REAL-TIME GLOBAL RESEARCH
AstraZeneca PLC (AZN.L): Detailed efzinfotase alfa pediatric data support consensus ests but adult use may offer more upside
Research evidence excerpt
AstraZeneca PLC (AZN.L): Detailed efzinfotase alfa pediatric data support consensus ests but adult use may offer more upside
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29 Jun 2026 03:04:10 ET │ 11 pages
AstraZeneca PLC (AZN.L)
Detailed efzimfotase alfa pediatric data support consensus ests but
adult use may offer more upside
CITI’S TAKE Buy
Detailed late-breaking data from the MULBERRY PIII trial of efzimfotase Price (26 Jun 26 16:30) £143.18
alfa in paediatric (2 to <12 years) hypophosphatasia were presented over Target price £180.00
the weekend at the ICCBH meeting, following positive headline data
Expected share price return 25.7% released late-March. In our view, data supports efzim as self-
administered pediatric treatment Q2W and underscores our $1.2bn peak Expected dividend yield 1.7%
pediatric forecast given more convenient dosing vs AZN’s incumbent Expected total return 27.4%
product Strensiq’s (6-12x Q2W), lower injection volume and lack of Market Cap £222,074M
weight-based dosing. However, we also see upside to risk adjusted US$293,881M
consensus peak sales $1.8bn and c3% to our DCF from potential
adult/adolescent use based on the HICKORY trial, which accounts for
$3bn of our $4.4bn unadjusted peak sales, should full data/publication
(likely ASBMR in October in our view) support filing in paediatric onset Graham ParryAC
adults/adolescents. AZN’s headline PR revealed trial showed clinically +44-20-7508-3661
meaningful benefit in a combination of prespecified subgroups of graham.parry@citi.com
adolescents and adults with paediatric-onset hypophosphatasia despite Amy Y Mao
missing primary endpoint. Maintain Buy.
Leolie Telford-Cooke Ph.D.
Statistically significant and clinically meaningful improvement in children bone Jelena Milovic
health.
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