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FDA grants bexicaserin orphan drug status for Dravet syndrome

Published 09/19/2024, 08:37 AM
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LA JOLLA, Calif. - Longboard Pharmaceuticals, Inc. (NASDAQ:LBPH), a biopharmaceutical company specializing in neurological disease treatments, has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for bexicaserin, its drug candidate for Dravet syndrome treatment.


The FDA's Orphan Drug designation is intended to facilitate drug development for rare diseases, offering benefits such as tax credits for clinical trials, user fee waivers, and potential market exclusivity for seven years post-approval. The Rare Pediatric Disease designation could lead to a Priority Review Voucher (PRV) for Longboard, which can expedite the review process for another product or be monetized by selling it to another sponsor.


Dr. Randall Kaye, Longboard's Chief Medical Officer, expressed appreciation for the FDA's support, which bolsters the company's efforts to address the significant unmet medical needs of Developmental and Epileptic Encephalopathies (DEEs) patients. Longboard anticipates initiating its global Phase 3 DEEp program, beginning with the DEEp SEA trial for Dravet syndrome, in the upcoming weeks.


Bexicaserin, also known as LP352, is a selective 5-HT2C receptor superagonist with no observed impact on related receptor subtypes. It is part of Longboard's portfolio that targets G protein-coupled receptors (GPCRs). The company's commitment to advancing bexicaserin is supported by positive data from a Phase 1b/2a clinical trial. Another candidate, LP659, is in development for neuroinflammatory conditions, having completed a Phase 1 trial.


It is important to note that bexicaserin and LP659 are investigational compounds and have not yet been approved for marketing by the FDA or any other regulatory authority. The forward-looking statements included in Longboard's press release reflect the optimism of the company's executives but are subject to risks and uncertainties inherent in drug development.


This report is based on a press release statement from Longboard Pharmaceuticals, Inc.


In other recent news, Longboard Pharmaceuticals has seen several significant developments in its drug development programs and market analysis. Following the company's Investor & Analyst Day, H.C. Wainwright increased its price target for Longboard to $80, maintaining a Buy rating. The company's focus was on its Phase 3 Developmental and Epileptic Encephalopathies (DEEs) treatment program, specifically bexicaserin (LP352). The program has been expanded to cover a broader range of DEEs, a strategic shift facilitated by the FDA's Breakthrough Therapy Designation (BTD) received on July 1.


Longboard also plans to initiate a Phase 3 program by the end of 2024 and expects Phase 1 data for another therapeutic candidate, LP659, in the second quarter of 2024. Meanwhile, Baird maintains an Outperform rating and a steady price target of $60.00 for Longboard's stock, following the company's detailed plans for phase 3 trials of bexicaserin. Truist Securities initiated coverage on Longboard with a Buy rating, setting a price target of $60, suggesting that bexicaserin could outperform the current market leader in epilepsy treatment.


These developments reflect the confidence of multiple firms in Longboard's growth prospects and its potential to address significant unmet needs in epilepsy treatment. As these are recent developments, investors and stakeholders are closely watching Longboard's progress in its DEE treatment landscape.


InvestingPro Insights


As Longboard Pharmaceuticals, Inc. (NASDAQ:LBPH) advances its clinical programs with recent FDA designations for bexicaserin, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, Longboard holds a market capitalization of $1.14 billion, reflecting investor confidence despite the company's challenges. However, the stock's P/E ratio stands at -13.7, indicating that the market currently does not expect earnings to cover its share price.


InvestingPro Tips suggest a mixed picture for Longboard. On the positive side, the company holds more cash than debt on its balance sheet (InvestingPro Tip #0), which could provide financial flexibility as it moves forward with its Phase 3 trials. Moreover, liquid assets surpass short-term obligations (InvestingPro Tip #8), further indicating a robust short-term financial position. Yet, analysts have revised their earnings downwards for the upcoming period (InvestingPro Tip #1), which may reflect concerns about the company's profitability in the near term. Additionally, it's notable that the stock has experienced a substantial decline over the last week (InvestingPro Tip #2), which could be a point of consideration for potential investors.


For those interested in delving deeper into Longboard Pharmaceuticals' financial metrics and future prospects, InvestingPro offers a comprehensive list of additional tips, providing a more nuanced understanding of the company's performance and potential investment opportunities.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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