UK-based Mallinckrodt has revealed that its experimental therapy for Niemann-Pick Type C failed to hit targets in a recently completed registration trial.

The firm did not reveal any specific data, but said that VTS-270 did not show a statistically significant separation from placebo.

However, as US regulators indicated to Mallinckrodt at a meeting back in August that the drug’s potential approvability will be based on the totality of data, not a single study or endpoint, the firm’s review of data from the Phase IIb/III trial, including the longer term open label portion, "continues to proceed and is being assessed in combination with several other available data sources."

"A better understanding of the potential benefit of VTS-270 will emerge as the company carefully considers the totality of data available and continues to work with the primary investigators and the FDA to determine the best path forward," the firm said in an SEC filing.

Mallinckrodt acquired VTS-270 for around $1.2 billion via its purchase of Sucampo in January this year.

NPC is a progressive, irreversible, chronically debilitating - and ultimately lethal - genetic disease, caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen.

Current treatment is focused on managing specific symptoms of the condition in each individual, as well as preventing secondary complications.

VTS-270, a mixture of 2-hydroxypropyl-B-cyclodextrin, targets cholesterol and sphingolipid storage. The firm says preliminary studies have shown that it can promote transport of the cholesterol that would normally accumulate in the lysosomes of cells carrying the mutation responsible for the disease, restoring normal cholesterol metabolism and regulation.