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Ionis announces Biogen to advance salanersen into SMA registrational studies based on positive interim Phase 1 results
– Investigational salanersen (ION306/BIIB115) developed using novel Ionis antisense chemistry with the potential to achieve high efficacy and once-yearly
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Investigational salanersen (ION306/BIIB115) developed using novel Ionis antisense chemistry with the potential to achieve high efficacy and once-yearly dosing –\n\n\n– Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersen –\n\n\n– Biogen is engaging with regulators to advance salanersen to registrational stage studies –\n\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that its partner, Biogen, shared positive topline results from the Phase 1 study of salanersen (ION306/BIIB115), an investigational antisense oligonucleotide (ASO) being developed for the potential treatment of spinal muscular atrophy (SMA). Leveraging the same mechanism of action as SPINRAZA® (nusinersen) but designed to achieve greater potency, salanersen has the potential to achieve high efficacy and enable once-yearly dosing. Both dose levels tested, 40 mg and 80 mg, given once a year, were generally well-tolerated and led to substantial slowing of neurodegeneration, as shown by reductions in neurofilament. Exploratory clinical outcome data show clinically meaningful improvements in function and attainment of new World Health Organization (WHO) milestones over one year. These data will be presented today at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif.\n\n\nThe Phase 1 single ascending dose study was designed to evaluate the safety, tolerability and pharmacokinetics of salanersen. The trial consisted of two parts: Part A, a randomized and placebo-controlled segment in healthy adult male volunteers and Part B, an open-label segment in pediatric participants with SMA who previously received ZOLGENSMA® (onasemnogene abeparvovec) and had investigator-reported suboptimal clinical status. Interim results are from Part B (n=24) in individuals who received either 40 mg or 80 mg salanersen once a year. In participants with elevated baseline concentrations of neurofilament light chain (NfL), indicating ongoing neurodegeneration, initiation of salanersen led to mean reductions in NfL of 70% at 6-months which were sustained through the one-year dosing interval.\n\n\n“These encouraging interim results reflect the p...