Voyager Therapeutics Q3 2024 Earnings Call Transcript

Quartr
Provided by Quartr
November 12, 2024

There are 12 speakers on the call.

Operator

Good afternoon, and welcome to the Voyager Therapeutics Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen only mode. There will be a question and answer session at the end of this call. Please note that today's call is being recorded. A replay of today's call will be available on the Investors section of the company website approximately 2 hours after completion of this call.

Operator

I would now like to turn the call over to Trista Morrison, Chief Corporate Affairs Officer at Voyager.

Speaker 1

Thank you, and good afternoon. We issued our Q3 2024 financial results press release this afternoon. The press release and 10 Q are available on our website. Joining me on today's call are Doctor. Al Sandrock, our Chief Executive Officer Doctor.

Speaker 1

Toby Ferguson, our Chief Medical Officer and Doctor. Todd Carter, our Chief Scientific Officer. We will also be joined for the Q and A portion of the call by Nathan Jorgensen, our Chief Financial Officer. Before we get started, I would like to remind everyone that during this call Voyager representatives may make forward looking statements as noted in Slide 2 of today's deck. These statements are based on our current expectations and beliefs.

Speaker 1

They are subject to risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings, which are available on our website for additional detail. Now, I will turn the call over to Al.

Speaker 2

Good afternoon, everyone, and thank you for joining us. Please turn to Slide 3. At Voyager, we are leveraging the power of human genetics to discover and deliver transformative medicines that address the root cause of neurological diseases. Our 4 pillars of value include: 1st, our pipeline of 4 wholly owned and 14 partnered programs. During the Q3, we completed enrollment and dosing in the single ascending dose trial of VY7523, our anti tau antibody for Alzheimer's disease.

Speaker 2

We also recently saw encouraging third party clinical data with another anti tau antibody for Alzheimer's disease, which Toby will talk about shortly. Our second pillar of value is our industry leading tracer platform for the discovery of novel AAV capsids to enable CNS gene therapy. These IV delivered CNS targeted capsids underlie all of our wholly owned and partner gene therapy programs and we expect INDs for 3 of these programs next year. 3rd, we have blue chip partnerships with some of the world's experts in neurology and gene therapy, including Neurocrine, Novartis and Alexion. We continue to progress these partnerships through the Q3 with Novartis signing on for a 5th partner program partnership and Neurocrine nominating a 3rd development candidate.

Speaker 2

Finally, we continue to explore the potential to leverage receptors we have identified to shuttle non viral genetic medicines into the brain. Ultimately, we aim to expand from gene therapy and antibodies into other modalities of neurogenetic medicine broadening our impact. With that, I'll turn the call over to Toby to talk more about our anti tau antibody and some of the recent third party data in the space.

Speaker 3

Thanks, Al, and good afternoon. Please turn to Slide 4. This slide summarizes the 4 wholly owned programs and 14 partner programs that Al mentioned earlier. Today, we are going to focus on our tau targeting programs for Alzheimer's disease. Turning to Slide 5, the point I want to make here is that we view tau as a critically important target for the treatment of Alzheimer's disease.

Speaker 3

As you can see in the image on the left of this slide, the spread of pathological tau as identified on tau PET imaging correlates closely to Alzheimer's disease progression as measured by BROCK clinical pathologic staging. Additionally, exploratory clinical data from Biogen's Vibeti tau knockdown program showed a reduction in pathological tau as well as potentially greater slowing of clinical disease progression than seen with anti amyloid antibodies. So in short, we think targeting tau has the potential to impact Alzheimer's disease progression and do so in a significant way. This is why Voyage is pursuing 2 approaches to targeting tau, our anti tau antibody VY7523 and our tau silencing gene therapy. On Slide 6, I want to highlight how the team selected our anti tau antibody.

