TIDMPRTC
PureTech Health PLC
07 December 2022
7 December 2022
PureTech Health plc
PureTech Founded Entity Vor Bio Announces First AML Patient
Successfully Transplanted with its Investigational Trem-cel (VOR33)
and Tolerated Mylotarg(TM)
Trem-cel (formerly VOR33) successfully manufactured and
engrafted normally
Blood counts successfully maintained following post-transplant
treatment with Mylotarg
Vor Bio also announced the pricing of an underwritten offering
and a private placement, with combined gross proceeds of
approximately $115.8 million
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the
"Company"), a clinical-stage biotherapeutics company, noted that
its Founded Entity, Vor Bio (Nasdaq: VOR), a clinical-stage cell
and genome engineering company, today announced initial clinical
data from VBP101, its Phase 1/2a multicenter, open-label,
first-in-human study of tremtelectogene empogeditemcel or
"trem-cel" (formerly VOR33) in patients with acute myeloid leukemia
(AML). The data observed from the first treated patient support the
potential of a trem-cel transplant to be successfully manufactured,
to engraft normally, and to maintain blood counts following
treatment with the CD33-targeted therapy Mylotarg. The clinical
trial continues to enroll patients and additional data are expected
in 2023.
Vor also separately announced today the pricing of an
underwritten offering and a private placement, with combined gross
proceeds of approximately $115.8 million. PureTech's percentage
ownership of Vor Bio as at November 4, 2022 was approximately 8.2
percent on a beneficial ownership basis, prior to the
transaction.
The full text of the announcement from Vor is below:
First AML Patient Successfully Transplanted with Vor Bio's
Investigational Trem-cel (VOR33) and Tolerated Mylotarg(TM)
-- Trem-cel (formerly VOR33) successfully manufactured and engrafted normally
-- Blood counts successfully maintained following post-transplant treatment with Mylotarg
-- Conference call scheduled for today, December 7 at 8:00am ET
CAMBRIDGE, Mass., Dec. 07, 2022 -- Vor Bio (Nasdaq: VOR), a
clinical-stage cell and genome engineering company, today announced
initial clinical data from VBP101, its Phase 1/2a multicenter,
open-label, first-in-human study of tremtelectogene empogeditemcel
or "trem-cel" (formerly VOR33) in patients with acute myeloid
leukemia (AML). The data observed from the first treated patient
support the potential of a trem-cel transplant to be successfully
manufactured, to engraft normally, and to maintain blood counts
following treatment with the CD33-targeted therapy Mylotarg. The
clinical trial continues to enroll patients and additional data are
expected in 2023.
"These early engraftment data represent the first time genome
engineering has been used to genetically alter donor cells by
removing an antigen present on blood cells, thereby allowing
treatment using a CD33 targeted therapy while protecting normal
blood cells," said Dr. Robert Ang, Vor Bio's President and Chief
Executive Officer. "These encouraging data represent the first
clinical validation of our platform to potentially enable
next-generation transplants for patients with blood cancers. We
look forward to sharing additional data updates in 2023."
Trem-cel Displayed Normal Engraftment
A product dose of 7.6 x10(6) CD34(+) viable cells/kg, with a
CD33 editing efficiency of 88% was manufactured. Following
myeloablative conditioning, trem-cel was infused with no infusion
reactions. The patient achieved neutrophil engraftment 10 days
post-transplant which was within expectations for CD34-enriched
transplants. Platelet recovery was observed on Day 22.
Hematopoietic cell sub-population reconstitution was robust with
over 90% of peripheral blood cells negative for CD33 expression,
and 100% donor chimerism was achieved. These data provide
proof-of-concept that trem-cel can engraft as expected and that
CD33 does not appear to be biologically necessary for engraftment
and hematopoietic reconstitution.
Mylotarg Tolerated at Initial Dose Level
The patient received Mylotarg at a dose of 0.5 mg/m(2) . At this
dose, Mylotarg saturates CD33 antigen in patients with
relapsed/refractory AML(1) , and in the original Phase 1 trial of
Mylotarg(2) , neutropenia was observed across dose levels starting
at 0.25mg/m(2) within 14 days of infusion. No treatment related
adverse events and no liver enzyme changes were observed through
day 20 following Mylotarg dosing. No negative impacts to neutrophil
and platelet counts were observed through day 20, suggesting
tolerability at this initial dose level.
"The unmet medical need for AML is significant and hematopoietic
cell transplant is the best hope for these patients," said Brenda
Cooper, M.D., Professor of Medicine in the Cellar Therapy Program
at University Hospitals, Seidman Cancer Center, and an investigator
in the VBP101 study. "Early treatment data in the first patient
show that trem-cel can engraft normally and maintain normal
hematopoiesis following Mylotarg dosing, which typically causes
severe cytopenias. These data support the promise of this
approach."
___________
(1) Mylotarg ODAC 2017
(2) Sievers 1999 Blood 93:3678
Conference Call & Webcast Information
Members of the Vor Bio management team, joined by Dr. Brenda
Cooper, will conduct a live conference call and webcast today at
8:00 am Eastern Time.
Listeners can register for the webcast via this link .
Analysts wishing to participate in the Q&A session should
use this link .
A replay of the webcast will be available via the investor
section of the Company's website at www.vorbio.com approximately
two hours after the call's conclusion.
About AML
AML is the most common type of acute leukemia in adults and one
of the deadliest and most aggressive blood cancers, affecting
20,000 newly diagnosed patients each year in the United States.
