Fate Therapeutics Reports First Quarter 2024 Financial Results and Business Updates
09 Mayo 2024 - 3:01PM
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 reported business highlights and financial results for the
first quarter ended March 31, 2024.
“We made great progress across three key areas of clinical focus
– we treated the first patient with our off-the-shelf FT819 CAR
T-cell therapy for autoimmunity, we initiated patient enrollment
without conditioning chemotherapy for our ADR-armed FT522 CAR NK
cell therapy, and we treated the first patient with our
multiplexed-engineered FT825 CAR T-cell therapy for solid tumors,”
said Scott Wolchko, President and Chief Executive Officer of Fate
Therapeutics. “As we look toward the middle of the year, we plan to
expand clinical development in autoimmunity with our off-the-shelf
FT819 and FT522 programs, where we believe our iPSC product
platform is highly differentiated and has the potential to overcome
numerous challenges that hinder treatment of patients with cell
therapies, such as the requirement for apheresis and Cy / Flu
conditioning, extended hospitalization, risk of secondary
malignancies, and limited access. In addition, while we continue
with the preclinical assessment of our next-generation,
BCMA-targeted cell product candidates, we do not intend to further
advance FT576 into Phase 1 dose expansion in relapsed / refractory
multiple myeloma.”
FT819 iPSC-derived CAR T-cell Program
- First SLE Patient Treated in
Phase 1 Autoimmunity Study. The multi-center, Phase 1
clinical trial for patients with systemic lupus erythematosus (SLE)
is designed to evaluate the safety, pharmacokinetics, and anti-B
cell activity of FT819, the Company’s off-the-shelf CD8αβ+ T-cell
product candidate that incorporates a novel CD19-targeted 1XX
chimeric antigen receptor (CAR) construct into the T-cell receptor
alpha constant (TRAC) locus (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.
- Phase 1 BCM Study Data
Establish Proof-of-Concept for Autoimmune
Disease. At the American Society of Gene and
Cell Therapy (ASGCT) 27th Annual Meeting, the Company today
presented preclinical and translational data from its FT819 Phase 1
study in relapsed / refractory B cell malignancies (BCM), which
showed that a single dose of FT819 exhibits multiple mechanisms
implicated in generating an immune reset in patients with B
cell-mediated autoimmune diseases. Clinical observations included:
dose-dependent pharmacokinetics, reaching Cmax at Day 8 with
persistence through the first two weeks in the periphery; rapid,
deep, and sustained CD19+ B cell depletion in the peripheral blood
using standard Cy / Flu and alternative conditioning chemotherapy
regimens; patient case studies of secondary and tertiary tissue
trafficking, infiltration, and activity with CD19+ B cell
elimination in tissue; and patient case studies of plasma cell
depletion and B cell reconstitution, which showed recovery of naïve
B cells with little to no recovery of activated memory B cells or
plasmablasts.
- Dose-escalation Completed in
Phase 1 BCM Study with Further Clinical Development to Focus
Exclusively on Autoimmunity. The Company has
successfully completed dose escalation in its Phase 1 BCM study,
demonstrating safety and tolerability of FT819 at a single dose up
to 1.08 billion cells (NCT04629729), and intends to pursue further
clinical development exclusively in autoimmunity. 43 heavily
pre-treated patients (B cell lymphoma, n=25; chronic lymphocytic
leukemia, n=12; and acute lymphocytic leukemia, n=6) were treated
with conditioning chemotherapy and a single dose of FT819 across
five dose levels. The safety and tolerability profile of FT819 was
favorable, with no dose-limiting toxicities, no events of any grade
of immune effector-cell associated neurotoxicity syndrome (ICANS)
or graft-versus-host disease (GvHD), and low incidence (14%) of
only low-grade cytokine release syndrome (CRS). There were no study
discontinuations or deaths related to FT819. Clinical responses
were observed across all three histologies. In 17 patients with
aggressive large B cell lymphoma, 12 (71%) of whom had previously
received autologous CD19-targeted CAR T-cell therapy, the overall
response and complete response rates were 47% and 24%,
respectively.
FT825 / ONO-8250 iPSC-derived CAR T-cell
Program
- First Patient Treated in Phase
1 Study with HER2-targeted CAR T-cell for Advanced Solid
Tumors. Under its collaboration with Ono
Pharmaceutical Co., Ltd. (Ono), the Company is conducting a
multi-center, Phase 1 study to assess the safety, pharmacokinetics,
and activity of FT825 / ONO-8250 as monotherapy and in combination
with monoclonal antibody therapy in patients with advanced solid
tumors (NCT06241456). The first patient was diagnosed with
HER2-positive gastroesophageal junction (GEJ) adenocarcinoma, had
progressed after receiving multiple lines of treatment including
HER2-targeted therapies, and was administered standard conditioning
chemotherapy followed by a single dose of FT825 / ONO-8250 as
monotherapy at 100 million cells. Designed using the Company’s iPSC
product platform, FT825 / ONO-8250 incorporates seven synthetic
controls of cell function including a novel cancer-specific
H2CasMab-2 CAR, which has exhibited similar potency with greater
specificity for cancer cells expressing HER2 compared to
trastuzumab in preclinical studies.
