Financial Performance - Agios Pharmaceuticals reported a loss of $1.85 per share in Q4 2025, which is narrower than the Zacks Consensus Estimate of a loss of $1.97 and compared to a loss of $1.74 per share in the same quarter last year [1][10] - Total revenues for Q4 2025 were $20 million, exceeding the Zacks Consensus Estimate of $10 million [1] - For the full year 2025, total revenues reached $54 million, reflecting a 48% year-over-year increase [10] Product Performance - The lead drug, mitapivat, is marketed as Pyrukynd and Aqvesme, with Pyrukynd approved for treating hemolytic anemia in adults with pyruvate kinase deficiency and Aqvesme for anemia in adults with alpha- or beta-thalassemia [2] - Pyrukynd generated $16 million in product revenues in the U.S., marking a 49% year-over-year increase and a 24% sequential increase [5] - Aqvesme was launched in the U.S. in December 2025 and is reported to have a strong start [3] Market Developments - Outside the U.S., mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications, with a positive opinion from the European Medicines Agency for label expansion in thalassemia [4] - Agios recorded $4 million in revenues from Pyrukynd in the ex-U.S. market, primarily due to inventory stocking as the market transitioned to commercial supply [8] Research and Development - Research and development expenses increased by approximately 6.4% year-over-year to $88.1 million in Q4 2025, driven by higher costs related to pipeline development [9] - The company is developing mitapivat for sickle cell disease (SCD) and plans to engage with the FDA in Q1 2026 before filing for approval [11][13] - Agios is also developing another candidate, tebapivat, for SCD, with patient enrollment completed and top-line results expected in the second half of 2026 [13] Stock Performance - Over the past year, Agios' stock has decreased by 15.7%, while the industry has seen an increase of 17.9% [4]

Group 1 - Goldman Sachs raised the 12-month target price for Agios Pharmaceuticals from $25 to $28 while maintaining a "Neutral" rating [1] - The firm believes that Agios will realize its first wave of value through the U.S. launch of Aqvesme in the short term [1] - In the medium term, the focus will be on when the prescription revenue mismatch will converge [1] - The long-term outlook depends on whether Agios can achieve further success in the sickle cell disease indication [1]

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].