Biogen is advancing its investigational therapy, salanersen, as a follow-up to Spinraza (nusinersen), into pivotal registrational (Phase III) studies for spinal muscular atrophy (SMA) after positive interim Phase I results.235
Salanersen is an antisense oligonucleotide (ASO) designed to be more potent than Spinraza, allowing for once-yearly dosing compared to Spinraza’s quarterly regimen.5
Interim Phase I data showed salanersen was generally well-tolerated, with most adverse events being mild or moderate (primarily pyrexia and upper respiratory tract infection).5
In the trial, salanersen led to a 70% mean reduction in neurofilament light chain (NfL) at six months, indicating a substantial slowing of neurodegeneration.5
Half of treated patients achieved new WHO motor milestones (e.g., walking, crawling, standing, sitting) that they previously could not accomplish or required assistance for, and experienced meaningful improvements on HFMSE and RULM motor function scales.5
Participants in the trial included pediatric SMA patients who had previously received Novartis’ gene therapy Zolgensma but still had suboptimal clinical outcomes.5
Sources:
2. https://ir.ionis.com/news-releases/news-release-details/ionis-announces-biogen-advance-salanersen-sma-registrational
3. https://firstwordpharma.com/story/5975970
5. https://www.clinicaltrialsarena.com/news/biogen-salanersen-advance-phase-iii/