Frequency Therapeutics Commences Dosing in its Phase 2a Study of FX-322 for Sensorineural Hearing Loss; FDA Grants FX-322 Fast Track Designation

WOBURN, Mass.--(BUSINESS WIRE)-- Frequency Therapeutics (Nasdaq: FREQ), a clinical-stage biotechnology company focused on harnessing the body’s innate biology to repair or reverse damage caused by a broad range of degenerative diseases, today announced that dosing has commenced in a Phase 2a clinical study of FX-322, a locally administered investigational drug candidate designed to regenerate auditory hair cells and restore hearing function. The objectives of the Phase 2a study are to further establish the positive hearing signal observed in Frequency’s Phase 1/2 study of FX-322, as well as continue to evaluate safety and define the potential dosing regimen. FX-322 is being developed for the treatment of sensorineural hearing loss (SNHL), the most common type of hearing loss.

The company also announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for FX-322. This designation is intended to facilitate development of new therapies for serious conditions with unmet medical need, expedite review, and enable more frequent engagement between the company and the FDA regarding study planning and design. At present, there are no FDA-approved medicines for SNHL. In a Phase 1/2 study, FX-322 demonstrated a statistically significant and clinically meaningful improvement in key measures of hearing loss, including clarity of sound and word recognition, with no serious adverse events observed.

“Starting our Phase 2a study shows continued momentum as we further our understanding of the potential of FX-322, both in measures of loudness and clarity, and work to develop a disease modifying treatment for the millions of people living with sensorineural hearing loss,” said David Lucchino, Chief Executive Officer of Frequency Therapeutics. “We are also pleased that the FDA has granted Fast Track designation for FX-322 and we look forward to continuing our ongoing work with the agency as our study progresses. We want to thank all of the patients participating in this study and the clinicians, audiologists and staff at sites across the U.S. for their efforts and collaboration.”

Frequency’s progenitor cell activation (PCA) approach uses small molecules to activate progenitor cells within the body to create functional tissue. Progenitor cells are present throughout the body and programmed to develop and differentiate into specific cell types within an organ. FX-322 is a combination of proprietary small molecules designed to restore hearing function by activating inner ear progenitor cells in the cochlea to induce hair cell regeneration and improve hearing function.

The Phase 2a clinical trial is a multi-center, randomized, double-blind, placebo-controlled, single- and repeat-dose study of FX-322, expected to enroll approximately 96 adults aged 18 to 65 with stable SNHL at 12 sites in the U.S. Patients will be randomized to one of four groups, each of which will receive four injections, once per week, at weekly intervals starting at the initial visit. The key efficacy endpoints of this trial are word recognition (WR), words-in-noise (WIN), and standard pure tone audiometry. Exploratory efficacy endpoints are extended high frequency pure tone audiometry, the Tinnitus Functional Index (TFI), and the Hearing Handicap Inventory in Adults (HHIA).

Top-line data from the study are expected in the second half of 2020.

Additional study information is available at clinicaltrials.gov. The identification number is: NCT04120116.

About Sensorineural Hearing Loss

Sensorineural hearing loss is the most common form of hearing loss, resulting from damage to the hair cells in the inner ear or problems with the nerve pathways that convert sound waves from the inner ear to the brain. Hair cells are commonly lost due to chronic noise exposure, or as a result of aging, certain viral infections or exposure to ototoxic drugs. The World Health Organization (WHO) estimates that there are currently more than 800 million adults with hearing loss globally and that 1.1 billion children and adults ages 12 to 35 years old are at risk for hearing loss from recreational noise exposure. According to the U.S. National Institutes of Health, more than 90 percent of those with hearing loss are affected by SNHL.

About Frequency Therapeutics

Frequency Therapeutics is a leader in the development of medicines designed to activate progenitor cells within the body to treat degenerative diseases. The company’s progenitor cell activation (PCA) approach stimulates progenitor cells to create functional tissue with the aim of developing disease modifying therapies. The company’s lead product candidate, FX-322, is designed to regenerate auditory hair cells to restore hearing function. In a FX-322 Phase 1/2 study, the company observed statistically significant and clinically meaningful improvement in key measures of hearing function in patients with sensorineural hearing loss. The company is evaluating additional diseases where its PCA approach could create functional tissue, including a development program in multiple sclerosis.

Headquartered in Woburn, Mass., Frequency has a development and commercialization agreement with Astellas Pharma Inc. for FX-322, for which it retains U.S. rights, as well as additional collaboration agreements with academic and nonprofit research organizations including The Scripps Research Institute, Massachusetts Eye and Ear, and the Massachusetts Institute of Technology. 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 intended purpose of Fast Track designation, the treatment potential of FX-322, the design and enrollment of the Phase 2a clinical trial of FX-322, the timing of top-line data from the Phase 2a clinical trial, estimates of the size of the hearing loss population and population at risk for hearing loss, and the potential application of the 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 company has incurred and will continue to incur significant losses and is not and may never be profitable; 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; the lengthy, expensive and uncertain process of clinical drug development and regulatory approval; 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; costly and damaging litigation, including related to product liability, 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 laws and regulations, including healthcare and environmental, health, and safety laws and regulations; failure to obtain, maintain and enforce protection of patents and other intellectual property; security breaches or failure to protect private personal information; attracting and retaining key personnel; and ability to manage growth.

These and other important factors discussed under the caption “Risk factors” in the company’s final prospectus filed with the Securities and Exchange Commission (SEC) on October 4, 2019 relating to its Registration Statement on Form S-1 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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