On January 12, 2026 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines in oncology and autoimmune diseases, reported the Company’s achievements in 2025 and outlined key objectives and anticipated milestones for 2026, which will be the subject of Nurix’s corporate update at the 44th Annual J.P. Morgan Healthcare Conference today at 4:30 p.m. PT, in San Francisco.

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"2025 was a defining year for Nurix, having advanced our potentially best-in-class BTK degrader, bexobrutideg, into pivotal development for patients with relapsed or refractory CLL," said Arthur T. Sands, president and chief executive officer of Nurix. "As we enter 2026, we are focused on executing the DAYBreak CLL-201 study and initiating the confirmatory Phase 3 trial, DAYBreak CLL-306. We are also excited to advance our pipeline of wholly owned and partnered programs in inflammation and autoimmune diseases, including our new tablet formulation of bexobrutideg, our IRAK4 degrader program partnered with Gilead, and our STAT6 degrader program partnered with Sanofi. With our pivotal DAYBreak program underway, a strong balance sheet, and multiple catalysts across oncology and immunology, we believe Nurix is exceptionally well positioned to make 2026 a transformative year for the Company and the field of targeted protein degradation."

2025 Select Accomplishments and Business Highlights

Potential Best-in-Class BTK degrader, Bexobrutideg, in CLL

Presented new and updated clinical and preclinical data supporting a potential best-in-class BTK degrader profile. New and updated clinical data were presented in December 2025 at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH2025) that provide a maturing clinical picture of bexobrutideg’s efficacy, durability, and tolerability. Highlights included an 83% objective response rate, including two complete responses (4.3%) in CLL patients with a median of four prior lines of treatment. Responses were durable and deepened over time with a median progression-free survival estimated at 22.1 months, which is highly competitive with currently approved agents in a similar line of therapy. Bexobrutideg was well tolerated with no dose-limiting toxicities across all doses tested. New preclinical data were presented in October 2025 that support bexobrutideg’s potential best-in-class BTK degrader profile, demonstrating superior degradation potency, broad coverage of clinically relevant BTK mutations, and exquisite selectivity. These findings strengthen the Company’s conviction that bexobrutideg may prove to be a clinically superior medicine for the treatment of patients with CLL and other B-cell driven diseases.

Successfully addressed FDA’s Project Optimus with the selection of the 600 mg once daily dose for pivotal development in r/r CLL. The selection of the 600 mg dose was supported by data from a randomized cohort within the Phase 1b study comparing 200 mg and 600 mg in accordance with Project Optimus and reflects alignment with global regulators, including the U.S. Food and Drug Administration, the UK Medicines and Healthcare products Regulatory Agency, and the European Medicines Agency. The results, subsequently reported at ASH (Free ASH Whitepaper)2025, demonstrated a trend toward a higher objective response rate and longer progression-free survival with the 600 mg dose without an increase in adverse events. The clearance by regulators of the 600 mg dose allows Nurix to optimize bexobrutideg’s therapeutic effect, providing patients the opportunity to regain control of CLL that has progressed or has failed to respond to other therapies.

Initiated pivotal clinical development in patients with relapsed or refractory CLL. In October 2025, Nurix initiated enrollment in the DAYBreak CLL-201 pivotal Phase 2 study (NCT07221500). This single-arm, global study is evaluating bexobrutideg at 600 mg once daily in patients with relapsed or refractory CLL whose disease progressed following treatment with a BTK inhibitor and a BCL2 inhibitor. The trial is designed to support accelerated approval of bexobrutideg in triple-exposed CLL/SLL patients. Nurix plans to initiate a randomized confirmatory Phase 3 trial in 2026 to support full approval of bexobrutideg.

First-in-class CBL-B Inhibitor NX-1607

Presented positive Phase 1a clinical data demonstrating immune activation and clinical activity. New Phase 1a clinical data for NX-1607 were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October and the 2025 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November, demonstrating dose-dependent pharmacologic activity and signals of clinical activity across a diverse set of 82 patients with eleven different tumor types. Clinical activity was observed through reductions in tumor-specific biomarkers such as prostate-specific antigen (PSA) in prostate cancer and carcinoembryonic antigen (CEA) in colorectal cancer. Notably, there was a confirmed partial response in a patient with micro-satellite stable colorectal cancer (MSS CRC), a tumor type typically unresponsive to immune checkpoint therapy. As anticipated, treatment with NX-1607 led to increased peripheral T cell activation and proliferation, indicating active T-cell receptor engagement and immune responsiveness, suggesting its potential as an active immune-oncology agent with a unique mechanism distinct from PD-1/PD-L1 therapies.

Advancing Pipeline in Inflammation and Autoimmune Diseases

Introduced new tablet formulation of bexobrutideg into Phase 1 testing to support an IND for inflammation and autoimmune indications. In 2025, Nurix initiated a Phase 1 single ascending and multiple ascending dose (SAD/MAD) study to evaluate pharmacokinetics (PK), pharmacodynamics (PD), and safety of a new tablet formulation of bexobrutideg. This study is intended to support an IND filing and enable expansion into inflammatory and autoimmune indications beginning in 2026.

Partner Gilead initiated Phase 1 testing of IRAK-4 degrader. In April 2025, Nurix announced that the FDA cleared the Investigational New Drug (IND) application for GS-6791 (previously NX-0479), a novel, potentially best-in-class oral degrader of IRAK4 being developed in collaboration with Gilead Sciences. GS-6791 is designed to selectively degrade IRAK4, a key signaling protein that drives inflammation in autoimmune and inflammatory diseases. Gilead subsequently initiated an ongoing Phase 1 trial in healthy volunteers, the results of which will inform Nurix’s option for a 50/50 co-development and U.S. profit share.

Partner Sanofi advanced STAT6 degrader program into IND enabling studies: In June 2025, Nurix announced that Sanofi exercised its option to extend its license for Nurix’s STAT6 program, including the development candidate NX-3911. NX-3911 is an oral, highly selective degrader of STAT6, a key transcription factor within the IL-4/IL-13 signaling pathways that drive inflammation in allergic and type 2 inflammatory conditions, which currently is in IND enabling studies. Sanofi is responsible for all development activities, and Nurix retains an option for a 50/50 U.S. profit share and co-promotion after initial clinical proof of concept.

Corporate and Leadership

Strengthened financial position to support execution of bexobrutideg pivotal program and expansion into inflammation and autoimmune disease. In October 2025, Nurix closed an underwritten registered offering of 24,485,799 shares of its common stock, providing gross proceeds to Nurix of $250.0 million, before deducting underwriting discounts and commissions and other offering expenses payable by Nurix. In addition, Nurix earned $47.0 million in non-dilutive capital through its strategic collaborations with Gilead, Sanofi and Pfizer. Nurix is well capitalized with pro forma cash/investments of $663.8 million1.

Strengthened leadership with appointments of chief commercial officer and new board members with deep experience in drug development and commercialization. In 2025, Nurix announced the hiring of John Northcott as chief commercial officer, bringing extensive U.S. and global commercial leadership experience, including both pre-launch planning and on-market commercialization in hematology, oncology and a wide range of other therapeutic areas. In addition Nurix also appointed two board members: Roy Baynes, MB.Bch., M.Med., Ph.D., who has extensive experience in the development of innovative, blockbuster medicines during a distinguished career in hematology and oncology, and Roger Dansey, M.D., senior leader in drug development and operations with over 25 years of executive experience in pharmaceutical and biotech companies across both U.S. and international markets.

2026 Outlook: Executing the Next Phase of Growth

Execute pivotal development pathway in CLL:
Enrollment of pivotal Phase 2 trial – DAYBreak CLL-201
Initiation of a confirmatory Phase 3 study in patients with r/r CLL in the 2L+ setting comparing bexobrutideg monotherapy to pirtobrutinib – DAYBreak CLL-306
Initiation of a Phase 1b/2 clinical study in patients with CLL in combination with other therapeutic agents including venetoclax (BCL-2 inhibitor)
Advance Degrader Programs in I&I:
Bexobrutideg – data from new tablet formulation SAD/MAD study supporting IND in I&I
GS-6791 IRAK4 degrader – potential Phase 1 results2
NX-3911 STAT6 degrader – potential IND filing by Sanofi2
Report Ongoing Clinical Data Updates:
Bexobrutideg Phase 1a/b CLL cohorts
Bexobrutideg Phase 1a/b NHL cohorts
Zelebrudomide Phase 1a cohorts
Progress Research and Development Pipeline:
Leverage DEL-AITM platform to fuel wholly owned and partnered drug discovery programs
Earn additional research milestones and potential licensing fees from its collaborations with Gilead, Sanofi, and Pfizer.

