AstraZeneca studies optimistic knowledge from the Section III trial of efzimfotase alfa
AstraZeneca has reported optimistic outcomes from its efzimfotase alfa Section III medical trial program for hypophosphatasia (HPP), a uncommon metabolic bone illness.
The worldwide program included two placebo-controlled, randomized research and one active-controlled, open-label, randomized pediatric swap research.
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Extra data
196 kids, adolescents and adults with each child- and adult-onset HPP throughout 22 international locations have been included.
Efzimfotase alfa, an investigational enzyme substitute remedy, is designed to offer a smaller injection quantity and fewer frequent dosing in comparison with Strensiq (asfotase alfa).
The MULBERRY Section III trial, concentrating on kids aged two to 12 years with HPP who had not beforehand obtained Strensiq, met its main endpoint.
It confirmed a statistically important and clinically significant enchancment in bone well being at week 25 as measured by radiographic world impression of change (RGI-C) rating in comparison with placebo.
Secondary endpoints, together with rickets severity rating (RSS), bodily perform (six-minute stroll check) and motor ability (Pediatric Outcomes Knowledge Assortment Instrument or PODCI), additionally supported the good thing about efzimfotase alfa on this age group.
Outcomes from the CHESTNUT Section III trial confirmed that efzimfotase alfa was properly tolerated in pediatric sufferers switching from Strensiq and maintained the established remedy profit on bone well being at week 25.
Within the HICKORY Section III research of adolescents and adults 12 years and older with HPP who had not beforehand been handled with Strensiq, efzimfotase alfa confirmed numerical enchancment within the six-minute stroll check, however didn’t attain statistical significance for the first endpoint.
Nonetheless, nominally important enhancements in fatigue discount and mobility have been noticed amongst subgroup populations.
Within the MULBERRY, CHESTNUT and HICKORY research, the protection profile of efzimfotase alfa was acceptable.
Alexion, Marc Dunoyer, CEO of AstraZeneca Uncommon Illness, mentioned: “The efzimfotase alfa medical program, consisting of three world Section III research, was the primary to enroll sufferers with each pediatric and grownup HPP with heterogeneous manifestations past bone.
“We’re inspired by the enhancements noticed on this affected person inhabitants, which displays a variety of severity and medical options. Collectively, these outcomes help the potential of efzimfotase alfa to remodel the remedy paradigm for individuals with this uncommon illness.”
In December 2025, AstraZeneca’s mixture remedy for domestically superior or metastatic non-small cell lung most cancers failed to satisfy the first endpoint of a section III trial.
