Regulus Therapeutics' (RGLS) CEO Kleanthis Xanthopoulos on Q2 2014 Results - Earnings Call Transcript

Aug. 6.14 | About: Regulus Therapeutics (RGLS)

Call Start: 17:00

Call End: 17:40

Regulus Therapeutics (NASDAQ:RGLS)

Q2 2014 Earnings Conference Call

August 6, 2014 05:00 p.m. ET

Executives

Amy Conrad – Senior Director, Investor Relations and Corporate Communications

Kleanthis G. Xanthopoulos – President, Chief Executive Officer

Paul C. Grint – Chief Medical Officer

Neil W. Gibson – Chief Scientific Officer

Dan Chevallard – Vice President, Finance and Accounting

Analysts

Eric Schmidt – Cowen & Company

Bill Tanner – FBR Capital Markets

Christopher James – Brinson Patrick Securities

Liana Moussatos – Wedbush Securities

Operator

Good afternoon, ladies and gentlemen. And welcome to the Regulus Therapeutics Second Quarter 2014 Conference Call. My name is Latoya, and I will be your coordinator for today.

I would now like to turn the conference over to Amy Conrad, Senior Director, Investor Relations and Corporate Communications. Please proceed.

Amy Conrad

Good afternoon and thank you for joining us. On behalf of Regulus Therapeutics, I would like to welcome everyone to our conference call for the quarter ended June 30, 2014. I hope you've all had a chance to review today's press release. If you have not and you need a copy, you can visit our website at www.regulusrx.com.

Joining me on today's call are Kleanthis Xanthopoulos, Ph.D., President and Chief Executive Officer; Paul C. Grint M.D. , Chief Medical Officer; Neil Gibson, Ph.D., Chief Scientific Officer; and Dan Chevallard, Vice President, Finance and Accounting.

During today's call, Kleanthis will provide introductory remarks, following Neil will provide an update on our microRNA therapeutic programs and Dan will summarize our second quarter 2014 financial results. Following your questions, Kleanthis will wrap up the call.

Before we begin, I would like to remind you that this call will contain forward-looking statements concerning Regulus' future expectations, plans, prospects, corporate strategy and performance, 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 filings with the SEC.

In addition, any forward-looking statements represent our views only as of the date of this webcast, and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligations to update such statements.

At this time, I would like to turn the call over to Kleanthis.

Kleanthis G. Xanthopoulos

Thank you, Amy. Welcome everyone and thank you for joining us this afternoon. The second quarter of 2014 has been a productive period for Regulus. We made solid advancements over the last quarter in recent weeks on both the corporate and scientific fronts which continue to solidify our leadership position in the field of microRNA therapeutics.

Let me start with the business front. Yesterday we announced a significant highlight that further advances our promising biomarker technology under our Regulus microMarkers division. We entered into a new agreement and expanded our relationship with Biogen Idec to identify microRNA signatures for the treatment of multiple sclerosis. Under the terms of the new agreement we’ll receive $2 million upfront in our eligible future milestone payments related to the identification of microRNA signatures for MS.

The expansion is a continuation of the work performed since we began our collaboration with Biogen Idec in August 2012. Previously we conducted the largest known MS profiling study and profiled over 400 serum samples from healthy volunteers in MS patients. In that study using microRNA expression as indicators, we differentiated between healthy and diseased tissue from serum and this work provided the foundation for the next step of our research with Biogen Idec.

We now are very excited to apply our technology platform to approximately twice as many samples. Importantly, we will now profile whole blood samples from a cohort MS patients treated with Biogen Idec MS therapy.

Overall, we’re very pleased with the advancements in our biomarker work and believe that we’ve developed a highly robust reproducible platform to extract, profile and analyze microRNA. We look forward to continue to build out this platform to support our pipeline and our collaborators and partners programs.

On the corporate front we were pleased to be able to recruit the best talent which is essential to maintain our leadership in the microRNA field. In June, Dr. Paul Grint joined us as Chief Medical Officer, a critical member of our Executive Management Team and he will responsible for advancing and expanding our clinical microRNA pipeline. Paul is a recognized biotech leader with more than 20 years of experience in biologics and small molecule drug development, including the successful development of numerous commercial products in oncology, anti-infectives and immunology for both domestic and international markets. Paul’s experience and guidance is a tremendous asset to our team as we continue to build a meaningful clinical portfolio. As we already told you, you’ll be hearing from Paul later on the call.

