Clinical Trials
In April 2015, safety and efficacy results from a 215-patient Phase 2b clinical trial for ABSSSI (defined below) of our lead antibiotic compound, Brilacidin, were presented at the 25th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID). In July 2015, at an End-of-Phase 2 Meeting, Cellceutix and FDA discussed data supporting advancement into Phase 3, as well as the basic elements of a brilacidin Phase 3 program in ABSSSI. The Phase 3 program would include two Phase 3 ABSSSI studies, as required by FDA Guidance (October 2013). In addition, the first study would include an interim analysis after a portion of the patients has been enrolled. This would provide an early assessment of both safety and efficacy. As part of the agreement, the Company would submit a Pediatric Study Plan (PSP) within 60 days of the End-of-Phase 2 Meeting. The Company is currently evaluating potential Contract Research Organizations (CROs) and clinical sites for the global Phase 3 program.
In June 2015, Cellceutix’s presented an overview of the Company’s ongoing Phase 1 clinical trial to evaluate the safety and preliminary efficacy of Kevetrin for patients with advanced solid tumors at the annual American Society of Clinical Oncology (ASCO) meeting in Chicago. The study has demonstrated the safety of Kevetrin administration over repeated cycles. In addition, increases in p21 expression in peripheral blood lymphocytes were shown in a number of the patients in multiple cancers and p21 activity appears to be dose related. The study is near completion at the Dana-Farber Cancer Institute and Beth Israel Deaconess Medical Center, as we are presently enrolling the final cohort of the Phase 1 study. We believe we have reached the maximum tolerated dose (MTD), but with no dose limiting toxicities in this cohort. Exposure to Kevetrin as measured by plasma concentrations have been achieved which are greater than concentrations shown to induce apoptosis in non-clinical studies. The final cohort evaluates at least 6 patients, as is typical for a Phase 1 study of this type.
In July 2015, the U.S. Food and Drug Administration (FDA), granted Orphan Drug Designation to Kevetrin for the treatment of ovarian cancer.
In August 2015, Cellceutix announced the commencement of the Company’s Phase 2 trial of Prurisol for the treatment of plaque psoriasis, and patient recruitment is underway. Cellceutix is developing Prurisol under guidance from the U.S. Food and Drug Administration that a 505(b)(2) designation is an acceptable pathway to expedite development of the compound.
In August 2015, Cellceutix announced the addition of two clinical sites for the Company’s ongoing Phase 2 clinical trial of Brilacidin-OM for the treatment and prevention of oral mucositis in patients with head and neck cancer, bringing the total number of sites in the study to date to five. Cellceutix is evaluating additional sites for inclusion in the study. Increasing patient access to the trial is important in the Company’s efforts to discover an effective, safe therapy for prevention of oral mucositis, and to prove the anti-inflammatory properties of Brilacidin in a clinical setting. Laboratory studies have shown Brilacidin to have both antibacterial and anti-inflammatory properties.
Research Collaborations
1. HDP Mimics.
Advancing the Platform and Developing Compounds with Activity Against Fungi
We have entered into a research collaboration with Fox Chase Chemical Diversity Center (FCCDC) which has led to an award in June 2014 of a Phase 2b Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Disease (NIAID). This $1.5 million dollar grant (over 2 years) will be directed into developing this technology platform on host defense protein (HDP) mimics (defensin-mimetics) and for treatment of disseminated fungal infections, particularly those caused by Candida species.
Developing Compounds with Activity Against Gram-Negative Bacteria
We are evaluating several host defense protein (HDP) mimics as novel antibiotic compounds, with activity against specific strains of Enterobacteriaceae, including multi-drug resistant Klebsiella pneumonia, and other Gram-negative pathogens such as Acinetobacter baumannii and Pseudomonas aeruginosa. In December 2014, Fox Chase Chemical Diversity Center received a 1-year extension on a subcontract from the University of Massachusetts (Amherst) under a UO1 National Institute of Health grant. The subcontract of $565,440 will be used to continue research on the development of our platform technology of host defense protein mimics (HDP mimics or defensin-mimetics) to combat serious and life-threatening infections caused by multi-drug resistant Gram-negative bacteria.
2. Kevetrin. There is a potential for use of Kevetrin in multiple tumor types, and in combination with other chemotherapeutic agents. In 2012, we entered into an agreement with Beth Israel Deaconess Medical Center (BIDMC), a teaching hospital of Harvard Medical School, on an innovative research project with Kevetrin. The Medical Center wishes to exploit the nuclear and/or mitochondrial pro-apoptotic function of p53 in melanoma and renal cell carcinoma, two types of cancer that are particularly resistant to therapy. At the conclusion of the Phase 1 study we will engage in discussions with the hospital as to the logistics and costs, net of grants, to move this project forward into Phase 2 studies of renal cell carcinomas.
The University of Bologna in Italy (the “University”) and The Italian Cooperative Study Group on Chronic Myeloid Leukemia (ICSG on CML) and Acute Leukemia (GIMEMA Group) plan on evaluating Kevetrin in patients with Acute Myelogenous Leukemia (AML). The study proposed is a Phase 2 trial evaluating Kevetrin as a single agent or in combination with cytarabine in patients with AML. Over 100 patients are expected to be enrolled in the trial. The protocol was submitted in May 2015 by the principal investigator to the institutional committee. There have been delays in communication due to the summer schedules in Italy. We are awaiting comments on the protocol and only then can we have a clearer picture of the timing of the study. This is an important trial for Kevetrin as these AML patients will be receiving Kevetrin on multiple consecutive days, which we believe will increase p53 activity, hence its anti-tumor activity. The primary objective of this trial is to evaluate the rate of complete remission of AML in patients receiving Kevetrin alone or in combination with cytarabine. We believe that if the trial shows clinical activity of Kevetrin or Kevetrin plus cytarabine in the treatment of AML, a disease that the American Cancer Society estimates accounts for 20,830 new cases and 10,460 deaths annually in the United States, we will see a substantial rise in interest in Kevetrin for potential use in leukemias; however, there can be no guarantee that the trials will proceed on time or will yield positive results. In June 2013, we signed a Material Transfer Agreement with the University of Texas, MD Anderson Cancer Center. MD Anderson intends to utilize in vivo and in vitro methods to research specific pathways, gene expression, mechanism of action and apoptotic activity of Kevetrin in a range of concentrations and time points in both mutant and wild-type p53 Myeloma and Lymphoma cell lines. The National Cancer Institute estimates that more than 24,000 individuals will be diagnosed with myeloma in the United States in 2014, and more than 11,000 will die from this disease. They also wish to study our other cancer compounds against a broad array of Multiple Myeloma cell lines that are resistant to today’s FDA-approved chemotherapies. MD Anderson is covering the expenses of the research, with Cellceutix only supplying the drugs.
In April 2015, safety and efficacy results from a 215-patient Phase 2b clinical trial for ABSSSI (defined below) of our lead antibiotic compound, Brilacidin, were presented at the 25th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID). In July 2015, at an End-of-Phase 2 Meeting, Cellceutix and FDA discussed data supporting advancement into Phase 3, as well as the basic elements of a brilacidin Phase 3 program in ABSSSI. The Phase 3 program would include two Phase 3 ABSSSI studies, as required by FDA Guidance (October 2013). In addition, the first study would include an interim analysis after a portion of the patients has been enrolled. This would provide an early assessment of both safety and efficacy. As part of the agreement, the Company would submit a Pediatric Study Plan (PSP) within 60 days of the End-of-Phase 2 Meeting. The Company is currently evaluating potential Contract Research Organizations (CROs) and clinical sites for the global Phase 3 program.
In June 2015, Cellceutix’s presented an overview of the Company’s ongoing Phase 1 clinical trial to evaluate the safety and preliminary efficacy of Kevetrin for patients with advanced solid tumors at the annual American Society of Clinical Oncology (ASCO) meeting in Chicago. The study has demonstrated the safety of Kevetrin administration over repeated cycles. In addition, increases in p21 expression in peripheral blood lymphocytes were shown in a number of the patients in multiple cancers and p21 activity appears to be dose related. The study is near completion at the Dana-Farber Cancer Institute and Beth Israel Deaconess Medical Center, as we are presently enrolling the final cohort of the Phase 1 study. We believe we have reached the maximum tolerated dose (MTD), but with no dose limiting toxicities in this cohort. Exposure to Kevetrin as measured by plasma concentrations have been achieved which are greater than concentrations shown to induce apoptosis in non-clinical studies. The final cohort evaluates at least 6 patients, as is typical for a Phase 1 study of this type.
In July 2015, the U.S. Food and Drug Administration (FDA), granted Orphan Drug Designation to Kevetrin for the treatment of ovarian cancer.
In August 2015, Cellceutix announced the commencement of the Company’s Phase 2 trial of Prurisol for the treatment of plaque psoriasis, and patient recruitment is underway. Cellceutix is developing Prurisol under guidance from the U.S. Food and Drug Administration that a 505(b)(2) designation is an acceptable pathway to expedite development of the compound.
In August 2015, Cellceutix announced the addition of two clinical sites for the Company’s ongoing Phase 2 clinical trial of Brilacidin-OM for the treatment and prevention of oral mucositis in patients with head and neck cancer, bringing the total number of sites in the study to date to five. Cellceutix is evaluating additional sites for inclusion in the study. Increasing patient access to the trial is important in the Company’s efforts to discover an effective, safe therapy for prevention of oral mucositis, and to prove the anti-inflammatory properties of Brilacidin in a clinical setting. Laboratory studies have shown Brilacidin to have both antibacterial and anti-inflammatory properties.
Research Collaborations
1. HDP Mimics.
Advancing the Platform and Developing Compounds with Activity Against Fungi
We have entered into a research collaboration with Fox Chase Chemical Diversity Center (FCCDC) which has led to an award in June 2014 of a Phase 2b Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Disease (NIAID). This $1.5 million dollar grant (over 2 years) will be directed into developing this technology platform on host defense protein (HDP) mimics (defensin-mimetics) and for treatment of disseminated fungal infections, particularly those caused by Candida species.
Developing Compounds with Activity Against Gram-Negative Bacteria
We are evaluating several host defense protein (HDP) mimics as novel antibiotic compounds, with activity against specific strains of Enterobacteriaceae, including multi-drug resistant Klebsiella pneumonia, and other Gram-negative pathogens such as Acinetobacter baumannii and Pseudomonas aeruginosa. In December 2014, Fox Chase Chemical Diversity Center received a 1-year extension on a subcontract from the University of Massachusetts (Amherst) under a UO1 National Institute of Health grant. The subcontract of $565,440 will be used to continue research on the development of our platform technology of host defense protein mimics (HDP mimics or defensin-mimetics) to combat serious and life-threatening infections caused by multi-drug resistant Gram-negative bacteria.
2. Kevetrin. There is a potential for use of Kevetrin in multiple tumor types, and in combination with other chemotherapeutic agents. In 2012, we entered into an agreement with Beth Israel Deaconess Medical Center (BIDMC), a teaching hospital of Harvard Medical School, on an innovative research project with Kevetrin. The Medical Center wishes to exploit the nuclear and/or mitochondrial pro-apoptotic function of p53 in melanoma and renal cell carcinoma, two types of cancer that are particularly resistant to therapy. At the conclusion of the Phase 1 study we will engage in discussions with the hospital as to the logistics and costs, net of grants, to move this project forward into Phase 2 studies of renal cell carcinomas.
The University of Bologna in Italy (the “University”) and The Italian Cooperative Study Group on Chronic Myeloid Leukemia (ICSG on CML) and Acute Leukemia (GIMEMA Group) plan on evaluating Kevetrin in patients with Acute Myelogenous Leukemia (AML). The study proposed is a Phase 2 trial evaluating Kevetrin as a single agent or in combination with cytarabine in patients with AML. Over 100 patients are expected to be enrolled in the trial. The protocol was submitted in May 2015 by the principal investigator to the institutional committee. There have been delays in communication due to the summer schedules in Italy. We are awaiting comments on the protocol and only then can we have a clearer picture of the timing of the study. This is an important trial for Kevetrin as these AML patients will be receiving Kevetrin on multiple consecutive days, which we believe will increase p53 activity, hence its anti-tumor activity. The primary objective of this trial is to evaluate the rate of complete remission of AML in patients receiving Kevetrin alone or in combination with cytarabine. We believe that if the trial shows clinical activity of Kevetrin or Kevetrin plus cytarabine in the treatment of AML, a disease that the American Cancer Society estimates accounts for 20,830 new cases and 10,460 deaths annually in the United States, we will see a substantial rise in interest in Kevetrin for potential use in leukemias; however, there can be no guarantee that the trials will proceed on time or will yield positive results. In June 2013, we signed a Material Transfer Agreement with the University of Texas, MD Anderson Cancer Center. MD Anderson intends to utilize in vivo and in vitro methods to research specific pathways, gene expression, mechanism of action and apoptotic activity of Kevetrin in a range of concentrations and time points in both mutant and wild-type p53 Myeloma and Lymphoma cell lines. The National Cancer Institute estimates that more than 24,000 individuals will be diagnosed with myeloma in the United States in 2014, and more than 11,000 will die from this disease. They also wish to study our other cancer compounds against a broad array of Multiple Myeloma cell lines that are resistant to today’s FDA-approved chemotherapies. MD Anderson is covering the expenses of the research, with Cellceutix only supplying the drugs.
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