Phoenix Tissue Repair, Inc., a biotechnology company focused on developing transformational disease-modifying treatments for dystrophic epidermolysis bullosa (DEB), today announced that the first patient has been dosed in the Phase 1/2, first in-human trial of PTR-01 (BridgeBio Pharma designation BBP-589), a protein replacement therapy for recessive DEB (RDEB). DEB and RDEB are rare genetic multisystem disorders associated with severe skin blistering, and treatment is currently limited to palliative symptom management.

"Dosing the first patient in the clinic is an important milestone for the development of PTR-01 as a potential treatment for RDEB," said Dr. Neil Kirby, President and Chief Executive Officer of Phoenix Tissue Repair. "The community of RDEB patients, families and physicians urgently need effective treatments for this devastating disease. With an approach that aims to target the root cause of the disease, we are optimistic that PTR-01 could provide hope to patients and their families."

DEB is a rare genetic disorder symptomatic from birth that is caused by mutations in the gene encoding collagen type VII protein (C7). Symptoms include extreme skin and mucosal fragility and can also include erosions and scarring of other mucous membranes throughout the body. Severe comorbidities are common, and patients require intensive and constant care. DEB, and RDEB in particular, is associated with a considerable reduction in quality-of-life and life span. There are currently no approved disease-modifying therapies for any form of DEB.

The PTR-01-001 Phase 1/2 trial will enroll 14 patients who will each be dosed over a 10-week period. The primary objective of the trial is to evaluate the safety, tolerability and pharmacokinetics of PTR-01 in RDEB patients. The trial will also assess various secondary endpoints.

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