Neurocrine Biosciences, Inc. · NASDAQ:NBIX
Neurocrine Biosciences — Investor Day (December 2023)
53 slides from Neurocrine Biosciences's investor day presentation, Dec 5, 2023
Slide 1: Neurocrine Biosciences 2023 Analyst Day
Neurocrine Biosciences 2023 Analyst Day December 5, 2023
Slide 2: Dr. Kevin Gorman, Chief Executive Officer
Dr. Kevin Gorman CHIEF EXECUTIVE OFFICER 2
Slide 3: Neurocrine Biosciences Attendees
Neurocrine Biosciences Attendees Kevin Gorman, Ph.D. Matt Abernethy Eric Benevich David Boyer Chief Executive Officer Chief Financial Officer Chief Commercial Officer Chief Corporate Affairs Officer Julie Cooke Ingrid Delaet, Ph.D. Kyle Gano, Ph.D. Dimitri E. Grigoriadis, Ph.D. Chief Human Resources
Slide 4: Agenda
Agenda TIME (EST PM) Topic Speaker(s) 1:00 – 2:00 Welcome / CEO Opening Remarks Kevin Gorman (R&D Focus) R&D Vision and Strategy Jude Onyia Jude Onyia, Eiry Roberts, Kyle Gano, Jerold Chun, Grace R&D Q&A Session Liang 2:00 – 3:15 A Brief History of CRF Kevin Gorman / Dimitri Grigoriadis (CAH Focus)
Slide 5: Forward-Looking Statements
Forward-Looking Statements 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 regarding the potential benefits to be derived from certain of our product
Slide 6: Where Are We Today?
Where Are We Today? • Discovered and Developed Three Novel FDA-Approved Programs • Deep Expertise in Neuroscience Drug Development • Fully-Integrated Organization with Both R&D and Commercial Capabilities • Growing Blockbuster Commercial Product in INGREZZA with Strong IP Protection • Future Blockbu
Slide 7: Building and Maximizing the Pipeline
# of Programs by Stage Building and Maximizing the Pipeline Phase 1 5 Phase 2 8 Phase 3 4 NDA 1 Phase 1 Phase 2 Phase 3 NDA Milestone Neurology valbenazine* Sprinkle Formulation VMAT2 Inhibitor PDUFA: 4/30/2024 valbenazine* Dyskinetic Cerebral Palsy VMAT2 Inhibitor Phase 3 Ongoing NBI-8271042 EE-CSW
Slide 8: Dr. Jude Onyia, Chief Scientific Officer
Dr. Jude Onyia CHIEF SCIENTIFIC OFFICER 8
Slide 9: R&D Vision
R&D Vision Top 5 Global Leadership in Neuroscience Five Year Plan Long-Term Steady State Produce 20 Development Candidates* Produce One Commercial Product Every Two Years • “20 in 5” Goal Will be a Product of Internal Research and External Innovation • Requires Several “Shots on Goal” Per Year and S
Slide 10: R&D Strategy
R&D Strategy Build Breadth and Depth Strengthen Therapeutic Area Expertise Strengthen Existing Modality + Build New Modalities Shift Focus to Higher Probability “Best & Next-In Class” Targets Deploy External Innovation to Accelerate R&D Goal: Deliver A Diversified Portfolio Across Therapeutic Areas
Slide 11: Committed to Improving Our Probability of Success
Committed to Improving Our Probability of Success Focus Areas to Improve Probability of Success Deep Understanding Clinical and / or Genetic Guiding of Disease and Unmet Need Validation Principles Right Targets Translationally Relevant Leverage Right Drugs Endpoints Shared Pathophysiological Mechani
Slide 12: Therapeutic Areas of Focus
Therapeutic Areas of Focus 12
Slide 13: Therapeutic Areas of Focus
Therapeutic Areas of Focus Prioritizing Heavier Investment in Neurology vs. Neuropsychiatry Today Neurology Neuropsychiatry (25%) (50%) Neuroendocrinology Neuroimmunology (15%) (10%) 13
Slide 14: What Do We Want To Add Over Next Five Years?
