Core Viewpoint - Agios Pharmaceuticals received FDA approval for mitapivat (AQVESME), leading to an 18% increase in share price and a price target raise by BofA Securities from $32 to $34 while maintaining a Buy rating [1] Group 1: FDA Approval and Market Impact - Mitapivat is now the only approved therapy for both transfusion-dependent and non-transfusion-dependent alpha- and beta-thalassemia [2] - Despite delays around the PDUFA date, confidence in the approval was high due to prior regulatory clearance in Saudi Arabia and a favorable opinion from the European Medicines Agency's CHMP [2] Group 2: Drug Launch and Sales Projections - The drug's REMS requirements and black box warning related to hepatocellular injury are consistent with expectations and are not anticipated to significantly hinder adoption [3] - The launch execution for thalassemia is expected to be a major focus for 2026, with availability anticipated in late January 2026 [3] - BofA projected peak sales of approximately $1 billion for the thalassemia indication, with initial uptake expected to be gradual due to REMS certification requirements [4] Group 3: Probability of Success and Market Share - Following the approval, BofA raised the probability of success in thalassemia to 100% from 85% and modestly increased peak market share assumptions [4]

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Company Overview - Agios Pharmaceuticals (AGIO) shares increased by 18.6% to $29.17, following a significant trading volume, contrasting with a 13.5% decline over the past four weeks [1][2] FDA Approval - The stock surged after the FDA approved Aqvesme (mitapivat) for treating adult patients with alpha- or beta-thalassemia, making it the only FDA-approved drug for both non-transfusion-dependent and transfusion-dependent forms of the disease [2] Financial Expectations - The company is projected to report a quarterly loss of $1.96 per share, reflecting a year-over-year decrease of 12.6%. Expected revenues are $11.14 million, which is a 3.8% increase from the same quarter last year [3] - The consensus EPS estimate for Agios Pharmaceuticals has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Agios Pharmaceuticals is part of the Zacks Medical - Biomedical and Genetics industry, where Enanta Pharmaceuticals (ENTA) also operates. ENTA shares rose by 5.7% to $16.6, with a 15% return over the past month [5] - Enanta Pharmaceuticals has an unchanged consensus EPS estimate of -$0.54, which represents a 48.6% improvement compared to the previous year [6]

Core Viewpoint - Goldman Sachs raised the 12-month target price for Agios Pharmaceuticals (AGIO.US) from $25 to $28, indicating a potential upside of approximately 14%, while maintaining a "Neutral" rating. The analysis focuses on the market prospects and risk assessment of its core drug, Aqvesme (mitapivat), following FDA approval [1]. Group 1: Drug Approval and Market Potential - Aqvesme is the first and only drug approved for treating adult transfusion-dependent (TD) and non-transfusion-dependent (NTD) alpha or beta thalassemia, based on significant efficacy data from Phase 3 ENERGIZE-T and ENERGIZE trials [1][2]. - In the ENERGIZE trial, 42.3% of patients in the Aqvesme group achieved a hemoglobin increase of ≥1 g/dL, compared to only 1.6% in the placebo group (p<0.0001) [2]. - In the ENERGIZE-T trial, 10% of patients in the Aqvesme group became transfusion-independent within 48 weeks, while only 1% in the placebo group achieved this [2]. Group 2: Sales Forecast and Market Size - Goldman Sachs increased the probability of Aqvesme's market launch for thalassemia from 90% to 100%, setting the annual U.S. price at $425,000, higher than the $335,000 for Pyrukynd, which is used for pyruvate kinase deficiency [2]. - The management has identified approximately 4,000 "easiest" patients to target initially: 2,000 in the TD population and 2,000 with significant symptoms or low hemoglobin in the NTD group. The total addressable population in the U.S. is estimated at around 6,000, with NTD patients making up two-thirds [3]. - Goldman Sachs forecasts Aqvesme sales of $69 million for FY2026, with a gradual increase in prescriptions and revenue expected to align by late 2026 or early 2027, reaching a global peak of approximately $600 million by 2033 [3]. Group 3: Financial Projections and Valuation - Goldman Sachs adjusted revenue forecasts for 2025-2027 to $36.5 million, $47 million, and $146 million, respectively, while maintaining a trend of narrowing losses, projecting a loss of $6.25 per share in 2027, about $1 less than previous estimates [4]. - The target price of $28 is derived using a 100% risk-adjusted DCF model, with a WACC of 17% and a perpetual growth rate of 3%. This valuation includes the successful commercialization of existing assets [4]. - The company is still in a net loss position, and if commercialization speeds are below expectations, financing needs may arise again [4]. Group 4: Strategic Considerations - Goldman Sachs believes Agios will realize initial value from Aqvesme's U.S. launch in the short term, with medium-term focus on the alignment of prescriptions and revenue, and long-term potential hinging on the success of expanding indications for sickle cell disease [5]. - The target price corresponds to a revenue multiple of about ten times the 2027 revenue forecast, which, while not cheap, is considered reasonable within the narrative of rare disease blockbuster products [5]. - Investors are advised to closely monitor the prescription curve and liver safety signals starting in Q1 of next year, as alignment in these areas will be crucial for maintaining interest from event-driven funds [5].

