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Edited Transcript of VYGR earnings conference call or presentation 26-Feb-19 9:30pm GMT

Q4 2018 Voyager Therapeutics Inc Earnings Call

CAMBRIDGE Mar 1, 2019 (Thomson StreetEvents) -- Edited Transcript of Voyager Therapeutics Inc earnings conference call or presentation Tuesday, February 26, 2019 at 9:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Allison Dorval

Voyager Therapeutics, Inc. - CFO & Principal Accounting Officer

* G. Andre Turenne

Voyager Therapeutics, Inc. - CEO, President & Director

* Matthew P. Ottmer

Voyager Therapeutics, Inc. - COO

* Matthew S. Osborne

Voyager Therapeutics, Inc. - VP of Corporate Affairs, Communications & IR

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Conference Call Participants

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* Dane Vincent Leone

Raymond James & Associates, Inc., Research Division - Research Analyst

* Jackson Dean Harvey

Nomura Securities Co. Ltd., Research Division - Research Analyst

* Jeff Hung

Morgan Stanley, Research Division - Equity Analyst

* Laura Christianson

Cowen and Company, LLC, Research Division - Research Associate

* Yanan Zhu

Wells Fargo Securities, LLC, Research Division - Associate Analyst

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Presentation

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Operator [1]

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Good afternoon, and welcome to the Voyager Therapeutics Fourth Quarter and Year-end 2018 Financial Results and Corporate Highlights Conference Call. (Operator Instructions) Please be advised that this call is being recorded at the company's request.

At this time, I'd like to turn the call over to Matt Osborne, Voyager's Vice President of Corporate Affairs, Communications and Investor Relations. Please proceed.

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Matthew S. Osborne, Voyager Therapeutics, Inc. - VP of Corporate Affairs, Communications & IR [2]

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Thank you. Good afternoon, and welcome to the conference call. This afternoon, we issued a press release, which outlines the results and corporate highlights for the fourth quarter and year-end of 2018 and provides our corporate goals and financial guidance for 2019. The release is available at voyagertherapeutics.com.

Before we begin, just a reminder that the forward-looking statements included in this call represent the company's view as of today, February 26, 2019. Voyager disclaims any obligation to update these statements to reflect future events or circumstances, except as required by law. Please refer to today's press release as well as Voyager's filings with the SEC for information concerning risk factors that could cause actual results to differ materially from those expressed or implied by such statements.

With that, I'll pass the call over to Andre.

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [3]

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Thank you, Matt, and good afternoon, everyone. Welcome to our year-end call and review of corporate highlights. I'll start by summarizing for you our key developments since the start of 2018. Next, I'll discuss the 2 strategic collaborations we've entered into since the start of this year and how these deals support our strategic vision of becoming a leading, fully-integrated biopharmaceutical company. I'll then present our 2019 goals before turning it over to Allison, who will outline our financial results and guidance.

Voyager has had a tremendous 2018 and start of 2019. We took important steps in advancing our lead and pipeline programs. We've continued to invest in our products, discovery engine, vectorized antibody platform and novel capsids. We've expanded our manufacturing capabilities. We've significantly strengthened our management team. We've also entered into major new collaborations. I'll give you more detail on each of these developments and how they position us for success in 2019 and beyond.

So first for VY-AADC for Parkinson's disease, we initiated the randomized, placebo-controlled Phase II RESTORE-1 trial. In December, we held a productive Type B meeting with the FDA, gaining clarity on their earlier feedback. We incorporated their guidance to conduct 2 adequate and well-controlled trials and to the design of the RESTORE-1 trial and the planned RESTORE-2 trial. We've started enrolling RESTORE-1, and we've recently held 2 well-attended investigator meetings.

