REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 |
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
SHELL COMPANY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
Title of each class |
Trading Symbol |
Name of each exchange on which registered | ||
* | Not for trading, but only in connection with the registration of the American Depositary Shares. |
Accelerated filer ☐ |
Non-accelerated filer ☐ |
Emerging growth company |
U.S. GAAP ☐ | by the International Accounting Standards Board ☒ |
Other ☐ |
Auditor Firm ID: |
Auditor Name: Deloitte Statsautoriseret |
Auditor Location: | ||||||
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II-1 |
• | the timing or likelihood of regulatory filings and approvals for our product candidates; |
• | our expectations regarding the commercial availability of TransCon hGH, known by its brand name SKYTROFA (lonapegsomatropin-tcgd), in the United States and related patient support services; |
• | the commercialization of TransCon hGH and our other product candidates, if approved; |
• | our commercialization, marketing and manufacturing capabilities of TransCon Growth Hormone, or TransCon hGH, and our other product candidates and associated devices; |
• | the scope, progress, results and costs of developing our product candidates or any other future product candidates, and conducting preclinical studies and clinical trials; |
• | our pursuit of oncology as our second of three independent therapeutic areas of focus, and our development of a pipeline of product candidates related to oncology; |
• | our expectations regarding the potential market size and the size of the patient populations for TransCon hGH and our other product candidates, if approved for commercial use; |
• | our expectations regarding the potential advantages of TransCon hGH and our other product candidates over existing therapies; |
• | our ability to enter into new collaborations; |
• | our expectations with regard to the ability to develop additional product candidates using our TransCon technologies and file Investigational New Drug Applications, or INDs, or similar for such product candidates; |
• | our expectations with regard to the ability to seek expedited regulatory approval pathways for our product candidates, including the potential ability to rely on the parent drug’s clinical and safety data with regard to our product candidates; |
• | our expectations with regard to our current and future collaboration partners to pursue the development of our product candidates and file INDs or similar for such product candidates; |
• | our development plans with respect to TransCon hGH and our other product candidates; |
• | our ability to develop, acquire and advance product candidates into, and successfully complete, clinical trials; |
• | the implementation of our business model and strategic plans for our business, TransCon hGH and our other product candidates and technologies, including global commercialization strategies; |
• | the scope of protection we are able to establish and maintain for intellectual property rights covering TransCon hGH and our other product candidates; |
• | estimates of our expenses, future revenue, capital requirements, our needs for additional financing and our ability to obtain additional capital; |
• | our financial performance; |
• | developments and projections relating to our market conditions, competitors and industry; and |
• | the potential effects on our business of the worldwide COVID-19 pandemic. |
• | The global pandemic caused by COVID-19 could materially adversely impact our business, including our clinical trials, supply chain operation, regulatory timelines and commercial activities. |
• | We have a limited operating history and we may incur significant losses in the future, which makes it difficult to assess our future viability. |
• | We may require substantial additional financing to achieve our goals, and a failure to obtain this necessary capital when needed on acceptable terms, or at all, could force us to delay, limit, scale back or cease our commercialization activities, product development or any other or all operations. |
• | We are substantially dependent on the success of TransCon hGH and our other product candidates, which may not be successful in nonclinical studies or clinical trials, receive regulatory approval or be successfully commercialized. |
• | Clinical drug development involves a lengthy and expensive process with uncertain outcomes, and we may encounter substantial delays in our clinical studies. Furthermore, results of earlier studies and trials may not be predictive of results of future trials. |
• | Interim, “top-line” and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. |
• | Our sales and marketing efforts for TransCon hGH may not be effective and we may not be successful in our commercial efforts. |
• | Competition in the biotechnology and pharmaceutical industries is intense and our competitors may discover, develop or commercialize products faster or more successfully than us. If we are unable to compete effectively our business, results of operations and prospects will suffer. |
• | We rely on third-parties to manufacture our preclinical and clinical drug supplies, and we rely on third-parties to produce commercial supplies of TransCon hGH and its device components. |
• | Our operating results may vary significantly from period to period and these variations may be difficult to predict. |
• | The parent drug, drug substance, drug product and other components of TransCon hGH and our other product candidates are currently acquired from certain single-source suppliers. The loss of these suppliers, or their failure to supply could materially and adversely affect our business. |
