Agios Pharmaceuticals Inc Stock Price, News & Analysis

Agios Pharmaceuticals (NASDAQ: AGIO) reported Q2 2025 financial results, highlighting $12.5 million in PYRUKYND net revenues, a significant increase from $8.6 million in Q2 2024. The company maintains a strong financial position with $1.3 billion in cash and equivalents.

Key developments include the pending FDA review of PYRUKYND's sNDA for thalassemia with a PDUFA date of September 7, 2025, and anticipated topline results from the RISE UP Phase 3 trial in sickle cell disease by year-end. The company reported a net loss of $112.0 million for Q2 2025, compared to $96.1 million in Q2 2024, with increased R&D expenses of $91.9 million and SG&A expenses of $45.9 million.

Notable operational progress includes 248 unique patients completing prescription enrollment forms and 142 patients currently on PYRUKYND therapy. The company also secured a European distribution agreement with Avanzanite Bioscience and received IND clearance for AG-236 in polycythemia vera treatment.

Agios Pharmaceuticals (Nasdaq: AGIO), a company focused on cellular metabolism and PK activation therapies for rare diseases, will host its second quarter 2025 financial results conference call and webcast on July 31, 2025, at 8:00 a.m. ET.

The presentation will be available through the company's website investor section, with a replay accessible approximately two hours after the event.

Agios Pharmaceuticals (Nasdaq: AGIO), a leader in cellular metabolism and PK activation therapies, has appointed Dr. Jay Backstrom to its Board of Directors. Dr. Backstrom, who will serve on the Science and Technology Committee, brings extensive experience in advancing clinical programs and regulatory approvals, particularly in rare diseases.

Dr. Backstrom's recent roles include serving as President and CEO of Scholar Rock, where he led the company's spinal muscular atrophy program through Phase 3 development. His previous positions include Executive VP of Research and Development at Acceleron Pharma and Chief Medical Officer at Celgene Corporation, where he contributed to regulatory approvals in beta thalassemia and myelodysplastic syndromes.

Avanzanite Bioscience has secured an exclusive agreement with Agios Pharmaceuticals to commercialize and distribute PYRUKYND® (mitapivat) across Europe. PYRUKYND®, the first-in-class oral pyruvate kinase activator, is approved for treating PK deficiency in adults by both the European Commission and UK's Healthcare Regulatory Agency. The partnership covers the European Economic Area, UK, and Switzerland, with potential expansion to future indications. Avanzanite, which tripled its revenue in Q1 2025, plans to expand into 32 European countries including Italy, France, UK, Romania, and Spain. The collaboration aims to provide a capital-efficient pathway for rare disease therapies in Europe, eliminating the need for costly local infrastructure. PK deficiency is an ultra-rare inherited condition causing premature red blood cell breakdown and chronic anemia.

Agios Pharmaceuticals (NASDAQ: AGIO), a company focused on cellular metabolism and pyruvate kinase activation for rare diseases, has announced its upcoming participation in the Goldman Sachs 46th Annual Global Healthcare Conference. The presentation is scheduled for June 9, 2025, at 8:40 a.m. ET. Investors and interested parties can access the live webcast through the Investors section of Agios' website under "Events & Presentations." A replay will remain available on the company's website for a minimum of two weeks after the presentation.

Agios Pharmaceuticals (NASDAQ: AGIO) will present new data on its pyruvate kinase (PK) activators, mitapivat and tebapivat, at the 30th EHA Congress in Milan. Key highlights include:

The ACTIVATE-KidsT Phase 3 study of mitapivat in children with PK deficiency showed meaningful reduction in transfusion burden, though not meeting statistical criteria. The ESTIMATE Phase 2 trial demonstrated sustained three-year efficacy of mitapivat in sickle cell disease patients. Preclinical data revealed tebapivat's potential in reducing red blood cell sickling in sickle cell disease samples.

The company will present 14 studies across various rare blood disorders including thalassemia, sickle cell disease, PK deficiency, and myelodysplastic syndromes at the conference scheduled for June 12-15, 2025.

Agios Pharmaceuticals (NASDAQ: AGIO), a biotechnology company focused on cellular metabolism and PK activation therapies for rare diseases, has announced its upcoming participation in the 2025 RBC Capital Markets Global Healthcare Conference. The presentation is scheduled for May 21, 2025, at 8:00 AM ET. Investors and interested parties can access the live webcast through the Investors section of Agios' website under "Events & Presentations." A replay will remain available on the company's website for a minimum of two weeks after the presentation.

Agios Pharmaceuticals (NASDAQ: AGIO), a company focused on cellular metabolism and PK activation therapies for rare diseases, has announced its upcoming participation in the BofA Securities 2025 Health Care Conference. The presentation is scheduled for May 14, 2025, at 8:00 AM PT/11:00 AM ET. Investors and interested parties can access the live webcast through the Investors section of Agios' website under "Events & Presentations." A recording of the presentation will remain available on the company's website for a minimum of two weeks after the event.

Agios Pharmaceuticals (NASDAQ: AGIO) reported its Q1 2025 financial results and business updates. PYRUKYND generated $8.7 million in net revenue, up from $8.2 million in Q1 2024, with 136 patients currently on therapy. The FDA is reviewing PYRUKYND's sNDA for thalassemia treatment with a PDUFA date of September 7, 2025. The Phase 3 RISE UP study for sickle cell disease is progressing with topline results expected in late 2025. Financially, Agios reported a net loss of $89.3 million for Q1 2025, compared to $81.5 million in Q1 2024. The company maintains a strong cash position of $1.4 billion as of March 31, 2025. R&D expenses increased to $72.7 million, while SG&A expenses rose to $41.5 million due to commercial preparation activities.