Next generation lysine gingipain inhibitor COR588 with novel structure and improved pharmacologic and safety profile prioritized for Alzheimer’s development

Additional pipeline includes therapeutics in development for coronavirus infection, periodontal disease, and prevention of oral squamous cell carcinoma

Preliminary, unaudited 2021 year-end cash, cash equivalents, and investments of $126.7 million allows for strategic flexibility

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Feb. 1, 2022-- Cortexyme, Inc. (Nasdaq: CRTX), a clinical-stage biopharmaceutical company pioneering upstream therapeutic approaches to improve the lives of patients diagnosed with degenerative diseases, today provided a business update and announced key milestones for 2022.

Christopher Lowe, Cortexyme’s interim chief executive officer, chief operating officer, and chief financial officer, said, “Cortexyme is committed to following the evidence as we identify the most efficient path to expand our pipeline through both internal and external development. We believe that there are several promising paths to meaningfully advance our pipeline forward. This includes targeting the expansion of our pipeline in several therapeutic areas, including leveraging our proprietary protease inhibitor expertise for periodontal disease, a new indication targeted at preventing the development of oral squamous cell carcinoma where P. gingivalis infection plays an integral role, and coronavirus infection. With $126.7 million in cash on hand as of the end of 2021, Cortexyme is well-positioned to leverage our proprietary science and pioneering pipeline through both internal and external opportunities.”

Anticipated Milestones for 2022

COR588 in Mild to Moderate Alzheimer’s Disease

• COR588 possesses novel compound properties designed for potency and safety with an improved pharmacologic profile, including once daily oral administration.
• COR588 has a higher safety margin compared to its predecessor COR388 in preclinical toxicology studies to date.
• COR588 is currently completing a Phase 1 SAD/MAD trial in a cohort of healthy participants in Australia with study results expected in the second quarter of 2022.
• Following the Phase 1 study, and subject to continued interactions with FDA, Cortexyme plans to advance COR588 to a Phase 2 trial to assess dose ranging, biomarker response, and further evaluate the safety profile.

Atuzaginstat (COR388) in Oncology for Prevention of Oral Squamous Cell Carcinoma

• Cortexyme is announcing a potential new indication for its lysine gingipain inhibitor, atuzaginstat (COR388), to prevent the development of oral/head and neck squamous cell carcinoma (O/HNSCC). P. gingivalis infection is associated with the development of O/HNSCC, and also with a significantly worse overall survival in patients with P. gingivalis positive O/HNSCC. Most cases of O/HNSCC are preceded by high-risk oral potentially malignant diseases (OPMD), including oral pre-malignant dysplasia, proliferative verrucous leukoplakia (PVL), and carcinoma-in-situ.
• Non-clinical studies and peer-reviewed literature support the testing of atuzaginstat as a therapy for P. gingivalis positive, high-risk OPMDs and could reduce the incidence and progression to oral squamous cell carcinoma based upon the mode of action and the evidence demonstrating the benefits of atuzaginstat on P. gingivalis-driven tumor growth and PDL1 expression.
• Cortexyme will be sharing a comprehensive update of evidence generated to date. The company held a pre-IND meeting with FDA and plans to submit an IND to the Division of Oncology in the first quarter of 2022.

Atuzaginstat (COR388) in Mild to Moderate Alzheimer’s Disease

• Cortexyme intends to present additional analysis and insights from its extensive GAIN Trial data set at upcoming scientific meetings, including AD/PD 2022, the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, a hybrid event taking place March 15-20, 2022, in Barcelona, Spain.
• The company will provide additional updates on atuzaginstat, as appropriate, pending continued engagement with FDA.
• Cortexyme believes that top-line results from its Phase 2/3 GAIN Trial of atuzaginstat established proof of concept for the ability of its lysine gingipain inhibitor to successfully target and inhibit the infectious bacteria Porphyromonas gingivalis (P. gingivalis).
• The GAIN Trial also demonstrated the relationship between the reduction of P. gingivalis infection and the slowing of cognitive decline in Alzheimer’s disease with atuzaginstat treatment in a prespecified population of patients based on diagnosis of infection.
• Cortexyme intends to utilize the robust data set from the GAIN Trial to inform the ongoing development of its next generation lysine gingipain inhibitor, COR588, for Alzheimer’s disease, in addition to developing atuzaginstat for non-Alzheimer’s indications, such as periodontal disease and select oncology indications where P. gingivalis-driven infection contributes to disease progression.

