Promising responses seen
with Agios leukemia drug in study
Send a link to a friend
[December 12, 2017] By
Bill Berkrot
(Reuters) - Nearly a third of patients with
an advanced form of a fast-progressing leukemia who carry a specific
genetic mutation experienced a complete or near complete response to an
experimental Agios Pharmaceuticals drug, according to data from an early
stage trial released on Monday.
|
The study tested Agios' ivosidenib, a once-a-day pill, in patients
with an IDH1 gene mutation who had relapsed or were not helped by
prior treatment for acute myeloid leukemia (AML).
Of 125 patients available for evaluation in the expansion of a Phase
I study, 30.4 percent experienced a complete response or a complete
response with partial hematologic recovery (CRh), researchers
reported. Both represent no detectable sign of the cancer, but with
the latter category blood counts have not fully returned to normal.
The complete response rate was 21.6 percent, while 8.8 percent had a
CRh at the time of data readout. The overall response rate was 41.6
percent, according to data unveiled at the American Society of
Hematology conference in Atlanta.
The median duration of response was 9.3 months for patients who
achieved a complete response. That fell to 6.5 months when including
all responders.
This new data "is compelling and demonstrates impressive
single-agent efficacy with durable responses in these high-risk
patients," Dr. Courtney DiNardo, the study's lead investigator from
MD Anderson Cancer Center in Houston, said in a statement.
Median overall survival data was not yet available for patients who
achieved complete response or CRh, meaning more than half of those
patients were still alive at the time of data cutoff. Median overall
survival was 9.3 months for the partial responders versus just 3.9
months for those not helped by the drug.
Agios, which holds full rights to ivosidenib, said it plans to use
the early data to seek swift approval of the medicine, a similar
path that led to the August U.S. approval of Idhifa, which Agios
shares with Celgene Corp..
[to top of second column] |

Idhifa is approved to treat relapsed AML in patients with a similar
genetic mutation known as IDH2. Combined, IDH1 and IDH2 mutations
account for about 20 percent of all AML cases.

The vast majority of AML patients do not respond to chemotherapy and
need new treatments for the rapidly progressing deadly blood cancer.
Serious but not fatal side effects reported in 8 percent of
ivosidenib patients included above normal white blood cell counts
and QT elongation, a type of heart arrhythmia.
(Reporting by Bill Berkrot; Editing by Steve Orlofsky)
[© 2017 Thomson Reuters. All rights
reserved.] Copyright 2017 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed. |