Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage
biopharmaceutical company developing novel therapeutics for the
treatment of rare diseases, today announced the publication of the
comparison of results from the Company’s Phase 3 placebo-controlled
study (C601) and open-label extension study (C602) evaluating
investigational, once-daily DCCR (Diazoxide Choline)
Extended-Release tablets in patients with Prader-Willi syndrome
(PWS), to data from the PATH for PWS (PATH) natural history study,
in the Journal of Neurodevelopmental Disorders. The article,
entitled, “Behavioral Changes in Patients with Prader-Willi
Syndrome Receiving Diazoxide Choline Extended-Release Tablets
Compared to the PATH for PWS Natural History Study,” can be found
HERE.
Data from DCCR-treated participants in the C602/C602 cohort were
compared to results from a cohort of comparable participants from
PATH using the same caregiver-completed questionnaires to measure
hyperphagia (Hyperphagia Questionnaire for Clinical Trials [HQ-CT])
and PWS-related behaviors (the Prader-Willi Syndrome Profile
[PWSP]) in six domains: aggressive behaviors, anxiety,
compulsivity, depression, disordered thinking, and
rigidity-irritability.
Hyperphagia: Participants treated with DCCR
showed highly statistically significant and clinically meaningful
improvement with DCCR administration relative to participants in
the PATH study at 6 and 12 months (p<0.001 for both
comparisons).
PWS-related behaviors: As with hyperphagia,
highly statistically significant and clinically meaningful
improvements were seen in DCCR treated participants compared to
those in the PATH study in all behavioral domains of the PWSP at 6
and 12 months (p≤0.003 for all domains).
“The PATH for PWS study is an important evaluation of the
natural history of individuals with PWS,” said Dr. Theresa Strong,
Director of Research Programs for FPWR. “These highly significant
improvements with long-term DCCR treatment compared to the PATH
data suggest that DCCR has the potential to provide much needed and
substantial improvement in the lives of those living with PWS and
their families. We are excited to see DCCR advance through the
regulatory process and look forward to continuing to support Soleno
in its efforts.”
“DCCR has the potential to substantially improve the quality of
life for individuals with PWS and their families,” said PATH Study
Principal Investigators, Jennifer Miller, M.D. and Shawn
McCandless, M.D. “These data clearly demonstrate that long-term
treatment with DCCR resulted in changes in hyperphagia and other
behavioral complications of PWS that are meaningfully improved
compared to the natural history of the disease. We believe these
results support the significant potential of DCCR in PWS and are
eager to offer a much-needed treatment option to patients in need,
if approved.”
About the DCCR C601/602 Dataset
C602 an open-label extension study enrolled participants who
completed DESTINY PWS (C601), an international, multi-center,
randomized, double-blind, placebo-controlled study of DCCR in 127
PWS patients at 29 sites in the U.S. and UK.
About PATH for PWS
The PATH from PWS (PATH) study
(https://www.clinicaltrials.gov/study/NCT03718416) is a recently
completed study that was concurrently recruited with DESTINY PWS
and sponsored by the Foundation for Prader-Willi Research (FPWR).
The key objective of PATH is to advance the understanding of the
natural history in individuals with PWS, particularly long-term
behavioral changes in the syndrome.
About PWSThe Prader-Willi Syndrome Association
USA estimates that PWS occurs in one in every 15,000 live births.
The hallmark symptom of this disorder is hyperphagia, a chronic and
life-threatening feeling of intense, persistent hunger, food
pre-occupation, extreme drive to food seek and consume food that
severely diminish the quality of life for patients with PWS 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.,
obesity, diabetes, cardiovascular disease) and mortality (e.g.,
stomach rupture, 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 and 92.9% rated body composition as either 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 DCCR (Diazoxide Choline) Extended-Release
TabletsDCCR is a novel, proprietary extended-release
dosage form containing the crystalline salt of diazoxide and 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 for the therapeutic use of diazoxide, diazoxide
choline and DCCR in patients with PWS. The DCCR development program
is supported by data from five completed Phase 1 clinical studies
in healthy volunteers and three completed Phase 2 clinical studies,
one of which was in patients with PWS. In the PWS Phase 3 clinical
development program, 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. Diazoxide choline has received Orphan Drug Designation
for the treatment of PWS in the U.S. and E.U., and Fast Track and
Breakthrough Designations in the U.S.
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 (diazoxide choline) extended-release tablets, a
once-daily oral tablet for the treatment of PWS, recently completed
its Phase 3 development program to support a planned NDA
submission. 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 the 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 the projected timeline of our NDA
submission, whether FDA will agree with our interpretation of the
data or the adequacy of data to support an NDA, the FDA’s review of
our NDA, 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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