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 ...

产品研发 - 公司在2024年第一季度取得了积极的Thalassemia Phase 3 ENERGIZE试验结果[12] - Mitapivat有望成为第一个获批用于所有地中海贫血亚型的疗法[11] - PYRUKYND是一种新型口服疗法，有望成为最佳疗法，改善贫血，减少SCPCs并改善患者的感觉和功能[15] - 公司的早期阶段管线致力于解决罕见疾病的病理生理学问题，满足高度未满足的需求[16] - PYRUKYND®有潜力成为首个获得所有地中海贫血亚型批准的疗法[20] - PYRUKYND®扩展到患有更大患者群的疾病，为首个和最佳疗法提供了重要的短期增长潜力[26] - PYRUKYND®在美国、欧盟和英国获得成人丙酮酸激酶（PK）缺乏症的批准，正在对儿童PK缺乏症、地中海贫血和镰状细胞病进行调查[26] - PYRUKYND®第一个3期项目涵盖了所有地中海贫血患者范围[33] 业绩展望 - PYRUKYND®的净销售额为8.2百万美元，较2023年第四季度增长15%[25] 临床试验结果 - ENERGIZE-T试验设计反映了真实世界的临床治疗动态[14] - ENERGIZE研究的主要终点已达成，显示出与安慰剂相比，米他匹韦治疗显著增加了血红蛋白反应率[35] - 米他匹韦治疗显示出与安慰剂相比的显著增加血红蛋白反应率[36] - 基线定义为随机分配前42天内的所有评估的平均值，不包括RBC输血后8周内的Hb浓度评估[37] - 调整后的反应率差异估计值、95%置信区间和p值基于Mantel-Haenszel分层加权方法进行调整[38] - 100 毫克 mitapivat 治疗显示出与安慰剂相比在血红蛋白反应率上的显着增加[41] - Mitapivat 治疗组患者的镰状细胞疼痛危机的年化发作率较安慰剂组低[43] - PYRUKYND® 在镰状细胞病中的 Phase 3 主要终点为血红蛋白反应和镰状细胞疼痛危机的年化发作率[46]

财务数据和关键指标变化 - 第一季度PYRUKYND净收入为8.2百万美元,较2023年第一季度增加2.6百万美元 [48] - 销售成本为0.6百万美元 [49] - 研发费用为68.6百万美元,较2023年第一季度增加1.3百万美元,主要由于工艺开发费用增加抵消了人员相关支出的减少 [49] - 销售、一般及管理费用为31百万美元,较上年同期增加2.6百万美元,主要由于商业活动增加 [50] - 公司现金、现金等价物和有价证券余额约为714.3百万美元,预计可为公司运营和资本支出提供资金支持,至少到2026年 [51] 各条业务线数据和关键指标变化 - 非输血依赖性地中海贫血患者的ENERGIZE研究取得积极结果,达到主要和次要终点 [8][9][21] - 输血依赖性地中海贫血患者的ENERGIZE-T研究预计于第二季度报告数据,公司计划在今年底前向FDA提交涵盖ENERGIZE和ENERGIZE-T的单一监管申请 [10][28] - 镰状细胞病RISE UP III期研究预计于年底前完成入组,并于明年报告数据 [11][29] - 苯丙酮尿症AG-181 I期研究已开始给药,公司期待引入新机制治疗 [30] - 低危骨髓增生异常综合征AG-946 II期研究计划于2024年中期启动 [31] - 儿童PK缺乏症ACTIVATE-kids和ACTIVATE-kids-T研究进展顺利,预计分别于今年中期和2024年中期报告数据 [31] 各个市场数据和关键指标变化 - 美国约有6,000名诊断的成人地中海贫血患者,其中4,000名为非输血依赖型,目前无可用治疗方案 [34] - 美国约有2,000名输血依赖型地中海贫血患者,目前也无口服治疗选择 [34] - 公司正积极准备在2025年前在美国推出mitapivat治疗地中海贫血,并于2026年推出治疗镰状细胞病 [13][35] - 公司还计划通过与合作伙伴在海湾地区等海外市场推广mitapivat [42] 公司战略和发展方向及行业竞争 - 公司认为mitapivat凭借其独特的机制改善红细胞健康,有望成为多种溶血性贫血的首创和最佳治疗选择,有潜力成为数十亿美元的产品线 [12][54] - 公司正在加强商业团队,为mitapivat在地中海贫血和镰状细胞病的潜在上市做好准备 [13][35] - 公司认为mitapivat在溶血性贫血领域的临床数据优势,以及口服给药的便利性,有望在与竞争对手Novo Nordisk的PK激活剂etavopivat中脱颖而出 [66][67][88] 管理层对经营环境和未来前景的评论 - 公司对mitapivat在地中海贫血和镰状细胞病的潜力充满信心,认为其有望成为多适应症的数十亿美元产品线 [12][54] - 公司认为mitapivat的独特机制和一致的临床数据,为其在溶血性贫血领域的领先地位奠定了基础 [24][26][54] - 公司对mitapivat在PK缺乏症、地中海贫血和镰状细胞病的III期研究结果充满期待,预计未来24个月将是催化剂丰富的时期 [11][55] 问答环节重要的提问和回答 问题1 **Gregory Renza 提问** 询问ENERGIZE-T研究数据时间表更新的原因以及患者转入公开标签延长研究的机制 [61][62][63] **Sarah Gheuens 回答** 最后几名患者是否选择转入公开标签延长研究是影响时间表的关键因素,公司希望尽可能缩短时间表 [63][64] 问题2 **Divya Rao 提问** 询问ENERGIZE-T研究主要终点的统计学假设 [71][72] **Sarah Gheuens 回答** 公司根据既往研究数据和监管反馈设计了该研究,对主要终点的统计学假设有充分的理论依据,但具体细节暂未披露 [72] 问题3 **Alec Stranahan 提问** 询问公司是否需要针对输血依赖和非输血依赖地中海贫血患者采取不同的销售策略 [87][88][89] **Tsveta Milanova 回答** 公司计划采取相同的销售策略,因为两类患者的基础病理学相同,处方医生群体也有重叠,但会根据患者特点在执行中有所侧重 [89][90]

Agios Pharmaceuticals (AGIO) came out with a quarterly loss of $1.45 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to loss of $1.47 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 11.59%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $1.64 per share when it actually produced a loss of $1.72, delivering a surprise of -4.88%.Over the last four quarters, ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 001-36014 AGIOS PHARMACEUTICALS, INC. (Exact Name of Registrant as Specified in Its Charter) Delaware 26-0662915 (State or Other Jurisdiction of (I ...

Exhibit 99.1 Agios Reports Business Highlights and First Quarter 2024 Financial Results – Topline Data Readout from the Phase 3 ENERGIZE-T Study of Mitapivat in Adults with Transfusion- Dependent Alpha- or Beta-Thalassemia now expected in Q2 2024 – – Announced Positive Results from the Phase 3 ENERGIZE Study of Mitapivat in Adults with Non-Transfusion- Dependent Alpha- or Beta-Thalassemia in January – – Phase 3 Readouts from Mitapivat RISE UP Study in Sickle Cell Disease, ACTIVATE-KIDS and ACTIVATE KIDS-T i ...

– Report Highlights Company's Focus on Red Blood Cell Health; Diversity, Equity & Inclusion; and Strong Corporate Governance – – Aligns with the Sustainability Accounting Standards Board (SASB) Standards for the Biotechnology and Pharmaceuticals Industry and the United Nations Sustainable Development Goals (UN SDGs) – CAMBRIDGE, Mass., April 09, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today ...