Q4 2021 Voyager Therapeutics Inc Earnings Call
Operator: Thank you.
Operator: Thank you for calling Conference ID please.
Operator: It is the Voyager Therapeutics.
Operator: Do you happen to have the conference ID number?
Rachel Smith: No, there's no number.
Operator: That's what was provided as the ID, the name of the call.
Operator: It's Q4 2021.
Operator: Thank you.
Operator: One moment please.
Operator: Okay.
Operator: May I have the spelling of your first and last name please?
Rachel Smith: Rachel, R-A-C-H-E-L, Smith, S-M-I-T-H.
Operator: Your company name please?
Rachel Smith: Ayara, A-I-E-R-A.
Operator: Thank you.
Operator: Your line will be on hold until the conference begins.
Operator: Thank you.
Operator: Good morning, and welcome to Voyager Therapeutics' fourth quarter and full year 2021 conference call.
Operator: All participants are now in listen-only mode.
Operator: There will be a question and answer session at the end of this call.
Operator: Please be advised this call is being recorded at the company's request.
Operator: I would now like to turn the call over to Julie Bjurik, Vice President of Finance at Voyager.
Julie Bjurik: Thank you, Operator.
Julie Bjurik: Good morning, everyone, and welcome to this conference call to discuss our most recent license option agreement
Julie Bjurik: and to review our fourth quarter and full year 2021 results, both announced this morning.
Julie Bjurik: A replay of today's call will be available on the Investor section of our website approximately two hours after completion of this call.
Julie Bjurik: After our prepared remarks, we will open the call for Q&A.
Julie Bjurik: As a reminder, various remarks that we make during this call about the company's future expectations, plans, and prospects
Julie Bjurik: constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.
Julie Bjurik: Actual results may differ materially from these indicated by these forward-looking statements as a result of various important factors,
Julie Bjurik: including those discussed in the risk factors section of our most recent annual report on Form 10-K, which is on file with the SEC,
Julie Bjurik: and as updated by our subsequent filing.
Julie Bjurik: In addition, any forward-looking statements represent our views only as of today
Julie Bjurik: and should not be relied upon in representing our views as of any subsequent date.
Julie Bjurik: Except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change.
Julie Bjurik: I'm joined today by members of Voyager's leadership team, including our Interim Chief Executive Officer, Michael Higgins,
Julie Bjurik: our Chief Business Officer, Alan Nunnally, and our Interim Chief Scientific Officer, Glenn Pierce,
Julie Bjurik: Board Director, Al Sandrock, and Chief Operating Officer, Robin Schwartz will also join the Q&A portion of the call.
Julie Bjurik: Now I will turn the call over to Michael.
Michael Higgins: Thanks, Julie, and good morning, everyone.
Michael Higgins: I'm extremely pleased to share that earlier this morning we announced the License Options Agreement with Novartis
Michael Higgins: for target-specific access to our proprietary Tracer AAV capsids.
Michael Higgins: This transaction is the second major licensing agreement around our novel capsids in the last five months
Michael Higgins: and provides Novartis with access to our novel AAV capsids and options to license our capsids for potential use
Michael Higgins: with three undisclosed CNS targets plus options to access capsids for two additional targets to be determined in the future.
Michael Higgins: The transaction further validates the potential of our Tracer capsid discovery platform
Michael Higgins: to broadly enable a new wave of AAV gene therapies across the field and across our internal pipeline.
Michael Higgins: It's also complementary to our agreement with Pfizer announced last October.
Michael Higgins: That transaction provided Pfizer with target-specific access and option rights to our Tracer capsids
Michael Higgins: for exclusive use with one CNS target and one cardiovascular target.
Michael Higgins: Together, these major transactions reinforce our corporate strategy to pursue internal
Michael Higgins: and external applications of our Tracer platform.
Michael Higgins: Importantly, the targets and transgenes involved in the Novartis and Pfizer collaborations are distinct from those planned for our internal pipeline.
Michael Higgins: Outside of these agreements, we continue to retain global rights to our TRACER AAV capsid platform, as well as all capsids arising from its use with other targets.
Michael Higgins: We believe there is considerable potential for future business development opportunities around the TRACER platform,
Michael Higgins: given the focused nature of the deals we've done to date, as well as our expanding and maturing capsid library.
Michael Higgins: We view these agreements as critical validation for the potential of the TRACER capsid discovery platform
Alan Nunnally: For more on this, I'd like to now turn the call over to Glenn to review the progress we're making with our platform and programs.
Michael Higgins: to enable gene therapies that may target desired cells and tissues with greater specificity at lower doses
Glenn Pierce: Thank you, Alan.
Michael Higgins: and with fewer off-target risks than conventional AAV serotypes.
