Kadmon Data Published in Science Signaling Show ROCK2 Signaling as a Key Modulator of T Follicular Helper Cell Function in Autoimmune Diseases

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.

This press release contains forward-looking statements. These
forward-looking statements are based on management’s expectations and
are subject to certain factors, risks and uncertainties that may cause
actual results, outcome of events, timing and performance to differ
materially from those expressed or implied by such statements. The
information contained in this press release is believed to be current as
of the date of original issue. Kadmon expressly disclaims any obligation
or undertaking to release publicly any updates or revisions to any
forward-looking statements contained herein to reflect any change in our
expectations with regard thereto or any change in events, conditions or
circumstances on which any such statements are based.

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