Travere Therapeutics, Inc.

Hello, and welcome to the Travere Therapeutics first quarter financial results and corporate update. My name is Brandon, and I'll be your operator for today. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session, during which you may dial star 1 if you have a question. Please note this conference is being recorded, and I will now turn it over to Chris Klein. You may begin, sir.

Great. Thank you, Brandon. Good afternoon, and welcome to Travere Therapeutics first quarter 2021 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 Dube. 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 would 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 guaranteed to 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 views as of the date such statements are made, May 6, 2021. Interviewee 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?

We remain dedicated... Sorry, technical difficulties. We remain dedicated to the key priorities that we believe will enable us to strengthen our position of leadership in the rare disease community. And the first quarter of this year was another example of our organization's ability to execute. For us, this centers on developing our pipeline for potential first-in-class programs targeting rare diseases that have significant unmet needs. Earlier this year, the Every Life Foundation published the National Economic Burden of Rare Disease Study. The study found that on average it takes approximately 6.3 years for a person living with a rare disease to find a diagnosis, and that patients typically see upwards of 17 specialists along their diagnostic journey. Overall, the study estimated that the direct medical cost associated with rare diseases in the US was more than $400 billion in 2019. This is especially relevant given the challenges the rare nephrology community faces due to the limited innovation over the past few decades for diseases like FSGS and IJ nephropathy. For example, FSGS is considered to have one of the worst prognoses amongst primary glomerular diseases. It is estimated that more than 50% of FSGS patients with persistent nephrotic proteinuria will reach end-stage kidney disease within 10 years. This translates to over 2,000 people each year reaching FSGS-related end-stage kidney disease in the U.S. Patients today have a long diagnostic journey, experience progressive loss of kidney function, and become dependent upon transplant and dialysis. This is why we have great urgency in pursuing our goal of developing sparsentin to become a new treatment standard for FSGS and IgA nephropathy if approved. We remain encouraged by the interim proteinuria data that we reported from the duplex study in February. The data showed that treatment with sparsentin resulted in a 60% greater relative likelihood of achieving FPRE when compared to erbosartan. The data also showed that sparsentin has been generally well tolerated. And overall, the safety profile between treatment groups in the study at the time of the analysis were generally comparable, which is encouraging. We are in the process of engaging with regulatory agencies to discuss potential accelerated approval submissions for FSGS. As such, we will not be in a position to provide a regulatory update today, but remain on track to do so as planned before the end of the first half of the year. In parallel, our PROTECT study of sparsensin in IJ nephropathy continues to advance and is nearing completion of enrollment. The study continues to receive broad support from the nephrology community and from patients and their families. Importantly, we remain on track for a top line readout from the interim proteinuria assessment in the third quarter of this year. And we look to the data from PROTECT. We remain encouraged by the consistency with which sparsentin has demonstrated its ability to meaningfully reduce proteinuria throughout its development. We also continue to see additional trial-level analyses published in the space, specifically in IG nephropathy, that clearly link the benefit of proteinuria reduction with beneficial outcomes in EGFR. Importantly, these are consistent with how we have designed our PROTECT study, and we are looking forward to those results coming up soon. We also continue to be excited about PEG-tobatinase, the new non-proprietary name for our TBT058 molecule in development for classical homocystinuria, or HCU. There are no approved treatments that specifically address the underlying genetic cause of HCU. The literature suggests that without control of HCU, approximately 25% of patients by age 16 and 50% of patients by age 29 have thromboembolism, which can lead to heart attack and stroke. Available treatments are often inadequate and patients also face challenges with compliance and adhering to a difficult, low-protein diet, especially as they age. So we believe that there is a meaningful opportunity to help the more than 7,000 patients estimated to be living with classical HCU in the U.S. and Europe and in need of an effective treatment option. Our team is working with investigators and patients to advance the ongoing Phase I-II study. And we continue to be encouraged by the potential to ultimately make pegtobatinase the first disease-modifying therapy for classical homocystinuria. We look forward to providing additional updates on the program later this year. Our leadership position in the rare disease community is also built upon our ability to consistently deliver our approved therapies to patients. In the first quarter, we experienced some challenges in new patient referrals from the ongoing pandemic. but our commercial organization continued to deliver, and the underlying strength of our business remains. Importantly, we have maintained consistent access and support for our approved treatments through the challenges over the last year. And while we had a slower than anticipated month for new patient starts in January and February as a result of the resurgence of COVID in the fall, we did see new patient referrals meaningfully increase in March and through April. This provides us with the confidence that we can achieve our full-year guidance of mid-single-digit growth and ultimately draw upon our expertise and resilience to be successful in delivering sparsentin if approved. Let me now turn the call over to Noah for updates from the pipeline. Noah?