Harmony Biosciences FY Conference Summary Company Overview - **Company**: Harmony Biosciences (NasdaqGM:HRMY) - **Key Product**: WAKIX, a treatment for narcolepsy Industry Insights - **Market Size**: Approximately 80,000 diagnosed narcolepsy patients, with an additional 80,000 to 90,000 undiagnosed patients, indicating significant growth potential in the narcolepsy treatment market [10][10] - **Polypharmacy**: The narcolepsy market is characterized by high polypharmacy, with patients often on multiple therapies [8][8] Key Product Performance - **WAKIX Growth**: - Achieved a record increase of 500 patients in Q3, the highest since launch, following a steady growth pattern of adding 100 to 400 patients per quarter over the last six years [5][5] - Currently, 8,100 patients are on WAKIX therapy, indicating substantial room for growth in a market of 80,000 diagnosed patients [10][10] Competitive Landscape - **Oxybate Generics**: Anticipation of multiple Xyrem generics entering the market next year, which may lower costs for oxybate therapy but is expected to have limited impact on WAKIX due to its unique non-scheduled status [22][22] - **Orexin Agonists**: - Takeda's orexin agonist is expected to launch in late 2026, but the impact on WAKIX is anticipated to be small initially, with a gradual effect over time [28][28] - WAKIX is perceived as well-tolerated and effective, maintaining a strong position among healthcare providers [27][27] Future Developments - **Orexin Agonist Program**: Harmony has initiated a phase one PK/PD program for its own orexin agonist, with plans to accelerate to phase two and three based on initial data [30][30] - **Next-Gen Pitolisant**: - A gastro-resistant formulation of Pitolisant (Pitolisant GR) is on track for NDA submission in early 2027, aiming to improve patient adherence and outcomes [41][41] - A high-dose formulation (Pitolisant HD) is also in development, with phase three trials for narcolepsy and idiopathic hypersomnia set to begin [44][44] Clinical Insights - **Fatigue Management**: Fatigue, a common issue in narcolepsy, will be a secondary outcome in clinical trials for both narcolepsy and idiopathic hypersomnia, aiming to differentiate the product further [45][45] - **Clemizole for Pediatric Epilepsies**: Harmony is in phase three trials for clemizole hydrochloride in Dravet and Lennox-Gastaut syndromes, with top-line data expected in late 2026 [50][50] Conclusion - Harmony Biosciences is positioned for growth in the narcolepsy market with WAKIX, while also exploring new opportunities with orexin agonists and next-generation formulations of Pitolisant. The company is actively addressing unmet medical needs in both narcolepsy and pediatric epilepsy markets.

Core Insights - Harmony Biosciences has initiated a Phase 1 clinical trial for BP1.15205, a potential best-in-class orexin 2 receptor agonist aimed at treating central disorders of hypersomnolence, including narcolepsy and idiopathic hypersomnia [1][2] - The trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BP1.15205, with topline data expected in 2026 [1][2] - The drug has shown high potency and selectivity in preclinical studies, with a favorable safety profile and potential for once-daily dosing [1][2] Company Overview - Harmony Biosciences is focused on developing innovative therapies for rare neurological diseases, emphasizing a commitment to patients with unmet medical needs [6] - The company was established in 2017 and is headquartered in Plymouth Meeting, Pennsylvania [6] Product Development - BP1.15205 is part of Harmony's orexin program, which aims to expand its sleep-wake franchise and provide additional treatment options for patients with narcolepsy and hypersomnolence disorders [3] - The Phase 1 trial is being conducted by Bioprojet Pharma in the European Union, with Harmony holding an exclusive licensing agreement for development and commercialization in the U.S. and Latin America [3] Market Context - Narcolepsy affects approximately 170,000 Americans, characterized by excessive daytime sleepiness and cataplexy, with a significant impact on daily functioning [4] - Idiopathic hypersomnia is another debilitating disorder marked by excessive daytime sleepiness despite adequate sleep, affecting patients' cognitive functions [5]

Q3 2025 Financial Results & Business Update November 4 , 2025 Copyright © 2024 Harmony Biosciences. All rights reserved. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2024 net product revenue, expectations for ...

