Voyager Therapeutics Inc. Q1 2023 Earnings Call
Okay.
Okay.
Good morning, and welcome to wager Therapeutics first quarter 2023 countries call.
All participants are in a listen only mode. There will be a question answer session at the end of this call.
Please be advised that this call is being recorded at the company's request.
A replay of today's call will be available on the investors section of the company's website approximately two hours after completion of this call.
I would now like to turn the call over to Pete pounds shoot.
Chief Financial Officer. Please go ahead.
Thank you and good morning.
Joining me on the call today are doctors.
Yeah.
Dr. Tom Carter, our Chief Scientific Officer.
We issued our Q1 2023 financial results press release this morning.
The press release and 10-Q.
You are available on our website.
We plan to provide a brief summary of key highlights on the quarter.
And reserve the majority of time for Q&A.
In a moment I will turn the call over to al.
Before I do that I.
I want to remind everyone that during this call OIBDA Representatives may make forward looking statements.
As noted on slide two of today's deck.
These forward looking statements include future expectations plans and prospects.
All forward looking statements are inherently uncertain and.
And are subject to risks and uncertainties that may cause actual results to differ materially from.
From those indicated by these forward looking statements.
You are encouraged to review and understand the various material risks and uncertainties facing the company.
As described in the company's most recent annual report.
On Form 10-K filed with the SEC.
As with the timing of today's quarterly report on Form 10-Q.
There has been no material changes to the risk factors described in our interim report.
All of our SEC filings are available on the company's website.
Now it is my pleasure to turn the call over to Hap.
Thank you Pete and good morning, everyone.
Please turn to slide three.
I'd like to take a moment to recognize the incredible innovation happening right now in Europe Therapeutics and gene therapy.
We believe we are witnessing a renaissance in neuro therapeutics Jeff.
Jeff This year, the second disease modifying therapy for Alzheimer's disease received accelerated approval.
And the first drug approved for <unk> ataxia.
We've seen breakthroughs in treating negative symptoms of schizophrenia, something for which there are no approved therapies.
Just two weeks ago, the FDA granted accelerated approval to an antisense oligonucleotide for <unk> amyotrophic lateral sclerosis.
Importantly, the FDA basically approval in finding the treatment driven redemption.
And they are reasonably likely to predict clinical benefit in <unk> ALS patients, establishing a precedent for a biomarker based path to accelerated approval.
I Hope this will drive further new treatments for patients suffering from this devastating disease.
At the same time, the gene therapy field is coming of age.
We have recently seen the FDA approval of the first gene therapy for hemophilia B.
Gene therapies operating important potential advances in treating duchenne muscular dystrophy and hemophilia a are approaching produce the dates and through that we may see the accelerated approval path utilized for gene therapy.
Importantly, long term data on <unk>, one of the first gene therapy approved recently demonstrated durability of effect after seven five years, which positions have called transformational.
Voyager sits at the intersection of narrow therapeutics and gene therapy, and we believe we are uniquely positioned to leverage the investments in both fields.
To date, the delivery of gene therapies into the central nervous system has proven challenging.
Approaches to inject these therapies into the brain correct, our various CSF spaces have not been very successful.
Soldiers tracer capsid discovery platform is the foundation of our approach to solving this delivery challenge.
<unk> scientists have engineered multiple capsid libraries, each with more than 20 million novel variants of AAV nine an AAV capsid.
Select those novel capsid that display greatly increased transaction in the central nervous system following intravenous delivery.
We have leveraged the capsid to advance our <unk> and our partners CNS gene therapy programs.
Several of which are now advancing towards clinical trials.
This is our Voyager is enabling the future of neuro genetic medicine and from where I sit it's an incredibly exciting place to be.
Moving to slide four I will briefly review our investment rationale.
Our first pillar of value is our tracer capsid discovery platform, which I just discussed.
And the preclinical studies, our novel Capsid delivered intravenously.
Demonstrated more than 100 call higher transgene expression in the brain compared to conventional AAV nine cap suits.
