Agios Pharmaceuticals Inc.

AGIO

Held by 8 specialist biotech funds

High Convergence

8 funds opened new positions. Next phase2 readout (AG-348): Aug 2020. Short interest: 11.8% of float.

BiotechnologyCEO: Brian GoffIPO: 2013Website ↗

Agios Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of medicines in the field of cellular metabolism and adjacent areas of biology. The company offers PYRUKYND (mitapivat) an activator of both wild-type and a variety of mutant pyruvate kinase, PK, enzymes for the treatment of hemolytic anemias; and AG-946 that is in Phase I clinical study for treating hemolytic anemias and other indications. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Catalysts

Phase 2PositiveQ3 2020AG-348ThalassemiaNCT03692052

Primary: Percentage of Participants Achieving a Hemoglobin Response (HR) (Up to 12 weeks)Enrollment:20/20Sites: 4NA, TREATMENT

Phase 3PositiveQ4 2023Placebo Matching MitapivatNon-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-ThalassemiaNCT04770753

Phase 3Q2 2024Placebo Matching MitapivatTransfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-ThalassemiaNCT04770779

Phase 3Q2 2024MitapivatPediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic AnemiaNCT05144256

Primary: Percentage of Participants Achieving Transfusion Reduction Response (TRR) (Week 9 to Week 32)Enrollment:49/49Sites: 22RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3Q4 2024MitapivatPediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic AnemiaNCT05175105

Primary: Percentage of Participants Achieving a Hemoglobin (Hb) Response (Baseline up to Week 20)Enrollment:30/30Sites: 19RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3Q4 2025MitapivatSickle Cell DiseaseNCT05031780

Phase 2Q1 2026TebapivatMyelodysplastic SyndromesNCT05490446

Primary: Phase 2a: Proportion of Participants With Hemoglobin (Hb) Response (Baseline, Week 8 through Week 16)Enrollment:87/87Sites: 45NON_RANDOMIZED, TREATMENT

Phase 2Q1 2026AG-120Chondrosarcoma, Chondrosarcoma, Grade 2, Chondrosarcoma, Grade 3NCT04278781

Phase 2Q2 2026TebapivatSickle Cell DiseaseNCT06924970

Phase 2Jun 30, 2026MitapivatAnemiaNCT07055243

Phase 3Q1 2029MitapivatNon-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-ThalassemiaNCT07517133

Primary: Percentage of Participants Who Achieved a Hemoglobin (Hb) Response (Baseline, Week 12 through Week 24)Enrollment:Target 45RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3Q2 2029Mitapivat Matched PlaceboTransfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-ThalassemiaNCT07506863

Primary: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48 (Baseline, through Week 48)Enrollment:Target 54RANDOMIZED, QUADRUPLE, TREATMENT

Agios Pharmaceuticals Inc.

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