During 2018, we announced positive long-term results from the Phase Ib open-label, dose-escalating trial, VY-AADC. In November, we shared data from all 3 cohorts, with follow-up data from patients to 3 years' post-treatment in Cohort 1, 2 years in Cohort 2 and 18 months in Cohort 3. We're encouraged with the long-term follow-up data, as we believe it continues to demonstrate that VY-AADC can restore the brain's ability to make dopamine and provide patients with better response to levodopa therapy, allowing more control of their motor function, similar to their response during the earlier stages of the disease.

Our preclinical pipeline programs also progressed very well during the year. We're focused on advancing potentially best-in-class programs that have a high probability of success into the clinic. Our ongoing efforts to optimize the capsid, transgene and delivery approaches are applied to each of our preclinical programs. We leveraged the onetime delivery of these vectors in unique ways to target areas of the brain and spinal cord as well as the relevant cell types within those regions that most prominently contribute to disease manifestations.

In the fall at the ASGCT meeting, we provided preclinical update from both our ALS and Huntington's disease programs. For SOD1 ALS, with delivery of VY-SOD102 into the spinal cord of large animals, we were able to achieve significant reduction of SOD1 gene expression throughout the spinal cord, including an over 80% reduction of SOD1 in cervical motor neurons, which are critical for maintaining respiration.

And for our Huntington's program, administration of VY-HTT01 in the putamen and thalamus of large animals resulted in significant reduction of HTT gene expression in deeper tissues and outer layers of the brain. Preclinical toxicology studies are underway with a potential to file INDs for both of these programs later this year.

We also advanced our discovery programs, novel capsids and vectorized antibody efforts. On the latter, the advancements made with AbbVie on tau vectorized antibody approach will be directly leveraged in our extended work on an alpha-synuclein vectorized antibody.

On the manufacturing front, we entered into strategic arrangements with Brammer Bio and Fujifilm Diosynth to add flexibility and scale across our pipeline, while we continue to add to our in-house capabilities around our core competencies and process and analytical development. We'll leverage our internal expertise in baculo/Sf9 manufacturing as we partner with best-in-class contract manufacturers to ensure adequate clinical and commercial supply.

Over the past months, we've also significantly strengthened our executive management team with the appointment of Omar Khwaja as Chief Medical Officer, Bob Hesslein as General Counsel; and Allison Dorval as Chief Financial Officer. I'm excited to formally begin working with Omar and Bob in the coming weeks and to continue to partner with Allison in her new role.

And lastly, to conclude the summary of key achievements, since the start of 2018, we've entered into 3 enabling and strategic collaborations: one, with Neurocrine, and two, with AbbVie. As previously discussed, the Neurocrine collaboration aligns us with a true leader in movement disorders. We'll clearly benefit from our access to Neurocrine's experience, insight and contributions. We look forward to partnering with them on Parkinson's and FA and to selecting our 2 new joint discovery programs.

We also announced 2 separate collaborations with AbbVie in the past year. We've had a productive collaboration on tau vectorized antibodies since last February. And just last week, on Friday, we announced the expansion of our relationship towards a promising new vectorized antibody target in alpha-synuclein.

I want to move now to comment on how these transactions support our strategy of becoming the leading, fully-integrated gene therapy company focused on severe neurological diseases. Having the benefit of a product engine with more high-quality targets that we can pursue alone, we're advancing programs in 3 ways. In one way, as is the case for our 2 collaborations with AbbVie and 2 discovery programs with Neurocrine, we advance novel discovery programs with a partner from the start, but don't retain future commercial rights. The partner fully funds or pre-funds the research, and we're eligible for near-term milestones or development payments, both prior to and after turning over the program to the partner at IND or Phase I. These payments in turn allow us to fund our own pipeline in a non-dilutive way. We're eligible for up to more than $300 million in preclinical milestone payments over the coming years across these programs.

In another way, as is the case for our AADC and FA programs with Neurocrine and our HD program with Sanofi, we advance existing development programs and candidates with the support of partners' funding and resources, but retain co-development and co-commercialization rights. This provides us with a form of risk sharing with highly capable partners and the ability to derive significant value from multiple programs.

