Investigational Drug
Emavusertib
TrialFetch AI Analysis
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Overview
Emavusertib (CA-4948) is an investigational oral drug studied in blood cancers and, in combination with other treatments, in solid tumors. Human results are available, but most are preliminary findings from small, early-stage trials; they do not establish clinical benefit. (ashpublications.org)
Mechanism of action
Emavusertib inhibits interleukin-1 receptor–associated kinase 4 (IRAK4) and also inhibits FLT3. IRAK4 participates in inflammatory signaling that can activate NF-κB, a pathway involved in cancer-cell survival. The rationale for combining emavusertib with the BTK inhibitor ibrutinib is to block two signaling routes implicated in B-cell lymphoma. (ashpublications.org)
Efficacy
- Primary central nervous system lymphoma: In a phase 1/2 trial of emavusertib plus ibrutinib, 11 of 24 patients treated for at least 28 days responded (46%), including six complete responses. Seven of 19 patients previously exposed to a BTK inhibitor responded. These figures exclude patients who stopped treatment before 28 days. A later, company-reported July 2026 update found responses in 10 of 39 previously exposed patients when all treated patients were counted (26%); this update has not been presented here as a peer-reviewed result. (academic.oup.com)
- Higher-risk myelodysplastic syndrome: In a 2024 conference abstract, emavusertib alone produced marrow complete remission in 4 of 13 response-evaluable patients with specified spliceosome mutations. (ashpublications.org)
- Pancreatic cancer: In a 2026 phase 1 abstract, emavusertib with gemcitabine and nab-paclitaxel produced partial responses in 3 of 15 evaluable patients (20%). The study had no comparison group, so the contribution of emavusertib cannot be determined. (ascopubs.org)
Safety
In the lymphoma combination study, 11 of 33 patients (33%) had grade 3 or higher treatment-related adverse events; neutropenia was reported in five (15%). Across 145 patients with acute myeloid leukemia or higher-risk myelodysplastic syndrome treated in the leukemia trial, 41 (28%) had grade 3 or higher treatment-related adverse events. In the pancreatic-cancer combination study, grade 3 neutropenia occurred in 10 of 21 patients (48%), and one had grade 4 pancytopenia. Adverse events in combination studies cannot necessarily be attributed to emavusertib alone. (academic.oup.com)
Further reading
- 2025 lymphoma trial abstract
- 2024 myelodysplastic syndrome trial abstract
- 2026 pancreatic-cancer trial abstract
Last updated: Sep 2026
Active trials using Emavusertib
Found 3 active trials using this drug:
TrialFetch AI summary: Adults with measurable, unresectable or metastatic colorectal adenocarcinoma, ECOG 0–2, and no prior chemotherapy or antiangiogenic therapy may enroll; MSI-H/dMMR disease is excluded. Treatment adds oral emavusertib, an investigational IRAK4/FLT3 inhibitor, to first-line FOLFOX plus bevacizumab, with bevacizumab starting in cycle 1 or 2.
ClinicalTrials.gov ID: NCT06696768
TrialFetch AI summary: Adults with metastatic or unresectable pancreatic ductal adenocarcinoma that progressed on or after fluorouracil-based therapy receive emavusertib, an investigational IRAK4/FLT3 inhibitor, plus gemcitabine and nab-paclitaxel. Prior gemcitabine/nab-paclitaxel for advanced disease is not permitted.
ClinicalTrials.gov ID: NCT05685602
TrialFetch AI summary: Adults with previously untreated unresectable or metastatic gastric, gastroesophageal junction, or esophageal cancer and ECOG 0–1 receive mFOLFOX7 plus emavusertib (CA-4948), an investigational IRAK4/FLT3 inhibitor. HER2-negative patients also receive nivolumab; the HER2-positive expansion cohort receives trastuzumab and pembrolizumab.
ClinicalTrials.gov ID: NCT05187182
