Q1 2020 Earnings Call
At this time, all participants in listen only mode.
Operator: All participants are in a listen-only mode and the Webcast. I'll now turn the call over to Kate Rausch, 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 and review WAVE's first quarter 2020 operating results. On the call with me here today are Dr. Paul Bolno, our President and CEO, David Guerrero, Interim CFO, Dr. Mike Panzera, our Chief Medical Officer, and Dr. Chandra Vargeese, Senior Vice President, Drug Discovery. This morning, we issued a news release detailing our first quarter results. Please note that this news release is available in the Investor section of our website, www.waveslifesciences.com. The slide presentation that accompanies this webcast will also be available on our website following this call.
As a reminder, this call is being recorded and with that I'll now turn the call over to Kate wrong.
It doesn't investor Relations at Life Sciences. Please go ahead.
Thank you operator good morning.
Thank you for joining us today to discuss our recent business progress.
<unk> first quarter 2020 operating results.
On the call with meeting here today are Dr., Paul Bono, our president and CEO.
The guy there in terms CFO Dr., Mike and there are chief Medical Officer, Dr., Charles Johndroe Marquee Senior Vice President drug discovery.
This morning, we issued a news release detailing our first quarter results. Please note that this news release is available in the Investor section of our website Www Dot Weve life Sciences Dot com.
The five 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.
Operator: 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-K for the year ended December 31st, 2019, and our quarterly report on Form 10-Q for the quarter ended March 31st, 2020. 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 Bolno, President and CEO of WAVE Life Sciences. Paul?
These statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described these forward looking statements.
Doctors that could cause actual results to differ are discussed in the press release issued today and then are actually see filings, including our annual report on form 10-K for the year ended December 31st 2019, and our quarterly report on form 10-Q quarter ended March 31st 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 the to Pabon, L., President and CEO of with Life Sciences Paul.
Kate Rausch: Thanks, Kate. Good morning, and thank you for joining us today. I'll start today's call with a few introductory remarks. Next, Dave Galliero will discuss our financial results. Mike Panzaro will provide an update on the progress of our neurology pipeline. And finally, Chandra Vargeese will provide an exciting update on our emerging RNA editing platform capabilities. During the first quarter, I am proud that our team's commitment, along with the close partnership and support of our clinical trial site, allowed both PrecisionHD clinical trials to continue despite the challenges presented by the COVID-19 pandemic. We have also continued to progress work that will allow us to submit clinical trial applications for two additional neurology programs in the second half of this year, our SNP3 program for Huntington's disease and our C9ORF72 And today, we are announcing our first in vivo RNA editing data, delivering on one of our key corporate goals for 2020. Like everyone else, we are operating in the midst of the COVID-19 pandemic, an evolving and dynamic situation. While the unknown duration of the pandemic makes it difficult to predict or plan for potential longer-term impacts on our business.
Thank you good morning, and thank you for joining us today.
I'll start todays call with a few introductory remarks, there. They are will discuss our financial results, Mike and there will provide an update on the progress of our neurology pipeline.
Finally, counterparties will provide an exciting update on our emerging Arnie editing platform capability.
During the first quarter I'm proud that our team committed along with the close partnership in support of our clinical trial site has allowed both precision HD clinical trials to continue despite the challenges presented by the Kobin 19.
We've also continued to progress work that will allow us to submit clinical trial application for two additional neurology programs in the second half of this year.
Our snipped three program for Huntingtons disease, and Arsene onboard to 72 program for Alex in front of temporal dementia.
And today, we're announcing our first invivo ornate editing data delivering on one of our key corporate goals for 2020.
Like everyone else, we're operating in the mid the coven, 19, pandemic and evolving and dynamic situation.
While the unknown duration of depends on it makes it difficult to predict her plans for potential longer term impacts to our business, we have leaned into the challenge and maintained to consistently proactive approach.
Paul B. Bolno: We have leaned into the challenge and maintained a consistently proactive approach. We quickly established an internal COVID-19 response team that includes expertise from across all areas of our organization. This team meets on a regular basis and has facilitated our ongoing operations by focusing on three priorities. We are safeguarding the health and well-being of our employees, as well as patients and caregivers. The majority of our team has been working from home since mid-March, and we have implemented strict protective measures for the small number of employees whose work requires them to be on-site in our labs and manufacturing facilities.
We quickly established and internal Tobin 19 response team and include expertise from across all areas of organization.
This team meets on a regular basis and has facilitated our ongoing operations by focusing on three priority.
First.
We are safe guarding the health and wellbeing of our employees as well as patients caregivers explanation.
Majority of our team has been working from home in March and Weve implemented strict protective measures for the small number of employees, whose work requires them on site in our lab and manufacturing facility.
Second.
Paul B. Bolno: Second, we are focused on advancing our programs while adapting to new challenges in the current environment. Importantly, even before the pandemic, we had been actively investing in our manufacturing facility, managing our supply chain, vendors, and inventory levels to limit the potential for disruption. As a result, we have sufficient materials stored locally to enable us to continue to manufacture our products for at least 12 months.
We are focused on advancing our programs, while adapting to new challenges in the current environment.
Importantly, even before the pandemic, we had been actively investing in our manufacturing facility managing our supply chain, there's an inventory levels limits the potential for disruption.
As a result, we have sufficient material store locally to enable us to continue to manufacture products for at least 12 month.
My Kids Arab we'll share more details on how we've been able to maintain momentum with our two global clinical trial.
Paul B. Bolno: Mike Panzera will share more details on how we've been able to maintain momentum with our two global clinical trials. Finally, we are working to create opportunities in this new environment. For example, we have found ways to creatively engage with our stakeholders, including virtual meetings with researchers, clinicians, advocates, and broader patient communities, all while maintaining social distance.
Finally, we're working to create opportunities in this new environment for example.
We have found ways to creatively engage with our stakeholders, including virtual meetings with researchers clinicians advocate and broader patient community, all while maintaining social distances.
Additionally, we are working to get ahead of future opportunities and challenges, including planning for an expansion of on site activities in our lab manufacturing suite as well as remaining prepared for a second wave run a virus outbreak.
Paul B. Bolno: Additionally, we are working to get ahead of future opportunities and challenges, including planning for an expansion of on-site activities in our labs and manufacturing suite, as well as remaining prepared for a second wave of coronavirus outbreaks. For patients and families with devastating neurodegenerative diseases, progress cannot come fast enough. Knowing that they are counting on us has kept us going and continues to motivate everyone at WAVE.
For patients and families with devastating neurodegenerative diseases progress cannot come fast enough.
Knowing that they are counting on US is kept is going and continues to motivate everyone at wave.
Our innovative pipeline is focused on neurology.
