Neurocrine Biosciences, Inc. (NASDAQ:NBIX) just announced that that the FDA has cancelled the Psychopharmacologic Drugs Advisory Committee meeting for INGREZZA. The meeting, which was originally scheduled to take place on February 16, 2017, will now be skipped, and the drug will carry straight through to the PDUFA date, scheduled for April 11, 2017.
This is a bit of an unusual move, and that’s why there’s a bit of indecision among markets as to how to respond. The company initially gained on the news, but is now pretty much flat, despite plenty of shares trading hands and a fair amount of volume coming to the fore. It’s rare, but it’s not unheard of, and it can be interpreted a few different ways.
We’ve seen advisory panel meetings cancelled because the FDA wants to issue a CRL, and is aware of this long before the PDUFA, so doesn’t want to waste the panel’s time. We’ve also seen it happen, however, where the FDA knows ahead of time that it wants to give a drug the green light, and gain, doesn’t feel it necessary to waste a panel’s time.
What’s the case here, then?
First, let’s look at the drug.
INGREZZA is seeking approval for the treatment of a condition called tardive dyskinesia. Dyskinesia is a disorder characterized by involuntary, repetitive body movements. With this particular type of the condition, and as the tardive element of the name suggests, the involuntary movements are slow in onset (or at least, slower than that standard definition of the condition). It’s really difficult to treat, with no real effective SOC in place right now, and it’s brought on by long term use of of antipsychotic drugs (or short term, high dose) or in children and infants as a side effect from usage of drugs for gastrointestinal disorders.
The drug is what’s called a VMAT2 inhibitor. VMAT2 is a protein that is concentrated primarily in the brain, and it is responsible for aiding and monitoring the movement of what are called monoamines – things like dopamine and serotonin – across neurons and synapses, which are the brain’s nerve cells.
When VMAT2 is inhibited, as Neurocrine Biosciences, Inc. (NASDAQ:NBIX) is attempting to do with INGREZZA, not as much monoamine is able to pass across the synapses in the brain, and this – theoretically – should have a degradatory impact on the involuntary movement associated with tardive dyskinesia.
So that’s the drug and how it works, but MOA is not going to be the major element of the FDA’s decision come PDUFA – it’s all about the data.
The data that the NDA is based on derived from two trials, one called KINECT2 and one called KINECT3, which were phase II and phase III studies respectively. Obviously, the phase III is going to factor into the agency’s decision a little more heavily than the phase II.
The endpoint was change in Abnormal Involuntary Movement Scale (AIMS) six weeks into the trial. AIMS is exactly as it sounds, and the company was looking for a reduction as indicative of efficacy. When the data hit, it demonstrated a reduction of 3.1 points more in the active group than was shown in the placebo group, and the p-value for the study proved beyond doubt that the study is significant.
So this brings us to our final take on the FDA’s decision to overlook the panel review and take this drug straight to PDUFA. When looked at objectively, there’s a large unmet need here, in a condition that doesn’t really have too many other treatments developing through another company’s pipeline (and definitely none as advanced in terms of time-to-commercialization as INGREZZA). In other words, if the FDA doesn’t give this one the thumbs up, then sufferers are going to have to wait a long time for an alternative.
The data was sound, and proved the drug safe, and we think that even a modest benefit is worthy of the agency giving INGREZZA a green light. With this noted, we expect that this news is positive for Neurocrine Biosciences, Inc. (NASDAQ:NBIX), and that the FDA is going to pass INGREZZA for commercialization come PDUFA on April 11. Of course, nothing is certain, and there may be something in the application we’re not privy to that has served as a red flag. Without knowing that for sure, though, we’re going with a positive bias.
Note: This article is written by Mark Collins and originally published at Market Exclusive.