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Regenxbio Inc. (NASDAQ:RGNX)
Q2 2020 Earnings Call
Aug 6, 2020, 4:30 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good afternoon and welcome to the REGENXBIO Second Quarter 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will be given at that time. As a reminder, this conference call is being recorded.

I would now like to turn the call over to Mr. Patrick Christmas, Chief Legal Officer for REGENXBIO. You may begin.

Patrick J. Christmas II, J.D. -- Chief Legal Officer

Good afternoon and thank you for joining us today. With us are Ken Mills, REGENXBIO's President and Chief Executive Officer; Dr. Steve Pakola, our Chief Medical Officer; and Vit Vasista, our Chief Financial Officer. Earlier this afternoon, REGENXBIO released financial and operating results for the second quarter ended June 30, 2020. The press release reporting our financial results is available on our website at www.regenxbio.com.

Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory, product development and other business plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend and other words of similar meaning.

Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2019, and comparable sections of REGENXBIO's quarterly report on Form 10-Q and other filings, which are on file with the Securities and Exchange Commission and available on the SEC's website.

Any information we provide on this conference call is provided only as of the date of this call, August 6, 2020. And we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially.

I would now like to turn the call over to Ken Mills, President and Chief Executive Officer of REGENXBIO. Ken?

Ken Mills -- President and Chief Executive Officer

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us for a second time this week. Some of us today are updating live from our offices in Rockville, Maryland, and we hope that everyone is safe and well. On today's conference call we will provide a few updates on recent progress, advancing our pipeline of gene therapies, and Vit will provide an update on financial results for the second quarter of 2020. Steve is here as well for updates and questions about our clinical programs, including our latest RGX-314 data.

We're now a little more than halfway through the year, of course, and I'm pleased to report that 2020 has been very productive for REGENXBIO, despite the challenging backdrop of the COVID-19 pandemic. We've made important strides in our clinical development programs and key decisions that have allowed us to broaden our pipeline while evaluating and prioritizing promising gene therapy candidates. As I mentioned earlier this week, we held a conference call that was led by Steve and with several of our leading retinal specialists to share an update on the RGX-314 program for the treatment of wet AMD. So I invite all of you to listen to that call if you haven't already done so.

The data from our Phase 1/2a study of RGX-314 has provided us with evidence of a compelling clinical profile for the potential treatment of wet AMD in a broad range of patients. Patients from this study in Cohorts 3, 4, and 5 have demonstrated stable to improved visual acuity and retinal thickness, as well as meaningful reduction in anti-VEGF injection burden out to one year. And as we reported in April, we also have data from Cohort three out to two years, which gives us confidence in the long-term durability of this gene therapy. We will use the data and our learnings from this first-in-human study to inform the design of our pivotal program, which we expect to initiate in the second half of this year. We look forward to providing further details in the months to come.

Also announced earlier this week, we had advanced our Phase 2 trial for the suprachoroidal delivery of RGX-314 using the SCS Microinjector for the treatment of wet AMD. We planted those in the first patient in this randomized controlled study known as AAVIATE in the coming weeks and expect interim data from the first patient cohort by the end of 2020. We also plan to use the suprachoroidal delivery route for the treatment of diabetic retinopathy, which is a leading cause of vision loss, affecting approximately 8 million people in the United States. We expect to initiate a Phase 2 trial in the second half of 2020, with interim data expected in 2021.

In July, we reported an update on the progress of clinical programs for rare genetic diseases, and Steve will now update on the current status and expected milestones related to these programs. Steve?

Steve Pakola, M.D -- Chief Medical Officer

Thanks, Ken. As previously announced, we have completed dosing of three patients in the second cohort of our Phase 1/2 study of RGX-121 for the treatment of MPS II. These patients were dosed intracisternally and RGX-121 was reported to be well tolerated in patients across two dose levels with no drug related serious adverse events. We expect to provide additional data from both cohorts in the study, as well as a program update later this year. We also expect to provide a program update for our Phase 1/2 clinical trial of RGX-111 for the treatment of MPS I by the end of 2020.

