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Edited Transcript of AKCA.OQ earnings conference call or presentation 5-May-20 8:30pm GMT

Q1 2020 Akcea Therapeutics Inc Earnings Call

CAMBRIDGE Jun 13, 2020 (Thomson StreetEvents) -- Edited Transcript of Akcea Therapeutics Inc earnings conference call or presentation Tuesday, May 5, 2020 at 8:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Alex G. Howarth

Akcea Therapeutics, Inc. - COO

* Angelyn Lowe

Akcea Therapeutics, Inc. - Executive Director of Corporate Communications & IR

* Damien McDevitt

Akcea Therapeutics, Inc. - CEO & Director

* Kyle Jenne

Akcea Therapeutics, Inc. - Chief Commercial Officer

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Conference Call Participants

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* Chad Jason Messer

Needham & Company, LLC, Research Division - Senior Analyst

* Nathaniel Tower

Stifel, Nicolaus & Company, Incorporated, Research Division - Associate

* Yanan Zhu

Wells Fargo Securities, LLC, Research Division - Associate Analyst

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Presentation

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Operator [1]

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Good afternoon and welcome to the Akcea Therapeutics, Inc. First Quarter 2020 Conference Call. As a reminder, this call is being recorded.

I will now turn the call over to Angelyn Lowe, Akcea's Executive Director of Corporate Communications and Investor Relations. Ms. Lowe, please begin.

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Angelyn Lowe, Akcea Therapeutics, Inc. - Executive Director of Corporate Communications & IR [2]

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Thank you, Jason. Hello, everyone. Thank you for joining today's call. With me today are Damien McDevitt, our Chief Executive Officer; Kyle Jenne, our Chief Commercial Officer; and Alex Howarth, our Chief Operating Officer.

As a reminder, this conference call includes forward-looking statements regarding the financial outlook for Akcea, Akcea's businesses, and the therapeutic and commercial potential of Akcea's products in development. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of TEGSEDI, WAYLIVRA and our pipeline medicines is a forward-looking statement and should be considered an at-risk statement.

Such statements are subject to certain risks and uncertainties, including those related to the impact COVID-19 could have on our business, including, but not limited to, the impact on our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics and in the endeavor of building a business around such medicines. Akcea's 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 Akcea's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements.

These and other risks concerning Akcea's programs are described in additional detail in Akcea's most recent quarterly report on Form 10-Q and in the most recent annual report on Form 10-K on file with the SEC. Copies of these and other documents are available from the company. In addition, earlier today, we issued a press release and related financial tables, including a reconciliation of GAAP to our reported non-GAAP financial measures that we will discuss today. To read this release and access the slides that accompany today's call, please visit the Investors section of our website.

Now I'll turn the call over to Damien.

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [3]

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Thank you, Angelyn. Good afternoon, everyone, and thank you for joining today's call. We had a strong start to the year as we continue to advance our mission of delivering transformative treatments to people living with serious and rare diseases. We ended the first quarter with $421 million of cash and short-term investments on the balance sheet, putting us in a very strong position to execute on our strategic priorities in 2020 and beyond.

Our strategy is to successfully execute on our 2 commercial launches and to advance the transformational programs that we currently have in development, while also adding additional rare disease medicines to our pipeline, both from Ionis and from third parties. In addition, we will partner certain medicines that have potential to treat broader disease indications in order to bring our medicines to more patients faster. This gives us the ability to reinvest a portion of the capital from our broader disease partnerships into our core rare disease business.

To successfully execute on our strategy, it is critical that we have a strong team in place. In the first quarter, we continued to add to and build upon the strengths of the leadership team and Board of Directors. We welcome Dr. Amber Salzman as our newest board member, and as you know, in late March, I was appointed Chief Executive Officer by our Board of Directors. Since joining the company as Interim CEO back in September, I have seen firsthand the outstanding capabilities of the Akcea team in advancing our development and commercialization strategies as well as our unsurpassed level of commitments to making a positive difference for patients affected by serious and rare diseases. I'm very honored to serve this organization's both talented people and innovative therapies. I look forward to continuing to build upon the Akcea's successes as we plan for many important positive developments in the years ahead.

