UNITED THERAPEUTICS Corp

Previous wording

•Expectations of revenues, expenses, profitability, cash flows, and growth in the number of patients being treated with our products, including continued growth in sales of our newest product, Tyvaso DPI, and anticipated growth in the number of patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) being treated with our Tyvaso products;

Current wording

•Expectations of revenues, expenses, profitability, cash flows, and growth in the number of patients being treated with our products, including continued growth in sales of Tyvaso DPI, and anticipated growth in the number of patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) being treated with our Tyvaso products;

Previous wording

•The impact of competing therapies on sales of our commercial products, including the impact of generic versions of Remodulin; established therapies such as Uptravi®; and therapies such as Merck’s Winrevair™ and Liquidia’s recently-approved Yutrepia;

Current wording

•The impact of competing therapies on sales of our commercial products, including the impact of generic versions of Remodulin; established therapies such as Uptravi®; and newer therapies such as Merck’s Winrevair™ and Liquidia’s Yutrepia;

Previous wording

•Tyvaso DPI, a dry powder inhaled formulation of the prostacyclin analogue treprostinil, approved by the FDA in May 2022 to improve exercise ability in patients with pulmonary arterial hypertension (PAH) and PH-ILD. We initiated commercial shipments of Tyvaso DPI to our U.S. distributors in June 2022.

Current wording

•Tyvaso DPI, a dry powder inhaled formulation of the prostacyclin analogue treprostinil, approved by the FDA in May 2022 to improve exercise ability in patients with pulmonary arterial hypertension (PAH) and PH-ILD.

Previous wording

•Nebulized Tyvaso, a nebulized liquid inhaled formulation of treprostinil, approved by the FDA to improve exercise ability in patients with PAH. Nebulized Tyvaso was also approved by the FDA in March 2021 to improve exercise ability in patients with PH-ILD. In addition, marketing authorization applications for Nebulized Tyvaso to treat PAH and/or PH-ILD have also been approved in other countries including Argentina, Israel, and Japan, with applications pending in various other countries in Latin America, Asia, and the Middle East.

Current wording

•Nebulized Tyvaso, a nebulized liquid inhaled formulation of treprostinil, approved by the FDA to improve exercise ability in patients with PAH. Nebulized Tyvaso was also approved by the FDA in March 2021 to improve exercise ability in patients with PH-ILD. Nebulized Tyvaso has also been approved in various countries outside of the United States.

Previous wording

•Remodulin, a continuously-infused formulation of treprostinil, approved by the FDA for subcutaneous and intravenous delivery to diminish symptoms associated with exercise in patients with PAH. Remodulin has also been approved in various countries outside of the United States. In February 2021, we launched U.S. sales of the Remunity Pump, a next-generation subcutaneous infusion system for Remodulin developed under an exclusive development and license agreement with DEKA Research & Development Corp. (DEKA). In January 2025, DEKA obtained FDA clearance of a new version of the Remunity Pump, which is intended to improve the patient experience by making the pump easier to use and will be offered only as a patient-filled device. We plan to launch this new system, called RemunityPRO™, later this year.

Current wording

•Remodulin, a continuously-infused formulation of treprostinil, approved by the FDA for subcutaneous and intravenous delivery to diminish symptoms associated with exercise in patients with PAH. Remodulin has also been approved in various countries outside of the United States. In February 2021, we launched U.S. sales of the Remunity Pump, a next-generation subcutaneous infusion system for Remodulin developed under an exclusive development and license agreement with DEKA Research & Development Corp. (DEKA). In September 2025, we launched a new patient-filled version of the Remunity Pump, called RemunityPRO, which is intended to improve the patient experience by making the pump easier to use.

Previous wording

Our total revenues consist primarily of sales of the commercial products noted above, including the delivery devices (in the case of Tyvaso DPI, Nebulized Tyvaso, and Remodulin). We have entered into separate, non-exclusive distribution agreements with Accredo Health Group, Inc. and its affiliates (Accredo) and Caremark, L.L.C. (CVS Specialty) to distribute Tyvaso DPI, Nebulized Tyvaso, Remodulin, the Remunity Pump, and Orenitram in the United States, and we have entered into an exclusive distribution agreement with Cencora Global Procurement Ltd., an affiliate of Cencora, Inc. (formerly known as AmerisourceBergen Corporation), to distribute Unituxin in the United States. We also sell Nebulized Tyvaso, Remodulin, and Unituxin to distributors internationally. We sell Adcirca through Lilly’s pharmaceutical wholesale network. To the extent we have increased the price of any of these products, increases have typically been in the single-digit percentages per year, except for Adcirca, the price of which is set solely by Lilly. We also derive revenues from the sale of commercialex vivo lung perfusion services, which are presented under Other within Note 11—Segment Information to our consolidated financial statements included in this Quarterly Report on Form 10-Q.

Current wording

Our total revenues consist primarily of sales of the commercial products noted above, including the delivery devices (in the case of Tyvaso DPI, Nebulized Tyvaso, and Remodulin). We have entered into separate, non-exclusive distribution agreements with Accredo Health Group, Inc. and its affiliates (Accredo) and Caremark, L.L.C. (CVS Specialty) to distribute Tyvaso DPI, Nebulized Tyvaso, Remodulin, the Remunity Pump, the RemunityPRO Pump, and Orenitram in the United States, and we have entered into an exclusive distribution agreement with Cencora Global Procurement Ltd. to distribute Unituxin in the United States. We also sell Nebulized Tyvaso, Remodulin, and Unituxin to distributors internationally. We sell Adcirca through Lilly’s pharmaceutical wholesale network. To the extent we have increased the price of any of these products, increases have typically been in the single-digit percentages per year, except for Adcirca, the price of which is set solely by Lilly. We also derive revenues from the sale of commercialex vivo lung perfusion services, which are presented under Other within Note 11—Segment Information to our consolidated financial statements included in this Quarterly Report on Form 10-Q.

Previous wording

We settled litigation with Sandoz related to its abbreviated new drug application (ANDA) seeking FDA approval to market a generic version of Remodulin, and in March 2019, Sandoz announced the availability of its generic product in the United States. We have also entered into similar settlement agreements with other generic companies, some of which have also launched sales of generic versions of Remodulin. Through June 30, 2025, we have seen limited erosion of Remodulin sales as a result of generic treprostinil competition in the United States. We are currently engaged in litigation with Sandoz and its marketing partner, RareGen (now a subsidiary of Liquidia Corporation, the parent company of Liquidia), related to the infusion devices used to administer Remodulin subcutaneously. We understand that generic treprostinil was initially launched

Current wording

We settled litigation with Sandoz related to its abbreviated new drug application (ANDA) seeking FDA approval to market a generic version of Remodulin, and in March 2019, Sandoz announced the availability of its generic product in the United States. We have also entered into similar settlement agreements with other generic companies, some of which have also launched sales of generic versions of Remodulin. Through September 30, 2025, we have seen limited erosion of Remodulin sales as a result of generic treprostinil competition in the United States. We are currently engaged in litigation with Sandoz and its marketing partner, RareGen (now a subsidiary of Liquidia Corporation, the parent company of Liquidia), related to the infusion devices used to administer Remodulin subcutaneously. We understand that generic treprostinil was initially launched by Sandoz/RareGen for use only by intravenous infusion. In May 2021, Sandoz/Liquidia Corporation announced that Sandoz’s generic treprostinil was made available for subcutaneous use, following FDA clearance of a cartridge that can administer the product via the Smiths Medical CADD MS-3 pump. In addition, Liquidia has announced it is developing a new subcutaneous

Previous wording

by Sandoz/RareGen for use only by intravenous infusion. In May 2021, Sandoz/Liquidia Corporation announced that Sandoz’s generic treprostinil was made available for subcutaneous use, following FDA clearance of a cartridge that can administer the product via the Smiths Medical CADD MS-3 pump. In addition, Liquidia has announced it is developing a new subcutaneous infusion system for its generic treprostinil product. See Note 12—Litigation, to our consolidated financial statements included in this Quarterly Report on Form 10-Q.

Current wording

infusion system for its generic treprostinil product. See Note 12—Litigation, to our consolidated financial statements included in this Quarterly Report on Form 10-Q.

Previous wording

We settled litigation with Watson Laboratories, Inc. (Watson) and Actavis Laboratories FL, Inc. (Actavis) related to their ANDAs seeking FDA approval to market generic versions of Nebulized Tyvaso and Orenitram, respectively, before the expiration of certain of our U.S. patents. Under the settlement agreements, Watson and Actavis can market their generic versions of Nebulized Tyvaso and Orenitram in the United States beginning in January 2026 and June 2027, respectively, although they may be permitted to enter the market earlier under certain circumstances. In May 2022, we settled litigation with ANI Pharmaceuticals, Inc. (ANI) regarding its ANDA seeking FDA approval to market a generic version of Orenitram. Under the settlement agreement, ANI can market its generic version of Orenitram in the United States beginning in December 2027, although it may be permitted to enter the market earlier under certain circumstances. Competition from these generic companies could reduce our net product sales and profits.

Current wording

We settled litigation with Watson Laboratories, Inc. (Watson) and Actavis Laboratories FL, Inc. (Actavis) related to their ANDAs seeking FDA approval to market generic versions of Nebulized Tyvaso and Orenitram, respectively, before the expiration of certain of our U.S. patents. Under the settlement agreements, Watson and Actavis can market their generic versions of Nebulized Tyvaso and Orenitram, respectively, in the United States beginning in January 2026 and June 2027, respectively, although either or both of them may be permitted to enter the market earlier under certain circumstances. In May 2022, we settled litigation with ANI Pharmaceuticals, Inc. (ANI) regarding its ANDA seeking FDA approval to market a generic version of Orenitram. Under the settlement agreement, ANI can market its generic version of Orenitram in the United States beginning in December 2027, although it may be permitted to enter the market earlier under certain circumstances. Competition from these generic companies could reduce our net product sales and profits.

Previous wording

We are engaged in patent litigation with Liquidia concerning Yutrepia. Specifically, we allege that Yutrepia infringes a patent we own covering the treatment of PH-ILD to improve exercise capacity in patients suffering from PH-ILD by inhaling treprostinil at specific dosages (the PH-ILD patent). If we are successful in this litigation, we believe Liquidia will be required to remove PH-ILD as a labeled indication for Yutrepia until the expiration of our patent in February 2042. We are also engaged in litigation with Liquidia alleging trade secret misappropriation. In this case, we allege that a former executive of ours misappropriated trade secrets related to Tyvaso when he utilized them as an executive of Liquidia to aid in the development of Yutrepia. Finally, we are engaged in separate litigation against Liquidia alleging that Yutrepia infringes a patent that claims a method of treating pulmonary hypertension using inhaled treprostinil delivered in a specified dosage using a specified dosage regimen. This patent expires in May 2027.

Current wording

We are engaged in patent litigation with Liquidia concerning Yutrepia. Specifically, we allege that Yutrepia infringes a patent we own covering the treatment of PH-ILD to improve exercise capacity in patients suffering from PH-ILD by inhaling treprostinil at specific dosages. If we are successful in this litigation, we believe Liquidia will be required to remove PH-ILD as a labeled indication for Yutrepia until the expiration of our patent in February 2042. We are also engaged in litigation with Liquidia alleging trade secret misappropriation. In this case, we allege that a former executive of ours misappropriated trade secrets related to Tyvaso when he utilized them as an executive of Liquidia to aid in the development of Yutrepia. Finally, we are engaged in separate litigation against Liquidia alleging that Yutrepia infringes a patent that claims a method of treating pulmonary hypertension using inhaled treprostinil delivered in a specified dosage using a specified dosage regimen. This patent expires in May 2027.

Previous wording

Liquidia has also sued us, alleging infringement of a patent with claims directed to the treatment of pulmonary hypertension by administering specified amounts of treprostinil via a dry powder inhaler in a specified number of breaths. We filed a motion to dismiss or, alternatively, a motion to stay, the case based on the argument that we co-own the asserted patent based the alleged trade secret misappropriation discussed above.

Current wording

Liquidia has also sued us, alleging infringement of a patent with claims directed to the treatment of pulmonary hypertension by administering specified amounts of treprostinil via a dry powder inhaler in a specified number of breaths. We filed a motion to dismiss or, alternatively, a motion to stay, the case based on the argument that we co-own the asserted patent based on the alleged trade secret misappropriation discussed above.

Previous wording

We devote substantial resources to our various clinical trials and other research and development efforts, which are conducted both internally and through third parties. From time to time, we also license or acquire additional technologies

Current wording

We devote substantial resources to our various clinical trials and other research and development efforts, which are conducted both internally and through third parties. From time to time, we also license or acquire additional technologies and compounds to be incorporated into our development pipeline. Our operating expenses include the costs described below.

Previous wording

The TETON 1 and TETON 2 studies were prompted by data from the INCREASE study of Nebulized Tyvaso for the treatment of PH-ILD, which demonstrated in a post-hoc analysis that treatment with Nebulized Tyvaso resulted in significant improvements in percent predicted FVC at weeks 8 and 16, with subjects having an underlying etiology of IPF showing the greatest improvement (week 8: 2.5 percent; p=0.038 and week 16: 3.5 percent; p=0.015). Further, open-label extension (OLE) data published in 2023 showed that these improvements in FVC were sustained for at least 64 weeks. For those patients who received placebo during the INCREASE study, marked improvements in FVC were observed following transition to Nebulized Tyvaso during the OLE study. These data points, combined with substantial preclinical evidence of antifibrotic activity of treprostinil, suggest that Nebulized Tyvaso may offer a treatment option for patients with IPF. We believe there are approximately 100,000 IPF patients in the United States.

Current wording

The TETON 1 and TETON 2 studies were prompted by data from the INCREASE study of Nebulized Tyvaso for the treatment of PH-ILD, which demonstrated in a post-hoc analysis that treatment with Nebulized Tyvaso resulted in significant improvements in percent predicted FVC at weeks 8 and 16, with subjects having an underlying etiology of IPF showing the greatest improvement (week 8: 2.5 percent; p=0.038 and week 16: 3.5 percent; p=0.015). Further, open-label extension (OLE) data published in 2023 showed that these improvements in FVC were sustained for at least 64 weeks. For those patients who received placebo during the INCREASE study, marked improvements in FVC were observed following transition to Nebulized Tyvaso during the OLE study. These data points, combined with substantial preclinical evidence of antifibrotic activity of treprostinil and the results of the TETON 2 study, suggest that Nebulized Tyvaso may offer a treatment option for patients with IPF. We believe there are approximately 100,000 IPF patients in the United States.