Lastly, on the scientific front, our confidence in our innovative approach to treating disease with microRNA therapeutics is now higher than ever. We know that the biology of microRNA is well suited to treat complex, multi-factorial diseases and we believe that microRNA dis-regulations will provide us with tremendous opportunities to apply our technology platform across many disease areas.

To that end, we focus internally and with our partners for several attractive therapeutic opportunities in oncology, orphan diseases such as Alport Syndrome, metabolic and inflammatory diseases and HCV. Our goals under the Clinical Map Initiative remain on track. The phase 1 clinical study of RG 101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of HCV is progressing very well and we tend to communicate there before year end.

We are also on track to file an IND to test RG 012, an anti-miR targeting microRNA 21 for the treatment of Alport Syndrome in clinical studies and our preclinical portfolio continues to also mature. In addition to our growing confidence in our technology, the broader RNA therapeutic space continue to mature with late stage clinical advancements in both anti-sense and RNAI and attention from large pharma is evidenced by a [relevant] [ph] acquisition announced early this week.

When I reflect on RNA therapeutics and the transformative potential that they represent, I think our [many products disruptive] [ph] therapeutics that have had a great impact on human health. Small molecule therapeutics, protein therapeutics and lately and specifically monoclonal antibodies. However, all these great approaches can only target about 2% of the human genome collectively. I believe that the next evolution of drug development is based on RNA therapeutics which have the ability to target everything that is transcribed in human genome which is about 60%.

With our growing confidence in our technology and the continued advancements in the overall space, we believe microRNA therapeutics will become a new and major class of drugs with broad therapeutic application to disrupt a traditional drug development model.

I’ll now turn it over to Paul who is going to take us through our clinical pipeline. Paul?

Paul C. Grint

Thank you very much Kleanthis and good afternoon to everyone. I’m delighted to join the experienced Regulus team as Chief Medical Officer and I’m excited about the clinical potential of targeting microRNA.

On today’s call, I’ll focus my remarks on RG 012 program and will then turn the call over to Neil to review the RG 101 program, our preclinical portfolio and our Regulus microMarkers division.

RG 012 is an anti-miR targeting microRNA 21 for the treatment of Alport Syndrome, a life threatening genetic kidney disease with no approved therapy and is a key program under Clinical Map Initiative. Recently we were pleased to receive orphan drug designation for RG 012 from the U.S. FDA. According to the FDA, the orphan drug designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis for prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

Alport Syndrome meets these criteria and is an orphan disease. The disease is driven by genetic mutation in the type IV collagen family of proteins. The impact of the mutation in the collagen gene results in disruption to the structure of glomerular basement membrane, increased expression of microRNA 21, increased fibrosis and progressive loss of renal function which eventually leads to end-stage renal disease, requiring dialysis or kidney transplantation.

This orphan drug designation is the first to Regulus and underscores our focus on treating orphan diseases like Alport Syndrome. We’re encouraged by the FDA’s recognition for the need of innovative new treatments such as microRNA therapeutics to treat orphan disease. Following helpful discussions with the U.S. FDA at the recent pre-IND meeting regarding our clinical development plans for RG 012, we expect to file an IND application by the end of this year.

In the near term we expect to initiate the natural history of disease study to learn more about the actual progression of Alport Syndrome by documenting the change in certain renal biomarkers and glomerular filtration rate or GFR. In the first half of 2015, we expect to initiate a phase 1 clinical study in normal healthy volunteers and then transition to Alport Syndrome patients in a phase 2 proof of concept study due to start later next year.

In addition to the emerging clinical map of RG 012, we expect to strengthen this preclinical profile over the next several quarters. In prior preclinical studies we demonstrated potent inhibition of microRNA 21 both in vitro and in vivo RG 012. The decrease in rate progression in renal fibrosis and up to 50% increase in life span of the mice harboring a collagen 403 mutation.

