Advancing Life-Changing Discoveries in Neuroscience · 2020. 11. 9. · Q3 2020 Earnings...
Transcript of Advancing Life-Changing Discoveries in Neuroscience · 2020. 11. 9. · Q3 2020 Earnings...
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neurocrine.comneurocrine.com
Advancing Life-Changing
Discoveries in Neuroscience
Q3 2020
Corporate Presentation
November 9, 2020
Nasdaq: NBIX
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J.P. Morgan Healthcare Conference 2020Q3 2020 Earnings Presentation
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: 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 launch of ONGENTYS; our financial and operating performance, including our future expenses; our
collaborative partnerships; expectations regarding the impact of COVID-19 on our business; expectations regarding our ability to adapt our business to the evolving
COVID-19 pandemic, mitigate its impact on our business and maintain business continuity, including our ability to protect the safety and well-being of our employees,
to continue to support uninterrupted supply of INGREZZA, including patient and healthcare provider access to INGREZZA, 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: 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 press release 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. Reconciliations of non-GAAP financial results to the most directly comparable GAAP financial
results are included at the end of this press release, which has been filed with the SEC in a Current Report on Form-8-K dated as of event date herewith. In addition,
Neurocrine provides guidance regarding combined research and development and sales, general and administrative expenses on both a GAAP and non-GAAP basis.
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Q3 2020 Earnings Presentation 3
Neurocrine Q3 2020 Highlights & Q4 Key Activities
TRx = Total Prescriptions; FDA = U.S. Food and Drug Administration; COMT = Catechol-O-Methyltransferase; UPDRS = Unified Parkinson’s Disease Rating Scale; TD = Tardive Dyskinesia; CAH = Congenital Adrenal Hyperplasia;
SCN8A-DEE = SCN8A Developmental and Epileptic Encephalopathy; CSWS = Continuous Spikes and Waves During Sleep
Q3 2020 Highlights Q4 2020 Key Activities
▪ INGREZZA® (valbenazine) Net Product Sales
– $254MM with ~45,100 TRx in Q3 2020
– $753MM with ~133,000 TRx YTD through Q3
▪ ONGENTYS® (opicapone) Launched in the U.S.
– ONGENTYS: The First and Only FDA-Approved Once Daily COMT Inhibitor, Decreases “Off” Time and Increases “On” Time Without Troublesome Dyskinesia When Added to Levodopa / Carbidopa
▪ Presented New Long-Term Data Demonstrating One-Time Treatment with NBIb-1817 Continued To:
– Improve Motor Function, including “On” Time Without Troublesome Dyskinesia and UPDRS III Scores
– Reduce the Amount of Medication Used In Patients With Parkinson’s Disease After Three Years
▪ Continued Focus on INGREZZA Commercial Execution Including “Talk About TD” Disease State Awareness Campaign
▪ Grow Awareness and Adoption of ONGENTYS Via Educational Initiatives and Product Sampling
▪ Advance Clinical Programs and Enroll Patients in:
– Single, Global Registrational Study of Crinecerfont in Adults
– Crinecerfont Pediatric Phase II Proof-of-Concept Study
– Phase III Registration Study of Valbenazine to Treat Chorea in Huntington Disease
▪ Address FDA Feedback for NBI-921352 in SCN8A-DEE Indication; Progress With Plans for Adult Focal Epilepsy Indication
▪ Initiate Phase II Study for NBI-827104 in CSWS, a Rare Pediatric Epilepsy
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Q3 2020 Earnings Presentation 4
Commercially Available Products and Pipeline Candidates
* Mitsubishi Tanabe Pharma has commercialization rights in East Asia. † AbbVie has global commercialization rights. ‡ BIAL retains commercialization rights outside U.S. and Canada. ¶ Voyager Therapeutics has co-commercialization option for U.S. market following the ongoing Phase II RESTORE-1 study
PROGRAM INDICATION PHASE 1 PHASE 2 PHASE 3
valbenazine* Chorea in Huntington Disease
ezaladcigeneresoparvovec¶ (VY-AADC)
Parkinson’s Disease
NBI-921352 (XEN901) Rare Pediatric Epilepsy: SCN8A-DEE
NBI-827104 (ACT-709478) Rare Pediatric Epilepsy: Continuous Spikes and
Waves During Sleep
crinecerfont Congenital Adrenal Hyperplasia (Adults)
crinecerfont Congenital Adrenal Hyperplasia (Pediatric)
elagolix† Polycystic Ovary Syndrome
NBI-1065844 (TAK-831) Negative Symptoms of Schizophrenia
NBI-1065845 (TAK-653) Treatment-Resistant Depression
NBI-1065846 (TAK-041) Anhedonia in Depression
New indication
Registrational
PIPELINE
Tardive Dyskinesia
*
Endometriosis
†
COMMERCIALLYAVAILABLE †
Uterine FibroidsParkinson’s Disease
‡
Psy
chia
tric
Dis
ord
ers
End
ocr
ine
Ne
uro
logi
cal
Neurocrine Biosciences has global rights unless otherwise noted.
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Q3 2020 Earnings Presentation 5
Financial Summary$ Millions, Except Non-GAAP Earnings Per Share
Item Q3 ’20 Q3 ’19 Financial Highlights / Comments
Revenue
- Product Sales, Net- Collaboration Revenue
$258.5
254.1
4.4
$222.1
198.1
24.0
INGREZZA Sales of $254M Representing YoY Growth of 28%;
Prior Year Collaboration Revenue Included $20M Event-Based
Milestone for Elagolix
Non-GAAP R&D Expense 60.3 38.3Increase Due Primarily to Increased Investment Across Expanded
Pipeline Programs and Headcount Costs
Non-GAAP SG&A Expense 94.6 71.2Increase Due Primarily to Increased Investment in Marketing
Initiatives and Headcount Costs
Non-GAAP Net Income 96.1 86.7 10th Straight Quarter of Positive Non-GAAP Net Income
Non-GAAP Earnings per Share, Diluted $0.97 $0.90 8% YoY Growth
Cash and Investments (Period End) $1,126.1 $875.0 Cash Balance Increase Driven by Operating Income
All income statement items, except revenue, are non-GAAP financial measures—see reconciliations accompanying the presentation
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Q3 2020 Earnings Presentation 6
Revised 2020 GAAP and Non-GAAP Expense Guidance$ Millions
▪ Guidance Range Reflects Increased Investment in R&D, Including Three Registrational Programs and Mid-Stage Pipeline, Continued Investments in INGREZZA and Marketing Costs Associated with U.S. Launch of ONGENTYS
▪ GAAP Guidance:
– Includes:
✓$120 Million Paid to Takeda for Exclusive License for Right to Develop and Commercialize Certain Compounds in Takeda’s Early-to-Mid-Stage Psychiatry Pipeline
✓$45 Million Paid to Idorsia Upon Exercising Option to License the Global Rights to NBI-827104 (ACT-709478)
✓$20 Million Paid to Bial for the U.S. Approval of ONGENTYS
✓ Includes Approximately $105 Million of Share-Based Compensation
– Does Not Include Any Other Potential Future Milestones or In-Process Research and Development Costs Associated with Current Collaborations or Potential Future Business Development Activities
▪ Non-GAAP Guidance:
– YoY Increase Driven by Higher Investment in Pipeline Programs, Commercial Marketing Initiatives and Increased R&D and SG&A Headcount Costs
Combined R&D and SG&A Expenses2019
Actuals
2020 Updated
Guidance Range
2020 Previous
Guidance Range
GAAP Basis $554 $880 - $900 $850 - $900
Non-GAAP Basis $469 $590 - $610 $570 - $610
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Our MedicinesOur Patients
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J.P. Morgan Healthcare Conference 2020Q3 2020 Earnings Presentation 8
1st FDA-Approved Treatment for Adults with Tardive Dyskinesia (TD) – Launched in 2017
▪ Rapid Improvement in Involuntary Movements
▪ Generally Well Tolerated
▪ Ease-of-Use: One Capsule, Once Daily
Most Prescribed and Most Preferred TD Therapy
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Q3 2020 Earnings Presentation 9
Tardive Dyskinesia (TD): An Overview
* Neurocrine Data on File
TD is a movement
disorder characterized by
uncontrollable, abnormal
and repetitive movements
of the face, torso and/or
other body parts, which
may be disruptive and
negatively impact patients
TD can look different
day-to-day. Symptoms
can be severe and are
often persistent and
irreversible
1 in 5U.S. adults live with
a mental illness
TD, one of the
challenges
associated with
mental illness, is
estimated to affect
at least
500,000 people in the U.S.
TD is caused by
prolonged use of
antipsychotics
commonly prescribed to
treat schizophrenia,
bipolar disorder and
depression, and
certain anti-nausea
medications
According to a survey* of
patients with TD, the
condition affects their:
Ability to sleep
61%
Ability to exercise
39%
Self-esteem
68%
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Q3 2020 Earnings Presentation
$130 $136
$181$198
$238$231
$268$254
22.924.2
31.6
34.8
42.1 41.5
46.445.1
10
20
30
40
50
60
$50
$100
$150
$200
$250
$300
Q4 '18 Q1 '19 Q2 '19 Q3 '19 Q4 '19 Q1 '20 Q2 '20 Q3'20
INGREZZA Net Sales INGREZZA TRx (30 day)
10
INGREZZA Net Sales Growth of 28% vs. Q3 2019
10
INGREZZA Net Sales and ~Total Prescriptions (TRx)
Net
Pro
du
ct
Sale
s (
$ i
n M
illio
ns)
Ap
pro
xim
ate
TR
x(in
Th
ou
san
ds)
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Q3 2020 Earnings Presentation 11
Valbenazine: Chorea in Huntington Disease Phase III Study to Treat Chorea in Adult Patients with Huntington Disease
An involuntary movement disorder estimated
to affect approximately 90% of the 30,000 HD
patients in the U.S. HD is a rare
neurodegenerative disorder in which
neurons within the brain break down. Patients
with chorea in HD develop abnormal, abrupt
or irregular movements
Common symptoms of chorea can affect
various body parts and interfere with speech,
swallowing, posture and gait
Need for chorea treatment options with
better safety profile as current treatments
are associated with increased risk of
depression and suicidality
▪ Targeted symptom control of chorea movements
as measured by the Unified Huntington Disease
Rating Scale (UHDRS) and Total Maximal Chorea (TMC)
▪ Promising safety profile without troublesome
side effects
▪ Phase III study initiated with data expected in 2021
Chorea in Huntington Disease (HD) Valbenazine*
* Valbenazine in Huntington disease is investigational and not approved in the U.S.
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J.P. Morgan Healthcare Conference 2020Q3 2020 Earnings Presentation 12
1st and Only FDA-Approved Once-Daily COMT Inhibitor for Parkinson’s DiseaseLaunched in the U.S. in Late Q3 2020
▪ Provides Significant Reduction of Daily “Off” Time; Increase in Good “On” Time
– Add-on treatment to levodopa/carbidopa prolongs clinical effects and helps patients achieve more consistent motor symptom control
▪ One Capsule, Once Daily
– Convenient for patients and may lessen daily pill burden levels for PD patients
▪ Demonstrated Safety and Tolerability Profile
– Not associated with diarrhea or discoloration of body fluids
Under license from
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Q3 2020 Earnings Presentation 13
Parkinson’s Disease (PD): An Overview
Parkinson’s disease (PD)
is a chronic, progressive
and debilitating neuro-
degenerative disease
PD is caused by low
dopamine levels produced
in the brain. Dopamine
helps transmit signals
between the areas of the
brain that control all
purposeful movements
1 million PD affects
people in the U.S. 2 out of 3PD patients on carbidopa/
levodopa (standard-of-care) new diagnoses annually50,000
2nd most common neurodegenerative
disorder following
Alzheimer’s disease
Levodopa loses
effectiveness as
disease progresses
requiring dose and
frequency escalation
to achieve motor
symptom control
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J.P. Morgan Healthcare Conference 2020Q3 2020 Earnings Presentation 14
1st FDA-Approved Oral Treatment for Women with Moderate-to-Severe Endometriosis Pain in Over a Decade - Launched in 2018
▪ Less Estrogen = Less Painful Endometriosis Lesions
– Addresses three most common types of endometriosis pain: painful periods, pelvic pain between periods, pain with sex*
▪ Oral Administration
– Two dosage options based on severity of symptoms and treatment objectives
▪ Safety and Tolerability Profile
– Proven efficacy and safety in largest endometriosis clinical program
* There are two different dosage options of ORILISSA: 150 mg (taken once a day) or 200 mg (taken twice a day). Only the 200 mg dose was proven to work for pain with sex.
Neurocrine Biosciences discovered and
developed through Phase II; AbbVie
received FDA approval and responsible
for commercialization
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J.P. Morgan Healthcare Conference 2020Q3 2020 Earnings Presentation 15
1ST FDA-Approved Oral Medication to Manage Heavy Menstrual Bleeding due to Uterine Fibroids in Pre-Menopausal Women
Neurocrine Biosciences discovered and
developed elagolix through Phase II;
AbbVie received FDA approval and
responsible for commercialization
▪ Clinically Meaningful Reduction in Heavy Menstrual Bleeding– 7 out of 10 women no longer experiencing heavy menstrual bleeding vs. 1 out of 10 women on placebo– Reduced heavy menstrual bleeding by 50% within the first month of use
▪ Non-Surgical Oral Administration– Oral combination of elagolix and estradiol/norethindrone acetate helps achieve a balance between the
reduction of heavy bleeding and associated hypoestrogenic side effects– Twice daily (morning and evening) dosing at approximately the same time each day, with or without food
▪ Safety and Tolerability Profile – The most common adverse reactions occurring in ≥5% of women receiving ORIAHNN in clinical trials were
hot flush, headache, fatigue, and metrorrhagia. These are not the only possible side effects of ORIAHNN– May increase chances of heart attack, stroke, or blood clots, especially in smokers over 35 years of age
with high blood pressure– Use of ORIAHNN should be limited to 24 months due to the risk of continued bone loss, which may not
be reversible– See important safety information, including BOXED WARNING on THROMBOEMBOLIC AND
VASCULAR EVENTS at rxabbvie.com
https://urldefense.proofpoint.com/v2/url?u=http-3A__click.e.abbvie.com_-3Fqs-3D93e60964449b439d4a9a6265d936c0698333514c8fe98739d1868a6ed8b393c946a661fd1405106ca65643599767e58c5a23f1d2f463da83&d=DwMDaQ&c=pBpWB3g5lxYDsRBNURdZrQ&r=0cVEm4nBRTB31_fN7d4gWs3agBSm6rGH_lBPOjVLyps&m=c6-cK7rGVsAUERS7dmW2KvvaJlVV5aea0RNhcIlFfcY&s=2oh5NgI2peko2PpSV8_nwDiRAGsO6GBmdayym-06-WI&e=https://www.rxabbvie.com/
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Q3 2020 Earnings Presentation 16
Crinecerfont*: Classic Congenital Adrenal HyperplasiaInitiated Single Phase III Global Registrational Study in Adults
Phase II Pediatric Study Ongoing**
▪ Potent, selective, orally active, non-peptide
corticotropin releasing factor type 1 (CRF1)
receptor antagonist
Rare genetic disorder caused by enzyme
deficiency which leads to reduced adrenal
steroids and excess androgen levels with up
to 30,000 people impacted in the U.S. and up
to 50,000 people in Europe
Complex and highly variable symptoms
including adrenal crisis, virilization, hirsutism,
precocious puberty, fertility problems and
abnormal growth
Excess corticosteroid treatment leads
to additional clinical problems including
bone loss, Cushing’s disease and
metabolic syndrome
* Crinecerfont is investigational and not approved in any country
** Study resumed following temporary pause due to COVID-19
Congenital Adrenal Hyperplasia Crinecerfont
Cushingoid FeaturesCentral Obesity Osteoporosis
Insulin ResistanceImpaired Glucose Tolerance
Glucocorticoid
Adrenal CrisisIncreased Adrenal
AndrogenHirsutism
AmenorrheaInfertility
Hypertension Mineralocorticoid
Low Blood PressureSalt Loss
Fatigue, Lack of EnergyIncreased Requirements
for Glucocorticoid Replacement
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Q3 2020 Earnings Presentation
NBI-921352*
for SCN8A-DEE
and adult
focal epilepsy
NBI-827104*
for CSWS
Research
collaboration
Research
collaboration
17
v
NBIb-1817*
for Parkinson’s disease
Friedreich’s ataxia
Two undisclosed CNS programs
* Investigational and not approved any country.
v
NBI-1065844*
for Negative Symptoms
of Schizophrenia
NBI-1065845* for Treatment
Resistant Depression
NBI-1065846* for Treatment of
Anhedonia in Depression
Four undisclosed
preclinical programs
Gene Therapy
Opportunity to Expand Footprint
into Key Areas of Neuroscience
with Novel Modalities
Precision Medicine
Establish Strong Presence to Address
Unmet Medical Needs in Epilepsy and
Other Neurological Disorders
Neuropsychiatry
Develop Potential First-in-Class
Programs For Under-Served
Psychiatric Disorders
Expanding Reach: Innovative Partners with Novel Science to Address Unmet Medical Need
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Q3 2020 Earnings Presentation 18
NBIb-1817*: Gene Therapy for Parkinson’s Disease
▪ One-time treatment restores AADC enzyme activity,
enhances the conversion of levodopa and
restores motor function
▪ Improvement in ON time and reduction in OFF
time at 1-year timepoint
▪ >7-year shift in disease progression seen at 1 year
as measured by modified Hoehn and Yahr scale
▪ Durable expression of the AADC enzyme observed
at 15 years post-administration in non-human primates
Moderate to Advanced PD NBIb-1817†
Severe, debilitating loss of motor function
including rigidity, postural instability,
gait freezing and difficulty with speech
and swallowing
Loss of neurons and critical AADC enzyme
in the midbrain that produce dopamine leads
to progressive loss of motor function and less
responsiveness to levodopa
One million patients with PD in the U.S., with
moderate to advanced stages of PD typically
occurring four years after diagnosis
* In-licensed from Voyager Therapeutics† NBIb-1817 is investigational and not approved in any country.
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Q3 2020 Earnings Presentation 19
NBI-921352*: Selective NaV1.6 Inhibitor for Rare Pediatric EpilepsyEngaging With FDA to Discuss Request for Additional Non-Clinical Data to Enable Pediatric Trial in SCN8A-DEE†
Ashley
▪ First potent and selective inhibitor to precisely target
the sodium channel affected by the genetic mutation of
SCN8A - NaV1.6
▪ Impact the lives of SCN8A-DEE patients and additional
1 million patients with focal seizures, 50% of whom
are refractory to existing treatments
▪ Potential Initiation of Phase II study in SCN8A-DEE
in 2021
▪ Program has potential to address other central
nervous system diseases → Currently developing
plans in adult focal epilepsy
SCN8A-DEE NBI-921352‡
* In-licensed from Xenon Pharmaceuticals†SCN8A-DEE (SCN8A developmental and epileptic encephalopathy)
‡ NBI-921352 is investigational and not approved in any country.
