Applied Therapeutics Charts Path Forward After FDA Setback, Eyes European Approval and Pipeline Expansion

Applied Therapeutics (NASDAQ: APLT) received a warning letter from the FDA concerning its study of govorestat, a drug intended to treat the rare genetic disease galactosemia. This follows last week’s FDA rejection of the drug’s marketing application, citing deficiencies.

The warning letter identifies issues with electronic data capture and a dosing error during the trial’s dose-escalation phase, which resulted in slightly lower dosing levels in a limited number of patients. Applied Therapeutics stated that these issues had been addressed through prior communications and provided detailed records to the FDA.

The company plans to respond to the FDA within 15 business days to address the concerns. Applied Therapeutics’ CEO, Shoshana Shendelman, reaffirmed the company’s commitment to working with the FDA to determine a clear path forward for govorestat.

Govorestat is designed to treat galactosemia by targeting the aldose reductase enzyme to prevent the accumulation of toxic galactitol, a major cause of complications in patients. Despite initial clinical data showing promise in reducing galactitol levels, the drug’s Phase III trial fell short of its primary efficacy endpoint.Applied Therapeutics’ European approval process for govorestat remains on track, with a decision expected in the first quarter of 2025. Additionally, the company is advancing a promising pipeline, including candidates targeting diabetic cardiomyopathy and diabetic retinopathy, which address significant unmet medical needs.

The company is also pursuing govorestat as a treatment for SORD deficiency, with plans to file a New Drug Application for this indication in early 2025.

In September 2024, Applied Therapeutics had $99 million in cash and was debt-free. Over the past year, the company burned through $82 million, leaving it with an estimated 14 months of cash runway as of September. With a market capitalization of $236 million, the company’s cash burn represents approximately 35% of its market value.

Despite the recent setbacks, Applied Therapeutics remains focused on its broader pipeline and upcoming regulatory milestones to drive growth and innovation.