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Also known as BIIB115
Salanersen · 4 trials · 3 indications
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.
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.
An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A serious adverse event (SAE) is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
| 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. |
| Part A: Cohort 1: BIIB115 Dose 1 | EXPERIMENTAL | Participants will receive a single dose of BIIB115, Dose 1, via IT bolus injection, on Day 1. |
| Part A: Cohort 2: BIIB115 Dose 2 | EXPERIMENTAL | Participants will receive a single dose of BIIB115, Dose 2, via IT bolus injection, on Day 1. |
| Part A: Cohort 3:BIIB115 Dose 3 | EXPERIMENTAL | Participants will receive a single dose of BIIB115, Dose 3, via IT bolus injection, on Day 1. |
| Part A: Cohort 4: BIIB115 Dose 4 | EXPERIMENTAL | Participants will receive a single dose of BIIB115, Dose 4, via IT bolus injection, on Day 1. |
| Part A: Cohorts 1-4: BIIB115-Matching Placebo | PLACEBO_COMPARATOR | Participants will receive a single dose of BIIB115-matching placebo, via IT bolus injection, on Day 1. |
| Part B: Cohort 5: BIIB115 Dose 3 | EXPERIMENTAL | Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 3, via IT bolus injection at two separate time points. |
| Part B: Cohort 6: BIIB115 Dose 4 | EXPERIMENTAL | Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 4, via IT bolus injectionat two separate time points. |
| Part B: Long Term Extension (LTE): BIIB115 Dose 4 | EXPERIMENTAL | Pediatric SMA participants previously treated with onasemnogene abeparvovec who have completed Part B and are eligible will receive five doses of BIIB115, Dose 4, via IT bolus injection at separate time points for up to 60-month duration. |
| 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. |
| BIIB115 | DRUG | Administered as specified in the treatment arm |
| BIIB115-Matching Placebo | DRUG | Administered as specified in the treatment arm |
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...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 13 | PHASE3 | Nusinersen, Salanersen |
| Novartis AG Sponsored ADR | NVS | 7 | PHASE3 | Onasemnogene abeparvovec |
| Biohaven Ltd. | BHVN | 1 | PHASE3 | Taldefgrobep Alfa |
| Scholar Rock Holding Corp. | SRRK | 2 | PHASE3 | Apitegromab |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Hoffmann-La Roche | RHHBY | 2 | PHASE2 | Risdiplam, RO7204239 |
Salanersen is an investigational RNA therapy being developed for the treatment of spinal muscular atrophy (SMA), a neurological condition. It is being studied in people with genetically diagnosed SMA, including babies treated before symptoms appear, babies previously treated with onasemnogene abeparvovec, and participants aged 15 to 60 who are new to SMA treatment or were previously treated with risdiplam.
Salanersen is an antisense oligonucleotide, an RNA therapy belonging to the -sen class of antisense agents. It is designed to act on RNA rather than a protein target. Biogen is studying it for spinal muscular atrophy, a condition caused by deficiency of the SMN protein, though the specific RNA target has not been disclosed in the available program information.
Salanersen is developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen is also known as the developer of other SMA therapies. The drug is referred to as BIIB115 in company and trial documentation.
Salanersen is in Phase 3 clinical development for spinal muscular atrophy. It has not been approved by the FDA and remains investigational. The program includes four trials in total, all currently active, with three Phase 3 studies recruiting and one Phase 1 study active but not recruiting. Salanersen holds a Breakthrough Therapy designation from the FDA.
Salanersen is being studied in four trials. NCT07444476 is a Phase 3 study in participants aged 15 to 60 with SMA. NCT07444450 is a Phase 3 study in babies previously treated with onasemnogene abeparvovec. NCT07221669 is a Phase 3 study in babies treated before symptoms appear. NCT05575011 is a Phase 1 study in healthy adult males and pediatric participants.
Yes, salanersen and BIIB115 are the same investigational drug. BIIB115 is the code name used by Biogen Inc. in its clinical trial documentation, while salanersen is the international nonproprietary name. Both names refer to the same antisense oligonucleotide being developed for spinal muscular atrophy.