Innovation Pharmaceuticals Inc (IPIX)

[georgejjl]

Cellceutix: A New Standard Of Care In Oral Mucositis In The Making

About half a million people annually suffer from oral mucositis, a painful side effect of chemoradiation with no FDA-approved prevention medication.

Current therapies are mostly just coatings to help numb the pain and modestly support healing – a prophylactic should dominate the market.

Interim data from CTIX’s Phase 2 trial showed only 22.2% of patients treated with Brilacidin-OM developed severe oral mucositis (Placebo group = 70%).

More than just for oral mucositis – Brilacidin’s antibiotic, anti-inflammatory and immunomodulatory qualities are supported by mid-stage clinical trials for other indications.

Throughout years of discovery, many companies have reported being on the edge of something truly effective to treat oral mucositis, yet they all seem to come up short. Speaking in the broad sense, most treatments are little more than coatings to help ease the symptoms of the often-excruciating side effect of radiation and chemotherapy therapy for head and neck cancer patients.

That's why most hospitals whip up their own concoctions of "magic mouthwash," typically a mix of antibiotics, antifungals, corticosteroids, antacids and antihistamines. A lack of efficacy comparatively to magic mouthwashes also underscores why insurance companies don't want to reimburse for most other rinses. That and the fact that once severe oral mucositis has presented, medical bills start shooting upward, so there's little value to reimbursing for these types of non-preventative medicines from an insurance company's perspective.

The FDA still hasn't approved anything for preventing oral mucositis. It's not as if there isn't a market; it's that no one has been able to successfully develop a reliable, safe product. Some 500,000 people a year are affected by oral mucositis in the U.S. Additional costs associated with the condition are estimated at up to $25,000, so it's an attractive market opportunity.

In the meantime, patients will be given things like Amifostine, Gelclair, Zilactin, doxepin, keratinocyte and any number of drug cocktails to try and lessen the pain and ulcers associated with oral mucositis.

The winds of change are blowing on that front, though. Cellceutix Corp. (OTCQB: OTCQB:CTIX) on Monday reported interim results from their company-sponsored Phase 2 trial of Brilacidin-OM for oral mucositis. In the ongoing trial, Brilacidin-OM is being evaluated for safety and efficacy to prevent the onset and reduce the symptoms of oral mucositis in head and neck cancer patients undergoing chemoradiation.

The double-blind trial, which Cellceutix hopes to wrap-up this year, is designed to enroll 60 patients (30 in the B-OM group and 30 in placebo group). Per protocol, patients are required to swish and spit Brilacidin-OM 3x daily for 49 days.

The primary endpoints are evaluation of the safety and tolerability of Brilacidin-OM and the incidence of severe oral mucositis experience across seven weeks of radiation. Severe oral mucositis is defined as a Grade 3 or above score on the World Health Organization's Oral Mucositis Grading Scale. WHO's Scale for OM goes from Grade 0 (none) to Grade 4 (ulceration/pseudomembrane formation, patient can't tolerate solid or liquid food). Grade 3 is identified as ulceration/pseudomembrane with the patient on a liquid diet.

Notice the main goal of the trial is squarely pointed at preventing oral mucositis.

A secondary endpoint was looking at the duration of severe oral mucositis.

19 patients were evaluable for the interim analysis, 9 in the treatment group and 10 in the placebo group. The data showed that 7 out of 10 patients in the placebo group, or 70%, ended up with severe oral mucositis.

Of the 9 patients treated with Brilacidin-OM, only 2 (22.2%) developed Grade 3 oral mucositis. And those that did experience severe OM didn't have to cope with it as long. The mean duration for staying at or above Grade 3 was 10.5 days for the two patients on Brilacidin, compared to 14 days for the seven patients on placebo.

No serious adverse events were attributed to Brilacidin, proving in yet another trial that the novel drug is safe.

Granted, the sample size is small, but that's the type of data that should get investors excited and get the attention of big pharma as a true prophylactic medicine for oral mucositis.

What's most interesting here is the big picture for Brilacidin. Discovered at the University of Pennsylvania, the non-peptide, small molecule compound is modeled after host defense proteins (hence the name "defensin-mimetic"), which are the front-line of defense in the body's innate immune system. Due to its structure, the compound is amazingly diverse, have antibiotic, anti-inflammatory and immunomodulatory qualities.

In a Phase 2b study, Brilacidin stood toe-to-toe with the blockbuster antibiotic daptomycin to treat Gram-positive acute bacterial skin and skin structure infections, showing non-inferiority with a similar safety profile for a single dose of Brilacidin versus a 7-day therapy regimen for daptomycin. To be clear, the antibiotic properties in one infusion of Brilacidin rivaled that of a drug doing over $1 billion in annual sales that takes seven days to complete the course.

On the anti-inflammatory and immunomodulatory front, Brilacidin is being evaluated in retention enema form to treat ulcerative proctitis/ulcerative proctosigmoiditis, two versions of inflammatory bowel disease. Interim data from the ongoing Phase 2 study, which is in its third and final cohort and should be completed in the next three months, showed all 12 patients treated with Brilacidin experienced a clinically meaningful response as measured by the Modified Mayo Disease Activity Index.

As discussed above, the oral mucositis trial - which leverages the anti-inflammatory and immunomodulatory properties first to fight onset of oral mucositis and then likely gets some benefit from the antibiotic activity should the mouth develop any infection - also has provided very optimistic data to date.

When you start thinking about the natural extensions of Brilacidin and the next logical indications for growth and value building, you'll see that they abound. Certainly, rectal mucositis should enter the equation. How about radiation proctitis/colitis? In reality, the whole inflammatory bowel disease spectrum becomes a possibility.

So, when Cellceutix discloses as much as it can about big pharma being interested, it doesn't necessarily mean that they are particularly focused on one thing (although they could be). The greatest value resides in novel experimental drugs that can bridge many indications. Because a drug like Brilacidin is completely novel, big pharma is going to move cautiously and want to see several pieces of data demonstrating its potential before they put their money on the line in a partnership, much less and acquisition.

It seems logical that is what is happening and that Cellceutix is delivering the necessary clinical data. Staying grounded on facts, it seems only a matter of time before the company achieves its stated goal of striking a partnership for late-stage development of one of the drugs in its pipeline.

That's not without recognizing that Cellceutix has downside risk, just like any other biotech or OTC company. The company must secure additional capital above and beyond its share purchase agreement with Aspire Capital to fund additional studies and cover G&A expenses in the coming years. To that point, dilution remains a concern with shares trading around $1.00 following several hit pieces that were apparently part of a short attack on the stock.

I believe that Cellceutix has its risk mitigated because not only does it have its defensin-mimetic franchise, it also has Kevetrin, a novel compound acting upon the p53 pathway to treat cancer currently in a Phase 2 for ovarian cancer, and Prurisol, an ester of abacavir that in a Phase 2b trial as a new oral treatment for psoriasis. Both drugs have incredible upside potential, but I'll save that discussion for a later article and encourage investors to look into the litany of data published by credible sources to learn more about them in the meantime.

For this exercise, I wanted to stay focused on Brilacidin and put a circle around the fact that the data to date is very encouraging for a breakthrough in an indication like oral mucositis all by itself, while touching on the idea that there is overlap underscored by the multiple properties of the compound that are being further documented on a nearly monthly basis. The bottom line here is that if the data continues to hold its current course, I believe a partnership that catalyzes this stock once again is in the cards for Cellceutix and its shareholders.

https://seekingalpha.com/article/4059084-cellceutix-new-standard-care-oral-mucositis-making

Good luck and GOD bless,

George