RGN-137, an experimental therapy for wound healing in epidermolysis bullosa (EB), is expected to advance into a Phase 3 trial, backed by a joint venture created by RegeneRx licensee GtreeBNT and YuYang DNU.
The treatment was granted orphan drug designation by the U.S. Food and Drug Administration, and received FDA permission to start a Phase 3 efficacy study in the U.S. The clinical trial is expected to start in 2019, according to a RegeneRx news release.
The joint venture between the two companies, called Lenus Therapeutics, is a U.S. subsidiary of the drug’s licensee GtreeBNT. The latter will contribute all intellectual property rights and the development results, while YuYang will invest about $17.8 million in the program.
RGN-137 is a gel formulation to be applied directly to the skin. Its active ingredient is thymosin beta-4 (Tβ4), a lab-made copy of a small, natural protein produced by the body which has wound healing and anti-inflammatory properties.
The gene that provides instructions to make Tβ4 is one of the first to be activated when an injury occurs, suggesting that it contributes significantly to the healing process.
RGN-137 aims to offer a novel approach for wound healing, as it targets multiple cellular pathways that contribute to wound formation, including cell death, blood vessel growth, collagen deposition, and tissue inflammation.
According to RegeneRX, a Phase 1 trial, completed in 2004, showed no serious adverse events and no drug-related adverse events in healthy subjects topically administered (applied to the skin) with RGN-137.
Also, a Phase 2 placebo-controlled trial evaluating RGN-137 for accelerating wound healing in EB (NCT00311766) was completed in 2011. Results showed that daily treatment with 0.03% RGN-137 gel, up to 56 days, improved wound healing, with no serious adverse events reported.
Now, the joint venture is planning to conduct a small open-label study (NCT03578029) this year to attract a big pharmaceutical company in the drug, and then launch a Phase 3 trial in 2019. The open-label study is not yet recruiting.
The Phase 3 study planned for next year is expected to enroll approximately 200 subjects in different centers around the globe, including the U.S.
RGN-137 is also being tested for other indications, specifically in the treatment of late stage pressure ulcers (wounds that result from prolonged pressure on the skin), and venous stasis ulcers (wounds that results from malfunctioning venous valves causing increase pressure in veins, usually in the legs).