A new candidate treatment for motor neuron disease (MND) will be evaluated through the UK MND Research Institute’s innovative EXPERTS-ALS clinical trial platform.
Biotechnology company Raya Therapeutic Inc. has announced that smilagenin (RT1999), an investigational drug designed to protect nerve cells, has been selected for inclusion in EXPERTS-ALS. The first patient is expected to be given the drug before the end of 2026. This selection represents an independent scientific endorsement of RT1999’s potential as a disease-modifying therapy for ALS.
A faster way to identify promising treatments
Finding new treatments for MND is challenging, and traditional clinical trials can take many years to deliver results. EXPERTS-ALS was created to change that.
The platform uses an adaptive, biomarker-led design to test promising therapies in smaller groups of participants over a shorter timeframe, typically 6 to 9 months. By measuring levels of neurofilament light chain (NfL), a blood protein that reflects nerve cell damage, researchers can detect early signals of whether a treatment is having a biological effect.
This allows the team to make informed, more rapid decisions about which therapies should advance to large-scale trials. For people living with MND, where the average survival is just three years after diagnosis, this speed is critical.
About this new drug
RT1999 is an oral therapy that functions as a neurotrophic factor modulator, which regulates the production of neuroprotective proteins in response to neuronal stress signals. This approach preserves the body’s natural balance while protecting against disease conditions.
The drug has demonstrated meaningful neuroprotective and disease-modifying activity across multiple preclinical models relevant to ALS. In clinical testing, RT1999 was evaluated in a large Phase 2B trial in Parkinson’s disease, where post-hoc analyses identified clear signs of disease-modifying activity, including meaningful slowing of motor decline. Raya Therapeutic has received Orphan Drug Designations from both the U.S. FDA and European Medicines Agency for the treatment of ALS.
Strengthening coordinated trial infrastructure
EXPERTS-ALS is led by Professor Chris McDermott at the University of Sheffield and Professor Martin Turner at the University of Oxford, spanning 11 UK clinical centres, soon to increase to 17. The trial is funded by the National Institute for Health and Care Research (NIHR) and supported by major MND charities.
The inclusion of RT1999 demonstrates how collaboration and innovative trial design can accelerate the search for effective treatments. By bringing together researchers, clinicians, funders, and industry within a unified framework, the UK MND Research Institute is shortening the pathway from laboratory discovery to patient benefit.
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