Takeda Pharmaceuticals announced that a preliminary look at the results of their recently completed phase-IIb trial of their TAK-611 ERT (enzyme replacement therapy) for MLD “topline results … did not meet primary and secondary endpoints.” The company is analyzing the results and continues to list TAK-611 in its pipeline according to statements made during this week’s earnings call.

MLD families in the trial will continue to receive infusions as part of the extension period of the trial while the rest of the data is analyzed and formal decisions are made and announced, perhaps later this year.

Remember that five different study groups are part of this trial, entering with varying levels of disease progression and ages.  

The formal analysis of this data will take some time.  The company did not formally announce what slice(s) of data were used to draw these initial conclusions.

MLD Foundation has been working with ERT researchers since 2005.  ERT is successfully used to treat other lysosomal disorders.