Paul B. Bolno: Our innovative pipeline is focused on neurology. Notably, we continue to make excellent progress on the multiple preclinical CNS programs we are advancing with our partner Takeda. In the first quarter, we achieved target validation in vivo with a lead compound for a second program, and we expect to achieve target validation for a third program later in 2020. Kate is excited about the progress we are making, and we look forward to sharing further updates on these programs. Beyond neurology, our platform capabilities offer the opportunity for us to help more patients, including those living with genetically defined diseases of the liver and eye. To that end, we continue exploring opportunities to advance our two preclinical ophthalmology programs, USH2A for Usher syndrome type 2A and Rho P23H for retinitis pigmentosa, both of which have demonstrated positive preclinical data.
Notably we continued to make excellent progress on the multiple preclinical CNS programs, we are advancing with our partner Takeda.
In the first quarter, we achieve target validation in vivo with a lead compound for a second program and we expect to achieve target validation for a third program later in 2020.
Takeda is excited about the progress, we're making and we look forward to sharing further updates on these programs.
Beyond neurology, our platform capabilities offer the opportunity for us to help more patients, including those living with genetically defined diseases of the liver and I.
To that and we continue exploring opportunities to advance our two preclinical ophthalmology program to wafer ushers syndrome type two way and rope. He 23 age for retinitis Pigmentosa, both of which have demonstrated positive preclinical data.
We didn't have haddock diseases, while the collaboration with Pfizer concluded or later this month, we are today announcing our initial eat our mediated arnie editing data in the liver of non human primates.
And we expect our initial these target for eight are to be focusing a panic disease.
We are building, our harney editing technology, the platform capability and see opportunity to imply that other therapeutic areas over time or in collaboration with potential partners.
Paul B. Bolno: Within hepatic diseases, while the collaboration with Pfizer concluded earlier this month, we are today announcing our initial ADAR-mediated RNA editing data in the liver of non-human primates, and we expect our initial disease target for ADAR to be focused on hepatic diseases. We are building our RNA editing technology as a platform capability and see opportunity to apply this to other therapeutic areas over time or in collaboration with potential partners. I will now turn this call over to Dave Galliero for a review of our financials.
I will now turn this call over to Dave here for a review of our financial Dave.
Thanks, Paul.
First quarter 2020, we reported a net loss of $47.5 million compared to $44.2 million for the same period in 2019.
Research and development expenses were $41.2 million in the first quarter 2020, compared to $40.1 million for the same period than the prior year.
The increase in research and development expenses in the first quarter was primarily due to increased external expenses related to our clinical and preclinical activities.
Moving our HD programs, we don't see nine or 72 programs and less than a CD.
And separation costs associated with the workforce reductions implemented in February 2020.
Partially offset by decrease six rental expenses related to our DMD programs due to our December 2019 decision to discontinue the Super Justin program and to seize development of our other DMD program.
Dave Galliero: Thanks, Paul. For the first quarter of 2020, we reported a net loss of $47.5 million compared to $44.2 million for the same period in 2019. Research and development expenses were $41.2 million in the first quarter of 2020, compared to $40.1 million for the same period in the prior year. The increase in research and development expenses in the first quarter was primarily due to increased external expenses related to our clinical and preclinical activities, including our HD programs and our C9ORF72 program for ALS and FTD, and separation costs associated with the workforce reduction implemented in February 2020. Partially offset by decreased external expenses related to our DMD programs due to our December 2019 decision to discontinue the Subra Joslin program and to cease development of our other DMD programs.
General and administrative expenses were $13 million for the first quarter 2020, compared to $10.9 million for this and other than the prior year.
The increase in general and administrative expenses in the first quarter was mainly driven by separation costs associated with the workforce reductions implemented in February 20 Twond.
We ended the first quarter 2020, with approximately $121 million in cash and cash equivalents, including $20 million in research support funding receipt from Takeda and the first quarter under our collaboration.
As a reminder, we expect to begin to realize the results of our overall cost reduction efforts, including our February 2020 workforce reduction in the second quarter 2020.
We expect that our existing cash and cash equivalents together with expected didn't committed cash from our existing collaboration will enable us to fund our operating and capital expenditure requirements into the third quarter 2021.
I'll now turn call over to Dr., Michael Bender, Our Chief Medical Officer, who will provide an update on neurology programs Mike.
Thanks, Dave.
Today, we'll be giving an update on where things stand with our clinical development programs. However, I'd like to begin with yet another publication that highlights the importance of what we're doing and huntingtons disease by working to develop the leal selective treatments.
Dave Galliero: General and administrative expenses were $13 million for the first quarter of 2020, compared to $10.9 million for the same period in the prior year. The increase in general and administrative expenses in the first quarter was mainly driven by separation costs associated with the workforce reduction implemented in February 2020. We ended the first quarter of 2020 with approximately $121 million in cash and cash equivalents, including $20 million in research support funding received from Takeda in the first quarter under our collaboration. As a reminder, we expect to begin to realize the results of our overall cost reduction efforts, including our February 2020 workforce reduction, in the second quarter of 2020. 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 third quarter of 2021. I will now turn the call over to Dr. Michael Banthera, our Chief Medical Officer, who will provide an update on our neurology programs.
This recent publication by plus get all the nature adds to a growing body of evidence that preserving wild type Huntington will be essential to impacting clinical outcomes at this disease demonstrating that Huntington is that the center of the regeneration transcriptome.
Playing in the central role and neuroplasticity after injury.
Turning to an update on our precision HD clinical trials since the started the pandemic in the first quarter, we have been keenly focused on ensuring the safety of our study teams patients and investigators while continuing to advance our trials and mitigate future potential risks.
As you know this is a dynamic situation.
We've been monitoring Vigilantly and proactively doing what we can to enable our patients to continue treatment, while ensuring the data quality required to enable definitive results.
As a reminder, precision 81, and precision HD, two or evaluating the safety Tolerability pharmacokinetics and pharmacodynamics of single and multiple doses W. Ve, one too low one on one and W. E. One two or one or two in adult patients with early manifest each D who cares.
Snip, one or snipped two respectively.
W. Ve, one to one or one and W. ve, one two or one or two are the first and only compounds in clinical development designed to selectively target. The mute new we'll have to Huntington gene transcript, while leaving the wild type Huntington relatively intact.
Michael Linden: Thanks Dave. Today, we'll be giving an update on where things stand with our clinical development program. However, I'd like to begin with yet another publication that highlights the importance of what we are doing in Huntington's disease by working to develop allele-selective treatment. This recent publication by Poplusky et al in Nature adds to a growing body of evidence that preserving wild-type Huntington will be essential to impacting clinical outcomes, demonstrating that Huntington is at the center of the regeneration transcriptome, playing an essential role in neuroplasticity after injury.
