Alnylam Pharmaceuticals, Inc.
Q2 2017 Earnings Call Transcript

Published: 10 Aug 2017

[Operator:]

Ladies and gentlemen, thank you for standing by, and welcome to the Alnylam Pharmaceuticals Conference Call to discuss Second Quarter 2017 Financial Results. There will be a question-and-answer session to follow. Please be advised that this call is being taped at the company's request. I would now like to turn the call over to the company.

[Christine Regan Lindenboom:]

Good afternoon. I'm Christine Lindenboom, Vice President of Investor Relations and Corporate Communications at Alnylam. With me today are John Maraganore, our Chief Executive Officer; Barry Greene, President; Akshay Vaishnaw, Executive Vice President of R&D; and Manmeet Soni, Chief Financial Officer. In addition, Yvonne Greenstreet, Executive Vice President, Chief Operating Officer, is in the room and available for Q&A. For those of you participating via conference call, the slides we have made available via webcast can also be accessed by going to the Investors page of our website, www.alnylam.com. The press release and related financial tables including a reconciliation of GAAP and non-GAAP net loss that we will discuss today can be found on the Investors page of our website. We believe non-GAAP net loss provides useful information to management and investors regarding our financial conditions and results of operations. During today's call, as outlined on slide 2, John will provide some introductory remarks and provide general context, Akshay will review recent clinical updates, Manmeet will review our financials and Barry will provide a brief summary of upcoming milestones before opening the call for your questions. Before we begin, I would like to remind you that this call will contain remarks concerning Alnylam's future expectations, plans and prospects, which constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recently quarterly report on file with the SEC. In addition, any forward-looking statements represent our views only as of the date of this recording and should not be relied upon as representing our view of any subsequent date. We specifically disclaim any obligation to update such statements. With that, I'd like to turn the call over to John.

[John M. Maraganore, Ph.D.:]

Thanks, Christine, and thank you, everyone, for joining us this afternoon. During the second quarter of 2017 and recent period, we made continued and meaningful progress in advancing our investigational RNAi therapeutics through clinical trials and toward the market. 2017 promises to be a pivotal year for our company, as we open the envelope on our first Phase 3 results and prepare to make the anticipated regulatory filings for marketing approval and, assuming approval, make the transition to a commercial organization. In addition, we aim to start three separate Phase 3 programs in 2017, positioning us to fulfill our Alnylam 2020 plan of becoming a multiproduct, commercial stage company with a deep and sustainable clinical development pipeline by the end of 2020. This is a profile that has been rarely achieved in biotech. Of course, right now, all eyes are on patisiran, our investigational RNAi therapeutic, which we're developing for the treatment of patients with polyneuropathy due to hereditary ATTR amyloidosis. Specifically, we look forward to reporting top line data from the APOLLO Phase 3 trial in the coming weeks, and assuming positive results, expect to submit an NDA by year end with an MAA following shortly thereafter. We believe we have strong reasons to be encouraged about APOLLO, based on the continuing promising results that we've seen from our ongoing Phase 2 open-label extension, or OLE, study of patisiran, where we've seen evidence for potential halting or in fact improvement of neuropathy progression in patients. Moreover, we believe the potential clinical efficacy for a TTR lowering mechanism of action was also validated by the recent positive top line Phase 3 results for inotersen, the investigational antisense oligonucleotide targeting TTR. Accordingly, we very much look forward to seeing the important results from APOLLO. In the meanwhile, we're very much in a build phase preparing for our potential transition to a commercial stage company in 2018. Barry will review some of these activities later, but I also encourage you to listen to the webcast of our patisiran RNAi Roundtable from last week, where we discussed our commercial readiness efforts in much more detail. Without a doubt, we're excited about the medical affairs, manufacturing, and commercial organizations and capabilities we're building to support our initial commercialization efforts in the U.S., Canada, and Western Europe, and we look forward to extending these efforts globally to support givosiran and future programs where we've retained global rights. We've also recently made great progress with our mid- to late-stage clinical programs, including fitusiran for hemophilia, givosiran for porphyria, and, with our partners at The Medicines Company, inclisiran for hypercholesterolemia. The ATLAS Phase 3 program for fitusiran in hemophilia A and B patients, with or without inhibitors, was initiated earlier this summer, and we're pleased to get this program up and going. We're also aiming to start the givosiran Phase 3 study by end of the year, and The Medicines Company has announced agreement with the FDA on a Phase 3 program for inclisiran, with the study in patients with atherosclerotic cardiovascular disease expected to commence in late 2017. Strategically, these three Phase 3 programs, in addition to patisiran, position Alnylam to potentially have a steady flow of commercial launches on essentially an annual basis from 2018 going forward. Of course, we're also very excited about the recent data presentations for what we believe to be the transformative potential of fitusiran, givosiran and inclisiran. Finally, as we shared during our RNAi Roundtable Webinar earlier today on revusiran, we have included our investigation into the imbalanced mortality that led to the discontinuation of that product's development in the fourth quarter of 2016. As part of this investigation and as we reviewed earlier today, we explored a broad range of potential hypothesis. There was no clinical evidence for revusiran-related cardio toxic effect and there was no evidence for PK or PD-based toxicity from either TTR knockdown or mobilization. Of course, it's impossible to prove a negative, so we cannot fully exclude the contribution of the drug. But as we discussed earlier today, there was some evidence that lower than expected mortality on the placebo arm at the time of discontinuation contributed to the observed mortality imbalance and thus, it is possible the imbalance may have been a chance finding. Importantly, we continue to remain confident that the findings with revusiran, a first-generation GalNAc-conjugate do not read through to the rest of our GalNAc platform and we believe recently reported data from across our pipeline continue to strengthen this confidence. Now, with those introductory comments, I'd like to now turn the call over to Akshay to review our pipeline progress in more detail. Akshay?

