Breaking News for SORD Deficiency Patients

Therapeutic Research In Accelerated Discovery (TRIAD) partner Applied Therapeutics announces today positive results from the 12-month interim analysis of Govorestat (AT-007) in the ongoing INSPIRE Phase 3 Trial in Sorbitol Dehydrogenase (SORD) Deficiency.

The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.

The objective of this pre-specified, 12-month interim analysis was to evaluate early indicators of govorestat treatment effect in order to inform future regulatory discussions and support a potential New Drug Application (NDA) submission, due to the urgent need for treatment and absence of any other options for patients with SORD Deficiency.

The 12-month interim analysis was comprised of a clinical efficacy primary endpoint based on correlation of sorbitol with composite clinical outcome measures, and a pharmacodynamic (PD) biomarker primary endpoint based on sorbitol reduction.

Interim Analysis Results:

  • Demonstrated statistically significant correlation between sorbitol level and the prespecified CMT-FOM composite clinical endpoint (10-meter walk-run test, 4 stair climb, sit to stand test,6-minute walk test and dorsiflexion) (p=0.05).
  • Govorestat treatment provided sustained reduction in sorbitol level in patients with SORD Deficiency over 12 months of treatment, which was statistically significant compared to placebo (p<0.001).
  • Govorestat treatment also resulted in a highly statistically significant effect (p=0.01) on the CMT Health Index (CMT-HI), an important patient-reported outcome measure of disease severity and well-being, which was a secondary endpoint in the study. Aspects of the CMT-HI that demonstrated a treatment effect included lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.
  • Govorestat was safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups.

HNF has been a proud partner in the patient recruitment of this trial through GRIN, educational webinars, focus groups, social media campaigns and dedicated website content. Our team continues to support Applied Therapeutics in increasing awareness of SORD Deficiency amongst the patient community and the importance of considering genetic testing for those patients suspected to have SORD Deficiency.

For more info on patient-initiated genetic testing, visit HNF’s CMT Genie 

To participate in CMT research, enroll in HNF’s Global Registry for Inherited Neuropathies, GRIN here.

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