With Sickle Cell Results For Pyrukynd, Agios Enters Increasingly Competitive Market

The company has significantly built up its pipeline since divesting its oncology business to Servier, with multiple Phase III readouts for Pyrukynd expected in 2024-2025.

Agios announced topline Phase II results from RISE UP • Source: Shutterstock

Agios Pharmaceuticals, Inc.’s topline results from the Phase II portion of its Phase II/III RISE UP trial of Pyrukynd (mitapivat) in sickle cell disease (SCD) show potential to significantly expand the addressable market for the drug, albeit in a space that has grown increasingly competitive in the past few years. They also indicate how rapidly the company has developed its pipeline since selling off its oncology business to Les Laboratoires Servier in 2020.

Agios announced 26 June the results from the 79 patients enrolled into the first portion of the Phase II/III study,...

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