REG - Syncona Limited - Autolus presents clinical data updates at EHA

SynconaAnnouncement

10/06/2022 08:15

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RNS Number : 4504O  Syncona Limited  10 June 2022

Syncona Limited

 

Autolus presents clinical data updates at EHA Congress

 

10 June 2022

 

Syncona Ltd, a leading healthcare company focused on founding, building and
funding global leaders in life science, notes that its portfolio company,
Autolus Therapeutics Plc (Nasdaq: AUTL) ("Autolus"), has today announced the
publication of clinical data across multiple programmes at the European
Hematology Association (EHA) Congress, being held between 9-12 June, 2022.

 

Key highlights are as follows:

 

·     Positive early safety and efficacy data from AUTO4 in 10 patients
with T cell lymphoma (TCL), with AUTO4 demonstrating a tolerable safety
profile. As of 26 April 2022, 9 patients were evaluable for efficacy and all 3
evaluable patients treated at the highest dose level achieved complete
metabolic responses (CMR) one month post treatment. 2 of these patients remain
in ongoing CMR at months 3 and 6 respectively, with the other patient
relapsing after 3 months.

 

·   Encouraging early safety and efficacy data in AUTO1/22 in paediatric
acute lymphoblastic leukaemia (pALL), with 9 out of 11 patients achieving
complete response. At a median follow up of 8.7 months, 6 of the 9 responding
patients were in minimal residual disease (MRD) negative complete remission.

 

·    Early safety and efficacy data in AUTO1 (obe-cel) in
relapsed/refractory primary central nervous system lymphoma (PCNSL) from 6
patients.

 

·    Early safety and efficacy data in obe-cel in relapsed/refractory B
cell non-Hodgkin's lymphoma (B-NHL) and chronic lymphocytic leukaemia (CLL).
obe-cel continues to display a favourable safety profile. Of the 20 patients
evaluable for efficacy, the overall response rate was 18 out of 20 patients.

 

The full text announcement from Autolus is contained below and can be accessed
at: https://www.autolus.com/investor-relations
(https://www.autolus.com/investor-relations) . Management will host a
conference call and webcast on 13 June 2022 at 7:30 am ET/12:30 pm BST to
discuss the EHA data. To listen to the webcast and view the accompanying slide
presentation, please go to the events
(https://www.autolus.com/investor-relations/events/) section of Autolus'
website.

 

 ENDS 

 

Copies of this press release and other corporate information can be found on
the company website at: www.synconaltd.com (http://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 risk

 

 

Enquiries

 

Syncona Ltd

 

Natalie Garland-Collins / Fergus Witt

Tel: +44 (0) 7714 916615

 

FTI Consulting

 

Ben Atwell / Julia Bradshaw / 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
founding and building companies to deliver transformational treatments to
patients in areas of high unmet need.

 

Our strategy is to found, build and fund companies around exceptional science
to create a diversified portfolio of 15-20 globally leading healthcare
businesses 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.

 

Autolus Therapeutics Presents Clinical Data Updates at the European Hematology
Association Congress

 

-     AUTO4 shows high level of clinical activity with a novel targeting
approach for patients with T Cell Lymphoma

-     AUTO1/22 demonstrates encouraging and durable responses in children
ineligible for commercial CAR T product

-     Obe-cel shows high level of sustained clinical activity in B-NHL
patients and first activity in Primary CNS Lymphoma

Conference call to be held on Monday June 13, 2022 at 7:30 am EST/12:30 pm
BST

 

LONDON, June 10, 2022 -- Autolus Therapeutics plc (Nasdaq: AUTL), a
clinical-stage biopharmaceutical company developing next-generation programmed
T cell therapies, today announces the publication of clinical data across
multiple programs at the European Hematology Association (EHA) Congress, being
held June 9-12, 2022.

 

Autolus will hold a conference call on Monday June 13 2022 at 7:30 am EST /
12:30 pm BST, which will include participation from; Dr. Steven Horwitz, M.D.,
Department of Medicine, Lymphoma Service, Memorial Sloan Kettering Cancer
Center; Dr. Kate Cwynarski, Chair UK T cell Lymphoma Group, Consultant
Hematologist, University College London Hospital; and Autolus' management
team.

 

"We are excited to be presenting this first clinical data for two new product
candidates, AUTO4 with its unique targeting approach for T cell lymphoma and
AUTO1/22 a dual targeting CAR T product for the treatment of children with
ALL," said Dr. Christian Itin, CEO of Autolus. "With obe-cel progressing
towards pivotal data in the FELIX trial in adult patients with ALL, we are
pleased to show obe-cel's broader utility in B-NHL patients, mirroring the
high level of activity and well manageable safety profile we have seen in
previous trials."

