Novartis Presents New Data from Large European Study Reinforcing the Benefit of First-Line Tasigna in Newly-Diagnosed Patients with CML

Novartis has announced results from the Phase IIIb ENEST1st study in over 1000 patients with newly-diagnosed, BCR-ABL positive chronic myeloid leukemia (CML), confirming the benefits of first-line Tasigna treatment seen in earlier trials. The final results of this large study, conducted in 26 European countries, were presented at the 20th Congress of the European Hematology Association (EHA) in Vienna. 

"These results show patients with Bmyeloid leukemia, Tasigna, CR-ABL positive CML in chronic phase receiving first-line treatment with Tasigna achieved rapid and high rates of molecular response and had a very low rate of progression to advanced disease," said Dr Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology, Jena University Hospital, Germany. "These findings from 26 countries, which are a collaboration of 307 trial sites and 14 standardized laboratories monitoring the incidence of MR4.0 as primary endpoint, confirm and complement data from the pivotal ENESTnd trial."

The primary endpoint in ENEST1st was the rate of Molecular Response 4 (MR4.0) at 18 months. MR4.0, which is a 4 log reduction in BCR-ABL, represents a very low level of detectable BCR-ABL, the cause of Ph+ CML (measured as BCR-ABL <=0.01% on the International Scale [BCR-ABLIS] or undetectable BCR-ABL in cDNA with >=10,000 ABL transcripts).

At 18 months, 38.4% of Tasigna-treated patients (n=1,052) reached MR4.0. These data demonstrate high rates of early and deep molecular response with Tasigna. The rate of disease progression in the study was low, with six patients, or 0.6%, advancing to the accelerated phase/blast crisis stage of the disease. Despite the higher median age of patients in ENEST1st than in previous Tasigna studies, the safety results were consistent with the known safety profile of Tasigna. The most common adverse events (AE) were rash, itch and headache. Grade 3/4 AEs related to hepatotoxicity and pancreatitis occurred in 0.4% and 0.6% of patients, respectively. Grade 3/4 thrombocytopenia (low blood platelet count) and neutropenia (low white blood cell count) occurred in 6.0% and 4.8% of patients, respectively.

"Our commitment to CML remains strong and is exemplified by this trial, as well as further study of deep molecular response with Tasigna and the possibility for some patients with Ph+ CML to stop their treatment and achieve sustained treatment-free remission," said Bruno Strigini, President, Novartis Oncology. "In addition, we are developing new compounds with different mechanisms of action, which could help address the resistance to existing medications that some patients experience."