Tab-cel® U.S. BLA Accepted Under Priority
Review With PDUFA Action Date of January 15, 2025
ATA3219 Lupus Nephritis and Severe Systemic
Lupus Erythematosus Study Initiation Expected Q4 2024; Initial
Clinical Data Expected Mid-2025
Enrolling ATA3219 Non-Hodgkin’s Lymphoma Study;
Initial Clinical Data Expected Q1 2025
Effective September 9, 2024, Pascal Touchon To
Assume Role of Chairman of the Board of Directors of Atara; Cokey
Nguyen, Ph.D., Currently Chief Scientific and Technical Officer, To
Be Appointed President and CEO
Cash Runway Into 2027 Enables Key Pipeline
Readouts
Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell
immunotherapy, leveraging its novel allogeneic Epstein-Barr virus
(EBV) T-cell platform to develop transformative therapies for
patients with cancer and autoimmune diseases, today reported
financial results for the second quarter 2024, recent business
highlights, and key upcoming milestones for 2024.
“Building on the recent BLA acceptance with Priority Review for
tab-cel, we are making significant progress with the agency towards
the target action date of January 15, 2025, while supporting our
partner Pierre Fabre with their U.S. launch preparation,” said
Pascal Touchon, President and Chief Executive Officer of Atara.
“During the quarter we continued to advance the clinical
development of our lead CAR T program, ATA3219, and remain on track
to deliver key value creating milestones within the next 12 months.
This is highlighted by initial data from our non-Hodgkin’s lymphoma
study anticipated in the first quarter of 2025, which we believe
will provide a read-through for ATA3219’s potential in autoimmune
disease. On that front, we plan to initiate our ATA3219 Systemic
Lupus Erythematosus trial in the fourth quarter, including the
cohort without lymphodepletion, with initial data expected in
mid-2025.”
Dr. Touchon continued, “Following the landmark milestone of the
world’s first-ever approval of an allogeneic T-cell therapy and
with the potential first U.S. approval approaching, we are
advancing our differentiated allogeneic CAR-T programs into the
clinic. With the Company in a strong position, I have decided to
move into the role of Chairman for personal reasons to dedicate
more time to my family. I look forward to having a very active and
strategic advisory role as board chair and continuing to help Atara
create value with the potential tab-cel U.S. approval and initial
clinical data with ATA3219. I believe the future of Atara is bright
under the leadership of Cokey Nguyen, Ph.D. who will be promoted to
the role of President and CEO. Cokey is a visionary leader in the
cell therapy field, and our Board of Directors values his deep
commitment to our staff and to patients as well as his expertise
across the breadth of our business.”
“I admire the strong foundation we built under Pascal’s
leadership. I am honored to serve as Atara’s CEO at this pivotal
time to continue our journey to get tab-cel approved in the U.S.
and to unlock the disruptive potential of our allogeneic CAR-T
platform,” said Cokey Nguyen, Ph.D. “I look forward to working
alongside our world-class and innovative teams to rapidly innovate
and strive to develop better cell therapy treatment options for
patients.”
Tabelecleucel (tab-cel® or Ebvallo™) for Post-Transplant
Lymphoproliferative Disease (PTLD)
- U.S. Food and Drug Administration (FDA) accepted the filing of
Atara’s Biologics License Application (BLA) for tabelecleucel
(tab-cel®) indicated as monotherapy for treatment of adult and
pediatric patients two years of age and older with Epstein-Barr
virus positive post-transplant lymphoproliferative disease (EBV+
PTLD) who have received at least one prior therapy. For solid organ
transplant patients, prior therapy includes chemotherapy unless
chemotherapy is inappropriate
- The BLA has been granted Priority Review with a Prescription
Drug User Fee Act (PDUFA) target action date of January 15,
2025
- The data package for the filing includes pivotal and supportive
data covering more than 430 patients treated with tab-cel across
multiple life-threatening diseases
- The BLA submission is supported by the latest pivotal ALLELE
study data-cut that demonstrated a statistically significant 48.8%
Objective Response Rate (ORR) (p<0.0001) and favorable safety
profile consistent with previous analyses
- Atara received a $20 million milestone payment from Pierre
Fabre Laboratories in August 2024, following the acceptance of the
tab-cel BLA, with the potential to receive a $60 million milestone
payment from Pierre Fabre contingent upon FDA approval of the
tab-cel BLA
ATA3219: CD19 Program in Lupus Nephritis (LN)
- Atara expects to initiate a Phase 1 study of ATA3219 as a
monotherapy for the treatment of systemic lupus erythematosus (SLE)
