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The principal factors and uncertainties that make investing in shares of our common stock risky include, among others:
+Added: Risks Related to Our Financial Position, Need for Additional Capital and Limited Operating History
● We have a limited operating history and while we are moving toward becoming a commercial-ready biopharmaceutical company, some of our product candidates are early in development and we have no products approved for commercial sale, which may make it difficult for you to evaluate our current business and likelihood of success and future viability.
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● Our ability to generate revenue and achieve profitability depends significantly on our ability to achieve several objectives relating to the discovery, development and commercialization of our product candidates, if approved.
−Removed: ● We will require substantial additional capital to finance our operations.
+Added: ● We will require substantial additional capital to finance our operations, which may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce and/or eliminate one or more of our research and drug development programs or future commercialization efforts.
−Removed: ● Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
+Added: Risks Related to the Discovery, Development and Commercialization of Our Product Candidates
● We are substantially dependent on the success of our lead product candidates, sevasemten and EDG-7500 .
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● Clinical drug development involves a lengthy and expensive process with an uncertain outcome.
−Removed: The clinical trials of our product candidates may not demonstrate safety and efficacy to the satisfaction of the FDA, European Medicines Agency (EMA) or other comparable foreign regulatory authorities or otherwise produce positive results and the results of preclinical studies and early clinical trials may not be predictive
−Removed: of future results.
+Added: The clinical trials of our product candidates may not demonstrate safety and efficacy to the satisfaction of the FDA, European Medicines Agency (EMA) or other comparable foreign regulatory authorities or otherwise produce positive results and the results of preclinical studies and early clinical trials may not be predictive of future results.
We may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
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Because the target patient populations of our programs are small and the addressable patient population may be even smaller, we must be able to successfully identify patients and capture a significant market share to achieve profitability and growth.
+Added: Risks Related to Regulatory Approval and Other Legal Compliance Matters
● The regulatory approval processes of the FDA, EMA and other comparable foreign regulatory authorities are lengthy, time consuming and inherently unpredictable.
If we are ultimately unable to obtain regulatory approval of our product candidates, we will be unable to generate product revenue and our business will be substantially harmed.
+Added: Risks Related to Employee Matters, Managing Our Growth and Other Risks Related to Our Business
● Our success is highly dependent on our ability to attract and retain highly skilled executive officers and employees.
+Added: Risks Related to Our Intellectual Property
● Our success depends on our ability to protect our intellectual property and our proprietary technologies.
+Added: Risks Related to Our Dependence on Third Parties
● We rely, and expect to continue to rely, on third parties to conduct our clinical trials and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials, research and studies, which may harm our business.
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We have a limited operating history and while we are moving toward becoming a commercial-ready biopharmaceutical company, some of our product candidates are early in development and we have no products approved for commercial sale, which may make it difficult for you to evaluate our current business and likelihood of success and future viability.
−Removed: We are a clinical stage biopharmaceutical company with a limited operating history upon which you can evaluate our business and prospects.
+Added: We are a late-stage clinical biopharmaceutical company with a limited operating history upon which you can evaluate our business and prospects.
We are developing precision medicines for rare neuromuscular diseases which is an unproven and highly uncertain undertaking and involves a substantial degree of risk.
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As a result, it may be more difficult for investors to accurately predict our likelihood of success and viability than it could be if we had a longer operating history.
−Removed: In addition, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors and risks frequently experienced by clinical-stage biopharmaceutical companies in rapidly evolving fields.
+Added: In addition, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors and risks frequently experienced by clinical-stage biopharmaceutical companies in rapidly
+Added: evolving fields.
As we continue moving toward commercialization, we will need to transition from a company with a research and development focus to a company capable of supporting commercial activities.
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We have incurred significant net losses since our inception, have not generated any revenue to date and have financed our operations principally through private placements of our convertible preferred stock and public offerings of our common stock.
−Removed: Our net loss was $40.7 million and $117.6 million for the three and nine months ended September 30, 2025, respectively.
−Removed: As of September 30, 2025, we had an accumulated deficit of $496.2 million.
+Added: Our net loss was $49.0 million for the three months ended March 31, 2026.
+Added: As of March 31, 2026, we had an accumulated deficit of $595.4 million.
We are advancing sevasemten, EDG-7500, and EDG-15400 in clinical development.
Our other programs, including EDG-003, are in preclinical discovery and research stages.
−Removed: As a result, we expect that it will be several years, if ever, before we receive approval to commercialize a product and generate revenue from product sales.
+Added: As a result, we expect that it will be a couple of years, if ever, before we receive approval to commercialize a product and generate revenue from product sales.
Even if we succeed in receiving marketing approval for and commercializing one or more of our approved product candidates, we expect that we will continue to incur substantial research and development and other expenses in order to discover, develop and market additional potential products.
We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future.
+Added: For example, we anticipate that our general and administrative expenses will increase in the future as we scale our organization to support clinical advancement, regulatory readiness, and future commercial planning activities.
The net losses we incur may fluctuate significantly from quarter to quarter such that a period-to-period comparison of our results of operations may not be a good indication of our future performance, particularly since we expect our expenses to increase if and when our product candidates progress through clinical development as product candidates in later stages of clinical development generally have higher development costs than those in earlier stages, primarily due to the increased size and duration of later-stage clinical trials.
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Our business depends entirely on the successful discovery, development, regulatory approval and commercialization of product candidates.
−Removed: We have no products approved for commercial sale and do not anticipate generating any revenue from product sales for the next several years, if ever.
+Added: We have no products approved for commercial sale and do not anticipate generating any revenue from product sales for the next couple of years, if ever.
Our ability to generate revenue and achieve profitability depends significantly on our ability, or any future collaborator’s ability, to achieve several objectives, including:
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Changes in the manufacturing process or facilities will require further comparability analysis and approval by the FDA before implementation, which could delay our clinical trials and product candidate development, and could require additional clinical trials, including bridging studies, to demonstrate consistent and continued safety and efficacy.
−Removed: We will require substantial additional capital to finance our operations.
+Added: We will require substantial additional capital to finance our operations, which may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce and/or eliminate one or more of our research and drug development programs or future commercialization efforts.
−Removed: As of September 30, 2025, we had $563.3 million in cash, cash equivalents and marketable securities.
+Added: As of March 31, 2026, we had $499.6 million in cash, cash equivalents and marketable securities.
We expect our current cash, cash equivalents and marketable securities will be sufficient to fund our current operating plan for at least the next 12 months.
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We also expect to incur costs associated with operating as a public company.
−Removed: Our cash, cash equivalents and marketable securities will not be sufficient for us to fund any of our product candidates through regulatory approval, and we will need to raise additional capital to complete the development and commercialization of our products.
+Added: Our cash, cash equivalents and marketable securities will not be sufficient for us to fund any of our product candidates through regulatory approval, and we will need to raise additional capital
+Added: to complete the development and commercialization of our products.
Accordingly, we will need to obtain substantial additional funding in order to continue our operations.
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In the event that we would need to obtain additional funding, our ability to raise or access capital may be affected by macroeconomic events and disruptions to the U.S.
−Removed: banking and financial sectors.
+Added: banking and financial sectors and adequate financing may not be available to us on acceptable terms, or at all.
Failures of banks and other financial institutions, such as Silicon Valley Bank in March 2023, or issues in the broader U.S.
−Removed: financial system may impact the broader capital markets, and in turn, may impact our ability to access those markets.
+Added: financial system may impact the broader capital markets, and in turn, may impact our ability to access those
Further, a tightening of credit markets and lending standards could it make more difficult for us to raise capital through either debt or equity offerings on commercially reasonable terms or at all.
−Removed: Attempting to secure additional financing may divert our management from our day-to-day activities, which may adversely affect our ability to develop our product candidates.
−Removed: Our failure to raise capital as and when needed or on acceptable terms would have a negative impact on our financial condition and our ability to pursue our business strategy, and we may have to delay, reduce the scope of, suspend or eliminate one or more of our research-stage programs, clinical trials or future commercialization efforts.
−Removed: Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
−Removed: Until such time, if ever, as we can generate substantial revenues, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources, which may dilute our stockholders or restrict our operating activities.
−Removed: As summarized in the risk factor entitled, “ We will require substantial additional capital to finance our operations.
−Removed: If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce and/or eliminate one or more of our research and drug development programs or future commercialization efforts .”, we have previously raised capital under our shelf registration statement that was filed on April 1, 2022 with the SEC that became effective on May 5, 2022 and was amended on January 19, 2024.
−Removed: On May 10, 2024, we filed an automatic shelf registration statement on Form S-3ASR that allows us to undertake various equity and debt offerings and entered into the Leerink Sales Agreement under which we may offer and sell shares of common stock, having aggregate sales proceeds of up to $175.0 million from time to time, through the Leerink ATM.
−Removed: Pursuant to the automatic shelf registration statement, on April 3, 2025, we closed an underwritten registered direct offering of 9,935,419 shares of our common stock for net proceeds $187.1 million after deducting underwriting discounts and commissions and offering expenses.
−Removed: Adequate additional financing may not be available to us on acceptable terms, or at all.
+Added: If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms may include liquidation or other preferences that adversely affect your rights as a stockholder.
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If we raise additional funds through upfront payments or milestone payments pursuant to strategic collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us.
−Removed: If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
+Added: Until such time, if ever, as we can generate substantial revenues, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources, which may dilute our stockholders or restrict our operating activities.
+Added: Further, attempting to secure additional financing may divert our management from our day-to-day activities, which may adversely affect our ability to develop our product candidates.
+Added: Failure to raise capital as and when needed or on acceptable terms would have a negative impact on our financial condition and our ability to pursue our business strategy, and we may have to delay, reduce the scope of, suspend or eliminate one or more of our research-stage programs, clinical trials or future commercialization efforts.
Our ability to utilize our net operating loss carryforwards and certain other tax attributes to offset future taxable income may be limited.
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Similar rules may apply under state tax laws.
−Removed: We may have experienced such ownership changes in the
−Removed: past, and we may experience ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which are outside our control.
+Added: We may have experienced such ownership changes in the past, and we may experience ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which are outside our control.
We have not conducted any studies to determine annual limitations, if any, that could result from such changes in the ownership of our stock.
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Changes in tax laws, as well as other factors, could cause us to experience fluctuations in our tax obligations and effective tax rates and otherwise adversely affect our tax positions and/or our tax liabilities.
−Removed: For example, beginning in 2022, the Tax Act eliminated the option to deduct research and development expenditures currently and requires taxpayers to capitalize and amortize them over five or fifteen years, and this requirement may impact our effective tax rate and our cash tax liability in future years.
−Removed: On July 4, 2025, the U.S.
−Removed: federal tax legislation commonly referred to as the One Big Beautiful Bill Act (the “OBBB Act”) was enacted, which makes a number of changes to U.S.
−Removed: federal income tax law, including permanently suspending the requirement to capitalize and amortize domestic research and development expenditures and permitting such deductions on a current basis.
−Removed: We are currently evaluating the full impact of the OBBB Act on us.
−Removed: Further, many countries, and organizations such as the Organization for Economic Cooperation and Development have proposed implementing changes to existing tax laws, including a proposed 15% global minimum tax (Pillar Two) that has been implemented by several countries in 2024 and is being considered for implementation by other jurisdictions.
−Removed: However, on June 28, 2025, the G7 released a joint statement that it had reached an understanding with the United States for a side-by-side system based on certain accepted principles, including that U.S.-parented groups, such as ours, would be exempt from certain provisions of Pillar Two.
