Novartis’ Phase III trial investigating Scemblix (asciminib) as a first-line therapy in Philadelphia chromosome-positive chronic myeloid leukaemia (CML) has met its primary endpoints.

The two primary endpoints compared the major molecular response (MMR) rate of Scemblix to investigator-selected tyrosine kinase inhibitors (TKI), such as imatinib, nilotinib, dasatinib, or bosutinib, and compared Scemblix’s efficacy to imatinib alone at week 48.

Novartis said that Scemblix demonstrated clinically meaningful and statistically significant results on both primary endpoints. However, the company has not yet released a detailed analysis.

Scemblix-treated patients suffered fewer adverse events (AEs) and treatment discontinuations compared to investigator-selected TKIs. There were no new safety signals identified.

The study remains ongoing with a key secondary endpoint of the proportion of patients that achieve MMR at week 96 and a safety endpoint of discontinuation of study treatment due to an AE (TTDAE) by week 96.

The study is also investigating other endpoints including MR4, MR4.5, complete haematological response (CHR), time to treatment failure, event-free survival, failure-free survival, progression-free survival, and overall survival.

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By GlobalData

South Australian Health and Medical Research Institute professor Dr Tim Hughes said: “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.”

The next scheduled data readout is planned for week 96. Details will be presented at an upcoming medical conference and included as part of regulatory submissions in 2024.

ASC4FIRST (NCT04971226) is a Phase III, global, head-to-head, multi-centre, open-label, randomised study of oral Scemblix 80mg versus investigator-selected first-generation or second-generation TKIs.

The trial enrolled 405 adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukaemia at the chronic phase (Ph+ CML-CP).

Scemblix is already approved for CML

Scemblix targets the ABL myristoyl pocket 15,16 and was designed to be highly specific and minimise off-target kinase-mediated effects, which translates into an improved safety and tolerability profile compared to the current standard of care.

Scemblix is approved in more than 60 countries, having been given US Food and Drug Administration approval in 2021. The treatment is approved to treat adults with Ph+ CML-CP who have previously been treated with two or more TKIs. In some countries, including the US, Scemblix is also approved in patients with Ph+ CML-CP with the T315I mutation.

According to GlobalData’s Pharmaceutical Intelligence Centre, Scemblix has a predicted global sales forecast of $1.6bn in 2029. Novartis has registered 30 trials for Scemblix, two planned, 20 ongoing and eight completed.

GlobalData is the parent company of Clinical Trials Arena.