Studies Show Triolex for Parkinson's Disease Crosses the BBB in Mice

Harbor BioSciences, Inc., which is investigating the use the company’s proprietary compound Triolex as a treatment for Parkinson’s disease (PD) with funding from The Michael J. Fox Foundation (MJFF), recently announced positive results in initial preclinical studies. These studies in orally treated mice demonstrate that Triolex crosses the blood-brain barrier in significant quantities.

Harbor BioSciences designed these studies to determine if Triolex, an anti-inflammatory, small-molecule compound believed to reduce inflammation in the brain, was capable of crossing the blood-brain barrier. The results described here show that more than 50% of levels of Triolex measured in plasma were found in mouse brains after oral administration. These positive findings support rodent efficacy studies, which are expected to be completed before the end of this year.

The terms of the collaboration call for MJFF to fund up to approximately $150,000 toward preclinical development of Triolex in rodents. If these studies are successful, additional funding may be awarded by MJFF to continue the clinical development of Triolex for the treatment of PD.

“Anti-inflammatory approaches to PD are of increasing interest to Parkinson’s researchers, but even the most effective potential treatment must cross the blood-brain barrier in order to be translated into practical treatments for PD patients,” said Todd Sherer, PhD, Chief Program Officer, The Michael J. Fox Foundation. “We are encouraged by the initial data showing that Triolex may accomplish both of these goals, and look forward to partnering with Harbor BioSciences to continue vetting this novel compound in the hope of speeding its path to the clinic.”

Harbor BioSciences believes Triolex may decrease chronic inflammation in the brain, thereby protecting neurons whose loss would otherwise lead to the damage associated with PD. This belief is based on several factors: the company’s previously reported data regarding the anti-inflammatory effects of Triolex in human clinical studies; attenuation of neuroinflammation in rodent models; and the extensive literature describing the benefits of related natural compounds in both humans and animal PD models. To date, Triolex has been well tolerated when administered to more than 180 people with type 2 diabetes and healthy volunteers.

“It is a privilege to be collaborating with MJFF to assess the potential benefits of Triolex in patients suffering from PD,” commented James M. Frincke, Harbor BioSciences’ Chief Executive Officer. “If Triolex demonstrates an effect on regulating key inflammatory cytokines in PD patients similar to that observed in animal models and in type 2 diabetes patients, it should provide an entirely new approach to the treatment of this debilitating disease. We are also hopeful that if Triolex is able to reduce neuro-inflammation and protect neurons in PD, it may also be useful in other inflammation-driven neurodegenerative disorders, such as Alzheimer’s disease and multiple sclerosis.”

Harbor BioSciences is a development-stage company with two product candidates that recently completed Phase I/IIa clinical trials: Apoptone (HE3235) in patients with late-stage prostate cancer, and Triolex (HE3286) in obese type 2 diabetes mellitus patients. Apoptone and Triolex represent two of the lead candidates from Harbor BioSciences’ small molecule platform based on metabolites or synthetic analogs of endogenous human steroids.