[Akshay K. Vaishnaw:]

Thanks, John, and good afternoon, everyone. As John noted, we have indeed made strong progress in our pipeline of investigational RNAi therapeutics. Let's begin with our programs in hATTR amyloidosis, which includes patisiran and ALN-TTRsc02. This is really a big year for patisiran with our Phase 3 APOLLO study readout in the coming weeks. As John commented, we believe we have good reasons to be encouraged by the prospects of patisiran based on our Phase 2 OLE study results and the mechanism validating Phase 3 top line efficacy results for inotersen. At the same time, we're also advancing ALN-TTRsc02 as a subcutaneous alternative. We expect to advance this program into Phase 3 in 2018. Now, the (8

[Manmeet Singh Soni:]

Thanks, Akshay. Good afternoon, everyone, and thanks for joining us today. In addition to the significant progress with our pipeline as Akshay mentioned, we further strengthened our cash position during the second quarter with the recent stock offering. I'll be referring to slide 24 for a discussion of our second quarter 2017 financial results. We entered the second quarter of 2017 with a strong position with approximately $1.25 billion in cash, cash equivalents and marketable investments, including restricted investments. As previously announced, during the second quarter of 2017, we sold 5 million shares of common stock in an underwritten public offering for net proceeds of $355.2 million. In addition, Sanofi Genzyme exercised its right to purchase in a concurrent private placement shares of common stock at the public offering price resulting in additional proceeds of $21.4 million. The net proceeds from the public offering in private placements were received during the quarter. These proceeds will provide support for our general corporate operations, including the continued growth of manufacturing, quality, commercial and medical affairs capabilities to support our transition to a commercial stage biopharma company. The GAAP net loss was $118.4 million or $1.34 loss per share in the second quarter of 2017 as compared to net loss of $90.1 million or $1.05 loss per share for the same period in the previous year. We also disclosed non-GAAP net loss in our earnings release. Non-GAAP net loss is calculated by excluding stock-based compensation expense from GAAP net loss. Please see the appendix included in the slides made available for download with today's call for details regarding GAAP to non-GAAP reconciliation. Non-GAAP net loss for the second quarter of 2017 was $94.4 million or $1.07 loss per share as compared to a net loss of $74.3 million or $0.87 loss per share for the same period in the previous year. Our GAAP revenues were $15.9 million in the second quarter of 2017 as compared to $8.7 million in the second quarter of 2016. Revenue for the second quarter of 2017 included $14.4 million from the company's alliance with Sanofi Genzyme and $1.5 million from the company's alliance with The Medicines Company. The increase in revenues in the quarter ended June 30, 2017 as compared to the prior year period was due primarily to higher cost reimbursement revenue from the company's alliance with Sanofi Genzyme. Moving to expenses, R&D expenses were $90.6 million in the second quarter of 2017 as compared to $83.2 million in the second quarter of 2016. The increase in R&D expenses for the quarter ended June 30, 2017 as compared to the prior year period was due primarily to additional manufacturing expenses related to company's late stage clinical trials. In addition, stock-based compensation