Stanley Crooke - Founder, Executive Chairman, CEO and President Wade Walke - VP of Corporate Communications and IR Lynne Parshall - COO and Director Elizabeth Hougen - CFO and SVP of Finance.

Eric Schmidt - Cowen and Company Yale I-Eh Jen - Laidlaw & Company Stephen Willey - Stifel, Nicolaus & Company Jessica Fye - JPMorgan Chase & Co Benjamin Burnett - Leerink Partners.

Welcome to Ionis Pharmaceuticals First Quarter 2017 Financial Results Conference Call. As a reminder, this call is being recorded. Leading the call today from Ionis is Dr. Stan Crooke, Ionis' Chairman and CEO. Dr. Crooke, please begin..

Good morning and thank you for joining us on today's conference call to discuss our first quarter 2017 financial results and business highlights. 2017 is off to a great start. Biogen reported sales of SPINRAZA of over $47 million in the first quarter, significantly exceeding consensus estimates.

Biogen received a positive CHMP opinion for SPINRAZA, recommending a broad indication, puts the drug on track for approval in the EU shortly. We received $75 million from Bayer to advance both IONIS-FXIRx and its LICA follow-on, IONIS-FXI-LRx. Importantly, this expands the opportunity for our FXI franchise (inaudible) forward.

And the collaboration with Novartis worth up to more than $1.6 billion plus royalties help in commercialize new AKCEA drugs for large indications. As part of this collaboration, AKCEA initiated a Phase IIb dose-ranging study for APO(a)-LRx in patients with high Lp(a).

We reported positive Phase III data from the volanesorsen APPROACH study in patients with FCS. And we continued our strong financial performance, ending the first quarter with a pro forma operating income of $35 million and pro forma net income of $24 million.

With the SPINRAZA launch gaining momentum, volanesorsen moving toward the market, Phase III data for IONIS-TTRRx imminent, a large and growing pipeline supported by our efficient technology platform, we believe we have the elements in place to achieve sustained, long term financial growth.

Joining me in today's call are Lynne Parshall, Operating Officer; Beth Hougen, Chief Financial Officer; Sarah Boyce, Chief Business Officer; Paula Soteropoulos, Chief Executive Officer of Akcea; Wade Walke, Vice President of Corporate Communications, Investor Relations.

And now Wade, will you please read our forward-looking language statement?.

Thanks, Dan. A reminder to everyone that this conference call includes forward-looking statements regarding financial outlook for Ionis, Ionis' business, the business of Akcea Therapeutics and the therapeutic and commercial potential of Ionis' technology and products in development.

Any statement describing Ionis' goals, expectations, financial or other projections, intentions or belief, including the commercial potential of SPINRAZA, IONIS-TTRRx and volanesorsen is a forward-looking statement and should be considered an at-risk statement.

Such statement is subject to certain risks and uncertainties, particularly those inherent in the process of discovery, developing and commercializing drugs that are safe and effective for use as human therapeutics and any endeavor of building a business around such drugs.

Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements.

Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements.

These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2016 which is on file with the SEC. Copies of this and other documents are available from the company. And now I'd like to turn the call over to Lynne..

Thank you, Wade and good morning. SPINRAZA has brought a life-changing therapy to patients with SMA, their families and the physicians who treat them. We're proud to have brought this therapy to patients who need it. We continue to be pleased with the dedication, passion and commitment of our partners at Biogen.

SPINRAZA sales for the quarter totaled over $47 million and we earned over $5 million from these sales. Patient demand is high and physicians are motivated to prescribe SPINRAZA which are both important factors as the launch builds momentum. We believe this strong start sets up for continued growth in the SPINRAZA launch trajectory.

Biogen has already been successful in streamlining access to treatment at the 40 to 50 SMA centers in the United States, with additional U.S. centers beyond these specialty centers now treating patients. As of April 21, there were 88 sites across 36 states administering SPINRAZA and 203 sites had submitted start forms.

Additionally, Biogen reported coverage on more than 165 insurance plans, including commercial and Medicaid plans as of April 21. Biogen estimates that 75% of commercially insured patients are covered by a plan that has an established policy for SPINRAZA, half of which have a policy with broad access.

And today, while the majority of patients treated have been Type 1, Biogen continues to seek coverage approvals for patients with Type 2 and Type 3 SMA. And many patients covered by plans without stated coverage or with policies restricted to Type 1 SMA have, nevertheless, been approved for reimbursement.

At the end of April, Biogen received a positive CHMP opinion for SPINRAZA, recommending approval with a broad label in the EU. We're looking forward for approval in the EU shortly. Biogen also expects approval in Canada and Japan this year.

Biogen has submitted regulatory filings for SPINRAZA in Australia and Switzerland and anticipates filings in additional countries this year. New data were presented last month at the AAN Annual Meeting that continued its support of robust efficacy and safety profile of SPINRAZA across a broad range of patients with SMA.

Data from the end of study analysis of the CHERISH study in children with later-onset SMA demonstrated a highly statistically significant and clinically meaningful improvement in motor function scores in SPINRAZA-treated patients compared to untreated patients.

Let me explain what it means for patients when we talk about motor function and milestone scores. When a child improves by a point or 2 on the Hammersmith Motor Function Scale, it means that child may not be able to do something as simple as brush her hair, raise his hand or climb a curb on a walk to school with a friend.

SPINRAZA's real impact is demonstrated in these simple but profound achievements. In the CHERISH study, children treated with SPINRAZA achieved on average nearly 4 points improvement from baseline on the Hammersmith motor function score at month 15, leading to unprecedented improvement such as crawling and standing with assistance.

By comparison, untreated children declined by an average of 1 point over the same period. Biogen also presented new interim data at AAN from the NURTURE study in presymptomatic infants with genetically diagnosed SMA. At the time of the interim analysis, all infants were alive without the need for permanent ventilation.

Further, most of the infants in the study achieved new motor milestones and growth parameter gains. What's important to note is that SPINRAZA-treated infants achieved these milestones on essentially the same time line as would be expected of a healthy infant.

The NURTURE data further demonstrates the value of initiating treatment with SPINRAZA as early as possible. We also achieved successes beyond SPINRAZA in the first quarter.

In January, we and Akcea entered into a strategic collaboration and option agreement with Novartis to develop and commercialize 2 of Akcea's drugs, AKCEA-APO(a)-L Rx and AKCEA-APOCIII-L Rx. This collaboration has a potential value of up to more than $1.6 billion plus royalties up to the low 20% range.

We have received $175 million from Novartis already.

By pairing Novartis' expertise and global resources with Akcea's specialized sales force focused on lipid disorders, we believe this collaboration will maximize the commercial potential of each drug to treat the millions of patients who suffer from cardiovascular disease because of their high Lp(a) or high triglycerides.

As the next step in this collaboration, in March, Akcea started a Phase IIb dose-ranging study of AKCEA-APO(a)-L Rx in patients with high Lp(a) to determine the dosing regimen for the planned Phase III outcome study.

Later this year, Akcea plans to initiate a Phase IIb dose-ranging study for AKCEA-APOCIII-L Rx to determine the dose and regimen for the planned Phase III outcome study for that drug.

Novartis will be responsible for globally developing and commercially launching each drug, including conducting and paying for the cardiovascular outcome studies, assuming Novartis exercises its option to license each drug.

Importantly, Akcea has no obligation to participate in the funding of these outcome studies and yet Akcea has the right to co-commercialize any drug Novartis licenses through the specialized sales force it is building to commercialize volanesorsen.

Late last year, we reported positive results from an important study of IONIS-FXIRx in patients with end-stage renal disease on dialysis that showed that the drug worked and was well tolerated. Then in the first quarter, we announced that our partner, Bayer, paid us $75 million to advance both IONIS-FXIRx and the LICA drug, IONIS-FXI-LRx.

