Kadmon Presents Preclinical Data Supporting the Therapeutic Potential of ROCK Inhibition in Pulmonary Fibrosis

March 28, 2017

Kadmon Presents Preclinical Data Supporting the Therapeutic Potential of ROCK Inhibition in Pulmonary Fibrosis

-- Data Validate the Role of ROCK Signaling in Fibrotic Disease --

NEW YORK--(BUSINESS WIRE)-- Kadmon Holdings, Inc. (NYSE:KDMN) (“Kadmon”) today announced preclinical data demonstrating the importance of the Rho-associated coiled-coil kinase (ROCK) signaling pathway in the pathogenesis of pulmonary fibrosis, supporting the therapeutic potential of ROCK inhibition to treat the disease. The data will be presented today as a poster at the 2017 Keystone Symposia on Injury, Inflammation and Fibrosis, taking place in Snowbird, UT.

“These data demonstrate that ROCK inhibition blocks multiple cell signaling mechanisms that drive pulmonary fibrosis, including α-SMA expression and endothelial and epithelial cell function,” said Masha Poyurovsky, PhD, Vice President, Head of Molecular Signaling at Kadmon. “By targeting ROCK, we can disrupt fundamental aspects of the TGFβ signaling pathway to potentially treat pulmonary fibrosis.”

Recent studies conducted by Kadmon and others have shown that ROCK signaling plays a key role in the development of pulmonary fibrosis, or scarring of the lungs that can lead to organ failure and death. Specifically, the ROCK pathway mediates cell signaling of Transforming Growth Factor-β (TGFβ), a master regulator of wound healing response that is essential for proper functions of multiple tissues. By targeting ROCK, Kadmon researchers believe that they can inhibit fibrosis while avoiding the toxicities associated with broad TGFβ inhibition.

To investigate the therapeutic potential of ROCK inhibition in fibrosis, Kadmon researchers generated a series of potent small molecule ROCK inhibitors and assessed their ability to modulate cellular mechanisms underlying aberrant signaling in pulmonary fibrosis. The findings demonstrated that ROCK inhibition blocks the expression of α-smooth muscle actin (α-SMA), the defining component of fibrosis-causing myofibroblast cells. Along with targeting α-SMA expression, ROCK inhibition reduces the activity of endothelial cells and epithelial cells, major contributors to the progressive organ injury associated with fibrosis. In summary, researchers determined that ROCK activity is essential to the fibrotic process and that ROCK inhibition shows potential in treating pulmonary fibrosis.

“Through our ROCK research platform, we continue to establish the importance of ROCK signaling in fibrotic disease,” said Harlan W. Waksal, M.D., President and CEO at Kadmon. “These findings further support the clinical development of our ROCK inhibitor KD025, for which we have three ongoing Phase 2 clinical trials, including in idiopathic pulmonary fibrosis.”

About Kadmon Holdings, Inc.

Kadmon Holdings, Inc. is a fully integrated biopharmaceutical company developing innovative products for significant unmet medical needs. We have a diversified product pipeline in autoimmune and fibrotic diseases, oncology and genetic diseases.

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For Kadmon Holdings, Inc.
Investor Relations
Ellen Tremaine, 646-490-2989
Maeve Conneighton, 212-600-1902

Source: Kadmon Holdings, Inc.