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Ionis Pharmaceuticals, Inc. (IONS 1.88%)
Q3Â 2020 Earnings Call
Nov 4, 2020, 11:30 a.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Good morning, and welcome to the Ionis Pharmaceuticals Third Quarter 2020 Financial Results Conference Call. [Operator Instructions]
At this time, I would like to turn the call over to Wade Walke, Vice President, Investor Relations to lead off the call. Please go ahead.
D. Wade Walke -- Vice President, Investor Relations
Thank you, Brandon. Before we begin, I encourage everyone to go to the Investor section of the Ionis website to find the press release and related financial tables, including a reconciliation of 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 have also posted slides on our website that accompany our discussion today.
With me on today's call are Brett Monia, our Chief Executive Officer; Beth Hougen, Chief Financial Officer; Onaiza Cadoret, our Chief Corporate Development and Commercial Officer; and Richard Geary, Executive Vice President of Development. And joining us for Q&A, we have Eric Swayze, our Executive Vice President of Research and Kyle Jenne, Chief Commercial Officer of Akcea.
I would like to draw your attention to Slide 3 which contains our forward-looking language. We'll be making forward-looking statements which are based on our current expectations and beliefs. 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 Brett.
Brett P. Monia -- Chief Executive Officer
Thanks, Wade. And good morning, and thank you for joining us on today's call. As we near the end of 2020, we are well positioned to achieve our next stage of growth. Since our last update, we took the very important step of acquiring Akcea. We believe that together as one company we're stronger and more efficient with an even greater ability to achieve success today and well into the future.
This transaction also represents a significant step forward in the evolution and execution of our commercial strategy, by combining Akcea's commercial capabilities with the Ionis-owned pipeline, we move closer to fully maximizing the value of these medicines. And we're very pleased with the excellent progress we're making across our Ionis-owned pipeline and our pipeline overall.
Our five ongoing Phase 3 studies continue to progress well. We also remain on track to begin our sixth Phase 3 study when we initiate Phase 3 testing of APOCIII-LRx in FCS patients later this year. We're also evaluating larger indications for this medicine with an additional pivotal study potentially starting next year.
Beyond our Phase 3 pipeline, we continue to make excellent progress with our medicines in mid-stage development. The IONIS-ENAC-2.5Rx data we reported last month provided additional support for our rapidly expanding platform of inhaled antisense medicines. Vupanorsen, IONIS-Factor XI LRx and IONIS-HBVRx all advanced further in development with each medicine entering Phase 2b studies.
ION541, one of our four ALS medicines in development entered a Phase 1/2 study in patients with nearly all forms of ALS regardless of family history. IONIS-PKK-LRx has completed enrollment in a Phase 2 study in patients with hereditary angioedema, which is due to read out next year and is also under evaluation in a Phase 2 investigator study in hospitalized patients with severe COVID-19 complications in Brazil.
And we further extended the reach of our technology this past quarter through a collaboration with Genuity Science, whose unique approach for new target identification is expected to enhance our already robust drug discovery and development capabilities.
With these achievements, we are closer than ever to reaching our goal of 10 or more marketing applications through 2025, which should result in a large number of new marketed products. Importantly, we remain financially strong and on track to achieve our 2020 guidance.
Coming up, on December 7, we will be hosting our virtual Investor Day. During the presentation, we look forward to sharing our commercial plans, provide several development program updates and highlight some of the great progress we're making that is extending the reach of our technology. More information about this event will be released soon.
And with that, I'll hand the call over to Beth to take us through our Q3 financial results. Onaiza will then talk about the Akcea integration and Richard will review our recent pipeline achievements. After Richard, I'll wrap up our prepared remarks before taking your questions.
Now, over to Beth.
Elizabeth L. Hougen -- Executive Vice President, Finance and Chief Financial Officer
Thank you, Brett. As we approach year end, we remain financially strong and well capitalized, with the resources to achieve our goal of significant growth. Our acquisition of Akcea is one of the steps we have taken this year to achieve this goal. By integrating the two companies into one, our business is further strengthened in numerous ways, including improving our already strong financial position.
We now retain more value from Akcea's rich pipeline and commercial products. Additionally, we can use Akcea's current cash and future cash flows to advance our strategic priorities. And beginning next year, we expect to realize meaningful cost synergies from our integration activities.
We delivered strong financial results in the third quarter, with revenues increasing by 10% compared to the prior quarter and we earned operating income and net income both on a non-GAAP basis.
SPINRAZA generated global sales of $495 million in the third quarter, resulting in $74 million in royalty revenue to Ionis. At the end of September, there were over 11,000 patients on SPINRAZA treatment worldwide, with growth driven by markets outside the United States. With the significant number of untreated SMA patients across numerous established and emerging markets, together with SPINRAZA's proven efficacy and safety profile, we and Biogen continue to see opportunities for growth.
Biogen's DEVOTE study is progressing well. This study is evaluating the potential for higher doses of SPINRAZA to deliver even greater benefit to SMA patients of all ages. Enrollment in the open-label safety cohort is now complete with enrollment in the randomized pivotal cohort to follow next. In early next year, Biogen plans to initiate the RESPOND study to evaluate SPINRAZA's benefit in patients with a sub-optimal clinical response to gene therapy.
TEGSEDI and WAYLIVRA continued to deliver growth in the third quarter. Product sales were $19 million, an increase of more than 15% compared to last quarter and a nearly 60% increase over last year. We continue to see growth in the number of patients on TEGSEDI across North America and Europe.
We believe patients and physicians are choosing TEGSEDI in part due to its subcutaneous self-injection, enabling patients to take this therapy at home. This is one of the important benefits of TEGSEDI, which has been especially important during the ongoing pandemic.
In the United States, patients are starting and remaining on therapy. Thanks, in part, to the excellent service provided by Akcea's patient support program, Akcea Connect. We continued to see growth with Akcea's genetic testing program with over 2,200 physicians and an increasing number of tests conducted to-date.
In Europe, we successfully achieved reimbursement and recognized revenue from multiple new markets, including Portugal, home to a large endemic hATTR patient population. And in Canada, TEGSEDI reimbursement is in place in the largest provinces and through multiple private payers. We also moved closer to achieving public reimbursement for all TEGSEDI patients in Canada. In Latin America, PTC Therapeutics is focused on new patient finding, while continuing to negotiate pricing in Brazil.
Now, turning to WAYLIVRA, where we are generating revenue from a growing number of EU markets. In the U.K., Akcea finalized pricing and reimbursement with NICE. And in Latin America, PTC filed for marketing authorization in Brazil earlier this year and is working to expand access in other Latin American markets. And in the United States, we plan to refile with the FDA next year.
During the third quarter, we earned R&D revenues of $65 million. This revenue included over $50 million from our neurological disease franchise, primarily driven by several Biogen-partnered programs. We expect a substantial increase in R&D revenue in the fourth quarter, including the $75 million milestone payment from Pfizer.
