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Ionis Pharmaceuticals, Inc. (IONS 1.61%)
Q1Â 2019 Earnings Call
May. 9, 2019, 11:30 a.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Good morning and welcome to the Ionis Pharmaceuticals Q1 2019 Financial Results Conference Call. As a reminder, this call is being recorded.
At this time, I would like to turn the call over to Wade Walke, Vice President, Investor Relations to lead off the call. Please begin.
Wade Walke -- Vice President, Investor Relations
Thank you, Gary. Before we begin, I encourage everyone to go to the Investors section of the Ionis website to find the press release and related financial tables, including a reconciliation of the GAAP to non-GAAP financial measures that we will discuss today.
We believe non-GAAP financial results better represent the economics of our business and how we manage our business. We've also posted slides on our website that accompany our discussion today. With me on the call, are Stan Crooke, Chairman of the Board and Chief Executive Officer; Beth Hougen, Chief Financial Officer; Brett Monia, Chief Operating Officer and joining us for Q&A will be Damien McDevitt, Chief Business Officer and Frank Bennett, Senior Vice President of Research.
I would like to draw your attention to slide three, which contains our forward-looking language statement. We will begin -- we will be making forward-looking statements which are based on our current expectations and lease. These statements are subject to certain risks and uncertainties and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional detail.
And with that, I'll turn the call over to, Stan.
Stanley Crooke -- Chief Executive Officer
Thanks Wade, and good morning everyone and thank you for joining us on today's call. We've had a busy week of various calls, so thanks. You may have noticed that this week we unveiled our new brand. We think it better expresses our commitment to patients and the passion that we have for the science that creates life-transforming medicine. The picks and we forces our culture, our technology, our business model and our position as a (inaudible) for life.
So then I'll turn to Q1. We are off to a strong start building on the momentum from last year. Our first quarter financial results significantly outperformed the first quarter of 2018, putting us well on track to achieve or perhaps more likely potentially exceed our 2019 financial guidance. Importantly, we achieved these strong financial results while investing aggressively and broadly in our business, including commercializing two medicines through our full year of next year and advancing our pipeline in technology. It's been rather blockbuster performance that are continued in the first quarter and continue to significantly contribute to our financial results.
SPINRAZA that has glazed new ground and is the established standard of care in the treatment of previously failed and untreatable disease SMA. Even today, two plus years after marketing Biogen is continuing to generate new results and gain further understanding of SPINRAZA's benefit in patients with all forms of SMA.
Recent clinical experience continues to demonstrate durability of benefit in patients with SMA with some of these patients having been treated for over six years. More than that these results demonstrate the benefit is not only durable but it's also increasing over time with the longer treatment resulting in even better benefit. So in sum, all of the information that we have about SPINRAZA leads us here, that is that if we treat infants before they become symptomatic most of those infants seem to develop quite normal, healthy babies. So, the earlier we treat the better and the longer we treat the better.
In addition, we are working with Biogen on follow-on medicine for SMA with the potential to offer the same exceptional benefit as SPINRAZA with less frequent dosing. We anticipate moving this new medicine into development soon. Having completed the first full quarter of the TEGSEDI launch, we're encouraged by the initial sales and the feedback that we're getting from physicians and patients. Payers in the US have been receptive to TEGSEDI and we are also seeing good progress in expanding access in the --WAYLIVRAis now our third and the such medicine approved just over two years.
We at IONIS are very pleased that the EU recognizes the value (inaudible) patients with FCS and we look forward to making this new medicine available to SES patients who desperately needed. Our pipeline of Phase III and near-Phase III programs also achieved significant value-driving milestones in this quarter. Our medicines targeting SOD1 ALS and Huntington's disease have entered Phase III development and new data from ongoing clinical trials on these medicines continue to support the potential of these medicines to improve the lives of patients with these fatal and debilitating diseases.
The next wave of medicines advancing toward Phase III this year include our likely for (inaudible) LPA driven cardiovascular disease and are like to program for TTR amyloidosis. These programs are examples of the advances in our technology that have enhanced the performance of medicines in the clinic and enabled us to broaden our pipeline to address not only rare diseases but many of the common diseases that affect millions of patients. Including these four medicines, we have at least 10 more medicines that have potential to enter Phase III trials by the end of next year and of course that's a very exciting agenda for us and those patients.
Now I'll turn the call over to Beth to provide us the financial update.
Elizabeth Hougen -- Chief Financial Officer
Thank you, Stan. In the first quarter of this year, we significantly outperformed the first quarter of last year. Our total revenues more than doubled to nearly $300 million resulting in operating and net income on both the GAAP and non-GAAP basis. Our non-GAAP operating and net income in the first quarter were $167 million and $126 million respectively. In addition, we further strengthened our balance sheet by ending the first quarter with $2.3 billion of cash. Given our strong financial results, we are optimistic that we are on track to achieve and possibly exceed our guidance of non-GAAP net income. And as we gain more clarity on the rest of the year, we may update our guidance as we have in previous years.
