Astellas Licenses AviadoBio Experimental FTP Gene Therapy

Rare Daily Staff

Astellas Pharma has secured an exclusive option and license agreement for AVB-101, AviadoBio’s experimental AAV-based gene therapy in phase 1/2 development for patients with frontotemporal dementia with progranulin mutations for up to $30 million upfront and the purchase of a $20 million equity stake.

Frontotemporal dementia with progranulin mutations (FTD-GRN) is a form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed.

Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. AviadoBio is also eligible to receive up to nearly $2.2 billion in license fees and milestone payments plus royalties if Astellas exercises its option.

The first clinical study, ASPIRE-FTD, is an open-label phase 1/2 study initiated in October 2023. Clinical trial sites are currently open in Poland, the Netherlands, Spain, and the United States, with additional sites planned to open in multiple countries.

In April 2024, AviadoBio announced that the first patient was treated with AVB-101 in the ASPIRE-FTD trial, and since then two additional patients have also been dosed.

“This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies,” said Lisa Deschamps, CEO of AviadoBio. “Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.”

Photo: Lisa Deschamps, CEO of AviadoBio