Radius Planning Pivotal Trial of Cannabidiol RAD011

Radius Health has acquired the development and commercialization rights to a synthetic oral cannabidiol therapy known as RAD011, with plans to start a pivotal Phase 2/3 clinical trial in Prader-Willi syndrome (PWS) this year.

Having finalized its acquisition deal with Benuvia Therapeutics, the company expects to initiate the trial in the second half of 2021 following discussions with the U.S. Food and Drug Administration (FDA).

“RAD011 has the potential for broad clinical applicability. We intend to use PWS as the anchor indication and will further investigate additional orphan disease opportunities in due course,” Kelly Martin, CEO of Radius, said in a press release.

Under the agreement, Radius acquired rights to RAD011 for $12.5 million, with an additional $15 million payment contingent on successfully meeting milestones in PWS development. The therapy is part of an orphan drug program in which PWS is the first indication. Orphan drugs are therapies developed to treat rare diseases that, without special status and incentives from the government, would be unprofitable to produce. 

PWS is characterized by poor muscle tone and growth, and by hyperphagia (excessive appetite), weight gain, behavioral problems, and intellectual disability in childhood.

As a cannabidiol — an active chemical of the cannabis plant, devoid of mind-altering effects — RAD011 functions by targeting multiple signaling pathways and receptors involved in regulating hyperphagia and anxiety, the symptoms often ranked as the two most significant by caregivers of PWS patients, according to Radius. Benefits in behavioral problems also may be achieved, the company stated.

“Currently, there is no treatment for Prader-Willi syndrome. For many patients with the disorder, ameliorating some of the most difficult symptoms, such as insatiable appetite and anxiety, would be life-changing for them and their families,” said Lynne M. Bird, MD, medical director of the PWS Clinic at Rady Children’s Hospital.

Early data from an abbreviated Phase 2 clinical trial of RAD011 indicated that the treatment may help reduce hyperphagia and weight. 

The design of the upcoming trial of RAD011 will be informed by findings from previous clinical studies conducted in the PWS patient population. Top-line data could be released within about two years after the study begins, according to Radius.

The FDA granted RAD011 fast track designation in 2017 and orphan drug designation in 2020 for the treatment of hyperphagia and weight loss in PWS. Both designations convey financial and regulatory incentives for clinical development.