Regulus Therapeutics Inc (RGLS) 2014 Q2 法說會逐字稿

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.

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, PhD, President and Chief Executive Officer; Paul Grint, MD, Chief Medical Officer; Neil Gibson, PhD, Chief Scientific Officer and Dan Chevallard, Vice President Finance and Accounting.

During today's call, Kleanthis will provide introductory remarks, Paul and 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 statement represents 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 the obligation to update such statements.

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

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 and 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 advancements of 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 will 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 are now very excited to apply our technology platform to approximately twice as many samples.

Importantly, we will now profile whole blood samples from a cohort of MS patients treated with Biogen Idec MS therapy. Overall, we are very pleased with the advancement in our biomarker work and believe we that have developed a highly robust reproducible platform to extract, profile, and analyze mircoRNAs. We look forward to continue to build out this platform to support our pipeline in our collaborators and partners programs.

On the corporate front, we're 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 executive management team and he will be 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 in the call.

Lastly on the scientific front, our confidence in our innovative approach in treating disease with microRNA therapeutics is now higher than ever. We know that the biology of microRNAs is well-suited to treat complex multi-factorial diseases and we believe that microRNA disease regulation should provide us with tremendous opportunities to apply our technology platform across many of disease areas. To that end we focus internally and with our partners on 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 one clinical study of RG-101, GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of HCV, is progressing very well and we intend to communicate data before year's end. We are also on track to file an IND to test for RG-102 anti-miR targeting microRNA-21 for the treatment of Alport syndrome in clinical studies, and our pre-clinical portfolio continues to mature. In addition to our growing confidence in our technology, the broader RNA therapeutic space continues to mature with late stage clinical advancements in both antisense and RNAi [and attention from lots pharma] by a relevant acquisition announced earlier this week.

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

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

I will now turn it over to Paul, who's going to take us through our clinical pipeline. Paul?

Thank you very much Kleanthis and good afternoon to everyone. I am delighted to have join the experienced Regulus team as Chief Medical Officer, and I'm excited about the clinical potential of taunting microRNAs. On today's call, I will focus my remarks on the RG-102 program and then will then turn the call over to Neil to review the RG-101 program, our pre-clinical portfolio in our Regulus microMarkers division.

RG-102 targeting is an anti-miR microRNA 121 for the treatment of Alport syndrome, a life threatening genetic kidney disease with no approved therapy and is a key program under our clinical map initiative. Recently, we were pleased to receive orphan drug designation for RG-102 from the US FDA.

According to the FDA, the orphan drug designation program provides orphan status to drugs and biologics which are defined as both intended for the safe and effective treatment, diagnosis, or prevention of rare disease 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 mutations in the type IV collagen family of proteins. The impact of the mutation in the collagen gene results disruption of the glomerular basement membrane, increased expression of micro RNA 21, increased fibrosis and progressive loss of renal function, which eventually leads to end-stage renal disease requiring dialysis or kidney transplantation.

This orphan destination is the first for Regulus and underscores our focus on treating orphan diseases like Alport syndrome. We are encouraged by the FDA's recognition for the need of innovative new treatments, such as microRNA therapeutics to treat orphan diseases. Following helpful discussions with the US FDA at a recent pre-IND meeting regarding our clinical development plans for RG-012, we to file an IND application by the end of this year.

In the near term, we expect to initiate a 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 rates with 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 for RG-012, we expect to strength its pre-clinical portfolio over the next several quarters. In prior pre-clinical studies we demonstrate potent inhibition of microRNA 21 both in vitro and in vivo with RG-012, a decrease in the rates projection renal fibrosis and up to a 50% increase in lifespan of the mice harboring a cold 4A3 mutation.

Later this year, we expect to report our first pre clinical combination data of RG-012 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 indication of chronic kidney disease.

Together with the key opinion leaders we believe that ACE inhibitors are not sufficient to treat the actual progression of Alport syndrome. The pre-clinical combination data will be important to demonstrate the utility of adding a microRNA to this emerging standard of clinical care. We look forward to reporting continued progress on this exciting program.

I'll now turn the call over to Neil to summarize our RG-101 program, our pre-clinical portfolio and our biomarkers platform.

Neil?

Thank you, Paul. During the second quarter we were pleased with our scientific progress across our therapeutic pipeline and within our Regulus microMarkers division.

