Q2 2020 miRagen Therapeutics Inc Earnings Call

[music].

Greetings and welcome to the marriage and therapeutic Q2 2020 earnings conference call. At this time, all participants are to listen only mode. A question and answer session will follow the formal presentation. If anyone should require operator systems. During this conference. Please press star zero on your telephone keypad. As a reminder, this conference is being recorded I will now.

The conference over to Dan three of life Science fighters. Thank you you may begin.

Thank you operator, good afternoon, everyone and welcome to our second quarter 2020 conference call.

Today after the market close we issued a press release, providing our second quarter financial results and business updates.

A replay of today's call will be available on the Investor section.

Our website approximately one hour after its completion.

After our prepared remarks, we will open up the call forgive me.

Before we begin I would like to remind everyone. At 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 today, except as required by law that.

We disclaim any obligation to update or revise these forward looking statements in light of new information for future if that's.

Factors that could cause actual results or outcomes to differ materially from those expressed in.

Implied by such forward looking statements are discussed in greater detail and our most recent filings on form 10-K.

And our other periodic reports on forms 10-Q, an 8-K filed with the FCC.

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

Thank you Dan Good afternoon, everyone and thank you for joining us for our corporate update call for the second quarter 2020.

I'm joined today by the rest of our leadership team Deanna Escobar, our Chief Medical Officer, Jason Leverone, Our Chief Financial Officer, and Lee Roush, Our Chief operating officer.

First I would like to express my gratitude and appreciation to the whole marriage and team for all that they're doing to help advance Mike or any targeted therapies as a potential new class of medicines.

Maridjan, we believe in the potential of therapeutic micro targeting and in our ability to develop potentially transformative medicines for patients in need across a wide range of diseases.

Before I hand, the call over to Deanna to provide a review of our programs I would like to take this opportunity to talk about marriage and progress and our expectations going forward.

I'm pleased with our business performance on multiple fronts and encouraged by the continued commitment of our team to deliver results. During these challenging times.

As we entered the second half of the year. We believe we're on track to achieve many of our 2020 goals, which will help position the company for future growth and continued innovation.

During the first half of 2020, we release clinical data for cobalt marson in adult T cell leukemia lymphoma, or 80, Ll, which we believe supports the continued development in this indication.

We are scheduled to meet with the FDA to present, our data and discuss the development path for cobalt Marson in 80, El al and expect to receive their guidance before the ended the year.

We also recently announced that Kobo Marson received orphan drug designation from the FDA for the treatment of T cell lymphoma, which includes 80 Ll and CTCL.

Further to our progress with color marson. Despite the challenges that koeppen 19 has caused to many clinical trials, we continue to treat and evaluate a majority of that CTCL patients in our phase two solar trial.

Finally in the second quarter. We also released preclinical data for our next generation targeted Mir 29, mimic MRG to two nine.

Which is our product candidate for the potential treatment of pulmonary fibrosis.

We have additional preclinical studies underway and anticipate announcing new data from this program before the end of the year.

Before I turn the call over I'm pleased to welcome Lee Roush to Maridjan as Chief operating officer.

We joined US in June.

And brings deep experience across the business landscape in the biotech industry.

We're excited to welcome lead to the marriage and family and are enthusiastic about her future contributions as we continue to build marriage and as a leader in micro targeting medicines.

Now, let me turn the call over to our Chief Medical Officer, Deanna Escobar to discuss our lead programs.

Thanks, Bill It is my pleasure to join the call today.

First let me start with couple of Marson.

As a reminder, we enrolled 37 patients in our phase two study of couple of Marson called Sawlog.

Glenn to assess the rate of an objective response in this game that is durable for four months define us a 50% upgrading equipment in the severity of the patient the skin disease over the entire body as measured by the end Swat score.

In our last quarterly update we had mentioned that we had seen an impact on clinical activities at some sites, where this solid trial is being conducted as a result of the covet 19 pandemic.

Were pleased to report today, the majority of the patients into solid trial have continued to receive uninterrupted treatment and have undulation clinical response and the impact does covet 19 on this trial to date has been limited to a small number of patients.

As of July 17, 2020.

34.

37, CTCL patients are being followed for response on 32 have continued to received and interrupted treatment with couple of Martin.