What Do We Want To Add Over Next Five Years? Neurology Neuropsychiatry Neuroendocrinology Movement Disorders Schizophrenia Congenital Adrenal Hyperplasia Epilepsy Depression Metabolic Disease Neuromuscular Bipolar Mania Rare Endocrinology Neurodegenerative/ Alzheimer’s Disease Psychosis Neurodevelop
Slide 15: Modalities
GD NEYROGRINE » Modalities
Slide 16: Modalities Expand the Range of Therapeutic Innovation
Modalities Expand the Range of Therapeutic Innovation Biologics Science Technological Advances Antibodies Gene Therapies + Strategy Peptides Small Molecules + Investment Target Specificity & Molecular Weight 16
Slide 17: New Opportunities Via Modalities Across Therapeutic Areas
New Opportunities Via Modalities Across Therapeutic Areas Tech Platforms Small Molecules Peptides Antibodies Gene Therapy Neuropsychiatry ✓ ? ? Neurology ✓ ✓ ✓ ✓ Neuroendocrinology ✓ ✓ ✓ ✓ Neuroimmunology ✓ ✓ ✓ ✓ 17
Slide 18: Where Do We Point The Engine?
Where Do We Point The Engine? Doing All 3 Game Changer We Can to Win? 75 % Opportunity 2 [Unmet Need] 1 Should We Do This? Focus on High Confidence and High Opportunity Targets 1 2 3 4 5 Confidence / Ability to Deliver [Biology/Chemistry/Clinical] 18
Slide 19: Molecule Design & Engineering
Molecule Design & Engineering Developing a Competitive Advantage at Neurocrine Biosciences EFFICACY SAFETY Design Principles — 1) Better pharmacology 1) Improve safety Patient Outcome Centric 2) Added pharmacology 2) Improve specificity Maximize Efficacy 3) Better time action 3) Reduce immunogenicit
Slide 20: Leveraging External Innovation as a Multiplier
Leveraging External Innovation as a Multiplier 20
Slide 21: Leveraging External Innovation for Over 30 Years
Leveraging External Innovation for Over 30 Years We Remain Agnostic To Deal Structure OPTION-TO-LICENSE FOR RISK COMMERCIAL PRODUCT MANAGEMENT Increase Topline Growth, Accelerate Presence in Disease States Supported by Pipeline Programs • Relevant for Early-Stage Deal • Limits Neurocrine Resources U
Slide 22: Internal R&D Will Meet External Innovation to Build Our Pipeline
Internal R&D Will Meet External Innovation to Build and Sustain Our Pipeline Treatment Company Targets/Modalities Examples Commercial: ORILISSA®, ORIAHNN® (GnRH) Phase 3: Crinecerfont (CRF) GPCRs Phase 2: NBI-’568 (Muscarinic M4) Phase 1: NBI-’570 (Muscarinic M1/M4 Dual) Symptomatic Enzymes, Transpo
Slide 23: Emerging Pipeline
Emerging Pipeline: Building Blocks for Rapid / Sustainable Portfolio Growth 23
Slide 24: Early R&D Trends Are Positive With Increased Number of Assets
Early R&D Trends Are Positive With Increased Number of Assets and Faster Timeframe Leading to Robust Early Clinical Pipeline • 10 New Development Candidates since 2021 (Includes Internal and External Compounds) Number of Development Number of Eligible Candidates by Year Phase 1 Candidates • 6 Clinic
Slide 25: 2024 Portfolio Pipeline Snapshot
2024 Portfolio Pipeline Snapshot Discovery and Preclinical Clinical Development NDA Select Programs Phase I Phase II Phase III NBI-770 — NMDA valbenazine — sprinkle #1 #9 #1 NBI-567 — M1 Preferring Ag valbenazine — ATS NR2B NAM (Oral) #2 #10 #2 NBI-569 — M4 Preferring Ag NBI-568 — Schizophrenia valb
Slide 26: 2024 Portfolio Pipeline Snapshot
2024 Portfolio Pipeline Snapshot Therapeutic Areas AREAS THERAPEUTIC Modalities Targets Small Molecules 20% Neuropsychiatry 50% Novel Neurology (25%) (50%) Neuroendocrinology Neuroimmunology (15%) (10%) Biologics 50% 80% Clinically Or Genetically Validated 26