with kidney involvement (lupus nephritis [LN]) in Q4 2024 with
initial clinical data anticipated in mid-2025
- The Phase 1 open-label, dose-escalation study is designed to
evaluate safety, preliminary efficacy, pharmacokinetics, and
biomarkers of a single dose of ATA3219 administered to LN subjects
refractory to one or more lines of treatment. Subjects will receive
lymphodepletion treatment followed by ATA3219 at a dose of 40, 80,
or 160 x 106 CAR+ T cells. Each dose level is designed to enroll
3-6 subjects
- Atara is positioned to potentially expand ATA3219 Phase 1 study
into additional autoimmune indications via the same Investigational
New Drug (IND) application previously cleared for the LN study
- Preclinical data supporting the potential of ATA3219 in SLE was
presented in poster presentation at the International Society for
Cell & Gene Therapy meeting. The data demonstrated that ATA3219
CAR T cells led to complete CD19-specific B-cell depletion against
SLE or multiple sclerosis patient peripheral blood mononuclear
cells
- Additional preclinical data presented in the poster showed that
ATA3219 CAR T cells, which incorporate the next-generation 1XX
costimulatory domain, released lower levels of pro-inflammatory
cytokines while maintaining cytotoxic function and potency in
response to stimulation with CD19+ target cells when compared to
autologous CAR T controls. Mitigating inflammatory cytokine release
that is typically seen with standard CD19 CAR T signaling may lead
to reduced toxicity and better tolerability if confirmed in
clinical trials
ATA3219: CD19 Program in Severe Systemic Lupus Erythematosus
(SLE) Without Lymphodepletion
- Atara plans to expand the Phase 1 LN study of ATA3219 and add a
new cohort in severe SLE without lymphodepletion (LD) in Q4 2024
with initial clinical data anticipated in mid-2025
- Eligible subjects with severe SLE will receive ATA3219 at a
dose of 40, 80, or 240 × 106 CAR+ T cells
- The elimination of LD is designed to further simplify the
treatment regimen and to potentially provide a differentiated
safety profile to patients without comprising efficacy which may
improve patient access
- There is compelling clinical and scientific rationale
supporting the potential to eliminate the need for LD based on the
EBV T-cell backbone and unique features of ATA3219, including: 1)
low alloreactivity risk and favorable safety in over 600 patients
treated without LD, due to T-cell receptor EBV specificity and
partial human leukocyte antigen matching; 2) expansion and
persistence data without LD correlating to efficacy in patients
treated with tab-cel; and 3) the inclusion of clinically validated
features into ATA3219 such as the 1XX costimulatory domain and
memory phenotype that increase potency and persistence
ATA3219: CD19 Program in Non-Hodgkin’s Lymphoma (NHL)
- Atara continues opening sites and initiating enrollment of a
multi-center, Phase 1 open-label, dose-escalation clinical trial of
ATA3219 in NHL, including large B-cell lymphomas, follicular
lymphoma, and mantle cell lymphoma, with initial clinical data
anticipated in Q1 2025
- Study designed to evaluate safety, preliminary efficacy,
pharmacokinetics, and biomarkers. Subjects will receive LD
treatment followed by ATA3219 at a dose of 40, 80, 240, or 480 x
106 CAR+ T cells. Each dose level is designed to enroll 3-6
patients
- Previously presented preclinical data demonstrated superior in
vivo persistence and CD19-specific anti-tumor efficacy compared to
an autologous CD19 CAR T benchmark with no observed toxicity or
alloreactivity
ATA3431: CD19/CD20 Program for B-Cell Malignancies
- Preclinical data presented at ASH 2023 demonstrated early
evidence of potent antitumor activity, long-term persistence, and
superior tumor growth inhibition compared to an autologous
CD19/CD20 CAR T benchmark
- Dual CD19 and CD20 targeting designed to address CD19 escape
and tumor variability and may provide additional efficacy in
lymphoma
- Atara is progressing toward an IND submission in H2 2025
Leadership Updates
- Effective September 9, 2024:
- Cokey Nguyen, Ph.D., the Company's current Chief Scientific and
Technical Officer, will be promoted to the role of President and
CEO and Pascal Touchon, the Company's current President and CEO,
will transition to the role of Chairman of the Board of
Directors
- Cokey Nguyen, Ph.D., will be appointed to the Company’s Board
of Directors
- Carol Gallagher, Pharm.D., current Chair of the Board, will
transition to become Independent Lead Director
Second Quarter 2024 Financial Results
- Cash, cash equivalents and short-term investments as of June
30, 2024 totaled $35.3 million, as compared to $46.2 million as of
March 31, 2024
- Q2 2024 accounts receivable totaled $2.4 million; however, this
