Cell Therapeutics (CTIC):

Cell Therapeutics never disappoints. Cell announced some more positive efficacy data on their drug Pixantrone. For those that aren’t familiar with Cell’s lead product I’ll go through a brief overview. Pixantrone is currently under the FDA’s standard review process for refractory non-Hodgkin’s Lymphoma. The drug was designed to be a possible replacement for the standard cancer therapy doxorubicin. You see, cancer patients can only take so much doxorubicin in their lifetimes before it damages their hearts. So researchers have long been trying to develop a drug that does not hurt patients hearts, but is equally as effective as doxorubicin, hence we have Pixantrone. Pixantrone was designed with the thought of having all of the efficacy traits of doxorubicin without having the cardiotoxicity problems associated with it. The data announced this morning updated data from their pivotal Phase III trial, so let’s take a look.

From the get go, the efficacy data looks pretty darn good, the Progression Free Survival data is strong at 5.6 months versus 2.6 months for the comparator arm. And the Complete response rate is especially strong at 25.7% versus 7.1%. Most of this should be expected however given the efficacy data that was originally released when the Phase III trial was finished. I am constantly asked if I have changed my estimate on a 50/50 approval possibility. And I have to say honestly that I still am 50/50 on approval for Pixantrone. If the drug was evaluated on the merits of efficacy alone then there would be no problems, but it doesn’t work that way. The FDA measures safety and efficacy arbitrarily and there is no gold standard when it comes to trade-offs in the eyes of the FDA’s Oncology Chief advisor Dr. Pazdur. I have personally seen this guy send many cancer drugs back to the starting line for miniscule data questions and safety reasons. And in the end he usually ends up approving the drug only after delaying the approval process by years, in fact, some suspect that this guy may have actually set cancer drug development back by years because of his overly tight standards. You see, when companies have to run another clinical trial to confirm data it is a costly expense that puts drug development dollars into running another redundant clinical trial. Some of the drugs that he has sent back for trials are: Dendreon’s Provenge and Genzyeme’s Clolar. Both drugs are obviously effective, yet the cancer chief consistently wants to double and triple check safety data because he had a relative that had cancer that had a hard time with treatments.

I’m gonna be practical here. I think Pixantrone will have to scrap with the FDA through the regulatory process. Why? Because there is still a minor cardiotoxicity issue with the drug. And the FDA is pretty unclear as to what the actual trade off for safety and efficacy is. Cell said in the press release that 8.8% of patients had a cardiotoxic event compared to only 4.5% of the comparator arm. Now granted this isn’t a huge change and some of us might think the trade-off for a more effective drug is worth it, but I can guarantee you one thing here, the FDA will most certainly question Cell Therapeutics over it. They probably will have a zillion questions lined up for Cell when they convene a panel meeting. And if they can’t answer all of the questions then there could be a problem. The other concern that I think the FDA will have is that the trial’s sample is smaller than originally expected. I know that Cell met with the FDA to reduce the trial’s patient size, but I worry that given the lingering Cardiotoxic event questions, the FDA’s cancer chief might not be able to make a desisive call and err on the side of playing it safe and asking for another trial to be performed. This is why the FDA has become a black box of sort, because while if I had cancer I might be willing to take the drug given the risks, the FDA might not think its risk/reward profile is beneficial enough for patients. So I will have to stay with my original 50/50 approval estimate because of my worries over the FDA’s cancer chief’s erratic approval calls, I mean this is the same guy that sent Dendreon’s Provenge back for another clincial trial even though the entire cancer community wanted the drug to get approved and the drug was strongly effective in its trials, that whole mess was a total blackeye for the FDA. So we have to be practical here and take that into account. In the meantime, I expect the FDA will at least set up a panel meeting to discuss the drug at which point we will be able to see all of the FDA’ briefing documents.

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