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 ...

Cantor公司分析师埃里克·施密特表示："这比Pyrukynd(该药物的另一商品名)33.5万美元的批发 采购成本价格略有溢价。我们原本预期是平价定价。" 在实施一项必需的安全计划后，Aqvesme预计将于下个月晚些时候上市。 该药物将附带关于肝细胞损伤的黑框警告，并要求在治疗的前24周内每四周进行一次肝功能检测。 药品标签还将建议肝硬化患者不要使用。 至少有两位分析师表示，药品标签内容和相关要求符合预期。 该公司首席财务官塞西莉亚·琼斯在周三的分析师电话会议上表示，该药物每位患者的年治疗费用 约为42.5万美元。 美国食品药品监督管理局(FDA)批准了Agios Pharmaceuticals(AGIO.US)药物用于治疗某种血液疾病的 扩大适应症，该股股价周三大幅上涨18.63%。 该公司周二晚间宣布，其化学名为米他匹伐(商品名：Aqvesme)的药物获批准，成为针对非输血依 赖性和输血依赖性α或β地中海贫血患者的首个口服疗法。 地中海贫血是一种遗传性血液疾病，影响人体产生血红蛋白和健康红细胞的能力。 该药物(商品名：Pyrukynd)已于2022年获得FDA批准，用于治疗成人丙酮酸激酶缺乏症所致的红 ...

至少有两位分析师表示，药品标签内容和相关要求符合预期。 地中海贫血是一种遗传性血液疾病，影响人体产生血红蛋白和健康红细胞的能力。 在实施一项必需的安全计划后，Aqvesme预计将于下个月晚些时候上市。 该药物将附带关于肝细胞损伤的黑框警告，并要求在治疗的前24周内每四周进行一次肝功能检测。药品 标签还将建议肝硬化患者不要使用。 美国食品药品监督管理局(FDA)批准了Agios Pharmaceuticals(AGIO.US)药物用于治疗某种血液疾病的扩 大适应症，该股股价周三大幅上涨18.63%。 该公司周二晚间宣布，其化学名为米他匹伐(商品名：Aqvesme)的药物获批准，成为针对非输血依赖性 和输血依赖性α或β地中海贫血患者的首个口服疗法。 该公司首席财务官塞西莉亚.琼斯在周三的分析师电话会议上表示，该药物每位患者的年治疗费用约为 42.5万美元。 该公司针对的是美国约6000名成人地中海贫血患者群体，其中约4000名患者在药物上市初期即有机会获 得治疗。 此次最新批准是基于一项后期研究，在该研究中，与安慰剂组相比，接受该药物治疗的患者在血红蛋白 反应方面显示出统计学上的显著改善。 Cantor公司分析师 ...

Core Viewpoint - Agios Pharmaceuticals has received FDA approval for mitapivat, a treatment for anemia in adults with alpha or beta thalassemia, which will be marketed as AQVESME in the United States [2]. Group 1: FDA Approval - The FDA has approved mitapivat for treating anemia in adults with alpha or beta thalassemia [2]. - The drug will be marketed under the brand name AQVESME in the United States [2]. Group 2: Investor Relations - Morgan Sanford, Vice President of Investor Relations, is leading the conference call to discuss the approval and its implications [2]. - The company has provided access to presentation slides on its website for investors [2].

Company Overview - Agios Pharma has seen a stock return of -29% over the past year, underperforming the market by 47%, which supports a cautious investment stance [1] Leadership and Background - The company has a co-founder, Brendan, who has a Ph.D. in organic synthesis from Stanford University and has experience working with major pharmaceutical companies and biotech startups [1]

Key Points The new indication is relatively rare in the U.S., but pricing is substantial, and the need for the treatment is significant. The positive update turns the company's fortunes around after a disappointing update earlier in the year. 10 stocks we like better than Agios Pharmaceuticals › Shares in Agios Pharmaceuticals (NASDAQ: AGIO) soared by more than 18% as of 11 a.m. today. The move follows the company's receipt of approval from the Food and Drug Administration (FDA) for AQVESME (mitapiv ...