On March 31, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, reported a business and strategy update and reported 2021 financial results (Press release, Abeona Therapeutics, MAR 31, 2022, View Source [SID1234611234]).

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"We are committed to developing novel cell and gene therapies for patients with rare diseases with no approved treatment options," said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. "We are focused on EB-101 and, having recently achieved target enrollment in our pivotal Phase 3 VIITAL study, have increased confidence that we will share topline results in the third quarter of 2022. We also expect animal proof-of-concept data from our preclinical eye programs beginning in the second half of 2022 that could support pre-IND meetings with the FDA. We believe the strategic steps announced today reflect the operating discipline needed to extend our cash runway beyond these near-term catalysts."

Strategy and Business Update

●Following a comprehensive portfolio review, Abeona announced today that it is focusing research and development (R&D) resources on the topline data readout for its EB-101 pivotal Phase 3 VIITAL study while the Company actively pursues a potential commercialization partner for EB-101.
●Target enrollment was achieved in Abeona’s EB-101 pivotal Phase 3 VIITAL study for recessive dystrophic epidermolysis bullosa (RDEB). The Company anticipates topline results for the co-primary endpoints related to wound healing and pain reduction measured at 24 weeks post treatment in the third quarter of 2022. Abeona received positive feedback from the U.S. Food and Drug Administration (FDA) related to a Type B meeting on the proposed Chemistry, Manufacturing and Controls (CMC) requirements of the EB-101 development program, gaining alignment on the characterization and validation plans that could support a potential Biologics License Application (BLA) for EB-101 in RDEB.

●In connection with its shift in priorities, the Company has intensified its pursuit of a strategic partnership to take over development activities for ABO-102, and has ceased build-out of additional AAV manufacturing space. As part of the FDA’s feedback on the Transpher A Statistical Analysis Plan (SAP) in January 2022, the agency recommended that all participants be followed to an age of at least 60 months, which would shift timing of the neurocognitive outcomes data readout to late-2024/early-2025, as compared to the Company’s prior projection of the second quarter of 2023.
●As part of the Company’s portfolio prioritization, Abeona will discontinue development of ABO-101 for MPS IIIB.
●Abeona plans to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based gene therapies using the novel AIM capsid platform and internal AAV vector research programs. Abeona will present results from testing of novel AAV capsids in non-human primates at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting being held on May 1-4, 2022 in Denver, CO. The preclinical data could support pre-IND meetings with the FDA for Abeona’s undisclosed eye gene therapy indications. The Company previously reported preclinical data showing the potential for AIM AAV vectors to efficiently target the photoreceptor and retinal epithelium cell layers after intravitreal injection, creating the potential for new pipeline candidates that can address multiple ophthalmic disorders.
●The strategic changes will reduce the Company’s operating expenses and extend the estimated runway of current cash resources to mid-2023.
●In December 2021, Abeona raised approximately $17.5 million in aggregate gross proceeds, before underwriting discounts and commissions and other offering expenses, from an underwritten public offering of common stock.
●Joseph Vazzano was appointed as Chief Financial Officer (CFO) at Abeona, and will serve as the Company’s principal financial officer and principal accounting officer effective March 31, 2022. Mr. Vazzano previously served as CFO of Avenue Therapeutics, Inc. (Nasdaq: ATXI), where he secured multiple equity financings for Avenue and served in a leadership role for signing a complex, two-stage acquisition of the company with future contingent value rights.

Full Year 2021 Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $50.9 million as of December 31, 2021, compared to $96.0 million as of December 31, 2020. Net cash used in operating activities was $65.7 million for the full year of 2021, including a $20 million payment in November 2021 in accordance with a settlement agreement with REGENXBIO. Net cash used in operating activities was $35.0 million for the full year of 2020.

License and other revenues for the full year of 2021 were $3.0 million, compared to $10.0 million in 2020. The revenue in 2021 resulted from a clinical milestone achieved in December 2021 under a sublicense agreement with Taysha Gene Therapies for ABO-202 for CLN1 disease.

R&D expenses were $34.3 million for the full year of 2021, compared to $30.1 million in 2020. General and administrative (G&A) expenses were $22.8 million for the full year of 2021, compared to $23.8 million in 2020.

Net loss was $84.9 million for the full year of 2021, or a $0.86 basic and diluted loss per common share as compared to a net loss of $84.2 million, or a $0.91 basic and diluted loss per common share, in 2020. The net loss in 2021 included a non-cash goodwill impairment charge of $32.5 million. The impairment charge has no impact on the Company’s cash position, cash flow from operating activities, and does not have any impact on future operations.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, Thursday, March 31, 2022 at 8:30 a.m. ET, to discuss its full year 2021 financial results and business update. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 851784 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.