Itamar Medical, an international medical technology company developing noninvasive diagnostic technology using the Peripheral Arterial Tone (PAT) Signal, announced today the use of the company's patented Endo-PAT2000 in BioMarin Pharmaceutical Inc.'s phase 2 study of patients with Sickle Cell Disease (SCD). The Endo-PAT2000 was used to measure endothelial function in a study designed to evaluate whether the drug 6R-BH4 can improve endothelial function in SCD patients.
Using measurements obtained from Endo-PAT tests, BioMarin announced Oct. 15 that the oral administration of 6R-BH4 is associated with an improvement of endothelial function in SCD patients. According to the study's key findings, Endo-PAT measurements showed that patients treated with escalating doses of 6R-BH4 showed improved endothelial function at weeks 8, 12 and 16, and that the improvement appeared to be dose dependent. Endo-PAT measurements indicate that patients with endothelial dysfunction at baseline, 56% of the study's patient population, improved to the normal range, with a demonstrated improvement at all dose levels.
Sickle Cell Disease is a chronic, debilitating, lifelong disease that affects a population of approximately 100,000 people in the U.S. In light of new medical research that has shifted focus away from the physical sickling of red blood cells towards the deteriorating health of blood vessels in SCD patients, BioMarin's trial evaluated changes in the physiological and biochemical markers of endothelial function, which underlie key aspects of Sickle Cell Disease.
In BioMarin's announcement, Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin stated: "The Endo-PAT device is a new reproducible method to measure the effect of endothelial dysfunction on small vessel function and has been adopted by the ongoing Framingham study of cardiovascular risks and outcomes. With this current SCD study, we have an indication that 6R-BH4 is active in improving endothelial function, and we hope to establish a clear relationship between the degree of endothelial dysfunction and the frequency and severity of sickle cell crises using a survey of SCD patients with the Endo-PAT test."