PART I. FINANCIAL INFORMATION [Financial Statements](index=3&type=section&id=Item%201.%20Financial%20Statements) Unaudited statements show a net loss, a significant deficit, and substantial doubt about the company's going concern status Condensed Balance Sheet Data (in thousands) | Metric | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $7,421 | $12,068 | | Total Assets | $8,338 | $13,290 | | Total Liabilities | $31,608 | $32,773 | | Accumulated Deficit | $(289,481) | $(262,486) | | Total Stockholders' (Deficit) Equity | $(23,270) | $(19,483) | Condensed Statements of Operations (in thousands, except per share data) | Metric | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Research and development | $29,771 | $26,723 | | General and administrative | $4,573 | $4,372 | | Loss from operations | $(34,278) | $(31,025) | | Net Loss | $(26,995) | $(30,990) | | Basic and diluted net loss per share | $(1.97) | $(3.59) | - The company has incurred substantial operating losses since inception and has an accumulated deficit of **$289.5 million** as of June 30, 2023[187](index=187&type=chunk) - Management has concluded there is **substantial doubt about the company's ability to continue as a going concern**, as cash is only expected to fund operations through Q3 2023[196](index=196&type=chunk) - In February 2023, the company expensed a **$2.2 million** non-refundable milestone payment for the FDA's acceptance of the Biologics License Application (BLA) for cosibelimab[244](index=244&type=chunk) - The company closed registered direct offerings in February, April, and May 2023, raising total net proceeds of approximately **$21.3 million**[188](index=188&type=chunk)[189](index=189&type=chunk)[190](index=190&type=chunk) - Subsequent to the quarter end, the company closed another registered direct offering with gross proceeds of approximately **$10.0 million** in July 2023[191](index=191&type=chunk)[310](index=310&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the cosibelimab BLA submission, a significant deficit, increased R&D expenses, and the need for future financing - The company submitted a BLA to the FDA for cosibelimab, which is under review with a PDUFA goal date of **January 3, 2024**[312](index=312&type=chunk) Comparison of Operating Expenses (in millions) | Expense Category | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Research & Development | $13.9 | $12.1 | $29.8 | $26.7 | | General & Administrative | $2.3 | $2.1 | $4.6 | $4.4 | - The increase in R&D expenses was primarily due to **$15.7 million** in commercial manufacturing costs for cosibelimab and a **$3.2 million** PDUFA fee[333](index=333&type=chunk) - The company's cash is only sufficient to fund operations through **Q3 2023**, raising substantial doubt about its ability to continue as a going concern[359](index=359&type=chunk) - For the six months ended June 30, 2023, net cash used in operating activities was **$26.0 million**, while financing activities provided **$21.4 million**[344](index=344&type=chunk)[361](index=361&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, the company is not required to provide disclosures about market risk - As a smaller reporting company, the company is not required to provide information under this item[362](index=362&type=chunk) [Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective, with no material changes to internal controls - The CEO and CFO concluded that the company's disclosure controls and procedures are **effective** as of June 30, 2023[365](index=365&type=chunk) - There were **no material changes** in internal control over financial reporting during the fiscal quarter ended June 30, 2023[366](index=366&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any litigation expected to have a material adverse effect - The company is not involved in any litigation that it believes could have a **material adverse effect** on its financial position or results of operations[368](index=368&type=chunk) [Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant financial, strategic, operational, and regulatory risks impacting its future viability [Risks Related to Our Finances and Capital Requirements](index=30&type=section&id=Risks%20Related%20to%20Our%20Finances%20and%20Capital%20Requirements) The company has a history of losses, faces a going concern risk, and requires substantial additional capital - The company has an accumulated deficit of **$289.5 million** as of June 30, 2023, and expects to incur continued losses[370](index=370&type=chunk)[388](index=388&type=chunk) - There is **substantial doubt about the company's ability to continue as a going concern**, with cash sufficient only through Q3 2023[404](index=404&type=chunk)[397](index=397&type=chunk) - Raising additional capital is necessary but may cause **significant dilution** to existing stockholders or require relinquishing valuable rights[406](index=406&type=chunk)[407](index=407&type=chunk) [Risks Related to our