Soleno Therapeutics (SLNO) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company overview and disease background

• Focuses on rare diseases, primarily Prader-Willi syndrome (PWS), with a team of about 60 people.

• PWS is a genetic neurobehavioral disorder affecting 1 in 15,000 births, with 10,000–20,000 patients in the U.S.

• Hallmark symptoms include hyperphagia, behavioral abnormalities, and need for lifelong supervision.

• Current standard of care is growth hormone, which does not address core symptoms like hyperphagia.

Drug profile and mechanism

• DCCR is a once-daily pill targeting KATP channels, reducing appetite by modulating hypothalamic neurons.

• The drug uses a choline salt for higher solubility and efficacy at lower doses than the parent molecule.

• Demonstrates both reduction in fat mass and increase in muscle mass, unlike GLP-1s.

Clinical development and efficacy

• Phase 3 program included a 2:1 randomized, double-blind study with 127 patients across 29 sites.

• Initial primary endpoint was not met due to COVID-19 impact, but pre-COVID and open-label extension data showed significant benefits.

• Randomized withdrawal study confirmed sustained improvements in hyperphagia and behavior.

• Hyperphagia scores improved by over 50%, with effects plateauing after 9–10 months.

• Behavioral and metabolic markers, including leptin and adiponectin, showed robust improvements.