Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage
biopharmaceutical company dedicated to bringing a first-in-class
pipeline of induced pluripotent stem cell (iPSC)-derived cellular
immunotherapies to patients with cancer and autoimmune diseases,
today announced that the first patient with systemic lupus
erythematosus (SLE) has been treated in the Phase 1 autoimmunity
study of FT819, the Company’s off-the-shelf, CD19-targeted chimeric
antigen receptor (CAR) T-cell program. In addition, at the American
Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, the
Company today presented translational data from the Phase 1 study
of FT819 in relapsed / refractory B-cell malignancies (BCM) and
initial clinical observations from the Phase 1 study of its FT522
off-the-shelf, CD19-targeted CAR NK cell program in relapsed /
refractory B-cell lymphoma (BCL). Data from these programs
highlight the scientific rationale and demonstrate key therapeutic
mechanisms of activity for the treatment of B cell-mediated
autoimmune disease.
The multi-center, Phase 1 autoimmunity study of
FT819 is designed to assess safety, pharmacokinetics, and anti-B
cell activity for patients with moderate-to-severe SLE
(NCT06308978). The first patient, a 27 year-old woman diagnosed
with SLE over ten years ago who has refractory disease despite
having been treated with multiple standard-of-care therapies,
received conditioning chemotherapy followed by a single dose of
FT819 at 360 million cells. The patient was discharged after a
three-day hospital stay without any notable adverse events. In a
"first-of-kind" translational assessment using a sample of the
patient’s blood obtained prior to administration of conditioning
chemotherapy, FT819 induced rapid and potent depletion of the
patient’s CD19+ B cells in an ex vivo cytotoxicity assay.
“The seminal data with autologous CAR-T cell
therapy demonstrating early and long-lasting remissions in patients
with certain B cell-mediated autoimmune diseases is remarkable, and
we are very excited to bring potentially novel therapeutic
solutions with disease-modifying potential to our patients”, said
Jennifer Medlin, M.D., and Principal Investigator at the University
of Nebraska Medical Center. “These solutions may extend to
off-the-shelf cell products, such as FT819, which may have the
potential to overcome critical challenges that could limit patient
access to CAR-T for autoimmune diseases, such as the requirement
for apheresis, conditioning chemotherapy, extended hospitalization,
and risk of significant adverse events including secondary
malignancies.”
“We are excited to bring our iPSC product platform
and our first product candidates to patients with autoimmune
diseases, where preclinical and translational data from our
off-the-shelf FT819 CAR T-cell program and our FT522 CAR NK cell
program demonstrate key therapeutic mechanisms of activity for
autoimmunity,” said Scott Wolchko, President and Chief Executive
Officer of Fate Therapeutics. “We believe these programs have a
favorable safety profile, offer patient access and convenience, and
can deliver the breadth and depth of B cell depletion necessary to
induce immune reset in patients with B-cell mediated autoimmune
diseases.”
Translational Data for FT819 iPSC-derived
CAR T-cell Program
FT819 is the Company’s off-the-shelf,
CD19-targeted, 1XX CAR T-cell product candidate comprised of CD8αβ+
T cells with a memory phenotype and high CXCR4 expression to
promote tissue trafficking. Translational data presented today at
ASGCT from the Company’s Phase 1 BCM study show that a single dose
of FT819 exhibited multiple therapeutic mechanisms implicated in
generating an immune reset in patients with B cell-mediated
autoimmune disease. Clinical data highlighted today at ASGCT
include:
- Blood samples taken from 23 patients
treated for relapsed / refractory B cell lymphoma showed rapid and
deep CD19+ B cell depletion, with sustained suppression of B cells,
in the periphery during the initial 30-day period following
administration of standard conditioning chemotherapy and
FT819;
- Patient case studies demonstrating
secondary and tertiary tissue trafficking, infiltration, and
activity, with complete elimination of CD19+ cells in tissue;
and
- Patient case studies of plasma cell
depletion and B-cell reconstitution showing recovery of naïve and
immature phenotypes, with little to no recovery of activated memory
B cells or plasmablasts.
Notably, the Company also presented patient case
studies demonstrating the capacity of FT819 to induce rapid, deep,
and sustained B-cell depletion without the use of fludarabine as a
conditioning agent. Collectively, these data support the potential
of FT819 to reset the immune system of patients with autoimmune
diseases, including as an add-on therapy to commonly-used treatment
regimens. The presentation is available on the Company’s website
here.
