Content analysis
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H.S. sophomore Good
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New words:
abundance, apoB, beneficiary, biosimilar, Canadian, cap, compensatory, conference, constitute, duly, HHS, ISR, John, largest, lifting, longest, motion, NeuroBo, plc, postponed, readout, renal, reopen, repurchased, rulemaking, session, Stanley, thereunto, tie, undersigned, upcoming, video
Removed:
anniversary, Boston, BROADEN, commenced, comprised, constrained, demonstrating, Dublin, expanded, fat, feet, FPL, functionality, Gemphire, improvement, infection, landlord, launching, leasehold, leasing, lessor, list, Novelion, refused, satisfying, space, square, statistically, sublease, tenant, updated, urinary
Financial report summary
?Competition
AMGEN • Novelion Therapeutics • Regeneron Pharmaceuticals • Arrowhead Pharmaceuticals • Novartis • Alnylam Pharmaceuticals • Dicerna Pharmaceuticals • Arcturus Therapeutics • NeuroBo Pharmaceuticals • BridgeBio PharmaRisks
- We have a limited operating history and may never become sustainably profitable.
- We have experienced significant turnover in our top executives, and our business could be adversely affected by these and other transitions in our senior management team or if any of the resulting vacancies cannot be filled with qualified replacements in a timely manner.
- We have mostly incurred losses since our inception.
- We will require substantial additional funding to achieve our goals. If we fail to obtain timely funding, we may need to curtail or abandon some of our programs.
- If the results of clinical testing indicate that any of our medicines are not suitable for commercial use, we may need to abandon one or more of our drug development programs.
- Even if our medicines are successful in preclinical and earlier-stage clinical studies, the medicines may not be successful in later-stage clinical studies.
- We may not have appropriately designed the planned and ongoing clinical studies for our medicines in development to support submission of a marketing application to the FDA and foreign regulatory authorities or demonstrate safety or efficacy at the level required by the FDA and foreign regulatory authorities for product approval.
- Clinical studies for our medicines in development may not demonstrate safety or efficacy at the level required by the FDA and foreign regulatory authorities for product approval.
- If we or our partners fail to obtain regulatory approval for our medicines in development, or additional approvals for TEGSEDI and WAYLIVRA, we or our partners cannot sell them in the applicable markets.
- We may not be able to benefit from orphan drug designation for WAYLIVRA, TEGSEDI or any of our other medicines.
- We may expend our limited resources to pursue a particular medicine or indication and fail to capitalize on medicines or indications that may be more profitable or for which there is a greater likelihood of success.
- Our medicines could be subject to regulatory limitations following approval.
- The development and commercialization of TEGSEDI and WAYLIVRA may place strain on our management team’s time and attention and may divert our management team’s attention from our other existing products.
- If we cannot optimize and maintain effective marketing and sales capabilities or enter into agreements with third parties to market and sell TEGSEDI and WAYLIVRA, we may not generate significant product revenue from TEGSEDI or WAYLIVRA.
- If we are unable to rely on third-party specialty channels to distribute our medicines to patients we may be unable to generate adequate revenue.
- If the market does not accept our medicines, including TEGSEDI, WAYLIVRA and our medicines in development, we are not likely to generate substantial product revenue or become profitable.
- The patient populations suffering from FCS and FPL are small and have not been established with precision. If the actual number of patients is smaller than we estimate, or if we cannot raise awareness of these diseases and diagnosis is not improved, our revenue and ability to achieve profitability from WAYLIVRA may be adversely affected.
- If we or our partners fail to compete effectively, WAYLIVRA, TEGSEDI and our medicines in development will not contribute significant revenue.
- If government or other third-party payers fail to provide adequate coverage and payment rates for TEGSEDI, WAYLIVRA and our medicines in development, our revenue and prospects for profitability will be limited.
- Certain of our medicines may compete with our other medicines, which could reduce our expected revenues.
- If we cannot manufacture our medicines or contract with a third party to manufacture our medicines at costs that allow us to charge competitive prices to buyers, we will not be able to operate profitably.
- We depend on Ionis and third parties to conduct our clinical studies for our medicines and any failure of those parties to fulfill their obligations could adversely affect our development and commercialization plans.
- Ionis controls the direction of our business, and the concentrated ownership of our common stock will prevent you and other stockholders from influencing significant decisions.
- We intend to license additional medicines from Ionis but may not be successful in completing such licenses or on favorable terms, which may adversely affect our long-term strategy as a company and the value of our stock. If we are successful in completing additional licenses with Ionis, we could be exposed to additional risk of shareholder derivative litigation.
- If Ionis sells a controlling interest in our company to a third party in a private transaction, you may not realize a change of control premium on shares of our common stock, and we may become subject to the control of a presently unknown third party.
- Certain of our directors and officers may have actual or potential conflicts of interest because of their positions with Ionis.
- We will incur incremental costs as a standalone company.
- We may not be able to fully realize the expected benefits of our license agreements with Ionis.
- Risks Related to Our Intellectual Property
- If we breach our obligations under any of our license agreements with Ionis, we could lose our rights to WAYLIVRA, TEGSEDI and our other medicines in development.
- Intellectual property litigation could cause us to spend substantial resources and prevent us from pursuing our programs.
- We will not seek to protect our intellectual property rights in all jurisdictions throughout the world and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.
- If we do not obtain additional protection under the Hatch-Waxman Amendments and similar foreign legislation by extending the patent protection for TEGSEDI, WAYLIVRA and our medicines in development, our business may be materially harmed.
- Changes in United States patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
- If we and our partners do not adequately protect the trademarks and trade names for our products, then we and our partners may not be able to build name recognition in our markets of interest and our business may be adversely affected.
- Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
- Risks Related to Our Business and Industry
- We will need to optimize the size of our organization, and we may experience difficulties in managing optimization.
- If we do not progress in our programs as anticipated, the price of our securities could decrease.
- The loss of key personnel, or an inability to attract and retain highly skilled personnel, could make it more difficult to run our business and reduce our likelihood of success.
- We are exposed to potential product liability claims, and insurance against these claims may not be available to us at a reasonable rate in the future or at all.
- Because we use biological materials, hazardous materials, chemicals and radioactive compounds, if we do not comply with laws regulating the protection of the environment and health and human safety, our business could be adversely affected.
- A variety of risks associated with operating our business and marketing our medicines internationally could materially adversely affect our business.
- The impact on us of the vote by the United Kingdom to leave the European Union cannot be predicted.
- If a natural or man-made disaster strikes our development or manufacturing facilities or otherwise affects our business, it could delay our progress developing and commercializing our medicines.
- Our business and operations would suffer in the event of computer system failures.
- Risks Related to Our Common Stock
- We are an "emerging growth company" and as a result of the reduced disclosure and governance requirements applicable to emerging growth companies, our common stock may be less attractive to investors.
- The market price for our common stock may be volatile, which could contribute to the loss of your investment.
- Sales of a substantial number of shares of our common stock by our existing stockholders in the public market may cause our stock price to decline.
- We could be subject to additional tax liabilities.
- Our ability to use our net operating loss carryovers and certain other tax attributes may be limited.
- We could be subject to securities class action litigation.
- Provisions in our amended and restated certificate of incorporation, our amended and restated bylaws and Delaware law may have anti-takeover effects that could discourage an acquisition of us by others, even if an acquisition would be beneficial to our stockholders, and may prevent attempts by our stockholders to replace or remove our current management.
Management Discussion
- Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
- This financial review presents our operating results for each of the two years in the period ended December 31, 2019, and our financial condition at December 31, 2019. Year-to-year comparisons between 2018 and 2017 have been omitted from this Annual Report on Form 10-K, but may be found in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of our Annual Report on Form 10-K for the fiscal year ended December 31, 2018, which specific discussion is incorporated herein by reference. Except for the historical information contained herein, the following discussion contains forward-looking statements which are subject to known and unknown risks, uncertainties and other factors that may cause our actual results to differ materially from those expressed or implied by such forward-looking statements. We discuss such risks, uncertainties and other factors throughout this report and specifically under Item 1A of Part I of this report, "Risk Factors." In addition, the following should be read in connection with the information presented in our consolidated financial statements and the related notes to our consolidated financial statements as indexed on page F-1.
- We are a commercial stage biopharmaceutical company developing and marketing transformative medicines to treat patients with serious and rare diseases. Our large and potentially advancing pipeline of medicines in late-stage development and on the market allows us to capitalize on our strengths in supporting patients, healthcare professionals and caregivers in treating rare and serious diseases. To optimize the value of our medicines to treat larger patient populations, we add to our own capabilities the strengths of partners to provide additional expertise, resources and commercial capabilities. We believe our strong relationship with our majority shareholder, Ionis Pharmaceuticals, Inc., or Ionis, should allow us to continue to expand our pipeline.