+Added: Further, many countries, and organizations such as the Organization for Economic Cooperation and Development (the OECD) have proposed implementing changes to existing tax laws, including a proposed 15% global minimum tax (Pillar Two) that has been implemented by several countries in 2024 and is being considered for implementation by other jurisdictions.
+Added: On January 5, 2026, the OECD announced a “side-by-side” elective safe harbor that exempts U.S.-parented multinational entities from some of the Pillar Two rules (including the 15% global minimum tax) for fiscal years beginning on or after January 1, 2026.
Any of these developments or changes in U.S.
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In addition, our clinical trial sites could experience delays in collecting, receiving, and analyzing data from patients enrolled in our clinical trial for sevasemten due to limited staff at such sites, limitation or suspension of on-site visits by patients, or patients’ reluctance to visit the clinical trial sites during a public health emergency.
−Removed: As a result, research and development expenses and general and administrative expenses may vary
−Removed: significantly if there is an increased impact from public health emergencies on the costs and timing associated with the conduct of our clinical trial and other related business activities.
+Added: As a result, research and development expenses and general and administrative expenses may vary significantly if there is an increased impact from public health emergencies on the costs and timing associated with the conduct of our clinical trial and other related business activities.
In the event of a public health emergency, we could be required to develop and implement additional clinical trial policies and procedures designed to help protect subjects from such diseases.
−Removed: During the COVID-19 pandemic, the FDA issued various COVID-19 related guidance documents for sponsors and manufacturers, many of which have expired or were withdrawn with the expiration of the COVID-19 public health emergency declaration on May 11, 2023, although some COVID-19 related guidance documents continue in effect.
−Removed: Any continued and prolonged public health crisis could have a material negative impact on our business, financial condition and operating results.
To the extent public health emergencies or outbreaks adversely affect our business, financial condition and operating results, it may also have the effect of heightening many of the risks described in this “Risk Factors” section.
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Sevasemten and EDG-7500 will require additional clinical development, expansion of manufacturing capabilities, marketing approval from government regulators, substantial investment and significant marketing efforts before we can generate any revenues from product sales.
−Removed: We are not permitted to market or promote sevasemten, EDG-7500 , or any other product candidate before we receive marketing approval from the FDA and comparable foreign regulatory authorities, and we may never receive such marketing approvals.
−Removed: While we announced topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker, data from MESA, an open label extension that is providing continued access to individuals with Becker, and for our multipart Phase 2 CIRRUS-HCM trial of EDG-7500, from Part A, the single-dose arm in patients with obstructive HCM (oHCM) and Part B and Part C in patients with oHCM and nonobstructive HCM (nHCM), the FDA may disagree with our interpretation of the data and may require additional clinical testing before we can seek regulatory approval and begin commercialization, if at all.
+Added: We are not permitted to market or promote sevasemten, EDG-7500 , EDG-15400, or any other product candidate before we receive marketing approval from the FDA and comparable foreign regulatory authorities, and we may never receive such marketing approvals.
+Added: While we announced topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker, data from MESA, an open label extension that is providing continued access to individuals with Becker, encouraging observations from the LYNX Phase 2 trial in participants with Duchenne across functional measures, initial results from the FOX Phase 2 trial in participants with Duchenne, and for our multipart Phase 2 CIRRUS-HCM trial of EDG-7500, from Part A, the single-dose arm in patients with obstructive HCM (oHCM) and Part B and Part C in patients with oHCM and nonobstructive HCM (nHCM), and interim safety results from ongoing Part D trial in patients with oHCM and nHCM, the FDA may disagree with our interpretation of the data and may require additional clinical testing before we can seek regulatory approval and begin commercialization, if at all.
The success of sevasemten and EDG-7500 will depend on several factors, including the following:
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Sevasemten is currently being studied in multiple Phase 2 clinical trials, EDG-7500 is currently in a multipart Phase 2 trial, and EDG-15400 is currently in a Phase 1 trial with healthy adults.
−Removed: However, research and
−Removed: development related to novel therapeutics is inherently risky.
+Added: However, research and development related to novel therapeutics is inherently risky.
A product candidate can unexpectedly fail at any stage of preclinical and/or clinical development.
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The outcome of preclinical studies and early-stage clinical trials may not be predictive of the success of later clinical trials.
−Removed: For example, the primary endpoint of the GRAND CANYON cohort may not be met even though the endpoint trended towards improvement as a secondary endpoint in the earlier cohorts of the CANYON trial, and the trends observed so far in the CANYON trial may not be seen and or may not be statistically significant in the GRAND CANYON cohort.
+Added: For example, the primary endpoint of the GRAND CANYON cohort may not be met even though the endpoint trended towards improvement as a secondary endpoint in the earlier cohorts of the CANYON trial, and the trends observed so far in the CANYON trial may not be seen and may not be statistically significant in the GRAND CANYON cohort.
In addition, our product candidates may fail to show the desired safety and efficacy in clinical development despite positive results in preclinical studies or having successfully advanced through initial clinical trials.
−Removed: We may also discover that the half-life of our product candidates renders them unsuitable for the therapeutic applications we
+Added: We may also discover that the half-life of our product candidates renders them unsuitable for the therapeutic applications we have chosen.
As a result, we cannot assure you that any clinical trials that we conduct will demonstrate consistent or adequate efficacy and safety to support marketing approval.
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We have experienced delays in completing our clinical trials and may experience additional delays in initiating or completing additional clinical trials.
−Removed: We may also experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent receipt of marketing approval or our ability to commercialize our product candidates, including:
+Added: We may also experience numerous unforeseen events during, or
+Added: as a result of, clinical trials that could delay or prevent receipt of marketing approval or our ability to commercialize our product candidates, including:
● receipt of feedback from regulatory authorities that requires us to modify the design of our clinical trials;
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● selection of clinical end points that require prolonged periods of clinical observation or analysis of the resulting data;
−Removed: ● a facility manufacturing our product candidates or any of their components being ordered by the FDA or comparable foreign regulatory authorities to temporarily or permanently shut down due to violations of
−Removed: cGMPs , regulations or other applicable requirements, or infections or cross-contaminations of product candidates in the manufacturing process;
+Added: ● a facility manufacturing our product candidates or any of their components being ordered by the FDA or comparable foreign regulatory authorities to temporarily or permanently shut down due to violations of cGMPs , regulations or other applicable requirements, or infections or cross-contaminations of product candidates in the manufacturing process;
● any changes to our manufacturing process that may be necessary or desired;
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● third-party contractors not performing data collection or analysis in a timely or accurate manner;
−Removed: ● interruptions resulting from geo-political actions, including war, such war and instability in the Middle East and other regional or geo-political conflicts given that the Company conducts trials internationally, including in Israel and certain countries in Europe and Australasia;
+Added: ● interruptions resulting from geo-political actions, including war, such as war and instability in the U.S., Iran, Israel and Lebanon and other regional or geo-political conflicts given that the Company conducts trials internationally, including in Israel and certain countries in Europe and Australasia;
● third-party contractors becoming debarred or suspended or otherwise penalized by the FDA or other government or regulatory authorities for violations of regulatory requirements, in which case we may need to find a substitute contractor, and we may not be able to use some or all of the data produced by such contractors in support of our marketing applications;
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In addition, many of the factors that cause, or lead to, termination or suspension of, or a delay in the commencement or completion of, clinical trials may also ultimately lead to the denial of regulatory approval of a product candidate.
−Removed: Any delays to our clinical trials that occur as a result could shorten any period during which we
−Removed: may have the exclusive right to commercialize our product candidates and our competitors may be able to bring products to market before we do, and the commercial viability of our product candidates could be significantly reduced.
+Added: Any delays to our clinical trials that occur as a result could shorten any period during which we may have the exclusive right to commercialize our product candidates and our competitors may be able to bring products to market before we do, and the commercial viability of our product candidates could be significantly reduced.
Any of these occurrences may harm our business, financial condition and prospects significantly.
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In addition, our product candidates may be used in combination with certain other therapies, including corticosteroids, which may have undesirable side effects.
−Removed: If our product candidates are associated with undesirable side effects or have unexpected characteristics in preclinical studies or clinical trials when used alone or in combination with other approved products or investigational new drugs we may need to interrupt, delay or abandon their development or limit development to more narrow uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe or more acceptable from a risk-benefit perspective.
+Added: If our product candidates are associated with undesirable side effects or have unexpected characteristics in preclinical studies or clinical trials when used alone or in combination with other approved products or investigational new drugs we may need to interrupt, delay or abandon their development or limit development to more narrow uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent,
+Added: less severe or more acceptable from a risk-benefit perspective.
Treatment-related side effects could also affect patient recruitment or the ability of enrolled subjects to complete the trial or result in potential product liability claims.
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Any of these developments could materially harm our business, financial condition and prospects.
−Removed: Further, if any of our product candidates obtains marketing approval, toxicities associated with such product candidates previously not seen during clinical testing may also develop after such approval and lead to a requirement to conduct additional clinical safety trials, additional contraindications, warnings and precautions
−Removed: being added to the drug label, significant restrictions on the use of the product or the withdrawal of the product from the market.
+Added: Further, if any of our product candidates obtains marketing approval, toxicities associated with such product candidates previously not seen during clinical testing may also develop after such approval and lead to a requirement to conduct additional clinical safety trials, additional contraindications, warnings and precautions being added to the drug label, significant restrictions on the use of the product or the withdrawal of the product from the market.
We cannot predict whether our product candidates will cause toxicities in humans that would preclude or lead to the revocation of regulatory approval based on preclinical studies or early stage clinical trials.
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Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy traits despite having progressed through preclinical studies and initial clinical trials.
−Removed: Although we have announced positive results from our preclinical studies and clinical trials, we do not know whether sevasemten , EDG-7500, or EDG-15400 will perform in current or future clinical trials as they have performed in preclinical studies or earlier clinical trials, nor do we know whether any product candidate in our EDG-003 cardiometabolic discovery program will perform in current or future preclinical studies or future clinical trials as it has in prior preclinical studies.
+Added: Although we have announced positive results from our preclinical studies and clinical trials, we do not know whether sevasemten, EDG-7500, or EDG-15400 will perform in current or future clinical trials as they have performed in
+Added: preclinical studies or earlier clinical trials, nor do we know whether any product candidate in our EDG-003 cardiometabolic discovery program will perform in current or future preclinical studies or future clinical trials as it has in prior preclinical studies.
For example, the primary endpoint of the GRAND CANYON cohort may not be met even though the endpoint trended towards improvement as a secondary endpoint in the earlier cohorts of the CANYON trial, and the trends observed so far in the CANYON trial may not be seen and or may not be statistically significant in the GRAND CANYON cohort.
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Our ability to enroll eligible patients may be limited or may result in slower enrollment than we anticipate.
−Removed: For sevasemten trials, we completed our open-label ARCH trial (single site) and our CANYON Phase 2 clinical trial (multiple sites), as well as completed enrollment of the DUNE Phase 2 exercise challenge study (single site), GRAND CANYON, the pivotal cohort of our Phase 2 CANYON clinical trial in individuals with Becker (multiple sites), and the LYNX and FOX Phase 2 studies in Duchenne (multiple sites).
−Removed: We have also completed enrollment for an industry-sponsored, global, prospective registry investigating the natural history of
−Removed: adults with Becker aged 18 years and older.
+Added: For sevasemten trials, we completed our open-label ARCH trial (single site), our CANYON Phase 2 clinical trial (multiple sites), and the DUNE Phase 2 exercise challenge study (single site), as well as completed enrollment of the GRAND CANYON, the pivotal cohort of our Phase 2 CANYON clinical trial in individuals with Becker (multiple sites), and the LYNX and FOX Phase 2 studies in Duchenne (multiple sites).
