Servier Announces Phase 3 Clinical Success of Ivosidenib in Combination with Azacitidine as First-Line Therapy for IDH1-Mutated AML

Recently, Servier announced that it will present the results of a Phase 3 clinical trial at the American Society of Hematology (ASH) Annual Meeting. The trial evaluates Tibsovo (ivosidenib), an oral selective IDH1 inhibitor, in combination with azacitidine as a first-line treatment for acute myeloid leukemia (AML) patients with IDH1 mutations. The combination of Tibsovo and azacitidine met the primary endpoint of the trial, significantly extending patients' event-free survival (EFS) compared to placebo plus azacitidine (HR=0.33 [95% CI 0.16, 0.69]; P=0.0011).

In addition, the combination of Tibsovo and azacitidine also reached the secondary endpoints of overall survival (OS) and complete remission rate (CR). The median OS in the Tibsovo/azacitidine group was 24.0 months, compared to 7.9 months in the control group (HR=0.44, p=0.0005). The CR in the Tibsovo/azacitidine group was 47.2%, compared to 14.9% in the control group (p<0.0001).

AML is a blood and bone marrow cancer characterized by rapid disease progression and is the most common acute leukemia affecting adults. Most AML patients eventually relapse, and the prognosis for relapsed/refractory AML is poor, with a 5-year survival rate of approximately 27%. In 6% to 10% of AML patients, mutations in the IDH1 enzyme block normal hematopoietic stem cell differentiation, increasing the risk of acute leukemia.

Ivosidenib is an orally available, selective small molecule IDH1 inhibitor initially developed by Agios Pharmaceuticals. It has previously been approved by the U.S. FDA as a monotherapy for adult patients with relapsed/refractory AML carrying IDH1 mutations and for untreated IDH1-mutated AML adult patients who are unable to undergo intensive induction chemotherapy due to being aged ≥75 years or having comorbidities. In August this year, it received FDA approval for the treatment of cholangiocarcinoma. In China, CStone Pharmaceuticals has submitted its new drug application, which has also been included in the proposed priority review for the treatment of adult patients with relapsed or refractory AML carrying IDH1 mutations.

References:

[1] New Data at ASH 2021 Reinforces the Strength of Servier's Hematology Portfolio. Retrieved November 7, 2021, from https://www.prnewswire.com/news-releases/new-data-at-ash-2021-reinforces-the-strength-of-serviers-hematology-portfolio-301417135.html

(Original text has been abridged)

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