We have shown high levels of CNS gene expression at low doses and we have demonstrated the ability to target specific cell such as neurons are up while <unk> targeting the liver and dorsal root ganglion cells.
Look forward to sharing more data on our capsid at <unk> later this month.
Yes.
Our second pillar of value is our CNS pipeline.
We are advancing four programs through late research and towards R&D.
Two of these programs are wholly owned our humanized anti Tau antibody for all time, this disease and <unk> gene therapy program for AOS.
The other two are GBA, one gene therapy program for Parkinson's disease, and our pretax in gene therapy program for <unk> ataxia are being co developed with Neurocrine.
Our third pillar of value is partnerships, we completed <unk> option and license agreement with Pfizer and Novartis.
<unk> executed strategic collaborations around our pipeline programs with Neurocrine.
And we are exploring more such transactions.
Turning to slide five we continued to make progress advancing our CNS pipeline.
I'll note a few recent highlights.
During the first quarter, we selected a lead humanized anti Tau antibody candidate <unk> for the treatment of Alzheimers disease.
In March we presented new data at the <unk> meeting highlighting the differentiating characteristics of this lead candidate.
Last month, we received pre IMD written feedback from the FDA for <unk>.
Voyager continues to expect to initiate GOP toxicology studies this year to enable an IND filing in the first half of 2024.
Another change this quarter was to the timeline for our <unk> gene therapy program.
Voyager previously said, we expected to identify our lead development candidate for this program in the first half of this year that has moved out to the second half of this year as we continue to evaluate data from this program to identify the optimal development candidate.
We plan to provide updated guidance on the RMB timeline once we select a development candidate.
Given where we are today, we expect the RMB to occur in mid 2025.
Our pretax <unk> gene therapy program for pre Brexit back yet and our TBA, one gene therapy program for Parkinson's disease, and other GBA one mediated diseases, both continue to advance in collaboration with Neurocrine.
I am pleased with the progress we're making here.
Additionally, during the first quarter, we launched two new early stage gene therapy program, combining <unk> with our novel intravenous tracer cabinets.
One combined to <unk> enabled specific knockdown of <unk>.
HCP and Msas III for the treatment of Huntington's disease.
The other uses <unk> to reduce tau expression in the brain for the treatment of Alzheimer's disease.
I'd like to now turn the call over to Pete <unk> to discuss our financial results for the quarter.
Thanks Al.
I will cover some key financial points on this call and refer you to our press release and 10-Q issued today for further details.
Please turn to slide six.
We announced Q1 2023 collaboration revenue of 175.
Composed of $69 5 million from the.
2023, nurturing collaboration agreement or the GBA one program.
$79 million from the Novartis option exercises and $2 million from the 2019 Neurocrine collaboration agreement activities.
Our R&D expense was $18 6 million.
An increase of $4 2 million as compared to Q1 <unk>.
Two drew.
Driven by increased head count.
Increased program related to R&D spend.
And milestone fees.
And offset by decreased facility costs.
Our G&A expenses were $9 million for the first quarter of 2023 as compared to $7 7 million for the same period in 2022.
The increase in G&A expenses was primarily a result of increased compensation costs driven.
Driven by head count increases.
As a result of strong revenues in Q1 2023, the company had net income of $124 million.
Resulting largely from our collaboration revenues.
As well as increase of one.
$1 8 million in other income.
Due primarily to increased interest revenue from cash marketable securities.
Regarding the balance sheet, Voyager reported $273 3 million in cash cash equivalents and marketable securities.
At the close of March 31, 2023.
We also had a receivable at the close of Q1 2023 from Novartis is $25 million option payment received in April .
Together this resulted in pro forma cash cash equivalents and marketable securities.
$298 3 million for the close of the quarter.
Of note deferred revenue increased by $18 7 million.
Related primarily to the upfront payment from Neurocrine allocated to the three new discovery programs of $74 4 million.
Offset by the recognition of $54 million in revenue from the Novartis option to exercise.
The company has a sound balance sheet.
Enabled by our non diluted collaboration revenues.
We expect that our balance sheet plus expected reimbursements.
