Exhibit 99.1 neuroinnovation Meeting Patient Needs Growing Value INVESTOR PRESENTATION | JUNE 2020 NYSE: BHVN © 2020 Biohaven Pharmaceuticals Inc. All rights reserved.
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 our product candidates, our planned clinical trials for our rimegepant, vazegepant (BHV-3500), troriluzole, BHV-5000 and verdiperstat development programs, the timing of the availability of data from our clinical trials, the timing of our planned regulatory filings, the timing of and our ability to obtain and maintain regulatory approvals for our product candidates and the clinical potential utility of our product candidates, alone and as compared to other existing or potential treatment options. These statements involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements and from the Company's current expectations. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. The forward-looking statements in this presentation represent our views as of the date of this presentation. Subsequent events and developments may cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no obligation to do so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. For further information regarding these risks, uncertainties and other factors, you should read the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the Securities and Exchange Commission (the ”SEC”) on February 26, 2020 and the Company’s other reports filed with the SEC. References to www.biohavenpharma.com in this presentation are not intended to, nor shall they be deemed to, incorporate information on such website into this presentation by reference. This presentation also contains market data and other statistical information that are based on independent industry publications, reports by market research firms or published independent sources. Some market data and statistical information are also based on the Company's good faith estimates, which are derived from management's knowledge of its industry and such independent sources referred to above. While the Company is not aware of any misstatements regarding the market and industry data presented herein, such data involve risks and uncertainties and are subject to change based on various factors. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 2
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 approach to support independent by advancing first-in-class products across commercialization and best-in-class therapies 3 technology platforms Biohaven is focused on innovative pharmaceutical solutions to address unmet needs in the treatment of neurological diseases JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 3
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 Intranasal Acute Migraine Intranasal Acute Migraine COMPLETED-POSITIVE COMPLETED-POSITIVE COMPLETED-POSITIVE COMPLETED-POSITIVE COMPLETED-POSITIVE 4Q2020/1Q2021 Topline TRORILUZOLE TRORILUZOLE TRORILUZOLE BHV-5000 GLUTAMATE Phase 2/3 Phase 2/3 Phase 3 Phase 1 Alzheimer’s Disease (AD) OCD Spinocerebellar Ataxia Neuropsychiatric modulation Passed Interim Analysis 2Q2020 Topline Initiated 1Q2019 Indications LPLT 4Q2020 VERDIPERSTAT VERDIPERSTAT MPO Phase 3 Phase 3 Multiple System Atrophy Amyotrophic Lateral Sclerosis inhibition 2021 Topline 2Q2020 Planned Start Pre-Clinical Amyotrophic Neurological Lateral TDP-43 UC1MT Indications Sclerosis Rimegepant: formerly BHV-3000; Vazegepant: formerly BHV-3500; MPO: Myeloperoxidase; TDP-43: TAR DNA-binding protein 43; UC1MT: monoclonal antibody targeting extracellular metallothionein JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 4
CGRP Receptor Antagonist Platform: Redefining the Treatment of Migraine
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 attacks2 • Reduced functionality, disrupting daily lives, work productivity, and social interactions2,4 • Severity, frequency, and characteristics of migraine vary among patients, and often within the same patient over time2 Regardless of frequency, severity, or use of preventative treatment, all patients require effective acute treatment for their migraine attacks. References: 1. Burch R, Rizzoli P, Loder E. Headache. 2018;58(4):496–505. 2. The American Headache Society. Headache. 2019;59(1):1–18. 3. Bonafede M, Cai Q, Capell K. JMCP. 2017;23(11):1169–1176. 4. Martelletti P et al. J Headache Pain. 2018;19(1):115. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 6
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 migraine-related ER visits incurred $3,125 MORE total all-cause costs vs those with less than 2 visits3 Direct health care costs associated with migraine (including interventions) are significant and affect patients, their families, and employers4 References: 1. The American Headache Society. Headache. 2019;59(1):1-18. 2. Migraine Resource Center website. https://www.mdedge.com/neurology/ migraineresourcecenter/article/160613/headache-migraine/migraine-often-results. Accessed August 8, 2019. 3. Bonafede M, Cai Q, Cappell K. JMCP. 2017;23(11):1169-1176. 4. Bonafede M, Sapra S, Shah N et al. Headache. 2018;58(5):700-714. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 7
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 with their current migraine treatment plan2 § Migraine patients twice as likely to use opioids, and had 1.8x the number of opioid scripts per patient3 References: 1. Migraine.com. https://migraine.com/infographic/migraine-is-more-than-a-headache-its-a-life-ache/. Published August 30, 2016. Accessed September 9, 2019. 2. Data on file, Biohaven Pharmaceuticals 3. Bonafede M, Cai Q, Cappell K. JMCP. 2017;23(11):1169-1176. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 8
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% experience AEs return of migraine Approximately 2.6M acute therapies can currently available within 24 hours2 pain/symptoms within in the US4 cause MOH5 treatments1 24 hours3 References: 1. Lipton RB et al. Headache. 2017;57(7):1026-1040. 2. Derry CJ et al. Cochrane Database Syst Rev. 2012;2(2):CD008615. 3. Géraud G et al. Headache. 2003;43(4):376- 388. 4. Buse DC et al. Headache. 2017;57(1):31-44. 5. Schwedt TJ et al. J Headache Pain. 2018;19(1):38. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 9
Now FDA Approved JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 10
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 seconds— relief and returned to normal function in just before use1 no water necessary1,2 60 minutes with Nurtec ODT3,* Important Safety Information: If a serious hypersensitivity reaction occurs, discontinue Nurtec ODT and initiate appropriate therapy. Serious hypersensitivity reactions have included dyspnea and rash, and have occurred days after administration. *37% (n=246/669) of patients experienced pain relief vs 31% (n=213/682) on placebo (P<0.05). 22% (n=149/669) of patients experienced freedom from functional disability vs 16% (n=108/682) on placebo (P=0.0025).3,4 1. Nurtec ODT [prescribing information]. New Haven, CT: Biohaven Pharmaceuticals, Inc; 2020. 2. Catalent. https://consumerhealth.catalent.com/over-the-counter/zydis-odt-technology/. Accessed February 20, 2020. 3. Croop R et al. Lancet. 2019;394(10200):737-745. 4. Data on file. Biohaven Pharmaceuticals; 2019. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 11
Nurtec ODT Blister Pack (8-count) JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 12
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“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, Pearson S D, Rind D M. Acute Treatments for Migraine: Effectiveness and Value. Institute for Clinical and Economic Review, February 25, 2020. http://icer-review.org/material/acute-migraine-evidence-report/ JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 18
• 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 of migraine: a randomised, phase Positive Phase 3 3, double-blind, placebo-controlled trial." Lancet 394(10200): 737-745. Trials, 1-Year Safety • *Patients may have been double counted if enrolled in both the long-term safety study 201, and a phase 3 trial. ODT, orally dissolving tablet. APRILJUNE 20202020 BIOHAVEN INVESTOR PRESENTATION 1919
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, discontinue Nurtec ODT and initiate appropriate therapy. Serious hypersensitivity reactions have included dyspnea and rash, and have occurred days after administration in less than 1% of patients. Adverse Reactions: The most common adverse reaction was nausea (2% of patients). JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 20
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 to Nurtec ODT or any of its components. Warnings and Precautions: If a serious hypersensitivity reaction occurs, discontinue Nurtec ODT and initiate appropriate therapy. Serious hypersensitivity reactions have included dyspnea and rash, and can occur days after administration. Adverse Reactions: The most common adverse reaction was nausea (2% in patients who received Nurtec ODT compared to 0.4% in patients who received placebo). Hypersensitivity, including dyspnea and rash, occurred in less than 1% of patients treated with Nurtec ODT. Drug Interactions: Avoid concomitant administration of Nurtec ODT with strong inhibitors of CYP3A4, strong or moderate inducers of CYP3A or inhibitors of P-gp or BCRP. Avoid another dose of Nurtec ODT within 48 hours when it is administered with moderate inhibitors of CYP3A4. Use in Specific Populations: Pregnant/breast feeding: It is not known if Nurtec ODT can harm an unborn baby or if it passes into breast milk. Hepatic impairment: Avoid use of Nurtec ODT in persons with severe hepatic impairment. Renal impairment: Avoid use in patients with end- stage renal disease. Please see full Prescribing Information available at www.nurtec.com JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 21
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 Observation Phase • Assess Migraine Frequency and Disability Dispense Start treatment Stop treatment 1. Patients were dispensed open label rimegepant 75 mg in bottles containing 30 tablets; A maximum of 1 tablet per calendar day was permitted JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 22
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} (n=1031) (n=481) Mean (SD) 5.6 (3.50) 8.5 (4.20) Median 4.9 7.8 • To further test safety, patients in additional EOD+PRN group received scheduled every other day dosing (EOD) and could also dose PRN on nonscheduled dosing days for up to 3 months (n=286): median usage = 14.2 tablets per month (based on 28-day month) Tablets per 4 weeks are based on 4-week (28-day) intervals in the long-term treatment period. Data on file. Study 201: Biohaven Pharmaceuticals; 2019. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 23
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 Disability course of 1-year 21 20.7 19 17 19.1 - 48.3% Mean MIDAS Total Score Mean MIDAS Total Moderate 17.5 16.4 15 0 weeks 12 Weeks 24 Weeks 36 Weeks 52 weeks MIDAS, Migraine Disability Assessment. Validated, 5-item instrument that queries migraine-related absenteeism and lost productivity at work, school, and home, as well as missed family, social, and leisure activities Reference: Biohaven presents data demonstrating reduction in migraine-related disability and improvement in patient reported outcomes after oral treatment with rimegepant at the international headache conference late breaking session. Biohaven Pharmaceuticals website. https://www.biohavenpharma.com/investors/news-events/press-releases/09-09-2019. Published September 9, 2019. Accessed September 12, 2019. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 24
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 12 10.7 migraine 9.6 8.9 8.9 presenteeism items were seen 10 throughout treatment follow-up 8 10 9.4 8.7 8.4 Presenteeism 6 Days when 4 2 migraine interfered 0 with work, school, baseline 12 24 36 52 household, social ABSENTEEISM PRESENTEEISM or leisure activities Week Reference: Biohaven presents data demonstrating reduction in migraine-related disability and improvement in patient reported outcomes after oral treatment with rimegepant at the international headache conference late breaking session. Biohaven Pharmaceuticals website. https://www.biohavenpharma.com/investors/news-events/press-releases/09-09-2019. Published September 9, 2019. Accessed September 12, 2019. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 25
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 baseline of moderate-to-severe 30% migraine days per month with rimegepant 20% Severe Migraine Days/ 75 mg (n=244) - to - 10% 0% Patients Showing ≥50% Reduction in Moderate All <14 ≥14 n=244 n=218 n=26 Migraine Frequency in Observation (days) Reference: Lipton R et al. Presented at the 2019 American Academy of Neurology Annual Meeting; May 4-10, 2019; Philadelphia, PA. Poster P235LB JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 26
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 number of migraine days per month • Orally administered rimegepant 75 mg, approved earlier this year for the acute treatment of migraine, is the only CGRP targeting therapy to demonstrate efficacy in both the acute and preventive treatment of migraine • Biohaven plans to engage the FDA and European Medicines Agency to submit a sNDA and MAA, respectively, for rimegepant for the indication of preventive treatment of migraine JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 27
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=370) Study 305 History of 4 to 18 migraine days R End of Study (N=741) per month 1:1 Visit Placebo (n=371) R randomization Objective: Assess Rimegepant 75 mg versus placebo on safety, efficacy and tolerability in the preventive treatment of migraine JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 28
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 baseline in monthly migraine days at month 3 compared to a 3.5 day -3 reduction in the placebo group, p < 0.01. • Baseline MMDs = 10.1 -4 during observation period -3.5 across all evaluable subjects -5 -4.3 Mean Change from Baseline in in Baseline from Change Mean a Monthly Migraine Days at 3 Months 3 at Days Migraine Monthly p < 0.01 -6 a Figure shows model-based estimates of the change from baseline for evaluable mITT cohort. MMD monthly migraine days. EOD every other day dosing JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 29
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 baseline, in -20 moderate to severe migraine days per month at month 3, compared to 41.5% for placebo, p < 0.05 -30 • Baseline MMDs = 10.1 during observation period -40 across all evaluable subjects -41.5 -50 -49.1 Moderate to Severe Migraine Days/mo Migraine Severe to Moderate % Patients Showing ≥50% Reduction in -60 p < 0.05 a Figure shows model-based estimates of the change from baseline for evaluable mITT cohort. Moderate-Severe pain intensity. MMD monthly migraine days. EOD every other day dosing JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 30
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 of migraine days per month, and key secondary outcome measure assessing the proportion of patients who achieved a ≥50% reduction in moderate-to-severe migraine days at month 3 • Patients receiving the drug every other day experienced a statistically significant 4.3 day reduction from baseline in monthly migraine days, compared to 3.5 day reduction in the placebo group, p < 0.01 • 49.1% of patients treated with NURTEC 75 mg showed a ≥50% reduction from their baseline number of moderate-to-severe migraine days at month 3 compared to 41.5% of placebo-treated patients, p < 0.05 • Nurtec™ ODT 75 mg was approved earlier this year for the acute treatment of migraine showing rapid onset within 60 minutes and durable benefits out to 48 hours with a single dose • Nurtec is part of Biohaven’s NOJECTIONTM CGRP receptor antagonist platform and is the first therapy to complete pivotal trials that demonstrate efficacy in both acute and preventive treatment of migraine JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 31
Commercial and Launch Readiness
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) JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 33
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 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 3434
Managed Markets Customer Engagements JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 3535
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” Accounts Completed 211,198,254 94.6% Scheduled 7,289,528 3.3% 97.9% Pending 4,751,116 2.1% Total 223,238,898 100.0% B Priority Accounts Lives % Total “B” Accounts Completed 64,290,107 62.8% Scheduled 29,334,656 28.7% 91.5% Pending 8,676,722 8.5% Total 102,301,485 100.0% JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 3636
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 advantaged coverage vs Ubrelvy on six state 20 Medicaid plans 0 • Medicare access on schedule May June (Actual) (Actual*) * Not fully reflected in MMIT or Fingertip Formularies covered lives data as of June 1st
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-moderate attacks2 Used for moderate-to-severe migraine7 • Mild-to-moderate that respond poorly Use to NSAIDs or caffeinated combinations2 Use with caution in patients who have:1,2 • Can cause serious gastrointestinal • Short-acting opioids have the • Coronary artery disease and cardiovascular side effects4 potential for physical dependence • Peripheral vascular disease • May be associated with delays in and/or psychological addiction7 Shortcomings • Uncontrolled hypertension absorption due to gastric hypomotility • May worsen patient's primary • Other vascular risk factors and during migraine attacks3 migraine disease through overuse7 disorders • Concomitant SSRI use *Excedrin Migraine contains NSAID and nonNSAID ingredients. References: 1. Rothrock JF, Friedman DI. American Headache Society website. https://americanheadachesociety.org/wp-content/uploads/2018/05/John_Rothrock_and_Deborah_Friedman_-_Triptans.pdf. Accessed September 9, 2019. 2. The American Headache Society. Headache. 2019;59(1):1-18. 3. Pardutz A, Schoenen J. Pharmaceuticals. 2010;3:1966-1987. 4. Varga Z, Sabzwari SRA, Vargova V. Cureus. 2017; 9(4):e1144. 5. Dodson H, Bhula J, Eriksson S et al. Cureus. 2018;10(4):e2439. doi:10.7759/cureus.2439. 6. Franklin GM. Neurology. 2014;83:1277-1284. 7. American Migraine Foundation website. https://americanmigrainefoundation.org/resource-library/opioid-therapy-migraine/. Published October 12, 2007. Accessed September 9, 2019. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 38
