Overall Response Rate of 71%, with a Favorable
Safety Profile
Findings to be Presented and Webcast Today at
8:45 a.m. ET at Kadmon’s Research and Development (“R&D”) Day
in New York
Kadmon Holdings, Inc. (NYSE:KDMN) (“Kadmon” or the “Company”)
today announced positive interim results from its ongoing Phase 2
clinical trial evaluating KD025, the Company’s Rho-associated
coiled-coil kinase 2 (“ROCK2”) inhibitor, in patients with
previously treated chronic graft-versus-host disease (“cGVHD”), a
serious complication following allogeneic bone marrow or stem cell
transplantation. In a preliminary analysis, KD025 demonstrated a
favorable safety and tolerability profile in all patients and an
overall response rate (“ORR”) of 71%.
KD025-208 is an ongoing Phase 2 clinical trial evaluating the
safety, tolerability and activity of KD025 in adults with
steroid-dependent or steroid-refractory cGVHD and active disease.
The dose-finding study 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 a preliminary analysis of data from the first cohort, KD025
200 mg QD (n=17), KD025 demonstrated clinically meaningful
responses in cGVHD patients, with an ORR of 71% (12/17), which
includes complete and partial responders. Of responders remaining
on KD025 through week 24, 7 of 8 (88%) sustained responses. In
addition to the 12 responders, two patients have stable disease and
remain on KD025 through week 24. Overall clinical benefit (response
and stable disease) occurred in 82% (14/17) of patients.
Sixty-seven percent (67%; 8/12) of responders saw an improvement in
symptoms as measured by the Lee cGVHD Symptom Scale score. To date,
no drug-related serious adverse events (SAEs) have been recorded,
and no drug-related elevations in liver function tests have been
observed. Importantly, 67% (8/12) of responders had reduction of
steroid doses and 67% (4/6) of patients had reduction of tacrolimus
doses.
“These positive interim findings indicate activity and a
favorable safety profile of KD025 in cGVHD, a fatal disease with no
approved therapies,” said John L. Ryan, M.D., Ph.D., Executive Vice
President, Chief Medical Officer of Kadmon. “Steroids are the
current standard therapy for cGVHD and have severe side effects
associated with long-term use. We are pleased to see that the
majority of patients in the first cohort have been able to reduce
their steroid doses, indicating that KD025 potentially offers a
well-tolerated treatment option for cGVHD patients.”
“We are encouraged by these initial activity and safety results,
which support the potential of KD025 to treat this unmet medical
need,” said Harlan W. Waksal, M.D., President and Chief Executive
Officer of Kadmon. “Of note, these data are from the lowest dose of
KD025 being studied, and we look forward to further exploring KD025
activity and safety in the two additional higher-dose cohorts.”
R&D Day and Webcast Information
Kadmon will present its findings today at 8:45 a.m. ET at the
Company’s R&D Day in New York. A live and archived webcast of
the event, with slides, will be available on the Investors section
of the Kadmon website at www.kadmon.com. The live
event is intended for institutional investors and sell-side
analysts only. For more information, please email Ellen Tremaine,
Manager, Investor Relations of Kadmon, at
ellen.tremaine@kadmon.com.
About cGVHD
cGVHD is a common and often fatal complication following
allogeneic bone marrow transplantation and hematopoietic stem cell
transplantation, which are procedures that are often used to treat
patients with cancers such as myeloma or leukemia. With cGVHD,
transplanted immune cells (graft) attack the patient’s body (host),
leading to inflammation and fibrosis in multiple tissues, including
skin, mouth, eye, joints, liver, lung and digestive tract.
About KD025
KD025 is a selective oral inhibitor of ROCK2, a signaling
pathway implicated in the pathogenesis of autoimmune and fibrotic
diseases. KD025 is being studied in three ongoing Phase 2 clinical
trials: a placebo-controlled trial in moderate to severe psoriasis,
an open-label trial in idiopathic pulmonary fibrosis and an
open-label trial in cGVHD.
About the Study
KD025-208 is an ongoing Phase 2 study evaluating the safety,
tolerability and activity of KD025 in 48 patients with
steroid-dependent or steroid-refractory cGVHD and active disease.
The primary endpoint is KD025 activity at 24 weeks in terms of ORR,
as defined by the 2014 National Institutes of Health (NIH)
Consensus Response Criteria for overall response. Secondary
endpoints include changes in corticosteroid and calcineurin
inhibitor dose. Exploratory endpoints include change in symptom
burden or bother using the Lee cGVHD Symptom Scale.
Enrollment in the first cohort (KD025 200 mg QD) is complete and
enrollment in the second cohort (200 mg BID) is nearly complete,
with 15 of 16 patients enrolled. The safety assessment of the 200
mg BID cohort is complete and supports initiating enrollment in the
400 mg QD cohort. Additional study results will be submitted for
presentation at upcoming scientific conferences.
About Kadmon Holdings, Inc.
Kadmon Holdings, Inc. 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.
Safe Harbor Statement
This press release contains forward-looking statements. Such
statements may be preceded by the words “may,” “will,” “should,”
“expects,” “plans,” “anticipates,” “could,” “intends,” “targets,”
“projects,” “contemplates,” “believes,” “estimates,” “predicts,”
“potential” or “continue” or the negative of these terms or other
similar expressions. Forward-looking statements involve known and
unknown risks, uncertainties and other important factors that may
cause our actual results, performance or achievements to be
materially different from any future results, performance or
achievements expressed or implied by the forward-looking
statements. These factors include statements regarding KD025, the
results of KD025 in KD025-208, including in the second and third
cohorts to be treated, as well as the long-term results of KD025 in
the first cohort, and the prospects for KD025 in other indications.
We believe that these factors also 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 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; (xx) our ability to achieve cost savings and other
benefits from our efforts to streamline our operations and to not
harm our business with such efforts; and (xxi) the use of proceeds
from our recent private placement. 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 May 15, 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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version on businesswire.com: http://www.businesswire.com/news/home/20170711005778/en/
Kadmon Holdings, Inc.Ellen Tremaine, 646-490-2989Investor
Relationsellen.tremaine@kadmon.comorMaeve Conneighton,
212-600-1902maeve@argotpartners.com
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