Good morning and welcome to Wave Life Sciences Second Quarter 2020 Conference Call. At this time, all participants are in a listen only mode. As a reminder, this call is being recorded and webcast. I will now turn the call over to Kate Roush, Head of Investor Relations 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 at Herzeway's Q2 2020 operating results. On the call with me today are Doctor. Paul Bono, our President and CEO Doctor. Mike Tanzera, our Chief Medical Officer, Head of Therapeutics Discovery and Development David Gallero, Interim CFO Doctor.
Chandra Varghese, Chief Technology Officer and Doctor. Ken Rhoads, SCP Therapeutics Discovery. Paul and Mike will provide opening remarks after which Dave will discuss our Q2 financial results. This morning, we issued a news release detailing our Q2 results. Please note that this news release is available in the Investors section of our website, www.wavelifesciences.com.
The slide presentation that accompanies this webcast will also be available on our website following this call. 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 a number of risks and uncertainties that could cause our actual results to differ materially from those described in these forward looking statements. The 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 ks the year ended December 31, 2019, and our quarterly report on Form 10 Q for the quarter ended June 30, 2020. We undertake no obligation to update or revise any forward looking statements for any reason.
I'd now like to turn the call over to Paul Bono, President and CEO of Wave Life Sciences. Paul?
Thanks, Kate. Good morning to everyone on the call and thank you for joining us today. I hope you and your families are staying healthy and safe during these challenging times. While we have been impacted by the global COVID-nineteen pandemic, we have continued to advance our clinical and preclinical neurology pipeline. Since the declaration of the pandemic in the United States back in March, we, like others, have been navigating a fluid and unprecedented environment and recent outbreaks around the world reminds us of the uncertainty that is likely to persist well into the second half of this year.
Built from our PRISM platform, our pipeline addresses a wide range of neurology targets and includes 11 programs that span discovery through clinical stage development. Since our last update, both our PRECISION HD clinical trials for patients with Huntington's disease have continued, with patients advancing through the 32 milligram dose cohorts. We now expect to report data from these trials in the Q1 of 2021. We have also continued to successfully advance the work required to submit the clinical trial applications for our SNP III program for Huntington's disease in the Q4 of this year. This 3rd allele selective program incorporates all of our learnings from our SNP 1 and 2 programs as well as the advancements in our platform, including new chemistry.
Importantly, targeting SNP3 increases the number of Huntington's disease patients who could potentially benefit from a new allele selective HD therapeutic. In addition, we are all excited about the progress that we are making to advance our C9orf72 program for ALS and FTD. These are 2 devastating diseases with high unmet need, and our approach has the potential to target one mutation within both diseases. Like SNP-three, this program is designed with new chemistry off the platform and the variant selective strategy was optimized with our PRISM technology. We remain on track to submit a clinical trial application for our C9 program in the Q4 of this year.
Lastly, our collaboration with Takeda for multiple CNS indications, including Alzheimer's disease and Parkinson's disease, continues to yield new PRISM platform, we have a lot of exciting work ongoing, much of which will be discussed during our upcoming analyst and investor research webcast at the end of this month. We continue to generate promising data with our new ADAR mediated RNA editing capability, and we expect to present additional in vivo editing data during this upcoming webcast as well as discuss the opportunity to apply this modality to neurology targets. The ability to edit RNA transcripts significantly expands our targetable landscape and adds to our existing toolbox and modalities for the design of novel RNA therapeutics. We also expect to present ADAR editing data at multiple scientific meetings this fall, including the Elegonucleotide Therapeutics Society, or OTS, Annual Meeting and the Keystone Symposium on RNA Editing and Modifications as well as announce our first RNA ADAR editing program later this year. More broadly, we have implemented novel chemistry enhancements across our pipeline and our next wave of neurology programs, including C9orf72, SNP3 and our RNA editing elevated weight size are all designed with this new chemistry.
We will discuss more details on our new PRISM chemistry platform for the first time during our research webcast. So I'd like to provide a brief update on our operations in this ongoing pandemic. In the Q2, we shifted from responding to the COVID-nineteen restrictions to developing and implementing a robust business continuity plan, enabling us to resume many of our critical on-site activities. We expanded a number of on-site laboratory and manufacturing staff over the past couple of months, while maintaining strict protective measures to ensure their health and safety. To date, more than 60% of our employees are now trained on these protective measures and eligible to work on-site.
