Biohaven Ltd (BHVN)

Biohaven Reports First-in-Human Dosing of Oral PKM2 Modulator, BHV-8100, Targeting Metabolic Restoration and ImmunomodulationJune 1, 2026 7:30 AM
PR Newswire (US) BHV-8100 is an orally administered, brain-penetrant PKM2 modulator; a novel therapeutic class addressing the bioenergetic and immunometabolic basis of systemic and central nervous system disorders First-in-human study initiated with dose escalation ongoing; preliminary data in healthy participants demonstrates favorable pharmacokinetics supporting convenient, once-daily dosing and well a tolerated profile at projected therapeutic exposuresPenetrates blood-brain barrier and reverses metabolic dysfunction with 3-fold improvement in glucose utilization in human, whole brain model (Bexorg BrainEx platform, physiologically reactivated brains from human donors); preferential metabolic rescue in Alzheimer's disease donor brains vs controlsExhibits robust beneficial effects across a spectrum of preclinical models of Alzheimer's, and multiple sclerosis: restores metabolic deficits, reduces inflammation and neurodegeneration, and enhances remyelination PKM2 modulation offers a potential new paradigm for treating large, underserved, and high-value indications in neurology, ophthalmology, and immunology NEW HAVEN, Conn., June 1, 2026 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of life-changing therapies for rare and common diseases, today announced the initiation of first-in-human (FIH) dosing for BHV-8100, its oral, brain-penetrant pyruvate kinase M2 isoform (PKM2) modulator. Dr. Bruce Car, Chief Scientific Officer of Biohaven, stated, "BHV-8100 represents a potential fundamental breakthrough in a new class of medicines with potential therapeutic effects in both systemic and brain disorders that involve metabolic and immune dysfunction. PKM2 acts as a bridge between metabolism and immune function. By modulating PKM2, we are striving to correct the core energy deficit known to underpin a number of inflammatory and neurological disorders including atopic dermatitis, Alzheimer's disease, and retinitis pigmentosa. Achieving this important milestone to advance a convenient oral, once-daily and brain-penetrant PKM2 modulator reflects Biohaven's mission to deliver innovative and potentially transformative therapies in areas of profound unmet medical need."BHV-8100 Mechanism of ActionPKM2 is the final, rate-limiting enzyme in glycolysis, converting phosphoenolpyruvate to pyruvate, and it serves as a master metabolic regulator in energy-intensive cells and tissues, including the brain, retina, and immune system. The accumulation of the less enzymatically active, dimeric form of PKM2 leads to bioenergetic deficits and drives disease pathogenesis. Conversion to its more active, tetrameric form by an allosteric PKM2 modulator, such as BHV-8100, can restore energy homeostasis and promote health.The brain and retina are two of the most metabolically demanding tissues in the body with a significant reliance on glucose to produce cellular energy as adenosine triphosphate (ATP). When glucose uptake and ATP generation are deficient, as has been documented in multiple neurodegenerative and retinal diseases, including aging, Parkinson's disease, multiple sclerosis, age-related macular degeneration (AMD), and retinitis pigmentosa, neurodegeneration and immune activation result.  Compelling evidence from preclinical models employing BHV-8100 and the literature suggest a breadth of disease indications for which BHV-8100 could provide significant benefit.   By pharmacologically increasing ATP generation from glycolysis and through fueling the tricarboxylic acid (TCA) cycle, as well as rebalancing glycolytic biosynthetic intermediates, neurons, photoreceptor cells and glial cells become resilient and are better able to sustain cell function, plasticity, and survival.Pierre Magistretti, M.D., Ph.D., Ibn Sina Distinguished Professor at King Abdullah University of Science and Technology, an expert in the effects of neuronal metabolism in disease and discoverer of the astrocyte-neuron lactate shuttle which is critical in neuronal homeostasis, commented, "Neurodegenerative and retinal diseases with early and worsening metabolic hypofunction develop into profoundly disabling conditions. BHV-8100 has the potential to change that. BHV-8100 provides astrocytic lactate to neurons, correcting metabolic dysfunction, improving bioenergetics and offering real hope to the millions of patients to reverse the relentless decline in cognition and eyesight."BHV-8100 is an oral, potent, selective, small-molecule PKM2 allosteric modulator designed for once-daily administration. By binding to and stabilizing the active tetrameric form of PKM2, BHV-8100 restores flux through the glycolytic pathway, producing three interconnected therapeutic effects:Rescues metabolic deficits:  Increased PKM2 activity drives cytoplasmic ATP production, while further fueling mitochondrial TCA cycle ATP production thereby correcting inadequate ATP levels in neurons, glia and retinal cells.  Adequate ATP enables cellular resilience based on restoring energetically expensive processes such as supporting am efficient unfolded protein response (UPR), antioxidant mechanisms, ion channel function, synaptic signaling, and axonal transport which all collectively deteriorate in neurodegeneration.Reduces neuroinflammation: PKM2 modulation to the tetramer form clears glycolytic intermediates, robustly suppressing pro-inflammatory cytokine production (including IL-1ß, TNF-a) and shifting microglia/macrophages toward anti-inflammatory, neuroprotective phenotypes. Th17 lymphocyte specific inhibition is noted in the neuroinflammatory processes associated with allergic encephalomyelitis (EAE).Rescues neurodegeneration: By correcting the energetic and inflammatory drivers of neuronal and glial stress, BHV-8100 protects against synapse loss, axonal degeneration, myelin loss and cell death in preclinical disease models, including EAE and cuprizone-induce myelopathy.BrainEx Human Translational Data: Compelling Preclinical Confirmation of Novel PKM2 Modulating Pharmacology, Pharmacokinetics, Target Engagement, and Human BiologyBiohaven's ongoing collaboration with Bexorg's BrainEx platform allows for early testing of brain penetrance, target engagement, target pharmacology and dose-response relationships in human brain prior to Phase 1 initiation. BrainEx uses ex-vivo, decedent human brains with specific disease diagnoses to generate uniquely powerful translational data prior to Phase 1 first-in-human dosing. Key findings from administration of BHV-8100 in BrainEx demonstrated:Excellent blood-brain barrier penetration confirmed in intact human brains.  Target blood plasma and brain drug concentrations determined in other preclinical models were employed in the BrainEx platform, allowing precise determination of brain uptake of drug.Dose- and brain concentration-pharmacology response relationships confirmed in samples from input and output blood vasculature, brain tissue and cerebrospinal fluid, allowing optimal dose targetingUp to 3 times improvement in cerebral glucose utilization and lactate production, direct readouts of enhanced glycolytic flux, confirming expected PKM2 activation in intact human brain tissue (see Figure 1).   Maintenance of the enhanced glycolytic flux well after removing drug in the blood supply suggests a durable PKM2 tetramer state, further amplifying efficacyPreferential metabolic rescue in Alzheimer's disease: BHV-8100 demonstrated selectively greater glucose utilization improvements in donor brains from individuals diagnosed with Alzheimer's disease versus cognitively normal controls, suggesting that BHV-8100's activity occurs precisely where the metabolic deficit is most severe, a highly favorable mechanistic profile for disease modification.First-in-Human Dosing Initiated in Ongoing Phase 1 StudyBiohaven initiated a Phase 1 FIH study of BHV-8100 in healthy participants to characterize the safety, tolerability and pharmacokinetics of BHV-8100. Preliminary pharmacokinetic (PK) and safety data from the ongoing study:Confirmed PK profile is consistent with oral, once-daily dosingAchieved projected therapeutic exposuresDemonstrated well tolerated profile at projected therapeutic exposures, with most AEs being mild and spontaneously resolvingBharat Awsare, M.D., Executive Medical Director, Clinical Development at Biohaven stated, "The initiation of first-in-human dosing for BHV-8100, a brain-penetrant PKM2 modulator, ushers in a potential new paradigm for a broad range of systemic as well as neurodegenerative, neuroinflammatory, retinal, and age-related diseases. Through our pioneering discovery engine, we are advancing an innovative drug candidate into the clinic that targets the fundamental metabolic impairments underpinning these diseases in ways that simply have not been possible before."Brain Penetration Allows for Extending Beyond Systemic Disorders: Potential in Aging, Neurodegenerative Disorders and Eye Disorders The prevalence and economic burden of neurodegenerative and neuroinflammatory diseases are enormous. Alzheimer's disease alone affects over 6.5 million Americans, with global prevalence exceeding 55 million. Annual costs of treating patients with Alzheimer's disease exceed $300 billion in the United States. Parkinson's disease affects approximately 1 million Americans, and multiple sclerosis affects nearly 1 million U.S. adults. Retinal disease represents a compelling aspect of the BHV-8100 opportunity including Age-Related Macular Degeneration, Diabetic Retinopathy, Inherited Retinal Dystrophies and others. The global retinal disease market is projected to exceed $15 billion annually by 2030, with oral, disease-modifying therapies representing the single most sought-after unmet need in the field. Biohaven believes BHV-8100 could address this gap across multiple retinal indications.About BiohavenBiohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE™ and TRAP™ extracellular protein degradation for immunological diseases; myostatin inhibition for neuromuscular and metabolic diseases, including obesity; and PKM2 activation for neurodegenerative and retinal diseases. For more information, visit www.biohavenpharma.com.Forward-Looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue," "plan," "will," "believe," "may," "expect," "potential," "potentially," and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing, potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments, and events may differ materially from those in the forward-looking statements as a result of various factors, including: the expected timing, commencement, and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations." The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law.MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.
BrainEx is a trademark of Bexorg, Inc.Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli @Dr ExtremeMedia Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer @Punch Drunk View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-reports-first-in-human-dosing-of-oral-pkm2-modulator-bhv-8100-targeting-metabolic-restoration-and-immunomodulation-302786647.htmlSOURCE Biohaven Ltd. Original: Biohaven Reports First-in-Human Dosing of Oral PKM2 Modulator, BHV-8100, Targeting Metabolic Restoration and Immunomodulation