Finally, in the third way, as is the case for our SOD1 ALS program and additional programs to be disclosed, we advance promising programs on our own and retain full upside value. In 2019, we intend to add to this portfolio fully-retained assets through both internal and BD efforts, which takes me to our goals for this year.

2019 will be an important year for the company as we advance our programs, continue to invest in the platform and further execute towards our goal to become the leading, fully-integrated gene therapy company focused on severe neurological diseases. Next quarter, we plan to provide 12-month safety and motor function data from the open-label Phase I trial of VY-AADC in patients with Parkinson's disease treated with the posterior infusion trajectory. Next, we plan to complete activation of trial sites and continue to enroll patients in the RESTORE-1 trial. During the year, we plan to provide longer-term safety, biomarker, motor function and quality-of-life data from Cohorts 1 to 3 from the ongoing open-label Phase I trial of VY-AADC.

We plan to advance our ALS SOD1 and the Huntington's disease programs towards clinical trials. Preclinical pharmacology and toxicology studies are underway to support potential filings of IND applications for both programs later this year.

In addition, we plan to advance our Friedreich's ataxia program towards identification of lead candidates and IND-enabling preclinical pharmacology and toxicology studies.

And finally, we'll continue to identify and evaluate potential business development opportunities and continue to invest in the discovery programs, vectorized antibody platform and new capsid development.

So as this summary fully captured, it's a very exciting time for Voyager. I look forward to updating you on our progress against these goals and our other developments throughout the year.

With that, I'll turn it over to Allison, who will review the financials.

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Allison Dorval, Voyager Therapeutics, Inc. - CFO & Principal Accounting Officer [4]

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Thanks, Andre. I'll spend the next few minutes reviewing the financials and guidance before we move to Q&A.

Voyager reported a GAAP net loss of $22.5 million or $0.70 per share for the fourth quarter ended December 31, 2018, compared to a GAAP net loss of $11.8 million or $0.40 per share for the fourth quarter of 2017. GAAP net loss was $88.3 million or $2.75 per share for the full year ended December 31, 2018, compared to a GAAP net loss of $70.7 million or $2.64 per share for the same period in 2017.

Collaboration revenues of $2.0 million for the fourth quarter of 2018 compared to collaboration revenues of $6.3 million for the fourth quarter of 2017. Collaboration revenues of $7.6 million for the full year ended 2018, compared to collaboration revenues of $10.1 million for the same period in 2017.

Collaboration revenues reflect the recognition of payments for research and development services that we provided for various programs under the Sanofi Genzyme and AbbVie collaboration agreements and can vary based on quarterly assessments of our efforts under the collaborations. The decrease in collaboration revenues for the fourth quarter and full year 2018 compared to the same periods in 2017 reflect the onetime recognition of amounts allocated to Sanofi Genzyme's rights in the Parkinson's program in the fourth quarter of 2017 and the January 1, 2018, adoption of the new revenue accounting standards, which modified our recognition methodology. These decreases were partially offset by revenue from the collaboration agreement with AbbVie, entered into in February 2018.

R&D expenses of $16.9 million for the fourth quarter ended December 31, 2018, compared to $13.3 million for the fourth quarter of 2017. R&D expenses of $64.9 million for the year ended December 31, 2018, compared to $62.3 million for the same period in 2017. The increase in R&D expenses related primarily to expenditures associated with the development of our pipeline, including costs related to our RESTORE-1 Phase II clinical trial for VY-AADC and increased personnel and facility costs to support the advancement of our pipeline programs.

General and administrative expenses of $8.3 million for the fourth quarter ended December 31, 2018, compared to $5.4 million for the fourth quarter of 2017. G&A expenses of $33.8 million for the year ended December 31, 2018, compared to $19.7 million for the same period in 2017. The increase in G&A expenses is primarily due to increased professional fees, including consulting and legal costs, in addition to personnel and facility costs to support our growing business.

