BofA raised the firm’s price target on Agios Pharmaceuticals (AGIO) to $34 from $32 and keeps a Buy rating on the shares following FDA approval for mitapivat to make it the only drug approved in both transfusion-dependent and non-transfusion-dependent alpha- or beta- thalassemia. The REMS and black box for hepatocellular injury were in line with the firm’s expectations and BofA doesn’t expect this to impact uptake, the analyst tells investors. Claim 70% Off TipRanks This Holiday Season Published first on ...

Shares of Agios Pharmaceuticals surged nearly 15% in premarket trading on Wednesday after the US Food and Drug Administration approved an expanded use of its drug mitapivat, strengthening investor opt... ...

CAMBRIDGE, Mass., Dec. 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has not yet issued a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia and tha ...

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]

Core Viewpoint - Agios Pharmaceuticals Inc. experienced a significant stock decline following the release of Phase 3 trial results for mitapivat in treating sickle cell disease, despite meeting its primary endpoint of hemoglobin response [1][10]. Trial Results - The RISE UP Phase 3 trial met its primary endpoint, showing a statistically significant improvement in hemoglobin response with 40.6% of patients in the mitapivat arm achieving this response compared to 2.9% in the placebo arm [2][3]. - Mitapivat also demonstrated a reduction in the annualized rate of sickle cell pain crises (SCPCs), with rates of 2.62 in the mitapivat arm versus 3.05 in the placebo arm, although this did not achieve statistical significance [3][4]. - Statistically significant improvements were observed in two key secondary endpoints: average change in hemoglobin concentration and levels of indirect bilirubin from baseline [4][6]. Safety and Efficacy - The safety profile of mitapivat in this trial was consistent with previous studies, with an average change in hemoglobin concentration of 7.69 g/L in the mitapivat arm compared to 0.26 g/L in the placebo arm, indicating a statistically significant improvement [6]. - The average change in indirect bilirubin was -16.03 µmol/L in the mitapivat arm versus 0.88 µmol/L in the placebo arm, also showing a statistically significant improvement [7]. Future Plans - Agios plans to submit a marketing application for mitapivat in the U.S. for sickle cell disease after a pre-supplemental New Drug Application meeting with the FDA in early 2026 [8]. - The company is also focused on other commercial and pipeline milestones, including the potential U.S. approval of Pyrukynd (mitapivat) for thalassemia, expected in December 2025 [8][9]. Stock Performance - Following the trial results, Agios Pharmaceuticals' stock fell by 49.00%, trading at $23.20, near its 52-week low of $23.41 [10].

Summary of Agios Pharmaceuticals Update / Briefing (November 19, 2025) Company Overview - **Company**: Agios Pharmaceuticals (NasdaqGS:AGIO) - **Focus**: Development of innovative medicines for rare diseases, particularly sickle cell disease Industry Context - **Disease**: Sickle cell disease, characterized by complex and severe symptoms, with a median life expectancy in the U.S. of late 30s - **Current Treatment Landscape**: Limited treatment options available, with a strong need for new therapies Key Points from the Call Clinical Trial Results - **RISE UP Phase III Trial**: - **Primary Endpoint**: Mitapivat achieved statistical significance in hemoglobin response - **Secondary Endpoint**: A trend towards a reduction in the annualized rate of sickle cell pain crises (14% reduction, p-value 0.12) was observed [2][21] - **Patient Population**: 40.6% of patients in the mitapivat arm achieved a significant hemoglobin response (defined as at least a 1 gram per deciliter increase) [20][33] - **Efficacy**: - Average hemoglobin concentration increased by 0.7 grams per deciliter compared to placebo [22] - Indirect bilirubin levels decreased significantly, indicating reduced hemolysis [24] - Fatigue scores improved, although not statistically significant [25][38] Mechanism of Action - **Mitapivat**: An oral allosteric activator of red blood cell pyruvate kinase, which enhances ATP levels and improves red blood cell membrane integrity, potentially reducing hemolysis and vasoocclusion [15][16] Safety Profile - **Adverse Events**: Generally well-balanced between mitapivat and placebo arms, with no significant drug-induced liver injury observed [29][32] - **Serious Adverse Events**: More reported in the placebo arm than in the mitapivat arm [29] Market Potential - **Unmet Need**: Significant unmet need for effective treatments in sickle cell disease, with acute care costs in the U.S. approximating $2 billion per year [38] - **Commercial Strategy**: Plans to submit a marketing application for mitapivat in the U.S. after a pre-SNDA meeting with the FDA in Q1 of 2026 [34][41] Future Directions - **Pipeline Development**: Ongoing phase II trials for tebipivat (a more potent PK activator) and other programs targeting different conditions [41] - **Financial Discipline**: Commitment to reducing operating expenses to maximize shareholder value [42] Additional Insights - **Patient Quality of Life**: Improvement in fatigue is a critical outcome, as fatigue is a major symptom affecting patients' daily lives [38] - **Clinical Context**: The results indicate that while not all patients respond, a significant portion shows clinically meaningful benefits, reinforcing the need for diverse treatment options [35][39] Conclusion - The RISE UP trial results for mitapivat demonstrate promising efficacy and safety in treating sickle cell disease, addressing a critical unmet need in the market. The company is poised to advance its regulatory strategy and continue developing its pipeline to enhance treatment options for patients.

RISE UP Phase 3 Trial Results - Mitapivat met the primary endpoint with a 40.6% hemoglobin response rate, defined as ≥1.0 g/dL increase in average Hb from Week 24 through Week 52 compared to baseline, significantly higher than the 2.9% in the placebo arm[38] - There was a trend favoring mitapivat with a 14% reduction in the annualized rate of sickle cell pain crises (SCPCs) compared to placebo, although this did not reach statistical significance[39] - Mitapivat showed a statistically significant improvement in average change from baseline in hemoglobin concentration, with a 7.43 g/L difference compared to placebo[41] - Mitapivat significantly reduced the average change from baseline in indirect bilirubin compared to placebo, with a difference of -16.91 µmol/L[46] - The trial showed a trend in favor of mitapivat with a 14% reduction in the annualized rate of hospitalizations for SCPC compared to placebo[50] - Percent reticulocytes substantially decreased from baseline in the mitapivat arm vs placebo[52] Safety and Discontinuation - The safety profile observed in the RISE UP Phase 3 trial was consistent with previous mitapivat sickle cell disease trials, with no new safety signals identified[57] - The discontinuation rate in the double-blind treatment period was low, with 13% in the mitapivat arm and 18.8% in the placebo arm[34] Next Steps - The company intends to submit a marketing application for mitapivat in the U S for sickle cell disease after a pre-sNDA meeting with the FDA in Q1 2026[61, 68]

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]