Q2 2021 Dicerna Pharmaceuticals Inc Earnings Call

August 9, 2021

[music].

Good morning, ladies and gentlemen, and welcome to the base rent of Pharmaceuticals second quarter 2021 earnings Conference call. As a reminder, this conference is being recorded at the company's request I will now turn the call over to your house attorneys Sobek upstream IR. Please go ahead.

Thank you operator, good morning, everyone and thank you for joining us to review dikes, there on a second quarter 2021 financial results and operational highlights.

For anyone who hasn't yet had a chance to review our results we issued a press release earlier. This morning, which is available under the investors and media tab on our website I'd like to turn on Dot com.

You May also listen to this conference call via webcast on our website, which will be archived beginning approximately 2 hours. After this call is completed.

Speaking on today's call will be debt service, President and Chief Executive Officer, Doug Fambrough, who will review our program portfolio and strategy and our Chief Financial Officer, Doug Pagan, who will review our second quarter financials.

We also have Sriram Rodriguez, our Chief Medical Officer, Jim Weissman, Our Chief operating Officer, Rob <unk>, Our Chief commercial officer, and Bob Brown, Our Chief Scientific officer available today to address questions during the Q&A session.

Following our remarks, we will open the line for your questions.

I'd like to remind listeners that management may make forward looking statements on today's call pertaining to the company's finances business and operations, including the discovery development and commercialization of our product candidates and technology platform and the therapeutic potential thereof, the success of our collaboration and any potential future collaborations.

Such forward looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements.

Applicable risks and uncertainties include those relating to our preclinical research and clinical program and other risks identified under the heading risk factors included in our most recent forms 10-Q and form 10-K, while we may elect to update these forward looking statements at some point in the future. We specifically disclaim any obligation to do so if our views.

Now I'd like to turn the call over to Doug Fambrough Zone, as President and CEO Doug.

Thanks, Tony Good morning, everyone.

Since we just did our Fi Ax 2 day to call on Thursday, I'd like to start today at a high strategic level before discussing particular programs.

Arnie I is a powerful modality 24 years since its academic discovery encompassed a long period of technology maturation, leading to the breakthrough of gallon that mediated delivery of highly modified RNA Duplexes, which has led to multiple approved products with more product candidates at N D. A.

Age.

Last Thursday afternoon day sort of added our first to this list as our lead product candidates. The dozer on demonstrated a strong safety profile and impressive activity profile and the primary hyperoxaluria type 1 or P. H 1 population.

These data we believe sets up a high likelihood of P. H 1 approval in the U S Europe, Japan, and other markets and we intend to submit an NDA to the FDA in Q.

Q4.

The <unk> 2 pivotal data directly demonstrates the strength of our Galaxy RNA technology platform.

I know everyone on the line today is highly sophisticated and can recognize that relative to other approved biotech modalities, such as monoclonal antibodies and AAV gene therapy.

The ability to practice Arnie I on a cutting edge critical mass level is remarkably concentrated in a small number of companies and we're proud to be 1 of them. It.

It was less than 4 years ago that day Sirona was a microcap company trading at a little over $3 per share no collaborations no galaxy clinical data.

In less than $100 million on the balance sheet today, we in Q2 with almost $710 million. The large majority coming from technology monetization with 6 collaboration 6 different companies engaged in moving day, starting on Galaxy molecules forward 6 clinical stage programs, including the dose or in 9 more.

That have entered preclinical development and our platform now expense R&D I delivery to tissues beyond the liver opening a deep well of opportunities to exploit for years to come.

That's a pretty good 4 years.

What really excites me, though is what we can accomplish over the next 4 years in that time, we expect in a dozer, Inc to be approved and available for patients in major markets around the globe, we expect to Blue belt Sessa round for Alpha 1 antitrypsin deficiency associated liver disease or a T. L D.

Where we hold 100% of U S rights and which is now early in phase 2.

We expect to move this deep into pivotal development, we anticipate the RG 6346 program for chronic HBV.

Which is in a phase II combination trial being conducted by Roche will have further progressed in clinical development and completed its 5 phase 2 cohorts and with success. We will have opted in to co fund pivotal development, bringing our U S. Co promotion option, we expect our D. C. R. A D program for alcohol use disorder.

To achieve human proof of concept and has moved to advanced trials potentially breaking open a vast underserved market.

We expect multiple programs from our Novo collaborations who have entered development and we expect to have opted in to 2 of those but only after having seen positive human clinical evidence, which is to say we get to choose the likely winners after seeing some clinical data.

We expect current development programs from Lilly.

AZ slash lexi on.

And boehringer ingelheim to be moving through the clinic, we expect new programs to be entering the clinic on average 1 per quarter over the next 2 years and potentially beyond that at a similar rate amongst those we expect to have taken multiple new galaxy and or Galaxy plus proprietary.

Grams, well into clinical development. Indeed, we initiated early development of 2 galaxy plus non liver wholly owned programs, which will debut when they are closer to clinical entry.

It'll be quite a ride over these next 4 years and we have the capital to execute on this because our current cash runway with expected revenue from existing collaborations runs into 2025.

It's longer than we've previously disclosed I'll return to that in a moment in the context of the dos <unk> and <unk> 2 pivotal trial results.

But first let's come down from the high level to the actual corporate updates for the second quarter as well as subsequent events.

<unk> with the assessor in.

That we are developing for the treatment of a T. L. P last month, we announced interim results from our phase 1 study, which met our objective demonstrating strong dose dependent reductions in serum alpha 1 antitrypsin in healthy volunteers with administration of a single dose of Bell CECI room in this.

Analysis of the 4 completed active treatment dose cohorts.

0.11 point or 3 point O and 6 point.

<unk> <unk> milligrams per kilogram Bell Susser on was found to have an acceptable safety profile and was generally well tolerated 1 more dose cohort 12 milligrams per kilogram is ongoing and our objective is to present the results from all dose cohorts at an upcoming medical conference.

We also began patient dosing in a randomized multi dose double blind placebo controlled phase 2 Estrella trial Bell Susser on in June the Estrella Phase II study includes a 24 week cohort and a 48 week cohort to be conducted in parallel each with up to 27 participants.

You'll have a diagnosis of P is easy tight T.

<unk> deficiency and a T L D.

As the study progresses, we are looking forward to having a better understanding of the cells of belts essar and its potential to treat the underlying cause of a T. L D.

Also last month, we announced the FDA acceptance of our IND for <unk>, our investigational Galaxy arent AI therapeutic candidate for the treatment of alcohol use disorder, we had filed that IND in late June.

And we are now clear to begin phase 1 we plan to initiate a 24 week randomized double blind placebo controlled phase 1 trial this quarter to evaluate the safety and Tolerability pharmacokinetics and pharmacodynamics of single ascending doses of D. C. R.

<unk> in healthy volunteers. The trial will also assess the interaction between D. C. R. <unk> D treatment and alcohol consumption using standardized ethanol interaction assessments, which will provide critical pharmacodynamic data to inform the development strategy for that high potential program.

From a collaboration standpoint, we announced in May the Fda's acceptance of Eli Lilly's IND for Mel wide free 819469 targeting L. P. A 4 cardiovascular disorders L. P. Little a sometimes referred to this is the <unk>.

Clinical stage investigational Galaxy RNA eye candidate from our collaboration with them.

It triggered a $10 million milestone payment to us and pave the way for literally to begin phase 1 dosing of the compound which began in June.

