Agios Pharmaceuticals(AGIO) - 2025 Q2 - Quarterly Results

Business and Operational Highlights Agios reported strong PYRUKYND® commercial performance, advanced its R&D pipeline, and prepared for potential U.S. approval for thalassemia - The company is focused on the upcoming PDUFA goal date of September 7, 2025, for the potential U.S. approval of PYRUKYND for thalassemia[3] - Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease are expected by the end of the year, with a potential U.S. commercial launch in 2026[3][5][10] - Entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND in the European Economic Area, the UK, and Switzerland[6] Commercial Performance – PYRUKYND® (mitapivat) PYRUKYND® net revenue reached $12.5 million in Q2 2025, driven by steady growth in patient enrollment and therapy PYRUKYND® Q2 2025 Performance | Metric | Q2 2025 | Q2 2024 | QoQ Growth (vs Q1 2025) | | :--- | :--- | :--- | :--- | | Net Revenue | $12.5 million | $8.6 million | N/A | | Unique Patient Enrollments | 248 | N/A | 6% | | Patients on Therapy | 142 | N/A | 4% | R&D Highlights R&D pipeline advanced with PYRUKYND's sNDA for thalassemia, progress in sickle cell and LR-MDS trials, and AG-236 IND clearance - PYRUKYND (mitapivat): - Thalassemia: sNDA under active FDA review with a PDUFA goal date of September 7, 2025 - Sickle Cell Disease: Topline Phase 3 (RISE UP) results on track for year-end 2025[10] - Tebapivat: - Sickle Cell Disease: Dosed the first patient in a Phase 2 trial - Lower-risk Myelodysplastic Syndromes (LR-MDS): Phase 2b trial enrollment is progressing, with completion targeted by the end of 2025[8][10] - Early Pipeline: - Received Investigational New Drug (IND) clearance for AG-236, an siRNA targeting TMPRSS6 for treating polycythemia vera (PV)[10] Second Quarter 2025 Financial Results Agios reported a net loss of $112.0 million in Q2 2025, driven by increased R&D and SG&A expenses, with $1.3 billion in cash Q2 2025 Key Financial Metrics (in millions, vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Net Product Revenue | $12.5 million | $8.6 million | | R&D Expenses | $91.9 million | $77.4 million | | SG&A Expenses | $45.9 million | $35.5 million | | Net Loss | $112.0 million | $96.1 million | - The increase in R&D expenses was primarily due to a $10.0 million regulatory milestone payment to Alnylam for AG-236[13] - The increase in SG&A expenses was mainly attributable to increased commercial activities in preparation for the potential approval of PYRUKYND in thalassemia[13] - The company ended the quarter with $1.3 billion in cash, cash equivalents, and marketable securities, expected to fund operations, potential launches, and pipeline expansion[5][13] Financial Statements Consolidated financial statements show $1.47 billion in total assets and a net loss of $112.0 million for Q2 2025, or ($1.93) per share Consolidated Balance Sheet Data As of June 30, 2025, Agios reported $1.34 billion in cash, $1.47 billion in total assets, and $1.37 billion in stockholders' equity Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $1,339,404 | $1,532,031 | | Total assets | $1,471,237 | $1,663,199 | | Stockholders' equity | $1,369,555 | $1,540,956 | Consolidated Statements of Operations Data For Q2 2025, net product revenue was $12.5 million, with loss from operations at $127.1 million and a net loss of $112.0 million, or ($1.93) per share Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Product revenue, net | $12,455 | $8,615 | | Total operating expenses | $139,511 | $114,432 | | Loss from operations | $(127,056) | $(105,817) | | Net loss | $(112,020) | $(96,118) | | Net loss per share | $(1.93) | $(1.69) |