Investor Presentation - NEUROINNOVATION We Demand More for Patients - Biohaven Pharmaceuticals
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NEUROINNOVATION ®
We Demand More for Patients.
Investor Presentation
March 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, BHV-1200, Taldefgrobep Alfa, and BHV-7000 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, UC1MT and Kv7. 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, 2021, filed with the Securities and Exchange Commission on Feb 25, 2022, 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. MARCH 2022 BIOHAVEN INVESTOR PRESENTATION
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.
$190M $463M 1.6M
TRxs OF
4Q 2021 REVENUE FY 2021 NURTEC® ODT
TO DATE
NDA
DEEP
FILING
1H22 PIPELINE
BREAKING NEWS
NEURO
CHANNEL
B I O S C I E N C E S
INNOVATION
®
RIMEGEPANT Kv7 MYOSTATIN
EMA CHMP PLATFORM TARGETING AGENT
POSITIVE OPINION ACQUISITION LICENSE
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION
Strategic Platforms & Deep Pipeline PRECLINICAL PHASE 1 PHASE 2 PHASE 3
Filing for Approval
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 | SPINOCEREBELLAR ATAXIA (SCA)
GLUTAMATE Troriluzole
BHV-4157 | OBSESSIVE-COMPULSIVE DISORDER (OCD)
MPO Verdiperstat BHV-3241 | AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MYOSTATIN BHV-2000 | SPINAL MUSCULAR ATROPHY (SMA)
CD38 BHV-1100 MULTIPLE MYELOMA
Kv7 Kv7 BHV-7000 REFRACTORY FOCAL SEIZURES
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 5
Ellie Actual Nurtec® ODT Patient
Source: 1. TRX numbers 2/18/22, IQVIA SMART, accessed 2/28/22. Market definition for class share is based on Nurtec® ODT, Ubrelvy®, and Qulipta®
CGRP
Strong and Steady Growth
$526M $190M
Launch to date
net product revenue1
$136M
$93M
$44M
$35M
$10M $463M FY21
$18M
2Q20 3Q20 4Q20 1Q21 2Q21 3Q21 4Q21
1. 2/25/2022 Biohaven Earnings Call and Press Release — NURTEC® ODT achieved net product revenue of $190 million
for the fourth quarter of 2021. Net product revenue for NURTEC ODT in 2021 totaled approximately $463 million, resulting
in launch to date net product revenue of $526 million with over 1,600,000 prescriptions filled since initial product launch in
March 2020.
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 8
CGRP
Oral CGRP Class Continues to Show Robust Market Growth
Nurtec® ODT leads in TRx at 50.4% share
1 Total Rx Volume (3/11)¹ 2 Total Rx Share (3/11)1
65%
60%
34,202 Nurtec ODT 55%
50% 50.4% Nurtec ODT
27,781 Ubrelvy 45%
40% 41.0% Ubrelvy
35% Prevention
Approval
30%
25%
20%
15%
5,828 Qulipta 10%
8.6% Qulipta
5%
0%
10/16
10/30
11/13
11/27
12/11
12/25
10/15
10/29
11/12
11/26
12/10
12/24
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
2/4
3/4
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
2/18
10/15
10/22
10/29
11/12
11/19
11/26
12/10
12/17
12/24
12/31
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
1/21
1/28
2/11
2/18
2/25
3/11
5/7
7/2
7/9
8/6
9/3
1/7
2/4
3/4
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 – 3/11/22, IQVIA SMART, accessed 3/21/22. Note: Market definition for share calculations is based on Nurtec ODT, Ubrelvy, and Qulipta
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION
CGRP
Oral CGRPs: Fastest Growing Class in Large U.S. Migraine Market
60.0%
50.0%
Triptans
40.0%
% U.S. Migraine Market Share
Topiramate
In 2021
oral CGRPs reached
30.0% ~$1B in net sales while
only accounting for
5-6% of migraine scripts
20.0%
Total migraine market
expected to grow to
CGRP mAbs
~40+ million scripts
10.0%
Oral CGRPs
Botox
0.0%
Jan Ma r Ma y Jul Sep Nov Jan Ma r Ma y Jul Sep Nov Jan Ma r Ma y Jul Sep Nov Jan
2019 2019 2019 2019 2019 2019 2020 2020 2020 2020 2020 2020 2021 2021 2021 2021 2021 2021 2022
Source: IQVIA NPA monthly TRx through Feb 2022
MARCH 2022 BIOHAVEN INVESTOR PRESENTATIONCGRP
Oral CGRPs Have Significant Growth Opportunity Ahead vs Triptans
TRx Volume vs Triptans¹ NBRx Volume vs Triptans²
570,752
Quarterly Quarterly
3,891,364
NBRx volume NBRx volume
25%
15%
144,960
708,454
4Q21 4Q21 4Q21 4Q21
CGRP orals3 Triptans CGRP orals3 Triptans
Source: 1. TRX numbers cover 4Q2021, IQVIA XPO, accessed 1/17/22. 2. NBRx numbers cover 4Q2021, IQVIA XPO, accessed 1/17/22. 3. CGRP orals = Nurtec ODT, Ubrelvy, and Qulipta.
