Fate Therapeutics Initiates Phase 2 PROTECT Study of ProTmune™ for Prevention of Acute Graft-versus-Host Disease
September 18 2017 - 8:00AM
Independent Data Monitoring Committee Conducts
Phase 1 Safety Review and Unanimously Recommends Opening of Phase 2
Enrollment
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 Company
initiated enrollment in the Phase 2 stage of PROTECT, a combined
open-label Phase 1 / blinded Phase 2 clinical trial of ProTmune™
for the prevention of acute graft-versus-host disease (GvHD) in
patients with hematologic malignancies undergoing matched unrelated
donor hematopoietic cell transplantation (HCT). Thirteen U.S.
centers are currently open for enrollment in the Phase 2 stage of
PROTECT.
“Acute GvHD is the leading cause of early morbidity and
mortality in patients undergoing allogeneic transplant. We are
excited to initiate the randomized, controlled and blinded Phase 2
stage of PROTECT and assess the potential of ProTmune to deliver
transformative benefits to cancer patients,” said Chris Storgard,
M.D., Chief Medical Officer of Fate Therapeutics. “We thank the
study’s independent data monitoring committee for its Phase 1 data
review. Once all seven Phase 1 subjects progress to Day 100
post-HCT, we expect to present ProTmune Day 100 efficacy data,
including acute GvHD, cancer relapse and survival, at the 2017
American Society of Hematology annual meeting.”
The Phase 2 stage is assessing the safety and efficacy of
ProTmune in 60 subjects, where subjects are being randomized, in a
1:1 ratio, to receive either ProTmune or a conventional matched
unrelated donor mobilized peripheral blood cell graft. The primary
efficacy endpoint of PROTECT is incidence of acute GvHD by Day 100
post-HCT, where prospective clinical studies have shown that 40% to
80% of patients undergoing matched unrelated donor transplant
experience Grades 2-4 acute GvHD. There are currently no approved
preventive therapies and very few treatment options for acute
GvHD.
PROTECT Phase 1 Safety & Manufacturing Data
On September 14, 2017, the study’s four-member independent data
monitoring committee conducted a review of all available Phase 1
data. Based on its review, the committee unanimously recommended
initiation of the randomized, controlled and blinded Phase 2 stage
of PROTECT.
The Phase 1 review by the committee included data on the first
seven subjects receiving ProTmune. Underlying hematologic diseases
included three subjects with acute lymphoblastic leukemia (ALL),
three with acute myeloid leukemia (AML) and one with
myelodysplastic syndrome (MDS). All subjects met the Day 28 safety
objectives of neutrophil engraftment and survival, and reached Day
28 without any events of graft failure or serious adverse events
related to ProTmune. The median time to neutrophil engraftment was
18 days [14-22 days].
ProTmune was successfully manufactured at four clinical sites.
Cell viability and CD34+ cell recovery (mean +/- SD) were 87.7 +/-
7.5% and 90.5 +/- 14.6%, respectively. The cell-surface protein
CXCR4 (mean +/- SD), a key pharmacological biomarker of product
potency, was expressed on 67.7 +/- 10.2% of CD34+ cells as compared
to approximately 5% of CD34+ cells contained in a conventional
hematopoietic cell graft.
About Acute Graft-versus-Host DiseaseAcute GvHD
is a severe immunological disease that commonly arises in patients
during the first weeks following allogeneic HCT when the
newly-transplanted donor immune cells attack the patient’s tissues
and organs, resulting in a potentially fatal immune system
reaction. Prospective clinical studies have shown that 40% to 80%
of patients undergoing matched unrelated donor transplant
experience Grades 2-4 acute GvHD, with most incidents occurring by
Day 60 post-HCT despite the use of standard prophylaxis regimens.
The disease is the leading cause of early morbidity and mortality
in matched unrelated donor transplant, where death directly
attributable to acute GvHD or its treatment occurs in 10% to 20% of
patients. There are currently no approved preventive therapies and
very few treatment options for acute GvHD.
About ProTmune™ProTmune™ is an investigational
next-generation hematopoietic cell graft for the prevention of
acute GvHD in patients undergoing allogeneic HCT. ProTmune is
manufactured by pharmacologically 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. ProTmune has been granted Orphan
Drug and Fast Track Designations by the U.S. Food and Drug
Administration, and Orphan Medicinal Product Designation by the
European Medicines Agency.
About Allogeneic Hematopoietic Cell
TransplantationThere are approximately 30,000 allogeneic
HCT procedures performed globally each year according to the Center
for International Blood and Marrow Transplant Research. The
procedure is performed with curative intent most often for patients
with acute leukemias and myelodysplastic syndromes. While long-term
curative rates are high for patients achieving disease-free
survival at two years, patients face a multitude of
life-threatening complications, including GvHD and cancer relapse,
during the initial weeks and months following allogeneic HCT. In
fact, the GvHD-free, relapse-free survival (GRFS) rate is only
about 30% during the first year following allogeneic HCT.
About Fate Therapeutics, Inc. Fate Therapeutics
is a clinical-stage biopharmaceutical company dedicated to the
development of programmed cellular immunotherapies for cancer and
immune disorders. The Company's hematopoietic cell therapy pipeline
is comprised of NK- and T-cell immuno-oncology programs, including
off-the-shelf product candidates derived from engineered induced
pluripotent cell lines, and immuno-regulatory programs, including
product candidates to prevent life-threatening complications in
patients undergoing hematopoietic cell transplantation and to
promote immune tolerance in patients with autoimmune disease. 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, the ability of ProTmune to prevent, or reduce the
incidence or severity of, graft-versus-host disease, severe
infections, disease relapse or mortality, the potential safety of
ProTmune in the treatment of diseases, the timing and success of
the Company’s PROTECT clinical trial, including the Company’s
ability to generate Day 100 efficacy data from the Phase 1 stage of
the trial and the timing thereof, and the Company’s ability to
enroll patients in, and conduct, the Phase 2 stage of the trial.
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 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: Christina Tartaglia Stern Investor
Relations, Inc. 212.362.1200 christina@sternir.com
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