WAKIX® (pitolisant) Net Revenue of $184.7 Million for First Quarter 2025; Representing 20% Growth Year-over-Year; Reiterates Guidance of $820-$860M
Net Income Grew 19% Year-over-Year, Building on Four Consecutive Years of Profitability; Increased Cash and Investments to Over $600 Million on Balance Sheet
Completed Recruitment of Phase 3 Registrational Trial of ZYN002 in Fragile X Syndrome; On Track for Topline Data in Q3
BP1.15205, Potential Best in Class Orexin, Data to Be Presented at SLEEP 2025 Conference in June
On Track for Initiation of Next-Generation Pitolisant-HD Phase 3 Registrational Trials in Narcolepsy & IH in Q4
Conference Call and Webcast to be Held Today at 8:30 a.m. ET
PLYMOUTH MEETING, Pa.–(BUSINESS WIRE)–Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced strong year-over-year revenue growth for WAKIX® of 20% in the first quarter 2025, on its way to a potential $1B+ opportunity in narcolepsy alone and poised for additional growth from its next-gen pitolisant development programs. The company has demonstrated four consecutive years of profitability and has grown its cash and investments position to over $600M.
“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 Chief Executive Officer of Harmony Biosciences. “Our next major clinical milestone, topline data readout from our Phase 3 registrational trial of ZYN002 in patients with Fragile X syndrome, the RECONNECT Study, is on track for Q3. A positive readout could put us on a path toward the first ever approved treatment for this patient community. I am proud of the unique profile we have created at Harmony, a profitable, self-funding biotech company, with a robust pipeline, that has the potential to help hundreds of thousands of patients living with unmet medical needs while creating significant, long-term value.”
Franchise Highlights
Sleep/Wake Franchise
WAKIX in Narcolepsy
- Net Revenue was $184.7 million for Q1 2025
- 2025 Net Revenue projected between $820 to $860 million
- The average number of patients on WAKIX increased to approximately 7,200 for Q1 2025 and we exited the quarter with approximately 7,300 patients
Pitolisant HD (high-dose)
- Higher dose and optimized pharmacokinetic profile designed for greater efficacy in narcolepsy; development program to pursue multiple additional indications
- Phase 3 registrational trial in narcolepsy designed for greater efficacy in excessive daytime sleepiness and cataplexy; also to include endpoint on narcolepsy-related fatigue in pursuit of differentiated label
- Phase 3 registrational trial in IH to include endpoint on sleep inertia in pursuit of differentiated label
- On track to initiate Phase 3 registrational trials in both narcolepsy and IH in Q4 2025 with potential PDUFA dates in 2028
- Utility patents filed out to 2044 for narcolepsy and IH
Pitolisant GR (gastro-resistant)
- Pivotal bioequivalence study initiated in March 2025
- Topline data readout anticipated in Q3 2025 with potential PDUFA date in 2026
- Utility patents filed out to 2044
Orexin-2 receptor agonist (BP1.15205)
- Comprehensive preclinical safety and efficacy data to be presented at SLEEP 2025 (June)
- Potential to be best-in-class orexin-2 receptor agonist based on a novel chemical scaffold, preclinical potency, selectivity and safety data, as well as its potential for once-a-day dosing
- IMPD submission on track for mid-2025; first-in-human study expected to initiate 2H 2025 with clinical data anticipated in 2026
Neurobehavioral Franchise
ZYN002
- Completed recruitment of Phase 3 registrational trial, the RECONNECT Study, in patients with Fragile X syndrome (FXS); on track for topline data readout in Q3
- RECONNECT Study is designed to confirm the positive findings from the prespecified analysis of the primary outcome in the subgroup of patients with complete methylation from the Phase 2/3 CONNECT Study
- Promising new open-label extension (OLE) data shows benefit in patients with FXS
- Participants in the OLE trial demonstrated clinically meaningful improvements in behavioral symptoms as measured by the Aberrant Behavior Checklist – Community (ABC-CFXS Irritability)
- More than 60% of participants achieved clinically meaningful improvement of at least 9 points on the ABC-CFXS Irritability scores out to 3 years
- Potential to be the first and only approved treatment for patients with FXS; 80,000 patients in the U.S. and Harmony possesses global rights
- Prepared to initiate Phase 3 registrational trial in 22q11.2 deletion syndrome (22q) in Q4 2025 (pending positive data from the RECONNECT Study)
Rare Epilepsy Franchise
EPX-100 (clemizole hydrochloride)
- Most advanced development program in the 5HT2 (serotonin) agonist class
- Recruitment ongoing for Phase 3 registrational trial in Dravet syndrome (ARGUS Study) with topline data anticipated in 2026
- Recruitment ongoing for Phase 3 registrational trial in patients with Lennox-Gastaut syndrome (LIGHTHOUSE Study) with topline data anticipated in 2026
EPX-200 (lorcaserin hydrochloride)
- Proven mechanism of action in developmental and epileptic encephalopathies (DEEs) confirmed via non-clinical and clinical data
- Currently in IND enabling stage
First Quarter 2025 Financial Results
Net product revenue for the quarter ended March 31, 2025, was $184.7 million, compared to $154.6 million for the same period in 2024. The 20% growth versus the same period in 2024 is primarily attributed to strong commercial sales of WAKIX driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the U.S.) and the broad clinical utility of WAKIX across the approximately 9,000 HCPs that we call on (about 5,000 of whom do not participate in an oxybate REMS program).
GAAP net income for the quarter ended March 31, 2025, was $45.6 million, or $0.78 earnings per diluted share, compared to GAAP net income of $38.3 million, or $0.67 earnings per diluted share, for the same period in 2024. Non-GAAP adjusted net income was $60.4 million, or $1.03 earnings per diluted share, for the quarter ended March 31, 2025, compared to Non-GAAP adjusted net income of $50.7 million, or $0.88 per diluted share, for the same period in 2024.
Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release.
Harmony’s operating expenses include the following:
- Research and Development expenses were $34.5 million in the first quarter of 2025, as compared to $22.2 million for the same quarter in 2024, representing a 56% increase;
- Sales and Marketing expenses were $30.7 million in the first quarter of 2025, as compared to $27.2 million for the same quarter in 2024, representing a 13% increase;
- General and Administrative expenses were $31.2 million in the first quarter of 2025, as compared to $25.7 million for the same quarter in 2024, representing a 22% increase; and
- Total Operating Expenses were $96.5 million in the first quarter of 2025, as compared to $75.1 million for the same quarter in 2024, representing a 29% increase.
As of March 31, 2025, Harmony had cash, cash equivalents and investments of $610.2 million, compared to $576.1 million as of December 31, 2024.
2025 Net Product Revenue Guidance
Expect full year 2025 net product revenue of $820 million to $860 million.
Conference Call Today at 8:30 a.m. ET
We are hosting our first quarter 2025 financial results conference call and webcast today, beginning at 8:30 a.m. Eastern Time. The live and replay webcast of the call will be available on the investor relations page of our website https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial 800-267-6316 (domestic) or 203-518-9783 (international), and reference passcode HRMYQ125.