 

"This year's EHA is an important meeting for Autolus with four presentations
providing updates from ongoing clinical studies," said Dr. Martin Pule, Chief
Scientific Officer of Autolus. "In an oral presentation we will present AUTO4
clinical data for the first time. These data suggest that AUTO4 has the
potential to become an important therapeutic option for patients with T cell
lymphoma. In a second presentation, we will present our finding from clinical
testing of AUTO1/22. These data show that AUTO1/22 can induce remission in
children with B-ALL, including in those whose disease was not successfully
treated with commercial CAR T product. Further, data suggest that AUTO1/22 can
prevent antigen escape. In two additional presentations, we demonstrate
incremental obe-cel data in B-NHL and B-CLL, as well as some early data in
PCNSL. Obe-cel continues to have consistent safety and efficacy data across
these indications."

 

"As clinicians, we are always searching for new strategies to address unmet
needs in aggressive blood cancers," said Dr. Steven Horwitz,
M.D., Department of Medicine, Lymphoma Service, Memorial Sloan Kettering
Cancer Center, New York. "T Cell Lymphomas are particularly challenging, and
I've been following Dr. Pule's strategy of CAR T targeting based on the
mutually exclusive expressions of TRBC1 or TRBC2 with great interest. Any
advance in bringing new effective therapies to patients with T cell lymphomas
is of great importance."

 

Data presentations:

 

1.   Title: Safety and preliminary efficacy findings of AUTO4, a
TRBC1-targetting CAR, in relapsed/refractory TRBC1 positive selected T Cell
Non-Hodgkin Lymphoma

Session Title: Gene therapy and cellular immunotherapy - Clinical 2

Session date and time: Saturday, June 11 2022 16:30 - 17:45 CEST

Session room: Hall Strauss 1-2

Final Abstract Code:  S261

Presenting Author: Kate Cwynarski

Conclusions: As of April 26 2022, 10 patients with TRBC1-positive r/r T-cell
lymphoma (Peripheral T-cell lymphoma Not Otherwise Specified (PTCL-NOS),
Angioimmunoblastic T-cell lymphoma (AITL), Anaplastic Large cell lymphoma
(ALCL)) have been treated with AUTO4 in a Phase I dose escalation trial. Three
patients had prior stem cell transplantation. After lymphodepletion with
Flu/Cy, patients received either 25, 75, 225 or 450 x 10(6) CAR T cells. AUTO4
demonstrated a tolerable safety profile, with no patient experiencing any dose
limiting toxicities, and no neurotoxicity/immune effector cell-associated
neurotoxicity (ICANS) and no Grade 3 or higher infections. CRS was only seen
at the highest dose level of 450 x 10(6) CAR T cells (Grade 3 in 1 patient;
Grade 1-2 in 3 patients). As of 26 April 2022, 9 patients were evaluable for
efficacy. At the highest dose level 3 of the 3 patients dosed achieved a
complete metabolic remission (CMR) at 1 month. 2 of these patients remain in
ongoing CMR by PET-CT at Month 3 and 6 respectively, whilst the 3(rd) relapsed
at 3 months.

 

2.   Title: Dual antigen targeting with co-transduced CD19/22 CAR T cells
for relapsed/refractory ALL (AUTO1/22)

Session Title: Gene therapy and cellular immunotherapy - Clinical 1

Session date and time: Saturday, June 11 2022 11:30 - 12:45 CEST

Session room: Hall Strauss 1-2

Final Abstract Code:  S259

Presenting Author: Sara Ghorashian

Conclusions: As of May 27 2022, in 11 treated patients, we have reproducibly
generated a product that is balanced in CD19 and CD22 CAR expression, with
predominance of dual CAR T cells and having a mostly central memory phenotype.
To date and in Kymriah-ineligible patients, AUTO1/22 has demonstrated a
favorable safety profile. There have been no incidences of severe CRS, and one
Grade 4 ICANS which was indistinguishable from chemotherapy-related
leukoencephalopathy. We have seen excellent CAR T expansion, with only 4
patients losing CAR T persistence at the last follow up. Overall, 9 out of 11
patients achieved complete response, and there were 2 non-responders. Notably,
2 out of 3 patients with CD19-ve disease achieved complete response
demonstrating the efficacy of the CD22 CAR. Two patients relapsed with
CD19+CD22+ disease, a further patient had emergence of molecular MRD and all
these events were associated with lack of CAR T Cell persistence. No antigen
negative relapses were seen in responding patients. At a median follow up of
8.7 months, 6 of 9 responding patients were in MRD-negative complete remission
(1-12 months) and the median duration of b-cell aplasia has not been reached.

 

3.   Title: Safety and efficacy findings of AUTO1, a fast off-rate CD19 CAR,
in relapsed/refractory Primary CNS Lymphoma

Session Title: Poster session

Session date and time: Friday, June 10 2022 - 16:30 - 17:45 CEST

Final Abstract Code:  P1460

Presenting Author: Claire Roddie

Conclusions: Excellent AUTO1 expansion was observed in the peripheral blood by
qPCR, with persistence in all treated patients at last follow-up. No grade
>/=3 CRS was observed using IV or I-VEN AUTO1 administration. Two cases of
grade 3 ICANS were reported following IV infusion. In the first case the
patient had several neurological deficits that evolved despite ICANS treatment
and were compatible with progressive PCNSL, as confirmed with the month 1 MRI
scan. The second case was a patient whose neurological deficits improved with
steroids/anakinra. Encouraging response rates were observed: of 6 patients
evaluable for efficacy following IV AUTO1, the ORR was 4/6 (67%), with 2 CRs
and 2 PRs. These four responding patients are without disease progression at
last follow up. Two patients died from progressive PCNSL on study. Longer
follow-up is needed and enrolment of additional patients is ongoing.

