Regenacy Partners with CMTA to Advance Development of Ricolinostat for Treating CMT

Regenacy Pharmaceuticals is partnering with the Charcot-Marie-Tooth Association (CMTA) to help develop the investigative therapy ricolinostat and evaluate its potential for Charcot-Marie-Tooth (CMT) in animal models.

The collaboration is expected to speed the advancement of the treatment candidate for clinical trials in CMT patients.

Regenacy and CMTA — a patient-led, nonprofit organization dedicated to finding a cure for CMT — will work together to better understand the role of the HDAC6 enzyme in CMT and investigate if ricolinostat, a potent and selective inhibitor of the enzyme, effectively treats CMT in animal models. 

The project is based on previous preclinical research showing that HDAC6, the histone deacetylase 6 enzyme, is a potential therapeutic target in axonal CMT, or CMT2. The enzyme seems to contribute to a deficient transport of molecules within nerve fibers, or axons, leading to the damage and malfunctioning of nerves.

In the study, treatment with ricolinostat (also known as ACY-1215) was able to revert the motor and sensory nerve fiber deficits in a mouse model of CMT2.

The treatment increased innervation at the nerve cell-muscle interfaces, or neuromuscular junctions, and improved the conduction of motor and sensory nerve impulses.

Ricolinostat is a promising drug candidate under development for other conditions, as well. Regenacy recently launched a Phase 2 trial (NCT03176472) to investigate the effectiveness of the compound for treating pain due to another type of peripheral neuropathy caused by diabetes.

The investigative therapy is also in clinical trials as a single agent or in combination with other drugs for the treatment of several types of cancer, including multiple myeloma and breast cancer.

“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,” Matt Jarpe, PhD, vice president of research and development at Regenacy Pharmaceuticals, said in a news release.

“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.”

According to Regenacy, ricolinostat has demonstrated an excellent safety and tolerability profile in Phase 1 and Phase 2 clinical trials.

Now, the preclinical results obtained by the Regenacy-CMTA partnership are anticipated to speed up the translation of the HDAC6 inhibitor into an effective therapy against CMT.

“Current treatment options for Charcot-Marie-Tooth disease, and related disorders, are very limited so each potential new therapy can make a critical difference for patients in need,” said David Herrmann, MD, professor at the University of Rochester, member of the CMTA STAR advisory board, and recently appointed scientific adviser of Regenacy.