Last month, we reported data from a patient with severe MPS I who was dosed intracisternally under a single patient investigator initiated IND at CHOC Children's. At week 12, after administration of RGX-111, an increase in IDUA enzyme activity was detected, as well as a sustained decrease in heparan sulfate in the cerebrospinal fluid. In addition, neurocognitive evaluations indicated that the patient continued to acquire cognitive developmental skills at a normal rate at week 32. We are very encouraged by the clinical and biochemical indicators for this study.

We are committed to continuously evaluating our portfolio of single administration gene therapy candidates to drive innovation for patients, and further leveraging the expertise of our team. We have been working for some time on our RGX-181 program for the treatment of the CNS manifestations of CLN2 disease, also known as Batten disease. As we work closely with the community and gotten to know many of the patients and families, we were struck by the tremendous unmet need for therapies to address ocular manifestations of the disease.

Building upon our understanding of gene therapy in the eye, we are bringing forward a new program, RGX-381 for the treatment of ocular manifestations of CLN2. RGX-381 is designed to use the AAV9 vector to deliver tripeptidyl peptidase 1 or TPP1 gene directly to the retina. We believe one-time administration of RGX-381 could provide a durable source of TPP1 activity in the retina thereby potentially preventing visual decline. We plan to submit an IND for this program in the second half of 2020 and initiate enrollment in the first half of 2021. We have also opened two non-interventional natural history studies to better understand the ocular manifestations of CLN2 disease.

This expansion reflects our commitment to the CLN2 patient community, and we'll build upon the collaborations and relationships established through the RGX-181 program, which also continues to move forward. We expect to file an IND for RGX-181 by the end of this year and initiate enrollment in a Phase 1/2 trial in the first half of 2021.

With that, I'll turn it back over to Ken.

Ken Mills -- President and Chief Executive Officer

Thank you, Steve. These are encouraging updates. Now, additionally, today we're announcing that we've made a difficult decision to discontinue internal development of RGX-501 for the treatment of Homozygous Familial Hypercholesterolemia or HoFH. RGX-501 was one of our first clinical stage gene therapy programs and we're grateful for the support of the HoFH patient community over the years. However, we've seen the landscape of treatment options shift dramatically since we began the program with the emergence of several new therapies that lower LDL cholesterol in these populations. The target product profile has changed, so while our gene therapy approach supported signs of biological activity at the second dose level, we acknowledge that further pharmacology work is needed. We will look for opportunities to support continued advancement of this program through business development, and certainly focus on ways to share our clinical trial data that benefits patients and foster scientific discovery in cooperation with the investigators in the future.

And just to wrap up, our plans through the second half of 2020 also include progress on our potential AAV8 based treatment for HAE, where our preclinical data has demonstrated biological activity in an animal model of inflammatory edema and inhibition of kallikrein comparable to other anti-kallikrein treatments and we have plans to announce more details around the treatment for neuromuscular disorder using AAV8.

With that, I will now like to turn the call over to Vit for review of the financials.

Vit Vasista -- Chief Financial Officer

Thank you, Ken. REGENXBIO ended the quarter on June 30, 2020 with cash, cash equivalents and marketable securities totaling $339.2 million compared to $400 million as of December 31, 2019. The decrease was primarily attributable to net cash used in operating activities of $57.1 million. Revenues were $16.6 million for the three months ended June 30, 2020 compared to $7.9 million for the same period in 2019. The increase was primarily attributable to $11 million increase in royalty revenue recognized on net sales of Zolgensma in the second quarter of 2020. Total net sales of Zolgensma since launch in May, 2019 are greater than $707 million [Phonetic] and REGENXBIO is eligible to receive a milestone payment of $80 million upon the achievement of $1 billion in cumulative net sales of Zolgensma.

Research and development expenses were $38.1 million for the three months ended June 30, 2020 compared to $29.5 million for the same period in 2019. The increase was primarily attributable to personnel related costs as a result of increased headcount, expenses associated with conducting clinical trials for our lead product candidates, laboratory and facilities costs, and externally sourced services for preclinical, regulatory and manufacturing-related activities.

