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Pivotal Trials Overview

For patients with NYHA class II–III obstructive HCM,

Proven in 2 Phase 3 Pivotal Trials—CAMZYOS Consistently Demonstrated Benefit in Meeting the Primary Endpoint and Secondary Endpoints vs Placebo (LVOT Obstruction and NYHA Class)1

Comparisons between the 2 studies cannot be made.

EXPLORER-HCM

A 30-week, phase 3 clinical trial for patients with
NYHA class II–III obstructive HCM (N=251).1

At baseline, 92% of patients were on background
therapy
(BB or CCB).2

Review full study design >

VALOR-HCM

A 16-week, phase 3 clinical trial for patients with severely symptomatic obstructive HCM (N=112) who were eligible for SRT.1

At baseline, 95% of patients were on background therapy (BB, CCB, disopyramide, or combination therapy).3

Review full study design >

Significant Benefit in Both Primary Endpoints1

Primary endpoints explained

EXPLORER-HCM: The primary composite functional endpoint was defined as achieved with an improvement of pVO2 by 1.5 mL/kg/min or more and improvement in NYHA class by at least 1 or an improvement of pVO2 by 3.0 mL/kg/min or more and no worsening in NYHA class.1,2

VALOR-HCM: The primary endpoint was a composite of patients who remained eligible for SRT (per 2011 AHA/ACC Guidelines) at week 16 or decided to proceed with SRT prior to or at week 16. Patients who discontinued treatment or whose response status could not be assessed at the end of the 16-week dosing period were classified as SRT eligible.1,3

2x as many patients taking CAMZYOS

achieved the primary composite functional endpoint (symptoms and exercise capacity) vs placebo; 37% with CAMZYOS (n=45/123) vs. 17% with placebo (n=22/128).

Difference (95% CI): 19% (9, 30); P=0.0005

See EXPLORER-HCM primary
composite functional endpoint data >

>3x as many patients were able to avoid SRT at 16 weeks

(no longer guideline-eligible for SRT at Week 16 and did not choose to undergo SRT by Week 16) with CAMZYOS vs placebo. 18% (n=10/56) of patients taking CAMZYOS met the primary composite endpoint vs 77% (n=43/56) taking placebo. Two patients in each group decided to proceed with SRT.

Difference (95% CI): 59% (44, 74); P<0.0001

See VALOR-HCM primary composite endpoint data >

Significantly Reduced Obstruction1

Both studies evaluated changes in LVOT obstruction

-47 mmHg mean reduction

of post-exercise LVOT peak gradient from baseline with CAMZYOS vs -10 mmHg with placebo.

Difference (95% CI): -35 mmHg (-43, -28); P<0.0001

See EXPLORER-HCM LVOT obstruction data >

-39 mmHg mean reduction

of post-exercise LVOT peak gradient from baseline with CAMZYOS vs -2 mmHg with placebo.

Difference (95% CI): -38 mmHg (-49, -28); P<0.0001

See VALOR-HCM LVOT obstruction data >

Significantly Reduced Symptoms1

Both studies evaluated changes in NYHA class

65% of patients receiving CAMZYOS

(n=80/123) improved by ≥1 NYHA class from baseline vs 31% in the placebo group (n=40/128).

Difference (95% CI): 34% (22, 45); P<0.0001

See EXPLORER-HCM NYHA class data >

63% of patients receiving CAMZYOS

(n=35/56) improved by ≥1 NYHA class from baseline vs 21% in the placebo group (n=12/56).

Difference (95% CI): 41% (25, 58); P<0.0001

See VALOR-HCM NYHA class data >

Comparisons between the 2 studies
cannot be made.

