Cerevance Presents Positive Results from Phase 1 Study of CVN293 at American Academy of Neurology 2025 Annual Meeting
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.
Cerevance ha annunciato risultati positivi per la fase 1 dello studio clinico su CVN293, un nuovo inibitore del KCNK13 mirato alla neuroinfiammazione, durante il Meeting Annuale dell'Accademia Americana di Neurologia 2025. Lo studio, randomizzato, in doppio cieco e controllato con placebo, ha coinvolto 72 adulti sani, valutando dosi singole fino a 1.000 mg e dosi multiple fino a 750 mg per 14 giorni.
I risultati chiave hanno mostrato che CVN293 è stato ben tollerato con:
- 100% di completamento da parte dei partecipanti
- Nessun evento avverso grave o limitante per dose
- Solo effetti collaterali lievi legati al trattamento
- Alti livelli di esposizione nel sistema nervoso centrale
- Esposizione plasmatica generalmente proporzionale alla dose
L'azienda ha anche presentato dati sulla loro piattaforma NETSseq e solengepras, una terapia non dopaminergica in fase di sviluppo per il morbo di Parkinson, inclusi i risultati dello studio ASCEND di fase 2 su pazienti con Parkinson in fase precoce e non trattati.
Cerevance anunció resultados positivos de la fase 1 del ensayo clínico para CVN293, un nuevo inhibidor de KCNK13 dirigido a la neuroinflamación, en el Encuentro Anual de la Academia Americana de Neurología 2025. El estudio, aleatorizado, doble ciego y controlado con placebo, involucró a 72 adultos sanos, evaluando dosis únicas de hasta 1,000 mg y dosis múltiples de hasta 750 mg durante 14 días.
Los hallazgos clave mostraron que CVN293 fue bien tolerado con:
- Tasa de finalización del 100% de los participantes
- Sin eventos adversos graves o limitantes por dosis
- Solo efectos leves relacionados con el tratamiento
- Altos niveles de exposición en el sistema nervioso central
- Exposición plasmática generalmente proporcional a la dosis
La empresa también presentó datos sobre su plataforma NETSseq y solengepras, una terapia no dopaminérgica en desarrollo para la enfermedad de Parkinson, incluidos los resultados del estudio ASCEND de fase 2 en pacientes con Parkinson temprano y no tratados.
세레반스(Cerevance)는 CVN293의 1상 시험 긍정적인 결과를 발표했습니다. 이는 신경 염증을 표적으로 하는 새로운 KCNK13 억제제이며, 2025년 미국 신경학회 연례 회의에서 발표되었습니다. 이 무작위 이중 맹검 위약 대조 연구는 72명의 건강한 성인이 참여했으며, 1,000mg까지의 단일 용량과 14일 동안 750mg까지의 다중 용량을 평가했습니다.
주요 결과는 CVN293이 잘 견뎌졌음을 보여주었습니다:
- 참여자 완주율 100%
- 심각하거나 용량 제한적인 부작용 없음
- 치료와 관련된 경미한 효과만
- 중추 신경계에서의 높은 노출 수준
- 일반적으로 용량 비례적인 혈장 노출
회사는 또한 NETSseq 플랫폼 및 솔렌게프라스(solengepras)에 대한 데이터를 발표했습니다. 이는 파킨슨병 치료를 위한 비도파민성 요법으로, 초기 치료를 받지 않은 파킨슨병 환자에 대한 2상 ASCEND 연구 결과를 포함합니다.
Cerevance a annoncé des résultats positifs d'essai de phase 1 pour CVN293, un nouvel inhibiteur de KCNK13 ciblant la neuroinflammation, lors de la Réunion Annuelle de l'Académie Américaine de Neurologie 2025. L'étude randomisée, en double aveugle et contrôlée par placebo a impliqué 72 adultes en bonne santé, évaluant des doses uniques allant jusqu'à 1 000 mg et des doses multiples allant jusqu'à 750 mg sur 14 jours.
Les résultats clés ont montré que CVN293 était bien toléré avec :
- Taux de complétion de 100 % des participants
- Aucun événement indésirable grave ou limitant la dose
- Seulement des effets légers liés au traitement
- Niveaux élevés d'exposition dans le système nerveux central
- Exposition plasmatique généralement proportionnelle à la dose
L'entreprise a également présenté des données sur sa plateforme NETSseq et solengepras, une thérapie non dopaminergique en développement pour la maladie de Parkinson, y compris les résultats de l'étude ASCEND de phase 2 chez des patients parkinsoniens précoces non traités.
Cerevance hat positive Ergebnisse der Phase-1-Studie für CVN293 bekannt gegeben, einen neuartigen KCNK13-Inhibitor, der auf Neuroinflammation abzielt, auf dem Jahrestreffen der American Academy of Neurology 2025. Die randomisierte, doppelblinde, placebo-kontrollierte Studie umfasste 72 gesunde Erwachsene und bewertete Einzeldosen von bis zu 1.000 mg und Mehrfachdosen von bis zu 750 mg über 14 Tage.
Wichtige Ergebnisse zeigten, dass CVN293 gut vertragen wurde mit:
- 100% Abschlussrate der Teilnehmer
- Keine schweren oder dosierungsbegrenzenden unerwünschten Ereignisse
- Nur leichte behandlungsbedingte Effekte
- Hohe Expositionsniveaus im zentralen Nervensystem
- Allgemein dosisproportionale Plasmakonzentration
Das Unternehmen präsentierte auch Daten zu ihrer NETSseq-Plattform und solengepras, einer nicht-dopaminergen Therapie in Entwicklung für die Parkinson-Krankheit, einschließlich der Ergebnisse der Phase-2-ASCEND-Studie bei frühen, unbehandelten Parkinson-Patienten.
- 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)
- 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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