29 unchanged sentences
● Our product candidates may not achieve adequate market acceptance among physicians, patients, healthcare payors and others in the medical community necessary for commercial success.
−Removed: ● The patient population suffering from Duchenne, Becker and Limb-girdle muscular dystrophy is small and has not been established with precision.
+Added: ● The patient population suffering from Duchenne muscular dystrophy (Duchenne), Becker muscular dystrophy (Becker) and Limb-girdle muscular dystrophy is small and has not been established with precision.
● The regulatory approval processes of the FDA, EMA and other comparable foreign regulatory authorities are lengthy, time consuming and inherently unpredictable.
11 unchanged sentences
We commenced operations in 2017, have no products approved for commercial sale and have not generated any revenue.
−Removed: In July 2022, we initiated the first of four Phase 2 clinical trials for our lead product candidate, sevasemten , and we initiated a Phase 2 clinical trial in April 2024 of our product candidate EDG-7500.
+Added: In July 2022, we initiated the first of four Phase 2 clinical trials for our lead product candidate, sevasemten , and we are enrolling Part B and Part C of a multipart Phase 2 clinical trial with our product candidate EDG-7500 for people with HCM.
We have not yet initiated clinical trials for any other product candidate, including product candidates from our EDG-003 cardiometabolic discovery program.
8 unchanged sentences
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 $31.5 million and $60.0 million for the three and six months ended June 30, 2024, respectively.
−Removed: As of June 30, 2024, we had an accumulated deficit of $304.8 million.
+Added: Our net loss was $34.1 million and $94.2 million for the three and nine months ended September 30, 2024, respectively.
+Added: As of September 30, 2024, we had an accumulated deficit of $338.9 million.
We are advancing sevasemten and EDG-7500 in clinical development.
5 unchanged sentences
The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenue.
−Removed: Our prior losses and expected future losses have had and will continue to have an adverse effect on our working capital,
−Removed: our ability to fund the development of our product candidates and our ability to achieve and maintain profitability and the performance of our stock.
+Added: Our prior losses and expected future losses have had and will continue to have an adverse effect on our working capital, our ability to fund the development of our product candidates and our ability to achieve and maintain profitability and the performance of our stock.
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.
28 unchanged sentences
If we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis.
−Removed: Our failure to become and remain profitable would decrease the value of our company and could impair our ability to maintain or further our research and development efforts, raise additional necessary capital, grow our business and continue our operations.
+Added: Our failure to become and remain profitable would decrease
+Added: the value of our company and could impair our ability to maintain or further our research and development efforts, raise additional necessary capital, grow our business and continue our operations.
We may also experience delays in developing a sustainable, reproducible and scalable manufacturing process or transferring that process to commercial partners, which may prevent us from completing our clinical trials or commercializing our product candidates on a timely or profitable basis, if at all.
2 unchanged sentences
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 June 30, 2024, we had $511.8 million in cash, cash equivalents and marketable securities.
+Added: As of September 30, 2024, we had $492.5 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.
3 unchanged sentences
Our estimate as to how long we expect our existing cash, cash equivalents and marketable securities to be able to continue to fund our operations is based on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect.
−Removed: Changing circumstances, some of which may be beyond our control, could cause
−Removed: us to consume capital significantly faster than we currently anticipate, and we may need to seek additional funds sooner than planned.
+Added: Changing circumstances, some of which may be beyond our control, could cause us to consume capital significantly faster than we currently anticipate, and we may need to seek additional funds sooner than planned.
Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a very time-consuming, expensive and uncertain process that takes years to complete.
40 unchanged sentences
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.
−Removed: Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making acquisitions, engaging in acquisition, merger or collaboration transactions, selling or licensing our assets, making capital expenditures, redeeming our stock, making certain investments, declaring dividends or encumbering our
−Removed: assets to secure future indebtedness.
+Added: Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making acquisitions, engaging in acquisition, merger or collaboration transactions, selling or licensing our assets, making capital expenditures, redeeming our stock, making certain investments, declaring dividends or encumbering our assets to secure future indebtedness.
Such restrictions could adversely impact our ability to conduct our operations and execute our business plan.
10 unchanged sentences
Similar rules may apply under state tax laws.
−Removed: 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.
+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
+Added: 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.
8 unchanged sentences
Any of these developments or changes in U.S.
−Removed: federal, state, or international tax laws or tax rulings could adversely
−Removed: affect our effective tax rate and our operating results.
+Added: federal, state, or international tax laws or tax rulings could adversely affect our effective tax rate and our operating results.
There can be no assurance that our effective tax rates, tax payments, or tax credits and incentives will not be adversely affected by these or other developments or changes in law.
29 unchanged sentences
For example, we experienced delays in enrolling patients for our Phase 1 clinical trial for sevasemten .
−Removed: 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 the pandemic.
+Added: In addition, our clinical trial sites could experience delays in collecting, receiving, and analyzing data from patients enrolled in our
+Added: 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 the pandemic.
As a result, research and development expenses and general and administrative expenses may vary significantly if there is an increased impact from COVID-19 or other public health emergencies on the costs and timing associated with the conduct of our clinical trial and other related business activities.
35 unchanged sentences
Our future operating results are dependent on our ability to successfully develop, obtain regulatory approval for and commercialize EDG-7500, product candidates from our research program currently focused on cardiometabolic indications, or EDG-003, and our lead product candidate, sevasemten .
−Removed: EDG-7500 is currently in a Phase 2 trial.
+Added: EDG-7500 is currently in a multipart Phase 2 trial.
However, research and development related to novel therapeutics is inherently risky.
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Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials even after achieving promising results in preclinical testing and earlier-stage clinical trials, and we cannot be certain that we will not face similar setbacks.
−Removed: Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of their drugs.
+Added: Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain
+Added: marketing approval of their drugs.
Furthermore, the failure of any of our product candidates to demonstrate safety and efficacy in any clinical trial could negatively impact the perception of our other product candidates and/or cause the FDA or other regulatory authorities to require additional testing before approving any of our product candidates.
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Under certain circumstances, we may be required to report some of these relationships to the FDA or comparable foreign regulatory authorities.
−Removed: The FDA or comparable foreign regulatory authority may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected
−Removed: interpretation of the study.
+Added: The FDA or comparable foreign regulatory authority may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected interpretation of the study.
The FDA or comparable foreign regulatory authority may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the clinical trial itself may be jeopardized.
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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
+Added: 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.
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.
