Biohaven Pharmaceutical Holding Co Ltd

Biohaven Pharmaceutical — Investor Presentation (June 2020)

76 slides from Biohaven Pharmaceutical's investor presentation, Jun 2, 2020

  1. Slide 1: Investor Presentation June 2020

    Exhibit 99.1 neuroinnovation Meeting Patient Needs Growing Value INVESTOR PRESENTATION | JUNE 2020 NYSE: BHVN © 2020 Biohaven Pharmaceuticals Inc. All rights reserved.

  2. Slide 2: Disclaimer

    Disclaimer This presentation contains forward-looking statements within the meaning of “safe harbor” provisions of The Private Securities Litigation Reform Act of 1995, including: statements about Biohaven Pharmaceutical Holding Company Ltd. (The ”Company”) and our plans to develop and commercialize

  3. Slide 3: About Biohaven

    About Biohaven is a clinical-stage biopharmaceutical company, headquartered in New Haven, CT LISTED ON THE NEW YORK STOCK EXCHANGE (BHVN) PIPELINE INCLUDES PROVEN LEADERSHIP in industry and academic COMMITTED TO IMPROVING settings with a unique FULL OPERATIONS THE LIVES OF PATIENTS 6 development app

  4. Slide 4: Biohaven's Neuroinnovation Portfolio Late Stage Development Pipeline

    Biohaven’s Neuroinnovation Portfolio Late Stage Development Pipeline NURTEC™ ODT RIMEGEPANT RIMEGEPANT RIMEGEPANT VAZEGEPANT VAZEGEPANT CGRP FDA Approved Phase 3 Phase 3 Phase 3 Phase 2/3 Phase 3 antagonism Zydis® ODT Acute Migraine Acute Migraine Long-Term Safety Study Preventive Migraine Intranasa

  5. Slide 5: CGRP Receptor Antagonist Platform: Redefining the Treatment of Migraine

    CGRP Receptor Antagonist Platform: Redefining the Treatment of Migraine

  6. Slide 6: Migraine Significantly Impairs Patients' Lives

    Migraine Significantly Impairs Patients’ Lives Migraine affects ~15% of the US population1 with prevalence highest during peak employment years (25–55 years of age)3 1 in 5 women2 20,000 per 100,000 women 1 in 16 men2 6,250 per 100,000 men • Chronic neurological disease characterized by disabling at

  7. Slide 7: Migraine Attacks: A Leading Cause of Economic Burden in US

    Migraine Attacks: A Leading Cause of Economic Burden in US Annual total costs for migraine in the US are estimated at $27 BILLION1 ~25% OF ER VISITS FOR MIGRAINE were followed by a second ER visit for headache in <6 months2 In a study of over 850,000 patients with migraine, patients with 2 or more m

  8. Slide 8: Low Satisfaction With Current Acute Treatments and High Abandonment Rates

    Patients Report Low Satisfaction With Current Acute Treatments and High Abandonment Rates Across the Spectrum of Therapies ABANDONMENT RATES OF ACUTE MIGRAINE TREATMENTS1 PRESCRIPTION NSAIDs TRIPTANS ERGOT DERIVATIVES ANALGESICS 50% 45% 38% 35% § In a large survey, only 40% reported satisfaction wit

  9. Slide 9: Oral CGRP Receptor Antagonists Address Unmet Needs of Current Acute Treatments

    Oral CGRP Receptor Antagonists Address Unmet Needs of Current Acute Treatments Cardiovascular Medication Overuse Nonresponders Cannot tolerate Recurrence Contraindications Headache (MOH) Up to 55% of people do not 17% to 40% experience Overuse of many current have sufficient relief with 43% experien

  10. Slide 10: Now FDA Approved

    Now FDA Approved JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 10

  11. Slide 11: The Convenience of a Single Nurtec ODT Dose

    The Convenience of a Single Nurtec ODT 75 mg Dose The first and only CGRP receptor antagonist available in a quick-dissolve ODT1 Ready Set Go Nurtec ODT is removed from Placed under or on the tongue, it In clinical trials, some patients experienced pain the blister pack immediately dissolves within

  12. Slide 12: Nurtec ODT Blister Pack (8-count)

    Nurtec ODT Blister Pack (8-count) JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 12

  13. Slide 13: JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 13

    JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 13

  14. Slide 14: JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 14

    JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 14

  15. Slide 15: JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 15

    JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 15

  16. Slide 16: JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 16

    JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 16

  17. Slide 17: JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 17

    JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 17

  18. Slide 18: ICER Report Cost Effectiveness Quote

    “In our analysis of the cost effectiveness…we found that for patients for whom triptans are not effective, not tolerated, or are contraindicated…rimegepant…[is] cost effective at commonly used thresholds.” ICER Report Feb 25th 2020, Page ES18 Atlas S, Touchette D, Agboola F, Lee T, Chapman R, Pearso

  19. Slide 19: Rimegepant: Three Positive Phase 3 Trials, 1-Year Safety

    • Lipton, R. B., et al. (2019). "Rimegepant, an Oral Calcitonin Gene-Related Rimegepant: Three Peptide Receptor Antagonist, for Migraine." N Engl J Med 381(2): 142-149. • Croop, R., et al. (2019). "Efficacy, safety, and tolerability of rimegepant orally disintegrating tablet for the acute treatment

  20. Slide 20: Rimegepant Safety Exposure and Adverse Events

    2% 1,798 patients were exposed to No individual AE had an incidence greater than 2%1;3* 113,000 doses of rimegepant IMPORTANT SAFETY INFORMATION Contraindications: Hypersensitivity to Nurtec ODT or any of its components. Warnings and Precautions: If a serious hypersensitivity reaction occurs, discon

  21. Slide 21: Important Safety Information

    Important Safety Information INDICATION Nurtec™ ODT (rimegepant) is indicated for the acute treatment of migraine with or without aura in adults. Limitations of Use Nurtec ODT is not indicated for the preventive treatment of migraine. IMPORTANT SAFETY INFORMATION Contraindications: Hypersensitivity

  22. Slide 22: Rimegepant 1-Year Long-Term Safety Study Design

    Rimegepant 1-Year Long-Term Safety to Capture Real World Patient Usage Patients Received Free SCREENING/ TREATMENT PERIOD END of Treatment Access to Daily Supply1 BASELINE up to 52 Weeks or Early D/C Visit of Rimegepant for 1 year • Assess Long-Term Safety 30-day • Determine Patient Utilization Obse

  23. Slide 23: Median Monthly Usage Over 1-Year with PRN Dosing

    Median Monthly Usage: < 8 pills/month over 1-Year with PRN Dosing • PRN protocol allowed dosing up to once per calendar day Rimegepant 75 mg Tablets Treated Subjects Average rimegepant exposure [tablets per 4 weeks] PRN Dosing PRN Dosing 2–8 cohort {attack history/mo} 9–14 cohort {attack history/mo}

  24. Slide 24: Rimegepant Reduced Total Migraine Disability (MIDAS) by ~50%

    Rimegepant Reduced Total Migraine Disability (MIDAS) by ~50% in 1-Year Study Migraine Disability Assessment Score Severe 35 33 33.9 P<0.0001 versus baseline for all time points 31 Rimegepant demonstrated 29 improvements in mean 27 change from baseline by 25 ~50% for total MIDAS over the 23 Level of

  25. Slide 25: Rimegepant Reduces Lost Productivity Time by Approximately 50%

    Rimegepant 75 mg Up to Once Daily Reduces Lost Productivity Time by Approximately 50% Absenteeism Days Mean Item Score of missed work, 20 17.8 18 school, household, P<0.0001 versus baseline 16 Significant improvements social or leisure 16.2 for all time points activities due to 14 in absenteeism and

  26. Slide 26: Reduction in Moderate-to-Severe Migraine Days Irrespective of Baseline Frequency

    Rimegepant Shows Reduction in Moderate-to-Severe Migraine Days Irrespective of Baseline Migraine Frequency RIMEGEPANT LONG-TERM SAFETY – STUDY 201, INTERIM ANALYSIS 21FEB2019 o M 60% 48.4% 48.6% 50% 46.2% 48.4% of subjects in the EOD+PRN cohort 40% during month 3 achieved ≥50% reduction from baselin

  27. Slide 27: Positive Results in Pivotal Trial for Preventive Treatment of Migraine

    Biohaven Achieves Positive Results in Pivotal Trial of Oral NURTEC™, Dosed Every Other Day, for the Preventive Treatment of Migraine • Rimegepant 75 mg, dosed every other day, demonstrates statistically significant superiority, compared to placebo, on the primary endpoint of reduction in the mean nu

  28. Slide 28: Study BHV3000-305 Design

    Study BHV3000-305: Phase 3 Randomized Study Investigating the Safety and Efficacy of Rimegepant 75 mg for Preventive Treatment of Migraine Rimegepant 75 mg EOD Dosing Baseline Treatment Phase Observation Period Dosed Every Other Day (EOD) End of Study Visit 28 Day Up to 12 Weeks Rimegepant 75 mg (n=

  29. Slide 29: Rimegepant Met Primary Endpoint in Monthly Migraine Days

    Rimegepant Met Primary Endpoint – Showed Superiority to Placebo in Reduction of Monthly Migraine Days (MMD) with 75 mg Every Other Day (EOD) Dosing Placebo Rimegepant 75 mg Every Other Day Every Other Day N=347 N=348 -1 • Rimegepant 75 mg EOD treated patients demonstrated -2 4.3 day reduction from b

  30. Slide 30: Rimegepant EOD Met Key Secondary Endpoint

    Rimegepant EOD Met Key Secondary Endpoint – Superiority to Placebo in % Patients with ≥50% Reduction from Baseline in Moderate-Severe MMDs at 3 Months Placebo Rimegepant 75 mg Every Other Day Every Other Day N=347 N=348 a 0 -10 • 49.1% of rimegepant-treated subjects achieved ≥50% reduction, from bas

  31. Slide 31: Nurtec First CGRP Medication with Dual-Therapy Action

    Oral NURTEC™ is First CGRP Targeting Medication to Show “Dual-Therapy Action” with Both Acute and Preventive Treatment Benefits • Patients treated with oral NURTEC (rimegepant 75 mg), dosed every other day, demonstrated superiority over placebo on the primary endpoint of reduction in the mean number

  32. Slide 32: Commercial and Launch Readiness

    Commercial and Launch Readiness

  33. Slide 33: Nurtec ODT 'Surround Sound' Marketing Plan

    Nurtec™ ODT ‘Surround Sound’ Marketing Plan Core HCP Rep Promotion >500 Key Characteristics: Ø Competitive share of voice Consumer Omni-Channel Peer-2-Peer Ø Highly targeted (Awareness & Activation) (Engagement & Education) Ø Modern, ‘social first’ mindset HCP Omni-Channel (Awareness & Engagement) J

  34. Slide 34: Launch Payer Mix for CGRP Migraine Prevention Class

    Launch Payer Mix for CGRP Migraine Prevention Class (6 Months) Payment Type Commercial / Assistance 88.3% 89.3% 89.3% 87.1% Programs Medicare 6.0% 4.2% 5.2% 6.0% Cash 2.8% 3.7% 2.8% 3.7% Medicaid 2.6% 2.9% 2.7% 3.2% Totals 100% 100% 100% 100% *Ubrelvy data through 3 weeks of launch ending 2/7/20 JUN

  35. Slide 35: Managed Markets Customer Engagements

    Managed Markets Customer Engagements JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 3535