FT522 iPSC-derived CAR NK Cell Program
- Conditioning-free Treatment Arm
of Phase 1 BCL Study Open for Enrollment. FT522 is the
Company’s off-the-shelf, CD19-targeted CAR NK cell product
candidate and its first to incorporate Alloimmune Defense Receptor
(ADR) technology, which is designed to reduce or eliminate the need
for administration of conditioning chemotherapy to patients
receiving cell therapies. In its ongoing multi-center, Phase 1
clinical trial of FT522 in patients with relapsed / refractory
B-cell lymphoma (BCL) (NCT05950334), patient enrollment has now
been initiated in the first three-dose cohort at 300 million cells
per dose without conditioning chemotherapy (Regimen B). Three
patients have been treated with conditioning chemotherapy in the
first three-dose cohort at 300 million cells per dose (Regimen A).
There were no dose-limiting toxicities and no events of any grade
of CRS, ICANS, or GvHD, and dose escalation is ongoing at 900
million cells per dose.
- ASGCT Presentation Data Show
ADR Differentiation and Proof-of-Concept for
Autoimmunity. At the ASGCT conference, the
Company today presented preclinical data as well as early
translational data from its ongoing Phase 1 BCL study. 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. The Company also shared initial clinical observations from
the first two patients treated with FT522 in Regimen A, which
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.
FT576 iPSC-derived CAR NK Cell Program
- Dose Escalation Completed in
Phase 1 Multiple Myeloma Study. The Company has completed
dose escalation in its multi-center, Phase 1 clinical trial of
FT576, its BCMA-targeted CAR NK cell product candidate, for
relapsed / refractory multiple myeloma (NCT05182073). Using a
standard conditioning chemotherapy regimen, 12 patients were
treated with a three-dose treatment schedule at 1 billion cells per
dose (n=6) or at 2.5 billion cells per dose (n=6) as monotherapy or
in combination with CD38-targeted monoclonal antibody therapy.
There were no dose-limiting toxicities and no events of any grade
of CRS, ICANS or GvHD. Response assessment is ongoing at 2.5
billion cells per dose. Five of six (83%) heavily pre-treated
patients achieved a clinical response at 1 billion cells per dose,
including two penta-exposed patients treated with FT576 as
monotherapy that achieved very good partial responses. The Company
is continuing to assess the FT576 Phase 1 dose-escalation dataset
and to preclinically evaluate the potential of its next-generation,
BCMA-targeted cell product candidates, including for the treatment
of multiple myeloma and autoimmune diseases, and does not intend to
further advance FT576 into Phase 1 dose expansion in relapsed /
refractory multiple myeloma.
First Quarter 2024 Financial Results
- Cash & Investment
Position: Cash, cash equivalents and investments as of
March 31, 2024 were $391.1 million, which includes net proceeds
from the closing of the Company’s approximately $80 million
underwritten offering of common stock at $5.50 per share and
approximately $20 million concurrent private placement of
pre-funded warrants at $5.499 per pre-funded warrant in the first
quarter of 2024.
- Total Revenue: Revenue
was $1.9 million for the first quarter of 2024, which was derived
from the Company’s conduct of preclinical development activities
for a second collaboration candidate targeting an undisclosed solid
tumor antigen under its collaboration with Ono.
- Total Operating
Expenses: For the first quarter of 2024, GAAP operating
expenses were $53.0 million, including research and development
expenses of $32.1 million and general and administrative expenses
of $20.9 million. Such amounts included $11.0 million of non-cash
stock-based compensation expense.
- Shares Outstanding:
Common shares outstanding were 113.8 million, pre-funded warrants
outstanding were 3.9 million, and preferred shares outstanding were
2.8 million, as of March 31, 2024. Each preferred share is
convertible into five common shares.
Today's Conference Call and WebcastThe Company
will conduct a conference call today, Thursday, May 9, 2024 at 5:00
p.m. ET to review financial and operating results for the quarter
and full year ended March 31, 2024. In order to participate in the
conference call, please dial (833) 630-1956 (domestic) and (412)
317-1837 (international). The live webcast can be accessed under
"Events & Presentations" in the Investors section of the
Company's website at www.fatetherapeutics.com. The archived webcast
will be available on the Company's website beginning approximately
two hours after the event.
