— GAIN Trial enrollment complete with 643 participants
— GAIN Trial interim analysis on schedule to complete in December 2020
Cortexyme, Inc. (Nasdaq: CRTX), a clinical-stage biopharmaceutical company pioneering potential therapeutics for Alzheimer’s and other degenerative diseases, today announced financial results for the third quarter 2020 and provided an update on its business.
"We are pleased with the strong participation in the GAIN Trial and deeply appreciate the support and dedication of patients and the medical community as we approach the interim analysis before the end of this year," said Casey Lynch, Cortexyme’s chief executive officer, co-founder, and chair. "With a strong balance sheet and a talented team, we remain confident that Cortexyme is well positioned to advance new therapeutic options for patients with Alzheimer’s and other degenerative diseases."
GAIN Trial Updates: Evaluating Atuzaginstat, a New Potential Therapy for Alzheimer’s Disease
The Phase 2/3 GAIN Trial of atuzaginstat (COR388) in mild to moderate Alzheimer’s disease (AD) enrolled on time with final enrollment of 643 participants. Target enrollment was exceeded by approximately 12% in light of uncertainties which may be created by the global pandemic. Strong demand allowed the incremental enrollment to occur within projected timelines.
Cortexyme remains on track to conduct an interim analysis in the GAIN Trial in December 2020. Management will remain blinded to the interim analysis, which is being conducted by an independent Data Monitoring Committee (DMC). This interim analysis will be conducted after approximately 100 participants in each of the GAIN Trial’s three arms reach 24 weeks of treatment. The co-primary endpoints for the GAIN Trial’s interim analysis are change from baseline in ADAS-Cog11 and CDR-SB versus placebo. The four possible recommendations from the DMC after the interim analysis, and the only information that will be conveyed to management, are:
End the study early for overwhelming efficacy on either dose of atuzaginstat vs. placebo on the co-primary endpoints p< 0.005
Continue as planned
Increase sample size by up to 100 participants/arm based on favorable trends
End the study early for overwhelming futility, placebo vs. atuzaginstat p<0.05
A research abstract on the design of the GAIN Trial and its baseline biomarkers was the subject of an oral presentation at the 13th Clinical Trials on Alzheimer’s Disease (CTAD) Conference on November 5, 2020. The presentation, "Phase 2/3 GAIN trial of atuzaginstat (COR388), a novel bacterial virulence factor inhibitor for the treatment of Alzheimer’s disease: Update and baseline data," highlights key biomarker data for the first 40-50% of participants in the GAIN Trial. All patient samples analyzed to date had evidence of P. gingivalis IgG in serum at baseline indicating immune response to systemic P. gingivalis infection, with 72% showing very high levels associated with higher infection and more severe periodontal disease. Additionally, the vast majority of subjects showed CSF biomarkers consistent with recently defined cutoffs for Alzheimer’s disease including amyloid β (Aβ) 42/40 ratio, tau and p-tau. As expected, 65% of the study participants are ApoE4 carriers who have been stratified across the three treatment groups. These data further reinforce the gingipain hypothesis and the design of the GAIN Trial to enroll the appropriate population for testing atuzaginstat.
Scientific Updates: Generating New Evidence and Expanding Our Pipeline
As atuzaginstat advances through late-stage clinical development, Cortexyme and external collaborators continue to present and publish new research and study data to advance the gingipain hypothesis for Alzheimer’s pathogenesis and identify additional development opportunities. Recent scientific presentations and research accomplishments are the following.
Cortexyme scientists presented new data in a poster at the Society for Immunotherapy of Cancer’s (SITC) 35th Anniversary Annual Meeting on November 9, 2020 entitled "PD-L1 is induced by the periodontal pathogen Porphyromonas gingivalis and can be blocked by small molecule gingipain inhibitors, including atuzaginstat." P. gingivalis has been linked to oral, esophageal, gastrointestinal, and pancreatic cancer and there is accumulating evidence that bacterial presence is correlated with worse disease prognosis. This study demonstrated that PD-L1 expression is increased by P. gingivalis infection in an esophageal cell line and this induction is blocked by either the lysine gingipain inhibitor atuzaginstat or an arginine gingipain inhibitor COR613. Infection resulted in nuclear β-catenin and disruption of the Wnt pathway complex regulating β-catenin function, and this is also blocked by gingipain inhibition. Tumor immune evasion markers PD-L1, PD-L2, and CTLA4 ligand CD80 were also induced by Pg infection on primed M2 macrophages, further supporting a role for Pg infection in blocking functional tumor immune surveillance.
COR588, a novel lysine gingipain inhibitor from Cortexyme’s library, remains on track with IND-enabling studies. Clinical studies are expected to begin in Q3 2021.
Cortexyme completed additional screening of its proprietary library of small molecules for a possible treatment for coronaviruses. The Company has identified inhibitors of the 3CL protease of SARS-CoV-2 and other coronaviruses in its library of small molecules which block viral replication in cells. Cortexyme is now proceeding to in vivo efficacy and toxicology testing.
Financial Results for the Quarter Ended September 30, 2020
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents, and short and long-term marketable securities as of September 30, 2020, were $197.9 million, and includes approximately $117.6 million of net proceeds raised in Cortexyme’s private placement offering completed in February 2020. Cortexyme expects current cash, cash equivalents and marketable securities will be sufficient to fund its operating and capital expenditures through 2022 and the completion of the GAIN Trial.
Research and Development (R&D) Expenses: For the quarter ended September 30, 2020, R&D expenses were $17.0 million, primarily due to costs related to the research and development of atuzaginstat and the GAIN Trial.
General and Administrative (G&A) Expenses: For the quarter ended September 30, 2020, G&A expenses were $4.9 million. The expense was primarily attributable to personnel-related expenses, insurance, professional and legal fees, and stock-based compensation.
Net Loss: For the quarter ended September 30, 2020, net loss was $21.5 million, or a loss of $0.73 per basic share. Weighted average shares outstanding for the quarter ended September 30, 2020 was 29,488,739.
About Cortexyme, Inc.
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 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 translation to human of pre-clinical data, the pre-clinical results for our product candidates, the timing and success of our clinical trials and related data including the outcome of the interim analysis, the potential of atuzaginstat to treat Alzheimer’s disease and the potential therapeutic application in oncology, our ability to fund planned operating and capital expenditures, the timing of announcements and updates relating to our clinical trials and related data, the timing of and our ability to enroll patients into our clinical trials, and the potential therapeutic benefits, safety and efficacy of our product candidate 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.
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