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AZN Posts Data From Hypophosphatasia Program on Efzimfotase Alfa
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Key Takeaways
AstraZeneca's efzimfotase alfa met primary endpoints in MULBERRY, improving bone health versus placebo.
CHESTNUT study showed maintained benefits and favorable safety in children switching from Strensiq.
HICKORY study missed the primary endpoint but showed mobility, pain, and function gains in some HPP patients.
AstraZeneca (AZN - Free Report) announced positive data from a late-stage clinical program that consisted of three phase III studies evaluating its pipeline candidate, efzimfotase alfa (ALXN1850), for treating certain patients with hypophosphatasia (HPP), a rare, inherited metabolic disease.
The clinical program consisted of the phase III MULBERRY study, which evaluated efzimfotase alfa in children aged two to less than 12 years with HPP who have not been previously treated with AZN’s HPP drug, Strensiq (asfotase alfa). The phase III CHESTNUT study evaluated efzimfotase in pediatric patients aged two to less than 12 years with HPP, who have been treated with 6 mg/kg per week of Strensiq for at least six months before the study began.
Data from AZN’s MULBERRY & CHESTNUT Studies
Data from the MULBERRY study showed that treatment with efzimfotase alfa led to a statistically significant and clinically meaningful improvement in bone health from baseline versus placebo, as measured by the Radiographic Global Impression of Change (RGI-C) score at week 25, the study’s primary endpoint.
Additionally, there was a significant improvement in the key secondary endpoint, the change from baseline in Rickets Severity Score (RSS) at week 25. Other secondary measures, including physical function (Six-Minute Walk Test) and motor proficiency, also supported the overall clinical benefit of efzimfotase alfa in the given patient population.
Meanwhile, data from the CHESTNUT study showed that treatment with efzimfotase alfa was well tolerated and had a favorable safety profile in children aged two to under 12 who switched from Strensiq, while maintaining the treatment benefit of Strensiq on bone health at week 25.
Year to date, shares of AstraZeneca have risen 7.2% compared with the industry’s rise of 0.7%.
Image Source: Zacks Investment Research
Data from AZN’s HICKORY Study on Efzimfotase Alfa
The above-mentioned clinical program also included the phase III HICKORY study, which investigated efzimfotase alfa in adolescents aged 12 to less than 18 years and adults with HPP who had not been previously treated with Strensiq.
Data from the same showed a numerical improvement following treatment with efzimfotase alfa but failed to achieve statistical significance in the primary endpoint of Six-Minute Walk Test (6MWT) versus placebo at week 25 in the given patient population.
In adolescents and adults with pediatric-onset HPP, treatment with efzimfotase alfa demonstrated nominally statistically significant and clinically meaningful benefits in mobility, as measured by 6MWT, as well as key secondary endpoints measuring physical function and pain reduction versus placebo.
Management said that data from the HICKORY study showed meaningful improvements in mobility, physical function, pain, and fatigue, highlighting the potential of efzimfotase alfa to improve patient outcomes.
Overall, across the three studies, treatment with efzimfotase alfa was generally well-tolerated and had an acceptable safety profile.
Management plans to present data from these studies at an upcoming medical conference and share the same with regulatory bodies worldwide.
Efzimfotase alfa is an investigational enzyme replacement therapy designed to offer smaller injection volumes and less frequent dosing than Strensiq, while improving care for HPP patients.
AZN’s Zacks Rank & Stocks to Consider
AstraZeneca currently carries a Zacks Rank #3 (Hold).
Over the past 60 days, estimates for Prothena’s 2026 earnings per share have increased from 20 cents to 36 cents, while loss per share estimates for 2027 have narrowed from $1.20 to 98 cents. PRTA’s shares have risen 1.7% year to date.
Prothena’s earnings beat estimates in one of the trailing four quarters and missed on the remaining three occasions, with the average negative surprise being 24.69%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ 2026 earnings per share have increased from $8.28 to $8.99, while the same for 2027 have increased from $9.25 to $10.10. ANIP’s shares have fallen 2.6% year to date.
ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have increased from 63 cents to $1.75, while the same for 2027 have increased from $1.55 to $2.91. LQDA’s shares have risen 9.4% year to date.
