TIDMPRTC
PureTech Health PLC
10 September 2021
10 September 2021
PureTech Health plc
PureTech Founded Entity Vor Biopharma Announces VOR33 Granted
U.S. FDA Fast Track Designation for Acute Myeloid Leukemia
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the
"Company"), a clinical-stage biotherapeutics company, is pleased to
note that its Founded Entity, Vor Biopharma (Nasdaq: VOR) ("Vor")
announced that the U.S. Food and Drug Administration ("FDA") has
granted Fast Track designation to VOR33, Vor's lead engineered
hematopoietic stem cell ("eHSC") therapeutic candidate for the
treatment of acute myeloid leukemia ("AML").
VOR33, the lead product candidate from Vor's novel scientific
platform, consists of CRISPR genome-edited hematopoietic stem and
progenitor cells that have been engineered to lack CD33. Once
infused, VOR33 is designed to protect patients' healthy cells from
anti-CD33 therapies, such as VCAR33 or Mylotarg(TM) (gemtuzumab
ozogamicin). VOR33 is intended to replace standard of care
transplants for AML patients who are at high risk of relapse and
has the potential to seamlessly integrate into current transplant
settings.
Vor is actively enrolling in its Phase 1/2a clinical trial for
AML patients who currently have limited treatment options. Vor
remains on track to report VOR33's initial clinical data in the
first half of 2022. Vor is also currently exploring the use of its
genome engineered hematopoietic stem cell platform in combination
with multiple therapeutic modalities.
The full text of the announcement from Vor is as follows:
VOR33 Granted U.S. FDA Fast Track Designation for AML
CAMBRIDGE, Mass., Sept. 09, 2021 - Vor Biopharma (Nasdaq: VOR),
a clinical-stage cell and genome engineering company, today
announced that the U.S. Food and Drug Administration (FDA) has
granted Fast Track designation to VOR33, the Company's lead
engineered hematopoietic stem cell (eHSC) therapeutic candidate for
the treatment of acute myeloid leukemia (AML).
VOR33 consists of CRISPR genome-edited hematopoietic stem and
progenitor cells that have been engineered to lack CD33. Once
infused, VOR33 is designed to protect patients' healthy cells from
anti-CD33 therapies, such as VCAR33 or Mylotarg(TM) (gemtuzumab
ozogamicin). VOR33 is intended to replace standard of care
transplants for AML patients who are at high risk of relapse and
has the potential to seamlessly integrate into current transplant
settings.
"Receiving Fast Track designation is an important milestone for
Vor, which signals the FDA's recognition of the serious and
life-threatening medical condition of patients facing acute myeloid
leukemia and the potential of VOR33 to address this unmet medical
need," said Robert Pietrusko, PharmD, Vor's Chief Regulatory and
Quality Officer. "We will continue to work closely with the FDA to
expedite the development of VOR33, which is now actively enrolling
in its Phase 1/2a clinical trial for AML patients who currently
have limited treatment options. We continue to remain on-track to
report VOR33's initial clinical data in the first half of
2022."
VOR33 is the lead product candidate of Vor's novel scientific
platform, which has the mission to create next-generation,
treatment-resistant transplants that unlock the potential of
targeted cancer therapies by leveraging advances in cell therapy
and gene editing. Vor is currently exploring the use of its genome
engineered hematopoietic stem cell platform in combination with
multiple therapeutic modalities.
Fast Track designation is intended to facilitate development and
expedite review of products designed to treat serious and
life-threatening conditions with unmet medical needs. The
designation is granted upon the FDA's review of data that
demonstrate this potential, along with a product development
program that is adequately designed to address the unmet medical
need. Therapeutic candidates receiving Fast Track designation may
be eligible for priority review and accelerated approval if certain
conditions are met.
About Vor Biopharma
Vor Biopharma is a clinical-stage cell and genome engineering
company that aims to transform the lives of cancer patients by
pioneering an engineered hematopoietic stem cell (eHSC) therapeutic
platform that unlocks 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 cancer cells while sparing healthy
cells.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. The words "believe," "continue," "could," "estimate,"
"expect," "intend," "may," "plan," "potential," "project,"
"should," "target," "will," "would," and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Forward-looking statements in this press release include the
Company's statements regarding the potential efficacy of VOR33,
potential benefits VOR33 may receive in connection with its Fast
Track designation and reporting initial clinical data from the
VOR33 Phase 1/2a clinical trial in the first half of 2022. The
Company may not actually achieve the plans, intentions, or
expectations disclosed in these forward-looking statements, and you
should not place undue reliance on these forward-looking
statements. Actual results or events could differ materially from
the plans, intentions and expectations disclosed in these
forward-looking statements as a result of various factors,
including: uncertainties inherent in the initiation and completion
of preclinical studies and clinical trials and clinical development
of the Company's product candidates; availability and timing of
results from preclinical studies and clinical trials; whether
interim results from a clinical trial will be predictive of the
final results of the trial or the results of future trials;
expectations for regulatory approvals to conduct trials or to
market products; the impact of the COVID-19 pandemic on the
Company's business, including its preclinical studies and clinical
trials and availability of funding sufficient for the Company's
foreseeable and unforeseeable operating expenses and capital
expenditure requirements. These and other risks are described in
greater detail under the caption "Risk Factors" included in the
Company's most recent annual or quarterly report and in other
reports the Company has filed or may file with the Securities and
Exchange Commission. Any forward-looking statements contained in
this press release speak only as of the date hereof, and the
Company expressly disclaims any obligation to update any
forward-looking statements, whether because of new information,
future events or otherwise, except as may be required by law.
About PureTech Health
PureTech is a clinical-stage biotherapeutics company dedicated
to discovering, developing and commercializing highly
differentiated medicines for devastating diseases, including
inflammatory, fibrotic and immunological conditions, intractable
cancers, lymphatic and gastrointestinal diseases and neurological
and neuropsychological disorders, 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, is comprised of 25 therapeutics and therapeutic
candidates, including two that have received both U.S. FDA
clearance and European marketing authorization, as of the date of
PureTech's most recently filed Half Year Report and corresponding
Form 6-K. All of the underlying programs and platforms that
resulted in this pipeline of therapeutic 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
expectations regarding the potential efficacy of VOR33, potential
benefits VOR33 may receive in connection with its Fast Track
designation and the anticipated timing of the initial clinical data
from the VOR33 Phase 1/2a clinical trial in the first half of 2022,
and Vor'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
Allison Mead Talbot Ben Atwell, Rob Winder
+1 617 651 3156 +44 (0) 20 3727 1000
amt@puretechhealth.com ben.atwell@FTIconsulting.com
This information is provided by Reach, the non-regulatory press
release distribution service of RNS, part of the London Stock
Exchange. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
Reach is a non-regulatory news service. By using this service an
issuer is confirming that the information contained within this
announcement is of a non-regulatory nature. Reach announcements are
identified with an orange label and the word "Reach" in the source
column of the News Explorer pages of London Stock Exchange's
website so that they are distinguished from the RNS UK regulatory
service. Other vendors subscribing for Reach press releases may use
a different method to distinguish Reach announcements from UK
regulatory news.
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.
END
NRAFFFFLAAIAIIL
(END) Dow Jones Newswires
September 10, 2021 02:00 ET (06:00 GMT)
Puretech Health (LSE:PRTC)
Historical Stock Chart
From Jun 2024 to Jul 2024
Puretech Health (LSE:PRTC)
Historical Stock Chart
From Jul 2023 to Jul 2024