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 110
100,000
120,000
140,000
160,000
20,000
40,000
60,000
80,000
5/25/2018
MARCH 2022
6/15/2018
7/6/2018
7/27/2018
8/17/2018
9/7/2018
9/28/2018
10/19/2018
11/9/2018
11/30/2018
12/21/2018
1/11/2019
2/1/2019
2/22/2019
3/15/2019
4/5/2019
4/26/2019
5/17/2019
6/7/2019
6/28/2019
7/19/2019
8/9/2019
8/30/2019
9/20/2019
10/11/2019
11/1/2019
11/22/2019
12/13/2019
1/3/2020
1/24/2020
2/14/2020
3/6/2020
3/27/2020
4/17/2020
5/8/2020
5/29/2020
6/19/2020
CGRP Weekly TRx
7/10/2020
BIOHAVEN INVESTOR PRESENTATION
7/31/2020
8/21/2020
9/11/2020
10/2/2020
10/23/2020
11/13/2020
12/4/2020
12/25/2020
Source: IQVIA SMART: TRx Volume to 3/11/2022, accessed 3/21/22. 1. Oral CGRP = Ubrelvy, Qulipta, Nurtec® ODT; 2. Injectable mAb = Emgality, Ajovy, Aimovig
1/15/2021
2/5/2021
2/26/2021
3/19/2021
4/9/2021
4/30/2021
5/21/2021
6/11/2021
7/2/2021
7/23/2021
8/13/2021
9/3/2021
9/24/2021
10/15/2021
11/5/2021
11/26/2021
12/17/2021
1/7/2022
1/28/2022
Injectable mAb2
Oral CGRP1
Orals CGRPs Have Driven CGRP Growth in 2020 and Beyond (vs mAbs)
2/18/2022
12
3/11/2022CGRP
Rimegepant Global Market Opportunity
2 Prevention
(Dual Therapy)
4 1
Acute Migraine 15+ Acute or Dual
Thru 2022/2023
APPROVALS APPROVALS SUBMISSIONS
Europe
Dual1 | POSITIVE OPINION
Japan
Dual1 | 1H23
Lebanon
Acute | 2022/23 China
Acute | 2H22
Israel Kuwait
Dual1 | APPROVED Acute | APPROVED
Korea
Acute | 2H22
Qatar
Bahrain Acute | 2022/23
Acute | 4Q20
US
Dual1 | APPROVED Saudi Arabia
Acute | 1Q21 United Arab Emirates
Acute | APPROVED
Singapore
Dual1 | 2H22
APPROVALS Oman
Acute | 2022/23
SUBMISSIONS
NDA PLANNED 1. Includes dual-therapy: acute and prevention
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 13CGRP
Delivering Dual Therapy Innovation
to Patients with Migraine Around the Globe
GLOBAL PARTNERSHIP
>1B
EU POSITIVE OPINION
MULTI-BILLION-
Market Size: 112 million
DOLLAR MARKET
OPPORTUNITY
CHINA/KOREA Global Patients
LIVING WITH MIGRAINE
Positive Phase 3 Data
Market Size: 120 million
ROW: Japan | Middle East …
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 14CGRP
CHINA/KOREA: Rimegepant 4th Positive Pivotal
Phase 3 Study - Profile Consistent with Prior Trials
• Study met coprimary
Rapid Onset Pain Relief Demonstrated by Rimegepant endpoints with superiority
over placebo (p < 0.0001):
• 2 hr Pain Freedom
% of Patients with Pain Relief1
Placebo (n=666) • 2 hr Freedom from Most
90 Rimegepant 75 mg (n=674)
75% *
Bothersome Symptom
80
70
67%*** • Rapid onset and durable
58%* efficacy profile superior to
60
placebo (p < 0.0001) on:
50
37%* • 2 hr Pain Relief
40
30 • 2 hr Normal Function
20 • No Need for Rescue Med
10 within 24 hr
0 • 2 to 24 hr Pain Freedom2
45 min 90 min 2 hr 3 hr
Time • 2 to 48 hr Pain Freedom2
1. Pain Relief defined as patients having mild-pain or no-pain during specified interval. 2. Sustained Pain Freedom is defined as patients having mild-to-no-pain at 2 hours and
continuing as such to the end of the specified interval. Estimates computed using the mITT subjects and CMH methods. Subjects using rescue medications at or before the
assessment, and subjects not providing data, are classified as failures. *** significant p < 0.0001 vs. placebo; * nominal p < 0.001 vs. placebo
Study BHV3000-310
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 15CGRP
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.
