- FDA Type C meeting minutes provide clarity on the Phase 2/3 study design for RAD011
- 505(b)(2) regulatory strategy: acceptable approach upon completion of PK & food effect studies
- Based on results, a single well controlled study could serve as the basis for marketing approval
- RAD011 had previously been granted Orphan Drug and Fast Track Designation by the FDA
- Study initiation targeted by end of year or early first quarter of 2022
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
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)
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
About Prader-Willi Syndrome
PWS, an orphan disease, is a complex genetic disorder with clinical manifestations on the endocrine and neurological systems. Clinical signs of PWS develop throughout childhood, with hyperphagia and anxiety ranked as the key clinical features seeking medical attention by caregivers of individuals with PWS. Hyperphagia is a relentless, insatiable, pathological drive to eat that requires caregivers to strictly manage access to food through the locking of cabinets and refrigerators. PWS is recognized as the leading genetic cause of life-threatening obesity in children. As life-threatening hyperphagia persists into adulthood, metabolic syndrome expressed through obesity and diabetes can develop and contribute to morbidity and mortality. In addition to food-related behaviors, the behavioral symptoms commonly observed in PWS include high irritability, habitual skin picking, oppositional defiance and cognitive rigidity. There are currently no approved therapies to treat this disorder’s hyperphagia, irritability, or metabolic aspects. In the
Investigational drug RAD011 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. Radius is initially targeting Prader-Willi syndrome (PWS) and anticipates initiating a seamless pivotal Phase 2/3 study for patients with PWS in the second half of 2021.
Radius is a commercial biopharmaceutical company committed to serving patients with unmet medical needs in endocrinology and other therapeutic areas. Radius’ lead product, TYMLOS® (abaloparatide) injection, was approved by the
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations with respect to our SCOUT-15 and SCOUT-16 clinical trials and timing of receipt of topline results therefrom.
These forward-looking statements are based on management's current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the adverse impact the ongoing COVID-19 pandemic is having and is expected to continue to have on our business, financial condition and results of operations, including our commercial operations and sales, clinical trials, preclinical studies, and employees; quarterly fluctuation in our financial results; our dependence on the success of TYMLOS, and our inability to ensure that TYMLOS will obtain regulatory approval outside the
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Source: Radius Health Inc.