We will be sufficient to meet our planned operating expenses and capital expenditures into 2025.
I will now turn the call back over to al. Thank you Pete.
Turning to slide seven as you can see Voyager has had a productive start to 2023 we.
We began the year by securing a $175 million upfront payment in strategic collaboration with Neurocrine Biosciences, followed by Novartis is opt in decision on capsid to two neurologic disease targets, triggering another $25 million payment.
These transactions have strengthened our balance sheet and enabled us to further advance our platform and pipeline.
As discussed we selected a development candidate for our anti Tau antibody program for Alzheimer's disease.
We aim to select a development candidate for the AOS and <unk>. One program later this year and we launched two new early stage gene therapy programs for Huntington's disease and Alzheimers disease.
In addition, I am thrilled to welcome George scan goes to our board of directors as we announced in our press release this morning.
George just wanted to most accomplished executives and the entire biotech industry, having served as CEO of beer Biogen and <unk>.
Is that the experience building biotech company that deliver a highly innovative therapies to patients while creating value for shareholders will be invaluable to us as we strive to develop voyager into a leaner and narrow genetic medicine.
Looking forward, we continue our work to breakthrough the barriers constraining the field of gene therapy and neurology.
We will continue to share the exciting data we are generating at scientific conferences, including at GCT later this month.
Importantly, our pipeline advancement is leaning towards what we view as multiple opportunities for value creation.
As we look towards 2024 and 2025, we anticipate the potential for multiple RMB filings across our wholly owned and collaborative and our licensed programs.
This translates into multiple opportunities to earn milestone payments and even more importantly, as clinical trials began several shots on goal to establish human proof of concept for our novel capsid.
Furthermore, there is potential to see early biomarker based evidence of disease impact and some of these very difficult CNS indications.
And of course, we continue to engage in active discussions with potential partners around our platform and pipeline.
In summary, it's been a great start to 2023 and as always it is all due to the incredible Voyager team.
I look forward to continuing our momentum throughout the year.
With that we're happy to take any questions you may have operator.
Thank you.
To ask a question. Please press star one on your telephone.
And as well wait for your name to be announced before you actually asked a question one moment, while we prepare our Q&A officers.
The first question that I have today, we will be coming from Jay Olson of Oppenheimer. Your line is open.
Oh, Hey, congrats on all the progress and thank you for taking our questions.
We're interested in the work Youre doing on Tom.
You are advancing an anti tau antibody in initiating its Cao knockdown gene therapy program and could potentially have a vector is anti tau antibody can you just talk about how your optimized.
Development strategy and prioritize all of your different Tao targeting modalities and then eventually do you think that anti Tau therapies can be mono therapy or do you think there could.
Could be combined with anti a beta therapies and then finally can you just talk about.
How you balance the two.
Tradeoffs of advancing your Tau therapies independently versus seeking a partner. Thank you.
Thanks, guys. Those are great questions. So first of all what we like about towers that we believe.
Very important involve validated target for Alzheimer's disease.
And because of that we're taking multiple approaches against this target.
And.
As you pointed out we have an antibody against the C terminal domain apparel.
The development strategy there is to move forward with the.
The passive antibody one.
And see whether or not lead but we get proof of concept in other words to see whether or not we can block the spread of power by looking at him.
Tau pet scan.
We think this is very feasible and we can do it with not that many patients over a one year period.
If we obtain proof of concept that we have.
A couple of choices there.
One is we can continue to proceed with the antibody to power all the way through to approval or we add add or we can initiate a vector is anti tau antibody program.
So that would be the.
Sort of.
Crossroads that we will see there.
In addition, we have bids vector is tayo knockdown, which we think is an additional short against the very important targets.
Yes.
This will be different in the sense that it doesn't rely on antibodies by binding to extra sales.
This is a this approach is to decrease the expression of all forms of Tau intracellular and exercise are there and we saw some preliminary evidence of that approach at the <unk> meeting.
With key Biogen ion is active against apparel, where they were able to see effects on Tau pet imaging.
