corporate presentation - investors
TRANSCRIPT
Corporate Presentation
January 2022
Safe Harbor Statement
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This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements.
Such forward-looking statements include statements regarding, among other things, our plans to commercialize CAPLYTA for bipolar depression and schizophrenia; our estimates of the size of the prescriber base; whether clinical trial results will be predictive of future real-world results; whether CAPLYTA will serve an unmet need; our beliefs about the potential utility of our product candidates; and our development efforts and plans. All such forward-looking statements are based on management’s present expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, the following: there are no guarantees that CAPLYTA will be commercially successful; we may encounter issues, delays or other challenges in commercializing CAPLYTA; the COVID-19 pandemic may negatively impact our commercial plans and sales for CAPLYTA; the COVID-19 pandemic may negatively impact the conduct of, and the timing of enrollment, completion and reporting with respect to, our clinical trials; whether CAPLYTA receives adequate reimbursement from third-party payors; the degree to which CAPLYTA receives acceptance from patients and physicians for its approved indications; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; results achieved in CAPLYTA in the treatment of schizophrenia and bipolar depression following commercial launch of the product may be different than observed in clinical trials, and may vary among patients; any other impacts on our business as a result of or related to the COVID-19 pandemic; risks associated with our current and planned clinical trials; we may encounter unexpected safety or tolerability issues with CAPLYTA following commercial launch for the treatment of schizophrenia or bipolar depression or in ongoing or future trials and other development activities; our other product candidates may not be successful or may take longer and be more costly than anticipated; product candidates that appeared promising in earlier research and clinical trials may not demonstrate safety and/or efficacy in larger-scale or later clinical trials or in clinical trials for other indications; our proposals with respect to the regulatory path for our product candidates may not be acceptable to the U.S. Food and Drug Administration; our reliance on collaborative partners and other third parties for development of our product candidates; and the other risk factors detailed in our public filings with the Securities and Exchange Commission. All statements contained in this presentation are made only as of the date of this presentation, and the Company undertakes no duty to update this information unless required by law.
• Fully integrated biopharmaceutical company
• Founded in 2002 based on Nobel Prize-winning science
Intra-Cellular Therapies, Inc. (ITCI)
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Our Mission
Develop innovative treatments to improve the lives of individuals with neuropsychiatric and neurologic disorders in order to reduce the burden on patients and their caregivers
CAPLYTA® (lumateperone)
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Please see important safety information, including Boxed Warning on slide 18 and full Prescribing Information at https://www.intracellulartherapies.com/docs/caplyta_pi.pdf
New Indication
FDA-approved for the treatment of bipolar depression in adultsONLY FDA-approved treatment for depressive episodes
associated with bipolar I or bipolar II disorder in adults as
monotherapy and as adjunctive therapy with lithium or
valproate
Previously approved
Treatment of schizophrenia in adults
CAPLYTA® (lumateperone) is Approved for a Broad Range of Adult Patients with Bipolar Depression
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MonotherapyAdjunctive
(to lithium or valproate)
Bipolar I Bipolar II Bipolar I Bipolar II
CAPLYTA (lumateperone)
Quetiapine1
Olanzapine/Fluoxetine2
Lurasidone3
Cariprazine4
✓ ✓ ✓ ✓
✓ ✓
✓
✓ ✓
✓
CAPLYTA (lumateperone) is FDA-approved for the treatment of depressive episodes associated with bipolar I or bipolar II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults.1. Drugs@FDA: FDA-Approved Drugs. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/020639s069lbl.pdf. Accessed Dec 20, 2021. 2. Drugs@FDA: FDA-Approved Drugs. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/021520s053lbl.pdf. Accessed Dec 20, 2021. 3. Drugs@FDA: FDA-Approved Drugs. https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/200603s035lbl.pdf. Accessed Dec 20, 2021. 4. Drugs@FDA: FDA-Approved Drugs. https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/204370s006lbl.pdf. Accessed Dec 20, 2021.
Demonstrated Efficacy and Favorable Safety and Tolerability in Bipolar Depression in Adults
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Safety profileSimilar to placebo:• Extrapyramidal symptoms (EPS) changes including akathisia • Weight, fasting glucose, total cholesterol, and triglycerides• Mean changes in prolactin
Most common adverse reactions**Somnolence/sedation, dizziness, nausea, and dry mouth
Convenient dosingOnce daily, with or without foodStart and stay, with no titration required
CAPLYTA (lumateperone) is FDA-approved for the treatment of depressive episodes associated with bipolar I or bipolar II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults.
