Q4 2019 Earnings Call
Good afternoon, and welcome to the Voyager Therapeutics fourth quarter for year 2019 financial results Conference call.
Unknown Executive: Good afternoon, and welcome to the Voyager Therapeutics 4th Quarter and 4 Year 2019 Financial Results Conference Call. At this time, all participants are in a listen-only mode.
This time all participants on in listen only mode. This call is being webcast live on the Western media section of voyages website at Voyager Therapeutics dotcom.
Unknown Executive: This call is being webcast live on the Investor & Media section of Voyager's website at voyagertherapeutics.com. This call is the property of Voyager Therapeutics, and recordings, reproduction, or transmission of this call without the express written consent of Voyager Therapeutics is strictly prohibited. Please be advised that this call is being recorded. I would now like to introduce Paul Cox, Head of Investor Relations at Voyager.
This call is the property of Voyager therapeutics and recordings reproduction or transmission of this call without the expressed written consent of Voyager therapeutics is strictly prohibited.
Please be advised that this call is being recorded I would now like to introduce all top.
Head of Investor Relations at Voyager.
Paul Cox: Good afternoon, and thank you for joining us. With me on the call today are Andre Turin, our President and Chief Executive Officer, Omar Khawaja, Chief Medical Officer and Head of R&D, and Allison Dorval, Chief Financial Officer. This afternoon, after market close, we issued a press release that outlines the financial results and corporate highlights for the fourth quarter and full year 2019. The release is available at voyagertherapeutics.com. Before we begin, just a reminder that the four forward-looking statements included in this call represent the company's views as of today, March 3, 2020. Voyager disclaims any obligation to update these statements to reflect future events or circumstances, except as required by law. Please refer to today's press release, as well as Voyager's filings with the SEC, for information concerning risk factors that could cause actual results to differ materially from those expressed or implied by such statements. With that, I will turn the call over to Andre.
Good afternoon, and thank you for joining us.
With me on the call today, or Andre <unk>, our president and Chief Executive Officer.
<unk>, Chief Medical officer, and head of R&D.
And Allison Dorval, Chief Financial Officer.
This afternoon after market close we issued a press release, which outlines the financial results in corporate highlights for the fourth quarter and full year 2019.
There really is available at Voyager therapeutics Dot com.
Before we began just a reminder that the forward looking statements included in this call represent the company's views as of today March 3rd twice what it.
Border disclaims any obligation to update these statements to reflect future events or circumstances, except as required by law.
Please refer to today's press releases as well as voyagers filings with the FCC for information concerning risk factors that could cause actual results could differ materially from those expressed or implied by such statements.
With that I will turn the call over to Andre.
Andre Turin: Thank you, Paul, and good afternoon, everyone. Welcome to our Q4 earnings and corporate update call. I'll begin by walking you through our recent updates. Omar will discuss our pipeline programs and plans, and Allison will close with our financial results and guidance. Once we've concluded our remarks, we'll take questions in the Q&A session.
Thank you Paul and good afternoon, everyone welcome to our Q4 earnings and corporate update call I'll begin by walking you through our recent updates Omar will discuss our bottling program than plan and Alison will close what our financial results and guidance.
Once we've completed our remarks, we'll take out was questions in a <unk> session.
Andre Turin: We continue to make progress to establish Voyager as the leading gene therapy company focused on severe neurological diseases. We believe that our combined expertise in gene therapy and neuroscience gives us the focus and ability to drive innovation in this area. In 2019, we continued to build on our experience and to enrich our toolbox for CNS gene therapy. A key feature of the Voyager pipeline is that our lead programs for Parkinson's disease and Huntington's disease are leveraging targeted surgical delivery of our gene therapies to very precise regions of the brain. We believe that this intraparenchymal approach has the important advantage of potentially bypassing certain historical and present challenges of systemically delivered AAV gene therapies, such as limited brain transduction, dose-limiting distribution to untargeted tissues and organs, systemic immunogenicity, and production at large scale. At the same time as we've advanced these two lead programs, we've continued to invest in the discovery of novel capsids specifically engineered to enhance blood These efforts may significantly broaden our ability to develop gene therapies that deliver a wide variety of payloads to specific regions, cells, and genetic targets in the brain.
We continue to make progress to establish Voyager de leading gene therapy company focused on severe neurological diseases.
We believe that our combined expertise and gene therapy and narrow sign.
Just a focus and ability to drive innovation in this area.
In 2019, we continued to build on our experience and to enrich our tool box for CNS gene therapy.
A key feature of the Voyager pipeline is that our lead programs for Parkinsons disease and Huntingtons disease.
We're leveraging a targeted surgical delivery of our gene therapies to very precise regions of the brain.
We believe that this interoperable approach has the important to the advantage of potentially bypassing certain historical and present challenges of systemically delivered Avi gene therapies, such as limited brain transaction dose limiting distribution to untargeted tissues and organs.
That make immunogenicity and production at large scale.
At the same time as we advance. These two lead programs. We've continued to invest in a discovery of novel Capsids, specifically engineered to enhance blood brain barrier penetrant.
These efforts may significantly broaden our ability to develop gene therapies that deliver a wide variety of payloads two specific regions cells and genetic targets in the CNS.
Another key feature of the Voyager pipeline is that the as we've shown that are Parkinsons program. We've developed a manufacturing process that we believe enables the production of high quality Avi gene therapies of commercial scale.
Andre Turin: Another key feature of the Voyager pipeline is that, as we've shown with our Parkinson's program, we've developed a manufacturing process that we believe enables the production of high-quality AAV gene therapies at commercial scale. We'll continue to invest in these leading capabilities and expertise in AAV production and manufacturing as we advance our pipeline. Turning to our program updates, our lead program, BYADC, is currently being evaluated in the RESTORE-1 clinical trial in Parkinson's disease patients. Along with our partner Neurocrin, we recently updated the trial protocol after seeking and receiving FDA feedback. The protocol amendments are intended to enhance the patient experience in the trial, while also ensuring that the overall VYDC clinical program is designed to support the BLA filing. In addition to implementing these changes to RESTORE-1, we plan to initiate a global study in RESTORE-2 in the second half of 2020. We also expect to provide important longer-term data this year from the VYDC Phase 1B program. In total, these data will represent more than 60 patient years of data on VY-ADC. Next, we turn to our lead, wholly owned program, VY-HTTL1 for Huntington's Disease.
Well continue to invest in these leading capabilities and expertise and Avi production and manufacturing as we advance or pipeline.