There are also 2 Lilly partnered programs in IND, enabling studies. In addition to the 2 clinical stage programs also Wednesday, Boehringer Ingelheim accepted <unk> <unk>, an investigational galaxy RNA candidate for the treatment of Nash to advance to the develop.

<unk> stage. This candidate acceptance triggered a single digit multimillion dollar preclinical milestone 2 day sirna and preclinical work is underway and earlier this year, we added to the development pipeline. Our second candidate under the Novo collaboration finally, we have 2 programs in our age.

Z slash Alexia on collaborations that are progressing through IND, enabling studies.

Now, let's turn attention back to the dose right.

As you all know we presented top line data from our <unk> 2 pivotal clinical trial last week and the dose or on successfully and decisively hit both the primary and key secondary endpoints, while showing a strong safety profile.

The results were driven by strong oxalate reduction activity in patients with ph, 1 well patients with ph 2 showed inconsistent results.

Sequentially, we see a path to approval in the U S and elsewhere for the ph, 1 indication, but not the th 2 indications and ph..1 we believe that the dose of rent is highly competitive with the currently approved product for ph..1 we are reiterating our guidance of <unk>.

Q4, NDA submission for nodosa in and we'll continue our development program, including the <unk> 7 and <unk> 8 clinical trials for patients with ph, who have compromised renal function for our pediatric respectively.

With a goal of subsequent supplemental NDA submissions to add those populations to the dose are on label.

With respect to ph too we are reviewing all of our data preclinical and clinical and conferring with experts in the ph field to understand. This result, we will continue to monitor the patients with ph to from the <unk> 2 trial, who have all rolled over into our <unk> 3 open label extension study.

Extended observations may or may not provide additional insight in any event, we do not see a near term path to including ph too and our proposed new dose drug label.

In the meantime, we look forward to reading out on <unk> for single dose study in patients with ph type 3 upon review of that data, we will decide how to proceed and ph 3.

Up to now we have articulated the strategy of fully integrating our company using the dosing Ren as the vector and rationale for building out our commercial function in the United States.

That strategy was based on achieving the label with at least ph 1 ph 2.

With both of those indications and data in ph 1 comparable to what was achieved in <unk> 2.

We were projecting the ability to ramp sales to where our commercialization efforts would be cash flow positive in a reasonably short period of time.

Without page 2 on the label, however, ramping our commercial efforts to cash flow positive is more uncertain.

With substantially more capital investment required along the way.

I do not believe that is the right choice for dice sirna anti starting to shareholders.

Consequently, we have decided to seek a commercial collaboration partner or partners to make <unk> available across all major markets, including the U S subject to approvals occur.

Accordingly, we plan to forego investment in commercial infrastructure at this time.

This approach will reduce our planned spend and is the basis for extending our runway guidance from the prior into 'twenty 'twenty 4.2 into 2025.

Importantly, this does not signal any reduction in investment in our pipeline on the contrary, we may choose to increase investment in our pipeline as we fully processed the <unk> 2 data and refine our plans.

Normally at this point I'd be turning over the call to sriram around our Chief Medical officer for additional color on our progress. However, having just had <unk> 2 topline data call last Thursday, and recently, having spoken to many of you about our best belt sensor on the phase 1 data we are instead going to proceed.

To the financial discussion Sri is here with me and is available for questions. After the prepared remarks. So I will now turn the call over to Doug Pagan Chief Financial Officer, Doug.

Thank you Doug I'd like to briefly walk through the key financial results for the second quarter of 2021, and direct you to our press release outlining these results into our quarterly report on form 10-Q, which was filed with the SEC. This morning.

Net loss for the second quarter, 2021 was $40.8 million or.

<unk> <unk> 53 per share compared to $31.8 million or <unk> 43 per share for the same period last year.

Revenue for the second quarter totaled $41.3 million compared to $44 million in the same period last year.

As of June 32021, we had approximately $162 million of current deferred revenue and approximately $268.6 million of non current deferred revenue.

Additionally, we have recorded deferred income related to our sale of royalty rights to royalty pharma $1.1 million of which is current and $178.7 million non current.

R&D expenses for the second quarter totaled $56.1 million compared to $53.4 million per the same period last year the.

The year over year change was primarily driven by increases in facilities and employee related expenses, resulting from the higher R&D head count necessary to support our expanding pipeline and collaboration agreement.

These increases were largely offset by a decrease in external direct R&D expenses.

G&A expenses for the second quarter of 2021 totaled $25.5 million compared to $26 million per the same period last year the.

The year over year change was driven primarily by increased professional services.

We expect G&A expenses to remain comparable with the current period as we cease our plans to build in house commercial operations for the dose range.

Year to date, we have received $74.5 million in milestone fee and reimbursement payments from our collaboration partners, primarily Novo and Roche and continued to guide to receiving 83 point I'm, sorry $83 million for the full year 2021.

These payments represent an important source of proceeds and demonstrate the value. We expect these collaboration program to continue to generate as they mature.

As of June 32021, we had $709.6 million in cash cash equivalents and held to maturity investments.

Compared to $544.9 million as of March 31, 2021.

This cash balance with our revised operating plans and projected proceeds from existing collaborations should provide us runway into 2025.

As Doug mentioned, we will be seeking 1 or more partners for global commercialization of nodosa them.

Economics from any potential array arrangements have not been factored into our stated runway projections.

Our strong balance sheet will allow discern it to continue to invest in advancing our clinical pipeline supporting our collaboration partners and expansion of our platform technologies.

That concludes my review of the financials I would now like to open the call for questions operator.

Ladies and gentlemen, if you have a question at this time. Please press. The Star then the number 1 key on your Touchtone telephone. If your question has been answered or you wish to remove yourself from the queue. Please press the pound key to your first question comes from Jonathan Miller with Evercore ISI. Your line is open.

Inc.

Thanks, So much guidance I guess I'll start by asking what sort of a commercialization partner or partners. You are looking for for ph..1 is this more of a major pharma co co promoter co commercialization sort of a deal or are you looking for contract sales can you give us a little bit more color about what youre looking for on that global commercialization.

And then secondly also on on ph I guess now that you've had the weekend to think it over do you have any hypothesis on ph to failure at this point.

And obviously you are still following those patients, but do you have any clarity for us on when we might expect to hear an update from you on what what happened biologically there and then thirdly I know you mentioned youre waiting for <unk> to make a final call on this.

But assuming fox for and ph D look good.

Would your plan at this point be to include page 3 in the same NBA, ASP, which 1 or would that be a separate submission.

Hey, Jonathan it's <unk> I appreciate the questions I'll tackle the first 1 related to partnerships for in a dose range I think.

Best way to look at it is we're looking at this as an out licensing opportunity not as a contract sales.

Uh huh.

Strategy. So we will be looking at and have continued to speak with both global biopharmaceutical companies as well as significant regional players all of them have greet acumen in the rare disease space, We will continue those conversations.

Provide our updates as soon as we have gone to conclusion of those discussions.

Thanks, Rob.

So.

John.

You asked for clarity on non ph too.

The Peach to result, with kind of a shock to low ph field certainly are interactive with some some kols and there was I think a fair.

Currently broad expectation that there would be activity.

In ph to end.

There isn't at the current time, leading hypothesis for why we didn't see consistent results with a monthly dosing regimen.

Those are on particularly after the parents signal from a single dose.

In patients.

That means its really not possible for me to give guidance on when some clarity maybe provided but I don't see a path in the near term for there to be any clarity and it's going to take some time to understand and I think it's going to trigger some.

Biochemical level work in academia wouldn't we're not going to be doing that here.

With respect to ph 3.

Debates operating assumption should be our NDA will be for ph 1 only.

We will see what the results are from a single dose of <unk>, 3 and assess them, but I think anyone planning or projecting should assume the NDA filing is just the H 1.

Thanks, John.

Thanks, so much guys.

And your next question comes from Manny for Haar with SV building rank your line is open.

Hey, Good morning. This is Rick on the line from Mani Thanks for taking our questions.

So first.

What are some of your expected timeline for getting visibility into establishing commercial partnerships for ph..1 is there any guidance you can provide here and I guess also do you anticipate that timelines would be different based on whether you choose to have a single partner for commercialization or multiple partners here.

Hey, Rick it's Rob in terms of our partnership discussions those are ongoing growth.

I had mentioned both globally and.

With regional players I don't see it as a rate limiting step in terms of making a dose for an available we're continuing first and foremost with filing the NDA and Anda is a key resource document for filings beyond the U S. So think about it this way.

We're on track as guided to Q4 filing of the NDA concurrent to that we're continuing the discussions with both global and regional players.

I would expect us to.

The next couple of quarters be able to come back with.

Clear conclusions in terms of what we're going to be doing within the dosing.

The United States, and as well with a partner in the U S.

Okay got it thanks that's helpful.

But you are also taking a step back.

What does this change in strategy, meaning per commercial commercialization plans for the rest of the wholly owned pipeline.

Just trying to think is there any read through here for the rest of the pipeline is there a certain threshold.

To meet to start building commercial infrastructure or is it more likely that we're going to see commercialization partners.

On the pipeline.

So Rick I don't think Theres any read through for the subsequent programs. The next 1 likely to come down to that stage would be the <unk> T program, which has a focused call points. Similarly.

Which would be further out has a focused call point in addiction medicine, both of which would be appropriate for us to build out a specialty sales force.

When that comes to pass.

The key thing we're looking at in deciding to fully forward integrate and become a commercial company is that we're confident that that investment is going to generate a return and that we're going to be cash flow positive.

I think we set a reasonably short period of time.

And then that will be a source of cash in the company. If we don't have confidence in that then it doesn't make sense to make that investment that will be made on a case by case basis, but the next opportunities could support building out that infrastructure.

Alright, perfect I appreciate the detail here, thanks for taking my questions again.

Your next question comes from Matthew.

Kumar with Goldman Sachs. Your line is open.

Hey, everyone. Thanks for taking our questions. So I wanted to start with Alpha 1 antitrypsin. So can you kind of walk through the peak day planned for choosing belt says, Iran over the on myeloma, ATR and AIA drug it so something.

That's not publicly available about the on <unk> drug that kind of makes you favorite belts as Iran, or how should we think about the kind of election, but true Bill says ran over over that on a drug.

So my view that took into account.

Decision between <unk> element I sirna took into account all of the preclinical and clinical data to date as well as the logistics of working with our own compound versus working with on the islands compound. There are quite a few things that are not publicly available about VL myeloma.

<unk> and our own frankly that weigh on that decision.

And it's not a decision we're going to revisit.

Okay, and then thinking about the kind of extra hepatic space.

Can you give more visibility, but kind of where are you thinking about kind of pursuing.

Previously discussed the CNS space kind of how are you thinking about that now on how are you doing.

Granularly timelines for putting extra hepatic compounds into the clinic.

So I am really pleased that the breadth of delivery to other tissues that we're seeing.

So in the CNS, we are partnered with Lilly and so I think much of the CNS work will be under that collaboration, but we've talked about adipose tissue and muscle on tumor associated immune cells and there are other tissues. We haven't mentioned that we're achieving delivery I do notice that.

Certain other tissues are a focus for other companies, notably muscle would fall into that category and I think overall, there are probably relatively fewer number of opportunities in muscle.

I would like to see di sirna in more of a white space.

<unk> field running room. So you when we revealed our programs I think we will see that we're probably tacking in a slightly different direction than some of the other oligonucleotide companies going outside the liver.

Drawing on the strength of Galaxy plus to get the other tissue types.

The preclinical timelines are now.

The plan the best you can but that Covid is really disrupted preclinical timelines with respect to things like non human primate availability.

And.

We're not going to guide right now on the clinical entry timing.

And we do want to be fairly close to a clinical entry before we talk about what we're doing because we do expect.

People to jump into the programs we're doing so.

Yeah.

What I can say is that we have 2 programs that we have initiated development on with lots of clinical candidates gone into manufacturing all of that planning to preclinical entry is underway and we will give more detail on a couple of quarters probably.

Excellent thanks very much.

And your next question comes from Stephen Willey with Stifel. Your line is open.

Yeah. Good morning, Thanks for taking the questions maybe just a couple of quick clinical ones.

And Australia can can can you talk about.

The extent of fibrosis, but you're allowing patients who will be.

Eligible to enroll that study or are these going to be a threes or fours are you, allowing cirrhotic patients into that study and then.

With respect to AED.

In terms of patient eligibility I mean, obviously this is a healthy volunteer population healthy volunteers I guess do consume alcohol.

Is there any kind of restrict channel or do you have some kind of.

Guardrails around consumption in that study or at least attempt to.

Just for the first phase 1 experience.

Yes.

In the Hyatt <unk> in Australia. Our goal is to enroll the population that gives us a good read on the benefits of knocking down the mutant protein and potential consequent benefits on liver histology. So we are enrolling abroad.

Population with representative fibrosis.

So that's that's ongoing.

<unk> data to make sure that we've designed the study in a manner that gives us a good read on seeing a difference in the size of the study that we have.

We have designed.

Certainly the worst case patients.

The compensated cirrhotic.

On the other part of this trial.

Other than that will be on a broad range.

And AED Steven could you repeat your question on that for me. Please yes, Im just kind of curious so.

The healthy volunteer experience for <unk> is there some kind of eligibility criteria around alcohol consumption in those patients who are going to enroll I'm just kind of curious if theres going to be any kind of.

Any kind of really efficacy signal that could emerge out of that study just based on the fact that youre going to see healthy volunteers consume alcohol during a 24 week period post dose.

Yeah. So I think we expect to do this in a stepwise manner.

And that's the first goal of the phase 1 single ascending doses to establish safety and Tolerability and as Doug said duo very controlled exposure to ethanol as a way of gathering the pharmacodynamic data that confirms for us whether on what we have established the biological stayed in the human of achieving the necessary not only to deliver in what.

In fact that has.

We're going in our guidance, we will of course be for people to avoid alcohol intake my expectation is that over time. The phase 1 study will give us a read on what people's reaction to drinking alcohol outside of the trial might be but coming in in the initial safety single ascending dose our goal is on making sure that the exposure both on the <unk>.

On the controlled setting.

To make sure that we have a clear read on what the PD of the Douglas, but over time and make sure that in the multiple ascending dose we should be able to gather more reads on efficacy.

Yes.

So in essence, we do expect.

2 to learn a lot about the physiological response of.

Someone who has taken the <unk> to the intake of alcohol that is not the approvable endpoint. The approvable endpoint will be likely harm reduction in patients who have <unk>. That's.

