Agios Pharmaceuticals, Inc.

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

Thank you, Operator. Good morning, everyone, and welcome to AGO's conference call and webcast. to discuss our third quarter 2024 financial results and recent business highlights. You can access the 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, 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 we set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. With that, please 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. We are especially focused on rare diseases that result in the dysfunction and destruction of red blood cells, including pyruvate kinase deficiency, or PKD, thalassemia, sickle cell disease, and low-risk myelodysplastic syndrome, or MDS. As red blood cells comprise over 80% of all cells in the human body, optimizing red cell health represents an important path to improved individual health. Our lead product, Midipivet, a pyruvate kinase activator, has a novel mechanism of action that improves red blood cell metabolism and increases the amount of energy, or ATP, available to support red blood cell health. We are proud to have delivered positive data in Phase III programs in PKD and thalassemia, and we are excited about the prospects of Minipivet for sickle cell disease. Complementing those data readouts and the continued progress across our current pipeline, I am delighted to confirm today that Agios has received $1.1 billion in milestone payments following FDA approval in August of voracidinib, which originated at Agios. These payments include a $905 million payment from Royalty Pharma in connection with the voracidinib royalty purchase agreement Agios announced in May 2024, and a $200 million payment from Servier in connection with Agios' divestiture of its oncology business in 2021. Given the positive phase 3 data readouts of midipivette in thalassemia accomplished this year and the recently announced achievement of full enrollment of the phase 3 rise-up sickle cell disease study, we aim to deploy this strong cash base to prepare for the potential launches of midipivette in thalassemia in 2025 and in sickle cell disease in 2026. Beyond this significant infusion of capital, we also made important progress across our advancing clinical pipeline. First, we announced top-line data from the Phase III Activate Kids T study of midipivet in children with PK deficiency who were regularly transfused, our first pediatric study of midipivet. And second, we achieved two milestones in our clinical development program for lower-risk MDS. We announced that the FDA granted orphan drug designation to our potent PK activator, Tebapivet, formerly known as AG946, for the treatment of myelodysplastic syndromes, or MDS. And we have initiated enrollment and begun patient dosing in the Phase 2B study of Tebapivet in lower-risk MDS, an area with profound unmet need and significant market growth. Importantly, as we round out a very productive and successful 2024, we are on track to achieve our key remaining milestone for this year, submission of an SNDA for mid to pivot in thalassemia by the end of the year, seeking a broad label that includes adults living with all subtypes of thalassemia. Sarah will provide a detailed update on our progress and upcoming milestones across R&D in just a few minutes. Leveraging the positive data observed in the ENERGYZE and ENERGYZE-T Phase III studies of midipivet, our expanding commercial organization is actively preparing for a potential U.S. launch of midipivet in thalassemia in 2025. Sveta will provide greater detail on the market opportunity in thalassemia and the team's robust preparation, 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 third quarter with a very strong cash position with approximately $1.7 billion in cash and investments on the balance sheet, which includes the $1.1 billion in milestone payments we received following FDA approval of voracitinib. And before handing off to Sarah, I want to emphasize how incredibly proud we are for all that's been achieved thus far in 2024. how intensely focused we remain on finishing the year strongly, and our enthusiasm for continued progress in 2025 and beyond. With that, I'll now turn the call over to Sarah.