Chong Kun Dang Unveils Clinical Phase 1 and Non-Clinical Study Results of Charcot-Marie-Tooth Treatment New Drug 'CKD-510'

International Congress of Peripheral Nerves Annual Conference Presentation
Safety and Tolerability Confirmed in European Phase 1 Clinical Trial

Jongkundang exterior.

[Asia Economy Reporter Lee Gwan-joo] Chong Kun Dang announced on the 18th that it presented the results of the European Phase 1 clinical trial and non-clinical studies of the Charcot-Marie-Tooth treatment new drug 'CKD-510' at the Peripheral Nerve Society (PNS) Annual Meeting held in Miami, USA, from the 14th to the 17th (local time).

CKD-510 is a next-generation drug candidate applying a non-hydroxamic acid platform technology that inhibits histone deacetylase 6 (HDAC6), and is being developed as a treatment for Charcot-Marie-Tooth disease. This presentation covered the Phase 1 clinical trial of CKD-510 conducted in Europe, marking the first disclosure of clinical results for an HDAC6 inhibitor using the non-hydroxamic acid platform technology.

Chong Kun Dang stated that CKD-510 demonstrated excellent safety and tolerability in a Phase 1 clinical trial involving 87 healthy adults. Meaningful results were confirmed in the pharmacokinetic profile, which shows how much the drug is absorbed and eliminated over a certain period in the body, and in the dose-dependent inhibition of HDAC6 activity, securing the possibility of developing CKD-510 as a once-daily oral treatment.

The non-clinical study results of CKD-510 were also included. In non-clinical studies conducted on disease animal models, CKD-510 selectively inhibited HDAC6, improving peripheral nervous system axonal transport function and preventing abnormal protein aggregation, thereby confirming its efficacy as a drug that improves motor function through this mechanism.

Charcot-Marie-Tooth disease is a hereditary peripheral neuropathy caused by genetic abnormalities. It is a rare disease characterized by muscle atrophy and deformity of the hands and feet, loss of motor and sensory functions, making walking and daily life difficult. Currently, there are no approved treatments worldwide. CKD-510 was designated as an orphan drug by the U.S. Food and Drug Administration (FDA) in March 2020.

A Chong Kun Dang official said, “Based on the results of this European Phase 1 clinical trial, we plan to rapidly proceed with a global Phase 2 clinical trial targeting Charcot-Marie-Tooth patients,” adding, “We will accelerate the development of treatments for Charcot-Marie-Tooth disease, which has a high unmet medical need, to contribute to improving the quality of life for patients with rare diseases.”