Speaker 3

Started with more than 700 antibodies across the mid domain and C terminus of tau. We first focused on antibodies targeting pathological tau. This was important with the anti amyloid antibodies. Several that failed such as solanezumab or bapanezumab were shown to have insufficient target engagement with aggregated forms of amyloid beta, while the approved anti amyloid antibodies all target the aggregated species. We narrowed the funnel through further in vitro and in vivo studies.

Speaker 3

We evaluated antibodies targeting various epitopes across tau, including in the mid domain and the C terminus. In a mouse C model of human pathological tau spread, the C terminal targeted antibody AB01, the mirroring version of BY-seven thousand five hundred and twenty three decreased the spread of injected pathological human tau by approximately 70%. Based on these data and the specificity for pathological tau, we selected BY-seven thousand five hundred and twenty three as our clinical candidate. We also conducted a series of head to head studies in the same model against other anti tau antibodies. These data are summarized on Slide 7.

Speaker 3

In the first head to head study, we evaluated murine-seven thousand five hundred and twenty three against murine versions of Biogen's Gosinurumab and Lilly's zafaginamab. These antibodies both targeted the N terminus of tau and both failed the primary endpoints in clinical trials. Both were ineffective at reducing tau spread in the model, while our C terminal targeted antibody again blocked tau spread. This gave us confidence that the model had negative predictive value. In the second head to head study, we compared our C terminal targeted antibody to UCB's burnapap, which targets the mid domain.

Speaker 3

In our models, both antibodies inhibited tau spread. Based on these data, we were eager to see the results from the vipanapap clinical trial, because we thought that if this antibody could impact tau in a clinical trial, then our model might also have positive predictive value. Turning to Slide 8, I want to summarize the recent propranumab data shared at the 2024 CTAD meeting. I want to start by noting that the primary endpoint CDR stomach boxes was not met in the full study population. This is important to acknowledge.

Speaker 3

Now I would like to note that propranumab inhibited the accumulation of tau in human brain by 33% to 58%. We saw this as establishing that an antibody can be used to inhibit the spread of pathological tau in the brain. This finding should not be underestimated. As I think that after the failure of the N terminal anti tau antibodies, there was uncertainty in industry as to whether an antibody approach could impact tau accumulation. As it turns out, it can.

Speaker 3

I also want to note that propranumab slowed cognitive decline by 21% to 25% versus placebo per ADAS COG14. Additionally, in subgroup analyses, it seems that patients with the greatest reduction in tau burden had more consistent clinical benefit, although we will need to see more detailed PKPD and PD clinical correlation analyses. Finally, propanumab demonstrated an acceptable safety profile with brain hemorrhagic and inflammatory changes similar to placebo. What does this mean for Voyageur's VY-seven thousand five hundred and twenty three? I would say that these data give us increasing confidence that antibody targeting the appropriate epitope of tau can slow the accumulation of tau in the brain of Alzheimer's patients.

Speaker 3

And that this slowing may offer a clinically significant benefit in some patients. This was a top line data presentation and there are certainly more work to be done here and we look forward to seeing the additional data. Slide 9 provides an overview of our Phase 1 clinical development plan for VY-seven thousand five hundred and twenty three. During the Q3, we completed enrollment and dosing of healthy volunteers in our single ascending dose trial. We expect to report top line safety and pharmacokinetic data in the first half of next year.

Speaker 3

We expect to initiate a multiple ascending dose trial in patients with early Alzheimer's disease next year and generate initial Taufat imaging data in the second half of twenty twenty six. At this point, we don't see anything in the brepranumab data that would shift our thinking here. If anything, the data reaffirmed that Tau PET imaging is the critical outcome we want to focus on and we have planned a very efficient trial focused on that outcome. We found the safety profile of propranumab encouraging and look forward to exploring the full range of dosing possibilities. I mentioned earlier that we are advancing 2 approaches to targeting tau for Alzheimer's disease.

Speaker 3

We focused a lot on the antibody approach today in light of the recent third party data. But I will now turn the call over to Todd to touch on our other approach.