expense increased as a result of the company's accounting for the achievement of certain performance-based stock option awards during the second quarter 2017 in connection with the initiation of the ATLAS Phase 3 program for fitusiran. G&A expenses were $45.8 million in the second quarter of 2017 as compared to $18 million in the second quarter of 2016. The increase in G&A expenses for the quarter ended June 30, 2017 as compared to the prior year period was due primarily to an increase in commercial and medical affairs head count to support corporate growth and to prepare for the potential launch of the company's post-commercial product. With respect to guidance for 2017, we remain on track to end the year with greater than $1 billion in cash, cash equivalents and marketable investments including $150 million in restricted investments. We believe this provides Alnylam a very strong balance sheet at the end of 2017 to support development and commercial activities in 2018 and beyond. With that, I will now turn the call over to Barry. Barry?

[Barry E. Greene:]

Thanks, Manmeet. As you heard from John and Akshay, we're looking forward to some critical data points this year, which, if positive, will push us closer to Alnylam 2020 profile, marking our transition from a late-stage R&D company to a multiproduct, commercial organization with a sustainable development pipeline, achieving a profile, as John said, rarely achieved in the biopharmaceutical industry. As part of this transition, we're very focused on expanding our medical affairs, quality, manufacturing and commercial capabilities to support the potential for multiple consecutive product launches in 2018, 2019, 2020 and beyond. These efforts include building our capabilities in the United States, Canada and Western Europe for patisiran and fitusiran and growing globally with givosiran and in future products. As John commented earlier, I encourage you to listen to the webcast of our recent patisiran RNAi Roundtable to understand the comprehensive medical affairs and commercial (28

[Christine Regan Lindenboom:]

Thank you, Barry. Operator, we will now open the call for questions. As a reminder, to those dialed in, we would like you to ask to limit yourself to one question each and then get back in the queue if you'd like to ask additional questions.

[Operator:]

Our first question comes from the line of Ritu Baral with Cowen. Your line is open.

[Ritu Baral:]

Hi, guys. Thanks for taking the question. Folks, as you look towards the registration path and potential approval of patisiran, do you plan on presenting some of the revusiran safety analysis that you presented earlier in the webcast earlier today, just to in an effort to address KOL concerns, any lingering KOL concerns about the side effects, just given the mixed phenotype of some of these patients? And a quick follow-up to that, is can you address some of the commercial strategies that you might have around the patisiran launch to increase diagnosis, I think like skin biopsies was one mentioned in your previous webcast.

[John M. Maraganore, Ph.D.:]

Yes. Great. I mean, Ritu, those are great questions. Akshay should comment as well, but the brief answer on revusiran is that it's a completely different molecule in a different setting and there's really – we obviously share the data with investigators, we share the data with FDA and EMA as well. And so they're generally aware of those data, but they certainly regard those data as being distinct and related to a completely different molecule. Akshay, anything more to...

