B-UP Due Diligence About Ulcerative

New Post Public Reply Private Reply Replies (3) Message Board
sox0407
806
B-UP Due Diligence

About Ulcerative Proctitis

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.

http://www.cellceutix.com/press-release/2016/...-proctitis

Trial Design

The primary objective of the P-o-C trial is to assess the frequency of clinical and endoscopic remission with Brilacidin administered per rectum in subjects with active UP or UPS after 6 weeks of treatment. Secondary objectives include evaluation of safety and tolerability of Brilacidin when administered per rectum, evaluation of clinical remission at Week 2 and Week 4, assessment of systemic exposure and/or pharmacokinetics of Brilacidin when administered per rectum, assessment of the efficiency of Brilacidin by biomarker evaluation of biopsy samples for interleukin (IL)-6 and IL-1beta, and estimation of statistical power for subsequent trial(s) in UP and UPS.

The P-o-C trial will include 18 patients divided evenly into three cohorts. Cohort A is receiving 50 milligrams (mg) of Brilacidin once daily for 42 days. Dosing will be increased to 100mg and 200mg 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 and adverse events) after 21 days of therapy for all six patients in each cohort before proceeding with initiating enrollment in the subsequent cohort.

http://www.cellceutix.com/press-release/2016/...-proctitis

Second Cohort Update (1/23/2017)

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.

http://www.cellceutix.com/press-release/2017/...d-outcomes

First/Second Cohort Update (12/7/2016)

Review of safety data from the first cohort revealed that Brilacidin, administered for 6 weeks as a retention enema, at 50 mg once daily, appeared well-tolerated, with no measurable systemic absorption detected. This drug profile, at the lowest dose, allowed the approval of dose escalation to the second cohort (100 mg, once daily). Clinically meaningful improvements in symptoms of UP/UPS were also demonstrated in the first cohort, as measured by physician assessments and patient reported outcomes, further supported with endoscopic evaluation of disease activity.

http://www.cellceutix.com/press-release/2016/...ve-colitis

First Cohort Update (10/10/2016)

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.

http://www.cellceutix.com/press-release/2016/...ve-colitis

First Patient Update (07/18/2016)

Cellceutix has been advised that the first patient in Cohort A remains on study. The clinical site administering Brilacidin to this patient has informed the Company, “Both the patient and the site staff have been amazed how the study drug works as all the symptoms decreased significantly within the 1st week of treatment.”

http://www.cellceutix.com/press-release/2016/...-proctitis

Q1 2017 Conference Call

During September we received initial comments on the first patient enrolled in our Phase 2 trial of Brilacidin for the inflammatory bowel disease, IBD, ulcerative proctitis or ulcerative proctosigmoiditis which detailed a significant decrease in symptoms within one week of Brilacidin activity. Early in the current quarter we followed that up with even better news of clinically meaningful improvements in the first four patients of the first cohort who completed the trial at the lowest dosing level. Yes, the lowest grossing level. Dr. Bertolino will provide some more color on the results. But the key is the fact that Brilacidin concentrations in the patient's plasma at all timeframes were basically undetectable. This is incredibly important in the safety profile that is being built for the FDA. We believe that a highly effective new drug with minimal systemic exposure will have competitive advantages to capture market share in a market for IBD medicine, the vision gain forecast to client to $9.3 billion in 2019.

Unidentified Analyst

Brilacidin seems to have very broad potential across a number of indications. Can you comment on why you chose to start with inflammatory bowel disease and ulcerative proctitis? Can you expand on the critical path for these indications and what other indications would you consider pursuing?

Leo Ehrlich

There is currently a significant medical need for effective treatment of inflammatory bowel disease. We look for massive markets with unmet needs, so IBD certainly qualified. The simplest way for us to do a proof-of-concept evaluation was to first explore ulcerative proctitis, ulcerative proctosigmoiditis. We have certainly been encouraged by preliminary results if it holds true that we can achieve significant clinical benefits with localized treatment and no significant systemic drug levels. We also anticipate potential for use in expanding IBD indications and in a number of dermatological diseases.

Unidentified Analyst

Dr. Bertolino, can you explain more about what is known of the science underlying the anti-inflammatory actions of Brilacidin?

