RegeneRx Biopharmaceuticals announced in a press release that GtreeBNT Co., Ltd., has received a positive response from the FDA for a phase 3 clinical trial design for RGN-137 to treat epidermolysis bullosa.
RGN-137 has Thymosin beta 4 as an inactive pharmaceutical ingredient in a dermal wound healing gel, according to RegeneRx, a clinical-stage drug development company.
Epidermolysis bullosa is a rare inherited skin disease, in which patients develop recurrent blisters and scars in the skin throughout the body beginning at birth, which can lead to serious infections, severe pain and sometimes death, according to the release.
The FDA had previously granted RGN-137 an orphan designation, according to RegeneRx.
There is no approved prescription treatment for EB, which is estimated to affect 25,000 to 30,000 people in the United States, according to the release.
While current treatment focuses on minimizing and relieving certain symptoms and controlling infection, RGN-137 has the potential to accelerate wound healing, reduce inflammation and upregulate the production of laminin-5, a protein that affects tissue integrity that is defective in patients with epidermolysis bullosa. Animal models have shown that RGN0137 has been effective in reducing scarring, according to the release.
The phase 3 trial is planned as a randomized, multi-center, double-blind, placebo-controlled study of the efficacy and safety of RGN-137, which will be topically administered to approximately 200 patients with epidermolysis bullosa at clinical sites throughout the US, according to the release. It is expected to be begin in the third quarter of 2017.