Harmony Biosciences (HRMY) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Business transformation and financial position

• Company has undergone significant transformation, expanding its pipeline and diversifying its portfolio over the past year.

• Maintains a strong cash position with $434 million and continues to generate positive cash flow and profitability.

• Revenue from WAKIX is projected at $700–$720 million in net sales for the year, with a patient base nearing 7,000.

• WAKIX is positioned as a differentiated, non-scheduled treatment in narcolepsy, appealing to a broad prescriber and patient base.

• The company is targeting a billion-dollar-plus opportunity in adult narcolepsy, with further growth expected from label expansion.

WAKIX market positioning and clinical utility

• WAKIX is the only non-scheduled FDA-approved treatment for narcolepsy, used by both Type 1 and Type 2 patients.

• About 80% of WAKIX use is as add-on therapy, reflecting the polypharmacy nature of the market.

• Its non-scheduled status allows broader access, with 9,000 potential prescribers versus 4,000 for oxybates.

• WAKIX is effective for both excessive daytime sleepiness and cataplexy, with a strong safety and efficacy profile.

• Prescribing trends are shifting toward Type 2 narcolepsy, mirroring clinical presentations.

Expansion into idiopathic hypersomnia (IH)

• Pursuing a supplemental NDA for WAKIX in IH, with submission planned for Q4.

• IH represents a significant unmet need, with only one approved treatment (Xywav), which is a scheduled, restricted-access product.

• Phase III INTUNE study showed strong signals in open-label phase; long-term safety and efficacy data are being collected.

• Real-world evidence from Europe and compassionate use programs support the benefit-risk profile.

• Commercial infrastructure is ready for IH, with high overlap among prescribers and existing sales channels.