FDA puts the brakes on a rare disease biotech’s PhIII as regulators wait for an update — shares crumble
Little Abeona Therapeutics $ABEO is back in trouble this morning.
The biotech reported that the FDA is slamming the brakes on their Phase III study of EB-101 for rare cases of recessive dystrophic epidermolysis bullosa, which is characterized by fragile skin that blisters quickly.
The drug uses gene transfer to deliver COL7A1 genes into a patient’s own skin cells, which are then put back into the patient’s skin for improved wound healing. It has RMAT and breakthrough status at the FDA but doesn’t have approval for the Phase III.
It won’t get that green light until the company provides “additional data points” on transport stability.
The biotech’s shares were quickly hammered on the news, dropping more than 20%.
João Siffert, the CEO of Abeona, got the job last fall after Alexion vet and then new CEO Carsten Thiel was booted after the board accused him of personal misconduct. The biotech started the day with a market cap of $160 million.
“Initiating the VIITAL pivotal Phase III trial for EB-101 is the Company’s top priority,” said Siffert in a statement. “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. Looking ahead, we believe that completion of our CMC work coupled with the durable safety and efficacy data, now out to five years in some patients, will ultimately be critical to support a future Biologics License Application.”