So we think it's a very important target, we think having multiple approaches.
Thats helpful against this target and we think we can obtain proof of concept pretty rapidly in the clinic.
Taking advantage of Tau pet imaging.
The final question relates to whether or not we're going to partner.
<unk>.
We're always talking to partners and we're always open to anything.
We could go to proof of concept by ourselves we have the capability to do that and we I believe we have the resources to do that however.
We're open to anything.
And.
But ultimately I can't imagine that we would go all the way to commercialization for a disease as large as all segments would be so we will need to partner that program. The question is when and we have some choices there.
Finally, your question about combination.
Or a monotherapy I believe.
I think we just started the <unk>.
Era of disease modifying therapies for Alzheimer's disease lift the approval accelerated approval of two anti a beta antibodies as we all know the efficacy modest and the 25% to 30% range on the CVR sum of boxes.
We'd love to get better treatment effects, and one approach might be to combine.
With a tile reducing approach, whether it's an antibody or knockdown.
And so I think that we're going to.
We are likely to test combination treatments.
Because I think one Tao spreading starts to occur you may need to address how independently separate from the anti amyloid antibodies or in combination with them.
So I hope that answers all your questions Jay.
That was perfect. Thank you so much for taking our questions.
Thank you.
Thank you one moment, while we prepare for the next questions.
And our next question will be coming from Jack Allen again, please wait for your name to be called before you ask the question. Your line is open.
Jack Allen your line is open.
Oh, sorry about that I was on mute. Thank you so much for taking my questions and congratulations on all the progress I wanted to stick with the <unk>.
<unk> as well and I wanted to ask al.
Sure you are aware of the Eli Lilly results from that recent study, where they measure Tau and stratify patients by Paul I was wondering if you had any early thoughts on a specific population within the Alzheimers disease group that you'd look to enroll in any comments you have around the differential effects. It seems like you guys.
You saw in there higher versus intermediate tower cohorts.
The hijacked that's a really interesting question I look forward to seeing the data at a scientific conference, but from what I gather the people who had the higher Tau burden when they started the treatment had less of a treatment effect.
And I think that underscores I think but I was just saying that we may need to combine with a tower.
Tayo approach a <unk> targeted therapy.
And.
And so in terms of the population.
Even by even when we get enrolled patients with mild cognitive impairment, which is the early symptoms the earliest symptomatic disease.
Liza is pretty far advanced in terms of molecular pathophysiology right I mean, not only is it.
As amyloid and accumulating for up to 20 years and have reached essentially a maximum burden in the brain. We see Tao has already started to progress at many of these patients and so I think that the.
Even when you do the combination youre going to need to go to an early stage of patients at least Mci and maybe even earlier.
Got it great. Thank you so much for that insight and then I was also wondering if you had any comment on the competitive landscape as it relates to powered any other programs are closely monitoring the early proof of concept stage.
Well I think that.
There are a number of mid domain targeted antibodies that are now approaching.
Readouts from efficacy trial to my understanding.
So that will be very interesting in our hands, we had multiple mid domain antibodies that we could have humanized ourselves and our hands. They werent as consistently effective and the spreading assay in the animal studies, where we take human pathological tayo inject them into the animals and look at the spread of pathological towers.
So so we're very hopeful that our <unk> antibody use remains differentiated but I think it would be wonderful to see.
Efficacy with the mid domain antibodies. These alzheimers disease patients need something additional I believe in addition to the anti amyloid therapies.
And just wanted to add that debt from our studies at least in our preclinical models, it's pretty clear that the epitope matters.
So there Nathan in antibodies.
Of our own network.
Antibodies and Delaware are C terminal antibody as Al mentioned gave us the most robust and strongest effect. So I think that as we've seen these antibodies come to the clinic it will be very informative.
Field to learn hopefully what works and then meeting with us.
Great. Thank you, so I'll sort of color and congratulations again on the progress.
Thanks Jack.
Thank you.
One moment, while we prepare for the next question and our next question will be coming from CL Naidu.
TD Cowen.
<unk> Your line is open.
