Fate Therapeutics Receives FDA Orphan Drug Designation for ProTmune™ in Allogeneic Hematopoietic Cell Transplantation
September 26 2016 - 8:00AM
Fate Therapeutics, Inc. (NASDAQ:FATE), a biopharmaceutical company
dedicated to the development of programmed cellular immunotherapies
for cancer and immune disorders, announced today that the U.S. Food
and Drug Administration (FDA) has granted Orphan Drug Designation
for ProTmune™, the Company’s lead product candidate that is
currently undergoing Phase 1/2 clinical investigation. The FDA
designation is for “prevention of graft-versus-host disease in
patients undergoing allogeneic hematopoietic cell transplantation”
and broadly covers diseases, including blood cancers and genetic
disorders, for which the procedure is performed.
“The granting of both orphan drug and Fast Track designations
for ProTmune validates the product candidate’s unique therapeutic
potential to address life-threatening complications and improve the
curative potential of allogeneic HCT,” said Scott Wolchko,
President and Chief Executive Officer of Fate Therapeutics.
“Graft-versus-host disease is a significant cause of morbidity and
mortality in patients undergoing allogeneic HCT and there are no
FDA-approved therapies to prevent its occurrence. Through our
development of ProTmune, we seek to transform the allogeneic HCT
paradigm by providing immunocompromised patients a
therapeutically-optimized donor graft containing immune cells with
reduced alloreactivity and enhanced infection-fighting and
anti-tumor properties.”
Graft-versus-host disease (GvHD) is a severe immunological
complication that arises when newly-transplanted donor immune cells
attack the patient’s tissues and organs, resulting in a potentially
fatal immune system reaction. Despite the use of protocols to
prevent its occurrence, up to 50 percent of patients experience
GvHD. Additionally, GvHD is treated systemically with
immunosuppressive agents, with only about half of patients
responding to treatment. The systemic use of these agents increases
a patient’s risk of severe infection from bacteria, viruses and
fungi as well as cancer relapse, further compromising the curative
potential of allogeneic HCT.
The FDA provides orphan designation to drugs and biologics which
are intended for the safe and effective treatment, diagnosis or
prevention of rare diseases and disorders that affect fewer than
200,000 people in the United States. Orphan drug designation would
provide seven years of market exclusivity in the United States,
with certain exceptions, if market approval is granted for
ProTmune. Orphan designation also qualifies a company for
various development incentives, including tax credits for qualified
clinical testing and a waiver of PDUFA filing fees.
About ProTmune™ProTmune™ is an investigational
programmed cellular immunotherapy undergoing clinical development
for the prevention of acute GvHD and cytomegalovirus (CMV)
infection in patients undergoing allogeneic HCT. The cell therapy
is produced by modulating a donor-sourced, mobilized peripheral
blood graft ex vivo with two small molecules (FT1050 and FT4145) to
enhance the biological properties and therapeutic function of the
graft’s immune cells. The programmed mobilized peripheral blood
graft is administered to a patient as a one-time intravenous
infusion.
About Fate Therapeutics, Inc.Fate Therapeutics
is a biopharmaceutical company dedicated to the development of
programmed cellular immunotherapies for cancer and immune
disorders. The Company’s cell therapy pipeline is comprised of
immuno-oncology programs, including off-the-shelf NK- and T-cell
cancer immunotherapies derived from engineered induced pluripotent
cells, and immuno-regulatory programs, including hematopoietic cell
immunotherapies for protecting the immune system of patients
undergoing hematopoietic cell transplantation and for regulating
autoimmunity. Its adoptive cell therapy programs are based on the
Company’s novel ex vivo cell programming approach, which it applies
to modulate the therapeutic function and direct the fate of immune
cells. Fate Therapeutics is headquartered in San Diego, CA. For
more information, please visit www.fatetherapeutics.com.
Forward-Looking StatementsThis release contains
"forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements
regarding the therapeutic and market potential of ProTmune™, the
Company’s progress and plans for its clinical investigation of
ProTmune, and the ability of ProTmune to prevent, or reduce the
incidence or severity of, acute graft-versus-host disease, severe
infections, including cytomegalovirus infection, and disease
relapse. These and any other forward-looking statements in this
release are based on management's current expectations of future
events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
These risks and uncertainties include, but are not limited to, the
risk of cessation or delay of planned development and clinical
activities for a variety of reasons (including any delay in
initiating or enrolling patients in clinical trials, or the
occurrence of any adverse events or other results that may be
observed during development), the risk that results observed in
prior preclinical studies of ProTmune may not be replicated in
subsequent studies or clinical trials, the risk that ProTmune may
not produce therapeutic benefits or may cause other unanticipated
adverse effects, and the risk that the Company may allocate its
financial and other resources to programs or product candidates
that ultimately have less therapeutic or commercial potential than
other product opportunities. For a discussion of other risks and
uncertainties, and other important factors, any of which could
cause the Company’s actual results to differ from those contained
in the forward-looking statements, see the risks and uncertainties
detailed in the Company’s periodic filings with the Securities and
Exchange Commission, including but not limited to the Company’s
most recently filed periodic report and, from time to time, in the
Company’s other investor communications. The Company is
providing the information in this release as of this date and does
not undertake any obligation to update any forward-looking
statements contained in this release as a result of new
information, future events or otherwise.
Contact:
Jesse Baumgartner
Stern Investor Relations, Inc.
212.362.1200
jesse@sternir.com
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