Cerevance, Inc.

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.

CVN293 was generally well-tolerated in healthy volunteers for up to 14-days of continuous dosing Dose-dependent exposure was observed with evidence of robust brain penetration Data supports potential advancement into Phase 2 for neurodegenerative diseases characterized by neuroinflammation such as Frontotemporal Dementia, Amyotrophic Lateral Sclerosis, and Alzheimer’s Disease Nov. 11, 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 reported positive results from its Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) clinical study of CVN293 in healthy volunteers. CVN293 is designed to inhibit KCNK13, a potentially novel target involved in activating the NLRP3 inflammasome, with the aim of reducing neuroinflammation and attenuating disease progression in a variety of neurodegenerative disorders. KCNK13 was identified by Cerevance’s proprietary NETSseq platform. “We are very encouraged by the clinical data for CVN293, observing that it was generally well-tolerated and achieved favorable brain penetration,” said Mark Carlton, Ph.D., chief scientific officer and co-founder of Cerevance. “CVN293 selectively inhibits KCNK13, a gene selectively expressed in microglia with minimal expression in peripheral tissues. This selective targeting may contribute to its favorable tolerability profile. By inhibiting KCNK13 to reduce NLRP3 activation, CVN293 may offer a disease-modifying approach for challenging CNS disorders.” CVN293 was generally well-tolerated in healthy participants with single doses up to 1000mg and multiple doses of up to 375mg twice daily for 14 days. No serious adverse events were reported and all participants completed the study. All related treatment-emergent adverse events were mild. Dose-dependent increases in CVN293 exposure were observed, achieving plasma concentrations that align with clinically relevant levels as indicated by preclinical studies. CSF sampling demonstrated high brain penetrance of CVN293. Clinical safety and pharmacokinetic profile of CVN293 is favorable for potential advancement into Phase 2 studies. The Phase 1 study of CVN293 was a first-in-human, randomized, placebo-controlled, single-center, SAD and MAD study in 72 healthy adult volunteers. The primary objective of the study was to evaluate the safety, tolerability, and pharmacokinetics of oral CVN293. In the SAD portion of the study, a single dose of CVN293 ranging from 3 mg to 1000 mg or placebo were administered to 48 healthy adult participants (CVN293 n=36; placebo n=12). In the MAD portion of the study, CVN293 doses from 50 mg (25 mg twice daily) to 750 mg (375 mg twice daily) or placebo were administered to 24 healthy adult participants (CVN293 n=18; placebo n=6) over 14 consecutive days. CVN293 is a highly-selective oral inhibitor targeting KCNK13, a potassium two pore domain channel subfamily K member 13, which is implicated in neuroinflammation and central nervous system disorders. By targeting KCNK13, identified to be solely expressed in microglia with Cerevance’s proprietary NETSseq platform, CVN293 aims to reduce the activation of the NOD-like receptor family, pyrin domain containing 3 (NLRP3) inflammasome specifically in the brain. This inflammasome plays a critical role in the activation of microglia, which are associated with neurodegenerative disorders such as Alzheimer’s disease and frontotemporal dementia. In contrast to other therapies that target NLRP3 which may suppress aspects of the peripheral immune system, we believe the selective inhibition of KCNK13 by CVN293 in microglia may provide better tolerability relative to other approaches targeting NLRP3 and has the potential to act as a disease-modifying treatment by specifically targeting neuroinflammation in the brain. 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 novel targets, even those 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.

BOSTON, Oct. 21, 2024 (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, has published the full results from the Phase 2 clinical trial of solengepras, in eClinicalMedicine , a peer-reviewed journal published by The Lancet Discovery Science Suite . The results demonstrated that solengepras, a potentially first-in-class, oral, once-daily, non-dopaminergic, GPR6 inverse agonist, significantly reduced OFF time in individuals with Parkinson’s disease and was generally well-tolerated. The Phase 2 clinical trial met the key efficacy endpoint and demonstrated a reduction in average daily OFF time from baseline at Day 27, as recorded over two days using a self-reported patient motor diary. On Day 27, the change from baseline in daily OFF time was -1.6 hours (p 5%) were headache and nausea which were generally mild and transient. “We are excited to share the results of this important Phase 2 study with the broader scientific community through this publication in eClinicalMedicine,” said chief executive officer, Craig Thompson. “The data demonstrated solengepras’ potential to be a meaningful therapeutic option for individuals with Parkinson’s disease, addressing not only motor symptoms but also overall quality-of-life.” For more information on the Phase 2 clinical trial and to access the full publication, visit About Solengepras (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 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). About Parkinson’s Disease 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 pro About Cerevance 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, N uclear E nriched T ranscript S ort seq uencing (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. Contacts Cerevance: Johnna Simões, ir@cerevance.com Media: Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868