Travere Therapeutics, Inc.

Good day. Thank you for standing by. Welcome to Travier Therapeutics' fourth quarter and full year 2021 financial results and corporate update. At this time, all participants' lines are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Mr. Chris Klein. Thank you. Please go ahead.

Thank you, Buena. Good afternoon, and welcome to Travere Therapeutics' fourth quarter and full year 2021 financial results and corporate update call. Thank you all for joining us. Today's call will be led by our Chief Executive Officer, Dr. Eric Duvet. Eric will be joined for the prepared remarks by Dr. Jule Inrig, our Chief Medical Officer, Peter Herma, our Chief Commercial Officer, and our Chief Financial Officer, Laura Clegg. Dr. Bill Rote, Senior Vice President of Research, will join us for the Q&A session. Before we begin, I'd like to remind everyone that statements major in 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 statement. 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 view only as of the date such statements are made, February 24, 2022. And Travere specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. Let me now turn the call over to Eric.

Eric? Thank you, Chris, and good afternoon, everyone. 2021 was an outstanding year for Travere. Our pipeline of potential first-in-class rare disease treatments delivered three positive top-line readouts from our ongoing studies. We made significant regulatory progress and established pathways to potential accelerated approvals for our lead candidates, sparsentin, in both IgA nephropathy and focal segmental glomerular sclerosis, or FSGS. We delivered 6% growth in net product sales from our commercial products, despite the ongoing challenges from COVID, and a generic entrant for thiola. And we continue to strengthen our ability to successfully deliver our treatments to the rare disease community in the future. I'll touch on each of these areas briefly. First, regarding the pipeline, we are incredibly pleased with the outcomes from both of our Phase III studies of sparsentin, duplex in FSGS and protect in IJ nephropathy. With positive top-line interim readouts from both studies, sparsentin has established a robust body of evidence around its ability to reduce proteinuria in patients with rare kidney diseases. Importantly, the sparsentin readouts have positioned us for three potential regulatory submissions this year. I am pleased to report that we remain on track to submit an application for accelerated approval under subpart H for IgA nephropathy during this quarter. If that application is accepted and granted priority review, we would anticipate a PDUFA date for sparsentin and IgA nephropathy towards the end of this year. We are also on track to submit to the FDA additional EGFR data from the ongoing duplex study in the first half of this year. If at that time the data further strengthen the prediction of long-term benefits in the study as we should expect they should, We anticipate submitting an MDA for accelerated approval for FSGS around the middle of this year. Together with our European partner, V4 Pharma, we also remain on track to submit a combined IgA nephropathy and FSGS MAA submission for conditional approval for sparsentin in Europe, pending additional support of EGFR data from Duplex. As you can see, we have a very exciting year ahead for sparsentin. Also from the pipeline, in December, we reported positive top line data from the ongoing phase one, two composed study of PEG-2-batinase in classical homocysteineuria, or HCU, a rare and devastating metabolic disorder. We believe these data support the potential for PEG-2-batinase to become the first therapy targeting the underlying cause of HCU for the more than 7,000 people in the US and Europe who are not able to adequately control their HCU with the available treatment options today. Having achieved this milestone also reinforces confidence in our strategy of bringing in external programs to further address the unmet needs of rare disease patients while diversifying and building our growth potential. This year, we look forward to engaging with regulators to establish next steps for a pivotal development program while we also gain experience with formulation enhancements and further explore the dose-response curve in the final plan cohort of the study. On the commercial side of the business, we continued our track record of strong execution. Our results in 2021 marked the sixth straight year of organic growth. We believe this illustrates the strength of our commercial capabilities and our ability to identify, treat, and support patients living with rare kidney and liver conditions. For 2022, we currently expect to see continued growth of our bile acid products, but we do expect pressure on the Fiola business. As a result, our consecutive growth streak in total net product sales is not likely to continue this year. But with the potential for Sparsentin to launch as early as the end of this year, we look forward to returning to growth again in the future. Importantly, our business planning has been accounting for this. Peter and his team are continuing to build upon our commercial strengths to translate our experience and establish relationships for successful launches of sparsentin in the U.S. Finally, I'd like to introduce Dr. Jule Enrich, our new chief medical officer. Jule joined us in January and has taken over for NOAA following his planned transition. Jule is a nephrologist who brings to Travere more than 15 years of expertise in drug development, clinical trial planning and execution, global regulatory engagement, and medical oversight. She has a clear passion for championing positive change for patients and a deep history of advancing new treatment options for rare diseases, specifically in rare nephrology. She joins our team at an exciting time as we approach the next inflection point on the path to bringing innovation to patients living with IgA nephropathy and FSGS. With that, let me turn the call over to Jula for the clinical update. Jula?