We are delighted to share some positive news related to two new EB treatments which are currently being developed.

BridgeBio announces positive results from the Phase II trial of PTR-01, for the treatment of Reccesive Dystrophic Epidermolysis Bullosa (RDEB)

BridgeBio Pharma and its partner company, Phoenix Tissue Repair, announced positive results from their Phase II clinical trial of PTR-01, which treats Recessive Dystrophic Epidermolysis Bullosa (RDEB). PTR-01 is a protein replacement therapy, which is administered using a syringe and IV drip. The treatment is designed to replace the faulty Collagen VII protein in RDEB, potentially healing the chronic wounds associated with the condition.

The aim of the Phase II clinical trial was to evaluate how effective PTR-01 is for wound healing, as well as the safety and tolerability of the treatment. Executive chairman of Phoenix Tissue Repair, Sanuj K. Ravindran, M.D. stated “Our data shows that treatment with PTR-01 led to rapid, consistent, and durable wound healing”.

The data BridgeBio shared at the Society for Investigative Dermatology (SID) Annual Meeting 2022 showed that PTR-01 was well-tolerated over an eighteen-week treatment period in patients with RDEB. The patients who were treated with PTR-01 experienced a 50% or greater reduction of wound surface area in 80% of the target wounds by the end of the treatment, compared to baseline data.

The drug is showing the ability to treat many different wound types and sizes. 86% of recurrent wounds and 75% of chronic wounds showed positive results after being treated with PTR-01. 89% of large wounds and 77% of small wounds experienced the 50% or greater reduction of wound surface area.

In addition to effective wound healing, all five patients who completed the study reported a decrease in pain over the course of treatment with PTR-01.

This successful result has prompted Phoenix Tissue Repair to initiate a Phase II extension study, to ensure these positive effects are seen over a longer period.

For more information on PTR-01:https://bridgebio.com/news/bridgebio-pharma-affiliate-phoenix-tissue-repair-announces-positive-results-from-phase-2-trial-of-ptr-01-a-protein-replacement-therapy-for-the-treatment-of-recessive-dystrophic-epidermolysis-bullosa/

 

Tetra Bio-Pharma granted Orphan Drug Designation by EMA for a cannabinoid cream, PPP004, to treat EB

Tetra Bio-Pharma’s cannabinoid cream ‘PPP004’ (containing THC-CBD) has been granted orphan drug designation by the European Medicines Agency (EMA) to treat Epidermolysis Bullosa. Orphan Drug Designation (ODD) is granted to medications which aim to treat, prevent, and diagnose chronic or life-threatening conditions that affect less than 5 in 10,000 people in the European Union.

Tetra Bio-Pharma is a leader in the development of biopharmaceuticals containing cannabinoids and other medicinal plant-based elements. This cream is composed of components of the cannabis plant, cannabidiol (CBD) and tetrahydrocannabinol (THC).

PPP004 was developed to help manage the pain and itch associated with EB, which have a huge impact on daily life.

There are multiple advantages that come with ODD, such as…

· Cost reduction in drug development / clinical trials.

· Marketing exclusivity in the EU for 10 years following authorization.

· Access to specialized medical expertise from authorities

· Access to a quicker review process

The cream will be packaged in a metered-dose, airless pump system to ensure that both CBD and THC are given in equal amounts of the same dose. It also protects the compounds from degrading, caused by exposure to light and oxygen.

Tetra have stated they are ready to test PPP004 in human clinical trials

For more information on PPP004 (containing THC-CBD): https://ir.tetrabiopharma.com/newsroom/press-releases/news-details/2022/Tetra-Bio-Pharma-Receives-EMA-Orphan-Drug-Designation-for-a-Novel-Topical-Therapeutic-Containing-CBD/default.aspx