Neurocrine Biosciences, Inc. · NASDAQ:NBIX
Neurocrine Biosciences — Q3 2011 Earnings Presentation
31 slides from Neurocrine Biosciences's earnings presentation, Oct 31, 2011
Slide 1: Neurocrine
V/ Neurocnine
Slide 2: Forward-looking statements and risks
This presentation includes early and late stage clinical data and projections based thereon that involve a number of risks and uncertainties. These risks include the specific risk that the clinical development of the products discussed will not successfully proceed to later stage clinical trials or
Slide 3: Neurocrine Overview
Neurocrine Overview • Neurological and Endocrine Based Diseases • Diverse Pipeline » GnRH Phase III program partnered with Abbott (Endometriosis) » GnRH Phase II program partnered with Abbott (Uterine Fibroids) » VMAT2 program in Phase II, wholly owned, multiple indications » Urocortin2 program in P
Slide 4: A Pipeline of Innovation
A Pipeline of Innovation Program and Indication Preclinical Phase 1 Phase 2 Phase 3 Women’s and Men’s Health Endometriosis Pain Elagolix Uterine Fibroids Elagolix Endometriosis, Oncology GnRH Antagonists Central Nervous System Post-Traumatic Stress Disorder GSK-561679 Tardive Dyskinesia NBI-98854 Sc
Slide 5: Elagolix GnRH Program
Urocortin 2 for Elagolix Congestive GnRH Program Heart Failure (CHF)
Slide 6: A First-in-Class Opportunity
A First-in-Class Opportunity Elagolix Product Attributes Improved tolerability Rapid No flare recovery effect time Bone Fast Onset neutral Oral No Dosing Elagolix tolerance 6
Slide 7: A Pipeline Within a Program
A Pipeline Within a Program Follow-On Elagolix NBI 98341 NBI 97934 Primary Indications Primary Indications Oncology Endometriosis Uterine Fibroids Men’s Health Prostate Breast Additional Indications Menorrhagia PCOS Adenomyosis Primary PMDD ART Dysmenorrhea 7
Slide 8: Endometriosis is Pain
Endometriosis is Pain “It’s disabling. I’ll do everything I have to do but I don’t feel like I am BLEEDING reaching my full potential… it’s honestly a huge disadvantage.” PAIN “I live my life based on this (endometriosis). I have to INFERTILITY rearrange my whole life based on when this is going to
Slide 9: Current Activities
Current Activities • Finalizing Phase III Protocol for Endometriosis following productive end of phase II meeting with FDA » Endpoints – Non-Menstrual Pelvic Pain and Dysmenorrhea » Statistical Methods – Responder Analysis » Trial Design – Two trials, six-month placebo controlled, (>800 patients/stu
Slide 10: Uterine Fibroids (Leiomyomas)
Uterine Fibroids (Leiomyomas) Symptoms & Pathophysiology • Benign tumors of muscle and connective tissue that Disease develop within or are attached to the uterine wall • Also known as leiomyoma • Leiomyomas are estrogen sensitive and have estrogen receptors Patho- • Fibroids tend to regress after m
Slide 11: Uterine Fibroids Symptoms
Uterine Fibroids Symptoms Pain and Menorrhagia Common Urinary incontinence 30.0% • Hemorrhagia • Menorrhagia Pelvic or bladder • Pelvic pain 81.0% pressure • Bloating • Cramping Pelvic pain 63.0% • Chronic fatigue • Headaches Bleeding - Heavy or very 94.0% • Anemia heavy • Depression 0% 20% 40% 60%
Slide 12: Uterine Fibroids Treatment Continuum
Uterine Fibroids Treatment Continuum Suboptimal Options Option Issues Wait for menopause It could be a while… NSAIDS (e.g. Advil) Insufficient pain relief Opioids Tolerance and addiction Contraceptives (combination or Insufficient pain relief; risks with specific OCs (bone progestin-based) loss) GnR
Slide 13: Surgical Options (US)
Surgical Options (US) 250,000 Hysterectomies Annually Greater 650,000 Hysterectomies Performed Annually >12 MM Worldwide 300,000 258,774 250,000 200,000 172,516 150,000 123,226 110,903 100,000 50,000 0 Endometriosis Uterine Fibroids Uterine Prolapse Other 13 (1) Whiteman et al Am J Obstet & Gynecol
Slide 14: Elagolix Intellectual Property
Elagolix Intellectual Property Market Exclusivity Provides Market Expansion Opportunities 6 Issued U.S. Patents covering: • Elagolix composition of matter • Pharmaceutical composition • Methods of using elagolix Composition of matter patent expires 2024 (not including patent term extension of ap
Slide 15: VMAT2 Inhibitor Program
Urocortin 2 for VMAT2 Congestive Inhibitor Program Heart Failure (CHF)
Slide 16: VMAT2 Inhibition: Potential for Multiple Conditions
