The FoCus trial is a major milestone because it’s a large randomized control Phase III study in Wilson’s disease. The goal of this study was to evaluate the benefit of tiomolibdate choline, a novel albumin three-parted complex activator in development for Wilson’s disease. This new drug was compared to the standard of care in Wilson’s disease patients with neurologic symptoms...
The FoCus trial is a major milestone because it’s a large randomized control Phase III study in Wilson’s disease. The goal of this study was to evaluate the benefit of tiomolibdate choline, a novel albumin three-parted complex activator in development for Wilson’s disease. This new drug was compared to the standard of care in Wilson’s disease patients with neurologic symptoms. So 207 patients were randomized to receive either tiomolibdate choline or standard of care for 48 weeks, followed by an optional long-term extension of five years. And importantly, more than half of the patients had neurological symptoms at baseline. Another important strength of the study is that all neurological assessments using the UW-DRS Part III were retro-blinded, ensuring an objective evaluation of neurological outcomes. So at the European Academy of Neurology Congress, we specifically analyzed the subgroup of patients with neurological Wilson’s disease because this is where the greatest unmet medical need remains. As I told you, these patients are at risk of paradoxical neurological worsening after treatment initiation, and neurological recovery is often incomplete despite long-term therapy. So concerning the results, what impressed me most was the remarkable consistency of the findings. Across neurological, psychiatric, hepatic, and global clinical outcomes, tiomolibdate choline performed as well as or better than standard of care. Global clinical improvement assessed by the clinical global impression scale was significantly greater with tiomolibdate choline. When we focused on neurological function using the UW-DRS Part III, patients treated with tiomolibdate choline continued to improve throughout the 48-week study, where improvements essentially plateaued in the standard of care group. Importantly, this finding was highly consistent. Whether we define neurological improvement using the minimal clinically important difference or more demanding improvement thresholds, a greater proportion of patients improved with tiomolibdate choline than with standard of care. To me, this is the most important finding. The benefit was observed consistently across multiple clinical relevant neurological endpoints, making it unlikely that the results were driven by a single statistical analysis. And finally, the overall safety profile remained favorable across the clinical development program, with more than 645 patients’ years of exposure and a very low incidence of treatment-related serious neurological or psychotic adverse events. Overall, these findings transcend the evidence that tiomolibdate choline could become an important therapeutic option for patients with neurological recent disease, a population in whom new treatment options have been needed.
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