Pegcetacoplan/APL-2

What does the drug do?

Pegcetacoplan is an investigational medication developed by Apellis Pharmaceuticals that inhibits the activity of a protein called C3, which is involved in a specific immune response called the complement system. Incorrect activation of the complement system can cause inflammation and damage to healthy motor neurons. C3 protein levels have been found to be increased at motor end plates (the junction between nerves and muscles) in those with MND. Pegcetacoplan is designed to block the action of the C3 protein and reduce inflammation which might help to slow disease progression in those with MND.

This drug has already been successful in several trials for a rare blood disorder so a phase 1 trial for it’s use in MND was not needed, as pegcetacoplan has already been found to be safe and well-tolerated.

Phase 2 - MERIDIAN

The phase 2 trial to assess the safety and efficacy of pegcetacoplan in MND, called MERIDIAN, is taking place at sites across the U.S, Australia and Europe, including two sites in the UK. This trial aims to recruit 228 participants and has two stages, each one lasting for a year. The first stage involves the participants being randomly assigned to receive either a placebo or pegcetacoplan for a year. The second stage of the trial will allow all participants to be treated with pegcetacoplan for a year. You can find out more about the trial here.

The trial has fully enrolled in the UK and Republic of Ireland.

Latest News

2020 - Dosing began in participants.

Last updated: 05/08/2022