Biohaven Ltd.

Previous wording

Our actual results and timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, among other things, may differ materially from the forward-looking statements contained in this Quarterly Report on Form 10-Q. Statements made herein are as of the date of the filing of this Form 10-Q with the SEC and should not be relied upon as of any subsequent date. Even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this Quarterly Report on Form 10-Q, they may not be predictive of results or developments in future periods. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Current wording

Our actual results and timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, among other things, may differ materially from the forward-looking statements contained in this Quarterly Report on Form 10-Q. Statements made herein are as of the date of the filing of this Quarterly Report on Form 10-Q with the SEC and should not be relied upon as of any subsequent date. Even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this Quarterly Report on Form 10-Q, they may not be predictive of results or developments in future periods. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Previous wording

In the fourth quarter of 2025, we initiated strategic portfolio and cost-optimization measures to prioritize three key, late-stage, clinical programs that we believe have the greatest potential for value generation. These key clinical programs include Kv7 ion channel modulation for epilepsy; Molecular Degrader of Extracellular Proteins (“MoDE”) and Targeted Removal of Aberrant Protein ("TRAP") extracellular protein degradation for immunological diseases; and myostatin-activin pathway targeting agent for neuromuscular and metabolic diseases, including obesity (collectively, the "key programs").

Current wording

In the fourth quarter of 2025, we initiated strategic portfolio and cost-optimization measures to prioritize three key, late-stage, clinical programs that we believe have the greatest potential for value generation. These key clinical programs include Kv7 ion channel modulation for epilepsy; Molecular Degrader of Extracellular Proteins (“MoDE”) and Targeted Removal of Aberrant Protein ("TRAP") extracellular protein degradation for immunological diseases; and a myostatin-activin pathway targeting agent for neuromuscular and metabolic diseases, including obesity (collectively, the "key programs").

Previous wording

Biohaven MoDE and TRAP 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. The first extracellular protein degraders in the clinic, three MoDE and TRAP degraders have now been dosed in Phase 1 trials.

Current wording

Biohaven MoDE and TRAP 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. Lead Extracellular Degrader, BHV-1300 MoDE has entered Phase 3, with BHV-1400 lead TRAP planned to enter Phase 3 in the second half of 2026.

Previous wording

BHV-1400, Biohaven's first TRAP molecule, is designed to specifically target the pathogenic driver of IgA nephropathy ("IgAN"), galactose deficient IgA1 ("Gd-IgA1") without suppressing the healthy immune system. BHV-1400 has been dosed in a clinically concluded Phase 1 study in normal healthy volunteers and continues to be dosed in an expansion cohort of IgAN patients with plans to initiate the pivotal study in IgAN patients in the second half of 2026. Data from the first, and lowest, dose cohort and each subsequent cohort thereafter of BHV-1400 demonstrated clear differentiation from competitors in the IgA nephropathy space, with deep, rapid lowering of Gd-IgA1 within hours and preservation of host immunoglobulins ("Ig") including IgG, IgA, IgE, and IgM. These results have now been re-capitulated in the first IgAN patients dosed, with improvements noted in hematuria, proteinuria, and eGFR (as defined below) within the first month of dosing.

Current wording

BHV-1400, Biohaven's first TRAP molecule, is designed to specifically target the pathogenic driver of IgA nephropathy ("IgAN"), galactose deficient IgA1 ("Gd-IgA1") without suppressing the healthy immune system. BHV-1400 has been dosed in a clinically concluded Phase 1 study in normal healthy volunteers and continues to be dosed in an expansion cohort of IgAN patients with plans to initiate the pivotal study in IgAN patients in the second half of 2026. Data from the first, and lowest, dose cohort and each subsequent cohort thereafter of BHV-1400 demonstrated clear differentiation from competitors in the IgA nephropathy space, with deep, rapid lowering of Gd-IgA1 within hours and preservation of host immunoglobulins ("Ig") including IgG, IgA, IgE, and IgM. These results have now been recapitulated in the first IgAN patients dosed, with improvements noted in hematuria, proteinuria, and eGFR (as defined below) within the first month of dosing.

Previous wording

In the preliminary data reported, BHV-1300 was safe and well-tolerated in subcutaneous doses up to 2000 mg with no clinically significant increases in ALT, AST, or bilirubin, no clinically significant reductions in albumin, and no clinically significant increases in cholesterol over the four-week dosing period compared to placebo. There were no clinically significant reductions in IgG3, IgA, IgE, or IgM compared to baseline. Most AEs were mild and self-resolving, and there were no serious or severe AEs. A Phase 1b study was initiated to evaluate the effect in participants with Graves' disease.

Current wording

In the preliminary data reported, BHV-1300 was safe and well-tolerated in subcutaneous doses up to 2000 mg with no clinically significant increases in ALT, AST, or bilirubin, no clinically significant reductions in albumin, and no clinically significant increases in cholesterol over the four-week dosing period compared to placebo. There were no clinically significant reductions in IgG3, IgA, IgE, or IgM compared to baseline. Most adverse events ("AEs") were mild and self-resolving, and there were no serious or severe AEs. A Phase 1b study was initiated to evaluate the effect in participants with Graves' disease.

