Neurocrine Biosciences Inc. (NBIX)
Phase II Results of VMAT2 Inhibitor NBI-98854 for Treatment of Tardive Dyskinesia Call
March 27, 2012 08:00 am ET
Kevin Gorman - President & CEO
Jane Sorenson - IR
Chris O'Brien - CMO
Thomas Wei - Jefferies
Robyn Karnauskas - Deutsche Bank
Phil Nadeau - Cowen & Company
Jon LeCroy - MKM
Joshua Schimmer - Leerink Swann
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Good day everyone and welcome to this Neurocrine announces Phase II Results of the VMAT2 Inhibitor call. At this time all sites are on a listen-only mode (Operator Instructions). As a reminder today's call may be recorded and it's now my pleasure to introduce our first speaker, President and CEO, Mr. Kevin Gorman. Please go ahead sir.
Thank you very much and thank everyone for joining us this morning. I'm joined here with Chris O'Brien, our Chief Medical Officer and Tim Coughlin, our CFO. Before we get started, I would like Jane Sorenson to read our Safe Harbor statement please.
Good morning. I want to remind you of Neurocrine’s Safe Harbor precautions. Certain statements made in the course of this conference call that state the company's or management's intentions, hopes, believes, expectations or predictions of the future are forward-looking statements which are subject to risks and uncertainties. Information concerning factors that could cause the actual results to differ materially from those contained in or implied by the forward-looking statements is contained in the company's SEC filings, including but not limited to the company's annual report on Form 10-K and quarterly reports on Form 10-Q.
Copies of these filings may be obtained by visiting the Investor Relations page on the company's website at www.neurocrine.com. Any forward-looking statements are made only as of today's date and we undertake no obligation to update these forward-looking statements to reflect subsequent events or circumstances. Kevin?
Thank you, Jane. So you have had a chance to see the press release from last night on our VMAT2 trial. The dataset was not as clean as we would have expected and we are compelled to perform an ad hoc analysis, but it's in the ad hoc analysis that the drug displayed substantial efficacy. We are going to spend the majority of this call with your questions. We'll start out with Chris giving you a brief overview. One last thing before Chris goes through the trial with you is that you may have also seen this morning that we have another press release. We are very pleased to see that Abbott has received just minor comments from the FDA on their SPA for elagolix. They have already responded to the agency and expect to receive full approval on the SPA shortly, all aspects of the starting of this trial, Abbott is going full speed ahead and we expect this trial as Abbott has directed us to start next quarter. So without any more delay, Chris as I said is going to give a brief overview of our VMAT2 trial and then we would like to take your questions. Chris?
Thanks very much, Kevin and good morning. Thanks to those for joining us. If you are having difficulty of tracking through the webex or you need to refer to the slides that I am going to be using, they are also available on our website posted this morning. So we will walk through those. So let's start by moving to slide number three which is entitled The Tardive Dyskinesia Study, but I wanted to remind people of the intent, the purpose of the design of this early Phase II study. If you recall, when we started, the study kicked off last fall, the initiative was to see if we could get proof of concept very early in this drug discovery program.
At the time, when we had two weeks of tox data and [APIM] solution. So we designed a small cross-over trial that would allow us to detect a 3 point difference in patient score when they were either in the placebo period of the cross-over trial or the active period of the cross-over trial. So in this case, individual patients are used as their own control or so called within subject comparison and with that in mind, what we know about the AIMS, we knew that the sample size estimate would be approximately 8 subjects in each arm of the study for a total of approximately 32 people for randomization.
As you will hear, we go through the data today there were 37 subjects that actually qualify for randomization and of that, the intention to treat or ITT population was a total of 32 subjects. ITT in this case defined as subjects who had gotten drug and who had data from both variants, period one and period two. Subjects were randomized either a 12.5 mg or 50 mg and they were also randomized into the sequence of exposure. So they could have gotten placebo first and then active or vice versa.
So four groups of eight turns out that we ended up having an intent to treat population of 32 and we will walk through the numbers with them as it stands. The schematic on slide four since we showed you, use that again. There are two treatment periods and the important thing is that the primary endpoint of the study was a comparison of the scores at the end of one treatment period to the scores at the end of another treatment period. So active versus placebo. Again powered to detect a three-point difference and as you will hear patients came into the study with baseline scores in the 14 to 15 range on the AIMS with a standard deviation of just under four points.