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Lixte Biotechnology Holdings, Inc. (Lixte)

a publicly traded (OTCQB: LIXT) drug discovery company, Lixte has a developed unique, proprietary, first in-class protein phosphatase inhibitor, LB-100, that has potential widespread use as an anti-cancer agent alone and in combination with standard anti-cancer cytotoxic drugs, radiation, and immune checkpoint blockers. The 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, the lead clinical compound, LB-100, an inhibitor of protein phosphatase PP2A, was well tolerated at doses associated with objective regression (significant tumor shrinkage) and/or arrest of tumor progression in patients with advancing cancers despite best available standard therapy.

The Company also developed another class of compounds that are novel histone deacetylase inhibitors (HDACi), designated by the Company as the LB-200 series of compounds. Compounds of both types also have potential use in the prevention and treatment of neurodegenerative diseases. However, the LB-100 series consists of novel structures, which have the potential to be first in their class and may be useful in the treatment of not only several types of cancer, but also vascular and metabolic diseases. The LB-200 series contains compounds which have the potential to be the most effective in its class and may be useful for the treatment of chronic hereditary diseases, such as Gaucher’s disease, in addition to cancer and neurodegenerative diseases. LB-200 has not yet advanced to the clinical stage and would require additional capital to fund further development. Accordingly, because of its focus on the clinical development of LB-100 and analogs for cancer therapy, the Company has decided not to actively pursue the pre-clinical development of its LB-200 series of compounds at this time.

Extensive preclinical studies show that LB-100 itself inhibits a spectrum of human cancers with acquired abnormalities in PP2A function. When combined with standard cytotoxic drugs and/or radiation, LB-100 potentiates their effectiveness against hematologic cancers and solid tumors without enhancing toxicity. In addition, as recently shown, at low doses, LB-100 increases the effectiveness of PD-1 immune-checkpoint blockade by activating cytotoxic T cells and CAR-T cells. The addition of LB-100 may general way to enhance the effectiveness of anticancer immunotherapy with both anti-PD-1 and anti-CTLA-4 drugs. Thus, LB-100 may be a true breakthrough in the treatment of many cancers.

Lixte’s immediate goal is to demonstrate the clinical therapeutic benefit of LB-100 in important cancers expected to be vulnerable to inhibition of PP2A based on molecular and in vivo animal data. These include a Phase 1b/2 trial of LB-100 in myelodyplastic syndrome in progress at the Moffitt Cancer Center, Tampa, FL (NCT03886662) and a Phase 1b/randomized 2 trial of doxorubicin with and without LB-100 in first line advanced sarcoma in Spain led by the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas , GEIS) to open in 2021. In addition, the National Cancer Institute has initiated a clinical pharmacologic study (NCTO30227388) to determine the extent of penetration of the LB-100 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.

There is another intriguing possible use of LB-100 at some future date to treat the symptoms of a rare genetic disorder called Angleman Syndrome (AS), affecting about 20,000 persons in the US and 500,000 worldwide. A group of neuroscientists in China and Japan recently reported that Lixte’s lead clinical compound, LB-100, improved muscle strength, movement coordination and learning in a mouse model of AS. This is a completely unanticipated finding. Whether LB-100 could possibly be of therapeutic benefit in AS is certainly unknown but Lixte has entered into an agreement with the Foundation for Angelman Syndrome Therapeutics (FAST) to collaborate in supporting preclinical studies at The University of California, Davis to verify the apparent benefit of LB-100 in a mouse model of AS. If LB-100 reduces AS signs in the rodent model, FAST and Lixte have agreed to discuss further collaboration to assess the potential benefit of LB-100 in patients with AS.

Header Background

Lixte Biotechnology Holdings, Inc. (Lixte)

a publicly traded (OTCQB: LIXT) drug discovery company, Lixte has a developed unique, proprietary, first in-class protein phosphatase inhibitor, LB-100, that has potential widespread use as an anti-cancer agent alone and in combination with standard anti-cancer cytotoxic drugs, radiation, and immune checkpoint blockers. The 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, the lead clinical compound, LB-100, an inhibitor of protein phosphatase PP2A, was well tolerated at doses associated with objective regression (significant tumor shrinkage) and/or arrest of tumor progression in patients with advancing cancers despite best available standard therapy.

The Company also developed another class of compounds that are novel histone deacetylase inhibitors (HDACi), designated by the Company as the LB-200 series of compounds. Compounds of both types also have potential use in the prevention and treatment of neurodegenerative diseases. However, the LB-100 series consists of novel structures, which have the potential to be first in their class and may be useful in the treatment of not only several types of cancer, but also vascular and metabolic diseases. The LB-200 series contains compounds which have the potential to be the most effective in its class and may be useful for the treatment of chronic hereditary diseases, such as Gaucher’s disease, in addition to cancer and neurodegenerative diseases. LB-200 has not yet advanced to the clinical stage and would require additional capital to fund further development. Accordingly, because of its focus on the clinical development of LB-100 and analogs for cancer therapy, the Company has decided not to actively pursue the pre-clinical development of its LB-200 series of compounds at this time.

Extensive preclinical studies show that LB-100 itself inhibits a spectrum of human cancers with acquired abnormalities in PP2A function. When combined with standard cytotoxic drugs and/or radiation, LB-100 potentiates their effectiveness against hematologic cancers and solid tumors without enhancing toxicity. In addition, as recently shown, at low doses, LB-100 increases the effectiveness of PD-1 immune-checkpoint blockade by activating cytotoxic T cells and CAR-T cells. The addition of LB-100 may general way to enhance the effectiveness of anticancer immunotherapy with both anti-PD-1 and anti-CTLA-4 drugs. Thus, LB-100 may be a true breakthrough in the treatment of many cancers.

