▸ Enacted and future legislation or judicial action may increase the difficulty and cost for us to market our approved products or commercialize any other drug candidates we may acquire or license and affect the prices we may obtain.· · · · ● 1 ▸ If future reimbursement for our products is less than we project, or rebate obligations associated with them are substantially greater than we expect, our future net revenue and profitability could be materially diminished.· · · · ● 1 ▸ Our strategy of seeking to acquire or in-license innovative technical platforms or earlier stage drug development programs outside of the neuromuscular disease space may not be successful.· · · · ● 1 ▸ Tariffs could adversely affect our business.· · · · ● 1 ▸ The successful commercialization of our approved products or any we may develop will depend in part on the extent to which government authorities and health insurers establish adequate reimbursement levels and pricing policies.· · · · ● 1 ▸ We have been, and may continue to be, forced to litigate to enforce or defend our intellectual property, which could be expensive, time-consuming, and unsuccessful, and result in the loss of valuable assets.· · · · ● 1 ▸ If our suppliers were to be unable to supply us with adequate supply of our drugs, it could have a material adverse effect on our ability to successfully commercialize our drug candidates.· · · ● ● 2 ▸ group Risks Related to our Business and the Marketing of Approved Products· · · ● ● 2 rw ▸ Risks Related to the Development of Additional Drug Products and Indications· · · ● ● 2 ▸ The healthcare industry is highly regulated, subject to stringent regulatory standards and other applicable laws, and we may be the subject of unexpected changes in interpretation or enforcement, any of which may adversely impact our business.· · · ● ● 2 ▸ We anticipate being subject to increasing focus by our investors, regulators, customers, and stakeholders on environmental, social & governance (ESG) disclosures.· · · ● ● 2 rw ▸ We are subject to environmental, health, and safety laws and regulations, which could increase our costs or restrict our operations.· · · ● ● 2 ▸ Because of risks associated with taking FYCOMPA®, potential patients may be reluctant to start treatment with FYCOMPA® or may discontinue use.· · ● ● ● 3 ▸ Because the target patient population for certain of our products is small, we must achieve significant market share and obtain relatively high per-patient prices for our products to achieve meaningful gross margins.· · ● ● ● 3 rw ▸ Delaware law and our certificate of incorporation and by-laws contain provisions that could delay and discourage takeover attempts that stockholders may consider favorable.· · ● ● ● 3 ▸ If our pre-clinical studies or our clinical studies and trials are unsuccessful or significantly delayed, our ability to commercialize our products will be impaired.· · ● ● ● 3 ▸ If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection obtained is not sufficiently broad, we may not be able to compete effectively in our markets.· · ● ● ● 3 ▸ Our business and operations could suffer in the event of system failures or security or data breaches due to cyber-attacks, or cyber intrusions, including ransomware, phishing attacks and other malicious intrusions.· · ● ● ● 3 ▸ Our drug products may fail to receive the degree of market acceptance by physicians, patients, third-party payers or others in the medical community necessary for commercial success, which would negatively impact our business.· · ● ● ● 3 rw ▸ Our strategy of seeking to acquire or in-license innovative technical platforms or earlier stage drug development programs may not be successful.· · ● ● ● 3 rw ▸ group Risks Related to Government Regulation· · ● ● ● 3 ▸ group Risks Related to our Intellectual Property· · ● ● ● 3 ▸ Sales of shares, or the perception in the market that the holders of a large number of shares intend to sell shares, could reduce the market price of our common stock.· · ● ● ● 3 ▸ We are dependent on our licensing partner for supplies of AGAMREE® and FYCOMPA®.· · ● ● ● 3 rw ▸ We rely on third parties to conduct our pre-clinical studies and clinical studies and trials, and if they do not perform their obligations to us we may not be able to obtain approval for additional indications.· · ● ● ● 3 ▸ Pressure on drug product third-party payor coverage, reimbursement and pricing may impair our ability to be reimbursed at prices or on terms sufficient to provide a viable financial outcome.· ● ● ● ● 4 rw ▸ We could be impacted by the viability of our suppliers.· ● ● ● ● 4 ▸ In that regard, in January 2023, the FDA reported that while it is complying with the 11th Circuit decision in Catalyst’s favor with respect to FIRDAPSE®, going forward the FDA intends to continue to apply its regulations tying the scope of orphan· · · ● · 1 ▸ Changes to the ODA or successful legal challenges to the FDA’s interpretation of the ODA may affect our ability to obtain or subsequently maintain orphan drug exclusivity or affect the scope of orphan drug exclusivity for our products.· · ● · · 1 ▸ For all of these reasons, we may not be able to compete successfully.· · ● · · 1 ▸ group Index to Financial Statements· · ● · · 1 ▸ group Risks Related to the Development of Drug Products· · ● ● · 2 ▸ group Risks associated with our pending Paragraph IV litigation· · ● · · 1 ▸ group Risks related to Our Business· · ● ● · 2 ▸ We may fail to meet our publicly announced guidance or other expectations about our business and future operating results, which would cause our stock price to decline.· · ● · · 1 ▸ Our failure to develop safe, effective, and/or commercially viable products would have a material adverse effect on our business, prospects, results of operations and financial condition.· ● · · · 1 ▸ group Our strategy of seeking to acquire or· ● · · · 1 ▸ group The commercial success of our drug products, including FIRDAPSE· ● · · · 1 ▸ Under our License Agreements, we have the right to bring legal action against any alleged infringers of the patents we license. In that regard, in October 2020, we filed lawsuits against Jacobus and the specialty pharmacy marketing Ruzurgi· ● · · · 1 ▸ We cannot assess the impact on our business of the public concerns expressed by a U.S. Senator and a vocal group of neuromuscular physicians and patients with LEMS about the pricing of our product.