David L. Lucchino, cofounder, president, and CEO of Frequency Therapeutics

Astellas Pharma will partner with Frequency Therapeutics to develop Frequency’s lead candidate—the Phase IIa-bound sensorineural hearing loss regenerative therapeutic FX-322—through a collaboration that could generate more than $625 million for the Woburn, MA-based developer of small-molecule, progenitor cell activation drugs.

Under an exclusive license inked by the companies, Astellas agreed to oversee development and commercialization of FX-322 worldwide except the U.S., where Frequency will assume those responsibilities.

FX-322 is a proprietary combination of small-molecule drugs designed to restore hearing function by activating inner ear progenitor cells already present in the body to induce hair cell regeneration. While FX-322 targets hearing restoration, Frequency says its Progenitor Cell Activation (PCA) platform may have the potential to touch upon a variety of therapeutic areas.

On April 9, Frequency announced positive results from a Phase I/II clinical trial (NCT03616223) that showed FX-322 to be well-tolerated following a single intratympanic injection, with no serious adverse events. Improvements in hearing function compared to placebo were observed in multiple FX-322 treated adult patients with stable sensorineural hearing loss, who had a medical history consistent with either chronic noise exposure or idiopathic sudden sensorineural hearing loss.

Frequency said it plans to launch a Phase IIa study of FX-322 in the fourth quarter.

The companies will jointly conduct global clinical studies and coordinating commercial launch activities for FX-311, hoping to win approval for the drug as the first treatment indicated for the most common type of hearing loss.

According to NIH statistics, approximately 15% of American adults (37.5 million) aged 18 and over reported some trouble hearing as of 2016—of which approximately 90% were affected by sensorineural hearing loss.

$80 million upfront

Astellas agreed to pay Frequency $80 million upfront, up to $545 million tied to achieving development ad commercial milestones, and royalties on any future ex-U.S. product sales.

“FX-322 is a program that focuses on the mechanism of regeneration,” Naoki Okamura, representative director corporate executive vice president, Chief Strategy Officer, Astellas, said Wednesday in a statement. “Astellas is committed to exploring all types of partnership opportunities to turn cutting-edge science and technological advances into value for patients.”

Regenerative medicine—including pluripotent stem cell products for patients with high unmet needs—is one of Astellas’ 10 areas of therapeutic interest. The other nine are oncology, urology, nephrology, immunology, neuroscience, muscle diseases, ophthalmology, vaccines, and gene therapy. Astellas is also interested in digital health and other healthcare businesses it calls “Rx+”.

“Collaborating with Astellas provides us an opportunity to work with a partner that has deep, global clinical development and commercial expertise and shares our focus in pursuing novel regenerative medicines for patients with diseases where there are no therapeutic options,” added Frequency CEO David Lucchino.

Founded in 2015, Frequency was established to develop small molecule drugs that activate progenitor cells within the body to restore healthy tissue. The company was formed to commercialize the PCA technology that emerged from discoveries by Bob Langer, ScD, of MIT and Jeff Karp, PhD, who holds positions at Harvard Medical School, the Harvard Stem Cell Institute, the Broad Institute, the Harvard-MIT Division of Health Sciences and Technology, and Brigham and Women’s Hospital.

Frequency’s science also builds upon research contributions by Xiaolei Yin, PhD, and other members of Karp’s lab at Harvard and Brigham & Women’s.

Speaking with GEN last year, Langer discussed how Frequency’s PCA technology compared with a competing approach under study by University College London’s (UCL’s) Ear Institute that entered clinical trials at about the same time as Frequency Therapeutics’ program. UCL’s approach, which incorporates a gamma-secretase inhibitor, forces stem cells to convert directly into other types of cells.

“We, however, engage a different part of the signaling pathway. Rather than force cells to become something different, we’re causing precursor cells to behave in a native manner, mimicking the biology that humans experience during development,” Langer explained.

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