Frequency Therapeutics has finished a first-in-human study of its progenitor cell activation therapy, FX-322, in nine adults with severe to profound sensorineural hearing loss.
The company’s candidate is a combination of small molecules that are designed to activate progenitor cells and trigger the formation of new hair cells in the inner ear.
The patients enrolled in the trial were all scheduled to receive a cochlear implant within 24 hours of intratympanic injection of Frequency’s therapy. Researchers evaluated the safety of administering FX-322 while the patient was awake and also assessed the compound’s local and systemic bioavailability.
“The goal of this landmark first-in-human study was to prove the safety and tolerability of FX-322 at a dose that has been effective in restoring hearing in animals,” lead investigator Dr. Stephen O’Leary said in prepared remarks.
“This goal was achieved by showing that FX-322 was well tolerated with no drug related adverse events reported. Further, the results validated the feasibility of using a standard intratympanic injection to deliver FX-322 locally to the inner ear. In addition, we found that FX-322 successfully diffused from the middle ear to the perilymph fluid in the cochlea with minimal systemic drug exposure.”
“FX-322 has transformative therapeutic potential, as it is designed to activate dormant progenitor cells in the inner ear so that they will multiply and mature into new, fully functional hair cells to restore hearing,” chief medical officer Dr. Peter Weber added. “This is the first time a progenitor cell activator has been looked at in humans, and these promising results bode well for the continued development of FX-322 for hearing restoration and other potential PCA™ therapeutics to address additional disease indications.”
“Our Phase I study provides crucial data on safety and pharmacokinetics associated with intratympanic delivery of FX-322 as we move forward to clinical trials to monitor efficacy,” Dr. Daniel Lee, chair of Frequency’s clinical advisory board, said. “FX-322 represents a new class of targeted small molecule therapies in Frequency’s PCA™ portfolio with the potential to transform the field of regenerative medicine.”
“The completion of this study is a milestone for the clinical development of FX-322, a first-of-its-kind candidate with the potential to treat hearing loss for millions of people,” co-founder, president & CEO David Lucchino touted.
“Frequency is pioneering the field of PCA regenerative medicine to tackle a wide range of disease indications where no effective therapeutic solutions are available. The successful study in Australia validates our groundbreaking approach and sets the stage for our development of more progenitor cell activators for additional disease indications. We are grateful for the tremendous efforts made by the team at Frequency, our clinical investigators and most importantly the patients who have volunteered to undertake this revolutionary endeavor.”