Autolus Therapeutics plc

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We may not obtain or maintain the benefits associated with orphan drug designation, including market exclusivity or favorable pricing.

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Obe-cel in B-ALL received orphan drug designation from the FDA in November 2019 and orphan medicinal product status from the European Commission in March 2022. We may seek orphan drug designation for other indications or product candidates. Even if we were to obtain orphan drug designation for a product candidate, we may not obtain orphan exclusivity and that exclusivity may not effectively protect the drug from the competition of different drugs for the same condition, which could be approved during the exclusivity period. Additionally, after an orphan drug is approved, the FDA could subsequently approve another application for the same drug for the same indication if the FDA concludes that the later drug is shown to be safer, more effective or makes a major contribution to patient care.

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Orphan drug exclusive marketing rights may be lost if the FDA or European Commission (on the basis of the opinion of the EMA) later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the drug to meet the needs of patients with the rare disease or condition. In the EU, alongside applying for marketing authorization, the applicant must submit a report to European Medicine Agency's Committee for Orphan Medicinal Products (“COMP”) demonstrating that the criteria for orphan designation are still met; if they are not, the medicine loses its orphan status. An applicant can choose to withdraw their application for orphan designation in the EU at any point before the COMP adopts an opinion. The failure to obtain an orphan drug designation for any product candidates we may develop, the inability to maintain that designation for the duration of the applicable period, or the inability to obtain or maintain orphan drug exclusivity could reduce our ability to make sufficient sales of the applicable product candidate to balance our expenses incurred to develop it, which would have a negative impact on our operational results and financial condition. For example, based on feedback from the COMP and the results of the latest COMP assessment, in June 2025, we voluntarily withdrew obe-cel from the EU register of orphan medicinal products. Following a thorough evaluation, we have determined not to proceed with the launch of AUCATZYL in Europe in the near term, in part due to the difficulty of achieving a commercially viable price in the absence of orphan drug designation.

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Our products and product candidates are subject to government price controls in certain jurisdictions that may affect our ability to ability to receive adequate coverage and reimbursement for AUCATZYL, which could make it difficult for us to sell AUCATZYL profitably.

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The current Trump U.S. presidential administration is pursuing policies to reduce regulations and expenditures across government including at the U.S Department of Health and Human Services (“HHS”), the FDA, the CMS and related agencies. These actions, presently directed by executive orders or memoranda from the Office of Management and Budget, may propose policy changes that create additional uncertainty for our business. For example, on September 30, 2025, the current Trump administration announced the first agreement with a major pharmaceutical company that requires the drug manufacturer to offer, through a direct to consumer platform, U.S. patients and Medicaid programs prescription drug Most-Favored Nation pricing equal to or lower than those paid in other developed nations, with additional mandates for direct-to-patient discounts and repatriation of foreign revenues. Other recent actions and proposals include, for example, (1) reducing agency workforce; (2) directing program cuts; (3) rescinding a Biden administration executive order tasking the Center for Medicare and Medicaid Innovation, or CMMI, to consider new payment and healthcare models to limit drug spending and eliminating the Biden administration’s executive order that directed the HHS to establishing an AI task force and developing a strategic plan; (4) directing the HHS and other agencies to lower prescription drug costs for Medicare through a variety of initiatives, including by improving upon the Medicare Drug Price Negotiation Program and establishing Most-Favored-Nation pricing for pharmaceutical products; (5) imposing tariffs of imported pharmaceutical products; (6) directing certain federal agencies to enforce existing law regarding hospital and plan price transparency and by standardizing prices across hospitals and health plans; and (7) as part of the Make America Healthy Again (MAHA) Commission’s recent Strategy Report, working across government agencies to increase enforcement on direct-to-consumer pharmaceutical advertising. These actions and policies may significantly reduce U.S. drug prices, potentially impacting manufacturers’ global pricing strategies and profitability, while increasing their operational costs and compliance risks. On April 1, 2025, the CMS published Healthcare Common Procedure Coding System (“HCPCS”) application summaries and coding determinations, as well as the Outpatient Prospective Payment System (“OPPS”) Addendum B payment rates. AUCATZYL was included in these CMS publications, formalizing reimbursement for patients on government programs such as Medicare and Medicaid. The CMS policy splits the therapeutic dose of AUCATZYL into two administrations for coding and billing purposes. Because AUCATZYL is administered in two infusions approximately ten days apart, the CMS policy to bill each infusion separately may delay our and our treatment centers’ ability to recognize revenue from a particular treatment. We are working with our authorized trea…

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We operate in a global economy, and our business depends on a global supply chain for the development, manufacturing, and distribution of our pharmaceutical products, and for the advancement of our preclinical and clinical development programs. There is inherent risk, based on the complex relationships among the U.S. and the countries in which we conduct our business, that political, diplomatic, and national security factors can lead to global trade restrictions and changes in trade policies and export regulations that may adversely affect our business and operations. The current international trade and regulatory environment is subject to significant ongoing uncertainty. We source significant quantities of biological materials, active pharmaceutical ingredients (“API”), precursor chemicals, and specialized equipment which are critical to the manufacture of our CAR T products from international suppliers, with substantial reliance on foreign manufacturers. In addition, we manufacture all of our commercial supplies of AUCATZYL and clinical supplies of our other product candidates in the United Kingdom. Tariff policies, particularly those affecting pharmaceutical products, could materially increase our costs of goods and/or costs of manufacture and reduce our profitability, including as a result of our inability to adjust pricing in formulary-based markets. Recent and potential future changes in international trade policies, particularly regarding pharmaceutical-specific tariffs, present material risks to our operations and financial performance. Even if our own products are not directly subject to tariffs, the administration of a complex, rapidly changing tariff scheme by limited government personnel at the various U.S. ports of entry may prove challenging, and may create delays in our provision of commercial or clinical supply.

