NEW YORK - Applied Therapeutics, Inc. (NASDAQ:APLT) reported positive interim results from its Phase 3 INSPIRE trial for govorestat, indicating significant benefits in treating SORD Deficiency, a rare hereditary neuropathy. The trial met its primary endpoints, including a statistically significant correlation between sorbitol levels and clinical outcomes, and a significant reduction in sorbitol with govorestat treatment compared to placebo.
The 12-month interim analysis revealed a notable correlation between sorbitol levels and the composite clinical outcome measures (p=0.05), as well as a sustained reduction in sorbitol levels in patients treated with govorestat over a 12-month period (p<0.001). Additionally, the treatment demonstrated a highly significant effect on the CMT Health Index (CMT-HI), a patient-reported outcome measure, with improvements noted in lower limb function, mobility, fatigue, pain, sensory function, and upper limb function (p=0.01).
Govorestat, an Aldose Reductase Inhibitor, has been shown to reduce sorbitol levels in patients with SORD Deficiency, a condition characterized by mutations in the Sorbitol Dehydrogenase gene leading to toxic sorbitol accumulation. This accumulation results in motor neuron degeneration and loss of mobility and motility.
The safety profile of govorestat was comparable to the placebo, with a similar incidence of adverse events reported between the active and placebo-treated groups. The results support sorbitol as a key factor in disease severity and progression.
Based on the interim data, Applied Therapeutics plans to request a pre-NDA meeting with the neurology division of the US FDA to discuss potential approval. The company's commitment to addressing rare diseases with no existing treatments is underscored by the potential of govorestat to slow disease progression and improve patient well-being.
The final clinical outcomes of the INSPIRE trial will be assessed at 24 months, with the 10-meter walk run test as the primary clinical efficacy endpoint. Applied Therapeutics will host a conference call and webcast to discuss the interim analysis and the treatment's prospects for SORD Deficiency.
This article is based on a press release statement from Applied Therapeutics.
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