Company: AGIO
Filing Date: 2025-10-30
Form Type: 10-Q
Source: 0001439222-25-000116
Chunk: 69

Company: AGIOS PHARMACEUTICALS, INC.
Filing Date: 2025-10-30
Form: 10-Q
Item: Part II, Item 1A
Chunk 69
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 Merck and Bristol-Myers Squibb Company, or BMS, are marketing a therapy to treat beta thalassemia and LR MDS, and are conducting clinical trials for alpha thalassemia and LR MDS patients that are erythropoiesis-stimulating agent naïve and non-transfusion dependent; Geron Corporation recently announced FDA approval of a treatment for adults with LR MDS with transfusion-dependent anemia; Takeda Pharmaceutical Company Limited, or Takeda with Keros Therapeutics, Inc. are developing a therapy to treat LR-MDS; Novartis International AG is marketing a therapy and has another molecule in clinical trials to treat SCD; Emmaus Life Sciences is marketing a therapy to treat SCD; BioMarin Pharmaceutical Inc. is marketing and conducting clinical trials for therapies to treat PKU; PTC Therapeutics, Inc. has a recently approved treatment for PKU; Pfizer Inc., CSL Behring, and Cellarity, Inc. are conducting clinical trials for therapies in SCD; Novo Nordisk A/S is conducting clinical trials for the treatment of alpha and beta thalassemia and SCD; Sanofi is conducting clinical trials for a therapy in SCD;  Genetix Biotherapeutics Inc. (formerly bluebird bio) is marketing a gene therapy to treat transfusion-dependent beta-thalassemia and SCD; Vertex, with CRISPR, is marketing a gene therapy targeting SCD and transfusion-dependent beta-thalassemia; Disc Medicine, Inc., or Disc, is conducting a clinical trial for a potential treatment for SCD; Fulcrum Therapeutics Inc. is conducting clinical trials for a potential treatment for SCD; BMS is conducting clinical trials for a potential treatment for SCD; Otsuka Pharmaceutical Co., Ltd, NGGT Inc., and Maze Therapeutics, Inc. are conducting clinical trials for potential treatments for PKU; PharmaEssentia Corp and Incyte Corporation are marketing therapies to treat PV, and Protagonist Therapeutics with Takeda, Ionis Pharmaceuticals, Inc. with Ono Pharmaceutical Co., Ltd, Italfarmaco S.p.A., Disc, Merck & Co., Inc., GluBio Inc., and Silence Therapeutics are developing therapies to treat PV; Rocket Pharma LTD is developing a therapy for the treatment of PK deficiency; and a number of other biotechnology companies have product candidates in clinical development in similar indications as ours.

There are a variety of treatment