Inozyme Pharma, Inc. (Nasdaq: INZY) (“the
Company” or “Inozyme”), a clinical-stage biopharmaceutical company
developing innovative therapeutics for rare diseases that affect
bone health and blood vessel function, today reported financial
results for the third quarter ended September 30, 2024, and
provided business highlights.
“As we close in on the end of a highly productive year, our
focus remains firmly on advancing INZ-701 across each of our
clinical programs,” said Douglas A. Treco, Ph.D., CEO and Chairman
of Inozyme Pharma. “The recent presentation of promising interim
data from our Phase 1 data from our calciphylaxis program
highlights our progress, and we remain on track to report interim
data from our Phase 1b ENERGY 1 trial in ENPP1 Deficiency by
year-end. With a growing body of clinical evidence supporting the
potential for INZ-701 to serve as a meaningful therapy across
multiple indications of high unmet need, we are committed to our
mission to bring novel treatment options to patients facing rare
diseases affecting bone health and blood vessel function.”
Recent Highlights
Pipeline
- Presentations at American Society for Bone and Mineral
Research (ASBMR) 2024 Annual Meeting. In September 2024,
the Company presented new data at ASBMR 2024 in Toronto, Canada,
demonstrating the progression and impact of ENPP1 Deficiency and
ABCC6 Deficiency in children. These findings underscored the urgent
need for innovative therapies to address the severe cardiovascular
and musculoskeletal complications associated with these conditions.
In addition, the Company and GACI Global highlighted the launch of
the PROPEL Registry designed to further understanding of the burden
of illness and progressive nature of ENPP1 Deficiency and
early-onset ABCC6 Deficiency (NCT06302439).
ENPP1 Deficiency
- ENPP1 Deficiency Review Series Publication. In
September 2024, a series of comprehensive review articles on ENPP1
Deficiency was published in the French journal of pediatrics,
Archives de Pédiatrie. Authored by expert clinicians and
researchers in bone health, the articles collectively highlight the
complexities of ENPP1 Deficiency, underscoring both established and
emerging insights into the disease’s presentation and
management.
Calciphylaxis
- Phase 1 Trial of INZ-701 in Patients with End-Stage
Kidney Disease (ESKD) Undergoing Hemodialysis. In October
2024, the Company announced positive interim data demonstrating
INZ-701 was well-tolerated and significantly increased plasma
pyrophosphate (PPi) levels in patients with ESKD on dialysis. Low
PPi levels are linked to the development of calciphylaxis, a rare
and life-threatening complication of ESKD, as well as the
associated morbidity and mortality. Data were featured at the
American Society of Nephrology’s Kidney Week 2024 in San Diego, CA.
Subject to regulatory review and sufficient funding, the Company
plans to initiate a registrational study in calciphylaxis in
2025.
Corporate
- Board of Directors Appointment. In October
2024, the Company announced the appointment of Erik Harris to its
Board of Directors. Mr. Harris, who currently serves as Chief
Commercial Officer and Executive Vice President at Ultragenyx
Pharmaceutical Inc., brings to the Company over 20 years of
commercial expertise within the biopharmaceutical industry.
Anticipated Milestones
- ENPP1 Deficiency
- Complete enrollment of ENERGY 3 pivotal trial in pediatric
patients by the end of 2024
- Initiation of the ENERGY 2 pivotal trial in infants with ENPP1
Deficiency outside the United States in the fourth quarter of
2024
- Release interim data from the ENERGY 1 Phase 1b trial in
infants in the fourth quarter of 2024
- Release topline data from the ENERGY 3 pivotal trial in
pediatric patients in early 2026
- ABCC6 Deficiency
- Initiation of pivotal clinical trial of INZ-701 in pediatric
patients with ABCC6 Deficiency in 2025, subject to regulatory
alignment and sufficient funding.
- Calciphylaxis
- Initiation of a pivotal trial of INZ-701 in patients with
calciphylaxis in 2025, subject to regulatory alignment and
sufficient funding.
Third Quarter 2024 Financial Results
- Cash Position and Financial Guidance. Cash,
cash equivalents, and short-term investments were $131.6 million as
of September 30, 2024. Based on its current plans, the Company
anticipates its cash, cash equivalents, and short-term investments
as of September 30, 2024, will enable the Company to fund cash flow
requirements into the fourth quarter of 2025.
- Research and Development (R&D) Expenses.
R&D expenses were $19.9 million for the quarter ended September
30, 2024, compared to $13.3 million for the prior-year period.
- General Administrative (G&A) Expenses.
G&A expenses were $5.0 million for the quarter ended September
30, 2024, compared to $4.7 million for the prior-year period.
- Net Loss. Net loss was $24.6 million, or $0.39
net loss per share, for the quarter ended September 30, 2024,
compared to $16.6 million or $0.29 net 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 Calciphylaxis and the PPi-Adenosine
PathwayCalciphylaxis (also known as calcific uremic
arteriolopathy, or CUA) is a rare disorder with a high mortality
rate that predominantly 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. This leads to poor blood
flow, blood clots, painful skin ulcers, serious infections, and
often death, with a reported one-year survival rate of
approximately 50%. Currently, there are no approved therapies for
calciphylaxis. 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.
The PPi-Adenosine Pathway plays a critical role in regulating
both pathologic mineralization and intimal proliferation. The ENPP1
enzyme generates PPi, a potent inhibitor of pathologic
mineralization, by hydrolyzing extracellular adenosine
triphosphate. Additionally, adenosine, produced by the CD73 enzyme
regulates intimal proliferation, preventing the abnormal growth of
smooth muscle cells within blood vessels, which can contribute to
vascular occlusion. Recent genetic research has shown that
polymorphisms in the ENPP1 or CD73 genes have
been linked to an increased risk of arterial calcification in ESKD
patients and/or calciphylaxis, further substantiating the role of
the PPi-Adenosine Pathway in this condition.
INZ-701 is designed to restore PPi levels and increase adenosine
production, addressing both key elements of the PPi-Adenosine
Pathway. By normalizing these processes, INZ-701 has the potential
to prevent the progression of calciphylaxis, which could offer a
promising therapeutic solution for this high-risk and underserved
patient population.
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 and calciphylaxis programs, 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) |
| |
|
|
|
| |
September 30,2024 |
|
December 31,2023 |
|
Cash, cash equivalents and investments |
$ |
131,608 |
|
|
$ |
188,589 |
|
| Total
assets |
$ |
143,361 |
|
|
$ |
200,847 |
|
| Total
liabilities |
$ |
60,573 |
|
|
$ |
60,368 |
|
|
Additional paid-in-capital |
$ |
443,476 |
|
|
$ |
426,362 |
|
|
Accumulated deficit |
$ |
(360,880 |
) |
|
$ |
(285,930 |
) |
| Total
stockholders' equity |
$ |
82,788 |
|
|
$ |
140,479 |
|
|
Condensed Consolidated Statements of Operations and
Comprehensive Loss(Unaudited) |
| |
| |
|
Three Months Ended September 30, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Operating expenses: |
|
|
|
|
|
Research and development |
|
$ |
19,890 |
|
|
$ |
13,341 |
|
|
General and administrative |
|
|
4,961 |
|
|
|
4,733 |
|
| Total
operating expenses |
|
|
24,851 |
|
|
|
18,074 |
|
| Loss
from operations |
|
|
(24,851 |
) |
|
|
(18,074 |
) |
| Other
income (expense): |
|
|
|
|
|
Interest income |
|
|
1,778 |
|
|
|
2,369 |
|
|
Interest expense |
|
|
(1,416 |
) |
|
|
(953 |
) |
|
Other (expense)income, net |
|
|
(81 |
) |
|
|
20 |
|
| Other
income, net |
|
|
281 |
|
|
|
1,436 |
|
|
Net loss |
|
$ |
(24,570 |
) |
|
$ |
(16,638 |
) |
| Other
comprehensive income (loss): |
|
|
|
|
|
Unrealized gains on available-for-sale securities |
|
|
290 |
|
|
|
53 |
|
|
Foreign currency translation adjustment |
|
|
(1 |
) |
|
|
(182 |
) |
| Total
other comprehensive income (loss) |
|
|
289 |
|
|
|
(129 |
) |
|
Comprehensive loss |
|
$ |
(24,281 |
) |
|
$ |
(16,767 |
) |
| Net loss
attributable to common stockholders—basic and diluted |
|
$ |
(24,570 |
) |
|
$ |
(16,638 |
) |
| Net loss
per share attributable to common stockholders—basic and
diluted |
|
$ |
(0.39 |
) |
|
$ |
(0.29 |
) |
|
Weighted-average common shares outstanding—basic and diluted |
|
|
63,276,851 |
|
|
|
56,758,395 |
|
|
|
|
|
|
|
| |
|
Nine Months Ended September 30, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Operating expenses: |
|
|
|
|
|
Research and development |
|
$ |
60,758 |
|
|
$ |
36,864 |
|
|
General and administrative |
|
|
16,101 |
|
|
|
15,973 |
|
| Total
operating expenses |
|
|
76,859 |
|
|
|
52,837 |
|
| Loss
from operations |
|
|
(76,859 |
) |
|
|
(52,837 |
) |
| Other
income (expense): |
|
|
|
|
|
Interest income |
|
|
6,182 |
|
|
|
5,306 |
|
|
Interest expense |
|
|
(4,136 |
) |
|
|
(2,052 |
) |
|
Other expense, net |
|
|
(137 |
) |
|
|
(42 |
) |
| Other
income (expense), net |
|
|
1,909 |
|
|
|
3,212 |
|
|
Net loss |
|
$ |
(74,950 |
) |
|
$ |
(49,625 |
) |
| Other
comprehensive income (loss): |
|
|
|
|
|
Unrealized gains on available-for-sale securities |
|
|
137 |
|
|
|
279 |
|
|
Foreign currency translation adjustment |
|
|
8 |
|
|
|
(152 |
) |
| Total
other comprehensive income |
|
|
145 |
|
|
|
127 |
|
|
Comprehensive loss |
|
$ |
(74,805 |
) |
|
$ |
(49,498 |
) |
| Net loss
attributable to common stockholders—basic and diluted |
|
$ |
(74,950 |
) |
|
$ |
(49,625 |
) |
| Net loss
per share attributable to common stockholders—basic and
diluted |
|
$ |
(1.20 |
) |
|
$ |
(1.02 |
) |
|
Weighted-average common shares outstanding—basic and diluted |
|
|
62,334,482 |
|
|
|
48,494,175 |
|
Contacts
Investors:Inozyme PharmaStefan Riley, Senior Director of IR and
Corporate Communications(857)
330-8871Stefan.riley@inozyme.com
Media:Biongage CommunicationsTodd Cooper(617)
840-1637Todd@biongage.com
Inozyme Pharma (NASDAQ:INZY)
過去 株価チャート
から 10 2024 まで 11 2024
Inozyme Pharma (NASDAQ:INZY)
過去 株価チャート
から 11 2023 まで 11 2024
