Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) reported robust financial and operational results for the first quarter of 2025, underscoring the company’s continued momentum and strategic focus on advancing its late-stage pipeline while reaffirming full-year revenue guidance. The company achieved a 20% year-over-year revenue increase for WAKIX® (pitolisant), its flagship product for narcolepsy, positioning it as a potential billion-dollar opportunity in the U.S. market alone. Harmony also reported its fourth consecutive year of profitability and a cash and investments balance exceeding $600 million.
“Building off of our strong foundation of commercial success, we are poised for significant momentum throughout the rest of the year, driven by the upcoming catalysts from our robust, late-stage pipeline,” said Jeffrey M. Dayno, M.D., President and CEO of Harmony Biosciences. “Our next major clinical milestone is the topline data readout from our Phase 3 registrational trial of ZYN002 in Fragile X syndrome, the RECONNECT Study, expected in the third quarter. A positive result could pave the way for the first-ever approved treatment for this patient population.”
Strong Commercial Performance in Sleep/Wake Disorders
Harmony’s Sleep/Wake franchise continues to anchor its commercial success. In Q1 2025, WAKIX generated net revenue of $184.7 million, up from $154.6 million in the same period last year. The number of patients on therapy increased to approximately 7,200 during the quarter, with an exit rate near 7,300. The company reiterated its 2025 revenue guidance for WAKIX, projected in the range of $820 to $860 million.
Advancing Next-Generation Pitolisant Programs
To extend the clinical and commercial life of pitolisant, Harmony is advancing two novel formulations:
- Pitolisant HD (High-Dose): Engineered for enhanced efficacy in narcolepsy and other hypersomnia conditions, Pitolisant HD is poised to begin two Phase 3 registrational trials in Q4 2025—one in narcolepsy and another in idiopathic hypersomnia (IH). These trials will incorporate endpoints on fatigue and sleep inertia to support differentiated labeling. Harmony has filed utility patents through 2044 for these indications.
- Pitolisant GR (Gastro-Resistant): A pivotal bioequivalence study for this formulation began in March 2025, with topline data expected in Q3 2025. If successful, a potential FDA approval (PDUFA) could be secured by 2026. Patents for Pitolisant GR also extend to 2044.
Additionally, the company is developing an Orexin-2 receptor agonist (BP1.15205), which could emerge as a best-in-class therapy based on its novel scaffold, preclinical efficacy, and once-daily dosing potential. Harmony plans to submit an IMPD in mid-2025 and initiate a first-in-human study in the second half of 2025, with initial clinical data anticipated in 2026.
Pipeline Progress in Neurobehavioral Disorders
ZYN002, a transdermal cannabidiol gel, is the lead candidate in Harmony’s neurobehavioral franchise. The RECONNECT Study, a Phase 3 trial in Fragile X syndrome (FXS), has completed recruitment, with topline results expected in Q3 2025. The study aims to confirm previous positive outcomes in a genetically defined subgroup from the earlier CONNECT study.
Open-label extension data from RECONNECT continue to show promise. Over 60% of participants exhibited at least a 9-point improvement in the ABC-CFXS Irritability subscale, a measure of behavioral symptoms, sustained over a three-year period.
If approved, ZYN002 could become the first treatment for FXS, which affects an estimated 80,000 individuals in the U.S. Harmony holds global rights and is also preparing to initiate a Phase 3 trial in 22q11.2 deletion syndrome (22q) by Q4 2025, pending favorable RECONNECT data.
Rare Epilepsy Franchise Development
In the rare epilepsy space, Harmony is progressing with two lead compounds:
- EPX-100 (clemizole hydrochloride): The most advanced candidate in the 5HT2 agonist class, currently enrolling in two Phase 3 registrational trials: the ARGUS Study for Dravet syndrome and the LIGHTHOUSE Study for Lennox-Gastaut syndrome. Topline data are expected in 2026.
- EPX-200 (lorcaserin hydrochloride): This candidate, supported by preclinical and early clinical data, is in the IND-enabling phase targeting developmental and epileptic encephalopathies (DEEs).
First Quarter 2025 Financial Overview
Harmony delivered a strong financial performance for Q1 2025:
- Net Product Revenue: $184.7 million, up 20% from $154.6 million in Q1 2024.
- GAAP Net Income: $45.6 million, or $0.78 per diluted share, compared to $38.3 million, or $0.67 per diluted share in Q1 2024.
- Non-GAAP Adjusted Net Income: $60.4 million, or $1.03 per diluted share, versus $50.7 million, or $0.88 per diluted share in Q1 2024.
This performance reflects strong commercial uptake of WAKIX and expanded reach across a market of approximately 80,000 diagnosed narcolepsy patients in the U.S. Notably, Harmony’s promotional efforts target around 9,000 healthcare professionals (HCPs), including 5,000 not participating in oxybate Risk Evaluation and Mitigation Strategy (REMS) programs, further broadening access.
Operating Expenses
Harmony continues to invest in its growth trajectory:
- Research and Development (R&D): $34.5 million in Q1 2025, up 56% from $22.2 million in Q1 2024.
- Sales and Marketing: $30.7 million, a 13% increase from $27.2 million in Q1 2024.
- General and Administrative (G&A): $31.2 million, up 22% from $25.7 million in the prior year period.
- Total Operating Expenses: $96.5 million, representing a 29% year-over-year increase.
These increases are largely driven by accelerated pipeline development and expanded commercialization activities.
Positioned for Long-Term Growth
With a strong balance sheet, multiple near-term catalysts, and a diversified late-stage pipeline, Harmony Biosciences is strategically positioned for sustained long-term growth. The company remains focused on delivering innovative therapies to address unmet needs in central nervous system (CNS) disorders, while creating substantial value for shareholders.
As the company looks ahead to major clinical readouts and additional regulatory milestones, Harmony continues to build on its foundation as a profitable, self-sustaining biotechnology company with global ambitions.
About Narcolepsy
Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed.