amount does not include the $20 million milestone payment owed by
Pierre Fabre related to the tab-cel BLA acceptance, which was
received in August 2024
- On August 9, 2024, Pierre Fabre and Atara entered into an
agreement for Pierre Fabre to purchase certain existing tab-cel
intermediate inventory from Atara for $15.5 million, which is
expected to be received from Pierre Fabre in September 2024
- Together, cash, cash equivalents, short-term investments,
accounts receivable as of June 30, 2024, the $20 million tab-cel
BLA acceptance milestone payment, and the $15.5 million tab-cel
intermediate inventory purchase amount total $73.2 million
- Net cash used in operating activities was $10.6 million for the
second quarter 2024, as compared to $52.8 million in the same
period in 2023
- Q2 2024 net cash used in operating activities included a $20
million cash payment received from Pierre Fabre for a milestone
payment achieved in March 2024, whereas Q2 2023 had no such cash
receipts
- Total revenues were $28.6 million for the second quarter 2024,
as compared to $1.0 million for the same period in 2023. Total
revenues increased by $27.6 million year over year, primarily due
to revenue recognized as a result of additional obligations for the
expanded partnership with Pierre Fabre and accelerated recognition
of existing deferred revenue due to the planned transition of
substantially all activities relating to tab-cel at the time of BLA
approval and transfer to Pierre Fabre
- Total costs and operating expenses include non-cash stock-based
compensation, depreciation and amortization expenses of $7.7
million for the second quarter 2024, as compared to $13.8 million
for the same period in 2023
- Research and development expenses were $33.3 million for the
second quarter 2024, as compared to $56.1 million for the same
period in 2023
- Research and development expenses include $3.3 million of
non-cash stock-based compensation expenses for the second quarter
2024, as compared to $7.2 million for the same period in 2023
- General and administrative expenses were $8.9 million for the
second quarter 2024, as compared to $13.3 million for the same
period in 2023
- General and administrative expenses include $3.0 million of
non-cash stock-based compensation expenses for the second quarter
2024, as compared to $5.4 million for the same period in 2023
- Atara reported net losses of $19.0 million, or $3.10 per share,
for the second quarter 2024, as compared to $71.1 million, or
$16.91 per share, for the same period in 2023
2024 Outlook and Cash Runway
- Atara expects full year 2024 operating expenses to decrease by
approximately 35% from 2023
- The large majority of the year-over-year operating expense
reduction began in Q2 2024 and is expected to continue for the
remainder of the year
- Atara expects that cash, cash equivalents, short-term
investments, and accounts receivable as of June 30, 2024, plus the
items noted below, in total will enable funding of planned
operations into 2027:
- $20 million milestone payment for the acceptance of the tab-cel
BLA received from Pierre Fabre in August 2024 and $60 million
contingent upon the approval of the tab-cel BLA;
- $15.5 million purchase by Pierre Fabre of tab-cel intermediate
inventory to be received in September 2024 and additional
anticipated purchases of tab-cel inventory through the
manufacturing transfer date by Pierre Fabre;
- anticipated reimbursement for tab-cel global development costs
through the BLA transfer by Pierre Fabre;
- operating efficiencies resulting from completed workforce
reductions;
- the planned transition of substantially all activities relating
to tab-cel at the time of the BLA transfer to Pierre Fabre
potentially as early as Q1 2025, which will further reduce
quarterly operating expenses; and
- anticipated royalties from sales of tab-cel by Pierre Fabre in
the U.S. post BLA approval
About ATA3219
ATA3219 combines the natural biology of unedited T cells with
the benefits of an allogeneic therapy. It consists of allogeneic
Epstein-Barr virus (EBV)-sensitized T cells that express a CD19 CAR
construct for the treatment of CD19+ relapsed or refractory B-cell
malignancies, including B-cell non-Hodgkin’s lymphoma and B-cell
mediated autoimmune diseases including systemic lupus
erythematosus. ATA3219 has been optimized to offer a potential
best-in-class profile, featuring off-the-shelf availability. It
incorporates multiple clinically validated technologies including a
modified CD3� signaling domain (1XX) that optimizes expansion and
mitigates exhaustion, enrichment during manufacturing for a less
differentiated phenotype for robust expansion and persistence and
retains the endogenous T-cell receptor without gene editing as a
key survival signal for T cells contributing to persistence.
