Good day, everyone, and welcome to today's Neurocrine Biosciences Update. At this time, Please note this call may be recorded. I'll be standing by if you should need any assistance. It is now my pleasure to turn the program over to Matt Aberneck, Chief Financial Officer.
Good morning, and thank you for joining our call today on short notice to discuss the collaboration announced earlier this morning with Oager Therapeutics. During this call, we will be making forward looking statements. These statements are subject to certain risks and uncertainties. And their actual results may differ materially. I encourage you to review the risk factors discussed in our latest SEC filings.
Joining me on the call from Neurocrine is Kevin Gorman, Chief Executive Officer Kyle Gaynell, Chief Business Development Officer Irene Roberts, our Chief Medical Officer and Eric Benevich, our Chief Commercial Officer. Also on our call, we are being joined by Voyager's CEO, Andre Turent and Chief Scientific Officer, Dina Saabh. During this call, Kevin and Kyle will provide insight into the collaboration, and we will then open it up for Q And A. We'll be ending this call around 8:30 Eastern Time to allow Andre and Dina to be available for their call, which begins at 8:45 Eastern Time. With that, I will now hand the call over to Kevin Gorman.
Thanks, Matt, and thank you, Andre and Dina, for joining the call. Good morning. Good morning, guys. It's great to have you. This is the first of many calls and many in persons that we're going to be having over the years.
We've really enjoyed getting to know the Voyager team, particularly Andre and Dina through this process and very much look forward to a productive collaboration across the four programs that we're working on. Now given our focus on movement disorders and the anticipated launch of opicapone for Parkinson's disease in 2020, Obviously, we're very excited about the phase 2 Parkinson's program, V Y ADC, that Voyager has been sharpening through the clinic for some time now and the encouraging results seen so far in the early studies. But this collaboration is much, much more, just as Voyager is much, much more than just the Parkinson's program. And I'm incredibly excited about the potential impact on patients through the Friedreichs at Taxia and the 2 other undisclosed programs that we're going to be working on. I believe this collaboration is going to catapult, knurk and Envoyager to the forefront joint discovery and development within the gene therapy area for neuroscience.
When you think about collaborations and the way that both Entre and I approach this. To be successful, you have to think about what each party brings to the table. And in the ideal situation to ensure that some of the parts is worth more to get better than a part, Voyager brings a tremendous amount of expertise in gene therapy. Neurocrine brings a rich history in CNS drug discovery and development and now commercialization. Combining our track record with Voyager's leading approach to gene therapy will clearly allow us to pursue life changing medicines.
In addition, each of our respective organizations are going to be advanced beyond what we could have done apart from one another. Now with that as backdrop as to why did we do the deal and why we're so excited Kyle will now give you a bit more insight into the collaboration structure and also respective programs. Once Kyle is done, we'll turn it over
to your questions. Kyle? Thank you, Kevin, and fully agree about what it takes to form a strong collaboration and believe the structure here aligns with the strategic goals that both Kevin and Andre established for this partnership at the outset and our respective companies. Over the past year and more intensely over the past 6 months, we have spent a considerable time, effort and energy performing diligence on these programs and engaging with external experts and KOLs. Needless to say, what we found was to the collaboration announced today, Let me provide a bit of insight on the lead programs and also on the collaboration structure.
Voyager's lead program, BYADC in Parkinson's disease us in a familiar disease state given our partnership with Beal and our anticipated launch of Picopone next year. Where BYADC fits into the Parkinson's Treatment algorithm is help those who have moderate to severe disease and no longer have good control of on time with L dopa. Here, the goal is to deliver the M time aromatic amino acid decarboxylase or AADC to the region of the brain responsible for the processing with dopamine. In doing so, VY ADC has the potential to provide a one time treatment for the repletion of AADC and importantly, to restore dopamine production response to LDopa. Results through phase 1 are encouraging with a sustained increase in on time with up to 3 years of data at this point.
Voyager initiated a phase 2 study RESTORE-one with the 1st patient dose in December of 2018 a second pivotal trial is planned for next year. Switching gears now and talking about the Friedreasetaxi program, This is a program currently at the preclinical stage. Friedreucetaxia is autosomal recessive disease, characterized by reduced propathy and cardiac disease. Oftentimes, patients end up with a loss of sensation, wheelchair dependency, and serious cardiac symptoms, which can lead to death. Currently, there are no approved treatments for previous attacks yet.