Core Insights - Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) is recognized as a promising growth stock by analysts, with a Buy rating reiterated by Truist Financial on October 24 [1] - The company reported strong Q3 2025 performance for its WAKIX product, generating approximately $239 million in revenue, marking a 29% year-over-year growth [2] - The increase in revenue was attributed to a record rise in the average number of patients, which grew by 500 to reach 8,100 during the quarter [2] - Harmony has also raised its revenue guidance for 2025 to a range of $845 million to $865 million, up from the previous range of $820 million to $860 million [3] - The company focuses on developing and commercializing therapies for rare neurological diseases and other unmet medical needs [3]

Core Insights - Harmony Biosciences announced strong preliminary results for Q3 2025, driven by the performance of WAKIX, which shows significant market potential [1][6] - The company raised its 2025 revenue guidance to a range of $845 million to $865 million, up from the previous range of $820 million to $860 million [6] Financial Performance - WAKIX generated approximately $239 million in net revenue for Q3 2025, reflecting a 29% year-over-year growth [6] - The average number of patients using WAKIX increased by approximately 500, reaching 8,100 patients in Q3 2025, indicating a strong trajectory towards potential blockbuster status [6] Upcoming Events - Harmony will report full Q3 2025 financial results on November 4, 2025, and will host a conference call at 8:30 a.m. ET on the same day [2][3]

Core Insights - Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) is identified as a promising low-priced growth stock, with analysts at H.C. Wainwright maintaining a 'Buy' rating but lowering the price target from $70 to $55, indicating a potential upside of nearly 100% [1] - The company faced a setback with its Phase 3 RECONNECT trial for ZYN002, which did not meet its primary endpoint for treating Fragile X syndrome, attributed to a higher-than-expected placebo response [2] - Despite halting the development of ZYN002 for 22q11.2 deletion syndrome, Harmony continues to demonstrate commercial strength through its product WAKIX and maintains a solid balance sheet, supporting ongoing R&D and business development efforts [3] Company Overview - Harmony Biosciences Holdings, Inc. is a Pennsylvania-based pharmaceutical company focused on therapies for rare and neurological diseases, founded in 2017 with an emphasis on empathy and innovation [4]

Group 1 - Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) is considered one of the best affordable biotech stocks to invest in, with a Buy rating maintained by UBS analyst Ashwani Verma and a price target set at $50.00 [1] - The company reported fiscal Q2 2025 results with net revenue of $200.5 million for the quarter, reflecting a 16% year-over-year growth [2] - Harmony Biosciences is initiating next-generation Pitolisant HD Phase 3 registration trials for Narcolepsy and Idiopathic Hypersomnia, targeting PDUFA in 2028 [3] Group 2 - The product WAKIX enhances histamine signaling in the brain by binding to H3 receptors and is part of a strong pipeline aimed at treating rare neurological diseases [4]

Financial Performance - Harmony Biosciences reported Q2 2025 net product revenue of $200.5 million, a 16% year-over-year increase compared to $172.8 million in Q2 2024 [8, 36] - The company's non-GAAP adjusted net income for Q2 2025 was $53.8 million, a 120% increase from $24.5 million in Q2 2024 [36] - Harmony Biosciences is confirming its full-year 2025 revenue guidance of $820 million to $860 million [8] - Cash, cash equivalents, and investments totaled $672.3 million as of Q2 2025, compared to $434.1 million in Q2 2024, a 55% increase [6, 38] WAKIX Performance - WAKIX is approaching blockbuster status, representing a $1 billion opportunity in narcolepsy alone [9] - The average patient count for WAKIX grew by approximately 400 to approximately 7,600 patients [8] - WAKIX has strong payer coverage of >80% of lives [8] Pipeline Development - Harmony Biosciences plans to initiate Phase 3 registrational trials for Pitolisant HD in narcolepsy and idiopathic hypersomnia (IH) in Q4 2025 [17, 29] - Topline data from the Phase 3 RECONNECT study of ZYN002 in Fragile X Syndrome (FXS) is expected in Q3 2025 [17, 18] - EPX-100 Phase 3 registrational trials are ongoing in Dravet syndrome (ARGUS study) & Lennox-Gastaut syndrome (LIGHTHOUSE study), with topline data anticipated in 2026 [17, 33]

Q1 2025 Financial Results & Business Update May 6, 2025 Copyright © 2025 Harmony Biosciences. All rights reserved. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2024 net product revenue, expectations for the gr ...