• | The regulatory approval processes of the U.S. Food and Drug Administration, the European Medicines Agency and comparable authorities are lengthy, time consuming, and inherently unpredictable. If we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed. |
• | If we are sued for allegedly infringing intellectual property rights of third parties, it will be costly and time consuming, and an unfavorable outcome in such litigation could harm our business. |
Item 1 |
Identity of Directors, Senior Management and Advisers |
Item 2 |
Offer Statistics and Expected Timetable |
Item 3 |
Key Information |
A. |
Reserved. |
B. |
Capitalization and Indebtedness |
C. |
Reasons for the Offer and Use of Proceeds |
D. |
Risk Factors |
• | completing research and preclinical and clinical development of our product candidates; |
• | on our own, or together with our strategic collaboration partners, obtaining additional regulatory approvals for our products and product candidates; |
• | negotiating favorable terms of and entering into collaboration, licensing or other arrangements; |
• | our ability to commercialize or co-promote, and/or the ability of our collaboration partners to successfully commercialize, our products and product candidates; |
• | developing and sustaining a scalable manufacturing process for TransCon hGH and for any of our other product candidates, if approved, and establishing and maintaining supply and manufacturing relationships with third-parties that can conduct the process and provide adequate, in amount and quality, products to support clinical development and the market demand for TransCon hGH and for our other product candidates, if approved; |
• | obtaining market acceptance of TransCon hGH, and of our other product candidates, if approved, as viable treatment options; |
• | addressing any competing technological and market developments; |
• | identifying, assessing, acquiring, in-licensing and/or developing new product candidates; |
• | maintaining, protecting, and expanding our portfolio of intellectual property rights, including patents, trade secrets, and know-how, and our ability to develop, manufacture and commercialize our product candidates and products without infringing intellectual property rights of others; and |
• | attracting, hiring, and retaining qualified personnel. |
• | the manufacturing, selling and marketing costs associated with TransCon hGH and with our other product candidates, if approved, including the cost and timing of building our sales and marketing capabilities; |
• | the timing, receipt, and amount of sales of, or royalties on, TransCon hGH and any future products; |
• | the sales price and the availability of adequate third-party coverage and reimbursement for TransCon hGH and for our other product candidates, if approved; |
• | our ability to establish and maintain strategic partnerships, licensing or other arrangements and the financial terms of such agreements; |
• | our ability to collect payments which are due to us from collaboration partners (if any), which in turn is impacted by the financial standing of any such collaboration partners; |
• | the progress, timing, scope, results and costs of our preclinical studies and clinical trials and manufacturing activities for our product candidates, including the ability to enroll patients in a timely manner for clinical trials; |
• | the time and cost necessary to obtain regulatory approvals for our product candidates and the costs of post-marketing studies that could be required by regulatory authorities; |
• | the cash requirements of any future acquisitions or discovery of product candidates; |
• | the number and scope of preclinical and discovery programs that we decide to pursue or initiate; |
• | the potential acquisition and in-licensing of other technologies, products or assets; |
• | the time and cost necessary to respond to technological and market developments, including further development of our TransCon technologies; |
• | the achievement of development, regulatory and commercial milestones resulting in the payment to us from collaboration partners of contractual milestone payments and the timing of receipt of such payments, if any; |
• | our progress in the successful commercialization and co-promotion of TransCon hGH and of our other product candidates, if approved, and our efforts to develop and commercialize our other existing product candidates; and |
• | the costs of filing, prosecuting, maintaining, defending and enforcing any patent claims and other intellectual property rights, including litigation costs and the outcome of such litigation, including costs of defending any claims of infringement brought by others in connection with the development, manufacture or commercialization of our product candidates. |
• | the outcome and successful execution of our ongoing and planned clinical trials of TransCon hGH and our other product candidates; |
• | our ability and that of any collaboration partners to establish and maintain commercial-scale manufacturing processes for our products, product candidates and device components; |
• | whether our product candidates’ safety, purity, potency, tolerability and/or efficacy profiles will be satisfactory to the European Medicines Agency (“EMA”), the FDA and similar regulatory authorities to warrant marketing approval; |
• | whether the EMA, the FDA or similar regulatory authorities will require additional clinical trials prior to approving our product candidates, if ever; |
• | the prevalence and severity of adverse side effects of TransCon hGH and our other product candidates; |
• | the occurrence of adverse events that implicate the TransCon technologies, including among any out-licensed product candidates; |
• | the timely receipt of necessary marketing authorizations or certifications for our product candidates and associated device components from the FDA, similar regulatory authorities and notified bodies; |