Glenn Pierce: We have oriented Voyager to address some of the most critical challenges facing gene therapy, potentially unlocking the tremendous opportunity this modality holds to help patients.
Michael Higgins: We believe that our approach could enable a new wave of AAV gene therapies to treat serious disease, both in the CNS and beyond.
Glenn Pierce: With first-generation AAV gene therapies, tissue-specific targeting is typically suboptimal and the therapeutic index is often poor.
Michael Higgins: These transactions also strengthen Voyager's balance sheet, extending our cash runway into 2024
Glenn Pierce: As a result, many developers have encountered major toxicity issues if they dose high enough to achieve efficacious levels, with toxicity limiting the potential for optimal efficacy in many cases.
Michael Higgins: and providing greater flexibility as we pursue internal development efforts.
Michael Higgins: forward to keeping you up to date as we continue to move the business forward.
Glenn Pierce: In fact, a recent analysis revealed that clinical trials evaluating cell and gene therapies accounted for approximately 40% of all clinical holds in 2021.
Michael Higgins: The Novartis and Pfizer agreements include substantial upfront and potential downstreet economics.
Glenn Pierce: The ability to produce capsids capable of delivering a payload to specific tissues, whether that's the CNS or cardiac tissue or other parts of the body, with limited exposure elsewhere, is critical to the success of AAV gene therapy.
Michael Higgins: Our incremental investment requirements for these programs are very low under these transactions,
Glenn Pierce: We believe that Voyager has developed a core technology with our tracer capsid platform that may enable meaningful therapeutic benefit for patients.
Michael Higgins: as they are designed to tap into our ongoing platform investments and allow us to expand that investment into our programs.
Glenn Pierce: Tracer allows us to evaluate millions of different possible modifications to existing AAV capsids and select only those capsids that display increased transduction in specific tissues or cells.
Michael Higgins: So thanks, and we will stay in touch.
Michael Higgins: In a moment, I will turn the call over to Alan to discuss the Novartis agreement in more detail.
Glenn Pierce: This approach takes advantage of two distinct methods that historically have not been used by others in this field.
Michael Higgins: But first, I'd like to say a few words about the direction we're taking as a company.
Glenn Pierce: The first is to perform this initial screening along with a series of subsequent screens in non-human primates so that we're selecting for capsids that show enhancements in a species very closely related to humans, not those that have only shown activity in mice, which often don't translate well into human beings.
Michael Higgins: And please reach out to us if you have any follow-up questions in the future.
Michael Higgins: Voyager is leading the evolution of next-generation AAV gene therapies,
Glenn Pierce: Second, we measure the performance of our capsids at each step of the screening process by evaluating the expression level of messenger RNA or mRNA.
Michael Higgins: as evidenced by groundbreaking non-human primate data from our tracer Capsids
Glenn Pierce: In order to produce mRNA, an AAV vector directed to a CNS target must pass through the blood-brain barrier, find the target cells in the brain or spinal cord, get through the cell membrane into the endosomes, get out of the endosomes into the nucleus, produce double-stranded DNA, and then produce mRNA.
Michael Higgins: and our recent collaborations with Novartis and Pfizer.
Glenn Pierce: It's a very complicated process that we don't need to fully understand.
Michael Higgins: Our tracer platform has shown the ability to identify highly differentiated novel Capsids
Glenn Pierce: Thus, when we measure production of the desired mRNA in the target tissue, we've got confidence that we are selecting capsids that not only get across the blood-brain barrier, but that get into cells and deliver and express their payload productively.
Michael Higgins: Have a great day.
Michael Higgins: with substantially enhanced tropism for CNS and other tissue targets in non-human primate studies.
Operator: Take care.
Glenn Pierce: The TRACER approach has enabled us to identify and find capsids that surpass the delivery capabilities of the parental vectors that we're working with, including AAV9 and AAV5, by considerable margins in non-human primates and other animal species.
Michael Higgins: We have proven experience in the design and development of diverse payloads and prosecution of preclinical validation studies.
Glenn Pierce: An initial set of our proprietary AAV vectors from our first campaign to readout have demonstrated superior blood-brain barrier penetration, enhanced cardiac muscle tropism, and increased transgene expression in target tissues compared to conventional AAV capsids when dosed intravenously in non-human primates, which we believe are a much better model for projecting performance in humans than rodents.
Michael Higgins: We're also capable of manufacturing essentially everything required to evaluate most early-stage gene therapy candidates.
Glenn Pierce: One of our AAV9 derived TRACER capsids from this first campaign achieved a more than 1,000-fold increase in transgene mRNA expression compared with AAV9.
Operator: Ladies and gentlemen, this concludes today's presentation.
Michael Higgins: We're utilizing these capabilities with our novel Capsids to develop therapeutic programs that have the potential
Glenn Pierce: This initial effort also identified an additional capsid that has shown enhanced tropism for cardiac tissue and de-targeting of the dorsal root ganglia compared to conventional AAV.
