The U.S. Food and Drug Administration granted Breakthrough Therapy Designation status to the Abeona Therapeutics Inc.’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa.

EB-101 is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa.

The designation enables collaborative discussions with senior FDA personnel, priority review and an expedited approval process for drug candidates where preliminary clinical trials indicate that a therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases.

“EB-101 is an autologous gene-corrected therapeutic approach that utilizes a patient’s own cells, genetically engineering them to produce the correct version of collagen which helps hold skin onto the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” says Timothy J. Miller, Ph.D., Abeona’s President and CEO, in a news release. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal Phase 3 trial as we advance EB-101 for patients with this debilitating disease.”

The Breakthrough Therapy designation is based on data from the Phase 1/2 EB-101 clinical trial, which demonstrated significant wound healing (greater than 50 percent healed) in treated wounds for over two years. The criteria for this particular designation requires preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. 

This is the first Breakthrough Therapy designation for Abeona since the FDA initiated the program in 2013.

The Company will work with the FDA on the final Phase 3 clinical trial design, planned to commence early 2018, and will provide an update on the program in the coming months. 

The EB-101 program has been granted Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Medicines Agency (EMA).

About EB-101 Phase 1/2 Clinical Trial

In the recent Phase 1/2 clinical 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 safety and efficacy, where wound healing after EB-101 administration was compared to control untreated wounds from a supporting natural history study that evaluated 128 patients. Secondary endpoints included expression of collagen C7 and restoration of anchoring fibrils at three and six-months post-administration. Clinical data were presented at the Society of Investigative Dermatology (SID) conference by Stanford collaborators, and demonstrated that EB-101 treated wounds were significantly healed >50 percent for more than two years post-administration. The data included:

Wound healing, defined as >50 percent closure after EB-101 administration, was observed in:

   ---   100 percent (36/36 treated wounds, n=6 subjects) at 3 months;

   ---   89 percent (32/36 treated wounds, n=6 subjects) at 6 months;

   ---   83 percent (20/24 treated wounds, n=4 subjects) at 12 months;

   ---   88 percent (21/24 treated wounds, n=4 subjects) at 24 months;

   ---   100 percent (6/6 treated wounds, n=1 subject) at 36 months post-administration.

Collagen VII (C7) expression: C7 and morphologically normal NC2 reactive anchoring fibrils were observed in EB-101 treated wounds up to two years post-administration.

Importantly, data from a supportive natural history study of 1,436 wounds from 128 patients with RDEB, established by Stanford and EBCare Registry, were also presented at the conference and to the FDA. Notably, 13 RDEB patients with a total of 15 chronic wounds were treated with an allograft product, including Apligraf® and Dermagraft®. Of these wounds treated with allografts, only 7 percent (1/15 treated wounds) remained healed after 12 weeks, and 0 percent (0/15 treated wounds) remained healed after 24 weeks. This is a meaningful finding of the natural history study, as there are no approved therapies for RDEB patients that demonstrate significant wound closure after two months post-application.