Soleno Therapeutics recently shared data from their DESTINY PWS study, an international, placebo-controlled Phase 3 study of DCCR (Diazoxide Choline Controlled-Release) drug trial. To further establish the effects of DCCR administration on hyperphagia, a randomized withdrawal period was initiated for 77 participants who had been taking DCCR for more than 2 years. During the withdrawal period, half of the patients continued DCCR while the other half received placebo. Here are three key takeaway from the study:
1. Efficacy Data is Encouraging: The DCCR drug trial showed promising efficacy data, indicating a significant reduction in hyperphagia (excessive hunger) and improved weight management among participants. This is a noteworthy step toward improving the lives of those with PWS.
2. DCCR has Favorable Safety and Tolerability: DCCR is generally well tolerated. No new safety concerns have been identified.
3. Potential FDA Approval: Based on these positive results, Soleno Therapeutics intends to seek FDA approval for DCCR as a treatment for Prader-Willi syndrome. This is a significant development as there are currently no FDA-approved medications specifically for PWS.
To provide a deeper understanding of their groundbreaking announcement, Soleno actively engaged with the Prader-Willi community earlier this month during two presentations at FPWR’s Annual PWS | SYS Research Symposium and Family Conference. These sessions were recorded and are now readily accessible for you to gain valuable insights.
Session Recording: Q&A With Soleno CEO, Anish Bhatnagar
In this session, Anish Bhatnagar, CEO of Soleno Therapeutics, takes center stage to address pressing questions from the PWS community. Learn more about next steps for DCCR in this short interview.
Session Recording: DCCR Data Presentation from the 2023 PWS Research Symposium
In this recorded session from the 2023 PWS Research Symposium, Dr Jennifer Miller presents topline results from the study.
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