Michael Higgins: to demonstrate the performance of our Capsids in humans that may ultimately provide much-needed therapeutic benefit to underserved patients.
Glenn Pierce: More recently, a new set of TRACER AAV9 variants have demonstrated improved CNS targeting in both non-human primates and rodents, with certain capsids capable of exhibiting preferential tropism for glial cells.
Michael Higgins: Our potential to help patients extends far beyond what we may achieve with our internal development programs
Glenn Pierce: Achieving glial cell tropism in addition to the capsids that have been previously demonstrated, neuronal tropism, is important because it may enable more effective targeting of certain CNS diseases.
Operator: Thank you once again for your participation.
Michael Higgins: to include an array of therapeutic opportunities that could result from the licensing agreements and partnerships now underway.
Glenn Pierce: We are performing further TRACER screening campaigns to identify additional proprietary AAV9 and AAV5 derived capsids targeting multiple tissue and cell types inside and outside the CNS.
Michael Higgins: The data we've generated over the past year for our highly differentiated Capsids have been particularly exciting
Glenn Pierce: In parallel, we are further refining the promising capsids already identified to enhance desirable characteristics for use in gene therapies to treat a broad range of diseases.
Michael Higgins: in demonstrating our ability to cross the blood-brain barrier
Operator: You may now disconnect.
Glenn Pierce: We look forward to presenting more data from our expanding capsid library in the spring.
Michael Higgins: and access specific tissues and cell types in the brain in non-human primates.
Glenn Pierce: We anticipate our advances in capsid development will have a direct impact on our ability to advance Voyager's programs.
Michael Higgins: We are also moving beyond the CNS, applying our tracer technology to discover enhanced Capsids for targeting other tissues.
Glenn Pierce: We believe we are well-positioned to develop best-in-class programs thanks to our proprietary TRACER capsids that may broaden the therapeutic windows needed to successfully treat diseases with serious unmet medical needs.
Michael Higgins: We expect tracer Capsids to power our in-house pipeline of gene therapy programs.
Glenn Pierce: We're focused on programs with the capacity for target validation at an early point in the development process, establishing more efficient paths to preclinical and clinical proof of concept and the potential to provide meaningful therapeutic benefit.
Michael Higgins: In addition to collaborations with two leading companies in gene therapy,
Glenn Pierce: We believe our capsids may enable programs across our portfolio with systemic IV delivery that harnesses the full vasculature of the brain to reach desired cells while lowering the risk of dose-limiting toxicities.
Michael Higgins: the recent addition of Al Sanrock to a board of directors and executive committee is indicative of the progress we're making.
Glenn Pierce: Our programs leveraging the TRACER capsids for IV delivery include gene replacement programs for spinal muscular atrophy and diseases linked to GBA1 mutations, including Parkinson's disease, Lewy body dementia, and Gaucher disease.
Michael Higgins: Al is a scientific visionary with a long track record of spotting promise in early research.
Glenn Pierce: Knockdown programs for SOD1 ALS and Huntington's disease, and vectorized antibody programs for HER2-positive breast cancer brain metastases and diseases associated with tau pathology.
Michael Higgins: He is already playing an important role working with the leadership team to help shape future
Glenn Pierce: In each of these areas, we believe that our approach has the potential to result in transformational, new therapeutic approaches for patients facing devastating conditions.
Michael Higgins: strategies for our tracer platform and therapeutic programs, as well as beginning his efforts
Glenn Pierce: We expect to present preclinical data on some of our programs at a spring medical conference.
Michael Higgins: to further expand our external collaborations with scientific leaders and industry partners.
Glenn Pierce: One example of the progress we are making is our presentation at last November's Society for Neuro-Oncology Annual Meeting,
Michael Higgins: I'm also thrilled that Robin Swartz has been promoted to Chief Operating Officer.
Glenn Pierce: where we shared positive preclinical data in mice on our IV-administered vector encoding an anti-HER2 antibody payload to target HER2-positive brain metastases utilizing a tracer AAV capsid.
Michael Higgins: Robin is a proven and respected leader whose passion for patients is reflected in her deep
Glenn Pierce: HER2-positive breast cancer patients with brain metastases face major unmet needs.
Michael Higgins: commitment to Voyager.
Glenn Pierce: While approved anti-HER2 therapies are effective for peripheral disease, they have a limited ability to reach the CNS in sufficient concentrations to treat brain metastases.
Michael Higgins: Her 25 years of operating experience from multiple roles at Sanofi Genzyme are a tremendous
Glenn Pierce: By using one of our proprietary CNS-targeted capsids to deliver a vectorized antibody payload, and by utilizing cells within the brain to produce therapeutic antibodies,
Michael Higgins: asset for Voyager.