Slide 27: "Must Win" Areas of Focus
“Must Win” Areas of Focus VMAT2 CRF-R1 Muscarinic Gene Inhibitors Antagonist Programs Therapy Neuropsychiatry / Neuropsychiatry / Neuroendocrinology (+) Neurology Neurology Neurology NBI-890 Crinecerfont NBI-568 GBA1 (Oral QD / Low Dose / (M4 Agonist) Parkinson’s Disease / LAI) CRF R1 Follow-On Gauc
Slide 28: Our R&D Strategy Is Delivering Results
Our R&D Strategy Is Delivering Results Top Talent Bench of Chemists and Biologists in Place Developed Engineering Capabilities Across a Variety of Diversified Modalities Focused on our Core Therapeutic Areas (TAs) of Interest Advanced Early Portfolio is Now Diversified Across TAs and Modalities Pre-
Slide 29: R&D Q&A Session
GD NEYROGRINE » R&D Q&A Session
Slide 30: Dr. Dimitri Grigoriadis, Chief Research Officer
Dr. Dimitri Grigoriadis CHIEF RESEARCH OFFICER 30
Slide 31: Neurocrine Biosciences: Founded on a CRF Platform
Neurocrine Biosciences: Founded on a CRF Platform 30+ years of R&D on CRF1 Receptor Antagonists for Neuroendocrine Diseases Positive Phase 3 1981 Pediatric CAH 2023 CRF Identified Recent Positive Phase 3 Results for Crinecerfont in Adult AND Pediatric CAH Positive Human Phase 3 CRF2 Receptor Adult C
Slide 32: Neurocrine Biosciences: Our History with CRF
Neurocrine Biosciences: Our History with CRF R&D Efforts Across the Spectrum of the CRF System GPCRs, Neuroendocrine Peptides and Binding Protein CRF1 Receptor Antagonists NBI-30775 | Phase 2 NBI-34041 | Phase 2 GW876008 | Phase 2 5 Industry-Wide Clinical Programs Discovered NBI-77860 | Phase 2 / As
Slide 33: Dr. Eiry Roberts, Chief Medical Officer
Dr. Eiry Roberts CHIEF MEDICAL OFFICER 33
Slide 34: Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency
Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (21OHD CAH) 21OHD CAH Results in: • Impaired Synthesis of Cortisol and (Often) Aldosterone • Excess Adrenal Androgen Production Treatment Must Balance Consequences of: • Supraphysiologic Glucocorticoid (GC) Doses • High ACTH and
Slide 35: Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency
Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (21OHD CAH) High ACTH and Androgen Excess TARTs Cortisol Excess From Supraphysiologic GC Dosing Hirsutism, Infertility amenorrhea Psychological 21OHD CAH Results in: Diabetes, Abnormal and/or early effects insulin resistance Sho
Slide 36: Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency
Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (21OHD CAH) Cortisol Excess From Supraphysiologic GC Dosing Obesity Cardiovascular striae Diabetes, Psychological High ACTH and Androgen Excess insulin resistance 21OHD CAH Results in: risk Short stature Fractures, effects Hirsu
Slide 37: Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency
Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (21OHD CAH) 21OHD CAH Results in: Reduced GC Dosing crinecerfont Androgen Control • Impaired Synthesis of Cortisol and (Often) Aldosterone • Excess Adrenal Androgen Production Treatment Must Balance Consequences of: • Supraphysi
Slide 38: CAH Panel Discussion
CAH Panel Discussion MODERATED BY JEAN L. CHAN AND BOB FARBER 38
Slide 39: Congenital Adrenal Hyperplasia Panelists
Congenital Adrenal Hyperplasia Panelists Richard Auchus, M.D., Ph.D. Lesley Holroyd University of Michigan CAH Patient Advocate, (Adult Endocrinologist, CAH Expert) CARES Foundation Board Member Kyriakie Sarafoglou, M.D. Alexandra Dubois University of Minnesota CAH Parent Caretaker Advocate, (Pediat
