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Item 1. Business

2026-03-02 compared with 2025-03-03 · 490 added, 429 removed, 809 unchanged (53% of the section changed)

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We are advancing our innovative portfolio of therapeutics, leveraging our proven drug development experience and multiple proprietary drug development platforms.

Our extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders;

extracellular protein degradation for immunological diseases;

Transient Receptor Potential Melastatin 3 ("TRPM3") antagonism for migraine and neuropathic pain;

Tyrosine Kinase 2/Janus Kinase 1 ("TYK2/JAK1") inhibition for neuroinflammatory disorders;

glutamate modulation for obsessive-compulsive disorder (“OCD”) and spinocerebellar ataxia ("SCA");

myostatin inhibition for neuromuscular and metabolic diseases, including spinal muscular atrophy ("SMA") and obesity;

and antibody recruiting bispecific molecules ("ARMs") and antibody drug conjugates ("ADCs") for cancer.

Separation from Biohaven Pharmaceutical Holding Company Ltd.

On October 3, 2022, Biohaven Pharmaceutical Holding Company Ltd.

(the “Former Parent”) completed the distribution (the “Distribution”) to holders of its common shares of all of the outstanding common shares of Biohaven Ltd.

and the spin-off of Biohaven Ltd.

from the Former Parent (the “Spin-Off”) described in Biohaven’s Information Statement attached as Exhibit 99.1 to Biohaven’s Registration Statement on Form 10, as amended (Reg.

Collectively, we refer to the Distribution and Spin-Off throughout this Annual Report on Form 10-K ("Annual Report") as the "Separation." As a result of the Separation, Biohaven Ltd.

became an independent, publicly traded company as of October 3, 2022, and commenced regular way trading under the symbol “BHVN”’ on the New York Stock Exchange (the "NYSE") on October 4, 2022.

Where we describe historical business activities in this report, we do so as if the Former Parent’s activities related to such assets and liabilities had been performed by the Company.

(the “Former Parent”) completed the distribution to holders of its common shares of all of our outstanding common shares and the spin-off of Biohaven Ltd.

from the Former Parent (the “Separation”).

As a result of the Separation, Biohaven became an independent, publicly traded company as of October 3, 2022, and commenced regular way trading under the symbol “BHVN”’ on the New York Stock Exchange on October 4, 2022.

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.

Our 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").

The product candidates chart below summarizes our most advanced programs, including those that are key clinical stage programs and some of our preclinical programs that are beginning to advance to the clinic.

Product Candidates

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We hold the worldwide rights to substantially all of our product candidates.

MoDE and TRAP Degraders

Bispecific Molecular Degraders of Extracellular Proteins and TRAP Degraders

Biohaven Molecular Degrader of Extracellular Protein (“MoDE”) and Targeted Removal of Aberrant Protein ("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.

Our lead MoDE, BHV-1300, has demonstrated deep lowering of IgG > 80% in Phase 1 clinical trials and is being developed as a proprietary subcutaneous formulation in conjunction with an autoinjector for easy-to-use self-administration.

Data in the first Graves' patient dosed with BHV-1300 demonstrated complete suppression of autoantibodies targeting the TSH receptor and normalization of T3 and T4 within one month of dosing.

We plan to initiate a pivotal trial in 2026.

BHV-1400 , Biohaven's first TRAP molecule, is designed to specifically target the pathogenic driver of 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 2026.

Data from the first, and lowest, dose cohort 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 within the first month of dosing.

BHV-1300 has demonstrated deep lowering of IgG1, 2 and 4 in Phase 1 clinical trials and is being developed as a proprietary subcutaneous formulation in conjunction with an autoinjector for easy-to-use self-administration.

BHV-1300 was rationally designed to spare IgG3, potentially allowing for preservation of host defense.

BHV-1300 is being developed for the treatment of common immune mediated-diseases, such as Graves' Disease and Rheumatoid Arthritis ("RA").

Graves' Disease is a disease in which IgG1 autoantibodies stimulate the thyroid to produce excess thyroid hormone.

Targeted removal of disease-causing IgG has the potential to eliminate the pathogenic thyroid-stimulating antibody and modify the disease.

Graves' Disease is estimated to impact 1% of the population globally.

RA is a chronic autoimmune disease estimated to affect 1 to 2% of the global population.

RA primarily affects the joints, causing pain, swelling, stiffness, and loss of function.

We evaluated the effect of single and multiple doses of BHV-1300 in cynomolgus monkeys.

In September 2023, we reported data from confirmatory studies that showed a 75-80% reduction of IgG levels two days after a single dose and over 90% of IgG lowering after three doses.

Maximal lowering across FcRn inhibitors is 60-80% within approximately 7 to 21 days after initiation of single or multiple doses, respectively, in cynomolgus.

In contrast, a single dose of BHV-1300 lowers IgG by approximately 75 to 80% after approximately 2 days, and after three rapid doses to greater than 90% lowering.

The length of significant exposure to BHV-1300 is approximately one day within the dosage interval compared to continuous exposure required of the FcRn inhibitors.

Mechanism-related liabilities of FcRn inhibitors seen in animals and humans, including hypoalbuminemia and hypercholeresterolemia, are not expected and do not occur with BHV-1300 in cynomolgus.

See figures below comparing the speed and depth of lowering to FcRn inhibitors.

In January 2024, we reported preclinical pharmacodynamic single dose data with BHV-1300 which demonstrated the Biohaven IgG degrader technology allows for co-administration with Fc-containing biologics.

The PK of Humira® was unaltered after being dosed 12 hours after BHV-1300 administration (see figure below).

* Adapted from BLA 761154, IND 116471, Study no.

Our Phase 1 Study with BHV-1300

The Phase 1 SAD study examining BHV-1300 in healthy subjects was initiated in the first quarter of 2024.