Turning to our program update our lead program be why do you see is currently being evaluated and to restore one clinical trial in Parkinson's disease patients.
Along with our partner NERC, Ryan we recently updated the trial protocol after seeking and receiving FDA feedback.
The protocol amendments are intended to enhance the patient experience in the trial, while also ensuring that the overall the why D.C. clinical program. It is designed to support the BLE filing.
In addition to implementing these changes to restore one we plan to initiate a global.
That study and restore too in the second half of 2020.
We also expect to provide important longer term data this year from that'd be why do you see phase one be program.
In total these data will represent more than 60 patient years of data on the why a b C.
Turning to our lead wholly owned program the why HD video on for Huntingtons disease. We're currently in the process of conducting and the reviewing preclinical R&D, enabling studies.
Andre Turin: We're currently in the process of conducting and reviewing preclinical IND-enabling studies. We plan to provide an update in the second quarter of this year regarding the program, including plans to file an IND application. We also plan to initiate a prospective observational study of HD patients in the middle of the year. We're also excited to continue our momentum on our Vectorized Antibody programs, our other preclinical programs, and our novel capsid efforts. We plan to present updates on these programs and efforts during 2020. Finally, we continue to invest in building Voyager for long-term leadership in neurological gene therapy, and this includes a continued focus on adding expertise and talent. We recently made a few key hires, including Jennifer Hunt as Senior Vice President of Development Operations, Julianne Muscat as Vice President of Quality, Gan Wei as Vice President of Technical Development, and last but not least, Paul Cox, who just kicked off the call, as Head of Investor Relations. We're thrilled to have each of them on the Voyager team. With that, I'll now turn the call over to Omar to provide more detail on our pipeline programs. Okay, Omar?
We plan to provide an update than the second quarter of this year regarding the program, including plans to file and I'm the application.
We also plan to initiate a prospective observational study of HD patients in the middle of the year.
We're also excited to continue our momentum on our victory as anybody programs are at or preclinical programs and our novel Capsid efforts, we plan to present the updates on these programs and efforts during 2020.
Finally, we continue to invest in building Voyager for long term leadership and neurological gene therapy and this includes a continued focus on adding expertise on town.
We recently made a few key hires including Jennifer on.
Senior Vice President of development of operations, Julian Muscat, as Vice President of quality Gannaway, as Vice President of technical development and last but not least ballcocks. We just kicked off the call as head of Investor Relations. We're thrilled to have each of them on the Voyager team.
With that I'll now turn the call over to Omar to provide more detail on our pipeline programs Omar.
Omar Khawaja: Thank you, Andre. I'd now like to walk through some of these program updates in more detail. First, I'll discuss the updates for the Parkinson's disease program, which is partnered with Neurocrin. As a reminder, our approach is to deliver the gene for the AADC enzyme locally to the putamen using an AAV viral vector. The goal is to use a one-time infusion of a small volume of gene therapy to create a stable reservoir of AADC enzyme in the putamen capable of converting levodopa to dopamine. The result is a regulatable system controllable by exogenous levodopa, the standard of care oral medication. We think there is an important advantage to this approach, as physicians and patients are able to titrate oral levodopa medication to the patient's individual need while avoiding side effects due to excess dopamine systemically or in other brain regions.
Thank you Andre.
I'd now like T walk through some of these program updates in more detail fast I'll discuss the update for the Parkinson's disease program, which has partnered with no crane.
As a reminder, our approach is to deliver the Jane for the AIDC enzyme locally to the putamen using an HIV viral back to.
The goal is to use a onetime infusion of a small volume as gene therapy to create a stable reservoirs AIDC enzyme in the putamen capable of converting leave the type of that to me.
The results as a regular couple system controllable by exogenously, the tied to the standard of care oral medication.
We think there is an important advantage because that's a page I physicians and patients are able to titrate oral leaves the type of medication to the patients individual need while avoiding side effects due to access doesn't mean systemically or another brain regions.
The phase one clinical programs. So the way AIDC was designed to establish safety and optimized spacing on delivery.
Omar Khawaja: The Phase I clinical program for VYA-ADC was designed to establish safety and optimize dosing and delivery. The PD1101 trial is evaluating escalating doses across three cohorts of five patients each using the transfrontal surgical delivery route, while the PD1102 trial is studying 8 patients and focused on using a posterior trajectory delivery. In our Phase I study results to date, we have observed that by replacing the AADC activity through our one-time gene therapy, we see increased AADC enzyme activity, as measured by F-Dopa PET scans, and improvements in motor function and quality of life in study participants across multiple endpoints, together with a significantly reduced need for oral levodopa medication. As Andre mentioned, we expect to present final three-year data from the PD1101 trial and two-year data from the PD1102 trial at medical congresses this year.
PT 11, I, one trial is evaluating escalating doses across three cohorts of five patients age using the trans frontal surgical delivery, but.
Well the PD 11, I two trial studying a patient and focused on using opposed to reject treat delivery.
You know phase one study results to date, we have upset that by replacing the ADCC activity through all onetime gene therapy.
We see increased AIDC enzyme activity as measured by asked at the pet scans and improvements in nights, a function and quality of life and study participants across multiple endpoint together with a significantly reduced need for or at least the type of medication.
As Andres mentioned, we expect to present final three at data from the PD 11, I, one trial and to your data from the PD 11, no two trial medical Congresses. This year.
Omar Khawaja: These longer-term results from patients with advanced Parkinson's disease at the time of BYA-ADC treatment may speak to the durability of effect and provide important evidence of sustained clinical benefit in the context of the predicted course of disease progression for Parkinson's disease. We believe these data, which include a number of patient-reported, physician-assessed, and objective endpoints, will be important given the decline in motor function and quality of life that Parkinson's disease patients experience over multiple years at this stage of disease. For example, we know that in longer-term studies of patients receiving other types of therapy, such as deep brain stimulation or alternative dopaminergic formulations, patients experience declining good on time, worsening motor symptoms, as well as increases in levodopa-equivalent doses after three years.
These long term results from patients with advanced Parkinson's disease at the time the fee like AIDC treatment.
Nice speak to place a joke those heap effect and provide important evidence of sustained clinical benefit.
In the context of the predictive pool of disease progression Parkinson's disease.
We believe these data which include a number of patient reported position assessed.
The objective endpoint.
We'll be important given that the kind of <unk> function and quality of life. The Parkinson's disease patients experience over multiple years at this stage of disease.
