Dr. Giulio Frontino and Dr. Maurizio Delvecchio of IRCCS San Raffaele Hospital, Milan, Italy. Unified Care for Diverse Challenges: Crafting National Guidelines for WS from Diagnosis to Care. You can listen to the presentation by following the link provided. https://youtu.be/PssLjWZgsFE
Head to the link to listen to it now. https://youtu.be/zQ-WrsgEi3o
Dr. Fumihiko Urano’s research updates along with Amylyx Pharmaceuticals’ update on their ongoing clinical trial involving AMX0035. It’s an incredible chance to learn more about the latest advancements in this field. Listen to the updates by clicking on the link provided: https://youtu.be/WCUNVi90EGo
Tim Barrett update for Winter TreatWolfram newsletter
Dear friends and colleagues,
I hope everyone is keeping OK and managed to enjoy at least some good weather this Summer.
We are getting to the end of the TREATWOLFRAM trial, and the last participants will be having their last visits this October. We then hope to collect all outstanding data from study sites by the end of the year. The Clinical Trials Unit will then ‘lock’ the database so that our statistician can do the analysis. We expect to be told whether the treatment is effective or not, by the end of March or April 2025. As soon as I am told the results, I will write a report to our funder, the UK Medical Research Council; and I will of course share results through Wolfram Syndrome UK. Our trials unit team, Amy Lamb, Lewis James, Victoria Homer and Darren Barton, are working hard to make sure all the information that participants have kindly provided, will be ready to be analysed when the trial ends in October 2024.
Dr. Fumihiko Urano
Washington University School of Medicine, USA
Dear Friends,
I hope you had a wonderful summer. As the number of patients referred to us continues to grow, our team feels an even greater urgency to develop safe and effective treatments for Wolfram syndrome. I’m pleased to share the latest updates with you:
Ongoing Clinical Trial of AMX0035 in patients with Wolfram Syndrome
We have been collaborating with Amylyx Pharmaceuticals to advance the development of AMX0035, a novel oral medication with the potential to halt or delay the progression of Wolfram syndrome, based on its mechanisms of action and pre-clinical studies. The trial began in April 2023, and the interim results were announced in April 2024. According to Amylyx, the interim analysis of the ongoing Phase 2 clinical trial of AMX0035 for Wolfram syndrome, which involved eight participants assessed at Week 24, revealed encouraging outcomes. Participants demonstrated improvements in pancreatic function and glycemic control, as indicated by C-peptide levels and other markers of glucose metabolism. This contrasts with the typical disease progression expected in Wolfram syndrome. All eight participants met the prespecified responder criteria, showing either improvement or stabilization of their condition, as assessed by both the Patient Global Impression of Change (PGIC) and Clinician Global Impression of Change (CGIC) scales. Furthermore, the majority of participants reported some improvement in vision. Importantly, AMX0035 was generally well-tolerated by all participants, supporting its potential as a therapeutic option for this rare and devastating disorder.
Dr. Fumihiko Urano
Washington University School of Medicine, USA
Dear Friends,
Thank you sincerely for your invaluable support of our collaborative study alongside Prilenia Therapeutics.
One promising candidate is the Sigma 1 receptor, and we have initiated a collaboration with Prilenia, a biotech company specializing in this field. Together, we are investigating the effects of a drug called pridopidine, which targets the Sigma 1 receptor, in cellular models of Wolfram syndrome. We are grateful to the Snow Foundation in collaboration with Ellie White Foundation for their generous donations, which enables us to conduct this crucial study using cells derived from Wolfram syndrome patients. The study is currently in progress as we explore various experimental conditions to assess the efficacy of pridopidine. We are committed to providing you with regular updates on our progress.
AMYLYX PHARMACEUTICALS
Helios Update
Earlier in February 2024, Amylyx Pharmaceuticals announced that enrollment was completed in the Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol) for the treatment of Wolfram syndrome.
HELIOS is a 48-week exploratory, open-label proof of biology study assessing the effect of AMX0035 on a number of measures, including pancreatic function (mainly pertaining to diabetes), visual function, and Wolfram syndrome disease rating instruments.
The study, being conducted at Washington University in St. Louis, has enrolled 12 adult participants living with Wolfram syndrome, and preliminary results are anticipated in the second half of 2024. Data from this initial study will play an important role in giving Amylyx information needed to make decisions for potential future trials. Amylyx looks forward to sharing the results with the community, including at scientific meetings, when they are available.
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