Good morning, Thanks for taking our questions and <unk> congratulations on the progress a couple from us keeping on the tower Fame.
In terms of the Tau antibody that you've chosen as the development candidate how does that antibody compare to the one that produced the data I see 2022 is it simply the humanized version.
Of that prior antibody or are there any other changes.
Sure.
It's the so we have shown data on a number of antibodies.
Youre talking about from a IC included.
Data from multiple panels of antibody is different epitopes. The antibody. We're taking forward is a humanized version of lead antibody from that.
That presentation.
It's a humanized version of the antibody that produce the 71% to 74% declines in.
Yes, yes, okay.
That's helpful and then second on the.
GOP talks it sounds like Youre going to complete the GOP talks and be able to file an IND within a year.
It strikes us as faster than average how can you get that done. So quickly is that based on feedback from the FDA or just prior experience.
It's both actually.
<unk>.
There have been other humanized antibodies against pathologic proteins. The tricky part here is that the protein.
The target for the antibody is not expressed in wild type animals, which is typically with Houston tox species talks pizza right.
So in order to look at on target toxicity, you have to look in animal models, where that's the only place that you have the pathological tower.
Being expressed.
Those models.
Animals don't do well.
I mean die prematurely because of the disease and so you have to work within the limitations of that but so we do have experience with humanized antibodies against pathological proteins in the brain and so we've drawn on that experience, but also we have FDA feedback.
And so are our preclinical toxicology plan is based on both.
Great and then one last housekeeping question for Peter Peter the $25 million milestone that was received in April was that all can be booked in Q2 versus because theyre going to be an amortizing product.
So we from a Rev. Rec perspective recorded that obviously in Q1, but cash is in Q2.
Perfect.
You bet and the numbers got it thank you.
Thank you.
While we prepare for the next question.
And our next question will be coming from <unk>.
I'm, sorry, Xeon Xu of Wells Fargo. Your line is open.
Hi, Thanks for taking our questions.
And perhaps how question in a soft one ask question so for Tao.
How would you think about the criteria for success.
Once you.
Get the Tau antibody pet imaging data.
Given the findings.
How antisense as you mentioned before.
I am guessing mainly if tau reduction.
Pet is less potent.
Then if there is still a reason that the antibody approach can be a reason that led that modality to be further pursued.
I'm sorry, the way. The question is could you give more color on the delay for the nomination of the candidate is it more about payload or vector optimization or some other reasons. Thank you.
I think those are great questions I'll take the first one and Todd will take the second one on the tower.
Well first of all the antisense oligonucleotide is injected into the vehicle.
And so and our antibody as an intravenous drug that we're going to inject every four weeks. So that's a pretty big difference right. There in the mode of administration.
We will obviously compare to two others that are showing data on Tau, we do have a minimum product profile that takes into account our competition.
And so we will move forward. If we have first of all we have to have a statistically significant reduction of the spread of the tower and then well compared to competitors and see if there is there is space for <unk>.
Additional treatment.
Just keeping in mind that the different modes of administration that the higher ease of use use of intravenous antibody.
And the fact that.
Sure.
I think there is room for more than one treatment.
As a physician I always liked having multiple options for my patients.
Todd.
Yes, so on <unk> as you pointed out.
Development candidate in therapy is the combination of the cats. It ended a payload.
<unk>, we're continuing to work and evaluate the trans genes in our cabinets.
<unk> continued to perform very well.
Anybody on the call to check out the HTC key presentations next week, we have a couple of.
Symposium presentation, and oral presentations and posters on our novel assets.
Seeing quite reproducible results from continued performance across both our print area.
We're trying to do is identify the optimal payload.
The optimal caps it to meet our candidate criteria.
And we hope to do that in the back half of this year.
Got it thanks for taking my questions.
Thank you one moment, while we prepare for the next question.
And our next question.
We will be coming from Laura Chico of Wedbush. Your line is open.
Good morning, Thanks, very much for taking the question I guess staying on the Pal team I wanted to ask it.
