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 reported financial results for the third quarter ended
September 30, 2023 and provided business highlights.
“We are excited to advance INZ-701 into the ENERGY-3 pivotal
trial in pediatric patients with ENPP1 Deficiency. Our ongoing
trial of INZ-701 in adults with ENPP1 Deficiency showed early signs
of clinical impact and supports the potential of treating children
with this condition,” said Douglas A. Treco, Ph.D., CEO of Inozyme
Pharma. “We continue to expand our pipeline and therapeutic focus
with INZ-701,” Dr. Treco added. “In addition to planning a Phase 3
trial in patients with ABCC6 Deficiency, recent data presented by
our collaborators at the American Society of Nephrology Kidney Week
2023 support our plans to evaluate INZ-701 in patients with
calciphylaxis, a rare but often fatal complication of hemodialysis
associated with end-stage kidney disease.”
Recent Highlights
- ENERGY-3 Pivotal Trial of INZ-701 in Pediatric Patients
with ENPP1 Deficiency. Patient recruitment is underway,
and the Company remains on track to report topline data in
mid-2025.
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ENPP1 Deficiency. The Company announced positive interim
safety, pharmacokinetic (PK), pharmacodynamic (PD), and exploratory
efficacy data from the ongoing trial. Exploratory efficacy data
reported suggested clinical benefit for ENPP1 Deficiency, including
improvement in key biomarkers, patient-reported outcomes (PROs),
and functional outcomes. Dosing is ongoing in the Phase 2 portion
of the trial, and the Company is on track to report topline
clinical data from the first three cohorts through 48 weeks in the
first quarter of 2024.
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ABCC6 Deficiency (pseudoxanthoma elasticum, or PXE). The
Company announced positive interim safety, PK, PD, and exploratory
efficacy data from the ongoing trial. Improvements in the Global
Impression of Change (GIC), a PRO, were observed in all three dose
cohorts. Dosing is ongoing in the Phase 2 portion of the trial, and
the Company is on track to report topline clinical data through 48
weeks in the first quarter of 2024.
- Genomics England Generation Study. The Company
highlighted the inclusion of Generalized Arterial Calcification of
Infancy (GACI), which is caused by mutations in the ENPP1 or ABCC6
genes, in the Genomics England’s Generation Study. The Generation
Study is scheduled to begin in late 2023, with the goal of
sequencing the genomes of more than 100,000 infants and paving the
way for potential widespread implementation of whole-genome
sequencing in newborn screening.
- Medical Conference Presentations. The Company
announced the presentation of three posters at the American Society
for Bone and Mineral Research (ASBMR) 2023 Annual Meeting and an
oral presentation at the American Society for Nephrology (ASN)
Kidney Week 2023.
Anticipated Milestones
- ENPP1 Deficiency
- Topline data from first three cohorts of ongoing Phase 1/2
trial in adults through 48 weeks – Q1 2024
- Initiation of the ENERGY-2 pivotal trial in infants, Ex-U.S. –
Q2 2024
- Interim data from the ENERGY-1 Phase 1b trial in infants – 2H
2024
- Topline data from the ENERGY-3 pivotal trial in pediatric
patients – Mid-2025
- ABCC6 Deficiency
- Topline data from ongoing Phase 1/2 trial in adults through 48
weeks– Q1 2024
- Initiation of Phase 3 clinical trial in adults, subject to
regulatory review and sufficient funding – Q4 2024
- Calciphylaxis
- Initiation of SEAPORT-1 Phase 1 trial designed to assess
safety, tolerability, PK, and PD of INZ-701 in patients with
end-stage kidney disease (ESKD) receiving hemodialysis – 1H
2024
- Interim data from SEAPORT-1 Phase 1 trial in patients with ESKD
receiving hemodialysis – Q4 2024
Third Quarter 2023 Financial Results
- Cash Position and Financial Guidance. Cash,
cash equivalents, and short-term investments were $192.4 million as
of September 30, 2023. Based on its current plans, the Company
anticipates its cash, cash equivalents, and short-term investments
as of September 30, 2023 will enable the Company to fund cash flow
requirements into Q4 2025.
- Research and Development (R&D) Expenses.
R&D Expenses were $13.3 million for the quarter ended September
30, 2023, compared to $12.2 million for the prior-year period.
- General and Administrative (G&A) Expenses.
G&A expenses were $4.7 million for each of the quarters ended
September 30, 2023 and 2022.
- Net Loss. Net loss was $16.6 million, or $0.29
loss per share, for the quarter ended September 30, 2023, compared
to $16.4 million, or $0.38 loss per share, for the prior-year
period.
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.
ENERGY-3 Pivotal Trial of INZ-701 in Pediatric Patients
with ENPP1 Deficiency
The ENERGY-3 pivotal trial is a multicenter, randomized,
open-label trial in pediatric patients with ENPP1 Deficiency. The
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 pyrophosphate (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 injection. Based on recommendations
from the Food and Drug Administration (FDA), the primary endpoint
of plasma PPi should be supported by consistent trends in
appropriate secondary endpoints such as Radiographic Global
Impression of Change (RGI-C), Rickets Severity Score (RSS), and
Growth Z-score. Based on the agreed Paediatric Investigational Plan
(PIP) with the Paediatric Committee of the European Medicines
Agency (PDCO), plasma PPi and RGI-C are co-primary endpoints, with
a relaxed p-value of <0.2 for RGI-C.
