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SACT-1

Development stage: Phase 1
Lead developer: Aptorum
Modality: Small Molecules
Administration: Oral

Overview

SACT-1 is an orally administered, repurposed small molecule drug developed by Aptorum Group for the treatment of neuroblastoma and other cancers. Its mechanism of action involves stimulating tumor cell death and inhibiting the N-myc proto-oncogene protein (MYCN), which is commonly amplified in high-risk or relapsed neuroblastoma patients. Preclinical studies have shown that SACT-1 reduces MYCN protein levels and decreases ERK5 phosphorylation, suggesting activity against MEK5/ERK5 signaling pathways. SACT-1 has received orphan drug designation for neuroblastoma and is being investigated as an adjunctive therapy to standard-of-care chemotherapy in relapsed or refractory high-risk neuroblastoma[1][3][4].

Other names: rilpivirine suspension

Targets

MYCN (N-myc proto-oncogene protein)

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