Slide 40: Crinecerfont Phase 3 Study Results (Adults & Pediatrics)
Crinecerfont Phase 3 Study Results (Adults & Pediatrics) 40
Slide 41: CAHtalyst Adult Study Design
CAHtalystTM Adult Study Design Open- Double-Blind Placebo Controlled Treatment Open-Label Treatment Screening Label (6 months) (one year) Extension* Crinecerfont Crinecerfont Placebo Day 1 Week 24 Month 18* Participants randomized to Continued access to crinecerfont placebo will receive crinecerfont
Slide 42: CAHtalyst Pediatric Study Design
CAHtalystTM Pediatric Study Design Open- Double-Blind Placebo Controlled Treatment Open-Label Treatment Screening Label (28 weeks) (24 weeks) Extension* Crinecerfont Crinecerfont Placebo Day 1 Week 4 Week 28 Week 52* Primary Endpoint Participants randomized to Continued access to crinecerfont Measur
Slide 43: CAHtalyst Adult and Pediatric Study Design Comparisons
CAHtalystTM Adult and Pediatric Study Design Comparisons Similarities Differences Key Endpoints Focused on Androgen Reduction and Ordering of Endpoints (Primary vs. Steroid Dose Reduction with Androgen Control Key Secondary) • Same definition of endpoints, same target dose Management of Steroid Redu
Slide 44: CAHtalyst Adult and Pediatric Study Baseline Characteristics
CAHtalystTM Adult and Pediatric Study Baseline Characteristics Adult Study Pediatric Study Study Characteristic Key Takeaways (N = 182) (N = 103) Male / Female 51% Male 52% Male Nearly Equal Male and Female (Proportion of Total Subjects) 49% Female 48% Female Average Age 31 Years Old 12 Years Old Br
Slide 45: CAHtalyst Adult and Pediatric Study Androgen Reduction
CAHtalystTM Adult and Pediatric Study Androgen Reduction Percent Change* in Androstenedione at Week 4 (Following Glucocorticoid Stable Period) Adult Study Pediatric Study Study Characteristic Key Takeaways (N = 182) (N = 103) Substantial and Meaningful Reduction in Patients Receiving Crinecerfont -4
Slide 46: CAHtalyst Glucocorticoid Dose Reduction While Maintaining Androgen Control
CAHtalystTM Adult and Pediatric Study Glucocorticoid Dose Reduction While Maintaining Androgen Control Percent of Subjects Achieving a Glucocorticoid Daily Dose ≤ 11 mg/m2/day While Maintaining Androgen Control Adult Study @ Pediatric Study @ CAHtalystTM Trial Participants Key Takeaways Week 24 Week
Slide 47: CAHtalyst Adult and Pediatric Study Safety and Tolerability
CAHtalystTM Adult and Pediatric Study Safety and Tolerability • Crinecerfont Treatment was Generally Well-Tolerated with Few Serious Adverse Events (SAEs), None Were Assessed as Related to Crinecerfont • Most Common Adverse Events During the Double-Blind, Placebo-Controlled Period of the Adult Study
Slide 48: CAH / Crinecerfont Q&A Session
CAH / CRINECERFONT Q&A SESSION 48
Slide 49: Dr. Kevin Gorman, Chief Executive Officer
Dr. Kevin Gorman CHIEF EXECUTIVE OFFICER 49
Slide 50: Key Milestones in 2024 and Longer Term
Key Milestones in 2024 and Longer Term 2024 Longer Term • April PDUFA for Valbenazine Oral Granules Sprinkle • Anticipated Crinecerfont Approved in U.S. (2025) and in Formulation Europe (2026) • Submitting New Drug Application for Crinecerfont in Adult and • Advancement of 2 Gene Therapy Programs In
Slide 51: Open Q&A Session
GD NEYROGRINE » OPEN Q&A SESSION
Slide 52: Thank You!
GD NEYROGRINE » THANK YOU!
Slide 53: Neurocrine Biosciences 2023 Analyst Day
Neurocrine Biosciences 2023 Analyst Day December 5, 2023
Neurocrine Biosciences — Investor Day (December 2023) on 6ix