Core Points - The conference call is focused on the top line results from the pivotal Phase III RISE UP trial of mitapivat for treating sickle cell disease [2] - The call includes key executives from Agios Pharmaceuticals, indicating a comprehensive discussion on the trial results and future implications [4] Company Overview - Agios Pharmaceuticals is actively engaging with investors to discuss significant trial outcomes, showcasing its commitment to transparency and investor relations [2][4] - The company is preparing to share detailed findings from the RISE UP trial, which is crucial for its product pipeline and market strategy [2] Industry Context - The Phase III RISE UP trial represents a critical step in the development of treatments for sickle cell disease, a condition with significant unmet medical needs [2] - The results from this trial could have implications for the broader pharmaceutical industry, particularly in the area of rare diseases and innovative therapies [2]

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) stock plunged on Wednesday after the company released topline results from RISE UP Phase 3 data for mitapivat in sickle cell disease.AGIO is testing key support levels. See what the experts say hereThe trial met its primary endpoint of hemoglobin response, with mitapivat demonstrating a statistically significant improvement compared to placebo.Also Read: FDA Delays Decision On Agios Pharmaceuticals’ Blood Disorder Drug For Expanded UseDataWhile mitapivat also showed ...

Agios Pharmaceuticals (NasdaqGS:AGIO) Update / Briefing November 19, 2025 08:00 AM ET Company ParticipantsEllen Horste - Equity Research AssociateSarah Gheuens - Chief Medical Officer and Head of Research and DevelopmentBrian Goff - CEOMorgan Sanford - Head of Investor RelationsBiree Andemariam - Professor of MedicineConference Call ParticipantsTessa Romero - Senior Analyst of Biotechnology Equity ResearchEmily Bodnar - Biotech Equity Research AnalystEric Smith - Senior Biotechnology AnalystLuca Issi - Seni ...

Forward-looking statements Topline results Phase 3 RISE UP in sickle cell disease November 19, 2025 This presentation and various remarks we make during this presentation contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of mitapivat; Agios' plans for future meetings with, or submissions to, regulators, including the FDA; Agios' plans for the development of mitapivat, t ...

Core Insights - Agios Pharmaceuticals announced topline results from the RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase activator, for patients with sickle cell disease, demonstrating significant improvements in hemoglobin response and hemolysis markers [1][5][6] Trial Results - The trial met its primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving a significant increase in hemoglobin levels compared to 2.9% in the placebo group [12] - Mitapivat showed a statistically significant improvement in average hemoglobin concentration and indirect bilirubin levels, but the reduction in annualized rate of sickle cell pain crises did not achieve statistical significance [2][3][12] - The safety profile of mitapivat was consistent with previous trials, with a similar proportion of adverse events reported in both mitapivat and placebo groups [19] Patient Outcomes - Patients who achieved hemoglobin response experienced clinically meaningful benefits in the annualized rate of sickle cell pain crises and hospitalizations, as well as improvements in fatigue scores [4][12][13] - The average change in PROMIS Fatigue score was -5.19 for hemoglobin responders, indicating a clinically meaningful improvement [13] Regulatory and Future Plans - Agios plans to engage with the FDA to discuss the findings and intends to submit a marketing application for mitapivat for sickle cell disease in early 2026 [15][16] - The company is also focused on the potential U.S. approval of PYRUKYND for thalassemia, anticipated in December 2025, and will take steps to reduce operating expenses [16] About Sickle Cell Disease - Sickle cell disease is a rare inherited blood disorder characterized by abnormal hemoglobin production, leading to severe complications and increased healthcare needs [18]

Core Insights - Agios Pharmaceuticals is presenting new data on its pyruvate kinase (PK) activators, mitapivat and tebapivat, at the upcoming European Hematology Association (EHA) Congress in Milan, Italy, from June 12-15, 2025 [1][2] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and PK activation, focusing on therapies for rare diseases [1][17] - The company has developed PYRUKYND (mitapivat), a first-in-class pyruvate kinase activator for treating hemolytic anemia in adults with PK deficiency [9][17] Clinical Data Presentation - A total of 14 presentations and publications will be shared at EHA 2025, highlighting the efficacy and safety of PK activation in treating rare blood disorders [3][2] - Key presentations include: - Results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children with PK deficiency, showing a clinically meaningful reduction in transfusion burden [6] - Long-term data from the ESTIMATE Phase 2 trial of mitapivat in sickle cell disease, demonstrating sustained efficacy and tolerability over three years [6] - Preclinical data on tebapivat's potential in reducing red blood cell sickling in sickle cell disease patients [6] Research Focus Areas - The presentations will cover serious conditions with limited treatment options, including sickle cell disease, thalassemia, PK deficiency, and myelodysplastic syndromes [2][6] - Ongoing investigations include the expression patterns of PKM2 in patients with myelodysplastic syndromes, supporting the potential of tebapivat in lower-risk MDS [6] Collaboration and Community Engagement - Agios aims to strengthen its collaboration with the global hematology community through these presentations at EHA [2]