NASDAQ:ABEO
READ THE FULL ABEO RESEARCH REPORT
Business Update
Type A Meeting with FDA Complete; BLA Resubmission in 2H24
Abeona Therapeutics, Inc. (NASDAQ:ABEO) is currently working on the resubmission of the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Recently, Abeona completed a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the resubmission. During the meeting, Abeona shared data and reports addressing nearly all the deficiencies noted in the Complete Response Letter (CRL). The company and the FDA came to preliminary alignment on those issues pending formal review of the data. There are two remaining outstanding items related to sterility assays and identity assays, with validation currently ongoing under protocols that incorporate FDA feedback. The company remains on track to resubmit the BLA for pz-cel before the end of the year. Following acceptance of the BLA, we anticipate a PDUFA date 6 months from the date of resubmission.
In regards to commercial preparation, the company is now focused on payer discussions such that patients can have greater and faster access upon approval. The company recently announced that the Centers for Medicare and Medicaid Services (CMS) has granted a product-specific procedure code ICD-10-PCS for pz-cel. If pz-cel is approved, the code will allow for efficient and accurate documentation, billing, and analysis of inpatient hospital procedures using pz-cel. The code will go into effect on Oct. 1, 2024. In addition, CMS assigned Medicare reimbursement of pz-cel to Pre-Major Diagnostic Category, Medicare Severity Diagnosis Related Group 018 (Pre-MDC MS-DRG 018), which is among the highest available inpatient hospital reimbursement levels for cell and gene therapies.
The company reported it is continuing discussions with the payer community and these discussions are generating positive results, which is likely to lead to favorable coverage and broad patient access following approval of pz-cel. Discussions are also continuing with experienced EB centers and the company remains on track for potential launch approximately 3 months after the potential pz-cel approval.
Beacon Therapeutics to Evaluate AAV204 Capsid for Ophthalmology Indications
In July 2024, Abeona announced an agreement with Beacon Therapeutics in which Beacon will evaluate Abeona’s AAV204 capsid for the development and commercialization of gene therapies for select ophthalmology indications. The agreement will give Beacon 12-months to evaluate AAV204 with an option to take a worldwide, non-exclusive license to use AAV204 in up to five gene or disease targets. Abeona will receive an upfront payment upon Beacon’s exercise of its option to license AAV204, with additional payments upon certain development, regulatory, and sales milestones along with tiered royalties on sales of products incorporating AAV204. The targets that Beacon would license are distinct from those currently in development at Abeona.
Financial Update
On August 12, 2024, Abeona announced financial results for the second quarter of 2024. The company did not report any revenue in the second quarter of 2024. R&D expenses in the second quarter of 2024 were $9.2 million compared to $8.5 million for the second quarter of 2023. The increase was primarily due to increased salary and related costs partially offset by decrease clinical and development work. G&A expenses in the second quarter of 2024 were $8.6 million compared to $5.0 million for the second quarter of 2023. The increase was primarily due to increased salaries, pre-commercial preparation costs, non-cash stock-based compensation, and professional fees.
Abeona exited the second quarter of 2024 with approximately $123.0 million in cash, cash equivalents, short term investments, and restricted cash. We estimate the company now has sufficient capital to fund operations into 2026, which does not take into account any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher (PRV), if awarded by the FDA. As of August 2, 2024, the company had approximately 43.3 million shares outstanding and, when factoring in stock options and warrants, a fully diluted share count of approximately 55.1 million.
Conclusion
The company continues to be on track to resubmit the BLA in the second half of 2024 following a successful Type A meeting with the FDA. The discussions with payers and EB centers appear to be going well, which should lead to a successful commercial launch of pz-cel, if approved. With no changes to our model our valuation remains at $7.50.
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