Later this year, we expect to report our first preclinical combination data of RG 012 with the Angiotensin Converting Enzyme or ACE inhibitor. ACE inhibitors are now emerging as the standard of care in patients with Alport syndrome used to treat proteinuria or abnormal amounts of protein in the urine, a key indicator of chronic kidney disease. Together with the key opinion leaders, we believe that ACE inhibitors are not sufficient to treat the actual progression of the Alport syndrome. The preclinical combination data will be important to demonstrate the utility of adding a microRNA therapy to this emerging standard of clinical care. We look forward to reporting continued progress on this exciting program.

I will now turn the call over to Neil to summarize our RG 101 program, our preclinical portfolio and our biomarkers platform. Neil?

Neil W. Gibson

Thank you, Paul. During the second quarter we were pleased with our scientific progress across our therapeutic pipeline within our Regulus microMarkers division. Under the Clinical Map Initiative, we continue to evaluate RG 101 in the Phase 1 study and as Paul mentioned, we continue to advance RG 012 towards clinical development. We are also on track to nominate third clinical candidate by the end of the year.

The Phase 1 clinical study of RG 101, a GalNAc conjugated anti-miR targeting miR 122 for the treatment of HCV is currently ongoing and consists of four parts. Part one is a single ascending dose in healthy volunteer subject. Part two, a multiple ascending dose study in healthy volunteer subject. Part three is a single dose drug interaction study of RG 101 in combination with an approved direct acting anti-viral in healthy volunteer subject. And part four is the single dose study in HCV patients to assess the safety and viral reduction which is designed to demonstrate human proof of concept.

The primary objective in the study is to evaluate safety and tolerability and the secondary objective is to evaluate pharmacokinetics viral load reduction and any impact on oral direct acting anti-viral may have on the pharmacokinetics of RG 101.

Up till 100 healthy volunteers subjects in HCV patients are planned to be enrolled in the study. By the end of the year we expect to report human proof of concept results from part four of the study, which is a key corporate goal under our Clinical Map Initiative. If favorable, these results may validate our microRNA technology platform and may set the stage for success in future clinical trials across the pipeline. In addition to the progress on RG 101, we also continue to pursue several additional microRNA targets to potentially expand our therapeutic pipeline. During the quarter we continued to optimize anti-miRs in multiple therapeutic programs including miR 21 and miR 221 in oncology, miR 19 for a number of oncology indications and miR 103, 107 in metabolic diseases for potential clinical development.

Under the clinical map initiative, we intend to nominate a third candidate for clinical development which may come from proprietary efforts or from a partner program at the end of the year. Lastly, we also continue to advance our biomarkers technology platform within our Regulus microMarkers division. As Kleanthis mentioned earlier on the call, we at least have expanded a relationship with Biogen Idec to identify microRNA as biomarkers for MS. Under the scope of the new work, we will apply our technology platform to larger number of whole blood samples from a cohort of MS patients treated with Biogen Idec therapy.

To-date we profiled approximately 3,000 clinical samples in a wide variety of disease state including MS, chronic kidney disease, fatty liver, rheumatoid arthritis and others. And these new experiments will significantly strengthen our platform to extract profile in our microRNA. In addition to the MS, we have an arrangement with another leading commercial stage pharmaceutical company to explore microRNAs as biomarkers for specific patient populations. And we maintain several academic research collaborations focused on the identification of microRNAs as biomarkers in multiple disease areas.

The Regulus microMarkers division also supports their own therapeutic pipeline. For example, in the upcoming natural historic disease study for RG 012, we plan to apply a Regulus microMarkers technology to look for microRNA signatures in both the urine and blood of the Alport syndrome patient.

To summarize, it's been a very productive quarter and recent periods for us on the scientific front and we look forward to reporting additional progress.

With that I will turn the call over to Dan to summarize our second quarter 2014 financial results.

Dan Chevallard

Thank you, Neil and good afternoon. During the second quarter of 2014, we continued to maintain a strong financial position and ended the quarter with $103.5 million in cash, cash equivalents and short term investments. We recognized revenue of approximately $700,000 in the second quarter 2014 compared to revenue of $4.8 million for the same period in 2013. Revenue during these periods consisted primarily of amortization of upfront payments received from our strategic alliances and collaboration, which we recognize over our estimated period of performance.

As Kleanthis mentioned earlier on the call, we will receive $2 million as an upfront payment from Biogen Idec associated with our new collaboration agreements. This upfront payment will have been received after the close of the second quarter 2014 and no revenue recognition associated with the payment was included in the second quarter of financial reported today.

Total operating expenses were $13.8 million in the second quarter of 2014 compared to $9.4 million for the same period in 2013. More specifically, our research and development expenses were $10.8 million in the second quarter of 2014 compared to $7.7 million for the same period in 2013. This increase was primarily driven by cost associated with our Clinical Map Initiative including our Phase 1 clinical study of RG101, R&D enabling cost for RG012 and the continued advancement of other therapeutic programs.

We expect our research and development expenses to continue to increase as we initiate additional clinical studies and IND enabling our other regulatory filing activities in the future. Our general and administrative expenses were $3 million in the second quarter of 2014 compared to $1.7 million for the same period in 2013. This change was primarily driven by an increase in salaries and related employee cost and other operating expense associated with general business activity.

Our net loss for the second quarter of 2014 was $12 million compared to a net loss of $7.3 million for the same period in 2013. Our resulting basic and diluted net loss per share was $0.28 and $0.29 respectively for the second quarter of 2014 compared to $0.20 for the same period in 2013.

As previously guided, we expect to finish 2014 with at least $75 million in cash, cash equivalent and short-term investments.

With that, I like to turn it back over to Kleanthis to wrap up the call.

Kleanthis G. Xanthopoulos

Thank you, Dan. In closing, we’ve had a very productive first half of 2014 on the business and scientific fronts and our confidence in our microRNA technology continues to grow. Here is why we are excited about our business. Over the next several quarters we have multiple important catalysts in the horizon under our Clinical Map Initiative. By the end of the year, we have a unique opportunity to report our first human proof of concept results with RG 101, which favorably may validate our microRNA technology in advanced to broader RNA therapeutic space.

In the coming weeks, we expect to advance the RG 012 program by initiating a natural history of disease study to gather greater information about the progression of Alport syndrome in patients. And we expect to expand our therapeutic pipeline by nominating a third microRNA candidate for clinical development before the end of the year thus setting the stage to have three clinical programs in 2015. In short we are pleased with our recent accomplishment and we hope to extend our good track record through the remainder of the year.

With that let me now open the forum for questions. Operator?

Question-and-Answer Session

Operator

(Operator Instructions) And the first question is from Eric Schmidt of Cowen & Company. Your line is open.

Eric Schmidt – Cowen & Company

Good afternoon and thanks for taking my questions. I guess first on RG 101 for I guess maybe Kleanthis or Paul, just wondering what the forum is going to be for the release of data by year end, is that a press release or a medical conference?

Paul C. Grint

This is Paul, that's a good question, I mean clearly once we have all the data and information in, we will be looking at it and we will decide what the best way to release that information is, at this point in time we haven’t firmly decided which is the best way to go.

Kleanthis G. Xanthopoulos

Eric, if you think AASLD is typically towards the end of October, I think we are not going to be ready for that, it’s a little too early. The next big conference is into 2015. So most likely we are going to have either a press release followed by a call or some sort of a broader communication.

Eric Schmidt – Cowen & Company

Okay. Thanks for that clarification. On RG 012 and the phase 1 study in healthies, is that purely a safety study or do you think you will see or even try to identity anything in the biomarker arena that might be [inaudible] related?

Paul C. Grint

So, this is Paul again, we are currently finalizing the design of that study, I mean, the key goals of the study is, it is a first in man study for RG 012, so the goal primarily is - the plan is going to be single ascending dose study. So the key end points there obviously is safety, tolerance and PK. We will be taking samples and obviously we’ll wait to see what we see in the study. But generally this is in otherwise fit, healthy, relatively young people, the key goals I’ve already outlined.

Eric Schmidt – Cowen & Company

I guess last question, it sounds like you are just talking more and more about the role of microRNAs in rare orphan diseases. Should we expect that the entire sort of next set of clinical candidates from Regulus internally are going to be directed at orphan disorders?

