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Soleno Therapeutics Announces Submission of New Drug Application to the U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets for the Treatment of Prader-Willi Syndrome

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Soleno Therapeutics has submitted a New Drug Application (NDA) to the U.S. FDA for its drug DCCR (diazoxide choline) extended-release tablets, aimed at treating Prader-Willi syndrome (PWS) in individuals aged four and older suffering from hyperphagia.

This submission is a pivotal step for Soleno, offering a potential new therapeutic solution for PWS, a rare condition characterized by life-threatening overeating and other severe symptoms.

DCCR has received Breakthrough, Fast Track, and Orphan Drug designations in the U.S., as well as Orphan Drug designation in the E.U. The FDA will decide on the NDA's acceptance within 60 days, and Soleno has requested Priority Review, which could shorten the review period to six months.

Positive
  • Submission of NDA for DCCR to the FDA is a major milestone.
  • DCCR targets Prader-Willi syndrome, addressing unmet medical needs.
  • DCCR has Breakthrough, Fast Track, and Orphan Drug designations in the U.S. and Orphan Drug designation in the E.U.
  • Priority Review requested, potentially reducing review time to six months.
Negative
  • The FDA has yet to accept the NDA for review, creating uncertainty.
  • The success of DCCR is contingent on FDA approval, which is not guaranteed.

The submission of the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets is a significant marker in the progression of Soleno Therapeutics' pipeline. DCCR's potential in addressing hyperphagia in Prader-Willi Syndrome (PWS) is particularly noteworthy given the limited treatment options currently available for this rare genetic disorder. The Breakthrough and Fast Track Designations indicate a substantial need and promise in clinical efficacy, streamlining the approval process. With an imminent FDA review, stakeholders should be attentive to interim efficacy and safety data released during this period, which could impact Soleno’s trajectory and market reception.

The Orphan Drug Designation provides additional financial incentives and market exclusivity, important for small biopharmaceutical companies specializing in rare diseases. This regulatory framework can foster innovation by mitigating development costs and ensuring a sustainable path to market. Investors should evaluate the long-term market potential considering the unique competitive positioning and the significant unmet medical need in the PWS community.

From a financial perspective, the NDA submission for DCCR could herald substantial upside potential for Soleno Therapeutics. Notably, the Orphan Drug Designation suggests a future period of market exclusivity post-approval, which is beneficial for pricing power and revenue generation. The anticipated six-month Priority Review, if granted, could expedite commercialization, aligning with strategic revenue milestones and potentially enhancing investor confidence. However, investors should remain cautious of inherent risks associated with regulatory dependencies and the possibility of FDA requests for additional data, which could delay approval and impact financial forecasts.

Examining the financial statements, it's vital to assess the cash reserves and funding adequacy for the commercialization phase. Potential revenue from DCCR could significantly alter Soleno's financial landscape, but attention should be given to cash burn rates and the need for strategic partnerships or financing rounds to support ongoing operations and marketing efforts post-approval.

REDWOOD CITY, Calif., June 28, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia.

“Submission of the DCCR NDA to the FDA marks a significant milestone not only for Soleno, but for people living with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “This regulatory submission advances us one important step closer to our goal of bringing to market a new therapeutic for individuals with PWS that addresses the life-threatening hyperphagia and other key aspects of this serious and rare condition. We look forward to working with the FDA throughout the review process. We extend sincere gratitude to the team at Soleno, investigators, study site teams, advocacy organizations and most importantly, the individuals with PWS and their families who were instrumental in completing our DCCR development program.”

DCCR has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U. The FDA has 60 days to determine whether the NDA is accepted for review. Soleno has requested Priority Review of the NDA, which would provide a target review period of six months by the FDA after the NDA has been accepted.

About PWS
The 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 individuals 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 mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents 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 Tablets
DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals 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 individuals 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 recently submitted an NDA to the FDA, supported by its Phase 3 development program, for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS). 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 the timing of any regulatory process, acceptance by the FDA of Soleno’s NDA, 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 FDA’s filing and / or review of Soleno’s 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 Ritchie
LifeSci Advisors, LLC
212-915-2578


FAQ

What is the latest news on Soleno Therapeutics' NDA submission for DCCR?

Soleno Therapeutics has submitted a New Drug Application (NDA) to the U.S. FDA for DCCR (diazoxide choline) extended-release tablets for treating Prader-Willi syndrome.

What condition is Soleno Therapeutics' DCCR designed to treat?

DCCR is designed to treat Prader-Willi syndrome (PWS), specifically targeting hyperphagia in individuals aged four and older.

What designations has DCCR received in the U.S. and E.U.?

DCCR has received Breakthrough, Fast Track, and Orphan Drug designations in the U.S., and Orphan Drug designation in the E.U.

What is the next step for the NDA submitted by Soleno Therapeutics?

The FDA has 60 days to decide whether to accept the NDA for review. Soleno has requested Priority Review, which could shorten the review period to six months.

What is Soleno Therapeutics' stock symbol?

Soleno Therapeutics' stock symbol is SLNO.

Soleno Therapeutics, Inc.

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