Frequency Therapeutics, a progenitor cell development company based in Massachusetts, began a trial for hearing restoration using its small molecule drug candidate FX-322 from the company’s Progenitor Cell Activation (PCA) regeneration platform.
This will be the first time testing a progenitor cell activator on adult patients with stable sensorineural hearing loss (SSHL).
Carl LeBel, executive VP of clinical development at Frequency Therapeutics told us, “The primary focus of the study…is safety in a patient population with confirmed stable sensorineural hearing loss. However, the measurements we are using to assess safety are the same type of measurements that we’ll be using to assess efficacy in future studies, but there are no efficacy endpoints in this trial. Hearing improvement is not a goal of this study.”
“We believe our approach, PCA Regeneration, overcomes this barrier [not being able to access the inner-ear] and we validated our approach to delivering FX-322 to the inner ear in a first-in-human study last year,” LaBel continued.
While the previous study was the first-in-human study, this clinical trial will be the first to be conducted in adult patients with SSHL.
Currently around 48 million people in the US have SSHL. Pre-clinical work has shown potential for FX-322 to have significant impact in patients with noise-induced hearing loss.
Tissue regeneration with PCA, targets cells within the body instead of removing cells to deliver therapeutics, said LeBel.
Furthermore, he stated that other types of regenerative medicine had previously encountered issues in delivery, sustainability, and maintenance when trying to fix cells outside the body.
Progenitor cells are similar to stem cells but the cells can differentiate to form one or more kinds of cells; however, they cannot divide or reproduce infinitely. The cells do have the capacity to self-renew but a lower capacity than that of a stem cell.
This allows the cells to regenerate into a cell needed to treat the affected issue within the body, thus the body to redevelop the progenitor cells needed without manipulation outside the body.
“With PCA Regeneration, we’re taking that complexity out of the picture, leaving cells where they reside in the body, and using local delivery techniques to provide a trigger to awaken dormant cells and invite them to regenerate naturally,” he said.
PCA Regeneration is a new therapeutic approach that has potential to treat beyond hearing loss to areas like skin-related diseases, demyelinating diseases, type 1 diabetes, muscle regeneration and gastrointestinal diseases.
LeBel said, “This approach creates a regenerative response with the potential to repair damaged cells and restore healthy tissue in a less complex and potentially safer manner than traditional cell and gene therapy.”
The study is a randomized, double-blind, placebo-controlled trial. FX-322 will be given by intratympanic administration.