Alnylam Pharmaceuticals Inc (ALNY) 2012 Q3 法說會逐字稿

Good afternoon, ladies and gentlemen, and welcome to the Third Quarter 2012 Alnylam Pharmaceuticals Earnings Conference Call. My name is Chris, and I will be your conference moderator for today. (Operator Instructions) As a reminder, this conference is being recorded for replay purposes, and at this time I would now like to turn the conference over to your presenter for today, Ms. Cynthia Clayton. Ma'am, you may proceed.

Good afternoon. I'm Cynthia Clayton, Vice President of Investor Relations and Corporate Communications at Alnylam. With me today are John Maraganore, Chief Executive Officer; Barry Greene, President and Chief Operating Officer; Akshay Vaishnaw, Executive Vice President and Chief Medical Officer; and Mike Mason, Vice President of Finance and Treasurer. 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 at www.alnylam.com.

During today's call and as outlined in slide 2, John will provide some introductory remarks and provide some general context. Akshay will summarize the clinical progress with our Alnylam 5 x 15 and partner program; Mike will review our financials and guidance; and Barry will provide a brief summary of our business highlights and goals before we open up the call for your questions.

Before we begin and as you can see on slide 3, 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 provision 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 recent 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 views as of any subsequent date. We specifically disclaim any obligations to update such statements. I will now turn the call over to John.

Thanks, Cynthia. Welcome, and thanks, everyone, for joining us this afternoon. Our activities and accomplishments this quarter and recent period reflect very important scientific and clinical progress for Alnylam and, in fact, for the entire field of RNAi therapeutics. Highlights include the positive results we reported in our clinical programs and continued execution on our Alnylam 5 x 15 product strategy.

Importantly, we reported positive data from a Phase 1 study of ALN-TTR02, the lead program within our Alnylam 5 x 15 efforts, where we show that a single dose of drug results in rapid, dose-dependent, durable, specific and RNAi-mediated knockdown of serum transthyretin, or TTR, the disease-causing protein in transthyretin-mediated amyloidosis.

Specifically, we showed TTR knockdown of up to 94% that was sustained to nearly 80% at one month. We believe these data document an unprecedented level of clinical activity for RNAi therapeutics and serve as an important industry milestone in the advancement of this new class of medicines to patients. In the meanwhile, we continue to enroll patients in our Phase 2 multi-dose study of ALN-TTR02 in ATTR patients.

With the recent accomplishments in our pipeline efforts, we continue to be convinced that with Alnylam 5 x 15, we have a very exciting opportunity to advance RNAi therapeutics focused on genetically defined targets for diseases that currently have limited treatment options for patients and their caregivers. We also believe that this product strategy creates an important path and a compelling path for overall value creation for our shareholders.

The other highlight for the quarter and recent period is our success in business development. Indeed, we completed three significant deals during the quarter and recent period including an alliance for ALN-VSP in China with Ascletis; an alliance with Monsanto for applications of Alnylam RNAi technology in agriculture; and an alliance in our ALN-TTR program in Japan, and other Asia Pacific countries with Genzyme. Based in large part on our recent pipeline successes, we believe that this business development efforts will continue going forward, and we're having many excellent discussions.

We also had major success in our noncore areas, such as micro-RNA therapeutics and our efforts with Regulus, where they completed deals with AstraZeneca and Biogen Idec, and then completed their initial public offering. In fact, when you combine our efforts at Alnylam with those of Regulus, we were able to bring in just over $135 million in new capital to fund our collective RNA therapeutic efforts.

In summary, it has really been an excellent period for Alnylam. Our science is working, and we believe that we have unambiguously demonstrated that we can achieve robust RNAi effects in man. Moreover, we are advancing an exciting pipeline of innovative medicines that have potential breakthrough potential in high unmet need clinical settings. This includes four programs in clinical development today, and when we look out over the next 12 months or so, we expect that this will grow to six programs into clinic, and at least one program in Phase 3 development.

Finally, we are supporting our business with new alliances, with outstanding partners that we believe can make a big difference in advancing our technology for medicines to customers or patients while we retain key product rights to build maximal value. With that, I'll now turn the call over to Akshay for more detail review of our clinical activities, our pipeline, and our scientific progress. Akshay?

