NEW YORK - Applied Therapeutics, Inc. (NASDAQ: NASDAQ:APLT), a clinical-stage biopharmaceutical company, has announced the progression of its New Drug Application (NDA) for govorestat, a proposed treatment for Classic Galactosemia. The company recently completed a late-cycle review meeting with the FDA, which has decided that an Advisory Committee meeting, initially set for October 9, 2024, will no longer be necessary. The FDA's review is moving forward as planned, with post-marketing requirements discussions expected in October and a Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024.
Classic Galactosemia is a rare genetic metabolic disorder that impairs the body's ability to process the simple sugar galactose, leading to serious neurological complications. Applied Therapeutics is aiming to provide the first potential treatment for the disease with govorestat, which has demonstrated clinical benefits in children with Galactosemia in studies.
Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor (ARI) currently in development for the treatment of several rare neurological diseases. The drug has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and SORD Deficiency, and Orphan Drug Designation from the U.S. FDA for the treatment of Galactosemia, PMM2-CDG, and SORD Deficiency.
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, expressed satisfaction with the collaborative dialogue with the FDA and emphasized the company's commitment to the Galactosemia community, highlighting the importance of patient access programs, physician awareness, and payer engagement.
The company's pipeline also includes other novel drug candidates targeting unmet medical needs, such as Diabetic Cardiomyopathy and Diabetic Retinopathy.
This article is based on a press release statement and presents the facts around Applied Therapeutics' ongoing FDA review process for govorestat.
In other recent news, Applied Therapeutics' new drug application for govorestat (AT-007), a treatment for galactosemia, is currently under review by the FDA. Both William Blair and RBC Capital Markets have maintained an Outperform rating on the company, highlighting its focus on developing treatments for rare diseases. The anticipation of the FDA's decision on govorestat is seen as a pivotal period for the company, as its approval could transform the treatment landscape for galactosemia. William Blair expressed confidence in the data supporting govorestat, suggesting its efficacy could lead to FDA approval. RBC Capital Markets also noted positive feedback from the FDA on Applied Therapeutics' regulatory roadmap, which includes the potential for an accelerated approval submission for SORD, another genetic disorder. The recent developments indicate a favorable risk/benefit profile for patients with limited treatment options. These developments are part of the company's broader strategy to address rare diseases, with long-term revenue projections estimated at $710 million.
InvestingPro Insights
As Applied Therapeutics, Inc. (NASDAQ: APLT) advances through the regulatory process with the FDA for its drug candidate govorestat, investors and industry observers are closely monitoring the company's financial health and market performance. According to InvestingPro data, Applied Therapeutics holds a market capitalization of approximately $534.04 million. The stock's performance has been quite volatile, with the price having retreated significantly over the last week and month, by -10.75% and -20.51% respectively, although it has seen a high return over the last year with an increase of 134.85%.
The company's valuation metrics present a mixed picture. Applied Therapeutics is trading at a high Price / Book multiple of 7.38, which may suggest the stock is relatively expensive compared to the company's book value. Moreover, the company is not profitable over the last twelve months, and analysts do not anticipate it will be profitable this year either, as reflected in the negative P/E ratio of -3.39.
Two InvestingPro Tips that stand out in relation to Applied Therapeutics include the fact that the company holds more cash than debt on its balance sheet, which provides a degree of financial stability, and that it has liquid assets that exceed short-term obligations, indicating a strong liquidity position. These factors could be particularly relevant for investors considering the risks and opportunities associated with the company's ongoing FDA review process for govorestat.
For those looking for more in-depth analysis, there are an additional 11 InvestingPro Tips for Applied Therapeutics available at InvestingPro, which provide a comprehensive look at the company's financials, market performance, and analyst expectations. These insights can help investors make more informed decisions in the context of the company's current developments.
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