On July 9, 2025 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical stage pharmaceutical company, reported that the medical journal Nature has published findings by a team of physician-scientists that validate LIXTE’s ongoing clinical trials with its proprietary compound LB100 for Ovarian and Colorectal cancers (View Source) (Press release, Lixte Biotechnology, JUL 9, 2025, View Source [SID1234654349]).
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A team led by principal investigator Amir Jazaeri, MD, professor of Gynecologic Oncology and Reproductive Medicine at The University of Texas MD Anderson Cancer Center, studied survival outcomes of Ovarian Clear Cell Carcinoma (OCCC) patients treated with immune checkpoint blockade therapy (clinicaltrials.gov identifier: NCT03026062). The ;study showed that patients having tumors with inactivating mutations in PPP2R1A – the major scaffold subunit of protein phosphatase 2A (PP2A) – had significantly better overall survival, compared with patients who did not have this mutation in their tumors.
Inactivating mutations in PPP2R1A are known to reduce the enzymatic activity of PP2A, which is the target of LIXTE’s lead compound LB-100. Tumors with mutations in PPP2R1A were found to have increased the interferon gamma response pathway, which is known to be associated with improved immune checkpoint responses.
LIXTE is currently investigating the activity of LB-100 in combination with checkpoint immunotherapy in two clinical trials. The first is enrolling patients with OCCC, led by Dr. Jazaeri at MD Anderson Cancer Center, and also is open at Northwester University. In this trial, LIXTE is collaborating with GSK to test LB-100 in combination with dostarlimab (anti PD1). In the second trial, at the Netherlands Cancer Institute, LIXTE is collaborating with Roche to test LB-100 in combination with atezolizumab (anti PDL1) in colon cancer patients.
"Not only did we identify a new biomarker for improved survival with immunotherapy in ovarian cancer, but we also confirmed the correlation of this biomarker with survival benefit in other cancer types," said Dr. Jazaeri, who was co-senior author of the Nature article. "Since PPP2R1A mutations are relatively uncommon, we believe the same benefits may be possible by targeting the PPP2A pathway using drugs, which we currently are evaluating in a clinical trial at MD Anderson."
Bas van der Baan, LIXTE’s Chief Scientific Officer, added, "This work extends a body of pre-clinical evidence indicating that LB-100 is strongly synergistic with checkpoint immunotherapy in a range of cancer types. We look forward to the first results of our clinical studies in the second half of this year."