nationwide allergy.net is a scam. They are setting patients up to receive 2 formulations of immunotherapy simultaneously with the sole intent of maximum billing of the patient's insurance (for the injectable formulation, which is billable) with the full knowledge and expectation that the patient will not initiate or attempt to complete the treatment. The patient is enticed into participating because they get the second (sublingual, non-billable) formulation free of charge. Probably a kick-back in there too somewhere. So shady!!

It is a shame that Arrayit is associated with them. The microarray testing platform is legitimate. I toured their facility and reviewed their allergy testing panels several years ago just as they were getting underway for allergy.

Haha! How funny, my first research job out of college was in this lab (Dr. Abrams). I worked with fruit flies and learned to do some basic molecular biology before moving to Boston to work in immunology. I actually got my name on my first publication as a research assistant there. I doubt he's involved in the K2 trial, but perhaps he could be helping with the genomic mapping stuff....anything is possible I suppose.

The info on Clintrials.gov states the expected end date for K2a is 12/31/17. Keep in mind that this is a sequential study, which means the first cohort has to complete 3 weeks of study + 3 weeks of post treatment evaluation. Also, the 5 patients will not start on the same date....they have to be recruited, consented, and pass all inclusion/exclusion criteria.

I'd conservatively allow at least 3-4 months for each cohort to complete. You also have to allow a reasonable amount of time to schedule a meeting of the DMC to review patient safety issues and determine if cohort #2 can proceed. Assuming this will likely occur over the Summer, when docs are notoriously hard to get into 1 room together, it is easy to see why this trial could take 10-12 moths to complete.

It would be amazing if this is being conducted at UT Southwestern or Baylor, but I imagine its being run at another for-profit cancer treatment & research center.

The last 5-6 weeks have been horrible for all of biopharma. Go look at IBB since 9/12 and you can almost overlay exactly with CTIX's chart. The recent downturn has nothing specific to do with CTIX study progression or management. It has everything to do with sour attitudes about biotech as a whole due to the upcoming election.

Many folks in this sector seem to believe Clinton will aggressively go after BP profits....probably somewhat true, but not to the extreme that the fear mongers are predicting. At the end of the day, sick people still want new meds, and they want them fast. That all costs money to accomplish....so Relax!

I don't see a turn north coming until after the election, and probably not until beginning of 2017. But please please please stop spreading the same "sky is falling" nonsense on here. It is not based in fact and only muddies the water.

Can someone remind me who our CRO is???


RE: "He has worked diligently with our CRO (for the planned Phase 3 ABSSSI study). They already have selected the first sites for the study that can start once we get the final protocol approval from the FDA. I wish him and his family well in his new endeavor," said Mr. Ehrlich

Whoa! Whoa! Whoa! Re: the most recent SA article. Does anyone else believe that this article could have been written by the same mo-fo's who penned the garbage Mako article from last August?

I have a few problems with this article and worry that it appears to be a well-crafted piece set on manipulation. Let me state clearly at the onset here that I am long CTIX and I am heavily invested since 2013, shortly after the PYMX acquisition. I am simply trying to point out my personal concerns about the tone (and intent) of this article.

Problem #1 – I agree with a few of the other skeptical comments on the article claiming that it is not balanced. While I found that none of the articles’ claims were uniquely deceiving, it was the profusely “rosey” tone and the intentional disregard of certain troubling aspects about the company and its drugs in development that made me furrow my eyebrows.

Problem #2 – the article’s countless charts touting the potential for success based on CTIX’s current stockpile of drugs and position in the marketplace reeks of bait & switch while entirely glancing over some real concerns like the selection of the Board of Directors, and a lack of expertise in the executive office.

Problem #3 (and most controversial I’m sure) - the article shares an eerily similar tone and structure as the Mako hit piece written in August of last year, albeit with a completely alternate goal. I know many will label this as conspiracy run amuck, but if you dig a little deeper and analyze the writing more closely, it could almost be written by the same person. My rationale (i.e. paranoia) is based on the following:

1. Both articles come from an Anonymous author (or authors) – I do not understand the desire to write a lengthy and heavily sourced blog article on SA and remain completely anonymous. In both the Mako case and in this case, it seems plausible that the intent is to drive the market’s perception one way or another for personal gain. A lot of work went into the articles, and it stands to reason that only someone with something substantial to gain would go to that effort.