Please see full Prescribing Information including Boxed Warning at https://www.intracellulartherapies.com/docs/caplyta_pi.pdf
* In these studies, CAPLYTA 42 mg also showed statistically significant improvement in the key secondary endpoint relating to clinical global impression (CGI) of bipolar disorder
**≥5% and twice the rate of placebo
The efficacy of CAPLYTA 42 mg was demonstrated in two Phase 3 studies by meeting the primary endpoint of change from baseline in the MADRS total score at week 6*
Bipolar Depression is a Highly PrevalentUnderserved Condition
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Few approved treatments for bipolar I depression
Need for treatment options that are effective with favorable safety and tolerability profiles
11 million adults
4.4%
Depressive episodes Longer and recurring more often than manic/hypomanic episodes
Greater functional impairment, morbidity and mortality3
• Higher risk of suicide
1. National Institute of Mental Health. Bipolar Disorder. https://www.nimh.nih.gov/health/statistics/bipolar-disorder. Accessed Dec 9, 2021. 2. Merikangas KR et al., Arch Gen Psychiatry. 2011 March ; 68(3): 241–251. 3. Post RM. J Clin Psychiatry. 2005;66(Suppl 5):5-10. 4. American Psychiatric Association. (2013). Diagnostic and statistical manual of mental disorders (5th ed.). 5. Karanti A, et al. Bipolar disorders. 2020;22(4),392-400. 6. Swartz HA, et al. Bipolar II Disorder: Recognition, Understanding, and Treatment. DC: APA Publishing; 2019.
Bipolar Disorder lifetime prevalence in US adults1
Prior to CAPLYTA, only one approved treatment for bipolar II depression and only as monotherapy
Prevalence of Bipolar I and II are similar2
Bipolar II, a more complex condition4,5,6
Depressive episodes last longer and are more severe than Bipolar I
Higher risk of suicide than Bipolar I
~60% of patients with Bipolar II are initially diagnosed as unipolar (major depressive disorder (MDD))
Expanding Prescriber Base Following CAPLYTA Approval in Bipolar Depression
*Accounting for ~80% of branded schizophrenia and bipolar prescriptions 8
Salesforce expansion complete: ~320 Neuroscience sales specialists utilizing hybrid model
43,000+*
prescribers treating bipolar depression +schizophrenia• Psychiatrists• NP/PA• PCPs
23,000prescribers treatingschizophrenia• Primarily Psychiatrists• NP/PA
Strong Market Access Position
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of Medicare / Medicaid lives
of Commercial lives
Effective patient support program
>80%
>95%
Lumateperone Programs Extend Across Multiple Major Neuropsychiatric Conditions
Schizophrenia
Adjunctive treatment of MDD
2.4+ M patients1
~ 11 M patients2
19.4 M patients3
1. NIH. https://archives.nih.gov/asites/report/09-09-2019/report.nih.gov/nihfactsheets/ViewFactSheete76f.html?csid=67&key=S#S. Accessed Dec 9, 2021. 2. National Institute of Mental Health. Bipolar Disorder. https://www.nimh.nih.gov/health/statistics/bipolar-disorder. Accessed Dec 9, 2021. 3. National Institute of Mental Health, Major Depression. https://www.nimh.nih.gov/health/statistics/major-depression.shtml. Accessed Dec 9, 2021. 4. Perugi G, Angst J, Azorin JM, et al: Mixed features in patients with a major depressive episode: the BRIDGE-II-MIX study. J Clin Psychiatry2015; 76:e351–e358.5. McIntyre RS, Soczynska JK, Cha DS, et al. The prevalence and illness characteristics of DSM-5-defined "mixed feature specifier" in adults with major depressive disorder and bipolar disorder.
Depressive episodes associated with Bipolar I or II disorder as monotherapy and as
adjunctive treatment with lithium or valproate
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Patients w/ MDD or Bipolar Depression w/ Mixed Features
About 1/3 of patients with MDD and bipolar depression4, 5
P O T E N T I A L t o E X P A N D O P P O R T U N I T Y w i t h
O N G O I N G D E V E L O P M E N T
P R O G R A M S
CAPLYTA (lumateperone) is FDA-approved for the treatment of depressive episodes associated with bipolar I or bipolar II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults. The safety and efficacy has not been established for other uses.
