- First Patient Dosed in Phase 1/2
MYCHELANGELO™ I Trial of
OTX-2002
- OTX-2002 Granted Orphan Drug Designation by U.S. FDA for the
Treatment of Hepatocellular Carcinoma
- OTX-2101 for MYC-Driven Non-Small Cell Lung Cancer Selected
as Second Omega Epigenomic Controller™ Development
Candidate
- $148.3 Million in Cash, Cash
Equivalents and Marketable Securities as of September 30, 2022
CAMBRIDGE, Mass., Nov. 8, 2022
/PRNewswire/ -- Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"),
a clinical-stage biotechnology company pioneering the first
systematic approach to use mRNA therapeutics as a new class of
programmable epigenetic medicines by leveraging its OMEGA
Epigenomic Programming™ platform, today announced financial results
for the third quarter ended September 30,
2022 and highlighted recent Company progress.
"The significant progress we are making on all fronts across our
development pipeline is exciting, including the initiation of the
MYCHELANGELO™ clinical program for OTX-2002, which represents the
first-ever dosing of an epigenomic controller in a patient and
marks a significant milestone in our journey to bring novel and
programmable mRNA therapeutics to patients," said Mahesh Karande, President and Chief Executive
Officer of Omega Therapeutics. "We were also delighted to announce
our next development candidate, OTX-2101 for the treatment of
patients with MYC-driven non-small cell lung cancer. Our focus is
on advancing our lead programs, OTX-2002 and OTX-2101, as well as
driving additional discovery and preclinical assets forward to
further expand our pipeline."
Recent Corporate Highlights
Development Pipeline and Platform
- First Patient Dosed in Landmark MYCHELANGELO I Clinical
Trial for OTX-2002, the First-Ever Omega Epigenomic
Controller™ (OEC): The Phase 1/2 study is the first-ever
study to evaluate this new class of programmable mRNA therapeutics
designed to treat or cure serious diseases through precision
genomic control. The study will evaluate the safety, tolerability,
pharmacokinetics, pharmacodynamics, and preliminary antitumor
activity of OTX-2002 as a monotherapy (Part 1) and in combination
with standard of care therapies (Part 2) in patients with relapsed
or refractory hepatocellular carcinoma (HCC) and other solid tumor
types known for association with the c-Myc (MYC) oncogene. The
study is expected to enroll approximately 190 patients at clinical
trial sites in the United States,
Asia, and Europe.
- OTX-2002 Granted Orphan Drug Designation by U.S. Food and
Drug Administration (FDA) for Hepatocellular Carcinoma
(HCC): OTX-2002 is a rationally engineered, novel and
programmable mRNA therapeutic designed to downregulate MYC
expression pre-transcriptionally through epigenetic modulation
while potentially overcoming MYC autoregulation. The FDA's Orphan
Drug Designation Program provides orphan status to drugs intended
for the treatment, diagnosis or prevention of rare diseases that
affect fewer than 200,000 people in the
United States.
- OTX-2101 for MYC-Driven Non-Small Cell Lung Cancer (NSCLC)
Selected as Second Omega Epigenomic Controller Development
Candidate: OTX-2101 is the second candidate in this new class
of programmable mRNA therapeutics designed to downregulate MYC
expression pre-transcriptionally through epigenetic modulation
while potentially overcoming MYC autoregulation. Preclinical data
presented at the 2022 American Society of Gene & Cell Therapy
(ASGCT) Annual Meeting demonstrated OTX-2101 potently downregulates
MYC in multiple NSCLC cell lines. OTX-2101 effectively reduced
tumor growth in vivo and was well tolerated in murine
xenograft models, further supporting its clinical potential.
Investigational New Drug (IND)-enabling studies for OTX-2101 are
underway.
- Completed Development Candidate-enabling Activities for
Several OECs: Beyond HCC and NSCLC, the Company continues to
advance multiple OECs from the OMEGA Epigenomic Programming
platform through preclinical studies. The CXCL 1-8-targeting OEC
has been characterized in preclinical studies and has potential in
several indications including neutrophilic asthma, acute
respiratory distress syndrome (including COVID-related), oncology,
and dermatological and rheumatological indications, representing a
potential franchise opportunity. The Company continues additional
preclinical work for its OEC development programs spanning
oncology, multigenic diseases including immunology, regenerative
medicine, and select monogenic diseases.
Corporate
- Rainer Boehm Appointed to Board of Directors: Mr. Boehm
joined the Board on August 30, 2022.
He serves on the Company's audit and compensation committees. He
brings over 30 years of successful and diverse clinical,
managerial, drug development, and commercialization experience to
Omega.
Third Quarter 2022 Financial Results
As of September 30, 2022, the
Company had cash, cash equivalents and marketable securities
totaling $148.3 million.
Research and development (R&D) expenses for the third
quarter of 2022 were $20.7 million,
compared to $12.3 million for the
third quarter of 2021. The $8.4
million increase in R&D expense was primarily driven by
an increase in personnel-related expenses, external manufacturing
costs, and study costs in support of the advancement of our
programs.
