ARMGO Pharma, Inc. has made a revolutionary breakthrough in medicine with its Rycal ARM210, a small molecule drug set to change the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).
The first patient has been enrolled in a Phase 2 proof of concept clinical trial with ARM210, conducted by the renowned Amsterdam University Medical Center (Amsterdam UMC) in the Netherlands and the prestigious Mayo Clinic in Rochester, Minnesota. This trial aims to investigate the safety and efficacy of ARM210 in CPVT and promises to usher in a new era in the treatment of this potentially life-threatening condition.
CPVT is a rare genetic heart disease causing dangerous arrhythmias and affecting 1 in every 10,000 people. Sadly, the fatality rate of CPVT is high if left untreated: as many as 50% of individuals with the disease do not make it to the age of 40.
To understand the cause of CPVT, we look to mutations in RyR2, a crucial intracellular calcium-release channel. These mutations render the channel leaky, leading to the onset of the severe arrhythmias during times of exercise or stress.
ARM210 is an exciting development in the field of disease-modifying therapies, as it has the potential to treat CPVT by repairing dysfunctional RyR2 channels. A successful solution has been demonstrated in animal models, where high-resolution structures of mutant RyR2 have been used to stably bind and stabilize leaky RyR2 channels, restoring normal function to affected individuals. This unique therapy is also being studied for potential applications in other RyR-mediated cardiac and skeletal muscle diseases, such as Ryanodine Receptor-1 Related Myopathy (RYR1-RM).
ARMGO Pharma is thrilled to be taking a groundbreaking step forward in the CPVT community with its launch of the ARM210 clinical trial. This trial is a potential game-changer in the search for a disease-modifying treatment for CPVT, and is set to make big waves in the research of Ryanodine Receptors therapies.
ARMGO Pharma is confident in the success of this trial and is eager to commence the program with esteemed CPVT experts Arthur Wilde, M.D., Ph.D. and Michael Ackerman, M.D., Ph.D. The company is also committed to growing its portfolio of Rycal-based treatments for Ryanodine Receptor-related disorders. Gene Marcantonio, M.D. Ph.D., Chief Executive Officer of ARMGO Pharma, proudly stated: “This trial represents an important milestone for ARMGO and the CPVT community.”
ARMGO was awarded a major distinction by the FDA in 2020: an orphan drug designation and a rare pediatric disease designation for its experimental treatment ARM210, a potential therapy for those with CPVT.
The upcoming phase 2 trial of the drug is backed by the FDA through an Orphan Products Development (OPD) grant (1R01FD007279). This marks an exciting step forward in potentially transforming the lives of those living with the life-threatening condition.