Soleno Therapeutics Announces Peer-Reviewed Publication of Results from Long-Term Open-Label Study of DCCR in Prader-Willi Syndrome
06 Novembro 2023 - 10:00AM
Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a
clinical-stage biopharmaceutical company developing novel
therapeutics for the treatment of rare diseases, today announced
publication of one-year results from the Phase 3 DESTINY PWS study
and the open-label extension period of Study C602 evaluating
investigational, once-daily DCCR (Diazoxide Choline)
Extended-Release tablets for patients with Prader-Willi syndrome
(PWS). The paper, entitled “Diazoxide Choline Extended-Release
Tablet in People with Prader-Willi Syndrome: Results from Long-Term
Open-Label Study,” was published in the peer-reviewed journal
Obesity and can be found online here.
“We are pleased to publish results from the long-term study of
DCCR demonstrating significant, sustained reductions in
hyperphagia,” said Anish Bhatnagar, M.D., Chief Executive
Officer of Soleno Therapeutics. “Together with positive
results from our recently completed randomized withdrawal period of
Study C602, DCCR has shown significant and clinically meaningful
benefits for the most burdensome symptoms of PWS. We believe it has
the potential to be a safe and effective therapy for patients with
PWS. We anticipate submitting an NDA to the FDA for DCCR in
mid-2024.”
The publication features results from 52-week administration of
DCCR in participants with PWS enrolled in DESTINY PWS and/or Study
C602, the long-term open-label extension study of participants who
completed DESTINY PWS. The primary efficacy endpoint was change in
hyperphagia total score from baseline, as measured using the
Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Other
endpoints included behavioral assessments using the PWS Profile
Questionnaire (PWSP), body composition, hormonal and metabolic
measures, disease severity and safety.
Key Highlights from the Publication:
Primary Endpoint: Hyperphagia
- Statistically significant, clinically meaningful decreases in
HQ-CT total score (mean [SE]) from baseline in overall population
after receiving DCCR for 52 weeks (-9.9 [0.77]; p<0.0001).
- Highly significant, clinically meaningful decreases in HQ-CT
total score from baseline were also observed after receiving DCCR
for 13, 26, and 39 weeks (all p<0.0001).
- In participants with severe hyperphagia at baseline, 52-week
DCCR treatment resulted in significantly greater reduction in HQ-CT
score compared to the overall population (-15.2 [1.39];
p<0.0001).
Other Endpoints
- PWS related behaviors: Statistically
significant improvements in all behavioral domains of the PWSP,
which consist of aggressive behaviors, anxiety, compulsivity,
depression, disordered thinking, and rigidity/irritability (all
p<0.0001).
- Body composition: Significant improvements in
lean body mass:fat mass ratio were seen (p=0.0005), consistent with
increases in lean body mass (p<0.0001) and no significant
changes in body fat mass.
- Hormonal and metabolic measures: Significant
reductions in serum leptin (p<0.0001) and insulin (p=0.0004) and
improvement in Homeostatic Model Assessment for Insulin Resistance
(HOMA-IR) (p=0.0033), consistent with the proposed mechanism of
action of DCCR in PWS. In addition, a significant increase in
adiponectin was observed (p<0.0001).
- Disease severity: Significant reduction in
disease severity as assessed by both clinicians and caregivers
using Clinical Global Impression of Severity and Caregiver Global
Impression of Severity scores, respectively (both
p<0.0001).
- Safety: DCCR was well-tolerated overall.
Safety results are consistent with the safety profile of DCCR, with
the most common drug-related treatment-emergent adverse events of
hypertrichosis, peripheral edema, and hyperglycemia, the majority
of which were grade 1 and infrequently resulting in study drug
discontinuation.
DCCR has orphan designation for the treatment of PWS in the U.S.
and EU and Fast Track designation from the U.S. FDA. Soleno
recently announced positive topline results from its randomized
withdrawal phase of Study C602. The U.S. Food and Drug
Administration (FDA) previously acknowledged that data from this
study has the potential to support an NDA submission for DCCR.
Soleno anticipates submission of an NDA to the FDA in mid-2024.
About PWSThe Prader-Willi Syndrome Association
USA estimates that PWS occurs in one in every 15,000 live births in
the U.S. 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., 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.
Diazoxide choline has received Orphan Drug Designation for the
treatment of PWS in the U.S. and E.U., and Fast Track Designation
in the U.S.
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 on the therapeutic use of diazoxide 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 Phase 3 DESTINY PWS 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. After one-year of open-label DCCR, participants
experienced significant, clinically meaningful reductions from
baseline in hyperphagia and other common PWS behaviors. In a
randomized withdrawal period of Study C602, participants who were
randomized to placebo had highly statistically significant
increases from baseline in HQ-CT total score compared to those
randomized to continue on DCCR (p = 0.0022).
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 extended-release tablets, a once-daily oral tablet
for the treatment of Prader-Willi syndrome (PWS), is currently
being evaluated in a Phase 3 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 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 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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