Thanks, John, and hello, everyone. As John mentioned, we've had an extremely rewarding and productive quarter on the clinical front. I am going to focus on the recent progress with our Alnylam 5 x 15 programs beginning with the recap of data we presented from our ALN-TTR02 program.

As all of you are aware, ALN-TTR02 is the flagship program in our 5 x 15 product strategy. It is aimed at the treatment of TTR-mediated amyloidosis, or ATTR. ATTR is a devastating hereditary and often fatal disease caused by mutations in the TTR gene. As an orphan disease, ATTR afflicts approximately 50,000 people worldwide and is associated with significant morbidity and a mean life expectancy of just 5 to 15 years from symptom onset.

It is clear that new therapies are needed for the treatment of ATTR, and we believe that our mechanism of action silencing the disease-causing TTR gene leading to knockdown of the circulating TTR protein, has the potential to generate a profound therapeutic impact.

In July, we reported data from our Phase 1 trial with ALN-TTR02. The trial was conducted as a randomized, single blind, placebo-controlled, single ascending dose study with 17 healthy volunteer subjects. The primary objective of the study was to evaluate the safety and tolerability of a single dose of ALN-TTR02. In addition, pharmacodynamic and clinical activity for ALN-TTR02 were measured based on circulating serum TTR protein levels through at least day 56 following the single dose.

As you can see on slide 8, results from the study showed that a single dose of ALN-TTR02 resulted in rapid, dose-dependent, durable and specific knockdown or serum TTR levels which appeared to be RNAi-mediated, with the overall results being highly significant with a p-value of less than 0.0001.

Even at doses as low as 0.15 milligram per kilogram, substantial serum TTR suppression was achieved where a mean 82% reduction was observed at nadir. This was extended at the next dose level of 0.3, where we showed an 87% mean TTR knockdown, and then at 0.5, where we showed 94% level of TTR knockdown.

ALN-TTR02 exhibited a rapid onset of action and a durable response. Over 50% knockdown in TTR was achieved by day 3, and all of the 0.15, 0.3 and 0.5 milligram per kilogram subjects, and nadir levels were reached between days 10 and 14.

In terms of durability, ALN-TTR02 sustained a 67% TTR knockdown at day 28 at the 0.3 milligram per kilogram dose. And a nearly 80% TTR knockdown at 28 days at 0.5 milligram per kilogram. All this after just one single dose of the drug. We believe that this pharmacodynamic profile supports at least once-a-month and possibly even once-every-other-month dosing going forward, a very compelling profile for a potential breakthrough therapy in this clinical setting.

ALN-TTR02 was found to be generally safe and well tolerated in this study, consistent with our broader clinical experience of RNAi therapeutics using LMP formulations where we have now treated over 100 patients or subjects with over 325 doses of drug and for durations of over two years.

These results provide key human proof of concept and associated clinical relevance as we advance this medicine to patients for the treatment of ATTR, a devastating orphan genetic disorder.

We are also continuing to enroll patients in a Phase 2 study with ALN-TTR02 in ATTR patients. The study will look at the safety and tolerability of multiple doses of ALN-TTR02, as well as serum TTR knockdown that occurs with two doses of drug. We expect this study to be complete and to share the results in mid-2013.

In general, we look forward to continuing to share clinical data from our ALN-TTR02 program and, assuming positive results in the Phase 2 study, our goal is to advance to a pivotal trial by the end of 2013.

As you can see on slide 10, we also made strong progress in our ALN-TTRsc program, which uses our GalNAc conjugate delivery platform enabling subcutaneous delivery. Specifically, at recent scientific meetings we have shown potent, dose-dependent and durable knockdown of serum TTR with ALN-TTRsc in preclinical models.