A P P R O V E D I N D I C A T I O N S
Adjunctive Treatment of MDD
• Two Phase 3 adjunctive treatment of MDD studies ongoing– Study 501 and Study 502 are global 6-week, randomized, double-blind, placebo-controlled, multicenter,
clinical trials in adult patients with MDD who are having inadequate response to antidepressant monotherapy (ADT)
– Inadequate response to ADT is defined as less than 50% improvement
• Study 503 is an open label roll-over study to assess safety for 6 months
ADT + Lumateperone 42 mg
ADT + Placebo
ADT + Lumateperone 42 mg
ADT + Placebo
Study 501 (n ~470)
Study 502 (n ~470)
1 2 3 4 5 6
Assessment Weeks
Screening PeriodUp to 2 weeks
Primary EndpointChange in MADRS
total score at Week 6
Key Secondary Endpoint
Change in CGI-S
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Studying Patients with Bipolar Depression or MDD who Exhibit Mixed Features
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Study 403: Studying lumateperone 42 mg as monotherapy in patients with Bipolar depression or MDD with mixed features
Mixed features
• ~ 1/3 of patients with MDD and bipolar depression exhibit mixed features1,2
• Patients exhibit manic symptoms below the clinical threshold for mania/hypomania during depressive episode
• Poor response to antidepressants, greater symptom severity, a higher risk of suicide attempts, and severe illness with more comorbidities
• In MDD patients, it is a significant risk factor for the development of Bipolar I or Bipolar II disorder
CAPLYTA (lumateperone) is FDA-approved for the treatment of depressive episodes associated with bipolar I or bipolar II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults. The safety and efficacy have not been established for other uses.
1. Perugi G, Angst J, Azorin JM, et al: Mixed features in patients with a major depressive episode: the BRIDGE-II-MIX study. J Clin Psychiatry2015; 76:e351–e358.2. McIntyre RS, Soczynska JK, Cha DS, et al. J Affect Disord. 2015; 172:259–264.
Screening PeriodUp to 2 weeks
Lumateperone 42 mg
Placebo
Primary EndpointChange in MADRS
total score at Week 6
Key Secondary Endpoint
Change in CGI-S
(n ~450)
Lumateperone Long-Acting Injectable (LLAI)
• A Phase 1 single ascending dose study evaluates the pharmacokinetics (PK), safety and tolerability of LLAI in patients with stable symptoms of schizophrenia
• Initial conduct has been completed and results from this study will inform the dosing strategy for the multiple ascending dose study and for the efficacy study
• LLAI is designed to maintain therapeutic levels of lumateperone for at least 1 month
• Program includes the development of other formulations
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Oral Lumateperone is FDA-approved for the treatment of depressive episodes associated with bipolar I or bipolar II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate and for the treatment of schizophrenia in adults. The safety and efficacy have not been established as a long-acting injectable.
MDD- Adjunctive Therapy
LAI Program
Robust Pipeline with Multiple Platforms
PDE 1Inhibitors
PRECLINICAL PHASE 1 PHASE 2 PHASE 3
Lenrispodun (ITI-214) – Parkinson’s Disease
ITI-214 and other PDE1 inhibitors -Other indications incl. cancer
PLATFORM
ITI-333Pain & Mood Disorders
Opioid Use Disorder
Lenrispodun (ITI-214) – Heart Failure
Lumateperoneand follow-on compounds
The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established.
Bipolar depression and MDD with mixed features
Agitation in pts with probable Alzheimer’s disease
Dementia-related psychosis
Depressive disorders in the elderly
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ITI-1284 ODT-SL
A Robust Pipeline Beyond Lumateperone
ITI-1284-ODT-SL
15The safety and efficacy of investigational agents have not been established.
ITI-1284 ODT-SL is a deuterated form of lumateperone, a new molecular entity formulated as an oral disintegrating tablet for sublingual administration
Programs focused on the treatment of agitation in patients with probable Alzheimer’s disease, dementia-related psychosis and certain depressive disorders in the elderly
PDE 1 Inhibitors
PDE1’s unique properties provide broad therapeutic opportunities
Our portfolio of PDE1 inhibitors are being developed to treat diseases in which PDE1 activity is increased and/or deleterious immune cell changes lead to poor outcomes including certain cancers
Lenrispodun, our lead PDE1 inhibitor, is in Phase 2 development for Parkinson’s disease
We are studying the anti-tumor effects of our PDE1 inhibitors that act (in preclinical models) to enhance the host immune systems ability to fight cancer
ITI-333
Our 5-HT2A antagonist and μ-opioid receptor partial agonist provides potential utility in the treatment of opioid use disorder (overdose prevention and reversal interventions) and acute & chronic pain
Phase 1 single ascending dose study evaluating safety, tolerability, and pharmacokinetics has been completed
Achieved plasma exposures at or above those required for efficacy
In this study, ITI-333 was generally safe and well tolerated
Key Financial Metrics
KEY METRICS
Total Cash, Cash Equivalents, and Investmentsand restricted cash1 $413 million
Total Debt2 $0.0 million
Common Shares Outstanding2 81,377,406
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1. As of December 31, 2021 (unaudited). This amount is preliminary, unaudited and may change, was prepared by management and is based on the most current information available to management, and is subject to completion by management of the financial statements as of and for the year ended December 31, 2021, or the 2021 financial statements, including completion of the review procedures, final adjustments and other developments that may arise between now and the time the financial results for this period are finalized, and completion of the audit of the 2021 financial statements
2. As of September 30, 2021 (unaudited)
Management Team
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Sharon Mates, PhD Founder, Chairman, President & Chief Executive Officer
Mark Neumann Executive Vice President & Chief Commercial Officer
Michael I. Halstead, Esq. Executive Vice President & General Counsel
John A. Bardi Senior Vice President, Market Access, Policy and Government Affairs
Robert Davis, PhD Senior Vice President & Chief Scientific Officer
Suresh Durgam, MD Senior Vice President & Chief Medical Officer
Larry Hineline Senior Vice President of Finance & Chief Financial Officer
Michael Olchaskey, PharmD Senior Vice President, Head of Regulatory Affairs
Karen Sheehy, Esq. Senior Vice President & Chief Compliance Officer
Juan Sanchez, MD Vice President, Corporate Communications and Investor Relations
CAPLYTA® (lumateperone) Warnings and Precautions Are Those Common to the Antipsychotic Class
18Please see full Prescribing Information including Boxed Warning at https://www.intracellulartherapies.com/docs/caplyta_pi.pdf
Important Safety Information
Boxed Warnings:
Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. CAPLYTA is not approved for the treatment of patients with dementia-related psychosis.
