Biohaven Pharmaceutical Holding Co Ltd

Biohaven Pharmaceutical — Leerink Fireside Chat June 2021

23 slides from Biohaven Pharmaceutical's conference presentation, Jun 29, 2021

  1. Slide 1: Leerink Fireside Chat | June 2021

    NYSE:BHVN Ellie, living with migraine © 2021 Biohaven Pharmaceuticals. All rights reserved. Leerink Fireside Chat | June 2021 neuroinnovation Exhibit 99.1

  2. Slide 2: Disclaimer

    Disclaimer This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including: statements about Biohaven Pharmaceutical Holding Company Ltd. (The ”Company”) and our plans relating to the commercialization and sales of NURTEC® O

  3. Slide 3: Biohaven's Leading CGRP-Signaling Franchise

    Biohaven’s Leading CGRP-Signaling Franchise to Treat: Migraine, Neuro-inflammatory and Neuro-immune Disorders BIOHAVEN INVESTOR PRESENTATION 3 Nurtec® ODT Rapid Dissolving Zavegepant Next-GEN CGRPs Intranasal Oral Launched 1Q20 2nd Ph 3 trial underway 4Q20 Ph 3 prevention trial started 1Q21 Multiple

  4. Slide 4: Nurtec ODT: First and Only Medication Proven to Treat & Prevent Migraine

    Dissolving the line between acute and preventive treatment of migraine1-3 ACUTE Treatment PREVENTIVE Treatment The maximum dose in a 24-hour period is 75 mg.1 Up to 18 doses of Nurtec ODT can be taken per month.1 1. Nurtec ODT. Package insert. Biohaven Pharmaceuticals Inc. 2. Croop R et al. Lancet.

  5. Slide 5: June 2021

    JUNE 2021 BIOHAVEN INVESTOR PRESENTATION 5

  6. Slide 6: Oral CGRP Class Continues to Show Robust Market Growth

    3/ 13 3/ 27 4/ 10 4/ 24 5/ 8 5/ 22 6/ 5 6/ 19 7/ 3 7/ 17 7/ 31 8/ 14 8/ 28 9/ 11 9/ 25 10 /9 10 /2 3 11 /6 11 /2 0 12 /4 12 /1 8 1/ 1 1/ 15 1/ 29 2/ 12 2/ 26 3/ 12 3/ 26 4/ 9 4/ 23 5/ 7 5/ 21 6/ 4 6/ 18 Oral CGRP Class Continues to Show Robust Market Growth: Nurtec leads in NBRx share at 51.4% BIOHA

  7. Slide 7: Near-Term Milestones Beyond Migraine

    Near-Term Milestones Beyond Migraine BIOHAVEN INVESTOR PRESENTATION 7 Migraine Acute Treatment APPROVED FDA Approved May 27, 2021 Multiple system atrophy Amyotrophic lateral sclerosis Spinocerebellar ataxia Top-line 3Q21 Complete enrollment 4Q21 Top-line 4Q21-1Q22 JUNE 2021 Migraine Preventive Tx AP

  8. Slide 8: Orphan Disease Opportunities

    Orphan Disease Opportunities BIOHAVEN INVESTOR PRESENTATION 8 Multiple System Atrophy Amyotrophic Lateral Sclerosis Spinocerebellar Ataxia 3 Devastating Diseases 3 Phase 3 Readouts Over Next Year Targeting 3 Global Orphan Drug Approvals 2022/2023 JUNE 2021

  9. Slide 9: Multiple System Atrophy (MSA) is a Rare Disease with High Unmet Need

    • Neurodegenerative disease that presents like Parkinson’s but is rapidly progressive and fatal • Prevalence: 2–5 per 100,000 • Clinical symptoms • Parkinsonism: characteristic tremor (not responsive to L-DOPA), rigidity, dysarthria, falls • Cerebellar ataxia • Autonomic failure: orthostatic hypoten

  10. Slide 10: Verdiperstat (BHV-3241) is a Myeloperoxidase Inhibitor

    • Myeloperoxidase (MPO) enzyme • Key mediator of oxidative and inflammatory processes that lead to neurodegeneration • Promotes alpha-synuclein aggregation • Increased in human MSA brains in areas of neurodegeneration1 • Verdiperstat • Potent, first-in-class, brain-penetrant MPO inhibitor • Develope

  11. Slide 11: Verdiperstat Clinical Experience

    • Studied in approximately 250 subjects (healthy volunteers, Parkinson’s disease, MSA) • Generally safe and well tolerated • Demonstrated target engagement (↓ blood MPO activity) • Reduced neuroinflammation (microglial activation) in Parkinson’s disease • Positron Emission Tomography imaging showed

  12. Slide 12: Verdiperstat Phase 2 Study in MSA Completed by AstraZeneca

    • Phase 2 study: randomized double-blind controlled trial • N=61 subjects (MSA-C, 34; MSA-P, 24) • Randomized to 12 weeks treatment • Placebo BID vs Verdiperstat 300 mg BID vs Verdiperstat 600 mg BID • Outcome measures: UMSARS*, PET, safety (labs, AE’s, ECGs, vitals) • Generally safe and well tolera

  13. Slide 13: Verdiperstat Phase 3 Study in MSA is Ongoing, Topline Data Expected 3Q21

    DESIGN • Sample size: 336 subjects • Randomization: 1:1 • Dose: Verdiperstat 600 mg BID vs. Placebo • Primary outcome measure: modified Unified MSA Rating Scale (modified with FDA guidance) • Sites: US, EU (UK, FR, GER, AUS, IT) STATUS • Study initiated Jul 2019 (US), Feb 2020 (EU) • LPFV: Jul 2020

  14. Slide 14: Verdiperstat Selected for First-Ever Platform Trial in ALS

    BIOHAVEN INVESTOR PRESENTATION 14 Verdiperstat Selected for First-Ever Platform Trial in ALS FDG, MAY 2021JUNE

  15. Slide 15: Rationale for Studying Verdiperstat in ALS

    Rationale for Studying Verdiperstat in ALS BIOHAVEN INVESTOR PRESENTATION 15 • Targets well accepted ALS disease mechanisms (oxidative stress / nitrosative stress, microglial activation / neuroinflammation) in a physiologically relevant manner • MPO may also play a role in increasingly recognized AL

  16. Slide 16: Neuroinflammation in ALS Indicated by [11C]-PBR28 TSPO Imaging

    Neuroinflammation in ALS Indicated by [11C]-PBR28 TSPO Imaging BIOHAVEN INVESTOR PRESENTATION 16 TSPO, translocator protein - positron emission tomography Increased in ALS vs. controls Co-Localizes with Cortical Thinning Correlates with ALS Disease Severity Alshikho MJ, et al. Ann Neurol. 2018 Jun;8

  17. Slide 17: Verdiperstat Phase 3 Healey ALS Platform Trial Ongoing

    • DESIGN • Sample size: 160 subjects • Randomization: 3:1 • Dose: 600 mg BID vs. Placebo • Primary outcome measure: ALS Functional Rating Scale – Revised (ALS-FRS-R) • Sites: 50 sites (North East ALS Consortium) • STATUS • Enrollment expected to complete by 4Q21 • COLLABORATOR: Verdiperstat Phase 3

  18. Slide 18: Cerebellar Ataxias

    Cerebellar Ataxias • Characterized by: • Poor balance with falls • Dysarthria / dysphagia • Incoordination of limbs • Cognitive impairment • Postural or kinetic tremor • Oculomotor dysfunction • Spinocerebellar ataxia (SCA; >40 subtypes) • Affects 1–5.6 per 100,000 (estimated 3,200–18,000 in US) • N

  19. Slide 19: Troriluzole Study in Spinocerebellar Ataxia (SCA) vs Natural History Cohort

    • Post-hoc analysis of patients enrolled in long-term extension of Phase 2b/3 troriluzole SCA trial • Primary efficacy endpoint: change from baseline in the Total SARA Score after 48 weeks • Patients from BHV4157-201 trial versus eligibility criteria matched Ashizawa Natural History cohort: • SCA Ge

  20. Slide 20: Troriluzole Study BHV4157-206 Randomized Controlled Trial in SCA

    Key Entry Criteria • SCA genotypes (SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA10) Design • Sample size: 230 subjects • Randomization: 1:1 • Stratification: by SCA genotype • Dose: Troriluzole 200 mg QD vs. Placebo QD • Primary Outcome: Modified SARA Scale • FDA aligned outcome measure Status • Phase 3

  21. Slide 21: Biohaven 2021 Growth Drivers

    Biohaven 2021 Growth Drivers BIOHAVEN INVESTOR PRESENTATION 21 Nurtec (Rimegepant) • Treating migraine continuum • Single simple clear dosing with ODT • Significant lifecycle expansion Zavegepant • Intranasal ultrarapid formulation • Oral formulation for chronic migraine • Multiple additional nonmig

  22. Slide 22: Biohaven Labs: Next-Generation Bispecific Platforms for Pipeline Growth

    Biohaven Labs: Chemistry and Discovery Next-Generation Bispecific Platforms for Pipeline Growth BIOHAVEN INVESTOR PRESENTATION 22 Binds liver (ASGPR) Binds protein target Bifunctional small molecule Target Binder Reactive group Directing group Binding to IgG1/2/4 antibody Conjugation and release of

  23. Slide 23: Thank You!

    THANK YOU! www.biohavenpharma.com NYSE:BHVN

Biohaven Pharmaceutical Holding — Leerink Fireside Chat June 2021 on 6ix