We look forward to working with Voyager to advance this important development candidate and the potential to help patients with this serious disease. Finally, As we approach this collaboration, we had several disease states of high interest that we believe Voyager could help us pursue. We have not yet begun work on these programs, but we'll provide updates in the future as these progress from discovery to development. Now let's turn to the collaboration structure. Our press release provides a good overview of the economics.
In general, we are paying $165,000,000 in cash, including $115,000,000 upfront and $50,000,000 in equity at $11.96 per share based on a 25 percent premium over a trailing average. In addition, we We will fund development for these programs with Voyager being eligible to receive various development, regulatory and commercial milestones along with royalties on sharing arrangement where there was share in the cost and forego certain milestones and royalties. As you can see, we have a lot to be excited about and look forward
to this collaboration Thanks, Kyle. Because this call is short, we plan on going no longer, than until 8:30 am. We wanted to keep our introductory statements very brief. At this point, I would like to open it up for your questions.
We'll take our first question from Charles Duncan of Cantor. Your line is open.
Okay. Hi, guys. Thanks for taking my questions. I'll be quick. I just had a couple of, clarifying questions And then one on the Parkinson's program.
Regarding the milestone payments, are these equally split between the 4 programs and then within a program across development, regulatory and commercial milestones or If this is not the case, how should we think about near term outflows say in the next 1 to 3 years for Neurocrine?
Yes. Thanks, Chad. This is Matt, and I appreciate the question. So, you'll see it in an 8 K filed with with Voyager today or this morning. It's out there.
You can see the mix by program and also the split between the development regulatory milestones and then also commercial. So that should be able to give you an insight. It's about 600,000,000 across the programs on the development regulatory front and then also and then $1,100,000,000 on the commercial side of the equation. As it relates to ongoing investment, I'll provide additional insight into what we expect to fund for 2019 on our earnings call next week. And to be clear in the deal itself, we will be covering all costs associated with these programs up into a point when Voyager does have the option to elect into a profit sharing arrangement on the Parkinson's and also the FA program.
So hopefully that gives you the clarity you're looking for, Chaz,
Yes, it does. And on that FA program, it's sixty-forty is, is it 60 Neurocrine or is it 60?
Yes, that's correct. It's a 60 neurocrine.
Okay. And then regarding the Parkinson's program. Frankly, this is a program I've been very interested in for a long time. We used to actually cover Voyager when I say at my last firm. And so pretty cool program from a longer term commercial perspective this makes a lot of sense in terms of synergies for where Neurocrine is.
But could you help us understand your perspective on the clinical value potential in this patient population. I think Kyle referred to one time treatment. At least relative to the administration paradigm. I mean, in your KOL work, what was kind of the perspective on that?
Thanks, Chaz. Irene, do you want to answer that, please?
Yes, I can do that. Thanks, Chaz. Thanks for the question. Certainly, in our KOL work, we know and understand that there is still significant unmet clinical need in part instance patients and especially those that have troublesome, motor fluctuations and of which that is a significant proportion of the parking disease population over time. We're particularly excited about this approach given, as you mentioned, that it is a one time treatment and that it really classifies very much as a precision related medicine in that regard in that this enzyme and the gene carrying this enzyme is delivered to exactly the right part of the brain where it needs to be to form dopamine locally and therefore provide a significant clinical So we do believe there's a lot of opportunity for this medication to add significant value for patients with no to fluctuations moving forward.
The, obviously, the initial population that would be of significant interest is those that have are currently receiving treatments like DBS Obviously, that's a small proportion of the Parkinson's patients, but we believe the opportunity is much broader than that given the data that we've seen date. And we're very interested in continuing to partner with Voyager in their current pivotal program, phase twothree. In order to understand
congrats on the collaboration.
Thank you. We'll move next to Geoff Meacham of Barclays.
Hey guys, this is Scott on for Jeff. Just one quick question. So level, how do you view the ultimate life cycle management of opicapone with the new AATC asset? Thanks.
Thank you for that, Scott. Actually, we see these 2 drugs as being very complimentary. They can be used in the same patient population So they overlap quite nicely. They are both working in order to re store the dopamine balance in these, in these Parkinson's patients to lower the amount of dopamine that needs to be used by that are left open to be used by the physician and to ultimately, lead to a greater on time and obviously less off time. So we enjoy starting next year.
