Agios reports positive mitapivat data in sickle cell disease

Agios Pharmaceuticals (NASDAQ:AGIO) announces that clinical proof-of-concept has been established based on a preliminary analysis in Phase 1 trial of mitapivat (AG-348), an oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R enzymes in patients with sickle cell disease.

Mitapivat has been shown to decrease 2,3-diphosphoglycerate and increase adenosine triphosphate, and through this mechanism, it may reduce hemoglobin S polymerization and red blood cell sickling.

The study has enrolled nine patients to date. Seven of eight (88%) of patients experienced a hemoglobin increase, with five of eight patients (63%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline (range 1.0-2.7 g/dL).

Adverse events reported were generally consistent with those previously reported in pyruvate kinase deficiency or that are to be expected in the context of sickle cell disease.

Pharmacodynamics and biomarker data support mitapivat’s proposed mechanism of action.

The company is focused on advancing mitapivat to pivotal development, with the goal of initiating a pivotal study next year.

https://seekingalpha.com/news/3582535-agios-reports-positive-mitapivat-data-in-sickle-cell-disease