Trem-cel + Mylotarg demonstrated engraftment,
shielding, broadened therapeutic window, and patient benefit
VCAR33ALLO demonstrates encouraging biomarker
data at lowest dose
New asset VADC45 with significant potential
opportunities across oncology, gene therapy, and autoimmune
disorders
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the
"Company"), a clinical-stage biotherapeutics company dedicated to
changing the lives of patients with devastating diseases, noted
that its Founded Entity, Vor Bio (Nasdaq: VOR), a clinical-stage
cell and genome engineering company, announced new clinical data
from its ongoing Phase 1/2 VBP101 study of patients with
relapsed/refractory AML receiving trem-cel followed by Mylotarg™.
The data demonstrated reliable engraftment, shielding from Mylotarg
on-target toxicity, a broadened Mylotarg therapeutic window, and
early evidence of patient benefit.
The full text of the announcement from Vor is as follows:
New Clinical Data Validates Vor Bio’s
Approach of Using Shielded Transplants to Deliver Targeted
Therapies
Trem-cel + Mylotarg demonstrated engraftment,
shielding, broadened therapeutic window, and patient benefit
VCAR33ALLO demonstrates encouraging biomarker
data at lowest dose
New asset VADC45 with significant potential
opportunities across oncology, gene therapy, and autoimmune
disorders
CAMBRIDGE, Mass., Sept. 05, 2024 -- Vor Bio (Nasdaq: VOR), a
clinical-stage cell and genome engineering company, today announced
new clinical data from its ongoing Phase 1/2 VBP101 study of
patients with relapsed/refractory AML receiving trem-cel followed
by Mylotarg™. The data demonstrated reliable engraftment, shielding
from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic
window, and early evidence of patient benefit.
“We are encouraged by this data and the potential benefit that
trem-cel in combination with Mylotarg may offer to patients in a
disease that has extremely poor outcomes even after transplant,”
said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “With this
data, we plan to explore a registrational trial while we continue
to pursue other synergistic opportunities for Vor Bio’s platform
such as VCAR33ALLO and VADC45.”
The data released today included 18 patients treated with
trem-cel of which ten had received Mylotarg as of the data cut-off
date of July 19, 2024. The data demonstrated:
- Reliable engraftment, with 100% of patients achieving primary
neutrophil engraftment (median 9 days) and robust platelet
recovery (median 16.5 days). High CD33 editing efficiency (median
89%, range 71-94%) and full myeloid chimerism at Day 28.
- Shielding of the blood system, with maintained neutrophil and
platelet counts across multiple Mylotarg doses of 0.5, 1, and 2
mg/m2.
- Broadened therapeutic index for Mylotarg with drug exposure
represented by AUC which is related to efficacy, consistent with
labeled Mylotarg doses, and with maximal concentrations, measured
by Cmax and related to veno-occlusive disease, well below known
toxic range.
- Early evidence suggesting patient benefit as measured by
relapse-free survival when compared to published high-risk AML
comparators1.
“All the hope I had in the safety of this approach has been
supported by the data from this trial thus far,” said Guenther
Koehne, MD, PhD, an investigator on the VBP101 study and Deputy
Director and Chief of Blood & Marrow Transplant and Hematologic
Oncology at Miami Cancer Institute of Baptist Health South Florida.
“I look forward to treating my next patients at high risk of
relapse on this trial as their outcomes are otherwise limited with
standard transplants.”
Vor Bio plans to approach the U.S. Food & Drug
Administration to discuss a pivotal trial design for trem-cel +
Mylotarg by around year end.
Continued progress with VCAR33ALLO
- VCAR33ALLO represents another potentially significant
synergistic treatment option after trem-cel.
- The VBP301 study continues enrolling patients with initial
focus on relapsed/refractory AML post-transplant.
- Vor Bio is encouraged by in vivo CAR-T expansion data from
three patients treated to date, all at the lowest dose of 1 x 106
CAR+ cells/kg.
Vor Bio announced today, a new preclinical asset, VADC45,
which has a number of potential opportunities in oncology, gene
therapy, and autoimmune disorders.
- VADC45 is an ADC that targets the CD45 protein. CD45 is a
well-validated target for a wide variety of blood cancers with
clinical proof of concept. The linker-payload used in VADC45 is
also clinically validated.
- VADC45 has the potential to treat a number of diseases,
including treatment of hematologic malignancies, as a targeted
conditioning agent for gene therapies such as for sickle cell
disease, holistic immune reset for autoimmune disorders, and for
Vor Bio’s approach of combining this asset with epitope
modification of CD45 to shield healthy stem cells.
- Vor Bio already has robust preclinical data for VADC45 and is
progressing IND-enabling studies to enable future Phase 1
studies.
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 potential of its product candidates to
positively impact quality of life and alter the course of disease
in the patients it seeks to treat, the timing of regulatory filings
and initiation of clinical trials, the timing and pace of patient
enrollment and dosing in clinical trials and the availability of
data therefrom, the expected safety profile of its product
candidates, its intentions to use VCAR33ALLO in combination with
trem-cel as a Treatment System, the potential of trem-cel to enable
targeted therapies in the post-transplant setting including
Mylotarg and CD33-targeted CAR-Ts, and the ability of VADC45 to
treat hematologic malignancies and to be used as a targeted
conditioning agent for gene therapies, as a holistic immune reset
for autoimmune disorders, and in combination with opitope
modification of CD45 to shield healthy stem cells. 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; uncertainties regarding 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 and Vor
Bio’s ability to continue as a going concern. 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 clinical-stage biotherapeutics company dedicated
to giving life to new classes of medicine to change the lives of
patients with devastating diseases. The Company has created a broad
and deep pipeline through its experienced research and development
team and its extensive network of scientists, clinicians and
industry leaders that is being advanced both internally and through
its Founded Entities. PureTech's R&D engine has resulted in the
development of 29 therapeutics and therapeutic candidates,
including two that have received both U.S. FDA clearance and
European marketing authorization and a third (KarXT) that has been
filed for FDA approval. A number of these programs are being
advanced by PureTech or its Founded Entities in various indications
and stages of clinical development, including registration enabling
studies. 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.
For more information, visit www.puretechhealth.com or connect
with us on X (formerly Twitter) @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be
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’s Bio’s statements
regarding the potential of its product candidates to positively
impact quality of life and alter the course of disease in the
patients it seeks to treat, the timing of regulatory filings and
initiation of clinical trials, the timing and pace of patient
enrollment and dosing in clinical trials and the availability of
data therefrom, the expected safety profile of its product
candidates, its intentions to use VCAR33ALLO in combination with
trem-cel as a Treatment System, the potential of trem-cel to enable
targeted therapies in the post-transplant setting including
Mylotarg and CD33-targeted CAR-Ts, and the ability of VADC45 to
treat hematologic malignancies and to be used as a targeted
conditioning agent for gene therapies, as a holistic immune reset
for autoimmune disorders, and in combination with opitope
modification of CD45 to shield healthy stem cells. 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, 2023,
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.
1 Araki et al. JCO 2016; Jentzsch et al. Blood Cancer Journal
2022.
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