Update on the ION283 Program from Telethon

Dear Members of the International Lafora Patient Community,

Following our previous communications, we would like to provide an important update regarding the ION283 program.

We have been informed that Ionis has decided to transfer the development rights of ION283 to another organization, different from Fondazione Telethon. As this decision rests entirely with Ionis, it is not under our control, as we have consistently clarified in our previous updates.

From the outset, our only priority has been — and will remain — to ensure that people living with Lafora disease have a real opportunity for a therapeutic option: in this spirit, we have donated all the work and materials developed over the past months to the organization identified to continue the program, in order to facilitate the fastest possible progress.

We are fully aware of the sense of urgency experienced by families and of how deeply time matters in the context of this disease. For this reason, we believe that enabling continuity — regardless of the actors involved — is the most responsible choice in the best interest of patients.

At the same time, we wish to reiterate Fondazione Telethon’s close commitment to the Lafora community: we will continue to support scientific research through the avenues that can best safeguard all patients, in full respect of internationally recognized standards, regulatory requirements, and the principles of scientific rigor and excellence.

Please be assured that we have done — and will continue to do — everything within our reach to support the Lafora community.

We remain close to you and committed to sharing further updates as soon as reliable information becomes available.

With respect and commitment,

Alessandra Camerini

Head of Relations with Patients and Patient Organisations