While the wider biotech space was enjoying something of a unexpected return to positive sentiment on Wednesday and Thursday, buoyed by the lifting of the potential investigations into pricing practices that came about on the back of a Donald Trump election win, Anthera Pharmaceuticals Inc (NASDAQ:ANTH) was not so fortunate.
The company put out data from a phase III trial of its lead development asset, Blisibimod, and things don’t look good for the drug’s prospects. Endpoints were missed, and management had very little to say as far as reassuring markets of forward potential is concerned, and the company lost more than 35% of its market capitalization on the release.
The question now, is what’s next? Here’s our take.
Before we get into what happened with the outcome, let’s take a look at the drug in question, and the trial that has – seemingly – spelled the end of its run in this indication.
The drug, as mentioned, is called Blisibimod, and it’s under investigation for the treatment of a condition called Systemic Lupus Erythmatosus (SLE). SLE is an autoimmune disorder that mainly impacts women, although some men do suffer from the condition (the ratio in the US is close to 9:1 in favor of women), with a peak incidence age of between 15-45. There are around 1.5 million sufferers right now in the US, and more than 5 million globally, and there’s no really effective cure for the condition available on the market. A few off label symptom treatments exist, and some targeted therapies, but nothing that really goes after and corrects the underlying cause.
SLE is caused by an over-proliferation of B cells (or at least, that’s the current theory). B cells, in healthy patients, mediate immune responses and have an impact ion certain inflammatory processes. Normally, B cells die through normal necrotic behavior (apoptosis, etc.) so as to avoid this over-proliferation. When these processes don’t work as they should, B cells become too high in number, and start to cause issues such as SLE. One of the major players against B cell necrosis is what’s called B-cell activating factor (BAFF). BAFF (for some reason, scientists aren’t 100% sure why) promotes B cell survival, and it’s this BAFF that Anthera was going after with Blisibimod.
The drug was billed as a selective antagonist of BAFF, which binds to the factor and, in doing so, essentially makes it redundant. Reduction in active BAFF should lead to a reduction in B cell survival, and a reduction in B cell survival means more B cells undergo the standard necrosis process. Less B cells, less response, and less SLE.
That’s the theory, at least.
Now, it doesn’t look like it holds much water.
The announcement just put out by the company revealed that, in the phase III in question, called CHABLIS-SC1, the drug failed to meet its primary endpoint based on what’s called the SLE Responder Index-6 (SRI-6), at 52 weeks. This is a pretty standard industry measurement of efficacy in SLE targets, and so the primary fell in line with what it would have needed to if it was to have a chance of gaining approval. Basically, Anthera needed to show that Blisibimod improved response by way of this scale compared to a placebo arm.
From a raw numbers perspective, it did. 47% of patients in the blisibimod arm versus 42% of patients in the placebo arm achieved the endpoint. Unfortunately for Anthera, however, and equally so for its shareholders, the difference was deemed not stat sig (we don’t have the p-value, but we’ll keep an eye out for it), and non stat sig means no hit.
So what’s next?
Well, things don’t look good. Often when a trial fails to meet an endpoint like this, management will play on the potential for a revival based on a deeper data analysis. Revivals don’t often happen, but at least management optimism serves up some level of reprieve to a knee jerk market response. Here, however, even management doesn’t seem optimistic. William Shanahan, M.D., Anthera’s Chief Medical Officer, basically just thanked the teams and the patients, and said they would make the data available for others in the “scientific community in the future which we believe will help to further inform the development of treatments for severe lupus.”
Bottom line, this one looks like a dead end. We’ll keep an eye on things to see if there’s anything that suggests our thesis might be premature, but it’s hard to see any reconciliation against a positive endpoint on this one, whatever the data reveals going forward.