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NASDAQ:ABEO
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Business Update
BLA Submitted; Awaiting FDA Decision on Acceptance and Priority Review
On September 26, 2023, Abeona Therapeutics, Inc. (NASDAQ:ABEO) announced that the company had submitted the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) to seek approval for prademagene zamikeracel (pz-cel; formerly EB-101), the company’s engineered cell therapy being developed for recessive dystrophic epidermolysis bullosa (RDEB). Abeona requested Priority Review, which if granted would shorten the review time from the usual 10 months to six months from acceptance of the BLA. We anticipate the FDA’s decision regarding acceptance of the BLA and whether Priority Review will be granted by the end of November 2023.
In November 2022, Abeona announced positive topline results from the Phase 3 VIITAL™ study showing pz-cel met the two co-primary endpoints while exhibiting a clean safety and tolerability profile. In May 2023, Abeona announced additional data from the Phase 3 VIITAL™ study, including data showing pz-cel improved would healing in as early as six weeks, a significant reduction in pain for pz-cel-treated wounds compared to control wounds, and numerous quality of life improvements. For a full discussion of the Phase 3 VIITAL data please see our initiation report (here) and our previous update (here).
During the third quarter conference call, the company reiterated that it will have the capacity at launch to treat approximately 120 patients per year. Abeona will be onboarding approximately five to seven treatment centers, which are each equipped to care for one to two EB patients per month and are geographically dispersed across the U.S. The company recently conducted an advisory board meeting with eight prominent physicians, at which time there was a discussion concerning patient and caregiver testimonials, the clinical data and care coordination needed for surgical application of pz-cel was discussed, and before and after pictures were shared. The physician’s provided very constructive feedback and expressed interest in initiating onboarding discussions.
The company is also working with commercial and government health insurance systems to ensure broad access for all patients. Early feedback has been positive and the company is currently using a “7-digit price point” for peak sales estimates, which the company currently puts at $500 million. Abeona will look to ramp up production over time from the initial 120 patients per year based on the experience with the initial treatment centers and the peak sales estimate was made assuming capacity will expand to approximately 500 patients per year over the next five years.