In nonhuman primate studies, ALN-TTRsc showed an approximately 80% reduction of TTR at doses as low as 2.5 milligram per kilogram in a weekly subcutaneous dosing regimen. In addition, in single dose and multi-dose preclinical safety studies in rodents and nonhuman primates, ALN-TTRsc was found to be generally safe and well tolerated with a no adverse effect level, or NOAEL, greater than or equal to 300 milligram per kilogram, and a therapeutic index exceeding 100-fold, including the absence of any injection site reactions or elevations in proinflammatory markers.

Our second area of major development focus on Alnylam has been our hemophilia program. We are advancing ALN-AT3 in RNAi therapeutic targeting antithrombin, or AT. Data were presented at multiple scientific meetings over the quarter and recent period from this program.

Our findings as shown on slide 12, show that subcutaneous administration of ALN-AT3, which also employs GalNAc-siRNA conjugate delivery approach, results in potent, dose-dependent and durable findings in AT in preclinical models, and that AT reduction can normalize thrombin generation in an animal model of hemophilia, establishing key proof of concept for this program.

Specifically, ALN-AT3 demonstrated potent activity in both mice and nonhuman primates with an ED50 for AT plasma protein knockdown of approximately 1 milligram per kilogram after a single subcutaneous dose.

Based on nonhuman primate studies, nadir knockdown levels of AT were achieved by about day 15, with effects lasting over 22 days. In multi-dose rodent studies, once weekly subcutaneous administration of ALN-AT3 resulted in an ED50 and ED80 for AT plasma protein knockdown of 0.25 milligram per kilogram, and 0.75 milligram per kilogram, respectively.

But more in studies performed in mouse models of hemophilia, ALN-AT3 was found to achieve dose-dependent knockdown of endogenous AT and to significantly increase, in fact normalize thrombin generation.

We are very excited about the potential for ALN-AT3 in hemophilia and other bleeding disorders, and believe that these new data support a once-a-week or twice-a-month subcutaneous dosing paradigm for this RNAi therapeutic, which we believe could become a game-changer for hemophilia patients.

We also continue to advance additional Alnylam 5 x 15 programs, including ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia, ALN-HPN and RNAi therapeutic targeting the hepcidin pathway for the treatment of refractory anemia, and ALN-TMP, an RNAi therapeutic targeting TMPRSS6, or TMPRSS6, for the treatment of hemoglobinopathies.

At the OTS meeting just last week, we presented new data demonstrating generation of a GalNAc-siRNA conjugate in our ALN-PCS program. Specifically, as you can see on slide 13, the new ALN-PCSsc drug candidate demonstrated potent knockdown of the PCSK9 target gene with an ED50 below 0.3 milligram per kilogram of a single subcutaneous dose. We expect that this new subcutaneous program will be an important part of a partnership that we aim to form to advance our ALN-TTR-PCS program into Phase 2.

Finally, we are also pleased to report complete data from our Phase 2b trial with ALN-RSVO1 in lung transplant patients in September. With these results in hand, we are meeting with US and European regulatory authorities to determine next steps for this program. Pending the outcome of those discussions, we will determine the appropriate path forward for this program and communicate them to you at the end of this year.

We made tremendous progress with our Alnylam 5 x 15 and partnered clinical programs, and we look forward to continuing to share updates of these programs with you in the coming months. With that, I'd like to now turn the call over to Mike, for review of our financials. Mike?

Thanks, Akshay, and good afternoon, everyone. Turning to slide 15, we continue to maintain a strong financial profile ending the third quarter of 2012 with $295.8 million in cash, cash equivalents and marketable securities, which excludes the upfront payment of $22.5 million from our recent alliance with Genzyme, which was received in the fourth quarter.

Our GAAP revenues for the third quarter of 2012 were $16.8 million as compared to $20.8 million in the third quarter of 2011. Q3 revenues consist primarily of $9.3 million of collaboration revenues related to our alliance with Roche, which has signed its rights and obligations to Arrowhead Research Corporation during 2011, and $5.5 million of revenues related to our alliance with Takeda.

Looking ahead, we expect revenues to decrease due to the completion of amortization of Roche, Arrowhead deferred revenue during the third quarter of 2012, partially offset by revenues from the Monsanto alliance, which will be amortized on a straight line basis over five years.

