Inozyme Pharma Publishes Preclinical Data Supporting INZ-701 as a Potential Therapy for a Broad Range of Serious Rare Diseases Impacting Bone Health and Blood Vessel Function
In This Article:
- Treatment with INZ-701 shown to prevent intimal proliferation in both ENPP1-deficient and wild-type mice, supporting its potential therapeutic application beyond traditional ERT -
BOSTON, July 25, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases that affect bone health and blood vessel function, today announced the publication of preclinical data supporting the potential of INZ-701, the Company’s lead ENPP1 enzyme replacement therapy (ERT) development candidate, to treat a broad range of diseases mediated by the PPi-Adenosine Pathway, which regulates mineralization and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). The article titled, “Inhibition of Vascular Smooth Muscle Cell Proliferation by ENPP1: The Role of CD73 and the Adenosine Signaling Axis”, published in the journal Cells can be accessed via this link.
“The data published in Cells highlights the potential of INZ-701 to inhibit intimal proliferation, an important advancement for treating rare diseases driven by pathological dysregulation of vascular smooth muscle cell overgrowth,” said Yves Sabbagh, Ph.D., Chief Scientific Officer of Inozyme Pharma and one of the senior authors on this publication. “INZ-701’s ability to address disruptions in the PPi-Adenosine Pathway positions it as a promising therapeutic candidate, offering hope across multiple rare diseases.”
Overview of Findings
The PPi-Adenosine Pathway is critical for regulating bone health and blood vessel function. The ENPP1 enzyme is a vital component of this pathway and plays an essential role in generating inorganic pyrophosphate (PPi), a key regulator of mineralization, and adenosine, a key regulator of intimal proliferation. Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurologic conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL).
INZ-701 is designed to increase PPi and adenosine, addressing deficiencies in these molecules and offering the potential to treat multiple rare diseases driven by disruptions in the PPi-Adenosine Pathway.
The Cells publication highlights the following preclinical findings with INZ-701 (ENPP1-Fc):
In vitro Experiments:
-
ENPP1-Fc was tested on vascular smooth muscle cells (VSMCs), which are the major cells in the walls of blood vessels.
-
Adding ENPP-1-Fc and ATP to VSMCs led to the production of AMP, which was subsequently converted to adenosine, reducing cell proliferation.
-
Both AMP and adenosine independently inhibited VSMC growth.
-
Blocking the CD73 enzyme, which converts AMP to adenosine, reduced the accumulation of adenosine, and suppressed the anti-proliferative effects of ENPP1/ATP.