Mallinckrodt's VTS-270 flop puts Acthar back into focus

Anyone wondering what had happened to Mallinckrodt’s VTS-270 in Niemann-Pick C disease finally got their answer yesterday: its pivotal trial flopped. The company had been due to report results mid-year, but snuck out the news alongside third-quarter results. Mallinckrodt does not appear to have given up on VTS-270, which it gained through the $1.2bn acquisition of Sucampo: the company pointed to an unexpected lack of disease progression in the placebo arm of the study, adding that it would meet the FDA to see whether there was a path forward for the project. There is no cure for Niemann-Pick C disease, a rare genetic condition that leads to neurodegeneration and death. Assuming VTS-270 is a dud, hope now rests with Orphazyme’s arimoclomol citrate, due to yield data from a phase II/III trial in 2020. Meanwhile, it is a good job for Mallinckrodt that sales of its blockbuster, H.P. Acthar Gel, are declining more slowly than expected – and data are due next year in yet another indication, amyotrophic lateral sclerosis. But the VTS-270 failure makes Mallinckrodt’s pipeline look even less likely to make up for future Acthar losses.

Niemann-Pick disease clinical pipeline (all phase III)
Project Company Pharma class Subtype Trial Primary completion
VTS-270 Mallinckrodt Cholesterol modulator Niemann-Pick C NCT02534844 Failed
Arimoclomol citrate Orphazyme Superoxide dismutase 1 chaperone Niemann-Pick C NCT02612129 Oct 2020
Olipudase alfa Sanofi Human recombinant acid sphingomyelinase Niemann-Pick A/B  Ascend (NCT02004691) Jan 2024
Source: EvaluatePharma, clinicaltrials.gov.

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