Rare Skin Disease Update: Abeona's EB-101 Performs Well in a Phase 3 study of RDEB

There is currently no approved treatment for recessive dystrophic epidermolysis bullosa, a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life.

Abeona Therapeutics Inc.’s investigational EB-101 performed well in a Phase 3 study of recessive dystrophic epidermolysis bullosa (RDEB).

Both co-primary endpoints were met, with the majority (81.4%) of randomized EB-101–treated wounds demonstrating ≥50% healing compared with 16.3% of untreated control wounds, and a significantly greater reduction in pain severity observed in randomized EB-101−treated wounds (3.07 mean pain reduction from baseline) compared with untreated control wounds (0.90 mean pain reduction from baseline) at six months, according to additional results from the Phase 3 VIITAL study which were presented at the International Societies for Investigative Dermatology (ISID) Meeting in Tokyo, Japan.

At earlier time points, meaning weeks 6 and 12, the percentage of wounds with ≥50%, ≥75%, and complete healing, as well as pain reduction, was greater for randomized EB-101–treated wounds and achieved statistical significance over untreated control wounds, the study showed.

In addition to significantly reducing pain, patient-reported outcomes related to itch and blistering showed significantly greater improvement with EB-101 treatment. Caregiver-reported outcomes related to wound care and overall impression of wound pain showed consistent trends for improvement.

Moreover, EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience.

“Following the positive topline VIITAL study results reported in November 2022, the additional data reported at ISID further highlights the value proposition of EB-101 by demonstrating improved wound healing and pain reduction at 6, 12 and 24 weeks compared to control wounds following a one-time application of EB-101,” says Vish Seshadri, Chief Executive Officer of Abeona, in a news release.  “Furthermore, EB-101 demonstrated improvement in patient-reported and caregiver-reported outcomes for itch severity and blistering. In totality, these results highlight the potential for EB-101 to meaningfully improve quality of life of RDEB patients.”

About EB-101

EB-101 is an autologous, engineered cell therapy currently being developed for the treatment of RDEB.  Treatment with EB-101 involves using gene transfer to deliver the functional COL7A1 gene into a patient’s own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing. EB-101 has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA.