Investor Presentation - NEUROINNOVATION We Demand More for Patients - Biohaven Pharmaceuticals
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NEUROINNOVATION ® We Demand More for Patients. Investor Presentation February 2022 Ellie, living with migraine © 2022 Biohaven Pharmaceuticals. All rights reserved. NYSE:BHVN
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® ODT, the potential approval and commercialization of other product candidates, the effect of the ongoing COVID-19 pandemic on the Company, the expected timing, commencement and outcomes of the Company's planned and ongoing clinical trials for our rimegepant (BHV-3000), zavegepant (BHV-3500), BHV-2100, troriluzole, BHV-5500, verdiperstat, BHV-1100 and BHV-1200 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, the clinical potential utility of our product candidates, alone and as compared to other existing or potential treatment options, and the potential advancement of our early phase programs including ARM™, MATE™, MoDE™, TRPM3, TDP-43 and UC1MT. 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, the Company 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. 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. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of Biohaven's Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission on March 1, 2021, and Biohaven's subsequent filings with the Securities and Exchange Commission. 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. Safety information and the full prescribing information for Nurtec ODT can be found at Nurtec.com. February 2022 BIOHAVEN INVESTOR PRESENTATION 2
NEURO INNOVATION ® Combining exceptional drug development expertise with an entrepreneurial attitude to uncover a new and better way to treat neurological and neuropsychiatric diseases We Demand More for Patients.
$1.2+ TOTAL UPFRONT & BILLION MILESTONE VALUE Bringing Dual Therapy Innovation to Patients Around the Globe
Strategic Platforms and Deep Pipeline Filing for Approval PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MARKETED NURTEC ODT | MIGRAINE ACUTE NURTEC ODT | MIGRAINE PREVENTION NURTEC ODT | MIGRAINE EXPANSION STUDIES PHASE 4 STUDIES NURTEC ODT | CHILD ADOLESCENT MIGRAINE Rimegepant BHV-3000 | PAIN ADJACENCIES (TRIGEMINAL NEURALGIA, SINUSITIS, TMJ) CGRP BHV-3000 | NON-MIGRAINE INDICATIONS (PSORIASIS, UNDISCLOSED) BHV-3500 | MIGRAINE ACUTE (NASAL) BHV-3500 | MIGRAINE PREVENTION (ORAL) Zavegepant BHV-3500 | ACUTE TREATMENT OF LUNG INFLAMMATION/COVID-19 BHV-3500 | ASTHMA BHV-4157-206 | SPINOCEREBELLAR ATAXIA (SCA) GLUTAMATE Troriluzole BHV-4157 | OBSESSIVE-COMPULSIVE DISORDER (OCD) MPO Verdiperstat BHV-3241 | AMYOTROPHIC LATERAL SCLEROSIS (ALS) ARM™ BHV-1100 MULTIPLE MYELOMA MATE™ BHV-1200 COVID-19 TRPM3 BHV-2100 NEUROPATHIC PAIN BIOHAVEN LABS MoDE™ BHV-TBD MULTI MODALITY DEGRADERS FOR NEUROIMMUNE DISORDERS TDP-43 BHV-TBD NEURODEGENERATIVE DISEASES UC1MT BHV-TBD INFLAMMATORY AND AUTOIMMUNE DISEASES February 2022 BIOHAVEN INVESTOR PRESENTATION 7
CGRP Unparalleled CGRP Receptor Antagonist Franchise Zavegepant Next-GEN CGRPs RAPID DISSOLVING INTRANASAL ORAL MULTIPLE FORMULATIONS 5 ADVANCED MOLECULES APPROVED NDA Filing Phase 3 Prevention ACUTE FEB 2020 1H2022 Started 1Q2021 PREVENTION MAY 2021 February 2022 BIOHAVEN INVESTOR PRESENTATION 9
