From GlobeNewswire.: 2025-02-13 06:31:00

Agios Pharmaceuticals filed for regulatory approval of Mitapivat (PYRUKYND®) in the U.S., European Union, Saudi Arabia, and UAE for thalassemia treatment, with a PDUFA goal date of September 7, 2025. Enrollment for the Phase 3 RISE UP study of Mitapivat in Sickle Cell Disease has been completed, with topline results expected in late 2025. The early- and mid-stage pipeline at Agios presents a strong foundation for innovation and growth. PYRUKYND generated $10.7 million in net revenue in Q4, with $1.5 billion in cash, cash equivalents, and marketable securities as of December 31, 2024.

Agios Pharmaceuticals had a transformative year in 2024, achieving key milestones like filing for regulatory approval for Mitapivat in thalassemia and completing enrollment in the Phase 3 RISE UP study for sickle cell disease. With a strong balance sheet and experienced team, Agios aims to maximize PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively, drive long-term growth, and deliver value for shareholders, healthcare professionals, and patients.

In Q4 2024, PYRUKYND generated $10.7 million in net revenue, with 223 unique patients completing enrollment forms and 130 patients on PYRUKYND therapy. Positive results from the ENERGIZE-T Phase 3 trial for thalassemia led to regulatory applications filed in the U.S., EU, Saudi Arabia, and UAE. The Phase 3 RISE UP study for sickle cell disease has enrolled over 200 patients worldwide. Positive topline results from the ACTIVATE-Kids Phase 3 study of Mitapivat in children with PK deficiency were reported. An orphan drug designation was granted to tebapivat for the treatment of MDS, and 16 abstracts highlighting new data on Mitapivat and tebapivat were presented at ASH 2024. David Schenkein, M.D., will step down from Agios’ Board of Directors but continue as a strategic advisor. Agios plans to achieve key milestones in 2025, including FDA decision on PYRUKYND for thalassemia and Phase 3 results for sickle cell disease. Financially, fourth quarter 2024 saw a net product revenue increase to $10.7 million. R&D expenses were $82.8 million, with a net loss of $96.5 million. Cash, cash equivalents, and securities were $1.5 billion, supporting future launches and pipeline expansion. Agios will host a conference call to discuss these results. Agios Pharmaceuticals announced potential benefits of PYRUKYND® (mitapivat), tebapivat (AG-946), AG-236, and AG-181. Plans include drug development and strategic vision for 2025. Risks and uncertainties may impact stock price appreciation and business operations. Consolidated balance sheet data for December 31, 2024, shows cash, cash equivalents, and marketable securities totaling $1,532,031. Revenue for 2024 was $36,498 with a net loss of $425,737. Contact Agios Pharmaceuticals for more information.



Read more at GlobeNewswire.:: Agios Reports Fourth Quarter and Full Year 2024 Financial