AstraZeneca (AZN) AZN Meet The Management at ASBMR 2026 summary

Strategic priorities and rare disease focus

• Long-range plan targets $80bn total revenue by 2030 and sustained growth beyond 2030; ambition is risk-adjusted and not dependent on future M&A.

• Rare disease plans prioritize high-unmet-need areas with best- or first-in-class potential; >10,000 rare diseases are known, and fewer than 10% have approved treatments.

• Priority areas include haematology, nephrology and transplant, neurology, bone and endocrine disorders, cardiology and amyloidosis, and rare tumours.

HPP and efzimfotase alfa profile

• HPP is a lifelong, inherited multisystem disorder caused by deficient alkaline phosphatase; manifestations include skeletal disease, fractures, pain, fatigue, weakness, respiratory failure and tooth loss.

• Efzimfotase alfa is a second-generation ERT designed for HPP patients aged >2 years across the disease spectrum; Q2W dosing contrasts with 3–6 weekly Strensiq injections.

• Phase III included 196 patients across 22 countries: treatment-naive children, patients switching from Strensiq, and treatment-naive adolescents and adults; studies included MULBERRY, HICKORY and CHESTNUT.

• MULBERRY week-25 median RGI-C was +1.67 with efzimfotase alfa versus 0 with placebo (p=0.0003), with improvements in physical function, motor proficiency and quality of life.

• CHESTNUT met its primary safety objective; adverse-event incidence was similar after switching from asfotase alfa, and bone-health, physical-function and quality-of-life benefits were maintained.

HICKORY Phase III findings

• HICKORY randomized 124 treatment-naive patients aged ≥12 years; at week 25, 6MWT change was 33.7m versus 23.7m with placebo (LS mean difference 17.05m, p=0.1675), missing the primary endpoint.

• In the open-label extension, 6MWT distance increased 61.9m by week 49 for patients on efzimfotase alfa and 43.0m after placebo patients switched at week 25.

• Secondary outcomes favored efzimfotase alfa; FACIT-Fatigue improved significantly (difference 5.74, nominal p=0.0016), alongside improvements in pain interference and treatment satisfaction.

• Pediatric-onset patients had significant improvements in 6MWT, sit-to-stand and pain; adult-onset patients had significant FACIT-Fatigue improvement.