Syncona
Limited
Autolus
reports full year 2023 Financial Results and business
updates
14 March 2024
Syncona Ltd, a leading life science
investor focused on creating, building and scaling 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 full
year ended December 31, 2023. A copy of the announcement is set out
below, with key highlights as follows:
Positive regulatory and clinical
progress:
· Biologics License Application (BLA) submitted for lead obe-cel
therapy, a potentially transformational treatment for r/r adult
B-cell acute lymphoblastic leukaemia (B-ALL), to the US Food and
Drug Administration (FDA); PDUFA action date, the date the FDA is
expected to respond by, is set for November 16,
2024
·
Presented pooled analysis of FELIX Phase Ib/II
study demonstrating prolonged event free survival and low
immunotoxicity across all r/r B-ALL cohorts at the American Society
of Haematology (ASH) meeting in December 2023
·
Phase I dose confirmation study in refractory
systemic lupus erythematosus (SLE) has first site open for
enrolment in Q1 CY2024, extending obe-cel into autoimmune diseases,
with initial clinical data expected in H2 CY2024
Post period end, strong financial
position established:
·
Entered a strategic
collaboration with BioNTech aimed at advancing both companies'
autologous CAR T programs; included $200 million equity investment
and $50 million upfront to Autolus
·
Completed underwritten offering in February 2024,
raising gross proceeds of $350 million
· The two transactions enable Autolus to drive the full launch
and commercialisation of obe-cel in r/r adult ALL and establish the
company as a potential leader in the delivery of CAR T therapy to
patients with autoimmune diseases
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 8:30 am EDT / 12:30 pm 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/
[ENDS]
Enquiries
Syncona Ltd
Annabel Clark / Fergus
Witt
Tel: +44 (0) 7714 916615
FTI Consulting
Ben Atwell / Natalie Garland-Collins
/ 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 and building
companies to deliver transformational treatments to patients in
areas of high unmet need.
Our strategy is to create, build and
scale companies around exceptional science to create a diversified
portfolio of 20-25 globally leading healthcare businesses, across
development stage and therapeutic areas, for the benefit of all our
stakeholders. We focus on developing treatments for patients by
working in close partnership with world-class academic founders and
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.
Copies of this press release and
other corporate information can be found on the company website
at: www.synconaltd.com 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 Full Year 2023
Financial Results
and Business Updates
· Announced strategic collaboration and equity
investment from BioNTech for aggregate proceeds of $250 million
upfront, plus underwritten offering of ADSs for $350 million, for
gross proceeds of $600 million received in February
2024
·
Submitted a Biologics License Application
(BLA) for obecabtagene autoleucel (obe-cel), a potentially
transformational treatment for relapsed/refractory (r/r) adult
B-cell Acute Lymphoblastic Leukemia (ALL), to the US Food &
Drug Administration (FDA); Prescription Drug User Fee Act (PDUFA)
target action date November 16, 2024
·
Successfully completed first facility
inspection and obtained a Manufacturer's Importation
Authorization (MIA) from the Medicines and Healthcare
products Regulatory Agency (MHRA), enabling
the commercial product
supply for obe-cel at The Nucleus manufacturing
facility
·
Submitted a Market Authorization Application
(MAA) for obe-cel in r/r adult ALL with the European Medicines
Agency (EMA)
·
Conference call to be held today at 08:30 am
EDT/12:30 pm GMT: Conference call participants should pre-register
using the link at the bottom of this press
release
LONDON, March 14,
2024 - Autolus Therapeutics plc (Nasdaq:
AUTL), a clinical-stage biopharmaceutical company developing
next-generation programmed T cell therapies, today announced its
operational and financial results for the full year ended December
31, 2023.
"We're delighted to be starting 2024 in such
a strong financial position; our recently announced strategic
alliance with BioNTech, coupled with two equity financing
transactions, raised gross proceeds of $600 million. Combined with
our 2023 ending cash of $240 million, this enables us to drive the
full launch and commercialization of obe-cel in r/r adult ALL and
establish Autolus as a potential leader in the delivery of CAR T
therapy to patients with autoimmune
diseases," said Dr. Christian Itin,
Chief Executive Officer of Autolus.
"2023
was a transformational year for the Company. Our lead program,
obe-cel, demonstrated strong data in B-ALL in the pivotal FELIX
study, we fully validated our commercial manufacturing facility,
The Nucleus, to support our regulatory submissions and we submitted
our first BLA for obe-cel to the United States Food and Drug
Administration (FDA) in November, with a PDUFA target action date
of November 16, 2024. We also just submitted an MAA to the European
Medicines Agency (EMA)."
