Swiss pharmaceutical giant Novartis (NYSE: NVS) unveiled longer-term results from the pivotal Phase III ASC4FIRST study for its Scemblix (asciminib) at the 66th American Society of Hematology (ASH) annual meeting. The study demonstrated superior major molecular response (MMR) rates for Scemblix at week 96 compared to standard-of-care (SoC) tyrosine kinase inhibitors (TKIs).
ASC4FIRST Study Design and Findings
The ASC4FIRST study compared Scemblix to investigator-selected SoC TKIs, including imatinib, nilotinib, dasatinib, and bosutinib, as well as to imatinib alone in adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). The longer-term results indicated an increasing difference in Scemblix MMR rates versus SoC, imatinib, and second-generation TKIs (2G TKIs). Over 22% more patients treated with once-daily Scemblix achieved MMR at week 96 compared to all investigator-selected SoC TKIs, and nearly 30% more patients achieved MMR at week 96 compared to imatinib alone. The Scemblix MMR rate was 15.1% higher compared to 2G TKIs, with rates of 72% versus 56.9%. Additionally, patients treated with Scemblix achieved deeper rates of molecular responses (MR4 and MR4.5) compared with investigator-selected SoC TKIs.
Scemblix’s Mechanism of Action and Global Approval Status
Scemblix is the first CML treatment that specifically targets the ABL Myristoyl Pocket, also known as a STAMP inhibitor in scientific literature. In the US, Scemblix received accelerated approval for the treatment of newly diagnosed adults with Ph+ CML-CP and is also approved for previously treated adult patients with Ph+ CML-CP. In China, the drug is awaiting regulatory decisions with its market filing accepted for review in June this year.-Fineline Info & Tech