VMAT2 Inhibition: Potential for Multiple Conditions Neurocrine Development Strategy Tardive Dyskinesia Currently approved therapies: None Tourette’s NBI-98854 Syndrome Currently approved therapies: None Schizophrenia Currently approved therapies: dopamine receptor antagonists (atypical/typical
Slide 17: Normal VMAT2 and Dopamine Activity
Normal VMAT2 and Dopamine Activity VMAT2 Dopamine Dopamine Transporter Dopamine Receptors
Slide 18: Tardive Dyskinesia Pathophysiology
Tardive Dyskinesia Pathophysiology Excessive Dopamine and Receptor Sensitivity
Slide 19: VMAT2 Inhibition Reduces Vesicular Dopamine
VMAT2 Inhibition Reduces Vesicular Dopamine NBI 98854
Slide 20: Selective VMAT2 Inhibition
Selective VMAT2 Inhibition Normalization of Dopaminergic Tone
Slide 21: Objectives of Exploratory Phase IIa Trial
Objectives of Exploratory Phase IIa Trial NBI-98854-1001 • Assess safety and tolerability of NBI-98854 » Doses of 12.5 mg, 25 mg and 50 mg over 12 days of dosing with 7-day wash-out post treatment » Schizophrenia or schizoaffective disorder subjects with moderate or severe Tardive Dyskinesia (i.e.,
Slide 22: AIMS-Abnormal Involuntary Movement Scale
AIMS-Abnormal Involuntary Movement Scale • Clinician-rated scale to assess severity of dyskinesia during structured neurological examination (developed in 1976) • Seven body regions assessed for involuntary movement » Facial or Oral Movements Expression, Lips, Jaw, Tongue » Extremity Movements E
Slide 23: Positive Phase IIa Results – AIMS (Items 1-7)
Positive Phase IIa Results – AIMS (Items 1-7) Mean Score and Study Period*: 12 Days of Dosing 16 14.3 12.8 12 41% reduction in AIMS with treatment 8.4 8 4 0 Baseline End of Dosing Post Treatment n=6 n=6 n=6 * Mean AIMS scores reflect the following: Baseline: Day –1 and Day 1 pre-dose End of Dosing:
Slide 24: Initial Phase II Study Summary
Initial Phase II Study Summary • NBI-98854 is generally safe and well tolerated at doses up to 50 mg per day during 12 days of exposure » Stable to improved BPRS, transient mild or moderate AEs, one SAE post treatment • Pharmacokinetic data consistent with predictions » Dose proportional exposure •
Slide 25: 2011 Clinical Development VMAT2
2011 Clinical Development VMAT2 • Pre-IND meeting held in June 2011 • Submitted IND to FDA Division of Psychiatry • CMC work on solid dose formulation ongoing • Placebo controlled two-week Phase II study in tardive dyskinesia initiated in September 2011 • Three month toxicology data available in lat
Slide 26: Design of Second Phase II VMAT2 study
Design of Second Phase II VMAT2 study • Tardive dyskinesia patients • Randomized, double-blind, placebo controlled, cross-over trial • 32 patient study (four arms) • Once-daily NBI-98854 (12.5mg and 50mg) • Two-week dosing period • Abnormal Involuntary Movement Scale (AIMS) • Within-subject comparis
Slide 27: Phase II Study Design
Phase II Study Design NBI-98854-1101: 2-Period Cross-over Study (14 Days/Period) Treatment Period 1 Treatment Period 2 Placebo NBI-98854 (12.5mg qd) NBI-98854 (12.5mg qd) Placebo Placebo NBI-98854 (50mg qd) NBI-98854 (50mg qd) Placebo
Slide 28: VMAT2 Composition of Matter Patent
VMAT2 Composition of Matter Patent • Notice of Allowance for the Company’s proprietary Vesicular Monoamine Transporter 2 inhibitor NBI-98854 » Received notice from USPTO June 2011 • May 2029 patent expiration date • With Hatch/Waxman term could extend to 2034 » Maximum period of exclusivity is 14 ye
Slide 29: Planned Milestones for 2011
Planned Milestones for 2011 •March 2011 end of Phase II meeting with FDA for elagolix use in endometriosis •April 2011 VMAT2 Phase IIa study results •Mid-2011 open IND for VMAT2 in United States •Composition of Matter Patent for VMAT2 •Sept 2011, Elagolix presentation at World Endometriosis Con
Slide 30: Planned Milestones for 2012
Planned Milestones for 2012 • Q1 Results of 14-day placebo controlled VMAT2 Phase II study • Q1 Three month toxicology results VMAT2 • Q2 Initiate Phase IIb three-month tardive dyskinesia study in Schizophrenia patients • Q2 Initiate Phase IIb three-month tardive dyskinesia study in bipolar and depr
Slide 31: Financial Highlights
Financial Highlights Year Ended Nine-Months Ended December 31, 2010 September 30 , 2011 Revenues and other Income $ 39.3 million $ 69.0 million Operating Expenses $ 47.2 million $ 32.8 million Net Income (Loss) $ (8.0) million $ 36.2million Cash, Investments, Receivables $ 135.1 million $ 140.2 mill
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