The partner of CStone Pharmaceuticals announced the publication of results from the Phase 3 AGILE trial of TIBSOVO® (ivosidenib tablets) in the New England Journal of Medicine (NEJM). The AGILE trial is a global Phase 3 double blinded placebo-controlled study in adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) comparing TIBSOVO in combination with the chemotherapy azacitidine to azacitidine in combination with placebo. The study met the primary and all key secondary endpoints including overall survival.

Servier is actively working with the FDA and health authorities across the globe to potentially bring this new indication to market, and CStone is pursuing submission for this indication in China as well. AML is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with approximately 20,000 new cases estimated in the U.S. each year. In China, there are about 75.3 thousand new cases of leukemia each year and approximately 59% are AML patients.

The majority of patients with AML eventually relapse. Relapsed or refractory AML has a poor prognosis. The five-year survival rate is approximately 29.5%.

IDH mutations are present in about 6 to 10 percent of AML cases. The data from the global Phase 3 AGILE study, showed TIBSOVO is the first IDH1 mutation specific targeted therapy to demonstrate improved event-free survival (EFS) and overall survival (OS) in combination with azacitidine compared to azacitidine plus placebo. Treatment with TIBSOVO in combination with azacitidine demonstrated a statistically significant improvement in event-free survival (hazard ratio [HR] = 0.33, 95% confidence interval [CI]: 0.16, 0.69, 1-sided P = 0.0011).

The combination of TIBSOVO with azacitidine showed a statistically significant improvement in overall survival  (HR = 0.44 [95% CI 0.27, 0.73]; 1-sided P = 0.0005), with a median OS of 24.0 months vs. 7.9 months in the placebo + azacitidine arm. In addition, complete remission (CR) rate was 47.2% (n = 34/72) for TIBSOVO in combination with azacitidine vs.

14.9% (n = 11/74) for placebo plus azacitidine (P < 0.0001). CR + CR with partial hematologic recovery rate (CR + CRh rate) was 52.8% (n = 38/72) for TIBSOVO in combination with azacitidine vs. 17.6% (n = 13/74) for placebo plus azacitidine (P < 0.0001).

Objective response rate (ORR) was 62.5% (n = 45/72) for TIBSOVO in combination with azacitidine vs. 18.9% (n = 14/74) for placebo plus azacitidine (P < 0.0001).