What changed in RIGEL PHARMACEUTICALS INC's 10-K — 2024 vs 2025
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Paragraph-level year-over-year comparison of RIGEL PHARMACEUTICALS INC's 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.
+299 added−836 removedSource: 10-K (2026-03-03) vs 10-K (2025-03-04)
Top changes in RIGEL PHARMACEUTICALS INC's 2025 10-K
299 paragraphs added · 836 removed · 227 edited across 1 sections
- Item 1A. Risk Factors+299 / −836 · 227 edited
Item 1A. Risk Factors
Risk Factors — what could go wrong, per management
227 edited+72 added−609 removed444 unchanged
Item 1A. Risk Factors
Risk Factors — what could go wrong, per management
227 edited+72 added−609 removed444 unchanged
2024 filing
2025 filing
Biggest changeIn addition to data privacy requirements, cybersecurity requirements are laid down in various laws in the EU and the UK, the key ones being: (i) the GDPR (as discussed above), which requires controllers and processors to implement appropriate technical and organizational measures to safeguard personal data to a level of security appropriate to the data protection risk; (ii) the UK Network and Information Systems Regulation 2018 (NIS Regulations), and (iii) the EU Network and Information Systems Security 2 Directive (NISD2). 46 Table of Contents The GDPR does not provide for a specific set of cybersecurity requirements or measures to be implemented, but rather requires a controller or processor to implement appropriate cyber and data security measures in accordance with the then-current risk, the state of the art, the costs of implementation and the nature, scope, context and purposes of the processing.
Biggest changeIn addition to data privacy requirements, cybersecurity requirements are laid down in various laws in the EU and the UK, the key ones being: (i) the GDPR (as discussed above), which requires controllers and processors to implement appropriate technical and organizational measures to safeguard personal data to a level of security appropriate to the risk; (ii) the UK Network and Information Systems Regulations 2018 (NIS Regulations), and (iii) the EU Network and Information Systems 2 Directive (NISD2).
Parts 46, 50, and 56. States, such as Colorado, Utah and California, have passed or are considering legislation or regulation governing the development or use of artificial intelligence technologies, supplementing the existing consumer protection, FDA and other regulatory guidance that may apply to the use of AI technologies in our business, and which may impact our use of technology.
Parts 46, 50, and 56. States, such as Colorado, Utah and California, have passed or are considering legislation or regulation governing the development or use of artificial intelligence (AI) technologies, supplementing the existing consumer protection, FDA and other regulatory guidance that may apply to the use of AI technologies in our business, and which may impact our use of technology.
The CER is enforceable on a national EU Member State level by the competent authorities, and allows EU Member States to set penalties as long as they are effective, proportionate, and dissuasive. Our entities may be in scope of the CER where they qualify as critical entities within the meaning of CER.
CER is enforceable on a national EU Member State level by the competent authorities, and allows EU Member States to set penalties as long as they are effective, proportionate, and dissuasive. Our entities may be in scope of CER where they qualify as critical entities within the meaning of CER.
The CER has not to date been transposed by all EU Member States despite the deadline for doing so having passed. In the EU, a number of new laws related to digital data and AI have recently entered into force, are expected to enter into force in the foreseeable future, or have been proposed and are being considered.
CER has not to date been transposed by all EU Member States despite the deadline for doing so having passed. In the EU, a number of new laws related to digital data and AI have recently entered into force, are expected to enter into force in the foreseeable future, or have been proposed and are being considered.
Our future funding requirements will depend upon many factors, many of which are beyond our control, including, but not limited to: ● the costs to commercialize our products in the US, or any other future product candidates, if any such candidate receives regulatory approval for commercial sale; ● the progress and success of our clinical trials and preclinical activities (including studies and manufacture of materials) of our product candidates conducted by us; ● our ability to secure patent and regulatory protection; ● our ability to secure a favorable price or a positive HTA assessment; ● potential future impacts, if any, of a global pandemic; 57 Table of Contents ● the costs and timing of regulatory filings and approvals by us and our collaborators; ● the progress of research and development programs carried out by us and our collaborative partners; ● any changes in the breadth of our research and development programs; ● the ability to achieve the events identified in our collaborative agreements that may trigger payments to us from our collaboration partners; ● our ability to acquire or license other technologies or compounds that we may seek to pursue; ● our ability to manage our growth; ● competing technological and market developments; ● the costs and timing of obtaining, enforcing and defending our patent and other intellectual property rights; and ● expenses associated with any unforeseen litigation, including any arbitration and securities class action lawsuits.
Our future funding requirements will depend upon many factors, many of which are beyond our control, including, but not limited to: • the costs to commercialize our products in the US, or any other future product candidates, if any such candidate receives regulatory approval for commercial sale; • the progress and success of our clinical trials and preclinical activities (including studies and manufacture of materials) of our product candidates conducted by us; • our ability to secure patent and regulatory protection; • our ability to secure a favorable price or a positive HTA assessment; • potential future impacts, if any, of a global pandemic; • the costs and timing of regulatory filings and approvals by us and our collaborators; 54 Table of Contents • the progress of research and development programs carried out by us and our collaborative partners; • any changes in the breadth of our research and development programs; • the ability to achieve the events identified in our collaborative agreements that may trigger payments to us from our collaboration partners; • our ability to acquire or license other technologies or compounds that we may seek to pursue; • our ability to manage our growth; • competing technological and market developments; • the costs and timing of obtaining, enforcing and defending our patent and other intellectual property rights; and • expenses associated with any unforeseen litigation, including any arbitration and securities class action lawsuits.
Actual results may differ materially from projected results for various reasons, including the following, as well as risks identified in other risk factors: ● the efficacy and safety of any of our product candidates, including as relative to marketed products and product candidates in development by third parties; ● pricing (including discounting or other promotions), reimbursement, product returns or recalls, competition, labeling, adverse events and other items that impact commercialization; 56 Table of Contents ● the rate of adoption in the particular market, including fluctuations in demand for various reasons; ● potential future impacts, if any, including a global pandemic; ● lack of patient and physician familiarity with the drug; ● lack of patient use and physician prescribing history; ● lack of commercialization experience with the drug; ● actual sales to patients may significantly differ from expectations based on sales to wholesalers; and ● uncertainty relating to when the drug may become commercially available to patients and rate of adoption in other territories.
Actual results may differ materially from projected results for various reasons, including the following, as well as risks identified in other risk factors: • the efficacy and safety of any of our product candidates, including as relative to marketed products and product candidates in development by third parties; • pricing (including discounting or other promotions), reimbursement, product returns or recalls, competition, labeling, adverse events and other items that impact commercialization; 53 Table of Contents • the rate of adoption in the particular market, including fluctuations in demand for various reasons; • potential future impacts, if any, including a global pandemic; • lack of patient and physician familiarity with the drug; • lack of patient use and physician prescribing history; • lack of commercialization experience with the drug; • actual sales to patients may significantly differ from expectations based on sales to wholesalers; and • uncertainty relating to when the drug may become commercially available to patients and rate of adoption in other territories.