2. Both tout their investing shrewdness and the more recent article claims a hefty stake in CTIX by the author(s) – seven figures by their own account, which would equate to at least 600,000 shares at the most recent closing prices. Such a stake would certainly be positioned to benefit substantially from even a 2-fold move in the share price.

3. Both articles make use of 5 summary bullet points at the onset of the article that are essentially counterpoints of each other. This could certainly be intentional by the most current author, but it certainly raised my eyebrows when they were lined up side by side.

4. Both articles make use of superfluous charts and graphs to create a somewhat dizzying concoction of evidence in support of their case. It almost seems as both are using some form of smoke and mirrors, the most recent trying to hide an otherwise biased attempt to pump stock’s public perception (the other openly attempting to annihilate it).

5. Both authors claim to own a treasure trove of additional data and resources to back up their positions, yet both articles only seem to skim the surface of the actual meat of the arguments in favor of (or against) the drugs in development. Overall, both authors demonstrated a similar cursory understanding of the actual science, while touting a much deeper appreciation that they really can’t back up in print. Therefore, it seems once again, both articles could have been written by the same person who has a limited understanding of the actual science, but who is instead (at least in this most recent case) a wolf in sheep’s clothing intent on driving the stock price in a particular direction, and at a particular time.

In summary, I am 100% in support of CTIX and its pipeline. Also, I’m not a sophisticated investor. I am a scientist (over 20 years in Immunology & Microbiology) and clinical consultant who is a self-proclaimed devil’s advocate and skeptic, and what I see in the most recent SA article is something that made my eyebrows raise right away…in other words, my BS detector started beeping….not loudly, but enough for me to write this friendly “buyer beware” post. My theory is that these are individuals who are building the roller coaster so they can ride it over and over. On the other hand, it is plausible that the article is nothing more than a slightly-less-than-well-balanced summary of the company.

1. Peer review of the data = increased legitimacy of the science, the clinical development team, and the company as a whole
2. Publication in a high impact journal = greater exposure to the science and clinical pipeline
3. Thorough explanation of the materials & methods, and a review of specific patient outcomes = further insight into the potential success of the drug
4. P21 data! Enough said.

I asked about the company's publication plan and Leo responded with "which drug?" I said, all of them, but Kevetrin Phase 1 data to start. He deferred to Dr. Menon who gave a very vague answer about how the manuscript has been submitted and it takes time due to the lengthy review process.

Basically, I got the feeling that Dr. Menon is either too busy, too distracted, or having health problems and cannot dedicate the time to put publishing as a priority (or some combo of all three). I also wonder how much experience he has writing manuscripts as lead author (I'm thinking very little) and also with human clinical study data (probably none).

He didn't disclose the journal where the manuscript was submitted, and he did not clarify the time frame for eventual publication, so I can only assume that the paper is in the review process, which I agree can take a considerable amount of time. There's a lot of back and forth with the reviewers and having to respond or make edits tot he paper. Not to mention, I believe a number of investigators at Dana Farber are probably co-authors so I assume it is difficult to track them down to review changes.

People have clambered on about visibility of the company and attracting new investors. I would have thought publishing data is a [relatively] cheap and sure fire way of gaining attention. I would like to see the pre-clinical and mechanism of action data on Kevetrin published (not just as a poster at a conference), as we all as the pre-clinical stuff on Prurisol. I think this is all very noteworthy data that would easily get accepted in a top tier journal.

So, a strategic publication plan is on my holiday wish list for CTIX, along with a VP of Clinical Development, a VP of Regulatory Affairs, a reliable IR firm, and up-listing to Nasdaq (duh!).

The hamster swishing comment was meant as a joke (presumably an obvious one).

I don't think anyone here is trying to quantitatively predict outcomes in humans based on results in rodent studies. I believe the sentiment by most on this board is that we are hopeful....hopeful that the patient's qualitative experience in the Ph2 B-OM are comparable to what was seen in the pre-clinical hamster study.

I will argue, however, that it is certainly reasonable to expect that the B-OM outcomes may be quantitatively similar to those seen in hamsters....otherwise, what's the point in declaring a rodent model as a gold standard for pre-clinical evaluation, if it isn't predictive of results in humans?!? Isn't that the purpose of having an animal model in the first place....because it is predictive??

