12 November 2024
Syncona
Limited
Autolus
reports third quarter 2024 Financial Results and business
updates
Syncona Ltd, (the "Company"), a
leading life science investor focused on creating, building and
scaling a portfolio of global leaders in life science, notes that
its portfolio company, Autolus Therapeutics plc (Nasdaq: AUTL)
("Autolus"), announced its financial results and business updates
for the third quarter ended 30 September 2024. Key highlights are
as follows:
·
The U.S. Food and Drug Administration
(FDA) granted marketing approval for AUCATZYL® (obe-cel)
for the treatment of adult patients with relapsed or refractory
B-cell precursor acute lymphoblastic leukaemia (r/r
B-ALL)
·
US commercial launch of
AUCATZYL® initiated
·
Biologics License
Application (BLA) approval triggers $30m milestone payment to
Autolus from Blackstone
·
Obe-cel is under regulatory review in
both the EU and the UK, with marketing authorisation submissions
accepted by the European Medicines Agency (EMA), and the UK
Medicines and Healthcare products Regulatory Agency
(MHRA)
·
Matthias Will M.D. appointed as Chief
Development Officer, effective September 30, 2024
·
Cash and cash equivalents of $657.1
million as at 30 September, 2024
The announcement can be accessed on
Autolus' investor website at https://www.autolus.com/investor-relations/news/ and
the full text of the announcement from Autolus is contained
below.
Autolus management will host a
conference call today, at 8:30am EST / 13:30pm GMT, to discuss the
company's financial results and provide a general business update.
To listen to the webcast and view the accompanying slide
presentation, please go to: https://www.autolus.com/investor-relations/events/
Enquiries
Syncona Ltd
Natalie Garland-Collins / Fergus
Witt
Tel: +44 (0) 20 3981 7912
FTI
Consulting
Ben Atwell / Tim Stamper
Tel: +44 (0) 20 3727 1000
About Syncona
Syncona's purpose is to invest to
extend and enhance human life. We do this by creating, building and
scaling companies to deliver transformational treatments to
patients in areas of high unmet need.
We aim to build and maintain a
diversified portfolio of 20-25 globally leading life science
businesses, across development stage, modality and therapeutic
area, for the benefit of all our stakeholders. We focus on
developing treatments that deliver patient impact by working in
close partnership with world-class academic founders and
experienced management teams. Our balance sheet underpins our
strategy, enabling us to take a long-term view as we look to
improve the lives of patients with no or poor treatment options,
build sustainable life science companies and deliver strong
risk-adjusted returns to shareholders.
Forward-looking statements - this announcement contains
certain forward-looking statements with respect to the portfolio of
investments of Syncona Limited. These statements and forecasts
involve risk and uncertainty because they relate to events and
depend upon circumstances that may or may not occur in the future.
There are a number of factors that could cause actual results or
developments to differ materially from those expressed or implied
by these forward-looking statements. In particular, many companies
in the Syncona Limited portfolio are conducting scientific research
and clinical trials where the outcome is inherently uncertain and
there is significant risk of negative results or adverse events
arising. In addition, many companies in the Syncona Limited
portfolio have yet to commercialise a product and their ability to
do so may be affected by operational, commercial and other
risks.
Syncona Limited seeks to achieve returns over the long term.
Investors should seek to ensure they understand the risks and
opportunities of an investment in Syncona Limited, including the
information in our published documentation, before
investing.
Autolus Therapeutics Reports Third Quarter 2024 Financial
Results and Business Updates
· AUCATZYL® (obecabtagene autoleucel) approved
by US FDA on November 8, ahead of target PDUFA date of November 16;
US commercial launch initiated
· BLA approval triggers $30m
milestone payment to Autolus from Blackstone
· Marketing authorizations for
obe-cel under review with both the MHRA EMA
· Matthias Will M.D. appointed
as Chief Development Officer, effective September 30,
2024
· Conference call to be held
today at 08:30 am EDT/13:30 pm BST: conference call participants
should pre-register using the link at the bottom of this press
release
LONDON, Nov. 12, 2024 - Autolus
Therapeutics plc (Nasdaq: AUTL), an early commercial-stage
biopharmaceutical company developing next-generation programmed T
cell therapies, today announces its financial results for the third
quarter ended September 30, 2024, and provides additional
operational and clinical updates.
