Novartis on Monday announced positive results in Phase III trial comparing Scemblix (asciminib) with conventional treatment in patients with chronic myeloid leukemia.
According to the company’s statement, ASC4FIRST is the first and only randomized head-to-head Phase III trial comparing a CML treatment vs. approved standard-of-care first- and second-generation TKIs.
“The trial met both primary endpoints of major molecular response (MMR) rate for Scemblix compared to investigator-selected TKIs (imatinib, nilotinib, dasatinib, and bosutinib) and compared to imatinib, demonstrating clinically meaningful and statistically significant results for both endpoints1. Scemblix showed a favorable safety and tolerability profile with fewer adverse events (AEs) and treatment discontinuations vs. investigator-selected standard-of-care TKIs,” the company said in a statement.
The ASC4FIRST data showed no new safety signals compared to the established safety profile of Scemblix.
“We are very encouraged by these results given that a significant proportion of patients with newly diagnosed chronic myeloid leukemia, or CML, do not achieve their treatment goals,” said Prof. Tim Hughes, MD, South Australian Health & Medical Research Institute (SAHMRI). “There remains a significant need in first-line therapy of CML for tolerable treatment options, allowing people with CML to balance their treatment alongside their quality of life.”
Improvements in treatment have transformed CML into a chronic disease, with a life expectancy similar to that of the general population, making tolerability an important treatment goal9. While many patients with CML may benefit from available TKI therapy, intolerance and AEs remain a primary reason for TKI therapy discontinuation, with discontinuation rates due to AEs of up to 25% by five years. Additionally, over 60% of newly diagnosed CML patients fail to meet 12-month molecular response goals.
“We are excited that Scemblix may help people newly diagnosed with CML achieve their treatment goals while continuing to live their lives,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “Given the chronic nature of their condition, patients often need to be on TKI therapy for many years, so treatment options that are well tolerated and highly efficacious are crucial to support adherence. This study outcome builds on our 20-year legacy in CML innovation as we strive to continue to address the remaining unmet needs for people living with this blood cancer.”
The trial remains ongoing, with the next scheduled data readout planned for week 96, which will evaluate the key secondary endpoint (MMR at week 96) as well as additional secondary endpoints.
Details will be presented at an upcoming medical conference and included as part of regulatory submissions in 2024, the company stated.
