Inozyme Pharma Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE) - Seite 2

ENPP1 Deficiency

Nine patients were initially enrolled in the ongoing Phase 1/2 clinical trial across three dose cohorts of INZ-701 (0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=3)). For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

Exploratory Biomarker Data

Exploratory biomarker data were collected throughout the study to provide evidence of the potential for disease modification with ongoing treatment with INZ-701. Notable changes in key biomarkers were observed and support the clinical hypothesis, including:

• Meaningful reduction of fibroblast growth factor-23 (FGF-23) observed. Most patients with ENPP1 Deficiency have elevated levels of FGF-23, which leads to increased phosphate wasting and hypophosphatemia, a key driver of osteomalacia and rickets.
• Serum phosphate (Pi) levels increased over time, in the absence of phosphate and active vitamin D supplementation, which were withheld from patients during the study.
• Statistically significant correlation between increase in plasma pyrophosphate (PPi) and decrease in FGF-23 observed at one week post first dose.
• Upward trends observed in bone specific alkaline phosphatase (BSAP) levels from baseline, which signal biological activity in bone tissue.

Exploratory Efficacy Data

Outcome measures were collected to assess potential clinical benefit with ongoing treatment with INZ-701 and to inform the design and patient selection of future trials in adolescents and adults. Notable changes in PROs and functional outcomes were observed in all cohorts, including:

• Concordant improvement in GIC scores reported by patients (P-GIC) and clinicians (C-GIC), and no patient showed a deterioration from baseline.
• High responder rates in Patient-Reported Outcome Measurement Information Scales (PROMIS) of Pain Intensity, Fatigue and Pain Interference.¹
• Trend of improvement in 6-minute walk test (6-MWT). Subgroup analysis of 6-MWT results showed greater improvement in patients with lower baseline values and stable results over time in patients with higher baseline values.
• Subgroup analysis of patients who presented with arthritis/arthralgia at baseline showed improvement in 6-MWT, and increased spine bone mineral density (BMD) and bone mineral content (BMC), as measured by dual x-ray absorptiometry (DEXA).

Pharmacodynamic (PD) and Pharmacokinetic (PK) Data