REG - Syncona Limited - Autolus reports Q3 2025 Financial Results
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RNS Number : 3011H Syncona Limited 12 November 2025
12 November 2025
Syncona Limited
("Syncona" or the "Company")
Autolus Therapeutics Reports Third Quarter 2025 Financial Results and Business
Updates
Syncona Ltd, a leading life science investor, notes that its portfolio company
Autolus Therapeutics plc (Nasdaq: AUTL) ("Autolus") announced its operational
and financial results for the third quarter ended 30 September 2025. Key
highlights are as follows:
· The company reported Q3 2025 AUCATZYL® net product revenue of $21.1
million and deferred revenue of $7.6 million; 60 authorized treatment centres
achieved ahead of target
· Clinical execution and data generation to support market growth and
expansion continues; data in severe refractory systemic lupus erythematosus
(srSLE) show no ICANS or high-grade CRS, demonstrate achievement of definition
of remission in SLE (DORIS) in 83% (n=5/6) of patients and complete renal
response (CRR) in 50% (n=3/6) of patients
· Leadership team bolstered to support next phase of growth and
optimization of business operations
Autolus' announcement is copied below and can be accessed at the company's
investor website at https://www.autolus.com/investor-relations/news
(https://www.autolus.com/investor-relations/news) . To listen to the webcast
and view the accompanying slide presentation, please go to:
https://www.autolus.com/investor-relations/events/
(https://www.autolus.com/investor-relations/events/) .
Enquiries
Syncona Ltd
Annabel Clark
Tel: +44 (0) 20 3981 7940
FTI Consulting
Ben Atwell / Natalie Garland-Collins / Tim Stamper
Tel: +44 (0) 20 3727 1000
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 2025 Financial Results and
Business Updates
· Company reports Q3 2025 AUCATZYL® net product revenue of $21.1
million and deferred revenue of $7.6 million; 60 authorized treatment centers
achieved ahead of target
· Clinical execution and data generation to support market growth and
expansion continues; data in severe refractory systemic lupus erythematosus
(srSLE) show no ICANS or high-grade CRS, demonstrate achievement of definition
of remission in SLE (DORIS) in 83% (n=5/6) of patients and complete renal
response (CRR) in 50% (n=3/6) of patients
· Leadership team bolstered to support next phase of growth and
optimization of business operations
· Conference call to be held today at 8:30 am EST / 1:30 pm GMT;
conference call participants should pre-register using the link at the bottom
of this press release
LONDON & Gaithersburg, MD, November 12, 2025 -- Autolus Therapeutics
plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company
developing, manufacturing and delivering next-generation programmed T cell
therapies, announces its operational and financial results for the third
quarter ended September 30, 2025.
"Through three quarters of launch, we are encouraged by our progress to
increase the overall market in r/r B-ALL, reaching patients who previously may
not have been considered for CAR T therapy. With mounting experience we see
physician enthusiasm for AUCATZYL increasing, validated by real world data
from the ROCCA Consortium to be presented at the ASH Annual Meeting in
December," said Dr. Christian Itin, Chief Executive Officer of Autolus.
"Despite an expected temporary lag in Q3 sales based on the change in CMS
reimbursement policy that occurred in Q2, we executed well on new patient
starts and project a strong full year of sales."
Dr. Itin continued, "Building on our strong commercial and manufacturing
performance, we enter our next phase of growth for obe-cel focused on three
key objectives. First, increasing market share within the indicated adult ALL
population; second, based on strong Phase 1 data sets we are conducting
potential pivotal studies in pediatric ALL and in severe lupus nephritis to
broaden the utility and commercial opportunity of obe-cel; and finally, we
continue to innovate on manufacturing technology as a foundation for further
expansion of access to CAR T therapies."
Product and Pipeline Updates:
· AUCATZYL® Launch
o Autolus reported net product revenue of $21.1 million for the three months
ended September 30, 2025 and deferred revenue of $7.6 million as of September
30, 2025.
o The Company has 60 centers fully activated in the U.S. as of November 12,
2025, achieving the target of 60 activated centers prior to year-end.
o Patient access to AUCATZYL continues to increase, with coverage secured for
greater than 90% of total U.S. medical lives.
o Data from the ROCCA (Real-World Outcomes Collaborative for CAR T in Adult
ALL) database evaluating patient characteristics, toxicity and response after
real-world administration of AUCATZYL (obecabtagene autoleucel) and TECARTUS
(brexucabtagene autoleucel) for relapsed acute lymphoblastic leukemia with r/r
ALL will be presented at the American Society of Hematology (ASH) Annual
Meeting.
