Cerevance, a clinical-stage biopharmaceutical company, announced positive Phase 1 clinical trial results for CVN293, an investigational oral inhibitor targeting KCNK13 for the treatment of neurodegenerative diseases. The Phase 1 data showed that CVN293 demonstrated good central nervous system exposure and was generally well-tolerated, marking a significant milestone for this novel therapeutic approach.
Novel Mechanism Targets Neuroinflammation
CVN293 represents a highly selective oral inhibitor of the potassium two pore domain channel subfamily K member 13 (KCNK13). By selectively inhibiting KCNK13, which serves as a potentially novel regulator of NLRP3 inflammasome activation, CVN293 aims to reduce neuroinflammation and slow disease progression in a variety of neurodegenerative disorders. This mechanism may provide a disease-modifying approach for challenging central nervous system disorders.
The therapeutic potential extends beyond neurodegeneration, as modulation of KCNK13 may also have therapeutic benefit in obesity, expanding the potential applications of this novel target.
Proprietary Platform Drives Discovery
KCNK13 was identified through Cerevance's proprietary Nuclear Enriched Transcript Sort sequencing (NETSseq) platform, which enables the identification of subtle molecular changes that drive disease progression. The platform analyzes brain tissue from a diverse pool of over 20,000 donors, ranging in age from 8 to 104, and including both healthy individuals and those with various central nervous system disorders.
NETSseq allows researchers to identify targets that are expressed at very low levels, are present in rare cell types, or change over time as a disease progresses. This transformative tool provides deep insights into the molecular drivers of disease within specific cell populations, expanding understanding and treatment of neurodegenerative diseases.
Upcoming Scientific Presentations
Cerevance will present its findings at the Alzheimer's Association International Conference (AAIC) 2025, being held in Toronto from July 27-31, 2025. The company will deliver two poster presentations:
The first presentation, scheduled for Monday, July 28, 2025, will focus on "CVN293, an Investigational Inhibitor of KCNK13 Targeting NLRP3-Mediated Neuroinflammation in Neurodegenerative Disease: A Phase 1 Study to Investigate the Safety, Tolerability, and Pharmacokinetics."
The second presentation, scheduled for Wednesday, July 30, 2025, will explore "NETSseq unveils deep molecular insights into astrocyte function in Alzheimer's disease in the human brain," highlighting the proprietary platform's role in exploring the key potential role of astrocytes in Alzheimer's disease.
Broader Pipeline Development
CVN293 represents the third investigational treatment in Cerevance's pipeline focused on cell type-specific therapies for neurodegenerative and central nervous system-controlled metabolic disorders. The company's most advanced investigational treatment, solengepras, is currently in Phase 3 development and has the potential to be a first-in-class, oral non-dopaminergic therapy for both the motor and non-motor symptoms of Parkinson's disease.
The company's second investigational therapy, CVN766, is designed to be a highly selective oral antagonist of the orexin 1 receptor for the potential treatment of binge eating disorder and schizophrenia. CVN293 represents a potentially novel intervention point for neurodegenerative disorders and obesity, demonstrating the company's commitment to addressing multiple challenging therapeutic areas through innovative approaches.