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 System Depression Driving Impairment REYVOW may cause central nervous system (CNS) depression, including REYVOW may cause significant driving dizziness and sedation. 9.1 Controlled Substance impairment. In a driving study, administration Because of the potential for REYVOW to cause sedation, other cognitive REYVOW contains lasmiditan, a of single 50 mg, 100 mg, or 200 mg doses of and/or neuropsychiatric adverse reactions, and driving impairment, Schedule V controlled substance. REYVOW significantly impaired subjects’ REYVOW should be used with caution if used in combination with alcohol ability to drive. or other CNS depressants. 9.2 Abuse …With all doses of REYVOW, Patient who cannot follow this advice should Serotonin Syndrome subjects reported statistically In clinical trials, reactions consistent with serotonin syndrome were not take REYVOW. significantly higher “drug liking” reported in patients treated with REYVOW who were not taking any other drugs associated with serotonin syndrome. Serotonin syndrome may also scores than placebo, indicating that Prescribers and patients should be aware that REYVOW has abuse potential…. patients may not be able to assess their own occur with REYVOW during coadministration with serotonergic drugs… driving competence and the degree of Medication Overuse Headache impairment caused by REYVOW. Overuse of acute migraine drugs (e.g., ergotamines, triptans, opioids, or a combination of these drugs for 10 or more days per month) may lead to exacerbation of headache… JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 39
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 soluble and high free fraction • US composition of matter protection to March 20311 • Multiple potential routes of delivery hCGRP Ki = 23 pM • Nasal, inhalation and oral • Vazegepant Achieved Positive Topline Results in Pivotal Phase 2/3 Study Rimegepant • 10 and 20 mg achieved statistical superiority to placebo on regulatory endpoints of pain freedom and freedom from most bothersome symptom at 2 hours • Vazegepant showed evidence of rapid onset with pain relief as early as 15 minutes, return to normal function at hCGRP Ki = 32 pM 30 min and sustained benefits through 48 hours 1. Patent expiration, not including patent term adjustment or any potential patent term extensions JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 40
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 Placebo (n=401) 61% 61% Vazegepant 10 mg (n=391) 60 Vazegepant 20 mg (n=402) 50% 46% 45% 50 54% 43% 40% 39% 40 30% 42% 27% 30 36% 33% 18% 20 15% 25% 10 10% 0 // % of Patients with Pain Relief with Pain % of Patients 15 min 30 min 60 min 120 min 2-24 hr 2-48 hr Time Single Dose of Vazegepant, No Rescue Meds 1. Pain Relief is defined as patients who have either mild-pain or no-pain during the specified interval. 2. Sustained Pain Relief is defined as patients having mild-to-no-pain at 2 hours and continuing to the end of the specified interval. Estimates computed using the mITT population and CMH methods (mean ± Alpha’s Standard Error). Subjects using rescue medications at or before the assessment, and subjects not providing data, are classified as failures. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 41
GLUTAMATE PLATFORM Therapies for Neurologic and Neuropsychiatric Indications
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 TRANSMISSION MOOD NEURODEGENERATION NEUROTROPHIC CANCER STRESS PAIN SPINOCEREBELLAR ATAXIA ACTION POTENTIAL CELL RETT SYNDROME SURVIVAL COGNITION NEUROTOXICITY SEIZURES Biohaven is focused on normalizing glutamate to treat disease JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 43
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 INVESTOR PRESENTATION 44
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 tolerated, safe in clinical settings at approved dose • Originally marketed by Sanofi, received FDA approval in 1995 LIMITATIONS • In 2013, the FDA approved the first generic ✘ Twice daily dosing, low bioavailability versions of riluzole ✘ Fasting required for 6 hours/day, • Doses above 100 mg for efficacy not approved can’t be taken with meals due to dose-dependent liver effects ✘ Dose dependent LFT liability1 ✘ Marked PK variability ✘ High drug burden relative to efficacy2 ✘ Only one approved indication (ALS) 1. LFT = liver function test 2. Poor oral bioavailability results in a high liver burden relative to efficacy as ~40% is either not absorbed or is metabolized in the liver JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 45
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
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
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 neurologic and neuropsychiatric indications Prodromal Trichotillomania Friedreich’s Ataxia Alzheimer’s Disease ALS (High Dose) Hoarding Disorder Sporadic Ataxia Other Ataxias Essential Tremor* * = Third-party study / collaboration ongoing or planned (ADCS Collaboration for AD; ET Study Group Collaboration for ET) JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 48
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