"With the U.S. Food and Drug
Administration (FDA) having approved
AUCATZYL® (obe-cel) for the treatment of adult
B-cell Acute Lymphoblastic Leukemia (B-ALL) patients, we are all
systems go with our commercial efforts in the US across the
Company," said Dr. Christian
Itin, Chief Executive Officer of Autolus. "This first
FDA approval is just the beginning for Autolus; we have great
belief in our pipeline and our manufacturing capabilities and are
excited for the future."
Key
updates and anticipated milestones:
· AUCATZYL® was approved by the FDA for the
treatment of adult patients with relapsed and refractory B-cell
acute lymphoblastic leukemia on November 8, 2024.
· Obe-cel in r/r adult B-ALL -
The FELIX Study and regulatory updates
o Obe-cel is under regulatory review in both the EU and the UK,
with marketing authorization submissions accepted by the European
Medicines Agency in April 2024, and the UK Medicines and Healthcare
products Regulatory Agency in August 2024.
o Post
period, Autolus submitted obe-cel for appraisal by the U.K.
National Institute for Health and Care Excellence (NICE) and
Autolus looks forward to working with NICE and NHS England to make
obe-cel available to patients in England and Wales, if
approved.
o Autolus presented updated data from the pivotal Phase 1b/2
FELIX study at the Society of Hematologic Oncology (SOHO) meeting
in August 2024 which demonstrated the rationale for tumor burden
(TB)-guided dosing by analyzing the impact of bone marrow (BM)
blast percentage in patients treated with obe-cel. The data
demonstrated the importance of administering a split dose and
highlighted the differentiation of obe-cel based on its unique
biding properties and tumor burden-guided approach.
o Post
period, Autolus presented data at the 2024 Lymphoma, Leukemia &
Myeloma Congress on October 16-19. The poster presentation
suggested that adult patients with r/r B-ALL achieve comparable
outcomes irrespective of the timing of stem cell transplant (SCT)
pre or post obe-cel, suggesting no further benefit of consolidative
transplant based on this post-hoc analysis. Additionally, obe-cel
given as a sole treatment to patients with lower Tumor Burden (TB)
at Lymphodepletion (LD) was associated with better
outcomes.
· Obe-cel in B-cell mediated
autoimmune diseases
o The
Phase 1 dose confirmation study (CARLYSLE) in refractory systemic
lupus erythematosus (SLE) patients is ongoing and Autolus expects
to complete enrolment and patient dosing, as well as present
initial data in Q1 2025. The Company anticipates that full data
with adequate follow-up will be targeted for 2H 2025 at a medical
conference.
· Pipeline programs in
collaboration with University College London
o Clinical programs AUTO8, AUTO6NG and AUTO1/22 are progressing
and the Company is planning data updates for all programs in
2025.
Operational Updates:
· The
FDA approval for AUCATZYL triggers a $30 million milestone payment
to Autolus from Blackstone in accordance with the terms of the
collaboration agreement between the parties. In addition,
Autolus will make a £10 million regulatory milestone payment to UCL
Business Ltd. in accordance with the license agreement between the
parties.
· In
September 2024, Autolus announced the appointment of Matthias Will
M.D. as Chief Development Officer, effective September 30, 2024.