+Added: We have also completed enrollment for an industry-sponsored, global, prospective registry investigating the natural history of adults with Becker aged 18 years and older.
However, we may not be successful in achieving our goal of establishing natural history reference data points.
−Removed: For EDG-7500 trials, we completed the Phase 1 trial in healthy subjects and for CIRRUS-HCM, our multipart Phase 2 trial, we completed Part A, the single-dose arm in patients with oHCM, completed enrollment of Part B and Part C in patients with oHCM and nHCM, respectively, and initiated enrollment of Part D.
−Removed: However, we may experience difficulty with enrollment and/or maintenance of patients in Part B, Part C, or Part D of the CIRRUS-HCM trial.
+Added: For EDG-7500 trials, we completed the Phase 1 trial in healthy subjects and for CIRRUS-HCM, our multipart Phase 2 trial, we completed Part A, the single-dose arm in patients with oHCM and Part B and Part C in patients with oHCM and nHCM, respectively, as well as completed enrollment of Part D.
+Added: However, we may experience difficulty with enrollment and/or maintenance of patients in Part D of the CIRRUS-HCM trial.
Additionally, we initiated a Phase 1 trial of healthy adults with our product candidate EDG-15400 with future disease target of HFpEF.
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In addition, the process of finding and diagnosing subjects may prove costly.
−Removed: Further, the treating physicians in our clinical trials may also use their medical discretion in advising patients enrolled in our clinical trials to withdraw from our studies to try alternative therapies.
+Added: treating physicians in our clinical trials may also use their medical discretion in advising patients enrolled in our clinical trials to withdraw from our studies to try alternative therapies.
We expect patient enrollment to be affected because our competitors have ongoing clinical trials for programs that are under development for the same indications as our product candidates, and patients who would otherwise be eligible for our clinical trials could instead enroll in clinical trials of our competitors’ programs.
−Removed: Patient enrollment for our current or any future clinical trials may be affected by other factors, including:
+Added: Patient enrollment for our current or any future clinical trials has been and could in the future be affected by factors such as:
● size and nature of the patient population;
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Enrollment delays in our clinical trials may result in increased development costs for our product candidates and jeopardize our ability to obtain marketing approval for the sale of our product candidates.
−Removed: Furthermore, even if we are able to enroll a sufficient number of
−Removed: patients for our clinical trials, we may have difficulty maintaining participation in our clinical trials through the treatment and any follow-up periods.
+Added: Furthermore, even if we are able to enroll a sufficient number of patients for our clinical trials, we may have difficulty maintaining participation in our clinical trials through the treatment and any follow-up periods.
We have limited resources and are currently focusing the majority of our efforts on developing sevasemten and EDG-7500 for particular indications.
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Our resource allocation decisions may cause us to fail to capitalize on viable commercial drugs or profitable market opportunities.
−Removed: Our spending on current and future research and development activities for sevasemten , EDG-7500, EDG-15400, and our EDG-003 cardiometabolic discovery program may not yield any commercially viable products.
+Added: Our spending on current and future
+Added: research and development activities for sevasemten , EDG-7500, EDG-15400, and our EDG-003 cardiometabolic discovery program may not yield any commercially viable products.
If we do not accurately evaluate the commercial potential or target markets for sevasemten , EDG-7500, EDG-15400, or the product candidates we are currently researching, such as those from our EDG-003 cardiometabolic discovery program, we may relinquish valuable rights to our product candidates or programs through collaboration, licensing or other strategic arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate or program.
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In May 2024, Nippon Shinyaku Co.
−Removed: announced that no statistical significance was observed between the treatment group and the placebo group in VILTEPSO’s
−Removed: confirmatory study.
−Removed: This result may affect VILTEPSO’s accelerated FDA approval.
+Added: announced that no statistical significance was observed between the treatment group and the placebo group in VILTEPSO’s confirmatory study.
+Added: In November 2025, Sarepta announced that AMONDYS 45 and VYONDYS 53 missed their primary endpoint in the confirmatory study.
+Added: These results may affect these three drugs’ accelerated FDA approval.
In June 2022, PTC Therapeutics presented new topline results with Translarna (ataluren), for patients with nonsense mutation Duchenne, a subset of the disease that impacts between 10% and 15% of patients.
−Removed: It remains unclear if the data will lead to FDA approval of Translarna, for which the company resubmitted the NDA in October 2024.
Translarna has been conditionally approved in the European Union and Brazil for ambulatory patients aged 2 years and older with Duchenne resulting from a nonsense mutation in the dystrophin gene.
−Removed: However, in March 2025, the European Commission (the EC) adopted the negative opinions issued by the Committee for Medicinal Products for Human Use of the EMA for the renewal of conditional marketing authorization of Translarna.
−Removed: While this action effectively removes Translarna’s marketing authorization in the European Economic Area, individual countries within the EU can leverage existing legislations to allow continued use of Translarna.
−Removed: In June 2023, the FDA approved Sarepta’s Biologics License Application seeking accelerated approval of their microdystrophin gene therapy, Elevidys (delandistrogene moxeparvovec), for the treatment of ambulant individuals with Duchenne between the ages of four to five years.
+Added: However, in March 2025, the European Commission adopted the negative opinions issued by the Committee for Medicinal Products for Human Use of the EMA for the renewal of conditional marketing authorization of Translarna.
+Added: While this action effectively removes Translarna’s marketing authorization in the European Economic Area, individual countries within the EU can leverage existing legislation to allow continued use of Translarna.
+Added: In February 2026, PTC Therapeutics withdrew its application to the FDA for Translarna in nonsense mutation Duchenne after receiving feedback on its filing.
+Added: In June 2023, the FDA approved Sarepta’s Biologics License Application seeking accelerated approval of their microdystrophin gene therapy, Elevidys (delandistrogene moxeparvovec), for the treatment of ambulant
+Added: individuals with Duchenne between the ages of four to five years.
In June 2024, the FDA granted Elevidys full approval for the treatment of ambulatory individuals aged 4 years and older, and accelerated approval for the treatment of non-ambulatory individuals aged 4 years and older.
−Removed: Other companies focused on developing genetic based therapies for Duchenne that target dystrophin mechanisms include Solid Biosciences Inc., Genethon, Dyne Therapeutics, Avidity Biosciences, REGENXBIO, Wave Life Sciences, and Entrada Therapeutics.
−Removed: Satellos Bioscience, Inc.
−Removed: is developing an orally administered small molecule drug designed to address deficits in muscle repair and regeneration, and is conducting a Phase 1b trial in adult patients with Duchenne.
−Removed: Gene editing treatments that are in preclinical development are also being pursued by Vertex and Sarepta Therapeutics.
+Added: However, in November 2025, the FDA revised the Elevidys indication to limit to ambulatory individuals 4 years or older and added black box warnings about risks of acute and fatal liver injuries.
+Added: Other companies focused on developing genetic based therapies for Duchenne that target dystrophin mechanisms include Solid Biosciences Inc., Genethon, Dyne Therapeutics, Avidity Biosciences, a Novartis Company, REGENXBIO, Wave Life Sciences, and Entrada Therapeutics.
+Added: In September 2025, Avidity announced positive topline and functional Phase 1/2 data for del-zota, demonstrating a statistically significant increase in dystrophin in individuals with Duchenne amenable to exon 44 skipping.
+Added: In December 2025, Dyne announced top line Phase 1/2 data for zeleciment rostudirsen (z-rostudirsen) demonstrating a statistically significant increase in dystrophin in individuals with Duchenne amenable to exon 51 skipping.
+Added: Gene editing treatments that are in preclinical development are also being pursued by Vertex Pharmaceuticals and Sarepta.
We are also aware of several companies targeting non-dystrophin mechanisms for the treatment of Duchenne.
In March 2024, the FDA approved Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy in patients aged six years and older.
+Added: The European Commission granted Duvyzat a conditional approval in June 2025.
Moreover, in June 2021, Italfarmaco released top line Phase 2 data for givinostat in Becker.
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In June 2025, Capricor Therapeutics, Inc.
−Removed: announced that the FDA has granted ODD to Deramiocel, the company’s lead cell therapy candidate, for the potential treatment of BMD.
+Added: announced that the FDA has granted ODD to Deramiocel, the company’s lead cell therapy candidate, for the potential treatment of Becker, and this candidate is currently under regulatory review.
+Added: Satellos Bioscience, Inc.
+Added: is developing an orally administered small molecule drug designed to address deficits in muscle repair and regeneration and announced functional data from a Phase 1b trial in adult patients with Duchenne in May 2025.
With EDG-7500, we expect to face competition from existing products and products in development.
−Removed: Current pharmaceutical treatment is intended to improve diastolic filling in both oHCM and nHCM and reduce left ventricular outflow tract gradient in oHCM patients only.
−Removed: The goal of current therapies is to achieve meaningful symptom relief.
−Removed: Non-vasodilating beta blockers and non-dihydropyridine calcium channel blockers are the first-line therapies for symptomatic oHCM and nHCM patients.
+Added: Current first-line pharmaceutical treatment for patients with oHCM and nHCM consists of non-vasodilating beta blockers and non-dihydropyridine calcium channel blockers.
Commonly prescribed beta-blockers are atenolol, propranolol, and metoprolol.
Verapamil and diltiazem are calcium channel blockers used in the treatment of symptomatic oHCM and nHCM.
−Removed: For oHCM patients who do not respond to beta blocker therapy, disopyramide (either Norpace, marketed by Pfizer, or a generic form marketed by several other companies) and/or Camzyos (mavacamten), a cardiac myosin inhibitor (CMI), may also be added.
−Removed: In the field of emerging treatments for HCM, competitors include Bristol-Myers Squibb (BMS), Cytokinetics, Imbria Pharmaceuticals, Lexicon, and Celltrion.
−Removed: Cytokinetics is developing a CMI, aficamten (CK-274), for which positive oHCM Phase 3 results were announced in 2023.
−Removed: In December 2024, the FDA accepted the aficamten New Drug Application (NDA) with a Prescription Drug User Fee Act (PDUFA) target action date recently delayed from the original date of September 26, 2025 to December 26, 2025.
−Removed: In June 2023, Cytokinetics initiated another Phase 3 active-comparator clinical trial of aficamten compared to metoprolol in symptomatic oHCM patients.
+Added: For oHCM patients who remain symptomatic, a sodium channel blocker with negative ionotropic drug properties may also be added, typically disopyramide (either Pfizer’s Norpace, marketed by Pfizer, or a generic form marketed by several companies) and/or Camzyos (mavacamten), a cardiac myosin inhibitor (CMI), may also be added.
+Added: In the field of emerging treatments for HCM, competitors include Bristol-Myers Squibb (BMS), Cytokinetics, Imbria Pharmaceuticals, Lexicon Pharmaceuticals, and Braveheart Bio.
+Added: BMS markets Camzyos (mavacamten), a CMI intended for the treatment of adults with symptomatic NYHA class II-III oHCM.
+Added: To date, Camzyos (mavacamten) has secured marketing approvals in the US, Europe, and other countries across five continents.
+Added: In December 2025, Cytokinetics received approval from the FDA and the China National Medical Products Administration for the treatment of oHCM for its CMI aficamten, marketed as Myqorzo.
+Added: Myqorzo became available in the US in January 2026.
+Added: In February 2026, Cytokinetics received approval from the European Commission for the treatment of NYHA class II-III oHCM in adult patients.
In the second quarter of 2024, BMS and Cytokinetics initiated a study of mavacamten and aficamten, respectively, in pediatric population with symptomatic oHCM.
