Oct 23, 2025 8:05 AM Eastern Daylight Time Harmony Biosciences Preannounces Strong Q3 2025 WAKIX Performance; Raises 2025 Revenue Guidance Share PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced preliminary results for Q3 2025, highlighted by the following: "Our robust performance this quarter highlights the continued strength of WAKIX and significant market opportunity that remains ahead. I am incredibly proud of our team's focus and executional excel ...

Core Insights - The health care sector is experiencing a trend of oversold stocks, presenting potential buying opportunities for undervalued companies [1][2] Group 1: Oversold Stocks - MoonLake Immunotherapeutics (NASDAQ: MLTX) has an RSI of 21.5, with a stock price drop of approximately 86% over the past month, reaching a 52-week low of $5.95. The stock saw a 13% increase to close at $8.43 [3][7] - CorVel Corp (NASDAQ: CRVL) has an RSI of 27.2, with a stock price decline of around 14% over the past month, hitting a 52-week low of $72.15. The stock rose 0.6% to close at $73.83 [4][7] - Harmony Biosciences Holdings Inc (NASDAQ: HRMY) has an RSI of 28.9, with a stock price decrease of about 24% over the past month, reaching a 52-week low of $26.26. The stock gained 2% to close at $27.28 [4][7] Group 2: Company Updates - MoonLake Immunotherapeutics reported week-16 results from its Phase 3 VELA-1 and VELA-2 trials, using HiSCR75 as the primary endpoint [7] - CorVel's CFO Brandon T. O'Brien stepped down, impacting the company's stock performance [7] - Harmony Biosciences Holdings announced topline results from its Phase 3 RECONNECT Study for ZYN002 in Fragile X syndrome, highlighting a catalyst-rich pipeline and plans for future trials [7]

Core Insights - The health care sector is experiencing a trend of oversold stocks, presenting potential buying opportunities for undervalued companies [1][2] Group 1: Oversold Stocks - MoonLake Immunotherapeutics (NASDAQ: MLTX) has an RSI of 21.5, with a stock price drop of approximately 86% over the past month, reaching a 52-week low of $5.95. Recent price action saw a 13% increase, closing at $8.43 [3][7] - CorVel Corp (NASDAQ: CRVL) has an RSI of 27.2, with a stock price decline of around 14% over the past month, hitting a 52-week low of $72.15. The stock rose 0.6% to close at $73.83 [4][7] - Harmony Biosciences Holdings Inc (NASDAQ: HRMY) has an RSI of 28.9, with a stock price decrease of about 24% over the past month, reaching a 52-week low of $26.26. The stock gained 2% to close at $27.28 [4][7] Group 2: Company Developments - MoonLake Immunotherapeutics reported week-16 results from its Phase 3 VELA-1 and VELA-2 trials, using HiSCR75 as the primary endpoint [7] - CorVel's CFO Brandon T. O'Brien stepped down, impacting the company's stock performance [7] - Harmony Biosciences released topline results from its Phase 3 RECONNECT Study for ZYN002 in Fragile X syndrome, highlighting a catalyst-rich pipeline and plans for future trials [7]

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]

Core Insights - Harmony Biosciences Holdings, Inc. (HRMY) shares fell by 16.56% on September 24 following the announcement of the failure of the late-stage RECONNECT study for ZYN002 in Fragile X syndrome (FXS) [1][2] - The RECONNECT study was a randomized, double-blind, placebo-controlled trial aimed at assessing the efficacy and safety of ZYN002, a transdermal gel cannabidiol for patients aged 3 to under 30 years with FXS [1][2] - The study did not meet its primary endpoint of improving social avoidance, attributed to a higher-than-expected placebo response rate [2] Company Performance - Year-to-date, HRMY has experienced a 22.2% decline, contrasting with a 5.3% gain in the industry [3] - The company's lead drug, Wakix (pitolisant), generated revenues of $200.5 million in the second quarter [5][7] - HRMY plans to initiate phase III registrational trials for a high dose of pitolisant in narcolepsy and idiopathic hypersomnia in the fourth quarter of 2025 [5][7] Pipeline Expansion - HRMY has broadened its pipeline into rare epilepsy through the acquisition of Epygenix Therapeutics, adding EPX-100 and EPX-200 to its portfolio [6][7] - EPX-100 is currently in phase III registrational trials for Dravet Syndrome and Lennox-Gastaut Syndrome, while EPX-200 is in the pre-IND phase for developmental and epileptic encephalopathies [6]

