BridgeBio Partnered Protein Replacement Therapy Shows Promising Action In Painful Skin Blistering Disorder

BridgeBio Pharma Inc BBIO and affiliate company Phoenix Tissue Repair announced data from the Phase 2 trial of PTR-01 in recessive dystrophic epidermolysis bullosa (RDEB).
PTR-01 is an intravenously-administered recombinant collagen 7 (rC7) protein replacement therapy.
RDEB is one of the most severe forms of epidermolysis bullosa, characterized by severe and painful skin blistering.
The data exhibited that PTR-01 was well tolerated when given once per week for 4 weeks and then every other week for 14 weeks.
In addition, treatment with PTR-01 led to rapid, consistent, and durable wound healing, as observed in the reduction of wound surface area and clinician-reported assessments.
Specifically, over 80% of target wounds (21/26) demonstrated a 50% or greater reduction in wound surface area at the end of treatment (day 120) compared to baseline.
This response was observed in a breadth of wound types.
Notably, all patients who completed the study (N=5) reported a decrease in pain over treatment with PTR-01.
There was a 36% mean reduction in total pain from the end of the study compared to baseline.
Phoenix Tissue Repair has initiated a Phase 2 extension study.
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