Announcement • Jan 10

Inozyme Pharma, Inc. Announces Positive Interim Data for Inz-701 in Infants and Young Children with Enpp1 Deficiency and Key Program Updates

Inozyme Pharma, Inc. announced positive interim data from its ENERGY 1 trial and Expanded Access Program (EAP) evaluating INZ-701 in infants and young children with ENPP1 Deficiency, completion of enrollment in the ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency and regulatory guidance for the ASPIRE pivotal trial in children with ABCC6 Deficiency. Positive Interim Data from the ENERGY 1 trial and Expanded Access Program Interim data from the ENERGY 1 trial (three infants) and the EAP (two infants and one child -2.5 years old) evaluated patients with generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 Deficiency. Patients were treated with INZ-701 for periods of three weeks to 22 months. The data presentation can be accessed here on Inozyme’s Investor Relations site. Key results include: Improved Survival: 80% of infants treated with INZ-701 survived beyond their first year, compared to a historical survival rate of approximately 50%. Reduction in Arterial Calcifications: Substantial reductions or stabilization of arterial calcifications were observed in all surviving patients, including complete resolution in some instances. There was no evidence of progression of arterial calcification in any patient. Improved Heart Function: Stabilization or improvement in left ventricular ejection fraction (LVEF) was noted in all surviving patients. Reduced Risk of Rickets: No radiographic evidence of rickets was observed in patients evaluated beyond one year of age and at-risk of rickets development (n=3), supported by stabilization or increases in serum phosphate levels. Favorable Safety Profile: INZ-701 was well-tolerated, with no serious treatment-related adverse events in infants and young children. Observed treatment-related events were limited to mild injection site reactions. Across studies to-date low, often transient, anti-drug antibody (ADA) levels were noted in some children and adults, with no impact on pharmacokinetics (PK) or pharmacodynamics (PD). In the ENERGY 1 trial and EAP, higher ADA levels in some infants significantly affected PK and PD. In infants with high ADA levels, data collected pre- and post-dosing demonstrated substantial transient increases in PPi and drug exposure following INZ-701 administration, consistent with the clinical effects observed. ADAs were not associated with adverse events in any patient. Enrollment Complete in ENERGY 3 Pivotal Trial The Company today announced completion of enrollment in its ENERGY 3 pivotal trial of INZ-701 in patients with ENPP1 Deficiency aged >1 to <13 years. Based on recommendations from the U.S. Food and Drug Administration (FDA), the primary endpoint of plasma PPi should be supported by consistent trends in appropriate clinical endpoints, such as radiographic global impression of change (RGI-C), a measure for progression or improvement of rickets. As per agreement with the European Medicines Agency (EMA), plasma PPi and RGI-C are co-primary endpoints, with a relaxed p-value of <0.2 for RGI-C. With 25 patients enrolled, the trial’s 2:1 randomized design provides >90% power to detect meaningful differences in RGI-C between treatment and control groups. Strong patient interest and scheduled screenings may result in the enrollment of additional participants in January 2025. Inozyme anticipates completing the one-year dosing period for all patients by January 2026, with topline data expected in early 2026. Inozyme is advancing the development of INZ-701 in ABCC6 Deficiency. In April 2024, the Company reported topline data from an open-label, dose-escalation study in adults, along with findings from a natural history study documenting the significant disease burden in patients with the early-onset form of the disease, known as GACI Type 2 (GACI-2). The adult study demonstrated positive improvements in vascular and retinal pathology after 48 weeks of treatment with INZ-701, as well as normalization of PPi levels at the highest dose tested, supporting further development in additional age groups. The natural history study revealed a high disease burden characterized by childhood strokes, arteriopathy, cardiovascular complications, and early mortality. Further research has identified a substantial pediatric population with ABCC6 Deficiency, underscoring the significant unmet medical need in this group. The natural history study data, supplemented by literature reports, has informed the design of the Company’s planned randomized, controlled ASPIRE trial of INZ-701 in children with ABCC6 Deficiency. The proposed primary endpoint, comprising major adverse clinical events over a two-year treatment period, has been reviewed and received preliminary support from U.S. and EU regulators. The trial is expected to enroll approximately 70 patients from infancy up to <18 years old with biallelic or monoallelic ABCC6 Deficiency. Inozyme is currently refining the study design to harmonize feedback from the FDA and EMA. The Company plans to continue regulatory engagement over the coming months to finalize the trial protocol. Pending ongoing regulatory review and the availability of financial resources, Inozyme aims to initiate the ASPIRE trial in early 2026.

Announcement • Mar 27

Inozyme Pharma, Inc. to Report Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency and ENPP1 Deficiency

Inozyme Pharma, Inc. announced that it will discuss topline data from its ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency (pseudoxanthoma elasticum or PXE), and from all cohorts of its ongoing Phase 1/2 trial of INZ-701 in adults with ENPP1 Deficiency. In addition, the event will feature an update on the Company’s Natural History Study in pediatric patients with ABCC6 Deficiency, including presentations from the following key opinion leaders who will provide an overview of the unmet need in ABCC6 Deficiency. ABCC6 Deficiency is a progressively debilitating condition of the vasculature and soft tissue that is estimated to affect approximately 1 in 25,000 to 1 in 50,000 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. Pediatric patients who survive the first year of life may develop neurological disease, including stroke, and cardiovascular disease secondary to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), which is characterized by pathologic mineralization in blood vessels and soft tissues clinically affecting the skin, eyes, and vascular system. There are no approved therapies for 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. ENPP1 Deficiency is a progressively debilitating condition of the vasculature, soft tissue, and skeleton with a prevalence of approximately 1 in 64,000 pregnancies worldwide. Although ENPP1 Deficiency was initially described in patients with biallelic ENPP1 Deficiency (homozygous or compound heterozygous mutations), many patients with monoallelic ENPP1 Deficiency (heterozygous mutations) have clinical symptoms, potentially increasing the worldwide prevalence. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1) and approximately 50% of infants 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. 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. INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. INZ-701 metabolizes ATP to generate PPi, a natural inhibitor of mineralization, and AMP, which can be processed to phosphate and adenosine, the latter being a natural inhibitor of intimal proliferation. In preclinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating disorders such as ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis. Clinical data to date have demonstrated that INZ-701 was generally safe and well tolerated and meaningfully increased PPi levels in multiple clinical trials.

Total Shareholder Yield	-4.4%
Future Dividend Yield	0%
Dividend Growth	n/a
Next dividend pay date	n/a
Ex dividend date	n/a
Dividend per share	n/a
Payout ratio	n/a

Segment	Dividend Yield
Company (INZY)	n/a
Market Bottom 25% (US)	1.4%
Market Top 25% (US)	4.2%
Industry Average (Biotechs)	2.3%
Analyst forecast (INZY) (up to 3 years)	0%

Data	Last Updated (UTC time)
Company Analysis	2025/07/03 10:09
End of Day Share Price	2025/06/30 00:00
Earnings	2025/03/31
Annual Earnings	2024/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Edward White	H.C. Wainwright & Co.
Joseph Stringer	Needham & Company
Dae Gon Ha	Stifel, Equities Research

This company has been acquired

Inozyme Pharma Dividends and Buybacks

Dividend criteria checks 0/6

Key information

Recent dividend and buyback updates

Recent updates

New major risk - Revenue and earnings growth

Inozyme Pharma, Inc.(NasdaqGS:INZY) dropped from NASDAQ Composite Index

Price target decreased by 70% to US$4.00

Insufficient new directors

BioMarin Pharmaceutical Inc. (NasdaqGS:BMRN) agreed to acquire Inozyme Pharma, Inc. (NasdaqGS:INZY) for approximately $260 million.

Inozyme Pharma, Inc., Annual General Meeting, Jun 25, 2025

Inozyme Pharma, Inc. Auditor Raises 'Going Concern' Doubt

Inozyme Pharma, Inc. Announces Workforce Reduction of Approximately 25% of Employees Implemented in the First Quarter of 2025

New minor risk - Market cap size

Price target decreased by 8.0% to US$18.00

New minor risk - Share price stability

Inozyme Pharma, Inc. Announces Positive Interim Data for Inz-701 in Infants and Young Children with Enpp1 Deficiency and Key Program Updates

Does Inozyme Pharma (NASDAQ:INZY) Have A Healthy Balance Sheet?

New major risk - Revenue and earnings growth

New major risk - Revenue and earnings growth

Inozyme Pharma, Inc. Announces Positive Interim Data from Phase 1 Seaport 1 Trial of INZ-701

Inozyme Pharma, Inc. Announces Appointment of Erik Harris to its Board of Directors

New major risk - Financial position

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Inozyme: Q4 Of 2024 Phase 1 INZ-701 Data Could Lead To Indication Expansion

Inozyme Pharma Announces FDA Fast Track Designation for INZ-701 in ABCC6 Deficiency

New major risk - Revenue and earnings growth

Price target decreased by 7.5% to US$19.43

New major risk - Revenue and earnings growth

Inozyme Pharma, Inc., Annual General Meeting, Jun 18, 2024

Inozyme Pharma (NASDAQ:INZY) Has Debt But No Earnings; Should You Worry?

Inozyme Pharma, Inc. Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with Abcc6 Deficiency (PXE) and ENPP1 Deficiency

CEO & Chairman exercised options and sold US$52k worth of stock

New minor risk - Share price stability

Inozyme Pharma, Inc. to Report Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency and ENPP1 Deficiency

New major risk - Revenue and earnings growth

Inozyme: Several Catalysts To Move Rare Disease Pipeline Programs Forward

Inozyme Pharma Highlights Inclusion of Generalized Arterial Calcification of Infancy in Genomics England's Generation Study of Rare Conditions

Inozyme Pharma, Inc. Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE)

New minor risk - Share price stability

Price target decreased by 9.3% to US$20.50

New major risk - Shareholder dilution

New major risk - Revenue and earnings growth

Inozyme Pharma, Inc. has completed a Follow-on Equity Offering in the amount of $60 million.

Price target increased by 8.5% to US$22.60

Inozyme Pharma, Inc. Announces Updates on Global Development Strategy of INZ-701 for the Treatment of ENPP1 Deficiency

Inozyme Pharma Announces Dosing of First Infant with ENPP1 Deficiency in Phase 1b Trial of INZ-701

Insufficient new directors

Price target decreased by 13% to US$19.17

Inozyme Pharma, Inc. Reports Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701

Price target increased by 8.5% to US$21.80

Inozyme Pharma, Inc. Provides Business Updates

Inozyme Pharma, Inc. Announces First Self-Administration of Inz-701 in Ongoing Enpp1 Deficiency Phase 1/2 Clinical Trial

Inozyme Pharma Announces Publication of Comprehensive ENPP1 Variant Database

Price target increased to US$23.20

Inozyme Pharma GAAP EPS of -$0.38

Inozyme Pharma, Inc. Announces Positive Preliminary Data from Phase 1/2 Clinical Trial of INZ-701 in Subjects with ABCC6 Deficiency (pseudoxanthoma elasticum or PXE)

Inozyme Pharma releases positive early-stage data on candidate for ABCC6 Deficiency

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Inozyme Pharma, Inc.(NasdaqGS:INZY) dropped from Russell 2500 Index

Inozyme Pharma, Inc. Appoints Kurt Gunter as Chief Medical Officer

Price target decreased to US$29.40

Inozyme Pharma, Inc. Provides Update for Phase 1/2 Clinical Trials in ABCC6 Deficiency and ENPP1 Deficiency

We're Keeping An Eye On Inozyme Pharma's (NASDAQ:INZY) Cash Burn Rate

Inozyme Pharma, Inc. Announces Positive Preliminary Data from Phase 1/2 Clinical Trial of INZ-701 in Patients with ENPP1 Deficiency

Price target decreased to US$30.00

Inozyme Pharma, Inc. Announces Management Changes

High number of new directors

Here's Why We're Not Too Worried About Inozyme Pharma's (NASDAQ:INZY) Cash Burn Situation

Price target decreased to US$32.25

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Inozyme Pharma Receives Orphan Drug Designation for INZ-701 from the European Medicines Agency for the Treatment of ABCC6 Deficiency

Inozyme Pharma, Inc.(NasdaqGS:INZY) dropped from Russell 2500 Growth Index

President exercised options and sold US$65k worth of stock

Inozyme Pharma, Inc. Announces Acceptance of First European Clinical Trial Application for Phase 1/2 Clinical Trial of Inz-701 in Abcc6 Deficiency

We're Not Very Worried About Inozyme Pharma's (NASDAQ:INZY) Cash Burn Rate

Senior Vice President of Finance Stephen Basso has left the company

Inozyme Pharma, Inc. Presents Preclinical Data Suggesting Utility of INZ-701 as a Potential Treatment for ABCC6 Deficiency

Member of Scientific Advisory Board has left the company