Business Strategy, Structure, and Organization](index=34&type=section&id=Risks%20Related%20to%20our%20Business%20Strategy%2C%20Structure%2C%20and%20Organization) Future success depends entirely on commercializing product candidates and acquiring new assets, as the company has no products for sale - The company's future is entirely dependent on its ability to develop, obtain approval for, and commercialize its product candidates[420](index=420&type=chunk)[421](index=421&type=chunk) - The BLA for cosibelimab relies on foreign clinical data, and the FDA may require additional U.S. studies, causing delays and expense[432](index=432&type=chunk) - Future growth depends on acquiring or in-licensing new products, which presents numerous financial and integration risks[427](index=427&type=chunk)[434](index=434&type=chunk) [Risks Inherent in Drug Development and Commercialization](index=35&type=section&id=Risks%20Inherent%20in%20Drug%20Development%20and%20Commercialization) Drug development is high-risk, with unpredictable clinical trials, potential regulatory delays, and intense competition - The outcome of early clinical trials is **not necessarily predictive of future results**, and candidates may fail in later stages[435](index=435&type=chunk)[436](index=436&type=chunk) - The cosibelimab BLA may be impacted by the FDA's potential inability to audit clinical sites in **Russia and Ukraine**, which could delay approval[450](index=450&type=chunk) - The company faces **significant competition** from firms with substantially greater capital and commercialization resources[375](index=375&type=chunk)[376](index=376&type=chunk) - Even if approved, products may not gain market acceptance, and securing **adequate reimbursement** is uncertain but critical for success[55](index=55&type=chunk)[380](index=380&type=chunk)[382](index=382&type=chunk) [Risks Related to Reliance on Third Parties](index=44&type=section&id=Risks%20Related%20to%20Reliance%20on%20Third%20Parties) The company is heavily dependent on third-party manufacturers and CROs, which reduces direct control and introduces significant risk - The company relies on third-party contract manufacturers, increasing the risk of **supply shortages and quality issues**[35](index=35&type=chunk)[352](index=352&type=chunk)[353](index=353&type=chunk) - Reliance on third-party CROs is critical, and their failure to perform could compromise clinical data and **delay regulatory approval**[32](index=32&type=chunk)[33](index=33&type=chunk)[349](index=349&type=chunk) - The company's strategy relies on clinical data from third parties, which could prove to be **inaccurate or unreliable**[41](index=41&type=chunk)[42](index=42&type=chunk) [Risks Relating to Legislation and Regulation Affecting the Biopharmaceutical and Other Industries](index=47&type=section&id=Risks%20Relating%20to%20Legislation%20and%20Regulation%20Affecting%20the%20Biopharmaceutical%20and%20Other%20Industries) The company operates in a highly regulated environment subject to risks from legislative changes and complex healthcare laws - The company is subject to new legislation that may **increase compliance costs** and adversely affect its ability to market products[2](index=2&type=chunk) - Uncertainty from the Affordable Care Act (ACA) and potential action against high drug prices by the Biden administration could **negatively impact profitability**[3](index=3&type=chunk)[4](index=4&type=chunk)[8](index=8&type=chunk) - Proposed rules on price transparency and drug importation could **adversely impact the industry** by influencing drug pricing[1](index=1&type=chunk) - Relationships with customers are subject to healthcare fraud and abuse laws, where violations can lead to **criminal sanctions and civil penalties**[10](index=10&type=chunk)[11](index=11&type=chunk) [Risks Related to Intellectual Property and Potential Disputes with Licensors Thereof](index=51&type=section&id=Risks%20Related%20to%20Intellectual%20Property%20and%20Potential%20Disputes%20with%20Licensors%20Thereof) Commercial success depends on obtaining and defending intellectual property, which is costly, uncertain, and reliant on licensors - The inability to maintain sufficient patent protection could **impair commercialization** by allowing competitors to develop similar products[15](index=15&type=chunk)[16](index=16&type=chunk) - The patent process is **expensive, time-consuming, and uncertain**, and patents may be challenged through legal proceedings[18](index=18&type=chunk)[19](index=19&type=chunk)[21](index=21&type=chunk) - The company depends on its licensors to maintain and enforce IP for its product candidates and has **limited control** over their strategies[27](index=27&type=chunk)[28](index=28&type=chunk)[29](index=29&type=chunk) - The company may become involved in **expensive lawsuits** to protect its patents or defend against infringement claims[52](index=52&type=chunk)[53](index=53&type=chunk)[71](index=71&type=chunk) [Risks Relating to