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Kadmon Announces Positive Topline Results from Phase 2 Study of KD025 in Idiopathic Pulmonary Fibrosis

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-- Conference Call and Webcast Tomorrow at 8:30 a.m. ET --

Kadmon Holdings, Inc. (NYSE:KDMN) today announced topline results from
an ongoing Phase 2 clinical trial evaluating KD025, its Rho-associated
coiled-coil kinase 2 (ROCK2) inhibitor, in patients with idiopathic
pulmonary fibrosis (IPF) who were previously treated with or offered
pirfenidone and/or nintedanib. KD025 was well tolerated and demonstrated
clinical benefit, with a median decline in forced vital capacity (FVC),
a measure of lung function, of 48 mL at week 24, compared to a median
decline of 175 mL in patients treated with best supportive care (BSC),
an absolute difference of 127 mL and a relative difference of 73%.

"KD025 represents a novel mechanism of action in IPF by inhibiting ROCK,
a central regulator of fibrosis that mediates several pro-fibrotic
responses, including stress fiber formation, myofibroblast activation
and pro-fibrotic gene transcription," said Kevin F. Gibson, MD,
Professor and Medical Director, Dorothy P. and Richard P. Simmons Center
for Interstitial Lung Disease, University of Pittsburgh Medical Center
and lead investigator of the study. "In this proof-of-concept trial,
KD025 has demonstrated clinical activity at 24 weeks and was well
tolerated, with no apparent safety signals, potentially offering a new
option for patients with IPF."

In the open-label trial (KD025-207), patients who were previously
treated with or offered pirfenidone and/or nintedanib were randomized
2:1 to receive KD025 400 mg QD monotherapy or BSC. The primary endpoints
were safety and tolerability of KD025 and change in FVC from baseline to
24 weeks. Patients have the option to continue treatment with KD025
beyond 24 weeks.

As of a data cutoff date of February 1, 2018, 20 evaluable patients have
completed 24 weeks of KD025 treatment, and 9 evaluable patients in the
BSC arm have completed 24 weeks of follow-up. Approximately 44% of all
patients enrolled in the trial had received prior treatment with
pirfenidone and/or nintedanib. Following are key results:

  • The median decline in FVC at 24 weeks was 48 mL in the KD025 arm,
    compared to a median decline of 175 mL in the BSC arm, a relative
    difference of 73%.
  • The median decline in FVC % predicted from baseline to week 24 was 1%
    in KD025 patients, compared to a median decline of 2% in BSC patients,
    a relative difference of 50%.
  • Treatment with KD025 reduced the proportion of patients who
    experienced IPF progression: At 24 weeks, 20% of KD025 patients
    experienced FVC % predicted decline ≥5%, compared to 44% of BSC
    patients, a relative difference of 55%.
  • KD025 patients experienced less FVC decline on an annualized basis
    relative to the year prior to enrollment: Evaluable patients
    randomized to KD025 had an annualized decline in FVC of 126 mL in the
    year prior to randomization, compared to an annualized decline of 32
    mL at 24 weeks of KD025 treatment.
  • KD025 was well tolerated, with no drug-related serious adverse events.
    In addition, 90% of patients who received KD025 for 24 weeks have
    elected to continue KD025 treatment beyond week 24.

"We are pleased with today's results, which demonstrate the activity and
tolerability of KD025 in IPF, including in patients who have received
prior therapy with approved agents," said Harlan W. Waksal, M.D.,
President and CEO at Kadmon. "These findings support the therapeutic
potential of ROCK inhibition in IPF and further validate Kadmon's ROCK
inhibitor platform, which is being applied across programs in fibrotic
diseases as well as inflammatory diseases."

Kadmon plans to present the results from this study at the American
Thoracic Society (ATS) International Conference in May 2018.

Conference Call and Webcast

Kadmon will host a conference call and webcast on February 13, 2018, at
8:30 a.m. ET to discuss top-line results from the KD025-207 study. To
access the webcast, please visit the Investors section of www.kadmon.com,
under "Presentations & Events." A replay of the webcast will be archived
on the Company's website for 30 days.

Dial-in Information:
(866) 762-3021
Conference ID: 369-4419

About KD025

KD025 is a selective oral inhibitor of ROCK2, a signaling pathway
involved in the pathogenesis of multiple chronic diseases. Published
research by Kadmon and academic institutions has demonstrated that KD025
regulates fibrotic processes and aberrant immune responses. Kadmon is
conducting Phase 2 clinical trials of KD025, including in idiopathic
pulmonary fibrosis (IPF) and chronic graft-versus-host disease (cGVHD).

About IPF

IPF is a progressive fibrotic disease of the lungs, with a median
survival of 3 to 5 years from the time of diagnosis. IPF is thought to
be caused by repetitive environmental injury to the lining of the lung
airways and the resulting abnormal wound-healing responses. IPF patients
are in need of new therapies that provide clinical benefit.

About Kadmon Holdings, Inc.

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

Forward-Looking Statements

This press release contains forward-looking statements. Such
statements may be preceded by the words "may," "will," "should,"
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"projects," "contemplates," "believes," "estimates," "predicts,"
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our actual results, performance or achievements to be materially
different from any future results, performance or achievements expressed
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factors include, but are not limited to, (i) the initiation, timing,
progress and results of our preclinical studies and clinical trials, and
our research and development programs; (ii) our ability to advance
product candidates into, and successfully complete, clinical trials;
(iii) our reliance on the success of our product candidates; (iv) the
timing or likelihood of regulatory filings and approvals; (v) our
ability to expand our sales and marketing capabilities; (vi) the
commercialization of our product candidates, if approved; (vii) the
pricing and reimbursement of our product candidates, if approved; (viii)
the implementation of our business model, strategic plans for our
business, product candidates and technology; (ix) the scope of
protection we are able to establish and maintain for intellectual
property rights covering our product candidates and technology; (x) our
ability to operate our business without infringing the intellectual
property rights and proprietary technology of third parties; (xi) costs
associated with defending intellectual property infringement, product
liability and other claims; (xii) regulatory developments in the United
States, Europe and other jurisdictions; (xiii) estimates of our
expenses, future revenues, capital requirements and our needs for
additional financing; (xiv) the potential benefits of strategic
collaboration agreements and our ability to enter into strategic
arrangements; (xv) our ability to maintain and establish collaborations
or obtain additional grant funding; (xvi) the rate and degree of market
acceptance, if any, of our product candidates; (xvii) developments
relating to our competitors and our industry, including competing
therapies; (xviii) our ability to effectively manage our anticipated
growth; (xix) our ability to attract and retain qualified employees and
key personnel; and/or (xx) our ability to achieve cost savings and
benefits from our efforts to streamline our operations and to not harm
our business with such efforts. More detailed information about Kadmon
and the risk factors that may affect the realization of forward-looking
statements is set forth in the Company's filings with the U.S.
Securities and Exchange Commission ("SEC"), including the Company's
Quarterly Report on Form 10-Q filed pursuant to Section 13 of the
Securities Exchange Act of 1934, as amended, with the SEC on November 9,
2017. Investors and security holders are urged to read these documents
free of charge on the SEC's web site at
www.sec.gov.
The Company assumes no obligation to publicly update or revise its
forward-looking statements as a result of new information, future events
or otherwise.

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