Q2 2023 Voyager Therapeutics Inc Earnings Call
Good morning, and welcome to the Voyager Therapeutics second quarter 2023 conference call. All participants are now in a listen only mode there'll be a question and answer session. At the end of this call. Please be advised that this call is being recorded.
At the company's request.
I would now like to turn the call over to Pete French you Chief Financial Officer. Please go ahead.
Thank you and good morning.
Joining me on the call today or a doctor al Sandrock, Our Chief Executive Officer, Dr. Todd Carter, a chief scientist golf Sir.
We issued our queue to 2023 financial results press release this morning.
The press release and the 10-Q are available on our website.
In a moment I will turn the call over it at all.
Before I do this I want to remind everyone.
During this call Voyager Representatives may make forward looking statements I just noticed slide to today's Jack.
These forward looking statements include future expectations.
And prospects.
I'll forward looking statements are inherently uncertain and 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.
Form 10-K .
Filled with the S. P C.
As of the following of today's quarterly report on Form 10-Q.
There have been no material changes to the risk factors described in our annual report.
Oh F C. C filings are available on the company's website now.
Now it is my pleasure to turn the call over to al. Thank you and good morning, everyone.
Please turn to slide three.
I'd like to start by recognizing the incredible innovation happening right now in narrow therapeutics.
Gene therapy.
I talked about this on our last call.
Since then we continue to see tremendous progress.
Therapeutics front, just last month like kind of Mab received full F. D. A approval for all drivers disease as well as Medicare coverage.
All those other recent F D a approval and the narrow therapeutic space, including approvals of medicines for free drags ataxia and amyotrophic lateral sclerosis.
Gene therapy field also continues to advance with recent F. D. A approved both of the first gene therapies for Duchenne muscular dystrophy and hemophilia a.
Voyager sits at the intersection of narrow therapeutics and gene therapy rebuild.
We believe we are uniquely positioned to leverage the advancements in both fields and importantly to combine them.
The date, the delivery of gene therapies into the central nervous system or C. N S as proven challenging.
Purchase to inject these therapies into the brain parenchyma or various cerebral spinal fluid spaces generally have not been successful and continued to resolve and setbacks for other companies.
Would you recognize this back in 2021, that's why we pivoted to intravenous delivery leveraging the vasculature to penetrate the C. N S. Something we have enabled through our innovative capsid design.
Moving to slide for.
Briefly review our investment rationale.
That form pipeline partnerships and potential.
First the platform.
Voyager uses our trade Sir caps of discovery platform to generate multiple families of novel captives, that's robust CNS tropism following Ivy delivery.
We have presented data demonstrating strong transaction to multiple areas within the brain and activity across multiple species.
Our most recent data at the American Society for gene himself therapy or S. G. C. T 2023 conference showed greater than 50% transduction in multiple brain areas at the relatively low dose of 212 P. G as per kilogram marmosets.
Second pipeline.
We are advancing for C. N S programs through late stage research and towards IMD filings, including are wholly owned anteater antibody for all Simers disease and S. O D. One gene therapy program for a L. S.
Currently you have three wholly owned assets targeting all simers disease, including the new early research in an initiative just announced today to advance a vectorized N T a beta antibody.
We will talk more about this in a minute.
Third partnerships Voyager.
Voyager has generated more than $200 million this year alone.
Dilutive partnering revenue.
And Q2, we executed a license agreement with Sangamo around prion disease, bringing our total number of partnered programs to 11.
These programs provide opportunities for additional milestone.
Or royalty revenue to Voyager as well as opportunities to generate data with our capsid.
And most importantly to help patients.
Finally potential.
Specifically the potential to expand from gene therapy and to other approaches of neuro genetic medicine.
As those of you who attended our standing room only target.
T know.
We are making good progress in our receptor program.
After identifying a receptor for one of our capsid families.
Now also identified a lie again for this receptor which has many of the characteristics required for transport of macromolecules across the blood brain barrier or BBB.
We are exploring the potential to leverage the receptor to shuttled nonviral genetic medicines across the BBB.