1 unchanged sentence
Patients in our ongoing and planned clinical trials may in the future suffer other adverse events or other side effects not observed in our preclinical studies or previous clinical trials.
−Removed: For example, in the single ascending dose trial for sevasemten , dose limiting somnolence was observed at the 135 mg level.
−Removed: In addition, in the MAD trial for sevasemten , the most common adverse events were dizziness and somnolence, all of which were mild and transient.
−Removed: In the Phase 1b clinical trial, the most common adverse event was dizziness, which was mild and transient and was consistent with observations from the ARCH trial of sevasemten in adults with Becker.
+Added: For example, in the single ascending dose (SAD) trial for sevasemten , dose limiting somnolence was observed at the 135 mg level.
+Added: In addition, in the multiple ascending dose (MAD) trial for sevasemten , the most common adverse events were dizziness and somnolence, all of which were mild and transient.
+Added: In the ARCH trial of sevasemten in adults wit Becker, the most common adverse events were dizziness, fall, and arthralgia, which were mild and transient.
Sevasemten or other product candidates may be used in pediatric populations for which safety concerns may be particularly scrutinized by regulatory agencies.
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Product candidates in clinical trials may fail to demonstrate sufficient safety and efficacy to the satisfaction of the FDA, EMA and other comparable foreign regulatory authorities despite having progressed through preclinical studies.
−Removed: Regulatory authorities may also limit the scope of later-stage trials until we have demonstrated satisfactory safety, which could delay regulatory approval, limit the size of the patient population to which we may market our product candidates, or prevent regulatory approval.
+Added: Regulatory authorities may also limit the scope of later-stage trials until we have demonstrated satisfactory safety, which could delay regulatory
+Added: approval, limit the size of the patient population to which we may market our product candidates, or prevent regulatory approval.
In some instances, there can be significant variability in safety and efficacy results between different clinical trials of the same product candidate due to numerous factors, including changes in trial protocols, differences in size and type of the patient populations, differences in and adherence to the dose and dosing regimen and other trial protocols and the rate of dropout among clinical trial participants.
5 unchanged sentences
We may not be able to initiate or continue clinical trials for our product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials to such trial’s conclusion as required by the FDA, EMA or other comparable foreign regulatory authorities.
−Removed: Patient enrollment is a significant factor in
−Removed: the timing of clinical trials.
+Added: Patient enrollment is a significant factor in the timing of clinical trials.
Our ability to enroll eligible patients may be limited or may result in slower enrollment than we anticipate.
−Removed: We completed our open-label ARCH trial and completed enrollment of the DUNE Phase 2 exercise challenge study of sevasemten at a single clinical trial site.
−Removed: Our other Phase 2 clinical trials are being conducted at multiple sites.
+Added: For sevasemten trials, we completed our open-label ARCH trial and completed enrollment of the DUNE Phase 2 exercise challenge study at a single clinical trial site and our CANYON Phase 2 clinical trial at multiple sites.
+Added: All other Phase 2 clinical trials are also being conducted at multiple sites.
We have initiated 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 and identifying future eligibility for recruitment into our planned registrational trial in Becker.
+Added: In addition, we completed the Phase 1 trial with EDG-7500 in healthy subjects and the Part A single-dose arm of the multipart Phase 2 Cirrus-CM trial in patient with oHCM.
+Added: However, we may experience difficulty with enrollment and/or maintenance of patients in the ongoing enrollment of Part B and Part C of the Phase 2 trial.
We are developing product candidates for severe muscle diseases with limited patient pools from which to draw for clinical trials.
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Our inability to enroll a sufficient number of patients for our clinical trials would result in significant delays or may require us to abandon one or more clinical trials altogether.
−Removed: Enrollment delays in our clinical trials may
−Removed: result in increased development costs for our product candidates and jeopardize our ability to obtain marketing approval for the sale of our product candidates.
+Added: 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.
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.
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The biotechnology and biopharmaceutical industries are characterized by rapidly advancing technologies, intense competition and a strong emphasis on proprietary and novel products and product candidates.
−Removed: Our competitors have developed, are developing or may develop products, product candidates and processes competitive with our product candidates.
+Added: Our competitors have developed, are developing or may develop products, product candidates and processes
+Added: competitive with our product candidates.
Any product candidates that we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future.
11 unchanged sentences
In May 2024, Nippon Shinyaku Co.
−Removed: announced that no statistical significance was
−Removed: observed between the treatment group and the placebo group in VILTEPSO’s confirmatory study.
+Added: announced that no statistical significance was observed between the treatment group and the placebo group in VILTEPSO’s confirmatory study.
This result may affect VILTEPSO’s 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 plans to engage with the FDA and submit an NDA in 2024.
+Added: It remains unclear if the data will lead to FDA approval of Translarna, for which the company resubmitted the NDA in July 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.
3 unchanged sentences
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, PepGen, Dyne Therapeutics, Avidity Biosciences, REGENXBIO, and Entrada Therapeutics.
+Added: Other companies focused on developing genetic based therapies for Duchenne that target dystrophin mechanisms include Solid Biosciences Inc., Genethon, PepGen, Dyne Therapeutics, Avidity Biosciences, REGENXBIO, Wave Life Sciences, and Entrada Therapeutics.
In June 2024, Pfizer announced its gene therapy Phase 3 study failed to meet the primary and key secondary endpoints and is no longer under development.
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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 obstructive hypertrophic cardiomyopathy (oHCM) and nonobstructive hypertrophic cardiomyopathy (nHCM) and reduce left ventricular outflow tract gradient in oHCM patients only.
+Added: Current pharmaceutical treatment is intended to improve diastolic filling in both obstructive hypertrophic cardiomyopathy
+Added: (oHCM) and nonobstructive hypertrophic cardiomyopathy (nHCM) and reduce left ventricular outflow tract gradient in oHCM patients only.
The goal of current therapies is to achieve meaningful symptom relief.
3 unchanged sentences
For oHCM patients who remain symptomatic, disopyramide (either Norpace, marketed by Pfizer, or a generic form marketed by several companies) or Camzyos (mayacamten) may also be added.
−Removed: In the field of emerging treatments intended to treat HCM, competitors include Bristol-Myers Squibb (BMS), LianBio, Cytokinetics, Imbria Pharmaceuticals, and Celltrion.
+Added: In the field of emerging treatments intended to treat HCM, competitors include Bristol-Myers Squibb (BMS), Cytokinetics, Imbria Pharmaceuticals, and Celltrion.