· Obe-cel data in pediatric r/r B-ALL
o Data from the ongoing Phase Ib/II CATULUS study evaluating the safety and
efficacy of obe-cel in patients under 18 years with CD19-positive r/r B-ALL or
B-cell Non-Hodgkin Lymphoma (NHL) will be presented at the American Society of
Hematology Annual Meeting. Data show the safety profile of obe-cel in
pediatric patients is consistent with that previously reported in adults, with
low rates of high-grade cytokine release syndrome (CRS) and immune effector
cell-associated neurotoxicity syndrome (ICANS). Overall response rate (ORR)
was high at 95% and nearly 90% of responders had ongoing remission at data
cut-off. Additional data will be presented in a poster presentation at the ASH
Annual Meeting on December 7, 2025, from 6:00 - 8:00pm ET.
o In October 2025, the U.S. Food and Drug Administration (FDA) granted
regenerative medicine advanced therapy (RMAT) designation to obe-cel for the
treatment of pediatric patients with r/r B-ALL. The RMAT designation is a
program created under the 21st Century Cures Act to accelerate development and
regulatory review of regenerative medicine therapies, including cell
therapies, intended to treat serious or life-threatening diseases.
· Obe-cel in lupus nephritis (LN)
o Data from the Phase 1 CARLYSLE clinical trial were presented
(https://autolus.gcs-web.com/news-releases/news-release-details/autolus-therapeutics-presents-clinical-data-updates-american-1)
on October 28, 2025, at the American College of Rheumatology (ACR) Convergence
2025. Data in severe refractory systemic lupus erythematosus (srSLE) suggests
obe-cel is well tolerated with no ICANS or high-grade CRS. Preliminary
efficacy data demonstrate achievement of definition of remission in SLE
(DORIS) in 83% (n=5/6) of patients and complete renal response (CRR) in 50%
(n=3/6) of patients. All responses and remissions are ongoing with no evidence
of disease activity at a median follow-up of 8.9 months (range 6-13.8 months).
o Additional findings from the ongoing CARLYSLE study will be presented in an
oral presentation at the American Society of Hematology (ASH) Annual Meeting
2025 on December 8, 2025, at 11:30am ET. Data show the B-cell reconstitution
profiles suggest that obe-cel may induce a reset of pathologic autoimmunity.
Updated Phase 1 data with longer follow-up, and data in patients who received
100×10(6) CAR T-cells will be presented.
o The Company has previously aligned with the FDA on the Phase 2 trial design
and potential registrational path to approval. Data to date supported
progressing into the Phase 2 LUMINA trial. The first patient is expected to be
dosed prior to year-end 2025.
· Obe-cel in progressive MS
o Autolus is advancing obe-cel into initial clinical development in
progressive MS. The first patient in the BOBCAT trial was dosed
(https://autolus.gcs-web.com/news-releases/news-release-details/autolus-therapeutics-announces-first-patient-dosed-phase-1-trial)
in October 2025. The Phase 1 trial, expected to include up to 18 adult
patients, will determine the safety, tolerability, and preliminary efficacy of
obe-cel in participants with refractory progressive forms of multiple
sclerosis. The primary endpoint is to assess safety and tolerability of
obe-cel. Key secondary endpoints include evaluating the preliminary efficacy
of obe-cel using change from baseline in standard efficacy measures.
· Early-stage pipeline programs and collaborations
o In November 2025, Moderna announced that the first patient has been dosed in
a Phase 1/2 study of mRNA-2808, an investigational mRNA-based T-cell engager
for participants with relapsed or refractory multiple myeloma. mRNA-2808
utilizes an Autolus proprietary binder that was licensed to Moderna in 2022.
o Long term follow up from the AUTO8 MCARTY study in multiple myeloma will be
presented in a poster presentation at the ASH Annual Meeting:
Abstract ID: abs25-14286
Title: CAR-T cells co-targeting BCMA and CD19 in RRMM: Results from the UKMRA
Phase 1 MCARTY trial in relapsed refractory multiple myeloma
Date: December 7, 06:00 PM - 08:00 PM EST
Location: OCCC - West Halls B3-B4
Presenting Author: Lydia Lee, MBBS, MRCP, MRCPath (Haem), PhD
The first patient in the AUTO8 ALARIC study in AL Amyloidosis is expected to
be dosed prior to year-end 2025.
o Autolus' translational programs with UCL continue to fuel its early-stage
pipeline, providing a cost-efficient path to development to support long-term
growth.
