Kadmon Receives FDA Guidance on Pivotal Clinical Trial Design for KD025 in Chronic Graft-Versus-Host Disease
April 10 2018 - 8:00AM
Business Wire
Kadmon Holdings, Inc. (NYSE:KDMN) (the “Company”) today
announced that it has received final minutes from a March 2018 Type
C meeting with the U.S. Food and Drug Administration (FDA)
regarding the development pathway for KD025, the Company’s
Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the
treatment of chronic graft-versus-host disease (cGVHD). Based on
the FDA’s guidance, Kadmon plans to initiate an open-label,
two-arm, pivotal Phase 2 clinical trial to support the potential
registration of KD025 in cGVHD.
In the planned pivotal trial (KD025-213), patients will be
randomized to receive one of two dose levels of KD025: 200 mg QD or
200 mg BID; 48 patients will be enrolled into each arm. Either
KD025 dose may be considered by the FDA for the registrational
dose. The study will enroll adults with active cGVHD who have
received at least two prior lines of systemic therapy for cGVHD.
The primary endpoint is the Overall Response Rate (ORR), defined as
the percentage of patients who meet the 2014 National Institutes of
Health (NIH) Consensus Conference overall response criteria,
supported by a key secondary endpoint of Duration of Response
(DOR).
“We are pleased with the FDA’s guidance, which provides us with
a clear regulatory path forward to support a submission for
potential approval,” said Harlan W. Waksal, M.D., President and CEO
at Kadmon. “Furthermore, this trial design allows us to evaluate
two dose levels of KD025, either of which could meet the target for
potential registration. We look forward to advancing KD025 through
this pivotal study.”
About KD025
KD025 is a selective oral inhibitor of ROCK2, a signaling
pathway that mediates cell movement, shape, differentiation and
function and is dysregulated in many chronic diseases, including
cGVHD. Previously reported data from an ongoing Phase 2 clinical
trial of KD025 in cGVHD (KD025-208) demonstrated an ORR of
approximately 66%. KD025 was also well tolerated, with no
drug-related serious adverse events (SAEs). In October 2017, KD025
received orphan drug designation from the FDA for KD025 in
cGVHD.
About cGVHD
cGVHD is a common and often fatal complication following
hematopoietic stem cell transplantation, a procedure that is often
used to treat patients with cancers such as myeloma or leukemia.
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,
fibrotic and genetic diseases.
Forward Looking Statements
This press release contains forward-looking statements. Such
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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. We believe that these 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 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; (xxi) the use of proceeds from
our recent public offerings; (xxii) the potential benefits of any
of our product candidates being granted orphan drug designation;
(xxiii) the future trading price of the shares of our common stock
and impact of securities analysts’ reports on these prices; and/or
(xxiv) other risks and uncertainties. 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 (the “SEC”),
including the Company’s Annual Report on Form 10-K for the fiscal
year ended December 31, 2017. Investors and security holders are
urged to read these documents free of charge on the SEC’s website
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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Kadmon Holdings, Inc.Ellen Tremaine, 646-490-2989Investor
Relationsellen.tremaine@kadmon.com
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