REG - Syncona Limited - Autolus submits key regulatory filing for obe-cel

SynconaAnnouncement

27/11/2023 12:15

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RNS Number : 7714U  Syncona Limited  27 November 2023

 

 

 

Syncona Limited

 

Autolus submits key regulatory filing for lead programme

 

27 November 2023

 

Syncona Ltd, a leading life science investor focused on creating, building and
scaling a portfolio of global leaders in life science, notes that its
portfolio company, Autolus Therapeutics Plc (Nasdaq: AUTL) ("Autolus"), has
announced that it has submitted its Biologics License Application (BLA) to the
US Food and Drug Administration (FDA) for obecabtagene autoleucel (obe-cel)
for patients with relapsed/refractory (r/r) adult B-Cell Acute Lymphoblastic
Leukaemia (ALL).

 

The BLA submission is based on data from the pivotal FELIX study of obe-cel in
adult r/r B-ALL. The data was presented at the 2023 American Society of
Clinical Oncology (ASCO) Annual Meeting in June 2023 and longer-term follow up
data will be presented at the upcoming Annual Meeting of the American Society
for Hematology (ASH) in December 2023.

 

Autolus has also made significant progress in preparing its manufacturing
footprint for the planned commercial launch of obe-cel, with its new 70,000
sq. foot commercial manufacturing facility, The Nucleus, which has an initial
designed capacity of 2,000 batches per year. Pending approval from the U.S.
Food and Drug Administration for the BLA submission, the company expects to
initiate its commercial launch of obe-cel in the US in CY2024.

 

Autolus also expects to file a Marketing Authorisation Application for obe-cel
in r/r adult ALL with the European Medicines Agency (EMA) in the first half of
CY2024.

 

Chris Hollowood, CEO of Syncona Investment Management Limited, said: "We are
very pleased to see Autolus deliver on this milestone, and we are proud that a
company that was co-founded by Syncona in 2014 has progressed a potentially
transformational therapy through to late-stage development and is now working
towards the point of regulatory approval. We look forward to our continued
close collaboration with the company as it works with the FDA on the
regulatory approval process."

 

Martin Murphy, Director on the Board of Autolus Therapeutics, said: "This is a
significant achievement for Autolus and an exciting moment for patients. The
recent data released from the FELIX pivotal trial showed the potential impact
of obe-cel and we look forward to seeing further long-term follow up data
published at the ASH conference in December 2023."

 

The announcement can be accessed on Autolus' investor website at
https://www.autolus.com/investor-relations/news
(https://www.autolus.com/investor-relations/news) and the full text of the
announcement from Autolus is contained below.

 

 ENDS 

 

Enquiries

 

Syncona Ltd

 

Annabel Clark / Fergus Witt

Tel: +44 (0) 20 3981 7940

 

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.

 

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 Submits Biologics License Application to U.S. Food and
Drug Administration for obecabtagene autoleucel (obe-cel) for Patients with
Relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (ALL)

·      BLA submission includes results from pivotal Phase 2 FELIX study
evaluating obe-cel in relapsed/refractory (r/r) adult B-cell Acute
Lymphoblastic Leukemia (ALL)

·      Company on track to submit a marketing authorization application
to the European Medicines Agency (EMA) in the first half of 2024

 

LONDON, November 27, 2023 - Autolus Therapeutics plc (Nasdaq: AUTL), a
clinical-stage biopharmaceutical company developing next-generation programmed
T cell therapies, today announces that it has submitted a Biologics License
Application (BLA) to the U.S. Food and Drug Administration (FDA) for
obecabtagene autoleucel (obe-cel). Obe-cel is Autolus' lead investigational
chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients
with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia
(ALL).

 

The BLA submission is based on data from the Pivotal Phase 2 FELIX study of
obe-cel in adult r/r B-ALL. The data which were presented at the 2023 American
Society of Clinical Oncology (ASCO) Annual Meeting in June 2023 and will be
updated at the upcoming Annual Meeting of the American Society for Hematology
Meeting in December in San Diego.

 

"We are looking forward to continuing working with the FDA through the
regulatory approval process, commented Dr. Christian Itin, Chief Executive
Officer of Autolus. "I would like to thank the treating physicians, patients,
caregivers, and the dedicated team at Autolus for their support, trust and
commitment for the program to reach this important milestone."

 

Autolus plans to submit a Marketing Authorization Application for obe-cel in
relapsed/refractory ALL to the European Medicines Agency (EMA) in the first
half of 2024.

 

Obe-cel has been granted Orphan Drug Designation by the FDA, Orphan Medical
Product Designation by the EMA, Regenerative Medicine Advanced Therapy (RMAT)
designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA
for adult r/r B-ALL.

 

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, the Company 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. Clinical trials of
obe-cel have demonstrated that this "fast off-rate" profile reduces 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, are being prepared for regulatory
submissions with the FDA and EMA. 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 Ib/II clinical trial of obe-cel enrolled adult patients with
relapsed / refractory B-precursor ALL. The trial had a Phase Ib component
prior to proceeding to the single arm, Phase II clinical trial. The primary
endpoint is overall response rate, and the secondary endpoints include
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 

 

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 development of
Autolus' product candidates, the status of clinical trials (including, without
limitation, expectations regarding the data that is being presented, the
expected timing of data releases and development, as well as completion of
clinical trials) and development timelines for the Company's product
candidates. 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
on March 7, 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.

 

Contact:

 

Julia Wilson

+44 (0) 7818 430877

j.wilson@autolus.com (mailto:j.wilson@autolus.com)

 

Susan A. Noonan

S.A. Noonan Communications

+1-917-513-5303

susan@sanoonan.com (mailto:susan@sanoonan.com)

 

Lauren Williams

Investase

+44 23 9438 7760

lauren@investase.com

 

 

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