# A Randomized, Double-blind, Placebo-Controlled, Parallel-Group, Phase II Study to Evaluate the Safety and Efficacy of CT1812 in Subjects with Mild to Moderate Alzheimerʼs Disease

> **NIH NIH R01** · COGNITION THERAPEUTICS, INC. · 2021 · $13,634,548

## Abstract

ABSTRACT. Soluble oligomers of beta amyloid (Aβ) protein are the most potent neuroactive structural form of
this protein and evidence suggests they cause the synaptotoxic changes resulting in cognitive decline in
Alzheimer's disease (AD) (Selkoe and Hardy 2016, Viola and Klein 2015). Cognition Therapeutics, Inc.
(CogRx) is developing CT1812, the first brain penetrant small molecule that selectively clears toxic oligomers
from the brain into the cerebrospinal fluid (CSF). This drug candidate displaces AβOs bound to neuronal
receptors at synapses and protects synapses from toxic oligomer effects. When administered once daily for 28
days to mild to moderate AD patients, CT1812 significantly reduces concentrations of synaptic degeneration
markers in AD patient CSF, and phospho-tau. CT1812's mechanism of action can potentially halt or slow
cognitive decline, significantly alleviating the suffering of AD patients. An ongoing study COG0201 (SHINE)
funded by the NIA under the award AG058660 is evaluating the safety and efficacy of two doses of CT1812 on
cognitive function in a Phase 2 randomized, double-blind, placebo-controlled parallel group clinical trial in mild
to moderate AD patients. This important proof of concept trial will be critical for CT1812's continued
development. Excellent progress has been made on this study and award. Study COG0201 is proceeding well
at nine clinical research sites across the US and Australia. Regulatory interactions have resulted in changes to
the study design which have increased per patient costs; this competing revision application requests
additional funds to enroll sufficient patients and reach the appropriate number of patients per group and
adequately measure changes in cognitive function (N=48/group) and complete a fully powered trial (revised
AIM 1). Adequately powered assessment of clinical outcomes is vital to CT1812's further development.
Completion of this study in AD patients will inform the design and methods of the subsequent disease
modification Phase III trials with CT1812. Advancement of CT1812 clinical development would substantially
improve the lives of the 50 million people worldwide suffering from AD, for whom no disease-modifying
pharmacological treatments exist.

## Key facts

- **NIH application ID:** 10208480
- **Project number:** 3R01AG058660-03S1A1
- **Recipient organization:** COGNITION THERAPEUTICS, INC.
- **Principal Investigator:** ANTHONY O CAGGIANO
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $13,634,548
- **Award type:** 3
- **Project period:** 2018-09-15 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10208480

## Citation

> US National Institutes of Health, RePORTER application 10208480, A Randomized, Double-blind, Placebo-Controlled, Parallel-Group, Phase II Study to Evaluate the Safety and Efficacy of CT1812 in Subjects with Mild to Moderate Alzheimerʼs Disease (3R01AG058660-03S1A1). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10208480. Licensed CC0.

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