Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease
biopharmaceutical company developing novel therapeutics for the
treatment of abnormal mineralization, today announced a partnership
with Rady Children’s Institute for Genomic Medicine (RCIGM) to
advance and evaluate a novel newborn screening technology to
facilitate diagnosis of genetic diseases. The partnership includes
several leading genomics, biotechnology companies and patient
advocacy groups and focuses specifically on a diagnostic and
precision medicine guidance tool called BeginNGS™, which
incorporates rapid Whole Genome Sequencing (rWGS®) to currently
screen newborns for approximately 400 genetic diseases.
“Newborn screening will be essential to identifying and
initiating timely intervention in children with rare genetic
disorders like GACI (generalized arterial calcification of infancy)
as we advance INZ-701 through clinical testing,” said Catherine
Nester, vice president, physician and patient strategies at Inozyme
Pharma. “We look forward to working with Rady Children’s Institute
for Genomic Medicine, and with the BeginNGS consortium, to advance
the use of this promising screening technology.”
RCIGM is in a pilot evaluation that aims to supplement existing
newborn screening protocols at birthing hospitals throughout the
United States. The pilot program’s goal is for BeginNGS to become
the genetic disease screening standard, with testing expanding to
approximately 1,000 [disorders] and sequencing of 3.7 million
newborns annually. Founding members of the public-private BeginNGS
consortium include Inozyme, Alexion, Travere Therapeutics, and
several patient advocacy groups that are helping to advance this
program.
“RCIGM helped pioneer the use of rWGS for diagnosis of
genetic disease in intensive care settings,” said Stephen
Kingsmore, MD, DSc, president and CEO of RCIGM. “With the
proven clinical utility of diagnostic rWGS , we are using that
experience to screen, diagnose, and help treat genetic conditions
at or before onset of symptoms. Through a public-private consortium
of leading organizations such as Inozyme, and advocacy groups in
pediatrics, genetics, biopharma, biotech, and information
technology, we aim to scale newborn sequencing to every
life-threatening childhood genetic disease, RCIGM believes now is
the time to end the diagnostic and therapeutic odyssey for all
children with treatable genetic diseases.”
BeginNGS developed through a research collaboration with
Alexion; AstraZeneca’s Rare Disease group; Illumina, Inc.; TileDB;
Fabric Genomics; and Genomemon, which uses rWGS to diagnose and
identify treatment options for genetic conditions before symptoms
begin. This approach represents an advance over current pediatric
uses of rWGS that focus mainly on children who are already
critically ill. Once a diagnosis is made, BeginNGS uses
Genome-to-Treatment (GTRx™), a tool that provides immediate
treatment guidelines to help physicians understand genetic
conditions and their available treatment options.
Addressing the Need for Enhanced Newborn Screening
Tools
Traditional newborn screening is one of the most successful
public health programs in the United States. Of nearly 4 million
babies born annually, 98 percent are tested in the first days of
life. The BeginNGS test identifies serious childhood diseases that
have effective treatments. States currently screen for only 31 to
76 of the hundreds of severe, childhood genetic diseases that have
available treatments. Adding a new condition to the screening
protocol is slow (5 to 6 years per condition), laborious, and
costly. In the last decade, WGS has increased in speed, diagnostic
performance, and scalability. BeginNGS will not replace the current
biochemical newborn screening paradigm; rather, it is designed to
complement the newborn screening processes and infrastructure that
are already in place.
“We are thrilled at the prospect of newborn screening to assist
in early identification of infants affected by ENPP1 Deficiency and
ABCC6 Deficiency via Inozyme’s collaboration with Rady Children’s
Institute for Genomic Medicine. Early diagnosis is crucial to
improving a baby’s chances of survival and long-term health if they
have these rare and devastating diseases,” said Christine O’Brien
and Liz Molloy, co-presidents of GACI Global.
About Rady Children’s Institute for Genomic
Medicine
Rady Children’s Institute for Genomic Medicine is transforming
pediatric critical care by advancing disease-specific healthcare
for infants and children with rare disease. Discoveries at the
Institute are enabling rapid diagnosis and targeted treatment of
critically ill newborns and pediatric patients at Rady Children’s
Hospital-San Diego and a growing network of more than 60 children’s
hospitals nationwide. The vision is to expand delivery of this
life-changing technology to enable the practice of Rapid Precision
Medicine™ at children’s hospitals across the nation and the world.
RCIGM is a non-profit, research institute embedded within Rady
Children’s Hospital and Health Center.
About Inozyme Pharma
Inozyme Pharma, Inc. (Nasdaq: INZY) is a
clinical-stage rare disease biopharmaceutical company developing
novel therapeutics for the treatment of diseases of abnormal
mineralization impacting the vasculature, soft tissue, and
skeleton. Through our in-depth understanding of the biological
pathways involved in mineralization, we are pursuing the
development of therapeutics to address the underlying causes of
these debilitating diseases. It is well established that two genes,
ENPP1 and ABCC6, play key roles in a critical mineralization
pathway and that defects in these genes lead to abnormal
mineralization. We are initially focused on developing a novel
therapy, INZ-701, to treat the rare genetic diseases of ENPP1 and
ABCC6 Deficiencies. INZ-701 is currently in Phase 1/2 clinical
trials for the treatment of ENPP1 Deficiency and ABCC6
Deficiency.
