At Soleno, we are researching and developing medicines to treat rare genetic disorders, beginning with Prader-Willi syndrome (PWS), a rare and life-threatening neurobehavioral, metabolic and endocrine disorder characterized by hyperphagia (insatiable hunger), neurocognitive and behavioral difficulties. Our priority is to bring the first approved treatment to patients to manage the most burdensome aspects of this devastating disorder.
The 5 highest priority needs to address in the development of a new PWS therapeutic according to caregivers were:
- reducing hunger
- improving behavior around food
- improved metabolic health (reduces fat/increases muscle)
- reduced temper outburst frequency/severity
- reduced obsessive compulsive behavior
DCCR has the potential to treat other diseases.
DCCR may have the potential to address unmet needs in other disorders such as SH2B1 deficiency obesity, obesity associated with mutations in PCSK1 mutation, and SIM1 deficiency obesity. DCCR also has the potential to benefit patients who have life-threatening hypoglycemia, such as those with hyperinsulinism and Glycogen Storage Disease Type 1 (GSD 1).
Soleno has received Orphan Drug Designation for diazoxide choline, the active ingredient in DCCR, for the treatment of GSD 1 in the United States.
Soleno has received Orphan Drug Designation for DCCR for the treatment of PWS in the United States and in Europe and has been granted a Fast Tack Designation by FDA.
Based on the therapeutic potential of KATP channel activation in the treatment of endocrine disorders characterized by hyperphagia, the accumulation of excess body fat, and leptin and insulin resistance, we believe DCCR may have the potential to treat other disorders such as SH2B1 deficiency obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), and SIM1 deficiency obesity. In addition, it also has the potential to benefit patients who have life-threatening hypoglycemia, such as those with hyperinsulinism and Glycogen Storage Disease Type 1a, for which Orphan Designation has been granted in the United States.