On March 2, 2026 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, 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.
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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 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 (Free ESMO Whitepaper) 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 2026
BHV-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 2026
Capital 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 Highlights
R&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.
(Press release, Biohaven Pharmaceutical, MAR 2, 2026, View Source [SID1234663191])