Chris Viehbacher
President and Chief Executive Officer at Biogen
Thank you very much, Chuck. Good morning, all. I think we released a very good set of results this morning, ahead of expectations, but of course, we're all too consciously aware that really what most of you are interested in is where is Biogen going? And to that end, we outlined five priorities that we believed we needed to achieve to put Biogen in a position to be able to grow again sustainably, and I think in the first nine months of the year, we've made an awful lot of progress, and indeed, I would say, the third quarter was a particularly busy quarter.
To remind you all what those priorities were, really was to focus our teams and our resources on new product launches, and that is a little easier said than done, we're a company that has a long heritage in the treatment of multiple sclerosis, and teams get very passionate about patient outcomes and working with physicians and to move them to new areas does require a concerted effort. The other thing we wanted to do is to stabilize and grow again, those existing products that still have market exclusivity for a significant period of time, notably VUMERITY and SPINRAZA.
The third thing was to really look at our cost base. Although, we had a relatively mature product portfolio, we had one of the higher opex to sales ratios among our peer group, and we needed to address that, but more than that, we needed to really reallocate our resources. Fourth was to really look at our research and development pipeline, particularly for the longer-term growth outlook. We have really taken a deep dive into research and development, looked at those products -- projects that perhaps no longer fulfilled their original target product profile, where the practice of medicine had changed, where the probability of success had changed, and we have terminated those programs so that we can focus on those assets that we think have the most promise, and I think we have a number of those, any one of which could actually meaningfully add to our longer-term growth.
And the final thing was, we said right at the outset, we were interested in external growth. We always knew that the LEQEMBI launch was going to be a gradual launch. We always knew that also even the Zuranolone launch was a non-conventional launch, and to de-risk that profile, we wanted to look at external growth, and of course, we have been able to do that. So as I look at where we are, in the third quarter we actually had LEQEMBI was the first anti-amyloid antibody to receive traditional approval for early Alzheimer's disease.
ZURZUVAE was a mixed bag, we got an important indication with postpartum depression, but of course we missed on the major depressive disorder. As expected, we received Schedule IV listing from the DEA, and we also had QALSODY approved for treating a genetic cause of ALS. This is not necessarily a product that is going to be of interest to many of you from the revenue potential, but scientifically this is a major milestone, and validating the biomarker neurofilament I think will enable so many researchers to find further treatments for ALS and perhaps other diseases.
As I noted, we closed the acquisition of Reata Pharmaceuticals and that gives us a whole new growth opportunity. SKYCLARYS is off to a very strong launch and we'll talk about that in a minute, but it also builds out our rare disease portfolio. As you know, we are trying to move into some adjacencies just given the risk of the neurological conditions that we have tended to focus on and rare diseases has been a logical place for us to go. Biogen has been very successful with the launch of SPINRAZA and we think we can do the same with SKYCLARYS.
And as I mentioned earlier, we had the Fit for Growth program and this wasn't just about cost reduction because we do want to reinvest some of that, but we really needed to simplify the organizational structure to empower the organization more and move -- more of the decision making closer to markets and customers. We have ended up taking an entire layer, enterprise-wide out of the organization and in some parts of the organization, even two layers of that. So we do think those cost savings will add meaningfully to our earnings per share as we look forward, but I'm also looking forward to a significant change culturally in how we allocate capital and the agility and the ability to take decisions in the organization.
Could I move to the next slide, please? So let's talk about LEQEMBI, a subject that I'm sure all of you are very interested in. We have always guided that this was going to be a gradual launch, and we know that partly from the ADUHELM experience, but also just from the fundamentals of what we're doing. This is a product that needs to be administered within a treatment process or care network, and those care networks did not exist at the time of the launch, so they have to be built and doing that requires actually quite a significant change to the work patterns within clinics, and while IDNs and clinics are working really hard to put these in place, it of course takes time.
And I think a terrific example of that is the announcement recently by the Cleveland Clinic, and we all know that the Cleveland Clinic is one of the most widely respected medical centers anywhere in the world and they recently announced that they had just infused their first patient with LEQEMBI months after the approval, and I think that just speaks to the complexity that we're dealing with and in a lot of ways, we not only are pioneering science, we're pioneering this commercial approach. So, of course, we have an aim of getting to 10,000 patients by the end of March. We're at 800 now. What gives us the confidence that we think we can get there?
