$IPIX - iPharm Reports Very Encouraging Interim Analysis of Phase 2 Drug Candidate Brilacidin for Severe Oral Mucositis (OM) in Head and Neck Cancer Patients; High Potential for Preventative Treatment
Study showed a markedly reduced rate of Severe OM (WHO Grade = 3): Active Arm (Brilacidin): 2 of 9 patients (22.2 percent); Control Arm (Placebo): 7 of 10 patients (70 percent)
BEVERLY, MA – March 27, 2017 (GLOBE NEWSWIRE) iPharm fka Cellceutix Corporation, (IPIX fna OTCQB:CTIX) (“the Company”), today presented positive results from the Company's ongoing randomized, double-blind Phase 2 study of Brilacidin in the prevention and control of Oral Mucositis (OM) in patients receiving chemoradiation for treatment of Head and Neck Cancer. In a preliminary Interim Analysis, a marked reduction in incidence of Severe OM (WHO Grade = 3) was observed in patients treated with Brilacidin who received at least 55 Gy cumulative units of radiation. Study results, complementing favorable data released last week in the treatment of Ulcerative Proctitis/Proctosigmoiditis with Brilacidin, further establish Brilacidin as a novel anti-inflammatory drug candidate with potentially broad applications.
Presented below is information on Cellceutix’s clinical trial of Brilacidin for OM, including preliminary Interim Analysis of 19 patients (9 administered Brilacidin; 10 administered placebo) who have reached or passed the planned visit at the end of 5 weeks on study, and received a cumulative radiation dose of at least 55 Gy.
STUDY DESIGN
CTIX-BRI-205 is a Phase 2 randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of Brilacidin as an oral rinse in preventing and controlling OM in patients receiving chemoradiation therapy for Head and Neck Cancer. The study is anticipated to enroll approximately 60 patients in the United States, 30 each to Brilacidin treatment or to placebo (water). Brilacidin (45 mg/15 ml oral rinse—“swish and spit”) is administered 3 times daily across 7 weeks (49 days). Pharmacokinetics of Brilacidin are to be evaluated if there is measurable systemic exposure (from drug concentrations in plasma).
The Brilacidin OM trial uses a World Health Organization (WHO) OM Grading Scale, a common measurement tool in assessing the presence and severity of OM, as defined below.
WHO Scale for OM
• Grade 0 = None • Grade 1 = Erythema and Mouth Pain Soreness; no Ulceration/Pseudomembrane formation • Grade 2 = Ulceration/Pseudomembrane formation; solid diet • Grade 3 = Ulceration/Pseudomembrane formation; liquid diet • Grade 4 = Ulceration/Pseudomembrane formation; not able to tolerate a solid or liquid diet (except enough liquid for medication)
Primary Endpoints of the Brilacidin OM trial are:
• Incidence of Severe OM (WHO Grade = 3) experienced during radiation therapy (across 7 weeks) by patients with Head and Neck Cancer receiving a cumulative radiation dose of at least 55 Gy in the course of their chemoradiation treatment • Safety and Tolerability of Brilacidin
A Secondary Endpoint included in the Interim Analysis is:
• Duration of Severe OM (WHO Grade = 3)
Pharmacokinetics of Brilacidin is determined from:
• Brilacidin concentrations in plasma
INTERIM ANALYSIS
Preliminary efficacy and safety data from 19 patients who met the criteria for evaluation were reviewed. To be included, patients needed to have reached or passed the planned visit at the end of 5 weeks on study, and have received a cumulative radiation dose of at least 55 Gy. Patients receiving Brilacidin, as compared to patients on placebo, showed a markedly reduced rate of Severe OM (WHO Grade = 3). Additionally, Brilacidin was generally safe and well-tolerated.