Previous wording

We are conducting two phase 3 studies of Nebulized Tyvaso, called TETON 1 and TETON 2, in patients with idiopathic pulmonary fibrosis (IPF). TETON 1 is being conducted in the United States and Canada, and TETON 2 is being conducted outside the United States and Canada. Enrollment in the TETON 2 study was completed in July 2024 after enrolling a total of 597 patients, and TETON 1 completed enrollment in January 2025 with 598 patients. We are also conducting a phase 3 study of Nebulized Tyvaso called TETON PPF for the treatment of progressive pulmonary fibrosis (PPF); we enrolled the first patient in TETON PPF in October 2023. The primary endpoint of each TETON study is the change in absolute forced vital capacity (FVC) from baseline to week 52.

Current wording

We are also conducting a phase 3 study of Nebulized Tyvaso called TETON PPF for the treatment of progressive pulmonary fibrosis (PPF); we enrolled the first patient in TETON PPF in October 2023. The primary endpoint of the TETON PPF study is the change in absolute FVC from baseline to week 52.

Previous wording

The TETON PPFstudy was also prompted by a post-hoc analysis of data from the INCREASE study. PPF is a group of ILD conditions that exhibit progressive, self-sustaining fibrosis, and a similar disease course to IPF. PPF includes idiopathic interstitial pneumonias, autoimmune ILDs, chronic fibrosing hypersensitivity pneumonitis, and fibrotic ILDs related to environmental/occupational exposure. Due to the similarities in the mechanism of fibrosis between IPF and PPF, we anticipate that anti-fibrotic therapies will impact disease progression similarly in patients with these conditions. Therefore, based on the FVC improvements in subjects with IPF observed in the INCREASE study, we are conducting a single pivotal study, TETON PPF, to evaluate the safety and efficacy of Nebulized Tyvaso for the treatment of PPF. We are targeting enrollment of 698 patients in this study. We believe there are at least 60,000 PPF patients in the United States, but the number could be significantly greater as some estimates indicate the U.S. patient population could exceed 180,000.

Current wording

The TETON PPF study was also prompted by a post-hoc analysis of data from the INCREASE study. PPF is a group of ILD conditions that exhibit progressive, self-sustaining fibrosis, and a similar disease course to IPF. PPF includes idiopathic interstitial pneumonias, autoimmune ILDs, chronic fibrosing hypersensitivity pneumonitis, and fibrotic ILDs related to environmental/occupational exposure. Due to the similarities in the mechanism of fibrosis between IPF and PPF, we anticipate that anti-fibrotic therapies will impact disease progression similarly in patients with these conditions. Therefore, based on the FVC improvements in subjects with IPF observed in the INCREASE study, we are conducting the TETON PPFstudy to evaluate the safety and efficacy of Nebulized Tyvaso for the treatment of PPF. We are targeting enrollment of 698 patients in this study. We believe there are at least 60,000 PPF patients in the United States, but the number could be significantly greater as some estimates indicate the U.S. patient population could exceed 180,000.

Previous wording

Both the FDA and the European Medicines Agency (EMA) have granted orphan designation for treprostinil to treat IPF. The FDA denied our orphan designation application for PPF. If the TETON studies are successful, we plan to seek FDA approval to expand the Nebulized Tyvaso label to include IPF and PPF. We also plan to seek FDA approval to expand the Tyvaso DPI label to include IPF and PPF, following completion of any FDA-required bridging studies. We and our distributors will also consider seeking amendments to the marketing authorizations for Nebulized Tyvaso in other countries where it is approved, to include IPF and/or PPF indications, and we will also consider seeking approval of Nebulized Tyvaso for these indications in countries where it is not yet approved.

Current wording

If the TETON studies result in FDA approval to add IPF and/or PPF to the Nebulized Tyvaso label, we also plan to seek FDA approval to expand the Tyvaso DPI label to include IPF and/or PPF, as applicable, following completion of any FDA-required bridging studies. We and our distributors will also consider seeking amendments to the marketing authorizations for Nebulized Tyvaso in other countries where it is approved, to include IPF and/or PPF indications, and we will also consider seeking approval of Nebulized Tyvaso for these indications in countries where it is not yet approved. Both the FDA and the European Medicines Agency have granted orphan designation for treprostinil to treat IPF, but not for PPF.

