Luke Heagle - IR, W2O pure Communications William Marshall - President and CEO Jason Leverone - CFO.

Jonathan Miller - Evercore ISI Liana Moussatos - WedBush Madhu Kumar - B Reilly FBR.

Good afternoon everyone, and welcome to the miRagen Therapeutics First Quarter 2018 Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. And as a reminder, today's call is being recorded.

And now I'd like to turn the conference over to Luke Heagle from W2O Pure. Please go ahead, sir..

Thank you, and good afternoon, everyone. On the call today are miRagen's President and Chief Executive Officer, Bill Marshall; Chief Financial Officer, Jason Leverone; Executive Vice President of Research and Development, Paul Rubin; and Chief Business Officer, Adam Levy.

Before we begin, I would like to remind everyone that this conference call and webcast will contain forward-looking statements about the company. These statements are subject to risks and uncertainties that could cause actual results to differ. Please note that these forward-looking statements reflect our opinions only as of the date of this call.

We will undertake no obligation to revise or publicly release the results of any revisions to these forward-looking statements in light of new information or future events.

Factors that could cause actual results or outcomes to differ materially from those expressed in or implied by such forward-looking statements are discussed in greater detail in our most recent filings on Form 10-K and our other periodic reports on Forms 10-Q and 8-K filed with the SEC.

I would now like to turn the call over to Bill Marshall, President and Chief Executive Officer of miRagen. Bill..

Thanks, Luke. Good afternoon, and thank you for joining us for our corporate update call for the first quarter 2018. We had a strong start to the year, with the initiation of two clinical trials, introduction of new preclinical stage ophthalmology programs and completion of refinancing that helps fund the company into early 2020.

We continue to advance, de-risk and build confidence in our pipeline of high potential RNA-targeted therapies, making miRagen uniquely position to bring RNA-targeted therapies to patients in need. First, we recently initiated two Phase 1 clinical trials for MRG-110 in collaboration with our partner, Servier.

This represents an important part of our strategy to not only develop product candidates on our own but to collaboratively extend the potential applications of our platform with partners who can bring valuable expertise and resources.

We also unveiled a new preclinical program evaluating mimics of the microRNA-183/96/182 cluster, which has been shown to play an important role in the establishment and maintenance of neurosensory cells, including photoreceptors and hair cells which are associated with hearing.

Last week at the 2018 Association for Research in Vision and Ophthalmology or RVO Annual meeting, we presented preclinical data for mimics of the microRNA-183 cluster, for potential use in retinal degeneration, a pathological condition that impact thousands of patients annually and is characterized by gradual loss of photoreceptors and eventual blindness.

Also at RVO, we presented data from our preclinical work advancing MRG-201, a microRNA-29 mimic as a therapeutic candidate with the potential to treat pathological fibrotic conditions of the eye.

These programs highlight our commitment to leverage the unique power of microRNA biology in biological process control to potentially address areas of large unmet medical need such as vision loss.

Our most advanced program exploring cobomarsen or MRG-106, a microRNA-155 inhibitor as a therapeutic strategy to treat hematological malignancies continues to advance.

We remain on track to initiate the SOLAR Phase II trial evaluating cobomarsen in patients with which is the most common form of cutaneous T-cell lymphoma or CTCL, in the second half of 2018.

As a reminder, we anticipate the trial will evaluate the safety and efficacy of 300 milligram of cobomarsen given by intravenous infusion versus an active control. We expect to enroll approximately 65 patients per treatment group.

Based on discussions with the FDA we believe a successful outcome for the primary endpoint overall response rate of 50% or greater improvement in the severity of a patient's skin disease over the patient's entire body maintained for at least four consecutive months, ORR4 with no evidence of disease progression in the blood, lymph nodes or viscera could potentially allow us to apply for accelerated approval of cobomarsen in the United States.

Yesterday we released additional clinical results from our ongoing Phase 1 study in CTCL, highlighting observations of durable improvements in patient's quality of life. We plan to release additional results at an upcoming medical meeting this quarter.