We have also preliminarily identified two receptors for additional families of our tracer cabinets and we are conducting confirmatory research to further validate these discoveries.
While this program is early.
Increasingly excited about the potential here to expand the reach of our technology into other approaches of neuro genetic medicine.
Moving to slide five as you can see Voyager is advancing quite a robust pipeline.
However, we are doing so efficiently before.
The four programs depicted in blue at the bottom of the slide are funded and executed by partners.
Do not require significant investment of time or money from Voyager.
Moving up the slide the seven programs depicted in yellow represent our collaborative programs with American for these programs voyager's fully reimbursed for our collaborative research.
The six programs depicted in orange at the top of the slide represent our wholly owned pipeline.
This is where I will focus today.
Turning to slide six you can see that are wholly owned pipeline now includes three programs for all Simers disease.
Our lead program is our humanized anti tiro antibody, which is advancing towards initiation of buying D. Enabling studies this year.
We continue to expect to file an INV in the first half of 2024.
Additionally, we continued to conduct early research on our Taio gene silencing program, which we introduced earlier this year.
This program utilizes Vectorized S irna delivered with a tracer capsid to reduce taio expression in the brain.
Today, we are introducing another early research program in our Alzheimer's disease franchise.
This program, we are combining vectorized anti a beta antibody with a tracer capsid I will turn the call over to Todd who will talk more about this program momentarily.
But first I want to explain why Voyager has chosen to focus three of our six only own programs on all famous disease.
On slide seven a glance across the top row highlight some of the recent progress with anti amyloid antibodies.
<unk> represents tremendous first steps toward modifying the course of all Simers disease.
Turning to the second row milestones on this slide there is an increasing body of data demonstrating the role of Tao and Altzheimer's disease.
I think of amyloid as the trigger and the bullet.
There's a tipping point at which increasing amounts of amyloid caused how to spread and thats spread of towers, what causes narrowed regeneration.
Data reinforced this demonstrating that anti amyloid treatment showed greater clinical benefit in patients with lower Cao burden.
Ultimately, we need to better understand the clinical efficacy of anti amyloid treatments by stage and subtype we.
We may already be starting to see evidence of complete responders partial responders and non responders to empty amyloid treatment based on recent phase III data <unk>.
Complete responders may not need any further treatment than anti amyloid.
But partial responders may be appropriate for a combination of anti amyloid an antique out their fees.
And non responders to anti amyloid might be candidates for switching to antique tile monotherapy.
In short this is a disease that affects millions of people.
The field is making great progress against multiple targets and there is still much work to be done.
Now I will turn the call over to Todd to talk about our new anti amyloid gene therapy program.
Thank you al Please turn to slide eight.
As L mentioned, there are now multiple FDA approved anti amyloid antibodies for Alzheimer's disease.
We think of Vectorized anti amyloid gene therapy may offer the benefit of providing similar disease modifying efficacy with a single dose.
Additionally, on more research is needed.
Is biologic rationale to suggest the gene therapy approach to targeting amyloid may reduce the risk of amyloid related imaging abnormalities were ARIA.
And a gene therapy approach anti amyloid antibodies are steadily secreted by cells in the central nervous system and.
Thus, we would be avoiding high antibody concentrations that necessarily follow intravenous antibody infusions.
Moreover, the antibody would first engaged the beta amyloid deposited in and around amyloid plaques.
Rather than the beta amyloid depositing around blood vessels.
Both mechanisms may reduce the risk of Maria.
Please your has a long history of antibody expertise elite.
<unk> program is or anti Kelly anybody Eli <unk> zero one.
Although the white one is not a gene therapy. We previously shared data at the Alzheimer's Association International Conference in 2022.
<unk>, we had picked her eyes antibodies from this program and achieved substantial anti tell anybody expression in the hippocampus cortex and CSS of mice.
Which was sustained seven months after a single administration.
In addition, we have also vectorized an anti her to anybody for the potential treatment of brain metastases from breast cancer.
So this gives you a flavor for voyager's working antibodies as a whole and didn't pick the rising antibody specifically.