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 Company’s results of operations, financial condition,
anticipated operating expenses and cash runway, and sufficiency of
its cash and cash equivalents to fund its operations, as well as
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 initiation of additional
clinical trials and additional dose cohorts in ongoing clinical
trials of the Company’s product candidates, the 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, the Company’s clinical and product development
strategy, and the Company’s expectations regarding progress and
timelines, and the objectives, plans and goals of its collaboration
with Ono. 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), the risk that its
product candidates may not produce therapeutic benefits or may
cause other unanticipated adverse effects, the risk that the
Company may not comply with its obligations under and otherwise
maintain its collaboration agreement with Ono, the risk that
research funding and milestone payments received by the Company
under its collaboration may be less than expected, and the risk
that the Company may incur operating expenses in amounts greater
than anticipated. 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.
|
Condensed Consolidated Statements of Operations and
Comprehensive Loss(in thousands, except share and
per share data)(unaudited) |
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2024 |
|
|
2023 |
|
|
|
|
|
Collaboration revenue |
|
$ |
1,925 |
|
|
$ |
58,980 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
32,138 |
|
|
|
65,629 |
|
General and administrative |
|
|
20,855 |
|
|
|
21,943 |
|
Total operating expenses |
|
|
52,993 |
|
|
|
87,572 |
|
Loss from operations |
|
|
(51,068 |
) |
|
|
(28,592 |
) |
Other income (expense): |
|
|
|
|
|
|
Interest income |
|
|
4,149 |
|
|
|
3,694 |
|
Change in fair value of stock price appreciation milestones |
|
|
(1,394 |
) |
|
|
1,718 |
|
Other income |
|
|
309 |
|
|
|
4,299 |
|
Total other income (expense),
net |
|
|
3,064 |
|
|
|
9,711 |
|
Net loss |
|
$ |
(48,004 |
) |
|
$ |
(18,881 |
) |
Other comprehensive gain
(loss): |
|
|
|
|
|
|
Unrealized gain (loss) on available-for-sale securities, net |
|
|
(209 |
) |
|
|
1,208 |
|
Comprehensive loss |
|
$ |
(48,213 |
) |
|
$ |
(17,673 |
) |
Net loss per common share,
basic and diluted |
|
$ |
(0.47 |
) |
|
$ |
(0.19 |
) |
Weighted-average common shares used tocompute basic and diluted net
loss per share |
|
|
101,104,345 |
|
|
|
98,054,687 |
|
|
|
|
|
|
|
|
|
Condensed Consolidated Balance Sheets(in
thousands)(unaudited) |
|
|
|
March 31, |
|
|
December 31, |
|
|
|
2024 |
|
|
2023 |
|
|
|
|
|
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
121,322 |
|
|
$ |
41,870 |
|
Accounts receivable |
|
|
858 |
|
|
|
1,826 |
|
Short-term investments |
|
|
262,222 |
|
|
|
273,305 |
|
Prepaid expenses and other current assets |
|
|
9,973 |
|
|
|
14,539 |
|
Total current assets |
|
|
394,375 |
|
|
|
331,540 |
|
Long-term investments |
|
|
7,595 |
|
|
|
980 |
|
Operating lease right-of-use
asset |
|
|
60,620 |
|
|
|
61,675 |
|
Other long-term assets |
|
|
107,302 |
|
|
|
112,022 |
|
Total assets |
|
$ |
569,892 |
|
|
$ |
506,217 |
|
|
|
|
|
|
|
|
Liabilities and
stockholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable and accrued expenses |
|
$ |
38,808 |
|
|
$ |
32,233 |
|
Deferred revenue |
|
|
159 |
|
|
|
685 |
|
Operating lease liability, current portion |
|
|
6,402 |
|
|
|
6,176 |
|
Total current liabilities |
|
|
45,369 |
|
|
|
39,094 |
|
Operating lease liability, net
of current portion |
|
|
95,668 |
|
|
|
97,360 |
|
Stock price appreciation
milestones |
|
|
2,740 |
|
|
|
1,346 |
|
Stockholders’ equity |
|
|
426,115 |
|
|
|
368,417 |
|
Total liabilities and
stockholders’ equity |
|
$ |
569,892 |
|
|
$ |
506,217 |
|
|
|
|
|
|
|
|
Contact:Christina TartagliaStern Investor
Relations, Inc.212.362.1200christina.tartaglia@sternir.com
Fate Therapeutics (NASDAQ:FATE)
Gráfica de Acción Histórica
De May 2024 a Jun 2024
Fate Therapeutics (NASDAQ:FATE)
Gráfica de Acción Histórica
De Jun 2023 a Jun 2024