Although the impact has been limited to date the covered Nitin pandemic is ongoing and will likely continue to provide challenges due us our clinicians and patients in the trial.

As we prioritize safety. It is important to note that the collection of patient data for the assessment of the primary endpoint in this trial cannot be performed remotely.

Emberson patient visits a clinical trial sites are required to evaluate each patient's m. sought an objective response for four consecutive months.

Consequently, we will continue to assess the impact on patient dosing and this is monitoring that may require additional time on drug to allow for that more complete assessment of couple of Marcellus activity.

We look forward to providing additional updates on the status of the solid trial later this year.

As part of our brought our strategy to us at the potential of couple Marston to treat Meanwhile, 55 elevated blood cancers.

We're evaluating couple more sending their first in human phase one expansion indication trial in patients with 80 allow.

Earlier in the year, we announced positive data from this trial is specifically in the subset of 18 patients with with Seadrill disease after chemotherapy treatments.

This included the observation that Google Marson prolong. This is a steadily station a median survival time in patients with aggressive 80, Ll that has per system to seadrill deceased up to chemotherapy and other therapies.

Did you see stabilization in these patients was marked by a decreasing biomarkers of tumor cells activation and from your operation providing evidence of the biological mechanism of couple of marson.

We believe that the overall survival biomarker and safety data, we observed for Colo Marson. In this trial provides the basis for a continued development in patients with aggressive ATM.

Especially considering that historically this patients have shown a very poor prognosis.

The next step for this program is to finalize development plan for a couple of Martin in ATM now based on discussions with the FDA.

During the second quarter of 2020, we requested a meeting with FDA to disgusted development path for couple of Marson in 80 allow and what currently preparing for a meeting to take place before the end up 22 any.

Turning into play process.

Well develop in multiple micro and they may make compounds that replaced micro are in a 29, which is found an abnormally low levels in a number of pathologic I'll talk about it conditions.

Micro and they 29 is believed to play an important role in the regulation of numerous processes that contribute to fight process. We believe that increasing the levels of micro and 829 would that were proprietary micro in a mimics could provide benefits to patients with pathological deposition connective tissue in an organ or tissue.

We are currently developing two distinct Mike or in a 29 mimics rim Larson and MRG to nine for the treatment of various forms of pathological type process.

Today, I will focus on MRG to nine and the data we announced earlier this quarter.

As a reminder, MRG to nine is being developed a systemic antibiotic for potential treatment of patients with idiopathic pulmonary fibrosis or yes.

We believe that the efficacy and safety profiles of NRG to nine positions it asset potential differentiated approach for IP, yes.

This program is supported in part by a grant in collaboration with the NIH and Yale University.

In June we announced additional preclinical abroad, and safety and in vitro human efficacy data for MRG to Twonine would suggest that Mir 29 replacement may represent a novel part of the time in the treatment of IP, yes.

These data were discussed by our director of research breast among memory Onek Halo alcohol held on June 23 2020.

In summary of what mentioned in their patient targeted mid 29 mimics demonstrated potent targets pathway down regulation in normal human lung fibroblast in vitro.

The next regulated college and secretion Invepar logica lung fibrosis and showed an anti fibrotic effect in precision cut lung slices.

As we move from those interest that is an ex vivo studies, we've seen numerous times that Mir 29 blogs fibrosis in blue Mycin, India's pulmonary fibrosis with increased potency of a red Larson.

And can be doses cynically by either intravenous our subcutaneous administration.

In addition, toxicology studies have shown an adverse events on Oregon histology.

Because either in this data we have already advance MRG to nine into a non human primate toxicology study and expect to report the additional preclinical safety and efficacy data before the end of 2020.

I want to say that we are grateful for their continued support that Yale and the NIH.

As you can probably tell by our cold in June and by our comments today, we're excited by this data.

The plan advancement into non human Primate studies.

I want to think again, our cable else on the June call, which included Duck does have a random emptiness of well Cornell Medicine Teresa Barnes from the coalition for pulmonary fibrosis, and Dr. necessarily kaminski of the yellow school of medicine.

Each Gail brought a unique perspective to the call an offer insights into why are work an idea is so important.

We're also evaluating red Larsen in continuous fibrosis and local antibiotic indications, we're encouraged by our progress today and plan to provide additional updates during future calls.

