Core Insights - Harmony Biosciences will present new open-label extension data from the Phase 3 ARGUS trial for EPX-100 (clemizole hydrochloride) at the 2025 American Epilepsy Society Annual Meeting [1][2] - The ARGUS trial is currently enrolling participants, with poster presentations scheduled for December 8, 2025 [2] - EPX-100 is being developed for the treatment of Dravet syndrome and Lennox-Gastaut syndrome, targeting central 5-HT2 receptors to modulate serotonin signaling [2][3] Company Overview - Harmony Biosciences is focused on developing innovative therapies for rare neurological diseases, established in 2017 and headquartered in Plymouth Meeting, PA [4] - The company emphasizes a commitment to addressing unmet medical needs through novel science and community programs [4] Product Information - EPX-100 is administered orally twice a day in a liquid formulation and is based on a proprietary zebrafish drug screening platform [2] - Dravet syndrome, which EPX-100 targets, has an estimated incidence rate of 1 in 15,700 in the US and is primarily caused by SCN1A gene mutations [3]

Core Insights - Harmony Biosciences announced positive results from its pivotal bioequivalence study for pitolisant gastro-resistant formulation, indicating its potential as a next-generation treatment for narcolepsy [1][2] - The company plans to submit a New Drug Application (NDA) in early 2026, targeting a PDUFA date in Q1 2027, with utility patent applications filed for exclusivity until 2044 [1][3] Bioequivalence Study Results - The pivotal bioequivalence study confirmed that 17.8mg of pitolisant gastro-resistant is bioequivalent to existing 17.8mg WAKIX tablets, with AUC and Cmax ratios of 108.46% and 99.65% respectively [2] - No new safety or tolerability issues were reported, and 100% of patients successfully initiated treatment at the therapeutic dose without the need for titration [2] Market Position and Future Developments - The progress on pitolisant gastro-resistant formulation highlights the accelerating momentum of the pitolisant franchise, which is nearing blockbuster status in narcolepsy [3] - Harmony is also developing pitolisant HD, a high-dose formulation aimed at enhancing efficacy for excessive daytime sleepiness and other symptoms in narcolepsy and idiopathic hypersomnia [3] Company Overview - Harmony Biosciences is focused on developing innovative therapies for rare neurological diseases, emphasizing a commitment to patients with unmet medical needs [19]

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]

Core Insights - Harmony Biosciences Holdings, Inc. will participate in several upcoming investor conferences, indicating active engagement with the investment community [1] Conference Details - Citi's 2025 Global Healthcare Conference will take place in Miami, FL on December 2, 2025 [1] - The 8th Annual Evercore Healthcare Conference will also be held in Miami, FL, featuring a fireside chat on December 2, 2025, at 2:35 p.m. ET [1] - Piper Sandler's 37th Annual Healthcare Conference is scheduled in New York, NY, with a fireside chat on Wednesday [1]

Core Insights - Harmony Biosciences reported quarterly earnings of $0.87 per share, missing the Zacks Consensus Estimate of $0.92 per share, but showing an increase from $0.79 per share a year ago, resulting in an earnings surprise of -5.43% [1] - The company posted revenues of $239.46 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 4.59% and up from $186.04 million year-over-year [2] - Harmony Biosciences shares have declined approximately 14.8% year-to-date, contrasting with the S&P 500's gain of 16.5% [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is $1.05 on revenues of $239.18 million, and for the current fiscal year, it is $3.23 on revenues of $864.24 million [7] - The estimate revisions trend for Harmony Biosciences was mixed prior to the earnings release, resulting in a Zacks Rank 3 (Hold) for the stock, indicating expected performance in line with the market [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Harmony Biosciences belongs, is currently ranked in the top 39% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