CGRP Nurtec® ODT 2021 Achievements $190M Preliminary Net >1,375,000 Sales 4Q2021 TRxs of Nurtec® ODT 40% to Date Sequential Growth Over 3Q $462M 55% New to 2021 Net Sales Brand Rx Share 1. 1/6/2022 Biohaven Press Release — NURTEC® ODT achieved net product revenue of $190 million for the fourth quarter of 2021. Preliminary net product revenue for NURTEC ODT in 2021 totaled approximately $462 million, with over 1,370,000 prescriptions filled since initial product launch in March 2020. 2. New to Brand Rx numbers 3/13/20 – 12/24/21, IQVIA NPA-MD, accessed 1/3/22 February 2022 BIOHAVEN INVESTOR PRESENTATION 10
CGRP Strong and Steady Growth $526M $190M Launch to date net product revenue1 $136M $93M $44M $35M $10M $18M 2Q20 3Q20 4Q20 1Q21 2Q21 3Q21 4Q21 1. 1/6/2022 Biohaven Press Release — NURTEC® ODT achieved net product revenue of $190 million for the fourth quarter of 2021. Preliminary net product revenue for NURTEC ODT in 2021 totaled approximately $462 million, resulting in launch to date net product revenue of $526 million with over 1,370,000 prescriptions filled since initial product launch in March 2020. February 2022 BIOHAVEN INVESTOR PRESENTATION 11
CGRP One Simple 75 mg Dosage Strength to Treat and Prevent Migraine Attacks1,2 For the acute treatment of migraine and the preventive treatment of episodic migraine in adults Finally, the first and only medication proven1,2: FAST LASTS • One rapid dissolving tablet that works quickly to • Treats or prevents for up to 48 hours at a time resolve pain and return many patients back to for many patients1,3,7 normal activities in 1 hour1–4 • Reduction in mean monthly migraine days • Demonstrated preventive effect within 1 week (MMDs) for many patients through 12 weeks for many patients5 of treatment1,2,6 Ellie So you can can TREAT PREVENT Actual Nurtec® ODT Patient Help put the power of migraine control in your patient’s hands ‡Exploratory analysis. Subjects had ≥ 1 day of efficacy data in the observation period and in the first week of the double-blind treatment period.5 Back to normal activities = Return to normal function 1. Nurtec ODT. Package insert. Biohaven Pharmaceuticals Inc. 2. Croop R, Lipton RB, Kudrow D, et al. Oral rimegepant for preventive treatment of migraine: a phase 2/3, randomised, double-blind, placebo-controlled trial. Lancet. 2020;397(10268): 51-60. doi:10.1016/S0140-6736(20)32544-7. 3. Croop R, Goadsby PJ, Stock DA, et al. Efficacy, safety, and tolerability of rimegepant orally disintegrating tablet for the acute treatment of migraine: a randomised, phase 3, double-blind, placebo-controlled trial. Lancet. 2019;394(10200):737-745. doi: 10.1016/S0140-6736(19)31606-X. 4. Data on File. RIM108. Biohaven Pharmaceuticals Inc. 5. Lipton RB, Coric V, Stock EG, et al. Efficacy, safety, and tolerability of rimegepant 75 mg orally dissolving tablet for the acute treatment of migraine: a phase 3, double-blind, randomized, placebo-controlled trial (study 303). Abstract presented at: 61st Annual Scientific Meeting of the American Headache Society; Philadelphia, PA. Session IOR05; July 11, 2019. 6. Lipton RB, Croop R, Jensen CM, et al. Rapid Decrease in Migraine Days With Rimegepant: Results From a Post Hoc Analysis of a Phase 2/3, Randomized, Double- Blind, Placebo-Controlled Trial. Virtual Poster presented at: American Headache Society 2021 Annual Meeting; June 3-6, 2021. 7. Data on File. RIM118. Biohaven Pharmaceuticals Inc. February 2022 BIOHAVEN INVESTOR PRESENTATION 12
