Financial Performance - WAKIX® (pitolisant) net revenue for Q1 2025 was $184.7 million, representing a 20% growth year-over-year[1] - GAAP net income for Q1 2025 was $45.6 million, or $0.78 earnings per diluted share, compared to $38.3 million, or $0.67 earnings per diluted share for Q1 2024[12] - Non-GAAP adjusted net income for Q1 2025 was $60.4 million, or $1.03 earnings per diluted share, compared to $50.7 million, or $0.88 earnings per diluted share for Q1 2024[12] - Net product revenue for Q1 2025 was $184.733 million, a 19.5% increase from $154.615 million in Q1 2024[44] - Gross profit for Q1 2025 was $152.739 million, compared to $127.131 million in Q1 2024, reflecting a gross margin improvement[44] - Operating income for Q1 2025 was $56.245 million, up from $52.033 million in Q1 2024, showing operational efficiency[44] - Net income for Q1 2025 was $45.560 million, compared to $38.334 million in Q1 2024, representing an increase of 18.5%[44] - GAAP reported net income per diluted share rose to $0.78 in Q1 2025 from $0.67 in Q1 2024, marking a 16% increase[46] - Non-GAAP adjusted net income per diluted share increased to $1.03 in Q1 2025, up from $0.88 in Q1 2024, a growth of 17%[46] Expenses and Investments - Research and Development expenses increased by 56% to $34.5 million in Q1 2025 compared to $22.2 million in Q1 2024[15] - Total operating expenses were $96.5 million in Q1 2025, representing a 29% increase from $75.1 million in Q1 2024[15] - Research and development expenses increased to $34.540 million in Q1 2025 from $22.189 million in Q1 2024, indicating a focus on product development[44] - Stock-based compensation expense increased to $12,450,000 in Q1 2025 from $10,434,000 in Q1 2024, an increase of 19%[46] Patient and Product Development - The average number of patients on WAKIX increased to approximately 7,200 for Q1 2025, with approximately 7,300 patients at the end of the quarter[7] - Recruitment for the Phase 3 registrational trial of ZYN002 in Fragile X syndrome is complete, with topline data expected in Q3 2025[11] - The company is on track to initiate Phase 3 registrational trials in narcolepsy and idiopathic hypersomnia (IH) in Q4 2025[7] - The company plans to submit a supplemental New Drug Application (sNDA) for pitolisant in idiopathic hypersomnia, expanding its product indications[42] - Harmony Biosciences is focused on developing therapies for rare neurological diseases, addressing significant unmet medical needs[41] - The company has received FDA Fast Track designation for ZYN002 for the treatment of behavioral symptoms in patients with Fragile X Syndrome[34] Financial Guidance and Assets - 2025 net revenue guidance is projected between $820 million and $860 million[14] - Total assets as of March 31, 2025, were $1.055 billion, up from $999.200 million at the end of 2024[45] - Cash and cash equivalents increased to $488.998 million from $453.001 million, indicating improved liquidity[45] Other Financial Metrics - Non-cash interest expense for Q1 2025 was $166,000, slightly down from $180,000 in Q1 2024[46] - Depreciation expense decreased significantly to $7,000 in Q1 2025 from $163,000 in Q1 2024[46] - The income tax effect related to non-GAAP adjustments improved to $(3,776,000) in Q1 2025 from $(4,350,000) in Q1 2024[46] - Amortization expenses remained consistent at $5,961,000 for both Q1 2025 and Q1 2024[46] - The weighted average number of shares of common stock used in non-GAAP diluted per share calculations was 58,524,566 in Q1 2025, compared to 57,597,627 in Q1 2024[46]

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 ...

