Cognition Therapeutics (CGTX) investor relations material

Mechanism of action and clinical rationale

• CT1812 targets toxic oligomers by binding to TMEM97 and PGRMC1 on neurons, causing a conformational change that reduces oligomer binding and restores synaptic health, as shown in multiple studies including SEQUEL.

• The drug differs from antibodies by not removing material from vessel walls, potentially impacting safety.

• Overlapping pathology between Alzheimer's and Lewy body dementia (DLB) supports CT1812's use in both, as many patients show both amyloid and alpha-synuclein abnormalities.

Current management and unmet needs in DLB

• DLB patients face delayed diagnosis, often requiring multiple specialists and over a year to confirm.

• Management is symptomatic, involving a mix of treatments for GI, neuropsychiatric, and movement symptoms, with significant limitations and risks from antipsychotics.

• No approved therapies exist for DLB, highlighting the need for effective disease-modifying options.

SHIMMER study in DLB and regulatory strategy

• SHIMMER showed benefits across neuropsychiatric, cognitive, movement, and fluctuation domains, with the strongest effects in neuropsychiatric symptoms.

• The company is preparing for a Type C meeting with the FDA in January to discuss endpoints and trial design, aiming for endpoints that best reflect efficacy and patient health.

• A composite endpoint is likely due to the drug's broad effects, and the company is collaborating with regulators to define the best approach.

• Parallel discussions are ongoing with European authorities for Alzheimer's, but only one set of trials will be run at a time.