[{"id":"gami-ep1","name":"Overall response rate (previously treated)","type":"PRIMARY","unit":"%","p_value":"0.013 (vs null hypothesis of 40%)","results":[{"n":"17/27","ci":"42-81","arm":"gami-ptx","label":"63% (17/27)","value":63}],"description":"ORR in previously treated primary HLH patients. HLH is a life-threatening hyperinflammatory condition, mostly in infants. Any response that bridges to curative bone marrow transplant (HSCT) is clinically meaningful."}]

Infections 56% (expected given HLH-associated immunosuppression). Infusion reactions 27% (8% mild, 5% moderate; one-third at first infusion). Hypertension 41%. Pyrexia 24%. 1 discontinuation due to AE (disseminated histoplasmosis). Median time to response: 8 days.

FDA accelerated approval Nov 2018. Small N (34) reflects ultra-rare disease. 63% ORR with 70% bridging to HSCT in previously treated primary HLH is clinically meaningful. The big optionality is expanding to IDS (sepsis) via EMBRACE - massive TAM but very early and high-risk.

Anti-IFN-gamma monoclonal antibody. Primary HLH is driven by uncontrolled IFN-gamma production from hyperactivated T cells and NK cells. Neutralizing IFN-gamma calms the cytokine storm and allows bridging to curative HSCT.