Based on the interest of the clinical investigators and families, Soleno will continue to make DCCR available to patients enrolled in the current program. The duration of C602 has been increased from 9 to 12 months, and Soleno is in the process of initiating 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,” said Dr. Anish Bhatnagar, Chief Executive Officer of Soleno. “Our enrollment has been impacted by the number of PWS trials currently ongoing in the US and
Collaboration with Casimir
Soleno has entered into a collaboration with
“The 21st Century Cures Act defines the importance of individual patient experience to the FDA’s regulatory decision making process. We are excited to be working with a group as innovative as Casimir to collect these meaningful, individualized outcomes,” said Dr. Bhatnagar. “Their work in designing outcome measures for other rare disease therapies, notably EXONDYS 51, the first
“We are delighted to work with Soleno on the DCCR program for PWS,” said Mindy Leffler, President of Casimir. “Based on our extensive conversations with caregivers, physicians and family members of PWS patients, we believe we can bring significant value to the program by identifying aspects of DCCR’s efficacy that may otherwise not be captured. PWS patients have significant unmet needs and we look forward to collaborating with Soleno to better understand the potential efficacy of DCCR.”
DESTINY PWS is a randomized, double-blind, placebo-controlled study of once-daily oral administration of DCCR versus placebo in approximately 100 patients with a confirmed diagnosis of PWS. Patients who complete DESTINY PWS have the option to enroll into C602.
In July 2018, the U.S. Food and Drug Administration (FDA) designated the investigation of DCCR for the treatment of PWS to be a Fast Track development program. Diazoxide choline has also received orphan designation for the treatment of PWS in the U.S. and in the E.U.
For further information about DESTINY PWS (NCT03440814), please visit: www.clinicaltrials.gov.
About Diazoxide Choline Controlled-Release (DCCR) Tablet
Diazoxide choline controlled-release tablet is a novel, proprietary extended-release, crystalline salt formulation of diazoxide, which is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection on the therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase I clinical studies in healthy volunteers and three completed Phase II clinical studies, one of which was in PWS patients. In the PWS Phase II study, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and abnormal lipid profiles.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s lead candidate, DCCR, a once-daily oral tablet for the treatment of PWS, is currently being evaluated in a Phase III clinical development program.
For more information, please visit www.soleno.life.
This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, our ability to complete the Phase III DESTINY PWS study during 2020. We may use terms such as "believes," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should," "approximately" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this presentation. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in Soleno’s annual and quarterly reports filed with the
Source: Soleno Therapeutics