Today, Amylyx Pharmaceuticals provided an update on its plans to advance its oral compound ‘AMX0035’ through the clinical development process for the treatment of ALS. They have announced that they will submit a Marketing Authorisation Application to the European Medicines Agency (EMA). In addition, following promising results of the CENTAUR trial, Amylyx plans to initiate a phase 3 clinical trial in Europe and the United States. TRICALS is very excited to be collaborating with Amylyx on their phase 3 trial. The full press release can be accessed here.
To request marketing of a new drug in the United States, a pharmaceutical company needs to submit a ‘New Drug Application’ to the US Food and Drug Administration (FDA). The FDA has expressed an interest in seeing data from an additional placebo-controlled clinical trial prior to receiving a New Drug Application (NDA) from Amylyx for AMX0035. To meet this request and to continue to build upon previous data supporting AMX0035 for the potential treatment of ALS, Amylyx is preparing a phase 3 clinical trial in Europe and the United States.
TRICALS is looking forward to be working together with Amylyx on the European part of the study. Currently, TRICALS is the largest European research initiative to find a cure for ALS. Forty-four top research centres in 15 countries have joined hands with patient organisations and fundraisers to improve and accelerate ALS clinical trials. Together we aim to find the right treatment, for the right patient, at the right time.
“ALS is a devastating progressive disease that impacts patients and their families not only physically, but also mentally and emotionally,” said Leonard H. van den Berg, Professor of Neurology at UMC Utrecht in the Netherlands and Chairman of TRICALS. “After decades of ALS trial failures, AMX0035 has given us the hope that a new potential treatment option may be on the horizon for those living with ALS. We remain highly encouraged as AMX0035 continues to move through the regulatory review process, and are excited for the Phase 3 clinical trial.”
The trial is expected to begin enrollment in Q3 of this year. Amylyx also intends to submit a Marketing Authorisation Application to the European Medicines Agency (EMA) in Q4 of this year. This is necessary to obtain approval to market a medicine within the European Union.
We will post updates on our website and in our newsletter, once they are available. Would you like to receive the TRICALS newsletter? Click here to subscribe!
The CENTAUR Trial
CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. The trial was led by investigators at the Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium. As published in the New England Journal of Medicine (NEJM), the study met its primary efficacy endpoint of slowing ALS progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). The rates of adverse events were similar between the AMX0035 and placebo arms of the study. However, discontinuations related to adverse events occurred more frequently in the AMX0035 arm than in the placebo arm. You can read more about this study in our news article.