Cytokinetics is developing a CMI, aficamten (CK-274), for which positive topline Phase 3 oHCM trial results were reported in December 2023.
In June 2023, Cytokinetics initiated another Phase 3 active-comparator clinical trial of aficamten compared to metoprolol in symptomatic oHCM patients (MAPLE trial).
+Added: In the second quarter of 2024, BMS and Cytokinetics initiated a study of mavacamten and aficamten, respectively, in pediatric population with symptomatic oHCM.
BMS and Cytokinetics are also exploring their respective CMIs in ongoing Phase 3 nHCM clinical trials, and both are also developing next generation CMIs for the treatment of symptomatic HCM, CK-271 and MYK-224, respectively.
1 unchanged sentence
Non-CMI targeting drugs in development include IMB-101(Imbria Pharmaceuticals), a prodrug of a 3-ketoacyl CoA thiolase enzyme inhibitor, CT-G20 (Celltrion), an anti-arrhythmic cibenzoline succinate, and trientine dihydrochloride (Univar Solutions), a selective copper II chelator.
−Removed: Lexeo Therapeutics announced plans to investigate sotagliflozin, an SGLT1 and SGLT2 inhibitor, in a Phase 3 study of obstructive and non-obstructive HCM patients.
+Added: Lexicon Pharmaceuticals announced plans to investigate sotagliflozin, an SGLT1 and SGLT2 inhibitor, in a Phase 3 study of obstructive and non-obstructive HCM patients.
In March 2024, the results of a Phase 2 trial in nHCM of IMB-101 were published.
2 unchanged sentences
We are aware of several preclinical HCM programs including:
−Removed: JN-210, a microRNA activating
−Removed: gene therapy approach being developed by Jaan Biotherapeutics;
+Added: JN-210, a microRNA activating gene therapy approach being developed by Jaan Biotherapeutics;
HTX-001, an antisense oligonucleotide approach being developed by Haya Therapeutics;
−Removed: CDR348T and CDR641L, both are non-coding RNA-based therapies being developed by Cardior Pharmaceuticals.
+Added: CDR348T and CDR641L, both are non-coding RNA-based therapies being developed by Cardior Pharmaceuticals (acquired by Novo Nordisk in May 2024).
We are also aware of several early-stage preclinical HCM assets being developed by DiNAQOR in collaboration with BioMarin Pharmaceuticals (BMN-293/DINA-001) and Lexeo Therapeutics (LX2022), both are gene therapy approaches for genetic HCM.
+Added: In April 2024, BioMarin announced the discontinuation of the development of BMN-293.
Many of these current and potential competitors have significantly greater financial, manufacturing, marketing, drug development, technical and human resources and commercial expertise than we do.
6 unchanged sentences
Our competitors also may obtain marketing approval from the FDA, EMA or other comparable foreign regulatory authorities for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market.
−Removed: Even if the product candidates we develop achieve marketing approval, they may be priced at a significant premium over competitive products if any have been approved by then, resulting in reduced competitiveness.
+Added: Even if the product candidates we develop achieve marketing approval, they may be priced at a significant premium over competitive
+Added: products if any have been approved by then, resulting in reduced competitiveness.
Technological advances or products developed by our competitors may render our technologies or product candidates obsolete, less competitive or not economical.
2 unchanged sentences
From time to time, we may publicly disclose preliminary, interim or topline data from our clinical trials.
−Removed: For example, on April 15, 2024, we announced positive two-year topline results from the ARCH open label trial of sevasemten in adults with Becker.
+Added: For example, on April 15, 2024, we announced positive two-year topline results from the ARCH open label trial of sevasemten in adults with Becker and 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.
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.
5 unchanged sentences
Interim data from clinical trials that we may complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data becomes available.
−Removed: Adverse changes between interim data and final data could significantly harm
−Removed: our business and prospects.
+Added: Adverse changes between interim data and final data could significantly harm our business and prospects.
Further, additional disclosure of interim data by us or by our competitors in the future could result in volatility in the price of our common stock.
9 unchanged sentences
Furthermore, we may also find that the uses of our proprietary drug discovery platform are limited because alternative uses of our therapeutics prove not to be safe or effective.
−Removed: Even if we are successful in building our pipeline, the potential product candidates that we identify may not be suitable for clinical development, including as a result of being shown to have harmful side effects or other characteristics that indicate that they are unlikely to be products that will receive marketing approval or achieve market acceptance.
+Added: Even if we are successful in building our pipeline, the potential product candidates that we identify may not be suitable for clinical development, including as a result of being shown to have harmful side
+Added: effects or other characteristics that indicate that they are unlikely to be products that will receive marketing approval or achieve market acceptance.
Further, because our product candidates and development programs are based on our proprietary drug discovery platform, adverse developments with respect to one of our programs may have a significant adverse impact on the actual or perceived likelihood of success and value of our other programs.
8 unchanged sentences
Patients may not be able to tolerate sevasemten or any other product candidates in combination with other therapies or dosing of sevasemten in combination with other therapies may have unexpected consequences.
−Removed: Even if any of our product candidates were to receive marketing approval or
−Removed: be commercialized for use in combination with other existing therapies, we would continue to be subject to the risks that the FDA, EMA or other comparable foreign regulatory authorities could revoke approval of the therapy used in combination with any of our product candidates, or safety, efficacy, manufacturing or supply issues could arise with these existing therapies.
+Added: Even if any of our product candidates were to receive marketing approval or be commercialized for use in combination with other existing therapies, we would continue to be subject to the risks that the FDA, EMA or other comparable foreign regulatory authorities could revoke approval of the therapy used in combination with any of our product candidates, or safety, efficacy, manufacturing or supply issues could arise with these existing therapies.
In addition, it is possible that existing therapies with which our product candidates are approved for use could themselves fall out of favor.
42 unchanged sentences
We estimate that Duchenne occurs in approximately 35,000 patients in the US, EU (5) and Japan.
−Removed: Becker has a much lower incidence of approximately 1
−Removed: in every 18,450 live male births.
+Added: Becker has a much lower incidence of approximately 1 in every 18,450 live male births.
We estimate that Becker occurs in approximately 12,000 patients in the US, EU (5) and Japan.
13 unchanged sentences
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.
+Added: The review process
+Added: typically takes more than a year and requires two key votes of experts in NBS to move forward.
In February 2023, the first key vote took place and the ACHDNC decided that more information was needed before proceeding to the second vote.