With multiple medicines on the market, our commercial revenue continues to grow. In the first quarter, our commercial revenues were $68 million compared to $42 million last year. Our increased commercial revenue was driven by substantial SPINRAZA royalties and the contribution of TEGSEDI product sales. We are encouraged by SPINRAZA's better than expected global sales of $518 million in the first quarter. Global sales increased by 10% compared to the fourth quarter and 42% compared to last year.
We earned commercial revenue from SPINRAZA royalties of $60 million, up 46% increase compared to last year. As a reminder, while our SPINRAZA royalty rate resets at the end of each year because of Spinraza's strong first quarter performance, we have nearly reached the highest year. The worldwide sales growth was driven by multiple factors including adolescent and adult SMA patients initiating treatment in the US and increased penetration in major geographies outside the US.
At the end of the first quarter, there were over 7,500 patients on SPINRAZA treatment around the globe, including more than 7,000 commercial patients from over 40 countries as well as patients from the SPINRAZA expanded access program and clinical trial.
Biogen believes the global SMA patient population is significantly larger than initial estimates, which supports their expectation of continued growth from patient uptake in new market. In the US, continued penetration among it's adolescent and adult SMA patients is expected to be a significant driver of growth. The TEGSEDI launch continues to show strength with $7 million in sales in the first quarter and $9 million since the launch began late last year. In the US, physicians and patients are motivated by the potential benefit of TEGSEDI treatment. Patients on TEGSEDI are primarily being treated by cardiologists, neurologists and hematologists. AKCEA is seeing good progress with naive patients starting TEGSEDI treatments. Patients are also transitioning to commercial drug from the early access program and open label extension study. Notably, we are seeing patients switching to TEGSEDI from both competitive medicines in the US and EU.
We and AKCEA committed to expanding access to TEGSEDI for hATTR patients around the world. Last month, the first reimbursement process in the EU was completed with mice issuing a positive recommendation for TEGSEDI in England. Reimbursement discussions are also ongoing in other EU countries.In addition, PTC Therapeutics is continuing to work with Brazilian regulators toward approval.
With the recent approval of WAYLIVRA, AKCEA is preparing to launch in the EU with Germany, expected to come online next quarter. We anticipate the launch of WAYLIVRA to rollout across the EU and a similar sequence to TEGSEDI with additional EU countries launching next year. And in Latin America, PTC is laying the groundwork to deliver WAYLIVRA to patients there. In recognition of the EU approval of WAYLIVRA, we earned a $6 million milestone payment from PTC.
R&D revenue continues to be a significant and sustainable source of revenue for us. It is a particularly important element of our financial strength because it funds of large portion of our non-GAAP R&D expenses. In the first quarter, we are in $229 million in R&D revenue from our successful collaboration which accounted for 77% of our total revenue.
Our significant R&D revenue in the first quarter was driven in large part by the license fee we earned from Novartis. For the full year, we expect the mix of commercial and R&D revenues to be more balanced. In the first quarter, we earned $36 million from amortization of upfront payments and milestone payments we earned from our partners reflects the value we create as our medicines advance. For example, we earned $35 million milestone payment from Roche in the first quarter when they advance IONIS-HTTRx into Phase III development for Huntington's disease.
License fees are additional significant element of our R&D revenue as demonstrated by the $150 million we earned from Novartis when they licensed AKCEA-APO(a)-LRx.
We also earned license fees when companies license our technology to their partners. For example, last month Alnylam announced a partnership in which they licensed our technology to Regeneron. Once this transaction closes, we expect to earn $20 million from Alnylam.
Our non-GAAP operating expenses in the first quarter increased slightly to $130 million compared to the same quarter last year. This increase was primarily due to our investment in commercializing TEGSEDI globally. The fact that we can prepare to commercialize TEGSEDI and advance our pipeline of over 40 medicines with such a modest increase in expenses clearly sets us apart from our peers.
We also recognized $31 million of income tax expense during the quarter. The increase in our income tax expense was primarily due to our expectation that we will generate US federal and state taxable income this year. Our income tax expense has two components. The first component relates to our federal taxes, we expect to use our deferred tax assets to fully offset our US federal taxable income and therefore we don't anticipate paying our federal income taxes with cash.
The other component of our expense relates to the estimated taxes we will pay in cash for our state income taxes. Although we are recording the expense this year, we will not have to make the majority of the payment for this liability until the first quarter of 2020. Our financial results in the first quarter put us on track to achieve and possibly exceed our 2019 financial guidance. In large part because of the payment we earned from Novartis in the first quarter, we have already achieved approximately 40% of our full year revenue guidance of more than $725 million.
We are also on track to meet and possibly exceed our guidance of non-GAAP operating income of more than $100 million and to be profitable on the bottom line, on a non-GAAP basis. We continue to project that we will have approximately $2 billion in cash at year-end. We have accomplished these strong results by continuing to successfully leverage our efficient platform technology and our partners resources to advance both partnered and Ionis owned medicines and looking to the remainder of this year and into the future, we have confidence that we can continue to deliver sustainable financial growth while maintaining our focus on ever increasing innovation to drive value to patients in need.
And with that, I'll turn the call over to Brett to provide an update on our pipeline.
