Tab-cel U.S. BLA Submission Not Currently
Expected for Q2 2022 as Further FDA Engagement and Alignment
Required; Anticipated EU Approval On-Track for Q4 2022 Under
Accelerated Assessment
ATA188 Granted FDA Fast Track Designation in
Both Non-Active PPMS and Non-Active SPMS with Phase 2 EMBOLD Study
Interim Analysis Planned for Q2 2022
Entered Long-Term Strategic Manufacturing
Partnership with Fujifilm with Upfront Cash Payment of USD 100
Million
Conference Call and Webcast Today at 1:30 p.m.
PST/ 4:30 p.m. EST
Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell
immunotherapy, leveraging its novel allogeneic EBV T-cell platform
to develop transformative therapies for patients with cancer and
autoimmune diseases, today reported financial results for the
fourth quarter and full year 2021, recent business highlights and
key upcoming catalysts for 2022.
“With our EMA filing for tab-cel® and a strategic commercial
collaboration with Pierre Fabre, ATA188’s FDA Fast Track
designations and new data reinforcing its potential to reverse or
halt disability in progressive multiple sclerosis, 2021 provided
strong validation for Atara and our industry-leading allogeneic
T-cell pipeline,” said Pascal Touchon, President and Chief
Executive Officer of Atara. “2022, which has already delivered two
landmark publications confirming EBV as the leading cause of MS in
advance of our interim analysis from our ATA188 Phase 2 study, and
a long-term strategic manufacturing partnership with Fujifilm, will
be a critical year as we progress our strategic priorities.
Although further engagement and alignment is required with FDA to
determine the path forward for a BLA submission in the U.S., we
anticipate a groundbreaking EU approval this year that will
position tab-cel as the first ever allogeneic off-the-shelf T-cell
therapy available for patients.”
Tabelecleucel (tab-cel®) for Post-Transplant
Lymphoproliferative Disease (PTLD)
- Atara has performed extensive studies demonstrating analytical
comparability between the tab-cel manufacturing process versions
used for the pivotal study and that intended for commercialization
- Comprehensive comparability analyses included all 74 available
product lots manufactured by Atara and covered 21 key attributes
associated with potency, purity, and alloreactivity. Atara believes
analytic comparability between tab-cel process versions has been
demonstrated based on well-established statistical methodology and
application of ICH1 guidelines and is further supported by
significant and consistent clinical experience
- These comparability data analyses were submitted to the
European Medicines Agency (EMA) through our MAA filing in November
2021 and the review, under Accelerated Assessment, is progressing
as planned following receipt of EMA day 80 Critical Assessment
report, with anticipated approval in Q4 2022
- A Type B CMC meeting was conducted in late February 2022 with
the U.S. Food & Drug Administration (FDA) review team to
discuss and potentially align on comparability between commercial
and pivotal clinical trial products
- Preliminary meeting responses and discussion did not result in
alignment on comparability and the FDA has initially recommended
Atara conduct a clinical study with commercial product as they do
not agree that comparability has been demonstrated
- Atara has responded with additional questions to clarify the
FDA’s view and has suggested several alternative approaches to
progress to a BLA submission given tab-cel is a BTD product that
addresses an urgent medical need and has the potential to save the
lives of patients with an ultra-rare, often fatal disease with no
approved therapeutic options
- Additional interactions with the Agency are expected, including
receipt of final Type B CMC meeting minutes. However, based on the
preliminary feedback received from the FDA, Atara does not
currently expect to file a BLA in Q2 2022
- Atara expects further engagement with FDA on potential pathways
to a BLA submission for tab-cel and plans to provide a further
update during its next quarterly call
- After entering an exclusive commercialization collaboration in
Q4 2021, Atara is working with Pierre Fabre to prepare for the
launch of tab-cel in Europe while adapting its U.S.
commercialization preparation to account for possible delays in
submission and potential approval of a BLA in the U.S.