1Represents cash balance as of August 31, 2025, plus the net proceeds from the October 2025 registered direct offering

2Statements include Nurix estimate for partnered programs using industry standard timelines based on current stage of development (not official guidance of partners).

About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, highly selective small molecule degrader of BTK currently being evaluated in the DAYBreak CLL-201 clinical trial (NCT07221500), a pivotal single-arm Phase 2 study of bexobrutideg in patients with relapsed or refractory chronic lymphocytic leukemia. Bexobrutideg also continues to be studied in the NX-5948-301 Phase 1a/1b clinical trial (NCT05131022) in patients with relapsed or refractory B cell malignancies.

About NX-1607
NX-1607 is an investigational first-in-class oral inhibitor of the E3 ligase Casitas B-lineage lymphoma proto-oncogene B (CBL-B) being developed for immuno-oncology indications, including a range of solid tumor types. CBL-B is a cytoplasmic E3 ubiquitin ligase that negatively regulates T cell activation, making it an attractive target for immuno-oncology and offering a novel therapeutic approach to treat solid tumors. Inhibition of CBL-B in preclinical studies reverses T cell exhaustion, alleviates tumor induced immunosuppression, and may also exert direct antitumor effects. Nurix is evaluating NX-1607 in an ongoing Phase 1 trial in adults in a range of oncology indications. This study includes a thorough investigation of both dose and schedule in the Phase 1a portion. Additional information on the NX-1607 clinical trial can be accessed at www.clinicaltrials.gov (NCT05107674).

(Press release, Nurix Therapeutics, JAN 12, 2026, View Source [SID1234661962])

On January 12, 2026 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, reported that France’s National Agency for Medicines and Health Products Safety (ANSM) has approved an updated national treatment protocol for botensilimab (BOT) plus balstilimab (BAL) under France’s Autorisation d’Accès Compassionnel (AAC) framework.

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The updated protocol expands France’s previously granted AAC authorization for BOT+BAL in patients with microsatellite-stable (MSS) metastatic colorectal cancer (mCRC) without active liver metastases to eligible patients with certain ovarian cancers and soft-tissue sarcomas—diseases with substantial unmet medical need after standard options have been exhausted.

The revised protocol is designed to broaden the eligibility criteria, allowing more patients with advanced solid tumors to have early access to BOT+BAL under reimbursed compassionate use, and to enhance monitoring and treatment procedures in participating hospitals.

BOT+BAL is a chemotherapy- and radiation-free immunotherapy combination being evaluated in clinical studies and available in compassionate access settings authorized by ANSM. In studies conducted to date, antitumor activity has been observed in heavily pretreated patients, including in tumor types that have historically shown limited responsiveness to standard immunotherapy approaches.

France’s AAC pathway enables hospital-based access for patients with serious or life-threatening diseases who lack appropriate therapeutic alternatives. Treatment under AAC is governed by a national protocol that standardizes the conditions of use, including patient eligibility, treatment administration, effectiveness and safety data collection and follow-up under France’s national health system oversight.

For eligible French patients treated in hospital under AAC, BOT+BAL is fully reimbursed.

Expanded AAC eligibility

Under the updated national protocol, reimbursed AAC access to BOT+BAL is authorized in France for eligible adult patients with:

MSS metastatic colorectal cancer without active liver metastases, following progression on standard therapies;
Platinum-refractory or platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, after exhaustion of approved treatment options; and
Advanced or metastatic soft-tissue sarcomas, including multiple high-grade histologies, following failure of standard therapies.
Significance of the update

By extending fully reimbursed AAC access across colorectal cancer, ovarian cancer, and sarcoma, France has implemented a multi-tumor early access framework for a single investigational immunotherapy combination under one nationally standardized protocol. This represents an uncommon level of national early-access authorization and enables consistent hospital access to an investigational treatment while maintaining ANSM oversight and structured patient follow-up as additional clinical and real-world evidence continues to develop.

BOT+BAL remains investigational and is not approved for commercial marketing in France or elsewhere.

(Press release, Agenus, JAN 12, 2026, View Source;Balstilimab-for-Ovarian-Cancer-and-Soft-Tissue-Sarcomas/default.aspx [SID1234661978])

On January 12, 2026 Kainova Therapeutics ("Kainova" or "the Company"), a catalyst for breakthrough treatments for patients in immuno-oncology and inflammation, reported a transformative rebrand. The new name and brand reflect the Company’s clinical momentum, global focus, and readiness for the next chapter of strategic growth, anchored by a mature pipeline of G Protein-Coupled Receptor (GPCR)-modulating therapies.

