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 Updates
Corporate 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; and
Taldefgrobep 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 alfa
Novel 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 ...
