Cortexyme, Inc., a clinical-stage pharmaceutical company focused on Alzheimer’s and other degenerative diseases, today announced the first cohort has been dosed in the initial trial of its phase 1 clinical development program for COR388, the company’s lead compound. COR388 is a first-in-class bacterial protease inhibitor that targets a pathogen identified in brain tissue and cerebral spinal fluid of patients with Alzheimer’s disease (AD). It represents a promising new approach to addressing a disease estimated to affect more than 5.4 million Americans.
“Alzheimer’s disease impacts millions of patients and their caregivers, and puts a huge burden on healthcare systems worldwide. The failure of so many large clinical trials targeting amyloid beta over the last decade illustrates the urgent need for new therapeutic targets, generated by scientifically sound hypotheses of Alzheimer’s pathogenesis, to guide development of novel and safe therapeutics,” said Samer Kaba, M.D., Cortexyme’s chief medical officer. “COR388 represents a completely new paradigm for the treatment of AD. Cortexyme’s ongoing phase 1 clinical development program is an important step in evaluating this investigational therapy that addresses an underlying disease driver.”
COR388 is the first compound developed by Cortexyme to advance into clinical trials. The company’s drug discovery and development efforts are based on wide-ranging scientific evidence demonstrating Alzheimer’s symptoms and pathology are triggered by a specific pathogen discovered in the brains of patients with the disease by Cortexyme’s co-founder and chief scientific officer, Stephen Dominy, M.D. COR388 is designed to inhibit this pathogen in a way that broad spectrum antibiotics cannot, rescuing neurons from bacterial toxicity and preventing further cognitive decline and dysfunction.
Sponsored by Cortexyme, the new phase 1 trial is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and pharmacokinetics of a single dose of COR388. This single ascending dose study will enroll 40 subjects into five cohorts of healthy volunteers. The phase 1 clinical development program for COR388 also includes a multiple ascending dose study in older healthy volunteers and AD patients that is planned to start shortly. This second study will enroll 36 subjects in four cohorts and will assess the safety, tolerability, and pharmacokinetics of COR388 administered in older populations for up to 28 days. In addition, this study will evaluate target engagement biomarkers and other exploratory pharmacodynamic measures in its AD cohort.
“COR388’s advancement into the clinic is an important milestone for Cortexyme as we develop therapies for degenerative diseases that pose major challenges for patients, families, and healthcare systems at large,” said Casey Lynch, chief executive officer and co-founder of Cortexyme. “Moving from seed funding to successful Investigational New Drug application in just over three years underscores our commitment to effective, streamlined development intended to speed new therapies to the patients who need them.”
Based in South San Francisco, California, Cortexyme is a privately held, clinical-stage pharmaceutical company developing therapeutics to alter the course of Alzheimer’s and other degenerative disorders. Cortexyme is targeting a specific, undisclosed infectious pathogen tied to neurodegeneration and chronic inflammation in humans and animal models. The company’s lead compound, COR388, is the subject of an ongoing phase 1 clinical trial; additional proprietary small molecules are moving forward in preclinical development. Cortexyme’s investors include Pfizer, Takeda Ventures, Lamond Family, Dolby Family Ventures, Breakout Ventures, and Breakout Labs. For more information on Cortexyme, visit www.cortexyme.com.