For patients with NYHA class II–III
obstructive HCM,

Primary composite functional endpoint

To achieve the primary composite functional endpoint, patients needed to achieve either ≥1 NYHA class improvement and an increase of ≥1.5 mL/kg/min in peak oxygen consumption (pVO2) or no worsening NYHA class and an increase of ≥3.0 mL/kg/min in pVO2.1,2

At week 30, 37% (n=45/123) of patients taking CAMZYOS achieved the primary composite functional endpoint vs 17% (n=22/128) with placebo (95% CI: 9, 30; P=0.0005).1,2

Secondary endpoint: mean change in post-exercise LVOT peak gradient
from baseline to week 301,2

CAMZYOS
Significantly
Reduced LVOT
Obstruction
vs
Placebo1,2

LVOT obstruction was assessed by post-exercise LVOT peak gradient. Treatment difference (95% CI): -35 mmHg (-43, -28);P<0.0001
Secondary Endpoint LVOT Mean Change  CAMZYOSTM (mavacamten) vs Placebo, Chart Secondary Endpoint LVOT Mean Change  CAMZYOSTM (mavacamten) vs Placebo, Chart

Guideline-based threshold for surgery consideration (>50 mmHg)2

Guideline-based diagnostic criteria for obstruction (>30 mmHg)2

Data based on all randomized patients who received ≥1 dose of study treatment. LVOT gradient was analyzed with all available data without imputation.2

CI=confidence interval; LVOT=left ventricular outflow tract obstruction; SD=standard deviation.

The efficacy of CAMZYOS was evaluated in EXPLORER-HCM, a phase 3, double-blind, randomized, placebo-controlled trial that enrolled 251 adult patients with symptomatic (New York Heart Association [NYHA] class II and III) obstructive HCM, with left ventricular outflow tract (LVOT) peak gradient ≥50 mmHg, and left ventricle ejection fraction (LVEF) ≥55% at rest or with provocation.1 Patients were randomized in a 1:1 ratio to receive CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks.1

All subjects were initiated on CAMZYOS 5 mg (or matching placebo) once daily, and the dose was periodically adjusted to optimize patient response (decrease in LVOT gradient with Valsalva maneuver), maintain LVEF ≥50%, and was further informed by plasma concentrations of CAMZYOS.1,2

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

Prespecified exploratory analysis of post-exercise LVOT gradient

Exploratory endpoints: proportion of patients achieving post-exercise LVOT peak gradient <50 mmHg or <30 mmHg at week 302

74% (n=75/101) of patients on CAMZYOS had
a post-exercise LVOT gradient <50 mmHg
and
21% (n=22/106) in the placebo arm2*

57% (n=64/113) of patients on CAMZYOS had
a post-exercise LVOT gradient <30 mmHg
and
7% (n=8/114) in the placebo arm2†

While these exploratory endpoints were prespecified, they were not powered for significance.3

  • *Only patients with baseline post-exercise LVOT peak gradient of at least 50 mmHg were assessed.2
  • Only patients with baseline post-exercise LVOT peak gradient of at least 30 mmHg were assessed.2
Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

For patients with NYHA class II–III
obstructive HCM,

Primary composite functional endpoint

To achieve the primary composite functional endpoint, patients needed to achieve either ≥1 NYHA class improvement and an increase of ≥1.5 mL/kg/min in peak oxygen consumption (pVO2) or no worsening NYHA class and an increase of ≥3.0 mL/kg/min in pVO2.1,2

At week 30, 37% (n=45/123) of patients taking CAMZYOS achieved the primary composite functional endpoint vs 17% (n=22/128) with placebo (95% CI: 9, 30; P=0.0005).1,2

Secondary endpoint: mean change in pVO2 consumption from baseline
to week 301,2

Significant Improvement in pVO2 in Patients Receiving CAMZYOS vs Placebo1,2

Treatment difference (95% CI): 1.4 (0.6, 2.1); P<0.0006
Secondary Endpoint pVO2 Consumption Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart Secondary Endpoint pVO2 Consumption Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart

Mean change in pVO2 from baseline to week 30 was 1.4 mL/kg/min (SD, 3.1) for patients taking CAMZYOS and -0.1 mL/kg/min (SD, 3.0) for patients taking placebo.1

pVO2 measures peak oxygen consumption during cardiopulmonary exercise testing. Data based on all randomized patients who received ≥1 dose of study treatment.2,3

  • Data based on all randomized patients who received ≥1 dose of study treatment.2

CI=confidence interval; SD=standard deviation.

