Agios Pharmaceuticals(US:AGIO)2025-05-01 12:08Business Overview - Agios views PYRUKYND® as having a multi-billion dollar market opportunity [9] - Agios aims to maximize the potential of the PYRUKYND® franchise and progress key pipeline programs in 2025 [11] - Agios anticipates several clinical and regulatory catalysts in 2025, including a potential FDA approval for PYRUKYND® in thalassemia with a PDUFA goal date of September 7, 2025 [13] Clinical Development - Approximately 67% of thalassemia patients are non-transfusion dependent, representing about 4,000 adults in the U S [19] - Approximately 33% of thalassemia patients are transfusion dependent, representing about 2,000 adults in the U S [19] - Agios's PYRUKYND® has the potential to be the first and only approved therapy for all subtypes of thalassemia [23] - The Phase 3 RISE UP study of PYRUKYND® in sickle cell disease completed enrollment in October 2024, with topline data expected in late 2025 [31] - Tebapivat's Phase 2b study in lower-risk MDS expects patient enrollment completion in late 2025 [34, 41] Commercial Performance & Financials - PYRUKYND® net revenue was $8 7 million for Q1 2025, compared to $8 2 million in Q1 2024 [68] - Agios had $1 4 billion in cash, cash equivalents, and marketable securities as of March 31, 2025 [68, 74]