A randomized Phase 1b trial evaluating the pharmacodynamics of ilofotase alfa in adults with hypophosphatasia
Authors' conclusion
These data demonstrate that single-dose ilofotase alfa enhances AP activity and results in dose-dependent reductions in primary disease-specific biomarkers without undesired effects on mineral homeostasis.
Study context
- Population
- Adults with hypophosphatasia; at baseline all had reduced alkaline phosphatase (AP) activity and elevated pyridoxal-5'-phosphateall completed
- Participants
- 12 adults
- Comparison
- Ilofotase alfa 0.8 mg/kg vs 3.2 mg/kg IV
- Primary endpoint
- Changes in plasma levels of AP substrates inorganic pyrophosphate and pyridoxal-5'-phosphate
Methodological detailsPhase 1b, single-center, open-label, randomized 1:1, single dose
- Phase 1b
- Single-center
- Open-label
- Randomized 1:1
- Single dose
Primary result
As reported in the abstract
10-d follow-up
- Greatest reduction in inorganic pyrophosphate and pyridoxal-5'-phosphate 2 h after start of dosing in both groups
- Inorganic pyrophosphate returned to baseline more rapidly with 0.8 mg/kg vs 3.2 mg/kg
Further findings
Circulating AP activity
- Mean AP activity peaked 24 h after dosing, then declined but remained above the lower limit of normal throughout
Ilofotase alfa pharmacokinetics
- Dose-proportional increase in ilofotase alfa, peak concentration 1-h post-infusion
Safety
- Treatment-emergent adverse events, all mild
- 8
Original publication
A randomized Phase 1b trial evaluating the pharmacodynamics of ilofotase alfa in adults with hypophosphatasia. J Bone Miner Res. Published 11 December 2025. doi:10.
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