AstraZeneca's Alexion bet continues to pay off as danicopan improves hemoglobin levels in phase 3

AstraZeneca’s $39 billion acquisition of Alexion last year continues to look like money well spent. The latest good news is that the oral factor D inhibitor danicopan can improve hemoglobin levels when added to the approved drugs Ultomiris or Soliris.

The interim analysis of the phase 3 ALPHA trial looked at danicopan as an add-on C5 inhibitor therapy for 63 patients with a blood disease called paroxysmal nocturnal hemoglobinuria (PNH) who experience extravascular haemolysis (EVH). EVH occurs when a type of white blood cell normally found in the liver and spleen ingests antibody-coated red blood cells.

The trial met its primary endpoint of changing hemoglobin levels from baseline at 12 weeks. The study also hit key secondary endpoints including patients not requiring a blood transfusion and a reduction in fatigue, according to the press release.

The combination of danicopan plus Ultomiris or Soliris demonstrated superiority compared to the two approved drugs and a placebo. Danicopan was generally well tolerated, and there were no clinically meaningful differences in safety results observed between the danicopan and placebo cohorts.

“C5 inhibitors are a proven treatment option for patients living with PNH, yet a small percentage may continue to experience anemia and burden of transfusion due to clinically significant EVH; however, it is not life-threatening,” said Catholic University of Korea’s Jong-Wook Lee, M.D., Ph.D., who was the trial’s investigator.

“These data show that danicopan has the potential to resolve clinically significant EVH while allowing patients to remain on standard of care treatment with Ultomiris or Soliris,” Lee added in the Sept. 16 release.

Like danicopan, Soliris was also acquired by AstraZeneca from rare disease-focused Alexion, who secured its approval as the first PNH treatment back in 2007. A decade later, AstraZeneca’s Ultomiris was also approved for the condition. As a result of the U.K.-based Big Pharma’s acquisition of Boston-based Alexion in 2021, AstraZeneca now has the option of trialing combinations of the therapies.

Of the two approved therapies, Soliris has proved the biggest earner in recent months, bringing in a total of over $2 billion in the first half of the year compared to $853 million from Ultomiris.

Danicopan has already been granted breakthrough and orphan-drug tags by the FDA. Alexion is also evaluating the drug in a phase 2 trial as a monotherapy for a type of macular degeneration called geographic atrophy.

Another asset from the Alexion acquisition that has proved successful is ALXN1840, which AstraZeneca sees as a potential blockbuster in a rare genetic disorder called Wilson disease.