Speaker 4

Thanks, Toby. Please turn to Slide 10. In addition to our antibody based approach, we are also advancing the tau silencing gene therapy program. This program deploys a tau targeted siRNA packaged in an ID administered tracer capsid. Using this approach, we have demonstrated robust reductions in human tau, mRNA and protein across the brain following a single IV administration in mice that express human tau as shown on this slide.

Speaker 4

It is also important to note that the recent BIVV80 data that Toby mentioned showed that reducing tau expression was associated with favorable trends on clinical outcomes and pathological tau as measured by PET imaging. We view these data as supportive for a knockdown approach and believe that the one time IVD therapy could provide benefits over repeated intrathecal therapy. This program remains on track for U. S. IND and Health Canada CTA filings in 2026.

Speaker 4

I will now turn the call back over to

Speaker 2

Al. Thanks, Todd. As you can see on Slide 11, Voyager continues to deliver on expectations for 2024. We have advanced our pipeline, our platform and our partnerships. Our strong cash position of $345,000,000 at the end of the Q3 is expected to provide runway into 2027, enabling multiple data readouts.

Speaker 2

This cash figure doesn't include the recent payments from Novartis or Neurocrine, which were received in October, nor does it contemplate any future potential milestones from our 14 partnered programs. With a robust slate of clinical milestones expected into the next 12 months to 24 months, a maturing partnership portfolio with top tier collaborators and cash runway into 2027, we believe Voyager is poised to drive significant value creation over both the near and long term. Finally, I'd like to thank all of our employees for their hard work and dedication to improving the lives of patients. With that, we will open the call for questions. Operator?

Operator

Thank you. At this time, we will conduct a question and answer session. Our first question comes from Phil Nando at TD

Speaker 5

Cohen. Good afternoon. Thanks for taking our questions and congrats on the progress. I guess first a follow-up to your comments on the Taupek Imaging. We're curious whether you have more insight as to how to translate the Taupe imaging results like we saw from Roche UCB into cognitive improvements.

Speaker 5

Any additional thoughts now that you've had a bit more time to let the MISTI-ten data set in? Do you think it's a question of selecting the right patients, selecting the right endpoint? And any thoughts on how to get that TAPAD imaging data to translate into a clinical improvement in cognitive benefits?

Speaker 3

So maybe I'll start that question. This is Toby. So I think fundamentally, I think the I'll reemphasize the point that the where the top of the imaging, we saw clear data in the whole population that you did not you slowed the accumulation of tau. And then I think the other point I'd make is that when you looked in some of the other populations, particularly the lower tau populations, you saw perhaps a slightly greater less than an accumulation in that context. So I do think as we move forward, really understanding the population will be critical in the terms of perhaps towel burden, which seems to be the main point.

Speaker 3

And then I think the other point I think is we'll look forward to understanding the full dose response in terms of sort of the tau reduction in the context of PKPD. We look forward to that data from UCB moving forward. Those are my initial thoughts.

Speaker 2

And I would just add that this relationship between Alpek effects and disease effects on clinical outcomes are agnostic to the drug or to the approach, right? So there are more than one there's more than one drug targeting tau over the next couple of years. I think we'll learn this relationship better, what degree of tau PET imaging translates to what clinical outcome. And I agree with Toby that selecting the right patients, particularly with respect to tau burden, for example, may be very important.

Speaker 5

Perfect. That's helpful. And then a follow-up on the SOD1 silencing gene therapy for ALS. You note in the press release and in your prepared remarks that the initial trial could generate proof of concept based on biomarkers. Is that something that we could possibly see in 2026?

Speaker 5

Is that something that's within 12 months of the U. S. IND?

Speaker 3

So we haven't guided to timing of the biomarker results. The points I can make is that 1, we'll go into the SOD1 population of course and 2, we'll start in at doses with the potential efficacious given the severity of the disease. In that context, we do think results would be reasonably forthcoming.