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 Neurobiology (2018) JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 50
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-controls1 Aged-control Vehicle control (sec) Time Exploration Time (min) Morris water maze 2,3 Reduces p-tau2 Control Riluzole- TauP301L treated tau- Riluzole + overexpressing 13/Actin TauP301L animals - perform like CP (Relative Ratio) (Relative RLZ treated Errors Maze Water controls Control TauP301L Riluzole + TauP301L 1. Pereira Proc Natl Acad Sci 2014; 2.Hunsberger J Neurochem 2015; 3. Hunsberger, Metab Brain Dis 2016; 4. Pereira Molecular Psychiatry 2017; 5. Okamoto Transl Psychiatry 2018 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 51
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 Assessment Scale (ADAS)-Cog11 42 days 48 weeks • Clinical Dementia Rating-Sum of Boxes (CDR-SB) Troriluzole • Secondary efficacy endpoints 280 mg QD • Brain volumes: MRI imaging • Activities of daily living: ADCS-ADL R • Neuropsychiatric: NPI Placebo QD • Neuropsychological: NTB • Cognitive: MMSE/MoCA • Sample size: 292 subjects Passed interim Futility • Randomization: 1:1 Analysis December 2019 • Collaborator: Alzheimer’s Disease Cooperative Study JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 52
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 100,000 (estimated 3,200–18,000 in US) • Neurodegeneration of cerebellum and input/output tracts • Relentlessly progressive, often fatal (aspiration) • Increasing disability over time (requiring wheelchair, assistance with activities of daily living) • The 6 most common genotypes caused by triplet repeat expansion mutations, sharing many phenotypic features • Genetic anticipation in some: subsequent generations affected at earlier Spinocerebellar Ataxia type 2 Cerebellar and brainstem volume loss ages and with greater severity • Other ataxias have similar core features • Can be recessive, dominant, immune, mitochondrial, post-stroke, e.g., Friedreich’s ataxia, ataxia telangiectasia, multiple system atrophy — cerebellar type JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 53
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) from baseline in the Total SARA ± Score after 48 weeks • Patients from BHV4157-201 trial Total in Change 2 Difference: -1.41 ± 0.411 versus eligibility criteria matched (95% confidence interval of Ashizawa Natural History cohort: (from baseline -2.22 to -0.60) suggesting • SCA Genotype therapeutic benefits of • SCA1, SCA2, SCA3, SCA6 troriluzole (p=0.0007) • Age at baseline • 18 to 75 years of age SARA Score SARA • Gender 1. Matched on eligibility criteria Least Squares Mean Squares Least 2. ANCOVA model with fixed effects for cohort, • SARA Score at baseline sex, & SCA genotype with age and baseline • ≥ 8 and ≤ 30, and SARA scores as covariates • Initial SARA gait item score ≥ 2 Troriluzole Study (BHV4157-206) in Spinocerebellar Ataxia (SCA) Achieved Phase 2/3 start in 1Q19 SARA: Scale for the Assessment and Rating of Ataxia JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 54
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 • Stratification: by SCA genotype R Troriluzole • Dose: Troriluzole 200 mg QD vs. 200 mg QD Placebo QD Placebo QD • Primary Outcome: Modified SARA Scale • FDA aligned outcome measure Status • Phase 3 (initiated 1Q19) SARA: Scale for the Assessment and Rating of Ataxia JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 55
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 categorized as ‘responders’ continue to suffer • Leading many patients to seek invasive treatments • Deep brain stimulation • Ablative neurosurgery 1992 1994 1996 1997 1. Hirschtritt ME et al (2017). JAMA 317(13):1358-1367. 2. Pallanti S et al (2006). Prog Neuropsychopharmacol Biol Psychiatry 30(3):400-412. JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 56
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 mice (-/-) Increased activity in Glutamate modulating agents 2 (SLC1A1 on chromosome exhibit OCD-like behaviors cortico-striatal-thalamic show promise in OCD patients 9p24) associated w/OCD1 SAPAP3 is an ortholog of (CST) pathway3 GWAS studies are focusing DLGAP3, in the same family attention on DLGAP1, a post- as a gene implicated by OCD MRS studies: synaptic scaffolding molecule GWAS studies Glutamate dysfunction at glutamate synapse in OCD suggested by some studies4,5 Spinal fluid studies: Increased glutamate in OCD patients6 1. Arnold et al 2006; 2. Aida et a; 2015; 3. Baxter et al 1987, 1988, 1992; Nordhal et al 1989; Swedo et al 1989; Sawle et al 1991; Rubin et al 1992, 1995; Adams et al 1993; Perani et al 1995; Adams et al 1993; Perani et al 1995; McGuire et al 1994; Breiter et al 1996; Rausch et al 1996; 4. Rosenberg et al 2000; 5. Bolton et al 2001; 6. Chakrabarty et al 2005 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 57