In the first quarter, we continued to make great progress with TEGSEDI and WAYLIVRA. TEGSEDI is commercially available in 12 countries, and WAYLIVRA is approved in Europe and is commercially available in Germany and is now available in Austria. We are making excellent progress in expanding into new countries and expect these new countries to enable revenue growth. Kyle will talk more about the progress we're making with TEGSEDI and WAYLIVRA later on the call.

We're delighted that AKCEA-APO(a)-LRx was recently granted Fast Track Designation by the U.S. Food and Drug Administration as the potential treatment for people at significant risk for cardiovascular disease due to elevated levels of lipoprotein(a). Novartis continues to make great progress with this program. And earlier in the quarter, we reported positive Phase II results for both AKCEA-APOCIII-LRx and AKCEA-ANGPTL3-LRx, which we're now referring to as vupanorsen. With the completion of these 2 studies, we now have data for more than 1,100 patients treated in the clinical setting with LICA medicines, hundreds of which are from the Akcea pipeline. Of those 1,100, a substantial number of patients have been treated for 1 year. As expected, we are seeing consistent positive results across the LICA platform. Our approved medicines are subcutaneous self-injections that are ideal for treatment at home. This is one of our many advantages, and we've seen it magnified in the wake of the COVID-19 pandemic. Thankfully, our patients have been able to stay on therapy while following recommendations from WHO and CDC. It is important to us that our patients who rely on our medicines during this time can obtain them, and we have several programs in place to make sure that happens quickly and seamlessly.

At the present time, many businesses around the world are dealing with adjusting to the recent coronavirus pandemic. We took immediate action to mitigate risks to our business. We've quickly adjusted to the new normal as our workforce continues to work remotely. It's still relatively early, and the situation remains fluid. That said, we have seen a little bit of impact to our business. And we'll discuss that later on the call.

Looking ahead, we are excited about a number of upcoming milestones. We anticipate additional WAYLIVRA and TEGSEDI country launches throughout the year. While WAYLIVRA in the U.S., we are on track to refile with FDA this year, and we look forward to providing an update on this in the coming months. From the pipeline, we anticipate sharing a more detailed development plans for AKCEA-APOCIII-LRx as we prepare to initiate the Phase III study for FCS later this year. We will also be presenting the Phase II data for both AKCEA-APOCIII-LRx and vupanorsen at future medical conferences. Furthermore, we continue to closely collaborate with Ionis to identify and potentially license a novel rare disease medicine from their wholly owned pipeline and to search for potential third-party medicines that complement our rare diseases focus and capabilities.

With that, I'll now turn the call over to Kyle to discuss our commercial efforts in more detail.

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [4]

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Thank you, Damien, and good afternoon, everyone. We continue to make steady progress with our 2 commercial products, TEGSEDI and WAYLIVRA, and we are seeing positive results. Total product revenue was $15 million in the first quarter, an increase of 8% from the fourth quarter of 2019 and a continuation of the sequential growth that we saw throughout 2019. We are competing effectively in countries where we have launched, and we are progressing with pricing and reimbursement negotiations in additional countries for both TEGSEDI and WAYLIVRA. We have the foundation in place for both commercial products as we look ahead in 2020.

Right now a challenge that all companies face is COVID-19. Like most companies, we are working to determine the impact the crisis will have on our commercial business. The impacts that we have been experiencing have been anticipated and managed effectively by our team, including limited face-to-face HCP engagements, challenges for patients to see their health care professional and delays in diagnosis. To date, COVID-19 has not impacted our ability to fulfill patient demand for our products. Our industry-leading patient support program, Akcea Connect, has continued without interruption during the pandemic as nurse case managers have been conducting audio and video calls with patients for disease and drug education. We continue to assess the impact of the crisis on our HCPs, our patients and our business, and we will adjust our strategy and approach as the situation unfolds.

Regarding TEGSEDI, we demonstrated continued growth from Q4 to Q1. We consistently hear from patients and doctors that they prefer a solution for treatment at home, and we believe that the ability of TEGSEDI to provide an at-home subcutaneous self-injection continues to be the driver of TEGSEDI's growth.