Glenn Pierce: we've demonstrated a durable effect against CNS tumors in several mouse models.
Michael Higgins: With that, I'll turn it over to Allen.
Glenn Pierce: We believe our ability to make an impact for patients and create value extends beyond our own pipeline with the potential application of our tracer capsids in our partners' programs.
Alan Nunnally: Thank you, Michael.
Glenn Pierce: Our arrangements with partners provide the opportunity to enable new medicines, leveraging the substantial development knowledge and capabilities of these larger organizations.
Alan Nunnally: This is an exciting time for Voyager.
Glenn Pierce: We believe that we'll be able to further expand our impact through additional partnerships.
Alan Nunnally: Our agreements with Novartis and Pfizer substantially expand the number of therapeutic programs
Julie Bjurik: I'll now hand it over to Julie to review financial highlights.
Alan Nunnally: in which our proprietary capsids may be deployed and highlight the potential of our tracer
Julie Bjurik: Thank you, Glenn.
Alan Nunnally: platform to generate future business development opportunities, particularly as our novel capsid
Julie Bjurik: In light of the time we have devoted to today's exciting news and the time remaining, I'll limit and focus my remarks this morning on our cash position and how our transaction with Novartis extends our runway.
Alan Nunnally: library expands within and beyond the CNS, and initial tracer-derived capsids are further
Julie Bjurik: Please refer to our press release and 10-K issued earlier today for details of our financial results for the fourth quarter and full year of 2021.
Alan Nunnally: refined.
Julie Bjurik: Voyager is in a strong financial position as we advance our platform and portfolio forward.
Alan Nunnally: Moreover, these agreements have generated $84 million of aggregate upfront payments
Julie Bjurik: Our cash, cash equivalent in marketable securities as of December 31st, 2021, were $132.5 million.
Alan Nunnally: that substantially strengthen our balance sheet.
Julie Bjurik: This does not include the $54 million upfront payment we are entitled to receive from Novartis under the recently announced license option agreement.
Alan Nunnally: I'd like to provide an overview of the license option agreement with Novartis we just announced.
Julie Bjurik: With the upfront payments from our Novartis and Pfizer agreement, our cash runway now extends into 2024, enabling the company to achieve potential further advances in our tracer platform development and pipeline program.
Alan Nunnally: Novartis may exercise options to license novel AAV capsids generated from Voyager's tracer
Julie Bjurik: With that, I will hand it back to Michael.
Alan Nunnally: capsid discovery platform for potential use with three undisclosed CNS targets and options
Michael Higgins: Thank you, Julie.
Alan Nunnally: to access capsids for two additional targets to be agreed on in the future.
Michael Higgins: We've made tremendous progress since we announced the strategic shift for Voyager about nine months ago to focus on maximizing the opportunities created by our tracer platform and realigning our pipeline to deploy this next generation technology.
Alan Nunnally: Voyager will receive $54 million upfront and is entitled to receive up to $37.5 million
Michael Higgins: We've published very exciting data on some of our first tracer capsids and look forward to presenting more.
Alan Nunnally: in exercise fees for options for the three initial CNS targets exercisable by Novartis
Michael Higgins: Our licensing agreements with leading gene therapy companies provide additional validation for the value of our platform.
Alan Nunnally: within 12 months of signing.
Michael Higgins: And we've enhanced our leadership team with highly accomplished executives.
Alan Nunnally: In addition, Novartis may elect to evaluate capsids for up to two additional targets to
Michael Higgins: Looking to the spring, we have several goals we're working towards.
Alan Nunnally: be agreed in the future, subject to their availability, for $18 million upon selection
Michael Higgins: We expect to release additional data on our capsid discovery campaigns.
Alan Nunnally: of each target, and a $12.5 million exercise fee for selection of the capsid for each target.
Michael Higgins: We've already disclosed some promising data on AAV5-based capsids and capsids targeting glial cells, and we look forward to building on that.
Alan Nunnally: Voyager will also be eligible to earn up to $1.5 billion in potential development, regulatory,
Michael Higgins: On the therapeutic pipeline side, we're continuing to progress our programs toward IND-enabling studies and expect to release further preclinical data in the coming months.
Alan Nunnally: and commercial milestones for products utilizing Voyager-licensed capsids, as well as mid-
Michael Higgins: We're also optimistic about the potential for additional BD transactions anchored by the growing potential of our capsids and therapeutic programs.
Alan Nunnally: to high-single-digit tiered royalties based on net sales of Novartis products incorporating
Michael Higgins: I've never been more enthusiastic about the potential of our technology and of our company to make a meaningful and positive impact on the lives of patients.
Alan Nunnally: the licensed capsids.
