Welcome to our dedicated page for Harmony Biosciences Holdings news (Ticker: HRMY), a resource for investors and traders seeking the latest updates and insights on Harmony Biosciences Holdings stock.
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) is a commercial-stage pharmaceutical company committed to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Established in 2017 and headquartered in Plymouth Meeting, PA, Harmony focuses on rare and orphan diseases, particularly those involving the central nervous system.
Harmony's flagship product is WAKIX® (pitolisant). This molecule is specifically designed to increase histamine signaling in the brain by binding to H3 receptors. It is used for the treatment of excessive daytime sleepiness (EDS) and cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019 and continues to demonstrate strong market performance.
In recent developments, Harmony has initiated a global Phase 3 TEMPO study to evaluate pitolisant for treating EDS and behavioral symptoms in patients with Prader-Willi syndrome (PWS). The U.S. FDA granted Orphan Drug designation to pitolisant for this condition, further underscoring the potential of this therapy to address significant unmet medical needs.
The company's financial performance remains robust, with a 31% increase in net product revenue in Q4 2023 compared to the same period in 2022. Harmony’s strategic growth continues with key acquisitions, such as Epygenix Therapeutics, Inc., which expands its pipeline with promising treatments for rare forms of epilepsy like Dravet syndrome and Lennox-Gastaut syndrome.
Harmony recently announced the FDA's priority review for its supplemental New Drug Application (sNDA) for WAKIX for the treatment of EDS or cataplexy in pediatric patients aged six and older with narcolepsy. This decision highlights the pressing need for pediatric treatment options and demonstrates Harmony's commitment to broadening the clinical utility of its products.
Beyond narcolepsy, Harmony is exploring pitolisant's potential for other rare conditions, including Idiopathic Hypersomnia and Myotonic Dystrophy Type 1 (DM1). The company presented encouraging Phase 2 data showing pitolisant's efficacy in reducing EDS and fatigue in DM1 patients, paving the way for a pivotal Phase 3 study.
Harmony's collaborative efforts extend to partnerships with Bioprojet for the development of TPM-1116, an orexin-2 receptor agonist with potential applications in narcolepsy and other sleep/wake disorders. These initiatives align with the company's mission to deliver innovative treatments that improve the lives of patients living with rare neurological diseases.
For more information, please visit www.harmonybiosciences.com.
On June 25, 2024, Harmony Biosciences announced that the FDA denied a Citizen Petition filed by a short seller, which challenged the safety and efficacy of WAKIX (pitolisant) for treating excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The FDA confirmed WAKIX's favorable benefit-risk profile, rejecting the petition's requests, including the drug's withdrawal and transition to a Compassionate Use Program. Furthermore, the FDA had recently approved a supplemental New Drug Application (sNDA) for WAKIX, extending its use to pediatric patients aged 6 and older with narcolepsy. Harmony Biosciences plans to launch at least one new product or indication annually over the next five years and aims to extend the pitolisant franchise beyond 2040 with next-generation formulations.
Harmony Biosciences (Nasdaq: HRMY) has received FDA approval for its drug WAKIX® (pitolisant) to treat excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy. This approval follows a Phase 3 study by Bioprojet, which also led to European Medicines Agency approval for the same use last year. While WAKIX will now be available for pediatric EDS, FDA has issued a complete response for its use in treating cataplexy in this age group. WAKIX, first approved in 2019 for adult narcolepsy, is the only FDA-approved non-scheduled treatment for this condition. The company plans to discuss further pathways for cataplexy treatment in pediatric patients with the FDA.
Harmony Biosciences (Nasdaq: HRMY) has reported positive data from its Phase 2 study on pitolisant for treating excessive daytime sleepiness (EDS) and fatigue in myotonic dystrophy type 1 (DM1) patients. The study showed significant improvement in EDS and fatigue symptoms with higher doses of pitolisant compared to placebo. The results encourage further development of pitolisant using the Next-Generation 2 (NG2) formulation for a pivotal Phase 3 study. The study included a randomized, double-blind, placebo-controlled design with a total of 25 participants. Both primary and secondary efficacy endpoints indicated greater mean improvements for pitolisant over placebo, and adverse event rates were similar across groups. Pitolisant, marketed as WAKIX® in the U.S., is currently FDA approved for narcolepsy-related EDS and cataplexy but remains investigational for DM1.
Harmony Biosciences announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference.
The event will take place on June 10, 2024, in Miami Beach, FL.
The management team will engage in a fireside chat at 9:20 a.m. ET.
Investors can access a webcast of the session on Harmony's investor relations page.
Harmony Biosciences Holdings, Inc. reported strong first-quarter financial results with a net revenue growth of 30% year-over-year. The company is accelerating its growth strategy by extending the revenue potential of its Pitolisant franchise beyond 2040. They are strengthening their sleep/wake leadership with new drug applications for Pitolisant in Idiopathic Hypersomnia, pediatric exclusivity, and positive pharmacokinetic data on next-generation formulations. Harmony is also diversifying into rare epilepsy through an acquisition, reiterating 2024 net product revenue guidance of $700 - $720 million. The company expects to launch new products or indications annually over the next five years, with multi-billion-dollar revenue potential extending beyond 2040.
Harmony Biosciences acquired Epygenix Therapeutics, Inc., adding a rare epilepsy franchise to its CNS pipeline. The lead candidate, EPX-100, is in a pivotal trial for Dravet Syndrome with topline data expected in 2026. EPX-100 holds Orphan Drug and Rare Pediatric Disease designations. Harmony paid $35 million in cash with potential payments up to $130 million based on milestones.
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