AMO Pharma announced initiation of patient recruitment in an interventional study of AMO-01, the company's investigational Ras-ERK pathway inhibitor, in the treatment of Phelan-McDermid syndrome. The clinical trial is being conducted by the Icahn School of Medicine at Mount Sinai (ISMMS) in New York.
"Treatment of Phelan-McDermid syndrome represents a significant area of unmet need in healthcare, and AMO Pharma is grateful to the research team at Mt. Sinai as well as the Phelan McDermid Syndrome Foundation for their commitment to this landmark research effort," said Michael Snape, chief executive officer of AMO Pharma. "Research thus far indicates that AMO-01 could have important applications in the treatment of patients living with Phelan-McDermid syndrome in the years ahead."
AMO Pharma will provide AMO-01 and grant funding for this investigator led study in subjects with Phelan-McDermid syndrome aged 12 years or older who have a history of epilepsy. Phelan-McDermid syndrome is a rare genetic condition caused by a chromosomal deletion. The most common characteristics found in these patients are intellectual disability of varying degrees, delayed or absent speech, symptoms of autism spectrum disorder, low muscle tone, motor delays and epilepsy.
Recent studies suggest that extracellular signal-regulated kinase (ERK) plays a critical role in synaptic plasticity (the ability of certain synapses to strengthen or weaken over time) and neurodevelopment. AMO-01 is an inhibitor of the Ras-ERK pathway. In pre-clinical efficacy studies, AMO-01 has been shown to rescue the neuronal phenotype of the multiple knockout mouse models of intellectual disability. This provides strong scientific evidence to support the conclusion that inhibition of the Ras-ERK cascade may have therapeutic benefit in the treatment of intellectual disabilities in humans.