Oct 11, 2024

Safety Clinical Trial Launched for Usher Syndrome 3 Drug

Research News

Researchers hope the molecule will slow the vision and hearing loss associated with USH3.

A Phase I clinical trial has been launched in Perth, Australia, to evaluate the safety and tolerability of an emerging oral drug known as BF844 for the treatment of Usher syndrome type 3 (USH3). The clinical trial will also evaluate the drug’s pharmacokinetics, i.e., how the drug is metabolized and distributed throughout the body. The trial will be completed in September 2025.

The trial, the first for an USH3 treatment, was launched by the USH III initiative, a non-profit organization dedicated to advancing research and developing treatments for USH3. The Foundation Fighting Blindness funded lab studies to prepare for the trial through the Brint Translational Research Program.

"This is a momentous step in our more than two-decade-long mission to develop a therapy for patients suffering from USH3. I am humbled and deeply grateful to the many people and organizations whose generous support  made this possible," said Cindy Elden, president and co-founder of the Usher III Initiative.

“We are delighted to have helped the Usher III Initiative with getting BF844 to the clinical stage. Moving a potential therapy out of lab studies and into a human trial is always challenging. We applaud the Usher III Initiative for their persistence and tenacity in making this clinical trial possible,” said Chad Jackson, PhD, senior director, preclinical translational research program, Foundation Fighting Blindness. “I’d like to especially thank Ms. Elden, Dr. Mahdi Farhan, and Ms. Georgia Horn for their dedication to this effort.”

Usher syndrome is a genetic condition causing progressive vision and hearing loss. More than 15 genetic subtypes of Usher syndrome have been identified. USH3 is caused by mutations in the gene CLRN1.