In summary, our pipeline continues to advance and we see opportunities for each of our programs with important milestones set for the second half of the year.

And while the Carbonite team pandemic has created some uncertainty in our ability to accurately guide on the timing of certain milestones we remain confident in our ability to deliver important results from this program.

With that I will now turn the call over to Jason lever Rodney our Chief Financial Officer to provide a review of our financial results Jason.

Thank you Deanna good afternoon, everyone. Today's press release, we reported our second quarter 2020 financial results.

We ended the quarter with 30.6 million in cash and cash equivalents.

This compared to 26.8 million of cash cash equivalents in short term investments at the end of last year.

Net cash used in operating activities was 6.8 million for the quarter, which was down from 8.5 million last quarter and $7.2 million during the second quarter of last year.

We believe that our current cash cash equivalents will be sufficient to fund the company's operations.

Third quarter of 2021.

Revenue was 0.2 million for the quarter compared to 2.5 million for the second quarter of 2019.

The decrease is primarily due to a decrease in contract revenue attributable to a prior license and collaboration agreement.

Research and development expenses were 3.8 million for the quarter this compared to 8.6 million for the second quarter last year.

Significant decrease in R&D expenses was expected and primarily due to decreases in clinical and manufacturing activities associated with the solar trial as well as lower personnel related costs during the quarter.

General and administrative expenses were 2.7 million for the quarter compared to 2.9 million for the second quarter of last year. The decrease in January expenses was due primarily to decrease personnel related cost, which were partially offset by increases in legal costs during the quarter.

Finally, our net loss was 6.4 million or 12 cents per share for the quarter. This compared to $8.9 million were 29 cents per share for the second quarter of last year.

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

At this time it will be conducting a question and answer his question. If you would like to ask a question. Please press star one on your telephone keypad confirmation tone would indicate your line is in the question Q you May Prestart too if you would like to remove your question from the Q for participants using speaker equipment. It may be necessary to pick up the handset before pressing star Keith one.

Well, we pull for questions.

Yeah.

Our first question is from Jonathan Miller with Evercore ISI.

Hi, guys. Thanks for taking my question I have couple here I guess first it seems like the HTML meeting with the FDA has maybe slipped a little bit again and I wonder if that's an actual delay or are you just still waiting for from guidance on timing from the agency.

Secondly, I guess given that the majority of solar patients have been able to get on interrupted treatment.

You have any idea when we might be able to see data I know, we were expecting pretty good we're expecting it this quarter. Obviously, that's been interrupted do you have a sense of how many patients have had interrupted data collection and.

What that means for overall data timing there and then lastly, oh, the mobile partner ready program that you expressed an interest in.

Developing oh license relates to brick collaboration on.

Can you say, which you think has gotten the most interest from the rest of the industry.

Yes. Thanks, John appreciate the questions today, so for I'm HTL all front, what Weve indicated is that we do have a meeting scheduled with the agency.

We're not guiding on the exact timing of that meeting and the other kind of factors that we're taking into account are really just the anticipated time to receive the feedback and really digested and then be able to discuss it in greater detail. So we're simply kind of guiding it towards.

The up before the end of the year. However, our last guidance on this was we anticipated.

Getting the meetings scheduled and having it occur. So we are on track for what we expected.

On the solar front.

Good again, we have about 32 patients that have received on interrupted doses. There are 34 of the 37 that we originally put in this study remain on the study. The you know the factors at play here. There is the continuity of the dosing occurring and the other one is the.

Ability to measure the I'm swap scores so what we want to do obviously for this interim analysis is really.

Select the highest quality information from.

The data that we're getting from these patients and there is.

Based on either some.

The patients are affected by a break in dosing or because of the.

Inability to have contiguous.

Swap measurements, we are simply monitoring this at this point.

And we'll provide further guidance as soon as possible on when we anticipated.

Overall I would say you know we we originally were quite concerned about the.

The effects of this we're happy to report that there are some effects, but again our goal here is really to ensure that we have the sort of optimal level of quality data that can come out of this study or the interim analysis that that provides us the ability for some clear decision making.

On the last front in terms of the.

The partner ball and sort of a outreach in terms of programs, we've had discussions around a variety of different programs in the area.

