Earnings Labs

Ionis Pharmaceuticals, Inc. (IONS)

Q4 2014 Earnings Call· Fri, Feb 27, 2015

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Transcript

Operator

Operator

Good morning and welcome to Isis Pharmaceuticals’ Year End Financial Result Conference Call. All participants will be in listen-only mode. [Operator Instructions]. After today’s presentation, there will be an opportunity to ask questions. Please note this event is being recorded. Leading the call today from Isis is Dr. Stan Crooke, Isis’ Chairman and CEO. Dr. Crooke, you may begin.

Dr. Stan Crooke

Analyst

Thank you. Good morning everybody, and thanks for joining us on today’s call to discuss our year-end financial results. As we begin, I do want to acknowledge that tomorrow is International Rare Disease Day. We do have a large and important and exciting pipeline of treatments for severe rare diseases. And everyone at Isis is committed to bringing these important new medicines to these patients who clearly need them. On the call today Lynne will share with you our 2014 successes and provide an update on the progress we’re making with our new lipid focused subsidiary Akcea Therapeutics. Beth will review our 2014 financial results and provide guidance for 2015. And then I’ll finish by putting the events that already happened this year in the context and give you a preview on what to look forward to in the remainder of 2015. Joining me on today’s call are Lynne Parshall, Chief Operating Officer; Beth Hougen, Chief Financial Officer; Sarah Boyce, Chief Business Officer and Paula Soteropoulos, Chief Executive Officer of Akcea Therapeutics; and finally, Wade Walke, Vice President of Corporate Communications and Investor Relations. And so, Wade, will you read our forward-looking language statement please?

Wade Walke

Analyst

Thanks, Stan. A reminder to everyone that this conference call includes forward-looking statements regarding the financial outlook for Isis, Isis’ business and the therapeutic and commercial potential of Isis’ technologies and products and development. Any statement describing Isis’ goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential in KYNAMRO, ISIS-APOCIIIRx, ISIS-MNRx and ISIS-TTRRx 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 to the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor building a business around such drugs. Isis’ 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 Isis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis’ programs are described in additional detail in Isis’ Annual Report on Form 10-K for the year-ended December 31, 2013, and in its most recent quarterly report on the Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the company. Now, I’d like to turn the call over to Lynne.

Lynne Parshall

Analyst

Thank you, Wade. 2014 was another year of growth for Isis. Pleased with value of the advancements we’ve made in our technology, our pipeline and our business strategy have been recognized by the market, creating value for patients and shareholders. We had successes in every element of our business and those successes have established a strong base and upon which to build in 2015. Isis is a leader is a leader in developing RNA targeted drugs. We have 38 drugs in development. We are the only company to have commercialized RNA targeted drugs. Our pipeline continues to mature and today includes several near-term commercial opportunities. We have six different drugs in Phase 3 studies for nine different indications including three drugs for which we are conducting the Phase 3 studies, ISIS-SMNRx, ISIS-TTRRx and ISIS-APOCIIIRx. These drugs are focused on orphan indications in which patients have limited or no therapeutic options. Each of these has Phase 3 data planned for 2016/2017. Two of these drugs are partnered with major pharmaceutical companies, Biogen Idec and GSK who are adding considerable value to the programs including preparing them for approval and commercialization. In fact, Biogen and GSK each plans to initiate additional studies to support these drugs which they will be conducting in parallel with the work we’re doing. The third drug, ISIS-APOCIIIRx is in our sweet spot, lipids. And this is an asset we have put into our new subsidiary Akcea Therapeutics. With Akcea, we plan to build the world class lipid therapeutics company with the unique line of first-in-class drugs to treat lipid disorders. In December, the groundbreaking Phase 2 data from two of our programs was highlighted in two New England Journal of Medicine articles, one ISIS-APOCIIIRx and on ISIS-Factor XIRx. ISIS-Factor XIRx publication showed that inhibiting Factor XI, a…

