UTHR — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-08-05

• Phase 3 ADVANCE OUTCOMES study successful; NDA submitted to the FDA

• in the TETON-PPF study. While estimates vary, we believe the size of the U.S. PPF population is approximately 200,000 patients.

• We are developing an oral triple combination therapy consisting of ralinepag, an endothelin receptor antagonist (ERA), and a PDE-5 inhibitor. We have completed formulation development and dosage design work and plan to engage with the FDA on our proposed clinical development strategy. Our triple com…

• We are developing a dry powder inhalation (DPI) version of ralinepag. In August 2025, we exercised the option under our license and collaboration agreement with MannKind Corp. to develop ralinepag DPI utilizing the dry powder formulation technology used to manufacture Tyvaso DPI. We believe the half…

• efficacy seamlessly without moving through separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals. The first transplant in this study occurred in the fourth quarter of 2025, and the study is ongoing.

Text removed vs the prior filing · source: 10-Q · 2026-05-06

• We are developing an oral triple combination therapy consisting of ralinepag, an endothelin receptor antagonist (ERA), and a PDE-5 inhibitor. We have completed formulation development and dosage design work and plan to engage with the FDA on

• our proposed clinical development strategy following submission of our NDA for ralinepag extended-release tablets. Our triple combination therapy is intended to provide a convenient means of dosing patients with existing first-line oral therapies for PAH (an ERA and a PDE-5 inhibitor), along with on…

• We are developing a dry powder inhalation (DPI) version of ralinepag. In August 2025, we exercised the option under our license and collaboration agreement with MannKind Corp. to develop ralinepag DPI utilizing the dry powder formulation technology used to manufacture Tyvaso DPI. We believe the half…

• In July 2025, we submitted an IND to the FDA related to our anticipated EXTEND clinical study of our UThymoKidney product. In August 2025, the FDA cleared this IND, enabling us to commence this study, which we expect will be similar in size and

• scope to the EXPAND study described above. We are engaging with the FDA on a potential IND for a study of our UHeart product.

Text added vs the prior filing · source: 10-Q · 2026-08-05

• purposes. Any negative attention, threats, or acts of vandalism directed against our animal research or manufacturing activities could impede the operation of our business.

• and Orenitram revenues could be offset because of the IRA’s Part D redesign, which went into effect in 2025 and resulted in an increase in the number of patients able to afford these therapies. The amount of the offset, if any, is inherently uncertain and difficult to measure and predict.

• The 2026 Medicare Physician Fee Schedule final rule amended the Medicare Part B “average sales price” regulations, including by mandating that, as part of the manufacturer “bona fide service fee” determination, manufacturers submit reasonable assumptions and, for all new service vendor contracts as …

• These legal reforms, as well as the IRA and other healthcare legislative or regulatory reform measures that may be adopted in the future, have resulted in, and may in the future result in, downward pressure on the payment for any of our approved products, and may adversely impact our business. Any r…

• It is difficult to predict the impact, if any, that future federal or state legislation, or executive actions, might have on the use of and reimbursement for our products in the United States, such as the potential for the importation of generic versions of our products, for reimbursement reductions…

Text removed vs the prior filing · source: 10-Q · 2026-05-06

• Lilly manufactures and supplies Adcirca for us. We use Lilly’s pharmaceutical wholesaler network to distribute Adcirca. If Lilly is unable to manufacture or supply Adcirca or its distribution network is disrupted, it could delay, disrupt, or prevent us from selling Adcirca.

• rules and guidelines related to promotion and advertising can result in adverse regulatory actions by the FDA and its international counterparts — such as warning letters, enforcement actions, civil lawsuits, or criminal prosecution.

• The IRA and other healthcare reform measures that may be adopted in the future may result in additional downward pressure on the payment that we receive for any approved product, and may adversely impact our business. Any reduction in reimbursement from Medicare and other government programs may res…

• It is difficult to predict the impact, if any, that future federal or state legislation, or executive actions, might have on the use of and reimbursement for our products in the United States, such as the potential for the importation of generic versions of our products, for price caps under state l…

• If HRSA develops a new theory of liability, we may face enforcement action or penalties as well as adverse publicity. Such an outcome may also prompt other parties to challenge our policies. It is also possible that covered entities could bring an action against us under the administrative dispute r…