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PLYMOUTH MEETING, Pa. - Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY), a profitable pharmaceutical company with annual revenue of $714.73 million and an impressive 78% gross profit margin, shared updated data from its ongoing study on ZYN002, a synthetic cannabidiol gel, showing potential benefits for patients with Fragile X syndrome (FXS), a rare genetic condition. The company presented these findings at the American Academy of Neurology Annual Meeting on Tuesday in San Diego. According to InvestingPro analysis, Harmony maintains strong financial health with a "GREAT" overall score.
The Open-Label Extension (OLE) study, which followed earlier trial phases, indicated improvements in irritability-related symptoms in children, adolescents, and adults with FXS. FXS is characterized by a range of intellectual disabilities and behavioral symptoms and currently lacks FDA-approved treatments.
During the OLE trial, participants who had previously taken part in the CONNECT-FX and FAB-C trials were observed for changes in their behavior. Results showed that over 60% of participants in both the treatment and placebo-to-treatment groups experienced a clinically meaningful improvement in behavioral symptoms. Caregivers also reported improvements in patient behavior, with 73.3% of those continuing ZYN002 treatment and 72% transitioning from placebo noting beneficial changes after three years. The stock, currently trading at $28.40 near its 52-week low, appears undervalued according to InvestingPro's Fair Value analysis, suggesting potential upside for investors interested in this promising development.
The study involved 240 patients, aged between 3 to 17 years, with dosages of ZYN002 adjusted based on weight. While adverse events were reported, only a small fraction led to discontinuation of treatment, and the most common side effect was pain at the application site.
ZYN002 is a THC-free, synthetic cannabidiol formulated for transdermal delivery and has received Orphan Drug Designation and Fast Track designation from the FDA for FXS treatment. Harmony Biosciences is currently conducting the Phase 3 RECONNECT study, aiming to further evaluate ZYN002's efficacy and safety.
Harmony Biosciences, established in 2017, focuses on developing therapies for rare neurological diseases. The company's commitment to addressing unmet medical needs in conditions like FXS is underscored by the potential of ZYN002 to become the first approved treatment for this disorder. With a solid balance sheet showing more cash than debt and strong cash flows to cover interest payments, Harmony appears well-positioned to continue its development programs. Discover more detailed insights and 11 additional ProTips about HRMY with a comprehensive Pro Research Report, available exclusively on InvestingPro.
The information in this article is based on a press release statement from Harmony Biosciences Holdings, Inc.
In other recent news, Harmony Biosciences Holdings, Inc. announced the appointment of Adam Zaeske as its new Executive Vice President and Chief Commercial Officer, succeeding Jeffrey Dierks. This leadership change comes as the company focuses on the growth of its narcolepsy drug, WAKIX, and advancing its late-stage pipeline assets. Meanwhile, Mizuho Securities has increased its price target for Harmony Biosciences to $44, maintaining an Outperform rating, citing the company's promising pipeline and potential for multiple product launches. H.C. Wainwright has adjusted its price target to $70 from $75, keeping a Buy rating, following Harmony's fourth-quarter and full-year 2024 financial results. The firm remains confident in Harmony's projected revenues, despite a recent FDA setback for WAKIX in treating idiopathic hypersomnia. Cantor Fitzgerald has reaffirmed its Overweight rating with a $54 price target, highlighting the company's progress in clinical trials and its focus on rare neurological conditions. Additionally, Harmony Biosciences has expanded its board with the appointment of Ron Philip, a seasoned biopharmaceutical executive, to support its strategic growth and business development efforts. These developments underscore Harmony Biosciences' ongoing efforts to strengthen its leadership and advance its clinical programs.
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