EAAT2 modulators(solute carrier family 1 member 2 modulators), NMDA receptor antagonists(Glutamate [NMDA] receptor antagonists), Nav1.5 blockers(Sodium channel protein type V alpha subunit blockers)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersOther Diseases+ [3]

Active Indication

Machado-Joseph DiseaseObsessive-Compulsive DisorderSpinocerebellar Ataxias+ [6]

Inactive Indication

Alzheimer DiseaseAnxiety DisordersBrain metastases+ [3]

Originator Organization

Yale University

Active Organization

Biohaven Pharmaceuticals, Inc.Polaris Group

Kazia Therapeutics Ltd.

+ [7]

Inactive Organization-

Drug Highest PhaseNDA/BLA

First Approval Date-

RegulationFast Track (US), Orphan Drug (US)

Structure

Molecular FormulaC15H17ClF3N5O4S

InChIKeyZRZLEMQBDKNTLJ-UHFFFAOYSA-N

CAS Registry1926204-76-3

Clinical Trials associated with Trigriluzole

NCT06552260 / Not yet recruitingEarly Phase 1IIT

A Surgical Window of Opportunity Clinical Trial of Troriluzole in Recurrent IDH Wild-Type Glioblastoma

This research study is studying troriluzole as a possible treatment for recurrent glioblastoma.
The name of the study drug involved in this research study is:
-Troriluzole (a tripeptide prodrug of riluzole)

Start Date01 Feb 2025

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+2]

CTR20220688 / RecruitingPhase 3

一项Troriluzole ？助治疗强迫症受试者的多中心、48周、开放标签安全性研究

[Translation] A Multicenter, 48-week, Open-label Safety Study of Troriluzole as an Adjunct to the Treatment of Obsessive-compulsive Disorder Subjects

本研究的主要目的是在根据主方案（？BHV4157-302 和BHV4157-303 研究）定义的对OCD标准治疗（SOC）反应不佳的OCD受试者中，评价troriluzole作为辅助治疗的长期安全性和耐受性。

[Translation]

The primary objective of this study was to evaluate the long-term safety and tolerability of troriluzole as adjunctive therapy in subjects with OCD who had an inadequate response to standard of care (SOC) for OCD as defined in the master protocol (?BHV4157-302 and BHV4157-303 studies).

Start Date31 Jan 2023

Sponsor / Collaborator

Bioshin Ltd.

[+1]

CTR20212534 / RecruitingPhase 3

一项Troriluzole用于辅助治疗强迫症的随机、双盲、安慰剂对照试验

[Translation] A randomized, double-blind, placebo-controlled trial of troriluzole as an adjunctive treatment for obsessive-compulsive disorder

本研究的主要目的是与安慰剂相比，基于Y-BOCS评分的变化，评价Troriluzole作为辅助治疗用于对当前OCD治疗效果不佳的OCD受试者的疗效。

[Translation]

The primary objective of this study was to evaluate the efficacy of troriluzole as adjunctive therapy compared with placebo in subjects with OCD who were refractory to current OCD treatments based on changes in Y-BOCS scores.

Start Date19 Jul 2022

Sponsor / Collaborator

Bioshin Ltd.

[+1]

100 Clinical Results associated with Trigriluzole

100 Translational Medicine associated with Trigriluzole

100 Patents (Medical) associated with Trigriluzole

Literatures (Medical) associated with Trigriluzole

01 Aug 2024·Neurology and Therapy

Video-Based Kinematic Analysis of Movement Quality in a Phase 3 Clinical Trial of Troriluzole in Adults with Spinocerebellar Ataxia: A Post Hoc Analysis

Article

Author: Perlman, Susan ; Maclaine, Grant D H ; L'Italien, Gilbert J ; Khera, Rohan ; Coric, Vladimir ; Potashman, Michele H ; Schmahmann, Jeremy D ; Beiner, Melissa W ; Oikonomou, Evangelos K

INTRODUCTION:

Traditional methods for assessing movement quality rely on subjective standardized scales and clinical expertise. This limitation creates challenges for assessing patients with spinocerebellar ataxia (SCA), in whom changes in mobility can be subtle and varied. We hypothesized that a machine learning analytic system might complement traditional clinician-rated measures of gait. Our objective was to use a video-based assessment of gait dispersion to compare the effects of troriluzole with placebo on gait quality in adults with SCA.

METHODS:

Participants with SCA underwent gait assessment in a phase 3, double-blind, placebo-controlled trial of troriluzole (NCT03701399). Videos were processed through a deep learning pose extraction algorithm, followed by the estimation of a novel gait stability measure, the Pose Dispersion Index, quantifying the frame-by-frame symmetry, balance, and stability during natural and tandem walk tasks. The effects of troriluzole treatment were assessed in mixed linear models, participant-level grouping, and treatment group-by-visit week interaction adjusted for age, sex, baseline modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA), and time since diagnosis.

RESULTS:

From 218 randomized participants, 67 and 56 participants had interpretable videos of a tandem and natural walk attempt, respectively. At Week 48, individuals assigned to troriluzole exhibited significant (p = 0.010) improvement in tandem walk Pose Dispersion Index versus placebo {adjusted interaction coefficient: 0.584 [95% confidence interval (CI) 0.137 to 1.031]}. A similar, nonsignificant trend was observed in the natural walk assessment [coefficient: 1.198 (95% CI - 1.067 to 3.462)]. Further, lower baseline Pose Dispersion Index during the natural walk was significantly (p = 0.041) associated with a higher risk of subsequent falls [adjusted Poisson coefficient: - 0.356 [95% CI - 0.697 to - 0.014)].

CONCLUSION:

Using this novel approach, troriluzole-treated subjects demonstrated improvement in gait as compared to placebo for the tandem walk. Machine learning applied to video-captured gait parameters can complement clinician-reported motor assessment in adults with SCA. The Pose Dispersion Index may enhance assessment in future research. TRIAL REGISTRATION-CLINICALTRIALS.

GOV IDENTIFIER:

NCT03701399.

01 Mar 2023·Pharmaceutical patent analyst

Riluzole and Its Prodrugs for the Treatment of Alzheimer’s Disease

Article

Author: Al-Horani, Rami A

Current medications for Alzheimer’s disease help manage symptoms and behavioral problems. Nevertheless, they do not slow the progression of cognitive decline or dementia. A potential approach for treating Alzheimer’s disease is to target neurons that are sensitive to disease pathobiology such as glutamatergic neurons. Several patents disclosed methods for treating Alzheimer’s disease by administering riluzole or its prodrugs. Clinical trials revealed that 6 months treatment using riluzole or troriluzole is associated with a slower decline in the tomographic measures of the positron emissions of cerebral glucose metabolism in Alzheimer’s patients. The proposed strategy claims to prevent and/or slow the cognitive decline of Alzheimer’s patients and to enhance global functioning. These claims may also pave the way for other glutamate modulators to be used for Alzheimer’s disease.

01 Jan 2023·Drug and alcohol dependence

Troriluzole inhibits methamphetamine place preference in rats and normalizes methamphetamine-evoked glutamate carboxypeptidase II (GCPII) protein levels in the mesolimbic pathway

Article

Author: Rawls, Scott M ; Potula, Raghava ; Roper, Abigail ; Zhao, Pingwei ; Reitz, Allen B ; Wiah, Sonita ; Shekarabi, Aryan ; Farkas, Daniel J ; Watson, Mia N

Riluzole, approved to manage amyotrophic lateral sclerosis, is mechanistically unique among glutamate-based therapeutics because it reduces glutamate transmission through a dual mechanism (i.e., reduces glutamate release and enhances glutamate reuptake). The profile of riluzole is favorable for normalizing glutamatergic dysregulation that perpetuates methamphetamine (METH) dependence, but pharmacokinetic and metabolic liabilities hinder repurposing. To mitigate these limitations, we synthesized troriluzole (TRLZ), a third-generation prodrug of riluzole, and tested the hypothesis that TRLZ inhibits METH hyperlocomotion and conditioned place preference (CPP) and normalizes METH-induced changes in mesolimbic glutamate biomarkers. TRLZ (8, 16 mg/kg) reduced hyperlocomotion caused by METH (1 mg/kg) without affecting spontaneous activity. TRLZ (1, 4, 8, 16 mg/kg) administered during METH conditioning (0.5 mg/kg x 4 d) inhibited development of METH place preference, and TRLZ (16 mg/kg) administered after METH conditioning reduced expression of CPP. In rats with established METH place preference, TRLZ (16 mg/kg) accelerated extinction of CPP. In cellular studies, chronic METH enhanced mRNA levels of glutamate carboxypeptidase II (GCPII) in the ventral tegmental area (VTA) and prefrontal cortex (PFC). Repeated METH also caused enhancement of GCPII protein levels in the VTA that was prevented by TRLZ (16 mg/kg). TRLZ (16 mg/kg) administered during chronic METH did not affect brain or plasma levels of METH. These results indicate that TRLZ, already in clinical trials for cerebellar ataxia, reduces development, expression and maintenance of METH CPP. Moreover, normalization of METH-induced GCPII levels in mesolimbic substrates by TRLZ points toward studying GCPII as a therapeutic target of TRLZ.

News (Medical) associated with Trigriluzole

16 Dec 2024

·www.prnewswire.com

Biohaven Reports Positive Phase 1 Degrader Data, Achieving Deep Targeted IgG Reductions in the Lowest Subcutaneous Dose Tested; Announces NDA Submission for Troriluzole in SCA and Provides Other Key Program Updates

