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Agios’s mitapivat meets endpoints in Phase III thalassemia trial

2024-01-04

临床3期临床结果上市批准基因疗法细胞疗法

Mitapivat is a pyruvate kinase activator and is sold as Pyrukynd for the treatment of haemolytic anaemia with pyruvate kinase deficiency. Image Credit: r.classen / Shutterstock.

Mitapivatrmaceuticals’s mitapivat met primary and two kePyrukyndary endpoints in a Phahaemolytic anaemiaial inpyruvate kinase deficiency pyruvate kinasenon-transfusion-dependent alpha- or beta-thalassemia.

Agios Pharmaceuticalss tmitapivatr regulatory approval of mitapivat as a treatment for thalassemia by the end of the year.transfusion-dependent alpha- or beta-thalassemia

The regulatory filing will also include data from the secomitapivatIII ENERGISE-T studythalassemia79) of mitapivat in adults with transfusion-dependent alpha-thalassemia or beta-thalassemia. The topline data from the trial is expected in mid-2024, as per a 3 January press release.

Mitapivat is a pyruvate kinase activator, which was approved as a treatment for haemolytic anaemia in adumitapivatpyruvate kinase transfusion-dependent alpha-thalassemiae brbeta-thalassemiad for the haemolytic anaemia indication. It generated $7.4m in net US sales in Q3 FY 2023, as per Agio’s financials.

Mitapivatt for thalassemia therapies is forecasted to grow from being worth apprhaemolytic anaemia2023 to over $4.pyruvate kinase deficiency pyruvate kinase per GlobalData analysis.Pyrukynd haemolytic anaemia Agio

GlobalData is tthalassemiaompany of Pharmaceutical Technology.

One of the major drivers for growth Pharmaceutical Technologyive gene therapies. In November 2023, CRISPR-based gene editing therapy Casgevy (exagamglogene autotemcel) developed by Vertex Pharmaceuticals and CRISPR Therapeutics was conditionally approved in the UK as a treatment for transfusion-dependent beta-thalassemia.

Phase III dataCasgevy exagamglogene autotemcel Vertex Pharmaceuticals CRISPR Therapeutics transfusion-dependent beta-thalassemia

A total of 194 patients were enrolled in the Phase III ENERGIZE trial (NCT04770753). Of these, 122 in the mitapivat arm and 62 in the placebo arm completed the 24-week double-blind period of the study.

The study’s primary endpoint was defined as an increase of ≥1g/dL in average haemoglobin concentrations frmitapivat2 through week 24 compared to baseline. This haemoglobin response was seen in 42.3% of patients in the mitapivat arm, compared to 1.6% of patients in the placebo arm.

The Mitapivat group also demonstrated statistically significant improvements in the functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) score and average haemoglobin concentration from weeks 12 to mitapivated to baseline.

The Mitapivatncidence of adverse events was similar in both arms. However, four participants in the mitapivat arm had to discontinue fatigueal due tFatiguese events.

Another therapy being developed for the treatment of thalassemia is Jasper Therapeutics’s antibody cmitapivatng therapy briquilimab. The treatment was designed to limit the toxicity of stem cell therapies. It is currently in Phase I/II trial (NCT05357482) in beta-thalassemia and sickle cell disease patients.

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