Q3 2022 WAVE Life Sciences Ltd Earnings Call
Good morning, and welcome to the wave Life Sciences third quarter 2022 financial results Conference call.
At this time all participants are in a listen only mode. As a reminder, this call is being recorded and webcast I'll now turn the call over to Kate Rausch V. P of Investor Relations and corporate Affairs at Wave Life Sciences. Please go ahead.
Thank you operator, good morning, and thank you for joining us today to discuss our recent business progress and review our third quarter 2022 financial results.
Joining me in the room today are Dr. Pappano, President and Chief Executive Officer, and really quite sharp Chief Development Officer, and Tom Moran Chief Financial Officer.
Press release issued this morning, and a slide presentation to accompany this webcast are available on the investors section of our website Www Dot wave life Sciences.
Before we begin I would like to remind you that discussions during this conference call will include forward looking statements.
These statements are subject to several restaurants certainties that could cause our actual results to differ materially from those described in these forward looking statements.
Factors that could cause actual results to differ are discussed in the press release issued today and in our SEC filings, including our annual report on Form 10-K for the year ended December 31, 2021 and our quarterly report on Form 10-Q for the quarter ended September 30 of 2022.
We undertake no obligation to update or revise any forward looking statement for any reason.
I'd now like to turn the call over to Paul.
Thanks, Kate good morning, and thank you all for joining us I.
I will start today's call with some recent highlights and then introduce our Chief Development Officer, Anne Marie will be quite sharp.
Emory will provide updates on our most advanced therapeutic program and finally, Kyle will discuss our financials.
2022 has been a pivotal year for wave.
At the start of this year, we said, we would deliver data update from three separate clinical trials as well as it adds an entirely new modality Youre candidate nomination.
To date, we have delivered the first two clinical data updates including results from our ongoing study.
Study, showing potent and durable target engagement with W. E E.
And most recently the first clinical data from our select HD trial for Huntington's disease, indicating a real collective target engagement, but W. B E.
Both these datasets validated our novel chemistry, and supported the clinical translation of our discovery and development platform.
We're on track to deliver clinical data from our Duchenne muscular dystrophy open label clinical trial later this quarter.
By designing the oligonucleotide to efficiently harness eight our enzymes, we haven't locked entirely new biology with our platform.
In the third quarter, we selected our first RNA editing or aim or development candidates W. V E <unk> for Alpha one antitrypsin deficiency or <unk>.
In September we held a virtual event spotlighting the biology of this disease.
Our RNA editing therapeutic approach and existing unmet need that we hope to address with W. E E.
We were joined by a well known clinical expert in the treatment of ATB. Dr pilot Bogart with shared perspectives on the current treatment landscape and the benefit of having one therapy, such as OPEC to address both liver and lung manifestations of AED without permanent genetic alteration.
I invite you to tune in to the replay on our website he met with live events.
Preclinical data for WB.
Were also presented for the first time in a scientific setting at the Oligonucleotide Therapeutics Society meeting last month.
Yes.
At Ots and other recent forums, we continue to highlight the broad applicability of waves unique oligonucleotide backbone.
This includes using immersive up regulate gene applying prism chemistry to SCR.
To achieve remarkably potent and durable violence in preclinical models.
This coming week, our Chief Technology Officer, John drove argued will be presenting these data in other results that demonstrate the versatility of Weibo platform at a special symposium at the back.
Alongside other innovators because all of these great chemistry.
Before turning the call over to our Chief Development Officer, Anne Marie I would like to take this time to briefly introduce her to those working again.
And Murray joined wave at the beginning of this year as a senior leader at a clinical development organization.
Previously he spent eight years at Roche holding leadership positions of increasing responsibility in global drug development and global program leadership within the neurology and rare disease therapeutic area, particularly in Huntington's disease.
We are very excited to have Emory right through her expanded role in September as we know her deep experience across clinical development and commercial activities as well as regulatory and compliance will be critical to wave as well.
Our innovative pipeline.
With that I'll now turn the call over to Anne Marie Thanks, Paul.
I'm pleased to be speaking with you all this morning and to share exciting progress we've made across our development program.
My decision to join weight.
This year was grounded on three things first how much of the company's novel Oligonucleotide platform second the robust pipeline Brexit on areas of significant unmet need and that the innovative approach to drug development.
Successfully advancing each of our three ongoing clinical trials sharing data at key points and adapting our studies to refine this level on frequency along way.
We showed data from both our ILS and F G and H B trial shown in dark blue on the slide and we are on track to deliver data from yet another clinical trial, our DMD study in this quarter.
In addition, we have successfully advanced waves or is there a six our development candidate for AIG D. Initiating <unk> DNA Berthing preclinical studies and positioning us well to begin clinical development Nextgen I will describe the progress in each program and the slides that follow.
I'd like to start by focusing on page eight I think.
Deeply involved in the therapeutic development for this devastating debate for the past several years and prior to joining weight and through that experience have come to appreciate the criticality of preserving wild type Huntington protein as parts for therapeutic approach to H D.
Huntington's disease is a monogenic autosomal dominant I'm fully punishment genetic disease, which affects the entire bright.
At the time, the accumulation of toxic mutant Huntington protein as it comes to the beneficial effects of the wild type protein leading to disease progression.
So it is understood that the mutant Huntington protein as toxic and that the wild type Huntington protein as essential and placed several critical roles in the central nervous system and these include regulation of sign up tick on protein transport promoting awareness survival and formation of function of cilia, which are essential to regulate <unk>.
Just one.
And any of these pathways could be expected to adversely impact response.
<unk> western in patients.
So it remains an open question as to whether there is any space level of wild type Huntington knockdown in the stretching of mutual Huntington knockdown.
In H D and given HD patients already begin lines with 50% less wild type as compared to healthy individuals.
Our program is the only one able to answer. This question is why did the reserves right is the only allele selective therapy in clinical development.
With interest equal dosing, we're able to take advantage of targeted CNS delivery without invasive surgical procedures and without board systemic exposure that result.
RP is incredibly encouraging initial data suggests with our next generation <unk> chemistry, we are indeed selected they engage with target.
As we reflect on the path of why did you reserve for each of the clinic, demonstrating potent and selective target engagement and relevant preclinical models with coach our development process.
On the left you can see the steps we followed.
First allele selectivity was demonstrated in vitro and Ips senior Orange with <unk> prior to optimization and further using reserves reached target engagement data from the back HD transgenic mouse, along with CNS tissue concentration from nonhuman primates, we were able to model the likely pharmacologically active dose or the reserve.
Humans. This allowed us to start the trial at dose levels predicted to engage target and guided dose selection.
On the right you can see the data we shared in September .
This was the first data update from US, let H D clinical trial in <unk> and H D patients with the SNP three play more cushion.
I've reached five days the main reduction in CSF mutant Huntington was 22% from baseline and approximately 35% of some hedges to keep us.
In addition, wild type Huntington protein levels over the same period, a pay consistent with allele selectivity.
These analyses we pulled the data from the <unk> 60 milligram cohorts and as a reminder, patients dosed with 90 milligrams were not included as none had reached 85 at the time of the analysis.
We have just presented this update at the H S. Jamie.
These results are very encouraging and in consultation with our safety monitoring Committee, which is sorry did you expand all three cohorts 30, 60, and 90 milligram Tibet to define the treatment effect undecided the optimal dose for the multi dose phase of the study.
Dosing in the expanded cohort is already underway and we are on track to share data from the expanded single dose cohorts in the first half of 2023.
Moving to <unk> and 531, our first muscle biopsy data from our open label clinical trial in boys with DMD amenable to exon 53, skipping as expected this quarter.
This will also be the first clinical data with its blasting or the Gannett oligonucleotide containing a pan modifications.
Preclinical data in <unk> mice, and nonhuman primates support the potential for Pan chemistry to significantly improve both plasma and muscle concentrations overall first generation PSP compounds.
These increases in tissue concentrations were associated at clinically relevant doses with improved skipping efficiency in the double knockout mouse improve muscle respiratory function and resulted in 100% overall survival and a knockout mouse model, which typically exhibit yesterday right.
Right.
We have now completed dose escalation of <unk> hundred one to the 10, Meg ticket does and move forward into the multi dose phase of the study this.
This dose is the human equivalent level in the range of dose just explode in the double knockout mouse and that resulted in improved function in 100% survival.
The study is open label, we can shed the pharmacokinetic data from all four doses explode in the single dose phase of the study.
Slide 12 illustrates plasma concentrations of <unk> III, one over a period of 20 days following single IV doses at both dose levels.
Our first generation PSP compounded all set up.
Remember five mixed the cake was the top dose administered in the now discontinued phase III study.
Approximately one week following dosing concentrations measured with one make the case of advisory one exceeded those of five K Cup stupid Ashton.
Plasma concentrations of 10, Meg to kick off with <unk>.
531 were approximately 58 fold greater than those that you achieved the stupidest thing overall.
These data demonstrate a unique and distinct pharmacological profile within 531 as compared to a first generation chemistry and make us very hopeful about the day two we expect this quarter.
The initial cohort in our clinical trial includes boys with DMD amenable to exon 53 skipping to a transition from the single dose portion of the study and are now receiving three additional cohorts of <unk> three one every other week followed by a muscle biopsy.
To date, and 531 in Pittsburgh, and well tolerated there have been no SAE and no events meeting certain criteria all adverse events have been mild with the exception of a moderate COVID-19 infection.
It's important to recognize that dystrophin protein levels with other oligonucleotides have been reported following up to 48 weeks of treatment.
While we will report results from this initial cohort after three biweekly doses and with that in mind. The purpose of this data set is to confirm as suggested by the plasma and animal data that Pan chemistry leads to higher muscle tissue concentrations and improved interest earlier uptake as compared with our cash generation compounds.
Of course, exon skipping and dystrophin protein is also being measured and we expect to share. These data on <unk> hundred one tissue concentrations and localization exon skipping activity and dystrophin expression like in this quarter plus.
Positive data from the initial cohort would drive us to expand the study with additional patients and we'd also speak to regulators about the potential package to pursue an accelerated approval pathway.
Patients with exon 53 amenable mutations represents approximately 8% to 10% of the DMD population.
The board the promise of the NT <unk> hundred one program is that a positive signal opens up the possibility of applying comparable technology to other splicing oligonucleotides and planning is underway to explore this possibility.
As mentioned positive data from the initial cohort with not only drive us to expand our exon 53 study, but we would also accelerate work and other excellence with Pn modify splicing oligonucleotides, enabling us to leverage our previously developed multi X all expansion strategy.
Moving to focus Jeannine, all clinical trial for <unk> in patients with <unk> 72 associated Alice and S. T D.
I'm pleased to report that dosing continues in the multi dose cohorts as a reminder, we shut our first clinical update in April of this year, where we demonstrated robust durable and statistically significant target engagement. Following just a single dose of waves or was there at all.
Based on the potency and durability of on target effects and our last day SMB meeting it was decided to expand multi dose portion of focusing nine to evaluate quarterly dosing.
Typically patients in the 20 milligram single dose cohort will move to a 20 milligram Fortunately dose cohort and we are also open to 10 milligram.
Quarterly dose cohort.
With potency and durability, enabling our ability to move to quarterly dosing regimen, we would expect to be more attractive to patients.
Got to share additional data from all focusing on cohorts in the first half of 2023.
In the fourth quarter, we also initiated a rising labor extension to provide patients from this focusing on trial the opportunity to continue to receive like there was a recall.
Having established target engagement through the balancing mechanism and we hope later this year the splicing mechanism, we move to our next innovation waves. There is there are six it's RNA editing development candidate for IHG D.
This past September we shared a comprehensive update on the preclinical data sets that support this candidate as a potential first and best in class treatment for this disease.
In the left panel, we demonstrated the efficacy of waves. There was there are shifts in the <unk> mouse model, which is well established model in the sales and after backing weeks. There was a seven fold increase in serum IHA with.
