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.

Financial Data and Key Metrics Changes - Harmony Biosciences reported net revenue of $239.5 million for Q3 2025, representing a 29% year-over-year growth [5][20] - The company raised its full-year net revenue guidance from $820-$860 million to $845-$865 million [5][22] - Cash generation for the quarter was robust at $106 million, bringing total cash and cash equivalents to $778 million as of September 30, 2025 [5][21] Business Line Data and Key Metrics Changes - Wakix achieved net sales of $239.5 million in Q3 2025, with an average increase of 500 patients added, resulting in approximately 8,100 average patients by the end of Q3 [9][20] - The performance of Wakix is attributed to its unique position as the only non-scheduled treatment option, leading to high brand awareness and broad payer coverage [9][10] Market Data and Key Metrics Changes - The company is rapidly approaching blockbuster status for Wakix in narcolepsy, with expectations of exceeding $1 billion in annual revenue from this indication alone [6][11] - The market for narcolepsy treatments is expected to expand with the introduction of new therapies, but Harmony remains confident in Wakix's continued growth [45][46] Company Strategy and Development Direction - Harmony aims to become a leading patient-focused CNS company, focusing on innovative treatments for unmet medical needs [7][8] - The company has a robust late-stage pipeline with multiple catalysts expected in the coming years, including the initiation of two phase 3 trials for Pitolisant HD [7][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's growth trajectory, driven by strong fundamentals and execution across the organization [24][91] - The management team highlighted the importance of maintaining a profitable, self-funding biotech profile while pursuing strategic opportunities to enhance the product portfolio [8][22] Other Important Information - The IND for Pitolisant HD has been submitted to the FDA, with plans to initiate phase 3 trials for both narcolepsy and idiopathic hypersomnia by the end of 2025 [7][14] - The company is actively evaluating business development opportunities to expand its pipeline and product offerings [43][44] Q&A Session Summary Question: Can you elaborate on the trajectory of Wakix evolving into 2026? - Management noted that the strong performance is driven by patient adds, with a record increase of 500 patients in Q3, and they expect this momentum to continue into Q4 and 2026 [27][30] Question: What were the GI AEs seen with the GR formulation? - No new safety or tolerability issues were observed with the Pitolisant GR formulation, and all patients initiated treatment at the therapeutic dose without issues [34][35] Question: Thoughts on BizDev and M&A after the Zygel failure? - The company remains focused on strategic business development, actively evaluating innovative assets to enhance its pipeline [42][43] Question: How is new patient growth coming for Wakix? - Growth is attributed to both activating new prescribers and deeper penetration within existing writers, supported by strong marketing and promotional efforts [90]

Financial Data and Key Metrics Changes - The company reported net revenues of $184.7 million for Q1 2025, representing a 20% increase year-over-year from $154.6 million in Q1 2024 [35][36] - Non-GAAP adjusted net income for Q1 2025 was $60.4 million, or $1.03 per diluted share, compared to $50.7 million, or $0.88 per diluted share in the prior year quarter, reflecting a growth of 19% [37][38] - The company ended Q1 2025 with over $610 million in cash, cash equivalents, and investments on the balance sheet [39] Business Line Data and Key Metrics Changes - Net product revenue for Wakix in Q1 2025 was $184.7 million, a 20% increase year-over-year, indicating strong demand in the narcolepsy market [7][19] - The company is confident in reaching over $1 billion in revenue from narcolepsy alone, with guidance for total net revenue in 2025 set between $820 million and $860 million [21][39] Market Data and Key Metrics Changes - The company has achieved over 50% penetration within approximately 5,000 healthcare providers (HCPs) as of the end of Q1 2025, with continued growth in prescribers beyond those enrolled in the oxybate REMS program [20][21] - The narcolepsy market is estimated to have around 80,000 diagnosed patients, with the company tapping into this potential through its differentiated product profile [21][80] Company Strategy and Development Direction - The company aims to leverage its strong balance sheet to build a robust late-stage pipeline, with plans to have up to six programs in Phase III trials by the end of 2025 [9][12] - The focus is on developing next-generation formulations of pitolisant, targeting unmet patient needs and extending the growth of the pitolisant franchise into the mid-2040s [21][22] - The company is also exploring opportunities in adjacent rare CNS assets to further strengthen its pipeline [12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to navigate the shifting geopolitical landscape and potential impacts from new tariffs, emphasizing operational independence [12][14] - The company is optimistic about its late-stage pipeline, with expectations of delivering one or more new product or indication launches each year over the next several years, with peak sales potential exceeding $3 billion [15][40] Other Important Information - The company has a unique operational foundation in the U.S., with exclusive licensing rights and intellectual property associated with its products domiciled in the U.S. [14] - The company is actively working on a secondary manufacturing site for Wakix in the U.S. to enhance supply chain resilience [12] Q&A Session Summary Question: What are the powering assumptions for the ZYN-two study and risk mitigation strategies? - The study is powered more than 90% for the primary endpoint, with rigorous criteria and checks in place to ensure the right patients are included [42][45][46] Question: What are the initial impressions of the commercial team and strategies supporting Wakix? - The new Chief Commercial Officer expressed excitement about the team's focus on patient needs and the strong performance of Wakix, with plans to evaluate and implement potential improvements [43][49][51] Question: How does the company view the competitive landscape for orexin receptor agonists? - The company sees the market as still emerging, with a focus on the high potency of its BP1.5205 compound and the potential for polypharmacy in treating narcolepsy [53][57][70] Question: What are the drivers of revenue guidance for 2025? - Continued sequential additions of patients on therapy and strong underlying business fundamentals are expected to drive revenue growth [75][78] Question: What is the timeline for filing the NDA for ZYN-two if positive data is obtained? - The company aims to file the NDA as soon as possible following positive data, with expectations for priority review due to the significant unmet need [87][91]