Novartis is hoping that Scemblix (asciminib) will become the physician’s first choice of therapy for chronic myeloid leukaemia in chronic phase (CML-CP) after it has shown to be more tolerable than the standard of care (SOC) in a Phase IIIb trial.
Scemblix is a first-in-class STAMP inhibitor which received accelerated approval by the US Food and Drug Administration (FDA) in 2021 in previously treated CML in adult patients. On 29 October 2024, the FDA granted accelerated approval to the drug as a first-line therapy in CML-CP.
Interim data presented at the American Society of Clinical Oncology (ASCO) 2025 meeting, held from 30 May to 3 June in Chicago, from the ongoing ASC4START (NCT05456191) study showed the drug met its primary endpoint of superior tolerability.
In the study, 568 patients were randomised to receive either Scemblix 80mg daily or nilotinib 300mg twice daily, a SOC tyrosine kinase inhibitor (TKI), with the primary endpoint looking at time to treatment discontinuation due to adverse events (TTDAE), including deaths to AEs.
Patients were evaluated for an average of 9.7 months with this preliminary data cutoff happening after 50 trial-reported AEs. At this cutoff, 10.9% of patients on Scemblix had discontinued treatment compared with 17.3% of patients on nilotinib, achieving the primary endpoint with a cause-specific hazard ratio of 0.45.
Novartis added that the study is ongoing with further analyses planned for tolerability and efficacy of Scemblix with a 65-event cut-off. It follows the ASC4FIRST study (NCT04971226) for efficacy in which Scemblix outperformed SOC TKI with a major molecular response (MMR) rate of 74.1% at week 96 compared with 52% for those on SOC TKIs after 96 weeks of treatment.
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By GlobalDataThe company hopes this additional tolerability data will support physician preference for Scemblix as a first-line therapy for patients with CML-CP. While the data may convince physicians, its ability to become SOC will depend on the agencies and payers said Dr. Andreas Hochhaus, principal investigator and director of the University Tumor Center (UTC) at the Jena University Hospital, Germany.
Speaking to Clinical Trials Arena, Hochhaus said: “We did this study to convince the agencies in different countries for reimbursement because SOC means you need reimbursement, and it’s important to show all kinds of data and that means efficacy, but also tolerability. In CML, we have improved survival almost to the survival expectation of the normal population. Therefore, this cannot be improved anymore, so you need other endpoints such as quality of life and AEs..”