Previous wording

Ralinepag is a next-generation, once-daily, oral, selective, and potent prostacyclin receptor agonist that we are developing for the treatment of PAH. A phase 2 study of an immediate-release formulation of ralinepag in 61 PAH patients (40 patients on active ralinepag, 21 on placebo) met its primary endpoint, showing a 29.8 percent reduction (p=0.03) in median pulmonary vascular resistance (PVR, the force or resistance that blood encounters as it flows through the blood vessels in the lungs) after 22 weeks of treatment with ralinepag compared with placebo. After participation in the phase 2 study, 45 patients entered into an OLE study to further determine if ralinepag may be safe and effective for long-term use to treat patients with PAH. The study found that ralinepag had a manageable side effect profile, with a decrease in side effects for patients who continued taking ralinepag over time. Moreover, two years after entering the OLE study, the study showed that ralinepag improved the ability to exercise as the 6MWD significantly increased by a mean of 36.3 meters (p=0.004), and over 85 percent of patients

Current wording

Ralinepag is a next-generation, once-daily, oral, extended-release, selective, and potent prostacyclin receptor agonist that we are developing for the treatment of PAH. A phase 2 study of an immediate-release formulation of ralinepag in 61 PAH patients (40 patients on active ralinepag, 21 on placebo) met its primary endpoint, showing a 29.8 percent reduction (p=0.03) in median pulmonary vascular resistance (PVR, the force or resistance that blood encounters as it flows through the blood vessels in the lungs) after 22 weeks of treatment with ralinepag compared with placebo. After participation in the phase 2 study, 45 patients entered into an OLE study to further determine if ralinepag may be safe and effective for long-term use to treat patients with PAH. The study found that ralinepag had a manageable side effect profile, with a decrease in side effects for patients who continued taking ralinepag over time. Moreover, two years after entering the OLE study, the study showed that ralinepag improved the ability to exercise as the 6MWD significantly increased by a mean of 36.3 meters (p=0.004), and over 85 percent of patients remained stable in their functional class. Additionally, hemodynamic measures (metrics to

Previous wording

remained stable in their functional class. Additionally, hemodynamic measures (metrics to measure how well the heart is working) taken either one or two years after entering the OLE study demonstrated significant improvements (p=0.05) in both median PVR and mean pulmonary arterial pressure (the pressure in the blood vessels connecting the heart).

Current wording

measure how well the heart is working) taken either one or two years after entering the OLE study demonstrated significant improvements (p=0.05) in both median PVR and mean pulmonary arterial pressure (the pressure in the blood vessels connecting the heart).

Previous wording

In June 2025, we concluded enrollment of the ADVANCE OUTCOMES study, which is a phase 3, event-driven clinical trial of an extended-release formulation of ralinepag in PAH patients with a primary endpoint of time to first clinical worsening event. ADVANCE OUTCOMES is a global, multi-center, placebo-controlled trial that includes patients on approved oral background PAH therapies. We expect to continue accruing clinical worsening events in this study through the end of 2025, and release topline data from the study in the first half of 2026. In October 2023, the data monitoring committee for the ADVANCE OUTCOMES study completed a routine, unblinded safety review of data from nearly 510 patients enrolled in the study, and unanimously recommended continuation of the trial without modification.

Current wording

In June 2025, we concluded enrollment of the ADVANCE OUTCOMES study, which is a phase 3, event-driven clinical trial of an extended-release formulation of ralinepag in PAH patients with a primary endpoint of time to first clinical worsening event. ADVANCE OUTCOMES is a global, multi-center, placebo-controlled trial that includes patients on approved oral background PAH therapies. We expect to continue accruing clinical worsening events in this study through the end of 2025, and release topline data from the study in the first half of 2026.

Previous wording

In December 2024, we submitted an Investigational New Drug application (IND) to the FDA related to our UKidney product. In January 2025, the FDA cleared this IND, which enables us to commence a clinical trial. This study, which we refer to as the EXPAND study, is expected to enroll an initial cohort of six end-stage renal disease (ESRD) patients, expanding to up to 50 participants, and we intend to use the results of this study to support a Biologics License Application (BLA) with the FDA. This study is designed as a combination phase 1/2/3 trial (sometimes referred to as a “phaseless” study) to evaluate safety and

Current wording

In December 2024, we submitted an Investigational New Drug application (IND) to the FDA related to our UKidney product. In January 2025, the FDA cleared this IND, which enables us to commence a clinical trial. This study, which we refer to as the EXPAND study, is expected to enroll an initial cohort of six end-stage renal disease (ESRD) patients, expanding to up to 50 participants, and we intend to use the results of this study to support a Biologics License Application (BLA) with the FDA. This study is designed as a combination phase 1/2/3 trial (sometimes referred to as a “phaseless” study) to evaluate safety and efficacy seamlessly without moving through separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals. The study has been initiated, and we expect the first transplant to occur shortly.

Previous wording

Based on recent FDA interactions, we do not intend to conduct further preclinical animal studies of our UThymoKidney and UHeart products. As such, in July 2025, we submitted an IND to the FDA related to our anticipated EXTEND clinical study of our UThymoKidney product. Upon clearance of this IND, we intend to conduct a study of this product similar in size and scope to the EXPAND study of our UKidney product. We are also preparing to submit an IND for a clinical study of our UHeart product, which we refer to as the EXPRESS study, in the near term.

Current wording

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.

