Mallinckrodt Niemann-Pick disease therapy fails pivotal trial

Mallinckrodt plc (NYSE:MNK) said VTS-270 showed no significant separation from placebo in a Phase IIb/III trial to treat Niemann-Pick disease type C1 (NPC1). Mallinckrodt CSO and EVP Steven Romano disclosed the result on a conference call Tuesday to discuss the company's 3Q18 earnings.

The double-blind, international trial enrolled 51 patients with neurologic manifestations of NPC1 and evaluated the change from baseline to week 52 in NPC Clinical Severity Score as its primary endpoint. On the call, Romano said patients in both treatment arms unexpectedly showed no disease progression over the 52-week trial and subsequently no benefit could be observed. He said Mallinckrodt will continue to review the data and work with FDA to determine next steps...