Cerevance Presents Positive Results from Phase 1 Study of CVN293 at American Academy of Neurology 2025 Annual Meeting
Rhea-AI Summary
Cerevance announced positive Phase 1 trial results for CVN293, a novel KCNK13 inhibitor targeting neuroinflammation, at the American Academy of Neurology 2025 Annual Meeting. The randomized, double-blind, placebo-controlled study involved 72 healthy adults, evaluating single doses up to 1,000 mg and multiple doses up to 750 mg over 14 days.
Key findings showed that CVN293 was well-tolerated with:
- 100% participant completion rate
- No severe or dose-limiting adverse events
- Only mild treatment-related effects
- High levels of central nervous system exposure
- Generally dose-proportional plasma exposure
The company also presented data on their NETSseq platform and solengepras, a non-dopaminergic therapy in development for Parkinson's disease, including results from the Phase 2 ASCEND study in early, untreated Parkinson's patients.
Positive
- Successful completion of Phase 1 trial with 100% participant retention
- Strong safety profile with no severe adverse events
- Demonstrated robust brain penetration
- Dose-proportional plasma exposure achieved
- Advanced pipeline with multiple clinical-stage candidates (CVN293 and solengepras)
Negative
- Early-stage development - still far from market approval
- data on actual therapeutic efficacy
Insights
Cerevance's positive Phase 1 results for CVN293 represent an important early development milestone that de-risks this novel KCNK13 inhibitor program. The clean safety profile—with no severe adverse events, treatment discontinuations, or clinically meaningful changes in vital signs—provides a strong foundation for further development in neurodegenerative disorders.
Two critical findings make these results particularly significant for a CNS-targeting compound: First, the robust brain penetration demonstrated through cerebrospinal fluid sampling addresses one of the most challenging hurdles in CNS drug development, as many promising candidates fail due to inadequate brain exposure. Second, the dose-proportional pharmacokinetics suggest predictable exposure at different dose levels, simplifying future dosing strategies.
While Phase 1 studies primarily assess safety rather than efficacy, establishing this favorable tolerability profile in healthy volunteers is a necessary stepping stone before testing in patient populations. The targeting of neuroinflammation through KCNK13 inhibition represents a differentiated approach to neurodegenerative diseases, potentially addressing a key driver of disease progression rather than merely treating symptoms.
Cerevance's broader pipeline appears strategically diversified, with solengepras for Parkinson's disease already advancing in Phase 2 and the proprietary NETSseq platform generating additional novel targets with specific brain expression. This suggests a sustainable discovery engine beyond their current clinical candidates.
The successful Phase 1 results for CVN293 highlight the potential of targeting KCNK13 (a potassium channel) to address neuroinflammation—a common pathological mechanism underlying multiple neurodegenerative disorders. Neuroinflammation involves chronic immune activation in the brain that contributes to neuronal damage, and effective modulation of this process has proven challenging in drug development.
The high CNS exposure demonstrated in this study is particularly noteworthy, as the blood-brain barrier presents a significant obstacle for many drug candidates. Adequate concentration at the target site is essential for therapeutic efficacy in central nervous system disorders, and these pharmacokinetic data suggest CVN293 overcomes this critical hurdle.
Cerevance's approach of targeting cell type-specific pathways, as enabled by their NETSseq platform, represents a more precise strategy than traditional approaches that often affect multiple cell types indiscriminately. This selectivity could potentially improve efficacy while reducing off-target effects.
Their second clinical candidate, solengepras, further demonstrates the platform's versatility by targeting GPR6 for Parkinson's disease through a non-dopaminergic mechanism. This approach could potentially avoid the motor complications associated with traditional dopamine replacement therapies—addressing a significant unmet need in Parkinson's treatment.
While these early results are promising, the true test will come in later-stage trials that evaluate efficacy endpoints in patient populations. Nevertheless, establishing this favorable safety and pharmacokinetic foundation is an essential first step in clinical development.
- CVN293 was generally well-tolerated in healthy volunteers with evidence of robust brain penetration, supporting utility of KCNK13 inhibitors for neurodegenerative diseases characterized by neuroinflammation
- Additional AAN 2025 poster presentations highlight the company’s NETSseq platform and solengepras, a novel non-dopaminergic investigational treatment in clinical development to treat Parkinson’s disease
BOSTON, April 08, 2025 (GLOBE NEWSWIRE) -- Cerevance, a clinical-stage biopharmaceutical company advancing multiple cell type-specific therapies for the treatment of neurodegenerative, psychiatric, and central nervous system-controlled metabolic disorders, today announced results from its Phase 1 trial of CVN293, a novel KCNK13 inhibitor targeting neuroinflammation. The data showed that CVN293 was generally well-tolerated in healthy adults following single and 14-day multiple dosing and showed evidence of robust brain penetration. The data was presented today in a poster session at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego.