Our Platform and Data](index=55&type=section&id=Risks%20Relating%20to%20Our%20Platform%20and%20Data) Operations are vulnerable to IT system failures and cyber-attacks, which could compromise confidential data and cause reputational harm - The business is dependent on IT systems and vulnerable to **cyber-attacks**, which could compromise confidential information[81](index=81&type=chunk)[82](index=82&type=chunk)[83](index=83&type=chunk) - A security breach could lead to the loss of proprietary information, **harm the company's reputation**, and result in liability[86](index=86&type=chunk) - The risk of cyber-attacks has generally increased, and the company may be unable to anticipate or prevent all security threats[84](index=84&type=chunk)[85](index=85&type=chunk) [Risks Relating to Our Control by Fortress Biotech Inc.](index=57&type=section&id=Risks%20Relating%20to%20Our%20Control%20by%20Fortress%20Biotech%20Inc.) Fortress Biotech holds a voting majority, creating potential conflicts of interest and stockholder dilution through its equity grants - Fortress holds a **voting majority** and can control all matters requiring stockholder approval, creating potential conflicts of interest[90](index=90&type=chunk)[96](index=96&type=chunk) - Under the Founders Agreement, Fortress receives an annual **2.5% equity grant**, which dilutes other stockholders' holdings[91](index=91&type=chunk)[97](index=97&type=chunk) - Agreements with Fortress might not reflect terms that would have resulted from **arm's-length negotiations** with unaffiliated parties[92](index=92&type=chunk)[98](index=98&type=chunk) [Risks Related to Conflicts of Interest](index=58&type=section&id=Risks%20Related%20to%20Conflicts%20of%20Interest) Significant conflicts of interest exist due to overlapping leadership with parent company Fortress and collaborator TGTX - The Chairman of the Board is also the CEO of TGTX, with whom the company has collaboration agreements, creating **potential conflicts of interest**[93](index=93&type=chunk)[100](index=100&type=chunk)[101](index=101&type=chunk) - Shared directors with Fortress could create conflicts, especially as Fortress is not required to notify the company of corporate opportunities[102](index=102&type=chunk)[103](index=103&type=chunk) [General Risks](index=58&type=section&id=General%20Risks) The company is exposed to general business risks including public health crises, employee misconduct, and stock price volatility - Public health issues like the **COVID-19 pandemic** could adversely impact business by delaying clinical trials and disrupting supply chains[104](index=104&type=chunk)[105](index=105&type=chunk)[107](index=107&type=chunk) - The company is exposed to risks of **employee misconduct**, including noncompliance with FDA regulations and fraud laws[109](index=109&type=chunk)[110](index=110&type=chunk) - The company's common stock price has been **highly volatile** and is likely to remain so, increasing the risk of securities litigation[117](index=117&type=chunk)[118](index=118&type=chunk) [Recent Sales of Unregistered Securities](index=61&type=section&id=Item%202.%20Recent%20Sales%20of%20Unregistered%20Securities.) Information regarding recent sales of unregistered securities was previously disclosed in other SEC filings - Information regarding recent sales of unregistered securities has not been furnished in this report, as it was previously included in other filings[122](index=122&type=chunk) [Defaults Upon Senior Securities](index=61&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) The company reports no defaults upon its senior securities during the period - None[124](index=124&type=chunk) [Mine Safety Disclosures](index=61&type=section&id=Item%204.%20Mine%20Safety%20Disclosures.) This item is not applicable to the company's operations - Not applicable[126](index=126&type=chunk) [Other Information](index=61&type=section&id=Item%205.%20Other%20Information.) The company reports no other information for disclosure under this item - None[128](index=128&type=chunk) [Exhibits](index=62&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the report, including corporate amendments and required officer certifications - The report includes several exhibits, such as amendments to the company's certificate of incorporation and officer certifications under Sarbanes-Oxley[130](index=130&type=chunk) Signatures [Signatures](index=63&type=section&id=Signatures_summary) The report was duly signed and authorized by the President and CEO on August 14, 2023 - The report was signed on **August 14, 2023**, by James F. Oliviero, President and Chief Executive Officer[132](index=132&type=chunk)[158](index=158&type=chunk)

Licensed from the Dana-Farber Institute, Cosibelimab is a fully-human anti-PD-L1 mAb with a dual mechanism of action Safe Harbor Statement This presentation may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. For such forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act ...

