smilagenin (RT-1999) / Raya Therap - LARVOL DELTA

The Combination Of RT1999 And RT1972 From Preclinical To Clinical Development (ENCALS 2026) - "This study is intended to initially determine safety and tolerability of the combination in persons living with ALS, as well as identify biomarkers for the future development. Conclusion Currently presented preclinical data of the combination, and the previous clinical development of the compounds separately, are the foundation for future early-stage clinical development of this combination of RT1972 and RT1999 for the treatment of ALS."

Preclinical

RT1999 Is Neuroprotective Across Multiple Models Of ALS And Neurodegeneration (ENCALS 2026) - "Additional results from the in vivo and in vitro studies support neuroprotection at the level of the cell soma (motor neuron survival and TDP-43 pathology) and on axonal outgrowth (sciatic nerve crush and in vitro).Conclusion The presented preclinical and past clinical data (in Parkinson's disease) from RT1999 warrants further clinical development of RT1999 to assess its potential for the treatment of ALS. We are excited that this drug has been selected for the EXPERTS-ALS trial run in the UK."

Alzheimer's Disease • CNS Disorders • Movement Disorders • Parkinson's Disease • BDNF • TARDBP

Raya Therapeutic Announces Selection of RT1999 (Smilagenin) onto the EXPERTS-ALS Clinical Trial Platform (Firstwordpharma Press Release) - "RT1999 is expected to dose its first patient before the end of 2026....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."

Trial status • Amyotrophic Lateral Sclerosis

Raya Therapeutic Announces Oral Presentation and Poster at International Symposium on ALS/MND in Montreal December 6-8 (EIN News) - "Raya Therapeutic Inc...announces the presentation of the Phase 2 ROCK-ALS study data for its lead compound, fasudil (RT1968) for the treatment of Amyotrophic Lateral Sclerosis (ALS) by Dr. Paul Lingor (Principal Investigator) on Friday, December 6th at the 35th International Symposium on ALS/MND in Montreal...The company would also like to announce a poster presentation...entitled: 'The novel combination of 2 clinical-stage molecules demonstrates a beneficial effect in preclinical models of ALS.' Raya is also developing 2 novel small molecules, RT1972 and RT1999, each of which have neuroprotective effects in ALS preclinical models...Raya believes that this is the first presentation of a combination of two new chemical entities across multiple preclinical models of ALS."

P2 data • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders