Cellceutix Phase 2 Trial Initial Data Shows Potential of Brilacidin as a Novel Anti-Inflammatory Drug Candidate for the Induction of Remission of Mild-to-Moderate Ulcerative Colitis
BEVERLY, Mass., Oct. 10, 2016 (GLOBE NEWSWIRE) -- Cellceutix Corporation, (CTIX) (“the Company”), a clinical stage biopharmaceutical company developing innovative therapies with dermatology, oncology, anti-inflammatory, and antibiotic applications, is pleased to announce today interim results observed for the first four patients treated with Brilacidin for Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS), a type of Inflammatory Bowel Disease (IBD). Clinically meaningful improvements were demonstrated, as measured by physician assessments, patient reported outcomes and endoscopic evaluation of disease activity.
The ongoing Phase 2, open-label, proof-of-concept trial comprises three sequential cohorts (6 patients per cohort), with progressive dose escalation by cohort—50 mg, 100 mg, and 200 mg, respectively. Treatment with Brilacidin by daily enema administration is performed for 42 days.
Four (4) of 6 patients to date in the lowest dosing cohort (50 mg) have completed the study. Comparison to baseline after 6 weeks of treatment showed:
All 4 patients experienced a clinically meaningful response, as measured by the Modified Mayo Disease Activity Index (MMDAI).
At Day 42, on the Partial MMDAI (accounting for Stool Frequency, Rectal Bleeding, and Physician’s Global Assessment scores), 2 of 4 patients achieved full response (100% reduction) and the other 2 patients had notable improvement (50% reduction).
At Day 42, on the MMDAI (equivalent to Partial MMDAI + Endoscopy score; completed by 3 of 4 patients), 1 of 3 patients achieved full response (100% reduction) and 2 of 3 patients had notable improvement (50% reduction). One patient who had demonstrated a full response on the Partial MMDAI (as defined in first bullet point) did not consent to the final endoscopy, so their data cannot be included in these initial results.
At Day 42, on the Partial MMDAI (accounting for Stool Frequency, Rectal Bleeding, and Physician’s Global Assessment scores), 2 of 4 patients achieved full response (100% reduction) and the other 2 patients had notable improvement (50% reduction).
At Day 42, on the MMDAI (equivalent to Partial MMDAI + Endoscopy score; completed by 3 of 4 patients), 1 of 3 patients achieved full response (100% reduction) and 2 of 3 patients had notable improvement (50% reduction). One patient who had demonstrated a full response on the Partial MMDAI (as defined in first bullet point) did not consent to the final endoscopy, so their data cannot be included in these initial results.
Patient quality of life, as assessed by the Short Inflammatory Bowel Disease Questionnaire (SIBDQ), was improved after 6-weeks of treatment with Brilacidin.
Brilacidin was generally well-tolerated and patients maintained stable normal vital signs during treatment.
For all 4 patients, measurements of drug concentrations in plasma showed all levels, across all time points, to be below the lower limit of quantification (i.e., <100 ng/mL), which is consistent with very limited systemic exposure from administration per rectum by enema. In comparison, based on a previously completed and successful Phase 2b clinical trial of Brilacidin in the treatment of serious skin infections, maximum concentrations of the drug in plasma administered by intravenous (iv) dosing, at 0.6 mg/kg and 0.8 mg/kg, were approximately 9,000 ng/mL and 12,000 ng/mL, respectively.
“These results are extremely encouraging. They support early clinical validity of the unique immunomodulatory properties of Brilacidin. We anticipate the anti-inflammatory properties of Brilacidin alone will lead to multiple beneficial applications of the drug candidate beyond its already demonstrated effect in antibacterial indications,” said Arthur P. Bertolino, MD, PhD, MBA, Cellceutix President and Chief Medical Officer.
Leo Ehrlich, Chief Executive Officer of Cellceutix, commented: “We are delighted to see such promising responses to treatment in the first group of patients given the lowest dose of Brilacidin. The open label design of this trial allowed us to take an early look. Inflammatory Bowel Disease, which affects millions worldwide, poses a real medical need for patients who we hope may eventually benefit from our lead defensin mimetic drug candidate. The immunomodulatory and anti-inflammatory properties are at the core of effectively treating many diseases and conditions, such as IBDs and oral mucositis. As we are initiating 2-3 more sites to our ongoing oral mucositis (OM) trial this month and continue to report results from this UP trial, we expect to continue to grow many horizontal and vertical pipeline opportunities with the Brilacidin franchise.”
Continued reporting of results from this ongoing Phase 2 proof-of-concept clinical trial is anticipated over the coming months.
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About Brilacidin
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.
About UP/UPS
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 will include 18 patients divided evenly into three cohorts. Cohort A is receiving 50 milligrams (mg) of Brilacidin once daily administered per rectum as a retention enema for 42 days. Dosing will be 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 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, 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.
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 first-in-class lead drug candidates and is now advancing them toward market approval, while actively seeking strategic partnerships. Cellceutix’s anti-psoriasis drug Prurisol completed a Phase 2 trial and Cellceutix is now preparing to launch a Phase 2b study. Prurisol is a small molecule that acts through immune modulation and PRINS reduction. Cellceutix’s anti-cancer drug Kevetrin concluded a Phase 1 clinical trial at Harvard Cancer Centers’ Dana Farber Cancer Institute and Beth Israel Deaconess Medical Center, and Cellceutix is now preparing its plans for a Phase 2 study. 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 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 atwww.cellceutix.com.
Forward-Looking Statements:
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 including statements concerning projected timelines for the initiation and completion of clinical trials, our future drug development plans, other statements regarding future product developments, including with respect to specific indications, and any other statements which are other than statements of historical fact. These statements involve risks, uncertainties and assumptions that could cause Cellceutix’s actual results and experience to differ materially from anticipated results and expectations expressed in these forward looking statements. Cellceutix has in some cases identified forward-looking statements by using words such as “anticipates,” “believes,” “hopes,” “estimates,” “looks,” “expects,” “plans,” “intends,” “goal,” “potential,” “may,” “suggest,” and similar expressions. Among other factors that could cause actual results to differ materially from those expressed in forward-looking statements are Cellceutix’s need for, and the availability of, substantial capital in the future to fund its operations and research and development; including the amount and timing of the sale of shares of common stock to Aspire Capital; the fact that Cellceutix’s compounds may not successfully complete pre-clinical or clinical testing, or be granted regulatory approval to be sold and marketed in the United States or elsewhere. A more complete description of these risk factors is included in Cellceutix’s filings with the Securities and Exchange Commission. You should not place undue reliance on any forward-looking statements. Cellceutix undertakes no obligation to release publicly the results of any revisions to any such forward-looking statements that may be made to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events, except as required by applicable law or regulation.
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