  36. Slide 36: Market Access Presentation Penetration

    Market Access Pie Deck/Clinical Presentation Penetration Completed / Scheduled Presentations All Targeted Accounts Total Lives % Total All Accounts Completed 275,488,361 84.6% Scheduled 36,624,214 11.3% 95.9% Pending 13,427,838 4.1% Total 325,540,383 100.0% A Priority Accounts Lives % Total “A” Acco

  37. Slide 37: Nurtec ODT Formulary Access Update: 120M Covered Lives

    Nurtec ODT Formulary Access Update: 120M Covered Lives 140 • Launch to date, 120M covered lives 120 with recent contracted formulary wins: • CVS 100 • Aetna 80 • HealthPartners • and key PBMs/health plans 60 • Medicaid access ahead of plan: Covered Lives (MM) Lives Covered 40 • Nurtec ODT with advan

  38. Slide 38: Established Treatments: Options for Acute Treatment of Migraine

    Established Treatments: Options for Acute Treatment of Migraine Triptans NSAIDs Opioids Butorphanol, morphine, Examples Sumatriptan, naratriptan, zolmitriptan1 Diclofenac, ketoprofen, flurbiprofen2,3 hydromorphone2,5 Migraine-specific Yes1 No3 No6 treatment? • Moderate or severe attacks OR2 Mild-to-

  39. Slide 39: REYVOW (Lasmiditan) Serotonin 1F Receptor Agonist

    REYVOW™ (Lasmiditan): Serotonin (5-HT) 1F Receptor Agonist Label Indication: Acute Treatment of Migraine DRIVING IMPAIRMENT CNS DEPRESSION, SEROTONIN SYNDROME Advise patients not to drive & MEDICATION OVERUSE HEADACHES Schedule V for 8 hours (detoxification may be necessary) 5.2 Central Nervous Syst

  40. Slide 40: Vazegepant: First Third-Generation Intranasal CGRP Receptor Antagonist

    Vazegepant: First and Only Third-Generation, Vazegepant Structural Intranasal CGRP Receptor Antagonist Diversity vs. Rimegepant FIRST INTRANASAL FORMULATION OF A SMALL MOLECULE CGRP RECEPTOR ANTAGONIST Vazegepant • Superior chemical attributes • Potent antagonist at the human CGRP receptor • Highly

  41. Slide 41: Vazegepant Early Separation From Placebo, Durable Through 24-48 Hours

    Vazegepant 10 mg and 20 mg Demonstrated Early Separation From Placebo at 15, 60 and 120 Minutes, Durable Through 24–48 Hours VAZEGEPANT PHASE 2/3 — STUDY BHV3500-201, 10MG & 20MG INTRANASAL Pain Relief to 2 Hours1 & Sustained Pain Relief 2-24/48 Hours2 Post-Single Dosing with Vazegepant Intranasal P

  42. Slide 42: Glutamate Platform

    GLUTAMATE PLATFORM Therapies for Neurologic and Neuropsychiatric Indications

  43. Slide 43: The Role of Glutamate: Present in 90% of Brain Synapses

    The Role of Glutamate: Present in 90% of Brain Synapses EXCITOTOXICITY NORMAL FUNCTION Diseased State Healthy State STRESS DEMENTIA RESILIENCE MEMORY AMYOTROPHIC LATERAL SCLEROSIS LEARNING SYNAPTO- DEPRESSION ANXIETY PLASTICITY NEURONAL ABNORMAL CELL GROWTH CONNECTIONS NEURO- STROKE MELANOMA TRANSMI

  44. Slide 44: Glutamate Mechanisms of Action in CNS

    Glutamate Mechanisms of Action in CNS 1 Glutamate Transporter Modulation • Troriluzole 2 Glutamate NMDA Receptor Antagonism • BHV-5000 1 Third-party clinical trials provide basis for exploration of troriluzole and BHV-5000 in 2 multiple neurologic and neuropsychiatric disorders JUNE 2020 BIOHAVEN IN

