For patients with NYHA class II–III obstructive HCM,

In EXPLORER-LTE, CAMZYOS® Consistently Reduced Obstruction, and Improved Symptoms (NYHA Class) Through ~3.5 Years1

On this page:         Primary Objective         LVOT Obstruction         NYHA Class         Cardiac Biomarker         Cardiac Structure

EXPLORER-LTE: Primary Objective–Safety and Tolerability

In the pivotal EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group were dizziness (27% vs 18%) and syncope (6% vs 2%).2

EXPLORER-LTE: LVOT Obstruction

In the pivotal EXPLORER-HCM trial, -49 (34) mmHg mean change (SD) in Valsalva LVOT gradient with CAMZYOS (n=123) vs -12 (31) mmHg with placebo (n=128) from baseline to Week 30.2

Interim analysis from EXPLORER-LTE

CAMZYOS Demonstrated Consistent and Sustained Reductions in Mean Valsalva LVOT Gradient to Week 1801

Exploratory objective (interim data): Mean Valsalva LVOT gradient from baseline* to Week 1801,2

While this exploratory endpoint was prespecified, it was not powered for significance

Important LVOT gradient thresholds3:

  • A resting or provoked LVOT gradient of ≥50 mmHg is the obstructive criteria for SRT in patients with drug-refractory symptoms.1,3

Additional EXPLORER-LTE LVOT data1:

  • An LVOT peak gradient of ≥30 mmHg is indicative of obstruction
  • Overall, 83% (n=191) of patients achieved a reduced Valsalva LVOT gradient of ≤30 mmHg, or below obstructive levels at the time of analysis (data cutoff)
* Baseline is defined as last non-missing measurement before the first dose of CAMZYOS in MAVA-LTE.1
Based on the results of the 91 patients who have reached Week 180.1


LVOT=left ventricular outflow tract; SRT=septal reduction therapy.

Interim analysis from EXPLORER-LTE

Overall, ~83% of Patients No Longer Had Obstruction at the Time of Analysis (Data Cutoff; n=191)1,3§

Proportion of patients who achieved a Valsalva LVOT gradient of ≤30 mmHg by visit1,3

chart-mean-valsalva-lvot-explorer-lte

This exploratory endpoint was derived from a post-hoc analysis and was not powered for significance.

Based on the AHA/Multisociety Guideline for HCM, obstruction is considered present if peak LVOT gradient is ≥30 mmHg.3
§ Centrally read.1

EXPLORER-LTE: NYHA Class

In the pivotal EXPLORER-HCM trial 65% of patients (n=80/123) taking CAMZYOS improved by ≥1 NYHA class vs 31% (n=40/128) taking placebo from baseline to Week 30. Approximately 73% of the randomized patients were NYHA class II and 27% were NYHA class III at baseline.3

Interim analysis from EXPLORER-LTE

~78% of Patients Improved by ≥1 NYHA Class From Baseline to Week 1801||

Secondary objective: Proportion of patients with at least 1 NYHA class improvement from baseline to Week 180 (n=95)1

78-37 image

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

Of the patients (n=95) at the Week 180 visit1:

  • 63 patients (66%) were NYHA Class I
  • 29 patients (31%) were NYHA Class II
  • 3 patients (3%) were NYHA Class III

Additional EXPLORER-LTE NYHA class data1:

  • One patient worsened from NYHA Class II to NYHA Class III at Week 180 and had ongoing atrial fibrillation unrelated to CAMZYOS during this time
|| Time points for NYHA functional class assessments included Weeks 12, 48, 108, 120, 132, 144, 156, and 180 as per the trial protocol.
Baseline is defined as last non-missing measurement before the first dose of CAMZYOS in MAVA-LTE.
# Percentage of patients who achieved improvement of ≥2 classes were also included in the percentage of those who improved by ≥1 class.


