Scemblix® Outperforms Standard Treatments in Phase III Myeloid Leukemia Trial

Novartis' Scemblix® has shown superior efficacy and safety in treating newly diagnosed chronic myeloid leukemia (CML) compared to standard TKIs in a Phase III trial. Scemblix® achieved a nearly 20% higher major molecular response (MMR) rate at week 48 and reported fewer adverse events, offering a promising frontline treatment option.

Novartis has announced that its drug Scemblix® (asciminib) has demonstrated superior efficacy and a favorable safety profile in the treatment of chronic myeloid leukemia (CML) compared to existing treatments. The findings, presented at the 2024 American Society of Clinical Oncology (ASCO) meeting, reveal that Scemblix outperformed standard-of-care tyrosine kinase inhibitors (TKIs) in newly diagnosed patients.

Dr. Tim Hughes from the South Australian Health & Medical Research Institute (SAHMRI) highlighted the importance of these findings: “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.”

Superior Efficacy and Safety Profile

The Phase III ASC4FIRST trial, which included 405 adult patients, showed that Scemblix® achieved major molecular response (MMR) rates at week 48 in 67.7% of patients. This is nearly 20% higher than the MMR rates for those treated with investigator-selected standard TKIs (49.0%) and almost 30% higher compared to imatinib alone (40.2%). This represents a significant milestone, as TKIs have been the cornerstone of CML treatment, transforming it from a fatal disease to a manageable chronic condition.

Moreover, the safety profile of Scemblix® was notably better than that of the existing treatments. Patients on Scemblix® reported fewer grade 3 or higher adverse events (AEs). The rate of treatment discontinuation due to side effects was also halved compared to those on standard TKIs.

Breakthrough Therapy Designation

Scemblix has already received Breakthrough Therapy Designation from the U.S. FDA, and the new data will likely support expanded use for patients who have not previously been treated for CML. Currently, Scemblix is approved in over 70 countries, including the U.S., for adults who have received at least two prior TKI therapies.

“The compelling ASC4FIRST data highlight the potential of Scemblix to achieve better results than standard-of-care in newly diagnosed adults, while maintaining a favorable safety and tolerability profile. These results reinforce Scemblix as a proven treatment in Ph+CML-CP, as we continue to build on our 20-year legacy in CML innovation,” said Dr. Shreeram Aradhye, President, Development and Chief Medical Officer, Novartis.

Addressing Unmet Needs in CML Treatment

Despite the advancements brought by TKIs, many CML patients still face challenges such as unmet molecular response goals and treatment intolerance. Gerald Clements, a CML caregiver and patient advocate, emphasized the significance of effective and well-tolerated treatments: “Effective care that can be tolerated long-term is a key unmet need. By potentially bringing Scemblix to patients when they are first diagnosed, they may have an opportunity to be on a highly effective treatment while also maintaining their day-to-day from the start.”

The ASC4FIRST trial continues, with the next analysis scheduled for week 96 to further evaluate MMR and other key secondary endpoints. Analysts predict that with an MMR rate greater than 60% and a discontinuation rate less than 10%, Scemblix® could become the new standard of care for CML.