BHV-1300 achieved deep lowering of targeted IgG, with reductions > 60% in the lowest subcutaneous dose tested in the MAD. Subcutaneous BHV-1300 achieved rapid and progressive lowering of IgG within hours of each weekly dose administration, and pharmacodynamic effects were sustained relative to baseline over the four-week period. The optimized subcutaneous formulation also showed substantially less inter-patient variability in the MAD compared to the previously reported intravenously administered BHV-1300. BHV-1300 has been safe and well tolerated across the ongoing Phase 1 without any dose limiting toxicity to date. All AEs have been mild, with no SAEs or discontinuations related to study drug. Dose escalation continues with the optimized subcutaneous formulation to explore the full range of IgG reductions possible with BHV-1300. Additional Phase 1 data will be presented upon completion of the remaining subcutaneous cohorts in 1Q25. Laboratory data from the MAD confirm a differentiated safety profile compared to competitor agents as BHV-1300 has had no clinically significant reductions in albumin, liver function test abnormalities or increases in cholesterol at week 4 relative to baseline. Further enhancing the competitive safety profile, BHV-1300 was rationally designed to spare IgG3 with plasma IgG3 levels over the course of the MAD preserved through the end of study week 4 to allow for healthy immune effector functioning. Submitted new drug application (NDA) to US FDA for troriluzole in spinocerebellar ataxia (SCA), following completion of pre-NDA meeting in 4Q 2024. Troriluzole has Orphan Drug and Fast-Track designations and qualifies for potential Priority Review. Announced completion of enrollment during 4Q 2024 in the BHV-7000 pivotal 3-week, Phase 2/3 bipolar trial, several months ahead of timelines. Expanded and advanced the molecular degraders of extracellular proteins (MoDE) clinical program to include next-generation autoantibody specific degraders that selectively remove pathogenic antibodies while preserving healthy immune functioning, with regulatory acceptance of 3 novel drug candidate INDs and/or CTAs in 4Q24: IgA nephropathy: Initiated Phase 1 dosing with BHV-1400, a novel IgAN investigational therapy designed to selectively degrade pathogenic galactose deficient IgA1 (Gd-IgA1) while sparing normal IgA. In addition to rapid and sustained lowering of Gd-IgA1, BHV-1400 is expected to result in less potential for respiratory, mucosal or central nervous system infections compared to broader IgA lowering or immunosuppressive strategies in development by competitors. Autoimmune cardiomyopathy: Initiated Phase 1 dosing with BHV-1600, a novel investigational therapy designed to selectively degrade b1 adrenergic receptor (b1AR) autoantibodies. Biohaven also completed an INTERACT meeting with FDA regarding BHV-1600 in 4Q 2024 and gained alignment for the study design to pursue an accelerated approval pathway in peripartum cardiomyopathy (PPCM), a rare autoimmune life-threatening disease with no approved therapy. IgG mediated diseases: IND opened for BHV-1310, an optimized and selective IgG1, IgG2, and IgG4 degrader in 4Q 2024 with first dosing planned for 1Q 2025. Entered into an agreement with Ypsomed to develop and manufacture BHV-1300 in an easy-to-use, autoinjector for self-administration. Ypsomed is a leading provider of autoinjector technology used in commercialized products for convenient patient use. The Ypsomed device is expected to be used across all MoDE programs through development and commercial use, derisking this aspect of the development program as well as providing seamless transitions and allowing for significant data generation on the device in advance of future NDA filings. NEW HAVEN, Conn., Dec. 16, 2024 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of life-changing therapies to treat a broad range of rare and common diseases, today highlighted the achievement of several clinical and regulatory milestones across its proprietary Molecular Degrader of Extracellular Proteins (MoDE™) platform as well as its glutamate modulation and ion channel programs. Figure 1: BHV-1300 easy-to-use autoinjector for pivotal trials in 2025, developed and manufactured in collaboration with Ypsomed. Ypsomed, a Swiss device maker and global leader in the development of innovative injection systems, is well-known for its design, development and production of user-friendly injectors for self-medication in various diseases including the treatment of metabolic disorders. This device is expected to be used across all MoDE programs through development and commercialization, providing seamless transitions and allowing for significant data generation on the device in advance of future NDA filings. Figure 2: (A) IgA nephropathy is caused by excess production of galactose deficient IgA1 and deposition in the renal glomerulus, setting off an inflammatory cascade and renal damage. Figure 2: (B) BHV-1400 degrades galactose deficient IgA1 (red) in vivo in preclinical models. With BHV-1400 administration compared to vehicle administration, galactose deficient IgA1 clears from the renal glomerulus (inset) over hours. BHV-1400 was designed with precision to remove the pathogenic driver of IgA nephropathy and preserve components of the immune system crucial to fighting infections, such as meningitis. Figure 3: Autoantibodies target the b1-adrenergic receptor (b1AR) to cause cardiac dilation and heart failure.1 BHV-1600 selectively removes autoantibodies to b1AR, and has been dosed in humans, with recent FDA alignment on a clinical development program to most efficiently deliver a potentially transformative therapeutic to patients with peripartum cardiomyopathy. Subcutaneously administered BHV-1300 achieved deep lowering of targeted IgG, with reductions > 60% in the lowest subcutaneous dose tested in the ongoing multiple ascending dose (MAD) study. Subcutaneous BHV-1300 achieved progressive reduction in IgG within hours of each weekly dose administration in the MAD, and pharmacodynamic effects were sustained relative to baseline over the four-week study period. BHV-1300 has been safe and well-tolerated across the Phase 1 study. There were no clinically significant effects on albumin or liver function, and no increases in cholesterol were noted. Further enhancing the competitive safety profile and as intentionally designed, plasma IgG3 levels were preserved through the end of study week 4 to allow for healthy immune effector functioning. All AEs were mild, any drug-related AE resolved, and there were no discontinuations due to study drug related AEs. The optimized subcutaneous formulation in the MAD also showed substantially less inter-patient variability compared to previously reported intravenous BHV-1300. Escalating dose level cohorts of subcutaneous BHV-1300 are ongoing to explore the full range of IgG reductions possible with BHV-1300 for a wide range of future disease indications. Tova Gardin MD, MPP, Biohaven's Chief Translational Officer, commented, "The results of subcutaneously delivered BHV-1300 from the first and lowest MAD dose cohort represent a monumental step forward for our MoDE platform with deep and rapid reduction in targeted IgG. Our results highlight the selectivity and precision of BHV-1300 in potentially treating IgG mediated immune diseases while the regulatory acceptance of three next generation degraders showcases the future of the MoDE technology in autoantibody disease. With speed, selectivity, and depth of lowering, BHV-1300 has the potential to transform the treatment of autoimmune disease and has paved the way for rapid innovation across the degrader platform. With the mechanistic validation of BHV-1300 and performance of the optimized subcutaneous formulation in Phase 1, we have begun the manufacturing of a convenient autoinjector that will further differentiate our approach in the clinic. The early autoinjector development to ensure a commercial ready, patient-administered device for BHV-1300 was important to derisking this aspect of our MoDE development program." Biohaven licensed the MoDE technology from Yale University with high potential differentiation for the treatment of autoimmune diseases, including rapid degradation of targets within hours, deep reductions that can be titrated by dose level and frequency, simple-to-use patient