10806XAB8

As of June 30, 2026, we held cash, cash equivalents and marketable securities of $720.2 million. Our cash equivalents consist of amounts invested in money market funds, agency discount notes, and high investment grade fixed income securities that are primarily invested in U.S. government securities and treasury bills. Our marketable securities consisted of high investment grade fixed income securities that were invested in U.S. treasury bills and notes and agency discount notes. We do not enter into investments for trading or speculative purposes and have not used any derivative financial instruments to manage our interest rate risk exposure. We have not been exposed, nor do we anticipate being exposed to material risks due to changes in interest rates. We do not believe that our cash, cash equivalents and marketable securities have a significant risk of default or illiquidity. As of June 30, 2026, our 2027 Notes, 2029 Notes, 2031 Notes, and 2033 Notes had principal balances of $550.0 million, $747.5 million, $575.0 million, and $632.5 million, respectively, which bear fixed interest rates that are not subject to variability as a result of changes in interest rates. Inflationary factors, such as increases in the cost of our raw materials, clinical supplies, interest rates and overhead costs may adversely affect our operating results. We do not believe that inflation has had a material impact on our financial position or results of operations during the periods presented. Significant adverse changes in inflation and prices in the future could result in material losses.

As of the date of this Quarterly Report on Form 10-Q, we were not party to any material legal proceedings. In the future, we may become party to legal proceedings and claims arising in the ordinary course of business. Although the results of litigation and claims cannot be predicted with certainty, we do not believe we are party to any claim or litigation the outcome of which, if determined adversely to us, would individually or in the aggregate be reasonably expected to have a material adverse impact on our financial position, results of operations or cash flows. Regardless of the outcome, litigation can have an adverse effect on us because of defense and settlement costs, diversion of management resources and other factors.

• The business is substantially dependent on market acceptance and successful commercialization of Attruby and Beyonttra, with revenue highly sensitive to physician adoption, payer coverage, and competitive pricing.
• Proposed U.S. regulations (GLOBE and GUARD) and executive actions seek to impose most-favored-nation pricing and mandatory rebates on Medicare drugs, which could significantly reduce net product revenue from U.S. sales.
• The company relies entirely on third-party manufacturers for commercial and clinical supply, with single-source suppliers for key products, exposing it to supply disruptions, quality failures, and regulatory non-compliance.
• Clinical development faces substantial risks including enrollment delays for rare disease trials, potential adverse events for novel gene therapy candidates, and the possibility that earlier trial results will not predict later success.
• Intellectual property challenges, including potential third-party infringement claims, patent validity disputes, and dependence on in-licensed technologies, could block or limit commercialization of key products.
• The company has significant debt ($2.5 billion) and royalty obligations, and may require additional capital; failure to obtain funding could force delays or termination of development and commercialization efforts.