Tab-cel for Potential Additional Indications
- The multi-cohort Phase 2 study evaluating tab-cel in six
additional patient populations for EBV+ immunodeficiency-associated
lymphoproliferative diseases (IA-LPDs) and other EBV-driven
diseases is currently enrolling in the U.S. and EU
- First data from the multi-cohort study is on track for
presentation in 2023
ATA188 for Progressive Multiple Sclerosis (MS)
- Recent landmark studies further validate Atara’s approach and
EBV-targeted platform. Publications in Science and Nature provide
compelling new epidemiological evidence that EBV is the leading
cause of MS and mechanistic evidence showing how EBV infection can
initiate and propagate the autoimmune attack on the brain in
MS
- The FDA granted Fast Track designation to ATA188 for non-active
primary progressive multiple sclerosis (PPMS) and non-active
secondary progressive multiple sclerosis (SPMS), two populations
with high unmet medical need and limited treatment options
- Encouraging data presented at ECTRIMS 2021 from the ongoing
Phase 1 open-label extension study of ATA188 with up to 39 months
follow-up demonstrated that 20 out of 24 patients had sustained
disability improvement or stabilization of disease on expanded
disability status scale (EDSS)
- Patients who achieved sustained EDSS improvement at any time
showed significant increase in Magnetization Transfer Ratio (MTR)
in non-enhancing T2 lesions at 12 months compared to baseline,
suggestive of remyelination
- Atara continues to advance enrollment in the Phase 2 EMBOLD
study, with target enrollment of 80 patients expected soon after a
planned formal interim analysis (IA) in Q2 2022
- The IA will include efficacy, safety, and biomarker data to
inform adjustments to sample size, if needed, to optimize
likelihood of Phase 2 success, and confirm the Company’s current
development strategy moving forward
- A key data point at the time of the IA will be EDSS improvement
at six months for applicable patients. In the Phase 1 study, EDSS
improvement at six months was >85 percent predictive of
achieving sustained EDSS improvement at 12 months, the primary
endpoint of EMBOLD
- Following the IA, Atara plans to communicate next steps for the
program, including rationale, while still maintaining the integrity
of the study. Atara also plans to interact with the FDA following
the IA to discuss potential development pathways for ATA188
- Prior to conducting the IA in Q2 2022, the Company will host a
public webcast, Atara MS Day, for the investor community on March
22nd to review the key drivers generating excitement around our
potentially transformative ATA188 MS program. The Company will also
communicate new areas of development, including exploratory work on
a differentiated EBV vaccine with encouraging pre-clinical
data
CAR T Programs
- Atara is building an innovative next-generation CAR T platform,
which includes key features needed for efficacy and persistence,
such as the use of PD-1 dominant negative receptor (DNR) and 1XX
CAR co-stimulatory signaling domain technologies
- The CAR T platform retains the endogenous T-cell receptor
(TCR), shown to be essential for T-cell persistence and
survival
ATA2271/ATA3271 (Solid Tumors Over-Expressing
Mesothelin)
- The global strategic collaboration for autologous ATA2271 and
allogeneic ATA3271 with Bayer continues to progress
- Encouraging early safety and persistence of ATA2271 from lower
dose cohorts of the ongoing Phase 1 dose-escalation trial in
patients with advanced mesothelioma was presented at the ESMO
Immuno-Oncology Congress in December 2021
- Memorial Sloan Kettering Cancer Center (MSK) notified the FDA
of a fatal serious adverse event (SAE) associated with a patient
treated in the third, higher dose cohort in the ongoing Phase 1,
MSK-conducted dose-escalation clinical study of autologous
mesothelin CAR T, ATA2271
- MSK has voluntarily paused enrollment of new patients in the
ATA2271 study on a temporary basis while additional information
regarding the case is gathered and reviewed. The FDA notified MSK
of its agreement with this approach
- Subject to the outcome of this review and resumption of
enrollment of new patients in this study we expect to provide a
data update from this Phase 1 study in 2022
- The single case involved a patient with a history of multiple
malignancies and other comorbidities undergoing treatment for
advanced recurrent mesothelioma
- The temporary pause in ATA2271 study enrollment does not impact
the IND-enabling work currently underway to advance ATA3271, a
separate off-the-shelf, allogeneic CAR-T therapy targeting
mesothelin using next-generation PD-1 DNR and 1XX CAR technologies
for patients with advanced mesothelioma, with an expected IND
filing in Q4 2022
- Atara’s pipeline programs, including ATA3271, ATA3219, tab-cel,
and ATA188 all utilize the Company’s allogeneic EBV T-cell
platform, the safety and tolerability of which has been
demonstrated by clinical studies and experience in approximately
