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, 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 that the US 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 US 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 US. 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.
Source: Frequency Therapeutics