On 26 October, Cortexyme, a clinical-stage pharmaceutical company developing novel treatments for altering the course of Alzheimer’s disease (AD) and other neurodegenerative disorders, reported top-line results from its Phase II/III GAIN Trial. This study is a randomised, double-blind and placebo-controlled study assessing the efficacy, safety and tolerability of two dose levels (40 and 80mg) of COR388 oral capsules in people with mild to moderate AD. COR388 is an oral small-molecule inhibitor of gingipains, which are proteins that are released by bacteria species (p.gingivalis) that have been identified in brain tissue and cerebral spinal fluid of people with AD.
643 participants were recruited in the study. Results showed that the study failed to meet its co-primary endpoints as measured by ADAS-Cog11 and ADCS-ADL at end of the treatment period (48 weeks) in the overall population. In a group of participants, representing up to half of the participants, with P. gingivalis detected in their saliva, the drug showed 57% slowing of cognitive decline as measured by ADAS-Cog11 in the 80mg group compared to placebo, but failed to show significant benefits on the ADCS-ADL co-primary outcome. The most common adverse events were gastrointestinal, such as diarrhea and nausea. Cortexyme will present further results from the GAIN trial at the upcoming 14th Clinical Trials on Alzheimer’s Disease (CTAD 2021) conference to be held on 9-12 November 2021.