Phase 3 Trial of EB-101 for RDEB Meets Target Enrollment

Teresa Carvalho, MS avatar

by Teresa Carvalho, MS |

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RDEB, Phase 3 Trial, EB-101 | Epidermolysis Bullosa News | illustration of clinical trial graph

The pivotal Phase 3 VIITAL clinical trial of EB-101, Abeona Therapeutics’ experimental cell therapy for wound healing in people with recessive dystrophic epidermolysis bullosa (RDEB), has met its enrollment target.

The company also announced that top-line results from the VIITAL trial (NCT04227106) are expected in the in third quarter of this year.

“Meeting the target accrual of VIITAL marks an important milestone for Abeona and underscores the high unmet need in RDEB where there are currently no approved therapies,” Vish Seshadri, PhD, CEO of Abeona, said in a press release.

RDEB is caused by mutations in the gene COL7A1, which is responsible for producing a portion of type VII collagen, a protein needed to keep skin layers attached. Mutations in COL7A1 disrupt the production of type VII collagen, resulting in severe skin blisters and wounds.

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EB-101 is a gene-corrected cell therapy that is designed to deliver in the lab a healthy version of COL7A1 into a patient’s own skin cells. Those cells are then transplanted back into the patient to facilitate wound healing.

VIITAL is aimed at assessing the safety and effectiveness of EB-101 in at least 10 participants, ages 6 and older, recruited at Stanford University Medical Center in Palo Alto, California, and UMass Memorial Medical Center in Worcester, Massachusetts.

To be eligible, participants must have 36 or more large, chronic wound pairs — with a minimum of 20 square centimeters in size, one wound treated, the other left untreated — for at least six months.

This study has two main objectives. The first one is to evaluate the number of wounds that heal at least 50% from baseline, or the start of the study. This will be assessed by comparing wound pairs 24 weeks (nearly six months) after transplant.

The other main goal is pain reduction associated with changing wound dressings that are used to clean, cover, and protect the wound. This will be evaluated by the mean differences in Wong-Baker FACES scale scores between wound pairs at the end of the study.

“We thank the patients, families, and the clinical investigators at Stanford and UMass for participating in this study,” Seshadri said. “We look forward to sharing topline study results in the third quarter of 2022.”

Results of a Phase 1/2a study (NCT01263379), also conducted at Stanford University, found significant healing and pain reduction in large chronic wounds treated with EB-101 for up to six years.

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to EB-101. This designation is given to investigational therapies for serious or life-threatening diseases primarily affecting children and with fewer than 200,000 patients in the U.S. If EB-101 is approved, Abeona will get a voucher from the FDA that can be converted to obtain priority review of a subsequent treatment.