of preserved neurocognitive development in younger patients, dose-related reduction in CSF heparan sulfate, and durable reduction of liver volume. No treatment-related serious adverse events have been reported[34][35] - ABO-101 for MPS IIIB: Ongoing Phase I/II trial shows clear signals of biologic effect with reduction of disease-specific biomarkers. The therapy has been well tolerated to date[39] - ABO-202 for CLN1 (Batten Disease): An Investigational New Drug (IND) application was approved in May 2019 based on preclinical data showing increased survival and improved function in animal models[43] - The company is developing next-generation therapies using its novel AIM™ capsid technology, which aims for improved tissue targeting and the potential to evade immunity to wildtype AAV vectors[13][53] Manufacturing Abeona established a cGMP manufacturing facility in Cleveland, Ohio, to control its supply chain, support clinical production, and develop scalable processes - The company established a state-of-the-art cGMP production facility in Cleveland, OH for manufacturing gene and cell therapies, aiming for flexibility, scale, and reduced development risk[14][54] - The facility build-out is phased, with the first two phases completed in 2018 and 2019 for clinical production and quality control labs. The final phase will be a commercial AAV facility[54] - Manufacturing processes are being developed for both autologous cell therapy (EB-101) and AAV-based vectors using transfection in HEK293 cells, with a focus on scalable suspension technology[55][56][60] Intellectual Property The company's intellectual property strategy combines in-house development with exclusive licenses for key programs and the AIM™ Capsid platform, including an agreement with REGENXBIO - The company has secured exclusive licenses for its key programs: MPS IIIA/IIIB from Nationwide Children's Hospital, CLN3 from the University of Nebraska, RDEB from Stanford University, and AIM™ capsids and CLN1 technology from UNC at Chapel Hill[65][67][68][69][70] - Abeona entered into a license agreement with REGENXBIO Inc. for its NAV AAV9 vector, involving a $20 million upfront payment, up to $100 million in annual fees, and potential milestones and royalties[73] - The company actively seeks patent protection for vector engineering, production processes, composition of matter, and methods of administration for its product candidates[16][64] Government Regulation Abeona's products are subject to extensive U.S. and EU regulations, including multi-phase clinical trials, expedited pathways for rare diseases, and post-approval compliance with healthcare laws - Gene therapy products are regulated by the FDA's Center for Biologics Evaluation and Research (CBER) and require an Investigational New Drug (IND) application before human clinical trials can begin[76][77] - The company's product candidates have received several expedited regulatory designations, including Orphan Drug, Fast Track, Breakthrough Therapy, and Regenerative Medicine Advanced Therapy (RMAT), which can accelerate the development and review process[26][111][113][117] - In the EU, advanced therapy medicinal products are governed by Regulation 1394/2007/EC, requiring a Marketing Authorization Application (MAA) to be reviewed by the EMA. Orphan designation in the EU provides 10 years of market exclusivity[134][137] - Commercialization is subject to healthcare laws like the federal Anti-Kickback Statute and data privacy regulations such as HIPAA in the U.S. and GDPR in Europe[142][145] Competition Abeona faces intense competition from pharmaceutical and biotechnology companies with greater resources, competing on efficacy, safety, cost, and regulatory approval speed - The company competes with major multinational pharmaceutical companies, specialized biotech firms, and research institutions, many of which have substantially greater financial, technical, and human resources[152] - Competitors may develop products that are safer, more effective, or less expensive, or obtain regulatory approval more rapidly, which could render Abeona's treatments obsolete or non-competitive[152][155] - Abeona is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases[9] - The company's lead clinical programs are EB-101 for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 for Sanfilippo syndrome type A (MPS IIIA), and ABO-101 for Sanfilippo syndrome type B (MPS IIIB)[9] - Key strategic pillars include advancing clinical programs, utilizing the novel AIM™ capsid technology, establishing commercial-scale manufacturing, forming strategic partnerships, and growing its intellectual property portfolio[11][12][14][15][16] Risk Factors The company faces significant risks in novel gene and cell therapy development, including unpredictable timelines, regulatory hurdles, manufacturing complexities, financial losses, and the need for substantial additional capital - The novelty of gene and cell therapies makes it difficult to predict development time and cost, with only a few such products approved in the U.S. and EU[164] - The company may face substantial delays in clinical studies, such as the clinical hold received for the EB-101 Phase III trial in September 2019, which was subsequently lifted in December 2019[170][172] - Manufacturing of product candidates is complex. For EB-101, challenges include biopsy viability and cell proliferation rates. The company currently