Travere Therapeutics, Inc.

Good day, and thank you for standing by, and welcome to Previer Therapeutics Third Quarter 2021 Financial Results and Corporate Update Call. At this time, all participants are in a listen-only mode. Please be advised that this call is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to Chris Klein, Senior Vice President, Best Relations, Corporate Communication. Please go ahead.

Thank you, Justin. Good afternoon, and welcome to Travere Therapeutics' third quarter 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 Dubay. Eric will be joined for the prepared remarks by Dr. Noah Rosenberg, our Chief Medical Officer, Peter Herma, 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 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 factors section in our Forms 10Q and 10K file with the SEC. In addition, any forward-looking statements represent our view only as of the date such statements are made, October 28, 2021. The interviewer specifically disclaims any obligation to update such circumstances that reflect future information, events, or circumstances. With that, let me now turn the call over to Eric. Eric?

Thank you, Chris. And by the way, happy birthday today. Good afternoon, everyone. I am proud of the progress made across all facets of our business during the third quarter. First, I am pleased to report that we achieved several significant milestones related to our goal of positioning sparsentin to ultimately become the new treatment standard for IJ nephropathy and FSGS if approved. In August, we reported that our pivotal Phase III PROTECT study of sparsentin in IJ nephropathy achieved its primary proteinuria endpoint with statistical significance in its pre-specified interim analysis. The result exceeded our expectations by demonstrating a greater than three-fold reduction in proteinuria from baseline compared to those patients receiving the active control erbisartan. And at the time of the interim assessment, sparsentin was generally well-tolerated and consistent with the observed safety profile to date. This is the first time a single non-immunosuppressive agent has demonstrated this magnitude of effect on proteinuria reduction in a large, well-controlled study. And we know from our engagement with the nephrology community that a treatment with these attributes is precisely what physicians are seeking to help prevent progression to end-stage kidney disease. I'm very pleased to report today that we have completed our pre-NDA interactions with the FDA for sparsentin in IJ nephropathy. Notably, the FDA agreed that the interim analysis from the PROTECT study supports submission of an application for accelerated approval under subpart H. Based upon the agency's feedback and alignment on our structure of the planned NDA, we expect to submit our application for accelerated approval in the U.S. in the first quarter of next year. We also made meaningful progress on our sparsentin program for FSGS during the quarter. Most importantly, we gained alignment with the FDA on our plan to provide the agency with additional EGFR data from the ongoing duplex study in the first half of 2022. which enables us to continue on the accelerated approval pathway for FSGS. If the additional EGFR data further strengthen the prediction of long-term benefit in the study, as we expect they should, we anticipate submitting an NDA for accelerated approval for FSGS in the middle of next year. We are pleased that we now have a clear regulatory path to potential accelerated approval for sparsentin for both IgA nephropathy and FSGS in the US. And we look forward to achieving those submissions next year. I'm also pleased to share that we have formalized our plan to pursue a combined IgA nephropathy and FSGS MAA submission for sparsentin in Europe. This strategic decision will allow us to pursue the most expedited path to making sparsentin available for both IgA nephropathy and FSGS in Europe. And we believe it will increase the probability of success for achieving optimal market access across regions. Additionally, this option allows us to potentially request accelerated assessment for the review process. We will continue our engagements with the EMA and expect the MAA application to be submitted mid-next year. once the planned additional EGFR data from the duplex study can be incorporated, if supported. Also, as it relates to Europe, we were very pleased to recently enter into a joint collaboration and licensing agreement with V4 Pharma for the commercialization of sparsentin in Europe, Australia, and New Zealand. This collaboration aligns the strength of two leaders in rare nephrology with a shared mission to making sparsentin the new treatment standard for IJ nephropathy and FSGS if approved. And it significantly increases our probability of success across all launches, as it will allow us to have the dedicated focus needed for successful launches in these substantial markets. The collaboration also recognizes the value sparsentin can bring in Europe and strengthens our financial foundation, with potential milestone payments totaling up to $845 million and royalties on sales in these regions of up to 40%. We look forward to working closely with V4 as they integrate Sparsentin into their portfolio and prepare for potential launches in Europe, Australia, and New Zealand beginning in 2023. Beyond Sparsentin, we continue to be encouraged by the potential for our novel PEG-debatinase program in classical homocystinuria, or HCU. We believe that it has the potential to become the first disease-modifying therapy for the more than 7,000 people in the U.S. and Europe who are not adequately responding to current treatment options. We remain on track for a preliminary assessment from the ongoing Phase I-II trial, now named the COMPOSE study, before year-end. And we look forward to establishing next steps for the program if the data are encouraging as we expect. Lastly, I'd like to highlight the performance of our commercial organization in the third quarter. Our execution led to another quarter of year-over-year growth across all approved products, despite the ongoing challenging environment created by the COVID-19 pandemic. This performance underscores our confidence in the ability to successfully deliver sparsentin and pegdabatinase if approved. Let me now turn the call over to Noah for the clinical update. Noah?