The collaborations with TGTX each contain single material performance obligations under Topic 606, which is the granting of a license that is functional intellectual property. The Company's performance obligation was satisfied at the point in time when TGTX had the ability to use and benefit from the right to use the intellectual property. The performance obligations of the original agreements were satisfied prior to the adoption of Topic 606. The performance obligation of the amendment to the collaboration ...

The issuance of a patent does not foreclose challenges to its inventorship, scope, validity or enforceability. Therefore, our owned and licensed patents may be challenged in the courts or patent offices in the United States and abroad. Such challenges may result in loss of exclusivity or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number 001-38128 CHECKPOINT THERAPEUTICS, INC. (Exact name of registrant as specified in its char ...

Therapeutics NASDAQ: CKPT Corporate Presentation September 2022 Checkpoint Therapeutics Safe Harbor Statement This presentation may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. For such forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Such statements incl ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number 001-38128 CHECKPOINT THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) ...

Financial Performance - For the three months ended March 31, 2022, revenue was approximately $52,000, a decrease of approximately $16,000 compared to $68,000 for the same period in 2021[141]. - As of March 31, 2022, the accumulated deficit was $216.7 million, with expectations of continued significant operating losses for the foreseeable future[149]. - Net cash used in operating activities was approximately $19.6 million for the three months ended March 31, 2022, compared to approximately $4.7 million for the same period in 2021[152]. - Net cash provided by financing activities was $6.3 million for the three months ended March 31, 2022, down from $23.9 million for the same period in 2021[154]. - The company currently believes its cash and cash equivalents are sufficient to fund anticipated operating cash requirements for at least one year from the date of the report[176]. - The company expects its financial condition and operating results to fluctuate significantly from quarter to quarter and year to year due to various uncontrollable factors[174]. Research and Development - Research and development expenses for the three months ended March 31, 2022, were approximately $14.7 million, an increase of $10.5 million from $4.2 million in the same period in 2021[143]. - The company anticipates a decrease in research and development expenses for the remainder of 2022[144]. - The company anticipates substantial increases in expenses if any product candidates are submitted for marketing approval or if additional clinical trials are initiated[167]. - The ongoing Phase 1 clinical trial of cosibelimab aims to support U.S. BLAs and comparable applications, with discussions held with the FDA regarding the use of exclusively foreign clinical data[199]. - The company has invested a significant portion of its resources in the acquisition and development of product candidates, which are currently in preclinical development or clinical trials[196]. Product Development and Approval - The company has not received approval for the sale of any product candidate and has not generated any product sales[135]. - The company has not generated any revenue from the sale of its development stage products to date and is uncertain when, or if, it will generate any revenue[168]. - The company is not permitted to market any product candidates until receiving regulatory approval, which may never occur[202]. - The success of product candidates in clinical trials is uncertain, with a high rate of failure in later stages of development[208]. - The company has not received marketing approval for any of its product candidates from regulatory authorities in any jurisdiction[226]. - The process of obtaining marketing approval is expensive and may take many years, with substantial variability based on product complexity and regulatory changes[227]. - Delays in obtaining approval or failure to obtain approval for product candidates could materially impair the company's ability to generate revenue[229]. - Regulatory authorities may approve product candidates for fewer or more limited indications than requested, impacting commercialization prospects[230]. - The FDA's accelerated approval regulations may allow for marketing approval based on surrogate endpoints, but meeting these criteria is not guaranteed[228]. - The company may need to abandon or limit development of product candidates if serious adverse effects are identified during clinical trials[233]. Market and Competitive Landscape - Market acceptance of product candidates is crucial for revenue generation, with factors such as efficacy, safety, and reimbursement playing significant roles[258]. - The company anticipates facing increasing competition from generic versions of branded products, particularly as patents expire, which could impact market share and pricing strategies[256]. - Competitors may develop treatments for target indications more quickly, reducing the commercial opportunity for the company's product candidates[251]. - The biotechnology and pharmaceutical industries are subject to rapid technological change, posing a risk of obsolescence for the company's product candidates[252]. Regulatory and Compliance Risks - Regulatory compliance is critical, as failures could lead to significant penalties, product recalls, and damage to reputation[277]. - The company must obtain FDA approval for any proposed product brand names, and delays in this process could adversely impact business[250]. - Legislative changes, such as the ACA, could