Scembix (ascitinib) first line for CML – pro
SCEMBLIX is indicated for the treatment of adult patients with:Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs)
Ph+ CML in CP with the T315I mutation. It was denied becasue of no T315I mutation.
However, the Phase III ASC4FIRST trial was a late-breaking abstract at the 2024 American Society of Clinical Oncology (ASCO) meeting. Scemblix® (asciminib) demonstrated superior major molecular response (MMR) rates at week 48 compared to investigator-selected standard-of-care (SoC) tyrosine kinase inhibitors (TKIs) imatinib, nilotinib, dasatinib and bosutinib, and compared to imatinib alone in patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). Scemblix also showed a numerical improvement in MMR at week 48 vs. second generation (2G) TKIs (nilotinib, dasatinib and bosutinib)1. Additionally, Scemblix demonstrated a favorable safety and tolerability profile, with fewer adverse events (AEs) and treatment discontinuations vs. both imatinib and 2G TKIs. Asciminib, a BCR::ABL1 inhibitor specifically targeting the ABL myristoyl pocket,was published in May 2025 yo offer better efficacy and safety and fewer side effects than currently available frontline ATP-competitive tyrosine kinase inhibitors (TKIs). The restriction to a particular mutation is no longer possible to maintain.
Hughes TP, Hochhaus A, Takahashi N, et al. ASC4FIRST, a pivotal ph 3 study of asciminib (ASC) vs investigator-selected tyrosine kinase inhibitors (IS TKIs) in newly diagnosed patients (pts) with chronic myeloid leukemia (CML): primary results [abstract]. Presented at: American Society for Clinical Oncology (ASCO) 2024 Annual Meeting; May 31 – June 4, 2024; Chicago, IL.
Hochhaus A, Saglio G, Hughes TP, et al. Long-term benefits and risks of frontline nilotinib vs imatinib for chronic myeloid leukemia in chronic phase: 5-year update of the randomized ENESTnd trial. Leukemia. 2016;30(5):1044-1054. doi:10.1038/leu.2016.5
Elisabetta Abruzzese,CML 25 Years Later — Poised for Another Breakthrough? N Engl J Med 2024;391:955-957
VOL. 391 NO. 10
Andreas Hochhaus, Dr.Med., Jianxiang Wang, M.D., Dong-Wook Kim, M.D., Ph.D., Dennis Dong Hwan Kim, M.D., Ph.D., Jiri Mayer, M.D., Yeow-Tee Goh, M.B., B.S., M.Med., Philipp le Coutre, M.D., Asciminib in Newly Diagnosed Chronic Myeloid Leukemia. N Engl J Med 2024;391:885-898
DOI: 10.1056/NEJMoa2400858 VOL. 391 NO. 10 May 21 2024