Monday's ETF Movers: SPXL, BBSC
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Aug 04 2025
0mins
Source: NASDAQ.COM
ETF Performance: The JPMorgan BetaBuilders U.S. Small Cap Equity ETF is down approximately 1.9% in afternoon trading, with Agios Pharmaceuticals and Twist Bioscience showing significant declines of 17.9% and 14.3%, respectively.
Author's Perspective: The opinions expressed in the article are solely those of the author and do not necessarily represent Nasdaq, Inc.
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Analyst Views on AGIO
Wall Street analysts forecast AGIO stock price to rise
9 Analyst Rating
6 Buy
3 Hold
0 Sell
Moderate Buy
Current: 28.330
Low
25.00
Averages
36.14
High
62.00
Current: 28.330
Low
25.00
Averages
36.14
High
62.00
About AGIO
Agios Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on developing and delivering transformative therapies for patients living with rare diseases. It markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for debilitating hemolytic anemia. Its lead product candidate in its portfolio, PYRUKYND (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. It is also developing tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes, or LR MDS, and hemolytic anemias; AG-181, its phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU; and AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., targeting the transmembrane serine protease 6, or TMPRSS6 gene for the potential treatment of polycythemia vera, or PV.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Agios Receives EU Approval for PYRUKYND
- Drug Approval: Agios Pharmaceuticals and its partner Avanzanite Bioscience announced that the European Commission has granted marketing authorization for their lead product PYRUKYND to treat transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking the second indication for the drug in the EU.
- Clinical Trial Support: The approval is based on a positive opinion from the European Medicines Agency (EMA) and relies on results from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated the efficacy and safety of Mitapivat in adults with thalassemia.
- Market Launch Plan: Under an exclusive agreement with Agios, Avanzanite Bioscience will commercialize and distribute PYRUKYND across the European Economic Area, the UK, and Switzerland, with CEO Adam Plich stating their commitment to collaborate with local authorities to drive a successful launch and ensure broad access.
- Stock Price Dynamics: AGIO's stock has traded between $22.24 and $46.00 over the past year, closing at $28.14, down 2.26%, and falling to $27.60 in after-hours trading, indicating a cautious market reaction to the new drug approval.
See More
Avanzanite Launches PYRUKYND in the EU
- Drug Approval: Avanzanite, in partnership with Agios, has received marketing authorization for PYRUKYND in the EU, introducing a new oral treatment option for adult patients with transfusion-dependent and non-transfusion-dependent alpha or beta-thalassaemia, marking its second indication in the EU.
- Clinical Trial Support: This approval is based on results from the global randomized double-blind ENERGIZE and ENERGIZE-T Phase 3 trials, demonstrating significant efficacy in improving patient quality of life and reducing transfusion burden, highlighting its clinical importance.
- Market Access Commitment: Avanzanite is committed to collaborating with local health authorities to ensure broad access to PYRUKYND across the EU, aiming to help all thalassaemia patients access this new therapy, reflecting the company's dedication to patient care.
- Commercial Platform Advantage: This launch marks Avanzanite's fourth drug introduction in the rare disease sector, showcasing its strong commercial platform across 32 European countries and its capability to partner with biotech innovators to enhance treatment accessibility for rare diseases.
See More
PYRUKYND Approved in EU for Thalassemia Treatment
- Regulatory Milestone: PYRUKYND (mitapivat) has received marketing authorization from the European Commission, becoming the only approved treatment for both transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking a significant advancement for thousands of patients.
- Clinical Trial Validation: This approval is based on results from global randomized Phase 3 trials, demonstrating PYRUKYND's efficacy in alleviating anemia symptoms and improving quality of life, highlighting its substantial clinical significance.
- Market Expansion Strategy: Agios has entered into an exclusive agreement with Avanzanite Bioscience for the commercialization and distribution of PYRUKYND across the European Economic Area, further solidifying its position in the global rare disease market.
- Global Recognition: With approvals in the U.S., Saudi Arabia, UAE, and now the EU, PYRUKYND reflects widespread demand and potential market opportunities globally, indicating promising future sales growth.
See More
Agios Pharmaceuticals Presents New Data on Mitapivat at EHA 2026
- RISE UP Trial Results: At EHA 2026, Agios will present detailed efficacy and safety data from the RISE UP Phase 3 trial, demonstrating that patients treated with mitapivat showed significant improvements in average hemoglobin levels over 52 weeks and reduced markers of hemolysis, indicating the drug's potential in treating sickle cell disease.