Now turning towards guidance. We ended 2018 with $155.8 million in cash, cash equivalents and marketable debt securities. And we expect to receive an additional $230 million from the upfront payments on our Neurocrine and AbbVie collaborations, inclusive of the proceeds from our planned sale of $50 million of common stocks to Neurocrine.

We expect operating expenses to be in the range of $130 million to $140 million, as we advance our robust pipeline and invest in the infrastructure to support our ongoing and discovery programs, novel capsids and vectorized antibody efforts. These operating expenses exclude any reimbursement of development costs under our collaboration agreements, since these amounts will be recorded as collaboration revenue.

As a reminder, Neurocrine will be funding all development costs on the 4 collaboration programs, including for Parkinson's and Friedreich's ataxia, the 2 programs for which we have opt-in rights.

Based on our current operating plan, we expect to end 2019 with $280 million to $290 million of cash, cash equivalents and marketable debt securities, which we believe to be sufficient to meet our operating needs into mid-2022.

With that, we would like to now open the call up for questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Dane Leon with Raymond James.

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Dane Vincent Leone, Raymond James & Associates, Inc., Research Division - Research Analyst [2]

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A couple from me. Maybe on the AbbVie deal, I just want to ask the expansion of the strategic relationship was great. I'm just curious, can you give us a little bit of color of what you've actually kind of delivered in that relationship today that kind of helped catalyze the expansion of the partnership?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [3]

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Yes, thanks for the question, Dane. So we're almost exactly a year into the relationship with AbbVie on tau, and it's been a very close working relationship. So it's -- both parties are contributing. And at a high level, what we've done so far has been to select, clone, vectorize a number of different research test antibodies targeting tau. We've been evaluating this in various models looking at bio-distribution and pharmacology and starting to work in animal models using novel AAV capsids to be able to evaluate these. So quite a bit of work done in a short amount of time, working hand-in-hand with them. And it was interesting for us to be able to leverage all of that to be able to work with AbbVie, to be able to add a target that we think is very compelling. So I think from both perspectives, short amount of time under the belt, but quite a bit of positive momentum and a lot of interest in applying it to another target.

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Dane Vincent Leone, Raymond James & Associates, Inc., Research Division - Research Analyst [4]

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Great. And if I could just follow up with 1/2 on the investigator meeting that you recently held. A few points of interest there from your commentary. One, just in terms of the feedback from your investigators that are now in the RESTORE program, you had -- there's a number of endpoints that thankfully have all seemed correlated with the administration of VY-AADC. But the feedback from these investigators in the clinical community that they've been expressing as you move towards the pivotal studies, what's your feeling of where they're really focused on out of the real critical endpoints that they think are going to be more clinically impactful, whether it's the UPDRS or the reductions in levodopa load on a daily basis? Just kind of curious to hear the updated thoughts there? And then the second kind of part of that is, where are you at with integrating some of the MRI-guided technology and now approved V-TAG technology with investigators and what's been the reception?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [5]

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Yes, thanks for these questions. I'll ask Matt to start with addressing these.

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Matthew P. Ottmer, Voyager Therapeutics, Inc. - COO [6]

