Two experimental ALS therapies showed promise in recent clinical developments. Interim Phase 1/2 data suggest the antisense oligonucleotide QRL-201 restores a critical nerve protein and may slow disease decline, with a Phase 3 trial planned for 2027. Meanwhile, the oral therapy neflamapimod has been selected for testing in the UK's EXPERTS-ALS platform study, with first results expected this spring.
The FDA granted Fast Track designation to COYA 302 for ALS. Phase 1 data showed tolerability and biomarker effects, and the phase 2 ALSTARS trial is underway.
JAMA Neurology published Phase 2b PARADIGM results for PrimeC in ALS, showing slower functional decline, fewer ALS-related complications and longer survival. NeuroSense is preparing a Phase 3 trial and received Brazilian patent protection through October 2042.
PrimeC, an oral combination therapy, showed safety and signals of clinical benefit in a phase 2b trial, while PLL001, a gut-targeting therapy, demonstrated positive safety and tolerability in a phase I/II study for amyotrophic lateral sclerosis.