RARE Daily

Abeona Raises $35M, Reports Positive Topline Results in Pivotal Study of EB-101 for RDEB Patients

November 3, 2022

Rare Daily Staff

Abeona Therapeutics said it raised $35 million in a private financing and at the same time reported positive topline data from its pivotal phase 3 VIITAL study assessing the safety and efficacy of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa.

The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds.

“We are very pleased with the topline results from our pivotal VIITAL study, which reinforce the strong value proposition of EB-101 as a potential one-time therapy to both significantly improve wound healing and reduce pain for the most disabling, challenging to treat wounds in patients with RDEB,” said Vish Seshadri, CEO at Abeona. “The VIITAL study is differentiated from any other pivotal study in RDEB by the co-primary endpoint measuring patient-reported pain. We believe the significant result in this endpoint supports EB-101’s potential for improving the daily life of RDEB patients.”

Recessive dystrophic epidermolysis bullosa (RDEB) is 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. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

EB-101 is an autologous, engineered cell therapy currently being developed for the treatment of RDEB. The EB-101 VIITAL study is a randomized clinical trial with target enrollment of at least 10 to 15 RDEB patients with approximately 36 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the 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.

The pivotal phase 3 VIITAL study evaluated the efficacy, safety, and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB. The large chronic wounds randomized and treated in VIITAL measured greater than 20 cm2 of surface area and had remained open for a minimum of six months and a maximum of 21 years (mean 6.2 years). The co-primary endpoints of the study were: 1) the proportion of RDEB wound sites with greater than or equal to 50 percent healing from baseline, comparing randomized treated with matched untreated (control) wound sites at the six-month timepoint, as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between randomized treated and matched untreated (control) wounds at the six-month timepoint. The study allowed for wounds not included in the randomized primary efficacy analysis to receive EB-101 treatment (n=14 non-randomized wounds).

EB-101 significantly improved wound healing vs. control at six months. The co-primary endpoint measuring greater than 50 percent wound healing and other endpoints measuring greater than 75 percent and complete wound healing at six months were all met.

Photo: Vish Seshadri, CEO at Abeona

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