AL-S Pharma has released new data from the phase 2 trial evaluating its drug AP-101 in patients with amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative disease that affects the motor neurons of the brain and spinal cord. Symptoms vary from person to person. Some forms of ALS begin with limb weakness, while others start with bulbar symptoms. All forms ultimately lead to loss of independence and a markedly shortened lifespan. Median survival remains three to five years, and diagnosis is often delayed.
AP-101 helps the body’s immune system clear misfolded superoxide dismutase 1 (SOD1) by inhibiting its spread in the central nervous system. The investigational human-derived antibody was given Orphan Drug Status by the US FDA, the EMA and Swissmedic.
Results from the global phase 2 clinical trial provide additional evidence of clinically meaningful disease modification and prolonged survival supported by reductions in key neuroaxonal injury biomarkers, serum neurofilament light chain (NfL) and cerebrospinal fluid phosphorylated neurofilament heavy chain (pNfH) after six months of treatment.
The trial met its primary endpoint relating to safety and tolerability. Adverse events were comparable to placebo and no AP-101-induced antibody responses were reported.
Angela Genge, chief medical officer of AL-S Pharma, said: “The data shows something we rarely see in ALS – objective evidence of clinically meaningful disease modification that tracks directly with prolonged survival.
“The phase 2 study results with AP-101 are consistent with the hypothesis that targeting misfolded SOD1 is a disease-modifying approach in ALS. We believe AP-101 could fundamentally transform the treatment of ALS.
“We look forward to continue advancing the AP-101 clinical programme so that we can bring a much-needed new treatment option to people living with ALS rapidly.”
AL-S Pharma aims to initiate a phase 3 clinical trial of AP-101in ALS at the end of 2026.