WOBURN, MASSACHUSSETTS – Biotech startup, Frequency Therapeutics, announced late last month the successful completion of a first-in-human study of the company’s investigative compound FX-322, the company’s lead Progenitor Cell Activation (PCA) candidate for hearing restoration. The preliminary results were recently presented at a lecture last month at the US-Japan Symposium on Drug Delivery.

According to the company, FX-322 is a “proprietary combination of small molecules designed to transiently activate inner ear progenitor cells and enable the creation of new inner ear hair cells in the cochlea.”

“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. 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.” –Stephen O’Leary, M.D., lead investigator for the study

Getting Closer to Cochlear Hair Cell Restoration in Humans?

The Phase 1 safety study, which enrolled 9 adult subjects with severe to profound sensorineural hearing loss, was conducted at the Royal Victoria Eye and Ear Hospital in Melbourne, Victoria, Australia.

The study participants received intratympanic injections of FX-322 within a 24 hour period prior to cochlear implantation. The results confirmed the safety and tolerability of the compound in the awake patient, and according to the company, “we were able to confirm the bioavailability both locally and systemically.”

“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. 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.” –Peter C. Weber, M.D., Chief Medical Officer of Frequency

The successful results of the safety trial help lay the groundwork for future clinical trials in patients with moderate hearing losses that are not candidates for cochlear implantation.

Hailing the successful completion of the Phase 1 trial, President and CEO of Frequency Therapeutics, David Lucchino commented: “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.”

Further information on the FX-322 301 study can be found here.

Frequency Announces Creation of Regenerative Medicine Advisory Board

Shortly after the company announced the completion of its first-in-human trial with FX-322, it also announced that it has brought together a group of renowned scientists and researchers with expertise in the fields of stem cell biology and regenerative medicine.

The newly created PCA Regenerative Medicine Scientific Advisory Board “will help guide and support Frequency’s leadership in advancing a next-generation approach to regenerative medicine with the potential to fully realize the power of the body’s own repair capabilities.”

Members of the Board include:

Sean Morrison, Ph.D. , Director of the Children’s Medical Center Research Institute (CRI), UT Southwestern, the Mary McDermott Cook Chair in Pediatric Genetics, the Kathryne and Gene Bishop Distinguished Chair in Pediatric Research, and a Howard Hughes Medical Institute Investigator

, Director of the Children’s Medical Center Research Institute (CRI), UT Southwestern, the Mary McDermott Cook Chair in Pediatric Genetics, the Kathryne and Gene Bishop Distinguished Chair in Pediatric Research, and a Howard Hughes Medical Institute Investigator Siddhartha Mukherjee, M.D., D.Phil. , Assistant Professor of Medicine at Columbia University, Pulitzer Prize winning author

, Assistant Professor of Medicine at Columbia University, Pulitzer Prize winning author Lee Rubin, Ph.D. , Professor of Stem Cell and Regenerative Biology at Harvard University, Director of Translational Medicine at the Harvard Stem Cell Institute

, Professor of Stem Cell and Regenerative Biology at Harvard University, Director of Translational Medicine at the Harvard Stem Cell Institute Amy Wagers, Ph.D., the Forst Family Professor of Stem Cell and Regenerative Biology at Harvard University

“We are honored to welcome this esteemed group of individuals to help steer Frequency’s scientific direction towards ground-breaking discoveries in multiple disease indications through the use of small molecules to activate the body’s repair processes,” said Chris Loose, Ph.D., Co-founder and Chief Scientific Officer of Frequency.

Source: Frequency Therapeutics