With the upcoming reimbursement decision for the anti amyloid antibodies is there any learnings there that Kevin and form or perhaps.
Change your development strategy for <unk> and then one for Pete can you just remind us. It's obviously been a big year in terms of milestones coming in the potential for any remaining milestone payments in 2023. Thanks guys.
Thanks, Laura on the reimbursement question Thats, a really important question and we have a lot to learn I think as the year goes on this year.
I think we have to be mindful of the total number of patients that are going to require treatment and we don't want to do anything that will be.
Unhelpful to society, we want to help patients and we want to make sure patients have access to the drug.
So I think theres a lot for all of us to learn in the coming years.
So, but yes, I think youre pointing to that to the concept that if theres a combination treatment. That's too that's two drugs that have to be reimbursed and so we'll have to take that into account.
Yes, Laura with regards to your second question. So we.
We don't provide a lot of forward looking guidance with regards to future milestones I know, we've shared publicly the milestones relative to all of our partnerships and relationships that we have an aggregate and kind of put some breakdown but.
Hello, Thats been redacted.
I can say at this way.
With regard to our existing relationships there are some potential milestones that we could be getting.
More of a near term I would say it that way.
But again, we don't guide specifically with regards to those and so I think that's probably where we would leave it for now hopefully thank you Sir.
Yes, thank you very much guys.
Thank you Sam.
The next question.
And our next question will be coming from Joon Lee of Trust security.
Jeremy Your line is open.
Hey, hi, thanks for taking our questions.
For the Huntington's program. It's interesting that you are taking both an allele specific again non allele specific approaches with Smith MSH targeting can you elaborate a little bit on your strategy here and do you plan to include multiple <unk> and a similar cost structure address multiple sniffs.
And would you also combine SNP as well as on the MSA to let's say the construct.
Yes. Thanks.
I'll start and I'll ask Todd to fin.
Finish the answer.
So I would say that in some ways folks are kind of the nobody else specific both targets because MSH three targets the expansion.
Only occurs off the mutant.
And obviously, the <unk> specific mutant Huntington targets.
This is an enviable specific approach so in some ways I look on it as both are kind of.
Preserves both targets.
Targeting both of these were preserving the expression of wild type Huntington, which I think is the goal.
Because I think theres a lot of concern that we need to preserve wild type Huntington expression. So so so and then yes, I mean I'll, let Todd.
Answer, but our aim is to combine both.
Alright back to rise, thus Rnas into the same vector so.
Yes, so thats right out so we did plan, Jim and incorporating OSA RNA that.
The payloads for the <unk> is relatively small so that's not a problem to hit multiple.
A single vector.
We can drill devices trying to approach.
Now that said we can explore.
Valuate the independently as well so thats always an option that continues to move forward with.
We plan on going after MSH Street Pam <unk>.
Pan manner, which would affect the.
<unk>, specifically and then the approach to the allele specific Huntington is a sip based approach.
10, offset down in Illinois.
So Jay you are right.
Net based approach, we're not going to be able to treat every single Huntington station <unk>.
Start with the most commonly yields.
And then ultimately we may need to do a second.
Youll specific MH.
Http.
Program and so forth.
Okay.
Got it. Thank you. Thank you and I have a follow up on the TARP program.
You have some other.
People ability of multiple programs multiple shots on goal.
<unk>.
It does approach to targeting extra solar species as well as the interests of our species with the SRA is there one species and your view of that is probably more important to address.
Let me say, both but which one has more like.
This is as a more dangerous pathogen anymore.
Well I wish I knew the answer to that question I think that's part of the complexity here.
There are multiple forms if you will.
Tao in the extra cellular space.
And.
It's pretty certain that targeting just began terminal containing.
Extra cellular species or probably it doesn't work because the multiple approaches have been tried for those.
I think that I don't have an answer maybe Todd.
With the pathologic species.
I doubt that.
No.
More along the line yes.
Yes, more along the lines of.
Intracellular versus out yourself, because youre SRA approach type of target I believe interstellar species preferentially.
Yes.
