Organovo Announces Target Validation Ahead of Schedule, Accelerating Timeline for First Crohn’s Disease Drug Candidates
November 15 2022 - 8:05AM
Organovo Holdings, Inc. (Nasdaq: ONVO), a three-dimensional biology
(3D biology) company focused on delivering scientific and medical
breakthroughs using novel technologies including 3D bioprinting,
today announced that it has successfully advanced the use of its
first inflammatory bowel disease (IBD) model and has achieved the
next milestone, target validation. The company announced in May
2022 that it had achieved a successful Crohn’s disease model that
demonstrates key aspects of patient biology that differ from a
non-diseased state and that the model would be used to find and
characterize therapeutics. The Crohn’s disease model has since been
utilized to understand the biology of disease and identify specific
gene targets. Treating some of those targets, by introducing a
compound directly to the 3D tissue model that affects the target,
has now been shown to reproducibly reduce disease. Multiple targets
are now considered validated, and they represent specific druggable
opportunities that the company plans to leverage. Organovo plans to
advance at least one target program to medicinal chemistry by the
end of 2022 to build a proprietary new drug for Crohn’s disease.
“The Organovo team has successfully identified
disease regulated genes from our Crohn's disease model and
importantly, identified a subset that are disease-driver genes,”
said Jeff Miner, Ph.D., Organovo’s Chief Scientific Officer. “These
disease-drivers directly affect the disease when modulated and
represent superb therapeutic targets” he added.
Organovo Executive Chairman Keith Murphy
commented, “Organovo’s progress towards our goal has been driven by
a strong, dedicated scientific team that has continued to rapidly
advance our programs. We are pleased to have this step completed
earlier than expected, and plan to continue to meet or exceed
stockholder expectations as we move therapeutics to the clinic to
meet unmet medical needs.”
In September 2020, Organovo announced that it
had updated its business model to capitalize on its human 3D tissue
technologies in drug discovery, building disease models to find
effective therapies that are more likely to succeed in the clinic
than drugs discovered using animal models. We have focused on the
creation of three-dimensional intestinal disease models to discover
therapeutic opportunities that leverage the insights possible from
the truer human biology achievable in 3D human disease models.
More than 1 million people in North America
suffer from Crohn’s and its prevalence is increasing globally.
Because of its progressive nature, more than 70% will require at
least one surgical intervention to relieve symptoms. Current drugs
can slow progression in about 50% of patients, but none are
curative. Defects in the intestinal lining and inflammation can
lead to abdominal pain, severe diarrhea, fatigue, weight loss,
malnutrition, and lower quality of life. Chronic inflammation leads
to ulcers, fistulas and bowel obstructions caused by fibrosis.
Organovo believes that its 3D tissue technologies are well suited
to drug and target discovery in IBD because they contain a
functional intestinal epithelium that is affected by disease and a
stromal layer demonstrating disease-dependent fibrosis, key targets
for therapy.
The drug discovery process using 3D tissues
consists of several stages. Two early steps that are uniquely
important in Organovo’s approach to drug discovery are creation of
a disease model followed by target discovery and validation. The
steps involved in each require groundbreaking science that involves
the application of tools specific to our approach. We believe the
achievement of these goals represents a strong step forward and
lowering of risk. The remaining steps after validation of a target
are typical pharma development steps to advance a drug to Phase 1
clinical trials. The next phase will be medicinal chemistry, the
creation and evaluation of new chemical entities to pick the best
drug candidate. Organovo plans to do this work through an outside
contract research organization. Organovo is on track to have its
medicinal chemistry program result in an investigative new drug
(IND) application by 2025 in alignment with our previously
communicated objective to have multiple INDs by that time.
Medicinal chemistry, preclinical safety studies,
and other common tasks that follow consist of common practices
across pharmaceutical development, and are not unique to 3D drug
discovery, but can benefit greatly from the additional insights
that can be provided by testing candidates in human tissue 3D
models as the candidates are screened and developed. Since these
steps are more typical, the risk profile is similar to traditional
pharmaceutical development at these stages. However, upon entering
the clinic and seeking evidence of efficacy in humans, we believe
that our use of human 3D disease models to select drug candidates
will result in significantly higher probability of success.
Organovo is working on additional IBD disease
models representing different aspects of disease, each of which can
represent a significant advancement over previous tools. We will
also seek to leverage validated targets and scientific knowledge
from these disease models into business development deals and
partnerships with pharmaceutical companies.
About Organovo
Organovo is an early-stage biotechnology company
that is developing and utilizing highly customized 3D human tissues
as dynamic models of healthy and diseased human biology for drug
development. The company’s proprietary technology is being used to
build functional 3D human tissues that mimic key aspects of native
human tissue composition, architecture, function and disease.
Organovo’s advances include cell type-specific compartments,
prevalent intercellular tight junctions, and the formation of
microvascular structures. Management believes these attributes can
enable critical complex, multicellular disease models that can be
used to develop clinically effective drugs for selected therapeutic
areas.
Forward Looking Statements
Any statements contained in this press release
that do not describe historical facts constitute forward-looking
statements as that term is defined in the Private Securities
Litigation Reform Act of 1995. Any forward-looking statements
contained herein are based on current expectations, but are subject
to a number of risks and uncertainties. These risks and
uncertainties and other factors are identified and described in
more detail in the Company’s filings with the SEC, including
its Annual Report on Form 10-K filed with
the SEC on June 10, 2022, as such risk factors are
updated in its most recently filed Quarterly Report on Form 10-Q
filed with the SEC on November 10, 2022. You should not place undue
reliance on these forward-looking statements, which speak only as
of the date that they were made. These cautionary statements should
be considered with any written or oral forward-looking statements
that the Company may issue in the future. Except as required by
applicable law, including the securities laws of the United
States, the Company does not intend to update any of the
forward-looking statements to conform these statements to reflect
actual results, later events or circumstances or to reflect the
occurrence of unanticipated events.
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