administration, excellent tolerability without deleterious effects on albumin or cholesterol, and the flexibility to be used in conjunction with Fc-containing biologics (unlike other IgG lowering agents). Additionally, BHV-1300 is unique with its selective removal of targeted IgG (IgG1, IgG2 and IgG4 over IgG3). IgG3 plays a critical role in the immune response and in activating effector functions crucial for combating bacteria, parasites, and viruses. BHV-1300 was designed to spare IgG3 to avoid the broad immunosuppression associated with other IgG lowering approaches and to allow for an immune response to combat potential infections while delivering therapeutic actions in autoimmune disease. Results of the ongoing Phase 1 trial validate the selectivity of BHV-1300 and the adaptability of the platform to engage targets of interest with precision. In addition to the new Phase 1 data with BHV-1300, Biohaven also announced regulatory acceptance of three INDs and/or CTAs for its next-generation MoDE molecules to target other immune mediated diseases. Two of these novel MoDEs, BHV-1400 and BHV-1600, represent the platform's first autoantibody specific degraders, sparing the body's healthy antibodies (IgG, IgA, etc.) to function normally while clearing disease-causing antibodies. Dosing in humans has been initiated for BHV-1400, a novel IgA nephropathy (IgAN) investigational therapy designed to selectively degrade galactose deficient IgA1 (Gd-IgA1) without immunosuppression. IgAN is a rare disease affecting approximately 60,000 individuals in the United States. It is often diagnosed in the second or third decade of life, progresses over decades, and can result in kidney failure leading to the need for hemodialysis. Approved therapies broadly suppress the immune system or target the downstream consequences of immune damage without targeting the autoimmune cause of disease. For a disease which is diagnosed in young adults and treatment may be required over the lifespan, BHV-1400 is highly differentiated, engineered to clear the pathogenic nidus of disease, Gd-IgA1, and preserve the individual's immunoglobulins (IgG, IgA, IgE, IgM), immune cells, and complement system (Figure 2). Thus, BHV-1400 is expected to result in less potential for respiratory, mucosal or central nervous system infections compared to broader IgA lowering or immunosuppressive strategies in development. Additionally, IgAN clinical trials have a well-established pathway for accelerated approval. Biohaven also initiated dosing in humans with BHV-1600 in 4Q 2024. BHV-1600 is a MoDE engineered to rapidly degrade pathogenic autoantibodies to the b1-adrenergic receptor (b1AR). BHV-1600 is the first rationally designed investigative treatment in development for peripartum cardiomyopathy (PPCM), a rare type of heart failure that occurs during pregnancy or soon after birth that has no currently approved therapy. In b1AR autoantibody-driven cardiomyopathy, autoantibodies bind b1AR and induce cardiomyocyte toxicity and heart failure (Figure 3).1 PPCM affects mothers at an incredibly vulnerable period: previously healthy, individuals with PPCM develop profound heart failure, struggling with new symptoms as their child experiences its first days and often first years of life. Without disease-specific therapies, women with PPCM can develop heart failure and may emergently require left ventricular assistive devices or heart transplant. Given PPCM is a life-threatening disease with no current treatment and the potential for BHV-1600 to rapidly degrade pathogenic b1AR autoantibodies, Biohaven completed an INTERACT meeting with the FDA in 4Q 2024 and gained alignment on a clinical development program to pursue an accelerated approval pathway for BHV-1600 in PPCM. An IND has also been accepted for BHV-1310, an optimized and selective IgG1, IgG2, and IgG4 degrader. Dosing is expected in humans in 1Q 2025. In addition to key updates in the MoDE clinical development program, Biohaven also announced critically important advances related to its late-stage glutamate and ion channel platforms. A new drug application (NDA) was submitted to the US FDA for troriluzole in the treatment of all genotypes of spinocerebellar ataxia (SCA), following the completion of a pre-NDA meeting in 4Q 2024. Troriluzole was previously granted Orphan Drug and Fast-Track designations by FDA, and qualifies for potential Priority Review. Biohaven recently reported positive topline pivotal results in SCA in September 2024, demonstrating that troriluzole slowed disease progression by 50-70% over the 3-year study period. SCA is a rare, life-threatening, progressively debilitating neurodegenerative disease that affects approximately 15,000 people in the US, and 24,000 in Europe and the United Kingdom. Troriluzole has been safe and well-tolerated in over 8 years of clinical trial experience. There are no FDA approved treatments for SCA and troriluzole is the first investigational agent to show disease slowing in its clinical development program. Biohaven further announced the completion of enrollment in a pivotal BHV-7000 Phase 2/3 trial in bipolar disorder in 4Q 2024. BHV-7000 is a selective activator of Kv7 potassium channels that offers a novel and compelling mechanism of action for the treatment of bipolar disorder and an excellent tolerability at all doses evaluated in previous studies without the central nervous system adverse effects, such as somnolence and other CNS-related effects, that typically limit the use of other mood stabilizing medications. The trial completed enrollment several months ahead of anticipated timelines, reflecting the high unmet need for new treatments in bipolar disorder. The Phase 2/3 double-blind, placebo-controlled study enrolled approximately 256 patients. Patients were randomized to receive BHV-7000 75 mg once daily or placebo over a 3-week treatment period. The primary outcome measure of the study is the change from baseline to week 3 in mania symptom severity, as measured by the Young Mania Rating Scale (YMRS). Secondary objectives include response and remission rates, early onset of efficacy, depression symptom severity as measured by the Montgomery-Åsberg Depression Rating Scale (MADRS), and safety. Irfan Qureshi MD, Chief Medical Officer of Biohaven, commented, "Biohaven continues to propel cutting edge science into reality in the clinic. Biohaven's proprietary MoDE degrader platform is a technology that has the potential to revolutionize precision treatment of a spectrum of common and rare autoimmune diseases. Targeted removal of IgG, b1AR autoantibodies and galactose deficient IgA1 has the potential to transform the treatment of rheumatoid arthritis, myasthenia gravis, autoimmune cardiomyopathy, IgAN, and many other immune mediated disorders. While advancing new compounds in immunology and inflammation, our R&D teams have also delivered important milestones for our ion channel and glutamate modulating agents. We are proud to have completed enrollment in the pivotal bipolar study with BHV-7000, months ahead of timelines and eagerly await topline results. And, our NDA submission for spinocerebellar ataxia represents the culmination of over 8 years of innovation, research, and commitment to patients with this devastating neurodegenerative disease. We believe troriluzole is a transformative breakthrough that offers new hope to individuals and families suffering from SCA who currently have no therapeutic options to alter the course of this relentless disease." Dr. Qureshi added, "We are extremely excited about the rapid progress across the portfolio and optimistic about the opportunities we have to make a positive impact on patients' lives and create value in 2025 and beyond." About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and nonclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA (spinocerebellar ataxia); myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. For more information, visit . Forward-looking Statements This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. MoDE is a trademark of Biohaven Therapeutics Ltd. Investor Contact: Jennifer Porcelli Vice President, Investor Relations [email protected] +1 (201) 248-0741 Media Contact: Mike Beyer Sam Brown Inc. [email protected] +1 (312) 961-2502 SOURCE Biohaven Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 1Fast TrackOrphan DrugBreakthrough Therapy