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The name "Kainova" combines "Kairos," the opportune or decisive moment, and "Nova," symbolizing innovation and renewal. It embodies the Company’s transformation and ambition, reflecting its drive to innovate, determination to deliver, and commitment to therapies that truly transform lives. Built on a strong scientific foundation, a proprietary discovery platform, an integrated development approach, and experienced leadership, the new brand underscores Kainova’s commitment to clinical excellence, value creation, and lasting impact.

"The rebrand is not a break from our scientific or strategic roots", said Sean A. MacDonald, Chief Executive Officer of Kainova Therapeutics. "It is the clearer expression of who we are today and the impact we strive to deliver for patients. It highlights a continuation between our past successes and the growth path we envision for the future. Above all, Kainova Therapeutics embodies our belief that better is always possible."

Leveraging decades of scientific and medical expertise and a proven track record of collaborations with pharma, Kainova is advancing innovative, clinically differentiated therapies designed to unlock the untapped potential of GPCRs.

Kainova employs a fully integrated, forward-thinking approach to GPCR drug discovery and smart development, moving programs efficiently from concept to clinic and strengthening its position as a leader in the field. The Company’s leading programs include DT-7012, a Treg-depleting anti-CCR8 antibody with competitive properties in Phase I/II trials for solid tumors, DT-9046, a first-in-modality, pre-IND biased PAR2 antagonist in inflammation, and DT-9081, a Phase II-ready EP4 receptor antagonist for solid tumors.

With operations in North America, France, and Australia, and a growing clinical pipeline, Kainova is entering its next chapter, shaping the future of GPCR-modulating therapies, transforming lives, and expanding its global reach.

The Kainova leadership will be in San Francisco during the J.P. Morgan Healthcare Conference, January 12–15, 2026, and welcomes the opportunity to share how the Company is advancing its clinical pipeline of GPCR-modulating therapies to create a meaningful impact for patients.

(Press release, Domain Therapeutics, JAN 12, 2026, View Source [SID1234661989])

On January 12, 2026 Olema Oncology presented its corporate presentation.

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(Presentation, Olema Oncology, JAN 12, 2026, View Source [SID1234661963])

On January 12, 2026 Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported its broad portfolio progress at the 44th Annual J.P. Morgan Healthcare Conference. A copy of the slide presentation is available on the Events and Presentations section of the Biohaven website.

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "As we prioritize key programs across our portfolio that can efficiently deliver clear value for patients and investors, we are excited to highlight the first patient experiences using our proprietary MoDE and TRAP extracellular degraders at the annual J.P. Morgan Healthcare Conference. The early data continues to signal the important role that this transformative science can have in the treatment of immunological disease and the potential for our technology to provide differentiated treatments for patients suffering from immune-mediated conditions such as IgA nephropathy and Graves’ disease. Our lead programs represent just the beginning of this platform; we aim to transform immunological disease therapy by selectively removing disease-causing proteins as they circulate in the blood and body, fulfilling the potential of true precision immunology to target the root cause of diseases without immunosuppression."

"We’ve dosed our first patient in our obesity program with taldefgrobep that introduces a dual-action therapy to not only reduce fat but also build lean muscle mass, addressing a critical unmet need in metabolic health," continued Dr. Coric "We expect topline data from this Phase 2 obesity proof-of-concept trial in 2026. In addition, we continue to advance a clinically validated approach for treating focal epilepsy that targets Kv7 ion channels with pivotal topline data expected in 2026. Our advancements and discovery expertise demonstrate our commitment to developing innovative treatments that address a wide range of patient needs."

Biohaven 2026 Portfolio Review and Anticipated Milestones

Biohaven is positioned to achieve significant milestones in 2026 across key programs targeting indications with high unmet need. Throughout its clinical-stage portfolio, combined with its innovative discovery engine, the company is advancing a broad range of innovative medicines that represent paradigm-shifting treatment options.