Arthur P. Bertolino

Brilacidin is an immunomodulatory and anti-inflammatory agent. It inhibits the production of a number of key molecular targets, TNF-alpha, IL- 1 beta, MCP-1, MMP-9 and IL-6. Recent encouraging results in ulcerative proctitis, ulcerative proctosigmoiditis reassure us that the science is meaningful.

http://seekingalpha.com/article/4023682-cellc...transcript

Interim Data

"A: Let me first touch on the ulcerative proctitis (UP) and oral mucositis (OM) trials. The UP trial is progressing smoothly and barring any unforeseen complications, we think that we will have some interim results sometime next quarter. This is a proof-of-concept study, which I believe can have a significant impact on the value of the Brilacidin franchise by serving as a gateway to other gastrointestinal diseases and conditions.”

http://seekingalpha.com/article/3988240-inter...olino#alt2

Ulcerative Proctitis

Abstract

Ulcerative proctitis is an idiopathic mucosal inflammatory disease involving only the rectum and is therefore an anatomically limited form of ulcerative colitis. Diagnosis is made based on clinical presentation, endoscopic appearance, and histopathology. Additionally, other etiologies of proctitis are excluded. The course of the disease is variable ranging from complete resolution to easily maintained remission to frequent relapses or refractory disease. Extension of inflammatory changes involving the proximal colon occurs in some cases. Rectal 5-aminosalicylic acid (5-ASA) or steroids are the initial treatments of choice with oral 5-ASA, sulfasalazine, or steroids used for treatment failures or patients unable to tolerate rectally administered drugs. Immunomodulators like azathioprine and 6-mercaptopurine have been used successfully in small groups of patients who have not responded to 5-ASA or steroids. Oral or rectal 5-ASA products maintain remission but long-term steroid use should be avoided. Rare cases may require surgical therapy.

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2780078/

Foam Preparations

Abstract

Patients with ulcerative colitis uniformly have disease involving the distal colon. When patients have disease limited to the left colon or symptoms suggestive of active rectal inflammation, guidelines recommend topical rectal therapies as first-line agents either as monotherapy or in conjunction with oral products. Rectal delivery modalities offer the advantage of delivering high local concentrations of active medication to the site of maximal inflammation with minimization of systemic side effects. Methods of rectal administration include suppositories, liquid enemas and foams. Suppositories are limited to the treatment of rectal disease, and patients often have difficulty retaining the liquid enema secondary to its high volume and consistency. Rectal foams reliability extend to the descending and sigmoid colon with application. Foams are further characterized by increased viscosity, lower volumes, finer dispersion on the colonic mucosa, and increased adhesiveness to the colonic mucosa compared with liquid enemas. Additionally, rectal foam agents demonstrate equal efficacy to their liquid enema counterparts yet consistently yield better patient tolerance, lower incidence of side effects, and increased patient acceptability. Currently available agents include 5-aminosalicylic acid and corticosteroids, both first and newer generation. This review focuses on clinical trials assessing efficacy, tolerability, and patient preferences for these agents as well as describing the currently available rectal foam products.

http://www.ncbi.nlm.nih.gov/pubmed/21235478

Placebo Response Rate

Experience with foam preparations has demonstrated better retention and adherence, as well as more uniform distribution of medication in the distal colon and rectum, as compared with enemas and suppositories.

Analysis of the individual trials showed that 38.3% and 44% of patients in the budesonide arm achieved remission at 6 weeks as compared with 25.8% and 22.4% of the placebo groups (P=0.0324 and P<0.0001, respectively). Combined results showed a remission rate of 41.2% with budesonide foam and 24% with placebo (P<0.0001).

http://www.medpagetoday.com/meetingcoverage/acg/42311

Current and Future Therapies

Ulcerative colitis (UC) and Crohn’s disease (CD) are severe ailments affecting the digestive system.

UC is a gastrointestinal (GI) disease that is localized to the large intestine, or colon, where inflammation can affect either the entire organ or a portion of it. Approximately 1.86 billion patients have been diagnosed with UC globally, with 1.54 billion patients currently receiving treatment. Traditional therapies have yielded $4.18 billion in annual sales around the world, a figure expected to increase to $6.85 billion by 2022 with the approval of various pipeline drugs.

CD can affect any part of the GI tract, but most commonly involves both the large and small intestines. Although CD is more severe than UC, the global prevalence is much lower, with only 1.3 million patients diagnosed and 0.8 million who currently receive treatment. Still, traditional therapies have resulted in an impressive $3.17 billion in global market sales, which is predicted to increase to $4.20 billion by 2022.

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4123809/

Potential Market

The global ulcerative colitis (UC) market value will increase steadily over the coming years, growing from almost $4.2 billion in 2012 to approximately $6.6 billion by 2022, at a Compound Annual Growth Rate (CAGR) of 4.7%, according to a new report from research and consulting firm GlobalData.

