Financial Data and Key Metrics Changes - Harmony Biosciences reported net product revenue 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% [36][34] - The company ended Q1 2025 with over $600 million in cash and cash equivalents, positioning it well for future growth and pipeline development [34][36] Business Line Data and Key Metrics Changes - The core commercial business, particularly Wakix for narcolepsy, continues to show strong growth, achieving a 20% year-over-year increase in net revenue [20][21] - The company is on track to reach over $1 billion in revenue from narcolepsy alone, with a confirmed net revenue guidance for 2025 in the range of $820 million to $860 million [22][36] - The pipeline includes eight assets across 13 development programs, with plans to have up to six programs in Phase III trials by the end of 2025 [10][24] Market Data and Key Metrics Changes - Harmony's market penetration for Wakix has reached over 50% among approximately 5,000 healthcare providers, with continued growth in prescribers beyond those enrolled in the oxybate REMS program [21][22] - The company is actively monitoring the competitive landscape, particularly with the introduction of generic alternatives to oxybate, but has not seen significant changes in payer coverage for Wakix [56][57] Company Strategy and Development Direction - Harmony aims to leverage its strong balance sheet to expand its late-stage pipeline, focusing on rare neurological diseases with high unmet medical needs [12][13] - The company is preparing for significant clinical milestones, including the Phase III trial of ZYN-two for Fragile X syndrome, with top-line data expected in Q3 2025 [8][30] - Harmony's operational foundation is U.S.-based, which provides a unique advantage in navigating potential geopolitical risks and supply chain challenges [15][13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to deliver continued growth, driven by a robust pipeline and strong commercial performance [16][38] - The company is optimistic about the potential for new product launches and indications over the next several years, with peak sales potential exceeding $3 billion [16][38] - Management highlighted the importance of patient-centric drug development and the ongoing commitment to addressing unmet medical needs in neurology [23][24] Other Important Information - The company has made significant progress in its clinical development programs, with a focus on addressing symptoms such as fatigue in narcolepsy, which currently lacks approved treatments [25][68] - Harmony's unique commercial model and differentiated product profile for Wakix continue to drive its success in the narcolepsy market [10][21] Q&A Session Summary Question: What are the powering assumptions for the ZYN-two study and risk mitigation strategies? - The ZYN-two study is powered more than 90% for the primary endpoint, with rigorous criteria for patient selection and extensive training for caregivers to ensure accurate assessments [40][42] Question: What changes or tweaks might be implemented in the commercial strategies for Wakix? - The new Chief Commercial Officer expressed excitement about the existing strategies and emphasized a focus on maintaining strong performance while preparing for future opportunities [41][48] Question: How does Harmony view the competitive landscape for orexin receptor agonists? - Harmony sees the market as still emerging, with a belief that narcolepsy will remain a polypharmacy market, necessitating multiple treatment options for patients [50][68] Question: What are the drivers of revenue guidance for 2025? - The primary driver of growth is the continued sequential addition of patients on therapy, with a strong and stable progression of average patients on Wakix [73][75] Question: How quickly could Harmony file an NDA for ZYN-two if data is positive? - If the results are positive, Harmony aims to file the NDA as soon as possible, anticipating a priority review due to the significant unmet need in the Fragile X patient population [86][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]