Hypertrophic Cardiomyopathy Association supports study of potential treatment for nonbstructive HCM
Imbria Pharmaceuticals is partnering with the Hypertrophic Cardiomyopathy Association (HCMA) to help expedite patient recruitment for its ongoing FORTITUDE-HCM study looking at the first drug specifically developed to treat symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). No treatments are currently approved for the debilitating disease.
The randomized, double-blinded, placebo-controlled phase 2b clinical trial is evaluating the investigational drug ninerafaxstat for safety, tolerability and efficacy. Fourteen centers in the United States and U.K. are involved in the trial.
According to Imbria Pharmaceuticals, the investigational therapy was designed to optimize mitochondrial energy production in cardiomyocytes without adverse impact on left ventricular ejection fraction, heart rate or blood pressure. The company said ninerafaxstat is a partial fatty acid oxidation (pFOX) inhibitor that shifts myocardial substrate utilization in favor of glucose oxidation to generate more ATP per unit of oxygen consumed, increasing myocardial metabolic efficiency.
HCMA is supporting trial recruitment by educating the HCM community about the study. The goal is to connect more patients and clinicians with trial information to help speed enrollment. This includes highlighting the FORTITUDE-HCM study at the Imbria-sponsored HCMA Annual Patient Meeting on Oct. 18.
The trial needs to enroll 165 patients. There are currently 67 patients who have agreed to participate.
“Every step forward in clinical research brings us closer to improving the lives of those living with nHCM,” Lisa Salberg, founder and CEO of HCMA, said in a statement. “By supporting patient recruitment for FORTITUDE-HCM and spotlighting this collaboration at our annual meeting, we aim to increase awareness of this potential treatment and connect patients and families with meaningful opportunities to advance research and improve care.”
The study's primary endpoint is a change from baseline in the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS). Secondary endpoints include additional measurements of symptoms from the KCCQ and measurements of functional capacity from standardized cardiopulmonary exercise testing (CPET).
HCM is the most common inherited cardiac disease, which causes abnormal thickening of the heart muscle and leads to heart failure. It is estimated that roughly one-third of HCM patients have nHCM.
Without an effective treatment, HCM patients previously had their symptoms managed, but they would eventually require a heart transplant. But there was an explosion of interest in finding and treating HCM patients earlier when mavacamten became the first effective drug treatment approved in 2022. However, the drug is only effective in obstructive HCM (oHCM) and is not effective in nHCM patients.
Imbria has said phase 2a data showed compelling safety and efficacy potential in an approvable endpoint. The company also plans to initiate a Phase 2b clinical trial in patients with heart failure with preserved ejection fraction, which is another area of unmet need without effective treatments.
Dave Fornell has covered healthcare for more than 17 years, with a focus in cardiology and radiology. Fornell is a 5-time winner of a Jesse H. Neal Award, the most prestigious editorial honors in the field of specialized journalism. The wins included best technical content, best use of social media and best COVID-19 coverage. Fornell was also a three-time Neal finalist for best range of work by a single author. He produces more than 100 editorial videos each year, most of them interviews with key opinion leaders in medicine. He also writes technical articles, covers key trends, conducts video hospital site visits, and is very involved with social media. E-mail: [email protected]