Clinical Update on an Anti-Alzheimer Drug Candidate CT1812: A Sigma-2 Receptor Antagonist.

Purpose: This review article summarizes the progress and latest findings related to the investigational drug candidate CT1812, which is currently in phase 2 clinical trials for Alzheimer's disease (AD). The article outlines the development of this promising molecule and provides insights into its mechanism of action as sigma-2 receptor (S2R) antagonist along with the positive outcomes of various clinical trials. Literature mentioning AD therapeutics that specifically target amyloid-beta (Aβ) oligomers is limited even though these oligomers are established as the most neurotoxic forms of the Aβ protein. This timely article highlights the potential of CT1812 as a breakthrough in AD therapeutics, providing a new avenue for addressing the neurotoxic forms of Aβ and advancing the field toward a potential cure for AD.

Methods: The literature includes articles searched from PubMed and Google Scholar along with a comprehensive discussion of all the clinical research trials undertaken for CT1812. The review includes 12 clinical trials; of the total citations identified, 10 have been used to support the results of published trials.

Findings: The positive outcomes in the multiple clinical trials conducted on CT1812 indicate the emergence of an effective and promising drug candidate for AD. The article mentions a gap in the literature regarding AD therapeutics specifically targeting Aβ oligomers, which reveals lack of established treatments addressing Aβ oligomers, making the novel approach of CT1812 noteworthy.

Implications: Clinical treatments available today provide symptomatic relief, however, any drug providing a potential cure for AD remains an unanswered question. S2Rs mediated oligomer binding in addition to synaptic toxicity suggest the potential usefulness of CT1812 in AD treatment. Efficacy and safety of CT1812 in further clinical trials could represent a significant advancement in the field, offering a potential treatment that goes beyond the symptomatic relief and aimed at addressing the core mechanisms associated with AD.