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 a regulatory update for its global development
strategy of INZ-701 for the treatment of ENPP1 Deficiency following
recent meetings with the United States (U.S.) Food and Drug
Administration (FDA) and the Paediatric Committee (PDCO) of the
European Medicines Agency (EMA).
“We are pleased to have finalized our pediatric pivotal trial
design with PPi, a well-established natural inhibitor of
mineralization, as a primary endpoint in the U.S. and a co-primary
endpoint in the EU. We have already observed that INZ-701
meaningfully increased PPi levels in our ongoing trial of INZ-701
in adults with ENPP1 Deficiency and, based on our discussions with
regulators in the U.S. and EU, we believe we have a clear path
forward in our clinical development program for the treatment of
ENPP1 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme
Pharma.
ENERGY-3 Trial Design – Planned Pivotal Trial in
Pediatric Patients with ENPP1 Deficiency
The Company plans to initiate the ENERGY-3 pivotal trial, a
multicenter, randomized, open label trial in pediatric patients
with ENPP1 Deficiency in October 2023. The ENERGY-3 trial is
expected to enroll up to 33 patients between the ages of one and
less than 13 years across multiple sites globally and is designed
primarily to assess the efficacy and safety of INZ-701 in pediatric
patients with ENPP1 Deficiency. Enrollment criteria for the trial
include a confirmed genetic diagnosis of ENPP1 Deficiency,
radiographic evidence of skeletal abnormalities and low plasma PPi.
Patients will be randomized in a 2:1 ratio to an INZ-701 arm or a
control arm (conventional therapy, i.e., oral phosphate and active
vitamin D) for 52 weeks, followed by an open label extension period
during which all patients may receive INZ-701. INZ-701 will be
administered at a 2.4 mg/kg once weekly dose via subcutaneous (SC)
injection.
ENERGY-3 is a single, multicenter, clinical trial with
differences in the statistical treatment of endpoints, based on
guidance from the FDA and PDCO, as follows:
U.S. |
EU |
Primary Endpoint – Change in plasma PPi from
baseline |
Co-Primary Endpoints – Change in plasma PPi from
baseline and Radiographic Global Impression of Change (RGI-C) score
(p<0.2) |
Secondary Endpoints - RGI-C score, Rickets
Severity Score (RSS), Growth Z-score and pharmacokinetics (PK) |
Secondary Endpoints – RSS, Growth Z-score and
PK |
Based on recommendations from the FDA, the primary endpoint of
plasma PPi should be supported by consistent trends in appropriate
secondary endpoints. Based on the agreed Paediatric Investigational
Plan (PIP) with PDCO, plasma PPi and RGI-C are co-primary
endpoints, with a relaxed p-value of <0.2 for RGI-C.
Planned Pivotal Trials in Infants and Adolescent/Adult
Patients with ENPP1 Deficiency
The Company plans to conduct the ENERGY-2 pivotal trial, an open
label, single arm trial in infants with ENPP1 Deficiency, based on
the Paediatric Investigational Plan (PIP) agreed upon by PDCO. The
trial is expected to be initiated outside of the U.S. The trial’s
co-primary endpoints will be change in plasma PPi from baseline and
survival. The trial is expected to enroll up to 12 infants between
birth and up to 12 months of age. Primary endpoint data from this
trial will be compared to a natural history control group with
patients matched on covariates associated with mortality.
Discussions are ongoing with the FDA regarding the design of a
potential pivotal trial of INZ-701 in infants with ENPP1 Deficiency
in the U.S.
Pending regulatory discussions and appropriate financial
resources, the Company also plans to conduct the ENERGY-4 pivotal
trial, a multicenter, randomized, controlled trial in adolescents
and adults with ENPP1 Deficiency. In the U.S., the trial’s sole
primary endpoint is expected to be change in plasma PPi from
baseline, supported by trends in appropriate secondary endpoints,
and in the EU, the trial’s co-primary endpoints are expected to be
change in plasma PPi from baseline and bone mineral
content/density. Subject to regulatory review, the trial is
expected to enroll up to 30 patients 13 years and older, and
patients will be randomized in a 2:1 ratio to an INZ-701 arm or a
control arm (conventional therapy, i.e., oral phosphate and active
vitamin D).
Basis for Planned Marketing Applications
Based on regulatory feedback from the FDA and EMA, positive data
from the ongoing and planned clinical trials of INZ-701 in patients
with ENPP1 Deficiency, including comprehensive data demonstrating
clinical impact of plasma PPi, could provide the basis for the
Company’s submission of marketing applications in both the U.S. and
EU. These data will include final results from the Company’s
ongoing Phase 1/2 trial in adult patients with ENPP1 Deficiency,
available results from the Company’s ongoing ENERGY-1 trial, a
Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency,
available results from the planned pivotal ENERGY-2 trial in
infants to be initiated ex-U.S., and final results from the planned
pivotal ENERGY-3 trial in pediatric patients.
If these marketing applications are approved, the Company
expects to commercially launch INZ-701 for infant and pediatric
patients as early as the second half of 2026. Data from the planned
ENERGY-4 trial in adolescent and adult patients with ENPP1
Deficiency may provide a basis for a supplemental marketing
application.
Anticipated Milestones for ENPP1 Deficiency
Program
- Interim data from Cohorts 1-3 in the Phase 2 portion of the
ongoing Phase 1/2 trial in adults – September 2023
- Initiation of ENERGY-3 trial, a pivotal trial in pediatric
patients – October 2023
- Topline data from Cohorts 1-3 in the Phase 2 portion of the
ongoing Phase 1/2 trial in adults – Q1 2024
- Initiation of ENERGY-2 trial, a pivotal trial in infants, ex
U.S. - Q2 2024
- Interim data from ENERGY-1 trial, a Phase 1b trial in infants –
2H 2024
- Topline data from ENERGY-3 trial, a pivotal trial in pediatric
patients – Mid-2025
Cash Runway Guidance
The Company expects to report cash, cash equivalents, and
short-term investments of approximately $140.2 million as of June
30, 2023. The estimated cash, cash equivalents, and short-term
investments amount is preliminary and unaudited, represents
management’s estimate as of the date of this press release, is
subject to completion of the Company’s financial closing procedures
for the quarter ended June 30, 2023 and does not present all
necessary information for a complete understanding of the Company’s
financial condition as of June 30, 2023, or the Company’s results
of operations for the quarter ended June 30, 2023. The actual
financial results may differ materially from the preliminary
estimated financial information.
Based on its current plans, the Company anticipates its cash,
cash equivalents, and short-term investments as of June 30, 2023,
will enable the Company to fund cash flow requirements into the
first quarter of 2025.
Conference Call
Inozyme will host a conference call and webcast to discuss its
global development strategy for INZ-701 in patients with ENPP1
Deficiency today, July 26, 2023 at 8am ET. The live webcast will be
accessible through the Investor Relations section of Inozyme’s
website under News & Events. To access the live call by phone,
dial 1-877-270-2148 (domestic) or 1-412-902-6510 (international)
and ask to be connected to 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. The estimated genetic prevalence of ENPP1
Deficiency is approximately 1 in 64,000 pregnancies worldwide.
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
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 clinical trials for the
treatment of ENPP1 Deficiency and ABCC6 Deficiency.
For more information, please
visit www.inozyme.com and follow us
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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 timing and
design of our clinical trials, the potential benefits of INZ-701,
the timing and contents of our planned global development strategy,
the availability and timing of clinical trial data, planned
regulatory filings and the basis for such filings, the timing of
the planned commercial launch of INZ-701, if approved, and the
period over which we believe that our existing cash, cash
equivalents and short term investments will be sufficient to fund
our cash flow requirements. 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, EMA, 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; obtain
clinically meaningful results with respect to novel endpoints;
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 (SEC), as well as discussions of
potential risks, uncertainties, and other important factors, in the
Company's most recent filings with the SEC. 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
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