Soleno Therapeutics Receives Breakthrough Therapy Designation from U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome (PWS)
April 29 2024 - 7:00AM
Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage
biopharmaceutical company developing novel therapeutics for the
treatment of rare diseases, today announced that the U.S. Food and
Drug Administration (FDA) has granted Breakthrough Therapy
Designation to diazoxide choline for the treatment of adults and
children ages 4 years and older with genetically confirmed
Prader-Willi syndrome (PWS) who have hyperphagia. The designation
reflects the Agency’s determination that, based on an assessment of
the preliminary data from the Phase 3 clinical development program,
diazoxide choline may demonstrate substantial improvement on a
clinically significant endpoint(s) over available therapies.
“The granting of Breakthrough Therapy Designation, the first for
a drug being developed for the treatment of PWS, marks another
important milestone for our DCCR clinical development program,”
said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno
Therapeutics. “This important designation is confirmation that the
FDA views PWS as a serious condition and is an indication of DCCR’s
potential to be a safe and effective treatment for PWS. We remain
focused on preparing our NDA submission for DCCR in PWS, which we
continue to expect will occur in mid-2024.”
The FDA's Breakthrough Therapy Designation is intended to
expedite the development and review of drugs in the U.S. that are
intended to treat a serious condition, when preliminary clinical
evidence indicates the drug may demonstrate substantial improvement
over available therapy on a clinically significant endpoint(s).
With Breakthrough Therapy Designation, FDA provides intensive
guidance and organizational commitment involving senior managers in
a proactive, collaborative, cross-disciplinary review, and may also
allow for priority review and other actions to expedite review.
Diazoxide choline has Orphan Drug Designation for PWS in the
U.S. and E.U., as well as Fast Track Designation in the U.S.
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 diazoxide choline, the crystalline salt of
diazoxide, and is administered once-daily. 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 extended-release tablets, a once-daily oral tablet,
recently completed its Phase 3 development program in patients with
PWS 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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