Caladrius Biosciences Receives FDA Fast Track Designation for CLBS03 to Treat Recent Onset Type 1 Diabetes
July 28 2016 - 07:30AM
Caladrius Biosciences, Inc. (NASDAQ:CLBS) (“Caladrius” or the
“Company”), a cell therapy company combining an industry-leading
development and manufacturing services provider, PCT, with a select
therapeutic development pipeline, announces today that its product
candidate CLBS03 (autologous expanded polyclonal regulatory T
cells, or Tregs) was granted Fast Track designation by the US Food
and Drug Administration (FDA) for the treatment of type 1 diabetes
mellitus (TID), making it the first known therapeutic candidate for
treatment of T1D to receive the designation. CLBS03 has received
Orphan Drug designation from the FDA as well as Advanced
Therapeutic Medicinal Product (ATMP) classification from the
European Medicines Agency. CLBS03 is currently being studied in a
landmark Phase 2 clinical trial in collaboration with Sanford
Health, The Sanford Project: T-Rex Study, which is expected to
complete enrollment of the first defined cohort of 18 patients in
the coming weeks.
Fast Track is a process designed to facilitate the development
and expedite the review of drugs, biologics or treatments for
serious conditions, thereby filling unmet medical needs. Through
the Fast Track program, a product may be eligible for priority
review at the time of a Biologic License Application (BLA) or New
Drug Application (NDA) filing and may also be eligible to submit
completed sections of the BLA/NDA on a rolling basis before the
complete application is submitted. These expedited processes can
significantly cut down the development time and cost associated
with bringing a therapy to market. Furthermore, the therapy's
sponsors are eligible for more frequent written communication and
meetings with the FDA, the benefit of which may be to foster a
pivotal study design which more closely meets the FDA’s needs,
thereby creating a more efficient and rapid pathway to
approval.
The scientific basis for treating T1D with CLBS03 derives from
the use of Tregs to treat autoimmune diseases caused by imbalances
in an individual’s immune system. This innovative approach seeks to
restore immune balance by enhancing Treg cell number and function.
Tregs are a natural part of the human immune system and regulate
the activity of T effector cells, which are responsible for
protecting the body from viruses and other foreign antigens. When
Tregs function properly, only harmful foreign materials are
attacked by T effector cells. In autoimmune diseases, deficient
Treg activity permits the T effector cells to attack the body’s own
beneficial cells, for example, insulin-producing pancreatic beta
cells in the case of T1D.
CLBS03 is a personalized, autologous medicine consisting of each
patient’s own Tregs, which have been expanded in number and
functionally enhanced by a proprietary method developed by a
collaboration between PCT and the University of California, San
Francisco. The program is supported by promising published
early clinical work conducted by respected leaders in the area of T
regulatory cell science. Two Phase 1 clinical trials of this
technology in T1D patients demonstrated safety and tolerance,
feasibility of manufacturing, infused Treg persistence and an early
indication of efficacy1,2. In particular, one of those trials
provided supportive evidence of the utility of Tregs for T1D in
pediatric patients 8 to 16 years of age with new onset T1D2. In
that open label study, the authors reported that treatment with
expanded autologous Tregs preserved function of pancreatic beta
cells and reduced the need for exogenous insulin in the majority of
patients treated.
“Obtaining Fast Track designation is a key milestone in our
regulatory and development strategy for CLBS03. It
underscores the great need for innovative treatments, such as
CLBS03, in the treatment of T1D and allows for the acceleration of
its development,” said David J. Mazzo, PhD, Chief Executive Officer
of Caladrius. “We are making excellent progress advancing the
U.S.-based Phase 2 clinical program of CLBS03 to treat T1D and look
to complete enrollment of the first cohort of 18 patients in the
coming weeks. This, coupled with our Orphan Drug and Fast Track
designations, should make CLBS03 an even more attractive
opportunity for a potential partner.”
- Bluestone, J., et al. Type 1 diabetes immunotherapy using
polyclonal regulatory T cells. Science Translational
Medicine, 2015 Nov;7(315): pp. 315ra189.
- Marek-Trzonkowsa, N., et al. Therapy of type 1 diabetes with
CD4(+)CD25(high)CD127-regulatory T cells prolongs survival of
pancreatic islets – results of one year follow-up. Clinical
Immunology. 2014 Jul;153(1): 23-30.
About The Sanford Project: T-Rex
Study
The landmark study is a prospective, randomized,
placebo-controlled, double-blind Phase 2 clinical trial to evaluate
the safety and efficacy of CLBS03 as a treatment for T1D with
residual beta cell function in approximately 111 subjects age 12 to
17 in two cohorts (18 subjects followed by 93 subjects). The study
is being conducted in collaboration with Sanford Research, a
subsidiary of Sanford Health. Subjects will be randomized into one
of three groups and will receive either a high dose of CLBS03, a
low dose of CLBS03 or placebo. The key endpoints for the
trial are the standard medical and regulatory endpoints for a T1D
trial and include preservation of C-peptide, an accepted measure
for pancreatic beta cell function; insulin use; severe hypoglycemic
episodes; and glucose and hemoglobin A1c levels.
About Caladrius Biosciences
Caladrius Biosciences, Inc., through its subsidiary, PCT, is a
leading development and manufacturing partner to the cell therapy
industry. PCT works with its clients to overcome the
fundamental challenges of cell therapy manufacturing by providing a
wide range of innovative services including product and process
development, GMP manufacturing, engineering and automation, cell
and tissue processing, logistics, storage and distribution, as well
as expert consulting and regulatory support. PCT and Hitachi
Chemical Co., Ltd. have entered into a strategic global
collaboration to accelerate the creation of a global commercial
cell therapy development and manufacturing enterprise with deep
engineering expertise. Around the core expertise of PCT,
Caladrius strategically develops select product candidates, which
currently includes an innovative therapy for type 1 diabetes based
on a proprietary platform technology for immunomodulation. For more
information, visit www.caladrius.com.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements reflect management’s current
expectations, as of the date of this press release, and involve
certain risks and uncertainties. All statements other than
statements of historical fact contained in this press release are
forward-looking statements, including statements regarding the
realization of the benefits of fast track designation for CLB03,
the achievement of clinical milestones for CLB03 including the
completion of enrollment for the initial cohort of 18 patients and
the establishment of a partnership for CLBS03. The Company’s
actual results could differ materially from those anticipated in
these forward-looking statements as a result of various factors.
Factors that could cause future results to materially differ from
the recent results or those projected in forward-looking statements
include the “Risk Factors” described in the Company’s Annual Report
on Form 10-K filed with the Securities and Exchange Commission
(“SEC”) on March 15, 2016, and in the Company’s other periodic
filings with the SEC, including: risks related to: (i) our
expected continued losses and negative cash flows; (ii) our
anticipated need for substantial additional financing; (iii) the
significant costs and management resources required to comply with
the requirements of being a public company; (iv) the
possibility that a significant market for cell therapy may not
emerge; (v) the potential variability in PCT’s revenues; (vi) PCT’s
limited manufacturing capacity; (vii) the need to improve
manufacturing efficiency at PCT; (viii) the limited marketing staff
and budget at PCT; (ix) the logistics associated with the
distribution of materials produced by PCT; (x) government
regulation; (xi) our intellectual property; (xii) cybersecurity;
(xiii) the development, approval and commercialization of our
products; (xiv) enrolling patients in and completing, clinical
trials; (xv) the variability of autologous cell therapy; (xvi) our
access to reagents we use in the clinical development of our cell
therapy product candidates; (xvii) the validation and establishment
of manufacturing controls; (xviii) the failure to obtain regulatory
approvals outside the United States; (xix) our failure to realize
benefits relating to “fast track” and “orphan drug” designations;
(xx) the failure of our clinical trials to demonstrate the safety
and efficacy of our product candidates; (xxi) our current lack of
sufficient manufacturing capabilities to produce our product
candidates at commercial scale; (xxii) our lack of revenue from
product sales; (xxiii) the commercial potential and profitability
of our products; (xxiv) our failure to realize benefits from
collaborations, strategic alliances or licensing arrangements;
(xxv) the novelty and expense of the technology used in our cell
therapy business; (xxvi) the possibility that our competitors will
develop and market more effective, safer or less expensive products
than our product candidates; (xxvii) product liability claims and
litigation, including exposure from the use of our products;
(xxviii) our potential inability to retain or hire key employees;
and (xxix) risks related to our capital stock. The Company’s
further development is highly dependent on, among other things,
future medical and research developments and market acceptance,
which are outside of its control.
CONTACTS:
Investors:
LHA
Anne Marie Fields
Senior Vice President
Phone: +1-212-838-3777
Email: afields@lhai.com
Media:
Caladrius Biosciences, Inc.
Eric Powers
Director, Communications and Marketing
Phone: +1-212-584-4173
Email: epowers@caladrius.com
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