EXPLORER-LTE durable efficacy

EXPLORER-LTE is an open-label, single-arm, ongoing extension of the Phase 3 EXPLORER-HCM trial which investigated the use of CAMZYOS in patients with symptomatic NYHA class II and III obstructive HCM^1–3

In EXPLORE-HCM, ~2x as many patients achieved the primary composite endpoint* with CAMZYOS vs placebo at Week 30 (37% [n=45/123] vs 17% [n=22/128], respectively; 95% CI: 8.67 to 30.13; p=0.0005)^2,3

*Primary composite endpoint defined as a ≥1.5 mL/kg/min increase in pVO2 and ≥1 NYHA class improvement or a ≥3.0 mL/kg/min increase in pVO2 and no worsening of NYHA class.^2,3

While the endpoints in the EXPLORER-LTE trial were prespecified, they were not powered for statistical significance.

CAMZYOS sustained an improvement in post-exercise (Valsalva) LVOT obstruction through to Week 180¹

Change in mean Valsalva LVOT gradient from baseline* to Week 180¹

Mean change ± SD from baseline to Week 180 in Valsalva LVOT gradient (central-read) was –55.3 ± 33.7 mmHg (n=91)

Adapted from Garcia-Pavia P et al. 2024.¹*BL values represent those from the beginning of the MAVA-LTE, EXPLORER-LTE cohort, not the beginning of the parent study (EXPLORER-HCM).¹

83%

(n=191/231) of patients achieved a Valsalva LVOT gradient of ≤30 mmHg from baseline to Week 180¹

CAMZYOS showed a sustained improvement in NYHA class, with 66% of patients achieving NYHA class I (asymptomatic) by Week 180¹

Change in NYHA class distribution from baseline to Week 180¹

Adapted from Garcia-Pavia P et al. 2024.¹

78%

(n=74/95) of patients improved by ≥1 NYHA class from baseline to Week 180¹

CAMZYOS reduced NT-proBNP levels from baseline to Week 180¹

Change in NT-proBNP from baseline* to Week 180¹

Median change (IQR) from baseline to Week 180 in NT-proBNP was –562 ng/L (-1162.5, -209; n=88)

Adapted from Garcia-Pavia P et al. 2024.¹
*BL values represent those from the beginning of the MAVA-LTE, EXPLORER-LTE cohort, not the beginning of the parent study (EXPLORER-HCM).¹

54% (n=50/93) of patients had a normal NT-proBNP level (<124 ng/L) at Week 180 vs 10% (n=22/230) at baseline¹

>6x

reduction in NT-proBNP levels at Week 180 vs baseline¹

CAMZYOS sustained an improvement in patient-reported frequency and severity of shortness of breath through to Week 180¹

HCMSQ-SoB domain score*: Mean change from baseline to Week 180¹

Mean change (SD) from baseline to Week 180 in HCMSQ-SoB was –3.7 (3.7) points; n=90

Adapted from Garcia-Pavia P et al. 2024.¹*The HCMSQ-SoB domain score measures the frequency and severity of shortness of breath. The HCMSQ-SoB domain score ranges from 0 to 18, with lower scores representing less shortness of breath.¹

>2x

mean improvement in HCMSQ-SoB domain score at Week 180 vs baseline¹

Mean LVEF remained above 50% with CAMZYOS from baseline to Week 180¹

Change in LVEF from baseline* to Week 180¹

Mean change (SD) from baseline to Week 180 in LVEF was –11% (8.9%); n=93

Adapted from Garcia-Pavia P et al. 2024.¹

At the data cutoff (31 August 2023), 20 patients experienced transient reductions in site-read LVEF <50% resulting in treatment interruption. All 20 patients recovered LVEF to ≥50% after treatment interruption, 14 resumed treatment, and 6 discontinued treatment (1 later re-enrolled)¹

Please refer to the CAMZYOS Summary of Product Characteristics for further information on dosing, initiation and safety.

BL, baseline; HCMSQ SoB, Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness of Breath; IQR, interquartile range; LTE, long-term extension; LVEF, left ventricular ejection fraction; LVOT, left ventricular outflow tract; NT-proBNP, N-terminal pro-B-type natriuretic peptide; NYHA, New York Heart Association; SD, standard deviation.

References

Garcia-Pavia P et al. Eur Heart J. 2024. ehae579.
CAMZYOS (mavacamten) Summary of Product Characteristics.
Olivotto I et al. Lancet. 2020;396(10253):759–769.

3500-GB-2400410 | November 2024

CAMZYOS sustained an improvement in post-exercise (Valsalva) LVOT obstruction through to Week 1801

83%

CAMZYOS showed a sustained improvement in NYHA class, with 66% of patients achieving NYHA class I (asymptomatic) by Week 1801

78%

CAMZYOS reduced NT-proBNP levels from baseline to Week 1801

>6x

CAMZYOS sustained an improvement in patient-reported frequency and severity of shortness of breath through to Week 1801

>2x