ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
☒ | Accelerated filer | ☐ | ||||
Non-accelerated filer |
☐ | Smaller reporting company | ||||
Emerging growth company |
Item 1. |
BUSINESS |
• | we seek to have the capability to develop first-in-class |
• | we seek to develop drugs that either can differentiate themselves in competitive markets by addressing aspects of CNS diseases and other diseases which are not adequately treated by currently marketed drugs or can be effective with fewer side effects. |
• | continue to commercialize CAPLYTA, which has been approved by the FDA for the treatment of schizophrenia in adults, in the United States; |
• | commercialize CAPLYTA, which has been approved by the FDA for the treatment of bipolar depression in adults, in the United States; |
• | complete the development of lumateperone for additional neuropsychiatric indications, such as MDD; |
• | expand the commercial potential of lumateperone by investigating its usefulness in additional neurological areas, such as autism spectrum disorder, and in additional neuropsychiatric indications, such as sleep disorders associated with neuropsychiatric and neurological disorders; |
• | continue to advance our other product candidates in clinical development such as lenrispodun, for the treatment of CNS and other disorders; ITI-1284, for the treatment of neuropsychiatric disorders and behavioral disturbances in dementia; and ITI-333, for substance use disorders, pain and psychiatric comorbidities including depression and anxiety; and |
• | advance the earlier stage product candidates in our pipeline. |
Summary Description of Patent or Patent Application |
United States or Foreign Jurisdiction |
Expiration Date | ||
ITI-007 Product Patent (approved drug product—lumateperone tosylate—in any pharmaceutical form) |
Granted: |
March 12, 2028 (US: does not include expected 6-month extension in US for pediatric studies) |
Summary Description of Patent or Patent Application |
United States or Foreign Jurisdiction |
Expiration Date | ||
ITI-007 Crystal Form Patent (approved drug product—lumateperone tosylate—in solid crystalline form) |
Granted: |
December 1, 2029 (US; does not include expected 6-month extension for pediatric studies; additional patent term extension possible through 2033**);March 12, 2029 (ex-US) | ||
Pending in IL, IN | ||||
ITI-007 Dosage and Method of Treatment Patents (including schizophrenia, bipolar depression, sleep disorder indications) |
Granted: Pending: US (continuation), EP, IN, KR (divisional), MX (divisional) |
December 28, 2029 (US; does not include expected 6-month extension for pediatric studies; additional patent term extension possible through 2033**);May 27, 2029 (ex-US) | ||
ITI-007 Residual Symptoms Patent (treatment of negative/residual symptoms of schizophrenia) |
Granted: Pending: US (continuation), AU (divisional), JP (divisional), EP (divisional), IN, KR (divisional), MX (divisional), CA, BR IL, CN |
December 3, 2034 (US and ex-US; does not include expected US 6-month extension for pediatric studies;) | ||
Patents for Additional Dosage Forms |
Granted: Pending: US (continuation), AU, CA, CN, EP, IN, IL, JP, KR, MX, RU |
2037-2039 | ||
Patents for Additional Indications (including post-traumatic stress disorder, impulse control disorder, symptoms associated with dementia, acute depression, and acute anxiety) |
Granted: Granted |
2033-2034 |
* | Orange-Book listed U.S. patents (NB: U.S. 8,598,119 and U.S. 9,586,960 have been requested for delisting as they have been superseded by RE48,839 and RE48,825, respectively) |
** | We have filed patent term extension applications on two U.S. patents. The U.S. Patent and Trademark Office, or USPTO, has not completed its review of these applications. In the United States, we are permitted to extend the term of one U.S. patent for lumateperone or the use thereof. Accordingly, on completion of the USPTO’s review of our patent term extension applications, we must select one of the two patents to which any patent term extension granted will attach. Patent terms may be subject to change not only due to potential patent term extensions but also to any terminal disclaimer that reduces patent term, as well as other factors. Because the U.S. patent laws and related judicial interpretations change, modifications or new interpretations of the laws may impact our patent terms. |
• | identifying and validating targets; |
• | screening compounds against targets; |
• | preclinical studies and clinical trials of potential pharmaceutical products; and |
• | obtaining FDA and other regulatory clearances. |
• | capital resources; |
• | research and development resources; |
• | manufacturing capabilities; and |
• | sales and marketing. |
• | completion of extensive preclinical laboratory tests, animal studies, and formulation studies in accordance with the FDA’s Good Laboratory Practice, or GLP, regulations; |
