Syncona
Limited
Quell provides updates on
QEL-001 and LIBERATE trial
5 June 2024
Syncona Ltd, a leading life science
investor focused on creating, building and scaling global leaders
in life science, notes that its portfolio company, Quell
Therapeutics (Quell), has announced that it is advancing QEL-001,
its autologous, multi-modular engineered CAR-Treg cell therapy,
into the efficacy cohort of the LIBERATE Phase I/II trial in liver
transplant patients. Quell's decision to advance the trial follows
successful completion of dosing in the initial safety cohort of
three patients, from which data were presented yesterday at the
American Transplant Congress. A copy of Quell's announcement is set
out below, with key highlights as follows:
· Data
presented from this first-in-human study show QEL-001 to be safe
and well tolerated, with no serious adverse events or dose-limiting
toxicities reported to date
· The
study confirmed that QEL-001 cells migrated to the transplanted
liver following infusion, with persistence of those infused cells
for up to six months
· The
data support further investigation of QEL-001 CAR-Treg therapy to
induce operational tolerance in liver transplant patients and
decrease the side effects of immunosuppressants (IS)
· Quell
will now proceed with evaluating QEL-001 in the efficacy cohort of
liver transplant patients, assessing the induction of tolerance at
two and 12 months after full IS withdrawal
· The
decision to advance the trial to the efficacy cohort follows the
initial data review and subsequent approval by the trial's
independent Data Safety and Monitoring Board (DSMB)
Elisa Petris, Lead Partner of Syncona Investment Management
Limited and Non-executive Director of Quell Therapeutics,
said: "This is the first time a
therapy of this kind has been assessed in humans as a potential
treatment for supporting successful liver transplantation. We are
encouraged by the initial data showing QEL-001 to be safe and well
tolerated, and look forward to the trial advancing to efficacy
evaluation. Patients undergoing liver transplantation currently
face the prospect of lifelong immunosuppression, which can
seriously impact quality of life, and we are excited about Quell's
novel approach to address this."
[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
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documentation, before investing.
Quell Therapeutics Advances
QEL-001, its Multi-modular Engineered CAR-Treg Cell Therapy, into
Efficacy Cohort of LIBERATE Phase 1/2 Trial in Liver Transplant
Patients
Progress in the LIBERATE trial was presented at the American
Transplant Congress today
London, UK-
June 4, 2024 - Quell Therapeutics Ltd
("Quell"), a leader in developing engineered
T-regulatory (Treg) cell therapies for serious medical conditions
driven by the immune system, announces that it is advancing
QEL-001, its autologous engineered CAR-Treg cell therapy, into the
efficacy cohort of the LIBERATE Phase 1/2 trial in liver transplant
patients. The decision to advance the trial follows the successful
completion of dosing in the initial safety cohort (n=3) and
subsequent approval by the trial's independent Data Safety and
Monitoring Board (DSMB) following its review of the clinical
data.
Progress in the LIBERATE study was presented
today at the American
Transplant Congress in Philadelphia, PA, USA by Prof.
Alberto Sánchez-Fueyo, Professor of Hepatology and Academic
Director of the Institute of Liver Studies at Kings College London,
and co-founder of Quell. The presentation concluded:
·
Tregs from liver transplant patients can be isolated and
expanded to generate engineered CAR-Tregs with improved safety and
therapeutic characteristics (including the proprietary Foxp3
Phenotype Lock™ and a safety switch) for clinical use.
·
Infusion of QEL-001 was shown to be safe and well tolerated
in the safety cohort (n=3)
·
Engraftment and trafficking of QEL-001 to the liver allograft
and persistence of infused cells for up to six months in the
periphery post infusion was confirmed.
·
These initial data support further investigation of QEL-001
CAR-Treg therapy in HLA-A2 mismatched liver transplant patients to
induce operational tolerance and decrease the side-effects of
immunosuppressive (IS) therapy.
Quell will now proceed with evaluating QEL-001
in the efficacy cohort of liver transplant patients, which will
assess the induction of tolerance at two and 12 months after full
withdrawal of IS therapy. The ability to wean patients off IS at
the two-month evaluation point is expected to be highly predictive
of achieving longer-term operational tolerance.