Beth Hougen

Analyst

Thank you, Lynne. In 2014, we significantly improved upon our pro forma NOL guidance and substantially exceeded our cash guidance. We ended the year in a strongest financial position in our history with nearly $730 million in cash and pro forma NOL of $16 million and we reported net income $32 million in the fourth quarter. Significant contributors to our profitable fourth quarter were nearly $70 million in milestone payments and more than $30 million related to our ownership in Regulus. These results illustrate that we are effectively executing our business strategy and that our business model is working as designed to consistently generate cash revenues as partnered programs succeed. Our strong financial position has allowed us to develop and expand our pipeline and to make important advances in our technology, all while maintaining a moderate expense level. Today in our pipeline, we have one drug for every 12 Isis employees. This level of productivity is only possible because of the efficiency of the antisense technology we have developed and continue to advance coupled with our business model by which we leverage our partners’ resources. Our pro forma NOL of $16 million in 2014 was significantly better than our guidance of a pro forma NOL I the low $50 million range. Our revenue was $214 million, up 34% increase over our guidance, reflecting against the successes we had with our partnered programs during the year. The most significant component of revenue for us is milestone payments we earned as our programs advance. And last year, nearly two-thirds of our revenue came from milestone payments including $22 million from AstraZeneca, $22 million from GSK and $80 million from Biogen Idec. And we continued to experience in our existing partnerships. So, far this year, we have achieved $27 million in milestone payments…

Dr. Stan Crooke

Analyst

Thanks Beth. So, we certainly are pleased with where we are financially. And we’re also pleased with the trajectory. We look forward to where we’re heading. So, let me begin by putting a few recent events in context for you. And hopefully that will allow you to better appreciate how I think the progress this year is likely to generate increased value for patients and for shareholders. Just this week, we reported clinical results for ISIS-PKKRx. This new addition to our severe and rare disease portfolio is for the treatment of patients with hereditary angioedema HAE. We think that this is a program that with our capabilities and our resources, we can easily move forward IRR. HAE is characterized by rapid and painful attacks and inflammation throughout the body including face, larynx and trachea. The attacks are always painful and debilitating and can be fatal. HAE is caused by inappropriate activation of prekallikren or PKK. Our drug is designed to effect the cause of the disease by reducing production of PKKs and directly therefore blocking biological processes that lead to HAE attacks. In our early work, we evaluated several steps in the disease to the pathway to find the best target. That’s one of the great strengths of the antisense technology; it’s possible to look at everything. And not surprisingly, PKK appear to give us the best results. It enabled us to target the source of the disease. So, in our preclinical studies, we observed significant improvements in disease models of HAE, 40% to 50% with only 40% to 50% reduction of PKK. And we recently completed this initial clinical trial in healthy volunteers. And in this study, we observed a significant dose dependant reduction to PKK of upto 95%, and great safety and tolerability for the drug. So, this…

Operator

Operator

[Operator Instructions].

Operator

Operator

Our first question today comes from Jim Birchenough with BMO Capital.

Jim Birchenough

Analyst

Hi, guys. Congratulations on all the progress. Just on the TTR update we’ll get at AAN, could you maybe give us a better sense of what we should be looking for in those two podium presentations? And then just had a couple of follow up questions.

Dr. Stan Crooke

Analyst

As you know, of course Phase 3 study is blinded. So, won’t be reporting any Phase 3 data. But we do have some ancillary studies that are in progress in patients. And that will give us the opportunity to talk about both the ability of the drug to reducing targeting patients and also its tolerability.

Jim Birchenough

Analyst

Will there be any functional data Stan, anything on their conduction that sort of thing?

Dr. Stan Crooke

Analyst

I think I’d like to leave it there, Jim. So, the presentations are just being put together now, and I haven’t actually seen the actual presentation. So, I would leave it there.

Jim Birchenough

Analyst

So, maybe bigger picture question, with the advantage you have with the LICA technology, can you go back and conjugate something like KYNAMRO and create a novel second generation drug that you retain yourselves?

Dr. Stan Crooke

Analyst

First of all, we can do exactly that but our deal with Genzyme gives them ownership of KYNAMRO well of the target.

Jim Birchenough

Analyst

And maybe just a final question for Beth, just so we get a better sense of the sustainability of the revenues you see this year and how confident you are in the guidance. Could you maybe discuss how you go about giving revenue guidance when there are so many moving parts around milestones and a lot of it’s contingent on things going right? I’m imagining there is probability adjusting. How do you do that? And then, are these revenues, do you think as model forward that there will be sustainable when you look at in aggregate what’s coming in terms of all these programs in development.

