About the RATIFY trial
RATIFY (Randomized AML Trial In FLT3 in patients 0.7) and ITD low allelic mutation fraction (0.05-0.7). All three subtypes treated with PKC412 (midostaurin) demonstrated improved OS versus placebo. Allogeneic hematopoietic stem cell transplantation (SCT) was allowed. PKC412 (midostaurin) benefited patients regardless of whether they went on to receive a SCT.
No statistically significant differences were observed in the overall rate of grade 3 or higher hematologic and non-hematologic adverse events (AEs) . A total of 37 deaths were reported, with no difference in treatment-related deaths observed between groups.
"The RATIFY study, in partnership with the Alliance for Clinical Trials in Oncology, reflects our relentless pursuit to develop targeted therapies that can improve and extend people's lives," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "Based on the results of this trial, we plan to move forward with global regulatory submissions for PKC412 (midostaurin) in the first half of 2016."
In order to help identify patients who may have a FLT3 mutation and potentially benefit from treatment with PKC412 (midostaurin), Novartis is collaborating with Invivoscribe Technologies, Inc. who will lead regulatory submissions for a companion diagnostic.
About the RATIFY trial
RATIFY (Randomized AML Trial In FLT3 in patients
DEC 06, 2015
Study in partnership with the Alliance for Clinical Trials in Oncology is the first large controlled trial to show overall survival benefit in FLT3-mutated AML
AML is the most common acute leukemia in adults, but has the lowest survival rate; no change in treatment strategy in more than 25 years,
Currently there are no approved targeted AML treatments; worldwide regulatory submissions for PKC412 (midostaurin) to begin in 2016
Basel, December 6, 2015 - Novartis today announced positive results from the global Phase III RATIFY (CALGB 10603) clinical trial. In the study, adult patients under 60 years of age with newly-diagnosed FLT3-mutated acute myeloid leukemia (AML) who received the investigational compound PKC412 (midostaurin) plus standard induction and consolidation chemotherapy experienced a 23% improvement in overall survival (OS) (hazard ratio [HR] = 0.77, P = 0.0074) compared to those treated with standard induction and consolidation chemotherapy alone. The median OS for patients in the PKC412 (midostaurin) treatment group was 74.7 months (95% confidence interval [CI]: 31.7, not attained), versus 25.6 months (95% CI: 18.6, 42.9) for patients in the placebo group.
The trial evaluated the addition of either PKC412 (midostaurin) or placebo to daunorubicin/cytarabine in the induction phase, followed by high-dose cytarabine in the consolidation phase; patients who achieved complete remission after consolidation chemotherapy continued treatment with PKC412 (midostaurin) or placebo as a single agent for up to one year.
The data, collected and analyzed in partnership with the Alliance for Clinical Trials in Oncology, are from the largest clinical trial in FLT3-mutated AML to date, with 3,279 patients screened and 717 study participants from around the world. Results will be presented today at the 57th Americ