According to a story from PR Newswire, the biopharmaceutical company Constant Pharmaceuticals recently announced that it plans to develop its experimental compound TXA127 as a therapy for epidermolysis bullosa (EB). This development will be done in collaboration with several epidermolysis bullosa focused nonprofit organizations, including the Epidermolysis Bullosa Medical Research Foundation, DEBRA (Austria), the Epidermolysis Bullosa Research Partnership, and Cure-EB. Constant focuses on the development of therapies for connective tissue diseases, neuromuscular disorders, and stroke recovery.
About Epidermolysis Bullosa
Epidermolysis bullosa is a group of genetic conditions which are most characterized by unusual blistering of the mucous membranes and skin. These painful blisters can occur with only minor friction or abrasion. The disease can be fatal in severe cases. These disorders are all caused by genetic mutations; the various subtypes of epidermolysis bullosa are classified by which genes are affected. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer. There is no cure for the condition; treatment primarily focuses on symptom management, such as controlling pain, cleaning wounds, prevention or treatment of complications, nutritional support, and controlling infections. Males and females are affected at equal rates. The disease can seriously shorten lifespan, and in severe cases, patients do not survive beyond infancy. There is a dire need for more effective treatment approaches for epidermolysis bullosa. To learn more about these disorders, click here.
The development plan as of this juncture will consist of the reformulation of TXA127 into an orally available form. Modifying the drug in this way is necessary as TXA127 is currently administered using a subcutaneous injection, which would not be suitable for epidermolysis bullosa. Using this new formulation, Phase 2 clinical trials will be conducted both in Europe and the US. Prior research with TXA127 using animal models of the most severe form of epidermolysis bullosa, known as recessive dystrophic epidermolysis bullosa, suggested that the experimental drug could offer meaningful benefit to patients; the effect may be even more pronounced in less severe types.
Hopefully, this endeavor will result in a new, more effective treatment for this devastating disorder.