−Removed: Cytokinetics is also exploring its CMI in an ongoing Phase 3 nHCM clinical trial.
+Added: Positive results from the BMS pediatric study were reported at the American College of Cardiology conference in March 2026.
In April 2025, BMS reported that its Phase 3 study of mavacamten in nHCM failed to meet its dual primary endpoints.
−Removed: Non-myosin targeting drugs in development include ninerafaxstat/IMB-101(Imbria Pharmaceuticals), a partial fatty acid oxidation (pFOX) inhibitor, CT-G20 (Celltrion), an anti-arrhythmic cibenzoline succinate, and trientine dihydrochloride (Univar Solutions), a selective copper II chelator.
−Removed: In November 2023, Imbria announced Phase 2 nHCM topline results of ninerafaxstat, and in the second quarter of 2025, initiated a Phase 2b nHCM trial of ninerafaxstat.
−Removed: In the third quarter of 2024, Lexicon Pharmaceuticals initiated a Phase 3 trial of sotagliflozin, an SGLT1 and SGLT2 inhibitor,
−Removed: in patients with symptomatic oHCM and nHCM.
−Removed: We have limited knowledge of CT-G20’s Phase 1 oHCM trial status, while the trientine Phase 2 oHCM clinical trial is ongoing.
+Added: In May 2026, Cytokinetics announced positive topline results from its Phase 3 trial of aficamten in patients with symptomatic nHCM meeting its dual primary endpoints.
+Added: Lexicon Pharmaceuticals is currently conducting a Phase 3 study of Sotagliflozin, an SGLT 1/2 inhibitor, for the treatment of oHCM and nHCM.
+Added: In March 2026, Braveheart Bio announced positive results from its Phase 2 trial with HRS/BHB-1893 in oHCM and is planning to initiate a global Phase 3 clinical trial in oHCM in 2026.
+Added: Other drugs in development that do not target cardiac myosin include Imbria Pharmaceuticals’ ninerafaxstat (IMB-101), a partial fatty acid oxidation (pFOX) inhibitor, and Univar Solutions’ trientine dihydrochloride, a selective copper II chelator.
+Added: In November 2023, Imbria announced Phase 2 nHCM topline results of ninerafaxstat with full results published in March 2024, and in the second quarter of 2025, initiated a Phase 2b nHCM trial of ninerafaxstat.
+Added: In the third quarter of 2024, Lexicon Pharmaceuticals initiated a Phase 3 trial of sotagliflozin, an SGLT1 and SGLT2 inhibitor, in patients with symptomatic oHCM and nHCM.
+Added: We have limited knowledge of CT-G20’s Phase 1 oHCM trial status,
+Added: while the trientine Phase 2 oHCM clinical trial is ongoing.
A myosin binding protein C3-targeting gene therapy candidate, TN-201, is being developed by Tenaya Therapeutics for genetic HCM.
2 unchanged sentences
JN-210, a microRNA activating gene therapy approach being developed by Jaan Biotherapeutics;
−Removed: HTX-001, an antisense oligonucleotide approach being developed by Haya Therapeutics for the treatment of nHCM;
+Added: HTX-001, an antisense oligonucleotide approach being developed by Haya Therapeutics;
CDR348T and CDR641L, both are non-coding RNA-based therapies being developed by Cardior Pharmaceuticals (acquired by Novo Nordisk in May 2024).
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From time to time, we may publicly disclose preliminary, interim or topline data from our clinical trials.
−Removed: In sevasemten, on April 15, 2024, we announced positive two-year topline results from the ARCH open label trial of sevasemten in adults with Becker, on December 16, 2024, we announced positive topline data from the Phase 2 CANYON trial of sevasemten in individuals with Becker, and, on June 26, 2025, we announced encouraging topline data from our Phase 2 Duchenne trials, LYNX and FOX, and positive data from MESA, an open label extension trial that is providing continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, including the GRAND CANYON pivotal cohort, or DUNE .
−Removed: In EDG-7500, on September 19, 2024, we announced positive topline data from the Phase 1 trial of EDG-7500 in healthy subjects and the Part A single-dose arm of the Phase 2 multipart CIRRUS-HCM trial in patients with oHCM and, on April 2, 2025, we
−Removed: announced positive topline results from Part B and Part C of the Phase 2 multipart CIRRUS-HCM trial in patients with oHCM and nHCM.
+Added: In sevasemten, on April 15, 2024, we announced positive two-year topline results from the ARCH open label trial of sevasemten in adults with Becker, on December 16, 2024, we announced positive topline data from the Phase 2 CANYON trial of sevasemten in individuals with Becker, on June 26, 2025, we announced encouraging topline data from our Phase 2 Duchenne trials, LYNX and FOX, and positive data from MESA, an open label extension trial that is providing continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, including the GRAND CANYON pivotal cohort, or DUNE, and on March 10, 2026, we announced additional positive data from MESA.
+Added: In EDG-7500, on September 19, 2024, we announced positive topline data from the Phase 1 trial of EDG-7500 in healthy subjects and the Part A single-dose arm of the Phase 2 multipart CIRRUS-HCM trial in patients with oHCM, on April 2, 2025, we announced positive topline
+Added: results from Part B and Part C of the Phase 2 multipart CIRRUS-HCM trial in patients with oHCM and nHCM, and on December 24, 2025, we announced favorable interim safety results from Part D of the Phase 2 multipart CIRRUS-HCM trial in patients with oHCM and nHCM.
These interim updates are based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data related to the particular study or trial.
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If we fail to stay at the forefront of technological change in utilizing our proprietary drug discovery platform to create and develop product candidates, we may be unable to compete effectively.
−Removed: Our competitors may render our approach obsolete or limit the commercial value of our product candidates, by advances in existing technological approaches or the development of new or different approaches, potentially eliminating the
−Removed: advantages in our drug discovery process that we believe we derive from our research approach and proprietary technologies.
+Added: Our competitors may render our approach obsolete or limit the commercial value of our product candidates, by advances in existing
+Added: technological approaches or the development of new or different approaches, potentially eliminating the advantages in our drug discovery process that we believe we derive from our research approach and proprietary technologies.
By contrast, adverse developments with respect to other companies that attempt to use a similar approach to our approach may adversely impact the actual or perceived value of our proprietary drug discovery platform and potential of our product candidates.
58 unchanged sentences
A newborn screening initiative was put into place with the goal of identifying and providing care for every child born with Duchenne muscular dystrophy and achieving Recommended Uniform Screening Panel (RUSP) status.
−Removed: An Ohio newborn screening (NBS) program was announced in April 2024 in which all newborns in the state of Ohio are screened for Duchenne.
A newborn screening pilot program in New York State tested this and other aspects of a comprehensive newborn screening program at a large scale.
2 unchanged sentences
Two other pilot programs have been successfully conducted.
−Removed: In June 2022, Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne, announced that the organization submitted a nomination package to add Duchenne to the RUSP to the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), initiating the review process.
−Removed: The review process typically takes more than a year and requires two key votes of experts in NBS to move forward.
−Removed: In February 2023, the first key vote took place and the ACHDNC decided that more information was needed before proceeding to the second vote.
−Removed: The work to finalize the required additional evidence was completed and a meeting was scheduled with the ACHDNC for May 8, 2025.
−Removed: However, the current administration dissolved the ACHDNC before the meeting took place, leaving the process for submission and vote uncertain.
−Removed: The RUSP still exists, and advocates are currently seeking to understand the pathway to present the evidence package and receive approval to add Duchenne/Becker to the RUSP.
−Removed: Additionally, advocates at the state level are working to add Duchenne/Becker to their state level NBS programs.
+Added: An Ohio newborn screening (NBS) program was announced in April 2024 in which all newborns in the state of Ohio are screened for Duchenne.
+Added: In December 2025, the U.S.
+Added: Department of Health and Human Services (HHS) announced the addition of Duchenne to the RUSP.
+Added: Advocates at the state level are working to add Duchenne/Becker to their state level NBS programs.
Ohio and Minnesota are actively screening and Arizona, Massachusetts, and New York have passed legislation but screening has not started.
There are 12 states with pending legislation or committee hearings and 34 states with no activity.
−Removed: Uncertainties remain, including, among other things, ascertaining the pathway for additions to the RUSP and the adequacy of the evidence package.
−Removed: Furthermore, even if Duchenne is added to the RUSP, all states may not be able to effectively implement a NBS program.
+Added: Uncertainties remain and all states may not be able to effectively implement a NBS program.
This could reduce the identifiable patient population for the diseases we seek to treat and result in our therapies not being able to be initiated early in the course of the disease.
−Removed: Additionally, the potentially addressable patient population for each of our product candidates may be limited or may not be amenable to treatment with our product candidates, and new patients may become increasingly
−Removed: difficult to identify or gain access to, which would adversely affect our results of operations and our business.
+Added: Additionally, the potentially addressable patient population for each of our product candidates may be limited or may not be amenable to treatment with our product candidates, and new patients may become increasingly difficult to identify or gain access to, which would adversely affect our results of operations and our business.
Further, even if we obtain significant market share for our product candidates, because the potential target populations are very small, we may never achieve profitability despite obtaining such significant market share.
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We intend to evaluate select external opportunities to strategically expand our pipeline.
−Removed: While we plan to leverage our leadership team’s prior business development experience as we evaluate potential in-licensing and acquisition opportunities to expand our portfolio, we may not be able to identify suitable licensing or acquisition opportunities, and even if we do, we may not be able to successfully secure such licensing and acquisition opportunities.
+Added: While we plan to leverage our leadership team’s prior business development experience as we evaluate potential in-licensing and acquisition opportunities to expand our portfolio, we may not be able to identify suitable licensing or acquisition opportunities, and even if we do, we may not be able to successfully secure such licensing and acquisition
+Added: opportunities.
The licensing or acquisition of third-party intellectual property rights is a competitive area, and several more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary.
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There is significant uncertainty related to third-party payor coverage and reimbursement of newly approved products.
−Removed: In the United States, for example, principal decisions about reimbursement for new products are typically made by the Centers for Medicare & Medicaid Services (CMS), an agency within the U.S.
−Removed: Department of Health and Human Services (HHS).
+Added: In the United States, for example, principal decisions about reimbursement for new products are typically made by the Centers for Medicare & Medicaid Services (CMS), an agency within the HHS.
CMS decides whether and to what extent a new product will be covered and reimbursed under Medicare, and private third-party payors often follow CMS’s decisions regarding coverage and reimbursement to a substantial degree.
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Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to warn of dangers inherent in the product, negligence, strict liability, or a breach of warranties.
−Removed: Claims could also be asserted under
−Removed: state consumer protection laws.
+Added: Claims could also be asserted under state consumer protection laws.
If we cannot successfully defend ourselves against product liability claims, we may incur substantial liabilities or be required to limit testing and commercialization of our products.
22 unchanged sentences
Regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data are insufficient for approval and require additional preclinical, clinical or other data.
−Removed: Even if we eventually complete clinical testing and receive approval for our product candidates, the FDA, EMA and other comparable foreign regulatory authorities may
−Removed: approve our product candidates for a more limited indication or a narrower patient population than we originally requested or may impose other prescribing limitations or warnings that limit the product’s commercial potential.
+Added: Even if we eventually complete clinical testing and receive approval for our product candidates, the FDA, EMA and other comparable foreign regulatory authorities may approve our product candidates for a more limited indication or a narrower patient population than we originally requested or may impose other prescribing limitations or warnings that limit the product’s commercial potential.
We have not submitted for, or obtained, regulatory approval for any product candidate, and it is possible that none of our product candidates will ever obtain regulatory approval.
