Agios Reports First Quarter 2025 Financial Results and Recent Business Highlights

Core Insights - Agios Pharmaceuticals is progressing with the regulatory approval of PYRUKYND (mitapivat) for thalassemia, with a PDUFA goal date set for September 7, 2025 [1][2] - The Phase 3 RISE UP study for mitapivat in sickle cell disease is on track, with topline results expected by late 2025 and a potential U.S. commercial launch in 2026 [1][2] - The company reported a net revenue of $8.7 million for PYRUKYND in Q1 2025, showing a slight increase from $8.2 million in Q1 2024 [1][3] Financial Performance - PYRUKYND generated net revenue of $8.7 million in Q1 2025, up from $8.2 million in Q1 2024, with 234 unique patients completing prescription enrollment forms, a 5% increase from Q4 2024 [3][5] - R&D expenses for Q1 2025 were $72.7 million, compared to $68.6 million in Q1 2024, primarily due to increased workforce-related expenses and clinical trial costs [6] - SG&A expenses rose to $41.5 million in Q1 2025 from $31.0 million in Q1 2024, attributed to increased commercial activities in preparation for potential PYRUKYND approval [7] Cash Position - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.4 billion, down from $1.5 billion at the end of 2024 [8] - The company anticipates that its financial resources will support the potential launches of PYRUKYND in thalassemia and sickle cell disease, as well as the advancement of existing programs [8] Clinical Development - The FDA accepted the supplemental New Drug Application for PYRUKYND for both non-transfusion-dependent and transfusion-dependent thalassemia patients [3][11] - The Phase 3 RISE UP study for sickle cell disease has completed enrollment with over 200 patients worldwide [3][11] - Positive topline results were reported from the ACTIVATE-Kids Phase 3 study of mitapivat in children with PK deficiency, marking a significant advancement in pediatric treatment [3] Corporate Developments - Krishnan Viswanadhan joined Agios as Chief Corporate Development and Strategy Officer, focusing on corporate strategy and long-term growth initiatives [4]