materially affect the company's business, including increased manufacturer rebates and compliance costs[304]. - Compliance with healthcare laws and regulations may involve substantial costs and expose the company to significant penalties if found in violation[324]. Operational Risks - The company has limited resources to identify and execute acquisitions or in-licensing of third-party products, competing with larger pharmaceutical companies[205]. - Patient enrollment challenges in clinical trials could lead to significant delays and increased development costs[221]. - The company relies on third-party contract manufacturers for production, which introduces risks related to compliance and timely delivery of products[273]. - The reliance on third parties for clinical trials and manufacturing increases the risk of delays and impacts on commercialization efforts[285]. - There is a limited number of suppliers for raw materials, and disruptions in the supply chain could significantly delay clinical trials and regulatory approvals[288]. Funding and Financial Stability - The company will require substantial additional funding to complete the development and commercialization of its product candidates, which may not be available on acceptable terms[175]. - Limited product liability insurance coverage may expose the company to substantial liabilities from product claims, affecting financial stability[272]. - The company faces ongoing litigation regarding the constitutionality of the ACA, which may significantly impact its operations[309]. Intellectual Property - The company’s commercial success depends on obtaining and maintaining patent protection, which is costly and time-consuming[326]. - The patent position is uncertain, with potential litigation and varying international laws affecting patent rights[329]. - Changes in patent laws or interpretations may diminish the value of patents or narrow their scope, impacting competitive positioning[329].

Company Classification and Financial Status - The company is classified as an "emerging growth company" and may remain so until it achieves total annual gross revenue of at least $1.07 billion or a market value exceeding $700 million [155]. - The company has opted out of the extended transition period for new or revised financial accounting standards, which may complicate financial comparisons with other public companies [158]. - The company is a smaller reporting company and will remain so until its voting and non-voting common shares held by non-affiliates exceed $250 million or annual revenues exceed $100 million [159]. Product Development and Clinical Trials - The company currently has no drug products for sale and is entirely dependent on the future success of its product candidates [163]. - The company has invested significantly in the acquisition and development of product candidates, which are currently in preclinical development or clinical trials [164]. - The company intends to use data from ongoing clinical trials to support U.S. marketing approval applications for its product candidates [169]. - The company faces substantial risks in drug development, including the high rate of failure in clinical trials and the unpredictability of regulatory approval [179]. - The company may disclose top-line or preliminary data from clinical trials, which are subject to change and should be viewed with caution [181]. - Delays in clinical trials may increase costs and hinder revenue generation for the company [185]. - Patient enrollment challenges could lead to significant delays in clinical trials, increasing development costs and limiting financing options [192]. - Regulatory authorities may impose suspensions or terminations of clinical trials due to various factors, including safety issues and funding shortages [186]. - The ongoing military conflict in Europe may disrupt clinical trial activities in affected regions, potentially delaying product development [194]. - The FDA's accelerated approval process may not be met, causing further delays in obtaining marketing approval for product candidates [199]. - Regulatory scrutiny remains high, and any adverse side effects identified during trials could lead to abandonment or limitation of product development [203]. Regulatory and Compliance Risks - The company faces potential restrictions on product marketing and distribution due to regulatory compliance issues, which could lead to fines or product recalls [215]. - Regulatory approval by the FDA is limited to specific indications where clinical safety and efficacy have been demonstrated, impacting the company's ability to market its products [217]. - Regulatory authorities may require costly post-marketing studies or impose restrictions that could affect the commercialization of approved products [212]. - Public concern regarding drug safety could delay regulatory approvals and increase scrutiny of clinical trial data [209]. - Compliance with healthcare laws and regulations is critical, as violations could lead to significant penalties and operational restructuring [298]. - The company is subject to various legislative and regulatory changes that could adversely affect its ability to market products and raise capital [277]. Manufacturing and Supply Chain Challenges - The company relies on third-party manufacturers for product production, and any failure in compliance or timely production could delay commercialization and result in lost revenues [246]. - The company must navigate intense competition from other pharmaceutical and biotechnology firms, which may develop more effective or cost-efficient alternatives [224]. - There are limited suppliers for raw materials necessary for manufacturing, and disruptions in the supply chain could significantly delay clinical trials and product launches [261]. - The company does not have any manufacturing facilities and is dependent