Financial Data and Key Metrics Changes - The company reported net revenue of $239.5 million for Q3 2025, representing a 29% year-over-year growth [5][20] - Cash generation was robust at $106 million, bringing the total cash and cash equivalents to $778 million as of September 30, 2025 [5][22] - Non-GAAP adjusted net income for Q3 2025 was $63.5 million, or $1.8 per diluted share, compared to $57.3 million, or $0.99 per diluted share in the prior year quarter [21][22] Business Line Data and Key Metrics Changes - The WCAGX product line achieved an average of 8,100 patients by the end of Q3, with a record increase of approximately 500 new patients added during the quarter [9][10] - The company raised its full-year revenue guidance from $820 million to a range of $845 million to $865 million, reflecting strong demand for WCAGX [12][23] Market Data and Key Metrics Changes - The company noted that WCAGX is rapidly approaching blockbuster status, with expectations of exceeding $1 billion in annual revenue from narcolepsy alone [6][25] - The company has secured important new payer coverage wins, which continue to expand its already broad payer coverage [11] Company Strategy and Development Direction - The company aims to become a leading patient-focused CNS company, delivering innovative treatments for unmet medical needs [7] - The late-stage pipeline includes multiple catalysts, with plans to initiate two Phase III trials for Pitolacin HD by the end of 2025 [6][14] - The company is focused on strategic business development to enhance its pipeline and product portfolio [46][67] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth trajectory, driven by strong fundamentals and execution across the organization [12][113] - The company anticipates ongoing investments in R&D to advance its late-stage pipeline, with five Phase III registrational programs expected by year-end [23][24] Other Important Information - The company has filed utility patents for both Pitolacin GR and HD, securing potential exclusivity until 2044 [16][76] - The IND for Pitolacin HD has been submitted to the FDA, with target PDUFA dates set for 2028 [14][16] Q&A Session Summary Question: Can you elaborate on the trajectory of WCAGX evolving into 2026? - Management noted that the performance is driven fundamentally by patient adds, with a record increase of 500 patients in Q3, and strong fundamentals are expected to carry forward into Q4 and 2026 [30][33] Question: What were the GI AEs seen with the GR formulation? - No new safety or tolerability issues were observed with the Pitolacin GR formulation, and all patients were able to initiate treatment at the therapeutic dose without issues [35][36] Question: What are the current thoughts on business development and M&A? - The company remains focused on strategic business development, actively evaluating several opportunities to enhance its pipeline [46][67] Question: How is new patient growth coming from? - New patient growth is primarily from deeper penetration within existing prescribers, with a stable breakdown of NT1 and NT2 patients [120][97] Question: Can you discuss the EPX-100 epilepsy program? - The company is excited to share efficacy data at the upcoming American Epilepsy Society meeting and continues to recruit patients for ongoing studies [87][88]

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]

Drug Approvals and Clinical Trials - Pitolisant was approved by the FDA for the treatment of EDS in adult patients with narcolepsy in August 2019, with commercial launch initiated in November 2019[123]. - The FDA granted Orphan Drug designation to pitolisant for the treatment of PWS in February 2024, with the Phase 3 TEMPO study initiated in Q1 2024[125]. - The Phase 2 trial for pitolisant in adult patients with DM1 showed clinically meaningful improvements in EDS and fatigue, with topline results announced in Q4 2023[125]. - The pivotal bioequivalence study for Pitolisant GR is anticipated to have topline data readout in Q4 2025, with a PDUFA date expected in Q1 2027[129]. - The company received positive pre-clinical data for BP1.15205, an orexin-2 receptor agonist, with first-in-human studies expected to begin in Q4 2025[130]. - A research collaboration agreement with CiRC Biosciences was established in June 2025 to develop candidates for refractory epilepsies and treatment-resistant narcolepsy[131]. - The FDA did not approve the sNDA for expanding the WAKIX label for pediatric patients with cataplexy in June 2024, but a Type A meeting was held to discuss a resubmission path[127]. - The company is committed to obtaining pediatric exclusivity for WAKIX, supported by recent FDA approvals and ongoing studies[128]. - The RECONNECT study for ZYN002 in Fragile X Syndrome did not meet its primary endpoint due to a higher than expected placebo response rate, leading to a review of the full data set[134]. - EPX-100 is currently in two Phase 3 registrational clinical trials, the ARGUS Study for Dravet Syndrome and the LIGHTHOUSE Study for Lennox-Gastaut Syndrome[135]. Financial Performance - Net product revenue for the three months ended September 30, 2025, was $239.5 million, a 28.7% increase compared to the same period in 2024, driven by a 26.8% increase in units shipped[156]. - For the nine months ended September 30, 2025, net product revenue reached $624.7 million, reflecting a 21.7% increase year-over-year, primarily due to an 18.0% increase in units shipped[156]. - The cost of product sales for the three months ended September 30, 2025, was $59.7 million, up from $42.8 million in the same period in 2024[155]. - Research and development expenses for the three months ended September 30, 2025, were $55.0 million, significantly higher than $25.4 million in the same period in 2024, reflecting increased clinical program efforts[155]. - Interest expense decreased by $0.7 million, or 16.7%, for the three months ended September 30, 2025, and decreased by $2.2 million, or 16.4%, for the nine months ended September 30, 2025, compared to the same periods in 2024[164]. - Interest income increased by $0.8 million, or 16.2%, for the three months ended September 30, 2025, and increased by $2.0 million, or 14.3%, for the nine months ended September 30, 2025, compared to the same periods in 2024[165]. - The company achieved a net income of $136.2 million for the nine months ended September 30, 2025, compared to $96.0 million in 2024, marking a 42% increase[183][184]. Expenses and Cost Management - The company expects sales and marketing expenses to rise in the near- and mid-term to support WAKIX's commercialization and potential new indications[147]. - General and administrative expenses are anticipated to increase due to hiring additional personnel and compliance costs associated with being a public company[150]. - Cost of product sales increased by $16.9 million, or 39.4%, for the three months ended September 30, 2025, and increased by $27.4 million, or 26.7%, for the nine months ended September 30, 2025, compared to the same periods in 2024[157]. - Research and development expenses increased by $29.6 million, or 116.5%, for the three months ended September 30, 2025, and increased by $28.5 million, or 25.6%, for the nine months ended September 30, 2025, compared to the same periods in 2024[159]. - Sales and marketing expenses increased by $2.0 million, or 7.2%, for the three months ended September 30, 2025, and increased by $7.0 million, or 8.4%, for the nine months ended September 30, 2025, compared to the same periods in 2024[162]. - General and administrative expenses increased by $1.2 million, or 4.3%, for the three months ended September 30, 2025, and increased by $13.5 million, or 16.6%, for the nine months ended September 30, 2025, compared to the same periods in 2024[163]. Cash Management and Investments - As of September 30, 2025, the company had cash, cash equivalents, and investments of $778.4 million, outstanding debt of $170.0 million, and retained earnings of $138.4 million[168]. - For the nine months ended September 30, 2025, cash provided by operating activities was $222.0 million, up from $144.3 million in the same period of 2024, representing a 54% increase[182]. - Net cash used in investing activities for the nine months ended September 30, 2025, was $22.0 million, a decrease from $60.3 million in 2024, indicating improved cash management[185][186]. - The company reported net cash used in financing activities of $6.0 million for the nine months ended September 30, 2025, compared to $8.3 million in 2024, reflecting a reduction in financing costs[187][188]. - As of September 30, 2025, the company had $685.2 million in investments, with an immediate 10% change in market interest rates not expected to materially impact the investment portfolio[194]. - The company had $170.0 million in borrowings outstanding as of September 30, 2025, with interest rates based on a margin ranging from 2.50% to 4.00% depending on the leverage ratio[195]. Acquisitions and Agreements - In October 2023, the company acquired Zynerba Pharmaceuticals, gaining global rights to develop and commercialize ZYN002, a synthetic cannabidiol gel for transdermal delivery[134]. - In April 2024, the company acquired Epygenix Therapeutics, obtaining an exclusive license for EPX-100, targeting Dravet Syndrome and Lennox-Gastaut Syndrome, with a total addressable market of approximately 5,000 and 35,000 patients respectively[135]. - The company acquired HBS-102, a MCHR1 antagonist, with encouraging results from preclinical studies reported in Q3 2024[133]. - The company entered into a License and Commercialization Agreement with Bioprojet in July 2022, obtaining exclusive rights to develop new products based on pitolisant in the U.S. and Latin America[129]. - The company entered into a sublicense agreement with Bioprojet in April 2024, paying an upfront license fee of $25.5 million and potentially up to $367.5 million upon achieving development and sales milestones[177]. - The company entered into a research collaboration with CiRC Biosciences, Inc., paying an upfront fee of $15.0 million and potential milestone payments of $2.0 million for each of the two candidates[179][180]. - A clinical milestone for ZYN002 was achieved in September 2025, triggering a $15.0 million payment to contingent value rights holders, expected to be paid in Q4 2025[181].

