On September 2, 2020 Aptorum Group Limited (NASDAQ: APM, Euronext Paris: APM) ("Aptorum Group"), a biopharmaceutical company specializing in the development of new therapies, particularly for orphan diseases and oncological indications, reported new positive results 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 chemotherapy therapy (SOC) (Press release, Aptorum, SEP 2, 2020, View Source [SID1234564334]).

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Separately, SACT-1 has also been studied for its in vitro activity against over 300 cancer cell lines and has shown positive results in a number of types of cancer including colorectal cancer, leukemia and lymphoma, etc. .

Our repositioned drug candidate, SACT-1, is in the preparation phase with a view to regulatory filing of an IND (Investigational New Drug) file with a view to considering the start of phase Ib / IIa clinical trials according to the 505 procedure ( b) (2) of the US FDA.

" Neuroblastoma is one of the most common solid cancer in children, representing 8 to 10% of all childhood tumors, about 15% of all cancer deaths in children 1 . For the high-risk group of patients, the 5-year survival rate for this disease is approximately 40-50%, as observed by the American Cancer Society 2 based on existing treatment. We are very happy to see the progress of our SACT-1, one of our first active ingredients from our SMART-ACT platform.. We are pleased to observe the significant effect of SACT-1 on tumor reduction when used in combination with standard chemotherapy in our latest in vivo studies. In addition, we believe that SACT-1 may have potential applications in a number of other types of cancer, including non-orphan cancers, which we will continue to study further for their wider application , "said Dr Clark. Cheng, Medical Director and Executive Director of the company.

The summary of our in vivo assessment against neuroblastoma and our in vitro assessment against other cancers is discussed below.

Assessment of neuroblastoma in vivo

Based on initial data from a recent study we conducted in a mouse model of neuroblastoma using a xenograft model, SACT-1 was administered orally daily at 60 mg / kg in combination with standard chemotherapy treatment (SOC), which resulted in statistically significant tumor reduction (unpaired student t-test, p <0.01) from day 15 to 22, compared to the control group who received treatment only standard (SOC) by chemotherapy. This is because the combination reduced tumor size by up to 54.2% in the first 22 days compared to the control group (SOC only). The combination of SACT-1 appears to be effective in accelerating effect of SOC in the early stages (day 1 to day 7 compared to the control group). This confirms our previous conclusions that SACT-1 is effective in accelerating the effect of SOC in the early stages (day 1 through day 7 compared to the control group). Which further reinforces our previous observationin vitro showing that SACT-1 promotes tumor DNA damage and tumor cell death.

Assessment of other types of cancer in vitro

In addition, the activity of SACT-1 was also examined in vitro in a panel of more than 300 cancer cell lines. As in our previous findings on neuroblastoma cell lines, SACT-1 exhibits similar anti-tumor efficacy for one or more other important cancer types, including, but not limited to colorectal cancer, leukemia, and cell lines. cell lymphoma. Therefore, besides treating neuroblastoma, SACT-1 may have potential applications in the treatment of other cancers. Based on this finding, the company plans to continue in vivo studies on the efficacy of SACT-1 against other types of cancers aimed at maximizing the potential of SACT-1.

About SACT-1

As part of Aptorum Group’s SMART-ACT platform, SACT-1 was discovered on our SMART-ACT platform focused on untreated orphan diseases. SACT-1 is a repositioned drug intended for the treatment of neuroblastoma (and potentially other types of cancer), particularly in combination with standard treatment (SOC) with chemotherapy. The mechanism of SACT-1 has been shown in vitro to improve DNA damage and tumor cell death.