Breaking the mould?
More news this week from the EXPERTS-ALS platform.
EXPERTS-ALS, the first flagship project of the UK MND Research Institute, has been now operational for about 8 months. Five drugs are already in the queue for screening with 3 in play currently. The purpose of the platform is to accurately measure NfL biomarker change in an open label study over a maximum of 6 months. The primary ambition is an attempt to ‘break the mould’ of expensive phase II trials which typically take 3 years or more, often producing ambiguous results, only to fail in even larger and more expensive phase III trials. Let’s fail much earlier or preferably find better evidence, faster.
In their press release, on 2nd September 2025, Raya Therapeutic, a Canadian bio-tech company, announced that one of their 5 new investigational drug candidates is to be scheduled to use the platform. The candidate brings with it strong preclinical evidence that justifies the move to human study. Reflecting the globally growing interest in EXPERTS-ALS, this is likely to be only the first of many novel compounds screened.
This new commitment shows the urgency from Raya to gain a deeper understanding of their candidate drug to help them assess its true viability for our condition. We hope this is part of a new era where our community can see how much potential a drug has in the shortest possible time. We appreciate Raya both choosing the EXPERTS-ALS platform but also their willingness to signup to this new vision.
We believe that this reimagining of phase II trials has the potential to bring forward much better candidate drugs and speed their assessment.
United2EndMND
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