· ● · · · 1 ▸ We cannot predict how any such laws or regulations, or new laws or regulations that have yet to be proposed, will affect the pricing of our product, of orphan drugs generally, or of pharmaceutical products generally.· ● ● ● · 3 rw ▸ group All of our patent rights for FIRDAPSE● ● · · · 2 ▸ Broad-based business or economic disruptions could adversely affect our ongoing or planned research and development activities. Global health concerns, such as the● ● · · · 2 ▸ group Certain provisions of our certificate of incorporation and● ● · · · 2 ▸ Commissioner Scott Gottlieb to allow pharmacies and manufacturers who were previously making this drug to be permitted to resume providing it.● · · · · 1 ▸ group Development of additional indications for Firdapse● · · · · 1 ▸ group Even with the FDA approval of FIRDAPSE● ● · · · 2 ▸ If these third-party tests are unsuccessful, or if they show significant health risk to the test subjects, our development efforts may also be adversely affected.● · · · · 1 ▸ If we are unable to successfully obtain rights to suitable product candidates or technologies, our business, financial condition and prospects for growth could suffer. In addition, acquisitions and● ● · · · 2 ▸ If we rely on a sole source of supply to manufacture our products we could be impacted by the viability of our supplier.● · · · · 1 ▸ group In other countries where FIRDAPSE● ● · · · 2 ▸ In the United States, orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages, and● ● · · · 2 ▸ group Our ability to commercialize Firdapse● · · · · 1 ▸ Our business involves a high degree of risk. You should carefully consider the risks and uncertainties described below, and all of the other information contained in this Form● ● · · · 2 ▸ group Our strategy is to continue to successfully commercialize FIRDAPSE● ● · · · 2 ▸ Our success depends on our ability to continue to successfully commercialize FIRDAPSE● ● · · · 2 ▸ The FDA and other regulatory authorities generally approve products for particular indications. Firdapse● · · · · 1 ▸ The Rights Plan was implemented through our entry into a rights agreement with Continental Stock Transfer & Trust Company, as rights agent, and the declaration of a● · · · · 1 ▸ The occurrence of any of these events could have a material adverse effect on our business, financial condition, results of operations or prospects.● ● · · · 2 ▸ The process of proposing, negotiating and implementing a license or acquisition of a product candidate is lengthy and complex, and we may be unable to● · · · · 1 ▸ There can be no assurance as to how these matters will affect our business or results of operations.● · · · · 1 ▸ We are dependent on our relationships and license agreements, and we rely upon the patent rights granted to us pursuant to the license agreements.● ● · · · 2 ▸ We are licensed in Florida as a virtual drug manufacturer, which means we have no● ● · · · 2 ▸ We cannot guarantee that the design of, or data collected from, that trial or any of our clinical trials conducted under a Special Protocol Assessment (SPA) will be sufficient to support filing or approval of an NDA.● · · · · 1 ▸ group We do not currently have the ability to independently conduct● ● · · · 2 ▸ group We may not be able to sufficiently● · · · · 1 ▸ group We will only obtain regulatory approval to commercialize Firdapse● · · · · 1 ▸ Business or economic disruptions or global health concerns could seriously harm our development efforts and increase our costs and expenses.● ● ● ● ● 5 ▸ Enacted and future legislation or judicial action may increase the difficulty and cost for us to commercialize FIRDAPSE®, FYCOMPA®, AGAMREE®, or any other drug candidates we may acquire or license and affect the prices we may obtain.● ● ● ● ● 5 rw ▸ Failure can occur at any stage of our drug development efforts.● ● ● ● ● 5 ▸ Future sales of our common stock may cause our stock price to decline.● ● ● ● ● 5 ▸ Our business is subject to substantial competition.● ● ● ● ● 5 ▸ Our business may require additional capital.● ● ● ● ● 5 rw ▸ Our employees, sales agents and consultants may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.● ● ● ● ● 5 ▸ Our internal computer systems, or those of our contract research organizations and other key vendors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs.● ● ● ● ● 5 ▸ Our success will depend significantly on our ability to operate without infringing the patents and other proprietary rights of third parties.● ● ● ● ● 5 ▸ The obligations incident to being a public company place significant demands on our management.● ● ● ● ● 5 ▸ The regulatory approval process is lengthy, and we may not be able to obtain all of the regulatory approvals required to manufacture and commercialize our drug products in which we are licensed to them.● ● ● ● ● 5 rw ▸ The trading price of the shares of our common stock has been and could in the future be highly volatile.● ● ● ● ● 5 ▸ There are also general risk factors relating to us that you should consider that relate to our business and to our common stock.● ● ● ● ● 5 rw ▸ We are highly dependent on our small number of key personnel and advisors.● ● ● ● ● 5 ▸ We do not intend to pay cash dividends on our common stock in the foreseeable future.● ● ● ● ● 5 ▸ We face a risk of product liability claims and may not be able to obtain adequate insurance.● ● ● ● ● 5 ▸ We may encounter difficulties in managing our growth, which would adversely affect our results of operations.● ● ● ● ● 5 ▸ We may face significant delays in our clinical studies and trials due to an inability to recruit patients for our clinical studies and trials or to retain patients in the clinical studies and trials we may perform.● ● ● ● ● 5 ▸ We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights.● ● ● ● ● 5 ▸ We will need to continue to develop and maintain distribution and production capabilities or relationships to be successful.● ● ● ● ● 5 
