48 unchanged sentences
Failure to obtain this necessary capital when needed may force us to delay, limit, or terminate our product development efforts or other activities.
−Removed: As of June 30 , 2020, our available cash, cash equivalents, and investments were $817.5 million.
+Added: As of September 30 , 2020, our available cash, cash equivalents, and investments were $765.5 million.
We expect we will likely need additional capital to continue to commercialize our products, and to develop and obtain regulatory approval for, and to commercialize, all of our product candidates.
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We cannot guarantee that any clinical studies will be conducted as planned or completed on schedule, if at all.
−Removed: A failure of one or more clinical studies can occur at any stage of testing, and our future clinical studies may not be successful.
+Added: A failure of one or more clinical studies can occur at any stage of testing, and our future clinical
+Added: studies may not be successful.
Product candidates that have shown promising results in early-stage clinical studies may still suffer significant setbacks in subsequent clinical studies.
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Results from investigator-sponsored studies or compassionate-use studies may not be confirmed in company-sponsored studies or may negatively impact the prospects for our programs.
−Removed: Additionally, given the nature of the rare diseases we are seeking to treat, we often have to devise newly-defined endpoints to be tested in our studies, which can lead to some subjectivity in interpreting study results and could result in regulatory agencies not agreeing with the validity of our endpoints, or our interpretation of the clinical data, and therefore denying approval.
+Added: Additionally, given the nature of the rare diseases we are seeking to treat, we often have to devise newly-defined endpoints to be tested in our studies, which can lead to some subjectivity in interpreting study results and could result in regulatory agencies not agreeing with the validity of our endpoints, or our interpretation of the clinical data, and therefore delaying or denying approval.
Given the illness of the patients in our studies and the nature of their rare diseases, we may also be required or choose to conduct certain studies on an open-label basis.
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In these circumstances, we work closely with the regulatory authorities to define the approval path and may have to qualify outcome measures as part of our development programs.
−Removed: Additionally, many of the disease states we are targeting, such as LC-FAOD, are highly heterogeneous in nature, which may impact our ability to determine the treatment benefit of our potential therapies.
+Added: Additionally, many of the disease states we are targeting are highly heterogeneous in nature, which may impact our ability to determine the treatment benefit of our potential therapies.
This lengthy and uncertain approval process, as well as the unpredictability of the clinical and nonclinical studies, may result in our failure to obtain regulatory approval to market any of our product candidates, or delayed regulatory approval, which would significantly harm our business, results of operations, and prospects.
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Additionally, the FDA, Health Canada, and the EMA have each expressed interest in further regulating biotechnology, including gene therapy and genetic testing.
−Removed: For example, the EMA, which governs the development of gene therapies in the EU and may issue new guidelines concerning the development and marketing authorization for gene therapy products, advocates a risk-based approach to the development of a gene therapy product.
+Added: For example, the EMA, which governs the development of gene therapies in the EU and
+Added: may issue new guidelines concerning the development and marketing authorization for gene therapy products, advocates a risk-based approach to the development of a gene therapy product.
Agencies at both the federal and state level in the United States, as well as U.S.
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Further, as one of the goals of Phase 1 and/or 2 clinical trials is to identify the highest dose of treatment that can be safely provided to study participants, adverse side effects, including serious adverse effects, have occurred in certain studies as a result of changes to the dosing regimen during such studies.
+Added: For instance, patients in our Phase 1/2 study of GTX-102 experienced a serious adverse event of lower extremity weakness believed to be related to local inflammation due to GTX-102 at the highest doses of the product.
Gene therapy product candidates using AAV vectors, like DTX301, have been associated with immunologic reaction to the capsid protein or gene at early time points after administration.
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Companion diagnostics are subject to regulation by FDA and similar regulatory authorities outside the United States as medical devices and require regulatory clearance or approval prior to commercialization.
−Removed: In the United States, companion diagnostics are
−Removed: cleared or approved through FDA’s 510(k) premarket notification or premarket approval, or PMA, process.
+Added: In the United States, companion diagnostics are cleared or approved through FDA’s 510(k) premarket notification or premarket approval, or PMA, process.