Under the Clinical Map Initiative we continued to evaluate to RG-101 in the phase 1 study in as Paul mentioned we continue to advance RG-012 towards clinical development. We're also on track to nominate a third clinical candidate by the end of the year.

The phase 1 clinical study of RG-101, a GalNAc-conjugated anti-miR targeting miR122 for the treatment of HCV is currently ongoing and consists of four parts. Part one is a single ascending dose in healthy volunteer subjects. Part two a multiple ascending dose study in healthy volunteer subjects. Part three is a single dose drug-drug interaction study of RG-101 in combination with an approved direct acting antiviral in healthy volunteer subjects. And part four, a single dose study in HCV patients to assess the safety and viral load reduction which is designed to demonstrate human proof of concept.

The primarily objective in the study is to evaluate safety and tolerability and the secondary objective is to evaluate pharmacokinetics, viral load reduction and any impact an oral direct-acting antiviral may have on the pharmacokinetics of RG-101 Up to 100 healthy volunteer subjects and HCV patients our planned to be enrolled in the study.

By the end of the year, we expect to report human proof of concept results from part 4 of the study, which is a key corporate goal under our clinical mapping initiative. If favorable, these results may validate our microRNA technology platform in 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 mirR-21 and miR-221 in oncology, miR19 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 our proprietary efforts or from a partner program by 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 are pleased to have expanded our relationship with Biogen Idec to identify microRNAs as biomarkers for MS. Under the scope of the new work, we will apply our technology platform to a larger number of whole blood samples from a cohort of MS patients treated with a Biogen Idec therapy. To date, we have profiled approximately 3,000 clinical samples in a wide variety of disease states, including MS, chronic kidney disease, fatty liver, rheumatoid arthritis and others. These new experiments will significantly strengthen our platform to extract, profile and analyze microRNAs.

In addition to the MS work, 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 history disease study for RG-012, we plan to apply 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 period for us on the scientific front and we look forward to reporting additional progress.

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

Thank you, Neil, and good afternoon. During the second quarter of 2014, we continue 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 of 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 collaborations, which we recognized over our estimated period of performance.

As Kleanthis mentioned earlier on the call, we will received $2 million as of upfront payment from Biogen Idec associated with our new collaboration agreement. 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 financials reported today.

Total operating expenses were $13.8 million in the second quarter of 2014, compared to $9.4 million for the same period 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 costs associated with our Clinical Map Initiative, including our phase 1 clinical study of RG-101, IND enabling costs for RG-012 and continued advancement of other therapeutic programs. We expect our research and development expenses to continue to increase as we initiate additional clinical studies in IND enabling or 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 costs and other operating expenses associated with general business activities.

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 equivalents, and short-term investments.

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

Think you, Don,. In closing, we had very productive first half of 2014 on the business and scientific front and are confident in our microRNA technology continues to grow. Here's why we are excited about our business.

Over the next several quarters we have multiple important catalysts on the horizon under our Clinical Map Initiative. The end of the year, we have a unique opportunity to report our first human proof of concept results with RG-101. We see favorably may validate our microRNA technology and advance the broader RNA therapeutic space.

In the coming weeks expect to advance the RG-102 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, setting the stage to have three clinical programs in 2015.

In short, we're pleased with our recent accomplishments and we hope to extend our good track record through the remainder the year. With that, now let me open the forum for questions. Operator?

(Operator Instructions)

Eric Smith, Cowen and Company.

Thanks for taking my questions. I guess first on RG-101 for, I guess maybe for Kleanthis or Paul, just wondering what the [form] is going to be for the release of data by year end? Is that a press release or a medical conference?

This is Paul. That's a good question. Clearly, once we have all the data and information in, we will be looking at it and 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.

And if you think (inaudible) typically towards the end of October, I think we're not going to be ready for that. It's a little too early. The next big conference is into 2015, so most likely we're going to have either a press release followed by a call or some sort of a broader communication.

On RG-102 and the Phase I study in the HealthEase, is that purely a safety study or do you think you'll see or even try to identify anything in the biomarker arena that might be [trotafact] related?

This is Paul again. We're currently finalizing the design in that study. The key goal of the study is it is a first-in-man study for RG-012, so the goal primarily is -- the plan is it's going to be a single ascending-dose study, so the key end points there are obliviously safety, tolerance and PK.

We will be taking samples and obviously will 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.

I guess last question, it sounds like you're 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?

Not necessarily, Eric We let biology speak for the indication.

