Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease
biopharmaceutical company developing novel therapeutics for the
treatment of pathologic mineralization and intimal proliferation,
today announced dosing of the first patient in its ENERGY-1 trial,
a Phase 1b clinical trial of INZ-701 in infants with ENPP1
Deficiency.
“Initiation of the ENERGY-1 trial in infants is an important
milestone as we continue to advance INZ-701 with the goal of
improving the lives of patients with ENPP1 Deficiency across all
age groups. We are committed to a global program to identify and
treat all newborns with this condition,” said Kurt Gunter, M.D.,
senior vice president and chief medical officer of Inozyme.
“Infants diagnosed with ENPP1 Deficiency face a high mortality
risk in the first months of life. Those who survive this critical
period often develop severe symptoms that adversely affect lifelong
health and quality of life. I am excited to serve as a principal
investigator in this trial as a first step towards delivering a
potentially lifesaving therapy for this patient population,”, said
David R. Weber, M.D., MSCE, Medical Director of the Center of Bone
Health, Division of Endocrinology and Diabetes at the Children’s
Hospital of Philadelphia (CHOP).
ENERGY-1 is a Phase 1b, single arm, open label clinical trial
designed to primarily assess the safety, tolerability,
pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in
infants with ENPP1 Deficiency. The trial is expected to enroll up
to eight infants between the ages of one and 12 months across
multiple sites in the United States and Europe. Patients will
receive subcutaneous doses of INZ-701 during the treatment period
of 52 weeks and may continue to receive INZ-701 in an extension
period beyond 52 weeks. Doses range from 0.2 mg/kg once weekly
through 0.6 mg/kg twice weekly, with the ability to increase the
dose further depending on the results of PK/PD and safety data.
Additional outcome measures include evaluation of plasma
pyrophosphate (PPi) levels, survival, growth, development,
functional performance, cardiac function, and exploratory
biomarkers.
Planned ENPP1 Deficiency Program Updates
The Company plans to host a conference call in July 2023 to
provide a program update on global development plans for INZ-701 in
patients with ENPP1 Deficiency. The update will cover regulatory
agreements on pivotal trial designs, including plans for our
pivotal trial of INZ-701 in pediatric patients which is planned to
begin in Q3 2023, an overview of the ENPP1 Deficiency opportunity,
and ongoing patient identification efforts.
About ENPP1 Deficiency
ENPP1 Deficiency is a progressive condition that manifests as a
spectrum of diseases. The estimated genetic prevalence of ENPP1
Deficiency is approximately 1 in 64,000 pregnancies. 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 intimal
proliferation (overgrowth of smooth muscle cells inside blood
vessels), resulting in myocardial infarction, stroke, or cardiac or
multiorgan failure. Approximately 50% of infants with ENPP1
Deficiency die within six months of birth. Children with ENPP1
Deficiency typically develop rickets, a condition diagnosed as
autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while
adults can develop osteomalacia (softened bones). ARHR2 and
osteomalacia lead to pain and mobility issues. Patients can also
exhibit signs and symptoms of hearing loss, arterial and joint
calcification, and cardiovascular complications. There are no
approved therapies for ENPP1 Deficiency.
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
Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and
ABCC6 Deficiency.
About Inozyme Pharma
Inozyme Pharma, Inc. (Nasdaq: INZY) is a clinical-stage rare
disease biopharmaceutical company developing novel therapeutics for
the treatment of diseases impacting the vasculature, soft tissue,
and skeleton. We are 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 Phase 1/2 clinical trials
for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
For more information, please visit www.inozyme.com and follow us
on LinkedIn, Twitter, 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 design of our clinical
trials, the potential benefits of INZ-701 and timing and contents
of our planned program update. 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; comply with the 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.
Contacts
Investors:Inozyme PharmaStefan Riley, Director of IR and
Corporate Communications(857)
330-8871stefan.riley@inozyme.com
Media:SmithSolveMatt Pera(973)
886-9150matt.pera@smithsolve.com
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