CLEVELAND – Abeona Therapeutics Inc. (NASDAQ:), a clinical-stage biopharmaceutical firm, introduced immediately the resubmission of its Biologics License Software (BLA) to the U.S. Meals and Drug Administration (FDA) for prademagene zamikeracel (pz-cel), an investigational gene remedy meant for the remedy of recessive dystrophic epidermolysis bullosa (RDEB).
The corporate’s CEO, Vish Seshadri, expressed confidence that the resubmission addresses all objects recognized within the FDA’s earlier Full Response Letter, together with the Chemistry Manufacturing and Controls (CMC) necessities. The resubmission follows a Kind A gathering held in August 2024, the place Abeona and the FDA agreed on the content material of the resubmission.
The FDA’s Full Response Letter, issued in April 2024, didn’t increase considerations concerning the scientific efficacy or security information of pz-cel, nor did it require extra scientific trials for approval. The BLA resubmission is supported by information from the pivotal Section 3 VIITAL™ examine and a Section 1/2a examine. Abeona expects the FDA to set a brand new Prescription Drug Person Payment Act (PDUFA) goal motion date upon acceptance of the BLA resubmission.
Prademagene zamikeracel (pz-cel) is a cell-based gene remedy developed for RDEB, a uncommon genetic pores and skin dysfunction attributable to mutations within the COL7A1 gene. The remedy includes utilizing the affected person’s personal genetically corrected pores and skin cells to specific collagen VII, which is critical for binding the dermis to the dermis. Pz-cel has obtained a number of designations from the FDA, together with Regenerative Medication Superior Remedy and Breakthrough Remedy.
Abeona’s manufacturing facility, which produced pz-cel for the Section 3 trial, is ready for business manufacturing pending FDA approval. The corporate additionally has a portfolio of AAV-based gene therapies for ophthalmic ailments and is exploring next-generation AAV capsids for a spread of ailments.
This information article is predicated on a press launch assertion and supplies an replace on Abeona’s efforts to carry its gene remedy, pz-cel, nearer to approval and business availability for sufferers with RDEB.
In different latest information, Abeona Therapeutics has made important strides in its operations, with a concentrate on gene remedy developments. The corporate has expanded its board with the addition of Dr. Bernhardt G. Zeiher and Dr. Eric Crombez, who carry intensive expertise in drug improvement and scientific packages for uncommon genetic problems. Abeona has additionally obtained a product-specific process code from the Facilities for Medicare and Medicaid Companies for its gene remedy candidate pz-cel, which might streamline hospital billing and reimbursement processes.
The corporate is nearing the resubmission of its Biologics License Software for pz-cel, anticipated within the latter half of 2024. Abeona has additionally partnered with Beacon Therapeutics to discover potential gene therapies for eye ailments, specializing in Abeona’s patented AAV204 capsid.
Monetary providers companies H.C. Wainwright and Stifel have reaffirmed their constructive outlook on Abeona, highlighting the potential of the corporate’s gene remedy developments. The corporate reported a internet earnings of $7.4 million for Q2 of 2024 and efficiently closed a $75 million securities providing in Might 2024. These developments underscore Abeona’s ongoing dedication to advancing its gene remedy initiatives.
InvestingPro Insights
As Abeona Therapeutics Inc. (NASDAQ:ABEO) resubmits its Biologics License Software for prademagene zamikeracel, traders ought to contemplate some key monetary metrics and insights from InvestingPro.
In response to InvestingPro information, Abeona’s market capitalization stands at $276.78 million, reflecting the market’s present valuation of the corporate’s potential. This comparatively modest market cap underscores the significance of the FDA’s choice on the BLA resubmission for the corporate’s future.
An InvestingPro Tip signifies that Abeona holds additional cash than debt on its stability sheet, which might present monetary flexibility as the corporate navigates the regulatory course of and prepares for potential commercialization of pz-cel. This robust money place aligns with the corporate’s readiness for business manufacturing talked about within the article.
Nonetheless, one other InvestingPro Tip notes that Abeona is rapidly burning via money, which isn’t unusual for clinical-stage biopharmaceutical corporations investing closely in analysis and improvement. This money burn fee emphasizes the significance of attaining regulatory milestones and probably bringing pz-cel to market.
The inventory has proven robust efficiency not too long ago, with InvestingPro information revealing a 72.24% worth whole return over the previous 12 months and a powerful 85.76% return over the past six months. This constructive momentum might replicate investor optimism concerning the firm’s progress with pz-cel and its potential approval.
It is price noting that InvestingPro affords 11 extra suggestions for Abeona Therapeutics, offering a extra complete evaluation for traders occupied with deeper insights into the corporate’s monetary well being and market place.
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