Core Insights - Harmony Biosciences Holdings, Inc. has a strong track record of exceeding earnings estimates, particularly in the last two quarters with an average surprise of 19.15% [1][5] - The company reported earnings of $0.85 per share in the last quarter, surpassing the Zacks Consensus Estimate of $0.74 per share by 14.86% [2] - The previous quarter also showed a positive surprise, with actual earnings of $0.79 per share against an expected $0.64 per share, resulting in a 23.44% surprise [2] Earnings Estimates and Predictions - Recent changes in earnings estimates for Harmony Biosciences have been favorable, with a positive Earnings ESP of +33.09%, indicating increased analyst optimism about its near-term earnings potential [5][8] - The combination of a positive Earnings ESP and a Zacks Rank of 3 (Hold) suggests a high likelihood of another earnings beat, with historical data showing that such combinations lead to positive surprises nearly 70% of the time [6][8] - The next earnings report for Harmony Biosciences is anticipated to be released on May 6, 2025 [8]

Core Viewpoint - Jazz Pharmaceuticals and Harmony Biosciences are significant players in the neuroscience sector, particularly in sleep disorders, with Jazz having a more diversified portfolio and stronger financial performance compared to Harmony [1][2][22]. Group 1: Company Overview - Jazz Pharmaceuticals markets several neuroscience drugs, including the oral sleep disorder drug Xywav and the CBD drug Epidiolex, while Harmony Biosciences has one marketed drug, Wakix, for narcolepsy [1][10]. - Jazz derives over two-thirds of its total revenues from its neuroscience franchise, while Harmony generates all its revenue from Wakix sales [3][11]. Group 2: Financial Performance - Jazz's total revenues for 2024 rose 6% year over year to $4.1 billion, with $2.9 billion from neuroscience and $1.1 billion from oncology drugs [9]. - Harmony's 2024 sales increased 23% year over year to $715 million, with expectations for 2025 sales between $820 million and $860 million, indicating an 18% year-over-year growth [11]. Group 3: Product Pipeline and Development - Jazz's Xywav sales rose 16% year over year to $1.5 billion in 2024, and Epidiolex sales increased 15% year over year to $972 million [4][5]. - Harmony is developing next-generation formulations of Wakix and has acquired Zynerba Pharmaceuticals to explore the cannabis space [12][13]. Group 4: Market Position and Valuation - Jazz has a diversified product lineup, including five oncology drugs, which accounted for 29% of its 2024 product revenues [6]. - Harmony's shares trade at a higher price/earnings (P/E) ratio of 8.63 compared to Jazz's 5.07, indicating that Harmony is more expensive [19]. Group 5: Investment Outlook - Jazz is viewed as a better investment option due to its diversified portfolio, rising EPS estimates, and robust cash reserves, while Harmony's reliance on a single product presents a concentration risk [22][23]. - Jazz holds a Zacks Rank 2 (Buy), while Harmony has a Zacks Rank 3 (Hold), reinforcing Jazz's favorable investment standing [24].

Small biotech companies can sometimes offer great benefits, with one or more potentially disruptive drugs in their pipeline. However, most of the times I am not interested because this is more than offset by a multitude"Fundamental Options" would be the title of my investing style, because I combine fundamental analysis with the power of options. I use Fundamental Analysis to quantitatively and qualitatively assess individual stocks and ETFs, and I pursue various strategies: Income oriented, especially BDCs ...

Core Viewpoint - Harmony Biosciences (HRMY) has faced challenges, including a 10.8% decline in stock price over the past six months, primarily due to a Refusal to File (RTF) letter from the FDA regarding pitolisant for excessive daytime sleepiness in idiopathic hypersomnia [1][20] Group 1: Company Performance - The stock has underperformed compared to the industry, which declined by 9.7%, and the S&P 500 index during the same period [1] - Despite the setback, the fourth-quarter results reported on February 25 were better than expected, leading to a slight recovery in shares [3] - The lead drug, Wakix, is a first-in-class therapy that has driven growth since its initial FDA approval in 2019, with expected net revenues between $820 million and $860 million for 2025 [7][8] Group 2: Drug Development and Pipeline - The company aimed to expand the label of pitolisant for excessive daytime sleepiness in adult patients with idiopathic hypersomnia, but the submission faced regulatory challenges [4][5] - Two next-generation formulations, pitolisant Gastro-Resistant and pitolisant High-Dose, are in development, with phase III studies planned for late 2025 [9][10] - The acquisition of Zynerba Pharmaceuticals added ZYN-002 to the portfolio, currently in a phase III trial for Fragile X Syndrome, with data expected in Q3 2025 [11][12] Group 3: Financial Position and Valuation - As of December 31, 2024, the company had cash, cash equivalents, and investments totaling $576.1 million, with long-term debt of $163 million, indicating a sound cash position [14] - HRMY's shares trade at a price/sales ratio of 2.27x forward sales, lower than its historical mean of 4.23x but higher than the industry average of 1.70x [15] - The estimate for 2025 earnings per share has decreased to $2.89 from $3.16 over the past 30 days, reflecting a downward trend in earnings expectations [17] Group 4: Market Outlook - The narcolepsy market in the U.S. had an approximate net sales value of $2.9 billion in 2023, with expectations for continued growth [19] - The company believes its pipeline has the potential to deliver more than $3 billion in net revenues in the coming years, driven by successful development of its candidates [19] - The company is advised to maintain investment in HRMY, with potential for value increase from favorable data readouts later in the year [20]