General and administrative expenses were $15.6 million for the three months ended June 30, 2020, compared to $13.4 million for the same period in 2019. The increase was primarily attributable to personnel related costs as a result of increased head count and professional fees for advisory and other services. Net loss was $33.8 million or $0.91 basic and diluted net loss per share for the three months ended June 30, 2020 compared to a net loss of $1.5 million or $0.04 basic and diluted net loss per share for the same period in 2019.

As of June 30, we had approximately 37.3 million common shares outstanding. Based on our current operating plan, REGENXBIO bio expects its balance in cash, cash equivalents and marketable securities to fund the completion of its internal manufacturing capabilities and clinical advancements of its product candidates into 2022.

With that, I will turn the call back to Obi-Ken Kenobi, may the force be with you.

Ken Mills -- President and Chief Executive Officer

Okay, Vit. As we've mentioned before, REGENXBIO has an extensive footprint in gene therapy, aside from our own internal pipeline. And our NAV Vectors are the basis of many partnered product candidates, including one marketed product Novartis’ Zolgensma and several others in late stage clinical development. The extraordinary milestones and achievements from our partners, as well as the progress in our internal pipeline provide additional validation to our proprietary NAV technology platform and further demonstrate a transformational impact that can come from a one-time administration of gene therapy.

Now as Vit mentioned, we continue to track the positive progress on Zolgensma which uses the NAV AAV9 vector. On its recent earnings call Novartis cited, strong global momentum for the therapy, despite COVID-19, with newborn screening continuing to progress in the United States and additional growth driven by geographic expansion outside of the U.S. We're also pleased to see the positive regulatory developments in Europe this quarter with the conditional approval of the intravenous formulation and the robust uptake in Japan, since its first month of launch in that market. I continue to be encouraged by the great potential for this therapy.

It's truly exciting to be experiencing the momentum that we have at the company today. And after more than a decade of steadfast effort and focus our business includes a broad and expanding pipeline of gene therapies. We're on the cusp of initiating our first pivotal trial for RGX-314, a remarkable milestone. We look forward to making further strides towards improving lives, through the curative potential of single-administration gene therapies, and remain dedicated to this mission. I'm proud of and grateful to our talented and dedicated employees who drive our business forward, despite the challenging backdrop of the COVID-19 pandemic.

And with that, we'll end this portion of the call and we'd like to open up for questions. Operator?

Questions and Answers:

Operator

Thank you. [Operator Instructions] And our first question comes from Gena Wang with Barclays. Please go ahead.

Gena Wang -- Barclays -- Analyst

Thank you for taking my questions. I have two questions. First one is regarding the 314, suprachoroidal Phase 2 trial design. First, have you thought about using two doses for -- two injections for the first dose so that it can cover broader space on the back of the eye? And also how's the retreatment criteria compared to the subretinal procedure? And then my second question is regarding the CLN2 programs. Can you give us a little bit more color regarding the symptom onset for the ocular versus CMS? And how much overlap this could be for the patients? And how would you decide the patient selection?

Ken Mills -- President and Chief Executive Officer

Hi, Gena. This is Ken. Those are great questions. I'm going to -- yes, Steve, you're on. Thank you.

Steve Pakola, M.D -- Chief Medical Officer

Okay. All right, great. You see the realities of being in different spots in times of COVID. So thanks, Gena, for those good questions. Yes. So suprachoroidal, the existing experience with suprachoroidal delivery using this specific device SCS Microinjector from Clearside is with 100 microliters. And that's where our first cohort is going to be looking at the high dose number of GCs per eye within 100 microliters. So that highest dose that we were able to get to in the subretinal study.

So you raised a good point given the preclinical data that suggests that with suprachoroidal delivery, even with one injection you get about a half of the globe or the retinal surface covered with a 100 microliter suprachoroidal injection. So that could have been a possibility to consider looking at, for example, 50 microliters in two separate injections or a 100 microliters. We decided right off the bat to take it step by step and first start with a 100 microliters single injection, but going right up to that 2.5E11 GC dose there. But we are going to in the second cohort use that same concentration and give a second dose of a 100 microliters, a 180 degrees on the other side of the eye to do exactly what you're raising, have the potential to cover the entire retinal surface.

