Benzinga | Sep 24, 2020 08:09AM EDT

Cortexyme Announces GAIN Trial Of Atuzaginstat In Alzheimer's Disease Has Exceeded Study Enrollment Target Of 570 Patients

- Interim analysis of the Phase 2/3 GAIN Trial expected to occur before year-end 2020

Cortexyme, Inc. (NASDAQ:CRTX) today announced that its GAIN Trial for Alzheimer's disease (AD) has exceeded its enrollment target of 570 patients. GAIN is a randomized, double-blind, placebo-controlled Phase 2/3 trial of atuzaginstat (COR388), Cortexyme's lead investigational medicine, in patients with mild to moderate AD. The GAIN Trial's protocol includes an interim analysis on the study's co-primary cognitive and global endpoints after approximately 300 patients reach 24 weeks of treatment, and this interim analysis is expected to occur before year-end 2020. Top-line results of the GAIN Trial's final analysis, to be performed once all study subjects complete the one-year study, are expected in Q4 2021.

The GAIN Trial was designed to enroll patients with mild to moderate Alzheimer's disease, randomized 1:1:1 to receive atuzaginstat 40 mg twice a day, 80 mg twice a day a day, or placebo. GAIN Trial enrollment has already exceeded the enrollment targets in the three double-blind arms. Although new screening of patients for the double-blind arms has ended, eligible patients who have already screened for the study will continue to enroll over the next several weeks.

"The GAIN Trial is an important study of a potential new approach to addressing Alzheimer's. We are pleased the trial has reached this milestone on schedule, and we look forward to completing the trial's interim analysis before year-end 2020," said Michael Detke, M.D., Ph.D., Cortexyme's Chief Medical Officer. "The significant need for new therapeutic options for Alzheimer's patients grows every day, and we are encouraged by the body of evidence supporting atuzaginstat's mechanism of action, which is 'upstream' of other approaches."

Atuzaginstat targets the toxic proteases, or gingipains, produced by P. gingivalis, which have been discovered in greater than 90% of post-mortem brains of patients with AD and shown to produce Alzheimer's pathology in infected animals. P. gingivalis is best known as a keystone bacterium in the development of periodontal disease. The GAIN Trial includes a periodontal sub-study, in which approximately 40% of GAIN Trial participants are also assessed for endpoints of efficacy in periodontal disease. Of the sub-study participants enrolled to date, greater than 90% had moderate to severe periodontal disease at baseline.

For more information about the GAIN Trial, visit www.GAINtrial.com.