And this new program Vectorized anti a beta antibody and delivered it using a novel Kansas.
Preliminary data and mice as shown target engagement with amyloid plaques following a single Ivy administration of the Vectorized antibody with the BBB penetrance catch it.
We assume this using vectorized murine antibodies and Victor Iced humanized antibodies and we are currently evaluating antibody payloads with our trace of candidates.
I will now turn the call back to al.
Thank you Todd.
Turning to slide nine as you can see Voyager continues to execute on our milestones with.
We secured partnerships with Pfizer Neurocrine, Novartis, and now Sangamo and the company as well Capitalised with approximately $273 million in cash on our balance sheet.
We selected a development candidate for our anti Tiro antibody program for all Simers disease.
And we launched three new early stages gene therapy programs, one for Huntington's disease, and two for all Simers disease.
We also continued to add incredible talent to our team last quarter. We welcome George scan goes to our board of directors.
Earlier this month, we appointed Jacquelyn Fahy Sandell, as our Chief legal officer, and she has already adding tremendous value.
Looking forward, we continue our work to break through the barriers constraining the fields of gene therapy and neurology.
We expect to identify a lead candidate for our wholly owned as so do you want <unk> gene therapy program by the end of this year.
As we look towards 2024 and 2025, we anticipate the potential for multiple IMD filings across are wholly owned and collaborative and or licensed programs.
This translates into multiple opportunities to earn milestone payments and even more importantly, once clinical trials begin several shots on goal to establish human proof of concept for our tracer cabinets.
Furthermore, there is potential to see early biomark 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, it's a very exciting time at Voyager and we look forward to continued execution this year and next.
With that we are happy to take any questions you may have operator.
Thank you so much for centuries, and as a reminder to ask a question you will need to press star one.
One on your telephone the way they are.
Question can you start one one again.
And could you stand by while he compiled acuity roster.
Your first question comes from the lineup.
The security's. Please ask you a question.
And thanks for taking our questions.
The Alzheimer's programs are you able to share any information regarding the choice of the antibody.
<unk> and Uhm, how do you plan to mitigate risks associated with area for example, and I know you mentioned.
Basically.
Not on vessels and curious uhm. If there are certain antibodies are aware of that can do that.
And last name would you be able to see it.
Alrighty I can be in the same contract I'm thinking maybe that's X ray style.
Four two pronged strategy and I have a quick follow up.
Thanks June this is alexandra, but we haven't disclosed which ain't that much of the empty amyloid antibodies, we're gonna vectorized yet.
We have a choice.
But and we're doing experiments.
Several of them now or at least more than one.
Anyway.
We will disclose that at the right time in the future but.
The risk of Oreos Rio and.
Nicely pointed out the reasons why we think we're going to minimize the risk of already over that drives antibody. Nevertheless.
Something to keep in mind, there's two things I would say here one is that the risk of area is mostly in the first six months and one year.
And so we're still a concern we may wait until most of the risk task.
And therefore use the G therapy is more of a maintenance after the initial treatments for six to 12 months.
And then the second thing is that we are we have some preliminary experiments regulating gene expression with a small molecule, which we may be able to then turn on or turn awkward adjustment dose in the future. So.
Both are things we are actually contemplating at this point in terms of an AI.
You're right that we haven't vectorized.
Hi, Taro knockdown program.
At the present time, we don't plan on combining the two into the same vector.
Got it thank you for that and.
Deal with Sangamo, it's really interesting because they got the license or cast for the prion disease yet.
Months later prevail a subsidiary of would be like living American agreement with <unk> to develop a novel cash set for prevails Parkinson's program.
Using it amusing at the same time, we would appreciate it as a kid elaborate.
On.
On the deal, giving you our competitive position on a similar gene therapy program for purchases. Thank you.
Yeah. Thanks June .
I really can't speak to prevail strategy here, however, I can verify that check about did come to us.
The capsule for the prime disease programs.
Alright.
<unk>.
Just a quick follow up I mean, obviously.
Are you concerned at all are there are there are mechanisms in place to ensure that whatever they may learn from Europe half of it is not.
