BEVERLY, MA – January 23, 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, is pleased to update shareholders following its recent monthly conference call with the three clinical sites conducting Brilacidin’s Phase 2 Proof-of-Concept (PoC) study as a new treatment for ulcerative proctitis/ulcerative proctosigmoiditis (UP/UPS), limited forms of ulcerative colitis (UC).
Site investigators report enthusiastic feedback from patients currently receiving treatment in the second cohort with Brilacidin at 100 mg daily (by retention enema) for six weeks —comments included how the treatment already has greatly improved or eliminated their symptomatic complaints, daily functioning and overall quality of life. There is significant interest by new patients wishing to participate in the third (final) cohort once enrollment opens.
All six patients in the completed first cohort were treated with 50 mg of Brilacidin, once daily for six consecutive weeks (42 days), as a retention enema. The initial data showed Brilacidin to be well-tolerated with no severe adverse events reported and no detection of measurable systemic absorption. Preliminary review of endoscopic images and video from Screening and Day 42 in patients in the first cohort showed meaningful improvements for 5 of the 6 patients, including noticeable reductions in ulceration and bleeding. Such endoscopic data reflect the ability of Brilacidin to help clear Gastrointestinal (GI) tract mucosa in patients suffering from UC. The Food and Drug Administration (FDA), in 2016 draft guidance for UC, has conveyed the “ideal primary efficacy assessment tool” would include an “endoscopic and histological assessment scale,” in addition to a “patient-reported outcome instrument.” This guidance reinforces the importance of endoscopic remission as a critical efficacy measure toward gaining drug approval.
Endoscopic assessments, pharmacokinetic profiles, as well as patient/physician-reported outcomes, collectively, suggest that, even at the lowest dosing level (50 mg), Brilacidin offers potential to deliver a clinically meaningful, safe, and expedient therapeutic response among trial participants. These are the positive signals one looks for in a Proof-of-Concept study, to attract partnering companies. Registration trials for filing a New Drug Application (NDA) will require a final formulation and statistically powered studies with a control arm, which are not part of this PoC trial. The Company believes that while additional clinical data are needed to further corroborate findings, should future data remain consistent across the entire trial, Brilacidin could potentially emerge as a treatment for a wide range of Inflammatory Bowel Disease (IBD) indications. Moreover, as costly biologic therapies account for approximately 10 percent of overall pharmacy spending in the U.S., the need for less expensive treatments—an attribute Brilacidin can potentially offer the marketplace due to its lower cost of production—is becoming a real economic imperative.
Leo Ehrlich, Chief Executive Officer at Cellceutix, commented, “We unequivocally believe that Brilacidin can emerge as a novel drug in treating Ulcerative Colitis and other debilitating IBD conditions, which affect millions of people worldwide. Endoscopic assessments from the first cohort are particularly telling. We excitedly anticipate finishing the next two cohorts and sharing complete trial findings in the first half of 2017. Simply put, I believe that Brilacidin shows the potential to become one of the most sought after IBD drugs.”
Cellceutix looks forward to continuing to share data from the ongoing Proof-of-Concept trial and plans to present complete findings at a future Scientific Conference.
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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 atopic dermatitis. 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 Infection (ABSSSI)—qualifying it for Fast Track and possible Priority FDA Review and a potential 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. Additional 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 will include 18 patients divided evenly into three cohorts. Cohort A has received 50 milligrams (mg) of Brilacidin once daily administered per rectum as a retention enema for 42 days. Dosing increases 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 will be performed at screening and at the end of treatment/Day 42 (± 3 days). Per protocol, a safety committee will review safety and retention data (clinical laboratory findings, physical examination findings, vital signs, adverse events, use of concomitant medications, retention times) after 21 days of therapy for all six patients in each cohort before proceeding with initiating enrollment (dosing) into the subsequent cohort.