文章核心观点 - 公司在thalassemia和儿童PK缺乏症领域取得重大进展,分别完成了ENERGIZE-T和ACTIVATE-KidsT III期临床试验 [1][3][6] - 公司与Royalty Pharma签署了9.05亿美元的vorasidenib版权交易协议,预计在FDA批准后将获得11亿美元总收益 [2][6] - 公司PYRUKYND®在Q2实现了8.6百万美元的净销售收入,患者人数持续增加 [4][7] - 公司现金、现金等价物和有价证券余额为6.453亿美元,有望为潜在的PYRUKYND®在地中海贫血和镰状细胞病的上市做好准备 [12] 分组1: 临床试验进展 - 完成了ENERGIZE-T III期临床试验,达到了主要和所有关键次要终点 [1][7] - 在欧洲血液学会2024年会上报告了ENERGIZE III期临床试验的积极结果 [7] - 完成了ACTIVATE-KidsT III期临床试验的患者入组,并公布了积极的初步结果 [3][7] - 计划在2025年公布ACTIVATE-Kids III期临床试验的结果 [7] 分组2: 商业化和财务表现 - PYRUKYND®在Q2的净销售收入为8.6百万美元,较上年同期增长28% [4][7] - 截至2024年6月30日,公司现金、现金等价物和有价证券余额为6.453亿美元 [12] - 公司与NewBridge Pharmaceuticals签署了分销协议,将在海湾合作委员会地区推广PYRUKYND® [7] - 公司预计现金、现金等价物和有价证券将为PYRUKYND®在地中海贫血和镰状细胞病的潜在上市提供资金支持 [12] 分组3: 公司发展战略 - 计划在2024年底前,基于ENERGIZE和ENERGIZE-T研究的结果,向FDA提交PYRUKYND®用于治疗所有类型地中海贫血的补充新药申请 [1][6][7] - 计划在2024年底前完成RISE UP III期临床试验的患者入组,用于评估PYRUKYND®治疗镰状细胞病 [7] - 计划在2024年中期启动AG-946用于治疗低危骨髓增生异常综合征的II期b期临床试验 [7] - 预计Servier的vorasidenib用于治疗IDH突变弥漫性胶质瘤将在2024年8月20日获得FDA批准 [7]

– ACTIVATE-KidsT is Agios’ First Pediatric Data Readout;Safety Results Consistent with Safety Profile for Mitapivat Previously Observed in Adultswith PK Deficiency Who are Regularly Transfused – – Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the Primary Endpoint of Transfusion Reduction Response and for the Secondary Endpoints of Transfusion-Free Response and N ...

The market expects Agios Pharmaceuticals (AGIO) to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended June 2024. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates.The earnings report, which is expected to be released on August 1, 2024, might help the stock move higher if these key numbe ...

CAMBRIDGE, Mass., July 18, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced the company will host a conference call and live webcast on Thursday, August 1, 2024, at 8:00 a.m. ET to report its second quarter 2024 financial results and other business highlights. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's w ...

Agios Pharmaceuticals (AGIO) shares soared 6.5% in the last trading session to close at $45.22. The move was backed by solid volume with far more shares changing hands than in a normal session. This compares to the stock's 29.9% gain over the past four weeks. The rise in share price is attributable to the company’s progress with its label expansion studies on sol-marketed Pyrukynd. Earlier this month, management reported that the phase III ENERGIZE-T study, evaluating the drug in transfusion-dependent (TD) ...

– ENERGIZE is the First Study to Demonstrate Efficacy of an Oral Treatment for Non-Transfusion-Dependent Alpha- and Beta-Thalassemia – – Additional ENERGIZE Poster Presentation Highlights Improvements in Fatigue and Exercise Capacity in Patients Treated with Mitapivat Compared to Placebo – – Agios to Webcast Virtual Investor Event on June 16, 2024 at 10 a.m. Eastern Time or 4 p.m. Central European Summer Time – CAMBRIDGE, Mass., June 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a ...

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 ...

Shares of Agios Pharmaceuticals (AGIO) rose more than 20% on Monday after management reported that the phase III ENERGIZE-T study, evaluating its sole marketed drug, Pyrukynd (mitapivat) in certain thalassemia patients, achieved its primary and key secondary endpoints. A total of 258 adult participants with transfusion-dependent (TD) alpha- or beta-thalassemia were enrolled in the ENERGIZE-T study. These patients were randomized into two treatment arms — 171 patients who received a 100mg oral dose of Pyruky ...

– 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. Marketi ...

– 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. Marketi ...