Federal NOLs generated prior to 2018 will continue to be governed by the NOL carryforward rules as they existed prior to the adoption of the Tax Cuts and Jobs Act (Tax Act), which means that generally they will expire 20 years after they were generated if not used prior thereto. Many states have similar laws.
Federal NOLs generated prior to 2018 will continue to be governed by the NOL carryforward rules as they existed prior to the adoption of the Tax Cuts and Jobs Act (TCJA), which means that generally they will expire 20 years after they were generated if not used prior thereto. Many states have similar laws.
Our ability to use our federal and state NOLs to offset potential future taxable income and related income taxes that would otherwise be due is dependent upon our generation of future taxable income before the expiration dates of the NOLs, and we cannot predict with certainty when, or whether, we will generate sufficient taxable income to use all of our NOLs.
Our ability to use our federal and state NOLs to offset potential future taxable income and related income taxes that would otherwise be due dependent upon our generation of future taxable income before the expiration dates of the NOLs, and we cannot predict with certainty when, or whether, we will generate sufficient taxable income to use all of our NOLs.
To help patients afford our products, we have a manufacturer-sponsored patient assistance program that helps financially needy patients in the US access our therapies. This type of program has become the subject of enforcement scrutiny in recent years.
To help patients afford our products, we have a manufacturer-sponsored patient assistance program that helps eligible financially needy patients in the US access our therapies. This type of program has become the subject of enforcement scrutiny in recent years.
If we or others identify additional undesirable side effects caused by our products after approval: ● regulatory authorities may require the addition of labeling statements, specific warnings, contraindications, Dear Healthcare Provider letters, press releases, field alerts, or other communications containing warnings or other safety information about our products to physicians and pharmacies; 50 Table of Contents ● regulatory authorities may withdraw their approval of the product and require us to take our approved drugs off the market or suspend their commercialization until the identified issues have been satisfactorily addressed; ● we may be required to change the way the product is administered, conduct additional clinical trials, change the labeling of the product, or implement a Risk Evaluation and Mitigation Strategy (REMS); ● we may have additional limitations on how we promote our drugs; ● third-party payors may limit coverage or reimbursement for our products; ● sales of our products may decrease significantly; ● we may be subject to litigation or product liability claims; and ● our reputation may suffer.
If we or others identify additional undesirable side effects caused by our products after approval: • regulatory authorities may require the addition of labeling statements, specific warnings, contraindications, Dear Healthcare Provider letters, press releases, field alerts, or other communications containing warnings or other safety information about our products to physicians and pharmacies; • regulatory authorities may withdraw their approval of the product and require us to take our approved drugs off the market or suspend their commercialization until the identified issues have been satisfactorily addressed; • we may be required to change the way the product is administered, conduct additional clinical trials, change the labeling of the product, or implement a Risk Evaluation and Mitigation Strategy (REMS); • we may have additional limitations on how we promote our drugs; • third-party payors may limit coverage or reimbursement for our products; • sales of our products may decrease significantly; • we may be subject to litigation or product liability claims; and • our reputation may suffer.
The degree of market acceptance of our product candidates, if approved for commercial sale, will depend on a number of factors, including the following: ● relative convenience and ease of administration; ● the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; ● the willingness of physicians to change their current treatment practices; ● any additional support that may be required to administer the treatment to patients; ● the willingness of hospitals and hospital systems to include our product candidates as treatment options; ● demonstration of efficacy and safety in clinical trials; ● the prevalence and severity of any side effects; ● the ability to offer product candidates for sale at competitive prices; ● the price we charge for our product candidates; ● the strength of marketing and distribution support; and ● the availability of third-party coverage and adequate reimbursement and the willingness of patients to pay out-of-pocket in the absence of such coverage and adequate reimbursement.
The degree of market acceptance of our product candidates, if approved for commercial sale, will depend on a number of factors, including the following: • relative convenience and ease of administration; • the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; • the willingness of physicians to change their current treatment practices; • any additional support that may be required to administer the treatment to patients; • the willingness of hospitals and hospital systems to include our product candidates as treatment options; • demonstration of efficacy and safety in clinical trials; • the prevalence and severity of any side effects; • the ability to offer product candidates for sale at competitive prices; 51 Table of Contents • the price we charge for our product candidates; • the strength of marketing and distribution support; and • the availability of third-party coverage and adequate reimbursement and the willingness of patients to pay out-of-pocket in the absence of such coverage and adequate reimbursement.
Should Annora or any other third parties receive FDA approval of an ANDA for a generic version of fostamatinib or a 505(b)(2) NDA with respect to fostamatinib, and if our patents covering fostamatinib were held to be invalid (or if such competing generic versions of fostamatinib were found to not infringe our patents), then they could introduce generic versions of fostamatinib or other such 505(b)(2) products before our patents expire, and the resulting competition would negatively affect our business, financial condition and results of operations.
Should any third parties receive FDA approval of an ANDA for a generic version of our products or a 505(b)(2) NDA with respect to our products, and if our patents covering our products were held to be invalid (or if such competing generic versions of our products were found to not infringe our patents), then they could introduce generic versions of our products or other such 505(b)(2) products before our patents expire, and the resulting competition would negatively affect our business, financial condition and results of operations.
Discovery after approval of previously unknown problems with any of our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in actions such as: ● restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials; ● restrictions on product manufacturing processes; ● restrictions on the marketing of a product; ● restrictions on product distribution; ● requirements to conduct post-marketing clinical trials; ● untitled or warning letters or other adverse publicity; ● withdrawal of products from the market; ● refusal to approve pending applications or supplements to approved applications that we submit; ● recall of products; ● refusal to permit the import or export of our products; ● product seizure; ● fines, restitution or disgorgement of profits or revenue; ● refusal to allow us to enter into supply contracts, including government contracts; ● injunctions; or 78 Table of Contents ● imposition of civil or criminal penalties.
Discovery after approval of previously unknown problems with any of our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in actions such as: • restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials; • restrictions on product manufacturing processes; • restrictions on the marketing of a product; • restrictions on product distribution; • requirements to conduct post-marketing clinical trials; • untitled or warning letters or other adverse publicity; • withdrawal of products from the market; • refusal to approve pending applications or supplements to approved applications that we submit; • recall of products; • refusal to permit the import or export of our products; • product seizure; • fines, restitution or disgorgement of profits or revenue; • refusal to allow us to enter into supply contracts, including government contracts; • injunctions; or • imposition of civil or criminal penalties.
We have also entered into exclusive commercialization agreements with third parties to commercialize fostamatinib outside the US, and we plan to further enter partnership with existing or other third parties to commercialize our products outside the US in the future.
We have also entered into exclusive commercialization agreements with third parties to commercialize our products outside the US, and we plan to further enter partnership with existing or other third parties to commercialize our products outside the US in the future.
The GDPR imposes obligations on controllers, including, among others: ● accountability and transparency requirements, requiring controllers to demonstrate and record compliance with the GDPR and to provide more detailed information to data subjects regarding the processing of their personal data; ● requirements to process personal data lawfully including specific requirements for obtaining valid consent where consent is the lawful basis for processing; ● obligations to consider data protection when any new products or services are developed and designed (including e.g., to limit the amount of personal data processed); ● obligations to comply with data protection rights of data subjects including a right: (i) of access to, erasure of, or rectification of personal data, (ii) to restriction of processing or to withdraw consent to processing, (iii) to object to processing or to ask for a copy of personal data to be provided to a third party, and (iv) not to be subject to solely automated decision-making; and ● an obligation to report personal data breaches to: (i) the data protection supervisory authority without undue delay (and no later than 72 hours) after becoming aware of the personal data breach, where feasible, unless the personal data breach is unlikely to result in a risk to the data subjects’ rights and freedoms; and (ii) affected data subjects, where the personal data breach is likely to result in a high risk to their rights and freedoms.
The GDPR imposes obligations on controllers, including, among others: • accountability and transparency requirements, requiring controllers to demonstrate and record compliance with the GDPR and to provide detailed information to data subjects regarding the processing of their personal data; • requirements to process personal data lawfully including specific requirements for obtaining valid consent where consent is the lawful basis for processing; 42 Table of Contents • obligations to consider data protection when any new products or services are developed and designed (including e.g., to limit the amount of personal data processed); • obligations to comply with data protection rights of data subjects including a right: (i) of access to, erasure of, or rectification of personal data, (ii) to restriction of processing or to withdraw consent to processing, (iii) to object to processing or to ask for a copy of personal data to be provided to a third party, and (iv) not to be subject to solely automated decision-making; and • an obligation to report personal data breaches to: (i) the data protection supervisory authority without undue delay (and no later than 72 hours) after becoming aware of the personal data breach, where feasible, unless the personal data breach is unlikely to result in a risk to the data subjects’ rights and freedoms; and (ii) affected data subjects, where the personal data breach is likely to result in a high risk to their rights and freedoms without undue delay.
As we acquire or produce more FDA approved inventory quantities in the future, our inventory cost in the balance sheet and cost of product sales will reflect the full cost of acquiring or producing such products.
As we acquire or produce more FDA approved inventory quantities, our inventory cost in the balance sheet and cost of product sales will reflect the full cost of acquiring or producing such products.
Because the degree of future protection for our proprietary rights is uncertain, we cannot assure that: ● we were the first to make the inventions covered by each of our pending patent applications; ● we were the first to file patent applications for these inventions; ● others will not independently develop similar or alternative technologies or duplicate any of our 60 Table of Contents technologies; ● any of our pending patent applications will result in issued patents; ● any patents issued to us or our collaborators will provide a basis for commercially viable products or will provide us with any competitive advantages or will not be challenged by third parties; ● we will develop additional proprietary technologies that are patentable; ● we will obtain a supplementary protection certificate that will extend the protection afforded by the patent to the product with a marketing authorization; or ● the patents of others will not have a negative effect on our ability to do business.
Because the degree of future protection for our proprietary rights is uncertain, we cannot assure that: • we were the first to make the inventions covered by each of our pending patent applications; • we were the first to file patent applications for these inventions; • others will not independently develop similar or alternative technologies or duplicate any of our technologies; • any of our pending patent applications will result in issued patents; • any patents issued to us or our collaborators will provide a basis for commercially viable products or will provide us with any competitive advantages or will not be challenged by third parties; • we will develop additional proprietary technologies that are patentable; • we will obtain a supplementary protection certificate that will extend the protection afforded by the patent to the product with a marketing authorization; or • the patents of others will not have a negative effect on our ability to do business.
These provisions: ● establish that members of our Board of Directors may be removed only for cause upon the affirmative vote of stockholders owning a majority of our capital stock; ● authorize the issuance of “blank check” preferred stock that could be issued by our Board of Directors to increase the number of outstanding shares and thwart a takeover attempt; ● limit who may call a special meeting of stockholders; ● prohibit stockholder action by written consent, thereby requiring all stockholder actions to be taken at a meeting of our stockholders; ● establish advance notice requirements for nominations for election to our Board of Directors or for proposing matters that can be acted upon at stockholder meetings; ● provide for staggered terms for our Board of Directors; and ● provide that the authorized number of directors may be changed only by a resolution of our Board of Directors.
These provisions: • establish that members of our Board of Directors may be removed only for cause upon the affirmative vote of stockholders owning a majority of our capital stock; • authorize the issuance of “blank check” preferred stock that could be issued by our Board of Directors to increase the number of outstanding shares and thwart a takeover attempt; • limit who may call a special meeting of stockholders; • prohibit stockholder action by written consent, thereby requiring all stockholder actions to be taken at a meeting of our stockholders; • establish advance notice requirements for nominations for election to our Board of Directors or for proposing matters that can be acted upon at stockholder meetings; • provide for staggered terms for our Board of Directors; and 82 Table of Contents • provide that the authorized number of directors may be changed only by a resolution of our Board of Directors.
If our collaborators are unable to successfully complete clinical trials, delay commercialization of fostamatinib or do not invest the resources necessary to successfully commercialize fostamatinib in international territories where it has been approved, this could reduce the amount of revenue we are due to receive under these license agreements, resulting in harm to our business and operations.
If our collaborators are unable to successfully complete clinical trials, delay commercialization of our products or do not invest the resources necessary to successfully commercialize our products in international territories where it has been approved, this could reduce the amount of revenue we are due to receive under these license agreements, resulting in harm to our business and operations.
In connection with clinical trials of our product candidates, we may face the following risks among others: ● the product candidate may not prove to be effective; ● the product candidate may cause harmful side effects; ● the clinical results may not replicate the results of earlier, smaller trials; ● we or third parties with whom we collaborate, may be significantly impacted by force majeure events; ● we, or the FDA or similar foreign regulatory authorities, may delay, terminate or suspend the trials; ● our results may not be statistically significant; ● patient recruitment and enrollment may be slower than expected; ● patients may drop out of the trials or otherwise not enroll; and ● regulatory and clinical trial requirements, interpretations or guidance may change.
In connection with clinical trials of our product candidates, we may face the following risks among others: • the product candidate may not prove to be effective; • the product candidate may cause harmful side effects; • the clinical results may not replicate the results of earlier, smaller trials; • we or third parties with whom we collaborate, may be significantly impacted by force majeure events; • we, or the FDA or similar foreign regulatory authorities, may delay, terminate or suspend the trials; 80 Table of Contents • our results may not be statistically significant; • patient recruitment and enrollment may be slower than expected; • patients may drop out of the trials or otherwise not enroll; and • regulatory and clinical trial requirements, interpretations or guidance may change.
