TIDMPRTC
PureTech Health PLC
29 May 2019
29 May 2019
PureTech Health plc
PureTech Health Affiliate Vor Biopharma Publishes
Proof-of-Concept Study of its Lead Programme in the Scientific
Journal Proceedings of the National Academy of Sciences
Preclinical study supports Vor's approach for overcoming
important toxicities encountered by targeted immunotherapies for
blood cancers
Paper co-authored by Vor co-founder Dr Siddhartha Mukherjee
PureTech Health plc (LSE:PRTC) ("PureTech Health"), an advanced
biopharmaceutical company developing novel medicines for
dysfunctions of the Brain-Immune-Gut (BIG) axis, is pleased to note
that its affiliate Vor Biopharma ("Vor") today announced the
publication of an important paper in the scientific journal
Proceedings of the National Academy of Medicines supporting Vor's
novel approach to treating cancer via engineered hematopoietic stem
cells (HSCs).
Vor's approach is designed to address a serious limitation of
most targeted immunotherapies: the targets they bind are present on
both healthy cells and cancer cells. In the case of haematological
cancers, this poor discrimination can result in devastating
collateral damage to healthy immune cells and severe toxicities.
Vor's lead programme, VOR33, is a novel HSC therapy consisting of
donor-derived HSCs that are engineered to lack the cell surface
protein CD33. When removed from the cell surface, VOR33 cells do
not appear to have any measurable changes to their biological
function but are shown to be highly resistant to attack from
CD33-targeted immunotherapies. When infused into a patient, the
engineered stem cells are designed to mature and differentiate into
a full spectrum of healthy immune and blood cells that would be
untouched by the cancer treatment. As the preclinical research
published today suggests, this approach has the potential to
minimize immunotherapy toxicities and maximize the potency of
anti-CD33 therapies.
Bharatt Chowrira, JD, PhD, president and chief of business and
strategy at PureTech Health, said: "Vor's novel approach is
designed to reboot the patient's immune system with an infusion of
healthy blood stem cells that are protected from the toxic effects
of targeted immunotherapies. This approach has the potential to
minimize the toxicities associated with immunotherapies and greatly
expand the reach of this class of medicines, well beyond what is
currently possible. We are excited and encouraged by these results
and look forward to Vor's progress in advancing its lead candidate
VOR33 and other therapies built around engineered HSCs."
The full text announcement from Vor Biopharma is as follows:
Vor Biopharma Announces Publication of Proof-of-Concept Study of
its Lead Programme in the Scientific Journal Proceedings of the
National Academy of Sciences
Preclinical study supports Vor's approach for empowering
targeted immunotherapies through hematopoietic stem cell
editing
Paper co-authored by Vor co-founder Dr Siddhartha Mukherjee
BOSTON, May 29, 2019 - Vor Biopharma, an immuno-oncology company
pioneering engineered hematopoietic stem cell (HSC) therapies for
hematologic malignancies, today announced a publication in the
scientific journal Proceedings of the National Academy of Sciences
(PNAS) titled "Gene-Edited Stem Cells Enable CD33-Directed Immune
Therapy for Myeloid Malignancies." The research describes the
successful editing of HSCs that are engineered to be selectively
deficient in the myeloid lineage antigen CD33. These CD33-deficient
HSCs were shown to successfully engraft into the bone marrow of
their host. Furthermore, there was evidence these cells could
functionally repopulate the hematopoietic system. When subjected to
aggressive doses and regimens of CD33-targeted immuonotherapies in
a murine model of acute myeloid leukaemia (AML), Vor-engineered
HSCs were protected from depletion by the immunotherapy while
cancer cells were selectively targeted.
These newly published results are the basis for Vor's lead
product candidate, VOR33, and outline Vor's unique therapeutic
approach. The paper was co-authored by Vor co-founder Siddhartha
Mukherjee, MD, DPhil, of Columbia University Irving Medical Center,
and his team.
Vor's approach is designed to enable broadly targeting lineage
antigens, which are attractive targets but face serious
limitations, since they are expressed on both healthy cells and
cancerous cells. Currently, targeting lineage antigens for treating
hematologic malignancies depletes the healthy blood cells of the
targeted lineage, causing severe toxicities. These toxicities have
prevented the successful development or broader use of several
otherwise promising drugs. In patients, Vor-engineered HSCs are
expected to mature and differentiate into healthy blood cells that
do not display a particular lineage antigen, therefore making that
lineage antigen tumour-specific and safe to target.
The preclinical research published today suggests that VOR33
cells will produce healthy blood cells that will not display the
lineage antigen CD33, and therefore will not be depleted by
CD33-targeted therapies. The publication further indicates that
this approach minimizes on-target toxicities and maximizes the
potency of lineage-targeted immunotherapies, including
antibody-drug conjugates, bispecific antibodies, and chimeric
antigen receptor (CAR) T cells.
"We are hoping to protect the patient's immune system from
damage during targeted immunotherapy treatment. This approach has
the potential to transform the use of immunotherapies in
hematologic diseases by enabling the use of highly potent drugs,
improving dosing, and fundamentally changing the treatment
paradigm. The novelty of the strategy is that rather than focus
only on the cancer, we are making the host - the patient -
resistant to the therapy, so that the cancer remains vulnerable.
