Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05626855 | Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab | PHASE3 | ACTIVE NOT_RECRUITING | 238 | — | — | Apr 17, 2023 | May 2, 2029 | May 25, 2025 | 49 | United States, Belgium +7 |
| NCT05156320 | Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam | PHASE3 | COMPLETED | 188 | — | — | Apr 14, 2022 | Dec 18, 2024 | Jan 22, 2026 | 50 | United States, Belgium +7 |
| NCT07047144 | A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy | PHASE2 | RECRUITING | 52 | — | — | Sep 15, 2025 | Mar 1, 2029 | May 1, 2026 | 25 | United States, Belgium +5 |
Incidence of TEAEs and SAEs by severity
The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
Apitegromab concentrations in serum
Total latent myostatin concentrations in serum
Change from baseline in the raw score of the Bayley Scale of Infant and Toddler Development, Fourth Edition - Gross Motor Subscale (BSID-4 GMS) at 48 weeks. BSID-4 GMS is a standardized assessment commonly used to evaluate development across 5 domains in infants and young children. It consists of 58 items, scored from 0 to 2 for each item, with higher scores indicating better gross motor development.
| Arm | Type | Description |
|---|---|---|
| Treatment Period | EXPERIMENTAL | Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period |
| Main Efficacy Population (Apitegromab 10 mg/kg) | EXPERIMENTAL | Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 10 mg/kg for up to 52 weeks. |
| Main Efficacy Population (Apitegromab 20 mg/kg) | EXPERIMENTAL | Aged 2-12 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. |
| Main Efficacy Population (Placebo) | PLACEBO_COMPARATOR | Aged 2-12 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. |
| Exploratory Subpopulation (Apitegromab) | EXPERIMENTAL | Aged 13-21 years at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks. |
| Exploratory Subpopulation (Placebo) | PLACEBO_COMPARATOR | Aged 13-21 years at Screening. Participants were randomized to receive placebo for up to 52 weeks. |
| Apitegromab low dose + SMN Therapy | EXPERIMENTAL | Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period. Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam). |
| Apitegromab high dose + SMN Therapy | EXPERIMENTAL | Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period. Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam). |
| Name | Type | Description |
|---|---|---|
| Apitegromab | DRUG | Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength. |
| Placebo | DRUG | Placebo was administered every 4 weeks by intravenous (IV) infusion. |
| Nusinersen | DRUG | Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information. |
| Risdiplam | DRUG | Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information. |
Inclusion Criteria: * Patients have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial. (For TOPAZ, completed is defined as completion of Visit EC14 in Extension Period C or participating in TOPAZ at the time the trial is ended. For SAPPHIRE, com...