Foundation Funds Early Stage Clinical Trial of a Potential Nonsense Mutation Treatment

The Cystic Fibrosis Foundation awarded up to $1.61 million to Eloxx Pharmaceuticals Inc. to conduct a U.S.-based Phase 2 clinical trial of an investigational drug that could potentially treat people with cystic fibrosis who have a nonsense mutation.

| 2 min read

The Cystic Fibrosis Foundation awarded up to $1.61 million to Eloxx Pharmaceuticals Inc. for an early Phase 2 study of its investigational drug ELX-02 that could potentially “read through” premature stop signals in people with CF with nonsense mutations (also known as “x” or “stop” mutations). These mutations cause the cells to stop the production of the CFTR protein midway through the process, resulting in shortened, non-functional protein that the cell recognizes as defective and destroys.

If the clinical trials are successful, ELX-02 could potentially treat people with cystic fibrosis who have at least one nonsense mutation. Currently, this group of individuals does not have a drug that addresses the underlying cause of the disease, and identifying these types of treatments for all people with CF is a top priority of the Foundation.

The U.S. clinical trial will take place at sites within the Foundation's Therapeutics Development Network, the largest CF clinical trials network in the world. The Phase 2 clinical trial will enroll up to 8 patients and evaluate the safety and tolerability of ELX-02 in people with CF who have at least one G542X mutation. A European version of the study will enroll up to 16 patients. A summary of the initial results is expected before the end of 2019.

Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease. Learn more about the potential treatments the Foundation is exploring for people with nonsense and other rare mutations. For more information on this study, please see the Eloxx press release.

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