We are also evaluating cobomarsen in three other indications within the current Phase 1 trial, adult T-cell leukemia lymphoma, diffused large B-cell lymphoma and chronic lymphocytic leukemia. We expect to release interim Phase 1 data in at least one of these expansion indications in the second half of 2018.

Turning to MRG-201, in the first half of 2018 we intend to initiate a double blinded randomized Phase II clinical trial to evaluate MRG-201 in subjects with a pre-disposition for a keloid formation. We are also investigating MRG-201 in preclinical studies for the potential treatment of ocular and lung fibrosis.

Recently at RVO we presented preclinical data suggesting that MRG-201 may function as a potential therapeutic agent to inhibit corneal or retinal fibrosis. The preclinical study showed that delivery of MRG-201 through various routes of administration resulted in functional uptake of the product candidate.

It also demonstrated that MRG-201 reduced the expression of multiple collagens and other microRNA-29 target genes important in fiber genesis, indicating that it may inhibit corneal and retinal fibrosis.

Patients suffering from ocular fibrosis have limited treatment options and this data demonstrates the possible utility of MRG-201 to provide therapeutic benefit to these patients in need. We are also evaluating mirror 29 mimics for the treatment of lung fibrosis and expect to release preclinical in-vivo data later this year.

Now turning to MRG-110, an inhibitor of microRNA-92 and the lead product candidate under our collaboration with Servier, I'm happy to note that MRG-110 is our third product candidate to enter human clinical trials.

In March 2018 Servier initiated their Phase 1 clinical trial of MRG-110 intended to support additional clinical studies that could allow for its eventual use in the potential treatment of heart failure. In that trial MRG-110 is being evaluated for safety and tolerability in a systemic dosing protocol.

The trial is expected to enroll 49 male subjects aged 18 to 45. Under the terms of our agreement with Servier, Miragen earned a milestone of €3 million for the dosing of the first patient in the Phase 1 clinical trial.

In preclinical models MRG-110 accelerated the formation of new blood vessels which resulted in improved vacuolization and improved functional outcomes. We also observed an acceleration in time to healing of wounds that received MRG-110 injections compared to controls treated with placebo or standard of care.

Based on these findings the collaboration decided to evaluate MRG-110 for its potential use in indications such as surgical infusions, severe lacerations or chronic wounds.

In May we initiated a second Phase 1 trial designed to evaluate the safety tolerability and pharmacokinetics of MRG-110 after intradermal injection in healthy volunteers reducing induced wounds through biopsy. The study is also intended to measure a variety of exploratory pharmacodynamic end points.

Such as molecular biomarker changes, blood flow and healing rate. The bio-up marker information for MRG-110 obtained in this clinical trial is also intended to support clinical studies for the treatment of heart failure.

We believe there is a significant need for therapies to treat a variety of wound, that are difficult to care for because of insufficient vascularization. The delay or lack of healing in these patients can result in significant morbidities, including infection, herniation and even death.

We recently presented some of the new preclinical data at the Wound Healing Society annual meeting. Showing the potential of MRG-110 to accelerate wound healing in healthy skin and in pathological condition.

In summary, we believe we made important progress advancing our pipeline of microRNA therapeutic candidate, while continuing to maintain organizational focus on our lead program. We look forward to advancing both cobomarsen and MRG-201 into Phase 2 clinical trial later this year and further advancing our other product candidate during 2018.

We are excited about the opportunities that RNA focus discovery and development platform could provide. We believe that RNA target therapies represents an important therapeutic approach in a variety of medical conditions with unmet need and miRagen is well positioned to be a leader in the field on several fronts.

With that, I will now turn the call over to Jason Leverone, our Chief Financial Officer, to review the financial results we have reported earlier today.

Jason?.

Thank you Bill. And good afternoon everyone. I appreciate the opportunity to provide our first quarter 2018 financial results.

As Bill mentioned we had a strong to the year, we made important advances in the development of MRG-110 with our partner Servier which led to miRagen earnings its first development milestone under our agreement with Servier during the quarter.

Servier has been an important partner to miRagen for a number of years and we look forward to developing MRG-110 now our third clinical stage product candidate, together with Servier.