Additionally, we recently obtained a favorable award in our arbitration proceeding with Alnylam. This award pertains to fees arising from Alnylam's agreement with Sanofi related to Fitusiran and its TTR products. The final judgment has not been determined, but we expect it will add meaningful revenue in the fourth quarter.
Our non-GAAP operating expenses in the third quarter increased compared to last year, primarily driven by our Phase 3 program for AKCEA-TTR-LRx and progress with our other Ionis-owned medicines. With these results, we achieved operating income of $9 million and net income of $5 million for the third quarter, both on a non-GAAP basis.
Early last month, we used our strong balance sheet to successfully complete our acquisition of Akcea. Following the acquisition, we remain in a strong financial position with an estimated pro forma cash balance of $1.8 billion. Importantly, our entire cash balance is now available to us to advance Ionis' strategic priorities and to build value for our patients and our shareholders.
We also anticipate achieving meaningful cost synergies as we progress the integration activities. And, our financial statements will be much simpler. For example, beginning next year, we will no longer include a non-controlling interest adjustment on our P&L or balance sheet.
With our third quarter results and our projections for increased revenues in the fourth quarter, we are on track to achieve our 2020 financial guidance of meaningful profitability. Our financial strength including our substantial cash position enables us to continue to invest in areas that we believe will accelerate growth.
And with that, I'll turn the call over to Onaiza to provide an update on the Akcea integration and progress with our commercial strategy.
Onaiza Cadoret-Manier -- Executive Vice President, Chief Corporate Development and Commercial Officer
Great. Thank you, Beth. With the acquisition of Akcea now closed, our process to merge the two companies into one is well under way. The transition team, which is made up of cross-functional leadership from both companies is following an established playbook to achieve full integration, while focusing on three primary goals.
Our highest priority is to ensure our patients continue to receive TEGSEDI and WAYLIVRA without interruption and with a high level of personalized support. Second, we are focused on ensuring our employees remain highly engaged and productive. And finally, we are looking to advance our commercial plans for the Ionis-owned pipeline with the addition of Akcea's commercial capabilities.
As we integrate the two companies, we are carefully reviewing all aspects of our business processes to ensure we are driving the greatest value for patients and shareholders. Through Akcea, we now have access to payer, health economics and outcomes research, medical affairs and other capabilities to support our Ionis-owned pipeline.
We look forward to adapting these capabilities and accelerating our go-to-market strategies as our medicines advance toward the market. At our Investor Day next month, I look forward to discussing our commercial plans for our neurological and other rare disease programs, as well as TTR LICA and APOCIII LICA.
And with that, I'll turn the call over to Richard to discuss our key pipeline highlights.
Richard S. Geary -- Executive Vice President, Development
Thank you, Onaiza. As Brett said, we've continued to advance our pipeline in the third quarter with significant achievements across our neurological, pulmonary and cardiovascular disease franchises.
Starting with the neuro pipeline, the Tominersen Phase 3 study in patients with Huntington's disease remains on track for data and potential regulatory filing in 2022. Ahead of the Phase 3 data, our partner Roche plans to provide an update from the Phase 1/2 open-label extension and natural history studies next year. The Phase 3 study of AKCEA-TTR-LRx in patients with TTR polyneuropathy also continues to progress.
Our ALS program has continued to grow with four medicines now in our pipeline for the treatment of ALS. The Phase 1/2 study of ION541, our first medicine designed to address nearly all forms of ALS regardless of family history, is well under way. The Phase 3 study of Tofersen in patients with SOD1-ALS is progressing with data expected in the second half of next year.
Also next year, Biogen plans to initiate a study of Tofersen in pre-symptomatic SOD1-ALS patients with the potential to delay or prevent disease onset. The Phase 1/2 study of IONIS-C9Rx in patients with C9-ALS remains on track for data next year. And ION363, our first Ionis-owned ALS medicine for the treatment of ALS patients with mutations in the FUS gene, remains on track to enter a registrational study next year. Looking beyond these programs, we continue to advance multiple earlier stage programs for the treatment of ALS.
Now, from our growing pulmonary franchise, we recently reported positive IONIS-ENAC-2.5Rx healthy volunteer data, demonstrating significant and substantial reductions in ENaC levels in the lung with attractive safety and tolerability. Importantly, these data also support inhalation as a viable route of delivery for antisense medicines more broadly, gives us greater confidence for positive results in our ongoing study in cystic fibrosis patients, and opens a path for us to broaden our pulmonary franchise to numerous new diseases of the lung representing a significant opportunity for growth.
In fact, we look forward to initiating the study of IONIS-ENAC-2.5Rx in patients with COPD later this year. We also made significant progress in the development of IONIS-PKK-LRx. Enrollment in the Phase 2 study in patients with hereditary angioedema or HAE is now complete with data expected next year.
Earlier this year, results from a compassionate-use study with our PKK program were published in the New England Journal of Medicine demonstrating its potential to reduce the frequency of attacks in patients with HAE. IONIS-PKK-LRx also recently advanced into an investigator study in patients hospitalized with severe COVID-19 complications in Brazil, designed to prevent the formation of bradykinin, this medicine has the potential to significantly reduce a cause of the severe complications characteristic of the COVID-19 virus.
Turning now to our cardiovascular disease franchise; the Phase 3 study of Pelacarsen in patients with Lp(a)-driven cardiovascular disease continues to progress with data expected in 2024. Our Phase 3 study of AKCEA-TTR-LRx in patients with TTR cardiomyopathy also continues to progress. We also made excellent progress with our mid-stage cardiovascular disease pipeline.
Pfizer recently initiated a Phase 2b dose-finding study of vupanorsen, which is expected to readout in the second half of next year. This study is being conducted in patients with dyslipidemia, despite stable LDL lowering treatment, which is representative of its planned Phase 3 program focused on the over 6 million patients in the U.S. at high risk for cardiovascular disease, despite maximum LDL-C lowering therapy.
To support this strategy, Pfizer is planning three Phase 3 studies of vupanorsen, including a large cardiovascular outcome study and studies in patients with elevated triglycerides and severe hypertriglyceridemia. We expect all three of these studies to get under way in 2022.
We and AstraZeneca look forward to providing an update on progress of our medicine targeting PCSK9 for the treatment of cardiovascular disease at the AHA later this month. AZ's presentation at AHA will include updates from both the subcutaneous and oral formulations of this medicine.
And before the end of the year, we look forward to initiating a Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS. These achievements move us closer to 10 or more marketing applications through 2025.
And with that, I'll turn the call back over to Brett to close this portion of the call.
Brett P. Monia -- Chief Executive Officer
Thank you, Richard. In closing, I'm pleased to say that Ionis is stronger than ever. We are financially strong and have taken several important steps recently to further strengthen our financial position. With the Akcea acquisition now complete, we are one company with one leadership team and one set of strategic goals positioning us well for the future.