Brett Monia -- Chief Operating Officer
Thanks, Beth. As you can see from this slide, 2019 is already off to a strong start with numerous successes and important value driving achievements across the business. Just this week, we and AKCEA were extremely pleased to announce European approval of WAYLIVRA to treat people with genetically confirmed FCS who are at high risk for pancreatitis.
Because TEGSEDI and WAYLIVRA share much of the same infrastructure in Europe, preparations to launch WAYLIVRA are well under way and in Germany, AKCEA's commercial team is already in place. And in the US and Canada, our discussions with regulators continue as we pursue a path forward to expand WAYLIVRA access to FCS patients worldwide.
Turning attention to our neurology programs. We believe antisense technology is the leading drug discovery platform to treat a range of devastating neurological diseases, as such the AAM conference is an important opportunity every year for us and our partners to highlight the progress of our neurological disease programs.
This year, Biogen presented data from ongoing studies of SPINRAZA and the Phase I-II study of IONIS-SOD1Rx, which we now refer to as tofersen. In addition, Roche presented data for IONIS-HTTRx in Huntington's disease and AKCEA represented data from the NEURO-TTR open-label extension study for TEGSEDI.
SPINRAZA data from the SHINE, open label extension study and the ongoing nurture steady continue to show increased benefit for longer SPINRAZA treatment with no change to the established favorable safety profile reinforcing SPINRAZA place as the trusted choice in the treatment of all patients with SMA.
In the SHINE open label extension study in children and adolescents SMA patients, long term SPINRAZA treatment continues to demonstrate durability of benefit with some estimate patients now treated for up to six years and the benefit is not only durable but it also increases over time with longer treatment resulting in greater benefit. Additionally, these data further reinforced SPINRAZA's established and highly favorable safety profile. In the nurture study, we continue to see that when SPINRAZA treatment is initiated in pre-symptomatic infants, most patients are developing and achieving motor milestones like their healthy counterparts.
Switching gears, data presented by Biogen from the Phase I-II study of Tofersen in patients with SOD1-ALS demonstrated market reductions of in SOD1 protein which were associated with benefit in measures of ALS disease progression after only three months of treatment.
Based on these data, along with the positive safety profile, Biogen has initiated a Phase III study by adding a new patient cohort to the existing study protocol. This study could potentially support in accelerated path to regulatory approvals.
Moving onto our Huntington's program. As a reminder, data from the Phase I-II study of IONIS-HTTRx in patients with Huntington's disease, demonstrated for the first time substantial reductions in mutant Huntingtin protein, the known cause of the disease. Results from this study were published this week in the New England Journal of Medicine, underscoring the medical community's excitement for IONIS-HTTRx and its potential to treat patients with this devastating disease.
We and Roche are encouraged by the performance of IONIS-HTTRx, today in the open label extension study. Data from this study presented at AAN demonstrate how long-term treatment produces sustained reductions in mutant Huntingtin protein and these reductions are maintained with bimonthly dosing. A nine month data from this study provide support for Roche's decision to change the Phase III study protocol to explore less frequent dosing regimens.
We believe this change will greatly simplify the operation of the Phase III study as less frequent dosing is more manageable and desirable for patients and physicians. Although this introduces a slight delay in the study initially, by easing the burden on study sites in patients with less frequent dosing visits, we don't expect this the way it extend the study and may even accelerate time to completion.
In total, Roche's established a comprehensive clinical program that provides the potential for a rapid path for patients. We look forward to additional data from the OLE study later this year or early next year. Also at AEN, data were presented from the NEURO TTR open label extension study of TEGSEDI. These data demonstrated long-term efficacy in HTTR patients with polyneuropathy. Patients who initiated TEGSEDI treatment during the blinded portion of the Phase III NEURO TTR study, before entering the OLE demonstrated long-term benefit in both neurological disease progression and measures of quality of life.
Moreover, patients treated with placebo in the NEURO TTR study who then went on to receive TEGSEDI in the OLE achieved rapid and sustained benefit as well. And importantly, safety and tolerability continued to be easily manageable with no new safety concerns identified in the study.
We also have two LICA medicines advancing toward Phase III studies this year AKCEA-APO(a)-LRx for treatment of millions of people with LPA driven cardiovascular disease worldwide, is our most advanced like a program. With our partner Novartis, AKCEA-APO(a)-LRx is advancing toward the Phase III cardiovascular outcome study later this year with patients expected to begin enrolling early next year.
We're pleased with the progress Novartis has already made including opening (ph) epidemiology study to more precisely estimate the prevalence of patients around the world LPA treating cardiovascular disease. Importantly Novartis commitments this program including their decision to license Akcea APO(a)-LRx is further validation of our game changing LICA technology to treat very large patient populations. We and AKCEA are also making excellent progress in advancing Akcea TTR LRx , our like to follow medicine to treat people with all forms of TTR amyloidosis. The Phase I-II study of AKCEA TTR LRx is proceeding well and we'll be ramping up shortly.