Both studies has continued to progress we have benefited from the prioritization of disease modifying studies by health authorities, the infrequent dosing regimen and the global distribution of our clinical trial sites.
Most importantly, the commitment of our patience and investigators has remained steadfast speaking to the importance of the work we're doing in the high unmet needs those suffering from Huntingtons disease.
Michael Linden: Turning to an update on our PrecisionHD clinical trials, since the start of the pandemic in the first quarter, we have been keenly focused on ensuring the safety of our study teams, patients, and investigators while continuing to advance our trials and mitigate future potential risks. As you know, this is a dynamic situation that we have been monitoring vigilantly and proactively, doing what we can to enable our patients to continue treatment while ensuring the data quality required to enable definitive results. As a reminder, PrecisionHD1 and PrecisionHD2 are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of WVE120101 and WVE120102 in adult patients with early manifest HD who carry SNP1 or SNP2, respectively. WVE-12101 and WVE-120102 are the first and only compounds in clinical development designed to selectively target the mutant allele of the Huntington gene transcript while leaving the wild-type Huntington relatively intact.
As all health care systems are currently under significant stress some precision Ht trial sites have been impacted by the pandemic.
However, I'm pleased to report that we currently remain on track to liver data from both the precision SG trials in the second half of the year.
Specifically for precision HD too we expect to report data from the 32 milligram cohort, which was initiated in January as well as data from each of the previous individual cohorts.
Similarly for precision AG, one trial, which remains blinded we initiated the 32 milligram cohort in March as planned and we expect to report results from the individual core Hertz and this trial, including 32 milligram in the second half also.
Of course should global restrictions continue for worsen the ability do out valuate patience is plans in each of these studies has the potential be impacted.
For both precision has two trials, we continue to assess the ability to advance to a next higher dose, which will be determined by single dose safety results of 32 milligram cohort and available pharmacokinetic data as well as our pre existing preclinical.
Packages.
Our third Leo selective HD program arsenic three program continues to advance towards clinical development.
Michael Linden: Both studies have continued to progress. We have benefited from the prioritization of disease-modifying studies by health authorities, the infrequent dosing regimen, and the global distribution of our clinical trial sites. Most importantly, the commitment of our patients and investigators has remained steadfast, speaking to the importance of the work we are doing and the high, unmet needs of those suffering from Huntington's. However, as all health care systems are currently under significant stress, some PrecisionHD trial sites have been impacted by the pandemic.
As a quick reminder, approximately 40% of the achieve population has three and with overlap up to 80% of the age depopulation contains at least one of snip one step to endorse net three.
One of three program is designed with novel with a novel chemistry advancement from our Prism platform and we were particularly excited by the Nvvault data we have seen to date in preclinical models, we expect to initiate clinical development, meaning submission of a clinical trial application or C.T.A. fortunate three in the second half of this year.
Turning now to our see nine or 72 program, which is also approaching the clinic.
Michael Linden: However, I am pleased to report that we currently remain on track to deliver data from both the PRECISION-HG trials in the second half of the year. Specifically, for PrecisionHD2, we expect to report data from the 32 mg cohort, which was initiated in January, as well as data from each of the previous individual cohorts. Similarly, for the Precision HD1 trial, which remains blinded, we initiated the 32 milligram cohort in March as planned, and we expect to report results from the individual cohorts in this trial, including 32 milligrams in the second half also. Of course, should global restrictions continue or worsen, the ability to evaluate patients' plans in each of these studies has the potential to be impactful.
This program aims to address amyotrophic lateral sclerosis, and frontotemporal dementia caused by mutations in that see nine or 72 gene.
This is the most common cause a familiar lay a lesson FTD and the strong genetic risk factor for the sporadic forms of disease.
This program is designed to selectively in Potently silenced the transcripts that contain the hickson nucleotide repeat which drives the formation of toxic Arnie and I'm normal proteins and bring tissue.
Similar to arsenic three pro <unk> program are seen nine program also uses new chemistry off of our platform and we have demonstrated proof of principle and transgenic animal models.
And these in vivo studies, we've shown <unk> knockdown of both the repeat exchange transcripts and the associated that peptides, while avoiding a reduction in wild type protein.
We expect to submit a C.T.J. for seen or North Sea nine or 72 program also in the second half for the year.
Michael Linden: For both precision HD trials, we continue to assess the ability to advance to the next higher dose, which will be determined by single-dose safety results in the 32-milligram cohort and available pharmacokinetic data, as well as our pre-existing preclinical package. Meanwhile, our third allele-selective HD program, our SNP3 program, continues to advance towards clinical development. As a quick reminder, approximately 40% of the HD population has SNP3, and with overlap, up to 80% of the HD population contains at least one of SNP1, SNP2, and or SNP3.
And with that I'll handover the call to Chandra Varghese.
Thank you Mike Good morning, everyone today, I'll provide an exciting obtain formal owning editing.
I mean anything is that yes, modality to come off as well as impactful.
Yes significant capabilities to the advancement.
We have made in all of these slicing and handling.
Actually down although modalities by focusing on omni heat shield additional flow Johnson's sandwell distinct benefiting all the gene editing.
Including the ability to use individuals proteins.
Michael Linden: Our SNP3 program is designed with a novel chemistry advancement from our PRISM platform, and we are particularly excited by the in vivo data we have seen to date in preclinical models. We expect to initiate clinical development, meaning submission of a clinical trial application or CTA, for SNP3 in the second half of this year. Turning now to our C9ORF72 program, which is also approaching... This program aims to address amyotrophic lateral sclerosis and frontotemporal dementia caused by mutations in the C9R72 gene. This is the most common cause of familial ALS and FTD and a strong genetic risk factor for sporadic deformity. This program is designed to selectively and potently silence the transcripts that contain the hexonucleotide repeat, which drives the formation of toxic RNA and abnormal proteins in brain tissue.
Now I'll agree pad capable building and replaceable. Thanks.
Thanks, Doug protein be hardness, and you know all agonising dnbi acting on it I mean needs to effecting aim to gene editing, providing ample opportunity across a wide variety of diseases.
Do you see on that right now.
The last Cooper Zeon other mediums amenable to aided G.
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Mitch networks and so.
So for this tells me and I didn't think against potential clinical chunky.
You never know Oh.
Ill pathogenic humans nims in.
Yes, I mean publish that.
Could be corrected mid 80 gene editing.
Beyond offering a bad because overall gene editing.
Michael Linden: Similar to our SNP3 program, our C9 program also uses new chemistry off of our platform, and we have demonstrated proof-of-principle in transgenic animal models. In these in vivo studies, we showed potent knockdown of both the repeat-containing transcripts and the associated addict peptides while avoiding a reduction in wild-type. And with that, I'll hand over the call to Chandra Vargeese.