Speaker 2

And maybe I could add that certainly in the case of the Tufersen clinical development plan and the trials that have been published in the New England Journal, you can start to see effects on SOD1 levels in spinal fluid and about is it 12 weeks?

Speaker 3

About 12 weeks. 12 weeks, Toba, yes.

Speaker 2

And then effects on plasma neurofilament, which is a surrogate outcome that's reasonably likely to predict clinical outcomes and was the basis of approval by FDA of Tufersen. That measurement should also yield data in the 1st say 6 months or so. So if that's any precedent, that gives you an idea of the timing that it takes to see both biological effect and the outcomes on clinically relevant surrogate outcomes like NfL.

Speaker 5

That's very helpful. And last question, an accounting question from us. The $15,000,000 from Novartis to $3,000,000 from Neurocrine, how will those be booked? Are those going to be booked in the Q4 of this year? Or will they be spread out over the life of those agreements?

Speaker 2

It will be booked in the Q4.

Speaker 5

Perfect. Thanks for taking our questions. Congrats again on the partners.

Speaker 2

Thanks, Phil.

Operator

Our next question comes from Jack Allen at Baird.

Speaker 6

Great. Thanks so much for taking the questions and congrats on the progress. I wanted to ask about an aspect of the Voyager story that's more in its infancy and that being the blood brain shuttle programs. I was hoping you could just provide some context as to where those programs sit and when you maybe could look to bring an asset into the clinic. I know there's been a lot of activity in the space with AbbVie's acquisition of Alieda and then also Roche providing some initial proof of concept data from their beta amyloid asset there as well.

Speaker 2

Well, Jack, I'll start and I'll ask Todd to provide more color. But I would say that we're very excited about the potential of leveraging the receptors we identify through our capsids to shuttle other sort of macromolecules across the BBB. And as you pointed out trontinumab, the data with trontinumab, which leverages transferrin receptor is looking very promising as an approach to get anti amyloid antibodies into the brain. And so we're very excited about that. We're still in the preclinical stages.

Speaker 2

We haven't been we're not close enough to the clinic yet to guide on when we would enter the clinic. Todd, do you want to provide more color on that?

Speaker 7

Thanks, Al. So Jack, as you do point out, we are excited by it. We know that these receptors and we do have different receptors, different capsid families, they can mediate transcytosis delivery across the blood brain barrier for a large molecule, the AAV capsid. So there's reason to believe that they should be able to deliver other things. Hopefully, in the future, we'll be able to share data on that.

Speaker 7

I think the other aspect is that we're seeing with receptors such as transferrin that there's the opportunity for additional receptors to have an impact because we expect that each receptor could have its own profile, both for pharmacokinetics and safety that could really give an opportunity for different receptors for different diseases and indications.

Speaker 6

Great. That's great context. Maybe just one brief follow-up. You've been incredibly successful in partnering your gene therapy library of capsids, even in the context of these preclinical assets. Any comments as it relates potential partnerships around the blood brain shuttle programs?

Speaker 6

And is that something you have an appetite for before moving this into the clinic?

Speaker 2

Well, Jack, this is Al. Yes, I mean, I'm always interested and willing to talk to potential partners. This is exactly the kind of thing where there could be expertise in other companies that could really be very complementary to our shuttle program. So always open to talking to potential partners about these kinds of things and really accelerate the program.

Speaker 3

Great. Thanks so much.

Operator

Our next question comes from Joon Lee at Truist Securities.

Speaker 8

Hey, congrats on the progress and thanks for taking our questions. Your plans for 7,523 match study in 2025 imply that you may be seeing something positive in SAD study or am I being too liberal with my interpretation? And so the SAD study due in first half of twenty twenty five, in addition to safety, what are you tracking that could give us some incremental conviction on the drug? Thank you.

