Company: APM
Filing Date: 2025-04-30
Form Type: 20-F
Source: 0001213900-25-037669
Chunk: 118

Company: Aptorum Group Ltd
Filing Date: 2025-04-30
Form: 20-F
Item: Item 4
Chunk 118
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 cancer, and classified as an orphan disease,
that forms in certain types of nerve tissue and most frequently in the adrenal glands as well as spine, chest, abdomen or neck, predominantly
in children, especially for those aged 5 years and below. For the high-risk group, which is close to 20% (Annu Rev Med. 2015; 66: 49 - 63.)
of total new patient population per year, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer
Society (https://www. cancer. org/cancer/neuroblastoma/detection-diagnosis-staging/survival-rates. html). The current high drug treatment
cost for high risk patients can average USD200,000 per regimen (all 6 cycles) (https://www. cadth. ca/sites/default/files/pcodr/Reviews2019/10154DinutuximabNeuroblastoma_fnEGR_NOREDACT-ABBREV_Post_26Mar2019_final. pdf). In addition, most pediatric patients often do not tolerate or survive the relevant chemotherapy stage which, subject to further clinical
studies, may be positively addressed by the SACT-1 candidate due to the potential synergistic effects when applied with standard chemotherapy.

In our studies, SACT-1 has
been shown to be effective against numerous neuroblastoma cell lines, of which 2 are MYCN-amplified cells, which represent the high-risk
neuroblastoma patient group. In addition, by using a bliss score as a quantitative measure of the extent of drug interaction, Aptorum
Group has seen a high and robust synergism between SACT-1 and traditional chemotherapy in vitro (Figure 4), indicating a potential efficacy
enhancement/dose reduction of the chemotherapy.

Figure 4

Figure 4: synergism between
SACT-1 and traditional chemotherapy in vitro

In addition, in our study,
the maximum tolerable dose of SACT-1 in a rodent model was determined to be higher than 400mg/kg. Compared with the MTD of standard chemotherapy
such as paclitaxel (20-30mg/kg) (Clin Cancer Res. 5(11):3632-8) and cisplatin (6mg/kg) (BMC Cancer 17: 684 (2017)), the safety profile
of SACT-1 appears to be very impressive. Based on our