Inozyme Pharma, Inc. (Nasdaq: INZY) (“the
Company” or “Inozyme”), a clinical-stage rare disease
biopharmaceutical company developing innovative therapeutics for
rare diseases that affect bone health and blood vessel function,
today reported financial results for the second quarter ended June
30, 2024, and provided business highlights.
“We are making strides in advancing INZ-701 through our clinical
programs, with several significant milestones expected by
year-end,” said Douglas A. Treco, Ph.D., Chief Executive Officer
and Chairman of Inozyme’s Board of Directors. “Notably, we expect
to provide an update on our planned pathway to approval for the
ABCC6 Deficiency program and present the first look at clinical
data from our ongoing infant ENPP1 Deficiency trial, along with
interim data from the SEAPORT 1 trial in calciphylaxis. Our
unwavering dedication to developing transformative treatments for
patients with rare diseases linked to the PPi-Adenosine Pathway
drives us forward with a profound sense of urgency.”
Recent Highlights
Pipeline
- Publication of Preclinical Data in
Cells. In July 2024, the Company published
preclinical data supporting the potential of INZ-701 to treat a
broad range of diseases mediated by the PPi-Adenosine Pathway,
which regulates mineralization and intimal proliferation (the
overgrowth of smooth muscle cells inside blood vessels). The
article titled, “Inhibition of Vascular Smooth Muscle Cell
Proliferation by ENPP1: The Role of CD73 and the Adenosine
Signaling Axis”, was published in the journal Cells.
ENPP1 Deficiency
- Presentation and Symposium at the 11th International
Conference on Children’s Bone Health (ICCBH). In June
2024, results from a radiographic study describing skeletal
features of pediatric patients with ENPP1 Deficiency were presented
at the 11th International Conference on Children’s Bone Health in
Salzburg, Austria. In addition, the Company hosted a sponsored
symposium titled, “Recognizing ENPP1 Deficiency - An overlooked
cause of hypophosphatemic rickets”.
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ENPP1 Deficiency. In April 2024, the Company announced
positive topline data indicating that the previously-reported
favorable safety, immunogenicity, and clinical outcome data were
maintained through 48 weeks in Cohorts 1-3. Data from Cohort 4
support once weekly dosing in ongoing and future clinical trials.
Data were subsequently featured at the European Calcified Tissue
Society Congress (ECTS) 2024 in May in Marseille, France and the
Endocrine Society’s Annual Meeting (ENDO) 2024 in June in
Boston.
ABCC6 Deficiency
- FDA Fast Track Designation Granted for INZ-701 in ABCC6
Deficiency. In July 2024, the Company announced that the
U.S. Food and Drug Administration (FDA) has granted Fast Track
designation to INZ-701 for the treatment of ABCC6 Deficiency
(manifesting as pseudoxanthoma elasticum, or PXE).
- Phase 1/2 Clinical Trial of INZ-701 in Adults with
ABCC6 Deficiency. In April 2024, the Company announced
positive topline safety and immunogenicity data, with clinical
improvements in vascular pathology, visual function, and patient
reported outcomes (PROs). Data were subsequently featured at the
ECTS 2024. Subject to regulatory review and sufficient funding, the
Company expects to initiate a pivotal trial in pediatric patients
with ABCC6 Deficiency in Q1 2025.
- Natural History Studies and Development Plans for
INZ-701 in Pediatric ABCC6 Deficiency. In April 2024, the
Company reported initial findings from natural history studies
which indicated a substantial disease burden among pediatric
patients with ABCC6 Deficiency, manifesting as a high incidence of
major clinical events, notably stroke, severe neurological disease,
and severe cardiovascular disease, occurring early in life.
Anticipated Milestones
- ENPP1 Deficiency
- Complete enrollment of ENERGY 3 pivotal trial in pediatric
patients – Q3 2024
- Initiation of the ENERGY 2 pivotal trial in infants, Ex-U.S. –
Q4 2024
- Interim data from the ENERGY 1 Phase 1b trial in infants – Q4
2024
- Topline data from the ENERGY 3 pivotal trial in pediatric
patients – 2H 2025
- ABCC6 Deficiency
- Initiation of pivotal clinical trial in pediatric patients,
subject to regulatory review and sufficient funding – Q1 2025
- Calciphylaxis
- Interim data from SEAPORT 1 Phase 1 trial in patients with
end-stage kidney disease (ESKD) receiving hemodialysis – Q4
2024
Second Quarter 2024 Financial Results
- Cash Position and Financial Guidance. Cash,
cash equivalents, and short-term investments were $144.5 million as
of June 30, 2024. Based on its current plans, the Company
anticipates its cash, cash equivalents, and short-term investments
as of June 30, 2024, will enable the Company to fund cash flow
requirements into Q4 2025.
- Research and Development (R&D) Expenses.
R&D expenses were $21.8 million for the quarter ended June 30,
2024, compared to $11.7 million for the prior-year period.
- General Administrative (G&A) Expenses.
G&A expenses were $5.9 million for the quarter ended June 30,
2024, compared to $4.7 million for the prior-year period.
- Net Loss. Net loss was $27.0 million, or $0.44
loss per share, for the quarter ended June 30, 2024, compared to
$15.6 million or $0.35 loss per share for the prior-year
period.
About ENPP1 DeficiencyENPP1 Deficiency is a
serious and progressive rare disease that affects blood vessels,
soft tissues, and bones. Individuals who present in utero or in
infancy are typically diagnosed with generalized arterial
calcification of infancy (GACI Type 1), with about 50% of these
infants not surviving beyond six months. Children with this
condition typically develop rickets, specifically
autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while
adolescents and adults may develop osteomalacia, or softened bones.
ARHR2 and osteomalacia cause pain and difficulty with movement.
Additionally, patients may experience hearing loss, calcification
in arteries and joints, and heart problems.
Biallelic ENPP1 Deficiency affects approximately 1 in 64,000
pregnancies worldwide. Initially, it was believed to only impact
individuals with two copies of the mutated gene. However, many
individuals with just one copy of the mutated gene (monoallelic
ENPP1 Deficiency) also exhibit severe symptoms. This suggests that
the worldwide prevalence of ENPP1 Deficiency may be much higher
than current estimates, which are based solely on biallelic cases.
Currently, there are no approved therapies for ENPP1
Deficiency.
About ABCC6 DeficiencyABCC6 Deficiency is a
progressive and debilitating rare disease that affects blood
vessels and soft tissues. Infants with ABCC6 Deficiency are
diagnosed with generalized arterial calcification of infancy (GACI
Type 2), which is similar to GACI Type 1, the infant form of ENPP1
Deficiency. Pediatric patients who survive beyond the first year of
life may develop neurological disease, including strokes, and
cardiovascular diseases due to ongoing vascular calcification and
stenosis. In older individuals, ABCC6 Deficiency manifests as
pseudoxanthoma elasticum (PXE), characterized by abnormal
mineralization in blood vessels and soft tissues, affecting the
skin, visual function, and vascular system.
Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to
1 in 50,000 individuals worldwide. Initially, it was believed to
only impact individuals with two copies of the mutated gene.
However, many people with just one copy of the mutated gene
(monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This
suggests that the worldwide prevalence of ABCC6 Deficiency may be
much higher than current estimates, which are based solely on
biallelic cases. Currently, there are no approved therapies for
ABCC6 Deficiency.
About CalciphylaxisCalciphylaxis (also known as
calcific uremic arteriolopathy, CUA) is a 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
inorganic pyrophosphate (PPi) and is characterized by pathologic
mineralization (i.e., calcification) and intimal proliferation (the
overgrowth of smooth muscle cells inside blood vessels) of the
vasculature in the skin and fatty tissue leading to poor blood
flow, blood clots, painful skin ulcers, serious infections, and
death. Patients with calciphylaxis have a reported one-year
survival rate of approximately 50%. The estimated incidence of
calciphylaxis is approximately 3.5 per 1,000 patients with ESKD
with approximately 5,000 new patients presenting annually across
major addressable markets. Currently, there are no approved
therapies for calciphylaxis.
About Inozyme PharmaInozyme Pharma is a
pioneering clinical-stage biopharmaceutical company dedicated to
developing innovative therapeutics for rare diseases that affect
bone health and blood vessel function. We are experts in the
PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic
pyrophosphate (PPi), which regulates mineralization, and adenosine,
which controls intimal proliferation (the overgrowth of smooth
muscle cells inside blood vessels). Disruptions in this pathway
impact the levels of these molecules, leading to severe
musculoskeletal, cardiovascular, and neurological conditions,
including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and
ossification of the posterior longitudinal ligament (OPLL).
Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein
enzyme replacement therapy (ERT) designed to increase PPi and
adenosine, enabling the potential treatment of multiple diseases
caused by deficiencies in these molecules. It is currently in
clinical development for the treatment of ENPP1 Deficiency, ABCC6
Deficiency, and calciphylaxis. By targeting the PPi-Adenosine
Pathway, INZ-701 aims to correct pathological mineralization and
intimal proliferation, addressing the significant morbidity and
mortality in these devastating diseases.
For more information, please
visit https://www.inozyme.com/ or follow Inozyme
on LinkedIn, X,
and Facebook.
Cautionary Note Regarding Forward-Looking
StatementsStatements 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 initiation,
timing, and design of our planned clinical trials, availability of
data from clinical trials, the potential benefits of INZ-701, our
regulatory strategy, including our planned pathway to approval for
the ABCC6 Deficiency program, 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, ABCC6 Deficiency, and
calciphylaxis; 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) |
|
|
June 30,2024 |
|
December 31,2023 |
Cash, cash equivalents and investments |
$ |
144,523 |
|
|
$ |
188,589 |
|
Total Assets |
$ |
155,712 |
|
|
$ |
200,847 |
|
Total Liabilities |
$ |
61,292 |
|
|
$ |
60,368 |
|
Additional
paid-in-capital |
$ |
430,827 |
|
|
$ |
426,362 |
|
Accumulated deficit |
$ |
(336,310 |
) |
|
$ |
(285,930 |
) |
Total stockholders'
equity |
$ |
94,420 |
|
|
$ |
140,479 |
|
|
Condensed Consolidated Statements of Operations and
Comprehensive Loss(Unaudited) |
|
|
|
Three Months Ended June 30, |
|
|
|
2024 |
|
|
|
2023 |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
$ |
21,758 |
|
|
$ |
11,666 |
|
General and administrative |
|
|
5,907 |
|
|
|
4,728 |
|
Total operating expenses |
|
|
27,665 |
|
|
|
16,394 |
|
Loss from operations |
|
|
(27,665 |
) |
|
|
(16,394 |
) |
Other income (expense): |
|
|
— |
|
|
|
— |
|
Interest income |
|
|
2,030 |
|
|
|
1,610 |
|
Interest expense |
|
|
(1,395 |
) |
|
|
(771 |
) |
Other expenses |
|
|
(3 |
) |
|
|
(28 |
) |
Other income (expense),
net |
|
|
632 |
|
|
|
811 |
|
Net loss |
|
$ |
(27,033 |
) |
|
$ |
(15,583 |
) |
Other comprehensive income
(loss): |
|
|
|
|
Unrealized gains (losses) on available-for-sale securities |
|
|
3 |
|
|
|
76 |
|
Foreign currency translation adjustment |
|
|
(1 |
) |
|
|
11 |
|
Total other comprehensive
income (loss) |
|
|
2 |
|
|
|
87 |
|
Comprehensive
loss |
|
$ |
(27,031 |
) |
|
$ |
(15,496 |
) |
Net loss attributable to
common stockholders—basic and diluted |
|
$ |
(27,033 |
) |
|
$ |
(15,583 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.44 |
) |
|
$ |
(0.35 |
) |
Weighted-average common shares
outstanding—basic and diluted |
|
|
61,943,960 |
|
|
|
44,860,279 |
|
|
|
|
|
|
|
|
Six Months Ended June 30, |
|
|
|
2024 |
|
|
|
2023 |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
$ |
40,868 |
|
|
$ |
23,523 |
|
General and administrative |
|
|
11,141 |
|
|
|
11,240 |
|
Total operating expenses |
|
|
52,009 |
|
|
|
34,763 |
|
Loss from operations |
|
|
(52,009 |
) |
|
|
(34,763 |
) |
Other income (expense): |
|
|
|
|
Interest income |
|
|
4,404 |
|
|
|
2,937 |
|
Interest expense |
|
|
(2,720 |
) |
|
|
(1,099 |
) |
Other expenses |
|
|
(55 |
) |
|
|
(62 |
) |
Other income (expense),
net |
|
|
1,629 |
|
|
|
1,776 |
|
Net loss |
|
$ |
(50,380 |
) |
|
$ |
(32,987 |
) |
Other comprehensive income
(loss): |
|
|
|
|
Unrealized gains (losses) on available-for-sale securities |
|
|
(153 |
) |
|
|
226 |
|
Foreign currency translation adjustment |
|
|
9 |
|
|
|
30 |
|
Total other comprehensive
income (loss) |
|
|
(144 |
) |
|
|
256 |
|
Comprehensive
loss |
|
$ |
(50,524 |
) |
|
$ |
(32,731 |
) |
Net loss attributable to
common stockholders—basic and diluted |
|
$ |
(50,380 |
) |
|
$ |
(32,987 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.81 |
) |
|
$ |
(0.74 |
) |
Weighted-average common shares
outstanding—basic and diluted |
|
|
61,858,119 |
|
|
|
44,293,577 |
|
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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