The ongoing DESTINY PWS Phase 3 trial is halfway to its target enrollment of patients with Prader-Willi syndrome (PWS) to evaluate the impact of once-daily treatment with diazoxide choline controlled-release (DCCR) tablets.
Importantly, more than 90% of the patients who have completed the study so far have chosen to continue treatment with DCCR tablets in the 12-month, open-label C602 safety extension study (NCT03714373).
The DESTINY PWS trial (NCT03440814) is still recruiting patients with PWS, age 4 and older, at about 20 sites across the United States and Europe. Find more information on locations and contacts here.
“Our enrollment has been impacted by the number of PWS trials currently ongoing in the U.S. and Europe, but with current strong recruitment trends, we expect to conclude enrollment around the end of the year,” Anish Bhatnagar, MD, CEO of Soleno Therapeutics, said in a press release.
Given the delay in the enrollment phase compared with its initial plans, the company expects to announce results from DESTINY PWS in the first half of 2020, rather than the prior expected date of late 2019.
Soleno is also preparing another open-label study for patients who complete the C602 study.
“We are encouraged by the continued interest of families and investigators in keeping patients on DCCR,” Bhatnagar said.
Soleno is developing DCCR to prevent the production and release of the neuropeptide Y (NPY) and agouti-related protein (AgRP), which are thought to contribute to the underlying mechanisms that trigger excessive appetite in PWS patients.
DCCR has received orphan drug designation for the treatment of PWS in the United States and Europe, and fast track status in the U.S. These designations are expected to support its clinical development, and expedite its regulatory review and approval for this indication.
Results from a Phase 1/2 trial (NCT02034071) have shown that treatment with DCCR can effectively reduce total body fat, in particular belly fat. Patients treated with daily 4.2 mg/kg DCCR (the highest tested dose) experienced a 6.1% reduction in their total body fat mass, as well as loss of fat in their trunk, legs, and arms, over a period of 10 weeks.
In the DESTINY PWS trial, researchers will compare the impact of once-daily administration of DCCR (75–450 mg) versus a placebo on approximately 105 patients diagnosed with PWS, over a treatment period of 13 weeks.
Researchers will evaluate changes in body fat, questionnaire-based hyperphagia (overeating) scores, and clinical progress estimated by clinicians and caregivers.
Soleno also announced that it has established a collaboration agreement with Casimir to help design outcome measures that could capture the real-world impact of DCCR treatment on PWS patients’ quality of life.
“Based on our extensive conversations with caregivers, physicians, and family members of PWS patients, we believe we can bring significant value to the [DCCR] program by identifying aspects of DCCR’s efficacy that may otherwise not be captured,” said Mindy Leffler, president of Casimir. “PWS patients have significant unmet needs and we look forward to collaborating with Soleno to better understand the potential efficacy of DCCR.”