AgentNipocalimab (anti-FcRn)

MechanismLowers pathogenic IgG

EfficacyDurable Hgb response ~3x placebo

StatusFDA Priority Review (wAIHA)

PositiveNot yet (filing)

ENERGY

NCT04119050 · S300 · Fattizzo · phase 2/3

• Nipocalimab (anti-FcRn) vs placebo in warm autoimmune haemolytic anaemia
• Durable haemoglobin response roughly three times placebo by week 24 (about 24% vs 8%)
• Mean haemoglobin improvement of at least 1 g/dL by week 1
• Already marketed as Imaavy for myasthenia gravis; wAIHA filing holds FDA Priority Review

Why it matters: The first targeted randomised data in warm AIHA, a condition with no licensed targeted therapy.

Early phaseProof of concept

BCMA CAR-T in ITP

LB5003 · Shu · 8 patients dosed

• BCMA-directed CAR-T for refractory primary immune thrombocytopenia
• 8 dosed, 4 evaluable, all 4 achieved complete response
• 1 patient relapsed at 9 months
• No severe adverse events reported

Why it matters: Targeting the plasma cells behind pathogenic antibody is a landmark concept, but the numbers are tiny and lymphodepletion in thrombocytopenia carries risk.

Targeted therapy is reaching conditions once managed only with broad immunosuppression.Nipocalimab in warm AIHA and BCMA CAR-T in ITP.