Previous wording

In May 2026, we reported updated data from our ongoing Phase 1b study of BHV-1300 in patients with Graves’ disease. In the study, weekly administration of BHV-1300 1000 mg subcutaneously achieved mean reductions of pathogenic TSHR-IgG1 autoantibodies of greater than 80% by week 12 in patients with Graves’ hyperthyroidism. Among participants with elevated thyroid hormones despite concurrent anti-thyroid drug therapy, normalization of free T4 occurred at a median of 3 weeks and normalization of free T3 occurred at a median of 5 weeks after the first administration of BHV-1300. Based on current data, BHV-1300 has been safe and well-tolerated through 12 weeks of dosing, with most adverse events ("AEs") mild and self-resolving, no serious adverse events (" SAEs"), no clinically significant increases in cholesterol or ALT/AST/bilirubin, no clinically significant reductions in albumin, and no clinically significant reductions in IgG3, IgA, IgE, or IgM relative to baseline. Based upon these Phase 1b results, we have initiated a pivotal trial of BHV-1300 in Graves’ disease and expect to pursue additional follow-on studies in other autoimmune diseases. The study is a randomized, double-blind, placebo-controlled study in approximately 300 adults with Graves’ hyperthyroidism evaluating normalization of T3, T4, and TSH at 26 weeks absent an antithyroid drug.

Current wording

In May 2026, we reported updated data from our ongoing Phase 1b study of BHV-1300 in patients with Graves’ disease. In the study, weekly administration of BHV-1300 1000 mg subcutaneously achieved mean reductions of pathogenic TSHR-IgG1 autoantibodies of greater than 80% by week 12 in patients with Graves’ hyperthyroidism. Among participants with elevated thyroid hormones despite concurrent anti-thyroid drug therapy, normalization of free T4 occurred at a median of 3 weeks and normalization of free T3 occurred at a median of 5 weeks after the first administration of BHV-1300. Based on current data, BHV-1300 has been safe and well-tolerated through 12 weeks of dosing, with most AEs mild and self-resolving, no serious adverse events ("SAEs"), no clinically significant increases in cholesterol or ALT/AST/bilirubin, no clinically significant reductions in albumin, and no clinically significant reductions in IgG3, IgA, IgE, or IgM relative to baseline. Based upon these Phase 1b results, we have initiated a pivotal trial of BHV-1300 in Graves’ disease and expect to pursue additional follow-on studies in other autoimmune diseases. The study is a randomized, double-blind, placebo-controlled study in approximately 300 adults with Graves’ hyperthyroidism evaluating normalization of T3, T4, and TSH at 26 weeks absent an antithyroid drug.

Previous wording

BHV-1400 has been safe and well-tolerated across the ongoing Phase 1 study. Most AEs were mild and self-resolving, there were no discontinuations due to study drug AEs, and there were no serious or severe study drug AEs. 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 4-week dosing period. There were no clinically significant reductions in other immunoglobulins including IgG, IgA, IgE, or IgM relative to baseline. Based upon the rapid and deep reductions of Gd-IgA1 observed with subcutaneous ("SC") BHV-1400, we have expanded our Phase 1 study of BHV-1400 in patients with IgAN, and ultimately plan to initiate a pivotal trial using urine protein-creatinine ratio ("UPCR") as a surrogate endpoint for accelerated approval.

Current wording

BHV-1400 has been safe and well-tolerated across the ongoing Phase 1 study. Most AEs were mild and self-resolving, there were no discontinuations due to study drug AEs, and there were no serious or severe study drug AEs. 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 3-week dosing period. There were no clinically significant reductions in other immunoglobulins including IgG, IgA, IgE, or IgM relative to baseline. Based upon the rapid and deep reductions of Gd-IgA1 observed with subcutaneous ("SC") BHV-1400, we have expanded our Phase 1 program of BHV-1400 to include patients with IgAN, and ultimately plan to initiate a pivotal trial using urine protein-creatinine ratio ("UPCR") as a primary endpoint for traditional approval.

Previous wording

In April 2022, we closed the acquisition from Knopp of Channel, a wholly owned subsidiary of Knopp owning the assets of Knopp’s Kv7 channel targeting platform, pursuant to the Purchase Agreement. The acquisition of the Kv7 channel targeting platform added the latest advances in ion-channel modulation to our neuroscience portfolio. Opakalim (formerly known as KB-3061 and also referred to as BHV-7000), the lead asset from the Kv7 platform is an activator of Kv7.2/Kv7.3, a key ion channel involved in neuronal signaling and in regulating the hyperexcitable state in epilepsy.

Current wording

In April 2022, we closed the acquisition from Knopp Biosciences LLC (“Knopp”) of Channel Biosciences, LLC ("Channel"), a wholly owned subsidiary of Knopp owning the assets of Knopp’s Kv7 channel targeting platform, pursuant to the Purchase Agreement. The acquisition of the Kv7 channel targeting platform added the latest advances in ion-channel modulation to our neuroscience portfolio. Opakalim (formerly known as KB-3061 and also referred to as BHV-7000), the lead asset from the Kv7 platform is an activator of Kv7.2/Kv7.3, a key ion channel involved in neuronal signaling and in regulating the hyperexcitable state in epilepsy.

Previous wording

In May 2026, we subsequently reported updated data showing that 54% of participants had a ≥50%RR over any consecutive 6-month period in the OLE study (n>100). These results are comparable to the ≥50%RR published for other investigational agents in the class such azetukalner (which has reported 56% of patients with a ≥50%RR over any consecutive 6-month period from its Phase 2b OLE data). Opakalim was well-tolerated in the OLE study with a low incidence of CNS adverse events, consistent with prior studies with opakalim (see figure below).

Current wording

In May 2026, we reported updated data showing that 54% of participants had a ≥50%RR over any consecutive 6-month period in the OLE study (n>100). These results are comparable to the ≥50%RR published for other investigational agents in the class such as azetukalner (which has reported 56% of patients with a ≥50%RR over any consecutive 6-month period from its Phase 2b OLE data). Opakalim was well-tolerated in the OLE study with a low incidence of CNS adverse events, consistent with prior studies with opakalim (see figure below).

