Applied Therapeutics, Inc. (Nasdaq: APLT) announced today that it will present comprehensive data from its INSPIRE Phase 3 trial of govorestat (AT-007) at the upcoming Peripheral Nerve Society (PNS) 2025 Annual Meeting in Edinburgh, Scotland. The presentation, scheduled for May 18, will feature full 12-month clinical results along with new topline data from the 18-month and 24-month timepoints.
The late-breaking abstract was selected for oral presentation, highlighting the significance of these extended results for patients with Sorbitol Dehydrogenase (SORD) Deficiency, a rare subtype of Charcot-Marie-Tooth (CMT) disease. This presentation comes before patients transition to the open-label extension phase of the study.
Dr. Evan Bailey, Senior Vice President of Clinical Development at Applied Therapeutics, will deliver the presentation titled "INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study."
The INSPIRE trial represents a critical advancement in addressing SORD Deficiency, which currently has no approved treatments. This double-blind, placebo-controlled registrational trial was designed to evaluate govorestat's efficacy in this patient population. Applied Therapeutics previously released topline results from the 12-month analysis in February 2024.
Understanding SORD Deficiency and the Treatment Approach
SORD Deficiency is characterized by progressive peripheral neuropathy resulting from mutations in the sorbitol dehydrogenase gene. This genetic defect leads to toxic accumulation of sorbitol in peripheral nerves, causing progressive muscle weakness, sensory loss, and disability.
Govorestat works as a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI), targeting the metabolic pathway that produces sorbitol. By inhibiting aldose reductase, govorestat aims to prevent sorbitol accumulation and potentially slow or halt disease progression.
"The extended data from the INSPIRE trial will provide valuable insights into the long-term efficacy and safety profile of govorestat in SORD Deficiency patients," noted a company spokesperson. "These results are particularly important as we evaluate the drug's potential to modify disease progression over extended treatment periods."
Regulatory Status and Broader Applications
Govorestat has garnered significant regulatory support, receiving Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and CMT disease. The U.S. Food and Drug Administration (FDA) has granted it Orphan Drug Designation for Galactosemia, phosphomannomutase 2 deficiency, and SORD Deficiency.
Additionally, the drug has received Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, as well as Fast Track designation for Galactosemia, underscoring its potential importance in treating these rare conditions.
Beyond SORD Deficiency, Applied Therapeutics is developing govorestat for multiple rare metabolic disorders, including Classic Galactosemia and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). This broad development program highlights the potential versatility of aldose reductase inhibition in addressing various rare diseases with limited treatment options.
Clinical Trial Design and Significance
The INSPIRE Phase 3 trial was meticulously designed to assess govorestat's impact on disease progression and symptom management in SORD Deficiency patients. The trial's extended duration—with data now available through 24 months—provides a more comprehensive understanding of the drug's long-term effects.
"Long-term data is particularly crucial in progressive neurological disorders like SORD Deficiency," explained an independent neurologist familiar with the condition. "Demonstrating sustained efficacy over two years would represent a significant advancement in our approach to treating this debilitating condition."
The presentation at the PNS Annual Meeting will also outline plans for future randomized studies, suggesting Applied Therapeutics' commitment to further developing govorestat and expanding the evidence base for its use in SORD Deficiency.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates for rare diseases with significant unmet medical needs. The company's approach centers on targeting validated molecular pathways with innovative therapies.
Govorestat represents the company's lead drug candidate and exemplifies its strategy of addressing rare metabolic disorders through targeted intervention in well-understood disease pathways. Applied Therapeutics continues to advance its pipeline of rare disease treatments, with govorestat at the forefront of these efforts.
As the company prepares to share these extended results at the PNS Annual Meeting, the rare disease community awaits potential new insights into this promising therapeutic approach for SORD Deficiency and other related conditions.
