Q4 and FY 2025 Financial Results and Business Highlights Conference call for investors and analysts 12 February 2026 4 2026 strategic priorities – driving long-term value creation | Execute high-impact launch for AQVESME | | --- | | (mitapivat) in thalassemia | | Potential to expand PK activation franchise into | | sickle cell disease and LR-MDS | | Unlock future value in hematology and other rare | | disease by advancing early-stage pipeline | | Ensure long-term sustainability through disciplined | | capit ...

Business Overview - Agios is positioned for growth and aims for profitability with its existing commercial portfolio[3, 4] - The company has a strong foundation in hematology and aspires to become a sustainable rare disease company[5] - Agios estimates a total global market size of over $10 billion by 2030 for its current pipeline indications[11] Product Pipeline and Approvals - AQVESME (mitapivat) is now approved in the U S for anemia in adults with alpha- or beta-thalassemia, addressing approximately 4,000 addressable patients at launch[7, 17, 23] - AQVESME has a potential to deliver $1 billion in global peak-year-sales across PKD and thalassemia indications[24] - Pyrukynd is approved in the U S , EU, and UK for Pyruvate Kinase Deficiency[7] Clinical Development - In Sickle Cell Disease, 40.6% of patients achieved Hemoglobin response (≥1 g/dL) in the mitapivat trial[37] - Phase 1 tebapivat data demonstrates a 1.2 g/dL increase (2mg) and 1.9 g/dL increase (5mg) in mean change from baseline in Hb across dosing cohorts at Day 28 in SCD patients[44] - In LR-MDS, 40% of low transfusion burden cohort achieved transfusion independence with tebapivat[51]

Core Viewpoint - Agios Pharmaceuticals has received FDA approval for mitapivat, a treatment for anemia in adults with alpha or beta thalassemia, which will be marketed as AQVESME in the United States [2]. Group 1: FDA Approval - The FDA has approved mitapivat for treating anemia in adults with alpha or beta thalassemia [2]. - The drug will be marketed under the brand name AQVESME in the United States [2]. Group 2: Investor Relations - Morgan Sanford, Vice President of Investor Relations, is leading the conference call to discuss the approval and its implications [2]. - The company has provided access to presentation slides on its website for investors [2].

Core Viewpoint - Agios Pharmaceuticals' shares increased by over 18% following FDA approval for AQVESME (mitapivat) to treat anemia in thalassemia patients, with management outlining positive marketing and pricing strategies [1][5]. FDA Approval - Mitapivat is designed to address thalassemia, an inherited blood disorder that results in insufficient hemoglobin production, leading to fatigue and increased morbidity. The drug enhances the strength and longevity of red blood cells, potentially reducing fatigue and transfusion needs for patients [2]. Marketing Plan - The management indicated an "addressable launch population" of 4,000 patients in the U.S. for thalassemia, with a proposed annual treatment cost of $425,000 [3]. Sales Potential - Mitapivat is already FDA approved for Pyruvate Kinase Deficiency (PKD), and management anticipates the drug could achieve $1 billion in global peak-year sales across both PKD and thalassemia indications. This potential may increase with demographic trends bringing more thalassemia patients to the U.S. market [4]. Company Outlook - The recent approval marks a turnaround for the company after a previous setback with mitapivat in a sickle cell disease trial, positioning the company for rapid sales growth [5].