"Beyond
B-ALL, we see a significant opportunity for obe-cel in autoimmune
disease. Our Phase 1 dose confirmation trial in refractory SLE is
now open for enrollment. We believe obe-cel's clinical profile,
together with our commercial product delivery base and
infrastructure, will help to drive an accelerated and
differentiated expansion in autoimmune diseases and we look forward
to sharing initial data from the study in late 2024."
"For
now, we remain fully focused on preparing for a potential obe-cel
launch and successfully transitioning Autolus to a commercial stage
company. Pre-commercial and product delivery activities are well
underway, and we are on track to make obe-cel available to B-ALL
patients as soon as possible, following a potential
approval."
Key obecabtagene autoleucel (obe-cel)
updates and anticipated milestones:
·
Obe-cel in relapsed / refractory (r/r) adult
ALL - The FELIX Study
o Obe-cel BLA for r/r B-ALL submitted to the
FDA in November 2023; PDUFA target action date of November 16,
2024. A marketing authorization application (MAA) to the European
Medicines Agency (EMA) was just submitted and an MAA submission to
the MHRA in the UK is planned for the second half of
2024.
o Pooled analysis of the FELIX Phase 1b/2
study presented at ASH in December 2023 demonstrated prolonged
event free survival and low overall immunotoxicity across all
cohorts in r/r B-ALL, and particularly in patients with low
leukemic burden at lymphodepletion. Additionally, data from a
pooled analysis from the ALLCAR19 study and FELIX
Phase 1b in r/r B-ALL showed durable remissions with obe-cel as a
stand-alone therapy in a subset of patients after a median follow
up of >3 years. Further long-term data from the FELIX study is
anticipated at medical conferences in 2024.
·
Obe-cel in B-cell mediated autoimmune
diseases
o The Phase 1 dose confirmation study in
refractory systemic lupus erythematosus (SLE) patients is open for
enrollment; initial clinical data expected in late 2024.
Pipeline clinical trials, in collaboration with University
College London (UCL), updates and anticipated
milestones:
·
AUTO8 in Multiple
Myeloma - Phase 1 MCARTY Study
o AUTO8 is a next-generation product candidate
for multiple myeloma, which includes two CARs for the multiple
myeloma targets, BCMA and CD19. Initial data from the MCARTY Phase
1 study in multiple myeloma presented at ASH in December 2023
showed AUTO8 was well tolerated, with responses observed in all
patients. Further updates from the MCARTY study are anticipated
during 2024.
·
AUTO6NG in Neuroblastoma - Phase 1 MAGNETO
Study
o AUTO6NG contains a CAR that targets GD2
alongside additional programming modules to enhance the activity
and persistence. A Phase 1 clinical study in children with r/r
neuroblastoma was opened for enrollment in the fourth quarter of
2023.
Post-period:
Strategic developments:
Strategic alliance with BioNTech SE
(Nasdaq: BNTX)
In
February 2024, BioNTech and Autolus announced a strategic CAR T
cell therapy collaboration to advance their pipelines and expand
late-stage programs, for $50 million cash upfront and up to $582
million in potential option exercise and milestone payments.
Additionally, Autolus sold $200 million of ADSs to BioNTech in a
concurrent private placement financing transaction.
Overview:
- BioNTech has right to utilize
Autolus' manufacturing capacity, know-how and cost-efficiencies to
accelerate the planned clinical development and commercialization
of BNT211
- BioNTech to support launch
and expansion of development program of Autolus' lead cell therapy
candidate obe-cel and will receive a royalty on net
sales
- BioNTech has
co-commercialization options for Autolus' AUTO1/22 and AUTO6NG
programs, and an option to access a suite of Autolus target binders
and cell programming technologies
Underwritten
offering
In
February 2024, Autolus completed an underwritten registered direct
offering in the United States at a price of $6.00 per ADS, for
total gross proceeds of $350 million.
Recent Operational
Updates:
·
In March 2024, following the most recent GMP
inspection by the MHRA in February 2024, The Nucleus manufacturing
facility in Stevenage obtained a Manufacturer's Importation
Authorization (MIA), together with the accompanying GMP
certificate. This authorization enables Autolus to manufacture
products for global commercial and clinical supply at The Nucleus,
effective as of March 18, 2024.
·
In February 2024, Autolus promoted Dr. Chris
Williams to Chief Business Officer and Alex Driggs to Senior Vice
President, Legal Affairs and General Counsel. Chris has been with
the Company since its inception, having negotiated on behalf of UCL
the spin off and formation of Autolus. Alex has been with Autolus
since 2018 in the role of General Counsel.
