Positive Phase 1b Results for ARM210 in RYR1-Related Myopathies Treatment

ARMGO Pharma has announced the successful results of a Phase 1b clinical trial for Rycal® ARM210, a treatment aimed at Ryanodine Receptor 1 Related Myopathies (RYR1-RM). The study, published in eClinicalMedicine, demonstrated the safety and tolerability of daily doses of 120 mg and 200 mg of ARM210 in patients with RYR1-RM. Notably, the 200 mg daily dose showed promising signs of clinical efficacy by reducing fatigue and enhancing proximal muscle strength.
The research supports the allosteric mechanism of ARM210, which works to repair the mutated and 'leaky' Ryanodine Receptor 1 channels that are the root cause of RYR1-RM. The Phase 1b trial, a collaborative effort with the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institutes of Health (NIH), confirmed the drug's safety profile and pharmacokinetics over a 29-day period in adult patients with the disease.
ARM210's development will proceed to a Phase 2 trial, which will be a randomized, placebo-controlled study to further evaluate its potential as a disease-modifying treatment for RYR1-RM. The trial's positive outcomes are a significant step forward, offering hope to patients suffering from this chronic muscle disorder. ARMGO Pharma's CEO, Gene Marcantonio, M.D., Ph.D., expressed optimism about the trial's results and the drug's future development.
The RYR-1 Foundation, which supports research and advocacy for RYR1-RM, also welcomed the study's findings, viewing them as a promising development for the RYR1-RM community. ARM210 is also in Phase 2 clinical development for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), another orphan indication related to mutations in the RYR2 gene. The drug has received orphan drug designation for both RYR1-RM and CPVT from the FDA.

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