United Therapeutics Corporation

Good morning and welcome to the United Therapeutics Corporation fourth quarter 2023 earnings webcast. My name is David and I will be your conference operator today. All participants on the call of the webcast will be in listen-only mode until the question and answer portion of the earnings call. If you would like to ask a question during that time, simply press star and one on your telephone keypad. If you would like to withdraw your question, please press star, then two. Please note this event is being recorded. I would now like to turn the conference over to Dewey Steadman, Head of Investor Relations at United Therapeutics.

Thank you, Dave, and good morning. It's my pleasure to welcome you to the United Therapeutics Corporation fourth quarter 2023 earnings webcast. Remarks today will include forward-looking statements representing our expectations or beliefs regarding future events. These statements involve risks and uncertainties that may cause actual results to differ materially. Our latest SEC filings, including Forms 10-K and 10-Q, contain additional information on these risks and uncertainties. We assume no obligation to update those forward-looking statements. Today's remarks also may discuss the progress and results of clinical trials or other developments with respect to our products. These remarks are intended solely to educate investors and are not intended to serve as the basics for medical decision-making or to suggest that any products are safe and effective for any unapproved or investigational uses. Full prescribing information for the products are available on our website. Accompanying me on today's call are Dr. Martine Rothblatt, our Chairperson and Chief Executive Officer, Michael Bigwitz, our President and Chief Operating Officer, James Edgman, our Chief Financial Officer and Treasurer, Dr. Lee Peterson, our Executive Vice President of Product Development and Xenotransplantation, and Pat Poisson, our Executive Vice President of Technical Operations. Note that James and I will be participating in one-on-one meetings at the 2023 UBS European Healthcare Conference on February 27th in London. Michael James and I will participate in a fireside chat and one-on-one meetings at the TD Cowan Healthcare Conference on March 5th in Boston. And Pat Poisson and I will participate in a fireside chat and one-on-one meetings at the Lyric Partners Global Biopharma Conference on March 12th in Miami. Our scientific, commercial, and medical affairs teams will present at the American College of Cardiology 73rd Scientific Sessions April 6th through 8th in Atlanta, the International Society for Heart and Lung Transplantation, April 10th through 13th in Prague, and the American Thoracic Society International Conference on May 17th through 22nd in San Diego. And now I will turn the call or the webcast over to Dr. Rothblatt for an overview of our fourth quarter 2023 financial results and the business activities of United Therapeutics. Martin?

