Kadmon Initiates Phase 2 Clinical Trial Evaluating KD025 in Chronic Graft-Versus Host Disease

NEW YORK–(BUSINESS WIRE)– Kadmon Holdings, Inc. (NYSE:KDMN) today announced that the first patient
has been dosed in a Phase 2 clinical trial of KD025, the Company’s
rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment
of chronic graft-versus-host disease (cGVHD). The randomized,
open-label, 24-week study examines the safety, tolerability and activity
of KD025 dosed at 200 mg once daily (QD), 200 mg twice daily (BID) or
400 mg QD in 48 cGVHD patients in the United States.

cGVHD is a common and often fatal complication following allogeneic stem
cell transplantation in which donor immune cells attack the recipient’s
body, leading to fibrosis in multiple organs. Preclinical research
conducted by Kadmon in collaboration with Bruce Blazar, MD, of the
University of Minnesota and recently published in the journal Blood demonstrated
that ROCK2 inhibition with KD025 down-regulated clinical symptoms in
multiple murine models of cGVHD. KD025 treatment also down-regulated the
pro-inflammatory signaling pathways involved in cGVHD pathogenesis and
simultaneously up-regulated regulatory cell function in these murine
models, reducing overall cGVHD progression while preserving normal
immune function.

“Kadmon’s preclinical research has demonstrated the potential of ROCK2
inhibition to treat a number of autoimmune and fibrotic diseases,
including cGVHD, a serious condition with limited treatment options,”
said Harlan W. Waksal, M.D., President and Chief Executive Officer of
Kadmon. “In this Phase 2 study, we hope to further assess the ability of
KD025, our lead ROCK2 inhibitor, to ameliorate the cGVHD process.”

In addition to cGVHD, Kadmon is investigating the potential of KD025 to
treat other autoimmune and fibrotic diseases. The company is conducting
an ongoing Phase 2 clinical study of KD025 for the treatment of
idiopathic pulmonary fibrosis, with a planned Phase 2 study in moderate
to severe psoriasis.

About Kadmon Holdings, Inc.

Kadmon Holdings, Inc., is a fully integrated biopharmaceutical company
engaged in the discovery, development and commercialization of small
molecules and biologics to address disease areas of significant unmet
medical need. Kadmon is developing product candidates within autoimmune
and fibrotic diseases, oncology and genetic diseases.

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