• | submission to the FDA of an Investigational New Drug application, or IND, for human clinical testing, which must become effective before human clinical trials may begin; |
• | performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the drug for each proposed indication; |
• | submission to the FDA of an NDA after completion of all clinical trials; |
• | satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the API and finished drug product are produced and tested to assess compliance with current Good Manufacturing Practices, or cGMPs; |
• | satisfactory completion of FDA inspections of clinical trial sites to assure that data supporting the safety and effectiveness of product candidates has been generated in compliance with Good Clinical Practices; and |
• | FDA review and approval of the NDA prior to any commercial marketing or sale of the drug in the United States. |
• | Phase 1 usually involves the initial introduction of the investigational drug into a limited population, typically healthy humans, to evaluate its short-term safety, dosage tolerance, metabolism, pharmacokinetics and pharmacologic actions, and, if possible, to gain an early indication of its effectiveness. |
• | Phase 2 usually involves trials in a limited patient population to (i) evaluate dosage tolerance and appropriate dosage; (ii) identify possible adverse effects and safety risks; and (iii) evaluate preliminarily the efficacy of the drug for specific targeted indications. Multiple Phase 2 clinical trials may be conducted by the sponsor to obtain information prior to beginning larger and more expensive Phase 3 clinical trials. |
• | Phase 3 trials, commonly referred to as pivotal studies, are undertaken in an expanded patient population at multiple, geographically dispersed clinical trial centers to further evaluate clinical efficacy and test further for safety by using the drug in its final form. |
• | The federal Anti-Kickback Law, which prohibits, among other things, knowingly or willingly offering, paying, soliciting or receiving remuneration, directly or indirectly, in cash or in kind, to induce or reward the purchasing, leasing, ordering or arranging for or recommending the purchase, lease or order of any health care items or service for which payment may be made, in whole or in part, by federal health care programs such as Medicare and Medicaid; |
• | The federal civil False Claims Act, which prohibits, among other things, individuals or entities from knowingly presenting, or causing to be presented, a false or fraudulent claim for payment of government funds or knowingly making, using or causing to be made or used, a false record or statement material to an obligation to pay money to the government or knowingly concealing or knowingly and improperly avoiding, decreasing or concealing an obligation to pay money to the federal government; |
• | The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal liability for knowingly and willfully executing a scheme to defraud any healthcare benefit program, knowingly and willfully embezzling or stealing from a health care benefit program, willfully obstructing a criminal investigation of a health care offense, or knowingly and willfully making false statements relating to healthcare matters; |
• | The federal Physician Payment Sunshine Act, being implemented as the Open Payments Program, requires certain pharmaceutical manufacturers to engage in extensive tracking of payments and other transfers of value to physicians and teaching hospitals, and to submit such data to the Centers for Medicare and Medicaid Studies, or CMS, which will then make all of this data publicly available on the CMS website; and |
• | Analogous state laws and regulations, including state anti-kickback and false claims laws, which may apply to items or services reimbursed under Medicaid and other state programs or, in several states, |
apply regardless of the payer, as well as other state laws that require pharmaceutical companies to report expenses related to the marketing and promotion of pharmaceutical products, prohibit certain gifts or payments to health care providers in the state, and/or require pharmaceutical companies to implement compliance programs or marketing codes of conduct. |
Item 1A. |
RISK FACTORS |
• | In order to execute our business plan and achieve profitability, we need to effectively commercialize CAPLYTA, which received FDA approval in December 2019 for the treatment of schizophrenia in adults and in December 2021 for the treatment of bipolar depression in adults. |
• | If we do not obtain regulatory approval of lumateperone for other indications in the United States, or for any indication in foreign jurisdictions, we will not be able to market lumateperone for other indications or in other jurisdictions, which will limit our commercial revenues. |
