Agios Pharmaceuticals, Inc. reports developments as a commercial-stage biopharmaceutical company focused on rare diseases and hematology. Its recurring updates center on mitapivat, an oral pyruvate kinase activator marketed as PYRUKYND® and AQVESME™, including product revenue, U.S. and ex-U.S. commercialization, thalassemia approvals, and activity in pyruvate kinase deficiency, sickle cell disease, and other hemolytic anemias.
Company news also covers FDA interactions, supplemental application activity, conference presentations, and pipeline progress for tebapivat, a next-generation PK activator, and other rare disease programs. International commercialization themes include regulatory approvals and distribution activity in Gulf Cooperation Council markets through NewBridge Pharmaceuticals.
Silence Therapeutics (SLN) agreed to a cash acquisition by Merck KGaA, Darmstadt, Germany, valuing its equity at about $1.3 billion. The October 19 agreement offers $20.00 per American Depositary Share (ADS), a 130% premium to the October 16 close and an 82% premium to the 30-day volume-weighted average price of $10.99. Enterprise value is $1.1 billion. The U.K. scheme of arrangement is expected to close in the first quarter of 2027, subject to shareholder, court and regulatory approvals.
The deal centers on divesiran, an RNA-based therapy for polycythemia vera, a disorder involving excess red blood cells. Its Phase 2 SANRECO trial reported an 88% response rate versus 19% with placebo. Silence ADSs rose 125.58% to $19.58 on October 19. Separately, Disc Medicine (IRON) reported that its Phase 3 APOLLO trial of bitopertin met all primary and secondary endpoints; its shares rose 38.86%.
Agios Pharmaceuticals (AGIO) will host its third-quarter 2026 financial results conference call and webcast on November 5, 2026. The event begins at 8:00 a.m. ET and will cover financial results and business highlights. The live webcast will be accessible through the company website’s Investors section under “Events & Presentations,” with a replay available approximately two hours after the event.
Agios (Nasdaq: AGIO) reported second quarter 2026 net product revenue of $44.7 million from mitapivat (PYRUKYND/AQVESME), up from $12.5 million a year ago, driven by the U.S. AQVESME thalassemia launch and growing ex-U.S. PYRUKYND sales. Net loss was $100.7 million versus $112.0 million in 2025. R&D expenses rose to $100.8 million, reflecting a $25 million upfront payment to license cevidoplenib for immune thrombocytopenia; SG&A increased to $51.5 million on launch activities.
According to Agios, the FDA granted Priority Review to the mitapivat sNDA in sickle cell disease with a November 1, 2026 PDUFA date, and the EC approved PYRUKYND for a broad adult thalassemia population. The company advanced AG-236 into a planned Phase 2/3 program in polycythemia vera, initiated a Phase 1b trial of AG-181 in PKU, and discontinued tebapivat in LR-MDS and sickle cell disease. Cash, cash equivalents and marketable securities were $964.8 million as of June 30, 2026.
Agios (Nasdaq: AGIO) reported topline Phase 2 results for tebapivat, an oral pyruvate kinase (PK) activator, in adults with sickle cell disease. In the 12-week, double-blind, placebo-controlled trial (n=59), participants were randomized 2:2:2:1 to tebapivat 2.5 mg, 5.0 mg, 7.5 mg once daily or placebo. The primary endpoint, hemoglobin response ≥1.0 g/dL from Weeks 10–12 versus baseline, was achieved by 43.8% (2.5 mg), 47.1% (5.0 mg), 29.4% (7.5 mg) and 33.3% (placebo). Safety and tolerability were consistent with prior sickle cell trials.
Because the data did not show a sufficiently differentiated profile versus other PK activators, Agios decided not to continue tebapivat development in sickle cell disease. The company remains focused on mitapivat, its foundational PK activator, which is under FDA Priority Review via an sNDA for sickle cell disease, with a PDUFA goal date of November 1, 2026.
Agios (Nasdaq: AGIO) announced that the U.S. FDA has accepted its supplemental New Drug Application for mitapivat in sickle cell disease and granted Priority Review under the accelerated approval pathway.
The FDA’s PDUFA goal date is November 1, 2026. The sNDA is supported by the global Phase 2 and 3 RISE UP trials in patients ≥16 years and more than 1,300 patient‑years of clinical experience. Mitapivat is already approved in the U.S. for PK deficiency (2022) and thalassemia (2025).
Agios (Nasdaq:AGIO) will host a conference call and live webcast on July 30, 2026 at 8:00 a.m. ET to discuss its second quarter 2026 financial results and business highlights.
The webcast will be available in the Investors section of the company website, with a replay accessible about two hours after the event.
Agios (Nasdaq: AGIO) presented detailed 52-week Phase 3 RISE UP results for oral PK activator mitapivat in sickle cell disease at EHA 2026.
Mitapivat showed statistically significant hemoglobin responses, clinically meaningful reductions in transfusion burden and healthcare utilization for hemoglobin responders, and a safety profile similar to placebo with no treatment-related deaths.
Agios (Nasdaq: AGIO) signed an exclusive global license with Oscotec for cevidoplenib, a next‑generation oral SYK inhibitor for immune thrombocytopenia (ITP), with up to $1 billion peak U.S. sales potential. Oscotec receives $25 million upfront, up to $140 million in milestones, plus tiered royalties.
Cevidoplenib showed clinically meaningful platelet responses and was well tolerated in a Phase 2 ITP trial, despite the primary endpoint not reaching statistical significance. Agios plans to start Phase 3 in 1H 2028 and keeps 2026 operating expense guidance roughly flat versus 2025, excluding the upfront payment.
Agios (Nasdaq: AGIO) reported Phase 2b results for tebapivat in lower-risk myelodysplastic syndromes (LR-MDS). The trial did not meet the predefined threshold for transfusion independence, and Agios will not advance tebapivat in LR-MDS, though it remained well tolerated with no new safety signals observed.
Agios (Nasdaq: AGIO) received European Commission marketing authorisation for PYRUKYND (mitapivat), an oral pyruvate kinase activator, to treat anaemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia, with orphan designation.
Under an exclusive agreement, Avanzanite will commercialise and distribute PYRUKYND across the EEA, UK and Switzerland, leveraging its pan-European rare disease platform.