NYHA class distribution over time1

image-nyha-class-distribution-over-time

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

EXPLORER-LTE: Cardiac Biomarker

In the pivotal EXPLORER-HCM trial, the proportion of geometric mean ratio of NT-proBNP between the CAMZYOS group and placebo group was 0.20 (95% CI: 0.17, 0.24) from baseline to Week 30.2

Interim analysis from EXPLORER-LTE

Normalized NT-proBNP Observed in More Than Half of Patients on CAMZYOS at Week 1801

Exploratory objective (interim data): Median NT-proBNP from baseline** to Week 1801

chart-nt-probnp-explorer-lte

While this exploratory endpoint was prespecified, it was not powered for significance. The clinical significance of the NT-proBNP findings is unknown.1

  • At baseline, ~10% (n=22/230) of patients presented with a NTproBNP concentration of <124 ng/L, indicative of normal range1
  • The proportion of patients with NT-proBNP levels in the normal range increased to ~28% at Week 4 (n=62/218), and ~54% at Week 180 (n=50/93)1
  • At Week 180, the median (IQR) plasma concentration of NT-proBNP was 118 (58, 304) ng/L1
** Baseline is defined as last non-missing measurement before the first dose of CAMZYOS in MAVA-LTE.1

EXPLORER-LTE: Cardiac Structure

In the pivotal EXPLORER-HCM trial, the mean (SD) reduction from baseline in left atrial volume index (LAVI) in the CAMZYOS group (-7.5 [7.8] mL/m2) versus no change in the placebo group (-0.1 [8.7] mL/m2).2

Interim analysis from EXPLORER-LTE

Changes in LAVI Over the 180 Week Treatment Period With CAMZYOS1,5

Exploratory objective (interim data): Mean LAVI from baseline†† to Week 1801,5

chart-mean-lavi-explorer-lte

While this exploratory endpoint was prespecified, it was not powered for significance. The clinical significance of the LAVI findings is unknown.1

†† Baseline is defined as last non-missing measurement before the first dose of CAMZYOS in MAVA-LTE.1

The EXPLORER-LTE cohort of the MAVA-LTE trial sought to evaluate the safety and efficacy of CAMZYOS starting at 5 mg in adults who had completed the EXPLORER-HCM trial.* The EXPLORER-LTE cohort (n=231) is a single-arm study without an active comparator.1,5,6

 

Dose adjustments could be made at Weeks 4, 8, 12, and during subsequent visits based on the patient’s Valsalva LVOT gradient and LVEF, except for Week 24, in which the patient’s stress TTE was used. Unscheduled dose adjustments could occur at Week 24 and every 12 weeks thereafter. Treatment was temporarily interrupted if LVEF <50%. Mean time from EXPLORER-HCM end of study to Day 1 of EXPLORER-LTE was 66.5 days (range: 3-359 days).1,5,6

 

* Patients enrolled in EXPLORER-LTE were part of either the CAMZYOS or placebo arm in the EXPLORER-HCM trial.1,5,6
This is not consistent with the current approved dosing schedule per the U.S. Full Prescribing Information.2

References:

  1. Garcia-Pavia P, Oreziak A, Masri A, et al. Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J. 2024; ehae579. doi:10.1093/eurheartj/ehae579
  2. CAMZYOS. [package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2024.
  3. Ommen SR, Ho CY, Asif IM, et al. 2024 AHA/ACC/AMSSM/HRS/PACES/SCMR guideline for the management of hypertrophic cardiomyopathy: a report of the American Heart Association/American College of Cardiology Joint Committee on Clinical Practice Guidelines. Circulation. 2024;149(23):e1239-e1311. doi:10.1161/CIR.0000000000001250
  4. Data on file. Princeton, NJ: Bristol-Myers Squibb Company; 2024.
  5. Garcia-Pavia P, Oreziak A, Masri A, et al. Long-term safety and efficacy of mavacamten in obstructive hypertrophic cardiomyopathy: up to 3.5-year follow-up results of the EXPLORER cohort of MAVA-Long-Term Extension study. Presented at European Society of Cardiology; August 30-September 2, 2024; London and online. Oral presentation 84470.
  6. Rader F, Oręziak A, Choudhury L, et al. Mavacamten treatment for symptomatic obstructive hypertrophic cardiomyopathy: interim results from the MAVA-LTE study, EXPLORER-LTE Cohort. J Am Coll Cardiol. 2024;12(1):164-177.


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3500-US-2400621   04/25

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