Agios Pharmaceuticals, Inc.

Good morning and welcome to AGO's first quarter 2025 conference call. At this time, all participants are on a listen-only mode. There will be a question-answer session at the end. Please be advised that this call is being recorded at AGO's request. I would like to turn the call over to Chris Taylor, Vice President of Investor Relations and Corporate Communications for AGOs.

Thank you, Operator. Good morning, everyone, and welcome to AGIOS conference call and webcast to discuss our first quarter 2025 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, we'll hear from 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. Today, we are fortunate to have multiple late-stage programs nearing critical readouts or potential commercialization. and have a very strong balance sheet. We are maintaining our focus on executing against the objectives we've laid out, including the ongoing regulatory reviews of our thalassemia program, for which we have seen continued and consistent FDA engagement. And with approximately $1.4 billion of cash on hand and a disciplined approach to capital allocation, We believe we will have financial independence to fund the company through new approvals and product launches while advancing our pipeline, all of which is especially notable in today's market environment. Our lead product, Pyrakyne, or Mitipivet, 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 have the exciting prospect of two additional commercial launches to support our potential multi-billion dollar opportunity for pyrokine. We are planning for a potential approval and launch in thalassemia in the U.S. in September of this year, followed by sickle cell disease in 2026. Beyond pyrokine, our early and mid-stage pipeline is robust and poised for clinical advancement, offering a strong foundation for innovation and long-term growth. And finally, supporting it all is our highly experienced team and the strong balance sheet I noted earlier. We look at 2025 as a breakout year for Agios as we focus on three key priorities. First, maximizing the potential of the Piratine franchise, including the pursuit of new indications. Second, advancing and diversifying our key pipeline programs. And third, strategically focusing our capital deployment to sustain and drive our growth. We have begun the year with a strong start executing towards important milestones. Earlier this year, We announced positive top-line results from the ACTIVATE Kids Phase III trial of midiPIVAT in pediatric patients with PK deficiency who are not regularly transfused. We also anticipate some exciting developments for our mid- and early-stage pipeline programs. For TevaPIVAT, our novel PK activator, we expect to complete enrollment in the ongoing Phase IIb study in low-risk MDS by year-end and to initiate enrollment in 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 TMPR6 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, now only four months away, 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 holds significant value for shareholders and have transformative potential for patients. Before I turn it over to Sarah, I'd like to formally welcome Krishnan Viswanathan, who joined us in March as Chief Corporate Development and Strategy Officer, having previously served as the President and Chief Operating Officer of Bee Biopharma and in various senior roles at both Bristol-Myers Squibb and Celgene. His diverse experience and strategic vision will be instrumental in maximizing the potential of our current assets while also exploring potential expansion opportunities. With that, let me now turn it over to Sarah.