The primary goal of most phase 1 clinical trials is to prove safety, basically to show that a drug, therapy or treatment doesn't cause severe adverse reactions and/or death. With the news out of Ziopharm today (May 18th, 2016) it seems that goal is being met. They reported that their on going phase I trial of Ad-RTS-hIL-12 with Veledimex for patients with Glioblastoma has enrolled 11 patients with only 1 death.
The company cites the median follow up as being 6.2 months, which compares well given that this form of aggressive brain cancer typically comes with a prognosis of death in as little as 3 or as many as 7 months, depending on what treatments have already been administered according to the release.
It sounds good, and it is. This trial is, so far, meeting the primary goal of a phase 1 trial, its proving itself to be safe. But the data is more than a little thin and leaves lots of questions.
The first and most obvious question relates to the median figure of 6.2 months. Median, as anyone who's taken even a high school level course in statistics knows, represents the middle point. Given that this trial data represents 11 patients, this median figure means that 6 patients have been in the trial for 6.2 months and longer, and 5 have been in less than 6.2 months.
Is this important? I think it is, definitely. Of those 5 cases that have been in the trial less than 6.2 months...How long have they been enrolled? Is it a few weeks, a few days? The PR doesn't say.
Why not include that data, let the world know what the shortest and longest number of months in the trial is? Instead of just citing a median figure why not include more information so that shareholders and prospective investors can make a more informed decision.
Instead of just saying "Overall median follow-up for patients enrolled in the trial is 6.2 months with 10 of 11 still alive". Why not add...."with the range being from 1 month to 11 months"? Another significant point is the one death. At what month did it occur? Was it at 1 month, at 2....at 10?
I can't help but question why more data wasn't included. Its only 11 patients, why not give the time in for each? It wouldn't be hard, I'm sure the data is readily at hand.
Obviously there are only 6 patients who have been enrolled for 6.2 months or longer, that much is not in question. But presenting data on just 6 patients might seem almost desperate. Did they include patients only enrolled for a month or two to boost the number so that it would appear more meaningful and then only cite the median figure in an effort to spin the news? In my opinion that is a very legitimate question.
If you go to clinicaltrials.gov you can read about this trial:
If you check that site you will see that the estimated enrollment is 48 patients. So 11 is less than 25% of the estimated total, and the study start date is listed as June 2015. If they did start in June 2015, then they've been dosing about 1 patient per month on average. The estimated primary completion date is given as December of this year, that's for the primary outcome measure, safety.
That means they're going to have to start enrolling patients at a much faster pace if they want to have any deep and meaningful data by the end of the year.
That means they're going to have to start enrolling patients at a much faster pace if they want to have any deep and meaningful data by the end of the year.
The estimated date for completion of the full study is given as December 2018.
This is notable because Ziopharm, as everyone already knows, only projects having enough cash to maybe last to the end of 2017, and they fully disclose that they will be in need of raising more capital.
That has me thinking that this PR with very little deep or meaningful data was perhaps pushed out in an effort to prop up a falling share price. If another secondary is coming sometime next year, and I view that as a slam dunk, then the higher the PPS is, the less dilution that will be required. And with all the share based compensation it certainly seems logical to me that the powers that be within the company would want to shore up the falling share price.
Thoughts anyone? As I've disclosed previously I do own put contracts so my views are not without bias.
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