• | our ability and that of any collaboration partners to successfully commercialize TransCon hGH or to commercialize our other product candidates, if approved for marketing and sale by the FDA or similar regulatory authorities, including educating physicians and patients about the benefits, administration and use of such products; |
• | achieving and maintaining compliance with all applicable regulatory requirements; |
• | acceptance of TransCon hGH, or our other product candidates, if approved, as safe and effective by patients and the medical community; |
• | acceptance of the device components of our combination products and combination product candidates, if approved, including the TransCon hGH auto-injector and the TransCon PTH drug delivery device and associated Bluetooth connectivity features, by patients and the medical community; |
• | the availability, perceived advantages, relative cost, relative safety and relative efficacy of alternative and competing treatments; |
• | obtaining and sustaining an adequate level of coverage and reimbursement for TransCon hGH and our other product candidates by third-party payors; |
• | the effectiveness of our and any collaboration partners’ marketing, sales and distribution strategies and operations; |
• | our ability and that of any collaboration partners, or any third-party manufacturer we contract with, to manufacture supplies of TransCon hGH and/or our other product candidates and to develop, validate and maintain commercially viable manufacturing processes that are compliant with current good manufacturing practice, or cGMP, or similar requirements; |
• | enforcing intellectual property rights in and to TransCon hGH and our other product candidates; |
• | avoiding third-party interference, opposition, derivation or similar proceedings with respect to our patent rights, and avoiding other challenges to our patent rights and patent infringement claims; and |
• | continued acceptable safety profiles of TransCon hGH and of our other product candidates following any potential approval. |
• | generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation or continuation of clinical trials; |
• | reach consensus with regulatory authorities on study design or implementation of the clinical trials and/or obtain regulatory authorization to commence a trial; |
• | reach agreement on acceptable terms with prospective contract research organizations (“CROs”) and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; |
• | identify, recruit and train suitable clinical investigators; |
• | obtain institutional review board (“IRB”) or ethics committee approval at each site; |
• | manufacture, test, release, validate or import sufficient quantities of drug product for use in a trial; |
• | recruit, screen and enroll suitable patients to participate in a trial; |
• | have patients complete a trial or return for post-treatment follow-up; |
• | ensure that clinical sites observe trial protocol or continue to participate in a trial; |
• | address any patient safety concerns that arise during the course of a trial; |
• | address any conflicts with new or existing laws or regulations; or |
• | initiate or add a sufficient number of clinical trial sites. |
• | the research methodology used and our TransCon technologies may not be successful in creating potential product candidates; |
• | competitors may develop alternatives that render our product candidates obsolete or less attractive; |
• | product candidates we develop may nevertheless be covered by third-parties’ intellectual property rights or other types of exclusivity and we may not be able to obtain a license from such third-party or the license terms may not be acceptable to us; |
• | the market for a product candidate may change during our program or we may discover that such market was smaller than initially expected so that such a product may become financially unfeasible to continue to develop; |
• | a product candidate may be demonstrated to have harmful side effects or not to be effective, or otherwise not to meet other requirements for regulatory approval; |
• | a product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; and |
• | a product candidate may not be accepted as safe and effective by patients, the medical community or third-party payors, or reimbursable by third-party payors, if applicable. |
• | the safety, purity, potency and/or efficacy of the products as demonstrated in clinical trials; |
• | the prevalence and severity of any side effects and overall safety profile of the product; |
• | the perceived safety of the TransCon technologies; |
• | the convenience and features of the auto-injector or drug delivery device used to administer the drug; |
• | the clinical indications for which the product is approved; |
• | acceptance by physicians, major operators of clinics and patients of the product as a safe and effective treatment and their willingness to pay for them; |
• | relative convenience and ease of administration of our products; |
• | the potential and perceived advantages of our product candidates over current treatment options or alternative treatments, including future alternative treatments; |
• | the availability of supply of our products and their ability to meet market demand; |
• | marketing and distribution support for our product candidates; |
• | the quality of our relationships with patient advocacy groups; and |
• | coverage and reimbursement policies of government and other third-party payors. |
• | regulatory authorities may withdraw their approval of the product or seize the product; |
• | we, or any collaboration partners, may be required to recall the product; |
• | additional restrictions may be imposed on the marketing of the particular product or the manufacturing processes for the product or any component thereof, including the imposition of a REMS or requirements for similar actions, such as patient education, certification of health care professionals or specific monitoring; |
