BOSTON, April 30, 2019 /PRNewswire/ --Phoenix Tissue Repair, Inc., a biotechnology company focused on developing disease-modifying treatments, today announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation for PTR-01, an investigational protein replacement therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a rare genetic disorder associated with severe skin blistering and treatment is currently limited to palliative options.
The fast track process at the FDA is designed to facilitate the development and expedite the review of drugs to treat serious conditions with unmet medical needs. Under such designation, Phoenix Tissue Repair is eligible to engage in more frequent interactions with the FDA to discuss the development plan for PTR-01, including the design of proposed clinical trials and use of biomarkers. Fast track-designated drugs may also qualify for priority review, which can further expedite the FDA review process.
"Patients and their caregivers have been waiting far too long for treatments that go beyond palliative care for RDEB, and fast track designation is an important step forward in the development of PTR-01," said Dr. Neil Kirby, President and Chief Executive Officer of Phoenix Tissue Repair. "We continue to work diligently to advance what we believe could be the first systemic therapeutic agent that aims to target the root cause of the disease."
Phoenix Tissue Repair also announced it has opened enrollment of the second cohort in its ongoing Phase 1/2 trial in adult RDEB (PTR-01-001 trial). The PTR-01-001 trial is a randomized saline-controlled, single-blind, multiple ascending dose, dose-escalation and multi-center study. Patients will be enrolled into one of three cohorts. Cohorts 1, 2 and 3 will consist of 2, 4 and 8 patients respectively. During the treatment period a total of 3 doses of PTR-01 and 3 doses of saline control will be administered to all patients for a total of 6 doses in a cross-over design over a 10-week period. The primary objective of the trial is to evaluate the safety and tolerability of PTR-01 in RDEB patients. Additionally, the trial will assess biologic activity through skin biopsy evaluation of C7 and the presence of anchoring fibrils to potentially provide proof of concept. Wound healing and clinically meaningful patient reported outcomes will also be evaluated. To learn more about the PTR-01 Phase 1/2 clinical trial, please visit www.clinicaltrials.gov and search the identifier NCT03752905.
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). C7 is essential for the formation of anchoring fibrils, structures which connect the uppermost two layers of the skin, the epidermis and dermis. 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 presents as recurrent, painful blistering and scarring of the skin; ulcerations of the mouth, tongue, and dental caries. In addition to the cutaneous and oral symptoms, severe forms are associated with erosions and scarring of mucous membranes of the eye, esophagus, genitals, and anus. Joint contractures, mutilating deformities of hands and feet, malnutrition, growth retardation, recurrent infections, and a significantly increased risk for squamous cell carcinoma are also common. There are currently no approved disease-modifying therapies for any form of DEB, and the standard of care focuses on wound and pain management.
Phoenix Tissue Repair is advancing an investigational therapy known as PTR-01, a protein replacement therapy which uses a recombinant collagen type VII (rC7) for the treatment of RDEB. PTR-01 is designed to be systemically available through intravenous delivery. Phoenix Tissue Repair acquired worldwide rights to PTR-01 in 2017. Preclinical studies of PTR-01 have demonstrated C7 staining in basement membranes with de novo anchoring fibril formation and improved survival in models of RDEB.
PTR-01 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency.
About Phoenix Tissue Repair and BridgeBio
Phoenix Tissue Repair is a Boston-based company that is part of the BridgeBio family. BridgeBio is a clinical-stage biotech company developing novel, genetically targeted therapies to improve the lives of patients. Founded in 2015 by a team of industry veterans, the company has built a robust portfolio of transformative assets, each housed in its own subsidiary, ranging from pre-clinical to late stage development in multiple therapeutic areas including dermatology, cardiology, oncology and endocrinology. The company's focus on scientific excellence and rapid execution aims to translate today's discoveries into tomorrow's medicines.
This press release contains forward-looking statements. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements relating to expectations, plans, and prospects regarding Phoenix Tissue Repair's clinical development plan, clinical trial results, timing and completion of clinical trials and ability to take advantage of expedited FDA review for PTR-01. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, Phoenix Tissue Repair's ability to advance PTR-01 in clinical development in accordance with its plans, the results from any clinical trials and nonclinical studies of PTR-01 and the nature of Phoenix Tissue Repair's interactions with regulatory authorities. Moreover, Phoenix Tissue Repair operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of Phoenix Tissue Repair's management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. All forward-looking statements in this press release are based on information available to Phoenix Tissue Repair as of the date hereof, and Phoenix Tissue Repair disclaims any obligation to update these forward-looking statements except as required by law.
Investor Contact: LeYi Wang, PhD Vice President, Business Development (650) 391-9740 [email protected]