Constant Pharmaceuticals recently announced plans to begin a development program for TXA127 for the treatment of epidermolysis bullosa (EB). The potential therapy is expected to enter Phase 2 clinical trials in EB patients in 2020.
The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) previously granted TXA127 orphan drug status for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe form of EB.
Now, the development program — a collaboration among Constant Pharmaceuticals and the EB-focused charities DEBRA Austria, Cure-EB, the Epidermolysis Bullosa Research Partnership (EBRP), and the Epidermolysis Bullosa Medical Research Foundation (EBMRF) — aims to conduct a Phase 2 clinical trial in both Europe and the U.S.
“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,” Richard Franklin, PhD, CEO of Constant Pharmaceuticals, said in a press release.
“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,” Franklin said.
Previous studies with TXA127 demonstrated significant clinical effects in an animal model of RDEB. According to the studies, conducted at Germany’s University of Freiburg, the therapy had an anti-fibrotic effect in mouse models, and reduced symptoms analogous to mitten deformities (fusion of the hands and feet by a thin membrane of skin) as seen in RDEB patients.
TXA127 is a pharmacological formulation of the naturally occurring peptide Angiotensin (1-7), which is part of the “alternative” renin-angiotensin-system (RAS). Angiotensin (1-7) has been shown to counteract the detrimental effects of the “classical” RAS pathway, associated with vasoconstriction, fibrosis, and inflammation, among other conditions.
“DEBRA Austria is delighted to be able to provide further support toward 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,” said Rainer Riedl, CEO of DEBRA Austria.
“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,” Riedl added.
Before TXA127 enters Phase 2 clinical trials, the plan is to develop an oral formulation, which is more suitable for EB patients. TXA127 is currently delivered as a daily subcutaneous injection.