neurocrine.comneurocrine.com
JP Morgan Healthcare Presentation
January 11, 2021
Nasdaq: NBIX
Safe Harbor Statement and Non-GAAP Financial Measures
2
In addition to historical facts, this presentation contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but
are not limited to, statements related to: our preliminary unaudited financial information; the benefits to be derived from our products and product candidates; the
value our products and/or our product candidates may bring to patients; the continued success of INGREZZA; our financial and operating performance, our
collaborative partnerships; expectations regarding the impact of COVID-19 on our business, including patient and healthcare provider access to INGREZZA, our
ability to continue our ongoing clinical trials and other development activities, and to otherwise advance our business objectives; and the timing of completion of our
clinical, regulatory, and other development activities and those of our collaboration partners. Among the factors that could cause actual results to differ materially
from those indicated in the forward-looking statements are: risks and uncertainties associated with items that may be identified during the financial statement closing
process that cause adjustments to the estimates included in this presentation; our future financial and operating performance; risks associated with the
commercialization of INGREZZA and ONGENTYS; the impact of the COVID-19 pandemic on our business and the business operations of our customers; risks and
uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and
uncertainties related to any COVID-19 quarantines, shelter-in-place, social distancing and other government orders that are currently in place or that may be put in
place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to
the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to INGREZZA
and our product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding
our products or product candidates; risks associated with our dependence on AbbVie for the commercialization of ORILISSA and ORIAHNN, as well as the continued
development of elagolix; risks associated with our dependence on BIAL for manufacturing activities for ONGENTYS, and our ability to manage BIAL; risks that
clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 or other reasons, may not be
successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world
results or of results in subsequent clinical trials; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that
our products, and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side
effects, adverse reactions or incidents of misuse; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report
on Form 10-Q for the quarter ended September 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this presentation after the date
hereof.
This presentation refers to certain non-GAAP financial measures. These non-GAAP financial measures should not be considered replacements for, and should be
read together with, the most comparable GAAP financial measures.
3
Neuroscience Company Well-Positioned for Sustained and Long-Term Growth
Strong Commercial Capabilities; Experienced Sales Team
4 Approved Products
INGREZZA Blockbuster Status
ONGENTYS Launched Q3 2020
R&D Focus on Neurological, Endocrine, Psychiatric Disorders
Deep Pipeline
3 Programs in Pivotal Studies
Multiple Mid-Stage Programs
Strong Financial Position>$1.1B Cash and Investments(as of Q3 2020)
Generating Strong Free Cash Flow
‡
‡ Under License from
4
Uniquely Focused on Key Areas of Neuroscience
P S Y C H I A T R YE N D O C R I N O L O G YN E U R O L O G Y
5
4 Treatments Commercially Available Across 4 Indications
* Mitsubishi Tanabe Pharma has commercialization rights in East Asia. ‡ Under License from † AbbVie has global commercialization rights.
Uterine Fibroids
†
Endometriosis
†
Parkinson’s Disease
‡
Tardive Dyskinesia
*
Neurocrine Biosciences Exclusive and Partnered Commercial Products
PROGRAM INDICATION PHASE 1 PHASE 2 PHASE 3 PARTNERUPCOMING
MILESTONES 2021
valbenazine* Chorea in Huntington Disease Phase III Completion
NBIb-1817¶ Parkinson’s DiseaseInitiate Registrational
Study
NBI-921352 Rare Pediatric Epilepsy: SCN8A-DEE Ongoing Dialogue with FDA
NBI-827104 Rare Pediatric Epilepsy: CSWS Ongoing Phase II
crinecerfont Congenital Adrenal Hyperplasia (Adults) Ongoing Phase III
crinecerfont Congenital Adrenal Hyperplasia (Pediatric) Initiate Phase III
elagolix† Polycystic Ovary Syndrome Ongoing Phase Il
NBI-1065844§ Negative Symptoms of Schizophrenia Ongoing Phase II
NBI-1065845∥ Treatment-Resistant Depression Initiate Phase II
NBI-1065846∥ Anhedonia in Depression Initiate Phase II
6
Robust Pipeline With Multiple Mid-Stage Programs
Registrational
New Indication / Registrational
Registrational
En
do
cri
no
log
yN
eu
rolo
gy
Psych
iatr
y
Neurocrine Biosciences has global rights unless otherwise noted.
