Company: APM
Filing Date: 2025-07-15
Form Type: DRS
Source: 0001213900-25-063906
Chunk: 216

Company: Aptorum Group Ltd
Filing Date: 2025-07-15
Form: DRS
Chunk 216
---
 can mean slightly different things to different researchers or companies, but in this document, we refer to Lead Discovery as the process of identifying one or more small molecules with the desired activity against the identified targets. Leads can be identified through one or more approaches, which can depend on the target and what, if any, previous knowledge exists. • Lead Optimization: In this stage of the drug discovery process, the aim is to produce a preclinical drug candidate by maintaining the desired and favorable properties in the lead compounds, while repairing or reducing deficiencies in their structures. For example, to optimize the chemical structures to improve, among others, efficacy, reduce toxicity, improve metabolism, absorption and pharmacokinetic properties. • CTA -Enabling Studies: Includes all the essential studies such as GLP toxicology studies, pharmacology and efficacy, pharmacokinetics, in vitro metabolism, CMC studies, and the data of which are used for CTA submission. • IND -Enabling Studies: Includes all the essential studies such as GLP toxicology studies, pharmacology and efficacy, pharmacokinetics, in vitro metabolism, CMC studies, and the data of which are used for IND submission. • In vitro validation: At this stage, the efficacy and safety of a drug candidate are assessed at cellular levels. • In vivo validation: At this stage, the efficacy, safety and pharmacokinetic of a drug candidate are assessed in animal models. • IND Preparation and Submission: Preparation of a package of documents for different sections such as CMC, clinical, nonclinical, etc. and getting them reviewed, approved and final checked and followed by submission to regulatory agencies. Human clinical trials are typically conducted in three sequential phases that may overlap or be combined: • Phase 1.Phase 1 includes the initial introduction of an investigational new drug into humans. These studies are closely monitored and may be conducted in patients but are usually conducted in healthy volunteer subjects. These studies are designed to determine the metabolic and pharmacologic actions of the drug in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. During Phase1, sufficient information about the drug’s pharmacokinetics and pharmacological effects should be obtained to permit the design of well -controlled, scientifically valid, Phase 2 studies. Phase 1 studies also evaluate drug metabolism, structure -activityrelationships, and the mechanism of action in humans. These studies also determine which investigational drugs are used as research tools to explore biological phenomena or disease processes. The total number of subjects