Aptorum Group Limited
Smart-Act - Drug Discovery Platform
A repurposed small molecule drug discovered from our SMART-ACT® platform, which has the potential to help develop drugs with well-established safety profiles in a time- and cost-effective manner
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orphan disease
- Targeted indication is neuroblastoma, an orphan disease predominantly occurs in children under 5
- In our studies, SACT-1 has been shown to:
- Enhance DNA damage and tumor cell death in vitro
- Promote neuroblastoma tumor reduction with standard of care chemotherapy in vivo
- Exhibit similar anti-tumor efficacy in vitro across major cancer types, such as colorectal cancer and triple negative breast cancer
- After completion of a Pre-IND Meeting with US FDA in February 2021, we are on track to open an IND to commence clinical studies in H2 2021, ultimately leading to NDA submission under 505(b)(2)