Atuzaginstat (COR388) in Periodontal Disease

• Cortexyme intends to develop an oral formulation of its proprietary gingipain inhibitors, including atuzaginstat, to address periodontal disease in a non-systemic way that engages and inhibits gingipains from P. gingivalis, penetrating and disrupting biofilms directly in the oral cavity.
• The company believes this type of oral formulation can compare favorably to traditional antibiotics through their ability to penetrate biofilms at therapeutic concentrations and address their lack of demonstrated efficacy in treating chronic periodontal disease.

COR803 in Coronavirus Infection

• COR803 is a novel patent-pending small molecule 3CLpro inhibitor discovered and developed by Cortexyme based on its expertise in cysteine protease inhibition. 3CLpro, or Mpro, is a validated antiviral drug target shown to be essential in viral replication of SARS-CoV-2.
• COR803 is differentiated from other COVID-19 therapeutics with its covalent irreversible target binding resulting in high antiviral potency, with selectivity, and systemic exposure utilizing oral, intranasal, or subcutaneous administration – enabling potential clinical utility in multiple settings, including outpatient and inpatient use.
• The target of COR803 is highly conserved across coronavirus strains observed to date and, therefore, is expected to broadly address both current and future coronavirus infection.
• Cortexyme is progressing IND-enabling preclinical studies for COR803.

Cortexyme has not completed preparation of its financial statements for the fourth quarter or full year of 2021. The cash, cash equivalents and investments presented as of December 31, 2021 are preliminary and unaudited and are thus inherently uncertain and subject to change as the company completes its financial results for 2021. Cortexyme is in the process of completing the company’s customary year-end close and review procedures as of and for the year ended December 31, 2021, and there can be no assurance that its final results for this period will not differ from these preliminary, unaudited amounts. The company’s independent registered public accounting firm has not audited, reviewed, compiled, or performed any procedures with respect to such preliminary data for the fourth quarter and year ended December 31, 2021.

About Cortexyme

Cortexyme, Inc. (Nasdaq: CRTX) is a clinical stage biopharmaceutical company pioneering upstream therapeutic approaches designed to improve the lives of patients diagnosed with degenerative diseases. Cortexyme’s innovative approach targets a specific, infectious pathogen called P. gingivalis found in the brain of Alzheimer’s patients and other organs and tied to degeneration and inflammation in humans and animal models. The company’s causation evidence for Alzheimer’s disease and the mechanism of its novel therapeutic has been independently replicated and confirmed by multiple laboratories around the world, as well as published in peer-reviewed scientific journals. To learn more about Cortexyme, visit www.cortexyme.com or follow @Cortexyme on Twitter.

Forward-Looking Statements

Statements in this news release contain “forward-looking statements” that are subject to substantial risks and uncertainties. Forward-looking statements contained in this news release may be identified by the use of words such as “anticipate,” “expect,” “believe,” “plans,” “intends,” “will,” “may,” “should,” “estimate,” “project,” “outlook,” “runway,” “forecast,” “potential” or other similar words. Examples of forward-looking statements include, among others, the strategic development path for atuzaginstat, including with respect to COR388, COR588, and other programs and indications, including with respect to COR803; its business plans, internal and external development of the pipeline, strategy, planned FDA submissions and clinical trials and timeline, prospects, and milestone expectations; cash position and the expected cash runway; the timing and success of the company’s clinical trials and related data, including plans and the ability to initiate, conduct and/or complete current and additional studies; the potential of atuzaginstat to treat Alzheimer’s disease and other indications; the timing of announcements and updates relating to its clinical trials and related data; the potential therapeutic benefits, safety and efficacy of the company’s product candidate or library of compounds; and statements about its ability to obtain, and the timing relating to, further development of atuzaginstat and other programs or indications, regulatory submissions and interactions with regulators, and related response and decisions, and approvals with respect to the company’s drug product candidate. 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 the company expects. 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 Cortexyme’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 1, 2021, its Quarterly Report on Form 10-Q filed with the SEC on October 29, 2021, and other reports as filed with the SEC. Forward-looking statements contained in this news 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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Cortexyme Contact:
Stacy Roughan
Cortexyme, Inc.
Vice President, Corporate Communications & Investor Relations
ir@cortexyme.com

Source: Cortexyme, Inc.