Dave Technology, Yes, yes, among other plans in automating.
And that you don't mediated editing technology has emerged reactively recently.
Beyond the whole spread of the mopping this technology for potential could appear to use.
It was and Doug will be.
Mitch Awards, let's call it mid agenda to be excited you must toping.
But yes casting team all came mainly a bunch.
Under the latent potential all for good effect.
These proteins.
All again nucleotides are totally chemically modified.
Chandra Vargeese: Thank you, Mike. Good morning, everyone.
The increase is tablet.
Chandra Vargeese: Today, I'll provide an exciting update on our RNA editing platform. RNA editing is the newest modality to come off of our PRISM platform, adding a significant capability to the advancements that we have made in RNA splicing and silencing. Ashley Durham, another medallion, by focusing on RNA. We feel that this approach offers several distinct benefits over gene editing, including the ability to use endogenous proteins. Ease of delivery, titratable dosing, and reversible effort.
And duration of activity.
Typically there's plenty visas are fewer.
Importantly, our ability to precisely control.
The backbone.
Yes.
Maximize they and dog eat dog activity.
Lastly, we use a simplified deleverage tribe.
Meaning no. He may be best days are no nano particles.
It had a beautiful investigation.
Are you, saying simple galnac conjugated oligonucleotides.
Hepatic targets.
Well.
We also continued to build the army anything capability all four prism platform.
Chandra Vargeese: The endogenous protein we harness, ADAR, or adenosine deaminases acting on RNA, leads to effective A to G editing, providing ample opportunity across a wide variety of diseases. As you see on the right, The Landscape of Genetic Variants Amenable to ADGs, Everything is very long, in the tens of thousands, which represents a rich source for discovery and identification of potential clinical trials.
<unk> expenses automate technology could be used across maybe this kind of beauty indications.
I actually had previously described.
We have achieved very efficient automate editing beach or they are oligonucleotides.
Across a variety of cell lines, including non human primate I'm human primary qualified.
As shown here in this slide we have observed potent durable dose dependent on everything we see chemically distinct Stevie appeal oligonucleotides, why galnac mediated upkeep.
Chandra Vargeese: It has been published that nearly half could be corrected with A to G editing. Beyond Offering Advantages Over Gene Editing, WAVE's technology is unique among other players in RMA education.
Hi, I can't there's in vitro our next tasks what's to determine whether these the in vitro results, Pennsylvania.
Chandra Vargeese: Endogenous ADAR-mediated editing technology has emerged relatively recently, and we are at the forefront of developing this technology for potential therapeutic use. Bios, Endogenous Protein, which avoids the risk of immunogenicity from exogenous proteins, such as Cas13 or Chimeric ADOT, and the related potential off-target effects of these proteins. Our oligonucleotides are fully chemically modified, which increases stability and duration of activity. And typically, there are 30 bases or fewer.
Two in vivo system, which they did.
The first time.
Yes.
Indeed on anything results in non human primate.
Well, that's Debbie vetoes six non human primates.
Three groups.
Subcutaneously once a day coinciding.
Clean chemically distinct arnie.
The oligonucleotides.
The coke liver biopsy samples.
Based on to these posts lots of those you boldly that sequence off the targets targeted side.
Chandra Vargeese: Importantly, our ability to precisely control the chirality of the backbone enables us to maximize the endogenous ADAR activity. Finally, we use a simplified delivery strategy. Meaning, no AAV vectors or nanoparticles. In our initial therapeutic investigation, we are using simple galvanic conjugated oligonucleotides for a hepatic target. However, we have continued to build the RNA editing capability of our PRISM platform and expect this RNA technology could be used across various therapeutic indications. As we have previously described, we have achieved very efficient RNA editing in vitro with our oligonucleotide, including non-human primates and human primary parasites. As shown here in this slide, we have observed potent and durable dose-dependent RNA editing with three chemically distinct stereopure oligonucleotides via GalNac-mediated uptake. Having achieved this in vitro, our next task was to determine whether these in vitro results translate, which they did.
As you can see on the chart on the left.
He detected.
50% anything as compared to baseline indeed.
And then right you can see as Sanger sequencing thought for the targeted oligonucleotides showing key editing had that target sites.
Well. This is an ongoing proof of concept study im very excited by decent solved.
Onto our knowledge. These are the first successful automated editing and bond human planning.
Importantly, we have achieved sniffles, using our dog eat dog technology, which we believe makes them more compelling.
I would like to.
That pardon team for delivering these results I missed a very challenging environment asked me continues to adapt our work streams as it goes all the ongoing comeback.
Chandra Vargeese: For the first time, we are sharing in vivo RNA editing results in non-human primates. For this study, we dosed six non-human primates in pre-grow, subcontinuously, once a day, for 5 days, with 3 chemically distinct RNA editing oligonucleotides. We check liver biopsy samples and, two days post last dose, an evaluated sequence of the targeted sites. As you can see, on the chart on the left, we detected up to 50% editing as compared to baseline in VB.
No look at home.
Clearly demonstrated successful army anything individual across multiple consequence, which supports the potential of this technology to be applied across a variety of disease targets.
Our in B.
Well also be process.
In a poster tomorrow at the American Society of gene. So quickly on the old meeting, which is being held motion.
Yeah.
We expect.
I'm showing no additional invivo Igor mediated army anything began.
Chandra Vargeese: On the right, you can see a Sanger sequencing plot for the targeted oligonucleotides, showing clear editing at the target site. While this is an ongoing proof-of-concept study, we are very excited by these results. And to our knowledge, these are the first successful RNA editing results in non-human primates. Importantly, we have achieved these results using our endogenous ADOT technology, which we believe makes them more compelling. I would like to add that I am proud of our team for delivering these results amidst a very challenging environment as we continue to adapt our work streams as a result of the ongoing pandemic. Now, looking ahead, we have previously demonstrated successful RNA editing in vitro across multiple transplants, which supports the potential of this technology to be applied across a variety of disease targets.
We also expect to announce our first automate editing program and apply to indication now.
No no I'll turn the call back to Paul for closing remarks.
Thanks Sandra.
Clothing, we've had a solid start to the year, we have already delivered on our first milestone and are on track to achieve several more this year, including multiple C.T. a submission.
And data from both our precision HD clinical trials.
I want to close by taking the time to thank the entire weve team as well as our partners in these endeavors, namely our investigators and patient.
Without the steadfast commitment we can never be where we are today.
The wave team has worked tirelessly to keep our clinical trials ongoing our preclinical program and our discovery work on track all while supporting each other and our community in this difficult time.