In May 2024, we reported preliminary results from our Phase 1 study of BHV-1300.

These results in healthy subjects demonstrated that BHV-1300 rapidly and selectively lowers IgG in a dose-dependent manner in the first 4 cohorts completed to date (see figure below).

Preliminary IgG lowering data was consistent with modeling, with dose- and time-dependent IgG lowering observed even in initial low-dose cohorts.

Some subjects experienced IgG reductions as low as 50 to 70% of baseline.

BHV-1300 demonstrated reduction of IgG without significantly impacting LFTs, albumin, LDL cholesterol or other serum labs.

BHV-1300 has been safe and well tolerated to date, with no serious or severe adverse events.

Most adverse events ("AEs") were mild, deemed unrelated to study drug and resolved spontaneously.

As expected from the selectivity of the molecule for IgG, when compared to placebo, there were no meaningful reductions in average IgA, IgM or IgE levels during the week after dosing.

No adverse trends have been observed in vital signs or ECGs.

Modeling suggested additional cohorts in the Phase 1 study would achieve greater than 70% lowering of IgG utilizing doses compatible with subcutaneous administration.

The Phase 1 study has also compared intravenous and subcutaneous administration of BHV-1300.

Subcutaneous administration of BHV-1300 demonstrated an average of approximately 44% higher than expected exposure compared to the dose-equivalent intravenous formulation without injection site reactions.

This new human data confirms the feasibility of our plan for convenient dosing of BHV-1300 with a patient self-administered subcutaneous autoinjector.

In the fourth quarter of 2024 we provided an update to our Phase 1 study of BHV-1300, reporting that subcutaneously administered BHV-1300 achieved deep lowering of targeted IgG (IgG1, IgG2, IgG4, over IgG3), with reductions over 60% in the lowest subcutaneous dose tested in the ongoing MAD study.

Subcutaneous BHV-1300 achieved progressive reduction in IgG within hours of each weekly dose administration in the MAD, and pharmacodynamic effects were sustained relative to baseline over the four-week study period.

BHV-1300 has been safe and well-tolerated across the Phase 1 study.

There were no clinically significant effects on albumin or liver function, and no increases in cholesterol were noted.

Further enhancing the competitive safety profile and as intentionally designed, plasma IgG3 levels were preserved through the end of study week 4 to allow for healthy immune effector functioning.

All AEs were mild, any drug-related AE resolved, and there were no discontinuations due to study drug related AEs.

The optimized subcutaneous formulation in the MAD also showed substantially less inter-patient variability compared to previously reported intravenous BHV-1300.

In addition, we announced that we entered into an agreement with Ypsomed to develop and manufacture BHV-1300 in an easy-to-use, autoinjector for self-administration intended for commercialization.

In March 2025, we provided further updates to our ongoing Phase 1 study of BHV-1300.

In the four-week Phase 1 study, subcutaneously administered BHV-1300 at a dose of 1000 mg weekly achieved rapid, deep and sustained reductions

in total IgG of up to 84%, with a median reduction of 80%.

Reductions occurred within hours of each dose, were progressive, and were sustained compared to baseline over the four-week period.

In May 2025, we released new data from our clinically concluded Phase 1 study of BHV-1300.

In the Phase 1 multiple-dose study, subcutaneously administered BHV-1300 achieved IgG reductions up to 87%.

Median maximum reductions of 83% were achieved within 18 days (see figure below).

We recently reported the 1000 mg weekly dose achieved rapid, deep and sustained reductions in total IgG of up to 84%, with a median reduction of 80% by Week 4.

Reductions at all doses occurred within hours of administration, were progressive, and effects were durable between dosing intervals.

The range of IgG lowering enabled by different dose levels of BHV-1300 offers tunability and flexibility in dosing paradigm, with higher doses planned for management of acute conditions, and lower, less frequent dosing planned for the management of chronic disease.

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 has been initiated to evaluate the effect in participants with Graves' disease.

We plan to initiate a pivotal trial of BHV-1300 in Graves’ disease in 2026 and expect to pursue additional follow-on studies in other autoimmune diseases.

We are evaluating and have not yet finalized potential clinical trial designs, including size and primary and secondary endpoints.

In January 2025, we announced that the first-in-patient clinical experience with 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.

BHV-1400 is the first TRAP degrader introduced by Biohaven, a selective MoDE which is being developed to target Gd-IgA1, an aberrant immunoglobulin that drives IgA Nephropathy (see figure below).

Specific removal of pathogenic Gd-IgA1 and associated circulating immune complexes with preservation of normal IgA potentially permits disease remission without incurring an infection risk.

We shared preliminary data demonstrating the chimeric antibody-ASGPR ligand conjugate specifically mediated endocytosis of Gd-IgA1, as opposed to normal IgA1 and IgA2, in an endocytosis assay with ASGPR-expressing cell lines, and that TRAP degraders successfully internalize and degrade these immune-complexes.

In mice, after deposition of Gd-IgA1 in the glomerular mesangial matrix followed by administration of BHV-1400, Gd-IgA1 is progressively removed and no longer detected by six hours.

Our Phase 1 Study with BHV-1400

We initiated Phase 1 studies of BHV-1400 in the fourth quarter of 2024.

The first-in-human ("FIH") trial is a randomized, open-label, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability, PK, and PD of BHV-1400 in healthy volunteers.

In the first quarter of 2025, Biohaven announced deep and selective lowering of Gd-IgA1 with the first dose cohort tested in the SAD.

Subjects achieved median Gd-IgA1 lowering of 60% within 4 hours of dose administration without clinically significant lowering of healthy immunoglobulins IgA, IgE, IgM, or IgG.

As a next generation TRAP degrader, BHV-1400 is a potential therapeutic for the treatment of IgA nephropathy, and highlights the precision of MoDE platform molecules in their ability to selectively remove a pathogenic disease-causing protein without suppressing the healthy immune system.

