Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics Business Update Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. As a reminder, today's program is being recorded. And now, I'd like to introduce your host for today's program, Ian Estefan, Senior Vice President, Chief of Staff and Corporate Affairs.
Please go ahead.
Thank you, Jerome, and thank you all for joining today's call and happy holidays to all. Earlier this morning, we issued a press release regarding a licensing agreement with Roche for rights to our microdystrophin gene therapy for Duchenne muscular dystrophy in territories outside the United States. The press release is available on our website at www.sarepta.com. Joining us on the call today are Doug Ingram, Sandy Mahatme, Bo Cumbo, Doctor. Gilmore O'Neill and Doctor.
Louise Rodino Klapac. After our formal remarks, we'll open the call for Q and A. I'd like to note that during this call, we will be making a number of forward looking statements. Please take a moment to review our slides on the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties, any of which are beyond Sarepta's control.
Actual results could materially differ from these forward looking statements, and any such risks could materially and adversely affect the business, the result of operations and the trading prices of Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10 Q filed with the Securities and Exchange Commission, as well as the company's other SEC filings. The company does not undertake any obligations to publicly update its forward looking statements based on subsequent events or circumstances. And with that, I'll turn the call over to our CEO, Doug Echamps.
Doug? Thank you, Ian. Good afternoon and thank you all for joining us to discuss our strategic ex U. S. Partnership with Roche that was announced earlier today.
I have been in biopharma for nearly 25 years. Over those decades, I have often resisted licensing relationships. In part because I have held a very high standard that for a licensing relationship to work, I have to be convinced not merely that the economic terms were compelling, but that with a partner we could do much more than we could do alone. As we commence this very thorough and competitive process of evaluating an ex U. S.
License for our DMD gene therapy candidates, which is SRP-nine thousand and one, we set for ourselves a very high bar. We insisted first that the economics must be unprecedented, given the unprecedented opportunity of this gene therapy candidate. And we insisted that if we were to consider a license, we had to be convinced that with a partner we could greatly accelerate our mission and strategy to be the world's leader in rare genetic medicine and along the way to transform the lives of patients. And this partnership meets those standards brilliantly. The deal will be transformational for Sarepta.
Indeed, it will be of lasting strategic importance to Sarepta and it will work to accelerate our mission. But before I discuss the transformative impact and strategic rationale, let me provide the broad deal terms. As you will have seen in our press release earlier today, we have entered into a strategic partnership with Roche, pursuant to which Roche will have the right and responsibility to gain regulatory approval for, to launch and to commercialize our microdystrophin gene therapy program SRP-nine thousand and one in territories outside of the United States. Consistent with our strategic goals, Sarepta will have control over and remain responsible for the global development plan and the manufacturing plan for SRP-nine thousand and one. And of course retains all rights to SRP-nine thousand and one in the United States.
While there is a separate option for ex U. S. Rights to certain other DMD programs, which I will discuss momentarily, this partnership does not contain any options or substantive rights to any of our non DMD programs either in our gene therapy or our RNA platforms. In exchange for the right to distribute SRP-nine thousand and one in ex U. S.
Territories, Roche will compensate Sarepta as follows. First, Roche will pay Sarepta $1,150,000,000 at closing, comprised of a $750,000,000 upfront fee and the acquisition of $400,000,000 of Sarepta common stock at a premium price of $158.59 per share. 2nd, Sarepta will also receive up to an additional $1,700,000,000 of payments based on regulatory and sales milestones. 3rd, Roche will additionally reimburse Sarepta 50% of all global development expenses associated with SRP 9,001. And if there are any unique territory specific development expenses that may arise, Roche will pay 100 percent of them.
4th and very importantly, as Roche is successful ex U. S, we will significantly participate. Roche will pay royalties on net sales anticipated to be in the mid teens. And finally, as I mentioned earlier, Roche also has an option to acquire rights to other DMD programs, both in gene therapy and RNA limited to ex U. S.
Territories. That option cannot be exercised for 2 years. If it is exercised, it will come with additional milestones, cost sharing obligations and royalty obligations. So summarizing the financials, assuming success, the non risk adjusted payments inclusive of royalties could reasonably exceed $10,000,000,000 Judge justify the upfront payment alone, this transaction by about an order of magnitude is the largest licensing transaction in cell or gene therapy history. But more, it is also the largest single asset ex U.
