Wednesday, September 20, 2023
Cerevance, a privately held clinical-stage biopharmaceutical company dedicated to the advancement of innovative precision therapeutics for central nervous system (CNS) disorders through its proprietary Nuclear Enriched Transcript Sort sequencing (NETSseq) platform, has announced the initiation of a Phase 1 clinical trial to evaluate the safety, tolerability, and pharmacokinetics of CVN293.
Craig Thompson, CEO of Cerevance, expressed enthusiasm about advancing CVN293, a selective small-molecule inhibitor targeting the novel KCNK13, into Phase 1 clinical development. KCNK13 was identified as a selective modulator of neuroinflammation using NETSseq, showcasing the potential of Cerevance's platform in identifying precise neuroscience targets. Thompson emphasized that this milestone underscores the company's commitment to translating scientific discoveries into potential therapies for neuroinflammatory conditions.
The Phase 1 trial encompasses both a single ascending dose (SAD) and multiple ascending dose (MAD) study. Its objective is to evaluate the safety, tolerability, and pharmacokinetics of orally administered CVN293 in healthy participants. During the SAD phase, subjects will receive single doses, while the MAD phase involves repeated doses of CVN293. The study follows a double-blind, randomized, placebo-controlled, dose-escalation design, enrolling a total of 64 healthy male and female participants aged 18 to 55 years, with 40 subjects participating in the SAD study and 24 subjects in the MAD study. Each cohort will consist of 8 subjects, randomized at a 6:2 ratio to receive either CVN293 or a placebo, respectively.
CVN293 is an investigational and highly selective inhibitor of KCNK13, a target identified by NETSseq as predominantly expressed in brain microglia. Brain microglia play a central role in neuroinflammation and have implications in various neurodegenerative diseases, including Amyotrophic Lateral Sclerosis, Alzheimer's disease, and severe age-related macular degeneration. The low expression levels of KCNK13 in peripheral macrophages, in contrast to other pursued targets, suggest that CVN293 may effectively address brain inflammation without compromising peripheral immune function, a critical consideration for aging patients with fragile immune systems. The potential benefits of CVN293's selective action hold promise for a precise neuroscience approach to treating neuroinflammation.