Meridian: Apellis APL2-ALS-206

Condition: Amyotrophic lateral sclerosis


Key Inclusion Criteria:

  • Sporadic amyotrophic lateral sclerosis (ALS) diagnosed as definite, probable, or laboratory-supported probable
  • At least 18 years old
  • Onset of ALS symptoms within 72 weeks prior to screening

Key Exclusion Criteria:

  • Confirmed or suspected other causes of neuromuscular weakness
  • Diagnosis of another neurodegenerative disease
  • Significant cognitive impairment, clinical dementia, or psychiatric illness that may increase individual’s risk or confound study outcomes

Study Name

A Phase II, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Subjects With Amyotrophic Lateral Sclerosis (ALS) (IRB no. 00001294)


This study focuses on individuals who have been diagnosed with amyotrophic lateral sclerosis (ALS) and are currently showing signs of weakness due to ALS. The purpose of the study is to determine whether an investigational drug called pegcetacoplan (also known as APL-2) can slow the progression of ALS. Researchers will assess the effects of the drug and evaluate participants for any side effects. The study will also evaluate how the body processes pegcetacoplan and how well it is tolerated. Participants will be randomly assigned to receive either pegcetacoplan or placebo (inactive substance).

Pegcetacoplan acts by turning down part of the immune system that helps remove disease-causing pathogens; this system is called the complement system. Under normal conditions, the complement system helps remove pathogens and damaged cells without hurting the body. However, uncontrolled or improper activation of the complement system can sometimes damage the body. Activation of the complement system is believed to play a role in the development and progression of ALS.

Principal Investigator