Neurocrine Biosciences, Inc. · NASDAQ:NBIX
Neurocrine Biosciences — Non-Deal Roadshow (January 2005)
34 slides from Neurocrine Biosciences's roadshow presentation, Jan 31, 2005
Slide 1: Neurocrine Biosciences
CO it eA ie Neurocrine Biosciences
Slide 2: This presentation includes early and late stage clinical data and projections based thereon that
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: Research & Development Pipeline
Research & Development Pipeline RESEARCH AND DEVELOPMENT PIPELINE Program Targeted Indication Status Indiplon IR Insomnia NDA Indiplon MR Insomnia NDA GnRH Antagonist Endometriosis, Benign Prostatic Hyperplasia Phase I Altered Peptide Ligand Multiple Sclerosis Phase II Altered Peptide Ligand Type 1
Slide 4: Indiplon for Insomnia
Indiplon for Insomnia
Slide 5: Commercial Operations Overview
Commercial Operations Overview Indiplon
Slide 6: Commercial Operations Current Status / Future Developments
Commercial Operations Current Status / Future Developments — Indiplon Marketing Team (5) in place, Co-Managing Alliance Marketing with Pfizer — Sales Director Hired — Outsourced Vendors Selected for Recruiting, Sales Force, Systems, Fleet, etc. — Territory Design Completed for 200 person Sales Organ
Slide 7: Regulatory Indiplon
Regulatory Indiplon
Slide 8: Evolution of NDA Submissions
Evolution of NDA Submissions • Paper NDAs • Electronic (paper) NDAs • eCTD (fully electronic) NDAs (July/August ’04)
Slide 9: Submission Plan
Submission Plan MR NDA Module 1 MR Module 1 IR IR NDA Module 1 Patent Info/Cert Financial Disclosure Module 2 Module 2 MR Module 2 IR Re-file IR and MR Common Table of Contents DP - MR Table of Contents DP-IR NDA’s following Sections January FDA meeting. Module 3 API Module 3 DP/MR Module 3 DP/IR Mo
Slide 10: Indiplon Development
Indiplon Development IND – Phase III Division of Neuropharmacological Drug Products (Neuropharm) Phase III Division of Anesthesia and Critical Care Pre-Registration NDA – Neuropharm NDA Review Neurology Psychiatry
Slide 11: Clinical Update Indiplon
Clinical Update Indiplon
Slide 12: Summary of Phase III - IR Results
Summary of Phase III - IR Results p-values 0209 0115 0113 35d R1 0215 0114 MOTN Transient (LPS) ITT (LSO) ITT 3 mos (LSO) ITT (LSO) ITT (LPS) ITT (V4) Week 1 Week 2 Month 1 Month 2 Month 3 10 20 10 20 10 20 5 10 5 10 10 20 10 20 10 20 LPS (ITT) SE SE 1st third SE 2nd third TST WASO WASO 7hr NAASO LS
Slide 13: Summary of Phase III - MR Results
Summary of Phase III - MR Results p-values 0221 MR 0310 MR 0217 MR 0211 MR 0212 MR 0306 MR 2wk Adult Transient 2wk Elderly 3 Month (sTST) Elderly 35d (WASO) 2wk (sTST) (LPS) (sTST) Elderly (sTST) W1 W2 W1 W2 M1 M2 M3 N 1/2 N 15/16 N 29/30 W1 W2 30 30 20 30 20 30 20 30 20 30 20 30 20 30 20 30 20 30 1
Slide 14: Development Pipeline
Development Pipeline
Slide 15: Small Molecule / Oral GnRH Antagonist
Small Molecule / Oral GnRH Antagonist
Slide 16: Target for Development of Small Molecule GnRH Antagonists
Target for Development of Small Molecule GnRH Antagonists — Oral bioavailability • Avoiding monthly injections of agonists — No agonist activity • Avoiding “flare” phenomenon of agonists — Able to modulate hormone levels rather than cause medical castration • Avoiding deleterious effects of medicall
Slide 17: WW Market Value & Opportunities
WW Market Value & Opportunities Endometriosis & BPH 1,2 Significant Unmet Need in Market Value, Major Brands Men & Women’s Health 2003 Sales, WW (>$5.5 B) Prevalence 5,900 Endometriosis GnRH Agonists (TOTAL) $2,533 Diagnosed 1,305 GnRH Agonists Treated - 115 (Endometriosis) $417 Significant GnRH Ago
Slide 18: GnRH Program Status
GnRH Program Status Study 0301 PK/PD effects of NBI-56418 in females 9 Tolerability of single and 7 day dosing in females 9 Study 0402 PK/PD effects of NBI-56418 in males 9 Tolerability of single dosing in males 9 Study 0405 PK/PD effects of 42 days NBI-56418 in females ongoing Tolerability of 42 da
Slide 19: Effect of Multiple Doses on Estradiol
NBI 56418-0301 Effect of Multiple Doses 50 mg QD and 100 mg BID on Estradiol NBI-56418 able to achieve targeted Estradiol concentration range 250 Placebo Dosing period 50 mg QD 200 100 mg BID Estradiol (pg/ml) 150 ineffective 100 50 target estradiol concentration bone loss 0 0 1 2 3 4 5 6 7 8 9 10 m
