KCNK13

CVN293 was generally well-tolerated in healthy volunteers with evidence of robust brain penetration, supporting utility of KCNK13 inhibitors for neurodegenerative diseases characterized by neuroinflammationAdditional 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 100% of participants completing the trial. There were no severe or dose-limiting adverse events (AEs), treatment-related discontinuations or clinically meaningful changes in vital signs or laboratory parameters. All related AEs were considered mild. In the SAD and MAD parts of the study, CVN293 plasma exposure increased in a generally dose proportional manner. Cerebrospinal fluid (CSF) sampling demonstrated high levels of central nervous system (CNS) exposure with CVN293. Additional Cerevance Presentations at AAN 2025The 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 CVN293CVN293 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 CerevanceCerevance 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 MediaApril Dovorany, adovorany@realchemistry.com, +1-262-909-8739

Topline results from Phase 2 ASCEND trial of solengepras as a monotherapy treatment for early-stage Parkinson’s to be presentedFirst presentation of data from Phase 1 trial of novel KCNK13 inhibitor for CNS diseases targeting neuroinflammationAdditional meeting presentations highlight potential of proprietary NETSseq platform to identify novel therapeutic targets for neurodegenerative diseases ‍BOSTON, March 27, 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 upcoming presentations at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2025) being held April 1-5 in Vienna, and the American Academy of Neurology (AAN) Annual Meeting being held April 5-9 in San Diego. Details of the oral presentation at AD/PD™ 2025 are as follows: Title: Results of ASCEND: A Phase 2 Trial of Solengepras (CVN424) as an Investigational Monotherapy for Treatment of Early Parkinson’s Disease Presenter: Aaron L. Ellenbogen, DO, MPHSession Date/Time: Tuesday, April 1, 2025, 1:15 p.m. CEST / 4:15 a.m. PT Details of the poster presentations at the AAN 2025 Annual Meeting are as follows: Title: NETSseq Identifies Novel Therapeutic Targets for Neurodegenerative Diseases (#3592) Presenter: Sagar Vaidya, M.D., Ph.D.Session Date/Time: Sunday, April 6, 2025, 11:45 a.m. PT Title: A First-in-Human Phase 1 Study to Investigate the Safety, Tolerability, and Pharmacokinetics of CVN293, an Investigational Inhibitor of KCNK13 Targeting NLRP3-Mediated Neuroinflammation (#3282) Presenter: Sagar Vaidya, M.D., Ph.D.Session Date/Time: Tuesday, April 8, 2025, 8 a.m. PT Title: Solengepras (CVN424): A Novel GPR6 Inhibitor in Clinical Development as an Investigational Therapy for Parkinson’s Disease (#2892) Presenter: Kelvin L. Chou, M.D., FAANSession Date/Time: Wednesday, April 9, 2025, 8 a.m. PT 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 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

Solengepras is a potentially first-in-class, oral, non-dopaminergic investigational therapy in development for Parkinson’s disease ARISE will evaluate the efficacy of solengepras as an adjunctive therapy to levodopa and other background Parkinson’s disease medications Company expects to report topline data in the first half of 2026 Nov. 18, 2024 -- 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, has dosed the first patient in ARISE, a Phase 3 clinical trial evaluating the efficacy of solengepras as a potential adjunctive treatment for individuals with Parkinson’s disease. “Parkinson’s disease is the fastest growing neurological disorder globally, highlighting a significant need for more improved and effective therapies,” shared Craig Thompson, chief executive officer of Cerevance. “Unlike traditional Parkinson’s treatments that aim to restore dopamine levels, solengepras is designed to selectively target and modulate the specific brain circuits responsible for controlling movement and non-motor functions, without relying directly on dopamine pathways. This novel mechanism of action is designed to reduce motor complications such as dyskinesia and “OFF” periods, which we believe may offer potentially improved tolerability and potential impact on non-motor symptoms for individuals with Parkinson’s disease.” ARISE is a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial, with a plan to enroll approximately 330 patients with three or more hours of “OFF” time per day. The primary endpoint is the change from baseline in the total daily “OFF” time at Week 12. Secondary endpoints include the change in “ON” time without troublesome dyskinesia, evaluations of motor function and quality of life, as assessed by the Movement Disorder Society-Unified Parkinson’s disease Rating Scale (MDS-UPDRS), the Epworth Sleepiness Scale (ESS), and the use of digital technologies, such as the Cogstate PD Battery (for the evaluation of cognition) and the Modality System (for the evaluation of motor/non-motor impairment). The trial will also evaluate the safety and tolerability of solengepras. “The initiation of ARISE builds on the encouraging Phase 2 data we have generated and represents an exciting chapter in Cerevance’s journey to address the unmet needs of individuals with Parkinson’s disease,” added Sagar Vaidya, M.D., Ph.D., chief medical officer of Cerevance. “We look forward to advancing solengepras through this pivotal trial and moving closer to potentially bringing a new, non-dopaminergic therapy to the Parkinson’s community.” 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 both a monotherapy (Phase 2 ASCEND clinical trial) and as an adjunctive therapy to levodopa and other anti-Parkinsonian medication (Phase 3 ARISE clinical trial). Parkinson’s disease is a progressive neurodegenerative disorder that is characterized by both motor symptoms, such as tremor, rigidity, and bradykinesia/akinesia, and non-motor symptoms, such as mood changes, apathy, and cognitive deficits. Globally, Parkinson’s disease is the fastest growing neurological disorders, affecting more than ten million people worldwide and approximately one million people in the United States. The current standard of care has primarily relied on dopaminergic therapies, which lose effectiveness over time and are associated with side effects that result in challenging risk-benefit profiles. Cerevance is focused on advancing cell type-specific therapies for the treatment of neurodegenerative, psychiatric and central nervous system (“CNS”)-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 product candidate, solengepras (CVN424), 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 product candidate, CVN766, is designed to be a highly selective oral antagonist of the orexin 1 receptor that we plan to evaluate in a Phase 2 study for the potential treatment of binge eating disorder and schizophrenia. Our third product candidate, CVN293, is a highly selective investigational oral inhibitor targeting potassium two pore domain channel subfamily K member 13 (KCNK13) and represents a potentially novel intervention point for neurodegenerative disorders by reducing neuroinflammation. We plan to evaluate CVN293 in a Phase 2 study for the potential treatment of frontotemporal dementia. The content above comes from the network. if any infringement, please contact us to modify.