By Fineline Cube 
Biogen (NASDAQ: BIIB) announced this week topline results from the Phase I study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) for the treatment of spinal muscular atrophy (SMA).
Drug Mechanism and Development
Salanersen shares the same mechanism as Spinraza (nusinersen) but is engineered for enhanced potency. This advancement could potentially enable once-yearly dosing. The interim analysis conducted to determine whether to advance salanersen into registrational studies focused on SMA patients previously treated with gene therapy.
Clinical Results
The drug demonstrated a favorable tolerability profile and significant slowing of neurodegeneration. This was evidenced by reduced neurofilament levels in patients tested at both 40 mg and 80 mg (annual administration).
Collaboration and Future Plans
Developed by Ionis Pharmaceuticals, Inc., salanersen was licensed to Biogen for global development, manufacturing, and commercialization. Biogen is currently engaging with global health authorities to finalize the Phase III study design.-Fineline Info & Tech