The remainder of our team has adapted well to working from home. Overall, everyone at Wave has embraced and excelled in new ways of working, and their flexibility and ingenuity also mirrors what we are seeing among the broad network of researchers and patients who are participating in our studies. Our supply chain has remained intact, ensuring we have sufficient material to drive our preclinical and clinical pipeline forward. In the month of May, Doctor. Ken Rhoads joined our team as Senior Vice President, Therapeutics Discovery, and I'm very excited to have him on board.
Ken comes to Wave with decades of experience discovering and advancing therapeutic candidates in the neurology space. He will help build our pipeline by identifying new targets and therapeutic areas of focus and guiding the research needed to advance candidates to the clinic. You will hear from Ken at our upcoming Analyst and Investor Research webcast. I'd now like to turn the call over to Doctor. Michael Panzara for an update on our clinical and preclinical neurology programs.
Mike?
Thanks, Paul. Today, I will begin with an update on our PRECISION HD1 and PRECISION HD2 clinical trials for the treatment of Huntington's disease. During the COVID-nineteen pandemic, we have remained keenly focused on ensuring the safety of our study teams, patients and investigators. While both clinical trials have continued to advance, clinical trial sites, patients and investigators are facing new or continued restrictions. Progress has continued and we have been successful at identifying the patients needed to complete the 32 milligram cohorts for both studies.
While we are working to enroll these patients as safely and efficiently as possible, we now anticipate being able to share data from all treatment cohorts for both SNP1 and SNP2 studies as well as data from our open label extension trials in the Q1 of 2021. At the beginning of the pandemic and the time of our last update, we were hopeful that we could keep our studies on track because of the prioritization disease modifying studies by health authorities, the infrequent dosing regimen of the precision HD trials and based on the locations of our clinical trial sites. While this has certainly been helpful, the ongoing impact of the coronavirus has been unavoidable. We believe that selectively lowering of mutant Huntington will be critical to achieving long term clinical benefit with any therapeutic in HD, which is why we are expanding our commitment to HD patients with the development of our 3rd allele selective program, SNP-three. Preparations continue towards beginning clinical development with the submission of a clinical trial application or CTA for SNP-three, which is expected to occur in the Q4 of 2020.
We have leveraged the learnings from both our first HD clinical programs in terms of candidate screening, identification and in vivo target validation as well as advancements in our platform when designing our SNP3 compound, which incorporates a novel chemistry advancement from our PRISM platform. Beyond the advances in developing our lead SNP3 compound, over the past several years, we have significantly evolved our methods to identify patients eligible for SNP3 targeting and measurement of fluid biomarkers. When we started this journey, we were told that identifying patients with relevant SNPs through a process called SNP phasing was not possible or too burdensome to be practical for therapeutic development. Since then, we have clearly proved SNP phasing can be accomplished successfully and efficiently. We not only confirm the feasibility of SNP phasing in a prospective observational study, which was recently published in Neurology Genetics, but we have successfully phased and identified more than enough patients appropriate for
our
studies in a timeline to enable enrollment. Taking this approach to the next level, last year we entered into a collaboration with Asurion for the development and commercialization of a potential companion diagnostic for each of our SNP-one and SNP-two programs. Just recently, we expanded this agreement to enable us to use their scalable technology for patient selection in our upcoming clinical trial for SNP3. Our SNP3 program has the potential to also broaden our reach into the HD population. This program addresses a significant portion of the Huntington's disease population on its own as approximately 40% of the HD population has SNP3 and observation we have confirmed using patient samples from our observational study.
Today together with our SNP1 and SNP 2 programs, we have the potential to address up to 80% of the HD population. To remind you why we believe allele selectivity matters, patients with Huntington's disease have approximately half the level of wild type huntingtin protein as healthy patients. And there was a growing understanding around the importance of minimizing any effects on wild type huntingtin given the role it plays in so many critical biological functions, both in the central nervous system and systemically. The supporting data not only continues to grow as does the recognition of this fact in conversations with the HD community. I'm sure those of you who listened in on the virtual Huntington's Disease Society of America or HDSA Congress in June noticed this.
We've also been told that it would never be possible to measure wild type Huntington in the CSF of HD patients. We have turned our focus to this scientific challenge and have made progress. We believe that it is critical to understand the impact of potential treatments on wild type huntingtin and look forward to keeping you updated as we strive to complete this work in time for our data readouts. Now turning focus to our C9orf72 program targeting the most frequent genetic cause of the familial forms of myotrophic lateral sclerosis or ALS and frontotemporal dementia or FTD, 2 neurodegenerative diseases that are both devastating and fatal. C9orf72 genetic mutations are also a strong genetic risk factor for sporadic forms of these diseases.
Our C9R72 targeting compound is designed to selectively and potently silence the hexanucleotide repeat containing transcripts, which drives the formation of toxic RNA and proteins in the central nervous system. Our clinical candidate heads had striking effects in transgenic animal models with potent and durable knockdown of both the repeat containing transcripts and associated dipeptide while leaving C9R72 protein levels relatively intact. The ability to measure dipeptide repeats in the CSF of ALS and FTT patients is a critical biomarker of target engagement as well as other biomarkers in neurodegeneration will afford us the opportunity to rapidly assess for signs of efficacy in the clinic. We plan to include both ALS and FTD patients in our clinical trial with the intention to advance development in both indications. We hope to share more details on this clinical trial design later this year once the details are agreed upon with key stakeholders, including the regulatory authorities.
We continue to expect to submit a CTA in the Q4 of this year. Similar to our SNP-three program, our C9 program takes advantage of innovations from our PRISM platform that have led to a compound with the potential to be highly differentiated from other therapeutic approaches. Doctor. Ken Rhoads will discuss this program in more detail during our upcoming research webcast. So with that, I'll turn it over to Dave Gallero for a review of our financials.
Thanks, Mike. For the Q2 of 2020, Wave reported a net loss of $40,500,000 compared to $41,900,000 for the same period in 2019. Research and development expenses were $31,500,000 in the Q2 of 2020 compared to $41,600,000 for the same period in the prior year. The decrease in research and development expenses in the Q2 was primarily due to decreased external expenses related to suvodirsen due to our December 2019 decision to discontinue the program, partially offset by increased external expenses related to our clinical and preclinical activities, including our HD programs and our C9orf72 program for ALS and FTD. General and administrative expenses were $10,200,000 for the Q2 of 2020 compared to $11,600,000 for the same period in the prior year.
The decrease in general and administrative expenses in the Q2 of 2020 was mainly driven by decreased headcount resulting from the work force reduction implemented in February 2020. WAVE ended the Q2 of 2020 with approximately $94,000,000 in cash and cash equivalents. We expect that our existing cash and cash equivalents, together with expected and committed cash from our existing collaboration, will enable us to fund our operating and capital expenditure requirements into the Q4 of 2021. As a reminder, we do not include potential milestone payments and other uncommitted payments related to our Takeda collaboration in our cash runway. I will now turn the call back over to Paul for closing remarks.
Paul?
Thanks, Dave. Wave has made significant progress this year despite challenges posed by the global pandemic. We continue to advance our neurology pipeline with 2 CTA submissions expected in the 4th quarter and data readouts from our PRECISION HD trials expected in the Q1 next year. In the near term, we have several updates planned to be included in our upcoming research webcast presentation, including C9orf72 preclinical data, in vivo data on our ADAR editing modality and data highlighting our new chemistry enhancements from our platform. The research webcast will be held August 25, and we hope you can tune in.
With that, we'll open up the call for questions. Operator?
Thank you. We have our first question comes from the line of Eun Yang from Jefferies. Your line is open. Please go ahead.
Hi. This is Suji Dao in for you and thanks for taking the question.
So I
have a question about the SNF basing that Paul mentioned. You mentioned that SNP phasing was burdensome earlier. Does it mean that the SNP III clinical trial will use different protocol for SMITH 8 compared to the earlier HC1, HC2 program? And then could you please give a quick enrollment update for the preclinical HD1 program, please? Thank you.
Sure. I'll pass the call to Mike. But just to begin, I think what Mike was saying was that it was presented to us that this would be a challenge. We have been able to do that from the beginning and have continued to provide enhancements to that. I'll let Mike continue to comment on the SNP phasing assay.
Mike?
Yes. Hi. It's exactly what Paul said basically. What I had said is that we were always told when we started this process of identifying patients to have this allele specific approach that there would be a lot of burden and it wasn't really feasible to do in a practical way to do clinical trial. But from the beginning, we've managed to do that.
We've managed to do the study that I mentioned published. We managed to do it in the trials. And the new approach will be working with our partner, Surajin, to develop sort of the next generation, phasing approach that allows it more amenable to a companion diagnostic. So we've moved it along. We've managed to use it efficiently.
And we're looking forward just continuing to do that. And I think the second part of your question was about enrollment. We don't give specifics about enrollment, but what I can say is luckily we've been able to identify patients for the filling of the cohorts, including their SNF phasing and we're just working to get them into the studies.
Okay, great. Thank you.
We have our next question comes from the line of Mani Faroohar from SVB Leerink. Your line is open. Please go ahead.
Hey, guys. Thanks for taking the question. A couple of quick ones. We'll start with the boring finance question. I'm looking at how you guys have fairly rapidly reduced your cash burn in the follow on to the suvodirsen data.
Can you give us a sense of how much of your spend this quarter to last quarter was separation costs regarding that? And how much incremental cost there is to come out? It's a little bit challenging to get to your 4Q 2021 to your 4Q 2021 cash runway guidance for us, and I think we're going to be over modeling it. Moving to clinical trial operations, you guys have seen a series of delays in your Huntington's enrollment. And taking your own published data versus what we know about the epidemiology of Huntington's, you should have been able to enroll, assuming you're somewhere near the speed that Roche Ionis did, essentially the entire study from 1 or 2 large sites given the epidemiological data you've given us regarding the prevalence of SNPs.
So what do you think the reasons are that patients are avoiding your study? Are there changes you can make to your to either your R and D leadership or to your R and D strategy and execution, to avoid future underperformance in enrollment speed and clinical development? Thanks.
Thank you. So we'll take the first question first and then I'll let Dave follow-up with some of the financial guidance. But yes, as you pointed out, there
was the substantial reduction related to FTE costs
as well as operating costs related to finances?
Sure, Paul. So as you saw, we did have reduced cash from Q1 to Q2. In addition, as we reported in our 10 Q this morning, we've also earned approximately $20,000,000 on our ATM since March of this year. And so that's also adding to our extended cash runway.
And the second part of the question, Mike, do you want to talk about the speed of enrollment?
Yes. I mean, I think that there are patients out there. We've managed to identify them. We've actually started to be able to work with the sites to get through this pandemic. But let's just remember, we have this pandemic going on.
And just give me I'll give you a concrete example. I mean, we have you're right.
We have
half of our sites are out there in Australia, just to give you an example. Half many of our sites are in Australia. And we had a whole group of patients scheduled to come into the study beginning last Wednesday. And then Monday, Melbourne announced a sudden lockdown and that just essentially wiped out a whole group of patients that could have gone into the study at that point because of the lock down in Melbourne. And so we had to redirect efforts then to other places.
So I mean, this is a very complex and fluid environment. I'd say the issues of this year are certainly very different than the issues of last year. And I think that we have adapted we made changes last year to improve upon our recruitment this year, but it's not a patient interest issue. It's not a patient interest issue at all. We actually have there's a lot of interest in the community.
And I think that, we're in a we're just right now trying to navigate a very complex situation.
That makes a lot of sense to me. But given the severity and the relentless decline associated with Huntington, unsurprisingly, there is a lot of patient interest in any potential therapy, I get that. But again, what are the structural changes, Paul, that you're thinking of making in your research leadership, headcount, etcetera, to avoid delays like this? For example, one could have chosen for one APAC enrollment site, one could have chosen for example a place like New Zealand, which has much less of a COVID impact or other places where you would have exposed yourself to less of this risk. Given the study has been going on for a couple of years, I would have thought you would have considered that.
So again, how are you thinking of changing research leadership strategy and personnel if necessary?
Yes. So I think the key is prior to this, to your point on New Zealand, we only knew about that New Zealand was less impacted more recently. So getting sites up and running during a pandemic and shifting that wasn't there.