Well, I think we have a number of green shoots here, signs of progress. The first is, as we look at our internal metrics of intent to treat and patient demand, we are seeing all of those things progress extremely nicely. The FDA not only provided traditional approval, but CMS actually moved very quickly. The day of traditional approval, as they promised, they actually have provided reimbursement, and the patient registry has so far, from what we hear from the market, been relatively easy to use. We had some confusion around the reimbursement of amyloid PET, and CMS has clarified that. Now, of course, it's going to take a little time for that to flow down through to the max, but I think that will also relieve some of the confusion out there.
I think one of the most interesting things is we've got 60% of the top 100 targeted IDNs now having P&T approval, and one of the things that really gives me a lot of inspiration is, usually these P&T committees meet twice a year, but a number of them actually have organized special meetings just for LEQEMBI and not waited until the next meeting and that says to me that there's a recognition of the importance of this treatment and being able to get patients on treatment. So where we can also go from here?
Remember, a year ago from here, there was still skepticism about whether reducing amyloid plaques would really have a benefit, and it wasn't really until the Clarity study was finally presented at CTAD last year that we really had, for the first time, clear, compelling evidence of the benefit of removing these amyloid plaques. And now, of course, we can go and say, all right, that's tremendous, but why is that so tremendous? Well, for years, we've been trying to develop antibodies, and those antibodies failed, and that's what gave rise to the skepticism, which were the right patients, which was the right antibody that was going to get the right amount of drug into the brain? And, LEQEMBI is really the first one to show that clear, compelling evidence that that has occurred.
Now, of course, we all want to get fancy, and that's where we're going, and we just had CTAD this year and think about what we've just done. We are generating more data to really demonstrate the benefit of this treatment. We've seen, for instance, that the subcutaneous treatment is going to work, that we have comparability with the infusion. And this means so much for the convenience of patients, but this is no mean task either, other doctors have tried to do this. How do you get enough drug through the muscle tissue and into the brain? That has been achieved and is a major milestone.
We've been looking at maintenance dosing. What happens when you've cleared the plaque? Does the plaque come back? Well, we have 24-month data now that shows a lot of benefit of staying on treatment. Then the question is still, who's the right patient? And data were shown with the early stage patients with low levels of tau, and those are fascinating data. We had 76% of those patients stable over the course of measurement. And, very intriguing and very interesting, we actually saw with 60% of those patients that we actually saw some clinical benefit as measured by the CDR Sum of Boxes. Completely unexpected, that generated an awful lot of discussion at CTAD.
So now, of course, we're also looking at executing on geographic expansion. We've had the recent approval of Japan, and I'm traveling to Japan early in the New Year to be with my friend and colleague, the CEO of Eisai, to launch the LEQEMBI in Japan. And of course, we've got global filings under review in the EU, China, and 10 other markets. So this is one where we're going to have to be patient, but all the signs are green at this moment, and for us internally, we see a launch that is on track. But as we've always said, there's no real analogs, and every month we learn something new.
If I could move to the next slide, please, Chuck. Now, let's talk about SKYCLARYS, something that is, much different. And as you know, we now have 1,180 start forms to date, with about 860 patients actually on drug. When we look at all of the known analogs, we're actually exceeding all of those, including SPINRAZA at the same point in time. Now, we have to be a little cautious because we all know that there were likely to have been a number of patients ready and waiting by physicians. And I think that was even more of the case because you may recall that the product was actually approved in the spring, but then delayed for a couple of months due to a technical and temporary challenge on supply, and so I think there was an anticipation.
Nonetheless, there is a very strong desire to see this product come, and we're actually seeing a lot of requests from countries around the world to make SKYCLARYS available, and that just speaks to, I think, the understanding that this is the very first treatment that has ever been approved for Friedreich's ataxia. This is an incredibly debilitating disease that affects so many young people right in the prime of their life, and so it's extremely important that they benefit from that.
We had about $43 million of sales in the third quarter. One of the things that we are now working on, and I think this is where Biogen can really add value, is, why is there 1,180 on start forms and 860 on drug? Well, there are a number of things, trying to get reimbursement, we need genetic tests, we need to measure your liver enzymes before you go on the product, and one of the differences from SPINRAZA is that they're not all in centers. They could be out there in primary care, physician care, but Biogen is well equipped to do that.
We are used to providing genetic tests. We don't worry about the reimbursement. We provide those. We have mobile labs so that we can help patients who are not near to major medical centers to get, for instance, the lab enzymes done, and also, we know how to pull through these start forms and navigate the difficult reimbursement situation. So I think, not only is there an advantage for Biogen in getting this important medicine to patients around the world, but I think even in the United States, we can actually make this more rapidly available to patients.
So with that, I'll turn it over to Priya.