Let's now turn to our progress with TEGSEDI in the U.S., where there is positive momentum. The hATTR compass genetic testing program now has more than 1,800 physicians testing. The total number of tests continues to increase as well as the number of patients with a confirmed hATTR diagnosis. These newly diagnosed hATTR patients are starting on TEGSEDI. Physicians with TEGSEDI experience are choosing to put appropriate patients on therapy, and physicians that have not used TEGSEDI before are choosing to treat patients with TEGSEDI for the first time. In Q1, we made progress with market access by securing another long-term contract with a major payer in the U.S. Today, we have 94% of the total U.S. commercial lives covered, and we have negotiated long-term coverage that secures 75% of commercial lives through 2023. These are positive signs of progress and demonstrate our ability to compete effectively in the U.S. market. Outside of the U.S., we are expanding the total number of patients on commercial TEGSEDI in those countries where we have secured pricing and reimbursement. We are seeing our first countries launch in Southern Europe, which is an important region for us to enter because of its endemic patient population. In Spain, we've recently finalized pricing and reimbursement, and we were able to do so very quickly. It took us just 6 months to finalize negotiations when typically it takes 1 to 2 years. There are a couple of reasons for the fast turnaround: first, our team did a great job of communicating the need for patients to be treated with TEGSEDI in this market; and second, the Ministry of Health of Spain recognized the tremendous value that TEGSEDI brings to patients.

Early in the second quarter, we finalized pricing and reimbursement in Austria. And in Canada, we have entered into a Letter of Intent with a pan-Canadian Pharmaceutical Alliance, or pCPA, for TEGSEDI and secured multiple agreements with private payers. In Brazil, PTC is having ongoing discussions with CMED, which we expect to be completed in Q2. Brazil is the single most important market for PTC and has the highest prevalence of hATTR amyloidosis with polyneuropathy worldwide. TEGSEDI is the first and only self-administered silencer approved in Brazil for the treatment of Stage 1 or 2 polyneuropathy in adult patients with hATTR to improve quality of life. We are expanding access to hATTR patients across numerous geographies, and we are pleased with the ongoing progress that the commercial team is making. Overall, with TEGSEDI, we continue to be optimistic about the steady progress we are making and the momentum we are seeing in terms of new patient starts as well as the breadth and depth of prescribing physicians.

Turning to WAYLIVRA. WAYLIVRA is the only approved treatment for FCS patients in the EU. WAYLIVRA is commercially available in Germany and became commercially available in Austria this past quarter, and we continue to enroll patients in our ATU in France, which is a reimbursed early access program. Our team is focused on building FCS awareness with HCPs, supporting patient advocacy groups, identifying additional patients and securing market access as we follow the typical launch sequence in Europe. We are seeing more FCS patients initiated on WAYLIVRA therapy in countries where we have commercial access. In the U.K., we are in the early stages of our process to secure reimbursement with NICE. Currently, our WAYLIVRA submission is paused due to the COVID-19 pandemic. We look forward to continuing discussions with NICE when conditions improve. In the U.S., our discussions with the FDA are ongoing, and we are on track to refile this year. The PTC team continues to work to provide access to WAYLIVRA to patients across Latin America, and the goal is to file in Brazil with Anvisa in the second half of 2020. Our team is working diligently as we execute on both TEGSEDI and WAYLIVRA launches, and we look forward to keeping you updated on our progress.

I'll now turn the call over to Alex to discuss our pipeline and our financials.

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Alex G. Howarth, Akcea Therapeutics, Inc. - COO [5]

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Thank you, Kyle. I'll start with the AKCEA-TTR-LRx. The CARDIO-TTRansform and NEURO-TTRansform Phase III studies are underway with both the hereditary and wild-type forms of TTR amyloidosis. This represents another important milestone in further demonstrating Akcea and Ionis's commitment to bringing new treatment options to patients around the world living with TTR amyloidosis or ATTR. Both the wild-type and hereditary forms of ATTR are underdiagnosed and fatal with limited treatment options available. In regards to COVID-19, patients who are currently part of our NEURO-TTRansform and CARDIO-TTRansform Phase III studies have remained on study, and patient identification and site activation remains ongoing.

In March, we had temporarily paused enrollment to assess the situation and determine the best course of action on a site-by-site basis. Based on this assessment, in close consultation with investigators and sites and in accordance with local authorities, we've now lifted our temporary pause, and enrollment in both studies has resumed. Importantly, we do not expect this brief pause to significantly impact the time lines of these studies.