Edit: pre-clinical studies on B-OM were in hamsters, not mice (source: CTIX Corporate Presentation, Nov 2013). I suppose its easier to teach hamsters to swish an oral rinse. In either case, it doesn't change my assessment.

Haha! No food at the meeting, but I did see a nice Cellceutix baseball cap in one of the offices, and thought it would have been nice to get one of those. I was secretly hoping for a swag bag of goodies, but alas, all we got was a print out of the "Forward-Looking Statements" disclaimer. Yay!

How will B-OM stand up to SGX942 -

Obviously, we don't have efficacy data on B-OM, and others have aptly pointed out that fact. However, IF our Ph2 results are anything like the preclinical mouse outcomes, then this should be a no-brainer. Here is my rationale:

1. Dosing regimen - B-OM is given 3x a day, for 7 weeks as an oral rinse. It was mentioned at the meeting yesterday that this consists of a 16mL solution (Mint flavored??? HA!) and is taken at home by the patient. So, which would you prefer...go into the oncology clinic and get set up for IV infusion for an hour on the SGX drug, or swish around some B-OM oral rinse at home a few times a day?

2. Outcomes - it was just reported that average duration of severe OM was decreased by 50% in the SGX Ph2 study, and 67% in the heavy radiation group. While this sounds impressive on first reading, think about what that means....it basically says that almost every one still got a bad case of OM, but the duration went from 30 days to 15 days (on average). That's still 2 weeks of debilitating ulceration in the mouth and throat....or imagine having the worst case of strep throat for 2 weeks. B-OM is believed to help reduce the overall severity of OM, including the duration of symptoms. If you look back at the mouse data, the inflammation in the oral cavity was significantly reduced, to the point that OM scores were at or near their lowest category. If we can achieve even half that result in humans, then the data will be very compelling compared to SGX [IMHO].

3. Study design - I went to clinicaltrials.gov and looked closely at the details of both the SGX study and B-OM, and noticed something that caught my eye. The Primary Purpose of the SGX study is listed as "Treatment" whereas B-OM is listed as "Supportive Care". Here are the definitions from the FDA:

Primary Purpose: Treatment - protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition

Primary Purpose: Supportive Care - protocol designed to evaluate one or more interventions where the primary intent is to maximize comfort, minimize side effects or mitigate against a decline in the subject's health or function. In general, supportive care interventions are not intended to cure a disease.

I'm not savvy enough to understand the differences of these as far as trial design, but I am imagining that the "Treatment" classification might require a higher threshold for statistical significance and different outcomes measures, as compared to "Supportive Care". If so, then this could bode well for CTIX come time to submit the NDA. In other words, B-OM is not intending to "Cure a disease", but rather provide prevention and relief from debilitating side effects of chemo & radiation. SGX on the other hand may be required to demonstrate some clinical effect on the condition itself (think clinical immunology or biomarkers to show treatment effect).

So, this is a long post, but I wanted to provide my thoughts on the potential of B-OM, and how I'm not even sweating the fact that we appear to be slightly behind in the development timeline. In the end, I expect B-OM to be very competitive, and possibly the treatment of choice [if approved].

P.S., yes, I attended the meeting and had some very nice conversations with folks (employees and investors), and came away very impressed. I plan to provide a more detailed account of my experience and takeaways when I have a chance (long flight back to TX last night).

Do I need to RSVP for the meeting?

I will be in Boston this weekend and I plan on attending the meeting on Tuesday. Can someone let me know if I need to RSVP or do I just show up at 10am? This is my first time attending something like this.

Just a small correction, and I think most are aware, but Dr. Frei passed in 2013. The description you posted talks about him in the present tense. I believe the CTIX website has him appropriately listed as 'In Memorium'.

Dr. Frei was a brilliant oncologist and a tenacious researcher, and I agree that his endorsement of Kevetrin probably helped pave the way to get the drug into human trials. Those at DF and elsewhere in the oncology research community continue to revere his name. Seeing him affiliated with CTIX caused me to raise an eyebrow when I was first researching this company.

I read the article and other than poor editing, I don't see anything that appears to make a false claim regarding Dr. Menon's PhD. It simply states that Dr. Frei admitted Menon as a student in his Harvard lab "under his guidance". This is very different than being admitted by Harvard University as a PhD student by a specific program.

