Investigators have outlined the results of a small, single-arm study of a new drug for Niemann-Pick type C disease which inspired a $200 million biotech buyout earlier this year.
The drug is cyclodextrin, or at least a unique mixture of 2-hydroxypropyl-ß- cyclodextrins (HPßCD) that Vtesse had moved into a pivotal study after gleaning positive data from a study with 14 patients. Sucampo bagged the biotech and its drug for $200 million in April, taking over control of the Phase III.
According to Daniel Ory of Washington University School of Medicine in St Louis — the first author of a new paper published in The Lancet — researchers recruited 14 NPC patients, who typically die of the disease 10 to 15 years after it’s diagnosed.
Delivered in the spinal column every two weeks for 18 months, the symptoms of 7 of the 14 patients improved, with some regaining an ability to speak as researchers tracked scores on gait, cognition and speech.
To be sure, this is no cure. The researchers say that the scores of patients for the point system they used worsened by 1.2 per year on average. But that compares to the historical data they used from an earlier study, as there was no placebo arm, with an expected 2.9 point decline. And investigators say that they tracked hearing loss as a serious adverse event in the study.
“Some of the patients began this trial without the ability to speak, and now they speak,” Ory said. “There is a slowing of the decline, but we were surprised to see trends toward improvement in a few categories. Compared with the historical data, half of the patients in this study saw an improvement or no worsening in the neurological severity score.”
Added Ory: “A therapy that causes hearing loss is not ideal. But since the disease itself causes hearing loss, we felt that this side effect may be a reasonable trade-off, given the alternative decline and death that the disease also causes.”
Researchers at the NIH’s National Institute of Child Health and Human Development designed and conducted the study. And they say that they were also able to track a positive influence on several key biomarkers for the disease, which is caused by the buildup of cholesterol in brain cells.
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