Financial Data and Key Metrics Changes - The company reported net product revenues of $714.7 million for 2024, representing a 23% year-over-year growth. In Q4 alone, net revenue was $201.3 million, reflecting a 20% increase from $168.4 million in the prior year quarter [11][25][55]. - Non-GAAP adjusted net income for Q4 2024 was $63 million, or $1.08 per diluted share, compared to $42.8 million or $0.73 per diluted share in the prior year quarter [56]. - The company ended Q4 with approximately $576 million in cash and cash equivalents, reflecting robust cash generation of approximately $219.8 million from operations in 2024 [55][57]. Business Line Data and Key Metrics Changes - The average number of patients on WAKIX increased to approximately 7,100 in Q4, with a sequential increase of about 300 patients from the previous quarter [25][26]. - The growth in WAKIX prescriptions was primarily driven by adult narcolepsy patients, constituting approximately 95% of the diagnosed narcolepsy opportunity [27]. Market Data and Key Metrics Changes - The company anticipates continued growth in WAKIX, with net revenue guidance for 2025 set at $820 million to $860 million, reflecting strong expectations for the year [58]. - The company is on track to achieve a $1 billion plus opportunity in narcolepsy alone, with a robust commercial infrastructure supporting this growth [30][31]. Company Strategy and Development Direction - The company aims to become a leading CNS company by advancing its late-stage pipeline to deliver one or more new product or indication launches each year [9]. - The strategic focus includes the development of next-generation formulations of pitolisant, such as pitolisant high-dose (HD) and pitolisant gas resistant (GR), to enhance patient care and extend revenue growth into the mid-2040s [31][40]. - The company is actively engaged in identifying additional opportunities to expand its leadership in Sleep/Wake, neurobehavioral disorders, and rare epilepsies [22]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth strategy, emphasizing that 2025 is shaping up to be a transformational year with key clinical milestones expected [17][62]. - The company remains committed to addressing unmet medical needs in idiopathic hypersomnia and narcolepsy, despite recent challenges with the supplemental new drug application for pitolisant in idiopathic hypersomnia [15][16]. Other Important Information - The company announced its first generic settlement agreement with Novagen Pharma, reinforcing the strength of its intellectual property portfolio [12][100]. - The company is on track to initiate pivotal Phase 3 registration trials for pitolisant HD in both narcolepsy and idiopathic hypersomnia in Q4 2025, with anticipated PDUFA dates in 2028 [40][41]. Q&A Session Summary Question: Can you provide updates on ZYN002 and the Phase 3 study? - Management expressed satisfaction with the ongoing trial and noted that they are enrolling patients aged three to 30 years, with a good proportion rolling over into the long-term extension study [66][71]. Question: Why should investors feel confident about Pitolisant HD's success in IH patients? - Management highlighted the evidence of efficacy from previous studies and the optimized formulation of pitolisant HD, which is expected to deliver larger efficacy for excessive daytime sleepiness and target symptoms like sleep inertia [76][79]. Question: How confident is the company in the timeline for Pitolisant HD trials given competition? - Management indicated confidence in the recruitment process due to established relationships with clinical trial sites and the robust signal shown in previous studies [84][88]. Question: What is the status of remaining patent challenges? - Management confirmed ongoing litigation processes and mentioned a Markman hearing scheduled for March, with a trial set for 2026 [96][99]. Question: What would success look like for the ZYN002 trial? - Success would be defined by demonstrating a statistically significant and clinically meaningful outcome on the primary endpoint, with supportive data from the previous CONNECT study [104][109].