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Edited Transcript of AKCA.OQ earnings conference call or presentation 5-Nov-19 9:30pm GMT

Q3 2019 Akcea Therapeutics Inc Earnings Call

CAMBRIDGE Nov 9, 2019 (Thomson StreetEvents) -- Edited Transcript of Akcea Therapeutics Inc earnings conference call or presentation Tuesday, November 5, 2019 at 9:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Damien McDevitt

Akcea Therapeutics, Inc. - Interim CEO & Director

* Kathleen Gallagher

Akcea Therapeutics, Inc. - VP of Communications & IR

* Kyle Jenne

Akcea Therapeutics, Inc. - Chief Commercial Officer

* Michael F. MacLean

Akcea Therapeutics, Inc. - CFO

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Conference Call Participants

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* Gil Joseph Blum

Needham & Company, LLC, Research Division - Analyst

* James William Birchenough

Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst

* Nathaniel Tower

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

* Ritu Subhalaksmi Baral

Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst

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Presentation

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Operator [1]

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Good afternoon, and welcome to the Akcea Therapeutics Third Quarter 2019 Conference Call. As a reminder, this call is being recorded. I will now turn the call over to Kathleen Gallagher, Akcea 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, Faith. Hello, everyone. Thank you for joining today's call. With me today are Damien McDevitt, our interim Chief Executive Officer; Mike MacLean, our Chief Financial Officer; and Kyle Jenne, our Chief Commercial 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 and development. Any statements describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of TEGSEDI, WAYLIVRA and our pipeline of 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 in the 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 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 to 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. - Interim CEO & Director [3]

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Thank you, Kath. Good afternoon, everyone, and thank you for joining today's call. I want to start off by sharing how excited I am to lead Akcea. I believe in our mission, our team and our innovative medicines. Since being appointed interim CEO a little more than a month ago, I've worked alongside our dedicated employees, and I've heard firsthand the real and positive impact we're having on patients' lives. I have now seen the strong foundation that has been built here, and I'm impressed with the talent and engagements across the organization. I've also spent time with our key partners, Novartis, PTC and now Pfizer, and have been impressed with their commitments to our medicines.

Our strategy is to continue to execute on our 2 commercial launches as well as deliver on the transformational programs that we currently have in development. I look forward to leveraging this infrastructure as we expand our pipeline and build upon our relationship with Ionis. As we announced, when I joined Akcea, we have a firm commitment from Ionis to license rare disease medicines that are complementary to our pipeline, and we will continue to evaluate licensing additional third-party assets.

To ensure we can successfully execute this strategy, we've added a key member to our executive team. I am pleased to introduce our new Chief Commercial Officer, Kyle Jenne. Kyle has been with Akcea since 2017, leading the U.S. commercial organization for both TEGSEDI and WAYLIVRA, and has deep knowledge of the ATTR and FCS communities. He brings a wealth of experience to his new role, including more than 20 years in the industry, of which almost half were in the rare diseases space. With the team we have in place, we have the background and experience to drive our company's growth.

This quarter, we also entered into a worldwide exclusive licensing agreement with Pfizer for AKCEA-ANGPTL3-LRx to treat patients with certain cardiovascular and metabolic diseases. Through this agreement, Akcea and Ionis will receive a $250 million upfront license fee and are eligible to receive development, regulatory and sales milestone payments of up to $1.3 billion and tiered double-digit royalties. This agreement is still subject to HSR clearance.

We were thrilled to announce this deal. AKCEA-ANGPTL3-LRx has the potential to treat very broad patient populations, and advancing this medicine to patients is a task well suited for a large pharma company. Pfizer is the ideal partner for this program because of their expertise in both cardiovascular health and in commercializing therapies targeting large markets.

TEGSEDI continues with steady progress. We reported approximately $12 million in sales for the third quarter, which is an approximately 20% growth quarter-over-quarter. We are seeing some promising progress in the market, which Kyle will speak to in more detail.

In the third quarter, we launched WAYLIVRA in Germany. WAYLIVRA is the only treatment approved for patients with FCS, and we are now treating our first patients with commercial drug. We've made a lot of progress with the pipeline, which I will discuss later in the call.