Turning to our first quarter 2018 financials, we ended the quarter with approximately $78.1 million in cash and cash equivalents, including the $37.9 million in net proceeds from our public offering in February. Net cash used in operations for the quarter, was $7.4 million compared to $8.2 million for the first quarter of 2017.

Based on our current cash position, we believe our current resources will be sufficient to fund our operations into early 2020. Turning to the income statement, we recognized $4.8 million in total revenue during the quarter. This compared to $0.5 million during the first quarter of 2017.

The increase in revenue was due primarily to the $3.7 million milestone we earned and recognized as revenue under our agreement with Servier. We also recognized additional revenue related to an increase in research and development activity, reimbursable to us by Servier as we advance MRG-110 into clinical development during the quarter.

Overall, research and development expenses for the quarter increase to $6.4 million this compared to $4.1 million for the first quarter of 2017. The increase in R&D expenses was due primarily to higher personnel cost as we added to our research and development team over the last 12 months.

We also incurred higher technology license fees and clinical development expenses to support our now three clinical stage programs. General and administrative expenses were at $3.0 million for the quarter, this compared to $3.3 million for the first quarter of 2017.

The decrease in G&A expenses was due primarily to lower professional fees in 2018, when compared to higher merger related charges we incurred during the first quarter of 2017. This decrease was partially offset by higher personnel related costs as we expanded our general and administrative team over the last 12 months.

With that, I'll ask the operator to open the call for questions.

Operator?.

Thank you. [Operator Instructions] And we'll go first to Jonathan Miller at Evercore ISI..

Hi guys. Thanks for taking the question. I want to ask a little bit about MRG-201 in the fibrosis trial that you're planning on starting.

For the keloid patients, there has been some confusion over the past few years over how many patients there are exactly, where they are? What can you tell us about your expectation for enrollment there how many centers you'll need and what the timeline is on that trial? Secondly for MRG-201.

In the ocular fibrosis setting, how do you expect the formulation to be different than how should we think about the effects of that formulation difference on the development that way..

Great. Hey Jonathan, thanks for the questions. So on the first front, in the Phase II trial in keloid we spent a lot of time in conversations with key opinion leaders and folks that treat patients with Keloids. We've been able to identify several centers around the country that have databases with the relatively large numbers of patients in them.

And we will initiate those sites in the recruitment of the patients. For the study we've designed a very powerful study where we are looking at inter-patient control analysis of biopsies in keloid formers.

So because we can do powering on and sort of usual work on pretreatment post-treatment analysis and do it within patient where we're comparing placebo versus treatment we are able to do trial and power it sufficiently to lead to the outcomes we desire to see with the relatively small number of patients.

And we feel at this point with the centers that we have onboard that we are well positioned to be able to recruit this without a problem.

In terms of the second question on ocular fibrosis, what we reported at RVO and what we've been looking at rather extensively is the effects of MRG-201 on fibrosis that occurs both in the retina as well as the cornea.

And to that end we've explored both intravitreal injection focused in the treatment of retinal conditions and subconjunctival in the treatment of cornea conditions. And what we have done to date is the preclinical studies evolved in fairly simple formulations of MRG-201 again with the intravitreal administration and subconjuctival administration.

As I mentioned we do see good biodistribution, biomarker changes that are consistent with this potentially having a clinical benefit. The third thing that we've looked at was conditions where there is some sort of a break in the barrier to the eye.

So in the conditions we were looking at this was an out borne [ph] burn which them compromises the impact eye. And in that case what we are able to show quite nicely is that even topical administration the compound leads to distribution and activity of the compound in the eye.

So in that case if we can identify certain indications where we would have a commensurate either alternations or cut in the eye or traumatic damage to the eye, we would be working towards looking at some formulations that may increase the viscosity or wettability of the compound to keep it in place in the eye.

But no sort of major differences in formulation, obviously dose and dose form and concentration would likely be different in applications in the eye versus other potential applications for MRG-201..

Great. Thanks. For the preclinical programs, sticking with the eye and progressive blindness there. How do you view the overlap with other competitor programs that are also looking at modulations of progressive blindness disorders.