Phase 1/2 Clinical Trial of INZ-701 in Adults with ENPP1
Deficiency
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 plasma
pyrophosphate (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.
Phase 1/2 Clinical Trial of INZ-701 in Adults with ABCC6
Deficiency
The ongoing Phase 1/2 open-label clinical trial enrolled 10
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 pyrophosphate (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 that increases
PPi levels for further development. 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 Calciphylaxis
Calciphylaxis is a serious and rare disorder with a high
mortality rate that mostly affects patients with end-stage kidney
disease (ESKD). The disease is associated with low levels of plasma
pyrophosphate (PPi) and is characterized by pathologic
mineralization and intimal proliferation of the vasculature in the
skin and fatty tissue, leading to blood clots, painful skin ulcers,
infections, and death. Patients with calciphylaxis have a reported
one-year survival rate of approximately 50%. The estimated
incidence of calciphylaxis is at least 1,800 new patients per year
in the United States, and there are currently no approved
therapies.
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
multiple 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 multiple 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 timing and
design of our clinical trials, the availability and timing of data
from clinical trials, the potential benefits of INZ-701, 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 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.
Condensed Consolidated Balance Sheet Data
(Unaudited) |
|
|
|
|
|
September 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and investments |
$ |
192,442 |
|
|
$ |
127,866 |
|
Total
assets |
$ |
205,702 |
|
|
$ |
139,195 |
|
Total
liabilities |
$ |
45,489 |
|
|
$ |
20,801 |
|
Additional paid-in-capital |
$ |
424,671 |
|
|
$ |
333,356 |
|
Accumulated deficit |
$ |
(264,386 |
) |
|
$ |
(214,761 |
) |
Total
stockholders' equity |
$ |
160,213 |
|
|
$ |
118,394 |
|
Condensed Consolidated Statements of Operations and
Comprehensive Loss(Unaudited) |
|
|
|
|
Three Months Ended September 30, |
Nine Months Ended September 30, |
|
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
$ |
13,341 |
|
|
$ |
12,191 |
|
|
$ |
36,864 |
|
|
$ |
34,012 |
|
General and administrative |
|
|
|
4,733 |
|
|
|
4,721 |
|
|
|
15,973 |
|
|
|
15,130 |
|
Total
operating expenses |
|
|
|
18,074 |
|
|
|
16,912 |
|
|
|
52,837 |
|
|
|
49,142 |
|
Loss
from operations |
|
|
|
(18,074 |
) |
|
|
(16,912 |
) |
|
|
(52,837 |
) |
|
|
(49,142 |
) |
Other
income (expense): |
|
|
|
|
|
|
|
|
|
Interest income, net |
|
|
|
1,416 |
|
|
|
737 |
|
|
|
3,254 |
|
|
|
1,118 |
|
Other expense, net |
|
|
|
20 |
|
|
|
(197 |
) |
|
|
(42 |
) |
|
|
(493 |
) |
Other
income, net |
|
|
|
1,436 |
|
|
|
540 |
|
|
|
3,212 |
|
|
|
625 |
|
Net loss |
|
|
$ |
(16,638 |
) |
|
$ |
(16,372 |
) |
|
$ |
(49,625 |
) |
|
$ |
(48,517 |
) |
Other
comprehensive income (loss): |
|
|
|
|
|
|
|
|
|
Unrealized gains (losses) on available-for-sale securities |
|
|
|
53 |
|
|
|
(60 |
) |
|
|
279 |
|
|
|
(417 |
) |
Foreign currency translation adjustment |
|
|
|
(182 |
) |
|
|
(20 |
) |
|
|
(152 |
) |
|
|
(78 |
) |
Total
other comprehensive income (loss) |
|
|
|
(129 |
) |
|
|
(80 |
) |
|
|
127 |
|
|
|
(495 |
) |
Comprehensive loss |
|
|
$ |
(16,767 |
) |
|
$ |
(16,452 |
) |
|
$ |
(49,498 |
) |
|
$ |
(49,012 |
) |
Net loss
attributable to common stockholders—basic and diluted |
|
|
$ |
(16,638 |
) |
|
$ |
(16,372 |
) |
|
$ |
(49,625 |
) |
|
$ |
(48,517 |
) |
Net loss
per share attributable to common stockholders—basic and
diluted |
|
|
$ |
(0.29 |
) |
|
$ |
(0.38 |
) |
|
$ |
(1.02 |
) |
|
$ |
(1.36 |
) |
Weighted-average common shares and pre-funded warrants
outstanding—basic and diluted |
|
|
|
56,758,395 |
|
|
|
43,657,718 |
|
|
|
48,494,175 |
|
|
|
35,755,695 |
|
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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