Company: AGIO
Filing Date: 2025-04-25
Form Type: DEF 14A
Source: 0001193125-25-096719
Chunk: 56

Company: AGIOS PHARMACEUTICALS, INC.
Filing Date: 2025-04-25
Form: DEF 14A
Chunk 56
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 reduction response. The study also demonstrated a statistically significant reduction in additional measures of transfusion reduction response compared to placebo as assessed by the three key secondary endpoints. In addition, a higher proportion of patients in the PYRUKYND arm compared to the placebo arm achieved the secondary endpoint of transfusion independence. Based on the results of the ENERGIZE and ENERGIZE-T trials, in December 2024 we announced that we filed regulatory applications for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the FDA, the European Medicines Agency, or EMA, and Kingdom of Saudi Arabia and United Arab Emirates health authorities. In January 2025, the FDA accepted our supplemental new drug application with standard review and granted a Prescription Drug User Fee Act goal date of September 7, 2025.

We also continued to advance PYRUKYND in SCD and PK deficiency. We completed enrollment of the phase 3 portion of RISE UP, a phase 2/3 study evaluating the efficacy and safety of PYRUKYND in SCD patients 16 years of age or older. The European Commission also adopted a positive decision for the designation of mitapivat as an orphan medicinal product for the treatment of SCD. With respect to PK deficiency, we announced topline data from ACTIVATE-kidsT and completed enrollment for ACTIVATE-kids, which are double-blind phase 3 studies evaluating the efficacy and safety of PYRUKYND as a potential treatment for PK deficiency in regularly transfused and not regularly transfused patients between one and 18 years old, respectively. During 2024, we also made further progress commercializing PYRUKYNDfor the treatment of hemolytic anemia in adults with PK deficiency, recording $36.5 million in total revenue for the year ended December 31, 2024, compared to $26.8 million for the year ended December 31, 2023.

In addition to the above achievements, we continued to advance our pipeline across our portfolio. With respect to tebapivat, our novel PK activator, we initiated patient enrollment in our phase 2b study in patients with lower-risk myelodysplastic syndromes, or LR MDS. Tebapivat was also granted orphan drug designation for the treatment of MDS by the FDA. In addition, we dosed the first participants in our phase