Inozyme Pharma, Inc. (Nasdaq: INZY), 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 first quarter ended March
31, 2023, and provided business highlights.
“We are continuing to advance INZ-701 and look forward to
initiating a pivotal trial for pediatric patients with ENPP1
Deficiency in the third quarter of this year, subject to receipt of
regulatory approval. These patients represent a population with
high unmet medical need and they and their caregivers eagerly
anticipate the planned clinical trial," said Douglas A. Treco,
Ph.D., chief executive officer and chairman of the Company’s board
of directors. “Our team is executing well, and we continue to
collect clinical data from our ongoing trials in adults with ENPP1
Deficiency and ABCC6 Deficiency, which will provide important
insights into the clinical effects of INZ-701.”
Business Highlights
- Regulatory Update. The Company has reached
agreement with the European Medicines Agency (EMA) on a Pediatric
Investigational Plan (PIP) for the study of INZ-701 in pediatric
patients with ENPP1 Deficiency.
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ENPP1 Deficiency. The Company dosed the first adult
patient with ENPP1 Deficiency in an additional dose cohort designed
to investigate the potential for once-weekly dosing of INZ-701 in
the ongoing trial.
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ABCC6 Deficiency (pseudoxanthoma elasticum or PXE). The
Company announced the first self-administration of INZ-701 in the
open-label Phase 2 portion of the ongoing clinical trial.
- Medical Conference Presentations. Yves
Sabbagh, Ph.D., the Company’s senior vice president and chief
scientific officer, presented the recently announced topline data
from the ongoing Phase 1/2 clinical trial of INZ-701 in patients
with ENPP1 Deficiency at the European Calcified Tissue Society
(ECTS) on April 17, 2023. Kurt Gunter, M.D., the Company’s senior
vice president and chief medical officer, will present these data
at the European Congress of Endocrinology (ECE 2023) scheduled May
13-16.
Anticipated Milestones
- ENPP1 Deficiency
- Initiation of ENERGY-1 – Phase 1b clinical trial to evaluate
the safety, tolerability, pharmacokinetic and pharmacodynamic
profile of INZ-701 in infants – Q2 2023
- Initiate scientific advice process with EMA regarding our
comprehensive development plan covering all age groups– Q2
2023
- Interim data from the Phase 2 portion of the ongoing trial in
adults – Q3 2023
- Initiation of pivotal trial in pediatric patients, subject to
regulatory approval – Q3 2023
- ABCC6 Deficiency
- Interim data from the Phase 2 portion of the ongoing trial in
adults – Q4 2023
First Quarter 2023 Financial Results
- Cash Position and Financial Guidance – Cash,
cash equivalents, and short-term investments were $130.9 million as
of March 31, 2023. Based on its current plans, the Company
anticipates its cash, cash equivalents, and short-term investments
as of March 31, 2023 will enable the Company to fund cash flow
requirements into the fourth quarter of 2024.
- Research and Development (R&D) Expenses –
R&D expenses of $11.9 million for the quarter ended March 31,
2023 were relatively consistent with R&D expenses of $11.8
million for the prior-year period.
- General and Administrative (G&A) Expenses
– G&A expenses were $6.5 million for the quarter ended March
31, 2023, compared to $5.0 million for the prior-year period. The
increase was primarily related to the expenses recorded for the
transition and separation agreement entered into with our former
chief executive officer.
- Net Loss – Net loss was $17.4 million, or
$0.40 loss per share, for the quarter ended March 31, 2023,
compared to $16.9 million, or $0.71 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 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
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 with three patients per
dose cohort. Patients received a single dose and then began twice
weekly dosing one week later. Doses were selected based on
preclinical studies and PK/PD modeling. The Phase 1 dose-escalation
portion of the trial seeks to identify a safe, tolerable dose that
increases PPi levels, and that can be used for further clinical
development. The open-label Phase 2 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 ectopic calcification, skeletal, vascular, and
physical function, patient-reported outcomes, and exploratory
biomarkers.
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 is designed to 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 with three patients per
dose cohort. Patients received a single dose and then began twice
weekly dosing one week later. Doses were selected based on
preclinical studies and PK/PD modeling. The Phase 1 dose-escalation
portion of the trial was designed to identify a safe, tolerable
dose for further development that increases PPi levels. The
open-label Phase 2 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 include evaluations of ectopic
calcification, vascular and retinal function, patient- reported
outcomes and exploratory biomarkers.
About INZ-701
INZ-701 is a clinical-stage 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 Phase 1/2 clinical trials for
the treatment of adult 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 Phase 1/2 clinical trials
for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
For more information, please visit www.inozyme.com and follow us
on LinkedIn, 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 of our planned
clinical trials, the availability of data from clinical trials,
timing of planned regulatory meetings, the potential benefits of
INZ-701, and the sufficiency of the Company's cash resources. 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 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, 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)(in
thousands) |
|
|
March 31, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and short-term investments |
$ |
130,930 |
|
|
$ |
127,866 |
|
Total assets |
$ |
141,796 |
|
|
$ |
139,195 |
|
Total liabilities |
$ |
38,449 |
|
|
$ |
20,801 |
|
Additional
paid-in-capital |
$ |
335,544 |
|
|
$ |
333,356 |
|
Accumulated deficit |
$ |
(232,165 |
) |
|
$ |
(214,761 |
) |
Total stockholders'
equity |
$ |
103,347 |
|
|
$ |
118,394 |
|
|
Condensed Consolidated Statements of Operations and
Comprehensive
Loss (Unaudited)(in
thousands, except share and per share data) |
|
|
|
Three Months Ended March 31, |
|
|
|
2023 |
|
|
|
2022 |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
$ |
11,857 |
|
|
$ |
11,814 |
|
General and administrative |
|
|
6,512 |
|
|
|
5,025 |
|
Total operating expenses |
|
|
18,369 |
|
|
|
16,839 |
|
Loss from operations |
|
|
(18,369 |
) |
|
|
(16,839 |
) |
Other income (expense): |
|
|
|
|
Interest income, net |
|
|
999 |
|
|
|
60 |
|
Other expense, net |
|
|
(34 |
) |
|
|
(105 |
) |
Other income (expense),
net |
|
|
965 |
|
|
|
(45 |
) |
Net loss |
|
$ |
(17,404 |
) |
|
$ |
(16,884 |
) |
Other comprehensive income
(loss): |
|
|
|
|
Unrealized gains (losses) on available-for-sale securities |
|
|
150 |
|
|
|
(132 |
) |
Foreign currency translation adjustment |
|
|
19 |
|
|
|
(15 |
) |
Total other comprehensive
income (loss) |
|
|
169 |
|
|
|
(147 |
) |
Comprehensive
loss |
|
$ |
(17,235 |
) |
|
$ |
(17,031 |
) |
Net loss attributable to
common stockholders—basic and diluted |
|
$ |
(17,404 |
) |
|
$ |
(16,884 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.40 |
) |
|
$ |
(0.71 |
) |
Weighted-average common shares
outstanding—basic and diluted |
|
|
43,720,578 |
|
|
|
23,686,351 |
|
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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