25 unchanged sentences
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.
+Added: In the United States, for example, principal decisions about reimbursement for new products are typically
+Added: made by the Centers for Medicare & Medicaid Services (CMS), an agency within the U.S.
Department of Health and Human Services (HHS).
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As a result, we may be unable to obtain sufficient insurance at a reasonable cost to protect us against losses caused by product liability claims that could have an adverse effect on our business and financial condition.
−Removed: Also, our insurance policies may have various exclusions, and we may be subject to a product liability claim for which we have no coverage.
+Added: Also, our insurance policies may have various exclusions, and we may be subject to a product liability claim for
+Added: which we have no coverage.
We may have to pay any amounts awarded by a court or negotiated in a settlement that exceed our coverage limitations or that are not covered by our insurance, and we may not have, or be able to obtain, sufficient capital to pay such amounts.
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Our product candidates are and will continue to be subject to extensive governmental regulations relating to, among other things, research, testing, development, manufacturing, safety, efficacy, approval, recordkeeping, reporting, labeling, storage, packaging, advertising and promotion, pricing, marketing and distribution of drugs.
−Removed: Rigorous preclinical testing and clinical trials and an extensive regulatory approval process must be successfully completed in the United States and in many foreign jurisdictions before a new drug can be approved for marketing.
+Added: Rigorous preclinical testing and clinical trials and an extensive regulatory approval process must be successfully
+Added: completed in the United States and in many foreign jurisdictions before a new drug can be approved for marketing.
Obtaining approval by the FDA, EMA and other comparable foreign regulatory authorities is costly, unpredictable, typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the type, complexity and novelty of the product candidates involved.
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In addition, the FDA has the authority to require a Risk Evaluation and Mitigation Strategy (REMS) plan as part of approving an NDA, or after approval, which may impose further requirements or restrictions on the distribution or use of an approved drug.
−Removed: These requirements or restrictions might include limiting prescribing to certain physicians or medical centers that have undergone specialized training, limiting treatment to patients who meet certain safe-use criteria and requiring treated patients to enroll in a registry.
+Added: These requirements or restrictions might include limiting prescribing to certain physicians or medical centers that have undergone
+Added: specialized training, limiting treatment to patients who meet certain safe-use criteria and requiring treated patients to enroll in a registry.
These limitations and restrictions may significantly limit the size of the market for the drug and affect reimbursement by third-party payors.
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If we or a regulatory agency discover previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facilities where the product is manufactured, a regulatory agency may impose restrictions on that product, the manufacturing facility or us, including requiring recall or withdrawal of the product from the market or suspension of manufacturing.
−Removed: addition, failure to comply with FDA, EMA and other comparable foreign regulatory requirements may subject our company to administrative or judicially imposed sanctions, including:
+Added: In addition, failure to comply with FDA, EMA and other comparable foreign regulatory requirements may subject our company to administrative or judicially imposed sanctions, including:
● delays in or the rejection of product approvals;
33 unchanged sentences
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.
−Removed: 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.
+Added: 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
+Added: 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.
Further, even after obtaining orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs with different active moieties may be approved for the same condition.
12 unchanged sentences
The FDA considers a clinical benefit to be a positive therapeutic effect that is clinically meaningful in the context of a given disease, such as irreversible morbidity or mortality.
−Removed: For the purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image,
−Removed: physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit.
+Added: For the purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit.
An intermediate clinical endpoint is a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit.
6 unchanged sentences
Similarly, there can be no assurance that after subsequent FDA feedback we will continue to pursue or apply for accelerated approval or any other form of expedited development, review or approval, even if we initially decide to do so.
−Removed: Furthermore, if we decide to submit an application for accelerated approval or under another expedited regulatory designation (e.g., breakthrough therapy designation), there can be no assurance that such submission or application will be accepted or that any expedited development, review or approval will be granted on a timely basis, or at all.
+Added: Furthermore, if we decide to submit an application for
+Added: accelerated approval or under another expedited regulatory designation (e.g., breakthrough therapy designation), there can be no assurance that such submission or application will be accepted or that any expedited development, review or approval will be granted on a timely basis, or at all.
The FDA or other comparable foreign regulatory authorities could also require us to conduct further studies prior to considering our application or granting approval of any type.
11 unchanged sentences
pharmaceutical industry.
−Removed: Since its enactment, there have been judicial and congressional
−Removed: challenges to certain aspects of the ACA.
+Added: Since its enactment, there have been judicial and congressional challenges to certain aspects of the ACA.
In June 2021, the United States Supreme Court held that Texas and other challengers had no legal standing to challenge the ACA, dismissing the case without specifically ruling on the constitutionality of the ACA.
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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.
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At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
−Removed: A number of states are considering or have recently enacted state drug price transparency and reporting laws that could substantially increase our compliance burdens and expose us to greater liability under such state laws once we begin commercialization after
−Removed: obtaining regulatory approval for any of our products.
+Added: A number of states are considering or have recently enacted state drug price transparency and reporting laws that could substantially increase our compliance burdens and expose us to greater liability under such state laws once we begin commercialization after obtaining regulatory approval for any of our products.
For example, FDA recently authorized the state of Florida to import certain prescription drugs from Canada for a period of two years to help reduce drug costs, provided that Florida’s Agency for Health Care Administration meets the requirements set forth by the FDA.
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We cannot be sure whether additional legislative changes will be enacted, or whether FDA regulations, guidance or interpretations will be changed, or what the impact of such changes on the marketing approvals of our product candidates, if any, may be.
−Removed: In addition, increased scrutiny by Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.
+Added: In addition, increased scrutiny
+Added: by Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.
The regulatory framework for privacy and personal information security issues worldwide is rapidly evolving and is likely to remain uncertain for the foreseeable future.
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Failure to comply with the GDPR may result in fines up to €20,000,000 or up to 4% of the total worldwide annual turnover of the preceding financial year, whichever is higher, and other administrative penalties.
−Removed: The GDPR increases our responsibility and liability in relation to applicable personal data that we or our CROs and other contractors and service providers may process, and we may be required to put in place additional measures in an effort to comply with the GDPR and with other laws and regulations in the EU, including those of EU member states, relating to privacy and data protection.
+Added: The GDPR has increased our responsibility and liability in relation to applicable personal data that we or our CROs and other contractors and service providers may process, and we may be required to put in place additional measures in an effort to comply with the GDPR and with other laws and regulations in the EU, including those of EU member states, relating to privacy and data protection.