[Akshay K. Vaishnaw:]

Yes. And to the point that patisiran stands in and of itself and we look at this data obviously very thoroughly. But as an appetizer, I mean if you look at the Phase – OLE data, significant number of patients there with (31

[John M. Maraganore, Ph.D.:]

Okay. And then the commercial question, Barry, you want to (32

[Barry E. Greene:]

Yes. Ritu, as you're aware, there's a significant amount of disease awareness going on in neurology and cardiovascular settings and there are number of programs to try decrease the patient journey and get diagnosis done earlier, including genetic testing, biopsies and other mechanisms to try to help. But the real key is to make sure that people in neurology and cardiovascular setting are aware of the disease. So that it's on the list of potential diagnosis and we're well on our way to doing that.

[Yvonne Greenstreet:]

(32

[John M. Maraganore, Ph.D.:]

I think that's a great point, Yvonne, and I would just finally add that we're taking a very significant leadership position in this field as from a company perspective and I think that the KOLs and the patient communities realize that we're all about helping diagnosis, helping to find patients and then ultimately when new medicines are approved and available, those medicines will become available to them for (33

[Ritu Baral:]

So, am I hearing you correctly, genetic testing is more practical than biopsies for something like this?

[Barry E. Greene:]

I mean, it's – Ritu, (33

[Ritu Baral:]

Great. Thanks for taking all the questions.

[John M. Maraganore, Ph.D.:]

Thanks, Ritu.

[Operator:]

Thank you. Our next question comes from the line of Maury Raycroft with Jefferies. Your line is open.

[Maury Raycroft:]

Hi. Thanks for taking my question. So, the RNAi webinars have been really helpful. So thanks...

[John M. Maraganore, Ph.D.:]

Good.

[Maury Raycroft:]

...for putting this together. So, for the presentation earlier today, I was drawn to slide 44 where you compared the annualized exposure levels of revusiran to the other programs that you have.

[John M. Maraganore, Ph.D.:]

Yes.

[Maury Raycroft:]

I'm just wondering if you can comment on the relative exposure differences between patisiran and givosiran and if this could translate to, A, differences in any way?

[John M. Maraganore, Ph.D.:]

Yes. So, well, I mean, I don't have the numbers in front of me, Maury, but patisiran is given as a 0.3 mg per kg infusion once every three weeks, so 21 milligrams every three weeks. So the total exposure is under 1 gram of (34

[Maury Raycroft:]

I think so. Yes.

[John M. Maraganore, Ph.D.:]

Good.

[Maury Raycroft:]

I'll hop back in the queue. Thank you.

[John M. Maraganore, Ph.D.:]

Great.

[Operator:]

Thank you. Our next question comes from the line of Geoff Meacham with Barclays. Your line is open.

[Geoffrey Meacham:]

Hey, guys, afternoon. Thanks for the question.

[John M. Maraganore, Ph.D.:]

Absolutely. Thank you, Geoff.

[Geoffrey Meacham:]

Yes. Just wanted to ask a couple, John, on your commercial readiness for patisiran. First, I know, not directly comparable but are there any lessons to be learned from the (36

[John M. Maraganore, Ph.D.:]

Yes. Those are great questions. Let me just say one thing for starters, which is that the beginning of lessons learned from (37

[Yvonne Greenstreet:]

(37

[John M. Maraganore, Ph.D.:]

Absolutely. And then commercially, Barry, do you want to comment a little bit on Geoff's questions?

[Barry E. Greene:]

Yes. Sure. It does start, Geoff, with the strength of the target product profile, what kind of patients we're enrolling, the impact we have. And we think that if APOLLO has the kind of data that we're seeing in the open-label extension study, where a significant number of majority of patients have stabilized or improved in their disease, then the value proposition is considerably better than (38

[Geoffrey Meacham:]

It does. Yes. But I just wanted to – does the (39

[Barry E. Greene:]

Yes. We did. So, probably the best way to think about it is there's certain countries that have well-organized centers of diagnosis, places like France, for example, where significant number of patients are captured. There's other countries, Germany, United States that are far less organized and one of the things that we're doing is setting up centers of excellence so that patients can be screened rapidly and diagnosed rapidly. (40

[Geoffrey Meacham:]

Right. Okay. That's helpful. Thanks, guys.

