Aptorum Group Announces Further Positive Data on SACT-1 Against Neuroblastoma and Other Potential Tumor Types

HONG KONG, Sept 1, 2020 - (ACN Newswire) - - Aptorum Group Limited (NASDAQ: APM, Euronext Paris: APM) ("Aptorum Group"), a biopharmaceutical company focused on the development of novel therapeutics including orphan diseases and oncology indications, announced further positive data from its latest in vivo studies showing significant activity against neuroblastoma tumor reduction when treated with its lead compound SACT-1 in combination with standard of care (SOC) chemotherapy. Separately, SACT-1 was also screened for its in vitro activity against over 300 cancer cell lines and showed positive results in a number of cancer types including in particular colorectal cancer, leukemia and lymphoma, etc.

Our repurposed drug candidate, SACT-1 is undergoing preparation for IND submission and is on track for regulatory application to target to commence phase 1b/2a clinical trials under the US FDA's 505(b)(2) pathway.

"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. We are delighted to see the progress of our SACT-1, one of our first assets from our SMART-ACT platform. We are extremely excited to observe SACT-1's significant effect on tumor shrinkage when used in combination with standard of care chemotherapy in our latest in vivo studies. Moreover, we believe that SACT-1 may have potential applications in a number of other cancer types, including non-orphan cancers, which we will be continuing to investigate further for its wider application," said Dr. Clark Cheng, the Chief Medical Officer and Executive Director of the company.

Summary of our in vivo assessment against neuroblastoma and in vitro assessment against other cancers are discussed below.

Neuroblastoma In Vivo Assessment

Based on the initial 22 day data of a recent study we conducted in a xenograft mouse model of neuroblastoma, SACT-1 was orally administered daily at 60mg/kg in combination of SOC chemotherapy brought a statistically significant tumor shrinkage (unpaired student's t-test, p