Chong Kun Dang released the findings of European phase 1 and pre-clinical trials of CKD-510, an investigational drug for Charcot-Marie-Tooth, at the annual meeting of the Peripheral Nerve Society (PNS), held in Miami from May 14-17.
According to the company, CKD-510 uses non-hydroxamic acid platform technology that inhibits histone deacetylase 6 (HDAC6).
Chong Kun Dang’s phase 1 study of CKD-510 conducted in Europe is the first trial of an HDAC6 inhibitor that used non-hydroxamic acid platform technology.
PNS selected CKD-510 as a late-breaking poster. The late-breaking poster designation allows additional opportunity to present new results or outcomes expected to affect the related area significantly even after the deadline for data submission to the conference.
Chong Kun Dang said CKD-510 demonstrated excellent safety and tolerability in healthy 87 adults in the phase 1 study. The company also found meaningful outcomes in the drug candidate’s in vivo kinetic profiles and inhibition of HDAC6. This secured the possibility of developing it as an oral, once-daily treatment.
Chong Kun Dang’s presentation also included the results of the pre-clinical trial of CKD-510 on animal models with the disease. The treatment candidate selectively inhibited HDAC6 to improve the axon transport function of the peripheral nervous system and prevented abnormal protein aggregation from improving motor function.
Charcot-Marie-Tooth is an inherited disorder that causes damage to the peripheral nerves. The disease makes it difficult to walk or carry normal life because of muscle atrophy in the hands and feet and loss of motor and sensory functions. There is no authorized treatment for the disease.
In March 2020, the FDA designated CKD-510 an orphan drug to treat Charcot-Marie-Tooth.
“Based on the European phase 1 trial outcomes, we will promptly conduct a global phase 2 study on Charcot-Marie-Tooth patients,” an official at Chong Kun Dang said. “We will accelerate the development of Charcot-Marie-Tooth treatment with high unmet medical needs and contribute to improving the quality of life of rare disease patients.”
The annual meeting of PNS is the world’s largest peripheral nerve educational event where over 1,000 peripheral nerve researchers, clinicians, and healthcare professionals participate in sharing the latest knowledge.