Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced positive preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three patients treated in the Phase 1 portion of its ongoing first-in-human Phase 1/2 clinical trial of INZ-701 in adult patients with ENPP1 Deficiency. At the 0.2 mg/kg dose level of INZ-701, all three patients showed rapid, significant, and sustained increases in PPi levels. Preclinical findings demonstrated PPi as a key predictive biomarker of therapeutic benefit in ENPP1 Deficiency.
“ENPP1 Deficiency is a devastating disease, and patients currently have no approved therapies,” said Axel Bolte, MSc, MBA, Inozyme’s co-founder, president, and chief executive officer. “We are greatly encouraged by the data reported today, as they represent the first clinical evidence supporting the potential of INZ-701 to address an urgent medical need. The data from the lowest dose cohort demonstrated that INZ-701 was able to rapidly and significantly increase PPi levels in these patients, with a potential for therapeutic benefit. We look forward to additional data from patients at the next dose levels in our ongoing clinical trial.”
Summary of Preliminary Data
The range of PPi levels across three patients at screening was 132-333 nM.
The range of PPi levels measured at six hours after the first dose was 581-1239 nM, an approximately 4-fold mean increase from screening across the three patients.
The mean PPi level during the 32-day dose evaluation period across the three patients was 1356 nM, an approximately 5-fold mean increase from screening across the three patients.
The range of peak PPi levels observed during the 32-day dose evaluation period across the three patients was 1082-2416 nM, and was comparable to data from our study of healthy subjects (n=10), which showed PPi levels between 1002 nM and 2169 nM.