This is important because it allows us to continue advancing the parent drug while expanding the collaboration by moving forward with the LICA drug which has a potential for a broader indication.

In the second half of 2017, we plan to initiate a Phase IIb study evaluating IONIS-FXIRx in patients with end-stage renal disease to optimize the design of the Phase III program. And we plan to initiate a Phase I study of IONIS-FXI-LRx early next year.

After these studies, Bayer will be responsible for globally developing and commercializing these drugs. In March, we and Akcea announced positive results from the Phase III APPROACH study of volanesorsen in patients with FCS.

FCS is a severe rare disease affecting approximately 3,000 to 5,000 patients worldwide, characterized by extremely high levels of triglycerides. These extremely high triglycerides result in multiple daily and chronic manifestations such as recurrent abdominal pain and acute and potentially life-threatening pancreatitis attacks.

Patients also experience emotional and cognitive symptoms such as memory impairment and what patients describe as brain fog. Today, FCS patients have no effective treatment for their disease.

We believe that volanesorsen has the potential to be the first treatment option for patients suffering from the daily burden as well as the life-threatening risks of FCS.

In the Phase III APPROACH study, volanesorsen achieved its primary endpoint, resulting in profound nearly 80% reductions in triglycerides with an absolute reduction of triglycerides of more than 1,700 milligrams per deciliter. The triglyceride-lowering effects we observed will maintain throughout the 12-month study period.

Importantly, volanesorsen also reduced the instance of pancreatitis attacks and the frequency and severity of abdominal pain. These top line data were presented last month at the Annual European Atherosclerosis Society Meeting. We identified one safety issue during the Phase III program. Serious thrombocytopenia was observed in 3 patients.

Each of these patients' platelet levels recovered following cessation of dosing. We now know that FCS patients are prone to greater-than-normal fluctuations in platelet levels and that volanesorsen can contribute to the thrombocytopenia seen in some of these patients.

Once we identified the potential for serious thrombocytopenia in FCS patients, we implemented more frequent platelet monitoring which allowed us to better manage this issue in patients. Routine platelet monitoring will be part of our patient-centric commercial strategy.

Platelet monitoring is simple and can be performed by any lab and we're exploring various ways to make this monitoring as convenient as possible for patients.

We believe, based on its overall efficacy and safety data, that volanesorsen has demonstrated a favorable risk benefit profile in patients with FCS who suffer every day from a very severe and life-threatening disease. Akcea, in collaboration with Ionis, is well along in preparing the final marketing applications for volanesorsen in the third quarter.

It's also well along in putting in place the commercial infrastructure to be ready to launch volanesorsen next year. Akcea is building an integrated commercial team which is planned to eventually be about 75 to 100 people in the U.S., EU and Canada. FCS is a terrible orphan disease that causes daily symptoms and shortens life expectancy.

However, as with many orphan diseases with no treatment options, FCS is under-diagnosed and poorly understood. In preparation for an effective launch, Akcea is working to raise awareness of FCS, identify patients, improve diagnosis and quantify the burden of this disease.

One way Akcea is doing this is by building a database of FCS patients through communication with physicians and patient organizations and using tools such as electronic medical record database searches.

By employing a simple search algorithm for patients with persistent high triglycerides, a history of pancreatitis and ruling out other potential causes, Akcea has been able to systematically work with centers and integrated health care systems to identify additional FCS patients.

Akcea's work to understand and quantify the burden of FCS has established them as leaders in understanding FCS. An example of this work is the recently published interim results for the IN-FOCU.S. survey which Akcea commissioned, to capture the holistic burden of disease for patients, including physical, cognitive and psychosocial burdens.