I mean,
I think we had the diversity of clinical trial sites that earlier this year had things on a positive trajectory. I think to Mike's point, we did make changes both in our operating relationship internally with our global CRO network. We enhanced our clinical development team, adding additional physicians more recently, to again continue to focus on delivering and executing on the study. As Mike said, the patients are identified. So this isn't an identification issue.
It's now just closing it out and hence that shift.
Okay. Thanks for taking the questions, guys. What's that? I said thanks for taking my questions.
Thank you, Madam.
We have our next question comes from the line of Salim Syed from Mizuho. Your line is open. Please go ahead.
Hey, Paul. Hey, Mike. Hey, Dave. Thanks for all the color, guys. Just a few from me, if that's okay, all on the Huntington's program.
So Paul or Mike, could you maybe just give us some color how you guys are thinking about the bogey here, given Roche Hyannis is 60% knockdown on both the mutant and the wild type. What do you think you need to have if you're just knocking down the mutant in order to be successful here in the upcoming data set? Number 2 is when we looked at the pooled, the data from the that you disclosed in December, which included the 16 milligram, on the 95% confidence interval, you went up as high as 25% knockdown on the mutant Huntington. And I'm just wondering here if there's anything that suggests that the 16 milligram is actually hitting close to the 25% than something lower than that, whether it was the 12% or closer to the 0 on the lower end of the confidence interval? And then lastly, if you just give us some color on any data that you may be getting in the blinded aggregated setting, if you have access to NFL data perhaps or if there's anything you're looking at there in the blinded aggregated data?
Thank you.
I'll pass the call on to Mike to answer the question specifically, but it's an interesting transition over because I think we are excited to have continued OLE data in that Q1 as well to review at the time we had the 32 milligram data. So in aggregate, a lot of data to evaluate. Mike, do you want to speak to the numbers?
Well, yes. So I mean, I think that, in terms of as you all know, I think that what is a meaningful reduction, in mutant Huntington is really not firmly been established. I mean, if you think about what you mentioned the 40% to 60%, knockdown that's often quoted, the actual study that's being conducted is essentially if you are at HD meetings, the last HD meeting really where anybody could attend was the CHDI meeting in Palm Springs. The target of 20%, 25% was being spoken about, essentially by Roche in terms of what they felt was minimum meaningful when you're considering a knockdown that includes both mutant and Huntington. So that's the number that's actually been out there, but you hear people talking about as meaningful.
So we would think that if it's meaningful in the setting of non specific knock down, certainly that's going to be meaningful in the setting of selective knock down. So but the true answer to that until you have clinical effect and see how that all translates, it's really not clear. So we're obviously we said many times that we were dissatisfied with the interim analysis that we saw. We wanted to go higher. We have gone higher.
And now that's we're looking forward to seeing that data. In terms of ongoing in terms of ongoing assessments of biomarkers and those types of things, we are the assay that type of stuff is very tightly held and restricted and we share safety data with our safety monitoring committee that is unblinded, where they go and they after each cohort as we've talked about, talk about dose escalation. We review blinded information, but we do not review blinded biomarker information. That is not a standard review process at WAVE.
Got it. Thanks, Mike. Mike, I don't know if maybe I missed it. The on the 16 meg, can you maybe just opine on that a little bit?
Again, we've been clear that, we've shared that interim analysis, which was pooled data, the pooled active cohorts versus the pooled placebo cohorts. We've never really gotten into the individual knockdown of each cohort separately. That's all going to be part of the data readout in the Q1.
Okay.
Thanks so much.
Interesting, it's early to single data as well as OLE data too. So we'll have more opportunities for continued dosing. So more to come.
Thanks, Paul.
We have our next question comes from the line of Paul Matteis from Stifel. Your line is open. Please go ahead.
Hey, thanks for taking the question. This is Alex on for Paul. Just I guess I'm curious for your next clinical program SNP3 and the C9orf programs. You mentioned the CTA filing in 4Q. Just wondering if you could give us an idea of when you think you could start dosing, if that's all going to be ex U.
S. Or if you're thinking about filing IND sometime soon as well? And then on your AD and PD programs, curious if you could give us any more color on the source of targets that you're looking at? Thanks.
That's great. As it relates to C9 and SNP3 updates regarding the CTA, Mike, do you want to comment on?
