RCS - PureTech Health PLC - PRTC's Vor Shares New AML Data Validating Approach

Puretech HealthAnnouncement

06/09/2024 07:00

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RNS Number : 1725D  PureTech Health PLC  06 September 2024

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

 

VCAR33(ALLO) demonstrates encouraging biomarker data at lowest dose

 

New asset VADC45 with significant potential opportunities across oncology,
gene therapy, and autoimmune disorders

 

PureTech Health plc (https://puretechhealth.com/) (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

 

VCAR33(ALLO) 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 VCAR33(ALLO) 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/m(2).

·      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 C(max) 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 comparators(1).

 

"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 VCAR33(ALLO)

·      VCAR33(ALLO) 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 10(6) 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
(https://www.globenewswire.com/Tracker?data=aXCcaMXVUhB6WEH78P3fay4Gr6aZE7Zs2tcHf0VwzIWK77IQUgOLQSOYxZ1fqWFy8nxATpEISEg-weTTr1KfLUAwSQD5C_dSlmJ6N1Gm8KNP7_3D0Mfhu2eZDe__0n96nA3yaJSzjqtzjmSsYsu7Ka8LTW-ed-wHkH3JKxZC8jKh4v1s9S51GL89wUolKbmzGVxAGuk_UzZT3pjGYtV4NGNdbAPsjxUz-9B-MrOcdiU=)
.

 

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 VCAR33(ALLO) 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
(http://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 VCAR33(ALLO) 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 (mailto:publicrelations@puretechhealth.com)

Investor Relations

IR@puretechhealth.com (mailto:IR@puretechhealth.com)

 

UK/EU Media

Ben Atwell, Rob Winder

+44 (0) 20 3727 1000

puretech@fticonsulting.com (mailto:puretech@fticonsulting.com)

 

US Media

Nichole Bobbyn

+1 774 278 8273

nichole@tenbridgecommunications.com
(mailto:nichole@tenbridgecommunications.com)

 

 

(1) Araki et al. JCO 2016; Jentzsch et al. Blood Cancer Journal 2022.

 

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