Moving to expenses, R&D expenses were $22.1 million in the third quarter of 2012, as compared to $24.3 million in the prior year period. The decrease is due primarily to lower clinical trial and manufacturing expenses related to our ALN-RSV and ALN-VSP programs partially offset by additional expenses related to the advancement of our ALN-TTR program.

G&A expenses were $12.8 million in the third quarter of 2012, as compared to $9 million in the third quarter of 2011. The increase was due primarily to an increase in consulting and professional service fees related to business and legal activities.

Regarding year-end guidance, we now expect to end 2012 with greater than $280 million in cash, which will continue to provide us with a strong balance sheet as we advance our RNAi therapeutics through clinical trials and towards the market.

Our previous guidance was to end the year with greater than $250 million. This increase is due to upfront payments from new alliances with Monsanto and Genzyme. As we outlined in our press release, the new guidance excludes any potential payments related to a resolution, if any, of our ongoing litigation.

This concludes the financial highlights and I'll now turn the call over to Barry. Barry?

Thanks, Mike. As you heard this afternoon, we are demonstrating with human clinical data that the RNAi pathway can be harnessed to create high impact, innovative medicines. In addition to the substantial advancements with our pipeline, we also made tremendous progress in our business development efforts in the recent period through new collaborations with Genzyme, Monsanto and Ascletis.

Notably, we recently formed a strategic alliance with Genzyme to advance our TTR amyloidosis program in Japan and the broader Asia Pacific region. Genzyme, clearly the industry innovator and leader in bringing orphan drugs to patients in need, will leverage its proven regulatory and commercial capabilities in the Japanese and broader Asian market to advance Alnylam TTR programs, which includes ALN-TTR02 and ALN-TTRsc. Very importantly, Alnylam maintains all other rights consistent with our plans to develop and directly commercialize this potential breakthrough medicine in North and South America, Europe and rest of world.

Under the terms of the agreement, Genzyme has made an upfront cash payment of $22.5 million to Alnylam. In addition, Alnylam is eligible to receive certain success-based milestone payments totaling $50 million. Furthermore, Genzyme will make tiered royalty payments to Alnylam that are expected to yield an effective rate in the mid-teens to mid-twenties on sales of ALN-TTR in the territories covered by Genzyme. The royalties in this agreement represent a very attractive way for Alnylam to participate in the success of ALN-TTR in Japan and other Asian countries.

We are very excited to form this alliance with Genzyme, and we believe that as a result of this new alliance, ALN-TTR will get to patients in Japan and other Asia Pacific countries sooner, and that our drug will reach these markets much faster.

We are also excited about the partnership we formed with Monsanto, a leader in the field of agriculture. This collaboration facilitates broad use of our intellectual property and technology in agricultural applications and enables additional value creation for Alnylam, helps center our focus on human therapeutics. The alliance brings Alnylam's broad RNAi-based IP and proprietary technologies to Monsanto's new BioDirect, which aims to deliver innovative biological solutions for farmers.

The deal includes $29.2 million in upfront payments from Monsanto to Alnylam. Alnylam is also eligible to receive milestone payments and additional funding for collaborative research efforts. In addition, Alnylam is eligible to receive royalty payments on products utilizing Alnylam intellectual property.

As noted earlier, we also formed a strategic collaboration with Ascletis to develop ALN-VSP for the treatment of liver cancer in China. This collaboration provides Ascletis, a privately-held US/China joint venture pharmaceutical company, with the exclusive rights to develop and commercialize ALN-VSP in China, including Hong Kong, Makow and Taiwan.

With this collaboration, we're eligible to develop ALN-VSP globally for the partner's advancement in a region where hepatocellular carcinoma is a major health concern. And as we retain all rights in the rest of the world, this partnering strategy provides future opportunities for Alnylam to advance this novel therapeutic in other markets.

In the recent period, there was also a good deal of progress made in our efforts outside of our primary focus of RNA therapeutics. This is particularly true for Regulus Therapeutics, the company we formed with Isis to focus on development and commercialization of microRNA therapeutics. They successfully completed an IPO and are now trading on NASDAQ under the ticker RGLS.

