Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its investigational drug, govorestat, did not meet the primary endpoint in a late-stage clinical trial for classic galactosemia, a rare and potentially life-threatening genetic disease.
Classic galactosemia is a rare inherited condition in which the body is unable to process galactose, a sugar found in many foods. Without proper treatment, toxic substances build up in the body, leading to serious complications including speech and developmental delays, movement problems, and reproductive issues. Currently, there are no approved therapies that address the underlying causes of the disorder, and treatment options are limited to dietary restrictions.
Applied Therapeutics developed govorestat, an aldose reductase inhibitor, with the aim of reducing harmful byproducts in the body and improving clinical outcomes for patients with classic galactosemia. The company’s Phase III ACTION-Galactosemia Kids trial was designed to evaluate whether the drug could improve symptoms and quality of life for children affected by the disorder.
According to the company’s announcement, the trial did not achieve its main goal: there was no statistically significant difference between patients treated with govorestat and those given a placebo in the primary endpoint, which measured improvement in clinical outcomes. The news is a blow to both Applied Therapeutics and the galactosemia community, who had hoped that the drug could offer the first disease-modifying treatment for this challenging condition.
Despite the disappointing primary results, Applied Therapeutics highlighted some positive findings from secondary endpoints and exploratory measures, suggesting that govorestat may have potential benefits for certain aspects of the disease. The company indicated that it plans to further analyze the trial’s data to better understand these findings and determine if there is a path forward for the drug in classic galactosemia or other indications.
The failure of govorestat to meet its primary endpoint in this trial underscores the challenges of developing therapies for rare diseases, which often have complex biology and small patient populations. For patients and families affected by classic galactosemia, the news is especially difficult, as it represents a missed opportunity for a much-needed treatment.
Applied Therapeutics’ stock price dropped sharply following the announcement, reflecting investor disappointment. The company has not yet indicated whether it will pursue additional clinical development for govorestat in classic galactosemia, but it remains engaged in research for other rare disease indications.
As the field of rare disease drug development continues to advance, the results from this trial highlight both the potential and the uncertainty inherent in bringing new therapies to patients with unmet medical needs. For now, the search for an effective treatment for classic galactosemia continues.