We are achieving a great deal of success across the business. Our pipeline continues to advance well and deliver value for patients and investors. Our five Phase 3 studies are advancing with a sixth expected to start by year's end. Furthermore, we look forward to numerous value-driving catalysts in the remaining months of this year and next year, including data from the Phase 3 study of Tofersen in patients with SOD1-ALS in 2021. We believe that Tofersen has the potential to be the first disease-modifying medicine approved for patients with ALS.
We've also made excellent progress in our mid-stage pipeline. We successfully completed several important Phase 3 studies, advanced inhaled delivery and initiated several important Phase 3 studies. Our success this year puts us in an even better position to achieve our goal of 10 or more marketing applications through 2025, which we expect will result in a large number of new marketed products.
We are also very proud of TEGSEDI winning the Prix Galien USA Award for Best Biotechnology Product in 2020. Our vision in the discovery and development of TEGSEDI was to improve the lives of patients living with devastating effects of TTR amyloidosis, a rare genetic disease that greatly impacts the lives of families over generations.
This is Ionis' second transformational medicine to win the prestigious award. The first being SPINRAZA, which transformed the treatment landscape for SMA, the leading genetic cause of infant death. This award is a testament to the hard work and dedication of our employees who work tirelessly to improve the lives of patients suffering with serious diseases.
And with that, I'll open the call for questions.
Questions and Answers:
Operator
We will now begin the question-and-answer session. [Operator Instructions] Our first question comes from Do Kim with BMO Capital Markets. Please go ahead.
Do Kim -- BMO Capital Markets Corp. -- Analyst
Hi, thanks for taking my questions. A question on the ENaC inhaled ASO, the Phase 1 data. Just wanted to get your thoughts on how the PK data tracked with the reduction of ENaC mRNA as the dose escalated? Do you think you saw a dose response? And whether you had any thoughts on why a highest dose at three times weekly didn't show much of a reduction?
Brett P. Monia -- Chief Executive Officer
So, Do, I'll take that and thanks for the question, first of all. I'll take a stab at it and then I'll toss it over to Richard to expand. So, our Phase 1 data in which we -- in normal volunteers, in which we demonstrated excellent safety, tolerability in normal volunteers with dose-dependent reductions in ENaC, was very consistent with what we expected based on a lot of preclinical data.
The effects, as I said, were dose-dependent and we achieved significant -- statistically significant reductions in ENaC that are well beyond the reductions that we expected to produce efficacy based on preclinical data we've generated so far. And now, as we've said, as Richard highlighted, we're now in a Phase 2 study in patients with CF to demonstrate clinical benefit, which we hope to share next year.
Richard, do you want to get into the PK relationship a little bit more in detail?
Richard S. Geary -- Executive Vice President, Development
Yes. So, we saw the dose dependent reductions in ENaC and that did track with our pharmacokinetics. And the highest bolus dose, which was given less frequently than lower doses given more frequently did track -- PK did track with the effect we saw. And I think that is what has given us confidence as we've moved forward in the CF population and also are on the verge of initiating the COPD study.
Do Kim -- BMO Capital Markets Corp. -- Analyst
Great, that's very helpful. And just a quick question on the ION541 for most types of ALS, do you think that that therapy will also be effective in the familial ALS, like SOD1 and C9Rx [Phonetic] and would you consider a strategy or Biogen consider a strategy for a combo with the mutant specific therapies?
Brett P. Monia -- Chief Executive Officer
The ION541 is targeting ataxin 2 and for all forms potentially of ALS. And yes, we -- there is strong rationale for this target by knocking it down with 541 to show efficacy in genetic forms or hereditary forms of ALS, as well as sporadic. Eric, more on that?
Eric E. Swayze -- Executive Vice President, Research
Yes, sure. So, the mechanism of action of this drug is it modulates TDP-43 pathology by down-regulating -- by reducing the level of ataxin 2, which has been shown to be important in that pathology and most forms of ALS involve some pathology of TDP-43. So, we'd expect that drug to work in any form of ALS that has that type of pathology and treat most all forms of ALS.
Brett P. Monia -- Chief Executive Officer
And your question about combination. Yes, theoretically, you can envision a scenario in which combining a SOD1 with an ataxin 2 inhibitor, which provide even greater benefit, we'll have to see how the data shakes out in the clinic. I mean, SOD1, we think, is going to show great efficacy in the clinic and we'll see how much better we can achieve beyond that in those particular patients. But from a mechanistic standpoint, you can see the rationale for added benefit, yes.
Do Kim -- BMO Capital Markets Corp. -- Analyst
Great. Congrats on the progress, and thanks for taking my questions.
Brett P. Monia -- Chief Executive Officer
Thanks, Do.
Operator
Our next question comes from Chad Messer with Needham & Company. Please go ahead.
Gil Blum -- Needham & Company -- Analyst
Hello, everyone. This is Gil on for Chad, and thank you for taking our questions. I have a bit of a more general question. So, as to the Akcea platform and the new commercial capabilities, how transferable are they to a cross indications? I mean, could you envision this platform being used, for example, to promote drugs in neurological indications?
Brett P. Monia -- Chief Executive Officer
That's a great question. We're very excited about the reacquisition of Akcea because as Onaiza said in her discussion earlier, it really accelerates one of our top priorities, it is to build out our commercial capabilities and we think that -- and we're building our strategy for that. And as you said, we're planning to share some details on that strategy in December at Investor Day, and we do believe it will be broadly applicable, these capabilities to areas that we want to invest in, including neurological.
Onaiza, would you like to expand on that?
Onaiza Cadoret-Manier -- Executive Vice President, Chief Corporate Development and Commercial Officer
Sure. Yes, great question. You know in any -- building out any kind of rare disease capabilities, there are some that are transferable and leveraged across therapeutic areas, right? We think about these things as our health economics capabilities, the payer space, our access hub as it is adapted to the therapeutic area or areas where we see that the capabilities can be applied and really build a very customized approach to neurology, as well as to the lipid cardiology space that the team has been in.
But as you know that when you look at more of the marketing and sales people that will obviously be based on kind of the customer types and we wouldn't see a lot of overlap there. But with polyneuropathy as well as -- going into our neurology portfolio, we're looking to see whether we're finding some good customer-facing synergies there as well. So, hopefully that gives a good flavor.
Gil Blum -- Needham & Company -- Analyst
Thank you. That's very helpful. And maybe a question for Richard on ENaC. Just to remind us, is there something special in the ENaC agent's chemistry or a [Indecipherable] so that makes it more absorbable in the lung -- just a little bit of color on that.
Richard S. Geary -- Executive Vice President, Development
So, as far as cell uptake, oligonucleotides delivered to the lung by aerosol are well taken up in multiple cell types. So, the general state of cell uptake, there is nothing really special about the chemistry for uptake. However, ENaC is a 2.5 or a CF [Phonetic] bicyclic sugar modification, which makes it very much more potent than the [Indecipherable] modifications. And so, we've increased potency and we have the similar distribution within the lung.
Gil Blum -- Needham & Company -- Analyst
All right. Excellent. And just one last question from me. So, you mentioned the post-marketing study with SPINRAZA in gene therapy failures. I'm assuming this is taking forward of a label expansion, is that a right way to look at it?
Brett P. Monia -- Chief Executive Officer
Biogen has not talked about how, assuming a positive outcome in the RESPONSE study, would impact a label change and that sort of thing. So, we don't want to get too far ahead on that. But, we are feeling pretty confident that SPINRAZA will demonstrate benefit -- significant benefit to patients who are performing sub-optimally on gene therapy, as I'm sure you're aware, Gil, there is anecdotal -- quite a bit of anecdotal news out -- information out there on patients who don't perform well as well as desired on gene therapy even with not in SPINRAZA.
And what Biogen wants to do is take that anecdotal information and turn it into a controlled trial, so they can prove it. And, obviously, they're doing that to enhance the value of SPINRAZA to patients and to shareholders. So, I wouldn't put that outside the realm of possibility, but I don't want to get too far ahead already here.
Gil Blum -- Needham & Company -- Analyst
Alright. Much appreciate the color, and congratulations on all the progress. Thank you.
Brett P. Monia -- Chief Executive Officer
Thanks, Gil.
Operator
Our next question comes from Jim Birchenough with Wells Fargo. Please go ahead.
Jim Birchenough -- Wells Fargo -- Analyst
Yes, hi guys. Congrats on all the progress, and thanks for taking the question. I guess, the first question is just related to the integration of Akcea and the synergies and how you see that affecting your ability to remain sustainably profitable and cash flow positive. And then I have a couple of questions on the pipeline. Thanks.
Brett P. Monia -- Chief Executive Officer
I'll let Beth take the first one Jim.
Elizabeth L. Hougen -- Executive Vice President, Finance and Chief Financial Officer
Hey, Jim. Good morning. So, I think -- so the way I would think about the Akcea integration is it absolutely moves us down the path of being closer to commercializing our Ionis-owned pipeline ourselves. And of course you know that pipeline continues to expand and advance, and it's been a key focus of ours certainly throughout this year and will continue to be as we go into the future years.
We will see meaningful synergies, cost synergies from the integration certainly putting two public companies together. There is some obvious low hanging fruit in that we won't have to sustain cost structures for two public companies going forward.
And then, as far as sustained profitability, our focus is really on our Ionis-owned pipeline and building that pipeline and expanding that pipeline, building these commercial capabilities off of the -- combined with the capabilities that we've now acquired wholly from Akcea and also looking for ways to invest to expand -- extend the reach of the technology. And so, there -- that's our highest priority, profitability is still important to us, but it is not our first and top priority.
Jim Birchenough -- Wells Fargo -- Analyst
Got it. That's very helpful. And then maybe just on ION449 and the AHA presentation. I know it's embargoed, but how should we think about what you're hoping to show vis-a-vis the subcu? And then if you've got a successful oral approach, what other candidates make most sense for the technology?
Brett P. Monia -- Chief Executive Officer
Yes. You're referring to the -- thanks, Jim. You're referring to the PCSK9 programs.
Jim Birchenough -- Wells Fargo -- Analyst
Right.
Brett P. Monia -- Chief Executive Officer
And we have [Technical Issues] formulation and the other is the oral program, both in clinical testing as I said. At the AHA, we're going to have data, clinical data on the subcu program showing reductions in PCSK9, LDL cholesterol that we are very excited about that we think have the potential to really differentiate this drug from all other drugs that are in development or are on the market today as a potential best-in-class medicine.
The oral program will also be updated at American Heart Meeting and we'll principally focus on the rationale, the strategy, the preclinical data supporting the clinical testing, in which the conclusion from that oral program will be that we think that we've solved commercially viable oral delivery as a once a day tablet that will drive reductions in target in the liver by PCSK9 to levels comparable to the subcu program I just referred to.
There may be also some clinical data from the oral program. But in the abstract, it's focused entirely on the preclinical data so far. But I also want to expand on that a little Jim. In addition to the PCSK9 program for oral, we have prioritized several programs here at Ionis for oral development that we think will provide a significant advantage differentiating -- our ability to differentiate from competition and we're planning on touching on the Ionis-owned oral programs a little bit at Investor Day in December.
Jim Birchenough -- Wells Fargo -- Analyst
Great. Thanks for taking that question. I guess, the final question is just on timelines for the neuro rare disease programs. What's the earliest we could see data from one of those Prion disease before Alexander disease, is that more of a 2022 dynamic or could we see some data from that next year?
Brett P. Monia -- Chief Executive Officer
So, we're excited about starting several of those clinical trials next year and really some of them in the first half, early next year. And as you know, because they are rare diseases and severe diseases, we are expecting a very fast clinical [Indecipherable] some of them just one study. So, we'll be providing an update on the initiation of those studies and our development strategy next year early on, but I would expect the time frame that you're thinking of is about right for clinical data.
Jim Birchenough -- Wells Fargo -- Analyst
Great. Thanks for taking the question, guys.
Brett P. Monia -- Chief Executive Officer
Thanks, Jim.
Operator
Our next question comes from Joel Beatty with Citi. Please go ahead.
Joel Beatty -- Citigroup -- Analyst
Hi. Thanks for taking the question. This is a follow-up to the last question on oral PCSK9. Just to clarify, for the biomarker data on like PCSK9 and LDL lowering for the oral molecule, is there a potential for that to come at AHA or how -- if not, how much later could that come?
Brett P. Monia -- Chief Executive Officer
The oral -- as I said, we will be presenting with AstraZeneca an update on both programs, subcutaneous and oral. The subcu will have pharmacodynamic data on PCSK9 and LDL lowering. These are on patients on top of statins. And -- whereas the oral program will principally focus on preclinical data that will set up -- that will really provide the strategy, the justification for developing this platform for oral delivery. That will certainly include PD data on preclinical models, but the clinical data we're saving into the next year for oral.
Joel Beatty -- Citigroup -- Analyst
Okay, got it. And then thinking about the potential for oral technology more broadly in your pipeline, could you talk a little bit about how broadly applicable the oral PCSK9 data would be for other agents and also how quick that could be applied to other agents in your pipeline?
Brett P. Monia -- Chief Executive Officer
Yes. It's what we've engineered for oral delivery. Again, this is looking at, it's a once-a-day tablet that will achieve target reductions comparable to any of our subcu programs that we've developed or are in development today. And the focus is on Gen 2.5 chemistry with LICA, chemistry so we get the potency of the two chemistries that really we think solves commercially viable oral delivery.
We're advancing forward several programs from the Ionis-owned pipeline now, we prioritize them and we'll talk about them soon in the Investor Day and we're expecting to move one or two of these drugs into development next year for oral delivery.
Joel Beatty -- Citigroup -- Analyst
Great, thanks for the update.
Brett P. Monia -- Chief Executive Officer
Yes. In addition to PCSK9. Thanks, Joel.
Operator
Our next question comes from Chi Fong with Bank of America. Please go ahead.
