Lixte Biotechnology Holdings, Inc. (Lixte)

is a clinical-stage public (LIXT) pharmaceutical company dedicated to discovering drugs for more effective treatments for cancer. Lixte has identified molecular signaling pathways altered in disease states and designed compounds that can safely target them in animal models. The current drug portfolio includes inhibitors of protein phosphatases that are critical to cell division and DNA damage repair and inhibitors of protein deacetylases that regulate pathways of gene expression and protein degradation.

The phosphatase inhibitors enhance the effectiveness of cytotoxic anti-cancer drugs in general as well as radiation therapy making them potentially useful for the treatment of many, if not all, cancers in combination with existing standard chemotherapy regimens and the evolving targeted cytotoxic therapies of personalized cancer medicine. In addition, in a pre-clinical study done with collaborators at the National Cancer Institute, the lead compound, LB-100, enhanced the antitumor activity on a major immune checkpoint inhibitor raising the possibility that in addition to enhancing the potency of standard cytotoxic anti-cancer drugs, LIXTE’s phosphatase inhibitor may enhance the rapidly developing immunotherapy regimens. LB-100, a novel small molecule PP2A inhibitor, recently completed a Phase 1 clinical trial and has the potential to be first-in-class. In this trial, LB-100 was associated with stabilization of a variety of advanced cancers that had been progressing despite extensive prior treatment without dose-limiting toxicity.

The deacetylase inhibitors have anti-cancer activity and in model systems correct enzymes deficiencies underlying several inborn metabolic diseases including Gaucher and von Hippel-Lindau disease and neurofibromatosis type 2.

Lixte's cancer drug development strategy has led to the discovery of novel compounds with the potential to be therapeutically useful against several other important but seemingly disparate diseases. The phosphatase inhibitors are in pre-clinical development for reducing the extent of tissue damage following stroke, heart attack, and septic shock; and, the deacetylase inhibitors for prevention and treatment of neurodegenerative diseases, traumatic brain injury, and topically for fungal dermatitis (see Product Development).