• | we, or any collaboration partners, may be subject to fines, injunctions or the imposition of civil or criminal penalties; |
• | regulatory authorities may require additional warnings on the label, including “boxed” warnings, or issue safety alerts, Dear Healthcare Provider letters, press releases or other communications containing warnings or other safety information about the product; |
• | we could be sued and held liable for harm caused to patients; |
• | the product may become less competitive; and |
• | our reputation may suffer. |
• | filing new formulation patent applications on drugs whose original patent protection is about to expire; |
• | filing an increasing number of patent applications that are more complex and costly to challenge; |
• | filing suits for alleged patent infringement that automatically delay FDA or foreign regulatory authorities approval; |
• | developing patented controlled-release or other “next-generation” products, which may compete with TransCon product candidates; |
• | establishing exclusive contracts with third-party payors; or |
• | changing product claims and product labeling. |
• | significantly greater name recognition, financial, marketing, research, drug development and technical and human resources than we have at every stage of the discovery, development, manufacturing and commercialization process and additional mergers and acquisitions in the biotechnology industries may result in even more resources being concentrated in our competitors; |
• | more extensive experience in commercializing drugs, conducting preclinical testing, conducting clinical studies, obtaining regulatory approvals, challenging patents and in manufacturing and marketing pharmaceutical products; |
• | products that have been approved or are in late stages of development; and |
• | collaboration arrangements in our target markets with leading companies and research institutions. |
• | the safety and effectiveness of such product candidates; |
• | the timing of and specific circumstances relating to regulatory approvals for these product candidates; |
• | the availability and cost of manufacturing, marketing and sales capabilities; |
• | the effectiveness of our marketing and sales capabilities; |
• | the price of our product candidates; |
• | the availability and amount of third-party reimbursement for our product candidates; and |
• | the strength of our patent position. |
• | our ability to hire and retain required sales and marketing personnel; |
• | our ability to provide sufficient training to develop and strengthen the technical expertise of our sales and marketing personnel; |
• | our ability to provide required support materials and resources to our sales personnel to help them educate physicians and healthcare providers regarding TransCon hGH and its proper administration; and |
• | our resources to meet and timely fulfill supply obligations to our customers. |
• | our collaboration partners have the unilateral ability to choose not to develop a collaboration product for one or more indications for which such product has been or is currently being evaluated, and our collaboration partners may choose to pursue an indication that is not in our strategic best interest or to forego an indication that they believe does not provide significant market potential even if clinical data is supportive of further development for such indication; |
• | our collaboration partners may choose not to develop and commercialize our collaboration products in certain relevant markets; |
• | our collaboration partners may take considerably more time advancing our product candidates through the clinical and regulatory process than we currently anticipate, which could materially delay the achievement of milestones and, consequently the receipt of milestone payments from our collaboration partners; |
• | our collaboration partners have substantial discretion under their respective agreements regarding how they structure their efforts and allocate resources to fulfill their obligations to diligently develop, obtain regulatory approval for and commercialize our collaboration products; |
• | our collaboration partners control all aspects of commercialization efforts under their respective license agreements and may change the focus of their development and commercialization efforts or pursue higher-priority programs and, accordingly, reduce the efforts and resources allocated to their collaborations with us; |
• | our collaboration partners are solely responsible for obtaining and maintaining all regulatory approvals and we or our collaboration partners may fail to develop a commercially viable formulation or manufacturing process for our product candidates, and we or our collaboration partners may fail to manufacture or supply sufficient drug substance for commercial use, if approved, which could result in lost revenue under such collaborations; |
• | our collaboration partners may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements; |
• | if any of our agreements with our collaboration partners terminate, we will no longer have any rights to receive potential revenue under such agreement, in which case we would need to identify alternative means to continue the development, manufacture and commercialization of the affected product candidates, alone or with others; |
• | our collaboration partners have the discretion to sublicense their rights with respect to our collaboration technology in connection with collaboration product candidates to one or more third-parties without our consent; and |
• | our collaboration partners may be pursuing alternative technologies or developing alternative products, either on their own or in collaboration with others, that may be competitive with products on which they are collaborating with us or which could affect our collaboration partners’ commitment to the collaboration. |
