Soleno Therapeutics Announces Presentation of Body Composition Results from DESTINY PWS, a Phase III Trial of DCCR in Prader-...
November 03 2020 - 4:01PM
Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a
clinical-stage biopharmaceutical company developing novel
therapeutics for the treatment of rare diseases, today announced
presentation of body composition data from the Company’s Phase III
trial, DESTINY PWS (C601), evaluating once-daily Diazoxide Choline
Controlled Release (DCCR) tablets for patients with Prader-Willi
Syndrome (PWS). The results were presented today by Parisa Salehi,
M.D., Clinical Director of the Prader-Willi Syndrome Clinic at
Seattle Children’s Hospital, in a late-breaking oral presentation
at The Obesity Society’s ObesityWeek® 2020 meeting. Dr. Salehi
presented the data on behalf of the DESTINY PWS Investigators.
DESTINY PWS is a randomized, double-blind, placebo-controlled
Phase III study of once daily oral administration of DCCR in 127
PWS patients conducted at 29 sites in the U.S. and U.K. The
objective of the study was to assess the efficacy and safety of
DCCR in subjects with genetically-confirmed PWS aged four years and
older and weighing between 20 and 134 kg. Patients who completed
the double-blind study enrolled in study C602, an ongoing
open-label, extension study. Updated top-line results were
previously announced in September 2020 that demonstrated DCCR’s
beneficial impact on hyperphagia, the predominant symptom of PWS,
other behaviors and body composition abnormalities typical of
PWS.
Key results for effects on body composition and adipokines
following DCCR treatment were presented at ObesityWeek 2020 as
follows:
Fat mass and body mass:
- Significant reductions in fat mass (p=0.0027) were observed
after 13 weeks of double-blind treatment- Greater mean
reductions in fat mass were observed in those participants weighing
more than 100 kg at baseline (placebo-adjusted difference -4.82
kg)
- Linear exposure-response relationship for fat mass was
significant with greater fat loss occurring at higher circulating
drug concentrations (p<0.0001)
- Trend towards increased lean body mass for DCCR compared to
placebo (p=0.058)
- Significant increase in lean body mass/fat mass ratio
(p=0.001)
Leptin:
- Significant reduction in leptin while it increased in
placebo-treated subjects (p<0.0001)
- Linear exposure-response relationship for leptin was
significant with greater reductions in leptin occurring at higher
circulating drug concentrations (p<0.0001)
- Reductions in leptin were greater than would be predicted
solely by the loss of body fat, suggesting an improvement in leptin
sensitivity
Adiponectin:
- Adiponectin, a cardioprotective hormone, increased in
DCCR-treated subjects, but decreased in placebo-treated
participants (p<0.0001)
“We are pleased to present further results from the DESTINY PWS
study that demonstrate DCCR’s effect on body composition. In the
DCCR group compared to placebo, we have observed a significant
reduction in fat mass and leptin. Improvements in leptin
sensitivity, as suggested by the results, may have an important
impact on regulating hyperphagia,” said Dr. Salehi. “The data
presented to date show that DCCR has the potential to manage both
behavioral and co-morbid metabolic components of PWS.”
“These data represent additional means by which DCCR may address
the significant unmet medical needs and the life-threatening
comorbidities associated with PWS,” said Anish Bhatnagar, M.D.,
Chief Executive Officer of Soleno Therapeutics. “We remain focused
on advancing DCCR as a potential treatment for PWS and look forward
to our regulatory interactions to establish the path forward for
making DCCR available as a treatment for PWS patients.”
About PWSThe Prader-Willi Syndrome Association
USA estimates that one in 15,000 live births in the U.S. 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 function, or behavioral aspects of
the disorder. Diazoxide choline has received Orphan Drug
Designation for the treatment of PWS in the U.S. and EU, and Fast
Track Designation in the U.S.
About Diazoxide Choline Controlled-Release (DCCR)
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 data from five completed Phase
I clinical studies in healthy volunteers and three completed Phase
II clinical studies, one of which was in PWS patients. In the PWS
Phase III 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 other metabolic
parameters.
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, Diazoxide Choline Controlled-Release (DCCR) tablets, a
once-daily oral tablet for the treatment of Prader-Willi Syndrome
(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 within the meaning of Section
21E of the Securities Exchange Act of 1934, as amended. All
statements other than statements of historical facts contained in
this press release are forward-looking statements, including
statements regarding timing of any regulatory process or ultimate
approvals and determining a path forward for DCCR for the treatment
of PWS. In some cases, you can identify forward-looking statements
by terms such as "may," "will," "should," "expect," "plan,"
"anticipate," "could," "intend," "target," "project,"
"contemplates," "believes," "estimates," "predicts," "potential" or
"continue" or the negative of these terms or other similar
expressions. These forward-looking statements speak only as of the
date of this press release and are subject to a number of risks,
uncertainties, and assumptions, including the risks and
uncertainties associated with market conditions, as well as risks
and uncertainties inherent in Soleno’s business, including those
described in the company's prior press releases and in the periodic
reports it files with the SEC. The events and circumstances
reflected in the company's forward-looking statements may not be
achieved or occur and actual results could differ materially from
those projected in the forward-looking statements. Except as
required by applicable law, the company does not plan to publicly
update or revise any forward-looking statements contained herein,
whether as a result of any new information, future events, changed
circumstances or otherwise.
Corporate Contact:Brian RitchieLifeSci
Advisors, LLC212-915-2578
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