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Sure. Matt Ottmer. So a couple of different questions in there. So for the investigators, I break it down into -- with the RESTORE 1 trial, we're expanding 2 new clinical sites beyond the core group that have been involved with the program for a number of years. And so as we have initial discussions, I'd say what is most compelling to them and then when they see the totality of the data, is -- it's very remarkable how strong the alignment is across all of the measures. Recalling that for this program, one of the things that really stands out is that it's very elegant in terms of replacing the enzyme, being able to see that you're actually getting enzyme activity in the part of the brain where it was not previously evident as a result of the Parkinson's, then over time seeing the reduction in the oral medication across levodopa and then seeing that translate into both improvement around diary as well as patient and physician measures of quality of life. So seeing that all heading in a consistent direction and between the early cohorts at a lower dose and then increasing the dose and improving the delivery, seeing a dose-dependent response, that's what really gets them excited about the potential of the program. Then when you shift into this being a potentially pivotal study and then really focusing on the regulatory aspect and the placebo-control [plant], the conversations are very much around the ability to impact the primary endpoint, which is the diary reported improvement in good ON time at 12 months. That's the one that we've really focused on and in looking at optimizing the way we run this study and we guide the titration of the oral medications to kind of land them in the optimized point, again in a blinded manner. But based on the Phase I experience, the expectation that there's going to be a reduction in the oral medication, that's the part that the trial is really focused on, of course, then expecting the secondary measures to line up as we've seen in the Phase I. But it's really that gold standard of the regulatory. To the second part of your question, delivery is something that we've invested a lot of time and focus on through the Phase I. We're moving forward with the MRI Interventions' ClearPoint System, which is really well adopted across the functional neurosurgery community and was what we used in the Phase I studies. We are also introducing, as you described, our own device developed internally so that we can at sites that have not yet adopted the ClearPoint System, we have the flexibility to provide a fit-for-purpose device, that now has the 510(k) approval from the -- from FDA. We are rolling that out as we activate sites and expect that to be happening in parallel and so that we would have that flexibility as we move forward with the program. Our point of view is that we do not have a preference between the 2. It's really a site-specific decision on how they want to use it.

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Operator [7]

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Our next question comes from Christopher Marai with Nomura.

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Jackson Dean Harvey, Nomura Securities Co. Ltd., Research Division - Research Analyst [8]

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This is Jackson Harvey on for Christopher Marai. I was just curious on the AbbVie collaboration with the vectorized antibodies. If you could just speak a little bit to how you see this fitting into the competitive landscape as more technologies for crossing the blood-brain barrier emerge? In particular, I'm thinking of Denali's transferrin receptor program and even AbbVie's own dual-targeted antibodies?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [9]

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Yes, thanks for the question, Jackson. So it is something that's been a challenge for the [sales] for many, many years. So it's a natural thing and a good thing that there's a number of approaches being attempted to get better penetrance of the -- through the blood-brain barrier. What we leverage here is our delivery capability with our novel capsids to be able to encode for an antibody or an antibody fragment sequence to be able to get the payload broadly distributed to the right cells across the CNS. So we will tell as we get to advance our efforts into animal models and eventually, we hope in nonhuman primates and eventually into the clinic to see how we fare. But this ability to get a significantly enhanced barrier to get across -- to get delivery of a therapeutic payload across the CNS is something that we are seeing early in our efforts and is the basis for -- for this. Now the -- one of the advantages with vectorizing an antibody is that you can -- that's one of the payloads that you can incorporate. If we have success with our novel capsids in being able to achieve this, you can then have the number of permutations for other therapeutic payloads that you can deliver with that same vehicle. So these are things that we're going to look to, in step, explore as well. But to start with the expert in antibody development in AbbVie and to work on 2 compelling targets, we thought was the -- just the right place to start.

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Operator [10]

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Our next question comes from Jim Birchenough with Wells Fargo Securities.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [11]

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This is Yanan on for Jim. So I wanted to just start with a few questions on the Parkinson's disease program. And then I have a follow up on the Huntington's disease program. So for the Parkinson's disease program, would you be able to provide any incremental update on enrollment in RESTORE-1? And also, would you report putamen coverage and AADC enzyme activity data, the biomarker data before -- at some time before the clinical data readout? And also, is there any possibility for some sort of interim analysis for RESTORE-1 and/or RESTORE-2?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [12]

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Yes, thanks for the questions, Yanan, on the PD program. So we are yet to formally start the active collaboration with our partner, Neurocrine, on this, waiting for HSR clearance. So we will work closely with our new partner to share further updates about the enrollment status with the study. But as we were mentioning and commenting on earlier, we just held at the start of this year a pair of investigator meeting where we got the whole community of investigators to get reenergized about where we stand with the program and coordinate it among themselves between referring site and surgical site. So there's a lot of momentum that's very fresh with this. And with now the resources of our partner, we expect we'll be able to really do great progress with the continued enrollment of the program. So we will provide updates as appropriate once we have an alignment with Neurocrine. As to the interim analysis, we consider this in the development of the optimal studies here. But as this will serve as 1 of 2 pivotal trials, we determined that there is too much of a statistical hit to take an interim look. So we will look to have a readout to that 12-month primary endpoint. But again, we will work with Neurocrine to be able to provide periodic updates on the progress on all fronts on our lead program.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [13]

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Great. That's very helpful. And on the Huntington's disease program, just wondering, would you be able to compare your candidate with competitors' AAV miRNA candidate, for example, in terms of the miRNA scaffold differences as well as whether the ability to silence inside the nucleus might be important?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [14]

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Yes, thanks, Yanan. I'll start here. And Dinah could not participate, she is presenting actually at the -- an HD conference as we speak. I'll ask Matt to also chime in here. So Matt, why don't you start and I will complete the response.

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Matthew P. Ottmer, Voyager Therapeutics, Inc. - COO [15]

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Sure. So yes -- so I think as you're aware, it's difficult to compare data across different constructs, different trials, particularly in Huntington's, where there isn't a gold standard animal model in the large mammals. The different companies, different products are being tested in different models, different species, different time points, different methods. With that being said, we're very excited about the data that we shared at ASGCT this past fall where we saw, as a result of our further optimization on the dosing and the delivery, a very strong knockdown across the 3 different places that we measured. We saw an average 68% in the caudate, 67% in the putamen and then 32% in cortical neurons. That data, we think, is a very strong profile and excited to move that forward on track for later -- filing an IND later this year. In terms of the second part of your question in terms of differentiation within the cellularity, I think -- again, we wouldn't want to comment on our competitor's constructs, but we're highly confident that we've got a strong profile with what we've seen with very robust preclinical modeling up to this point.

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Operator [16]

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Our next question comes from Laura Christianson with Cowen.

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Laura Christianson, Cowen and Company, LLC, Research Division - Research Associate [17]

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I'm just curious about how the activation of sites is going for RESTORE-1? I know you're targeting 24. But how many have been activated thus far? And do the centers that have not yet been activated have eligible patients in queue waiting to enroll? And if you have any guidance for how long you think enrollment will take, that would be helpful as well.

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [18]

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Yes, thanks, Laura for the question. I'll ask Matt also to...

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Matthew P. Ottmer, Voyager Therapeutics, Inc. - COO [19]

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Sure. So we're not going to give specific numbers on activation. I think as you're aware, clinicaltrials.gov is a bit of a lagging indicator in terms of activation. But we've made very strong progress this year. In the number of 24, again, it depends on how you count in terms of the number of neurology sites and surgical sites. So we have targeted 9 surgery sites and a total of 19 treating neurology sites. Some of those are the same. And in some cases, the treating neurology sites are separate. We're also, as a result of our FDA discussions at the end of last year, as we look at refining this and now working with Neurocrine, exploring whether or not to add additional sites for a larger-sized study that would also support a second pivotal study and also thinking towards the future of having a strong delivery base to support commercialization. So we expect the activating sites really for the next couple of years across this program to make sure that we can drive the strong enrollment of the correct number of -- type of patients. The second part of your question around, even for those sites that are not yet activated, they certainly have to comply with IRB standards that they cannot be consenting or having super-detailed discussions with patients ahead of IRB approval. But they know these patients quite well and they have ideas in mind, even coming into the investigator workshops of kind of refining among their different potential patients who would be ideal for this study. So that translates into strong momentum as sites activate. And one of the things that we're working with the sites on, and we look forward to welcoming Neurocrine into the collaboration, is how to even more broadly increase that awareness, so that adding on to our Michael J. Fox Foundation partnership, increasing awareness of this trial, we feel will really raise the ability to bring in the right patients into the study. As we described in the past, of course, we want to go fast, but not at the expense of losing the signal that will result from having the right patients measured appropriately. And so we're very comfortable with the progress we're making there.