For example, we like that had a longtime studies of patients receiving other types of therapy, such as deep brain stimulation for alternatives that's in tonnage at formulations.
Patients experienced declining good on time, Wessling motor symptoms as well as increases and leave it open equivalent basis after three years.
The way I'd say is currently being evaluated and there were still one study a randomized sham surgery controlled double blinded phase two trial.
Omar Khawaja: BYADC is currently being evaluated in the RESTORE-1 study, a randomized sham surgery-controlled, double-blinded Phase 2 trial. As Andre mentioned, we are now implementing a trial protocol amendment based upon our discussions with the FDA. The protocol amendments include plans to increase enrolment to approximately 85 patients and randomize patients to receive BYADC treatment or sham surgery on a 2-to-1 basis as opposed to the prior 1-to-1 randomization scheme. We're also reducing exploratory assessments not relevant to support registration.
As Andres mentioned, we are now implementing a trial protocol amendment based upon our discussions with the FDA.
The protocol amendments include plans to increase enrollment to approximately 85 patients and randomized patients to receive B.Y. AIDC treatment well sham surgery on a two to one basis as opposed to the prior one to one randomization scheme.
We're also reducing exploratory assessment, it's not relevant support registration.
Omar Khawaja: These protocol modifications are expected to enhance the experience of patients and sites participating in the trial and allow RestoreOne to act as one of two adequate and well-controlled trials to support registration of VYA-ADC for the treatment of Parkinson's disease. Importantly, there are no changes to the primary efficacy endpoint or key secondary endpoint. We expect to update the Restore One enrolment timeline following implementation of this protocol amendment. We also expect to initiate the second pivotal trial, Restore2, together with Neurocrin, in the second half of 2020. Turning now to VYHTT01, our lead wholly-owned program for the treatment of Huntington's disease. VOI-HCT01 is an AAV-based gene therapy encoding a novel microRNA designed to knock down human Huntington's mRNA. We're pursuing a novel delivery paradigm by targeted local delivery of VY-HTT01 to both the Batayman and Thalamus, leveraging both the primary site of disease pathology as well as the thalamus's extensive neuronal projections to the cortex.
These post call modifications are expected to enhance the experience of patients and sites participating in the trial.
Well I was still want to act as one of two out of put them well controlled trial to support registration to see why AIDC as a treatment Parkinson's disease.
Importantly, there are no changes to the primary efficacy endpoint all key secondary endpoints.
We expect to update but we're still one enrollment timelines falling implementation at this place called Amendment.
We also expect to initiate the second pivotal trial was still team together with no credit in the second half of Twentytwenty.
Turning now to Vyšehrad teaser I was a lead Holly and program for the treatment Huntingtons disease.
See why HD teeth, everyone is a navy based gene therapy and coating a novel might call are a nice design to knocked out in human Huntingtons Edmar night.
We are pursuing a novel delivery paradigm by targets had local delivery vyšehrad teaser one they simply timing and tell them enough.
Leveraging by supply beside just disease pathology. That's why this is telling us has extensive nevado projections to the kautex.
We have for he previously presented data demonstrating that delivery as B.Y. I just it seems you're right one into the precise minutes elements of non human primates resulted in white, but widespread distribution of the white HDTV right one back to GE knives, and robust enjoyable knockdown of H.T. I'm on I M protein across both destroyed.
Omar Khawaja: We have previously presented data demonstrating that delivery of VYHTT01 into the putamen and thalamus of non-human primates resulted in widespread distribution of VYHTT01 vector genomes and robust and durable knockdown of HTT mRNA and protein across both striatum and cortex. We've also presented data, including recently at the CHDI Annual Therapeutics Conference. That VY-HTT01 treatment results in a significant and dose-dependent lowering of human HTT mRNA and protein in the striatum of YAK128 mice, a well-characterized transgenic mouse model of Huntington's disease. Concomitant with significant human HCT reductions, VY-HCT01-treated YAK128 mice showed significant improvement in motor function as compared to vehicle-treated animals.
And of course Act.
We've also presented data, including recently at the CHD I know Therapeutics conference.
That B Y S. T T zero, one treatment results and significant dose dependent lowering of human HCT Emerald I M protein in the strides in the CEOC onetime P eight mice.
I will characterize transgenic mouse model of Huntingtons disease.
Cognizant was significant human H.T. reductions P. why HGTV right. One treated yuck 128 mice showed significant improvement in motor function as compared to vehicle treated animals.
We are engaged in the ongoing conduction review of preclinical studies for the Vyšehrad T Zero, one program and expect to provide an update in the second quarter. This year, including all times to fall and I envy application.
We're also planning to begin a prospective observational study in patients with light play dry animal and Ali manifest huntingtons disease in the Twentytwenty.
This study will evaluate the clinical and biological evolution, that's Perry manifest huntingtons disease patient.
By examining longitudinal changes I was a 12 month in clinical Nora imaging molecular digital biomarker endpoints as they relate to the question of Huntingtons disease symptoms.
Omar Khawaja: We're engaged in the ongoing conduct and review of preclinical studies for the VY-HCT01 program and expect to provide an update in the second quarter of this year, including our plans to file an IMD application. We're also planning to begin a prospective observational study of patients with late prodromal and early manifest Huntington's disease in mid-2020. This study will evaluate the clinical and biological evolution of peri-manifest Huntington's disease patients by examining longitudinal changes over 12 months in clinical neuroimaging, molecular, and digital biomarker endpoints as they relate to progression of Huntington's disease symptoms. Patients participating in the observational study may also be eligible for later enrollment in the VY HDT01 Clinical Trial Program. Finally, we continue to advance our earlier stage research programs, including wholly owned efforts, as well as our efforts with our collaboration partners, ADVI and NeuroCrim. These initiatives include our Friedrichs-Ataxia program, currently in preclinical development, our vectorized antibody programs for tau and alpha-synuclein, and our efforts on new research programs, as well as the discovery and characterization of novel AAV capsules. With time to give updates on all of these initiatives in 2020, I'll now pass the call on to Alison.
Patients participating in the observational study may also be eligible for later enrollment in the T Y edge DTC right, one clinical trial probably fine.
Finally, we continue to advance our earliest stage research programs, including wholly owned assets as well as our assets without collaboration partners at the American.
These initiatives include all Friedreichs ataxia paragraph currently in preclinical development.
Well that twice antibody programs with Kolon Alpha Synuclein.
And our efforts on new research programs as well as the discovery and characterization of novel Avi captive.
Time to give updates across all of these initiatives and Twentytwenty I'll now pass the call onto the Allison.
Thanks, Omar I'll now review the highlights of our financial results and guidance.
We ended 2019, and a strong financial position with 281.5 million in cash cash equivalents and marketable debt securities compared to 155.8 million at the end of 2018.
We booked collaboration revenues of 32.7 million in Q4, 2019, and 104.4 million for the year compared to 2.0 million and 7.6 million respectively for the same periods of 2018.
This increase reflects the recognition of amounts related to the Santa Fe Genzyme collaboration in June and amounts related to our Neurocrine and Abbvie Alpha Synuclein collaboration both of which became effective in Q1 of 2019.
Net loss was 12.6 million for Q4, 2019, and 43.6 million for the full year compared to 22.5 million, an 88.3 million respectively for the same periods of 2018.
Allison Dorval: Thanks, Omar. I'll now review the highlights of our financial results and guidance. We ended 2019 in a strong financial position with $281.5 million in cash, cash equivalents, and marketable debt securities, compared to $155.8 million at the end of 2018. We booked collaboration revenues of $32.7 million in Q4 2019 and $104.4 million for the year, compared to $2.0 million and $7.6 million, respectively, for the same periods of 2018. This increase reflects the recognition of amounts related to the Sanofi Genzyme collaboration in June and amounts related to our Neurocrin and AbbVie alpha-synuclein collaborations, both of which became effective in Q1 of 2019.
R&D expenses were 36.6 million for Q4, 2019, and 119.7 million for the year compared to 16.9 million 64.9 million respectively for the same period 2018.
The increase in R&D expenses were primarily related to external cost and employee related costs to support our pipeline programs, including advancing the restore one trial for Vyšehrad C.
DNA expenses were 9.9 million for Q4, 2019, and 36.3 million for the year compared to 8.3 million and 33.8 million respectively for the same periods of 2018.
The increase in DNA expenses was primarily related to employee and facility costs to support the advancement of our pipeline programs and operations.
Turning now to our financial guidance.
We expect to end 2020, with cash cash equivalents and marketable security between 150 and 170 million.
Based on current operating plan, we expect this cash balance along with the amount that we expect to receive for reimbursement of development costs from our Neurocrine collaboration will be sufficient to meet our need for projected operating expenses in capital expenditures into mid 2022.
Allison Dorval: The net loss was $12.6 million for Q4 2019, and $43.6 million for the full year, compared to $22.5 million and $88.3 million, respectively, for the same period of 2018. R&D expenses were $36.6 million for Q4 2019 and $119.7 million for the year, compared to $16.9 million and $64.9 million, respectively, for the same period of 2018. The increase in R&D expenses was primarily related to external costs and employee-related costs to support our pipeline programs, including advancing the RESTORE-1 trial for VYA-DC. G&A expenses were $9.9 million for Q4 2019 and $36.3 million for the year, compared to $8.3 million and $33.8 million, respectively, for the same periods of 2018. The increase in G&A expenses was primarily related to employee and facility costs to support the advancement of our pipeline programs and operations.
We believe we have established a strong capital position through a disciplined financial approach and strategic partnering strategy and we look forward to continuing our progress through multiple milestone events across our programs in 2020.
With that we would now like to open the call for questions operator.
Thank you.
The question at this time, you would need to press Star then one on your telephone to lift draw. Your question. Please press the pound Keith Please standby, we compile the culinary roster.
Our first question comes on the line of Laura Chico with Wedbush. Your line is now open.
Hi, Thanks, very much for taking my question I just have to for you I think you were mentioning in terms of the amendments that were filed a with regards to restore could you elaborate a little bit more on some of those specific changes I think specifically the exploratory measurements that you are going to be looking out and then maybe secondarily.
What gave FCS confidence to permit that change in the randomization schedule.
[laughter] Oh, thanks, Laura Yeah. So.
The main elements of the amendment really structures around.
HM two things one was going back to the FDA wed Oh, a proposal for they statistical analysis plan and.
Allison Dorval: Turning now to our financial guidance, we expect to end 2020 with cash and cash equivalents and marketable debt securities between $150 and $170 million. Based on our current operating plan, we expect this cash balance, along with the amount that we expect to receive for reimbursement of development costs from our NeuroCrim collaboration, will be sufficient to meet our needs for projected operating expenses and capital expenditures into mid-2022. We believe we have established a strong capital position through our disciplined financial approach and strategic partnering strategy, and we look forward to continuing our progress through multiple milestone events across our programs in 2020. With that, we would now like to open the call for questions. Operator?
It's incorporating the powering for efficacy, but also has a number of expirations safety. We came to an agreement on increasing the number of a study participants in Bristol one to 85, and then because the study has been active through a about a year or so with all say we had a number of.
Operational learnings from the conduct of the study at that approximately 20 sites that we have acted.
Given that this novel says I want to add to potentially adequate and well controlled trials to support the filing we decided to reduce the number of exploratory assessments that were being conducted.
These included things for example, a number of because that's where patients had to be in the Allstate, which I'll actually I'm comfortable for patients and so we we reduced the number of days.
Unknown Executive: Thank you. To ask a question at this time, you will need to press star then 1 on your telephone. To withdraw your question, please press the pound key. Please stand by while we compile the Q&A roster. Our first question comes from the line of Laura Chico with Wedbush. Your line is now open.
We remain that number or more detailed extraordinary here there are a psychological assessments that without any time consuming.
Oh, yes, yeah, I think well I think a century is the proposal based on the powering and safety database, which I've got them comfortable with the change in Randomizations game and the fact that the.
Laura Kathryn Chico: Thanks very much for taking the question. I just have two for you.
Laura Kathryn Chico: I think, Omar, you were mentioning, in terms of the amendments that were filed with regard to restoration, could you elaborate a little bit more on some of those specific changes? I think, specifically, the exploratory measurements that you are going to be looking at. And then, maybe secondarily, what gave FDA confidence to permit that change in the randomization schedule?
The amendment does not substantially change the eligibility criteria for enrollments in the study so that there isn't a significant changes a patient population that will be well enrolled under the amendment.
That's very helpful and I guess, maybe one follow up question then I'm just looking ahead to the longer term data that you mentioned out of the phase. One study yeah. I think one thing that we've struggled a little bit with was to put maybe some natural history contacts around the results. These are going to be you two and three year end points.
