Neurocrine Biosciences, Inc. · NASDAQ:NBIX
Neurocrine Biosciences — Q3 2024 Earnings Presentation
49 slides from Neurocrine Biosciences's earnings presentation, Oct 30, 2024
Slide 1: Advancing Life-Changing Discoveries in Neuroscience
Advancing Life-Changing Discoveries in Neuroscience Q3 2024 Corporate Presentation October 30, 2024 Nasdaq: NBIX
Slide 2: Safe Harbor Statement and Non-GAAP Financial Measures
Safe Harbor Statement and Non-GAAP Financial Measures 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
Slide 3: Well-Positioned for Sustained & Long-term Growth
Well-Positioned for Sustained & Long-term Growth Commercial R&D Focus Strong Financial Position * Neurology ~$1.9B TARDIVE DYSKINESIA AND CHOREA Neuroendocrinology Cash and Investments ASSOCIATED WITH HUNTINGTON’S DISEASE 2024 Annual Neuropsychiatry as of 9/30/2024 $2.30 - $2.32 Net Sales Guidance R
Slide 4: 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 5: Recent Highlights and Future Key Milestones and Activities
Recent Highlights and Future Key Milestones and Activities Q3 2024 / Recent Highlights Future Key Milestones and Activities ▪ Kyle W. Gano, Ph.D. succeeds Kevin Gorman as CEO ▪ Company Intends to Enter Into a $300M Accelerated Share ▪ INGREZZA® (valbenazine) Net Product Sales of $613M Repurchase Tra
Slide 6: Building and Maximizing the Pipeline
Building and Maximizing the Pipeline Phase 1 # of Programs by Stage Phase 2 Phase 3 NDA 5 6 2 2 Phase 1 Phase 2 Phase 3 NDA Neurology valbenazine* Dyskinetic Cerebral Palsy VMAT2 Inhibitor NBI-9213522 SCN8A-DEE Nav1.6 NBI-1076986 Movement Disorders M4 Antagonist Neuroendocrinology crinecerfont3 CAH:
Slide 7: Q3 2024 Financial Summary
Q3 2024 Financial Summary $ Millions, Except Non-GAAP Earnings Per Share Item Q3 2024 Q3 2023 Highlights / Comments Revenue $622 $499 INGREZZA Sales of $613M Represents YoY Growth of 26% - Product Sales, Net $617 $492 Driven by Strong Underlying Patient Demand and Improvement in Gross-to-Net Dynamic
Slide 8: YTD 2024 Financial Summary
YTD 2024 Financial Summary $ Millions, Except Non-GAAP Earnings Per Share Item 2024 YTD* 2023 YTD* Highlights / Comments Revenue $1,728 $1,372 YTD INGREZZA Sales of ~$1.7B Represents YoY Growth of - Product Sales, Net $1,709 $1,353 27% vs. YTD 2023 - Collaboration Revenue $18 $19 Increase Driven by
Slide 9: Raised 2024 INGREZZA Net Sales Guidance and Revised Expense Guidance
Raised 2024 INGREZZA Net Sales Guidance and Revised Expense Guidance 2023 2024 Previous 2024 Current Item ($ Millions) Comments Actuals Guidance Range Guidance Range Raised Sales INGREZZA Net Product Sales1 $1,836 $2,250 - $2,300 $2,300 - $2,320 Guidance GAAP R&D Expense2 $565 $665 - $695 $700 - $72
Slide 10: Corporate Sustainability: "A" Rating Reaffirmed at MSCI
Corporate Sustainability: “A” Rating Reaffirmed at MSCI and Rank in 11th Percentile for Biotech at Sustainalytics Our Purpose: Relieve Suffering for People with Great Needs, but Few Options Adhere to the highest Invest in our people Minimize our impact product quality and and communities on the envi
Slide 11: Our Medicines, Our Patients
© NEYROCRINE Our Medicines, Our Patients
Slide 12: Multiple Commercial Products
Multiple Commercial Products In the U.S. In the U.S. and Europe † * ENDOMETRIOSIS ADRENAL INSUFFICIENCY In Europe TARDIVE DYSKINESIA † CHOREA ASSOCIATED WITH HD UTERINE FIBROIDS CONGENITAL ADRENAL HYPERPLASIA HD = Huntington’s Disease *Mitsubishi Tanabe Pharma Corporation (MTPC) has commercializatio
Slide 13: INGREZZA
INGREZZA (valbenazine) capsules NEUROCRINE BIOSCIENCES
Slide 14: INGREZZA Quarterly Net Sales Performance
INGREZZA Quarterly Net Sales Performance ($Million) Q3 2024 Sales of $613M Represents $650 26% Year-Over-Year Growth vs. Q3 2023 $613 $600 $580 $550 $500 $506 $500 $486 $450 $440 $410 $399 $400 $350 Q4 '22 Q1 '23 Q2 '23 Q3 '23 Q4 '23 Q1 '24 Q2 '24 Q3 '24 14
Slide 15: Substantial Impact on TD Patients and Care Partners
Substantial Impact on TD Patients and Care Partners Movement disorder caused by prolonged use of antipsychotics and anti-nausea medications Uncontrollable, abnormal and repetitive movements >50% of patients experience meaningful emotional, social and psychological impact* Job Performance Low Self-Wo
Slide 16: Nascent Tardive Dyskinesia Market Presents Significant Opportunity
Nascent Tardive Dyskinesia Market Presents Significant Opportunity Approximately 75% of TD E S T I M AT E D T O A F F E C T Patients Remain Undiagnosed ~800,000 ✓ Only half of diagnosed patients receive treatment with a VMAT2 inhibitor like INGREZZA people in the U.S. Source: U.S.Claims Data and 31
Slide 17: INGREZZA Approved by the FDA for Chorea Associated with Huntington's Disease
INGREZZA® Approved by the FDA for the Treatment of Chorea Associated with Huntington’s Disease INGREZZA Simple once-a-day treatment targeted for symptom control of chorea movements Safety profile consistent with and supported by extensive safety data in tardive dyskinesia In randomized, double-blind
Slide 18: Driving Long-Term Growth for INGREZZA
Driving Long-Term Growth for INGREZZA Launched INGREZZA® SPRINKLE (valbenazine) oral granules formulation IP Protection to 2038 Additional indication to treat chorea associated with Huntington’s disease Salesforce and distribution expansion (Including Psychiatry and LTC Salesforce Expansion in Q3 20
Slide 19: Neuropsychiatry Pipeline
(5) NEUROCRINE BIOSCIENCES
Slide 20: NBI-1065845 (AMPA Potentiator): Positive Phase 2 Results in MDD
NBI-1065845* (AMPA Potentiator): Reported Positive Phase 2 Top- Line Study Results in Adults with Major Depressive Disorder Inadequate Response to Treatment NBI-1065845 (or NBI-’845) in Major Depressive Disorder (MDD) Potent first-in-class AMPA potentiator ~1/3 of the 16 million+ people in the • Ora
Slide 21: NBI-1065845: SAVITRI Phase 2 Study Summary Results
NBI-1065845* (AMPA Potentiator): TM SAVITRI Phase 2 Study Summary Results EFFICACY SAFETY AND TOLERABILITY • The study met its primary and key secondary endpoints • NBI-1065845 was generally well-tolerated • Once-daily, oral administration of NBI-’845 produced a • Most common adverse event was heada
Slide 22: Muscarinic Platform Includes Multiple Clinical Programs
Muscarinic Platform Includes Multiple Clinical Programs From M1 to M4 Selective Orthosteric Agonists M1 M1/M4 M4 “Cognition” DUAL “Psychosis” PREFERRING PREFERRING ▪ Phase 1 ▪ Phase 1 ▪ Phase 1 NBI-1117570 SELECTIVE NBI-1117567 NBI-1117569 SELECTIVE ▪ Phase 2: Schizophrenia Preclinical NBI-1117568 C
Slide 23: Developing Novel Muscarinic System Portfolio
Developing Novel Muscarinic System Portfolio Neurocrine Biosciences Advancing Muscarinic Portfolio (Largest in Industry) ➢ Phase 2 placebo-controlled study of NBI-1117568*, a selective M4 agonist, as a potential treatment for schizophrenia with positive Phase 2 proof-of-concept results ✓ NBI-1117568
