Trial results for a Phase 2a study investigating mavacamten in participants with heart failure with preserved ejection fraction (HFpEF) were posted on ClinicalTrials.gov on 2025-03-20. The study demonstrated geometric mean ratios to baseline at Week 26 of 0.7354 for resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) levels and 0.8645 for resting High Sensitivity Cardiac Troponin T (Hs-cTnT) levels.
Background
Mavacamten was investigated for its potential in heart failure with preserved ejection fraction (HFpEF). This Phase 2a study was designed as a proof-of-concept to evaluate the safety, tolerability, and preliminary efficacy of mavacamten on biomarker levels in participants with HFpEF who also had elevated NT-proBNP, with or without elevated cTnT.
Trial design
The study (NCT04766892) was a Phase 2a, proof-of-concept trial that enrolled 30 participants with Heart Failure With Preserved Ejection Fraction. The trial aimed to assess the safety, tolerability, and preliminary efficacy of mavacamten treatment on biomarker levels.
Key results
Key results from the trial included both safety and biomarker outcomes for the mavacamten group. Regarding safety, 23 participants experienced Treatment-Emergent Adverse Events (TEAEs), and 5 participants experienced Treatment-Emergent Serious Adverse Events (TESAEs). Notably, there were 0 participants with Adverse Events of Special Interest (AESIs). Other reported safety events included 4 participants with laboratory abnormalities reported as TEAEs, 4 participants with abnormal vital signs reported as TEAEs, and 5 participants with cardiac rhythm abnormalities reported as TEAEs.
For preliminary efficacy, assessed by biomarker levels at Week 26:
- The geometric mean ratio to baseline for resting N-Terminal Pro B-Type Natriuretic Peptide (NT-proBNP) levels was 0.7354.
- The geometric mean ratio to baseline for resting High Sensitivity Cardiac Troponin T (Hs-cTnT) levels was 0.8645.
What this means
The observed reductions in NT-proBNP and Hs-cTnT levels suggest a potential positive effect of mavacamten on cardiac biomarkers in participants with HFpEF. These biomarkers are indicators of cardiac stress and injury, respectively. The finding of 0 Adverse Events of Special Interest (AESIs) is also a notable safety observation in this Phase 2a proof-of-concept study. These preliminary findings will be crucial for informing the design of larger, more definitive clinical trials for mavacamten in the HFpEF population.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04766892, titled "A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT," were posted on 2025-03-20 on clinicaltrials.gov.