Syncona Limited
Autolus reports first quarter 2024 Financial
Results and business updates
17 May 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 first quarter ended 31 March, 2024. Key highlights
are as follows:
· Updates from
obe-cel for patients with relapsed/refractory (r/r) adult B-cell
acute lymphoblastic leukaemia (ALL):
o Longer follow
up and subset analyses from the pivotal FELIX Phase II trial to be
presented in upcoming oral presentations at ASCO and EHA
o Market
Authorization Application (MAA) for obe-cel accepted by the
European Medicines Agency (EMA)
· Two patients have
been enrolled in the Phase I trial of obe-cel in systemic lupus
erythematosus (SLE), with the study on track to release initial
data in H2 CY2024
· Autolus is well
funded to drive the full launch and commercialisation of obe-cel in
r/r adult ALL, with cash and cash equivalents at March 31, 2024
totalling $758.5 million
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 / 13:30 pm BST, 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
Natalie Garland-Collins / Fergus
Witt
Tel: +44 (0) 20 3981 7940
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 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 First Quarter
2024 Financial Results and Business Updates
·
Longer follow up and subset
analyses from the pivotal FELIX Phase 2 data to be presented in
oral presentations at ASCO and EHA
·
Two patients enrolled in Phase 1
Systemic Lupus Erythematosus (SLE) trial; study on track for
initial data end of 2024
·
Market Authorization Application
(MAA) for obe-cel in r/r adult ALL accepted by European Medicines
Agency (EMA)
·
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, May 17,
2024 - Autolus Therapeutics
plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company
developing next-generation programmed T cell therapies, today
announces its operational and financial results for the first
quarter ended March 31, 2024.
"We continue to engage with the FDA
in the regulatory review process for obecabtagene autoleucel
(obe-cel) in adult ALL as we head towards the PDUFA target action
date of November 16, 2024, and are driving commercial readiness
activities across the Company," said Dr. Christian Itin, Chief Executive
Officer of Autolus. "We're also delighted that our abstracts
from the pivotal FELIX Phase 2 trial have been accepted for oral
presentations at ASCO and EHA this year and we look forward to
sharing further long-term data and additional subset
analyses."
"In addition, the first two patients
have been enrolled into our dose confirmation trial (CARLYSLE) of
obe-cel in Systemic Lupus Erythematosus (SLE) and the study is on
track for initial data by end of 2024."
Key obe-cel updates and
anticipated milestones:
·
Obe-cel in
relapsed / refractory (r/r) adult B-cell Acute Lymphoblastic
Leukemia (ALL) - The FELIX Study
o Obe-cel Biologics License Application (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 accepted in April 2024. For
the UK we are evaluating a filing based on an international
recognition procedure.
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.
o Further long-term data from the FELIX study including
additional subset analysis will be presented in oral and poster
presentations at the American Society of Clinical Oncology annual
meeting (ASCO - May 31 - June 4, 2024), and European Hematology
Association congress (EHA - June 13 - 16, 2024)
respectively.
·
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. Two patients have been
enrolled and Autolus continues to expect initial clinical data 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. Enrollment of the initial
cohorts are complete and further updates from the MCARTY study are
anticipated in H2 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.
Strategic
developments:
·
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.
·
In February 2024, Autolus completed an
underwritten offering in the United States at a price of $6.00 per
ADS, for total gross proceeds of $350 million before underwriting
fees and offering expenses.
Operational
Updates:
·
In March 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 April 2024, Autolus announced that the European
Medicines Agency (EMA) had accepted its Marketing Authorization
Application (MAA) for obe-cel for patients with relapsed/refractory
(r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL). The MAA
submission was based on data from the pivotal Phase 2 FELIX study
of obe-cel in adult r/r B-ALL.
·
In April 2024, Autolus entered into a distribution
services agreement with a subsidiary of Cardinal Health to support
the ordering and distribution of obe-cel in the United States,
following the receipt of regulatory approval.
·
In April 2024, Autolus announced the appointment
of Mike Bonney as Chairman of the Board, and Ravi Rao M.D., as
Non-Executive Director. John H. Johnson advised the Board of his
decision to step down from his role as Chairman of the Board and
Non-Executive Director, effective April 1, 2024.