Biohaven Reports Positive Clinical Biomarker and Patient Data: First MoDE and TRAP Extracellular Protein Degraders Achieve Deep, Rapid, Selective Lowering of Disease-Driving Antibodies in Graves' Disease and IgA NephropathyMay 27, 2026 7:30 AM
PR Newswire (US) BHV-1300 demonstrated deep, rapid, and sustained lowering of pathogenic TSHR autoantibodies (TSHR-IgG1) in patients with Graves' hyperthyroidism receiving 1000 mg SC weekly, with mean reductions in TSHR-IgG1exceeding >80% over the 12-week study.Participants with Graves' overt hyperthyroidism, confirmed by elevated baseline thyroid tests despite being treated with anti-thyroid drug therapy (ATD), experienced normalization of thyroid hormones within weeks;  T4 normalization occurring at a median of 3 weeks and T3 at a median of 5 weeks after the first administration of BHV-1300.These preliminary patient data from an ongoing Phase 1b study highlight deep lowering of TSHR-IgG1 with BHV-1300 as a potentially disease-modifying approach to Graves' disease.BHV-1400 achieved rapid, deep, selective, and sustained lowering of Gd-IgA1 in patients with IgA nephropathy (IgAN), differentiating Biohaven's leading TRAP degrader from the complement and BAFF/APRIL inhibitor competition. Participants with IgAN administered BHV-1400 showed a rapid, > 60% mean reduction of Gd-IgA1  within hours, and a 70% mean reduction of within the first one-month of dosing. Observed reductions in Gd-IgA1 were deeper than has been reported with BAFF/APRIL inhibitors, APRIL-inhibitors, and CD38 inhibitors at these early time points. Preliminary observations also demonstrated an increase in eGFR, decrease in spot UPCR, and a decrease in hematuria with one month or less of dosing.BHV-1400 brings precision immunology to the treatment landscape of IgAN as it was rationally designed to selectively remove galactose-deficient IgA1 (Gd-IgA1), the pathogenic antibody driver of the disease while sparing healthy antibodies IgA, IgG, IgE, and IgM. Designed to target the disease driver while preserving healthy immunoglobulins, the complement system, and cell-mediated and humoral immunity, BHV-1400 offers a differentiated approach to current immunosuppressive therapies approved or in development.  Both BHV-1300 and BHV-1400 continue to demonstrate the paradigm shifting potential of its extracellular degrader platform to target pathogenic antibodies causing disease. BHV-1300 and BHV-1400 show a differentiated safety profile with no clinically significant increases in cholesterol, decreases in albumin, or increases in ALT, AST, or bilirubin. Most AEs were mild and self-resolving, there were no drug discontinuations throughout the patient studies, and no serious or severe AEs. Pivotal Phase 3 trials in Graves' Disease and IgA Nephropathy to initiate in the coming weeks by mid-2026. Both trials use a patient friendly, simple to self-administer autoinjector for BHV-1300 and BHV-1400.NEW HAVEN, Conn., May 27, 2026 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven") a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies with the potential to transform autoimmune diseases today reported compelling results regarding its proprietary extracellular MoDE™ (BHV-1300) and TRAP™ (BHV-1400) degrader platform.  The new patient data from its BHV-1300 MoDE degrader for Graves' disease and BHV-1400 TRAP degrader for IgA nephropathy is being presented today at Biohaven's R&D and Analyst Day as part of the 2026 Yale Innovation Summit at the Yale School of Management in New Haven, Connecticut.Tova Gardin, M.D., Chief Translational Officer of Biohaven, commented, "These new data in patients with Graves' disease and IgAN bridge scientific insights and Phase 1 findings – including pharmacodynamics and safety – to advance the programs into the upcoming Phase 3 studies planned for this summer. We continue to make good on our promise to translate the best of immunoscience into meaningful solutions for patients. Through focused innovation and execution, we have advanced the first MoDE and TRAP extracellular protein degraders, demonstrated their ability to rapidly and deeply reduce the antibodies that drive disease, and are encouraged by the biomarker data as well as early improvements in clinical outcomes noted by patients and investigators. As we enter pivotal trials in 2026 and continue to innovate with breakthrough science and precision tools, these data represent just the beginning of the impact we plan to deliver with our Biohaven proprietary extracellular degrader platform."Key Highlights from the Phase 1b Graves' Disease Patient Experience with BHV-1300:Weekly administration of BHV-1300 1000 mg achieved mean reductions of pathogenic TSHR-IgG1 of >80% by week 12 in patients with Graves' hyperthyroidism (see Figure 1), a deeper reduction in pathogenic TSHR-IgG1 than has been demonstrated with the FcRn inhibitor competition.Patients with elevated thyroid hormones experienced a normalization of free T4 at a median of 3 weeks and a normalization of free T3 within a median of 5 weeks (see Figure 2). Patients reported improvements in classic symptoms of hyperthyroidism including palpitations, fatigue, diarrhea, diaphoresis, tremor, and mood.BHV-1300 has been safe and well-tolerated through 12 weeks of dosing. Most AE's have been mild and self-resolving, there have been no drug-discontinuations, and no serious or severe AEs. There have been no clinically significant increases in cholesterol, decreases in albumin, or increases in ALT, AST or bilirubin.  Critically, BHV-1300 preserved IgG3 and demonstrated no clinically significant reductions in IgG3, IgA, IgE, or IgM relative to baseline, maintaining the immune protection that FcRn inhibitors — which lower all IgG subclasses including IgG3 — have not demonstrated.Malini Gupta, M.D.,  ECNU, FACE, Director of G2Endo- Endocrinology in Memphis, the 2025 AACE Thyroid Chair and investigator in the BHV-1300 study, commented, "We are excited by these initial data in patients with Graves' disease, which demonstrate BHV-1300's ability to profoundly reduce TSHR autoantibodies and to normalize thyroid function in patients who have limited options today." Dr. Gupta added, "The strong link between TSHR-IgG1 lowering and clinical response positions BHV-1300 as a potential first-and-best-in-class therapy for Graves' disease. We see substantial unmet need in this population with no current disease modifying therapy and are excited for our patients to participate in the BHV-1300 pivotal Phase 3 study this year."The positive Phase1b results in patients with Graves' disease who were hyperthyroid despite concurrent treatment with ATDs build on the observed dose-response relationship of BHV-1300 and literature that suggests deeper TSHR-IgG1 reductions are known to drive meaningfully higher efficacy compared to more modest lowering. Additional biomarker and pharmacokinetic data (n=8) regarding Graves' patients who were and were not hyperthyroid at baseline will be presented at the Biohaven R&D Day and will be available on the company's website. Based upon the pharmacokinetic and biomarker results with BHV-1300 in this Phase 1b study, Biohaven is advancing to a pivotal trial in Graves' disease in the coming weeks. The planned study design is a double-blind placebo-controlled study in adults with Graves' hyperthyroidism evaluating normalization of T3, T4, and TSH at 26 weeks absent an antithyroid drug.  Figure 1: BHV-1300 rapidly and robustly reduced TSHR autoantibodies and normalized free T4 and free T3 in consecutively treated hyperthyroid patients (n=3) who were already on ATDsFigure 2: Time to normalization of thyroid hormones after treatment with BHV-1300 in consecutively treated patients (n=3) with hyperthyroidism at baseline despite concurrent use of antithyroid drugs.Key Highlights from the Phase 1b IgA Nephropathy Disease Patient Experience with BHV-1400:Administration of BHV-1400, the Company's first TRAP, galactose-deficient IgA1 (Gd-IgA1) TRAP degrader for the treatment of IgA nephropathy (IgAN)  achieved deep, rapid, and sustained mean reductions of pathogenic Gd-IgA1 of >60% within 48 hours of subcutaneous administration and 70% within the one month of dosing in patients with IgAN (see Figure 3). These reductions are deeper than those demonstrated by BAFF-APRIL inhibitors, APRIL inhibitors, and CD38 inhibitors at this early time point.Reductions in Gd-IgA1 were associated with resolution of hematuria, decreases in spot UPCR, and increases in eGFR (see Figures 4-6). Participants also subjectively reported improvements in fatigue.BHV-1400 has been safe and well-tolerated throughout one month of dosing. Most AE's have been mild and self-resolving, there have been no drug-discontinuations, and no serious or severe AEs. There have been no clinically significant increases in ALT, AST or bilirubin, and no clinically significant reductions in other immunoglobulins relative to baseline.Effects were selective, with no clinically significant reductions observed in other immunoglobulins: IgA, IgG, IgE, or IgM.Dr. Jonathan Barratt, the Mayer Professor of Renal Medicine at University of Leicester and leading expert in the treatment of IgAN, commented on the new Phase 1b patient data, "These new data, which include data from my own patients, represent the next frontier of innovation for patients with IgA nephropathy. Currently approved treatments broadly suppress the immune system, and disease recurs upon their cessation, necessitating long term, potentially life long, treatment. There is a need for rapid acting and safe and rapid therapies that can be utilized throughout a patient's lifetime. The data I presented today show consistency between Phase 1 healthy subjects and patients with IgAN in BHV-1400's ability to deeply remove pathogenic Gd-IgA1. This reduction in Gd-IgA1 is associated with an immediate impact on patient outcomes rapidly without immunosuppression. I remain excited about BHV-1400, based on the Phase 1 patient data, as it rapidly and specifically targeted the fundamental abnormality in IgA nephropathy while sparing the rest of the immune system. It has the potential to take away the major driver for immune complex formation while leaving other antibodies unaffected, which means it potentially has efficacy with unrivaled safety."Figure 3: BHV-1400 rapidly and robustly reduced Gd-IgA1 in patient with IgAN.Figure 4: Associated changes to eGFR in IgAN patients administered one month of treatment with BHV-1400.Figure 5: Associated changes to UPCR in IgAN patients administered one month of treatment with BHV-1400.Based upon the rapid and deep reductions of Gd-IgA1 observed with subcutaneous BHV-1400, Biohaven plans to study BHV-1400 in patients with IgAN, initiating a pivotal trial in mid-2026 designed to support regulatory approval, evaluating urine protein-creatinine ratio (UPCR) and eGFR at 1 year.About Graves' DiseaseGraves' disease is the most common cause of hyperthyroidism, driven by autoantibodies stimulating the TSH receptor. A relapsing and remitting condition, Graves' disease affects 1% of the global population.Driven by an autoantibody (TSHR-IgG1) that binds the TSH receptor, Graves' disease causes multi-organ symptoms and is complicated by associated TSHR-IgG1 driven conditions: thyroid eye disease, neonatal Graves' disease, and pretibial myxedema. Current treatment options are limited to thyroid organ removal or ablation or anti-thyroid drugs, none of which target the underlying autoantibody driving the disease. Standard ATDs do not target the underlying driver of disease and leave patients still symptomatic and at risk for development of thyroid eye disease and pretibial myxedema. Among patients with Graves' disease recently exposed to antithyroid drugs, 93% report multiple ongoing symptoms, with 72% reporting five or more persistent symptoms. With no new FDA approved therapy in over seventy years, there is a high unmet need for a novel therapy targeting the root cause of disease.Biohaven's Lead MoDE degrader, BHV-1300, is poised to advance into its pivotal trial in Graves' disease mid-2026.  The presentation slides from Biohaven's R&D Day for the TRAP and MoDE degraders and its other platforms will be available on the Events and Presentations page of the Biohaven website just prior to their presentations.About BHV-1300BHV-1300, first MoDE, is a small molecule, extracellular IgG degrader, rationally designed to leverage the body's natural hepatic clearance mechanisms to selectively target and remove IgG1, IgG2, and IgG4, the underlying cause of many immune-mediated diseases.  BHV-1300 is mechanistically differentiated from FcRn inhibitors in multiple important ways: first, it selectively targets the disease-causing IgG subclasses (IgG1, IgG2, IgG4) while sparing IgG3, which mediates protection against bacteria, viruses, and parasites and which has not been demonstrated with FcRn inhibitors; second, as a small molecule, BHV-1300 does not interact with FcRn and therefore does not accelerate the clearance of co-administered Fc-containing biologic therapies — a meaningful clinical limitation of the FcRn inhibitor class; third, it avoids the off-target effects of cholesterol elevation, albumin reduction, and headache associated with first-generation FcRn inhibitors; and fourth, it is delivered in a convenient self-administered autoinjector.About IgA NephropathyIgA nephropathy is the leading cause of glomerular disease globally and is commonly diagnosed in individuals in their second and third decades of life, with most individuals progressing to renal failure over the ensuing 10-15 years. As a disease of the immune system, IgA nephropathy frequently returns even after renal transplant. While the 2021 Kidney Disease Improving Global Outcomes (KDIGO) treatment guidelines recommended only standard chronic kidney disease treatments, the 2025 guidelines emphasize the importance of treating the underlying immune disease by removing aberrant forms of IgA. "Galactose deficient IgA1 is the fundamental abnormality in IgA nephropathy," Dr. Barratt explained, "It's a group of IgA molecules that have changes to the sugars on the IgA1 hinge region that fundamentally change the way this antibody behaves. It promotes immune complex formation and it's these immune complexes that cause glomerular injury and damage and promote loss of kidney function."About BHV-1400BHV-1400, Biohaven's lead TRAP extracellular degrader, rapidly and selectively removes Gd-IgA1, the fundamental abnormality in IgA nephropathy, without adversely affecting any other immunoglobulins or components of protective immunity. BHV-1400's precision approach is differentiated from all existing and investigational therapies for IgAN: complement inhibitors, BAFF/APRIL inhibitors and non-selective inhibitors of IgA all suppress immunity.  BHV-1400 has been safe and well-tolerated across the ongoing Phase 1b study in IgAN patients. Most adverse events (AEs) were mild and self-resolving, there were no discontinuations due to AEs related to study drug, and there were no serious or severe AEs related to drug. There were no clinically significant increases in ALT, AST or bilirubin, no clinically significant reductions in albumin and no clinically significant increases in cholesterol relative to placebo over the one month dosing period. There were no clinically significant reductions in other immunoglobulins including IgG, IgA, IgE, or IgM relative to baseline.About BiohavenBiohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The Company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE and TRAP extracellular protein degradation for immunological diseases; and myostatin inhibition for neuromuscular and metabolic diseases, including obesity. For more information, visit www.biohavenpharma.com.Forward-Looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "potentially", "groundbreaking" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing to and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, including the studies of BHV-1300 and BHV-1400; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable US regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli @Dr ExtremeMedia Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer @Punch Drunk  View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-reports-positive-clinical-biomarker-and-patient-data-first-mode-and-trap-extracellular-protein-degraders-achieve-deep-rapid-selective-lowering-of-disease-driving-antibodies-in-graves-disease-and-iga-nephropathy-302783023.htmlSOURCE Biohaven Ltd. Original: Biohaven Reports Positive Clinical Biomarker and Patient Data: First MoDE and TRAP Extracellular Protein Degraders Achieve Deep, Rapid, Selective Lowering of Disease-Driving Antibodies in Graves' Disease and IgA Nephropathy

