Korean Biotech Licenses Rare Disease Candidate to Novartis for Up to $1.3 Billion

South Korean biotech Chong Kun Dang Pharmaceutical licensed it’s phase 1 experimental therapeutic for the treatment of Charcot-Marie-Tooth disease to Novartis in a deal potentially worth up to $1.3 billion, according to a report from The Korea Economic Daily.

Under their agreement, CKD grant Novartis exclusive ex-Korea rights to develop and commercialize the CKD-510, an investigational HDAC6 inhibitor for the rare hereditary disease Charcot-Marie-Tooth, also known as hereditary motor and sensory neuropathy, or peroneal muscular atrophy. Novartis will pay CKD $80 million upfront and potentially up to $1.8 billion in development and regulatory milestones, plus royalties if approved.

The deal is the largest out-licensing deal for Chong Kun Dang in its 80-year history. The company’s stock soared 26 percent in trading on the Korean Stock Exchange following the news.

Charcot-Marie-Tooth disease (CMT) is one of a group of disorders that cause damage to the peripheral nerves—the nerves that transmit information and signals from the brain and spinal cord to and from the rest of the body, as well as sensory information such as touch, back to the spinal cord and brain. CMT also can directly affect the nerves that control the muscles.

Progressive muscle weakness typically becomes noticeable in adolescence or early adulthood, but the onset of disease can occur at any age. Because longer nerves are affected first, symptoms usually begin in the feet and lower legs and then can affect the fingers, hands, and arms. Most individuals with CMT have some amount of physical disability, although some people may never know they have the disease.

CKD-510 is an inhibitor of histone deacetylase 6, or HDAC6, a family of enzymes that block cell cycle modulators. CKD developed it to regulate inflammatory diseases, including both cardiovascular and neurodegenerative disorders.

Chong Kun Dang demonstrated the efficacy of CKD-510 in treating cardiovascular diseases and other HDAC6-related disorders during preclinical trials, as well as its safety and tolerability in phase 1 trials in France. The U.S. Food and Drug Administration in March 2020 designated CKD-510 as an orphan drug to treat CMT.

With 10 new chemical entities in its pipeline, the company said it spends about 12 percent of its annual revenue on average every year to develop breakthrough therapies or first-in-class drugs using novel mechanisms, Kim Young-Joo, CEO and chairman of Chong Kun Dang Pharmaceutical told The Korean Economic Daily.