Michael Higgins: The range of diseases that could potentially be addressed with gene therapy is vast, but is often limited by the field's ability to deliver these therapies safely and effectively to the right organs and tissues.
Alan Nunnally: The targets for which Novartis will receive rights under the agreement will be distinct
Michael Higgins: We believe our tracer technology offers great promise to overcome these limitations and help unlock the potential of AAV gene therapies.
Alan Nunnally: from those planned for Voyager's internal pipeline.
Michael Higgins: With that, we thank you for your interest and support, and we'll open it up to Q&A.
Alan Nunnally: We retain global rights to all licensed capsids for use with other targets and to all other
Operator: If you'd like to ask a question at this time, please press the star, then the number one key on your touchtone telephone.
Alan Nunnally: applications of our tracer technology outside of the use of our capsids with two specific
Operator: To withdraw your question, press the pound key.
Alan Nunnally: transgenes that we licensed to Pfizer in the agreement we announced in October.
Operator: Again, that is star, then one, if you'd like to ask a question at this time.
Alan Nunnally: This agreement provides Pfizer similar access to our tracer platform for selection of novel
Operator: Our first question comes from Jack Allen with Baird.
Alan Nunnally: capsids for use with one central nervous system and one cardiovascular target in Pfizer's
Jack Allen: Great.
Alan Nunnally: development of AAV gene therapies.
Jack Allen: Thank you so much for taking the questions, and congratulations to the team on all the progress.
Alan Nunnally: As with our Novartis agreement, Voyager retains global rights to all tracer capsids for use
Jack Allen: I guess the first question I had was on the Pfizer partnership and any additional color you could provide with regard to how that's going to date.
Alan Nunnally: with other targets and to all other applications of our tracer technology.
Jack Allen: Have you delivered any of the, I guess, necessary constructs to Pfizer with regard to the first two transgenes?
Alan Nunnally: We received $30 million up front from Pfizer as part of that agreement, and we are entitled
Jack Allen: And any qualitative comments you can make on that partnership would be great.
Alan Nunnally: to receive up to $20 million in exercise fees in the aggregate for two options exercisable
Jack Allen: And then I have a few follow-ups as well.
Alan Nunnally: by Pfizer within 12 months of signing and are eligible to receive up to $580 million
Michael Higgins: Hi, Jack.
Alan Nunnally: in total development, regulatory, and commercial milestones and mid to high single-digit tiered
Michael Higgins: It's Michael.
Alan Nunnally: royalties.
Michael Higgins: Thanks very much for the question.
Alan Nunnally: We are exploring additional transactions with other parties that would provide target-specific access to our tracer capsids, both within and outside the CNS.
Michael Higgins: I think Allen can dive into that one first.
Alan Nunnally: We expect opportunities for outlicensing our capsids on a target-specific basis to expand as we extend our repertoire of tracer capsids with tropisms for varied tissue and cell types.
Alan Nunnally: Thanks a lot for the question, Jack.
Alan Nunnally: Given the broad applicability of the tracer platform to select capsids enhanced for any desired tropism, we hope to address one of the largest obstacles in gene therapy, namely to achieve specific transduction of target cells at doses that avoid off-target toxicities and afford wider therapeutic windows.
Alan Nunnally: So, as you know, from the first deal with Pfizer, there's a 12-month evaluation period during which we provide the constructs necessary for Pfizer to evaluate the capsids.
Alan Nunnally: So that's underway.
Alan Nunnally: It's in process.
Alan Nunnally: They've let us know which capsids they'd like to evaluate for right now, and that work is ongoing.
Alan Nunnally: We'll find out whether and which capsids they'd like to exercise on.
Alan Nunnally: They have until October of this year to make that decision.
Jack Allen: Got it.
Jack Allen: Great.
Jack Allen: And then on the Novartis program, I was hoping you could provide some insights with regard to some of the, you know,
Jack Allen: color around the CNS indications that Novartis is looking at and the relative size of those opportunities.
Jack Allen: And then just my last brief question was how to think about Voyager's advancement of the internal pipeline
Jack Allen: and when we might see an internal product move toward the clinic.
Jack Allen: I know you outlined that there's going to be some preclinical data this spring, but any guidance with regard to movement toward the clinic would be great.
Jack Allen: Thank you so much.
Alan Nunnally: Sure, Jack.
Alan Nunnally: So I'll take the first part of that and then hand it over to Glenn.
Glenn Pierce: So the CNS indications, the three CNS targets under the Novartis deal are undisclosed.
Glenn Pierce: So we can't comment on what those are except that they are in the CNS.
Glenn Pierce: So, you know, there is a focus on rarer diseases in CNS in terms of the limitations of the agreement that will be disclosed in due course.
Glenn Pierce: But it's not really very limiting when we talk about what rare disease is with respect to CNS.
