Agio’s Investors Disappointed With Data on Leukemia Drug Ivosidenib


In addition to data that will support the filing of IDH1 inhibitor invosidenib in refractory acute myeloid leukemia (AML), Agois is set to release data on first-line combination for presentation at the upcoming American Society of Hematology (ASH) conference. Agois has continued to say that it is on pace to file ivosidenib for refractory AML before the end of the year and has provided a peek at the data it will use to go along with the application at ASH in the form of two abstracts drawn from a phase 1 dose-escalation trial of the drug.

This includes data from the combinations of ivosidenib and its IDH2 inhibitor Idhifa, which is licensed to Celgene and approved in August, adding to its current first-line therapies for AML that could unlock a much larger market for the two drugs. Agios CEO David Schenkein commented on the biotech’s third quarter results, ““Early data from these phase 1 trials reinforce ongoing efforts at Agios and Celgene to advance the IDH inhibitors into the newly diagnosed setting.”

The company was satisfied about all of the results on the call, however investors seemed unsure as shares in the company fell almost 4% on the announcement on high-volume trading, bucking the general trend among other biotechs trumpeting their ASH data.

Firstly, the trial of 258 refractory AML patients with IDH1 mutations, accounting for around 6% to 10% of AML cases, Agios’ drug showed a complete remission rate including complete hematologic recovery of 21.6%, with 8.8% seeing a complete remission with partial hematologic recovery, giving an overall response rate of only 30%. Agios will provide investors additional data at ASH on secondary measures such as transfusion independence and overall survival, according to their Chief Medical Officer Chris Bowden.

The test results from newly diagnosed AML patients with IDH1 or 2 mutations, a group which collectively accounts for around 15% to 20% of all AML patients, revealed that adding ivosidenib or Idhifa to induction chemotherapy was generally well-tolerated. The overall response rate for ivosidenib in primary AML patients was 86% and 44% among patients whose AML secondary to another blood-related disorder such as myelodysplastic syndrome.

Bowden commented on the results, ““The early data is encouraging and validate our existing and planned late stage efforts in the front-line setting.” Bowden also suggested that the company should be able to give more information on the path toward regulatory approval in front-line AML at an investor event on Dec. 11.


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