Soleno Raises $15 Million to Advance Experimental Candidate for Prader-Willi Syndrome

Rare Daily Staff

Rare disease focused biotech Soleno Therapeutics said it priced an underwritten public offering for aggregate gross proceeds of approximately $14.8 million, before deducting the underwriting discount and other estimated offering expenses.

Proceeds from the offering will be used to fund Soleno’s current research and development efforts primarily focused on advancing its lead candidate, DCCR tablets for the treatment of Prader-Willi Syndrome (PWS), and to provide for general corporate purposes.

The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births in the United States. 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 such as obesity, diabetes, and cardiovascular disease, and mortality due to stomach rupture or choking, and accidental death due to food seeking behavior. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5 percent of respondents (parent and caregivers) rated hyperphagia and 92.9 percent 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.

DCCR (Diazoxide Choline Extended-Release) 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. Soleno has been in ongoing discussions with the FDA regarding additional data needed to support the submission of an NDA. Diazoxide choline has received Orphan Drug designation for the treatment of PWS in the U.S. and EU, and Fast Track designation in the U.S.

Photo: Anish Bhatnagar, CEO of Soleno Therapeutics