About ATA3431
ATA3431 is an allogeneic, bispecific CAR directed against CD20
and CD19, built on Atara’s EBV T-cell platform. The design consists
of a tandem CD20-CD19 design, with binders oriented to optimize
potency. Dual targets address the limitations of single antigen
loss and tumor variability. ATA3431 features a novel 1XX
costimulatory domain, memory phenotype, and retained, unedited
T-cell receptor. Preclinical data have demonstrated early evidence
of antitumor activity, long-term persistence, and superior tumor
growth inhibition compared to an autologous CD19/CD20 CAR T
benchmark.
Next-Generation Allogeneic CAR T Approach
Atara is focused on applying Epstein-Barr virus (EBV) T-cell
biology, featuring experience in over 600 patients treated with
allogeneic EBV T cells, and novel chimeric antigen receptor (CAR)
technologies to meet the current limitations of autologous and
allogeneic CAR therapies head-on by advancing a potential
best-in-class CAR T pipeline in oncology and autoimmune disease.
Unlike gene-edited approaches aimed at inactivating T-cell receptor
(TCR) function to reduce the risk for graft-vs-host disease,
Atara’s allogeneic platform maintains expression of the native EBV
TCR that promote in vivo functional persistence while also
demonstrating inherently low alloreactivity due to their
recognition of defined viral antigens and partial human leukocyte
antigen (HLA) matching. A molecular toolkit of clinically-validated
technologies—including the 1XX costimulatory domain designed for
better cell fitness and less exhaustion while maintaining
stemness—offers a differentiated approach to addressing significant
unmet need with the next generation CAR T.
About Atara Biotherapeutics, Inc.
Atara is harnessing the natural power of the immune system to
develop off-the-shelf cell therapies for difficult-to-treat cancers
and autoimmune conditions that can be rapidly delivered to patients
from inventory. With cutting-edge science and differentiated
approach, Atara is the first company in the world to receive
regulatory approval of an allogeneic T-cell immunotherapy. Our
advanced and versatile T-cell platform does not require T-cell
receptor or HLA gene editing and forms the basis of a diverse
portfolio of investigational therapies that target EBV, the root
cause of certain diseases, in addition to next-generation
AlloCAR-Ts designed for best-in-class opportunities across a broad
range of hematological malignancies and B-cell driven autoimmune
diseases. Atara is headquartered in Southern California. For more
information, visit atarabio.com and follow @Atarabio on X and
LinkedIn.
Forward-Looking Statements
This press release contains or may imply "forward-looking
statements" within the meaning of Section 27A of the Securities Act
of 1933 and Section 21E of the Securities Exchange Act of 1934. For
example, forward-looking statements include statements regarding:
(1) the development, timing and progress of tab-cel®, including the
BLA and potential indications, the potential characteristics and
benefits of tab-cel®, and the progress and results of, and
prospects for, the global partnership with Pierre Fabre
Laboratories involving tab-cel®, and the potential financial
benefits to Atara as a result of the global partnership with Pierre
Fabre Laboratories, including the receipt, timing and amount of any
payments to be received by Atara thereunder; (2) the development,
timing and progress of Atara’s AlloCAR-T programs (including
ATA3219 and ATA3431), including the timing of the start of any
clinical trials, the timing of the availability of data from such
clinical trials, the timing of submissions of regulatory
applications, and the potential benefits, characteristics, safety
and efficacy of such product candidates or product candidates
emerging from such programs; (3) Atara’s cash runway, the timing
and receipt of potential milestone and other payments, and
operating expenses, including Atara’s ability to fund its planned
operations into 2027; and (4) Atara’s planned transition of
substantially all activities relating to tab-cel at the time of the
BLA transfer to Pierre Fabre and the timing thereof. Because such
statements deal with future events and are based on Atara’s current
expectations, they are subject to various risks and uncertainties
and actual results, performance or achievements of Atara could
differ materially from those described in or implied by the
statements in this press release. These forward-looking statements
are subject to risks and uncertainties, including, without
limitation, risks and uncertainties associated with the costly and
time-consuming pharmaceutical product development process and the
uncertainty of clinical success; the COVID-19 pandemic and the wars
in Ukraine and the Middle East, which may significantly impact (i)
our business, research, clinical development plans and operations,
including our operations in Southern California and Denver and at
our clinical trial sites, as well as the business or operations of
our third-party manufacturer, contract research organizations or
other third parties with whom we conduct business, (ii) our ability
to access capital, and (iii) the value of our common stock; the
sufficiency of Atara’s cash resources and need for additional
capital; and other risks and uncertainties affecting Atara’s and
its development programs, including those discussed in Atara’s
filings with the Securities and Exchange Commission, including in
the “Risk Factors” and “Management’s Discussion and Analysis of
Financial Condition and Results of Operations” sections of the
Company’s most recently filed periodic reports on Form 10-K and
Form 10-Q and subsequent filings and in the documents incorporated
by reference therein. Except as otherwise required by law, Atara
disclaims any intention or obligation to update or revise any
forward-looking statements, which speak only as of the date hereof,
whether as a result of new information, future events or
circumstances or otherwise.