Omar Khawaja: Thanks Laura. The main elements of the amendment were really structured around two things. One was returning to the FDA with our proposal for the statistical analysis plan. Both incorporating the powering for efficacy but also the number of exposures for safety, we came to an agreement on increasing the number of study participants in Restore 1 to 85. And then, because the study has been active for about a year or so, we also had a number of participants who have been Operational Learnings from the conduct of the study at the approximately 20 sites that we have active. And given that this now will serve as one of two potentially adequate and well-controlled trials to support the filing, we decided to reduce the number of exploratory assessments that were being conducted.
I'm just wondering if you could point to certain population metrics or criteria that we should be using to help putting a little context around these results that are.
Anticipated to come out not coming months here.
Yeah, that's it I suddenly say there or you know that that was essentially two sources of data, which I think a helpful. So natural history. So one of looking at B. I control arms as well as a treatment arms or a other therapeutic approaches for example, deep brain stimulation or or other types of.
That's a magic formulations that studies that have collected long condensate had three to four years out from the initial on such treatment time I think.
Omar Khawaja: These included things, for example, a number of visits where patients had to be in the off state, which is actually uncomfortable for patients, and so we reduced the number of those. We also removed a number of more detailed exploratory neuropsychological assessments that were fairly time-consuming. The FDA, I think, essentially is the proposal based on the powering and safety database which got them comfortable with the change in the randomization scheme and the fact that the amendment does not substantially change the eligibility criteria for enrollment in the study so that there isn't a significant change in the patient population that will be enrolled under the amendment.
Those are going to be helpful for context, something so the 11 I won a three year data the second assessments of disease progression in all concerns and more natural history cohort [laughter] picky not just looking out the primary outcome measures such as diary will you pdrs, but other metrics.
But impact a patient close he has life and functioning side for example, a global disease a stage that modified done and you. All for example activities of daily living captured by the you can drs too and.
Omar Khawaja: That's very helpful. And I guess maybe one follow-up question then. Just looking ahead to the longer-term data that you mentioned from the Phase I study, you know, I think one thing that we've struggled a little bit with was to maybe put maybe some natural history context around the results. These are going to be, you know, two- and three-year endpoints. I'm just wondering if you could point to certain population metrics or criteria that we should be using to help put a little context around these results that are anticipated to come out in the coming months here.
The other.
Disease co Morbidities for example falls.
I think looking at natural history data sets, which out in the lunch I think will be helpful to get that complexity or the 11 I wasn't peso.
Thanks, very much guys.
Thanks, Laura.
Our next question comes from the line Charles Duncan with Cantor Fitzgerald Your line open.
Laura Kathryn Chico: Yeah, absolutely. So there are, you know, there are essentially two sources of data which I think are helpful for natural history. So one is looking at the control arms as well as the treatment arms of other therapeutic approaches, for example, deep brain stimulation or other types of dopaminergic formulations that studies have collected long-term data, three to four years out from the initial onset of treatment. And I think those are going to be helpful for context. So, for example, global disease stage, the modified hernia, for example, activities of daily living as captured by UPDRS2, and other disease comorbidities, for example, falls. So I think looking at natural history data sets, which are out in the literature, will be helpful to give context to the 1101 data.
[laughter]. Thank you hi, Andre and team congrats on a great your progress, especially partnering last year.
Wanted to ask a couple of more questions regarding their restore one change.
<unk> if you if we look at the drivers where there was there any new information I am I thinking mention operational.
Information that you could to the agency or are you able to enroll patients as you had anticipated or do you think that to the one is better than a one to one or is that not the driver to the change in restore mine.
Yeah no. Thanks.
The question so as we had guided last year.
We are we're looking at a 75 to 100 patients.
To get to the right level of the powering and increase of the safety exposure data on the program and.
Unknown Executive: Thanks very much, guys.
Unknown Executive: Thanks, Laura.
Charles Duncan: Thank you. Our next question comes from the line of Charles Duncan with Cantor Fitzgerald. Your line is now open.
Now with our partner with an character and we were able to map out the statistically what do we get from a one to one versus a two to one.
And Uh huh.
In discussion about the side I think we won't get some likely some benefit from the.
The proposition of having one in three chance versus wanting to chance of the sham surgery for the patients.
Charles Duncan: Thank you. Hi Andre and team. Congratulations on a great year of progress, especially partnering last year. I wanted to ask a couple of more questions regarding the Restore One change. If you look at the drivers, was there any new information? I think you mentioned operational information that you took to the agency. Are you able to enroll patients as you had anticipated, or do you think that a two-to-one is better than a one-to-one, or is that not the driver for the change in RestoreOne?
Enrolling in its into the trial. So they can only be a positive from the perspective have continued to enrollment, but it was a full review of the statistical plan and the safety exposure that let us to compare to the there was not much of a cost between the one to one and a two to one or two.
To justify sticking with the one to one as the first studies like this.
It makes sense and it seems to be a real positive in terms of.
Things forward could you could you see perhaps quicker time lines to two data on and how do you think about that.
Andre Turin: Yeah. No. Thanks, Chas, for the question.
Yeah. So we're going to provide an update once we get a little bit suffered during the implementation of the new products. All at the site level. That's a that's going to give us an hour partner more information to be able to map out a what the impact there would be on that completing a this study and getting.
Andre Turin: So, as we had guided last year, we were looking at 75 to 100 patients to get to the right level of powering and increase the safety exposure data on the program. And now, with our partner Neurocrin, we were able to map out statistically what we get from a one-to-one versus a two-to-one. And in discussions with the sites, I think we will likely get some benefit from the proposition of having one in three chance versus one in two chance of a sham surgery for the patients enrolling into the trial. So, that can only be positive from the perspective of continued enrollment, but it was a full review of the statistical plan and the safety exposure that led us to conclude that there was not much of a cost between the one-to-one and the two-to-one to justify sticking with the one-to-one for a study like this.
The data, but yes, we anticipate that.
As we discussed but investigators that a change in randomization scheme here.
And as the Omar.
Referred to.
Reducing some of the assessments that are more burden. Some on the patient may also even for further facilitate.
The decision to enter to study.
And my last question Andre is regarding restored to timing it seems like you've done a rigorous analysis some restore one.
Recently in discussions with agency I guess I'm wondering.
What are the rate limiting steps to kicking off for store to and can you provide any additional yet at least guide posts to think about the sizing of for store too.
Andre Turin: That makes sense, and it seems to be a real positive in terms of moving forward. Could you perhaps see quicker timelines for data, and how would you think about that?
Yeah no. Thanks for the other question. So we expect a will be in a position about our partner, but there are going to start. This at the end of the year generally speaking we expect that this study will very similar characteristics to restore one in terms of side.