S. License of any kind in all of biopharma history. We are very pleased that after a robust competitive process and very detailed diligence on the commercial opportunity, our data to date, our development strategy and importantly on our manufacturing approach and progress to date there. Roche came to the same view as Sarepta on this opportunity, on our plans and progress and made this significant investment in DMD with Sarepta. These are indeed unprecedented economics, but then again this opportunity is unprecedented.
Obviously, we are very pleased with the economics, but more important still, we are truly excited about the strategic rationale of this partnership and its importance to Sarepta's mission to bring a better life to as many Duchenne muscular dystrophy patients around the world as broadly and as rapidly as good science, the regulatory path and resources permit. So commenting on the strategic rationale. First, teaming with Roche serves our promise to work with urgency to bring therapy to the greatest number of patients with DMD. With its existing infrastructure, expertise and shared mission, Roche will accelerate our ex U. S.
Plans. And more than that, Roche broadens our ambition, as they will get to territories that Sarepta would be challenged to address in a similar timeframe even with our most ambitious approach. A world leader in biopharmaceutical sales and marketing, Roche Heppi demonstrated interest and expertise in neuromuscular therapies and rare disease and indeed in gene therapy. Globally, Roche is directly present in over 100 countries. Roche has nearly 100,000 employees marketing and promotion, patient advocacy, government affairs and on and on.
2nd, as we will have as much as $2,500,000,000 of cash available after closing to drive our strategy as we commence 2020. This partnership provides us both resources and importantly focus to execute our plans. To remind, our aspiration is to be the global leader in rare genetic medicine, continuing our RNA commercial success, while advancing our current and next generation RNA platforms, And continuing the rapid construction of our enduring gene therapy engine, one built on a hybrid manufacturing strategy, aimed to have the most significant capacity and expertise in the industry on the deepest pipeline of gene therapy candidates in biopharma today, on a gene therapy center of excellence that can continue to create 1st in class constructs to treat serious life threatening disease and on U. S. Launch readiness for SRP-nine thousand and one, one of the most significant late stage gene therapy candidates all of development.
Given the breadth of our ambition and the amount we have to execute to make our ambition a reality, attempting to fully build out our ex U. S. Infrastructure and the development of commercial access, government affairs, regulatory and all of the G and A expertise in countries and territories around the world at the same time that we're executing our ambitious RNA, gene therapy strategy would not only be very costly, but would be enormously distracting to our mission. So in summary, this transaction accelerates our ex U. S.
Plans. It takes our therapy to places around the globe that we could not reasonably have built to access in a reasonable period of time. It gives us the resources to be successful. It removes distraction and permits us to focus on our strategic priorities and it also ensures not only through sales milestones, but also through mid teen royalties that if and when Roche is successful in bringing SRP-nine thousand and one broadly across the globe, more patients will benefit and if so, the Sarepta shareholder will also be richly rewarded. Sarepta has a bold but achievable vision.
We intend to be the leader in genetic medicine for rare disease and our ex U. S. Relationship with Roche focused on SRP-nine thousand and one is in my view an immense leap forward in making that vision a reality. And with that, I will open the line for questions.
Your first question comes from the line of Debjit Chattopadhyay with H. C. Wainwright. You may ask your
question. Hi, guys. Good morning. This is Aaron on for Debjit. Congratulations on the deal.
And I wanted to ask just one question. So did Roche see all the available data from Study 1, including the NSAA data? And did they have access to the blinded data from Study 2? Thanks.
So Roche did an enormous amount of diligence as one would anticipate for an organization that was willing to make such a significant investment. I can't go into enormous amount of detail and everything they looked at might be inappropriate. They certainly had access to all the unblinded data from our studies, including our very first study 1. They've also had significant access under confidentiality to our manufacturing progress to date capacity process development, analytical development and the like. And so it was based on all of this entire transaction and the economics of this transaction certainly were informed by a significant amount of diligence and access to the teams across development, research, manufacturing, tech ops and commercial.
Your next question comes from the line of Alethia Young with Cantor. Please ask your question.
Hey, thanks for taking my questions and congrats on the deal this morning. I guess I just wanted to see if you could maybe frame for us how Roche might think about the ex U. S. Opportunity in DMD on a patient basis and or potentially on how they're thinking about maybe a sales opportunity basis? Thanks.
Broadly speaking, I'm sorry, apologies Alethia. Just broadly, what how do they see this commercial opportunity?
