Novartis' CML drug hits PhIII targets

Novartis' Phase III ASCEMBL study has met its primary goal showing that asciminib was superior to bosutinib in inducing a major molecular response (MMR) in patients with a certain from of chronic myeloid leukaemia (CML).

The study is assessing asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – in adults with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs).

Despite advances in CML care, many patients are still at risk of disease progression, and sequential TKI therapy may be associated with increased resistance and intolerance, the Swiss drug giant noted.

By specifically targeting the ABL myristoyl pocket, STAMP inhibition has the potential to help address resistance and intolerance in later treatment lines for CML. Additional studies are also underway to evaluate asciminib in earlier lines of therapy.

“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 effects16,” said John Tsai, Novartis' head of Global Drug Development and chief medical officer.

“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.”

The drug giant said it would submit the data for presentation at an upcoming medical meeting, as well as to regulatory authorities.