CLEVELAND – Abeona Therapeutics Inc. (NASDAQ:), a clinical-stage biopharmaceutical company, announced today the resubmission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for prademagene zamikeracel (pz-cel), an investigational gene therapy intended for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
The company’s CEO, Vish Seshadri, expressed confidence that the resubmission addresses all items identified in the FDA’s previous Complete Response Letter, including the Chemistry Manufacturing and Controls (CMC) requirements. The resubmission follows a Type A meeting held in August 2024, where Abeona and the FDA agreed on the content of the resubmission.
The FDA’s Complete Response Letter, issued in April 2024, did not raise concerns regarding the clinical efficacy or safety data of pz-cel, nor did it require additional clinical trials for approval. The BLA resubmission is supported by data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study. Abeona expects the FDA to set a new Prescription Drug User Fee Act (PDUFA) target action date upon acceptance of the BLA resubmission.
Prademagene zamikeracel (pz-cel) is a cell-based gene therapy developed for RDEB, a rare genetic skin disorder caused by mutations in the COL7A1 gene. The therapy involves using the patient’s own genetically corrected skin cells to express collagen VII, which is necessary for binding the epidermis to the dermis. Pz-cel has received several designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy.
Abeona’s manufacturing facility, which produced pz-cel for the Phase 3 trial, is prepared for commercial production pending FDA approval. The company also has a portfolio of AAV-based gene therapies for ophthalmic diseases and is exploring next-generation AAV capsids for a range of diseases.
This news article is based on a press release statement and provides an update on Abeona’s efforts to bring its gene therapy, pz-cel, closer to approval and commercial availability for patients with RDEB.
In other recent news, Abeona Therapeutics has made significant strides in its operations, with a focus on gene therapy advancements. The company has expanded its board with the addition of Dr. Bernhardt G. Zeiher and Dr. Eric Crombez, who bring extensive experience in drug development and clinical programs for rare genetic disorders. Abeona has also received a product-specific procedure code from the Centers for Medicare and Medicaid Services for its gene therapy candidate pz-cel, which could streamline hospital billing and reimbursement processes.
The company is nearing the resubmission of its Biologics License Application for pz-cel, expected in the latter half of 2024. Abeona has also partnered with Beacon Therapeutics to explore potential gene therapies for eye diseases, focusing on Abeona’s patented AAV204 capsid.
Financial services firms H.C. Wainwright and Stifel have reaffirmed their positive outlook on Abeona, highlighting the potential of the company’s gene therapy advancements. The company reported a net income of $7.4 million for Q2 of 2024 and successfully closed a $75 million securities offering in May 2024. These developments underscore Abeona’s ongoing commitment to advancing its gene therapy initiatives.
InvestingPro Insights
As Abeona Therapeutics Inc. (NASDAQ:ABEO) resubmits its Biologics License Application for prademagene zamikeracel, investors should consider some key financial metrics and insights from InvestingPro.
According to InvestingPro data, Abeona’s market capitalization stands at $276.78 million, reflecting the market’s current valuation of the company’s potential. This relatively modest market cap underscores the importance of the FDA’s decision on the BLA resubmission for the company’s future.
An InvestingPro Tip indicates that Abeona holds more cash than debt on its balance sheet, which could provide financial flexibility as the company navigates the regulatory process and prepares for potential commercialization of pz-cel. This strong cash position aligns with the company’s readiness for commercial production mentioned in the article.
However, another InvestingPro Tip notes that Abeona is quickly burning through cash, which is not uncommon for clinical-stage biopharmaceutical companies investing heavily in research and development. This cash burn rate emphasizes the importance of achieving regulatory milestones and potentially bringing pz-cel to market.
The stock has shown strong performance recently, with InvestingPro data revealing a 72.24% price total return over the past year and an impressive 85.76% return over the last six months. This positive momentum may reflect investor optimism about the company’s progress with pz-cel and its potential approval.
It’s worth noting that InvestingPro offers 11 additional tips for Abeona Therapeutics, providing a more comprehensive analysis for investors interested in deeper insights into the company’s financial health and market position.
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