Little Abeona gets big setback with FDA clinical hold on pivotal phase 3

FDA Building
The FDA will not let Abeona Therapeutics' phase 3 trial go ahead until "data points on transport stability" are sorted. (FDA)

Just under a year ago, Abeona Therapeutics rejected its CEO; now, it’s the biotech’s turn to be spurned, as the FDA has slapped the company with a clinical hold.  

The trial hold comes for the planned phase 3 test of EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications.

The FDA told the biotech it won’t give a green light to this test “until it submits to the FDA additional data points on transport stability of EB-101 to clinical sites.”

In a statement, the New York- and Cleveland-based company said it had been “working closely” with the U.S. regulator “to address and narrow open Chemical, Manufacturing and Controls (CMC) items and has been working to resolve this one item identified in the FDA clinical hold letter.”

It believes the CMC clearance for the so-called VIITAL trial will be gained in the fourth quarter.

“Initiating the VIITAL pivotal phase 3 trial for EB-101 is the company’s top priority,” said João Siffert, M.D., 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.

“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. We remain deeply committed to advancing EB-101 to provide a desperately needed treatment for RDEB patients.”

EB-101 works by using gene transfer to deliver COL7A1 genes into a patient’s own skin cells (keratinocytes) and transplanting them back to the patient.

It’s been an up-and-down year for the company: Last November, ex-CEO Carsten Thiel was sacked after an investigation into “allegations of misconduct towards colleagues” and was replaced by head of R&D Siffert on an interim basis. Siffert became Abeona's chief in February.

Industry veteran Thiel—who was formerly chief commercial officer and executive vice president at Alexion—had only been with Abeona for a few months, joining the company last April to spearhead its march toward commercialization of its cell and gene therapies.

In a strongly worded statement, Abeona said its decision stems from “personal misconduct that violated the company’s code of business conduct and ethics” and was not related to the condition of the company’s finances, operations or clinical programs. Thiel later became EUSA’s new president of Europe.

RELATED: EUSA Pharma nabs new executives, including the recently ousted Abeona CEO

The biotech’s stock was down nearly 12% yesterday on the news and fell more than 5% after-hours, with a market cap of $140 million.

It has several earlier-stage assets in its pipeline, including ABO-102 and ABO-101, AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively.