Core Insights - Beacon Biosignals and Harmony Biosciences have announced a collaboration to integrate quantitative EEG measurements into Harmony's Phase 3 studies for HBS-301 targeting Narcolepsy and Idiopathic Hypersomnia [1][2] Group 1: Collaboration Details - The collaboration aims to enhance the assessment of excessive daytime sleepiness (EDS) by incorporating objective EEG data alongside traditional patient-reported outcomes like the Epworth Sleepiness Scale (ESS) [2] - Beacon's FDA 510(k)-cleared Waveband EEG headband will be utilized to capture at-home brain data over consecutive nights, providing continuous EEG data to assess daytime sleepiness [3] Group 2: Objectives and Benefits - The integration of objective sleep EEG data is expected to improve the precision and interpretability of treatment outcomes, potentially accelerating the development of better therapies for hypersomnia disorders [4] - The collaboration seeks to bring new levels of objectivity and reliability to clinical endpoints, enhancing the evaluation of treatment benefits for patients with hypersomnia disorders [4] Group 3: Clinical Context - Narcolepsy and Idiopathic Hypersomnia are characterized by severe excessive daytime sleepiness, with current trial measures relying heavily on subjective patient-reported scales, which may not fully capture the disease burden [4] - The addition of quantitative EEG-based endpoints allows for a more robust characterization of sleep patterns, aiding in the identification of EEG biomarkers for dose selection and efficacy assessment [4] Group 4: Company Profiles - Beacon Biosignals specializes in AI-driven neurophysiology and precision drug development, utilizing wearable EEG technology to capture and analyze neural data for advancements in neurology and sleep medicine [6] - Harmony Biosciences focuses on developing innovative therapies for rare neurological disorders, with a portfolio that includes FDA-approved treatments for narcolepsy and a pipeline targeting sleep/wake disorders and epilepsy [7]

"The ARGUS trial is one of the most advanced development programs in the 5-HT2 (serotonin) agonist class and the effectiveness, safety and tolerability data of EPX-100 dosed BID from the open-label extension study are very encouraging,†said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. "These initial results support the advancement of our epilepsy franchise as we progress toward the topline data readout from the ARGUS trial in 2026.†EPX-100 is being evaluated as an inve ...

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.

Harmony Biosciences FY Conference Summary Company Overview - **Company**: Harmony Biosciences (NasdaqGM:HRMY) - **Industry**: Biotechnology, specifically focusing on treatments for narcolepsy and epilepsy Key Points and Arguments Business Performance and Growth - Harmony Biosciences has established a strong commercial foundation, particularly with its product Wakix, which targets narcolepsy and is projected to be a $1 billion-plus opportunity in this market [2][3] - The company is profitable and self-funding its activities, allowing for continued growth in both its pipeline and commercial portfolio [3][4] Wakix Franchise - Wakix has shown consistent performance with a steady increase in patient numbers, reporting an increase of 400 patients in Q2 and 500 patients in Q3, leading to an upward revision of revenue guidance from $820-$860 million to $845-$865 million for the year [6][7] - The product is differentiated as the only non-scheduled treatment for narcolepsy, which is well tolerated and has a unique mechanism of action [8][10] - The company plans to maintain its competitive edge through life cycle management strategies, including new formulations such as gastro-resistant (GR) and high-dose (HD) versions of pitolisant [10][11] Pipeline Developments - Harmony is advancing its pipeline with new formulations and additional assets, including orexin-2 agonists currently in phase one clinical trials and EPX-100 for epilepsy in phase three trials [3][4] - Upcoming data presentations at the American Epilepsy Society meeting are expected to provide new efficacy data for EPX-100 [3] Market Dynamics and Competitive Position - The narcolepsy market is characterized by a high discontinuation rate of 30%-50% for medications, but once patients are stabilized on Wakix, they tend to remain on therapy [17] - The company emphasizes that Wakix's unique profile will remain competitive despite the introduction of generics in the market [15][16] Future Strategies - The GR formulation aims to provide a faster therapeutic effect without the need for titration, while the HD formulation targets improved efficacy and new indications for fatigue in narcolepsy patients [11][19] - Harmony is also exploring business development opportunities to expand its commercial portfolio and pipeline further [34] Challenges and Considerations - The company is currently assessing the future of its Cannabidiol Gel (ZYN002) program following disappointing phase three results, with plans to provide updates after a thorough data review [31][32] - The transition from a single product focus to a multi-franchise business model requires careful organizational growth to ensure all programs receive adequate attention [33] Additional Important Insights - The company has a robust commercial engine and is looking to leverage its balance sheet to drive further value through business development [34] - The ongoing clinical trials and product developments are expected to enhance Harmony's position in the biotechnology sector, particularly in the treatment of sleep disorders and epilepsy [3][4]

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]