Previous wording

In February 2022, we announced a worldwide license agreement with BMS for the development and commercialization rights to taldefgrobep alfa (also known as BMS-986089 and now referred to as BHV-2000), a novel, Phase 3-ready anti-myostatin adnectin. Myostatin is a natural protein that limits skeletal muscle growth, an important process in healthy muscular development that can lead to improvements of lean mass and loss of adipose tissue by acting through the activin receptor type-2B ("ActRIIb"). In patients with neuromuscular diseases, active myostatin can critically limit the growth needed to achieve developmental and functional milestones. Myostatin inhibition is a promising therapeutic strategy for enhancing muscle mass and strength in a range of pediatric and adult neuromuscular conditions. In addition, preclinical and early clinical data suggest that blocking myostatin and downstream signaling through its receptors on skeletal muscle may produce physical and metabolic changes that are important to individuals living with overweight and obesity, including reducing body fat and improving insulin sensitivity while increasing lean muscle mass. Taldefgrobep’s novel mode of action inhibiting both myostatin directly and through the ActRIIb and its unique impact on body composition suggest it could be used as monotherapy or in combination with other anti-obesity medications.

Current wording

In February 2022, we announced a worldwide license agreement with Bristol Myers Squibb ("BMS") for the development and commercialization rights to taldefgrobep alfa (also known as BMS-986089 and now referred to as BHV-2000), a novel, Phase 3-ready anti-myostatin adnectin. Myostatin is a natural protein that limits skeletal muscle growth, an important process in healthy muscular development that can lead to improvements of lean mass and loss of adipose tissue by acting through the activin receptor type-2B ("ActRIIb"). In patients with neuromuscular diseases, active myostatin can critically limit the growth needed to achieve developmental and functional milestones. Myostatin inhibition is a promising therapeutic strategy for enhancing muscle mass and strength in a range of pediatric and adult neuromuscular conditions. In addition, preclinical and early clinical data suggest that blocking myostatin and downstream signaling through its receptors on skeletal muscle may produce physical and metabolic changes that are important to individuals living with overweight and obesity, including reducing body fat and improving insulin sensitivity while increasing lean muscle mass. Taldefgrobep’s novel mode of action inhibiting both myostatin directly and through the ActRIIb and its unique impact on body composition suggest it could be used as monotherapy or in combination with other anti-obesity medications.

Previous wording

Based on non-clinical and clinical data, Biohaven initiated a Phase 2 study of taldefgrobep in the management of obesity in the fourth quarter of 2025. In March 2026, we announced that enrollment in the study was complete. Topline results for the Phase 2 proof-of-concept study are expected in the second half of 2026. The study will evaluate the ability of taldefgrobep to reduce fat mass and total body weight while increasing lean muscle mass. The study is a placebo-controlled study evaluating two dosing schedules of taldefgrobep versus placebo. Approximately 150 participants will be randomized to receive taldefgrobep or matching placebo over a 24-week double-blind treatment period followed by an additional 24 weeks of OLE during which all participants will receive taldefgrobep. Key endpoints include the change in total body weight, lean mass, fat mass, and metabolic parameters, along with a comprehensive assessment of safety. See below for trial design.

Current wording

Based on non-clinical and clinical data, Biohaven initiated a Phase 2 study of taldefgrobep in the management of obesity in the fourth quarter of 2025. In March 2026, we announced that enrollment in the study was complete. The study is evaluating the ability of taldefgrobep to reduce fat mass and total body weight while increasing lean muscle mass. As originally designed, the study is a placebo-controlled study evaluating two dosing schedules of taldefgrobep versus placebo. Approximately 150 participants were randomized to receive taldefgrobep or matching placebo over a 24-week double-blind treatment period followed by an additional 24 weeks of OLE during which all participants will receive taldefgrobep. Key endpoints include the change in total body weight, lean mass, fat mass, and metabolic parameters, along with a comprehensive assessment of safety. See below for trial design.

Previous wording

◦BHV-1530: In July 2026, Biohaven announced plans to initiate combination cohorts evaluating BHV-1530 with monoclonal antibody Libtayo® (cemiplimab-rwlc), in the second half of 2026. In connection with these plans, we have entered into a clinical supply agreement with Regeneron Pharmaceuticals, Inc. ("Regeneron") under which we will sponsor and fund the planned combination clinical trial, and Regeneron will provide Libtayo. BHV-1530 is currently being studied in an ongoing Phase 1 dose-escalation trial in unselected patients with advanced urothelial cancer, head and neck squamous cell carcinoma, and non-small cell lung cancer who have failed standard-of-care therapy, as well as other tumor types harboring FGFR3 genomic alterations. Up to approximately 140 subjects are planned to be evaluated.

Current wording

◦BHV-1530: In July 2026, Biohaven announced plans to initiate combination cohorts evaluating BHV-1530 with monoclonal antibody Libtayo® (cemiplimab-rwlc), which began enrolling in the third quarter of 2026. In connection with these plans, we entered into a clinical supply agreement with Regeneron Pharmaceuticals, Inc. ("Regeneron") under which we will sponsor and fund the combination clinical trial, and Regeneron will provide Libtayo. BHV-1530 is currently being studied in an ongoing Phase 1 dose-escalation trial in unselected patients with advanced urothelial cancer, head and neck squamous cell carcinoma, and non-small cell lung cancer who have failed standard-of-care therapy, as well as other tumor types harboring FGFR3 genomic alterations. Up to approximately 140 subjects are planned to be evaluated.