There is a strong sense of momentum across the company. Even in my short tenure, we have had several positive achievements. Commercially, we've launched TEGSEDI in additional countries, and we've had success with a number of European pricing negotiations. We have further validation of our LICA technology with the recent Pfizer licensing of AKCEA-ANGPTL3-LRx, and we're progressing quickly towards the start of the Phase III program for AKCEA-TTR-LRx. Also, we have added experienced leaders to our team, and we have a strong balance sheet that allows us flexibility to continue to build the business. Together, these events and this momentum are positioning us for a promising future.

I'll now turn the call over to Kyle to discuss our commercial efforts for the quarter.

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

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Thank you, Damien, and hello, everyone. It's a pleasure to be here. I'm looking forward to meeting with you in person very soon.

Over my last couple of years at Akcea, I've had the opportunity to build the U.S. commercial team launching TEGSEDI. I've had the pleasure of working with the Akcea team to build the foundation that will support the future launches for WAYLIVRA and additional medicines that receive regulatory approvals.

The focus has always been on ensuring that patients have access to TEGSEDI and WAYLIVRA, and we have done that through building partnerships with physicians, patient advocacy organizations and payers. I believe in listening to the patient and physician communities to understand how we can best serve them. I will lead with that core philosophy as I take on this new role.

Last month marks 1 year since we launched TEGSEDI. We are seeing steady progress. Looking back at the last year, the team has executed well in an unprecedented, competitive environment for our rare disease launch. Through Q3, TEGSEDI has generated revenue from the U.S. and Germany and is now commercially available in 7 additional countries.

In the U.S., we continue to have success with market access for TEGSEDI. We continue to help patients gain access to our treatment through broad reimbursement coverage from commercial and government payers. Importantly, patients are being treated with TEGSEDI with low out-of-pocket cost.

We have continued to focus on raising awareness of the disease. We are seeing our efforts pay off with both neurologists and cardiologists better understanding the urgency to treat the polyneuropathy of hATTR amyloidosis and the importance of starting treatment early.

Our strategy to focus on academic centers and community-based physicians and institutions has resulted in an increase in new positions prescribing TEGSEDI.

We are also seeing this increase in diagnosis from our genetic testing program, hATTR Compass. We now have over 1,000 physicians using the program. Compass allows physicians to diagnose patients locally, and TEGSEDI allows them the independence to treat patients locally as well. This means physicians can treat patients in their local practice, and patients don't have to travel far distances for diagnosis or treatment. This is especially important for patients who are experiencing the debilitating symptoms of autonomic neuropathy, which makes travel incredibly difficult.

This past quarter, more than 40% of our new prescriptions were from first-time prescribers. Importantly, prescriptions from both cardiologists and neurologists are growing, and we are seeing repeat prescriptions from physicians who have experience with TEGSEDI. Coupled with new and repeat prescriptions, we are pleased with patients' compliance and persistence rates.

Outside of the U.S., we have completed the initial free pricing period and finalized pricing negotiations in Germany. We have received acceptance by both NICE and the Scottish Medicines Consortium. With access in all 4 U.K. countries, we are now treating patients there with TEGSEDI.

In addition, we successfully completed our reimbursement negotiations in Sweden and Austria and have launched in both countries. As expected, we are following the typical launch sequence in Europe and anticipate launching in additional countries in 2020.

AKCEA CONNECT is now a global program. We believe we are providing the highest level of patient and physician support across all commercial geographies. We continue to hear feedback that the AKCEA CONNECT team are strong partners for both patients and physicians. In the countries where we are pursuing reimbursement, we have early access programs up and running. Our partners in Latin America, PTC Therapeutics, recently announced the approval in Brazil. We are pleased to be the first approved RNA-targeted therapy for patients with the polyneuropathy of hATTR amyloidosis in Brazil and the only hATTR therapy globally for patients suffering with polyneuropathy to have quality of life mentioned in the indication statement. PTC immediately started launch activities for TEGSEDI in Brazil. Our partnership strategy has been a great way to accelerate the delivery of TEGSEDI to patients in Latin America, and they are working toward additional approvals and reimbursement in other countries. We are very pleased with all we have achieved with TEGSEDI in the past year, and we are looking forward to growing the business in 2020 and beyond.

Turning to WAYLIVRA. The team achieved a major milestone by launching and treating patients in Germany this past quarter. We have begun an ATU, which is a reimbursed early access program in France. We have a strong EAMS program in the U.K. as well as a number of early access programs in other European countries as we pursue reimbursement. Preparations are underway to launch in additional EU countries in 2020 and in Latin America with our partner, PTC. Our team is executing on both the TEGSEDI and WAYLIVRA launches, and the foundation that we have in place internationally is setting us up to commercialize additional products from our pipeline in the coming years.