400 patients in various disease areas with no SAEs, including
cytokine release syndrome (CRS), observed to date
ATA3219 (B-cell Malignancies)
- Atara is progressing ATA3219, our potential best-in-class,
allogeneic CAR T for B cell malignancies expressing CD19, and
expects to submit an IND in Q4 2022
- ATA3219 is an optimized approach to address high unmet medical
need, leveraging our next-generation 1XX CAR co-stimulatory
signaling domain and allogeneic EBV T-cell platform and does not
require TCR or human leukocyte antigen (HLA) gene editing
Allogeneic T-Cell Platform Development and Operations
- Atara entered a strategic manufacturing partnership in January
2022 with FUJIFILM Diosynth Biotechnologies (FDB), a subsidiary of
FUJIFILM Corporation (Fujifilm), under which Fujifilm will acquire
Atara’s T-cell Operations and Manufacturing (ATOM) facility for USD
100 million upfront, retaining current manufacturing and quality
staff at the site. Closing of the transaction is expected to occur
in April
- As part of the long-term supply agreement, which could extend
to 10 years following anticipated completion of the transaction,
FDB will also provide Atara with flexible capacity and specific
capability needed to manufacture and support the Company’s maturing
and promising pipeline
- Atara continues to leverage its recently established and now
fully operational Thousand Oaks-based Atara Research Center (ARC),
which houses the Company’s Pre-Clinical, Translational Sciences,
Manufacturing Process Sciences, and Analytical Development Teams to
further drive innovation
Fourth Quarter and Full Year 2021 Financial Results
- Cash, cash equivalents and short-term investments as of
December 31, 2021 totaled $371.1 million, as compared to $500.7
million as of December 31, 2020
- The December 31, 2021 cash balance includes $47.7 million of
net proceeds from the sale of 2,836,062 shares of common stock
through the Company’s ATM facilities in the fourth quarter, and a
$45.0 million upfront payment received under the Pierre Fabre
Commercialization Agreement, partially offset by operating cash
outflows
- Atara believes that its cash as of December 31, 2021, together
with the anticipated $100.0 million payable to Atara upon closing
of the strategic transaction with FDB, will be sufficient to fund
the Company’s planned operations into the fourth quarter of
2023
- Net cash used in operating activities was $34.3 million and
$220.5 million for the fourth quarter and fiscal year 2021,
respectively, as compared to $4.1 million and $180.8 million for
the same periods in 2020; the increase in operating cash usage in
2021 was primarily due to a $33.5 million increase in net loss and
increased usage of net working capital, partially offset by $45.0
million received under the Pierre Fabre Commercialization
Agreement
- Atara reported net losses of $93.3 million, or $0.96 per share,
and $340.1 million, or $3.63 per share, for the fourth quarter and
fiscal year 2021, respectively, as compared to $81.3 million, or
$0.95 per share, and $306.6 million, or $4.15 per share, for the
same periods in 2020
- Total operating expenses include non-cash expenses of $16.5
million and $63.0 million for the fourth quarter and fiscal year
2021, respectively, as compared to $13.6 million and $59.4 million
for the same periods in 2020
- Research and development expenses were $79.1 million and $282.0
million for the fourth quarter and fiscal year 2021, respectively,
as compared to $65.6 million and $244.7 million for the same
periods in 2020
- The increases in the fourth quarter and fiscal year 2021 were
primarily due to higher employee-related and overhead costs from
increased headcount, higher facility-related costs in support of
continuing expansion of research and development activities and
increased spending on research, development, and clinical trial
costs to further advance ATA188 and CAR T programs
- Research and development expenses include $8.4 million and
$32.1 million of non-cash stock-based compensation expenses for the
fourth quarter and fiscal year 2021, respectively, as compared to
$7.2 million and $31.5 million for the same periods in 2020
- General and administrative expenses were $21.8 million and
$78.8 million for the fourth quarter and fiscal year 2021,
respectively, as compared to $16.1 million and $64.4 million for
the same periods in 2020
- The increases in the fourth quarter and fiscal year 2021 were
primarily due to higher compensation-related costs from increased
headcount and activities to support an anticipated tab-cel
launch
- General and administrative expenses include $5.6 million and
$21.8 million of non-cash stock-based compensation expenses for the
fourth quarter and fiscal year 2021, respectively, as compared to
$4.3 million and $19.8 million for the same periods in 2020
Conference Call and Webcast Details
Atara will host a live conference call and webcast today,
Monday, February 28, 2022, at 4:30 p.m. EST to discuss the
Company’s financial results and recent operational highlights.