And in the second half of this year, we plan on presenting results from this Phase I-II study and to initiate the Phase III study in patients with polyneuropathy due to their hereditary TTR amyloidosis followed soon after by a Phase III study in patients with wild-type and hereditary GTR cardiomyopathy. In addition, our mid-stage pipeline continues to perform very well, setting us up nicely that potentially have at least 10 medicines in Phase III studies by the end of next year.
Looking to near-term upcoming catalysts, in the coming months, we expect AstraZeneca to complete a Phase II study of danvatirsen, our Generation 2.5 antisense therapy targeting STAT3. AstraZeneca recently stated that they anticipate advancing danvatirsen into pivotal studies. Additionally, we completed enrollment in a Phase II study of IONIS-FactorXIRx in patients with end-stage renal disease late last year and we're looking forward to data from the study around the middle of this year.
IONIS-FactorXIRx is the first anti-thrombotic medicines that selectively targets the intrinsic coagulation pathway as a novel strategy to achieve reduce clotting without the risk of increased bleeding. As a reminder, data from our Phase II study in approximately 300 patients undergoing total knee replacement, published in the New England Journal of Medicine demonstrates the IONIS-FactorXIRx is the first anti-thrombotic agents in development to demonstrate robust reductions in Factor XI activity and their thrombotic activity superior to the standard of care, low molecular weight heparin without significant risk of bleeding.
We believe our (inaudible) medicines targeting to Factor XI have the potential to be used broadly in patients at risk for thrombosis, especially patient populations with additional risks for bleeding. We also look forward to sharing data from the Phase 1 study of the LICA follow on IONIS-FactorXIRx around mid-year.
Following these studies, fire (ph) and advance one are both of these programs further into development. And later this year we plan to have data from additional clinical programs including HBV and acromegaly as well as from our first generation 2.5 LICA program. Also, Biogen recently selected a new target for a fatal neurological disease identified under our broad 2018 research collaboration.
This program is another excellent example of the productivity of our collaboration with Biogen, which we established to fully exploit our technology to identify even more innovative antisense medicines for neurological diseases. We look forward to this program and other novel medicines moving into development in the future.
And now I'll turn the call back over to, Stan to close this portion of the call.
Stanley Crooke -- Chief Executive Officer
Thanks, Brett. This year we are celebrating our 30th anniversary. I'm proud of the value that we have delivered to patients and the shareholders, I'm also proud of the way we've done. And looking to the coming years, we are confident that we have many opportunities for value creation that far exceeds the substantial value that we have delivered thus far. We intend to continue to build value for patients and shareholders as we continue to deliver revenue and earnings growth and advance, broaden and expand our mature pipeline and continue to advance our technology platform. we are particularly excited that in addition to advancing, one of the largest rare disease pipelines in the industry, we also have a substantial pipeline of medicines in development focused on bringing benefits to patients with more common health issues, AKCEA-APO(a)-LRx for LPA driven cardiovascular disease, and Ionis Factor XII LRx for chronic disorders are excellent examples of the potential transformative medicines addressing more common diseases that are advancing in our pipeline. Unlike others, who follow, we are creating substantial value by being leaders.
We have led and we continue to lead in advancing RNA targeted drug discovery. We have led and we continue to lead in creating maximum value for patients by addressing novel molecular targets that have potential to revolutionize the treatment of many disease. We are leading in focusing on maintaining our long-term innovation and identifying the optimal organization to commercialize each of the medicines in our pipeline, treating each medicine as the precious asset that we have, because of our business model, today, we are substantially profitable and sustainably profitable while launching two new medicines advancing a pipeline of more than 40 novel medicines and continuing to advance our technology.
We are committed to continuing to lead the industry in innovation. In aggregate, these attributes separate us very clearly from our peers and are the foundation of our growing competitive advantage.
With that, I'd like to turn the call over for Q&A. So, Gary, if you could set us up please?
Questions and Answers:
Operator
We will now begin the question-and-answer session.(Operator Instructions)The first question comes from Chad Messer with Needham & Company. Please go ahead.
Chad Messer -- Needham & Company -- Analyst
Great, thanks for taking my question. One thing we learned at AAN just sort of hanging around the posters and talking to people, it's the idea I guess the hypothesis that SOD1 reduction would be beneficial perhaps even in patients that do not have SOD1 driven disease. Just wondering what your guys thoughts on that are? And whether it's possible in the future if we saw that pursued clinically by Roche?
Stanley Crooke -- Chief Executive Officer
We agree with the notion. There are a variety of lines of evidence that suggests that lowering SOD1 could bring benefit in patients who have other forms of ALS and I would reserve the response to the second question, Chad for our partners at Biogen.
Chad Messer -- Needham & Company -- Analyst
Thanks.
Stanley Crooke -- Chief Executive Officer
Did I say Biogen?
Chad Messer -- Needham & Company -- Analyst
I may have spoken earlier. Sorry.
Stanley Crooke -- Chief Executive Officer
.Thanks.
Operator
The next question comes from Paul Matteis with Stifel. Please go ahead.