 

4.   Title: Safety and efficacy findings of AUTO1, a fast off-rate CD19 CAR,
in relapsed/refractory B-Cell Non-Hodgkin's Lymphoma (B-NHL), and chronic
Lymphocytic Leukemia (CLL) / Small Lymphocytic Lymphoma (SLL)

Session Title: Poster session

Session date and time: Friday, June 10 2022 - 16:30 - 17:45 CEST

Final Abstract Code:  P1459

Presenting Author: Claire Roddie

Conclusions: AUTO1 continues to display a favorable safety profile with no
ICANS or Grade ≥ 3 CRS. Long term persistence of AUTO1 in the peripheral
blood was demonstrated by qPCR. Of the 20 patients evaluable for efficacy, the
overall response rate was 18/20 (90%). In the B-NHL cohorts the CRR was 16/17
(94%) (FL: 7/7, MCL: 3/3, DBCL: 6/7). In the CLL cohort a best response of a
PR was achieved in 2/3 patients, notably both achieved MRD-negativity in their
marrow and both remain in PR at 10 and 6 months respectively.  Of the
responding MCL, DLBCL, FL and CLL patients, 17/18 (94%) are without disease
progression at last follow-up. One MCL patient relapsed six months following
treatment and 1 FL patient died in CR from COVID-19. Longer follow-up and
enrolment of additional MCL, FL, DLBCL and CLL patients is ongoing.

 

 

# # #

Conference Call

Management will host a conference call and webcast on June 13, 2022 at 7:30
am ET/12:30 pm BST to discuss the EHA data. To listen to the webcast and view
the accompanying slide presentation, please go to the events section
(https://www.autolus.com/investor-relations/news-and-events/events) of
Autolus' website.

 

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada
callers or (409) 217-8320 for international callers. Please reference
conference ID: 6594553. After the conference call, a replay will be available
for one week. To access the replay, please dial (855) 859-2056 for U.S. and
Canada callers or (404) 537-3406 for international callers. Please reference
conference ID: 6594553.

About Autolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing
next-generation, programmed T cell therapies for the treatment of cancer.
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 cancer
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 and solid tumors. 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. Designed to have a fast target binding off-rate to minimize
excessive activation of the programmed T cells, obe-cel may reduce toxicity
and be less prone to T cell exhaustion, which could enhance persistence and
improve the ability of the programmed T cells to engage in serial killing of
target cancer cells. In collaboration with Autolus' academic partner, UCL,
obe-cel is currently being evaluated in a Phase 1 clinical trials for B-NHL.
Autolus has progressed obe-cel to the FELIX trial, a potential pivotal trial
for adult ALL.

About obe-cel FELIX clinical trial

Autolus' Phase 1b/2 clinical trial of obe-cel is enrolling 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 is overall response rate, and the secondary endpoints include
duration of response, MRD negative CR rate and safety. The trial is designed
to enroll approximately 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 &
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 called CARPALL for patients
with r/r pediatric ALL. [NCT02443831
(https://clinicaltrials.gov/ct2/show/NCT02443831) ]

About AUTO4

AUTO4 is a programmed T cell product candidate in clinical development for T
cell lymphoma, a setting where there are currently no approved programmed T
cell therapies. AUTO4 is specifically designed to target TRBC1 derived
cancers, which account for approximately 40% of T cell lymphomas, and is a
complement to the AUTO5 T cell product candidate, which is in pre-clinical
development.  AUTO4 has been tested in a Phase 1 clinical trial, LibRA1 for
patients with peripheral T cell Lymphoma.

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 of
the obe-cel program; the future clinical development, efficacy, safety and
therapeutic potential of its product candidates, including progress,
expectations as to the reporting of data, conduct and timing and potential
future clinical activity and milestones; expectations regarding the
initiation, design and reporting of data from clinical trials; expectations
regarding regulatory approval process for any product candidates; the
collaboration between Autolus and Blackstone; the discovery, development and
potential commercialization of potential product candidates including obe-cel
using Autolus' technology and under the collaboration agreement; the
therapeutic potential for Autolus in next generation product developments of
obe-cel in B-cell malignancies; the potential and timing to receive milestone
payments and pay royalties under the strategic collaboration; and the
Company's anticipated cash runway. 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; possible safety and efficacy concerns; and
the impact of the ongoing COVID-19 pandemic on Autolus' business. 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 10, 2022, 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)

 

 

# # #

 

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