Key Takeaways AGIO says the FDA missed its Dec. 7 deadline for a Pyrukynd thalassemia sNDA decision.The sNDA remains under FDA review but no additional efficacy or safety data have been requested.Pyrukynd is already approved for treating hemolytic anemia in adults with PK deficiency.Agios Pharmaceuticals (AGIO) announced that the FDA has not yet given its decision related to the company’s supplemental new drug application (sNDA) seeking approval for its only marketed drug, Pyrukynd (mitapivat), for treating ...

CAMBRIDGE, Mass., Dec. 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has not yet issued a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia and tha ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) ranks among the most oversold biotech stocks to invest in. On November 20, Leerink Partners upgraded Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) from Market Perform to Outperform with a $34 price target. The rating upgrade comes despite Agios’ conflicting topline data for its Phase 3 RISE UP trial assessing mitapivat in sickle cell disease (SCD). The trial reached its primary goal, with 40.6% of patients receiving mitapivat demonstrating a 1.0 g/dL or higher increa ...

Core Insights - Agios Pharmaceuticals' shares dropped approximately 51% following mixed results from the phase III RISE UP study of Pyrukynd in sickle cell disease patients [1][6] - The study met its primary endpoint of improving hemoglobin levels but failed to reduce the annualized rate of sickle cell pain crises [1][7] Efficacy Results - Nearly 41% of patients treated with Pyrukynd achieved a hemoglobin response compared to about 3% in the placebo group [2] - Pyrukynd-treated patients showed a numerical reduction in sickle cell pain crises (2.62 vs. 3.05 for placebo) but did not achieve statistical significance [2][3] - Statistically significant improvements were observed in average hemoglobin concentration and indirect bilirubin levels, but no improvement in patient-reported fatigue was noted [3] Regulatory Plans - Despite mixed results, Agios plans to proceed with a regulatory filing for Pyrukynd, focusing on a subgroup of hemoglobin responders who showed clearer clinical benefits [4] - The company aims to submit this filing after discussions with the FDA in the first quarter of 2026 [4] Current Drug Status - Pyrukynd is already approved in the U.S. and Europe for treating hemolytic anemia in adults with pyruvate kinase deficiency [5] - A regulatory filing for label expansion to include non-transfusion-dependent and transfusion-dependent thalassemia patients is under FDA review, with a decision expected by December 7, 2025 [5] Stock Performance - Following the study results, Agios shares reached a 52-week low of $22.24, reflecting investor disappointment [6] - Year-to-date, Agios shares have declined by 32%, contrasting with a 17% growth in the industry [6] Competitive Landscape - Shares of Fulcrum Therapeutics rose over 18% after Agios' announcement, as Fulcrum is developing a different investigational drug for sickle cell disease [9]

Core Insights - Top Wall Street analysts have revised their outlook on several prominent stocks, indicating a shift in market sentiment and potential investment opportunities [1] Company Analysis - Analysts are considering buying RCEL stock, suggesting a positive outlook for the company [1]

Group 1 - Agios Pharmaceuticals, Inc. (AGIO) stock has experienced a significant decline, down 49% and trading at $23 per share, resulting in a market capitalization of approximately $1.4 billion [1] - The article highlights the importance of staying updated on stocks within the biotech, pharma, and healthcare sectors, emphasizing key trends and catalysts that influence market valuations [1][2] Group 2 - The investing group Haggerston BioHealth, led by a biotech consultant with over 5 years of experience, provides insights and detailed reports on more than 1,000 companies in the biotech and healthcare sectors [2] - Haggerston BioHealth offers various resources for investors, including product sales forecasts, integrated financial statements, discounted cash flow analysis, and market-by-market analysis [2]

Core Points - The conference call is focused on the top line results from the pivotal Phase III RISE UP trial of mitapivat for treating sickle cell disease [2] - The call includes key executives from Agios Pharmaceuticals, indicating a comprehensive discussion on the trial results and future implications [4] Company Overview - Agios Pharmaceuticals is actively engaging with investors to discuss significant trial outcomes, showcasing its commitment to transparency and investor relations [2][4] - The company is preparing to share detailed findings from the RISE UP trial, which is crucial for its product pipeline and market strategy [2] Industry Context - The Phase III RISE UP trial represents a critical step in the development of treatments for sickle cell disease, a condition with significant unmet medical needs [2] - The results from this trial could have implications for the broader pharmaceutical industry, particularly in the area of rare diseases and innovative therapies [2]