With <unk> compared with PBS control.
Importantly, mass spec data in the middle panel confirms that we are generating wild type.
Protein, which was functional as measured by the neutrophil elastase activity as shown in the right panel.
IND, enabling studies are ongoing and we are on track to submit <unk> in 2023.
I will now turn the call over to Colin Moran our CFO .
Thanks Anne Marie.
Net loss for the three months ended September 32000, $20 million to $39 million.
$6 $2 million in the same period in 2021.
We reported approximately $300000 in revenue for the third quarter of 2022.
Compared to $36 $4 million in the same period of 2021.
The decrease in revenue and the increase in net loss year over year was primarily driven by the amendment.
<unk> the prior year quarter.
R&D expenses were $27 $6 million for the third quarter of 2022.
Compared to $31 $1 million for the same period in 2021.
This was primarily due to decreased external expenses related to our agency program.
And decreases in other research and development expenses, partially offset by increased external expenses related to C&I and our other clinical and preclinical programs.
General and administrative expenses declined to $11 6 million third quarter 2022.
The $12 $9 million last year, primarily due to a decrease in share based compensation expense as well as decreases in other external general and administrative expenses.
We ended the quarter with we ended the third quarter with $122 million in cash cash equivalents and short term investments.
Our cash runway remains unchanged from previous guidance we.
We continue to expect that our existing cash cash equivalents and short term investments will enable us to fund our operating and capital expenditure requirements in 2020.
As a reminder, we do not include any potential milestones or opt in payments under our Takeda collaboration and our cash runway.
I'll now turn the call back over to Paul.
Thanks, Kyle we're excited about all the progress and clinical data generated to date for our pipeline with <unk>. We are unlocking new biology to diversify the applications of our platform and W. E. <unk> is the first demonstration of the terms of the potential of RNA editing.
Our discovery team is actively evaluating new RNA editing program in multiple disease areas, including CNS product and renal diseases.
As a reminder.
<unk> do not require llp's for AAV, which have unique delivery challenges tissue types.
We expect to deliver more updates in 2023 on our discovery pipeline.
We have a disruptive oligonucleotide platform with unique multimodal guide strength, enabling us to target the burst biology.
We are delivering clinical translation of our initial program and diversifying our pipeline with everything.
Partnerships remain a key priority to maximize the value of our platform support expansion of our pipeline and unlock value in GMP manufacturing.
We intend to collaborate with partners, who share our broad and ambitious vision for our platform.
We have also begun to deliver on initial manufacturing proof of concept work, including process development with the central GMP customers, we are well positioned to expand these efforts.
We set out to deliver three clinical updates this year and are on track to accomplish this goal with the expected DMV data this quarter.
Looking into 2023, we are positioned to deliver a continuous flow of data updates, including additional data from our HD and 99.
Nine FTE trial.
We will also move <unk> into clinical development, which is positioned to be the first ever RNA editing therapeutic in the clinic.
We're also well capitalized to execute on these milestones and with that we'll open up the call for questions operator.
Thank you.
As a reminder to ask a question you will need to press star one on your telephone please standby will be compile the Q&A roster.
One moment for our first question.
And our first question comes from Salim Syed from Mizuho. Your line is now open.
Great. Good morning, guys. Thanks, so much for the color and congrats on the progress a couple for me as we head into the DMD readout possible.
Paul obviously.
<unk> had some good data this year, both in <unk>, ALS and Huntington's with neurochemistry.
Sort of thinking about conceptually speaking.
Are there any risks in your mind as to what could derail the thesis into DMD, whether its biology or otherwise as we translate from preclinical to clinical.
Given that we've had targeted engagement for both FTE BLS and Asa datasets.
And then just related to that.
The open label are obviously getting close would just love to hear a quantitative bogey something like 3% that people should be thinking about here is that a good number for you guys.
The dose data today.
Or is it higher or lower just would love to get a quantitative bogey if that's possible. Thank you.
I'll start with your first question and thank you Felipe.
Because we have made extraordinary progress, but anytime we develop a medicine theres always the potential risks so it'd be remiss for not again about that.
