Astellas Secures $50M Option on AviadoBio Dementia Program

Astellas Pharma is making significant strides in the field of neurodegenerative disorders by investing $50 million in AviadoBio, a London-based biotechnology startup. This investment comprises a $20 million equity stake and up to $30 million in upfront payments. The agreement provides Astellas with an exclusive option to license AviadoBio’s AVB-101, an experimental gene therapy aimed at treating a specific form of frontotemporal dementia (FTD).

FTD is an early-onset dementia that severely affects cognitive functions such as attention control, memory, and problem-solving, typically resulting in death within three to 13 years of diagnosis. AviadoBio is targeting a version of this dementia caused by a mutation in the progranulin (PGN) gene, a protein essential for immune regulation in the brain.

Designed as a one-time treatment, AVB-101 aims to restore normal levels of progranulin in the central nervous system. The gene therapy involves delivering a functional copy of the PGN gene directly into the brain's thalamus, utilizing an adeno-associated virus (AAV) as a delivery mechanism. This approach has shown promise in preclinical studies, where it resulted in the expression of progranulin and reduced disease pathology in mice deficient in this protein.

AviadoBio was founded based on research from King’s College London and emerged from stealth mode in 2021 with $80 million in Series A funding. Its lead program, AVB-101, is currently undergoing a Phase 1/2 clinical trial, which began dosing its first patient earlier this year. The open-label study plans to enroll nine participants to evaluate the safety and efficacy of the gene therapy.

AviadoBio is not alone in its pursuit to treat FTD through progranulin restoration. Passage Bio is developing PBFT02, another gene therapy aimed at increasing progranulin levels, and has reached Phase 1/2 trials. Alector is taking a different route with AL100, an antibody drug designed to prevent progranulin degradation, which is currently in Phase 3 trials in partnership with GSK.

Astellas Pharma has identified gene therapy as a key growth area, particularly focusing on neuromuscular disorders, central nervous system conditions, and ophthalmology. This strategic direction was reinforced by the 2020 acquisition of Audentes Therapeutics, which brought a pipeline of gene therapies for various rare inherited disorders. However, some of Audentes’s programs faced challenges, including clinical holds and terminations for gene therapies aimed at Pompe disease and Duchenne muscular dystrophy.

In addition to the investment in AviadoBio, Astellas has previously engaged in option deals to expand its gene therapy portfolio. In 2021, the company made an equity investment in Taysha Gene Therapies, securing options to license gene therapies for giant axonal neuropathy (GAN) and Rett syndrome. Although Astellas did not exercise its option for the GAN program after Phase 1/2 data were reported, the option for the Rett syndrome program remains open.

Astellas has also entered other agreements to enhance its gene therapy capabilities. Last year, it licensed a preclinical gene therapy for X-linked myotubular myopathy from Kate Therapeutics and secured rights to use an AAV vector from 4D Molecular Therapeutics for ophthalmic diseases. The deal with AviadoBio includes potential applications of AVB-101 beyond FTD-GRN, with milestones and royalties that could total up to $2.18 billion if the option is exercised.

AviadoBio CEO Lisa Deschamps expressed optimism about the collaboration, highlighting its potential to address the significant unmet needs in frontotemporal dementia treatment. This partnership aims to leverage AviadoBio’s innovative gene therapy and delivery expertise with Astellas’s global development and commercialization capabilities.

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