8 August 2024
Syncona
Limited
Autolus reports second
quarter 2024 Financial Results and business
updates
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 second quarter ended
30 June 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 Autolus is on track for the potential US commercial launch of
obe-cel, with the FDA's target action date for reviewing its
biologics license application (BLA) set for 16 November
2024
o Longer follow up and subset analyses from the pivotal FELIX
Phase II trial presented at ASCO and EHA, with the majority of
responders showing durable responses and 40% of patients being in
ongoing remission without subsequent stem cell transplant (SCT) or
other intervention; a further data update is expected at ASH in
December 2024
o Market Authorisation Application (MAA) submitted to the
Medicine and Healthcare products Regulatory Authority (MHRA) in the
UK at the end of July 2024, as the MAA review process continues
with the European Medicines Agency (EMA)
·
A Phase I trial of obe-cel in systemic lupus
erythematosus (SLE) is ongoing, 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 30 June 2024 totalling $705.9
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, 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.
Copies of this press release and
other corporate information can be found on the company website
at: www.synconaltd.com
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
Second Quarter 2024 Financial Results
and Business
Updates
·
On track for
potential US commercial launch of obe-cel; PDUFA date November 16,
2024
·
Longer follow up
and subset analyses from pivotal FELIX Phase 2 data presented at
ASCO and EHA; majority of responders showed durable
responses; 40% of patients in ongoing remission without subsequent
stem cell transplant (SCT) or other intervention
·
A Market
Authorization Application (MAA) for obe-cel in relapsed/refractory
r/r adult B-cell Acute Lymphoblastic Leukemia (B-ALL) was submitted
to the Medicine and Healthcare products Regulatory Authority (MHRA)
in the UK at the end of July 2024. The MAA review process continues
with the 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, August 8, 2024
-
Autolus Therapeutics plc (Nasdaq: AUTL), a
clinical-stage biopharmaceutical company developing next-generation
programmed T cell therapies, today announces its financial results
for the second quarter ended June 30, 2024, and provides additional
operation and clinical updates.
"We are focused on driving
commercial readiness activities across Autolus to bring our lead
product obecabtagene autoleucel (obe-cel) to adult B-ALL patients.
Applications for marketing authorizations are under review by
regulatory agencies in the US, Europe and UK, and we are working
towards a US Food and Drug Administration (FDA) PDUFA target date
of November 16, 2024," said Dr.
Christian Itin, Chief Executive Officer of Autolus. "Data
from the FELIX Phase 1b/2 study with a median follow up of 21
months presented at ASCO and EHA indicate a long-term plateau
forming in event-free and overall survival rates. Stem cell
transplant post obe-cel did not appear to improve patient outcomes,
and patients with long-term persisting obe-cel appear to have
improved event free survival. The data support the potential for
obe-cel as a stand-alone therapy in a portion of r/r adult ALL
patients."
Key obe-cel updates and
anticipated milestones:
·
Obe-cel in r/r
adult B-ALL - The FELIX Study
o In the UK,
an MAA was submitted to the MHRA at the end of July 2024. The
Biologics License Application (BLA) is on track with the FDA,
working towards a Prescription Drug User Fee Act (PDUFA) target
action date of November 16, 2024. An MAA submitted to EMA was
accepted in April 2024.
o Pooled
analysis of the FELIX Phase 1b/2 study were presented at the
American Society of Oncology (ASCO) and European Hematology
Association (EHA) annual meetings in June 2024, with a median
follow-up of 21 months. Data showed stabilization of event-free
survival and overall survival following treatment with obe-cel,
with 40% of patients in ongoing remission. Of the 99 responders
following treatment with obe-cel, 18 received a subsequent stem
cell transplant (SCT) while in minimal residual disease
(MRD)-negative response but did not show improved survival compared
to patients who did not have a subsequent SCT. Patients with
prolonged obe-cel persistence experienced improved event-free
survival and data indicate that bridging of high tumor burden
patients with inotuzumab could be effective at reducing tumor
burden, cytokine release syndrome (CRS) and immune effector
cell-associated neurotoxicity syndrome (ICANS) without increasing
liver toxicity.
·
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. Autolus continues to
expect initial clinical data in late 2024.
·
Pipeline
programs in collaboration with University College
London
o Clinical
programs AUTO8, AUTO6NG and AUTO1/22 are progressing well and we
are planning data updates for all programs in 2025.
Operational
Updates:
·
During the quarter, Autolus promoted the following
individuals to Senior Vice President: Andrea Braun, Regulatory
Affairs; Markus Gruell, Corporate Quality; Claudia Mercedes Mayer,
Manufacturing Strategy and Technology; Chris Gray, Technical
Operations and Facilities and Dilip Patel, Market Access and
Pricing Strategy. These individuals bring significant leadership
experience and continue to drive Autolus' regulatory activities and
preparation for the potential commercialization and launch of
obe-cel.