In early days in terms of suprachoroidal we thought that was the best way to get the earliest and quickest learnings, while also leveraging how much has been learned from the over 1,000 eyes that have been dosed with the Clearside device using a 100 microliters single injection.

Your second question segwayed into our newest ocular program beyond RGX-314 subretinal and now suprachoroidal the RGX-381 for a CLN2 disease. And it's a great question in terms of when do you see the deterioration in the systemic and the neurologic side of the disease relative to the ocular side of the disease. And it's really that time course that interestingly is what allowed us to realize how much of a big unmet need exists, where given ERT treatment, which is addressing systemic complications for these patients. They're living longer, but now the really big unmet need that we're hearing from families repeatedly is that the patients are now going blind. So this has become a very clear area, where now, especially, for us with our experience and also our vectors, where we have real proof of concept and validation for retinal delivery, it makes sense for us to go in this direction.

If you think of the time course, it is a little later than the time course where you see the non-ocular manifestations initially evolve, but it is the type of thing that there's enough natural history out there in terms of how even the imaging evolution involves to help us decide what's the right age groups to enroll, even in early development to have patients where we're going to be likely to evaluate and have power to evaluate whether a gene therapy one-time treatment is actually having an impact on the natural course of the disease in terms of the ocular manifestations. And we're going to gather more information by starting as we announced two non-interventional natural history studies of CLN2 patients.

Gena Wang -- Barclays -- Analyst

Okay. Just like -- what about the rescue injection criteria for suprachoroidal comparison to the subretinal?

Steve Pakola, M.D -- Chief Medical Officer

Yes. Yes, thank you. I accidentally skipped over that one. So in our first-in-human study as we've often talked about, we had a very low bar for retreatment, so that stack the deck against us in terms of which patients and how many reinjections might occur. Now that we have actual proof of concept and clear evidence of beneficial effect across a range of doses, albeit with subretinal delivery, we feel we're in a very solid position to start moving that bar higher to a more traditional bar that you can see in some of the other later stage studies that have had retreatment criteria. So that's a good point. We're already right from the start with our suprachoroidal Phase 1/2 studies per SCS wet AMD, we'll be using kind of a more traditional retreatment criteria.

Ken Mills -- President and Chief Executive Officer

Thanks, Gena. Next question, operator. Hello?

Operator

Our next question comes from Geoff Meacham with Bank of America. Please go ahead.

Alec Stranahan -- Bank of America -- Analyst

Hey guys, this is Alec on for Jeff. Thanks for taking our questions. First on 501, could you help us understand a bit more around the decision to discontinue the program? I guess the evolution of the competitive landscape, but did the transaminases elevation or something on the efficacy side also inform your decision? And then on the CLN2 programs, could you provide some more color on how the preclinical work for 181 helped inform development of 381? And if you could also speak to the nuances of TPP1 delivery to the retina versus intracisternally in the case of 181, that would be great. Thanks.

Ken Mills -- President and Chief Executive Officer

Sure, Alec. Steve, maybe I'll start off here and you can jump in as needed.

Steve Pakola, M.D -- Chief Medical Officer

Right.

Ken Mills -- President and Chief Executive Officer

With respect to 501, we -- certainly as I mentioned, expect to bring when the investigators have had a chance to work through all of the data with us, bring forward more of the findings with respect to the science and the status of the patients that we've enrolled to date. I really think that with that understanding, what we saw Alec was what I described, which is certainly the landscape has changed.

