Company: APM
Filing Date: 2025-07-15
Form Type: DRS
Source: 0001213900-25-063906
Chunk: 226

Company: Aptorum Group Ltd
Filing Date: 2025-07-15
Form: DRS
Chunk 226
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 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 4 Figure 4: synergism between SACT -1and traditional chemotherapy in vitro In addition, in our study, the maximum tolerable dose of SACT -1in 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 -1appears to be very impressive. Based on our internal observations of pre -existinginformation from approved products, (subject to FDA’s approval and on a case -by -casebasis, a 505(b)(2) Application can rely in part on existing information from approved products (such as the FDA’s previous findings on safety and efficacy) or products in literature (such as data available). However, typically speaking, the applicant is nonetheless required to carry out a Phase 1 bridging study to compare the Reference Listed Drug and reference the established safety and efficacy information), SACT -1also exhibits a well -establishedsafety profile: at 150mg/day, the death rate was 0% in prior clinical studies with no dosage related adverse events (Table 1). In addition, the pharmacokinetic profile of SACT -1has also been reported (Table 2). 122 Table 1: Safety Profiles of SACT -1in Human Clinical Trials Table 2: The pharmacokinetic Profile of SACT -1in Humans We have developed a pediatric formulation of SACT -1to better address the needs of neuroblastoma patients who are exclusively children younger than 5. Positive data from our latest internal in vivostudies show significant activity against neuroblastoma tumor reduction when treated with the compound SACT -1in combination with standard of care (SOC) chemotherapy. Separately,