AM Pharma is developing ilofotase alfa, a proprietary recombinant human alkaline phosphatase, as an enzyme replacement therapy for adult hypophosphatasia (HPP). The subcutaneous treatment restores alkaline phosphatase activity, lowering disease‑related pyridoxal‑5′‑phosphate and inorganic pyrophosphate levels, which improves bone mineralization, muscle function, and pain. Phase 1b data show dose‑dependent normalization of HPP biomarkers and a favorable safety profile, positioning ilofotase alfa for Phase 2 trials to address the unmet need in adult HPP patients.
Funding
Funding not disclosed
Founders
Product
Problem
Adults with hypophosphatasia (HPP) often lack approved enzyme replacement therapies, leading to persistent bone mineralization defects, muscle weakness, pain, and reduced quality of life. Existing treatments are limited to pediatric-onset patients and are not indicated for adult-diagnosed cases, leaving a significant therapeutic gap.
Solution
AM‑Pharma is developing ilofotase alfa, a recombinant human alkaline phosphatase engineered from two human isoforms, as an enzyme replacement therapy for adult HPP. By restoring alkaline phosphatase activity, ilofotase alfa reduces elevated pyridoxal‑5′‑phosphate and inorganic pyrophosphate levels, addressing both skeletal and non‑skeletal manifestations of the disease. Phase 1b clinical data in adult patients demonstrated dose‑dependent normalization of disease biomarkers, increased alkaline phosphatase activity, and a favorable safety profile. The product is formulated for subcutaneous administration, enabling convenient outpatient treatment. Ongoing development positions ilofotase alfa for Phase 2 trials, aiming to provide a disease‑specific option for the underserved adult HPP population.
Target Audience
Primary customers are adult patients diagnosed with hypophosphatasia and their healthcare providers, including endocrinologists, metabolic bone specialists, and clinical researchers focused on rare metabolic bone disorders.
Features
- Proprietary recombinant alkaline phosphatase combining two human isoforms for high enzymatic activity and stability
- Subcutaneous formulation designed for convenient outpatient dosing
- Mechanistic action that lowers pathogenic pyridoxal‑5′‑phosphate and inorganic pyrophosphate, supporting bone mineralization and muscle function
- Demonstrated pharmacodynamic effects on all primary HPP biomarkers (PPi, PLP, PEA) in a Phase 1b adult study
- Extensive safety data from >1,000 patients across multiple indications, including healthy volunteers and sepsis‑associated acute kidney injury trials
- Potential to address both bone and extra‑skeletal symptoms due to lack of a bone‑targeting sequence