Astellas Pharma and AviadoBio have entered into an exclusive option and licence agreement worth over $2bn for an investigational dementia gene therapy.
AviadoBio’s candidate, AVB-101, is currently in phase 1/2 development for patients with frontotemporal dementia (FTD) with progranulin mutations.
Astellas will be given the option to receive a worldwide exclusive licence for the development and commercialisation rights to the asset in FTD with progranulin mutations and other potential indications.
In exchange, Astellas will make a $20m equity investment in AviadoBio and up to $30m in upfront payments, with AviadoBio also eligible to receive up to $2.18bn in licence fees and milestone payments, plus royalties, if Astellas exercises its option.
Accounting for less than one in 30 dementia cases, FTD is a form of early-onset dementia that typically leads to death within three to 13 years from diagnosis.
Symptoms typically begin between the ages of 45 and 64 years, and patients commonly experience personality changes, uncharacteristic behaviours, loss of language, apathy and reduced mobility.
AVB-101 is delivered as a one-time treatment using a minimally invasive procedure directly to the part of the brain called the thalamus, which has extensive connections throughout the brain, to restore progranulin levels in the frontal and temporal cortex.
Astellas’ chief strategy officer, Adam Pearson, said: “We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases.
“AVB-101 represents a truly innovative approach to the treatment of FTD with progranulin mutations and has the potential to be part of the next generation of gene therapy products through the creation of this agreement.”
Also commenting on the agreement, AviadoBio’s chief executive officer, Lisa Deschamps, said: “This strategic collaboration will combine our promising gene therapy candidate for FTD with progranulin mutations and delivery expertise with Astellas’ global capabilities in development and commercialisation of gene therapies.
“Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD with progranulin mutations and other neurological diseases.”