CGRP Fast: Rapid Onset Got Patients Back to Normal Activities and Reduced Migraine Frequency1-6 TREATING MIGRAINE ATTACKS PREVENTING THE NEXT ONE (Mean Percentage Change from the Observation Period) BACK TO NORMAL ACTIVITIES1-4 54%* 30% REDUCTION IN WEEKLY n=359 MIGRAINE DAYS AT WEEK 1‡,5,6 38%‡ n=255 32% n=216 26% -9.4% (95% CI -17.1, -1.8) 22%† n=176 n=149 16%† n=108 9%* n=63 7% n=45 -30.0% (95% Cl: -36.1, -23.9) 15 min 1 hr 2 hr 4 hr Nurtec ODT 75 mg (N=370) Placebo (N=371) Nurtec ODT 75 mg (N=669) Placebo (N=682) ‡Exploratory analysis. Subjects had ≥ 1 day of efficacy data in the observation period and in the first week of the double- †P=0.0025 ‡P
CGRP Lasts: Nurtec® ODT Delivers Sustained Efficacy for Lasting Migraine Control1–6 TREATING MIGRAINE ATTACKS PREVENTING THE NEXT ONE SUSTAINED RESPONSE FROM 2 TO 48 HOURS1-3 REDUCTION IN MONTHLY MIGRAINE DAYS (MMDs) AT MONTH 31,4-6 Nurtec ODT has a half-life of 11 hours1,2 63% of patients taking Nurtec ODT Approximately half of patients taking (n=90/142) who experienced freedom Nurtec decreased their moderate to 48 from pain at 2 hours maintained it through 48 hours vs 50% (n=37/74) 49% severe MMDs by ≥50% vs 41% of those taking placebo (n=171/348); P=0.0441,5,* HOURS patients1,2 OF PATIENTS A consistent trend in reduction of mean 14% of patients taking Nurtec ODT 75 mg (n=90/669) experienced freedom from pain from 2–48 hours vs 5% MMDs was shown month over month from of patients taking placebo (n=37/682); P
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CGRP Nurtec® ODT Commercial Success February 2022 BIOHAVEN INVESTOR PRESENTATION 17
CGRP Broad Commercial Coverage: High Impact Commercial PBM and Health Plan Wins 89% COVERAGE 240M+ TOTAL COVERED LIVES IN ALL CHANNELS February 2022 BIOHAVEN INVESTOR PRESENTATION 18
CGRP Oral CGRP Class Continues to Show Robust Market Growth Nurtec® ODT leads in TRx at 51.3% share 1 Total Rx Volume (1/21)¹ 2 Total Rx Share (1/21)1 60% 29,490 Nurtec ODT 55% 50% 51.3% Nurtec ODT 24,959 Ubrelvy 45% 40% 43.4% Ubrelvy 35% 30% Prevention Approval 25% 20% 15% 10% 3,040 Qulipta 5% 5.3% Qulipta 0% 1/24 2/21 3/20 4/17 5/15 5/29 6/12 6/26 7/10 7/24 8/21 9/18 10/2 1/22 2/19 3/19 4/16 4/30 5/14 5/28 6/11 6/25 7/23 8/20 9/17 10/1 1/21 10/16 10/30 11/13 11/27 12/11 12/25 10/15 10/29 11/12 11/26 12/10 12/24 10/15 10/22 10/29 11/12 11/19 11/26 12/10 12/17 12/24 12/31 2/7 3/6 4/3 5/1 8/7 9/4 1/8 2/5 3/5 4/2 7/9 8/6 9/3 1/7 4/23 4/30 5/14 5/21 5/28 6/04 6/11 6/18 6/25 7/16 7/23 7/30 8/13 8/20 8/27 9/10 9/17 9/24 10/1 10/8 11/5 12/3 1/14 1/15 5/7 7/2 7/9 8/6 9/3 1/7 Week ending Week ending KEY INSIGHTS • Nurtec TRx launch curve shows strong growth, overtaking Ubrelvy in Aug 2021, with the brand steadily growing and maintaining leadership • Oral CGRP market for migraine on track to reach blockbuster status in U.S. market alone Source: 1. TRX numbers 1/24/20 – 1/21/22, IQVIA SMART, accessed 1/31/22. Note: Market definition for share calculations is based on Nurtec ODT, Ubrelvy, and Qulipta February 2022 BIOHAVEN INVESTOR PRESENTATION 19
CGRP All Roads Lead to Blockbuster U.S. Market Opportunity TRIPTANS 40M OTHER RX Topiramate, Gabapentin, TCAs, MULTI-BILLION- Novel Injectables DOLLAR MARKET U.S. PATIENTS LIVING WITH MIGRAINE OPPORTUNITY OTCS Advil, Excedrin Migraine, Tylenol, Sudafed, Aleve, Aspirin UNDIAGNOSED February 2022 BIOHAVEN INVESTOR PRESENTATION 20
CGRP Rimegepant Global Market Opportunity 2 Prevention (Dual Therapy) 4 Acute Migraine 12 Acute and Dual By end of 2022 APPROVAL APPROVALS SUBMISSIONS Europe Dual | 1Q21 Japan China Ph 2/3 | 1H22 Lebanon Acute | 3Q21 Ph 3 | 4Q20 Israel Acute & Prevention | Kuwait Korea APPROVED Acute | APPROVED Ph 3 | 4Q20 Bahrain Qatar Acute | 4Q20 Acute | 1Q22 Saudi Arabia Acute | 1Q21 United Arab Emirates Acute | APPROVED APPROVALS Oman SUBMISSIONS Acute | 1Q22 PIVOTAL TRIALS 1 Billion Migraine Sufferers World Wide February 2022 BIOHAVEN INVESTOR PRESENTATION 21
CGRP Product Patent Awarded for ODT Formulation Extends Company's IP for CGRP Platform into 2039 New patent awarded to Biohaven by the United States Patent and Trademark Office for our drug product, Nurtec® ODT (rimegepant), in an ODT form Covers rimegepant as well as other CGRP inhibitors Patent expires in March 2039, not including possible extensions of up to 5 years February 2022 BIOHAVEN INVESTOR PRESENTATION 22
CGRP BHV-3500 Zavegepant: NDA Submission 1Q2022 Superior chemical attributes • Potent antagonist at the human CGRP receptor • Highly soluble and high free fraction First and Only • U.S. composition of matter protection to March 20341 Intranasal CGRP Receptor Antagonist Multiple potential routes of delivery Nasal, inhalation and oral Ultra Rapid Onset Within 15 Minutes First showed positive topline results in pivotal Phase 2/3 dose-ranging study 10 and 20 mg achieved statistical superiority to placebo on regulatory endpoints Ideal for Patients With of pain freedom and freedom from most bothersome symptom at 2 hours Nausea/Vomiting (approximately 50%) Recently achieved positive Phase 3 data in 2nd pivotal study replicating/extending prior results Zavegepant 10 mg met primary endpoints and showed ultra-rapid onset pain relief at 15 minutes, return to normal function at 30 minutes and sustained benefits through 48 hours 1. Patent expiration including anticipated patent term adjustment and potential patent term extensions February 2022 BIOHAVEN INVESTOR PRESENTATION 23
CGRP BHV-3500 Intranasal Zavegepant: Ultra Rapid Speed of Onset Demonstrated in Phase 3 Profile May Transcend The Traditional Boundaries of Older Legacy Intranasal Migraine Approaches Placebo (n=646) 59%** % of Patients with Pain Relief 60 Zavegepant 10 mg (n=623) INTRANASAL ZAVEGEPANT 10 mg demonstrated ultra-rapid onset of pain 50 relief that was superior to placebo 40 beginning at 15 minutes after a single 30%** dose (**p < 0.0015). 30 The impressive efficacy, safety and 16% ** After zavegepant treatment, these tolerability profile highlights the 20 patients who previously experienced potential to usher in a new era of 10 moderate to severe pain achieved non-oral CGRP targeting migraine reduction to mild or no pain. therapies that may transcend the 8% 20% 50% traditional boundaries of older 0 15 min 30 min 120 min legacy intranasal migraine approaches. 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 efficacy analysis set and CMH methods. Subjects using rescue medications at or before the assessment, and subjects not providing data, are classified as failures. ** Statistically significant vs. placebo in prespecified hierarchical testing which controlled type-1 error (data presented are mean ± asymptotic standard error). February 2022 BIOHAVEN INVESTOR PRESENTATION 24
CGRP CGRP Portfolio Expansion/Lifecycle Management First and only ACUTE AND migraine PREVENTION medication to treat and prevent FUTURE New indications Child and adolescent age PEDIATRICS groups CGRP MEDIATED: Psoriasis NON-MIGRAINE Asthma DISEASES Undisclosed Post-traumatic Headache Trigeminal Neuralgia MIGRAINE ADJACENCIES Undisclosed Sinusitis February 2022 BIOHAVEN INVESTOR PRESENTATION 25
G L U TA M AT E | M P O Biohaven Next-Generation Pipeline Platforms
GLUTAMATE Troriluzole: Targeted Lead-Indication Development Strategy Lead indications across an array of potential neurologic and neuropsychiatric indications Friedreich’s Ataxia Sporadic Ataxia Trichotillomania Spinocerebellar Ataxia Obsessive-Compulsive (SCA) Disorder (OCD) Other Ataxias Hoarding Disorder Essential Tremor February 2022 BIOHAVEN INVESTOR PRESENTATION 27
GLUTAMATE BHV-4157 Troriluzole Treated SCA Patients: Benefits Seen in Patients Treated for 1 Year Compared to Matched Ashizawa Natural History Cohort • Post-hoc analysis of patients enrolled in long- SCA Patients on Troriluzole1 after 48 weeks vs Natural History Cohort term extension of Phase 2b/3 troriluzole SCA trial Least Squares Mean2 Change in Total SARA Score (from baseline ± SE) • Primary efficacy endpoint: change from baseline in the Total SARA Score after 48 weeks • Patients from BHV4157-201 trial versus eligibility Difference: -1.41 ± 0.411 (95% confidence interval of criteria matched Ashizawa Natural History cohort: -2.22 to -0.60) suggesting • SCA Genotype therapeutic benefits of troriluzole (p=0.0007) • SCA1, SCA2, SCA3, SCA6 1. Matched on eligibility criteria • Age at baseline: 18 to 75 years of age 2. ANCOVA model with fixed effects for cohort, sex, & SCA genotype with age and baseline SARA • Gender scores as covariates 3. Ashizawa, T., et al. (2013). "Clinical characteristics of patients with spinocerebellar • SARA Score at baseline: ≥ 8 and ≤ 30, and ataxias 1, 2, 3 and 6 in the US; a prospective observational study." Orphanet J Rare Dis 8: 177 • Initial SARA gait item score ≥ 2 Troriluzole Study (BHV4157-201) Patient Benefits in LT Extension Supports Advancement to Phase 3 SARA: Scale for the Assessment and Rating of Ataxia February 2022 BIOHAVEN INVESTOR PRESENTATION 28
GLUTAMATE BHV-4157 Troriluzole: Phase 3 Randomized Controlled Trial in SCA • Post-hoc analysis of patients enrolled in long- term extension of Phase 2b/3 troriluzole SCA trial Screening Randomization Extension Phase Phase Phase 6 weeks 48 weeks 48 weeks • Primary efficacy endpoint: change from baseline in f-SARA Score after 48 weeks (FDA aligned) Troriluzole 200 mg QD • Trial design informed by Phase 2 study • SCA genotypes (SCA1, SCA2, SCA3, Troriluzole R 200 mg QD SCA6, SCA7, SCA8, SCA10) • Sample size: 230 subjects Placebo QD • Randomization: 1:1 • Troriluzole 200 mg QD vs. Placebo QD Troriluzole Study (BHV4157-206): Single Registrational Study, topline in 2022 SCA: Spinocerebellar Ataxia, f-SARA: Functional Scale for the Assessment and Rating of Ataxia February 2022 BIOHAVEN INVESTOR PRESENTATION 29
