Neurotrope, Inc. announced that its wholly-owned subsidiary, Neurotrope Bioscience, Inc., will conduct a preclinical study, in collaboration with a leading research institution, to examine the effect of bryostatin in Niemann-Pick type C (NPC) mice.
The study is being funded by several family foundations under the auspices of Support Of Accelerated Research for NPC Disease (SOAR-NPC).
This planned study will examine the effects of various dosing regimens of bryostatin in NPC mice over a brief treatment period. Specifically, the in vivo study will seek to confirm previous in vitro studies that suggest bryostatin may be effective in correcting the cholesterol transport defect in NPC. Neurotrope expects the results of the study to be available in the third quarter of 2015.
“We welcome and appreciate our collaboration with SOAR and view this as a significant step forward in Neurotrope’s Orphan drug programme. This study is designed to confirm, in mice, the results of recent NPC cellular studies. Reducing cholesterol accumulation is believed critical for the successful development of an effective therapy for NPC. We are very encouraged by results of the cellular study that suggest treatment with bryostatin can improve the cholesterol transport defect in NPC. We look forward to the results of this NPC mouse study in the coming months as we continue to work diligently to further the development of bryostatin as a therapeutic compound to treat this debilitating disease,” stated Charles S. Ramat, Neurotrope’ s president and chief executive officer
“This study is designed to examine the ability of bryostatin treatment to remove cholesterol from the brain which is critical for an effective therapy of NPC,” stated Dr. Warren W. Wasiewski, Neurotrope’s chief medical officer.
SOAR-NPC was created in 2007 to accelerate therapy development for one of the most devastating lysosomal diseases, Niemann Pick type C. Over the past five years, SOAR has made significant progress in developing a drug pipeline for NPC therapeutics and in the discovery of disease biomarkers to facilitate clinical testing of these drugs.
NPC mainly affects children who develop severe neurologic symptoms including gait disturbance and cognitive deficits early in life. There are approximately 3,500 patients in the US and a similar number in Europe. Patients with NPC have a gene defect that results in the loss of the normal NPC1 or NPC2 protein that is important for cholesterol trafficking. As a result of the gene defect, there is accumulation of cholesterol, and a number of other lipids such as sphingolipids, and gangliosides in various tissues, including the brain. Rates of disease progression and life expectancy vary widely; however, the majority of patients die between 10 and 25 years of age. Progressive neurological manifestations in NPC have a profound effect on quality of life for both the patients and their caregivers.
Neurotrope Bioscience Inc., the operating subsidiary of Neurotrope, Inc., was formed in October 2012 principally to license, develop and commercialize various novel therapeutic and diagnostic technologies from the Blanchette Rockefeller Neurosciences Institute (BRNI) which are focused on the development of conventional small molecules that are extraordinarily potent in the activation of the enzyme Protein Kinase C Epsilon (PKCe).