A star drug in Mallinckrodt’s $1.2B Sucampo buyout flops in pivotal trial

Some of the insiders at Mallinckrodt $MNK may be wondering today why they paid $1.2 billion for Sucampo just 10 months ago. One of the two late-stage drugs they bagged in the deal has failed the registration study for often fatal cases of Niemann-Pick type C.

Billed as a relatively economical approach to building their late-stage pipeline, Mallinckrodt — which was drummed out of PhRMA after becoming a punching bag for critics due to its pricing strategy — told investors Tuesday that VTS-270 flopped in the registration study.

Steven Romano

“In our recently completed registration trial, the product did not show a statistically significant separation from placebo,” noted Steven Romano, the company’s chief scientific officer.

But they are not writing it off. At least not yet.

Though their drug failed to outpace the placebo, Mallinckrodt says that they also didn’t see the expected decline in patients in the placebo group, giving them hope that there’s still something here worth keeping alive. That may be a lengthy stretch, but Romano insists they could salvage this one yet.

At a meeting in August, the FDA indicated to us that their view on the potential approvability of VTS-270 will be based on the totality of data, not a single study or endpoint. And in the coming months we will continue to work with the primary investigators and the FDA to clarify a potential path forward.

We understand the importance of pursuing this potential treatment for Niemann-Pick type C and based on our current assessment of the safety data, at this time, we believe continued treatment with VTS-270 in the ongoing open label portion of the trial is acceptable. Patients, their families, and our patient group partners should know that we remain deeply committed to this work.

The drug — a unique mixture of 2-hydroxypropyl-ß- cyclodextrins (HPßCD), which registered positive data from a tiny study with 14 patients — was originally from Vtesse, which Sucampo acquired in its $200 million buyout earlier in 2017.

The company’s shares nevertheless popped up about 10% by the end of the day, a reflection of investors’ appreciation for their Q3 numbers, which exceeded expectations.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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