With $30M Investment, Regenacy Plans Phase 2 Trial Testing Ricolinostat for CMT Type 2

Regenacy Pharmaceuticals received $30 million to help advance its lead candidate ricolinostat into Phase 2 studies for Charcot-Marie-Tooth (CMT) disease type 2, as well as diabetic and chemotherapy-induced peripheral neuropathy.

The money comes from a series A financing round, which startup companies need to raise capital. The funding was co-led by Cobro Ventures and Taiwania Capital Management Corporation, and joined by 3E Bioventures Capital, Yonjin Capital, VIVA Biotech, TA YA VENTURE, along with other undisclosed private investors.

“We are fortunate to have the strong support of investors aligned with our mission to provide lasting relief of all symptoms of diabetic peripheral neuropathy,” Simon Jones, PhD, Regenacy’s president and CEO, said in a press release.

The peripheral nervous system is key in the communication between the brain and spinal cord and all other parts of the body. Peripheral nerves send signals in two directions, relaying sensory information like cold feet from the extremities to the brain, but also sending signals from the brain to the rest of the body, such as those that tell muscles to contract.

When these nerves are damaged, patients have a form of peripheral neuropathy, and experience symptoms including weakness, numbness, and pain in the hands, feet, arms, and legs.

One of the most common problems in these patients is that the network of microtubules that transport information from one end of peripheral nerves to the other is destabilized.

This network is particularly important in nerve cells of the peripheral nervous system, since their axons (or nerve fibers) cover long distances. But it may become disrupted as a result of diabetes, the use of neurotoxic chemotherapeutic agents, or due to genetic mutations, as is the case in CMT type 2.

Ricolinostat is designed to restore the normal transport function of microtubules by blocking the function of histone deacetylase 6 (HDAC6). This protein prevents a chemical modification in the building blocks of microtubules, which is needed to help them create a stable network.

Preclinical studies in multiple animal models of peripheral neuropathies have demonstrated ricolinostat’s ability to restore nerve function, reversing pain and numbness. In a mouse model of CMT2, ricolinostat increased the communication between nerve and muscle cells, and improved conduction of motor and sensory nerve impulses.

Later Phase 1 (NCT02088398) and Phase 2 trials involving healthy volunteers found that the treatment is safe and well-tolerated, contrasting with other agents targeting a wide number of HDAC proteins.

“Our preclinical studies demonstrate that selectively inhibiting HDAC6 has the potential to safely restore nerve function in multiple peripheral neuropathies, and we are looking forward to advancing our lead program into Phase 2 studies this year,” Jones said.

The funding will first support a proof-of-concept Phase 2 trial (NCT03176472) in diabetic peripheral neuropathy (the lead indication for which ricolinostat is being developed), which will measure changes in pain intensity across 450 patients treated with either ricolinostat or a placebo for four weeks.

But Regenacy is also planning to advance ricolinostat to Phase 2 studies in CMT type 2 and chemotherapy-induced peripheral neuropathy, the company said.

“To me, the scientific reasoning behind HDAC6 inhibition for this indication is very clear, as it has significant potential to reverse the symptoms of and restore nerve fibers in different peripheral neuropathies by regulating neuronal microtubule function,” said William Chin, MD, a professor at Harvard Medical School and member of Regenacy’s board of directors.