Slide 24: Summary of Phase 2 Topline Results
Summary of Phase 2 Topline Results Once-Daily 20mg Dose: Efficacy, Safety, and Tolerability Results Support Advancement to Phase 3 20mg Once-daily Demonstrated Efficacy, Safety and Tolerability Profile Statistically Significant and Clinically Generally Safe and Well-tolerated Combined With Once-dail
Slide 25: Once-Daily 20mg Dose Demonstrated Significant Efficacy at Weeks 3-6
Once-Daily 20mg Dose Demonstrated Clinically Meaningful and Statistically Significant Efficacy at Week 3, 4, 5, and 6 0 20mg QD Efficacy Data PANSS Total Score Change from Baseline LS Mean1 ± SEM -5.7 Week 4 – Week 6 -5 -8.2 -7.7 Week 4 5 6** -9.6 PANSS Total Score -8.4 -10.2 -10 -10.8 -11.5 LS Mean
Slide 26: Significant Improvement in Additional Endpoints
Once-Daily 20mg Dose Demonstrated Statistically Significant Improvement in Additional Endpoints \ CGI-S Marder Factor — Positive Marder Factor — Negative Placebo 20mg QD Placebo 20mg QD Placebo 20mg QD Week 6 N=68 N=35 N=68 N=35 N=68 N=35 LS Mean Change from Baseline* -0.5 -1.2 -2.8 -5.8 -1.2 -3.1 L
Slide 27: NBI-'568 Was Generally Safe and Well Tolerated at All Doses Studied
NBI-’568 Was Generally Safe and Well Tolerated at All Doses Studied Treatment-Emergent Adverse Events Occurring in ≥ 5% of NBI-'568 All Treated Group Placebo 20mg QD 40mg QD 60mg QD 30mg BID All Treated \ N=70 N=40 N=39 N=34 N=27 N=140 Somnolence 2 (2.9) 5 (12.5) 2 (5.1) 7 (20.6) 1 (3.7) 15 (10.7) D
Slide 28: NBI-'568 is the First and Only Muscarinic M4 Selective Orthosteric Agonist
NBI-’568 is the First and Only Muscarinic M4 Selective Orthosteric Agonist in Clinical Development Type of Large Opportunity For NBI-'568, A Novel And Subtype Requires Endogenous Muscarinic Differentiated Asset Selectivity Ligand (Acetylcholine) Activation With no reliance on innate acetylcholine le
Slide 29: NBI-'568 Effect Size Comparable to Known Muscarinic Programs
NBI-’568 Effect Size Comparable to Known Muscarinic Programs and Leading Antipsychotics Clinical-Stage Muscarinic Programs Leading Approved Treatments 0.75 0.68 0.61 0.61 0.60 Effect Size 0.56 0.55 0.42 0.41 0.36 0.34 0.3 0.26 125mg / 30mg 30mg QD 125mg / 30mg 20mg 125mg / 30mg Zyprexa Risperdal Ser
Slide 30: Validation of Selective Orthosteric Agonist Mechanism
Validation of Selective Orthosteric Agonist Mechanism Strengthens Conviction In Opportunities For Industry Leading Muscarinic Portfolio Primary Mechanism Phase Therapeutic Areas Potential Areas For Development (M1-M4) NBI-1117568 NBI-1117568 M4 agonist 2 Alzheimer’s Disease NBI-1117567 M1 agonist 1
Slide 31: Valbenazine: ATS Study Will Inform Next-Generation VMAT2 Inhibitors
Valbenazine*: ATS Study Will Inform Development of Our Next-Generation VMAT2 Inhibitors Including NBI-1065890 (Currently in Phase 1) Adjunctive Treatment of Schizophrenia (ATS) Schizophrenia is one of the leading causes of disability worldwide, affecting up to 3.5M people in the U.S. alone. A seriou
Slide 32: Neuroendocrinology Pipeline
Neuroendocrinology Pipeline
Slide 33: Classic Congenital Adrenal Hyperplasia (CAH)
Classic Congenital Adrenal Hyperplasia (CAH) Rare Genetic Disorder Enzyme deficiency & reduced cortisol levels and excess androgen levels U.S. ~30,000* Europe ~50,000* Complex and Treatment Options Highly Stagnant for 70 Years Variable • Hormone replacement Symptoms • Do not address underlying issue