I'll now turn the call over to Mike to discuss our financials.

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Michael F. MacLean, Akcea Therapeutics, Inc. - CFO [5]

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Thank you, Kyle, and good afternoon, everyone. For Q3, we had total revenue of approximately $21 million and an operating loss of approximately $36 million on a non-GAAP basis, including non-GAAP operating expenses of $57 million. Our revenue includes approximately $12 million of product sales from TEGSEDI, which, as Damien mentioned, is an approximately 20% growth on a quarter-over-quarter basis. In addition, this quarter, we launched the WAYLIVRA in Germany, where the maintenance price of WAYLIVRA is approximately EUR 429,000 per year for a biweekly treatment regimen. WAYLIVRA will be sold as a single dose in prefilled syringes priced at EUR 16,500.

Our market access team is now working with the German authorities to agree to reimburse -- the reimbursement price for WAYLIVRA after the first 12 months. As Kyle mentioned, we have an ATU program for WAYLIVRA in France. We are billing and collecting for the product under the ATU program. And once we finalize our price in France, we will be able to recognize revenue.

We generated a substantial upfront payment of $250 million when we licensed Akcea angiopoietin-like 3-LRx to Pfizer last month. While we split all the economics under this agreement 50-50 with Ionis, we will settle our obligation to Ionis with 6.9 million shares of Akcea common stock, resulting in an ownership by Ionis of approximately 77%. Of the $250 million payment we anticipate receiving from Pfizer, we plan to recognize substantially all as revenue in the fourth quarter.

Further, PTC announced in October that they received approval from TEGSEDI -- for TEGSEDI in Brazil. With the approval, we earned a $4 million milestone. 60% of that amount will go to Ionis per the terms of our profit share agreement. We ended the third quarter with $253 million in cash and short-term investments. When added to the $250 million upfront license fee from our worldwide exclusive license agreement with Pfizer expected upon completion of the HSR review, we will have approximately $500 million in cash and short-term investments. With our substantial liquidity, we are well positioned to continue to execute on our strategy and broaden our pipeline.

I will now turn the call back to Damien.

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

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Thank you, Mike and Kyle. Let me start with an update on WAYLIVRA in the U.S. We remain encouraged by the safety and efficacy profile demonstrated by WAYLIVRA in Europe. In the U.S., we are continuing to work to find a path forward, and we believe that our experience in Europe could be helpful in progressing our discussions.

WAYLIVRA's Phase III APPROACH study was published in the August 8 issue of the New England Journal of Medicine. We were pleased to see the journal recognize the significance of the APPROACH data and the potential of WAYLIVRA to address an area of significant unmet need in the treatment of FCS.

We also announced positive results from the BROADEN study in familial partial lipodystrophy, or FPL, on our last earnings call. We continue to review the data with key experts in the field as we define our next steps.

Moving to AKCEA-TTR-LRx. Akcea and Ionis presented data from the Phase I clinical trial in a poster presentation at the Heart Failure Society of America Annual Meeting as well as at the European Amyloidosis Meeting. AKCEA-TTR-LRx is being developed with Ionis and will be studied in patients with both the hereditary and the wild-type forms of TTR amyloidosis or ATTR. The Phase I data demonstrates an impressive and meaningful reduction in the TTR protein while maintaining a positive safety and tolerability profile. With monthly treatment, we saw TTR reductions of up to 94% at the highest dose. Importantly, adverse events were mild, with the exception of 1 moderate headache. No adverse events led to an interruption in treatment, and there were no severe adverse events. These results are encouraging as we expand our commitment to the TTR amyloidosis community through the development of AKCEA-TTR-LRx. We continue to hear from physicians that the convenience of monthly self-administration would represent a significant advantage for patients living with this debilitating and stable disease.

We also presented the design of our Phase III program for AKCEA-TTR-LRx. The program includes 2 studies: one in patients with the hereditary form of TTR polyneuropathy; and the other in patients with wild-type and hereditary forms of TTR cardiomyopathy. The hereditary polyneuropathy study will consist of 140 patients and will compare AKCEA-TTR-LRx to the historical placebo from TEGSEDI's NEURO-TTR study. The primary endpoint analysis of the study is at 66 weeks to align with the placebo group from NEURO-TTR. The cardiomyopathy study is an event-driven outcome study. It will enroll approximately 750 patients and will compare AKCEA-TTR-LRx against standard of care, including patients taking tafamidis. In both studies, we are close to having sites up and running. We anticipate that the first patients will be treated soon.