Core Viewpoint - Harmony Biosciences Holdings, Inc. is under investigation for potential securities fraud and unlawful business practices following a negative FDA response regarding its drug pitolisant [1][2]. Group 1: Company Overview - Harmony Biosciences Holdings, Inc. trades on NASDAQ under the ticker HRMY [1]. - The company recently received a Refusal to File (RTF) letter from the FDA concerning pitolisant, which is intended for treating excessive daytime sleepiness in adult patients with idiopathic hypersomnia [2]. Group 2: Financial Impact - Following the FDA's announcement, Harmony's stock price dropped by $3.77 per share, representing a decline of 9.64%, closing at $35.32 per share on February 19, 2025 [2]. Group 3: Legal Context - Pomerantz LLP is leading the investigation into Harmony, focusing on whether the company and its officers engaged in securities fraud or other unlawful practices [1]. - Pomerantz LLP has a long history in corporate and securities class litigation, having recovered significant damages for victims of securities fraud [3].

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, which constitutes approximately 95% of the diagnosed narcolepsy opportunity [27] - The company anticipates net revenue guidance for WAKIX in 2025 to be between $820 million and $860 million [30][58] Market Data and Key Metrics Changes - The company continues to see strong double-digit growth in net revenue for WAKIX, demonstrating sustained demand in the narcolepsy market [25][26] - 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] Company Strategy and Development Direction - The company aims to become a leading CNS company focused on developing innovative treatments for patients with unmet medical needs, with a pipeline poised to deliver over $3 billion in net revenue [9][10] - The company is committed to defending its intellectual property and has announced its first generic settlement agreement with Novagen Pharma, reinforcing the strength of its IP portfolio [12][100] - 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 growth trajectory of WAKIX, projecting it to be a $1 billion plus opportunity in narcolepsy alone [11][12] - The company views 2025 as a transformational year, with key clinical milestones expected to reinforce its leadership in Sleep/Wake and rare CNS disorders [17][23] - Management acknowledged challenges with the supplemental new drug application for pitolisant in idiopathic hypersomnia but emphasized their commitment to the patient community [15][16] Other Important Information - The company has a robust late-stage pipeline with four ongoing Phase 3 registrational trials and plans to initiate six by the end of the year [36] - The company is advancing its next-generation pitolisant formulations, including pitolisant HD and pitolisant GR, designed to address unmet needs in narcolepsy and idiopathic hypersomnia [41][42] 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 on track for top-line data in Q3 2025. They are enrolling patients aged three to 30 years and have seen a good rollover rate into the long-term extension study [66][71] Question: What is the rationale for Pitolisant HD's likelihood of 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 enhanced therapeutic benefits [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 from 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]

Q4 & FY 2024 Financial Results & Business Update February 25, 2025 Copyright © 2025 Harmony Biosciences. All rights reserved. Forward-Looking Statements This presentation includes forward‐looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in these materials or elsewhere, including statements regarding Harmony Biosciences Holdings, Inc.'s (the "Company") future financial position, preliminary financ ...

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].