The big news out of the biotechnology at on midweek comes from $10 billion biotech company Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY). The company announced on Wednesday that it had data in hand from one of its lead investigator programs and, shortly after the announcement, reported the data by way of a press release. The release came as part of a collaborative report with Sanofi SA (ADR) (NYSE:SNY), the company with which Alnylam was partnered on the program.
So what did we learn?
The study in question was called APOLLO and it was set up to investigate a drug called patisiran. The two companies, Alnylam and Sanofi, are looking to pick up a regulatory approval for patisiran for the treatment of a condition called ATTR amyloidosis, which comes about when a protein called transthyretin builds up in the form of amyloid deposits in various parts of the body and causes problems associated with (and rooted in) the buildup in question.
It’s hereditary and there are basically no really effective treatment options on the market right now. Some symptom related therapies are available and these can serve to alleviate the condition to a degree, but as far as a cure goes, there is nothing available yet.
And that’s what Alnylam and Sanofi are trying to remedy.
The primary endpoint for the phase III trial was the change from baseline in what is called the modified neuropathy impairment score, which is an industry standard severity market for this condition, at 18 months compared with placebo. As per the latest data, the drug was able to improve the score by +7 as compared to placebo with a p-value of less than 0.00001. As far as statistical significance goes, you don’t really get any better than that. Markets generally look for a P value of less than 0.05 as indicative of treatment effect so what this value is saying is that there is about as low chance as there could possibly be that this improvement is the result of something else other than active drug administration.
The trial also hit on all of its secondary endpoints, many of which related to key biomarkers as well as some quality-of-life factors, again essentially demonstrating beyond doubt that this drug can be a game changer for this group of patients.
As per the report, there were no major safety issues associated with drug and adverse events were limited to grades well within those expected ahead of trial initiation.
So what comes next?
Well, this was a pivotal trial and the drug hit on its primary and all secondary endpoints so, the natural next step is to submit a registration application to the US Food and Drug Administration (FDA) in an attempt to get this drug on the shelves. We haven’t got any specific date by which the company hopes to get the application submitted, but Alnylam reported alongside the positive data that it expects to submit the NDA at some point during late 2017, so we are taking this to mean mid to late final quarter.
And this wasn’t the only impact of this release when it hit press on Wednesday. A couple of other companies have a vested interest in this drug, or disinterest as the case may be, and both of these have moved accordingly post-release.
First up we’ve got Arbutus Biopharma Corp (NASDAQ:ABUS). Arbutus is entitled to receive royalties on sales of patisiran as and when it hits markets so, as might be expected, the company is picking up some collateral strength in the wake of this positive news. At the close on Wednesday, company was up more than 20% on its daily open price.
Second comes Ionis Pharmaceuticals Inc (NASDAQ:IONS). For Ionis, things haven’t worked out quite as well. The company is developing a drug called inotersen right now and it’s doing so in exactly the same indication for which patisiran just demonstrated it can be extremely effective. If Alnylam is able to get the market first (and it’s looking very much like it will be able to) then it should be able to snatch up a substantial market share before inotersen even gets a looking. Again, as expected, this one is moving in line with the implications of this data – Ionis closed the day down by 9% at $53.88 per share.