Frequency Therapeutics Presents Results Demonstrating Sustained Improvement in Hearing Loss Patients Treated with FX-322
Evidence of Durable Hearing Improvements in Follow-Up with Patients from Phase 1/2 Study; Speech Intelligibility and Audibility Sustained for Up to 21 Months After Initial Dosing
Data Highlighted at the 2020
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.
“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 word recognition, 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,” said
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 increase of 87 percent in WR scores from their original baseline levels. 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,” said
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
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.
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 Phase 2a clinical trial, pace and timing of enrollment for the Phase 2a clinical trial, the timing of top-line data from the Phase 2a clinical trial, the results and implications of the Phase 1/2 durability of response data, the ability of our technology platform to provide patient benefit, the impact of COVID-19 on the Company’s on-going and planned clinical trials and business, increases in headcount, future milestone and royalty payments under the license and collaboration agreement with Astellas, estimates of the size of the hearing loss population and population at risk for hearing loss, the Company’s ability to advance its hearing program and further diversify its portfolio 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 impact of COVID-19 on the Company’s ongoing and planned clinical trials, research and development and manufacturing activities, the relocation of the Company’s offices and laboratory facilities, the Company’s business and financial markets; the Company has incurred and will continue to incur significant losses and is not and may never be profitable; the Company’s 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 or intellectual property or brought by stockholders; dependence on Astellas Pharma Inc. for the development and commercialization of FX-322 outside of
These and other important factors discussed under the caption “Risk factors” in the Company’s Form 10-Q filed with the
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