CRED Understanding Clinical Development 2024

08/10/2024

Role of Phase II studies

Like building………………

Build confidence

Clinical data – Building the key components of a Phase 2 study (dose, tolerability, safety, efficacy)

Lay the Foundations

Pre-Clinical Data – in vitro/in vivo data (PK/PD data)

The Organisation for Professionals in Regulatory Affairs

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Aims

What is already known when starting Phase 2 ● Preclinical efficacy data ( in vitro / in vivo data) ● Phase 1 information: – Tolerability (maximum tolerated dose) – Pharmacokinetic data (SAD/MAD data) – Pharmacodynamic data (receptor occupancy, biomarker information)

What do we want to know at the end of Phase 2 ● Unequivocal data to allow assessment of efficacy and safety to support a go/no-go decision for Ph 3 ● Clear justification of the dose for Phase 3; including treatment regimen and formulation refinement ● Design confirmatory Phase 3

Ultimately Phase 2 = Proof of Concept (POC)

The Organisation for Professionals in Regulatory Affairs

SAD = Single Ascending Dose; MAD = Multiple Ascending Dose

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