Novel Degrader Platforms: Biohaven Raises the Bar in Precision Immunology

Biohaven is pioneering scientific innovation in extracellular protein degradation, a transformational therapeutic approach for autoimmune diseases that focuses on the precise targeting and selective removal of disease-causing proteins. Based upon the groundbreaking work from the Spiegel Chemistry Lab at Yale University and exclusively licensed by Biohaven, the company’s MoDE and TRAP degrader platform offers selectivity, speed, and patient-friendly self-administration to remove disease-causing extracellular proteins from the body to potentially treat a wide range of diseases.

As part of an initiative to further advance and accelerate next-generation degrader development, Biohaven also announced entering into an memorandum of understanding with the King Abdullah University of Science and Technology (KAUST) to collaborate on discovery efforts and leverage the University’s technology capabilities including strengths in its Smart Health initiatives, generative AI, and supercomputing.

BHV-1400: Next-Generation TRAP Degrader for IgA Nephropathy (IgAN)

Biohaven’s highly selective next-generation TRAP degraders target a specific disease-causing protein for degradation after removal from the circulation. The company’s potential first-in-class TRAP degrader, BHV-1400, brings precision immunology to the treatment landscape of IgA nephropathy the most common primary glomerulonephritis worldwide. IgAN is caused by the excess production of galactose-deficient IgA1 (Gd-IgA1) which is bound by autoantibodies and forms complexes that deposit in the glomerular mesangium triggering a profibrotic and proinflammatory cascade, ultimately leading to renal failure.

The first clinical experience with BHV-1400 underscores its paradigm-shifting potential to selectively remove only aberrant Gd-IgA1, the disease-causing species in IgA nephropathy, while sparing normal IgA. First-in-patient dosing with BHV-1400, a next-generation TRAP degrader, achieved selective lowering of Gd-1gA1 within hours, translating to hematuria resolution, deep reductions in proteinuria, and eGFR improvements within weeks of dosing.

The first patient dosed, a young woman with early disease, normal baseline eGFR and chronic hematuria experienced rapid reductions in Gd-IgA1 associated with complete resolution of chronic hematuria and improvement in symptoms of fatigue within weeks of dosing. The second patient dosed, a man with late-moderate-to-severe loss of kidney function as evidenced by decreased baseline eGFR, experienced rapid reductions in Gd-IgA1 associated with robust reductions in proteinuria of greater than 60% and improvement in kidney function as measured by a 24% increase in GFR within weeks of dosing. Importantly, both patients showed no significant reductions in normal IgA, IgG, or IgM. Additional IgAN patients are being treated in the expansion cohort and plans are underway for initiating a pivotal trial of BHV-1400 in 2026.

In the ongoing Phase 1, BHV-1400 achieved rapid lowering of Gd-IgA1 with a mean reduction of >60% within hours. Maximum reduction exceeding 80% was observed following the first dose. This selective and rapid approach to immunoglobulin lowering represents a second-generation therapeutic approach to IgAN, potentially allowing for faster, meaningful disease control with less acute or long-term safety risks associated with complement inhibition or broad antibody suppression. As in healthy participants, BHV-1400 has been safe and well-tolerated in initial patients with IgAN. As of January 2026, there have been no drug-related adverse events in the Phase 1b expansion cohort.

BHV-1400 Directly Targets the Disease-Driver of IgA Nephropathy

BHV-1400 Early Disease Clinical Experience Complete Resolution of Hematuria Within Weeks of Dosing

BHV-1400 Advanced Disease Patient Rapid Improvement in Proteinuria and Increase in eGFR Within Weeks of Dosing

BHV-1400 Single Patient Data Highlights Rapid Proteinuria Reduction Where Competitor Requires Months.

BHV 1300: MoDE Degrader with Potential to Transform Care Across Multiple IgG Mediated Diseases

BHV-1300, Biohaven’s lead MoDE degrader, is currently in development for Graves’ disease, an IgG1-mediated autoimmune disease caused by autoantibodies that hyperstimulate the TSH receptor, causing hyperthyroidism and requiring surgical removal or chemical ablation of the thyroid or the chronic administration of anti-thyroid drugs. BHV-1300 has shown the potential for best-in-class reductions of IgG, with maximum reductions up to an 87% decrease from baseline within weeks of dosing and has been safe and well-tolerated in Phase 1 clinical studies.

Results from the first-in patient clinical experience with BHV-1300 also showed that it induced complete suppression of pathogenic TSH receptor-stimulating antibodies with normalization of previously elevated thyroid hormones within weeks after dosing. There were no clinically significant reductions in IgG3, IgA, IgE, IgM, or albumin, nor increases in cholesterol compared to baseline. Clinical findings to date position BHV-1300 for pivotal study initiation in Graves’ disease in 2026, as well as additional follow-on studies in other autoimmune diseases where its therapeutic pan-IgG depletion capability is expected to have significant potential.

Biohaven IgG Degrader Targets the Root Cause of a Broad Autoimmune Disease To Treat and Prevent Multi-Organ Complications

BHV-1300 First Graves’ Patient Dosed — Pathogenic Antibody Levels Undetectable and Thyroid Hormones Normalized Within First Month

Biohaven Discovery Engine for Next-Generation Degraders

With proof-of-concept now established in patients for the first clinically validated extracellular protein degrader platform, Biohaven’s discovery engine is prepared to advance next-generation MoDE and TRAP degraders as lead programs, including:

BHV-1420, a PLA2R autoantibody specific TRAP degrader, for membranous nephropathy;
BHV-1450, a IgG4 specific MODE degrader, for potential indications including pemphigus vulgaris and myasthenia gravis with anti-MuSK antibodies;
BHV-1440, a TSHR autoantibody specific TRAP degrader, as the next-generation of immune therapy for Graves’ disease and thyroid eye disease;
BHV-6500, a proinsulin and insulin autoantibody TRAP degrader, for type 1 diabetes;
BHV-1490, an IgM MoDE degrader for cryoglobulinemia, Waldenstrom’s macroglobulinemia and IgM neuropathy;
BHV-1310, a next generation IgG MoDE degrader, for management of rare IgG-mediated indications;
BHV-1600, a beta-1 adrenergic receptor autoantibody degrader, for cardiomyopathy; and,
Multiple undisclosed degrader targets in early discovery development.
Epilepsy Program: Revolutionizing the Treatment of Epilepsy with a Modern Kv7 Activator

Opakalim is a next-generation, selective Kv7 activator, targeting a clinically validated mechanism of action for the treatment of epilepsy. It is highly differentiated from ezogabine, a first-generation non-selective Kv7 activator previously approved for the treatment of focal seizures, and ezogabine analogs in development. Importantly, opakalim does not exhibit the GABA receptor activity seen with ezogabine and other antiseizure medicines (ASMs), which contribute to central nervous system (CNS) adverse effects and their poor tolerability in the clinic. Opakalim is being developed to address the significant unmet medical need that exists for novel ASMs having a favorable tolerability profile, especially those with mechanisms of action that are complementary to currently available ASMs.

Biohaven is currently conducting two Phase 2/3 randomized, double-blind, placebo-controlled studies (NCT06132893 and NCT06309966) comparing the efficacy of opakalim to placebo as an adjunctive therapy for refractory focal onset epilepsy, as well as an open-label extension (OLE) study (NCT06443463) 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 compelling efficacy and to deliver a highly 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.

These preliminary observations support the paradigm-shifting potential of opakalim to achieve antiseizure efficacy with a highly favorable and differentiated tolerability profile compared to other approved ASMs and those in development.

Pivotal topline results for opakalim in the treatment of focal epilepsy are expected in 2026.
Opakalim Offers Potential Best-in-Clinic Kv7-Activator Profile

Obesity Program: Targeting High Quality Weight Loss

Taldefgrobep alfa is Biohaven’s novel inhibitor of the myostatin-activin signaling pathway with the potential to achieve high quality weight loss in people living with obesity.

Biohaven initiated a taldefgrobep Phase 2 proof-of-concept study in obesity during the fourth quarter of 2025 (NCT07281495). This randomized, double-blind, placebo-controlled, 24-week, dose-ranging study is evaluating the efficacy and tolerability of once-weekly and once-monthly taldefgrobep as monotherapy, via self-administered autoinjector, in adults living with overweight and obesity. Approximately 150 participants will be enrolled. The primary outcome measure is percent change in total body weight from baseline to Week 24.

Taldefgrobep’s differentiated mode of action targets fat reduction, builds muscle, and increases bone density.

In the clinic, taldefgrobep has demonstrated beneficial changes in fat mass and lean mass in non-obese populations, including healthy adult participants in a Phase 1 study. Participants who received taldefgrobep once-weekly realized significant reductions in total body fat mass (>6%) and increases in lean muscle mass (up to 4%) after one month of dosing. Notably, these body composition parameters continued to demonstrate additional improvements after cessation of dosing associated with the persistence of the pharmacologically active taldefgrobep-myostatin complex and suggesting the drug may support extended dosing intervals. Recent nonclinical data demonstrates that this complex cam also potently inhibit the Activin E-ALK7 signaling axis within adipocytes, further underpinning the complementary mechanistic advantages of taldefgrobep in both growing muscle and reducing fat.

