Investors in ZIOP got quite excited by the news which just came out last week about the company's currently running Phase I trial for Ad-RTS-hlL-12 + veledimex. The news was shared across numerous social media platforms where stocks are discussed and even on twitter.
Why were investors excited? The subject line of the press release said it all, citing "Favourable Interim Survival Results". That led some Ziopharm investors, many who post to stocks sites and twitter, to draw a cause and effect relationship. Ten out of the eleven patients in the trial were still alive, ergo the trial was the reason these individuals were surviving.
If only science was that simple.
The first posting I ever wrote on Ziopharm was back in March of 2015 on Seeking Alpha where I expressed the opinion that ZIOP represented a speculative bubble. That was when ZIOP was trading over $13 per share. In the comment section I was roasted. One thing in particular that I wrote served as a point of a attack for the haters. In that posting I freely admitted that I do not have a medical or scientific background.
You can read that posting here: http://seekingalpha.com/instablog/15663412-joe_retail/3802236-ziopharm-oncology-a-speculative-bubble-poised-to-collapse
Lacking an in depth knowledge of medicine I was deemed ill equipped to judge such a "transformational" company as Ziopharm.
In light of the excitement over this most recent "news" it appears that there are many investors in Ziopharm with even less of a scientific background than your's truly. After all, I have taken science courses in both high school and university, and I at least understand the reasoning and rationale behind what is generally referred to as the Scientific Method.
Obviously those drawing a cause and effect relationship between this trial and patient survival do not. I can just picture some scratching their heads right now saying: "The Scientific what"????
This is a single arm study, there is no control group nor is there a placebo group. This is so rudimentary, but reading the comments on sites like StockTwits, Twitter, Yahoo, InvestorsVillage and InvestorsHub, its strikes me that a lot of posters have no idea what control and placebo groups are or why they're important. That or they're being willfully obtuse in an effort to lure in the Homer Simpson types of the investment world by touting the survival results as "proof" the therapy works
Now to be clear, a phase I study doesn't need either a control or placebo arm because the goal isn't to prove something like a survival rate. The primary goal is to simply prove safety, or efficacy if you prefer the fancier term. Basically they need to ensure the treatment doesn't cause death or adverse events, like say a heart attack. Secondary to safety is to determine the maximum tolerated dosage as well as any immune and biologic responses...if any.
On the primary objective of this study it appears that, at least at this early stage, its mission accomplished so far. Ten of the eleven patients are still alive and there's no news about Adverse Events or AEs in those ten individuals. That is excellent news in of itself. Had there been a number of AEs and/or deaths, then they would likely have to go back to the drawing board.
As for the secondary objectives, particularly immune system response, we have a "suggestion".
CEO Dr. Cooper says in the PR that the data they have "suggests" the results are exciting. What that means exactly, I haven't the foggiest idea. I'm assuming it to mean that the results may be exciting, or that ultimately they may not, there isn't enough information to go on. If the data was definitively exciting why qualify it by using the word "suggests".
As for survival rate, that will be either proved or disproved in later trials, and those trials will be much more in depth, involving more than just 10 or 11 patients I have no doubt.
So why are control and placebo arms important? I can't believe I'm explaining this, but its obvious from the posting I see on social media sites that a lot of people don't understand it, especially at InvestorVillage which strikes me as ground zero for the pumper crowd.
I'll deal with a placebo arm first. The placebo effect is well known. People receiving a sugar pill or something similar with zero medicinal value have been shown to experience healing results for years across a variety of trials and experiments. The relationship between the mind and healing is an area that most people recognize. Having a positive mental attitude boosts the immune system, while being depressed weakens immune response. A patient just knowing they're receiving a treatment, that can help in achieving positive results, even if the treatment is something as useless as a sugar pill.
A control group on the other hand receives no treatment at all, not the drug therapy or a sugar pill (placebo). The results of a drug undergoing a trial with three arms can then be measured against two variables, against those who had no treatment at all, and against those receiving a placebo. If the drug arm doesn't outperform the placebo and control groups then you have a failed trial. If the drug arm outperforms the other two, then you may be able to move onto the next step. And if the performance is significant enough you may have something worthy of FDA or similar approval, ultimately a marketable drug or therapy.
Now to be as clear as possible, this is not easy stuff, not when you're dealing with a disease like cancer. There are so many variables that it can be at best difficult and at worst impossible to run a 3 arm study. To get the best and most scientifically responsible results, then each arm should be identical. Same ages, sexes, races and with the same diseases at the same stages. There can be differences in the way different racial groups respond to treatments based on varying genetic make ups. Some populations are more prone to certain conditions than others.
With a disease like gliablastoma, I can't see being able to get 3 identical groups with the exact same characteristics, the same tumor locations and sizes, with the same treatment histories and everything else.
Obviously with a two or three arm trial they would want to at least approximate each group, so that they are at least somewhat similar, but you're never going to get perfectly identical groups.
As noted in the PR, the results so far are incredibly early. Only 6 patients treated 6.2 months or longer, and only 10 in total. According to the clinicaltrials website this trial is not estimated to be fully completed until December 2018, over 2 years from now.
So why tout survival results from a trial not designed to measure that variable? I am of the opinion, that as a public company, Ziopharm and its principles were looking to perhaps support a falling share price. There are a lot of Homer Simpson types out there who will draw cause and effect conclusions when at best all that can reasonably concluded is a correlation.
For those still unsure of the difference between cause and effect and correlation, I'll leave it to Lisa Simpson to explain it to you.
One last note, as noted in earlier postings about Ziopharm I own put options so my opinions are not without bias.
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