Soleno said Thursday after the close that the Data Safety Monitoring Board, or DSMB, which evaluated its diazoxide choline controlled-release, or DCCR, tablet for the treatment of Prader-Willi Syndrome, or PWS, has given the go-ahead for a Phase 3 trial dubbed DESTINY PWS without any changes.
PWS is a complex genetic condition affecting many parts of the body in infancy, and it manifests as weak muscle tone, feeding difficulties, poor growth and delayed development, according to the National Institutes of Health.
The DESTINY PWS trial is a randomized, double-blind, placebo-controlled study of once-daily oral administration of DCCR versus a placebo in about 100 patients with a confirmed PWS diagnosis.
The primary endpoint has been fixed as the change from the baseline hyperphagia score at Week 13.
Why It’s Important
The DSMB is a body of independent experts that monitors the safety of a treatment candidate. Following a review, the DSMB recommends one of the following three courses of action:
- Continuation of the study without modification.
- Modification of the study.
- Terminate the study due to safety concerns.
Now that the DSMB overhang is behind Soleno, the company can move forward unhindered in the development process
The pipeline asset has received Fast Track Designation for the indication in the U.S. and has also received Orphan Drug designation in both the U.S. and Europe.
Soleno said it is continuing to enroll patients, with 14 sites activated in DESTINY PWS.
Patients continue to roll over into C602, a nine-month, open-label extension study for patients who completed blinded treatment in the DESTINY PWS study.
Soleno shares were trading up 216.29 percent to $4.27 at the time of publication Friday.
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