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Ionis Pharmaceuticals, Inc. (IONS)
Morgan Stanley 15th Annual Global Healthcare Conference
September 13, 2017 02:10 PM ET
Beth Hougen - Chief Business Officer
Dr. Wade Walke - Corporate Communications
David Lebowitz - Morgan Stanley
Previous Statements by IONS
» Ionis Pharmaceuticals' (IONS) CEO Stanley Crooke on Q2 2017 Results - Earnings Call Transcript
» Ionis Pharmaceuticals' (IONS) CEO Stanley Crooke on Q1 2017 Results - Earnings Call Transcript
» Ionis Pharmaceuticals' (IONS) CEO Stan Crooke on Q4 2016 Results - Earnings Call Transcript
Now happy to have with me on stage from IONS’s Pharmaceuticals, Beth Hougen, the Chief Business Officer of IONS; and Dr. Wade Walke from Corporate Communications. Thank you very much for coming here today. It’s a different than last year when you're here because last year, you were assumed to be commercial and now you are commercial. So I guess, if you could start for us, tell us, where the Company is the whole, give a top level picture and then we’ll start drilling down.
Okay, thank you for having us. So, you’re right, a year ago we had just untwined our first of five positive Phase 3 studies, and that really started our transition to a commercial company. We’re now sitting here a year later having had the Phase 3, positive Phase 3 study. We are looking at SPINRAZA. It was a tremendous launch so far early day still, but very, very exciting. And we’ve got volanesorsen our Phase 3 drive for Akcea. That is now in registration in multiple reasons in the world U.S., Europe and Canada. I mean we would anticipate commercializing that through our subsidiary Akcea Therapeutics hopefully about this time next year.
And then inotersen, our Phase 3 drug and positive Phase 3 study in May. For that drug, we will go into registration in the U.S. and Europe, a little bit later this year, and that for TTR amyloidosis and we would anticipate being on the market with that drug probably about this time next year as well. So that really starts to cement our transition to a multiproduct profitable sustainably profitable company. And then behind that, we have a pipeline of 30 to 35 drugs that continues to grow with tremendous opportunities for commercial success embedded in that pipeline as well.
So just to make sure my pipeline charts are updated. How many drugs should be on the pipeline chart for IONS now?
I think that's about 38 or 39.
Dr. Wade Walke
37, I lose the track.
SPINRAZA, it's got launched very quickly after the approval in December. It certainly enjoyed the strong launch out of the gate with some very good quarter-over-quarter growth. Of course most recently the last quarter by Biogen had indicated some I guess challenges regarding some patients in SMA 2 population and natural history runs with it when they get the blue, so could you update us to what -- how should we view the SMA 2 population going forward? And what the challenges with administrating the drug in some of these patients mean?
So maybe start with just a little bit of history. SPINRAZA is on the market now in the U.S., in Europe. Recently approved in Japan, approved in Brazil and is really getting approval in pricing and reimbursement worldwide, so a tremendous opportunity for these patients without any other therapeutic option. In the Type 2 patients, the difficulty we're talking about is that some of these patients have had spinal surgeries possibly spinal fusion surgeries and then that could complicate the intrathecal injection that's required to dose SPINRAZA.
What we've found is that and Biogen has actually said, this was recently as a couple of days ago here at the conference that there are actually far fewer patients than the 60% of Type 2 patients who have had this spinal surgeries and given the efficacy of the drug and the strong demand by both patients and physicians, while it may take a little bit longer to out to find your work around and be able to dose SPINRAZA, they're finding ways to do it. It's really not a significant impediment to dosing the drug.
These patients really need this drug and they want it and we're finding that it's not only changing the way the SMA patients are cared forward in the standard of care, but it's also changing the way that the physicians are treating the symptoms of the disease, so that they can ensure that these patients can get access to SPINRAZA. So, that's for the prevalent population today, but if you think about the benefits of SPINRAZA could confer on patients.
What we know is that the earlier you dose these patients, the better their outcomes, and what we would hope is that you dose these Type 2 patients early on in either before symptoms or early on in their symptom onset. And you can avoid any of these types of final situations, and so they don't get the scoliosis. And therefore, you don't have that situation even to begin with. So, that's our view. We don't think there's any impact to t-cells and we're looking forward to continuing to ramp.
That actually brings us this next segway into the NURTURE data. NURTURE, pretty convincingly talked about the benefits of dosing earlier-and-earlier into the population and it connected to that is the need for screening at birth to try to ensure that they know about these patients before the disease actually starts physically manifesting. How is the -- I guess when we going to see the next data for NURTURE, number one? And next number two. How is that process of moving forward trying to find the way on a state-by-state basis to get early diagnosis just put into the new born children?