Cellceutix Releases Data Used in Receiving FDA Orphan Drug Designation of Kevetrin for Pancreatic Cancer

BEVERLY, MA--(Marketwired - January 22, 2016) - Cellceutix Corporation (OTC: CTIX) (the "Company"), a clinical stage biopharmaceutical company developing innovative therapies with oncology, dermatology, anti-inflammatory and antibiotic applications, today released information collected from research of Kevetrin for pancreatic cancer. The data was submitted to the U.S. Food and Drug Administration (FDA) as part of an application for an Orphan Drug designation for the indication, which was approved and disclosed yesterday, January 21, 2016. 

The laboratory work was a collaborative effort between Cellceutix and pancreatic cancer specialists at a world renowned clinic headquartered in Rochester, Minnesota (the "Clinic"). Cellceutix and the Clinic also collaboratively filed an application for a grant for a clinical trial of Kevetrin for pancreatic cancer.

Preclinical data

In vitro studies with the human pancreatic carcinoma cell line (MIA PaCa-2), which carries the R248W p53 mutation, showed that Kevetrin induced PARP cleavage, down regulated expression of HDAC2, HDAC6, c-Myc and showed synergistic induction of apoptosis in combination with doxorubicin.

In vitro Western blot studies showed that Kevetrin induced p21, the p53 target gene, in MIA PaCa-2 cells.

Poly ADP ribose polymerase (PARP) is involved in DNA repair and helps cells to maintain their viability. Cleavage of PARP serves as a marker of cells undergoing apoptosis. Kevetrin strongly induced PARP cleavage in MIA PaCa-2 cells within 24 hours. When doxorubicin was incubated in combination with Kevetrin, there was a synergistic increase in apoptosis. This suggests that proficient efficacy with lower toxicity may be achieved.

Histone deacetylase (HDAC) has been found to be dysregulated in many cancers. Upregulation of HDAC2 and HDAC6 is associated with advanced stage and poor prognosis. In the research, Kevetrin downregulated expression of both HDAC2 and HDAC6 in MIA PaCa-2 cells.

In vivo efficacy of Kevetrin in the pancreatic MIA PaCa-2 xenograft model showed that tumor volumes were significantly reduced by 82% during 3 weeks of dosing at 200mg/kg. In a separate experiment when Kevetrin alone was dosed at 150mg/kg the tumor volume was reduced by 54%. The combination of Kevetrin and irinotecan further reduced tumor volumes by 88% showing synergistic activity.

Weight changes observed during the daily dosing regimen suggest the combination of Kevetrin and Irinotecan is well-tolerated while being very efficacious in the animal model.

Clinical data

A Phase 1 clinical study was conducted with Kevetrin administered once weekly for 3 weeks with an off-week between cycles. Only one patient with pancreatic cancer was enrolled in the trial. The patient, who was diagnosed with pancreatic cancer received four full cycles of Kevetrin. Tumor measurements showed stable disease for more than three months.

Pancreatic Cancer

Last year, an estimated 48,960 adults (24,840 men and 24,120 women) in the United States were diagnosed with pancreatic cancer. It is estimated that 40,560 deaths (20,710 men and 19,850 women) from this disease occurred. Pancreatic cancer is the eighth most common cancer in women and the fourth leading cause of cancer death in men and women. A notoriously difficult disease to treat, pancreatic cancer has a five-year survival rate of only 7.2 percent.

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About Cellceutix:
Headquartered in Beverly, Massachusetts, Cellceutix is a publicly traded company under the symbol "CTIX". Cellceutix is a clinical stage biopharmaceutical company developing innovative therapies in multiple diseases. Cellceutix believes it has a world-class portfolio of compounds and is now engaged in advancing its compounds and seeking strategic partnerships. Cellceutix's anti-cancer drug Kevetrin is currently in a Phase 1 clinical trial at Harvard Cancer Centers' Dana Farber Cancer Institute and Beth Israel Deaconess Medical Center. In the laboratory Kevetrin has shown to induce activation of p53, often referred to as the "Guardian Angel Gene" due to its crucial role in controlling cell mutations. Cellceutix is in a Phase 2 clinical trial with its novel compound Brilacidin-OM for the prevention of Oral Mucositis in patients with head and neck cancer. Brilacidin-OM, a defensin mimetic compound, has shown in an animal model to reduce the occurrence of severe ulcerative oral mucositis by more than 94% compared to placebo. Cellceutix's anti-psoriasis drug Prurisol is in a Phase 2 trial. Prurisol is a small molecule that acts through immune modulation and PRINS reduction. Cellceutix's lead antibiotic, Brilacidin, has completed a Phase 2b trial for Acute Bacterial Skin and Skin Structure Infections, or ABSSSI. Top-line data have shown a single dose of Brilacidin to deliver comparable clinical outcomes to the FDA-approved seven-day dosing regimen of daptomycin. Brilacidin has the potential to be a single-dose therapy for certain multi-drug resistant bacteria (Superbugs). Cellceutix has formed research collaborations with world-renowned research institutions in the United States and Europe, including MD Anderson Cancer Center, Beth Israel Deaconess Medical Center, and the University of Bologna. More information is available on the Cellceutix web site at www.cellceutix.com.

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