The California-based drug development company FAScinate Therapeutics, Inc. announced positive results from the first part of a Phase 2 trial of KM-819, a compound being tested for the treatment of Parkinson’s disease.
KM-819 inhibits FAS-associated factor one (FAF1), a protein that plays a role in programed cell death and is associated with the accumulation of alpha-synuclein. In previous cell and animal studies, KM-819 protected dopaminergic cells and prevented alpha-synuclein from accumulating.
In part 1a of the Phase 2 trial, three dosage strengths of KM-819 demonstrated a favorable safety profile compared to placebo in healthy adults. Part 1b will evaluate the safety and pharmacokinetics of 200 mg, 400 mg, and 600 mg of KM-819 vs placebo in patients with Parkinson’s disease. Results will be used to determine the optimal dosage for study in part 2 of the Phase 2 trial, which will randomly assign patients with Parkinson’s disease to KM-819 or placebo for two years. Effectiveness will be evaluated using the Unified Parkinson’s Disease Rating Scale-Part 2: Activities of Daily Living.
In a press release, FAScinate Therapeutics stated “We are pleased with the prospects of KM-819 as a disease-modifying treatment for Parkinson’s disease as we progress through Phase 2 trials. Part 1a showed favorable safety even at very high doses, and we are excited to now initiate Part 1b. KM-819 shows great promise when it comes to the neuroprotection of dopaminergic cells, including GBA mutation, as well as in clearing alpha-synuclein.” The company also plans to test KM-819 for the treatment of multiple system atrophy.