BIOHAVEN PHARMAC.HLDG CO.

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 NEW HAVEN, Conn. , Nov. 12, 2024 /PRNewswire/ -- Biohaven Ltd.» Mehr auf prnewswire.com

NEW HAVEN, Conn. , Oct. 2, 2024 /PRNewswire/ -- Biohaven Ltd.» Mehr auf prnewswire.com

NEW HAVEN, Conn. , Oct. 1, 2024 /PRNewswire/ -- Biohaven Ltd.» Mehr auf prnewswire.com