Cortexyme provides regulatory update on development program for atuzaginstat in Alzheimer’s disease.

Cortexyme, Inc. received a letter from the FDA stating that a partial clinical hold has been placed on atuzaginstat (COR 388) impacting the open-label extension (OLE) phase of the company’s ongoing Phase II/III study, the GAIN Trial. Under the hold, no new participants will be enrolled in the OLE and currently enrolled OLE participants will be discontinued. Participants in the fully enrolled (N=643) double-blind, placebo-controlled randomized phase of the GAIN Trial will continue to receive study drug at their assigned dose, with top-line results from the double-blind GAIN Trial in Q4 2021. The partial clinical hold was initiated following the review of hepatic adverse events in the atuzaginstat trial by the FDA. These events have been reversible and without any known long-term adverse effects for the participants. Cortexyme will continue to collaborate with the FDA on the overall development program for atuzaginstat. The GAIN Trial is a randomized, double-blind, placebo-controlled Phase II/III trial that aims to recruit more than 570 participants at more than 90 sites in the United States and Europe. To participate in the study, subjects must be 55-80 years of age with a documented diagnosis of mild to moderate AD, among other criteria. Subjects will be randomized to one of two doses of COR 388 (twice daily 40mg or 80mg), or placebo. The primary endpoint for the study is mean change in the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-Cog 11) from baseline to 48 weeks. Secondary and exploratory endpoints include change in Alzheimer’s Disease Cooperative Study Group-Activities of Daily Living (ADCS-ADL), change in Clinical Dementia Rating-Sum of Boxes (CDR-SB), Winterlight Speech Assessment, cerebral spinal fluid biomarkers of infection, and other measures. -