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VALOR-HCM Secondary and Exploratory Endpoints

For SRT-eligible patients with NYHA class II-III obstructive HCM,

CAMZYOS®
Significantly
Reduced LVOT
Obstruction
vs
Placebo1

Secondary endpoint: mean change in post-exercise LVOT peak gradient
from baseline to week 161,2

Treatment difference (95% CI): -38 mmHg (-49, -28); P<0.0001
Secondary Endpoint: Post-exercise LVOT Peak Gradient Chart Secondary Endpoint: Post-exercise LVOT Peak Gradient Chart

CI=confidence interval; HCM=hypertrophic cardiomyopathy; LVOT=left ventricular outflow tract obstruction; NYHA=New York Heart Association; SD=standard deviation; SRT=septal reduction therapy.

Exploratory endpoint: Mean Change in Resting and Valsalva LVOT Peak Gradient Measured Over the 16-Week Treatment Period2

While these exploratory endpoints were prespecified, they were not powered for significance.2

LVOT peak gradient difference at rest2 Treatment difference (95% CI): -33.4 mmHg (-42.3, -24.5)
Exploratory Endpoint: LVOT Gradient Chart Exploratory Endpoint: LVOT Gradient Chart
LVOT peak gradient difference with Valsalva2 Treatment difference (95% CI): -47.6 mmHg (-58.2, -37.0)
Exploratory Endpoint: LVOT  Gradient Chart With Valsalva Exploratory Endpoint: LVOT  Gradient Chart With Valsalva

The efficacy and safety of CAMZYOS was evaluated in VALOR-HCM, a Phase 3, double-blind, randomized, placebo-controlled trial in 112 adult SRT-eligible patients with severely symptomatic drug-refractory obstructive HCM (NYHA Class III–IV, or Class II with exertional syncope or near syncope, LVOT peak gradient ≥50 mmHg at rest or with provocation, and LVEF ≥60%). Patients were randomized in a 1:1 ratio to receive a starting dose of 5 mg of CAMZYOS (n=56) or placebo (n=56) once daily for 16 weeks. Dosage was monitored and adjusted at Weeks 8 and 12 to optimize patient response (decrease in LVOT gradient with Valsalva) and maintain an LVEF ≥50%.1,2

Primary Composite Endpoint

18% (n=10/56) of patients taking CAMZYOS and 77% (n=43/56) taking placebo met the primary composite endpoint* (remained guideline eligible for SRT at week 16 or chose to undergo SRT† by or at week 16). Difference (95% CI): 59% (44, 74); P<0.0001.1

  • *The primary composite endpoint is based on the investigator's guideline-based recommendation for SRT. Patients who undergo SRT, terminate early, die, or cannot otherwise be assessed for SRT eligibility at the end of the 16-week, placebo-controlled treatment period will also be classified as meeting the primary composite endpoint.4
  • Two patients in the CAMZYOS group and 2 patients in the placebo group decided to proceed with SRT.1
Select Important Safety Information

ADVERSE REACTIONS
Based on the safety profile from the pivotal EXPLORER-HCM trial, there were no new adverse reactions identified in VALOR-HCM. See EXPLORER-HCM safety.

For SRT-eligible patients with NYHA class II-III obstructive HCM,

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

Secondary endpoint: proportion of patients with ≥1 NYHA class improvement from baseline to week 161,2

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

CI=confidence interval; HCM=hypertrophic cardiomyopathy; NYHA=New York Heart Association; SRT=septal reduction therapy.

Exploratory endpoint: NYHA class changes from baseline to week 16 in CAMZYOS and placebo2

Data below showing improvement of ≥ 2 classes was exploratory in nature and not powered for significance.