·
Dr. Edgar Braendle has notified the Company
that he will step down as Chief Development Officer to pursue other
opportunities. Edgar will continue to advise the company during the
BLA and MAA review process. Miranda Neville, SVP and obe-cel
Program Leader will run the Development team.
·
Autolus announced the appointment of
Elisabeth (Lis) Leiderman, M.D. and Robert W. Azelby to its Board
of Directors. Dr. Leiderman brings extensive transactional and
financial expertise, and Mr. Azelby brings more than 30 years of
biopharmaceutical leadership and commercial experience to Autolus'
Board.
Scientific
Publications:
· In January 2024, Autolus announced the publication of a paper
in ACS Chemical Biology entitled: 'Designer small molecule control system based
on Minocycline induced disruption of protein-protein
interaction' - Jha et al., ACS Chemical Biology (2024)
doi:10.1021/acschembio.3c00521; [Link]
· In February 2024, Autolus announced the publication of a paper
in Nature Communications entitled: 'Structure-Guided Engineering of
Immunotherapies Targeting TRBC1 and TRBC2 in T Cell
Malignancies' - Ferrari et al., Nat Commun 15, 1583 (2024)
doi:10.1038/s41467-024-45854-3; [Link]
· In March 2024, Autolus announced the publication of a paper in
Blood Cancer Journal entitled: 'Dual T-cell constant β chain (TRBC)1 and
TRBC2 staining for the identification of T-cell neoplasms by flow
cytometry - Horna et al., Blood Cancer J. 14, 34 (2024) doi:
10.1038/s41408-024-01002-0; [Link]
2024 Expected News
Flow:
Obe-cel FELIX data update at ASCO,
EHA & ASH
|
June & Dec 2024
|
Obe-cel Marketing Authorization
Application to MHRA
|
Second half 2024
|
Obe-cel U.S. FDA PDUFA target action
date
|
November 16, 2024
|
Obe-cel in autoimmune disease -
initial data from SLE Phase 1 study
|
Late 2024
|
Financial Results (Unaudited) for the Full Year Ended December
31, 2023
Cash and cash equivalents at
December 31, 2023 totaled $239.6 million, as compared to $382.4
million at December 31, 2023.
Total operating expenses, net for
the year ended December 31, 2023, were $179.7 million, as compared
to $143.4 million, for the year ended December 31, 2022.
Research and development expenses
increased from $117.4 million to $130.5 million for the year ended
December 31, 2023, compared to the same period in 2022. This change
was primarily due to increases in operating costs related to the
Company's new commercial manufacturing facility, contractual
milestone payments and employee salaries and related costs, a
decrease in our U.K. reimbursable R&D tax credits claimable
through the U.K. small and medium-sized entity (SME) scheme and
partially offset by decreases in clinical and manufacturing costs
related to the Company's obe-cel clinical product
candidate.
In prior years, Autolus reported the
R&D tax credits as income tax benefit on its statements of
operations. The Company has revised its financial presentation,
including the prior years, and will now present such tax credits as
a reduction in research and development expense. As a result,
income tax benefit has reduced by $19.5 million and $24.6 million
for the years ended December 31, 2023, and 2022, respectively, with
corresponding reductions in research and development expenses and
total operating expenses.
General and administrative expenses
increased from $31.9 million to $46.7 million for the year ended
December 31, 2023, compared to the same period in 2022. This
increase was primarily due to salaries and other employment-related
costs driven by an increase in general and administrative headcount
supporting the overall growth of the business, primarily relating
to pre-commercialization activities.
Net loss attributable to ordinary
shareholders was $208.4 million for the year ended December 31,
2023, compared to $148.8 million for the same period in 2022. The
basic and diluted net loss per ordinary share for the year ended
December 31, 2023, totaled $(1.20), compared to a basic and diluted
net loss per ordinary share of $(1.57) for 2022.