Space within Sangamo, or maybe with continuing Saturday.
I'm up at night.
Two competitors.
Wow.
We trust Sangoma I know the scientists <unk> very well.
Dot take good care of our of our cap said as well as any of the intellectual property around them.
Thank you and I can just regular progress.
Thank you so much.
Your next question comes from the line Alright, Chico Fetterbush. Please.
Please ask a question.
Hi, this is Sandra.
<unk>. Thank you for taking my question.
One question for Pete Pete.
How should we be thinking about R&D expensive as.
As you're heading into 2024.
You'll be in a position to file a 90 and so how much of a pickup should we be expecting here.
Ingrid.
I appreciate the question and we don't really provide tremendous guidance with regard.
<unk> or R&D expenses.
Going forward into 2024 at this point.
I would say that if you look at our R&D burden for the first six months of this year and in the most recent quarter or burn is up about 50 plus percent on the R&D side overall from a financial perspective, you see that from an Opex perspective.
From a cash flow perspective are overall burners.
Tracking nutrition to about 15% higher.
That's on a six month basis.
If you look at last year in 2022.
Byrne was $78 million for the full year. Our first six months of this year is $45 million on a net basis.
That translates to that 15% increase so hopefully that helps you.
Thank you for <unk> net.
You are.
<unk> I guess about burn you know it's a good concern I wouldn't say that we will continue to exercise financial discipline.
These early stage programs are relatively.
Not very intensive in terms of resource requirements for early stage programs and.
And I would also say that we choose programs based on our ability to rapidly achieved proof of concept and through the risk.
Quickly as possible in the clinic.
So, but I want to end by saying Ah reiterating that we will continue financial discipline and maintain a healthy cash around way.
And now I agree with you wholeheartedly I think that really shows in the cash balance that we have closed the quarter $273 million on the balance sheet.
Good runway going forward and we continue to reiterate that carries us and two 2025.
Thank you.
Thank you so much. Your next question comes from the lineup Jack Allen a scan. Please go ahead.
Listen to the team at all the progress made out of the corner and thanks for taking the question I guess the one.
The key question I have is around the town.
The first half of next year I guess.
Any additional color you can provide that relates to pre IMD activities I need to be completed to get that done and submitted.
Yeah, So Jack.
We did have feedback from the FDA in the form of a pre <unk> interaction earlier this year.
And so our timelines are based on what we learn on that feedback.
More needs to be done to get to an int.
The main thing of course is the toxicology studies.
Studies that we will be initiating shortly as.
As well as of course skewing Ah manufacturing. So we can start dosing patients and I'm happy to say that we are on track on all these fronts and <unk>.
Continue to believe will file and IMD in the first half of next year.
Okay. Thanks, so much.
Mmm how much your.
Your next question comes from the line of feeling that too TD Communists. Please go ahead.
First on the side, one AOS gene therapy program discussion a bit more detail the work you're doing to identify belief candidate.
What experiments in particular do you think will differentiate among the candidates that you have in development currently.
Okay question.
To answer that.
So thanks for the question.
We're really excited about our cats.
Across the AVP and we're in the process.
The optimal combining the optimal transgene with the optimal cabinet and so we're doing experiments and Naima primary bye now.
And you might be asking about how did you choose the particular.
<unk>.
Okay. So our process, we had a long history at dragging us Irna's at X laser.
He has a process by which we first screen identify <unk>.
And then begin screaming that the next generation components.
Oh variety of different characteristics.
Look at specificity for the change we look at the ability to knock it down with.
<unk>.
The city I'll make sure that we don't have a substantial off target effects and all of those things come together to identify the optimal transfer and if I remember correctly.
Use the amount specific caps.
One of the Specter I've, thus irna that you've got some very nice data in mouse models of <unk>.
<unk> that's true on the 293.
<unk> animal models greatly extended lifespan.
Capacity.
Presented that accomplishes as well.
That's really helpful. And then one last question.
From us the slides note that there are discussions ongoing for potential partnerships can you give us some sense of what type of partnerships are interested in what do you think would create value for them for the company and shareholders.