12 Nov 2024

·ir.biohaven.com

Biohaven Reports Third Quarter 2024 Financial Results and Recent Business Developments

Cash, cash equivalents, marketable securities and restricted cash as of October 2, 2024 totaled approximately $642 million Achieved positive topline trial results from pivotal trial with troriluzole in spinocerebellar ataxia (SCA) Troriluzole 200 mg QD dosed orally in patients with SCA met the study's primary endpoint on the change from baseline on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at 3 years in all study population genotypes Statistically significant superiority achieved on 9 consecutive, prespecified primary and secondary endpoints Both the study protocol and statistical analysis plan were submitted to, and reviewed by, the U.S. Food and Drug Administration (FDA) prior to topline data analysis Study designed in discussion with the FDA and utilized Phase 3 data and an external control of matched, untreated SCA subjects from the U.S. Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) in accordance with the FDA's Guidance on Real-World Evidence (RWE) of effectiveness CRC-SCA external control included contemporaneous natural history data gathered from 2010-2024 Planned New Drug Application (NDA) re-submission in 4Q 2024 Completed clarification meeting with CHMP Rapporteurs in 4Q 2024 and MAA documents are being updated to include the new positive BHV4157-206-RWE study data with broader indication to include all SCA genotypes Taldefgrobep alfa, a myostatin-inhibitor, Phase 3 topline data in spinal muscular atrophy (SMA) in 4Q 2024 and Phase 2 trial protocol in obesity expected in 4Q 2024 Advancing extracellular Molecular Degrader of Extracellular Protein (MoDE) programs 3 additional investigational agents expected to enter Phase 1 studies in the next quarter Anticipate Phase 1 update for BHV-1300, including subcutaneous formulation, before year-end Broad progress with TRPM3 antagonist Initiated pivotal Phase 2 trial evaluating BHV-2100, a TRPM3 antagonist, in the acute treatment of migraine Initiated separate proof of concept study with BHV-2100 in neuropathic pain Patient enrollment continues across 5 Phase 2/3 trials with Kv7 activator, BHV-7000, in epilepsy and mood disorders (bipolar and major depressive disorder (MDD)) with potential for multiple data readouts in 2025 Continued progress with antibody drug conjugate (ADC) portfolio BHV-1510 currently dosing cancer patients in Phase 1/2 study, advancing towards combination dosing of BHV-1510 with Libtayo® in 4Q 2024 Troriluzole 200 mg QD dosed orally in patients with SCA met the study's primary endpoint on the change from baseline on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at 3 years in all study population genotypes Statistically significant superiority achieved on 9 consecutive, prespecified primary and secondary endpoints Both the study protocol and statistical analysis plan were submitted to, and reviewed by, the U.S. Food and Drug Administration (FDA) prior to topline data analysis Study designed in discussion with the FDA and utilized Phase 3 data and an external control of matched, untreated SCA subjects from the U.S. Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) in accordance with the FDA's Guidance on Real-World Evidence (RWE) of effectiveness CRC-SCA external control included contemporaneous natural history data gathered from 2010-2024 Planned New Drug Application (NDA) re-submission in 4Q 2024 Completed clarification meeting with CHMP Rapporteurs in 4Q 2024 and MAA documents are being updated to include the new positive BHV4157-206-RWE study data with broader indication to include all SCA genotypes 3 additional investigational agents expected to enter Phase 1 studies in the next quarter Anticipate Phase 1 update for BHV-1300, including subcutaneous formulation, before year-end Initiated pivotal Phase 2 trial evaluating BHV-2100, a TRPM3 antagonist, in the acute treatment of migraine Initiated separate proof of concept study with BHV-2100 in neuropathic pain BHV-1510 currently dosing cancer patients in Phase 1/2 study, advancing towards combination dosing of BHV-1510 with Libtayo® in 4Q 2024 NEW HAVEN, Conn. , Nov. 12, 2024 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ( Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, today reported financial results for the third quarter ended September 30, 2024 , and provided a review of recent accomplishments and anticipated upcoming developments. Vlad Coric , M.D., Chairman and Chief Executive Officer of Biohaven , commented, "The team at Biohaven continues to advance multiple late and early stage assets that have the potential to change the current standard of care treatment paradigm across a number of diseases. Last quarter we announced positive topline results in our RWE trial assessing troriluzole for the treatment of spinocerebellar ataxia. Troriluzole (200 mg dosed orally) met the study's primary endpoint on the change from baseline in the f-SARA at 3 years in all study population genotypes, showed statistically significant superiority after both 1 and 2 years of treatment, and achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints. This was truly a watershed moment for the Company, given the implications of potentially bending the arc of one of the most intractable neurological diseases with no currently approved treatment options. SCA robs patients of their ability to speak, walk, and take care of themselves, and often shortens lifespans and has unfortunate intergenerational implications. We were encouraged by the robust dataset gathered to date and look forward to submitting an NDA to the FDA." Dr. Coric continued, "We continue to execute across our broad pipeline including the recent initiation of our Phase 2 trial with BHV-2100 in acute migraine. Migraine burden and disability remain high despite advances in treatment; we believe our TRPM3 antagonistic approach has the potential to be a highly-effective, non-sedating, non-opioid treatment for pain and migraine. In the final months of 2024, we expect to report on a number of updates including SMA topline data and data across our MoDE™ platform including a SAD/MAD update and multiple INDs spanning β1AR for the potential treatment of dilated cardiomyopathy, galactose deficient IgA for IgA nephropathy and a further optimized IgG degrader for use in rare diseases." Third Quarter 2024 and Recent Business Highlights Achieved positive topline results in pivotal study of troriluzole in SCA - In September 2024 , the Company announced positive topline results from pivotal Study BHV4157-206-RWE demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2. Additionally, troriluzole achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints. SCA patients treated with troriluzole showed a 50-70% slowing of disease progression, representing 1.5-2.2 years delay in disease progression over the 3-year study period. The Company intends to submit an NDA to the FDA for troriluzole in the treatment of all SCA genotypes in 4Q 2024. The development program for troriluzole has been granted orphan and fast track designations, and is eligible for priority review. European Medicines Agency marketing authorization remains under review and Biohaven completed a clarification meeting with CHMP Rapporteurs in 4Q 2024. MAA documents are being updated to include the new positive BHV4157-206-RWE study data with broader indication to include all SCA genotypes. Initiated Phase 2 trial evaluating BHV-2100 in the acute treatment of migraine - In September 2024 , the Company initiated a Phase 2 study of an orally administered TRPM3 antagonist, BHV-2100, in the acute treatment of migraine. The study is a randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of two doses (75 mg and 150 mg) of BHV-2100 in the acute treatment of migraine. The trial is designed to support registration with FDA-accepted co-primary endpoints of pain freedom and freedom from most bothersome symptom at 2 hours and is expected to enroll approximately 575 patients across 60 sites in the United States . Public offering - On October 2, 2024 , the Company closed its previously announced underwritten public offering of 6,052,631 of its common shares, which included the full exercise of the underwriters' option to purchase additional shares, at the public offering price of $47.50 per share. The net proceeds raised in the offering, after deducting underwriting discounts and estimated expenses of the offering payable by the Company, were approximately $269.9M . As of November 8, 2024 , we had 101,122,246 