How broad is the application of this mirror family?.

So thanks Jonathan it's a great question. So one of the things that really attracted us to this particular set of microRNAs is that there is a lot of genetic correlations here. So from both animal models where deletion of certain microRNAs will lead to certain phenotypes, both in hearing loss and vision loss.

There are also examples in human genetics that point us in this direction and as opposed to approaches that are really looking at either very specific mutations in particular genes that are inducers of issues in vision loss.

What we believe with this is the power of microRNA biology in modulating a pathway that's downstream and important in the maintenance and generation of a regime of genes, important in vision loss will likely give us broader application of this particular set of microRNAs than some of the other approaches and we're also interested in the potential of complementarity between these approaches to really lead to greater benefits to patients..

Great thanks very much..

Thank you..

And we'll go next to Liana Moussatos with WedBush..

Thank you for taking my question. Congratulations on all your progress. In the second half you mentioned having data from one of the - at least one of the expansion indications.

What do you hope to see there in order to move forward in at least one and which indication do you think is most likely to be the one that you report on?.

Thanks Liana. Great questions again and what we anticipate is again reporting out. We've treated patients with each of the conditions. What we've been focused on in addition to continuing to build our safety database the patients in these expansion indications are being treated with 600 milligram IV infusions.

So we will obviously build on the safety database in additional patient populations.

At the same time we're looking for alterations in blood counts, we're looking for alterations in lymph, we're looking for looking for general changes in sort of the progression of the natural history of the disease based on the patient's history and based on these observations the treatments the patients that we're treating now are certainly in disease that are more progressive so being able to then see alterations in the trajectory of the disease progression are going to be interesting observations as we move forward.

In terms of stratifying the indications we have been - we selected these three indications based on sort of the probabilities that microRNA-155 would be elevated. There is good reason to believe that in any of the indications there will likely be 155 elevation.

We've done preclinical studies in any of those diseases as well and shown promising preclinical data that supports the advancement. We're intrigued by the opportunity in ATLL because we've studied this is a lymphoma leukemia that's induced by HTLV1 and this is a human lymph tropic virus.

That virus actually cooax the native microRNA-155 in newer cells to advance its own life cycle and what happens is the effects on resistant say apoptosis, DNA repair mechanisms in other proliferative signaling leads to significant effects on progression of cancers as well. So we're particularly interested in that.

It's a very difficult to treat condition. There really are no good treatments available for it.

But we are intensively focused on all three of the applications and like I said in the second half we'll begin to report results that would be relevant in terms of altering blood cell profiles, lymph characteristics and looking at this relative to the natural history of the disease..

Thank you..

Thank you..

[Operator Instructions] And we'll go next to Yun Wang at Jefferies..

Hi, this is Suzy calling in for Yun. Thanks for taking my questions, then I question about cobomarsen.

Just wondering if the accelerated approval would be based on just on ORR4 or any other endpoints? And my other question is for 183 cluster is there something that you have to get chronically or is it just one time treatment?.

Thanks Suzy, great questions. So on the cobomarsen front, the primary endpoint will be ORR4.

Based on our discussions with the FDA and our recent meeting, we received guidance from them that ORR4 on its own as a primary endpoint, obviously based on review of the results by the agency could results in accelerated approval based on that primary endpoint alone. We will also be looking at additional endpoints.

Another key secondary endpoint will be progression free survival.

And the advice that we've gotten to-date has done that the primary endpoint alone could result in the accelerated approval, if the progression free survivals, the endpoint would read out at the time as ORR4, we believe that would then could then result in full approval out an accelerated basis, and we'll also be looking at patient reported outcomes.

These will be sort of tools that the patient reports out on itching and the pain in the tumors. And those are both important secondary and potential exploratory endpoints as we continue to move the compound forward.

Yesterday, we released the data on the first analysis from the Phase 1 that does show a correlation between reductions in Skindex-29 which is patient reported outcome of pruritus and pain and reductions in tumor severity and M slot scores.