Analysts and investors can participate in the conference call by
dialing 877-407-8291 for domestic callers and 201-689-8345 for
international callers, using the conference ID 13725930. A live
audio webcast can be accessed by visiting the Investors & Media
– News & Events section of atarabio.com. An archived replay
will be available on the Company's website for 30 days following
the live webcast.
About Atara Biotherapeutics, Inc.
Atara Biotherapeutics, Inc. (@Atarabio) is a pioneer in T-cell
immunotherapy leveraging its novel allogeneic EBV T-cell platform
to develop transformative therapies for patients with serious
diseases including solid tumors, hematologic cancers and autoimmune
disease. With our lead program in Phase 3 clinical development and
currently under review to support registration in Europe, Atara is
the most advanced allogeneic T-cell immunotherapy company and
intends to rapidly deliver off-the-shelf treatments to patients
with high unmet medical need. Our platform leverages the unique
biology of EBV T cells and has the capability to treat a wide range
of EBV-associated diseases, or other serious diseases through
incorporation of engineered CARs (chimeric antigen receptors) or
TCRs (T-cell receptors). Atara is applying this one platform, which
does not require TCR or HLA gene editing, to create a robust
pipeline including: tab-cel® (tabelecleucel) in Phase 3 development
for Epstein-Barr virus-driven post-transplant lymphoproliferative
disease (EBV+ PTLD); ATA188, a T-cell immunotherapy targeting EBV
antigens as a potential treatment for multiple sclerosis; and
multiple next-generation chimeric antigen receptor T-cell (CAR-T)
immunotherapies for both solid tumors and hematologic malignancies.
Improving patients’ lives is our mission and we will never stop
working to bring transformative therapies to those in need. Atara
is headquartered in South San Francisco and our leading-edge
research, development and manufacturing facility is based in
Thousand Oaks, California. For additional information about the
company, please visit atarabio.com and follow us on Twitter 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 potential benefits, safety and efficacy of tab-cel®; the
timing and progress of tab-cel®, including (i) data and analyses
from ALLELE study; (ii) tab-cel® clinical trials, and the
occurrence, timing and outcome of Atara’s interactions and
discussions with the FDA regarding a BLA submission for tab-cel®,
(iii) the timing and outcome of the MAA for tab-cel®, (iv) the
timing of the initiation or submission of the BLA for tab-cel®,
(iv) the timing of the EMA’s review of the MAA for tab-cel®, (v)
Atara’s ability to successfully advance the development of
tab-cel®, and (vi) Atara’s collaboration with Pierre Fabre for
commercializing tab-cel® in Europe, Middle East, Africa and other
emerging markets; (2) the potential benefits, safety and efficacy
of ATA188; the timing and progress of ATA188, including (i)
regulatory designations for ATA188 granted by FDA and the impact
thereof; (ii) the mechanistic link between EBV and multiple
sclerosis and the ability of ATA188 to specifically target such
link; (iii) data from ATA188 OLE study; (iv) ATA188 clinical
trials, (iv) Atara’s ability to successfully advance the
development of ATA188, and (v) partnering options for ATA188; (3)
the timing and progress of its CAR T programs, and the safety and
efficacy of product candidates emerging from such programs,
including (i) ATA2271 clinical trial, (ii) ATA3271 and ATA3219
preclinical development, (iii) progress of the strategic
collaboration with Bayer for ATA2271 and 3271, and (iv) Atara’s
ability to successfully advance the development of its CAR T
programs; (4) Atara’s research and development activities at ARC;
(5) Atara’s proposed sale of its ATOM manufacturing facility to
FUJIFILM Diosynth Biotechnologies (FDB), including (i) the parties
ability to consummate such transaction, including the timing
thereof, (ii) the potential benefits of such transaction to Atara,
including the potential financial benefits to Atara, (iii) the
proposed supply agreement between the parties and the duration and
benefits thereof, (iv) FDB’s ability to perform under the proposed
supply agreement and meet Atara’s requirements, (v) FDB’s potential
plans for ATOM, and (vi) Atara’s ability to retain its staff and
capabilities, and (6) Atara’s ability to advance development of its
other programs. 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
ongoing COVID-19 pandemic, which may significantly impact (i) our
business, research, clinical development plans and operations,
including our operations in South San Francisco and Southern
California 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 (SEC),
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.
