Agios Pharmaceuticals, Inc.

Good morning and welcome to ADIOS's first quarter 2023 conference call. At this time, participants are in a listen-only mode. There will be a question and answer session at the end. Please be advised this call is being recorded at ADIOS's request. I would now like to turn the call over to ADIOS's Chief Financial Officer, Cecilia Jones. Please go ahead.

Thank you, Operator. Good morning, everyone, and welcome to AGIOS first quarter 2023 conference call. You can access slides for today's call by going to the investor section of our website, AGIOS.com. On today's call, I am joined by our Chief Executive Officer, Brian Goff, Dr. Sarah Humes, our Chief Medical Officer and Head of Research and Development, and Sveta Milanova, our Chief Commercial 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. With that, I will turn the call over to Brian.

Thanks, Cecilia. Good morning, everyone, and thank you for joining us. Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. We continue to generate consistent and compelling data with our PK activators across multiple disease areas, highlighting the potential of this differentiated mechanism of action to transform patient function quality of life, and long-term outcomes in PK deficiency, thalassemia, sickle cell disease, and lower-risk MBS. Driven by the enthusiasm of our clinical investigators and the operational excellence of our research and development team, I'm pleased to report that we have closed screening in our Phase III studies in thalassemia, which together encompass the entire thalassemia population, and expect to complete enrollment of these studies later this month. On behalf of the AGIOS team, I'd like to thank the patients, caregivers, and physicians for their continued participation in these trials. As Sarah will describe in more detail, the rapid enrollment of these studies highlights the significant unmet need in these areas and bolsters our conviction in our broader clinical development strategy. In this context, we continue to advance our robust clinical stage pipeline and are on track to announce the data readout from the Phase 2 portion of the operationally seamless Phase 2-3 rise-up study of pyrokine in sickle cell disease and the go-no-go decision of Phase 3 in the middle of this year. Echoing the positive reception of both pyrokine and AG946 from investigators at our clinical trial sites, we continue to receive positive feedback from both patients and clinicians on the impact that pyrokine is having on individuals living with PK deficiency in the real world. We're encouraged to see that the efficacy of pyrokine observed in the clinical trial experience is translating to persistency on therapy in the real world, and we believe this has positive implications for the potential long-term impact of PK activation on how patients feel and function in not just PK deficiency, but also thalassemia, sickle cell disease, and lower risk MDS. Sveta will provide a detailed update on our commercial performance in just a few minutes. As you'll hear from Cecilia, we ended the first quarter of 2023 in an enviable cash position with approximately $1 billion on the balance sheet. As a reminder, as part of the divestiture of our oncology business to survey in April of 2021, we retain rights to a potential milestone upon FDA approval of voracidinib and royalties on potential U.S. net sales. We were therefore pleased to see that CERBIA's Phase III trial of voracitinib in patients with residual or recurrent IDH mutant low-grade glioma met both its primary endpoint and key secondary endpoints. These data underscore AGIOSA's strong track record in the discovery and development of therapeutics for disease areas with high unmet need which to date spans five approved indications across three separate products. In addition to the upcoming phase two data readout of pyrokine in sickle cell disease, we're expecting a number of additional milestones by the end of the year, including completing enrollment of the phase three studies of pyrokine in thalassemia, enrolling more than half of the patients in the Phase III Activate Kids and Activate Kids T studies of pyrokine and pediatric PK deficiency, completing enrollment of the Phase IIa study of AG946 in lower-risk MDS, and filing the IND for our PAH stabilizer for the treatment of PKU. Finally, looking forward to what we expect to be a catalyst-rich next few years We anticipate readouts from the Phase III studies of pyrokine and thalassemia in 2024 and readouts from the Phase III studies of pyrokine and sickle cell disease and pediatric PK deficiency in 2025. With that, I will now turn the call over to Sarah. Sarah?