We regret to share with our community that the biotechnology company Biogen has decided to discontinue development of their planned clinical trial for AGS. It’s our understanding that this decision has little to do with the potential and promise for this drug and that the company has discontinued a number of rare disease programs as a result of an updated business strategy. Drug development and clinical trials require huge investments of money and people, and the difficulty of doing this in the context of a poorly understood rare disease makes it all the more challenging. We’ve learned that drug companies need strong incentives to simplify clinical trials and that natural history data and clinical trial recruitment are two ways. For those reasons, we encourage AGS families to enroll in our data collection program with Citizen Health (https://agsaa.org/citizen) and join our global Contact Registry (https://agsaa.org/contact-registry). With a proper AGS census and health and medical data at the ready, we’ll be better prepared for the next opportunity.
We’d also like to share that work with drug at the center of Biogen’s clinical trial development could continue in spite of Biogen’s decision to walk away. The antisense oligonucleotide (ASO), developed by Ionic Pharmaceuticals (https://agsaa.org/news/2024/4/18/rna-targeted-therapy-offers-breakthrough-in-aicardi-goutires-syndrome), could be brought to a clinical trial by Ionis or another company with expertise in rare disease clinical trials. We’ll continue to pursue alternatives while helping to prepare our community for the demands of trial development.