[John M. Maraganore, Ph.D.:]

Thanks, Geoff.

[Operator:]

Thank you. Our next question comes from the line of Vincent Chen with Bernstein. Your line is open.

[Vincent Chen:]

Congratulations on the progress and thank you very much for taking my questions.

[John M. Maraganore, Ph.D.:]

Thank you.

[Vincent Chen:]

On the topic of payer discussions, it sounds like you probably had a range of discussion with payers around reimbursement. Could you provide us with a little bit more color on those? In particular, how are you thinking about what the appropriate pre-authorization and re-authorization criteria might be, and how might have patients' mix of neurological and cardiac symptoms figure into this or their stage of progression?

[John M. Maraganore, Ph.D.:]

So that's a great question. Barry will answer the elements that he thinks he should from a competitive perspective. If you could appreciate that we are in a competitive situation here. So, Barry, why don't you go ahead?

[Barry E. Greene:]

Yes. So to John's point, I mean, there's not a lot that we can say in detail at this point. And again, the results of APOLLO and the impact that patisiran has in the broad range of symptoms will help guide that. But the initial conversation we're having with payers are, first of all, raising awareness that it's a devastating disease. The cost – the burden of the disease is extreme. People can't work or are out of work, can't contribute to society, in certain countries obviously can't pay taxes. So, in the country-level discussions and the U.S. parallel discussions, there's appreciation for the disease and really an agreement that if we're able to halt or even, in some patients, reverse the disease, then again orphan-like pricing is in line with what they expect.

[Vincent Chen:]

I see. Very helpful. And then a quick question on the topic of patient identification which you alluded to earlier. At the last RNAi Roundtable about a week ago, the physician expert discussed sets of criteria. I believe they were termed red flag symptom clusters which a physician might use to assess where to suspect TTR amyloidosis and to decide whether to pursue additional testing. I remember those criteria looking fairly complicated. Is there some sense that you might be able to simplify these into a more easily remembered diagnostic scheme to help drive patient identification efforts?

[John M. Maraganore, Ph.D.:]

Well, let me turn it to Akshay Vaishnaw to comment on that from a medical perspective.

[Akshay K. Vaishnaw:]

Yes. I think from a medical perspective, the core of it is any patient with sensory and motor symptoms in the limbs, along with signs of impotence or gut symptoms or heart symptoms, particularly if there's a family history, it should be considered for either the genetic approach to look for mutation and/or a biopsy. And in centers of excellence, this is already being done. But I think as the drug becomes available and the safety and efficacy data become known and there's a viable treatment option for patients with TTR amyloidosis with a drug like patisiran, I think this tends to be adopted quickly in a more widespread fashion. Yvonne, did you have anything to add?

[Yvonne Greenstreet:]

No. I think, Akshay, that was very well answered.

[John M. Maraganore, Ph.D.:]

Good. Does that answer your question?

[Vincent Chen:]

Yes. Thank you very much and congratulations again.

[John M. Maraganore, Ph.D.:]

Thank you.

[Operator:]

Thank you. Our next question comes from the line of Paul Matteis with Leerink Partners. Your line is open.

