Benzinga | Jul 22, 2021 09:02AM EDT

Radius Health Announces Plans For Global Prader-Willi Syndrome Pivotal Study

Radius Health, Inc. ("Radius" or the "Company") (NASDAQ:RDUS) today announced that it has recently received the written meeting minutes from a June Type C meeting held with the U.S. Food and Drug Administration ("FDA") regarding RAD011, a synthetic cannabidiol oral solution.

RAD011 is initially to be utilized for the treatment of Prader-Willi Syndrome ("PWS"). RAD011 has previously been granted Orphan Drug and Fast Track Designation by the FDA. Based on the feedback received, Radius plans on initiating a pivotal Phase 2/3 global study for patients with PWS.

The main highlights from the FDA meeting minutes are set out below:

* Design characteristics and endpoints for a single seamless, pivotal Phase 2/3 study for PWS

* Results dependent, a single adequate and well-controlled study could serve as the basis for marketing approval

* Acceptability of 505(b)(2) regulatory pathway subject to completion and review of PK bridging and dedicated food effect studies

* Acceptability of nonclinical package to support the pivotal Phase 2/3 study

Paige Rivard, CEO of Prader-Willi Syndrome Association USA said, "We are encouraged by Radius' commitment to advance RAD011 for the treatment of debilitating symptoms associated with PWS, particularly hyperphagia. We look forward to supporting their team throughout their study by raising awareness of their planned study with key opinion leaders, caregivers and individuals within the PWS community, and providing a means to gather perspective of individuals with PWS and critical caregivers."

Phase 2/3 Study

Radius plans to initiate the pivotal Phase 2/3 study, to be branded as "SCOUT" (Synthetic Cannabidiol Oral Solution), by end of this year or early first quarter of 2022. With this current initiation timeline, it is anticipated top line results would be available in the second half of 2024.

The proposed study parameters, informed by several global advisory board meetings completed with leading KOLs, PWS advocacy organizations, and feedback from the FDA, are highlighted below:

* The pivotal Phase 2/3 study (SCOUT-015) will be a double-blind, placebo-controlled, seamless pivotal Phase 2/3 study in individuals with genetically-confirmed PWS, ages 8 to 65

* The seamless design 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

* Approximately 200 PWS individuals at 30+ global sites are planned for inclusion in SCOUT-015

* Primary endpoint: change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) from baseline

* Eligible individuals have the option to enroll in a long-term extension safety study (SCOUT-016)

Liz Messersmith, Ph.D. in Neuroscience and Senior Vice President at Radius stated, "We have established an exceptionally strong and highly experienced team of dedicated clinical, medical, biometrics, advocacy, regulatory and CMC talent to execute the pivotal study for PWS."

Dr. Messersmith added further that, "We intend to use PWS as the anchor indication for RAD011. Additional disease opportunities and clinical trial initiatives will be shared in due course."

Strategic CRO Partner Selected & DEA Scheduling Guidance Received

The Company is working closely with CTI Clinical Trial & Consulting, a world-leading CRO in rare and orphan disease research, to initiate and execute SCOUT-015 and SCOUT-016.

Important to the initiation and execution of the SCOUT program, Radius will move forward with RAD011 as not scheduled under the Controlled Substance Act ("CSA") based on guidance from the Drug Enforcement Administration ("DEA"). The guidance states if a product does not contain any quantity of synthetic THC (or any other controlled substance), it is not controlled under the CSA. RAD011 is not scheduled as it does have traceable amounts of THC.