Turning to vupanorsen, which is partnered with Pfizer. Earlier this quarter, we announced positive top line results from the Phase II study in a population of patients with hypertriglyceridemia, type 2 diabetes and nonalcoholic fatty liver disease or NAFLD. We met the primary endpoint, triglyceride lowering as well as significant reduction in additional lipid parameters in ANGPTL3. While we did not see reductions in liver fat or hemoglobin A1c, we are pleased with the evidence of reduction in cardiovascular risk factors. Vupanorsen generally had a favorable safety and tolerability profile. We plan to present detailed results at a future medical congress.

Given the results of this study, we see a lot of potential for vupanorsen in treating cardiovascular disease, and we are working with Pfizer to design the best development path to this medicine.

Next is an update on AKCEA-APO(a)-LRx. This medicine was granted Fast Track Designation by the U.S. Food and Drug Administration as a potential treatment for people who have significant risk of cardiovascular disease due to elevated levels of lipoprotein(a) or Lp(a). Fast Track Designation enables an expedited regulatory path to medicines, but addresses a significant unmet medical need for patients with serious diseases. This achievement underscores the significant value this medicine may bring to millions of patients worldwide with established Lp(a)-driven cardiovascular disease who currently have no effective therapeutic options.

The AKCEA-APO(a)-LRx Phase III HORIZON study is a 7,500-patient cardiovascular outcomes trial, and it's up and running with Novartis, and first patients are on treatment. Novartis is heavily invested in this program and its success.

And last, but certainly not least, this brings us to AKCEA-APOCIII-LRx, which we are also really excited about. In January, we announced positive top line results from the Phase II study of the AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia who are at risk or have established cardiovascular disease. We met the primary endpoint of triglyceride lowering as well as multiple secondary endpoints. At the highest monthly dose, we saw more than 90% of patients reach triglyceride levels below the recognized threshold for cardiovascular risk of 150 milligrams per deciliter. AKCEA-APOCIII-LRx also had a favorable safety and tolerability profile. Importantly, for AKCEA-APOCIII-LRx and for the broader LICA platform, there were no serious safety signals. We plan to present the more detailed data at a future medical conference.

We are thrilled that we are able to retain the right to AKCEA-APOCIII-LRx and energized to drive the development and commercialization strategy for this medicine. These data are very encouraging, and we're making excellent progress at the start of Phase III study in FCS later this year, a patient community and a market we know very well. We're committed to investing heavily in this medicine, and we're also exploring the potential for use in other rare and broad disease indications.

Now let's review our financials. For Q1, we had a total revenue of approximately $16 million and an operating loss of approximately $38 million on a non-GAAP basis, including non-GAAP operating expenses of $54 million. Our revenue includes approximately $15 million of product sales from TEGSEDI and WAYLIVRA, which, as Kyle mentioned, is approximately 8% growth quarter-over-quarter. We look forward to continued growth as we launch both medicines in additional countries this year. We ended the quarter with approximately $421 million in cash and short-term investments. With our strong balance sheet, we are well positioned to execute on our current strategic objectives and our vision for the future.

I'll now turn the call back over to Damien.

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [6]

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Thank you, Alex. To wrap up, we had a solid start to the year with lots to look forward to on the horizon. This quarter, we continue to make progress with our 2 commercial products, TEGSEDI and WAYLIVRA. We further advanced our broad pipeline of products, and we strengthened our management team and Board of Directors. We have a set of life-changing medicines that we believe can have a significant impact on patients who are currently underserved. We believe the positive data readouts for AKCEA-APOCIII-LRx and vupanorsen earlier in the quarter demonstrate the commercial potential of the LICA technology platform. With launches in additional countries on the horizon for TEGSEDI and WAYLIVRA, the commitment from Ionis to potentially license additional rare disease medicines that are complementary to our pipeline and capabilities, Akcea is very well positioned in 2020 and beyond.

I want to close by thanking all of our employees, our patients, caregivers and advocates for their shared dedication to advancing and supporting new therapies in serious and rare diseases. I'll now open up the line for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Chad Messer from Needham.