Approximately half of patients with AML who receive a hematopoietic
cell transplant (HCT) suffer a relapse of their leukemia, with
two-year survival rates of less than 20%, and relapse rates are
higher for patients with certain adverse risk features. The
fragility of engrafted hematopoietic stem cells prevents treatment
following transplant, giving the cancer a chance to return.
About the VBP101 Clinical Trial
VBP101 is a Phase 1/2a, multicenter, open-label, first-in-human
study of trem-cel in participants with AML who are undergoing human
leukocyte antigen (HLA)-matched allogeneic hematopoietic cell
transplant (HCT). Trem-cel is an allogeneic CRISPR/Cas9
genome-edited hematopoietic stem and progenitor cell (HSPC) therapy
product, lacking the CD33 protein. It is being investigated for
participants with CD33(+) AML at high risk for relapse after HCT to
allow post-HCT targeting of residual CD33(+) acute AML cells using
Mylotarg without toxicity to engrafted cells. Participants undergo
a myeloablative HCT with matched related or unrelated donor
CD34-selected HSPCs engineered to remove CD33 expression (trem-cel
drug product). Mylotarg is given after engraftment for up to four
cycles. The primary endpoint is the incidence of successful
engraftment, defined as the first day of 3 consecutive days of
absolute neutrophil count (ANC) 500 cells/mm(2) by day 28. Part 1
of this study is evaluating the safety of escalating Mylotarg dose
levels to determine the maximum tolerated dose (MTD) and
recommended Phase 2 dose. Part 2 will expand the number of
participants to evaluate the Mylotarg recommended Phase 2 dose. For
more information, visit:
https://clinicaltrials.gov/ct2/show/NCT04849910
About Trem-cel
Tremtelectogene empogeditemcel (trem-cel), formerly VOR33, is a
genome-edited hematopoietic stem and progenitor allogeneic donor
product candidate where CD33 has been deleted using genome
engineering. Transplant with trem-cel is designed to replace
standard of care transplants for patients suffering from AML and
potentially other blood cancers. Trem-cel has the potential to
enable powerful targeted therapies in the post-transplant setting
including CD33-targeted CAR-T cells.
About Vor Bio
Vor Bio is a clinical-stage cell and genome engineering company
that aims to change the standard of care for patients with blood
cancers by engineering hematopoietic stem cells to enable targeted
therapies post-transplant. For more information, visit:
www.vorbio.com .
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. The words "aim," "anticipate," "can," "continue," "could,"
"design," "enable," "expect," "initiate," "intend," "may,"
"on-track," "ongoing," "plan," "potential," "should," "target,"
"update," "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 Vor Bio's
statements regarding the feasibility of a trem-cel transplant to be
successfully manufactured, to engraft normally, to maintain blood
counts following treatment with Mylotarg following allogeneic
hematopoietic cell transplant and to be well tolerated, the
potential of Vor Bio's platform, and timing expectations for
additional release of clinical data. Vor Bio 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 Vor Bio'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 success of
Vor Bio's in-house manufacturing capabilities and efforts; and
availability of funding sufficient for its foreseeable and
unforeseeable operating expenses and capital expenditure
requirements. The interim data presented in this press release is
based on one patient and future results for this patient or
additional patients may not produce the same or consistent results.
These and other risks are described in greater detail under the
caption "Risk Factors" included in Vor Bio's most recent annual or
quarterly report and in other reports it 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 Vor Bio 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 biotherapeutics company dedicated to changing the
treatment paradigm for devastating diseases. 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 28 therapeutics and therapeutic
candidates, including two (Plenity(R) and EndeavorRx(R)) that have
received both U.S. FDA clearance and European marketing
authorization and a third (KarXT) that will soon be filed for FDA
approval, as of the most recent update by the Company. 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 unique insights in immunology and drug development.
PureTech Ownership
PureTech's percentage ownership of Vor Bio as at November 4,
2022 was approximately 8.2 percent on a beneficial ownership basis,
prior to the transaction. PureTech's ownership of Vor will be
updated in due course in certain of its public materials, including
its publicly-available investor deck, following completion of the
transaction.
For more information, visit www.puretechhealth.com or connect
with us on Twitter @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including without limitation those
related to Vor Bio's statements regarding the feasibility of a
trem-cel transplant to be successfully manufactured, to engraft
normally, to maintain blood counts following treatment with
Mylotarg following allogeneic hematopoietic cell transplant and to
be well tolerated, the potential of Vor Bio's platform, and timing
expectations for additional release of clinical data, and Vor's
future prospects, development plans, and strategies. The
forward-looking statements are based on current expectations and
are subject to known and unknown risks, uncertainties and other
important factors that could cause actual results, performance and
achievements to differ materially from current expectations,
including, but not limited to, those risks, uncertainties and other
important factors described under the caption "Risk Factors" in our
Annual Report on Form 20-F for the year ended December 31, 2021
filed with the SEC and in our other regulatory filings. 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, we
disclaim any obligation to update or revise these forward-looking
statements, whether as a result of new information, future events
or otherwise.
Contact:
PureTech
Public Relations
publicrelations@puretechhealth.com
Investor Relations
IR@puretechhealth.com
EU Media
Ben Atwell, Rob Winder
+44 (0) 20 3727 1000
ben.atwell@FTIconsulting.com
U.S. Media
Nichole Sarkis
+1 774 278 8273
nichole@tenbridgecommunications.com
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