What was important to us, particularly in the setting of advancing in Berkeley one.
Behind our previous experience with <unk>.
Two extensively model it in animal models of phenotype.
Attempt to predict.
As you referred to we've seen consistent modeling as we looked at silencing across benign HD in terms of thinking about how we establish our starting dose in the clinical studies and as we shared today with doses now up to 10 Meg per kg I think we've established the priority.
Paradigm, that's aligned with our preclinical models.
That has us optimistic I think it's important to remember in this transition goes into your second question around a numerical target.
Is that we are dosing this with three subsequent doses after establishing that so the dosing period is a lot smaller than where other studies have gone and therefore, we haven't laid out an exact target other than to say, we'd like to see the translation of the concentration.
We'd like to see the distribution different if you recall, we shared at the muscular Dystrophy Association meeting following subadar Austin that we werent getting exposure of the muscle cells two drugs, so we'd like to see that translate and we'll be showing that.
In this study and then looking at exon skipping as well as dystrophin are going to be important I think the numbers that you pointed out would be.
Exceedingly positive in a setting where we have this.
In.
This short timeframe to evaluate dystrophin being produced.
That if we see those numbers that would be extraordinarily encouraging to then do with Emory alluded to on the call, which is really to expand both the number of patients significantly powered study.
Superiority to other at 53 gifting oligonucleotides.
As well as drive.
To drive our path forward. So I think that's the targets to date.
Hopefully I answered your question.
Super helpful. Thanks, So much Paul.
And thank you.
And one moment our next question.
And our next question comes from Paul Matisse from Stifel. Your line is now open.
Hi, This is James on for Paul Thanks for taking our question.
I just had one on <unk> actually.
And maybe it's a little bit more theoretical but on the safety side I guess, what makes you comfortable.
There with the downside of recruiting aydar away from its kind of endogenous functions it'd be great just to hear kind of any color or thoughts you have on kind of what we know about those kind of endogenous functions and any risks associated with with leveraging leveraging it away from Ed. Thanks, So much.
No. It's a great question, because it's obviously something we spend a lot of time, both on the platform aspect as well as ultimately and running the preclinical candidate nomination.
Or or speaking to the platform aspect and we have shared these data at meetings.
It does have an endogenous function on an editing a transcript and so one of the studies that we have done in early on with looking at applying multiple <unk> within <unk>.
They'll look at whether or not you exhaust the enzyme from its function and what we were able to show is we can edit multiple transcripts within the cell and therefore realize that we're not capturing ADR and <unk> fell from preventing it from doing its normal function. So I think your point is spot on and something we kind of spent the early portion of our platform.
Relevant.
Our capability really studying and making sure we understood well as.
As we've been translated that into fixed it was important that obviously, we've done a lot of non GOP safety studies as part of our candidate nomination and important part of that.
Lots of companies define candidate nomination different ways, but we embed.
50, tolerability into that and again have seen good translation from our platform development into our program development and at this point are confident in the safety of the program. We're moving forward next steps are obviously, we're engaged now in GOP Tox studies to advance the program into a Cta submission next year.
Great. Thanks, so much.
Okay.
And thank you.
And one moment for our next question.
And our next question comes from Joon Lee from <unk> Securities. Your line is now open.
Hi, Thanks for taking our questions.
I'd love to hear and Marines views given her experience with Tom and understood that Roche what percent knockdown of mutant allele would be sufficient for clinical benefit in our view and and for what duration of follow up.
The duration of follow up that will be needed to show.
Great.
And I have a follow up.
Thanks.
Yes.
As we reported today from the initial data that we've already seen that we are in the range of about 20 tobacco.
With allele selective okay and this is what will it be aiming to replicate and as we move into the multi dose phase of the study.
With that magnitude of knockdown, how long do you think you would need to follow the patients to see a separation from placebo.
So.
<unk>.
Obviously ph D S.
<unk> slowly progressive disease, but you would expect to see something within six months to a year.
Okay.
We'll see.
Richmond with following based on the multi dose phase.
