Business
Agios Reports Fourth Quarter and Full Year 2022 Financial Results
– Completed Enrollment in Phase 2 RISE UP Study of PYRUKYND® (mitapivat) in Sickle Cell Disease – – On Track to Complete Enrollment in Phase 3 ENERGIZE and
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Completed Enrollment in Phase 2 RISE UP Study of PYRUKYND® (mitapivat) in Sickle Cell Disease – – On Track to Complete Enrollment in Phase 3 ENERGIZE and ENERGIZE-T Studies of PYRUKYND® in Thalassemia by Mid-2023 – – U.S. PYRUKYND® Launch Provides Capability Building Platform to Support Expected Future Product Growth and Expansion; Net Revenue of $4.3 Million in Q4 – – $1.1 Billion of Cash, Cash Equivalents and Marketable Securities as of Dec. 31, 2022 – CAMBRIDGE, Mass., Feb. 23, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended Dec. 31, 2022. “Over the past year, Agios has made significant progress toward our vision of transforming the lives of patients with rare diseases as we build a hematology franchise focused on diseases that share a common underlying pathophysiology, limited treatment options and profound unmet need,” said Brian Goff, chief executive officer at Agios. “In 2022, we received regulatory approvals in the U.S., EU and Great Britain for PYRUKYND® as the first and only disease-modifying treatment for adults with pyruvate kinase (PK) deficiency. We achieved our ambitious enrollment targets for our pivotal trials in thalassemia and sickle cell disease. We strengthened our company leadership with the appointments of new management team and Board members with deep expertise in rare diseases and global commercial strategy. We are poised for significant near- and long-term growth and look forward to a productive 2023, anticipating the readout of our Phase 2 sickle cell disease study and the completion of enrollment in our Phase 3 thalassemia studies, driving toward two additional PYRUKYND® indications by 2026.” Fourth Quarter 2022 & Recent Highlights PYRUKYND® U.S. Launch: Continued to execute launch, generating $4.3 million in U.S. net revenue for the fourth quarter of 2022, the third full quarter following FDA approval. A total of 105 unique patients have completed prescription enrollment forms, representing an increase of 25 percent over the third quarter. A total of 78 patients are on PYRUKYND® therapy, representing a 39 percent increase over the third quarter.PYRUKYND® Global Approvals: Received marketing authoriza...