BridgeBio Pharma’s Phoenix Tissue Repair to Highlight Interim Phase 1/2 Study Data in a Presentation at the Society for Pediatric Dermatology’s 45th Annual Meeting

Rhea-AI Summary

BridgeBio Pharma's affiliate, Phoenix Tissue Repair, announced the presentation of interim safety and tolerability data from a Phase 1/2 trial for PTR-01, a recombinant collagen 7 therapy aimed at treating recessive dystrophic epidermolysis bullosa (RDEB). The results will be shared virtually during the Society for Pediatric Dermatology’s Annual Meeting from July 10-12, 2020. PTR-01 has previously shown promising preclinical results, with Orphan Drug Designation granted by the FDA and EMA. Currently, no approved therapies exist for DEB, and the standard care is limited to wound management.

Positive

• PTR-01 has received Orphan Drug Designation from both the FDA and EMA.
• Preclinical studies indicate successful de novo anchoring fibril formation and improved survival in RDEB models.
• Interim data presentation indicates ongoing safety and tolerability in early clinical cohorts.

Negative

• None.

BOSTON, July 10, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate Phoenix Tissue Repair (PTR) today announced an upcoming presentation of interim data from an ongoing Phase 1/2 study of PTR-01 (BBP-589), an intravenously-administered recombinant collagen 7 protein replacement therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The presentation will be made during the Society for Pediatric Dermatology’s (SPD) 45th Annual Meeting, to be held virtually July 10-12, 2020.

The poster presentation, which includes safety and tolerability data observed so far in patients enrolled in cohorts 1-3, will be delivered by Anna L. Bruckner, MD, associate professor of dermatology and pediatrics at University of Colorado School of Medicine. The pre-recorded presentation will be available online to meeting registrants until December 31, 2020. The poster will also be available on the Phoenix Tissue Repair website.

Details for the presentation are below:

Title: Interim update from a Phase 1/2 trial examining the safety and tolerability of PTR-01, a collagen 7 protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa
Presenter: Anna L. Bruckner, MD

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). The C7 protein is essential for the formation of anchoring fibrils, structures which connect the epidermis and dermis—the uppermost two layers of the skin. 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 present as recurrent, painful blistering and scarring of the skin, as well as 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.  

About Phoenix Tissue Repair and PTR-01
Phoenix Tissue Repair is a Boston-based company that is an affiliate of BridgeBio Pharma, and is focused on advancing a novel systemic treatment for recessive dystrophic epidermolysis bullosa (RDEB).
PTR-01 is an investigational 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 BridgeBio Pharma, Inc.
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

Forward-Looking Statements
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 Relations Contact:
Mike Mangone, Ph.D.
Vice President, Business Development & Corporate Strategy
857-449-0970
info@phoenixtissuerepair.com

Media Relations Contact:
Carolyn Hawley
Canale Communications
(619) 849-5382
carolyn@canalecomm.com

FAQ

What is PTR-01 and how does it work?

PTR-01 is an investigational recombinant collagen 7 protein replacement therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB) by replacing the deficient protein systemically through intravenous delivery.

When and where will the interim data for PTR-01 be presented?

The interim data will be presented during the Society for Pediatric Dermatology’s 45th Annual Meeting, held virtually from July 10-12, 2020.

What are the key findings from the PTR-01 Phase 1/2 trial?

The key findings include interim safety and tolerability data from the first three cohorts of patients, indicating the treatment is being well-tolerated.

What is the significance of the Orphan Drug Designation for PTR-01?

The Orphan Drug Designation provides PTR-01 with benefits such as tax credits for clinical trials, potential market exclusivity, and eligibility for grants to assist in the development of the therapy.

What are the implications of the trial results for patients with RDEB?

If successful, PTR-01 could provide a much-needed disease-modifying therapy for RDEB patients, who currently have no approved treatments other than wound and pain management.