Soleno Therapeutics Announces Peer-Reviewed Publication of Results from Previously Completed Phase 3 Trial of DCCR for Treatment of Prader-Willi Syndrome

Rhea-AI Summary

Soleno Therapeutics (NASDAQ: SLNO) announced the publication of results from its Phase 3 DESTINY PWS trial for DCCR (Diazoxide Choline) Extended-Release tablets in treating Prader-Willi Syndrome (PWS) in the peer-reviewed Journal of Clinical Endocrinology and Metabolism. The promising trial outcomes have prompted the initiation of a randomized withdrawal phase in the ongoing extension study C602, potentially paving the way for a New Drug Application submission. PWS affects 1 in 15,000 births, marked by severe hunger and related health issues, underscoring the urgent need for effective treatments.

Positive

• Results from Phase 3 trial published in a peer-reviewed journal.
• Initiated randomized withdrawal period in the open-label Phase 3 extension study C602.
• Promising results from DCCR potentially supporting a New Drug Application submission.

Negative

• None.

REDWOOD CITY, Calif., Jan. 26, 2023 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that results from the Company’s previously completed Phase 3 DESTINY PWS (C601) trial of DCCR (Diazoxide Choline) Extended-Release tablets for the treatment of Prader-Willi Syndrome (PWS) have been published in a peer-reviewed journal. The article, entitled, “Diazoxide Choline Extended-Release Tablet in People with Prader-Willi Syndrome: A Double-Blind Placebo-Controlled Trial,” was published in The Journal of Clinical Endocrinology and Metabolism (JCEM) and can be found here.

“We are pleased that the data from the C601 study of DCCR in PWS have been published in a prestigious peer-reviewed journal, JCEM,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “Based on the promising results from the trial, as well as discussions with the U.S. Food and Drug Administration, we initiated a randomized withdrawal period of the open-label Phase 3 extension study C602, which has the potential to support the submission of a New Drug Application. Trial subjects are currently being randomized at our clinical sites.”

About PWS
The 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 % body composition 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. 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 Tablets
DCCR 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 PWS patients. In the PWS Phase 3 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, 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 Statements
This 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 Ritchie
LifeSci Advisors, LLC
212-915-2578

FAQ

What are the results of the Phase 3 DESTINY PWS trial for SLNO?

The Phase 3 DESTINY PWS trial results for DCCR showed promise in addressing symptoms of Prader-Willi Syndrome and were published in a peer-reviewed journal.

What is the significance of the publication in the Journal of Clinical Endocrinology and Metabolism for SLNO?

The publication in a prestigious journal validates the trial's results and enhances credibility, potentially influencing future clinical and regulatory decisions.

What are the next steps for Soleno Therapeutics regarding DCCR?

Following the trial results, Soleno has initiated a randomized withdrawal period in the extension study C602, which may support a New Drug Application submission.

What is Prader-Willi Syndrome and why is DCCR important for SLNO?

Prader-Willi Syndrome is a rare disorder affecting appetite and metabolism, and DCCR aims to address key symptoms, which currently lack effective treatments.