In May 2025, we announced further data from the Phase 1 study of BHV-1400.

In the Phase 1 study, 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% (see figure below).

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.

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 related AEs, and there were no serious or severe study drug related 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 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 as a surrogate endpoint for accelerated approval.

In the fourth quarter of 2025, we completed a meeting with the U.S.

Food and Drug Administration ("FDA") to align on a pivotal IgAN study design, which we expect to initiate in the first quarter of 2026.

We are evaluating and have not yet finalized potential clinical trial designs, including size and primary and secondary endpoints.

In January 2025, we announced that first dosing of 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 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.

Kv7 Platform Acquisition

In February 2022, we announced that we entered into a definitive agreement with Channel Biosciences, LLC, a subsidiary of Knopp Biosciences, LLC, to acquire a drug discovery platform targeting Kv7 ion channels, adding the latest advances in ion channel modulation to our growing neuroscience portfolio.

Kv7’s Role in Epilepsy, Mood Disorders, and Other Central Nervous System Disorders

Because of their fundamental role in health and their aberrant role in disease, ion channels in cell membranes represent a broad and important class of drug targets.

Sodium channels and potassium channels form the ionic basis of the action potential in electrically charged cells throughout the body (see figures below).

The Kv7 protein in particular forms a channel that exquisitely regulates the flow of charged potassium ions (K+) across cell membranes, repolarizing nerve cells and resetting them for normal action potential firing.

Kv7 channels include a family of channel subtypes, designated as Kv7.1 through Kv7.5, and they are formed by tetramers of identical or compatible subunits.

Some of these channel subtypes localize in nerve cells (neurons) while others can be found in cardiac muscle, smooth muscle, and other tissue types.

The Kv7 subunits, Kv7.2 and Kv7.3, are widely expressed in the brain, notably in the cortex and hippocampus, and together they form Kv7.2/7.3 heteromeric channels that produce the M-current ( I KM ), a critical regulator of neuronal excitability (see figures below).

Kv7.2/7.3 channels normally perform a natural “braking” function by regulating the electrical excitability and hyperexcitability of brain cells.

Dysfunction of these channels, due to genetic mutations or other factors, increases seizure risk, while augmenting the ‘open’ activity of these channels has been demonstrated to reduce neuronal hyperexcitability and seizure frequency in electrophysiology laboratories, in animal models, and, most importantly, in patients.

White, Role of Potassium Channel Ions in Epilepsy, Medscape.org

We are synthesizing novel Kv7.2/7.3 activators that improve on the selectivity, potency, and other characteristics of ezogabine (Potiga in the U.S.

and Trobalt (retigabine) in Europe), a drug approved in 2011 for the treatment of refractory epilepsy and voluntarily withdrawn from the market in 2017 because of poor tolerability and structure-related toxicities that limited its clinical use, and ezogabine-like compounds, while averting its negative attributes, including off-target activity at a different brain ion channel, gamma-aminobutyric acid (“GABA”) A receptor (“GABA A -R”).

Using a structure-based approach, supplemented by in silico modeling, we have identified structural features of our molecules critical to Kv7 activation.

We have applied these analyses to the generation of proprietary chemical leads structurally distinct from known Kv7 activators, including ezogabine and flupirtine, the only other approved Kv7 modulator, approved in Europe for the treatment of acute pain.

Our team has synthesized a large library of Kv7-activating molecules and are advancing them according to stringent criteria requiring improvements over ezogabine, including chemical stability, synthetic tractability, the avoidance of structural motifs associated with the generation of reactive metabolites and other unwanted, off-target activity, including GABA A -R activation.

Epilepsy, Major Depressive Disorder ("MDD"), and Bipolar Disorder are the initial indications we targeted with activators from our Kv7 platform.

In addition to their role in treating epilepsy, anti-seizure medications ("ASMs") are often used in the management of bipolar disorder and Kv7 activators have recently shown promise in the treatment of MDD.

While the use of ASMs is often accompanied by dose-limiting side effects, our Kv7 activators are specifically designed to target subtypes of Kv7 potassium channels without engagement of GABA A receptors.

The lack of GABA A -R activity potentially gives activators from our Kv7 platform a wide therapeutic window and is expected to result in an improved side effect profile, limiting the somnolence and fatigue often seen in patients receiving ASMs.

Opakalim (BHV-7000)

Opakalim (formerly referred to as BHV-7000), the lead asset from the Kv7 platform, is a potent Kv7.2/7.3 ion channel activator from a novel, bicyclic imidazole class that is structurally-distinct from other Kv7 modulators (e.g., ezogabine and related aniline compounds) with significant in vivo anticonvulsant activity and a wide therapeutic index.

In the most widely used and positively-predictive preclinical model of epilepsy, the maximal electroshock (“MES”) model, data for opakalim and ezogabine were collected in independent experiments (see figures below), measuring the activity of both compounds in preventing seizures (ED50) and recording the neurologic deficit five minutes prior to the MES test to calculate the tolerability index (“TI”).

The neurologic deficit is a behavioral index ranging from normal activity (score of 0) to a loss of righting reflex (score of 3).

As shown below, opakalim was demonstrated to have an ED50 = 0.5 mg/kg with almost no impact on behavior producing a TI > 40x.

In a separate rotarod experiment of preclinical tolerability, opakalim had no impact on rat motor behavior up to 30 mg/kg, the highest dose tested.

In contrast, ezogabine was 40x less potent (ED50 = 20 mg/kg) in the MES model with a narrow TI < 3x.

The narrow preclinical TI for ezogabine is consistent with the clinical

experience with the drug where side effects such as somnolence and dizziness limited its use at doses that prevented seizures in patients.

Phase 1 Clinical Development

In the second quarter of 2022, our Clinical Trial Application for opakalim was approved by Health Canada, and we subsequently began phase 1 clinical development.

