Two innovative therapies for the treatment of autoimmune diseases reach phase 3 clinical endpoints

Today, AstraZeneca and argenx announced that their investigational therapy met the primary endpoint in a Phase 3 clinical trial for the treatment of autoimmune diseases

Ultomiris (ravulizumab-cwvz), a long-acting complement C5 protein inhibitor developed by AstraZeneca's Alexion, met the primary endpoint in a Phase 3 clinical trial in the treatment of neuromyelitis optica spectrum disease (NMOSD), compared with an external placebo group.

NMOSD is a rare disabling central nervous system (CNS) autoimmune disease that affects up to hundreds of thousands of people worldwide

Complement protein C5 is at the end of the complement cascade, so targeting this protein modulates complement signaling activated by all three distinct pathways

Results from this clinical trial showed that no disease recurrence was observed in the 58 patients treated with Ultomiris at a median treatment duration of 73 weeks, meeting the trial's primary endpoint

argenx announced that its antibody therapy Vyvgart (efgartigimod alfa-fcab) targeting the neonatal Fc receptor (FcRn) met the primary endpoint in a Phase 3 clinical trial for the treatment of immune thrombocytopenia (ITP), comparable to placebo The proportion of patients who achieved sustained platelet remission was significantly higher

ITP is an autoimmune disease in which IgG autoantibodies attack platelets and reduce their production, leading to an increased risk of excessive bleeding and, in severe cases, anemia and even bleeding in the brain

Vyvgart is a human IgG antibody fragment that reduces circulating levels of IgG autoantibodies by binding to FcRn

The results of the trial showed that 21.

"Immune thrombocytopenia is a disabling autoimmune disease for which there is currently no clear standard of care

References:

[1] argenx Announces Positive Phase 3 Data from ADVANCE Trial of VYVGART® (efgartigimod alfa-fcab) in Adults with Primary Immune Thrombocytopenia.

[2] Ultomiris met primary endpoint in CHAMPION-NMOSD Phase III trial in adults with neuromyelitis optical spectrum disorder.