Agios Reports Strong Q4 and Full Year 2024 Results, Driven by PYRUKYND® Growth and Pipeline Advancements

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation therapies for rare diseases, reported its fourth quarter and full year 2024 financial results and recent business highlights on February 13, 2025. The company's strong performance was driven by the growth of its PYRUKYND® franchise and advancements in its pipeline.

Financial Highlights

* PYRUKYND® net revenue: $10.7 million in Q4, a 20% increase from Q3, primarily driven by year-end stocking and adjustments to certain revenue reserves.
* Total unique patients completing prescription enrollment forms: 223, representing a 6% increase over Q3.
* Patients on PYRUKYND therapy: 130, inclusive of new prescriptions and continued therapy.



Business Highlights

* Filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in the U.S., European Union, Kingdom of Saudi Arabia, and United Arab Emirates.
* Completed enrollment for the Phase 3 RISE UP study of mitapivat in sickle cell disease, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026.
* Received orphan drug designation for tebapivat (AG-946) for the treatment of lower-risk myelodysplastic syndromes (LR-MDS) from the U.S. Food and Drug Administration (FDA).
* Presented positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused.

Key Upcoming Milestones & Priorities

* Receive FDA regulatory approval for mitapivat in thalassemia by September 7, 2025.
* Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025.
* Advance and diversify key pipeline programs, including the Phase 2b study of tebapivat in LR-MDS.
* Strategically deploy capital to drive long-term growth and maximize the potential of PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively.

Agios' strong financial performance and pipeline advancements position the company well for a breakout year in 2025. With a robust balance sheet and a highly experienced team, Agios is focused on maximizing the potential of its PYRUKYND launches and advancing its key pipeline programs to bring significant value to shareholders, healthcare professionals, and patients.