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Phoenix Tissue Repair passes US FDA fast track designation for PTR-01to treat dystrophic epidermolysis bullosa.

M2 EQUITYBITES-May 1, 2019-Phoenix Tissue Repair passes US FDA fast track designation for PTR-01to treat dystrophic epidermolysis bullosa


Biotechnology company Phoenix Tissue Repair Inc revealed on Tuesday that it has received US Food and Drug Administration (FDA) fast track designation to facilitate the development and expedite the review of PTR-01 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic disorder associated with severe skin blistering and treatment is currently limited to palliative options.

According to the company, it is advancing PTR-01, an investigational protein replacement therapy that uses a recombinant collagen type VII (rC7) for the treatment of RDEB. PTR-01 is designed to be systemically available through intravenous delivery.

Under the company's preclinical studies, PTR-01 demonstrated C7 staining in basement membranes with de novo anchoring fibril formation and improved survival in models of RDEB.

In conjunction, the company has opened enrolment of the second cohort under its ongoing Phase 1/2 trial in adult RDEB (PTR-01-001 trial), a randomised saline-controlled, single-blind, multiple ascending dose, dose-escalation and multi-centre study. Patients will be enrolled into one of three cohorts. During the treatment period a total of three doses of PTR-01 and 3 doses of saline control will be administered to all patients for a total of six doses in a cross-over design over a ten week period.

The company said the primary objective of the trial is to evaluate the safety and tolerability of PTR-01 in RDEB patients, assess biologic activity through skin biopsy evaluation of C7 and the presence of anchoring fibrils to potentially provide proof of concept as well as evaluate the wound healing and patient reported outcomes.

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Publication:M2 EquityBites (EQB)
Date:May 1, 2019
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