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Edited Transcript of AKCA.OQ earnings conference call or presentation 6-Aug-19 8:30pm GMT

Q2 2019 Akcea Therapeutics Inc Earnings Call

CAMBRIDGE Aug 13, 2019 (Thomson StreetEvents) -- Edited Transcript of Akcea Therapeutics Inc earnings conference call or presentation Tuesday, August 6, 2019 at 8:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Jeffrey M. Goldberg

Akcea Therapeutics, Inc. - COO

* Kathleen Gallagher

Akcea Therapeutics, Inc. - VP of Communications & IR

* Michael F. MacLean

Akcea Therapeutics, Inc. - CFO

* Paula Soteropoulos

Akcea Therapeutics, Inc. - CEO & Director

* Sarah Boyce

Akcea Therapeutics, Inc. - President & Director

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Conference Call Participants

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* Benjamin Jay Burnett

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

* Chad Jason Messer

Needham & Company, LLC, Research Division - Senior Analyst

* Guyn Kim

BMO Capital Markets Equity Research - Analyst

* Nicholas M. Abbott

Wells Fargo Securities, LLC, Research Division - Associate Analyst

* Subhalaxmi T. Nambi

Cowen and Company, LLC, Research Division - Research Associate

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Presentation

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Operator [1]

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Good afternoon, and welcome to the Akcea Therapeutics Second Quarter 2019 Conference Call. As a reminder, this call is being recorded.

I will now turn the call over to Kathleen Gallagher, Akcea's Vice President of Corporate Communications and Investor Relations. Ms. Gallagher, please begin.

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Kathleen Gallagher, Akcea Therapeutics, Inc. - VP of Communications & IR [2]

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Thank you, Laura. Hello, everyone. Thank you for joining today's call. With me today are Paula Soteropoulos, our Chief Executive Officer; Sarah Boyce, our President; Mike MacLean, our Chief Financial Officer; and Jeff Goldberg, our Chief Operating Officer.

As a reminder, this conference call includes forward-looking statements regarding the financial outlook for Akcea, Akcea's business 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 drugs, is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and any endeavor of building a business around such drugs.

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 safe 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 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 Paula.

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [3]

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Thank you, Kath. Good afternoon, everyone. Thank you for joining us. Last month marked 2 years since our IPO. In that time, we've delivered on an aggressive agenda of launching 2 rare disease drugs, while progressing a large pipeline of therapies that has the potential to address severe diseases that are currently underserved.

For our rare disease drugs, we've launched TEGSEDI in the U.S., Germany and Canada. We reported $10 million in sales for TEGSEDI in Q2, which aligns with consensus. We see momentum building in the launch and growth in the hATTR market. As patients and physicians are gaining experience with TEGSEDI, we continue to hear positive feedback of the importance of this efficacious drug that gives patients the independence to treat on their own term.

For WAYLIVRA, we will be launching in Germany this month, and we are preparing to launch in additional countries in Europe in 2020.

For the pipeline, earlier today, we announced the top line results from our study of WAYLIVRA in patients with familial partial lipodystrophy or FPL. We are encouraged by these results, and we'll discuss them in further detail in the call.

We have delivered a positive Phase II result for AKCEA-APO(a)-LRx, or TQJ230, which resulted in Novartis exercising their option for that product. Novartis shares our enthusiasm and urgency to bring AKCEA-APO(a)-LRx to patients experiencing cardiovascular disease due to high levels of Lp(a). Novartis is in the final stages of the Phase III initiation for the AKCEA-APO(a)-LRx study. We will talk more about that trial design later in the call.

With AKCEA-TTR-LRx, we are expanding our commitment to the TTR community, including patients with wild-type and hereditary cardiomyopathy, and we are on track to initiate our Phase III program with Ionis by the end of this year.

The rest of our pipeline is progressing with the Phase II studies for both AKCEA-APOCIII-LRx and AKCEA-ANGPTL3-LRx reporting data early in 2020. All of this keeps us on track to close out 2019 with 2 commercial products, 2 programs in Phase II development and 2 drugs nearing Phase II data. We remain focused on delivering innovative solutions that improve the lives of those affected by serious and rare diseases.

And I will turn the call over now to Sarah.

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Sarah Boyce, Akcea Therapeutics, Inc. - President & Director [4]

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Thanks, Paula. We are seeing growth in the number of patients diagnosed with hATTR. We are also seeing increased physician and patient interest in TEGSEDI. This quarter's growth is primarily...

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [5]

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Sarah, you've gone on mute.

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Sarah Boyce, Akcea Therapeutics, Inc. - President & Director [6]

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Thank you, Paula.

We are seeing growth in the number of physicians and patients with hATTR. We are also seeing increased physician and patient interest in TEGSEDI. This quarter's growth is primarily driven by the United States, but we anticipate that the EU and Canada will play a larger role as we continue to work towards reimbursement in each country.

In the U.S., we are continuing our focus on the community physicians for whom disease education is critical to diagnose and support patient finding. Data from our marketing efforts support that there is a lot of potential for growth in diagnosis of polyneuropathy of hATTR.

Our field team continues to call on cardiologists, hematologists and neurologists. And we have seen -- in particular, we are pleased to see that we are gaining additional traction with the neurology community. This is important and a testament to the work of our field teams, who are spending a great deal of time focused on the neurology community, where we are seeing our messages on the urgency to treat hATTR early resonate.

The efforts of our field team are paying off. And this quarter, we saw an increase in TEGSEDI prescriptions in the United States of 50%. At this point, essentially, all patients have converted from early access programs and the OLE to commercial drug.

This past quarter -- as we look forward, we are confident that we are highly competitive with bringing on naïve patients who are identified and also seeking treatment. We are also seeing an increase in diagnosis from our genetic testing program, hATTR Compass. We recently announced the 1-year anniversary of the COMPASS program, and there are now over 800 physicians using this program. COMPASS allows physicians to diagnose patients locally and TEGSEDI allows them the independence to treat their patients locally as well. This is great for physicians who can keep their patients in their local practice and for patients who do not have to travel far distances to get their treatment.

Our market access team continues to do a great job working with patients and payers to make TEGSEDI available. This is excellent, especially at this point, in a rare disease launch. We attribute this success to the hard work of our market access team and leading up to and since launch as well as our AKCEA CONNECT team. A 30-day time from prescription to prescription being fulfilled is really excellent and something that's great to see. AKCEA CONNECT, our program of field-based nurse case managers, have an expertise in assisting patients and physicians through all aspects of TEGSEDI.

Outside of the U.S., we are working towards reimbursement in multiple European countries and in Canada. With the highly specialized technology guidance from NICE now implemented by the NHS, we will begin treating our first patients in England imminently.

In addition, we are seeing positive momentum in Canada. Our AKCEA CONNECT program is up and running in England and Canada and consistent with our global commitment. We believe we are providing the highest level of patient and physician support. We are working towards access in additional European countries, and we will update those as we launch.

Finally, the team at PTC is working towards the approval for TEGSEDI across Latin America. We are confident that they are the right partner with the right expertise to deliver TEGSEDI to patients in Latin America efficiently and in ways that align with our patient-centered focus.

This month, we are launching WAYLIVRA in Germany. WAYLIVRA is the only treatment available for patients with FCS. We believe there are approximately 1,000 patients eligible for treatment in Europe. The hallmark of FCS is extremely high triglycerides, which puts patients at risk of unpredictable and potentially fatal acute pancreatitis. Symptoms of FCS also include chronic complications due to permanent organ damage as well as major emotional and psychosocial issues.

