LIXTE Biotechnology Holdings, Inc.

LB-100 may be a true game changer in cancer treatment.

Protein phosphatases are ubiquitous enzymes that regulate many signaling networks important to cell growth, division and death. These enzymes, especially protein phosphatase 2A (PP2A), have long been appreciated as potentially important targets for anti-cancer drugs, but, because of their multi-functionality, it has been believed that their inhibition would be too toxic for clinical use. Lixte has shown that this is not the case. In a Phase 1 clinical trial, LB-100, an inhibitor of PP2A, was well tolerated at doses associated with significant tumor shrinkage (objective regression) and/or the arrest of tumor progression in patients with advancing cancers.

LB-100 has been shown to inhibit a spectrum of human cancers that have abnormalities in PP2A function.

In extensive preclinical studies, LB-100, when coupled with standard cytotoxic drug and/or radiation therapies, potentiates the effectiveness of such regimens against hematologic cancers and solid tumors without enhancing toxicity. In addition, as recently shown, low doses of LB-100 significantly increase the effectiveness of PD-1 immune-checkpoint blockade by activating cytotoxic T cells and CAR-T cells. The addition of LB-100 may be a breakthrough way to enhance the effectiveness of anticancer immunotherapy with both anti-PD-1 and anti-CTLA-4 drugs.

Our immediate goal is to demonstrate the clinical therapeutic benefit of LB-100 in major cancers that affect the largest populations.

Our focus is on cancers that are vulnerable to the inhibition of PP2A as an effective treatment based on molecular and in vivo animal data. Our pipeline includes a Phase 1b/2 trial of LB-100 in myelodysplastic syndrome in progress at the Moffitt Cancer Center in Tampa, FL (NCT03886662) and a Phase 1b/randomized 2 trial of doxorubicin with and without LB-100 in first line advanced soft tissue sarcoma in Spain led by the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas, or GEIS). In addition, the National Cancer Institute has initiated a clinical pharmacologic study (NCT03027388) of LB-100 to determine the extent of penetration of recurrent brain tumors. If positive, several centers wish to evaluate LB-100 plus standard therapy in patients with glioblastoma multiforme, the most common and deadly of adult brain cancers. Recently, we announced an agreement on a Phase 1b clinical trial with City of Hope, a world-renowned independent cancer research and treatment center. The trial (NCT04560972) will assess the combination of LB-100 with a standard regimen for untreated, extensive stage-disease small cell lung cancer (ED-SCLC).

LIXTE Biotechnology Holdings, Inc. (Nasdaq: LIXT) is a clinical-stage pharmaceutical company dedicated to discovering drugs for more effective treatments for many forms of cancer and other serious common diseases. A major driver of cancer is defects in the switches that turn the biochemical pathways in cells on and off. Most cancer research over the past 30 years has focused on the “on” switches because the “off” switches, especially the master “off” switch protein phosphatase (PP2A), were believed to cause intolerable toxicity in patients. LIXTE has achieved a breakthrough with its novel, first-in-class lead compound, PP2A inhibitor LB-100, by demonstrating that it is readily tolerated in cancer patients at doses associated with anti-cancer activity. This innovative approach encourages cancer cells, weakened by chemo or other cancer therapies, to continue to replicate, leading to the more efficient death and elimination of those cells from the body. LIXTE has partnered with top medical institutions and leading academic research centers to advance the clinical development of its compounds. The LB-100 compound, of which there are no competitors known to LIXTE currently in the clinic, is currently being tested in three active clinical studies with others in planning.