Taldefgrobep has an established safety profile that is well-suited for an indication in overweight and obesity. It has been previously evaluated in >700 clinical trial participants across 5 completed studies and an ongoing Phase 3 trial in spinal muscular atrophy. Throughout its clinical development, taldefgrobep has been well tolerated, with rates of muscle and gastrointestinal adverse events comparable to placebo.

Phase 2 topline results for taldefgrobep in the treatment of obesity are expected in 2026.
Taldefgrobep Monotherapy Dose-Ranging (Q1W and Q4W) Study Initiated

Advancement of Key Next Generation ADC Oncology Programs

BHV-1510 (Trop2 ADC): Preliminary Phase 1 results for BHV-1510 showed encouraging clinical activity with confirmed responses and a differentiated safety profile demonstrating clinical proof of principle with Biohaven’s novel topoisomerase 1 (TopoIx) payload. In a pretreated population of participants with advanced/metastatic cancer, the majority with prior PD-(L)1 treatment, BHV-1510 2.5 mg/kg IV Q3W plus cemiplimab resulted in a confirmed objective response rate of 72.7% (8/11), including 3/5 (60%) in NSCLC, 4/4 (100%) in endometrial cancer, and 1/2 (50%) in urothelial cancer. BHV-1510 demonstrated low rates of adverse events consistent with very low levels of unconjugated payload (such as hematological toxicities and diarrhea), and there were no cases of interstitial lung disease, thus indicating a differentiated safety profile of BHV-1510 from other Trop2 ADCs. The most frequent toxicity was oral mucositis/stomatitis, a known class effect that is manageable with supportive care. Based on these data, an expansion cohort has initiated in endometrial cancer for the combination of BHV-1510 plus cemiplimab.

Biohaven also announced the successful initiation of subcutaneous administration with BHV-1510, as the first and only Trop2 ADC in clinic with the potential for subcutaneous delivery. In the first patients, early data showed bioavailability in the predicted range with tumor shrinkage in first patient dosed. Subcutaneous delivery may offer several advantages over intravenous infusion, including a more patient-friendly experience and rapid administration (minutes vs hours). In addition, subcutaneous dosing may provide an optimized pharmacokinetic profile with lower Cmax and more stable exposure, which may support improved safety and efficacy.

BHV-1530 (FGFR3 ADC): Biohaven also provides an update of the ongoing first-in-human clinical trial of BHV-1530, a potential first-in-class FGFR3-directed ADC that also leverages the proprietary TopoIx payload. In the initial cohorts, no dose-limiting toxicities were observed, and no safety signals have emerged. Early tumor reduction was seen in a patient with an FGFR3 mutation as dosing enters the predicted efficacious range. The pharmacokinetic profile indicates a highly stable ADC, consistent with Biohaven’s next-generation platform technology. FGFR3 is a promising therapeutic target in patients with genetic alterations or overexpression including urothelial cancer and may also be relevant across other solid tumors with high unmet need.

Encouraging Early Clinical Activity and High Response Rates of BHV-1510 + Cemiplimab

Progress across portfolio of clinical and next-generation discovery programs:

Patient enrollment continues in pivotal Phase 2/3 clinical trial in early Parkinson’s disease with brain-penetrant TYK2/JAK1 inhibitor, BHV 8000.

Biohaven Discovery Engine: Next wave of innovative, therapeutics primed to advance forward at an intentional cadence.

Discovery has advanced multiple novel development candidates for future clinical development. Beyond the degrader platform, these include:
BHV-1955 targeting the oxytocin receptor centrally for the treatment of tinnitus;
BHV-2120, oral small molecule TRPM3 inhibitor for epilepsy
BHV-8555, a brain penetrant, oral small molecule preventing a-synuclein aggregation in Parkinson’s disease; and
BHV-8100, a brain-penetrant, oral small molecule activating the M2 isoform of pyruvate kinase (PKM2) for neurodegenerative disorders and aging.

(Press release, Biohaven Pharmaceutical, JAN 12, 2026, View Source [SID1234661979])