Abeona Reports Updated Data from Gene Therapy for EB

Rare Daily Staff

Abeona Therapeutics reported updated clinical data from the phase 1/2 trial for EB-101, the company’s gene-corrected skin graft cell therapy for patients suffering from recessive dystrophic epidermolysis bullosa, a rare and painful skin condition with life-threatening complications and shortened life expectancy.

People with recessive dystrophic epidermolysis bullosa lack functional type IIV collagen due to a mutation in COL7A1 gene. EB-101 is an autologous gene-corrected cell therapy that utilizes a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin onto the body.

The company said results demonstrate robust and durable clinical effects achieved throughout various timepoints post-administration. The results were presented during the American Society for Gene and Cell Therapy 21st Annual Meeting in Chicago.

“Currently there are no effective treatments available to reduce the severity of their symptoms,” said Carsten Thiel, CEO of Abeona. “The advancements made through this trial are clinically meaningful, showing significant and durable wound healing results and improved quality of life in these patients.”

The clinical trial included seven patients with 42 gene-corrected EB-101 grafts, with the first patient treated more than three years ago with lasting effects and closed wounds to date.

In the trial, EB-101 was administered to non-healing chronic wounds on each subject and assessed for wound healing at predefined time points. The trial met the primary endpoints for safety, tolerability and preliminary efficacy, where wound healing after EB-101 administration was compared to untreated wounds from a supportive natural history study that evaluated 128 patients with approximately 1,500 chronic and recurring RDEB wounds.

Secondary endpoints included expression of collagen C7 and restoration of anchoring fibrils at three- and six-months post-administration respectively. The wound healing effects of EB-101 were associated with meaningful reductions in pain and itch, as reported through patient recorded outcomes.

The data updated at the ASGCT conference included:

Wound healing (defined as greater than 50 percent closure after EB-101 administration) was observed in:

• 100 percent (42/42 treated wounds, n=7 subjects) at 3 months
• 90 percent (38/42 treated wounds, n=7 subjects) at 6 months
• 67 percent (24/36 treated wounds, n=6 subjects) at 12 months
• 88 percent (21/24 treated wounds, n=4 subjects) at 24 months
• 100% (6/6 treated wounds, n=1 subject) at 36 months

The company said it is working with the U.S. Food and Drug Administration to finalize the design of its pivotal phase 3 trial for the program and are defining the pathway forward for the study to begin later this year.

The EB-101 program has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations from the FDA and Orphan Drug Designation from the European Medicines Agency.

May 17, 2018
Photo: Carsten Thiel, CEO of Abeona