Thank you, Dewey. Good morning, everyone, and a good day to those who are on the other side of the oceans. Congratulations to the 1,200 Unitharians who work tirelessly every day to help us achieve our third straight quarter of record revenue and our second straight year of record revenue. We yet again achieve 20% plus quarterly and annual revenue growth for the fourth quarter and the full year On top of our record performance in the fourth quarter of 2023, earlier this year we received important external validation of the value of our Tyveso DPI business through a royalty transaction executed by our partners at Mankind Corporation. To put it simply, Mankind sold a tenth of their 10% royalty payment stream from us which equates to 1% of Tyveso DPI sales, and they sold it to Sagar Healthcare Partners for $150 million plus other milestones. That implies an external valuation of the entirety of the Tyveso DPI revenue stream of $15 billion before even factoring in the additional potential milestones. That's well above the current market cap for our entire business. And importantly, this transaction valuation is far above the Wall Street valuation of our entire company as a whole and is for only one of our many products. At United Therapeutics, we talk about being positioned for three waves of growth. And let's dive into the many reasons why we're so confident in our business. Our first wave of growth will come through our existing commercial business, led by Tyveso in PHILD. We continue to post solid growth in our current business, with our third consecutive quarter of record revenues for Tyveso and revenue growth for our U.S. remodeling business, despite the presence of competition on the market for the past five years. Our growth in PHILD and continued leadership in pulmonary arterial hypertension, also known as PAH, has led our nebulized Tyveso and our Tyveso DPI products combining to become the most prescribed prostacyclin therapy in the United States. Michael will go into detail on this exciting aspect of our commercial business. Our second wave of growth will come from our near-term pipeline, led by the Teton Studies in Pulmonary Fibrosis and the Advanced Outcomes Study of Relenopeg in PAH. These programs should enable us to continue our double-digit annual growth trend through the second half of the decade. I will provide updates on the Teton and Relenopeg programs shortly. Of course, both our first and second waves of growth are subject to clinical trial outcomes, regulatory approvals, new competitive entrance, and the potential impacts of the Inflation Reduction Act. But we feel good about our prospects for meeting these revenue growth targets. Our third wave of sustainable growth will come through the development, manufacture, and widespread use of manufactured organs and organ technologies to provide a solution to patients suffering from end-stage kidney, lung, heart, and liver disease. Now, moving to our near-term pipeline and second wave of growth, we have four key registration trials underway. Three TETON studies for pulmonary fibrosis and the Advanced Outcome Study for Relenopeg and Oral Therapy for Group 1 PAH. We also advanced our mural liver ELAP program toward the clinic with a recent and historic IND clearance by the FDA. Moving to Teton, we believe IPF represents a 100,000 patient opportunity in the United States with only two approved therapies that nearly slow lung function decline. Both TTON1 and TTON2 are enrolling patients, and at this time, we are aiming for full enrollment in both studies with 576 patients each by the end of this calendar year. Likewise, we believe PPF, or progressive pulmonary fibrosis, represents a 60,000-patient opportunity in the U.S. alone, And this disease is quite distinct from IPF or idiopathic pulmonary fibrosis. One of the two FDA-approved IPF therapies is also approved for PPF. And as in IPF, it only slows the decline of lung function in these fragile patients. The Teton PPF study dosed its first patients in the fourth quarter of 2023, right on schedule. And we expect this trial to enroll 698 patients. We believe there is a high probability of success in the three TITAN studies based on the IPF subset analysis of the increased study of nebulized tyveso in PHILD patients. Unlike the two IPF studies that are already on the market, Nebulized tibiaso in a safety endpoint showed an improvement, an improvement, actual improvement of lung function in the subset of patients that had IPF along with their pulmonary hypertension. That just gives so many people so much hope. Now let's move on to Relenopeg and our advanced outcome study in Group 1 PAH, which continues to enroll patients, and we expect completion of the study in 2025. We are in fact targeting 700 to 1,000 patients in this study, depending on the pace of accruing clinical worsening events. Relenopeg is a next-generation selective and potent prostacyclin receptor agonist, which we are developing as a once-daily oral therapy for PAH. We believe Relenopeg's once-daily dosing sustained release profile, and titratability could position it favorably against the other oral prostacyclin receptor agonists on the market, as well as other therapies for PAH patients. Relenoped provides 24-hour coverage with higher potency than the other oral prostacyclin agonists, as demonstrated by in vitro assays, and Relenopeg significantly demonstrated more than 20% improvement in pulmonary vascular resistance in a Phase II study. A long-term Phase II open-label study of Relenopeg also showed sustained improvement in six-minute walk distance out to more than two years. A manuscript recently published in the journal Advances in Therapy describes these exciting findings. Now, while we and others have made progress at extending lives and improving patient outcomes through tryprosinol and other therapies, the only known cure for PAH remains a lung transplant. That also is the case for IPF. The problem for PAH, IPF, and many other patients with end-stage organ disease is that there aren't enough donors and transplantable organs available to address the need. And for many organ donations, One life sadly must be lost to save another. We believe the best solution to this problem is to create a supply, an unlimited supply, of tolerable, transplantable, manufactured organs. Even better, with an unlimited supply of organs, transplant can become a consideration for therapy in countless end-stage organ diseases for which there are few good treatment options. Accordingly, We have been developing several investigational approaches using multiple technologies with different organs, all with this same goal in mind. The first is our ex vivo lung perfusion service, or EVLP, which has led to over 380 lives saved with lungs that have undergone EVLP in our facilities in Silver Spring, Maryland, and at the Mayo Clinic Jacksonville campus. Beyond EVLP, we have four platforms, xenotransplantation, regenerative medicine, 3D biome printing, and bioartificial organs. These four platforms cover four key organs, lung, heart, kidney, and liver. Starting with xenotransplantation, we continue to work with the FDA on a clinical path forward. We're underway with what we call pivotal preclinical studies in baboons at the request of the agency. Specifically, for our 10-gene program, we expect the last preclinical xenokidney transplant to occur in the first half of 24. We expect to meet with the agency later this year to discuss the IND and clinical protocol for human studies for 10-gene xeno organs. In parallel with the pivotal preclinical studies, the construction of our clinical scale designated pathogen-free facility, or DPF, in Virginia is complete, and we dedicated the facility earlier this month. We expect the facility to begin receiving pigs this quarter and for the facility to grow its population through the balance of 2024 in preparation for clinical studies in humans for both xenokidneys and xenoharts. Super, super exciting and wow, just breathing so much hope into the entire transplant space. Last month, we received FDA clearance of our investigational new drug application that allows the mural liver elapse system to enter human clinical trials. By the way, this non-registration study will be the first ever clinical study of a bioengineered organ. Mural liver ELAP is an external liver assist product, or ELAP, that is designed to provide liver support in the critical care setting. Acute liver failure is a devastating condition with no approved therapies. A liver transplant is often the only way to save these patients. The ELAP is intended to give the patient's liver a chance to heal itself, possibly reducing the need for liver transplantation. We look forward to providing more details on this program in the coming quarters. I'm thrilled that we're in such a great position at United Therapeutics. We have a solid commercial business posting record results with continued strong growth ahead, a pipeline of novel therapies that continue our strong double-digit revenue growth, and a long-term plan to address one of the largest critical unmet medical needs in medicine, all while helping our patients, employees, and shareholders succeed. I'll now turn the call over to our president, Michael Benkiewicz, who will give an overview of our commercial performance and expectations for potential competition this year. Michael?