INZ-701

INZ-701 is an experimental biologic therapy currently under development by Inozyme Pharma, a biopharmaceutical company specializing in the development of novel medicines for the treatment of rare metabolic diseases. INZ-701 is being developed for the treatment of Enzyme Deficiency Disorders such as Generalized Arterial Calcification of Infancy (GACI) and Pseudoxanthoma Elasticum (PXE).

Mechanism of Action

INZ-701 is a soluble, recombinant fusion protein that is designed to replace the deficient or inactive enzyme in patients with certain metabolic diseases. It is designed to correct the underlying enzyme deficiency that leads to the pathological mineralization observed in GACI and PXE. The therapy works by binding to and stabilizing pyrophosphate (PPi), a natural inhibitor of mineralization in the body.

Clinical Trials

As of 2021, INZ-701 is in the early stages of clinical development. The first Phase 1 clinical trial was initiated in 2020 to evaluate the safety, tolerability, and pharmacokinetics of INZ-701 in healthy volunteers. The results of this trial are expected to inform the design of future Phase 2 and Phase 3 trials in patients with GACI and PXE.

Potential Impact

If successful, INZ-701 could provide a much-needed treatment option for patients with GACI and PXE, two rare and currently untreatable metabolic diseases. These diseases are characterized by pathological mineralization, leading to severe morbidity and mortality. By correcting the underlying enzyme deficiency, INZ-701 could potentially halt or even reverse the pathological mineralization process, thereby improving patient outcomes.

See Also

• Biologic therapy
• Enzyme Deficiency Disorders
• Generalized Arterial Calcification of Infancy
• Pseudoxanthoma Elasticum
• Inozyme Pharma
• Clinical trial