Whether working from home are working in our labs and manufacturing Sweet entire organization has come together to adapt to our new reality.
Chandra Vargeese: Our in vitro data will also be presented in a poster tomorrow at the American Society of Gene and Cell Therapy annual meeting, which is being held virtually. Later this year, we expect to add or share additional in vivo ADAR-mediated RNA editing data, and we also expect to announce our first RNA editing program in a hepatic indication. Now, with that, I'll turn the call back to Paul for closing remarks.
I am grateful our team has risen to this challenge and for their dedication to bring potential new therapeutic to patients living with devastating diseases.
With that we'll open up the line.
Operator.
To ask a question really depressed charge one group telephone slips all your question. Please proceed with him he.
Please standby weaker how did he rene roster.
In our first question comes from Debbie.
Paul B. Bolno: Thanks, Chandra. In closing, we've had a solid start to the year. We have already delivered on our first milestone and are on track to achieve several more this year, including multiple CTA submissions and data from both our PRECISION-HD clinical trials. I want to close by taking the time to thank the entire WAVE, as well as our partners in these endeavors, namely our investigators and patients, without whose steadfast commitment we could never be where we are today. The WAVE team has worked tirelessly to keep our clinical trials ongoing, our preclinical programs, and our discovery work on track, all while supporting each other and our communities in this difficult time, whether working from home or working in our labs in manufacturing. The entire organization has come together to adapt to our new reality. I am grateful our team has risen to this challenge and for their dedication to bring potential new therapeutics to patients living with devastating diseases. With that, we'll open up the line. Operator
Todd.
With H.C. Wainwright.
You May proceed.
And on for Debjit I, just have a couple of question. So.
The phase one study told me nursing was halted due to inspections from the device is huge collection CSF. So I was wondering if.
Do you guys can tell for using a similar device there or how you might be making that risk.
Yes, Hi, Jeff. This is Mike Yeah. So that study that was halted was a frequent PK study tar tar knowledge, where they had a device and to take frequent lumbar puncture CSF samples, we're not using that device in the studies. These our monthly.
<unk> lumbar punctures for CSF collection and for administration.
Drugs.
Okay, great. Thank you and.
The Arnie editing platform looks interesting.
But my concern would be that.
Nick Administration could induce immunogenic response is similar to what we saw from two comparison.
Could you tell us about names that you guys have taken to mitigate that great, especially if there are differences in the cautions that you took quick fix the person.
Operator: To ask a question, please press star 1 on your telephone. To answer your questions, please press the tone. Please stand by while we have a Q&A session. And our first question comes from Debbie. Deb Chodley, with H.C. Wainwright. You may proceed. I just have a couple of questions.
Yeah I'll take the first part of this and this.
Like to talk about entendre I think the first steps, we're really in understanding now utilization of.
This administration Galnac.
Lower doses and night non Ivy Administration. In addition, I think as we've learned from dividends and continuing to characterize those drugs Preclinically is important and I think as like and share with repeat administration, even systemically at the deficit.
Michael Linden: The Phase 1 study of Tome Inertia was halted due to infections from the device that was used to collect the CSF. So I was wondering if you guys can tell us if you're using a similar device or how you might be mitigating that risk?
Paul B. Bolno: Yeah, hi, this is Mike. Yeah, so that study that was halted was a frequent PK study, to our knowledge, where they had a device in to take frequent lumbar puncture CSF samples. We are not using that device in the studies. These are monthly lumbar punctures for CSF collection and for administration of the drug.
The study with inconclusive not because of safety, but ultimately because of that because like I don't know if you have anything you would add to that.
No I mean, I'd just add that you know as we learn more and more about 'em administration distribution and getting the oligonucleotides to the target. It is about sudarsan was about getting enough oligonucleotide to target with the profile that enabled us to do that and I think right now where we are here you've heard this is a using galnac.
Paul B. Bolno: Okay, great, thank you. And so the RNA editing platform looks interesting, but my concern would be that systemic administration could induce immunogenic responses, similar to what we saw from fubidursin. So could you tell us about any steps you guys have taken to mitigate that risk, especially if there are differences in the precautions that you took with fubidursin?
And I think also we're in a very early stages here that as we.
Paul B. Bolno: Yeah, I'll take the first part of this and then hand this to Mike to talk about, and Chandra. I think the first steps were really in understanding the now utilization of subcutaneous administration of GalNec and lower doses and non-IV administration.
Learn more about this approach and optimize the oligonucleotide certainly immune activation is one of the criteria, we're going to use in in our screening of future candidates.
Okay, Great makes sense. Thanks, thanks for the answers guys.
Q.
Your next question comes from Tim I'm, sorry, with Mizuho Mizuho you May proceed.
Paul B. Bolno: of Stuva Dursen, continuing to characterize those drugs pre-clinically is important, and I think, as Mike can share, with repeat administration, even systemically at Stuva Dursen, the study was inconclusive, not because of safety, but ultimately because of efficacy. Mike, I don't know if you have anything you want to add to that.
Under a nice to hear your voice and earn in school couple of questions on the coded related impacts maybe Paul or Mike. One is how are you guys. Collecting these CSF samples are you seeing patients still coming in to the site.
What's been the impact there.
Michael Linden: No, I mean, I just want to add that, as we learn more and more about administration distribution and getting the oligonucleotides to the target, it is about Suvidursen was about getting enough oligonucleotides to target with the profile that enabled us to do that. And I think right now, where we are here, you've heard that this is using GalNec, and I think we're also in the very early stages here, as we learn more about this approach and optimize the oligonucleotides, certainly immune activation is one of the criteria we're going to use in our screening of future candidates.
Have you second is have you seen.
Right do you define impact differently between site activation and enrollment versus the already enrolled patient population.
And then lastly for any sites that have been particularly hard during the cobot 19 crisis.
Have you seen the enrollment tick back up at all high in the last couple of weeks as some of your parents have noted for the for their trials. This will not necessarily HD related but in general. Thank you.
Paul B. Bolno: Okay, great. It makes sense. Thanks. Thanks for the answers, guys.
Thanks, and I'll turn it over to Mike.
Yeah, actually now well I would say that this situation I think that I'm well first of all your first question about CSF samples and the patients coming in I wanted to things that we were most worried about but we have been very I'm encouraged by.
Paul B. Bolno: Thank you.
Operator: And our next question comes from Salim Syed with Mizzou Hill. Mizzou Hill, you may proceed. Andre, nice to hear your voice on Ernie's call. A couple questions on the COVID-related impacts. I don't know, maybe Paul or Mike.
