KainosMed Submits IND for Multiple System Atrophy Phase 2 Clinical Trial to MFDS

[Asia Economy Reporter Yoo Hyun-seok] Kainosmed announced on the 27th that it has submitted an Investigational New Drug application (IND) for the Phase 2 clinical trial of KM819, a candidate drug for the treatment of multiple system atrophy (MSA), a rare degenerative brain disease (CNS), to the Ministry of Food and Drug Safety.

KM819 is a drug currently being developed by Kainosmed as a treatment for Parkinson's disease and is preparing for Phase 2 clinical trials in the United States. Multiple system atrophy is a movement disorder similar to Parkinson's disease, characterized by rapid progression and high mortality, but currently has no treatment. The number of patients is rare, accounting for 5-10% of Parkinson's disease patients.

This clinical trial is the first clinical test of KM819 on patients, and it is particularly valuable as it will be conducted efficiently and rapidly in Korea compared to overseas trials such as those in the United States. Kainosmed has previously conducted efficacy experiments on KM819 for multiple system atrophy in collaboration with overseas researchers.

The Phase 2 clinical trial will be conducted at Seoul Asan Medical Center with 78 MSA patients. It includes randomized allocation, double-blind administration, placebo control, and open-label administration. The goal is to complete Phase 2 by 2023. Since MSA is a rare disease, conditional approval after Phase 2 is also expected. The study will be led by Professor Lee Jong-sik, a renowned expert in this field.

Multiple system atrophy is caused by the accumulation of a protein called alpha-synuclein, similar to Parkinson's disease. The difference is that this protein accumulates not in the brain's neurons but in the glial cells that protect and support neurons.

Kainosmed's KM819 inhibits the overexpression of a protein called FAF1, which promotes cell death, thereby preventing neuronal cell death and activating autophagy to prevent the accumulation of alpha-synuclein.

In animal experiments, KM819 reduced the accumulation of alpha-synuclein, improved motor function, and extended lifespan. The survival rate of rats administered KM819 was significantly higher than that of rats not administered the drug.

A company official stated, "We expect to confirm the efficacy of KM819 through the Phase 2 clinical trial for multiple system atrophy," adding, "This is also expected to be a positive signal for the success of the Parkinson's disease treatment clinical trials to be conducted in the United States."