OnCo
ideasIdea

A phase 0 fund to test academic compounds in humans with microdoses and imaging

Before investing in a full trial, give a few patients a tiny dose of a new compound and use scans and blood tests to see whether it reaches the tumour and hits its target. Fund these small studies as a matter of routine.

Phase 0 (exploratory IND) studies use microdoses or short courses to measure pharmacokinetics, target engagement (via PET tracers, tumour biopsies or pharmacodynamic markers) and biodistribution before committing to IND-enabling toxicology at full scale. They are cheap, fast and answer the question that kills most academic compounds late: does it get where it needs to go and do what it should in people? A dedicated fund pays for radiolabelling, tracer synthesis, phase 0 unit costs and biopsy-based pharmacodynamics for academic assets, with a rule that assets failing target engagement are dropped and published.

Hypothesis
Routing academic candidates through funded phase 0 studies kills at least 30% before IND-enabling investment and raises the phase 2 success rate of those that proceed compared with the historical academic pipeline, at a cost per candidate under $1 million.
Rationale
Regulatory frameworks for exploratory INDs exist in the US and EU; the NCI ran a phase 0 programme demonstrating feasibility with a PARP inhibitor. Immuno-PET and radiolabelled small molecules now allow direct measurement of tumour delivery for many modalities, including antibodies and ADCs.
What would test it
Fund phase 0 studies for ten academic assets and compare downstream attrition and cost with a matched historical cohort that went straight to IND-enabling work.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
2
Bottlenecks it attacks

Connected

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