Please also see the risk factor entitled, “If manufacturers obtain approval for generic versions of our products, or of products with which we compete, our business may be harmed.” In the future, there might be other claims that are subject to substantial uncertainties and unascertainable damages or other remedies, and the cost to defend may also be significant.
Please also see the risk factor entitled, “ If manufacturers obtain approval for generic versions of our products, or of products with which we compete, our business may be harmed. ” In the future, there might be other claims that are subject to substantial uncertainties and unascertainable damages or other remedies, and the cost to defend may also be significant.
Given the lack of clarity in the laws and their implementation, our reporting actions could be subject to the penalty provisions of the pertinent federal and state laws and regulations.
Given the lack of clarity in the laws and their implementation, our reporting actions could be subject to the penalty provisions of the pertinent state laws and regulations.
Despite our efforts to monitor evolving social media communication guidelines and comply with applicable laws, regulations and national and EU codes of conduct, there is risk that the unauthorized use of social media by us or our employees to communicate about our products or business, sharing of publications in unintended audiences 88 Table of Contents in other jurisdictions, or any inadvertent promotional activity or disclosure of material, nonpublic information through these means, may cause us to be found in violation of applicable laws and regulations, which may give rise to liability and result in harm to our business.
Despite our efforts to monitor evolving social media communication guidelines and comply with applicable laws, regulations and national and EU codes of conduct, there is risk that the unauthorized use of social media by us or our employees to communicate about our products or business, sharing of publications in unintended audiences in other jurisdictions, or any inadvertent promotional activity or disclosure of material, nonpublic information through these means, may cause us to be found in violation of applicable laws and regulations, which may give rise to liability and result in harm to our business.
As of December 31, 2024, we have not sold any shares of common stock under such Open Market Sale Agreement. We may also in the future enter into underwriting or sales agreements with financial institutions for the offer and sale of any combination of common stock, preferred stock, debt securities and warrants in one or more offerings.
As of December 31, 2025 , we have not sold any shares of common stock under such Open Market Sale Agreement. We may also in the future enter into underwriting or sales agreements with financial institutions for the offer and sale of any combination of common stock, preferred stock, debt securities and warrants in one or more offerings.
The HTA Regulation intends to foster cooperation among EU member states in assessing health technologies and provide a procedure for joint clinical assessments of medicinal products at a centralized level. It requires companies applying for products in scope to make relevant submissions for the joint clinical assessment, in line with a number of prespecified criteria.
The HTA Regulation intends to foster cooperation among EU member states in assessing health technologies and provides a procedure for joint clinical assessments of medicinal products at a centralized level. It requires companies applying for products in scope to make relevant submissions for the joint clinical assessment, in line with a number of prespecified criteria.
On June 28, 2024, the US Supreme Court issued an opinion holding that courts reviewing agency action pursuant to the Administrative Procedure Act “must exercise their independent judgment” and “may not defer to an agency interpretation of the law simply because a statute is ambiguous.” The decision will have a significant impact on how lower courts evaluate challenges to agency interpretations of law, including those by the FDA, DHHS, CMS and other agencies with significant oversight of the biopharmaceutical industry.
On June 28, 2024, the US Supreme Court issued an opinion holding that courts reviewing agency action pursuant to the Administrative Procedure Act (APA) “must exercise their independent judgment” and “may not defer to an agency interpretation of the law simply because a statute is ambiguous.” The decision will have a significant impact on how lower courts evaluate challenges to agency interpretations of law, including those by HHS, CMS, FDA and other agencies with significant oversight of the biopharmaceutical industry.
We cannot predict the likelihood, nature, or extent of health reform initiatives that may arise from future legislation or administrative action. However, we expect these initiatives to increase pressure on drug pricing. Further, certain broader legislation that is not targeted to the health care industry may nonetheless adversely affect our profitability.
We cannot predict the likelihood, nature, or extent of health reform initiatives that may arise from future legislation or administrative action. However, we expect these initiatives to increase pressure on drug pricing. Further, certain broader legislation that is not targeted to the healthcare industry may nonetheless adversely affect our profitability.
The commercial success of any product for which we have obtained regulatory approval, or for which we may obtain regulatory approval in the future will depend substantially on the extent to which the costs of our product or product candidates are or will be paid by third-party payors, including government health care programs and private health insurers.
The commercial success of any product for which we have obtained regulatory approval, or for which we may obtain regulatory approval in the future will depend substantially on the extent to which the costs of our product or product candidates are or will be paid by third-party payors, including government healthcare programs and private health insurers.
The following factors, in addition to other risk factors described in this section, may have a significant impact on the market price of our common stock: ● the progress and success of our clinical trials and preclinical activities (including studies and manufacture of materials) of our product candidates conducted by us; ● our ability to continue to sell our products in the US; ● our ability to enter into partnering opportunities across our pipeline; ● the receipt or failure to receive the additional funding necessary to conduct our business; ● selling of our common stock by large stockholders; ● presentations of detailed clinical trial data at medical and scientific conferences and investor perception 65 Table of Contents thereof; ● announcements of technological innovations or new commercial products by our competitors or us; ● the announcement of regulatory applications, such as Annora’s ANDA, seeking approval of generic versions of our marketed products; ● developments concerning proprietary rights, including patents; ● developments concerning our collaborations; ● publicity regarding actual or potential medical results relating to products under development by our competitors or us; ● regulatory developments in the US and foreign countries; ● changes in the structure of healthcare payment systems; ● litigation or arbitration; ● economic and other external factors or other disaster or crisis; and ● period-to-period fluctuations in financial results.
The following factors, in addition to other risk factors described in this section, may have a significant impact on the market price of our common stock: • the progress and success of our clinical trials and preclinical activities (including studies and manufacture of materials) of our product candidates conducted by us; • our ability to continue to sell our products in the US; • our ability to enter into partnering opportunities across our pipeline; • the receipt or failure to receive the additional funding necessary to conduct our business; • selling of our common stock by large stockholders; • presentations of detailed clinical trial data at medical and scientific conferences and investor perception thereof; • announcements of technological innovations or new commercial products by our competitors or us; • the announcement of regulatory applications, such as third parties seeking approval of generic versions of our marketed products; • developments concerning proprietary rights, including patents; • developments concerning our collaborations; 62 Table of Contents • publicity regarding actual or potential medical results relating to products under development by our competitors or us; • regulatory developments in the US and foreign countries; • changes in the structure of healthcare payment systems; • litigation or arbitration; • economic and other external factors or other disaster or crisis; and • period-to-period fluctuations in financial results.
