StarWise Therapeutics LLC based in Madison, WI has developed novel, safe HDAC6 inhibitors for use in the treatment of various neurological disorders. On the top of its list is the use of its new drugs in the potential treatment of the genetic disorder known as Charcot-Marie-Tooth disease, or CMT for short.

CMT is a progressive genetic disease of which there are many variants. CMT2 is a mutation in the sequence of the mitofusin 2 gene or MFN2. MFN2 is a protein that plays an essential role in the function of mitochondria which generate energy in the cells. In MFN2 patients, the energy produced by neuronal mitochondria is insufficient to sustain axons. As a consequence, axonal degeneration gradually occurs, causing muscle weakness, atrophy and deformities in the feet, legs, arms and hands. Those with CMT often lose the ability to walk and may become dependent upon assistive devices to remain mobile. Severe, chronic pain is common, and there is no cure.

StarWise Therapeutics and Waikato University in NZ have demonstrated that their key drug is able to restore sensory and motor function in the CMT2A mutant animals to performance levels akin to those of the wild type animals.

The Hereditary Neuropathy Foundation (HNF) is a non-profit 501(c)3 global organization whose mission is to increase awareness and accurate diagnosis of CMT, support patients and families with critical information to improve quality of life, and support research. HNF has notably developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government, and industry to support and develop treatments for CMT.

These two entities have now linked their expertise in order to advance the StarWise agents to the clinic. Key medicinal chemistry, biology, and clinical experts will be meeting in NYC this week to make plans for the filing of an Investigation New Drug (IND) application with the FDA for the treatment of this disorder. And this couldn’t be a more timely with the upcoming HNF-hosted FDA externally-led Patient-Focused Drug Development (PFDD) Meeting on September 28 in Washington, DC. StarWise and HNF are forging the way to accelerating therapies to the clinic for many CMT sub-types and for the first time CMT2A will be added to the that list. Stay-tuned as we will be reporting with research and clinical updates!