• | If our newly established sales and marketing capabilities or our third-party relationships for the commercialization of lumateperone are not effective, lumateperone may not be successfully commercialized. |
• | We have generated limited revenue from product sales and there is no guarantee that our revenue from the sale of CAPLYTA or other product candidates, if approved, will be substantial. |
• | There is no guarantee that our planned clinical trials for lumateperone will be successful. |
• | We expect our net losses to continue for at least a few years and are unable to predict the extent of future losses or when we will become profitable, if ever. |
• | We may require additional funding, which may not be available to us on acceptable terms, or at all, and, if not so available, may require us to delay, limit, reduce or cease our operations. |
• | Even though the FDA has granted approval of CAPLYTA for the treatment of schizophrenia and bipolar depression, the terms of the approval may limit its commercial potential. Additionally, CAPLYTA is still subject to ongoing regulatory requirements. |
• | Delays, suspensions and terminations in our clinical trials could result in increased costs to us, delay our ability to generate product revenues and therefore may have a material adverse effect on our business, results of operations and future growth prospects. |
• | Safety issues with our product candidates or approved product, or with product candidates or approved products of third parties that are similar to our product candidates, could give rise to delays in the regulatory approval process, restrictions on labeling or product withdrawal after approval. |
• | Preliminary and interim data from our clinical studies that we may announce or publish from time to time may change as more patient data become available. |
• | We rely on third parties to conduct our clinical trials and perform data collection and analysis, which may result in costs and delays that prevent us from successfully commercializing our product candidates. |
• | Even if we successfully complete the clinical trials of one or more of our product candidates, the product candidates may fail for other reasons. |
• | We are subject to ongoing regulatory obligations and restrictions with regard to lumateperone and, following regulatory approval of any of our product candidates, we will be subject to ongoing regulatory obligations and restrictions with regard to such product candidates, which may result in significant expense and limit our ability to commercialize lumateperone and our other potential products. |
• | CAPLYTA and our product candidates, if approved, may not gain acceptance among physicians, patients, or the medical community, thereby limiting our potential to generate revenues, which will undermine our future growth prospects. |
• | CAPLYTA has only recently been, and our other product candidates have never been, manufactured on a commercial scale, and there are risks associated with scaling up manufacturing to commercial scale. In particular, we will need to develop larger scale manufacturing processes that are more efficient and cost-effective to commercialize our product candidates which may not be successful. |
• | We rely on third-party manufacturers to manufacture and supply lumateperone and our other product candidates for us. If one of our suppliers or manufacturers fails to perform adequately or fulfill our needs, we may be required to incur significant costs and devote significant efforts to find new suppliers or manufacturers. We may also face significant delays in our clinical trials, regulatory approvals and product introductions and commercialization. |
• | We will need to continue to manage our organization and we may encounter difficulties with our staffing and any future transitions, which could adversely affect our results of operations. |
• | Our ability to compete may be undermined if we do not adequately protect our proprietary rights. |
• | Our ability to generate product revenues will be diminished if lumateperone or any of our other potential products does not receive coverage from payors or sell for inadequate prices, or if patients are unable to obtain adequate levels of reimbursement. |
• | Many of our competitors have greater resources and capital than us, putting us at a competitive disadvantage. If our competitors develop and market products that are more effective than lumateperone or our other product candidates, they may reduce or eliminate our commercial opportunity. |
• | The COVID-19 pandemic could continue to have a material impact on our business, financial condition and results of operations, including our commercial operations and sales, clinical trials and preclinical studies. |
• | Numerous factors could result in substantial volatility in the trading price of our stock. |
• | The price of our common stock could be subject to volatility related or unrelated to our operations. |