Ben Burnett -- Stifel -- Analyst
Thanks very much. This is Ben Burnett on for Paul. Just two quick ones. The first one on the TTR LICA program. Is there anything you can disclose I guess at this time with regards to the cardiomyopathy study that you're planning and I guess have you made a decision internally about a comparator arm and could this include tafamidis?
Brett Monia -- Chief Operating Officer
Sure Ben, this is Brent. Thanks for the question. So as I mentioned, as we mentioned in the earnings call just now we're wrapping up the Phase i study in the -- our LICA follow on is looking great and we will present some of that data second half of this year and we're putting the final touches on the Phase III designs, not just for cardiomyopathy but also for polyneuropathy and our discussions with regulators has gone very, very well. We are very pleased with how those discussions have proceeded.
There's two things to think about when designing clinical endpoints, as you know; one is what it takes to get a drug approved and then second, what it takes to get it to drug successfully commercialized. And that's an important balance. Tafamidis has set a significant bar and cardiologists like to see outcomes. They like to see hard survival improvements. And we think that will be a very important component to the design of our Phase III study. We think it should be and we think it needs to be. Whether or not, how the families plays into that is to be determined. We're very pleased that the tafamidis is approved, biotech cardiac patients need a drug. There is nothing out there for them and we put Pfizer in bringing that to the finish line.
But whether or not when we would do a head-to-head against tafamidis or some sort of combination with it or those sorts of things. We're just putting the final touches on that and those discussions are ongoing with Akcea and with regulators.
Stanley Crooke -- Chief Executive Officer
So then it won't be too long before we are able to disclose with some position with what that study will look like.
Chad Messer -- Needham & Company -- Analyst
Okay, understood. That's great color. I guess just one other quick one on the acromegaly. You mentioned you have a Phase II readout coming up. I guess I was just wondering if you could provide a little color on this. And I guess what would constitute success here and I don't know if you said it, but is this top line expected this year? Thank you very much.
Stanley Crooke -- Chief Executive Officer
The answer to the second question is, yes. We are excited about the growth hormone program. (inaudible) is a LICA form of the growth hormone drug and while there are agents that are used in patients with acromegaly, they have very substantial limitations. And based on fairly thorough examination of -- in animal models of various types, we believe that our growth hormones drug could have significant advantages both in side effects and efficacy compared to existing agents and that study is designed to give us initial guidance about whether our expectations are going to be achieved. So it's an important study as it rolled quite a bit faster that we expected, blind to safety, tolerability are pristine like the rest of our LICA drugs. So we're looking forward to sharing the data with you when we are looking forward to having exciting results.
Ben Burnett -- Stifel -- Analyst
Okay, great. Thank you.
Stanley Crooke -- Chief Executive Officer
You bet.
Operator
The next question comes from Mani Foroohar with SVP Leerink. Please go ahead.
Rick -- SVP Leerink -- Analyst
Hi, this is Rick on for Mani. Thanks for taking our questions. And congrats on all the data updates at AAN. I had a couple of questions about SOD1 ALS. My first is today, how common is the genetic screening in ALS patients? I guess I'm trying to get a sense of how many patients with SOD1 ALS are currently identified today and how easy or difficult it will be to identify eligible patients once tofersen is approved?
Stanley Crooke -- Chief Executive Officer
Well, first of all, thanks for the all the kind comments and I'll turn the question over to Frank.
Frank Bennett -- Senior Vice President of Research
Yes, so in major medical centers to treat ALS patients, almost all the ALS patients are screened for genetic variants that cause ALS, that's largely driven by the C9orf72 mutations with there, a new patients being identified. For those patients who have SOD1 ALS, it's been in their family for generations and so many of the families have seen a parent or a cousin or a brother succumb to the disease so it's pretty quickly, genetically identified that they're at risk for the disease. And I would guess with this data, the screening is going to be ramped up. And so I would anticipate that there are more patients that will be identified that have side one mutations as a contributor to ALS.
Stanley Crooke -- Chief Executive Officer
Do you want to comment on the number of patients that are currently identified as having a SOD1 driven ALS?.
Frank Bennett -- Senior Vice President of Research
Yes. So, approximately 2% of the patients -- 2% of all the ALS patients have SOD1 ALS patients and so that's probably in the US, close to 1,000 patients and with the similar number in Europe, they are identified and they do anticipate that that number will increase with the data that we just announced.
Stanley Crooke -- Chief Executive Officer
Thanks, Frank.
Rick -- SVP Leerink -- Analyst
Thanks. It's very helpful. Just speaking more generally about the programs in ALS, could you share with us if there's been any learnings or takeaways from the 2% of clinical data that are helping to shape the way you're thinking about the development of treatments for the C9orf72 ALS?.
Frank Bennett -- Senior Vice President of Research
Yes, I mean that -- what we identified with the SOD-1 ALS is really the first drug to ever be put into patients, that's on target. So we know what causes the disease and the drug directly targets toxic protein that's being produced, and so that gives us a lot of confidence that for another genetic variants of ALS, the C9orf72 patients that by directly targeting the gene that causes the disease and reducing the toxic protein that we have a high probability of being successful, we still have to prove that in the clinical space, but that gives us a lot of confidence. There are some movements that we're learning and along the process as far as what clinical endpoints are -- good solid clinical endpoints to use in the clinical studies, and we are incorporating those into this study and we're eliminating some other endpoints that were valuable for SOD1 patients and interpreting the data. So it's helping to streamline the C9ORF72 program.
