Ilofotase alfa

Ilofotase alfa is a recombinant human alkaline phosphatase enzyme replacement therapy, engineered from two human isoforms of the enzyme to optimise stability and activity. It is in development for hypophosphatasia (HPP) and was acquired as part of Ambrose’s purchase of AM-Pharma.

HPP is an inherited disease caused by low alkaline phosphatase activity, leading to bone fractures, tooth loss, muscle weakness, fatigue and pain. Today there is only one approved treatment, and it is for patients whose disease began in infancy or childhood. For adults with adult-onset HPP, including those with moderate to severe disease, there is no approved treatment option.

In a Phase 1b study in adult HPP patients, ilofotase alfa showed a positive safety profile and a dose-dependent effect on disease biomarkers. Its safety database spans nearly 700 patients. We believe it is appropriate to progress the asset in a Phase 2 trial, which could lead to accelerated or conditional approval through rare disease regulatory pathways.

Ilofotase alfa has Orphan Drug Designation for HPP from both the FDA and the EMA.

Indication

Hypophosphatasia (HPP) in patients diagnosed with adult-onset HPP, including many with moderate to severe disease.