Frequency Therapeutics Announces Successful Completion of First-in-Human Study of FX-322, Company’s Leading Candidate for Hearing Restoration

January 1, 2018

WOBURN, MASSACHUSSETTS – Biotech startup, Frequency Therapeutics, announced late last month the successful completion of a first-in-human study of the company’s investigative compound FX-322, the company’s lead Progenitor Cell Activation (PCA) candidate for hearing restoration. The preliminary results were recently presented at a lecture last month at the US-Japan Symposium on Drug Delivery.

According to the company, FX-322 is a “proprietary combination of small molecules designed to transiently activate inner ear progenitor cells and enable the creation of new inner ear hair cells in the cochlea.”


“The goal of this landmark First-in-Human study was to prove the safety and tolerability of FX-322 at a dose that has been effective in restoring hearing in animals. This goal was achieved by showing that FX-322 was well tolerated with no drug related adverse events reported. Further, the results validated the feasibility of using a standard intratympanic injection to deliver FX-322 locally to the inner ear. In addition, we found that FX-322 successfully diffused from the middle ear to the perilymph fluid in the cochlea with minimal systemic drug exposure.”

–Stephen O’Leary, M.D., lead investigator for the study



Getting Closer to Cochlear Hair Cell Restoration in Humans?



The Phase 1 safety study, which enrolled 9 adult subjects with severe to profound sensorineural hearing loss, was conducted at the Royal Victoria Eye and Ear Hospital in Melbourne, Victoria, Australia.

The study participants received intratympanic injections of FX-322 within a 24 hour period prior to cochlear implantation. The results confirmed the safety and tolerability of the compound in the awake patient, and according to the company, “we were able to confirm the bioavailability both locally and systemically.”  


“FX-322 has transformative therapeutic potential, as it is designed to activate dormant progenitor cells in the inner ear so that they will multiply and mature into new, fully functional hair cells to restore hearing. This is the first time a progenitor cell activator has been looked at in humans, and these promising results bode well for the continued development of FX-322 for hearing restoration and other potential PCA™ therapeutics to address additional disease indications.”

–Peter C. Weber, M.D., Chief Medical Officer of Frequency


The successful results of the safety trial help lay the groundwork for future clinical trials in patients with moderate hearing losses that are not candidates for cochlear implantation.

Hailing the successful completion of the Phase 1 trial, President and CEO of Frequency Therapeutics, David Lucchino commented: “The completion of this study is a milestone for the clinical development of FX-322, a first-of-its-kind candidate with the potential to treat hearing loss for millions of people.”

Further information on the FX-322 301 study can be found here.


Frequency Announces Creation of Regenerative Medicine Advisory Board


Shortly after the company announced the completion of its first-in-human trial with FX-322, it also announced that it has brought together a group of renowned scientists and researchers with expertise in the fields of stem cell biology and regenerative medicine.

The newly created PCA Regenerative Medicine Scientific Advisory Board “will help guide and support Frequency’s leadership in advancing a next-generation approach to regenerative medicine with the potential to fully realize the power of the body’s own repair capabilities.”

Members of the Board include:


  • Sean Morrison, Ph.D., Director of the Children’s Medical Center Research Institute (CRI), UT Southwestern, the Mary McDermott Cook Chair in Pediatric Genetics, the Kathryne and Gene Bishop Distinguished Chair in Pediatric Research, and a Howard Hughes Medical Institute Investigator
  • Siddhartha Mukherjee, M.D., D.Phil., Assistant Professor of Medicine at Columbia University, Pulitzer Prize winning author
  • Lee Rubin, Ph.D., Professor of Stem Cell and Regenerative Biology at Harvard University, Director of Translational Medicine at the Harvard Stem Cell Institute
  • Amy Wagers, Ph.D., the Forst Family Professor of Stem Cell and Regenerative Biology at Harvard University


“We are honored to welcome this esteemed group of individuals to help steer Frequency’s scientific direction towards ground-breaking discoveries in multiple disease indications through the use of small molecules to activate the body’s repair processes,” said Chris Loose, Ph.D., Co-founder and Chief Scientific Officer of Frequency. 


