Travere Therapeutics, Inc.

Ladies and gentlemen, and welcome to the Travere Therapeutics second quarter 2021 financial results and corporate update call. At this time, all participants are in a listen-only mode. Later, we will conduct question and answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star, then zero on your touchtone 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. Please go ahead.

Great. Thank you, Jerome. Good afternoon, everyone, and welcome to Travere Therapeutics' second quarter 2021 financial results and corporate update call. Thank you for joining us. 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 Herrmann, our Chief Commercial Officer, and our Chief Financial Officer, Laura Clay. Dr. Bill Rote, Senior Vice President of Research and Development, will join us for the Q&A session. Before we begin, I would 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. We 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, July 29, 2021. And Travere specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. With that, I'll now turn the call over to Eric. Eric?

Thank you, Chris, and good afternoon. At the outset of the year, we highlighted the progression of our pipeline as a key priority in order to strengthen our position in the rare disease community. At the heart of this priority is an organization-wide focus on positioning sparsentin to potentially become a new treatment standard for FSGS and IgA nephropathy if approved. People living with FSGS and IgA nephropathy have gone decades without adequate treatment options and limited innovation. As a result, each year thousands of patients with these disorders are advancing to kidney transplant or dialysis. It is a collective understanding of this significant unmet need that drives our organization every day to help pave the way for new therapeutic options for these patients. Being at the forefront of these efforts can come with both achieving significant milestones as well as challenges that must be overcome. This was evident in the first half of 2021 where we generated positive results from the interim proteinuria assessment of our pivotal duplex study of sparsentin and FSGS, but subsequently heard from FDA that the interim data were not adequate to support an accelerated approval submission this year. Despite this, we remain undeterred in our commitment to ultimately deliver sparsentin as a potential new treatment standard for FSGS. We remain confident in the interim data generated by the duplex study, the largest controlled interventional study in FSGS to date. These data build upon the strong proof of concept from the Phase II duet study and provide a consistent data package to further inform sparsentin's profile. At our upcoming scheduled Type A meeting this quarter, we look forward to continuing our constructive dialogue with FDA in an effort to pursue accelerated approval submission in the U.S. next year. Outside of the U.S., I am pleased to share that we have made progress on our ongoing regulatory discussions with the EMA. Based upon our latest interactions, we anticipate submitting an application for conditional marketing authorization of sparsentin for the treatment of FSGS in Europe by the end of this year. NOAA will go into a bit more detail shortly, but we believe that if we continue to progress according to plan, sparsentin could be approvable for FSGS in Europe by during the first half of 2023. We also remain excited about the potential for sparsentin to become an important new treatment option for patients with IgG nephropathy. IgG nephropathy is the most common cause of primary glomerular nephritis and a leading cause of end-stage kidney disease. If ultimately approved, we believe there to be between 75,000 and 120,000 addressable patients at launch in the US and Europe. with room for greater patient identification through increased awareness and diagnosis over time. Our phase three PROTECT study evaluating sparsentin in patients with IgA nephropathy is progressing well. And we remain on track for top line data from the 36 week interim proteinuria assessment next month. The interim assessment is designed to support potential accelerated approval submissions in the US and Europe. which could result in the first approval for IgA nephropathy as early as next year. Finally, in the pipeline, we are encouraged by progress made with the PEG-debatinase program, where we remain on track to see preliminary data from the Phase I-II study later this year. We will also be working to lay the foundation for our regulatory pathway in HCU by leveraging both data from the Phase I-II study and the ongoing natural history study. Lastly, I'd like to touch on our commercial portfolio. As we have talked about for the last several years, we believe our commercialization capabilities in the U.S. are a strategic strength for Travere's ability to deliver our current product candidates if approved. We have demonstrated a consistent ability to identify and treat new patients living with rare kidney and liver conditions. The second quarter is another example of our team's resilience. as evidenced by the rebound in performance following the impact of the pandemic in the first quarter. We experienced year-over-year growth across all of our products, which speaks to the continued important role they play for patients. Looking ahead, we continue to expect underlying demand for our products, but we do anticipate that the recently announced generic version of the original formulation of Fiola will have an unfavorable impact on our net product sales growth in the second half of the year. At this time, we are not in a position to reliably estimate what that impact will be, but we plan to provide updates as we learn more about the evolution of the market. This is a scenario that has been part of our business planning for some time, and we remain committed to the Cystinuria community. It also does not change our confidence in or plans to continue identifying and treating patients. and building upon our commercialization capabilities to successfully deliver sparsentin if approved. Let me now turn the call over to Noah for updates from the pipeline. Noah?