Beth Hougen

Analyst

Sure. So, you’re absolutely right, Jim. When we put together our revenue guidance, we look down the list of all of the opportunities we have to earn revenue from our partnerships, particularly milestones. And we go partner by partner, drug by drug, research program by research program and we look at those milestone opportunities. And we probablize them for timing and the probability of success. And then we aggregate those probablized amounts into our revenue guidance. So, we feel like there is tremendous opportunity to -- we are very confident that we can meet our revenue guidance. And in 2014 we provided guidance of about $160 million to $170 million worth of revenue and we ended the year in excess of $200 million. So, I think the approach that we take is very reasonable and it creates and opportunity for us to achieve our guidance. Does that answer your question?

Jim Birchenough

Analyst

Yes. And just as we look ahead, how sustainable do you think these revenues are? Can you grow off this base when you look in aggregate at what’s coming from all these different programs?

Beth Hougen

Analyst

Definitely. We continue to have tremendous productivity in our pipeline and in our partnered programs. And Biogen alone, we have our research collaborations in neurology that has a tremendous opportunity to continue to generate new targets and new drugs. And all of those as they advance will create milestone opportunities. And across all of our partnerships, we have the ability to earn license fees as our partners exercise their options to move those drugs forward towards the market.

Dr. Stan Crooke

Analyst

We also have a very high level of interest in the technology, the drugs in the pipeline. And so, we are very selective about what we are willing to partner and to whom and for what price? But certainly that’s another source of optimism for us.

Jim Birchenough

Analyst

Okay. Thanks for taking the questions.

Operator

Operator

Next question comes from Chad Messer with Needham & Company.

Chad Messer

Analyst · Needham & Company.

Great. Thanks for taking my question. I’m very interested in this strategy you’re employing for APOCIII, taking your generation 2.0 drug rapidly into now a couple of orphan indications and then using a LICA compound to go after perhaps some broader indications. It seems like this would be a strategy you could potentially reemploy in a number of areas. Could you maybe comment on whether you would agree with that observation and perhaps remind that where you have good LICA follow-on drugs; what programs you have done some more advanced work on LICA 4?

Dr. Stan Crooke

Analyst · Needham & Company.

Well, absolutely agree with what you had to say. And just to recall the basic strategy at Isis in terms of defining what drugs we develop is to create the maximum value in the initial phase of population. And to do that I think there are two steps that are uniquely available to us with the technology we have. The first is we take genetic information directly. Most of the targets that we’re really interested in are genetically validated. And we of course evaluate all of the relevant targets in a pathway and pick the best target as we did with PKK. And then the second step is to identify using very similar approaches, genetics; epigenetics; and phenotype, subsets of patients who we think are most likely to benefit in the greatest way from manipulating that specific target. And so we do that across the board in every part of the pipeline that we can. There are some diseases that don’t lend themselves to that easily but the vast majority do. Now, for the liver targets that we of course we have LICA follow-on opportunities for all of them. We also have LICA 2.5 opportunities and so each of these is a separate equation that we write. And we ask how far long is a parent drug; how -- do we have a good spot for the parent drug; should we replace the parent drug with the LICA. Each one of these opportunities is unique -- opportunity and a unique challenge and therefore, we write a unique equation. And that helps us decide when and how we are going to use the follow-on. With APOCIIIRx, we are far along. We think we’ve got a great looking drug for rare diseases. And with these two rare disease, we’ve got a super market opportunity which prices comparably, should give us great opportunities there and the LICA follow-on has its own place. So, each drug is different. And as the pipeline continues to evolve, you’ll see more and more of the drugs being 2.5 and LICA and 2.5 LICA because those are our best quality drugs. A lot of words, did I answer your question?

Chad Messer

Analyst · Needham & Company.

Yes, mostly. I mean maybe actually to move on to something different. So, I like the flexibility that the strategy of having two actual different drugs out there gives you, both in terms of pricing and then also partnering. Could you comment on whether you would see APOC drug for a larger population, so the LICA follow-on as something you would consider partnering down the road?

Dr. Stan Crooke

Analyst · Needham & Company.

Yes.

Chad Messer

Analyst · Needham & Company.

Okay, great. Thanks. I’ll get back in the queue.

Dr. Stan Crooke

Analyst · Needham & Company.

