Abeona to Submit More Data to FDA Before Starting Phase 3 Trial

Abeona Therapeutics (NASDAQ:ABEO) has been gearing up to begin its planned Phase 3 trial for EB-101, but the company hit a roadblock on Monday (September 23) after the US Food and Drug Administration (FDA) requested additional information on the drug. 

EB-101 is a cell therapy intended to treat recessive dystrophic epidermolysis bullosa (RDEB), which is a rare mutation of the COL7A gene that results in fragile skin. RDEB can cause severe pain and other impacts on quality of life.

In a press release, Abeona Therapeutics said the regulatory agency is asking for more data regarding transport stability of the drug to clinical sites.

Shares of the company took a hit on Monday, tumbling 11.96 percent to close the trading session at US$2.87. After-hours trading bumped its share price down an additional 5.23 percent to US$2.72 as of 5:33 p.m. EDT on Monday.

The Phase 3 trial, called VIITAL, is a top priority for the company, said Joao Siffert, CEO of Abeona.

“Efforts to gather supplemental data points on transport stability of EB-101 are ongoing and we are confident that the requested additional data will be submitted to the FDA promptly. We remain deeply committed to advancing EB-101 to provide a desperately needed treatment for RDEB patients.”

Abeona Therapeutics said it has been working with the FDA over the last year or so regarding Chemical, Manufacturing and Controls (CMC) items, and is now working to resolve the problem the agency identified in its clinical hold letter. According to the release, the company expects to receive CMC clearance for its Phase 3 trial in the fourth quarter of the year.

Once the Phase 3 trial gets the go ahead, enrolment will begin in Q4 2019 of 10 to 15 RDEB patients who will be treated with EB-101. The results will be compared to intra-patient untreated wounds.

In early 2018, Abeona Therapeutics received regenerative medicine advanced therapy (RMAT) designation for EB-101 in patients with RDEB. In its Phase 1/2 clinical trial of the treatment, EB-101 demonstrated a reduction in painful blisters resulting from injuries as well as improvements in wound healing. At the time, it was the first gene therapy RMAT designation awarded for the indication.

As it currently stands, there is no FDA-approved treatment for RDEB, which means, should everything go according to plan for the company, its treatment will be the first approved treatment for the indication.

Despite the share price drop on Monday, on TipRanks.com Abeona Therapeutics is currently ranked a “moderate buy” based off six analyst ratings. Shares of the company have an average price target of US$9.25, a 222.3 percent upside to its current price, with a high estimate of US$16 and a low of US$2.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.