Operational Updates:
Autolus announced leadership team changes to support the next phase of
commercial growth, margin improvement and market expansion.
· Cintia Piccina was appointed U.S. Chief Commercial Officer and
Country General Manager to lead ongoing momentum in AUCTAZYL® U.S. launch and
drive future growth. Cintia brings to Autolus extensive cellular therapy
experience having led teams that successfully launched and commercialized
multiple products in Novartis, Bluebird/2seventy bio, Allovir and Adaptimmune,
where she was most recently the Chief Commercial Officer.
· Miranda Neville was appointed Chief Technical Officer to drive
manufacturing optimization, succeeding David Brochu who will continue as an
advisor. Ms. Neville joined Autolus in 2018 from the consulting firm
AllianceBIO where she spent four years as a Partner and supported several
clinical stage and commercial biopharmaceutical companies. Ms. Neville began
her career at Human Genome Sciences (HGS). She spent 10 years at HGS in a
variety of roles including Manufacturing, Engineering & Program
Management, prior to its acquisition by GlaxoSmithKline.
· Patrick McIlvenny was appointed Senior Vice President, Finance and
Chief Accounting Officer. Before joining Autolus, Mr. McIlvenny served as
Senior Vice President, Chief Accounting Officer for Horizon Therapeutics plc,
until the acquisition of Horizon by Amgen, and in various finance roles of
increasing responsibilities at Ardagh Group S.A and Elan Corporation plc.
Prior to joining Elan, Mr. McIlvenny worked with PricewaterhouseCoopers and
Deloitte. Mr. McIlvenny is a Fellow of the Institute of Chartered Accountants
in England and Wales.
Dr. Itin commented, "Our new team members bring a breadth of leadership
experience and will focus on market growth, strategic planning and operational
excellence driving growth and efficiency of the ALL business. We are grateful
to our prior team members who were instrumental in setting our organization on
the right course for a successful launch."
Summary of Anticipated News Flow:
ALL: Initial clinical data from CATULUS trial in pediatric ALL December 7, 2025
SLE: Longer-term follow up data from CARLYSLE trial
December 8, 2025
LN: Expect to dose first patient in Phase 2 LUMINA trial By year-end 2025
ALA: Expect to dose first patient in Phase 1 ALARIC trial in AL amyloidosis By year-end 2025
ALL: acute lymphoblastic leukemia
SLE: systemic lupus erythematosus
LN: lupus nephritis
ALA: light-chain amyloidosis
Financial Results for the Quarter Ended September 30, 2025
Product revenue, net for the three months ended September 30, 2025, was $21.1
million. Deferred revenue balance at September 30, 2025, was $7.6 million,
representing product that was shipped and received by centers but not yet
dosed.
Cost of sales for the three months ended September 30, 2025, totaled $28.6
million. This amount includes the cost of all commercial product delivered to
the authorized treatment centers, including product delivered but not yet
recorded as product revenue which is captured as deferred revenue.
Additionally, cost of sales includes any cancelled orders in the period,
patient access program product, inventory reserves and write-offs and 3(rd)
party royalties for certain technology licenses.
Research and development expenses decreased from $40.3 million to $27.9
million for the three months ended September 30, 2025, compared to the same
period in 2024. This change was primarily due to commercial
manufacturing-related employee and infrastructure costs shifting to cost of
sales and inventory.
Selling, general and administrative expenses increased from $27.3 million to
$36.3 million for the three months ended September 30, 2025, compared to the
same period in 2024. This increase was primarily due to salaries and other
employment-related costs, driven by increased headcount supporting
commercialization activities.
Loss from operations for the three months ended September 30, 2025, was $71.6
million, as compared to $67.9 million for the same period in 2024.
Net loss was $79.1 million for the three months ended September 30, 2025,
compared to $82.1 million for the same period in 2024. Basic and diluted net
loss per ordinary share for the three months ended September 30, 2025, totaled
$(0.30), compared to basic and diluted net loss per ordinary share of $(0.31)
for the same period in 2024.
