Receive our newsletter – data, insights and analysis delivered to you
  1. Comment
April 6, 2021updated 07 Apr 2021 10:34am

LoA Update: AMO Pharma’s AMO-1 for PMS and epilepsy sees 13-point leap in regulatory potential following investigator-sponsored Phase II completion

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.

By William Newton

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 ClinicalTrials.gov.

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 Trials.gov. 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.

Related Companies

Content from our partners
Africa’s last mile: Building viable vaccine supply chains
Why this global life sciences COO believes relocation to Charleston, SC, was key to achieving next-level success
Patient-centric pharma logistics: How CRYOPDP delivers hope worldwide

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU