Q3 2023 Voyager Therapeutics Inc Earnings Call
Okay.
Okay.
Good afternoon, and welcome to the Voyager Therapeutics third quarter 2023 conference call. All participants are in listen only mode.
A question and answer session at the end of this call.
Be advised that this call is being recorded at the company's request.
Today's call will be available in 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 Beach Bundschuh Chief Financial Officer, you may begin.
Yeah.
Yeah.
Yeah.
Yeah.
Yeah.
Okay.
Sure.
Thank you and good afternoon.
Joining me on the call today is Dr Al Sandrock, our CEO.
And joining us for Q&A.
Todd Carter, our Chief Scientific Officer.
We issued our Q3 2023 financial results press release this afternoon.
The press release and 10-Q are available on our website.
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.
Boyd Your representatives may make forward looking statements.
Noted in slide two of today's deck.
These forward looking statements include future expectations prospects.
Implants.
All forward looking statements are inherently uncertain and are subject to risks and uncertainties that may cause actual results to differ materially 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 of the filing of today's quarterly report on Form 10-Q.
There have been no material changes to the risk factors described in our annual report.
All of our SEC filings are available on our company's website.
Now it is my pleasure to turn the call over to al.
Thank you Pete and good afternoon, everyone.
Please turn to slide three.
I'd like to start by briefly reviewing voyager's investment rationale platform pipeline partnerships and potential.
First the platform the delivery of gene therapies into the central nervous system or CNS.
Historically proven challenging.
<unk> is working to solve this delivery challenge with our tracer capsid discovery platform.
We have generated multiple families of novel capsid.
With robust CNS tropism following IV delivery.
We believe our captains are best in class because we have demonstrated high transduction in multiple brain areas at relatively low doses with <unk> targeting of the liver and dorsal root ganglion neurons.
We have also shown the ability to target neurons and glial cells blood brain barrier or BBB pennant trends across multiple species.
And an identified receptor that is also expressed in humans.
Second our pipeline.
We are advancing two wholly owned and two partnered CNS programs through late stage research and towards IND filings.
The most advanced is our anti Tau antibody program for all timers disease for which we anticipate an IND filing in the first half of 2024.
We were encouraged by the data presented by some of our peers at the recent clinical trials on all timers disease meeting.
Which provided for the first time and early clinical evidence showing that <unk> targeting therapies slowed cognitive decline.
These data strengthened our conviction in the value of Tau as an important therapeutic target for Alzheimer's disease.
Behind our anti Tau antibody, we have multiple opportunities to advance gene therapies enabled by our novel tracery capsid into the clinic in 2025, including potentially our wholly owned <unk> one program for amyotrophic lateral sclerosis or ALS.
Third partnerships boy.
Voyager has generated more than $200 million this year alone and non dilutive partnering revenue.
We currently have 11 partnered programs, which provide opportunities to achieve milestone and royalty revenue to generate data with our capsid and most importantly to help patients.
Finally.
Potential.
Specifically the potential to expand from gene therapy into other approaches of narrow genetic medicine.
We have identified multiple receptor is associated with our capsid families.
We are exploring the potential to leverage one of these which we call receptor acts to shuttled non viral genetic medicines across the blood brain barrier.
I am increasingly excited about the potential here to expand the reach of our technology into other approaches of narrow genetic medicine.
Moving to slide four.
As you can see Voyager is advancing quite a robust pipeline.
However, we are doing so efficiently.
Our wholly owned programs at the top of the slide the noted in Orange are the only programs we fund that.
The rest of our pipeline is funded by our partners.
During Q3, Voyager focused on advancing our prioritized pipeline programs towards the clinic.
We initiated GOP toxicology studies with V why Tao zero, one or humanized anti Tau antibody for Alzheimer's disease.
This program is on track for an IND filing in the first half of 2024.
We also continued to advance our <unk> gene therapy program for ALS.
This program is on track for a development candidate selection before the end of this year to support an IND in mid 2025.
As we move forward towards the clinic, we are thoughtfully building clinical expertise within the Voyager team, including in regulatory affairs toxicology and development operations.
I will make one more note before we leave this slide.
In September Alexia on Astrazeneca rare disease.
<unk> It had completed a definitive purchase and license agreement for our portfolio of preclinical rare disease gene therapies.
And enabling technologies from Pfizer.
The portfolio includes the license for a novel capsid generated from our trade surplus that form to enable a potential gene therapy program exclusively for an undisclosed rare neurologic disease target.
The assignment does not impact the terms of the licensing agreement.
We are thrilled to have them as a partner on this program, particularly given their public commitment to advancing next generation genomic medicines.
Turning to slide five as you can see Voyager continues to execute on our milestones.
So far this year, we secured partnerships with Neurocrine Novartis and Sangamo.
The company is well capitalized with approximately $253 million in cash on our balance sheet.
As of the close of Q3 2023.
We expect our cash cash equivalents and marketable securities along with expected reimbursements under the Neurocrine collaborations and interest income to provide runway into mid 2025.
We selected a development candidate and then initiated GOP toxicology studies for our anti Tau antibody program for all Cypress disease.
And we launched launched three early stage gene therapy programs.
One for Huntington's disease, and two for Alzheimer's disease.
Looking forward, we continued our work to advance neuro genetic medicines.
We expect to identify a lead candidate for our wholly owned <unk> <unk> gene therapy program by the end of this year.
As we look towards 2024 and 2025, we anticipate the potential for multiple IND filings across our wholly owned and collaborative and or licensed programs.
This translates into multiple opportunities to earn milestone payments and even more importantly, once clinical trials began several opportunities to establish human proof of concept for our tracer captives.
Furthermore, there is potential to see early biomarker based evidence of disease impact and some of these very difficult CNS indications.
We continue to engage in active discussions with potential partners regarding collaboration and licensing arrangements around our platform and pipeline.
In summary, Voyager continues to advance our lead programs towards the clinic, while maintaining our robust platform and early pipeline programs.
We look forward to continued execution this year and next.
I'd like to thank the wonderful employees at Voyager for their hard work to keep everything moving forward.
With that we're happy to take any questions you may have.
Operator.
Thank you, ladies and gentlemen, we like to ask a question. Please press star one on your Touchtone telephone.
For your name to be announced to withdraw your question you May Press Star one again please.
Please standby, while we compile the Q&A roster.
Okay.
Yes.
Sure.
Now first question is coming from the line of.
Jim Lee with <unk> Securities. Your line is open.
Hi, Thanks for taking our questions.
Congrats on the progress.
As you consider nominating.
Genetic medicine.
<unk>.
Can either do so with the.
Capex that you had or choose to use the so called receptor acts that you've discovered.
What are some considerations as you.
Okay.
Fixed route to go.
With strategies to us is that as each of our Quebec or.
Something else and I have a follow up.
Thanks, John I'll start and I'm sure Todd.
Some comments.
So we'll look at gene therapy remains our core technology and we're so excited by our assets.
That's going to be the first thing we think about rate.
Yes.
And.
But look genetic medicine.
I think it's best done with multiple modalities.
Yeah.
We are.
As we speak during this experiment.
Are you going to test, whether we can shuttle.
Other modalities into the brain using a ligand against receptor.
And if those experiments.
Paul.
And we're going to have more options available to us I view it very much as a toolbox.
We can choose the right modality for the right disease.
On the right motive action that we're seeing.
And I think we're headed to the point, where you will have multiple options.
Todd.
I think that.
Keying in on is an easier delivery whatever the modality sneaker delivery in a safe and tolerable dose to achieve efficacy.
One of the things that we're showing with our Nextgen taps.
Perhaps that we can deliver broadly perhaps CNS.
Okay.
I think there could be advantages.
<unk> therapy is an advantage.
Yes.
With delivery and targeting from our targets but.
The other modalities that we're starting to explore.
Different batches.
Great.
And your cash runway guidance into mid 2025, what's included in that assumption.
Are you able to provide any guidance on milestones we can expect over the next 12 months from from any one Im sorry 11 partnered programs. Thank you.
So Jim Thanks for the question I think.
First and foremost I would say that.
We continue to be very diligent our cash resources that are on our balance sheet.
What we did for you guys today as we just kind of updated the guidance to be up.
A bit more precise as to runway guidance for the business.
As you know our guidance is based on only the cash that we have physically sitting on the balance sheet.
It does not include any additional potential milestones that we would receive from the partnership partnered programs that I alluded to earlier.
That potentially could extend that guidance and runway as well as some further.
Yes.
Two earlier as well.
We continue to be very thoughtful and or potentially reduce further collaborations and deals from a licensing perspective. So that's kind of guidance with regards to the runway it does take us.
We said mid 2025.
In terms of inflection points and milestones.
Obviously, some big milestones inflection points that we talked about we've mentioned as part of this earnings call.
Plan on getting into the clinic in the first half of next year.
Specifically with regards to the Tau program.
So that would be filing the R&D and getting in the clinic and then obviously the type one program we talked about.
2025 with regards to file.
And advancing that program.
Is not include the milestones associated with several of our partnered programs which is.
We've referenced in the past, we do have some partnerships sangamo.
In regards to Europe.
They've moved to also advancing two programs for Neurocrine and saying, Yes, My one program.
In 2005.
So those are additional milestones that we're looking for down the road hopefully that helps with regards to guidance and also provide you with some context around milestones during this time period.
Yeah, no very helpful. Thank you.
Thank you.
Our next question coming from the line of Jack Allen with Baird. Your line is now open.
Alright, thanks, so much for taking my questions and congratulations to the team on the progress.
Maybe the first one on the timing of some of these updates you mentioned that the studies are ongoing as it relates to the shuttle program I was wondering when those preclinical studies might read out and when we could get a disclosure surrounding that and then similarly for the sod one candidate selection expected by the end of the year, how should we think about disclosures going forward as you talked about Canada.
And then I have a quick follow up as well.
So.
Todd to answer the first question and then I'll attempt to answer the second one.
Sure.
The shuttle program.
It's an early early stages of discovery right now.
At this early stage, we haven't really given any guidance as to timelines.
We're looking forward to being able to update you in the future on that.
A specific date yet.
As far as the <unk> one program.
We expect to announce when and if we have announced.
<unk> identified a development candidate, we'll let you know and also we will let you know when we start our GOP toxicology studies.
And generally speaking, we'll let you know.
Whether or not we're on track.
For the IND in mid 2025.
Great Great and then.
Shifting gears sort of Tau program I know, we just had <unk> recently.
Was wondering if you could speak to some of the evidence from the meeting that provide you additional support is what can move forward. This.
How antibody and then I know one of the questions around the meeting with taking antibodies and given them via IV or.
Or more rapid injections as well.
What are your thoughts as it relates to the initial formulation for the tap program.
Bringing this into the clinic.
Yes, so thanks, well I think the meeting continues to I think validate that Tau is a very exciting target.
For Alzheimers disease.
Actually the next target after the ammo empty amyloid treatments.
We'll produce hopefully.
New medicines for patients.
Thanks.
By that I'm, referring to the fact that we've already seen that.
Certainly the.
Knockdown approach will affect the spread of Tau by pet imaging.
And now we saw it as most recently meeting it seems to lead to a slowing of clinical decline.
Small numbers still early days, but I would say I would look at the data is encouraging.
I think it's also interesting that anti amyloid antibodies.
Really work best and people with a low taubert right and Thats predictable I believe because.
Many of us believe that.
Beta amyloid plaques for the formation of plaques seems to somehow triggered the spread of Tau.
So I think and in fact Tao appearing in certain parts of the brain.
In the in the medial temporal lobe is actually part of normal aging.
But it's only when you have the presence of amyloid plaques in the brain that you're starting to see it spread into other areas of the brand which is actually pathologic. So so so we continue to be excited.
<unk> Tau as a target and Thats why we have two programs.
<unk> being an antibody that we have seen.
Blocks the spread of Tau in animals.
Quite well actually quite robustly.
And then we have the.
<unk> Tao.
Not down approach Bacterize, sorry, RNA, which in some ways could mirror.
<unk>.
<unk>.
I've seen we hope it will mirror the efficacy seen.
With the <unk>.
Alright, good nucleotide approaches that also knocked down the expression of Tau.
Alright, thanks, so much that color and congratulations again on the progress.
Okay.
Thank you and our next question coming from the line of Jay Olson with Oppenheimer. Your line is open.
Oh, Hey, congrats on the progress and thank you for taking the question.
Just to follow up on.
<unk> data at <unk>.
Can you just talk about how your anti Tau antibody will be clinically differentiated from other antibodies and also other modalities like anti Tau Aso's.
Yes.
Our <unk> SA RNA.
We'll knock down the expression of all forms of Tau.
And.
You know.
And so in some ways mirrors.
Adv antisense against.
The mechanism is different but the.
The end result, we think will be pretty similar.
Of course, it will be differentiated from the avian that onetime treatment.
<unk> are one of our proprietary tracer derived capsid.
So we will deliver at IP, and we will be able to get.
Transduction across multiple brain regions pretty much across the CNS, which I think is.
It's a big advantage.
That's a very different approach from the anti Tau antibody, which.
We will not.
Address <unk>.
Intracellular Tau for example, we don't believe we think the antibody will bind to extra cellular forms of Tau and what we hope to do with that antibody is to block the spread of Tau.
So two very different approaches.
And we'll see which one works the best.
It could be that both works.
It would be great.
Excellent. Thank you so much and I had a follow up question if I could.
Assuming your first <unk> will be for your anti Tau antibody whats the cadence of subsequent <unk>.
Which are the next most likely candidates for filing.
And then maybe a financial question.
In the clinic next year can you just talk about how you plan to finance those clinical trials.
So let me start with your first question.
So after.
The <unk> that we anticipate for the <unk>.
<unk> antibody.
In 2024, and <unk> and a wholly owned program will be the SLP, one gene therapy program for AOS, which we anticipate we will file an IND in 2025.
In addition, our partners at Neurocrine have indicated that they plan to select and move to gene.
Gene therapy programs.
Into IMD.
By 2025.
Assume that means the Parkinson's and feed rates ataxia programs. The most advanced but you'll have to ask them, which two programs Amy.
Then.
Sangamo has indicated that they're planning to move our <unk> program.
Into the into <unk> in 2025, as well and then those are the ones. We know some of our other partners that are pure capsid licenses for all I know, they're planning to file an IND in 2025 as well.
We don't we don't know that for certain because we don't know their timelines.
But yeah so.
I hope to see a steady stream of Amd's for.
For the foreseeable future at Voyager with or without our partners.
Great Super helpful. And then maybe Peter if you could please comment on the <unk>.
Financial plans for funding the clinical trials next year.
Yes James.
With regards to the wholly owned programs.
<unk> just mentioned so those are our Tau antibody program and then also the ALS program.
We're building those programs on a wholly owned basis, where we're funding and financing those as we're advancing those both to IMT is wells towards declining so it is our assumption.
Model that as part of our cash runway guidance that we've provided to you and update it today.
So that is that's clearly our idea is to funded finance those and advance those law.
Of course, as we get to various milestones as we move forward in the future, we'll always be thoughtful about.
The next step and maybe partnerships around those programs, if we decide to do that but as of right. Now those are modeled into our cash guidance and runway with regards to the partner programs that outlook to where we have milestones that could come about.
Over the next couple of years, specifically in 2025 with regards to Airtran and Sangamo.
Any of those programs as those are financed off our balance sheet.
And so thats part of our overall financing and funding.
That way I think the one thing I would add to that is specifically on <unk>.
<unk> is becoming.
Pass through R&D expense perspective.
A bigger piece of our overall research and development expense on a quarterly as well as year to date basis.
To that note, we've actually added some non-GAAP guidance information at the back of the earnings release.
Hopefully helps you guys Youre stand kind of how much really doesn't sit on our balance sheet.
You look at our research and development expense growing on a quarterly basis or on a full year basis. So I think that provides a lot of kind of look through with regards to the.
The pass through that's associated with that partnership and again that does not sit on our balance sheet. So hopefully that helps check.
Great. That's super helpful. Thank you very much.
Yes.
Thank you and our next question coming from the line of Laura Chico with Wedbush. Your line is open.
Hey, Thanks, very much guys for taking the question I guess I just wanted to circle back with respect to see Tad then.
There was also some data showing some novel tell blood biomarkers at the meeting.
Just had a curiosity as youre thinking about preliminary clinical studies.
Curious if something along those lines would be use or how are you thinking about kind of categorizing patients in terms of their tow load that entry.
Thanks, Laura.
A lot going on in the field as Tao.
And in terms of the selection of patients at the present time, we're thinking that we will likely mirror what.
<unk> has done in terms.
Starting with early stage Alzheimer's disease people with.
Cognitive impairment plus early stages of dementia.
And.
We will likely also insist on uncertain, how burden because what we want to do certainly with antibody program is to block the spread of Tau. So it would be sort of like a region of interest.
Primary endpoint on the spread of Tau, so we'd have to take people.
Who are at one of the early Brock stages, probably stage two.
Which can be visualized by Tau pet imaging.
To see if we can block the spread of Tau. That's how we're thinking of in terms of new tower Biomarkers. There is a lot of fluid based biomarkers that are coming out a lot of P. Tau various types of P. Tau Biomarkers, which I think are also exciting.
And.
I think we will probably also evaluate those in our tau programs as well.
But I think for sprint.
Nothing like pet imaging to evaluate the spread of Tau, which is what we want to see if we can block with the anti Tau antibody.
Okay. That's super helpful. And then just beyond that I guess is Cal spreading just the primary criteria youre going to be evaluating for advancement or kind of a threshold and is there a specific degree your magnitude of effect that youre kind of hoping to see thanks very much.
Well, we saw a 70, 70% approximately 70% reduction in spreading.
From one kept the campus to the other side to the other at the campus.
Our animal models.
So we.
We have that as sort of an animal experiment, but in terms of the human experiment.
I think right now I'd be happy to see.
A statistically significant decrease and spreading.
Spreading.
First stage and a relatively small study and then hopefully by then we will know the clinical significance of that somebody will likely have drawn the.
We figured out the relationship between.
Spreading.
Our tau reduction and a certain brain region and the clinical consequence of that and so.
In this way actually not being first can be helpful.
It will be we'll learn from the people who have entered the clinic first.
Very helpful. Thank you.
Thank you.
And our next question coming from the line of.
With Wells Fargo. Your line is now open.
Alright, thanks for taking our questions.
A couple.
Question is on how and I actually wanted to ask about.
How about the few tad presentation for <unk>.
I was wondering.
If you can share your assessment.
About the.
The magnitude of the signal loss benefit.
How many function and also.
The time point at which these signals.
Was achieved are episodic.
And whether these sigma.
Taking all of the benefit in your mind.
Consistent with more of a reduction of the existing.
In our existing neurons that have.
These newer February tangles.
Or could it be also consistent with the threading during this timeframe.
I understand a little better the contribution that those two.
Two separate compartment too.
Potential.
Clinical benefit was after.
Alright, thank you.
Thanks Shannon.
So in terms of the magnitude I think it's B b.
Premature to really.
Understand I don't think we can fully understand the magnitude when you have 16 to 20 patients per group right. So very small sample size. So I think you'd be hard also.
Hey.
It's a.
<unk> that.
Chosen.
<unk>.
It's the first foray into the into the choice of patients. So I think.
So I would say that and then there is the matter of multiple different endpoints, but the nice thing is that it was seen across several endpoints. So there seems to be a high degree of consistency regardless of the endpoint.
Rather than focus on the magnitude I have been focusing more on the consistency of effect across multiple endpoints with respect to the time point.
It's not uncommon that you would see an effect on Tao.
Pet imaging and then to have a sort of a delayed effect on clinical outcomes.
This is something that we've seen.
In months and neuro degenerative disease that you get.
In effect on target engagement, you see an effect on target engagement and EBIT pharmacodynamic effects biological effects and that the clinical effects take longer to see and so the fact that they had to wait.
I believe as much as two years to see some of the effect does not surprised alright, thats, what you see with amyloid drugs to that you could see an effect on amyloid pet imaging within six months.
But.
But it takes longer to see the clinical effects and I would say you see something similar.
Pass as well.
In terms of your question.
Thank you hit on something that's pretty darn interesting, which is that.
Actual reduction in Tau pet signal.
Or at least.
In the regions.
And that's been surprising to me because I always use the bank of America February Tangles is pretty.
Highly aggregated and.
The fact that you can actually reduce the Tau pet signal.
Cancer specific for aggregated.
First of all the forms of power so.
So maybe that maybe there's more maybe bees narrow fibrillary tangles are not quite as.
Sort of end stage, if you will and that they can be this reversible aggregation are at or it can be removed.
It's interest is an intracellular.
It is an intracellular tango so I don't think its phagocytosis it must be that.
These aggregates are more dynamic and perhaps reversible than we once thought.
And then in terms of.
What causes the efficacy that you see whether the reduction in Tau and <unk> or is it a block spreading.
While both are being seen with <unk>. So it's hard for me to discern, which it is whether it's the pockets.
Interference with spreading our R&D actual reduction in salt.
Hard to discern, which based on the data at least as far as I'm concerned Todd I don't know whether you have any other thoughts on that I think you've covered it.
And overall, it's early data I think it's truly exciting.
Still have a lot to learn.
Got it very helpful. And then I was wondering obviously.
Sorry, the one program that will be the first gene.
Therapy.
Program.
Internal program to enter.
Clinic.
In 2025, but I was also wondering for the panel silencing gene therapy program.
How much.
The component with it needs to be different obviously, the the krieger sequels.
This is different and that was it.
I'm wondering is there also.
Differences differences in the.
Quite frankly it dependent.
A different cell type to the targeted so I'm just wondering how much.
Can the Tau gene silencing program that leverage.
The.
Firstly Flushing clinical gene therapy following program. Thank you.
So I'm not sure I fully understood your question Jonathan.
You understand it because if you did.
Yes go ahead, yes, I think the fundamental question is can we take earnings side, one to move forward quickly with <unk>.
<unk> opt out program.
I think the answer is fundamentally yes.
There are different components so.
There is the capsid and then theres the payload.
We're learning a lot about our novel capsid to our current programs and we'll continue to learn about them.
As we move along we may or May not select the same capsid protein knockdown say as we do for the sub one.
But he might select the St catch it.
Depends upon the particular characteristics.
Like Sars one.
Spinal cord motor neurons, probably astrocytes spinal cord and we wanted to get to motor neurons in the brain stem and perhaps the cortex as well.
For how we want to get.
Broader delivery cross cortex.
Final court is much less important for talent. So we're looking for different things from our catalyst in that context.
Just as an example in terms of the payload the Arcos <unk> SA RNA payloads.
Have a lot of experience with SA RNA is Victor I guess are in Asia at Voyager certainly deleveraging that protest that down as we are for the <unk> program. Obviously, the sequences will be different because we're targeting different teams, but in terms of off target et cetera.
Synergies in our knowledge base there.
We can we have the opportunity as different kinds of promoters.
In terms of the different cell types, you want to target. So the answer is I think we will learn.
We are learning a whole lot about our apps and our payloads will certainly employ all of that a lot of it will be transferable, but some will be disease and target specific.
And I would only add thanks, Todd and I understand the question I think.
Yes, I would add that for.
Tao.
I don't really care too much about knocking down and astrocytes.
They are on that we want to knock down the expression of Tau.
Cause right, they're both fact drives us irna's, we'll learn a lot from the first program that would be up.
The second I would also say that the caps that we could use the same capsid, we could use a different capsid.
Against different receptors or we can use different capsid to get the same receptor.
Which we already.
No that receptor <unk> been normal so.
Yes, I think there is a high potential for lots of learnings from our first program that may help us with the SEC.
And third and so forth programs.
Great Thats Super.
Helpful and lastly, I was wondering if I can ask a quick question.
On the X.
Offered.
Mainly interested in.
<unk> do you think this will be an asset that is ready to pursue a partnership interest.
Hello milestone in terms of preclinical.
Evidence.
Sure.
The interest come in at any time, and Youre ready to discuss it.
Thank you.
Yes.
I'm always ready to discuss.
Potential partnerships.
Sure.
Good partners always ready towards always open.
For me, but I want to see is in vivo evidence that we can shuttle.
Crow molecule, such as a nucleic acid or a protein.
Cross the BBB and get Dizzy.
<unk> affect pharmacodynamic effects.
In the brain.
If we see that.
Now I'm pretty I'm, even more excited.
And.
And then we can start to think about okay. What do we want a shuttle across.
And which targets that we want to pursue but it's that proof of concept if you will.
You can use leverage resection receptor X.
The shuttle things across the Pvp and produce a biological effect in the brain.
That's that will trigger a whole bunch of things, including potential partnership discussions as you mentioned.
Great. Thanks for the color.
Thank you.
And as a reminder, laser and gentlemen to ask a question. Please press star one one and our next question coming from the line of <unk> Kulkarni with Canaccord. Your line is now open.
Good afternoon. Thanks for taking my question is both that on the <unk> antibody.