Cash, cash equivalents and marketable securities at September 30, 2025,
totalled $367.4 million, as compared to $588.0 million at December 31, 2024.
The decrease was primarily driven by net cash used in operating activities and
impacted by a delayed cash receipt of approximately $20.1 million in the
Company's 2023 R&D tax credit expected from the UK HMRC.
Autolus estimates that, with its current cash and cash equivalents and
marketable securities, the Company is well capitalized to drive the launch and
commercialization of obe-cel in r/r B-ALL and to generate data in the LN and
pALL potential pivotal trials and MS Phase 1 trial
Financial Results for the Period Ended September 30, 2025
Selected Consolidated Balance Sheet Data
(In thousands)
September 30 December 31
2025 2024
Assets
Cash and cash equivalents $ 86,124 $ 227,380
Marketable securities - Available-for-sale debt securities $ 281,289 $ 360,643
Total current assets $ 514,577 $ 660,929
Total assets $ 661,947 $ 782,725
Liabilities and shareholders' equity
Total current liabilities $ 83,071 $ 60,743
Total liabilities $ 396,495 $ 355,400
Total shareholders' equity $ 265,452 $ 427,325
Selected 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,
2025 2024 2025 2024
Product revenue, net $ 21,144 $ - $ 51,049 $ -
License revenue 50 - - 50 - 10,091
Cost and operating expenses:
Cost of sales (28,643) - (71,039) -
Research and development expenses, net (27,892) (40,323) (82,056) (107,606)
Selling, general and administrative expenses (36,280) (27,330) (96,079) (67,410)
Loss on disposal of property and equipment - (223) (3) (223)
Impairment of operating lease right-of-use assets and related property and - - - (414)
equipment
Loss from operations (71,621) (67,876) (198,078) (165,562)
Total other (expenses) income, net (6,965) (14,196) 4,037 (27,428)
Net loss before income tax (78,586) (82,072) (194,041) (192,990)
Income tax expenses (532) (22) (3,155) (66)
Net loss attributable to ordinary shareholders (79,118) (82,094) (197,196) (193,056)
Other comprehensive (loss) income, net of tax (5,782) 27,010 24,254 28,094
Total comprehensive loss $ (84,900) $ (55,084) $ (172,942) $ (164,962)
Basic and diluted net loss per ordinary share $ (0.30) $ (0.31) $ (0.74) $ (0.77)
Weighted-average basic and diluted ordinary shares 266,141,431 266,084,589 266,136,518 255,480,521
Conference Call
Management will host a conference call and webcast today at 8:30am EST/1:30pm
GMT to discuss the company's financial results. Conference call participants
should pre-register using this link
(https://register-conf.media-server.com/register/BI9146f14735fc412bab1c8e128c95ad3e)
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 at
https://www.autolus.com/investor-relations-media/events/
(https://www.autolus.com/investor-relations-media/events/) .
About Autolus Therapeutics plc
Autolus Therapeutics plc (Nasdaq: AUTL) is an early commercial-stage
biopharmaceutical company developing, manufacturing and delivering
next-generation T cell therapies and candidates 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 and
controlled T cell therapies that are designed to better recognize target
cells, break down their defense mechanisms and eliminate these cells. Autolus
has a marketed therapy, 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
(http://www.autolus.com/) .
About AUCATZYL® (obecabtagene autoleucel, obe-cel)
AUCATZYL is a B-lymphocyte antigen CD19 (CD19) chimeric antigen receptor (CAR)
T cell therapy designed to overcome the limitations in clinical activity and
safety compared to current CD19 CAR T cell therapies. AUCATZYL is designed
with a fast target binding off-rate to minimize excessive activation of the
programmed T cells. AUCATZYL was approved by the FDA for the treatment of
adult patients with relapsed or refractory B-cell precursor acute
lymphoblastic leukemia on November 8, 2024, and was granted conditional
marketing authorization by MHRA in the UK and EMA in the EU in 2025.
INDICATION
AUCATZYL® is a CD19-directed genetically modified autologous T cell
immunotherapy indicated for the treatment of adult patients with relapsed or
refractory B-cell precursor acute lymphoblastic leukemia (ALL).
