AL-S Pharma tests how far SOD1 biology extends into sporadic ALS

Phase II data from AL-S Pharma suggest the biology of ALS may be less cleanly divided between SOD1-driven familial disease and TDP-43-driven sporadic disease than the field has often assumed. The results raise the possibility that some sporadic cases are also SOD1-driven; the task for Phase III will be to identify and stratify those patients.

At the AD/PD Alzheimer’s Disease and Parkinson’s Disease Conference in Denmark, Swiss biotech AL-S Pharma AG presented a fuller look at data from its Phase II study of AP-101, a mAb against anti-misfolded SOD1, in 52 patients with sporadic ALS and 21 with SOD1-mutant disease. The distinction matters because SOD1 has traditionally been seen as relevant mainly to the small subset of ALS patients with SOD1 mutations, while most sporadic ALS is thought to be driven by TDP-43 pathology...