Sure. Sure. I mean, typically, once you get your CTA in, obviously, it depends upon when you get your sites up and everything. But generally, we like to think about a quarter after your CTA is and you begin to be able to dose. So that's about what we've been finding.
Of course, that was certainly and that's typically when there's no COVID going on. So we'll have to see how that goes. But we're going to be targeting countries where we can do it despite COVID. And so, which again is a global we're going to take this globally and our intention to get this off the ground inside and outside of the U. S.
As it relates to the ADPD and other targets from Takeda, unfortunately, we are not at liberty to disclose the confidential targets as part of the collaboration. But as they make progress, we are working with them to be able to share more around those programs. But the team continues to push forward. And as we've said, we've been able to implement using our new chemistry similar to those that we're using for SNP3 and C9 in those programs as well. And we're excited to provide that update on what's happening on the research side of WAVE during our upcoming research day.
Thanks. And I guess one quick follow-up there. Could you give us a quick update on the ophthalmology program? I don't know if I saw it in the press release, just curious where that
stands? Thanks. Yes. So as it relates to the ophthalmology, we had done a lot of work looking at target engagement both in the back of the eye and the retina around USH 2A and Rho P23H. We haven't provided additional updates related to those programs as we look at the collaborative path forward to advance those programs.
Great. Thank you.
We have our next question comes from the line of Yaron River from Cowen. Your line is open. Please go ahead. Once again, Yaron Werber, your line is open. Please go ahead.
Hi, guys. This is Brendan on for Yaron. Thanks for taking the questions. Just a couple of quick ones from us, mostly on HD and kind of different dosing cohorts here. So you mentioned that the Q1 readout will include data through the 32 mg, but you've been considering maybe higher additional doses.
So I guess, first, I was hoping to see, like, what can we expect from the readout in Q1 in terms of time after treatment? I guess, namely how far out after the treatment will the readout include at that point? And then also how do you think we should think about the timeline for these programs? Would you potentially wait to proceed to pivotal until additional higher dose cohorts are fully enrolled in readout? And then I have one follow-up, if that's all right.
Thanks.
Mike, would you like to take the HSD question?
Yes. So again, you would expect at the time of the readout in the Q1, you're going to get the as you remember the design, the whole full 4 doses of the 32 milligram dose level, plus basically all the lower dose cohorts. So that will all be part of that. It will include the key biomarkers we've talked about, mutant. It will include we're striving to get our assessment of wild type done by then and actually you'll have clinical and safety and those types of things.
At that point, after we're done with the 32 milligram, we're also going to understand at that point, you'll understand whether we are going to a higher dose and we'll have all of that able to discuss that with you. In addition, you'll have the open label extension study data SNP 1 and SNP 2, whatever we have after a certain number of doses, similar, to what we're dosing in the PRECISION HD. So you'll have longer term dosing than you'll have with Precision in some patients with similar dosing. So it's a fairly large data set that we'll be sharing in that Q1 and with the hope of again sharing with all of you our thinking about what the next steps would be. In terms of the steps towards a pivotal, that will be driven by the degree of knockdown that we see across all the full cohorts.
Remember the interim was only a partial cohort in that not everybody had gotten out to day 140. So once we look at all of that, once we look at the OLE, once we look at the 32 milligram knockdown, that'll put us in a bit better position to determine the next steps towards our pivotal study, which we would anticipate given where we are that we should be able to begin those steps once we see that 32 milligram knockdown if that level is at a level that we feel is meaningful. So we're already beginning the thinking on how we do that.
Okay, great. Thanks very much. And just really quickly, I was also hoping, as you kind of mentioned, you guys are working to optimize the wild type detection asset. I was hoping that we can maybe get a little bit more color on kind of where that stands and maybe what's just left to optimize and how you might be and if you are looking to implement it differently than you have previously or kind of just where that is at this point? Thanks.
No, I'll definitely hand this to Mike. It's an exciting amount of work on the research side to answer a really important question for the field. Mike, would you like to?
Yes. Thanks for that. So as we discussed at the interim analysis, the mutant assay is an assay that is similar to whatever others are using to measure mutant. And at that time, we also discussed how we looked at the total Huntington in CSF samples. But we also said at the time that there were pretty significant limitations to that in terms of how you look at the mutant versus the total and the difficulties measuring the wild type because it's an indirect measurement.
