BridgeBio Pharma and Affiliate Phoenix Tissue Repair Announce First Patient Dosing In Phase 2 Trial of Protein Replacement Therapy for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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[{"type":"text","content":"BOSTON, Oct. 27, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) and affiliate Phoenix Tissue Repair today announced that the first patient has been dosed in a Phase 2 study of BBP-589 (also known as PTR-01), an intravenously-administered recombinant collagen 7 (rC7) protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a rare genetic disorder caused by mutations in the gene encoding collagen type VII (C7).\n RDEB is a devastating disease and is one of the most severe forms of epidermolysis bullosa, characterized by severe and painful skin blistering, as well as extreme fragility and scarring of mucous membranes throughout the body. There is currently no cure or effective treatment available for patients suffering from this disease. “BBP-589 is a potentially disease-modifying rC7 replacement therapy that we hope may improve the skin manifestations as well as the systemic symptoms these patients endure. The results observed in the Phase 1 study give us confidence in our approach, and we are excited to continue advancing this therapeutic candidate through the clinic,” said Sanuj K. Ravindran, M.D., executive chairman of Phoenix Tissue Repair. The Phase 2, open label study is designed to examine the effect of BBP-589 on wound healing and various systemic endpoints and to evaluate the long-term safety and tolerability of the drug candidate. The study plans to enroll six patients who will receive doses of BBP-589 intravenously in two regimens over 18 weeks, followed by 12 weeks of observation after completion of dosing to assess the durability of wound healing and other efficacy endpoints. To learn more about the BBP-589 Phase 2 clinical trial, please visit www.clinicaltrials.gov. About Dystrophic Epidermolysis Bullosa (DEB)DEB is a rare genetic disorder symptomatic from birth that is caused by mutations in the gene for a protein called collagen type VII (C7). The C7 protein is essential for the formation of anchoring fibrils, structures which connect the epidermis and dermis—the uppermost two layers of the skin. Patients with the recessive form of DEB (RDEB) tend to have particularly severe symptoms due to severe insufficiency of functional C7. Symptoms include extreme skin and mucosal fragility that present as recurrent, painful blistering and scarring of the skin, a...

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