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.