ARMGO Pharma, Inc. (ARMGO), a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs often called Rycals®, announced today the enrollment of the primary patient in a Phase 2 proof of concept clinical trial using its Rycal ARM210 (also often called S48168), for the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The trial, performed on the Amsterdam University Medical Center (Amsterdam UMC), Netherlands and the Mayo Clinic, Rochester MN, USA will investigate the protection and efficacy of ARM210 in CPVT.

CPVT is a rare genetic heart disease causing arrhythmia. It affects 1:10,000 individuals with a fatality rate of 30-50% by the age of 40 if left untreated. CPVT is attributable to dysregulation of intracellular calcium handling in cardiomyocytes resulting mainly from mutations in Ryanodine Receptor 2 (RyR2), an intracellular calcium-release channel. Such mutations render RyR2 channels leaky, resulting in inappropriate channel opening during times of exercise or stress, leading to severe and sometimes fatal arrhythmias.

ARM210 is a possible disease modifying therapy for CPVT because it repairs leaky RyR2 channels. By binding and stabilizing the leaky channel, ARM210 can restore normal function, as demonstrated in animal models and in high resolution structures of mutant RyR2. Along with CPVT, this unique mechanism of ARM210 has potential use in other RyR-mediated cardiac and skeletal muscle diseases, akin to the genetic muscle disorder Ryanodine Receptor 1-Related Myopathy (RYR1-RM).

This trial represents a vital milestone for ARMGO and the CPVT community, potentially providing a disease modifying treatment for CPVT and validating ARM210’s unique mechanism of motion for Ryanodine Receptors” stated Gene Marcantonio, M.D. Ph.D., Chief Executive Officer of ARMGO Pharma.

“We’re looking forward to advancing this vital clinical program in partnership with the leading CPVT experts Arthur Wilde, M.D., Ph.D. and Michael Ackerman, M.D., Ph.D. and we’re confident within the success of this trial. As well as, we’re constructing our portfolio to strengthen ARMGO’s position as a pioneer of Rycal-based treatments for Ryanodine Receptor-related disorders.”

ARMGO was granted orphan drug designation in addition to rare pediatric disease designation by the FDA in 2020 for using ARM210 as a possible treatment for patients with CPVT. The phase 2 trial is supported partly by an Orphan Products Development (OPD) grant from the FDA (1R01FD007279). Further information concerning the trial could be found at https://clinicaltrials.gov/ with identifier – NCT05122975.