Harmony Biosciences (HRMY) Investor Day 2024 summary

Strategic vision and business model

• Transitioning to a diversified neurology company with three CNS franchises: sleep/wake, neurobehavioral, and rare epilepsies, each targeting $1–2 billion peak sales opportunities.

• Aims to become the leading patient-focused CNS company, targeting unmet medical needs in sleep/wake, neurobehavioral, and epilepsy disorders.

• Profitable, self-funding biotech model, leveraging strong cash flow from WAKIX to fund pipeline expansion and business development.

• Unique, scalable commercial model using proprietary data analytics (HARP) to drive patient engagement, prescriber targeting, and lifecycle management.

• Ongoing business development to further expand the pipeline, supported by over $430 million on the balance sheet.

Pipeline and product development

• WAKIX (pitolisant) is a foundational asset in narcolepsy, with double-digit revenue growth, ~$1B+ opportunity, and a CAGR of ~45% from 2020-2024.

• Lifecycle management includes pitolisant GR (PDUFA 2026) and pitolisant HD (PDUFA 2028), both with provisional patents to 2044, aiming to extend franchise revenue into the 2040s.

• Next-generation orexin-2 agonist (BP1.15205) in-licensed, with best-in-class potential, high potency, selectivity, and once-daily dosing; IND submission planned for mid-2025.

• ZYN002, a synthetic cannabidiol gel for Fragile X syndrome and 22q11.2 deletion syndrome, is in phase III, with top-line data expected mid-2025 and potential first approval in 2026.

• Clemizole hydrochloride (EPX-100) for Dravet and Lennox-Gastaut syndromes is in phase III, with top-line data expected in 2026 and a favorable risk-benefit profile compared to current therapies.

Sleep/wake franchise developments

• WAKIX® continues strong growth, with 2024 net sales guidance of $700–$720M and significant room for further patient expansion.

• Pitolisant HD viewed as superior to WAKIX®, with anticipated strong uptake and favorable market access.

• sNDA for pitolisant in idiopathic hypersomnia (IH) on track for Q4 2024 submission, supported by robust Phase 3 INTUNE data.