Frequency Therapeutics Initiates Phase 1/2 Single Dose Clinical Trial to Evaluate FX-322, a First-in-Class Drug Candidate for Hearing Restoration

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Trial marks the first time a potential progenitor cell activator will be tested in adult patients with stable sensorineural hearing loss

Frequency Therapeutics
This trial marks the first time a PCA drug candidate with the potential to restore hearing has ever been tested in adult humans and gives hope to the 48 million people in the U.S. with stable sensorineural hearing loss

Frequency Therapeutics today announced that the first patients have been treated in a Phase 1/2 clinical trial to evaluate FX-322, a first-in-class drug candidate for hearing restoration from the company’s Progenitor Cell Activation (PCA) regeneration platform. The randomized, double-blind, placebo-controlled trial will assess the safety of FX-322 given by intratympanic administration in adult patients with stable sensorineural hearing loss (SSHL) who have a medical history consistent with either noise exposure or sudden hearing loss.

“This trial marks the first time a PCA drug candidate with the potential to restore hearing has ever been tested in adult humans and gives hope to the 48 million people in the U.S. with stable sensorineural hearing loss,” said David Lucchino, President, Co-founder and CEO of Frequency. “By using a proprietary combination of small molecules intended to awaken dormant progenitor cells, we are creating an entirely new therapeutic modality with the potential to activate the body’s existing regenerative capacity and ultimately cure diseased tissue without the complexities of traditional cell or genetic therapy. We are thrilled to launch this important trial with the support of our investigators, multiple clinical sites in the U.S. and most importantly the patient volunteers.”

The Phase 1/2 trial is a randomized, double-blind, placebo-controlled, multi-center study of FX-322, which will be administered in patients with stable sensorineural hearing loss. The trial is expected to enroll up to 24 patients at several leading otolaryngology clinics in the United States. Patients will receive either an injection of FX-322 or a placebo in one ear with an initial follow up visit after two weeks, and will continue to be monitored for the following three months.

“Hearing loss is a condition that has historically not received much attention despite the widespread nature of the issue, due in part to the difficulty of access to the inner ear environment,” said Dan Lee, M.D., Pediatric and Adult Otology and Neurotology, Massachusetts Eye and Ear Infirmary, Associate Professor of Otolaryngology, Harvard Medical School, and Chairman of Frequency’s Clinical Advisory Board. “The early work done by Frequency has shown real potential for FX-322 to have a significant impact in patients with noise induced hearing loss. While this is a safety study, it is an important first step to examining FX-322 in adult patients with sensorineural hearing loss.”

“This Phase 1/2 trial builds on the success of our first-in-human study completed last year, which showed FX-322 to be well-tolerated and validated the potential for cochlear penetration when delivered using a standard intratympanic injection in patients scheduled for cochlear implant surgery,” said Carl LeBel, Ph.D., Executive Vice President, Clinical Development of Frequency. “The clinical evaluation of FX-322 for hearing restoration is a priority for us based on the unmet medical need of millions of patients around the world who have lost varying degrees of hearing function from chronic noise exposure or sudden hearing loss, and we look forward to reporting the results from this study toward the end of 2018.”

Information on the Phase 1/2 clinical trial can be found at with the identification number: NCT03616223.

Virtually all hearing deficits in humans 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. There is no approved therapeutic option for chronic noise-induced hearing loss. 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. 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.

Tissue regeneration with Progenitor Cell Activation, or PCA Regeneration, is a new therapeutic approach to repairing damaged tissue and restoring health function in a less complex and potentially safer manner than traditional cell and gene therapy. The approach, 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, utilizes small molecules to awaken dormant progenitor cells already in the body and has the opportunity 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 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 ground-breaking therapy uses a proprietary combination of small-molecule drugs that induce progenitor cells to multiply and create new cells. While Frequency’s lead program targets hearing restoration, our PCA Regeneration platform has the potential to touch upon a wide breadth of disease indications.

Jeffrey S. Rosenbloom, M.D. is actively enrolling volunteers to participate in this study. If interested, please contact Alamo ENT Associates at 210-545-0404.

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Jeff Holland
Alamo ENT Associates
since: 11/2013
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