Change in NYHA class at Week 16.2
Proportion of Patients with NYHA Class Changes Chart Proportion of Patients with NYHA Class Changes Chart

The efficacy and safety of CAMZYOS was evaluated in VALOR-HCM, a Phase 3, double-blind, randomized, placebo-controlled trial in 112 adult SRT-eligible patients with severely symptomatic drug-refractory obstructive HCM (NYHA Class III–IV, or Class II with exertional syncope or near syncope, LVOT peak gradient ≥50 mmHg at rest or with provocation, and LVEF ≥60%). Patients were randomized in a 1:1 ratio to receive a starting dose of 5 mg of CAMZYOS (n=56) or placebo (n=56) once daily for 16 weeks. Dosage was monitored and adjusted at Weeks 8 and 12 to optimize patient response (decrease in LVOT gradient with Valsalva) and maintain an LVEF ≥50%.1,2

Primary Composite Endpoint

18% (n=10/56) of patients taking CAMZYOS and 77% (n=43/56) taking placebo met the primary composite endpoint* (remained guideline eligible for SRT at week 16 or chose to undergo SRT† by or at week 16). Difference (95% CI): 59% (44, 74); P<0.0001.1

  • *The primary composite endpoint is based on the investigator's guideline-based recommendation for SRT. Patients who undergo SRT, terminate early, die, or cannot otherwise be assessed for SRT eligibility at the end of the 16-week, placebo-controlled treatment period will also be classified as meeting the primary composite endpoint.3
  • Two patients in the CAMZYOS group and 2 patients in the placebo group decided to proceed with SRT.1
Select Important Safety Information

ADVERSE REACTIONS
Based on the safety profile from the pivotal EXPLORER-HCM trial, there were no new adverse reactions identified in VALOR-HCM. See EXPLORER-HCM safety.

For SRT-eligible patients with NYHA class II-III obstructive HCM,

CAMZYOS® Showed Significant Mean Improvement in KCCQ-23–CSS (Patient-Reported Total Symptom Score and Physical Limitations) vs Placebo1

Secondary endpoint: mean change in KCCQ-23–CSS from baseline to week 161

Treatment difference (95% CI): 9 (5, 14); P<0.0001
Secondary Endpoint: Least Squares Mean Change in KCCQ-23 Chart Secondary Endpoint: Least Squares Mean Change in KCCQ-23 Chart
  • The baseline mean (SD) KCCQ-23–CSS was 70 (16) for the CAMZYOS group (n=56) and 66 (20) for the placebo group (n=56)2
  • KCCQ-23–TSS: mean (SD) change from baseline to week 16 was 10 (16) for CAMZYOS and 2 (14) for placebo1
  • KCCQ-23–PL Score: mean (SD) change from baseline to week 16 was 10 (19) for CAMZYOS and 2 (17) for placebo1
  • The KCCQ-23–CSS is derived from the TSS and PL score of the KCCQ-23. The Clinical Summary Score ranges from 0 to 100, with higher scores representing less severe symptoms and/or physical limitations1

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 efficacy and safety of CAMZYOS was evaluated in VALOR-HCM, a Phase 3, double-blind, randomized, placebo-controlled trial in 112 adult SRT-eligible patients with severely symptomatic drug-refractory obstructive HCM (NYHA Class III–IV, or Class II with exertional syncope or near syncope, LVOT peak gradient ≥50 mmHg at rest or with provocation, and LVEF ≥60%). Patients were randomized in a 1:1 ratio to receive a starting dose of 5 mg of CAMZYOS (n=56) or placebo (n=56) once daily for 16 weeks. Dosage was monitored and adjusted at Weeks 8 and 12 to optimize patient response (decrease in LVOT gradient with Valsalva) and maintain an LVEF ≥50%.1,2

Primary Composite Endpoint

18% (n=10/56) of patients taking CAMZYOS and 77% (n=43/56) taking placebo met the primary composite endpoint* (remained guideline eligible for SRT at week 16 or chose to undergo SRT† by or at week 16). Difference (95% CI): 59% (44, 74); P<0.0001.1

  • *The primary composite endpoint is based on the investigator's guideline-based recommendation for SRT. Patients who undergo SRT, terminate early, die, or cannot otherwise be assessed for SRT eligibility at the end of the 16-week, placebo-controlled treatment period will also be classified as meeting the primary composite endpoint.3
  • Two patients in the CAMZYOS group and 2 patients in the placebo group decided to proceed with SRT.1
Select Important Safety Information

ADVERSE REACTIONS
Based on the safety profile from the pivotal EXPLORER-HCM trial, there were no new adverse reactions identified in VALOR-HCM. See EXPLORER-HCM safety.