The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or reduce the costs of healthcare and/or impose price controls may adversely affect, for example: ● the demand for our products, or our product candidates, if we obtain regulatory approval; 71 Table of Contents ● our ability to set a price that we believe is fair for our products; ● our ability to generate revenue and achieve or maintain profitability; ● the level of taxes that we are required to pay; and ● the availability of capital.
The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or reduce the costs of healthcare and/or impose price controls may adversely affect, for example: • the demand for our products, or our product candidates, if we obtain regulatory approval; • our ability to set a price that we believe is fair for our products; • our ability to generate revenue and achieve or maintain profitability; • the level of taxes that we are required to pay; and • the availability of capital.
In addition, exclusive marketing rights in the US for fostamatinib for the treatment of ITP, wAIHA or any future product candidate may be limited if we seek 80 Table of Contents approval for an indication broader than the orphan-designated indication or may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.
In addition, exclusive marketing rights in the US for fostamatinib for the treatment of ITP, wAIHA or any future product candidate may be limited if we seek approval for an indication broader than the orphan-designated indication or may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.
We commercialize our products in the US and we have entered into commercialization agreements with third parties to commercialize fostamatinib outside the US.
We commercialize our products in the US and we have entered into commercialization agreements with third parties to commercialize our products outside the US.
Furthermore, foreign sales of fostamatinib by our partners could be adversely affected by the imposition of governmental controls, political and economic instability, outbreaks of pandemic diseases, such as the COVID-19 pandemic, trade restrictions or barriers and changes in tariffs and escalating global trade and political tensions.
Furthermore, foreign sales of our products by our partners could be adversely affected by the imposition of governmental controls, political and economic instability, outbreaks of pandemic diseases, such as the COVID-19 pandemic, trade restrictions or barriers and changes in tariffs and escalating global trade and political tensions.
In addition, we cannot predict what additional ANDAs could be filed by Annora or other potential generic competitors requesting approval to market generic forms of our products, which would require us to incur significant additional expense and result in distraction for our management team, and if approved, result in significant decreases in the revenue derived from sales of our marketed products and thereby materially harm our business and financial condition.
In addition, we cannot predict what additional ANDAs could be filed by potential generic competitors requesting approval to market generic forms of our products, which would require us to incur significant additional expense and result in distraction for our management team, and if approved, result in significant decreases in the revenue derived from sales of our marketed products and thereby materially harm our business and financial condition.
In general, under the Code, if a corporation undergoes an “ownership change,” generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development credit carryforwards) to offset its post-change taxable income or taxes may be limited.
In general, under Section 174, if a corporation undergoes an “ownership change,” generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development credit carryforwards) to offset its post-change taxable income or taxes may be limited.
As a consequence of our license agreements with our collaboration partners, we rely heavily upon their regulatory, commercial, medical affairs, market access and other expertise and resources for commercialization of fostamatinib in their respective territories outside of the US.
As a consequence of our license agreements with our collaboration partners, we rely heavily upon their regulatory, commercial, medical affairs, market access and other expertise and resources for commercialization of our products in their respective territories outside of the US.
For example, the CCPA, as amended by the California Privacy Rights Act (CPRA), establishes a privacy framework applicable to for-profit entities that are doing business in California, including an expansive definition of personal information and data privacy rights for California residents (as consumers, business contacts and employees), and authorizes potentially severe statutory damages and creates a private right of action for certain data security breaches.
For example, the CCPA, as amended by the California Privacy Rights Act (CPRA), establishes a privacy framework applicable to for-profit entities that are doing business in California, 41 Table of Contents including an expansive definition of personal information and data privacy rights for California residents (as consumers, business contacts and employees), and authorizes potentially severe statutory damages and creates a private right of action for certain data security breaches.
The scope of CER includes entities designated as ‘critical’ under CER and includes (among other things) the health sector and the manufacturers of medical devices as ‘essential services.’ The CER imposes cybersecurity and resilience requirements in particular in relation to incidents with so-called ‘significant disruptive effects’ – which are incidents that are able to significantly impact the continuation of the critical infrastructure service offering in the EU.
The scope of CER includes entities designated as ‘critical’ under CER and includes (among other things) the health sector and the manufacturers of medical devices as ‘essential services.’ CER imposes cybersecurity and resilience requirements in particular in relation to incidents with so-called ‘significant disruptive effects’ – which are incidents that 44 Table of Contents are able to significantly impact the continuation of the critical infrastructure service offering in the EU.
On January 1, 2024, the MHRA launched a new International Recognition Procedure for Great Britain (England, Scotland and Wales) marketing authorization applications whereby the MHRA 67 Table of Contents will, when considering such applications, recognize the approval of medicines by trusted reference regulators in Australia, Canada, Switzerland, Singapore, Japan, United States and EU following its own abbreviated assessment.
On January 1, 2024, the MHRA launched a new International Recognition Procedure for Great Britain (England, Scotland and Wales) marketing authorization applications whereby the MHRA will, when considering such applications, recognize the approval of medicines by trusted reference regulators in Australia, Canada, Switzerland, Singapore, Japan, United States and EU following its own abbreviated assessment.
If we are unable to effectively train our sales force and equip them with compliant and effective materials, including medical and sales literature to help them appropriately inform and educate health care providers regarding the potential benefits and proper administration of our products, our efforts to successfully commercialize our products could be put in jeopardy, which would negatively impact our ability to generate product revenues.
If we are unable to effectively train our sales force and equip them with compliant and effective materials, including medical and sales literature to help them appropriately inform and educate healthcare providers regarding the potential benefits and proper administration of our products, our efforts to successfully commercialize our products could be put in jeopardy, which would negatively impact our ability to generate product revenues.
Data protection supervisory authorities have the power under the GDPR to (amongst other thing) impose fines for serious breaches of up to the higher of 4% of the organization’s annual worldwide turnover or €20 million (under the EU GDPR) or £17.5 million (under the UK GDPR).
Data protection supervisory authorities have the power under the GDPR to (amongst other things) impose fines for serious breaches of up to the higher of 4% of the organization’s annual worldwide turnover or €20 million (under the EU GDPR) or £17.5 million (under the UK GDPR).
We do not know if we will be able to maintain such consulting agreements or that such scientific advisors will not enter 68 Table of Contents into consulting arrangements with competing pharmaceutical or biotechnology companies, any of which may have a detrimental impact on our research objectives and could have an adverse effect on our business, financial condition and results of operations.
We do not know if we will be able to maintain such consulting agreements or that such scientific advisors will not enter into consulting arrangements with competing pharmaceutical or biotechnology companies, any of which may have a detrimental impact on our research objectives and could have an adverse effect on our business, financial condition and results of operations.
We cannot control the amount of resources that our partners dedicate to the commercialization of fostamatinib, and our ability to generate revenues from the commercialization of fostamatinib by our partners depends on their ability to achieve market acceptance of fostamatinib in its approved indications in their respective territories.