Andre Turin: Yeah, so we're going to provide an update once we get a little bit further in the implementation of the new protocol at the site level. That will give us and our partners more information to be able to map out what the impact would be on completing the study and getting data. But yes, we anticipate that, as we discussed with investigators, a change in the randomization scheme here and, as Omar referred to, reducing some of the assessments that are more burdensome on the patient may also even...
Yes.
Design and endpoints. So we until we finalize a barrel call or we won't be getting more specific but we expect that it's going to be quite a similar the one aspect that we will look to do with restore too that goes beyond restore won it.
Andre Turin: And my last question, Andre, is regarding Restore 2 timing. It seems like you've done a rigorous analysis on Restore 1 recently in discussions with the agency. I guess I'm wondering, what are the rate-limiting steps to kicking off Restore 2? And can you provide any additional, at least, guideposts to think about the sizing of Restore 2?
As to include a number of international sites. So this study is intended to be a normal study, whereas restore one on the has a U.S. sites.
Probably good right now that Mr. One is only U.S. sites. So appreciate the added color and and pay down the progress.
Andre Turin: Yeah, no, thanks for the question. So we expect that we'll be in a position with our partner Neurocrin to start this at the end of the year. Generally speaking, we expect that the study will have very similar characteristics to the restoration one in terms of size, design, and endpoint. So until we finalize that protocol, we won't be getting more specific, but we expect it's gonna be quite similar. The one aspect that we will look to do with Restore 2 that goes beyond Restore 1 is to include a number of international sites. So the study is intended to be a global study, whereas RestoreOne only has U.S.
Thank you.
Thank you.
From the line.
Yep.
Pat.
H.C. Wainwright your line is now open.
Hi, guys Aaron on for Debjit Ah Thanks for taking the question so.
Quickly can you tell us what exactly the powering assumptions are for restore one on time and the baseline changed as expected from the feeble.
Yeah. Thanks for your question Aaron. So this study is powered to be or is it is size to be well powered for showing statistical significance.
Charles Duncan: Probably good right now that Restore One is only a U.S. site. So I appreciate the added color and update on the progress.
The an hour or more against.
Placebo so that's a that's.
That's how we ended up running our statistical analyses.
Unknown Executive: Thank you.
Debjit Chattopadhyay: Thank you. Our next question comes from the line of Debjit Chattopadhyay with H.C. Wainwright. Your line is now open.
So we're looking for something obviously as a pivotal study a registrational study that's going to be a statistically significant and clinically meaningful and and that's what we achieve with 85 patients would that the two 200 amortization based on.
Unknown Executive: Hi guys, this is Aaron Onford from DevJet. Thanks for taking the question. So, real quickly, can you tell us what exactly the powering assumptions are for RestoreOne on time and the baseline change that's expected from placebo?
I'll see as dawn and based on a prior results at the Weve shown in the open label setting.
Okay.
And then second quick question.
Andre Turin: Thanks for your question, Aaron. So the study is sized to be well-powered to show statistical significance for an hour or more against a placebo. So that's how we ended up running our statistical analyses. So we're looking for something, obviously, as a pivotal study, a registrational study that's going to be statistically significant and clinically meaningful. And that's what we achieved with 85 patients with that 2-to-1 randomization based on the analyses done and based on the prior results that we've shown in the open-label setting.
So looking at H T T. A data preclinical data it looks like to determine in college knockdown of Huntington may not be as much of a problem that's critical.
So is there a critical number below which you are trying to lowered the huntingtons protein in in those regions.
It area their concerns about lowering the wild type Huntington too much in the straight.
Unknown Executive: Okay, and then a second quick question. So looking at the HTT data, preclinical data, it looks like putamen and caudate knockdown of Huntington may not be as much of a problem as cortical neuron lowering. So is there a critical number below which you're trying to lower the Huntington's protein in in those regions? Or are there concerns about lowering the wild type Huntington too much in the striatum? At the end, yeah.
Yep Yep.
Intended.
Sort of question.
So Omar Oliver.
Yes. This question you know a you know it's a great question I mean anything.
All the sales as a reaching a little bit to try and understand.
What degree of clinical not sound as necessary to predict clinical benefit is having the challenge. It's been it's that the majority of preclinical efficacy data is being done in mouse transgenic models were.
Unknown Executive: Thanks for the question.
Omar Khawaja: Yeah, so Omar, if you can address this question,
Omar Khawaja: Yeah, you know, it's a great question. I think that, overall, the field is reaching a little bit to try and understand what degree of cortical knockdown is necessary to predict clinical benefits. I mean, the challenge has been that the majority of preclinical efficacy data is being done in mouse transgenic models where primarily the striatum is the tissue of interest, and that's where the efficacy sort of dose setting has been anchored. You know, I think it's a bit of a moving target. I mean, I think it's unlikely with any therapeutic modality but gene therapy that we would get 100% reduction of wild type and mutant Huntington in any particular brain region. I think for the cortex, we're currently aiming to get doses that would give reductions in mutant Huntington protein in broad cortical regions in the ballpark of about 20%, but it's not as definitively anchored as the reductions in the striatum, which are at levels of around 60% reduction based on multiple efficacy studies in transgenic animals.
Primarily this fight them as a tissue of interest and that's why I fixed fee.
Sort of dose that they can be noncash.
The you know I think it's it's a bit of a moving target I mean, like I think it's unlikely with any therapeutic modality, but with the gene therapy that we would get 100% reduction of wild type and meet in Huntington in any particular brain regions I.
I think for the Kautex, where are we currently aiming to.
Get does just that would get production, then and you can understand protein and broad cortical region than in the ballpark of about 20%, but it if it's not add.
Definitively on cut as the.
The the reductions in the slide them, which all.
At levels of around 60% reduction based on multiple efficacy studies subs and transgenic animals.
Okay, Great I was very helpful. Thank you guys.
Debjit Chattopadhyay: Okay, great. That was very helpful. Thank you, guys. Thank you.
Thank you.
Our next question from the line Jeff.
Jeff Hung: Our next question comes from the line of Jeff Hung with Morgan Stanley. Your line is now open.
Sadly your line is now open.
Hi, This is hana on for Jeff We were running what remains outstanding I had the Huntington di di and then what do you need to observational study to be included in it and then what you expect a one do you expect to begin the phase one study. Thanks.