EXPLORER-HCM

A 30-week, phase 3 clinical trial for patients with
NYHA class II–III obstructive HCM (N=251).1

At baseline, 92% of patients were on background
therapy
(BB or CCB).2

Significant Benefit in
Both Primary Endpoints1

Primary endpoints explained

EXPLORER-HCM: The primary composite functional endpoint was defined as
achieved with an improvement of pVO2 by 1.5 mL/kg/min or more and improvement in NYHA class by at least 1 or an improvement of pVO2 by 3.0 mL/kg/min or more and no worsening in NYHA class.1,2

VALOR-HCM: The primary endpoint was a composite of patients who remained eligible for SRT (per 2011 AHA/ACC Guidelines) at week 16 or decided to proceed with SRT prior to or at week 16. Patients who discontinued treatment or whose response status could not be assessed at the end of the 16-week dosing period were classified as SRT eligible.1,3

2x as many patients taking CAMZYOS

achieved the primary composite functional endpoint (symptoms and exercise capacity) vs placebo; 37% with CAMZYOS (n=45/123) vs. 17% with placebo (n=22/128).

Difference (95% CI): 19% (9, 30); P=0.0005

Significantly Reduced Obstruction1

Both studies evaluated changes
in LVOT obstruction

-47 mmHg mean reduction

of post-exercise LVOT peak gradient from baseline with CAMZYOS vs -10 mmHg with placebo.

Difference (95% CI): -35 mmHg (-43, -28); P<0.0001

Significantly Reduced Symptoms1

Both studies evaluated changes
in NYHA class

65% of patients receiving CAMZYOS

(n=80/123) improved by ≥1 NYHA class from baseline vs 31% in the placebo group (n=40/128).

Difference (95% CI): 34% (22, 45); P<0.0001

Comparisons between the 2 studies
cannot be made.

VALOR-HCM

A 16-week, phase 3 clinical trial for patients with severely symptomatic obstructive HCM (N=112) who were eligible for SRT.1

At baseline, 95% of patients were on background therapy (BB, CCB, disopyramide, or combination therapy).3

Significant Benefit in
Both Primary Endpoints1

Primary endpoints explained

EXPLORER-HCM: The primary composite functional endpoint was defined as
achieved with an improvement of pVO2 by 1.5 mL/kg/min or more and improvement in NYHA class by at least 1 or an improvement of pVO2 by 3.0 mL/kg/min or more and no worsening in NYHA class.1,2

VALOR-HCM: The primary endpoint was a composite of patients who remained eligible for SRT (per 2011 AHA/ACC Guidelines) at week 16 or decided to proceed with SRT prior to or at week 16. Patients who discontinued treatment or whose response status could not be assessed at the end of the 16-week dosing period were classified as SRT eligible.1,3

>3x as many patients were able to avoid SRT at 16 weeks

(no longer guideline-eligible for SRT at Week 16 and did not choose to undergo SRT by Week 16) with CAMZYOS vs placebo. 18% (n=10/56) of patients taking CAMZYOS met the primary composite endpoint vs 77% (n=43/56) taking placebo. Two patients in each group decided to proceed with SRT.

Difference (95% CI): 59% (44, 74); P<0.0001

Significantly Reduced Obstruction1

Both studies evaluated changes
in LVOT obstruction

-39 mmHg mean reduction

of post-exercise LVOT peak gradient from baseline with CAMZYOS vs -2 mmHg with placebo.

Difference (95% CI): -38 mmHg (-49, -28); P<0.0001

Significantly Reduced Symptoms1

Both studies evaluated changes
in NYHA class

63% of patients receiving CAMZYOS

(n=35/56) improved by ≥1 NYHA class from baseline vs 21% in the placebo group (n=12/56).

Difference (95% CI): 41% (25, 58); P<0.0001

BB=beta blocker; CCB=calcium channel blocker; CI=confidence interval; HCM=hypertrophic cardiomyopathy; LVOT=left ventricular outflow tract; NYHA=New York Heart Association; SRT=septal reduction therapy.

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Reference:

  1. CAMZYOS [package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2023.
  2. Olivotto I, Oreziak A, Barriales-Villa R, et al; EXPLORER-HCM study investigators. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759-769.
  3. Desai MY, Owens A, Geske JB, et al. Myosin inhibition in patients with obstructive hypertrophic cardiomyopathy referred for septal reduction therapy. J Am Coll Cardiol. 2022;80(2):95-108.