Stanley Crooke -- Chief Executive Officer
And I think more broadly, It's just another manifestation of the value of having multiple entries in disease categories that are of strategic importance, that aggregate learning that one achieved by having multiple medicines for ALS, multiple medicines for Alzheimer's, multiple medicines for NASH and other problems, it should tremendously valuable and the other thing that we learned from every study since we have this integrated safety database that we publish all the time line is we learned a great deal about the safety tolerability of our agents -- as affected by disease, and affected by the different sequences that are in those medicines. So specifically to ALS a lot has actually been learned and as being learned but more generically that strategy, we think is really paying important dividends for us, particularly with comments, behaviors ASLs of similar of the same chemical class.
Rick -- SVP Leerink -- Analyst
Great, thanks for taking our questions.
Unidentified Speaker
You bet.
Operator
The next question is from David Lebowitz with Morgan Stanley. Please go ahead.
David Lebowitz -- Morgan Stanley -- Analyst
Thank you very much for taking my question. I was just curious if you could remind us on the size of the EAP program for TEGSEDI. How many -- how big was the program? And I guess how much of it has been transitioned thus far to commercial drug?
Brett Monia -- Chief Operating Officer
Sure David, Akcea has not disclose details on the numbers of patients in the EAP nor how may of those patients constitute the sales that they present yesterday. What we can say is that we have a very nice mix of patients on commercial right now from the EAP or -- as well as a lot of new scripts as well and the scripts being written and balance -- and we are pleased about the balance between the EU and the United States as well. But those details are still haven't been disclosed.
Stanley Crooke -- Chief Executive Officer
Yes. It's simply the number of new prescriptions is key factor that we're watching and what fraction of those come from our naive patients as opposed to those that have been previously exposed to TEGSEDI and we're pleased with what we're seeing.
David Lebowitz -- Morgan Stanley -- Analyst
Thanks for taking my question.
Stanley Crooke -- Chief Executive Officer
You bet.
Operator
The next question is from Jim Birchenough with Wells Fargo Securities. Please go ahead.
Yanan -- Wells Fargo Securities -- Analyst
Hi, thanks for taking the question. This is Yanan (ph) in for Jim. So first question is on SPINRAZA in adult patients, could you talk about your experience in terms of efficacy, for example for SPINRAZA in adult patients because it's a value driver due to the high prevalence and also I think that at SGCT, one doctor or presenter mentioned that some centers may not be treating adult patients due to -- for control data. Could you talk about maybe the extent of that kind of situation.
Stanley Crooke -- Chief Executive Officer
Of course, what we know is, it does not derive from a randomized, placebo-controlled trial. But what we have learned is the SPINRAZA appears to produce similar quality of responses and character of benefit in the adult patient as in the adolescent as in infants and the child with SMA. And I would refer you to Biogen for the-to answer the second question.
Yanan -- Wells Fargo Securities -- Analyst
Got it. Then on TEGSEDI, so just wondering, you commented that you have preserved patients switching from competing medicines. Could you elaborate a little bit on that for example, what drives patient to switch and also perhaps characterize a little bit more on your effort in increasing the diagnosis rate given that the vast majority of additional patients are remain undiagnosed?
Elizabeth Hougen -- Chief Financial Officer
Hi Yanan, Its Beth. Good morning. So on the question regarding conversion, what we are seeing, our patients converting both in the US and EU from both competitive products and I think from our perspective and Akcea's is perspective, TEGSEDI provides an important benefit in the independents that it allows these patients through the subcu once weekly administration and we believe that that is being realized in the marketplace. So that's our view on the conversions.
Stanley Crooke -- Chief Executive Officer
I think there's probably a great deal more information that's developing in patients who are and converting and there are issues with limited efficacy of perhaps one of the agents as being used in their real issues with IV infusion and that sort of thing, that are certainly drivers. But I don't think we know enough to comment definitively on the factors that are driving each conversions.
Operator
The next question comes from Ritu Baral with Cowen. Please go ahead.
Ritu Baral -- Cowen -- Analyst
Good morning everyone, thanks for taking the question. I wanted to ask you about another specific comment you made during the TEGSEDI launch. You mentioned that--you were seeing cardiologist, neurologists and hematologists prescribing, is that sort of rank order of the distribution of prescribing physicians that you're seeing and how is that mix as well as payer coverage evolving with continuing conversations with payers and insurance plans?
Stanley Crooke -- Chief Executive Officer
Well, we don't -- we're not going to answer explicitly the question of the number of ratio of physicians of various specialties because that's number going to change all the time. What I can say and I would again encourage you to talk to our colleagues at Akcea for more detail. What we can say is that we are pleased with the kinds of specialties that are involved in prescribing TEGSEDI and Beth if you want to handle the other question please.
