FDA Alignment Hinted for Applied Therapeutics' Rare Disease Drug

Applied Therapeutics may have reached a crucial milestone in the development of its rare disease drug, govorestat, as a new confirmatory study suggests potential alignment with the FDA. This development comes despite previous setbacks in clinical trials and highlights the complex landscape of rare disease drug development.

New Confirmatory Study Signals Potential FDA Agreement

A new study for govorestat, an aldose reductase inhibitor, has been posted on clinicaltrials.gov, indicating possible progress in the drug's development for sorbitol dehydrogenase deficiency (SORD). The study, which aims to enroll 155 patients across 16 global sites, is designed to evaluate the long-term effects of govorestat on functional, clinical, and patient-reported outcomes in individuals with Charcot-Marie-Tooth Type 2 or distal Hereditary Motor Neuropathy due to SORD.

William Blair analysts suggest that this new confirmatory study could mean the FDA finds previously collected data sufficient to support an accelerated approval for govorestat. However, Applied Therapeutics has yet to officially confirm whether this study signifies alignment with the FDA on the drug's development path.

Overcoming Previous Trial Setbacks

The potential breakthrough comes after govorestat failed to meet its primary endpoint in the Phase II/III INSPIRE trial earlier this year. Despite not significantly improving scores on the 10-meter walk-run test at 12 months compared to placebo, Applied Therapeutics remained committed to pursuing a new drug application. The company focused on positive secondary outcomes, including improvements in mobility and quality of life, as well as significant reductions in blood sorbitol levels—a key disease biomarker.

Evan Bailey, Senior Vice President of Clinical Development at Applied Therapeutics, expressed encouragement about the data supporting govorestat's ability to lower blood sorbitol levels and positively impact both functional and patient-reported outcomes. The company had previously indicated its intention to work with the FDA to determine the best path forward for govorestat, aiming for a 2025 filing.

Financial Considerations and Future Outlook

While the new study posting suggests potential progress, William Blair analysts also highlighted Applied Therapeutics' financial situation as a point of concern. As of June 30, the biotech reported $30.4 million in cash and cash equivalents, which analysts warn could pose "increased financial risk" given the company's "limited cash runway."

The rare genetic condition SORD, characterized by the accumulation of sorbitol sugars in nerves leading to sensory loss and muscle weakness, remains a critical area of unmet medical need. Govorestat's mechanism of action, which disables the aldose reductase enzyme to disrupt the sorbitol production pathway, represents a potentially innovative approach to treating this devastating disease.

As the pharmaceutical industry closely watches the development of govorestat, this case underscores the challenges and potential breakthroughs in bringing treatments for rare diseases to market. The coming months will be crucial in determining whether Applied Therapeutics can successfully navigate the regulatory landscape and advance govorestat towards potential approval.