Science
Collagen is an essential component of human skin and connective tissues. More than two-dozen types of collagen exist in the body, each one playing its own distinct and important role. Collagen type VII functions as nature’s Velcro, forming anchoring fibrils that secure the skin’s protective outer layer, the epidermis, to the thick under-layer known as the dermis. Mutations in the gene that encodes collagen type VII can disrupt its ability to anchor the epidermis, resulting in a genetic disease called Dystrophic Epidermolysis Bullosa (DEB). The disease can also affect the lining of the mouth, esophagus, eye, anus and vagina.
Two forms of the disease exist, based on how it is inherited. In the United States, the recessive and dominant forms of DEB each have a prevalence of one affected patient per million people. There is currently no cure or effective treatment available to patients.
Patients
In patients with recessive dystrophic epidermolysis bullosa (RDEB), the epidermis can separate from the dermis. Even minor friction or trauma can cause debilitating blistering, tearing and scarring of the skin, along with severe pain and itching. The internal skin lining the mouth and throat are affected, disrupting patients’ ability to eat. Many die prematurely from aggressive squamous cell carcinoma, a common complication of DEB. These patients have no treatment options beyond daily wound care, pain management and protective bandaging. The epidermolysis bullosa patient community has done extraordinary work to spread awareness of the unmet medical need that patients face.
Phoenix Tissue Repair is working to create a first-ever systemic treatment for RDEB by replacing the collagen proteins (C7) at the root of the disease. In November 2018, we initiated a Phase 1/2 clinical trial of PTR-01, a recombinant version of the human protein collagen type VII (rC7) in adult patients with recessive dystrophic epidermolysis bullosa (RDEB). In this trial, we are evaluating safety and tolerability of PTR-01, as well as clinical proof of concept in RDEB patient population. For more information about this trial, visit the posting on ClinicalTrials.gov, or contact us at info@phoenixtissuerepair.com
About PTR-01
Phoenix Tissue Repair is advancing an investigational therapy known as PTR-01, a systemic recombinant collagen type VII (rC7) for the treatment of Recessive Dystrophic Epidermolysis Bullosa. rC7 is a potentially disease-modifying drug that is delivered intravenously to patients, replacing defective collagen type VII with healthy collagen at the sites where it is needed.
Phoenix Tissue Repair acquired worldwide rights to PTR-01 from Shire Plc in 2017 and has initiated its first clinical trial (PTR-01-001). Preclinical studies of PTR-01 have shown that it selectively anchors in the skin and other tissues affected by an absence of collagen type VII. In four animal models of the disease, intravenous injections of PTR-01 promoted healing of DEB wounds.
These experiments have shown improvements in tissue structure, disease presentation and survival, indicating a restoration of natural skin architecture. For more information, please contact us at info@phoenixtissuerepair.com
News
PHOENIX TISSUE REPAIR IS A MEMBER OF THE BRIDGEBIO FAMILY
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. Founded in 2015, BridgeBio’s mission is 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 15 development programs includes product candidates ranging from early discovery to late-stage development.