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 agents that enhance glutamate uptake JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 58
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
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 benefit on primary outcome measure (change in Y-BOCS scores) Placebo (n=25) BOCS Total Score Total BOCS - Y Riluzole (n=25) *, p<0.05; Cohen’s d 0.59 Emamzadehfard et al. 2016 YBOCS, Yale-Brown Obsessive Compulsive Scale JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 60
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 + Troriluzole • Dose: Troriluzole 200 mg QD vs. 200 mg QD Placebo QD (in patients on R SOC + Troriluzole standard of care) 200 mg QD • Primary Outcome: Y-BOCS, SOC + a precedented outcome Placebo QD measure accepted by FDA STATUS Subjects (n=226) with Moderate to severe OCD and inadequate response to standard of care • Enrollment active • Expect to complete enrollment in SOC, standard of care 1Q2020 YBOCS, Yale-Brown Obsessive Compulsive Scale JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 61
MPO PLATFORM Therapies for Neuroinflammation
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 ataxia • Autonomic failure: orthostatic hypotension, urinary dysfunction, erectile dysfunction • Prognosis: more rapidly progressive than Parkinson’s disease • Time to loss of ambulation: 3.5–5 years • Mean survival from symptom onset: 6–10 years • Pathology: glial cytoplasmic inclusions (GCIs) containing alpha-synuclein • Disease mechanisms: oxidative stress and neuroinflammation • No disease modifying treatments • Symptomatic and palliative management JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 63
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 neurodegeneration1 • Verdiperstat • Potent, first-in-class, brain-penetrant MPO inhibitor • Developed by AstraZeneca (formerly AZ3241) 1. Neurotox Res 2012;21(4):393-404 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 64
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 PRESENTATION 65
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 neuroinflammation (microglial activation) in Parkinson’s disease • Positron Emission Tomography imaging showed decreased TSPO Jucaite et al., 2015. ligand binding JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 66
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 BHV-3241 600 mg BID • Outcome measures: UMSARS*, PET, safety (labs, AE’s, ECGs, vitals) • Generally safe and well tolerated • Emerging efficacy signals warrant further study in MSA • Dose proportional benefit on mean UMSARS decline • Dose proportional rates of clinically meaningful improvement • 600 mg dose with statistical trends (p<0.10) for superiority BaselineUMSARS ChangeTotalfrom over placebo on multiple UMSARS items • 600 mg dose shows numerical improvement on MSA Quality of life scale * UMSARS, Unified MSA Rating Scale: primary efficacy measure for the study JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 67
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 • Sites: US, EU (UK, FR, GER, AUS, IT), CHINA Placebo BID STATUS • FDA EOP2 Meeting completed • EU Scientific Advice received • Study initiated 3Q2019 JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 68
METALLOTHIONEIN BLOCKADE A Novel Anti-Inflammatory Therapeutic
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 BIOHAVEN INVESTOR PRESENTATION 70
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 progression in multiple inflammatory models • MT elevations demonstrated in patients with Inflammatory Bowel Disease (IBD) • MT is elevated in diabetes, multiple brain disorders, inflammatory liver disease, inflammatory skin disease, pulmonary arterial hypertension, neoplasia, etc. • Anti-MT monoclonal antibody (UC1MT) significantly reduces disease severity in multiple models tested Pro-inflammatory Progressive Inflammatory MT induction and effects of inflammation and stress release released MT MT synthesis UC1MT blockade JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 71
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
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 molecular mechanism • Demonstrated the ability reduce markers of inflammation by UC1MT • Strong global patent protection of Anti-MT antibody therapeutic in several disease areas JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 73
2020 Milestones/Commercial Transformation
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 clinical trial data for verdiperstat for the treatment of MSA OCD: Obsessive-Compulsive Disorder, MSA: Multiple System Atrophy JUNE 2020 BIOHAVEN INVESTOR PRESENTATION 75
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