Mallinckrodt's rare disease drug set back by confusing trial result
- Mallinckrodt executives revealed on a third quarter earnings call Tuesday that the company's drug candidate for a rare neurodegenerative disease called Niemann-Pick type C failed to beat out placebo in a Phase 2/3 trial.
- The pharma tested VTS-270 against placebo over 52 weeks. Given the degenerative nature of the disease, investigators had expected both trial arms to worsen, with slower disease progression rates in the active group. Instead, neither group showed disease progression, yielding the negative result.
- Treatment will continue in the ongoing open-label portion of the study, while Mallinckrodt says it will work with the Food and Drug Administration to find a path forward for the drug.
Mallinckrodt added VTS-270 to its pipeline when it bought Sucampo Pharmaceuticals last year for $1.2 billion. Mallinckrodt also picked up the gastrointestinal drug Amitiza (lubiprostone) in the deal, which it hoped would prop up its revenue as sales of H.P. Acthar Gel (repository corticotropin injection) have weakened recently.
Now, VTS-270 is facing a significant development challenge following the trial miss.
The company did not disclose specific data from the study, such as the statistical comparisons which would reveal how the drug performed versus placebo.
Mallinckrodt put forward a hypothesis that differences in the population studied, which included patients between 4 and 20 years of age, could have led to the confusing result.
"We'll look at things like severity of disease, age of the patients at the time of onset of the disease, the extent of their neurological condition which is a consequence of the disease as you well know," Steve Romano, chief science officer at Mallinckrodt, said on the call.
A rare inherited lysosomal storage disorder, Niemann-Pick type C generally presents in childhood, but there are cases of adolescent or adult onset. It affects just 500 or 600 people across the U.S., and the disease is ultimately fatal.
Most treatments are symptomatic, though Actelion's type 1 Gaucher disease drug Zavesca (miglustat) used off-label may slow progression. Zavesca has been approved in Europe, Canada and Japan for Niemann-Pick type C but was rejected by the Food and Drug Administration in 2010.
Despite the ultra-rare nature of the disease, VTS-270 had been pegged for peak sales potential of more than $300 million, Raymond James analyst Elliot Wilbur wrote in a Nov. 6 note to investors. He remained wary, however, on Mallinckrodt finding a path forward for the drug.
"While the message was that the potential FDA approval will be based on the totality of data according to the agency, [this] development represents another setback on the pipeline front," Wilbur added.
Follow Suzanne Elvidge on Twitter