Yeah. So thanks, well at a new can see we have a history of doing.
A variety of different types of deals.
I'd say on one enemies spectrum, we have a deal that we did with Pfizer Novartis, where we.
Basically captured licensing agreement.
We do very little work after after they choose the caps.
They have all the capabilities and house to prosecute those programs and.
And they run with it on the other extreme I would say we have the nerve.
Collaboration where it's more of a.
Program collaboration around GBA, one and three undisclosed neuro targets as well as the prior arrangements around feedbacks ataxia.
And for these <unk>.
Much more sort of research collaborators if you will.
Reimburse us for our expenses, but we are working hand in hand with them.
And.
But the progress of theirs, they make all the decisions we try to be as collaborative and helpful. As possible and then we have.
We have those I would say are the two extremes.
And where we can do anything in between those and where.
Look we want to help patients.
Grow shareholder value over time.
Will do will do whatever makes sense for for both.
That's very helpful. Thanks for taking our questions.
Mmm thank.
Thank you. So much. Your next question comes from the lineup Shannon to you of Wells Fargo Securities. Please ask you a question.
Alright, thanks for taking our country and <unk>. So I have a two part question for the new and.
Program. So first I know you don't want to the east coast asset, but may I ask would you be targeting <unk> <unk> <unk> <unk> <unk>.
I think the comments and engagement the plague does that mean, it's targeting.
Targeting the <unk>.
<unk>.
The tissue concentrating you want to achieve might be different from the negative and my body strategy. So far.
Concentration you are targeting.
Okay.
Hollywood beat her dose level that will be required to achieve that take.
Take your concentration thank you.
And I'll take the first question and I'll ask Bob to answer the second one.
In terms of the antibody.
We will not be targeting we will not be using antibodies that target the monitor.
Those darn work essentially a sore loser map is a clear example.
I just haven't works, we will be targeting we will be using antibodies that favor. These.
<unk> illegal nurse as well as insoluble fibrils and examples of that would include for example, aducanumab in mcadam at both of which are selective.
Or aggregated forms of data either as a large soluble oligomers, including prototype.
Or and as well as the February amyloid as president and the amyloid plaques.
And the reason why we choose those is that there is a very good validation, obviously human clinical trial and of course, they have been FDA approved.
One of them has even been fully approved recently, so so I think the the President's is there.
For that.
And do you want to take the second one.
I think so I want to confirm that I have.
The signal incorrectly it's a question about the.
Cetera antibody and how it differs from straight antibiotics is that right.
Right in the tissue concentration you plan to achieve and probably the dose level that might be required.
Thank you.
So I can't comment specifically on that.
Tissue concentrations in the doses will be going into clearly as it can take west of learning from the field on the E.
Antibody concentrations needed for efficacy and what we think potential advantage.
Transaction is beyond a single dose which of course could add.
Implications for being able to treat large numbers of patients.
But as we can talk to that in a call and we hope to avoid the potential reactions is already that you get when you and cheeses, an antibody necessarily get a peek Dylan.
Delivery at peak of antibody exposure.
The vascular system.
We'll be treating and a constitutive fashion so.
So we hope to avoid that high exposure and we hope that the encounter the plaques.
Brain before.
It's encountered in the blood vessels, we hope to have some advantages there as well.
So I would guess summary of that would be that we're sort of targeting the AUC side of things rather than the Max.
That we achieve with antibodies because see max's more related to ARIA and AUC, it's more related to efficacy.
I would also say that we know the concentrations and spinal fluid you have to achieve.
Both of those antibodies.
And they're in the typical range, 100.5% brand new plasma ratio. So so.
There's been a lot of published information from animals as to what antibiotic concentrations are needed for removal amyloid. So so so the nice thing is this is it the risk program in many ways, we're going after a highly validated target we have a <unk>.
Quantitative information that we can use for drug development, we understand the major side effects and thoughts about how to mitigate those and that's the kind of thing that we like to go after a highly validated targets.
Efficient path to proof of concept.
Got it thank.
Thank you.
Mhm.
