Applied Therapeutics Hints at FDA Alignment for Rare Disease Drug Govorestat

Applied Therapeutics has signaled potential progress in its development of govorestat, an aldose reductase inhibitor targeting sorbitol dehydrogenase deficiency (SORD), a rare genetic condition. The company recently posted details of a new confirmatory study on clinicaltrials.gov, suggesting a possible agreement with the FDA on the drug's path forward.

New Confirmatory Study Signals Potential Breakthrough

The newly posted study, which appeared on clinicaltrials.gov on September 25, aims to enroll 155 patients across 16 global sites. The trial will focus on patients diagnosed with Charcot-Marie-Tooth Type 2 or distal Hereditary Motor Neuropathy due to SORD. This long-term study is designed to evaluate govorestat's effects on functional, clinical, and patient-reported outcomes compared to placebo.

William Blair analysts interpret this development as a positive sign, suggesting that the FDA may have found previously collected data sufficient to support an accelerated approval for govorestat. However, Applied Therapeutics has yet to officially confirm whether this new study indicates alignment with the FDA on the drug's development path.

Govorestat's Journey: From Setback to Potential Comeback

In May, govorestat faced a significant setback when it failed to meet the primary endpoint in the Phase II/III INSPIRE trial. The drug did not significantly improve scores on the 10-meter walk-run test at 12 months compared to placebo. Despite this failure, Applied Therapeutics remained committed to pursuing a new drug application for govorestat, focusing on positive secondary outcomes.

Evan Bailey, Senior Vice President of Clinical Development at Applied, emphasized the drug's ability to lower blood sorbitol levels and positively impact functional and patient-reported outcomes. The company indicated its intention to work with the FDA to determine the best path forward, aiming for a 2025 filing.

Financial Considerations and Future Outlook

While the new study posting suggests potential progress, William Blair analysts have flagged Applied Therapeutics' financial situation as a concern. As of June 30, the biotech reported $30.4 million in cash and cash equivalents. Analysts warn that this limited cash runway could pose "increased financial risk" for the company as it pursues govorestat's development.

The confirmatory study, which has a primary completion date of October 2028, has yet to start enrolling patients. As Applied Therapeutics moves forward with govorestat's development, the pharmaceutical industry will be watching closely to see if this rare disease treatment can overcome its earlier setbacks and potentially provide a new option for patients with SORD.