For example, one of the programs we highlighted today, 103, 107 is a metabolic diseases, hardly a definition of orphan apply to that, but -- and a lot of our cancer programs, once you essentially stratify the patient population, may be considered orphan, but it's not our strategy to only [prosecute] orphan indication. We are going to let the biology microRNA speak to us.

Okay, thanks a lot.

Alan Carr, Needham & Company.

Hi, guys, it's actually Mark, on for Alan. Thanks for taking my call. I was wondering if I could ask a little bit more about the biomarker collaboration with [BI] -- biomarkers in general? I know that profiling [Messenger] RNA and using micro [raisers] has been problematic in the past in terms of doing predictive medicine. Do you guys think that microRNA has a different success rate than we've seen micro [rates] have in the past?

Let me make a few comments and then I'll let Neil get into a more detail answer because it's a very thoughtful and insightful question. Yes. We do believe microRNAs potentially are better [bionalyzed] than Messenger and then for a variety of reasons.

You're looking at -- starting with the complexity alone, you're 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 species, especially if you include alternative splicing variants, so simplicity is key.

Secondly, microRNAs have [involve] to control pathways. In [asat], they report changes in entire populations individual -- as opposed to individual genes, and we view this as more -- philosophically much more important to look at the [micronome] as opposed to the [transcriptor]. 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 disease that we looked in, we are very satisfied with our results so far.

I'll turn it over to Neil for further comments.

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

Great. Thank you very much.

Jim Birchenough, BMO Capital Markets.

Good afternoon. It's Nick in for Jim this afternoon. Just carrying on that theme, Neil, I think you just really identified that the goal here is the disease state, prediction and prognosis, but one could also imagine that for a drug that maybe has some acute side effects, that one could look at a biomarker indicative off 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 looking at what is the 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 diseased tissue or can you look at either acute or chronic safety -- potential safety signals, as well?

We can look at both the potential for efficacy associated with the therapy as well as potential toxicities, if you will, that may not be exempt by traditional means. So there are a number of papers out there that I have started to look at that they've used -- liver injury is an example where, with a couple of liver microRNAs that they can find in blood or serum in a much more sensitive way than even [ALT] to get to your exact point mix.

So I think the opportunity is there to do exactly that with both for markers or effects on efficacy as well as potential effects from a toxicity perspective.

Okay, thanks. And then on 101, so obviously you have data [remand] now. Is there anything that has surprised you about the very early clinical experience?

This is Paul. Right now the currently -- currently the study is still ongoing. I mean, the nature of the design it is, it is a placebo control-blinded study, so obviously we're waiting until the study is completely finished, going through the formal process of data cleanup and database lock before we are really looking at that information and we'll be able to comment on that.

All right. So nothing very untoward has happened there. I think we can complete. And then on [RO12], you mentioned doing some -- a combination of base inhibitors. Is there anything about the biology of ACE inhibition in [Mir] 21 inhibition that would lead you to think that it could be additive effects or even a synergy?

I'll let Neil comment, then I'll (inaudible) some comments afterward. Neil?

Well, (inaudible) we believe that ACE inhibitors 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, [they too] would be more than additives. But that's the experiments that Paul discussed that we will be talking about in an upcoming conference later this year when the data has matured in a manner that will allow us to talk about it properly.

To add to that, philosophically we believe that microRNAs, as I said before, control an entire pathway. It may be an individual therapy targets a member of that pathway. So because of the trip -- what we believe to be a transformative nature of the microRNA therapy, we don't see anything but potential positive outcome for combining a microRNA inhibitor with a direct therapy. And we already have an example like that in our preclinical work with microRNA 122 and the [protest] inhibitor for [HCV]. And we think that phenomenal will most likely be replicated in the microRNA 21 for all ports 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.

Thanks a lot for taking my questions.

Bill Tanner of FBR Capital Markets.

Yes, thanks for taking the question. I think couple for Neil and then, Kleanthis, maybe one for you. Neil, just as it relates to the Biogen agreement, I'm just curious. Just read the press release. 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 a new one was entered, just the rationale for that or what actually prompted that?

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 on to an assessment of microRNA signatures that we'd see in serum of MS samples that we had collected. And now this is now an extension and expansion of that, if you will, into looking at microRNAs in [liters] and looking at it in samples from well-controlled clinical studies that Biogen Idec have performed.

