Market Overview

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


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

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

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
, 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.

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.

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