Primary Efficacy Results Incidence of Severe OM (WHO Grade = 3)
• Active Arm (Brilacidin): 2 of 9 patients (22.2 percent) • Control Arm (Placebo): 7 of 10 patients (70 percent)
Secondary Efficacy Results Duration of Severe OM (WHO Grade = 3)
• Active Arm (Brilacidin): Mean 10.5 days (Range 3 to 18 days; 2 patients) • Control Arm (Placebo): Mean 14 days (Range 3 to 39 days; 7 patients)
Safety and Tolerability Profile
• Brilacidin administered as an oral rinse was generally well-tolerated by patients • Safety findings were typical for patients with Head and Neck Cancer being treated with chemoradiation; no treatment group differences were apparent on vital signs and clinical laboratory safety tests • Six patients (2 in Active Arm, 4 in Control Arm) experienced at least one adverse event categorized as serious. Nine Serious Adverse Events (SAEs) were reported for these 6 patients, and all were classified by the Investigator as Not Related to study drug • Incidence of Treatment-Emergent Adverse Events (TEAEs) o Majority of the TEAEs reported (Active Arm, 164 TEAEs; Control Arm, 143 TEAEs) were related to chemoradiation or the underlying study indication o No TEAEs were classified as Likely Related or Definitely Related to study treatment
Pharmacokinetics
• Plasma samples from 6 patients treated with Brilacidin were analyzed and all concentrations of Brilacidin were below the lower limit of quantification (i.e., < 10 ng/mL)
“These interim results suggest the potential for an even greater effective therapeutic response as formulation and dosing is further optimized,” commented Arthur P. Bertolino, MD, PhD, MBA, President and Chief Medical Officer at Cellceutix. "There currently is no existing preventative treatment available for OM patients in this population, with only limited therapies focusing on symptom relief. Already under Fast Track designation, should Brilacidin earn FDA approval for the treatment of Oral Mucositis, countless patients may no longer have to suffer from this horribly debilitating condition as a side effect of cancer treatment. The use of Brilacidin to prevent the onset of OM could even lead to an entirely new standard of care in this area as we strive to bring this drug to market.”
Cellceutix, over the past months, has added additional clinical sites to this study. Completion of the clinical trial is expected before year-end.
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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 a potential extra 5 years of United States market exclusivity upon drug approval.
Oral Mucositis (OM) is a frequent, painful and debilitating complication of chemoradiation commonly manifesting in the treatment of head and neck tumors. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak or eat (requiring the insertion of a feeding tube) and more susceptible to bacterial infections, with severe cases leading to hospitalization, with increased treatment costs of up to $25,000. Affecting over 500,000 people in the United States, there currently are no approved medications for the prevention of OM in this population, with only limited palliative care options available. Worldwide, the potential market for OM is expected to exceed $1 billion in the next few years.
$IPIX - Innovation Pharmaceuticals Phase 2 PoC Trial for Inflammatory Bowel Disease Achieves Induction of Remission in a Majority of Patients Treated with Brilacidin
BEVERLY, MA – July 13, 2017 (GLOBE NEWSWIRE) Innovation Pharmaceuticals, (OTCQB:IPIX) (“the Company”), a clinical stage biopharmaceutical company, today announces that a majority of patients treated with Brilacidin achieved Clinical Remission in its Phase 2, open-label, Proof-of-Concept (PoC) clinical trial evaluating Brilacidin for mild-to-moderate Ulcerative Proctitis / Ulcerative Proctosigmoiditis (UP/UPS), two types of Inflammatory Bowel Disease (IBD).
Brilacidin is being developed as a novel, non-corticosteroid, non-biologic treatment, with formulation development plans including oral tablets for the treatment of Ulcerative Colitis and Crohn’s Disease and foam and/or gel for the treatment of UP/UPS.
Results
Using Modified Mayo scoring, the study’s Primary Efficacy Endpoint of Clinical Remission at Day 42 (Week 6) was defined as: (i) an Endoscopy subscore ≤ 1; (ii) Rectal Bleeding subscore of 0; and (iii) improvement or no change from baseline in the Stool Frequency subscore.
Topline results, which include additional detailed review of patient data in Cohort A and Cohort B following interim analysis, showed favorable Clinical Remission rates that were similar across cohorts.
