Radius Health announced today their plans for a Pivotal Study for RAD011 for the treatment of hyperphagia in Prader-Willi syndrome. The Phase 2/3 study, SCOUT (Synthetic Cannabidiol Oral Solution), will evaluate safety and tolerability in PWS and is expected to begin by end of this year or early first quarter of 2022. With this current initiation timeline, it is anticipated that top-line results would be available in the second half of 2024.
What Is RAD011?
Investigational drug RADS011 is a pharmaceutical-grade synthetic cannabidiol oral solution, manufactured utilizing traditional pharmaceutical manufacturing processes. The product has purity specifications that meet standardized regulatory and quality control requirements and, compared to the process of developing a plant-derived product, the synthetic manufacturing process usually enables increased consistency and greater precision in the product supply. RAD011 has been assessed in over 150 patients across multiple indications and has potential utilization in multiple endocrine and metabolic orphan diseases. RAD011 has previously been granted Orphan Drug and Fast Track Designation by the FDA.
About the Phase 2/3 Study
A single Phase2/3 study design is planned to serve as the basis for market approval. Study highlights include:
- The pivotal Phase 2/3 study will be a double-blind, placebo-controlled, seamless pivotal Phase 2/3 study in individuals with genetically-confirmed PWS, ages 8 to 65
- The study will evaluate safety and tolerability across multiple dose groups in the Phase 2 portion, narrow the dose selection for the Phase 3 portion, and anticipated to enable one study to evaluate efficacy, safety and tolerability
- A screening and placebo lead-in period will precede the 26-week maintenance period 1
- Approximately 200 PWS individuals will be needed across 30+ global sites
- Primary endpoint: change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) from baseline
- Eligible individuals will have the option to enroll in a long-term extension safety study (SCOUT-016)