A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Apellis Pharmaceuticals' pegcetacoplan (APL-2) was evaluated in a Phase 2 clinical trial for amyotrophic lateral sclerosis (ALS). The study aimed to assess its efficacy and safety over 24 months. Despite the potential of pegcetacoplan as a complement inhibitor, the trial was terminated after failing to meet primary and key secondary endpoints at Week 52. This outcome raises concerns regarding the commercial viability of pegcetacoplan in the ALS market, which is characterized by limited treatment options and high unmet medical need. The competitive landscape includes established therapies like riluzole and edaravone, as well as emerging therapies targeting similar pathways. Investors and stakeholders should consider the implications of this trial's results on Apellis' pipeline and future strategic direction.
Indication: Amyotrophic Lateral Sclerosis
Modality: small molecule
Target: Complement component 3 (C3) inhibitor
Sponsor: Apellis Pharmaceuticals, Inc.
Source URL: ClinicalTrials.gov
Source updated: Detailed source ingestion pending
Ingested: Jul 21, 2026
Model: trialsignal-ai-v1
Validation: validated
Matched by target_normalized: Complement component 3 (C3) inhibitor
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Condition raw: Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Condition normalized: Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Modality raw: small molecule
Modality normalized: small molecule
Target raw: Complement component 3 (C3) inhibitor
Target normalized: Complement component 3 (C3) inhibitor