Vyglxia (troriluzole) / Biohaven - LARVOL DELTA

Troriluzole in Adult Participants With Spinocerebellar Ataxia (clinicaltrials.gov) - P3 | N=299 | Active, not recruiting | Sponsor: Biohaven Pharmaceuticals, Inc. | Trial completion date: Oct 2025 ➔ Jun 2026

Trial completion date • Ataxia • Movement Disorders

FDA Issues Complete Response Letter for Biohaven's VYGLXIA (troriluzole) New Drug Application for Spinocerebellar Ataxia (PRNewswire) - "The FDA issued a complete response letter (CRL) despite Study 206-RWE being reviewed by FDA and achieving statistical significance in the study's prespecified primary and secondary outcome efficacy endpoints. FDA cited issues that can be inherent to real-world evidence and external control studies including potential bias, design flaws, lack of pre-specification and unmeasured confounding factors....In the CRL, the FDA recommended that Biohaven meet with the Division to discuss the evidence that will be needed to support a future NDA for the treatment of SCA with troriluzole."

CRL • Ataxia

Troriluzole attenuates opioid intake, reinforcing efficacy, seeking behaviours, physical dependence and antinociceptive tolerance in rats. (PubMed, Br J Pharmacol) - "TRLZ, already in clinical trials for cerebellar ataxia, also reduced opioid taking and seeking as well as opioid-derived adverse effects in rats, supporting further study in treating opioid use disorders and pain management."

Journal • Preclinical • Addiction (Opioid and Alcohol) • Amyotrophic Lateral Sclerosis • Ataxia • CNS Disorders • Depression • Movement Disorders • Pain • Psychiatry • Substance Abuse

BED IN 46: Methamphetamine and Troriluzole (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: University of Kentucky | Not yet recruiting ➔ Recruiting

Enrollment open

Efficacy and Safety Study of Adjunctive Troriluzole in Obsessive Compulsive Disorder (clinicaltrials.gov) - P3 | N=700 | Active, not recruiting | Sponsor: Biohaven Pharmaceuticals, Inc. | Trial completion date: Jun 2025 ➔ Nov 2025 | Trial primary completion date: Jun 2025 ➔ Nov 2025 | Recruiting ➔ Active, not recruiting

Enrollment closed • Trial completion date • Trial primary completion date • Mood Disorders • Obsessive-Compulsive Disorder • Psychiatry

Efficacy and Safety Study of Adjunctive Troriluzule in Obsessive Compulsive Disorder (clinicaltrials.gov) - P3 | N=589 | Completed | Sponsor: Biohaven Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

Trial completion • Mood Disorders • Obsessive-Compulsive Disorder • Psychiatry

GBM AGILE: A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma (clinicaltrials.gov) - P2/3 | N=1280 | Recruiting | Sponsor: Global Coalition for Adaptive Research | Trial completion date: Jun 2028 ➔ Jun 2030 | Trial primary completion date: Jun 2026 ➔ Jun 2028

Trial completion date • Trial primary completion date • Brain Cancer • Glioblastoma • Hematological Disorders • Oncology • Solid Tumor

BED IN 46: Methamphetamine and Troriluzole (clinicaltrials.gov) - P1 | N=40 | Not yet recruiting | Sponsor: University of Kentucky

New P1 trial

Biohaven Reports First Quarter 2025 Financial Results and Recent Business Developments (PRNewswire) - "The Company completed a mid-cycle review meeting with the FDA of the NDA for troriluzole for the treatment of SCA. The FDA has not conveyed any intention of holding an Advisory Committee Meeting. The NDA had previously been accepted and granted priority review by the FDA with a Prescription Drug User Fee Act ('PDUFA') date expected in the third quarter of 2025....Pivotal topline data from two Phase 3 OCD trials expected in 1H 2025 and 2H 2025, respectively."

FDA event • P3 data: top line • PDUFA • Ataxia • Obsessive-Compulsive Disorder

FDA Extends PDUFA Date of Biohaven's Troriluzole NDA for Rare Disease Spinocerebellar Ataxia (PRNewswire) - "Biohaven...announced that the Division of Neurology 1 within FDA's Office of Neuroscience informed the Company that they are extending the PDUFA date for the troriluzole new drug application (NDA) for the treatment of spinocerebellar ataxia (SCA) by three months to provide time for a full review of Biohaven's recent submissions related to information requests from the FDA. The Division also informed Biohaven that it is currently planning to hold an advisory committee meeting to discuss the application, but no date has been scheduled. The FDA did not raise any new concerns in the letter. The FDA's decision regarding the NDA is now expected in 4Q 2025."

PDUFA • Ataxia

Prognostic value of plasma biomarkers for informing clinical trial design in mild-to-moderate Alzheimer's disease. (PubMed, Alzheimers Res Ther) - P2 | "Elevated baseline plasma NfL predicted more rapid clinical decline and MRI volume loss. Furthermore, increasing plasma NfL concentration over time was associated with worsening on the CDR-SB. Plasma NfL is an easily accessible biomarker that may enhance the design of clinical trials in mild-to-moderate AD."

Biomarker • Journal • Alzheimer's Disease • CNS Disorders • Aβ42 • GFAP • Plasma NfL • p-tau181

Withdrawal of applications (European Medicines Agency) - "An application for an initial marketing authorisation was withdrawn. Dazluma (troriluzole hydrochloride monohydrate) was intended for the treatment of spinocerebellar ataxia genotype 3 (SCA3), an inherited brain disorder that affects coordination and balance....An application to extend the therapeutic indication of Ngenla (somatrogon) in the treatment of adults with growth hormone deficiency was also withdrawn."

