Patient Follow-up Completed in VIITAL Trial of EB-101 for RDEB
Pivotal Phase 3 trial assessed wound healing 6 months after cell therapy
Patient follow-up has now been completed in VIITAL, a pivotal Phase 3 trial testing EB-101 in people with recessive dystrophic epidermolysis bullosa (RDEB), according to Abeona Therapeutics, the therapy’s developer.
Top-line results from VIITAL (NCT04227106) are expected before the end of November and, if positive, will support an application seeking EB-101’s approval in the U.S. The company is planning to submit that request to the U.S. Food and Drug Administration (FDA).
“Completion of the last patient’s 6-month follow-up visit marks a key milestone that enables us to report key findings from our Phase 3 VIITAL study of EB-101 in RDEB,” Vish Seshadri, Abeona’s CEO, said in a press release.
“We thank the patients, their families, and the clinical investigators, and plan to report topline results from this pivotal study within the next month,” added Seshadri.
RDEB is caused by mutations in the COL7A1 gene, which contains instructions for producing a portion of type VII collagen — a protein needed to maintain the structural integrity of the skin. Mutations in COL7A1 affect the production of type VII collagen, and leads to severe skin blisters and wounds in patients.
A gene-corrected cell therapy, EB-101 delivers a lab-made healthy copy of COL7A1 into patients’ skin cells. These cells are subsequently transplanted back into the patient to stimulate wound healing.
VIITAL aimed to enroll up to 15 patients, ages 6 and older, from Stanford University Medical Center, in Palo Alto, California, and UMass Memorial Medical Center, in Worcester, Massachusetts.
Patients had approximately 36 large chronic wound pairs in total. Each wound had a minimum size of 20 square centimeters (about 3.1 square inches). In each pair, one wound was treated, while the other was left untreated for at least six months.
The trial aimed to assess the number of wounds that healed by at least 50% from the study’s start. This was evaluated by comparing wound pairs at 24 weeks, or about six months, after the transplant.
The other main goal was evaluating any reduction in pain related to changing wound dressings — the bandages used to clean, cover, and protect patient sores. This was assessed by evaluating mean differences in Wong-Baker FACES scores between treated and untreated wounds at the end of the study.
Now, Abeona is checking and organizing all the results from the trial to ensure the database is closed and can be ready within a few weeks.
The therapy was given rare pediatric disease designation by the FDA. It offers incentives to support the clinical development of treatments for children with serious or life-threatening rare diseases, defined in the U.S. as those affecting fewer than 200,000 patients. One of the incentives is an accelerated FDA review.
EB-101 also was granted orphan drug status in both the U.S. and Europe. That designation is meant to advance the development of potential rare disease treatments by providing several benefits to their manufacturers. One of these benefits is entitlement to a period of market exclusivity upon approval, which lasts seven years in the U.S. and 10 years in Europe.