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 16CGRP
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); PCGRP MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 19
CGRP MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 20
CGRP Nurtec® ODT Commercial Success MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 21
CGRP
Broad Commercial Coverage:
High Impact Commercial PBM and Health Plan Wins
89%
COVERAGE
240M+
TOTAL COVERED LIVES
IN ALL CHANNELS
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 22CGRP
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 23“I am a retired Green Beret from the Army – 21 years, 7 combat tours. I have a TBI and bad migraine headaches… I started to take Nurtec ODT and I think it has changed my life. I used to live in my closet… between migraine headaches and all my other problems after 7 combat tours, I had lost all hope. Nurtec has made my life so much better. I have now started Blue Ridge Safe House. It’s a non-profit to help active duty guys like me.” — Greg, living with migraine
CGRP 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 26CGRP
Intranasal Zavegepant Achieved Positive Phase 3 Data
The only intranasal CGRP receptor antagonist
Ultra rapid migraine relief within 15 minutes
Ideal for patients with nausea/vomiting
(approx. 50%)
NDA Submission 1H2022
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 27CGRP
BHV-3500 Zavegepant: NDA Submission 1H2022
Superior chemical attributes
• Potent antagonist at the human CGRP receptor
• Highly soluble and high free fraction
• U.S. composition of matter protection to March 20341
Multiple potential routes of delivery
Nasal, inhalation and oral
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 The impressive efficacy, safety and
of pain freedom and freedom from most bothersome symptom at 2 hours tolerability profile highlights the
potential to usher in a new era of
non-oral CGRP targeting migraine
Recently achieved positive Phase 3 data in 2nd pivotal therapies that may transcend the
study replicating/extending prior results traditional boundaries of older
legacy intranasal migraine
Zavegepant 10 mg met primary endpoints and showed ultra-rapid onset pain relief approaches.
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 28CGRP
BHV-3500 Intranasal Zavegepant: Potential to Usher in a New Era of
Non-oral CGRP Targeting Migraine Therapies
Placebo (n=646) 59%**
% of Patients with Pain Relief
60 Zavegepant 10 mg (n=623)
NDA SUBMISSION 50
ON SCHEDULE 40
30%**
1H 2022 30
20 16% **
10
8% 20% 50%
0
15 min 30 min 120 min
INTRANASAL ZAVEGEPANT 10 mg
demonstrated ultra-rapid onset of pain relief
that was superior to placebo beginning at
15 minutes after a single dose (**p < 0.0015).
