August 26, 2020″Novartis announced that, at primary analysis, the Phase III ASCEMBL study met its primary endpoint of statistically significant superiority in major molecular response (MMR) rate at 24 weeks for asciminib (ABL001) vs. bosutinib.
The study evaluates asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – in adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs).
Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial.
“Our ability to treat patients with TKIs changed CML care forever. However, the risk of disease progression is a reality for many patients – especially those who experience resistance to sequential TKI therapy or those who cannot adhere to treatment due to the daily impact of intolerable side effects,” said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis.
“We are incredibly grateful to the patients and investigators around the world who participated in this study. These results with asciminib are a testament to our commitment to further transform CML care – this time through STAMP inhibition, by exploiting a natural regulatory mechanism of the ABL kinase.”
Data from the ASCEMBL trial will be submitted for presentation at an upcoming medical meeting, and results will be shared with regulatory authorities. The U.S. FDA has granted Fast Track designation for asciminib.
Findings from earlier studies of asciminib were presented during past annual meetings of the European Hematology Association.
Asciminib (ABL001)
Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP).
As a STAMP inhibitor, asciminib may help address tyrosine-kinase inhibitor (TKI)-resistance and intolerance in later treatment lines of chronic myeloid leukemia (CML)8-14, and it is being studied in several clinical trials in hopes of helping patients across multiple treatment lines of CML7,10,11,13-15,17.
About ASCEMBL
ASCEMBL is a Phase III, multicenter, open-label, randomized study comparing the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs).
Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial.
Novartis Commitment to CML
Our ongoing research in Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) has helped transform the disease from a fatal leukemia to a chronic condition in most patients. Novartis maintains an unwavering commitment to scientific innovation and access to care for patients worldwide.
As an organization committed to patients, Novartis continues to reimagine CML care by pursuing ambitious goals with courage, passion and commitment for the global CML community.”
https://www.novartis.com/news/media-releases/novartis-investigational-novel-stamp-inhibitor-asciminib-abl001-meets-primary-endpoint-phase-iii-chronic-myeloid-leukemia-study