Through the IPO, Regulus raised $50.9 million in gross proceeds. As a result of the IPO, Alnylam's ownership position in Regulus now stands at 17%. Regulus also had a busy period leading up to the IPO, including performing strategic collaborations with AstraZeneca and Biogen, who joined other partners, GSK and Sanofi.

We're excited that Regulus is now public. We believe they are positioned to build significant value in the years to come, and view our equity position as an opportunity to bring significant value to Alnylam shareholders.

As John mentioned earlier, we think it's terrific that our overall efforts, including business development and financing efforts at Alnylam Regulus during this last period have resulted in over $135 million in new capital investment for RNAi therapeutics and technology. We are very pleased with that accomplishment and look forward to continued success in the future.

Now, in closing, as Mike mentioned, we continue to maintain a very solid balance sheet and remain poised to execute our goals outlined here on slide 20, which include advancing ALN-TTR02 through the current Phase 2 trial with data in mid-2013, with a goal of starting our pivotal studies by end of 2013; our plan to file an IND for ALN-TTRsc by the end of 2012 with data in 2013; also filing an IND for ALN-AT3, a subcutaneously-administered RNAi therapeutic for the treatment of hemophilia to be filed in 2013; partnering our ALN-PCS program prior to the start of Phase 2; and advancing other Alnylam 5 x 15 programs toward Phase 1 with partnerships we aim to form.

We also believe we will form additional partnerships on other programs in technology, and as Mike highlighted, ending the year with over $280 million in cash.

In summary, I think it's clear that we are executing on our goal of driving important innovative RNAi therapeutics for patients in need. With that, I'd like to turn the call back over to the operator for your questions. Chris, can we have questions, please?

Thank you. (Operator Instructions) Our first question comes from the line of Geoff Meacham with JPMorgan. You may proceed.

Hi. This is actually Mike in for Geoff. Thanks for taking the question. I had a question on the hemophilia program. Sort of now that we've seen some of the Phase 3 data for Biogen's hemophilia program, I'm just curious if that in any way sort of changes how you view the market opportunities for the AT3 program?

Absolutely. Well, let me have Akshay and Barry address that question. Akshay, do you want to start?

Yes. You know, there are a number of very exciting things about the Biogen program, including potentially less frequent administration of intravenous replacement factors for Factor VIII or Factor IX.

I think more importantly for us and what's really exciting about the Alnylam approach is a completely different approach. It relies on subcutaneous dosing. There are really no programs in the hemophilia space that can afford subcutaneous dosing. It's an innovative approach targeting AT, or antithrombin, which will allow us to address several very important segments. So, there are these very high unmet need patients with inhibitors, who have no ability to take any replacement factor on a regular basis. So, I think there is tremendous possibility there.

And then with respect to other hemophilia A and B patients, administration of our ALN-AT3 drug could lead to less frequent dosing of the replacement factor, still potentially -- or even at one -- one possibility is that they might even be able to do without replacement factors if the product has an outstanding profile, which we hope that it does. So, there are lots of opportunities there in hemophilia itself.

And then not to mention rarer bleeding disorders, so these are deficiencies of some of the other clotting factors, like V or VII or X, XI, and we believe we can have an important role to play in those bleeding disorders as well. So, we see tremendous possibility for our subcutaneous approach across a range of targets.

And, Barry, anything to add on the commercial side?

Yes, I think Akshay outlined the opportunity very well. The other value that Biogen's efforts create is to help the prescribing physicians and patients understand that moving to a more convenient product will provide benefit. So, when we come out with a game-changing subcutaneous administration that resets the coagulation cascade and works in a fundamentally different way with data to support that, the market will be tuned to try different products. And as Akshay said, we have an opportunity to treat a broad range of patients with a very different approach. Once a week or once every other week subcutaneous administration, it should be highly amenable to both treating physicians and patients to try.

Mike, does that answer your questions?

Yes, that's great. Thank you.

Our next question comes from the line of Marko Kazul of Leerink Swann. You may proceed.

Hi. Good afternoon. I wanted to maybe start with a question on your ASH abstract, if you could review the AT3 abstract and what else might be presented at the meeting?

