Phase 1/2 MYCHELANGELO™ I Clinical Trial
Underway to Evaluate Safety, Tolerability and Preliminary Antitumor
Activity of OTX-2002
CAMBRIDGE, Mass., Nov. 2, 2022
/PRNewswire/ -- Omega Therapeutics, Inc. ("Omega"), a
clinical-stage biotechnology company pioneering the first
systematic approach to use mRNA therapeutics as a new class of
programmable epigenetic medicines, today announced that the U.S.
Food and Drug Administration (FDA) has granted Orphan Drug
Designation for OTX-2002, the company's first-in-class epigenomic
controller engineered to downregulate c-Myc (MYC), for the
treatment of hepatocellular carcinoma (HCC).
The company recently announced that the first patient had been
dosed in its Phase 1/2 MYCHELANGELO™ I clinical trial investigating
the safety, tolerability, pharmacokinetics, pharmacodynamics, and
preliminary antitumor activity of OTX-2002 as a monotherapy and in
combination with standard of care therapies in patients with
relapsed or refractory HCC and other solid tumor types known for
association with the MYC oncogene.
"HCC is a devastating illness that often develops resistance to
current standard of care therapeutics. The FDA's decision to grant
orphan drug designation for OTX-2002 underscores the need for novel
therapies to address HCC and the potential of epigenomic
programming to transform the treatment landscape," said
Mahesh Karande, President and Chief
Executive Officer of Omega Therapeutics. "We look forward to
continuing to work with clinical investigators, patients and the
FDA as we advance our MYCHELANGELO clinical program and evaluate
the potential of OTX-2002 to bring a new treatment option to the
liver cancer patient community."
About Orphan Drug
Designation
The FDA's Orphan Drug Designation program provides orphan status
to drugs defined as those intended for the treatment, diagnosis or
prevention of rare diseases that affect fewer than 200,000 people
in the United States. Orphan drug
designation qualifies the sponsor of the drug for certain
development incentives, including tax credits for qualified
clinical testing, prescription drug user fee exemptions and
seven-year marketing exclusivity upon FDA approval.
About Hepatocellular
Carcinoma
Hepatocellular carcinoma (HCC) is a leading cause of cancer
deaths worldwide and represents an unmet clinical need with few
therapeutic options. Tyrosine kinase inhibitors (TKIs) have been
used as a systemic therapy for HCC, but patients frequently develop
resistance with oncogenic MYC identified as a correlating
prognostic factor. The MYC oncogene is associated with aggressive
disease in up to 70% of patients with HCC.
About OTX-2002
OTX-2002 is a first-in-class Omega Epigenomic
Controller™ in development for the treatment of
hepatocellular carcinoma (HCC). OTX-2002 is an mRNA therapeutic
delivered via lipid nanoparticles (LNPs) and is designed to
downregulate MYC expression pre-transcriptionally through
epigenetic modulation while potentially overcoming MYC
autoregulation. MYC is a master transcription factor that regulates
cell proliferation, differentiation and apoptosis and plays a
significant role in more than 50% of all human cancers. OTX-2002 is
currently being evaluated in the Phase 1/2
MYCHELANGELO™ I trial in patients with relapsed or
refractory HCC and other solid tumor types known for association
with the MYC oncogene;
visit clinicaltrials.gov (NCT05497453) for more
details.
About Omega
Therapeutics
Omega Therapeutics, founded by Flagship Pioneering, is a
clinical-stage biotechnology company pioneering the first
systematic approach to use mRNA therapeutics as a new class of
programmable epigenetic medicines. The company's OMEGA Epigenomic
Programming™ platform harnesses the power of epigenetics, the
mechanism that controls gene expression and every aspect of an
organism's life from cell genesis, growth, and differentiation to
cell death. Using a suite of technologies, paired with Omega's
process of systematic, rational, and integrative drug design, the
OMEGA platform enables control of fundamental epigenetic processes
to correct the root cause of disease by returning aberrant gene
expression to a normal range without altering native nucleic acid
sequences. Omega's modular and programmable mRNA medicines, Omega
Epigenomic Controllers™, target specific epigenomic loci within
insulated genomic domains, EpiZips™, from amongst thousands of
unique, mapped, and validated genome-wide DNA-sequences, with high
specificity to durably tune single or multiple genes to treat and
cure diseases through Precision Genomic Control™. Omega is
currently advancing a broad pipeline of development candidates
spanning a range of disease areas, including oncology, regenerative
medicine, multigenic diseases including immunology, and select
monogenic diseases, including alopecia.
For more information, visit omegatherapeutics.com, or
follow us on Twitter and LinkedIn.
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 statements
regarding the timing and design of our Phase 1/2
MYCHELANGELO™ I clinical trial; the potential of
the OMEGA platform to engineer programmable epigenetic mRNA
therapeutics that successfully regulate gene expression by
targeting insulated genomic domains; expectations surrounding the
potential of our product candidates, including our lead OEC
candidate OTX-2002; and expectations regarding our pipeline,
including trial design, initiation of preclinical studies and
advancement of multiple preclinical development programs in
oncology, immunology, regenerative medicine, and select monogenic
diseases. These statements are neither promises nor guarantees, but
involve known and unknown risks, uncertainties and other important
factors that may cause our actual results, performance or
achievements to be materially different from any future results,
performance or achievements expressed or implied by the
forward-looking statements, including, but not limited to, the
following: the novel technology on which our product candidates are
based makes it difficult to predict the time and cost of
preclinical and clinical development and subsequently obtaining
regulatory approval, if at all; the substantial development and
regulatory risks associated with epigenomic controller machines due
to the novel and unprecedented nature of this new category of
medicines; our limited operating history; the incurrence of
significant losses and the fact that we expect to continue to incur
significant additional losses for the foreseeable future; our need
for substantial additional financing; our investments in research
and development efforts that further enhance the OMEGA platform,
and their impact on our results; uncertainty regarding preclinical
development, especially for a new class of medicines such as
epigenomic controllers; potential delays in and unforeseen costs
arising from our clinical trials; the fact that our product
candidates may be associated with serious adverse events,
undesirable side effects or have other properties that could halt
their regulatory development, prevent their regulatory approval,
limit their commercial potential, or result in significant negative
consequences; the impact of increased demand for the manufacture of
mRNA and LNP based vaccines to treat COVID-19 on our development
plans; difficulties manufacturing the novel technology on which our
OEC candidates are based; our ability to adapt to rapid and
significant technological change; our reliance on third parties for
the manufacture of materials; our ability to successfully acquire
and establish our own manufacturing facilities and infrastructure;
our reliance on a limited number of suppliers for lipid excipients
used in our product candidates; our ability to advance our product
candidates to clinical development; and our ability to obtain,
maintain, enforce and adequately protect our intellectual property
rights. These and other important factors discussed under the
caption "Risk Factors" in our Quarterly Report on Form 10-Q for the
quarter ended June 30, 2022, and our other filings with
the SEC, could cause actual results to differ materially
from those indicated by the forward-looking statements made in this
press release. Any such forward-looking statements represent
management's estimates as of the date of this press release. While
we may elect to update such forward-looking statements at some
point in the future, we disclaim any obligation to do so, even if
subsequent events cause our views to change.
Investor Contact:
Eva Stroynowski
617-949-4370
estroynowski@omegatx.com
Media Contact:
Jason
Braco
LifeSci Communications
646.751.4361
jbraco@lifescicomms.com
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SOURCE Omega Therapeutics