Abeona Therapeutics, a New York-based biotechnology company, plans to ask the Food and Drug Administration to approve its cell therapy for an uncommon and painful tissue disease following positive results from a small clinical trial.
Known as epidermolysis bullosa, or EB, the disease makes the skin incredibly fragile and prone to chronic blistering. There are several forms of EB, with some estimated to affect one out of every 50,000 newborns. Abeona’s study specifically enrolled 11 patients with a more severe form called “recessive dystrophic.”
Across these patients, researchers evaluated 43 pairs of large open wounds over six months. Half of the wounds received Abeona’s therapy, EB-101, while the others went untreated. Results released by the company Thursday showed that 81% of the treated wounds had healed by 50% or more at the study’s cutoff, compared to 16% in the control group.
Patients in the treatment group also experienced significant reductions in pain, as measured by a scale often used to understand pain levels in children. Additionally, Abeona said its therapy was well tolerated, and that researchers had not observed any serious adverse events related to treatment.
Having hit the study’s main goals on wound healing and pain reduction, Abeona intends to submit an approval application to the FDA sometime between April and June next year. The agency has already granted EB-101 both Orphan Drug as well as Rare Pediatric Disease designations, which carry certain commercial benefits if the drug is approved.
Abeona’s therapy consists of a patient’s own skin cells, which are engineered outside of the body to include functional copies of a certain gene. While defective in patients with recessive dystrophic EB, this gene normally makes a type of collagen that strengthens and supports the skin. In a statement, Abeona’s CEO Vish Seshadri said the newly released data “reinforce the strong value proposition of EB-101 as a potential one-time therapy” for the disease.
To date, the FDA has not approved treatments for any form of EB. An experimental drug developed by Scioderm and later bought by Amicus Therapeutics had advanced into late-stage human testing, but negative results led Amicus to discontinue testing several years ago.
EB is currently managed by the painful process of re-bandaging and cleaning open wounds. Patients are also at elevated risk of infection and, in severe cases, can face much shorter life expectancies.
“Our patient community needs options to address not only the healing of wounds but also the chronic pain and the acute treatment related pain of daily wound care associated with these wounds,” said Brett Kopelan, a board member of the Alliance for Regenerative Medicine and the executive director of Debra of America, a nonprofit that supports EB patients like his 15-year-old daughter.
“Today’s standard of care comprises hours of brutal and painful wound care, and EB-101’s promise to be a transformational option for RDEB patients is truly exciting,” Kopelan added in a statement provided by Abeona.
Alongside the trial results, Abeona said Thursday it would raise $35 million by selling shares of its common stock to a group of new and existing institutional investors in a private placement. Previously, Abeona had been in danger of getting delisted from the Nasdaq stock exchange, but in May the company announced it had won a six-month extension that goes through Nov. 14.
Abeona’s share price fell by more than 15% Thursday morning, to trade below $3.75.