Biohaven Reports New Clinical Data in Epilepsy with Opakalim, a Selective Kv7.2/7.3 Activator, Highlighting Seizure Control and Markedly Differentiated Tolerability ProfileMay 26, 2026 7:30 AM
PR Newswire (US) In a randomized, placebo-controlled, time-to-event trial in idiopathic generalized epilepsy (IGE), the median time to the second day with a generalized tonic-clonic seizure was 141 days in the opakalim 75 mg once-daily treatment group vs. 47 days in the placebo group, representing a 3-fold prolongation.In an ongoing open-label extension (OLE) study in focal epilepsy, an updated data analysis shows 54% of patients with focal epilepsy on opakalim 75 mg once-daily achieved a ≥50% reduction in seizure frequency over any consecutive six months of open-label treatment compared to pre-randomization baseline.A six-month update of opakalim compassionate use treatment in a Kv7-activation dependent patient with KCNQ2-Developmental and Epileptic Encephalopathy (KCNQ2-DEE) confirms clinical stability and ongoing seizure control. Overnight EEG at 6-months demonstrated a 50% reduction in seizure counts relative to pre-opakalim baseline.Opakalim has been well-tolerated across all studies (1000+ subjects to date) with a low incidence of adverse events (AEs) comparing favorably with approved and investigational antiseizure medicines.In the IGE study, the opakalim group reported no cases of somnolence, dizziness, fatigue, or memory impairment; and in the focal epilepsy OLE study, rates of these central nervous system (CNS) AEs were less than or equal to 5% each.Opakalim's tolerability profile is markedly differentiated from that of other investigational Kv7 activators reporting double-digit rates of CNS AEs in OLE studies.On track to receive top-line results in 2H 2026 from the first of two pivotal Phase 2/3 randomized, double-blind, placebo-controlled studies in refractory focal epilepsy to support registration.NEW HAVEN, Conn., May 26, 2026 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN), 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 reported new data from its selective Kv7.2/7.3 channel activator program, currently in Phase 2/3 studies for the treatment of focal epilepsy, and will present additional updates at its annual R&D Day held in conjunction with the Yale Innovation Summit at the Yale School of Management in New Haven, Connecticut on May 27, 2026. Opakalim is distinguished from other investigational Kv7 activators by its selectivity for Kv7.2/7.3 and lack of GABA receptor activity. Opakalim offers potential for easy-to-use, once-daily, orally-administered treatment without the need for titration to control seizures, and without the burdensome central nervous system (CNS) side effects frequently reported with approved and investigational antiseizure medicines (ASMs). The R&D Day presentation will showcase new and updated clinical data from: 1) a randomized, double-blind, placebo-controlled proof-of-concept study in idiopathic generalized epilepsy (IGE); 2) an ongoing focal epilepsy open-label extension (OLE) study; and 3) a six-month clinical update for a pediatric patient with KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE). Together, these emerging data reinforce opakalim's target engagement, differentiated profile and potential to address the unmet need for novel, effective, and well-tolerated ASMs.Jason Lerner, M.D., Medical Director, Research & Development and Epilepsy Development Lead at Biohaven, commented, "The opakalim data across IGE, focal epilepsy and KCNQ2-DEE are consistent and compelling. What stands out is not just that opakalim controls seizures — it's that it does so without the burdensome side effects that compromise quality of life and adherence for people with epilepsy. Dizziness, somnolence, fatigue, and memory impairment are reported in meaningful proportions of patients on existing and investigational ASMs. Opakalim's zero rates of somnolence, dizziness, fatigue, and memory impairment in our small IGE proof-of-concept study, and single-digit rates of CNS adverse events in our focal epilepsy program suggest that selective Kv7.2/7.3 activation is a fundamentally different and cleaner mechanism. I believe opakalim can offer people with epilepsy real seizure control without asking them to compromise their quality of life."Efficacy Signal in Highly Treatment-resistant IGE PopulationBiohaven to present results from a randomized, double-blind, placebo-controlled, time-to-event proof-of-concept study evaluating opakalim 75 mg once-daily in subjects with IGE with intractable GTC seizures (NCT06425159). The study's prespecified primary outcome measure was defined as the time to the second day with a GTC seizure during the 24-week double-blind treatment period. The study enrolled a total of 27 subjects (15 in the opakalim arm and 12 in the placebo arm). Formal statistical testing is not provided, as this proof-of-concept study was closed prior to reaching the study's prespecified sample size, due to enrollment challenges and strategic portfolio prioritization. The observed efficacy and tolerability signals are consistent with opakalim's mechanism and broader clinical dataset.Key Findings:Median time to second GTC seizure during the 24-week double-blind treatment period: 141 days on opakalim vs. 47 days on placebo, a 3-fold prolongation of time to second seizure event (Figure 1).33% of opakalim-treated subjects completed the 24-week double-blind phase without a second GTC seizure vs. 0% of placebo subjects.20% of opakalim-treated subjects completed the 24-week double-blind phase seizure-free vs. 0% of placebo subjects.Opakalim was well-tolerated with remarkably low rates of nervous system adverse events (AEs) including somnolence, dizziness, and fatigue — AEs that collectively occur in more than one-third of patients on competitor investigational Kv7 activator in long-term studies, and at even higher rates with approved ASMs.Efficacy Signal and Differentiated Safety Profile in Focal Epilepsy OLEBiohaven to also report updates from its focal epilepsy program, including a recent data analysis from the ongoing open-label extension study of opakalim in subjects with refractory focal epilepsy (NCT06443463). As of 1H 2026, the double-blind study completion rates were 95%; and rollover rates in the optional open-label extension were 95%, reflecting subject and investigator confidence in opakalim.Key Findings:In the opakalim 75 mg once-daily, six-month completers cohort (n>100), 54% of subjects achieved a ≥50% reduction in seizure frequency over any consecutive six months of OLE treatment compared to pretreatment baseline prior to randomization (Figure 2). This result is on par with the ≥50% responder rate reported for the investigational Kv7 activator azetukalner (56%), but opakalim achieves this comparable level of seizure control with a substantially lower burden of CNS AEs, including markedly lower rates of dizziness, somnolence, fatigue, and memory impairment.The AE profile was favorable and consistent with prior data, with a markedly lower incidence of AEs compared to approved and investigational ASMs (Figure 3). For example, in opakalim-treated subjects (75 mg), dizziness was reported in only 5.0% of subjects. By contrast, published long-term data from a competitor's Kv7 activator shows its open-label extension study reported dizziness in 25%, somnolence in 17%, memory impairment in 11%, and falls in 15% of subjects. These data suggest opakalim's selective Kv7.2/7.3 activation without GABA effects may translate into a meaningfully cleaner CNS tolerability profile than other ASMs.Clinical Stability and Ongoing Seizure Control in Kv7-Dependent KCNQ2-DEE PatientBiohaven to further provide a six-month update for the 9 year-old boy with refractory epilepsy due to KCNQ2-DEE being treated with compassionate use opakalim. The patient had a history of daily tonic seizures despite three concurrent ASMs, including a first-generation Kv7 activator, with prior attempts to taper first-generation Kv7 resulting in status epilepticus, ICU admission, and developmental regression. Following compassionate use authorization under a single-patient IND approved by the FDA, he was transitioned to opakalim at exposures calibrated to match the 75 mg dose being studied in the pivotal focal epilepsy trials.Key Finding:Now at the six-month mark, the patient remains clinically stable. Overnight EEG at six-months demonstrated a 50% reduction in seizure counts relative to pre-opakalim baseline. Opakalim has been well-tolerated over the six-month treatment period.Biohaven is on track to announce in 2H 2026 top-line results from the first of two pivotal Phase 2/3 randomized, double-blind, placebo-controlled studies in refractory focal epilepsy to support registration.About Opakalim Opakalim (BHV-7000) is Biohaven's next-generation, selective Kv7.2/7.3 potassium channel activator that targets a clinically validated mechanism of action for the treatment of epilepsy. Opakalim is differentiated from both first- and second-generation Kv7 activators by its selectivity: it preferentially activates the Kv7.2/7.3 heteromeric channels that are the predominant regulators of neuronal excitability, with substantially less activity at GABA receptors. This selectivity profile is hypothesized to underlie opakalim's favorable tolerability, including the low rates of somnolence, dizziness, and fatigue observed in clinical studies to date. Opakalim has been studied in more than 1,000 subjects across multiple clinical trials, consistently demonstrating a favorable tolerability profile. Biohaven is currently conducting two Phase 2/3 randomized, double-blind, placebo-controlled studies (NCT06132893 and NCT06309966) comparing the efficacy of opakalim to placebo as an adjunctive therapy for refractory focal onset epilepsy, as well as an open-label extension study (NCT06443463) to evaluate the long-term efficacy and safety of opakalim.About BiohavenBiohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE™ and TRAP™ extracellular protein degradation for immunological diseases; and myostatin inhibition for neuromuscular and metabolic diseases, including obesity. For more information, visit www.biohavenpharma.com.Forward-Looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "potentially", "groundbreaking" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, including the studies of opakalim; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable US regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli @Dr ExtremeMedia Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer @Punch Drunk  View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-reports-new-clinical-data-in-epilepsy-with-opakalim-a-selective-kv7-27-3-activator-highlighting-seizure-control-and-markedly-differentiated-tolerability-profile-302781743.htmlSOURCE Biohaven Ltd. Original: Biohaven Reports New Clinical Data in Epilepsy with Opakalim, a Selective Kv7.2/7.3 Activator, Highlighting Seizure Control and Markedly Differentiated Tolerability Profile

Biohaven Shares Edge Higher After Completing Enrollment in Obesity Drug TrialMarch 19, 2026 10:12 AM
IH Market News
Biohaven Ltd. (NYSE:BHVN) shares gained about 2% on Thursday after the company announced that enrollment has been completed in its Phase 2 clinical trial evaluating taldefgrobep alfa as a potential treatment for obesity. Topline results from the study are expected in the second half of 2026.The randomized, double-blind, placebo-controlled trial is testing both once-weekly and once-monthly dosing of taldefgrobep as a standalone therapy in adults who are overweight or obese. The study aims to enroll approximately 150 participants and includes a 24-week double-blind treatment phase followed by a 24-week open-label extension period.Taldefgrobep is designed to inhibit the myostatin-activin signaling pathway, a mechanism that targets both fat and muscle tissue. The primary endpoint of the trial measures the percentage change in total body weight from baseline to Week 24. Secondary endpoints include changes in total body fat mass and lean body mass.Earlier clinical research showed that taldefgrobep reduced fat mass while increasing lean muscle mass in healthy volunteers and other non-obese groups. In a Phase 1 trial, participants receiving the drug experienced reductions in total body fat mass of more than 6%, alongside increases in lean muscle mass of up to 4% after 29 days of treatment.According to Biohaven, taldefgrobep has been tested in more than 700 participants across clinical trials and has demonstrated a favorable safety profile, with low rates of serious adverse events or treatment discontinuations.Preclinical studies using obese mouse models also indicated that the drug, both as a monotherapy and in combination with a GLP-1 agonist, reduced fat mass and body weight while increasing lean muscle mass.The current Phase 2 study is being conducted at 20 clinical research sites across the United States.Biohaven stock price

Original: Biohaven Shares Edge Higher After Completing Enrollment in Obesity Drug Trial

Biohaven's Phase 2 Obesity Study with Taldefgrobep Alfa, a Novel Myostatin-Activin Pathway Inhibitor, Completes EnrollmentMarch 19, 2026 7:30 AM
PR Newswire (US)

Phase 2 study in obesity, evaluating treatment with once-weekly and once-monthly taldefgrobep alfa as monotherapy, is now fully enrolled; topline data expected in 2H 2026.Taldefgrobep is a novel inhibitor of the myostatin-activin signaling pathway, which directly targets both fat and muscle, offering the potential to achieve high-quality weight loss in people living with overweight and obesity.In previous clinical studies, taldefgrobep produced meaningful reductions in fat mass while increasing lean muscle mass in healthy adults and other non-obese populations; it has also demonstrated a highly favorable safety and tolerability profile in more than 700 trial participants.In nonclinical studies in obese mouse models, taldefgrobep, both as monotherapy and in combination with a GLP-1 agonist, generated significant reductions in fat mass and body weight, while increasing lean mass.NEW HAVEN, Conn., March 19, 2026 /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 announced the completion of enrollment in a Phase 2 proof-of-concept (PoC) study with its myostatin-activin pathway inhibitor (MAPI), taldefgrobep alfa, which offers the potential to achieve high-quality weight loss in people living with obesity. Topline data from the study are expected in 2H 2026.