I made up from my long winded; the short is the answer of the day.

Operator

Operator

Our next question comes from Alethia Young with Deutsche Bank.

Alethia Young

Analyst · Deutsche Bank.

Hey guys, thanks for taking my questions. One is just we know in talking about the partial lipodystrophy -- what have you seen back in the papers and the biology that implicate APOCIII to give you so much confidence? And then kind of in parallel to that, have you spoken to the FDA about your plans in Phase III yet?

Dr. Stan Crooke

Analyst · Deutsche Bank.

So, we have been in contact with the FDA and European regulatory agencies on our APOCIII franchise. We’ve not yet spoken to the FDA on the partial lipodystrophy. But based on the conversations we’ve had with both sets of regulators, we feel there is a lot of enthusiasm for APOCIIIRx and what we’re doing with it. And what we show with APOCIIIRx is that we can lower triglycerides in any patient population and we look at partial lipodystrophy as a triglyceride disease. And lipodystrophy patients have elevated APOCIIIRx to APOCIII. So, it’s I think pretty straight forward to think about the disease mechanistically as it fits appropriately in this space for APOCIIIRx. I think it’s more obvious choice in some ways in FCS where we’ve got the surprise that we were able to produce effects in patients with FCS. But I think it’s fairly straight forward, Alethia.

Alethia Young

Analyst · Deutsche Bank.

And then as far as these patients being in the same clinics, I mean is that why you have confidence that you can enroll these trials at the same time; is that their synergy is with the doctors there in the current clinical trial?

Dr. Stan Crooke

Analyst · Deutsche Bank.

That’s a part of it. The patient group is larger, so we feel that with the expertise that we have in lipid franchise, the fact that we know the centers, know the KOL, know the patients, we can take advantage of all of that as the value of actually having a franchise. And there is such excitement about APOCIIIRx in the community. And of course in doing that it also sets us up kind of thinking of a much better and be prepared to learn from the processes compared to commercialize. I think that’s all of this, the thing excites you most about this…

Alethia Young

Analyst · Deutsche Bank.

And just one last logistical one; you said that the discontinuation seemed little lower I think in the TTR, are you willing to disclose what your assumptions were heading into designing the Phase 3 study?

Dr. Stan Crooke

Analyst · Deutsche Bank.

No, but I will tell you, I will give you some color unless Lynne interrupts me and tells me I can’t. If you look at the study, I think the dropouts were like 40. Is that right Lynne, 40%, 50%, somewhere in that range?

Lynne Parshall

Analyst · Deutsche Bank.

40, yes.

Dr. Stan Crooke

Analyst · Deutsche Bank.

And with KYNAMRO, we had dropouts in one year studies; they were in the 20-plus range. And so, we modeled dropouts, it would be appropriate to those two considerations. And we’re seeing far less.

Alethia Young

Analyst · Deutsche Bank.

Okay, great. Thanks for taking my questions.

Dr. Stan Crooke

Analyst · Deutsche Bank.

Lynne, do you want to add or subtract anything from that?

Lynne Parshall

Analyst · Deutsche Bank.

No, I think that’s great.

Operator

Operator

Our next question comes from Stephen Willey with Stifel.

Stephen Willey

Analyst · Stifel.

Yes. Thanks for taking the questions. Just wondering if there is anything you can say about the proposed design GSK is contemplating in FAC with TTR just in terms of I guess to endpoint specifically, and whether or not they’re going to be pursuing something ventured and/or more functional based?

Dr. Stan Crooke

Analyst · Stifel.

Well, we certainly can’t share any of that today, Steve. The protocol, the study is getting underway here and we’ll be able to share that a little later but not today. Lynne, do you want to add anything to that.

Lynne Parshall

Analyst · Stifel.

Yes. GSK has asked us because they view it as sort of a competitive advantage, not to talk about it until the study starts. But we will be enthusiastic about talking of that, when they’re ready to start the study.

Dr. Stan Crooke

Analyst · Stifel.

We’re very pleased with -- I mean we’ve had of course quite a bit of time to really understand the space thoroughly as has GSK. And so, at least from the Isis side, we’re quite excited about the decisions that GSK is waiting.

Stephen Willey

Analyst · Stifel.