There are many synergies across Europe with the infrastructure we've got for TEGSEDI. Our team will be calling on lipid specialists, including specialized endocrinologists and cardiologists. In Europe, most patients with FCS will be treated at academic centers. We expect to launch WAYLIVRA in additional countries following the typical European launch sequence. In the U.S. and Canada, our regulatory discussions are ongoing. PTC is also gearing up to deliver WAYLIVRA to patients in Latin America.

Across the board, the team has made great progress to date. We see a lot of opportunity in both the rare diseases, and we are investing in disease education as we work to build for the long term. We are executing on our multiple launches by ensuring access to TEGSEDI and WAYLIVRA and as always, keeping our focus on patients.

Now over to Mike.

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Michael F. MacLean, Akcea Therapeutics, Inc. - CFO [7]

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Thank you. As you heard from Sarah, the team is executing on the TEGSEDI launch, and we look forward to adding additional sources of revenue from TEGSEDI as we gain access in other European markets.

For Q2 2019, we had total revenue of approximately $27 million and an operating loss of approximately $24 million on a non-GAAP basis, including non-GAAP operating expenses of $51 million. Our revenue includes approximately $10 million of product sales from TEGSEDI. As we have disclosed, our sales of TEGSEDI were generated in the U.S. and Germany. Our revenue growth is predominantly driven by adding 90 patients in the U.S. At $10 million in revenue from TEGSEDI sales, we are in line with consensus. We are pleased with the growth that we are seeing in our hATTR franchise.

Looking forward, we expect to add revenue in 2019 from WAYLIVRA as we begin to roll out our European launch, starting with Germany.

For clarity, WAYLIVRA will be sold as a single prefilled syringe. We will recognize revenue for WAYLIVRA under the title model similar to TEGSEDI. We anticipate our first revenue from WAYLIVRA will occur in Q3 2019.

Just like TEGSEDI, we set our price for the first year in Germany, while we work with the authorities to agree on an ongoing price for WAYLIVRA, starting after the first year. We will share the initial WAYLIVRA price when we launch, and we expect to be in the typical rare disease price range.

We ended Q2 with $296 million in cash and short-term investments. This includes a $6 million milestone payment from PTC on approval of WAYLIVRA, which we split 50-50 with Ionis.

Turning to our financial guidance. With $296 million in cash at the end of Q2, we believe we have sufficient cash on hand to carry out commercial activities for our products as well as to fund progression of our current pipeline into 2021. We are uniquely positioned with having 2 products on the market as well as the potential license fee under our collaboration with Novartis.

Further, we have a pipeline with drugs for both broad and rare diseases that represent meaningful economic opportunities for our company.

I will now turn the call over to Jeff to discuss the pipeline in further detail.

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Jeffrey M. Goldberg, Akcea Therapeutics, Inc. - COO [8]

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Thanks, Mike. We announced earlier today the results of our study of WAYLIVRA in patients with familial partial lipodystrophy, or FPL. The BROADEN study was a global study of 40 patients with FPL. The primary endpoint was reduction in triglycerides and a key secondary endpoint was reduction in hepatic fat, and we hit both of those endpoints in the study.

Although there was significantly lower triglycerides from baseline by 88% at 3 months compared to 22% reduction in placebo-treated patients with a p-value of less than 0.001, significant triglyceride lowering was maintained throughout the 12 months of the study.

In addition, there was a statistically significant mean reduction from baseline of 51.9% of liver fat in the liver-treated patients at 12 months compared to a 1.5% increase in the placebo group, with a p-value of 0.004.

Overall, we're encouraged by the safety and efficacy profile demonstrated by WAYLIVRA in patients with FPL. Most frequent adverse events in BROADEN were all mild-to-moderate in severity, including injection site reactions, the common cold, urinary tract infections and reductions of platelet levels. Importantly, there were no serious or severe platelet count decreases. These are the top line results. As we obtain additional data, we'll review it in its totality and discuss the data with key experts in the field to define our next steps.

