Harmony Biosciences Presents Initial Data on EPX-100 for Dravet Syndrome
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 08 2025
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Harmony Biosciences announced the presentation of initial open-label extension data from the company's ongoing Phase 3 ARGUS trial investigating EPX-100 for the treatment of Dravet syndrome, which showed clinically meaningful reductions in seizure activity in participants with DS along with a favorable benefit-risk profile. The data will be presented at the American Epilepsy Society annual meeting in Atlanta. Data from the eighteen participants in the OLE trial with at least six months' exposure to EPX-100 demonstrated the following: a median reduction of approximately 50% in countable motor seizure frequency per 28 days; 50% of these participants achieved at least a 50% reduction in CMS-28; EPX-100 was generally well-tolerated in participants receiving treatment for more than two years and approaching three years in the OLE phase. The most common treatment emergent adverse events were seizures, pyrexia and upper respiratory tract infection. There were no significant gastrointestinal adverse events and no additional laboratory testing or special monitoring is being performed in the trial. These data suggest an emerging benefit-risk profile for EPX-100, supported by reduction in seizure frequency and a favorable safety/tolerability profile.
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Analyst Views on HRMY
Wall Street analysts forecast HRMY stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for HRMY is 43.83 USD with a low forecast of 32.00 USD and a high forecast of 55.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
6 Analyst Rating
5 Buy
0 Hold
1 Sell
Moderate Buy
Current: 36.840
Low
32.00
Averages
43.83
High
55.00
Current: 36.840
Low
32.00
Averages
43.83
High
55.00
About HRMY
Harmony Biosciences Holdings, Inc. is a pharmaceutical company. It is focused on developing and commercializing therapies for patients living with rare neurological diseases as well as patients living with other neurological diseases who have unmet medical needs. Its product, WAKIX (pitolisant), is a first-in-class molecule with a novel mechanism of action designed to enhance histamine signaling in the brain by binding to H3 receptors. WAKIX was developed for the treatment of excessive daytime sleepiness (EDS) in pediatric patients, six years and older, with narcolepsy. HBS-102, an investigational compound, which is a melanin-concentrating hormone receptor 1 (MCHR1) antagonist. ZYN002 is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS) and 22q11.2 deletion syndrome (22q). It also focused on the rare epilepsy franchise in its expanding late-stage pipeline of CNS assets: EPX-100 and EPX-200.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Harmony Biosciences Expands Wakix Patent Life with New Treatments
- Wakix Success: Harmony Biosciences' Wakix narcolepsy treatment has been highly successful, nearing blockbuster status, but is expected to lose patent exclusivity in 2030, prompting the company to seek new variants to extend patent protection.
- New Drug Application Plans: Harmony aims to submit a new drug application for Pitolisant GR to the FDA by Q1 2027, which could extend patent protection into the 2040s, thereby enhancing its competitive position in the narcolepsy market.
- Clinical Trial Progress: Pitolisant HD is currently in phase 3 trials for treating idiopathic hypersomnia, with topline data expected in 2027, and a successful outcome could pave the way for FDA approval in 2028, expanding treatment indications.
- Multiple Treatment Exploration: Harmony is also conducting clinical trials for BP1.15205 and EPX-100, with the former expected to provide data by mid-year, while EPX-100 has shown significant reductions in seizure frequency among Dravet Syndrome patients, potentially leading to FDA approval in 2028 if results are favorable.
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Harmony Biosciences: Future Prospects and Challenges Ahead
- Patent Protection Extension: Harmony Biosciences' primary drug, Wakix, is set to lose patent exclusivity in 2030, but the development of Pitolisant GR could extend its patent protection into the 2040s, enhancing its competitive position in the narcolepsy market.
- Clinical Trial Progress: Pitolisant HD is currently in phase 3 trials aimed at treating sleep inertia in patients with idiopathic hypersomnia, with topline data expected in 2027, and positive results could pave the way for FDA approval in 2028.
- New Drug Pipeline: Harmony is also developing BP1.15205 and EPX-100, targeting various sleep/wake disorders and showing promise in treating Dravet and Lennox-Gastaut syndromes, with key data anticipated in 2024 that could influence future FDA decisions.
- Investor Confidence Issues: Despite Harmony's strong financial performance, lingering doubts from a 2023 short-seller report have negatively impacted investor sentiment, leading to poor stock performance; however, its relatively attractive valuation offers a margin of safety for potential investors.
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