Previous wording

◦BHV-8100 phase 1 study initiation: In the second quarter of 2026, we initiated FIH dosing of BHV-8100. The Phase 1 study is a SAD study in healthy participants designed to evaluate the safety, tolerability, and pharmacokinetics of BHV-8100. Each cohort is planned to enroll approximately 8 subjects total (6 subjects receive active drug and 2 subjects receive placebo). Dose escalation is ongoing. Preliminary data from the study demonstrate a pharmacokinetic profile consistent with once-daily oral dosing and a well-tolerated profile at projected therapeutic exposures, with most adverse events mild and spontaneously resolving. BHV-8100 is an orally administered, brain-penetrant activator of the M2 isoform of pyruvate kinase (“PKM2”), a novel therapeutic class designed to address the bioenergetic and immunometabolic basis of systemic, CNS and retinal disorders. PKM2 is the final, rate-limiting enzyme in glycolysis, converting phosphoenolpyruvate to pyruvate, and serves as a master metabolic regulator in energy-intensive cells and tissues, including the brain, retina, and immune system. BHV-8100 stabilizes the more highly active, tetrameric form of PKM2. The accumulation of the less active dimeric form leads to bioenergetic deficits and drives disease pathogenesis in multiple inflammatory and neurological disorders. Through stabilizing the PKM2 tetramer, BHV-8100 restores glycolytic flux and potentially metabolic deficits. Potential target indications include neurodegenerative diseases such as Alzheimer’s disease, multiple sclerosis and Parkinson's disease, ophthalmological conditions such as adult-onset macular degeneration, retinitis pigmentosa, and immunological disorders such as atopic dermatitis.

Current wording

◦BHV-8100 Phase 1 study initiation: In the second quarter of 2026, we initiated FIH dosing of BHV-8100. The Phase 1 study is a SAD study in healthy participants designed to evaluate the safety, tolerability, and pharmacokinetics of BHV-8100. Each cohort is planned to enroll approximately 8 subjects total (6 subjects receive active drug and 2 subjects receive placebo). Dose escalation is ongoing. Preliminary data from the study demonstrate a pharmacokinetic profile consistent with once-daily oral dosing and a well-tolerated profile with most adverse events mild and spontaneously resolving. BHV-8100 is an orally administered, brain-penetrant activator of the M2 isoform of pyruvate kinase (“PKM2”), a novel therapeutic class designed to address the bioenergetic and immunometabolic basis of systemic, CNS and retinal disorders. PKM2 is the final, rate-limiting enzyme in glycolysis, converting phosphoenolpyruvate to pyruvate, and serves as a master metabolic regulator in energy-intensive cells and tissues, including the brain, retina, and immune system. BHV-8100 stabilizes the more highly enzymatically active, tetrameric form of PKM2. The accumulation of the less active dimeric form leads to bioenergetic deficits and drives disease pathogenesis in multiple inflammatory and neurological disorders. Through stabilizing the PKM2 tetramer, BHV-8100 restores glycolytic flux and potentially reverses metabolic deficits. Potential target indications include neurodegenerative diseases such as Alzheimer’s disease, multiple sclerosis and Parkinson's disease, ophthalmological conditions such as adult-onset macular degeneration, retinitis pigmentosa, and immunological disorders such as atopic dermatitis.

Previous wording

Other (expense) income, net primarily consists of changes in the fair value of our forward contract and derivative liabilities, net investment income, and the changes in fair value of our note payable liability under the Note Purchase Agreement.

Current wording

Other (expense) income, net primarily consists of changes in the fair value of our forward contract and derivative liabilities, net investment income, and the changes in fair value of our note payable liability under the Note Purchase Agreement (the "Note Purchase Agreement" or "NPA") entered into by the Company with Beetlejuice SA LLC, an affiliate of Oberland Capital Management LLC ("Oberland").

Previous wording

Prior to settlement, the fair value of the forward contracts and derivative liabilities recognized in connection with the Knopp Amendment was determined using a Monte Carlo simulation of the Company's stock price over the respective duration and terms of each instrument being valued. Refer to Note 4, "Fair Value of Financial Assets and Liabilities," in our audited consolidated financial statements included in the 2025 Form 10-K for detail on valuation inputs and methodology. The fair value of these liabilities were recorded on the condensed consolidated balance sheets with changes in fair value recorded in other (expense) income, net in the condensed consolidated statements of operations and comprehensive loss.

Current wording

Prior to settlement, the fair value of the forward contracts and derivative liabilities recognized in connection with the amendment to the Purchase Agreement with Knopp (the "Knopp Amendment"), was determined using a Monte Carlo simulation of the Company's stock price over the respective duration and terms of each instrument being valued. Refer to Note 4, "Fair Value of Financial Assets and Liabilities," in our audited consolidated financial statements included in the 2025 Form 10-K for detail on valuation inputs and methodology. The fair value of these liabilities were recorded on the condensed consolidated balance sheets with changes in fair value recorded in other (expense) income, net in the condensed consolidated statements of operations and comprehensive loss.

Previous wording

We have historically outsourced all of the research and clinical development for our programs under a master services agreement with our subsidiaries, Biohaven Pharmaceuticals, Inc. ("BPI") and Biohaven Biosciences Ireland Limited (“BBIL”). Under these arrangements, both companies were profitable during the three and six months ended June 30, 2026 and 2025. BPI and BBIL are subject to taxation in the United States and Ireland, respectively. As such, in each reporting period, the tax provision includes the effects of the results of profitable operations of BPI and BBIL.

