Shots:
- The P-III (HICKORY) trial data assessed efzimfotase alfa (ALXN1850; Q2W, SC) vs PBO in 124 pts (≥12yrs.) with HPP who have not been previously treated with Strensiq;data were presented at ASBMR 2026
- At Wk. 25, ALXN1850 did not significantly improve 6MWT (1EP), achieving a LS mean difference of 17.05m, while FACIT-Fatigue showed nominally significant improvement; 30s-STS, LEFS & pain severity numerically favoured treatment. Post hoc analysis showed 45.2% achieved 6MWT MCID vs 30%
- Paediatric-onset pts showed nominally significant improvements in 6MWT, 30s-STS and pain severity, while adults improved in LEFS & FACIT-Fatigue. In the OLE, ALXN1850 sustained improvements through wk. 49, with pts switching from PBO at Wk. 25 showing similar benefits in mobility, function, pain & fatigue
Ref: AstraZeneca | Image: AstraZeneca | Press Release
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