RegeneRx Biopharmaceuticals, Inc., a clinical-stage drug development company focused on tissue protection, repair and regeneration, today announced that its licensee for RGN-137, GtreeBNT Co., Ltd., received a positive response from the U.S. FDA for its Phase 3 clinical trial design for RGN-137 to treat epidermolysis bullosa (“EB”). RGN-137 is a dermal wound healing gel that incorporates Thymosin beta 4 (“Tβ4”) as the active pharmaceutical ingredient. GtreeBNT is planning to enter into the Phase 3 trial in the U.S. during the third quarter of 2017.
EB is a rare inherited skin disease that is caused by genetic defects in anchoring between the epidermis and dermis, resulting in skin fragility. EB 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. RGN-137 previously received an orphan designation for EB from the U.S. FDA.
It is estimated that the number of patients with all types of EB is approximately 25,000 to 30,000 in the U.S. The global market size of EB treatment is estimated at approximately $1.0 billion annually. Since there is currently no approved prescription treatment for EB, RGN-137 is targeting a very significant unmet medical need. The current treatments available for EB primarily focus on minimizing and relieving certain of its symptoms and controlling infection. RGN-137 has the potential to reduce pain and suffering in EB patients by accelerating wound healing, reducing inflammation, and upregulating the production of laminin-5, a protein that affects tissue integrity throughout the body that is defective in patients with EB. RGN-137 has also been shown to reduce scarring in animal models, another problem affecting EB patients.
“Since we received an official positive response from the FDA for our clinical development plan for a Phase 3 EB trial, our clinical development plan for the treatment of EB with RGN-137 has been established. GtreeBNT now has the potential to move forward for an NDA submission. Furthermore, this agreement with the FDA has continued our momentum in developing new drugs to treat rare diseases,” stated Won S. Yang, president and chief executive officer of GtreeBNT.
The Phase 3 trial will be a randomized, multi-center, double-blind, placebo-controlled study to evaluate the efficacy and safety of RGN-137 topically administered to approximately 200 EB patients at clinical sites throughout the U.S. GtreeBNT will be sponsoring and funding the clinical trial.