Commercial is a hopefully commercialization next is we, as we filed the on track now to file the NDA for a pair of bone, that was commercializing it next year, we have a nice long patent life to opicapone. So we're going to invest heavily into that. And I think that really smooths the way and opens us up to be able to interact with all the physicians that we will be interacting with when we commercialize, V Y AADC. So they are very complimentary. I think that it allows us to really broaden the span of Parkinson's patients and bring the doctors 2 very complimentary but very different medications.
Do you have anything that you'd want to add to that, Eric?
Other than the fact that we're just really excited about the opportunity to bring another treatment option to these patients that are high unmet need in Parkinson's. You've got 2 different approaches to improving mode of control in these patients and we're really thrilled at the opportunity to bring a new approach to market.
We'll take our next question from Brian Skorney of Baird. Your line is open.
To everyone involved on the deal. Really exciting. Kevin, I was hoping maybe you just kind of contextualize the results that you've looked over gone on to the hood with AAD. Do you think a bit of a controversy is, around a little bit of dose relationship? And what adequate AADT expression you need and durability that you get and how that kind of matches up with what they're seeing in terms of on time without troublesome dyskinesia, as well as actual levodopa doses, So maybe just kind of in your review of the clinical data that you've seen so far, what do you think is the most kind of compelling 1 or 2 point around the efficacy?
Yes, thanks. Thanks, Brian. Obviously, we found the data very compelling. Although, you have to have the caveat at all the data thus far collected as open label. But to go more into the details of this, I'm going to take you back Irene and have her answer that question in more detail.
Irene?
Yes, thank you. Well, first of all, I think, and thanks very much Ryan for the question. I think we're in a very favorable position here with this approach because we are able to answer important associated with dose response and do that using both biomarkers and the clinical data. And so I think it was very appealing to us that we were able to, look from the data. Firstly, in terms of understanding how much of the dose was being delivered to the pertainment real time with the MRI imaging that went along with each injection Secondly, then the PET imaging that can be done in follow-up to the dosing and administration shows the level of expression that you're seeing of the AADC enzyme and allows you to very much understand the degree of activity that you're having within that individual patient.
And then as a result of that, we have seen in the phase 1 data, albeit open label that translate into very clinically significant benefits in both on time without troublesome dyskinesia of up to 2 point 7 hours per day out to 2 years after a single dose administration. And so I think there is a very clear correlation there that we're seeing. It becomes important as we move into placebo controlled trials now required for registration that we fully understand how to move forward in terms of that the approach in those trials. And we are part we'll look forward to partnering very closely with Voyager and with the agency in ensuring that we have a robust package there to answer the question around ultimate clinical benefit.
Great. Thank you.
Thank you. Our next question comes from Paul Matteis of Stifel.
Great. Thanks so much for taking my questions. Just again, on the clinical data side, I was wondering if Neurocrine team could comment on how you've gotten comfortable with the Voyager Parkinson's data given that there's no placebo arm and what you might expect for the performance of placebo in a study where patients are undergoing some sort of procedure and in Parkinson's, I think the placebo effect can be significant. And then secondarily, Can you just comment on separate from the deal value in this transaction? How much investment Neurocrine might have to employ, if any, in the build out of, of manufacturing?
Thanks so much.
I'll take the second part of your question, pretty quickly. On the investment side beyond just the funding for the programs, there is no there will be limited to no investment on getting scale up for manufacturing. So our investment and continued investment will really be the milestones and then also the funding of these programs. As I highlighted earlier. I will provide more insight into our 2019 investment into this collaboration on our call next week.
So I'll we'll go back to the first part of your question, for Irene.
Hey, Paul, thanks for the question. It's really obviously a very important question and one which we've given a lot of consideration to. Not surprising that the phase 1 data or data to this point for this program would be open label. That's very consistent with other gene therapy programs in this area. We do believe that the data from those phase 1 studies was compelling.