There have been treatments coming forward in development that are different than when we initiated this trial several years ago. Some of which are having significant lipid lowering effects and cholesterol lowering effects, clinically it has changed and raised the bar for our target product profile and as well as the risk benefit. And we definitely saw from dose level 2 with some of the early examples while we felt we could treat the manifestations of these transaminases elevations with prophylactic steroid protocol. I think we've acknowledged that steroid protocols in these patients are also things that elevate the cholesterol levels naturally, even in healthy patients. And so it's something that pharmacologically we wrestled with. And I think with respect to that data coming forward, we'll look to work with our investigators at Penn to bring additional data when we have an ability to do so with them.

On the 181 and 381 conversation, I think as Steve alluded to with respect to the answer to Gena's question, this really -- it wasn't so much the preclinical work that was going on around 181 as it was the engagement with the community in CLN2 Batten disease. Hearing about the approval of enzyme replacement therapy treatment for the CNS manifestations and how it was being absorbed and received in the market, evaluating the need for gene therapy brought up a real, additional unmet need in the ocular manifestations of the disease. And as Steve alluded to, there may be a bit more of a late onset here, but the prevalent population of patients currently on even existing therapy is there in waiting for some solution that's not met by existing cerebroventricular enzyme replacement therapy. And we wouldn't expect would be met by an intracisternal approach either.

So the idea was to leverage our understanding of subretinal delivery to call on experiences like our preclinical investigators and others have had with programs like Luxturna. And deliver the gene that encodes TPP1 directly to the site of action, where -- in the retinal cells, where the disease pathology is occurring. And so we view this on a going forward basis as probably something that's going to become not unusual for gene therapy with different routes of administration to treat these multi-system diseases. And in this case, we think a intracisternal approach for the CNS manifestations, which are quite severe as well as a intraocular approach to address the vision loss is going to be important for basically addressing all of the unmet need that can be addressed in CLN2.

Alec Stranahan -- Bank of America -- Analyst

Perfect. Thank you very much and congrats on the progress.

Ken Mills -- President and Chief Executive Officer

Sure. Thanks again, Alec, for dialing in. Operator, next question.

Operator

Our next question comes from Mani Foroohar with SVB. Please go ahead.

Mani Foroohar -- SVB -- Analyst

Hey guys, I have a couple of quick ones, the first one for Vit. How do you guys think like strategically around the Previal stake and any other possible equity stakes you could have as a result of other licensing transactions? Do you see those as strategic assets, purely financial assets to be monetized whenever the valuation is appropriate? Like, how do you view them? I propose being control assets in the terms of equity, you're just another pool of cash.

And then one for Kenobi. When you think about reviewing your gene therapy pipeline and changing standards and expectations, how high is the bar? Or what is the bar to consider deprioritizing or going back and looking for a different asset for an indication going through different life cycle management strategy for later stage asset, as opposed to something that's still in the early stages of HoFH was?

Ken Mills -- President and Chief Executive Officer

Thanks. Mani. Glad you got bumped up in the queue today and those are good questions. Vit, why don't you take the first one?

Vit Vasista -- Chief Financial Officer

Yes. Thanks, Mani, for the question. We view the assets that we have in licensing companies when we do take equity as just alternative pools of capital and a way to continue funding the company. The general wave performed incredibly well in terms of if you normalize them to what we would get on a cash transaction licensing basis. So that's kind of how we use our view, equity stakes in currently public companies.

Ken Mills -- President and Chief Executive Officer

And I think the question about managing the pipeline, look, I think for us, by the time something gets to a decision about later stages of development, we not only have established a strong foundation on the basis of proof of concept from clinical data, but we've also in parallel continued to do deep digging into kind of the pre-commercial and commercial readiness and an additional need. And that maturation process is certainly a big contributor to making decisions to advance things from proof of concept in-clinic stage to late stages of development, including pivotal, like with our subretinal RGX-314 program, which as I mentioned, this is the first example of us advancing something forward.

And I think that will be applied to programs on a going forward basis. It's something that we obviously have awareness of upstream when we're identifying programs for the portfolio, but obviously the contributions of what happens with respect to the proof of concept outcomes in the clinic, as well as the commercial landscape, our major and important factors that go into are continuing to advance those things through the funnel.

Thanks, Mani. Operator, next question.