Current wording

We have historically outsourced all of the research and clinical development for our programs under a master services agreement with our subsidiaries, Biohaven Pharmaceuticals, Inc. ("BPI") and Biohaven Bioscience Ireland Limited (“BBIL”). Under these arrangements, both companies were profitable during the three and nine months ended September 30, 2026 and 2025. BPI and BBIL are subject to taxation in the United States and Ireland, respectively. As such, in each reporting period, the tax provision includes the effects of the results of profitable operations of BPI and BBIL.

Previous wording

At June 30, 2026 and December 31, 2025, we continued to maintain a full valuation allowance against our net deferred tax assets, comprised primarily of research and development tax credit carryforwards and net operating loss carryforwards, based on management’s assessment that it is more likely than not that the deferred tax assets will not be realized.

Current wording

At September 30, 2026 and December 31, 2025, we continued to maintain a full valuation allowance against our net deferred tax assets, comprised primarily of research and development tax credit carryforwards and net operating loss carryforwards, based on management’s assessment that it is more likely than not that the deferred tax assets will not be realized. We will continue to evaluate the need for a valuation allowance on our deferred tax assets until there is sufficient positive evidence to support the reversal of all or some portion of these allowances. We anticipate the potential payments from the SKBP License Agreement could result in future earnings and believe sufficient positive evidence may become available to allow us to reach a conclusion that a significant portion of the valuation allowance on our Irish deferred tax assets will no longer be needed. Release of the valuation allowance would result in the recognition of certain deferred tax assets and a decrease to income tax expense for the period the release is recorded. However, the exact timing and amount of the valuation allowance release are subject to change on the basis of the level of profitability that we are able to actually achieve.

Previous wording

R&D expenses, including non-cash share-based compensation costs, were $100.8 million for the three months ended June 30, 2026, compared to $184.4 million for the three months ended June 30, 2025. The decrease of $83.6 million was primarily due to decreases in direct program spend and preclinical spend in 2026 as compared to the same period in the prior year. The decrease in direct program spend was largely due to our strategic reprioritization of programs which was implemented in the fourth quarter of 2025, as well as one-time developmental milestones recorded during the three months ended June 30, 2025 of $15.0 million and $10.0 million for our BHV-8000 and BHV-1530 programs, respectively.

Current wording

R&D expenses, including non-cash share-based compensation costs, were $101.9 million for the three months ended September 30, 2026, compared to $141.2 million for the three months ended September 30, 2025. The decrease of $39.3 million was primarily due to decreases in direct program spend and preclinical spend in 2026 as compared to the same period in the prior year. The decrease in direct program spend was largely due to our strategic reprioritization of programs which was implemented in the fourth quarter of 2025.

Previous wording

General and administrative expenses were $24.1 million for the three months ended June 30, 2026, compared to $27.3 million for the three months ended June 30, 2025. The decrease of $3.2 million was primarily due to decreased legal costs and employee costs, including non-cash share-based compensation expense. Non-cash share-based compensation expense was $7.2 million for the three months ended June 30, 2026, a decrease of $0.5 million as compared to the same period in 2025.

Current wording

General and administrative expenses were $24.6 million for the three months ended September 30, 2026, compared to $28.2 million for the three months ended September 30, 2025. The decrease of $3.6 million was primarily due to decreased employee costs, including non-cash share-based compensation expense. Non-cash share-based compensation expense was $6.6 million for the three months ended September 30, 2026, a decrease of $1.0 million as compared to the same period in 2025.

Previous wording

Other (expense) income, net was other expense of $12.0 million for the three months ended June 30, 2026, compared to other income of $13.8 million for the three months ended June 30, 2025. The decrease of $25.8 million was primarily due to increased non-cash losses related to changes in fair value of our notes payable liability under the NPA during the three months ended June 30, 2026, and gains recorded for the non-cash changes in fair value of our forward contracts and derivative liabilities recorded in connection with the Knopp Amendment during the three months ended June 30, 2025. See Note 6, "Notes Payable," to the accompanying condensed consolidated financial statements included in this Form 10-Q for discussion of the NPA and Note 11, "License, Acquisitions and Other Agreements,"for discussion of the forward contract and derivative liabilities recorded in connection with the Knopp Amendment.

Current wording

Other expense, net was $2.9 million for the three months ended September 30, 2026, compared to $3.8 million for the three months ended September 30, 2025. The decrease in expense of $0.9 million was primarily due to decreased non-cash losses related to changes in fair value of our notes payable liability under the NPA during the three months ended September 30, 2026, which were partially offset by gains recorded for the non-cash changes in fair value of our forward contracts and derivative liabilities recorded in connection with the Knopp Amendment during the three months ended September 30, 2025, and decreased investment income. See Note 6, "Notes Payable," to the accompanying condensed consolidated financial statements included in this Form 10-Q for discussion of the NPA and Note 11, "License, Acquisitions and Other Agreements,"for discussion of the forward contract and derivative liabilities recorded in connection with the Knopp Amendment.