Okay. And then, I know that there’s been obviously discussion about the improved 2.5 chemistry, but just kind of wondering when we might see kind of more comprehensive safety data from one of those studies. I know that we’ve done a handful of STAT3 data and I think there is some more (a)R and DMPK readouts scheduled for the end of the year. But I’m just kind of wondering what kind of dose durations are we going to see, I guess specifically from DMPK that may allow us to better assess the safety of 2.5?

Dr. Stan Crooke

Analyst · Stifel.

STAT3 is not terribly helpful because you’ve got STAT3 target adverse events that were predicted and have been encountered. With androgen receptor, that’s been very helpful because the target toxicity is reducing androgen receptor a minimal. And AstraZeneca has taken the drug to super high doses in patients with cancer. And so, we’ve had a chance to look at the dose of that drug upto 2,700 milligrams a week, which probably a 150 times what we would dose for any target. And we haven’t encountered any safety issues there yet. The DMPK work is -- we have normal volunteer experience and now we’re gaining patient experience and that’s had normal doses. And so, we’ll have very high dose experience, although for quite a number of months in patients with androgen receptor and then, real world dose experience for somewhat shorter period but months in both normal volunteers and myotonic dystrophy patients.

Stephen Willey

Analyst · Stifel.

And then I guess as we think about DMPK data towards the end of this year, I think it’s primarily set up to be kind of a safety and PK study, but just wondering if there is any kind of clinical efficacy that’s going to be gleaned out of that and maybe just what some of those endpoints are?

Dr. Stan Crooke

Analyst · Stifel.

Lynne, do you want to answer that? Maybe not. So, there are endpoints that evaluate both target reduction and muscle performance. But you’re right, the primary goal is to demonstrate safety and pharmacokinetics.

Stephen Willey

Analyst · Stifel.

Okay. Thanks.

Operator

Operator

The next question comes from Eric Schmidt with Cowen and Company.

Eric Schmidt

Analyst · Cowen and Company.

Thanks for taking my questions; just two quick ones. Stan, should I assume that since you mentioned AAN as a possible site for the SMA Phase ½ update, we won’t be seeing it at that forum and if that’s the case, when do you think we might see the data?

Dr. Stan Crooke

Analyst · Cowen and Company.

We mentioned AAN with regard to TTR, and that’s what you supplied.

Eric Schmidt

Analyst · Cowen and Company.

Not known, specifically asking about when we’ll see the SNA data. I’m guessing it’s not going to be at AAN since you didn’t mention it?

Dr. Stan Crooke

Analyst · Cowen and Company.

We have an analysis that will be done and it will be done on schedule. And so, there is quite shift with AAN but there are numerous opportunities. So, it won’t be in the conditions, right?

Eric Schmidt

Analyst · Cowen and Company.

Okay, thank you. And Beth, on the revenue guidance, just one follow-up to Jim’s question. Should we therefore assume that the 2015 guidance doesn’t include any new collaborations or do you also try and probability adjust those?

Beth Hougen

Analyst · Cowen and Company.

We probability adjust all of the opportunities for revenue, Eric. So, if there is -- if we do have a collaboration and assume any revenue from a new collaboration, the probability would have been probably fairly low because those are obviously very significant transactions and involve a lot of complexity.

Dr. Stan Crooke

Analyst · Cowen and Company.

The short answer is that new deals are probablized low enough that they really don’t constitute much in our projections.

Eric Schmidt

Analyst · Cowen and Company.

Thanks very much.

Dr. Stan Crooke

Analyst · Cowen and Company.

It’s just proven to do that right.

Operator

Operator

Our next question comes from Jessica Fye of J.P. Morgan.

Jessica Fye

Analyst

On the prekallikren product, can you maybe elaborate on your expectations around the clinical profile for that asset, in terms of what you believe the 90% reduction in prekallikren might mean for impact on attack rates in HEA patients? Also is there anything you can share about what dose or doses you plan to study in Phase 2, whether you expect to stick with once weekly dosing and maybe any color around injection site reactions in the Phase 1?

