Biohaven Showcases Innovative Neuroscience Portfolio with 20 Presentations at the 2024 American Academy of Neurology (AAN) Annual Meeting and Provides Other Business Updates

  • Biohaven’s first-in-class Molecular Degrader of Extracellular Proteins (MoDE™) technology targeting IgG removal, BHV-1300, was selected for an oral presentation at AAN highlighting its novel mechanism of action and the latest preclinical data demonstrating rapid, robust, and selective target removal
    • Biohaven announces it will present safety and IgG lowering data from its ongoing single ascending dose (SAD) study of BHV-1300 at the company’s annual R&D day at the Yale Innovation Summit in New Haven, Connecticut on May 29, 2024
  • Safety, tolerability, and target engagement data showing the differentiated profile of BHV-7000 as a potential best-in-class Kv7 activator will be presented as both oral and poster presentations; the Phase 2/3 focal epilepsy program is now enrolling patients
  • AAN Abstract of Distinction awarded to BHV-2100, which demonstrated potent reversal of pain in preclinical models and favorable initial safety and pharmacokinetic data in Phase 1 studies, highlighting the potential for TRPM3 antagonism as a novel nonopioid target to treat pain and migraine
  • Oral and poster presentations of troriluzole as a novel glutamate modulating agent with favorable pharmacokinetics, safety, and outcomes data in the potential treatment of neurologic and neuropsychiatric disorders
    • Biohaven announces the Data Monitoring Committee for its Phase 3 trial in obsessive-compulsive disorder convened in the second quarter to review the interim analysis and informed the Company that the study may continue
  • Additional efficacy and safety data regarding troriluzole in spinocerebellar ataxia will also be presented in both oral and poster format
  • Poster presentation of RESILIENT Phase 3 study design with taldefgrobep alfa in spinal muscular atrophy; study completed enrollment and topline results anticipated in the second half of 2024
    • Biohaven announces the FDA granted “rare pediatric disease” designation for taldefgrobep alfa thereby providing the potential to receive a priority review voucher (PRV) if ultimately approved for the indication of spinal muscular atrophy
  • Other presentations and posters include development of the PARCOMS, a new outcome measure for Parkinson’s disease clinical trials, application of machine learning in video assessment of gait, and data demonstrating a reduced risk for Parkinson’s disease in patients taking anti-inflammatory agents
  • Three oral presentations and 6 poster presentations from the Biohaven Clinical and Health Outcomes group describe the development and validation of novel scales and natural history studies in support of troriluzole and BHV-8000 development programs, the impact of immune-modulating therapy in Parkinson’s disease, the burden of illness in focal epilepsy and KCNQ2-DEE (BHV-7000), and the application of AI machine learning technology to quantify patient mobility with troriluzole in spinocerebellar ataxia

DENVER, CO and NEW HAVEN, Conn., April 13, 2024 /PRNewswire/ — Biohaven Ltd. (NYSE: BHVN) announced today that 20 abstracts, including 8 oral presentations and 12 posters, will be featured this weekend starting April 13th at the 2024 American Academy of Neurology (AAN) Annual Meeting, taking place in Denver, Colorado. The presentations highlight Biohaven’s leadership in neuroscience and extensive development programs evaluating novel therapies to treat neurological diseases, with abstracts covering programs that include Kv7 ion channel modulation, molecular degraders of extracellular protein (MoDEs), TRPM3 antagonism, TYK2/JAK1 inhibition, glutamate modulation, and myostatin inhibition.

Irfan Qureshi, M.D., Chief Medical Officer of Biohaven, commented, “The research being presented at the AAN Annual Meeting emphasizes Biohaven’s commitment to developing new therapeutic options across a range of neurological diseases. By targeting novel mechanisms of action, differentiated from currently available treatments and other therapies in development, and following innovative science, Biohaven continues to strive for better treatments for people living with neurological disorders. We are particularly honored that the AAN Science Committee selected our BHV-2100 (TRPM3) abstract as an AAN Abstract of Distinction, recognizing it as the top abstract in the pain category. Following the completion of Phase 1 studies in the first half of 2024, we look forward to initiating a Phase 2 study with BHV-2100 in migraine in the second half of the year and are excited by the potential for this novel nonopioid approach to treat pain.”

Vlad Coric M.D., Chief Executive Officer and Chairman of Biohaven, added, “Our leadership in neuroscience research is on full display at the AAN Annual Meeting with the breadth and depth of clinical, epidemiological, and preclinical programs highlighted in our scientific presentations. Central nervous system (CNS) disorders continue to represent one of the highest unmet medical needs facing our society and we must act urgently to bring better treatments to patients and improve clinical outcomes. We believe that the next generation of therapies for CNS disorders will include MoDEs for autoimmune disorders, ion channel modulation for epilepsy, migraine and other pain disorders, immune modulation for neurodegenerative disorders including Parkinson’s and Alzheimer’s diseases, myostatin targeting drugs for neuromuscular disorders and glutamate modulating agents for neuropsychiatric disorders. I am so proud of the team at Biohaven who are working tirelessly to alleviate the burden of these devastating disorders.”

The complete list of Biohaven’s accepted abstract titles is below. Full abstracts can be viewed online at https://index.mirasmart.com/AAN2024/

Oral Presentations:

  • Novel Bispecific Degrader BHV-1300 Achieves Rapid, Robust, and Selective IgG Reduction in Preclinical Models Including Nonhuman Primates
  • BHV-2100, a First-in-class TRPM3 Antagonist for the Treatment of Pain (AAN Abstract of Distinction)
  • Characterization of BHV-7000: A Novel Kv7.2/7.3 Activator for the Treatment of Seizures
  • Association of Anti-inflammatory Therapy Use with the Incidence of Parkinson’s Disease: A Person-Time Analysis Among Patients with Autoimmune Diseases
  • Re-weighting MDS-UPDRS Motor Items for Optimal Sensitivity to Parkinson’s Disease Progression in Untreated Patients Using Parkinson’s Progression Markers Initiative Data
  • Population Pharmacokinetic Modeling of Riluzole After Administration of a Next Generation Prodrug Troriluzole
  • Next Generation Prodrug Troriluzole: Increased Bioavailability of Riluzole with No Food Effect in Healthy Subjects
  • Matching-adjusted Indirect Comparison of Troriluzole Versus Untreated Natural History Cohort in Spinocerebellar Ataxia

Poster Presentations:

  • Phase 1 Study Evaluating the Safety and Tolerability of BHV-7000, a Novel, Selective Kv7.2/7.3 Potassium Channel Activator, in Healthy Adults
  • Novel, Selective Kv7.2/7.3 Potassium Channel Activator, BHV-7000, Demonstrates Dose-dependent Pharmacodynamic Effects on EEG Parameters in Healthy Adults
  • Determinants of Health-related Quality of Life of Patients with Focal Epilepsy: A Systematic Literature Review
  • Functional Impairments in Patients with KCNQ2-DEE: Associations Among Key Clinical Features
  • The Phase 3 RESILIENT Study: Taldefgrobep Alfa in Spinal Muscular Atrophy
  • Re-weighting MDS-UPDRS Part II Items for Optimal Sensitivity to Parkinson’s Disease Progression Using Parkinson’s Progression Markers Initiative Natural History Data
  • Safety, Tolerability, and Pharmacokinetics of Single and Multiple Rising Doses of a Next Generation Prodrug Troriluzole in Healthy Subjects
  • Troriluzole Exhibits Favorable Hepatic Safety Profile Across a Diverse Range of Disorders
  • No Clinically Relevant Effects of Hepatic Impairment on the Pharmacokinetics of a Next Generation Prodrug Troriluzole
  • Automated Video-based Characterization of Movement Quality in a Phase III Clinical Trial of Troriluzole in Subjects with Spinocerebellar Ataxia
  • Psychometric Validation of the Modified-functional Scale for the Assessment and Rating of Ataxia
  • Development of a Novel Composite Measure (SCACOMS) to Assess Disease Progression in Spinocerebellar Ataxia

Posters and presentations will be available on the Posters and Presentations page after the conference at www.biohaven.com.

About Biohaven

Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven’s extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; and antibody recruiting, bispecific molecules and antibody drug conjugates for cancer.

Forward-looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including “continue”, “plan”, “will”, “believe”, “may”, “expect”, “anticipate” and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven’s planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable U.S. regulatory requirements; the potential commercialization of Biohaven’s product candidates; the potential for Biohaven’s product candidates to be first in class therapies; and the effectiveness and safety of Biohaven’s product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven’s filings with the Securities and Exchange Commission, including within the sections titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations”. The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Contact:

Jennifer Porcelli

Vice President, Investor Relations

[email protected]

201-248-0741

Media Contact:

Mike Beyer

Sam Brown Inc.

[email protected]

312-961-2502

MoDE is a trademark of Biohaven Therapeutics Ltd.

Biohaven AAN 2024 Oral & Poster Presentations:

Functional Impairments in Patients with KCNQ2-DEE: Associations Among Key Clinical Features

Sunday 4/14/24: 8:00-9:00

P1- Poster Session 1

Colorado Convention Center – Exhibit Hall B-E

The Phase 3 RESILIENT Study: Taldefgrobep Alfa in Spinal Muscular Atrophy

Sunday 4/14/24: 11:45 – 12:45

P2 – Poster Session 2

Colorado Convention Center – Exhibit Hall

Association of Anti-inflammatory Therapy Use with the Incidence of Parkinson’s Disease: A Person-Time Analysis Among Patients with Autoimmune Diseases

Sunday 4/14/24: 1:00-3:00 (1:24-1:36)

S2 – Movement Disorders: Epidemiology and Clinical Aspects

Colorado Convention Center- Mile High 4CD

Re-weighting MDS-UPDRS Motor Items for Optimal Sensitivity to Parkinson’s Disease Progression in Untreated Patients Using Parkinson’s Progression Markers Initiative Data

Sunday 4/14/24: 1:00-3:00 (1:36-1:48)

S2 – Movement Disorders: Epidemiology and Clinical Aspects

Colorado Convention Center- Mile High 4CD

Population Pharmacokinetic Modeling of Riluzole After Administration of a Next Generation Prodrug Troriluzole

Sunday 4/14/24: 1:00 – 3:00 (2:12-2:24)

S3 – General Neurology 1

Colorado Convention Center – Four Seasons 2/3

Next Generation Prodrug Troriluzole: Increased Bioavailability of Riluzole with No Food Effect in Healthy Subjects

Sunday 4/14/24: 3:30 – 5:30 (4:30-4:42)

S5 – ALS and CMT: New Therapeutic Approaches

Colorado Convention Center – Four Seasons 1

BHV-2100, A First-In-Class TRPM3 Antagonist for the Treatment of Pain

Monday 4/15/24: 11:15-12:15 (11:27-11:39)

S13 – Pain Research

Colorado Convention Center 605

Troriluzole Exhibits Favorable Hepatic Safety Profile Across a Diverse Range of Disorders

Monday 4/15/24: 11:45 – 12:45

P4 – Poster Session 4

Colorado Convention Center – Exhibit Hall

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Rising Doses of a Next Generation Prodrug Troriluzole in Healthy Subjects

Monday 4/15/24: 11:45 – 12:45

P4 – Poster Session 4

Colorado Convention Center – Exhibit Hall

No Clinically Relevant Effects of Hepatic Impairment on the Pharmacokinetics of a Next Generation Prodrug Troriluzole

Monday 4/15/24: 11:45 – 12:45

P4 – Poster Session

Colorado Convention Center – Exhibit Hall

Automated Video-based Characterization of Movement Quality in a Phase III Clinical Trial of Troriluzole in Subjects with Spinocerebellar Ataxia

Tuesday 4/16/24: 8:00-9:00

P6- Poster Session 6

Colorado Convention Center – Exhibit Hall

Psychometric Validation of the Modified-functional Scale for the Assessment and Rating of Ataxias

Tuesday 4/16/24: 11:45-12:45

P7 – Poster Session 7

Colorado Convention Center – Exhibit Hall

Development of a Novel Composite Measure (SCACOMS) to Assess Disease Progression in Spinocerebellar Ataxia

Tuesday 4/16/24: 11:45-12:45

P7 – Poster Session 7

Colorado Convention Center – Exhibit Hall

Phase 1 Study Evaluating the Safety and Tolerability of BHV-7000, a Novel, Selective Kv7.2/7.3 Potassium Channel Activator, in Healthy Adults

Tuesday 4/16/24: 5:30 – 6:30

P8 – Poster Session 8

Colorado Convention Center – Exhibit Hall

Novel, Selective Kv7.2/7.3 Potassium Channel Activator, BHV-7000, Demonstrates Dose-dependent Pharmacodynamic Effects on EEG Parameters in Healthy Adults

Tuesday 4/16/24: 5:30 – 6:30

P8 – Poster Session 8

Colorado Convention Center – Exhibit Hall

Determinants of Health-related Quality of Life of Patients with Focal Epilepsy: A Systematic Literature Review

Wednesday 4/17/24: 8:00-9:00

P9- Poster Session 9

Colorado Convention Center – Exhibit Hall

Characterization of BHV-7000: A Novel Kv7/2/7.3 Activator for the Treatment of Seizures

Wednesday 4/17/24: 1:00-3:00 (2:00-2:12)

S29- Epilepsy Diagnostics and Therapeutics

Colorado Convention Center 605

Matching-adjusted Indirect Comparison of Troriluzole Versus Untreated Natural History Cohort in Spinocerebellar Ataxia

Wednesday 4/17/24: 3:30-5:30 (5:06-5:18)

S35 – Movement Disorders: Hyperkinetic Movement Disorders

Colorado Convention Center – Four Seasons 4

Re-weighting MDS-UPDRS Part II Items for Optimal Sensitivity to Parkinson’s Disease Progression Using Parkinson’s Progression Markers Initiative Natural History Data

Wednesday 4/17/24: 5:30-6:30

P11 – Poster Session 11

Colorado Convention Center – Exhibit Hall

Novel Bispecific Degrader BHV-1300 Achieves Rapid, Robust, and Selective IgG Reduction in Preclinical Models Including Nonhuman Primates

Thursday 4/18/24: 1:00 – 3:00 (1:36-1:48)

S43 – General Neurology 2

Colorado Convention Center – 108/110/112

 

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