Harmony Biosciences Reports Strong Q2 2024 Financials and Progresses Pitolisant High-Dose Program Toward 2028 PDUFA Date

Harmony Biosciences Holdings, Inc. reported a significant 29% increase in net revenue year-over-year for the quarter ending June 30, 2024, reaching $172.8 million. The company attributes this growth to robust sales of WAKIX® (pitolisant), a treatment for excessive daytime sleepiness (EDS) in patients with narcolepsy. The average number of patients on WAKIX increased to approximately 6,550, up by 250 patients sequentially. This reinforces the confidence in WAKIX as a promising market opportunity.

The development of the next-generation high-dose (HD) pitolisant program is advancing, with a PDUFA (Prescription Drug User Fee Act) date anticipated in 2028. This initiative aims to extend the pitolisant franchise well beyond 2040. Furthermore, Harmony successfully upheld the WAKIX patent in the U.S. Patent and Trademark Office, which denied a petition for reexamination filed by a short seller. This final decision is non-appealable, underpinning the company's confidence in its intellectual property rights.

Harmony has also recently received FDA approval for WAKIX to treat EDS in pediatric patients aged six years and older with narcolepsy. The commercial launch for this indication began on July 1, 2024. The company plans to submit a supplemental new drug application (sNDA) for pitolisant in idiopathic hypersomnia (IH) by the fourth quarter of 2024.

Other key developments include the advancement of the pitolisant gastro-resistant (GR) formulation, with a dosing optimization study scheduled for the fourth quarter of 2024 and a pivotal bioequivalence study in the first quarter of 2025. This formulation has a potential PDUFA date in 2026 and could offer patent protection until 2044.

Additional pipeline progress includes ongoing patient enrollment in the Phase 3 TEMPO study for Prader-Willi syndrome (PWS) and preparations for Phase 3 trials of ZYN002 in Fragile X syndrome (FXS) and 22q11.2 deletion syndrome (22q). For rare epilepsy, the pivotal registrational trial (ARGUS) for Dravet syndrome (DS) with EPX-100 is ongoing, and a Phase 3 trial for Lennox-Gastaut syndrome (LGS) is expected to commence later this year.

Financially, Harmony's operating expenses increased significantly, primarily due to an upfront licensing fee related to the 2024 Bioprojet Sublicensing Agreement and an in-process research and development (IPR&D) charge from acquiring Epygenix. Research and development expenses for the second quarter of 2024 rose to $63.6 million from $15.0 million in the same period of 2023. Sales and marketing expenses also saw an increase of 16%, while general and administrative expenses grew by 19%.

Despite these higher operating costs, Harmony achieved a GAAP net income of $11.6 million for the quarter ended June 30, 2024, compared to $34.3 million for the same period in 2023. The decrease in GAAP net income was primarily attributed to significant upfront licensing fees and acquisition-related charges. However, non-GAAP adjusted net income reached $60.6 million, or $1.05 per diluted share, up from $45.9 million, or $0.76 per diluted share, in the same period of the previous year.

Harmony has reiterated its net product revenue guidance for the full year 2024, expecting it to be between $700 million and $720 million. As of June 30, 2024, the company had cash, cash equivalents, and investments totaling $434.1 million.

Additionally, Harmony will host an Investor Day in New York City on October 1, 2024, to provide further insights into its strategic initiatives and pipeline progress. The company will also conduct a conference call and webcast to discuss the second quarter 2024 financial results.