Slide 34: Classic CAH 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 35: Classic CAH 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 36: Classic CAH 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 37: 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 38: CAHtalyst Study 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 39: CAHtalyst Adult and Pediatric Study Safety and Tolerability
CAHtalystTM Adult and Pediatric Study Safety and Tolerability • Crinecerfont Treatment was Overall 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 w
Slide 40: Neurocrine Next Steps Regarding Crinecerfont
Neurocrine Next Steps Regarding Crinecerfont • FDA Granted Breakthrough Therapy Designation and Priority Review For Crinecerfont for Adult and Pediatric Patients with CAH • Prescription Drug User Fee (PDUFA) Target Action Dates Set for December 29 for the Capsule Formulation and December 30 for the
Slide 41: Neurology Pipeline
© NEVROCRINE Neurology Pipeline
Slide 42: Valbenazine: Registrational Program in Dyskinetic Cerebral Palsy
Valbenazine*: Registrational Program in Dyskinetic Cerebral Palsy Dyskinetic Cerebral Palsy (DCP) A form of cerebral palsy (CP) that affects ~15% of the approximately 500K to 1M people in the U.S. diagnosed with the disease. Can result in a range of developmental delays, physical difficulties and in
Slide 43: Well-Positioned for Sustained & Long-term Growth
Well-Positioned for Sustained & Long-term Growth Commercial R&D Focus Strong Financial Position * Neurology ~$1.9B TARDIVE DYSKINESIA AND CHOREA Neuroendocrinology Cash and Investments ASSOCIATED WITH HUNTINGTON’S DISEASE 2024 Annual Neuropsychiatry as of 9/30/2024 $2.30 - $2.32 Net Sales Guidance R
Slide 44: GAAP to Non-GAAP Reconciliations
GAAP to Non-GAAP Reconciliations neurocrine.com
Slide 45: Condensed Consolidated Statements of Income
NEUROCRINE BIOSCIENCES NEUROCRINE BIOSCIENCES, INC. CONDENSED CONSOLIDATED STATEMENTS OF INCOME (in millions, except per share data) Revenues: Net product sales Collaboration revenue Total revenues Operating expenses: Cost of revenues Research and development Acquired in-process research and develop
Slide 46: Condensed Consolidated Balance Sheets
NEUROCRINE BIOSCIENCES, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited) September 30, December 31, (in millions) 2024 2023 Cash, cash equivalents, and marketable securities $ 1,228.0 $ 1,031.6 Other current assets 648.6 575.4 Total current assets 1,876.6 1,607.0 Deferred tax assets 454.4 362.
Slide 47: Reconciliation of GAAP to Non-GAAP Financial Results
NEUROCRINE BIOSCIENCES NEUROCRINE BIOSCIENCES, INC. RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS, (unaudited) ‘Three Months Ended Nine Months Ended September 30, September 30, (in millions, except per share data) 2024 2023 2024 2023 GAAP net income ! $ 129.8 $ 83.1 $ 238.2 $ 102.0 Adjustment
Slide 48: Reconciliation of GAAP to Non-GAAP Expenses
NEUROCRINE BIOSCIENCES NEUROCRINE BIOSCIENCES, INC. RECONCILIATION OF GAAP TO NON-GAAP EXPENSES (unaudited) Three Months Ended Nine Months Ended ‘September 30, ‘September 30, (in mittions) 24 2023 224 2023 GAAP cost of revenues $ 80 $ Ti U7 Ss 312 Adjustments: Non-cash amortization related to acquir
Slide 49: Advancing Life-Changing Discoveries in Neuroscience
Advancing Life-Changing Discoveries in Neuroscience Q3 2024 Corporate Presentation October 30, 2024 Nasdaq: NBIX
Neurocrine Biosciences — Q3 2024 Earnings Presentation on 6ix