Is that patients are wanting to come in they are wanting to come in for their visits they're wanting to come in for their CSF collections and their administration. So we've been fortunate the monthly visits allow the flexibility to schedule around what's happening at the site. So we've been fortunate in that situation and I think we having a very dedicated.
Paul B. Bolno: One question is, how are you guys collecting these CSF samples? Are you seeing patients still coming in to the sites? Or what's been the impact there? Have you, secondly, have you seen, do you define impact differently between site activation and enrollment versus the already enrolled patient population? And then lastly, for any sites that have been particularly hit hard during the COVID-19 crisis, have you seen enrollment pick back up at all in the last couple weeks, as some of your peers have noted for their trials as well? Not necessarily HD-related, but in general. Thank you.
They did patient population has mitigated much of what could potential their patients just saying I'm just gonna stay home I'm, a little worried I'm. So.
That's weve so that's been our experienced thus far I think with your other questions about whats essentially happening at different did it in terms of timing and different visits in how we look at.
Michael Linden: Thanks, I'll turn it over to Mike.
Michael Linden: Yeah, hi Salim. Well, I would say that in this situation, first of all, your first question about the CSF samples and the patients coming in. One of the things that we were most worried about, but we have been very encouraged by, is that patients are wanting to come in. They're wanting to come in for their visits, they're wanting to come in for their CSF collections, and their administration.
Some of the you know site activation versus enrollments.
I think basically all of these are factors as we're looking at different sites around the world different sites of different challenges different health authorities have different rules as you know the country different countries had been impacted in handled the quite differently. We have approached each one of the sites individually and whatever.
Michael Linden: So we've been fortunate; the monthly visits allow the flexibility to schedule around what's happening at the site. So we've been fortunate in that situation, and I think having a very dedicated patient population has mitigated much of the potential there for patients just saying, I'm just going to stay home, I'm a little worried. So that's been our experience thus far. I think with your other questions about what's happening,
Plan is best for them in terms of recruitment and monitoring of patients. We've done that that we've done it in a way that's consistent with our protocols that allows us to ensure the data quality and we've been fortunate I think it just happens to be where we've chosen the sites and the disease indication we've chosen.
And on into your last point over the last several weeks, obviously things have been loosening up around the world and we have are optimistic about where that's heading and that have that's been that optimism certainly has been conveyed from our sites.
Michael Linden: Essentially, happening at different times and in terms of timing and different visits and how we look at some of the site activation versus enrollment. I think basically all of these are factors as we're looking at different sites around the world; different sites have different challenges, and different health authorities have different rules. As you know, different countries have been impacted and have handled it quite differently. We have approached each one of the sites individually, and whatever plan is best for them in terms of recruitment and monitoring of patients, we have done that. But we've done it in a way that's consistent with our protocols, that allows us to ensure data quality, and we've been fortunate. I think it just happens to be where we've chosen the sites and the disease indication we've chosen. And to your last point, obviously, things have been loosening up around the world, and we are optimistic about where that's heading, and that optimism is certainly being conveyed from our sites.
Okay. Thanks, so much guys. Thank you.
And our next question comes from many so hard with SPD Leerink you May proceed.
I've into and Garmany just a quick question on our end just.
Following up on the curve it impact on the HD program have you had any.
Comes with enrollment treatment from enrollments on Thursday.
Milligram cohort purposes, and HD, one I see two programs, meaning like can you guide how many patients have been treated this point until we anticipate data from all 12 patients each cohort from the issue on program moving forward.
Mike Gerlich, Yeah, I'll take that so I think that our guidance is that as we said that we anticipate that when we present. These data in the second half when a release these days in the second half. It will include all patients in the cohort.
In terms of the individual we'd never comment on the individual enrollment, but again our diversity of sites are.
The disease indication selfie infrequent dosing the monitoring that's required has allowed us to if it given site might have reduce their enrollment or reduce their activity. We can always pivot to another site. So our intention is to share complete cohort data in the second half.
Michael Linden: Okay, thanks so much, guys. Thank you.
Paul B. Bolno: Thank you.
Operator: And our next question comes from Mani Foroohar with SBV Lyrinc. You may proceed. Arvinda and Virmani, just a quick question on our end.
Michael Linden: Just following up on the COVID impact on the HD program, have you had any problems with enrollment and treatment from enrollment in the 32 milligram cohort for the Precision HD1, HD2 programs? Meaning, can you guide how many patients have been treated at this point? And can we anticipate data from all 12 patients in each cohort from the HD1 program moving forward?
Great. Thanks, a lot that.
Thank you.
And our next question comes from Ian Young with Jefferies.
You mean Christie.
Q4 precision H.B. wanting to study Youre selection of.
Hi, Joe So from somebody can milligram are you gonna be announcing that before the data. There we are going to be expecting second half of a D. C O where at the same time.
Michael Linden: Michael Hickman
Michael Linden: Yeah, I'll take that. So, I think that our guidance is, as we said, that we anticipate that when we present these data in the second half, when we release these data in the second half, it will include all patients in the cohort. In terms of the individual, we never comment on the individual enrollment, but again, our diversity of sites, the disease indication itself, the infrequent dosing, and the monitoring that's required has allowed us to know if a given site might reduce their enrollment or reduce their activity; we can always pivot to another site. So our intention is to share complete cohort data in the second half.
So Paul.
Okay I'm going after I mean at this point, we're guiding to the clinical trials in that we're I think moving to the higher dose we haven't yet.
Now when we would be announcing next cohorts and publicly.
Obviously as we continue to progress in this that we will give up like.
Right and just to say with that the data that will be second half will be that 32 milligram cohort that you're asking about.
Okay, and then on any editing asset up here. It's just so in non human primates. He showed up to 50% of editing efficiency I don't know, maybe it's too early to ask a but for a clinical benefit to what kind of it did.
Paul B. Bolno: Great, thanks a lot guys. And our next question comes from Eun Yang with Jeffrey. You may proceed. Thank you for the precision HD 1 and 2 study. This is a question for you. In the last year, you have seen higher doses from 32 mg. Are you going to be announcing that before the data that we are going to be expecting in the second half of this year or at the same time?
He patients do you think you didn't need to achieve.
Thank you and then.
Understood and I'm happy to bring tender and I mean, I think a lot of it depends on target selection and again. The beginning part is these are the unoptimized starting point, though this is not yet the preclinical data around clinical program.
Michael Linden: This is Paul. Mike, you can keep coming after.
Paul B. Bolno: I mean, at this point, we're guiding to the clinical trials and that we're assessing moving to the higher dose. We haven't yet announced when we would be announcing the next cohorts and that publicly. Obviously, as we continue to progress and assess, we will give updates.
To be progress I think our target selection when one looks at it was I guess, he is where 50% correction or no.