• | production yields; |
• | quality control and assurance; |
• | shortages of qualified personnel; |
• | compliance with FDA and foreign regulations; |
• | production costs; and |
• | development of advanced manufacturing techniques and process controls. |
• | decreased demand for TransCon hGH and our other product candidates; |
• | injury to our reputation; |
• | withdrawal of clinical trial participants; |
• | costs to defend the related litigation; |
• | a diversion of management’s time and our resources; |
• | substantial monetary awards to trial participants or patients; |
• | regulatory investigations, product recalls or withdrawals, or labeling, marketing or promotional restrictions; |
• | loss of revenue; and |
• | the inability to commercialize or co-promote TransCon hGH or our other product candidates. |
• | expand our general and administrative functions; |
• | identify, recruit, retain, incentivize and integrate additional employees; |
• | manage our internal development efforts effectively while carrying out our contractual obligations to third-parties; |
• | establish and build a marketing and commercial organization; and |
• | continue to improve our operational, legal, financial and management controls, reporting systems and procedures. |
• | the timing of regulatory approvals, if any, for our product candidates; |
• | the amount and timing of revenue from product sales; |
• | the initiation of intellectual property litigation by third-parties or by us; |
• | the amount and timing of operating costs and capital expenditures relating to the expansion of our business operations and facilities; |
• | the timing of the commencement, completion or termination of collaboration agreements; |
• | the timing and amount of payments to us under collaboration agreements, if any; |
• | the introduction of new products and services by us, collaboration partners or our competitors; |
• | delays in preclinical testing and clinical studies; |
• | changes in regulatory requirements for clinical studies; |
• | costs and expenses associated with preclinical testing and clinical studies; and |
• | payment of license fees for the right to use third-party proprietary rights, if any. |
• | up-front, milestone and royalty payments, equity investments and financial support of new research and development candidates including increase of personnel, all of which may be substantial; |
• | exposure to unknown liabilities, including potential indemnification claims from a potential spin-off or out-license of certain of our intellectual property rights; |
• | disruption of our business and diversion of our management’s time and attention to develop acquired products, product candidates or technologies; |
• | incurrence of substantial debt or dilutive issuances of equity securities to pay for acquisitions; |
• | higher-than-expected acquisition and integration costs; |
• | lower-than-expected benefits, from out-licensing or selling our technology, intellectual property or any of our subsidiaries or, from in-licensing intellectual property or purchasing assets; |
• | write-downs of assets or goodwill or impairment charges; |
• | difficulty and cost in combining or separating the operations and personnel of any acquired or sold businesses with our existing operations and personnel; |
• | impairment of relationships with key suppliers or customers of any acquired or sold businesses due to changes in our senior management and ownership; and |
• | inability to retain key employees of any acquired businesses. |
• | different regulatory requirements for drug approvals in foreign countries; |
• | differing drug import and export rules; |
• | reduced protection for intellectual property rights in foreign countries; |
• | unexpected changes in tariffs, trade barriers and regulatory requirements; |
• | different reimbursement systems, and different competitive drugs; |
• | economic weakness, including inflation, or political instability in particular foreign economies and markets; |
• | compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; |
• | foreign taxes, including withholding of payroll taxes; |
• | foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country; |
• | workforce uncertainty in countries where labor unrest is more common than in the United States; |
• | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; |
• | potential liability resulting from work conducted by these distributors; |
• | regulatory and compliance risks that relate to maintaining accurate information and control over sales and activities that may fall within the purview of the Foreign Corrupt Practices Act, its books and records provisions, or its anti-bribery provisions; and |
• | business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters. |
• | the research methodology used may not be successful in identifying potential product candidates; or |
• | potential product candidates may, on further study, be shown to have inadequate efficacy, harmful side effects or other characteristics suggesting that they are unlikely to be effective or safe products, or that they may not be sufficiently differentiated or offer substantial improvement over the currently available treatment options or standard of care in a given therapeutic category. |
• | delays or difficulties in enrolling and retaining patients in our clinical trials, which could potentially have a negative impact on clinical trial timelines; |
• | delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff; |
• | significant increases in expenses required to manage impacts to our business to complete our planned operations within our projected timelines; |
• | diversion of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; |