When I was a PhD student at Harvard Medical School in the Division of Medical Sciences, Immunology PhD program, I rotated through various labs. It was not uncommon for the Principle Investigator (PI) to take on students from other universities. It was often considered prestigious to have completed part or all of your research work in a Harvard lab, so many universities in other countries will allow their students to finish their work on a "visiting student" status. It is very likely that this was the arrangement for Menon, but I can't be certain without all of the facts.

I can see how someone reading the article might conclude that Menon claimed to have gotten his PhD at Harvard, but in essence, its simply the author of the article not clarifying the facts. If anyone is at fault its the author and/or editor of the magazine.

For the record, I dropped out of Harvard after 3 years without earning my PhD, but I gained valuable experience and met some very dedicated scientists from all over the world, many who were probably just like Dr. Menon (based on second hand accounts from those who've had the pleasure of speaking with him). Despite having only a BS in Microbiology, I still consider myself more of an Immunologist....its my life passion.

I would argue that it is very difficult to compare CTIX with the other immunotherapeutic companies you cite. A small molecule drug like K (a true pharmaceutical by definition) is quite different than a therapy that requires harvesting someone's lymphocytes, culturing them ex-vivo (i.e. in a petri dish) to re-program and amplify them, and then infusing now several million cells back into the patient, all with the very real risk of triggering a rapid, and potentially irreversible "cytokine storm" that causes the patient to spike a horrible fever, and possibly die. Also, these therapies currently only work on soft tissue or blood cancers. They've shown limited success on solid tumors. But, when it works, it can appear to be a miracle-like cure...and one that will certainly capture a lot of media attention.

So, we should careful about how we compare technologies and whether possible BP interest in acquisition or partnership is past due.

Now, IMHO I would bet that BP is a little gun shy about a small, unproven company like CTIX, not because of the science, but more due to the unorthodox management structure. When you compare small companies similar in size and stage as CTIX, it is more common to find a deeper roster on the "About Us/Leadership" page on the website. Even the really small companies have some individuals on the mgmt team with some form of experience in Reg Affairs, Clinical Development, etc. as well as a more targeted and diverse group of individuals on the BOD. Its not a bad thing that CTIX is running very lean and choosing to outsource many of the activities that are commonly managed more carefully in house, but from an outsiders perspective, I can see how it looks like 2 or 3 guys with too much personal investment in a company and who are likely unwilling to negotiate a deal at a reasonable price. Personally, I don't mind that Leo & Co. are holding out for the BIG deal....seeing as it will benefit all of us longs. But, in doing so, we have to wait for the science to prove itself worthy of the price tag that Leo may be seeking. Again IMO, I think it will be worth the wait.

You are correct that the blinding of the study technically only refers to the study participants and the study site physicians. In some cases, the sponsor/company may have a clinical development team that is keeping tabs on the study, but usually this will be restricted to overall & per site enrollment numbers, serious adverse events, and possibly some general observations from the physicians at each study site (though these observations are very limited in a blinded study).

In the case of CTIX, the company is contracting with a CRO that is overseeing most, if not all aspects of the B-OM study. I seriously doubt Leo or Menon will see much of anything going on with the study until data is officially available after the data is locked and analyzed. This is actually a good thing!

In the case of Kevetrin Ph1 at Dana Farber, the study is open-label and there is no placebo, so everyone knows that anything that happens with the patients in that study (good or bad) could possibly be drug-related. The observations are probably shared with the company on a regular basis.

For B-OM, the company will be in the dark because every patient enrolled receives a patient ID number, all drug & placebo is barcoded by a third party, so nobody will know who is receiving live drug. CTIX will unlikely receive any substantial updates until after the CRO collects all data, closes out each study site, and prepares a final study report for CTIX management. It will be at this point that Leo an Menon will have an idea about how well B performed and they can then decide how to inform shareholders. In rare cases there may be a mid-protocol data evaluation....usually for compassionate use purposes.

Of course, I don't know the exact study design, or how data is being collected, but it is probably some form of self-reported symptoms from the patient with regular office visits so the clinical team can perform a physical exam and collect patient diaries (among other study required data). If everyone is showing signs of OM with similar severity, then that could be a bad indication. If everyone is showing very little or no OM, then that could also be a bad sign that the inclusion criteria was not appropriate, or there is a huge placebo effect. Ideally, about 50% of the study participants would exhibit a pretty severe case of OM with the other half showing mild or no symptoms of OM. However, because no single study site will no what % of patients are enrolled on live drug, they will not be able to make any interpretations on patient observations alone.

