Soleno Therapeutics (SLNO) Jefferies London Healthcare Conference 2024 summary

Regulatory and clinical development

• NDA for DCCR in Prader-Willi syndrome (PWS) submitted in June and accepted by FDA in August, with a PDUFA date set for December 27, 2024.

• DCCR holds Breakthrough, Fast Track, and Orphan designations in the U.S. and EU, and has Priority Review status.

• Clinical trials showed statistically significant improvements in hyperphagia and PWS-related behaviors, especially after adjusting for COVID-impacted data.

• Open-label extension and randomized withdrawal studies confirmed durable efficacy and safety, with improvements in metabolic markers and body composition.

• No significant difference in clinical outcomes between pediatric and adult populations.

Market opportunity and commercialization plans

• U.S. PWS market estimated at $2 billion, with over 10,000 identified patients and high diagnostic rates in newborns.

• 95% of healthcare providers surveyed are willing to prescribe DCCR; key prescribers are concentrated among 300 HCPs.

• Commercial strategy targets both pediatric and adult segments, focusing on endocrinologists and psychiatrists.

• Infrastructure for launch includes supply chain partners, payer engagement, field force recruitment, and ongoing advocacy.

• European market opportunity identified, with plans to submit an MAA in H1 2025 and openness to partnership for commercialization.

Intellectual property and stakeholder engagement

• DCCR protected by three patent families, with potential exclusivity in the U.S. through 2034 and up to 2039 for certain indications.

• Strong engagement with patient advocacy groups, including a petition with over 14,000 signatures supporting FDA review.

• Ongoing collaboration with scientific and medical communities, with recent data presented at conferences.

• Extensive analytics and stakeholder engagement to support launch and market access.