Novartis announced on Sunday, Dec. 6 that its Phase III RATIFY study for an acute myeloid leukemia (AML) drug called Midostaurin, showed promise in patients' survival rates.
FLT3 is a typical genetic mutation that occurs in patients with AML. Due to poor clinical outcomes, it is critical for new treatments to be developed.
Midostaurin or PKC412 is an experimental compound that halts multiple enzymes such as FLT3. Through this action, numerous important cell activities are regulated, thereby disrupting the ability of cancer cells to enlarge and spread.
Novartis conducted the largest FLT3-mutated AML clinical trial, which involves 717 study subjects aged below 60 years old from all around the world.
For the study, patients were given either midostaurin or placebo in addition to standard daunorubicin/cytarabine chemotherapy during the induction stage. In the consolidation stage, the participants were given high-dose cytarabine. After the consolidation phase, some continued to receive either midostaurin or placebo for one more year.
The findings of the trial showed that patients who received midostaurin had a 59 percent remission rate while those who received placebo had 53 percent. Median overall survival rate showed significant discrepancies between the two groups, with midostaurin patients having 74.7 months and placebo patients with 25.6 months. The same notable difference was also observed in the patients' median five-year event-free survival rate with the midostaurin and placebo groups exhibiting eight and three months survival respectively.
RATIFY trial chair Richard Stone said the results of the study is a much sought-after development for hematologist and the entire AML community.
To improve the survival rates of patients with AML, the first step is to identify patients with FLT3 mutation and can best benefit from midostaurin therapy. To achieve this, Novartis is partnering with Invivoscribe Technologies, Inc., which will head regulatory submissions.
"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," said Alessandro Riva, global head for Novartis Oncology Development and Medical Affairs.
The results of the clinical trials were presented at the 57th American Society of Hematology (ASH) Annual Meeting in Florida.