Johnson & Johnson (J&J) has been continually dominating the UC market due to the success of Remicade. However, Remicade’s patent is set to expire between 2015 and 2018, which will knock J&J’s sales down from approximately $2 billion in 2012 to $1.5 billion by 2022, according to GlobalData.

Another key player in the UC industry, AbbVie’s Humira, will also lose its patent during the forecast period, with sales slipping from $1.2 billion in 2012 to $569 million by 2022.

https://healthcare.globaldata.com/media-cente...globaldata



http://www.prnewswire.com/news-releases/anti-...52061.html

Dr. Francis A. Farraye – Scientific Advisor, Gastroenterology

Dr. Francis A. Farraye serves as Professor of Medicine, Clinical Director, Section of Gastroenterology and Co-Director, Center for Digestive Disorders, at Boston University School of Medicine. Dr. Farraye is a Fellow of the American College of Physicians, American Society of Gastrointestinal Endoscopy, American Gastroenterological Association and the American College of Gastroenterology. Across his professional career, he has published over 350 original manuscripts, abstracts and book chapters, with particular clinical interest in inflammatory bowel disease and colorectal cancer. Dr. Farraye has been named Humanitarian of the Year by the New England Chapter of the Chrohn’s & Colitis Foundation of America. Dr. Farraye received a MD from Albert Einstein College of Medicine in New York and a MSc (Epidemiology) from Harvard University.

http://www.cellceutix.com/senior-management-1/

Books

Questions & Answers About Ulcerative Colitis

https://books.google.com/books?id=45iIz7k14pw...mp;f=false

Curbside Consultations in IBD: 49 Clinical Questions

https://books.google.com/books?id=JQaR2Sk2Ek4...mp;f=false

Presentations

Health Maintenance for your IBD Patient (Slide 104)

http://www.galamericas.org/dallas/library/PPT...isease.pdf

The role of chromoendoscopy in ulcerative colitis patients: Should we incorporate the SCENIC recommendations? (Slide 10)

http://advancesinibd.com/archive/2015/present...BD_Med.pdf

Publications

https://www.researchgate.net/profile/Francis_...blications

YouTube

Diagnosis and Management of Colorectal Neoplasia in Inflammatory Bowel Disease

https://www.youtube.com/watch?v=kV5-9qwetmc

Innovation Pharmaceuticals Inc (IPIX) Stock Research Links

IPIX Board Company Profile Buy Rating Time & Sales News Filings Financials
Dont-give-up.jpg
Scroll down for more posts ▼

Top 10 Most Recent News Articles

Innovation Drives New Asia-Pacific Opportunities

Updated Category News Views 0

A Glimpse Into The Future: Asia-Pacific's Tech Landscape There's a unique transformation bubbling across the Asia-Pacific scene, with young minds pushing the envelope on technology and innovation. Take Nathapol's team from Thailand, for example. They're merging bionic tech with drones for disaster relief—bold, ambitious, and nearly clinching glory at the Lancang-Mekong...

Continue Reading
Aurra Markets Wins 'Most Transparent Broker' Award 2026

Updated Category News Views 2

Spotlight on Transparency in Trading Alright, so Aurra Markets snagged the 'Most Transparent Broker' award at the Money Expo in Mumbai this year. From the greasy streets of trading floors to the polished halls of Jio World Convention Centre, transparency is one of those golden words you hear tossed around, but like finding a needle in a haystack, it’s not always easy to...

Continue Reading
HashKey and ONERWAY Pave New Routes for Digital Payments

Updated Category News Views 3

New World of On-Chain Payments The landscape of on-chain payments is shifting like sands in a desert storm, but this time HashKey's Settlement Protocol (HSP) ties up with ONERWAY, giving a new direction. These players are looking to redefine how merchants transact with digital currency, tying up loose ends with fiat systems. Strategic Partnership: What’s At Stake? When...

Continue Reading
Better's 90-Day Plan: Growth Amidst Governance Gamble

Updated Category News Views 0

Unpacking the Ambitious 90-Day Plan When you hear Vishal Garg's name next to a 90-day sprint plan, it's not your everyday boardroom shuffle—you know there's grit behind the screen. Better Home & Finance (BETR) is angling for a massive turnaround with its latest rollout, but whether it's all sizzle and no steak is what we're here to figure out. Financial Metrics and...