Liquidia’s earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, with the average surprise being 39.38%.
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AZN Posts Data From Hypophosphatasia Program on Efzimfotase Alfa
Key Takeaways
AstraZeneca (AZN - Free Report) announced positive data from a late-stage clinical program that consisted of three phase III studies evaluating its pipeline candidate, efzimfotase alfa (ALXN1850), for treating certain patients with hypophosphatasia (HPP), a rare, inherited metabolic disease.
The clinical program consisted of the phase III MULBERRY study, which evaluated efzimfotase alfa in children aged two to less than 12 years with HPP who have not been previously treated with AZN’s HPP drug, Strensiq (asfotase alfa). The phase III CHESTNUT study evaluated efzimfotase in pediatric patients aged two to less than 12 years with HPP, who have been treated with 6 mg/kg per week of Strensiq for at least six months before the study began.
Data from AZN’s MULBERRY & CHESTNUT Studies
Data from the MULBERRY study showed that treatment with efzimfotase alfa led to a statistically significant and clinically meaningful improvement in bone health from baseline versus placebo, as measured by the Radiographic Global Impression of Change (RGI-C) score at week 25, the study’s primary endpoint.
Additionally, there was a significant improvement in the key secondary endpoint, the change from baseline in Rickets Severity Score (RSS) at week 25. Other secondary measures, including physical function (Six-Minute Walk Test) and motor proficiency, also supported the overall clinical benefit of efzimfotase alfa in the given patient population.
Meanwhile, data from the CHESTNUT study showed that treatment with efzimfotase alfa was well tolerated and had a favorable safety profile in children aged two to under 12 who switched from Strensiq, while maintaining the treatment benefit of Strensiq on bone health at week 25.
Year to date, shares of AstraZeneca have risen 7.2% compared with the industry’s rise of 0.7%.
Image Source: Zacks Investment Research
Data from AZN’s HICKORY Study on Efzimfotase Alfa
The above-mentioned clinical program also included the phase III HICKORY study, which investigated efzimfotase alfa in adolescents aged 12 to less than 18 years and adults with HPP who had not been previously treated with Strensiq.
Data from the same showed a numerical improvement following treatment with efzimfotase alfa but failed to achieve statistical significance in the primary endpoint of Six-Minute Walk Test (6MWT) versus placebo at week 25 in the given patient population.
In adolescents and adults with pediatric-onset HPP, treatment with efzimfotase alfa demonstrated nominally statistically significant and clinically meaningful benefits in mobility, as measured by 6MWT, as well as key secondary endpoints measuring physical function and pain reduction versus placebo.
Management said that data from the HICKORY study showed meaningful improvements in mobility, physical function, pain, and fatigue, highlighting the potential of efzimfotase alfa to improve patient outcomes.
Overall, across the three studies, treatment with efzimfotase alfa was generally well-tolerated and had an acceptable safety profile.
Management plans to present data from these studies at an upcoming medical conference and share the same with regulatory bodies worldwide.
Efzimfotase alfa is an investigational enzyme replacement therapy designed to offer smaller injection volumes and less frequent dosing than Strensiq, while improving care for HPP patients.
AZN’s Zacks Rank & Stocks to Consider
AstraZeneca currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Prothena (PRTA - Free Report) , ANI Pharmaceuticals (ANIP - Free Report) and Liquidia Corporation (LQDA - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Over the past 60 days, estimates for Prothena’s 2026 earnings per share have increased from 20 cents to 36 cents, while loss per share estimates for 2027 have narrowed from $1.20 to 98 cents. PRTA’s shares have risen 1.7% year to date.
Prothena’s earnings beat estimates in one of the trailing four quarters and missed on the remaining three occasions, with the average negative surprise being 24.69%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ 2026 earnings per share have increased from $8.28 to $8.99, while the same for 2027 have increased from $9.25 to $10.10. ANIP’s shares have fallen 2.6% year to date.
ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have increased from 63 cents to $1.75, while the same for 2027 have increased from $1.55 to $2.91. LQDA’s shares have risen 9.4% year to date.
Liquidia’s earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, with the average surprise being 39.38%.