Unknown Executive: Hi Hannah, this is Hannah on behalf of Jeff. We were wondering what remains outstanding ahead of the Huntington's IND, and then will you need the observational study to be included in it? And then what do you expect, when do you expect to begin the phase one study? Thanks.
Thank you Omar a young thanks to the question the <unk>.
Omar Khawaja: Thank you, Omar.
Omar Khawaja: Thanks for the question. For the IND filing, you know, we have a lot of bioanalytics data which are still coming in from our GLP studies, which are IND-enabling. And as we discussed on our last call, we are running the study out to 53 weeks, so that's an increased number of samples. And then we're looking at a number of bioanalytics, including the vector genomes, but also the microRNA itself, mRNA, and protein, and in multiple tissues and also brain regions. So at the moment, we're really going through that data and reviewing it and analyzing it. So that's the sort of...
The I'd be filing a you know we we have a lot of by analytics, which I'll still coming in from.
GLP studies, which I have DNA Blake.
And that as we guided on our last call. We all running the study out to 53 weeks. So that's an increased number of cell Bulls eye and then we're looking out a number of biospecifics from including the effects of genes, but also the Michael Jordan I itself, more and I am protein and in multiple tissues animals that brain regions say.
At the moment, we're really going through that time, reviewing its an eye on lighting it so that that.
Got to sort of.
Andre Turin: The piece that lies between this time point and the IND, the observational study is not gated on the timing of the IND, and so that is an active study set up now, and we anticipate that being active by mid-year, with the first patients coming into that.
Piece that lies between you know this type one and the I envy. The observation will study is not gates is on the timing of CRT D. And so that is then active study sites up now and we anticipate that I'd be accessed by a by mid year with with fast patients coming into that.
Andre Turin: We expect, just to add to that last point, we expect that in the observational study, we would have patients of a disease severity so that they would be quite similar to the ones to be targeted for our clinical trial of our therapeutics. So patients may also be eligible to participate in the study. They're not, as Omar said, it's not rate-limiting, it's not dependent, but we do expect that may be a barrier for patients to be able to participate in the study with the therapeutics.
We expect just to answer that last point, we expect a that observational study Oh, we would have patients of the disease severity. So that that would be quite similar to the wants to be targeted for.
Our clinical trial of.
Huh.
Or therapeutics, so with patients may be eligible to participate also win in the study there's not.
As as Omar said, it's not a rate limiting it's not dependent but we do expect that.
That may be a run in for patients to be able to participate in the study with the therapeutics.
Great. Thank you.
Unknown Executive: Great, thank you.
<unk>.
Unknown Executive: Thank you. Our next question comes from the line of Jim Bertinoff with Wells Fargo. Your line is now open.
Question comes from the line Jim.
With Wells Fargo. Your line is now open.
Hi, Thanks for taking the questions. This is it yet and doubting for Jim.
Jim Bertinoff: Hi. Thanks for taking the questions. This is Yanan dialing in for Jim. So first, just to follow up on the earlier question about powering, what is the assumption for placebo arms on time used in the modeling?
First just.
Just a follow up on the earlier question about powering what is the what was the assumption for placebo.
Arms on time, you've seen a in a modeling.
Andre Turin: Yeah, thanks, Yanan, for the question. So, there's a range of scenarios that were mapped against what has been seen in prior trials, including prior placebo effect and gene therapy trials at a similar time point, at one year. So, the modeling was based on some review of these and scenarios around them to get to the right powering. As we've shared previously, the range of the placebo effect against the good on time, which is our primary endpoint, has ranged from a fraction of an hour to an hour in a range of studies. So, in our modeling, we also incorporate scenarios that go to an hour plus of placebo effect to get to the appropriate powering.
Yep. Thanks, you answered a question so the there's a range of scenarios that were mapped.
Again, so to what has been seen in prior trials, including a prior placebo effect and the gene therapy trials at a similar time point at one year.
So the modeling was based on.
Huh.
Some review of these and scenarios around them to get to the right. The powering as we've shared previously the range of placebo effect against the good on time, which is our primary endpoint.
As ranged from a fraction of an hour to an hour in a range of studies. So in our modeling we also.
Incorporate scenario was that a.
Go to an hour plus of placebo effect to get to the appropriate bar.
Omar Khawaja: I don't know, Omar, if you want to add to this?
Well Mark you want to address no I think that's right. So we we made assumptions on a range for the potential to effect in the placebo arm.
Omar Khawaja: No, I think that's right. So we made assumptions on a range for the potential true effect in the placebo arm and then ran simulations to help us decide on the final sample size that would be necessary to capture that potential range of outcomes in the placebo effect.
And then ran simulations to help us decide on the final sample size that will be necessary to capture that that's attachment rates about in a placebo effect.
Got it very helpful and all are we restored to in terms I know you mentioned already the trial design, it's a yet to be finalized and you will communicate that later, but could we get a sense of whether the primary endpoint.
Yanan Zhu: I got it. Very helpful. And on Restore 2, I know you mentioned already the trial design is yet to be finalized, and you will communicate that later, but could we get a sense of whether the primary endpoint will be identical? I thought previously you kind of had some optionality of having Restore 1 read out and Restore 2 before Restore 2 is unblinded, so you have some optionality on Restore 2 and primary endpoint, but not sure. Could you update on your thoughts on that?
The idea cool Oh I thought previously.
Kind of have had to some optionality of.
Having restore why read out and restore to before we store to a his own blinded to fill that you had some optionality on every store to and primary end point, but not for could you update on a your thoughts on that.
Omar Khawaja: Yeah, I mean that situation still stands, so Restore 1 will be complete with top-line data before the need to finalize the SAP and database lock on Restore 2, so that optionality remains. I think, as Andre said earlier, Restore 2 will be substantially similar to Restore 1 in design and sample size, with the main difference being that it will incorporate a number of ex-US sites as well.
Yes, I mean that situation still that say that Bristol what will be complete with top line data before that need to finalize the SAP and database lock cornerstone to say that Optionality remains I think as Andre said earlier, we're still too, we oh well be substantially similar to.
Still one in design and sample size would that make different space that will incorporate a number of X U.S. sites as well.
Got it any potential for a competition of enrollment in the U.S. between the two trials when they were both.
Omar Khawaja: Got it. Any potential for competition for enrollment in the U.S. between the two trials when they're both running?
Omar Khawaja: Yeah, that's a great question. I think based on how we're seeing recruitment to date and also the number of sites that we're able to activate, we're not anticipating that there would be a risk of, you know, one study cannibalizing the other and that we'll handle this operationally. In other words, making sure that Restore One has priority for recruitment before sites are able to recruit patients into Restore
<unk>.
