13
March 2024
PureTech Health plc
PureTech Receives Orphan Drug Designation for LYT-200 in Acute
Myeloid Leukemia
LYT-200 is being advanced in hematological malignancies such
as acute myeloid leukemia (AML) and high-risk myelodysplastic
syndrome (MDS), and locally advanced/metastatic solid tumors,
including head and neck cancers
Phase
1b clinical trial evaluating LYT-200 in relapsed/refractory AML and
MDS patients is ongoing
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, today announced that the
U.S. Food and Drug Administration (FDA) has granted orphan drug
designation to LYT-200 for the treatment of AML.
"The current long-term survival
rates of patients with relapsed or refractory AML are very poor,
and there remains a tremendous unmet need for more effective
therapies," said Amir Fathi, MD, Director of the Leukemia Program
at Massachusetts General Hospital and lead investigator of the
trial.
LYT-200 is a fully human IgG4
monoclonal antibody (mAb) targeting galectin-9, a potent oncogenic
driver in leukemia cells and an immunosuppressive protein. LYT-200
has demonstrated direct cytotoxic, anti-leukemic effects through
multiple mechanisms and is being developed as a potential novel
treatment for hematological malignancies, such as
relapsed/refractory AML and high-risk MDS, as well as locally
advanced/metastatic solid tumors that have poor survival rates,
including head and neck cancers. LYT-200 is currently being
evaluated in an ongoing Phase 1b clinical trial in relapsed/refractory AML and MDS, both as a single agent and
in combination with standard-of-care venetoclax and hypomethylating
agents (HMA). Initial findings from this trial
were announced in 2023, and
additional data are expected to be presented in a scientific forum
in 2024.
"Orphan drug designation from the
FDA validates our belief that targeting galectin-9 with LYT-200 is
a novel, promising approach that may offer patients a better
tolerated, more effective treatment," notes Aleksandra Filipovic,
MD, PhD, Head of Oncology at PureTech.
The FDA grants orphan drug
designation to novel drug and biologic products for the treatment,
diagnosis, or prevention of conditions affecting fewer than 200,000
persons in the U.S. Orphan drug designation qualifies PureTech for
incentives under the Orphan Drug Act, including tax credits for
some clinical trials and eligibility for seven years of market
exclusivity in the U.S., if the drug is approved for
AML.
About LYT-200
LYT-200 is a fully human IgG4
monoclonal antibody targeting galectin-9 for the potential
treatment of hematological malignancies, such as acute
myeloid leukemia (AML) and high-risk myelodysplastic syndrome
(MDS), as well as locally advanced/metastatic solid tumors
that have poor survival rates, including head and neck cancers.
Galectin-9 is a potent oncogenic driver and immunosuppressor, and
in AML it has been described to work via cytotoxic CD8 T cells and
natural killer cells. A wide variety of preclinical data
underscores the importance of galectin-9 as a target and suggests a
potential opportunity for biomarker development. These data
demonstrate high expression of galectin-9 across various blood
cancers and solid tumor types and show that galectin-9 levels
correlate with poor survival in several cancers.
LYT-200 has demonstrated direct
cytotoxic, anti-leukemic effects through multiple mechanisms, as
well as synergy with standard of care chemotherapy and venetoclax
in preclinical models. In the ongoing,
Phase 1b clinical trial evaluating LYT-200
as a single agent in AML and MDS, LYT-200
has demonstrated a favorable safety and tolerability profile as
well as early signals of potential clinical activity. This trial is
ongoing, in addition to a Phase 1/2 adaptive
design trial for the potential treatment of advanced/metastatic
solid tumors. Consistent with its hub-and-spoke model, PureTech
intends to advance LYT-200 via its Founded Entity Gallop
Oncology.
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 28 therapeutics and therapeutic candidates,
including two (Plenity® and EndeavorRx®) that have received
both US 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
the LYT-200 development program and the timing for
results from ongoing clinical trials of LYT-200, and our future prospects, developments 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,
2022 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 Bobbyn
+1 774 278 8273
nichole@tenbridgecommunications.com
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