Sort of loss control and harm reduction being an assessment of their ability to regain more control over their consumption of alcohol. So on that direct sort of approvable endpoint, we're not going to get anything but there is a belief.

Believed to be very clear linkage between the physiological response and that behavior as seen by the rates of AED and people with naturally occurring a L. D. H 2 mutations non exactly the same thing we're doing but closely related so we should get the important physiological feedback.

And that's something we'll discuss and look forward to talking about it when we see it.

Alright, thanks for taking my questions.

And our next question comes from Yaron Werber with Cowen Your line is open.

Hi, guys. This is Brendan on for you Ron Thanks for taking my questions just to just a quick 1 from US it's really looking at the TD program.

I guess looking at the landscape here, what can you tell us maybe about.

Built on <unk>, specifically, and maybe more so youre kind of goals and expectations for the phase III.

That you think youre going to really be necessary to differentiate the drug I guess my point is really kind of given that there are other competitors a bit ahead of you on development in that space as well, where do you really see the bar.

For both us around there to stand out and maybe what kind of gives you confidence you can meet that thanks.

Hi, Brendan so.

There is 1 other company.

And phase 2 to treat.

L D. So exact.

Exactly a crowded field.

And I'm sure you're aware almost every pharmaceutical market has multiple interest.

But when does think about how the various products are differentiated in this case because of the mechanism of action is the same there isn't a mechanistic differentiation.

In the ph 1 indication.

We have a.

What I think is a very positive differentiation based on the mode of administration with our pre filled syringes that for offer administration by patients with ph 1 at home.

On their own.

Compared to the competitor product with SASSA health care professional involved and is weight based and some variable volume and so not not in pre filled syringes.

We shall see in <unk> as we head into pivotal trials on a competitor heads into pivotal trials.

What those.

Regimens are and so.

How we want to.

Present, the product relative to competitor, but I think the most important dynamic has to do with the fact that most of the diagnosed patients with <unk>.

<unk> liver disease.

Or in fact patients have <unk> lung disease as well because that has a clearer diagnostic path currently and with approved products has had a fair amount of patient finding that's gone over for a long period of time and I think there is a natural pairing between augmentation.

As a therapy for the lung and RNA I as a therapy for the liver and you can see that the competitor product as 1 of the collaboration with an augmentation provider. It is as it happens on minor interest in the augmentation market and so I think that presents a fairly obvious.

Counter sort of strategy for us with respect to pairing our RNA eye compound with augmentation that we intend to pursue there isn't any particular.

Time per.

Pressure.

To do that.

Right.

There I think that solution.

<unk> solution.

Providing associated with lung and liver.

Is going to be very interesting and it'll be too.

<unk> advantage to be with the market leader.

As opposed to with a more minor player.

Alright, great. Thanks very much.

Your next question comes from Luca <unk> with RPC, Inc.

RBC capital.

Your line is open.

Oh.

On the allocation from RBC capital market.

Just a few questions here, maybe the first on net mid dose you ran if I recall it correctly, probably guidance estimated peak revenue to your peak sales I should say between $500 billion. I'm wondering if you can comment on how youre thinking about peak market opportunity here now that you've seen the phase III.

And then maybe on business development wondering if there is any appetite to broaden the scope of your extra padding delivery via BD.

Recent deal between on your honest and bicycle and on myeloma Pepsi Dream. So wondering if this is something that you're also considering and then finally, maybe on day..1 wondering if you can comment on the timeline tier 4 on seeing the actual data in patients here. Thanks, so much.

Hi.

Sure.

Trying to right.

The questions. There I think that those are in the sales potential was the first 1 so I'm not going to get into detailed numbers here, but we had ph too in our forecast as being a significant minority.

Of the total sales.

Less than half, but significant amount for ph 3 relatively small fraction.

But th 2 was as a significant minority does taking an off label or not non of getting it is included in the label has a significant effect, but I'm not going to get more specific than that on our prior forecast, which obviously is no longer applicable given the H twos.

No longer included.

Shifting to business development around delivery.

That is something that we are considering and we've had conversations with players in the field.

Having said that.

We are very pleased with the delivery, we're getting with our in house technology, which as I mentioned is getting us to a pretty broad array of tissues. So we're not feeling a particular.

Need at this point.

2 formal collaboration with someone who can help us get to other tissues. We are already in a situation where the number of opportunities. We're considering far outstrips the number of programs that we can plausibly prosecute.

No.

It's definitely in the mix and there is some really interesting approaches out there.

But the strategy that we have taken in our research labs has been paying off and we're really pleased with our in house work.

Terrific and maybe timing of the <unk> data for from Australia is there any kind of.

Our guidance that we should think about it.

Alright, I forgot about that 1 sorry on it as quickly as is free.

You can do the math I mean, we are enrolling a study that has 2 cohorts with paired biopsies that happened at 24 weeks and 48 weeks with up to 27 subjects, we haven't fully decided on the timing of a potential interim analysis.

Study started enrolling earlier this year.

Getting some sites on board on Europe. So I think it'll be sometime we will need to have a decent number of subjects, having had the right number.

Paired biopsies to be able to meaningfully present information that isn't applicable and from which we can draw some conclusions.

Above the guidance I can give you right now.

Got it Super helpful guys. Thanks, so much.

Your next question comes from Yigal <unk> with Citigroup. Your line is open.

Hi, Doug. Thank you very much for taking the questions I just had a high level question.

At this point do you believe you have an optimal number of pharma partnerships are you willing to entertain additional partnership to further extend the footprint for the Galaxy technology platform.

Ladders. The case are there certain disease areas that you believe would be better suited for partnering versus developing on your own.

Sure Yigal happy to take this 1.

We've got a really nice set of collaborations and.

They're good partners, who work with.

Is there is a logistical challenge and adding more names to that list.

On our ability to meet clinical candidates definitely outstrips, our development capabilities and I think that's true for the foreseeable future.

And with an excess of opportunities and candidates.

To me and I think to the whole team here. It makes sense to have someone else take things forward are not going to take forward.

So there is still.

I think rationale in our strategy to enter into collaborations on opportunities that we don't intend to pursue on our own or capabilities to pursue opportunities are a lot broader than they were in 2018, when we did our lexi on it really feels.

And our pressure to bring in cash which was fairly strong at that period of time now is we are.

We have a lot of luxury of choosing our spots and so our.

Posture is a little different with respect to collaborations now it is to really spend our time figuring out what we're going to do.

And then later, we'll take the things that we're not going to do.

And we would like to find a path forward for those <unk>.

Some of the spaces that we are working and we've mentioned deliberate adipose tissue, they are clear and metabolic and sort of obesity indications associated with that debt.

Probably are not right for dice sirna.

I would note we are already collaborating with the 2 largest players on the and the metabolic space.

It would be nice.

To perhaps extend those collaborations into galaxy plus that wouldnt burden on the logistics.

Any more but it would.

Satisfy the goal of bringing in capital and making sure those programs move forward. So I see a role for the collaborations but whereas in 2018.2019. It was a primary part of the strategy. It's really a secondary part of strategy now and primary is building out our own pipeline.

And then the collaborations.

Will fit in.

In a secondary role, but it's still.

An important piece I think 1 of the things that all of us would like to do per or what.

We have as our vision statement here at <unk> Sirona is maximize the impact of RNA eye on medicine that means getting a lot of drugs. We can meet candidates can make a lot of candidates it's very modular.