Dr. Stan Crooke

Analyst

Well, let’s do it in reverse order. Injections are reactions with new groups of generation 2 drugs are really de minimis. We are having almost no problems with injection site reaction site. They mean to apply sub-cu [ph] drugs. They do produce a spot on the skin but nothing like what we face with KYNAMRO. There is a difference. And they are different because we made some of -- the answers allow us to make better drugs. And the more potent, in the PKK study, we had excellent safety and tolerability with really no issues reported. The Phase 2 program, we haven’t decided formally, but we will use dose. I imagine when we look at couple of doses but we haven’t decided yet. And it will be weekly. And our experience with these drugs is what we see in normal volunteers in terms of target reduction, it translates almost one to one in every patient group we’ve looked at. We don’t think we need to reduce PKK by 90% at all. In animal models, we see very good activity of 40% to 50% reduction of PKK. So, we’ll target greater than that. And the whole is that by getting rid of the substrate for the inappropriate activation which these patients test their problem, they don’t have the inhibitor, we should see really very substantial benefit in terms of both the frequency and severity, which acts.

Jessica Fye

Analyst

Okay, great. Maybe one more on Factor XI, I know you’ve talked about potentially partnering that asset. And I think in the prepared remarks you said you are getting interest, but then you also said you will take your time. What does that mean in terms of when we should expect a partnership?

Dr. Stan Crooke

Analyst

Thursday, March 26. It means we’ll tell you as soon as we decide on one. We have a lot of interest and it’s certainly an active conversation that’s in progress today with a number of companies. And we don’t need to partner we think we can handle all the next steps that should be taken. But we want to partner, so that we make the development program as broad as rapidly as possible. And on the other hand, we want to be sure that the partner we pick is a partner we’re going to be happy to live with for a long time and who’s going to make this drug better. So, I don’t know how to make it any more precise than that. Lynne, do you want to give a specific date?

Lynne Parshall

Analyst

Yes, I would say March 27th but no. But I just want to say there is nothing about this program that’s slowing down. As Stan said, we’re pursuing the next steps. So, we are moving the compound forward. And we have the ability to take our time and make sure that we find the right partner with the right development program for the right price. And plan to do that. There is no -- nothing to be gained from rushing.

Jessica Fye

Analyst

Got it. Thanks.

Lynne Parshall

Analyst

That’s a longest non-answer we’ve given today.

Operator

Operator

Next question comes from Mike Schmidt with Leerink

Mike Schmidt

Analyst · Leerink

Hi, good morning. I just had a follow-up on APOCIII. I was wondering -- I’m trying to better understand timelines around the LICA formation for instance for the severe hydro-glyceride population. Do you essentially have to go back to preclinical and Phase I studies before launching a large efficacy study, or is there an accelerated clinical path if you reformulate generation 2.0 drugs into LICA conjugated versions?

Dr. Stan Crooke

Analyst · Leerink

I wouldn’t refer to this reformulation. For me, something very specific that you’re putting in new formation; it’s new chemical entity. We think of it as a pro drug. We have already with LICA follow-on, said that for the lipid franchise, we’ve already done all the preclinical work necessary. And we do believe that we can abbreviate the initial clinical trial work and move very rapidly into efficacy study. But at the moment, we still feel that we need to work our way through an initial clinical trial and normal volunteers for most of the indication we go to patients. But it will be quite disastrous.

Mike Schmidt

Analyst · Leerink

Got it, okay. And then, I think you mentioned Biogen Idec and Glaxo both initiated some additional studies and for ISIS-TTRRx and SMNRx respectively. Could you just comment on what those studies are, and whether or what data we might see for those before Phase 3 trials read out?

Dr. Stan Crooke

Analyst · Leerink

Lynne, do you want to…

Lynne Parshall

Analyst · Leerink

Sure. Biogen Idec is initiating two clinical studies, one of them is a study that includes both new born screening as well as presymptomatic treatments of infants who are at new born screening diagnosed to have MSA. And they’re also initiating a study in patients who are too old for our infant study and too young for our childhood onset study. They’re getting both of those studies up and running. And as Stan mentioned, GSK is initiating the Phase 3 clinical trial in cardiomyopathy form of TTR amyloidosis. So, those are the studies that we were referring to. We may have some data coming out of the Biogen studies. The studies are just starting and they haven’t come with the presentation, publication plan for them yet. But obviously the Phase 3 cardiomyopathy study is the study that we will not have data on before the study that we’re finishing up the Phase 3 study, we are finishing up which is polyneuropathy study. That being said, as Stan said, we do have two podium presentations and we will be talking about the TTR program with data from some ancillary studies that we think is very interesting in terms of the program.