Antidepressants increased the risk of suicidal thoughts and behaviors in pediatric and young adults in short-term studies. All anti-depressant-treated patients should be closely monitored for clinical worsening, and for emergence of suicidal thoughts and behaviors. The safety and effectiveness of CAPLYTA have not been established in pediatric patients.
Contraindications: CAPLYTA is contraindicated in patients with known hypersensitivity to lumateperone or any components of CAPLYTA. Reactions have included pruritus, rash (e.g. allergic dermatitis, papular rash, and generalized rash), and urticaria.
Warnings & Precautions: Antipsychotic drugs have been reported to cause:Cerebrovascular Adverse Reactions in Elderly Patients with Dementia-Related Psychosis, including stroke and transient ischemic attack. See Boxed Warning above.Neuroleptic Malignant Syndrome (NMS), which is a potentially fatal reaction. Signs and symptoms include: high fever, stiff muscles, confusion, changes in breathing, heart rate, and blood pressure, elevated creatinine phosphokinase, myoglobinuria (and/or rhabdomyolysis), and acute renal failure. Patients who experience signs and symptoms of NMS should immediately contact their doctor or go to the emergency room.Tardive Dyskinesia, a syndrome of uncontrolled body movements in the face, tongue, or other body parts, which may increase with duration of treatment and total cumulative dose. TD may not go away, even if CAPLYTA is discontinued. It can also occur after CAPLYTA is discontinued.Metabolic Changes, including hyperglycemia, diabetes mellitus, dyslipidemia, and weight gain. Hyperglycemia, in some cases extreme and associated with ketoacidosis, hyperosmolar coma or death, has been reported in patients treated with antipsychotics. Measure weight and assess fasting plasma glucose and lipids when initiating CAPLYTA and monitor periodically during long-term treatment.Leukopenia, Neutropenia, and Agranulocytosis (including fatal cases). Complete blood counts should be performed in patients with pre-existing low white blood cell count (WBC) or history of leukopenia or neutropenia. CAPLYTA should be discontinued if clinically significant decline in WBC occurs in absence of other causative factors.Decreased Blood Pressure & Dizziness. Patients may feel lightheaded, dizzy or faint when they rise too quickly from a sitting or lying position (orthostatic hypotension). Heart rate and blood pressure should be monitored, and patients should be warned with known cardiovascular or cerebrovascular disease. Orthostatic vital signs should be monitored in patients who are vulnerable to hypotension.Falls. CAPLYTA may cause sleepiness or dizziness and can slow thinking and motor skills, which may lead to falls and, consequently, fractures and other injuries. Patients should be assessed for risk when using CAPLYTA.Seizures. CAPLYTA should be used cautiously in patients with a history of seizures or with conditions that lower seizure threshold.Potential for Cognitive and Motor Impairment. Patients should use caution when operating machinery or motor vehicles until they know how CAPLYTA affects them.Body Temperature Dysregulation. CAPLYTA should be used with caution in patients who may experience conditions that may increase core body temperature such as strenuous exercise, extreme heat, dehydration, or concomitant anticholinergics.Dysphagia. CAPLYTA should be used with caution in patients at risk for aspiration.
Drug Interactions: CAPLYTA should not be used with CYP3A4 inducers and moderate or strong CYP3A4 inhibitors.
Special Populations: Newborn infants exposed to antipsychotic drugs during the third trimester of pregnancy are at risk for extrapyramidal and/or withdrawal symptoms following delivery. Breastfeeding is not recommended. Use of CAPLYTA should be avoided in patients with moderate or severe liver problems.
Adverse Reactions: The most common adverse reactions in clinical trials w/CAPLYTA vs. placebo were somnolence/sedation, dizziness, nausea and dry mouth.
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Thank you