* Mitsubishi Tanabe Pharma has commercialization rights in East Asia. † AbbVie has global commercialization rights. ¶ Voyager Therapeutics has co-commercialization option for U.S. market following the ongoing Phase II RESTORE-1 study. § Takeda has co-commercialization option following the ongoing Phase II. ∥ Takeda has co-commercialization rights with option to opt out following certain development milestones.
SCN8A-DEE = SCN8A Developmental and Epileptic Encephalopathy
CSWS = Epileptic Encephalopathy with Continuous Spike and Wave During Sleep
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Strong Pipeline Momentum Through 2021Neurology
Endocrinology
Psychiatry
valbenazine
crinecerfont
Chorea in Huntington disease
Neurological Indication (New)
Congenital Adrenal Hyperplasia Ongoing Registrational Trial (Adults)
Initiate Registrational Trial (Pediatric)
Phase III Data Q4
Initiate Phase III
Phase 3 & Registrational Programs
NBI-827104 Rare Pediatric Epilepsy: CSWS
Neurological Indication (New)
Ongoing Phase II
Initiate Phase II
NBI-921352Rare Pediatric Epilepsy: SCN8A-DEE
Focal Onset Seizure in Adults
Ongoing Dialogue with FDA
Initiate Phase II
NBI-1065844 Negative Symptoms of Schizophrenia Phase II Data in 1st Half
NBI-1065845 Treatment-Resistant Depression Initiate Phase II
NBI-1065846 Anhedonia in Depression Initiate Phase II
Psychiatric Indication (New) Initiate Phase IIvalbenazine
Phase 2 & Phase 2 Ready Programs
P S Y C H I A T R YE N D O C R I N O L O G Y
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Advancing Life-Changing Treatments for Neurological Disorders
N E U R O L O G Y
• Rapid Improvement in Involuntary Movements
• Generally Well-Tolerated
• One Capsule, Once a Day
1st FDA-Approved Treatment for Tardive Dyskinesia (TD)
9
Approaching Blockbuster Status
9NEUROLOGY
$117
$410
$753
$993
$0
$200
$400
$600
$800
$1,000
$1,200
2017 2018 2019 2020
Net Sales and ~Total Prescriptions (TRx)
Net
Pro
du
ct
Sale
s (
$ i
n M
illio
ns)
15K TRx
72K TRx
133K TRx
176K TRx
Ap
pro
xim
ate
TR
x (
in T
ho
usan
ds)
144
72
36
0
108
180
216
10
Substantial Impact on TD Patients and Caregivers
Movement disorder caused by prolonged use of antipsychotics and anti-nausea medications
NEUROLOGY
Uncontrollable, abnormal
and repetitive movements >50% of patients experience meaningful
emotional, social and psychological impact*
* https://www.takeontd.com/ Source: IQVIA’s SMART Audit, Quarterly Data for Antipsychotic Class
Low Self-Worth
Psychiatric patients may
already have difficulty
gaining stability and social
acceptance
Isolation
Loss of physical control
may make patients more
likely to withdraw from
social situations
Job Performance
Patients believe TD
affects their ability to
perform their job
11
Nascent TD Market Presents Significant Opportunity
>500,000people in the U.S.
E S T I M AT E D T O A F F E C T
~80%
Source: Neurocrine Biosciences Data
NEUROLOGY
Increasing Antipsychotic Prescriptions (U.S.)
14.0
14.5
15.0
15.5
16.0
16.5
17.0
17.5
2017 2018 2019 2020
TR
x (
Mill
ion
s)
market is not yet diagnosed
~20%
patients with
TD diagnosed
12
Driving Long-Term Growth for INGREZZA
NEUROLOGY
Healthcare provider educational initiatives
Investing in telemedicinecapabilities
Recommends patients with TD associated with
antipsychotic therapy be treated with a VMAT2 inhibitor
Patient outreach programs
ONGENTYS and INGREZZA offering greater access to neurology practices
Pursuing New Indications: Chorea in Huntington Disease
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Phase III Top-Line Data Expected in Q4 2021; sNDA Planned Early in 2022
*Valbenazine in Huntington disease is investigational and not approved in the U.S.
