WOBURN, MASSACHUSETTS — Biotech startup Frequency Therapeutics 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.
Fast Track 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.
Promising Results from FX-322 for Hearing Loss Treatment
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. 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.”
–David Lucchino, Frequency Therapeutics CEO
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.
ABC news story on Frequency’s FX-322 drug:
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.