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Synonyms: BIIB-115 | BIIB115 | ION-306 | ION306
Compound class:
Nucleic acid
Comment: Salanersen (BIIB115/ION306) is proposed as a survival motor neuron 2 (SMN2) synthesis inducer. It is a splice-switching anti-sense oligonucleotide (ASO) that is designed to restore normal SMN2 gene expression and translation of the SMN2 protein. This mechanism is intended to slow neurodegeneration and improve motor function in patients with infantile-onset spinal muscular atrophy, a condition that is caused by loss of functional SMN protein [1]. Salanersen is synthesised with 2'-O-[2-(methylamino)-2-oxoethyl] (NMA) ribose modifications to enhance its pharmacological properties compared to its predecessor nusinersen [2]. The full sequence is [2'-O-(2-methylamino)-2-oxoethyl](m5C-sp-A-m5C-sp-m5U-m5U-sp-m5U-sp-m5C-sp-A-sp-m5U-sp-A-sp-A-sp-m5U-sp-G-sp-m5C-sp-m5U-sp-G-sp-G-sp-m5C), as detailed in the INN record for the agent.
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Summary of Clinical Use  |
| Salanersen was progressed as a clinical candidate for the treatment of infantile-onset spinal muscular atrophy (SAM). It is proposed for once-yearly administration. The US FDA granted Breakthrough Therapy Designation in June 2026, to expedite salanersen's development and review for approval. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT07444476 | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam | Phase 3 Interventional | Biogen | ||
| NCT07444450 | A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec | Phase 3 Interventional | Biogen | ||
| NCT07221669 | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) | Phase 3 Interventional | Biogen | ||
