Anthera Pharmaceuticals Inc (NASDAQ:ANTH) is heading into the close of the year more than 60% down on its early week market cap, on the release of some seemingly disappointing data from one of its lead studies. The data, relating to the efficacy and safety of a drug called Sollpura, failed to meet against a prespecified endpoint, and as a result, the trial has technically failed.
There’s a side note, however, and it’s an important one.
The data only very narrowly missed its goals, and there were some strong indications that it did so based on trial design limitations, and not on the back of Sollpura’s MOA. If this is the case (and Anthera is hoping to show that it is, with the carrying out of another phase III early next year), then the current decline may prove just a temporary blip on a longer term path to commercialization. In turn, it could present an opportunity to get into Anthera at a discount. It all relies on the company’s interpretation of the numbers, of course. With this noted, let’s take a look at that interpretation, and see if we can figure out the chances of it proving accurate.
So the drug is called Sollpura, and it’s going after a condition called exocrine pancreatic insufficiency, which is the inability to properly digest food due to a lack of digestive enzymes made by the pancreas. In humans, it’s pretty much always limited to patients with cystic fibrosis (although it can be associated with some other conditions as well). It’s the CF target Anthera is going after. In short, CF patients with EPI, trying to treat the EPI.
Current SOC in the space is porcine-derived, enterically-coated pancreatic enzyme replacement therapy (basically, taking lots and lots of pills that contain enzymes from pigs). It’s a huge dosing regimen to follow, goes against certain religious dietary requirements and can be completely unsuitable for certain patient groups – kids, patients that have trouble swallowing, that sort of thing.
Anthera’s Sollpura is in two forms – a powder designed for oral solution, and a pill that offers the full dose of a biotech derived ERT in just a few pills. Basically, all the problems associated with current SOC are overcome by one of these two forms of Sollpara. That is, of course, so long as the company can show it works.
There were two phase IIIs in place – one called SOLUTION and another called SIMPLICITY. Somewhat confusingly, the SOLUTION study is the one looking at the pill form, while SIMPLICITY is looking at the oral solution form. The one we’re interested in here is SOLUTION.
The trial had an endpoint of change in the Coefficient of Fat Absorption (CFA) non-inferiority margin. It was pitched against one of the current SOCs, Pancreaze. The study narrowly missed the CFA non-inferiority margin of what’s called the modified Intent to Treat (mITT) analysis; however, by additional pre-specified analyses of CFA (mITT-Baseline Observation Carried Forward and Per Protocol), the drug hit its target. Anthera thinks that the patients’ inability to up their doses quickly. Basically, there’s a limit on how much porcine derived enzyme we can tolerate. This limit shouldn’t technically apply to Sollpara (and there’s data to support this), yet it was applied for the purposes of the study.
For us, this is the key point.
The study hit on two different types of analysis, proving it does offer a viable solution for these patients – a solution that doesn’t come with all the issues associated with SOC. However, because of that narrow miss, the drug is (technically) inferior. This wouldn’t be enough to get it past the FDA, so in all likelihood, we’re going to see Anthera allow for an increased dosing in certain patients in a follow up trial. If this increased dosing boosts the data a little, and there’s every suggestion that it will, it should be enough to combine with the current numbers and seek approval.
Our bottom line on this is that we think there’s potential for a complete recovery here, based on the seemingly easily resolvable data miss. Couple that with the clear improvement in drug profile of Sollpara over current SOC (including Pancreaze) and we don’t think Anthera will struggle to push this one through to commercialization.
It just might take a little longer than expected.