First-in-human single ascending dose ("SAD") and multiple ascending dose ("MAD") studies were completed with an immediate release formulation.

In the SAD and MAD cohorts, 77 subjects received opakalim (N=58) or placebo (N= 19).

Thirty-nine SAD subjects were randomized to opakalim or placebo and thirty-eight MAD subjects were randomized to opakalim or placebo.

The rates of AEs by MedDRA System Organ Class

across the pooled SAD and MAD cohorts among subjects treated with opakalim and placebo are presented in the tables below.

Treatment‐emergent adverse events ("TEAEs") Occurring in ≥ 2 Subjects Receiving opakalim in SAD Cohorts

All AEs reported in the SAD cohorts were mild in severity.

TEAEs Occurring in ≥ 2 Subjects Receiving opakalim in MAD Cohorts

All AEs reported in the MAD cohorts were mild in severity, except 1 case of back pain (moderate severity, 40 mg) and 1 case of dizziness (moderate severity, 80 mg).

a Data are included from a separate study evaluating higher MAD doses.

b Data are pooled across studies.

In 2023, we initiated a Phase 1 open-label electroencephalogram ("EEG") study designed to evaluate the effects of opakalim on EEG parameters in healthy adults.

The study’s objective was to demonstrate opakalim target engagement in the cerebral cortex and to help refine dose selection for Phase 3 trials.

Study measures included continuous EEG monitoring, time locked pharmacokinetic ("PK") sampling, and changes in EEG spectral power post dose.

The Phase 1 EEG study was designed to evaluate qualitative changes from baseline in EEG spectral power after administration of single doses of opakalim (10, 25, or 50 mg) to 11 healthy male and female adult volunteers.

EEG spectral power is a measure derived from quantitative analysis of EEG signals that assesses the amount of rhythmic activity in different frequency bands, including delta [1-3.5 Hz], theta [3.5-7.5 Hz], alpha [7.5-13 Hz], beta [13-30 Hz], and gamma [30-100 Hz].

Changes in spectral power have been used to evaluate the risk, onset and progression of seizures, assess cognitive and behavioral impairments, and characterize the effects of anti-seizure medications ("ASMs"), and they may also have utility in refining dose selection in clinical trials of ASMs.

Spectral analysis was performed by Clouds of Care (Ghent, Belgium), a global leader in EEG analytics.

Interim data from the Phase 1 EEG study were presented in December 2023 at the American Epilepsy Society meeting in Orlando, Florida.

Opakalim was well-tolerated at all doses studied, without the typical CNS AEs associated with other ASMs, such as somnolence or cognitive/mood disturbances, and EEG data showed dose-dependent increases in brain spectral power in healthy subjects (Figure 1 below).

Unlike prior reports where EEG effects of a Kv7.2/7.3 activator showed the greatest power increase in the delta frequency band (Biondi et al.

2022), the highest spectral power increases with opakalim were seen in alpha, beta, and gamma frequency bands (Figure 2 below).

While changes in spectral power were observed across all frequency bands with opakalim, the minimal impact on slower frequencies (i.e., delta) is consistent with the low incidence of CNS adverse events, in particular somnolence, seen in the opakalim Phase 1

SAD/MAD studies.

EEG delta activity is associated with somnolence, an undesirable CNS adverse event often seen with other ASMs.

Opakalim shows dose-dependent increases in spectral power over all brain regions.

Opakalim effect on spectral power in resting -state EEGs.

Greatest impact observed on alpha frequency with minimal impact on delta and theta frequencies.

The preliminary EEG study results confirm the CNS activity of opakalim at projected therapeutic concentrations, dose-dependent and time-dependent changes in EEG spectral power, and are consistent with the quantitative EEG effects observed with other ASMs approved for the treatment of epilepsy.

Based on the results from the EEG study and the safety profile in SAD/MAD trials, along with PK data from a new once-daily extended-release (“ER”) formulation, Biohaven is exploring three oral doses of opakalim (once daily 25 mg ER, once daily 50 mg ER, and once daily 75 mg ER) in the Phase 2/3 clinical trials in epilepsy and mood disorders.

This dosing approach with a Kv7 activator will allow for assessment of distinct target concentrations over a wide range.

In December 2024, t the American Epilepsy Society meeting, we presented additional safety data with the opakalim once-daily extended-release formulation, which further demonstrated tolerability.

Epilepsy affects approximately 3.5 million Americans, or more than 1.2% of adults and 0.6% of children in the U.S., and more than 50 million patients worldwide, according to the World Health Organization (“WHO”).

It is the fourth most common neurological disorder, and many patients struggle to achieve freedom from seizures, with more than one third of patients requiring two or more medications to manage their epilepsy.

While the use of anti-seizure medications is often accompanied by dose-limiting side effects, our clinical candidate opakalim is specifically designed to target subtypes of Kv7 potassium channels without engagement of GABA A receptors.

The lack of GABA A -R activity potentially gives opakalim a wide therapeutic window which we expect to result in an improved side effect profile, limiting the somnolence and fatigue often seen in patients receiving anti-seizure medications.

We aim to bring this potassium channel modulator as a potential solution to patients with epilepsy who remain uncontrolled on their current regimens.

Our Clinical Trials for Opakalim in Epilepsy

Focal Epilepsy

In January 2024, we completed our End-of-Phase 2 meeting with the FDA to advance to Phase 3 trials and announced that more than 110 global clinical sites have been selected in the first of two focal epilepsy trials.

Enrollment in our Phase 2/3 program commenced in the first quarter of 2024.

The two pivotal studies evaluating the efficacy of opakalim in refractory focal epilepsy are planned as randomized, double-blind, placebo-controlled, 8- and 12-week trials with a primary endpoint of change from baseline in 28-day average seizure frequency in adults with focal epilepsy.

RISE 3 is evaluating 50 mg and 75 mg doses of opakalim (see figure below).