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FDA has also increased its scrutiny of foreign drug manufacturing facilities and other contractors based in China, especially with respect to the transfer of biological materials, genetic data, and other sensitive data of American patients to parties located in China.
−Removed: FDA’s “real-time” release of newly issued Complete Response Letters associated with withdrawn or abandoned applications, if applicable to any of our product candidates, can materially impact our competitive advantage and intellectual property.
+Added: “real-time” release of newly issued Complete Response Letters associated with withdrawn or abandoned applications, if applicable to any of our product candidates, can materially impact our competitive advantage and intellectual property.
It is unclear how our industry and our clinical programs will be impacted by policies and regulations implemented under the current administration and FDA commissioner, or other executive orders.
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536/2014 entered into application on January 31, 2022 and is intended to simplify the current rules for clinical trial authorization and standards of performance in EU.
−Removed: From January 31, 2025, any trials approved under the Clinical Trials Directive that continue running will need to comply with the Clinical Trials Regulation.
+Added: Any trials approved under the Clinical Trials Directive that continue running will need to comply with the Clinical Trials Regulation.
Complying with such new legislation or changes in healthcare regulation could be time-intensive and expensive, resulting in a material adverse effect on our business.
1 unchanged sentence
Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from those in the United States, including additional preclinical studies or clinical trials as clinical trials conducted in one jurisdiction may not be accepted by regulatory authorities in other jurisdictions.
−Removed: In many jurisdictions outside the United States, a product candidate
−Removed: must be approved for reimbursement before it can be approved for sale in that jurisdiction.
+Added: In many jurisdictions outside the United States, a product candidate must be approved for reimbursement before it can be approved for sale in that jurisdiction.
In some cases, the price that we intend to charge for our products is also subject to approval.
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Even after obtaining orphan drug designation, we may not be able to obtain or maintain orphan drug exclusivity for that product candidate.
−Removed: We may not be the first to obtain marketing approval of any product candidate for which we have obtained orphan drug designation for the orphan-designated indication due to the uncertainties associated with developing pharmaceutical products.
+Added: We may not be the first to obtain marketing approval of any product candidate for which we have obtained orphan drug designation
+Added: for the orphan-designated indication due to the uncertainties associated with developing pharmaceutical products.
In addition, exclusive marketing rights in the United States 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 we are unable to ensure that we will be able to manufacture sufficient quantities of the product to meet the needs of patients with the rare disease or condition.
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2021), in January 2023, the FDA published a notice in the Federal Register to clarify that while the agency complies with the court’s order in Catalyst , FDA intends to continue to apply its longstanding interpretation of the regulations to matters outside of the scope of the Catalyst order – that is, the agency will continue tying the scope of orphan-drug exclusivity to the uses or indications for which a drug is approved, which permits other sponsors to obtain approval of a drug for new uses or indications within the same orphan designated disease or condition that have not yet been approved.
−Removed: It is unclear how future litigation, including judicial challenges in view of the Supreme Court’s overturn of the Chevron doctrine legislation, agency decisions, and administrative actions under the current administration will impact the scope of the orphan drug exclusivity.
+Added: The Consolidated Appropriations Act of 2026, signed into law in February 2026, codified this longstanding FDA interpretation of the Orphan Drug Act, allowing the FDA to approve multiple versions of the same orphan drug for different subindications and subpopulations.
Where appropriate, we plan to secure approval from the FDA or comparable foreign regulatory authorities through the use of accelerated registration pathways.
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The accelerated approval pathway may be used in cases in which the advantage of a new drug over available therapy may not be a direct therapeutic advantage but is a clinically important improvement from a patient and public health perspective.
−Removed: If granted, accelerated approval is usually contingent on the sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify
−Removed: and describe the drug’s clinical benefit.
+Added: If granted, accelerated approval is usually contingent on the sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the drug’s clinical benefit.
If such post-approval studies fail to confirm the drug’s clinical benefit, the FDA may withdraw its approval of the drug.
1 unchanged sentence
Prior to seeking such accelerated approval, we will seek feedback from the FDA and will otherwise evaluate our ability to seek and receive such accelerated approval.
−Removed: For example, the FDA deemed the CANYON data alone insufficient for an accelerated approval of sevasemten.
+Added: For example, the FDA deemed the CANYON data alone
+Added: insufficient for an accelerated approval of sevasemten.
There can be no assurance that after our evaluation of the feedback and other factors we will decide to pursue or submit an NDA for accelerated approval or any other form of expedited development, review or approval.
8 unchanged sentences
We also cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative or executive action, either in the United States or abroad.
−Removed: For example, certain policies of the current U.S.
+Added: For example, certain policies of the U.S.
administration may impact our business and industry, which could impose significant burdens on, or otherwise materially delay, the FDA’s ability to engage in routine regulatory and oversight activities such as implementing statutes through rulemaking, issuance of guidance, and review and approval of marketing applications.
6 unchanged sentences
Accordingly, the ACA remains in effect in its current form.
−Removed: It is unclear how this Supreme Court decision, future litigation, or healthcare measures promulgated by the Biden administration will impact our business, financial condition and results of operations.
−Removed: On January 28, 2021, the Biden administration issued an executive order to initiate a special enrollment period for purposes of obtaining health insurance
−Removed: coverage through the ACA marketplace.
−Removed: The executive order also instructs certain governmental agencies to review and reconsider their existing policies and rules that limit access to healthcare, including among others, reexamining Medicaid demonstration projects and waiver programs that include work requirements, and policies that create unnecessary barriers to obtaining access to health insurance coverage through Medicaid or the ACA.
+Added: It is unclear how future litigation or healthcare measures promulgated by the government will impact our business, financial condition and results of operations.
Complying with any new legislation or changes in healthcare regulation could be time-intensive and expensive, resulting in a material adverse effect on our business.
2 unchanged sentences
In January 2013, President Obama signed into law the American Taxpayer Relief Act of 2012, which, among other things, reduced Medicare payments to several providers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.
−Removed: These laws may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on customers for our drugs, if approved, and accordingly, our financial operations.
+Added: These laws may result in
+Added: additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on customers for our drugs, if approved, and accordingly, our financial operations.
Moreover, there has been heightened governmental scrutiny recently over the manner in which drug manufacturers set prices for their marketed products, which has resulted in several congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.
1 unchanged sentence
Elimination of this cap may require pharmaceutical manufacturers to pay more in rebates than it receives on the sale of products, which could have a material impact on our business.
−Removed: In July 2021, the Biden administration released an executive order, “Promoting Competition in the American Economy,” with multiple provisions aimed at increasing competition for prescription drugs.
In August 2022, Congress passed the Inflation Reduction Act of 2022, which includes prescription drug provisions that have significant implications for the pharmaceutical industry and Medicare beneficiaries, including allowing the federal government to negotiate a maximum fair price for certain high-priced single source Medicare drugs, imposing penalties and excise tax for manufacturers that fail to comply with the drug price negotiation requirements, requiring inflation rebates for all Medicare Part B and Part D drugs, with limited exceptions, if their drug prices increase faster than inflation, and redesigning Medicare Part D to reduce out-of-pocket prescription drug costs for beneficiaries, among other changes.
3 unchanged sentences
For 2028, up to an additional 15 drugs, which may be covered under either Medicare Part B or Part D, will be selected, and for 2029 and subsequent years, up to 20 additional Part B or Part D drugs will be selected.
−Removed: Various industry stakeholders, including pharmaceutical companies, the U.S.
−Removed: Chamber of Commerce, and the Pharmaceutical Research and Manufacturers of America, have initiated lawsuits against the federal government asserting that the price negotiation provisions of the Inflation Reduction Act are unconstitutional.
+Added: Various industry stakeholders, including pharmaceutical companies and the Pharmaceutical Research and Manufacturers of America, have initiated lawsuits against the federal government asserting that the price negotiation provisions of the Inflation Reduction Act are unconstitutional.
Further, the current administration has issued executive orders focused on decreasing prescription drug prices, including directing the Secretary of Health and Human Services to establish a mechanism through which American patients can buy drugs directly from manufacturers who sell at a most-favored-nation price and directing the U.S.
Trade Representative and Secretary of Commerce to take action to ensure foreign countries are not engaged in practices that purposefully and unfairly undercut market prices and drive price hikes in the United States.
−Removed: In September and October 2025, the government announced the first two agreements with two major pharmaceutical companies to bring American drug prices in line with the lowest paid by other developed nations, requiring the companies to offer medicines at a deep discount off the list price when selling directly to American patients.
−Removed: Such agreements and other government measures that use most-favored-nation pricing targets for prescription drugs, including the use of international pricing reference to set drug prices in the United States, or increases generic and biosimilar drug entry sooner than expected, can have a material adverse effect on our industry, ability to set adequate pricing for new drugs to recover R&D costs, ability to attract potential investors and potential buyers in the future.
−Removed: Additionally, the OBBB Act includes provisions that will impact the United
−Removed: States healthcare system in various ways, including by cuts to Medicaid and introducing new participant work and eligibility requirements for Medicaid coverage, which are expected to significantly change the administration and applicability of Medicaid coverage.
+Added: In November 2025, CMS announced a voluntary initiative called the GENEROUS Model (GENErating cost Reductions fOr U.S.
+Added: Medicaid Model) to introduce the option of most-favored-nation pricing to the Medicaid program, whereby a drug manufacturer may voluntarily offer supplemental rebates to participating state Medicaid programs for a manufacturer’s covered outpatient drugs.
+Added: Government agreements with pharmaceutical companies and other measures that use most-favored-nation pricing targets for prescription drugs, including the use of international pricing reference to set drug prices in the United States, or that increase generic and biosimilar drug entry sooner than expected can have a material adverse effect on our industry, ability to set adequate pricing for new drugs to recover R&D costs, ability to attract potential investors and potential buyers in the future.
+Added: Additionally, the OBBB Act includes provisions that will impact the United States healthcare system in various ways, including by cuts to Medicaid and introducing new participant work and eligibility requirements for Medicaid coverage, which are expected to significantly change the administration and applicability of Medicaid coverage.
The OBBB Act also expanded the orphan drug exemptions under the Medicare Price Negotiation Program, including an amendment to exclude orphan designated drugs for one or more rare diseases or conditions, instead of only one disease/condition, with the initial price applicability year 2028 and after, from Medicare price negotiations, and providing that the time for measuring a former orphan drug’s eligibility for Medicare price negotiations will be calculated from the first day after the date of FDA approval for a non-orphan disease or condition, or an approval for which the drug does not have orphan drug designation.
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We cannot predict the full impact of the OBBB Act, executive orders, and new laws focused on reducing prescription drug prices or increasing domestic drug manufacturing capacity, or other measures that may be implemented by the current administration related to drug pricing, drug supply chain and manufacturing in the United States.
−Removed: The impact of ongoing and future judicial challenges as well as future legislative, executive, and administrative actions and any future healthcare measures and agency rules implemented by the current administration on us and the pharmaceutical industry as a whole is unclear.
+Added: The impact of ongoing and future judicial challenges as well as future legislative, executive, and administrative actions and any
+Added: future healthcare measures and agency rules implemented by the current administration on us and the pharmaceutical industry as a whole is unclear.
The implementation of cost containment measures, including the prescription drug provisions under the Inflation Reduction Act, as well as other healthcare reforms may prevent us from being able to generate revenue, attain profitability, or commercialize our product candidates if approved.
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These efforts may require substantial efforts and incurring significant costs.
−Removed: If our efforts to comply with the GDPR or other applicable EU laws and regulations are not successful, or are perceived to be unsuccessful, it could adversely affect our business in the EEA.
