ASCO 2024: Novartis' Scemblix Superior to Standard Care in CML

Novartis recently published encouraging findings from the Phase III ASC4FIRST clinical trial, highlighting that its tyrosine kinase inhibitor (TKI) Scemblix (asciminib) surpasses current standard of care (SoC) treatments as a first-line option for Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML).

The Phase III ASC4FIRST study (NCT04971226) was conducted as an open-label trial and its results were unveiled in a late-breaking abstract, with a detailed presentation scheduled at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, US, running until June 4.

The trial successfully achieved its primary goal, with Scemblix demonstrating a major molecular response (MMR) rate of 67.7% at week 48. This is a significant improvement over the 49% MMR rate seen with the SoC TKIs, which include Pfizer’s Bosulif (bosutinib), Bristol Myers Squibb’s Sprycel (dasatinib), and Novartis’ own Gleevec/Glivec (imatinib) and Tasigna (nilotinib). Furthermore, Scemblix showed an MMR rate of 69.3% at 48 weeks, compared to an MMR of 40.2% achieved with Gleevec.

Dr. Tim Hughes from the South Australian Health & Medical Research Institute (SAHMRI) commented on the findings, stating, “Scemblix is the first CML treatment to show significantly better efficacy compared to investigator-selected standard-of-care TKIs. When you combine superior response with the excellent safety and tolerability profile of Scemblix, we have a very promising potential frontline option for newly diagnosed patients to support them in achieving their treatment goals.”

This study also indicated that patients treated with Scemblix experienced lower rates of adverse events (AEs), specifically 38% for Grade 3 or more severe AEs. This is in contrast to the AE rates of 55% and 44% observed in the Gleevec monotherapy and SoC groups, respectively.

Scemblix functions by inhibiting a BCR-ABL tyrosine kinase protein, which is present in CML cells, thereby halting their growth and proliferation. The medication received accelerated approval from the US Food and Drug Administration (FDA) in 2021, particularly as a third-line treatment for patients with Ph+ CML in the chronic phase.

Looking ahead, Novartis intends to submit the data from this trial to the FDA through the Oncology Center of Excellence Real-Time Oncology Review (RTOR) program. According to Novartis’ financial report, Scemblix generated $136 million in sales during the first quarter of this year. Analysts at GlobalData project that the drug could potentially reach sales of approximately $2.4 billion by 2030.

These promising results reflect not only the efficacy but also the enhanced safety profile of Scemblix, making it a potentially groundbreaking option for individuals newly diagnosed with Ph+ CML.