NEW YORK--(BUSINESS WIRE)--Kadmon Corporation, LLC today announced the publication of preclinical data demonstrating that inhibition of rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway implicated in many autoimmune diseases, decreased the development and function of cells involved in the pro-inflammatory immune response while preserving normal immune system function. The data, published today in the journal Science Signaling, support the potential of ROCK2 inhibition to treat certain autoimmune diseases, including systemic lupus erythematosus (SLE).
The immune response is regulated in part by T follicular helper (Tfh) cells, which facilitate the production of antibodies to foreign antigens. In autoimmune disease, uncontrolled Tfh cell activity leads to high levels of auto-antibodies, causing organ dysfunction. The published data demonstrate that treatment with KD025, the Company’s selective ROCK2 inhibitor, blocks specific signaling pathways that regulate Tfh cell development and function while leaving the normal immune response intact. Specifically, in normal human T cells and in cells from SLE patients, ROCK2 inhibition with KD025 reduced the percentage of Th17-type Tfh cells, which are elevated in autoimmune disease settings, with no effect on Th1-type Tfh cells, helping to preserve normal humoral immune response. In a mouse model of lupus, KD025-treated animals showed a reduced percentage of Tfh cells in spleens as well as substantial improvements in both clinical and histological scores, further confirming the role of ROCK2 signaling in regulating immune response.
“The ROCK2 signaling pathway plays an instrumental role in controlling Tfh cells, which are key drivers of dysregulated antibody production in autoimmune disease settings,” said Alexandra Zanin-Zhorov, Ph.D., Vice President, Head of Immunology at Kadmon and corresponding author of the manuscript. “ROCK2 inhibition also prevented autoimmune disease progression in a mouse model, further highlighting the importance of ROCK2 signaling in modulating immune response.”
“ROCK2 inhibition selectively targets auto-aggressive immune responses while preserving normal immune function, representing a new paradigm of therapy for certain autoimmune diseases,” said Harlan W. Waksal, M.D., President and CEO at Kadmon. “The published data provide further support for our clinical programs studying selective ROCK2 inhibition with KD025 for the treatment of multiple autoimmune diseases, including SLE.”
About Kadmon Corporation
Kadmon Corporation, LLC is a fully integrated biopharmaceutical company focused on 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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