Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or “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
second quarter ended June 30, 2023, and provided business
highlights.
“We continue to make tremendous progress
advancing our clinical programs of INZ-701 in patients with ENPP1
Deficiency and ABCC6 Deficiency, with interim data from both
programs expected in September 2023,” said Douglas A. Treco, Ph.D.,
chief executive officer and chairman of Inozyme’s board of
directors. “Notably, following regulatory discussions with the FDA
and EMA, we have outlined a clear path forward toward supporting
potential regulatory filings in ENPP1 Deficiency.”
Recent Clinical Highlights
- Global Development Strategy
Update for INZ-701 in Patients with ENPP1 Deficiency. In
July 2023, Inozyme announced that based on guidance from the U.S.
Food and Drug Administration (FDA) and the Paediatric Committee
(PDCO) of the European Medicines Agency (EMA), change in plasma PPi
will serve as the primary endpoint in the U.S., supported by
consistent trends in appropriate secondary endpoints, and a
co-primary endpoint in the EU for the ENERGY-3 pivotal trial in
pediatric patients.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 ongoing Phase 1/2 trial in adults, available results from the
ongoing ENERGY-1 Phase 1b and planned ENERGY-2 pivotal trial in
infants, and final results from the planned ENERGY-3 pivotal 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. Pending
regulatory discussions and appropriate financial resources, the
Company also plans to conduct the ENERGY-4 pivotal trial in
adolescents and adults with ENPP1 Deficiency. Data from the planned
ENERGY-4 trial may provide the basis for supplemental marketing
applications.
- ENERGY-1 Phase 1b Trial of
INZ-701 in Infants with ENPP1 Deficiency. Dosing is now
underway in the Phase 1b ENERGY-1 trial designed to primarily
assess the safety, tolerability, pharmacokinetic, and
pharmacodynamic profile of INZ-701 in infants with ENPP1
Deficiency.
- Medical Conference
Presentations. In the second quarter, Yves Sabbagh, Ph.D.,
Inozyme’s senior vice president and chief scientific officer, and
Kurt Gunter, M.D., Inozyme’s senior vice president and chief
medical officer, presented previously reported interim data from
the ongoing Phase 1/2 clinical trial of INZ-701 in patients with
ENPP1 Deficiency at the European Calcified Tissue Society Congress
and European Congress of Endocrinology, respectively.
Recent Corporate Highlights
- Closed Underwritten
Offering. In August 2023, the Company closed an
underwritten public offering of 14,375,000 shares of its common
stock at a price of $4.80 per share, for net proceeds of
approximately $64.5 million from the offering, after deducting
underwriting discounts and commissions and estimated offering
expenses.
Anticipated Milestones
- ENPP1 Deficiency
- Interim data from Cohorts 1-3 in
the Phase 2 portion of the ongoing Phase 1/2 trial in adults – Sep.
2023
- Initiation of the ENERGY-3 pivotal
trial in pediatric patients – Oct. 2023
- Topline data from Cohorts 1-3 in
the ongoing Phase 1/2 trial in adults – 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
- Interim data from the Phase 2
portion of the ongoing Phase 1/2 trial in adults – Sep. 2023
- Topline data from the Phase 2
portion of the ongoing Phase 1/2 trial in adults – Q1 2024
- Initiation of Phase 2 clinical
trial of INZ-701 in adult patients with ABCC6 Deficiency, subject
to regulatory review and sufficient funding – Q4 2024
Second Quarter 2023 Financial Results
- Cash Position and Financial
Guidance. Cash, cash equivalents, and short-term
investments were $140.2 million as of June 30, 2023. Based on its
current plans, the Company now anticipates its cash, cash
equivalents, and short-term investments as of June 30, 2023,
together with approximately $64.5 million in proceeds from the July
2023 public offering, will enable the Company to fund its cash flow
requirements into Q4 2025.
- Research and Development
(R&D) Expenses. R&D expenses were $11.7 million
for the quarter ended June 30, 2023, compared to $10.0 million for
the prior-year period. This increase was primarily due to increased
chemistry, manufacturing, and controls expenses, clinical
development costs, and personnel-related expenses to support our
clinical trials.
- General and Administrative
(G&A) Expenses. G&A expenses were $4.7 million for
the quarter ended June 30, 2023, compared to $5.4 million for the
prior-year period. The decrease was primarily related to a decrease
in stock-based compensation expense and cost-saving
initiatives.
- Net Loss. Net loss
was $15.6 million, or $0.35 loss per share, for the quarter ended
June 30, 2023, compared to $15.3 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.
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.
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. 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 clinical trials for the
treatment of ENPP1 Deficiency and ABCC6 Deficiency.
For more information, please visit
www.inozyme.com and follow us
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 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.
|
|
|
|
Condensed Consolidated Balance Sheet
Data |
(Unaudited) |
|
|
|
|
|
June 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents, and short-term investments |
$ |
140,247 |
|
|
$ |
127,866 |
|
Total assets |
$ |
150,929 |
|
|
$ |
139,195 |
|
Total liabilities |
$ |
45,336 |
|
|
$ |
20,801 |
|
Additional paid-in-capital |
$ |
353,285 |
|
|
$ |
333,356 |
|
Accumulated deficit |
$ |
(247,748 |
) |
|
$ |
(214,761 |
) |
Total stockholders' equity |
$ |
105,593 |
|
|
$ |
118,394 |
|
|
|
|
|
Condensed Consolidated Statements of Operations and
Comprehensive Loss |
(Unaudited) |
|
|
|
|
|
|
Three Months Ended June 30, |
Six Months Ended June 30, |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
11,666 |
|
|
$ |
10,007 |
|
|
$ |
23,523 |
|
|
$ |
21,821 |
|
General and administrative |
|
|
4,728 |
|
|
|
5,384 |
|
|
|
11,240 |
|
|
|
10,409 |
|
Total operating expenses |
|
|
16,394 |
|
|
|
15,391 |
|
|
|
34,763 |
|
|
|
32,230 |
|
Loss from operations |
|
|
(16,394 |
) |
|
|
(15,391 |
) |
|
|
(34,763 |
) |
|
|
(32,230 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
Interest income, net |
|
|
839 |
|
|
|
321 |
|
|
|
1,838 |
|
|
|
381 |
|
Other expense, net |
|
|
(28 |
) |
|
|
(191 |
) |
|
|
(62 |
) |
|
|
(296 |
) |
Other income, net |
|
|
811 |
|
|
|
130 |
|
|
|
1,776 |
|
|
|
85 |
|
Net loss |
|
$ |
(15,583 |
) |
|
$ |
(15,261 |
) |
|
$ |
(32,987 |
) |
|
$ |
(32,145 |
) |
Other comprehensive income (loss): |
|
|
|
|
|
|
|
|
Unrealized gains (losses) on available-for-sale
securities |
|
|
76 |
|
|
|
(225 |
) |
|
|
226 |
|
|
|
(357 |
) |
Foreign currency translation adjustment |
|
|
11 |
|
|
|
(43 |
) |
|
|
30 |
|
|
|
(58 |
) |
Total other comprehensive income (loss) |
|
|
87 |
|
|
|
(268 |
) |
|
|
256 |
|
|
|
(415 |
) |
Comprehensive loss |
|
$ |
(15,496 |
) |
|
$ |
(15,529 |
) |
|
$ |
(32,731 |
) |
|
$ |
(32,560 |
) |
Net loss attributable to common stockholders—basic and diluted |
|
$ |
(15,583 |
) |
|
$ |
(15,261 |
) |
|
$ |
(32,987 |
) |
|
$ |
(32,145 |
) |
Net loss per share attributable to commonstockholders—basic and
diluted |
|
$ |
(0.35 |
) |
|
$ |
(0.38 |
) |
|
$ |
(0.74 |
) |
|
$ |
(1.01 |
) |
Weighted-average common shares andpre-funded warrants
outstanding—basic and diluted |
|
|
44,860,279 |
|
|
|
39,703,550 |
|
|
|
44,293,577 |
|
|
|
31,739,197 |
|
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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