Aptorum Group Limited announced that the group has submitted the relevant Phase 1b/2a clinical trial protocol of SACT-1, anally administered repurposed small molecule drug for the treatment of neuroblastoma to US FDA. The Phase 1b/2a study of SACT-1 submitted is for the combination with chemotherapy for first relapse or refractory high risk neuroblastoma. This Phase 1b/2a trial represents another key milestone for the company and one of the targeted strategic goals for the year of 2023.

Neuroblastoma is a solid tumor arising in the nervous system outside of the brain predominantly in pediatric patients. The clinical behavior of neuroblastoma is highly variable with majority cases being highly aggressive. SACT-1 has the potential to effectively target this disease and address the unmet demands of such.

The targeted objectives of the Phase 1b part of the study based on neuroblastoma patients to be enrolled is to determine the recommended phase 2 dose (RP2D) based on safety, pharmacokinetics and efficacy and the Phase 2a part of the study based on Neuroblastoma patients to be enrolled will be used to assess the preliminary efficacy of SACT-1. SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed). SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.