Consolidated Balance
Sheets
(Unaudited)
(In thousands)
December 31,
December 31,
2021
2020
Assets
Current assets:
Cash and cash equivalents
$
106,084
$
200,404
Short-term investments
264,984
300,255
Restricted cash - short-term
194
194
Accounts receivable
986
1,250
Prepaid expenses and other current
assets
12,373
21,170
Total current assets
384,621
523,273
Property and equipment, net
53,780
50,517
Operating lease assets
26,159
12,303
Restricted cash - long-term
1,200
1,200
Other assets
2,367
827
Total assets
$
468,127
$
588,120
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
17,368
$
7,118
Accrued compensation
25,150
20,458
Accrued research and development
expenses
13,451
15,813
Deferred revenue
40,760
33,455
Other current liabilities
9,057
6,057
Total current liabilities
105,786
82,901
Deferred revenue - long-term
55,708
27,795
Operating lease liabilities -
long-term
25,518
13,041
Other long-term liabilities
1,501
2,044
Total liabilities
188,513
125,781
Commitments and contingencies
Stockholders’ equity:
Common stock
9
8
Additional paid-in capital
1,744,695
1,586,616
Accumulated other comprehensive (loss)
income
(368
)
296
Accumulated deficit
(1,464,722
)
(1,124,581
)
Total stockholders’ equity
279,614
462,339
Total liabilities and stockholders’
equity
$
468,127
$
588,120
ATARA BIOTHERAPEUTICS,
INC.
Consolidated Statements of
Operations and Comprehensive Loss
(Unaudited)
(In thousands, except per
share amounts)
Three Months Ended
Twelve Months Ended
December 31,
December 31,
2021
2020
2021
2020
License and collaboration revenue
$
7,548
$
—
$
20,340
$
—
Operating expenses:
Research and development
79,134
65,554
282,001
244,650
General and administrative
21,817
16,143
78,801
64,402
Total operating expenses
100,951
81,697
360,802
309,052
Loss from operations
(93,403
)
(81,697
)
(340,462
)
(309,052
)
Interest and other income, net
84
398
367
2,447
Loss before provision for income taxes
$
(93,319
)
$
(81,299
)
$
(340,095
)
$
(306,605
)
Provision for income taxes
30
8
46
15
Net loss
$
(93,349
)
$
(81,307
)
$
(340,141
)
$
(306,620
)
Other comprehensive gain (loss):
Unrealized gain (loss) on
available-for-sale securities
(392
)
(231
)
(664
)
76
Comprehensive loss
$
(93,741
)
$
(81,538
)
$
(340,805
)
$
(306,544
)
Net loss per common share:
Basic and diluted net loss per common
share
$
(0.96
)
$
(0.95
)
$
(3.63
)
$
(4.15
)
Weighted-average shares outstanding used
to calculate basic and diluted net loss per common share
97,407
85,301
93,670
73,973
_____________________________
1 The International Council for
Harmonization of Technical Requirements for Pharmaceuticals for
Human Use
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220228005915/en/
Investors Eric Hyllengren 805-395-9669
ehyllengren@atarabio.com
Media Alex Chapman 805-456-4772 achapman@atarabio.com
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