KainosMed Announces Mechanism of FAF1 Inducing α-syn Accumulation, a Cause of Degenerative Brain Diseases

[Asia Economy Reporter Hyunseok Yoo] Kainosmed recently announced on the 25th that it has published a paper elucidating the mechanism of Parkinson's disease development, revealing that FAF1 (Fas-associated factor 1) induces the accumulation of alpha-synuclein (α-synuclein, α-syn), which is known as a major cause of degenerative brain diseases (CNS).

The paper was published in the FASEB Journal, jointly issued by the Federation of American Societies for Experimental Biology.

Based on the fact that FAF1 and alpha-synuclein coexist in the brains of Parkinson's disease patients, the research team investigated the pathological interaction between FAF1 and alpha-synuclein.

The researchers found that FAF1 increases the amount of alpha-synuclein by interfering with the autophagy pathway that degrades alpha-synuclein in dopaminergic neurons. When adeno-associated virus loaded with FAF1 (FAF1-AAV) was injected into the midbrain of mice, not only did alpha-synuclein accumulate, but autophagy was also inhibited.

KM819, which Kainosmed is developing as a treatment for Parkinson's disease, inhibits the function of FAF1, thereby ▲ preventing neuronal cell death and ▲ promoting the degradation of alpha-synuclein and inhibiting its aggregation by activating autophagy function, thus blocking the progression of Parkinson's disease as its mode of action.

The significance of this research result lies in identifying a new function of FAF1 in the development of Parkinson's disease, beyond the previously known role of FAF1 in inducing neuronal cell death, showing that FAF1 suppresses autophagy and induces alpha-synuclein accumulation.

A company representative stated, “This study reaffirms that KM819 is not merely a symptom reliever but a disease-modifying drug that halts the progression of Parkinson's disease. We will do our best to enter Phase 2 clinical trials in the United States this year to confirm the efficacy of KM819 in Parkinson's disease.”