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Frequency Therapeutics’ First-in-Human Safety Study of FX-322 for Hearing Restoration Achieves All Endpoints

- Landmark first step in the development of FX-322 as a potential first-in-class progenitor cell activator for hearing restoration -

- Preliminary results were presented at an invited lecture at the US-Japan Symposium on Drug Delivery on December 17, 2017 -

WOBURN, Mass., December 21, 2017 – Frequency Therapeutics, a clinical stage biotechnology company spearheading the movement to harness the body’s innate regenerative potential with its Progenitor Cell Activation (PCA™) regeneration platform, today announced the successful completion of a first-in-human study of FX-322, the company’s lead PCA™ candidate for hearing restoration. 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 loss of inner ear hair cells is the most common cause of sensorineural hearing loss. The study met all endpoints laid out in the first-in-human clinical trial design.

“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,” said Stephen O’Leary, M.D., lead investigator for the study, and the William Gibson Chair of Otolaryngology at The University of Melbourne. “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.”

The Phase 1 safety study was conducted at the Royal Victoria Eye and Ear Hospital in Melbourne, Victoria, Australia. The study enrolled 9 adult subjects with severe to profound sensorineural hearing loss who were scheduled to receive a cochlear implant in the 24 hours following intratympanic injection of FX-322. Our aim was to determine the safety and tolerability of FX-322 in the awake patient. Further, we were able to confirm the bioavailability both locally and systemically. This successful trial lays the groundwork for future trials in patients with moderate hearing loss who are not candidates for the cochlear implant and whose hearing can be studied over time.

“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,” said Peter C. Weber, M.D., Chief Medical Officer of Frequency and an Otolaryngologist at Boston Medical Center. “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 1 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,” said 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.”

“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,” said David Lucchino, President, Co-founder and CEO of Frequency. “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.”

For more information on the FX-322 301 study, please refer to using identifier NCT03300687. This study was completed under full cGMP/GCP conditions in accord with international regulatory bodies.  

Virtually all hearing deficits in human arise from damage and/or loss of key cells in the inner ear called sensory hair cells. These inner ear hair cells convert sound waves into nerve impulses. In adult mammals, unlike birds or reptiles, inner ear hair cells do not spontaneously regenerate following injury, although progenitor cells capable of regenerating hair cells remain present in the ear. Chronic noise induced hearing loss, Frequency’s lead program, is a significant unmet need with no approved therapeutic option. Around 48 million people are affected in the U.S. alone and the World Health Organization (WHO) estimates that 1.1 billion children and adults ages 12-35 years old are at risk for hearing loss from recreational noise alone. Hearing loss caused by prolonged exposure to excessive noise is observed in many professional environments, such as heavy construction sites or military training. However, repetitive exposure to everyday loud sounds associated with a busy subway or rail system, emergency vehicle sirens, musical concerts and the excessive use of headphones at high volumes can have a negative impact on hearing. Frequency’s therapeutic candidate for noise induced hearing loss, FX-322, is a proprietary combination of small-molecules drugs designed to transiently activate inner ear progenitor cells, create new hair cells, and improve hearing.

Frequency Therapeutics’ precise and controlled approach enables Lgr5+ progenitor cells to divide transiently and differentiate, much like what is seen in naturally regenerating organs such as the skin and intestine. Frequency activates ‘stemness’ in adult tissue through mimicking signals provided by neighboring cells (the stem cell niche) with small molecules, and this proprietary approach is known as the Progenitor Cell Activation (PCA™) platform. Frequency believes that PCA™ has the potential to yield a whole new category of disease-modifying therapeutics for a wide range of degenerative conditions. To fuel its drug discovery programs, Frequency is leveraging a PCA™ screening platform using primary human cells, including cochlear progenitor cells and progenitor cells from the GI tract. Potential applications include hearing loss, skin disorders and gastrointestinal diseases.

Frequency Therapeutics develops small molecule drugs to stimulate cells in the body to reverse biological deficits and restore healthy tissue. Through the transitory activation of these cells, Frequency enables disease modification without the complexity of genetic engineering. Our breakthrough therapy uses a proprietary combination of small-molecule drugs that induce dormant progenitor cells to multiply and create new cells. Our platform technology is founded on discoveries in progenitor cell biology by Bob Langer, Sc.D. at MIT and Jeff Karp, Ph.D., at Harvard, with contributions from Xiaolei Yin, Ph.D. and other members of the Karp Lab at Harvard and Brigham & Women's Hospital.

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