Dr. Will joins Autolus from Dren Bio, Inc., a privately held
biotech company, where he served as Chief Medical Officer. During
his tenure, Matthias led the expansion of the clinical team and
oversaw the submission of two INDs for candidates to potentially
treat hematologic cancers. Prior to that he served as Vice
President of Clinical Development for CRISPR Therapeutics where he
led the development of the company's allogeneic CAR T programs
targeting CD70 in T-cell lymphomas and renal cell carcinoma and the
early stage CD70-NK cell program in collaboration with NKarta
Inc.
|
Obe-cel FELIX data at American
Society of Hematology (ASH) meeting
|
December 2024
|
|
Obe-cel in autoimmune disease -
initial data from SLE Phase 1 study
|
Q1 2025
|
|
Initial data from PY01 trial of
obe-cel in pediatric ALL
|
H2 2025
|
|
SLE Phase 1 trial presentation at
medical conference
|
H2 2025
|
|
|
|
2024/2025 Expected News Flow:
Financial Results (Unaudited) for the Quarter Ended September
30, 2024
Cash and cash equivalents at
September 30, 2024 totaled $657.1 million, as compared to $239.6
million at December 31, 2023.
Total operating expenses, net for
the three months ended September 30, 2024 were $67.9 million, as
compared to $42.9 million for the same period in 2023.
Research and development expenses
increased from $32.3 million to $40.3 million for the three months
ended September 30, 2024, compared to the same period in 2023. This
change was primarily due to increases in employee salaries and
related costs, and clinical trial and manufacturing costs related
to obe-cel, partially offset by a decrease in professional fees and
an increase in our U.K. R&D tax credits that reduce R&D
expense.
General and administrative expenses
increased from $10.6 million to $27.3 million for the three months
ended September 30, 2024, compared to the same period in 2023. This
increase was primarily due to salaries and other employment-related
costs driven by increased headcount supporting
pre-commercialization activities.
Net loss was $82.1 million for the
three months ended September 30, 2024, compared to $45.8 million
for the same period in 2023. Basic and diluted net loss per
ordinary share for the three months ended September 30, 2024,
totaled $(0.31), compared to basic and diluted net loss per
ordinary share of $(0.26) for the same period in 2023.
Autolus estimates that, with its
current cash and cash equivalents, it is well capitalized to drive
the full launch and commercialization of obe-cel in r/r adult B-ALL
as well as to advance its pipeline development plans, which
includes providing runway to data in the first pivotal study of
obe-cel in autoimmune disease.
Financial Results for the
Quarter Ended September 30, 2024
Selected Unaudited Condensed
Consolidated Balance Sheet Data
(In thousands)
|
|
|
September 30,
2024
|
|
December 31,
2023
|
|
Assets
|
|
|
|
|
|
Cash and cash equivalents
|
|
$
|
657,067
|
|
$
|
239,566
|
|
Total current assets
|
|
$
|
718,114
|
|
$
|
275,302
|
|
Total assets
|
|
$
|
827,490
|
|
$
|
375,381
|
|
Liabilities and shareholders' equity
|
|
|
|
|
|
Total current liabilities
|
|
$
|
52,474
|
|
$
|
44,737
|
|
Total liabilities
|
|
$
|
350,525
|
|
$
|
263,907
|
|
Total shareholders'
equity
|
|
$
|
476,965
|
|
$
|
111,474
|
|
|
|
|
|
|
|
|
|
Selected Unaudited Condensed
Consolidated Statements of Operations and Comprehensive Loss
Data
(In thousands, except share and per share amounts)
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30,
|
|
Nine Months Ended
September 30,
|
|
|
|
|
2024
|
|
|
|
2023
|
|
|
|
2024
|
|
|
|
2023
|
|
|
License revenue
|
|
$
|
-
|
|
|
$
|
406
|
|
|
$
|
10,091
|
|
|
$
|
1,698
|
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
(40,323
|
)
|
|
|
(32,318
|
)
|
|
|
(107,606
|
)
|
|
|
(92,938
|
)
|
|
General and
administrative
|
|
|
(27,330
