Tazemetostat Trial Pulled

 Yesterday (February 4) was World Cancer Day. It's a day to encourage research, prevention, and awareness. The link will give you more information about some of the events that took place around the world yesterday, and some that will continue to take place. I had planned to post something about it, but I've been so busy lately that I never got the chance. I had forgotten all about the day until I saw a notice from the Follicular Lymphoma Foundation last night.

It wouldn't have been much of a post anyway. I mostly would have wished you a good day and encouraged you to eat some ice cream. So I hope it really was a good day for everyone.

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Instead, I want to comment on a news item that I saw a couple of days ago.

It's a post from a pharma investing site called "Ipsen’s Tazemetostat Trial in Tough Lymphoma Group Withdrawn: What Investors Should Know." I've written recently about my feelings about investment and finance with regards to my disease. This is an example of the kind of different perspective that a financial site brings over a medical site. 

It reports on a clinical trial involving Tazemetostat that was withdrawn -- stopped before it was really started.

Tazemetostat is an EZH2 Inhibitor that was given accelerated approval by the FDA in 2020. EZH2 is short for "Enhancer of Zeste Homolog 2," an enzyme that is controlled by the EZH2 gene. The job of EZH2 is to keep tumors from growing, so when it's not doing its job, it needs to be inhibited -- stopped by Tazemetostat. The EZH2 abnormality involves about 20% of FL patients, though it can also be effective on patients that don't have that known mutation. 

But "accelerated approval" really means "approval for now." It is usually given based on data from a smaller trial, and must then go through a confirmatory trial -- a larger trial, with more patients, that can show the same results as the small trial. 

The official web page for Tazemetostat says that the confirmatory trial has not yet been completed.  

The clinical trial that was stopped was called "NCT06068881: A Study to Assess Efficacy and Safety of Oral Tazemetostat in Adult Participants With Relapsed/​Refractory Follicular Lymphoma That Does Not Have an "EZH2 Gain-of-function" Genetic Mutation (Mandolin)."

This is not the confirmatory trial. The title is clear that it is meant to study patients who do not have the EZH2 mutation and who have had previous treatment.

What I find so interesting is that there is so little information about this. And that's true of pretty much every failed clinical trial. It is very rare that a researcher or a company will be brave enough to discuss their failures, which is too bad, because we learn so much from failure. (I'm sure the researchers learned a lot -- they just aren't sharing it with us.)

And failure is certainly common. Of all of the potential cancer treatments that start the process of getting approval (lab tests, then animal tests, them stage 1, stage 2, and stage 3 clinical trials on people), less than 10% ultimately get approved. 

As the article says, there was no announcement about this, just some changes to the official clinical trial page that indicated it had been stopped. Most clearly, there is a red box that says "Withdrawn" where there would normally be a green one that says "Recruiting." It also says "Never Started."

There are many reasons why this could have been the case. It's possible that some issues with Tazemetostat have come up that we don't know about yet. I'm not saying that's true, just that it's a reason -- but it does seem unlikely, since the trial was supposed to have started last September. It's also possible that funding was pulled and there was just not enough money to complete the trial in the way they wanted. It's also possible that they just had trouble getting participants (there is no information about locations for the study) -- with so many other exciting possible treatments, maybe there just weren't enough folks for this one. 

My big point here is that this is an example of how much we don't know as patients. There is so much to be excited about, especially around ASH and ASCO, as early research gets discussed. And much of that just never finds success. No one has to tell us about failures, even though I wish they would. 

The other pint is, sometimes those folks who look at this from a financial perspective can give us something good. I can imagine someone who is invested in the company that makes Tazemetostat, looking a couple of times a week at the clinical trials site, trying to get some insight as to whether it's a good investment. their financial incentive makes it worth talking about, even if someone with a medical interest doesn't pay any attention to it.

Perhas we'll get some updated information about Tazemetostat's confirmatory trial soon. In the meantime, the good news is, we have lots of options, and lots of reasons to be hopeful, even when the failures are invisible (or perhaps because they are invisible).