Oral presentation on DCCR’s mechanism of action in treating hyperphagia
Poster presentation on DCCR’s safety profile and implications for PWS Phase III study design
“The data presented on DCCR at this year’s FPWR continue to support the design of our ongoing Phase 3 trial,” said
- Neural mechanism underlying hyperphagia in PWS may be driven by excessive secretion of two neurotransmitters, NPY and AgRP, from NAG neurons of the hypothalamus, regulated by leptin and insulin through agonism of the KATP channel
- DCCR directly agonizes KATP channels in NAG Neurons, lowering NPY and AgRP secretion
- Directly agonizing KATP channels via diazoxide has demonstrated reductions of hyperphagia in multiple animal models of NPY-driven hyperphagia
- Consistent with this mode of action, reductions in hyperphagia were documented in a successfully completed Phase II clinical study of DCCR in PWS
- DCCR’s once-daily dosing demonstrates potential for increased safety over diazoxide
- Adverse events with DCCR are consistent with the known profile of diazoxide, with frequency and/or severity reduced with dose titration
- Dosing period of ≥ 10 days at each titration step effectively improved the safety profile
- Based on this dosing paradigm, which was also used in the PWS Phase II study, the ongoing Phase III trial includes titration steps lasting approximately two weeks each.
Electronic versions of both posters can be found on the Events & Presentations page of Soleno Therapeutics’ Investor Relations website: http://investors.soleno.life/events-and-presentations/event-calendar.
About Diazoxide Choline Controlled-Release 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 positive data from five completed Phase I clinical studies in various metabolic indications or 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 clinical development program of DCCR in PWS in 2019.
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
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Source: Soleno Therapeutics