Travere Therapeutics, Inc.

Good afternoon ladies and gentlemen and welcome to Travere Therapeutics fourth quarter and full year 2020 financial results and corporate update. At this time all participants are in the listen only mode. Later we will conduct a question and answer session and instructions will follow at that time. If anyone should require assistance during the conference please press star zero on your touchstone telephone. As a reminder this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Chris Klein. Thank you. Please go ahead, sir.

Thank you, Katrina. Good afternoon, and welcome to Travere Therapeutics' fourth quarter and full year 2020 Financial Results and Corporate Update call. Thank you for joining us. I hope you all remain well. Today's call will be led by our Chief Executive Officer, Dr. Eric Dubé. Eric will be joined for the prepared remarks by our Chief Medical Officer, Dr. Noah Rosenberg, Peter Herma, our Chief Commercial Officer, and our Chief Financial Officer, Laura Clegg. Dr. Bill Rote, 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 risks, uncertainties, and assumptions that may cause actual results, performance, and achievements to differ materially from those expressed or implied by the statements. Please see the forward-looking statement disclaimer on the company's press release issued earlier today, as well as the risk factor 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, March 1, 2021, and Travere 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, Chris, and good afternoon, everyone. We were very pleased with the beginning of the year on a high note with the duplex study recently achieving its interim proteinuria endpoint. And we look forward to discussing a bit more about that with you shortly. First, I would like to reflect on the excellent execution that we had in 2020. This is a direct result of our team members' hard work and dedication to our mission of identifying, developing, and delivering life-changing therapies to people living with rare disease. Our key objectives last year were focused on three areas aimed at strengthening our position as a leader in the rare disease community. The first was advancing our two pivotal phase three programs for sparsentin in FSGS and IJ nephropathy. In doing so, our goal was to continue to position sparsentin to potentially become a new treatment standard for these two rare kidney disorders if approved. Despite the ongoing pandemic, our clinical and operational teams have done a phenomenal job of ensuring patient safety and continued high quality in both the duplex study in FSGS and the PROTECT study in IJ nephropathy. Furthermore, their efforts led to both studies achieving the key enrollment milestones necessary to enable top-line readouts from the interim proteinuria endpoints, including PROTECT, which is now well ahead of its original schedule. Both of these interim readouts are designed to support potential accelerated approval and conditional marketing authorization submissions in the U.S. and Europe beginning this year. The second key objective was building upon our established commercialization capabilities to identify new patients that may benefit from our approved products, as well as to begin preparing our organization for a successful launch of sparsentin if approved. I am pleased to report that we maintain supply and access for our patients throughout the pandemic last year. We also continue to identify new patients for all of our approved products. In preparation for a potential launch of sparsentin, we furthered our understanding of the patient journey in both FSGS and IJ nephropathy. This includes a deeper understanding of patients' needs, where patients may be identified, the therapeutic profile physicians desire for their patients, and the future role that earlier diagnosis can play. Together with our established commercialization capabilities, we believe that these deeper insights provide us with a clear pathway to be successful in delivering sparsentin if approved. Our third key objective was to further diversify our pipeline through our disciplined business development efforts. In the fourth quarter of last year, we added to our pipeline TBT058, a novel investigational human enzyme replacement therapy that is currently in phase 1-2 development for the treatment of classical homocystinuria, or HCU. Current treatment options for HCU are limited and ineffective for many. Preclinical data suggests that TBT058 has the potential to be the first disease-modifying therapy in HCU. We believe this program is an ideal fit for our mission and expertise. It allows us to leverage our late-stage development and commercialization capabilities to potentially deliver a new treatment option for people living with HCU. And it provides the potential for meaningful growth on top of sparsentin. I am incredibly pleased with our organization's performance in 2020, which is even more notable given the challenges that we have all faced in adjusting to the COVID-19 pandemic. This execution led us to meeting or exceeding our objectives for last year, and importantly, it set us on a path for a potentially transformative year in 2021. This potential is driven by multiple exciting catalysts anticipated from our pipeline this year. As I mentioned earlier, our duplex study in FSGS achieved its pre-specified interim FSGS partial remission of proteinuria endpoint or FPRE after 36 weeks of treatment. Based on the data from the interim analysis, we intend to pursue submissions for accelerated approval of sparsentin for FSGS in both the U.S. and Europe. With the interim readout behind us, our focus turns to preparing for our upcoming regulatory interactions and further building towards our goal of delivering sparsentin as a new treatment standard in FSGS if approved. We look forward to engaging with regulators in the coming months and continuing our preparations for NDA and MAA submissions later this year. The interim assessments from Duplex and FSGS has also increased our confidence in the potential for sparsentin to have a meaningful treatment effect on proteinuria in IJ nephropathy. The ongoing pivotal PROTECT study in IgA nephropathy remains on track to report top-line results from the interim proteinuria assessment in the third quarter of this year. If successful, this milestone could be the next significant step towards potentially reaching our goal of delivering sparsensin as a new treatment standard in both FSGS and IgA nephropathy. While we certainly have a number of important updates planned for Sparsentin this year, we do also expect preliminary data from our ongoing phase one to proof of concept study of TBT058 in HCU to become available later this year. We are excited to have this program now fully in-house and to advance our understanding of the candidate and its potential in HCU. Our goal throughout the year will be to identify the most expeditious path forward to help address the significant unmet need for patients in this community. Let me now turn the call over to Noah for an update on our clinical programs. Noah?