Chi Fong -- Bank of America Merrill Lynch -- Analyst
Hey, this is Chi on for Jason Gerberry from Bank of America. Thanks for taking my questions. I guess, another follow-up on PCSK9. It looked like Ionis has already had some early PK/PD data with the oral formulation. Curious, if you have a chance to review that with AstraZeneca? And what kind of feedback have we heard from the collaborator? And I'm curious, if there is any sort of initial conversation about advancing the oral formulation to further in development maybe perhaps some time next year? And then I have a couple of follow-up after that. Thank you.
Brett P. Monia -- Chief Executive Officer
All right, Chi. I need to get a clarification on your question about the first question, what data are you referring to, I -- we haven't shared data on our latest oral platform outside of what has been submitted to the American Heart by Ionis and AZ so far, can you clarify?
Chi Fong -- Bank of America Merrill Lynch -- Analyst
Curious if you have seen any human PK/PD data with the oral formulation that will not be part of the AHA like internally, whether you and partner AstraZeneca have reviewed what the initial feedback you've heard from partner and whether there is a part of advancing the oral formulation in further development perhaps sometime next year even though you're not presenting the data ahead of time.
Brett P. Monia -- Chief Executive Officer
Absolutely, yes. Sure. We have PK data from the oral program from the clinic -- in the clinic already and we're encouraged by it. It's just a question of whether or not that study will be completed in time to share at the American Heart and the abstracts were submitted earlier this year, so really focused on the strategy, the rationale, the justification for a commercially viable oral delivery focused on preclinical data. But yes, we've seen -- we have clinical data on the oral program and we have more clinical data on the subcu program, which is why we're -- there'll be more data at the American Heart on the subcu.
And as for -- I think your other question was really related to other programs. Yes, we're advancing forward additional programs using oral delivery as the route of administration, focusing on the Ionis-owned pipeline and we'll provide an update on those programs soon and we expect one or more of those drugs to reach development next year.
The other thing I'll just add to that is that we continue to invest in research, in new chemistries and new formulations to further enhance oral delivery. So, this isn't, sort of, our only stand validating commercially viable oral delivery, we continue to prioritize this in our research group.
Chi Fong -- Bank of America Merrill Lynch -- Analyst
Awesome. I guess, my follow-up -- another question would be on SPINRAZA. Curious, I think between Roche and Biogen's earnings call, kind of, early too maybe about 200 patients initially switched from SPINRAZA to risdi. Do you think that's more reflective of pent-up demand? Or do you think that's, sort of, like the rate of attrition that we might see for the next couple of quarters going forward? And, I guess, another part of the question would be, you and Biogen, how confident are you with the SPINRAZA growth profile? Do you expect SPINRAZA to have year-over-year growth for 2021 versus 2020? Thanks.
Onaiza Cadoret-Manier -- Executive Vice President, Chief Corporate Development and Commercial Officer
I would say, as far as Q3 results are concerned, I wouldn't -- we and Biogen believe that those patients were really a function of pent-up demand. We don't anticipate seeing that as a trend on a go-forward basis. I think the way to think about growth on a year-over-year basis with SPINRAZA is to think about the efficacy profile and the safety profile that SPINRAZA has demonstrated in, right now 11 -- more than 11,000 patients in the real world in a commercial setting, as well as in the clinic. And the fact that those efficacy and safety profile has been well established across all types and ages of SMA patients and over many, many years. We've been studying SPINRAZA in the clinic and it's been on the market now combined for eight plus years, so we've got lots of months of data that supports the profile.
And then I think as you think about it, there is limit to data in the competition right now, particularly the oral and that data suggests that its benefit is limited in -- to really the younger patients. As you get older and your weight increases, you have less ability to take the medicine and see the benefit. So, we are really focused on the fact that SPINRAZA's efficacy and safety sets the bar, frankly, sets a very high bar. And that combined with the fact that there are more than 60,000 patients worldwide with SMA and most of those patients are outside the United States and in markets where Biogen has a commercial presence. So, we think all of those factors bodes well for SPINRAZA's growth trajectory in coming years, including next year.
Brett P. Monia -- Chief Executive Officer
And just to add to that. As Beth highlighted earlier on the call, we're also -- we also think that the studies that are in progress or planned for SPINRAZA bode well for SPINRAZA in the future too, including the ongoing DEVOTE study in patients with -- that have had some optimal performance on gene therapy, which is to start next year and RESPONSE study, as well as the DEVOTE study in progress looking at higher doses with SPINRAZA to demonstrating the greater efficacy. So, there's a lot going on to further enhance the potential growth of SPINRAZA.
Chi Fong -- Bank of America Merrill Lynch -- Analyst
Awesome. Thanks.
Brett P. Monia -- Chief Executive Officer
You got it.
Operator
Our next question comes from Luca Issi with RBC. Please go ahead.
Luca Issi -- RBC Capital Markets -- Analyst
Terrific. Thank you for taking my questions. Two questions; one, on the strategy and two, on the competitive landscape maybe. So, on the strategy, can you just talk maybe high level of how the acquisition of Akcea and the $1.8 billion pro forma cash position changes your BD strategy?
And two, on the competitive landscape, I think we've seen Novartis receiving orphan drug designation for their oral splicing modulator for Huntington. Wondering, if you have any thoughts on that approach and maybe what are some of the implications for your program? Thank you.
Brett P. Monia -- Chief Executive Officer
Yes. I'll take a stab at the first one and ask [Indecipherable] somebody can help me with that too and then Eric maybe can talk about the Huntington program. So, the acquisition of Akcea is -- as we highlighted is right in line with our strategy to build our -- to prioritize the Ionis-owned pipeline and build our commercial strategy for the future. And this acquisition is -- it accelerates our path toward that. Onaiza will -- and Ionis, in general, we'll talk about that strategy in more detail at our Investor Day in December, but it will focus on rare diseases, at least, to start with.
And we'll talk about the types of rare diseases and which ones we're going to prioritize to bring those forward. The Ionis platform, our technology is incredibly prolific. We continue to bring many new drugs into development each year. Some of those drugs will not conform necessarily to the commercial strategy that we're planning to initiate that we're implementing, I should say, now. So, we will continue to partner, business development will continue to be a very important aspect of Ionis to maximize the value of our pipeline, our technology, maximize growth for the company. We are a hybrid, we're a commercial organization and we're a partnering organization and we think that that's the best strategy for maximizing the value to all of our stakeholders and we'll continue to do so. So, I guess, I would say that the acquisition of Akcea and building our commercial capabilities and strategy will force us to partner less in rare diseases, but we'll still continue to partner in those areas that make most sense to Ionis.
Eric, do you want to take the Huntington...
Eric E. Swayze -- Executive Vice President, Research
Yes. So, I think you're talking about Branaplam and -- with the Novartis's orphan designation. That's an older drug, that's been around a while, it's been in Phase II trials for SMA also. So, I'll let you draw your conclusions about something that modifies the splicing of two different genes. I like our drug. Tominersen is in the lead in the Huntington's space, it's in the Phase III trial, as you know, scheduled to read out in 2022, lowering Huntington directly, I think, is the best strategy to deal with Huntington's disease and I'm perfectly comfortable and happy with our strategy with Tominersen and Roche.
