Abeona Therapeutics On Track for Biologics License Application submission in Q3 2023

Abeona Therapeutics, Inc. (Nasdaq: ABEO) has announced its financial results for the second quarter of 2023, along with significant progress toward key corporate milestones. The company is gearing up for the submission of a Biologics License Application (BLA) for EB-101, a treatment for recessive dystrophic epidermolysis bullosa (RDEB), in the third quarter of 2023.

Highlights:

EB-101 Commercialization: With a recent successful capital raise of $25 million through a registered direct offering, Abeona is now positioned to commence preparations for the commercial launch of EB-101 in the United States. This funding also extends the company’s operational runway well beyond the anticipated regulatory approval timeframe for EB-101.

 Progress in RDEB Treatment: Abeona has achieved significant milestones in the development of EB-101 for RDEB treatment. The company successfully completed the Process Performance Qualification manufacturing runs for both the retroviral vector (RVV) and the EB-101 drug product manufacturing. This achievement underscores Abeona’s validated process and its readiness for commercial production.

FDA Collaboration: Abeona has responded to the U.S. Food and Drug Administration’s (FDA) requests for additional data supporting comparability between two RVV sources used in EB-101 clinical studies. This data will be included in the chemistry, manufacturing, and controls (CMC) module for the EB-101 BLA submission.

Pre-BLA Meeting: The company has submitted a briefing package for a pre-BLA meeting with the FDA, scheduled for August 2023. This meeting aims to facilitate the smooth BLA submission process for EB-101 in the treatment of RDEB, which is anticipated in the third quarter of 2023.

Positive Clinical Data: Abeona presented additional efficacy and safety data from the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB at notable dermatology conferences. The promising top-line data from the VIITAL study had been reported in November 2022.

Preclinical Advancements: Abeona’s preclinical programs in gene therapy are making strides. The company successfully completed pre-Investigational New Drug Application (IND) meetings with the FDA regarding two preclinical gene therapy product candidates from its adeno-associated virus (AAV) ophthalmology programs. IND-enabling studies are set to begin in the latter half of 2023.

Financial Overview: As of June 30, 2023, Abeona had cash, cash equivalents, restricted cash, and short-term investments totaling $37.1 million. This is prior to the $25 million registered direct offering in July 2023. The company estimates that these resources, combined with the proceeds from the offering, will fund operations into the fourth quarter of 2024.

Financial Performance: In the second quarter of 2023, license and other revenues reached $3.5 million, compared to $1.0 million in the same period of 2022. Research and development expenses for Q2 2023 were $8.5 million, while general and administrative expenses totaled $5.0 million. The net loss attributable to common shareholders for Q2 2023 was $16.7 million, compared to a net loss of $7.9 million in Q2 2022.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s lead clinical program is EB-101, its investigational autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona’s fully integrated cell and gene therapy cGMP manufacturing facility produced EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and potential commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

 

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Abeona Therapeutics On Track for Biologics License Application submission in Q3 2023

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