• | Our management has broad discretion over the use of our cash and we may not use our cash effectively, which could adversely affect our results of operations. |
• | the efficacy, cost, approved use, and side-effect profile of CAPLYTA regimens relative to competitive treatment regimens for the treatment of schizophrenia and bipolar depression; |
• | the effectiveness of our commercial strategy for the marketing of CAPLYTA, including our pricing strategy and the effectiveness of our efforts to obtain adequate third-party reimbursements; |
• | maintaining and successfully monitoring commercial manufacturing arrangements for CAPLYTA with third-party manufacturers to ensure they meet our standards and those of regulatory authorities, including the FDA, which extensively regulate and monitor pharmaceutical manufacturing facilities; |
• | our ability to meet the demand for commercial supplies of CAPLYTA; |
• | the acceptance of CAPLYTA by patients, the medical community and third-party payors; and |
• | the effect of recent or potential health care legislation in the United States. |
• | the amount of product sales from lumateperone; |
• | the costs of maintaining and expanding our sales and marketing capabilities for lumateperone; |
• | the costs of preparing applications for regulatory approvals for lumateperone in additional indications other than in schizophrenia and bipolar depression, and potentially in jurisdictions other than the United States, and for other product candidates, as well as the costs required to support review of such applications; |
• | the costs of manufacturing and distributing lumateperone for commercial use in the United States; |
• | our ability to obtain regulatory approval for, and subsequently generate product sales from, lumateperone in additional indications other than in schizophrenia and bipolar depression or in jurisdictions other than the United States; |
• | the progress in, and the costs of, our preclinical studies and clinical trials and other research and development programs; |
• | the scope, prioritization and number of our research and development programs; |
• | the ability of any future collaborators and us to reach the milestones, and other events or developments, triggering payments under any future collaboration agreements or to otherwise make payments under such agreements; |
• | our ability to enter into new, and to maintain any existing, collaboration and license agreements; |
• | the extent to which any future collaborators are obligated to reimburse us for clinical trial costs under any future collaboration agreements; |
• | the costs involved in filing, prosecuting, enforcing and defending patent claims and other intellectual property rights; |
• | the costs of maintaining or securing manufacturing and supply arrangements for clinical or commercial production of lumateperone or our other product candidates; |
• | the costs of preparing applications for regulatory approvals for our product candidates; |
• | the costs of preparing for and establishing, or contracting for, sales and marketing capabilities if we obtain regulatory approvals for our product candidates; |
• | the costs involved in expanding the accounting and data management systems to support commercial operations; and |
• | the costs associated with litigation, including the costs incurred in defending against any product liability claims that may be brought against us related to lumateperone or our other product candidates. |
• | withdrawal of approval, addition of warnings or narrowing of the approved indication in the product label; |
• | requirement of a Risk Evaluation and Mitigation Strategy to mitigate the risk of off-label use in populations where the FDA may believe that the potential risks of use may outweigh its benefits; |
• | voluntary or mandatory recalls; |
• | warning letters; |
• | suspension of any ongoing clinical studies; |
• | refusal by the FDA or other regulatory authorities to approve pending applications or supplements to approved applications filed by us, or suspension or revocation of product approvals; |
• | restrictions on operations, including restrictions on the marketing or manufacturing of the product or the imposition of costly new manufacturing requirements; or |
• | seizure or detention, or refusal to permit the import or export of products. |
• | we may be required to conduct additional clinical trials or implement a Risk Evaluation and Mitigation Strategies program prior to or following approval; |
• | regulatory authorities may not approve our product candidates or, as a condition of approval, may require specific warnings and contraindications; |
• | regulatory authorities may withdraw their approval of the product and require us to take our drug off the market; |
• | we may have limitations on how we promote our drugs; |
• | sales of products may decrease significantly; |
• | we may be subject to litigation or product liability claims; and |
• | our reputation may suffer. |
