The likelihood of approval (LoA) for AMO Pharma’s AMO-01 in Phelan-McDermid Syndrome (PMS) with co-morbid epilepsy jumped 13 points as of 29 March, according to GlobalData’s LoA data. The score change was based on the completion of an investigator-sponsored, 6-patient Phase II at Mount Sinai Hospital on 31 March 2020, noted by a 24 March 2021 update to

London, UK-based AMO Pharma’s open-label Phase II (NCT03493607), which has not yet published results, used the number of adverse events during the eight weeks following administration as its primary endpoint. Secondary endpoints include physician-measured efficacy assessments such as the Clinical Global Impression – Improvement Scale and the Visual Analogue Scale. Subjects received a single, 6-hour intravenous infusion of AMO-01 at the onset of the study, totalling 120 mg/m2 of treatment, according to Clinical AMO Pharma provided AMO-01 and grant funding for the investigator-sponsored trial, according to a May 2018 company press release.

While the LoA prior to this news was 5%, GlobalData’s analysis using a combination of machine learning and a proprietary algorithm has raised the LoA to 18%. AMO-01 inhibits the Ras-extracellular signal-regulated kinase pathway inhibitor, which could improve behavioural abnormalities and seizures in patients with the neurodevelopment disorder PMS.

William Newton is a Healthcare Reporter for Clinical Trials Arena parent company GlobalData’s investigative journalism team. A version of this article originally appeared on the Insights module of GlobalData’s Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.