Financial Performance - Q3 2025 net product revenue reached $239.5 million, a 29% increase year-over-year compared to $186.0 million in Q3 2024[6, 36] - The company raised its full-year 2025 revenue guidance to $845 million - $865 million[6] - Non-GAAP adjusted net income increased by 11% from $57.3 million in Q3 2024 to $63.5 million in Q3 2025[36] - Cash, cash equivalents, and investments increased to $778.4 million in Q3 2025, a 54% increase compared to $504.7 million in Q3 2024[36, 38] WAKIX Performance - WAKIX average patients reached 8,100[11] - Approximately 500 average patient additions, representing the highest quarterly increase since launch[11] - Strong payer coverage of WAKIX at >80% of lives[11] Pipeline Development - The company plans to initiate Phase 3 registrational trials for Pitolisant HD in narcolepsy and IH in Q4 2025[20, 21] - Topline data from the pivotal bioequivalence study of Pitolisant GR is expected in Q4 2025[25, 33] - Phase 3 registrational trials are ongoing for EPX-100 in Dravet Syndrome (ARGUS study) and Lennox-Gastaut Syndrome (LIGHTHOUSE study), with topline data anticipated in 2026[19, 31, 33] - The Phase 3 RECONNECT study for ZYN002 in Fragile X Syndrome (FXS) did not meet the primary endpoint due to a higher-than-expected placebo response rate, and a review of the full data set is ongoing[19, 28]

Financial Performance - WAKIX franchise generated net revenue of approximately $239 million in Q3 2025, representing a 29% year-over-year revenue growth[2] - The company raised its 2025 revenue guidance to a range of $845-$865 million from the previous range of $820-$860 million[2] - Harmony Biosciences will report full Q3 2025 financial results on November 4th, 2025, before the U.S. financial markets open[2] - The preliminary results are based on management's initial review and are subject to revision, indicating potential changes in actual results[4] Product Performance and Market Potential - The average number of patients increased by approximately 500, reaching 8,100 average patients in Q3 2025, indicating a strong trajectory towards potential blockbuster status for WAKIX in narcolepsy[2] - WAKIX is approved for treating excessive daytime sleepiness and cataplexy in adult and pediatric patients with narcolepsy, showcasing its therapeutic potential[5] - The company is exploring the therapeutic potential of pitolisant in additional indications, which may enhance its market position[24] Strategic Focus and Development - Harmony is committed to developing innovative therapies for patients with rare neurological diseases, reflecting its strategic focus[21] - The company emphasizes the importance of favorable insurance coverage and reimbursement for WAKIX to support its commercialization efforts[24] - Harmony's growth strategy includes identifying and integrating additional products or candidates with significant commercial potential[24]