The fast-track program aims to speed up the approval of therapies for unmet needs in serious conditions. Woburn, Mass.-based Frequency’s FX-322 candidate is designed to stimulate the regrowth of sensory hair cells to treat chronic noise-induced hearing loss. The technology is based on activating progenitor cells – mature stem cells that can proliferate into different types of cells. The compound is injected into the middle ear in a slow-release gel.
The 96-patient Phase 2a study is designed to compare the drug with a placebo in people with stable sensorineural hearing loss, randomized to three treatment arms and a placebo arm over four consecutive weekly treatments. The first cohort will will receive one FX-322 dose and three placebo doses; the second arm will receive two FX-322 doses and two placebo doses; the third group will receive three FX-322 doses and one placebo dose; and the control arm will receive four placebo doses, according to ClinicalTrials.gov. The study is quadruple masked, meaning neither the participants, healthcare providers, trial investigators nor outcomes assessors will know whether the dose is experimental or placebo. Top-line data are slated for the second half of 2020, Frequency said.
The trial’s safety endpoints include the number of patients with treatment-related adverse events, abnormal changes from baseline in otoscopic examinations and abnormal changes from baseline in tympanometry, all from screening to 210 days. The primary efficacy endpoints are word recognition, words-in-noise and standard pure-tone audiometry. Exploratory efficacy endpoints include extended high-frequency pure-tone audiometry, Tinnitus Functional Index and Hearing Handicap Inventory in Adults scores.
“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,” CEO David Lucchino said in prepared remarks. “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.”
FREQ shares were off by -0.8% at $13.20 apiece today in mid-morning trading, down -5.7% from their $14-per-share debut.