This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to \< 7 years with NF1 related symptomatic, inoperable PN.
Phase 1 trials primarily test safety and dosing in a small number of participants (typically 20–100). Results do not yet speak to efficacy. FDA approval is typically many years away from this stage.
Phase 2 trials begin testing efficacy in a larger patient group (100–300). This is often where biotech binary events occur — positive Phase 2 data can significantly advance a company's pipeline narrative, while failures can be terminal for a program.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
AstraZeneca (AZN) is the sponsoring company for this trial. BiotechSign currently grades this company F (16/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.