REDWOOD CITY, Calif. - Soleno Therapeutics Inc. (NASDAQ: NASDAQ:SLNO), a biopharmaceutical company focused on developing treatments for rare diseases, announced today significant findings from its Phase 3 study evaluating DCCR tablets for Prader-Willi syndrome (PWS). The study results, comparing DCCR-treated participants to a natural history cohort, showed marked improvements in hyperphagia and PWS-related behaviors.
The research, comparing data from Soleno's Phase 3 study and its open-label extension with the PATH for PWS natural history study, was published in the Journal of Neurodevelopmental Disorders.
The findings indicate that the use of DCCR (Diazoxide Choline) Extended-Release tablets led to statistically significant improvements in both hyperphagia and behavioral domains associated with PWS after 6 and 12 months of treatment.
Hyperphagia, an unrelenting hunger that leads to life-threatening obesity, is a hallmark symptom of PWS. The study utilized caregiver-completed questionnaires to assess changes in hyperphagia and PWS-related behaviors such as aggression, anxiety, and depression. The results suggest DCCR could substantially enhance the quality of life for individuals living with PWS and their families.
The Phase 3 study, known as DESTINY PWS, involved 127 patients at 29 sites in the U.S. and UK. Participants who completed this study were enrolled in the open-label extension study C602. The PATH for PWS study, sponsored by the Foundation for Prader-Willi Research (FPWR), provided a natural history comparison group and aimed to deepen the understanding of PWS, especially long-term behavioral changes.
Dr. Theresa Strong from FPWR expressed optimism about the potential of DCCR to provide significant improvement for those with PWS. Principal Investigators of the PATH Study, Dr. Jennifer Miller and Dr. Shawn McCandless, echoed this sentiment, highlighting the meaningful improvement in behavioral complications of PWS observed with DCCR treatment.
DCCR has received Orphan Drug Designation for PWS treatment in the U.S. and E.U., as well as Fast Track and Breakthrough Designations in the U.S. Soleno is preparing for a planned NDA submission based on these promising results.
This information is based on a press release statement from Soleno Therapeutics. The company's forward-looking statements involve risks and uncertainties, including the FDA's acceptance and review of the NDA submission. Soleno has not provided a timeline for regulatory approval or commercial availability of DCCR for PWS treatment.
InvestingPro Insights
Soleno Therapeutics Inc. (NASDAQ: SLNO) has recently caught the attention of the biopharmaceutical industry with its promising Phase 3 study results for DCCR tablets. As the company prepares for its New Drug Application (NDA) submission, investors and industry observers are closely monitoring its financial health and market performance. Here are key insights based on the latest data from InvestingPro:
The company's market capitalization stands at approximately $1.52 billion, reflecting investor confidence in its growth potential, especially in light of the recent study findings. Despite challenging market conditions, Soleno Therapeutics has demonstrated a significant return over the last week, with a 13.43% increase in its stock price. Additionally, over the past six months, the stock has seen a large price uptick of 90.9%, signaling strong investor enthusiasm for the company's prospects.
From a financial perspective, Soleno Therapeutics has faced some headwinds, as indicated by its negative operating income of -$38.67 million over the last twelve months as of Q4 2023. This is consistent with its status as a development-stage biopharmaceutical company, where significant investment in research and development is typical before reaching profitability. However, according to InvestingPro Tips, analysts predict that the company will be profitable this year, providing a potential upside for investors.
For those interested in exploring further, InvestingPro offers additional InvestingPro Tips that delve deeper into Soleno Therapeutics' financial metrics and market performance. Currently, there are 11 more tips available on InvestingPro, which can be accessed at: https://www.investing.com/pro/SLNO. To enhance your investing strategy with these insights, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
Investors should note that Soleno does not pay a dividend to shareholders, which is typical for companies in the growth phase and those focusing on reinvesting earnings into research and development. The company's commitment to innovation in rare disease treatments remains a central part of its value proposition to the market.
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