These efforts may require substantial efforts and incurring significant costs.
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In the medium and longer terms, however, the relationship between the UK and EU in relation to aspects of data protection law remains unclear, which exposes us to further compliance risk.
−Removed: The UK also has issued its own standard contractual clauses, similar to the SCCs, that are required to be implemented.
+Added: The UK also has issued its own standard contractual clauses that are required to be implemented.
We may incur liabilities, expenses, costs, and other operational losses relating to the GDPR, the UK GDPR, and other laws and regulations in the EU and UK relating to privacy and data protection, including those of applicable EU member states in connection with any measures we take to comply with them.
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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 new data privacy rights (including the ability to opt out of certain disclosures of personal data), imposes new 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),
+Added: 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).
Aspects of the CCPA and its interpretation and enforcement remain uncertain.
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Additionally, if third parties we work with, such as vendors or service providers, violate applicable laws or regulations or our policies, such violations may also put our or our customers’ data at risk and could in turn have an adverse effect on our business.
−Removed: 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
−Removed: 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.
+Added: 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.
+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.
Inadequate funding for the FDA, the SEC and other government agencies 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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For example, in 2018 and 2019, the U.S.
−Removed: government shut down several times and certain regulatory agencies, such as the FDA and the SEC, had to furlough critical employees and stop critical activities.
+Added: government shut down several times and certain regulatory
+Added: agencies, such as the FDA and the SEC, had to furlough critical employees and stop critical activities.
If any prolonged government shutdown or disruption occurs, including due to any public health emergencies, resurgence in COVID-19 cases, travel restrictions, or COVID-19-related policies, staffing shortages, it could significantly impact the ability of the FDA and other regulatory authorities to timely review and process our regulatory submissions and provide feedback on our clinical development plans, which could have a material adverse effect on our business and our anticipated timelines.
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Our employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, suppliers and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.
−Removed: We are exposed to the risk that our employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, suppliers and vendors may engage in misconduct or other improper
+Added: We are exposed to the risk that our employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, suppliers and vendors may engage in misconduct or other improper activities.
Misconduct by these parties could include failures to comply with FDA regulations, provide accurate information to the FDA, comply with federal and state health care fraud and abuse laws and regulations, accurately report financial information or data or disclose unauthorized activities to us.
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These laws generally prohibit companies and their employees, agents, representatives, business partners, and third-party intermediaries from, directly or indirectly, offering, promising, giving or authorizing others to give anything of value, either directly or indirectly, to recipients in the public or private sector in order to influence official action or otherwise obtain or retain business.
−Removed: Our business is heavily regulated and therefore involves significant interaction with public
−Removed: officials, including officials of non-U.S.
+Added: Our business is heavily regulated and therefore involves significant interaction with public officials, including officials of non-U.S.
Additionally, in many other countries, hospitals are owned and operated by the government, and doctors and other hospital employees would be considered foreign officials under the FCPA.
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If we fail to comply with export and import regulations and such economic sanctions, penalties could be imposed, including fines and/or denial of certain export privileges.
−Removed: Moreover, any new export or import restrictions, new legislation or shifting approaches in the enforcement or scope of existing regulations, or in the countries, persons, or products targeted by such regulations, including as a result of a change in administration from the upcoming U.S.
−Removed: presidential election, could result in decreased use of our products by, or in our decreased ability to export our products to, existing or potential customers with international operations.
+Added: Moreover, any new export or import restrictions, new legislation or shifting approaches in the enforcement or scope of existing regulations, or in the countries, persons, or products targeted by such regulations, including the impact of the upcoming change in the U.S.
+Added: presidential administration , could result in decreased use of our products by, or in our decreased ability to export our products to, existing or potential customers with international operations.
Any decreased use of our products or limitation on our ability to export or sell our products would likely adversely affect our business.
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Additionally, wage inflation may interfere with our ability to hire or retain personnel.
−Removed: If we do not succeed in attracting and retaining qualified personnel, particularly at the management
−Removed: level, it could adversely affect our ability to execute our business plan and harm our operating results.
+Added: If we do not succeed in attracting and retaining qualified personnel, particularly at the management level, it could adversely affect our ability to execute our business plan and harm our operating results.
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.
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Furthermore, our advisors may have arrangements with other companies to assist those companies in developing products or technologies that may compete with ours.
−Removed: In particular, if we are unable to maintain consulting relationships with our scientific founders or if they provide services to our competitors, our development and commercialization efforts will be impaired and our business will be significantly harmed.
+Added: In particular, if we are unable to
+Added: maintain consulting relationships with our scientific founders or if they provide services to our competitors, our development and commercialization efforts will be impaired and our business will be significantly harmed.
If we are unable to establish sales or marketing capabilities or enter into agreements with third parties to sell or market our product candidates, we may not be able to successfully sell or market our product candidates that obtain regulatory approval.
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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 June 30, 2024, we had 97 full-time employees.
+Added: As of September 30, 2024, we had 108 full-time employees.
Of these employees, 84 are engaged in research or product development and clinical activities.
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Our computer systems, or those of any of our CROs, manufacturers, other contractors or consultants or potential future collaborators, may fail or suffer security or data privacy breaches or incidents or other unauthorized or improper access to, use of, or destruction of our proprietary or confidential data, employee data, or personal data, which could result in additional costs, loss of revenue, significant liabilities, harm to our brand and material disruption of our operations.
−Removed: Despite the implementation of security measures in an effort to protect systems that store our information, given their 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) and consultants, these systems are potentially vulnerable to breakdown or other damage or interruption from service interruptions, system malfunction, 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, business partners, and/or other third parties, or from cyber-attacks by malicious third
−Removed: 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 our system infrastructure 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: Despite the implementation of security measures in an effort to protect systems that store our information, given their 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) 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, 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 our system infrastructure or 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.
For example, in 2019, one of our CROs experienced a cybersecurity breach which resulted in unauthorized access to certain of our preclinical data.
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Any disruption or security incident resulting in any loss, destruction, unavailability, alteration, disclosure, disruption or dissemination of, or damage or unauthorized access to, our applications, any other data processed or maintained on our behalf or other assets, or for it to be believed or reported that any of these occurred, could cause us to incur costs, liability, and other financial harm and reputational damage and could contribute to delays in the development and commercialization of our product candidates.
