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GEN News Highlights: December 21, 2017

Frequency Completes Human Progenitor Cell Activation Study to Restore Hearing

  • Frequency Therapeutics successfully completed a first-in-human study evaluating its lead Progenitor Cell Activation (PCA™) candidate FX-322, which is in development to restore hearing in patients with hearing loss due to loss of inner ear hair cells. FX-322 is a proprietary combination of small molecules that are designed to activate inner ear progenitor cells and the generation of new inner ear hair cells in the cochlea. The completed Phase I study, carried out at the Royal Victoria Eye and Ear Hospital in Melbourne, Australia, enrolled nine adult patients with severe to profound sensorineural hearing loss who were scheduled to receive a cochlear implant within 24 hours after intratympanic injection of FX-322. 

    The trial, which met all its endpoints, was designed to demonstrate the safety and tolerability of FX-322. “This goal was achieved by showing that FX-322 was well tolerated with no drug-related adverse events reported,” commented lead study investigator Stephen O’Leary, Ph.D., who is the William Gibson Chair of Otolaryngology at the University of Melbourne. “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.”

    Frequency says the completed first-in-human trial lays a foundation for future studies in patients with moderate hearing loss, who aren’t eligible for cochlea implants, and whose hearing can be followed over time following treatment with FX-322. “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,” added Daniel J. Lee, M.D., chair of the Clinical Advisory Board of Frequency and associate professor, department of otolaryngology, Massachusetts Eye and Ear and Harvard Medical School. “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.”

    Founded in 2015, Frequency is developing small-molecule drugs that transiently activate Lgr5+ progenitor cells to differentiate and regenerate damaged tissues. Unlike other approaches, Frequency’s technology is designed to activate stemness and trigger progenitor cell differentiation, rather than force conversion of Lgr5+ cells into the desired cell type. Potential applications include hearing loss, skin disorders, and gastrointestinal disease, the firm claims. Frequency raised $32 million in Series A funding earlier this year to support development of its hearing restoration program.

    Successful completion of Frequency’s Phase I study comes a day after an international research team reported the use of Cas-9-based genome-editing technique to restore hearing in a mouse model of human genetically mediated deafness.