Agios Pharmaceuticals, Inc.

Good morning and welcome to AGO's Pharmaceuticals Third Quarter 2025 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 the call over to Morgan Sanford, Head of Investor Relations at AGO. Please go ahead, ma'am.

Thank you, Operator. Good morning, everyone. Thank you for joining us to discuss Agios Pharmaceuticals' third quarter 2025 financial results and business highlights. You can access the slides for today's call by going to the Investors section of our website, agios.com. Next slide, please. Please note we'll be making certain forward-looking statements today. Actual events and results could differ materially from those expressed or implied by any forward-looking statements because 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. Our third quarter earnings call agenda is shown on the next slide. Joining me on today's call are Brian Goss, Chief Executive Officer, Cecilia Jones, Chief Financial Officer, Sveta Milanova, Chief Commercial Officer, and Dr. Sarah Huynh, Chief Medical Officer and Head of Research and Development. Following prepared remarks, we will open the call for questions. With that, please move to the next slide, and I am pleased to turn the call over to Brian.

Thanks, Morgan. Good morning, everyone, and thank you for joining us on today's call to discuss our third quarter highlights, beginning on the next slide. With steady progress and a focused strategy, we have a clear path to unlock long-term shareholder value. First, we have multiple high-value catalysts in the coming months that position pyrokine, our foundational TK activator, to achieve its multibillion-dollar potential across TK deficiency, thalassemia, and sickle cell disease. Following the FDA's recent request for a REMS program, our PDUFA date for pyrokine thalassemia supplemental NDA has been extended to December 7th. We are actively engaged with the FDA and are leveraging this additional time to strengthen our engagement with the thalassemia community and further refine our launch planning. Additionally, we look forward to sharing top-line results from the Rise Up Phase 3 trial of pyrokine and sickle cell disease by year end. Meanwhile, we continue to advance our early and mid-stage pipeline, which includes our other PK activator, Tebapivet, for lower-risk myelodysplastic syndromes, or MDS, and sickle cell disease, AG181 for phenylketonuria, and AG236 for polycythemia vera. Importantly, our strong balance sheet with approximately $1.3 billion in cash and investments positions us to invest in a disciplined manner to both support our potential U.S. launches and advance our rare disease pipeline. Our third quarter highlights are summarized on the next slide. In the third quarter, we reported $12.9 million in net revenue, underscoring the strong value proposition of pyrokine. In August, we announced approval for pyrokine in adults with thalassemia in Saudi Arabia, our first global regulatory approval for this indication. And earlier this month, we also received a positive CHMP opinion recommending pyrokine for marketing authorization in Europe for the treatment of adults with thalassemia. And lastly, we achieved a key R&D priority by completing enrollment in the Phase IIb trial of Tevapivet in lower-risk MDS, and we anticipate top-line data early next year. With continued momentum across our commercial portfolio and pipeline, our team has demonstrated strong execution and agility keeping us firmly focused on our mission to deliver transformative medicines for patients. That agility is a competitive advantage as we work to transform the treatment landscape for thalassemia and sickle cell disease. Feedback from these communities through our recent global engagements reinforces the critical need for treatment innovation. Please move to the next slide and I'll turn the call over to Cecilia to provide commentary on our third quarter performance and four-year outlook. Cecilia?