Abeona readies manufacturing ahead of Phase 3 trial
- Abeona Therapeutics said it's finished the first 6,000 square feet of a planned 26,000-foot expansion of a manufacturing facility in Cleveland, Ohio.
- The progress means the company should be ready to manufacture its EB-101 cell therapy for recessive dystrophic epidermolysis bullosa in time for a Phase 3 study starting mid-year, Abeona said in a Monday statement.
- Abeona released the manufacturing progress report as part of its fourth quarter financial results. The company reported cash and equivalent holdings of $85 million, down from $112 million held at the end of the third quarter last year.
With biopharma companies often struggling to find contract manufacturing capacity for their complex therapies, Abeona is making a bet on in-house facilities.
"We believe that our platform provides us with distinct advantages, including flexibility, scale, reliability, and the potential for reduced development risk, cost, and faster times to market," the company stated in an annual financial filing with the Securities and Exchange Commission. The second stage of the expansion has started and includes an 8,000-square-foot lab space, the company said.
The Cleveland facility is "cost-effective and readily scalable," said Abeona CEO João Siffert told investors on a conference call Tuesday.
Abeona expects to be able to supply its EB-101 treatment for the upcoming Phase 3 study and to soon be ready for manufacturing on a commercial scale, Siffert said. The company will have enough capacity to allow treatment of as many as 120 patients a year from the outset and should be in a good position to move toward filing an application for U.S. regulatory approval if all goes to plan, he added.
The manufacturing work is encouraging, RBC Capital Markets analyst Kennen MacKay wrote in a note to investors. There's a "high likelihood" that Abeona will secure Food and Drug Administration Good Manufacturing Practice validation of the Cleveland facility in the second half of this year, he said.
Patients with epidermolysis bullosa have skin that is so fragile it blisters easily, even from something as minor as a rub or a scratch. Abeona's EB-101 is a gene-corrected autologous skin graft designed for patients with the most severe form of the disease, recessive dystrophic epidermolysis bullosa, or RDEB, which can cause a variety of complications and shortened lives.
The Phase 3 VITAL study will include between 10 to 15 patients with RDEB and assess how many patients' wounds have greater than 50% healing at three months. It will also include a general assessment of wounds and changes in pain and itch over the course of the trial, Siffert told investors.