lacks a backup manufacturer for EB-101 clinical trial supply[193][194] - The company has a history of losses, with an accumulated deficit of approximately $486.5 million as of December 31, 2019, and expects to incur future losses. It will require substantial additional capital to fund operations[302][303][304] - The company's ability to use its net operating loss carryforwards of approximately $241.9 million may be limited by ownership changes under U.S. federal income tax rules[329] - Recent and upcoming departures of senior management, including the Executive Chairman and Chief Financial Officer, could adversely affect the business and strategy[254][255] Unresolved Staff Comments There are no unresolved staff comments - Not Applicable[334] Properties The company leases its principal executive office in New York, manufacturing and lab space in Cleveland, and office space in Madrid, which are deemed sufficient Leased Properties | Location | Size (sq. ft.) | Type | Lease Expiration | | :--- | :--- | :--- | :--- | | New York, NY | 10,400 | Corporate HQ | January 2026 | | Cleveland, OH | 45,300 | Manufacturing/Lab/Office | December 2025 | | Madrid, Spain | 1,700 | Office | September 2020 | Legal Proceedings A putative securities class action lawsuit filed against the company and certain officers on November 1, 2019, was voluntarily dismissed by the plaintiff on January 10, 2020 - A securities class action lawsuit filed in November 2019, alleging false and misleading statements, was voluntarily dismissed by the plaintiff in January 2020[336] Mine Safety Disclosures This item is not applicable to the company - Not applicable[337] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Abeona's common stock trades on Nasdaq under 'ABEO', with no cash dividends planned, and the report details equity compensation plan information - The company's common stock trades on the Nasdaq Capital Market under the symbol 'ABEO'[339] - No cash dividends have ever been declared or paid, and none are anticipated in the foreseeable future[340] Equity Compensation Plan Information (as of Dec 31, 2019) | Plan Category | Number of securities to be issued upon exercise | Weighted-average exercise price | Number of securities remaining available for future issuance | | :--- | :--- | :--- | :--- | | Equity compensation plans approved by security holders | 6,055,395 | $8.17 | 911,456 | | Equity compensation plans not approved by security holders | - | - | - | | Total | 6,055,395 | $8.17 | 911,456 | Selected Financial Data The company reported no revenue in 2019, with net loss increasing to $76.3 million and total assets rising to $223.4 million due to increased cash Selected Statement of Operations Data (in thousands) | | 2019 | 2018 | 2017 | 2016 | 2015 | | :--- | :--- | :--- | :--- | :--- | :--- | | Total Revenues | $0 | $2,998 | $837 | $889 | $1,040 | | Research and development | $48,566 | $38,698 | $16,989 | $10,655 | $4,715 | | General and administrative | $20,705 | $20,106 | $10,943 | $13,290 | $14,320 | | Loss from operations | $(77,090) | $(58,166) | $(27,836) | $(23,881) | $(18,546) | | Net loss | $(76,282) | $(56,671) | $(27,319) | $(21,873) | $(14,526) | | Basic and diluted loss per common share | $(1.51) | $(1.19) | $(0.66) | $(0.64) | $(0.53) | Selected Balance Sheet Data (as of Dec 31, in thousands) | | 2019 | 2018 | 2017 | 2016 | 2015 | | :--- | :--- | :--- | :--- | :--- | :--- | | Total current assets | $132,390 | $88,851 | $140,592 | $69,421 | $40,568 | | Total assets | $223,382 | $174,399 | $178,766 | $111,058 | $80,055 | | Total liabilities | $44,952 | $40,354 | $8,668 | $11,960 | $12,334 | | Total stockholders' equity | $178,430 | $134,045 | $170,098 | $99,098 | $67,721 | Management's Discussion and Analysis of Financial Condition and Results of Operations Abeona's net loss increased to $76.3 million in 2019, while liquidity improved to $129.3 million cash, sufficient through Q2 2021, though more capital is needed Results of Operations In 2019, total revenues dropped to zero, R&D expenses increased by $9.9 million, and net loss widened to $76.3 million compared to 2018 Comparison of Operating Results (2019 vs. 2018, in millions) | Metric | 2019 | 2018 | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $0.0 | $3.0 | $(3.0) | | R&D Expenses | $48.6 | $38.7 | $9.9 | | G&A Expenses | $20.7 | $20.1 | $0.6 | | Net Loss | $(76.3) | $(56.7) | $(19.6) | | Loss Per Share | $(1.51) | $(1.19) | $(0.32) | - The increase in R&D expenses in 2019 was primarily driven by a $6.3 million increase in clinical and development work for gene and cell therapy candidates and a $4.3 million increase in salary and related costs[360] - Depreciation and amortization expenses increased by $5.4 million in 2019, mainly due to amortization of the REGENXBIO license cost and depreciation from the build-out of the Cleveland manufacturing facility[362] Liquidity and Capital Resources As of December 31, 2019, cash and equivalents increased to $129.3 million due to a $96.0 million public offering, with existing funds projected to last through Q2 2021, though more capital is needed Liquidity Snapshot (as of Dec 31, in millions) | Metric | 2019 | 2018 | | :--- | :--- | :--- | | Cash, Cash Equivalents & Short-term Investments | $129.3 | $85.0 | | Working Capital | $93.7 | $68.5 | | Accumulated Deficit | $(486.5) | $(410.2) | - In December 2019, the company closed a public offering of common stock and pre-funded