Case Study Candidate Selection and Lead Optimization
Benefits of Case:
- Generated a preclinical research plan designed to demonstrate the pipeline potential of the Client’s core drug discovery platform
- Identified the critical path for candidate screening and rapid preclinical proof of concept
- Established pharmacological, safety and chemical criteria for lead candidate selection
Background and Challenge:
A company was in the early stages of constructing a pipeline of proprietary leads using a novel chemistry platform with the ability to transform the absorption and tissue uptake of known drugs. PharmaDirections was tasked with designing a preclinical research strategy that could demonstrate the full potential of the platform in building a pipeline of targeted lead molecules, while accelerating the identification of potential development candidates.
PharmaDirections Strategy:
- Assemble an expert team to evaluate the key benefits of the Client’s core technology platform and highlight the critical success factors for lead generation
- Define those in vitro and in vivo studies most likely to generate useful data in demonstrating the robustness of the platform in delivering compounds of potential interest
- Establish objective criteria for lead candidate selection based on industry standard benchmarks for potency, selectivity, bioavailability, safety, physical properties and ‘drugability’
- Recommend ways of collecting and presenting comprehensive data sets to optimize interest by potential partners and investors
The Objectives:
PharmaDirections assembled an expert team with both general preclinical development and therapeutic area specific experience to review the technology and the data collected by the Client to date. PharmaDirections performed a gap analysis on the existing data and the Client’s current research plan to identify critical data needs and potential program pitfalls. These activities resulted in a cost- and time effective research strategy designed to highlight the principle strengths of the core technology platform in generating leads of interest while advancing the most attractive candidates rapidly through development. By setting criteria for the ‘drugability’ of the chemical leads as well as basic requirements for efficacy, safety and bioavailability in animal models, PharmaDirections was able to focus the Client on those compounds with the greatest potential for successful IND track development.
Value for the Client:
The Client was able to increase focus on critical path studies while avoiding the collection of data considered non-strategic to company and program goals. The proposed research plan resulted in convincing preclinical proof of concept while realizing time and cost savings by anticipating investor/partner questions and anticipating issues in downstream IND enabling activities.