Operator

Our next question comes from Gbola Amusa with Chardan. Please go ahead.

Sam Lee -- Chardan -- Analyst

Hi, this is Sam on behalf of Gbola. Thanks for taking our questions. I have a question and a follow-up. First with regard to epidemiology, if you had to represent the markets RGX-181 and RGX-381 targets in a Venn diagram. Is the circle for RGX-381 sitting completely inside that for RGX-181 or would the Venn diagram represent two intersecting circles where each product would have utility in distinct populations with some overlap in the middle?

Vit Vasista -- Chief Financial Officer

I think -- thanks for the question. The assessment has been that the vast majority of patients that present with CLN2 disease, genetically and phenotypically will emerge with manifestations that are both central nervous system, neurodegenerative, etc., as well as ocular and vision loss. As Steve alluded to, there can be a frame shift sometimes in that presentation, but I think in terms of how you're thinking about a Venn diagram, I'm a math guy so I like that analogy, there's significant overlap, but for the component of time.

So maybe the incident population is going to be coming in with obviously CNS symptoms first as I alluded to, but eventually we see prevalence in all patients, especially as gene therapy or existing standard of care can start to address the CNS manifestations that vision loss becomes a substantial and severe impact as well.

Steve Pakola, M.D -- Chief Medical Officer

And I'd add, that helps quite a bit in our learnings and talking to the patient community where these patients are in the healthcare system and for lack of a treatment option for the blinding eye disease aspect that these patients are going to develop. There isn't anything to be done. So some of these are sent but with an actual treatment, there's a very feasible approach to having these patients seen in a timely fashion. So if a potential therapy comes forward, there could be a great penetration for these needy patients.

Ken Mills -- President and Chief Executive Officer

Thanks for the question. Operator? We're back to the queue.

Operator

Our next question comes from Matthew Harrison with Morgan Stanley. Please go ahead.

Connor McGuinness Meehan -- Morgan Stanley -- Analyst

Hi, all. Thanks for taking the question. This is Connor on for Matthew. Also just two from us, for MPS types I and II can you just talk about how you plan to engage with regulators? And I guess to what extent do you want to see the data develop before you have conversations regarding those programs? And then just quickly on RGX-381 what steps do you need to complete before submitting the IND? Thank you.

Ken Mills -- President and Chief Executive Officer

Yes, I think Steve, I'll take this I guess and feel free with additional color. Thanks, Connor. I think, appreciate the focus on the neurodegenerative and CNS aspects of the pipeline. We have chosen, and I think we've said this before MPS I and MPS II have been decisions for us that were as much driven by the impact that gene therapy can have, but also the understanding of the natural history in these diseases and we've pointed to the examples of, I think the consensus among experts that we've engaged with about the correlation between heparan sulfate as a biomarker in degenerative neurological conditions in severe cases of MPS II and MPS I as significant.

We obviously had the benefit of natural history occurring because just like Steve alluded to with recently -- more recently CLN2 patients, MPS I and MPS II patients have been part of a system of care, but also treatment for years now. Thanks to the early interventions developed around enzyme replacement therapy. And so gene therapies coming into that with an understanding of an improvement of a profile, addressing additional unmet needs, but also that more rich natural history then maybe a de novo disease and then because of that, I think conversations even from the very beginning of this program have been able to be informed by a lot of what's known and you get that even when you would talk with people within the agency or within the community. So I think that's important point of emphasis there.

With respect to RGX-381, our guidance here is that we are expecting to file an IND by the end of this year. So it's August and typically when we're talking about something on the order of months, it really is about at a stage where we feel like we've established a lead candidate based on proof of concept work and gone through opportunities to sort of assess the type of dose we're using.

We I guess obviously have decided and stated that we're using the AAV9 vector, which is something we were already familiar and comfortable with, with respect to the profile of RGX-181 in the CNS. So that formulation and that approach with that vector and that transgene product were things where we already had foundational evidence of obviously transduction and expression in different cell types. So we're really doing and steering this now to final work that would prepare us for clinical readiness and safety around the ocular administration.