Finally, just touching briefly on the rest of the pipeline. Novartis is initiating the AKCEA-APO(a)-LRx Phase III cardiovascular outcome study and continues to anticipate treating patients early in 2020. Phase II studies of AKCEA-APOCIII-LRx and Akcea angiopoietin-like 3-LRx are ongoing, and we anticipate data from both early in 2020.

With over 1,100 patients treated across all LICA programs, we are very impressed with the robust efficacy and with the safety and tolerability profile of the platform to date.

We are executing on our 2 commercial launches. We are initiating our 2 Phase III programs, and we have 2 drugs nearing Phase II data as we head towards the end of the year. With launches in additional countries on the horizon for TEGSEDI and WAYLIVRA, the commitment from Ionis to license us additional rare disease medicines that are complementary to our pipeline and the progression of our current pipeline, Akcea is well positioned for the future.

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 the line of Gil Blum from Needham & Company.

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Gil Joseph Blum, Needham & Company, LLC, Research Division - Analyst [2]

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Just a first one for Kyle. Do we have any indication of maybe the proportion of patients on TEGSEDI who come from the community setting versus the academic?

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [3]

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First...

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Damien McDevitt, Akcea Therapeutics, Inc. - Interim CEO & Director [4]

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This is Damien. Handing it over to Kyle. Go for it, Kyle.

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [5]

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Yes, thanks for the question, and definitely look forward to connecting with you in person here in the near future.

So our efforts have been split between the academic settings as well as the community settings, as I referenced in my comments. We're not going to get into specific numbers in terms of where those are coming from, but we are seeing both community-based and academic-based physicians treating with TEGSEDI within the hATTR polyneuropathy space. So we're excited about that and pleased with the progress that we're making.

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Gil Joseph Blum, Needham & Company, LLC, Research Division - Analyst [6]

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All right. And a bit about -- so as you might have noted, Pfizer released some interesting numbers about diagnosis ratio and wild type. And it appears that they're getting to a pretty significant amount of diagnosed patients. Do you guys think that in a longer-term sense, this is growing the overall TTR market faster than expected?

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [7]

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Yes. So we are seeing the market expand. I think as Pfizer referenced, we're seeing growth, but not only in the wild-type space, but we are seeing increased diagnosis in the hereditary space as well. I think it's a competitive marketplace. There's a lot of education around the hereditary forms of the disease and polyneuropathy specifically. And we're seeing physicians begin to understand and diagnose the patients earlier with neurologic symptoms and looking to treat patients with hereditary forms of the disease sooner than what we had expected prior.

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Gil Joseph Blum, Needham & Company, LLC, Research Division - Analyst [8]

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Great. And just one last one. I know that the Vascepa AdCom is coming up next week. And are you guys expecting any read-through's to your own triglyceride-lowering program?

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

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I'm going to hand that -- thanks for the question. I'm going to hand that over to Kath.

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Kathleen Gallagher, Akcea Therapeutics, Inc. - VP of Communications & IR [10]

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Gil, obviously, we're always watching the competitive environment. I think our program is an ApoC-III inhibitor. And I think as we think about the ApoC-III LICA program, we're focused on the differentiated fact that we decreased not only triglycerides but ApoC-III as well. So we'll be watching as well, but we're really focused right now on our own Phase III data, which we're expecting to come out early next year.

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Operator [11]

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

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Unidentified Analyst, [12]

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This is [Jamison] on for Do. Congrats on the quarter. So a quick question on ANGPTL3. What indications are you thinking about going after? And how has talks been with Pfizer?

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

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Thanks, [Ting]. This is Damien here. I'll take the question. So we're very, very excited about the transaction with Pfizer for ANGPTL3. As you heard, we're receiving $250 million upfront. This sets a new high watermark for a medicine at the stage of development with our technology. We're eligible to receive very significant milestones, development, regulatory and sales milestones up to $1.3 billion, and we're getting tiered double-digit royalties on sales. And so Pfizer is a great partner for this medicine because this medicine can address broad patient populations. So we're looking at both cardiovascular and metabolic diseases. And because this is Pfizer's medicine, we're going to leave it to them to disclose which indications they're going to progress the medicine forward for.

