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,
today announced that it will present three posters at the American
Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting,
which is being held October 13-16, 2023 in Vancouver, BC, Canada.
Details of the presentations are as follows:
Title: A Phase 1/2 Open-Label, Multiple
Ascending Dose Clinical Study to Evaluate the Safety, Tolerability,
Pharmacokinetics, and Pharmacodynamics of INZ-701 in Adults with
ENPP1 DeficiencyFormat: Poster
PresentationDates: Friday, October 13,
2023/Sunday, October 15, 2023Session Times:
6:00-7:30 p.m. PT/ 1:30-3:00 p.m.
PTAuthor/Presenter: Yves Sabbagh, Ph.D. (Senior
Vice President and Chief Scientific Officer, Inozyme Pharma)
Title: A Phase 1/2 Open-Label, Multiple
Ascending Dose Clinical Study to Evaluate the Safety, Tolerability,
Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an
Open-Label Long-Term Extension Period in Adults with ABCC6
Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE): An
Interim AnalysisFormat: Poster
PresentationDates: Friday, October 13,
2023/Saturday, October 14, 2023 Session Times:
6:00-7:00 p.m. PT/ 1:30-3:00 p.m. PT
Author/Presenter: Yves Sabbagh, Ph.D. (Senior Vice
President and Chief Scientific Officer, Inozyme Pharma)
Title: ENPP1 enzyme replacement therapy
improves brain calcification but does not rescue skeletal phenotype
in a mouse model for craniometaphyseal
dysplasiaFormat: Poster
PresentationDate: Saturday, October 14,
2023Session Time: 1:30-3:00 p.m.
PTAuthor/Presenter: I-Ping Chen, D.D.S., Ph.D.
(Associate Professor, Oral Health and Diagnostic Sciences, UConn
Health)
About ENPP1 Deficiency
ENPP1 Deficiency is a progressive condition that manifests as a
spectrum of diseases. 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 adolescents and
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.
INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial
Design
The ongoing Phase 1/2 open-label clinical trial initially
enrolled nine adult patients with ENPP1 Deficiency at sites in
North America and Europe. The trial will primarily assess the
safety and tolerability of INZ-701 in adult patients with ENPP1
Deficiency, as well as characterize the pharmacokinetic (PK) and
pharmacodynamic (PD) profile of INZ-701, including evaluation of
the PD marker, plasma pyrophosphate (PPi) and other biomarker
levels. In the Phase 1 dose-escalation portion of the trial,
Inozyme assessed INZ-701 for 32 days at doses of 0.2 mg/kg, 0.6
mg/kg, and 1.8 mg/kg administered via subcutaneous injection twice
weekly, with three patients per dose cohort. Doses were selected
based on preclinical studies and PK/PD modeling. The Phase 1
dose-escalation portion of the trial sought to identify a safe,
tolerable dose that increases PPi levels, and that can be used for
further clinical development. Following completion of the Phase 1
portion of the first three cohorts, Inozyme dosed patients in a
fourth cohort at 1.2 mg/kg to investigate the potential for
once-weekly dosing of INZ-701. The open-label Phase 2 extension
portion of the trial is assessing long-term safety, PK, and PD of
continued treatment with INZ-701 for at least 48 weeks, where
patients may self-administer INZ-701. Exploratory endpoints include
evaluations of skeletal, vascular, physical function and
patient-reported outcomes.
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 generalized arterial calcification of infancy (GACI)
type 2, a condition that resembles GACI type 1, the infant form of
ENPP1 Deficiency. In older individuals, 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.
INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial
Design
The ongoing Phase 1/2 open-label clinical trial enrolled ten
adult patients with ABCC6 Deficiency at sites in the United States
and Europe. The trial will primarily assess the safety and
tolerability of INZ-701 in adult patients with ABCC6 Deficiency, as
well as characterize the pharmacokinetic (PK) and pharmacodynamic
(PD) profile of INZ-701, including the evaluation of levels of
plasma PPi and other biomarkers. In the Phase 1 dose-escalation
portion of the trial, Inozyme assessed INZ-701 for 32 days at doses
of 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg administered via
subcutaneous injection twice weekly, with three patients per dose
cohort. Doses were selected based on preclinical studies and PK/PD
modeling. The Phase 1 dose-escalation portion of the trial sought
to identify a safe, tolerable dose for further development that
increases PPi levels. The open-label Phase 2 extension portion of
the trial is assessing long-term safety, PK, and PD of continued
treatment with INZ-701 for at least 48 weeks, where patients may
self-administer INZ-701. Exploratory endpoints will include
evaluations of vascular, ophthalmologic, physical function and
patient-reported outcomes.
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 follow Inozyme
on LinkedIn, X (formerly
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 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.
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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