We are so grateful to the patients and physicians in the FPL community for their time and participation. We and Ionis have always focused on diseases where we can transform patients' lives. We are proud to add to the body of evidence around FPL, and we remain committed to sharing what we have learned with the patients and medical communities.

Switching to AKCEA-APO(a)-LRx, also known as TQJ230. This drug is intended for patients who have high levels of lipoprotein (a) or Lp(a), a genetic risk factor for cardiovascular disease that cannot be treated by existing lipid lowering therapies or controlled by diet and exercise. Novartis is in the final stages of initiation for the Phase III outcomes study and the team there has done a great job moving quickly and efficiently to get this large study underway.

The global study will be in approximately 7,500 patients who have elevated Lp(a) of 70-milligram per deciliter or higher and have an established history of cardiovascular disease. The study is designed with 2 primary outcome measures: one, focused on patients with an Lp(a) measurement greater than or equal to 70-milligram per deciliter; and one for patients with an Lp(a) measurement greater than or equal to 90-milligram per deciliter. The goal of the overall study is to reduce the risk of expanded major adverse cardiovascular events, or MACE. Secondary outcomes include reducing risk of CV death, nonfatal MI and nonfatal stroke, coronary heart disease as well as hospitalization and all-cause death. The dose is 80 milligrams monthly in a prefilled syringe. We're very excited about this study, and we look forward to supporting Novartis as they work to enroll first patients early next year.

We and Ionis are on track to initiate the Phase III program for AKCEA-TTR-LRx in patients with ATTR. We plan to initiate 2 studies, one in patients with hATTR with polyneuropathy and one in patients with both hereditary and wild-type TTR cardiomyopathy. Our Phase I/II study for AKCEA-TTR-LRx is ongoing. We plan to present the initial data in September, first at the upcoming European Amyloidosis Meeting in Berlin; and then again, at the Heart Failure Society of America Annual Meeting in Philadelphia.

Looking ahead, with enrollment complete in both Phase II studies of AKCEA-APOCIII-LRx and AKCEA-ANGPTL3-LRx, we continue to anticipate data early in the first half of 2020.

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

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [9]

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Thanks, Jeff. We are pleased with the progress the team has made in raising awareness and elevating the urgency to treat hATTR as well as the commercial execution of the TEGSEDI launch. We have 2 commercial products with TEGSEDI and the upcoming launch of WAYLIVRA. Our Phase III programs are on track to initiate this year for AKCEA-APO(a)-LRx and AKCEA-TTR-LRx. And further, AKCEA-APOCIII-LRx and AKCEA-ANGPTL3-LRx are moving towards data early next year.

We are looking forward to closing out 2019 with 2 commercial products, 2 Phase III programs initiated in 2 Phase II products nearing data. We continue to see momentum and growth in the hATTR market and are working to build the FCS market in Europe as well. We have a solid pipeline with exciting rare and broad indications that we believe can make a positive impact on patients' lives. We look forward to sharing our continued progress with you as we go into the second half of the year.

And now we'll open up the line for questions. You can go ahead, Laura.

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Questions and Answers

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Operator [1]

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(Operator Instructions) We have a first question from the line of Chad Messer of Needham Company.

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

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Great. Congratulations on the quarter and on the positive pivotal data in FPL. I think I'll start with TEGSEDI. Have you guys run across any [Amadeus] patients yet, maybe switching or combining?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [3]

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Chad, thanks for the question. We -- as we, I think, noted last -- yes, we continue to see some patients switch from both competitive drugs.

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

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Okay. Sure. Then maybe just on the FPL data. Very impressive, especially that liver fat reduction and the safety profile seems to corroborate something that you and Ionis have been saying for a while, which is that the thrombo seen in FCS seems to be something that's a lot worse in that indication than others. But I think having this exact same drug now in 2 indications can really help that. What -- can you talk about the regulatory strategy here? I mean, in Europe, going for a label expansion seems to make a lot of sense, but how do you approach the U.S. regulatory authorities with this positive study?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [5]

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Yes. Chad, we agree regarding the platelets and the inherent volatility in FCS is different from other populations, but I'll have Jeff take that question.

