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ROC-101 In The Clinic

In a recent Phase 1 clinical trial, AllRock Bio demonstrated that ROC-101 daily dosing achieved ROC-101 plasma exposures that are predicted to inhibit both ROCK1 and ROCK2 isoforms, and to inhibit ROCK activity and ROCK-dependent inflammatory cytokine secretion by PBMCs. Levels of ROC-101 in plasma met or substantially exceeded modelling and simulation of levels of a ROCK1&2 inhibitor successfully used to treat PH/PAH in academic studies in Asia (Jiang 2015). No pan-ROCK inhibitor is currently approved in the United States for the treatment of PH or PAH.

The ROC-101 mechanism of action targets both ROCK1 and ROCK2, resulting in a treatment for PAH and ILD-PH that is anti-proliferative, anti-inflammatory, and anti-fibrotic. This mechanism beneficially targets the pathologic process of pulmonary arterial remodeling and pulmonary fibrosis. ROC-101 builds on the approved pan-ROCK inhibitor in Japan, and was shown in investigator led trials, to reduce pulmonary arterial pressure and pulmonary vascular resistance, a 22% PVR reduction after a single IV dose (Xiao 2015).


ROC-101 is an oral, bioavailable, small molecule that exhibits highly potent and selective drug-like properties. Pre-clinical data shows that ROC-101 has increased selectivity over fasudil, another pan-ROCK inhibitor approved in Japan for subarachnoid hemorrhage.

Group 1 PH, or PAH, is a complex disease characterized by narrowing, remodeling, and vasoconstriction of the pulmonary arteries, which leads to increased pulmonary vascular resistance and elevated pulmonary arterial pressure. The pathophysiology of PAH involves various mechanisms including endothelial and vascular smooth muscle cell dysfunction with accompanying impaired vasodilation, smooth muscle cell proliferation that narrows the pulmonary artery lumen, inflammation, and vascular remodeling (Abe 2004).



Mortality in PAH remains high despite the use of combinations of approved treatments.

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