IMPORTANT SAFETY INFORMATION
WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, and SECONDARY
HEMATOLOGICAL MALIGNANCIES
• Cytokine Release Syndrome (CRS) occurred in
patients receiving AUCATZYL. Do not administer AUCATZYL to patients with
active infection or inflammatory disorders. Prior to administering AUCATZYL,
ensure that healthcare providers have immediate access to medications and
resuscitative equipment to manage CRS [see Dosage and Administration (2.2,
2.3), Warnings and Precautions (5.1)].
• Immune Effector Cell-Associated Neurotoxicity
Syndrome (ICANS), including fatal or life-threatening reactions, occurred in
patients receiving AUCATZYL, including concurrently with CRS or after CRS
resolution. Monitor for neurologic signs and symptoms after treatment with
AUCATZYL. Prior to administering AUCATZYL, ensure that healthcare providers
have immediate access to medications and resuscitative equipment to manage
neurologic toxicities. Provide supportive care and/or corticosteroids, as
needed [see Dosage and Administration (2.2, 2.3), Warnings and Precautions
(5.2)].
• T cell malignancies have occurred following
treatment of hematologic malignancies with BCMA- and CD19-directed genetically
modified autologous T cell immunotherapies [see Warnings and Precautions
(5.8)].
WARNINGS AND PRECAUTIONS
Cytokine Release Syndrome (CRS)
Cytokine Release Syndrome (CRS) occurred following treatment with AUCATZYL.
CRS was reported in 75% (75/100) of patients including Grade 3 CRS in 3% of
patients. The median time to onset of CRS was 8 days following the first
infusion (range: 1 to 23 days) with a median duration of 5 days (range: 1 to
21 days). The most common manifestations of CRS included fever (100%),
hypotension (35%), and hypoxia (19%).
Cytokine Release Syndrome (CRS) occurred following treatment with AUCATZYL.
CRS was reported in 75% (75/100) of patients including Grade 3 CRS in 3% of
patients. The median time to onset of CRS was 8 days (range: 1 to 23 days)
with a median duration of 5 days (range: 1 to 21 days). Sixty-eight percent of
patients (51/75) experienced CRS after the first infusion, but prior to the
second infusion of AUCATZYL with a median time to onset of 6 days (range: 1 to
10 days). Among patients with CRS, the most common manifestations of CRS
included fever (100%), hypotension (35%) and hypoxia (19%). The primary
treatment for CRS was tocilizumab (73%; 55/75), with patients also receiving
corticosteroids (21%; 16/75).
Prior to administering AUCATZYL, ensure that healthcare providers have
immediate access to medications and resuscitative equipment to manage CRS.
During and following treatment with AUCATZYL, closely monitor patients for
signs and symptoms of CRS daily for at least 7 days following each infusion.
Continue to monitor patients for CRS for at least 2 weeks following each
infusion with AUCATZYL. Counsel patients to seek immediate medical attention
should signs or symptoms of CRS occur at any time. At the first sign of CRS,
immediately evaluate the patient for hospitalization and institute treatment
with supportive care based on severity and consider further management per
current practice guidelines.
Neurologic Toxicities
Neurologic toxicities including Immune Effector Cell-associated Neurotoxicity
Syndrome (ICANS), which were fatal or life-threatening, occurred following
treatment with AUCATZYL. Neurologic toxicities were reported in 64% (64/100)
of patients, including Grade ≥ 3 in 12% of patients.
The median time to onset of neurologic toxicities was 10 days (range: 1 to 246
days) with a median duration of 13 days (range: 1 to 904 days). Fifty-five
percent of patients (35/64) experienced neurologic toxicities after the first
infusion but prior to the second infusion of AUCATZYL with a median time to
onset of 6 days (range: 1 to 11 days). Among patients with neurologic
toxicities, the most common symptoms (> 5%) included ICANS (38%), headache
(34%), encephalopathy (33%), dizziness (22%), tremor (13%), anxiety (9%),
insomnia (9%), and delirium (8%).
Immune Effector Cell-associated Neurotoxicity Syndrome (ICANS)
ICANS events occurred in 24% (24/100) of patients, including Grade ≥ 3 in 7%
(7/100) of patients. Of the 24 patients who experienced ICANS, 33% (8/24)
experienced an onset after the first infusion, but prior to the second
infusion of AUCATZYL. The median time to onset for ICANS events after the
first infusion was 8 days (range: 1 to 10 days) and 6.5 days (range: 2 to 22
days) after the second infusion, with a median duration of 8.5 days (range: 1
to 53 days). Eighty-eight percent (21/24) of patients received treatment for
ICANS. All treated patients received high-dose corticosteroids and 42% (10/24)
of patients received anti-epileptics prophylactically. Prior to administering
AUCATZYL, ensure that healthcare providers have immediate access to
medications and resuscitative equipment to manage ICANS.