·
In April 2024, Autolus announced that the EMA had
accepted its MAA for obe-cel for patients with r/r adult
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, if
it receives 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 stepped down from his roles
as Chairman of the Board and Non-Executive Director, effective
April 1, 2024.
2024 Expected News
Flow:
Obe-cel U.S. FDA PDUFA target action
date
|
November 16, 2024
|
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
|
Late 2024
|
Financial Results (Unaudited) for the Quarter Ended June 30,
2024
Cash and cash equivalents at June
30, 2024 totaled $705.9 million, as compared to $239.6 million at
December 31, 2023.
Total operating expenses, net for
the three months ended June 30, 2024 were $58.9 million, as
compared to $44.4 million for the same period in 2023.
Research and development expenses
increased from $33.2 million to $36.6 million for the three months
ended June 30, 2024, compared to the same period in 2023. This
change was primarily due to increases in operating costs related to
our new manufacturing facility, employee salaries and related
costs, and clinical trial and manufacturing costs related to
obe-cel, partially offset by an increase in our U.K. reimbursable
R&D tax credits that reduce R&D expense.
General and administrative expenses
increased from $11.1 million to $21.9 million for the three months
ended June 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 $58.3 million for the
three months June 30, 2024, compared to $45.6 million for the same
period in 2023. Basic and diluted net loss per ordinary share for
the three months ended June 30, 2024, totaled $(0.22), 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 June 30, 2024
Selected Unaudited Condensed
Consolidated Balance Sheet Data
(In
thousands)
|
|
|
|
|
|
|
June 30
|
|
December 31
|
|
|
|
|
|
|
|
2024
|
|
2023
|
Assets
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents
|
|
|
|
|
|
|
$
705,939
|
|
$
239,566
|
Total current assets
|
|
|
|
|
|
|
$
758,600
|
|
$
275,302
|
Total assets
|
|
|
|
|
|
|
$
853,620
|
|
$
375,381
|
Liabilities and shareholders'
equity
|
|
|
|
|
|
|
|
|
|
Total current liabilities
|
|
|
|
|
|
|
$
40,904
|
|
$
44,737
|
Total liabilities
|
|
|
|
|
|
|
$
325,776
|
|
$
263,907
|
Total shareholders'
equity
|
|
|
|
|
|
|
$
527,844
|
|
$
111,474
|
Selected Unaudited Condensed
Consolidated Statements of Operations and Comprehensive Loss
Data
(In
thousands, except share and per share amounts)
|
|
|
Three Months Ended June
30,
|
|
Six Months Ended June
30,
|
|
|
|
2024
|
|
2023
|
|
2024
|
|
2023
|
License revenue
|
|
|
$
-
|
|
$
-
|
|
$
10,091
|
|
$
1,292
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
(36,612)
|
|
(33,232)
|
|
(67,283)
|
|
(60,620)
|
General and
administrative
|
|
|
(21,903)
|
|
(11,122)
|
|
(40,080)
|
|
(20,406)
|
Loss on disposal of property and
equipment
|
|
|
-
|
|
(23)
|
|
-
|
|
(3,791)
|
Impairment of operating lease
right-of-use assets and related property and equipment
|
|
|
(414)
|
|
-
|
|
(414)
|
|
-
|
Total operating expenses, net
|
|
|
(58,929)
|
|
(44,377)
|
|
(97,686)
|
|
(83,525)
|
Total other income (expense), net
|
|
|
708
|
|
(1,135)
|
|
(13,233)
|
|
(1,812)
|
Net
loss before income tax
|
|
|
(58,221)
|
|
(45,512)
|
|
(110,919)
|
|
(85,337)
|
Income tax expense
|
|
|
(51)
|
|
(40)
|
|
(43)
|
|
(26)
|
Net
loss
|
|
|
(58,272)
|
|
(45,552)
|
|
(110,962)
|
|
(85,363)
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|
Foreign currency exchange
translation adjustment
|
|
|
1,026
|
|
5,300
|
|
1,084
|
|
10,941
|
Total comprehensive loss
|
|
|
$
(57,246)
|
|
$
(40,252)
|
|
$
(109,878)
|
|
$
(74,422)
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
ordinary share
|
|
|
$
(0.22)
|
|
$
(0.26)
|
|
$
(0.43)
|
|
$
(0.49)
|
Weighted-average basic and diluted
ordinary shares
|
|
|
266,025,783
|
|
173,860,491
|
|
255,131,873
|
|
173,843,249
|
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)
Obecabatagene autoleucel (obe-cel)
is a B-lymphocyte antigen CD19 (CD19) chimeric antigen receptor
(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 relapsed/refractory (r/r) Adult B-cell Acute
Lymphoblastic Leukemia (B-ALL) patients. The results of the FELIX
trial, a pivotal trial for adult B-ALL, have been submitted and
accepted by the FDA with a PDUFA target action date of November 16,
2024. 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 obe-cel
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 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