Agios Pharmaceuticals, Inc.

Good morning and welcome to RGO's first quarter 2024 conference call. At this time, all participants are in a listen-only mode. There will be a question and answer session at the end. Please be advised that today's conference is being recorded at RGO's request. I would now like to turn the call over to Chris Taylor, Vice President, Investor Relations and Corporate Communications for RGOs. Please begin. Chris Taylor, Vice President, Investor Relations and Corporate Communications for RGOs Thank you, Operator.

Good morning, everyone, and welcome to AGIOS conference call and webcast to discuss first quarter 2024 financial results and recent business highlights. You can access slides for today's call by going to the investor section of our website, agios.com. On today's call, I'm joined by our chief executive officer, Brian Goff, Dr. Sarah Hewins, chief medical officer and head of R&D, Sveta Milanova, our Chief Commercial Officer, and Cecilia Jones, Chief Financial Officer. Before we get started, I would like to remind everyone that some of the statements we make on this call will include forward-looking statements. Actual results and events could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. And with that, I'll turn the call over to Brian.

Good morning, everyone, and thank you for joining us. Our mission at Agios is to develop and deliver transformative medicines that elevate and extend the lives of patients living with rare diseases, and we are off to a fast start in 2024. Our foundation today leverages Medipivet's novel mechanism of action, which focuses on overall red blood cell health and has been a key driver for our recent clinical results. On January 3rd, we reported positive data from the Phase III energized study of our lead PK activator, Medipivet, marketed as pyrokine in patients with non-transfusion-dependent thalassemia. This study met both the primary endpoint of hemoglobin response rate, as well as both key secondary endpoints associated with change from baseline in facet fatigue score and average hemoglobin concentration, and we look forward to presenting these data at an upcoming medical meeting. As a reminder, non-transfusion-dependent thalassemia accounts for approximately two-thirds of thalassemia in the U.S. and has no FDA-approved treatment options. Despite not requiring regular transfusions, it is increasingly understood that these patients experience a significant impact on their quality of life, a wide range of serious morbidities, and an elevated risk of premature death due to chronic hemolysis and ineffective erythropoiesis. Based on these data, our team is actively preparing for a potential launch in thalassemia in the U.S. Complementing the ENERGYZE study in non-transfusion-dependent thalassemia, we continue to advance the Phase III ENERGYZE-T study of midipivet in transfusion-dependent thalassemia. We expect to report data from this study in the second quarter, a slightly more refined timeframe than previously communicated, And we plan to submit a single regulatory filing encompassing data from both Energize and Energize-T to the FDA by the end of the year. In parallel, we look forward to near-term milestones across several additional clinical programs in our pipeline, including completing enrollment in the Phase III portion of the Rise Up Study of Mitopivet in Sickle Cell Disease by the end of this year, and reporting data from four additional Phase III studies by the end of 2025. Sarah will provide a detailed update on our progress and upcoming milestones across R&D in just a few minutes. Given the consistent positive data we've generated across the MediPivot development program and the high unmet need in our target disease areas, we believe MediPivot has the potential to transform the course of multiple hemolytic anemias by improving red blood cell health and to become a multi-billion dollar franchise. To help realize the full commercial potential of Medipivet, and based on the strength of the energized data, our commercial organization is laser focused on building upon the infrastructure established through our current launch in pyruvate kinase deficiency, or PKD, to prepare for potential US launches of MidiPivot in thalassemia in 2025 and in sickle cell disease in 2026. Sveta will provide greater detail on the market opportunity in thalassemia and the team's robust preparation for launch, as well as an update on our current launch in PKD in just a bit. Finally, as you'll hear from Cecilia, we ended the first quarter with a strong cash position with approximately $714 million in cash and investments on the balance sheet. Importantly, we have the potential to further bolster our cash position in the near term as Servier announced FDA filing acceptance and priority review for a new drug application for voracitinib for the treatment of certain IDH mutant diffuse glioma. You'll recall that as part of the divestiture of Agios' oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of voracidinib and 15% royalties on potential U.S. net sales. If approved, voracidinib would become a first-in-class targeted therapy for patients with IDH mutant gliomas, and we look forward to the PDUFA With that, I'll turn the call over to Sarah.