Harmony Biosciences (HRMY) H.C. Wainwright "HCW@Home" Series summary

Commercial performance and growth outlook

• Wakix is projected to generate $714 million in 2024 and $870 million in 2025, with the franchise expected to surpass $1 billion in annual revenues by 2030.

• Wakix continues to show strong, steady growth, achieving a 20% increase in net sales year-over-year and reaching 7,200 average patients in Q1 2024.

• The commercial team is preparing for the launch of ZYN002, targeting a significant unmet need in Fragile X syndrome with an estimated 80,000 patients.

• The company maintains broad payer coverage (80% of lives) and leverages a unique commercial model with a single patient hub and specialty pharmacies.

• Guidance for 2025 expects continued patient growth, approaching 8,000 patients by year-end.

Pipeline development and innovation

• The pipeline includes eight assets across 13 development programs, with up to six in Phase 3 by year-end.

• ZYN002 (cannabidiol gel) in Fragile X syndrome is a major near-term catalyst, with top-line Phase 3 data expected this quarter and potential NDA submission soon after.

• EPX100 is in Phase 3 for Dravet and Lennox-Gastaut syndromes, with top-line data anticipated in 2026; differentiation is focused on safety and tolerability.

• Preclinical data for BP115205, an orexin-2 receptor agonist, shows high potency and dosing flexibility, with first-in-human studies planned for later this year.

• The company is also advancing next-generation pitolisant formulations (gastro-resistant and high-dose) to address GI symptoms and fatigue, with PDUFA dates in 2026 and 2028.

Strategic initiatives and capital allocation

• Over $600 million on the balance sheet enables self-funding of R&D and business development.

• Active business development includes a recent $15 million research collaboration with Cirq Biosciences for regenerative cell therapies targeting epilepsy and narcolepsy.

• The company is open to expanding its portfolio in sleep-wake, neurobehavioral, and epilepsy franchises, as well as adjacent orphan neuro opportunities.

• The commercial infrastructure is designed for efficient launches, leveraging centers of excellence and a closed distribution system.

• Dedicated business development team continues to seek early, mid, and late-stage opportunities.