TIDMPRTC
PureTech Health PLC
14 January 2021
PureTech Health plc
PureTech Founded Entity Vor Announces FDA Clearance of IND
Application for VOR33
Phase 1/2a clinical trial expected to begin in first half of
2021
BOSTON, January 14, 2021 - PureTech Health plc (LSE: PRTC,
NASDAQ: PRTC) ("PureTech" or the "Company") is p leased to note
that its Founded Entity, Vor Biopharma , a clinical-stage cell
therapy company pioneering engineered hematopoietic stem cell
(eHSC) therapies combined with targeted therapies for the treatment
of cancer, today announced that the U.S. Food and Drug
Administration (FDA) has cleared the company's Investigational New
Drug (IND) application for VOR33, an eHSC therapy candidate being
developed for the treatment of acute myeloid leukemia (AML). Vor
plans to initiate a Phase 1/2a clinical trial for VOR33 in the
first half of this year.
VOR33, consisting of hematopoietic stem cells that are
engineered to lack the CD33 protein, is a cell therapy candidate
intended to replace the standard of care in hematopoietic stem cell
transplant settings for patients with AML who are at high-risk for
relapse.
The full text of the announcement from Vor Biopharma is as
follows:
Vor Announces FDA Clearance of IND Application for VOR33
Phase 1/2a clinical trial expected to begin in first half of
2021
CAMBRIDGE, MA January 14, 2021 - Vor Biopharma , a
clinical-stage cell therapy company pioneering engineered
hematopoietic stem cell (eHSC) therapies combined with targeted
therapies for the treatment of cancer, today announced that the
U.S. Food and Drug Administration (FDA) has cleared the company's
Investigational New Drug (IND) application for VOR33, an eHSC
therapy candidate being developed for the treatment of acute
myeloid leukemia (AML). The company plans to initiate a Phase 1/2a
clinical trial for VOR33 in the first half of this year.
VOR33, consisting of hematopoietic stem cells that are
engineered to lack the CD33 protein, is a cell therapy candidate
intended to replace the standard of care in hematopoietic stem cell
transplant settings for patients with AML who are at high-risk for
relapse.
"Though advances have been made in the treatment of AML and
other myeloid malignancies, the median overall five-year survival
rate for patients diagnosed with AML remains under 30 percent,"
said Christopher Slapak, MD, Vor's Chief Medical Officer. "With the
development of VOR33, we are seeking to change the treatment
paradigm for AML and potentially other hematologic malignancies. We
engineered VOR33 to provide patients with a hematopoietic stem cell
transplant that we believe, upon hematopoietic reconstitution, will
be treatment resistant to CD33 targeted therapies, potentially
resulting in new treatment options and improved post-transplant
outcomes."
"Clearance of this IND is the culmination of an incredible team
effort at Vor and represents a key milestone for us," added Robert
Ang, MBBS, MBA, Vor's President and Chief Executive Officer. "This
brings us an important step closer to treating patients with our
potentially transformative therapy."
The Phase 1/2a trial is expected to enroll patients with
CD33-positive AML who are at high risk of relapse. The primary
goals of the trial are to evaluate tolerability and feasibility of
the VOR33 stem cell transplant, with a focus on confirming that
VOR33 can engraft normally. Following engraftment, patients will be
eligible to be treated with Mylotarg (Ò) , an FDA approved
CD33-directed antibody drug conjugate (ADC) therapy owned by
Pfizer, in order to potentially prolong leukemia-free survival and
provide evidence that VOR33 protects against the myelosuppression
that typically accompanies treatment with Mylotarg (Ò) .
About VOR33
VOR33 is Vor's lead product candidate, consisting of eHSCs that
we have engineered to lack the protein CD33, and is designed to
replace the standard of care in transplant settings for patients
suffering from AML and potentially other hematologic malignancies.
Once the VOR33 cells have engrafted, we believe that patients can
be treated with anti-CD33 therapies, such as Mylotarg (Ò) or, if
approved by the FDA, Vor's in-licensed CD33 chimeric antigen
receptor T-cell (CAR-T) therapy candidate, with limited on-target
toxicity, leading to durable anti-tumor activity and potential
cures. In preclinical studies, we have observed that the removal of
CD33 provided robust protection of VOR33 eHSCs from the cytotoxic
effects of CD33-directed therapies, yet had no deleterious effects
on the differentiation or function of hematopoietic cells.
About Vor Biopharma
Vor Biopharma is a clinical-stage cell therapy company that aims
to transform the lives of cancer patients by pioneering eHSC
therapies to create next-generation, treatment-resistant
transplants that unlock the potential of targeted therapies. By
removing biologically redundant proteins from eHSCs, we design
these cells and their progeny to be treatment-resistant to
complementary targeted therapies, thereby enabling these therapies
to selectively destroy cancerous cells while sparing healthy
cells.
Our platform could be used to potentially change the treatment
paradigm of both hematopoietic stem cell transplants and targeted
therapies, such as ADCs, bispecific antibodies and CAR-T cell
treatments, including Vor's in-licensed CD33 CAR-T.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company dedicated
to discovering, developing and commercializing highly
differentiated medicines for devastating diseases, including
intractable cancers, lymphatic and gastrointestinal diseases,
central nervous system disorders and inflammatory and immunological
diseases, among others. The Company has created a broad and deep
pipeline through the expertise of its experienced research and
development team and its extensive network of scientists,
clinicians and industry leaders. This pipeline, which is being
advanced both internally and through PureTech's Founded Entities,
as of the date of PureTech's most recently filed Registration
Statement on Form 20-F, was comprised of 24 products and product
candidates, including two that have received FDA clearance and
European marketing authorization. All of the underlying programs
and platforms that resulted in this pipeline of product candidates
were initially identified or discovered and then advanced by the
PureTech team through key validation points based on the Company's
unique insights into the biology of the brain, immune and gut, or
BIG, systems and the interface between those systems, referred to
as the BIG Axis.
For more information, visit www.puretechhealth.com or connect
with us on Twitter @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be
forward-looking statements, including statements that relate to our
product candidates and approach towards addressing major diseases,
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, expectations
regarding the initiation of a Phase 1/2a clinical trial for VOR33
in the first half of this year, the potential therapeutic benefits
of VOR33 and 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 U.S. media
Allison Mead Talbot Ben Atwell, Rob Winder Stephanie Simon
+1 617 651 3156 +44 (0) 20 3727 1000 +1 617 581 9333
amt@puretechhealth.com ben.atwell@FTIconsulting. stephanie@tenbridgecommunications.
com com
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