Agios Pharmaceuticals, Inc.

Good morning and welcome to AGIOS's fourth 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 this call is being recorded at AGIOS's request. I would now like to turn the call over to Chris Taylor, VP Investor Relations and Corporate Communications for AGIOS.

Thank you, Operator. Good morning, everyone, and welcome to AGIOS conference call and webcast. to discuss our fourth quarter and full year 2024 financial results and recent business highlights. You can access the 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, 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. 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. We are especially focused on rare diseases that result in the dysfunction and destruction of red blood cells, including pyruvate kinase deficiency, thalassemia, sickle cell disease, and lower-risk myelodysplastic syndromes, or MDS. Our lead product, PyroKind, 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. Today, we are pleased to share with you our results from the fourth quarter, as well as reflect on accomplishments throughout 2024 and our expectations for the exciting new year ahead. Our rare blueprint for success uniquely positions us to drive significant growth in shareholder value creation over both the near and the long term. First, we have the exciting prospect of two additional commercial launches to support what we consider to be a multi-billion dollar growth opportunity for our lead product, PyroKine. We are planning for a potential approval and launch in thalassemia in September of this year, followed by sickle cell disease in 2026. Second, our early and mid-stage pipeline is robust and poised for clinical advancement, offering a strong foundation for innovation and growth. And finally, supporting it all is our highly experienced team with a proven track record of executional excellence, And our strong balance sheet, which puts us in the enviable position of being able to independently grow the company and execute on these exciting opportunities. 2024 was an exceptional year of executional and scientific excellence at Agios, as shown in this slide, with checkmarks for each of the key milestones we projected one year ago. We meaningfully advanced each of our key programs, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our phase three rise-up study for sickle cell disease. And we continue to progress our early pipeline, building the foundation for sustainable long-term growth. After a transformative 2024, we believe 2025 is a breakout year for Agios. Over the next 12 months, we will focus on three key priorities. Number one, maximizing the potential of the PyroKine franchise. Number two, advancing and diversifying our key pipeline programs. And number three, strategically focusing our capital deployments to sustain and drive our growth. And building on all that was accomplished in 2024, We have another year ahead with compelling commercial, regulatory, and clinical milestones. Today, we announce top-line results from the ACTIVATE Kids Phase 3 Trial of Mitipivet in pediatric patients with PK deficiency who are not regularly transfused. This is Agios' first pediatric clinical program for Mitipivet in a rare hemolytic anemia, And we are excited for Sarah to share with you the positive top-line data from this study in just a moment. We also anticipate some exciting developments for our mid- and early-stage pipeline programs. For Tebapivet, our novel PK activator, formerly known as AG946, we expect to complete enrollment in the ongoing Phase IIb study in lower-risk MDS by year-end, and initiate a phase 2 study in sickle cell disease by mid-2025. Additionally, we expect to file an investigational new drug application for AG236, our siRNA targeting TEMPR6 inhibition intended for the treatment of polycythemia vera in mid-2025. And the most significant expected events for 2025 include the September 7th PDUFA goal date for our SNDA filing of pyrokine and thalassemia, and the phase three readout of the rise-up study of midipivet in sickle cell disease by year end. As you can see, this year promises to be exciting with multiple catalysts across our pipeline that hold significant value for shareholders and have transformative potential for patients. With those introductory comments, let me now hand it off to Sarah to review our exciting progress in R&D.