▎ WuXi AppTec content team editor
Today, AstraZeneca and Argenx respectively announced that their research therapies have reached key endpoints in Phase 3 clinical trials to treat autoimmune diseases.
Ultomiris (ravulizumab-cwvz), a long-acting complement C5 protein inhibitor developed by Alexion, a subsidiary of AstraZeneca, achieved a primary endpoint in a Phase 3 clinical trial for the treatment of optic neuromyelitis spectrum disease (NMOSD), significantly reducing the risk of recurrence in patients with anti-AQP4 antibody-positive NMOSD compared with the external placebo group.
NMOSD is a rare disability central nervous system (CNS) autoimmune disease that affects hundreds of thousands of people worldwide. The patient's main symptom is inflammatory lesions of the optic nerve and spinal cord. Patients with NMOSD often experience disease recurrence, and repeated attacks on their own tissues by the immune system can lead to a gradual accumulation of nerve damage and disability.
Complement protein C5 is at the end of the complement cascade, so targeting this protein regulates complement signaling activated by all 3 different pathways. In a variety of complement-mediated immune diseases, complement-mediated immune responses attack the patient's own healthy tissues and cells, leading to damage to different tissues and organs. Inhibiting the activity of C5 can inhibit the immune attack on itself, thereby alleviating the symptoms of the disease. Ultomiris has received FDA approval to treat a variety of autoimmune diseases, including paroxysmal sleepy hemoglobinuria and myasthenia gravis.
The results of this clinical trial showed that at a median treatment duration of 73 weeks, no recurrence of the disease was observed in the 58 patients treated with Ultomiris, reaching the primary endpoint of the trial. Detailed data will be released at future medical conferences and submitted to global regulators as soon as possible.
Argenx corporation announced that its antibody therapy Vyvgart (efgartigimod alfa-fcab), which targets neonatal Fcrns, has reached a primary endpoint in a Phase 3 clinical trial for the treatment of immune thrombocytopenia (ITP), with a significantly higher proportion of patients achieving sustained platelet response compared to placebo.
ITP is an autoimmune disease in which IgG autoantibodies attack platelets and reduce platelet production, leading to an increased risk of excessive bleeding and, in severe cases, can lead to anemia and even bleeding in the brain. Symptoms in many ITP patients are currently not fully controlled.
Vyvgart is a human IgG antibody fragment that reduces the level of IgG autoantibodies in the blood circulation by binding to FcRn. It has received FDA approval for the treatment of generalized myasthenia gravis.
The trial results showed that 21.8% (17/78) of the ITP patient group treated with Vyvgart met the platelet sustained response criteria, significantly better than the placebo group (5%, p=0.0316). Vyvgart also reached a series of key secondary finishes.
"Immune thrombocytopenia is an incapacitated autoimmune disease that currently has no clear standard of treatment." The lead investigator of the clinical trial, Professor Catherine Broome of Georgetown University, said, "These data show that platelet levels can improve rapidly to clinically significant levels after receiving Vyvgart treatment. We are excited that targeting pathogenic IgG autoantibodies may represent potential new strategies to help this patient population." ”
Resources:
[1] argenx Announces Positive Phase 3 Data from ADVANCE Trial of VYVGART (efgartigimod alfa-fcab) in Adults with Primary Immune Thrombocytopenia. Retrieved May 5, 2022, from https://www.globenewswire.com/news-release/2022/05/05/2436303/0/en/argenx-Announces-Positive-Phase-3-Data-from-ADVANCE-Trial-of-VYVGART-efgartigimod-alfa-fcab-in-Adults-with-Primary-Immune-Thrombocytopenia.html
[2] Ultomiris met primary endpoint in CHAMPION-NMOSD Phase III trial in adults with neuromyelitis optica spectrum disorder. Retrieved May 5, 2022, from https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/ultomiris-nmosd-ph-iii-trial-met-primary-endpoint.html
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