Speaker 3

This is Toby. Thanks for the question. Just to remind, the SAD study is in healthy volunteers. And so really the data we're looking from the SAD study is PK, initial immunogenicity and do we have the appropriate exposures to pick doses for the MAD study and some initial read on safety. So really it's not in patients, that's the key point.

Operator

Our next question comes from Jay Olson at Oppenheimer.

Speaker 9

Hey, congrats on the progress and thanks for providing this update. We have a few questions. Based on the peprenumab data that you saw, what's your latest thinking on the potential read across to your own 7,523 program? And is there any additional data from UCB that you would be interested in seeing? And then I have a follow-up.

Speaker 2

Well, look, I think, Jay, this is Al. I mean, this is the first time we've seen that an antibody could have biological effects in the brain in humans, right? And so that's I look on that as positive, right, that an antibody approach could affect the biology of how spreading in the brain. We even before we knew about the Pranavab data, we did say that there could be read through because we had tested the UCB antibody which binds to a mid domain region versus our antibody which binds to the C terminal region in an animal where we look for this we look at the spread of pathological tau in a mouse expressing human tau where we inject pathological tau from Alzheimer's brains into the animal. And we said before the CTAD, before we knew anything about the propranumab data that the UCB antibody was pretty comparable to our antibody in blocking the spread and actually distinguishable from the N terminal directed antibodies that did not block the spread in that same model and which also failed in the clinic.

Speaker 2

So and so we still believe that there could be read through. And we also expect that the safety perhaps would also be similar in the sense that the antibody is binding to extracellular tau. I would say the other differentiating feature of our antibody relative to the UCB antibody is the fact that our antibody specific for pathological forms of tau. We don't know how important that is. We'll find out, but that is another distinguishing feature.

Speaker 2

So that's where we stand, Jay.

Speaker 3

Bob, do you

Speaker 2

want to add anything?

Speaker 7

I agree with everything you said, Al. The other thing to mention, there was a question about additional data from UCB. And I think it will be important for us to see what additional data come out on some of the PK and PD, that PKPD relationship. As Alice pointed out, the relationship between reduction of tau accumulation and potential cognitive benefit that it appears is pretty interesting and we see that as very encouraging. Those are in some groups.

Speaker 7

I think seeing more detail about target engagement and that relationship will really be important to interpret the results.

Speaker 9

Great. That's wonderful. Thank you for that. And then maybe just as a follow on, how are you thinking about prioritizing 7,523 versus your tau silencing gene therapy? And then the potential for combining either of your anti tau approaches with anti amyloid antibodies?

Speaker 2

Well, I would say that they're both high priorities. Am I allowed to say that 2 things are both high priorities? Yes. Look, we've always said that power is a pretty very important target that we think is the next step in helping Alzheimer's patients that the anti amyloid treatments got us started, but we need to do better for patients. And so I think they're both high priorities.

Speaker 2

They're very different approaches in many ways. And so we'll find out which is the better approach. In terms of combining or with the anti amyloid antibodies, I do think that there could be a place for combining. I would say that it would be in those patients that seem to have a partial response to the anti amyloid antibodies. They have no response to the anti amyloid antibodies.

Speaker 2

There's no sense in continuing the drug. If they've had a full response to anti amyloid antibodies, which may be the case in some patients. If you're not progressing or at all on in your disease on anti amyloid antibodies, there'd be no reason to add anything else. So I think time will help us. Time will tell what the heterogeneity of the treatment response is to the anti amyloid antibody therapies in humans as we see the drugs being rolled out.

Speaker 2

And then by the time we're ready to think about that combination approach, I think we'll have more data on how many patients respond fully, how many respond not at all and how many respond partially. And but I would say that we would want data initially as monotherapy too, first to see that make sure that our drug is active and then have some understanding of the doses that we want to use.

Speaker 5

Great. Thanks so much for taking all the questions.

Speaker 2

You're welcome, Jay.

