CLEVELAND - Abeona Therapeutics Inc. (NASDAQ: NASDAQ:ABEO) disclosed on Monday that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) concerning its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), a gene-corrected therapy designed for patients with recessive dystrophic epidermolysis bullosa (RDEB).
The CRL, which follows a Late Cycle Review Meeting with the FDA in March, requests additional Chemistry Manufacturing and Controls (CMC) information. The FDA has not raised concerns about the clinical efficacy or safety data of pz-cel, nor has it requested new clinical trials. Still, the agency requires further validation of manufacturing and release testing methods.
Abeona's CEO, Vish Seshadri, expressed disappointment with the CRL but assured stakeholders of the company's commitment to addressing the FDA's requirements promptly. The company anticipates resubmitting the BLA with the requested CMC information in the third quarter of 2024.
The initial BLA submission, which received priority review designation, was supported by data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study, suggesting that pz-cel can deliver sustained wound healing and pain reduction with a single application on large and chronic wounds.
Abeona will host a conference call and webcast on Tuesday to discuss the details of the CMC information requested by the FDA. The webcast will be available on the company's investor relations website.
Prademagene zamikeracel is an investigational autologous therapy that aims to correct the genetic defect causing RDEB by incorporating a functional COL7A1 gene into the patient's skin cells. The treatment has received several designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy.
Abeona produces pz-cel at its gene and cell therapy manufacturing facility in Cleveland, Ohio, which is capable of supporting commercial production upon FDA approval.
This news is based on a press release statement from Abeona Therapeutics.
InvestingPro Insights
Amidst the recent FDA developments, Abeona Therapeutics Inc. (NASDAQ: ABEO) remains a company of interest for investors, especially considering the performance metrics and analysts' outlooks.
According to InvestingPro data, Abeona holds a market capitalization of $201.33 million, suggesting a significant presence in the biotech industry. Despite the challenges faced with the FDA's Complete Response Letter, analysts are optimistic about the company's sales growth in the current year, which is a positive sign for Abeona's market position.
InvestingPro Tips indicate that Abeona has more cash than debt on its balance sheet, a reassuring sign of financial health that may provide some stability as the company navigates the regulatory landscape. Moreover, the company's liquid assets exceed its short-term obligations, offering further evidence of a solid financial foundation.
Investors should note that Abeona has experienced a strong return over the last year, with a 135.09% price total return. This performance is complemented by a significant price uptick over the last six months, boasting an 82.85% return. These figures highlight the stock's recent momentum, which could be a factor for those considering an investment in Abeona.
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