Agios Pharmaceuticals, Inc.

Good morning, and welcome to AGO's fourth quarter 2023 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 this call is being recorded at AGO's request. I would now like to turn a call over to Chris Taylor, Vice President, Investor Relations, and Corporate Communications for AGO. Please go ahead.

Thank you, Operator. Good morning, everyone, and welcome to AGO's conference call and webcast. to discuss fourth quarter and full year 2023 financial results and recent business highlights. You can access slides for today's call by going to the Investors section of our website, agios.com. On today's call, I am joined by our Chief Executive Officer, Brian Goff, Dr. Sarah Hewins, Chief Medical Officer and Head of Research and Development, 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 events and results 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. With that, I'll turn the call over to Brian.

Thanks, Chris, and 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. Driven by this goal, we continue to generate consistent and compelling data across our industry-leading PK activator franchise. And with seamless cross-functional collaboration of the Agios team, we made remarkable progress advancing this mission in 2023. Highlighting this progress, we reported three key data readouts in the last 12 months. In June, we reported positive top-line data from the Phase 2 portion of the RISE-UP study of midipivet, our lead PK activator in sickle cell disease, followed by the full data set in December at ASHE. Despite the field's recent progress in sickle cell disease, there are no novel oral therapies that both improve anemia and reduce sickle cell pain crises, and that is precisely what we aim to deliver with Medipivac. In November, we reported positive data from the open-label Phase 2A study of our other PK activator, AG946, in lower-risk MDS. with 40% of patients achieving the transfusion independence endpoint. And just last month, we reported positive data from the Phase III Energize study of mitopivet in nontransfusion-dependent thalassemia. As a reminder, nontransfusion-dependent or NTD thalassemia accounts for approximately two-thirds of thalassemia in the U.S. and has no FDA-approved treatment option. Despite not requiring regular transfusions, NTD thalassemia patients experience significant impact on quality of life, a wide range of serious morbidities, and an elevated risk of premature death. Together, the consistency of data generated across the Mitipivac Development Program bolsters our conviction in the probability of success of our ongoing studies including two additional phase three readouts we expect by the end of this year. And this data highlights the potential of our PK activators to transform the course of multiple hematologic diseases. Sarah will provide more detail on our advancements and upcoming milestones across our pipeline in just a few minutes. Importantly, we believe our PK activation pipeline is well positioned with multiple near-term catalysts to become a multibillion-dollar franchise and deliver significant value. In parallel with advancing the late-stage mid-PIVAT development program across multiple indications, our commercial organization is laser-focused on building the infrastructure established through our current launch in PK deficiency to prepare for potential US launches of mid-PIVAT in thalassemia in 2025 and in sickle cell disease in 2026. Sveta will provide greater detail on the commercial opportunities for Midipivet in thalassemia, as well as an update on our current launch in PK deficiency in just a bit. Finally, as you'll hear from Cecilia, we ended 2023 with a strong cash position with approximately $806 million in cash and investments on the balance sheet. In addition, we continue to track Servier's progress toward the potential FDA approval of voracidinib given our retained economics for both a milestone and royalties. This is truly an exciting time at Agios with four additional phase three readouts and two potential launches expected on the horizon. We look forward to multiple opportunities to drive significant near-term value creation for patients, caregivers, and shareholders. With that, I'll now turn the call over to Sarah.