Luca Issi -- RBC Capital Markets -- Analyst
Super helpful. Thanks, guys.
Brett P. Monia -- Chief Executive Officer
Thanks, Luca.
Operator
Our next question comes from Paul Matteis with Stifel. Please go ahead.
Katie Schneeberger -- Stifel -- Analyst
Hi, this is Katie Schneeberger [Phonetic] on for Paul. And I just had a quick question. As we wait for the HAE results, what are you mainly looking for, whether it'd be reductions in attack rates or to extend treatment duration with LICA? And also if you could clarify the updated timing as well? That would be super helpful. Thanks.
Brett P. Monia -- Chief Executive Officer
Yes. Richard, why don't you take that one?
Richard S. Geary -- Executive Vice President, Development
Sure. Well, of course, the endpoint is going to be attack rate focused and it's also going to be on breakthrough in terms of attacks that occur well on treatment, which is one of the confounding issues with some of the existing products. So, what we expect to see is a best-in-class response. That's the approach we're taking, and the study is designed to evaluate that.
In addition to the question that you asked, which is to extend frequency of dosing; one of the issues with some of the existing platforms is that if they broaden their frequency out to more than two weeks or one month, they begin to have these breakthrough issues. So, in our open label program, we'll be looking at the ability for this medicine with its long half-life and long PD component to be able to be administered at even more frequent -- or less frequent more applicable, kind of, and competitive administration rates.
Brett P. Monia -- Chief Executive Officer
[Speech Overlap] answer your question? You got it. Thank you.
Katie Schneeberger -- Stifel -- Analyst
Yes.
Operator
Our next question comes from Yaron Werber with Cowen. Please go ahead.
Yaron Werber -- Cowen & Company -- Analyst
Yes. Hi, and thanks for taking. I have a couple of questions. The first one is maybe just a follow-on to the last one. Maybe on PKK, is the [Indecipherable] or do you view that to be, sort of, the most relevant competition, because the potency and less frequent dosing or do you feel that there's other compounds that are, kind of, bigger competitors to you? And then I have a quick follow-on.
Richard S. Geary -- Executive Vice President, Development
No, I think you nailed it. That's what -- that's our hurdle to beat and that's what we're aiming for and the design of the study allows us to evaluate that.
Yaron Werber -- Cowen & Company -- Analyst
Yes. And can you -- do you think you can dose it every -- I mean, it sounds like monthly potentially certainly doable, what about less frequently, like every two months? Or is that a stretch based on what you see in PD?
Richard S. Geary -- Executive Vice President, Development
No, the PK/PD actually supports every two months, and then it's just a matter of starting to get some experience with that regimen.
Brett P. Monia -- Chief Executive Officer
And weather -- obviously, weather a bimonthly versus monthly regimen from a marketing standpoint is -- actually represents an advantage for patients. But as Richard said, if we feel it does, we have the ability to move there.
Yaron Werber -- Cowen & Company -- Analyst
Yes. Okay, great. And final question, just moving to GHR-LRx, if I remember correctly, that Phase II data we should see that soon acromegaly, that is looking at Somavert naive patients, right? I don't think you're enrolling failures, correct me if I'm wrong. And then what does that mean for potentially the Phase III program, would it be naives or can you also go for the Somavert failures? Thank you.
Richard S. Geary -- Executive Vice President, Development
Yes. So, the initial studies that we've conducted are on Somavert failures, you could call it that. It's patients, who are on Somavert and uncontrolled bio-chemically. And then we have the second study that actually we'll report out next year in the naive patients. So, we're covering the entire spectrum, starting with the Somavert uncontrolled patients. Did that answer your question?
Yaron Werber -- Cowen & Company -- Analyst
Yes. So, the data right? So, this is going to be the second study and that's in the naive segment. And then based on that, is the thought, you know, assuming based on the prior data, we're expecting positive results from this Phase II. Is the thought then in Phase III to do sort of a head-to-head against Somavert to go to the failures maybe within a single-arm design or go to the naive, I mean, there's sort of two ways you can go there?
Richard S. Geary -- Executive Vice President, Development
Yes, the teams are still working out the designs and the regulatory interactions are yet before us. So I would be I think premature in giving you a design at this point.
Yaron Werber -- Cowen & Company -- Analyst
Okay, great. Thank you.
Brett P. Monia -- Chief Executive Officer
Thanks, Yaron.
Operator
Our next question comes from Vincent Chen with Bernstein. Please go ahead.
Vincent Chen -- Bernstein -- Analyst
Congrats on the progress and thanks for taking my questions. A couple of quick ones on the oral program and the oral delivery of ASOs. The first would simply be, I'm curious how would bio-availability for one of your ligand conjugate ASOs differ from another one, if they're dosed orally? I guess what I'm thinking about is, would you expect bio-availability, it probably looks pretty similar between different LICA drugs or would there be meaningful differences?
And I guess the second related would be, you mentioned earlier you're continuing to do research on new formulations and new chemistries to enhance oral delivery. I was wondering, could you provide some color on what are some of the things you can do on formulation or chemistry to try to boost oral bio-availability?
Brett P. Monia -- Chief Executive Officer
So, I'll start -- and thanks for your questions, Vince. And then I'll pass it on to Richard, who is one of the experts in oral delivery of oligonucleotides anywhere. But legally, yes, we have shown that the oral delivery -- oral bio-availability that we get with our LICA drugs is very similar to our non-LICA drugs. And what was very important to demonstrate was that the -- that bio-availability to be that -- to be comparable to what we've shown previously with non-LICA drugs, but also to be stable and be effective in target reduction in the liver. And we've shown also that this is transferable across several many different antisense drugs with Gen 2.5 chemistry and LICA. So, it is directly transferable.
And Richard, maybe you could talk a little bit about what we expect on first-pass and that sort of thing with the LICA drugs.
Richard S. Geary -- Executive Vice President, Development
Yes, on the LICA drugs, I mean, it really all comes down to potency. And that's why the subcu data that you're going to see is so important, and it starts to give the justification for why a molecule that gives you 10% bio-availability or something like that can be a very clinically relevant dosage formulation. So, it comes down to cost of goods and potency.
You asked the question about would a different LICA of a different chemistry give you different bio-availability, actually across our platform the oligonucleotide bio-availability is fairly comparable across the chemistry types. The difference with the LICA is that you present to the liver first versus the subcu, so you get a bit of a bang for your buck, if you will, by presenting first to the liver and allowing the liver to see the drug at low concentrations first with millions of copies of the GalNAc receptor on the parasites scooping up the drug as it passes through the liver first. So, that gives you that extra over and above what you would get with an unconjugated oligonucleotide, is that helpful?
