Kainos Medicine Receives FDA Approval of IND for Phase 2 Trial of KM-819
Kainos Medicine Receives FDA Approval of IND for Phase 2 Trial of KM-819
  • Hyun Duk Yang
  • 승인 2021.11.17 09:33
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KM-819, first-in-class disease-modifying therapeutic for PD
Inhibits Fas-Associated Factor1 (FAF1) protein

Kainos Medicine (hereafter Kainos for short), a clinical-stage biotech company headquartered in Seongnam, South Korea, specializing in the research and development of innovative medicines for brain diseases, cancers and infectious diseases, announced on November 16th that the U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) application for KM-819 as a potential treatment for Parkinson’s disease (PD).

Kainos’ IND application for phase 2 clinical studies was submitted in October by its subsidiary FAScinate Therapeutics based in San Diego, California, developing disease-modifying drugs for neurodegenerative diseases like PD and multiple system atrophy (MSA) that are caused by abnormal accumulation of alpha-synuclein proteins. In September, FAScinate had licensed KM-819 for clinical development and commercialization from Kainos.

KM-819 is the first-in-class disease modifying therapeutic for PD, protecting dopaminergic neurons from death by inhibiting the over-expression of the Fas-Associated Factor1 (FAF1) protein which is known to be proapoptotic. By blocking the FAF1, KM-819 also activates the function of autophagy, leading to the breakdown of alpha-synuclein which is a trigger of PD. KM-819 has shown the dopaminergic neuron protection in the PD animal models, along with the inhibition of behavioral abnormality. KM-819 has gone through its phase 1 clinical studies for PD and MSA in South Korea successfully.

The IND approval enables Kainos to initiate a clinical trial to evaluate safety, tolerability and preliminary efficacy of KM-819 for the treatment of PD. This phase 2 clinical trial is divided into two parts, 1a/1b and 2. The part 1a/1b is a dose-finding study (200 mg, 400 mg, and 800 mg of KM-819) in 18 healthy elderly subjects and 24 patients with PD. The part 2 is aimed at evaluating the safety, tolerability, and preliminary efficacy in 288 PD patients for two years in the U.S. 

As the efficacy was found in experiments using the stem cells of patients with the specific gene (GBA, encoding the lysosomal enzyme glucocerebrosidase) mutations, this clinical trial will include the patients with mutations in GBA.

If the plan proceed as expected, Kainos will become the first Korean pharmaceutical company to conduct a large-scale clinical trial of PD drug in the U.S.

Meanwhile, Kainos announced on October 20th that the Ministry of Food and Drug Safety of Korea approved the phase 2 study for MSA.

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