So the primary endpoint allowing for accelerated approval will be ORR4, secondary endpoint are PFS, progression free survival would then allow for full approval. And then 183, so I would say that at this point, we don't have a sufficient amount of data to understand if this could potentially be sort of a single administration regime.

The data that we have so far has been in a model system where you really do have a limited amount of time. These are mutations in a receptor in a model systems. The animals are weaned in the dark when they move into the light. There is a rapid deterioration in vision. So what we saw was with dosing we're able to blunt the reduction in visual acuity.

We're going to conduct some additional experiments. But based on the distribution and the eye it turns out is a pretty interesting compartment for nucleic acid therapeutics because there is a relatively low nucleus content which is one mechanism by which they get degraded.

The residence time and the amount of [indiscernible] that we see in the eye has a pretty long duration. So if multiple injections would be necessary, we believe that that could occur on a relatively infrequent basis. But today I can't really following answer the question of whether or not an acute treatment would be sufficient..

Thank you..

Thank you..

We'll go next to Madhu Kumar at B Reilly FBR..

Hello, hi guys, thanks for taking my question. So really two sets of questions.

First one relates to SOLAR trial, could you remind us what would be the comparator drug and basically how that does on metrics like ORR4 and progression free survival? And then secondly thinking about Cobomarsen in the Phase 1 trial in CTCL and [indiscernible] how does it perform in terms of when you get the start-up therapeutic response, and how does that kind of response kinetics inform the timing of the SOLAR trial, how long do we treat [ph] and you expect to see ORR4 to occur?.

Great. Thanks Madhu. Great questions as usual. So the - so let me address the SOLAR trial and the comparator agents. So what we discussed with the FDA was three potential comparator agents methotrexate, [indiscernible]. We really raised these three as the active controls based on the fact that they have all recently been analyzed in control studies.

What we know from the reports in those controlled studies for [indiscernible] and methotrexate the reported outcomes there were objective ORR4 of somewhere between 12% and 14% I believe 12% to 15%. In the case of [indiscernible] there wasn't an ORR4 reported.

It was reported as ORR, so not with the four month durability provision on it, the observations there were about a 7% ORR. And if we think about the secondary endpoint, progression-free survival, the observation from previous clinical trials were that [indiscernible] and methotrexate were about 3.4 months and [indiscernible] was about 3.1 month.

So we really modeled the powering of the SOLAR trial based on those three potential agents. We haven't guided yet on selection but we will do so as soon as we are rezoning in on what the comparator will be but it will be one of those three and it will be a single agent controlled comparison based on guidance from the FDA.

The second question around the cobomarsen Phase 1 and sort of the response kinetics, so, we have seen responses that onset as soon as 17 days after the initiation of treatment. We have seen other responses that don't really fully appear to gain traction until later in the course of treatment there.

The best correlation that we can draw is between the severity of the mSWAT score at the initiation of treatment has a factor on the length of time on product candidate prior to seeing responsiveness. But we tend to see responses over the course of several months.

The longer that we keep patients on drug, we see a deepening response generally I would say. This really has led us in the SOLAR trial to stipulate a specific intend to treat time which would be six months and we would than file the - the patients over this period of time.

As you know ORR4's definition is that as we monitor the patients if they are able to achieve a 50% reduction in the skin, the mSWAT score once they achieve that we start to clock, and as long as they can maintain that response well on drug for four month, than that response come to the positive in the analysis.

So there is a fair amount of heterogeneity in the time to response and like I said it really appears to be correlated with severity of mSWAT score for initiation of treatment..

Great, thanks very much..

Thanks Madhu..

It looks like we have no additional questions at this time, Dr. Marshall, I'll turn things back over to you sir..

Great, well thank you very much for your time today and for continuing to track our progress. We are proud of our achievements thus far this year and we will continue to work diligently towards outcomes that could benefit patients in the future.

We look forward to keeping you informed as we advance our pipeline in the year ahead and please feel free to reach out to us, if you have any additional questions. Thank you and have a great afternoon..

And ladies and gentlemen, once again that does conclude today's conference. Again, I'd like to thank everyone for joining us today..