common shares outstanding. Expected Upcoming Milestones: We believe Biohaven is well positioned to achieve significant milestones in 2024 and 2025 across numerous programs: Selective Kv7 Activator: Continue 5 ongoing Phase 2/3 trials with BHV-7000 in focal epilepsy, idiopathic generalized epilepsy, MDD, and bipolar disorder Troriluzole: NDA submission to FDA on track for 4Q 2024, following release of pivotal topline results in SCA in September 2024 Two Phase 3 trials with troriluzole in OCD; expect to conduct interim analysis of the second Phase 3 OCD trial in 4Q 2024 and report topline data from first Phase 3 OCD trial in 1H 2025 Taldefgrobep alfa: Report topline data from Phase 3 trial with taldefgrobep in SMA in 4Q 2024 Initiate Phase 2 trial with taldefgrobep in obesity in 4Q 2024 or early 2025 First-in-class TRPM3 Antagonist: Continue advancing enrollment in Phase 2 trial with BHV-2100 in acute migraine and neuropathic pain (laser-evoked potential experimental pain paradigm) TYK2/JAK1 Inhibitor: Complete SAD/MAD studies with BHV-8000 and advance to Phase 2 in the coming months MoDE™ Platform Submit a total of 4 INDs in 2024 Continue to advance Phase 1 SAD and MAD studies with subcutaneous BHV-1300, with a further study update in 4Q 2024 Next Generation ADC Platform: Advance Phase 1 Trop-2 directed program BHV-1510 in multiple tumor types Capital Position: Cash, cash equivalents, marketable securities and restricted cash as of October 2, 2024 totaled approximately $642 million , which includes net proceeds of $269.9 million from the public offering of 6,052,631 common shares completed on October 2, 2024 . Third Quarter 2024 Financial Highlights: Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $157.6 million for the three months ended September 30, 2024 , compared to $95.5 million for the three months ended September 30, 2023 . The increase of $62.1 million was due to additional and advancing clinical trials, including late Phase 3 and Phase 2/3 studies, and preclinical research programs in 2024, as compared to the same period in the prior year. Non-cash share-based compensation expense was $7.2 million for the three months ended September 30, 2024 , an increase of $5.0 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the third quarter of 2024, primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024. General and Administrative (G&A) Expenses: G&A expenses were $20.6 million for the three months ended September 30, 2024 , compared to $15.0 million for the three months ended September 30, 2023 . The increase of $5.5 million was partly due to increased non-cash share-based compensation expense, which was $5.0 million for the three months ended September 30, 2024 , an increase of $2.7 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the third quarter of 2024 primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024. Other Income, Net: Other income, net was $17.8 million for the three months ended September 30, 2024 , compared to other income, net of $4.7 million for the three months ended September 30, 2023 . The increase of $13.1 million was primarily due to non-cash changes in the fair value of our derivative liabilities recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment), as well as increased investment income, partially offset by changes in the fair value of our forward contract liability recorded in connection with the Knopp Amendment. Net Loss: Biohaven reported a net loss for the three months ended September 30, 2024 of $160.3 million , or $1.70 per share, compared to $102.6 million , or $1.50 per share, for the same period in 2023. Non-GAAP adjusted net loss for the three months ended September 30, 2024 was $164.1 million , or $1.74 per share, compared to $98.1 million , or $1.44 per share for the same period in 2023. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below. Non-GAAP Financial MeasuresThis press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, which are adjusted to exclude non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares, and changes in the fair value of derivative liabilities, which do not correlate to actual cash payment obligations in the relevant periods. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance and can assist investors in comparing Biohaven's performance between periods. In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this news release. About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. Forward-looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first in class therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission , including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. BIOHAVEN LTD. CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share amounts) (Unaudited) Three Months Ended September 30 , Nine Months Ended September30, 2024 2023 2024 2023 Operating expenses: Research and development $ 157,607 $ 95,517 $ 628,398 $ 238,468 General and administrative 20,561 15,030 66,782 43,872 Total operating expenses 178,168 110,547 695,180 282,340 Loss from operations (178,168) (110,547) (695,180) (282,340) Other income, net 17,805 4,686 36,288 18,757 Loss before (benefit) provision for income taxes (160,363) (105,861) (658,892) (263,583) (Benefit) provision for income taxes (59) (3,287) 687 (171) Net loss $ (160,304) $ (102,574) $ (659,579) $ (263,412) Net loss per share — basic and diluted $ (1.70) $ (1.50) $ (7.50) $ (3.86) Weighted average common shares outstanding— basic and diluted 94,372,159 68,320,125 87,936,923 68,258,757 BIOHAVEN LTD. CONSOLIDATED BALANCE SHEETS (Amounts in thousands, except share amounts) September 30, 2024 December 31, 2023 (Unaudited) Assets Current assets: Cash and cash equivalents $ 84,390 $ 248,402 Marketable securities 294,426 133,417 Prepaid expenses 55,168 35,242 Income tax receivable 5,318 13,252 Other current assets 1,198 12,133 Total current assets 440,500 442,446 Property and equipment, net 18,276 17,191 Intangible assets 18,400 18,400 Goodwill 1,390 1,390 Other non-current assets 31,957 33,785 Total assets $ 510,523 $ 513,212 Liabilities and Shareholders' Equity Current liabilities: Accounts payable $ 19,744 $ 15,577 Accrued expenses and other current liabilities 63,520 39,846 Forward contract liability 69,030 — Total current liabilities 152,294 55,423 Non-current operating lease liabilities 25,312 27,569 Derivative liability, non-current 12,320 — Other non-current liabilities 4,591 2,245 Total liabilities 194,517 85,237 Shareholders' Equity: Preferred shares, no par value; 10,000,000 shares authorized, no shares issuedand outstanding as of September 30, 2024 and December 31, 2023 — — Common shares, no par value; 200,000,000 shares authorized as of September30, 2024 and December 31, 2023 ; 94,899,193 and 81,115,723 shares issuedand outstanding as of September 30, 2024 and December 31, 2023 ,respectively 1,381,699 887,528 Additional paid-in capital 93,038 39,804 Accumulated deficit (1,158,871) (499,292) Accumulated other comprehensive income (loss) 140 (65) Total shareholders' equity 316,006 427,975 Total liabilities and shareholders' equity $ 510,523 $ 513,212 BIOHAVEN LTD. RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL MEASURES (Amounts in thousands, except share and per share amounts) (Unaudited) Three Months Ended September 30 , Nine Months Ended September30, 2024 2023 2024 2023 Reconciliation of GAAP to Non-GAAP adjusted net loss: GAAP net loss $ (160,304) $ (102,574) $ (659,579) $ (263,412) Add: non-cash share-based compensation expense 12,160 4,456 59,269 12,916 Add: (gain) loss from change in fair value of derivatives (15,990) — (17,030) — Non-GAAP adjusted net loss $ (164,134) $ (98,118) $ (617,340) $ (250,496) Reconciliation of GAAP to Non-GAAP adjusted net loss per share — basic and diluted: GAAP net loss per share — basic and diluted $ (1.70) $ (1.50) $ (7.50) $ (3.86) Add: non-cash share-based compensation expense 0.13 0.07 0.67 0.20 Add: (gain) loss from change in fair value of derivatives (0.17) — (0.19) — Non-GAAP adjusted net loss per share — basic and diluted $ (1.74) $ (1.44) $ (7.02) $ (3.67) MoDEs is a trademark of Biohaven Therapeutics Ltd. Libtayo is a registered trademark of Regeneron Pharmaceuticals, Inc. Investor Contact: Jennifer Porcelli Vice President, Investor Relationsjennifer.porcelli@biohavenpharma.com+1 (201) 248-0741 Media Contact: Mike Beyer Sam Brown Inc. mikebeyer@sambrown.com+1 (312) 961-2502 View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-reports-third-quarter-2024-financial-results-and-recent-business-developments-302303249.html SOURCE Biohaven Ltd.

