- Following a green light from the Food and Drug Administration in a Type-C meeting, rare diseases company Abeona Therapeutics is ready to kick off its Phase 3 trial of EB-101 gene therapy in recessive dystrophic epidermolysis bullosa.
- Abeona is discussing the design of the pivotal trial in children with so-called "butterfly disease" with the FDA and plans to begin the study in early 2018.
- The ongoing Phase 1/2 proof-of concept study of Abeona's autologous ex vivo gene therapy has shown that EB-101 treatment can lead to significant healing of wounds (>50%) up to three years post-treatment. Standard skin grafts typically last only a few months.
EB-101 is an autologous ex vivo gene therapy, designed to treat the rare disorder recessive dystrophic epidermolysis bullosa, the most severe form of the disease. Keratinocytes (skin cells) are taken from patients, the genetic defect is corrected by inserting an unmutated version of the gene COL7A1, and the cells returned as gene-corrected skin grafts to aid wound closure.
Analysts at Maxim Group were upbeat about the new step for EB-101, which could potentially make it the first therapeutic to market specifically for this life-changing and even life-threatening disorder. "In gene therapy, proof of concept can come with N = 'not that many'. With POC data in hand and guidance from the FDA, Abeona has a path forward for a pivotal program," wrote Maxim analyst Jason Kolbert.
While EB-101 is a treatment for a rare disease, which affects between one and three cases per million live births in the U.S., Maury Raycroft, equity analyst at Jefferies, estimates peak sales of around $107 million depending on pricing and treatment paradigms.
There are a number of other companies working in epidermolysis bullosa. Last month, Castle Creek Pharma enrolled the first patient in its Phase 2/3 DELIVERS clinical trial of CCP-020 (diacerin 1% ointment) in epidermolysis bullosa simplex (EBS), the most common version of epidermolysis bullosa.
InMed Pharmaceuticals is carrying out preclinical and formulation studies for its lead product, INM-750, with an aim to begin Phase 1 in 2018. Fibrocell Science is specifically targeting recessive dystrophic epidermolysis bullosa, and taking a similar approach with local treatment using corrected skin cells. FK-007, in development in collaboration with Intrexon Corporation, is in Phase 1/2, completing dosing of the first cohort in June 2017.