RXC007 (ROCK2 selective inhibitor)

Selective ROCK2 inhibitor for the treatment of interstitial lung diseases with an initial study in idiopathic pulmonary fibrosis

Phase 2a clinical study commenced October 2022, topline data expected Q1 2024





Phase 1

Phase 2

Phase 3

Upcoming Milestones


Selective Inhibitor

Idiopathic pulmonary fibrosis (IPF)

Phase 2a topline data – Q1 2024

RXC007 is a potent, highly selective and orally-active inhibitor that targets Rho-Associated Coiled-Coil Containing Protein Kinase 2 (ROCK2) which sits at a nodal point in cell signalling pathways, central to fibrosis. ROCK2 is therefore an important emerging drug target and RXC007 has the potential to treat several fibrotic diseases where ROCK2 is upregulated. These include the orphan disease idiopathic pulmonary fibrosis (IPF), a severe and life-threatening chronic lung condition with a very poor prognosis, similar to many cancers, and limited treatment options. With a median survival 3-5 years from diagnosis and over 170,000 patients in the US, EU5 and Japan alone, IPF is being targeted as the first indication for clinical development.

Our ROCK2 inhibitor, RXC007, has demonstrated potential preclinically to be an attractive agent for the treatment of fibrotic indications.

RXC007 has been shown to be safe and well tolerated in a Phase 1 clinical study in healthy volunteers and is now being progressed in a Phase 2a clinical study in patients with IPF.

Additionally, RXC007 has shown preclinical proof-of-concept in immune mediated models, which supports the development of RXC007 into other interstitial lung diseases beyond IPF.

Our Pipeline


Selective Inhibitor

Phase 2

Idiopathic pulmonary fibrosis (IPF)


Phase 2a topline data – Q1 2024

What is Fibrosis?

Fibrosis is an internal scarring process, which can occur in response to injury, where excess connective tissue is deposited in an organ or tissue – thereby impairing its function. Most chronic inflammatory diseases will result in fibrosis, with progressive injury resulting in organ failure. Fibrotic disease can occur in nearly any tissue in the body and contributes to 45% of deaths in the developed world. Solid organ (such as lung, liver or kidney) fibrosis can occur as a result of many different diseases and underlying health issues, including obesity or diabetes. Current therapeutic options are limited for these chronic and often life-threatening diseases.

Why target ROCK2 (Rho-associated protein kinase 2) for fibrotic diseases?

ROCK2 is an intracellular kinase with multiple cellular functions. ROCK2 signalling plays a key role in both the inflammatory component and the tissue remodelling that drives disease progression in many fibrotic conditions. ROCK2 expression and activity have been shown to be upregulated in acute inflammatory injury and in chronic diseases such as diabetes, metabolic syndrome and IPF.
ROCK2 has been shown to play important roles in the pro-fibrotic response of lung epithelial cells in IPF. Furthermore, ROCK2 activity is also upregulated in liver, lung and kidney models of fibrosis and has been shown to modulate activation of the hepatic stellate cells (central drivers of fibrosis in the liver) and mesangial cells (important drivers of fibrosis in the kidney). Selective inhibition of ROCK2 has the potential to prevent the hypotensive side effects typically associated with systemic pan-ROCK inhibitors (where both ROCK1 and ROCK2 are inhibited). Targeting ROCK2 has been clinically validated by other compounds currently in development, including KD025, a ROCK2 inhibitor recently approved by the FDA for chronic Graft vs Host Disease (cGvHD) now being developed by Sanofi following its acquisition of Kadmon in August 2021.

ROCK is a compelling, nodal target for fibrotic diseases. The role of ROCK2 in a diverse range of cellular processes allows RXC007 to have pleiotropic effects, affecting multiple cell signalling pathways associated with fibrosis.

ROCK2 sits at a nodal point in a cell signalling pathway, believed to be central to fibrosis

Figure 1: The ROCK signalling pathway

RXC007 demonstrates compelling efficacy in well-validated preclinical model of lung fibrosis

Figure 2: Therapeutic Bleomycin-induced Lung Mouse Model

RXC007 reduces fibrosis and collagen deposition in the lung and bronchoalveolar lavage fluid (BALF) in murine bleomycin-induced idiopathic pulmonary fibrosis (IPF) model with therapeutic dosing.

Programme summary

RXC007 (ROCK2 selective)


ROCK2 selective inhibitor (RXC007) for treatment of fibrosis


Phase 2a study


Idiopathic Pulmonary Fibrosis (IPF)
Interstitial Lung Disease (ILD)

Next Milestone

Q1 2024

Phase 2a topline data