−Removed: We cannot assure you that our data protection efforts and our investment in information technology, or the efforts or investments of CROs, consultants or other third parties, will prevent significant breakdowns in systems or will prevent, or have prevented, other cyber incidents that could disrupt our programs and operations and the development of our product candidates or result in loss, destruction, unavailability, alteration or dissemination of, or damage or unauthorized access to, our data and other data processed or maintained on our behalf or other assets, any of which could have a material adverse effect upon our reputation, business, operations or financial condition.
+Added: We cannot assure you that our data protection efforts and our investment in information technology, or the efforts or investments of CROs, consultants or other third parties, will prevent significant breakdowns in systems or will prevent, or have prevented, other cyber incidents that could disrupt our programs and operations and the development of our product candidates or result in loss, destruction, unavailability, alteration or dissemination of, or damage or unauthorized access to, our systems and data and other data processed or maintained on our behalf or other assets, any of which could have a material adverse effect upon
+Added: our reputation, business, operations or financial condition.
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.
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 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 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 facilities are located in Boulder, Colorado.
−Removed: 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,
−Removed: terrorist activity, pandemics or other disasters and do not have a recovery plan for such disasters.
+Added: 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.
In addition, we do not carry sufficient insurance to compensate us for actual losses from interruption of our business that may occur, and any losses or damages incurred by us could harm our business.
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● unexpected changes in tariffs, trade barriers, price and exchange controls and other regulatory requirements;
−Removed: ● economic weakness, including inflation, change in political condition, including as a result of the upcoming U.S.
−Removed: presidential election, or political instability in particular foreign economies and markets;
+Added: ● economic weakness, including inflation, change in political condition, including as a result of the upcoming change in the U.S.
+Added: presidential administration , or political instability in particular foreign economies and markets;
● compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
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● impact of the COVID-19 pandemic or other public health concerns on our ability to produce our product candidates and conduct clinical trials in foreign countries;
−Removed: ● production or supply shortages or other disruptions resulting from any events affecting raw material supply or manufacturing capabilities abroad, including, but not limited to, impacts due to the ongoing Ukraine-Russia war, addition of certain suppliers or companies to the Unverified List or other export restrictions under the Export Administration Regulations, implementation of other export controls, restrictions or sanctions, including those that result from a change in administration from the upcoming U.S.
−Removed: presidential election, that can impact the supply chain, our business, or business operations of our suppliers, contractors or partners;
+Added: ● production or supply shortages or other disruptions resulting from any events affecting raw material supply or manufacturing capabilities abroad, including, but not limited to, impacts due to the ongoing Ukraine-Russia war, addition of certain suppliers or companies to the Unverified List or other export restrictions under the Export Administration Regulations, implementation of other export controls, restrictions or sanctions, including impact of the upcoming change in the U.S.
+Added: presidential administration , that can impact the supply chain, our business, or business operations of our suppliers, contractors or partners;
● business interruptions resulting from geo-political actions, including war, such as the ongoing war in Ukraine and war and instability in Israel and the surrounding region, other regional or geo-political conflicts, and terrorism.
1 unchanged sentence
government has and continues to make significant additional changes in U.S.
−Removed: trade policy and may continue to take future actions, including those that result from a change in administration from the upcoming U.S.
−Removed: presidential election, that could negatively impact U.S.
+Added: trade policy and may continue to take future actions, including the impact of the upcoming change in the U.S.
+Added: presidential administration , that could negatively impact U.S.
For example, legislation has been introduced in Congress to limit certain U.S.
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: 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 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
+Added: States and China or other countries, what products and services may be 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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Pending patent applications cannot be enforced against third parties practicing the technology claimed in such applications unless, and until, patents issue from such applications, and then only to the extent the issued claims cover the technology.
−Removed: There can be no assurance that our patent applications or the patent applications of any licensor will result in additional patents being issued or that issued patents will afford sufficient protection against
−Removed: competitors with similar technology, nor can there be any assurance that the patents issued will not be infringed, designed around or invalidated by third parties.
+Added: There can be no assurance that our patent applications or the patent applications of any licensor will result in additional patents being issued or that issued patents will afford sufficient protection against competitors with similar technology, nor can there be any assurance that the patents issued will not be infringed, designed around or invalidated by third parties.
Even issued patents may later be found invalid or unenforceable or may be modified or revoked in proceedings instituted by third parties before various patent offices or in courts.
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These risks and uncertainties include the following:
−Removed: ● the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process, the noncompliance with which can result in abandonment or lapse of a patent or patent application, and partial or complete loss of patent rights in the relevant jurisdiction;
+Added: ● the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process, the noncompliance
+Added: with which can result in abandonment or lapse of a patent or patent application, and partial or complete loss of patent rights in the relevant jurisdiction;
● patent applications may not result in any patents being issued;
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It is also possible that we or any licensor will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection.
−Removed: In addition, although we enter into non-disclosure and confidentiality agreements with parties who have access to patentable aspects of our research and development output, such as our employees, outside scientific collaborators, CROs, third-party manufacturers, consultants, advisors and other third parties, any of these parties
−Removed: may breach such agreements and disclose such output before a patent application is filed, thereby jeopardizing our ability to seek patent protection.
+Added: In addition, although we enter into non-disclosure and confidentiality agreements with parties who have access to patentable aspects of our research and development output, such as our employees, outside scientific collaborators, CROs, third-party manufacturers, consultants, advisors and other third parties, any of these parties may breach such agreements and disclose such output before a patent application is filed, thereby jeopardizing our ability to seek patent protection.
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.
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Any patents that we own or in-license may be challenged or circumvented by third parties or may be narrowed or invalidated as a result of challenges by third parties.
−Removed: Consequently, we do not know whether our product candidates will be protectable or remain protected by valid and enforceable patents.
+Added: Consequently, we do not know whether our
+Added: product candidates will be protectable or remain protected by valid and enforceable patents.
Our competitors or other third parties may be able to circumvent our patents or the patents of any licensors by developing similar or alternative technologies or products in a non-infringing manner which could materially adversely affect our business, financial condition, results of operations and prospects.
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and foreign issued patents and pending patent applications owned by third-parties in the fields in which we are developing our product candidates.
−Removed: Because patent applications are maintained as confidential for a certain period of time, until the relevant application is published, we may be unaware of third-
−Removed: party patents that may be infringed by commercialization of any of our product candidates, and we cannot be certain that we were the first to file a patent application related to a product candidate or technology.
