• There are currently no FDA approved treatments to stop or reverse loss of function in CMT
• Ricolinostat is currently also being studied by Celgene as a cancer treatment
• Regenacy has limited rights to ricolinostat in some nervous system disorders

Regenacy Pharmaceuticals, LLC, announced on 8/2/18 a collaboration with the Charcot-Marie-Tooth Association (CMTA), a registered non-profit organization serving the hereditary neuropathy patient community, to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth (CMT) disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models. In addition, the company announced the appointment of David Herrmann, M.B.B.Ch., to its Scientific Advisory Board. Dr. Herrmann is Chief of the Neuromuscular Division & Director of the Peripheral Neuropathy Clinics and Cutaneous Innervation Laboratory in the Department of Neurology at the University of Rochester, and a member of the STAR (Strategy to Accelerate Research) advisory board for the CMTA.

“We are thrilled to have such a substantial collaboration to broaden our programs for ricolinostat into inherited forms of neuropathy where there is a tremendous unmet need,” said Matt Jarpe, Ph.D., Vice President of R&D of Regenacy Pharmaceuticals. “This alliance with the Charcot-Marie-Tooth Association will enable us to expand our understanding of the role of HDAC6 in neuropathic diseases and lead us closer to initiating clinical trials in CMT disease.”

CMT disease is a progressive and degenerative nerve disease that usually appears in adolescence or early adulthood. Symptoms can include muscle weakness, decreased muscle size, foot-drop, foot bone abnormalities, fatigue, balance problems, neuropathic and/or musculoskeletal pain, loss of feeling in the hands and feet and loss of coordination in the limbs. There are no FDA approved treatments to stop or reverse the loss of nerve function in CMT. Based on the terms of the collaboration, Regenacy has an opportunity to expand on the groundbreaking  work  of Dr. Ludo van den Bosch at University of Leuven and Dr. Andrew Grierson at University of Sheffield to show the role of HDAC6 in several forms of CMT Type 2. The collaboration will focus on evaluating the efficacy of ricolinostat in animal models of CMT to support initiation of clinical trials. This relationship is taking advantage of the extensive suite of expert preclinical testing capabilities the CMTA has assembled and makes available to groups such as Regenacy that want to evaluate therapeutic potential in a CMT disorder.

Ricolinostat is an HDAC6 inhibitor is also in clinical trials as a cancer treatment. Regenacy was formed when Celgene purchased assets from Acetylon Pharmaceuticals. Regenacy only has rights to ricolinostat in some nervous system disorders.