Understanding this disease is one of the best ways Akcea can prepare for an effective launch. Data from this survey will also be important for regulators, payers and health care professional. Later this month, 8 abstracts discussing volanesorsen, Akcea's disease burden and results from the IN-FOCU.S.

survey will be presented at the National Lipid Association Conference. In March, we filed a registration statement for Akcea with a plan to launch an IPO. Some have asked us why we're taking this step and why now.

First, let me explain what this means for both Ionis and Akcea and why we feel taking Akcea public represents the natural next step in Akcea's maturations, then I'll explain why we think now is the ideal time to take Akcea public. Ionis was established to create and advance antisense technology as a new drug discovery platform.

Our success to date from this endeavor has resulted in the creation of one of the largest pipelines in the industry. One of the keys to our success has been our unwavering focus on innovation in the field of RNA therapeutics.

As our business model has naturally evolved and our technology platform and pipeline have advanced, our success has resulted in a strong financial position.

As such, we're now able to retain a larger portion of the commercial value from our drugs in a number of ways, including develop our -- developing our drugs longer before partnering and creating subsidiaries like Akcea to develop and commercialize additional drugs in our pipeline.

We formed Akcea to maximize the potential of 4 complementary drugs for the treatment of cardiometabolic disease which we believe has the potential to create more value when developing and commercializing one synergistic pipeline than at separate programs.

The structure allows us to provide greater input and influence over the future of our drugs and continue to add our expertise and resources to these programs. We're able to do this without losing Ionis' intense focus on advancing antisense technology, discovering new medicines and advancing our pipeline.

Forming a separate company, as we did with Akcea, enabled us to handpick leaders to create a high-quality and expert organization which is focused and incentivized to maximize the commercial potential of the drugs in its pipeline.

For Akcea, we envision the team with the same entrepreneurial spirit and sense of mission we have at Ionis, but focused on Akcea's unique opportunities. We think this focused effort assures that Akcea will be as expert in the treatment of cardiometabolic lipid disorders as the Ionis team is in the antisense technology.

Structuring Akcea as an independent company allows us to attract a team that possesses these traits and locating Akcea in Cambridge allows us to take advantage of that area's strong pharmaceutical community.

The Akcea organization, headed by seasoned senior leaders, Paula Soteropoulos, Jeff Goldberg and Louis O'Dea, embodies the team we hoped to create.

Further, the structure enabled Akcea to recruit an independent Board of Directors made up of industry leaders with extensive experience perfectly tailored to Akcea's specific needs, including global commercialization. Given the maturity and richness of Akcea's pipeline, we believe that this is the right time to launch an IPO for Akcea.

Akcea now has positive Phase III data for volanesorsen in hand, a near term commercial opportunity with volanesorsen and a second indication in Phase III, a pipeline of 4 drugs to approach the next lipid cardiovascular risk factors, a strategic partnership with Novartis to support large indications for 2 of Akcea's drugs, the foundational support of Ionis and experienced leadership team and an independent Board of Directors with deep, relevant experience.

So just to recap before I turn the call over to Beth. We believe the successes we've achieved, coupled with commercial revenue from SPINRAZA and a growing revenue stream from our partners, represent the creation of real value and position us well for the future.

The success of SPINRAZA, along with the continued progress towards commercialization of volanesorsen and IONIS-TTRRx, should further enable our transition to a multiproduct profitable organization, delivering important medicines to patients with serious illnesses. And now I'll turn the call over to Beth..

Thank you, Lynne. In the first quarter, we continued our strong financial performance from 2016 and made further progress toward our goal of achieving sustained long term financial growth. We ended the first quarter with pro forma operating income of $35 million and pro forma net income of $24 million.

We also reported GAAP operating income and net income. With the approval of SPINRAZA late last year, Q1 was the first full quarter in which we added commercial revenues to our substantial base of R&D revenue. The launch of SPINRAZA is off to a strong start.

And as a result, we recognized $5.2 million of commercial revenue from SPINRAZA royalties in the first quarter. The addition of commercial revenue from SPINRAZA builds on the $324 million in R&D revenue we have already received from Biogen and the $90 million in approval milestone payments we anticipate receiving upon approval in the EU and Japan.

