New: 50% response rate at RP2D in pancreatic cancer, presented at ESMO GI 2025
Clinical · Phase 1b/2a · NCT05497778

Clinical evidence in pancreatic cancer

A study conducted by Dr. S. Pant at MD Anderson to characterize the safety and early signs of efficacy of lixumistat in combination with standard of care in patients with advanced pancreatic cancer recently completed (NCT05497778). This study defined the RP2D and provided impressive interim evidence of clinical activity (ESMO GI, July 2025).

50%
Response Rate at RP2D
5 of 10 evaluable patients
80%
Disease Control Rate
First 10 evaluable at RP2D
N=25
Total Study Size
COMBAT-PC · MD Anderson
No DLTs
Dose-Limiting Toxicities Observed
Across 100mg QOD to 1,200mg QD
Basic Science · OxPhos Biology · Metabolic Oncology

Lixumistat in Advanced Pancreatic Cancer

Lixumistat in combination with gemcitabine and nab-paclitaxel (standard of care) in patients with advanced PDAC. Principal Investigator: Dr. Shubham Pant, MD Anderson Cancer Center. Registry: NCT05497778.

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COMBAT-PC clinical study design: dose escalation phase (N=12) and cohort expansion phase, gemcitabine plus nab-paclitaxel plus lixumistat, with safety/tolerability primary and ORR/PFS/OS secondary endpoints
Clinical study design · COMBAT-PCNCT05497778
Early Results

Encouraging Efficacy Signal at the Recommended Dose

Among the first 10 evaluable patients at the RP2D, the clinical response (ORR) and disease control (DCR) rates were 50% and 80%, respectively. Median progression free survival (PFS) and overall survival (OS) were 7.4 and 18 months, respectively. Importantly, there were no dose limiting toxicities observed at the RP2D.

Data presented at ESMO GI (July 2025). Interim publication in progress.

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ESMO GI 2025 abstract 263P: a Phase 1b dose-escalation trial of gemcitabine and nab-paclitaxel in combination with lixumistat in advanced pancreatic cancer (COMBAT-PC), Welsch et al., MD Anderson
Abstract 263P · COMBAT-PC · ESMO GI 2025 · Welsch et al., MD Anderson — click to view the full PDF
Clinical Programme

Two near-term data inflection points.

Proof of concept in pancreatic cancer, with glioblastoma as the next planned indication. We welcome conversations with investors and partners.