6
September 2024
PureTech Health plc
PureTech Founded Entity Vor
Bio Announces New Clinical Data Validating 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
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.
Contact:
PureTech
Public Relations
publicrelations@puretechhealth.com
Investor Relations
IR@puretechhealth.com
UK/EU Media
Ben Atwell, Rob Winder
+44 (0) 20 3727 1000
puretech@fticonsulting.com
US
Media
Nichole Bobbyn
+1 774 278 8273
nichole@tenbridgecommunications.com
1 Araki et al. JCO 2016; Jentzsch et al. Blood Cancer
Journal 2022.