Intra-Cellular Therapies(ITCI) - 2020 Q4 - Annual Report

PART I Business Intra-Cellular Therapies is a biopharmaceutical company focused on CNS disorders, commercializing CAPLYTA and advancing a pipeline Overview and Product The company is a biopharmaceutical firm focused on CNS disorders, with CAPLYTA approved for schizophrenia and commercially launched - The company is a biopharmaceutical firm focused on small molecule drugs for neuropsychiatric and neurological disorders[13] - CAPLYTA (lumateperone) was approved by the FDA in December 2019 for the treatment of schizophrenia in adults (42mg/day)[13] - The commercial launch of CAPLYTA began in late March 2020, supported by a national sales force of approximately 240 representatives[14] - In clinical trials, CAPLYTA demonstrated a favorable safety and tolerability profile, with minimal effects on weight gain and metabolic parameters compared to placebo[15] Development Programs The company is advancing lumateperone for bipolar depression and other indications, alongside pipeline candidates for various neurological disorders - Lumateperone is in Phase 3 development for bipolar depression, with sNDAs submitted in February 2021 and a target action date in the second half of 2021[21][24] - A long-acting injectable (LAI) formulation of lumateperone is in Phase 1 development, with topline results expected in the second half of 2021[27] - ITI-1284, a deuterated lumateperone, is being developed as an oral disintegrating tablet for behavioral disturbances in dementia and depressive disorders in the elderly[29] - The PDE1 inhibitor program, led by ITI-214, is being developed for Parkinson's disease and heart failure, with a Phase 2 trial for Parkinson's planned for 2021[30] - ITI-333, a novel compound for substance use disorders and pain, entered a Phase 1 study in December 2020, with partial NIH HEAL Initiative funding expected[32] Disease and Market Overview This section outlines the target patient populations and market sizes for key indications, including schizophrenia, bipolar disorder, and dementia Target Market Size and Opportunity | Indication | U.S. Patient Population / Market Size (2020) | | :--- | :--- | | Schizophrenia | 2.4 million Americans / >$12 billion U.S. antipsychotic market | | Bipolar Disorder | >11 million adult Americans / ~$6 billion global market | | Dementia (Alzheimer's) | 5.8 million Americans with AD | | Parkinson's Disease | 1 million Americans / ~$3.5 billion global market | | Major Depressive Disorder | ~7% of U.S. adults annually / ~$6 billion global market (2019) | | Heart Failure | 6.5 million U.S. adults / ~$30.7 billion annual U.S. cost | | Opioid Use Disorder | >10 million Americans reporting misuse | Strategy, Intellectual Property, and Operations The company's strategy focuses on commercializing CAPLYTA, expanding indications, advancing pipeline, protecting IP, and relying on third-party manufacturing - The company's strategy focuses on commercializing CAPLYTA, expanding lumateperone into new indications, and advancing its pipeline candidates[55] Lumateperone (CAPLYTA) U.S. Patent Expiration | Patent Type | U.S. Expiration Date | | :--- | :--- | | Product Patent | March 2028 | | Crystal Form Patent | December 2029 (extension possible to 2033) | | Dosage and Method of Treatment | December 2029 (extension possible to 2033) | | Residual Symptoms | December 2034 | | Additional Dosage Forms | 2037-2039 | - The company relies on third-party contract manufacturers like Siegfried and Lonza for the commercial and clinical supply of lumateperone's active pharmaceutical ingredient (API)[68][69][72] - CAPLYTA competes with branded drugs like Latuda®, Rexulti®, and VRAYLAR®, and numerous generic antipsychotics[78] Risk Factors The company faces substantial risks related to CAPLYTA commercialization, clinical trials, funding, third-party manufacturing, competition, and the COVID-19 pandemic - The company's prospects are highly dependent on the successful commercialization of CAPLYTA for schizophrenia, its only approved product[121][125] - The company expects to incur net losses for several more years and will require substantial additional funding[121][143][144] - The COVID-19 pandemic could materially impact business operations, including commercial sales of CAPLYTA, clinical trials, and preclinical studies[123][218] - The company relies on third-party manufacturers like Siegfried and Lonza; any supplier failure could cause significant delays in clinical trials and commercialization[123][184] - The company has a limited number of authorized shares of common stock available for issuance, potentially impairing its ability to raise capital or conduct strategic transactions[258] Unresolved Staff Comments The company reports no unresolved staff comments from the SEC - None Properties The company's headquarters are in New York, NY, with office and lab space leased until March 2029 - The company's headquarters are located in New York, NY, occupying approximately 32,287 square feet of office and lab space with a lease expiring in March 2029[278] Legal Proceedings The company is not currently a party to any material legal proceedings - The company is not currently a party to any material legal proceedings[279] Mine Safety Disclosures This section is not applicable to the company - Not applicable PART II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's common stock trades on Nasdaq under 'ITCI', with 80,917,013 shares outstanding as of February 22, 2021 - Common stock trades on the Nasdaq Global Select Market under the symbol 'ITCI'[283] - As of February 22, 2021, there were 80,917,013 shares of common stock outstanding[283] Selected Financial Data This section is not applicable - Not applicable Management's Discussion and Analysis of Financial Condition and Results of Operations In 2020, the company launched CAPLYTA, generating $22.5 million in net sales, incurring a $227.0 million net loss, and ending with $658.8 million in cash and investments Results of Operations This section details the company's financial performance, highlighting the impact of CAPLYTA's commercial launch on revenues, expenses, and