  45. Slide 45: Riluzole (Rilutek): Use and Limitations

    Riluzole (Rilutek®): Use and Limitations UNMET MEDICAL NEED IN ALS Riluzole (Rilutek®) is approved for the treatment of BENEFITS patients with amyotrophic lateral sclerosis (ALS) ü Mechanism of action well understood and proven to extend survival ü Neuroprotective, survival benefit in ALS ü Well tol

  46. Slide 46: Troriluzole: Rational Drug Discovery to Optimize Therapy

    Troriluzole: Rational Drug Discovery to Optimize Therapy • Improved absorption • Enhanced bioavailability • Reduced drug burden • Reduced first pass metabolism • Favorable safety profile • Once-daily dosing JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 46

  47. Slide 47: Peptide Transporter Enhances Absorption of Troriluzole

    Peptide Transporter 1 Enhances Absorption of Troriluzole ACTIVE ABSORPTION IN INTESTINAL TRACT BY PEPT1 PepT1 PepT1 = Peptide Transporter 1 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 47

  48. Slide 48: Troriluzole: Targeted Lead-Indication Development Strategy

    Troriluzole: Targeted Lead-Indication Development Strategy Neurodegenerative Obsessive-Compulsive Cerebellar Disorders & Related Disorders Disorders Lead indications Mild-to-Moderate Obsessive-Compulsive Spinocerebellar across an array of Alzheimer’s Disease* Disorder Ataxia (SCA) potential neurolog

  49. Slide 49: Pathological Features of Alzheimer's Disease

    Pathological Features of Alzheimer’s Disease • Brain atrophy • Neuronal cell death • Amyloid and tau pathology • Synapse loss (most highly correlated with clinical symptoms) • Glial cell dysfunction Congdon Nat Rev Neurol. 2018;14(7):399-415 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 49

  50. Slide 50: Glutamate Transporter (GLT-1) Is Reduced in Human AD Brains

    Glutamate Transporter (GLT-1) Is Reduced in Human AD Brains HEALTHY HUMAN BRAIN ALZHEIMER'S DISEASE Human autopsy observations from AD and controls (n=13) GLT-1 immunoreactivity in temporal neocortex area in AD is significantly weaker than that in control A. Hosi et al. Neuropathology and Applied Ne

  51. Slide 51: Riluzole Rescues Symptoms, Function and Pathology in AD Animal Models

    Riluzole Rescues Symptoms, Function and Pathology in AD Animal Models Rescues Cognitive Symptoms Increases GLT-1 Glutamate Transporter4 Reduces Amyloid and Tau Pathology (Learning and Memory) 5 Young-control Y-maze Reduces amyloid plaque Riluzole-treated Aged-treated aged animals perform like young-

  52. Slide 52: Troriluzole Phase 2/3 Clinical Trial Design in AD

    Troriluzole Phase 2/3 Clinical Trial Design in AD TRORILUZOLE (BHV-4157) PHASE 2/3 • Key entry criteria • Diagnosis of mild to moderate Alzheimer’s disease with Mini-Mental State Exam (MMSE) score of 14–24 Screening Randomized • Co-primary efficacy endpoints Phase Phase • Alzheimer's Disease Assessm

  53. Slide 53: Cerebellar Ataxias: No FDA-Approved Medications for SCA

    Cerebellar Ataxias No FDA-Approved Medications for SCA • 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 100,00

  54. Slide 54: Troriluzole Treated SCA Patients vs Natural History Cohort

    Troriluzole Treated SCA Patients Treated for 1 Year Compared to Matched Ashizawa Natural History Cohort • Post-hoc analysis of patients SCA Patients on Troriluzole vs. Natural History Cohort enrolled in long-term extension of Phase 2b/3 troriluzole SCA trial • Primary efficacy endpoint: change SE) f

  55. Slide 55: Troriluzole Study BHV4157-206 Randomized Controlled Trial in SCA

    Troriluzole Study BHV4157-206 Randomized Controlled Trial in SCA Key Entry Criteria • SCA genotypes (SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, Screening Randomization Extension Phase Phase Phase SCA10) 6 weeks 48 weeks 48 weeks Design • Sample size: 230 subjects Troriluzole • Randomization: 1:1 200 mg QD

