Rostafuroxin – In development for genetically associated hypertension

Hypertension is a very large market with high unmet need. Over one-third of the adult population in the U.S. has hypertension and the incidence is increasing. (1) Hypertension is a highly heterogeneous disease and it is well known that ethnicity can impact response to different classes of agents. The majority of treated patients (50-85 percent globally) do not reach their therapeutic target for blood pressure control thus there remains substantial unmet medical need in the treatment of hypertension. Having high blood pressure puts you at risk for heart disease and stroke, which are leading causes of death in the United States. (2)


Rostafuroxin is a novel, phase 2 precision medicine targeting hypertensive patients with certain genetic profiles focused on the large and important resistant hypertension market. Endogenous ouabain pathways and adducin polymorphisms are known triggers of hypertension and approximately 20-25 percent of population have this appropriate genetic profile. Rostafuroxin is designed to be a potent and selective antagonist of ouabain and of the mutant adducin molecules and creates functional effects by enhancing renal tubular sodium reabsorption and increasing vascular tone thus offering the possibility to reduce or normalize blood pressure in a genetically identified subset of patients.

There have been two phase 2 studies completed with rostafuroxin. In a phase 2a trial, rostafuroxin demonstrated efficacy by reducing blood pressure in a European population in the select mutations. In the phase 2b study, the Company examined the antihypertensive effect of different doses of rostafuroxin in comparison with Losartan, assessed by office and ambulatory blood pressure monitoring in a hypertensive population selected according to a specific genetic profile. The phase 2b was conducted as a two-part study with the first part, conducted in Italy in 160 Caucasian patients and the second part conducted in 120 Chinese patients in Taiwan. Efficacy results for the phase 2b in the EU were positive and were similar to the phase 2a study. While the BP reduction in Caucasians was notable in both the 2a and 2b trials, blood pressure response in Chinese patients was notably less pronounced. The Company will be examining the potential reasons for the different response in Chinese patients, including differences between the populations in drug metabolism, oral bioavailability or drug Interaction with traditional Chinese medicines the patients may have been taking.

Rostafuroxin has the potential to be an important novel target therapy for patients with the appropriate genetic profile. The company is focused on finalizing an enhanced formulation, analytic measures and assessments and then plans to conduct business development to out-license rostafuroxin to a larger pharmaceutical company that has expertise and infrastructure in the very large and broad antihypertension market.


1. Centers for Disease Control and Prevention, National Center for Health Statistics. Underlying Cause of Death 1999-2013 on CDC WONDER Online Database, released 2015. Data are from the Multiple Cause of Death Files, 1999-2013, as compiled from data provided by the 57 vital statistics jurisdictions through the Vital Statistics Cooperative Program: Accessed on Feb 3, 2015.

2. Farley TA, Dalal MA, Mostashari F, Frieden TR. Deaths preventable in the U.S. by improvements in the use of clinical preventive services. Am J Prev Med. 2010;38(6):600–9.