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [20]

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I'll just add, Laura. In terms of guidance, we've just, last month in January, provided the guidance, that we expect RESTORE-1 should take us 15 to 21 months to fully enroll. So that -- with the progress we've made in a week, since we've made that guidance, we feel very good about the progress and being on track to be able to achieve that.

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Laura Christianson, Cowen and Company, LLC, Research Division - Research Associate [21]

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Perfect. That's great. And then just one more. I'm curious what would make you decide to hold on to an asset rather than partner. You had mentioned wanting to add more programs beyond just the SOD1 that you would advance independently? How do you think about how to separate programs into one of those kind of 3 pathways that you outlined earlier?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [22]

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Yes. So at the moment, as we've done revamping of the portfolio and added a lot of financial resources, capable partners, as I noted, the focus will be on adding additional wholly owned assets to the portfolio. So we have our discovery engine, we have ALS-SOD1, and we have additional programs coming out of there, potentially from the outside, that we'll look to be able -- with our added resources now to be able to advance forward. So -- but as we move forward, we'll continue to have that big picture that we have this ability to bring capable partners to the table. And the mix, again, gives us this ability to both finance the effort, keep important stake in big, important indications and then have our own wholly owned assets. So there will continue to be an assessment, given our circumstances. But for the near term, we will look to add potentially an asset to the wholly retained part of the strategy.

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Operator [23]

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Our last question comes from Jeff Hung with Morgan Stanley.

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Jeff Hung, Morgan Stanley, Research Division - Equity Analyst [24]

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For the SOD1 program, can you remind us what magnitude of knockdown you think you need to see in humans for a clinically meaningful slowing of disease?

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Matthew P. Ottmer, Voyager Therapeutics, Inc. - COO [25]

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Jeff, it's Matt Ottmer. I think it's a difficult one to answer, given that there hasn't been strong efficacy seen in humans yet. If we extrapolate from the animal models, we're certainly very comfortable that when you get above 50%, it translates well into rescue. We'd remind you again, we released some data at ASGCT this past fall that with our delivery directly into the spinal cord, note, we're seeing an average of 70% in the cervical region, 50% in the thoracic region, both of which are critical to retaining respiratory function. And so again, until it's actually demonstrated in humans, no one can know for sure. But we're feeling very strong about that data going into the clinic.

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Jeff Hung, Morgan Stanley, Research Division - Equity Analyst [26]

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Okay. And then, I guess, for AADC, given the co-promote option in the U.S., how are you thinking about the potential size of sales force you would need in that scenario versus previously?

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [27]

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I think it's very early in terms of we need to sit down with Neurocrine and kind of discuss where both companies are going to be at that point. One of the major advantages to Voyager from this collaboration is that we would have had to invest in building out that infrastructure well ahead of time and doing this on our own, probably even before we had all of the data available so that we could be fully prepared to have a strong launch. With Neurocrine's strong infrastructure that they already have and a highly successful launch of INGREZZA, we fully expect to kind of build on that and optimize it for Parkinson's. But again, they have other products that are going to be into Parkinson's ahead of VY-AADC. What that then looks like for Voyager is a couple of years away, but would not expect it to be a large sales force, just because it's a pretty targeted call on the movement disorder centers, but perhaps a secondary in the community neurology. But most of these more advanced patients end up in the movement disorder centers, which are larger academic areas.

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Operator [28]

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Ladies and gentlemen, this does conclude the Q&A portion of today's conference. I'd like to turn the call back to Andre for closing remarks.

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G. Andre Turenne, Voyager Therapeutics, Inc. - CEO, President & Director [29]

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Thank you, operator. And that does conclude our call. So thank you, everyone, for attending, for your questions. Look forward to updating everyone on our progress throughout the year.

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Operator [30]

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Ladies and gentlemen, it does conclude today's presentation. You may now disconnect and have a wonderful day.