The EMA indicated that a single pivotal trial would support a Marketing Authorisation Application. They also indicated their general acceptance of several key aspects of the proposed development plan, on which general agreement had been reached previously with the
The EMA expressed their support for change in hyperphagia compared to placebo as the primary endpoint for the study. In addition, the dosing paradigm proposed by Soleno for the study was accepted, as it was during Soleno’s scientific advice meeting with the
“We are encouraged by the constructive guidance we have received from both the
About Scientific Advice
The EMA provides scientific advice to facilitate the development and availability of new safe and effective medicines for patients. This prospective advice focuses on development strategies, including the design of studies and other tests, to ensure that no major objections will be raised during the Marketing Authorisation Application (MAA) evaluation. The scientific advice provided by the EMA is not legally-binding; however, following the EMA’s advice increases the probability of a positive outcome.
PWS is a rare and complex genetic neurobehavioral/metabolic disorder affecting appetite, growth, metabolism, cognitive function and behavior. The committee on genetics of the
About Diazoxide Choline Controlled-Release Tablet
Diazoxide choline controlled-release tablet is a novel, proprietary controlled-release, crystalline salt formulation of diazoxide, which is administered once-daily. The parent molecule, diazoxide, as an oral suspension, has been used for decades in thousands of patients in a few rare diseases in neonates, children and/or adults, but not in PWS. Essentialis conceived of and is pursuing an extensive patent portfolio relating to various aspects of the therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR development program is supported by positive data from two completed Phase II clinical studies and six completed Phase I clinical studies in various metabolic indications, as well as a pilot study in PWS patients. In the PWS pilot study, DCCR showed promise in addressing the hallmark symptoms of PWS, most notably hyperphagia. DCCR has received Orphan Drug Designation from the US FDA for the treatment of PWS.
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 initiate the Phase III clinical development program of DCCR in PWS by the end of 2017.
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 presentation will prove to be accurate. Additional factors that could materially affect actual results can be found in Soleno’s Form 10-Q filed with the
LifeSci Advisors, LLC
Source: Soleno Therapeutics