First Participants Dosed in a Phase 1 Clinical Trial of COR388

The pharmaceutical company Cortexyme announced that the first group of participants has been dosed in the Phase 1 clinical trial of its lead compound, COR388, for the treatment of Alzheimer’s disease.

Because no new successful developments have emerged in Alzheimer’s therapeutics in the last 15 years, researchers have been trying to discover new and innovative ways to treat the disease.

“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 [disease development], to guide development of novel and safe therapeutics,” Samer Kaba, Cortexyme’s chief medical officer, said in a press release.

COR388 is a first-in-class bacterial protease inhibitor designed to target a specific, undisclosed bacteria that has been identified in both the brain tissue and cerebrospinal fluid of Alzheimer’s patients. Stephen Dominy, MD, Cortexyme’s co-founder and chief scientific officer, has discovered that Alzheimer’s symptoms and pathology are triggered by the presence of this particular pathogen.

Broad-spectrum antibiotics are unable to specifically target this bacteria, and the use of COR338 has been shown to rescue neurons from bacterial toxicity, which prevents further cognitive decline.

“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,” Kaba said.

Two studies are planned for the phase 1 clinical development program for COR388.

The first study, currently underway (NCT03331900), is a randomized, double-blind, placebo-controlled, single-ascending dose study that will enroll 40 healthy volunteers. The primary goals of this study are safety, tolerability, and pharmacokinetics.

The second study, which is set to begin soon, is a multiple ascending dose study in older volunteers and Alzheimer’s patients to determine the safety, tolerability, and pharmacokinetics of COR338. This study will enroll 36 participants who will be treated for up to 28 days. This study also will determine whether COR388 engages its intended target and leads to other progress in Alzheimer’s patients.

Cortexyme is a clinical-stage pharmaceutical company focused on the development of therapeutics for Alzheimer’s disease and other degenerative diseases. Cortexyme’s lead candidate, COR388, has moved from seed funding to a successful investigational new drug application in just over three years.