We're pleased with SPINRAZA's early launch results in the U.S. and the potential for future growth not only in the U.S., but also in other markets. Importantly, we're tiered royalties on SPINRAZA sales. This means that as sales of SPINRAZA grow, so will our portion of those sales.

In contrast, the nominal third-party royalties we pay are fixed, not tiered. This means that as SPINRAZA's sales increase, our profit margin on those sales will increase. The addition of commercial revenue is a reflection of the evolution of our business and our progress towards sustained profitability.

Our financial statements now reflect this progress. You'll notice that we have modified the revenue section of our P&L to break out our commercial revenues from our R&D revenues. In addition, we've renamed the G&A line of our P&L to SG&A to better reflect the character of our expenses.

Our strong financial results were driven by the more than $110 million of revenue we earned in the quarter, an increase of more than 200% over the same period in 2016. In addition to commercial revenue for SPINRAZA, we earned $102 million in R&D revenue, primarily due to the expansion of our Bayer agreement and our collaboration with Novartis.

We generated $75 million in Bayer advance both IONIS-FXIRx and IONIS-FXI-LRx. And we recognized more than $65 million of the $75 million payment as revenue in the first quarter. We will amortize the remainder as we perform the development activities for these 2 drugs. In addition, we received $175 million from our collaboration with Novartis.

Under this collaboration, Novartis paid us a $75 million upfront payment and made a $100 million equity investment in Ionis at a substantial premium. The premium represents additional revenue under the collaboration.

Therefore, we will amortize the $75 million upfront payment and the premium which is $33 million, into revenue ratably as we conduct the Phase IIb studies for AKCEA-APO(a)-L Rx and AKCEA-APOCIII-L Rx. Our pro forma operating expenses for the first quarter were $75 million.

And as we anticipated, our first quarter expenses were essentially flat compared to the same quarter in 2016. During the remainder of 2017, we're forecasting an increase in our SG&A expenses as Akcea continues to prepare to launch volanesorsen in 2018. And we expect our R&D expenses to be lower as we concluded our costly Phase III program.

Because of the efficiency of our technology, even with climbing R&D expenses this year, we will continue to advance our earlier-stage drugs and add new drugs to our pipeline. We ended the first quarter with more than $860 million in cash which is nearly a 30% increase over our cash at the end of last year.

The increase was primarily from the $175 million we received from our collaboration with Novartis and the more than $100 million that we earned late last year and received in the first quarter of this year.

We're on track to meet our 2017 financial guidance of being breakeven or profitable at the operating line on a pro forma basis and a cash balance of more than $825 million. As the year goes on and we have more visibility on SPINRAZA sales, we plan to provide more specific guidance.

Beyond 2017, we look forward to adding to our substantial base of R&D revenue with growing commercial revenue for SPINRAZA royalty and the addition of volanesorsen product sales and IONIS-TTRRx royalty next year. We believe that we have the key elements in place to achieve sustained, long term financial growth.

The combination of multiple drivers of revenue, our efficient technology and our partnering strategy that maximizes the near- and long term potential of our drugs and supports prudent management of our expenses positions us well for continued financial strength. And now I'll turn the call over to Stan..

Thanks, Beth. Just one point to clarify a bit. Beth referred to $324 million in payments we received. Those are $324 million specific to SPINRAZA. Of course, we received substantially greater funds -- payments from Biogen for the entire sets of collaboration.

So SPINRAZA has given patients and families benefiting -- or suffering from SMA a life-changing therapy. This is the most important of the many reasons we're excited about SPINRAZA. Clearly, with first quarter sales of over $47 million, the launch is off to a strong start. We're confident that SPINRAZA has blockbuster potential.

Moving ahead to the remainder of 2017 and into 2018, we have multiple near term commercial opportunities and key upcoming date events. Moving on to strong initial sales of SPINRAZA. We look forward to continued expansion of the SPINRAZA launch in the U.S.