• | we may not be able to control the amount and timing of resources that our collaborators may devote to the drug candidates; |
• | our collaborators may experience financial difficulties; |
• | we may be required to relinquish important rights, such as marketing and distribution rights; |
• | business combinations or significant changes in a collaborator’s business strategy may also adversely affect a collaborator’s willingness or ability to complete its obligations under any arrangement; |
• | a collaborator could independently move forward with a competing drug candidate developed either independently or in collaboration with others, including our competitors; and |
• | collaborative arrangements are often terminated or allowed to expire, which would delay the development and may increase the cost of developing our drug candidates. |
• | fail to receive the regulatory approvals required to market them as drugs; |
• | be subject to proprietary rights held by others requiring the negotiation of a license agreement prior to marketing; |
• | be difficult or expensive to manufacture on a commercial scale; |
• | have adverse side effects that make their use less desirable; or |
• | fail to compete with product candidates or other treatments commercialized by our competitors. |
• | our ability to provide acceptable evidence of safety and efficacy; |
• | the scope of the approved indication(s) for the product; |
• | the inclusion of any warnings or contraindications in the product label; |
• | pricing and cost effectiveness, which may be subject to regulatory control; |
• | our ability to obtain sufficient third-party insurance coverage or reimbursement; |
• | effectiveness of our or our collaborators’ sales and marketing strategy; |
• | relative convenience and ease of administration; |
• | patient adherence to treatment; |
• | prevalence and severity of any adverse side effects; and |
• | availability of alternative treatments. |
• | imposition of an annual, nondeductible fee on any entity that manufactures or imports certain branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government health care programs; |
• | an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program, retroactive to January 1, 2010, to 23% and 13% of the average manufacturer price for most branded and generic drugs, respectively; |
• | expansion of health care fraud and abuse laws, including the False Claims Act and the Anti-Kickback Statute, new government investigative powers, and enhanced penalties for noncompliance; |
• | a Medicare Part D coverage gap discount program, in which manufacturers agreed to offer 50% point-of-sale |
• | extension of manufacturers’ Medicaid rebate liability to covered drugs dispensed to individuals who are enrolled in Medicaid managed care organizations; |
• | expansion of eligibility criteria for Medicaid programs; |
• | expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program; |
• | requirements to report certain financial arrangements with physicians and teaching hospitals, including reporting any “payments or transfers of value” made or distributed to prescribers, teaching hospitals and other health care providers and reporting any ownership and investment interests held by physicians and their immediate family members and applicable group purchasing organizations during the preceding calendar year; |
• | a requirement to annually report drug samples that manufacturers and distributors provide to physicians; and |
• | a Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research. |
• | we may not have been the first to make the inventions covered by our pending patent applications or issued patents; |
• | we may not have been the first to file patent applications for our products, product candidates or the technologies we rely upon; |
• | others may independently develop similar or alternative technologies or duplicate any of our technologies; |
• | our disclosures in patent applications may not be sufficient to meet the statutory requirements for patentability; |
• | any or all of our pending patent applications may not result in issued patents; |
• | we may not seek or obtain patent protection in all countries that will eventually provide a significant business opportunity; |
• | any patents issued to us or our collaborators may not provide a basis for commercially viable products, may not provide us with any competitive advantages or may be challenged by third parties; |
• | our proprietary technologies may not be patentable; |
• | others may design around our patent claims to produce competitive products which fall outside of the scope of our patents; |
• | others may identify prior art which could invalidate our patents; and |
• | changes to patent laws may limit the exclusivity rights of patent holders. |
• | identifying and validating targets; |
• | screening compounds against targets; |
• | preclinical studies and clinical trials of potential pharmaceutical products; |