[Paul A. Matteis:]

Great. Thanks so much for taking my questions. I had one for Akshay on APOLLO. And, Akshay, it struck me earlier today when you were speaking about the patisiran early data versus the (43

[Akshay K. Vaishnaw:]

Yes. I mean, (44

[John M. Maraganore, Ph.D.:]

I mean, we certainly – this is John, Paul. We certainly have done the analysis of the Phase 2 open-label study and it's shown that regardless of baseline disease severity that we're seeing very striking improvements in neuropathy impairment, certainly compared to the natural history but in some cases, actual negative changes, i.e. improvement. And then I think Akshay's point on inotersen and their comments, we haven't seen a lot of data from them of course. But their comments that the more severe patients also appreciated the benefit and then, finally, the diflunisal study results showed that even in the more advanced patients with diflunisal that there was a treatment benefit albeit it small which you would expect for a stabilizer compared to a lower TTR lowering agent, but it was there. And I think that gives us strong comfort around APOLLO in that dimension.

[Paul A. Matteis:]

Okay. Thank you. That's very helpful. And if I might just ask one quick question on a protocol dynamic for outlets, I'm wondering if you put any specific direction in the protocol for how physicians should be dosing bypassing agents in patients with inhibitors and how the dosing paradigm for bypassing agents and then you (46

[John M. Maraganore, Ph.D.:]

Yes. Great, Paul. Akshay, you want to handle that?

[Akshay K. Vaishnaw:]

Yes. I mean, Paul, in the context of our earlier development with patisiran, I think we learned a lot about how to at least from the context (46

[John M. Maraganore, Ph.D.:]

Yes. I will just add, Paul, that it is reassuring that in our Phase 1 and Phase 2 experience with fitusiran that we have had more breakthrough bleed treatment events with (47

[Akshay K. Vaishnaw:]

And to quantify that (48

[John M. Maraganore, Ph.D.:]

That's right.

[Akshay K. Vaishnaw:]

And basically the reason it's been treated with (48

[John M. Maraganore, Ph.D.:]

Yes. That's correct.

[Akshay K. Vaishnaw:]

(48

[John M. Maraganore, Ph.D.:]

Yes. That is correct.

[Paul A. Matteis:]

Okay. Great. Thank you very much for the detail. Appreciate it.

[Operator:]

Thank you. Our next question comes from the line of Alethia Young with Credit Suisse. Your line is open. Eliana Merle - Credit Suisse Securities (USA) LLC Hi, guys. This is Eliana in for Alethia. Thanks so much for taking the question. So, in terms of APOLLO, what data are you looking for in the cardiac subset population specifically? What do you think would be good or file-able data in the subset in your view? And is there any color you can give us on what the regulators might be looking for in the subset, like, if they have mentioned any specific endpoints they're focused on such as proBNP or (48

[John M. Maraganore, Ph.D.:]

Oh, Akshay, that's your question.

[Akshay K. Vaishnaw:]

Yes. Just on the outset, I would say all data are file-able. I mean, we are obliged, and in fact, to file all data. So, all cardiac data in addition to neuropathy and every other last data will be filed. Now, as to the question of what regulators are looking for and what makes into the label, I think from their perspective, the important things are they look at the nature of the patient population, what's being (49

[John M. Maraganore, Ph.D.:]

Thank you.

[Operator:]

Thank you. Our next question comes from the line of Anupam Rama with JPMorgan. Your line is open.

[Anupam Rama:]

Hey, guys. Thanks so much for taking the question. Maybe I could just clarify the timing of APOLLO here. I think as of recent conferences as well as last week on the Roundtable, you guys were talking about a mid to late September timeframe for APOLLO. This coming weeks mean that it could be like right after the holiday or even earlier, and what are the factors that might shift the timeline up or out in either direction? Thanks so much.

[John M. Maraganore, Ph.D.:]

Thanks, Anupam. No. It is mid to late September which is what we expect. The last few patients are now entering their last dose. And then once we lock the database and analyze the results, we will share the data with you as rapidly as we can. But it's going to be mid to late September. That's the expected timeline.

[Anupam Rama:]

Okay. Thanks so much for clarifying.

[John M. Maraganore, Ph.D.:]

Yes. Thanks, Anupam.

[Operator:]

Thank you. Our next question comes from the line of Ted Tenthoff with Piper Jaffray. Your line is open.