Previous wording

The following tables summarize our results of operations for the six months ended June 30, 2026 and 2025:

Current wording

The following tables summarize our results of operations for the nine months ended September 30, 2026 and 2025:

Previous wording

R&D expenses, including non-cash share-based compensation costs, were $204.6 million for the six months ended June 30, 2026, compared to $372.0 million for the six months ended June 30, 2025. The decrease of $167.3 million was primarily due to decreases in direct program spend and preclinical spend, and non-cash share-based compensation expense in 2026 as compared to the same period in the prior year. The decrease in direct program and preclinical spend was largely due to our strategic reprioritization of programs which was implemented in the fourth quarter of 2025, as well as one-time developmental milestones recorded during the six months ended June 30, 2025 of $15.0 million and $10.0 million for our BHV-8000 and BHV-1530 programs, respectively. The $25.7 million decrease in preclinical research programs was also due to an upfront share payment valued at $4.9 million and an accrual for an upfront cash payment of $5.0 million related to agreements entered into during the six months ended June 30, 2025.

Current wording

R&D expenses, including non-cash share-based compensation costs, were $306.5 million for the nine months ended September 30, 2026, compared to $513.1 million for the nine months ended September 30, 2025. The decrease of $206.6 million was primarily due to decreases in direct program spend, preclinical spend, and non-cash share-based compensation expense in 2026 as compared to the same period in the prior year. The decrease in direct program and preclinical spend was largely due to our strategic reprioritization of programs which was implemented in the fourth quarter of 2025, as well as one-time developmental milestones recorded during the nine months ended September 30, 2025 of $15.0 million and $10.0 million for our BHV-8000 and BHV-1530 programs, respectively. The $30.1 million decrease in preclinical research programs was also due to an upfront share payment valued at $4.9 million and an accrual for an upfront cash payment of $5.0 million related to agreements entered into during the nine months ended September 30, 2025.

Previous wording

Non-cash share-based compensation expense was $30.5 million for the six months ended June 30, 2026, a decrease of $17.9 million as compared to the same period in 2025. Non-cash share-based compensation expense was lower in 2026 primarily due to our annual equity incentive awards granted in the first quarter of 2026, which had a lower grant date fair value per share than the annual awards granted in the first quarter of 2025.

Current wording

Non-cash share-based compensation expense was $42.5 million for the nine months ended September 30, 2026, a decrease of $19.8 million as compared to the same period in 2025. Non-cash share-based compensation expense was lower in 2026 primarily due to our annual equity incentive awards granted in the first quarter of 2026, which had a lower grant date fair value per share than the annual awards granted in the first quarter of 2025.

Previous wording

General and administrative expenses were $50.7 million for the six months ended June 30, 2026, compared to $61.3 million for the six months ended June 30, 2025. The decrease of $10.6 million was primarily due to decreased non-cash share-based compensation expense and decreased legal costs during the six months ended June 30, 2026. Non-cash share-based compensation expense was $17.0 million for the six months ended June 30, 2026, a decrease of $8.5 million as compared to the same period in 2025. Non-cash share-based compensation expense was lower in 2026 primarily due to our annual equity incentive awards granted in the first quarter of 2026, which had a lower grant date fair value per share than the annual awards granted in the first quarter of 2025.

Current wording

General and administrative expenses were $75.3 million for the nine months ended September 30, 2026, compared to $89.5 million for the nine months ended September 30, 2025. The decrease of $14.3 million was primarily due to decreased employee costs, including non-cash share-based compensation expense, and decreased legal costs during the nine months ended September 30, 2026. Non-cash share-based compensation expense was $23.6 million for the nine months ended September 30, 2026, a decrease of $9.6 million as compared to the same period in 2025. Non-cash share-based compensation expense was lower in 2026 primarily due to our annual equity incentive awards granted in the first quarter of 2026, which had a lower grant date fair value per share than the annual awards granted in the first quarter of 2025.

Previous wording

Other (expense) income, net was other expense of $11.9 million for the six months ended June 30, 2026, compared to other income of $14.3 million for the six months ended June 30, 2025. The decrease of $26.2 million was primarily due to increased non-cash losses related to changes in fair value of our notes payable liability under the NPA during the six months ended June 30, 2026, gains recorded for the non-cash changes in fair value of our forward contracts and derivative liabilities recorded in connection with the Knopp Amendment during the six months ended June 30, 2025, and decreased investment income. See Note 6, "Notes Payable," to the accompanying condensed consolidated financial statements included in this Form 10-Q for discussion of the NPA and Note 11, "License, Acquisitions and Other Agreements,"for discussion of the forward contract and derivative liabilities recorded in connection with the Knopp Amendment.

Current wording

Other (expense) income, net was other expense of $14.8 million for the nine months ended September 30, 2026, compared to other income of $10.5 million for the nine months ended September 30, 2025. The decrease of $25.2 million was primarily due to increased non-cash losses related to changes in fair value of our notes payable liability under the NPA during the nine months ended September 30, 2026, gains recorded for the non-cash changes in fair value of our forward contracts and derivative liabilities recorded in connection with the Knopp Amendment during the nine months ended September 30, 2025, and decreased investment income. See Note 6, "Notes Payable," to the accompanying condensed consolidated financial statements included in this Form 10-Q for discussion of the NPA and Note 11, "License, Acquisitions and Other Agreements,"for discussion of the forward contract and derivative liabilities recorded in connection with the Knopp Amendment.

Previous wording

We recorded income tax provisions of $0.4 million and $0.3 million for the three months ended June 30, 2026 and 2025, respectively.

Current wording

We recorded income tax provisions of $0.6 million and $1.1 million for the nine months ended September 30, 2026 and 2025, respectively.

Previous wording

Since our inception, we have not generated any revenue and have incurred significant operating losses and negative cash flows from operations. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our product candidates. We expect to continue to incur significant expenses for at least the next several years as we advance our product candidates from discovery through preclinical development and clinical trials and seek regulatory approval and pursue commercialization of any approved product candidate. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to product manufacturing, marketing, sales and distribution, regulatory and commercial milestones and royalty payments. In addition, we may incur expenses in connection with the in-license or acquisition of additional product candidates.