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 29CGRP
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 30Deep Pipeline: Robust Portfolio Across All Stages of Development
TALDEFGROBEP
ZAVEGEPANT TRORILUZOLE VERDIPERSTAT BHV-7000
ALFA
• Approved for Acute • Positive Phase 3 • Phase 3 in OCD • Phase 3 in ALS • Phase 3 in SMA • In clinic 2022 10+ preclinical
and Prevention data intranasal programs in:
• Phase 3 in SCA
• Positive Phase 3 • Oral studies ongoing • Infectious disease
data China/Korea • Multiple studies in • Neuropathic pain
• Pediatric Phase 3 pain adjacencies • Neurodegeneration
studies on going • Multiple studies in • Neuroimmunology
• Non-migraine non migraine
indications being • Auto immune
explored • Epilepsy
• Pfizer Ex-US
Commercialization
opens door to
1B patients
CGRP GLUTAMATE MPO MYOSTATIN Kv7 ACTIVATOR PRECLINICAL
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 31Kv7 Platform Acquisition:
3rd-Generation Ion Channel Activators for Neurologic Disease
Best in Class Validated MOA Multiple Therapeutic
Potential for Epilepsy Applications
Wide therapeutic index and Fast follower strategy to accelerate Potential to treat seizure, bipolar
patents up to 2039 to patients disorder, pain and others
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 32Taldefgrobep Alfa In-licensed from BMS:
Potential Adjuvant Therapy for Neuromuscular Disorders
Target Phase 3 in 2022
Strong preclinical and clinical data to support
activity in Spinal Muscular Atrophy (SMA)
Clinical trial experience including
demonstrated safety in adults/pediatrics
Extends neuroscience
Issued IP through 2038, without extensions R&D collaboration
between organizations
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 33Clinical-Stage Milestones Milestone achieved
DRUG NAME INDICATION 1H2021 2H2021 1H2022 2H2022
Migraine prevention Approval
Migraine acute/prevention Europe Filing 1Q EU Positive Opinion
Migraine acute (China/Korea) Topline China/Korea Filing
Migraine (intranasal) Topline US Filing
Zavegepant
Small molecule/NCE
Migraine (oral) Start Phase 3
Spinocerebellar ataxia Topline
Troriluzole
NCE prodrug of riluzole
Obsessive-compulsive disorder Complete Enrollment
Verdiperstat Amyotrophic lateral sclerosis Complete enrollment Topline
NCE oral MPO inhibitor
Taldefgrobep Alfa Spinal muscular atrophy Start Phase 3
Anti-myostatin adnectin
BHV-7000 Focal seizures Clinic Start
Kv7 channel modulator
BHV-1100 Multiple myeloma Start Phase 1
ARM combo
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 34BIOHAVEN LABS
Bringing Value to Patients and Shareholders for Years to Come
Preclinical Novel Novel Indications with
Programs Small Molecule Large Molecule High Unmet
Approaches Approaches Medical Need
10+ 4 6 12+
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 35Epilepsy Affects Millions of Children and Adults,
Significantly Impacting Quality of Life
of epilepsy patients have focal epilepsy, whether
60% adult or child
25% of
of adult focal patients are refractory needing 2+
33% meds for difficult to control, severe seizures
children are
refractory
3.5M Seizures impact 1.2% of
adults and 0.6% of
children 1 out of 26
Risk profiles of anti-
seizure medications and
high rates of treatment-
Approximate resistant disease require
people will develop
number of 4thmost common epilepsy at some point highly individualized
epilepsy patients in neurological disorder management in adults
the U.S. alone
in 2021 Each year, more than
1 in 1,000 people SUDEP is the sudden, unexpected death of someone
with epilepsy die from with epilepsy, who was otherwise healthy.
SUDEP
CDC, NIH, Biohaven Research
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 36Kv7 Potassium Channels Are a Critical Regulator of CNS Hyperexcitability
Potassium
Sodium Channels A key missing piece in epilepsy treatment
Channels
Ex: cenobamate,
carbamazepine,
lacosamide
Ex: BHV-7000 and Clinically validated mechanism of action
XEN1101, ezogabine for treating focal epilepsy
GABA &
Strong rationale for development in
Glutamate
adjacent indications
Calcium
Channels
Ex: Ex: levetiracetam,
benzodiazepines,
topiramate
pregabalin BHV-7000: Potentially best-in-class
Kv7 potassium channel activator
MARCH
MARCH2022
2022 BIOHAVEN INVESTOR PRESENTATION 37Biohaven’s Kv7 Platform: BHV-7000 Is a Potentially
Best-in-Class Kv7 Potassium Channel Activator for Epilepsy
BHV-7000
Lead Asset from Novel Platform Highly Differentiated Profile Advancing to Clinic in 2022
• Broad series of structurally-distinct Kv7 • Potent activator of the Kv7.2/7.3 ion • Clinical studies expected in 2022
targeting compounds differentiated channel • Potential best-in-class clinical profile
from ezogabine and XEN1101 • GABA activity “dialed-out” to limit for refractory focal epilepsy
• BHV-7000 is highly effective in potential side effects such • FDA granted Rare Pediatric Disease
preclinical epilepsy assays as somnolence and fatigue designation for KCNQ2 epileptic
• BHV-7000 has patent life to 2039 with • Wide therapeutic index encephalopathy
broad geographical coverage • Stable to photooxidation
• Follow-on compounds have potential
for additional indications: other
epilepsy types, pain, and affective
disorders
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 381st & 2nd Gen Kv7 Activators Show Clinical Anti-seizure POC, But
Off-target Activities, Opportunity for 3rd Gen Kv7’s To Differentiate
EZOGABINE XEN1101 BHV-7000
• Unstable when exposed to light • XEN1101 and Ezogabine are • BHV-7000 is a potent activator of
significantly greater GABAA receptor Kv7 channels
• Label warnings for skin discoloration
activators than BHV-7000 in vitroa • BHV-7000 is effective and well-
• Black box warning for retinal
abnormalities/vision loss • GABAA receptor activation contributes to tolerated in preclinical seizure assays
GABAGABA
A Positive
A Positive
Allosteric
Allosteric
Modulation
Modulation
somnolence, dizziness, fatigue, diplopia
✱✱ ✱✱
50
60 60 ✱✱ ✱✱
**
Therapeutic Index
% Increase from baseline
from baseline
10 µM 10 µM BHV-7000
BHV-7000 40
GABAA Activation a
**
10 µM 10
EZOµM EZO
(% Baseline)
40 40 30
10 µM 10 µM XEN1101
XEN1101
20
% Increase
20 20
10
0 0
Ezogabine XEN1101 BHV-7000 Ezogabineb XEN1101b BHV-7000a
FDA Drug Safety Communication: Potiga (ezogabine) [04-26-2013] ** significantly different from BHV-7000, all tested at 10 µM
BHV-7000 is chemically stable BHV-7000 is selective for Kv7 BHV-7000 has a wide therapeutic
a
to photooxidation over GABAA receptors index preclinically a
a. Biohaven data on file (2022); b. Calculated as ratio of TD50 (rotarod) to ED50 (MES seizure assay) data presented by Xenon at Epilepsy Foundation Pipeline Conference, San Francisco (Feb 2018)
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 39BHV-7000 Demonstrates Potent Anti-seizure Efficacy and a Wide
Therapeutic Index, a Distinct Profile From 1st & 2nd Gen Kv7 Activators
a a
BHV-7000
BHV-7000 in rat MES, PO EZOGABINE
EZO in rat MES, PO
Protection (%) 100 3 100 3
Protection (%)
Neurological Deficit
Neurological Deficit
Neurological Score
Neurological Score
75 75
% Protection
% Protection
2 2
ED50 = 0.5 mg/kg
50=0.5mg/kg EDED = =20
50 50 20 mg/kg
mg/kg
50 TI>40x 50 TI 40x 1
< 3x 1
Seizure
Seizure
Therapeutic Therapeutic
25 25
Index a Index a
0 0 0 0
0 .0 1 0 .1 1 10 100 0 .0 1 0 .1 1 10 100
Dose (mg/kg) Dose (mg/kg)
Efficacy
Seizure BHV-7000
Protection n=10/group
BHV-7000 Seizure Protection EZOGABINE
Efficacy EZO n=6/group
Neurological
NS BHV-7000Deficit BHV-7000
n=10/group Neurological Deficit EZOGABINE
NS EZO n=6/group
BHV-7000 demonstrates a wide therapeutic index (>40x) which is much wider than reported
for both ezogabine (3rd Generation BHV-7000 Exhibits Highly Differentiated Profile,
Potentially Best-in-Class Kv7 Channel Activator for Epilepsy
Ezogabine XEN1101 BHV-7000
Activator, with Clinical and Activator, with Clinical and Activator, with Potent
Kv7.2/7.3
Preclinical Anti-Seizure Preclinical Anti-Seizure Preclinical Anti-Seizure
Activator
Activity Activity Activity
GABAA Activity GABAA activity present GABAA activity present Negligible GABAA activity
“dialed-out”
Wide Therapeutic 3x reported a,b 6x reported a >40x b
Index
a. Calculated as ratio of TD50 (rotarod) to ED50 (MES seizure assay) data presented by Xenon at Epilepsy Foundation Pipeline Conference, San Francisco (Feb 2018); b. Biohaven data on file (2022)
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 41Taldefgrobep Alfa:
Targeting a Differentiated Regulatory Pathway of Muscular Growth
The Most Advanced
Phase 3 Ready Myostatin Inhibitor
Mechanistically, taldefgrobep-myostatin complex
acts as ActRIIB receptor antagonist Safety confirmed with over 300 patients dosed in prior
clinical studies. Safety demonstrated in pediatrics
Myostatin negatively Taldefgrobep alfa binds to myostatin populations.