Group 1 - Shares in Agios Pharmaceuticals experienced a significant decline of 50% following mixed results from its Phase 3 trial of the sickle cell disease drug, mitapavit [1] - The Phase 3 trial had two primary endpoints, achieving success in one while failing to meet the other [2] - The trial demonstrated a statistically significant response in average hemoglobin levels, with 40.6% of treated patients showing improvement compared to 2.9% in the placebo group [3] Group 2 - Despite the failure to significantly reduce sickle cell pain crises (SCPCs), management highlighted the potential value of mitapavit due to its "anti-hemolytic profile" [5][7] - The company plans to engage with the FDA and submit a marketing application for mitapavit in the first quarter of 2026 [5][7] - The market reaction to the trial results suggests a potential overreaction, indicating that there may still be investment opportunities in Agios Pharmaceuticals [5]

Market Overview - U.S. stocks showed mixed performance with the Dow Jones index falling approximately 0.2% while NASDAQ and S&P 500 saw slight gains of 0.19% and 0.08% respectively [1] - Communication services sector rose by 0.8%, while energy stocks declined by 1.6% [1] Company Performance - Lowe's Companies, Inc. (NYSE:LOW) shares increased over 5% after exceeding Wall Street profit expectations for Q3, reporting adjusted earnings per share of $3.06, a 5.9% year-over-year increase, surpassing the consensus estimate of $2.97 [2][3] - The company slightly missed sales expectations but raised its full-year revenue target, focusing on professional customers and online growth [2] Commodity Market - Oil prices fell by 2.4% to $59.26, while gold prices increased by 0.3% to $4,077.20 [6] - Silver and copper also saw gains, with silver up 0.2% to $50.615 and copper rising 0.4% to $4.9945 [6] Stock Volatility - WeShop Holdings Limited (NASDAQ:WSHP) shares surged 413% to $169.32 following its Nasdaq debut [8] - Freight Technologies, Inc. (NASDAQ:FRGT) shares rose 67% to $1.1045 after launching its AI-powered freight rate prediction platform [8] - Semrush Holdings, Inc. (NYSE:SEMR) shares increased by 74% to $11.79 after Adobe announced acquisition plans [8] - Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) shares dropped 50% to $22.88 due to Phase 3 trial results not meeting statistical significance [8] - Carver Bancorp, Inc. (NASDAQ:CARV) shares fell 51% to $1.4702 after announcing plans to de-register its common stock [8] - Tempest Therapeutics, Inc. (NASDAQ:TPST) shares decreased by 47% to $4.94 following an acquisition announcement [8] International Markets - European shares mostly declined, with the eurozone's STOXX 600 down 0.03% and major indices like London's FTSE 100 and Germany's DAX 40 also falling [7] - Asian markets closed mixed, with Japan's Nikkei 225 and Hong Kong's Hang Seng both down, while China's Shanghai Composite and India's BSE Sensex gained [9] Economic Indicators - U.S. crude oil inventories decreased by 3.426 million barrels, significantly more than the expected 0.6 million [12] - U.S. exports rose by $0.2 billion to $280.8 billion, while imports fell by 5.1% to $340.4 billion, leading to a reduced trade deficit of $59.6 billion [12]

Core Viewpoint - Agios Pharmaceuticals Inc. experienced a significant stock decline following the release of Phase 3 trial results for mitapivat in treating sickle cell disease, despite meeting its primary endpoint of hemoglobin response [1][10]. Trial Results - The RISE UP Phase 3 trial met its primary endpoint, showing a statistically significant improvement in hemoglobin response with 40.6% of patients in the mitapivat arm achieving this response compared to 2.9% in the placebo arm [2][3]. - Mitapivat also demonstrated a reduction in the annualized rate of sickle cell pain crises (SCPCs), with rates of 2.62 in the mitapivat arm versus 3.05 in the placebo arm, although this did not achieve statistical significance [3][4]. - Statistically significant improvements were observed in two key secondary endpoints: average change in hemoglobin concentration and levels of indirect bilirubin from baseline [4][6]. Safety and Efficacy - The safety profile of mitapivat in this trial was consistent with previous studies, with an average change in hemoglobin concentration of 7.69 g/L in the mitapivat arm compared to 0.26 g/L in the placebo arm, indicating a statistically significant improvement [6]. - The average change in indirect bilirubin was -16.03 µmol/L in the mitapivat arm versus 0.88 µmol/L in the placebo arm, also showing a statistically significant improvement [7]. Future Plans - Agios plans to submit a marketing application for mitapivat in the U.S. for sickle cell disease after a pre-supplemental New Drug Application meeting with the FDA in early 2026 [8]. - The company is also focused on other commercial and pipeline milestones, including the potential U.S. approval of Pyrukynd (mitapivat) for thalassemia, expected in December 2025 [8][9]. Stock Performance - Following the trial results, Agios Pharmaceuticals' stock fell by 49.00%, trading at $23.20, near its 52-week low of $23.41 [10].