With a positive CHMP opinion received for SPINRAZA recommending a broad indication, we believe that SPINRAZA is on track for a rapid approval and launch in the EU, further expanding SPINRAZA's near term commercial potential. Biogen also expects approval in Canada and Japan this year.

Akcea plans to submit regulatory application for volanesorsen in the U.S., EU and Canada in the third quarter, preparing the launch of volanesorsen in 2018. We plan to report pivotal Phase III data from the NEURO-TTR study of IONIS-TTRRx this quarter. We and GSK are hard at work at writing the regulatory applications to file by the end of this year.

Later this year, we plan to provide updates on IONIS-TTRRx currently in a Phase I study. IONIS-HTTRx is the first potentially disease-modifying drug to be evaluated in patients with Huntington's disease. We also plan to report additional clinical results for multiple programs as the year progresses.

We plan to initiate studies on several novel drugs throughout the year, including Phase IIb studies on FXIRx and AKCEA-APOCIII-L Rx and Phase I studies on IONIS-TMPRSS6-LRx in patients with beta thalassemia, IONIS-MAPT Rx and anti-tol antisense drug in patients with Alzheimer's disease, partner with Biogen.

2017 will continue to see our pipeline advance and deepen in both severe rare diseases as well as in diseases that treat -- with larger patient populations. We also plan to extend the pipeline by addressing new diseases such as beta thalassemia.

The addition of commercial revenues from SPINRAZA royalties, coupled with our strong and growing base of R&D revenue, we're in the strongest financial position in the company's history. Well positioned to achieve our goal of sustained long term financial growth.

Both are drivers of revenue and efficient technology and a strategy that maximizes near- and long term potential of our drugs. We're now closer than ever to our goal of becoming a profitable multiproduct company delivering first-in-class and best-in-class medicines in patients with serious diseases.

And now with that, Kate, if you can set us up for our question and answers, please..

[Operator Instructions]. The first question comes from Eric Schmidt of Cowen and Company..

Maybe on SPINRAZA. I know that Lynne mentioned that patients can get broader access if they have Type 2 or 3 disease.

But in the weeks since the CHERISH data were presented at AAN, have you seen any payers change their blanket policy coverages?.

Lynne, do you want to answer that?.

Eric, there have been some. It's probably a better question to ask -- because these things, obviously, are changing on a day-to-day basis, it's probably a better question to ask Biogen. But Biogen is making all those data available to payers and we do expect that to be a successful offer..

Okay, that's great. Maybe I can just sneak in a quick science question perhaps for Stan. I think there was an article just last week and I can't remember now if it's nature or science on CAG repeats and the potential for the wild type repeat to actually be beneficial to protein trafficking targeting for destruction.

I just wondered if you have a view on that. I think some of your competitors have suggested that the Ionis targeting compound only going after -- well, I guess, going after indiscriminate wild type and mutant repeats might be disadvantaged relative to others in the field..

That's a really complicated question. And I think to some extent, it will depend on CAG repeats and the context of the CAG repeats and the biology that's involved. With regard to HTT, we're confident that we look carefully at the role of HTT normal and mutant.

We're looking at reduction of mutant HTT in our studies and so we're optimistic that, that drug is going to bring significant benefit as we move forward..

We'll move on to the next question. Yale Jen from Laidlaw..

From the NURTURE study that -- on presymptomatic SMA patients, you have a very encouraging data. I just wonder whether you will start to see patients of -- presymptomatic ones that are seeking for treatments.

And how would you see the potentials or reimbursement landscape for this one given the drug initially was approved for all symptomatic patients?.

We certainly do think that it's appropriate to treat SMA patients as early as possible. And we would expect, given the data, that others, including payers, would agree I think for the specifics of what's going on right now. I would recommend that you ask Biogen more details..

Maybe just one more question for volanesorsen. That you're also currently running a Phase III study for FPL. I know you had -- may anticipate to complete patient enrollment toward the end of this year.