regulates muscle growth to inhibit signaling
Non-Clinical studies showed muscle and bone
improvements
POC for mechanism of action supported by clinical
data and multiple disease models
Near-term inflection point of 1H2022 Phase 3 study
start, limited additional work needed to support a BLA
submission
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 42G 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 44GLUTAMATE
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 45GLUTAMATE
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 46GLUTAMATE
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 47GLUTAMATE
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 48MPO
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 49MPO
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 50M AT E ™ | M O D E ™ | A R M ™ | T D P - 4 3 | U C 1 M T | T R P M 3 | K v 7 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 52BIOHAVEN LABS
MATE™ Provides Unparalleled Versatility, With Off-the-Shelf Conjugation
To Add Any Molecule To Many Antibodies
Any Molecule Many Antibodies1
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 53BIOHAVEN 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 54BIOHAVEN 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 55BIOHAVEN LABS
TDP-43 First-in-Class Small Molecule Inhibitors for
Neurodegenerative Disorders
TAR-DNA binding protein-43 (TDP-43) is Implicated in Neurodegeneration1 Program Highlights
First-in-class small molecule
• TDP-43 is a multifunctional inhibitors of TDP-43
nucleic acid-binding protein aggregation identified
• Mutations cause familial and Target engagement
sporadic amyotrophic lateral demonstrated in vitro:
sclerosis (ALS) and Active in competition assays,
ligand-protein NMR, and
frontotemporal dementia computational docking
(FTD)
Target validation achieved
• Aggregates are the in vivo: Treatment enhances
neuropathological hallmark of survival in TDP-43 preclinical
ALS-FTD spectrum disorders assays
Klim JR. Trends Neurosci. 2021.
Small molecule inhibitors of TDP-43 aggregation for ALS and FTD
1. Klim, JR. Trends Neurosci. 2021 Jun;44(6):424-440. doi: 10.1016/j.tins.2021.02.008. ALS, amyotrophic lateral sclerosis. FTD frontotemporal dementia. TDP-43,
transactive response DNA-binding protein 43. NMR, nuclear magnetic resonance
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 56BIOHAVEN LABS
UC1MT: First-in-Class Anti-Metallothionein Antibody
to Treat IBD and other Inflammatory Diseases
Program Highlights
Extracellular metallothionein (MT) is implicated in inflammatory diseases Target-engagement: UC1MT
and humanized versions of
• MT is a stress response Extracellular Metallothionein Binds to Lymphocytes UC1MT bind with high affinity to
protein synthesized during MT increases proliferative MT increases TNFα production by MT1 and MT2
infection, inflammation, and capacity of stimulated lymphocytes stimulated macrophage-like cells
autoimmune disease Mitogen
Biomarker efficacy: UC1MT
MT LPS MT reduces biomarkers of
• Extracellular MT acts as a Mitogen TNFα inflammation
pro-inflammatory agent and receptor
provokes chemotactic cell In Vivo POC demonstrated:
movement lymphocytes UC1MT treatment provides
therapeutic benefits in
• UC1MT blocks MT- established disease in multiple
induced chemotaxis UC1MT Demonstrates In Vivo Efficacy in Mouse IBD Assay
in vivo assays (with better
A 0 .5 ⇩Metallothionein
M T , D is ta l C o lo n(colon) B 8 T N F ,a
⇩TNF D is(colon)
• UC1MT demonstrates in vivo ta l C o lo n
activity than anti-TNF & anti-
POC in mouse IBD assays IL12)
Area
Area
0 .4
% p o s itiv e a r e a
% p o s itiv e a r e a
6
(e.g., adoptive T cell transfer test): 0 .3
Potential clinical candidates
% Positive
% Positive
4
• Decreases MT levels 0 .2 identified from humanized
• Reduces TNF-a levels 0 .1
2
therapeutic mAbs
0 .0 0
T
12
le
F
ve
TT
UC1MT treatment disrupts extracellular
12
le
F
ve
T
N
11MM
ic
TN
11MM
ai
ic
L
ai
-T
IL
-I
eh
eh
N
C
ti-
ti
ti-
ti
N
C
UUC
UCU
V
an
an
MT signaling and reduces inflammation
V
an
an
IBD, inflammatory bowel disease. TNF-a, tumor necrosis factor alpha. 1. Issued U.S. Patent #9,353,175 “Use of antagonists targeting metallothionein to treat intestinal
inflammation” M. DeVos (University of Gent), D. Laukens (University of Gent), L. Devisscher (University of Gent), and M.A. Lynes (University of Connecticut), 2016.
