Company: APM
Filing Date: 2025-11-17
Form Type: F-1
Source: 0001213900-25-111548
Chunk: 210

Company: Aptorum Group Ltd
Filing Date: 2025-11-17
Form: F-1
Chunk 210
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/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 (2017)). Based on our internal observations of pre-existing information from approved products, (subject to FDA’s approval and on a case-by-case basis, 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 Drug and reference the established safety and efficacy information. At 150mg/day, the death rate was 0% in prior clinical studies of the Reference Drug with no dosage related adverse events (Table 1). In addition, the pharmacokinetic profile of the approved product (i.e., Reference Drug) has also been reported (Table 2). 122 Table 1: Safety Profiles of the Reference Drug in Human Clinical Trials Table 2: The pharmacokinetic Profile of the Reference Drug in Humans Positive data from our latest internal in vivostudies show significant activity against neuroblastoma tumor reduction when treated with the compound SACT-