Company: APM
Filing Date: 2025-10-06
Form Type: S-4
Source: 0001213900-25-096656
Chunk: 251

Company: Aptorum Group Ltd
Filing Date: 2025-10-06
Form: S-4
Chunk 251
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 developed under the Smart -ACT® drug discovery platform. SACT -1is one of the Company’s proprietary technologies. The approved drug, rilpivirine, which we refer to as the “Reference Drug,” was developed for the treatment of HIV/AIDS (human immunodeficiency virus/acquired immunodeficiency syndrome). It works by inhibiting HIV reverse transcriptase, an enzyme that HIV uses to convert its RNA into DNA, which is essential for the virus to replicate and integrate into the host cell’s genome. By using the Smart -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 -riskgroup, which is close to 20% (Annu Rev Med. 2015; 66: 49 – 63.) of total new patient population per year, the 5 -yearsurvival 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 -riskpatients 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 -1candidate due to the potential synergistic effects when applied with standard chemotherapy. In our studies, SACT -1has been shown to be effective against numerous neuroblastoma cell lines, of which 2 are MYCN -amplifiedcells, which represent the high -riskneuroblastoma 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 -1and traditional chemotherapy in vitro (Figure 4), indicating a potential efficacy enhancement/dose reduction of the chemotherapy. Figure