We cannot control the amount of resources that our partners dedicate to the commercialization of our products, and our ability to generate revenues from the commercialization of our products by our partners depends on their ability to achieve market acceptance of our products in its approved indications in their respective territories.
We cannot guarantee that these compliance activities will prevent or timely detect off-label promotion by sales representatives or other personnel in their communications with health care professionals, patients and others, particularly if these activities are concealed from us. Regulatory authorities in the US generally do not regulate the behavior of physicians in their choice of treatments.
We cannot guarantee that these compliance activities will prevent or timely detect off-label promotion by sales representatives or other personnel in their communications with healthcare professionals, patients and others, particularly if these activities are concealed from us. Regulatory authorities in the US generally do not regulate the behavior of physicians in their choice of treatments.
There is a significant trend in the health care industry by public and private payors to contain or reduce their costs, including by taking the following steps, among others: decreasing the portion of costs payors will cover, ceasing to provide full payment for certain products depending on outcomes, and/or not covering certain products at all.
There is a significant trend in the healthcare industry by public and private payors to contain or reduce their costs, including by taking the following steps, among others: decreasing the portion of costs payors will cover, ceasing to provide full payment for certain products depending on outcomes, and/or not covering certain products at all.
In particular, in March 2010, the Affordable Care Act was enacted, which substantially changed the way health care is financed by both governmental and private insurers, and continues to significantly impact the US pharmaceutical industry.
In particular, in March 2010, the Affordable Care Act was enacted, which substantially changed the way healthcare is financed by both governmental and private insurers, and continues to significantly impact the US pharmaceutical industry.
Geopolitical developments, including changes arising as a result of the recent US presidential election, may lead to further developments with respect to the imposition or threat of imposition of trade policies, tariffs, taxes and other limitations on cross-border operations. Such tensions could adversely impact us and our third-party manufacturers.
Geopolitical developments, including changes arising as a result of the 2024 US presidential election, may lead to further developments with respect to the imposition or threat of imposition of trade policies, tariffs (including retaliatory tariffs), taxes and other limitations on cross-border operations. Such tensions could adversely impact us and our third-party manufacturers.
Any product for which we have obtained regulatory approval, or 77 Table of Contents for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EMA and other comparable international regulatory authorities.
Any product for which we have obtained regulatory approval, or for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EMA and other comparable international regulatory authorities.
On December 1, 2022, the FDA approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with a susceptible IDH1 mutations as detected by an FDA-approved test. REZLIDHIA is our second commercial product and we believe is highly synergistic with our existing hematology-oncology focused commercial and medical affairs infrastructure.
In December 2022, the FDA approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with a susceptible IDH1 mutations as detected by an FDA-approved test. REZLIDHIA is our second commercial product and we believe is highly synergistic with our existing hematology-oncology focused commercial and medical affairs infrastructure.
In addition, we cannot be sure that our existing insurance coverage will continue to be available on acceptable terms or that our insurers will not deny coverage as to any future claim. 70 Table of Contents Future equity issuances or a sale of a substantial number of shares of our common stock may cause the price of our common stock to decline.
In addition, we cannot be sure that our existing insurance coverage will continue to be available on acceptable terms or that our insurers will not deny coverage as to any future claim. Future equity issuances or a sale of a substantial number of shares of our common stock may cause the price of our common stock to decline.
Any changes to the orphan drug provisions could change our opportunities for, or likelihood of success in obtaining, orphan drug exclusivity and would materially adversely affect our business, financial condition, results of operations, cash flows and prospects. Risks Related to Commercialization Our prospects are highly dependent on our commercial products.
Any changes to the orphan drug provisions could change our opportunities for, or likelihood of success in obtaining, orphan drug exclusivity and would materially adversely affect our business, financial condition, results of operations, cash flows and prospects. 76 Table of Contents Risks Related to Commercialization Our prospects are highly dependent on our commercial products.
Also, many of our competitors are large pharmaceutical companies that will have a greater ability to reduce prices for their competing drugs in an effort to gain market share and undermine the value proposition that we might otherwise be able to offer to payors.
Also, many of our 60 Table of Contents competitors are large pharmaceutical companies that will have a greater ability to reduce prices for their competing drugs in an effort to gain market share and undermine the value proposition that we might otherwise be able to offer to payors.
In 2023, FDA published guidance documents and a final rule which all concern clinical trial requirements. In June 2023, FDA published a draft guidance, E6(R3) Good Clinical Practice, which seeks to unify standards for clinical trial data for the International Council for 85 Table of Contents Harmonisation of Technical Requirements of Pharmaceuticals for Human Use member countries and regions.
In 2023, FDA published guidance documents and a final rule which all concern clinical trial requirements. In June 2023, FDA published a draft guidance, E6(R3) Good Clinical Practice, which seeks to unify standards for clinical trial data for the International Council for Harmonisation of Technical Requirements of Pharmaceuticals for Human Use member countries and regions.
We cannot completely eliminate the risk of accidental contamination or injury from the use, storage, handling or disposal of these animals and materials. In the event of contamination or injury, we could be held liable for damages that result or for penalties or fines that may be imposed, and such liability could exceed our resources.
We cannot completely eliminate the risk of accidental contamination or injury from the use, storage, handling or disposal of these animals and materials. In the event of contamination or injury, we could be held liable for damages that result or for penalties or fines that may be 64 Table of Contents imposed, and such liability could exceed our resources.
In addition, utilization of NOLs to offset potential future taxable income and related income taxes that would otherwise be due is subject to annual limitations under the “ownership change” provisions of Sections 382 and 383 of the Code and similar state provisions, which may result in the expiration of NOLs before future utilization.
In addition, utilization of NOLs to offset potential future taxable income and related income taxes that would otherwise be due is subject to annual limitations under the “ownership change” provisions of Sections 382 and 383 of the Section 174 and similar state provisions, which may result in the expiration of NOLs before future utilization.
Additionally, we have limited experience in conducting and managing the clinical trials necessary to obtain regulatory approval. If regulatory approval of a product is granted, this approval will be limited to those indications or disease states and conditions for which the product is demonstrated through clinical trials to be safe and efficacious.
Additionally, we have limited experience in conducting and managing the clinical trials necessary to obtain regulatory approval. 75 Table of Contents If regulatory approval of a product is granted, this approval will be limited to those indications or disease states and conditions for which the product is demonstrated through clinical trials to be safe and efficacious.
Generally, in place of such 49 Table of Contents clinical studies, an ANDA applicant usually needs only to submit data demonstrating that its product has the same active ingredient(s), strength, dosage form and route of administration and that it is bioequivalent to the branded product.
Generally, in place of such clinical studies, an ANDA applicant usually needs only to submit data demonstrating that its product has the same active ingredient(s), strength, dosage form and route of administration and that it is bioequivalent to the branded product.
