JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic...

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neurocrine.com neurocrine.com JP Morgan Healthcare Presentation January 11, 2021 Nasdaq: NBIX

Transcript of JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic...

Page 1: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

neurocrine.comneurocrine.com

JP Morgan Healthcare Presentation

January 11, 2021

Nasdaq: NBIX

Page 2: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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.

Page 3: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

Page 4: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 5: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

Page 6: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

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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

Page 7: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

Page 8: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 9: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

• Rapid Improvement in Involuntary Movements

• Generally Well-Tolerated

• One Capsule, Once a Day

1st FDA-Approved Treatment for Tardive Dyskinesia (TD)

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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

Page 10: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

Page 11: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

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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

Page 13: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 14: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 15: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

P S Y C H I A T R YN E U R O L O G Y

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Developing Treatment Options for Endocrine-Related Disorders

E N D O C R I N O L O G Y

Page 16: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

U.S.~30,000

E.U.~50,000

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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

Page 17: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA
Page 18: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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Congenital Adrenal Hyperplasia Disease Mechanism

Homeostasis CAH Patients

Page 19: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

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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

Page 20: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 21: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

N E U R O L O G Y E N D O C R I N O L O G Y

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Expanding Our Pipeline in Psychiatry

P S Y C H I A T R Y

Page 22: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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

Page 23: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

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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

Page 24: JP Morgan Healthcare Presentation - Neurocrine Biosciences · 2021. 1. 11. · antipsychotic therapy be treated with a VMAT2 inhibitor Patient outreach programs ONGENTYS and INGREZZA

neurocrine.comneurocrine.com

JP Morgan Healthcare Presentation

January 11, 2021

Nasdaq: NBIX