Harmony Biosciences (HRMY) investor relations material

Strategic vision and pillars

• Four main pillars: protect pitolisant IP to 2030s, grow WAKIX, advance orexin pipeline, and pursue business development with a strong balance sheet.

• Confident in IP protection through March 2030, supported by ongoing litigation and exclusive patents, with potential for settlement or extended exclusivity.

• Business development will focus on smaller, complementary neuroscience deals to drive revenue between 2028-2032, leveraging cash reserves.

Orexin 2 agonist program

• BP-205 is positioned as a potential best-in-class orexin 2 agonist, with unique structure, high potency, and once-daily dosing potential.

• Phase I data in healthy volunteers expected soon, with multiple ascending dose and sleep-deprived studies planned for late 2024 or early 2025.

• Broader development strategy includes targeting sleep-wake disorders and expanding into CNS indications like cognition, ADHD, and mood disorders.

• Differentiation expected through clinical profile, with focus on safety, tolerability, and efficacy at lower doses.

WAKIX franchise and lifecycle management

• WAKIX is on track for blockbuster status in 2024, driven by established efficacy, safety, and commercial innovation.

• NDA for gastro-resistant (GR) formulation submitted; target approval in Q1 2025, with utility patents extending to 2044.

• High-dose (HD) formulation aims for improved efficacy and fatigue reduction, with phase III trials in narcolepsy and idiopathic hypersomnia.

• GR and HD formulations expected to support franchise resilience post-2030, including new indications not covered by generics.