Our most recent discussions and Youll see our not discussions our most recent results that we've been working on publishing it around the MRG 110 program. This is a program indicated in new vessel growth and particularly in this setting a cardiovascular disease, we have ongoing discussions.

In that regard we have also some discussions ongoing in the area of of Rem Larson in the area of Paul It's more local one topical administration, whether that would be dermal scarring or okcular fibrosis and finally, we have had some discussions ongoing even in pipeline programs. So soon.

Earlier stage programs, where were we will continue to work on those and provide further guidance as we've got more solid.

Direction on those programs, but there are multiple different assets that we're having discussions on at this point.

Great. Thanks, very much I guess, one final one if I can sneak it in there I know, we're expecting more preclinical data coming from the two to nine the Nexgen 20 or 29 minutes program. This year I was wondering if you had at this point anymore guidance on timeline to do I, Andy enabling studies being complete.

You did.

And being able to move forward into the class.

Yes, John Thats, a great question. The the the outcomes of this there is an ongoing non human Primate study and we also have a couple of kind of smaller dose ranging and.

And.

Sort of a frequency of dosing studies that are ongoing those are really the final determinants of sort of clinical candidate designation and then once we have these all the data back from that we can make some decision we will have a clearer ability to guide on when we would anticipate high indeed, ambling studies and an eventual.

Hi, Andy.

Great. Thank you very much.

Thanks, Jeff.

Our next question comes from Liana Moussatos from Wedbush Securities. Please go ahead.

Good afternoon. This is andres for Leon.

[music].

I wanted to get your thoughts on.

I'm good regarding couple merson near communications as you're going to have with the FDA.

Those discussions.

Any.

Around any potential indication expansions into the other T cell leukemias or b cell malignancies.

You go get any thoughts on that thanks, Hey, sure. Thanks, San Andreas the the discussions are really focused on the development pathway for.

Kobo marson in adult T cell leukemia and lymphoma at this point the the focus really being a.

Type C meeting to understand the.

The uptake in guidance from the FDA on what will be required for additional clinical analysis of Kobo marson.

It from a broader perspective, we obviously are.

Great.

Intensely focused on additional opportunities for Kobo Marson, obviously CTCL as one of them. We've also reported previously some interesting data and Dl Bcl, but we're really taking this in a stepwise approach understand the the path towards potential approvals in different.

A potential indications, but our broader goal is really to seek a.

Broader indications and Mir 155 elevated.

Blood cancers. This is one step along the way, but this meeting is really focus on 80 Ll itself.

Okay. Thanks, I'll step back and thank you.

Our next question comes from travel already with Oppenheimer. Please go ahead.

Hey, Thanks for taking my question.

Can you remind us the mood.

Both the solar and.

Typically.

In calculating so.

Thanks.

Thanks, Trevor so the solar trial is really.

Very much a global trial, so the total number sites sort of beyond.

I don't really have.

Im off the top of my head, but we do have sites both in Europe in the United States.

And we have seen affects at sites in both of those.

Geographies and principally it has been.

Effects on the centers either re.

Orienting resources in the and the fight against covered or in sort of restricting principally the ability to monitored the endpoints in the studies. So theres a variety of kind of different effects that are going on we've been quite pleased to actually have the.

The patients be able to access dosing and we had previously set up the the in home infusion service to be able to continue to do that so there are a variety of factors at play again with the sort of.

Kind of alterations in the.

Kind of surge in coal, but even within the United States and just different geographies. There are just different local geographies that I've had different effects, but overall, we've been pleased with with progress on the on the study.

Got it thanks.

Thank you.

Hey, once again, if you would like to press to ask a question. Please press star one on your telephone keypad.

One moment when we pull for questions.

Okay. We have reached the end of our question and answer session I would like to turn the call back over to Bill Marshall for closing remarks.

Great we want to thank everyone for taking the time. This afternoon for an update on marriage and I also want to think.

Our employees again, our collaborators and our advisors for their dedication to our mission as we work to bring transformative new medicines to patients lastly, I want to acknowledge the healthcare workers and hospitals across the country and the other frontline workers for their selfless contributions in the ongoing battle with cobot nine.

Team.

Everyone. Please stay safe, thank you and goodbye.

This concludes today's conference. Thank you for your participation you may disconnect your lines at this time.

Okay.