Scemblix Shows Superior Results Versus Other TKIs in Some With CML

Treatment with Scemblix demonstrated superior results versus treatment with standard-of-care TKIs in some patients with chronic myeloid leukemia.

Treatment with Scemblix (asciminib) demonstrated positive outcomes in newly diagnosed patients with Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase, according to a news release from Novartis, the drug’s manufacturer.

Scemblix is a type of tyrosine kinase inhibitor (TKI) that blocks the BCR-ABL fusion protein, which can help destroy cancer cells and prevent them from growing any further, according to the National Cancer Institute.

The Philadelphia chromosome is an abnormal version of chromosome 22, which includes the fusion of the ABL gene and BCR gene. This gene fusion leads to the uncontrollable growth of immature white blood cells, which can build up in the blood and bone marrow, as defined by the National Cancer Institute.

A randomized phase 3 trial called ASC4FIRST included 405 newly diagnosed patients with Philadelphia chromosome-positive CML in chronic phase; patients were randomly assigned to receive either Scemblix or a TKI that was chosen by the investigators of the study as standard-of-care, the news release reported.

The trial’s primary endpoint (main result measured at the end of a study to see if treatment worked) included evaluating the efficacy of Scemblix compared with the investigator-selected TKI. Another primary endpoint included in the trial determined the efficacy of Scemblix compared with patients receiving the Gleevec (imatinib) TKI.

Both primary endpoints evaluated the major molecular response (MMR; the amount of the abnormal BCR-ABL gene in a patient’s blood or bone marrow is 0.1% or less than baseline) in patients at 48 weeks.

The trial met both of its primary endpoints, which demonstrated statistically meaningful results, according to the news release.

“We are very encouraged by these results given that a significant proportion of patients with newly diagnosed chronic myeloid leukemia, or CML, do not achieve their treatment goals,” Dr. Tim Hughes, South Australian Health & Medical Research Institute, said in the news release. “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.”

READ MORE: Scemblix Shows Greater Efficacy, Safety For Patients With Chronic Phase CML

In terms of safety, the news release noted that treatment with Scemblix showed a favorable safety and tolerability profile with fewer side effects. It was reported that there were fewer treatment discontinuations with Scemblix when compared with the investigator-selected standard-of-care TKIs.

According to the news release, the trial is on track to continue and will soon evaluate the secondary endpoints of the trial, including the MMR at 96 weeks. The trial’s researchers also aim to evaluate a safety endpoint regarding treatment discontinuation due to side effects by week 96.

“We are excited that Scemblix may help people newly diagnosed with CML achieve their treatment goals while continuing to live their lives,” Dr. Shreeram Aradhye, president, development and chief medical officer, Novartis, said in the news release. “Given the chronic nature of their condition, patients often need to be on TKI therapy for many years, so treatment options that are well tolerated and highly efficacious are crucial to support adherence. This study outcome builds on our 20-year legacy in CML innovation as we strive to continue to address the remaining unmet needs for people living with this blood cancer.”

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