+Added: Because patent applications are maintained as confidential for a certain period of time, until the relevant application is published, we may be unaware of third-party patents that may be infringed by commercialization of any of our product candidates, and we cannot be certain that we were the first to file a patent application related to a product candidate or technology.
Moreover, because patent applications can take many years to issue, there may be currently-pending patent applications that may later result in issued patents that our product candidates may infringe.
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We may also require licenses from third parties for certain technologies for use with future product candidates.
−Removed: We may be unable to acquire or in-license any compositions, methods of use, processes or other third-party intellectual property rights from third parties that we identify as necessary for our product candidates.
+Added: We may be unable to acquire or in-license any compositions, methods of use, processes or
+Added: other third-party intellectual property rights from third parties that we identify as necessary for our product candidates.
The licensing and acquisition of third-party intellectual property rights is a competitive area, and a number of 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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Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources.
−Removed: Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could compromise our ability to compete in the marketplace.
+Added: Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could
+Added: compromise our ability to compete in the marketplace.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation or other legal proceedings relating to our intellectual property rights, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation or other proceedings.
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If securities analysts or investors regard these announcements as negative, the perceived value of our existing products, programs or intellectual property could be diminished.
−Removed: Accordingly, the market price of shares of our
−Removed: common stock may decline.
+Added: Accordingly, the market price of shares of our common stock may decline.
Such announcements could also harm our reputation or the market for our future products, which could have a material adverse effect on our business.
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Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations.
−Removed: In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained.
+Added: In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this
+Added: combination of events has created uncertainty with respect to the value of patents, once obtained.
Depending on decisions by the U.S.
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Even if patents covering our product candidates are obtained, once the patent life has expired, we may be open to competition from competitive products.
−Removed: 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
−Removed: commercialized.
+Added: 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.
As a result, our patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
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Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United States or from selling or importing products made using our inventions in and into the United States or other jurisdictions.
−Removed: Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States.
+Added: Competitors may
+Added: use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States.
These products may compete with our product candidates, and our patents, the patents of any licensors, or other intellectual property rights may not be effective or sufficient to prevent them from competing.
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In these countries, the patent owner may have limited remedies, which could materially diminish the value of such patent.
−Removed: If we are forced to grant a license to third parties with respect to any patents
−Removed: relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations and prospects may be adversely affected.
+Added: If we are forced to grant a license to third parties with respect to any patents relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations and prospects may be adversely affected.
Geo-political actions in the United States and in foreign countries could increase the uncertainties and costs surrounding the prosecution or maintenance of our patent applications or those of any current or future licensors and the maintenance, enforcement or defense of our issued patents or those of any current or future licensors.
−Removed: For example, the United States and foreign government actions related to Russia’s invasion of Ukraine, which could be subject to change if there is a change in administration as a result of the upcoming U.S.
−Removed: presidential election, may limit or prevent filing, prosecution and maintenance of patent applications in Russia.
+Added: For example, the United States and foreign government actions related to Russia’s invasion of Ukraine, which could be subject to change as a result of the upcoming change in the U.S.
+Added: presidential administration , may limit or prevent filing, prosecution and maintenance of patent applications in Russia.
Government actions may also prevent maintenance of issued patents in Russia.
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Obtaining and maintaining our patent protection depends on compliance with various procedural, documentary, fee payment and other requirements imposed by regulations and governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
−Removed: Periodic maintenance fees, renewal fees, annuity fees and various other governmental fees on patents and/or applications will be due to the USPTO and various foreign patent offices at various points over the lifetime of our patents and/or applications and those of any licensors.
+Added: Periodic maintenance fees, renewal fees, annuity fees and various other governmental fees on patents and/or applications will be due to the USPTO and various foreign patent offices at various points over the lifetime of our
+Added: patents and/or applications and those of any licensors.
We have systems in place to remind us to pay these fees, and we rely on our outside patent annuity service to pay these fees when due.
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In addition, there could be potential trade name or trademark infringement claims brought by owners of other registered trademarks or trademarks that incorporate variations of our registered or unregistered trademarks or trade names.
−Removed: Over the long term, if we are unable to establish name recognition based on our trademarks and trade names, then we may not be able to compete
−Removed: effectively, and our business may be adversely affected.
+Added: Over the long term, if we are unable to establish name recognition based on our trademarks and trade names, then we may not be able to compete effectively, and our business may be adversely affected.
Our efforts to enforce or protect our proprietary rights related to trademarks, trade secrets, domain names, copyrights or other intellectual property may be ineffective and could result in substantial costs and diversion of resources and could adversely affect our financial condition or results of operations.
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We have entered into and may enter in the future into non-disclosure and confidentiality agreements to protect the proprietary positions of third parties, such as outside scientific collaborators, CROs, third-party manufacturers, consultants, advisors, potential partners, lessees of shared multi-company property and other third parties.
−Removed: We may become subject to litigation where a third-party asserts that we or our employees inadvertently or otherwise breached the agreements and used or disclosed trade secrets or other information proprietary to the third parties.
+Added: We may become subject to litigation where a third-party asserts that we or our employees inadvertently or otherwise
+Added: breached the agreements and used or disclosed trade secrets or other information proprietary to the third parties.
Defense of such matters, regardless of their merit, could involve substantial litigation expense and be a substantial diversion of employee resources from our business.
50 unchanged sentences
We have in-licensed patent applications that were generated through the use of U.S.
−Removed: government funding or grants, and may acquire or license in the future intellectual property rights that have been generated through the
+Added: government funding or grants, and may acquire or license in the future intellectual property rights that have been generated through the use of U.S.
government funding or grants.
24 unchanged sentences
Third parties have a significant role in the conduct of our clinical trials and the subsequent collection and analysis of data.
−Removed: These third parties are not our employees, and except for remedies available to us under our agreements with such third parties, we have limited ability to control the amount or timing of resources that any such third-party will devote to our clinical trials.
+Added: These third parties are not our employees, and except for remedies available to us under our agreements with such third
+Added: parties, we have limited ability to control the amount or timing of resources that any such third-party will devote to our clinical trials.
The third parties we rely on for these services may also have relationships with other entities, some of which may be our competitors.
9 unchanged sentences
In addition, our clinical trials must be conducted with product produced under current cGMP regulations.
−Removed: failure to comply with these regulations may require us to repeat clinical trials, which would delay the marketing approval process.
+Added: Our failure to comply with these regulations may require us to repeat clinical trials, which would delay the marketing approval process.