We expect to report topline results from RISE 3 in the second half of 2026.

RISE 2 Part A is evaluating 25 and 50 mg doses of opakalim, whereas Part B is evaluating the 75 mg dose of opakalim (see figure below).

The RISE 2 study was amended to add Part B with the higher 75 mg dose, thereby replicating the potential therapeutic benefits of the higher 75 mg dose in the RISE 3 study and optimizing the overall development plan for opakalim.

The Company expects estimated enrollment in each study to be 390 participants.

In addition, Biohaven is currently conducting an open-label extension ("OLE") study to evaluate the long-term efficacy and safety of opakalim in participants who completed either parent study.

Review of data from the ongoing open-label clinical trial experience with opakalim in focal epilepsy support the potential for opakalim to achieve efficacy and to deliver a favorable and differentiated safety profile.

Open-label treatment with opakalim demonstrated clinically meaningful reductions in seizure frequency compared to the pretreatment baseline observation period prior to randomization.

Specifically, 55% of participants showed ≥50% reductions in seizure frequency (≥50% responder rate), for those who completed at least 6 months of treatment with opakalim 75 mg once daily in the open-label study;

and this result is comparable to the ≥50% responder rate published for other investigational agents in the class such azetukalner (which has reported 56% of patients with a ≥50% responder rate over any consecutive best 6-month period from its Phase 2b OLE data).

Notably, the antiseizure effects of opakalim were correlated with plasma concentrations, based on a preliminary exposure-response analysis.

Opakalim was well-tolerated in the open-label study with a low incidence of CNS adverse events, consistent with prior studies with opakalim.

Myostatin Platform

Taldefgrobep Alfa (BHV-2000)

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), a novel Phase 3 asset.

Myostatin, a negative regulator of muscle growth, is a key member of the Transforming Growth Factor ("TGF-β") superfamily.

Taldefgrobep's novelty in a field of myostatin-activin pathway inhibitors is based on the mechanism where it binds to myostatin to both lower overall free myostatin levels, but also to function as a receptor antagonist to block myostatin signaling in skeletal muscles.

Recently generated data has shown that the taldefgrobep alfa-myostatin complex is stable, and blocks myostatin signaling to both myostatin and to a lesser extent to activin A potentially for a protracted period after cessation of dosing.

Blocking myostatin and activin signaling has been shown to improve muscle function and strength in a number of disease models for neuromuscular wasting along with physical and metabolic changes important to individuals living with overweight and obesity.

Blocking activin contributes to decreased adipose tissue and improved glucose homeostasis.

Clinical studies have confirmed that taldefgrobep improved lean body mass directly through increase on contractile muscle and loss of adipose tissue as demonstrated in both normal healthy volunteers and in patients with Duchenne

muscular dystrophy (“DMD”).

The mechanism of increasing overall muscle size and reducing adipose tissue volume provide development opportunities in both neuromuscular disease and individuals living with overweight and obesity.

The advanced taldefgrobep alfa anti-myostatin development program offers extensive human safety data, especially in the pediatric population.

Metabolic Disorders

Obesity is a disease of excess and/or abnormal deposits of adipose tissue and a current global public health crisis.

By 2030, it is expected that nearly one billion people will be living with obesity, including 50% of the adult and 25% of the adolescent U.S.

The primary driver of obesity-related morbidity and mortality is metabolically active visceral adipose tissue and associated deposits of adipose tissue in and around organs such as the heart, liver, kidneys, and muscle.

Taldefgrobep Alfa’s Role in the Management of Overweight and Obesity

Preclinical and clinical data have demonstrated the potential for anti-myostatin therapies to produce physical and metabolic changes that are highly relevant to individuals living with overweight and obesity, including reducing total body fat and visceral adiposity, and improving insulin sensitivity and bone mineral density, while increasing lean muscle mass.

Taldefgrobep’s novel mode of action and unique impact on body composition suggest it could be used as monotherapy or in combination with other anti-obesity medications.

In October 2023, we announced preclinical data demonstrating the ability of taldefgrobep to significantly reduce fat mass while increasing lean mass in an obese mouse model.

In a mouse model of diet-induced obesity, untreated mice exhibited an increase in fat mass of 31%, while the mice treated with taldefgrobep demonstrated increases in lean mass of 25% from baseline (p≤.0.001) and lost 11% of their baseline fat (p≤.0.001) compared to vehicle (placebo) treated mice.

Insulin and leptin levels were consistently lower in mice treated with taldefgrobep compared to the untreated mice.

There was no difference in food intake over time across the taldefgrobep and untreated mice, counter to what has been observed with incretin mimetics (e.g., semaglutide) which are consistently associated with a reduction in energy intake.

Our Clinical Trial for Taldefgrobep Alfa in Obesity

In a Phase 1 MAD study conducted by BMS in normal healthy volunteers, the cohort using the projected therapeutic 45mg dose of taldefgrobep demonstrated a continued improvement in lean mass and a reduction in fat mass during the 29 day dosing period that continued to increase through the observation period at 4 weeks post dosing (see figure below).

In May 2024, we announced preclinical data from a diet induced obesity mouse model, which showed treatment with taldefgrobep alfa together with a glucagon-like peptide-1 ("GLP-1") agonist produced greater reductions in body weight and fat mass, and a larger increase in lean muscle mass, compared to treatment with GLP-1 alone (see figure below).

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 and expects to report topline results in 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 open-label extension 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 Clinical Trials

Taldefgrobep was previously studied by Bristol Meyers Squibb ("BMS") and Roche in 4 completed clinical studies in healthy volunteers and subjects with DMD.

An estimated 360 subjects received taldefgrobep;

179 healthy subjects and 181 subjects with DMD.

Completed clinical pharmacology studies in healthy adult subjects included:

• CN001001 was a randomized, placebo-controlled, Phase 1 study designed to evaluate the safety, tolerability, PK, and PD of single and multiple ascending subcutaneous doses of taldefgrobep (vial) in healthy adults.

• CN001023 was a randomized, open-label, single dose, parallel-group, Phase 1 study designed to compare the bioavailability of subcutaneous injections in the arm, thigh, and abdomen and to evaluate the safety, tolerability, PK, and immunogenicity of taldefgrobep (pre-filled syringe) in healthy adults.

Completed clinical studies in subjects with DMD included:

• CN001006 was a multi-site, randomized, placebo-controlled, double-blind, dose-ranging, Phase 1b/2 study to evaluate the safety, tolerability, and PK of taldefgrobep in ambulatory boys with DMD aged ≥ 5 to < 11 years.

• CN001016 was a randomized, double-blind, placebo-controlled, Phase 2/3 study to assess the efficacy, safety, and tolerability of 2 doses of taldefgrobep in ambulatory boys with DMD aged ≥ 6 to < 12 years.

All studies supported the safety and tolerability of taldefgrobep with fixed doses from 35 mg up to 180 mg administered weekly, subcutaneously ("SC").

The pharmacokinetics and safety data from the Phase 1 studies supported the continued development with doses of 35 mg and 50 mg administered weekly SC.

In the Phase 3 Clinical Study, CN001016, a futility analysis based on this primary endpoint, conducted after approximately 30% of subjects had completed 48 weeks of study drug treatment, did not show any statistically significant treatment differences.

A summary of the development and outcome from these studies are published (Neurol Ther.

2024 Feb;13(1):183-219).

Other Non-Key Programs

In the fourth quarter of 2025, we initiated a strategic reprioritization of our development platforms and are now focused on our key programs to prioritize resources.

As a result, development of programs outside of our key programs (the "non-key programs") may be substantially downsized, paused or delayed.

Glutamate Modulation Platform

The most advanced product candidate from our glutamate receptor antagonist platform is troriluzole (previously referred to as trigriluzole and BHV-4157), which is currently in two Phase 3 trials in OCD and, for which our new drug

application (“NDA”) in Spinocerebellar Ataxia and marketing authorisation application ("MAA") have been accepted and are currently under review by the U.S.

FDA and European Medicines Agency ("EMA"), respectively.

Troriluzole is also being evaluated by the Global Coalition for Adaptive Research ("GCAR") as part of Glioblastoma Adaptive Global Innovative Learning Environment - NCT03970447 ("GBM AGILE"), a revolutionary patient-centered, adaptive platform trial for registration that tests multiple therapies for patients with newly-diagnosed and recurrent glioblastoma ("GBM").

The most advanced product candidate from our glutamate receptor antagonist platform is troriluzole (previously referred to as trigriluzole and BHV-4157).

Other product candidates include BHV-5500, an antagonist of the glutamate N-methyl-D-aspartate (“NMDA”) receptor and its oral prodrug BHV-5000.

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In July 2023, the FDA informed us that it would not review the submitted NDA application for troriluzole given that the study's primary endpoint was not met and thus, would not permit a substantive review.

In followup to the regulatory decision on the NDA application, we held followup meetings with the FDA regarding the SCA data.

In follow-up to the regulatory decision on the NDA application, we held follow-up meetings with the FDA regarding the SCA data.

We had constructive dialogue with the FDA regarding our SCA development program and potential future data analyses to address regulatory concerns in the previously issued refuse-to-file decision on the NDA application for SCA3.

In October 2023, the European Medicines Agency ("EMA") informed us that our Marketing Authorization Application ("MAA") for troriluzole (Dazluma) in the treatment of SCA3 was validated and was now under review by EMA's Committee for Medicinal Products for Human Use ("CHMP").

In October 2023, the European Medicines Agency ("EMA") informed us that our Marketing Authorization Application ("MAA") for troriluzole (Dazluma) in the treatment of SCA3 was validated and was then under review by EMA's Committee for Medicinal Products for Human Use ("CHMP").

In the fourth quarter of 2024, we completed a clarification meeting with the CHMP Rapporteurs.

The MAA documents were subsequently updated with a broader indication to include all SCA genotypes, in light of the new positive BHV4157-206-RWE study data (discussed below).

In March 2025, we decided to withdraw the troriluzole MAA in the EU for the treatment of adult patients with SCA.

This withdrawal decision was based on feedback indicating the EMA would not be able to conclude on new active substance ("NAS") status for troriluzole due to insufficient data.

NAS is an important designation recognizing and incentivizing development innovation, validating the differentiation of a new medicinal product for patients.

The novelty of troriluzole has been recognized by multiple issued patents globally, and we are committed to expeditiously providing appropriate data and/or argumentation to EMA given the evidence that warrants granting NAS, in the spirit in which the designation was intended.

We plan to generate data to work with EMA on next steps towards marketing authorization.

In September 2024, we announced positive topline results from pivotal Study BHV4157-206-RWE (NCT06529146) demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment.

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In February 2025, the FDA accepted for review our NDA for troriluzole for the treatment of adult patients with SCA and granted priority review.

The FDA's decision regarding the NDA is expected during the third quarter of 2025.

Based on FDA priority review timelines and if ultimately approved, we are prepared to commercialize troriluzole for SCA in the U.S.

In November 2025, we received a Complete Response Letter ("CRL") for the NDA seeking approval of troriluzole for SCA.

In the CRL, the FDA recommended that we meet with the Division of Neurology 1 within the FDA's Office of Neuroscience to discuss the evidence that will be needed to support a future NDA for the treatment of SCA with troriluzole.

Following receipt of the CRL, we held a Type A meeting with the FDA to initiate an appeal process, given the large number of patients who are currently being treated in the expanded access program.

We remain committed to working with the FDA to find a path forward for our NDA.