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Jeffrey M. Goldberg, Akcea Therapeutics, Inc. - COO [6]

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Yes. As we think about leveraging these data for regulatory purposes, certainly, we're pleased that, as you said, it expands the database and reinforces the safety profile that we've been talking about. In terms of next steps, this is just top line data. We're collecting up all of the data from the study. We're going to evaluate that with the KOL and the thought-leader community, and then we'll take it forward.

As you say, the next step in Europe could be to do a follow-on on top of the program we've already got. In the U.S., as you know, we've got ongoing dialogue and certainly, this will be included in that conversation, and we'll see where that goes.

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Chad Jason Messer, Needham & Company, LLC, Research Division - Senior Analyst [7]

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All right. Great. Yes, we'll stay tuned there. And then just real quick, and I apologize if I've missed something along the way. But for the angiopoietin-like 3, I thought there were some small studies in rare lipid disorders that were also out there. What's the status on those?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [8]

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Yes. We talked about the angiopoietin-like 3 in rare, we talked about having data in midyear. And so we're still analyzing the data. I will say that we have prioritized the FPL with [Livra]. So we're working through that. So yes, that will be coming.

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Chad Jason Messer, Needham & Company, LLC, Research Division - Senior Analyst [9]

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All right. Great. We'll look for that. And yes, I get it. NASH is a much more potentially exciting indication. All right.

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [10]

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We agree. Thanks, Chad.

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Operator [11]

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Your next question comes from the line of Do Kim of BMO Capital Markets.

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Guyn Kim, BMO Capital Markets Equity Research - Analyst [12]

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Congrats also on the positive BROADEN study data. Just starting on that, perhaps you could talk a little bit more about the platelet decline that you did see. How did that compare to the placebo arm? And does that mean that FPL patients won't require the monitoring rams that you have for FCS patients?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [13]

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Thanks, Do. We did see some platelet declines, but as we said, they were all mild to moderate. We had no serious or severe drop, so different than what we saw in FCS. And we also did monitor patients, and we anticipate that they would need to continue to be managed with platelet monitors. There is an effect, but not to the same extent as in FCS.

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Guyn Kim, BMO Capital Markets Equity Research - Analyst [14]

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Okay. And were there any clinical or functional endpoints of this study like you had for FCS pancreatitis? And what would physicians care about in the results?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [15]

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Jeff, do you want to take that?

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Jeffrey M. Goldberg, Akcea Therapeutics, Inc. - COO [16]

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Sure. So obviously, we're really excited about the triglyceride and liver fat. Those are pretty big and significant endpoints for us to be able to hit. And as we look at the data, there will be other factors looking at quality of life, looking at other parameters to really ensure that we've assessed this disease as fully as we can, given the study that we've got. So a lot more data to come.

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Guyn Kim, BMO Capital Markets Equity Research - Analyst [17]

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And a final question on the TEGSEDI sales. You said growth was primarily driven by the U.S. Does that suggest that there wasn't any growth in Germany; that you've saturated that study in that market?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [18]

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Sarah, do you want to take that?

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Sarah Boyce, Akcea Therapeutics, Inc. - President & Director [19]

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Sure. So we saw, as I said, a 50% increase in prescriptions in the United States. So we're really pleased with our progress there and most of that's been naïve patients.

Within Europe, we have, as you know, launched in Germany, and we have good progress. I'm pleased with how Germany is going, and we're also expanding out into additional countries.

So we have patients treated in Italy, Spain, Portugal, although we're working through the reimbursement processes there, so they're currently free of charge.

And then in the U.K., we're really looking forward to being able to have the first patient dosed with TEGSEDI in England, which should happen imminently.

So I think you'll see more of Europe coming into the next quarter, Do, but very, very pleased with the new prescription growth in the U.S. as well as the growth in the COMPASS program, which was also great to see.

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Operator [20]

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Your next question comes from the line of Paul Matteis of Stifel.

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Benjamin Jay Burnett, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [21]

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This is Ben Burnett on for Paul Matteis. One on just FPL as a disease indication. I guess how many FPL patients are diagnosed today and how do you see this -- hello?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [22]

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That's better. You had a real tough sentence there, so that's better. Try again.

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Benjamin Jay Burnett, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [23]

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Okay. Let me say that again. A question on just FPL and the diagnosis rate today and how you see the diagnosis rate evolving over time. And I guess, what changes that?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [24]

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So regarding diagnosis, right? This is a complex disease from a diagnosis perspective because these patients, as you know, have a disease caused by defective adipose tissue, which causes a maldistribution of fat. That's why they also have high triglyceride, high liver fat and also cardiovascular disease.

Look, many of the manifestations that doctors struggle with, with these patients start with women, for example, with very significant PCOS, so they're being seen by reproductive endocrinologists. They also have a pretty difficult insulin resistance, so being seen by diabetologists. So a lot of the work that would be needed for this patient population is to really educate and get these patients more formally diagnosed because they are looked at by their specific symptom or manifestation rather than a disease in totality. There are very few experts in the world that I can count on my hand. And so it requires a lot more education and broadening out the understanding of this disease to these other types of physicians who tend to see these patients, again, in those singular areas that they're treating them. There really are very few physicians looking at the totality of the disease.

So it's a long-winded way to say that we have to influence that diagnosis rate; that it will have to evolve over time for people to recognize that this is one disease, not these separate symptoms.

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Benjamin Jay Burnett, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [25]

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Right. Okay. Okay. And then, I guess, kind of on the same topic, did you disclose the discontinuation rate of the FPL study? And I guess, could you also comment on just the percentage of the patients who enrolled over to the open-label extension portion?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [26]

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No. We didn't talk about that level of data yet. And again, as we analyze the rest of the data and bring forward more of that data, we'll share that.

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Operator [27]

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And your next question comes from the line of Subbu Nambi of Cowen.

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Subhalaxmi T. Nambi, Cowen and Company, LLC, Research Division - Research Associate [28]

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Congrats on the great quarter. So my question was what drives the 22% knock down that you observed in the BROADEN trial in the placebo group?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [29]

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Thanks, Subbu. Jeff?

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Jeffrey M. Goldberg, Akcea Therapeutics, Inc. - COO [30]

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So Subbu, it's a fair question. Remember, these individuals have a whole bunch -- as Paula said, a whole bunch of metabolic derangement. We also mandated a strict diet during the trial. So it's possible that some of that was natural chance. It's also possible that some of that was due to the diet and just being in control and being aware of what's going on. But that's part of what we'll dig deeper into the data as we move forward. But either way, it was a very significant reduction in triglycerides with drug on board.

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Subhalaxmi T. Nambi, Cowen and Company, LLC, Research Division - Research Associate [31]

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I see. And I have a follow-up question on the APOCIII trial. I know it's early, but how would you define a successful trial for Novartis to opt in for this program as well?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [32]

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So Subbu, I think we -- based on Phase II data, we expect that we're going to hit the knockdown of APOCIII in triglycerides. I'm pretty -- I can say we're pretty confident in that.

So the study is really designed for -- of course, to hone in on a dose as well as safety. And so of course, we'd be looking at the same things that we looked at with APO(a) and I'm sure Novartis will as well. And given the confidence that we have with the reproducibility, or I should say consistency with like a drug, and we anticipate to see the same type of profile that we saw for APO(a). So we'll be looking at a clean profile of platelets and renal, et cetera. So I think those are 2 key important factors.

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Operator [33]

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(Operator Instructions) Your next question comes from the line of Jim Birchenough of Wells Fargo.