Phase 2Clinical ResultFast TrackFinancial StatementNDA

24 Sep 2024

·www.biospace.com

Biohaven Sets Stage for Comeback With Pivotal Study Results in Spinocerebellar Ataxia

iStock, Prostock-Studio The FDA previously refused to review Biohaven’s candidate in the indication due to a failed late-stage trial. However, the company is now planning to NDA in the fourth quarter of 2024. Biohaven on Monday unveiled topline pivotal data for its drug candidate troriluzole, touting significant symptomatic and functional improvements in patients with spinocerebellar ataxia. Troriluzole treatment led to “robust and clinically meaningful slowing of disease progression” with the investigational treatment slowing down patients’ rate of decline by 50% to 70% versus untreated comparators, according to the company, while delaying disease progression by 1.5 years to 2.2 years over the study’s three-year duration. Using the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) tool, which classifies disease progression as at least a two-point worsening in score, Biohaven found that untreated patients were more than four times more likely to progress, compared with patients on troriluzole. CEO Vlad Coric in a statement said that Biohaven’s spinocerebellar ataxia (SCA) program, which includes Monday’s pivotal study, “has provided the first evidence of a clinically meaningful treatment benefit as well as slowing disease progression in SCA patients.” “The need for treatments for this deadly neurodegenerative disease is urgent,” Coric said. “We look forward to interacting with regulatory agencies to bring troriluzole to patients with SCA.” Biohaven is gearing up for a New Drug Application (NDA) in the fourth quarter of 2024, backed by the results from Monday’s readout, as well as previous efficacy and safety data. SCA refers to a group of heritable neurodegenerative disorders that typically involve the progressive loss of voluntary control over movement. The disease class also leads to the gradual atrophy of several crucial parts of their nervous system, including cerebellum, brainstem and spinal cord. Patients suffer from compromised motor skills, often leading to falls and other related injuries. SCA can also manifest as speech impairments, difficulties swallowing and, ultimately, premature death. There are currently no approved treatments for SCA. Biohaven’s troriluzole is an investigational modulator of the glutamate neurotransmitter, which is the most abundant excitatory messenger in humans. By boosting the uptake of glutamate from the synapse, it helps lower the overall levels of the neurotransmitter in the synaptic space, preventing the over-excitation of neurons and addressing a key pathway in SCA. However, despite a promising mechanism of action, troriluzole’s development has run into several rough patches in recent years. In August 2023, the FDA refused to review Biohaven’s NDA for troriluzole, pointing out that the study used to support the application failed its primary endpoint. The biotech announced the Phase III failure in May 2022 . Troriluzole has also run into difficulties beyond SCA. In January 2021, Biohaven unveiled disappointing Alzheimer’s disease data for the drug candidate, revealing that it had failed to significantly outperform placebo at 48 weeks. Nearly a year earlier, troriluzole failed a Phase III study in generalized anxiety disorder. Still, William Blair analyst Myles Minter is optimistic about troriluzole’s chances moving forward. Minter in a note to investors said that they are “encouraged by the consistent signals of benefit … in a population with extremely limited treatment options.” The firm expects the FDA to call for an advisory committee meeting to discuss the differences between Monday’s readout and the first failed Phase III study.