• | interruption of key clinical trial activities, such as clinical trial site monitoring, due to limitations on travel imposed or recommended by federal or state governments, employers and others; |
• | limitations in employee resources that would otherwise be focused on the conduct of our clinical trials and commercialization activities, including because of sickness of employees or their families or the desire of employees to avoid contact with large groups of people; |
• | delays in receiving approval from local regulatory authorities to initiate our planned clinical trials; |
• | delays in clinical sites receiving the supplies and materials needed to conduct our clinical trials; |
• | interruption in global storage and shipping that may affect the supply of TransCon hGH or the transport of clinical trial materials, such as comparator drugs used in certain of our clinical trials; |
• | interruptions in our global supply chain with regards to clinical trials and commercial supply; |
• | changes in local regulations as part of a response to the COVID-19 outbreak which may require us to change the ways in which our clinical trials are conducted, which may result in unexpected costs, or to discontinue the clinical trials altogether; |
• | delays in necessary interactions with local regulators, ethics committees and other important agencies and contractors due to limitations in employee resources or forced furlough of government employees; |
• | refusal of regulatory authorities to accept data from clinical trials in these affected geographies; |
• | in conducting our clinical trials, suppliers may experience delays in providing necessary equipment, consumables and services, which could cause temporary delays in clinical trial activities; |
• | global demand for COVID-19 vaccines or COVID-19 therapeutics could result in contract manufacturers not having sufficient capacity to meet scheduled manufacturing. In addition, sourcing of certain types of raw materials, consumables and equipment could result in scheduled manufacturing being delayed or postponed; |
• | travel restrictions and local outbreaks of COVID-19 could restrict authorities from performing site inspections in connection with their review procedures of marketing applications for TransCon hGH, which could potentially delay the commercial launch in areas outside of the United States; and |
• | our commercial launch strategy, including for TransCon hGH, could be negatively impacted by patients not being able to see their physicians, and similarly, our commercial team not being able to meet in-person with physicians and payors, which could both have a negative impact on the commercial launch strategy. |
• | warning letters; |
• | civil and criminal penalties; |
• | injunctions; |
• | withdrawal of regulatory approval of products; |
• | product seizure or detention; |
• | product recalls; |
• | total or partial suspension of production; and |
• | refusal to approve pending NDAs or BLAs, MAA, or supplements to approved NDAs or BLAs or extensions or variations to marketing authorizations. |
• | the EMA, the FDA or other comparable foreign regulatory authorities may disagree with the design or implementation of our, or any collaboration partners’, clinical studies; |
• | the population studied in the clinical program may not be sufficiently broad or representative to assure safety in the full population for which approval is sought; |
• | the EMA, the FDA or comparable foreign regulatory authorities may disagree with the interpretation of data from preclinical studies or clinical studies; |
• | the data collected from clinical studies of our product candidates may not be sufficient to support the submission of an NDA or BLA, MAA, or other submission or to obtain regulatory approval in the United States, the EU or elsewhere; |
• | we, or any collaboration partners, may be unable to demonstrate to the EMA, the FDA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio for its proposed indication is acceptable; |
• | the FDA or comparable foreign regulatory authorities may fail to approve the manufacturing processes, test procedures and specifications, or facilities of third-party manufacturers responsible for clinical and commercial supplies; and |
• | the approval policies or regulations of the FDA or comparable foreign regulatory authorities may significantly change in a manner rendering our clinical data insufficient for approval. |
• | warning letters, fines or holds on clinical trials; |
• | restrictions on the marketing or manufacturing of the product, withdrawal of the product from the market or voluntary or mandatory product recalls; |
• | injunctions or the imposition of civil or criminal penalties; |
• | suspension or revocation of existing regulatory approvals; |
• | suspension of any of our future or ongoing clinical trials; |
• | refusal to approve pending applications or supplements to approved applications submitted by us; |
• | restrictions on our or our contract manufacturers’ operations; or |
• | product seizure or detention, or refusal to permit the import or export of products. |
• | the U.S. Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, directly or indirectly, in exchange for or to induce either the referral of an individual for, or the purchase, order or recommendation of, any good or service for which payment may be made under U.S. federal healthcare programs such as the Medicare and Medicaid programs. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it to have committed a violation; |
• | U.S. false claims laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid, or other third-party payors that are false or fraudulent. In addition, the government may assert that a claim including items or services resulting from a violation of the U.S. federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act; |