Continue Reading
Innovative Lighting Idea Brightens Indoor Lives

Updated Category News Views 2

Shedding Light on SUN ORB's Indoor Revolution Sometimes, the best ideas come from simple needs, like wanting a bit of sunshine in your life. That's the deal with this SUN ORB concept—an invention dreamed up by a chap from Oklahoma who's spent too much time away from the natural light outside. We're talking about a wellness-oriented lighting idea that has big ambitions...

Continue Reading
Alcatraz AI: A Silent Titan in Data Center Security

Updated Category News Views 0

Why Alcatraz AI is the Security of Choice Forget flashy press releases and splashy marketing campaigns. Alcatraz AI, a company operating out of the hustle and bustle of Cupertino, has managed to quietly dominate a key sector without many folks being the wiser. What's their claim to fame, you ask? They're the chosen gatekeepers for over half of the hyperscale data centers...

Continue Reading
Nonprofit Option Rises for Specialty Physicians

Updated Category News Views 1

Reimaging Choices for Specialty Physicians In the whirlwind of healthcare, specialty physicians often find themselves between a rock and a hard place. The traditional paths are either hug a major health system or auction off to private equity, each option chaining them to systems more interested in the balance sheet than in patient care. But now, a third door opens. One...

Continue Reading
Beverly Hills MD's Dermal Cream Wins Key Beauty Award

Updated Category News Views 1

A Surprise Win in the Beauty World Who doesn't love an underdog story, right? Beverly Hills MD has slid into the limelight with their Dermal Repair Complex snagging the award for 'Beauty Product of the Year.' Talk about sending shockwaves through the beauty industry! Now, these guys might not be a household name yet, but you better believe they've got everyone's attention...

Continue Reading
Exploring Before and After School Care Dynamics in PA

Updated Category News Views 1

Navigating the World of Before and After School Care The dad juggling a morning coffee and a laptop at a crowded diner—he’s not just browsing social media; he's flipping through options for his kid’s care. In Harrisburg, PA, before and after school care isn't just babysitting, it’s a lifeline for working parents balancing the chaos of modern schedules. Timothy R....

Continue Reading
Hamamatsu & AIxMed to Automate Cytology Workflows

Updated Category News Views 2

Why This Collaboration Matters Here comes an interesting tidbit from the world of cytology: Hamamatsu and AIxMed are banding together to tackle workflow inefficiencies. Picture this—two powerhouses coming together to create a tool that might just relieve lab technicians from the tedious task of manually checking cytology slides. In a world where efficiency is king, this...

Continue Reading

Top 5 Most Recently Viewed Articles

SISQUAL® WFM Lands Major Contract with Norwegian Police

Updated Category News Views 11

New Deal to Enhance Police Operations SISQUAL® WFM snagged a plum contract to pimp Norway’s boys in blue with some top-drawer workforce management solutions. The Portuguese outfit snagged the public tender, making headlines with Devoteam lending a hand on the ground. The Backbone of Law Enforcement Operations This ain’t just a standard-issue software sale, folks. If...

Continue Reading
Tesla Leads California’s EV Market Amid Declining Registrations

Updated Category News Views 92

Tesla Remains the Leader in California’s EV Market Electric vehicles (EVs) have continued to gain traction in California, with battery electric vehicle (BEV) registrations showing a modest growth of 1% year-on-year, totaling 293,109 units by September. This figure represents a significant portion of new vehicle registrations, indicating a shift in consumer preferences...

Continue Reading
Maximize Your Earnings with Alphabet Stock's Dividend Potential

Updated Category News Views 103

Understanding Alphabet Inc. and Its Dividend Potential Alphabet Inc. (NASDAQ: GOOGL) is one of the most influential and innovative companies in the technology sector. As the parent company of Google, Alphabet has consistently made headlines for its groundbreaking advancements and financial performance. Investors are keenly interested in Alphabet, particularly in light of...

Continue Reading
Mitel's CEM Award: Shaping Future Crisis Management

Updated Category News Views 377

Mitel's Recognition for Exceptional Crisis Management Solutions Mitel has recently been recognized for its groundbreaking Critical Event Management (CEM) solutions. This award underscores their role in enhancing resilience and ensuring continuity in times of crisis. The innovative approach adopted by Mitel CEM allows organizations to consolidate risk intelligence,...

Continue Reading
Pollo Campero Collaborates with New York City FC for 2025 Season

Updated Category News Views 367

Pollo Campero Teams Up with New York City FC Beloved fried chicken restaurant Pollo Campero has announced a vibrant new partnership with New York City FC. This alliance marks a significant collaboration, as Pollo Campero plans to engage with fans in an exciting way during the upcoming soccer season. Celebrating Community and Culture The partnership embodies a shared...

Continue Reading