Autolus estimates that, with its
current cash and cash equivalents and proceeds received from the
strategic alliance with BioNTech and the private placement and
underwritten equity financing, it is well capitalized to drive the
full launch and commercialization of obe-cel in r/r adult 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
Year Ended December 31, 2023
Selected Unaudited
Consolidated Balance Sheet Data
(In
thousands)
|
|
|
|
|
|
|
December
31,
|
|
|
|
|
|
|
|
2023
|
|
2022
|
Assets
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents
|
|
|
|
|
|
|
$
239,566
|
|
$
382,436
|
Total current assets
|
|
|
|
|
|
|
$
275,302
|
|
$
425,771
|
Total assets
|
|
|
|
|
|
|
$
375,381
|
|
$
490,274
|
Liabilities and shareholders' equity
|
|
|
|
|
|
|
|
|
|
Total current liabilities
|
|
|
|
|
|
|
$
44,737
|
|
$
46,366
|
Total liabilities
|
|
|
|
|
|
|
$
263,907
|
|
$
191,600
|
Total shareholders' equity
|
|
|
|
|
|
|
$
111,474
|
|
$
298,674
|
Selected Unaudited
Consolidated Statements of Operations and Comprehensive Loss
Data
(In
thousands, except share and per share amounts)
|
|
|
|
|
|
|
Year Ended December
31,
|
|
|
|
|
|
|
|
|
2023
|
|
2022
|
|
Grant income
|
|
|
|
|
|
|
$
-
|
|
$
166
|
|
License revenues
|
|
|
|
|
|
|
1,698
|
|
6,194
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
|
Research and development[1]
|
|
|
|
|
|
|
(130,481)
|
|
(117,354)
|
|
General and
administrative
|
|
|
|
|
|
|
(46,745)
|
|
(31,899)
|
|
Loss on disposal of property and
equipment
|
|
|
|
|
|
|
(3,791)
|
|
(515)
|
|
Impairment of operating lease
right-right-of-use and related property equipment
|
|
|
|
|
|
|
(382)
|
|
-
|
|
Total operating expenses, net
|
|
|
|
|
|
|
(179,701)
|
|
(143,408)
|
|
Total other expenses, net
|
|
|
|
|
|
|
(28,701)
|
|
(5,159)
|
|
Net
loss before income tax
|
|
|
|
|
|
|
(208,402)
|
|
(148,567)
|
|
Income tax benefit
(expense)1
|
|
|
|
|
|
|
19
|
|
(272)
|
|
Net
loss attributable to ordinary shareholders
|
|
|
|
|
|
|
(208,383)
|
|
(148,839)
|
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|
|
Foreign currency exchange
translation adjustment
|
|
|
|
|
|
|
9,906
|
|
(30,328)
|
|
Total comprehensive loss
|
|
|
|
|
|
|
$
(198,477)
|
|
$
(179,167)
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
ordinary share
|
|
|
|
|
|
|
$
(1.20)
|
|
$
(1.57)
|
|
Weighted-average basic and diluted
ordinary shares
|
|
|
|
|
|
|
173,941,926
|
|
94,993,400
|
|
Conference Call
Management will host a conference
call and webcast at 08:30 am EDT/12:30 pm GMT 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 clinical-stage
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 better
recognize target cells, break down their defense mechanisms and
eliminate these cells. Autolus has 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 obe-cel (AUTO1)
Obe-cel is a CD19 CAR T cell
investigational therapy designed to overcome the limitations in
clinical activity and safety compared to current CD19 CAR T cell
therapies. Obe-cel is designed with a fast target binding
off-rate to minimize excessive activation of the programmed T
cells. In clinical trials of obe-cel, this "fast off-rate" profile
reduced toxicity and T cell exhaustion, resulting in improved
persistence and leading to high levels of durable remissions in r/r
Adult ALL patients. The results of the FELIX trial, a pivotal trial
for adult ALL, have been submitted and accepted by the FDA with a
PDUFA target action date of November 16, 2024. A regulatory
submission to the EMA was made in the first half of 2024. In
collaboration with Autolus' academic partner, UCL, obe-cel is
currently being evaluated in a Phase 1 clinical trials for
B-NHL.
About obe-cel
FELIX clinical trial
Autolus' Phase 1b/2 clinical trial
of obe-cel enrolled adult patients with relapsed / refractory
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 CR 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 I 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, 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 Autolus' development and commercialization of its product
candidates, timing of data announcements and regulatory
submissions, its cash resources and the market opportunity for
obe-cel. 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 20-F filed with the Securities
and Exchange Commission, or the SEC, on March 7, 2023 and in
Autolus' Quarterly Report on Form 10-Q filed with the Securities
and Exchange Commission on November 9, 2023, 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:
Olivia Manser
+44 (0) 7780 471 568
o.manser@autolus.com
Julia Wilson
+44 (0) 7818 430877
j.wilson@autolus.com
Susan A. Noonan
S.A. Noonan
Communications
+1-917-513-5303
susan@sanoonan.com
[1] Includes the presentation
of our U.K. SME R&D Tax Credit with Income tax benefit as
contra research and development expense in the amounts of $19.5
million and $24.6 million for the years ended December 31, 2023,
and 2022, respectively.