INZ-701 for ENPP1 Deficiency

No longer recruiting at 7 trial locations

Overseen By Inozyme Clinical Trial Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Inozyme Pharma

Must not be taking: Vitamin D, Phosphate, Corticosteroids, others

Disqualifiers: Uncontrolled cardiovascular, Thyroid, Infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 5

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of INZ-701, an experimental treatment for individuals with ENPP1 Deficiency, a genetic condition affecting bone and cardiovascular health. The study will explore different doses to determine the most effective one for future use. Individuals diagnosed with ENPP1 Deficiency and specific genetic mutations related to it may be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

You may need to stop taking certain medications, like vitamin D, phosphate, and some others, if your doctor thinks it's safe. Check with your doctor to see if this applies to you.

Is there any evidence suggesting that INZ-701 is likely to be safe for humans?

Research has shown that INZ-701 is generally safe and well-tolerated by patients. Studies have found that the treatment significantly increases PPi levels, indicating its effectiveness. Reports describe its safety as favorable, with no major side effects noted in trials. Overall, the findings are encouraging, suggesting that patients tolerate the treatment well. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for ENPP1 deficiency, which often involves managing symptoms and complications, INZ-701 offers a novel approach by directly targeting the underlying cause of the condition. INZ-701 is designed to work by replenishing the deficient enzyme, ENPP1, which is crucial for proper mineralization and vascular health. This could potentially address the disease at its root rather than just alleviating symptoms. Researchers are excited about INZ-701 because it represents a shift towards a more targeted treatment that could significantly improve outcomes for patients with this genetic disorder.

What evidence suggests that INZ-701 might be an effective treatment for ENPP1 Deficiency?

Research has shown that INZ-701, the investigational treatment in this trial, might help treat ENPP1 Deficiency. This treatment replaces the ENPP1 enzyme, crucial for healthy bone growth. In earlier studies, INZ-701 positively affected bone health and mineral balance in individuals with ENPP1 Deficiency. These studies noted improvements in health outcomes over 48 weeks. This suggests that INZ-701 could help manage symptoms of ENPP1 Deficiency by addressing its underlying cause. ^{1 2 3 6 7}

Who Is on the Research Team?

Kurt Gunter, MD

Principal Investigator

Inozyme Pharma, Inc.

Are You a Good Fit for This Trial?

Adults aged 18 to <65 with a clinical diagnosis of ENPP1 Deficiency, confirmed by genetic testing, can join this trial. They must be able to complete the study and provide medical records. Women who can have children and men must agree to use contraception during the study.

Inclusion Criteria

Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)

In the opinion of the Investigator, must be willing and able to complete the Dose Evaluation Period

You have been diagnosed with ENPP1 Deficiency and have specific mutations in the ENPP1 gene.

Exclusion Criteria

Subjects who are pregnant, trying to become pregnant, or breastfeeding

Unable or unwilling to discontinue the use of any prohibited medication (examples include 1,25-dihydroxy vitamin D, phosphate, anti-FGF23 [eg, burosumab], calcimimetics, calcium-containing antacids, systemic corticosteroids, PTH suppressors). Discontinuation should be undertaken only if considered not detrimental and indicated by the subject's treating physician

You have had cancer, except for non-melanoma skin cancers or a specific type of early-stage cervical cancer, within the past 5 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Dose Evaluation

Participants receive multiple ascending doses of INZ-701 to assess safety, tolerability, pharmacokinetics, and pharmacodynamics

32 days

Multiple visits for dose administration and monitoring

Extension

Participants continue to receive INZ-701 with regular follow-up visits to monitor long-term safety and efficacy

Until INZ-701 is approved or an alternative study is available

Visits every 4 weeks until Week 48, then every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person) 30 days after last dose

What Are the Treatments Tested in This Trial?

Interventions

• INZ-701

Trial Overview

The trial is testing multiple doses of INZ-701, an enzyme replacement therapy for treating ENPP1 Deficiency. It aims to determine the safety, how well it's tolerated, its effects on the body (PK/PD), and find a suitable dose for further studies.

How Is the Trial Designed?

Group I: INZ-701 Experimental Treatment 1 Intervention

The study design of the Dose Evaluation Period is a MAD 3 + 3 with 3 dose cohorts. Additional cohorts may be added to evaluate an intermediate dose and/or an alternative dosing regimen of an existing dose level. Based on nonclinical findings and nonclinical pharmacology modeling, the initial planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg all twice weekly, not to exceed 3.6 mg/kg weekly. During the Extension Period, visits will be every 4 weeks until Week 48 and then every 12 weeks until the subject leaves the study. Subjects will complete an End of Study (EOS) Visit (Safety Follow-up Visit) 30 days after their last dose of INZ-701 (greater than 5 half-lives of INZ-701) for all subjects.

Who Is Running the Clinical Trial?

Inozyme Pharma

Lead Sponsor

Trials

10

Recruited

1,400+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06739980

Safety and Efficacy Study of INZ-701 in Patients With ...

The purpose of Study INZ701-108 (ENABLE) is to assess the safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency.

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11454822/

7217 Impact of INZ-701 on Bone and Mineral Metabolism ...

7217 Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency - Results from 48-week ...

3.

academic.oup.com

academic.oup.com/jes/article/8/Supplement_1/bvae163.441/7813475

7217 Impact of INZ-701 on Bone and Mineral Metabolism ...

7217 Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency - Results from 48-week ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04686175?intr=INZ-701&rank=5

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in ...

The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, ...

5.

biomarin.com

biomarin.com/news/press-releases/biomarin-strengthens-enzyme-therapy-business-with-acquisition-of-inozyme-pharma/

BioMarin Strengthens Enzyme Therapy Business with ...

INZ-701 is a potential first-in-class, subcutaneous enzyme replacement therapy that is being developed for infants, pediatric and adult patients ...

6.

inozyme.com

inozyme.com/scientific-focus/our-science/

Our Science

So far, clinical data have shown that INZ-701 was generally safe and well tolerated and that it meaningfully increased PPi levels in multiple clinical trials.

7.

medicine.yale.edu

medicine.yale.edu/news-article/new-data-reported-from-trials-of-drug-developed-at-yale-pathology-to-treat-rare-often-fatal-neonatal-disorder/

New Data Reported From Trials of Drug Developed at Yale ...

INZ-701 was generally well-tolerated and exhibited a favorable safety profile in both trials. The findings were shared as “highly encouraging” ...