warrants, raising net proceeds of $96.0 million[371][372] - The company believes its existing cash and cash equivalents are sufficient to fund operations through the second quarter of 2021[455] - Under the REGENXBIO license agreement, an $8 million payment (including $1 million interest) is due April 1, 2020, and a guaranteed $20 million payment is due November 4, 2020[374][388] Critical Accounting Policies and Estimates The company's critical accounting policies involve significant estimates for licensed technology, goodwill impairment, revenue recognition (ASC 606), R&D accruals, and share-based compensation using the Black-Scholes model - The company adopted ASC 606 (Revenue from Contracts with Customers) on January 1, 2018, resulting in a $6.3 million cumulative effect adjustment to stockholders' equity[401] - Licensed technology and goodwill are tested for impairment annually or more frequently if indicators are present. No impairments were recorded in 2017, 2018, or 2019[399][400][401] - The company accrues for R&D expenses by estimating the level of service performed and associated costs when invoices have not yet been received[406] - Share-based compensation for stock options is valued using the Black-Scholes model, with key assumptions including expected volatility (108% in 2019) and expected term (5.1 years in 2019)[407][519] Quantitative and Qualitative Disclosures About Market Risk The company's market risk exposure is limited, with minimal interest rate risk from short-duration U.S. government securities and no significant foreign currency or inflation impact - The company's exposure to interest rate risk is limited to its investment portfolio, which consists of short-duration U.S. government, agency, and treasury securities[410] - Exposure to foreign currency risk is not currently significant, and inflation has not had a material effect on the business[411] Financial Statements and Supplementary Data The company's audited consolidated financial statements and supplementary data are included in the report from pages F-1 through F-22 - The full financial statements are incorporated in the Annual Report on pages F-1 through F-22[413] Changes in and Disagreements With Accountants on Accounting and Financial Disclosure There were no changes in or disagreements with accountants on accounting and financial disclosure during the period - None reported[413] Controls and Procedures Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2019, with an unqualified audit opinion from Whitley Penn LLP - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2019[414] - Management's assessment concluded that the company maintained effective internal control over financial reporting as of December 31, 2019, based on the COSO 2013 framework[415] - The independent auditor, Whitley Penn LLP, audited the internal control over financial reporting and issued an unqualified opinion on its effectiveness[416][419] - No material changes were made to the internal control over financial reporting during the fourth quarter of 2019[417] Other Information There is no other information to report for this item - None[424] Part III Directors, Executive Officers and Corporate Governance Information on directors, executive officers, and corporate governance is incorporated by reference from the 2020 Proxy Statement, and the company has a Code of Business Conduct and Ethics - Information required by this item is incorporated by reference from the company's 2020 Proxy Statement[424] - The company has adopted a Code of Business Conduct and Ethics, available on its website[425] Executive Compensation The information regarding executive compensation is incorporated by reference from the company's 2020 Proxy Statement - Information required by this item is incorporated by reference from the company's 2020 Proxy Statement[427] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters The information regarding security ownership of certain beneficial owners, management, and related stockholder matters is incorporated by reference from the company's 2020 Proxy Statement - Information required by this item is incorporated by reference from the company's 2020 Proxy Statement[427] Certain Relationships and Related Transactions, and Director Independence The information regarding certain relationships, related transactions, and director independence is incorporated by reference from the company's 2020 Proxy Statement - Information required by this item is incorporated by reference from the company's 2020 Proxy Statement[427] Principal Accounting Fees and Services The information regarding principal accounting fees and services is incorporated by reference from the company's 2020 Proxy Statement - Information required by this item is incorporated by reference from the company's 2020 Proxy Statement[428] Part IV Exhibits, Financial Statement Schedules This section lists the financial statements and exhibits filed as part of the Form 10-K, including audited financial statements and key corporate and contractual documents - This item contains the list of financial statements filed with the report, which can be found on pages F-1 through F-22[430] - A list of exhibits filed with the report is provided, including key agreements such as the license agreement with REGENXBIO Inc. and various employment and compensation-related documents[431][432] Form 10-K Summary This item is not applicable - None[432]