Noventia replied to the open letter from the Lafora patient community that they were in the final stages of formalizing an agreement with Fondazione Telethon for the clinical development of ION283. Here are their answers to some of our questions:

“• Clinical Trial Phase: We are preparing to submit an application for a Phase 1 clinical study to the regulatory authorities by the end of this year. This study will primarily focus on safety, but will also include preliminary efficacy endpoints, and is expected to enroll 20 patients.

• Trial Sites: The coordinating center identified for the study is the IRCCS Institute of Neurological Sciences in Bologna, Italy, a center of excellence in this field. Should there be any delays in certification, we have contingency plans to involve one or more additional European centers to avoid unnecessary delays.

Regarding your question on site accreditation, in Italy, Phase 1 clinical centers must be certified under the specific AIFA regulation 809/2015. Our selected center in Bologna is in the process of obtaining this certification, which we anticipate will be finalized by the time of the study submission. For any potential international sites, while this Italian regulation does not apply, we will ensure they meet comparable high-quality standards. The thorough evaluation and selection of all clinical sites will be conducted as soon as the final protocol is ready.

• Transparency and Communication: We are committed to keeping the patient community updated. Once the clinical protocol is finalized, we will organize a dedicated meeting with patient representatives and associations to share all relevant details and answer your questions. Up to now, the Italian patient associations AILA and Tempo Zero have been regularly consulted and kept aligned through periodic meetings and updates. We will continue this approach, ensuring that all families remain informed as we move forward.”