Efficacy

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

A chart showing the study design and key eligibility criteria for the study. Patients were randomized 2:1 to receive either SCEMBLIX 40mg (n=157) or bosutinib 500 mg once daily (n=76)

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

Bar graph showing MMR Rate for SCEMBLIX (40 mg bid) n=157 compared to Bosutinib (500 mg qd) n=76. At Week 24 (primary endpoint), 25% scemblix vs 13% bosutinib. Differenced 12% (95% CI, 2.2-22; P=0.029e). At Week 48, 29% scemblix vs 13% bosutinib. Differenced 16% (95% CI, 5.7-26). At Week 96, 38% scemblix vs 16% bosutinib. Differenced 22% (95% CI, 11-33; P=0.001).

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

When I started SCEMBLIX… within 4 months, I had broken that 0.01%. That’s a major molecular response.

SCEMBLIX demonstrated durable response rates across nearly 2 years of data¹²

Cumulative results for MMR across time points compared 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.¹

Almost all SCEMBLIX patients who achieved MMR maintained it for more than 16 months (97% probability).¹²

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

Chart showing CCyR Rate at week 24. SCEMBLIX (40 mg bid) n=103f. Week 24 41% (95% CI, 31-51) vs Bosutinib (500 mg qd) n=62f. Week 24 24% (95% CI, 14-37).

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

Chart showing MR4 rates at week 96 and MR4.5 rates at week 96. MR4 at Week 96 SCEMBLIX (40 mg bid) 17% n=27/157 vs Bosutinib (500 mg qd) 11% n=8/76. MR4.5 at Week 96 SCEMBLIX (40 mg bid) 11% n=17/157 vs Bosutinib (500 mg qd) 5% n=4/76

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:

of patients with the T315I mutation received MMR by 24 weeks (n=45)

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

Efficacy

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

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

SCEMBLIX demonstrated durable response rates across nearly 2 years of data¹²

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

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

IMPORTANT SAFETY INFORMATION

INDICATIONS

Efficacy

ASCEMBL: The first 3rd-line+ Phase 3 trial in CML evaluating efficacy and safety vs a 2nd-generation TKI comparator1,2

SCEMBLIX is the first treatment to demonstrate superior response rates vs bosutinib in a Phase 3 trial1,4-11

SCEMBLIX demonstrated durable response rates across nearly 2 years of data12

SCEMBLIX demonstrated fast responses at Week 24 and increased depth of response at Week 961,3

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

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

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

SCEMBLIX demonstrated durable response rates across nearly 2 years of data¹²

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

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