This strategy has never been attempted in cancer," said Dr
Mukherjee. "This preclinical study demonstrates that Vor's
technology can clear leukaemia and repopulate healthy hematopoietic
cell populations through the use of engineered HSCs combined with a
CAR-T or an antibody-drug conjugate (ADC) or a range of other
targeted immunotherapy approaches. Remarkably, we saw that it was
also possible to use a CAR-T and an ADC in combination without
untoward myelosuppression. This research gives us great hope as we
advance toward the clinic with VOR33 and look to expand our novel
approach into other cancers."
In February 2019, Vor announced the completion of a $42 million
Series A financing round led by 5AM Ventures and RA Capital to
advance its lead candidate for the treatment of AML towards the
clinic, and to further build its pipeline to treat hematologic
malignancies. In November 2018, Vor announced that the United
States Patent and Trademark Office issued U.S. Patent No.
10,137,155, which covers compositions and methods related to using
modified HSCs to enable targeted immunotherapies. This patent, the
first of its kind and foundational for the field, established Vor
as the pioneer in developing modified HSC therapies to enable
targeted immunotherapy. Subsequent to this patent's initial filing,
additional laboratories successfully replicated key aspects
underlying the technology.
About VOR33
Vor's lead product candidate, VOR33, is designed to produce
healthy blood cells that lack CD33, potentially protecting them
from the toxic effects of CD33-targeted immunotherapies. CD33 is a
myeloid lineage antigen that is present in the majority of acute
myeloid leukaemias (AML) but is also expressed abundantly on normal
myeloid cells. Depletion of normal myeloid progenitor cells during
treatment for AML limits the dosage and duration of CD33-targeted
therapies. By empowering CD33-targeted therapy, VOR33 has the
potential to overcome current limitations.
About Vor Biopharma
Vor Biopharma is taking a fundamentally novel approach to treat
cancer by developing engineered hematopoietic stem cells (HSCs).
Vor's engineered HSCs are designed to generate healthy, functional
blood cells that lack the binding site of lineage antigen-targeted
therapies. Removal of the binding site protects these HSCs from
depletion by lineage antigen-targeted immunotherapies.
Vor's platform is broad and could potentially be used to vastly
improve the therapeutic window of several lineage antigen-targeted
therapies, such as antibody drug conjugates (ADCs), bispecific
antibodies, and chimeric antigen receptor T cells (CAR-Ts). This
technology platform is covered by broad intellectual property
exclusively licensed from Columbia University, as well as Vor-owned
intellectual property. Co-founded by PureTech Health (LSE: PRTC)
and Siddhartha Mukherjee, MD, DPhil, Vor is working with some of
the world's leading oncologists and immunologists to develop a
pipeline of potentially life-altering immunotherapies that extend
beyond what is possible with current treatment.
About PureTech Health
PureTech Health (LSE: PRTC) is an advanced biopharmaceutical
company developing BIG medicines for dysfunctions of the
Brain-Immune-Gut axis. The Company has gained deep insights into
the connection between these systems and the resulting role in
diseases that have proven resistant to established therapeutic
approaches. By harnessing this emerging field of human biology,
PureTech Health is developing new categories of medicines with the
potential to have great impact on people with serious diseases.
PureTech Health is advancing a rich pipeline of innovative
therapies with an unbiased, non-binary, and capital efficient
R&D model across its affiliates and its internal labs.
PureTech's affiliates include seven clinical-stage platforms,
including one product that has been cleared by the US Food and Drug
Administration (FDA) and a second product candidate that has been
filed with the FDA for review, and several other novel preclinical
programmes. The PureTech Health pipeline includes ground-breaking
platforms and therapeutic candidates that were developed in
collaboration with some of the world's leading experts.
PureTech's internal research and development is centred on
tissue-selective immunomodulation for the treatment of oncology,
autoimmune, and CNS-related disorders, with a near-term focus on
targeting newly-discovered, foundational immunosuppressive
mechanisms in oncology and novel approaches that harness the
lymphatic infrastructure.
For more information, visit www.puretechhealth.com or connect
with us on Twitter @puretechh.
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company's future prospects, developments and strategies. The
forward-looking statements are based on current expectations and
are subject to known and unknown risks and uncertainties that could
cause actual results, performance and achievements to differ
materially from current expectations, including, but not limited
to, those risks and uncertainties described in the risk factors
included in the regulatory filings for PureTech Health plc. These
forward-looking statements are based on assumptions regarding the
present and future business strategies of the company and the
environment in which it will operate in the future. Each
forward-looking statement speaks only as at the date of this press
release. Except as required by law and regulatory requirements,
neither the company nor any other party intends to update or revise
these forward-looking statements, whether as a result of new
information, future events or otherwise.
Contact:
Investors EU media US media
Allison Mead Talbot Ben Atwell, Rob Winder Tom Donovan
+1 617 651 3156 +44 (0) 20 3727 1000 +1 857 559 3397
amt@puretechhealth.com ben.atwell@FTIconsulting.com tom@tenbridgecommunications.com
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END
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