Agios Pharmaceuticals, Inc.

Good morning, and welcome to AGO's second quarter 2024 conference call. At this time, our 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 the call over to Chris Taylor, VP, Investor Relations and Corporate Communications for AGO.

Thank you, Operator. Good morning, everyone, and welcome to AGO's conference call and webcast to discuss second quarter 2024 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'm 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, our Chief Financial Officer. Before we get started, I'd 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. And with that, I'm pleased to turn the call over to Brian.

Thanks, Chris. 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. Our foundation leverages MediPivot's novel mechanism of action, which improves overall red blood cell health and has been a key driver of our positive clinical data readouts in recent years. I'm delighted to report that we continued our positive momentum in Q2 with multiple key milestones announced this quarter. First, we announced that the Phase III energized T study of midipivet met both the primary endpoint and all key secondary endpoints in adults with transfusion-dependent alpha or beta thalassemia. Importantly, ENERGYZE-T is the first Phase III study to demonstrate efficacy of an oral disease-modifying treatment for transfusion-dependent alpha and beta thalassemia. Second, data from the positive Phase III ENERGYZE study of mitopivet in adults with alpha or beta thalassemia who are not regularly transfused were presented by leading KOLs in two presentations at the recent European Hematology Association Congress in Madrid, including a plenary session. Taken together with the positive data from energized tea, we plan to submit an SMDA for mid to pivot in thalassemia based on all available data from both energized and energized tea by the end of 2024, seeking a label that covers people living with all subtypes of thalassemia. Third, we announced that AGIOS has agreed to sell its rights to a 15% royalty on potential U.S. net sales of Cervia's voracitinib to Royalty Pharma. Under the terms of the agreement, AGIOS will receive an upfront payment of $905 million upon approval of voracitinib by the FDA. Cecilia will revisit the details in a moment, and I would like to thank her for directing the process and structuring a tremendous agreement for Agios, our shareholders, and a win-win for both Agios and Royalty Pharma. This transaction will provide us with the financial independence to prepare for potential launches in thalassemia and sickle cell disease as we build a multi-billion dollar PK activation franchise as well as the ability to opportunistically expand our pipeline. And fourth, just this morning, we announced top line data from the Phase III Activate Kids T study of midipivet in children with PK deficiency who are regularly transfused. This is truly a pioneering study for AGIOS, a first pediatric study of midipivet and representative of our commitment to pursue the potential of midipivet as a treatment for children with rare diseases. We are also pleased to report that we have completed enrollment in the Activate Kids study, and we look forward to the top-line readout in 2025. Building on this progress, we look forward to several additional upcoming milestones, including completing enrollment in the Phase 3 portion of the Rise Up Study of Mitipivet in Sickle Cell Disease by the end of this year and reporting data next year. Sarah will provide a detailed update on our progress and upcoming milestones across R&D in just a few minutes. Given the consistent and compelling data we've generated across the Mitipivet development program and the high unmet need in each of our target disease areas, we believe Mitipivet has the potential to transform the course of multiple hemolytic anemias by improving red blood cell health and to become a multibillion-dollar franchise. Leveraging the positive data observed in the energized and energized T studies of midipivet, which together encompass all thalassemia subtypes, our expanding commercial organization is actively preparing for a potential U.S. launch of midipivet in thalassemia in 2025, as well as a potential launch 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 PK deficiency in just a bit. As part of our news release this morning, we were delighted to announce two updates related to the commercialization of pyrokine outside the U.S. First, we've entered into a distribution agreement with New Bridge Pharmaceuticals, a leading player in the Gulf region, and second, Midipivet has received Breakthrough Medicine designation from the Saudi FDA. Finally, as you'll hear from Cecilia, we ended the second quarter with a strong cash position with approximately $645 million in cash and investments on the balance sheet, and we have the potential to further bolster our cash position by an additional $1.1 billion upon the potential FDA approval of Oracidinib. With that, I'll turn the call over to Sarah.