ASCEMBL STUDY DESIGN

ASCEMBL: The first 3rd-line+ Phase 3 trial in CML evaluating efficacy and safety vs a 2nd-generation TKI comparator^1,2

Multicenter, randomized, active-controlled, and open-label study^1,2

Primary end point³

• MMR at Week 24

Key secondary end point³

• MMR at Week 96

Other select secondary end points²

• Cytogenetic response rates and MMR rates, at and by scheduled data collection time points

• Time to and duration of MMR

• Time to and duration of CCyR

• Safety and tolerability

Key baseline characteristics²

• Resistance to last prior TKI: 61% of patients in the SCEMBLIX arm vs 71% with bosutinib

• Intolerance to last prior TKI: 38% of patients in the SCEMBLIX arm vs 29% with bosutinib

• MCyR at baseline: 29% of patients in the SCEMBLIX arm vs 29% with bosutinib

• Percentages of patients who had previously received 2, 3, 4, or 5 or more prior lines of TKIs were 48%, 31%, 15%, and 6%, respectively

EFFICACY IN PATIENTS IN LATER LINES

SCEMBLIX is the first treatment to demonstrate superior response rates vs bosutinib in a Phase 3 trial^1,4-11

FOR ADULTS WITH Ph+ CML-CP, PREVIOUSLY TREATED WITH 2 TKIs¹

SCEMBLIX more than doubled the MMR rate at Week 96 vs bosutinib¹

• Median follow-up was 28 months¹

The median duration of treatment was 24 months (range: 0 to 46 months) for patients receiving SCEMBLIX and 7 months (range: 0 to 43 months) for patients receiving bosutinib.¹

SCEMBLIX demonstrated durable response rates across nearly 2 years of data¹²

Percentages of patients who had previously received 2, 3, 4, or 5 or more prior lines of TKIs were 48%, 31%, 15%, and 6%, respectively.¹ 

SCEMBLIX demonstrated fast responses at Week 24 and increased depth of response at Week 96^1,3

More patients achieved CCyR at Week 24 with SCEMBLIX vs bosutinib¹

• The CCyR rate at Week 96 was 40% (95% CI, 30-50) in patients receiving SCEMBLIX and 16% (95% CI, 8-28) for bosutinib. Note that any patients who achieved CCyR and later achieved MMR would not have been assessed for CCyR at Week 96. Of patients achieving CCyR during the study, 1 patient in the SCEMBLIX arm and 2 patients in the bosutinib arm lost response^1,3

More patients achieved MR4 and MR4.5 at Week 96 with SCEMBLIX vs bosutinib³

Learn more about the SCEMBLIX safety profile >

EFFICACY IN PATIENTS WITH THE T315I MUTATION

The clinical efficacy and safety of SCEMBLIX for the treatment of patients with Ph+ CML-CP who have the T315I mutation were evaluated in a multicenter, open-label study¹

SCEMBLIX demonstrated effectiveness in adult patients with T315I mutation¹

In a study of patients with the T315I mutation who received SCEMBLIX 200 mg bid:

MMR was achieved by 96 weeks in 49% (95% CI, 34-64) of the patients (n=45) treated with SCEMBLIX.¹

The median duration of treatment was 108 weeks (range: 2 to 215 weeks).¹  

• By Week 24, MMR was achieved in 58% (n=11/19) in ponatinib-naive patients vs 31% (n=8/26) of patients pretreated with ponatinib¹³

• Percentages of patients who had previously received 1, 2, 3, 4, or 5 or more TKIs were 18%, 31%, 36%, 13%, and 2.2%, respectively¹

Efficacy was based on 45 patients with Ph+ CML-CP who have the T315I mutation who received SCEMBLIX at a dose of 200 mg bid. Patients continued treatment until unacceptable toxicity or treatment failure occurred.¹

bid, twice daily; CCyR, complete cytogenetic response; CML-CP, chronic myeloid leukemia in chronic phase; IS, International Scale; MCyR, major cytogenetic response; MMR, major molecular response; MR, molecular response; qd, once daily; TKI, tyrosine kinase inhibitor.

MMR was defined as BCR::ABL1^IS ≤0.1%.¹
CCyR was defined as 0% of Philadelphia chromosome–positive metaphases in bone marrow aspirate with at least 20 examined.¹
MCyR was defined as 0% to 35% Ph+ metaphases.³
MR4 was defined as BCR::ABL1^IS ≤0.01%.²
MR4.5 was defined as BCR::ABL1^IS ≤0.0032%.²

^aMust meet the definition of treatment failure per the 2013 European LeukemiaNet recommendations. Patients meeting treatment failure criteria on bosutinib could be switched to SCEMBLIX.^2
bDefined as nonhematologic grade 3 or 4 toxicity while on therapy; persistent grade 2 toxicity, unresponsive to optimal management, including dose adjustments; or hematologic grade 3 or 4 toxicity while on therapy, recurrent after dose reduction to the lowest recommended dose.^2
cPatients will continue to receive study treatment for up to 96 weeks after the last patient’s first dose or 48 weeks after the last patient switches to SCEMBLIX, whichever is longer.^2
dEstimated using a common risk difference stratified by baseline MCyR status.^1
eEstimated using a Cochran-Mantel-Haenszel 2-sided test stratified by baseline MCyR status.^1
fCCyR analysis based on patients who were not in CCyR at baseline.¹