“The favorable tolerability and pharmacokinetic profile observed with CVN293 in this first-in-human study are promising indicators for its potential as a disease-modifying intervention for neurodegenerative diseases driven by neuroinflammation,” said Sagar Vaidya, M.D., Ph.D., chief medical officer of Cerevance. “We look forward to advancing our family of KCNK13 inhibitors for the treatment of neurodegenerative disorders characterized by neuroinflammation, where new therapeutic options are urgently needed.”
The randomized, double-blind, placebo-controlled Phase 1 single/multiple ascending dose (SAD/MAD) study evaluated the safety, tolerability and pharmacokinetics of oral CVN293 in 72 healthy adult volunteers. Study participants in the SAD part of the study received a single dose of CVN293 up to 1,000 mg or placebo (CVN293 n=36; placebo n=12) and those in the MAD part received repeated doses of CVN293 up to 750 mg (375 mg twice daily) or placebo over 14 days (CVN293 n=18; placebo n=6).
Results showed that CVN293 was generally well-tolerated following single and 14-day multiple dosing, with
Additional Cerevance Presentations at AAN 2025
The company presented a poster demonstrating the potential of its proprietary NETSseq platform to identify and/or validate cell-specific gene targets and biological insight into human brain disorders, which can be developed into novel therapies for neurodegenerative diseases. The poster detailed two clinical-stage novel therapeutic targets with specific expression in the brain that resulted from NETSseq: solengepras and CVN293.
Another poster presentation will detail the novel mechanism of action of solengepras, an oral, non-dopaminergic therapy in clinical development for the treatment of Parkinson’s disease. A brain-penetrant, specific inhibitor of GPR6, solengepras is designed to selectively target and modulate the brain circuits responsible for controlling motor and non-motor functions without directly affecting dopaminergic pathways. This novel mechanism of action is designed to reduce motor complications, such as dyskinesia, decrease periods of symptomatic worsening, known as “OFF” time, and improve functional non-motor symptoms. The poster will describe the primary results of the randomized, double-blind, placebo-controlled Phase 2 ASCEND study of solengepras as monotherapy in patients with early, untreated Parkinson’s disease. This data was previously presented at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2025).
About CVN293
CVN293 is an investigational, highly selective oral inhibitor of the potassium two pore domain channel subfamily K member 13 (KCNK13). By selectively inhibiting KCNK13, 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 (CNS) disorders and obesity. KCNK13 was identified by Cerevance’s proprietary NETSseq platform.
About Solengepras (formerly CVN424)
Solengepras is designed to provide a potentially novel approach to the treatment of Parkinson’s disease. Unlike dopaminergic therapies, which primarily act by replenishing, enhancing, or mimicking dopamine, solengepras is designed to selectively address the indirect pathway by modulating the GPR6 receptor. By inhibiting GPR6, solengepras aims to restore both motor and non-motor function without directly affecting dopamine levels or signaling, improving the relative balance between the direct and indirect pathways, and potentially reducing the risk of common side effects associated with dopaminergic therapies, such as dyskinesias and motor fluctuations. Solengepras is currently being evaluated as a once-daily, oral treatment for use as an adjunctive therapy to levodopa and other anti-Parkinsonian medication in the Phase 3 ARISE trial.
About Cerevance
Cerevance is focused on advancing cell type-specific therapies for the treatment of neurodegenerative, psychiatric and central nervous system-controlled metabolic disorders. Our proprietary platform, Nuclear Enriched Transcript Sort sequencing (NETSseq), allows us to identify targets that are expressed at very low levels, that are present in rare cell types, or that change over time as a disease progresses. Our 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. Our 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. Our third investigational treatment, CVN293, is a highly selective investigational oral inhibitor targeting potassium two pore domain channel subfamily K member 13 (KCNK13). CVN293 represents a potentially novel intervention point for neurodegenerative disorders and obesity. For more information, please visit www.cerevance.com and follow us on LinkedIn and X.
Contacts
Cerevance:
Johnna Simoes, ir@cerevance.com
Media
April Dovorany, adovorany@realchemistry.com, +1-262-909-8739
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