Lixumistat shows promise as treatment for pancreatic cancer
Oral therapy added to chemotherapy slowed cancer growth in early trial
Treatment with the once-daily oral therapy lixumistat, in addition to chemotherapy, slowed the growth of pancreatic cancer among participants with advanced disease in an early clinical trial, according to new data announced by lixumistat’s developer ImmunoMet Therapeutics.
“Our study of lixumistat … in combination with [chemotherapy] shows quite encouraging clinical effects in this small … study,” Shubham Pant, MD, a trial investigator at the University of Texas MD Anderson Cancer Center, said in a company press release.
The findings were presented at the 2025 American Society of Clinical Oncology Gastrointestinal Cancers Symposium held Jan. 23-25 in San Francisco. The poster was titled “A phase 1b dose escalation trial of gemcitabine and nab-paclitaxel in combination with lixumistat in patients with advanced pancreatic cancer.”
Lixumistat is designed to interfere with cancer cells’ ability to generate energy. The investigational therapy specifically targets the oxidative phosphorylation or OxPhos pathway, which is key for cellular energy generation.
Dean Welsch, CEO of ImmunoMet, called the pancreatic cancer PDAC, fully known as pancreatic ductal adenocarcinoma, “a highly aggressive malignancy defined by high unmet need, morbidity, and mortality,” noting that it’s the third-leading cause of cancer deaths in the U.S.
“There is substantial evidence that differential cancer cell metabolism, and oxidative phosphorylation (OxPhos) in particular, plays a key role in the development of resistance to current therapies,” Welsch said. “Based on these early clinical data we are hopeful that this best-in-class OxPhos inhibitor can improve outcomes for these patients.”
Phase 1 study testing lixumistat as treatment for advanced pancreatic cancer
A Phase 1 clinical trial (NCT05497778) — which is sponsored by the M.D. Anderson Cancer Center — is testing lixumistat in as many as 25 adults. In the first part of the study, patients were given one of two doses of lixumistat (400 or 800 mg/day), in addition to the standard chemotherapy drugs gemcitabine and nab-paclitaxel.
The study’s main goal is to assess safety-related outcomes. The most common side effects related to lixumistat were nausea, vomiting, skin rash, fatigue, and diarrhea, the results showed. These effects were generally not severe, per the researchers. But because substantial side effects were notably more common with the higher dose, the lower dose has been selected for further testing.
These findings suggest that the lixumistat combination may provide a viable novel therapeutic option for advanced PDAC [pancreatic ductal adenocarcinoma], warranting further investigation in larger studies.
To date, eight patients given the low dose of lixumistat were evaluable for efficacy outcomes. In all of them, there was evidence that the lixumistat/chemotherapy combo slowed cancer growth. Specifically, five patients had a partial response, meaning their tumors decreased in size, and the other three had stable disease — their tumors neither grew nor shrank.
The median time without disease progression was more than nine months, and median survival time was 18 months, according to the company.
“These findings suggest that the Lixumistat combination may provide a viable novel therapeutic option for advanced PDAC, warranting further investigation in larger studies,” said Pant, a professor of gastrointestinal medical oncology and investigational cancer therapeutics.
The clinical trial is still enrolling individuals with pancreatic cancer who have not received other treatments. All patients enrolled in the trial from this point will be given the lower dose that’s been selected for further testing. Recruitment is ongoing at the study’s one site in Houston.
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