• | U.S. federal criminal laws that prohibit executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; |
• | the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”), which imposes criminal and civil liability for, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program, or knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement, in connection with the delivery of, or payment for, healthcare benefits, items or services; similar to the U.S. federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; |
• | the U.S. federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics, and medical supplies to report annually to the CMS information related to payments and other transfers of value to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), certain non-physician practitioners (physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, anesthesiologist assistants and certified nurse midwives) and teaching hospitals, and ownership and investment interests held by physicians (as defined under statute) and their immediate family members; |
• | state law equivalents of each of the above U.S. federal laws, such as anti-kickback and false claims laws which may apply to items or services reimbursed by any third-party payor, including commercial insurers; |
• | state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the applicable compliance guidance promulgated by the U.S. federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; |
• | state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information; and |
• | additional clinical trials to be conducted prior to obtaining approval; |
• | changes to manufacturing methods; |
• | recall, replacement, or discontinuance of one or more of our products; and |
• | additional record keeping. |
• | protect our trade secrets; |
• | apply for, obtain, maintain and enforce patents; and |
• | operate without infringing upon the proprietary rights of others. |
• | we might not have been the first to invent or the first to file the inventions covered by each of our pending patent applications and issued patents; |
• | others may independently develop similar or alternative technologies or duplicate any of our technologies; |
• | the patents of others may have an adverse effect on our business; |
• | any patents we obtain or our in-licensed issued patents may not encompass commercially viable products, may not provide us with any competitive advantages or may be challenged by third-parties; |
• | any patents we obtain or our in-licensed issued patents may not be valid or enforceable; or |
• | we may not develop additional proprietary technologies that are patentable. |
• | protect and enforce our patents and any future patents issuing on our patent applications; |
• | enforce or clarify the terms of the licenses we have granted or may be granted in the future; |
• | protect and enforce trade secrets, know-how and other proprietary rights that we own or have licensed, or may license in the future; or |
• | determine the enforceability, scope and validity of the proprietary rights of third-parties and defend against alleged patent infringement. |
• | our ability to commercialize or obtain regulatory approval for TransCon hGH or our other product candidates, or delays in commercializing or obtaining regulatory approval; |
• | results from, or any delays in, clinical trial programs relating to TransCon hGH or our other product candidates; |
• | the effects on our business, operating results, prospects and financial condition of the worldwide COVID-19 pandemic; |
• | our ability to apply our TransCon technologies to therapeutic areas other than endocrinology, including the therapeutic area of oncology; |
• | announcements of regulatory approval or a complete response letter to our product candidates, or specific label indications or patient populations for use, or changes or delays in the regulatory review process; |
• | announcements relating to current or future collaborations or joint ventures; |
• | announcements of therapeutic innovations or new products by us or our competitors; |
• | announcements regarding the parent drugs that we use in developing our product candidates; |
• | adverse actions taken by regulatory authorities with respect to our clinical trials, manufacturing supply chain or sales and marketing activities; |
• | changes or developments in laws or regulations applicable to TransCon hGH or our other product candidates; |
• | any adverse changes to our relationship with any manufacturers or suppliers; |
• | the success of our testing and clinical trials; |
• | the success of our efforts to acquire, license or discover additional products or product candidates; |
• | any intellectual property infringement actions in which we may become involved; |
• | announcements concerning our competitors or the pharmaceutical industry in general; |
• | achievement of expected product sales and profitability; |
• | manufacture, supply or distribution shortages; |
• | actual or anticipated fluctuations in our operating results; |
• | EMA, FDA or other similar regulatory actions affecting us or our industry or other healthcare reform measures in the European Union, United States or in other markets; |
• | changes in the structure of healthcare payment systems; |
• | changes in financial estimates or recommendations by securities analysts; |
• | trading volume of the ADSs; |
• | sales or purchases of ordinary shares and/or ADSs by us, our senior management and board members, holders of the ADSs or our shareholders in the future; |
• | general economic and market conditions and overall fluctuations in the United States and international equity markets, including deteriorating market conditions due to investor concerns regarding inflation and hostilities between Russia and Ukraine; and |
• | the loss of any of our key scientific or senior management personnel. |
Item 4 |
Information on the Company |