For SRT-eligible patients with NYHA class II-III obstructive HCM,

CAMZYOS® Reduced Cardiac Biomarkers (NT-proBNP and Cardiac Troponin I) vs Placebo1

The clinical significance of the NT-proBNP and cardiac troponin I findings is unknown

Secondary endpoint: Change in NT-proBNP from baseline to week 161,2

Geometric mean ratio difference: (95% CI): 0.33 (0.27, 0.42)* P<0.001
Secondary Endpoint: Geometric Mean Change in NT proBNP Chart Secondary Endpoint: Geometric Mean Change in NT proBNP Chart

Secondary endpoint: Change in cardiac troponin I from baseline to week 161,2

Geometric mean ratio difference (95% CI): 0.53 (0.41, 0.70)* P<0.001
Secondary Endpoint: Geometric Mean Change in Cardiac Troponin 1 Chart Secondary Endpoint: Geometric Mean Change in Cardiac Troponin 1 Chart

*Geometric mean ratios <1.0 represent an x-fold decrease for CAMZYOS compared with placebo.2

CI=confidence interval; GM=geometric mean; HCM=hypertrophic cardiomyopathy; NYHA=New York Heart Association; NT-proBNP=N-terminal pro B-type natriuretic peptide; SRT=septal reduction therapy.

The efficacy and safety of CAMZYOS was evaluated in VALOR-HCM, a Phase 3, double-blind, randomized, placebo-controlled trial in 112 adult SRT-eligible patients with severely symptomatic drug-refractory obstructive HCM (NYHA Class III–IV, or Class II with exertional syncope or near syncope, LVOT peak gradient ≥50 mmHg at rest or with provocation, and LVEF ≥60%). Patients were randomized in a 1:1 ratio to receive a starting dose of 5 mg of CAMZYOS (n=56) or placebo (n=56) once daily for 16 weeks. Dosage was monitored and adjusted at Weeks 8 and 12 to optimize patient response (decrease in LVOT gradient with Valsalva) and maintain an LVEF ≥50%.1,2

Primary Composite Endpoint

18% (n=10/56) of patients taking CAMZYOS and 77% (n=43/56) taking placebo met the primary composite endpoint* (remained guideline eligible for SRT at week 16 or chose to undergo SRT† by or at week 16). Difference (95% CI): 59% (44, 74); P<0.0001.1

  • *The primary composite endpoint is based on the investigator's guideline-based recommendation for SRT. Patients who undergo SRT, terminate early, die, or cannot otherwise be assessed for SRT eligibility at the end of the 16-week, placebo-controlled treatment period will also be classified as meeting the primary composite endpoint.3
  • Two patients in the CAMZYOS group and 2 patients in the placebo group decided to proceed with SRT.1
Select Important Safety Information

ADVERSE REACTIONS
Based on the safety profile from the pivotal EXPLORER-HCM trial, there were no new adverse reactions identified in VALOR-HCM. See EXPLORER-HCM safety.

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References:

  1. CAMZYOS [draft package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2023.
  2. 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.
  3. Gersh BJ, Maron BJ, Bonow RO, et al. 2011 ACCF/AHA guideline for the diagnosis and treatment of hypertrophic cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. J Am Coll Cardiol. 2011;58(25):e212-e260.
  4. Desai MY, Wolski K, Owens A, et al. Study design and rationale of VALOR-HCM: evaluation of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy who are eligible for septal reduction therapy. Am Heart J. 2021;239:80-89.