So we want a lot of candidates to move forward I'm really pleased there are 15.

We want that number to keep growing and growing so I hope that gives you. Some perspective that is well short of any sort of guidance, but I think does it is informative to what our mindset is about how we're going to think about collaborations.

Thank you that's very helpful. Thanks, Doug.

And your next question comes from May on Mopani with B Riley Securities. Your line is open.

Hi, Yes. This is William wood on for Mike on Tommy. Thank you again for taking our questions and glad to see you being proactive with your corporate strategy the follow up.

From box to last week. So I was curious about what learnings might be applied from your ex U S licensing process and now that you've made the choice to make global.

Global rights available.

Hey, William it's Rob in terms of kind of some insights on.

Hi, a couple.

High degree of interest in.

And William net interest not only was in new Jersey and beyond the U S. But a number of the parties kept coming back to us and saying what about the U S.

So I think that puts us in a really good spot to continue these conversations both with global and regional players.

Bottom line is the same.

I was going to do.

On wherever we can to most appropriately appropriately put this into the market so that patients have an auction.

Non U S growth globally.

And frankly these conversations.

I feel put us in a good place to.

Work towards that so the key insight is that a number of the players that we've been talking to.

Some are some have had some interest in the U S as well, but we'll be continuing to look specifically across all the different stakeholders and.

Proceed accordingly.

Doug.

Thanks Ross.

Oh go ahead.

I didnt have anything more to add on.

I think we're going to wrap up but at this point operator is there.

Any additional question.

Alright got that.

That concludes our question and answer session I would now like to return the call over to Dr. Tom Farrell for closing remarks.

I want to thank everyone for participating this morning, I want to reiterate that.

<unk> II is an important milestone and I am really pleased with our platform that bodes very well for the future and I look forward to updating the additional galaxy programs as they mature and subsequent calls thanks, everybody Bye bye.

Ladies and gentlemen. This concludes today's conference. Thank you for your participation and have a wonderful day you may all disconnect.

[music].

Good morning, ladies and gentlemen, and welcome to the day is trying to Pharmaceuticals second quarter 2021 earnings Conference call. As a reminder, this conference is being recorded at the company's request I will now turn the call over to your house attorneys Covid I'm sorry IR. Please go ahead.

Thank you operator, good morning, everyone and thank you for joining us true dikes, there in our second quarter 2021 financial results and operational highlights.

For anyone who hasn't yet had a chance to review our results we issued a press release earlier. This morning, which is available under the investors on media tab on our website I'd like to turn on Dot com.

You May also listen to this conference call via webcast on our website, which will be archived beginning approximately 2 hours. After this call is completed.

Speaking on today's call will be that fairness, President and Chief Executive Officer, Doug Fambrough, Who'll review, our program portfolio and strategy and our Chief Financial Officer, Doug The Guy, who will review our second quarter financials.

We also have sriram around Yang our Chief Medical Officer, Jim Weissman, Our Chief operating Officer, Rob Shovlin, Nally, our Chief commercial officer, and Bob Brown, Our Chief Scientific officer are available today to address questions during the Q&A session.

Following our remarks, we will open the line for your questions.

Such forward looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements.

Now I'd like to turn the call over to Doug Fambrough, <unk>, President and CEO Doug.

Thanks, Tony Good morning, everyone.

Since we just did our Fi Ax 2 day to call on Thursday, I'd like to start today at a high strategic level before discussing particular programs RNA.

<unk> is a powerful modality the 24 years since its academic discovery encompassed a long period of technology maturation, leading to the breakthrough of gallon that mediated delivery of highly modified RNA Duplexes, which has led to multiple approved products with more product candidates at the NDA stage.

<unk>.

Last Thursday afternoon day Serta added our first to this list as our lead product candidate the dozer on demonstrated a strong safety profile and impressive activity profile and the primary hyperoxaluria type 1 or ph..1 population. These data we believe sets up a high likelihood of P. H 1.

Approval in the U S Europe, Japan, and other markets and we intend to submit an NDA to the FDA in Q4.

The <unk> 2 pivotal data directly demonstrate the strength of our galaxy RNA on technology platform I.

I know everyone on the line today is highly sophisticated and can recognize that relative to other approved biotech modalities, such as monoclonal antibodies and AAV gene therapy the.

The ability to practice RNA I on a cutting edge critical mass level is remarkably concentrated in a small number of companies and we're proud to be 1 of them.

It was less than 4 years ago that they saw on I was a microcap company trading at a little over $3 per share no collaborations no galaxy clinical data.

And less than $100 million on the balance sheet. Today, we ended Q2 with almost $710 million. The large majority coming from technology monetization with 6 collaboration 6 different companies engaged in moving day started on galaxy molecules forward 6 clinical stage programs, including the dozer in 9 more.

That have entered preclinical development and our platform now extends R&D I delivery to tissues beyond the liver opening a deep well of opportunities to exploit for years to come.

That's a pretty good 4 years.

What really excites me, though is what we can accomplish over the next 4 years in that time, we expect in the dozer on to be approved and available for patients in major markets around the globe.

We expect to lose Bell Sessa round for Alpha 1 antitrypsin deficiency associated liver disease, or a T. L D, where we hold 100% of U S rights and which is now early on phase 2 we.

We expect to move this deep into pivotal development, we anticipate the RG 6346 program for chronic HBV.

Which is in a phase II combination trial being conducted by Roche will have further progressed in clinical development and completed its 5 phase 2 cohorts and with success, we'll have opted in to co fund pivotal development, bringing our U S. Co promotion option, we expect our D C or D program for alcohol use disorder.

True to achieve human proof of concept and has moved to advanced trials potentially breaking open a vast underserved market.

We expect multiple programs from our Novo collaboration to have entered development and we expect to have opted in to 2 of those but only after having seen positive human clinical evidence, which is to say we get to choose the likely winners after seeing some clinical data.

We expect current development programs from Lilly.

AZ slash lexi on and Boehringer ingelheim to be moving through the clinic, we expect new programs to be entering the clinic on average 1 per quarter over the next 2 years and potentially beyond that at a similar rate amongst those we expect to have taken multiple new galaxy and or <unk>.

Galaxy plus proprietary programs well into clinical development. Indeed, we initiated early development of 2 galaxy plus non liver wholly owned programs, which will debut when they are closer to clinical entry.

It'll be quite a ride over these next 4 years and we have the capital to execute on this because our current cash runway.

With expected revenue from existing collaborations runs into 'twenty 25, that's longer than we've previously disclosed I'll return to that in a moment in the context of the dos <unk> and <unk> 2 pivotal trial results.

But first let's come down from the high level to the actual corporate updates for the second quarter as well as subsequent events star.

Starting with the assessor in.

That we are developing for the treatment of T. L. P last month, we announced interim results from our phase 1 study, which met our objective demonstrating strong dose dependent reductions in serum alpha 1 antitrypsin in healthy volunteers with administration of a single dose of Bell CECI room in this analysis.

Of the 4 completed active treatment dose cohorts at 0.11 point O 3 point O and 6 <unk> milligrams per kilogram they'll susser on was found to have an acceptable safety profile and was generally well tolerated 1 more dose cohort 12 milligrams per kilogram is ongoing.

And our objective is to present the results from all dose cohorts at an upcoming medical conference.