Probably yes. But like if they can think about patients as well, I mean, it's a big opportunity at QS. So I guess how many people they really think you guys can get if it's helpful or versus like maybe a sales opportunity?
Well, we are I can't speak for Roche on their sales projections and they wouldn't have shared those with us certainly. Probably would have gotten worse for them in the transaction had they to be fair. I will note this. There is an enormous there are enormous number of Duchenne muscular dystrophy patients living with and inevitably dying from Duchenne muscular dystrophy around the world. Know this, just one example.
Outside the United States, every single day and every hour of every day, at least one child is dying from Duchenne muscular dystrophy and it's unrelenting. In fact, it's more than just a patient an hour a day. At the end of that day, another dozen or so children would have died as well. So there is an enormous opportunity. How much of those patients we can get to is something we're going to have to work to.
But I will say, it is exciting to be with an organization like Roche that has to build infrastructure and expertise to get as broad as we can possibly get. I'll just give you one example of a potential opportunity in the future that was nowhere near our strategic thinking simply because we couldn't have anticipated getting there and that is for instance China. Now we've got a lot of work to do and Roche certainly has a lot of work to do to consider how one gets to China. But as many may know, there are a number of very significant biopharmaceutical multinational organizations that have found opportunities in China to treat disease. And in fact the healthcare system in China has really accelerated and matured in the last 5 to 10 years.
There are something like 60,000 Duchenne muscular dystrophy patients living in China and unfortunately suffering and ultimately dying in China. So there is just a breathtaking opportunity with Roche to expand the number of patients that might
Your next question comes from the line of Anupam Rama with JPMorgan. You may ask your question.
Hey, guys. Congrats on the deal and thanks for taking the question. Just a quick clarification question. The $1,700,000,000 in milestones, wondering how that breaks down in regulatory versus sales related milestones? Thanks so much.
Thanks a lot. We haven't provided that detail. I will say there are milestones along the way, near term, mid term and late term milestones, both regulatory and then sales milestones. So it is not all back ended as one example, but we haven't broken the milestones out in any detail.
Your next question comes from the line of Joel Beatty with Citi. You may ask your question.
Hi, this is Sean Egan on for Joel. Thanks for taking my questions and happy holidays. So with all the manufacturing being handled in house, can you provide some color on how velo is set up so you can accommodate a scenario where maybe demand far exceeds supply? And also once the prevalent population is treated, there an agreed upon minimum iCELLis capacity that will be locked into DMD for you guys?
So a couple of thoughts on that. First of all, from a manufacturing perspective, this won't our manufacturing plans and capacity build won't change based on the steel over the next approximately 24 months or so. But as we begin to get more mature and build this out and get a better line of sight in where Roche can take this therapy, then I think beyond that there is going to be the need to build additional capacity. By the time we're building that additional capacity, remember though, this will be an enormously de risked program, will be through 102, will be through 301, we'll have all the process of development and analytical development completely buttoned down at the time we're doing this. So what I would say is for the time being, we are on track and we have sufficient capacity to serve ours and Roche's needs in the short term.
But as we kind of get past 2021, we're going to have to do some thinking and then scale beyond what we already have if we can if for instance, we can begin to access regions, I guess just one example, China that have a breathtaking opportunity. So I wouldn't see an enormous change in our cost basis and expense basis for manufacturing over the next 18 to 24 months. But certainly after that, it may actually have to go up as we the opportunity would go up significantly. But of course, I think everyone would be very pleased to see that.
Your next question comes from the line of Fazeen Ahmad with Bank of America.
Doug, just wanted to get a sense from you about the importance that you're placing and retaining control of the clinical development aspects of the program. I ask because Roche has had some recent experience developing another rare disease drug and so potentially they could have developed relationships with key physicians in this space. And related to that, do you think that your partnership with them will be able to accelerate any timelines for any of the studies that are underway? And maybe can I squeeze in and ask whether or not you've completed Study 2 enrollment? Thanks.
So, well, first, I'll answer the last question first, which is the study 102 isn't complete, but it's near completion as you know with the complete at the end of the year. So we're doing very well there. Going back, so look, it was there was a couple of things to think about with respect to the clinical development. So we wrote an opportunity to review our clinical development plans. They were bought in on the plans and the approach that we're taking, which is a very positive thing.
