Novartis reports long-term data from the ENESTfreedom and ENESTop trials of Tasigna for Ph+ chronic myeloid leukemia

Long-term follow-up data from the ongoing, pivotal open-label ENESTfreedom and ENESTop trials showed sustained treatment-free remission (TFR) after stopping frontline and second-line Tasigna (nilotinib) therapy in eligible adult patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase (CP). Separate data demonstrate promising results for asciminib (ABL001), an investigational allosteric BCR-ABL inhibitor, in combination with three different tyrosine kinase inhibitors (TKIs) in heavily pre-treated Ph+ CML-CP patients .

The results will be presented at the 24th Congress of the European Hematology Association (EHA) in Amsterdam-. "We are pleased to report many of our Tasigna clinical-trial patients continue to maintain treatment-free remission for nearly four years with a low adverse event burden," said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis. "Long-term trials like ENESTfreedom and ENESTop, as well as promising Phase I data from asciminib, are helping us to reimagine medicine and the way CML is treated."

Results from the ENESTfreedom study showed that about 44% of patients remained in TFR (84/190) for 192 weeks after stopping frontline Tasigna treatment. The treatment-free survival rate at 192 weeks was nearly 49%. About 99% (90/91) and 92% (84/91) of patients who resumed nilotinib due to loss of major molecular response (MMR) during the TFR phase regained MMR and molecular response, respectively. Among 91 patients who resumed nilotinib, the most common adverse events (AEs) were nasopharyngitis (18.7%) as well as pruritus, fatigue and increased lipase (14.3% each). The majority of AEs were grade 1/21.

Consistent results were observed in the ENESTop trial: About 46% of patients remained in TFR (58/126) for 192 weeks after stopping second-line Tasigna treatment. The treatment-free survival rate at 192 weeks was over 50%. Among 59 patients who resumed nilotinib, the most common AEs were hypertension (20.3%) and arthralgia (13.6%). The majority of AEs were grade 1/22.

Novartis will also present data from a Phase I trial of asciminib in combination with ATP-competitive TKI in heavily-pretreated patients with Ph+ CML-CP.