Valbenazine*
Promising safety profile without devastating side effects
Targeted symptom control of chorea movement
NEUROLOGY
~90% of 30,000
Chorea in Huntington Disease
(HD) affects
HD patients in the U.S.
Rare neurodegenerative
disorder in which neurons
within the brain break down
Patients develop
involuntary abnormal,
abrupt or irregular
movements
Current treatment
associated with
increased risk of
depression, suicidality
1st and Only FDA-Approved Once-Daily
COMT Inhibitor for Parkinson’s Disease (PD)
Provides Significant Reduction of Daily “Off” Time;
Increase in Good “On” Time
– Add-on treatment to levodopa/carbidopa prolongs clinical effects
– Helps patients achieve more consistent motor symptom control
Demonstrated Safety and Tolerability Profile
– Not associated with diarrhea or discoloration of body fluids
Launched in Sept. 2020 in Virtual and Physical Environment
– Strong interest from neurologists
‡ Under License from
14NEUROLOGY
Only Therapy Delivering on the Promise of COMT Inhibition
Catechol-O-methyltransferase
†
†
‡
P S Y C H I A T R YN E U R O L O G Y
15
Developing Treatment Options for Endocrine-Related Disorders
E N D O C R I N O L O G Y
U.S.~30,000
E.U.~50,000
16
Classic Congenital Adrenal Hyperplasia (CAH)
• Enzyme deficiency
• Reduced adrenal steroids & excess androgen levels
Complex and Highly
Variable Symptoms
Rare Genetic Disorder
Treatment Options
Stagnant for 60 Years
• Hormone replacement
• Do not address underlying issue
ENDOCRINOLOGY
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Congenital Adrenal Hyperplasia Disease Mechanism
Homeostasis CAH Patients
Potentially Fatal Adrenal Crisis
Infertility
Low Blood Pressure
Salt Loss
Amenorrhea
Hirsutism
Severe Fatigue, Lack of Energy
UndertreatmentHyperandrogenism
Insulin Resistance
Central Obesity
Osteoporosis
Impaired Glucose Tolerance
Hypertension
Cushingoid Disorder
OvertreatmentHypercortisolism
19
Crinecerfont Meets the Challenges of the Standard of Care
ENDOCRINOLOGY
Potentially Fatal Adrenal Crisis
Infertility
Low Blood Pressure
Salt Loss
Amenorrhea
Hirsutism
Severe Fatigue, Lack of Energy
UndertreatmentHyperandrogenism
Insulin Resistance
Central Obesity
Osteoporosis
Impaired Glucose Tolerance
Hypertension
Cushingoid Disorder
Overtreatment Hypercortisolism
CRINECERFONT
Crinecerfont*
Phase III Global Registrational Study in Adults Ongoing
Phase III Pediatric Study Scheduled 2021
20ENDOCRINOLOGY
Potential Paradigm Shift in the Treatment of CAH
* Crinecerfont is investigational and not approved in any country
Potent Orally Active
Selective Well-Tolerated
N E U R O L O G Y E N D O C R I N O L O G Y
21
Expanding Our Pipeline in Psychiatry
P S Y C H I A T R Y
First-in-Class Early to Mid-Stage Compounds
22PSYCHIATRY
Takeda Partnership
Exclusive worldwide rights to early to mid-stage psychiatry compounds
NBI-1065844 Negative Symptoms of Schizophrenia
Ongoing Phase II With
Top-Line Data Expected
in 1st Half of 2021
NBI-1065845 Treatment-Resistant Depression Initiate Phase II in 2021
NBI-1065846 Anhedonia in Depression Initiate Phase II in 2021
23
Well-positioned for Sustained and Long-term Growth
‡ Under License from
‡
Achieved ~$1B in Annual Sales in
3.5 Years
Significant Opportunity for
GROWTH
Pipeline
>$1.1B Cash and Investments(as of Q3 2020)
Strong FinancialPosition
3 Pivotal Programs 7 Mid-to-late Stage Programs
neurocrine.comneurocrine.com
JP Morgan Healthcare Presentation
January 11, 2021
Nasdaq: NBIX