Kadmon Announces Updated Results of KD025 in cGVHD at EHA Congress

Kadmon Holdings, Inc. (NYSE: KDMN) today announced updated efficacy and
safety results from an ongoing Phase 2 clinical trial evaluating KD025
in patients with steroid-dependent or refractory chronic
graft-versus-host disease (cGVHD). Results from the study will be
presented in an oral session on Saturday, June 16th, 2018 at
the 23rd Congress of the European Hematology Association
(EHA) in Stockholm, Sweden. The presentation is now available on the
Kadmon website.

The updated study results include four additional months of patient
follow-up, through May 2, 2018. The Overall Response Rate (ORR)
continues to be 65% (11/17) in Cohort 1 (KD025 200 mg QD; n=17) and 69%
(11/16) in Cohort 2 (KD025 200 mg BID; n=16). Responses have been
durable, with 47% (8/17) of patients in Cohort 1 having sustained a
response for at least 20 weeks, up from 41% (7/17) in the last reported
analysis. Initial durability data from Cohort 2 demonstrated that 38%
(6/16) of patients have sustained a response for at least 20 weeks,
which is comparable to durability data observed in Cohort 1 at the same
median treatment duration. Durability data continue to mature across
Cohorts 1 and 2. Sixty-six percent (66%; 22/33) of all patients were
able to reduce doses of corticosteroids and 15% (5/33) have completely
discontinued steroid use. In addition, 55% (18/33) of patients across
Cohorts 1 and 2 saw a clinically meaningful improvement in cGVHD
symptoms, as measured by at least a 7-point decrease in the Lee Symptom
Scale score. KD025 continued to be well tolerated, with no
treatment-related serious adverse events and no apparent increased risk
of infection.

“KD025 continues to achieve robust and durable responses across multiple
organs affected by cGVHD, while simultaneously allowing the majority of
patients to taper doses of steroids,” said Amandeep Salhotra, M.D.,
Assistant Professor, Hematology and Hematopoietic Cell Transplantation,
City of Hope, Duarte, CA, and study investigator. “In addition, the
favorable tolerability profile of KD025 has allowed patients to continue
on treatment and maintain responses, highlighting its potential for
long-term use as a backbone of cGVHD therapy.”

Kadmon plans to initiate an open-label, pivotal study of KD025 in cGVHD
in Q3 2018, based on FDA guidance received in a Type C meeting. The
planned pivotal study (KD025-213) will enroll adults who have received
at least two prior lines of systemic therapy for cGVHD. Patients will be
randomized to receive KD025 200 mg QD or 200 mg BID (63 patients per
cohort), and either dose may be considered by the FDA for the
registrational dose. The primary endpoint is ORR, supported by a key
secondary endpoint of Duration of Response.

“These study results continue to show accumulation of clinical benefit
and underscore the significant potential of KD025 in cGVHD, particularly
in patients with multiple organs involved,” said Harlan W. Waksal, M.D.,
President and CEO at Kadmon. “We have a clear regulatory path forward
for KD025 and we look forward to initiating our pivotal study in the
third quarter of this year.”

About KD025-208

KD025-208 is an ongoing Phase 2 clinical trial of KD025, Kadmon’s
Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, in adults with
steroid-dependent or steroid-refractory cGVHD and active disease. The
dose-finding trial includes 48 patients divided into three cohorts at
different dose levels (KD025 200 mg QD, 200 mg BID and 400 mg QD),
enrolled sequentially following a safety assessment of each cohort. In
October 2017, KD025 received orphan drug designation from the U.S. Food
and Drug Administration for cGVHD.

About cGVHD

cGVHD is a potentially life-threatening condition following
hematopoietic stem cell transplantation. With cGVHD, transplanted immune
cells (graft) attack the patient’s cells (host), leading to inflammation
and fibrosis in multiple tissues, including skin, mouth, eye, joints,
liver, lung, esophagus and GI tract.

About Kadmon Holdings, Inc.

Kadmon Holdings, Inc. is a fully integrated biopharmaceutical company
developing innovative products for significant unmet medical needs. Our
product pipeline is focused on inflammatory and fibrotic diseases.

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