Previous wording

In February 2024, we inaugurated a designated pathogen-free (DPF) facility in Virginia and began populating the facility with animals during the first quarter of 2024. We expect this DPF facility to supply xenografts compliant with FDA current Good Manufacturing Practices (cGMP) for human clinical trials, with a target capacity of up to 125 organs per year. In 2024, we acquired land in Minnesota where we are constructing a second DPF facility for redundancy, and to support breeding of animals for future commercial use. In March 2025, we purchased land near Houston, Texas, where we are constructing a third DPF facility. While we believe these DPF facilities will be capable of producing organs for commercial use, we are planning to build additional and potentially larger cGMP DPF facilities for commercial use. While these projects will be capital-intensive, the timing and volume of these expenditures will be staggered and paced in a manner intended to balance our need to address market demand as soon as possible following FDA approval with the need to defer the most significant capital expenditures until we achieve certain clinical trial milestones.

Current wording

In February 2024, we inaugurated a designated pathogen-free (DPF) facility in Virginia and began populating the facility with animals during the first quarter of 2024. We expect this DPF facility to supply xenografts compliant with FDA current Good Manufacturing Practices (cGMP) for human clinical trials, with a target capacity of up to 125 organs per year. In 2024, we acquired land in Minnesota where we are constructing a second DPF facility for redundancy, and to support breeding of animals for future commercial use. In March 2025, we purchased land in Houston, Texas, where we are constructing a third DPF facility. While we believe these DPF facilities will be capable of producing organs for commercial use, we are planning to build additional and potentially larger cGMP DPF facilities for commercial use. While these projects will be capital-intensive, the timing and volume of these expenditures will be staggered and paced in a manner intended to balance our need to address market demand as soon as possible following FDA approval with the need to defer the most significant capital expenditures until we achieve certain clinical trial milestones.

Previous wording

Key accomplishments in our xenotransplantation program include the following:

Current wording

Additional key accomplishments in our xenotransplantation program include the following:

Previous wording

•First Successful Xenotransplants of Porcine Hearts. University of Maryland School of Medicine (UMSOM) surgeons have successfully transplanted UHearts into two living human patients. Each of these procedures were authorized by the FDA on a single-patient, expanded access (also called “compassionate use”) basis, and marked the first known examples of transplanting whole organs from gene-edited pigs to humans. The FDA’s compassionate use regulations allow a physician to apply to use an unapproved product outside of a clinical trial to treat an individual patient with a serious or immediately life-threatening disease or condition when no satisfactory alternative therapy is available. The first patient, who received a xenotransplant in January 2022, survived for approximately two months with the UHeart. In June 2022, data from this procedure were published in the New England Journal of Medicine. The second patient, who received a xenotransplant in September 2023, survived for approximately six weeks with the UHeart. We and our collaborators continue to evaluate data from these human transplants.

Current wording

•First Successful Xenotransplants of Porcine Hearts. University of Maryland School of Medicine (UMSOM) surgeons successfully transplanted UHearts into two living human patients. Each of these procedures were authorized by the FDA on a single-patient, expanded access (also called “compassionate use”) basis, and marked the first known examples of transplanting whole organs from gene-edited pigs to humans. The FDA’s compassionate use regulations allow a physician to apply to use an unapproved product outside of a clinical trial to treat an individual patient with a serious or immediately life-threatening disease or condition when no satisfactory alternative therapy is available. The first patient, who received a xenotransplant in January 2022, survived for approximately two months with the UHeart. In June 2022, data from this procedure were published in the New England Journal of Medicine. The second patient, who received a xenotransplant in September 2023, survived for approximately six weeks with the UHeart. We and our collaborators continue to evaluate data from these human transplants.

Previous wording

In September 2023, NYU surgeons completed a 61-day study of a UThymoKidney in a brain-dead organ donor maintained on artificial support. At the time, this experiment marked the longest documented case of a xenotransplanted organ functioning in a human body. Publications of the result of this experiment are expected in the near term.

Current wording

In September 2023, NYU surgeons completed a 61-day study of a UThymoKidney in a brain-dead organ donor maintained on artificial support. At the time, this experiment marked the longest documented case of a xenotransplanted organ functioning in a human body.

Previous wording

•ULobe™. The ULobe is a development-stage engineered lung lobe alternative made using a porcine lung scaffold that is decellularized and then re-cellularized with allogeneic cells. In 2023, our Regenerative Medicine Laboratory in Research Triangle Park, North Carolina (RTP) produced 450 decellularized lung scaffolds, 220 recellularized lungs, and 1.7 trillion human cells for use in recellularization.

Current wording

•ULobe™. The ULobe is a development-stage engineered lung lobe alternative made using a porcine lung scaffold that is decellularized and then re-cellularized with allogeneic human cells. In 2023, our Regenerative Medicine Laboratory in Research Triangle Park, North Carolina (RTP) produced 518 decellularized lung scaffolds, 240 recellularized lungs, and 1 trillion human cells for use in recellularization.