During and following AUCATZYL administration, closely monitor patients for
signs and symptoms of Neurologic Toxicity/ICANS. Following treatment with
AUCATZYL, monitor patients daily for at least 7 days. Continue to monitor
patients for at least 2 weeks following treatment with AUCATZYL. Avoid driving
for at least 2 weeks after each infusion. Counsel patients to seek medical
attention should signs or symptoms of neurologic toxicity/ ICANS occur. At the
first sign of Neurologic Toxicity/ICANS, immediately evaluate patients for
hospitalization and institute treatment with supportive care based on severity
and consider further management per current practice guidelines.
Prolonged Cytopenias
Patients may exhibit cytopenias including anemia, neutropenia, and
thrombocytopenia for several weeks after treatment with lymphodepleting
chemotherapy and AUCATZYL. In patients who were responders to AUCATZYL, Grade
≥ 3 cytopenias that persisted beyond Day 30 following AUCATZYL infusion were
observed in 71% (29/41) of patients and included neutropenia (66%, 27/41) and
thrombocytopenia (54%, 22/41). Grade 3 or higher cytopenias that persisted
beyond Day 60 following AUCATZYL infusion was observed in 27% (11/41) of
patients and included neutropenia (17%, 7/41) and thrombocytopenia (15%,
6/41). Monitor blood counts after AUCATZYL infusion.
Infections
Severe, including life-threatening and fatal infections occurred in patients
after AUCATZYL infusion. Non-COVID-19 infections of all grades occurred in 67%
(67/100) of patients. Grade 3 or higher non-COVID-19 infections occurred in
41% (41/100) of patients. AUCATZYL should not be administered to patients with
clinically significant active systemic infections. Monitor patients for signs
and symptoms of infection before and after AUCATZYL infusion and treat
appropriately. Administer prophylactic antimicrobials according to local
guidelines.
Grade 3 or higher febrile neutropenia was observed in 26% (26/100) of patients
after AUCATZYL infusion and may be concurrent with CRS. In the event of
febrile neutropenia, evaluate for infection and manage with broad-spectrum
antibiotics, fluids, and other supportive care as medically indicated.
Viral reactivation, potentially severe or life-threatening, can occur in
patients treated with drugs directed against B cells. There is no experience
with manufacturing AUCATZYL for patients with a positive test for human
immunodeficiency virus (HIV) or with active hepatitis B virus (HBV) or active
hepatitis C virus (HCV). Perform screening for HBV, HCV and HIV in accordance
with clinical guidelines before collection of cells for manufacturing.
Hypogammaglobulinemia
Hypogammaglobulinemia and B-cell aplasia can occur in patients after AUCATZYL
infusion. Hypogammaglobulinemia was reported in 10% (10/100) of patients
treated with AUCATZYL including Grade 3 events in 2 patients (2%).
Immunoglobulin levels should be monitored after treatment with AUCATZYL and
managed per institutional guidelines including infection precautions,
antibiotic or antiviral prophylaxis, and immunoglobulin replacement.
The safety of immunization with live viral vaccines during or following
treatment with AUCATZYL has not been studied. Vaccination with live viral
vaccines is not recommended for at least 6 weeks prior to the start of
lymphodepleting chemotherapy treatment, during AUCATZYL treatment, and until
immune recovery following treatment with AUCATZYL.
Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome
(HLH/MAS)
HLH/MAS including fatal and life-threatening reactions occurred after
treatment with AUCATZYL. HLH/MAS was reported in 2% (2/100) of patients and
included Grade 3 and Grade 4 events with a time of onset at Day 22 and Day 41,
respectively. One patient experienced a concurrent ICANS events after AUCATZYL
infusion and died due to sepsis with ongoing HLH/MAS that had not resolved.
Administer treatment for HLH/MAS according to institutional standards.
Hypersensitivity Reactions
Serious hypersensitivity reactions, including anaphylaxis, may occur due to
dimethyl sulfoxide (DMSO), an excipient used in AUCATZYL. Observe patients for
hypersensitivity reactions during and after AUCATZYL infusion.
