Inozyme Pharma (Nasdaq: INZY) has published preclinical data in the journal Cells, supporting the potential of INZ-701, their lead ENPP1 enzyme replacement therapy (ERT) candidate, to treat a broad range of rare diseases affecting bone health and blood vessel function. The study demonstrates INZ-701's ability to inhibit intimal proliferation in both ENPP1-deficient and wild-type mice, suggesting potential applications beyond traditional ERT.
Key findings show that INZ-701 increases levels of inorganic pyrophosphate (PPi)and adenosine, addressing deficiencies in the PPi-Adenosine Pathway. This pathway is critical for regulating bone health and blood vessel function. The research reveals ENPP1's role in adenosine production, explaining its dual action in preventing abnormal mineralization and inhibiting intimal proliferation. These results position INZ-701 as a promising therapeutic for multiple rare diseases driven by PPi-Adenosine Pathway disruptions.