Phase 3NDA

100 Deals associated with Trigriluzole

External Link

KEGG	Wiki	ATC	Drug Bank
-	Trigriluzole	-	DB15079

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Machado-Joseph Disease	NDA/BLA	US	Biohaven Ltd.	31 May 2023
Obsessive-Compulsive Disorder	Phase 3	CN	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	CA	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	IT	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	NL	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	PR	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	ES	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Obsessive-Compulsive Disorder	Phase 3	GB	Biohaven Pharmaceuticals, Inc.	29 Jan 2021
Spinocerebellar Ataxias	Phase 3	CN	Bioshin Ltd.	20 Nov 2020
Glioblastoma Multiforme	Phase 3	US	Vigeo Therapeutics, Inc.	30 Jul 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NEWS Manual	Phase 3	Spinocerebellar Ataxias	-	Troriluzole	ojltnkikwy(hbgybktnqg) = Biohaven said that the once-daily oral drug met the primary endpoint across all genotypes of SCA included in the study and that it showed statistically significant superiority after both one and two years compared to the external control groups. orwkeutvtj (lfhbucwfto ) Met View more	Positive	23 Sep 2024
AAN2024	Not Applicable	-	-	Troriluzole 200 mg	cehgrcgxlz(oxfrjhxlsu) = values were 40% and 50% higher after troriluzole 100 mg and 280 mg, respectively vs oral riluzole 50 mg vsylmqrejy (pncilynfhq ) View more	-	09 Apr 2024
				Troriluzole 280 mg
AAN2024	Not Applicable	-	-	Troriluzole 140mg QD	swncowxvoo(ospomlvgnk) = cfpregbmph qgclkweqsh (lyadogzqnh ) View more	-	09 Apr 2024
				Troriluzole 200mg QD	swncowxvoo(ospomlvgnk) = whbootwqag qgclkweqsh (lyadogzqnh ) View more
NCT03701399 (Phase III Clinical Trial of Troriluzole, AAN2024)	Phase 3	Spinocerebellar Ataxias	218	Troriluzole	awuverhque(qzdrgovpkp): adj. interaction coefficient = 0.58 (95% CI, 0.14 - 1.03)	Positive	09 Apr 2024
				Placebo
NCT03605667 (T2 Protect AD, CTgov)	Phase 2	Alzheimer Disease	350	Placebo oral capsule	gyexuekxuv(lfgzsiedgi) = mvagsznamw ibgubbzeoi (vcgvggpdio, nbpwnmncyr - dixheyqrpl) View more	-	06 Dec 2023
NCT03299166 (CTgov)	Phase 2/3	Obsessive-Compulsive Disorder	426	Troriluzole (Troriluzole - Randomization Phase)	vpdzffbpek(fhwvnytwrq) = ilojxaxpdy nohwrxnszv (exlyamsemy, hacjyvoygs - ztebyjjktx) View more	-	02 Jun 2023
				placebo+troriluzole (Placebo - Randomization Phase)	vpdzffbpek(fhwvnytwrq) = vdbhanenfx nohwrxnszv (exlyamsemy, oujufxizwo - mogrdrclcn) View more
NCT03829241 (CTgov)	Phase 3	Generalized anxiety disorder	881	Troriluzole (Troriluzole)	htjrcqtuky(srqxdkotbm) = jdaunmtvqs qxkpjzlbns (xsxezattfx, pscxfoehjb - nzjqoiwgqx) View more	-	28 Dec 2022
				Placebo+Troriluzole (Placebo)	htjrcqtuky(srqxdkotbm) = vuneprlskb qxkpjzlbns (xsxezattfx, velwzeagbo - mtbzpdijlr) View more
NCT03701399 (NEWS) Manual	Phase 3	Spinocerebellar Ataxias	-	troriluzole	zieuklysnf(vixvcdcypa) = etudrlmvrw mwnwhibheu (yygmduybuz ) View more	Negative	23 May 2022
				placebo	zieuklysnf(vixvcdcypa) = ugalhdfrwj mwnwhibheu (yygmduybuz ) View more
NCT02960893 (CTgov)	Phase 2/3	Spinocerebellar Ataxia 10 \| Machado-Joseph Disease \| Spinocerebellar Ataxia 8	141	Troriluzole (Troriluzole - Randomization Phase)	hcaemgmiip(xpiqtvtsfg) = orbzoaxhxq okkqopkuvn (astiyveukp, ivrnbdmntp - ogxwmncenq) View more	-	14 Aug 2020
				placebo (Placebo - Randomization Phase)	hcaemgmiip(xpiqtvtsfg) = qawttjhlre okkqopkuvn (astiyveukp, rdvqvrpako - aqwaxzlywb) View more
NCT03829241 (PRNewswire) Manual	Phase 3	Generalized anxiety disorder	-	troriluzole	jckvfsdcfk(teacwbjyfd) = okvcjpsqqy cvqhqubwrx (rewnuodloj, -10.23 to -8.32) View more	Negative	10 Feb 2020
				Placebo	jckvfsdcfk(teacwbjyfd) = xkkclldowo cvqhqubwrx (rewnuodloj, -10.34 to -8.36) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis