Neurocrine Biosciences, Inc. THE NEUROENDOCRINE COMPANY

Similar documents
Neurocrine Biosciences, Inc. THE NEUROENDROCRINE COMPANY

Safe Harbor Statement

35 th Annual J.P. Morgan Healthcare Conference

Medical Policy An independent licensee of the Blue Cross Blue Shield Association

Acorda Acquisition of Civitas Therapeutics. September 24, 2014

US Tardive Dyskinesia Market: Size, Trends & Forecasts ( ) December 2016

Corporate Presentation August 6, 2015

Supernus Pharmaceuticals

Supernus Pharmaceuticals

Radius Highlights Science-Driven Biopharmaceutical Company

Ingrezza. (valbenazine) New Product Slideshow

Presented at the American Psychiatric Association Annual Meeting May 20 24, 2017 San Diego, CA.

Small-Cap Research. Neurocrine Biosciences, Inc. (NBIX-NASDAQ) NBIX: KINECT-2 Hits, Shares Soar! UPDATE SUMMARY DATA ZACKS ESTIMATES

August 7, Q Financial Results

37 th Annual J.P. Morgan Healthcare Conference. January 9, 2019

Ingrezza. Ingrezza (valbenazine) Description

ACADIA Pharmaceuticals Issues Statement Reaffirming Benefit/Risk Profile of NUPLAZID

SAKURA 3 Open-Label Phase 3 Safety Study with DaxibotulinumtoxinA for Injection (RT002) for the Treatment of Moderate to Severe Glabellar Lines

Q3 18 Earnings Supplemental Slides

Developing & Commercializing Targeted Small Molecule Drugs in Cancer

AM-125 : Intranasal Betahistine

Small-Cap Research. Neurocrine Biosciences, Inc. (NBIX - Nasdaq) NBIX: NDA for Valbenazine in Tardive Dyskinesia to be Filed in 2016 SUMMARY DATA

Santhera to Acquire Option from Idorsia for Exclusive Sub-License to First-in-class Dissociative Steroid Vamorolone. Webcast, 21 November 2018

Ingrezza (valbenazine)

Innovation In Ophthalmics

NASDAQ: ZGNX. Company Presentation. October 2017

Xenazine. Xenazine (tetrabenazine) Description

Developing clinical stage small molecule therapeutics to treat hormonal and reproductive system disorders

Could your uncontrollable movements be tardive dyskinesia (TD)? There s a treatment with INGREZZA.

Revefenacin (TD-4208) Phase 3 Efficacy Results

Announcing FDA Approval of GOCOVRI TM

Amicus Establishes Gene Therapy Pipeline for Lysosomal Storage Disorders (LSDs) Conference Call and Webcast September 20, 2018

Company Presentation June 2016

Investor Presentation March 2015

STUDY 1 PHASE 3 TOP-LINE RESULTS. September 2017

Genta Incorporated. A Multiproduct Late-Stage Oncology Company

Sucampo Pharmaceuticals, Inc. Jefferies 2015 Healthcare Conference

Genomic Health. Kim Popovits, Chairman, CEO and President

European Commission approves ONGENTYS (opicapone) a novel treatment for Parkinson s disease patients with motor fluctuations

For Tardive Dyskinesia

ARQ 087 Overview. FGFR Inhibitor. March 2017

4Q and Full Year 2017 Financial Results Call February 7, 2018

Corporate Presentation

XP23829 PHASE 2 PSORIASIS TRIAL PRELIMINARY TOPLINE DATA PRESENTATION SEPTEMBER 15, 2015 COPYRIGHT 2015 XENOPORT, INC. ALL RIGHTS RESERVED.

Committed to Transforming the Treatment Paradigm for Migraine Prevention

Company Update with a Focus on Pipeline

ObsEva Reports Third Quarter 2018 Financial Results and Provides Business Update

Committed to Transforming the Treatment Paradigm for Migraine Prevention

Study 1 ( ) Pivotal Phase 3 Long-Term Safety Study Top-Line Results

February 20, 2019 MANAGEMENT CALL TO DISCUSS PHASE 2 PHOENIX RESULTS AND CKD PROGRAM UPDATES

Vesicular Monoamine Transporter Type 2 Inhibitors: deutetrabenazine (Austedo ), tetrabenazine (Xenazine ), valbenazine (Ingrezza )

AUSTEDO (deutetrabenazine) oral tablet INGREZZA (valbenazine) oral capsule XENAZINE (tetrabenazine) oral tablet Tetrabenazine oral tablet

N a s d a q : I N S Y

Targeting and Treating Cancer

TELECONFERENCE FY February 2015

AVEO and Astellas Report Final Overall Survival Results from TIVO-1

Corporate Presentation Asia Investment Series March 2018

Corporate Overview. February 2018 NASDAQ: CYTR

H. LUNDBECK A/S. Teleconference. 10 August PM CET. Financial results Second quarter 2011

Egalet Corporate Presentation

Alcobra Ltd. (NASDAQ:ADHD) June Dr. Yaron Daniely President & CEO

Cowen Healthcare Conference March 12, 2018

Jefferies 2016 Healthcare Conference. Reid Huber, PhD Chief Scientific Officer

Diagnostics for the early detection and prevention of colon cancer. Fourth-Quarter 2014 Earnings Call February 24, 2015

J.P. Morgan Healthcare Conference

Targeting and Treating Cancer

Lauren Silvernail, CFO & VP Corporate Development to present at 2005 Thomas Weisel Partners Healthcare Conference on September 7, 2005 at 3:50 pm EST

Jefferies Healthcare Conference

SPHERIX ANNOUNCES FIRST QUARTER 2010 FINANCIAL RESULTS

AVENUE THERAPEUTICS, INC. NASDAQ: ATXI JANUARY 2018

Synergy Pharmaceuticals TRULANCE (Plecanatide) Receives U.S. FDA Approval for the Treatment of Adults with Chronic Idiopathic Constipation

ObsEva Reports First Quarter 2018 Financial Results and Provides Business Update

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. August 2017

Jefferies Healthcare Conference. June 25, 2008

TELECONFERENCE Q November 2015

Third Quarter 2015 Earnings Call. November 9, 2015

Alexis Howerton On Starting Up From New Science

Memory Pharmaceuticals Establishes Plans for Clinical Program for MEM 3454 in Schizophrenia. -Broadens Roche Nicotinic Alpha-7 Alliance-

Xenazine. Xenazine (tetrabenazine) Description

Pimavanserin Top-Line Results Phase III Parkinson s Disease Psychosis Trial (-020 Study) Creating the Next Generation of CNS Drugs

Spectrum Pharmaceuticals

PROMISE 2 Top-Line Data Results January 8, 2018

Merrill Lynch Pharmaceutical, Biotechnology & Medical Device Conference New York, February 4, 2004

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. September 2017

Innovation In Ophthalmology

ESTABLISH 2 Top Line Data Release

Ganaxolone as a Treatment for Drug-Resistant Epilepsy in Children

Lilly Diabetes: Pipeline Update

May 10, 2016 Q & Business Update

- Linzagolix overall efficacy and safety maintained or improved at week 24

Zogenix Announces Positive Top-line Results from Pivotal Phase 3 Clinical Trial of ZX008 in Dravet Syndrome

34 th Annual J.P. Morgan Healthcare Conference

PLEO-CMT Top-line Results. Presentation October 16, 2018

Alcobra Ltd. (NASDAQ:ADHD) June 2016

May 2018 August 9, 2018

ARDELYX REPORTS POSITIVE T3MPO-2 PHASE 3 TRIAL RESULTS IN IBS-C

PROVIDED IN RESPONSE TO YOUR UNSOLICITED REQUEST FOR INFORMATION

Anti-IL-33 (ANB020) Program

Press Release. RedHill Biopharma Announces Enrollment of Last Patient in the BEKINDA Phase II Study for IBS-D

Nektar Investor & Analyst Call. Nektar & z Bristol-Myers Squibb Collaboration. February 14, 2018

Transcription:

Neurocrine Biosciences, Inc. THE NEUROENDOCRINE COMPANY

Safe Harbor Statement 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, to the benefits to be derived from Neurocrine's products and product candidates, including INGREZZA; the market potential of INGREZZA and our product candidates, and the value they may bring to patients; the timing of completion of clinical and other development activities and whether those activities culminate in product approvals; and whether results from our clinical trials can be replicated or are indicative of real-world results. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: Neurocrine's future financial and operating performance; risks and uncertainties associated with the commercialization of INGREZZA, including the likelihood of continued revenue growth of INGREZZA; risks or uncertainties related to the development of the Company's product candidates; risks and uncertainties relating to competitive products and technological changes that may limit demand for INGREZZA or a product candidate; risks associated with the Company's dependence on third parties for development and manufacturing activities related to INGREZZA and the Company s product candidates, and the ability of the Company to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding INGREZZA or the Company s product candidates; risks associated with the Company's dependence on AbbVie for the development and commercialization of elagolix; risks that clinical development activities may not be completed on time or at all; risks that clinical development activities may be delayed for regulatory 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 benefits of the agreements with BIAL and Mitsubishi Tanabe may never be realized; risks associated with the Company's dependence on BIAL for development and manufacturing activities related to opicapone; risks associated with the Company's dependence on Mitsubishi Tanabe for the development and commercialization of valbenazine in Japan and other Asian countries; risks that INGREZZA 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 the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company s Annual Report on Form 10-K for the year ended December 31, 2017. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof. 2

Clinical Pipeline Disease Program Stage of Development 1 2 3 NDA Commercial Partner Neurology Tardive Dyskinesia INGREZZA Tourette Syndrome valbenazine Asia Parkinson s Disease opicapone NBI Rights: US & Canada Endocrinology Endometriosis elagolix Uterine Fibroids elagolix Worldwide Congenital Adrenal Hyperplasia NBI-74788 3

N E U R O C R I N E B I O S C I E N C E S, I N C. INGREZZA (valbenazine) capsules First FDA Approved Treatment for Adults with Tardive Dyskinesia

INGREZZA Launch Update $116MM in Net Product Sales, First 8 Months Net Product Sales ($ in Millions) $70 $60 $50 $40 $30 $20 $10 $- INGREZZA Net Product Sales and ~TRx $64 9,100 $46 5,100 $6 745 May/June '17 Q3'17 Q4'17 10,000 9,000 8,000 7,000 6,000 5,000 4,000 3,000 2,000 1,000 - Approximate TRx 2017 Launch Highlights Salesforce Established for May 1, 2017 Launch Market Awareness Created for TD Broad Prescriber Base Realized 2018 Launch Priorities Continued Disease State Education Brand Awareness INGREZZA Net Product Sales INGREZZA TRx (30 Day) 5

INGREZZA Overview TARDIVE DYSKINESIA AFFECTS APPROXIMATELY 500,000 PATIENTS IN THE US Involuntary Movement Disorder Caused by Prolonged Antipsychotic Use for Bipolar, Schizophrenia, and Depression More than a 400% Increase in Antipsychotic Prescriptions from 1990-2015 (~65MM TRx in 2017) Results in Dysregulation of Basal Ganglia Pathways Responsible for Movement Control z EFFICACY: RAPID AND ROBUST AIMS Week Six LS Mean Change from Baseline of -3.2 (80mg) vs. -0.1 in Placebo (KINECT 3) Significant Change Observed at 2 Weeks in Clinical Trials LABEL: NO BOXED WARNING No Contraindications No Dose Cap For CYP2D6 Poor Metabolizers Concomitant Use with Psychiatric Medications USE: ONCE A DAY, NO TITRATION Can Be Taken With or Without Food One Week to Reach Recommended Dose No Dosing Restrictions After Treatment Interruptions 6

Tardive Dyskinesia Overview: Symptoms Oral and Facial Dyskinesia Abnormal tongue and lip movements Retractions of the corners of the mouth Abnormal eyelid closure or eyebrow movements Bulging of the cheeks Chewing movement Limb Dyskinesia Piano-playing finger movements Tapping foot movements Dystonic extensor postures of the toes Trunk Dyskinesia Shoulder shrugging Axial Dystonia Twisting of the trunk Rocking and swaying movements Rotatory or thrusting hip movements Tarsy D. Curr Treat Options Neurol. 2000;2(3):205-214. 7

KINECT 3: INGREZZA Reduction in Abnormal Involuntary Movement Scores at Each Study Visit Through Week Six Improvement AIMS Change From Baseline by Study Visit (ITT Population) WEEK 0: Baseline WEEK 2 WEEK 4 WEEK 6 n=76 n=70 n=79 n=76 n=70 n=77 n=73 n=64 n=73 n=69 n=63 n=70 LS Mean Change From Baseline (SEM) Placebo INGREZZA 40 mg Once Daily INGREZZA 80 mg Once Daily P values vs placebo: * <0.05 (nominial), ** <0.01 (nominal), 0.001. AIMS change from baseline at weeks 2 and 4 not controlled for multiplicity. Data presented for ITT analysis set. Change in AIMS score analyzed by MMRM model. Treatment differences determined by comparison of LS means. Hauser RA, et al. Am J Psychiatry. 2017. Mar 21: doi: 10.1176/appi.ajp.2017.16091037. [Epub ahead of print]. Data on file. Neurocrine Biosciences. 8

KINECT 3: AIMS Change From Baseline for INGREZZA Groups Long-Term Extension Period Improvement AIMS Mean Change (SEM) From Baseline (ITT Population) Placebo INGREZZA 40 mg Once Daily INGREZZA 80 mg Once Daily Placebo to INGREZZA 40 mg Placebo to INGREZZA 80 mg PBO Controlled INGREZZA Blinded Extension Period Off Drug WEEKS 0 2 4 6 8 16 32 48 52 DB, double-blind. Data presented for ITT analysis set. INGREZZA [package insert]. San Diego, CA: Neurocrine Biosciences; 2017. 9

N E U R O C R I N E B I O S C I E N C E S, I N C.» Pipeline Highlights» 2018 Milestones

Recent Highlights Elagolix For Uterine Fibroids Elagolix for Uterine Fibroids Phase III Topline Data (ELARIS UF-1 and II) - Both Phase III Studies Met Primary Endpoint (p<0.001) Responder rates of 68.5%% and 76.2% (vs. 8.7% and 10.1% in placebo, respectively). Clinical response defined as menstrual blood loss volume of <80 ml during month six AND a >50% reduction in menstrual blood loss volume from baseline to month six. Met all secondary endpoints at month six. Women in the studies continue in either post-treatment follow-up or a blinded six-month extension study. Data to Be Presented at an Upcoming Conference 11

Recent Highlights Opicapone For Parkinson s Disease Opicapone Moving Forward with NDA Filing - No Additional Phase III Trial Needed to Support NDA Filing - Target Date for NDA Filing in 1H of 2019 - Commercial Preparations Ongoing for a 2020 Launch 12

Opicapone: Reducing Off-Time For Patients with Parkinson s Disease Parkinson s Disease (PD): Lifelong, Incurable, Progressive 2 nd Most Common Neurodegenerative Disease Following Alzheimer s Disease Approximately One Million Patient Cases in the United States While Incidence Rates Expected To Remain Constant, Prevalence Will Increase As A Result Of The Aging Population Increasing Life Expectancy >10M Elderly People By 2020 Approximately Two-Thirds of Patients on L- dopa/c-dopa therapy COMT Inhibition Reduces Wearing-Off Off-time and Increases On-time On Without Time vs Troublesome Off TimeDyskinesia Start of COMT Inhibition Therapy Sources: Datamonitor and Medical Literature review 13

Opicapone At-a-Glance Potential Best-in-Class COMT Inhibitor HISTORY NCE Discovered And Fully Developed By BIAL EMA Approved In June 2016 As ONgentys (Opicapone) Commercially-available In UK, Germany, Spain (Italy and Portugal, 2018) Partnered With ONO in Japan (PMDA Filing in 2018) In-licensed By Neurocrine in February 2017 EXTENSIVE CLINICAL AND REGULATORY DEVELOPMENT 1200 Healthy Subjects In >25 Phase I Studies 1000 PD Subjects in Phase II-III Studies (2 Pivotal Studies) Positive Phase III Comparator Study with the Standard of Care THE OPPORTUNITY Once A Day Reduced Pill Burden Safe and Well Tolerated Significantly More Efficacious Relative to the Standard of Care 14

Recent Highlights NBI-74788 For Congenital Adrenal Hyperplasia Initiated Phase II Study in Adult CAH Patients - Phase II Data in Q2 2018 - Completed Nonclinical Studies to Support Development in Pediatric Population - FDA End of Phase II Meeting Planning Ongoing to Support the Initiation of the Pivotal Program in 2018 15

Hypothalamic-Pituitary-Adrenal Axis (Normal) Cortisol Present CRF ACTH Endogenous Cortisol Androgens CORTISOL NEGATIVE FEEDBACK LOOPS CONTROL CRF, ACTH AND ANDROGEN LEVELS 16

Hypothalamic-Pituitary-Adrenal Axis (21-OHP CAH) Cortisol Absent NO CORTISOL NEGATIVE FEEDBACK LOOPS UNCONTROLLED CRF, ACTH AND ANDROGEN LEVELS 17

Hypothalamic-Pituitary-Adrenal Axis (CAH) Cortisol Therapy Exogenous Cortisol Replace Missing Cortisol With Supra-physiological Concentrations Causes Severe Immune And Metabolic Side Effects 18

Hypothalamic-Pituitary-Adrenal Axis (CAH) CRF Antagonist Therapy CRF ACTH Exogenous Cortisol Replace Missing Cortisol With Physiological Concentrations In Combination with CRF Antagonist Will Better Control Disease and Reduce the Drive to Androgens Androgens 19

Screening 24-Hour Baseline PD Baseline/Study Entry Dosing Period 14-Days Follow-Up Period & Final Study Visit Phase II Study (Adult CAH): NBI-74788-CAH2001 Study Periods (Repeat for each Dose Cohort) NBI-74788 Dose Escalation Cohort 2: Dose 2 Cohort 1: Dose 1 Study methods: N = 16-20 young adult females and males (ages 18-50) PD measures: 17-OHP, androgens, ACTH, cortisol Standard PK and safety: PK sample collection, AEs, vitals, PEs, clinical labs 20

NBI-74788 Classical Congenital Adrenal Hyperplasia (CAH) Potentially First-in-Class CRF-R1 Antagonist THE DISEASE Autosomal recessive genetic disorder resulting in: Impaired cortisol biosynthesis, increased ACTH, and abnormal androgen accumulation Diagnosed at birth following near universal adoption of neonatal screening for 17-OHP excess Persistent, excessive androgens cause precocious puberty, fertility issues, virilization in females CURRENT TREATMENT Glucocorticoid treatment to replace cortisol as well as activate HPA negative feedback loop to reduce ACTH levels, requires excessive dosing (supra-therapeutic) Consequence: growth impairment, bone loss, and iatrogenic Cushing s Syndrome PREVALENCE 20,000-30,000 patients in US Qualifies for Orphan Drug Designation by FDA PROGRAM GOAL: CURRENTLY IN PHASE II (ADULTS) The treatment of classical congenital adrenal hyperplasia (CAH) associated with high adrenocorticotropin levels, androgen excess and adrenal steroid insufficiency Maintain physiologic glucocorticoid replacement while normalizing ACTH to reduce overproduction of androgens 21

2018 Milestones Pipeline Expanding and Advancing Program elagolix opicapone elagolix New NBI-74788 valbenazine Phase III Data For Uterine Fibroids Milestone FDA Meeting with FDA (Preparation for 1H 2019 NDA Filing) PDUFA Date For Endometriosis IND Submission And Initiation Of Phase I For Internally Discovered Small Molecule Phase II Data For CAH (Adults) Phase III Initiation For CAH (Adults) Phase II Initiation For CAH (Peds) Phase IIb Data for Tourette Syndrome 22

Four Years = Four Products and Six Indications Starting with INGREZZA in Tardive Dyskinesia, Pipeline Will Deliver Long-term Growth Tardive Dyskinesia Tourette Syndrome elagolix Endometriosis Uterine Fibroids opicapone Parkinson s Disease NBI-74788 Congenital Adrenal Hyperplasia 23