Long term treatment evidence from ENESTop and ENESTfreedom studies of Tasigna for Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase (CP).- Novartis.

New Novartis data from two long-term Treatment-free Remission (TFR) studies of Tasigna (nilotinib) in patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase (CP) will be presented during the 54th Annual Meeting of the American Society of Clinical Oncology (ASCO) in Chicago. Results from the open-label Phase II trials, ENESTop and ENESTfreedom , show sustained TFR in patients treated with both front-line and second-line Tasigna (nilotinib) therapy. The 144-week trials evaluate the potential to maintain molecular response (MR) after stopping therapy in eligible adult patients with Ph+ CML-CP.

"Treatment-free Remission is a new treatment goal in CML," said François-Xavier Mahon, Cancer Center of Bordeaux, Institut Bergonié and lead investigator of ENESTop. "Clinical studies like ENESTop and ENESTfreedom offer evidence that when a Ph+ CML-CP patient achieves a deep molecular response with Tasigna, along with other eligibility criteria, s/he can attempt TFR and have a nearly 50% chance of remaining treatment-free long-term. These results confirm an exciting opportunity for eligible patients - the opportunity to reduce time on drug for a chronic leukemia."

Data from ENESTop presented in an oral session (Abstract #7003) show that approximately half (48.4%; CI 95%, 39.4%-57.5%) of patients with Ph+ CML-CP who are eligible to stop second-line Tasigna therapy maintained disease remission over a prolonged period of time in the absence of treatment at 144 weeks of follow up, almost 3 years. Patients in this trial took Tasigna following a switch from Glivec (imatinib). ENESTop data also show that of the patients who restarted Tasigna due to loss of major molecular response (MMR=BCR-ABL/ABL <=0.1% IS), during the study period, nearly all (97.1%) regained MMR and 95.8% regained MR4. (BCR-ABL1 IS =< 0.0032%). Study authors stress that frequent scheduled and compliant monitoring is necessary to assess for loss of response. Results of ENESTop at 144-weeks are consistent with previously reported data at both 96- and 48-weeks.

A second long-term clinical trial, ENESTfreedom , is also part of the ASCO Scientific Program . The authors will report on TFR results at 144 weeks in patients who started front-line CML therapy with Tasigna. Results from ENESTfreedom will be shared with ASCO attendees on Monday, June 4 (Abstract #7063). In this trial, researchers found that almost half (46.8%; CI 95%: 39.6%-54.2%) of Ph+ CML-CP patients eligible to stop Tasigna treatment remained in MMR following treatment discontinuation. An update on the Phase III clinical trial design for Novartis' investigational BCR-ABL1 inhibitor, asciminib, will also be presented as part of the ASCO Scientific Program (Abstract #TPS7081).