Great.
Next question for <unk> are you looking to focus more on the liver or the lung aspects.
And how does that impact your decision or.
The types of patients you want to enroll in the trial. Thank you.
Yes, I'd be happy to hand, it back to Ann Marie but I think ultimately in developing the program at the very beginning and really thinking about it at the first opportunity to develop editing.
Looking at both.
And it is important to us in the development plan, obviously measure the plasma level.
Proteins that we can relate that to.
The IV protein confusion, we will also be embedding into that study liver biopsies to be able to look at it.
The decrease hopefully in liver aggregate. So we'll be building the compendium of that program together as we advance it.
The Z patients as we think about it.
But as we get into the patient aspect of the study.
Those patients that tend to progress starting with pulmonary disease progressed deliver so the important piece for us in the initial period of that study is less about functional outcome in early on more about biomarker driven outcome that will answer to your important question. Obviously, one from a platform perspective, the significance of editing and.
The ability to see the translation from preclinical models to human models, and then secondly, being able to continue to follow.
Those patients forward in terms of that.
Yes.
Thank you.
Thank you.
Thank you.
And one moment our next question.
One moment please.
And our next question comes from Union from Jefferies. Your line is now open.
Thank you. So you mentioned the color on Kashi is.
Through 2023.
Without any potential milestone from Takeda So question number one.
Are you expecting.
Milestone payments based on your development.
He's from Takeda in 2023.
If not why would it be the <unk>.
Thank you could receive a milestone and second question is given the.
Hum.
<unk> eliminated cash are you thinking about prioritizing your programs because you havent been quite excited about RNA editing, so why not prioritizing the program and kind of a conservative cash. Thank you.
Great question.
The two are not unrelated so obviously, we have a productive collaboration that's ongoing with Takeda that involves our nine AOS FTE program at our Huntington's program. So we are continuing to advance that and exactly to your point.
Those drivers have the potential for opt in that opt in triggers that 50, 50 profit split R&D split, which decreases our expenses and also had a series of milestones associated with that we look at that collaboration in those programs advancement as being very intertwined.
No.
How we are thinking about that to Kyle's point earlier on the call. We don't forecast that into our run rate statement. However, progress on any clinical success in those programs would be tied into those.
Central opt in payments and potential milestone payments. So we look at that as well.
Balancing those program.
Secondly, we're continuing to do really important meaningful work as we said on building out and expanding our central manufacturing capability and so we've been doing that work internally that helps support and bring in potential additional resources as it relates to HDD. We are excited about that and as you've seen over.
This past year, we've prioritized it placed the investment in it and along with the hard work of the team at wave have now developed a clinical candidate that we anticipate bringing in the clinic next year. So I think as we look forward. Yes, there are potential sources of cash inflows coming from manufacturing coming from milestone payments with our exist.
<unk> partner and as we said on prior calls our work and focus on additional business development activity to bring in non dilutive capital to fund the platform engine to continue to build on that editing capability and other oligonucleotide experience we built.
Can I ask you one follow up question regarding <unk>.
The cadence of Glen is there any data points.
Event to debt.
Partner has to opt in.
And there are drivers around proof of mechanism and I think as Anne Marie alluded to the datasets that are upcoming we think those data are consequential.
Having that discussion with Takeda.
You.
And thank you.
And one moment for our next question.
And our next question comes from Luca <unk> from RBC. Your line is now open.
Hello, everyone and thanks for taking our questions. This is Lisa on for Luka.
Congrats on the progress this quarter.
Just a couple on <unk>.
Oh, you are flagging, a cta filing in 2023.
But just wondering can you remind us what your regulatory strategy is part of the U S.
You had a pre IMD meeting with the FDA and what do you think are the main areas, where the FDA will probe given the novelty of the eight are editing technology.
Are they looking for off target editing longer GOP talks more bio distribution experiments any any color there would be much appreciated and I have a follow up thanks, Okay. I'll start with the last part and then hand it to Emory because I think she is best suited to answer the question, but I think in light of a number of the.