Inozyme Pharma was founded in 2017 by Joseph
Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel
Bolte, MSc, MBA, with technology developed by Dr. Braddock and
licensed from Yale University. For more information, please visit
www.inozyme.com.
About ENPP1 Deficiency
ENPP1 Deficiency is a heterogenous, progressive
condition with high infant mortality in the first six months of
life. Individuals who present in utero or in infancy are typically
diagnosed with generalized arterial calcification of infancy
(GACI), which is characterized by extensive vascular calcification
and neointimal proliferation (overgrowth of smooth muscle cells
inside blood vessels), resulting in stroke, cardiac or multiorgan
failure. Children with ENPP1 Deficiency typically experience
rickets and other skeletal manifestations, a condition also known
as autosomal-recessive hypophosphatemic rickets type 2 (ARHR2).
Adults may experience osteomalacia (softened bones), pain,
stiffness and impaired quality of life. There are no approved
therapies for ENPP1 Deficiency.
About ABCC6 Deficiency
ABCC6 Deficiency is a rare, severe, inherited
disorder caused by mutations in the ABCC6 gene, leading to low
levels of PPi. PPi is essential for preventing harmful soft tissue
calcification and regulating bone mineralization. ABCC6 Deficiency
is a systemic and progressively debilitating condition, which
affects more than 67,000 individuals worldwide. Infants with ABCC6
Deficiency are diagnosed with GACI type 2, a condition that
resembles GACI type 1, the infant form of ENPP1 Deficiency. In
older patients, ABCC6 Deficiency presents as pseudoxanthoma
elasticum (PXE), which is characterized by pathological
mineralization in blood vessels and soft tissues clinically
affecting the skin, eyes, and vascular system. There are no
approved therapies for ABCC6 Deficiency.
About INZ-701
INZ-701 is a clinical-stage enzyme replacement
therapy in development for the treatment of mineralization
disorders of the circulatory system, bones, and kidneys. In
preclinical studies, the experimental therapy has shown potential
to generate PPi and to restore it to appropriate physiological
levels, thereby preventing calcification in the vasculature and
kidneys, while at the same time normalizing bone mineralization.
Inozyme is developing INZ-701 for certain rare, life-threatening,
and devastating genetic disorders such as ENPP1 Deficiency and
ABCC6 Deficiency in which PPi levels are below the normal
physiological levels. INZ-701 is currently in Phase 1/2 clinical
trials for the treatment of ENPP1 Deficiency and ABCC6
Deficiency.
Cautionary Note Regarding
Forward-Looking Statements
Statements in this press release about future
expectations, plans, and prospects, as well as any other statements
regarding matters that are not historical facts, may constitute
"forward-looking statements" within the meaning of The Private
Securities Litigation Reform Act of 1995. These statements include,
but are not limited to, statements relating the partnership with
Rady Children’s Institute for Genomic Medicine. The words
"anticipate," "believe," "continue," "could," "estimate," "expect,"
"intend," "may," "plan," "potential," "predict," "project,"
"should," "target," "will," "would," and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Any
forward-looking statements are based on management's current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in, or implied by,
such forward-looking statements. These risks and uncertainties
include, but are not limited to, risks associated with the
Company's ability to conduct its ongoing Phase 1/2 clinical trials
of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and
maintain necessary approvals from the FDA and other regulatory
authorities; continue to advance its product candidates in
preclinical studies and clinical trials; replicate in later
clinical trials positive results found in preclinical studies and
early-stage clinical trials of its product candidates; advance the
development of its product candidates under the timelines it
anticipates in planned and future clinical trials; obtain,
maintain, and protect intellectual property rights related to its
product candidates; manage expenses; and raise the substantial
additional capital needed to achieve its business objectives. For a
discussion of other risks and uncertainties, and other important
factors, any of which could cause the Company's actual results to
differ from those contained in the forward-looking statements, see
the "Risk Factors" section in the Company's most recent Annual
Report on Form 10-K filed with the Securities and Exchange
Commission, as well as discussions of potential risks,
uncertainties, and other important factors, in the Company's most
recent filings with the Securities and Exchange Commission. In
addition, the forward-looking statements included in this press
release represent the Company's views as of the date hereof and
should not be relied upon as representing the Company's views as of
any date subsequent to the date hereof. The Company anticipates
that subsequent events and developments will cause the Company's
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so.
Contacts
Investors:Inozyme PharmaStefan Riley, Director of Investor
Relations(857)
330-8871stefan.riley@inozyme.com
Media: SmithSolve Matt Pera(973)
886-9150matt.pera@smithsolve.com
Inozyme Pharma (NASDAQ:INZY)
Historical Stock Chart
From Apr 2024 to May 2024
Inozyme Pharma (NASDAQ:INZY)
Historical Stock Chart
From May 2023 to May 2024