Thank you so much. Your next question comes from the lineup Matthew fashion point of Oppenheimer. Please go ahead.
Hey, guys congrats on other progress on.
<unk> and thanks for taking our questions. So we were wondering if you had any thoughts following biogen's acquisition of Riata, which obviously reflects a pretty high value for the friedrichs a taxi opportunity. So how are you thinking about any differentiating factors for your essay gene therapy program, obviously prisons.
Potentially versus Sky Claris.
Which you have collaborated with <unk> and any advantages as a potential later follower and then proceed if you could just please remind us any timing on the potential for milestones in that as a program that would be very helpful. Thank you both.
All right I'll start amount last Scott Pete to talk about the milestone, but yeah. So I'm excited.
<unk>.
Right right sat alone with the very first drug ever approved for FAA.
Shows evidence of disease modification.
The worst thing.
This was all published in neurology and I thought the data were pretty convincing or when I first saw the paper.
Published.
And so I think it's a breakthrough and I would love for US to also continue on that trend and make.
Further progress.
With Fas the complexity of this by complexity here is that is that involves the central nervous system, but also the higher and and the reality of drug really targets. The CNS side effects because the outcome measure that they look that was more oriented toward neurological outcomes and so obviously this is a.
The program.
In the hands of neural credit their decision, but I says, but.
It can't hurt to have.
Regulatory precedence established by another company.
Outcome measures that work and that have been accepted by regulators.
And look the tears.
If you want to replace the dream, it's auto Somal a recessive so.
The patient to having a disease basically aren't making protection.
We need to replace for tax and in some way.
And our approach of course is to is to use gene therapy.
And.
And you have to get the best right here, because too much of a taxi, which is probably not good either so so this is all in the capable hands of our colleagues nurturing and.
And I'll, let him answer the question more specific fees respect to the program.
Give that with regards to spit.
Specific guidance on milestones associated with the FAA program, which I think was the.
The second part of your question. So what we've provided in the past is kind of a full summary of what the 2019 Eurocrat agreement looks like in terms of future milestones those.
Those milestones or about $1.4 billion, approximately we have not guided to provide detailed guidance with regards to what the specific next milestones are on that program.
I do think what I would highly not just for <unk>, but also for some of our other partnerships. We do have a number of milestones that potentially could come do.
In the next couple of years here on all of those partnerships as al alluded to earlier, we've got <unk>.
11 partnered programs now seven with our current alone.
Three in the original 2019 agreement, which includes up bag.
Those milestones have not been factored into your cash runway as of yet and potentially could benefit us greatly as you move forward here, so that would be what I would say with regards to that.
Okay.
[noise] helpful really appreciate dancer.
Thanks, and congrats again.
Thanks very much.
Alright, and our next question comes from the lineup Ross <unk>. Please go ahead.
As opposed to the other programs of your pipeline. Thank you.
Yeah. Thanks for the question. So actually are we do we do not have a vectorized antibody against a program we have essentially a passive intravenously administered antibody monoclonal antibody vectorized.
After we achieved proof of biology.
We will achieve that.
If we do we have a choice of moving forward with either continuing with the past was the past those antibodies or going to factorization. So so that's so I just wanted to clarify that.
I mean.
The whole approach here is to choose programs, where we can get proof of biology very efficiently for example, the antibody to.
Taro, we think we can do a proof of biology experiment and humans would swap out 25 patients per dose.
And a one year study how.
How pet imaging, we think we can determine whether we block.
The spread of pathological tower.
And so so we liked that because it's a rapid path to the risking and proof of biology.
And then after that as I said, we haven't choices.
Arriving or not I hope that clarifies or answers your question.
Yeah. Thank you.
Mhm. Thank you so much.
And for center staring no further questions at this time I would now like to turn to clients fans back to you for closing remarks.
Well, thank you very much for all the great questions and.
If you have any further questions. Obviously, we are ready to.
Please call us and we'll do our best to answer them, but thank you very much for your interest and larger.
Thank you for centuries, and this concludes today's conference call. Thank you for participating and you may now disconnect have a good day.
Mmm.
[music].