We do have built into the agreement, not only the responsibility, but for the most part the control over the clinical development plan itself, that's Sarepta and that's generally important to us just from a control perspective. But also know this, we are extremely impressed with our colleagues at Roche. I can tell you not only by reputation, but now in the process of negotiating with them and interacting with them, we've only become more impressed with their attitude as a partner, their ability to move fast, but also of course their expertise across these areas. I think Roche had a competitive advantage over others because they understood these issues in ways that I think others may not have. So certain and we're going to have a joint development committee.
So they are going to participate with us in these plans and we are certainly going to be humble and take their advice. And I do think there is the opportunity, I won't over promise, but I do think there's an opportunity to make us better and probably even have us move faster, because we are associating ourselves with a very impressive, a very successful organization like Roche. So don't imagine that when we say that we're going to we control the clinical development that that means that we're not going to be a good partner and listen loudly to our partner, Roche, who again is we chose in large measure, not simply because they have extraordinary infrastructure, but they have great expertise.
Your next question comes from the line of Matthew Harrison with Morgan Stanley. You may ask your question.
Hey, good morning. Thanks for taking the question. I guess two parts for me. So one, can you just talk briefly about what sort of diligence Roche did around manufacturing and how confident you are in maintaining the timelines that you have set out, including dosing commercial patients, I mean, dosing patients with commercial supply drug by mid year? And then the second one is, can you just clarify related to the cost sharing?
Does that relate only to clinical development? Or does it also cover manufacturing development? Thanks.
Yes. So let me answer the first question. So it relates to clinical development. Manufacturing is our responsibility. So they are not reimbursing us for the manufacturing portion.
So it's all the clinical development, which is substantial. But those just I will just say broadly speaking, Roche and others had access to an enormous amount of detail on where we are from an manufacturing perspective and the progress that we've made from a capacity analytical development and process development perspective and that all in formed the transaction and the partnership that we have today. From a timeline perspective, I will continue to say what I've said for quite some time and we are on track to start Study 301 by about the middle of 2020. We have made a significant amount of progress. We're very, very pleased with the progress that we've made from a manufacturing perspective, both capacity, analytical development, process development and yield.
So we're very pleased at the progress we've made. We still have more to do. So certainly, as I said before, there is a risk to this, it's more of a temporal risk than a substantive risk we believe. But as it stands right now, we've made great progress. The team has done a brilliant job of getting ourselves prepared.
All of this relates both to the talent that we have built at Sarepta and along with our partners Brammer and Paragon, but also the fact that we were willing to make a significant investment in and bet on gene therapy manufacturing sometime ago. So we're very pleased where we are. The temporal risks are always the risks with this because there's still more engineering work to do and more lots to run, but we're very pleased with where we are so far.
Your next question comes from the line of Gena Wang with Barclays. You may ask your question.
Hi, thank you for taking my questions and congrats on the deal and happy holidays for everyone. This is David for Gena. I have a couple of questions here. So the first question is, did Roche ask for U. S.
Rights? And do you have any thoughts for future partnership for the U. S. Rights? And my second question is regarding the royalty rates.
Can you just provide some more color on the anticipated mid teen royalty rates? Is it tier royalty rate or is it flat? Thank you very much.
Okay. I'll answer both those questions. So first of all, we started this process and we were very clear in the process regarding the scope. The scope of this potential deal at the time we started was for one product SRP-nine thousand and one, hyper dystrophin and for ex U. S.
Rights. We were very, very clear from the beginning that that was the scope of our discussions and that we did not want people to take the opportunity to try to extend that scope either to other assets or to back into the U. S. Or to come back with something else. So this we were very, very directed about the scope that we wanted.
Certainly, you can envision that and I'm not suggesting this was Roche, but there certainly were those who would have suggested that we look at things like U. S. We had no interest in that. So the scope of this process was well defined by us at inception. On the royalty rates, as I said, the royalty rates are anticipated to be a mid tier a mid teen royalty rate.
They are not in the traditional sense tiered, meaning if we have mid teen royalties, it will be mid teen from sale 1. They don't, for instance, start low and accelerate the high as many royalties do. The royalty itself is based in part on based largely on the cost of goods that we'll ultimately achieve in manufacturing. The base case assumption for us is mid teens. One could envision a scenario in which we could do better than mid teens and certainly aspirationally, I think our tech ops colleagues would argue that they're looking for that.
