Raya Therapeutic announces RT1999 selection for EXPERTS-ALS trial
RT1999 (smilagenin) will dose first ALS patient by end of 2026. It received Orphan Drug Designations and shows preclinical neuroprotective activity.
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Biotechnology company developing ALS treatments
rayatherapeutic.comLast updated
In short: Raya advanced its ALS pipeline by securing a spot for RT1999 in the EXPERTS-ALS clinical trial and receiving multiple orphan drug designations.
RT1999 (smilagenin) will dose first ALS patient by end of 2026. It received Orphan Drug Designations and shows preclinical neuroprotective activity.
The pipeline features five clinical-stage compounds with unique mechanisms targeting ALS, PD, and neurodegenerative diseases. Each has established safety data. Raya plans Phase 2/3 trials within 12-18 months.
Raya highlights its pipeline of 5 pleiotropic NCEs for combination therapies in AD, PD, ALS, and FTD, with a planned ALS trial in 2027.
One of five clinical-stage compounds advances toward ALS approval in University of Sheffield-led platform trial. Positive data could lead to registration trial.
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...
Experimental therapy RT1999 will soon be tested in the UK EXPERTS-ALS platform trial designed to rapidly screen potential ALS treatments.
Positive data from this clinical study could lead to a registration trial of the compound for the treatment of ALS/MND. MONTREAL, QUEBEC, CANADA, September 2...
The orphan drug designations (ODDs) are from both the FDA & EMA and for the treatment of Amyotrophic Lateral Sclerosis (ALS). MONTREAL, QUEBEC, CANADA, March...
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