Michael Linden: Right, and just to say with that, the data that will be the second half will be that 32 milligram cohort that you're asking about.
They're diseases, where restoration of 50% and even less than 50% could actually changed diseases nickel.
Yeah.
Paul B. Bolno: And then there is RNA editing therapy.
Not that we wouldn't be as.
Paul B. Bolno: In non-human primates, you showed up to 50% editing efficiency. I don't know, maybe it's too early to ask, but for clinical benefits, what kind of editing efficiency do you think you would need to achieve?
As much as possible I think the hurdle of achieving what we would need to to be able to go into any number of diseases, we feel like.
That.
This initial exploratory program I think the exciting part is we'll give more updates as we move through the year is really on what targets are coming in one can see where our data will deliver on it.
Paul B. Bolno: I think, Eun, and then, you know, if Chandra has to add something, I'm happy to bring Chandra in. I mean, I think a lot of it depends on target selection. And, you know, again, the beginning part is these are the unoptimized starting points, so this is not yet the preclinical data around clinical programs, which continue to be progressed. But I think our target selection, you know, when one looks at heterozygous diseases where 50 percent correction could restore a normal phenotype, there are diseases where restoration of 50 percent and even less than 50 percent could actually change the disease. So the goal doesn't necessarily have to be 100 percent. (inaudible)
I'm trying to figure anything you want to add to that.
Well this is going on you know what what do you said this correctly, though in some big aegis, but they have those I guess.
Patient population 50, pushing everything wellness store of holding in time.
Hey.
In the last two questions if I missed the hirji appalled apologize, but the partner to programs with the Pfizer Oh Youre show you didn't like do not an eight editing.
Technology.
No. The R&D editing technology came outside of Pfizer Pfizer was only using the very early on chemistry.
Not even.
Turning to our more recent.
Are they editing it outside either.
Okay. Thank you very much.
And our next question comes from Whitney Graham with Guggenheim Securities You May proceed.
Paul B. Bolno: No, this is correct. You know, what you said is correct. In some diseases with a heterozygous patient population, 50% editing will restore a full phenotype. I see. And the last question, if I misheard you, Paul, I apologize, but the partner to programs...
[music].
Okay. Thanks for taking my question I'm on the precision issue trials are you guys in anyways limited on the ability to do is higher based on previous daughter preclinical data.
I would you like.
Yeah. So I'm no we based on our preclinical data we definitely have clearance to go higher we have to you I should say the window to go higher.
Paul B. Bolno: Are you also utilizing RNA editing technology?
Paul B. Bolno: No, the RNA editing technology came outside of Pfizer. Pfizer was only using the very early chemistry of WAVE. Thank you.
So that's part of the what we're looking at when we will be looking at all the did all the clinical data in terms of safety PK and then that safety window.
Paul B. Bolno: RNA editing is outside the scope of the Pfizer class.
Paul B. Bolno: Thank you very much.
Michael Linden: And our next question comes from Whitney Lamb with Guggenheim Securities. You may proceed. Hey, thanks for taking my question. On the PRECISION HD trials, are you guys in any way limited on the ability to do this higher based on previous data or preclinical data?
So, but we have the preclinical data does support a dose escalation.
Thanks, and then.
In terms of the data, we'll see in second half you kind of going over what we can expect to see will be similar to that kind of that's what's up on began in December.
Michael Linden: Thank you.
Yeah. So it will yes, there will be similar to what you're seeing there accepts that whether you'll be seeing as the individual cohorts because we now rather than that was an interim analysis of an ongoing study what you'll be seeing at the end of this year will be the complete data sets of the individually.
Michael Linden: Based on our preclinical data, we definitely have clearance to go higher, or I should say the window to go higher, so that's part of what we're looking at when we will be looking at all the clinical data in terms of safety PK and then that safety, but we have the preclinical data that does support a dose escalation.
Michael Linden: Thanks, and then, in terms of the data we'll see in the second half, can you kind of go over what we can expect to see? It will be similar to the kind of initial top line we got in December.
Cohort.
Thanks, I just had one last question you expect to receive any more cash payments rough friends from collaborations this year as we think about capital.
Michael Linden: Yes, it will be similar to what you've seen there, except that what you'll be seeing is the individual cohorts because, now, rather than that was an interim analysis of an ongoing study, what you'll be seeing at the end of this year will be the complete data sets of the individual cohorts.
I think we always contemplate doing business development.
Central opportunities those all have the central to bring in additional capital through pipeline programs as well it through the platform.
I think there are opportunities. In addition, our cash runway statements don't take into account.
The thing operations.
I think as we as we think long term I think there are opportunities for additional capital.
Paul B. Bolno: Thanks. And I just had one last question. Do you expect to receive any more cash payments or upfronts from collaborations this year as we think about cash runways?
Thank you.
Your next question comes from Paul MACI with Stifel, You made Christie.
[laughter].
Just a couple of questions from us today I'm just curious if you are thinking about this NIPT three program whether or not.
Paul B. Bolno: I think we always contemplate doing business development, and I think there are potential opportunities. Those all have the potential to bring in additional capital both through pipeline programs as well as through the platform. So I think there are opportunities. In addition, our cash runway statements don't take into account milestones from existing customers. I think as we think long term, there are opportunities for additional capital.
That initiation is going to be dinner at all by the top line from the HD. One each did you read outs and along with that if you. That's just start at higher doses pending that those data and then Additionally, just curious if you have any updates on.
The presentation of the full oximeter Sun data.
Thanks.
Thanks, I'll take the beginning intra and then like to follow up but I think what was important to us is in advancing the three it's a different scenario than advancing in within Dnbi, Our exon 53 program on the back.
Paul B. Bolno: And our next question comes from Paul Matias with FECAL. You may proceed. We just have a couple of questions from us today. I'm just curious if, thinking about the SNP3 program, whether or not that initiation is going to be gated at all by the top line from the HD1, HD2 readouts, and along with that, if you expect to start at higher doses, you know, pending that data. And then additionally, just curious if you had any updates on the presentation of the full Suvidarsan data. Thanks.
Yes.
Our commitment to Huntingtons disease or me that path.
For us represent different opportunity.
For data says is generally we had a pre clinical in vivo model that we could develop it off.
Uh huh.
But I think we remain committed to advancing.
Right.
Outside of independent.
He wanted to Mike I don't know if you want it.
Yes, sure as Paul said, I mean, we have a dataset on snipped three using new chemistry from the platform in vivo that makes US see this is a totally different molecules. We have each of our molecules are there optimized and specifically designed so.
Paul B. Bolno: Thanks. I'll take the beginning intro and then Mike will follow up.
