Abeona Therapeutics has overcome an FDA clinical hold to show that its cell therapy can heal wounds in patients with a rare connective tissue disease, teeing up an FDA filing in the new year.
Back in 2019, Abeona was left reeling from an FDA clinical hold on the autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, a rare connective tissue disorder that causes severe skin wounds and pain and can lead to systemic complications. The agency requested additional data on the therapy’s transport stability.
But now, the small biotech has overcome those challenges to present phase 3 evidence that EB-101 led to 50% wound healing and reduced pain, meeting the co-primary endpoints of the study called VIITAL. The 11 patients in the trial had large, chronic wounds that had been open for at least six months to as long as 21 years.
Abeona now has eyes on the FDA again, with a new drug application slated for the second quarter of 2023. The therapy was previously granted orphan drug and rare pediatric disease tags.
The company hopes that EB-101 can be a one-time therapy to improve healing and reduce pain in patients with the most challenging wounds caused by the debilitating disease, commonly known as butterfly disease.
Abeona CEO Vish Seshadri said the VIITAL trial differed from others conducted in the same disease because it used an endpoint of patient-reported pain, which means the results show that EB-101 can improve daily life for patients with the disease.
EB-101 was well tolerated with no serious treatment-related adverse events observed, however, two patients experienced at least one serious adverse event that were deemed unrelated to the treatment. Four patients reported treatment-emergent adverse events of procedural pain, muscle spasms and itchiness, while eight patients experienced infections unrelated to treatment.
Abeona intends to present data from the trial at a future medical meeting.