You alluded to this a little bit, but it was more related to the potential outcomes of a trial, but at this stage, we've conceptualizing any <unk> Tau burden based cutoffs to recruit patients into your clinical trials.
Yes.
I was thinking that we would probably want people.
Who have deep what would be the equivalent of <unk> stage two.
Maybe also pronged stage, three but right now honing in on <unk> stage two.
Which can be defined by Tau pet imaging.
Enroll those patients and then see whether or not we can block the progression to stage three and four if you will.
That's how we're thinking about it now so.
We're still doing a lot of the <unk>.
Thinking around data as wireless consulting with experts and investigators, but that's conceptually how we're thinking about it now.
Does that is that what you were asking does that answer your question Scott.
Yes, somewhat but a little bit of a follow up to that do you think blood based biomarker well enough to be helpful to you in your trial inclusion program.
I think blood based biomarkers.
Can get clarity as to which blood based biomarker.
Links up to stage two.
That would be helpful.
B.
You can imagine us doing is starting with a.
Starting to screening for patients with a blood test using a blood based biomarker and then taking those patients that are qualified based on that and then doing tau pet imaging to be sure that they have the right stage.
I could see it as a sequential sort of screening process. If you will starting with a more feasible and easier and cheaper.
Blood tests and then.
Moving to the more complex and more.
Our expenses.
Pet imaging.
Eight patients.
Got it and then given what you know so far about your antibody are there any special considerations, we should be aware of on your GOP Tox studies or are these relatively conventional ones.
Well.
Let me start by saying that we did have an interaction with FDA earlier this year.
Second I would say that there is one.
The word conventional tough because.
One of the things that we like about our antibody is that it is very specific.
Pathological forms of Tau.
In other words, it does not bind.
Normal Tau that we all have in our bodies and that every animal perhaps.
So if you want to look at on target toxicity.
Kind of hard to do that in a wild type animals, which is what's typically used for Tox studies. So.
You can imagine that's a little bit more complicated than I would say the conventional toxicology studies.
We've taken all that into account.
And we've had our interaction with FDA and we think we're on track for an IMD.
As we indicated in the first half of next year.
Got it thank you.
Sure.
Thank you.
And our next question coming from the line of <unk> with TD Cowen. Your line is now open.
Hi, This is the answer Phil Thanks for taking our question most of mine have been answered but.
You guys have talked a lot about potential partnerships and how our discussions are constantly ongoing but going forward is the focus going to be more on maybe generating a proof of concept in an early stage trial before initiating discussions with partners to pursue those larger later stage trials or.
Do you still think of it as like out licensing programs that are identified through that fair trade to our platform and then I have a follow up.
Yes so.
I would say that right now as you've seen we've done a variety of partnerships, including sort of relatively simple capsid licensing arrangements as well as partnerships around named programs such as the GBA One program for example.
And we're open to either.
Type of partnership.
And.
And your question was I think our we are going to increasingly look to get to proof of concept before we partner.
Well as we get more and more the financial wherewithal to do that we may want to get to proof of concept, but I never rule out anything. We're open we're open to talking to any partner about any type of deal that makes sense for us.
For our shareholders and for the patients most importantly.
We're conscious of the fact that of course.
The further you go into development the more value is created.
But on the other hand, there is more risk.
That should absorb as we get to that proof of concept. So it's always a.
We're always thinking about all these things value risk et cetera.
Okay. That's helpful and then on the Tau program with desktop studies done are there I guess what else needs to be done before the IND is ready to be there is it mostly just compiling everything and putting it all together.
Is this on our <unk> knockdown.
The white wine program.
We've already selected a development candidate.
So we're in the process. So we just initiated the GOP toxicology studies.
Have started the manufacturing obviously.
To manufacture enough material to do the GOP Tox studies, and if we want to start clinical trials. Shortly after we file in.
In the first half of thanks, Shar, we have to have enough product to be able to do the first.
Clinical studies and the work we're on track for all that.
Does that answer your question.
Yes, yes. It does thank you so much.
Okay.
Okay.
Thank you.
And I see no further questions in the queue. At this time I will now turn the call back over to Dr. Al Sandrock for any closing remarks.
And thank you to everyone for joining us today and please feel free to follow up with US directly if you have any further questions. Thanks again.
Ladies and gentlemen. This concludes today's presentation. Thank you once again for your participation you may now disconnect.
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