CHMP • Ataxia • Growth Hormone Deficiency (Adult)

A Surgical Window of Opportunity Clinical Trial of Troriluzole in Recurrent IDH Wild-Type Glioblastoma (clinicaltrials.gov) - P1 | N=27 | Recruiting | Sponsor: Eudocia Quant Lee, MD | Not yet recruiting ➔ Recruiting

Enrollment open • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Biohaven Reports Recent Business Developments and Fourth Quarter and Full Year 2024 Financial Results (PRNewswire) - "Glutamate Modulator (Troriluzole)...Topline data from two Phase 3 OCD trials in 1H 2025 and 2H 2025, respectively. Myostatin (Taldefgrobep alfa): Expect FDA meeting to discuss SMA registrational path in 1H 2025. Initiate taldefgrobep Phase 2 study in obesity in 1H 2025. Continue advancing enrollment in proof of concept trial with BHV-2100 in acute migraine; data from the laser-evoked potential study expected in 1H 2025...Initiate BHV-8000 Phase 2/3 study in Parkinson's disease in 1H 2025."

FDA event • New P2 trial • New P2/3 trial • P2 data • P3 data • Migraine • Muscular Atrophy • Obesity • Obsessive-Compulsive Disorder • Parkinson's Disease

Comparative Effectiveness of Troriluzole Versus Untreated Natural History Cohorts in Spinocerebellar Ataxia Leveraging Propensity Score Matching Methods (AAN 2025) - P, P3 | "Compelling and sustained treatment effects were observed out to 3 years when troriluzole-treated subjects were compared to 3 different matched untreated natural history cohorts, supporting that long-term daily dosing of troriluzole attenuates the progression of disease among SCA subjects."

HEOR • Ataxia • CNS Disorders • Movement Disorders

Troriluzole in Adult Participants With Spinocerebellar Ataxia (clinicaltrials.gov) - P3 | N=299 | Active, not recruiting | Sponsor: Biohaven Pharmaceuticals, Inc. | N=218 ➔ 299

Enrollment change • Ataxia • Movement Disorders

Biohaven Announces FDA Acceptance and Priority Review of Troriluzole New Drug Application for the Treatment of Spinocerebellar Ataxia (PRNewswire) - "Biohaven Ltd...announced that the US Food and Drug Administration (FDA) has accepted for review the Company's New Drug Application (NDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia (SCA) and has granted Priority Review....The FDA's decision regarding the NDA is expected within 6 months of filing (during 3Q2025). Based on FDA Priority Review timelines and if ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025....The NDA submission was based, in part, on positive topline results from Study BHV4157-206-RWE (NCT06529146)...The NDA also includes confirmatory and supportive data from Studies BHV4157-201 and BHV4157-206..."

FDA approval • FDA filing • Launch US • Priority review • Ataxia

Long-term Safety Study of Adjunctive Troriluzole in Subjects With Obsessive Compulsive Disorder (clinicaltrials.gov) - P3 | N=1200 | Enrolling by invitation | Sponsor: Biohaven Pharmaceuticals, Inc. | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Mood Disorders • Obsessive-Compulsive Disorder • Psychiatry

Efficacy and Safety Study of Adjunctive Troriluzule in Obsessive Compulsive Disorder (clinicaltrials.gov) - P3 | N=700 | Active, not recruiting | Sponsor: Biohaven Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Mood Disorders • Obsessive-Compulsive Disorder • Psychiatry

Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia. (PubMed, Neurol Ther) - P3 | "Psychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3."

Journal • Ataxia • Friedreich ataxia • Movement Disorders

Efficacy and Safety Study of Adjunctive Troriluzole in Obsessive Compulsive Disorder (clinicaltrials.gov) - P3 | N=700 | Recruiting | Sponsor: Biohaven Pharmaceuticals, Inc. | Trial completion date: Dec 2024 ➔ Jun 2025 | Trial primary completion date: Dec 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Mood Disorders • Obsessive-Compulsive Disorder • Psychiatry

Comparative Effectiveness of Troriluzole versus Untreated Natural History Cohorts in Spinocerebellar Ataxia Leveraging Propensity Score Matching Methods (ICAR-Ataxia 2024) - No abstract available

HEOR • Late-breaking abstract • Ataxia • Movement Disorders

Comparison of Two Matching Methods to Assess Effectiveness of Troriluzole versus Untreated Natural History Cohort in Spinocerebellar Ataxia (ICAR-Ataxia 2024) - No abstract available

Ataxia • Movement Disorders

A Systematic Literature Review on the Efficacy of Pharmacological Interventions in Ataxia (ISPOR-EU 2024) - "Across these, idebenone and riluzole (2 studies each) were the most commonly assessed interventions, followed by one study each for amantadine hydrochloride, deferiprone, luvadaxistat, omaveloxolone, rovatirelin, and troriluzole. Limited therapeutic options are available for ataxia, often focusing on symptomatic management rather than addressing the underlying cause. This SLR underscores a notable gap in treatments that directly target ataxia and highlights the potential pharmacological treatments in treating ataxia or slowing its progression."

Clinical • Review • Ataxia • CNS Disorders • Friedreich ataxia • Movement Disorders

Trial in Adult Subjects With Spinocerebellar Ataxia (clinicaltrials.gov) - P2/3 | N=141 | Completed | Sponsor: Biohaven Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

Trial completion • Ataxia • Movement Disorders