ONCOLOGY

Finding the Signal Inside a 17-Patient Trial

ARGX-111, an anti-MET monoclonal antibody for solid tumors, showed limited overall clinical activity in a Phase I dose-escalation study involving 17 patients.

Challenge

Approach

KEM® explored 2,719 logical relationships across 165 variables - clinical, cytokine, pharmacokinetic, and safety data from 17 patients, plus 40 preclinical animal studies - using association-rule mining to identify biomarker combinations predictive of treatment response and toxicity.

Results

KEM® identified a small but highly responsive patient subgroup at dose level #3, along with a pre-inflammatory cytokine profile strongly predictive of both anti-tumor efficacy and adverse events.

Business Outcome

Ariana's analysis informed the scientific understanding that supported argenx's decision to prioritize ARGX-113 over continued development of ARGX-111. ARGX-113 (efgartigimod) was subsequently developed into Vyvgart®, now approved in multiple autoimmune diseases, including generalized myasthenia gravis (gMG), chronic inflammatory demyelinating polyneuropathy (CIDP), and immune thrombocytopenia (ITP), and is argenx's flagship product and a multi-billion-dollar commercial success.