[Edward Tenthoff:]

Great. Thank you very much for the update, and I apologize if this question has been asked. I've been having telephonic issues, but I wanted to sort of at a higher level what preparation you're starting to make for success around APOLLO on the commercial side? I know that you were talking about some of the payer discussions and things like that. But in terms of specialty pharma, sales force, medical liaisons, maybe you can update us on where you are in that process. Thank you.

[John M. Maraganore, Ph.D.:]

Yes. Absolutely, Ted. And we touched on some of this, but you're asking a slightly different question here. So it's a useful question for us to answer. Barry, do you want to take it?

[Barry E. Greene:]

Yes, I'll touch – what we said on the call, Ted, is we encourage everybody to listen to the patisiran Roundtable. We went through this in a lot more detail. But I'll touch upon it quickly. We're built out now in the United States, in the major markets in Europe with leadership. We've got medical mostly in place. We have our supply chain in place. And we're really positioned, post-APOLLO, really to file the NDA and MAA, and then we'll be positioned next year to launch the drug with a built-out commercial force, medical force, and supply chain in place, including the payer and reimbursement. So, we're in pretty good shape after APOLLO.

[John M. Maraganore, Ph.D.:]

And a lot of the – I'll just add to that, Ted. We built out our European organization. We have our headquarters. We've got country managers in the key markets. We have access leadership in those markets locally. We've obviously done the same in the U.S. as well. And we're out there in force and getting ready to be there with this very, very important product.

[Edward Tenthoff:]

And how much does the pacing group help here in terms of sort of rallying and educating patients? I have to imagine this is a – it's a pretty motivated pacing group.

[John M. Maraganore, Ph.D.:]

Yes. The answer is a lot. Barry, do you want to comment any further on that?

[Barry E. Greene:]

Yes. I think the answer is a lot, and it depends on country. There are certain countries like the United States and France where there's sophisticated patient groups and other countries where the patient groups are just starting to form. And keep in mind, Ted, that like many orphan diseases, the course of the disease, the patient population is just getting known and just getting educated. So many countries are frankly forming strong and stronger patient advocacy. The voice of the patients certainly will matter here in a big way.

[Edward Tenthoff:]

Excellent. Thank you so much.

[John M. Maraganore, Ph.D.:]

Thank you, Ted.

[Operator:]

Thank you. Our next question comes from the line of Christopher James with Ladenburg. Your line is open.

[Christopher S. James:]

Hey, guys. Thanks for taking the questions. I guess assuming positive data from patisiran, maybe kind of piggyback on (54

[John M. Maraganore, Ph.D.:]

All right. So, thanks, CJ. I mean, it's a great question. Obviously, we're – it's too soon to give you the answer you'd like to have on that. And it's also something which is in a competitive landscape here. So we're going to be a little bit careful. But I think Barry's point is the right one. If patisiran demonstrates the type of value that we have seen in the Phase 2 open-label study, then this is a medicine that is going to provide enormous benefit for patients, first and foremost. It's in orphan setting, and it's going to be price consistent with orphan medicines that have high impact in an orphan setting. And I think that's where we should leave it at this point in time. Obviously, it's very, very data-driven, no doubt about it, and it's going to be something we spend a lot of time on in the back half of this year, early next year to get it right. But I think very importantly, we're having conversations with payers or having conversations both government and private payers. We're learning quite a bit about the landscape, and we certainly have put together an overall value dossier that we think supports the value proposition for the medicine if it hits its key efficacy and safety criteria. So we're optimistic that we can support an appropriately valued product.

[Christopher S. James:]

Got it. Thank you. And then maybe lastly, can you maybe walk us through, assuming positive data, steps through speaking to the FDA filing and do you expect an FDA panel? Thanks.

[John M. Maraganore, Ph.D.:]

Yes. Akshay, do you want to comment on that?

