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Salanersen · 3 trials · 2 indications
The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.
The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
| Arm | Type | Description |
|---|---|---|
| Salanersen | EXPERIMENTAL | Participants will receive a single dose of salanersen, 80 milligrams (mg) administered intrathecally on Day 1 in Part A. In Part B, participants will continue to receive four additional intrathecal doses of salanersen, 80 mg administered on Days 365, 730, 1095, and 1460. |
| Sham Procedure | SHAM_COMPARATOR | Participants will undergo a sham procedure on Day 1 in Part A. In Part B, participants will receive four intrathecal doses of salanersen, 80 mg administered on Days 365, 730, 1095, and 1460. |
| Treatment-Naïve Cohort | EXPERIMENTAL | Treatment-naïve participants will receive salanersen 80 milligrams (mg) by intrathecal (IT) lumbar puncture (LP) every 12 months for a total of five doses. |
| Risdiplam-Treated Cohort | EXPERIMENTAL | Risdiplam-treated participants will receive salanersen 80 mg by IT LP every 12 months for a total of five doses. |
| Name | Type | Description |
|---|---|---|
| Salanersen | DRUG | Administered intrathecally |
| Sham Procedure | PROCEDURE | A sham lumbar puncture is a skin-only needle prick at the usual lumbar puncture site. The needle does not enter the spinal canal. |
Key Inclusion Criteria: * Genetic documentation of 5q spinal muscular atrophy (SMA) homozygous gene deletion or mutation or compound heterozygous mutation. * 2 copies of the survival motor neuron 2 (SMN2) gene. * Onasemnogene Abeparvovec (OA) dose given at ≤ 42 days of age and screening initiated l...
Salanersen is an investigational antisense oligonucleotide being developed for spinal muscular atrophy (SMA), a genetic neuromuscular disease. It is being studied in presymptomatic babies with a genetic diagnosis of SMA, in babies previously treated with onasemnogene abeparvovec, and in adults aged 15 to 60 years with SMA who are new to treatment or previously treated with risdiplam.
Salanersen is an antisense oligonucleotide (ASO), a class of small molecules that work by binding to RNA to modify gene expression. Its specific molecular target is not publicly disclosed in the available clinical trial information.
Salanersen is being developed by Biogen Inc., a biotechnology company traded on the Nasdaq under the ticker symbol BIIB. Biogen is conducting Phase 3 clinical trials of Salanersen across multiple countries, including the United States, Australia, Brazil, China, and Japan.
Salanersen is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug has received Breakthrough Therapy designation from the FDA, which is intended to expedite the development and review of drugs for serious conditions.
Salanersen is being studied in three Phase 3 trials. NCT07221669 enrolls presymptomatic babies with genetically diagnosed SMA. NCT07444450 enrolls babies previously treated with onasemnogene abeparvovec. NCT07444476 enrolls participants aged 15 to 60 years with SMA who are new to treatment or previously treated with risdiplam. All trials are recruiting.
Yes, Salanersen is also known as BIIB115. Clinical trial records refer to the drug as Salanersen (BIIB115), indicating that BIIB115 is the development code used by Biogen for this investigational antisense oligonucleotide.