CKD-510, also known as lobisomide, is an investigational drug candidate. It is being evaluated as a potential new therapeutic option for specific medical conditions and is progressing through the established phases of clinical research to determine its properties and effects in humans.
Mechanism of Action
CKD-510 is classified as a selective histone deacetylase 6 (HDAC6) inhibitor. These enzymes act as regulators within cells by removing specific chemical tags from proteins, a process that can alter protein function and gene expression.
The HDAC6 enzyme has a specialized job related to the cell’s internal transport system. It helps regulate the movement of components along structures called microtubules, which function like highways for trafficking proteins and organelles throughout the cell. The HDAC6 enzyme can act as a “brake” on this transport system. This is especially impactful in nerve cells, or neurons, which are very long and rely heavily on this transport system to maintain their health and function.
By specifically blocking the HDAC6 enzyme, CKD-510 is designed to “release the brake” on this cellular machinery. This action restores or improves the flow of traffic along the microtubule highways. The enhanced transport of materials like mitochondria and other structural proteins is believed to be the primary therapeutic benefit of inhibiting HDAC6.
Investigated Therapeutic Applications
The primary therapeutic application being investigated for CKD-510 is Charcot-Marie-Tooth disease (CMT). CMT is a group of inherited genetic disorders that cause progressive damage to the peripheral nerves. These are the nerves outside of the brain and spinal cord, relaying signals to and from the limbs, leading to muscle weakness, atrophy, and sensory loss in the feet, legs, hands, and arms.
The rationale for using an HDAC6 inhibitor for CMT is directly linked to the biology of the disease. A problem in many forms of CMT is the disruption of axonal transport. Genetic mutations in CMT can lead to the production of faulty proteins that disrupt nerve structure and function, and impaired transport along the nerve’s axon exacerbates this damage.
By inhibiting HDAC6, CKD-510 aims to overcome this transport deficit, helping to clear protein aggregates and deliver materials to the far ends of the long nerve cells. Preclinical studies in animal models of CMT have shown that inhibiting HDAC6 can improve motor function and maintain the integrity of axons and their protective myelin sheath. The mechanism of HDAC6 inhibition is also being explored for other neurodegenerative diseases.
Clinical Trial Findings
The evaluation of CKD-510 has progressed through early-stage clinical trials. A Phase 1 first-in-human study was conducted in Europe to establish the drug’s safety, tolerability, and pharmacokinetic profile. This trial enrolled 87 healthy adult male volunteers and used a randomized, double-blind, placebo-controlled design.
The results from this initial phase were positive, indicating that CKD-510 demonstrated good safety and tolerability. The study also provided data on how the drug is absorbed, distributed, and processed by the body, confirming that it could be developed as a once-daily oral medication. Inhibition of the HDAC6 enzyme was also observed, confirming the drug was engaging its target.
Following the completion of the Phase 1 trial, the developer announced its intention to move forward with a global Phase 2 study to evaluate the drug’s effectiveness in patients diagnosed with Charcot-Marie-Tooth disease. Specific adverse events from the Phase 1 trial have not been detailed in publicly available information, but the overall conclusion was that the drug was well-tolerated.
Development and Future Outlook
CKD-510 is advancing in the clinical development pipeline. The drug is now set to enter Phase 2 studies to test its efficacy in patients. The drug was initially developed by the South Korean firm Chong Kun Dang (CKD) Pharmaceutical.
In a development in late 2023, Novartis acquired the exclusive rights to develop and commercialize CKD-510 worldwide, with the exception of South Korea. This agreement, valued at up to $1.3 billion, signals strong interest in the drug’s potential. In May 2025, Novartis submitted an Investigational New Drug (IND) application to the U.S. FDA to begin a Phase 2 trial.
The next step involves enrolling CMT patients in this Phase 2 trial to gather data on whether the drug can improve disease symptoms. If these results are promising, the path forward would involve larger, more definitive Phase 3 trials. These studies are required by regulatory bodies like the FDA before a drug can be considered for market approval, a process that offers no guarantee of success.