Thanks, Marko. Obviously, the ASH abstracts just went out, I guess, this morning, and we're going to be presenting two data updates, one from our hemophilia program and from our hemoglobinopathy program.

In the case of our hemophilia program, we're going to be showing some pretty exciting data using ALN-AT3 in nonhuman primates, where we demonstrated very nice results looking at knockdown of antithrombin, but also increase in thrombin generation. And as you know, Marko, thrombin generation is what's deficient in hemophilia patients, and now we're shown in nonhuman primates. These are obviously wild-type animals, not hemophilia animals that were able to achieve a significant increase in thrombin generation following administration of drug subcutaneously in those animal studies.

The second key study is going to be related to our TMPRSS6, T-M-P-R-S-S-6 program, which really has become an exciting approach for the treatment of hemoglobinopathy. And I'll leave the abstract for you and others for read, but it really demonstrates that knocking down TMPRSS6 can have in fact disease-modifying effects in a model of beta thalassemia, as well as in a model of hemochromatosis. And so we think these data are very exciting and clearly demonstrate, again, disease-modifying effect of this treatment. Akshay, do you want to add anything on both of those abstracts?

No. I mean, I think the hemophilia one you have outlined very nicely. Just to add to the TMPRSS6, you know, Marko, realizing that hemoglobinopathy such as beta thalassemia, sickle cell disease. But, really, these patients have no access to any disease-modifying approach at all. Essentially, the idea is to give them transfusions if they're running low in the hematocrit or hemoglobin count, treat the complications, and offload the iron, because at some point they get overloaded with iron. And what TMPRSS6 does, it abrogates the uptake of iron from the gut effectively by dramatically raising (inaudible) and hepcidin levels.

And what we found is that not only does that ameliorate the iron overload problem in a very nice model of beta thalassemia in the mouse, which is rather reminiscent of the human problem, but importantly it begins to correct the bone marrow defect and normalizes the alpha to beta globin ratios, which then has a knock-on effect in terms of improving the splenomegaly and the extramedullary hematopoiesis that is going on. And that's what brings about the real -- the modifying approach.

So, a lot of data in the abstract. We'll let you guys read that, but we're fundamentally excited about data that point to a disease-modifying approach and a dataset that has been well received by folks that treat this set of diseases.

And, of course, that's what we're limiting our comments now, Marko, on what's in the abstracts so that we don't [create] any embargos, and we'll obviously be presenting full data at the meeting, which might include additional data that we're not discussing right now.

If you don't mind my squeezing one on TTR. I was wondering if you could give us your updated thoughts on a possible cohort expansion strategy or what might be driving the mid-2013 data? Thanks.

Akshay?

Yes. Marko, just to reiterate, you've seen the Phase 1 data, we've shared them with everybody. They are obviously very interesting, exciting. They're driving the approval right now in the Phase 2 study. It's going well, and we've said about mid 2013. It looks to be good time frame to us to get the study done and share the data. We are fully anticipating positive data at the present time. Things are going well and onto Phase 3 by the end of 2013.

(Operator Instructions) Our next question comes from the line of Alan Carr, Needham & Company. You may proceed.

Hi. Thanks for taking my questions. So, RSV, you're going to be meeting with, I guess FDA and EMA by year end. I'm wondering what your options are there for next steps in terms of what sort of trial you run next? And then also regarding Monsanto, can you give us the sense of how development might progress here, you know, timelines to when something might be commercialized in a broad sense? Thanks.

Well, let me answer the Monsanto question first and give some context on the RSV question, and then Akshay should finish the RSV question. But on the Monsanto question, we're not really at liberty, Alan, to comment anymore than what is currently in the release. What I can say is that Monsanto is extremely committed to the technology and to the application of RNAi in their BioDirect program. They have, I think, a very clear vision around advancing biological strategies in ways that improve crops and obviously improve the ability of farmers to succeed in agriculture. And so it is something which we're very enthusiastic about their level of commitment to it, and we're very enthusiastic about working with them. There is probably no better company in the world to work with in the agriculture setting than Monsanto, but we can't give any further details on that.