Kleanthis G. Xanthopoulos

Not necessarily Eric, we let biology speak for the indication, for example, one of the programs we highlighted today, 103, 107 is in metabolic diseases hardly definition of orphan apply to that. But, in a lot of our cancer programs once you essentially stratify the patient population who may be considered orphan, but it's not our strategy to only prosecute orphan indications. We are going to let the biology microRNA speak to us.

Eric Schmidt – Cowen & Company

Okay. Thanks a lot.

Kleanthis G. Xanthopoulos

Thank you.

Operator

Thank you. The next question is from Alan Carr of Needham & Company. Your line is open.

Unidentified Analyst

Hi guys, this is actually Mark on for Alan. Thanks for taking my call, my question, I was wondering if I could ask a little bit more about the biomarker collaboration with BI, and just biomarkers sort of in general. I know that profiling messenger RNA and using microarrays has always been problematic in the past in terms of doing [inaudible] medicine. Do you guys think that microRNA has a different success rate than we have seen microarrays have in the past?

Kleanthis G. Xanthopoulos

Mark, let me make a few comments and I will let Neil get into a more detailed answer because it's a very thoughtful and insightful question. Yes, we do believe microRNA is potentially are better bioanalytes than messenger RNA for a variety of reasons. Starting with complexity alone, you are looking at about 800 or so human microRNAs that we know are functional and have been identified in the human genome. Obviously, a fraction of that 23,000 to 30,000 messenger RNA especially if include alternative splicing variance. So, simplicity is key. Secondly, microRNA have involved to control pathways and as such they report changes in entire pathways as opposed to individual genes and we view this as more, philosophically much more important to look at the micronorm as opposed to the transcript norm. And I think so far the data are very solid. Naturally, we don't expect for every disease there is going to be a microRNA that determines the state of that disease but for most of the diseases that we looked in we are very satisfied with our results so far. I will turn it over to Neil for further comments.

Neil W. Gibson

Only to add Mark that we are focusing obviously on a number of bodily fluids including blood, serum, urine etcetera and, we don't have to actually access tissue and the concept being that the disease state where it will actually manifest itself by difference in the microRNA pattern that we see in those bodily fluids compared with the normal state, and that will give us the ability to pick up signatures of both predictive and prognostic [inaudible].

Unidentified Analyst

Great. Thanks very much.

Operator

Thank you. The next question is from Jim Birchenough of BMO Capital Markets. Your line is open.

Unidentified Analyst

Good afternoon, it’s Nick in for Jim this afternoon. So just carrying on that thing Neil, I think you just sort of really identified the goal here is the disease state prediction and prognosis, but one could also imagine that for a drug that maybe have some acute side effects that one could look at, a biomarker indicative of the onset of acute side effects or let's say another drug in an unnamed company’s portfolio was associated with an infectious disease with very severe outcomes and maybe you are looking at what is sort of canary in the coal mine here for recapitulation of that disease or that disease appearing in a form that you don’t want. So is it just looking at diseases or so the disease tissue or can you look at either acute or chronic safety - potential safety signal as well?

Neil W. Gibson

We can look at both the potential or efficacy associated with the therapy as well as potential toxicity if you will that may not be observed by traditional means. So, there are a number of papers out there that have started to look at that. They have used liver injury as an example where with a couple of liver microRNAs they we can find in blood or serum in a much more sensitive way than even ALT to get to your exact point Nick. So, I think the opportunity is there to do exactly that both for markers or effects of efficacy as well as potential effects from a toxicity perspective.

Unidentified Analyst

Okay. Thanks and then on 101, so obviously you have data in man now. Anything that has surprised you about the very early clinical experience?

Paul C. Grint

So this is Paul. Right now currently the study is still ongoing, I mean, the nature of the design it is, it is the placebo controlled blinded study so obviously we are waiting until the study is completely finished going through the formal process of data cleanup, database log before we’re ready, looking at that information we will be able to comment on it.

Unidentified Analyst

Okay. So nothing very untoward has happened there, I think we can conclude. And then, on 012, you mentioned doing some combination with ACE inhibitors, is there anything about the biology of ACE inhibition and miR 21 inhibition that would lead you to think there could be additive effects or even a synergy?