It's really a natural extension and is moving to expand -- analyze -- we analyze in a number of different sample sets, blood, serum, et cetera.

Okay. That makes sense.

Bill, to comment a little further here, obviously no one would have liked to reengage into an extended collaboration if that first phase wasn't successful, right? So I think you can assume that the collaboration -- the first leg of the 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.

All right, then. No, that makes sense. I guess as it relates to 101, then, Neil, obviously we're hoping that it's safe. But as you get to the readouts for the ACV-infected individuals, what would constitute -- what's kind of the no-go, no-go? I'm assuming that it's a viral titer reduction.

Is it just in the sense that -- I guess you'd hate to come up with an answer that's sort of in the middle versus definitively positive or definitely negative, so unfair, maybe, to ask you to predict the future, but where do you -- what's the likelihood you think that you'll get a definitive answer one way or the other?

In the discussions we've had with our key opinion leaders have really focused on -- if we are not seeing a 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've been thinking about in discussion with the key opinion leaders.

So then you think that you would have scanned the range of doses that you could possibly give, I don't know if it's through an NTD or something like that,5 that if you didn't see if there, then you'd just think, okay, this just may -- we're not getting a two-log reduction, so this is maybe just not going to work.

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 CV patient. We do believe that we have an appropriate path in place that will help us be able to evaluate an appropriate dosing in HCV patients.

Okay. And then, Kleanthis, I had a question for you and it's maybe an unfair question, but I would be interested in your thoughts on the Roche proposed acquisition of [Santeros]. I mean, obviously you guys know well who your competitors are and I'm just curious your perspective as to does this look like -- or do you have a thought that there may be some assets that Roche is interested in? Do you think that they want access to the technology? Just your general perspective.

Happy to comment, Bill, but I do want to -- I have a few more things to what we were 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 gallon of conjugate chemistry, sugar chemistry that came from Isis, [circle contrainethyl]. And, of course, it's the first (inaudible) that we're testing. So we see this as a huge product value base if the whole study and the molecule being exposed now to over 100 subjects.

Let's -- in terms of end point, when we unlock the data and communicate it, remember we look at a monotherapy. We are going to be reading out a single dose, a single-dose, subcu, over 30 days, and we are going to be looking as a monotherapy for viral low reduction. So anything above two logs for a single dose over 30 days is going to be incredibly interesting to us, given that -- in combination of that with any direct antiviral will immediately bring the virus down to undetectable.

That, plus the fact that we hope to have multiple genotypes because 122, we think, will act and work equally well on genotype one, genotype threes, and any other genotypes. Back to your question.

To me, that symbolizes the interest that continues to exist in RNA therapies, specifically, with [Ross] is, of course, a little strange, given that they had a huge investment in RNAi therapies with Alnylam, and, of course, they divested that completely. So that's how [arowhead] was born only to turn back, but strategically we've seen that happen so many times in pharma -- different strategic directions. In this case I think the champion within Ross was John [Reid].

John has been on the Board of Isis for years, so he knows RNA therapies, he knows anti[sense] technologies, and I'm sure he thought that it's a very good first step for us to reengage.

Makes sense. Okay, thanks very much.

Christopher James, of 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 provable end points and maybe path to approval in Alport Syndrome?

Chris, this is Paul. You're correct. That was not surprising, subject to the positive discussions. It is early at this stage. What's important, I think, to consider is the natural history study that we're doing.

Basically, we are going to be starting this program in the near future. But, in essence, it's almost like a Phase II 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 a relatively frequent basis.

We are looking at -- clearly at some measured GFR, which is an important endpoint as well as other biomarkers. And data from that study, together with ongoing discussions, will help elucidate the types of regulatory end points that the FDA is going to want to see, or other regulatory agents are going to want to see at some point in the future for approval in this indication.

The pre-IND was really the start of the discussion.

Got it. That's helpful. Thank you.

Neil, do you have any more comments or --

No, I think Paul summed it up nicely.

Great. And then moving on to the Biogen collaboration. 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?

Chris, this will be looking at a 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. But it will actually allow us to evaluate the microRNA in samples that have been collected under well-controlled conditions.

Great. Thanks, and congrats.

(Operator Instructions)

The next question is from Leanna Mistasos of Wedbush Securities.

My question has been answered. Thank you.

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.

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

Ladies and gentlemen, that concludes today's conference call. Thank you for your participation. You may now disconnect. Goodbye.