Glenn Pierce: But maybe I'll pass it over to Glenn for the second part of your question.
Glenn Pierce: Thanks Alan.
Glenn Pierce: Hi Jack.
Glenn Pierce: This is Glenn.
Glenn Pierce: With regard to the pipeline, it's an early stage pipeline. We rebooted last August and really began to focus the pipeline entirely on the tracer capsids.
Glenn Pierce: So, this means intravenous delivery for a number of the targets, including the CMS targets that we have in the pipeline.
Glenn Pierce: We have not released any timelines at this point, but suffice to say it's early stage.
Glenn Pierce: We are developing the capsids.
Glenn Pierce: We're developing the production processes and doing the requisite preclinical work to move those into the clinic.
Jack Allen: Great.
Jack Allen: Thank you so much for taking the questions and congratulations again on the progress.
Jack Allen: Thanks.
Operator: Our next question comes from Laura Chico with Wedbush Securities.
Laura Chico: Thank you for taking the question.
Laura Chico: I guess I have one question with regards to, I guess, the overall strategy for Voyager.
Laura Chico: Could you talk a little bit about kind of the balance between the internal pipeline efforts and more of the business development strategies?
Laura Chico: I guess, you know, looking ahead over the next few years, how should we be thinking about kind of the balance between these two dynamics and is one a greater priority than the other?
Laura Chico: And then I have one quick follow-up.
Michael Higgins: Thanks for the question, Laura.
Michael Higgins: It's Michael.
Michael Higgins: Let me take that first and then the team can weigh in if they have any other pieces to add.
Michael Higgins: Obviously, both elements are critically important.
Michael Higgins: Ultimately, we're all about delivering products to patients and providing them benefit.
Michael Higgins: The strategy we've followed thus far is to take full advantage of the great work that the team's done with the Tracer platform.
Michael Higgins: So we have focused early on on getting out the capsids to some other major players in the field to allow those programs to move forward.
Michael Higgins: But we remain a therapeutic company and we are prepared to move forward.
Michael Higgins: We have made a big shift in our pipeline, as Glenn just mentioned, and there's work to be done there.
Michael Higgins: But you should expect that we'll continue to move forward our own proprietary pipeline and we'll continue to do transactions.
Alan Nunnally: Alan, you can talk a little more about kind of the types of transactions people might expect over the coming years.
Alan Nunnally: Sure.
Alan Nunnally: Happy to.
Alan Nunnally: Thanks, Laura.
Alan Nunnally: So there really is every possibility of continued business development.
Alan Nunnally: There are a lot of targets out there, both within the CNS and especially as we consider beyond the CNS.
Alan Nunnally: So there's plenty of opportunity for Voyager to have its own programs with prosecuting and also to help enable other partners in the programs that they're working on.
Alan Nunnally: We're not going to prosecute every target in CNS.
Alan Nunnally: No company is going to do that, let alone in the broader field beyond the CNS.
Alan Nunnally: So I think there's a lot of possible future opportunity in the business development side there, as well as the traditional collaboration where we work on programs together with partners.
Alan Nunnally: We've done some of that in the past, and that's something we'll continue to explore as we move forward here.
Michael Higgins: And, Laura, one additional comment is probably quite clear.
Michael Higgins: These early transactions, in light of kind of the market challenges that are out there and the need for capital for an organization in our state,
Michael Higgins: these early transactions are also quite helpful in us, kind of strengthening the balance sheet and allowing us to make these investments to kind of move the company forward.
Laura Chico: That's helpful, Michael.
Laura Chico: Maybe one quick follow-up then.
Laura Chico: So obviously Voyager has been involved in clinical trials previously with prior programs.
Laura Chico: I'm just curious, I think it was Glenn that made a comment kind of about the regulatory hurdles that some in the sector have faced.
Laura Chico: As you're moving towards clinical efforts with TRACER, I'm curious what additional resources do you need to have on board or are there certain gating factors outside kind of accumulation?
Laura Chico: of preclinical work that needs to occur before you can get to the clinic?
Laura Chico: Thank you.
Glenn Pierce: Hi, Laurie.
Glenn Pierce: This is Glenn.
Glenn Pierce: I don't think there are particular specific gating factors other than the normal gating factors required for putting an AAV gene therapy product into the clinic, which does have to follow a specific pathway based upon safety and efficacy.
Glenn Pierce: But it should be substantially different than the safety profile that's being offered by the conventional AAVs.
Glenn Pierce: So what that means is that while we will need to be on high alert for any safety issues, as everyone is with the conventional AAVs, we're anticipating a much broader therapeutic window with these capsids because they've been designed to offer that.
Laura Chico: Thank you.
Operator: Our next question comes from Divya Rao with Cowan and Company.
Divya Rao: Hi, this is Divya.
