Travere Therapeutics, Inc.

Good day and welcome to the Travere Therapeutics fourth quarter and full year 2023 financial results and corporate update conference call. Today's conference is being recorded. At this time, I would like to turn the conference call over to Ann Crotto. Please go ahead, ma'am.

Thanks, Jenny. Good afternoon and welcome to Travere Therapeutics fourth quarter and full year 2023 financial results and corporate update call. Thank you all for joining. Today's call will be led by our President and Chief Executive Officer, Dr. Eric Dubé. Eric will be joined in the prepared remarks by Dr. Jule Inrig, our Chief Medical Officer, Peter Harama, our Chief Commercial Officer, and Chris Klein, our Chief Financial Officer. Dr. Bill Roat, Senior Vice President of Research and Development, will join us for the Q&A session. Before we begin, I'd like to remind everyone that statements made during this call regarding matters that are not historical facts are forward-looking statements within the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees of performance. They involve known and unknown risk, uncertainties, and assumptions that may cause actual results, performance, and achievements to differ materially from those expressed or implied by the statement. Please see the forward-looking statement disclaimer on the company's press release issued earlier today, as well as the risk factors section in our Forms 10-Q and 10-K file with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made, February 15, 2024, and Trevier specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. With that, let me now turn the call over to Eric. Eric?

Thank you, Anne, and welcome, everyone. 2023 was a year of many great achievements for Travere as we worked towards our goal of breaking down barriers and treating rare diseases with historically little innovation. At the start of 2023, we gained the first approval from our pipeline, delivering sparsentin, or Silspari, as the first and only non-immunosuppressive treatment option for people living with IgA nephropathy, or IgAN. For decades, people living with IgAN, most of whom are diagnosed in their 20s and 30s, and on average face kidney failure in 10 years, had limited treatment options. We are proud to help lead the growing evolution of the treatment paradigm that we believe will ultimately see patients get diagnosed and initiate treatment earlier, and where physicians will ultimately utilize Philzari as a foundational treatment with its superior prognuri reduction and accrual of kidney preservation benefits. Following the grant of accelerated approval, Our team quickly initiated our comprehensive commercial launch plan and worked throughout the year to lay a strong foundation for Fils-Fari uptake. I am very pleased with our results. We continue to make substantial progress in physician demand, payer coverage, and revenue, the key aspects of the launch. Of note, Fils-Fari is the only recent launch in the rare kidney space that has seen consistent growth in demand each quarter in its first year, and we saw a meaningful inflection in net product sales to close out 2023. Last year, we encountered a challenge in our pursuit of a better outlook for the FSGS community. Specifically, the duplex study of sparsentin in FSGS did not achieve the results we had hoped. And following our FDA engagement, it was clear we would not be in a position to submit an S&DA at that time based on the duplex results alone. I am incredibly grateful and proud of how our colleagues at Travere learned and quickly adjusted We move quickly to align our investment in this program, implement a strategic restructuring of our organization to focus our resources, and concentrate our efforts to collaborate with regulators with the goal of identifying a potential regulatory path forward in the future. As for PEG-2-Batinase Development Program for Classical Homocystinuria, or HCU, we achieved important milestones in 2023 globally. We believe there are 7,000 to 10,000 patients diagnosed with HCU who are not in full control of their homocysteine levels. With better diagnoses and a future where an effective treatment is available, we believe this may grow by 50% or more over time. Last year, we generated additional exciting data from our Phase I to Compose study, which further demonstrates the potential for PEG-debatinase to become the only disease-modifying therapy for HCU. With these data, we worked closely with regulators to align on a phase three program, utilizing total home assisting reduction as a primary endpoint, and initiated that study before year end. All of our efforts last year positioned us to start 2024 with focus and a plan for execution across the board. Strong launch performance at Pillsbury remains our top priority for 2024, and we expect the momentum in our launch from the second half of 2023 will continue into the new year. We are also executing on three additional priorities aimed at broadening access to PhilSparry. Of note, we are on track to submit our S&DA this quarter to support conversion of PhilSparry from accelerated approval to full approval for IGAN in the U.S. Together with our partner CSLV4, we expect an opinion on conditional approval of PhilSparry in Europe from CHMP later this quarter. We're optimistic that this will be positive. And with our recent agreement with Rinalis, We are looking forward to aiding their development plans to ultimately enable access to filspari in Japan and other regions in Asia, where IGAN is an even more prevalent disease and leading cause of kidney failure. And importantly, we are excited about the opportunity we have with PEG-debatinase, our novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocysteineuria, or HCU. In 2024, our focus will be on enrolling our Phase III Harmony Trial and raising awareness of the need for innovative treatments for this rare disorder. Let me now turn the call over to Jula for a clinical update. Jula?