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Chad Jason Messer, Needham & Company, LLC, Research Division - Senior Analyst [2]

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On the Phase III trial for the APOCIII LICA, anything you could tell us about what that might look like? Is it going to pretty much look like the approach study? And I know you've mentioned other rare disease indications, potentially including really high triglycerides. And so there I was wondering if something akin to the COMPASS study would potentially be sufficient.

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [3]

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Chad, it's Damien here. Thank you for the question. So we've not disclosed any of the details of the FCS study. I mean this is a patient population we know very well, and we've run a study, Phase III pivotal study for FCS previously. So you can make an assumption that it won't be too dissimilar. In terms of other indications, obviously, we're looking at other opportunities where significant triglyceride-lowering may benefit patients, both in rare disease indications and also in larger disease indications. But we're still working through what those indications may look like. And we look forward to updating you in due course on that.

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Chad Jason Messer, Needham & Company, LLC, Research Division - Senior Analyst [4]

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Right. Great. And then maybe just one on the COMPASS, the genetic testing program that you have going. Can you tell us how many tests you're running and what sort of the hit rate looks like there?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [5]

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Yes, for that, I'm going to hand it over to Kyle to give you some information about the COMPASS study.

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [6]

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Chad, thanks for the question. So we're not disclosing all of the details around the COMPASS program, but we are communicating, obviously, the 1,800 physicians currently utilizing the program. We are seeing more physicians testing quarter-over-quarter. We are seeing more tests coming quarter-over-quarter, and we're also seeing positive hereditary confirmation coming back from those tests. And then if and when appropriate and the physician deems TEGSEDI is the right therapy, they're moving those patients on to TEGSEDI. So it's a program that is operating effectively and doing exactly what it's designed to do at this point.

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Operator [7]

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The next question comes from Do Kim from BMO Capital.

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Unidentified Analyst, [8]

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This is [Jameson] on for Do, and glad everyone is safe and doing well. So one from us on WAYLIVRA. You briefly mentioned the COVID-19 impact. How will this impact your launch trajectory? And any additional details on time lines for reimbursement in the U.K. and France would be great.

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [9]

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Thanks, [Jameson]. This is Damien here. I'll start and then hand it over to Kyle. We're off to a good start with WAYLIVRA in Europe in terms of the launch. It's still early, but remember it's the only medicine that's been approved for FCS patients with -- in Europe. Kyle, would you like to give a little bit more information around the launch sequence there?

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [10]

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Yes, I'd be happy to. So great question. We're progressing well. So Germany, we currently are launched as well as Austria. And then we have the ATU that is ongoing in France. The slowdown in the U.K., obviously, is due to COVID, as I communicated earlier, and we are still reaching back out to the agency there to try to see when we can reengage in those discussions. But typical launch sequence from there. So we'll look at Nordics, we'll look at Southern Europe, and we'll continue to navigate the pricing reimbursement process, as you would expect, in those countries moving forward.

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Operator [11]

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The next question comes from Jim Birchenough from Wells Fargo.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [12]

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This is Yanan dialing in for Jim. First, just curious for the COVID-19 situation, does it impact the monitoring of TEGSEDI?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [13]

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Thanks, Yanan, for the question. This is Damien. So let me just take a step back and say during COVID-19, we're very focused, obviously, on keeping our patients, employees and health care professionals safe and healthy during this time. That's really important to us. We took early and immediate action to mitigate risks to the business. For the commercial business, I'm going to let Kyle give you some sort of information around any impacts on the commercial business, including monitoring.

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [14]

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Yes. I'll speak to the Akcea Connect program in the U.S. first. That's our most robust program and the one that has fortunately been able to continue without any interruption during the pandemic. Our nurse case managers are able to be able to coordinate with patients in their home. They're doing audio and video calls with these patients. They're talking about disease education and drug education. Our third-party that handles our mobile lab service is able to meet with these patients and do the draws and collections on a weekly basis and able to conduct the testing, and we've had no interruption as it relates to the at-home monitoring process, which has worked very effectively in the U.S. Ex U.S., similarly, we've been able to work effectively with the institutions and the local agencies to make sure that those patients are able to be monitored per the label as well. So all is being handled, I think, appropriately and going well as it relates to the monitoring program across the countries that we're in.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [15]

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That's great to hear. And it's also great to hear that the subcutaneous route of administration kind of stands out, as you indicated, in this current environment. Just wondering, going forward, would there be any leading indicators of TEGSEDI growth that we can get an early read on?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [16]

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Kyle, would you like to talk about that?

