CAMBRIDGE, Mass., June 06, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that the time of the company's presentation at the Goldman Sachs 45th Annual Global Healthcare Conference has been updated to Monday, June 10, 2024 at 8:40 a.m. ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.c ...

文章核心观点 - Agios Pharmaceuticals (AGIO) 股价周一上涨超过20%，原因是其唯一上市药物Pyrukynd (mitapivat) 在ENERGIZE-T III期研究中达到了主要和关键次要终点，该研究评估了其在某些地中海贫血患者中的效果 [1][2][3] 研究细节 - ENERGIZE-T研究共有258名成年输血依赖性α或β地中海贫血患者参与，分为两组：171名患者接受100mg口服剂量的Pyrukynd，87名患者接受安慰剂，治疗期为48周 [2] - 研究数据显示，接受Pyrukynd治疗的患者在输血负担方面显著减少，主要终点达到：30.4%的患者达到输血减少响应，而安慰剂组为12.6% [2] - 研究还显示，在三个关键次要终点上，Pyrukynd组的患者也表现出显著减少，其中10%的患者达到次要终点，即至少连续八周无输血，而安慰剂组为1.1% [3] 市场潜力 - 目前没有批准用于治疗非输血依赖性地中海贫血患者的疗法，Agios估计在美国地中海贫血患者中，超过一半是非输血依赖性患者，Pyrukynd在此领域的批准将满足高度未满足的需求 [4] - ENERGIZE和ENERGIZE-T研究的结果增强了Pyrukynd成为TD和NTD地中海贫血患者首个口服疗法的潜力，Agios计划在2024年底前寻求Pyrukynd在所有地中海贫血亚型中的标签扩展，预计明年在此领域进行商业化 [4] 其他业务动态 - Agios正在评估进行II/III期RISE UP研究，评估mitapivat用于镰状细胞病(SCD)，去年报告的II期部分达到了主要终点，公司已启动III期部分，计划在2026年获得SCD批准 [5] - Agios与Royalty Pharma达成协议，出售vorasidenib潜在净销售额的特许权使用费，Agios将获得9.05亿美元的预付款，Royalty Pharma将获得15%的美国销售额特许权使用费，超过10亿美元后为12% [5][6] - 资金将用于扩展Agios的管线，增加的现金余额应为公司在地中海贫血和SCD领域推出Pyrukynd时提供财务独立性 [7] 股价表现 - 今年以来，Agios的股价已上涨96.8%，而行业平均涨幅为2.9% [5]

– Study Achieved the Primary Endpoint; Mitapivat Demonstrated a Statistically Significant Transfusion Reduction Response Compared to Placebo – – Statistical Significance Achieved for All Key Secondary Endpoints Evaluating Additional Measures of Reduction of Transfusion Burden Compared to Placebo – – ENERGIZE-T is the First Phase 3 Study to Demonstrate Efficacy of an Oral, Disease-Modifying Treatment for Transfusion-Dependent Alpha- and Beta-Thalassemia – – As Part of Global Submission Strategy, U.S. Market ...

– Study Achieved the Primary Endpoint; Mitapivat Demonstrated a Statistically Significant Transfusion Reduction Response Compared to Placebo – – Statistical Significance Achieved for All Key Secondary Endpoints Evaluating Additional Measures of Reduction of Transfusion Burden Compared to Placebo – – ENERGIZE-T is the First Phase 3 Study to Demonstrate Efficacy of an Oral, Disease-Modifying Treatment for Transfusion-Dependent Alpha- and Beta-Thalassemia – – As Part of Global Submission Strategy, U.S. Market ...

Shares of Agios Pharmaceuticals (AGIO) rose more than 20% on May 28 after it entered into a deal with Royalty Pharma (RPRX) to sell the royalty rights on potential net sales of vorasidenib, a brain cancer therapy.Vorasidenib was a part of Agios’ oncology business that was sold to France-based pharmaceutical company Servier in 2021. Per the sale terms, the company is entitled to receive a 15% royalty on potential net sales of the drug in the United States following a possible FDA approval.Currently, the Serv ...

– Royalty Pharma to Acquire Rights to Agios' 15% Royalty on Potential Vorasidenib U.S. Net Sales for $905 Million Upfront upon FDA Approval of Vorasidenib; Agios to Share in Economics Above Certain Revenue Thresholds – – Agios Retains Rights to $200 Million Milestone Payment from Servier upon FDA Approval of Vorasidenib – – In Total, Agios to Receive $1.1 Billion in Payments upon FDA Approval of Vorasidenib; PDUFA Action Date of August 20, 2024 – CAMBRIDGE, Mass., May 28, 2024 (GLOBE NEWSWIRE) -- Agios Phar ...

Shares of Agios Pharmaceuticals (AGIO) have gained 3.2% over the past four weeks to close the last trading session at $32.69, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $41.33 indicates a potential upside of 26.4%.The mean estimate comprises six short-term price targets with a standard deviation of $8.12. While the lowest estimate of $30 indicates an 8.2% decline from the c ...

Agios Pharmaceuticals (AGIO) closed the last trading session at $32.80, gaining 10.4% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $41.33 indicates a 26% upside potential.The mean estimate comprises six short-term price targets with a standard deviation of $8.12. While the lowest estimate of $30 indicates an 8.5% decline from the current price level, the most optimistic analyst ...

CAMBRIDGE, Mass., May 06, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that the company is scheduled to present at the BofA Securities 2024 Health Care Conference on Thursday, May 16, 2024, at 11:40 a.m. ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. A replay of the webcast w ...

CAMBRIDGE, Mass., May 03, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that the company is scheduled to present at the 2024 RBC Capital Markets Global Healthcare Conference on Tuesday, May 14, 2024, at 8:00 a.m. ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. A replay of the w ...