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WASHINGTON - Vanda Pharmaceuticals Inc. (NASDAQ: NASDAQ:VNDA), a biopharmaceutical company with impressive gross profit margins of 94.3% and a market capitalization of $258 million, has recently announced the development of a novel antisense oligonucleotide (ASO) therapeutic, VCA-894A, targeting a rare genetic mutation associated with Charcot-Marie-Tooth disease Type 2S (CMT2S). According to InvestingPro analysis, the company maintains strong financial health with more cash than debt on its balance sheet. The research article detailing this advancement was published in Molecular Therapy Nucleic Acids, a partner journal of Cell Press.
CMT2S is a hereditary neuromuscular disorder characterized by progressive muscle weakness and loss of motor function, with an estimated prevalence of fewer than one in one million people worldwide. The therapeutic, VCA-894A, has been designed specifically for a patient with a unique variant in the IGHMBP2 gene, which is responsible for this condition. While the company reported a revenue of $199 million in the last twelve months, analysts tracked by InvestingPro expect significant growth in net income this year.
Vanda’s President, CEO, and Chairman of the Board, Mihael H. Polymeropoulos, M.D., highlighted the significance of this development as a step towards personalized medicine, moving away from a one-size-fits-all treatment approach. He emphasized the role of routine and affordable genome sequencing in identifying genetic causes of diseases and facilitating the creation of targeted therapeutics.
The study demonstrated that VCA-894A could significantly improve neuromuscular function, as observed in Hesperos’ Human-on-a-Chip® neuromuscular junction model using patient-derived cells. The treatment showed reduced muscle fatigue and enhanced synaptic transmission, indicating a potential breakthrough in precision medicine and genetically tailored treatments.
The U.S. Food and Drug Administration (FDA) has granted VCA-894A an orphan designation, and it is expected to be administered to the patient it was developed for shortly. This experimental platform could pave the way for addressing unmet medical needs through genetic insights and precision medicine.
Vanda Pharmaceuticals is a global biopharmaceutical company dedicated to the development and commercialization of innovative therapies to improve patient lives and address high unmet medical needs. InvestingPro’s Fair Value analysis suggests the stock is currently undervalued, with a strong current ratio of 4.39 indicating excellent liquidity. Discover more insights about VNDA and 1,400+ other stocks through comprehensive Pro Research Reports available on InvestingPro.
The information in this article is based on a press release statement from Vanda Pharmaceuticals Inc.
In other recent news, Vanda Pharmaceuticals reported its fourth-quarter 2024 earnings, with revenue totaling $53.2 million, surpassing the forecast of $51.23 million. The company also exceeded expectations with an earnings per share (EPS) of -$0.08, compared to the anticipated -$0.17. For the full year, Vanda’s net loss was $18.9 million, a shift from the previous year’s net income of $2.5 million, attributed to increased competition and strategic investments. Vanda has set its 2025 revenue guidance to range between $210 million and $250 million. H.C. Wainwright raised its price target for Vanda to $20, maintaining a Buy rating, following the company’s financial results. The firm anticipates that revenue growth will accelerate with the expected launches of new products. Cantor Fitzgerald also maintained its Overweight rating with a $13 price target, expressing optimism about Vanda’s commercial trajectory. Additionally, Vanda announced changes to executive compensation, including adjustments to salaries and bonuses, as well as the awarding of restricted stock units.
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