Novartis receives FDA Breakthrough Therapy designations for investigational STAMP inhibitor asciminib (ABL001) in chronic myeloid leukemia

  • Designation in patients with chronic myeloid leukemia (CML) resistant or intolerant to prior treatments based on positive data from pivotal Phase III ASCEMBL trial evaluating asciminib, an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP)1,2 

  • Despite advances in CML care, many patients are at risk of disease progression, and sequential TKI therapy may be associated with increased resistance and intolerance3-9 

  • Breakthrough Therapy designation is granted to medicines being evaluated for serious conditions where early clinical evidence indicates the potential for substantial improvement over available therapy10 

  • Asciminib is in development across multiple treatment lines of CML; first regulatory filing in pre-treated patients anticipated in first half of 2021 under the US FDA Real-Time Oncology Review program11-17 

Basel, February 8, 2020 - Novartis today announced that asciminib - a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) - has been granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). Asciminib was also granted BTD for the treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.

Despite tremendous advances in CML treatment over the past few decades, some of these pre-treated patients struggle to meet treatment goals due to resistance and intolerance18-23.
With few remaining treatment options, patients in later lines of care may be at risk of progression3-9.

These FDA designations, which may allow for an expedited development and review of asciminib, were based on:

  • The pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif (bosutinib)* in patients with Ph+ CML in CP previously treated with two or more TKIs1,2

  • A Phase I trial that included patients with Ph+ CML, some of them harboring the T315I mutation24

Data from these trials were shared at the 2020 Annual Meeting of the American Society of Hematology (ASH), and details on positive findings can be found here.

The FDA previously granted Fast Track designation to asciminib, and Novartis plans for a submission in the first half of 2021 for review under the FDA Oncology Center of Excellence Real-Time Oncology Review program.

About asciminib (ABL001)
Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP)11-17. As a STAMP inhibitor, asciminib is being studied in patients with chronic myeloid leukemia (CML) who experience resistance or intolerance to two or more tyrosine-kinase inhibitors (TKIs), and in several clinical trials in hopes of helping patients across multiple treatment lines of CML11-17, 25-32.

About ASCEMBL
ASCEMBL is the first head-to-head clinical trial in chronic myeloid leukemia using a second-generation tyrosine-kinase inhibitor (TKI) as a comparator. As a Phase III, multicenter, open-label, randomized study, ASCEMBL was designed to evaluate superiority in major molecular response rate at 24 weeks of the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia-chromosome positive CML in chronic phase previously treated with two or more TKIs2. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial2.