REG - Syncona Limited - Autolus announces acceptance of BLA for obe-cel

SynconaAnnouncement

22/01/2024 12:15

For best results when printing this announcement, please click on link below:
http://newsfile.refinitiv.com/getnewsfile/v1/story?guid=urn:newsml:reuters.com:20240122:nRSV4684Aa&default-theme=true

RNS Number : 4684A  Syncona Limited  22 January 2024

Syncona Limited

 

Autolus announces acceptance of BLA for obe-cel

 

 

22 January 2024

 

Syncona Ltd, a leading healthcare company focused on creating, building and
scaling global leaders in life science, notes that its portfolio company,
Autolus Therapeutics plc (Nasdaq: AUTL) ("Autolus") has announced that the US
Food and Drug Administration (FDA) has accepted its Biologics License
Application (BLA) for obe-cel for patients with relapsed/refractory (r/r)
adult B-cell acute lymphoblastic leukaemia (ALL).

 

Highlights include:

 

·      Under the Prescription Drug User Fee Act (PDUFA), the FDA has set
a target action date of November 16, 2024 for reviewing the application, under
a standard review timeline consistent with recently approved CAR T therapies.
The FDA is not currently planning to hold an advisory committee meeting to
discuss this application

·      The BLA submission is based on data from the pivotal FELIX study
of obe-cel in adult r/r B-ALL, most recently presented at the Annual Meeting
of the American Society for Hematology (ASH) in December 2023

·      Autolus is on track to submit a marketing authorisation
application to the European Medicines Agency (EMA) in H1 CY2024

 

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

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 announces acceptance of Biologics License Application for
obecabtagene autoleucel (obe-cel) as a potential treatment for
relapsed/refractory Adult B-cell Acute Lymphoblastic Leukemia (ALL)

 

-     PDUFA Goal date is November 16, 2024

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

 

LONDON, January 22, 2024 -- Autolus Therapeutics plc (Nasdaq: AUTL), a
clinical-stage biopharmaceutical company developing next-generation programmed
T cell therapies, today announces that the U.S. Food and Drug Administration
(FDA) has accepted its Biologics License Application (BLA) for obecabtagene
autoleucel (obe-cel) for patients with relapsed/refractory (r/r) Adult B-Cell
Acute Lymphoblastic Leukemia (ALL). Under the Prescription Drug User Fee Act
(PDUFA), the FDA has set a target action date of November 16, 2024, a standard
review timeline consistent with recently approved CAR T therapies. The FDA is
not currently planning to hold an advisory committee meeting to discuss this
application.

 

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 were presented at the 2023 American
Society of Clinical Oncology (ASCO) Annual Meeting in June 2023, with updated
data presented at the Annual Meeting of the American Society for Hematology
Meeting (ASH) in December 2023.

 

"Acceptance of the BLA filing is an important milestone for Autolus and we
look forward to continuing our collaboration with the FDA during the review
cycle," commented Dr. Christian Itin, Chief Executive Officer of Autolus.
"With the PDUFA date set for November, we remain focused on preparing for the
potential launch of obe-cel."

 

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
(http://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. Autolus is conducting a Phase 1b study in
paediatric patients with ALL and aggressive B-NHL and iIn collaboration with
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 Company's
anticipated transition plans and timing from a clinical to commercial stage
company. 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:

 

Olivia Manser

+44 (0) 7780 471568

o.manser@autolus.com (mailto:o.manser@autolus.com)

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 (mailto:lauren@investase.com)

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact
rns@lseg.com (mailto:rns@lseg.com)
 or visit
www.rns.com (http://www.rns.com/)
.

RNS may use your IP address to confirm compliance with the terms and conditions, to analyse how you engage with the information contained in this communication, and to share such analysis on an anonymised basis with others as part of our commercial services. For further information about how RNS and the London Stock Exchange use the personal data you provide us, please see our
Privacy Policy (https://www.lseg.com/privacy-and-cookie-policy)
.   END  PFUQKBBQKBKDODB