Investigational therapies
Overview: There are ongoing studies for treating HCM or its symptoms. These are called investigational therapies or clinical trials.
In this section we are going to talk about them and their pathway to consumers.
Investigational therapies under clinical investigation
There are several new therapies under investigation for use in HCM. Below is a list of several medications at different stages of discovery, trial, or submission to the FDA (Food and Drug Administration - USA)
The MyPeak-1 clinical trial will research an investigational gene therapy called TN-201 for the treatment of HCM caused by mutations (changes) in the MYBPC3 gene. Changes in the MYBPC3 gene prevent certain cells from making a protein needed for the heart to pump as expected.
TN-201 is designed to reach the specific muscle cells that make the heart contract and relax with each beat.
MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM)
Status: Enrolling
MAPLE-HCM is a Phase 3, multi-center, randomized, double-blind active-comparator clinical trial of aficamten compared to metoprolol in patients with symptomatic obstructive HCM.
ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM)
Status: Enrolling
ACACIA-HCM is a Phase 3 randomized, placebo-controlled, double-blind, multi-center clinical trial designed to evaluate aficamten in patients with symptomatic nHCM.