Current wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from operations. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our product candidates. We may recognize license revenue in future periods under the SKBP License Agreement and the Ono License Agreement. The timing of revenue from upfront payments depends on the closing of the SKBP License Agreement, and revenue from milestones and royalty payments depends on the achievement of the related milestones, regulatory approval of the licensed product candidates and, in the case of royalties, their commercial success. We expect to continue to incur significant expenses for at least the next several years as we advance our product candidates from discovery through preclinical development and clinical trials and seek regulatory approval and pursue commercialization of any approved product candidate. In addition, if we obtain marketing approval for any of our product candidates that we commercialize ourselves, we expect to incur significant commercialization expenses related to product manufacturing, marketing, sales and distribution, regulatory and commercial milestones and royalty payments.

Previous wording

Historically, we have funded our operations primarily with funding from the Former Parent, including a cash contribution received at the Separation, proceeds from the sale of our common shares, and proceeds from the sale of senior secured notes under our Note Purchase Agreement. We have incurred recurring losses since our inception and expect to continue to generate operating losses for the foreseeable future.

Current wording

Historically, we have funded our operations primarily with funding from the Former Parent, including a cash contribution received at the Separation, proceeds from the sale of our common shares, and proceeds from the sale of senior secured notes under our Note Purchase Agreement. We have also recently received an upfront payment from Ono and are contracted to receive an upfront payment from SKBP upon the closing of the SKBP License Agreement, as well as additional contingent milestone payments from both. We have incurred recurring losses since our inception and expect to continue to generate operating losses for the foreseeable future.

Previous wording

As of June 30, 2026, we had cash and cash equivalents of $238.0 million and marketable securities of $29.8 million. Cash in excess of immediate requirements is invested in marketable securities and money market funds with a view to liquidity and capital preservation. We continuously assess our working capital needs, capital expenditure requirements, and future investments or acquisitions.

Current wording

As of September 30, 2026, we had cash and cash equivalents of $95.8 million and marketable securities of $65.4 million. In October 2026, we received an $80.0 million upfront payment under the Ono License Agreement. Cash in excess of immediate requirements is invested in marketable securities and money market funds with a view to liquidity and capital preservation. We continuously assess our working capital needs, capital expenditure requirements, and future investments or acquisitions.

Previous wording

Net cash used in operating activities was $235.0 million for the six months ended June 30, 2026 and $333.1 million for the six months ended June 30, 2025. The $98.0 million decrease in net cash used in operating activities for the six months ended June 30, 2026 was primarily due to a decrease in cash payments for direct R&D activities, including a one-time development milestone payment of $10.0 million for BHV-1530 during the six months ended June 30, 2025. This was partially offset by a one-time payment of $42.7 million made to Knopp during the six months ended June 30, 2026 related to the settlement of the 2025 Additional Consideration True-Up, and payment of the 2025 annual employee bonus in the first quarter of 2026, as compared to our 2024 annual bonus being paid in the fourth quarter of 2024.

Current wording

Net cash used in operating activities was $341.9 million for the nine months ended September 30, 2026 and $478.8 million for the nine months ended September 30, 2025. The $136.9 million decrease in net cash used in operating activities for the nine months ended September 30, 2026 was primarily due to a decrease in cash payments for direct R&D activities, including one-time development milestone payments of $15.0 million and $10.0 million for BHV-8000 and BHV-1530, respectively, during the nine months ended September 30, 2025. This was partially offset by a one-time payment of $42.7 million made to Knopp during the nine months ended September 30, 2026 related to the settlement of the 2025 Additional Consideration True-Up under the Knopp Amendment, as described in Note 11, “License, Acquisitions and Other Agreements," and payment of the 2025 annual employee bonus in the first quarter of 2026, as compared to our 2024 annual bonus being paid in the fourth quarter of 2024.

Previous wording

Net cash provided by investing activities was $60.0 million for the six months ended June 30, 2026, compared to net cash provided by investing activities of $149.3 million for the six months ended June 30, 2025. The $89.3 million decrease in net cash provided by investing activities was driven primarily by a decrease in proceeds from maturities of marketable securities, partially offset by a decrease in purchases of marketable securities, during the six months ended June 30, 2026, as compared to the same period in the prior year. See Note 3, "Marketable Securities," to the condensed consolidated financial statements for additional details.

Current wording

Net cash provided by investing activities was $24.7 million for the nine months ended September 30, 2026, compared to net cash provided by investing activities of $314.3 million for the nine months ended September 30, 2025. The $289.6 million decrease in net cash provided by investing activities was driven primarily by a decrease in proceeds from maturities of marketable securities, partially offset by a decrease in purchases of marketable securities, during the nine months ended September 30, 2026, as compared to the same period in the prior year. See Note 3, "Marketable Securities," to the condensed consolidated financial statements for additional details.

Previous wording

Net cash provided by financing activities was $182.8 million for the six months ended June 30, 2026 compared to net cash provided by financing activities of $250.2 million for the six months ended June 30, 2025. The decrease of $67.4 million was primarily driven by a decrease in proceeds from the issuance of notes payable, related to proceeds from our Note Purchase Agreement during the six months ended June 30, 2025. This was partially offset by proceeds from the issuance of common shares during the six months ended June 30, 2026 related to proceeds from the Equity Distribution Agreement.