If these third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our clinical trials in accordance with regulatory requirements or our stated protocols, we will not be able to obtain, or may be delayed in obtaining, marketing approvals for our product candidates and will not be able to, or may be delayed in our efforts to, successfully commercialize our product candidates.
35 unchanged sentences
Despite the contractual provisions employed when working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade secrets become known by our competitors, are intentionally or inadvertently incorporated into the technology of others or are disclosed or used in violation of these agreements.
−Removed: Given that our proprietary position is based, in part, on our know-how and trade secrets and despite our efforts to protect our trade secrets, a competitor’s discovery of our proprietary technology and
−Removed: confidential information or other unauthorized use or disclosure would impair our competitive position and may have a material adverse effect on our business, financial condition, results of operations and prospects.
+Added: Given that our proprietary position is based, in part, on our know-how and trade secrets and despite our efforts to protect our trade secrets, a competitor’s discovery of our proprietary technology and confidential information or other unauthorized use or disclosure would impair our competitive position and may have a material adverse effect on our business, financial condition, results of operations and prospects.
If we engage in future acquisitions or strategic partnerships, this may increase our capital requirements, dilute our stockholders, cause us to incur debt or assume contingent liabilities, and subject us to other risks.
18 unchanged sentences
Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s evaluation of a number of factors.
−Removed: Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA, EMA or comparable foreign regulatory authorities, the potential market for the subject product candidate, the costs and complexities of
−Removed: manufacturing and delivering such product candidate to patients, the potential of competing drugs, the existence of uncertainty with respect to our ownership of intellectual property and industry and market conditions generally.
+Added: Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA, EMA or comparable foreign regulatory authorities, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing drugs, the existence of uncertainty with respect to our ownership of intellectual property and industry and market conditions generally.
The potential collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such collaboration could be more attractive than the one with us for our product candidate.
53 unchanged sentences
● the impact of any natural disasters or public health emergencies, such as the COVID-19 pandemic;
−Removed: ● general economic, political, industry and market conditions including those as a result of the upcoming U.S.
−Removed: presidential election and the impact of increasing inflation.
+Added: ● general economic, political, industry and market conditions including the impact of the change in the U.S.
+Added: presidential administration and the impact of increasing inflation.
The realization of any of the above risks or any of a broad range of other risks, including those described in this “Risk Factors” section, could have a dramatic and adverse impact on the market price of our common stock.
12 unchanged sentences
● the need to conduct unanticipated clinical trials or trials that are larger or more complex than anticipated;
−Removed: ● competition from existing and potential future products that compete with sevasemten , EDG-7500, product candidates from our EDG-003 cardiometabolic discovery program and any of our other product candidates or programs, and changes in the competitive landscape of our industry, including consolidation among our competitors or partners;
+Added: ● competition from existing and potential future products that compete with sevasemten , EDG-7500, product candidates from our EDG-003 cardiometabolic discovery program and any of our other product candidates
+Added: or programs, and changes in the competitive landscape of our industry, including consolidation among our competitors or partners;
● any delays in regulatory review or approval of sevasemten , EDG-7500, product candidates from our EDG-003 cardiometabolic discovery program or any of our other product candidates;
6 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 upcoming U.S.
−Removed: presidential election;
+Added: ● the changing and volatile global economic and political environment, including as a result of the upcoming change in the U.S.
+Added: presidential administration ;
● increased impact from COVID-19 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 June 30, 2024, our executive officers, directors, affiliated holders of 5% or more of our capital stock and their respective affiliates beneficially owned approximately 22.8% of our outstanding common stock.
+Added: As of September 30, 2024, our executive officers, directors, affiliated holders of 5% or more of our capital stock and their respective affiliates beneficially owned approximately 22.7% of our outstanding common stock.
These stockholders, acting together, may be able to control matters requiring stockholder approval.
1 unchanged sentence
This concentration of ownership control may delay, discourage or prevent a change of control, including unsolicited acquisition proposals or offers for our common stock that you may feel are in your best interest as one of our stockholders, entrench our management and board of directors or delay or prevent a merger, consolidation, takeover or other business combination involving us that other stockholders may desire.
−Removed: The interests of this group of stockholders may not always coincide with your interests or the interests of other stockholders and they may act in a manner that advances their best interests and not necessarily those of other stockholders, including seeking a premium value for their common stock, and might affect the prevailing market price for our common stock.
+Added: The interests of this group of stockholders may not always coincide with your
+Added: interests or the interests of other stockholders and they may act in a manner that advances their best interests and not necessarily those of other stockholders, including seeking a premium value for their common stock, and might affect the prevailing market price for our common stock.
Sales of a substantial number of shares of our common stock in the public market could cause our stock price to fall.
2 unchanged sentences
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
−Removed: 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, 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.
11 unchanged sentences
We have elected to avail ourselves of this exemption from new or revised accounting standards and, therefore, will not be subject to the same new or revised accounting standards as other public companies that are not emerging growth companies.
−Removed: As a result, our financial statements may not be comparable to companies that comply with the new or revised accounting pronouncements as of public company effective dates.
+Added: As a result, our financial
+Added: statements may not be comparable to companies that comply with the new or revised accounting pronouncements as of public company effective dates.
We cannot predict if investors will find our common stock less attractive because we may rely on these exemptions.
22 unchanged sentences
To achieve compliance with Section 404 within the prescribed period, we have engaged in a process to document and evaluate our internal control over financial reporting, which is both costly and challenging.
−Removed: In this regard, we will need to continue to dedicate internal resources, potentially engage outside consultants and adopt a detailed work plan to assess and document the adequacy of our internal control over financial reporting, continue steps to improve control processes as appropriate, validate through testing that controls are designed and operating effectively, and implement a continuous reporting and improvement process for internal control over financial reporting.
+Added: In this regard, we will need to continue to dedicate internal resources, potentially engage outside consultants and adopt a detailed work plan to assess and document the adequacy of our internal control over financial reporting, continue steps to improve control processes as appropriate, validate through testing that controls are designed and operating
+Added: effectively, and implement a continuous reporting and improvement process for internal control over financial reporting.
The rules governing the standards that must be met for management to assess our internal control over financial reporting are complex and require significant documentation, testing and possible remediation to meet the detailed standards under the rules.
2 unchanged sentences
A control system, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the control system’s objectives will be met.
−Removed: Because of the inherent limitations in all control systems, no evaluation of
−Removed: 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.
+Added: 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.
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.
45 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.