americanpharmaceuticalreviewNovember 29, 2018
Tag: clinical development , Constant Pharmaceuticals , TXA127 , RDEB
Constant Pharmaceuticals plans to begin a development program for its compound, TXA127, for the treatment of the rare pediatric disease, epidermolysis bullosa in collaboration with four EB focused charities - DEBRA (Austria), Cure-EB, the Epidermolysis Bullosa Research Partnership (EBRP), and the Epidermolysis Bullosa Medical Research Foundation (EBMRF). The plan is for this program to first complete the development of an oral formulation of the TXA127 peptide and then conduct a Phase 2 clinical trial in Europe and the US with that formulation.
In work done at the University of Freiburg under the direction of Professor Bruckner-Tuderman, TXA127 showed significant clinical benefit in the animal model corresponding to the most severe form of EB, Recessive Dystrophic Epidermolysis Bullosa (RDEB), and showed positive effects in related in-vitro models. TXA127 demonstrated substantial anti-fibrotic effects and showed a reduction in the fusion of digits, a symptom in RDEB mice that is analogous to mitten deformity common in patients with severe RDEB.
"DEBRA Austria is delighted to be able to provide further support towards the clinical development of TXA127. This will build on the high-quality preclinical research in Freiburg that we have funded to date, that indicates the promise of TXA127 as a candidate to tackle the disabling effects of fibrosis. As a father of an EB patient, it is great to see advances in the development of drugs with possible major beneficial impact on the life of people who live with RDEB," said Dr. Rainer Riedl, CEO of DEBRA Austria.
"We are very pleased to have had the support of DEBRA (Austria) in sponsoring the preclinical investigations of TXA127 for the treatment of RDEB, and thankful to each of the charities for their interest in this important program going forward. There are currently no approved therapies for EB, and TXA127 may represent the first systemic therapy capable of targeting the multi-organ symptoms of this severe disease," said Dr. Richard Franklin, CEO of Constant Pharmaceuticals.
In the first part of the new program, Constant will complete the development of an oral formulation of TXA127. TXA127 is currently delivered as a daily subcutaneous injection, but this is not suitable for patients with EB.
"EB Research Partnership's venture philanthropy model is focused on working together with companies like Constant Pharmaceuticals to accelerate life-changing treatments for people with EB. We look forward to partnering with Constant on the development of TXA127 and are proud to share with them a commitment to innovation and urgency to deliver results to patients," said Michael Hund, Executive Director of EBRP.
The second component of the program will be a Phase 2 trial done in Europe and the US. That trial will examine a number of endpoints that are clinically important in EB, including systemic and cutaneous fibrosis.
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