• Presents progress and new anticipated milestones across portfolio of more than 10 assets and 6 therapeutic areas.
• Announces multiple advancements across the MoDETM (molecular degraders of extracellular proteins) platform and the next generation TRAPTM (targeted removal of aberrant protein) degraders, including:
  • IgA Nephropathy (IgAN) program: First-in-human dosing with BHV-1400, a next generation TRAP degrader, achieved rapi­d, deep, and selective lowering of only aberrant galactose-deficient IgA1 (Gd-IgA1), the antibody causing IgA nephropathy, while sparing normal IgA.
    • This selective and rapid approach to immunoglobulin lowering represents a second-generation therapeutic approach to IgAN, potentially allowing for strong disease control with less acute or long-term safety risks associated with complement inhibition or broad antibody suppression.
    • The first and lowest dose tested (125mg) of BHV-1400 in the ongoing Phase 1 achieved rapid lowering of Gd-IgA1 with a median reduction of 60% within four hours of administration after a single dose. Maximal reduction exceeding 70% was observed within eight hours. Reductions were sustained for days even after a single dose. This rapid reduction of Gd-IgA1 is unprecedented in drugs targeting Gd-IgA1 and could allow for potential indications in situations where rapid Gd-IgA1 lowering could be beneficial in addition to chronic active disease and long-term maintenance.
    • BHV-1400 has been safe and well-tolerated in the Phase 1 study to date and demonstrated no clinically significant changes in innate or adaptive immunity, including white blood cells and immunoglobulins IgG, IgA, IgE, and IgM. There were no clinically significant reductions in albumin, liver function test abnormalities, or increases in cholesterol compared to baseline.
    • No dose limiting toxicities have been observed in the Phase 1 to date and further dose escalation is planned to explore the full range of Gd-IgA1 reductions possible with BHV-1400.
    • A pivotal trial in IgA nephropathy is planned using an accelerated regulatory path forward upon completion of the Phase 1 trial.
  • Peripartum cardiomyopathy (PPCM) program: First-in-human dosing with BHV-1600, a TRAP degrader of β1AR autoantibodies, was initiated and has been well-tolerated to date after the first two dose cohorts without clinically relevant changes in innate or adaptive immunity, including white blood cells and immunoglobulins IgG, IgA, IgE, and IgM. There were no clinically significant reductions in albumin, liver function test abnormalities, or increases in cholesterol compared to baseline. β1AR autoantibodies are thought to cause peripartum cardiomyopathy, a rare form of heart failure that develops at the end of pregnancy or in the months following delivery and in severe cases, can be life-threatening. BHV-1600 has been shown to bind to β1AR autoantibodies in preclinical studies and biomarker levels will be measured in women with PPCM later in development.
    • Completed INTERACT meeting with FDA regarding BHV-1600 in 4Q 2024; gained alignment for the study design to potentially pursue an accelerated approval pathway in PPCM.
  • IgG degrader programs:
    • BHV-1300: Advanced optimized subcutaneous formulation with deep reductions of targeted IgG >60% demonstrated in the lowest dose cohort of the MAD, in line with modeling projections and with differentiated safety profile. There were no clinically significant reductions in IgG3, IgA, IgE, IgM, or albumin, no liver function test abnormalities, nor increases in cholesterol compared to baseline.
    • Phase 1 completing last remaining dose cohorts with the optimized subcutaneous formulation in 1H 2025.
    • Lead indication for BHV-1300 announced in Graves' Disease, a common autoimmune disorder affecting approximately 80 million people globally. Graves' Disease is caused by IgG1 autoantibodies that hyperstimulate the TSH receptor, causing hyperthyroidism can result in the need for surgical removal, chemical ablation of the thyroid, or need for chronic anti-thyroid drugs. Additional programs in rheumatoid arthritis and myasthenia gravis also continue to be pursued with BHV-1300 and 1310.
    • Study May Proceed letter received from FDA for BHV-1310 IND, a novel IgG degrader and Phase 1 initiating in 1H 2025. BHV-1300 and BHV-1310 are similar but will optimize therapeutic targeting and facilitate broader commercial development options.
  • Next generation degrader targets advancing in 2025 include: IgG4 specific degrader, PLA2R autoantibody degrader for membranous nephropathy, pro-insulin autoantibody degrader for type 1 diabetes, and TSH receptor autoantibody degrader as a selective follow-on asset for Graves' Disease.
• Progresses ion channel programs for bipolar, depression, epilepsy and pain:
  • BHV-7000, a selective activator of Kv7.2/7.3 potassium channels: Continues to advance in clinical trials for bipolar disorder, depression, and epilepsy with multiple readouts over the next year, including expected topline for bipolar in 1H 2025 and depression in 2H 2025.
  • BHV-2100, a TRPM3 ion channel antagonist: Preliminary data from an innovative laser-evoked hyperalgesia study with BHV-2100 reduced laser heat-induced pain and brain evoked potentials in healthy volunteers. These exciting results with BHV-2100 represent the first clinical proof of concept for the novel TRPM3 inhibitor class in pain, recapitulating powerful preclinical evidence of efficacy for this approach across a spectrum of pain disorders.
• Highlights expansion of Biohaven's antibody drug conjugate (ADC) portfolio in Oncology, including:
  • BHV-1510, a Trop2-directed ADC using a novel topoisomerase 1 (TopoIx) payload: Demonstration of early Phase 1 clinical activity, including tumor shrinkage, and safety with BHV-1510 in advanced or metastatic epithelial tumors.
    • Based upon these results, Biohaven has entered into an expanded collaboration agreement with GeneQuantum providing exclusivity to the TopoIx payload for up to 18 ADC targets.
  • BHV-1530, a FGFR3-directed ADC using the TopoIx payload: US IND clearance received for BHV-1530, potential indications include urothelial cancers and other solid tumors driven by FGFR3 alterations and/or upregulated FGFR3 protein expression. The first-in-human study is planned to commence in 1H 2025.
  • Announced a multi-target collaboration with Merus, an oncology-focused biotechnology company developing innovative, multi-specific (Biclonics® and Triclonics®) antibodies, to co-develop three programs encompassing highly differentiated next generation dual-targeted bispecific ADCs leveraging Biohaven's proprietary conjugation and payload technologies and Merus' leading Biclonics technology platform.
• Key late-stage programs advancing across important indications in 2025 also include:
  • BHV-8000, a highly selective, brain-penetrant TYK2/JAK1 inhibitor that avoids class risks associated with JAK2/3 inhibition, expected to initiate pivotal trials in Parkinson's and Alzheimer's disease in 2025.
  • Taldefgrobep alfa, a novel myostatin inhibitor optimized to block two key regulators of muscle and fat metabolism, continues to be developed in spinal muscular atrophy and obesity.
  • New Drug Application (NDA) for troriluzole in all SCA genotypes was submitted to the FDA, following completion of pre-NDA meeting in 4Q 2024. Troriluzole has Orphan Drug and Fast-Track designations and qualifies for potential Priority Review. Awaiting filing decision from FDA and preparing for potential commercial launch in 2025 if approved.

NEW HAVEN, Conn., Jan. 13, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE:BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative and transformative therapies to treat individuals with rare and common diseases, today highlighted broad portfolio progress at the 43rd Annual J.P. Morgan Healthcare Conference, including positive Phase 1 data for BHV-1400, its highly differentiated investigational therapeutic for IgA nephropathy. BHV-1400 is a second generation TRAP™ degrader from its proprietary MoDE™ platform. A copy of the slide presentation is available on the Events and Presentations section of the Biohaven website.