But I would for planning purposes assume that the base case is a mid teens royalty from $1 ex U. S.
Just as a reminder, we're only answering one question, so everyone has an opportunity to ask the question.
Your next question comes from the line of Whitney Ujjain with Guggenheim. You may ask your question.
Hey, Greg. I'll add my congrats for exciting news. Excuse me. Doug, you highlighted your $2,500,000,000 in cash post this deal. So just curious if that changes your strategy on the BD front either as it relates to additional assets or programs or even technologies that you might want access to as you advance the pipeline?
Thanks.
On the face of it, we've got an ambitious strategy as it is. We've already built for ourselves what I believe to be the most significant gene therapy pipeline that currently exists. We built a gene therapy center of excellence that can develop new constructs for us. We've already had the constructs built. We already have one that we've announced, which is Emery Dreyfus this year.
We're building an enormous amount of manufacturing capacity. Our goal is to build among the most significant manufacturing capacity and manufacturing expertise in gene therapy in all of the biopharmaceutical industry. So I would say from a planning perspective, this I wouldn't we're not going to use this as an opportunity to even further expand our ambition beyond where we are today, we do business development. We have been doing business development over time. We're going to be thoughtful going forward, both about finding opportunities if we think that they make economic sense and are compelling to bolster our pipeline.
We'll continue to look for things like that. We've been looking as well to providing additional tools for our gene therapy center of excellence, so that we can ensure that we can continue to advance with gene therapy. As you may have seen very recently, we did the STRIDE Bio transaction, which in addition to potentially 8 constructs, also gave us access to what we're really excited about, which is STRIDES innovative capsid technology. So we'll continue there, but I wouldn't assume that this is going to greatly change
our approach.
We've got a lot to do. One of the things we're trying to do in this transaction is ensure that we have focus and that we can execute on our current plans.
Your next question comes from the line of Danielle Brill with Piper Jaffray. You may ask your question. Hi, everyone. This is Nirav Shladd on for Danielle Brill. Congrats on the deal.
I just have one question. With this deal in mind, looking forward into the limb girdle program, would you be
able to give us some color
on how you would think about potentially partnering ex U. S. For Limb Girdle and if it would be just there to E or all their programs together, just some color on that?
Yes. So as it stands right now, we're not entertaining any partnerships ex U. S. For any of our other gene therapy constructs, including but not limited to limb girdle. This you should see this opportunity that stands here today as relating to this moment in time, relating to an opportunity to work with an extraordinary biopharmaceutical company that is global in nature and that provides us with the kind of focus and the resources to ensure or certainly hopefully increase the probability of being successful in our broad mission of being a genetic medicine powerhouse in rare disease.
I wouldn't assume based on this that we would definitively do an additional ex U. S. License for another construct. That's something we would have to consider at the time. As it stands right now, it's not something that we're entertaining at all.
And we were clear during the process that we were only considering right now SRP-nine thousand and one.
Your next question comes from the line of Joseph Schwartz with SVB Leerink. You may ask your question.
Great. Thanks very much and congrats as well. I was wondering if Roche specifically evaluated your ability to transition manufacturing to a suspension based system? We did that the answer is no, because it's not what we're looking at right now. As you may know, we've what we've transferred from Hyperstax to iCellis, we've built capacity both at Brammer and very significantly at Paragon.
We've made enormous strides in the process development work there and analytical development there as well as the just capacity engagement of capacity. We've got a great relationship and supply agreement with the plasma material maker of Aldebaran. So that's the way we're tracking right now. I will say we also have we do have intellectual expertise and we do have an exercise going on in our facility in Burlington, which is one of the areas where we're focused significantly on process development and the like to look at suspension. But for micro dystrophin that's not what we're focused on right now.
Your next question comes from the line of Salveen Richter with Goldman Sachs. You may ask your question.
Great. Thanks for taking the question. If you could just real quickly just walk us through your non risk adjusted sales payments from the deal around $10,000,000,000 I know you highlighted around the few steps that
are going to compensate,
but just a little more clarity on how you're getting to the 10,000,000,000
dollars Well, I would just take it's simple to take $1,150,000,000 $1,700,000,000 dollars at 100 of 1,000,000 of dollars of cost sharing and then the remainder would of course be ex U. S. Royalties on in the mid teens range. It's a number that one gets to even fairly modest penetration ex U. S.