+Added: If our efforts to comply with the GDPR or other applicable EU laws and regulations are not successful, or are perceived to be unsuccessful, it could adversely affect our business in the EEA and elsewhere.
Further, in July 2020, the Court of Justice of the European Union (CJEU) issued a decision invalidating the EU-U.S.
−Removed: Privacy Shield, which had enabled the transfer of personal data from the EU to the U.S.
+Added: Privacy Shield, which had enabled the transfer
+Added: of personal data from the EU to the U.S.
for participating companies, and questioning the continued validity of the European Commission’s standard contractual clauses (SCCs).
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On June 28, 2021, the European Commission issued an adequacy decision in respect of the UK’s data protection framework, allowing personal data transfers from EU member states to the UK to continue without requiring additional contractual or other measures.
−Removed: This decision is subject to renewal and may be revisited by the European Commission at any time.
−Removed: The United Kingdom has implemented modifications to its data protection framework in the UK Data (Use and Access) Act 2025 (DUAA), which was enacted on June 19, 2025.
−Removed: These amendments may impact the European Commission’s decision with respect to its adequacy decision regarding the UK’s data protection regime.
−Removed: The European Commission has proposed to renew the UK’s adequacy decision after assessing the DUAA, but additional procedural steps remain, causing some uncertainty to remain regarding the UK’s adequacy determination.
+Added: This decision was renewed in December 2025 to extend until December 2031, but is subject to renewal and may be revisited by the European Commission at any time.
+Added: The United Kingdom has modified its data protection framework in the UK Data (Use and Access) Act 2025 (DUAA), which was enacted on June 19, 2025.
In the medium and longer terms, the relationship between the UK and EU in relation to aspects of data protection law remains unclear, which exposes us to further compliance risk.
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For example, the CCPA requires covered businesses that process personal information of California residents to disclose their data collection, use and sharing practices.
−Removed: Further, the CCPA provides California residents with certain data privacy rights (including the ability to opt out of certain disclosures of personal data), imposes operational requirements for covered businesses, provides for civil penalties for violations as well as a private right of action for data breaches and statutory damages (that is expected to increase data breach class action litigation and result in significant exposure to costly legal judgements and settlements).
+Added: Further, the CCPA provides California residents with certain data privacy rights (including the ability to opt out of certain disclosures of personal data), imposes operational requirements for covered businesses, provides for civil penalties for violations as well as a private right of action for data breaches and statutory damages (that is expected to increase data breach class action litigation and result in significant exposure to costly legal judgments and settlements).
Aspects of the CCPA and its interpretation and enforcement remain uncertain.
−Removed: In addition, the CCPA was expanded on January 1, 2023,
−Removed: when the California Privacy Rights Act of 2020 (CPRA) became operative.
+Added: In addition, the CCPA was expanded on January 1, 2023, when the California Privacy Rights Act of 2020 (CPRA) became operative.
The CPRA, among other things, gives California residents the ability to limit use of certain sensitive personal information, establishes restrictions on the retention of personal information, expand the types of data breaches subject to the CCPA’s private right of action, provides for increased penalties for CPRA violations concerning California residents under the age of 16, and establishes a California Privacy Protection Agency to implement and enforce the legislation.
Although there are limited exemptions for clinical trial data under the CCPA, the CCPA and other similar laws could impact our business activities, depending on their interpretation.
−Removed: Additionally, other state legislatures have enacted or are currently contemplating, and may pass, their own data privacy and security laws, with potentially greater penalties and more rigorous compliance requirements relevant to our business.
+Added: Additionally, numerous other state legislatures have enacted or are currently contemplating, and may pass, their own data privacy and security laws, with potentially greater penalties and more rigorous compliance requirements relevant to our business.
Many of these laws are comprehensive privacy statutes that impose obligations similar to the CCPA.
For example, Colorado enacted a Colorado Privacy Act (CPA) in June 2021 that went into effect on July 1, 2023, with enforcement commencing on the same date.
−Removed: The Colorado Attorney General released its rules implementing the CPA on March 15, 2023.
+Added: The Colorado Attorney General released its rules implementing the CPA on March 15, 2023, and since has amended these rules in 2024 and 2025.
Connecticut, Utah and Virginia have also enacted legislation similar to the CCPA and the CPA that took effect in 2023;
Florida, Montana, Oregon and Texas have enacted similar legislation that took effect in 2024;
−Removed: Delaware, Iowa, Minnesota, New Hampshire, New Jersey, Nebraska and Tennessee have enacted similar legislation that has taken effect in 2025;
−Removed: and Indiana, Kentucky and Rhode Island have enacted similar legislation that will take effect in 2026.
−Removed: government also has instituted new rules, effective April 8, 2025, that prohibit or restrict transactions involving certain types and amounts of sensitive data between U.S.
+Added: Delaware, Iowa, Maryland, Minnesota, New Hampshire, New Jersey, Nebraska and Tennessee have enacted similar legislation that took effect in 2025;
+Added: Indiana, Kentucky and Rhode Island have enacted similar legislation that has taken effect in 2026;
+Added: and Alabama and Oklahoma have enacted similar legislation that will take effect in 2027.
+Added: government also has instituted rules, effective April 8,
+Added: 2025, that prohibit or restrict transactions involving certain types and amounts of sensitive data between U.S.
persons and foreign persons associated with specific countries of concern, including China.
−Removed: Among other things, these new rules require U.S.
+Added: Among other things, these rules require U.S.
businesses to seek assurances from certain foreign parties with which they share sensitive data (under certain types of agreements) that those parties will not further share that data with parties in countries of concern.
+Added: Further, other states have enacted laws that cover certain aspects of the collection, use, disclosure, and/or other processing of health information, such as Washington’s My Health, My Data Act, which, among other things, provides for a private right of action.
Additionally, state and foreign laws may apply generally to the privacy and security of information we maintain, and may differ from each other in significant ways, thus complicating compliance efforts and potentially requiring us to undertake additional measures to comply with them.
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Any failure or perceived failure by us or our service providers to comply with our applicable policies or notices relating to privacy or data protection, our contractual or other obligations to third parties, or any of our other legal obligations relating to privacy or data protection, may result in governmental investigations or enforcement actions, litigation, claims and other proceedings, harm our reputation, and could result in significant liability.
−Removed: Further, other states have enacted laws that cover certain aspects of the collection, use, disclosure, and/or other processing of health information, such as Washington’s My Health, My Data Act, which, among other things, provides for a private right of action.
Inadequate funding for and other disruptions at the FDA, the SEC and other government agencies, including government shutdowns, could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
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Government shutdown, departures and other changes in the agency personnel at the FDA can materially impact the continuity of our correspondence with the FDA, delay our clinical trials, and the timing for submitting our applications for FDA approval.
−Removed: In addition, government funding of the U.S.
−Removed: Securities and Exchange Commission (SEC) and other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.
+Added: In addition, government funding of the SEC and other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.
Disruptions at the FDA and other agencies, including delays or disruptions due to government shutdown, lapse in U.S.
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● HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act (HITECH) and their implementing regulations, also imposes obligations, including mandatory contractual terms, on covered entities, which are health plans, healthcare clearinghouses, and certain health care providers, as those terms are defined by HIPAA, and their respective business associates and their subcontractors, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
−Removed: ● the federal Physician Payments Sunshine Act requires applicable manufacturers of covered drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to CMS information regarding payments and other transfers of value made to covered recipients, including physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), certain non-physician healthcare
−Removed: professionals (such as nurse practitioners and physician assistants, among others), and teaching hospitals as well as information regarding ownership and investment interests held by physicians;
+Added: ● the federal Physician Payments Sunshine Act requires applicable manufacturers of covered drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to CMS information regarding payments and other transfers of value made to covered recipients, including physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), certain non-physician healthcare professionals (such as nurse practitioners and physician assistants, among others), and teaching hospitals as well as information regarding ownership and investment interests held by physicians;
● analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers;
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state and local laws that require the registration of pharmaceutical sales and medical representatives;
−Removed: state laws that govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
+Added: state laws that govern the privacy and security of health
+Added: information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
Efforts to ensure that our current and future business arrangements with third parties will comply with applicable healthcare and data privacy laws and regulations will involve substantial ongoing costs and may require us to undertake or implement additional policies or measures.
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We have adopted a code of conduct but it is not always possible to identify and deter misconduct by these parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
−Removed: If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant penalties, including civil, criminal and administrative penalties, damages, fines, disgorgement, imprisonment, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, integrity oversight and
−Removed: reporting obligations, contractual damages, reputational harm, diminished profits and future earnings and the curtailment or restructuring of our operations.
+Added: If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant penalties, including civil, criminal and administrative penalties, damages, fines, disgorgement, imprisonment, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, integrity oversight and reporting obligations, contractual damages, reputational harm, diminished profits and future earnings and the curtailment or restructuring of our operations.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on our business.
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We generally contract with third parties for the disposal of these materials and wastes.
−Removed: We cannot eliminate the risk of contamination or injury from these materials.
+Added: We cannot eliminate the risk of contamination or injury from these
In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources.
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These laws also require that we make and keep books and records that accurately and fairly reflect the transactions of the corporation and to devise and maintain an adequate system of internal accounting controls and compliance procedures designed to prevent violations of anti-corruption laws.
−Removed: There is no certainty that all of our
−Removed: employees, agents, representatives, business partners and third-party intermediaries, or those of our affiliates, will comply with applicable laws and regulations, for which we may be ultimately held responsible.
+Added: There is no certainty that all of our employees, agents, representatives, business partners and third-party intermediaries, or those of our affiliates, will comply with applicable laws and regulations, for which we may be ultimately held responsible.
Violations of these laws and regulations could result in whistleblower complaints, fines, severe civil or criminal sanctions, settlements, prosecution, enforcement actions, damages, adverse media coverage, investigations, loss of export privileges, disgorgement, and other remedial measures and prohibitions on the conduct of our business including our ability to offer our products in one or more countries.
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In particular, the loss of one or more of our executive officers could be detrimental to us if we cannot recruit suitable replacements in a timely manner.
−Removed: We do not maintain “key person” insurance for any of our executives or other
+Added: We do not maintain “key person” insurance for any of our executives or other employees.
We could in the future have difficulty attracting and retaining experienced personnel and may be required to expend significant financial resources in our employee recruitment and retention efforts.
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Additionally, we rely on our scientific founders and other scientific and clinical advisors and consultants to assist us in formulating our research, development and clinical strategies.
−Removed: These advisors and consultants are not our employees and may have commitments to, or consulting or advisory contracts with, other entities that may limit their availability to us.
+Added: These advisors and consultants are not
+Added: our employees and may have commitments to, or consulting or advisory contracts with, other entities that may limit their availability to us.
In addition, these advisors and consultants typically will not enter into non-compete agreements with us.
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In order to successfully implement our plans and strategies, we will need to grow the size of our organization, and we may experience difficulties in managing this growth.
−Removed: As of September 30, 2025, we had 136 full-time employees.
+Added: As of March 31, 2026, we had 154 full-time employees.
Of these employees, 118 are engaged in research or product development and clinical activities.
−Removed: In order to successfully implement our development and commercialization plans and strategies, we expect to need additional managerial, operational, sales, marketing,
−Removed: financial and other personnel.
+Added: In order to successfully implement our development and commercialization plans and strategies, we expect to need additional managerial, operational, sales, marketing, financial and other personnel.
Future growth would impose significant added responsibilities on members of management, including:
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● improving our operational, financial and management controls, reporting systems and procedures.