Elizabeth Hougen -- Chief Financial Officer
In terms of payers (inaudible) we've got -- we're in under contract with the top 10 payers in the country, we have relationships with all the payers. We've got the vast majority of covered lives under contract and so we're not seeing reimbursement as any of a detriment to TEGSEDI's commercial acceptance that we're having no issues that whatsoever. So we're very pleased with our relationships with payers.
Ritu Baral -- Cowen -- Analyst
Are you finding that you're having conversations about value based outcomes or agreements or any other conversations on prior authorization or step edits with certain plans?
Elizabeth Hougen -- Chief Financial Officer
We're more than happy to enter into a value-based agreements with payers. But in our conversations, they just haven't been interested in that. Their view is that for the patient population for TTR amyloidosis with polyneuropathy that value based agreements are just not meaningful. And so while we are open to the idea and continue to be open to the idea, we're not seeing that being requested from payers. And as I said, we've got TEGSEDI covered by the top at least the top 10 and relationships with all the rest.
Stanley Crooke -- Chief Executive Officer
Once again, I would refer you to our colleagues at Akcea for more definitive answers.
Ritu Baral -- Cowen -- Analyst
Got it. Thanks for the questions.
Stanley Crooke -- Chief Executive Officer
You bet.
Operator
The next question comes from Eliana Merle with Cantor Fitzgerald. Please go ahead.
Eliana Merle -- Cantor Fitzgerald -- Analyst
Hi guys, thanks so much for taking the question and congrats on all the update at AAN. So just another question on the SOD1 program, at 100 meg dose if I'm looking at the poster correctly, it looks like you're seeing about like a 37% reduction in the SOD1 protein. I guess what your plans for the dose levels going forward and I mean, do you have plans to dose escalate further, to increase the SOD1 reduction and I guess just to pose the question, like why not try to maximize the reduction of SOD1? Thanks.
Brett Monia -- Chief Operating Officer
Yes, so to recognize the measurement, the SOD1 and CSF not SOD1 and CNS tissues and so the SOD1 and CSF underestimates the amount that's being reduced in spinal cord and motor cortex and in the case of ALS. And so we identified that was a dose that was more than adequate to produce clinical benefit in pre-clinical models, I should say. And so we're very happy with the doses that we've identified and I don't anticipate us exploring higher doses for the studies,
Stanley Crooke -- Chief Executive Officer
Probably even more important than that is of how profound the evidence of benefit that was observed in 3-month study in the initial work that we've done. So optimizing dose in rare diseases is always a challenge and we think we've got a very effective dose and I wouldn't preclude overtime and other doses in schedules we studying but we're very pleased with the sort of responses we've gotten with this dose and we're going to continue to use it.
Eliana Merle -- Cantor Fitzgerald -- Analyst
Got it. That's very helpful. Thanks for the color. And then I guess just on Huntington's. Could you help us think about what we should expect in terms of further updates on the program as we progress through the year? I know you guys, it looks like you scheduled to have a presentation at the Huntington's meeting in late June. How should we think about what we might see at that on presentation?
Brett Monia -- Chief Operating Officer
Yes. So the Huntington's meeting in June is a patient meeting and so they'll have a presence where they're presenting to the patients. But I would not expect a scientific update. We're looking for medical meetings in latter part of the year or early next year to provide a next update on the open label extension. And then the Phase III study, of course it's a blinded studies, so there will be no updates on that.
Unidentified Speaker
And once again, I refer you to Roche.
Eliana Merle -- Cantor Fitzgerald -- Analyst
That's helpful. Thank you.
Operator
The next question is from Jessica Fye with JPMorgan. Please go ahead.
Jessica Fye -- JPMorgan -- Analyst
Hey, guys. Good morning afternoon and thanks for taking my question. Maybe just first one, just following up on the acromegaly LICO product. What's the dosing frequency there? Is it comparable to the SSD depot injections, and what's the target reduction of growth hormone receptor and how does that translate to IGF-1 reduction?
Stanley Crooke -- Chief Executive Officer
Well it's weekly dosing like -- no it's monthly dosing we're using for all of our LICA drugs. And as we have indicated for the rest of the LICA family of drugs, if we thought it would be better for the patient to go to quarterly dosing and it would be very simple for us to do it. Of course, we're reducing directly growth hormone receptor and in animals, we showed that there was an almost direct correlation between growth receptor reduction and you measure that with growth hormone binding protein in blood and IGF and of course, we don't know yet what that relationship is in patients that's going-that is one of the primary questions we're asking in the study and we expect to have preliminary answers to that question and lots of others later this year.
Jessica Fye -- JPMorgan -- Analyst
Okay. And then when we think about the TEGSEDI number reported for the quarter, it seems like share was about 20% just based on sales. I'm curious if you're seeing signs pointing to better share over time, maybe based on the feedback you're getting from AKCEA and if so can you talk a little bit about what the leading indicators might be there?
Stanley Crooke -- Chief Executive Officer
Yes again, as we think about the launch of TEGSEDI versus our competitor, we remember that we are on a quarter behind. And so we compare that $7 million to $12 million that was reported in the first quarter of this year. And so in that sense, we are very pleased with the market share that we have, I think it's still far too early to draw a firm conclusions about what market share TEGSEDI will have versus others. I would let the numbers tell that as the year progresses.
