Agios spotlights promising data for IDH1 drug, putting it on the path to FDA submission

Agios today played a few of the cards it plans to show the FDA later this year when it files for an approval of its IDH1 drug ivosidenib.

Just a few months since the FDA handed out an OK for the IDH2 drug Idhifa, which Celgene largely controls, Agios $AGIO says in its ASH abstract that the first group of 125 patients in their pivotal study of acute myeloid leukemia demonstrated a complete response rate of 21.6%, with 8.8% seeing a CR with partial hematologic recovery.

That combined response rate, if it holds up for Agios during a more in-depth dive at ASH in December, could set up an approval that could be worth hundreds of millions in annual sales, according to some of the analysts who follow the company.

The biotech’s shares, though, slid 4% this morning.

Michael Schmidt

Celgene gave back rights to the drug in rejigging its deal with Agios, which may now be on track to a key approval that would get it directly involved in marketing the drug.

Back in August Leerink’s Michael Schmidt noted:

The IDH1 drug represented “a more significant value driver for the company. AGIO’s IDH1 & 2 inhibitors address a ~$270M and ~$370M global market opportunity in relapsed/refractory AML, respectively, based on our updated estimates; potential front-line use in combination with approved or investigational agents reflects a significantly larger $1Bn+ commercial opportunity based on our recent due diligence should ongoing and planned front-line clinical trials succeed.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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