We also began patient dosing in a randomized multi dose double blind placebo controlled phase 2 estrella trial, a bell susser on in June the Estrella Phase III study includes a 24 week cohort and a 48 week cohort to be conducted in parallel each with up to 27 participants.

You'll have a diagnosis of P. I Z type.

<unk> deficiency and a T L D S.

As the study progresses, we are looking forward to having a better understanding of the cells of belts essar on its potential to treat the underlying cause of a T. L D.

Also last month, we announced the FDA acceptance of our IND for <unk>, our investigational Galaxy RNA eye therapeutic candidate for the treatment of alcohol use disorder, we had filed that I N D. In late June.

And we are now clear to begin phase 1 we plan to initiate a 24 week randomized double blind placebo controlled phase 1 trial this quarter to evaluate the safety and Tolerability.

<unk> kinetics, and pharmacodynamics of single ascending doses of D C or D and healthy volunteers. The trial will also assess the interaction between D. C. R. <unk> D treatment and alcohol consumption using standardized ethanol interaction assessments, which will provide <unk>.

Critical pharmacodynamic data to inform the development strategy for that high potential program.

From a collaboration standpoint, we announced in May the Fda's acceptance of Eli Lilly's I N D..4 L Y 3819469 targeting L. P. A 4 cardiovascular disorders L. P. Little a sometimes referred to this is the second.

Nicole stage investigational Galaxy RNA eye candidate from our collaboration with them.

It triggered a $10 million milestone payment to us and pave the way for Lilly to begin phase 1 dosing of the compound which began in June.

There are also 2 Lilly partnered programs and our IND, enabling studies. In addition to the 2 clinical stage programs also when day Boehringer Ingelheim accepted D. C. R. L. I N E 2 and investigational Galaxy RNA candidate for the treatment of Nash to advance to the development.

Age this candidate acceptance triggered a single digit multimillion dollar preclinical milestone 2 day sirna and preclinical work is underway.

And earlier this year, we added to the development pipeline, our second candidate under the Novo collaboration.

Finally, we have 2 programs in our AZ slash Alexia on collaborations that are progressing through IND, enabling studies.

Now, let's turn attention back to the Doser at Ash.

The results were driven by strong oxalate reduction activity in patients with ph, 1 well patients with ph 2 showed inconsistent results.

Sequentially, we see a path to approval in the U S and elsewhere for the ph, 1 indication, but not the th 2 indications.

N P. H 1 we believe that the dose of <unk> is highly competitive with the currently approved product for ph..1 we are reiterating our guidance of a Q4 NDA submission for nodosa rent and we'll continue our development program, including the <unk> 7 and <unk> 8 clinical trials.

For patients with ph, who have compromised renal function or our pediatric respectively.

With a goal of subsequent supplemental NDA submissions to add those populations to the new dose are on label.

Extended observations may or may not provide additional insight in any event, we do not see a near term path to including ph too and our proposed new dosing on drug label.

In the meantime, we look forward to reading out on <unk> for single dose study in patients with ph type 3 upon review of that data, we will decide how to proceed and ph 3.

Up to now we have articulated the strategy of fully integrating our company using the dosing Ren as the vector and rationale for building out a commercial function in the United States.

That strategy was based on achieving the label with at least ph 1 ph 2.

With both of those indications and data in ph 1 comparable to what was achieved in <unk> 2.

We were projecting the ability to ramp sales to where our commercialization efforts would be cash flow positive in a reasonably short period of time.

Without page 2 on the label, however, ramping our commercial efforts to cash flow positive is more uncertain.

With substantially more capital investment required along the way.

I do not believe that is the right choice for dice sirna anti sirna shareholders.

Consequently, we have decided to seek a commercial collaboration partner or partners to make the dozer in available across all major markets, including the U S subject to approvals occur.

Accordingly, we plan to forego investment in commercial infrastructure at this time.

This approach will reduce our planned spend and is the basis for extending our runway guidance from the prior into 'twenty 'twenty 4.2 into 2025.

Importantly, this does not signal any reduction and investment in our pipeline on the contrary, we may choose to increase investment in our pipeline as we fully processed the <unk> 2 data and refine our plans.

To the financial discussion Sri is here with me and is available for questions. After the prepared remarks. So I will now turn the call over to Doug Pagan Chief Financial Officer, Doug.

Net loss for the second quarter of 2021 was $40.8 million or <unk> 53 per share compared to $31.8 million or <unk> 43 per share for the same period last year.

Revenue per the second quarter totaled $41.3 million compared to $44 million in the same period last year.

As of June 32021, we had approximately $162 million of current deferred revenue and approximately $268.6 million of non current deferred revenue. Additionally.

Additionally, we have recorded deferred income related to our sale of royalty rights to royalty pharma.

$1.1 million of which is current and $178.7 million non current.

R&D expenses for the second quarter totaled $56.1 million compared to $53.4 million per the same period last year.

These increases were largely offset by a decrease in external direct R&D expenses.

G&A expenses for the second quarter of 2021 totaled $25.5 million compared to $20.6 million for the same period last year.

The year over year change was driven primarily by increased professional services.

We expect G&A expenses to remain comparable with the current period as we cease our plans to build in house commercial operations for the dose range.

These payments represent an important source of proceeds and demonstrate the value. We expect these collaboration program to continue to generate as they mature.

As of June 32021, we had $709.6 million in cash cash equivalents and held to maturity investments compared to $544.9 million.

As of March 31, 2021.

This cash balance with our revised operating plans and projected proceeds from existing collaborations should provide us runway into 2025.

As Doug mentioned, we will be seeking 1 or more partners for global commercialization of nodosa on economics from any potential array arrangements have not been factored into our stated runway projections.

Our strong balance sheet will allow <unk> to continue to invest in advancing our clinical pipeline supporting our collaboration partners and expansion of our platform technologies.

That concludes my review of the financials I would now like to open the call for questions operator.

Ladies and gentlemen, if you have a question at this time. Please press. The Star then the number 1 key on your Touchtone telephone. If your question has been answered or you wish turned on Luke's yourself from the queue. Please press the pound key your first question comes from Jonathan Miller with Evercore ISI.

<unk> is open.

And then secondly also on on ph I guess now that you've had the weekend to think it over do you have any hypothesis on ph to failure at this point.

And obviously you are still following those patients, but do you have any clarity for us on when we might expect to hear an update from you on with what happened biologically there and then thirdly I know you mentioned, you're waiting for for Fox, Florida to make a final call on this.

But assuming fox for in ph 3 look good.

Would your plan at this point be to include page 3 in the same NBA, ASP, which 1 or would that be a separate submission.

Hey, Jonathan it's <unk> I appreciate the questions I'll tackle the first 1 related to partnerships for any dose ran I think the best way to look at it is we're looking at this as an out licensing opportunity not as a contract sales.

Strategy. So we will be looking at and have continued to speak with both.

Global biopharmaceutical companies as well as significant regional players.

All of them have greet acumen in the rare disease space, we will continue those conversations.

Divide our updates as soon as we have gone to <unk>.

<unk> of those discussions.

Thanks, Rob.

Uh huh.

So.

John.

You asked for clarity on non ph too.

The Peach to result, with kind of a shock to low ph field certainly have interacted with some some kols and there was I think a fairly broad expectation that there would be activity.

In ph to end.

There isn't debt at the current time, leading hypothesis for why we didn't see consistent results with a monthly dosing regimen of <unk>.

Those are on particularly after the parents signal from a single dose.

In patients.

