Expanded Access

Imbria Pharmaceuticals is a clinical-stage company revolutionizing the treatment of cardiovascular disease by targeting cardiac energy metabolism. The company’s lead product candidate, ninerafaxstat, is being evaluated in Phase 2 clinical trials as a potential treatment for non-obstructive hypertrophic cardiomyopathy (nHCM), a debilitating disease with no currently approved treatments, and for heart failure with preserved ejection fraction (HFpEF), a large and growing unmet need.

We are not currently offering expanded access to our investigational medicine. Right now, the best way to access our therapy is by enrolling in a clinical trial.

Drugs being studied in clinical trials have unknown benefits and unknown risks that will not be understood until the clinical trials are complete.

Eventually, if results from the clinical trials are favorable, they will be submitted to the U.S. Food and Drug Administration (FDA) and other regulatory bodies for review of the drug’s safety and efficacy in order to seek approval for the drug. Obtaining regulatory approval for a new medicine is the best way to bring rapid access to the greatest number of patients who may benefit.

Sometimes patients may be able to access investigational drugs outside of a clinical trial. In the United States, this is possible through “expanded access,” sometimes also referred to as “compassionate use.” Unlike the use of an investigational drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for treatment purposes, rather than to gather data on safety and effectiveness.

In the early stage of drug development, it is critical to systematically obtain information about the safety and tolerability of the investigational drug in a controlled manner. It is also important to evaluate investigational drugs under controlled conditions to assess their efficacy. Complete data on safety, tolerability and effectiveness is not yet available for ninerafaxstat. Therefore, Imbria is not accepting expanded access requests at this time. We will reevaluate this policy in the future as more information from our clinical trials is available. If the policy changes, this webpage will be updated.

If you have questions about this policy or would like information about how to enroll in our clinical studies, please contact us at info@imbria.com. You can also obtain information about our studies at https://clinicaltrials.gov/.