Rate of Clinical Remission (% of patients achieving)
· 60% Cohort A (3 of 5 patients)* · 67% Cohort B (4 of 6 patients) · 75% Cohort C (3 of 4 patients)*
(*Among the 17 patients enrolled in the trial, one patient from Cohort A and one patient from Cohort C declined endoscopy at Day 42 and thus were not included in the analysis population of those achieving Clinical Remission, i.e., the total number of evaluable patients was 15.)
The percentage of evaluable patients (n=15) meeting individual component criterion of Clinical Remission was also favorable, with rates similar across cohorts:
(i) #Endoscopy subscore ≤ 1 (% of patients achieving)
· 80% Cohort A (4 of 5 patients) · 67% Cohort B (4 of 6 patients) · 75% Cohort C (3 of 4 patients)
(#Note: Investigator assessment of rectal and sigmoid mucosa up to 40 cm from anal verge.)
(ii) Rectal Bleeding subscore of 0 (% of patients achieving)
· 80% Cohort A (4 of 5 patients) · 100% Cohort B (6 of 6 patients) · 100% Cohort C (4 of 4 patients)
(iii) Stool Frequency subscore, improvement or no change from baseline (% of patients achieving)
· 100% Cohort A (5 of 5 patients) · 100% Cohort B (6 of 6 patients) · 100% Cohort C (4 of 4 patients)
Improvement in Quality-of-Life, using the Short Inflammatory Bowel Disease Questionnaire (SIBDQ), was reported with more than 60 percent of patients in each cohort achieving a ≥10-point or more improvement after six weeks of treatment. At least half of patients in Cohorts B and C also showed ≥20-point or more improvement.
Brilacidin for UP/UPS also was shown to be generally well-tolerated, with no Serious Adverse Events (SAEs) experienced by patients, which is consistent with the low levels of systemic absorption of Brilacidin observed in the trial.
The Company has presented detailed topline results from the Brilacidin-UP/UPS trial, as well as information on the ongoing Brilacidin Phase 2 trial in the treatment of Oral Mucositis, at the Drug Discovery & Therapy World Congress in Boston, MA, held today. A corresponding slide deck is now posted to the Company’s website at the link below:
“As a practicing gastroenterologist, it’s extremely exciting to see Brilacidin’s emergence as a possible novel treatment for Inflammatory Bowel Disease. Brilacidin’s unique mechanism of action and compelling clinical trial results support its potential,” said Francis A. Farraye, MD, MSc, Clinical Director, Section of Gastroenterology at Boston Medical Center, Professor of Medicine at Boston University School of Medicine and Scientific Advisor to Innovation Pharmaceuticals. “There exists a large unmet medical need in treating patients with Inflammatory Bowel Disease given its complex pathogenesis and high degree of variability in how patients respond to a given treatment. I look forward to continuing to work closely with the Innovation Pharmaceuticals team as they advance Brilacidin in the treatment of IBD.”
Arthur P. Bertolino, MD, PhD, MBA, President and Chief Medical Officer at Innovation Pharmaceuticals, commented: “We are incredibly pleased with the successful completion of this trial and the dataset that we now have in-hand. To see such strong results with a basic formulation adds further credibility to where we believe we can take Brilacidin in treating the full continuum of IBD, particularly when optimally formulated and administered. Combined with the Brilacidin data for treating Oral Mucositis as a preventative medicine, we now have two solid anchors supporting Brilacidin’s considerable and diverse therapeutic potential.”
Study Overview
In this Phase 2 PoC trial, a total of 17 patients received treatment across three sequential, dose-escalated cohorts—Cohort A (6 patients); Cohort B (6 patients); and Cohort C (5 patients). Patients received Brilacidin, once daily, at 50 milligrams (mg), 100 mg and 200 mg, respectively, administered per rectum as a retention enema for 42 days (6 weeks) of treatment. The Primary Efficacy Endpoint of the Brilacidin UP/UPS trial used Modified Mayo Disease Activity Index (MMDAI) scoring, a common measurement tool in managing Ulcerative Colitis preferred by many IBD specialists, to determine Clinical Remission at Day 42. Secondary Efficacy Endpoints included: change in MMDAI score, both Full and Partial, and change in patient Quality-of-Life as assessed by the Short Inflammatory Bowel Disease Questionnaire (SIBDQ).
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