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Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1

This article explores ongoing clinical trials investigating the use of INZ-701, a recombinant human enzyme, for treating rare genetic disorders called ENPP1 Deficiency and ABCC6 Deficiency. These conditions can cause serious complications like abnormal calcification of arteries and bones. The trials aim to evaluate the safety and effectiveness of INZ-701 in patients of various ages, from infants to adults, with these rare diseases.

Table of Contents

What is INZ-701?

INZ-701 is an investigational drug being developed to treat rare genetic disorders related to abnormal calcification in the body. Its full scientific name is “recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1” (also known as rhENPP1-Fc)[1]. This long name describes the structure of the drug – it’s a lab-made version of a human enzyme (ENPP1) attached to part of an antibody to help it last longer in the body.

What conditions does INZ-701 treat?

INZ-701 is being studied for the treatment of several rare genetic disorders:

  • ENPP1 Deficiency: A condition where the body doesn’t produce enough of the ENPP1 enzyme, leading to abnormal calcification (hardening) of blood vessels and other tissues[2].
  • Generalized Arterial Calcification of Infancy (GACI): A severe form of ENPP1 Deficiency that affects infants, causing dangerous calcification of arteries[3].
  • ABCC6 Deficiency: A related disorder that can also cause abnormal calcification[4].
  • Pseudoxanthoma elasticum (PXE): A form of ABCC6 Deficiency that primarily affects the skin, eyes, and blood vessels[4].

How does INZ-701 work?

INZ-701 is designed to replace the missing or defective ENPP1 enzyme in patients with these disorders. The ENPP1 enzyme plays a crucial role in regulating calcification in the body by producing a substance called inorganic pyrophosphate (PPi). PPi helps prevent calcium from building up in places it shouldn’t, like blood vessels and other soft tissues[1].

By providing a working version of the ENPP1 enzyme, INZ-701 aims to increase PPi levels in the body and reduce abnormal calcification[1].

Current Clinical Trials

Several clinical trials are currently underway to evaluate INZ-701:

  • ENERGY 3 Study: A Phase 3 trial for children (ages 1-12) with ENPP1 Deficiency[1].
  • ENERGY Study: A study for infants (under 1 year old) with ENPP1 or ABCC6 Deficiency[2].
  • ENERGY 2 Study: A Phase 3 trial for infants with ENPP1 Deficiency, focusing on improving survival and preventing complications[3].
  • ADAPT Study: A long-term safety study for patients who have previously received INZ-701 in other trials[4].

How is INZ-701 administered?

INZ-701 is given as a subcutaneous injection (an injection under the skin). In most trials, it is administered once weekly. The dose is typically calculated based on the patient’s weight, with a common dose being 2.4 mg per kilogram of body weight[1][3].

What is known about INZ-701’s efficacy?

While final results from the ongoing clinical trials are not yet available, researchers are evaluating INZ-701’s effectiveness in several ways:

  • Increasing PPi levels in the blood[1][3]
  • Improving survival rates in infants with severe disease[3]
  • Reducing calcification in blood vessels and other tissues[3]
  • Improving heart function and preventing heart failure[3]
  • Promoting normal growth and development in children[1][3]
  • Improving bone abnormalities and rickets (softening of bones)[1]

Safety Information

As INZ-701 is still in clinical trials, comprehensive safety information is not yet available. The ongoing studies are closely monitoring for any side effects or safety concerns. Some key safety measures being evaluated include:

  • Adverse events (side effects) and their frequency and severity[4]
  • Changes in vital signs, weight, and laboratory tests[4]
  • Effects on heart function and structure[4]
  • Development of antibodies against the drug[4]

Patients considering participation in a clinical trial should discuss potential risks and benefits with their healthcare provider.

Ongoing and Future Research

Research on INZ-701 is ongoing, with several studies at different phases. Future research will likely focus on:

  • Long-term safety and effectiveness
  • Optimal dosing for different age groups and conditions
  • Potential use in related disorders
  • Quality of life improvements for patients

As INZ-701 is still an investigational drug, it is not yet approved for general use. Patients interested in accessing this treatment should speak with their doctor about the possibility of participating in clinical trials.

Trial Name Age Group Main Objectives Key Endpoints
ENERGY 3 Children 1-12 years Evaluate efficacy and safety of INZ-701 in ENPP1 Deficiency Change in PPi levels, skeletal abnormalities (RGI-C score)
ENERGY Infants <1 year Assess safety, tolerability, PK, and PD of INZ-701 Adverse events, INZ-701 concentrations, PPi levels
ADAPT >1 year (long-term) Evaluate long-term safety of INZ-701 Adverse events, drug levels, PPi concentrations
ENERGY 2 Infants <1 year Assess efficacy and safety, focus on survival PPi levels, overall survival, cardiac function

FAQ

  • What is INZ-701? INZ-701 is a recombinant human enzyme that combines ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) with a part of an antibody. It's designed to replace the missing or defective ENPP1 enzyme in patients with ENPP1 Deficiency.
  • What conditions are being studied in these trials? The trials are studying ENPP1 Deficiency and ABCC6 Deficiency. These rare genetic disorders can cause abnormal calcification in arteries, joints, and other tissues, leading to serious health problems.
  • How is INZ-701 administered in these trials? INZ-701 is given as a subcutaneous injection (under the skin) once a week. The dose is usually based on the patient's weight, with a maximum of 2.4 mg/kg in most trials.
  • What are the main goals of these clinical trials? The main goals are to assess the safety of INZ-701, determine if it increases levels of a substance called pyrophosphate (PPi) in the body, and evaluate its effectiveness in improving symptoms and outcomes for patients with these rare diseases.
  • Who can participate in these trials? The trials include patients of various ages, from newborns to adults, depending on the specific study. Participants must have a confirmed genetic diagnosis of ENPP1 or ABCC6 Deficiency and meet other eligibility criteria specific to each trial.

Ongoing Clinical Trials on Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1

  • Study on INZ-701 for Improving Survival in Infants with ENPP1 Deficiency

    Recruiting

    1 1 1
    Investigated drugs:
    France Hungary Italy Spain Sweden

  • Long-term Safety Study of INZ-701 for Patients with ENPP1 and ABCC6 Deficiencies, Including Pseudoxanthoma Elasticum and Generalized Arterial Calcification of Infancy

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany

  • Study on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency

    Not recruiting

    1 1 1
    Investigated drugs:
    France Spain

  • Study on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency

    Not recruiting

    1 1
    Investigated drugs:
    Spain

Glossary

  • ENPP1 Deficiency: A rare genetic disorder where the body lacks a working ENPP1 enzyme, leading to low levels of pyrophosphate and abnormal calcification in various tissues.
  • ABCC6 Deficiency: Another rare genetic condition that can cause abnormal mineralization and calcification in the body, similar to ENPP1 Deficiency.
  • Pyrophosphate (PPi): A substance in the body that helps prevent abnormal calcification. Levels are often low in patients with ENPP1 Deficiency.
  • Subcutaneous injection: A method of giving medication by injecting it under the skin.
  • Recombinant protein: A protein produced using genetic engineering techniques, often used to create therapeutic drugs like INZ-701.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including how it's absorbed, distributed, and eliminated.
  • Pharmacodynamics (PD): The study of how a drug affects the body and its biological processes.
  • Adverse event (AE): Any unfavorable medical occurrence in a patient during a clinical trial, whether it's related to the study treatment or not.
  • Efficacy: How well a treatment works to improve a condition or disease.
  • Open-label study: A type of clinical trial where both the researchers and participants know which treatment is being given.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-inz-701-for-children-with-enpp1-deficiency/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-inz-701-for-infants-with-enpp1-or-abcc6-deficiency/
  3. http://clinicaltrials.eu/trial/study-on-inz-701-for-improving-survival-in-infants-with-enpp1-deficiency/
  4. http://clinicaltrials.eu/trial/long-term-safety-study-of-inz-701-for-patients-with-enpp1-and-abcc6-deficiencies-including-pseudoxanthoma-elasticum-and-generalized-arterial-calcification-of-infancy/