AstraZeneca’s efzimfotase alfa misses primary endpoint in HICKORY Phase III trial
AstraZeneca reported that its investigational enzyme replacement therapy efzimfotase alfa (ALXN1850) did not achieve statistical significance on the primary Six‑Minute Walk Test (6MWT) endpoint in the HICKORY Phase III trial for hypophosphatasia, showing a least‑squares mean difference of 17.05 metres versus placebo (p = 0.1675). The company highlighted nominally significant improvements in secondary endpoints such as FACIT‑Fatigue and noted a 2.5‑fold higher odds of achieving a clinically meaningful 6MWT response in a post‑hoc analysis. Open‑label extension data indicated continued functional gains through week 49.
Why it matters
The lack of statistical significance on the primary endpoint may delay regulatory approval, while the secondary and subgroup signals could support future filing discussions, according to the company’s statements. AstraZeneca’s shares rose modestly after the release, reflecting market reaction to the mixed results.
Key facts
- 1The trial compared 84 patients receiving efzimfotase alfa with 40 patients on placebo. securities.io
- 2At week 25, the LS mean difference in 6MWT change from baseline was 17.05 metres (SE 12.223; p = 0.1675). securities.io
- 345.2% of treated patients achieved the minimum clinically important difference in 6MWT versus 30.0% on placebo (odds ratio 2.50; 95% CI 1.06‑5.91; nominal p = 0.0363). securities.io
- 4FACIT‑Fatigue showed a nominally significant LS mean difference of 5.74 (SE 1.733; nominal p = 0.0016) versus placebo. securities.io
- 5In the paediatric‑onset subgroup (n = 60), the 6MWT LS mean difference was 35.6 metres (SE 16.87; nominal p = 0.0386). securities.io
- 6AstraZeneca’s shares closed up 1.1% in London and 0.3% in New York on the day of the announcement. marketscreener.com
Summary written by AlphAI AI Desk from 3 of 3 sources. Not investment advice. Figures are as stated by the linked sources.