Brett P. Monia -- Chief Executive Officer
And I would just add to that also the fact that these drugs are so stable and so long-acting allows us to have -- they accumulate in the hepatocyte, so -- but 10% bio-availability for a drug that has a half-life of 24 hours or less is not very commercially viable, because of the fluctuations you're going to get in bio-availability and all kinds of things. But the fact that our drugs are so long-lasting allows that bio-availability to be very significant and very important and justifies oral delivery with our platforms. So, that's a very important thing to also remember.
As far as the work we're doing on new formulations, stay tuned Vince. We have a lot of work in progress, lot of irons in the fire, we're not sharing our strategies there, but obviously we've set -- we've tackled stability with our drugs. So, we're really focused on penetration, getting more drug into the blood.
Vincent Chen -- Bernstein -- Analyst
I see. Well, I'll stay tuned then. Thanks for taking the question. Appreciate the insight, and congrats again on all the progress.
Brett P. Monia -- Chief Executive Officer
Thanks, Vince.
Operator
Our next question comes from Mani Foroohar with SVB Leerink. Please go ahead.
Rick Bienkowski -- SVB Leerink -- Analyst
This is Rick dialing in for Mani. Thanks for taking our questions. First, moving back to ENaC, could you maybe broadly discuss what lessons were learned from the ENaC readout? You know, there seems to have been good target engagement in the lung, so we were wondering what the main takeaways were? And how you're thinking about target selection for any other inhaled ASOs that are may be in development?
Brett P. Monia -- Chief Executive Officer
I would say that the key lessons learned are that our Gen 2.5 chemistry is outperforming our Gen 2 chemistry for aerosol delivery in the lung, based on the potencies that we get with that drug that Richard referred to earlier. It's really the first time we've shown statistically significant and substantial reductions in target with an aerosol antisense oligonucleotide in humans. So, that's a huge lesson learned and we think that gives us confidence that -- in our emerging pulmonary pipeline and overall, we have several other drugs in development for pulmonary diseases.
One of -- at least one of which we expect to reach development in the next year or in development potentially clinical -- well, clinical testing next year or the second drug. So -- and of course, the safety and the tolerability is a very important lesson learned that there is the doses that we're seeing these effects give us confidence that the preclinical data is predicting what we expected to see in humans, very well, which is very important, obviously.
Rick Bienkowski -- SVB Leerink -- Analyst
Got it. Thanks for that. Second question, [Indecipherable] to see if there's any update on the status of recruitment into the Phase III studies for TTR LICA, just are there any trends you've been seeing in enrollment over the last quarter and how you're currently thinking about time to data for polyneuropathy and cardiomyopathy?
Brett P. Monia -- Chief Executive Officer
So we're -- studies are enrolling, sites are being activated for both polyneuropathy and the cardiomyopathy Phase III studies and they're moving well. We've had some impact of COVID-19 in getting sites activated and so on, but overall we're not providing details, it's obviously a competitive situation that we're in and -- but we are pleased. We're pleased with the enrollment that we're seeing so far in both Phase III studies and we like our clinical trial designs, which are -- we think are enhancing enrollment.
There's no changes to what we said publicly on cardiomyopathy data and we're expecting the data to be out from the Phase III study in '24 time frame and the polyneuropathy in '23 time frame for the full data set and we also have an interim analysis baked into the polyneuropathy study. And if we -- and that data is due to come out in 2022.
Rick Bienkowski -- SVB Leerink -- Analyst
All right. Perfect. Thanks for taking our questions.
Brett P. Monia -- Chief Executive Officer
You got it.
Operator
Our next question comes from Yale Jen with Laidlaw & Company. Please go ahead.
Yale Jen -- Laidlaw & Company -- Analyst
Good afternoon, and thanks for taking the questions. The first one is regarding the vupanorsen Phase II -- after the Phase IIb study by Pfizer. What's the -- could you elaborate more in terms of Pfizer's plans for the pivotal study? You mentioned three studies, but maybe a little bit more color on that?
Brett P. Monia -- Chief Executive Officer
Yes. So, perfect pitch for Richard. Knock it out.
Richard S. Geary -- Executive Vice President, Development
Yes. Good question. So, the Phase IIb study is in dyslipidemic patients with a cardiovascular disease, high risk profile and it's in direct preparation for Phase III program that includes a cardiovascular outcome trial. Much will be learned, because these patients are hypertriglyceridemic as well. Much will be learned about dose selection also for the severe hypertriglyceridemic platform. And so, it is a dose range finding study that will setup the finalization of protocols for those two programs and those two populations.
Yale Jen -- Laidlaw & Company -- Analyst
And is there a third one, which is -- you will use the cardiovascular outcome as an endpoint and any colors on that study?
Richard S. Geary -- Executive Vice President, Development
Yes. The cardiovascular outcome trial is one trial and then the severe hypertriglyceridemic population is the -- they've planned a couple of Phase IIIs for that.
Yale Jen -- Laidlaw & Company -- Analyst
Okay, great. That's very helpful. I have one more question. One more question in terms of Akcea acquisition at this point. I know you will give more color at the Investor Day, but any other thoughts at least on the 10,000 foot angle in terms of is there any additional elements or change of elements need to be added after this acquisition?
Brett P. Monia -- Chief Executive Officer
Not really, Yale. We'll talk more about it as mentioned at Investor Day, how this is a key step toward our -- building our commercial strategy for the future. But really, I wouldn't say that there is any additional elements than what we've said today or what we've put out when we announced the closure -- closing of the acquisition. We're very excited about -- we think this is a very important strategic step in the growth of Ionis for many reasons that we've highlighted, that are both financial, as well as based on efficiencies and based on building our commercial plans for the future. So that's a lot right there and we will talk more about it in December. I really can't expand more than that on your question.
Yale Jen -- Laidlaw & Company -- Analyst
Okay, great. Thanks a lot. I really appreciate it. And congrats on the progress.
Brett P. Monia -- Chief Executive Officer
Thanks, Yale.
Operator
Our next question comes from Jessica Fye with J.P. Morgan. Please go ahead.
Jessica Fye -- J.P. Morgan -- Analyst
Hi, guys. Good morning. Thanks for taking my question. Another one on vupanorsen. It looks like the doses being studied in Phase IIb are generally higher than what was studied in Phase IIa. So, thinking about the non -- very high triglyceride population, kind of, the broader high CV risk group. I'm just curious, when you look at those higher doses, how much more efficacy you're hoping to achieve relative to the Phase IIa on endpoints like LDL-C?
Richard S. Geary -- Executive Vice President, Development
Yes. Great question. Really the primary endpoint is non-HDL-C, which is a group of lipid particles that are involved in atherosclerosis. So, the increase is a combination of an increase on triglycerides, increase on VLDL-C and LDL. So, it's not a single component look, it's basically the remnant cholesterol that remains in the circulation in patients with elevated triglycerides and cardiovascular disease. So, I don't know if that answers your question. The -- it's a dose range finding study and the doses are now being tested in patients with dyslipidemia and cardiovascular risk, which is a different population than the population we studied in our Phase IIa study.