Kleanthis G. Xanthopoulos

I will let Neil comment. And I will also, I can comment afterwards, Neil.

Neil W. Gibson

I will need to see in excess, we believe that ACE inhibitor and inhibition of miR 21 are tackling different mechanisms and thus different pathways and so the expectation is that at a minimum, they would be additive and hopefully there to more than additive. But that's the experiment that Paul discussed that we’re – be talking about in upcoming conference later this year when the data has matured in a manner that will allow us to talk about it publicly.

Paul C. Grint

Okay. Nick, to add to that philosophically we believe that microRNA as I said before control an entire pathway and maybe an individual therapy targets, a member of that pathway. So because of the (inaudible) what we believe to be a transformative nature of microRNA therapy, we don't see anything, but potential positive outcome for combining a microRNA inhibitor we’ve a direct therapy and we already have an example like that in our preclinical work with microRNA 122 and a inhibitor for HCV and we think that phenomena would most likely be replicated in the microRNA 21 for Alport in combination with an ACE inhibitor and we also believe that we can see that in additional diseases. So, we are exploring that very, very interesting avenue right now.

Unidentified Analyst

Thanks a lot for taking my questions.

Operator

Thank you. The next question is from Bill Tanner of FBR Capital Markets. Your line is open.

Bill Tanner – FBR Capital Markets

Yes, thanks for taking the question. I think couple for Neil and Kleanthis maybe one for you. Neil just as it relates to Biogen agreement, I am just curious, just read the press release, I mean it certainly reads like you guys have seen some data from some of the work that has been done thus far, so could you just explain maybe whatever you can about the mechanics as to why the 2012 agreement was terminated and new one was entered just the rationale for that and what actually prompted that?

Neil W. Gibson

Well in essence, the initial studies that we did with Biogen Idec were focused initially on validating the platform if you will and we successfully achieved that goal and then moving onto an assessment of microRNA signatures that we would see in serum of MS samples that they had collected. Now, this is now an extension and expansion of that if you will into looking at microRNAs in blood and looking at in samples from well controlled clinical studies that Biogen Idec have performed. So, it's really a natural extension and it's moving to expand the analyzed, we analyze in a number of different sample sets blood, serum etcetera.

Bill Tanner – FBR Capital Markets

Okay. That makes sense.

Kleanthis G. Xanthopoulos

Bill to comment a little further here. Obviously, no one would have liked to re-engage into an extended collaboration if that first phase wasn’t successful, I think you can assume that the collaboration, the first levers of collaboration was very successful. Now technically, the way the contract was done, we needed to terminate the first phase and then engage into a new contract which is what we announced.

Bill Tanner – FBR Capital Markets

Okay. Alright that makes sense. I guess, as it relates to 101 then, Neil obviously we are hoping that it's safe, but as you get to the readouts for the HCV infected individuals what would constitute – what’s kind of the no go, no go I am assuming that it's a viral (inaudible) reduction and maybe just in the sense that I guess you would hate to come up with an answer that's sort of in the middle versus definitely positive or definitely negative. So unfair maybe to ask you to predict the future, but what’s the likelihood you think that you will get a definitive answer one way or the other?

Neil W. Gibson

I mean, the discussions we have had with our key opinion leaders have really focused, and if we are not seeing two log viral load reduction in HCV patients, then that would cause us to take a pause and really think hard about the program so that just gives you a level of what we have been thinking about in discussions with key opinion leaders.

Bill Tanner – FBR Capital Markets

So then, you think that you would have scanned the range of doses that I guess you could possibly give to an MTD or something like that that if you didn’t see if there is any distinct okay, this is -- we are not getting it two log reduction so this is maybe just not going to work?

Neil W. Gibson

The way the study is set up is, obviously with once a month dosing and using the information from the single dose and healthy volunteers to really help select the dose for the HCV patients. So, we do believe that we have an appropriate path in place that will help us, be able to evaluate an appropriate dose in HCV patients.

Bill Tanner – FBR Capital Markets

Okay. That's correct and Kleanthis I had a question for you and it's maybe an unfair question, but I just will be interested in your thoughts on the [Roach] proposed acquisition of Santarus, obviously you guys know well who your competitors are and I am just curious your perspective has to the disease look like or do you have a thought that there may be some assets that Roach is interested in, do you think that they want access to the technology just a general perspective?

