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

Core Insights - Biohaven's BHV-1300 has demonstrated significant efficacy in reducing targeted IgG levels by over 60% in the lowest subcutaneous dose tested during the ongoing multiple ascending dose (MAD) study, with sustained pharmacodynamic effects over four weeks [1] - The safety profile of BHV-1300 is strong, with no clinically significant effects on liver function or cholesterol levels, and all adverse events reported were mild and resolved [1][2] - Biohaven is advancing its MoDE platform with the regulatory acceptance of three next-generation degraders, including BHV-1400 and BHV-1600, targeting specific immune-mediated diseases while preserving healthy antibodies [3][4] Group 1: BHV-1300 Development - BHV-1300 achieved rapid and deep reductions in IgG levels, indicating its potential for treating IgG-mediated immune diseases [2] - The optimized subcutaneous formulation of BHV-1300 shows less inter-patient variability compared to previous intravenous formulations [1] - The ongoing MAD study aims to explore the full range of IgG reductions possible with BHV-1300 for various disease indications [1] Group 2: MoDE Technology and Future Developments - Biohaven's MoDE technology allows for selective degradation of pathogenic antibodies while preserving IgG3, crucial for immune response [2] - BHV-1400 targets galactose deficient IgA1 for IgA nephropathy, a rare disease affecting approximately 60,000 individuals in the U.S., with a focus on preserving the immune system [4] - BHV-1600 is designed to degrade autoantibodies related to peripartum cardiomyopathy, a rare heart failure condition with no current approved therapies [5][6] Group 3: Regulatory and Clinical Milestones - Biohaven has submitted a new drug application (NDA) for troriluzole, aimed at treating spinocerebellar ataxia (SCA), a rare neurodegenerative disease affecting around 15,000 people in the U.S. [8] - The pivotal BHV-7000 Phase 2/3 trial for bipolar disorder has completed enrollment ahead of schedule, reflecting the urgent need for new treatments in this area [9] - Biohaven's IND for BHV-1310, an optimized IgG degrader, is expected to begin human dosing in Q1 2025 [7]