Novartis has reported positive results from the Phase III ASCEMBL study of the oral investigational treatment asciminib (ABL001) in patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP).
Data showed that the therapy nearly doubled the major molecular response (MMR) rate at 24 weeks as compared to Bosulif (bosutinib) in such patients.
A novel investigational treatment, asciminib targets the ABL myristoyl pocket (STAMP).
The multi-centre, open-label, randomised study analysed asciminib versus Bosulif in patients with Ph+ CML-CP.
Those with failure or intolerance to the recently administered TKI therapy were part of the trial that enrolled 233 patients who randomly received either asciminib 40mg twice daily or Bosulif 500mg once-a-day.
At 24 weeks, results showed that 40.8% of patients achieved a CCyR in the asciminib arm as compared to 24.2% in Bosulif arm.
Deep molecular response (DMR) rates were higher for patients in the asciminib arm versus those in the Bosulif arm.
In addition, about 50.6% of patients treated with asciminib had Grade ≥3 adverse events (AEs) as compared to 60.5% in Bosulif arm.
Novartis Global Drug Development head and chief medical officer John Tsai said: “We are very proud to once again advance a potentially transformative medicine, a novel STAMP inhibitor, for those who do not adequately respond or are intolerant to currently available TKIs.
“There is a clear need in later lines of therapy, and based on these results, we believe asciminib may become an important new development for patients.”
Asciminib obtained fast track designation from the US Food and Drug Administration (FDA).
Novartis plans to initiate submissions to the US and EU health authorities in the first half of next year.