Raptor Pharmaceuticals Corp found its "experimental brain disorder drug" showed a positive trend towards slower progression of Total Motor Score (TMS) in patients with Huntington's Disease.
A research team looked at 96 patients with Huntington's disease, which is characterized by a degeneration in the nerve cells in the brain that causes loss of movement and control, Reuters reported.
"The drug slowed the progression of muscle spasms, eye and hand movements and loss of balance compared to a placebo," Reuters reported.
During the 38-month-long study the patients were allowed to continue taking their baseine medications.
"We are very encouraged by these trial results and we believe that the significant slowdown of loss of muscle control in these early stage patients indicates that RP103 is potentially effective at slowing the progression of Huntington's disease," doctor. Christophe Verny, Lead Investigator for the Phase 2/3 clinical trial and Chief of Neurology Department at CHU d'Angers, said in a news release via the Wall Street Journal. "We look forward to working with Raptor to develop and implement a continuing access program so that we can continue to provide RP103 to the patients who participated in the study initiated by CHU d'Angers, and avoid any treatment interruption after they finish the study."
"We are very grateful to the patients who participated in the study and the French clinical network of physicians, led by Drs. Christophe Verny and Dominique Bonneau in Angers," Patrice Rioux, M.D., Ph.D., Chief Medical Officer at Raptor said in the news release. "We are thrilled to build on these results and will engage regulatory agencies to discuss the most efficient means to advance this program to potential approval. These results not only support the safety and efficacy of Raptor's RP103 in Huntington's disease, but also the rationale for testing the use of RP103 in other indications such as NAFLD, Leigh Syndrome and mitochondrial diseases."
The drug, codenamed RP103, is also being tested for the treatment of non-alcoholic fatty liver disease. Over the course of the study six Huntington's disease patients discontinued treatment, Reuters reported.
