FDA Grants Breakthrough Therapy Designation to Kadmon's KD025 for Chronic Graft-Versus-Host Disease
October 17 2018 - 9:00AM
Kadmon Holdings, Inc. (NYSE: KDMN) today announced that the U.S.
Food and Drug Administration (FDA) has granted Breakthrough Therapy
Designation to KD025, the company’s ROCK2 inhibitor, for the
treatment of patients with chronic graft-versus-host disease
(cGVHD) after failure of two or more lines of systemic therapy.
Kadmon is currently enrolling patients in a pivotal Phase 2
clinical trial of KD025 in patients with cGVHD.
The FDA selectively grants Breakthrough Therapy
Designation to expedite the development and review of a drug that
is intended to treat a serious condition and preliminary clinical
evidence indicates that the drug may demonstrate substantial
improvement over existing therapies.
“We are pleased that the FDA has recognized the
therapeutic potential of KD025 for cGVHD, a serious condition for
which new treatments are urgently needed,” said Harlan W. Waksal,
M.D., President and CEO at Kadmon. “This designation is a key
regulatory milestone and we look forward to working closely with
the FDA to expedite the development of KD025 for cGVHD.”
The Breakthrough Therapy Designation of KD025 in
cGVHD is supported by data from an ongoing Phase 2 clinical trial
(KD025-208). In the study, KD025 was well tolerated and
demonstrated clinical activity in approximately two-thirds of
patients across Cohort 1 (KD025 200 mg QD) and Cohort 2 (KD025 200
mg BID). Preliminary results from the trial were previously
presented at the 23rd Congress of the European Hematology
Association (EHA) in June 2018 and at the Blood and Marrow
Transplantation (BMT) Tandem Meetings in February
2018.
Enrollment is ongoing in KD025-213, the pivotal
Phase 2 clinical trial of KD025 in adults with cGVHD who have
received at least two prior lines of systemic therapy. KD025-213 is
an open-label trial in which patients are randomized to receive
either KD025 200 mg QD or 200 mg BID, enrolling 63 patients per
arm. Either KD025 dose may be considered by the FDA for the
registrational dose.
About KD025
KD025 is a selective oral inhibitor of
Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway
that modulates inflammatory response. In October 2017, the FDA
granted orphan drug designation to KD025 for the treatment of
cGVHD.
About cGVHD
cGVHD is a common and often fatal complication
following hematopoietic stem cell transplantation. In 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 product candidates
for significant unmet medical needs. Our product pipeline is
focused on inflammatory and fibrotic diseases.
Forward Looking Statements
This press release contains forward-looking
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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 and 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 August 9, 2018. 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.
Contact InformationEllen
Cavaleri, Investor Relations
646.490.2989ellen.cavaleri@kadmon.com
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