We rely on third parties to, among other things, manufacture and ship our commercial product, raw materials and product supply for our clinical trials, perform quality testing and supply other goods and services to help manage our commercial activities, our clinical trials and our operations in the ordinary course of business.
We rely on third parties to, among other things, manufacture and ship our commercial product, raw materials and product supply for our clinical trials, perform quality testing and supply other goods and services to help manage our commercial activities, our 49 Table of Contents clinical trials and our operations in the ordinary course of business.
Market acceptance of our products will depend on a number of factors, including: ● the timing of market introduction of the product as well as competitive products; ● the clinical indications for which the product is approved; ● acceptance by physicians, the medical community and patients of the product as a safe and effective treatment; ● potential future impacts, if any, due to the effects of a global pandemic and the global tensions arising from 81 Table of Contents the Russian-Ukrainian war and Hamas-Israel war; ● the ability to distinguish safety and efficacy from existing, less expensive generic alternative therapies, if any; ● the convenience of prescribing, administrating and initiating patients on the product and the length of time the patient is on the product; ● the potential and perceived value and advantages of the product over alternative treatments; ● the cost of treatment in relation to alternative treatments, including any similar generic treatments; ● pricing and the availability of coverage and adequate reimbursement by third-party payors and government authorities; ● a positive HTA concluding that the product is cost-effective and the HTA bodies issuing a positive recommendation for the use of the product as a first or second line of treatment for the granted therapeutic indication; ● the prevalence and severity of adverse side effects; and ● the effectiveness of sales and marketing efforts.
Market acceptance of our products will depend on a number of factors, including: • the timing of market introduction of the product as well as competitive products; • the clinical indications for which the product is approved; • acceptance by physicians, the medical community and patients of the product as a safe and effective treatment; • potential future impacts, if any, due to the effects of a global pandemic and the global geopolitical tensions; • the ability to distinguish safety and efficacy from existing, less expensive generic alternative therapies, if any; • the convenience of prescribing, administrating and initiating patients on the product and the length of time the patient is on the product; • the potential and perceived value and advantages of the product over alternative treatments; • the cost of treatment in relation to alternative treatments, including any similar generic treatments; • pricing and the availability of coverage and adequate reimbursement by third-party payors and government authorities; • a positive HTA concluding that the product is cost-effective and the HTA bodies issuing a positive recommendation for the use of the product as a first or second line of treatment for the granted therapeutic indication; 77 Table of Contents • the prevalence and severity of adverse side effects; and • the effectiveness of sales and marketing efforts.
Any de-prioritization of our clinical trials or delay 52 Table of Contents in regulatory review resulting from such disruptions could materially affect the completion of our clinical trials. In addition, as seen in the COVID-19 pandemic, pandemics could result in a significant disruption of global financial markets.
Any de-prioritization of our clinical trials or delay in regulatory review resulting from such disruptions could materially affect the completion of our clinical trials. In addition, as seen in the COVID-19 pandemic, pandemics could result in a significant disruption of global financial markets.
We may seek another collaborator or licensee in the future for clinical development and commercialization of our products, as well as our other 58 Table of Contents clinical programs, which we may not be able to obtain on commercially reasonable terms or at all.
We may seek another collaborator or licensee in the future for clinical development and commercialization of our products, as well as our other clinical programs, which we may not be able to obtain on commercially reasonable terms or at all.
Our network and storage applications and those of our contract manufacturing organizations, collaborators, contractors, CROs or vendors may be subject to unauthorized access or processing by hackers or breached due to 69 Table of Contents operator or other human error, theft, malfeasance or other system disruptions.
Our network and storage applications and those of our contract manufacturing organizations, collaborators, contractors, CROs or vendors may be subject to unauthorized access or processing by hackers or breached due to operator or other human error, theft, malfeasance or other system disruptions.
Accordingly, compliance 44 Table of Contents programs may require additional investment in resources, and could impact availability of previously useful data. Internationally, our operations abroad may also be subject to increased scrutiny or attention from foreign data protection authorities.
Accordingly, compliance programs may require additional investment in resources, and could impact availability of previously useful data. Internationally, our operations abroad may also be subject to increased scrutiny or attention from foreign data protection authorities.
In addition, the FDA may not approve any IND we or our collaborative partners may submit in a timely manner, or at all. 79 Table of Contents Before receiving FDA approval to market a product, we must demonstrate with substantial clinical evidence that the product is safe and effective in the patient population and the indication that will be treated.
In addition, the FDA may not approve any IND we or our collaborative partners may submit in a timely manner, or at all. Before receiving FDA approval to market a product, we must demonstrate with substantial clinical evidence that the product is safe and effective in the patient population and the indication that will be treated.
The process of obtaining regulatory approvals in the US and other foreign jurisdictions is expensive, and lengthy, if approval is obtained at all. 84 Table of Contents Our clinical trials may fail to produce results satisfactory to the FDA or regulatory authorities in other jurisdictions.
The process of obtaining regulatory approvals in the US and other foreign jurisdictions is expensive, and lengthy, if approval is obtained at all. Our clinical trials may fail to produce results satisfactory to the FDA or regulatory authorities in other jurisdictions.
Provisions of our Amended and Restated Certificate of Incorporation and our Amended and Restated Bylaws (our Bylaws), as well as provisions of Delaware law, could make it more difficult for a third party to acquire us, even if 87 Table of Contents doing so would benefit our stockholders.
Provisions of our Amended and Restated Certificate of Incorporation and our Amended and Restated Bylaws (our Bylaws), as well as provisions of Delaware law, could make it more difficult for a third party to acquire us, even if doing so would benefit our stockholders.
Product liability claims could divert management’s attention from our core business, be expensive to defend, and result in sizable damage awards against us that may not be covered by insurance. If we cannot successfully defend ourselves against claims that our products caused injuries, we will incur substantial liabilities.
Product liability claims could divert management’s attention from our core business, be expensive to defend, and result in sizable damage awards against us that may not be covered by insurance. If we cannot 48 Table of Contents successfully defend ourselves against claims that our products caused injuries, we will incur substantial liabilities.
In addition, acquisitions and in-licensing may entail numerous operational, financial and legal risks, including: ● potential failure of the due diligence process to identify significant problems, liabilities or other shortcomings or challenges of an acquired or licensed product candidate or technology, including problems, liabilities or other shortcomings or challenges with respect to intellectual property, product quality, partner disputes or issues and other legal and financial contingencies and known and unknown liabilities; ● inability to integrate the target company or in-licensed asset successfully into our existing business and inability to maintain the key business relationships of the target; 40 Table of Contents ● in an in-licensing or an asset acquisition of a product that is commercially available in the market, we may not be able to successfully transition the existing patients who are dependent to the acquired or in-licensed product, or successfully enter into a reimbursement coverage contracts that the existing patients were previously dependent into, or successfully enter into a contract with contract manufacturers to continue the production of the in-licensed or acquired product; ● assumption of unknown or contingent liabilities or incurrence of unanticipated expenses; ● exposure to known and unknown liabilities, including possible intellectual property infringement claims, violations of laws, tax liabilities and commercial disputes; ● incurrence of substantial debt, dilutive issuances of securities or depletion of cash to pay for acquisitions; ● incurrence of large one-time expenses and acquiring intangible assets that could result in significant future amortization expense and significant write-offs; ● higher than expected acquisition and integration costs; and ● inability to maintain uniform standards, controls, procedures and policies; There is a high risk that drug discovery and development efforts might not generate successful product candidates.