Biochemical level work in academia wouldn't we're not going to be doing that here.

With respect to ph 3.

The base operating assumption should be our NDA will be for ph 1 only.

We will see what the results are from a single dose of <unk>, 3 and assess them, but I think anyone planning or projecting should assume the NDA filing is just the H 1.

Thanks, John.

Thanks, so much guys.

And your next question comes from Manny for Haar with after the building rank on your line's open.

Hey, Good morning. This is Rick on the line from Mani Thanks for taking our questions.

So first.

What are some of your expected timeline for getting visibility into establishing commercial partnerships for ph..1 are there any guidance you can provide here and I guess also do you anticipate that timelines would be different based on whether you choose to have a single partner for commercialization or multiple partners here.

Hey, Rick it's Rob in terms of our partnership discussions those are ongoing growth.

I had mentioned both globally and.

With regional players I don't see it as a rate limiting step in terms of making the dose for an available we're continuing first and foremost with filing the NDA. The NDA as a key resource document for filings beyond the U S. So think about it this way.

We're on track as guided to Q4 filing of the NDA concurrent to that we're continuing the discussions with both global and regional players.

I would expect us to.

The next couple of quarters be able to come back with.

Clear conclusions in terms of what we're going to be doing with nodosa and outside the United States and as well with a partner in the U S.

Okay got it thanks that's helpful.

But it's also taking a step back.

What does this change in strategy, meaning per commercial commercialization plans for the rest of the wholly owned pipeline.

Just trying to think is there any read through here to the rest of the pipeline is there a certain threshold.

To meet to start building commercial infrastructure or is it more likely that we're going to see commercialization partners.

On the pipeline.

So Rick I don't think Theres any read through for the subsequent programs. The next 1 likely to come down to that stage would be the <unk> <unk> program, which has a focused call points. Similarly.

Which would be further out has a focused call point in addiction medicine, both of which would be appropriate for us to build out a specialty sales force.

When that comes to pass.

I think we set a reasonably short period of time.

And then that will be a source of cash in the company. If we don't have confidence in that then it doesn't make sense to make that investment that'll be made on a case by case basis, but the next opportunities could support building out that infrastructure.

Alright, perfect I appreciate the detail here, thanks for taking my questions again.

Your next question comes from Matt too.

Kumar with Goldman Sachs. Your line is open.

It's not publicly available about the online low made he drug dependent makes your favorite Bell says around or how should we think about the kind of election, but true Bell says ran over over that on a drug.

So my view that took into account.

On pound and our own frankly that weigh on that decision.

And it's not a decision we're going to revisit.

Okay, and then thinking about the kind of extra hepatic space.

Can you give more visibility, but kind of where are you thinking about kind of pursuing.

Previously discussed the CNS space kind of how are you thinking about that now on how you can get.

Granularly timelines for putting extra hepatic compounds into the clinic.

So I am really pleased at the breath of delivery to other tissues that we're seeing.

So in the CNS, we are partnered with Lilly and so I think much of the CNS work will be in that under that collaboration but we have talked about adipose tissue and muscle on tumor associated immune cells and there are other tissues. We haven't mentioned that we're achieving delivery I do notice that.

Certain other tissues are a focus for other companies, notably muscle would fall into that category and I think overall, there are probably relatively fewer number of opportunities in muscle.

I would like to see di sirna in more of a white space.

<unk> field running room. So you when we revealed our programs I think we will see that we're probably tacking in a slightly different direction than some of the other ligand really tied companies going outside the liver.

Drawing on the strength of Galaxy plus to get the other tissue types.

The preclinical timelines are now.

The plan the best you can but that Covid is really disrupted preclinical timelines with respect to things like non human primate availability.

Yeah.

We're not going to guide right now on the clinical entry timing.

And we do want to be fairly close to a clinical entry before we talk about what we're doing because we do expect.

People to jump into the programs we're doing so.

Yeah.

Excellent thanks very much.

And your next question comes from Stephen Willey with Stifel. Your line is open.

Yeah. Good morning, Thanks for taking the questions maybe just a couple of quick clinical ones.

And Australia can can can you talk about.

The extent of fibrosis, but you're allowing patients who will be.

Eligible to enroll that study or are these going to be F. Threes low fours are you, allowing cirrhotic patients into that study and then.

With respect to AED.

In terms of patient eligibility I mean, obviously this is a healthy volunteer population healthy volunteers I guess do consume alcohol.

Is there any kind of restrict channel or do you have some kind of.

Our guardrails around consumption in that study or at least attempt to.

Just for the first phase 1 experience.

Yes.

Hyatt <unk> in Australia, our goal is to enroll the population that gives us a good read on the benefits of knocking down the mutant protein and potential consequent benefits on liver histology. So we are enrolling abroad.

Population with representative fibrosis.

<unk>.

So that's that's ongoing.

Gold data is to make sure that we've designed the study in a manner that gives us a good read on seeing a difference in the size of the study that we have.

We have designed.

Certainly the worst case patients.

The compensated cirrhotic.

On the other part of this trial.

Rather than debt will be on a broad range.

And AED Steven could you repeat your question on that for me. Please yes, Im just kind of curious so in.

Any kind of really efficacy signal that could emerge out of that study just based on the fact that youre going to see healthy volunteers consume alcohol during a 24 week period post dose.

Yes, So I think we expect to do this in a stepwise manner.

Whether or not we have established the biological stayed in the human of achieving the necessary knockdown in the liver and what effect that has.

Ongoing in our guidance, we will of course be for people to avoid alcohol intake my expectation is that over time. The phase 1 study will give us a read on what people's reaction to drinking alcohol outside of the trial might be but coming in in the initial phase the single ascending dose our goal is on making sure that the exposure goes on low.

He is on the controlled setting.

To make sure that we have a clear read on what the CDO debt August but over time and make sure that on the multiple ascending dose we shouldn't be able to gather more reads on efficacy.

So in essence, we do expect.

2 to learn a lot about the physiological response of.

Someone who has taken <unk>.

2 the intake of alcohol that is not the approvable endpoint. The approvable end point will be likely harm reduction in patients who have <unk>, that's sort of loss control and harm reduction being an assessment of their ability to regain more control over their consumption of alcohol. So on that direct serve approvable endpoint, we're not going to get anything but.

There is a.

Believed to be very clear linkage between the physiological response and that behavior as seen by the rates of <unk> and people with naturally occurring a L. D. H 2 mutations not exactly the same thing we're doing but closely related so we should get the important physiological feedback.

And that's something we'll discuss and look forward to talking about it when we see it.

Alright, thanks for taking the questions.

And our next question comes from Yaron Werber with Cowen Your line is open.

Hi, guys. This is Brendan on for you Ron Thanks for taking the question.

Just a quick 1 from US I guess really looking at a 180 day program.

So I guess looking at the landscape here, what can you tell us maybe about bill.

<unk>, specifically and maybe more so youre kind of goals and expectations for the phase III.

That you think are going to really be necessary to differentiate the drug I guess my point is really kind of given that there are other competitors a bit ahead of you on development in that space as well, where do you really see the bar.

For both us around there to stand out and maybe what kind of gives you confidence you can meet them.

Hi, Brendan so there is 1 other company.

In phase 2 to treat.

L D. So.

Exactly a crowded field.

And I'm sure you're aware almost every pharmaceutical market has multiple entrance.