net loss Consolidated Statements of Operations (in thousands) | | For the Year Ended December 31, | | :--- | :--- | :--- | :--- | | | 2020 | 2019 | 2018 | | Revenues, net | $22,813 | $61 | $— | | Expenses | | | | | Cost of product sales | 1,895 | — | — | | Research and development | 65,782 | 89,125 | 132,167 | | Selling, general and administrative | 186,364 | 64,948 | 30,099 | | Total costs & expenses | 254,041 | 154,073 | 162,266 | | Loss from operations | (231,228) | (154,012) | (162,266) | | Interest income, net | (4,235) | (6,292) | (7,141) | | Net loss | $(227,006) | $(147,722) | $(155,127) | - Net product sales for 2020 were approximately $22.5 million from CAPLYTA, which launched in late March 2020, with no product sales in 2019[321] - Cost of product sales was low at $1.9 million (8.4% of net sales), as most product sold was manufactured before FDA approval and previously expensed to R&D[316][322] - Research and development (R&D) expenses decreased by 26% to $65.8 million in 2020, primarily due to a $15.1 million decrease in manufacturing expenses capitalized as inventory post-approval[325] - Selling, general and administrative (SG&A) expenses increased by 187% to $186.4 million in 2020, driven by CAPLYTA commercialization, including $55.2 million in sales-related labor and $41.5 million in commercialization costs[332][333] Liquidity and Capital Resources This section discusses the company's cash position, capital raising activities, and cash flow from operations, and its ability to fund future operations - As of December 31, 2020, the company had approximately $658.8 million in cash, cash equivalents, and available-for-sale investment securities[343] - In 2020, the company raised significant capital through two public offerings, with net proceeds of approximately $277.0 million in January and $357.8 million in September[340][342] - Net cash used in operations for 2020 was $230.3 million, primarily for selling and marketing costs for the CAPLYTA launch, clinical trials, and manufacturing[343][347] - The company expects existing cash to fund operations and capital expenditures for at least the next 12 months from the filing date[344] Quantitative and Qualitative Disclosures About Market Risk The company's primary market risk is interest rate sensitivity on its $658.8 million investment portfolio, alongside capital market risk affecting future fundraising ability - The company's primary market risk is interest rate sensitivity on its $658.8 million portfolio of cash, cash equivalents, and marketable securities, where declines would reduce future investment income[365] - The company also faces capital market risk, as its ability to raise additional funds through equity offerings depends on its stock price and market conditions[367] Financial Statements and Supplementary Data This section contains the index to the company's audited consolidated financial statements and schedules for the fiscal year ended December 31, 2020 - This item includes the Report of Independent Registered Public Accounting Firm and the company's consolidated financial statements for the years ended December 31, 2020, 2019, and 2018[369] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure This section is not applicable - Not applicable Controls and Procedures Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2020, with an unqualified audit report - The principal executive and financial officers concluded that disclosure controls and procedures were effective as of the end of the period covered by the report[371] - Management assessed the company's internal control over financial reporting as effective as of December 31, 2020, based on the COSO framework[374] - The independent registered public accounting firm issued an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2020[379] Other Information This section is not applicable - Not applicable PART III Directors, Executive Officers and Corporate Governance Information for this item is incorporated by reference from the company's Proxy Statement for the 2021 Annual Meeting of Stockholders - Information regarding directors, executive officers, and corporate governance is incorporated by reference from the Registrant's Proxy Statement for the 2021 Annual Meeting of Stockholders[389] Executive Compensation Information for this item is incorporated by reference from the company's Proxy Statement for the 2021 Annual Meeting of Stockholders - Information regarding executive compensation is incorporated by reference from the Registrant's Proxy Statement for the 2021 Annual Meeting of Stockholders[390] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information for this item is incorporated by reference from the company's Proxy Statement for the 2021 Annual Meeting of Stockholders - Information regarding security ownership is incorporated by reference from the Registrant's Proxy Statement for the 2021 Annual Meeting of Stockholders[391] Certain Relationships and Related Transactions, and Director Independence Information for this item is incorporated by reference from the company's Proxy Statement for the 2021 Annual Meeting of Stockholders - Information regarding certain relationships, related transactions, and director independence is incorporated by reference from the Registrant's Proxy Statement for the 2021 Annual Meeting of Stockholders[392] Principal Accountant Fees and Services Information for this item is incorporated by reference from the company's Proxy Statement for the 2021 Annual Meeting of Stockholders - Information regarding principal accountant fees and services is incorporated by reference from the Registrant's Proxy Statement for the 2021 Annual Meeting of Stockholders[393] PART IV Exhibits and Financial Statement Schedules This section lists the exhibits filed as part of the Annual Report on Form 10-K, including corporate governance documents, material contracts, and certifications - This item contains a list of all exhibits filed with the Form 10-K, including material contracts, corporate documents, and certifications by the CEO and CFO[395][396] Form 10-K Summary This section is not applicable - Not Applicable