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Operator [14]

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Your next question comes from the line of Jim Birchenough from Wells Fargo.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [15]

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Congrats on all the progress in the quarter. A couple of questions. I guess, first, just with TEGSEDI. Could you maybe comment on any leading indicators of continued growth, either with specifics or just general comments? If you think about patients in the queue, patients that have been captured by AKCEA CONNECT, patients that have been identified and you look at how those patients in prior quarters have flowed to becoming paying patients, could you maybe comment on the trends, if you can't give specific numbers? And I've got a follow-up.

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

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Thanks, Jim. Thanks for the question. I'll hand that over to Kyle.

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

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Yes, Jim. I look forward to meeting you as well in person here, hopefully, fairly soon. As it relates to the progress that we're making in leading indicators, obviously, we're very pleased with the progress. We've got 20% quarter-over-quarter growth in terms of revenue. So we're starting to see the build. And it's happening with existing prescribers. It's happening with new prescribers. It's happening across specialties, in cardiology as well as neurology. And we are obviously broadening and deepening the physician experience with TEGSEDI. So we are optimistic about the repeat prescriptions that we're seeing and the growth that we've seen quarter-over-quarter in terms of revenue.

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Michael F. MacLean, Akcea Therapeutics, Inc. - CFO [18]

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Jim, Mike here. I would just add, Kyle also pointed out that we are moving through the reimbursement cycle in EU countries. So those countries will start to come online here late in '19 and throughout 2020.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [19]

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And then just any comments on -- if you look at the U.S. market, in particular, as you're heading into the fourth quarter, are you seeing good growth in patients identified, in patients moving through the AKCEA CONNECT system? Just trying to get a sense of those type of leading indicators.

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Kyle Jenne, Akcea Therapeutics, Inc. - Chief Commercial Officer [20]

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Yes. And I'll speak specifically to the COMPASS genetic testing program. I think the fact that we have over 1,000 physicians now utilizing that program and we're continuing to add new patients identified through that program and positive patients being found through that program are indicators that the education around diagnosis as well as the effort from the physician community to actually seek out hereditary forms of the disease is working, so from an education standpoint.

On the AKCEA CONNECT side of things, we're very pleased with the way that the program is operating in the United States. We're getting very good feedback from physicians and patients as it relates to the support that AKCEA CONNECT provides and the difference that it's making for them to be able to get on and stay on therapy.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [21]

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And then maybe just a question for Damien. Just strategically, as you think about your ability to bring in additional assets from either Ionis or third parties, could you maybe talk about the decision criteria you have for assessing an asset and whether you choose one from Ionis or from a third party?

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

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Sure, Jim. Thanks for the question. So in terms of the Ionis pipeline medicines, so we're having discussions with Ionis about their wholly-owned candidate medicines, many of which I'm very familiar with, having worked at Ionis before.

Our focus is really on rare disease medicines that complements our infrastructure, our pipeline and our business focus. But we're keeping an open mind as we work through the evaluation of the medicines with Ionis. So as you know, this will be an arm's length business development transaction, and we're not giving sort of a time line on this, but we're making good progress, and we'll announce when the transaction is completed.

In terms of third-party medicines, we're looking, again, for medicines that will be complementary to our pipeline, our rare disease business focus. We, again, are keeping a very open mind as to the stage of the medicine, the modality of the medicine, and we'll update you in due course on progress with that.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [23]

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Maybe just to spread it around, one final question for Mike. Could you just remind me or remind us, rather, how revenues are booked in Brazil with PTC?

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Michael F. MacLean, Akcea Therapeutics, Inc. - CFO [24]

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Yes. So for PTC, the way that, that's going to work is there's an initial period. So it's a 25% royalty, but there's a period at the time of the launch that goes to the either 12 months or $10 million. So for the first 12 months, if they don't exceed $10 million, we do not get a royalty. And in the first 12 months, if they go over $10 million, we get a royalty for the amount -- of 25% of the amount that's over $10 million. And then it kicks into just a regular royalty stream.

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Operator [25]

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

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Nathaniel Tower, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [26]

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This is Nate on for Paul. On the cardiomyopathy Phase III study for TTR LICA, I noticed it's on top of standard of care. I'm just wondering, how are you going to handle tafamidis usage? Do you have a cap on the number of patients that can have baseline usage or any requirements around the duration of therapy prior to enrollment?

