On January 20, 2022 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported that the United States Food and Drug Administration (FDA) Office has granted Orphan Drug Designation to SACT-1, a repurposed small molecule compound for the treatment of patients with Neuroblastoma (Press release, Aptorum, JAN 20, 2022, View Source [SID1234605672]). Aptorum Group plans to file an Investigational New Drug Application (IND) to commence a phase 1b/2a clinical trial for SACT-1 to test the drug in neuroblastoma patients in 2022.
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Mr. Darren Lui, President and Executive Director of Aptorum Group says, "The granting of orphan drug designation for SACT-1 for the treatment of neuroblastoma is another important step forward in the development of our drug candidate and reflects both the FDA’s and Aptorum’s commitment to addressing the unmet clinical needs of patients with neuroblastoma." Further to our recently announced completion of Phase 1 clinical trial and patent grant for SACT-1, we are currently focusing on our IND preparation for entering into the exciting Phase Ib/2a clinical trials for SACT-1 in the United States."
About SACT-1
SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in our 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.
About Neuroblastoma
Neuroblastoma is one of the most prevailing solid tumor cancers in children, representing 8% – 10% of all childhood tumors, accounting for c. 15% of all cancer related deaths in the pediatric population1. For the high-risk patient group, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society2 based on existing treatment.
1 View Source,deaths%20in%20the%20pediatric%20population.