The company’s narcolepsy treatment shows strong growth, while its late-stage pipeline advances with five phase 3 trials and a novel orexin agonist.

Key takeaways:

• Harmony Biosciences has issued a 2026 net revenue guidance for WAKIX of $1.0 to $1.04 billion, positioning the narcolepsy drug for blockbuster status.
• Preliminary, unaudited net revenue for WAKIX in 2025 was approximately $868 million.
• The company is developing next-generation pitolisant formulations intended to extend the franchise into the 2040s.
• Its pipeline includes five ongoing phase 3 registrational trials in five distinct central nervous system indications.
• A phase 1 trial for a potential orexin-2 agonist is underway, with clinical data anticipated in mid-2026.

Harmony Biosciences Holdings Inc announced preliminary financial results for 2025 and issued revenue guidance for 2026, projecting that its narcolepsy drug WAKIX (pitolisant) will generate over $1 billion in net revenue.

The company reported preliminary, unaudited net product revenue for WAKIX of approximately $868 million for the full year 2025 and $243 million for the fourth quarter. The 2026 net revenue guidance is set between $1.0 and $1.04 billion.

“With WAKIX on track to achieve revenue of over $1 billion in narcolepsy in 2026, Harmony is entering its next phase of growth with significant momentum,” says Jeffrey M Dayno, MD, president and CEO of Harmony Biosciences, in a release. “Our proven commercial engine is driving exceptional performance with WAKIX, our next-gen formulations are positioned to extend and expand the pitolisant franchise well into the 2040s, and we continue to advance our late-stage pipeline with five ongoing phase 3 registrational trials toward five distinct CNS [central nervous system] indications.”

WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist approved by the US Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and for excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy.

In addition to the commercial performance of WAKIX, Harmony highlighted its late-stage pipeline, which includes five ongoing phase 3 trials for five separate central nervous system indications. The company is also advancing a potential orexin-2 agonist, BP1.15205, which is currently in a phase 1 trial with clinical data expected in mid-2026.

Pitolisant Franchise Strategy

• WAKIX: On track to achieve blockbuster status in narcolepsy
  • Net revenue projected between $1.0 billion to $1.04 billion for the full year ending December 31, 2026
  • Pitolisant in Prader-Willi syndrome
    • Phase 3 topline data readout in 2H 2026
    • Supports Pediatric Exclusivity for WAKIX: Fulfills the last regulatory requirement for six months of additional regulatory exclusivity on top of the longest patent for WAKIX
• Pitolisant GR (gastro-resistant): On track to extend pitolisant franchise into the 2040s
  • NDA submission in Q2 2026; anticipated PDUFA date in Q1 2027
    • Approximately 80-90% of patients with narcolepsy experience GI symptoms; pitolisant GR is designed to minimize the worsening of these symptoms
    • Ability to initiate treatment at therapeutic dose with no titration
  • Utility patents filed to extend franchise to the 2040s
• Pitolisant HD (high dose): Opportunity to expand pitolisant franchise with differentiated indications
  • Phase 3 registrational clinical trials ongoing in narcolepsy (ONSTRIDE 1) and idiopathic hypersomnia (ONSTRIDE 2)
    • Topline data in 2027; anticipated PDUFA date in 2028
    • Enhanced formulation with optimized PK profile, GR coating and higher dose to drive greater efficacy
    • Differentiated indications: fatigue in narcolepsy and sleep inertia in IH
  • Utility patents filed to expand franchise to the 2040s

Pipeline

• Orexin-2 receptor agonist (BP1.15205)
  • First subject dosed in phase 1 PK trial in Q4 2025
  • Phase 1 clinical PK data in mid-2026
  • Potential best-in-class orexin-2 receptor agonist based on a novel chemical scaffold, preclinical potency, selectivity, safety, and efficacy data, and potential for once-a-day dosing
• EPX-100 (clemizole hydrochloride)
  • One of the most advanced development programs in the 5HT2 (serotonin) agonist class
  • Enrollment ongoing for Phase 3 registrational trial in Dravet syndrome (ARGUS Study) with topline data anticipated in 1H 2027
    • Safety and effectiveness data from the open-label extension study in DS was presented at AES meeting in December 2025
  • Enrollment ongoing for Phase 3 registrational trial in patients with Lennox-Gastaut syndrome (LIGHTHOUSE Study) with topline data anticipated in 1H 2027

ZYN002 Update

• After an in-depth review of the data from the RECONNECT study, the ZYN002 program in Fragile X syndrome is being phased out and Harmony is no longer pursuing a 22q deletion syndrome indication.

Learn More About What Harmony Is Working On: