REDWOOD CITY, Calif. - Soleno Therapeutics, Inc. (NASDAQ: NASDAQ:SLNO) has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate diazoxide choline, aimed at treating Prader-Willi syndrome (PWS), a rare genetic disorder.
This marks the first such designation for a drug targeting PWS, particularly for patients four years and older who experience hyperphagia, an unrelenting hunger leading to excessive eating and life-threatening obesity.
The FDA's decision is based on preliminary data from Phase 3 clinical trials that suggest diazoxide choline could substantially improve upon existing therapies for PWS. The company is on track to submit a New Drug Application (NDA) for the drug in mid-2024.
Prader-Willi Syndrome is estimated to affect one in every 15,000 live births and is characterized by a number of symptoms including hyperphagia, behavioral issues, cognitive disabilities, and low muscle tone. As of now, no approved therapies specifically target the hyperphagia and related metabolic, cognitive, and behavioral aspects of PWS.
Diazoxide choline is an extended-release tablet taken once daily, and it has been patented by Soleno for its therapeutic use in PWS patients. The drug has also been granted Orphan Drug Designation in the U.S. and E.U., as well as Fast Track Designation in the U.S.
Soleno's CEO, Anish Bhatnagar, M.D., expressed that the FDA's designation acknowledges the seriousness of PWS and the potential of DCCR, the extended-release formulation of diazoxide choline, to provide a significant treatment option.
The Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious conditions, providing intensive guidance from the FDA, and potentially leading to priority review.
Soleno, a clinical-stage biopharmaceutical company, is focused on developing novel treatments for rare diseases. The company's lead candidate, DCCR, has completed Phase 3 development to support the upcoming NDA submission.
This news article is based on a press release statement from Soleno Therapeutics.
InvestingPro Insights
As Soleno Therapeutics, Inc. (NASDAQ: SLNO) makes significant strides with its drug candidate diazoxide choline, the financial health of the company comes into focus. According to InvestingPro data, Soleno holds a market capitalization of approximately $1.27 billion USD, reflecting investor confidence in the company's growth potential.
Despite a challenging financial performance in the last twelve months of Q1 2023, with an operating loss of -$38.67 million USD and an EBITDA of -$36.71 million USD, the company's stock has shown remarkable resilience with a one-year price total return of 1024.33%.
Two key InvestingPro Tips for investors to consider: Soleno is expected to grow its net income this year, which could signal a turnaround from the previous period's performance. Moreover, the company does not pay a dividend, which is typical for clinical-stage biopharmaceutical companies that often reinvest earnings back into research and development.
Investors looking to further analyze Soleno's financial metrics and future outlook can find additional insights on InvestingPro. There are currently 10 more InvestingPro Tips available, which can be accessed with an exclusive offer: use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription at https://www.investing.com/pro/SLNO.
With the FDA's Breakthrough Therapy Designation for diazoxide choline, Soleno's commitment to addressing the unmet needs of PWS patients is clear. The financial data and expert tips from InvestingPro provide a broader context for investors, highlighting both the opportunities and challenges that lie ahead for the company.
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