Company: APM
Filing Date: 2025-12-05
Form Type: 424B5
Source: 0001213900-25-118752
Chunk: 222

Company: Aptorum Group Ltd
Filing Date: 2025-12-05
Form: 424B5
Chunk 222
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-ACT Drug discovery platform, we repurposed the Reference Drug to treat neuroblastoma. Neuroblastoma is a rare form
of 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 6cycles) (https://www.cadth.ca/sites/default/files/pcodr/Reviews2019/10154DinutuximabNeuroblastoma_fnEGR_NO REDACT-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