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [17]

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Yes. So when I look at the markets, I think, first and foremost, the subcutaneous -- subcu at-home therapy is the most critical part to this, right? It's a key differentiator. We know that TEGSEDI is effective at knocking down the TTR protein. We know that it's effective in terms of the treatment of polyneuropathy. So that's really encouraging. The leading indicators, probably a number of physicians testing, as I indicated, is going to be the key part to this. So we've got a lot of physicians testing through the androgenetic testing program. That tells us that physicians are educated on the disease, they're looking for these types of patients, and they're actively engaged in diagnosis and then treating those physicians. The other indicator that I would bring up is our sequential launch ex U.S., the European countries that we're continuing to move across. Southern Europe is a key area for us, obviously, with the endemic population. The success that we've had in Spain that I referenced in my comments, the speed at which we were able to achieve that, the interest by the agency based on the therapy and the advantages that it has and being able to treat the patient population there that needs treatment is very, very encouraging to us. So I think between the testing that's occurring combined with the expansion that we're doing across Europe should be pretty good indicators in terms of progress that we're making, and we're optimistic about where we're headed.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [18]

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Wonderful. Last question from us. Any thoughts on additional asset from Ionis or third-party asset to in-license?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [19]

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Yanan, this is Damien, again. Yes, absolutely, I mean, that's part of our strategy to grow the pipeline. And we're very focused on working collaboratively with Ionis to find a new medicine to license that will fit in with our capabilities and our expertise and our interest levels. And of course, they're going to be rare disease medicines, and you're probably quite familiar with the Ionis' wholly owned pipeline. Also, we continue to look for third-party medicines. We're not looking for early-stage medicines. We're looking for mid- to late-stage medicines, closer to approval the better. And that program is still running. And of course, any medicines that we are to license into the company will fit in with our capabilities and our expertise. So no specific sort of news on that beyond that we're continuing to look actively at both our Ionis medicines and third-party medicines.

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Operator [20]

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(Operator Instructions) The next question comes from Paul Matteis from Stifel.

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Nathaniel Tower, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [21]

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This is Nate on for Paul. First, I just wanted to ask about the TTR studies and COVID. I know the pause was removed, but what percent of sites in the U.S. began reenrolling patients? And then maybe I missed this, were there actually any interruptions in dosing?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [22]

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So let me start with the second part of that, which was, no, there were no interruptions in dosing. All the patients who were on medicine remain on medicine. So that's that part. The second part, I don't know, Alex, if you have any further information about -- I don't think I've got any information about the first part of the question. Alex, do you?

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Alex G. Howarth, Akcea Therapeutics, Inc. - COO [23]

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Yes. Well, we're sort of -- we're not giving breakdown of the resumption of the sites. But all I can say is that the pause has been lifted, and we are of the opinion that actually there will be a nominal impact to the time line of the actual study.

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Nathaniel Tower, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [24]

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All right. Makes sense. And then maybe I'm reading a little too much into this, but I think you previously said your goal was to file WAYLIVRA this year in the U.S. and now you're saying you plan on it. Did you hear -- did you have any conversations with the FDA that increased your confidence about your ability to file that? And what were those if so?

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [25]

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Nate, thanks for the question. So we have been in discussions with the FDA, and we have a plan now to refile, and we're excited about that. We believe it's a great medicine that the benefits significantly outweigh any risks, and we'll provide an update on our progress over the coming months.

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Operator [26]

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There are no more questions in the queue. This concludes our question-and-answer session. I would like to turn the conference back over to management for any closing remarks.

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Damien McDevitt, Akcea Therapeutics, Inc. - CEO & Director [27]

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Okay. Thank you all for joining today. We look forward to keeping you updated on our progress as we continue to execute on the TEGSEDI and WAYLIVRA launches and build upon the strong foundation at Akcea. Have a great afternoon.

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Operator [28]

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