GLUTAMATE BHV-4157 Troriluzole Treated OCD Patients: Strong Signal Observed in Phase 2 POC Supports Advancement to Phase 3 STUDY BHV4157-202 Table 1: Troriluzole Effect on OCD in Phase 2/3 Trial1 Patients with moderate-to-severe OCD (Y-BOCS score Week ≥ 21) and inadequate response to standard of care Y-BOCS Total Change from Baseline 4 8 12 (N=115a, 111b) (N=108a, 96b) (N=102a, 99b) SAMPLE SIZE a. Placeboa -2.9 -3.6 -4.9 226 subjects b. Troriluzoleb -3.4 -5.1* -5.9 RANDOMIZATION p-value 0.451 0.041 0.220 1:1 1. BHV-4157-202 Final Unblinded Analysis YBOCS Total Change from Baseline by Week LSMeans from MMRM Model MITT Data Set DOSE Table 2: Troriluzole Effect on Patients with Severe OCD1 Troriluzole 200 mg QD vs Placebo QD (in patients on standard of care) Week Y-BOCS Total Change from Baseline 4 8 12 PRIMARY OUTCOME (N=47c, 49d) (N=45c, 42d) (N=43c, 44d) Y-BOCS, a precedented outcome measure accepted a. Placeboc -3.5 -3.1 -4.6 by FDA b. Troriluzoled -4.1 -6.0* -7.0 p-value 0.584 0.035 0.084 1. Patients at baseline with median Y-BOCS total scores > 26 (severe OCD symptoms). * p < 0.05 versus placebo Y-BOCS, Yale-Brown Obsessive Compulsive Scale February 2022 BIOHAVEN INVESTOR PRESENTATION 30
GLUTAMATE BHV-4157 Troriluzole: Two Phase 3 Trials Ongoing in Obsessive- Compulsive Disorder Two Phase 3 Studies BHV4157-302 (US only) BHV4157-303 (global) Screening Randomization Extension Study Phase Phase (BHV4157-209) Key Entry Criteria 42 days 10 weeks 48 weeks Moderate-to-severe OCD Inadequate response to SOC SOC + Troriluzole Design (identical for each Ph 3 study) 280 mg QD Troriluzole Sample size: 600 subjects R 280 mg QD Randomization 1:1 SOC + Troriluzole 280 mg vs. placebo, 1x daily Placebo QD Adjunctive therapy to SOC Primary Outcome Y-BOCS Troriluzole OCD Global Phase 3 program was initiated in 1Q21 OCD, obsessive-compulsive disorder; SOC, standard of care; Y-BOCS, Yale-Brown Obsessive-Compulsive Scale (FDA accepted outcome measure), LPFV, last patient first visit; LPLV, last patient last visit February 2022 BIOHAVEN INVESTOR PRESENTATION 31
MPO BHV-3241 Verdiperstat: Rationale for Studying in ALS • 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 ALS disease mechanisms mediated by peripheral myeloid cells, including those that migrate into the brain as well as those that remain in the periphery, suggesting relevance of MPO as a therapeutic target at both sites • Human ALS patients exhibit microglial activation/neuroinflammation measured by [11C]-PBR28 TSPO PET • Verdiperstat has demonstrated the ability to decrease TSPO signal in neurodegenerative disease patients • Neuroinflammation is increased in ALS ([11C]-PBR28 TSPO Imaging) Increased in ALS vs. controls Co-Localizes with Cortical Thinning TSPO PET, translocator protein - positron emission tomography; 1. Alshikho MJ, et al. Ann Neurol. 2018 Jun;83(6):1186-1197 February 2022 BIOHAVEN INVESTOR PRESENTATION 32