Operator

Our next question comes from Lily Insongo at Leerink.

Speaker 10

Hi, good afternoon and thank you for taking our question. So I guess two questions for me. So the first one, as we await for inpatient data for in 2026 for the tau targeted antibody, are there any plans to potentially test previously developed C terminal antibodies in preclinical models to try to further confirm the negative predictive values of the model you use in a more region specific approach?

Speaker 2

Well, if we if there's another antibody that's negative, I think that would be a pretty interesting study to do, particularly if it's not end terminal, right? So I'm not sure doing any more N terminal directed antibodies would add much, but if there's a different antibody targeting a different epitope that fails, then it might be interesting to do that experiment that you're proposing really.

Speaker 10

Yes, because it was my understanding that both Hoffman and Roche and then Lundbeck had some C terminal antibody. I think it could be really interesting preclinical data as we await. And I guess maybe segue into my second question. So regarding the UCB data and the challenges that they've had in the overall population, would that compel you to potentially start the development in patient in maybe a smaller, more targeted patient population? So I guess my question is, does the results of the study compel you to maybe have more stringent inclusion criteria in the initial inpatient study for the antibody?

Speaker 2

Yes. Hi, Lilly. This is Al and I'll start and I'll ask Toby to add his comments. But I would say that we would be very open to that idea. We would like to see if we can differentiate from the pranumab if at all possible.

Speaker 2

And it could be that the choice of patient population could be a way to boost the efficacy that we see particularly on the clinical outcome measures right and get more clarity. As Toby noted in his comments, there were subgroups particularly those that were pre specified. I think it was the APOE4 non carrier low tau burden patients who seem to do better and those were exactly the patients where the effect on tau accumulation was more pronounced. So I'd be open to that. Even though it's a smaller population, we're talking about a gigantic population overall, the Alzheimer's disease patients.

Speaker 2

And to have a targeted treatment for a smaller population that's still quite large by the way, is would be of interest. Tobey?

Speaker 3

So the value captured nicely. I think the only other comment I'd make is that when we did look at the provenumab data, they had segregated their low and high tau populations. There really weren't that many low tau patients in the study overall about a 80:twenty ratio approximately. And so I think more data there that is suggestive data, but we want to see sort of the breakdown of those data.

Speaker 10

Thank you. And maybe just a follow-up. So we will be waiting for initial safety and PK data in healthy volunteers in the first half of twenty twenty five. But I was wondering maybe if you could give a little more granularity in terms of when we should expect an update on the potential design of the inpatient study?

Speaker 2

I think she's interested in the design of the multiple ascending dose study.

Speaker 4

Yes, exactly.

Speaker 3

Thanks, Lily. So I think as we once we get closer to the start, which we'll start next year, we'll share the details of the design. Thank

Operator

you. Our next question comes from Patrick Trucchio at H. C. Wainworth and Co.

Speaker 11

Good afternoon. Thanks very much. Just a couple of questions for me. The first is just a follow-up on earlier comment regarding the appropriate patient population for the tau program for the antibody program. Can you discuss what level of tau PET burden do you think could be appropriate at baseline based on this learnings from this recent data and particularly as we approach that Phase 1b trial with 7,523?

Speaker 11

And then separately, as we think about or as we look ahead to the tau silencing gene therapy, I'm wondering if you can talk about the advantages or differences we should expect from the tau silencing gene therapy as compared to these tau targeting antibodies? And then secondly, what remains to be completed to ensure that the 2026 IND and CTA filings remain on track for the tau silencing gene therapy? And then lastly, if you could just talk about the anticipated capsid and advantages of the Voyager platform as compared to some of these others in the field developing a similar approach?

Speaker 2

Well, that's a lot of questions, Patrick, but we'll knock them off 1 by 1 here. So I'll take the first one and then maybe Toby can take the second and Todd you can take the third one. But anyway on the appropriate patients, the cutoff that UCB shared data with us was a pre specified cutoff, but it was not the median. In fact, there were many fewer patients I think in the low tau burden subgroup versus the high tau. I think that was because it was pre specified.

