News Release, January 31, 2024 – The RYR-1 Foundation, a leading nonprofit dedicated to advancing research on RYR-1-related diseases (RYR-1-RD), proudly shares the groundbreaking results of the Phase 1b clinical trial on Rycal® ARM210, also known as S48168. The trial focused on the treatment of Ryanodine Receptor 1 Related Myopathies (RYR1-RM) (also referred to as RYR-1-related diseases (RYR-1-RD)), an orphan neuromuscular disease. The RYR-1 Foundation played a pivotal role by providing crucial funding through a seed grant for the initial research that provided the justification for this transformative study. Rycals® were discovered by Dr. Andrew Marks, Chair of The RYR-1 Foundation’s Scientific Advisory Board.
Published in the journal eClinicalMedicine, the results demonstrated the safety, tolerability, and pharmacokinetics of 120 mg and 200 mg dosing of ARM210 daily in adult men and women affected by RYR-1-RD. The study, titled ‘Rycal S48168 (ARM210) for RYR1-related myopathies: A phase one, open-label, dose-escalation trial,’ authored by Dr. Joshua Todd et al., highlights the novel allosteric mechanism of action targeting the root cause of RYR1-RM: the mutated Ryanodine Receptor-1 (RYR-1). The principal investigator of this study and the senior author of the paper is Dr. Payam Mohassel, a member of The RYR-1 Foundation’s Scientific Advisory Board.
Dr. Mohassel shares, “This was truly a big team effort and wasn’t possible without the engagement of all the participants and their willingness to take it on, and the important partnership with The RYR-1 Foundation.”
Importantly, the Phase 1b trial demonstrated preliminary efficacy, particularly in the 200 mg dose group, showing alleviation of fatigue and improved proximal muscle strength in some of the trial participants. According to the authors, these promising results affirm the potential of ARM210 as a disease-modifying treatment for RYR-1-RD and warrant further development in a randomized, placebo-controlled Phase 2 trial.
Gene Marcantonio, M.D., Ph.D., Chief Executive Officer of ARMGO Pharma shared in ARMGO Pharma’s January 29 press release, “We are very pleased with the results of the RYR1-RM trial conducted together with the NIH, as the study has confirmed the safety and tolerability of ARM210, but most importantly, it has demonstrated for the first time that our Rycal®, ARM210, can reverse symptoms of this devastating, chronic muscle disease in a short treatment period. That is very promising.”
Michael F. Goldberg, M.D., M.P.H., Co-Chair of Research and Board President of The RYR-1 Foundation added, “We are elated by the publication of this important study, as it represents a beacon of hope for the many individuals and families from around the world who are affected by RYR-1-RD. The RYR-1 Foundation was instrumental in the execution of this clinical trial, not only by the funding of early research, but in its capacity as a patient advocacy organization. We look forward to the next stages in the development of this important drug.”
The RYR-1 Foundation expresses gratitude to the RYR-1-RD community and looks forward to continuing its collaborative efforts with ARMGO Pharma to bring hope and effective treatments to individuals and families affected by RYR-1-RD. For more information on the Phase 1b trial, please visit https://clinicaltrials.gov/study/NCT04141670.