Amendment Lowers Minimum Age Inclusion Criteria
to Four Years, from Eight Years, to Better Reflect Demographics of
PWS Population
Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a
clinical-stage biopharmaceutical company developing novel
therapeutics for the treatment of rare diseases, today announced a
protocol amendment to its ongoing Phase III trial, DESTINY PWS,
studying Diazoxide Choline Controlled-Release (DCCR) tablets in
Prader-Willi syndrome (PWS). The protocol amendment reduces the age
of inclusion to children as young as four years of age, from eight
years of age previously. The amendment is the result of U.S. Food
and Drug Administration (FDA) feedback regarding Soleno’s Initial
Pediatric Investigational Plan. Hyperphagia, the central
characteristic of PWS, is present in nearly all PWS patients by age
8, and can occur in children as young as four years of age. The
protocol amendment has been approved by an independent
Institutional Review Board and is in the process of being
implemented at clinical trial sites.
“The hallmark symptom of PWS is hyperphagia, an
unrelenting hunger that causes significant challenges to both
patients and their caregivers, which manifests in early childhood,”
said Jennifer L. Miller, M.D., Associate Professor in the Division
of Pediatric Endocrinology at the University of Florida and a
Principal Investigator in the Soleno study. “Managing hyperphagia
in PWS patients is a critical unmet need for which there are no
currently approved treatments. Because the parent molecule in
DCCR has been approved for use in neonates through adults for other
conditions, and at much higher daily doses than are being used in
this study, its safety profile is well understood. We are pleased
to continue evaluating this promising product candidate in PWS
patients with hyperphagia in all ages in the ongoing Phase III
trial.”
“This important protocol change to the ongoing
Phase III trial of DCCR in PWS allows enrollment of hyperphagic PWS
patients as young as four years of age, permitting inclusion of
those children with an earlier age of hyperphagia onset,” said Dr.
Anish Bhatnagar, Chief Executive Officer of Soleno. “We continue to
work with our investigators, as well as the PWS community, to
complete enrollment and advance this trial through the clinic as
expeditiously as possible.”
The DESTINY PWS Phase III trial 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. The primary endpoint is change
from baseline hyperphagia score at Week 13. The trial is being
conducted at approximately 15 sites in several states in the U.S.
Patients who complete the double-blind study are eligible to enroll
in an open-label, 9-month safety extension study. The double-blind
Phase III trial, which was initiated in May 2018, is expected to be
completed in the first half of 2019.
In July 2018, the FDA designated the investigation of DCCR for
the treatment of PWS to be a Fast Track development program. Prior
to this, diazoxide choline received orphan designation for the
treatment of PWS in the U.S. and in the E.U.
For further information about the trial (NCT03440814), please
visit: www.clinicaltrials.gov.
About PWSThe Prader-Willi
Syndrome Association USA estimates that one in 12,000 to 15,000
people in the US have PWS. The hallmark symptom of this disorder is
hyperphagia, a chronic feeling of insatiable hunger that severely
diminishes the quality of life for PWS patients and their families.
Additional characteristics of PWS include 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 can lead to significant morbidities (e.g., stomach
rupture, obesity, diabetes, cardiovascular disease) and mortality
(e.g., choking, accidental death due to food seeking behavior). In
a global survey conducted by the Foundation for Prader-Willi
Research, 96.5% of respondents (parent and caregivers) rated
hyperphagia 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. Diazoxide choline
has received Orphan Drug Designation for the treatment of PWS in
the U.S. and E.U.
About Diazoxide Choline
Controlled-Release TabletDiazoxide 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.
About Soleno Therapeutics,
Inc.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.
Forward-Looking StatementsThis
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 Securities and Exchange Commission, 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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