Measuring the wild type portion of that total pool. Measuring the wild type portion of that total pool. This is something that we've been spending a lot of time thinking about. We actually are looking on implementation of that and we're striving that when we reveal the data in the first quarter, we'll have an assessment using a new approach that will allow people to understand whether what is being measured in that total assay, what piece of that is wild type, what piece of that is mutant and then make that our intention is to sort of work with the community to make that a little bit more widely available should we be successful. Because we think this is an important thing that needs to be looked at and we don't think this is something that we alone should be using.
We think this is something that's important. And community that the partners we're working with are quite excited about the approach.
Yes, it's great. I mean moving away from this kind of dynamic range and ratio to kind of more concrete numbers is obviously the direction that we said at the beginning we would move to and the team has been working extraordinarily hard to advance it. So we're excited to keep pushing that forward.
Okay, great. Thanks guys.
Thank you.
We have our last question comes from the line of Eun Yang from Jefferies. Your line is open. Please go ahead.
Hi. This is Suji again. I have a quick question on the OLE data that's expected in the Q1 next year. Are the patients in the OLE currently receiving this drug
right now?
And then what do you expect to show?
And then the second question I had was for the SNP 3 Phase 1 trial that you're going to start. Is that design going to be similar to the SNP 1 and 2 2 program? Or do you expect to start that study in healthy volunteers first? Thank you.
Yes. So I'll pass the question to Mike, but the Phase III study in SYP-three will be in patients. But I'll let Mike continue to answer questions around the OLE and the SYP-three. Mike?
Yes. So the OLE, the way the doses are so everybody in the open label extension study is getting active drug. And the way the study is designed is that once a cohort is shown to be safe in the precision HD study. Patients are migrated up to whatever the highest dose the highest safe dose has been in the precision HD study. So patients are migrating up to the highest doses tested in that study.
And what we'll see in the Q1 are the basically the safety and the biomarker results of those patients over the duration that they've been exposed. In terms of SNP3 trial versus similarities to SNP1 and SNP2, certainly there'll be a lot of similarities, but they've also had a lot of learnings from the precision HD1 and HD2 studies that will be incorporated into the precision HD, what would likely be the precision HD3 study. And this study, in terms of phasing, as I mentioned earlier, we'll be using the new technology for SNP phasing through our partnership with Asurion.
We have another question from the line of vejid Chappellpathai from A. C. Wainwright. Your line is open. Please go ahead.
Hi, guys. Good morning. Thanks for the data updates and I'm sorry about the delays. So I wanted to ask and by the way, this is Aaron on for Debjit. I wanted to ask, are the newly identified patients for the precision studies all at the same site or are they geographically diverse?
And do you have more patients identified than are needed to fill out the cohort or is it exactly the number of patients that are required? And then finally, if your patient misses an injection or a follow-up, are they totally excluded or is there and a new patient is needed or is there how much flexibility is there?
Mike, would you like to take those?
Yes. I mean, all I will say is that we have multiple sites with patients in multiple backups. So there's no exact here because we're starting to think about additional cohorts and additional patients. So yes, we have more patients that have been phased that we need and then more sites than we need to do it.
Okay, great. Thank you. And then a quick follow-up. So with dose escalation beyond 32 milligrams be dependent on how full of a data set do you need to justify escalation beyond 32 milligrams? Do you need the full patient data set at 32 milligrams and out to 192 days?
Or is there some sort of partial amount that you're using?
No, we've never waited for the full 196 days. It is actually to be able to make that decision. As you recall, going from 8 to 16 and 16 to 32 was made before we had any biomarker data or had complete safety data, which based upon the safety of the single dose cohort and the review of an independent safety committee. So we haven't waited to completion before making those decisions.
Okay, great. Thanks guys. Thank you.
There are no questions at this time. I'll now turn the call back over to Doctor. Paul Boulno. Thanks,
everyone for joining the call this morning to review our Q2 update. And thanks to our employees for their hard work and commitment to patients. We look forward to speaking to you again soon at our upcoming research webcast. Have a nice day. Thank you.
Ladies and gentlemen, that does conclude our conference for today.