Cortexyme Announces Phase 2/3 Clinical Trial Based on Transformational Understanding of Alzheimer’s Disease Now Open for Enrollment

The GAIN Trial will assess whether a new investigational medicine
targeting P. gingivalis bacteria can slow or halt progression of
Alzheimer’s disease

Researchers seek more than 500 participants in the United States and
Europe; visit www.GAINtrial.com
for information

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Cortexyme, Inc. today announced the launch of the GAIN Trial, an
international Phase 2/3 clinical study of the company’s lead
investigational medicine, COR388, in subjects with mild to moderate
Alzheimer’s disease (AD). A first wave of U.S. trial sites are now
screening patients for participation, and additional trial centers in
the United States and Europe are expected to be open for enrollment in
the coming months. Potential participants and caregivers can learn more
about the study by visiting the trial website at www.GAINtrial.com.

The GAIN (GingipAIN Inhibitor for Treatment of Alzheimer’s Disease)
Trial is based on a growing body of scientific evidence that the
pathogenic bacterium Porphyromonas gingivalis, or P. gingivalis,
most commonly associated with chronic periodontal disease, can infect
the brain and cause AD. The trial is evaluating whether COR388, an oral
investigational medicine, can slow or halt the progression of AD by
inactivating the toxic proteins, or gingipains, released by P.
gingivalis that have been shown in animal models to damage and
destroy brain cells.

“The GAIN Trial is the first large, international clinical trial to test
a dramatically new way of understanding and treating Alzheimer’s
disease,” said Marwan Sabbagh, M.D., the director of the Cleveland
Clinic Lou Ruvo Center for Brain Health, one of the clinical research
centers for the study. “For decades, a significant portion of the
Alzheimer’s research community has focused on approaches related to
amyloid plaques found in the brains of patients with the disease. In the
GAIN Trial, we are evaluating a new and different mechanism of action
against Alzheimer’s. We hope to confirm the hypothesis that bacteria are
an ‘upstream’ trigger for Alzheimer’s, and that inactivating the toxic
proteins released by the bacteria can have a positive impact on stopping
or slowing the progression of this devastating disease.”

COR388 is a first-in-class, orally administered virulence factor
inhibitor that targets P. gingivalis gingipains. In a paper
published in Science Advances earlier this year, a team of
researchers, including Cortexyme’s chief scientific officer, Dr. Stephen
Dominy, M.D., detailed preclinical data on COR388’s ability to reduce
the bacterial load of a P. gingivalis infection, block production
of amyloid beta, reduce neuroinflammation, and protect neurons in the
hippocampus, a part of the brain that mediates memory. In late 2018,
Cortexyme presented encouraging
results from a Phase 1b clinical trial showing COR388 was
well-tolerated, and identifying positive trends across several
biomarkers and cognitive tests in patients with AD.

The GAIN Trial is a randomized, double-blind, placebo-controlled Phase
2/3 trial that aims to recruit more than 570 participants at more than
90 sites in the United States and Europe. To participate in the study,
subjects must be 55-80 years of age with a documented diagnosis of mild
to moderate AD, among other criteria. Subjects will be randomized to one
of two doses of COR388 (twice daily 40mg or 80mg), or placebo. The
primary endpoint for the study is mean change in the Alzheimer’s Disease
Assessment Scale-Cognitive Subscale 11 (ADAS-Cog 11) from baseline to 48
weeks. Secondary and exploratory endpoints include change in Alzheimer’s
Disease Cooperative Study Group-Activities of Daily Living (ADCS-ADL),
change in Clinical Dementia Rating-Sum of Boxes (CDR-SB), Winterlight
Speech Assessment, cerebral spinal fluid biomarkers of infection, and
other measures.

“The start of the GAIN Trial is an important milestone as we work to
bring new hope and options to patients with Alzheimer’s,” said Michael
Detke, M.D., Ph.D., Cortexyme’s chief medical officer. “The clinical
rationale for the study, and the data that support a bacterial driver of
Alzheimer’s disease, are compelling. We look forward to enrolling
subjects and learning more about the potential for COR388 to benefit the
Alzheimer’s patient community, which now numbers more than 5.7 million
people in the United States and 30 million individuals worldwide.”

About Cortexyme, Inc.

Cortexyme is a clinical-stage pharmaceutical company pioneering a novel
disease-modifying therapeutic approach to treat a key underlying cause
of Alzheimer’s disease and other degenerative diseases. Cortexyme is
targeting a specific, infectious pathogen found in the brain of
Alzheimer’s patients and tied to neurodegeneration and neuroinflammation
in animal models. The company’s lead investigational medicine, COR388,
is the subject of the GAIN Trial, an ongoing Phase 2/3 clinical study in
patients with mild to moderate Alzheimer’s. More information about the
trial can be found at www.GAINtrial.com.
To learn more about Cortexyme, visit www.cortexyme.com.

Contacts

Hal Mackins
For Cortexyme, Inc.
[email protected]
(415)
994-0040