Client Overview
The Challenge
The Phase 1 failure introduced uncertainty regarding the compound’s pharmacokinetic properties and clinical viability.
The client needed to rapidly recover program momentum without restarting from scratch—balancing scientific rigor with cost and timeline pressures.
The challenge lay in identifying new compounds that retained the desired pharmacological profile while improving exposure and clinical performance.
NGT Approach
Partnered with the client’s R&D and clinical development teams to conduct a comprehensive data mining and structural analysis of the original compound and its analogs.
Identified several structurally related candidates with improved in vitro profiles and favorable non-human primate PK characteristics.
Designed a streamlined development path to leverage existing safety data from the first compound, enabling faster transition to clinical evaluation.
Proposed and helped execute a Phase 0/1 microdosing study, allowing sub-therapeutic evaluation of multiple compounds in parallel—reducing both cost and risk.
Provided strategic oversight to ensure alignment with regulatory expectations while maintaining scientific integrity.
Facilitated cross-functional integration between discovery, preclinical, and clinical teams to accelerate decision-making.
Outcome / Impact
Rapid identification of new lead candidates through efficient use of existing data and focused experimentation.
Significant cost savings achieved by leveraging the prior safety package and executing a low-dose exploratory study design.
The client successfully advanced several promising molecules, one of which entered formal preclinical development as the new lead candidate within six months.
This structured approach reestablished program confidence, preserved investor momentum, and provided a validated blueprint for future compound rescue strategies.