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 host an
Investor and Analyst Event on Tuesday, September 26, 2023 at 8:00
a.m. Eastern Time.
During the webcast and conference call, members of Inozyme
management will review interim data from the Phase 2 portion of the
ongoing trial of INZ-701 in adult patients with ENPP1 Deficiency.
The Company will also provide an update on the safety and
pharmacodynamic data from the ongoing Phase 1/2 trial of INZ-701 in
adult patients with ABCC6 Deficiency (PXE).
Conference Call and Webcast Details
The live webcast will be accessible through the Investor
Relations section of Inozyme’s website under events.
Alternatively, the conference call may be accessed by dialing:
Domestic Dial-in Number: 1-877-270-2148International Dial-in
Number: 1-412-902-6510
Participants should ask to join the Inozyme
Pharma call.
For those unable to participate live, a replay will be available
in the Investor Relations section of Inozyme’s website for a
limited time following the event.
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,
pharmacokinetics, and pharmacodynamics of continued treatment with
INZ-701 for up to 48 weeks, where patients may receive doses of
INZ-701 at home depending on site-specific protocols. Exploratory
endpoints will 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 initially
enrolled nine 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 will assess long-term safety,
pharmacokinetics, and pharmacodynamics of continued treatment with
INZ-701 for up to 48 weeks, where patients may receive doses of
INZ-701 at home depending on site-specific protocols. 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 design of our
clinical trials, the potential benefits of INZ-701 and the timing
and contents of our planned interim data 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 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: SmithSolve Matt Pera(973)
886-9150matt.pera@smithsolve.com
Inozyme Pharma (NASDAQ:INZY)
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