Tag Archive for: ion283

January 20, 2026 Update Fondazione Telethon logo

Telethon’s Update on the ION283 Clinical Trial Pathway for Lafora Disease

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Fondazione Telethon Logo

Fondazione Telethon has kindly provided Lafora patient organizations with a statement that includes some clarifications about ION283.

As they note, Telethon does not currently have a contract with IONIS. Until they have a contract for the license, they have no authority to submit a protocol to the EMA/FDA for a trial. If they are successful in gaining the license for ION283, they plan to:

  • Start a clinical trial as soon as possible
  • Cover all the clinical costs of the trial
  • Allow European patients currently in the Safety Study to complete their visits in Italy and continue receiving the drug after the study concludes

We appreciate the clarifications from Telethon! We will continue to update the Lafora community as we learn of developments for ION283.

As a reminder, please email your questions or concerns to ion283@chelseashope.org, and we will advocate on your behalf.

Read the statement from Telethon
ION283 Clinical Pathway Update January 8, 2026

Update on the ION283 Clinical Pathway

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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.”

We share your frustration at this setback. As we learn more information from partner organizations, we will share it.
We are very grateful that Fondazione Telethon has made itself available to step up to help develop the drug, as well as being so communicative with our community. Let’s remain united and hopeful!
Noventia Pharma logo

Noventia’s Reply to Lafora Patient Community

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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.”

Dear Noventia Pharma Team, We are families affected by Lafora disease who are holding on to hope through your work. Your dedication to developing a treatment for Lafora means everything to us. We were excited when you shared the news of the ION283 license purchase and your intentions to create a clinical trial last year, but we are anxiously awaiting more details. We know that any future progress with ION283 will come from your company, so we kindly request a public update on what is next for the drug’s development after the safety study at UTSW. As you know, the current ION283 safety study includes only 10 children. While we are grateful this study is happening, many more families were heartbroken to learn our children could not participate. These families are now left waiting, watching their children’s condition progress, and praying for the next opportunity. We are counting on Noventia to help make that opportunity possible. For many of us, your work is the only hope we have left. Our children do not have time to wait. Lafora disease is aggressive and unforgiving—we are pleading for a chance to save our children’s lives. We desperately urge Noventia to share your plans and move forward quickly with a clinical trial or expanded access program so more children can receive this potentially life-saving treatment. You have our full support in this mission, and we are willing to assist however we can.

Open Letter to Noventia

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Dear Noventia Pharma Team,

We are families affected by Lafora disease who are holding on to hope through your work. Your dedication to developing a treatment for Lafora means everything to us. We were excited when you shared the news of the ION283 license purchase and your intentions to create a clinical trial last year, but we are anxiously awaiting more details. We know that any future progress with ION283 will come from your company, so we kindly request a public update on what is next for the drug’s development after the safety study at UTSW.

As you know, the current ION283 safety study includes only 10 children. While we are grateful this study is happening, many more families were heartbroken to learn our children could not participate. These families are now left waiting, watching their children’s condition progress, and praying for the next opportunity. We are counting on Noventia to help make that opportunity possible.

For many of us, your work is the only hope we have left. Our children do not have time to wait. Lafora disease is aggressive and unforgiving—we are pleading for a chance to save our children’s lives. We desperately urge Noventia to share your plans and move forward quickly with a clinical trial or expanded access program so more children can receive this potentially life-saving treatment. You have our full support in this mission, and we are willing to assist however we can.

We would greatly appreciate it if you could share any updates with us or join our community for a Q&A. Some of our questions include:

  • How soon would expanded access be available for those not included in the trial?
  • What will be the initial phase of the clinical trial conducted by Noventia?
  • Which international sites will be involved?
  • Are those sites accredited to conduct a clinical trial?

We look forward to your prompt response and a clear outline of Noventia’s intentions regarding ION283. We are grateful for everything you do for the rare disease community and remain hopeful that Noventia will act swiftly to help more families in this fight against Lafora disease.

With hope,

The Lafora patient community

Patient organizations:

 
Asociacion CEL LUZ logo
Chelsea's Hope Lafora Children Research Fund

Lafora warriors and their families:

Hanifa Ishaque

Maggie Blatz

Anthony Caycho

Durmisevic Amar

Camila Bicca Oliveira

Caroline Bicca Oliveira

Prakash

Dr. Sagheer Hanif

Zarana Rathod

Tammie

Gonzalo Bruquetas

أم حسین الطباطبائي

Samy Sharif

Gladi Gidanian

Bilal Bhat

Bies Mahabier

Jamie Hennon

Marianne Rodriquez

Marina Weiss & Gajic Family

Georgianna

Spiros Georgakis

Paola Pacca

Romina Varesi

Silvana Parlato

Sami Memon

Marija Mrvosevic

Colleen Baumgartner-Hirsh

Simona Fochetti

Joan Monda

Babeth Letort

Jenifer Merriam

Recai yıldız

Marija Novakovic

Veronique Gadomski

Pierre Gadomski

Nuray yıldız

Fatos Malaj

Maha Hashim Alansari

Carmine Lettieri

Moniqueca Barfield

Jette Daubjerg

Elpida Georgaki

Nadira Belalouache

Fasal Alansari

Nora Belalouache

Hodosy Karol & Hodosy Viola Sheila Barter

Cheryl Bentley

Nicoletta Berti

Jette Daubjerg

Vanessa Lucas

Christophe Lucas

Kim Rice MD

Jim Rice MD

Tanja Weber

Antonello Giorgino

Daryoosh Moghaddam

Muhammad Malik

Mara Gerloni

Pasquale Esposito

Andi Müller

Anantha & Gopalakrishnan T

Korina Georgaki

 

Download the open letter to Noventia