Mallinckrodt's Niemann-Pick C-type drug failed to meet test targets

UK-based Mallinckrodt has revealed that its experimental treatment for the Niemann-Pick C model failed to meet its targets in a recently completed registration trial.
the company did not disclose any specific data, but said there was no statistically significant difference between the VTS-270 and the placebo.
However, as U.S. regulators told Mallinckrodt at a meeting in August that the potential approval of the drug would be based on the overall data rather than a single study or endpoint, the company's review of IIb/III trial data, including the long-term open label section, "continues and is evaluated with several other available data sources." "
" will better understand the potential benefits of VTS-270 as the company carefully considers all available data and continues to work with key researchers and the FDA to determine the best way forward. The company said in SEC filings.
Mallinckrodt bought the VTS-270 in January for about $1.2 billion.
NPC is a progressive, irreversible and chronically debilitating genetic disease caused by defects in in-cell lipid transport, leading to excessive accumulation of lipids in the brain and liver and spleen.
current treatment focuses on managing the specific symptoms of each individual's condition and preventing secondary complications.
VTS-270 is a mixture of 2-hydroxypropyl-B-cyclodext, which targets cholesterol and lipid storage. The company says preliminary studies have shown that it can promote the transport of cholesterol, which usually accumulates in lysosomes that carry cells with mutations in the disease, returning to normal cholesterol metabolism and regulation. (This web article)