Rock Creek Pharmaceuticals Reports Human Proof-of-Principle Study

Rock Creek Pharmaceuticals, Inc., recently announced the results of a human proof-of- principle study with anatabine citrate, the company’s lead compound. Results from the study show that a single oral dose of anatabine citrate can significantly inhibit the activation of inflammatory proteins in the blood of human subjects. The company is developing this compound in a Phase I clinical trial under a Medicines and Healthcare Products Regulatory Agency (MHRA) regulatory protocol in the United Kingdom.

In December 2014, the company received a New Zealand Ministry of Health Ethics Committee approval to conduct a single-site, single dose, open-label human study, entitled Determination of the Blood Pharmacodynamic Effects following a Single Dose of Oral Anatabine Citrate in Normal, Healthy Volunteers.

The primary objective of the study was to determine whether a single dose of orally administered anatabine citrate could reduce the activation of inflammatory proteins in human white blood cells. Blood samples were taken from 10 healthy human subjects, collected before and after oral administration of anatabine citrate. The blood samples were immediately challenged with lipopolysaccharide (LPS), a well-known potent stimulator of inflammation. The blood samples were then evaluated to measure the activation of two proteins, NF-kB and STAT3, which are known to be central to inflammation. The study compared the amount of LPS-induced activation of NF-kB and STAT3 in the white blood cells before and after the administration of anatabine citrate. Activated NF-kB and STAT3 protein levels were measured by two different, well-established molecular biology methods.

Data analysis showed that there were statistically significant (p<0.05) reductions in LPS stimulated levels of both NF-kB and STAT3 in human white blood cells after a single oral dose of anatabine citrate. These data are consistent with several peer reviewed scientific papers showing that anatabine can reduce NF-kB and STAT3 activation in animal models of inflammatory diseases, as well as in several human cell lines as shown by in vitro studies. Additionally, evaluation of safety data showed that anatabine citrate was well tolerated by the study participants, and there were no safety concerns.

“This is the first time that anatabine citrate taken orally by human subjects has been shown to oppose the activation of these known inflammatory proteins in white blood cells. The data are entirely consistent with our proposed mechanism of action of anatabine citrate which forms the basis for our advancement of this compound into clinical trials for human inflammatory diseases,” said Ryan Lanier, PhD, Rock Creek Pharmaceuticals’ Chief Scientific Officer.

Rock Creek Pharmaceuticals’ Anatabine Citrate is a small molecule, cholinergic agonist that exhibits anti-inflammatory pharmacological characteristics, distinct from other anti-inflammatory drugs available, such as biologics, steroids, and non-steroidal anti-inflammatories. The company has sponsored extensive preclinical (in vitro and in vivo) studies resulting in peer reviewed and published scientific journal articles, covering models of Multiple Sclerosis, Alzheimer’s Disease, and Auto-Immune Thyroiditis. All these studies demonstrated the anti-inflammatory effects of Anatabine Citrate. In addition, the company’s compilation of human exposure, safety, and tolerability data, derived primarily from human clinical studies and post-marketing data collection of the previously marketed nutraceutical product, has provided important insights for clinical development.

Rock Creek Pharmaceuticals, Inc. is an emerging drug development company focused on the discovery, development, and commercialization of new drugs, formulations, and compounds that provide therapies for chronic inflammatory disease, neurologic disorders, and behavioral health. For more information, visit www.rockcreekpharmaceuticals.com.