We understand many families are wondering what’s next for the development of the ION283 drug. Unfortunately, we received an update today from A.I.L.A. and Fondazione Telethon that Noventia Pharma returned the license for ION283 and will no longer be bringing it to a trial.

Update on the ION283 Clinical Trial Pathway for Lafora Disease – Fondazione Telethon

“Dear Members of the International Lafora Patient Community,

First and foremost, we would like to thank you for your continued engagement, commitment, and trust: we are fully aware of the urgency faced by families affected by Lafora disease and of how deeply time matters in the context of a rapidly progressive condition.

For this reason, Fondazione Telethon shares your determination to move forward as quickly as possible, while ensuring that every step is taken responsibly, transparently, and in the best interest of patients’ safety and future access to ION283 treatment.

In light of the current situation, we would like to share an update on our ongoing efforts.

At present, Fondazione Telethon is actively working to establish a direct dialogue with Ionis, with two key priorities:

• • accelerating the start of the clinical trial as much as possible, and
  • obtaining additional scientific and clinical information on the investigational drug that is not yet fully available to us and that is essential to plan the next steps in a solid and informed way.

Our intention is to facilitate a pathway that allows the clinical program to move forward efficiently, while ensuring that decisions are based on the most complete and up-to-date data available.

We remain fully committed to transparency and open communication: as soon as further information becomes available and concrete timelines can be defined, we will promptly share updates with the international patient associations and the broader Lafora community.

We recognize the weight of expectations, concerns, and hopes carried by families every day: please be assured that Fondazione Telethon is working with the utmost sense of responsibility and urgency to help transform these hopes into a concrete and accessible clinical pathway.

Thank you for your continued collaboration and trust.”