Raya Therapeutic Announces Selection of RT1999 (Smilagenin) onto the EXPERTS-ALS Clinical Trial Platform
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Positive data from EXPERTS-ALS could accelerate RT1999 towards a registration trial; RT1999 to be presented as a poster at the MDA Conference
MONTREAL, QUEBEC, CANADA, March 9, 2026 /EINPresswire.com/ -- Raya Therapeutic Inc. (“Raya”), a mission-driven company focused on the treatment of ALS and other neurodegenerative diseases, today announces that RT1999 (smilagenin), one of its five clinical-stage compounds from its Raya CARES pipeline, has been selected for inclusion in the EXPERTS-ALS (EXPErimental medicine Route To Success in ALS) Platform Trial led by the University of Sheffield and the University of Oxford. RT1999 is expected to dose its first patient before the end of 2026.
This selection marks a significant milestone as Raya advance the development of RT1999 for the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). It also represents an independent scientific endorsement of the compound’s potential as a disease-modifying therapy for ALS. Raya has previously received Orphan Drug Designations (ODD) from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ALS with RT1999.
About the EXPERTS-ALS Platform Trial
EXPERTS-ALS is led by Professor Chris McDermott at the University of Sheffield and Professor Martin Turner at the University of Oxford. It involves 11 clinical centres across the UK, soon to increase to 17, and is sponsored by Sheffield Teaching Hospitals NHS Foundation Trust. It is a flagship study within the broader portfolio of the UK MND Research Institute, funded by the National Institute for Health and Care Research (NIHR) and Biomedical Research Centre Capacity Building funding schemes. Patient charities MND Association, My Name‘5 Doddie Foundation, the Darby Rimmer Foundation, and medical research charity LifeArc are providing additional support.
The platform assesses potential therapies through a randomized, multicenter, open-label, multi-arm trial that evaluates investigational medicines through the measurement of the blood biomarker neurofilament light chain (NfL), which is significantly elevated in ALS patients and correlates with disease progression and survival. RT1999 will be primarily evaluated to determine its impact on NfL levels. Secondary and exploratory endpoints include clinical and survival measures.
About RT1999 (Smilagenin): Mechanism of Action
RT1999 (smilagenin) is an investigational, orally administered, non-peptide, small-molecule drug that readily crosses the blood–brain barrier. RT1999 acts as a neurotrophic factor modulator, dynamically regulating the synthesis of key neuroprotective proteins, including glial cell line-derived neurotrophic factor (GDNF) and brain-derived neurotrophic factor (BDNF), in response to neuronal stress signals. Importantly, RT1999 does not simply elevate GDNF and BDNF indiscriminately; rather, its effects are modulatory, restoring neurotrophic factor levels under pathological conditions while preserving homeostatic balance — potentially conferring a safety advantage over continuous exogenous neurotrophic factor administration. This mechanism provides a compelling rationale for its application in ALS, where the progressive loss of upper and lower motor neurons is a hallmark of disease pathology.
Strong Preclinical Evidence in ALS Models
RT1999 has demonstrated meaningful neuroprotective and disease-modifying activity across multiple preclinical models relevant to ALS, including in vitro studies in primary spinal motor neurons and in vivo studies in four established rodent models of ALS.
Clinical Evidence: Safety and Signals of Efficacy from Phase 2B Parkinson's Disease Trial
RT1999 has previously been evaluated in a large, rigorous Phase 2B clinical trial in Parkinson’s disease (the CONFIDENT-PD study), a 28-week, multi-center, randomized, double-blind, placebo-controlled, parallel-group study enrolling 425 treatment-naïve patients with early-stage Parkinson’s disease.
Although the study did not meet its primary endpoint, independent post‑hoc analyses identified clear signs of disease‑modifying activity. Using more sensitive modern statistical methods, RT1999 180 mg showed a meaningful slowing of motor and functional decline, with the strongest effects seen on pure motor symptoms. These findings provide encouraging evidence that RT1999 may slow the progression of motor neuron degeneration.
RT1999 Selected for Poster Presentation at MDA 2026 Conference
Additionally, Raya is pleased to announce that RT1999 (smilagenin) has been selected for a poster presentation at the upcoming 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 8–11, 2026 in Orlando, Florida. The poster (number 497 LB) is titled:
“RT1999, smilagenin, to proceed into Phase 2 clinical trial with EXPERTS-ALS”
Members of the Raya team will be in attendance at the MDA conference and welcome the opportunity to discuss RT1999 and Raya’s broader pipeline with interested stakeholders, collaborators, and the ALS community.
About Raya Therapeutic
Raya is a mission-driven company focused on the treatment of ALS, PD and other neurodegenerative diseases, leveraging the latest techniques for the selection and development of disease-modifying therapies. The company has a robust pipeline of five distinct clinical stage compounds (Raya CARES Pipeline) that each target different pathways involved in motor neuron degeneration in ALS. The compounds were in-licensed following a rigorous selection process based on biological plausibility, clinical target engagement and functional clinical effects reflective of efficacy. This diversified approach enables the development of combination therapies, which may have a significant impact on disease progression.
For more information, visit www.rayatherapeutic.com
About NIHR
The mission of the National Institute for Health and Care Research (NIHR) is to improve the health and wealth of the nation through research. We do this by:
- Funding high quality, timely research that benefits the NHS, public health and social care;
- Investing in world-class expertise, facilities and a skilled delivery workforce to translate discoveries into improved treatments and services;
- Partnering with patients, service users, carers and communities, improving the relevance, quality and impact of our research;
- Attracting, training and supporting the best researchers to tackle complex health and social care challenges;
- Collaborating with other public funders, charities and industry to help shape a cohesive and globally competitive research system;
- Funding applied global health research and training to meet the needs of the poorest people in low and middle income countries.
NIHR is funded by the Department of Health and Social Care. Its work in low and middle income countries is principally funded through UK international development funding from the UK government.
Anjan Aralihalli
Raya Therapeutic, Inc.
admin@rayatherapeutic.com
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