Changes in marketing approval policies during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for each submitted 510(k) premarket notification, PMA or equivalent application types in jurisdictions outside the United States, may cause delays in the approval, clearance or rejection of an application.
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House of Representatives with the goal of combating the increased costs of prescription drugs.
−Removed: Although a number of these proposals and other measures, including the four executive orders announced by the U.S.
−Removed: Presidential administration in July 2020 aimed to lower drug prices, may require additional authorization to become effective, Congress and the U.S.
+Added: Although a number of these proposals and other measures, including the executive orders announced by the U.S.
+Added: Presidential administration in July 2020 and September 2020 aimed to lower drug prices, may require additional authorization to become effective, Congress and the U.S.
Presidential administration have each indicated that it will continue to seek new legislative and/or administrative measures to control drug costs.
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If competitors are able to obtain marketing approval for biosimilars referencing our products, our products may become subject to competition from such biosimilars, with the attendant competitive pressure and consequences.
−Removed: Additional competitors could enter the market with generic versions of our small-molecule product candidates, which may result in a material decline in sales of Dojolvi or future small-molecule product candidates.
+Added: Competitors could enter the market with generic versions of Dojolvi or our small-molecule product candidates, which may result in a material decline in sales of affected products.
Under the Hatch-Waxman Act, a pharmaceutical manufacturer may file an abbreviated new drug application, or ANDA, seeking approval of a generic copy of an approved innovator product.
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Accordingly, competitors could file ANDAs for generic versions of Dojolvi, or 505(b)(2) NDAs that reference Dojolvi.
−Removed: If there are patents listed for Dojolvi in the Orange Book, those ANDAs and 505(b)(2) NDAs would be required to include a certification as to each listed patent indicating whether the ANDA applicant does or does not intend to challenge the patent.
−Removed: We cannot predict whether any patents issuing from our pending patent applications will be eligible for listing in the Orange Book, how any generic competitor would address such patents, whether we would sue on any such patents, or the outcome of any such suit.
+Added: For any patents listed for Dojolvi in the Orange Book, those ANDAs and 505(b)(2) NDAs would be required to include a certification as to each listed patent indicating whether the ANDA applicant does or does not intend to challenge the patent.
+Added: We cannot predict how any generic competitor would address such patents, whether we would sue on any such patents, or the outcome of any such suit.
We may not be successful in securing or maintaining proprietary patent protection for products and technologies we develop or license.
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Any of these events, including if we are required to initiate new or additional sites in response to such events, could require us to incur substantial increased expenses, delay the development and commercialization of our product candidates, delay the timing of anticipated data releases, and impact our operating results.
−Removed: The COVID-19 pandemic may also impact the timing of review of our submissions and discussions with regulatory agencies, such as the FDA, with respect to our product candidates.
+Added: The COVID-19 pandemic may also impact the timing of review of our submissions.
+Added: The FDA has delayed certain key meetings or discussions with us related to one of our product candidates, which may adversely impact the progression of our clinical study for such product candidate.
The pandemic has also significantly impacted our commercialization efforts for our products, including the launch of our recently approved product, Dojolvi.
Social distancing measures and travel limitations have prevented our field sales and medical teams from meeting with health care professionals, customers and patients in person and it has become increasingly difficult to maintain consistent contact with our current patients or identify new patients for our commercialized products and product candidates.
−Removed: Further, certain of our patients may experience interruptions in insurance coverage due to job loss or change in employment status due to the economic impact from the pandemic.
−Removed: Effects from government budgetary
−Removed: constraints, either in the United States or internationally, due to the economic impact of the pandemic, such as changes to state coverage rules under Medicaid programs in the United States, could also impact continued insurance coverage and reimbursement for our products.
+Added: Further, certain of our patients may experience interruptions in insurance coverage due to job loss or change in
+Added: employment status due to the economic impact from the pandemic.
+Added: Effects from government budgetary constraints, either in the United States or internationally, due to the economic impact of the pandemic, such as changes to state coverage rules under Medicaid programs in the United States, could also impact continued insurance coverage and reimbursement for our products.
Any of these events could impact our ability to commercialize our products and adversely affect our operating results and revenue .