Divya Rao: I'm here for Phil.
Divya Rao: Congrats on the Novartis deal and the exciting progress.
Divya Rao: We just had a question on if there were any updates in securing a partner for the VYA-DC clinical program.
Divya Rao: Also, if there was any additional progress that's been made in addressing some of those imaging and clinical assessment concerns that the DSMB had.
Michael Higgins: Thank you.
Michael Higgins: So let me just give you a quick response and then we'll let Glenn give a little more detail on it.
Michael Higgins: So we haven't provided any updates with regard to the discussions or the progress around the program.
Michael Higgins: As you know, the program was put on hold some time ago and we remain in that position and believe that there are significant challenges in terms of the technical aspects of that.
Michael Higgins: So we don't expect to be providing significant updates on that program at this time.
Glenn Pierce: Yeah, this is Glenn.
Glenn Pierce: The clinical work that we did with Parkinson's, as well as the preclinical work with Huntington's disease both involved intraparenchymal delivery of AAV.
Glenn Pierce: We are of the opinion at this point that that is not necessarily a good idea based upon our collective experiences there.
Glenn Pierce: And that is the reason why the TRACER platform offers us so much potential.
Glenn Pierce: The ability to dose intravenously, get across the blood-brain barrier in sufficient quantities to offer a therapeutic effect.
Glenn Pierce: So we have switched our entire pipeline and planning over to intravenous delivery of these capsids for CNS diseases, as well as for non-CNS diseases.
Divya Rao: Thank you, that was very helpful.
Operator: Our next question comes from Yanan Zhu with Wells Fargo.
Yanan Zhu: Hi, thanks and congrats on the Novartis deal.
Yanan Zhu: First a couple of questions regarding that deal.
Yanan Zhu: This partnership seems to have higher economics compared with the Pfizer deal on a per target
Yanan Zhu: basis.
Yanan Zhu: Could you comment on why that is the case and have you had additional validation, additional
Yanan Zhu: data since the Pfizer deal?
Yanan Zhu: And also for the three targets, do you expect to deliver three different capsids or could
Yanan Zhu: it be the same, you know, one same target, sorry, one same capsid for all three targets?
Yanan Zhu: Thanks.
Alan Nunnally: Yeah, so thank you very much for the question.
Alan Nunnally: Indeed, you've correctly noted that the per target economics are increased in the Novartis
Alan Nunnally: deal relative to the Pfizer deal.
Alan Nunnally: Of course, every business development negotiation is different, but to your second point about
Alan Nunnally: what may be behind that, we have, of course, progressed our work on the capsids and our
Alan Nunnally: partner Novartis had the most up-to-date information that was available as we negotiated that deal.
Alan Nunnally: And I think the terms speak for themselves with regard to what we were able to achieve
Alan Nunnally: there.
Alan Nunnally: There is, you know, one difference between the two deals is that the three initial targets
Alan Nunnally: with Novartis are all CNS.
Alan Nunnally: In the prior deal with Pfizer, there is one CNS target and one cardiovascular target.
Alan Nunnally: So obviously there are always differences between any two deals and any two partners.
Alan Nunnally: So but there has been progress in the work and the most up-to-date information is provided
Yanan Zhu: to our partners in our negotiations and discussions.
Yanan Zhu: And I think there's one more part to your question, I'm sorry.
Yanan Zhu: Right, for the three targets in the Novartis deal, are you expecting to deliver three different
Yanan Zhu: capsids or could it be one capsid?
Alan Nunnally: Yeah, so it really could be either.
Alan Nunnally: We do think there's a reasonable hypothesis that some of our capsids, some of our best
Alan Nunnally: capsids may be workhorses that can be applicable for multiple targets.
Alan Nunnally: In fact, it may be the case that Voyager uses one capsid for one or more of its own pipeline
Alan Nunnally: programs and one or more of our partners might end up using the same capsid for some of theirs.
Alan Nunnally: It is equally possible, however, that a partner like Novartis may select three different capsids
Alan Nunnally: for three different CNS indications.
Alan Nunnally: It is important, obviously, to target the cells that you're after specifically.
Alan Nunnally: We've shown data for neurotrophic capsids as well as those that have glial tropism.
Alan Nunnally: Those can be important and it really depends on what the indications are.
Alan Nunnally: But we want the capsids to be fit for purpose and those purposes will depend on what the
Alan Nunnally: target is.
Yanan Zhu: That's very helpful.
Yanan Zhu: And then maybe one question on the platform.
Yanan Zhu: From a safety perspective for systemically delivered AAV gene therapies, I think liver
Yanan Zhu: toxicity as well as complement activation are some of the issues that other programs
Yanan Zhu: in the field have had.