Paul B. Bolno: But I think what's important to us is that in advancing SNF3, it's a different scenario than advancing within DMD, our Exxon 53 program on the back of Suvidur. Our commitment to Huntington's disease remains steadfast. SNP3 for us represents a different opportunity, a different data set that's been generated. We had a pre-clinical in vivo model that we could develop it off of, and I'll let Mike speak to that later. But I think we remain committed to advancing SNP3 outside of and independently from the data from PrecisionHP1 and 2. Mike, I don't know if you want to continue.
Sneak one instant to data will not influence our trust and moving forward here given our commitment to HD and the fact that these data stand on their own in terms of dosing again, we're in a very different place than we were with snip one's going to.
Where we did not have models to look at target engagement in the in vivo setting here we do.
And or dosing paradigm will be driven by what we've learned from knocked down.
Michael Linden: Yes, sure. As Paul said, we have a data set on SNP3 using new chemistry from the platform in vivo that makes us see this as a totally different molecule. As each of our molecules is optimized and specifically designed, so SNP1 and SNP2 data will not influence our interest in moving forward here, given our commitment to HD and the fact that these data stand on their own. In terms of dosing, again, we're in a very different place than we were with SNP1 and SNP2, where we did not have models to look at target engagement in the in vivo setting. Here we do. And our dosing paradigm will be driven by what we've learned from knockdown in the in vivo models, as well as in preclinical studies. So where we go in the clinic with dosing will be guided by a much more complete data set and based on the molecule itself and what was seen in these various in vivo studies.
In the in vivo models as well as our preclinical so.
Where we go in clinic with dosing will be guided by a much more complete.
Dataset and based on the molecule itself and what each what was seen in these various in vivo studies.
And Mike on the how many deanna yeah.
Yeah.
And then on in terms of the Super Dursun data excuse me I'm, what supercenters in data.
Basically I mean theres not much more to report then what we presented at the the M.D.A. meeting where.
We saw I'm quite clearly that.
While that regardless of how we looked for target engagement, we did not see it in those muscle biopsies and it does seem that the drug was there but he didn't 90% were was essentially in the extra cellular matrix as opposed to getting to target. So we believe that that's.
Michael Linden: And then in terms of the Suvadersan data, basically, I mean, there's not much more to report than what we presented at the MDA meeting where we saw quite clearly that regardless of how we looked for target engagement, we did not see it in those muscle biopsies. It does seem that the drug was there, but 80% to 90% was essentially in the extracellular matrix as opposed to getting to the target. So we believe that that's why...
The complete story in terms of what happened with Super Dursun and.
That's there's not anything new to report about that.
Okay, great. Thanks, guys.
<unk>.
And I saw me a final question comes from during winter, which.
Alan.
You May proceed.
Obviously really exciting moving into they are less an empty spaces there.
So I I, just kind of wanted to touch base on.
Michael Linden: This is a complete story in terms of what happened with Suva Durson. There's not anything new to report. Thanks, guys.
How you're thinking about the timing of these clinical programs. When you think you actually filed these high and decent if you think you'll need stepper ones for each space.
Paul B. Bolno: Alright, great. Thanks guys. And our final question comes from Euryn Warder with... Chao-Lin. You may proceed. I'm obviously really excited.
And if you really would try to kind of pursue them in parallel.
From there on out thanks, very much yeah. No. One is great question I'll pass it to Mike, but I appreciate you or your thoughts around how we think about you know north is both as a treatment for airlift he and the importance around how this disease progressive in both patients and how we're taking an approach that's looking broadly at both the diseases like do you want it.
Operator: Moving into the ALS.
Operator: http://thevenusproject.com
Operator: I just kind of wanted to touch base on
Operator: How What you're thinking about the timing of these clinical programs.
Operator: When
Operator: When you think you'll actually file these INDs, and if you think you'll need separate ones for each space, And if you really would, try to kind of pursue them in parallel from there on out. Thanks very much. Yeah, no, and it's a great question. I'll pass it to Mike, but I appreciate your thoughts around how we think about C9-NORF as both a... for ALS FPD and the importance of how this disease progresses in both patients and how we're taking an approach that's looking broadly at both of those diseases.
Huh.
Our strategy.
No. Thank you for that and I think that you highlight something it's very exciting about this up program that we.
Can target.
I'm a single area.
In effect to very important diseases in terms of the timing of the initial C.T.A. <unk> that is the second half as we've said in that C.T.A.
Ill is is being the strategy behind that right now it's being devised in working with the authorities on how we might be able to use that to rapidly get into both indications are not prepared to get into the details there but that is our intent our intent is to rapidly get into both disease indications targeting the single gene target.
Michael Linden: Thank you for that. I think that you highlight something that is very exciting about this program, that we can target a single area and affect two very important diseases. In terms of the timing of the initial CTA, that is the second half, as we've said, and that CTA is being – the strategy behind that right now is being devised and working with the authorities on how we might be able to use that to rapidly get into both indications. I'm not prepared to get into the details there, but that is our intent. Our intent is to rapidly get into both disease indications targeting this single area.
Okay. Do you think you would you like a like a phase one in healthy volunteers kind of a situation and then kind of branch out into the individual educations from there on hours, it's still a little the influx well I would say that weve unlikely to go in healthy volunteers in general with an intra typically administered drug here, but I could say that you know depopulation.
Michael Linden: Okay, do you think you would do like a phase one and...
Operator: Or is it still a little bit in flux?
And we would go into in the first studies would set us up well to go into them either indication.
Michael Linden: Well, I would say that we are unlikely to go into healthy volunteers in general with an intrathecally administered drug here, but I could say that the population we would go into in the first studies would set us up well to go into either indication.
Okay. Thanks very much.
Thank you.
Ladies and gentlemen, this now concludes that unique Washington Today's conference I'll now turn call back over to Dr., Paul Bono for any closing remarks.
Michael Linden: Thanks very much.
Operator: Thank you.
Paul B. Bolno: Ladies and gentlemen, this now concludes our Q&A portion of today's conference. I'll now turn the call back over to Dr. Paul Bolno for any closing remarks.
Thanks, everyone for joining the call. This morning to review, our first quarter update and thanks again to our employees for their hard work and commitment.
We look forward to updating you in the future on our ongoing progress if they have a nice day. Thank you.
Operator: Thank you everyone for joining the call this morning to review our first quarter update. And thanks again to our employees for their hard work and commitment. We look forward to updating you in the future on our ongoing progress. Stay safe and have a nice day. Thank you.
Ladies and gentlemen, Nike friction in today's conference. This now concludes that program and give me all disconnect everyone have a great thing.
[music].
Operator: Ladies and gentlemen, thank you for attending today's conference. This now concludes our program, and you may all disconnect. Everyone have a great day.