Mike Schmidt

Analyst · Leerink

Okay, got it. Thanks. And then last question for FCS and the partial lipid dystrophy indications, can you comment on how many patients are actually identified versus prevalence that can be a discrepancy sometimes for rare diseases? And I guess what are you doing to -- do you have activities in place to drive diagnoses rates of those rare patients ahead of a potential launch later on? Thank you.

Dr. Stan Crooke

Analyst · Leerink

Paula, maybe you can handle that?

Paula Soteropoulos

Analyst · Leerink

Sure. Mike, this is typically like you would see with other rare diseases where all of the patients are not known and identified. We see with FCS patient population of one in a million and with partial lipodystrophy it’s about two to three in a million. And so, we will be working to help drive diagnosis earlier. Eventually those patients do get found because of manifestation of the disease and they eventually get to a lipid specialist. What we want to do is drive that diagnosis earlier so that we can actually intervene earlier and actually help these patients really affect their disease. So, all of the decisive things that we would do from a commercial activity would be to really understand today how these patients are diagnosed, what is that journey to get them to the right specialist, how do we improve the diagnosis, how do we increase the awareness to -- not go with the specialist, they understand the disease but beyond those physicians actually be referring early and really increase that awareness and leveraging the patient community because they already know the ground flow of these patients in both diseases that are getting momentum with their voice around their disease and will enhance that.

Dr. Stan Crooke

Analyst · Leerink

We learned a lot from KYNAMRO and the work that Paula did when she was at Genzyme and in bringing awareness of the disease forward. And we think that’s helped a lot getting recruitment done in the trials and helped both lomidipide and KYNAMRO get launched.

Mike Schmidt

Analyst · Leerink

Okay, great. Thank you very much.

Operator

Operator

Our next question comes from Yale Jen with Laidlaw & Company.

Yale Jen

Analyst · Laidlaw & Company.

Good morning and congrats on good quarters. And most of my questions been answered, so here just a very brief one. Could you give us a little bit more color in terms of the partial lipodystrophy trial design if you have it and when you anticipate that to start? And then I have a short follow-up after that.

Dr. Stan Crooke

Analyst · Laidlaw & Company.

I’d like to defer that answer for just a little while. We’re finalizing all that now. And we’re working seriously to get the study up and running, I mean as fast as we possibly can. Paula, do you want to add anything to that?

Paula Soteropoulos

Analyst · Laidlaw & Company.

Yes. I think that that’s right. I mean we will get that study up and running as quickly as possible. And we’re in the middle of defining how that study looks like and can we know where the sites, it’s really defining the clinical protocol endpoints.

Dr. Stan Crooke

Analyst · Laidlaw & Company.

Give us just a little bit of time, we’ll share with you.

Yale Jen

Analyst · Laidlaw & Company.

Sure. I appreciate that. And also my understanding is that the partial lipodystrophy has both the familial portion as well as the acquired portion. I know the size of those two is very different. In this study, would you include patients from both sort of cohorts or you’d be more focusing on one of the two?

Paula Soteropoulos

Analyst · Laidlaw & Company.

Initially we’ll be focusing on the familial partial lipodystrophy.

Dr. Stan Crooke

Analyst · Laidlaw & Company.

It’s well defined patient population, so well defined severe rare population. And we think it’s in a size similar to FCS population though it gets larger. So, it fits in terms of the launch, in terms of payers, in terms of pricing and so on. So, one of the things we really like about the decision is that we have a really well-matched set of opportunities that we should be able to take advantage of more effectively.

Yale Jen

Analyst · Laidlaw & Company.

Okay, great. Thanks a lot.

Dr. Stan Crooke

Analyst · Laidlaw & Company.

I think we’ve covered all the questions. If that is the case, I do want to thank everyone for joining us today. We are pleased with where we are as we enter 2015 and we’re pleased with the value that’s been created. But we think the value that’s been created is just a tip of the iceberg. We think a lot more is to come. And we think a number of good decisions have made about the lipid franchise, substantially enhanced its value in addition to the other things that we’re doing that we think will bring added value to shareholders. Thanks very much.

Operator

Operator

The conference is now concluded. Thank you for attending today’s presentation. You may now disconnect.