Frank Greenway, M.D., Professor, Chief Medical Officer of the Clinical Trials Unit at Pennington Biomedical Research Center, Baton Rouge, LA stated, "We are at a watershed moment in the treatment of obesity, brought on by the introduction of highly effective therapies like the GLP-1 agonists. To realize optimal long-term health outcomes, however, we will need to look beyond maximizing weight reduction and remain vigilant around the importance of muscle mass in overall health and wellness. Investigational medications, like taldefgrobep, with the potential to meaningfully reduce body weight and adipose tissue, while increasing muscle mass can be an important addition to the anti-obesity armamentarium."The Phase 2 PoC study (NCT07281495) is a randomized, double-blind, placebo-controlled, dose-ranging study evaluating the efficacy and tolerability of once-weekly and once-monthly taldefgrobep as monotherapy, via self-administered autoinjector, in adults living with overweight and obesity. It includes a 24-week double-blind treatment period followed by 24-week open-label extension period. The study is targeting approximately 150 participants for randomization. The primary outcome measure is percent change in total body weight from baseline to Week 24, and secondary outcome measures are percent change in total body fat mass and in total body lean mass. The study is being conducted at 20 clinical sites across the US.Peter Ackerman, M.D., Senior Vice President of Clinical Development at Biohaven stated, "We are excited to evaluate taldefgrobep, as both a once-weekly and once-monthly dosing regimen, in an obese patient population. We believe taldefgrobep could represent an important new agent, as monotherapy and in combination with the current standard of care, that can help optimize high-quality weight loss in people living with obesity." Dr. Ackerman added, "New investigational therapies with novel modes of action are critical to maximizing long-term health benefits in people living with overweight and obesity. While there have been great recent advancements in the field of obesity medicine, there is still a lot of work to do in optimizing the management of a complex, heterogeneous condition that affects nearly half of the world's population. Our team is grateful to the investigators, their staff, and all participants involved in this important study."In previous clinical studies, taldefgrobep demonstrated beneficial changes in fat mass and lean mass in non-obese populations. Participants in a Phase 1 trial who received taldefgrobep realized significant reductions in total body fat mass (>6%) with commensurate increases in lean muscle mass (up to 4%) after 29 days of dosing. In that study, taldefgrobep benefit in body composition change was not fully realized until four weeks beyond the final dose was administered, suggesting the drug may support extended dosing intervals. In the Phase 3 study BHV2000-301, examining patients with a neuromuscular disorder, taldefgrobep-treated participants achieved statistically significant reductions in total body fat accumulation (9.7%; p700 clinical trial participants and has been well tolerated with low rates of serious adverse events (SAEs) and adverse events (AEs) leading to early discontinuation. Rates of muscle- and GI-related AEs have been generally comparable to placebo.In nonclinical studies, taldefgrobep has demonstrated the ability to have direct beneficial effects on lean muscle mass and adipose tissue by interrupting the signaling through activin receptors (ActRII) caused by transforming growth factor-beta (TGF-ß) ligands, such as myostatin and multiple activins (A, E, etc.). In a diet-induced obese (DIO) mouse model, taldefgrobep monotherapy showed significant improvements in total body weight, fat mass, and lean mass relative to vehicle. In DIO mice, taldefgrobep in combination with a GLP-1 agonist showed additive benefit across these endpoints.Data from multiple Phase 2 studies conducted with bimagrumab, a myostatin-activin pathway inhibitor, demonstrates a strong proof of concept for this therapeutic approach to obesity. In the BELIEVE study, participants dosed with bimagrumab as monotherapy, achieved reductions in total body fat mass comparable to high-dose semaglutide (2.4 mg) at one year (-25.3% vs. -24.8%, respectively). In the same study, bimagrumab-treated individuals saw a greater reduction in metabolically active visceral adipose tissue (-40.2% vs. -29.5%) and improvement in lean muscle mass compared semaglutide (+2.7% vs. -7.9%, respectively). Unfortunately, the safety profile of bimagrumab (high rates of GI- and muscle-related AEs and worsening of cholesterol levels) has complicated further development of this asset for an indication in chronic weight management. and worsening of cholesterol leves) has complicated further development of this asset for an indication in chronic weight management.About Taldefgrobep AlfaTaldefgrobep alfa is a novel fusion protein designed to inhibit signaling through activin transmembrane receptors (ActRII). Taldefgrobep targets free myostatin, forming stable complexes which bind to ActRII in a manner that prevents downstream signaling from transforming growth factor-beta (TGF-ß) ligands (including myostatin and activins) important in body composition change. Taldefgrobep's novel mechanism of action offers the potential for meaningful reductions in fat mass, increased lean mass, and improvements in multiple metabolic parameters.About BiohavenBiohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, obesity, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE™ and TRAP™ extracellular protein degradation for immunological diseases; TYK2/JAK1 inhibition for neuroinflammatory disorders; myostatin-activin pathway inhibition for neuromuscular and metabolic diseases; antibody recruiting bispecific molecules; and antibody drug conjugates for cancer. For more information, visit www.biohaven.com.Forward-looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected timing and amounts of funding under the NPA. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate", "potential first-in-class" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials including the Phase 2 taldefgrobep trial in people living with obesity (BHV2000-202); the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates and the expected timing thereof; the potential for Biohaven's product candidates to be successful therapies, including the potential for taldefgrobep as a treatment for overweight and obesity; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.Investor Contact:Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli @Dr ExtremeMedia Contact:Mike Beyer
Sam Brown Inc.
mikebeyer @Punch Drunk














View original content to download multimedia:https://www.prnewswire.com/news-releases/biohavens-phase-2-obesity-study-with-taldefgrobep-alfa-a-novel-myostatin-activin-pathway-inhibitor-completes-enrollment-302718076.htmlSOURCE Biohaven Ltd.

Original: Biohaven's Phase 2 Obesity Study with Taldefgrobep Alfa, a Novel Myostatin-Activin Pathway Inhibitor, Completes Enrollment

uniQure shares jump 36% after news of FDA gene therapy chief’s departureMarch 9, 2026 10:14 AM
IH Market News
Shares of uniQure BV (NASDAQ:QURE) surged 36% on Monday after reports emerged that the head of the U.S. Food and Drug Administration’s vaccines and gene therapies division will step down, triggering several analyst upgrades across the biotechnology sector. Other companies in the space also rallied, with REGENXBIO (NASDAQ:RGNX) rising 13% and Biohaven (NYSE:BHVN) gaining 23%.Vinay Prasad, who currently leads the FDA’s Center for Biologics Evaluation and Research division overseeing vaccines and gene therapies, is expected to leave the agency next month to return to academia. His tenure has been viewed by some industry observers as a period of heightened regulatory scrutiny for gene therapy developers.RBC Capital analyst Luca Issi upgraded uniQure from Sector Perform to Outperform and raised the firm’s price target to $35.00 from $11.00. The company had recently faced regulatory setbacks after the FDA indicated it would not approve its Huntington’s disease therapy based solely on comparisons to natural history data, a development uniQure described as a “key shift” from earlier feedback given before changes in FDA leadership.“We believe that Prasad’s departure is likely to open up a more balanced discussion on risk/reward for HD,” Issi wrote, adding that he now estimates a 50% probability that the therapy could eventually gain approval.Stifel analyst Paul Matteis also reacted positively to the news, calling Prasad’s exit “a big win for biotech, especially for companies in the rare disease space.” Matteis added that several gene therapy and rare disease developers had been under pressure as part of what he described as the “FDA risk off” basket, with Prasad reportedly overriding review teams to recommend negative outcomes in certain cases.An analyst at Truist noted that Prasad’s leadership “marked a sharp departure from the more flexible regulatory approach for rare and serious diseases” seen under his predecessor Peter Marks. According to the analyst, several companies encountered evolving regulatory expectations during development discussions while Prasad was in charge.uniQure is scheduled to hold a Type B meeting with the FDA in the second quarter.uniQure stock price

Original: uniQure shares jump 36% after news of FDA gene therapy chief’s departure

Biohaven Reports Recent Business Developments and Fourth Quarter and Full Year 2025 Financial ResultsMarch 2, 2026 4:05 PM
PR Newswire (US)

Prioritizing three key, late-stage clinical programs including Molecular Degrader of Extracellular Proteins (MoDETM) and Targeted Removal of Aberrant Protein (TRAPTM) extracellular protein degradation for immunological diseases, Kv7 ion channel modulation for epilepsy; and myostatin-activin pathway targeting obesity:Inflammation and Immunology:Graves' Disease: First-in-patient clinical experience with IgG MoDE degrader BHV-1300 resulted in complete suppression of disease-causing TSH receptor-stimulating antibodies, normalization of previously elevated thyroid hormones within weeks after dosing a patient with Graves' disease. BHV-1300 has shown the potential for best-in-class reductions of IgG, with maximum reductions of up to an 87% decrease from baseline within weeks of dosing in a study conducted in healthy volunteers. The Company intends to initiate a pivotal study with BHV-1300 in Graves' disease in the second half of 2026.IgA Nephropathy (IgAN): First dosing of BHV-1400 TRAP degrader in IgAN patients achieved early observations of both biomarker and clinical responses. BHV-1400 previously achieved rapid lowering of Gd-IgA1 with a mean reduction of >60% within hours and maximum reduction exceeding 80% in a study conducted in healthy volunteers. A pivotal study is set to initiate in the first quarter of 2026.Ion Channel:Epilepsy: In an ongoing open-label extension study of Biohaven's selective Kv7 channel activator, the majority of participants who completed at least 6 months of open-label extension (OLE) treatment with opakalim 75 mg once daily showed ≥50% reductions in seizure frequency compared to pretreatment baseline. Opakalim was well-tolerated in the OLE with a low incidence of central nervous system adverse events, representing a potential paradigm-shift for patients with a highly favorable and differentiated safety profile compared to other approved or investigational antiseizure medicines. Pivotal results for opakalim in the treatment of focal epilepsy are expected in the second half of 2026.Myostatin-Activin:Obesity: In 4Q 2025, Biohaven initiated a Phase 2 study in people living with overweight and obesity, with topline results expected in the second half of 2026. The study is evaluating the ability of taldefgrobep, administered once-weekly or once-monthly via autoinjector, to achieve high quality weight loss including reducing fat mass and total body weight while also increasing lean muscle mass.Disciplined approach to balance sheet management and capital preservation in place, as we continue positioning the portfolio for future value creation, including ongoing evaluation of potential strategic opportunities with non-priority programs:Broader Degrader Portfolio: With clinical proof-of-concept now established, Biohaven is positioned to advance multiple next-generation extracellular protein degraders targeting high value immune-mediated disease indications and explore strategic partnerships to advance the breadth of this platform technology. Potential applications include BHV-1420 in membranous nephropathy, BHV-1450 in pemphigus vulgaris, myasthenia gravis, and encephalitis, BHV-1440 in Graves' disease and thyroid eye disease, BHV-6500 in type 1 diabetes, BHV-1490 in cryoglobulinemia, Waldenstrom's macroglobulinemia and IgM neuropathy, BHV-1310 for management of rare IgG-mediated indications, and various additional undisclosed degraders.Highly differentiated, next-generation antibody drug conjugate (ADC) portfolio: Phase 1/2 study evaluating BHV-1510 (Trop2 ADC) as monotherapy and in combination with Regeneron's anti-PD-1 monoclonal antibody cemiplimab shows continued clinical activity with confirmed responses in non-small cell lung cancer, endometrial cancer, and urothelial cancer with a differentiated safety profile in the ongoing study. BHV-1510 is the first and only Trop2 ADC in clinic with potential subcutaneous route of administration. Additionally, first-in-class FGFR3 directed ADC, BHV-1530, continues with dose escalation and no dose limiting toxicities to date.TYK2/JAK1 inhibition: Pivotal clinical trial evaluating Biohaven's brain-penetrant TYK2/JAK1 inhibitor for the treatment of early Parkinson's disease continues to advance enrollment.Cash, cash equivalents, marketable securities and restricted cash as of December 31, 2025, totaled approximately $322.0 million.  Subsequent to December 31, 2025, the Company issued and sold an additional 17.2 million common shares for net proceeds of $178.9 million, including a directed common share sale to Janus Henderson Investors as a block transaction under the Company's "at-the-market" offering program.NEW HAVEN, Conn., March 2, 2026 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), 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 reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a review of recent accomplishments and anticipated upcoming developments.







Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "We made significant progress over the past year in advancing our pipeline of innovative therapies, particularly with our revolutionary degrader platform, the central pillar of our long-term strategic focus in immunology and inflammation. Biohaven is a leader in the field of targeted extracellular protein degradation with our MoDE and TRAP degraders, with a technology borne out of ground-breaking science exclusively licensed from Yale and developed internally by our impressive discovery engine. MoDE and TRAP degraders have demonstrated the ability to rapidly, profoundly, and selectively target the root causes of disease without compromising patients' healthy immune functions. To date, we have established strong IP across multiple disease targets, advanced next-generation drug candidates, developed easy-to-use autoinjectors, and, most critically, provided proof of concept with a robust body of evidence in non-clinical studies as well as in healthy volunteers and patients. Our degrader portfolio possesses the potential to address an array of well-validated targets with highly differentiated mechanisms of action, reinforcing our confidence in this platform as a meaningful driver of future value creation, and we have the unique opportunity to be the first to market with this innovative technology."  "In parallel, we advanced other key programs that underscore the breadth and balance of our pipeline. Our selective Kv7 program is an extremely promising approach for treating focal epilepsy, which has the potential to address the significant unmet medical need for a novel efficacious antiseizure medicine that is easy-to-use, does not burden patients with central nervous system (CNS) side effects like dizziness and somnolence, and does not make their comorbidities worse.  In fact, we were very excited to report preliminary data demonstrating strong signals of efficacy from our ongoing open label extension (OLE) trial of opakalim, including improvements in seizure frequency greater than or equal to 50% in the majority of participants treated with opakalim 75 mg once daily for at least six months in the OLE, coupled with an exceptional tolerability profile. We are on track to deliver pivotal topline data from this program this year. Shifting to our third and final key strategic focus area, with our myostatin-activin pathway inhibitor, taldefgrobep alfa, we are now exploring the opportunity to address critical gaps in the treatment of obesity. Specifically, taldefgrobep is designed to directly target fat, build muscle and increase bone density while avoiding the intolerable adverse effects that occur with other myostatin-activin inhibitors. We look forward to reporting topline data with this exciting program this year. Together, these assets reflect our focus on pursuing scientifically rigorous programs with the potential to deliver impactful therapies to patients, and critical milestones are on the horizon this year for all three of these core programs."Matt Buten, Biohaven's Chief Financial Officer, added, "Though progress across our portfolio was demonstrable, we redoubled efforts to execute with a renewed emphasis on financial discipline. During the final quarter of 2025, we initiated strategic portfolio and cost-optimization measures, carefully prioritized investments, aligned spending with clear development milestones, and maintained a prudent approach to capital allocation. This disciplined approach positions Biohaven to advance these three key programs with the highest probability of generating near term value efficiently while preserving flexibility to opportunistically support sustained innovation in our oncology portfolio and with other emerging targets."Full Year and Recent Business UpdatesCorporate updates:Initiated strategic cost optimization efforts across portfolio in 4Q 2025 to focus forward-looking spend on three value-driving, late-stage clinical programs that will prioritize resources:Lead TRAP and MoDE extracellular degraders, for IgA nephropathy (BHV-1400) and Graves' disease (BHV-1300);Opakalim, selective Kv7 ion channel activator, in pivotal studies for focal epilepsy; andTaldefgrobep alfa, myostatin-activin pathway inhibitor, in Phase 2 for obesity.In November 2025, we announced a restructuring of business priorities and an optimization of resource allocation, expected to achieve an approximately 60% reduction in annual direct R&D spend (which excludes personnel and share-based compensation). The fourth quarter results that we are releasing today reflect the commencement of those restructuring and optimization efforts. Subsequently, in 2026, we have reported positive early clinical experience from clinically validated, extracellular protein degraders using our proprietary MoDE and next-generation, highly selective TRAP degraders. In addition, subsequent to December 31, 2025, the Company issued and sold an additional 17.2 million common shares for net proceeds of $178.9 million, including a directed common share sale to Janus Henderson Investors as a block transaction under the Company's "at-the-market" offering program. As reported today, we are accelerating pivotal degrader trials using our proprietary MoDE and next-generation, highly selective TRAP degraders.  We continue to expect that the restructuring and optimization efforts that we commenced in the fourth quarter of 2025 will lead to a reduction in annual direct R&D spend, although the acceleration of these trials is expected to reduce the level of that reduction.Continued pipeline advancement and ongoing business activities through capital raising initiatives:In 2026, the Company issued and sold 17.2 million common shares for net proceeds of $178.9 million, including a $125 million in gross proceeds directed common share sale to Janus Henderson Investors as a block transaction under the Company's "at-the-market" offering program.In November 2025, the Company generated gross proceeds of approximately $200 million in an upsized public offering of common shares.In April 2025, the Company announced an investment of up to $600 million by Oberland Capital, comprised of $250 million funded at closing, $150 million available at the Company's option upon the achievement of regulatory milestones related to troriluzole, and up to $200 million available at the mutual agreement of the parties for permitted strategic acquisitions and related costs and expenses.Entered into MoU with KAUST: In January 2026, Biohaven entered into a memorandum of understanding (MoU) with the King Abdullah University of Science and Technology (KAUST) to collaborate on discovery efforts and leverage the University's technology capabilities including strengths in its Smart Health initiatives, generative AI, and supercomputing. The agreement was established as part of an initiative to further advance and accelerate next-generation degrader development.Key program updates:Gd-IgA1 TRAP Degrader (BHV-1400) and IgG MoDE Degrader (BHV-1300)   Biohaven MoDE and TRAP extracellular protein degraders harness selectivity, rapidity and patient-friendly self-administration to remove disease-causing proteins from the body to potentially treat a range of diseases. Each MoDE or TRAP degrader is a novel bispecific molecule that targets a specific form of disease-causing circulating protein and directs it to the liver for degradation by the endosomal/lysosomal pathway.IgAN Program: In January 2026, first dosing of TRAP degrader BHV-1400 in IgAN patients achieved early observations of both biomarker and clinical responses including: selective lowering of only the disease-causing galactose-deficient IgA1 (Gd-IgA1) while sparing off-target effects on healthy antibodies (IgA, IgM, IgE, IgG), resolution of blood in the urine (hematuria), deep reductions in proteinuria, and improvement in fatigue and kidney function (eGFR) within weeks.In May 2025, the Company shared results from the ongoing Phase 1 study in healthy volunteers, in which a single dose of BHV-1400 was subcutaneously administered at a dose of 500 mg and achieved rapid, deep and sustained reductions in Gd-IgA1 of up to 81%, with a median reduction of 66%. Reductions occurred within hours of each dose, were progressive, and were sustained for weeks after a single dose administration. Effects were selective, with no significant reductions observed in other immunoglobulins: IgA, IgG, IgE, or IgM.The pivotal IgAN study is expected to initiate in the first quarter of 2026.Graves' Disease Program: In January 2026, the Company announced first-in-patient clinical experience with IgG MoDE degrader BHV-1300 resulted in a complete suppression of disease-causing TSH receptor-stimulating antibodies with accompanying normalization of previously elevated thyroid hormones within weeks after dosing a patient with Graves' disease.In May 2025, the Company released positive data from its Phase 1 multiple-dose study in healthy volunteers, whereby SC administered BHV-1300 achieved IgG reductions up to 87%. Median maximum reductions of 83% were achieved within 18 days.Biohaven is planning a pivotal study of BHV-1300 in Graves' disease in the second half of 2026.Broad Degrader Portfolio: With clinical proof-of-concept now established, Biohaven is positioned to advance multiple next-generation degraders targeting high value immune-mediated disease indications and explore strategic partnerships to advance the breadth of this platform technology, including:BHV-1420, a PLA2R autoantibody specific TRAP degrader, for membranous nephropathy;BHV-1450, an IgG4 specific MoDE degrader, for potential indications including pemphigus vulgaris and myasthenia gravis with anti-MuSK antibodies;BHV-1440, a TSHR autoantibody specific TRAP degrader, as the next-generation of immune therapy for Graves' disease and thyroid eye disease;BHV-6500, a proinsulin and insulin autoantibody TRAP degrader, for type 1 diabetes;BHV-1490, an IgM MoDE degrader for cryoglobulinemia, Waldenstrom's macroglobulinemia and IgM neuropathy;BHV-1310, a next generation IgG MoDE degrader, for management of rare IgG-mediated indications;BHV-1600, a beta-1 adrenergic receptor autoantibody degrader, for cardiomyopathy; and,Multiple undisclosed degrader targets in early discovery development.Opakalim Next-generation, selective Kv7 activator, targeting a clinically validated mechanism of action for the treatment of epilepsy.Focal Epilepsy Program:In January 2026, the Company announced preliminary data from the ongoing OLE study in focal epilepsy, demonstrating clinically meaningful reductions in seizure frequency compared to pretreatment baseline. Specifically, the majority of participants treated with opakalim 75 mg once daily who completed at least six months of OLE treatment showed ≥50% reductions in seizure frequency compared to pretreatment baseline.Opakalim was well-tolerated in the OLE with a low incidence of CNS adverse events, representing a potential paradigm-shift for patients with a highly favorable and differentiated safety profile compared to other approved or investigational antiseizure medicines.Top-line results from the first of two pivotal studies of opakalim in the treatment of focal epilepsy are expected in the second half of 2026.Taldefgrobep alfaNovel inhibitor of the myostatin-activin signaling pathway with the potential to achieve high quality weight loss in people living with obesity.Obesity Program:Biohaven initiated a taldefgrobep Phase 2 proof-of-concept study in obesity in 4Q 2025; topline results are expected in the second half of 2026. This randomized, double-blind, placebo-controlled, 24-week, dose-ranging study is evaluating the efficacy and tolerability of once-weekly and once-monthly taldefgrobep as monotherapy, via self-administered autoinjector, in adults living with overweight and obesity.Taldefgrobep's differentiated mode of action targets fat reduction, building muscle, and increasing bone density.In a Phase 1 study, taldefgrobep has demonstrated beneficial changes in fat mass and lean mass in non-obese populations, including healthy adult participants. Participants who received taldefgrobep once-weekly realized significant reductions in total body fat mass (>6%) and increases in lean muscle mass (up to 4%) after one month of dosing.These body composition parameters continued to demonstrate additional improvements after cessation of dosing associated with the persistence of the pharmacologically active taldefgrobep-myostatin complex, suggesting the drug may support extended dosing intervals.Nonclinical data demonstrated that the complex can also potently inhibit the Activin E-ALK7 signaling axis within adipocytes, further underpinning the complementary mechanistic advantages of taldefgrobep in both growing muscle and reducing fat.Taldefgrobep has demonstrated a highly favorable safety and tolerability profile in >700 clinical trial participants studied to date.Other pipeline updates:As previously disclosed in 4Q 2025, development of programs outside of the key prioritized programs outlined above may be substantially downsized, paused or delayed as a result of the Company's strategic reprioritization. Antibody Drug Conjugates (ADCs)BHV-1510 is a next-generation ADC targeting TROP2-expressing carcinomas, or malignant neoplasms of epithelial origin. BHV-1530 is an FGFR3-directed ADC with potential indications in urothelial cancers and other solid tumors. BHV-1510: In December 2025, the Company presented data from the ongoing BHV1510-101 trial at the ESMO Immuno-Oncology Congress. BHV-1510, demonstrated encouraging early clinical activity and differentiated safety profile in a Phase 1 study in combination with the anti-PD-1 cemiplimab:In a pretreated population of participants with advanced/metastatic cancer and the majority with prior PD-(L)1 treatment, BHV-1510 2.5 mg/kg Q3W plus cemiplimab resulted in confirmed objective response rates 3/5 (60%) in non-small cell lung cancer (NSCLC), 4/4 (100%) in endometrial cancer, and 1/2 (50%) in urothelial cancer.There were low rates of adverse events attributed to unconjugated payload such as hematological toxicities and diarrhea, and there were no cases of interstitial lung disease, showing a differentiated safety profile of BHV-1510 from other Trop2 ADCs. The most frequent toxicity observed was oral mucositis/stomatitis. This is a well-known class effect which is manageable.The pharmacokinetic profile for BHV-1510 was favorable, the unconjugated payload concentration is low, indicating a highly stable ADC in the circulation.In January 2026, the Company announced the initiation of subcutaneous administration with BHV-1510, the first and only Trop2 ADC in clinic with the potential for subcutaneous delivery.BHV-1530: In December 2025, the Company noted that dose escalation continues in the ongoing Phase 1 study; no dose limiting toxicities have been observed to date. The study is evaluating BHV-1530 in patients with advanced solid tumors, including patients whose cancers have progressed on or are intolerant to standard therapy.TYK2/JAK1 Inhibition BHV-8000 is an oral, brain-penetrant, selective TYK2/JAK1 inhibitor with broad potential for neuroinflammatory and neurodegenerative disorders.Parkinson's disease (PD): Aligned with our current portfolio prioritization, a more focused execution plan concentrating on a select number of sites in the US has been implemented for the ongoing Phase 2/3 clinical trial in early Parkinson's disease to optimize efficiency and resource allocation.Biohaven Discovery Engine:Discovery has advanced multiple novel development candidates for future clinical development. Beyond the degrader platform, these include:BHV-1955 targeting the oxytocin receptor centrally for the treatment of tinnitus;BHV-2120, a brain-penetrant, oral, small molecule TRPM3 inhibitor for epilepsy;BHV-8555, a brain-penetrant, oral, small molecule preventing alpha-synuclein aggregation in Parkinson's disease; and,BHV-8100, a brain-penetrant, oral, small molecule activating the M2 isoform of pyruvate kinase (PKM2) for neurodegenerative disorders and aging.Expected Upcoming Milestones:We believe Biohaven is well positioned to achieve significant, value-creating milestones in 2026 across numerous programs:Lead TRAP and MoDE Extracellular Protein Degraders (BHV-1400 and BHV-1300)BHV-1400: Completed 4Q25 meeting with the U.S. Food and Drug Administration (FDA) to align on pivotal IgAN study design; study to initiate in 1Q 2026BHV-1300: Pivotal study initiation in Graves' disease expected in 2H 2026.Kv7 Activator (Opakalim): Continue two Phase 2/3 studies in focal epilepsy; initial topline results expected in the second half of 2026.Myostatin-Activin Pathway Inhibitor (Taldefgrobep alfa): Initiated Phase 2 study in obesity in 4Q 2025; topline results expected in the second half of 2026Capital Position:Cash, cash equivalents, marketable securities and restricted cash as of December 31, 2025, totaled approximately $322.0 million.  Subsequent to December 31, 2025, the Company issued and sold an additional 17.2 million common shares for net proceeds of $178.9 million and made a $42.7 million payment to Knopp Biosciences LLC (Knopp) to settle the 2025 Additional Consideration True-Up liability recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp in May 2024 (the Knopp Amendment).Fourth Quarter 2025 Financial Highlights:Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $121.9 million for the three months ended December 31, 2025, compared to $167.5 million for the three months ended December 31, 2024. The decrease of $45.5 million was primarily due to decreases in direct program spend, largely related to BHV-2100, opakalim and BHV-1530, which were partially offset by a $12.0 milestone payment for BHV-1510 during the fourth quarter of 2025 as well as increased program spend for our lead degraders, BHV-1300 and BHV-1400. The decrease in direct spend for BHV-1530 was primarily due to a one-time upfront payment of $6.0 million cash and non-cash issuance of common shares valued at $8.6 million for a development and license agreement entered into in the fourth quarter of 2024. Non-cash share-based compensation expense was $10.5 million for the three months ended December 31, 2025, an increase of $3.4 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $20.8 million for the three months ended December 31, 2025, compared to $22.5 million for the three months ended December 31, 2024. The decrease of $1.7 million was primarily due decreased personnel costs, excluding non-cash share based compensation.  Non-cash share-based compensation expense was $6.5 million for the three months ended December 31, 2025, an increase of $0.8 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.Other (Expense) Income, Net: Other (expense) income, net was other expense, net of $2.5 million for the three months ended December 31, 2025, compared to other income, net of $3.1 million for the three months ended December 31, 2024. The decrease of $5.6 million was primarily due to an increase in losses recorded for the change in the fair value the 2025 Additional Consideration True-Up derivative liability, decreased investment income, and a loss recognized on the impairment of a purchased loan commitment asset recorded in connection with the Note Purchase Agreement with Beetlejuice SA LLC, an affiliate of Oberland Capital Management LLC, entered into during the second quarter of 2025 (the NPA). These decreases were partially offset by an increase in gains related to changes in fair value of our notes payable liability under the NPA.Net Loss: Biohaven reported a net loss for the three months ended December 31, 2025 of $145.6 million, or $1.21 per share, compared to $186.8 million, or $1.85 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended December 31, 2025 was $107.9 million, or $0.90 per share, compared to $173.3 million, or $1.71 per share, for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.Full Year 2025 Financial HighlightsR&D Expenses: R&D expenses, including non-cash share-based compensation, were $635.1 million for the year ended December 31, 2025, compared to $795.9 million for the year ended December 31, 2024. The decrease was primarily due to a one-time non-cash expense during the year ended December 31, 2024 of $171.9 million paid in connection with the Knopp Amendment (the Knopp Amendment reduced our potential future milestone payments by $867.5 million and replaced the scaled high single digit to low teens royalty payment obligations with a flat royalty payment in the mid-single digits for the Kv7 programs). The decrease was also due to decreased program expense for BHV-2000, troriluzole, and opakalim. These decreases were partially offset by increased direct program spend for advancing clinical trials and preclinical research programs in 2025, including one-time developmental milestone payments of $15.0 million, $12.0 million, and $10.0 million for our BHV-8000, BHV-1510 and BHV-1530 programs, respectively, as well as increased non-cash share-based compensation expense. Non-cash share-based compensation expense was $72.8 million for the year ended December 31, 2025, an increase of $30.2 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.G&A Expenses: G&A expenses, including non-cash share-based compensation costs, were $110.3 million for the year ended December 31, 2025, compared to $89.2 million for the year ended December 31, 2024. The increase of $21.1 million was primarily due to increased non-cash share-based compensation expense and increased expenses related to fees incurred in connection with the NPA and other legal costs. Non-cash share-based compensation expense was $39.6 million for the year ended December 31, 2025, an increase of $10.2 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.Other Income (Expense), Net: Other income, net was other income of $8.0 million for the year ended December 31, 2025, compared to other income of $39.4 million for the year ended December 31, 2024. The decrease of $31.4 million was primarily due to an increase in losses recorded for the non-cash changes in the fair value of our forward contracts and derivative liabilities recorded in connection with the Knopp Amendment, decreased investment income, and other expense recognized to write-off an impaired asset related to the NPA during the year ended December 31, 2025. This was partially offset by an increase in non-cash gains related to changes in fair value of our notes payable liability under the NPA.Net Loss: The Company reported a net loss attributable to common shareholders for the year ended December 31, 2025 of $738.8 million, or $6.86 per share, compared to $846.4 million, or $9.28 per share for the same period in 2024. Non-GAAP adjusted net loss for the year ended December 31, 2025 was $597.0 million, or $5.55 per share, compared to $790.6 million, or $8.67 per share for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives.  A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.Non-GAAP Financial MeasuresThis press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, which are adjusted to exclude non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares, and changes in the fair value of derivative liabilities, which do not correlate to actual cash payment obligations in the relevant periods. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance and can assist investors in comparing Biohaven's performance between periods.In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this news release.About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE™ and TRAP™ extracellular protein degradation for immunological diseases; and myostatin inhibition for neuromuscular and metabolic diseases, including obesity.Forward-looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected timing and amounts of funding under the NPA. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates and regarding reduction in annual direct R&D spend, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates and the expected timing thereof; the potential for Biohaven's product candidates to be successful therapies; the effectiveness of restructuring of business priorities; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.BIOHAVEN LTD.CONSOLIDATED STATEMENTS OF OPERATIONS(Amounts in thousands, except share and per share amounts)(Unaudited)


Three Months Ended
December 31,
Twelve Months Ended
December 31,

2025
2024
2025
2024Operating expenses:







Research and development
$        121,945
$        167,473
$        635,065
$        795,871General and administrative
20,789
22,458
110,313
89,240Total operating expenses
142,734
189,931
745,378
885,111Loss from operations
(142,734)
(189,931)
(745,378)
(885,111)Other (expense) income, net
(2,470)
3,136
7,998
39,424Loss before provision (benefit) for income taxes
(145,204)
(186,795)
(737,380)
(845,687)Provision (benefit) for income taxes
351
48
1,442
735Net loss
$      (145,555)
$      (186,843)
$      (738,822)
$      (846,422)Net loss per share — basic and diluted
$             (1.21)
$             (1.85)
$             (6.86)
$             (9.28)Weighted average common shares outstanding— basic and diluted     
120,187,689
101,054,895
107,624,885
91,234,337 BIOHAVEN LTD.CONSOLIDATED BALANCE SHEETS(Amounts in thousands, except share amounts)






December 31, 2025
December 31, 2024

(Unaudited)

Assets



Current assets:



Cash and cash equivalents
$                229,957
$                  99,134Marketable securities
89,180
386,857Prepaid expenses
47,022
49,376Other current assets
2,170
3,105Total current assets
368,329
538,472Property and equipment, net
15,964
17,320Intangible assets
18,400
18,400Goodwill
1,390
1,390Other non-current assets
47,364
39,525Total assets
$                451,447
$                615,107Liabilities and Shareholders' Equity



Current liabilities:



Accounts payable
$                  11,643
$                  18,029Accrued expenses and other current liabilities
104,291
51,487Forward contract and derivative liability
—
84,710Total current liabilities
115,934
154,226Non-current operating lease liability
39,958
32,782Notes payable
238,900
—Other non-current liabilities
4,583
4,663Total liabilities
399,375
191,671Shareholders' Equity:



Preferred shares, no par value; 10,000,000 shares authorized, no shares issued
and outstanding as of December 31, 2025 and 2024
—
—Common shares, no par value; 200,000,000 shares authorized as of December
31, 2025 and 2024; 132,775,113 and 101,221,989 shares issued and
outstanding as of December 31, 2025 and 2024, respectively
1,934,276
1,656,702Additional paid-in capital
193,984
112,369Accumulated deficit
(2,084,536)
(1,345,714)Accumulated other comprehensive income
8,348
79Total shareholders' equity
52,072
423,436Total liabilities and shareholders' equity
$                451,447
$                615,107 BIOHAVEN LTD.RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL MEASURES(Amounts in thousands, except share and per share amounts)(Unaudited)






Three Months Ended
December 31,
Twelve Months Ended
December 31,

2025
2024
2025
2024Reconciliation of GAAP to Non-GAAP adjusted net loss:







GAAP net loss
$   (145,555)
$   (186,843)
$   (738,822)
$   (846,422)Add: non-cash share-based compensation expense
16,952
12,695
112,361
71,963Add: loss (gain) from change in fair value of derivatives
20,700
890
29,500
(16,140)Non-GAAP adjusted net loss
$   (107,903)
$   (173,258)
$   (596,961)
$   (790,599)








Reconciliation of GAAP to Non-GAAP adjusted net loss per share — basic and diluted:



GAAP net loss per share — basic and diluted
$          (1.21)
$          (1.85)
$          (6.86)
$          (9.28)Add: non-cash share-based compensation expense
0.14
0.13
1.04
0.79Add: loss (gain) from change in fair value of derivatives
0.17
0.01
0.27
(0.18)Non-GAAP adjusted net loss per share — basic and diluted
$          (0.90)
$          (1.71)
$          (5.55)
$          (8.67) MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.Investor Contact:Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli @Dr ExtremeMedia Contact:Mike Beyer
Sam Brown Inc.
mikebeyer @Punch Drunk 



View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-reports-recent-business-developments-and-fourth-quarter-and-full-year-2025-financial-results-302701521.htmlSOURCE Biohaven Ltd.

Original: Biohaven Reports Recent Business Developments and Fourth Quarter and Full Year 2025 Financial Results

Upward momentum. Hope it continues...

Keep thinking eventually we have to get some good news...

I feel like there is some accumulation happening in expectation
of what should be positive news in January.

Not as bad as it first looked. Future uncertain...

CRL from FDA, per Dew discussion on BTValue board.

Ouch!

Pre-market looks ugly

Another large dump about 2 o'clock.

Looks like a large dump at about noon. Hope we stabilize.

So I guess if we break 18. then should go deep into the twenties...

Good bump today. Hope we see some follow thru

Another pullback...why

Another nice bounce

Nice bounce

Would like to see this close above 16.
May break into the 20's if that happens

https://www.panabee.com/news/fda-drops-advisory-committee-for-biohaven-s-troriluzole-q4-decision-looms
Looks like a potential breakout

Been stuck in this range for a few months now

Pre-market bounce over 16. Hoping we run in to the 20's soon.

Strong showing so far today

Rebounding nicely...

Earnings were a downer. More bad news. This stock needs a positive catalyst fast...

This stock has really been beaten down. Some analysts still say it should be over $50.00.
Wonder what's going on...

nice chart Monksdream

BHVN 10Q due 11/11

Positive data and a strong gap up.

Looks like a lot of Call options thru October at a $50 Strike.
Some people expecting this to be in the upper fifties within the next month.

CYTK chart?

BHVN a buy under $35

A positive trend is developing...

Positive day and insiders have been buying. Should have more upside potential...

Wonder why the downward pressure on this stock. One institutional investor reduced it's holding but a few others bought in to more than offset that. strange...

This bio has a lot going for it and that share price growth is very healthy/ Heck, it quadrupled! in one year. Got some for my long term growth.

Biohaven Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Developments

Source: PR Newswire (US)
Driving clinical, regulatory, and operational excellence across five innovative platforms focused on immunology, neuroscience, and oncology:
Portfolio targeting large indications including obesity, epilepsy, bipolar disorder, depression, obsessive-compulsive disorder (OCD), migraine, pain, Alzheimer's disease, Parkinson's disease, multiple sclerosis, rheumatoid arthritis, and cancer. Also advancing potential novel treatments for rare autoimmune and inflammatory diseases, including myasthenia gravis, cardiomyopathy, spinal muscular atrophy (SMA) and IgA nephropathy.
Pivotal clinical data milestones expected across three distinct programs:
First-in-human Phase 1 study with BHV-1300 initiated in 1Q 2024. Preliminary immunoglobulin G (IgG) lowering data from the single ascending dose (SAD) portion of the study expected in late 1Q 2024/early 2Q 2024. The MAD portion of the study is being planned in a relevant patient population with the possibility of benefit from BHV-1300.
Phase 3 database lock for interim efficacy analysis with troriluzole in OCD remains on schedule for 1Q 2024, with results expected in 2Q 2024.
Phase 3 topline data in ongoing fully enrolled SMA study with taldefgrobep expected 2H 2024.
Multiple trial initiations and Investigational New Drug (IND) filings projected over next three years present potential for continued growth and value creation.
NEW HAVEN, Conn., Feb. 29, 2024 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), 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 reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a review of recent accomplishments and anticipated upcoming developments.

(PRNewsfoto/BIOHAVEN LTD)

Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "We embarked on a bold execution plan after completing our spin-off, re-emerging as a separate entity focused on immunology, neuroscience, and oncology. In just over a year's time, we made tremendous strides in progressing our programs, providing impressive and supportive pre-clinical and clinical outcomes for our key stakeholders, including patients, who may gain access to differentiated drugs for difficult to treat medical conditions.

Today we are focused on delivering outcomes across five platforms spanning inflammation and immunology, ion channel modulation, myostatin inhibition, glutamate modulation, and oncology. In the year ahead, we expect to deliver potentially groundbreaking data across three distinct programs. In the near-term, we expect to report first-in-human Phase 1 IgG lowering data with our pan-IgG degrader, BHV-1300, with broad potential application. Given the profoundly deep and rapid reduction in IgG levels observed in non-human primate studies, unique ability to co-administer with Fc-containing biologics, ease of self-administration, and favorable toxicology profile observed to date, we are eagerly awaiting the proof-of-concept human data. We also plan to report OCD Phase 3 topline data in the second quarter of 2024 from an interim analysis with our glutamate modulating agent, troriluzole. There has been little to no innovation in advancing treatments for this disorder in decades and a win in this Phase 3 trial would be a substantial clinical success for the three million patients across the OCD community, many of whom are plagued by debilitating intrusive thoughts and compulsions. And finally, we're expecting Phase 3 topline data in the second half of the year with our anti-myostatin compound, taldefgrobep alfa, for patients with SMA.

Dr. Coric continued, "Beyond data expectations, we are excited with the progress made across the balance of our portfolio, starting with our Kv7 ion channel activation program, where we have operationalized over 110 clinical trial sites in our focal epilepsy trials and are advancing development programs in generalized epilepsy, bipolar disorder, and major depressive disorder. Excitement is also mounting for the potential of taldefgrobep alfa in weight loss, a compound that we believe will maintain and grow muscle mass as a means of helping to address the global obesity crisis. We also expect to deliver additional trial initiations across our broad IgG degrader program targeting both large indications and rare autoimmune/inflammatory diseases, TYK2/JAK1 inhibition in neuroinflammatory disorders, TRPM3 antagonism initially in migraine and neuropathic pain, and with our antibody drug conjugates program in an array of oncology indications. We are passionate about helping patients in need, and excited about our portfolio, the skilled drug development team we have in place, and the vast opportunity in the year ahead."

Full Year and Recent Business Highlights

Ion Channel Platform - Milestones and Next Steps:

Kv7 Ion Channel Activation: Epilepsy & Neuropsychiatric Indications
BHV-7000, the lead asset from the Kv7 platform, is a selective activator of Kv7.2/Kv7.3, a key ion channel involved in regulation of neuronal signaling and hyperexcitability.

