AL-S Pharma Reports Positive Phase 2 Results of AP-101 in ALS

AL-S Pharma Reports Positive Phase 2 Results of AP-101 in ALS

By, Admin 5 September 2025

AL-S Pharma AG today announced positive topline Phase 2 results for AP-101, a first-in-class investigational antibody targeting misfolded SOD1, a pathological hallmark of amyotrophic lateral sclerosis (ALS).

Key Findings

  • Primary endpoint met: AP-101 demonstrated favorable safety and tolerability.
  • Clinically meaningful signals:
    • Improvements in survival and non-invasive ventilation outcomes.
    • Stabilization of disease staging and neurofilament biomarkers.
  • Broad relevance: First Phase 2 study of a SOD1-targeted therapy in both sporadic ALS and SOD1-mutation ALS.
  • Next steps: Results will be presented at upcoming ALS conferences and submitted to regulatory authorities later this year

Expert Commentary

“These Phase 2 results are an important step toward developing a disease-modifying therapy for ALS. We are grateful to participants, families, and global ALS experts who made this trial possible.”

  • Michael Salzmann, PhD, CEO, AL-S Pharma.

“This is the first controlled study testing a SOD1-targeted therapy across both sporadic and genetic ALS. The findings support misfolded SOD1 as a general disease driver in ALS.”

  • Prof. Peter Andersen, Umea University, principal investigator.

Upcoming Presentations

  • 36th International Symposium on ALS/MND
    - Top-line results of the Phase 2 proof-of-concept study of AP-101
    - Presenter: Prof. Dr. Angela Genge (McGill University, Canada)
    - Date: December 5, 2025 | Oral session
  • 2025 Annual NEALS Meeting
    - Baseline characteristics and analysis of misfolded SOD1 target levels in Phase 2
    - Presenter: Prof. Dr. Angela Genge
    - Date: October 2025 | Poster presentation

About AP-101

  • Mechanism: Human monoclonal antibody targeting misfolded SOD1, aiming to halt its spread in cerebrospinal fluid and spinal cord.
  • Indications: Sporadic ALS and SOD1-mutation ALS.
  • Regulatory status: Orphan Drug Designation granted by FDA, EMA, and Swissmedic.
  • Origin: Discovered using Neurimmune’s Reverse Translational Medicine™ (RTM™) platform.

About AL-S Pharma

  • Founded: 2016 by Neurimmune and TVM Capital Life Science.
  • Focus: Single-asset biotech advancing AP-101.
  • Funding: Backed by TVM Life Science Innovation I and Neurimmune.
  • Leadership: Includes experts from McGill University, Neurimmune, and TVM Capital.

Why It Matters

  • ALS remains a fatal neurodegenerative disease with very limited treatment options.
  • Targeting misfolded SOD1 could address a shared disease driver across both genetic and sporadic ALS.
  • Phase 2 data position AP-101 as a potential disease-modifying therapy, with regulatory engagement planned in 2025.

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