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Recent policy discussions have included potential targeted tariffs or other trade measures specifically aimed at pharmaceutical products and ingredients as part of broader healthcare cost control or national security initiatives. Unlike consumer goods, pharmaceuticals face unique regulatory constraints that make rapid supply chain adjustments particularly difficult and costly. Should the current tariffs hold or additional tariffs be imposed specifically targeting pharmaceutical imports, our production costs could rise significantly, and it would be difficult and costly to qualify alternative sources within another country with a lower tariff rate or within the United States, as developing and qualifying alternative sources typically requires at least 18-24 months and substantial investment and regulatory approvals. Moreover, the dynamic and unpredictable tariff and trade landscape creates substantial uncertainty and significant planning challenges for our operations.

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Changes in tariff classifications, country-of-origin requirements, or customs procedures can occur with limited notice. This uncertainty complicates our long-term investment decisions regarding manufacturing facilities, supply chain optimization, and research and development locations.

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Unlike many industries, our ability to pass increased costs to customers is limited by the structure of pharmaceutical pricing and reimbursement systems. Many of our products are included in formularies with pricing established through annual or multi-year contracts with commercial, third-party payors and pharmacy benefit managers, and reimbursement methodologies established by government programs, such as Medicare. These arrangements typically include fixed pricing terms that were negotiated prior to the implementation of the recently announced tariffs. As a result, and depending on the timing and scope of the implementation of these tariffs, cost increases due to tariffs may be difficult or impossible to pass through to customers until the next negotiation cycle, which could be up to 36 months away.

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Current or future tariffs will also result in increased research and development expenses, including with respect to increased costs associated with APIs, raw materials, laboratory equipment and research materials and components. Trade restrictions could result in delays to our development timelines, either by directly affecting the import of materials necessary for our clinical trials and commercialization efforts, or by creating bottlenecks at the U.S. ports of entry. Increased development costs and extended development timelines could place us at a competitive disadvantage compared to companies operating in regions with more favorable trade relationships and could reduce investor confidence and negatively impact our business, results of operations, financial condition and growth prospects.

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The complexity of announced or future tariffs may also increase the risk that we or our customers or suppliers may be subject to civil or criminal enforcement actions in the United States or foreign jurisdictions related to compliance with trade regulations. Foreign governments may also adopt non-tariff measures, such as procurement preferences or informal disincentives to engage with, purchase from or invest in United States entities, which may limit our ability to compete internationally and attract non-U.S. investment, employees, customers and suppliers. Foreign governments may also take other retaliatory actions against the United States entities, such as decreased intellectual property protection, increased enforcement actions, or delays in regulatory approvals, which may result in heightened international legal and operational risks. In addition, the United States and other governments have imposed and may continue to impose additional sanctions, such as trade restrictions or trade barriers, which could restrict us from doing business directly or indirectly in or with certain countries or parties and may impose additional costs and complexity to our business.

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Trade disputes, tariffs, restrictions and other political tensions between the United States and other countries may also exacerbate unfavorable macroeconomic conditions including inflationary pressures, foreign exchange volatility, financial market instability, and economic recessions or downturns. The ultimate impact of current or future tariffs and trade restrictions remains uncertain and could materially and adversely affect our business, financial condition, and prospects. While we actively monitor these risks, any prolonged economic downturn or escalation in trade tensions could materially and adversely affect our business, ability to access the capital markets or other financing sources, results of operations, financial condition and prospects. In addition, tariffs and other trade developments have and may continue to heighten the risks related to the other risk factors described in our Annual Report for the fiscal year ended December 31, 2024.

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Changes to promotion and advertising rules and regulations may potentially adversely impact sales of our products.

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In September 2025, the HHS and the FDA jointly announced a crackdown on deceptive direct-to-consumer (“DTC”) pharmaceutical advertising, including promotion through social media. The announcement follows the presidential memorandum and report by the Trump administration’s Make American Healthy Again Commission, which directed the FDA, HHS, Federal Trade Commission and Department of Justice to increase oversight of DTC pharmaceutical advertising, particularly associated with risk information on digital platforms. Although Autolus does not rely on DTC pharmaceutical advertising for sales of its products (including social media channels), there is an indirect relationship between our company and our patients. If the FDA further tightens or restricts the ability of pharmaceutical or biotech companies to engage directly with patients, this could potentially impact upon the demand for products and therapies in the United States.

Current report — wording carried from elsewhere

There were no material changes during the period covered in this Quarterly Report on Form 10-Q to the Risk Factors previously disclosed in our most recent Annual Report on Form 10-K.