Aptorum Group Receives FDA Orphan Drug Designation for its SACT-1 Repurposed Drug For The Treatment of Neuroblastoma

NEW YORK & LONDON & PARIS--(BUSINESS WIRE)--Jan. 20, 2022-- Regulatory News:

Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) (“Aptorum Group” or “Aptorum”), a clinical-stage biopharmaceutical company, is pleased to announce 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. 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.

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.