The efficacy of CAMZYOS was evaluated in EXPLORER-HCM, a phase 3, double-blind, randomized, placebo-controlled trial that enrolled 251 adult patients with symptomatic (New York Heart Association [NYHA] class II and III) obstructive HCM, with left ventricular outflow tract (LVOT) peak gradient ≥50 mmHg, and left ventricle ejection fraction (LVEF) ≥55% at rest or with provocation.1 Patients were randomized in a 1:1 ratio to receive CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks.1

All subjects were initiated on CAMZYOS 5 mg (or matching placebo) once daily, and the dose was periodically adjusted to optimize patient response (decrease in LVOT gradient with Valsalva maneuver), maintain LVEF ≥50%, and was further informed by plasma concentrations of CAMZYOS.1,2

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

For patients with NYHA class II–III
obstructive HCM,

Primary composite functional endpoint

To achieve the primary composite functional endpoint, patients needed to achieve either ≥1 NYHA class improvement and an increase of ≥1.5 mL/kg/min in peak oxygen consumption (pVO2) or no worsening NYHA class and an increase of ≥3.0 mL/kg/min in pVO2.1,2

At week 30, 37% (n=45/123) of patients taking CAMZYOS achieved the primary composite functional endpoint vs 17% (n=22/128) with placebo (95% CI: 9, 30; P=0.0005).1,2

Secondary endpoint: proportion of patients with ≥1 NYHA class improvement from baseline to week 301,2

Significantly More Patients Improved by ≥1 NYHA Class With CAMZYOS vs Placebo1,2

Treatment difference (95% CI): 34% (22, 45); P=0.0001

At baseline, approximately 73% of the randomized patients were NYHA class II and 27% were NYHA class III.1

Data based on all randomized patients who received ≥1 dose of study treatment. Missing NYHA class data at week 30 were imputed with the week 26 value for NYHA response. Patients with a nonevaluable NYHA secondary endpoint were considered nonresponders.2

The efficacy of CAMZYOS was evaluated in EXPLORER-HCM, a phase 3, double-blind, randomized, placebo-controlled trial that enrolled 251 adult patients with symptomatic (New York Heart Association [NYHA] class II and III) obstructive HCM, with left ventricular outflow tract (LVOT) peak gradient ≥50 mmHg, and left ventricle ejection fraction (LVEF) ≥55% at rest or with provocation.1 Patients were randomized in a 1:1 ratio to receive CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks.1

All subjects were initiated on CAMZYOS 5 mg (or matching placebo) once daily, and the dose was periodically adjusted to optimize patient response (decrease in LVOT gradient with Valsalva maneuver), maintain LVEF ≥50%, and was further informed by plasma concentrations of CAMZYOS.1,2

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

NYHA class changes from baseline to week 30 in CAMZYOS and placebo2

The data below were used to calculate the secondary endpoint related to NYHA class but were exploratory in nature and not evaluated for significance to compare between the treatment groups.2,3

Data used to Calculate Secondary Endpoint  NYHA Class Changes CAMZYOS™ (mavacamten) vs Placebo, Chart Data used to Calculate Secondary Endpoint  NYHA Class Changes CAMZYOS™ (mavacamten) vs Placebo, Chart

CI=confidence interval; NYHA=New York Heart Association.

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

For patients with NYHA class II–III
obstructive HCM,

Primary composite functional endpoint

To achieve the primary composite functional endpoint, patients needed to achieve either ≥1 NYHA class improvement and an increase of ≥1.5 mL/kg/min in peak oxygen consumption (pVO2) or no worsening NYHA class and an increase of ≥3.0 mL/kg/min in pVO2.1,2

At week 30, 37% (n=45/123) of patients taking CAMZYOS achieved the primary composite functional endpoint vs 17% (n=22/128) with placebo (95% CI: 9, 30; P=0.0005).1,2

KCCQ-23–CSS and HCMSQ-SoB With CAMZYOS and Placebo1,2

Secondary endpoint: mean change in KCCQ-23–CSS from baseline to week 301,2*

CAMZYOS showed significant mean improvement in KCCQ-23–CSS score (patient-reported symptom burden and physical limitations) vs placebo1,2


Treatment difference (95% CI): 9 (5, 13); P<0.0001
KCCQ-23 clinical–summary score: mean change from baseline over time1
Secondary Endpoint  KCCQ-23 Clinical Summary Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart Secondary Endpoint  KCCQ-23 Clinical Summary Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart

Mean baseline KCCQ-23 CSS (SD) was 71 (16) for the CAMZYOS group (n=99) and 71 (19) for the placebo group (n=97).1

KCCQ-23 TSS: change from baseline to week 30, mean (SD) was 12 (15) for CAMZYOS and 5 (16) for placebo.1

KCCQ-23 PL: change from baseline to week 30, mean (SD) was 15 (17) for CAMZYOS and 4 (15) for placebo.1

The KCCQ-23–CSS is composed of the physical limitations and total symptom burden scores of the KCCQ-23. The Clinical Summary Score ranges from 0 to 100, with higher scores representing less severe symptoms and/or physical limitations.1

Missing data were not imputed to summarize the baseline and change from baseline to week 30 values. Difference in mean change from baseline between treatment groups was estimated using a mixed model for repeated measures.1

Patient-reported outcomes were analyzed with all available data. Sensitivity analyses were conducted to assess the effect of the missing data. When missing data were imputed with unfavorable results for the CAMZYOS group, the estimated treatment effects of KCCQ-CSS remained significant (P<0.05).2

KCCQ-23–CSS=Kansas City Cardiomyopathy Questionnaire (23-item version)–Clinical Summary Score; LS=least squares; PL=physical limitations; SD=standard deviation; SE=standard error; TSS=total symptom score.