Yanan Zhu: Given that you have increased tropism for the CNS, could you talk about the relative
Yanan Zhu: increase compared with liver tropism or in terms of complement activation?
Yanan Zhu: What is the relative reduction of exposure in those aspects?
Glenn Pierce: Thanks.
Glenn Pierce: This is Glenn.
Glenn Pierce: It depends upon the capsid, but in general, these tracer capsids are offering two possibilities
Glenn Pierce: to increase the therapeutic window.
Glenn Pierce: The first possibility is if we could simply use a lower dose of the capsid, substantially
Glenn Pierce: lower dose.
Glenn Pierce: And by doing that, we are providing a lower dose to the organs that are, and tissues that
Glenn Pierce: are subject to the toxicity.
Glenn Pierce: The second aspect of this is that we have noticed in some of our tracer capsids that
Glenn Pierce: they are de-targeting specific organs.
Glenn Pierce: So for instance, we've got a capsid that is de-targeting the dorsal root ganglia.
Glenn Pierce: We have a capsid that is de-targeting the liver.
Glenn Pierce: And so by pulling those two levers, decreased dose versus de-targeting, and using them either
Glenn Pierce: separately or in combination, we think that expanding that therapeutic window in both
Glenn Pierce: directions will offer us some safety advantages over the conventional capsids that don't have
Glenn Pierce: much of a therapeutic window.
Yanan Zhu: Great.
Yanan Zhu: Thanks very much for the helpful information.
Operator: As a reminder, if you'd like to ask a question at this time, that is star, then one.
Operator: Our next question comes from Suman Kulkarni with Canaccord.
Sumant Kulkarni: Good morning, and thanks for taking my question.
Sumant Kulkarni: It's nice to see all the progress.
Sumant Kulkarni: Welcome to Dr. Sandrock, and congrats to Robin on the promotion.
Sumant Kulkarni: I have a few questions here.
Sumant Kulkarni: So one of your slides shows many-fold expressions for tracer capsids versus more traditional
Sumant Kulkarni: AAV9.
Sumant Kulkarni: Given this knowledge and the different expressions in various parts of the brain, are there any
Sumant Kulkarni: specific portions of the brain that you would like to keep for yourself?
Sumant Kulkarni: That's an interesting way to phrase that question.
Sumant Kulkarni: There is...
Sumant Kulkarni: These capsids do have differential probism for different regions of the brain, different
Sumant Kulkarni: structures within the brain, so you're absolutely correct about that.
Sumant Kulkarni: In terms of keeping anything for ourselves, we've identified a point line that we are
Sumant Kulkarni: interested in, and that pipeline we're continuing to make progress forward on.
Sumant Kulkarni: As you know, there are a lot of diseases, both neurological diseases as well as other
Sumant Kulkarni: diseases that have been approached with AAV therapies that continue to have serious effects.
Sumant Kulkarni: So our thinking really is that if we can get these capsids in combination with our programs,
Sumant Kulkarni: but more importantly, in combination with our partners, to patients that have these
Sumant Kulkarni: diseases, then everyone benefits, and that's really the key.
Sumant Kulkarni: From a strategic point of view, you've done a commendable job on refocusing your strategy
Sumant Kulkarni: now to external partnerships, but there are two very important positions in management
Sumant Kulkarni: that are still on an interim basis.
Sumant Kulkarni: What are your plans to remove that interim strategy?
Sumant Kulkarni: Yeah, so thanks for the question.
Michael Higgins: Glenn and I do remain on board as interim leaders working closely with the management
Michael Higgins: team.
Michael Higgins: Our view is the following, is that we are committed to the company and committed to
Michael Higgins: help us move forward, have conducted and continue to conduct research, and we feel like we
Michael Higgins: can attract the really strong candidates.
Michael Higgins: With this, this is validation of the work that we're doing here, and we think that will
Michael Higgins: further help our efforts.
Michael Higgins: But we're holding up a high bar to be sure we get the right coach that can help us create
Michael Higgins: the value in the future.
Michael Higgins: So we're confident we will get good individuals, and we're feeling good about that.
Michael Higgins: But we're taking the time to make sure it goes well.
Michael Higgins: We're also making sure Glenn and I are working closely with the team.
Michael Higgins: So we have the capabilities we need right now, and we feel like we're executing, and
Michael Higgins: we're going to keep the bar high and get the best candidates possible.
Sumant Kulkarni: Thank you.
Operator: I'm showing no further questions in queue at this time.
Michael Higgins: I'd like to turn the call back to Michael Higgins for closing remarks.
Michael Higgins: Well, thank you, everyone, for your attention and for your interest in Voyager.
Michael Higgins: We're really excited about the, not only the transaction that we announced this morning,
Michael Higgins: but particularly excited about the future.
Michael Higgins: We feel like we've made some nice progress over the past months, and we're looking