Troriluzole for OCD

OCD is a chronic neuropsychiatric disorder characterized by symptoms of obsessions (intrusive thoughts) and compulsions (repetitive behaviors) that can interfere with patients’ functional abilities.

According to the National Institute of Mental Health, the 12-month prevalence of OCD is 1% of the U.S.

adult population, and approximately half of these cases are characterized as severe.

First-line treatment for OCD includes cognitive behavioral therapy, selective serotonin reuptake inhibitors (“SSRIs”) and adjunctive use of atypical antipsychotics.

Nonetheless, up to 60% of patients have an inadequate response to conventional intervention strategies and some seek invasive neurosurgical procedures to ameliorate symptoms.

We are currently developing troriluzole as a potential treatment option for patients suffering from OCD.

Despite the significant public health burden, no novel mechanisms of action have been approved by the FDA for OCD in over two decades.

The rationale for use of troriluzole in OCD is supported by clinical data with its active metabolite, riluzole, in populations with OCD in open-label and placebo-controlled clinical trials as well as in preclinical, genetic and neuroimaging studies implicating the glutamatergic hyperactivity in the pathogenesis of OCD.

In multiple case studies, the use of riluzole in patients with refractory OCD has commonly been associated with meaningful improvement of symptoms.

A small-scale randomized controlled trial in adults with OCD conducted by a third party showed favorable trends for the use of riluzole in an outpatient setting.

Another randomized controlled third-party study demonstrated statistically significant therapeutic effects with the adjunctive use of riluzole as compared to adjunctive placebo in 50 adults with refractory OCD.

These clinical effects are consistent with findings such as genetic associations of glutamate transporter genes with OCD and increased glutamate concentrations in brain and cerebrospinal fluid of patients with OCD.

Taken together, we believed there was a clear rationale for advancement of troriluzole, a prodrug of riluzole, into a Phase 2 proof-of-concept trial in OCD.

We commenced a Phase 2/3 double-blind, randomized controlled trial on the use of troriluzole in adults with OCD in late 2017.

Results from the Phase 2/3 trial were announced in June 2020.

Troriluzole 200 mg administered once daily as adjunctive therapy in OCD patients with inadequate response to standard of care treatment showed consistent numerical improvement over placebo on the Yale-Brown Obsessive Compulsive Scale (“Y-BOCS”) at all study timepoints (weeks 4 to 12) but did not meet the primary endpoint at week 12.

Troriluzole treated subjects (n = 111) had a mean Y-BOCS improvement of -3.4 points from baseline versus -2.9 for placebo-treated (n = 115) subjects [difference -0.5 and p-value = 0.451] at week 4, -5.1 points (n = 96) versus -3.6 for placebo-treated (n = 108) subjects [difference -1.5 and p-value = 0.041] at week 8, and -5.9 points (n = 99) versus -4.9 for placebo-treated (n = 102) subjects [difference -1.0 and p-value = 0.220] at week 12.

Troriluzole’s safety profile was generally consistent with past clinical trial experience with its active metabolite, riluzole.

Treatment emergent adverse events (“TEAEs”) were mostly reported to be mild in intensity.

TEAEs that occurred in at least 5% of patients in the troriluzole group, and more frequently in the troriluzole group than in the placebo group, were headache, dizziness, fatigue, somnolence, nausea and nasopharyngitis.

Given the strong signal in the Phase 2/3 proof-of-concept study and after receiving feedback from the FDA in an End of Phase 2 meeting, in December 2020, we initiated enrollment in a Phase 3 program.

The Phase 3 program will have an estimated total enrollment of up to 700 participants in each trial with a primary endpoint of change from baseline on the Y-BOCS total score at week 4, 8 and 10.

The two Phase 3 randomized, double-blind, placebo-controlled trials that make-up our Phase 3 program for OCD are currently ongoing.

In January 2024, we announced plans to conduct a pre-planned interim analysis ("IA") to evaluate efficacy in the first of our two Phase 3 studies in OCD.

The IA was planned to be conducted by an independent Data Monitoring Committee ("DMC") after approximately 70% of subjects in the primary analysis population reached the primary endpoint.

The Data Monitoring Committee convened in the second quarter of 2024 to review the IA and informed the Company that the study may continue.

As such, we continue enrolling patients in the first Phase 3 study in OCD and expect that this study will be fully enrolled in the first quarter of 2025.

We expect to report topline data from the first Phase 3 study in OCD in the first half of 2025.

There was a similarly designed pre-planned IA for the second Phase 3 study in OCD conducted in the fourth quarter of 2024, and the DMC also informed us that this study may continue.

We expect to report topline data from the second Phase 3 study in OCD in the second half of 2025.

We commenced a Phase 2/3 double-blind, randomized, controlled trial to assess the efficacy of troriluzole in adults with obsessive-compulsive disorder ("OCD").

This trial was followed by two Phase 3 randomized, double-blind, placebo-controlled trials.

The first Phase 3 study in OCD was completed with no efficacy signal detected.

Given the results of the first study, enrollment in the OCD program was closed to allow resources to be applied to other development programs.

Troriluzole for Glioblastoma

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Myostatin Platform

Taldefgrobep Alfa (BHV-2000)

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), a novel Phase 3 asset.

Myostatin, a negative regulator of muscle growth, is a key member of the Transforming Growth Factor ("TGF-β") superfamily.

Taldefgrobep's novelty in a field of myostatin inhibitors is based on the mechanism where it binds to myostatin to both lower overall free myostatin levels, but also to function as a receptor antagonist to block myostatin signaling in skeletal muscles.

Blocking myostatin activity and signaling has shown to improve muscle function and strength in a number of disease models for neuromuscular wasting along with physical and metabolic changes important to individuals living with overweight and obesity.

Blocking activin contributes to decreased adipose tissue and improved glucose homeostasis.