Group 1 - U.S. stocks experienced a decline, with the Dow Jones index dropping over 150 points on Wednesday [1] - Freeport-McMoRan Inc. shares fell 16.4% to $37.94 after the company provided lower third-quarter sales guidance [1] - The company reported a tragic incident at the Grasberg Block Cave mine, resulting in two fatalities and five team members missing [1] Group 2 - Zhengye Biotechnology Holding Limited shares decreased by 28.2% to $5.02 [4] - YD Bio Ltd shares fell 15% to $17.91 [4] - Harmony Biosciences Holdings, Inc. shares dropped 14.6% to $27.36 after failing to meet primary endpoints in a clinical trial [4] - Needham reduced the price target for Harmony Biosciences from $48 to $41 [4] - Cohu, Inc. shares declined 12.7% to $19.85 following an announcement of convertible senior notes offering [4] - Rigel Pharmaceuticals, Inc. shares decreased by 12.7% to $29.69 [4] - BGM Group Ltd. shares fell 12.2% to $10.31 [4] - Worthington Enterprises, Inc. shares dipped 11.3% to $53.43 after reporting first-quarter results [4] - QMMM Holdings Limited shares decreased by 11% to $97.00 [4] - Astera Labs, Inc. shares fell 10.6% to $205.65 [4] - Sandisk Corporation shares dipped 8.2% to $97.68 [4]

Group 1: Market Overview - U.S. stocks experienced a decline, with the Dow Jones index dropping more than 150 points on Wednesday [1] Group 2: Company-Specific News - Freeport-McMoRan Inc. shares fell 16.4% to $37.94 after the company provided third-quarter sales guidance [1] - The company reported a tragic incident at the Grasberg Block Cave mine, resulting in the fatal injuries of two team members and five others remaining missing [1] - Zhengye Biotechnology Holding Limited shares decreased by 28.2% to $5.02 [4] - YD Bio Ltd shares fell 15% to $17.91 [4] - Harmony Biosciences Holdings, Inc. shares dropped 14.6% to $27.36 after its Phase 3 clinical trial for ZYN002 in Fragile X syndrome did not meet primary endpoints, leading Needham to lower its price target from $48 to $41 [4] - Cohu, Inc. shares declined 12.7% to $19.85 following the announcement of an offering of convertible senior notes [4] - Rigel Pharmaceuticals, Inc. shares fell 12.7% to $29.69 [4] - BGM Group Ltd. shares decreased by 12.2% to $10.31 [4] - Worthington Enterprises, Inc. shares dipped 11.3% to $53.43 after reporting first-quarter results [4] - QMMM Holdings Limited shares fell 11% to $97.00 [4] - Astera Labs, Inc. shares declined 10.6% to $205.65 [4] - Sandisk Corporation shares dipped 8.2% to $97.68 [4]

Summary of Harmony Biosciences Conference Call Company Overview - **Company**: Harmony Biosciences - **Founded**: October 2017 - **Key Executives**: Jeffrey Dayno (President and CEO), Sandip Kapadia (CFO) [4][5] Industry and Market Context - **Industry**: Biopharmaceuticals focusing on neurological disorders - **Market Size**: Approximately 80,000 patients diagnosed with narcolepsy [9] - **Pipeline**: Robust late-stage pipeline with eight assets across 13 development programs, including six in phase 3 by the end of the year [13] Core Products and Pipeline - **Wakix (Pitolisant)**: - Commercial product for narcolepsy, tracking towards $1 billion in revenue with a 16% sales growth in Q2 [41][43] - Pediatric exclusivity expected to extend market protection until Q3 2030 [42] - Current sales guidance for the year is $820 to $860 million [43] - **ZYN002**: - Targeting Fragile X syndrome, pivotal phase 3 program with top-line data readout expected soon [14][52] - Unique delivery method (transdermal) and synthetic formulation without THC, differentiating it from competitors [17] - Potential market opportunity with 80,000 patients in the U.S., with 60-70% fully methylated [38] - **EPX-100**: - In phase 3 trials for Dravet syndrome and Lennox-Gastaut syndrome [13] - **Erexin Program**: - First-in-human studies expected later this year [57] Key Points on ZYN002 - **Mechanism**: Targets endocannabinoid system disruption in Fragile X syndrome [15][16] - **Clinical Trials**: Phase 2 showed significant outcomes in patients with complete methylation of the FMR1 gene [18][23] - **Regulatory Engagement**: Positive interactions with FDA regarding trial design and endpoints [26][40] Growth Strategies - **Lifecycle Management**: - Introduction of gastro-resistant (GR) and high-dose (HD) formulations of Pitolisant to extend market presence [45][46] - Focus on differentiating new formulations to mitigate loss of exclusivity risks [51] - **Commercial Strategy**: - Strong payer access and broad prescriber universe to drive continued growth [44][50] Future Outlook - **Key Value Drivers**: - Anticipated positive data from ZYN002 and advancement of Pitolisant HD programs [52] - Potential for business development opportunities to expand the pipeline [54] - **Market Position**: - Harmony Biosciences is well-positioned with a strong balance sheet and active in business development [52][54] Additional Insights - **Regulatory Environment**: Consistent communication with FDA despite administrative changes [40] - **Patient-Centric Approach**: Emphasis on the need for approved therapies for conditions like Fragile X syndrome [24] This summary encapsulates the key points discussed during the Harmony Biosciences conference call, highlighting the company's strategic direction, product pipeline, and market opportunities.

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]

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].