Slide 20: NBI-56418: Next Steps
NBI-56418: Next Steps Next Steps Status Establish tolerability to 6 weeks 9 Complete exposure Determine estradiol response over 6 Sample analysis in progress; week duration to assist dose selection results by February for Phase II Initiate Phase II trial in endometriosis In planning, first patient Q
Slide 21: Urocortin for Congestive Heart Failure (CHF)
Urocortin 2 for Congestive Heart Failure (CHF)
Slide 22: NBI-69734: Urocortin 2 In Vitro Biology
NBI-69734: Urocortin 2 In Vitro Biology — A 38 amino acid peptide — A member of the CRF family of mammalian neuropeptides — Binds with high selectivity to CRF2 receptor subtype — CRF2 expressed in heart, vasculature, skeletal muscle, lung, intestine and adrenal cortex — Identified and cloned by W. V
Slide 23: NBI-69734: Comparison to Standard of Care
NBI-69734: Comparison to Standard of Care Therapeutic ↓ Preload ↓ Afterload ↑ CO Nitrates Yes No No Diuretics Yes No No Morphine No Yes No Inotropes No Some Yes Nesiritide Yes No Some Projected Clinical Yes Yes Yes Effects of Urocortin 2
Slide 24: Cardiac Effects of Urocortin (Pilot Study)
Cardiac Effects of Urocortin 2 (Pilot Study) 14 8 Cardiac Output (L/min) - bars Urocortin II (ng/mL) - area 12 7 6 10 5 8 4 6 3 4 2 2 1 0 0 Time (30 min intervals) Pre-Dose Placebo infusion 1-hour 50µg infusion 2-hour 100µg infusion Recovery 0-60min Washout 120-180min Washout 300-360min 390-840min
Slide 25: Hemodynamic Effects of Urocortin
Hemodynamic Effects of Urocortin 2 Required Pilot Goal Metric Results Assessment Heart Rate (Pulse) ≥ 15% ↑ 35% ↑ ; Mean Blood Pressure ≥ 5% ↓ 18% ↓ ; Cardiac Output ≥ 15% ↑ 68% ↑ ;
Slide 26: Planned Phase I Studies
Planned Phase I Studies — Primary Healthy Volunteer Study – Ongoing • Doses: placebo, 25 and 100µg in 1-hour infusion • Nov 2004 – Mar 2005 — Mild CHF Patient Study – 2Q05 • Doses: placebo, ≤50 and 100µg in 1-hour infusion • Duration: Apr – Jul 2005 • Criteria for successful outcome – Improved hemod
Slide 27: Development of Altered Peptide Ligands
Antigen Presenting Cell Development of MHC Altered Peptide Ligands TH2 Cells for the T-Cell TH1 APL APL T-Cell Treatment of Cytokines TH2 Autoimmune Diseases
Slide 28: APLs - Diabetes
APLs - Diabetes Type I Diabetes (NBI-6024) Phase II – Enrollment target of 188 achieved in 1Q04 • 94% retention rate – 25% completed 2-year dosing – Dose/schedule: 0.1/0.5/1.0 mg biweekly/monthly for 24 months – Interim analysis in 3Q05 – No safety issues – Endpoints: c-peptide, insulin use, HbA1c,
Slide 29: APLs - Multiple Sclerosis
APLs - Multiple Sclerosis Multiple Sclerosis (NBI-5788) Phase II –~ 40 sites in NA and EU –Enrollment: 40% complete; 100% by 2Q05; facilitated by addition of Eastern European countries in Dec ‘04 –Dose/schedule: 5 mg vs. placebo; 9 mos treatment; weekly for 5 wks then monthly –Primary Endpoint: MRI
Slide 30: CRF Receptor Antagonists
CRF STRESS STRESS CRF AVP Corticotrope Corticotrope Receptor Asn34 Arg35 Arg35 Lys36 Asn34 Lys Met38 Glu39 36 Glu39 38 Met Ile41 Ile41 Ile40 Ile40 ββ-Endorphin -Endorphin ACTH Gln30 Ser33 Gln30 Ser33 Cortex Cortex Adrenal Adrenal Gln29 Ala31 Gln29 Ala31 Leu37 Leu37 Antagonists Glu25 Gln26 Glu25 Gln2
Slide 31: Depression, Anxiety and IBS
Depression, Anxiety and IBS GlaxoSmithKline Collaboration • Program Status: – Compound selected as clinical candidate: Phase I, 4Q04 – Phase I single dose escalating trial complete – Two advanced preclinical candidates – Advanced lead compounds ready for the clinic in 2005 • Terms of the Collaborati
Slide 32: Financial Highlights
Financial Highlights
Slide 33: Statement of Operations
Statement of Operations Nine Months Ended September 30, 2004 and 2003 (in thousands) 2004 2003 Sponsored Research and Development $ 16,480 $ 81,651 License Fees and Milestones 49,803 29,306 Grant Income 408 986 Total Revenues $ 66,691 $ 111,943 Research and Development $ 81,662 $ 138,184 General and
Slide 34: Neurocrine Biosciences, Inc.
Neurocrine Biosciences, Inc.
Neurocrine Biosciences — Non-Deal Roadshow (January 2005) on 6ix