Brett P. Monia -- Chief Executive Officer
Yes. I think that's the key, Jess, is that it is a different patient population in the Phase II study. So, Pfizer wants to get the dose right for the Phase III -- for the several Phase III studies that Richard mentioned that they're planning to initiate, because it is a different patient population and the doses could be different and they want an optimal dose. So, going to higher doses is just part of that strategy.
Jessica Fye -- J.P. Morgan -- Analyst
Okay, makes sense. And just, kind of, sticking with this product and recognizing the lack of support for benefit in drugs that increase HDL, what you make of the declines observed with this product in HDL-C?
Brett P. Monia -- Chief Executive Officer
Can we take that, Rich?
Richard S. Geary -- Executive Vice President, Development
Yes. So, we've looked at this very carefully and you know that there is actually a genetic population that has a knockout of ANGPTL3. And in that population, they have extremely low HDL, extremely low LDL and triglycerides and they have no cardiovascular disease and they have a very long -- bunch of very long lived individuals. This is a small population in the hill country of Italy. So, now we're studying this in populations where we're actually knocking it down, not from birth, but in a selected population. So, I guess, what I would say is when we think about HDL, HDL is always -- has to be taken into consideration along with all the atherogenic lipids. What's been shown is that HDL alone does not -- by raising HDL does not apparently improve the cardiovascular outcomes of patients. But what we know is if you take down cholesterol, if you take down remnant cholesterol, then you do improve cardiovascular health of patients.
Jessica Fye -- J.P. Morgan -- Analyst
Got it. Thank you.
Brett P. Monia -- Chief Executive Officer
Thanks, Jess. Maybe we're running long, we'll take one more question and then we'll have to close.
Operator
Our last question comes from Myles Minter with William Blair Company. Please go ahead.
Myles Minter -- William Blair -- Analyst
Hi, thanks for taking the questions. Just maybe one on something we haven't talked about, the TMPRSS6 asset for beta thal. Just wondering, when we're going to see Phase II data next year, whether you've got clarity on that? And just in terms of the primary endpoint, can you maybe explain the clinically -- the clinical meaningfulness of a 1 gram per deciliter increase in hemoglobin in this patient population and not the transfusion-dependent population? Just trying to understand how to interpret that moving forward if this starts to go into a pivotal trial of some sorts? Thanks.
Brett P. Monia -- Chief Executive Officer
Yes. I'll take this and Richard can expand on anything worth highlighting beyond this. So, we're very excited about this program. We think the opportunity in beta thal intermedia, as well as other indications where -- that are plagued by low hepcidin levels are also very relevant for our TMPRSS6 inhibitor. As you know Myles, in Phase I, we not only showed good safety and tolerability, we showed significant increases in hepcidin levels and changes exactly in the direction based on our preclinical data with -- that that data would predict in iron and transferrin saturation.
In the intermedia patient population, there's two goals, right? One is to increase -- is to deal with the anemia and to increase hemoglobin to the extent that you highlighted, which is very meaningful endpoint in these patients, so potential approvable endpoint in the intermedia patient population. But what this drug can also do is reduce iron load in liver and in heart and reduce toxicities associated with iron overload in those organs and other organs as well. So, this is very unique drug potentially that has the ability to not only affect the hemo and improve and normalize potentially the anemia, but also the devastating effects of iron overload in various organs. And we have a wealth of preclinical data published in real good models of beta thalassemia. We're also exploring additional indications for this drug that are related, as I said, to miss -- dysfunctioning -- dysfunctional management of iron due to low hepcidin levels.
Timing for this study, we -- I don't think we've disclosed that today for the Phase II study that's in progress. It is a novel design that we can share data as -- potentially next year from the study, but I don't think we've come out on that yet as to when exactly that data we'll read out, Myles.
Myles Minter -- William Blair -- Analyst
Okay. So, that's helpful. Yes, go ahead.
Onaiza Cadoret-Manier -- Executive Vice President, Chief Corporate Development and Commercial Officer
Hey, Myles. I was just going to add to Brett's point that we just actually completed a market research survey and it shows that just from a clinicians' perspective that the dual benefit of managing both anemia and iron overload with a single agent is very high. So, we've gotten about 85% of clinicians out there that are looking for an iron-controlling product aside from the iron-chelating agent. So, the dual benefit continues to be a really unmet need.
Myles Minter -- William Blair -- Analyst
Understood. Last quick one from me, and I may be wrong here, but is there any reason why for the chronic hepatitis B program that GSK is running why they would have taken a non-market conjugated products in a Phase II as opposed to a LICA product?
Brett P. Monia -- Chief Executive Officer
Eric? Please go ahead.
Eric E. Swayze -- Executive Vice President, Research
Yes. I mean, the reason there is that the reductions in the antigens that were shown in the trial were not really different with the LICA compound, and that the parent molecule was actually performing very well. And then the next question you'll ask me is, why that is? And the answer is truthfully, we really don't know why that is, it's one of the -- it's the only program where we haven't seen that. But the specifics of that program is the parent molecule is performing better than the LICA and the LICA didn't give an advantage.
Brett P. Monia -- Chief Executive Officer
Yes. This is the only LICA where we have not seen a significant increase in potency with a LICA versus a non-LICA, which we're trying to understand it. But GSK is very excited about the drug that they licensed from us, as we are too. And I think that this is a potential -- has the potential for HBV cure in future, Myles.
Myles Minter -- William Blair -- Analyst
Fair enough. Thanks for squeezing me in. I appreciate it.
Brett P. Monia -- Chief Executive Officer
Thank you, Myles, and thank you, everybody. So, with that, I want to thank you everybody for participating on today's call. Remind you again of our upcoming Investor Day on December 7th. We're really excited about some of the progress. A lot of the progress that we're making will be shared in more detail. And then finally, wish all you a great day. Thank you.
Operator
[Operator Closing Remarks]
Duration: 77 minutes
Call participants:
D. Wade Walke -- Vice President, Investor Relations
Brett P. Monia -- Chief Executive Officer
Elizabeth L. Hougen -- Executive Vice President, Finance and Chief Financial Officer
Onaiza Cadoret-Manier -- Executive Vice President, Chief Corporate Development and Commercial Officer
Richard S. Geary -- Executive Vice President, Development
Eric E. Swayze -- Executive Vice President, Research
Do Kim -- BMO Capital Markets Corp. -- Analyst
Gil Blum -- Needham & Company -- Analyst
Jim Birchenough -- Wells Fargo -- Analyst
Joel Beatty -- Citigroup -- Analyst
Chi Fong -- Bank of America Merrill Lynch -- Analyst
Luca Issi -- RBC Capital Markets -- Analyst
Katie Schneeberger -- Stifel -- Analyst
Yaron Werber -- Cowen & Company -- Analyst
Vincent Chen -- Bernstein -- Analyst
Rick Bienkowski -- SVB Leerink -- Analyst
Yale Jen -- Laidlaw & Company -- Analyst
Jessica Fye -- J.P. Morgan -- Analyst
Myles Minter -- William Blair -- Analyst