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
|
May, 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 Quarter Ended March 31,
2024
Cash and cash equivalents at March
31, 2024, totaled $758.5 million, as compared to $239.6 million at
December 31, 2023.
Total operating expenses, net for
the three months ended March 31, 2024, were $38.8 million, as
compared to $39.1 million, for the same period in 2023.
Research and development expenses
increased from $27.4 million to $30.7 million for the three months
ended March 31, 2024, compared to the same period in 2023. This
change was primarily due to increases in operating costs related to
the Company's new commercial manufacturing facility, employee
salaries and related costs, clinical trial costs related to
obe-cel, and a decrease in our U.K. reimbursable R&D tax
credits claimable through the U.K. small and medium-sized entity
(SME) scheme. These were partially offset by decreases in
professional consulting fees, legal fees, manufacturing costs
related to obe-cel clinical supply, information technology
infrastructure fees and general office expenses.
General and administrative expenses
increased from $9.3 million to $18.2 million for the three months
ended March 31, 2024, compared to the same period in 2023. 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 was $52.7 million for the
three months March 31, 2024, compared to $39.8 million for the same
period in 2023. The basic and diluted net loss per ordinary share
for the three months ended March 31, 2024, totaled $(0.24),
compared to a basic and diluted net loss per ordinary share of
$(0.23) for 2023.
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
Quarter Ended March 31, 2024
Selected Unaudited Condensed
Consolidated Balance Sheet Data
(In
thousands)
|
|
|
|
|
|
|
March 31
|
|
December 31
|
|
|
|
|
|
|
|
2024
|
|
2023
|
Assets
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents
|
|
|
|
|
|
|
$
758,529
|
|
$
239,566
|
Total current assets
|
|
|
|
|
|
|
$
804,298
|
|
$
275,302
|
Total assets
|
|
|
|
|
|
|
$
901,436
|
|
$
375,381
|
Liabilities and shareholders' equity
|
|
|
|
|
|
|
|
|
|
Total current liabilities
|
|
|
|
|
|
|
$
43,985
|
|
$
44,737
|
Total liabilities
|
|
|
|
|
|
|
$
319,406
|
|
$
263,907
|
Total shareholders'
equity
|
|
|
|
|
|
|
$
582,030
|
|
$
111,474
|
Selected Unaudited Condensed
Consolidated Statements of Operations and Comprehensive Loss
Data
(In
thousands, except share and per share amounts)
|
|
|
|
|
|
|
Three Months Ended March
31,
|
|
|
|
|
|
|
|
|
2024
|
|
2023
|
|
License revenues
|
|
|
|
|
|
|
$
10,091
|
|
$
1,292
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
|
|
|
|
(30,671)
|
|
(27,388)
|
|
General and
administrative
|
|
|
|
|
|
|
(18,177)
|
|
(9,284)
|
|
Loss on disposal of property and
equipment
|
|
|
|
|
|
|
-
|
|
(3,768)
|
|
Total operating expenses, net
|
|
|
|
|
|
|
(38,757)
|
|
(39,148)
|
|
Total other expenses, net
|
|
|
|
|
|
|
(13,941)
|
|
(677)
|
|
Net
loss before income tax
|
|
|
|
|
|
|
(52,698)
|
|
(39,825)
|
|
Income tax benefit
|
|
|
|
|
|
|
8
|
|
14
|
|
Net
loss
|
|
|
|
|
|
|
(52,690)
|
|
(39,811)
|
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|
|
Foreign currency exchange
translation adjustment
|
|
|
|
|
|
|
58
|
|
5,641
|
|
Total comprehensive loss
|
|
|
|
|
|
|
$
(52,632)
|
|
$
(34,170)
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
ordinary share
|
|
|
|
|
|
|
$
(0.24)
|
|
$
(0.23)
|
|
Weighted-average basic and diluted
ordinary shares
|
|
|
|
|
|
|
222,170,707
|
|
173,825,825
|
|
Conference Call
Management will host a conference
call and webcast at 08:30 am EDT/13:30 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 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 accepted in April
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 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:
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