But so far, do you have any sort of update in terms of that study? And any colors on that?.

Well, the study is obviously a blinded Phase III study, so I really can't disclose -- I don't have any information to disclose about the performance of the drug in the study other than to say the study's going well and safety and tolerability appear to be effective..

And would that be -- the recruitment was to anticipate to complete toward the end of the year or early next year?.

We hope so. Recruitment is moving along. It -- there are very substantial protocol requirements. So again, I think we're on track and we believe we'll be able to meet our recruitment goals. But I think you'll just have to stay tuned and we'll keep you posted as the study progresses..

And the next question comes from Stephen Willey of Stifel..

Just a question on the SOD1 program. Can you maybe give us a little bit of an indication as to when we might see some initial data coming out of this? And I guess, just given the FDA's recent regulatory activity on an ex U.S.

product kind of fast-tracking approval in the states, just curious if there's any intention, perhaps, on either your or Biogen's part to maybe seek some regulatory guidance as that data starts to come in..

Steve, I don't remember exactly when we think we'll have data on SOD1.

Lynne, do you happen to remember?.

Yes. So we expect to have data next year on that. And we and importantly, Biogen have a very strong relationship with this division and would certainly expect to have an ongoing dialogue with them about this and our other neuro programs..

Remember that we had some encouraging data with an earlier SOD1 that we had, so this is a program that we're hopeful and optimistic about..

And then just a follow-up on the cardiorenal collaboration with AstraZeneca. I think there's been one target identified out of that to date.

Should we anticipate potentially hearing about another target by the end of this year?.

Yes. I think things are moving along in that collaboration very well..

The next question comes from Jessica Fye of JPMorgan..

Two quick ones. So I'm wondering if you can refine when in the quarter we'll see the NEURO-TTR top line and whether or not we should expect the press release to call out results from the cardiac subgroup. And then separately on volanesorsen.

As you prepare that filing, can you comment on whether you expect an AdCom for that product?.

No, no and no comment. No, we can't provide more precise timing and we'll provide the top line data once we have them at an appropriate level of detail as we discuss all that with our partner. And that's really all I can say today. And I think it's too early to speculate about whether there will be an advisory panel for volanesorsen or not.

I think the next step is get our dossier together, meet with regulatory agents -- agencies, as we have been doing in Europe and will be doing in the U.S. and then go from there. Sorry, I can't give more -- provide better information, but that's what I can say today..

That's fine. Just to clarify the question on whether we'll get the cardiac subgroup, I mean, kind of detail in the top line press release. Was that a definitive no, we will not or no comment? So maybe....

A highly definitive no comment..

The next question comes from Paul Matteis of Leerink Partners..

This is Ben Burnett in for Paul Matteis. I wanted to ask about the Huntington's program. I guess just maybe some clarification on the time line that we can expect to get an update from this program.

And also, I guess, what kinds of data do you -- can we expect from this update? And maybe just any color as to what you would view sort of as success here or maybe a certain percentage of knockdown.

Or I guess, how should we be thinking about success here?.

Yes. We're measuring as a primary indicator of whether we have the correct dose reduction of Huntington's proteins in CSF. And we will want to see a meaningful reduction of that protein. We're also looking at other measures, but that's the key measure.

And I think we said that we hope to have information about this by the end of the year or early next year. I think that's what we said.

Is that right, Wade?.

That's correct..

Yes..

We remain encouraged by what we're seeing in the clinical trial of -- in terms of tolerability and safety and are looking forward to working with our partners at Roche over the rest of the way..

This concludes the question-and-answer session. I would like to turn the conference back over to Dr. Crooke for any closing remarks..

If there are no further questions, I want to thank everyone for their interest and attention. And we think 2017 is off to a strong start financially and in all the other ways and look forward to keeping you posted on the progress that we make as the rest of the year unfolds. Thank you..

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect..