Trials of new drug for rare genetic heart disease enter Phase II

Heart attack patient with doctor

ARMGO Pharma has enrolled the first patient in a Phase II proof-of-concept clinical trial using its Rycal ARM210 (also known as S48168), for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT).

The trial, performed at the Amsterdam University Medical Center (Amsterdam UMC), Netherlands and the Mayo Clinic, Rochester MN, USA will investigate the safety and efficacy of ARM210 in CPVT.

CPVT is a rare genetic heart disease causing arrhythmia. It affects one in 10,000 people with a fatality rate of 30-50% by the age of 40 if left untreated.

CPVT is caused by 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, leading to inappropriate channel opening during periods of exercise or stress, resulting in severe and often fatal arrhythmias.

ARM210 is a potential disease modifying therapy for CPVT as it repairs leaky RyR2 channels. By binding and stabilising the leaky channel, ARM210 can restore normal function, as demonstrated in animal models and in high resolution structures of mutant RyR2.

In addition to CPVT, this unique mechanism of ARM210 has potential use in other RyR-mediated cardiac and skeletal muscle diseases, such as the genetic muscle disorder Ryanodine Receptor 1-Related Myopathy (RYR1-RM).

“This trial represents an important milestone for ARMGO and the CPVT community, potentially providing a disease modifying treatment for CPVT and validating ARM210’s unique mechanism of action for Ryanodine Receptors,” stated Gene Marcantonio, Chief Executive Officer of ARMGO Pharma. “We are looking forward to advancing this important clinical programme and are building our portfolio to strengthen ARMGO’s position as a pioneer of Rycal-based treatments for Ryanodine Receptor-related disorders.”

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