Harmony Biosciences (HRMY) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Leadership and strategic direction

• Leadership team brings decades of experience from major pharma and biotech firms, with recent additions strengthening management.

• Company is entering a new growth phase, leveraging a strong balance sheet and commercial success.

• Focus remains on both organic pipeline growth and business development to diversify revenue streams.

• Business development targets late-stage or on-market assets to bridge revenue post-2028.

• Multiple transactions are likely, with a preference for value-creating deals in sleep and rare CNS disorders.

Pipeline and R&D focus

• BP-205, an orexin 2 receptor agonist, is positioned as a potential best-in-class asset due to its potency, selectivity, and once-daily dosing profile.

• The molecule shows high potency and sufficient selectivity, with a unique chemical structure minimizing safety concerns.

• Clinical data to date indicate rapid onset and dose proportionality, supporting its use in hypersomnia and related disorders.

• Multiple phase II proof-of-concept studies are planned for mid-next year, targeting both sleep-wake and broader CNS indications.

• Plans include building a portfolio of orexin-2 agonists, not just BP-205, to address various market segments.

Business development and financial strategy

• Business development is a high strategic priority, with ongoing evaluation of M&A and licensing opportunities.

• Target assets are expected to contribute revenue between 2028 and 2032, aligning with WAKIX loss of exclusivity.

• The company is unlikely to allocate all available capital to a single transaction, preferring multiple deals.

• Nearly $1 billion in cash enables simultaneous investment in internal R&D and external BD opportunities.

• Strategic focus remains on sleep, epilepsy, and rare CNS disorders, leveraging existing expertise.