MPO BHV-3241 Verdiperstat: Phase 3 Healey ALS Platform Trial Ongoing DESIGN • Sample size: 160 subjects • Randomization: 3:1 Screening Phase Randomization Phase • Dose: 600 mg BID vs Placebo 6 weeks 24 weeks • Primary outcome measure: ALS Functional Rating Scale — Revised (ALSFRS-R) • Sites: 50 sites in the U.S. Verdiperstat 600 mg BID STATUS • Completed enrollment in 4Q2021 R COLLABORATOR Placebo BID Verdiperstat Study (BHV-3241) in Amyotrophic Lateral Sclerosis (ALS): Completed Enrollment February 2022 BIOHAVEN INVESTOR PRESENTATION 33
M AT E ™ | M O D E ™ | A R M ™ | T D P - 4 3 | U C 1 M T | T R P M 3 Biohaven Labs
BIOHAVEN LABS Customizable Bispecific Platforms MATE™ MoDE™ ARM™ Molecular Degraders of Extracellular Multimodal Antibody Therapy Enhancers Antibody Recruiting Molecules Proteins Directed Antibody Conjugation Extracellular Protein Degradation Directing Immune Engagement Next-generation protein Targets pathogenic extracellular Recruits endogenous drug conjugation targets for degradation immunoglobulin to bind a specific target ADCs, antibody-guided Reduction/elimination of target degraders damaging proteins Potential for improved ROA* vs biologics *ROA = Route of Administration February 2022 BIOHAVEN INVESTOR PRESENTATION 35
BIOHAVEN LABS MATE™ Provides Unparalleled Versatility, With Off-the-Shelf Conjugation To Add Any Molecule To An Antibody Any Molecule Any Antibody1 MATE • scFv conjugation • Cell surface target • Cytotoxic payload • Soluble target • Small molecule • Pathogen antigen • Peptide • Protein • Nucleic acid Homogeneous conjugates Applications in: Oncology (ADCs), Infectious Disease (COVID), Immunology (IgA Nephropathy degraders), etc. 1. IgG1, IgG2, or IgG4 February 2022 BIOHAVEN INVESTOR PRESENTATION 36
BIOHAVEN LABS MoDE™ Utilizes the Patients’ Liver to Clear Unwanted, Disease-Causing Proteins MoDE small molecules bind extracellular target proteins and cause them to be removed from the body through the liver Pathogenic target • Harnesses the body’s own machinery for degrading proteins protein and MoDEs in circulation • Extracellular protein targets are eliminated via the asialoglycoprotein receptor (ASGPR) Binds liver Binds protein (ASGPR) target • Protein targets are degraded via endolysosomal proteolysis February 2022 BIOHAVEN INVESTOR PRESENTATION 37
BIOHAVEN LABS ARM™ Enhances Recruitment of NK Cells and Increases Killing of Multiple Myeloma Cells Human Natural Killer (NK) BHV-1100 BHV-1100 CD38 Immunoglobulin (Ig) Cell Therapy CD38 ARM™ Targeting Therapy Off-The-Shelf CIML Multiple Mult Multipl NK Cell CIML Myeloma iple e NK Cell Cell Myelo Mye lom ma Fc Cell a Cell Fc Receptor CIML cytokine induced BHV-1100 is a memory like bispecific BHV-1100 + CIML NK cell molecule binding to both Ig and CD38 Binds to CD38 First patient completed Binds to human receptor on the therapy at Dana Farber immunoglobulin (Ig) surface of multiple Antibody Recruiting Molecule (ARM™) myeloma cells February 2022 BIOHAVEN INVESTOR PRESENTATION 38
BIOHAVEN LABS BHV-2100: First-In-Class TRPM3 Antagonist for Neuropathic Pain TRPM3 is a novel druggable target in the TRP channel family1 BHV-2100 HIGHLIGHTS Small molecule with good Preclinical models and human oral bioavailability and genetic validation implicate TRPM3 animal PK in pain signaling1,2 Preclinical efficacy in models of diabetic peripheral Knocking out or antagonizing neuropathic pain, TRPM3 reduces pain behaviors in chemotherapy-induced peripheral neuropathy, and multiple animal models of nerve injury neuropathic pain with diverse etiologies3,4 Currently in IND-enabling studies with plans to enter the clinic in 1H2023 Targeting TRPM3 may avoid some of the on-target toxicities seen with antagonizing other TRP channels1 BHV-2100 is a potential breakthrough as a non-opioid treatment for neuropathic pain 1: Koivisto et al, Nature Reviews Drug Discovery 2022; 2: Lötsch et al, J. Molecular Sciences 2020; 3: Vandewauw et al, Nature 2018; 4: Su et al, J. Neuroscience 2021 February 2022 BIOHAVEN INVESTOR PRESENTATION 39
