SCEMBLIX is a kinase inhibitor indicated 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).

With almost 4 y of follow-up in ASCEMBL, ASC continued to show greater efficacy and better safety/tolerability than BOS in pts with CML-CP after ≥2 prior TKIs. The robust safety profile of ASC was sustained through each analysis in ASCEMBL (wk 24, wk 96, and EOS Tx), confirming that pts receiving ASC can maintain a high level of response and continue Tx without experiencing late-emerging AEs.

This is a multicenter, phase IIIb, open-label study of ASC in pts with CML-CP...Deep molecular response (MR4; BCR::ABL1IS ≤0.01%) rates by wks 4, 12, and 24 were 27.8%, 31.6%, and 30.0% in cohort A and 5.9%, 11.8%, and 27.8% in cohort B, respectively...This interim analysis of the AIM4CML trial further demonstrates the tolerability and efficacy of ASC and is among the first clinical reports on QD dosing of ASC in pts with CML-CP.

A higher proportion of patients on asciminib than bosutinib remained on treatment and continued to derive benefit over time, supporting asciminib as a standard of care for patients with CML-CP previously treated with ≥2 TKIs

Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting marketing authorization for Scemblix® (asciminib) for the treatment of 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).

Adults with CML-CP treated with ≥2 prior TKIs were randomized 2:1 to asciminib 40 mg twice daily...Pts intolerant of their most recent TKI were eligible only if they had BCR-ABL1 on the International Scale >0.1% at screening….The novel STAMP inhibitor asciminib demonstrates continued superior efficacy and a limited adverse event profile…

Novartis today announced that the US Food and Drug Administration (FDA) accepted and granted Priority Review to the company’s New Drug Application (NDA) for asciminib (ABL001) in chronic myeloid leukemia (CML)...Priority Review granted based on positive data from the pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif® (bosutinib)* in patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP)...and data from a Phase I trial that included patients with Ph+ CML-CP harboring the T315I mutation.

Novartis today announced that asciminib...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.

...At baseline, ≥1 BCR-ABL1 mutation was present in 12.7% pts...asciminib, a first-in-class STAMP inhibitor, demonstrated statistically significant and clinically meaningful superiority in efficacy compared with BOS (primary objective), deeper MR rates, and a favorable safety profile. These results support the use of asciminib as a new treatment option in CML, particularly in R/I pts who received ≥2 prior TKIs.

Novartis announced today 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)....FDA has granted asciminib Fast Track designation.

...No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly BCR-ABL1 ratio > 0.1% IS according to central laboratory at the screening examination for patients intolerant to the most recent TKI therapy...

This is the first real-world study describing treatment patterns and clinical outcomes among patients with Ph+ CML-CP treated with asciminib in the US. The findings of real-world treatment efficacy based on achieving BCR::ABL1 ≤0.1% were consistent with the ASCEMBL trial. Patients who initiated asciminib after 2 previous TKIs had higher persistence and treatment response rates than those initiated on asciminib after ≥3 previous TKIs.

Asciminib monotherapy was well tolerated and showed promising clinical activity in pts with baseline BCR-ABL1IS ≤ 1%, with 75.0% remaining on therapy and in MMR at the data cutoff.