— GAIN Trial passes futility analysis and will continue to 1-year endpoint following the independent Data Monitoring Committee recommendation; topline results expected on time in December 2021 —
— Final study enrollment remains at 643; no sample size adjustment —
— Conference call and webcast today, Friday, December 4, 2020, at 8:30 a.m. EST / 5:30 a.m. PST —
Cortexyme, Inc. (Nasdaq: CRTX), a clinical stage biopharmaceutical company pioneering potential therapeutics for Alzheimer’s and other degenerative diseases, today announced that the independent Data Monitoring Committee (DMC) conducted a pre-planned interim analysis and recommended Cortexyme continue the Phase 2/3 GAIN Trial of atuzaginstat (COR388) as planned to the 1-year endpoint.
The interim analysis included approximately 300 patients who have reached 6 months of treatment in the GAIN Trial, a randomized, double-blind, placebo-controlled study of 40 and 80 mg BID of atuzaginstat. As part of the interim analysis, the DMC looked for futility, overwhelming efficacy (p< 0.005 on both co-primary outcomes), a sample size increase if needed to improve powering, as well as safety. Based on the interim analysis, topline data for the fully enrolled population of 643 subjects after the full 1-year treatment period is expected on time in December of 2021. The company remains blinded to all data.
Dr. Marwan Sabbagh, Director of the Cleveland Clinic Lou Ruvo Center for Brain Health and Principal Investigator of the GAIN trial said: “Given the tremendous unmet need in Alzheimer’s disease, it is imperative that we rapidly and efficiently study potential new treatments. We look forward to completing the study and sharing the results with the medical community and patients when the full trial results become available.”
“We are pleased with the DMC recommendation, which we believe supports the study design and statistical powering of the GAIN Trial. The totality of evidence around P. gingivalis and gingipain inhibition shows that blocking this upstream target may impact multiple aspects of disease progression and neurodegeneration,” said Michael Detke, M.D., Ph.D., Cortexyme’s Chief Medical Officer. “We are grateful to the DMC, the trial investigators, and the patients and caregivers for their participation in the trial as the study continues to its 1-year endpoint.”
Atuzaginstat targets the toxic proteases, or gingipains, produced by P. gingivalis, which have been discovered in greater than 90% of AD patients and shown to produce Alzheimer’s pathology and neurodegeneration in infected animals. P. gingivalis is best known as a keystone bacterium in the development of periodontal disease.
The GAIN (GingipAIN Inhibitor for Treatment of Alzheimer’s Disease) Trial is a Phase 2/3 trial evaluating the efficacy, safety, and tolerability of atuzaginstat (COR388), Cortexyme’s investigational gingipain inhibitor, in patients with mild to moderate Alzheimer’s disease. The co-primary endpoints for the GAIN trial are mean change in ADAS-Cog 11 and ADCS-ADL from baseline to 48 weeks versus placebo. Secondary and exploratory endpoints include change in CDR-SB, NPI, MMSE, Winterlight Speech Assessment, MRI volumetric measures and biomarkers of infection. The GAIN Trial also includes a sub-study measuring the efficacy of COR388 on symptoms of periodontal disease including gingival pocket depth. For more information on the trial, visit www.gaintrial.com.
Investor Conference Call and Webcast
Cortexyme management will discuss the interim analysis during a conference call beginning at 8:30 a.m. EST / 5:30 a.m. PST later today, Friday, December 4, 2020. To join the call, participants may dial (866) 221-1774 (domestic) or (270) 215-9925 (international) and provide the conference ID, 7778375. To listen to a live webcast of the conference call, visit the Investor Calendar page under the News & Events heading of the Cortexyme investor site (ir.cortexyme.com). The webcast will be archived at that location for one year.
An audio replay of the conference call will also be available by phone until 11:59 p.m. EST / 8:59 p.m. PST on Sunday, December 6, 2020. Access numbers for the phone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 7778375.
Cortexyme, Inc. (Nasdaq: CRTX) is a clinical stage biopharmaceutical company pioneering upstream therapeutic approaches designed to improve the lives of patients diagnosed with Alzheimer’s and other degenerative diseases. Based upon the evidence generated to date, Cortexyme is currently advancing its lead therapeutic candidate, atuzaginstat (COR388), in the GAIN Trial, an ongoing Phase 2/3 clinical trial in patients with mild to moderate Alzheimer’s disease. Cortexyme is targeting a specific, infectious pathogen found in the brain of Alzheimer’s patients and tied to neurodegeneration and neuroinflammation in animal models. To learn more about Cortexyme, visit www.cortexyme.com or follow @Cortexyme on Twitter.
Statements in this press release contain “forward-looking statements” that are subject to substantial risks and uncertainties. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “expect,” “believe,” “will,” “may,” “should,” “estimate,” “project,” “outlook,” “forecast” or other similar words. Examples of forward-looking statements include, among others, statements we make regarding our business plans and prospects, the timing and success of our clinical trials and related data, including the outcome and results of the GAIN trial, the potential of atuzaginstat to treat Alzheimer’s disease, the timing of announcements and updates relating to our clinical trials and related data, and the potential therapeutic benefits, safety and efficacy of our product candidate and/or library of compounds. Forward-looking statements are based on Cortexyme’s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict and could cause actual results to differ materially from what we expect. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled “Risk Factors” in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 16, 2020, our Quarterly Report on Form 10-Q filed with the SEC on November 12, 2020, and other reports as filed with the SEC. Forward-looking statements contained in this press release are made as of this date, and Cortexyme undertakes no duty to update such information except as required under applicable law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201204005235/en/
Chief Financial Officer
For Cortexyme, Inc.