COR388 is a first-in-class bacterial protease inhibitor designed to target a specific 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, which has not been disclosed.
Broad-spectrum antibiotics are unable to target this bacteria, and the use of COR338 was seen in early testing to rescue neurons from bacterial toxicity, potentially preventing further cognitive decline in patients.
Cortexyme announced the successful completion of its placebo-controlled, single-ascending dose Phase 1 trial (NCT03331900) in 34 healthy volunteers. The trial was the first of COR388’s clinical development plan and was designed to assess its safety, tolerability, and pharmacokinetics (the movement of drug into, through, and out of the body; essentially how the body affects a drug).
COR388 was found to be safe and well-tolerated by volunteers, ages 20 to 70. The small molecule produced a favorable pharmacokinetic profile and tissue distribution when given orally in different doses.
The company plans to open a Phase 2 proof of efficacy study in Alzheimer’s patients in 2019.
Cortexyme is also currently recruiting eight older healthy adults (ages 55 to 80) and 12 Alzheimer’s patients for its second Phase 1 trial (NCT03418688), evaluating the safety, tolerability, and pharmacokinetics of multiple and ascending doses of oral COR388 given for up to 28 days.
The trial — enrolling at sites in California, Florida and Ohio — will also assess how well COR388 engages its target and effects changes in Alzheimer’s patients.
The company also announced that it has completed a $76 million Series B financing round, which included investors such as Pfizer, Takeda Ventures, and Sequoia Capital.
“Cortexyme is glad to count among its supporters some of the world’s most successful investors in innovation, both in the pharmaceutical industry and beyond,” Casey Lynch, co-founder and chief executive officer of Cortexyme, said in a press release.
“Our streamlined, efficient approach to drug development allowed us to move from seed funding to phase 1 data in less than four years,” Lynch added. “We’re committed to continuing to move swiftly through phase 2 proof of efficacy studies in … patients suffering from Alzheimer’s and related conditions.”