FDA Clears Cognition Therapeutics’ IND Application for Geographic Atrophy

Cognition Therapeutics announced that its Investigational New Drug (IND) application has been cleared by the FDA for the investigation of CT1812 in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD).

According to the announcement, the Phase 2 MAGNIFY trial of CT1812, an oral drug candidate, is expected to be initiated by Cognition in 2023 for individuals with dry AMD who have measurable GA.

“We enjoyed a collaborative dialogue with the ophthalmology division of the FDA during our pre-IND meeting and are excited to assess the potential of CT1812 to impact GA and slow the decline in visual acuity in people with dry AMD,” said Anthony Caggiano, M.D., Ph.D., Cognition’s chief medical officer and head of R&D. “We believe that CT1812's oral systemic delivery combined with its potential to protect the retinal pigment epithelium (RPE) from damage in both the affected and fellow eyes, may represent a significant advantage to the millions of people with dry AMD who are at risk for permanent vision loss.”

The MAGNIFY study (COG2201) is a phase 2 trial that will be randomized and placebo-controlled. The trial is expected to enroll around 246 individuals who have been diagnosed with dry age-related macular degeneration (dry AMD) and have measurable geographic atrophy (GA). During the treatment period, various measures will be assessed, including changes in GA lesion size and best-corrected visual acuity, to determine if treatment can slow the loss of vision. Additionally, safety and efficacy will be evaluated.

“Expansion of our pipeline programs into GA is an excellent example of how we are leveraging our scientific expertise to tackle some of the most challenging diseases,” added Lisa Ricciardi, president and CEO of Cognition. “Looking ahead, I’m excited by our continued clinical progress as we advance CT1812 through ongoing studies and now into this new indication.”

About CT1812

CT1812 is a small molecule delivered orally that is still in its experimental stage. The molecule has been designed to penetrate both the blood-brain and blood-retina barriers and has selective binding properties to the sigma-2 (σ-2) receptor complex. This complex plays a vital role in regulating cellular processes like membrane trafficking and autophagy that can be damaged by toxic interactions with beta-amyloid (Aβ) oligomers, oxidative stress, and other stressors. Damage to sensitive cells such as RPE cells and neurons in the central nervous system (CNS) can result in a loss of function, leading to a loss of visual acuity and eventual vision loss in dry age-related macular degeneration (dry AMD).

Early proof-of-concept studies with CT1812 indicate a role of σ-2 receptor modulators in rescuing sensitive RPE cells from damage by stressors such as pathogenic proteins and oxidative stress, thus preserving this crucial component of the macula.

In addition to Cognitions’ new Phase 2 MAGNIFY clinical trial, CT1812 is being studied in three ongoing Phase 2 clinical trials: the SEQUEL study, which recently concluded enrollment of adults with mild-to-moderate Alzheimer’s disease; the SHINE study in individuals with mild-to-moderate Alzheimer’s disease; and the SHIMMER study in individuals with dementia with Lewy bodies. CT1812 has not been approved by the U.S. FDA or other regulatory agency.