Cortexyme Inc. (NASDAQ:CRTX) has announced that an independent Data Monitoring Committee (DMC) has completed a pre-planned preliminary analysis and recommended the company to go on with Phase 2/3 GAIN study of COR388 (atuzaginstat) as previously planned.
Cortexyme to continue Phase 2/3 GAIN study in atuzaginstat
The preliminary analysis included around 300 patients that reached six months of treatment in the GAIN study. The double-blind, randomized placebo-controlled trial evaluated 40 mg and 80 mg atuzaginstat BID. The DMC looked at sample size increase if necessary to enhance powering, safety, overwhelming efficacy, and futility as part of the preliminary analysis. According to the preliminary analysis, the company expects to release topline data of the 643 enrolled subjects after one full year of treatment in December 2021. Cortexyme remains blinded to all study data.
The GAIN study’s principal investigator, Dr. Marwna Sabbagh said that there is a significant unmet medical need in Alzheimer’s disease and thus it is important to quickly study possible new therapies. Sabbagh said that they are looking to finalizing the trial and share results with the medical community once full study results are available.
P. gingivalis present in 90% of AD patients
Michael Detke, the company’s Chief Medical officer stated that they are pleased with the recommendation from the DMC which supports the trial design and statistical powering of the GAIN study. He added that evidence totality around gingipain and P. gingivalis inhibition demonstrates that blocking the upstream target can impact several disease progression aspects and neurodegeneration. Michael lauded the efforts of investigators, the DMC as well as patients and their caregivers for participation in the study until its 1-year endpoint.
Cortexyme’s atuzaginstat targets gingipains or toxic proteases produced by P. gingivalis found in more than 90% of Alzheimer’s disease patients. It also produces Alzheimer’s pathology and neurodegeneration in patients. Interestingly P. gingivalis is a keystone bacterium that leads to periodontal disease development.
The GAIN Study will evaluate atuzaginstat’s safety, tolerability, and efficacy in mild to moderate AD. The study’s co-primary endpoints are median change in ADCS-ADL and ADAS-Cog 11 from baseline to week 48.