The mechanism of increasing overall muscle size and reducing adipose tissue volume provide development opportunities in both neuromuscular disease and individuals living with overweight and obesity.

The advanced taldefgrobep alfa anti-myostatin development program offers extensive human safety data, especially in the pediatric population.

Previous Clinical Trials

Taldefgrobep was previously studied by Bristol Meyers Squibb ("BMS") and Roche in 4 completed clinical studies in healthy volunteers and subjects with DMD.

An estimated 360 subjects received taldefgrobep;

179 healthy subjects and 181 subjects with DMD.

Completed clinical pharmacology studies in healthy adult subjects included:

Completed clinical studies in subjects with DMD included:

All studies supported the safety and tolerability of taldefgrobep with fixed doses from 35 mg up to 180 mg administered weekly, subcutaneously ("SC").

The pharmacokinetics and safety data from the Phase 1 studies supported the continued development with doses of 35 mg and 50 mg administered weekly SC.

treatment differences.

A summary of the development and outcome from these studies are published (Neurol Ther.

2024 Feb;13(1):183-219).

There are significant differences between a neuromuscular (DMD) and neurodegenerative disease (SMA).

The differences in residual muscle in SMA along with adjunctive survival of motor neuron (“SMN”) upregulation therapy support the continued development of taldefgrobep alfa in SMA.

Spinal Muscular Atrophy

Taldefgrobep Alfa (BHV-2000) for Spinal Muscular Atrophy

SMA is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness that is often fatal and typically diagnosed in young children.

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states and covers over 94% of all births.

In February 2023, we received Fast Track designation from the FDA for taldefgrobep alfa for the treatment of SMA.

We received orphan drug designation from the FDA for taldefgrobep in the treatment of SMA in December 2022 and from the European Commission in July 2023.

In April 2024, we announced that the FDA granted "rare pediatric disease" designation for taldefgrobep alfa.

The designation provides for the potential for taldefgrobep to receive a priority review voucher (“PRV”) if ultimately approved for the indication of SMA prior to September 30, 2026.

The rare pediatric disease PRV program began to sunset in December 2024 and will not apply for approvals after September 30, 2026.

Taldefgrobep Alfa’s Role in Spinal Muscular Atrophy

In the past few years, significant advancements were made to address the underlying cause of disease in SMA with the up-regulation of SMN1 and SMN2 expression which positions taldefgrobep as a potential combination therapy to enhance muscle performance.

Data from an SMA animal model study that shows advantages of combination SMN therapy with taldefgrobep and the extensive clinical data in DMD supported the advancement of taldefgrobep into a SMA Phase 3 study.

Other indications in muscle wasting diseases may be a follow-on for taldefgrobep along with other life-cycle opportunities.

Our Clinical Trial for Taldefgrobep Alfa in SMA

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Additional analyses of these subjects (n=123) who had measurable baseline myostatin (the pharmacological target of taldefgrobep) showed an improved efficacy signal within this myostatin-positive population (p=0.02).

Prespecified outcome measures in the overall study population analyzing the change from baseline in body composition at Week 48 demonstrated a greater reduction in the percent change in total body fat mass in the taldefgrobep arm compared to the placebo+SOC arm (p=0.008) as measured by dual energy x-ray absorptiometry ("DXA").

The taldefgrobep arm also showed numerically larger increases in lean muscle mass and bone density compared to the placebo+SOC arm.

Biohaven plans to engage the FDA in the first half of 2025 to discuss the potential registrational path forward and will present the study data at an upcoming conference.

Biohaven has begun engagement with the FDA to discuss the potential registrational path forward.

Data from the study was presented at the Cure SMA meeting in June 2025.

The optional long-term extension phase of the trial will remain ongoing pending further data analysis as well as regulatory discussions.

In February 2023, we received Fast Track designation from the FDA for taldefgrobep alfa for the treatment of SMA.

Fast Track designation enables important new drugs to reach patients earlier by facilitating more frequent communications with the FDA and expeditious review of a drug which treats a serious condition and fills an unmet medical need.

In December 2022, we received orphan drug designation from the FDA for taldefgrobep in the treatment of SMA and from the European Commission in July 2023.

In April 2024, we announced that the FDA granted "rare pediatric disease" designation for taldefgrobep alfa.

The designation provides for the potential for taldefgrobep to receive a priority review voucher (“PRV”) if ultimately approved for the indication of SMA prior to September 30, 2026.

The rare pediatric disease PRV program began to sunset in December 2024 and will not apply for approvals after September 30, 2026.

Taldefgrobep Alfa’s Role in Spinal Muscular Atrophy

Data from an SMA animal model study that shows advantages of combination SMN therapy with taldefgrobep and the extensive clinical data in DMD support the advancement of taldefgrobep into a SMA

Phase 3 study.

Other indications in muscle wasting diseases may be a follow-on for taldefgrobep along with other life-cycle opportunities.

Metabolic Disorders

Obesity is a disease of excess and/or abnormal deposits of adipose tissue and a current global public health crisis.

By 2030, it is expected that nearly one billion people will be living with obesity, including 50% of the adult and 25% of the adolescent U.S.

Taldefgrobep Alfa’s Role in the Management of Overweight and Obesity

Taldefgrobep’s novel mode of action and unique impact on body composition suggest it could be used as monotherapy or in combination with other anti-obesity medications.

In October 2023, we announced preclinical data demonstrating the ability of taldefgrobep to significantly reduce fat mass while increasing lean mass in an obese mouse model.

Insulin and leptin levels were consistently lower in mice treated with taldefgrobep compared to the untreated mice.

Our Clinical Trial for Taldefgrobep Alfa in Obesity

We plan to initiate a Phase 2 clinical trial of taldefgrobep in the management of metabolic disease in the first half of 2025.

We are evaluating and have not yet finalized potential clinical trial designs, including size and primary and secondary endpoints.

Ion Channel Platform