The Data Safety Monitoring Board (DSMB) has recommended that the trial known as DESTINY PWS for the diazoxide choline controlled-release tablet continue without any changes.
Shares in Soleno surged 223% on the Nasdaq index to $4.36.
The hallmark symptom of PWS is hyperphagia, a chronic feeling of insatiable hunger. Patients can also suffer from behavioral problems, cognitive disabilities, low muscle tone, excess body fat and incomplete sexual development.
“We are delighted with the DSMB’s positive recommendation to continue the Phase 3 trial as planned as it further supports DCCR’s safety profile,” Dr Anish Bhatnagar, CEO at Soleno, said in a statement. "We are continuing to enrol patients with 14 sites activated in DESTINY PWS."
He added that patients continue to roll over into C602, the nine-month, open-label safety extension study for patients completing treatment in the DESTINY PWS study.
The DESTINY PWS trial is a randomized, double-blind, placebo-controlled study of once-daily oral administration of DCCR in about 100 patients with a confirmed diagnosis of PWS.
Diazoxide choline has received orphan drug designation for the treatment of PWS in the US and EU.
Contact Giles at [email protected]
Follow him on Twitter @Gile74