In addition, acquisitions and in-licensing may entail numerous operational, financial and legal risks, including: • potential failure of the due diligence process to identify significant problems, liabilities or other shortcomings or challenges of an acquired or licensed product candidate or technology, including problems, liabilities or other shortcomings or challenges with respect to intellectual property, product quality, partner disputes or issues and other legal and financial contingencies and known and unknown liabilities; • inability to integrate the target company or in-licensed asset successfully into our existing business and inability to maintain the key business relationships of the target; • in an in-licensing or an asset acquisition of a product that is commercially available in the market, we may not be able to successfully transition the existing patients who are dependent to the acquired or in-licensed product, or successfully enter into a reimbursement coverage contracts that the existing patients were previously dependent into, or successfully enter into a contract with contract manufacturers to continue the production of the in-licensed or acquired product; • assumption of unknown or contingent liabilities or incurrence of unanticipated expenses; • exposure to known and unknown liabilities, including possible intellectual property infringement claims, violations of laws, tax liabilities and commercial disputes; • incurrence of substantial debt, dilutive issuances of securities or depletion of cash to pay for acquisitions; 38 Table of Contents • incurrence of large one-time expenses and acquiring intangible assets that could result in significant future amortization expense and significant write-offs; • higher than expected acquisition and integration costs; and • inability to maintain uniform standards, controls, procedures and policies.
Our ability to develop our product candidates, and our ability to commercially supply our products will depend, in part, on our ability to successfully obtain the active pharmaceutical ingredients and other substances and materials used in our product candidates from third parties and to have finished products manufactured by third parties in accordance with regulatory requirements and in sufficient quantities for preclinical and clinical testing and commercialization.
Our ability to develop our product candidates, and our ability to commercially supply our products will depend, in part, on our ability to successfully obtain the APIs and other substances and materials used in our product candidates from third parties and to have finished products manufactured by third parties in accordance with regulatory requirements and in sufficient quantities for preclinical and clinical testing and commercialization.
Supreme Court issued an opinion holding that courts reviewing agency action pursuant to the Administrative Procedure Act (APA) “must exercise their independent judgment” and “may not defer to an agency interpretation of the law simply because a statute is ambiguous.” The decision will have a significant impact on how lower courts evaluate challenges to agency interpretations of law, including those by CMS and other agencies with significant oversight of the healthcare industry.
In addition, on June 28, 2024, the US Supreme Court issued an opinion holding that courts reviewing agency action pursuant to the Administrative Procedure Act (APA) “must exercise their independent judgment” and “may not defer to an agency interpretation of the law simply because a statute is ambiguous.” The decision will have a significant impact on how lower courts evaluate challenges to agency interpretations of law, including those by CMS and other agencies with significant oversight of the healthcare industry.
Significant delays in clinical testing could negatively impact our product development costs and timing. Our estimates regarding timing are based on a number of assumptions, including assumptions based on past experience with 74 Table of Contents our other clinical programs.
Significant delays in clinical testing could negatively impact our product development costs and timing. Our estimates regarding timing are based on a number of assumptions, including assumptions based on past experience with our other clinical programs.
We are also subject to the risk that a person or government could allege such fraud or other misconduct, even if none occurred. Efforts to ensure that our business arrangements will comply with applicable healthcare laws and regulations will involve substantial costs.
We are also subject to the risk that a person or government could allege such fraud or other misconduct, even if none occurred. Efforts to ensure that our business arrangements will comply with applicable healthcare laws and 40 Table of Contents regulations will involve substantial costs.
In June 2022, we announced top-line 41 Table of Contents efficacy and safety data results of the trial, and the results did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population .
In June 2022, we announced top-line efficacy and safety data results of the trial, and the results did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population .
Post-grant proceedings are complex and expensive legal proceedings and there is no assurance we will be successful in any such proceedings. A post-grant proceeding could result in our losing our patent rights and/or our freedom to operate and/or require us to pay significant royalties.
Post-grant proceedings are complex and expensive legal proceedings and there is no assurance we will be successful in any such proceedings. A post-grant proceeding could result in our losing our patent 56 Table of Contents rights and/or our freedom to operate and/or require us to pay significant royalties.
In certain cases (e.g., where transfers are made in reliance on EU SCCs) a company must also carry out a so-called transfer privacy impact assessment (TIA).
In certain cases (e.g., where transfers are made in reliance on EU standard contractual clauses (EU SCCs)) a company must also carry out a so-called transfer privacy impact assessment (TIA).
For example, during the COVID-19 pandemic, we observed reduced patient-doctor interactions and our representatives had fewer visits with health care providers, which negatively affected our product sales.
For example, during the COVID-19 pandemic, we observed reduced patient-doctor interactions and our representatives had fewer visits with healthcare providers, which negatively affected our product sales.
Thus, there can be no assurance that we will be able to identify all factors that may cause our discount and rebate payment obligations to vary from period to period, and our actual results may differ significantly from our estimated allowances for discounts and rebates.
Thus, there can be no assurance that we will be able to identify all factors that may cause our discount and rebate payment obligations to vary from period 50 Table of Contents to period, and our actual results may differ significantly from our estimated allowances for discounts and rebates.
We have consumed substantial amounts of capital to date as we continue our research and development activities, including preclinical studies and clinical trials and for the commercialization of our products.
We have consumed substantial amounts of capital to date as we continue our research and development activities, including preclinical studies and clinical trials and for the commercialization of our products, as well as corporate development opportunities.
These discovery efforts may initially show promise in identifying potential product candidates, yet ultimately fail to yield product candidates for clinical development for a number of reasons.
These discovery efforts may initially show promise in identifying potential product candidates, yet ultimately fail to yield product candidates for clinical 37 Table of Contents development for a number of reasons.
Any drugs resulting from our research and development efforts, or from our joint efforts with our existing or future collaborative partners, might not be able to compete successfully with competitors’ existing or future products or obtain regulatory approval in the US or elsewhere.
Any drugs resulting from our research and development efforts, or from our joint efforts with our existing or future collaborative 61 Table of Contents partners, might not be able to compete successfully with competitors’ existing or future products or obtain regulatory approval in the US or elsewhere.
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