Updated Data Highlighted in Poster Presentation
at 10th International Meeting of Pediatric Endocrinology
Soleno Therapeutics, Inc. (NASDAQ:SLNO), a clinical-stage
biopharmaceutical company developing novel therapeutics for the
treatment of rare diseases, announced today that updated safety and
efficacy data from the pilot clinical trial of Diazoxide Choline
Controlled Release Tablet (DCCR) for the treatment of Prader-Willi
syndrome (PWS) were the subject of a poster presentation at the
10th International Meeting of Pediatric Endocrinology, being held
September 14-17 in Washington, D.C. The data were presented
by Virginia Kimonis, M.D., Division of Genetics and Metabolism,
School of Medicine, University of California, Irvine, and Principal
Investigator of the trial.
The purpose of the study was to assess the safety and efficacy
of multiple dose levels of DCCR in subjects ages 10-22 years with
genetically-confirmed PWS. The study consisted of a 10-week
open-label treatment phase, during which subjects were escalated
from 1.5 mg/kg to a maximum of 5.1 mg/kg of DCCR and then treated
stably for the remainder of the phase. Subjects with an improvement
in hyperphagia and/or an increase in resting energy expenditure
during the open-label phase were classified as responders and were
eligible to enter a 4-week double-blind placebo-controlled
withdrawal treatment phase, in which they were randomized to DCCR
or placebo. Thirteen subjects were enrolled; 11 subjects
completed the open-label phase, were classified as responders and
randomized into the double-blind phase.
Key efficacy results included a significant improvement from
baseline in hyperphagia (mean baseline score = 15 out of possible
scores of 0-34) starting within two weeks of DCCR treatment.
As previously reported, a highly statistically significant
reduction (-4.32, p=0.0055) was seen in hyperphagia from baseline
to the end of the open-label treatment phase. In addition,
greater improvement in hyperphagia from baseline was observed in
those subjects with moderate to severe hyperphagia who received
DCCR doses ≥ 4.2 mg/kg (the planned population and target dose for
the Phase III study) with a reduction of -7.83 (n =3). A
significant improvement in the number of subjects reporting one or
more aggressive and destructive behaviors was seen (p=0.006).
During the open-label treatment phase, a mean decrease in body
fat mass (p=0.02) and increases in lean body mass (p=0.003) and
lean body mass / fat mass ratio (p=0.004) were seen. These changes
were associated with a statistically significant reduction in waist
circumference (-3.45 cm, p=0.006), consistent with the loss of
visceral fat. Leptin levels were significantly decreased (p=0.007)
from baseline, while ghrelin levels remained unchanged
(p=0.93).
The safety profile of DCCR in this study was consistent with the
known profile of diazoxide. The most commonly reported
treatment-emergent adverse events observed are also common in PWS
patients, including glycemic impacts, peripheral edema, upper
respiratory tract infections, constipation, and somnolence. Insulin
sensitivity as measured by reduction HOMA-IR was improved.
“DCCR treatment of adolescent and adult PWS patients in this
study positively impacted a number of the highest priority unmet
needs in the disease, including hyperphagia and body fat,” said Dr.
Kimonis. “DCCR has the potential to become a safe and
effective therapeutic option for PWS patients and their
families. I look forward to the further clinical evaluation
of DCCR in PWS in a Phase III clinical trial.”
“The updated analyses of the data from the pilot study provide
further evidence of the efficacy of DCCR in patients with PWS,”
said Anish Bhatnagar, Chief Executive Officer of Soleno
Therapeutics. “We are finalizing the design of a randomized,
double-blind placebo-controlled study that will treat approximately
100 patients. The dose response data obtained from this
study will help inform dosing in and design of the upcoming Phase
III trial.”
DCCR has Orphan Designation for the treatment of PWS in the
U.S.
About PWSPWS 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 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
TabletDiazoxide 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 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.
About Soleno Therapeutics, Inc.Soleno
Therapeutics, Inc. 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 this product 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 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 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 Capnia’s Form 10-Q filed with the
Securities and Exchange Commission on August 11, 2017, including
under the caption titled "Risk Factors." Capnia 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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