Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) has reported a robust performance for the second quarter of 2025, with net revenue from its WAKIX® (pitolisant) franchise reaching $200.5 million, marking a 16% increase compared to the same period last year. The company also achieved an increase in its average patient population, adding 400 new patients to reach a total of 7,600 during this quarter. This growth trajectory positions WAKIX as a potential blockbuster in the narcolepsy treatment market, which is estimated at over $1 billion.
Significant Developments in Clinical Trials
In addition to its revenue growth, Harmony announced plans to share topline data from its Phase 3 registrational clinical trial of ZYN002 for Fragile X syndrome in the third quarter of 2025. This trial aims to build upon the promising results from the previously conducted CONNECT study, which highlighted significant improvements in symptoms for over half of the participants. ZYN002 is a synthetic cannabidiol product designed for transdermal delivery, ensuring stable blood levels and facilitating easier administration for patients who may struggle with oral medications.
Financial Strength and Future Outlook
Harmony has demonstrated financial resilience, maintaining profitability while investing in a late-stage pipeline. With over $672 million in cash and investments, the company continues to solidify its position as a self-sustaining biotech firm focused on neurological conditions. The CEO, Dr. Jeffrey M. Dayno, emphasized the vital need for treatments for the 80,000 U.S. families affected by Fragile X syndrome and expressed optimism about the potential impact of ZYN002.
Neurobehavioral Franchise Highlights
The upcoming Phase 3 trial for ZYN002 is expected to confirm earlier findings regarding its effectiveness for patients with Fragile X syndrome. The innovative formulation of ZYN002, delivered transdermally, presents a patient-friendly alternative that addresses compliance issues commonly faced by this demographic. With a solid safety profile established over eight years, the drug aims to fulfill an unmet need for effective treatments in this area.
WAKIX Continues to Gain Market Traction
WAKIX recorded impressive net revenue of $200.5 million in Q2 2025, with projections for total revenue between $820 million and $860 million for the year. The increase in average patient numbers reflects effective commercial strategies, particularly in the expansive narcolepsy market, which includes around 80,000 diagnosed patients in the U.S. Harmony’s ongoing commitment to research and development, including upcoming Phase 3 trials for higher-dose formulations, underscores its focus on enhancing treatment efficacy.
Introducing the Orexin-2 Receptor Agonist
Harmony is set to initiate its first-in-human study for BP1.15205, a novel orexin-2 receptor agonist, in the second half of 2025. This potential best-in-class treatment aims to offer a new avenue for addressing sleep disorders and is backed by compelling preclinical safety and efficacy data. With the anticipated commencement of clinical trials, the company seeks to enhance its therapeutic offerings significantly.
Expansion in Rare Epilepsy Treatments
The company is also advancing its EPX-100 program for Dravet syndrome and Lennox-Gastaut syndrome, with enrollment for Phase 3 trials currently ongoing. These conditions represent serious challenges in pediatric epilepsy, and Harmony’s commitment to developing effective treatments is evident through its innovative approaches, including utilizing phenotypic screening platforms.
Strategic Partnerships and Collaborations
Harmony has entered a research collaboration with CiRC Biosciences to develop regenerative therapies for neurological disorders. This partnership aligns with Harmony’s strategic goals and aims to create novel treatments for refractory epilepsy and treatment-resistant narcolepsy, potentially paving the way for groundbreaking therapies.
Recent Patent Settlements and Legal Developments
The company recently announced a settlement with Lupin Limited regarding patent infringement related to WAKIX. This agreement allows Lupin to introduce its generic version by January 2030, ensuring Harmony maintains its market exclusivity and intellectual property rights as it continues to defend against other generic competitors.
Financial Performance Overview
For the quarter ending June 30, 2025, Harmony reported a net product revenue of $200.5 million, reflecting a notable increase from $172.8 million in the same quarter of the previous year. This growth highlights the effectiveness of Harmony’s commercial strategies and its ability to penetrate the narcolepsy treatment market effectively.
Looking Ahead: Guidance and Projections
Harmony reaffirms its guidance for full-year 2025 net product revenue, projecting between $820 million and $860 million. The company is hosting a conference call to discuss its financial results and future plans, providing investors and stakeholders with insights into its ongoing strategies and market positioning.
Understanding Non-GAAP Financial Measures
In addition to standard GAAP measures, Harmony presents Non-GAAP financial metrics, such as adjusted net income, to provide a clearer picture of its operational performance. These measures, while not substitutes for GAAP, offer valuable insights into the company’s financial health and growth trajectory.
About WAKIX® (pitolisant)
WAKIX is an FDA-approved medication for treating excessive daytime sleepiness and cataplexy in narcolepsy patients. With its unique mechanism of action as a selective H₃ receptor antagonist, it enhances wakefulness by promoting histamine release. This innovative drug has received orphan drug and breakthrough therapy designations, emphasizing its importance in addressing the symptoms of narcolepsy.
Understanding Narcolepsy and Related Disorders
Narcolepsy is a chronic neurological disorder characterized by excessive daytime sleepiness and sudden bouts of sleep, impacting around 170,000 individuals in the U.S. Idiopathic hypersomnia, another debilitating condition, presents similar challenges, with patients experiencing unrelenting sleepiness despite adequate rest. Both conditions require effective management strategies to improve patients’ quality of life.
About ZYN002 and Fragile X Syndrome
ZYN002 is an innovative synthetic cannabidiol product designed for transdermal delivery, offering a potential treatment for Fragile X syndrome, a genetic disorder that causes intellectual disabilities and behavioral challenges. With no approved therapies currently available, ZYN002 addresses a critical need in the patient community.
About Harmony Biosciences
Founded in 2017, Harmony Biosciences is dedicated to developing therapies for rare neurological diseases, focusing on unmet medical needs. The company strives to create innovative solutions that empower patients through advanced scientific research and compassionate care.
Forward-Looking Statements
This announcement includes forward-looking statements concerning Harmony’s financial outlook, product development plans, and market strategies. These statements are subject to various risks and uncertainties that could affect actual outcomes, emphasizing the company’s commitment to transparency and stakeholder communication.