Previous wording

Over 600 patients have received lung transplants following use of our centralized EVLP service.

Current wording

Over 670 patients have received lung transplants following use of our centralized EVLP service.

Previous wording

We have budgeted approximately $710 million for capital expenditures during the third quarter of 2025 through the end of 2027 to construct additional facilities to support the development and commercialization of our products and technologies. This amount is primarily dedicated to construction of a new Tyvaso DPI manufacturing facility in RTP; construction of DPF facilities in Stewartville, Minnesota and Houston, Texas; and property acquired in July 2025 for future growth on our RTP campus. We plan to fund these capital expenditures using cash on hand.

Current wording

We have budgeted approximately $520 million for capital expenditures during the fourth quarter of 2025 through the end of 2027 to construct additional facilities to support the development and commercialization of our products and technologies. This amount is primarily dedicated to construction of a new Tyvaso DPI manufacturing facility in RTP; and construction of DPF facilities in Stewartville, Minnesota and Houston, Texas. We plan to fund these capital expenditures using cash on hand.

Previous wording

We operate in a highly competitive market in which several large pharmaceutical companies control many of the available PAH therapies. These pharmaceutical companies are well established in the market and possess greater financial, technical, and marketing resources than we do. In addition, Yutrepia was approved by the FDA in May 2025 for treatment of PAH and PH-ILD, and the product was launched commercially in June 2025. While we have not seen a material impact of Yutrepia’s launch to date, our Tyvaso DPI net revenues could be materially impacted if Yutrepia gains significant market share or causes material price erosion.

Current wording

We operate in a highly competitive market in which several large pharmaceutical companies control many of the available PAH therapies, including Merck, which received FDA approval for Winrevair (sotatercept-csrk) to treat PAH in March 2024. These pharmaceutical companies are well established in the market and possess greater financial, technical, and marketing resources than we do. In addition, Yutrepia was approved by the FDA in May 2025 for treatment of PAH and PH-ILD, and the product was launched commercially in June 2025. While we have not seen a material impact on our net revenues as a result of Winrevair or Yutrepia’s launch to date, our net revenues could be materially impacted if either or both of these products gain significant market share or cause material price erosion for our existing products.

Previous wording

(2)Net product sales include sales of infusion devices, including the Remunity Pump.

Current wording

(2)Net product sales include sales of infusion devices, including the Remunity Pump and the RemunityPRO Pump.

Previous wording

Total Tyvaso net product sales grew 18% to $469.6 million for the three months ended June 30, 2025, and 21% to $935.9 million for the six months ended June 30, 2025, as compared to $398.2 million and $770.7 million for the same periods in 2024, respectively. Tyvaso DPI net product sales increased for the three and six months ended June 30, 2025, as compared to the same periods in 2024, due to an increase in quantities sold of $54.6 million and $149.1 million, respectively and, to a lesser extent, a price increase, partially offset by higher gross-to-net revenue deductions. The increase in Tyvaso DPI quantities sold was primarily due to continued growth in the number of patients following the product’s launch (including growth in utilization by PH-ILD patients) and, to a lesser extent, increased commercial utilization following the implementation of the Medicare Part D benefit redesign under the Inflation Reduction Act. Nebulized Tyvaso net product sales increased for the three and six months ended June 30, 2025, as compared to the same periods in 2024, primarily due to higher quantities sold and, to a lesser extent, a price increase. The increase in quantities sold for the six months ended June 30, 2025 was primarily driven by the timing of orders by an international distributor in the first quarter of 2025 and does not precisely reflect trends in underlying patient demand.

Current wording

Total Tyvaso net product sales grew 10% to $478.0 million for the three months ended September 30, 2025, and 17% to $1,413.9 million for the nine months ended September 30, 2025, as compared to $433.8 million and $1,204.5 million for the same periods in 2024, respectively. Tyvaso DPI net product sales increased for the three and nine months ended September 30, 2025, as compared to the same periods in 2024, due to an increase in quantities sold of $58.1 million and $204.6 million, respectively, and, to a lesser extent, a price increase, partially offset by higher gross-to-net revenue deductions. The increase in Tyvaso DPI quantities sold was primarily due to continued growth in the number of patients following the product’s launch and, to a lesser extent, increased commercial utilization following the implementation of the Medicare Part D benefit redesign under the Inflation Reduction Act. Nebulized Tyvaso net product sales decreased for the three months ended September 30, 2025, as compared to the same period in 2024, primarily due to higher gross-to-net revenue deductions and a decrease in quantities sold. Nebulized Tyvaso net product sales increased for the nine months ended September 30, 2025, as compared to the same period in 2024, primarily due to a price increase and, to a lesser extent, an increase in international Nebulized Tyvaso net product sales, partially offset by higher gross-to-net revenue deductions.