Secondary Malignancies
Patients treated with AUCATZYL may develop secondary malignancies. T cell
malignancies have occurred following treatment of hematologic malignancies
with BCMA- and CD19-directed genetically modified autologous T cell
immunotherapies. Mature T cell malignancies, including CAR-positive tumors,
may present as soon as weeks following infusion, and may include fatal
outcomes. Monitor lifelong for secondary malignancies. In the event that a
secondary malignancy occurs, contact Autolus at 1-855-288-5227 for reporting
and to obtain instructions on the collection of patient samples for testing.
Adverse Reactions
The safety of AUCATZYL was evaluated in the FELIX study in which 100 patients
with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL)
received AUCATZYL at a median dose of 410 × 10(6) CD19 CAR-positive viable T
cells (range: 10 to 480 × 10(6) CD19 CAR-positive viable T cells with 90% of
patients receiving the recommended dose of 410 × 10(6) +/- 25%).
The most common serious adverse reactions of any Grade (incidence ≥ 2%)
included infections-pathogen unspecified, febrile neutropenia, ICANS, CRS,
fever, bacterial infectious disorders, encephalopathy, fungal infections,
hemorrhage, respiratory failure, hypotension, ascites, HLH/MAS, thrombosis and
hypoxia. Nine patients (9%) experienced fatal adverse reactions which included
infections (sepsis, pneumonia, peritonitis), ascites, pulmonary embolism,
acute respiratory distress syndrome, HLH/MAS and ICANS. Of the 9 patients,
five patients who died from infections had pre-existing and ongoing
neutropenia prior to receiving bridging therapy, lymphodepletion chemotherapy
treatment and/or AUCATZYL.
Please see full Prescribing Information
(http://www.autolus.com/AUCATZYL-USPI/) , including BOXED WARNING and
Medication Guide.
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 therapeutic
potential and expected clinical benefits of AUCATZYL/obe-cel (obecabtagene
autoleucel) for adult patients with r/r B-ALL and obe-cel in additional
indications including LN and progressive MS; Autolus' ability to generate
revenues from AUCATZYL, which is dependent upon maintaining significant market
acceptance among physicians, patients and healthcare payors; the effect of
payor reimbursement determinations and other market conditions on Autolus'
ability to recognize revenue from AUCATZYL sales; Autolus' ability to obtain
and maintain regulatory approval for obe-cel for adult r/r B-ALL in additional
territories and the timing thereof; expectations regarding the
commercialization and marketing of AUCATZYL for adult r/r B-ALL, including
expanding into additional territories and the related timing of reaching
patients in such territories; the development of obe-cel in autoimmune
indications and of additional product candidates, including statements
regarding the initiation, timing, progress and the results of clinical studies
or trials and related preparatory work; the period during which the results of
clinical studies or trials will become available; commercialization, marketing
and manufacturing capabilities and strategy for AUCATZYL; the timing or
likelihood of regulatory filings and approvals for product candidates, along
with regulatory developments in the US, EU, the UK and other foreign
countries; size and growth potential of the markets for AUCATZYL and product
candidates, if approved; and estimates regarding expenses, future revenue,
capital requirements and needs for additional financing. 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 the impact of worsening macroeconomic conditions on Autolus'
business, financial position, strategy and anticipated milestones, including
Autolus' ability to conduct ongoing and planned clinical trials; Autolus'
ability to obtain a clinical supply of current or future product candidates or
commercial supply of AUCATZYL or any future approved products; Autolus'
ability to obtain and maintain regulatory approval of its product candidates,
including AUCATZYL and potential expansions into additional indications;
Autolus' ability and plans in continuing to establish and expand a commercial
infrastructure in the US and to successfully launch, market and sell AUCATZYL
and any future approved products; Autolus' ability to successfully expand the
approved indications for AUCATZYL or obtain marketing approval for AUCATZYL in
additional geographies in the future; the delay of any current or planned
clinical trials, whether due to patient enrollment delays or otherwise;
Autolus' ability to successfully demonstrate the safety and efficacy of its
product candidates and gain approval of its product candidates on a timely
basis, if at all; competition with respect to market opportunities; the risk
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;
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 20, 2025 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 (mailto:a.cray@autolus.com)
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