BEVERLY, MA – March 8, 2017 (GLOBE NEWSWIRE) Cellceutix Corporation, (OTCQB: CTIX) (“the Company”), a clinical stage biopharmaceutical company developing innovative therapies with dermatology, oncology, anti-inflammatory, and antibiotic applications, today announced interim results in the first two cohorts of its ongoing Phase 2a clinical trial of Brilacidin for the induction of remission of mild-to-moderate Ulcerative Colitis. Patient recruitment to the third cohort (highest dose) is currently underway. Patients include those with Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS), two types of Inflammatory Bowel Disease (IBD).
The ongoing Phase 2, open-label, Proof-of-Concept (PoC) trial comprises three sequential cohorts (6 patients per cohort), with progressive dose escalation by cohort—50 mg (Cohort A), 100 mg (Cohort B), and 200 mg (Cohort C), respectively. Daily treatment with Brilacidin by enema administration is performed consecutively for 42 days.
All twelve (12) patients across the first two cohorts have completed their full dosing schedules in the study. Comparison to baseline after six weeks of treatment showed:
- All 12 patients experienced a beneficial response, as measured by the Modified Mayo Disease Activity Index (MMDAI).
- At Day 42, the Primary Efficacy Endpoint of Clinical Remission (accounting for Stool Frequency, Rectal Bleeding, and Endoscopy sub-scores) was met in half (n=6) of all patients (3 of 6 in Cohort A; 3 of 6 in Cohort B).
- Among the remaining patients (n=6) in Cohorts A and B not meeting all three criteria for Clinical Remission, two of the three criteria were achieved by all of these patients (defined as a Partial Response).
- Patient Quality-of-Life, as assessed by the Short Inflammatory Bowel Disease Questionnaire (SIBDQ), was improved in all 12 patients after six weeks of daily Brilacidin treatment. Specifically, over 40 percent of patients reported significant improvements, ranging approximately from 20 points to more than 50 points higher on the 70-point SIBDQ scale.
- Brilacidin was generally well-tolerated and patients maintained stable normal vital signs during treatment.
Measurements of drug concentrations in plasma continued to show limited systemic absorption of Brilacidin, with all values registering less than 100 ng/mL for all six patients in Cohort A and averaging approximately 200 ng/mL maximum concentrations across the six patients in Cohort B.
Patients for Cohort C (200 mg, the highest and final dosing group) are now being enrolled. Further detailed analyses, across all three cohorts once the trial is completed, will establish which dosing level provides the most favorable overall outcomes.
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Brilacidin is Cellceutix’s lead drug candidate in its defensin mimetic franchise. Modeled after Host Defense Proteins (HDPs), the “front-line” of defense in the immune system, it is a small, non-peptidic, synthetic molecule that kills pathogens swiftly and thoroughly. Just as importantly, Brilacidin also functions in a robust immunomodulatory capacity, lessening inflammation and promoting healing. Due to its unique properties, Cellceutix is studying Brilacidin’s effect on oral mucositis (under Fast Track designation) and on ulcerative proctitis/proctosigmoiditis (UP/UPS) in Phase 2 trials. Additional trials of Brilacidin are planned in other conditions, including: hidradenitis suppurativa and acne. Brilacidin is also being developed under FDA’s Qualified Infectious Disease Product (QIDP) designation as an antibacterial product for Acute Bacterial Skin and Skin Structure Infections (ABSSSI)—qualifying it for Fast Track and possible Priority FDA Review and an extra 5 years of United States market exclusivity upon drug approval.
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Ulcerative proctitis (UP), a limited type of ulcerative colitis (UC), is a mucosal inflammatory disease of unknown cause involving only the rectum. When it involves both the rectum and the distal colon, it is called Ulcerative Proctosigmoiditis (UPS). It is characterized by inflammation, redness, and ulcerations of the mucosa. The course of the disease is variable and ranges from complete resolution to easily maintained remission to chronic relapses or refractory disease. Diagnosis can occur at any point in life, with approximately 30-50 percent of patients developing more extensive UC. There is currently no cure. According to estimates provided by GlobalData, the worldwide UC market, which includes products for UP/UPS, is expected to increase at a compound annual growth rate of 4.7 percent, from $4.2 billion in 2012 to approximately $6.6 billion by 2022.
About Cellceutix’s Proof-of-Concept (PoC) UP/UPS Trial Design
This trial is being conducted in an overseas hospital/clinic setting with Brilacidin being administered with water in an enema. A foam formulation of Brilacidin for use in future studies is planned and would be expected to improve patient convenience and study results. The primary objective of Cellceutix’s Proof-of-Concept (PoC) trial is to assess the frequency of clinical remission (defined using Modified Mayo Disease Activity Index [MMDAI] scoring) with Brilacidin administered per rectum by enema in patients with active UP or UPS after 6 weeks of treatment. Secondary objectives include: evaluation of safety and tolerability of Brilacidin when administered per rectum; assessment of systemic exposure and/or pharmacokinetics of Brilacidin when administered per rectum; assessment of the efficacy of Brilacidin by change in MMDAI at Day 42/Week 6 and Partial MMDAI during treatment and by biomarker evaluation (from serum, feces, and rectum/sigmoid biopsy samples); evaluation of change in patient-reported quality of life (by the Short Inflammatory Bowel Disease Questionnaire); and estimation of statistical power for subsequent trial(s) in UP and UPS. The PoC trial includes 18 patients divided evenly into three cohorts. Cohort A receives 50 milligrams (mg) of Brilacidin once daily administered per rectum as a retention enema for 42 days. Dosing is increased to 100 mg and 200 mg once daily for 42 days for Cohort B and Cohort C, respectively. Endoscopic evaluation of the rectum and mucosa up to 40 cm from the anal verge is performed at screening and at the end of treatment/Day 42 (± 3 days). Per protocol, a Safety Committee reviews safety and retention data (clinical laboratory findings, vital signs, adverse events, retention times) after 21 days of therapy for all six patients in each cohort before proceeding with initiating enrollment into the subsequent cohort.