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Nicholas M. Abbott, Wells Fargo Securities, LLC, Research Division - Associate Analyst [34]

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It's Nick on for Jim this afternoon. And congratulations from us on the FPL data. Can you comment whether all patients receiving WAYLIVRA, benefited from a reduction in liver fat?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [35]

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Was the question if all patients benefited from a reduction in liver fat?

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Nicholas M. Abbott, Wells Fargo Securities, LLC, Research Division - Associate Analyst [36]

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Yes, who received WAYLIVRA.

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Jeffrey M. Goldberg, Akcea Therapeutics, Inc. - COO [37]

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So I think maybe the best way to answer that is we saw 50-something-percent reduction in liver fat in the mean and variation is -- obviously, there's some individual variation we'll take a look at as part of the totality of the data, but very significant reduction at the mean level.

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Nicholas M. Abbott, Wells Fargo Securities, LLC, Research Division - Associate Analyst [38]

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And then of the 1,000 patients in Europe, how many of those are currently in care for FPL? And what proportion of those have been educated or are aware WAYLIVRA?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [39]

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Yes, I would say, of the 1,000 patients in Europe, it is a low number in terms of awareness of the disease itself in terms of formal diagnosis. Many of these patients had very long journeys to diagnose it. Again, as I mentioned, that many of them were treated for the specific disease manifestations, again it could be TQS, it could be their severe insulin resistance, and it takes time for someone to actually say, "Wait, these all connect, and you have familial partial lipodystrophy." So there's still ongoing work for education and awareness.

So typical of a rare disease, where you don't have many options for a patient, you see a low awareness and that part of educating as maybe an option comes forward, that will include the awareness and hopefully getting patients to the right type of specialist who can treat their disease.

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Nicholas M. Abbott, Wells Fargo Securities, LLC, Research Division - Associate Analyst [40]

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Great. And when might we see this data presented at a medical meeting?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [41]

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Again, we just announced top line. So we need a bit of time to get through all of the data. So we don't have a time line yet on when we would be sharing that at a medical meeting.

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Operator [42]

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You have a follow-up question from the line of Paul Matteis of Stifel.

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Benjamin Jay Burnett, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [43]

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This is Ben Burnett again on for Paul Matteis. I apologize if this has already been addressed, but I wanted to ask about TEGSEDI. You mentioned that there was growth you're seeing in sales, primarily coming from the U.S. And can you comment on just kind of the split of scripts and whether or not they're coming from cardiologists versus neurologists and kind of the mix there?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [44]

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Yes, we're getting prescriptions from neurologists, cardiologists, hematologists. And we aren't talking about the specific split, but we're seeing growth in all of those specialties and most especially additional growth from neurologists as Sarah had mentioned.

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Benjamin Jay Burnett, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [45]

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Okay. And I guess, could you -- it's still early days, I understand. But is there anything you could say about the persistence on TEGSEDI?

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [46]

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Sarah, do you want to take that?

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Sarah Boyce, Akcea Therapeutics, Inc. - President & Director [47]

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Sure. While we're not commenting specifically, one thing I would say is how pleased we are with AKCEA CONNECT and the work that they're doing both on educating patients and physicians, setting expectations and also helping patients establish a routine. And all of that has by far exceeded our expectations, and we're really pleased with how that's going.

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Operator [48]

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(Operator Instructions) I'm showing no further questions at this time. I would now like to turn the conference back to the presenters.

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Paula Soteropoulos, Akcea Therapeutics, Inc. - CEO & Director [49]

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All right. Thanks, Laura. Well, thank you, everyone, for joining today. With our multiple ongoing launches and broad pipeline, we have a number of upcoming catalysts. We look forward to sharing updates with you as we continue to execute across the business. Have a great afternoon.

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Operator [50]

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Ladies and gentlemen, this concludes today's conference. Thank you for your participation, and have a wonderful day. You may all disconnect.