Financials
ATARA BIOTHERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(Unaudited)
(In thousands)
June 30,
December 31,
2024
2023
Assets
Current assets:
Cash and cash equivalents
$
31,314
$
25,841
Short-term investments
3,978
25,884
Restricted cash
146
146
Accounts receivable
2,422
34,108
Inventories
18,749
9,706
Other current assets
5,801
6,184
Total current assets
62,410
101,869
Property and equipment, net
2,317
3,856
Operating lease assets
48,948
54,935
Other assets
3,609
4,844
Total assets
$
117,284
$
165,504
Liabilities and stockholders’ equity
(deficit)
Current liabilities:
Accounts payable
$
5,253
$
3,684
Accrued compensation
7,269
11,519
Accrued research and development
expenses
2,014
17,364
Deferred revenue
107,582
77,833
Other current liabilities
26,149
31,826
Total current liabilities
148,267
142,226
Deferred revenue - long-term
567
37,562
Operating lease liabilities -
long-term
38,703
45,693
Liability related to the sale of future
revenues - long-term
36,448
34,623
Other long-term liabilities
4,167
4,631
Total liabilities
$
228,152
$
264,735
Stockholders’ (deficit) equity:
Common stock
—
—
Additional paid-in capital
1,909,097
1,870,123
Accumulated other comprehensive loss
(14
)
(204
)
Accumulated deficit
(2,019,951
)
(1,969,150
)
Total stockholders’ (deficit) equity
(110,868
)
(99,231
)
Total liabilities and stockholders’
(deficit) equity
$
117,284
$
165,504
ATARA BIOTHERAPEUTICS,
INC.
Condensed Consolidated
Statements of Operations and Comprehensive Loss
(Unaudited)
(In thousands, except per
share amounts)
Three Months Ended June
30,
Six Months Ended June
30,
2024
2023
2024
2023
Commercialization revenue
$
28,640
$
793
$
55,997
$
1,677
License and collaboration revenue
—
164
—
506
Total revenue
28,640
957
55,997
2,183
Costs and operating expenses:
Cost of commercialization revenue
4,627
2,895
6,612
3,111
Research and development expenses
33,332
56,141
78,838
118,297
General and administrative expenses
8,912
13,335
20,025
27,207
Total costs and operating expenses
46,871
72,371
105,475
148,615
Loss from operations
(18,231
)
(71,414
)
(49,478
)
(146,432
)
Interest and other income (expense),
net
(818
)
307
(1,299
)
576
Loss before provision for income taxes
(19,049
)
(71,107
)
(50,777
)
(145,856
)
Provision for income taxes
—
1
24
23
Net loss
$
(19,049
)
$
(71,108
)
$
(50,801
)
$
(145,879
)
Other comprehensive gain (loss):
Unrealized gain (loss) on
available-for-sale securities
41
304
190
1,134
Comprehensive loss
$
(19,008
)
$
(70,804
)
$
(50,611
)
$
(144,745
)
Basic and diluted net loss per common
share
$
(3.10
)
$
(16.91
)
$
(8.64
)
$
(34.89
)
Basic and diluted weighted-average shares
outstanding
6,143
4,204
5,883
4,181
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240812401307/en/
Investor and Media Relations: Jason Awe, Ph.D. Head of
Corporate Communications & Investor Relations (805) 217-2287
jawe@atarabio.com
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