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.
Ellie Actual Nurtec® ODT Patient
$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
R I M E G E PA N T | Z AV E G E PA N T CGRP Platform Franchise
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 10CGRP
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 11CGRP
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 12CGRP
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 ‡PCGRP
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); PFebruary 2022 BIOHAVEN INVESTOR PRESENTATION 15
February 2022 BIOHAVEN INVESTOR PRESENTATION 16
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 18CGRP
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 19CGRP
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 20CGRP
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 21CGRP
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 22CGRP
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 23CGRP
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 24CGRP
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 25G 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 27GLUTAMATE
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 28GLUTAMATE
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 29GLUTAMATE
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 30GLUTAMATE
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 31MPO
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 32MPO
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 33M 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 35BIOHAVEN 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 36BIOHAVEN 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 37BIOHAVEN 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 38BIOHAVEN 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 39BIOHAVEN 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 40BIOHAVEN 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 41Corporate 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 43Highly 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 442022 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 45Thank you!
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