PTR-01 / BridgeBio, Ligand - LARVOL DELTA

Improving the therapeutic potential of intravenous recombinant collagen VII as a protein replacement therapy for recessive dystrophic epidermolysis bullosa (SID 2025) - "However, the reduced affinity for DEJ ligands, lower thermal stability, and premature NC2 maturation likely impaired the ability of PTR-01 to assemble into AFs. We have since identified CHO clones that express only wild-type C7 and plan to develop this as an improved alternative to PTR-01."

Late-breaking abstract

Exploring the Financial Burden Associated with the Management of Dystrophic Epidermolysis Bullosa (DEB): A Systematic Literature Review (SLR) with Database Review of Future Disease-Modifying Therapies (DMTs) (ISPOR 2024) - "Nine clinical trials research 7 potential DMTs: advanced/gene therapies (EB-101, B-VEC, ALLO-ASC-SHEET, FCX-007), protein-replacement therapies (PTR-01, PTR-01-002), and a proteinase inhibitor (Polyphenon E). Previously, as there were no DMT options for patients with DEB treatment was symptomatic only. Future innovative DMTs should aim to prevent skin wounds, thereby decreasing the need for hospitalization and outpatient visits."

Review • Dermatology • Gene Therapies

Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) (clinicaltrials.gov) - P2b | N=2 | Completed | Sponsor: Phoenix Tissue Repair, Inc. | Enrolling by invitation ➔ Completed | Trial completion date: Nov 2022 ➔ Aug 2022 | Trial primary completion date: Nov 2022 ➔ Jun 2022

Trial completion • Trial completion date • Trial primary completion date • CRP

Extension Study to PTR-01-002 (clinicaltrials.gov) - P2b | N=2 | Enrolling by invitation | Sponsor: Phoenix Tissue Repair, Inc. | Not yet recruiting ➔ Enrolling by invitation | Trial primary completion date: Jun 2022 ➔ Nov 2022

Enrollment open • Trial primary completion date • CRP

Extension Study to PTR-01-002 (clinicaltrials.gov) - P2b; N=2; Not yet recruiting; Sponsor: Phoenix Tissue Repair, Inc.

Clinical • New P2b trial • CRP

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - P2; N=6; Completed; Sponsor: Phoenix Tissue Repair, Inc.; Active, not recruiting ➔ Completed; Trial primary completion date: May 2021 ➔ Aug 2021

Clinical • Trial completion • Trial primary completion date • CRP

[VIRTUAL] A phase 1/2 trial of PTR-01, a collagen 7 (C7) protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB) (SID 2021) - "Mild/moderate infusion-associated reactions occurred in four patients (2 each in Cohorts 3 and 4) and were managed with standard of care treatments (diphenhydramine, acetaminophen/ibuprofen, glucocorticoids). In this short-term study, PTR-01 has shown encouraging findings warranting further clinical trials. A longer Phase 2 study to assess efficacy is now enrolling."

Clinical • P1/2 data

PTR-01-001: A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P1/2; N=12; Completed; Sponsor: Phoenix Tissue Repair, Inc.; Active, not recruiting ➔ Completed

Clinical • Trial completion

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - P2; N=6; Active, not recruiting; Sponsor: Phoenix Tissue Repair, Inc.; Recruiting ➔ Active, not recruiting; Trial completion date: Dec 2021 ➔ Aug 2021

Clinical • Enrollment closed • Trial completion date • CRP

A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - P2; N=6; Recruiting; Sponsor: Phoenix Tissue Repair, Inc.

Clinical • New P2 trial • CRP

[VIRTUAL] PTR01--CF Patient Registry Data & Projects (NACFC 2020) - "Session Description The registry team members will be talking about the recent trends in the registry data, new features in CFSmartReports and other ongoing and accomplished since the last NACFC registry related projects."

Clinical

PTR-01-001: A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P1/2; N=12; Active, not recruiting; Sponsor: Phoenix Tissue Repair, Inc.; Recruiting ➔ Active, not recruiting; Trial primary completion date: Jul 2020 ➔ Nov 2020

Clinical • Enrollment closed • Trial primary completion date