viernes, 12 de junio de 2020

Agios drug shows strong response in patients with inherited blood disorder

Agios drug shows strong response in patients with inherited blood disorder

The Readout

Damian Garde & Meghana Keshavan

Agios bided its time — and has data to show for it

Agios Pharmaceuticals has been picked on by some investors for helping small numbers of patients while ignoring more profitable business opportunities. New data released early today should help quiet some of that criticism.

Data on Agios’ lead pipeline drug — one targeted at patients with two different types of thalassemia — reported positive results from its first clinical trial at the European Hematology Association meeting. While preliminary, they could set up a pivotal Phase 3 studies starting next year — one that, if successful, turn the drug, called mitapivat, into a blockbuster.

The data “show that mitapivat can address larger patient populations that still have unmet medical needs even with other therapies coming in,” said Chris Bowden, Agios’ chief medical officer.

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