Treatment of active idiopathic inflammatory myopathies by inhibiting FcRn: Pre-registration report of ALKIVIA, a phase 2/3 trial with efgartigimod

Samantha Griffiths

Treatment of active idiopathic inflammatory myopathies by inhibiting FcRn: Pre-registration report of ALKIVIA, a phase 2/3 trial with efgartigimod

Autori

ROHIT AGGARWAL (UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE, PITTSBURGH, PHILADELPHIA, USA – )
MASSIMILIANO MIRABELLA (UOC NEUROLOGIA, FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS, ROME; DIPARTIMENTO DI NEUROSCIENZE, SEZIONE DI NEUROLOGIA, CATHOLIC UNIVERSITY OF SACRED HEART, ROME, ITALY – NEUROLOGY)
ANTHONY A. AMATO (BRIGHAM AND WOMEN’S HOSPITAL HARVARD MEDICAL SCHOOL, BOSTON, MASSACHUSETTS, USA; – )
DESPOINA PAPADOPOULOU (ARGENX, GHENT, BELGIUM – )
BAS VAN DER WONING (ARGENX, GHENT, BELGIUM – )
PAUL DUNCOMBE (ARGENX, GHENT, BELGIUM – )
INGRID E. LUNDBERG (ARGENX, GHENT, BELGIUM – )

Presentatore

MASSIMILIANO MIRABELLA (UOC NEUROLOGIA, FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS, ROME; DIPARTIMENTO DI NEUROSCIENZE, SEZIONE DI NEUROLOGIA, CATHOLIC UNIVERSITY OF SACRED HEART, ROME, ITALY)

Modalità

Oral Communication

Abstract

“Introduction
Idiopathic inflammatory myopathies (IIM) include a potentially IgG-mediated group of diseases targeting muscle, skin, and other organs. Efgartigimod, a human IgG1 antibody Fc fragment that reduces total IgG and pathogenic IgG autoantibody levels through neonatal Fc receptor blockade, is being studied as a therapy for active IIM in the global phase 2/3 randomized, double‑blind, placebo‑controlled ALKIVIA trial.

Objective
To evaluate the efficacy and safety of subcutaneous (SC) efgartigimod PH20 (co-formulated with recombinant human hyaluronidase PH20) treatment compared with placebo in IIM, in addition to standard-of-care therapy.

Methods
Eligible participants (adults with active immune-mediated necrotizing myopathy, dermatomyositis, or polymyositis on stable oral corticosteroids and/or a single antimalarial/immunosuppressant) are recruited to the 24-week, phase 2 proof-of-concept (90 participants) or the consecutive, confirmatory, 52-week phase 3 stage (150 participants). Participants are randomized (1:1) to receive efgartigimod PH20 SC 1000 mg or placebo PH20 SC weekly, in addition to standard care. Participants completing either stage may roll over into the open-label extension.

Results
The primary endpoint (total improvement score [TIS]) using the 2016 American College of Rheumatology/European League Against Rheumatism myositis response criteria will be assessed at weeks 24 (phase 2) and 52 (phase 3). Key secondary endpoints include minimal/moderate response per the TIS and time to response. Other secondary outcomes (muscle strength/endurance, pain, fatigue, and corticosteroid sparing effect) will be evaluated. Safety will be assessed by incidence and severity of adverse events.

Conclusion
ALKIVIA aims to identify potentially IgG-driven IIM subtypes by evaluating the efficacy and safety of efgartigimod PH20 SC in IIM overall and in different subtypes.”