Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SBC-103 · 1 trial · 1 indication
TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A). A severe AE was defined as an AE that was incapacitating and required medical intervention. TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| SBC-103 | EXPERIMENTAL | Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first. Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C. Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg. |
| Name | Type | Description |
|---|---|---|
| SBC-103 | DRUG | - |
Key Inclusion Criteria: * A participant was greater than or equal to 2 years of age but less than 12 years of age at the time of informed consent. * Definitive diagnosis of MPS IIIB. * Documented developmental delay. Key Exclusion Criteria: * Received treatment with gene therapy at any time. * Pr...
SBC-103 is an investigational small molecule being developed for the treatment of Mucopolysaccharidosis IIIB, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
SBC-103 is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker symbol AZN. The drug is in Phase 1 clinical development for Mucopolysaccharidosis IIIB.
SBC-103 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. One Phase 1 clinical trial has been completed for this drug.
SBC-103 has one completed clinical trial, identified as NCT02324049. This Phase 1 study evaluated the safety, pharmacokinetics, and pharmacodynamics/efficacy of SBC-103 in patients with Mucopolysaccharidosis IIIB. The trial enrolled 11 participants across the United States, Spain, and the United Kingdom.
No alternative names for SBC-103 have been reported. The drug is being developed under the name SBC-103 by AstraZeneca PLC for the treatment of Mucopolysaccharidosis IIIB.