  56. Slide 56: OCD Treatments Are Limited and Need Is Vast

    OCD Treatments Are Limited and Need Is Vast • Few FDA-approved treatments • Serotonin reuptake inhibitors (SSRIs and clomipramine) are the only approved monotherapy • No new mechanisms for 30 years • With limited efficacy1,2 • <50% of patients respond well • 25–30% are refractory • Many of those cat

  57. Slide 57: Accumulating Evidence for Glutamatergic Dysfunction in OCD

    Accumulating Evidence for Glutamatergic Dysfunction in OCD ANIMAL NEURAL PATIENTS GENES MODELS NETWORKS IN THE CLINIC Genetic association ↑Glutamatergic activity Multiple neuroimaging Preliminary efficacy studies: worsens OCD behaviors studies: evidence: Glutamate transporter gene SAPAP3 knockout mi

  58. Slide 58: Neurochemical Evidence for Glutamate Dysregulation in OCD

    Neurochemical Evidence for Glutamate Dysregulation in OCD /water) 4 x10 CaudateGlutamatergic Healthy Control Treatment Naïve Concentration( Subjects OCD Patients n = 11 n = 11 Rosenberg et al. 2000 Consistent with pathologic hyperactivity in cortico-striatal circuit Suggests clinical testing of agen

  59. Slide 59: Neurochemical Evidence for Glutamate Dysregulation in OCD: CSF

    Neurochemical Evidence for Glutamate Dysregulation in OCD CSF Glutamate Concentrations Bhattacharyya et al. 2009 Consistent with pathologic hyperactivity in cortico-striatal circuit Suggests clinical testing of agents that enhance glutamate uptake JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 59

  60. Slide 60: Randomized Controlled Trial with Riluzole Augmentation

    Randomized Controlled Trial with Riluzole Augmentation • Outpatients with moderate-to-severe OCD severity (Y-BOCS score ≥ 21) and free of medication treatment for at least six weeks • Randomized to receive fluvoxamine (up to 200 mg/day) plus either placebo or riluzole (50 mg twice daily) * • Robust

  61. Slide 61: Troriluzole Study BHV4157-202 in OCD

    Troriluzole Study BHV4157-202 in Randomized Controlled Trial in OCD KEY ENTRY CRITERIA • Moderate-to-severe OCD and inadequate response to Screening Randomization Extension standard of care Phase Phase Phase 42 days 12 weeks 48 weeks DESIGN • Sample size: 226 subjects • Randomization: 1:1 SOC + Tror

  62. Slide 62: MPO Platform

    MPO PLATFORM Therapies for Neuroinflammation

  63. Slide 63: Multiple System Atrophy (MSA)

    Multiple System Atrophy (MSA) VERDIPERSTAT (BHV-3241) FOR NEUROINFLAMMATION • Rare, rapidly progressive and fatal neurodegenerative disease • Prevalence: 2–5 per 100,000 • Clinical symptoms • Parkinsonism: characteristic tremor (not responsive to L-DOPA), rigidity, dysarthria, falls • Cerebellar ata

  64. Slide 64: Verdiperstat (BHV-3241) is a myeloperoxidase inhibitor

    Verdiperstat (BHV-3241) is a myeloperoxidase inhibitor BHV-3241 FOR NEUROINFLAMMATION • 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 neurodegenerat

  65. Slide 65: Verdiperstat Neuroprotective Effects in MSA Animal Model

    Verdiperstat Neuroprotective Effects in MSA Animal Model1 BHV-3241 FOR NEUROINFLAMMATION Suppresses microglial activation Reduces GCIs of alpha-synuclein Rescues neurodegeneration Promotes functional (motor) improvements RLZ treated 1. Neurotox Res 2012;21(4):393-404 JUNE 2020 BIOHAVEN INVESTOR PRES

