Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

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by Forest Ray PhD |

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Abeona Therapeutics has restarted patient enrollment in the VIITAL Phase 3 clinical trial of EB-101, a potential gene-corrected cell therapy for people with recessive dystrophic epidermolysis bullosa (RDEB).

Recruitment into the VIITAL trial (NCT04227106), conducted at Stanford Medicine in California, had been placed on hold in March so the company could redirect its resources to COVID-19 patients and implement measures to ensure the safety of study participants and staff. More information about enrollment, including contacts, can be found here.

The study intends to evaluate the safety and effectiveness of EB-101, with a primary goal of obtaining at least 50% healing at 12 weeks compared with the start of the treatment. Approximately 15 RDEB patients (age 6 and older) with chronic wounds will be enrolled.

“Reinitiating enrollment in the VIITAL study is important for patients with RDEB and brings us a step closer toward concluding the clinical development of EB-101,” João Siffert, MD, Abeona’s CEO, said in a press release. “The unprecedented COVID-19 global health crisis has had a broad impact across our industry, and we will continue to monitor the pandemic to ensure the safety of trial participants, healthcare professionals and our employees.”

Epidermolysis bullosa is a group of rare skin disorders characterized by fragile skin and blistering. Mutations in the COL7A1 gene, containing instructions for making a key skin integrity protein called type VII collagen, cause RDEB.

Treatment with EB-101 involves delivering a healthy COL7A1 gene to a patient’s own skin cells cultured in a lab dish. The modified cells are  transplanted back into the patient via a surgically-implanted keratinocyte sheet placed on the wound site.

Abeona presented two posters at the Society for Pediatric Dermatology (SPD) 45th Annual Meeting (held virtually), which further supported the therapeutic potential of EB-101 and highlighted the need for effective RDEB treatments.

The first poster, “EB-101 Treatment of Large, Chronic Wounds Is Associated with Durable Healing and Pain Reduction in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB),” reported results of a Phase 1/2a study (NCT01263379), also conducted at Stanford, which showed that EB-101 durably reduced patients’ wound burden.

EB-101 was given to seven adults with severe RDEB, who had a total of 38 chronic open wounds for at least 12 weeks, and were treated from 2013 to 2017.

Wound healing of 50% or more was associated with no pain at treated sites for up to five years post-treatment. By comparison, patients reported pain at 53% of wound sites prior to treatment.

The VIITAL study will further assess the link between having fewer wounds and pain relief following EB-101 treatment.

“The data presented at SPD showed that EB-101 treatment of large, chronic wounds resulted in considerable and durable reduction in wound burden,” said Siffert, in a separate press release.

The second poster, “The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB),” detailed the full burden of RDEB on patients and their families. The information came from a review of 65 studies that assessed the clinical, human, and economic burden of the disorder.

Key observations included that large and chronic wounds, often painful, comprise a major clinical burden. Also, patients frequently experience anxiety and depression. Parents of children with RDEB often choose not to have more children, and report lack of energy for anything besides caring for their child. In addition, 50% of U.S. families dealing with RDEB described the economic impact of the disease as “high” or “severe.”

“The second poster at SPD helps to characterize the disease burden and management of RDEB, providing an important reminder of the extraordinary toll RDEB takes on quality of life, and underscores the need for therapies that reduce wound burden and the associated humanistic and economic impact,” Siffert said.