AM-Pharma has reported positive data from the Phase Ib study of ilofotase alfa for the treatment of hypophosphatasia (HPP), a rare inherited metabolic disease.
The open-label pilot study assessed the proprietary recombinant alkaline phosphatase, ilofotase alfa, as a potential enzyme replacement therapy in adult HPP patients.
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A total of 12 patients were divided into two treatment arms and given either ilofotase alfa 0.8mg/kg or 3.2mg/kg intravenously.
After dosing, they were followed for ten days to evaluate changes in the biochemical fingerprint of HPP, tolerability and safety.
The key goal of the study was to observe changes in serum levels of inorganic pyrophosphate (PPi) and pyridoxal-5’-phosphate (PLP) as compared to baseline.
The study demonstrated a maximum proportional PLP reduction of 35% in the 0.8mg/kg cohort and 66% in the 3.2mg/kg cohort.
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By GlobalDataFor PPi, the maximum proportional reduction was 36% in the 0.8mg/kg cohort and 77% in the 3.2mg/kg cohort.
Pharmacokinetics were found to be consistent as compared with earlier clinical studies and ilofotase alfa was well-tolerated.
AM-Pharma CEO Juliane Bernholz said: “The pronounced effect on all HPP disease-relevant biomarkers together with a positive safety and tolerability profile, consistent with previous clinical studies with ilofotase alfa, supports its further development as a treatment option for adult HPP patients who are currently underserved.”
Adults with HPP suffer from recurrent fractures, muscle weakness, joint diseases and pain.
Ilofotase alfa is also being developed for treating acute kidney injury.