|
)
|
|
|
(10,611
|
)
|
|
|
(67,410
|
)
|
|
|
(31,017
|
)
|
|
Loss on disposal of property and
equipment
|
|
|
(223
|
)
|
|
|
-
|
|
|
|
(223
|
)
|
|
|
(3,791
|
)
|
|
Impairment of operating lease
right-of-use assets and related property and equipment
|
|
|
-
|
|
|
|
(382
|
)
|
|
|
(414
|
)
|
|
|
(382
|
)
|
|
Total operating expenses, net
|
|
|
(67,876
|
)
|
|
|
(42,905
|
)
|
|
|
(165,562
|
)
|
|
|
(126,430
|
)
|
|
Total other expenses, net
|
|
|
(14,196
|
)
|
|
|
(2,965
|
)
|
|
|
(27,428
|
)
|
|
|
(4,777
|
)
|
|
Net
loss before income tax
|
|
|
(82,072
|
)
|
|
|
(45,870
|
)
|
|
|
(192,990
|
)
|
|
|
(131,207
|
)
|
|
Income tax (expense)
benefit
|
|
|
(22
|
)
|
|
|
21
|
|
|
|
(66
|
)
|
|
|
(5
|
)
|
|
Net
loss
|
|
|
(82,094
|
)
|
|
|
(45,849
|
)
|
|
|
(193,056
|
)
|
|
|
(131,212
|
)
|
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|
Foreign currency exchange
translation adjustment
|
|
|
27,010
|
|
|
|
(5,837
|
)
|
|
|
28,094
|
|
|
|
5,104
|
|
|
Total comprehensive loss
|
|
$
|
(55,084
|
)
|
|
$
|
(51,686
|
)
|
|
$
|
(164,962
|
)
|
|
$
|
(126,108
|
)
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
ordinary share
|
|
$
|
(0.31
|
)
|
|
$
|
(0.26
|
)
|
|
$
|
(0.77
|
)
|
|
$
|
(0.75
|
)
|
|
Weighted-average basic and diluted
ordinary shares
|
|
|
266,084,589
|
|
|
|
173,984,101
|
|
|
|
251,480,521
|
|
|
|
173,890,666
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| |
Conference Call
Management
will host a conference call and webcast at 8:30 am EDT/1:30 pm
BST to discuss the company's financial results and provide a
general business update. Conference call participants should
pre-register using this link to
receive the dial-in numbers and a personal PIN, which are required
to access the conference call.
A simultaneous audio webcast and
replay will be accessible on the events
section of Autolus'
website.
About Autolus Therapeutics plc
Autolus is a biopharmaceutical company developing next-generation,
programmed T cell therapies for the treatment of cancer and
autoimmune disease. Using a broad suite of proprietary and modular
T cell programming technologies, Autolus is engineering precisely
targeted, controlled and highly active T cell therapies that are
designed to recognize target cells, break down their defense
mechanisms and eliminate these cells. Autolus has an FDA approved
product, AUCATZYL®, and a pipeline of product candidates
in development for the treatment of hematological malignancies,
solid tumors and autoimmune diseases. For more information, please
visit www.autolus.com
About Aucatzyl® (obecabtagene autoleucel,
AUTO1)
AUCATZYL® is a B-lymphocyte antigen CD19 (CD19)
chimeric antigen receptor (CAR) T cell therapy approved by the FDA
for the treatment of relapsed/refractory (r/r) Adult B-cell Acute
Lymphoblastic Leukemia (B-ALL). Please see full Prescribing
Information, including BOXED WARNING and Medication
Guide. Obe-cel is designed with a fast target binding off-rate to
minimize excessive activation of the programmed T cells. In the EU
a regulatory submission to the EMA was accepted in April 2024,
while in the UK, an MAA was submitted to MHRA in July 2024. In
collaboration with Autolus' academic partner, University College
London, obe-cel is currently being evaluated in a Phase 1 clinical
trial for B-cell non-Hodgkin lymphoma (B-NHL).
About FELIX clinical trial
Autolus' Phase 1b/2 clinical trial of obe-cel enrolled adult
patients with r/r B-precursor ALL. The trial had a Phase 1b
component prior to proceeding to the single arm, Phase 2 clinical
trial. The primary endpoint was overall response rate, and the
secondary endpoints included duration of response, MRD negative
complete remission rate and safety. The trial enrolled over 100
patients across 30 of the leading academic and non-academic centers
in the United States, United Kingdom and Europe.
