Frequency Therapeutics, Inc. (Nasdaq: FREQ), a clinical-stage regenerative medicine company focused on developing therapeutics to activate a person’s innate regenerative potential to restore function, today shared the results from its FX-322-113 study, a placebo-controlled trial evaluating the administration of FX-322 in subjects with severe sensorineural hearing loss (SNHL). In the study, FX-322 was associated with a hearing signal as shown by improvements by four subjects in a sentence-in-noise test.

FX-322-113 is a double-blind, placebo-controlled Phase 1b study designed to assess the local and systemic safety of a single dose of FX-322, and to evaluate hearing responses in a cohort of individuals with severe SNHL (pure tone average deficit between 71-90 decibel (dB) hearing level), a patient population that may have considerable damage to their inner ears and where cochlear implants may be the only potential intervention to improve hearing. Subjects were randomized 4:1 and received either FX-322 or placebo in one ear. Safety, otologic and audiologic assessments were conducted at days 30 and 90 following administration of FX-322 or placebo.

To gain a more comprehensive understanding of the potential impact of FX-322 in this population, the Company evaluated hearing function using multiple tests of speech perception in both quiet and noisy backgrounds, including the Bamford-Kowal-Bench Sentence-in-Noise exam (BKB-SIN). BKB-SIN is a validated test designed for severe SNHL populations (including cochlear implant patients), measuring the change in signal-to-noise ratios (SNR) required for a subject to correctly repeat words in a sentence.

In the FX-322-113 study, BKB-SIN test improvements were observed in four subjects, all of whom exceeded the 95 percent critical difference of 3.1 dB SNR, with two subjects showing a 6 dB response. A single placebo patient had a 3.6 dB change. In the study, subjects did not show substantial changes in speech perception measures in quiet, the safety profile in the study was favorable and there were no treatment-related serious adverse events reported.

“Given the level of cochlear damage, identifying single words in any audiologic test can be very challenging for these individuals. Response to the BKB-SIN test and a 6 dB improvement in the signal-to-noise ratio in multiple severe subjects is highly encouraging and consistent with the improvement we see in some patients following cochlear implantation,” said René Gifford, Ph.D., Professor and Director, Cochlear Implant Research Laboratory Department of Hearing and Speech Science at Vanderbilt University and a member of Frequency’s clinical advisory board. “This is the real-world equivalent to being able to halve the distance between two people in a conversation, which can be very impactful on an individual’s ability to communicate and engage in society. It is exciting to consider the potential of a restorative therapeutic as a component of the treatment paradigm for individuals with severe SNHL, either as a stand-alone treatment or in combination with hearing technology such as hearing aids and cochlear implants, to improve patient outcomes.”

Brian Monson, Ph.D., an assistant professor in the Department of Speech and Hearing Science in the College of Applied Health Science at the University of Illinois added: “Based on data pointing to FX-322 being localized in the higher frequency portion of the cochlea, as well as the proposed mechanism of action, these promising results are suggestive both of a hearing signal and potential functional improvements enabling speech perception in a noisy environment. These study findings are important as this outcome cannot currently be achieved using hearing devices that amplify sound, further highlighting the potential for a restorative intervention to enhance speech clarity.”  

FX-322 is Frequency’s lead hearing restoration product candidate and is designed to regenerate auditory hair cells to restore hearing function. Frequency currently is enrolling a new Phase 2b (FX-322-208) study of subjects with noise-induced or sudden sensorineural hearing loss, with hearing loss severities primarily in the moderate to moderately severe ranges (35 – 85 dB).

“This is the third FX-322 study where we have observed a hearing signal, furthering our confidence in our clinical program and the potential of a regenerative treatment for sensorineural hearing loss. The learnings from this study, and all of our early-phase studies to date, have provided Frequency and the field overall with important insights as we extend our understanding of how our therapeutic candidate may provide clinically meaningful benefit to the millions of individuals with SNHL that today have no restorative treatment options,” said David L. Lucchino, Frequency’s Chief Executive Officer.

About Frequency Therapeutics

Frequency Therapeutics is leading a new category in regenerative medicine that aims to restore function – first in hearing loss and then in multiple sclerosis (MS) – by developing therapeutics that activate a person’s innate regenerative potential within the body through the activation of progenitor cells to restore lost function. Frequency’s hearing research focuses on cochlear restoration and auditory repair, and its lead asset, FX-322, is a small-molecule product candidate that is the first to show statistically significant and clinically meaningful hearing improvements in clinical trials for SNHL. Frequency is also following early restorative signals in MS to develop medicines with the same underlying regenerative science being brought to hearing loss.

Headquartered in Lexington, Mass., Frequency has an ex-U.S. license and collaboration agreement with Astellas Pharma Inc. for FX-322, as well as additional collaboration and licensing agreements with academic and nonprofit research organizations including Massachusetts Eye and Ear, Mass General Brigham, the Massachusetts Institute of Technology, the Scripps Research Institute and Cambridge Enterprises Limited. For more information, visit www.frequencytx.com and follow Frequency on Twitter @Frequencytx.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the interpretation and implication of the results and learnings of FX-322-113, the timing and design of the new Phase 2b trial of FX-322, including the type of SNHL that the enrolled patients will have, the treatment potential of FX-322, the ability of our technology platform to provide patient benefit, the ability to continue to develop our Progenitor Cell Activation (PCA) platform and identify additional product candidates, and the potential application of the PCA platform to other diseases, including MS.

These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of COVID-19 on the Company’s ongoing and planned clinical trials, research and development and manufacturing activities, the Company’s business and financial markets; the Company has incurred and will continue to incur significant losses and is not and may never be profitable; the Company’s need for additional funding to complete development and commercialization of any product candidate; the Company’s dependence on the development of FX-322; the unproven approach of the PCA platform and the inability to identify additional potential product candidates; the lengthy, expensive and uncertain process of clinical drug development and regulatory approval; the Company’s limited experience successfully obtaining marketing approval for and commercializing product candidates; the results of earlier clinical trials not being indicative of the results from later clinical trials; differences between preliminary or interim data and final data; adverse events or undesirable side effects; disruptions at the FDA and other regulatory agencies; failure to identify additional product candidates; new or changed legislation; failure to maintain Fast Track designation for FX-322 and such designation failing to result in faster development or regulatory review or approval; ability to seek and receive Breakthrough Therapy designation for FX-322; the Company’s ability to enroll and retain patients in clinical trials; costly and damaging litigation, including related to product liability or intellectual property or brought by stockholders; dependence on Astellas Pharma Inc. for the development and commercialization of FX-322 outside of the United States; misconduct by employees or independent contractors; reliance on third parties, including to conduct clinical trials and manufacture product candidates; compliance with changing laws and regulations, including healthcare and environmental, health, data privacy and safety laws and regulations; failure to obtain, maintain and enforce protection of patents and other intellectual property rights covering product candidates; security breaches or failure to protect private personal information; attracting and retaining key personnel; and the Company’s ability to manage growth.

These and other important factors discussed under the caption “Risk Factors” in the Company’s Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 15, 2021 and its other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While the Company may elect to update such forward-looking statements at some point in the future, it disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.

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