As always, this is all IMHO based on my limited clinical study experience. Personally, I have a high degree of confidence that Brilacidin will be a game changer, both as an anti-infective and as an anti-inflammatory. I am eager for results like everyone else, but I am patient to let the studies run their course.

Actually, it is unlikely that we will know if Brilacidin is working until after the trial is completed. There may be some hope or limited confidence if ~50% of the patients enrolled never develop OM symptoms. However, this study is randomized 1:1 which means that patients are enrolled into the trial based on their demographics so it is possible that any given study site could have all patients on drug, or all patients on placebo, or half and half, or any combination of drug:placebo participants. All that matters is that ~50% of study participants across all sites receive Brilacidin, and those patients should be similar in demographics to the placebo group.

Also, because the trial is double blinded, the company won't know who is on drug or placebo, nor will the physicians on site, or the patients. Everything will be barcoded, and all dispensed product (drug or placebo) will look identical. They will also make every effort to make sure both placebo and B-OM look and taste the same. The CRO that CTIX contracted to run the study will have another 3rd party company that is responsible for dispensing, labeling and shipping both the study drug and placebo. Until the study is completed and unblinded, no one will know who is getting Brilacidin. In some situations there can be an inadvertent unblinding due to unmistakeable differences in drug and placebo, but in most cases these companies do a good job keeping everyone in the dark.

I was involved in a few allergy immunotherapy studies (allergy shots & drops), and we would have patients in our study visit an environmental exposure chamber (EEC) after they had been treated for 1 year with either our drug, or placebo. The EEC was this room where study participants would sit and have ragweed pollen or pulverized cat hair blown into the room. When observing the patients from an adjacent room, we would see quite a few reaching frequently for kleenex, and we were praying those were the placebo patients....but we couldn't be certain until it was all said and done.

Time frame for enrollment of B-OM due to inclusion criteria....I don't know much about oncology clinical trials specifically, but I know a little about trial design in general. I am assuming the investigators are aiming to find patients who are most likely to develop moderate-to-severe OM following their radiation and chemo treatments. To do so, they set the inclusion criteria very carefully, and often times very selectively. Unfortunately this can lead to longer recruitment times.

This is important in a placebo-controlled trial to tease out whether the drug had its intended affect. So, the inclusion criteria are set very stringently to make sure patients are highly likely to develop significant OM. If the inclusion criteria were set more relaxed, there may be a higher likelihood that patients with less severe OM are included (or worse, those who have some spontaneous remission of their OM symptoms during the treatment period). Thus, it becomes a nightmare to calculate a statistically significant delta (i.e. change) for OM score, and whether it is due to the drug or not.

Also, the investigators cannot wait until OM has developed in this trial, because B needs to be administered prior to, or very early in the radiation/chemo protocol (the MOA for B-OM is really more of a preventative effect, though it will likely also provide curative benefit too). So, we have to go after a specific tumor type, in a specific location, with a specific chemo/rad treatment protocol, all with the knowledge that these lead to a high probability of moderate-to-severe OM.

The question then becomes, how long does it take to identify such highly specific patients, not to mention recruiting them into a new investigational drug trial for something ancillary to their cancer treatment? In a best case scenario, here is my projection, If we assume the following:

1. the first 3 sites were announced in late May/early June (per press release)
2. the sites came online every 2 weeks (takes time for the CRO to do site initiation visits)
3. the next 2 sites start recruiting in mid/late Aug
4. each site can recruit 1 patient per week into the B-OM protocol (may be wishful thinking...particularly in places like South Dakota)

We may be close to the half way point in recruitment (n=34 by my calculation), and it may take another 6-8 weeks to complete recruiting. The last patient will require the 7 week treatment + 28 day follow-up. This takes us into mid/late Dec. Allow another 1-2 months for patient drop-out/hiccups, etc, and now we are looking at mid-Q1 '16 before we complete the protocol. Then allow 1-2 months for data lock, site close-out visits, and then top-line data analysis (recall this is a CRO doing all of this...CTIX does not have a clinical dev team cracking on this). If recruitment is slower (e.g. 1 patient every 2-3 weeks) then this drags out to the predicted completion data of June-'16. Again, only a projection IMO.

BTW, first time poster and I'm long (i.e. married to) CTIX