[Akshay K. Vaishnaw:]

Yes. I think it would be conservative to assume that for a first-in-class agent in a disease like this, there would be an advisory committee, yes.

[Christopher S. James:]

Got it.

[John M. Maraganore, Ph.D.:]

And it's possible, CJ that because there are two agents in the field, that both agents might be discussed at the same advisory panel.

[Christopher S. James:]

Got it. Got it. Thank you, guys.

[John M. Maraganore, Ph.D.:]

All right. Thank you. Thank you.

[Christopher S. James:]

Bye-bye.

[Operator:]

Thank you. Our next question comes from the line of Mike King with JMP Securities. Your line is open.

[Mike G. King:]

Hey, guys. Thanks for taking the questions and fitting me in. Just two real brief ones. Just thinking with the theme of market preparation and patient out and physician outreach. I notice you guys opened a couple of websites recently to facilitate communication with both audiences. I'm just wondering if you would be willing to give us any kind of qualitative information about how many hits or how popular those websites have become? Have you gotten any feedback or new contacts from the sites at all?

[John M. Maraganore, Ph.D.:]

It's a great question. These have been recently put together, so I'm not sure we have a lot of real data. Anything to add, Barry?

[Barry E. Greene:]

I guess what I can say Mike is what you have noticed is that unbranded disease awareness aim towards healthcare providers was launched months ago, and very recently, you see this on social media and others, patient indication, again, unbranded has been launched. And I think it's fair to say it's very well received. And as John said earlier, we are absolutely seen as the leaders in trying to educate the market to look for this disease in patients that they might not have figured it out. So, it seems very well received and time will tell, but we think that will lead to short invitation journey in finding patients more rapidly.

[Mike G. King:]

Okay, great. Can I just ask you a quick question on sc02?

[Barry E. Greene:]

Yes.

[John M. Maraganore, Ph.D.:]

Yes.

[Mike G. King:]

I asked basically similar question last quarter. I just wondered if sort of the revusiran analysis and your progress with patisiran has changed your view of what your course – future course for sc02 might be? Is it still your thought that you may get approved on TTR reduction or do you think you may have to do – have a clinical endpoint for approval?

[John M. Maraganore, Ph.D.:]

Yes. It's a topic of active discussion internally, Mike. I think that – two things, really, one is we're very, very impressed with the data of our TTRsc02. I mean, it's stunning if you look at the updated data that we presented for the first time last week to look at that level of knockdown with the very, very small doses that are administered. I mean it is clearly supportive of quarterly, if not semiannual, dose regimens and it's very, very encouraging. Safety looks very good. And so we're very impressed with that. And we're very committed to developing it as a best-in-class med across the whole spectrum of ATTR amyloidosis ranging from patients that have polyneuropathy, patients that have cardiomyopathy, and even the wild-type setting over time. And the initial studies will be focused on really introducing this agent relatively quickly – as quickly as we can to the market, so that patients can have an option of patisiran or a subcu option that can be given as in frequently a once a quarter or twice a year. And so that is going to be a focus of ours exactly what endpoints we use in the studies and how we do the studies is still an active topic of internal discussion that will soon be a topic of discussion with the regulators as well. But we're very confident that there is a very attractive path forward for the drug and we remain very bullish around the prospects forward based on the data that we've got right now.

[Mike G. King:]

Great. Thanks so much for taking the questions.

[John M. Maraganore, Ph.D.:]

Good. Thanks, Mike.

[Operator:]

Thank you. And I'd now like to turn the call back to the company for closing comments.

[John M. Maraganore, Ph.D.:]

All right. Well, look, thanks everyone for joining us this afternoon. For those of you that joined us earlier and today, thanks for your commitment to Alnylam during the course of the afternoon. As I said earlier, 2017 is really a pivotal year for our company transitioning toward a commercial stage. We could not be more excited about all that and we look forward to speaking to you in the coming weeks to come. Thanks very much. Bye-bye.

[Operator:]

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone, have a wonderful day.