Two Innovative Therapies for Autoimmune Diseases Achieve Phase 3 Primary Endpoints and Feature in Recent IPO Filings

Today, AstraZeneca and argenx announced separately that their investigational therapies met the primary endpoint in Phase 3 clinical trials for autoimmune diseases.

Ultomiris (ravulizumab-cwvz), a long-acting complement C5 protein inhibitor developed by Alexion, a subsidiary of AstraZeneca, met the primary endpoint in a Phase 3 clinical trial for the treatment of neuromyelitis optica spectrum disorder (NMOSD). Compared with the external placebo group, it significantly reduced the relapse risk in anti-AQP4 antibody-positive NMOSD patients.

NMOSD is a rare disabling autoimmune disease of the central nervous system (CNS), affecting up to hundreds of thousands of people worldwide. The main symptoms in patients are inflammatory lesions in the optic nerve and spinal cord. Patients with NMOSD often experience disease relapses, where repeated attacks by the immune system on their own tissues lead to the gradual accumulation of nerve damage and disability.

Complement protein C5 is at the terminal end of the complement cascade, so targeting this protein can regulate complement signals activated by all three different pathways. In various complement-mediated immune diseases, complement-mediated immune responses attack the patient's own healthy tissues and cells, causing damage to different tissues and organs. Inhibiting the activity of C5 can suppress autoimmune attacks, thereby alleviating disease symptoms. Ultomiris has been approved by the FDA for the treatment of multiple autoimmune diseases, including paroxysmal nocturnal hemoglobinuria and myasthenia gravis.

The results of this clinical trial showed that at a median treatment duration of 73 weeks, no disease relapse was observed in the 58 patients treated with Ultomiris, achieving the primary endpoint of the trial. Detailed data will be presented at future medical conferences and promptly submitted to global regulatory authorities.

argenx Announces Vyvgart (efgartigimod alfa-fcab), an Antibody Therapy Targeting the Neonatal Fc Receptor (FcRn), Meets Primary Endpoint in Phase 3 Clinical Trial for Immune Thrombocytopenia (ITP), with a Significantly Higher Proportion of Patients Achieving Sustained Platelet Response Compared to Placebo.

ITP is an autoimmune disease where IgG autoantibodies attack platelets and reduce platelet production, increasing the risk of excessive bleeding. In severe cases, it can lead to anemia or even intracerebral hemorrhage. Currently, the symptoms of many ITP patients remain not fully controlled.

Vyvgart is a human IgG antibody fragment that reduces the levels of IgG autoantibodies in the bloodstream by binding to FcRn. It has been approved by the U.S. FDA for the treatment of generalized myasthenia gravis.

Trial results showed that 21.8% (17/78) of ITP patients in the Vyvgart-treated group achieved sustained platelet response, significantly superior to the placebo group (5%, p=0.0316). Vyvgart also met a series of key secondary endpoints.

"Immune thrombocytopenia is a disabling autoimmune disease with no clear standard treatment," said Professor Catherine Broome of Georgetown University, the principal investigator of this clinical trial. "These data show that platelet levels can rapidly improve to clinically significant levels after Vyvgart treatment. We are excited that targeting pathogenic IgG autoantibodies may represent a potential new strategy to help this patient population."

References:

[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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