WOBURN, MASSACHUSETTS – Frequency Therapeutics, Inc. (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 presented clinical results from a longer-term durability study of FX-322, showing that some patients with chronic sensorineural hearing loss had significant improvement in key measures of hearing that were sustained for up to 21 months.
These new data build upon Phase 1/2 study results previously detailed at the 2019 AAO-HNS conference that showed, for the first time, statistically significant improvements in word recognition (WR) scores in patients with chronic moderate to moderately severe SNHL.
These patients had received a single intratympanic dose of FX-322 in a double-blind, placebo- controlled study, and had been evaluated for 90 days. Subsequent testing of a subset of these patients between 13 and 21 months after initial dosing, showed sustained improvements in measures of speech intelligibility (clarity), and in some patients, increased audibility (volume) threshold values at a higher frequency.
FX-322 Treatment: Evidence of Durable, Sustained Hearing Improvements
Susan King, M.D., FACS, the lead investigator on both studies, detailed results today at the AAO-HNS 2020 Virtual Annual Meeting & OTO Experience.
“Historically, patients with acquired chronic SNHL do not regain their lost hearing. The FX-322 Phase 1/2 study has provided promising evidence of clinically meaningful improvements in WR, and based on the recent longer-term follow-up data, we believe that this benefit may be sustained for many months following a single dose of this investigational treatment”
–Susan King, M.D., FACS, lead investigator
“It is exciting to see sustained clinical benefits in these individuals, and while still early, also to see the potential for a therapeutic that may provide sustained speech intelligibility improvement, which remains an unmet need for hearing loss patients,” continued Dr. King, co-founder of the Ear Medical Group in San Antonio and Clinical Professor, Department of Otolaryngology, Head and Neck Surgery, The University of Texas Health Science Center at San Antonio.
Four of the patients that were observed to have statistically significant WR scores during the Phase 1/2 study had maintained those improvements in the follow up assessment. Three of these patients remained at statistically significant levels maintaining an average of 87 percent increase in word recognition from their original baseline level. The other patient maintained a 71 percent increase from their initial baseline, though that sustained improvement fell outside the range of statistical significance. An additional patient who did not achieve statistically significant change in WR during the Phase 1/2 study was also retested, and it was observed that their WR score had returned to baseline.
Moreover, three of the patients that maintained statistically significant WR scores also showed 10-15dB improvements at 8000Hz compared to their original threshold values at this frequency. The safety profile observed in the FX-322 Phase 1/2 study was favorable and no additional safety findings were observed in the follow-up period. Together, these longer-term clinical data demonstrate the potential for FX-322 to have a durable positive impact both on measures of hearing clarity and volume.
“Today’s announcement is another important building block as we further our understanding of FX-322’s drug potential and the patient populations we hope to treat. In addition to a favorable safety profile, we have generated compelling cochlear drug delivery data, and these newly presented results show that the FX-322 benefit to hearing loss may provide individuals with a long-lasting clinical benefit.
–David L. Lucchino, Frequency Therapeutics CEO
“Furthermore, we believe this provides additional evidence supporting the regenerative mechanism of our novel therapeutic approach and a potentially disease modifying treatment,” said David L. Lucchino, Frequency’s chief executive officer. “We look forward to sharing data from our ongoing Phase 2a study and to a better understanding of the impact of FX-322 on sound clarity and word recognition that is so critical to hearing health and overall health.”
About FX-322 and Sensorineural Hearing Loss
FX-322 is Frequency’s lead product candidate for the treatment of acquired SNHL. SNHL impacts more than 40 million people in the U.S. and hundreds of millions of people globally and is the primary cause of more than 90 percent of all hearing loss. Acquired SNHL typically results from damage to the hair cells of the inner ear into signals that are sent to the brain. These auditory sensory hair cells may be lost due to chronic noise exposure, aging, certain viral infections or exposure to drugs that are toxic to the ear. FX-322 is designed to activate progenitor cells (pre-programmed stem cells) and regenerate auditory hair cells in the inner ear (cochlea) to restore hearing function.
FX-322 is currently being evaluated in a Phase 2a study. The FX-322 Phase 2a study is a randomized, double blind, placebo-controlled, single- and repeat-dose study in which the Company plans to enroll up to 96 patients aged 18 to 65 with SNHL. The objectives of the Phase 2a study are to further establish the hearing signal observed in the completed Phase 1/2 study, evaluate the impact of multiple doses and provide deeper insights on endpoints and the appropriate patient population for future studies. FX-322 Phase 2a study subjects are randomized to receive either FX-322 or placebo in one ear, with the untreated ear acting as an additional measure of control. The study is using validated measures of hearing function including word recognition (WR), words-in-noise and pure tone audiometry. Tinnitus and quality-of-life measures will also be evaluated using the Tinnitus Functional Index and the Hearing Handicap Inventory for Adults, respectively. The Phase 2a study has four dose cohorts, and hearing function will be regularly tested over the course of seven months following the first dosing.
The FX-322 Phase 2a study remains ongoing and the Company is expected to report study data in the second quarter of 2021.
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, statistically significant and clinically meaningful improvements in key measures of hearing function in patients with sensorineural hearing loss were observed. FX-322 is being evaluated in an ongoing Phase 2a clinical study in patients with sensorineural hearing loss. The Company also is evaluating additional diseases where its PCA approach could create functional tissue, including a discovery program in multiple sclerosis.
Headquartered in Woburn, Mass., Frequency has a license and collaboration agreement with Astellas Pharma Inc. for FX-322, for which it retains U.S. rights, as well as additional collaboration and licensing agreements with academic and nonprofit research organizations including The Scripps Research Institute, Massachusetts Eye and Ear, Mass General Brigham and the Massachusetts Institute of Technology.
Source: Frequency Therapeutics