Soleno Therapeutics, Inc. (NASDAQ:SLNO), a clinical-stage
biopharmaceutical company developing novel therapeutics for the
treatment of rare diseases, today announced that the European
Medicines Agency’s (EMA) Committee for Orphan Medicinal Products
(COMP) has issued a positive opinion recommending Diazoxide Choline
Controlled-Release Tablet (DCCR) for designation as an orphan
medicinal product for the treatment of Prader-Willi Syndrome (PWS).
PWS is a rare and complex genetic neurobehavioral/metabolic
disorder affecting appetite, growth, metabolism, cognitive
function, and behavior.
“The COMP’s positive opinion recommending DCCR’s
designation as an orphan drug provides further validation of its
potential to treat PWS patients,” said Anish Bhatnagar, M.D., Chief
Executive Officer of Soleno. “PWS patients have significant unmet
needs for which there are currently no safe and effective treatment
options available. We appreciate EMA’s support for the development
of DCCR, and look forward to commencing our Phase III program.”
The COMP issues an opinion on the granting of
orphan drug designation, after which the opinion is submitted to
the European Commission (EC) for adoption. Orphan drug designation
by the EC provides regulatory and financial incentives for
companies to develop and market therapies that treat a
life-threatening or chronically debilitating condition affecting no
more than five in 10,000 persons in the European Union (EU), and
where no satisfactory treatment is available. In addition to a
10-year period of marketing exclusivity in the EU after product
approval, orphan drug designation provides incentives for companies
seeking protocol assistance from the EMA during the product
development phase, and direct access to the centralized
authorization procedure.
Soleno is currently finalizing the design of a
randomized, double-blind, placebo-controlled Phase III study for
DCCR that will treat approximately 100 PWS patients. This study is
anticipated to take approximately 9-12 months to complete and is
expected to be initiated at the end of 2017. DCCR previously
received orphan designation for the treatment of PWS in the
U.S.
About PWS
PWS is a rare and complex genetic
neurobehavioral/metabolic disorder affecting appetite, growth,
metabolism, cognitive function and behavior. The committee on
genetics of the American Academy of Pediatrics states PWS affects
both genders equally and occurs in people from all geographic
regions: its estimated incidence is one in 15,000 to 25,000 live
births. This disorder is typically characterized by hyperphagia, a
chronic feeling of insatiable hunger, behavioral problems,
cognitive disabilities, low muscle tone, short stature (when not
treated with growth hormone), the accumulation of excess body fat,
developmental delays, and incomplete sexual development.
Hyperphagia, in the absence of effective limitations to access to
food, can lead to morbid obesity. In a global survey conducted by
the Foundation for Prader-Willi Research, 96.5% of respondents
(parent and caregivers) rated hyperphagia, which is the unrelenting
hunger that severely diminishes the quality of life for patients
and their families, as the most important or a very important
symptom to be relieved by a new medicine. There are currently no
approved therapies to treat the hyperphagia/appetite, metabolic,
cognitive function, or behavioral aspects of the disorder.
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. Soleno 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 five
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.
About Soleno Therapeutics, Inc.
Soleno Therapeutics, Inc. (Soleno) is focused on
the development and commercialization of novel therapeutics for the
treatment of rare diseases. The company is currently advancing its
lead candidate, DCCR, a once-daily oral tablet for the treatment of
PWS, into a Phase III clinical development program at the end of
2017. Soleno, through its wholly owned subsidiary, Capnia, Inc.,
continues to market Capnia’s innovative medical device, the
CoSense® End-Tidal Carbon Monoxide (ETCO) monitor, which measures
ETCO and is used by hospitals to detect hemolysis in newborns. It
is expected that CoSense will be monetized and will not be a focus
for the company in the long term.
For more information, please visit
www.soleno.life.
Forward-Looking Statements
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
Securities and Exchange Commission on August 11, 2017, including
under the caption titled "Risk Factors." Soleno expressly disclaims
any intent or obligation to update these forward-looking
statements, except as required by law.
Contact:Brian RitchieLifeSci Advisors,
LLC212-915-2578
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