Kleanthis G. Xanthopoulos

Happy to comment Bill, but I do want to add a few more things to what we’re just discussing about 101, in that of course the design is such to demonstrate safety, tolerability pharmacokinetics of the compound, remember it's the first ever compound that has a GalNAc conjugated chemistry, sugar chemistry that came from [inaudible] and of course it's the first time dimmers that we’re testing so we see this as huge product validation if the whole study and the molecule been exposed not to over 100 subjects.

Let's say in terms of end point, when we unlock the data and communicate it, remember we look at a mono therapy, we are going to be reading out a single dose sub queue over 30 days and we are going to be looking as a mono therapy for viral load reduction. So anything above two logs in for a single dose of a 30 days is going to be incredibly interesting to us given that in combination of that with any direct anti-viral will immediately bring the virus down to undetectable. So that class of act that we hope to have multiple genotypes because 122, we think will act and work equally well on genotype 1 and genotype 3 and many other genotypes.

Now back to your question. I mean, to me that symbolizes the interest that continues to exist in RNA therapies specifically with Roach is of course a little strange given that they had a huge investment in RNAI therapies we’ve (inaudible) and of course they divested that completely so that's how (inaudible) only to turn back. But strategically we have seen that happen so many times in pharma, different strategic directions. In this case, I think the (inaudible), John has been on the board of ISS for years so he knows RNA therapies, he knows anti-sense technologies and I am sure he thought that it's a very good first step for us to re-engage.

Bill Tanner – FBR Capital Markets

Makes sense. Okay thanks very much.

Operator

Thank you. The next question is from Christopher James of Brinson Patrick Securities. Your line is open.

Christopher James – Brinson Patrick Securities

Hi, thanks for taking my questions and congrats on the recent progress. Can you expand perhaps on your discussion from the pre-IND meeting with the FDA, specifically can you characterize your discussion around approvable end points and maybe path to approval in Alport syndrome?

Paul C. Grint

Chris, this is Paul, you are correct, I mean that was in the not surprising subject of positive discussions, it is early at this stage. What’s important I think to consider is the natural history study that we are doing, basically we are going to be starting this program in the near future and in essence it's almost like a phase two study, but without a therapeutic intervention and what this allows us to do is to, in a good controlled or the clinical trial fashion follow these patients prospectively and on the relatively frequent basis we are looking at, clearly at major GFR which is an important end point. As well as other biomarkers and data from that study together with ongoing discussions will help elucidate the types of regulatory end points of the FDA it going to want to see or other regulatory agents that want to see at some point in the future for approval in this indication. So the pre-IND was really the – with the start of the discussion.

Christopher James – Brinson Patrick Securities

Got it, it's helpful. Thank you.

Kleanthis G. Xanthopoulos

Neil, do you have any more comments or –

Neil W. Gibson

No I think Paul has summed up nicely.

Christopher James – Brinson Patrick Securities

Okay. Great and then moving on to the Biogen collaboration, are you – will you be looking at subtypes of MS other than the relapsing form such as secondary progressive or primary progressive or are you specifically looking at just the relapsing form?

Paul C. Grint

Chris, this will be looking at number of different samples that have been collected in the Biogen Idec clinical studies. So, I can't really comment in more detail than that at this time. They will actually allow us to evaluate the microRNA profiles in samples that have been collected under well controlled conditions.

Christopher James – Brinson Patrick Securities

Great, thanks and congrats.

Operator

Thank you (Operator Instruction) and the next question is from Liana Moussatos of Wedbush Securities, your line is open.

Liana Moussatos – Wedbush Securities

My question has been answered. Thank you.

Operator

Thank you. There are no further questions in queue at this time. I would like to turn the call back over to Kleanthis for closing remarks.

Kleanthis G. Xanthopoulos

Thank you all for joining us this afternoon. We are, as we stated very pleased with our recent accomplishments and look forward to communicating the progress review over the next few months.

Operator

Ladies and gentlemen, that concludes today's conference call, thank you for your participation. You may not disconnect. Good bye.

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