on third parties for the manufacture of its product candidates, which could impair development efforts if these manufacturers do not perform satisfactorily [258]. - The company anticipates that future dependence on third parties for manufacturing may negatively impact profit margins and commercialization timelines [265]. - The company must ensure adequate oversight of third-party manufacturers to comply with regulatory standards, which adds complexity to its operational strategy [260]. Intellectual Property and Patent Risks - The patent prosecution process is costly and time-consuming, impacting the ability to secure necessary patent protections for product candidates [301]. - Failure to maintain patent protection could allow competitors to develop similar products, adversely affecting revenue generation and market competitiveness [300]. - The patent position of biotechnology and pharmaceutical companies is highly uncertain, with complex legal questions and significant litigation risks [304]. - U.S. patent laws may change, potentially limiting the ability to file patent applications or affecting the exclusivity periods for patent holders [305]. - The company may face substantial costs from third-party challenges to its patent rights, which could adversely affect its U.S. patent position [306]. - Even if patents are issued, they may not provide meaningful protection against competitors or prevent them from developing similar technologies [307]. - The issuance of a patent does not guarantee its validity or enforceability, and challenges could lead to loss of exclusivity or narrowed claims [308]. - The time required for product development may result in patents expiring before commercialization, limiting competitive advantage [309]. - The company may need to license intellectual property from third parties, which may not be available on commercially reasonable terms [312]. - The company relies on licensors to maintain and enforce intellectual property rights, which may lead to risks if licensors do not perform adequately [315]. - Litigation related to intellectual property claims can be expensive and time-consuming, potentially distracting management from core business activities [317]. - The company may face claims regarding the wrongful use of trade secrets by employees who previously worked for competitors, leading to potential litigation costs [331]. Financial and Market Risks - The company’s future growth depends on its ability to identify and acquire or in-license products that align with its strategic focus [172]. - The company may face operational and financial risks associated with future acquisitions or in-licensing opportunities, including exposure to unknown liabilities and integration challenges [173]. - There is significant uncertainty regarding third-party coverage and reimbursement for newly approved drugs, which could affect market acceptance and sales [232]. - The company intends to build a targeted specialist sales force to market its products, but establishing sales and marketing capabilities poses risks and could delay product launches [238]. - The company may struggle to gain market acceptance for its product candidates, which is crucial for generating revenue and achieving profitability [230]. - Cybersecurity risks are significant, with potential impacts from computer system failures, cyber-attacks, and unauthorized access, which could compromise confidential information [335]. - Legal and financial exposure from security incidents could adversely affect the company's business and reputation [342]. - Conflicts of interest may arise due to the Chairman of the Board also serving as the Executive Chairman, President, and CEO of TGTX, with whom the company has collaboration agreements [353]. - The company has outsourced operations to third-party vendors, increasing the risk of disclosure of confidential information [337]. - The company may not have received the best terms in agreements with Fortress compared to potential third-party negotiations [351].

Revenue - For the three months ended September 30, 2021, revenue was approximately $29,000, an increase of $1,000 compared to approximately $28,000 for the same period in 2020[159]. - For the nine months ended September 30, 2021, revenue was approximately $0.3 million, a decrease of $0.7 million compared to approximately $1.0 million for the same period in 2020[167]. Research and Development Expenses - Research and development expenses for the three months ended September 30, 2021, were approximately $9.4 million, an increase of $6.9 million from $2.5 million for the same period in 2020[161]. - Research and development expenses for the nine months ended September 30, 2021, were approximately $20.8 million, an increase of $12.6 million from $8.2 million for the same period in 2020[168]. - The company anticipates research and development expenses to increase for the remainder of 2021[162]. General and Administrative Expenses - General and administrative expenses for the three months ended September 30, 2021, were approximately $1.9 million, a decrease of $0.5 million from $2.4 million for the same period in 2020[164]. - General and administrative expenses for the nine months ended September 30, 2021, were approximately $6.4 million, an increase of $0.6 million from $5.8 million for the same period in 2020[169]. Operating Losses and Deficit - As of September 30, 2021, the accumulated deficit was $170.1 million, with expectations of continued significant operating losses[170]. Cash Flow - Net cash used in operating activities was approximately $16.8 million for the nine months ended September 30, 2021, compared to approximately $11.6 million for the same period in 2020[173]. - Net cash provided by financing activities was $36.3 million for the nine months ended September 30, 2021, compared to $27.6 million for the same period in 2020[176].