−Removed: Our future financial performance and our ability to successfully develop and, if approved, commercialize sevasemten , EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program and other product candidates will depend, in part, on our ability to effectively manage any future growth, and our management may also have to divert a disproportionate amount of its attention away from day-to-day activities in order to devote a substantial amount of time to managing these growth activities.
+Added: Our future financial performance and our ability to successfully develop and, if approved, commercialize sevasemten , EDG-7500, EDG-15400, product candidates from our EDG-003 cardiometabolic discovery program
+Added: and other product candidates will depend, in part, on our ability to effectively manage any future growth, and our management may also have to divert a disproportionate amount of its attention away from day-to-day activities in order to devote a substantial amount of time to managing these growth activities.
We currently rely, and for the foreseeable future will continue to rely, in substantial part on certain independent organizations, advisors and consultants to provide certain services, including key aspects of our research and development, clinical development and manufacturing.
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We have incurred costs to establish a cybersecurity risk management program, including through the implementation of security measures in an effort to protect systems that store our information.
−Removed: However, despite the implementation of those security measures and the implementation of our cybersecurity risk management program, given the size and complexity and the increasing amounts of information maintained on our internal information technology systems, and those of our third-party CROs, other contractors (including sites performing our clinical trials), service providers, suppliers, and consultants, these systems are
−Removed: potentially vulnerable to breakdown or other damage or interruption from service interruptions, system malfunction, technical errors, natural disasters, terrorism, war and telecommunication and electrical failures, as well as security breaches and incidents from inadvertent or intentional actions by our employees, contractors, consultants, service providers, business partners, and/or other third parties, or from cyber-attacks by malicious third parties (including supply chain cyber-attacks or the deployment of harmful malware, ransomware, denial-of-service attacks, social engineering and other means to affect service reliability and threaten the confidentiality, integrity and availability of information), which may compromise the confidentiality, integrity, or availability of our information systems or the information contained therein, or otherwise availability or lead to the loss, destruction, alteration, prevention of access to, disclosure, or dissemination of, or damage or unauthorized access to, our data (including trade secrets or other confidential information, intellectual property, proprietary business information, and personal information) or data that is processed or maintained on our behalf, or other assets, which could result in financial, legal, business and reputational harm to us.
−Removed: We, and CROs that we work with, have experienced cybersecurity incidents in the past (including one of our CROS as recently as 2025) and we cannot eliminate the risk that we and our CROs will experience them in the future.
−Removed: For example, in 2019, one of our CROs experienced a cybersecurity breach which resulted in unauthorized access to certain of our preclinical data.
−Removed: Additionally, in 2023, one of our CROs experienced a cyber-attack for which an investigation found that no unauthorized access to Edgewise data occurred in connection with this event.
+Added: However, despite the implementation of those security measures and the implementation of our cybersecurity risk management program, given the size and complexity and the increasing amounts of information maintained on our information technology systems, and those of our third-party CROs, other contractors (including sites performing our clinical trials), service providers, suppliers, and consultants, these systems are potentially vulnerable to breakdown or other damage or interruption from service interruptions, system malfunction, technical errors, natural disasters, terrorism, war and telecommunication and electrical failures, as well as security breaches and incidents from inadvertent or intentional actions by our employees, contractors, consultants, service providers, business partners, and/or other third parties, or from cyber-attacks by malicious third parties (including supply chain cyber-attacks or the deployment of harmful malware, ransomware, denial-of-service attacks, social engineering and other means to affect service reliability and threaten the confidentiality, integrity and availability of information), which may compromise the confidentiality, integrity, or availability of our information systems or the information contained therein, or otherwise availability or lead to the loss, destruction, alteration, prevention of access to, disclosure, or dissemination of, or damage or unauthorized access to, our data (including trade secrets or other confidential information, intellectual property, proprietary business information, and personal information) or data that is processed or maintained on our behalf, or other assets, which could result in financial, legal, business and reputational harm to us.
+Added: We, our CROs, and other vendors that we work with have experienced cybersecurity incidents in the past (including one of our vendors in 2026 and CROs of our in 2025, 2023, and 2019) and we cannot eliminate the risk that we, our CROs, and other vendors will experience them in the future.
While we regularly monitor the security of our systems, attackers have become very sophisticated in the way they conceal access to systems, and we may not be aware that we have been attacked.
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Any such event that leads to loss, damage, or unauthorized access to, or use, alteration, or disclosure, dissemination, or other processing of, personal information, including personal information regarding our clinical trial subjects or employees, or the perception that any such event has occurred, could harm our reputation directly, compel us to comply with federal and/or state breach notification laws and foreign law equivalents, subject us to mandatory corrective action, cause us to incur costs, and otherwise subject us to liability under laws and regulations that protect the privacy and security of personal information, which could result in significant legal and financial exposure and reputational damages that could potentially have an adverse effect on our business.
+Added: Advances in artificial intelligence (AI) and other technologies may increase our cybersecurity risks.
+Added: The already-increasing sophistication and expertise among hackers may be magnified through the malicious use of AI technologies that have the potential to dramatically increase the speed and impact of cyberattacks.
+Added: We, and our third-party CROs, other contractors (including sites performing our clinical trials), service providers, suppliers, and consultants also continue to incorporate artificial intelligence (AI) technologies into our solutions and otherwise in our business, and that may result in security incidents, magnify the impact of such incidents, or otherwise increase cybersecurity risks.
+Added: Generative AI tools, particularly those that employ large language models (LLMs), used by us or our third-party CROs, contractors or service providers can also pose significant risks of data leakage, which could lead to loss, disclosure, dissemination, or other unauthorized processing of our data (including trade secrets or other confidential information, intellectual property, proprietary business information, and personal information), data that is maintained or otherwise processed on our behalf, or other assets, which could result in financial, legal, business and reputational harm to us.
+Added: To mitigate such risks, we generally seek to limit authorized use of AI tools in our business to internal LLMs.
Notifications and follow-up actions related to a security breach or incident could impact our reputation and cause us to incur significant costs, including legal expenses and remediation costs.
−Removed: For example, the loss, corruption or (temporary or permanent) unavailability of clinical trial data from completed or future clinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the impacted data.
+Added: For example, the loss, corruption or (temporary or permanent) unavailability of clinical trial data from completed or future clinical trials
+Added: could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the impacted data.
We expect to incur significant costs in an effort to detect and prevent security breaches and incidents, and we may face increased costs and requirements to expend substantial resources in the event of an actual or perceived security breach or incident.
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Our operations are vulnerable to interruption by fire, earthquakes, power loss, telecommunications failure, terrorist activity, pandemics and other events beyond our control, which could harm our business.
−Removed: Our facilities are located in Boulder, Colorado.
+Added: Our facilities and many of our personnel are located in Boulder, Colorado.
We have not undertaken a systematic analysis of the potential consequences to our business and financial results from a major flood, blizzard, fire, earthquake, power loss, terrorist activity, pandemics or other disasters and do not have a recovery plan for such disasters.
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government administration and policy positions , that can impact the supply chain, our business, or business operations of our suppliers, contractors or partners;
−Removed: ● business interruptions resulting from geo-political actions, including war, such as the ongoing war in Ukraine and war and instability in the Middle East, other regional or geo-political conflicts, and terrorism.
−Removed: In particular, there is currently significant uncertainty about the future relationship between the United States and various other countries, most significantly China, with respect to trade policies, treaties, tariffs, taxes, any retaliatory actions in respect thereto, and other limitations on cross-border operations.
+Added: ● business interruptions resulting from geo-political actions, including war, such as the ongoing war in Ukraine and war and instability in the U.S., Iran, Israel, and Lebanon, other regional or geo-political conflicts, and terrorism.
+Added: In particular, there is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, tariffs, taxes, any retaliatory actions in respect thereto, and other limitations on cross-border operations.
government has and continues to make significant additional changes in U.S.
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trade and our business.
−Removed: For example, legislation in Congress known as the BIOSECURE Act has been re-introduced as an amendment to the National Defense Authorization Act to limit certain U.S.
−Removed: biotechnology companies from using equipment or services produced or provided by select Chinese biotechnology companies, and others in Congress have advocated for the use of existing executive branch authorities to limit those Chinese service providers’ ability to engage in business in the U.S.
−Removed: In February and April 2025, the current U.S.
−Removed: presidential administration imposed new tariffs on China and China responded with tariffs on select U.S.
−Removed: The proposed legislation, or similar laws and regulations in the future, could adversely impact our current or future third-party arrangements with certain companies, including those in China or Chinese-owned U.S.
+Added: For example, legislation in Congress known as the BIOSECURE Act was enacted in December 2025 as part of the 2026 National Defense Authorization Act, and places limitations on certain interactions with certain Chinese and other biotechnology firms that may pose a threat to United States national security;
+Added: additional proposals have been raised regarding possible executive actions to further limit those Chinese service providers’ ability to engage in business in the United States.
+Added: In addition, since February 2025, the U.S.
+Added: administration has imposed new tariffs of 10% - 145% on many products imported from China citing authorities provided for in the International Emergency Economic Powers Act (IEEPA), and China responded with retaliatory tariffs on select U.S.
+Added: These additional U.S.
+Added: tariffs implemented under IEEPA were rescinded on February 24, 2026, following a Supreme Court decision invalidating the use of IEEPA to authorize these tariffs, but the U.S.
+Added: government subsequently announced plans to implement a new “temporary import surcharge” of 15% on many of the same imports beginning February 24, 2026, under authorities provided for in Section 122 of the Trade Act of 1974.
+Added: These or other tariffs, actual or proposed legislation, or similar laws and regulations in the future, including changes to implemented bilateral trade deals between the U.S.
+Added: and China, could adversely impact our current or future third-party arrangements with certain companies, including those in China or Chinese-owned U.S.
companies, which could delay or impact our clinical trials and consequently delay or obstruct successful commercialization of our product candidates.
−Removed: We cannot predict what actions may ultimately be taken with respect to trade relations between the United States and China or other countries, what products and services may be subject to such actions
−Removed: or what actions may be taken by the other countries in retaliation.
+Added: We cannot predict what actions may ultimately be taken with respect to trade relations between the United States and China or other countries, what products and services may be
+Added: subject to such actions or what actions may be taken by the other countries in retaliation.
If we are unable to obtain or use services from existing service providers or become unable to export or sell our products to any of our customers or service providers, our business, liquidity, financial condition, and/or results of operations would be materially and adversely affected.
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jurisdictions could increase the delays, uncertainties and costs surrounding the prosecution of our patent applications, and the maintenance, enforcement, or defense of our issued patents.
−Removed: For example, the ability of the USPTO and other applicable patent authorities to properly administer their functions is highly dependent on the levels of funding available to the agency and their ability to retain personnel and fill key leadership appointments, among various factors.
+Added: For example, the ability of the USPTO and other applicable patent authorities to properly administer their functions is highly dependent on the levels of funding available to the agency, their ability to attract and retain personnel, and fill key leadership appointments, among various factors.
Termination of employees or delays in replacing or hiring for positions could significantly impact the ability of the USPTO and other applicable patent authorities to fulfill their functions and could greatly impact our ability to timely and adequately prosecute or maintain our patent applications, and our ability to timely and adequately maintain, enforce, or defend our issued patents.
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Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.
−Removed: As a result, our intellectual property may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
+Added: As a result, our intellectual property may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours for an extended duration, if at all.
If the scope of any patent protection we obtain is not sufficiently broad, or if we lose any of our patent protection, our ability to prevent our competitors from commercializing similar or identical product candidates would be adversely affected.
−Removed: The patent position of biopharmaceutical companies generally is highly uncertain, involves complex legal and factual questions, and has been the subject of much litigation in recent years.
−Removed: As a result, the issuance, scope,
−Removed: validity, enforceability and commercial value of our patent rights are highly uncertain.