Well that actually will target both I mean, it will target the synthesis of all forms of power. So if we decrease we will definitely increase intracellular which the antibody probably won't be able to do very much at all.
But it will also the events. They will also target extracellular tau too because that's the source of extracellular Tau is intracellular tau.
Okay.
Would you would you say that the <unk> would have a little bit higher probably a success story that is still TBD.
Yeah, but.
<unk> approach is going to have its own benefit risk profile and mode of administration issues and things So I think.
I think it's still too early to know to be certain which approach is going to be optimal for patients.
I think it's such an important target we wanted to go after it with multiple approaches.
Yes.
Mhm.
Thank you one moment, while we prepare for the next question.
And our next question will be coming from.
Sumit.
So excuse me Killarney.
Canaccord Your line is open.
Good morning, Thanks for taking my question and nice to see all the progress that the company had what clearly an exciting time to be targeting the specific conditions that you are.
So I have a question on your <unk> gene therapy program now that 2% is available.
The landscape has changed with respect to clinical trial recruitment and is there any medicine government specific preclinical model in combination with 2%.
Okay, I think I heard the first question.
I'll start and then im not sure I heard the second one but I think the first question relates to the feasibility of recruiting patients in the study.
Person being approved and I think there is first of all it's wonderful that patients with a terrible disease treatment options. So congratulations to biogen and ion is forgetting that across the finish line.
But for us.
We could either take patients who are already on per person and see.
If they require.
And the gene therapy, and see if the requirements for a person is decreased perhaps.
Two zero, but it could just be decreased.
And we can track neuro filament marker to know whether or not the treatment is adequate for example, another approach would be to go to untreated patients there may be parts of the world, where it's hard to access the person.
And so and so and then a third approach would be to go very early even before symptoms.
To get rid of the toxic gain of function autosomal dominant mutation. So those are the three approaches we are considering now.
Our prime will tell whether which one of these.
Best option, but I think it's.
But as I alluded to in my comments.
Fact that neuro thorough mint is considered.
By regulators that lease FDA as a validated surrogate.
You can measure it only helps in terms of making the development more feasible I believe.
And then I can get.
You may have to repeat the second question, because I'm not sure I heard it.
The second part of that was is there any embedded in running a specific make them Marty <unk> in combination with 2% to optimize the alright.
Hi.
So the question was whether we're doing.
Preclinical studies so there are a couple.
Couple of <unk>, one transgenic mouse models G 93, <unk> hundred 37 are et cetera.
We could.
Due to the combination study.
In vivo, but.
<unk>.
I'm not certain that that would be necessary before we did the study in patients.
We have a very good broad based biomarker that we can use to monitor patients.
In terms of the amount of injury to motor neurons.
And some of these animal models theyre not very predictive of what happens in the clinic.
Worry about relying too much on these animal models.
Got it thank you.
Thank you.
You're going to see we would like to ask a question. Please press star one one on your telephone.
The next question is coming from Jack Allen.
Baird Your line is open.
Great. Thank you so much for taking the follow up I just wanted to reach out and see if you have any comments around that.
On a non human primates.
Earlier this year there was a.
Ban importation of these critical research assets and I wanted to gauge your.
Awareness of this and any comments you have as it relates to your preclinical programs that you have ongoing.
Hi, Jack it's Todd So that's a great question I think the entire field.
<unk> very carefully we certainly are watching the nonhuman primate availability quite closely.
To date, we have not been adversely affected by nonhuman primate availability we've setup.
Agreements with multiple vendors to make sure we have options in case of initiatives should arise.
And all I can say is that we're giving our best to mitigate any risk and watching very closely.
Great. Thank you so much.
Thank you.
Includes today's Q&A session there are no more.
<unk> is in the queue I would like to call turn the call back over to Doctor Al Sandrock for closing remarks.
Thank you everyone for joining us today and for the great questions.
Feel free to follow up with US directly if you have any further questions. Thanks again.
Yes.
Thank you everyone for joining today's conference call. This concludes today's event you may all disconnect and everyone have a great rest of your day.
Okay.
[music].
Okay.