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

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So thanks for the question, Nate. Yes. So this study is a large study, 750 patients looking at cardiovascular death and cardiovascular clinical events. It's got wild-type and hereditary ATTR patients, and they're going to be, as you mentioned, on available standard of care. And then half of them are going to have TTR LICA added. The other half is going to have placebo added. And then it's 120-week readouts on that. So that's the design of the study. That's what we're -- we've set up, ready to go.

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Kathleen Gallagher, Akcea Therapeutics, Inc. - VP of Communications & IR [28]

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Nate, this is Kath, just following up on that. I think there isn't a cap on the number of patients that can be on tafamidis. And I think the study design, what we're excited about standard of care arm, is that we'll get a number of different readouts from this study. So we'll see some combination data, we'll see some direct head-to-head data, and that is part of the design because, obviously, we'll be doing a study in a number of countries where tafamidis is available and a number of countries where it's not. So I think that is really part of the design that we're really excited about.

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Nathaniel Tower, Stifel, Nicolaus & Company, Incorporated, Research Division - Associate [29]

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Got you. That's helpful, Kath. And then for both the Phase IIIs, can you guys talk about powering? Have you -- what's the story there?

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Kathleen Gallagher, Akcea Therapeutics, Inc. - VP of Communications & IR [30]

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No, we haven't gotten any of the powering yet, Nate. And I'm sure we'll be sharing more on the study, not necessarily powering, but we'll be sharing more on the study. It actually launches here imminently.

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Operator [31]

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(Operator Instructions) Your next question comes from the line of Ritu Baral from Cowen.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [32]

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First one, I guess, Damien, can you -- just a follow-up on Jim's question about how you plan on building out the pipeline. Is there a stage of development that you think is ideal for the Akcea value proposition? And even more specifically within rare disease, are you guys thinking about commercial synergies with the existing promotional effort? Or are you willing to sort of strike out in a new direction? And then I've got a follow-up.

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Damien McDevitt, Akcea Therapeutics, Inc. - Interim CEO & Director [33]

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Okay. Thank you, Ritu, for the question. Yes, I mean, I think we're keeping an open mind to the stage of development, but obviously, closer to the market is more interesting. And with the Ionis pipeline, we're looking at their mid-stage pipeline of wholly-owned assets with them together.

In terms of, yes, we're staying focused on rare diseases. That's where our strength is. That's where our mission is and looking for medicines that will complement our infrastructure, our commercial capabilities and the business focus.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [34]

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Got it. And then as you think about your LICA TTR Phase III, both the FAP and the FAC and wild-type of study, are there sort of -- are there elements that from a commercial aspect will differentiate LICA TTR from other knockdown agents? Are there any secondary endpoints where you believe will contribute to a differential profile?

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Damien McDevitt, Akcea Therapeutics, Inc. - Interim CEO & Director [35]

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Let me start on that. The -- if I understand the question right, it's around TTR LICA Phase III. So I think what I would say is that we're excited about the fact that it's a monthly injection. And we've heard from physicians community that monthly is most convenient for patients. Also, what we're very excited about is that it's basically TEGSEDI with a GalNAc, and that gives us great confidence given the positive improvements we've seen for patients taking TEGSEDI.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [36]

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Got it. And as far as secondary endpoints or anything within the trial design, anything to differentiate it from potential future competition in the field, whether we're talking TTR (inaudible)?

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Damien McDevitt, Akcea Therapeutics, Inc. - Interim CEO & Director [37]

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Okay. Kath -- I'm going to ask Kath to handle that.

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Kathleen Gallagher, Akcea Therapeutics, Inc. - VP of Communications & IR [38]

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Sure. Ritu, so I don't think there's anything specific in there, Ritu. I mean, to some degree, these are -- the studies are what they are. I think what's important is that we brought all of our collective learning. At this point, we have a lot of knowledge on TTR. And we can take everything that we've learned, especially from the patient experience, and we can put that into the study in a way that it is typical in rare disease. We didn't have that information when we launched the NEURO-TTR study. And so I think that this puts us in a pretty good position of strength going in, thinking about quality of life metrics and really thinking about what are the important things that we're looking at, which I think, in general, as a community, we've learned more over the last few years to kind of strengthen these future trial designs.

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Operator [39]

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And that concludes our Q&A session. I would now like to turn the conference back to you, Mr. Damien McDevitt.

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Damien McDevitt, Akcea Therapeutics, Inc. - Interim CEO & Director [40]

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Thank you 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 [41]

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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.