  66. Slide 66: Verdiperstat Clinical Experience

    Verdiperstat Clinical Experience BHV-3241 FOR NEUROINFLAMMATION • Studied in approximately 250 subjects (healthy volunteers, Parkinson’s disease, MSA) Baseline AZD3241, 4w AZD3241, 8w • Generally safe and well tolerated • Demonstrated target engagement (↓ blood MPO activity) • Reduced neuroinflammat

  67. Slide 67: Verdiperstat Phase 2 Study in MSA Completed by AstraZeneca

    Verdiperstat Phase 2 Study in MSA Completed by AstraZeneca BHV-3241 FOR NEUROINFLAMMATION • Phase 2 study: randomized double-blind controlled trial • N=61 subjects (MSA-C, 34; MSA-P, 2 4 ) Mean Change on UMSARS Total Score • Randomized to 12 weeks treatment • Placebo BID vs BHV-3241 300 mg BID vs BH

  68. Slide 68: Verdiperstat Plan for Phase 3 Study in MSA

    Verdiperstat Plan for Phase 3 Study in MSA BHV-3241 FOR NEUROINFLAMMATION DESIGN • Sample size: 252 subjects Screening Phase Randomization Phase • Randomization: 1:1 6 weeks 1 year • Dose: 600 mg BID vs. Placebo *based on Ph2 BHV-3241 600 mg BID • Primary outcome measure: Unified MSA Rating Scale R

  69. Slide 69: Metallothionein Blockade

    METALLOTHIONEIN BLOCKADE A Novel Anti-Inflammatory Therapeutic

  70. Slide 70: Metallothionein Can Alter Immune Response

    Metallothionein Can Alter Immune Response by Influencing Cellular Trafficking Stress-causing cell damage Healthy tissue Disease propagation cycle: more stress and Extracellular MT more inflammation recruits Metallothionein production and inflammatory cell release as a “danger signal” JUNE 2020 BIOHA

  71. Slide 71: Metallothionein: A Novel Target in the Immune Cascade

    Metallothionein: A Novel Target in the Immune Cascade • Metallothionein (MT) is an intracellular protein in non-stressed conditions • MT is released from cells/tissues that are stressed, damaged, or killed • Extracellular MT can act as a pro-inflammatory agent • MT acts as an agent of disease progre

  72. Slide 72: UC1MT Reduces Inflammation in Adoptive T Cell Transfer Model of IBD

    UC1MT Reduces Inflammation in the Adoptive T Cell Transfer Model of IBD Anti-MT performance exceeded conventional anti-TNF therapeutic JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 72

  73. Slide 73: UC1MT: First in Class Metallothionein Antagonist

    UC1MT: First in Class Metallothionein Antagonist for a Novel Pro-Inflammatory Target • UC1MT is a high affinity monoclonal antibody that recognizes both mouse and human MT1 and MT2 • Confirmed the role of MT involvement in multiple disease models with better activity than anti-TNF • Evidence of mole

  74. Slide 74: 2020 Milestones/Commercial Transformation

    2020 Milestones/Commercial Transformation

  75. Slide 75: 2020 Milestones & Next Steps

    2020 Milestones & Next Steps 1Q20 Rimegepant approval in acute treatment of migraine 1Q20 Phase 3 topline data for rimegepant in preventive treatment of migraine 2Q20 Phase 2/3 topline data of troriluzole in OCD 4Q20/ Phase 2/3 trial data of troriluzole in Alzheimer’s Disease 1Q21 2021 Phase 3 clini

  76. Slide 76: Neuroinnovation

    neuroinnovation Zydis® is a registered trademark of Catalent. WWW.BIOHAVENPHARMA.COM Rilutek® is a registered trademark of Covis Pharma B.V. NYSE: BHVN Reyvow is a trademark of Eli Lilly and Company © 2020 Biohaven Pharmaceuticals Inc. All rights reserved.

Biohaven Pharmaceutical Holding — Investor Presentation (June 2020) on 6ix