Preclinical and Initial Clinical
Observations for FT522 iPSC-derived CAR NK Cell Program
Data
FT522 is the Company’s off-the-shelf, CD19-targeted
CAR NK cell product candidate and its first to incorporate a novel
alloimmune defense receptor (ADR), which is designed to increase
the potency of off-the-shelf cell therapy and enable effective
treatment without administration of conditioning chemotherapy to
patients. Data highlighted today at ASGCT include:
- In a novel re-challenge assay using
peripheral blood mononuclear cells (PBMCs) from unmatched SLE
donors, FT522 uniquely drove rapid and deep CD19+ B cell depletion,
eliminated alloreactive T cells, and maintained functional
persistence, indicating that FT522 can function effectively in the
presence of an unmatched host immune system;
- In a preclinical in vivo
biodistribution study, FT522 showed dose-dependent trafficking,
infiltration, and residency in secondary and tertiary tissues
without cytokine support at human dose equivalency levels of 250
million cells per dose and 1 billion cells per dose (based on 20
gram mouse and 65 kilogram human allometric conversion); and
- In initial clinical observations from
the Company’s ongoing Phase 1 BCL study, the first two patients
treated with FT522 showed rapid, deep, and sustained B-cell
depletion in the periphery throughout the one-month treatment
cycle. In addition, both patients showed enhanced persistence of
FT522 in the periphery compared to clinical data observed with
FT596, a prior-generation CD19-targeted CAR NK cell without ADR
technology.
The Company intends to submit an Investigational
New Drug (IND) application to the U.S. Food and Drug Administration
(FDA) in the middle of 2024 for the treatment of various autoimmune
diseases with FT522, including without administration of
conditioning chemotherapy to patients. The presentation is
available on the Company’s website here.
About Fate Therapeutics’ iPSC Product
PlatformHuman induced pluripotent stem cells (iPSCs)
possess the unique dual properties of unlimited self-renewal and
differentiation potential into all cell types of the body. The
Company’s proprietary iPSC product platform combines
multiplexed-engineering of human iPSCs with single-cell selection
to create clonal master iPSC lines. Analogous to master cell lines
used to mass produce biopharmaceutical drug products such as
monoclonal antibodies, the Company utilizes its clonal master iPSC
lines as a starting cell source to manufacture engineered cell
products which are well-defined and uniform in composition, can be
stored in inventory for off-the-shelf availability, can be combined
and administered with other therapies, and can potentially reach a
broad patient population. As a result, the Company’s platform is
uniquely designed to overcome numerous limitations associated with
the manufacture of cell therapies using patient- or donor-sourced
cells. Fate Therapeutics’ iPSC product platform is supported by an
intellectual property portfolio of over 500 issued patents and 500
pending patent applications.
About Fate Therapeutics, Inc.Fate
Therapeutics is a clinical-stage biopharmaceutical company
dedicated to bringing a first-in-class pipeline of induced
pluripotent stem cell (iPSC)-derived cellular immunotherapies to
patients with cancer and autoimmune diseases. Using its proprietary
iPSC product platform, the Company has established a leadership
position in creating multiplexed-engineered master iPSC lines and
in the manufacture and clinical development of off-the-shelf,
iPSC-derived cell products. The Company’s pipeline includes
iPSC-derived natural killer (NK) cell and T-cell product
candidates, which are selectively designed, incorporate novel
synthetic controls of cell function, and are intended to deliver
multiple therapeutic mechanisms to patients. 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 advancement of and plans related to the
Company’s product candidates, clinical studies and preclinical
research and development programs, the Company’s progress, plans
and timelines for the clinical investigation of its product
candidates, including the initiation and continuation of enrollment
in the Company’s clinical trials, the timing and availability of
data from the Company’s clinical trials, the therapeutic and market
potential of the Company’s research and development programs and
product candidates, and the potential capabilities and benefits of
the Company’s iPSC product platform. 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 that the
Company’s research and development programs and product candidates,
including those product candidates in clinical investigation, may
not demonstrate the requisite safety, efficacy, or other attributes
to warrant further development or to achieve regulatory approval,
the risk that results observed in prior studies of the Company’s
product candidates, including preclinical studies and clinical
trials, will not be observed in ongoing or future studies involving
these product candidates, the risk of a delay or difficulties in
the manufacturing of the Company’s product candidates or in the
initiation and conduct of, or enrollment of patients in, any
clinical trials, the risk that the Company may cease or delay
preclinical or clinical development of any of its product
candidates for a variety of reasons (including requirements that
may be imposed by regulatory authorities on the initiation or
conduct of clinical trials, changes in the therapeutic, regulatory,
or competitive landscape for which the Company’s product candidates
are being developed, the amount and type of data to be generated or
otherwise to support regulatory approval, difficulties or delays in
patient enrollment and continuation in the Company’s ongoing and
planned clinical trials, difficulties in manufacturing or supplying
the Company’s product candidates for clinical testing, failure to
demonstrate that a product candidate has the requisite safety,
efficacy, or other attributes to warrant further development, and
any adverse events or other negative results that may be observed
during preclinical or clinical development), and the risk that its
product candidates may not produce therapeutic benefits or may
cause other unanticipated adverse effects. 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 press releases and other investor
communications. Fate Therapeutics 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 TartagliaStern
Investor Relations,
Inc.212.362.1200christina.tartaglia@sternir.com
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