• | obtaining FDA and other regulatory approvals; and |
• | commercializing pharmaceutical products. |
• | decreased demand for our products or product candidates that we may develop; |
• | injury to our reputation; |
• | withdrawal of clinical trial participants; |
• | initiation of investigations by regulators; |
• | costs to defend the related litigation; |
• | a diversion of management’s time and our resources; |
• | substantial monetary awards to trial participants or patients; |
• | product recalls, withdrawals or labeling, marketing or promotional restrictions; |
• | loss of revenue; |
• | exhaustion of any available insurance and our capital resources; |
• | the inability to commercialize our products or product candidates; and |
• | a decline in our stock price. |
• | the success of our commercialization of CAPLYTA in the United States for the treatment of schizophrenia and bipolar depression; |
• | timing and announcement of regulatory developments, submissions and approvals or preliminary, interim or final results of clinical trials; |
• | actual or anticipated quarterly variation in our results of operations or the results of our competitors; |
• | announcements of medical innovations or new products or product candidates by our competitors; |
• | issuance of new or changed securities analysts’ reports or recommendations for our stock; |
• | developments or disputes concerning our intellectual property or other proprietary rights; |
• | commencement of, or our involvement in, litigation; |
• | market conditions in the biopharmaceutical industry; |
• | any future sales of our common stock or other securities in connection with raising additional capital or otherwise; |
• | any major change to the composition of our board of directors or management; and |
• | general economic conditions and slow or negative growth of our markets. |
• | the accuracy of our estimates regarding expenses, revenues, uses of cash, cash equivalents and investment securities, capital requirements and the need for additional financing; |
• | our expectations regarding our commercialization of CAPLYTA, including the impact of COVID-19 on the commercialization of CAPLYTA and our ability to adapt our approach as appropriate; |
• | the duration and severity of the COVID-19 pandemic and its impact on our business; |
• | the supply and availability of and demand for our product; |
• | the initiation, cost, timing, progress and results of our development activities, non-clinical studies and clinical trials; |
• | the timing of and our ability to obtain and maintain regulatory approval, or submit an application for regulatory approval, of lumateperone and our other existing product candidates, any product candidates that we may develop, and any related restrictions, limitations, and/or warnings in the label of any approved product candidates; |
• | our plans to research, develop and commercialize lumateperone and our other current and future product candidates; |
• | the election by any collaborator to pursue research, development and commercialization activities; |
• | our ability to obtain future reimbursement and/or milestone payments from our collaborators; |
• | our ability to attract collaborators with development, regulatory and commercialization expertise; |
• | our ability to obtain and maintain intellectual property protection for our product candidates; |
• | our ability to successfully commercialize lumateperone and our other product candidates; |
• | the size and growth of the markets for lumateperone and our other product candidates and our ability to serve those markets; |
• | the rate and degree of market acceptance of any current or future products; |
• | the success of competing drugs that are or become available; |
• | regulatory developments in the United States and other countries; |
• | the performance of our third-party suppliers and manufacturers and our ability to obtain alternative sources of raw materials; |
• | our ability to obtain additional financing; |
• | our use of the proceeds from our securities offerings; |
• | any restrictions on our ability to use our net operating loss carryforwards; |
• | our exposure to investment risk, interest rate risk and capital market risk; and |
• | our ability to attract and retain key scientific, management or sales and marketing personnel. |
Item 1B. |
UNRESOLVED STAFF COMMENTS |
Item 2. |
PROPERTIES |
Item 3. |
LEGAL PROCEEDINGS |
Item 4. |
MINE SAFETY DISCLOSURES |
Item 5. |
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES |
Item 6. |
[RESERVED] |
Item 7. |
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
• | direct costs of formulating, manufacturing and packaging drug product; |
• | overhead costs consisting of labor, share-based compensation, shipping, outside inventory management and other miscellaneous operating costs; and |
• | royalty payments on product sales. |