[NCT04404660]
About AUTO1/22
AUTO1/22 is a novel dual targeting CAR T cell-based therapy
candidate based on obe-cel. It is designed to combine the enhanced
safety, robust expansion and persistence seen with the fast off
rate CD19 CAR from obe-cel with a high sensitivity CD22 CAR to
reduce antigen negative relapses. This product candidate is
currently in a Phase 1 clinical trial for patients with r/r
pediatric ALL. [NCT02443831]
About AUTO6NG
AUTO6NG is a next generation programmed T cell product candidate in
development for the treatment of both neuroblastoma and other
GD2-expressing solid tumors. AUTO6NG builds on preliminary proof of
concept data from AUTO6, a CAR targeting GD2-expression cancer cell
currently in clinical development for the treatment of
neuroblastoma. AUTO6NG incorporates additional cell programming
modules to overcome immune suppressive defense mechanisms in the
tumor microenvironment, in addition to endowing the CAR T cells
with extended persistence capacity. A Phase 1 clinical trial of
AUTO6NG in children with relapsed/refractory neuroblastoma was
opened for enrollment in the fourth quarter of
2023.
About AUTO8
AUTO8 is a next-generation product candidate for multiple myeloma
which comprises two independent CARs for the multiple myeloma
targets, B-cell maturation antigen (BCMA) and CD19. We have
developed an optimized BCMA CAR designed for improved killing of
target cells that express BCMA at low levels. This has been
combined with fast off rate CD19 CAR from obe-cel, with the aim of
inducing deep and durable responses and extending the durability of
effect over other BCMA CARs currently in development. This product
candidate is currently in a Phase I clinical trial for patients
with r/r multiple myeloma. [NCT04795882]
Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of the "safe harbor" provisions of the Private Securities
Litigation Reform Act of 1995. Forward-looking statements are
statements that are not historical facts, and in some cases can be
identified by terms such as "may," "will," "could," "expects,"
"plans," "anticipates," and "believes." These statements include,
but are not limited to, statements regarding the market opportunity
for AUCATZYL®, Autolus' development and
commercialization of its product candidates, and the timing of data
announcements and regulatory submissions. Any forward-looking
statements are based on management's current views and assumptions
and involve risks and uncertainties that could cause actual
results, performance, or events to differ materially from those
expressed or implied in such statements. These risks and
uncertainties include, but are not limited to, the risks that
Autolus' preclinical or clinical programs do not advance or result
in approved products on a timely or cost effective basis or at all;
the results of early clinical trials are not always being
predictive of future results; the cost, timing and results of
clinical trials; that many product candidates do not become
approved drugs on a timely or cost effective basis or at all; the
ability to enroll patients in clinical trials; and possible safety
and efficacy concerns. For a discussion of other risks and
uncertainties, and other important factors, any of which could
cause Autolus' actual results to differ from those contained in the
forward-looking statements, see the section titled "Risk Factors"
in Autolus' Annual Report on Form 10-K filed with the Securities
and Exchange Commission, or the SEC, on March 21, 2024 as well as
discussions of potential risks, uncertainties, and other important
factors in Autolus' subsequent filings with the Securities and
Exchange Commission. All information in this press release is as of
the date of the release, and Autolus undertakes no obligation to
publicly update any forward-looking statement, whether as a result
of new information, future events, or otherwise, except as required
by law. You should, therefore, not rely on these forward-looking
statements as representing Autolus' views as of any date subsequent
to the date of this press release.
Contact:
Amanda Cray
+1 617-967-0207
a.cray@autolus.com
Olivia Manser
+44 (0) 7780 471 568
o.manser@autolus.com
Susan A. Noonan
S.A. Noonan Communications
+1-917-513-5303
susan@sanoonan.com