Positive Interim Govorestat Results in SORD Deficiency Phase 3 Trial

Applied Therapeutics, a biopharmaceutical firm, has reported promising interim outcomes from its Phase 3 study, INSPIRE, which is assessing the efficacy of the oral medication govorestat for patients with SORD Deficiency, a rare genetic disorder that leads to progressive motor neuron degeneration. The study has met its primary and several secondary endpoints, indicating a significant reduction in sorbitol levels, the toxic sugar associated with the disease, in patients treated with govorestat compared to those on a placebo.

The 12-month analysis revealed a substantial correlation between reduced sorbitol levels and improvements in clinical outcomes, as measured by the CMT-FOM composite endpoint, which includes various physical performance tests. The drug also showed a positive impact on the CMT Health Index, a patient-reported measure of disease severity and quality of life, with significant benefits observed in areas such as lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.

Govorestat, an Aldose Reductase Inhibitor, works by inhibiting the conversion of glucose to sorbitol, thereby addressing the root cause of SORD Deficiency. The drug has been well-tolerated with a safety profile comparable to that of the placebo group. The company is encouraged by these results, which support the role of sorbitol as a key factor in disease progression and are indicative of the potential of govorestat to slow down the disease's advance.

Shoshana Shendelman, CEO of Applied Therapeutics, emphasized the company's dedication to developing innovative treatments for rare diseases where no options currently exist. The company is planning to engage with the U.S. FDA to discuss the possibility of drug approval based on the positive data obtained so far. The ongoing INSPIRE trial will continue to assess clinical outcomes at the 24-month mark, with the 10-meter walk run test as the primary endpoint.

Riccardo Perfetti, the Chief Medical Officer, expressed enthusiasm about the drug's effectiveness in reducing sorbitol levels and improving functional measures and patient-reported outcomes. The company is eager to work with regulatory bodies to expedite the approval process and make this treatment available to patients.

Dr. Michael Shy, the lead investigator of the INSPIRE trial and a specialist in neuromuscular medicine, applauded the significant impact of govorestat on patient-reported outcomes and its potential to address the unmet needs of individuals with SORD Deficiency.

Applied Therapeutics is also hosting an investor conference call to discuss these interim results in detail. The company is committed to advancing its pipeline of novel drug candidates targeting high-unmet medical needs, with govorestat being a key asset in the treatment of rare CNS metabolic diseases.