In short
Ninerafaxstat trihydrochloride monohydrate is an investigational chemical substance with no marketing authorisation, being studied as a potential treatment for symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). One Phase 2 trial is authorised, planning to enrol 210 participants to compare ninerafaxstat against placebo over 12 weeks.
Key points
- Ninerafaxstat is an investigational substance with no marketing authorisation.
- The single registered trial is a Phase 2 study in non-obstructive hypertrophic cardiomyopathy (nHCM).
- The trial plans to enrol 210 participants and is currently authorised.
- Participants receive 400 mg ninerafaxstat orally as a modified-release tablet or a matching placebo.
- The main measure is the change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) from baseline to Week 12.
- The study is randomised and double-blinded, meaning neither participants nor researchers know who gets which treatment.
What is ninerafaxstat?
Ninerafaxstat trihydrochloride monohydrate, also known simply as ninerafaxstat, is a chemically synthesised substance classified in the cardiovascular disease therapeutic area. It is an investigational substance, meaning it has no marketing authorisation and is not yet approved as a medicine. The product is supplied by Imbria Pharmaceuticals Inc.
How does ninerafaxstat work?
The registered trial data does not describe the mechanism of action of ninerafaxstat. What is known from the registry is that the substance is being tested in patients with symptomatic non-obstructive hypertrophic cardiomyopathy, a heart condition where the heart muscle is thickened but does not block blood flow out of the heart.
Which conditions is ninerafaxstat studied for?
- Non-obstructive hypertrophic cardiomyopathy (nHCM) — a condition in which the heart muscle is thickened, causing symptoms such as breathlessness and fatigue, but without significant obstruction to blood flow leaving the heart.
How is ninerafaxstat given?
In the registered trial, ninerafaxstat is given as a modified-release tablet taken orally at a dose of 400 mg. The trial also includes a placebo arm with tablets identical in appearance to the ninerafaxstat tablets.
What do the clinical trials show?
There is one registered clinical trial for ninerafaxstat, a Phase 2 study that has been authorised but has not yet reported results. The trial is randomised and double-blinded, meaning participants are assigned by chance to receive either ninerafaxstat or placebo, and neither the participants nor the researchers know which treatment is being given.
The trial plans to enrol 210 participants with symptomatic non-obstructive hypertrophic cardiomyopathy. The main outcome measure is the change from baseline to Week 12 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS), a questionnaire that measures how heart failure symptoms affect a person's quality of life and daily functioning.
What side effects are known?
No side effect information is available from the registered trial data at this time.