Reported BHV-7000 Phase 1 study results: In December 2023, the Company reported full results from the BHV-7000 Phase 1 SAD and multiple ascending dose (MAD) studies examining doses up to 120mg daily, demonstrating BHV-7000 was well-tolerated at all doses studied without the typical central nervous system (CNS) adverse effects associated with other anti-seizure medications (ASMs), such as somnolence and cognitive/mood disturbances. Results were consistent with previously reported preclinical data demonstrating BHV-7000's lack of GABAA receptor activation and lack of adverse impacts on neurobehavior in preclinical testing.
Demonstrated CNS target engagement: In December 2023, the Company reported additional data from a Phase 1 electroencephalogram (EEG) biomarker study, where BHV-7000 demonstrated dose-dependent target engagement in the brain as shown by dose-dependent effects on EEG spectral power across all frequency bands. While changes in spectral power were observed across all frequency bands with BHV-7000, the minimal impact on slower frequencies (i.e., delta) is consistent with the low incidence of CNS adverse events observed in the BHV-7000 Phase 1 SAD/MAD studies. EEG delta activity is associated with somnolence, an undesirable CNS adverse event commonly reported with other ASMs.
Developed once-daily formulation: In September 2023, the Company announced it had formulated an extended release, once-a-day tablet designed to achieve target therapeutic concentrations (25mg, 50mg and 75mg). This dosing approach with a Kv7 activator will allow for assessment of distinct target concentrations over a wide range, above and below projected efficacious EC50 drug concentrations, not previously feasible with drugs in this class.
Sites operationalized: In January 2024, the Company completed its End-of-Phase 2 meeting with FDA to advance to Phase 3 trials and announced that more than 110 global clinical sites have been selected in the ongoing focal epilepsy trial, with enrollment planned for 1Q 2024.
Upcoming trial initiations: The Company expects to initiate Phase 2/3 programs in focal epilepsy in 1Q 2024 and in generalized epilepsy in 2Q 2024; the Company also expects to initiate a Phase 2 study in major depressive disorder and a Phase 2/3 study in bipolar disorder in 1H 2024.
TRPM3 Ion Channel Antagonism: Migraine & Neuropathic Pain
BHV-2100 is an oral, selective TRPM3 antagonist offering a novel, non-addictive treatment for migraine and neuropathic pain.

Phase 1 study data supports evaluation in acute migraine: In January 2024, the Company detailed preliminary pharmacokinetic (PK) and safety data from an ongoing Phase 1 study; in the study, BHV-2100 was rapidly absorbed, achieved 90% inhibitory concentrations within one hour, and was well tolerated at projected therapeutic concentrations
Upcoming trial initiations: The Company expects to initiate a BHV-2100 Phase 2 study in acute migraine in 2H 2024 and conduct a POC study for neuropathic pain in 2H 2024.
Inflammation and Immunology Platform - Milestones and Next Steps:

Targeted Extracellular Protein Degradation:
Molecular Degraders of Extracellular Proteins (MoDEs™) uniquely harness the hepatic ASGPR receptor for efficient and safe removal of circulating pathogenic targets; BHV-1300, is an IgG degrader.

Reported on progress with BHV-1300: The Company has demonstrated the effect of a single dose of BHV-1300 in lowering IgG in nonhuman primates (NHPs), previously reporting over 75-80% reduction of IgG levels from baseline in three days. These data compare favorably to other IgG targeting agents, such as the FcRn inhibitor efgartigimod, where reduction of IgG levels, following a single dose, was shown to be approximately 50% in 5-7 days. In September 2023, the Company announced positive multiple dose, pharmacodynamic (PD) data from a NHP study with BHV-1300 demonstrating dose-dependent reductions of over 90% in IgG levels from baseline, suggesting the potential for achieving greater efficacy with finely calibrated, deeper IgG reductions as compared with existing standard of care FcRn targeting treatments.
Demonstrated potential for same-day, co-administration with Fc-containing biologics: In January 2024, the Company presented new NHP data demonstrating that Biohaven's IgG degrader technology allows for co-administration with Fc-containing biologics; PK of Humira® was unaltered after being dosed 12 hours after BHV-1300 administration.
Reported preclinical pharmacodynamic data with single dose of BHV-1310: In January 2024, the Company showed that a next generation and optimized IgG degrader, BHV-1310, allowed for much deeper 90% reductions in IgG after a single dose. Given the deep and rapid reductions observed, the Company believes BHV-1310 may have potential application in acute settings.
Unveiled plans for BHV-1600: next-generation, selective degrader targeting ß1-AR autoantibodies: In January 2024, the Company reported preclinical data demonstrating degradation of anti-ß-1AR antibody in mice. The Company expects to file an IND application and initiate a first-in-human Phase 1 study in 2H 2024. BHV-1600 will initially be evaluated in patients with dilated cardiomyopathy.
Near-term data expected: The Phase 1 SAD study examining BHV-1300 in healthy subjects was initiated in 1Q 2024 and the Company expects preliminary results in late 1Q 2024/early 2Q 2024. The FDA indicated that the MAD assessment of BHV-1300 should be performed in a relevant patient population. Upon completion of the SAD study, Biohaven is planning the MAD portion of the study in a relevant patient population with the possibility of benefit from BHV-1300.
Upcoming trial initiations: A total of 4 INDs are expected for the degrader program in 2024.
TYK2/JAK1 Inhibition:
BHV-8000 is an oral, brain-penetrant, selective TYK2/JAK1 inhibitor with broad potential for neuroinflammatory and neurodegenerative disorders.

Successfully completed single ascending dose cohorts; advanced multiple ascending dose cohorts in ongoing Phase 1 study of TYK2/JAK1 inhibitor, BHV-8000: In January 2024, the Company provided a program update for the ongoing Phase 1 study designed to evaluate the safety, tolerability, PK and PD of single and multiple ascending doses of BHV-8000 in healthy volunteers. The SAD cohorts have now completed dosing (10, 20 and 30 mg); in the MAD cohorts, the Company completed up to the 20 mg dose. Based on the preliminary data available, projected therapeutic concentrations of BHV-8000 were achieved and BHV-8000 was well tolerated with only mild adverse events reported.
Upcoming trial initiations: The Company expects to initiate a Phase 2 study in multiple sclerosis in 2H 2024, a Phase 2a study in prevention of amyloid therapy induced ARIA in 2H 2024, and Phase 2/3 studies in early Parkinson's disease and early Alzheimer's disease in 2H 2024.
Myostatin Platform - Milestones and Next Steps:
Taldefgrobep alfa is a novel myostatin inhibitor optimized to target myostatin and associated signaling pathways of muscle growth. Taldefgrobep alpha also has promise as a potential treatment for obesity and, in preclinical models as well as preliminary healthy human studies, has demonstrated meaningful reductions in fat mass, the primary pathogenic tissue in obesity, while increasing lean mass. Biohaven is also studying taldefgrobep in a global Phase 3 study in Spinal Muscular Atrophy to enhance muscle mass and function in patients treated with standard-of-care gene therapy treatments.

Preclinical data demonstrated taldefgrobep alfa reduces fat and improves lean mass: In October 2023, the Company presented preclinical data demonstrating the ability of taldefgrobep alfa to significantly reduce fat mass while increasing lean mass in an obese mouse model at The Obesity Society's annual ObesityWeek conference.
Completed enrollment in pivotal Phase 3 study in SMA: In September 2023, the Company announced that it had completed enrollment in RESILIENT, a pivotal Phase 3 study designed to evaluate the efficacy and safety of taldefgrobep as adjunctive therapy to enhance muscle mass and function in SMA patients treated with standard-of-care treatments. In July 2023, the Company announced that taldefgrobep received orphan drug designation (ODD) from the European Commission for the treatment of SMA. Taldefgrobep previously received Fast-Track and ODD from the FDA.
Topline data expected: The Company expects to announce Phase 3 topline data from the ongoing SMA study in 2H 2024.
Upcoming trial initiation: The Company expects to initiate a Phase 2 study in patients with obesity in 2Q 2024.
Glutamate Modulation Platform - Milestones and Next Steps:
Troriluzole is a novel glutamate modulator currently being evaluated in Phase 3 trials for obsessive-compulsive disorder as an adjunctive therapy in patients with an inadequate response to existing standard-of-care treatment.

Topline data from interim analysis in OCD expected in 2Q 2024: In January 2024, the Company shared an update on the ongoing Phase 3 trial in OCD. The Company remains on schedule for a database lock in 1Q 2024, with an interim efficacy analysis expected in 2Q 2024.
Biohaven has also continued to have constructive dialogue with the FDA regarding its SCA development program and potential future data analyses to address regulatory concerns in the previously issued refuse-to-file decision on its NDA application for SCA3. Biohaven will provide further updates on the SCA development program as warranted by any continued positive progress from the outcome of future regulatory interactions on this topic.
Next-Generation ADC Platform - Milestones and Next Steps:
Biohaven's antibody drug conjugate (ADC) technology is focused on novel conjugation chemistry with the potential to be superior to the current industry standard maleimide and lipophilic click chemistry. BHV-1510 is a TROP2 directed ADC, with a highly differentiated efficacy and safety profile providing an opportunity to broaden therapeutic margin, increase time on treatment, and improve efficacy; BHV-1500 demonstrated superior efficacy to Adcetris® (brentuximab vedotin) and improved survival in a mouse xenograft model.

Upcoming trial initiations: The Company completed its regulatory interactions to begin first-in-human studies with BHV-1510 (TROP2 directed ADC) and expects to initiate a Phase 1 trial in 2Q 2024. The company also expects to submit an IND for BHV-1500 (next-generation brentuximab ADC) in 2H 2024.
Corporate Updates:

Public offering: On October 5, 2023, the Company closed its previously announced underwritten public offering of 11,761,363 of its common shares, which included the full exercise of the underwriters' option to purchase 1,534,090 additional shares, at the public offering price of $22.00 per share. The net proceeds raised in the offering, after deducting underwriting discounts and estimated expenses of the offering payable by the Company, were approximately $242.4 million. As of February 26, 2024, we had 81,579,914 common shares outstanding.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant, value-creating milestones in 2024 across numerous programs:

Selective Kv7 Activator:

Initiate BHV-7000 Phase 2/3 program in focal epilepsy in 1Q 2024
Initiate BHV-7000 Phase 2/3 study in generalized epilepsy in 2Q 2024
Initiate BHV-7000 Phase 2/3 study in bipolar disorder in 1H 2024
Initiate BHV-7000 Phase 2 study in major depressive disorder in 1H 2024
Troriluzole:

Database lock in 1Q 2024 and report troriluzole Phase 3 interim efficacy analysis topline results in OCD in 2Q 2024
Taldefgrobep alfa:

Initiate taldefgrobep Phase 2 study in obesity in 2Q 2024
Report taldefgrobep Phase 3 topline results in SMA in 2H 2024
First-in-class TRPM3 Antagonist:

Initiate BHV-2100 Phase 2 study in acute migraine in 2H 2024
Conduct BHV-2100 POC study for neuropathic pain in 2H 2024
TYK2/JAK1 Inhibitor:

Initiate BHV-8000 Phase 2 study in Multiple Sclerosis in 2H 2024
Initiate BHV-8000 Phase 2a study in prevention of amyloid therapy induced ARIA in 2H 2024
Initiate BHV-8000 Phase 2/3 study in early Parkinson's disease in 2H 2024
Initiate BHV-8000 Phase 2/3 study in early Alzheimer's disease in 2H 2024
Extracellular protein degradation platform

BHV-1300 first-in-human clinical data demonstrating IgG lowering expected late 1Q 2024/early 2Q 2024
A total of 4 INDs are expected for the degrader program in 2024
Next Generation ADC Platform:

Initiate Phase 1 trial of BHV-1510 (TROP2 directed ADC) in 2Q 2024
File IND for BHV-1500 (next generation brentuximab ADC) in 2H 2024
Capital Position:

Cash, cash equivalents and marketable securities as of December 31, 2023 was $385.5 million, which includes $3.7 million of restricted cash.

Fourth Quarter 2023 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $134.8 million for the three months ended December 31, 2023, compared to $137.0 million for the three months ended December 31, 2022. The decrease of $2.2 million was primarily due to decreased non-cash share-based compensation expense during the three months ended December 31, 2023, and $5.2 million of one-time employee costs related to the Pfizer acquisition of Biohaven Pharmaceutical Holding Company Ltd. (the Former Parent) in the fourth quarter of 2022. This was partially offset by increased costs related to advancing additional clinical development program activities, including late Phase 2/3 studies and preclinical research programs, and recognition of one-time expenses of a $10.0 million cash payment and a $21.8 million non-cash issuance of common shares to acquire rights related to our agreement with Highlightll in the three months ended December 31, 2023, as compared to the same period in the prior year. Non-cash share-based compensation expense was $9.1 million for the three months ended December 31, 2023, a decrease of $60.3 million as compared to the same period in 2022. The decrease was primarily due to $61.7 million of expense allocated from the Former Parent recognized in connection with the settlement of outstanding Former Parent stock options and restricted stock units (RSUs) upon the effectiveness of the Former Parent's distribution to holders of all outstanding common shares of Biohaven and the spin-off of Biohaven from the Former Parent (the Separation) in the fourth quarter of 2022.

General and Administrative (G&A) Expenses: General and administrative expenses were $18.9 million for the three months ended December 31, 2023, compared to $76.4 million for the three months ended December 31, 2022. The decrease of $57.5 million was primarily due to decreased non-cash share-based compensation expense during the three months ended December 31, 2023, and $8.2 million of transaction-related expenses and $8.9 million of one-time employee costs related to the Pfizer acquisition of the Former Parent in the fourth quarter of 2022. Non-cash share-based compensation expense was $6.8 million for the three months ended December 31, 2023, a decrease of $39.5 million as compared to the same period in 2022. The decrease was primarily due to $39.7 million of expense allocated from the Former Parent recognized in connection with the settlement of outstanding Former Parent stock options and RSUs upon the effectiveness of the Separation in the fourth quarter of 2022.

Other Income (Expense), Net: Other income (expense), net was a net income of $7.7 million for the three months ended December 31, 2023, compared to a net expense of $1.8 million for the three months ended December 31, 2022. The increase of $9.6 million was primarily due to a $10.0 million impairment loss recognized during the fourth quarter of 2022 on our Artizan Series A-2 Preferred Stock Investment.

Net Loss: Biohaven reported a net loss for the three months ended December 31, 2023, of $144.8 million, or $1.81 per share, compared to $201.1 million, or $3.32 per share, for the same period in 2022. Non-GAAP adjusted net loss for the three months ended December 31, 2023 was $128.9 million, or $1.61 per share, compared to $77.3 million, or $1.27 per share for the same period in 2022. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and transaction-related costs incurred relating to the Company's spin-off from Biohaven Pharmaceutical Holding Company Ltd. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Full Year 2023 Financial Highlights

Note: As described in our Annual Report on Form 10-K, full year results for the year ended December 31, 2022 include direct and allocated expenses on a carve-out basis of accounting for the period prior to October 3, 2022, when the Company became a standalone public company.

R&D Expenses: R&D expenses, including non-cash share-based compensation, were $373.3 million for the year ended December 31, 2023, compared to $437.1 million for the year ended December 31, 2022. The decrease of $63.8 million was primarily due to a decrease of $91.5 million in personnel related costs primarily due to decreased non-cash share-based compensation expense, and reduced program spend for discontinued programs in 2023 compared to 2022. R&D expenses for the year ended December 31, 2022 also included a one-time $93.7 million expense for our Kv7 Platform acquisition, and a $25.0 million milestone relating to BHV-7000. The decrease was partially offset by increases in direct program spend for additional and advancing clinical trials, including late Phase 2/3 studies and preclinical research programs, and recognition of one-time expenses of a $10.0 million cash payment and a $21.8 million non-cash issuance of common shares to acquire rights related to our agreement with Highlightll, as compared to the same period in the prior year. Non-cash share-based compensation expense was $16.0 million for the year ended December 31, 2023, a decrease of $100.4 million as compared to the same period in 2022. Non-cash share-based compensation expense for the year ended December 31, 2022 included $108.7 million of expense allocated from the Former Parent, including $61.7 million of expense recognized in connection with the settlement of outstanding Former Parent stock options and RSUs upon the effectiveness of the Separation in the fourth quarter of 2022.

G&A Expenses: G&A expenses, including non-cash share-based compensation costs, were $62.8 million for the year ended December 31, 2023, compared to $130.9 million for the year ended December 31, 2022. The decrease of $68.1 million was primarily due to decreased non-cash share-based compensation costs. Non-cash share-based compensation expense was $12.8 million for the year ended December 31, 2023, a decrease of $64.4 million as compared to the same period in 2022. Non-cash share-based compensation expense for the year ended December 31, 2022 included $70.6 million of expense allocated from the Former Parent, including $39.7 million of expense recognized in connection with the settlement of each outstanding Former Parent stock option and RSU upon the effectiveness of the Separation in the fourth quarter of 2022.

Other Income (Expense), Net: Other income (expense), net was a net income of $26.5 million for the year ended December 31, 2023, compared to a net expense of $1.9 million for the three months ended December 31, 2022. The increase of $28.4 million was primarily due to increased net investment income of $14.5 million and increased service revenue from the Transition Service Agreement we entered into with the Former Parent of $5.2 million in 2023, as compared to the same period in the prior year, as well as a $10.0 million impairment loss recognized during the fourth quarter of 2022 on our Artizan Series A-2 Preferred Stock Investment.

Net Loss: The Company reported a net loss attributable to common shareholders for the year ended December 31, 2023 of $408.2 million, or $5.73 per share, compared to $570.3 million, or $12.75 per share for the same period in 2022. Non-GAAP adjusted net loss for the year ended December 31, 2023 was $379.4 million, or $5.33 per share, compared to $362.7 million, or $8.11 per share for the same period in 2022. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and transaction-related costs incurred relating to the Company's spin-off from Biohaven Pharmaceutical Holding Company Ltd. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Non-GAAP Financial Measures
This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, which are adjusted to exclude non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares, and transaction-related costs incurred relating to the Company's spin-off from Biohaven Pharmaceutical Holding Company Ltd., which are limited to a specific period of time and related to Biohaven Ltd. being established as a standalone public company. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance and can assist investors in comparing Biohaven's performance between periods.

In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this news release.

About Biohaven
Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; and antibody recruiting, bispecific molecules and antibody drug conjugates for cancer.

Forward-looking Statements
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first in class therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

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Biohaven Provides Overview of Clinical Progress, Regulatory Updates, and Pipeline Developments at R&D Day

Source: PR Newswire (US)
Submitted a new drug application (NDA) for troriluzole in Spinocerebellar Ataxia Type 3 (SCA3) to U.S. FDA in 2Q2023, marking the team's fourth NDA in approximately 3 years
Released additional data from Kv7 platform, including Phase 1 safety data by dose groups for BHV-7000 that further validates differentiated profile
Projected Phase 3 Spinal Muscular Atrophy trial to complete enrollment in 2023
Initiated Phase 1 study of brain penetrant TYK2/JAK1 inhibitor, BHV-8000, and anticipate beginning Phase 2 trial in Parkinson's disease next year
Highlighted robust pipeline with multiple INDs planned to be filed within the next year, including pan IgG degrader for multiple immune-mediated diseases in 2023
NEW HAVEN, Conn., May 31, 2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including ultra-rare disorders, will provide an overview of clinical progress, regulatory updates, and pipeline developments at an in-person R&D Day today, concurrently with the Yale Ventures' Innovation Summit 2023 taking place on May 31-June 1. Members of Biohaven's senior management team and key opinion leaders will meet with investors and research analysts.

(PRNewsfoto/BIOHAVEN LTD)

The clinical progress, regulatory updates, and pipeline developments to include:

Troriluzole in SCA: Submitted New Drug Application (NDA) to the U.S. FDA for troriluzole for the treatment of spinocerebellar ataxia type 3 (SCA3), an ultra-rare, genetically-defined, neurodegenerative disease associated with progressive disability, frequent falls, loss of ambulation, speech and swallowing impairment, and premature death that is the most common SCA genotype worldwide.
NDA supported by consistent treatment benefits observed in patients with genotype SCA3 in Study BHV4157-206 across multiple outcome measures including the change from baseline f-SARA at Week 48, CGI-I total score at Week 48, and a robust reduction in fall risk over the study period. A rigorous analysis by genotype was possible as patients were randomized by genotype strata at baseline prior to randomization in the pivotal Phase 3 48-week, double-blind, placebo-controlled phase of Study BHV4157-206.
SCA3 represented 41% of study participants, consistent with being the most common subtype. SCA3 affects approximately 10,600 people in North America and in the EU and Japan.
NDA further supported by a composite scale development (SCACOMS) and analysis of Study BHV4157-206, and confirmatory evidence of efficacy provided by data from the 3-year, long-term open-label (OLE) extension phase of two studies (BHV4157-206 and BHV4157-201) using a Matching Adjusted Indirect Comparison (MAIC) to an external control group.
Biohaven has previously received Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA.
Kv7 Platform: Highlighted progress and broad potential of Kv7 platform, including ongoing and planned development of BHV-7000 (Kv7.2/3 activator):
Phase 1 EEG study with BHV-7000 in 1H2023.
Pivotal studies with BHV-7000 in focal epilepsy and bipolar disorder planned to initiate in 2H2023.
Burgeoning evidence for therapeutic benefits of targeting Kv7 in diverse, high unmet need indications.
Bispecific Platform: Provided updates regarding planned INDs for targeted extracellular protein degradation franchise (including IgG, IgA, and autoantibody-specific degrader programs) and next-generation antibody-drug conjugate (ADC) technologies;
IND application for novel IgG degrader, BHV-1300, on track for submission in 2023.
TYK2/JAK1 Inhibition in Immune-Mediated Brain Disorders: Began dosing with BHV-8000 (an oral, brain-penetrant, dual TYK2/JAK1 inhibitor) in a ­­­Phase 1 study in normal healthy volunteers.

Taldefgrobep Alfa:
In Spinal Muscular Atrophy: Enrollment of approximately 225 patients in global Phase 3 trial now anticipated to complete in 2023.
In Metabolic Disorders: Planned Phase 2 trial initiation in 2H2023.
Vlad Coric M.D., CEO and Chairman of Biohaven, commented, "Today's R&D Day review of our robust pipeline highlights Biohaven's continued dedication to advance novel therapeutics for brain disorders and builds upon our team's successes with the prior approvals of Nurtec® ODT and Zavzpret™ that have changed the treatment paradigm in migraine."

"The NDA we submitted for troriluzole for SCA3 represents approximately seven years of effort by the Biohaven team to bring forward a potentially new treatment for this ultra-rare disease. Advancing a new investigational drug for a rare disease that has no current therapy is a multi-year process that is the culmination of not only our own internal efforts but also close coordination with patient advocacy groups including the National Ataxia Foundation (NAF), leading academic researchers and regulatory agencies. Rare brain diseases are particularly challenging to research given relatively small populations of patients to run / participate in clinical trials, the need to rely on real-world data and often a lack of standardized ratings scales or biomarkers. We could not have advanced the SCA program this far without the leadership from the National Ataxia Foundation that has supported basic science research as well as the development of natural history cohorts in SCA that serve as an external control for longitudinal studies. The Biohaven clinical trials in SCA were a first of its kind in this area and utilized a newly developed rating scale (the functional SARA or f-SARA) that was developed in close consultation with the FDA using standard regulatory pathways to elucidate this new scale. We look forward to interactions with the US FDA, EMA and other regulatory agencies across the globe as our submissions advance in the review process," Dr. Coric added.

"We are pleased that our decades-long investment in ataxia research and understanding of disease progression has accelerated treatment development for SCA," said Andrew Rosen, Executive Director of the National Ataxia Foundation. "Thank you to all NAF members who participated in these important trials."

Bruce Car PhD, Biohaven's CSO, stated, "Given the lack of efficacious therapies for many brain disorders, we must urgently evaluate new mechanisms and approaches to change the treatment paradigm in this therapeutic area. Our R&D Day today demonstrates the commitment that we have given to this effort with exciting new approaches ranging from our submission of a glutamate modulator in SCA to immune modulation using small molecule degraders to ion channels targeting agents for epilepsy and mood disorders, as well as a biologic treatment to enhance muscle growth in SMA. Our scientific and clinical team is poised to file multiple new INDs from our deep pipeline and complete late-stage clinical trials in the next couple of years. It is an exciting time at Biohaven for our passionate employees, patients and investors as we advance these novel investigational drugs in our pipeline."

The Research & Development Day presentations will be made available following the conclusion of the program on https://ir.biohaven.com/events-presentations/events.

About Biohaven
Biohaven is a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including rare disorders. Biohaven's experienced management team brings with it a track record of delivering new drug approvals for products for diseases such as migraine, depression, bipolar disorder and schizophrenia. The company is advancing a pipeline of therapies for diseases with little or no treatment options, leveraging its proven drug development capabilities and proprietary platforms, including Kv7 ion channel modulation for epilepsy and neuronal hyperexcitability, glutamate modulation for obsessive-compulsive disorder and spinocerebellar ataxia, myostatin inhibition for neuromuscular diseases, and brain-penetrant TYK2/JAK1 inhibition for immune-mediated brain disorders. Biohaven's portfolio of early- and late-stage product candidates also includes discovery research programs focused on TRPM3 channel activation for neuropathic pain, CD-38 antibody recruiting, bispecific molecules for multiple myeloma, antibody drug conjugates (ADCs), and targeted extracellular protein degrader platform technology (MoDE™) with potential application in neurological disorders, cancer, and autoimmune diseases. For more information, visit www.biohaven.com.

Forward-looking Statements
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the Food and Drug Administration; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven's product candidates; the potential for Biohaven's product candidates to be first in class therapies; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this new release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

MoDEs is a trademark of Biohaven Therapeutics Ltd.

Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
+1 (201) 248-0741

Media Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
+1 (312) 961-2502

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SOURCE Biohaven Ltd.


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Long I love the move after the offering, Looking like it's being done correctly. Expect another pop when announced offering is over.

Biohaven Shares Hit 52-Week High After Public Offering Prices
10:40 am ET October 21, 2022 (Dow Jones) Print

By Chris Wack


Biohaven Ltd. shares were up 17% to $13.98 Friday after the biopharmaceutical company said it priced a public offering of 25 million common shares at $10.50 each.

The stock hit a 52-week high of $15.33 earlier in the session.

The company has granted the underwriters a 30-day option to buy up to an additional 3.75 million shares.

The offering is expected to close on Tuesday, subject to the satisfaction of customary closing conditions.

Biohaven said it intends to use the proceeds received from the offering for general corporate purposes.

Back in here $13.24 options look good also