EB-101 Cell Therapy Continues to Show Efficacy in Recessive Dystrophic Epidermolysis Bullosa


The phase 3 VITAL study, which is currently enrolling, will evaluate the investigational cell therapy agent further.

The autologous, gene-corrected cell therapy EB-101 is efficacious in long-term wound healing and relieving pain in patients with recessive dystrophic epidermolysis bullosa (RDEB), according to updated results from a Phase 1/2a clinical trial (NCT01263379) announced by Abeona Therapeutics.1

At 3 years post-treatment, 69% (n = 18/26) of treated wounds had wound healing of at least 50% from baseline; at 4 years 93% (n = 14/15) met this criterion, at 5 years, 80% (n = 12/15), and at 6 years, 80% (n = 4/5).

“RDEB is a debilitating and life-threatening rare genetic disorder with high rates of morbidity and mortality, without an approved treatment option," said Vishwas Seshadri, PhD, MBA, head of research & clinical development, Abeona, in a statement.1 "It is important that potential new treatments can durably address large, chronic wounds, which are the most severe wounds that cause substantial pain in patients with RDEB.” 

The phase 1/2a trial enrolled participants with RDEM that were at least 18 years of age, had 2 COL7A1 genetic mutations and chronic open wounds of at least 20 cm2 for at least 12 weeks. Researchers cultured autologous keratinocytes from biopsies of intact skin and transduced them with retrovirus containing full-length COL7A1 to form the gene-corrected epidermal sheets of EB-101 measuring 25 cm2. The investigational agent is designed to enable normal Type VII collagen expression and facilitate wound healing. EB-101 was then transplanted onto 38 chronic wound sites in 7 participants between 2013 and 2017. 

READ MORE: Phase 3 B-VEC Data on Horizon for DEB

Investigators assessed wound healing and pain assessment during study visits and conducted a follow-up survey 3 to 6 years after treatment that collected patient-reported outcomes such as change in pain from baseline on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse). The survey revealed that 76% of treated wounds with at least 50% healing were associated with improved pain scores, with 53% associated with “much or very much improved” pain scores.

New data reported included investigator assessment of presence of pain in treated wounds up to 6 years after EB-101 treatment. Pain was absent in all treated wounds at 3 years (n=26), 4 years (n=15), 5 years (n=15) and 6 years (n=5) of follow up, compared with 53% of treated wounds with pain at baseline (n=20/38).

EB-101 is being evaluated further in the currently recruiting phase 3, open-label VITAL clinical trial (NCT04227106). The study aims to enroll 10 to 15 participants with RDEB and to treat around 35 large, chronic wound sites in total. The study’s primary end points will be the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 and pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24. Investigators will also collect patient-reported outcomes and safety data.2

“The updated Phase 1/2a results showed safety and durable efficacy follow up, with EB-101 treated wounds continuing to show a considerable reduction in both wound burden and associated long-term pain for up to six years. We are excited about the data and look forward to further investigating EB-101’s potential to provide durable benefit in our ongoing pivotal Phase 3 VIITAL study,” Seshadri added.1

1. Abeona Therapeutics announces updated EB-101 Phase 1/2a clinical results in recessive dystrophic epidermolysis bullosa at the society for Pediatric Dermatology 46th Annual Meeting. News release. Abeona Therapeutics. July 7, 2021. https://www.globenewswire.com/news-release/2021/07/07/2258859/0/en/Abeona-Therapeutics-Announces-Updated-EB-101-Phase-1-2a-Clinical-Results-in-Recessive-Dystrophic-Epidermolysis-Bullosa-at-the-Society-for-Pediatric-Dermatology-46th-Annual-Meeting.html
2. Phase 3, open-label clinical trial of EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). NIH US National Library of Medicine. January 27, 2021. https://clinicaltrials.gov/ct2/show/NCT04227106
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