Biotech startup, Frequency Therapeutics, announced today that the first patients have been treated in a Phase 1/2 clinical trial to evaluate the company’s investigative compound FX-322, the company’s lead Progenitor Cell Activation (PCA) candidate for hearing restoration. The randomized, double-blind, placebo-controlled trial will assess the safety of FX-322 given by intratympanic administration in adult patients with stable sensorineural hearing loss (SSHL) who have a medical history consistent with either noise exposure or sudden hearing loss.

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.”

FX-322 Hearing Loss Drug Trial

The clinical trial is expected to enroll up to 24 patients at several leading otolaryngology clinics in the United States. Patients will receive either an injection of FX-322 or a placebo in one ear with an initial follow up visit after two weeks, and will continue to be monitored for the following three months.

“This Phase 1/2 trial builds on the success of our first-in-human study completed last year, which showed FX-322 to be well-tolerated and validated the potential for cochlear penetration when delivered using a standard intratympanic injection in patients scheduled for cochlear implant surgery. The clinical evaluation of FX-322 for hearing restoration is a priority for us based on the unmet medical need of millions of patients around the world who have lost varying degrees of hearing function from chronic noise exposure or sudden hearing loss, and we look forward to reporting the results from this study toward the end of 2018.” –Carl LeBel, Ph.D., Executive Vice President, Clinical Development

“This trial marks the first time a PCA drug candidate with the potential to restore hearing has ever been tested in adult humans and gives hope to the 48 million people in the U.S. with stable sensorineural hearing loss,” said David Lucchino, President, Co-founder and CEO of Frequency.

Akouos Raises $50 Million for Hearing Loss Gene Therapy R&D

BOSTON, MASSACHUSETTS — Biotechnology startup, Akouos, announced that it has raised $5o million in seed A funding. The company, which was spun out of the Harvard Innovation Labs, is developing novel therapies and delivery systems to prevent hearing loss and restore hearing in genetically defined populations.

The company is said to be the first to use a precision medicine approach to hearing loss.

The gene therapy being developed by Akouos is designed to work by using a special type of virus as the delivery vehicle for genetic material to target cells, delivered surgically to the inner ear via the round window membrane. The process is currently in the preclinical stage, but the company’s initial program is aiming for FDA review in 2019.

“The five year plan is to have multiple gene therapies in clinical development with a lead demonstrating clinical efficacy by that time” –Manny Simons, CEO of Akouos, in comments to Business Insider

Akouos also announced the formation of a scientific advisory board, which is made up of recognized experts in gene therapy and hearing loss:

Luk Vandenberghe, Ph.D. , chair and founder – director of the Grousbeck Gene Therapy Center, Massachusetts Eye and Ear; assistant professor of ophthalmology, Harvard Medical School

, chair and founder – director of the Grousbeck Gene Therapy Center, Massachusetts Eye and Ear; assistant professor of ophthalmology, Harvard Medical School Jean Bennett, M.D., Ph.D. – F.M. Kirby Professor of Ophthalmology, University of Pennsylvania

– F.M. Kirby Professor of Ophthalmology, University of Pennsylvania Michael McKenna, M.D. , founder – chief medical officer, Akouos, and the Joseph B. Nadol, Jr. Chair at Massachusetts Eye and Ear

, founder – chief medical officer, Akouos, and the Joseph B. Nadol, Jr. Chair at Massachusetts Eye and Ear William Sewell, Ph.D. , founder – professor of otolaryngology, Massachusetts Eye and Ear and Harvard Medical School

, founder – professor of otolaryngology, Massachusetts Eye and Ear and Harvard Medical School Richard Smith, M.D. , founder – founding director of the Iowa Institute of Human Genetics and the Molecular Otolaryngology and Renal Research Laboratories; professor of otolaryngology, pediatrics, medicine and molecular physiology & biophysics, University of Iowa

, founder – founding director of the Iowa Institute of Human Genetics and the Molecular Otolaryngology and Renal Research Laboratories; professor of otolaryngology, pediatrics, medicine and molecular physiology & biophysics, University of Iowa Aaron Tward, M.D., Ph.D. – assistant professor in residence, department of otolaryngology – head and neck surgery, University of California at San Francisco

The funding raised by the company will support the advancement of the first program into human studies. It will also help advance the pipeline behind it throughout clinical development and help build out the team.

Source: Frequency Therapuetics, Akouos