Inozyme Pharma, Inc. (Nasdaq: INZY) (“the
Company” or “Inozyme”), a clinical-stage rare disease
biopharmaceutical company developing novel therapeutics for the
treatment of pathologic mineralization and intimal proliferation,
today announced that the Company will present recently announced
topline data from the Company’s ongoing Phase 1/2 clinical trials
of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency
(manifesting as pseudoxanthoma elasticum, or PXE), during oral
presentations at two upcoming medical conferences.
Details regarding the presentations are as follows:
The European Calcified Tissue Society Congress (ECTS) 2024 being
held May 25-28, 2024 in Marseille, France.
Title: Impact of
INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical
Outcomes in Adults with ENPP1 Deficiency – Results from 48-week
Phase 1/2 Open Label StudyPresentation Number:
COP05Session Title: Concurrent Oral Presentations
1: Rare Bone DiseasesDate: Sunday, May 26,
2024Time: 9:45 – 9:55 CEST / 3:45am – 3:55am
ETLocation: CallelonguePresenter:
Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific
OfficerTitle: Safety and Exploratory Efficacy of
INZ-701 in Adults with ABCC6 Deficiency Manifesting as
Pseudoxanthoma Elasticum – Results from 48-week Phase 1/2 Open
Label Study Presentation Number:
P237Session Title: Concurrent Oral Poster
Presentations 1: Clinical/Public HealthDate:
Sunday, May 26, 2024Time: 18:15 – 18:20 CEST/
12:15pm – 12:20pm ETLocation:
CallelonguePresenter: Yves Sabbagh, Ph.D., Senior
Vice President and Chief Scientific Officer
The Endocrine Society’s Annual Meeting (ENDO) 2024 being held
June 1-4, 2024, in Boston, Massachusetts.
Title: Impact of
INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical
Outcomes in Adults with ENPP1 Deficiency-Results from 48-week Phase
1/2 Open Label StudyAbstract Number:
7217Session Title: Oral Abstract and
Rapid-FireDate: Monday, June 3,
2024Time: 2:45pm – 3:00pm
ETLocation: Boston Convention & Exhibition
Center (BCEC): 258ABC – BCECPresenter: Kurt
Gunter, M.D., Senior Vice President and Chief Medical Officer
About ENPP1 Deficiency
ENPP1 Deficiency is a progressively debilitating condition of
the vasculature, soft tissue, and skeleton with a prevalence of
approximately 1 in 64,000 pregnancies worldwide. Although ENPP1
Deficiency was initially described in patients with biallelic ENPP1
Deficiency (homozygous or compound heterozygous mutations), many
patients with monoallelic ENPP1 Deficiency (heterozygous mutations)
have clinical symptoms, potentially increasing the worldwide
prevalence. Individuals who present in utero or in infancy are
typically diagnosed with generalized arterial calcification of
infancy (GACI Type 1) and approximately 50% of infants 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 progressively debilitating condition of
the vasculature and soft tissue that is estimated to affect
approximately 1 in 25,000 to 1 in 50,000 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.
Pediatric patients who survive the first year of life may develop
neurological disease, including stroke, and cardiovascular disease
secondary to ongoing vascular calcification and stenosis. In older
individuals, ABCC6 Deficiency presents as pseudoxanthoma elasticum
(PXE), which is characterized by pathologic 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 (ERT) in development for the treatment of rare
disorders of the vasculature, soft tissue, and skeleton. INZ-701
metabolizes adenosine triphosphate (ATP) to generate PPi, a natural
inhibitor of mineralization, and AMP, which can be processed to
phosphate and adenosine, the latter being a natural inhibitor of
intimal proliferation. In preclinical studies, the experimental
therapy has shown potential to prevent pathologic mineralization
and intimal proliferation, which can drive morbidity and mortality
in devastating disorders such as, ENPP1 Deficiency, ABCC6
Deficiency, and calciphylaxis. Clinical data to date have
demonstrated that INZ-701 was generally well tolerated, exhibited a
favorable safety profile, and meaningfully increased PPi levels in
multiple clinical trials.
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 development for
the treatment of ENPP1 Deficiency, ABCC6 Deficiency and
calciphylaxis.
For more information, please
visit https://www.inozyme.com/ or follow
Inozyme on LinkedIn, X,
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, Senior 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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