Fortress Biotech Announces Orphan Drug Designations for CAEL-101 in Amyloidosis
April 05 2017 - 8:00AM
Fortress Biotech, Inc. (NASDAQ:FBIO) (“Fortress”), a
biopharmaceutical company dedicated to acquiring, developing and
commercializing novel pharmaceutical and biotechnology products,
today announced that the U.S. Department of Health & Human
Services has confirmed the transfer of two U.S. Food and Drug
Administration (FDA) Orphan Drug Designations for CAEL-101 (also
known as 11-1F4) from Columbia University (“Columbia”) to
Fortress’ subsidiary, Caelum Biosciences, Inc. (“Caelum”). The two
Orphan Drug Designations include the use of CAEL-101 as a
therapeutic agent for patients with AL amyloidosis, and the use of
CAEL-101 as a radio-imaging agent in amyloidosis. Caelum
in-licensed CAEL-101 from Columbia in January 2017.
CAEL‐101 is a fibril‐reactive monoclonal antibody (mAb)
currently being evaluated in a Phase 1b study for the treatment of
AL amyloidosis, a rare systemic disorder caused by an abnormality
of plasma cells in the bone marrow. Misfolded amyloid proteins
produced by plasma cells cause buildup in and around tissues,
nerves and organs, gradually affecting their function. This can
cause progressive and widespread organ damage, and high mortality
rates.
Dr. Lindsay A. Rosenwald, Fortress Biotech’s Chairman, President
and Chief Executive Officer, said, “The transfer of these two
Orphan Drug Designations is significant for Caelum, as they will
provide additional market exclusivity and financial benefits that
will enable Caelum to advance CAEL-101 as a potential improved
treatment option to AL amyloidosis patients in need. Preliminary
data for CAEL-101 has demonstrated biomarker activity in a Phase
1a/1b study that may indicate the therapy’s ability to safely
promote amyloid elimination and the subsequent improvement of organ
function. We look forward to reporting full data from the Phase
1a/1b study later this year, and plan to initiate a Phase 2 study
in 2018.”
FDA Orphan Drug Designation is granted to investigational
therapies that address rare medical diseases or conditions that
affect fewer than 200,000 people in the U.S. Orphan Drug status
provides benefits to drug developers including assistance in the
drug development process, tax credits for clinical costs,
exemptions from certain FDA fees and seven years of marketing
exclusivity.
About Caelum BiosciencesCaelum Biosciences,
Inc. (“Caelum”), a Fortress Biotech (NASDAQ:FBIO) Company, is a
clinical-stage biotechnology company developing treatments for rare
and life-threatening diseases. Caelum’s lead asset, CAEL-101
(11-14F), is a novel antibody in Phase 1b clinical trials for the
treatment of patients with amyloid light chain (“AL”) amyloidosis,
a rare systemic disorder that results in the buildup of misfolded
amyloid proteins in and around tissues, nerves and organs,
gradually affecting their function. Interim Phase 1a/1b data
presented at the American Society of Hematology’s (ASH) 58th Annual
Meeting in December 2016 support CAEL-101’s potential to be a safe
and well-tolerated therapy that promotes amyloid resolution.
CAEL-101 has received Orphan Drug Designation from the U.S. Food
and Drug Administration (FDA) as a therapeutic agent for patients
with AL amyloidosis, and as a radio-imaging agent in amyloidosis.
For more information, visit www.caelumbio.com.
About Fortress BiotechFortress Biotech, Inc.
(“Fortress”) is a biopharmaceutical company dedicated to acquiring,
developing and commercializing novel pharmaceutical and
biotechnology products. Fortress develops and commercializes
products both within Fortress and through certain of its subsidiary
companies, also known as Fortress Companies. Additionally, Fortress
recently acquired a controlling interest in National Holdings
Corporation (NASDAQ:NHLD), a diversified independent brokerage
company (together with its subsidiaries, “NHLD”). In addition to
its internal development programs, Fortress leverages its
biopharmaceutical business expertise and drug development
capabilities and provides funding and management services to help
the Fortress Companies achieve their goals. Fortress and the
Fortress Companies may seek licensings, acquisitions, partnerships,
joint ventures and/or public and private financings to accelerate
and provide additional funding to support their research and
development programs. For more information, visit
www.fortressbiotech.com.
Forward-Looking StatementsThis
press release may contain “forward-looking statements” within the
meaning of Section 27A of the Securities Act of 1933 and Section
21E of the Securities Exchange Act of 1934, as amended. Such
statements include, but are not limited to, any statements relating
to our growth strategy and product development programs and any
other statements that are not historical facts. Forward-looking
statements are based on management’s current expectations and are
subject to risks and uncertainties that could negatively affect our
business, operating results, financial condition and stock price.
Factors that could cause actual results to differ materially from
those currently anticipated include: risks relating to our growth
strategy; risks relating to the results of research and development
activities; risks relating to the timing of starting and completing
clinical trials; our ability to obtain, perform under and maintain
financing and strategic agreements and relationships; uncertainties
relating to preclinical and clinical testing; our dependence on
third-party suppliers; our ability to attract, integrate, and
retain key personnel; the early stage of products under
development; our need for substantial additional funds; government
regulation; patent and intellectual property matters; competition;
as well as other risks described in our SEC filings. We expressly
disclaim any obligation or undertaking to release publicly any
updates or revisions to any forward looking statements contained
herein to reflect any change in our expectations or any changes in
events, conditions or circumstances on which any such statement is
based, except as required by law.
Contacts:
Lucy Lu, M.D., Executive Vice President & Chief Financial Officer
Fortress Biotech, Inc.
781-652-4500; ir@fortressbiotech.com
Fortress Biotech Media Relations
Laura Bagby
6 Degrees
(312) 448-8098; lbagby@6degreespr.com
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