AviadoBio Ltd. ("AviadoBio") and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

FTD is a devastating 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 etc.), 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. ^1,2,3,4

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. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option.

Lisa Deschamps, CEO, AviadoBio
"As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. 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. 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."

Adam Pearson, Chief Strategy Officer, Astellas
"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-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need."

1. Onyike CU and Diehl-Schmid J. Int Rev Psychiatry. 2013;25(2):130–137;
2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310; 
3. Onyike CU. Neuroepidemiology. 2011;37:166–167; 
4. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122;