BIOHAVEN LABS 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 Demonstrated the ability disease models with better reduce markers of activity than anti-TNF inflammation by UC1MT Strong global patent Evidence of protection of Anti-MT molecular antibody therapeutic in mechanism several disease areas February 2022 BIOHAVEN INVESTOR PRESENTATION 40
BIOHAVEN LABS Novel Development Program Targeting TDP-43 TDP-43: TAR-DNA protein-43 is Implicated in Neurodegeneration1 • TDP-43 is a multifunctional nucleic acid-binding protein TDP-43 • Mutations cause familial and Small molecule inhibitors of sporadic amyotrophic lateral TDP-43 aggregation sclerosis (ALS) and identified frontotemporal dementia Treatment enhances survival (FTD) in TDP-43 preclinical models • Aggregates are the neuropathological hallmark of ALS-FTD spectrum disorders Klim JR. Trends Neurosci. 2021. Small molecule inhibitors of TDP-43 for ALS and FTD February 2022 BIOHAVEN INVESTOR PRESENTATION 41
Corporate Overview
Pfizer Deal Financials Strong Revenue Growth and Capital Position $1.2+ BILLION REVENUE CAPITAL TOTAL UPFRONT AND MILESTONE VALUE $190M $526M $520M** $500M $125M STRUCTURE 4Q2021 NET REVENUE 3Q2021 – CASH, AND PFIZER AVAILABLE FROM BIOHAVEN RUNS R&D GLOBALLY NET REVENUE SINCE LAUNCH MARKETABLE SECURITIES PAYMENT SIXTH STREET *Estimated **Before 4Q2021 Cash Spend 1Q2022 FINANCING PFIZER EXECUTES COMMERCIALIZATION EX-U.S. $500M $190M RECEIVED IN CASH & STOCK AT 25% MARKET PREMIUM (~$173 SHARE) UP TO $740M $136M IN SALES AND OTHER MILESTONES DOUBLE-DIGIT $93M ROYALTIES ON EX-U.S. NET SALES PFIZER TO PAY RELATED EX-U.S. $44M BMS AND RPI ROYALTIES $10M $35M $18M 2Q20 3Q20 4Q20 1Q21 2Q21 3Q21 4Q21 February 2022 BIOHAVEN INVESTOR PRESENTATION 43
Highly Experienced Drug Development Leadership Elyse Stock, MD CMO Dave Stock, PhD Kim Gentile Biostats Clinical Operations 25+ Ashwini Ghatpande, MS Marianne Frost, MA Medical Writing Regulatory YEARS Amy O’Donnell, JD, MD PV: Safety NEURO BJ Jones, MBA Commercial AVERAGE EXPERIENCE INNOVATION Cliff Bechtold, MS Donnie McGrath, MD GM Ireland BD/BioShin Ed Kim, MD Warren Volles, JD Medical Affairs Legal Charlie Conway, PhD Rajesh Kumar, PhD CSO CMC Gene Dubowchik, PhD Chemistry February 2022 BIOHAVEN INVESTOR PRESENTATION 44
2022 Milestones • Continue U.S. market growth of NURTEC® ODT in migraine • Broaden NURTEC ODT indications and market potential through lifecycle expansion studies • Submit NDA for Zavegepant • Advance CGRP proof of concept studies • Psoriasis • Asthma • Implement global collaboration with Pfizer • EU approval • Market launches • Announce topline data • Troriluzole for SCA and OCD • Verdiperstat for ALS • File multiple INDs from Biohaven Labs February 2022 BIOHAVEN INVESTOR PRESENTATION 45
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