The Kansas City Cardiomyopathy Questionnaire (KCCQ) has been used to capture and quantify patient-reported outcomes in a disease-specific fashion.3

The efficacy of CAMZYOS was evaluated in EXPLORER-HCM, a phase 3, double-blind, randomized, placebo-controlled trial that enrolled 251 adult patients with symptomatic (New York Heart Association [NYHA] class II and III) obstructive HCM, with left ventricular outflow tract (LVOT) peak gradient ≥50 mmHg, and left ventricle ejection fraction (LVEF) ≥55% at rest or with provocation.1 Patients were randomized in a 1:1 ratio to receive CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks.1

All subjects were initiated on CAMZYOS 5 mg (or matching placebo) once daily, and the dose was periodically adjusted to optimize patient response (decrease in LVOT gradient with Valsalva maneuver), maintain LVEF ≥50%, and was further informed by plasma concentrations of CAMZYOS.1,2

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

Secondary endpoint: mean change in HCMSQ-SoB from baseline to
week 301,2

CAMZYOS showed significant mean improvement in HCMSQ-SoB (patient-reported frequency and severity of shortness of breath) vs placebo1

Treatment difference: -2 (-2, -1); P<0.0001

HCMSQ shortness of breath domain: mean change from baseline over time1

Secondary Endpoint  HCMSQ-SoB Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart Secondary Endpoint  HCMSQ-SoB Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart

Mean baseline HCMSQ-SoB domain score (SD) was 5 (3) for both CAMZYOS (n=108) and placebo (n=109) groups.1The HCMSQ-SoB domain score measures the frequency and severity of shortness of breath. The HCMSQ-SoB scale score ranges from 0 to 18 with lower scores representing less shortness of breath.1 Missing data were not imputed to summarize the baseline and change from baseline to week 30 values. Difference in mean change from baseline between treatment groups was estimated using a mixed model for repeated measures.1 Patient-reported outcomes were analyzed with all available data. Sensitivity analyses were conducted to assess the effect of the missing data. When missing data were imputed with unfavorable results for the CAMZYOS group, the treatment effects of HCMSQ-SoB remained significant (P<0.05).2

  • HCMSQ-SoB=Hypertrophic Cardiomyopathy Symptom Questionnaire-Shortness of Breath subscore.

The HCMSQ-SoB is an assessment created expressly for the EXPLORER-HCM
study that measures the frequency and severity of shortness of breath.4

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

For patients with NYHA class II–III
obstructive HCM,

Primary composite functional endpoint

To achieve the primary composite functional endpoint, patients needed to achieve either ≥1 NYHA class improvement and an increase of ≥1.5 mL/kg/min in peak oxygen consumption (pVO2) or no worsening NYHA class and an increase of ≥3.0 mL/kg/min in pVO2.1,2

At week 30, 37% (n=45/123) of patients taking CAMZYOS achieved the primary composite functional endpoint vs 17% (n=22/128) with placebo (95% CI: 9, 30; P=0.0005).1,2

Exploratory Endpoint: Mean Change in NT-proBNP Over the 30-Week Treatment Period1

Changes in NT-proBNP Levels for Patients Receiving CAMZYOS and Placebo1,2

The reduction in NT-proBNP was 80% greater with CAMZYOS than for placebo. The clinical significance of these findings is unknown.1,2

Treatment difference (95% CI): 0.20 (0.17, 0.24)

While this endpoint was prespecified, it was not powered for significance.3

Exploratory Endpoint NT-proBNP Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart Exploratory Endpoint NT-proBNP Mean Change CAMZYOS™ (mavacamten) vs Placebo, Chart

NT-proBNP is a marker for negative outcomes in patients with obstructive hypertrophic cardiomyopathy.4

CI=confidence interval; NT-proBNP=N-terminal pro B-type natriuretic peptide.

The efficacy of CAMZYOS was evaluated in EXPLORER-HCM, a phase 3, double-blind, randomized, placebo-controlled trial that enrolled 251 adult patients with symptomatic (New York Heart Association [NYHA] class II and III) obstructive HCM, with left ventricular outflow tract (LVOT) peak gradient ≥50 mmHg, and left ventricle ejection fraction (LVEF) ≥55% at rest or with provocation.1 Patients were randomized in a 1:1 ratio to receive CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks.1

All subjects were initiated on CAMZYOS 5 mg (or matching placebo) once daily, and the dose was periodically adjusted to optimize patient response (decrease in LVOT gradient with Valsalva maneuver), maintain LVEF ≥50%, and was further informed by plasma concentrations of CAMZYOS.1,2

Select Important Safety Information

ADVERSE REACTIONS
In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%).
Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

Please see additional Important Safety Information below, including Boxed WARNING.

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References:

  1. CAMZYOS. [draft package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2022.