Lixte’s immediate goal is to demonstrate the clinical therapeutic benefit of LB-100 in important cancers expected to be vulnerable to inhibition of PP2A based on molecular and in vivo animal data. These include a Phase 1b/2 trial of LB-100 in myelodyplastic syndrome in progress at the Moffitt Cancer Center, Tampa, FL (NCT03886662) and a Phase 1b/randomized 2 trial of doxorubicin with and without LB-100 in first line advanced sarcoma in Spain led by the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas , GEIS) to open in 2021. In addition, the National Cancer Institute has initiated a clinical pharmacologic study (NCTO30227388) to determine the extent of penetration of the LB-100 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.

There is another intriguing possible use of LB-100 at some future date to treat the symptoms of a rare genetic disorder called Angleman Syndrome (AS), affecting about 20,000 persons in the US and 500,000 worldwide. A group of neuroscientists in China and Japan recently reported that Lixte’s lead clinical compound, LB-100, improved muscle strength, movement coordination and learning in a mouse model of AS. This is a completely unanticipated finding. Whether LB-100 could possibly be of therapeutic benefit in AS is certainly unknown but Lixte has entered into an agreement with the Foundation for Angelman Syndrome Therapeutics (FAST) to collaborate in supporting preclinical studies at The University of California, Davis to verify the apparent benefit of LB-100 in a mouse model of AS. If LB-100 reduces AS signs in the rodent model, FAST and Lixte have agreed to discuss further collaboration to assess the potential benefit of LB-100 in patients with AS.

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Lixte Biotechnology Holdings, Inc. (Lixte)

a publicly traded (OTCQB: LIXT) drug discovery company, Lixte has a developed unique, proprietary, first in-class protein phosphatase inhibitor, LB-100, that has potential widespread use as an anti-cancer agent alone and in combination with standard anti-cancer cytotoxic drugs, radiation, and immune checkpoint blockers. The 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, the lead clinical compound, LB-100, an inhibitor of protein phosphatase PP2A, was well tolerated at doses associated with objective regression (significant tumor shrinkage) and/or arrest of tumor progression in patients with advancing cancers despite best available standard therapy.

The Company also developed another class of compounds that are novel histone deacetylase inhibitors (HDACi), designated by the Company as the LB-200 series of compounds. Compounds of both types also have potential use in the prevention and treatment of neurodegenerative diseases. However, the LB-100 series consists of novel structures, which have the potential to be first in their class and may be useful in the treatment of not only several types of cancer, but also vascular and metabolic diseases. The LB-200 series contains compounds which have the potential to be the most effective in its class and may be useful for the treatment of chronic hereditary diseases, such as Gaucher’s disease, in addition to cancer and neurodegenerative diseases. LB-200 has not yet advanced to the clinical stage and would require additional capital to fund further development. Accordingly, because of its focus on the clinical development of LB-100 and analogs for cancer therapy, the Company has decided not to actively pursue the pre-clinical development of its LB-200 series of compounds at this time.

Extensive preclinical studies show that LB-100 itself inhibits a spectrum of human cancers with acquired abnormalities in PP2A function. When combined with standard cytotoxic drugs and/or radiation, LB-100 potentiates their effectiveness against hematologic cancers and solid tumors without enhancing toxicity. In addition, as recently shown, at low doses, LB-100 increases the effectiveness of PD-1 immune-checkpoint blockade by activating cytotoxic T cells and CAR-T cells. The addition of LB-100 may general way to enhance the effectiveness of anticancer immunotherapy with both anti-PD-1 and anti-CTLA-4 drugs. Thus, LB-100 may be a true breakthrough in the treatment of many cancers.

Lixte’s immediate goal is to demonstrate the clinical therapeutic benefit of LB-100 in important cancers expected to be vulnerable to inhibition of PP2A based on molecular and in vivo animal data. These include a Phase 1b/2 trial of LB-100 in myelodyplastic syndrome in progress at the Moffitt Cancer Center, Tampa, FL (NCT03886662) and a Phase 1b/randomized 2 trial of doxorubicin with and without LB-100 in first line advanced sarcoma in Spain led by the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas , GEIS) to open in 2021. In addition, the National Cancer Institute has initiated a clinical pharmacologic study (NCTO30227388) to determine the extent of penetration of the LB-100 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.

There is another intriguing possible use of LB-100 at some future date to treat the symptoms of a rare genetic disorder called Angleman Syndrome (AS), affecting about 20,000 persons in the US and 500,000 worldwide. A group of neuroscientists in China and Japan recently reported that Lixte’s lead clinical compound, LB-100, improved muscle strength, movement coordination and learning in a mouse model of AS. This is a completely unanticipated finding. Whether LB-100 could possibly be of therapeutic benefit in AS is certainly unknown but Lixte has entered into an agreement with the Foundation for Angelman Syndrome Therapeutics (FAST) to collaborate in supporting preclinical studies at The University of California, Davis to verify the apparent benefit of LB-100 in a mouse model of AS. If LB-100 reduces AS signs in the rodent model, FAST and Lixte have agreed to discuss further collaboration to assess the potential benefit of LB-100 in patients with AS.