Physical and psychological symptoms
include recurrent seizures of all types,
developmental delays, learning difficulties,
muscle spasms, poor coordination, sleep
problems and autistic-like features
Rare form of early-onset epilepsy with
occurrence of seizures beginning in the first
18 months of life and a high incidence of
sudden unexpected death in epilepsy
No approved treatments with off-label
options associated with poor outcomes,
safety and tolerability
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Q3 2020 Earnings Presentation 20
NBI-827104*: Selective CaV Inhibitor for Continuous Spikes and Waves During Sleep (Rare Pediatric Epilepsy)
Ashley
▪ First potent and selective inhibitor to precisely
target calcium channels 3.1, 3.2 and 3.3
▪ Impact the lives of CSWS patients and over 1 million
patients with adult generalized seizures
▪ Initiate Phase II study in CSWS in Q4 2020
▪ Potential fast track to approval in CSWS given
significant clinical need and lack of treatment options
▪ Program has potential to address other central
nervous system diseases
Continuous Spikes and Waves During Sleep (CSWS) NBI-827104†
* In-licensed from Idorsia Pharmaceuticals† NBI-1065844 is investigational and not approved in any country
Progressive decline in cognitive,
behavioral and psychiatric functioning
impacting all language, communication,
attention and social interaction. Impairments
are typically severe
Rare childhood epilepsy characterized by
onset seizures between 2 and 12 years
of age
No approved treatments with off-label
options associated with poor outcomes,
safety and tolerability
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Q3 2020 Earnings Presentation 21
NBI-1065844*: Potential First-in-Class D-Amino Acid Oxidase (DAAO) Inhibitor
Ashley
▪ Potent first-in-class DAAO inhibitor
▪ Hypofunction of glutamatergic signaling has been
implicated in the pathophysiology of schizophrenia
▪ Exogenously administered D-Serine and sodium
benzoate (a weak DAAO inhibitor) have improved
PANSS (Positive and Negative Syndrome Scale)
scores in patients with schizophrenia
▪ Ongoing Proof-of-Concept Phase II study with data
expected in mid-2021
Negative Symptoms of Schizophrenia NBI-1065844†
* In-licensed from Takeda Pharmaceuticals† NBI-1065844 is investigational and not approved in any country
Negative symptoms include avolition,
apathy diminished expression, and
attentional impairment
Over One million diagnosed patients in the
U.S. with approximately 800K treated
No approved treatments specifically
indicated for the negative symptoms
of schizophrenia
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Our Vision for the Future
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Q3 2020 Earnings Presentation 23
Transformation into Fully Integrated Neuroscience-Focused Company: Well-Positioned for Sustained Growth
Strategic Partnerships
Solid Financial Position to
Invest
>$1.1B Cash and Investments
(as of Q3 2020)
Proven R&D with Strong Multi-Stage
Pipeline
Four Approved Medicines in
Four Indications; Three Additional
Programs in Pivotal Studies and Multiple Mid-Stage Programs
Strong Commercial Capabilities
INGREZZA Nearing Blockbuster Status;
ONGENTYS Launched in Q3 2020;
Experienced, Neuro/Psych Field
Sales Team
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neurocrine.comneurocrine.com
GAAP to Non-GAAP Reconciliations
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Q3 2020 Earnings Presentation 25
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Q3 2020 Earnings Presentation 26
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Q3 2020 Earnings Presentation 27
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neurocrine.comneurocrine.com
Advancing Life-Changing
Discoveries in Neuroscience
Q3 2020
Corporate Presentation
November 9, 2020
Nasdaq: NBIX