Inozyme Pharma Highlights Inclusion of Generalized Arterial Calcification of Infancy (GACI) in Genomics England’s Generation Study of Rare Conditions
10 Oktober 2023 - 2:30PM
Inozyme Pharma, Inc. (Nasdaq: INZY)
(“Inozyme” or the “Company”), a clinical-stage rare disease
biopharmaceutical company developing novel therapeutics for the
treatment of pathologic mineralization and intimal proliferation,
highlighted the inclusion of Generalized Arterial Calcification of
Infancy (GACI), which is caused by mutations in the ENPP1 or ABCC6
genes, in the Genomic England’s Generation Study. This research
initiative, embedded within the UK’s National Health Service (NHS),
was developed to advance early detection and treatment of rare
genetic conditions, with the goal of setting a new standard for
newborn screening programs.
“We commend Genomics England for recognizing the crucial
importance of early detection of GACI as we advance INZ-701 into
clinical trials in infants. Including ENPP1 and ABCC6 in the
initial list of genes selected for this groundbreaking study
represents the hard work and dedication of the advocates, families,
and healthcare professionals who work tirelessly for those living
with this disease,” said Catherine Nester, R.N., Inozyme’s senior
vice president of healthcare professionals and patient engagement.
“The Generation Study has the potential to make a significant
impact on the creation of more comprehensive newborn screening
programs that save lives by empowering families to find appropriate
care for their babies as quickly as possible.”
The Generation Study is scheduled to begin in late 2023, with
the goal of sequencing the genomes of more than 100,000 infants and
paving the way for potential widespread implementation of
whole-genome sequencing in newborn screening. Genomics England
collaborated with NHS experts as well as scientists, healthcare
professionals (HCPs), and people living with rare conditions to
select 223 individual conditions for its current screening
program.
GACI is a rare genetic disorder that is fatal in 50 percent of
affected infants by six months of age due to severe and
pathological vascular calcification and neointimal proliferation
(overgrowth of smooth muscle cells inside blood vessels). The
disease can lead to the potential failure of major organs, such as
the heart, lungs, and kidneys, and it is caused by ENPP1 and ABCC6
Deficiencies, rare disorders for which there are currently no
approved treatments. Inozyme is developing INZ-701, an enzyme
replacement therapy, for the treatment of rare disorders like GACI
that impact the vasculature, soft tissue, or skeleton.
To learn more about Genomics England and its Generation Study,
read the full announcement here.
About INZ-701
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme
replacement therapy in development for the treatment of rare
disorders of the vasculature, soft tissue, and skeleton. In
preclinical studies, the experimental therapy has shown potential
to prevent pathologic mineralization and intimal proliferation (the
overgrowth of smooth muscle cells inside blood vessels), which can
drive morbidity and mortality in devastating genetic disorders such
as ENPP1 Deficiency and ABCC6 Deficiency. INZ-701 is currently in
clinical trials for the treatment of ENPP1 Deficiency and ABCC6
Deficiency.
About Inozyme Pharma
Inozyme Pharma, Inc. is a clinical-stage rare disease
biopharmaceutical company developing novel therapeutics for the
treatment of diseases impacting the vasculature, soft tissue, and
skeleton. Inozyme is developing INZ-701, an enzyme replacement
therapy, to address pathologic mineralization and intimal
proliferation which can drive morbidity and mortality in these
severe diseases. INZ-701 is currently in clinical trials for the
treatment of ENPP1 Deficiency and ABCC6 Deficiency.
For more information, please visit www.inozyme.com or
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and Facebook.
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 to the availability of
data from clinical trials, and the potential benefits of INZ-701.
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 clinical trials
of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; enroll
patients in ongoing and planned trials; 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; comply with covenants under its outstanding loan
agreement; 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.
ContactsInvestors:Inozyme
PharmaStefan Riley, Director of IR and Corporate
Communications(857)
330-8871stefan.riley@inozyme.com
Media: SmithSolve Matt Pera(973)
886-9150matt.pera@smithsolve.com
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