Current wording

Net cash provided by financing activities was $183.0 million for the nine months ended September 30, 2026 compared to net cash provided by financing activities of $250.3 million for the nine months ended September 30, 2025. The decrease of $67.3 million was primarily driven by a decrease in proceeds from the issuance of notes payable, related to proceeds from our Note Purchase Agreement during the nine months ended September 30, 2025. This was partially offset by proceeds from the issuance of common shares during the nine months ended September 30, 2026 related to proceeds from the Equity Distribution Agreement.

Previous wording

In the event that by the reporting deadline of March 1, 2027, our audited financial statements for the year ended December 31, 2026 or any year thereafter for the term of the agreement, are subject to any qualification, emphasis of matter or statement as to “going concern” or scope of audit, subject to certain exceptions, we would be in breach of our financial statement delivery covenant under the Note Purchase Agreement. In such event, if such requirement was not amended or waived by the Purchasers, the Purchasers could have the right to exercise their remedies under the Note Purchase Agreement, which could include, but not be limited to, declaring an event of default and accelerating payment of outstanding amounts thereunder (which amounted to $250.0 million as of June 30, 2026), plus a required premium as noted above.

Current wording

In the event that by the reporting deadline of March 1, 2027, our audited financial statements for the year ended December 31, 2026, or any year thereafter for the term of the agreement, are subject to any qualification, emphasis of matter or statement as to “going concern” or scope of audit, subject to certain exceptions, we would be in breach of our financial statement delivery covenant under the Note Purchase Agreement. In such event, if such requirement was not amended or waived by the Purchasers, the Purchasers could have the right to exercise their remedies under the Note Purchase Agreement, which could include, but not be limited to, declaring an event of default and accelerating payment of outstanding amounts thereunder (which amounted to $250.0 million as of September 30, 2026), plus a required premium.

Previous wording

In October 2023, we entered into the Equity Distribution Agreement pursuant to which we may offer and sell common shares having an aggregate offering price of up to $150.0 million from time to time through or to the sales agent, acting as our agent or principal. In August 2024, we entered into an amendment to the Equity Distribution Agreement pursuant to which we may offer and sell common shares having an aggregate offering price of up to $450.0 million. In May

Current wording

In October 2023, we entered into the Equity Distribution Agreement pursuant to which we may offer and sell common shares having an aggregate offering price of up to $150.0 million from time to time through or to the sales agent, acting as our agent or principal. In August 2024, we entered into an amendment to the Equity Distribution Agreement pursuant to which we may offer and sell common shares having an aggregate offering price of up to $450.0 million. In May 2026, we entered into a second amendment to the Equity Distribution Agreement pursuant to which we may offer and sell common shares having an aggregate offering price of up to $682.0 million.

Previous wording

During the six months ended June 30, 2026, we sold and issued 17,164,940 common shares under the Equity Distribution Agreement, as amended, for net proceeds of approximately $178.9 million. In total, as of June 30, 2026, we have sold and issued 21,413,528 common shares under the Equity Distribution Agreement, as amended, for net proceeds of approximately $325.1 million. As of June 30, 2026, additional common shares having an aggregate offering price of up to $350.0 million remain available to be issued.

Current wording

During the nine months ended September 30, 2026, we sold and issued 17,164,940 common shares under the Equity Distribution Agreement, as amended, for net proceeds of approximately $178.9 million, all in the first quarter of 2026. In total, as of September 30, 2026, we have sold and issued 21,413,528 common shares under the Equity Distribution Agreement, as amended, for net proceeds of approximately $325.1 million. As of September 30, 2026, additional common shares having an aggregate offering price of up to $350.0 million remain available to be issued.

Previous wording

We expect that our cash, cash equivalents and marketable securities, as of the date of this Quarterly Report on Form 10-Q, will be sufficient to fund operating and financial commitments, and other cash requirements for at least one year after the issuance date of these financial statements.

Current wording

We expect that our cash, cash equivalents and marketable securities, as of the date of this Quarterly Report on Form 10-Q, and upfront payments from our recently executed out-license agreements will be sufficient to fund operating and financial commitments, and other cash requirements for at least one year after the issuance date of these financial statements. See Note 1, “Nature of the Business and Basis of Presentation.”

Previous wording

To execute our business plans, we will require funding to support our continuing operations and pursue our growth strategy. Until such a time as we can generate significant revenue from product sales or royalties, if ever, we expect to finance our operations through public or private equity financings, debt financings or other capital sources, including collaborations with other companies or other strategic transactions. We may not be able to obtain financing on acceptable terms, or at all. The terms of any financing may adversely affect the holdings or the rights of our shareholders. If we are unable to obtain funding, we could be forced to delay, reduce, or eliminate some or all of our research and development programs, product portfolio expansion or commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations.

Current wording

To execute our business plans, we will require funding to support our continuing operations and pursue our growth strategy. Until such a time as we can generate significant revenue from product sales or royalties, if ever, we expect to finance our operations through public or private equity financings, debt financings or other capital sources, including collaborations and licensing agreements with other companies or other strategic transactions. We may not be able to obtain financing on acceptable terms, or at all. The terms of any financing may adversely affect the holdings or the rights of our shareholders. If we are unable to obtain funding, we could be forced to delay, reduce, or eliminate some or all of our research and development programs, product portfolio expansion or commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations.

Previous wording

During the six months ended June 30, 2026, there were no material changes to our critical accounting policies as reported in our annual consolidated financial statements included in the 2025 Form 10-K.

Current wording

During the nine months ended September 30, 2026, there were no material changes to our critical accounting policies as reported in our annual consolidated financial statements included in the 2025 Form 10-K.