Speaker 2

Hopefully, we'll see more data on the full range of tau burden and to understand better what the cutoff needs to be. And so right now, I mean, all we have is that one piece of data to go on. I think we would need more to be more specific about the tau burden. Toby, you can let me know if you disagree with that. I do not know.

Speaker 2

Okay. And then on the tau KD, think you were asking, how do we make sure that we stay like I'll be on track. And so maybe, Tobey and Todd, do you want

Speaker 3

to help me with those questions? Sure. Maybe I'll start and ask Todd to fill in. So the preclinical development is proceeding. I think the key next steps are sort of moving them all through forward.

Speaker 3

And then of course, I think the pivotal step we'll do in the toxicology studies.

Speaker 7

Right. Standard IND enabling studies would be part of that whole process. You also asked about differentiation for the tau knockdown versus the antibody approach, I believe. And I think the BIV80 data, which is an ASO delivered that has shown some really interesting clinical data to date versus the antibody approaches. But what we've seen in the BIV-eighty data are not only signs that you could reduce the accumulation of tau, but you may be able to reduce tau burden, pathological tau burden as measured by PET, in some cases below starting burden, which is a pretty remarkable finding.

Speaker 7

When you look at those sorts of data and even with the recent rapanimab data that also seem to suggest that an impact on tau PET imaging results can impact clinically. I think there is a accumulating evidence in the field that impacting tau accumulation can have a clinical benefit. With the gene therapy approach, of course, here we're talking about a once and done approach. We can dose once. We know that our vectors and this gets into a little bit of the Voyager platform question as well that our capsids can deliver quite broadly throughout the CNS.

Speaker 7

We can get delivery not only to the, I'll call it, the shallower regions of the brain, but also the deeper structures with that single dose. And so we think there could be some substantial benefits for tau knockdown approach. It is also an intracellular effect. The antibody has an impact presumably based on the prion spreading hypothesis as the pathological tau spreads from cell to cell. So an intracellular reduction of tau could have an impact not only in reducing that cell to cell spread, but also in an intracellular way.

Speaker 7

So those two approaches, I think, are both interesting. There are signs of both those mechanisms that they could work. And in the end, it's even possible there could be combination approaches.

Speaker 2

So, Tobey, do you want to add on potential clinical outcomes?

Speaker 3

So there I think the BIIB080 data in particular really emphasized along with the recent propranumab data, the importance of tau PET and to Todd's prior point in the BIIB080 knockdown based approach or a knockdown based approach, you can see the removal of both pre existing pathological tau and prevention of further accumulation and in somewhat earlier timeframe 6 months or so, which is distinct from slowing accumulation. And this is particular to the tau knockout approach. In addition, you can also see clear signals in the CSF biomarkers, a total tau for example and other tau species. And so there is a chance based on biomarkers to get a relatively quick read on how the molecule is performing.

Speaker 2

And what about the do you think the effect size? Mean it's hard, right?

Speaker 3

It's early data that the I mean if you look at the propensity score matching that Biogen performed, they looked at a comparison to another study called Tango, they looked at the natural history study And there that effect size was 2 to 2.5 points on CDR small boxes. As a reminder, the effects on the amyloid deficit of 0.4 to 0.9 points approximately on CDR small boxes. Potentially larger effect size as well, which gives you an additional opportunity to potentially say the total signal with smaller sample size.

Speaker 11

Great. Very helpful. Thanks so much.

Operator

I'm showing no further questions at this time. I would now like to turn it back to Al for closing remarks.

Speaker 2

Thank you everyone for joining us today and we look forward to speaking with you again soon.

Operator

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.

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Earnings Conference Call
Voyager Therapeutics Q3 2024
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