General and administrative (G&A) expenses for the third
quarter of 2022 were $5.2 million,
compared to $4.5 million for the
third quarter of 2021. The $0.7
million increase in G&A expense was primarily driven by
an increase in personnel-related expenses to support business
growth.
Net loss for the third quarter of 2022 was $25.8 million, compared to $18.5 million for the third quarter of 2021,
driven predominantly by increased R&D and G&A expenses to
support the Company's growth and operations as a public
company.
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™, are designed to 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
MYCHELANGELOTM 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 OTX-2002 and
OTX-2101; 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 most recent Quarterly Report on Form
10-Q and our Annual Report on Form 10-K for the year ended
December 31, 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 and Media Contact:
Eva Stroynowski
617.949.4370
estroynowski@omegatx.com
Media Contact:
Jason
Braco
LifeSci Communications
646.751.4361
jbraco@lifescicomms.com
Omega Therapeutics,
Inc.
|
Condensed
consolidated statements of operations and comprehensive
loss
|
(in thousands,
except share and per share amounts)
|
|
|
Three Months
Ended
September 30,
|
|
|
Nine Months
Ended
September 30,
|
|
|
2022
|
|
|
2021
|
|
|
2022
|
|
|
2021
|
|
Collaboration revenue
from related party
|
$
|
595
|
|
|
$
|
—
|
|
|
$
|
1,338
|
|
|
$
|
—
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
20,670
|
|
|
|
12,289
|
|
|
|
54,329
|
|
|
|
33,222
|
|
General and
administrative
|
|
5,198
|
|
|
|
4,459
|
|
|
|
16,466
|
|
|
|
10,911
|
|
Related party expense,
net
|
|
712
|
|
|
|
473
|
|
|
|
2,342
|
|
|
|
1,235
|
|
Total operating
expenses
|
|
26,580
|
|
|
|
17,221
|
|
|
|
73,137
|
|
|
|
45,368
|
|
Loss from
operations
|
|
(25,985)
|
|
|
|
(17,221)
|
|
|
|
(71,799)
|
|
|
|
(45,368)
|
|
Other expense,
net:
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
(expense), net
|
|
184
|
|
|
|
(339)
|
|
|
|
(26)
|
|
|
|
(741)
|
|
Change in fair value
of warrant liability
|
|
—
|
|
|
|
(970)
|
|
|
|
—
|
|
|
|
(1,310)
|
|
Other income
(expense), net
|
|
2
|
|
|
|
2
|
|
|
|
(50)
|
|
|
|
(7)
|
|
Total other income
(expense), net
|
|
186
|
|
|
|
(1,307)
|
|
|
|
(76)
|
|
|
|
(2,058)
|
|
Net loss
|
$
|
(25,799)
|
|
|
$
|
(18,528)
|
|
|
$
|
(71,875)
|
|
|
$
|
(47,426)
|
|
Net loss per common
stock attributable to common
stockholders, basic and diluted
|
$
|
(0.54)
|
|
|
$
|
(0.57)
|
|
|
$
|
(1.50)
|
|
|
$
|
(3.41)
|
|
Weighted-average common
stock used in net loss
per share attributable to common stockholders,
basic and diluted
|
|
47,854,965
|
|
|
|
32,303,540
|
|
|
|
47,837,490
|
|
|
|
13,898,089
|
|
Comprehensive
loss:
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
$
|
(25,799)
|
|
|
$
|
(18,528)
|
|
|
$
|
(71,875)
|
|
|
$
|
(47,426)
|
|
Other comprehensive
loss:
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on marketable securities
|
|
89
|
|
|
|
—
|
|
|
|
(855)
|
|
|
|
—
|
|
Comprehensive
loss
|
$
|
(25,710)
|
|
|
$
|
(18,528)
|
|
|
$
|
(72,730)
|
|
|
$
|
(47,426)
|
|
Omega Therapeutics,
Inc.
|
Condensed
Consolidated Balance Sheets
|
(in
thousands)
|
|
|
September 30,
|
|
|
December
31,
|
|
|
2022
|
|
|
2021
|
|
Assets
|
|
|
|
|
|
Cash and cash
equivalents
|
$
|
76,614
|
|
|
$
|
186,482
|
|
Marketable
securities
|
|
71,732
|
|
|
|
38,845
|
|
Other
assets
|
|
21,719
|
|
|
|
8,006
|
|
Total
assets
|
$
|
170,065
|
|
|
$
|
233,333
|
|
Liabilities and
stockholders' equity
|
|
|
|
|
|
Liabilities
|
$
|
36,414
|
|
|
$
|
32,705
|
|
Stockholders'
equity
|
|
133,651
|
|
|
|
200,628
|
|
Total liabilities
and stockholders' equity
|
$
|
170,065
|
|
|
$
|
233,333
|
|
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SOURCE Omega Therapeutics