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 57BIOHAVEN LABS
TRPM3 First-in-Class Small Molecule Antagonists
for Neuropathic and Other Persistent Pain States
Program Highlights
TRPM3 is a novel druggable target in the TRP ion channel family1 Clinical Candidate BHV-2100:
• Preclinical models and human Small molecule with good oral
bioavailability and animal PK
genetic validation implicate
TRPM3 in pain signaling1,2 Preclinical efficacy from in
vivo assays for diabetic
• Knocking out or antagonizing peripheral neuropathic pain,
TRPM3 reduces pain behaviors chemotherapy-induced
peripheral neuropathy, and
in animal models of neuropathic
nerve injury
pain with diverse etiologies3,4
IND-enabling studies
• Targeting TRPM3 may avoid initiated
some of the on-target toxicities
Discovery Program focused on
seen with antagonizing other structurally distinct chemical series
TRP channels1 to provide optionality for therapeutic
approaches
BHV-2100 is a potential breakthrough as a non-opioid treatment for 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 58BIOHAVEN LABS
Kv7.2/7.3 Potassium Channel Activators For Epilepsy, PROGRAM HIGHLIGHTS
Affective Disorders & Pain Clinical Candidate BHV-7000:
Small molecule with good oral
Target: Kv7.2/7.3 Activators of Voltage-Gated Potassium Channels bioavailability and animal PK
• Kv7.2/7.3 is a voltage-gated Preclinical efficacy in
clinically-predictive assay for
potassium channel, 4 subunits focal epilepsy (maximal electric
each with 6 membrane spanning shock (MES))
domains together form ion pore
Wide therapeutic index
• Kv7.2/7.3 channel opening preclinically provides
creates ion flux (“M-current) opportunity
known to critically regulate
IND-enabling studies initiated
neuronal excitability
• Therapeutic opportunities: Discovery Program focused on
refractory focal epilepsy, serious chemically distinct molecules and
Kv7.3 (aka KCNQ3) greatly
Current (µA)
pediatric encephalopathy amplifies current when multiple disease targets
(KCNQ2-EE), Dravet & LGS combined with Kv7.2 (aka
KCNQ2) and contributes to
syndromes, Major Depressive neuronal excitability
Disorder, Pain and Migraine Kv7.2 Kv7.3 Kv7.2+3
(KCNQ2) (KCNQ3) (KCNQ2+3)
Clinically validated mechanism of action for treating focal epilepsy (ezogabine, XEN1101)
Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare central nervous system diseases considered serious epileptic encephalopathies
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 59Corporate Overview
Capital Position ($ Millions)
Cash, cash equivalents, and marketable securities
$367
@ December 31, 2021
Proceeds received January 4, 2022 from strategic collaboration
and equity purchase transactions with Pfizer
$500 ~$1B
IN LIQUIDITY
Cash immediately available to draw from Sixth Street financing $125
MARCH 2022 BIOHAVEN INVESTOR PRESENTATIONPfizer Deal Financials
Strong Revenue Growth and Capital Position
$1.2+ BILLION
REVENUE CAPITAL TOTAL UPFRONT AND
MILESTONE VALUE
$190M $526M $367M $500M $125M STRUCTURE
4Q2021 NET REVENUE 4Q2021 – CASH, AND PFIZER AVAILABLE FROM BIOHAVEN RUNS R&D GLOBALLY
NET REVENUE SINCE LAUNCH MARKETABLE SECURITIES PAYMENT SIXTH STREET
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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION 62Highly 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
MARCH 2022 BIOHAVEN INVESTOR PRESENTATIONTHE FUTURE
LOOKS BRIGHT!
2 Marketed Indications: Acute and Prevention
8 Phase 3
5 Phase 2
2 Phase 1
10+ Preclinical
MARCH 2022 BIOHAVEN INVESTOR PRESENTATION2022 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
• Submit multiple INDs from Biohaven Labs
MARCH 2022 BIOHAVEN INVESTOR PRESENTATIONThank you!
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