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AstraZeneca : Efzimfotase alfa demonstrated improvements in bone health in treatment-naïve paediatric patients with hypophosphatasia achieving median difference of 1.67 vs placebo in RGI-C Score at week 25 in MULBERRY Phase III trial
AstraZeneca : Efzimfotase alfa demonstrated improvements in bone health in treatment-naïve paediatric patients with hypophosphatasia achieving median
About this update from Astrazeneca Plc
Efzimfotase alfa demonstrated favourable safety profile and tolerability in treatment-naïve patients and those switching from Strensiq across largest Phase III clinical programme in HPP including MULBERRY, CHESTNUT and HICKORY trialsLate-breaking results support potential of efzimfotase alfa as self-administered treatment taken every two weeks for patients with HPP Positive results from the MULBERRY Phase III trial showed that efzimfotase alfa (ALXN1850), an investigational enzyme replacement therapy, demonstrated a statistically significant and clinically meaningful improvement in bone health in children (2 to <12 years of age) with hypophosphatasia (HPP) who have not been previously treated with Strensiq (asfotase alfa) as measured by Radiographic Global Impression of Change (RGI-C) Score at week 25 compared to placebo.1 These late-breaking results were presented today in an oral session at the 12th International Conference on Children's Bone Health (ICCBH) in Montreal, Canada. In the MULBERRY trial, patients treated with efzimfotase alfa achieved an observed median RGI-C Score of 1.67 at week 25 compared to an observed median score of 0 in the placebo group, with a median difference of 1.67 (95% CI: 0.66, 2.00; p=0.0003).1 The study also met its key secondary endpoint in bone health as measured by Rickets Severity Score (RSS), demonstrating a statistically significant improvement at week 25, with an observed median of -1.00 in the efzimfotase alfa group compared to 0 in the placebo group and a median difference of -1.00 (95% CI: -2.00, -0.33; p=0.0008).1 In addition, efzimfotase alfa demonstrated improvements in secondary endpoints in physical function and quality of life at week 25, including a nominally significant median difference of 9.0 (95% CI: 1.0, 22.0; nominal p=0.0234) in Paediatric Outcomes Data Collection Instrument (PODCI) Global Function normative score and a clinically meaningful improvement in Six-Minute Walk Test (6MWT) with a median difference of 34.5m (95% CI: -27.0, 120.0; nominal p=0.4785) compared to placebo.1 In the CHESTNUT trial, efzimfotase alfa demonstrated a similar incidence of treatment-emergent adverse events (TEAEs) at week 25 in children (2 to <12 years of age) who switched from Strensiq (90.5%) compared to those who remained on Strensiq (86.4%) with a favourable safety profile. Key secondary endpoints showed that...