Your next question comes from the line of Brian Abraham with RBC. You may ask your question. Hey, guys. Thanks for taking
my question and congratulations on the deal. I was wondering if you could give us a general sense of the degree of OpEx that's allocated currently to the micro dystrophin development program? And I guess I'm just trying to get a sense for how having Roche take on half these development costs might impact the future OpEx trajectory? Thanks.
We haven't given a projection for that for 2020. We'll reflect on that and perhaps provide some additional color on that early next year. But it is significant. I mean, I will say, this is a very significant set of programs for micro dystrophin from a global perspective. Remember, in addition to what we're doing right now, which is what we call the Study 102 study, we're doing a significant collection of studies, all of which I use the phrase Study 3, but truthfully it's a series of other studies that gets at ambulatory patients, non ambulatory patients, younger kids, some additional kids in between those ranges.
So it is multinational, significantly multicenter, much larger than 102. So there is a significant amount of resource required for clinical development program going forward. And it will be an enormous benefit to us to have not only the guidance and expertise of Roche and their team, as we think about global development, but also this cost sharing and the fact they are going to pay for half of all of the global development work and as it relates to any specific territory, specific work, they're going to pay 100% of that. So it will be significant, but I don't have an exact number on that right now.
Your next question comes from the line of Tim Schump with BTIG. You may ask your question.
Hey, Doug. Congrats on the deal. Just to clarify, I guess, if Roche is going to pay for half of the R and D costs going forward with this program, are they also going to be taking on half the cost for Study 3 as well?
Yes. The answer is yes to that.
And maybe if I can just slip one in. You mentioned there was a competitive bidding process. Obviously, a lot of large cap pharma companies looking for gene therapy programs. I mean, there any sort of number you can throw out in terms of how many companies had interest in the ex U. S.
Rights for the DMD gene therapy program?
All I'll say broadly is there was significant amount of interest. It was a very competitive process. It was competitive in 2 ways, competitive amongst various large multinational organizations. And in a very real sense, it was competitive because we had a standalone thesis that had to be overcome as well. And it became obvious by the end that Roche was the preferred partner for host of reasons.
And we're obviously thrilled with the economics, but far more the economics we're thrilled with the partner and what this can mean for Sarepta and what it can mean for patients, because this really will trend. If we're successful and if Roche is successful with us, they are going to be countless patients around the world that would otherwise have been robbed of their movement and then ultimately of their life by Duchenne muscular dystrophy, which could be different if this therapy meets the profile that we're hoping that it does. And that's and that this partnership is going to empower that.
Your next question comes from the line of Vincent Chen with Bernstein. Please ask your question.
Thank you very much for taking the question
and congrats on the data. Following up quickly on earlier questions on the economics of manufacturing, when you talk about the mid teens, what portion of this is covering the cost of manufacturing? And if cost has been lower or higher than you currently expect, what happens to the royalty rate? And how should we think about Sarepta's share of the economic value of the U. S.
Opportunity going forward?
I apologize. You kind of broke up. I think what you're asking was sort of how does the royalty flex on net sales? Is that what I understand?
Yes. Sorry. I was asking
when you think about
the mid teens royalty, what part of this goes towards COGS versus other? And then if COGS comes in lower or higher, what happens to the total relapsed rate?
Okay, great. So first of all, understand, we sell we will sell the therapy to Roche at the standard cost of goods, okay? And then on top of that, they will pay us a mid teens royalty on net sales. So none of that mid teens royalty goes to cost recovery from our cost of goods. We will be fully reimbursed for all of our standard cost of goods.
And then on top of that, we look at this mid teens royalty. As I said, the reason we say, we anticipate mid teens royalty is that there is a schedule that would describe the royalty rates based on various cost of goods assumptions. And so again, if we were in the optimistic case, we'll be higher than mid teens. But I just think for planning purposes, we ought to take the base case assumption and that will be mid teens. But it will not be there wouldn't be no part of that mid teens that is any part of cost of goods recovery that we are paid 100% of our standard cost of goods before the royalty is imposed.
Your next question comes from the line of Liisa Bayko with JMP Securities. You may ask your question. Just in terms of as you think about commercialization, how much influence will you have on issues like global pricing and some various access questions? Thanks.