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If members of our management and other key personnel in critical functions across our organization are unable to perform their duties or have limited availability due to illness from COVID-19, we may not be able to execute on our business strategy and/or our operations may be negatively impacted.
+Added: Further, despite the safety protocols we may implement, employees may still contract COVID-19 at the workplace if and when we ease restrictions to our facilities and our employees return to work on-site.
The magnitude of the adverse effect on our business operations will depend, in part, on the length and severity of the restrictions and other limitations on our ability to conduct our business in the ordinary course.
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If the associated research and development effort is abandoned, the related assets will be written-off and we will record a noncash impairment loss on our statement of operations.
−Removed: We have not recorded any impairments related to our intangible assets through the end of June 30, 2020 .
+Added: We have not recorded any impairments related to our intangible assets through the end of September 30, 2020 .
We may not be successful in our efforts to identify, license, discover, develop, or commercialize additional product candidates.
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As a result, increases or decreases in the stock price of Arcturus common stock will result in accompanying changes in the fair value of our investments and impact , and cause substantial volatility in, our operating results for the reporting period.
−Removed: For instance, the increases in fair value of the Arcturus investments recognized in the Condensed Consolidated Statements of Operations for the three and six months ended June 30 , 2020 were $ 95.2 million and $ 102.9 million, respectively, primarily due to the higher Arcturus stock price at June 30 , 2020 compared to December 31, 2019.
−Removed: Such gain in fair value of the investment resulted in net income for the three months ended June 30, 2020 and decreased the amount of our net loss for th e six months ended June 30 , 2020.
−Removed: If the Arcturus stock price had been lower at June 30 , 2020 compared to December 31, 2019, we may have reported a net loss for the quarter and an even greater net loss for the six months ended June 30, 2020.
+Added: For instance, the changes in fair value of the Arcturus investments recognized in the Condensed Consolidated Statements of Operations for the three and nine months ended September 30 , 2020 were a $ 11.5 million decrease and a $ 91.3 million increase , respectively, primarily due to the lower Arcturus stock price at September 30 , 2020 compared to June 30, 2020 and the higher Arcturus stock price at September 30, 2020 compared to December 31, 2019 , respectively .
+Added: The gain in fair value of the investment for the nine months ended September 30, 2020 decreased the amount of our net loss during that period and the loss in fair value of the investment for the three months ended September 30, 2020 increased the amount of our net loss during that period .
+Added: If the Arcturus stock price had been lower at September 30 , 2020 compared to December 31, 2019, we may have reported an even greater net loss for the three and nine months ended September 30, 2020.
As the fair value of our investment in Arcturus is dependent on the stock price of Arcturus, which has recently seen wide fluctuations, the value of our investments and the impact on our operating results may similarly fluctuate significantly from quarter to quarter and year to year such that period-to-period comparisons may not be a good indication of the future value of the investments and our future operating results .
46 unchanged sentences
Pursuant to our 2014 Incentive Plan, or the 2014 Plan, our management is authorized to grant stock options and other equity-based awards to our employees, directors, and consultants.
−Removed: At June 30 , 2020, 3,304,531 shares were available for future grants under the 2014 Plan.
+Added: At September 30 , 2020, 3,186,920 shares were available for future grants under the 2014 Plan.
Through January 1, 2024, the number of shares available for future grant under the 2014 Plan will automatically increase on January 1 of each year by the lesser of 2,500,000 shares or 4% of all shares of our capital stock outstanding as of December 31 of the prior calendar year, subject to the ability of our compensation committee to take action to reduce the size of the increase in any given year.
Pursuant to our 2014 Employee Stock Purchase Plan, or 2014 ESPP, eligible employees can acquire shares of our common stock at a discount to the prevailing market price.
−Removed: At June 30 , 2020, 3,287,825 shares were available for issuance under the 2014 ESPP.
+Added: At September 30 , 2020, 3,287,825 shares were available for issuance under the 2014 ESPP.
Through January 1, 2024, the number of shares available for issuance under the 2014 ESPP will automatically increase on January 1 of each year by the lesser of 1,200,000 shares or 1% of all shares of our capital stock outstanding as of December 31 of the prior calendar year, subject to the ability of our compensation committee to take action to reduce the size of the increase in any given year.
38 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.