Obviously, the question that you ask about placebo response is a very important one. I think that speaks very directly to our ongoing upcoming collaboration with Voyager in terms of that we have the most robust and appropriately powered trials in addition to that, we anticipate working together on the statistical plan to ensure that that's as robust as it needs to be. And that we have alignment on that with the FDA. And so I think we look forward to that collaboration and we look forward to being able to use both our knowledge in this space, historical data from Parkinson's disease trials and ensuring that all of that is incorporated into a very robust package moving forward. We know based on recent feedback that the size of the RESTORE-one trial has been increased and we are and we'll continue to work with Voyager and ensuring that we're putting up both of our sets of expertise together to ensure the most robust program possible.
Thanks, Irene. I think Voyager had said that that study was originally powered for a 2 hour difference between drug and placebo. Is there any update to that or is it still in the works?
I can't comment on that at this point, Paul. We need to have those more detailed conversations. Obviously, in collaboration with Voyager moving forward. But we're certainly very much looking forward to that. I'm very excited about the prospect of working together.
All this is
Kyle. I just want to interject a few things. Obviously, nerve, and we've looked at a lot of programs over the years, a lot of placebo trial trials. And what you don't typically see in those programs are movements of all the endpoints, quality of life measures, things of that sort of all move with increases or decreases in dose depending on how you look at it. And with this program, what we are sin as the mechanism and the durability of the response and the mechanism AADC, that scientific rationale is solid.
We know that role in Parkinson's disease quite nicely. So the next piece is durability. And we have functional data with AADC and non human primates out to 15 years. We've got durability of response and subjects in the phase 1 study out to 3 years. And if you look at the cohorts that were in the phase 1s, across the different doses.
At every measure that's important in Parkinson's disease, you see a dose response and you also see decreases available usage with increasing doses of AADC. So I think that the whole program in its entirety really gives you good comfort that moving into the pivotal trials that this is going to provide a program, but this is going to provide a result that's going to be very positive for patients.
Okay. All right. Thanks Kyle. Appreciate it.
Our next question comes from Biren Amin of Jefferies.
Hi guys. Thanks for taking my questions and congrats on the deal. Maybe just to start on the Parkinson's disease program. Are you planning to initiate the RESTORE-two Phase III trial, this year, or are you going to wait for the readout and data from RESTORE-one before you start that study?
Yes. So, thanks for the question. And Irene has mentioned, we're going to engage deeply with the Voyager team and, and make sure on both the ReStore 1. And then what we're going to do with the second pivotal is adequately powered and structured to give us the answer that we'd be trying to find in those trial. So stay tuned as we engage in those conversations, we'll provide updates along the way.
And also what I would add to that, Barrett, is that our goal here is to really work with Voyager in moving this program and actually all four of our programs along. And so we all have a sense of urgency with these programs. So we want to be very smart about this, but we want to be very nimble keep up and move them quickly.
Got it. And then if Voyager ops in after the RESTORE-one data, what milestones would they forego? Can you just outline what the structure of the deal would look like if they opt in on the Parkinson's program and on the FA program?
Yes, we're not going to get into all the exact details of what goes in or what goes out. We'll likely provide more color as that would be exercised. But they would forgo some of the milestones. And then they would then start sharing in the cost of of the program at that time.
Okay. Thank you.
Yes, this is a last question. From, I believe on a pump.
Mr. Rama, your line is open of JP Morgan.
Hey, guys. Thanks so much for taking the question and congrats on the deal. Just a quick one for me. What are the gating factors to understanding the way and opportunities for the 2 undetermined programs? And what are the timelines there?
Thanks so much. Well, on a problem, there's several programs that we have at Neurocrine had, through our research and our ideas that we really wanted to explore. And as we've talked about these over the years here, we've we've often said it's to ultimately do what we want to do is not utilizing a therapeutic modality of orally active small molecules. It's another therapeutic modality, ideally, gene therapy. This gives us the opportunity to do that.
So what you're going to see is that, that Voyage and ourselves are going to be working through these, this really goes to the heart of the talented Voyager in guiding these programs along in the early stages and getting them ready for the clinic. Once they're ready to hit the clinic, that's when Neurocrine is going to is going to step in on these programs and usher them through. So it's really going to be from the once they enter the clinic going forward that you're going to, that you're going to start hearing from us on these. So I'd like to thank everyone today. And I hope that you, that you now will in a few minutes be going over to the to the Voyager call.
And once again, I'd like to thank Andre and Dana for joining us on this call. And we are very much looking we'd be kicking off this collaboration. Thank you, everyone.
This does conclude today's Neurocrine Biosciences Update. You may now disconnect