• | internal recurring costs, such as costs relating to labor and fringe benefits, materials, supplies, facilities and maintenance; and |
• | fees paid to external parties who provide us with contract services, such as pre-clinical testing, manufacturing and related testing, clinical trial activities and license milestone payments. |
• | salaries and related benefit costs of a dedicated sales force; |
• | sales operation costs; and |
• | marketing and promotion expenses. |
• | salaries and related benefit costs; |
• | patent, legal, and professional costs; and |
• | office and facilities overhead. |
For the Year Ended December 31, |
||||||||||||
2021 |
2020 | 2019 | ||||||||||
Revenues, net |
$ |
83,803 |
$ | 22,813 | $ | 61 | ||||||
Expenses |
||||||||||||
Cost of product sales |
8,035 |
1,895 | — | |||||||||
Research and development |
88,845 |
65,782 | 89,125 | |||||||||
Selling, general and administrative |
272,611 |
186,364 | 64,948 | |||||||||
|
|
|
|
|
|
|||||||
Total costs & expenses |
369,491 |
254,041 | 154,073 | |||||||||
|
|
|
|
|
|
|||||||
Loss from operations |
(285,688 |
) |
(231,228 | ) | (154,012 | ) | ||||||
Interest income, net |
1,568 |
4,235 | 6,292 | |||||||||
Income tax expense |
(6 |
) |
(13 | ) | (2 | ) | ||||||
|
|
|
|
|
|
|||||||
Net loss |
$ |
(284,126 |
) |
$ | (227,006 | ) | $ | (147,722 | ) | |||
|
|
|
|
|
|
2021 |
2020 | |||||||
External costs |
$ |
53,166 | $ | 38,791 | ||||
Internal costs |
35,679 | 26,991 | ||||||
|
|
|
|
|||||
Total Research and development expenses |
$ |
88,845 | $ | 65,782 | ||||
Lumateperone costs |
$ |
48,633 | $ | 32,884 | ||||
Manufacturing costs of drug candidates |
4,960 | 5,585 | ||||||
Share- based compensation |
11,456 | 8,415 | ||||||
Other projects and overhead |
23,796 | 18,898 | ||||||
|
|
|
|
|||||
Total Research and development expenses |
$ |
88,845 | $ | 65,782 | ||||
|
|
|
|
• | completion of extensive pre-clinical laboratory tests, animal studies, and formulation studies in accordance with the FDA’s Good Laboratory Practice, or GLP, regulations; |
• | submission to the FDA of an Investigational New Drug application, or IND, for human clinical testing, which must become effective before human clinical trials may begin; |
• | performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the drug for each proposed indication; |
• | submission to the FDA of a New Drug Application, or NDA, after completion of all clinical trials; |
• | satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the active pharmaceutical ingredient, or API, and finished drug product are produced and tested to assess compliance with current Good Manufacturing Practices, or cGMPs; |
• | satisfactory completion of FDA inspections of clinical trial sites to assure that data supporting the safety and effectiveness of product candidates has been generated in compliance with Good Clinical Practices; and |
• | FDA review and approval of the NDA prior to any commercial marketing or sale of the drug in the United States. |
Item 7A. |
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK |
Item 8. |
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA |
Index to Financial Statements and Financial Statement Schedules |
Number | |||
F-1 | ||||
F-3 |
||||
F-4 |
||||
F-5 |
||||
F-6 |
||||
F-7 |
||||
F-8 |
Item 9. |
CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE |
Item 9A. |
CONTROLS AND PROCEDURES |
• | pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the Company; |
• | provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the Company are being made only in accordance with authorizations of management and directors of the Company; and |
• | provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of the Company’s assets that could have a material effect on the financial statements. |
Item 9B. |
OTHER INFORMATION |
Item 9C. |
DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS |
Item 10. |
DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE |
Item 11. |
EXECUTIVE COMPENSATION |
Item 12. |
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS |
Item 13. |
CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE |
Item 14. |
PRINCIPAL ACCOUNTANT FEES AND SERVICES |
Item 15. |
EXHIBITS AND FINANCIAL STATEMENT SCHEDULES | |
Item 15(a). |
The following documents are filed as part of this annual report on Form 10-K: | |
Item 15(a)(1) and (2) |
See “Index to Financial Statements and Financial Statement Schedules” at Item 8 to this Annual Report on Form 10-K. Other financial statement schedules have not been included because they are not applicable or the information is included in the financial statements or notes thereto. | |
Item 15(a)(3) |
Exhibits |
Exhibit Number |
Exhibit Description |
Filed Herewith |
Incorporated by Reference herein from Form or Schedule |
Filing Date |
SEC File/ Reg. Number | |||||||
10.23 | Form of Director Restricted Stock Unit Agreement under the 2018 Equity Incentive Plan.* | 8-K |