They will so I was broadly speaking that we have a number of joint committees. I said, for instance, joint development committee, we'll obviously have a joint commercialization committee and the likes. So we'll have some influence on them. Ultimately, however, just as we said that we'll be responsible for development, Roche will be responsible for commercialization, including but not limited to things like access and reimbursement pricing and the like. So they will have control over those issues, as Gilmore rightly does ex U.
S, of course, not related to the U. S. So we were obviously this relationship only deals with ex U. S. Not U.
S. So the end Roche is going to be responsible for commercialization, for the cadence of launches, for pricing, access reimbursement, HCA process, regulatory process itself. Obviously, while we have we'll have visibility into that and the joint commercialization committee regarding that, we're very comfortable with that because of Roche and because we are very excited to be with a company with prowess frankly of Roche. And so I think we'll benefit from the fact that they're going to drive that.
Your next question comes from the line of Tim Lugo with William Blair. You may ask your question.
Hey, this is Lachlan
on for Tim. Thanks for taking the questions. So you mentioned that Roche will be responsible for any country specific or region specific development plans if they're required. Based on your discussions thus far, do you have are there any territories where you expect there will be additional requirements beyond what you currently have ongoing in the U. S?
We have not flagged any right now. So that will require us to sit and talk more about exactly what regions Roche is considering, whether there is some unique requirement in the region that we haven't anticipated. The global plan, the plan that we've built based on informed by our views in the U. S, informed by our views and communication and discussion with the European regulatory authorities and informed by discussions we've had, for instance, with Japanese regulatory authorities. That global plan, we're going to share those costs fifty-fifty.
We haven't identified any country specific additional requirements, but if something it came up in the future, then they would pay 100%. But we don't have a particular requirement that we've together flagged right now.
Your next question comes from the line of Christopher Marai with Nomura. You may ask your question.
Hey, good morning. Thank you for the question and congratulations. Could you maybe comment on Roche's diligence on other DMD assets at Sarepta? It sounds obviously like it was a Duchenne focused collaboration. For instance, have they done any work on the PPMO or PMO?
And then maybe quickly on the casimersen NDA submission, where is that at? Thank you.
All right. So broadly speaking, I will answer that, Chassis. So this was focused on SRP-nine thousand and one. We certainly provided information in a broad sense about our PMO and PPMOs and gave background information and other information to Roche and the Roche team. But if you really want to talk about the diligence that was done that supported this, it really was focused on SRP-nine thousand and one.
It was focused on the development plan itself. It really pressure tested the development plan. It was really significantly focused, of course, our manufacturing and manufacturing progress to date, tech ops and of course the commercial opportunity itself. So really I would say, you're looking at the diligence you should assume that 99% or more was focused on SRP-nine thousand and one. And then on Cazoneercyn, again, I think I've told other people, we had I had demanded of the team, in fairness it was me, that they not submit for casimersen until we had the ruling on Golodirsen.
We've got that means that the casimersen original filing, which would have been submitted shortly after the August 19 approval for Golodirsen is now still, it has to be updated. The team is working on that right now. And certainly, we intend to submit in 2020 for casimersen. And we believe we have a very clear pathway for casimersen subject to the review by the division
Presenters, I'm showing no further questions at this time. I would now like to hand the conference over back to you, Guy.
Thank you very much. Let me close by simply saying that, of course, Sarepta has much to do in 2020. We have an enormous amount of progress to make and we have some very seminal moments for this organization in 2020, both across our gene therapy and SRP-nine thousand and one, our RNA platform as well, both with golodirsen and casimersen, but also our PPMO limb girdles and of course our micro dystrophin program. So we've got a lot to do. 2020 will be an extraordinarily important year for us.
This relationship with Roche is going to play a big part of what we're doing, because we have as we track into 2020, as I said before, we will have an enormous amount of resources to ensure that our ambition can be met from a resource perspective. As I said, we will have once this deal closes as much as $2,500,000,000 available to us to drive our performance. And of course, we will be as I hope everyone would recognize, we will be good stewards with the resources in front of us. And that's why we don't intend to change our ambition. We're ambitious enough as it stands.
And we have the focus to get things done next year. So we're really excited about this. We're really excited about our partnership with Roche and the validating nature of that. And with all of that, I look forward to talking to all of you in 2020. In the interim, I would ask everyone to have a restful and happy holiday period.
Thank you.
Ladies and gentlemen, this concludes today's conference. Thank you for your participation and happy holidays everyone. You may all disconnect.