[Asia Economy Reporter Hyunseok Yoo] Kainosmed announced on the 25th that it received approval from the Ministry of Food and Drug Safety on the 22nd for the clinical trial plan (IND) of its clinical phase 2 candidate drug (KM-819) for the treatment of multiple system atrophy (MSA), a rare degenerative central nervous system (CNS) disease.
Kainosmed has advanced KM-819, which demonstrated excellent efficacy in cell and animal models and high safety in phase 1 clinical trials, into phase 2. Phase 2 is a proof-of-concept (POC) trial primarily aimed at evaluating drug efficacy in actual patients.
Multiple system atrophy is a movement disorder similar to Parkinson's disease. However, unlike Parkinson's disease, there are no clear symptom-relieving drugs, making symptom treatment difficult. Moreover, the disease progresses very rapidly, significantly shortening life expectancy, making it a fatal condition.
This disease manifests as motor dysfunction due to loss of brain function caused by neuronal death in certain brain regions, with symptoms worsening over time, resulting in a survival period of about 6-10 years. Many global pharmaceutical companies have attempted to develop treatments for multiple system atrophy but have not yet succeeded. Some are currently in clinical trials. Kainosmed’s KM-819 differs completely from other companies in its target and drug mechanism, and if successful, it has the advantage of potentially greater efficacy.
KM-819 is a drug currently being developed by Kainosmed as a treatment for Parkinson's disease and recently filed an IND for phase 2 clinical trials with the FDA in the United States. KM-819 is a disease-modifying drug that halts or slows disease progression, representing a fundamental treatment rather than a symptom-relieving drug. KM-819 is a first-in-class drug candidate that inhibits the protein FAF1, which promotes cell death. It protects nerve cells, inhibits apoptosis, and activates autophagy, promoting the degradation of alpha-synuclein and suppressing its accumulation.
KM-819 has demonstrated outstanding efficacy not only in Parkinson's disease but also in multiple system atrophy through numerous studies. In particular, in animal experiments jointly conducted with researchers from the University of California, San Diego, KM-819 showed higher-than-expected effects in reducing alpha-synuclein accumulation, improving motor function, and extending lifespan. The survival rate of rats administered KM-819 was significantly higher than that of untreated rats.
This clinical trial will be conducted at Seoul Asan Medical Center with 78 patients diagnosed with multiple system atrophy. Notably, the trial will use the Unified Multiple System Atrophy Rating Scale (UMSARS) questionnaire to assess clinical symptoms, and FP-PET imaging technology, which can accurately measure the death of dopamine cells?one of the causes of multiple system atrophy?will be applied to all patients for clinical efficacy evaluation.
A Kainosmed official stated, "Multiple system atrophy is a rare degenerative brain disease with no available treatment," adding, "We will accelerate drug development to support patients and their families." The official further commented, "If KM-819’s efficacy is proven in this domestic multiple system atrophy trial, we can expect positive results in overseas clinical trials for Parkinson's disease as well."
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