Luke Devey,
Chief Medical Officer, Quell Therapeutics, said:
"We are very excited to enter the next
phase of this clinical trial with QEL-001 in the liver
transplantation setting. This is an important trial for liver
transplant patients, who still face the prospect of lifelong
systemic immunosuppression and the serious complications and
negative impact on quality of life that brings, including increased
rates of malignancies, infections and renal failure. LIBERATE has
been designed to test a new, potentially transformational paradigm
for these patients, with QEL-001 designed to bring targeted and
durable immune tolerance and eliminate their need for chronic
immunosuppression."
Notes to
Editors
About
QEL-001
QEL-001 is a first-in-class, antigen-specific
CAR-Treg cell therapy, designed using Quell's unique multi-modular
engineered Treg platform. QEL-001 comprises three proprietary
modules: a chimeric antigen receptor (CAR) for tissue targeting,
the Foxp3 Phenotype Lock™ module, and a safety switch. The QEL-001
CAR is specific for HLA-A2, which localizes its activity to the
site of the transplanted organ in HLA-A2 mismatched liver
transplant patients (i.e. HLA-A2 negative recipients who received
an HLA-A2 positive donor liver).
About the
LIBERATE Trial
The LIBERATE trial (NCT05234190)
is a multi-center, first-in-human, open-label, single-arm Phase 1/2
study evaluating the safety and efficacy of QEL-001 in
approximately 18 HLA-A2 mismatched liver transplant
patients, 1-5yr post liver transplant, at
sites in the UK, Belgium and Spain.
Safety cohort (n = 3) - No ATG conditioning and
no removal of immuno-suppression to assess safety of QEL-001
therapy at 28 days post infusion.
Efficacy cohort (n = c.15) - ATG administration
followed by dosing of QEL-001 and complete weaning of
immunosuppression to assess the induction and durability of
operational tolerance at two and 12 months after full withdrawal of
immunosuppressive therapy (Endpoints 1 and 2,
respectively).
In both cohorts, immune monitoring assays will
be used to track QEL-001 CAR-Treg cells in whole blood and liver
tissue allowing safety and efficacy assessments and providing
translational information apply to the advancement of QEL-001 and
Quell's broader Treg cell therapy pipeline.
About Liver
Transplantation
Approximately 15,000 patients receive new liver
transplants in the US and EU5 each year and the standard of care
for liver transplant patients is to receive systemic
immunosuppression for the rest of their lives.
Current treatments, such as calcineurin
inhibitors, cause significant non-liver comorbidities including
increased rates of certain malignancies and infections, diabetes
and cardiovascular disease, as well as renal failure. These
complications result in poor post-transplant survival with the
10-year survival outcome of liver transplant patients being around
60-65% and comparable to that of common malignancies, such as colon
cancer, cancer of the uterus or Non-Hodgkin's lymphoma.
Further, long-term immunosuppressive therapy is
nephrotoxic, leading to progressive and irreparable kidney damage
that eventually requires dialysis and transplant.
About Quell
Therapeutics
Quell Therapeutics is the world leader in
developing engineered T-regulatory (Treg) cell therapies that aim
to harness, direct and optimize their immune suppressive properties
to address serious medical conditions driven by the immune
system.
The Company is leveraging its pioneering Foxp3
Phenotype Lock™ technology, unique multi-modular platform and
integrated manufacturing capabilities to design and develop a
pipeline of highly engineered Treg cell therapies with greater
potential for persistence, potency and stability than earlier
generations of Treg cell therapy approaches.
Quell's lead candidate QEL-001 is being
developed to induce operational tolerance following liver
transplantation, with the potential to protect the post-transplant
liver without the need for chronic immunosuppressive medications.
Quell is also advancing additional programs in neuroinflammatory
and autoimmune diseases internally and in partnership with
AstraZeneca. www.quell-tx.com.
Contacts
Luke Henry, Chief Business Officer
Quell Therapeutics
IR@quell-tx.com
Media: Mark Swallow, Frazer Hall, Sandi
Greenwood
MEDiSTRAVA
Quell-Tx@Medistrava.com
Investors: Corey Davis, PhD
LifeSci Advisors
cdavis@lifesciadvisors.com