Tuesday, April 16, 2024

In Phase III Chronic Myeloid Leukemia Trial, Novartis’ Scemblix® Outperforms Standard TKIs

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Novartis has announced positive results from the ASC4FIRST Phase III trial, comparing Scemblix® (asciminib) with other tyrosine kinase inhibitors (TKIs) in newly diagnosed patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This groundbreaking trial has significant implications for the treatment of chronic myeloid leukemia, a type of blood cancer.

The ASC4FIRST trial, a randomized head-to-head Phase III study, is the first of its kind to compare a chronic myeloid leukemia treatment against approved standard-of-care first- and second-generation TKIs. Scemblix demonstrated superior major molecular response (MMR) rates at week 48 compared to standard TKIs (imatinib, nilotinib, dasatinib, and bosutinib). The results showed clinically meaningful and statistically significant differences in both primary endpoints – efficacy of Scemblix versus investigator-selected TKIs and efficacy versus imatinib.

One of the key aspects of Scemblix is its favorable safety and tolerability profile. Fewer adverse events (AEs) and treatment discontinuations were observed with Scemblix compared to standard TKIs. Moreover, no new safety signals were noted, aligning with the established safety profile of Scemblix. Prof. Tim Hughes, MD, from the South Australian Health & Medical Research Institute (SAHMRI), highlighted the importance of these findings. Given that a significant proportion of newly diagnosed chronic myeloid leukemia patients do not achieve treatment goals, there is a pressing need for tolerable treatment options. Scemblix offers the possibility of balancing treatment with quality of life.

Scemblix Surpasses Standard TKIs in Groundbreaking Phase III Myeloid Leukemia Treatment Study

Treatment advancements have transformed CML into a chronic disease, with life expectancy similar to the general population. However, intolerance and AEs remain the primary reasons for discontinuing TKI therapy. Over 60% of newly diagnosed CML patients fail to meet 12-month molecular response goals. Scemblix presents an opportunity for patients to achieve treatment goals while maintaining their lifestyle.

Shreeram Aradhye, M.D., President of Development and Chief Medical Officer at Novartis, expressed excitement about Scemblix’s potential to help newly diagnosed CML patients. The chronic nature of CML often requires long-term TKI therapy, making well-tolerated and efficacious treatment options essential for adherence. The ASC4FIRST trial remains ongoing, with the next data readout scheduled for week 96, focusing on the key secondary endpoint (MMR at week 96) and additional secondary endpoints. These details will be presented at an upcoming medical conference and included in regulatory submissions in 2024.

ASC4FIRST (NCT04971226) is a multi-center, open-label, randomized study of oral Scemblix 80 mg QD versus investigator-selected first- or second-generation TKI in 405 adult patients with newly diagnosed Ph+ CML-CP. The study’s primary endpoint is to compare the efficacy of asciminib vs. investigator-selected TKI and imatinib, based on the proportion of patients achieving MMR at week 48. The study also includes a key secondary endpoint of the proportion of patients achieving MMR at week 96 and additional secondary safety and efficacy endpoints.

Myeloid Leukemia

Chronic Myeloid Leukemia Treatment with Enhanced Safety Profile, Now Approved in Over 60 Countries

Scemblix is a groundbreaking chronic myeloid leukemia treatment that specifically targets the ABL myristoyl pocket and is designed to minimize off-target kinase-mediated effects. This specificity leads to an improved safety and tolerability profile compared to the current standard of care. Scemblix is approved in over 60 countries, including the US and EU, for adults with Ph+ CML-CP previously treated with two or more TKIs. In some countries, including the US, it is also approved for patients with the T315I mutation.

In conclusion, Scemblix represents a significant advancement in the treatment of Ph+ CML-CP, offering a new option for patients who experience resistance or intolerance to existing TKI therapies. Its potential across multiple treatment lines, both as monotherapy and in combination, highlights its versatility and importance in the evolving landscape of CML treatment.


Resource: Novartis, January 05, 2023

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