Agios Pharmaceuticals Inc.’s stock (AGIO, +2.51%) experienced a significant surge of 19.6% early Wednesday following the announcement of positive results from a Phase 3 trial for a treatment targeting a rare blood disorder.
Promising Findings from the Energize Trial
The trial, known as Energize, focused on evaluating the effectiveness of Agios Pharmaceuticals Inc.’s mitapivat therapy in patients with non-transfusion-dependent (NTD) alpha- or beta-thalassemia. Encouragingly, the trial successfully met its primary endpoint of hemoglobin response.
Secondary Endpoints Highlight Potential Benefits
In addition to meeting its primary endpoint, the Energize trial also achieved positive outcomes in two secondary endpoints. It demonstrated significant improvements in both the change from baseline in FACIT-Fatigue Score and hemoglobin concentration.
Expert Opinions Support the Potential of Mitapivat
Dr. Ali Taher, a respected professor of medicine specializing in hematology and oncology, and the director of the Naef K. Basile Cancer Institute at the American University of Beirut Medical Center, expressed enthusiasm regarding the trial results. Dr. Taher stated that the findings reinforce the potential of mitapivat to become the first oral therapy for all NTD thalassemia patients, including those with alpha- or beta-thalassemia.
Future Prospects and Regulatory Approval
With these promising results, Agios Pharmaceuticals Inc. plans to seek regulatory approval for mitapivat by the end of 2024. This further highlights the potential of this therapy to make a significant impact in treating NTD thalassemia.
Market Performance
Over the last 12 months, Agios Pharmaceuticals Inc.’s stock has experienced a decline of 17%. In comparison, the S&P 500 has shown a growth of 24% during the same period.
S&P 500 symbol: SPX, -0.57%