Agios Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in a research engine, multiple novels, and investigational therapies in preclinical development. It focuses on cellular metabolism and classical hematology. The company was founded by Lewis Clayton Cantley, Tak W. Mak, Craig B. Thompson and Shin-Shan Michael Su on August 7, 2007 and is headquartered in Cambridge, MA.
Clinical Trials
149
88 active
Approvals
4
Total approvals
Agencies
1
Regulatory bodies
Founded
2008
Active, not recruiting
82
55.0%
Approved For Marketing
1
0.7%
Completed
36
24.2%
Enrolling By Invitation
1
0.7%
Not yet recruiting
5
3.4%
Recruiting
21
14.1%
Terminated
3
2.0%
- Agios Pharmaceuticals terminated development of tebapivat for lower-risk myelodysplastic syndromes after a 65-patient mid-stage trial failed to meet predefined efficacy thresholds. - The pyruvate kinase activator was tested in three once-daily oral doses over 24 weeks, with the primary endpoint being transfusion independence for at least eight weeks. - Despite the setback in blood cancer, Agios will continue evaluating tebapivat for sickle cell disease with early results anticipated in the second half of 2026. - The company's shares declined 1.7% following the announcement, though analysts noted the outcome wasn't surprising given the trial's inherent risks.
- Compass Therapeutics appointed Arjun Prasad as Chief Commercial Officer and Cynthia Sirard as Chief Medical Officer, both effective January 1, 2026. - Prasad brings extensive commercial experience including over 10 successful oncology launches, notably the blockbuster VORANIGO launch in 2024 and TIBSOVO for biliary tract cancer patients. - Sirard contributes more than two decades of oncology drug development expertise, having guided multiple programs through clinical development, pivotal trials, and regulatory approval. - The appointments strengthen Compass's execution capabilities as the company advances tovecimig and its broader immuno-oncology pipeline toward potential commercialization.
- The FDA has not issued a regulatory decision on Agios Pharmaceuticals' supplemental New Drug Application for mitapivat in thalassemia treatment, extending beyond the December 7, 2025 PDUFA goal date. - Agios is collaborating with the FDA to finalize labeling documents and Risk Evaluation and Mitigation Strategy materials, with no additional efficacy or safety data requested. - The FDA has not provided a timeline for its regulatory decision, while the supplemental application remains under active review for treating adult patients with alpha- or beta-thalassemia. - The application seeks approval for both non-transfusion-dependent and transfusion-dependent thalassemia patients, representing a potential expansion of mitapivat's therapeutic indications.
- Mitapivat met its primary endpoint of hemoglobin response with 40.6% of patients achieving significant improvement compared to 2.9% on placebo (p<0.0001). - The drug failed to achieve statistical significance in reducing sickle cell pain crises, showing only a trend toward improvement with 2.62 versus 3.05 annualized events. - Patients who achieved hemoglobin response experienced clinically meaningful benefits in pain crisis reduction and fatigue improvement. - Agios plans to submit for U.S. regulatory approval after meeting with FDA in Q1 2026.
- Agios Pharmaceuticals' mitapivat (Pyrukynd) met one of two primary endpoints in the Phase 3 Rise Up trial, significantly improving hemoglobin levels in 41% of sickle cell disease patients compared to 3% on placebo. - The drug failed to achieve statistical significance in reducing annualized sickle cell pain crises, with treated patients experiencing 2.6 crises per year versus 3.1 in the placebo group. - Despite mixed results, Agios plans to meet with FDA in early 2026 to discuss a supplemental new drug application for sickle cell disease indication. - The company's stock price fell nearly 50% following the announcement, while awaiting a December 7 FDA decision on mitapivat's approval for beta thalassemia.
- Avanzanite Bioscience raised €32 million in Series A funding from MVM Partners to expand its pan-European rare disease commercialization platform across 32 countries. - The company reported record Q3 2025 performance with revenue tripling year-over-year and growing over 20% quarter-on-quarter since launching three orphan drugs. - Avanzanite's integrated business model addresses Europe's fragmented healthcare landscape by coordinating pricing, reimbursement, and regulatory strategies for rare disease therapies. - The funding will support new product partnerships, asset acquisitions, and scaling of regulatory and commercial operations to improve patient access to orphan medicines.
- ResearchAndMarkets.com released a comprehensive report analyzing 1,775 clinical-stage partnering deals in pharma and biotech from 2020-2025, providing detailed financial terms and strategic insights. - The report includes headline values, upfront payments, milestone structures, and royalty ranges for Phase I-III partnerships, with direct access to SEC-filed contracts where available. - Clinical-stage licensing agreements typically grant exclusive rights covering Phase I, Phase II, and Phase III trials, often involving collaborative R&D and co-development strategies. - The analysis profiles the top 25 most active clinical-stage dealmakers and examines the highest-value partnerships to help companies benchmark terms and optimize negotiation strategies.
- Pomerantz LLP is investigating Agios Pharmaceuticals for potential securities fraud following the company's announcement of a three-month FDA approval delay for PYRUKYND (mitapivat) in thalassemia treatment. - The FDA extended the PDUFA goal date for PYRUKYND's supplemental New Drug Application from September to December 7, 2025, affecting approval for alpha- or beta-thalassemia patients. - Agios stock fell 11.03% or $4.48 per share to close at $36.13 on September 4, 2025, following the regulatory delay announcement. - PYRUKYND is an oral pyruvate kinase activator designed to treat both non-transfusion-dependent and transfusion-dependent thalassemia patients.
- Agios' PYRUKYND (mitapivat) received a positive CHMP opinion for treating adults with thalassemia in Europe, marking a significant regulatory milestone for this first-in-class pyruvate kinase activator. - The approval is supported by two global Phase 3 trials, ENERGIZE and ENERGIZE-T, which demonstrated efficacy in both non-transfusion-dependent and transfusion-dependent thalassemia patients. - An estimated 18,000 to 23,000 children and adults are living with thalassemia in the U.S. and five largest European countries, representing a significant unmet medical need. - Mitapivat works by activating pyruvate kinase to increase cellular energy, improving red blood cell health and reducing hemolytic anemia symptoms in affected patients.
- A comprehensive 2025 clinical trials review reveals the current global research landscape for acquired autoimmune hemolytic anemia, analyzing trial data across regions, phases, and sponsor types. - The report identifies prominent pharmaceutical companies including Sanofi, Novartis, AstraZeneca, and Johnson & Johnson as key players conducting trials for this rare hematological disorder. - Analysis covers enrollment trends over five years and provides insights into trial success rates through comparative analysis of completed versus uncompleted studies. - The database compilation from over 80 clinical trial registries worldwide offers strategic intelligence for investment decisions and optimal trial location selection.
