Study of Therapeutic to Restore Hearing Aims to Show Speech Perception Improvements in Individuals with Noise-Induced or Permanent Sudden Sensorineural Hearing Loss
Company Aligned with FDA on Speech Perception Primary Endpoint
Readout Expected in Q1 2023
Frequency Therapeutics, Inc. (Nasdaq: FREQ), a clinical-stage regenerative medicine company focused on developing therapeutics to activate a person’s innate potential to restore function, today announced that it has completed enrollment of its placebo-controlled Phase 2b study of FX-322 in adults with acquired sensorineural hearing loss (SNHL). The FX-322-208 study, which enrolled 142 individuals, is designed to show improvement in a pre-specified measure of speech perception. The Company plans to release study data in the first quarter of 2023.
“I am very pleased with our team’s execution of this study for the first potential treatment to restore hearing for those with SNHL. The 208 study was rigorously designed to ensure the stability of an individual’s hearing prior to entering the trial and to exclusively enroll those with the types hearing loss where we observed the strongest hearing improvement in prior FX-322 studies. FX-322 continues to have a favorable safety profile and we are aligned with FDA on the primary speech perception endpoint. With a successful outcome of this single-dose study, our intent is to advance the program into Phase 3 trials,” said David L. Lucchino, Frequency’s chief executive officer.
Mr. Lucchino continued: “We are grateful to all the study volunteers, clinicians and site staff for their time and commitment to this trial. We believe the high level of interest from patients and healthcare providers in this study further demonstrates the need for a novel, disease modifying hearing loss treatment to expand the standard of care for the millions of individuals with sensorineural hearing loss.”
FX-322-208 Study Design
FX-322-208 is a prospective, randomized, double-blinded, placebo-controlled, multi-center Phase 2b study designed to evaluate the efficacy of a single administration of FX-322 on speech perception in subjects aged 18-65 with hearing loss associated with either noise-induced or permanent idiopathic sudden SNHL. The study enrolled 142 participants, exceeding the original enrollment target of approximately 124, and is being conducted at 28 clinical sites across the US.
The Company previously aligned with the US Food and Drug Administration (FDA) on the use of the specific speech perception primary endpoint. With improved speech perception, individuals may hear words more clearly, a critical unmet need for individuals with hearing loss. The FX-322-208 study is powered at 80% (significance level of 0.05) to observe a statistically significant and clinically meaningful improvement in speech perception at day 90 following dosing, with study responders defined as individuals exceeding the upper 95% confidence interval in the speech perception test. The Company has not publicly disclosed the specific test used for the primary endpoint to maximize the rigor of the study and mitigate potential bias.
During the study, subjects participate in a range of audiologic exams, including pure-tone audiometry, word recognition in quiet, word recognition in noise, the Tinnitus Functional Index (TFI), as well as multiple patient-reported outcome measures including Frequency’s proprietary patient reported outcome instrument (RADIAL) in acquired SNHL. All subjects are required to have a documented audiogram from at least six months prior to screening and most patients are evaluated over a 270-day period following dosing. The study’s rigorous design includes a lead-in phase with multiple baseline measures. Subjects with instability of baseline tests are disqualified from participation in the study. Study audiometry testing sessions are recorded and monitored by third party audiologists to ensure consistency and identify any anomalies related to how tests were conducted.
In prior studies, the Company observed the greatest concentration of speech perception improvements in individuals with permanent sudden or noise-induced sensorineural hearing loss in the moderate to lower severe hearing loss range. These learnings informed the design and inclusion criteria for the FX-322-208 study. More than 200 individuals have been dosed with a single injection of FX-322 in prior or ongoing studies, and the drug candidate has continued to exhibit a favorable safety profile with no drug-related serious adverse events.
About Sensorineural Hearing Loss
Sensorineural hearing loss is the most common form of hearing loss, typically resulting from damage to sensory hair cells in the cochlea. These cells convert sound waves to signals sent to the brain which are interpreted as speech and sound. Sensory hair cells are lost due to chronic noise exposure, aging, certain viral infections or exposure to drugs that are toxic to the ear. This type of hearing loss impacts around 40 million individuals in the U.S. alone.
About Frequency Therapeutics
Frequency Therapeutics is leading a new category in regenerative medicine that aims to restore human function – first in hearing loss and then in multiple sclerosis – by developing therapeutics that activate a person’s innate regenerative potential within the body through the activation of progenitor cells. Frequency’s hearing research focuses on cochlear restoration and auditory repair, and its lead asset, FX-322, is a small-molecule combination product candidate that is the first to show statistically significant and clinically meaningful hearing improvements in clinical trials for sensorineural hearing loss. 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, and the Scripps Research Institute.
For more information, visit www.frequencytx.com and follow Frequency on Twitter @Frequencytx.
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 timing and design of the Phase 2b study (FX-322-208), including the timing of results and the ability of design features to reduce bias, the commencement of any future FX-322 trials, the interpretation and implications of the results and learnings of other FX-322 clinical studies, the treatment potential of FX-322, estimates of the size of the hearing loss population, the acceptance by the FDA of particular endpoints in the Company’s trials, and the potential application of the progenitor cell activation (PCA) platform to other diseases.
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 August 9, 2022 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.