+Added: The ability of biotech companies to protect their intellectual property through patents is highly uncertain, involves complex legal and factual questions, and has been the subject of much litigation in recent years.
+Added: result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain.
Our pending and future patent applications and those of any licensor may not result in patents being issued which protect our product candidates or which effectively prevent others from commercializing competitive product candidates.
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and foreign issued patents and pending patent applications exist in the fields in which we are developing product candidates.
−Removed: There may be third-party patents or patent applications with claims to materials, formulations, methods of manufacture or methods for treatment related to the use or manufacture of our product candidates.
+Added: There are third-party patents or patent applications that may have claims to materials, formulations, methods of manufacture or methods for treatment related to the use or manufacture of our product candidates.
There are numerous U.S.
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Although no third-party has asserted a claim of patent infringement against us as of the date of this periodic report, others may hold proprietary rights that could prevent our product candidates from being marketed.
−Removed: For example, we are aware of an issued patent that claims a method of treatment based upon a general mode of action.
+Added: For example, we are aware of an issued patent that claims a method of treatment based upon a general mode of action and a pending patent application that generically claims a composition of matter.
These claims could be alleged to cover sevasemten in certain treatment indications.
−Removed: While we believe that these patents are difficult to enforce and that we would have valid defenses to these claims of patent infringement, we cannot be certain that we would prevail in any dispute and we cannot be certain how an adverse determination would affect our business.
+Added: While we believe that this patent and pending patent applications will be difficult to enforce and that we would have valid defenses to these claims of patent infringement, we cannot be certain that we would prevail in any dispute and we cannot be certain how an adverse determination would affect our business.
It is possible that a third party may assert a claim of patent infringement directed at any of our product candidates.
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The outcome following legal assertions of invalidity is unpredictable.
−Removed: Additionally, we may be subject to claims of patent infringement during those proceedings, and delays caused by the federal agencies may increase the time period that we are subject to such claims.
+Added: In addition, we may be subject to claims of patent infringement during those proceedings, and delays caused by the federal agencies may increase the time period that we are subject to such claims.
For example, administrative changes, including reduced personnel and budgets experienced by the Patent and Trial Appeal Board, could further delay our ability to timely challenge any such patents.
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In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock.
−Removed: Such proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing or distribution activities.
+Added: Such proceedings could be expensive, substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing or distribution activities.
We may not have sufficient financial or other resources to conduct such proceedings adequately.
−Removed: Some of these third parties may be able to sustain the costs of such proceedings more effectively than we can because of their greater financial
+Added: Some of these third parties
+Added: may be able to sustain the costs of such proceedings more effectively than we can because of their greater financial resources.
Uncertainties resulting from the initiation and continuation of patent proceedings could compromise our ability to compete in the marketplace.
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To prevent infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming.
−Removed: In addition, in a patent infringement proceeding, a court may decide that a patent we own or in-license is not valid, is unenforceable and/or is not infringed.
+Added: In addition, in a patent infringement proceeding, a court may decide that a patent we own or in-license is invalid, is unenforceable and/or is not infringed.
If we or any of our potential future collaborators were to initiate legal proceedings against a third-party to enforce a patent directed at one of our product candidates, the defendant could counterclaim that our patent or the patent of any licensor is invalid and/or unenforceable in whole or in part.
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As is the case with other pharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents.
−Removed: Obtaining and enforcing patents in the pharmaceutical industry involve a high degree of
−Removed: technological and legal complexity.
+Added: Obtaining and enforcing patents in the pharmaceutical industry involve a high degree of technological and legal complexity.
Therefore, obtaining and enforcing pharmaceutical patents is costly, time consuming and inherently uncertain.
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If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights.
−Removed: Such an outcome could have a material adverse effect on our business.
+Added: Any such outcome could have a material adverse effect on our business, results of operations, and prospects.
Even if we are successful in defending against such claims, litigation could result in substantial costs and distraction to management and other employees.
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Patent term extension may also be available in certain foreign countries upon regulatory approval of our product candidates.
−Removed: However, we may not be granted an extension because of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements.
+Added: However, we may not be granted an extension because of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or
+Added: otherwise failing to satisfy applicable requirements.
Moreover, the applicable time period or the scope of patent protection afforded could be less than we request.
If we are unable to obtain patent term extension or restoration or the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our revenue could be reduced, possibly materially.
−Removed: Further, if this occurs, our competitors may take advantage of our investment in development and trials by referencing our clinical and
−Removed: preclinical data and launch their product earlier than might otherwise be the case.
+Added: Further, if this occurs, our competitors may take advantage of our investment in development and trials by referencing our clinical and preclinical data and launch their product earlier than might otherwise be the case.
Additionally, administrative changes at the USPTO or other applicable patent authorities, such as reduced hiring and/or funding, may result in delays in issuance of a patent or in accrual of patent term extension, thereby reducing the amount of patent term extension that could otherwise be received.
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If such an event were to occur, it could have a material adverse effect on our business.
−Removed: In addition, a decree was adopted by the Russian government in March 2022, allowing Russian companies and individuals to exploit inventions owned by patentees that have citizenship or nationality in, are registered in, or have predominately primary place of business or profit-making activities in the United States and other countries that Russia has deemed unfriendly without consent or compensation.
+Added: addition, a decree was adopted by the Russian government in March 2022, allowing Russian companies and individuals to exploit inventions owned by patentees that have citizenship or nationality in, are registered in, or have predominately primary place of business or profit-making activities in the United States and other countries that Russia has deemed unfriendly without consent or compensation.
Consequently, we would not be able to prevent third parties from practicing our inventions in Russia or from selling or importing products made using our inventions in and into Russia.
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In addition, we rely on the protection of our trade secrets, including unpatented know-how, technology and other proprietary information to maintain our competitive position.
−Removed: Although we have taken steps to protect our trade secrets and unpatented know-how, including entering into confidentiality agreements with third parties, and confidential information and inventions agreements with employees, consultants and advisors, we cannot provide any assurances that all such agreements have been duly executed, and any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches.
+Added: Although we have taken steps to protect our trade secrets and unpatented know-how, including entering into confidentiality agreements with third parties, and confidential information and inventions agreements with employees, consultants and advisors, we cannot provide
+Added: any assurances that all such agreements have been duly executed, and any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches.
Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable.
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We may enter into license agreements in the future to advance our research or allow commercialization of product candidates.
−Removed: These and other licenses may not provide exclusive rights to use such intellectual property and
−Removed: technology in all relevant fields of use and in all territories in which we may wish to develop or commercialize our technology and products in the future.
+Added: These and other licenses may not provide exclusive rights to use such intellectual property and technology in all relevant fields of use and in all territories in which we may wish to develop or commercialize our technology and products in the future.
In addition, subject to the terms of any such license agreements, we may not have the right to control the preparation, filing, prosecution, maintenance, enforcement, and defense of patents and patent applications covering the technology that we license from third parties.
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In addition, the agreements under which we license intellectual property or technology from third parties are complex, and certain provisions in such agreements may be susceptible to multiple interpretations.
−Removed: The resolution
−Removed: of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations, and prospects.
+Added: The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations, and prospects.
Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects.
19 unchanged sentences
(2) government action is necessary to meet public health or safety needs;
−Removed: or (3) government action is necessary to meet requirements for public use under federal regulations (also referred to as “march-in rights”).
+Added: or (3) government action is necessary to meet requirements for public use under federal
+Added: regulations (also referred to as “march-in rights”).
government exercised its march-in rights in our future intellectual property rights that are generated through the use of U.S.
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This preference for U.S.
−Removed: industry may be waived by the federal agency that provided the funding if the owner or assignee of the intellectual property can show that reasonable but unsuccessful efforts have been made to grant licenses on similar
−Removed: terms to potential licensees that would be likely to manufacture substantially in the United States or that under the circumstances domestic manufacture is not commercially feasible.
+Added: industry may be waived by the federal agency that provided the funding if the owner or assignee of the intellectual property can show that reasonable but unsuccessful efforts have been made to grant licenses on similar terms to potential licensees that would be likely to manufacture substantially in the United States or that under the circumstances domestic manufacture is not commercially feasible.
This preference for U.S.
133 unchanged sentences
● general economic, political, industry and market conditions including the impact of changes in the U.S.
−Removed: government administration and policy positions, the U.S.
+Added: government administration and policy positions, any U.S.
federal government shutdown, and the impact of any increase in inflation.
22 unchanged sentences
● future accounting pronouncements or changes in our accounting policies;
−Removed: ● the changing and volatile global economic and political environment, including as a result of the recent change in the U.S.
−Removed: presidential administration ;
+Added: ● the changing and volatile global economic and political environment;
● increased impact from public health pandemics on the costs and timing associated with the conduct of our clinical trial and other related business activities.
6 unchanged sentences
Our affiliated principal stockholders and management own a significant percentage of our stock and will be able to exert significant control over matters subject to stockholder approval.
−Removed: As of September 30, 2025, our executive officers, directors, affiliated holders of 5% or more of our capital stock and their respective affiliates beneficially owned approximately 22.0% of our outstanding common stock.
+Added: As of March 31, 2026, our executive officers, directors, affiliated holders of 5% or more of our capital stock and their respective affiliates beneficially owned approximately 21.3% of our outstanding common stock.
These stockholders, acting together, may be able to control matters requiring stockholder approval.
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Moreover, certain holders of our common stock have rights, subject to conditions, to require us to file registration statements covering their shares or to include their shares in registration statements that we may file for ourselves or other stockholders.
−Removed: Registration of these shares, under the Securities Act, would result in the shares becoming freely tradeable in the public market, subject to the restrictions of Rule 144 in the case of our affiliates.
+Added: Registration of these shares, under the Securities Act of 1933, as amended (Securities Act), would result in the shares becoming freely tradeable in the public market, subject to the restrictions of Rule 144 in the case of our affiliates.
In addition, shares registered under Form S-8 to register shares of our common stock reserved for issuance under our equity compensation plans become available for sale in the public market subject to the satisfaction of applicable vesting arrangements and the exercise of such options and, in the case of our affiliates, the restrictions of Rule 144.
3 unchanged sentences
The trading market for our common stock is influenced by the research and reports that securities or industry analysts publish about us, our business or our market.
−Removed: We currently have research coverage from a limited number
−Removed: of securities or industry analysts.
+Added: We currently have research coverage from a limited number of securities or industry analysts.
If no or few new securities or industry analysts commence coverage of us, the stock price may be negatively impacted.
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Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud will be detected.
−Removed: If we are not able to comply with the requirements of Section 404 of the Sarbanes-Oxley Act, or if we are unable to maintain proper and effective internal controls, we may not be able to produce timely and accurate financial statements.
+Added: If we are not able to comply with the requirements of Section 404 of the Sarbanes-Oxley Act, or if we are unable to maintain proper and effective internal controls, we may not be able to produce timely and accurate
+Added: financial statements.
If that were to happen, the market price of our stock could decline and we could be subject to sanctions or investigations by the stock exchange on which our common stock is listed, the SEC or other regulatory authorities.
9 unchanged sentences
This risk is especially relevant for us because biotechnology companies have experienced significant stock price volatility in recent years and we may be the target of this type of litigation in the future.
−Removed: Securities litigation against us could result in substantial costs and divert our management’s attention from other business concerns, which could seriously harm our business.
+Added: Securities litigation against us could result in substantial costs and divert our management’s attention from other business concerns.
We do not intend to pay dividends on our common stock in the foreseeable future, so any returns will be limited to the value of our common stock.
32 unchanged sentences
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.