WOBURN, Mass.--(BUSINESS WIRE)-- Frequency Therapeutics, a clinical stage biotechnology company, today announced positive results from a Phase 1/2 safety trial to evaluate FX-322, an investigational drug candidate designed to facilitate hearing restoration. The trial assessed the safety of a single dose of FX-322 given by intratympanic administration in adult patients with stable sensorineural hearing loss (SSHL) who had a medical history consistent with either chronic noise exposure or idiopathic sudden sensorineural hearing loss. In the Phase 1/2 study, FX-322 was safe and well tolerated following a single intratympanic injection with no serious adverse events. In addition, improvements in hearing function, including audiometry and word scores, were observed in multiple FX-322 treated patients.

“Hearing loss affects millions of people and current treatments are unable to restore hearing once it is lost,” said Susan Marenda King, M.D., a Neurotologist at the Ear Medical Group in San Antonio, TX and lead enroller in the clinical trial. “The study results for FX-322, which show signs of functional hearing improvement, are very promising and represent an exciting step toward a potential treatment for patients.”

“While the focus of this study was safety, we are excited to see initial results in sensorineural hearing restoration as there are currently no treatments to restore hearing for these patients,” said David L. Lucchino, President, Co-founder and CEO of Frequency. “Furthermore, this data provides support for our small-molecule-driven regenerative medicine platform, which has the opportunity to address numerous degenerative diseases.”

Frequency plans to present the Phase 1/2 results at a major otolaryngology meeting in 2019 and to submit the results for publication. The company believes the results of this study support the advancement of FX-322 into a multiple dose Phase 2a study, which it plans to initiate later this year.

“If successful, our lead development program would represent the first time ever that hearing could be restored in humans with a therapeutic,” said Marc Cohen, Frequency’s Executive Chairman. “We are defining a new mode of regenerative medicine: activating progenitor cells within your body to repair damaged tissue.”

About the FX-322 Phase 1/2 Trial

The trial was a randomized, double-blind, placebo-controlled, single-dose, multi-center study to assess the safety of FX-322 in 23 patients with stable sensorineural hearing loss. Patients received an intratympanic injection of either a high-dose or low-dose volume of FX-322 or placebo in one ear. Patients were evaluated at an initial follow up visit two weeks following injection and were monitored for three months. The trial was conducted at several leading otolaryngology clinics in San Antonio, TX.

Information on the safety clinical trial can be found at clinicaltrials.gov with the identification number: NCT03616223.

ABOUT CHRONIC SENSORINEURAL HEARING LOSS

Virtually all hearing deficits in humans arise from damage and/or loss of sensory hair cells in the inner ear that convert sound waves into nerve impulses. In adult mammals, inner ear hair cells do not spontaneously regenerate following injury, although progenitor cells capable of regenerating hair cells remain present in the ear in a dormant state. There is no approved therapeutic option for chronic sensorineural hearing loss. Approximately 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 exposure. Hearing loss caused by prolonged exposure to excessive noise is observed in many professional environments, such as heavy construction sites or military training. In addition, repetitive exposure to everyday loud sounds associated with modern life (e.g. busy subway or rail systems, emergency vehicle sirens, musical concerts and the excessive use of headphones at high volumes) can also have a negative impact on hearing. Frequency’s therapeutic candidate for chronic sensorineural hearing loss, FX-322, is a proprietary combination of small-molecule drugs designed to transiently activate inner ear progenitor cells to create new hair cells and improve hearing.

ABOUT PCA REGENERATION

Tissue regeneration with Progenitor Cell Activation, or PCA Regeneration, is a new therapeutic approach designed to repair damaged tissue and restore healthy function in a less complex manner, while potentially providing safety advantages over traditional cell and gene therapy. The approach is based on discoveries in progenitor cell biology from the labs of Bob Langer, Sc.D., at MIT and Jeff Karp, Ph.D., at Harvard Medical School and Brigham and Women’s Hospital. PCA Regeneration utilizes small molecules in a manner designed to awaken dormant progenitor cells already in the body and carries the potential to address numerous degenerative diseases such as hearing loss, demyelinating diseases, skin disorders and gastrointestinal conditions.

ABOUT FREQUENCY THERAPEUTICS

Frequency Therapeutics develops small molecule drugs designed to stimulate progenitor cells already in the body to reverse biological deficits and restore healthy tissue. Through the transitory activation of these cells, Frequency seeks to enable disease modification without the complexity of genetic engineering. Our ground-breaking therapy uses a proprietary combination of small-molecule drugs designed to induce targeted progenitor cells to multiply and create new cells. While Frequency’s lead program targets hearing restoration, our PCA Regeneration platform has implications for many disease indications. www.frequencytx.com.

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