Advancing Life-Changing Discoveries in Neuroscience · 2020. 11. 9. · Q3 2020 Earnings...

neurocrine.comneurocrine.com

Advancing Life-Changing

Discoveries in Neuroscience

Q3 2020

Corporate Presentation

November 9, 2020

Nasdaq: NBIX

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.

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


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.


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


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

Our MedicinesOur Patients

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


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%

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)


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.


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


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


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


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/


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

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


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.


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


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


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

Our Vision for the Future


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


GAAP to Non-GAAP Reconciliations


Advancing Life-Changing

Discoveries in Neuroscience

Q3 2020

Corporate Presentation

November 9, 2020

Nasdaq: NBIX

Date post:	25-Jan-2021
Category:	Documents
Upload:	others
View:	1 times
Download:	0 times

Advancing Life-Changing Discoveries in Neuroscience · 2020. 11. 9. · Q3 2020 Earnings...

Documents