Previous wording

Orenitram net product sales increased for the three and six months ended June 30, 2025, as compared to the same periods in 2024, due to an increase in quantities sold of $10.6 million and $24.0 million, respectively and, to a lesser extent, a price increase, partially offset by higher gross-to-net revenue deductions. The increase in quantities sold was driven, at least in part, by increased commercial utilization following the implementation of the Medicare Part D benefit redesign under the Inflation Reduction Act.

Current wording

Orenitram net product sales increased for the three and nine months ended September 30, 2025, as compared to the same periods in 2024, due to an increase in quantities sold of $11.7 million and $35.7 million, respectively and, to a lesser extent, a price increase. The increase in quantities sold was driven, at least in part, by increased commercial utilization following the implementation of the Medicare Part D benefit redesign under the Inflation Reduction Act.

Previous wording

We have entered into contracts with all of the major pharmacy benefit managers for Medicare Part D and commercial insurance plans, which provide rebates on utilization of Tyvaso DPI and, in some cases, Orenitram and Nebulized Tyvaso. We entered into these rebate agreements to encourage access to these therapies. Many of these rebates went into effect beginning in the second half of 2024, which have impacted our net revenues since such time by increasing gross-to-net deductions for the relevant products. These rebate contracts are effective at least through 2025.

Current wording

We have entered into contracts with all of the major pharmacy benefit managers for Medicare Part D and commercial insurance plans, which provide rebates on utilization of Tyvaso DPI and, in some cases, Orenitram and Nebulized Tyvaso. Many of these rebates went into effect beginning in the second half of 2024 and have impacted our net revenues since such time by increasing gross-to-net deductions for the relevant products.

Previous wording

(2) Net product sales include sales of infusion devices, including the Remunity Pump.

Current wording

(2) Net product sales include sales of infusion devices, including the Remunity Pump and the RemunityPRO Pump.

Previous wording

Cost of sales, excluding share-based compensation. Cost of sales for the three and six months ended June 30, 2025 increased as compared to the same periods in 2024, primarily due to an increase in: (1) royalty expense resulting from a growth in revenues; and (2) reserve expense resulting from increased production.

Current wording

Cost of sales, excluding share-based compensation. Cost of sales for the three and nine months ended September 30, 2025 increased as compared to the same periods in 2024, primarily due to increases in: (1) royalty expense resulting from a growth in revenues; and (2) inventory reserve expense.

Previous wording

(1)External research and development primarily includes fees paid to third parties (such as clinical trial sites, contract research organizations, and contract laboratories) for preclinical and clinical studies and payments to third-party contract manufacturers before FDA approval of the relevant product.

Current wording

(1)External research and development primarily includes fees paid to third parties (such as clinical trial sites, contract research organizations, and contract laboratories) for preclinical and clinical studies and payments to third-party contract manufacturers before regulatory approval of the relevant product.

Previous wording

(2)Internal research and development primarily includes salary-related expenses for research and development functions, internal costs to manufacture product candidates before FDA approval, and internal facilities-related expenses, including depreciation, related to research and development activities.

Current wording

(2)Internal research and development primarily includes salary-related expenses for research and development functions, internal costs to manufacture product candidates before regulatory approval, and internal facilities-related expenses, including depreciation, related to research and development activities.

Previous wording

Research and development, excluding share-based compensation. Research and development expense for the three months ended June 30, 2025 decreased as compared to the same period in 2024, primarily due to $36.0 million in upfront non-refundable license payments for drug delivery device technologies in 2024, partially offset by: (1) increased expenditures in 2025 related to manufactured organ and organ alternative projects; and (2) a $5.0 million milestone payment made in 2025 for drug delivery device technologies.

Current wording

Research and development, excluding share-based compensation. Research and development expense for the three months ended September 30, 2025 increased as compared to the same period in 2024, primarily due to: (1) increased expenditures related to manufactured organ and organ alternative projects; and (2) a $5.0 million milestone payment for drug delivery technologies.

Previous wording

Research and development expense for the six months ended June 30, 2025 increased as compared to the same period in 2024, primarily due to: (1) an increase of $35.0 million related to milestone payments for drug delivery device technologies; (2) increased expenditures related to manufactured organ and organ alternative projects; and (3) $6.6 million related to adjustments to the fair value of contingent consideration obligations for manufactured organ and organ alternative projects, partially offset by $36.0 million in upfront non-refundable license payments for drug delivery device technologies in 2024.

Current wording

Research and development expense for the nine months ended September 30, 2025 increased as compared to the same period in 2024, primarily due to: (1) an increase of $40.0 million related to milestone payments for drug delivery technologies; (2) increased expenditures related to manufactured organ and organ alternative projects; and (3) $6.9 million related to adjustments to the fair value of contingent consideration obligations for manufactured organ and organ alternative projects, partially offset by (4) $36.0 million in upfront non-refundable license payments for drug delivery technologies in 2024.

Previous wording

(1)Excluding impairment of PP&E. See Impairment of PP&Esection below.

Current wording

(1)Excluding impairment of PP&E and litigation accrual. See Impairment of PP&Eand Litigation accrual sections below.