The news flow within the editing space, particularly editing on DNA I think its really reflective of why we chose RNA editing with a capability.
Reversible he added the transcript and so I think that coupled with the benefits of manufacturing of <unk> are both very important as we think about progressing into our regulatory discussions, but I'll, let <unk> take the regulatory conversation.
And progress with our DLP Tox studies, so we have not yet initiated our interactions with the regulatory authorities and as you've alluded to of course, the outcome of the GOP Tox studies will be instrumental in the discussion of what the regulators.
With one to say, but based on the non guilty.
We have already in hand.
We showed that we have a comes up with a price of oil which makes it useful for continuing development. I think it's also important to go back to that original theme again that what we're going to regulators to have a conversation or RNA editing, we're still talking about it I'll, let get nucleoside approach much in the same way I think about these as heartbeat RNA guidance, rather that will develop.
Things are very analogous to the endogenous mechanism, we're talking about using an RNA therapeutic the silence with RNA say, it's short SA RNA with ego to or slight thing. This is just another oligonucleotide mechanism that we're using now to engage the Dodge.
Hadar.
Very different from other approaches where you are inserting both a protein to edit coupled with guides that then target DNA permanently. So it is it is a different call.
Thanks, that's helpful Paul and MRI.
Just maybe one more question on <unk>.
We have seen Reese.
Recently, <unk> announced that they can get approved on a serum biomarker.
I'm just wondering what was your reaction to that news and maybe how are you thinking about implications for your own program.
I mean, I think like everybody else we.
Anticipating and watching that data very carefully I think it was affirmative to us that indeed, the plasma concentrations of this protein are important I think as we watched that it does open up.
A potential path I think that that's only one size you remember of our program. So.
That aspect is how we think about replacement and helping the lung, but these patients also have potential liver complications and so I think as we said earlier, our clinical part will really be driven to address both so that we can.
Liver aggregate in addition to protein level.
Really giving ourselves a single therapy that comprehensive, but obviously encouraged by that data.
With the realization that the protein we're generating is endogenous protein. So there is a.
We're looking at the measure not just having the protein expressed in having a big infusion.
Infusion remember you gave a protein and then you watch the degradation of that propane will really establishing an increasing base of high levels of patients that we see the patients.
And allowing them when the body it needed to be manufactured that Theyre sympathizing LIBOR wildlife proceeds so interesting to follow and obviously encouraging as we watch.
Yeah, and I would just say that this kind of regulatory precedent.
Great for the field, great for patients and we support any action taken by regulators, but will help expedite therapies to patients with rare diseases.
Great. Thanks for taking our question.
Thank you and thank you.
And one moment for our next question.
And our last question comes from Manny <unk> from Seb Securities. Your line is now open.
Hey, guys. Thanks for squeezing us in just a quick clarification on the DMD program you talked about.
Some are speculating about possibly moving to a larger study is adequately powered to show differentiation versus versus the existing agents.
Exon skipping.
Does that.
Should I interpret that as you guys plan to do a head to head study with superiority endpoints.
And if not how should I interpret that.
Yes. Thank you for the clarification question.
No we're not planning on having we don't have a deal we're not planning on running a head to head study with program.
Celebrated approval I think the key for US is we do know where the buyers have been set from other programs that we want to assure that that study is substantially powered both to deliver on superior dystrophin to those program as well as potential in terms of design.
Support of them reset discussions with regulators rather accelerated approval pathway.
Okay. So you bet you've made in the cost and the kind of a cross trial comparison sort of sense exactly.
Rachel referring.
There is still a significant unmet need in this space.
Okay. Thanks.
Really helpful. Thanks, guys. Thank.
Thank you and thank you.
I will now turn the call back over to Dr. Paul <unk> for closing remarks.
Okay.
Thank you everyone for joining the call. This morning. This is an exciting time for our organization I am grateful that every wave employees for their dedication and unrelenting focus on our mission and on the patients and families. We serve have a great day.
I would now like.
This concludes today's conference call. Thank you for participating you may now disconnect.
The conference will begin shortly to raise your hand during Q&A you can dial star one one.
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