- Long-term Efficacy Validation: In the ENERGIZE Phase 3 trial, 95% of patients opted to transition into the open-label extension period, showing that mitapivat treatment was well-tolerated and maintained clinically meaningful hemoglobin improvements for up to 127 weeks, further supporting its long-term use.
- Patient Feedback Improvement: Among patients receiving mitapivat, approximately 38.9% achieved a hemoglobin response during the double-blind period, with significant improvements in fatigue scores, highlighting the drug's positive impact on enhancing patients' quality of life.
- Global Academic Exchange: Agios will conduct 10 presentations and publications at EHA 2026, showcasing its research advancements in rare blood diseases, further solidifying its leadership position in the biopharmaceutical industry.
See More
Agios Submits sNDA for Accelerated Approval of Mitapivat in Sickle Cell Disease
- Clinical Trial Design: Agios' supplemental New Drug Application (sNDA) submission is based on an agreement with the FDA requiring a confirmatory clinical trial to demonstrate the clinical benefit of mitapivat in reducing transfusion burden in sickle cell disease, with approximately 159 patients expected to be enrolled over a 52-week period.
- Key Endpoint Setting: The primary endpoint of the confirmatory trial is the transfusion-free rate from Week 4 to Week 52, aiming to showcase mitapivat's effectiveness in lowering transfusion needs compared to placebo, reflecting its potential market value in sickle cell disease treatment.
- Clinical Data Support: In the RISE UP Phase 3 trial, mitapivat demonstrated significant improvements in hemoglobin concentration and reduced hemolysis, translating clinically into meaningful reductions in pain crises and hospitalizations, indicating its strong potential in treating sickle cell disease.
- Market Outlook: Agios expects to receive notice of the sNDA filing acceptance from the FDA in the third quarter of 2026, and if approved, mitapivat will become the first PK activator approved in the U.S., addressing the urgent need for new therapies among sickle cell disease patients.
See More
Agios Pharmaceuticals Shares Rise 11.2% Following Strong Earnings Report
- Stock Surge: Agios Pharmaceuticals saw an 11.2% increase in shares this week, marking a positive turnaround for the year, reflecting market confidence in the company's future, particularly following the successful launch of Aqvesme.
- Significant Sales Growth: Aqvesme's sales reached $20.7 million in Q1 2026, a substantial increase from $8.7 million in the same quarter of 2025, which not only boosts overall revenue but also establishes a solid foundation for future market demand.
- New Drug Progress: Management confirmed alignment with the FDA, expecting to file for accelerated approval of mitapivat for sickle cell disease in Q2 2026, despite facing competition from Novo Nordisk, indicating strong market potential.
- Optimistic Future Outlook: Management is optimistic about the Phase 2 trial results for tebapivat, expected in the first half of 2026 for MDS and the second half for SCD, which will provide new momentum for the company's growth trajectory.
See More
Agios Receives EU Approval for PYRUKYND
- Drug Approval: Agios Pharmaceuticals and its partner Avanzanite Bioscience announced that the European Commission has granted marketing authorization for their lead product PYRUKYND to treat transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking the second indication for the drug in the EU.
- Clinical Trial Support: The approval is based on a positive opinion from the European Medicines Agency (EMA) and relies on results from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated the efficacy and safety of Mitapivat in adults with thalassemia.
- Market Launch Plan: Under an exclusive agreement with Agios, Avanzanite Bioscience will commercialize and distribute PYRUKYND across the European Economic Area, the UK, and Switzerland, with CEO Adam Plich stating their commitment to collaborate with local authorities to drive a successful launch and ensure broad access.
- Stock Price Dynamics: AGIO's stock has traded between $22.24 and $46.00 over the past year, closing at $28.14, down 2.26%, and falling to $27.60 in after-hours trading, indicating a cautious market reaction to the new drug approval.
See More
Avanzanite Launches PYRUKYND in the EU
- Drug Approval: Avanzanite, in partnership with Agios, has received marketing authorization for PYRUKYND in the EU, introducing a new oral treatment option for adult patients with transfusion-dependent and non-transfusion-dependent alpha or beta-thalassaemia, marking its second indication in the EU.
- Clinical Trial Support: This approval is based on results from the global randomized double-blind ENERGIZE and ENERGIZE-T Phase 3 trials, demonstrating significant efficacy in improving patient quality of life and reducing transfusion burden, highlighting its clinical importance.
- Market Access Commitment: Avanzanite is committed to collaborating with local health authorities to ensure broad access to PYRUKYND across the EU, aiming to help all thalassaemia patients access this new therapy, reflecting the company's dedication to patient care.
- Commercial Platform Advantage: This launch marks Avanzanite's fourth drug introduction in the rare disease sector, showcasing its strong commercial platform across 32 European countries and its capability to partner with biotech innovators to enhance treatment accessibility for rare diseases.
See More
PYRUKYND Approved in EU for Thalassemia Treatment
- Regulatory Milestone: PYRUKYND (mitapivat) has received marketing authorization from the European Commission, becoming the only approved treatment for both transfusion-dependent and non-transfusion-dependent alpha or beta-thalassemia, marking a significant advancement for thousands of patients.
- Clinical Trial Validation: This approval is based on results from global randomized Phase 3 trials, demonstrating PYRUKYND's efficacy in alleviating anemia symptoms and improving quality of life, highlighting its substantial clinical significance.
- Market Expansion Strategy: Agios has entered into an exclusive agreement with Avanzanite Bioscience for the commercialization and distribution of PYRUKYND across the European Economic Area, further solidifying its position in the global rare disease market.
- Global Recognition: With approvals in the U.S., Saudi Arabia, UAE, and now the EU, PYRUKYND reflects widespread demand and potential market opportunities globally, indicating promising future sales growth.
See More
Agios Pharmaceuticals Presents New Data on Mitapivat at EHA 2026
- RISE UP Trial Results: At EHA 2026, Agios will present detailed efficacy and safety data from the RISE UP Phase 3 trial, demonstrating that patients treated with mitapivat showed significant improvements in average hemoglobin levels over 52 weeks and reduced markers of hemolysis, indicating the drug's potential in treating sickle cell disease.
- Long-term Efficacy Validation: In the ENERGIZE Phase 3 trial, 95% of patients opted to transition into the open-label extension period, showing that mitapivat treatment was well-tolerated and maintained clinically meaningful hemoglobin improvements for up to 127 weeks, further supporting its long-term use.
- Patient Feedback Improvement: Among patients receiving mitapivat, approximately 38.9% achieved a hemoglobin response during the double-blind period, with significant improvements in fatigue scores, highlighting the drug's positive impact on enhancing patients' quality of life.
- Global Academic Exchange: Agios will conduct 10 presentations and publications at EHA 2026, showcasing its research advancements in rare blood diseases, further solidifying its leadership position in the biopharmaceutical industry.
See More
Agios Submits sNDA for Accelerated Approval of Mitapivat in Sickle Cell Disease
- Clinical Trial Design: Agios' supplemental New Drug Application (sNDA) submission is based on an agreement with the FDA requiring a confirmatory clinical trial to demonstrate the clinical benefit of mitapivat in reducing transfusion burden in sickle cell disease, with approximately 159 patients expected to be enrolled over a 52-week period.
- Key Endpoint Setting: The primary endpoint of the confirmatory trial is the transfusion-free rate from Week 4 to Week 52, aiming to showcase mitapivat's effectiveness in lowering transfusion needs compared to placebo, reflecting its potential market value in sickle cell disease treatment.
- Clinical Data Support: In the RISE UP Phase 3 trial, mitapivat demonstrated significant improvements in hemoglobin concentration and reduced hemolysis, translating clinically into meaningful reductions in pain crises and hospitalizations, indicating its strong potential in treating sickle cell disease.
- Market Outlook: Agios expects to receive notice of the sNDA filing acceptance from the FDA in the third quarter of 2026, and if approved, mitapivat will become the first PK activator approved in the U.S., addressing the urgent need for new therapies among sickle cell disease patients.
See More
Agios Pharmaceuticals Shares Rise 11.2% Following Strong Earnings Report
- Stock Surge: Agios Pharmaceuticals saw an 11.2% increase in shares this week, marking a positive turnaround for the year, reflecting market confidence in the company's future, particularly following the successful launch of Aqvesme.
- Significant Sales Growth: Aqvesme's sales reached $20.7 million in Q1 2026, a substantial increase from $8.7 million in the same quarter of 2025, which not only boosts overall revenue but also establishes a solid foundation for future market demand.
- New Drug Progress: Management confirmed alignment with the FDA, expecting to file for accelerated approval of mitapivat for sickle cell disease in Q2 2026, despite facing competition from Novo Nordisk, indicating strong market potential.
- Optimistic Future Outlook: Management is optimistic about the Phase 2 trial results for tebapivat, expected in the first half of 2026 for MDS and the second half for SCD, which will provide new momentum for the company's growth trajectory.
See More
