Novartis is making significant strides in the field of Charcot-Marie-Tooth (CMT) disease treatments, having recently entered into a substantial $1.3 billion deal with Chong Kun Dang Pharmaceutical Corp (CKD). The South Korean pharmaceutical company announced on November 6th that it has signed a licensing agreement with Novartis, granting the Swiss multinational pharmaceutical giant the exclusive rights to develop and commercialize CKD-510, an investigational drug with FDA orphan drug designation for the treatment of CMT.

As part of the agreement, Novartis will pay CKD an initial upfront fee of $80 million. Additionally, CKD stands to receive milestone payments totaling $1.2 billion over the course of CKD-510’s development and approval stages. Furthermore, CKD will be entitled to separate royalties based on the drug’s sales. CKD-510, developed by Chong Kun Dang, functions as an inhibitor of histone deacetylase 6 (HDAC6). Preclinical trials conducted by the Korean pharmaceutical company demonstrated the drug’s efficacy in treating cardiovascular diseases and other HDAC6-related disorders. Phase 1 trials in France also confirmed its safety and tolerability.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to CKD-510 in March 2020 for the treatment of CMT. Orphan drug status is assigned to investigational medicines targeting rare diseases, offering developers incentives such as tax credits for qualified clinical trials and exemption from user fees in the United States.

Novartis has secured exclusive rights to develop and market CKD-510 in international markets, excluding Korea, where Chong Kun Dang retains the rights. This agreement follows closely on the heels of Novartis’ acquisition of DTX-1252, another potential therapeutic for CMT1A, in July. It signals Novartis’ strategic commitment to expanding its CMT portfolio.

The significance of this collaboration is underscored by the substantial financial investment and the broader landscape of CMT research. HDAC6 has long been a focal point of CMT research, with initiatives supported by the Charcot-Marie-Tooth Association’s Strategy to Accelerate Research (CMTA-STAR). The transaction’s scale reflects the progress made in positioning CMT as an attractive proposition for pharmaceutical companies, one of the strategic aims of STAR.

The Charcot-Marie-Tooth Association (CMTA) is closely monitoring these developments and will share additional information with the CMT community as it becomes available.

About HDAC6 Inhibition:

HDAC6 is an enzyme that regulates mitochondrial function and intracellular transport along microtubules. Peripheral nerves, due to their length, are particularly sensitive to disruptions in this transport, leading to pain, numbness, and loss of muscle control, often exacerbated by mitochondrial dysfunction. Animal studies in various peripheral neuropathy models have identified HDAC6 inhibition as a promising therapeutic approach to restore nerve function and alleviate symptoms.

Source: Korea Biomed

Published: November 30, 2023