Yeah. That's a great question I think based on how we're staying recruitment to date and also the number one sites that were able to activate were not anticipating that they would be a risk or eight I want to study cannibalizing the other and that will handle that operationally in other words, making sure that was still one has a party for recruitment.
For a sites are able to recruit patients into the store too.
Got it. Thank you very much will take another question.
Yanan Zhu: Got it. Thank you very much for taking the question.
Unknown Executive: Thank you.
Thank you.
Jay Olson: Thank you. Our next question comes from the line of Jay Olson with Oppenheimer. Your line is now open.
Thank you.
From the line of Jay Olson with Oppenheimer Your line.
Hi, this is solvent.
Sylvain Turcan: Hi, this is Sylvain Turcan on behalf of Jay Olson. Thanks for taking my question and congratulations on the quarter. Could you just talk about some changes in regional disease management that you're looking at when designing Restore 2 versus Restore 1?
Thanks for taking my question congrats on the quarter.
Just to talk about some changes and.
Looking at.
<unk>.
<unk>.
Omar Khawaja: Yeah, so one of the things that will account for in the design of Restore2 will be variations in standards of care that go across geographies. You know, one of the big drivers of that is the availability of deep brain stimulation. And so what we're looking for, at least for the majority of Restore2 ex-US sites, are countries where deep brain stimulation remains part of the availability of care so that there isn't a complete separation in the management of patients. So that's going to be an important part of that. So as we look at Europe and Japan and other countries where we're really anticipating going to countries where deep brain stimulation remains an option for patients.
Yeah. So I mean, one of the things that will account for in the design of course to two will be variations in standard of had a standard of care that.
Got it across geographies.
One of the big drivers that that is the available if he has deep brain stimulation and so what we're looking for at least for the majority of restored to actually website or countries, where deep brain stimulation remains positive.
Availability of cast say that there isn't a complete separation and the management of patients. So that's good to be an important part of that so is as we look had a Europe and Japan in other countries, where were really anticipating going to countries, where deep brain stimulation remains an option for patients.
Great. Thanks.
Omar Khawaja: All right, great, thank you. And in terms of the preclinical data sets that you're still generating for your Huntington's disease IND filing, could you maybe tell us what there's left and, specifically, maybe what you may publish or present this year that could be useful for us to look at?
In terms of.
So do you still generating.
Huntingtons disease.
Maybe.
Maybe tell us what that's left.
Maybe one.
<unk>.
That could be useful for us.
Omar Khawaja: Yeah, absolutely. So, you know, we're really, at the moment, in the phase of completing and analyzing bioanalytics from the large number of samples that have been taken during the GLP studies up to 53 weeks. So it's a significant number of animals and a significant number of tissues that we're analyzing, as well as brain regions. So that's really where the
Yeah, Lilly said you'd I believe we're really at the moment in the phase or completing analyzing biospecifics from the large number of samples that had been taken during the GLP studies up to 53 weeks say, it's a significant number of animals and the significant numbers.
Tissues that were analyzing as well as brain regions like that's really why the teams activity is predominantly on the to the so the I'd be filing the second another preparations for the phase one as well as the observational study, which are now very advanced done. So that's a lot of team activity on that.
Omar Khawaja: The second are the preparations for the Phase 1 as well as the observational study, which are now very advanced, and so there's a lot of team activity on that. We would anticipate presenting our non-human primate long-term data from our GLP studies at an appropriate time, but the predominant focus right now is on the IND preparation and readiness for dosing a patient in Phase 1.
We would anticipate presenting our non human primate long data from non GLP studies.
I couldn't appropriate time, but the predominant focus right now is on the I'd preparation and readiness for dicing and patient in phase one.
Sylvain Turcan: Great! Thank you so much.
Great. Thank you so much.
Unknown Executive: Thank you. Our next question comes from the line of Brian Scorning with Bayard. Your line is now open.
Thank you.
[noise]. Thank you.
On the line.
Brian Scorning: Hi, this is Jack. I'm calling in on behalf of Brian.
With Baird Your line is not.
Hi, This is Jack long in for Brian. Thank you. So much for taking your question are we just have one really brief one on the sod one program I think previously you had indicated that you would look to partner that program I was wondering if you're just talking about your plans moving forward. There I know it's sold preclinical but are you looking to partner that are you going to develop that internally now moving forward.
Jack: Thank you so much for taking our questions. We just have one really brief one on the SOD1 program. I think previously you had indicated that you would look to partner with that program. I was wondering if you could just talk about your plans moving forward there. I know it's still preclinical, but are you looking to partner with that or are you going to develop that internally now moving forward? Thank you.
<unk>.
Yeah. Thanks for your question Jack.
Andre Turin: Thanks for the question, Jack. So we are in discussions with the parties about SOD1 but also broader efforts in ALS, and that's been our criteria in the mix of other discussions about corporate development efforts both on the inbound and potentially on partnering certain of our assets. We've been prioritizing our effort, but on SOD1, we continue to have some low-level activity as we've shifted the focus of our resources on advancing towards the HD, IND, and continue to have discussions with potential partners. As I said, it would potentially include some effort broader than SOD1, leveraging what we are learning from SOD1 to be able to apply to other forms of ALS.
So we.
Our in discussions with the parties about sod one but also brought her efforts in L.S. and that's been our criteria to in the mix of the other discussions about and corporate development efforts, both on the inbound and potentially on partnering certain of our.
That's.
We've been prioritizing our effort, but it on.
Sod one we continue to have some low level activity as we have shifted the focus of our Uh huh.
Resources on advancing towards the HD, Hi, Andy and continue to have a discussion with a potential partners.
Like I said it would.
Potentially include.
Some effort a broader than sort of one leveraging what we are learning.
From a sort of one to be able to apply to other forms of illness.
Awesome. Thank you so much.
Jack: Awesome, thank you so much. Thank you.
Thank you.
Andre Turn: Thank you. This concludes today's question and answer session. I would now like to turn the call back to Andre Turn for closing remarks.
<unk>.
Today's question and answer session I would now like to China.
Perfect. Thank you everyone for joining our call today and I look forward to the next importantly, we have to update you on our progress. Thank you.
Andre Turn: Perfect. So thank you everyone for joining our call today, and I look forward to the next opportunity we have to update you on our progress. Thank you.
Ladies and gentlemen.
Today's conference call. Thank you for participating you may now disconnect.
Unknown Executive: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
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Unknown Executive: .....