The clinical hold, the company explained, is due to concerns about EB-101’s stability during transport to clinical sites.
Abeona officials say the company is working closely with the FDA to resolve the issues.
RDEB is a rare and incurable form of epidermolysis bullosa (EB), a genetic skin disorder in which a patient’s skin blisters and tears easily, causing painful wounds and scarring. Patients are often called “butterfly children” because their skin is so fragile, like a butterfly’s wings. The disorder is caused by genetic mutations in the COL7A1 gene, which provides instructions for making a protein called type 7 collagen (COL7) that is essential for maintaining healthy skin.
EB-101 is a form of gene-corrected cell therapy that involves delivering the corrected version of the COL7A1 gene to patients’ skin cells cultured in a lab dish. These cells are then transplanted back to the patients, so that their skin regains the ability to produce COL7.
In a prior Phase 1/2 clinical trial (NCT01263379), EB-101 was well-tolerated by RDEB patients and led to significant and sustained improvements in wound healing over the course of five years. EB-101 led to continuous production of COL7 in patients’ skin cells up to two years after treatment, and did not pose any drug-related side effects to study participants.
Abeona is now planning to launch the Phase 3 trial, called VIITAL, to continue exploring the therapeutic potential of EB-101 for the treatment of RDEB.
However, the FDA recently sent a letter to the company ordering that the trial be put on hold until Abeona provides additional data showing that the therapy maintains its stability during the process of transport and delivery to patients.
Abeona has been working closely with the FDA for the past year to tackle issues related to Chemical, Manufacturing and Controls (CMC). Its officials now are committed to providing additional evidence of the therapy’s transport stability to address the concerns raised by the agency.
“Initiating the VIITAL pivotal Phase 3 trial for EB-101 is the company’s top priority,” João Siffert, MD, CEO of Abeona, said in a press release. “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.”
Despite the setback, Abeona is still expecting to receive the FDA’s go-ahead to initiate VIITAL before the end of the year.
“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.”