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 serine/threonine 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 cancers in combination with existing standard chemotherapy regimens and with the evolving targeted cytotoxic therapies of personalized cancer medicine. In addition, in a pre-clinical study done with scientists under collaborative research and development agreements at the National Institutes of Health, the lead compound, LB-100, enhanced the antitumor activity of a major immune checkpoint inhibitor raising the possibility that in addition to improving the efficacy of standard cytotoxic anti-cancer drugs, LIXTE’s phosphatase inhibitors may potentiate immunotherapy regimens (Ho 2017). LB-100 recently completed a Phase 1 clinical trial in which it was associated with objective regression/or stabilization of advanced cancers that had been progressing despite extensive prior treatment without dose-limiting toxicity (Chung 2016). The next step is to demonstrate the activity of LB-100 against specific cancers in Phase 2 studies.

Lixte's strategy of attacking novel enzyme targets has unexpectedly led to the discovery that its phosphatase inhibitors may be therapeutically useful not only in cancer but for treatment of important non-neoplastic diseases including stroke and heart attack, vascular complications of type 2 diabetes, and severe depression.

The deacetylase inhibitors show preclinical activity in models for prevention and treatment of neurodegenerative diseases, traumatic brain injury, and topically, for fungal dermatitis. The deacetylase inhibitors are currently not under active development by Lixte and are available for licensing (see Drug Pipeline).