Jessica Fye -- JPMorgan -- Analyst
Okay, great. And maybe just the last one. I think Novartis on their call was highlighting the durability of benefit with Zolgensma and I guess while acknowledging that maybe parents still want to do that all they can to treat kids with SMA. So I'm curious if you expect any disruption in type I new starts on SPINRAZA when that product launches? Just any-or any kind of, like, lag in starting SPINRAZA in patients can they go on gene therapy?
Stanley Crooke -- Chief Executive Officer
I do think that the data Roche presented are encouraging -- Novartis sorry. But the way I think that this is not about that drug or any other drug but about SPINRAZA. So SPINRAZA is proven to work and proven to work extraordinarily well. There are 7,500 patients on treatment, we've treated patients for more than six years. We know absolutely know that if we treat SMA patients before symptom onset then most of the infants that were developed like normal healthy children, it's really hard to be healthy and we also know that we have great benefit in the longer we treat to better in older patients.
And finally, we have an established essentially pristine safety database with many thousands of years of patient treatment. So as a parent, if I had a baby diagnosed with SMA, there is no way I would use other less study agent with unknown levels of durable efficacy and side effects on my baby in place of SPINRAZA. So that's speaking for myself but I can't imagine many parents to feel in the other way.
Jessica Fye -- JPMorgan -- Analyst
Okay great. Thank you.
Stanley Crooke -- Chief Executive Officer
You bet. One more question and then we'll close.
Operator
And that question comes from Yale Jen with Laidlaw & Company. Please go ahead.
Yale Jen -- Laidlaw & Company -- Analyst
Thanks folks for squeezing me in and congrats on the progress. Just a follow-up on the tofersen and that data release at AAN that Biogen provided some clinical data there including as rating scale and lung function improvement. So would you mind comment a little. So would you my comment a little bit more for more details on what do you see that data and how would that potentially translate to that ultimate benefits to the patients ?
Brett Monia -- Chief Operating Officer
Yes, so the ALS FRS is standard rating scale for ALS that's what all companies use for our determining whether there's an improvement or decline in ALS patients outcome. And generally untreated in the general population, there was about a 1 to 1.5 point drop per month in the disease and over 3 months that's a 4.5 point drop which I think is what they saw in this study as well for this, the general population and the rapidly progressing patient populations they declines even faster and what we observed as we stabilize the disease in these patients over three months there they essentially was no decline, which is truly remarkable in ALS patients. Generally they're trying to ferret out small changes in the slopes these declines and they're just not powered well enough to determine the effect or is this is really the first study to show stabilization of the disease and more impressively to stabilized the most rapidly progressing form of the disease in these patients. So there are a fewer at the posters at the meeting, but yes, they kind of swing through the crowd of participants there that's one of the more exciting things that's happened in the ALS in the last 20-25 years.,
Stanley Crooke -- Chief Executive Officer
And I think it's a little too early to comment on the various components of the rating scale, but at least at preliminary first blush, it looks like the improvements are occurring pretty much across-the-board.
Yale Jen -- Laidlaw & Company -- Analyst
Yes. And just curious, do you anticipate or do the people anticipate the drug that get approved if the current trends, or at least short-term trend continues or maintained.
Stanley Crooke -- Chief Executive Officer
Yes.
Yale Jen -- Laidlaw & Company -- Analyst
Okay, great, thanks a lot. Appreciate it.
Stanley Crooke -- Chief Executive Officer
Thank you. Well first, thanks everyone for your attention and interest in the work that we're doing. I want to close by emphasizing our financial performance. Our business model is working and our first quarter financial results were very impressive and very impressive in the context of aggressively spending across the Board in every single element of the business and so we think that puts us in a uniquely strong position to continue to expand and invest and we have great things to invest in. We have the technology that is generating medicine in an extraordinarily efficient way and medicines that are truly transformational. So this is indeed the beginning of another extraordinarily exciting year for IONIS and we'll be telling you about it, and we look forward to talking about the coming months. Thanks.
Operator
The conference is now concluded. Thank you for attending today's presentation. You may now disconnect.
Duration: 59 minutes
Call participants:
Wade Walke -- Vice President, Investor Relations
Stanley Crooke -- Chief Executive Officer
Elizabeth Hougen -- Chief Financial Officer
Brett Monia -- Chief Operating Officer
Frank Bennett -- Senior Vice President of Research
Unidentified Speaker
Chad Messer -- Needham & Company -- Analyst
Ben Burnett -- Stifel -- Analyst
Rick -- SVP Leerink -- Analyst
David Lebowitz -- Morgan Stanley -- Analyst
Yanan -- Wells Fargo Securities -- Analyst
Ritu Baral -- Cowen -- Analyst
Eliana Merle -- Cantor Fitzgerald -- Analyst
Jessica Fye -- JPMorgan -- Analyst
Yale Jen -- Laidlaw & Company -- Analyst