Source: Frequency Therapeutics



  1. This is wonderful news for hard of hearing or deaf people who have not yet had cochlear implant surgery, as I have bilaterally. Even though it would not benefit me, perhaps people in future decades will be fortunate enough to have their hearing restored as a result of this research.

  2. I am a recording engineer for cbs

    I am close to going on disability for my hearing loss

    Is there any chance I could be part of the human trial for the cochlear drug?


  3. I am interested in participating in the human trial for the cochlear drug. I have severe hearing loss in one ear and some high frequency loss in the other. I am interested in knowing what would qualify a person to be a test subject.

    1. Reach out to the company. This is a website that reports on industry news and developments, not affiliated with this company.

  4. I would like to be a part of the clinical trials. I have severe hearing loss in one ear, and some high frequency loss in the other. I am 59 years old . I am a career musician , so some of my loss is from exposure to loud sound. I also think I have some loss due to orotoxic antibiotics.

    1. You should reach out to the company and see if that’s possible. This website is just reporting news.

  5. Hello! My 13 year old daughter on the 25th off December, woke up feeling her left ear clogged and started to hear differents sounds, went to ER they said she got fluid so antibiotics were prescribed. She’s has been dealing with ringing and stress/anxiety miss a lot of school, but catching up My question would be, When do you think this would be available? I really, really appreciate all your hard work. Thank you so much!! Sad Mom 🙁

    1. She needs to see an ENT straight away to rule out sudden sensoneural gearing loss not a general doctor! Steroids should be given immediately as there is only a small window to do so before damage becomes permanent.

      1. Hi Meg, yes after the first ER I went to 2 more clinics and no one knew that Steroids was the way to go.. 🙁 I already went to 1 ENT 3 weeks later, and he told me that her Colchear doesn’t work 🙁 and order MRI witch came out normal, after that we went exactly 1 month later to another specialist a Neurotologist/Otologist he give my daughter 3 shots in her ear drum, but still her left ear is severe.. I feel bad and mad that this doctors didn’t know how to treat this, I trust their knowledge, How is this possible that in the 21first century they just said ..We don’t know what cause this, is called Idiopathic..

        1. This is the same case i am having right now i lost left to profound right is at severe . only steriods prednisone helps but it should be reduced gradually. If it is reduced suddenlly . it gets worse than before only good doctors prescribes correctly

    2. Hi sad mom, I’m in the same boat as your daughter. I woke up one day with that clogged feeling went to numerous doctors, got prednisone, intratympanic injections, etc. Nothing worked and I have high frequency hearing loss along with loud tinnitus… Had anxiety/panic attacks. I have learned to cope with it but at times I feel suicidal. It’s been 6 years now and hoping something like this can help us and everyone with hearing issues.

  6. When do they plan on starting phase two of their trial? What will phase two be testing?

  7. My son who is 4 years old having hearing problem on both side which is profound not yet had implant surgery… wearing hearing aids….pls suggest me … I am really interested in knowing…. when will be available to us….sad mom…

    1. Sir can u tell me. it eligible for small childrens pls..reply me fast…

      1. Sir can u tell me. it eligible for small childrens pls..reply me fast…

        1. Reach out to the company. This is a website that reports on industry news and developments, not affiliated with this company mentioned in the article: Frequency Therapuetics.

          1. Sir….how will be available in India

      2. Sir
        I have otosclerosis problem in both ear stapedectomy operation in one ear but operation fail hearing further worst in operated ear using hearing aid in one ear from may 2013 can your medicine help me restoring my hearing please reply me and when your medicine will be available in market please inform me very anxious waiting for your medicine daily search your website please launch it quickly

  8. I am a 26 year old cochlear implant recipient. I became profoundly deaf at age 8 due to bacterial meningitis.

    Can FX-322 help in my case ? Do meningitis survivors still retain the cochlear “supporting” (progenitor) cells ?

  9. When the second phase clinical trials are going to start? How long it will take to launch the product into the market?

    1. I sure hope this gets on the market soon. It is hard for me to hear even with hearing aids and is very embarrassing. I can’t hear on the phone.

  10. I have profound hearing loss in left and severe in right if you need patients for teail please let me know

  11. I have profound on left and sever hearing loss at right . if u nned patient for trial please let me know

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