AstraZeneca's Investigational Drug Shows Durable Clinical Benefit In Patients With Rare Blood Disease

AstraZeneca Plc AZN released results from the 24-week and long-term extension (LTE) period of the ALPHA Phase 3 trial of danicopan as an add-on to C5 inhibitor therapy Ultomiris or Soliris in patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH). A total of 86 patients were randomized. 

The data showed that danicopan as an add-on to the standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) continued to demonstrate clinical benefits for PNH patients.

Data showed that improvements in mean hemoglobin levels and absolute reticulocyte count (ARC) levels, demonstrated at 12 weeks, were maintained through 48 weeks.

Data showed that the significant improvements in hemoglobin levels observed at 12 weeks [LSM (SEM) change 2.94 (0.21) g/dL] continued at 24 weeks [LSM (SEM) change 3.17 (0.30) g/dL] among patients treated with danicopan plus Ultomiris or Soliris and were sustained through 48 weeks.

All key secondary endpoints met superiority in favor of danicopan plus Ultomiris or Soliris compared to placebo plus Ultomiris or Soliris at 12 weeks, and data showed benefits were maintained at 24 weeks in the danicopan-danicopan arm.

All key secondary endpoints showed meaningful improvement at 24 weeks in patients who switched from placebo to add-on treatment with danicopan at 12 weeks, including ARC levels and percentage of patients with transfusion avoidance, two indicators of potential EVH.

Additionally, mean LDH levels were maintained from baseline through 48 weeks in both treatment arms, demonstrating effective control of terminal complement activity and IVH with Ultomiris or Soliris.

Danicopan is generally well tolerated, and no new safety concerns were identified.

Price Action: AZN shares are up 0.28% at $63.43 during the premarket session on the last check Monday.

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