Here’s What The Portola Pharmaceuticals Announcement Really Means

Portola Pharmaceuticals Inc (NASDAQ:PTLA) is attracting a fair amount of attention in the biotechnology space as we head into the close of this week.

The company just put out an update on one of its lead development programs and has taken a hit on the development. Here’s a look at what happened and what comes next for the company.

So, the development in question relates to a drug called AndexXa, which Portola is developing for patients anticoagulated with an oral or injectable Factor Xa inhibitor who experience a serious uncontrolled or life-threatening bleeding event or who require urgent or emergency surgery.

A BLA for the asset was submitted back in August last year and the FDA, after a bit of back and forth, extended its review target on the drug from February 3, 2018, to May 4, 2018, with the latter now in place as a PDUFA date.

Well, supposedly.

On a conference call, however, yesterday, Portola management put this PDUFA in doubt. Here’s a snippet from the call, which is taken from a transcript provided and published at Seeking Alpha here:

“We also received a communication from the FDA this past Friday that included information we thought was important to share. FDA indicated in addition to the ANNEXA-4 study and the usual care cohort, what we call the UCC, they also asked for an additional data on some other patients in a randomized study. We are in the process of seeking clarity from the agent and will provide additional details as we learn more. In the meantime, we are moving forward with the review process and making commercial launch preparations.”

So, the uncertainty is rooted in the timing of this trial and – specifically – whether or not it’s going to take place before or after the PDUFA comes around. In other words, is the agency asking for a trial to shore up the application before it’s willing to issue an approval on the drug or is the trial just a post-marketing study to clarify administration questions.

If it’s the former, there’s a real concern as there’s essentially no way that Portola can complete a trial and submit the data, and then the FDA review the data, before the current, already extended, PDUFA comes around. This being the case, then, we’d almost certainly see another extension and, by proxy, another setback for the company on this program.

So, what comes next?

Well, as the snippet above highlights, Portola management now needs to sit down with the FDA and figure out exactly what the FDA expects when it said it wants another trial. Once this information is in place (and subsequently relayed to shareholders) it will be far easier to figure out the implications of the development from a time (and, of course, money) perspective going forward.

Right now, then, it’s just a case of sitting tight and waiting for additional information. This is a strong program and one that’s supported by some pretty solid (albeit not perfect) data, so even if an additional pre-approval trial is required, it doesn’t necessarily mean that the program is in jeopardy. Of course, and even with the just mentioned situation in place, shareholders would almost certainly rather the FDA’s request relate to a post-marketing trial so as to allow the company to get its asset on shelves in the US as soon as possible and to start generating sales revenues from it near term.

Right now, mid-session in the US on Friday, the company trades for a share price just shy of $31 a piece and a market cap (at this price) of $2.02 billion – down nearly 2% on the day’s open price.

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