On RSV, I want to be really clear that there are, I think, a range of different options that can occur here. One option is that after discussing the results with regulators both in the US and Europe, we and our partners at Cubist and Kyowa Kirin decide that it's not necessarily worth proceeding, and so that's one side of it. And that may be an outcome that we decide with them.

The other outcome is that we clearly define and delineate a path forward for approval largely focused on a Phase 3 trial that would support approval. And that we all feel good about that path forward and that Cubist and Kyowa Kirin make the commitment to proceed with that.

So, those are the bookends. I'll throw it to Akshay to comment, if he has anything to add further than that. But those are basically the bookends. And the discussions are very much scheduled and we'll have more information for you by year's end.

That's exactly right, John, and we look forward to doing that.

Yes, good.

All right. Thanks very much.

Our next question comes from the line of Meghan Dow with MOV. You may proceed.

Hi, everyone. Thanks for taking the question. We just had a couple questions. You've had such great progress from preclinical data and your Phase 1 data moving forward. We were curious if this was -- if you can expand anymore on some of the points here in your press release of an agreement with a major pharmaceutical company, if you can expand on that at all? And I was also curious how you see the relationship with Alnylam and Regulus as the two companies moving forward as separate entities but with the lines [desired]?

Sure. Those are great questions, Meghan. Well, so let's start with your first one as relates to the news we announced today in our quarterly release related to our Alnylam biotherapeutics effort. This is a new partnership that we formed with a global top five major pharma company that has an interest in biotherapeutics, and we are working with them to use our technologies to improve biologics manufacturing that they're engaged in. And that's all we're really saying about that technology or that partnership at this time. Obviously, we hope it succeeds in their efforts and obviously that will be attractive to Alnylam in the future if it does so. So, that's what we can say there.

And then your other question, Regulus is a great one. We're obviously very pleased that Regulus was successful in their IPO, and we remain committed to Regulus and their success. We have board representation on Regulus, but Regulus is an independent company and obviously we own 17% of it and are committed to it for the long term. But we do believe that it represents value to Alnylam shareholders because as Regulus builds value, our ownership, our equity ownership in Regulus will grow in value, and that ultimately delivers value back to our shareholders. So, we're pleased with our IPO. It clearly was an important step forward. And we're also pleased with the deals they did with AstraZeneca and Biogen Idec over the same recent period. So, very busy period with Regulus and clearly a very successful year.

Certainly. Excellent. And just one quick follow-up question. Can you expand at all on any of the discussions you've been having for the PCSK9 program?

Well, what I can say in very general terms is there remains very significant interest in the target. We do have discussions that are ongoing and when they're ready to be talked about in final form, we'll be talking about them in final form. We're encouraged by the progress that our program has made, not only the clinical data, the very strong clinical data that has been generated, which again we feel is very much in line with what's been achieved in single dose, nonstatin-containing studies with the PCSK9 antibodies, as highlighted by some of the results that came out today at the American Heart meeting, where we're seeing a 50% decrease in LDLC, which is comparable to what is being seen with the antibodies. But, of course, our approach is differentiated in that we're blocking the synthesis of PCSK9, and we think that's going to be important as it relates to optimal efficacy.

But the other data point I think that's quite important is the generation of a GalNAc conjugate lead for that program, which enables subcutaneous dosing, and that's going to be very important in that setting. And that will be a program that will be wrapped into the overall program with the partnership that we will form. So, we think that's very encouraging and we'll look forward to updating you when we can talk about a specific partnership that has been accomplished.

Excellent. Congratulations, guys.

And we have no further questions at this time. I would now like to turn the call back over to Alnylam for any closing remarks.

Great. Well, thanks, everyone. Look, Alnylam continues to lead the development and advancement of RNAi therapeutics to patients. We are very proud of our recent clinical data and business accomplishments, and we look forward to sharing more results on both dimensions throughout the remainder of the year. Thank you very much. Bye-bye.

Ladies and gentlemen, that concludes today's conference. Thank you so much for your participation. You may now disconnect. Have a great day.