Target, across the briefings

BTK across 6 cancers

BTK is on the target list of 6 of the briefings. A drug aimed at it carries an FDA label naming the disease in 2, and is approved for some indication in 6. In the reference cohorts of the 5 with a mutation landscape it is altered in at least 5% of patients in 1 and in under 2% in 3 (Mantle cell lymphoma, Chronic lymphocytic leukemia, Marginal zone lymphoma) — a target there by expression, dependency or drug label, not by mutation.

DiseaseLabel statusFurthest stage (Open Targets)Distinct drugsCell-therapy trialsAltered in the reference cohort
Mantle cell lymphomaapproved here Acalabrutinibapproved205 via BTK0.0% mut n=29
Chronic lymphocytic leukemiaapproved here Acalabrutinibapproved202 via BTK0.19% mut n=1074
Marginal zone lymphomaapproved elsewhereapproved201 via BTK0.0% mut n=43
Diffuse large B-cell lymphomaapproved elsewhereapproved20—2.8% mut n=1001
Follicular lymphomaapproved elsewhereapproved20—6.22% mut n=193
Waldenström macroglobulinaemiaapproved elsewhereapproved200 via BTKno landscape

"Approved here" means a drug Open Targets records as aimed at BTK carries an FDA label that names that disease, per the briefing's openFDA stage. "Approved elsewhere" means the drug is approved for some other indication. Stage is the furthest Open Targets clinical stage among drugs aimed at the gene, in any indication; the database is reported as it is, and it lags some approvals. Cell-therapy trials are counted on the antigen the briefing maps the gene to, from ClinicalTrials.gov. The last column is the disease's mutation landscape: non-silent mutation, high-level amplification and deep deletion as percentages of the reference cohort's patients, each over its own denominator; "not on panel" means the reference cohort could not have called the gene.