But when does think about how the various products are differentiated in this case because the mechanism of action is the same there isn't a mechanistic differentiation.

On the ph 1 indication.

We have a.

What I think is a very positive differentiation based on the mode of administration with our Prefilled syringes that for offer administration by patients with ph 1 at home.

On their own.

Compared to the competitor product with SASSA health care professional.

Walt and his weight based and so variable volume and so not not in pre filled syringes.

We shall see in <unk>.

As we head into pivotal trials on a competitor heads into pivotal trials.

What those.

Regimens are and so.

Assess how we want to.

At present, the product relative to competitor, but I think the most important dynamic has to do with the fact that most of the diagnosed patients with AA.

<unk> liver disease.

As a therapy for the lung and RNA I as a therapy for the liver and <unk> see that the competitor product as sort of a collaboration with an augmentation provider. It is as it happens on minor interest in the augmentation market and so I think that presents a fairly obvious.

Counter sort of strategy for us with respect to pairing our RNA compounds with augmentation that we intend to pursue there isn't any particular.

Time.

<unk>.

To do that.

Right.

There I think that <unk>.

Solution.

<unk> associated with lung and liver.

Is going to be very interesting and it'll be 2.

<unk> advantage to be with the market leader.

As opposed to with a more minor player.

Alright, great. Thanks very much.

Your next question comes from Luca <unk> with RPC, Inc.

RPC capital.

Your line is open.

Oh.

Yeah, low cases from RBC capital market.

A few questions here, maybe the first on net net dose you rank if I recall it correctly, probably guidance estimated peak revenues to your peak sales I should say between $501 billion. I'm wondering if you can comment on how youre thinking about peak market opportunity here now that you've seen the phase III.

And then maybe on business development wondering if there's any appetite to broaden the scope of your extra padding delivery via BD.

Seen recent deals between now you're honest and bicycle and volume and Pepsi Dream. So wondering if this is something that you're also considering and then finally, maybe on day..1 wondering if you can comment on the timeline for seeing the actual data in patients here. Thanks, so much.

Hi.

<unk>.

Trying to right.

Keep track of the questions. There I think that those are in the sales potential was the first 1 so I'm not going to get into detailed numbers here, but we had ph too in our forecast as being a significant minority.

Of the total sales.

Less than half, but significant amount for ph 3 relatively small fraction.

But <unk> was a as a significant minority does taking on auto label or not getting it is included in the label has a significant effect, but I'm not going to get more specific than that on our prior forecast, which obviously is no longer applicable given ph twos.

No longer included.

Shifting to business development around delivery.

That is something that we are considering and we've had conversations with players in the field.

Having said that.

We are very pleased with the delivery, we're getting with our in house technology, which as I mentioned is getting us to a pretty broad array of tissues. So we're not feeling a particular.

Need at this point.

No.

It's definitely in the mix and there are some really interesting approaches out there.

But the strategy that we have taken in our research labs has been paying off and we're really pleased with our in house work.

So on and maybe timing of the <unk> data for from Australia is there any kind of.

Guidance that we should think about it.

Alright, I forgot about that 1.3 audits quicker.

Study started enrolling earlier this year.

Getting some sites on board on Europe. So I think it will be sometime we will need to have a decent number of subjects having had.

On the right number.

Paid biopsies to be able to meaningfully present information that is into accretable and from which we can draw. Some conclusions. So that's above the guidance I can give you right now.

Got it Super helpful guys. Thanks, so much.

Your next question comes from Yigal <unk> with Citigroup. Your line is open.

Hi, Doug. Thank you very much for taking the questions I just had a high level question.

At this point do you believe you have an optimal number of pharma partnerships are you willing to entertain additional partnerships to further extend the footprint for the Galaxy technology platform <unk>.

Or is the case are there certain disease areas that you believe would be better suited for partnering versus developing on your own.

Okay.

Sure Yigal happy to take this 1.

We've got a really nice set of collaborations and.

They're good partners to work with.

Is there is a logistical challenge and adding more names to that list.

Yes.

Our ability to make clinical candidates definitely outstrips, our development capabilities and I think that's true for the foreseeable future.

With an excess of opportunities and candidates.

To me and I think to the whole team here.

It makes sense to have someone else take things forward are not going to take forward.

So there is still.

And our pressure to bring in cash which was fairly strong at that period of time. Now is we are we have a lot of luxury of choosing our spots and so our posture.

Posture is a little different with respect to collaborations now it is to really spend our time figuring out what we're going to do.

And then later, we'll take the things that we're not going to do.

And we would like to find a path forward for those.

Some of the spaces that we are working and we've mentioned deliberate adipose tissue. They are clear metabolic and sort of obesity indications associated with that debt.

Probably are not rate per day sirna.

I would note we're already collaborating with the 2 largest players on the metabolic space.

It would be nice.

To perhaps extend those collaborations into galaxy plus wouldnt burden the logistics.

Any more but it would satisfy the goal of bringing in capital and making sure those programs move forward. So I see a role for the collaborations but whereas in 2018.2019. It was a primary part of the strategy. It's really a secondary part of strategy now and primary is building out our own per.

<unk>.

And then the collaborations will.

It will fit in.

And a secondary role, but it still.

An important piece I think 1 of the things that all of us would like to do per our.

What we have is our vision statement here at <unk> Sirona is maximize the impact of RNA eye on medicine that means getting a lot of drugs. We can meet candidates can make a lot of candidates it's very modular.

So we want a lot of candidates to move forward I'm really pleased there are 15.

We want that number to keep growing and growing so I hope that gives you. Some perspective that is well short of any sort of guidance, but I think does it is informative to what our what our mindset is about how we're going to think about collaborations.

Thank you that's very helpful. Thanks, Doug.

And your next question comes from May on non Paani with B Riley Securities. Your line is open.

Hi, Yes. This is William wood on for Mike on Tommy. Thank you again for taking our questions on glad to see you being proactive with your corporate strategy the follow up.

From box to last week or so on.

I was curious about what learnings might be applied from your ex U S licensing process and now that you have made the choice to make.

Global rights available.

Hey, volume, it's Rob in terms of kind of.

Some insights on behind a couple.

High degree of interest.

William net interest not only was a new Dresser, Inc.

Beyond the U S, but a number of the parties kept coming back to us and saying we'd love the U S.

That puts us in a really good spot to continue these conversations both with global and regional players.

Bottom line is.

Sure he is going to do.

Wherever we can to most appropriately appropriately put this into the market so that patients have an auction.

Not only in the U S but globally.

Frankly these conversations.

I feel put us in a good place to.

Work towards that so the key insight is that a number of the players that we've been talking to.

Some are some have had some interest in the U S as well, but we'll be continuing to look specifically across all the different stakeholders and.

Proceed accordingly.

Doug.

Thanks Ross.

Oh go ahead.

I didnt have anything more debt.

I think we're going to wrap up but at this point operator is there.

Any additional question.

Alright.

That concludes our question and answer session I would now like to return the call over to Dr. Tom Farrell for closing remarks.

I want to thank everyone for participating this morning on or reiterate that.

<unk> II is an important milestone and I am really pleased with our platform that bodes very well for the future and I look forward to updating additional galaxy programs as they mature and subsequent calls thanks, everybody Bye bye.

Ladies and gentlemen. This concludes today's conference. Thank you for your participation and have a wonderful day you may all disconnect.

Q2 2021 Dicerna Pharmaceuticals Inc Earnings Call

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