And of course, I guess it would be remiss without adding. We're using the subretinal delivery approach. So we're also able to borrow from that clinical experience as well as approved product experience when we head into the clinic here. Steve, did I miss anything on that?

Steve Pakola, M.D -- Chief Medical Officer

Well, I'll just say as the Padawan learner here, nothing to add. Well done.

Ken Mills -- President and Chief Executive Officer

Good point. Thanks for extending the analogy.

Connor McGuinness Meehan -- Morgan Stanley -- Analyst

Understood. Thank you.

Ken Mills -- President and Chief Executive Officer

Operator? Yes. Thanks, Connor. Have a good one.

Operator

Our next question comes from Luca Issi with RBC Capital. Please go ahead.

Luca Issi -- RBC Capital -- Analyst

Terrific. Thanks for taking my question. Luca Issi from RBC. Maybe the first on RGX-314, how are you thinking about a pivotal trial design here? Should we assume that the study is conceptually similar to the Port Delivery System trial that Roche recently run? Or are there any differences from that trial that you'd like to highlight? And then maybe as a quick follow on to that question, how should we think about the costs of that trial? And then the second is, are there any updates on the IP dispute with Passage? Thank you.

Steve Pakola, M.D -- Chief Medical Officer

Okay. I can take the RGX-314 pivotal design question first and then pass-off on the latter part of the question. So thanks, Luca. Yes, so you raised the Port Delivery System and the Archway pivotal design. So that delivery approach is somewhat similar in that, it's certainly shooting for increasing durability, but it's also very different in terms of still being the paradigm of needing repeated injections. It's just that you can get it down to once every six months by being able to inject into -- in dwelling device.

So that aspect winds up being very important also from a design standpoint. So I'd say, they had to consider the aspect of definitely being reinjecting into the Port Delivery System every six months. So that could allow them to think differently about how they're looking at visits leading up to that reinjection. In our case, this is intended to be a onetime treatment option at least for a majority of patients and in the other patients decrease the frequency of injection.

So, but I'd say at least in a general sense, you can think of the same type of pivotal design considerations, we'd be thinking about. So whether, even with those differences aside in a wet AMD population, we know BCVA is the required pivotal endpoint. We know with standard of care, albeit frequent routine injections that in that setting it makes sense to look at a active comparator and considering non-inferiority. So a lot of this, Luca, is sounding like similarities.

Beyond that, also other similarities based on the patient population and of what type of duration of follow-up you might need in terms of safety and efficacy, and also the general size.

So it's at least a precedent out there for people to look at. We are aware of that, but we certainly rolled up our sleeves and looked at our particular target product profile, our particular results that we're seeing in how we're going to finalize the design of our pivotal study.

Ken Mills -- President and Chief Executive Officer

Yes. Thanks, Steve. That's the first part of the question or first question. Luca, the second on the IP dispute with Passage. No there's nothing to update with respect to that communication. We've notified them of obviously our concerns over their infringement of intellectual property and they've received that and they're aware of it and that's where that stands. I do expect that there will be updates on actions to be taken though with respect to our IP in general, and especially for companies that are in later stages of development and companies that are heading towards commercialization, Passage, of courses is I mean, still at a preclinical stage, we've notified them and others though -- of these infringing actions in our view.

And it's now time for us to transition to take action where the focus will be on those companies that are in later stages of development or heading towards commercialization. We think this is important. We've been saying this for a while about representing the interests of our shareholders with respect to this IP and so we'll be updating on this in the near future

Operator

Thank you. At this time, we have no further questions. Turning the call back to final remarks for Ken.

Ken Mills -- President and Chief Executive Officer

Okay. Well, thanks everyone. Thank you operator. And thanks everyone who joined us today. We appreciate the questions. We appreciate the interest. Appreciate those of you that spent two days with us reviewing data and reviewing progress. We're incredibly excited about the opportunity for REGENXBIO to deliver value for patients, to deliver on our mission and of course, for our partners, stakeholders, and shareholders. We hope that everyone stays safe, stays well and have a great night.

Operator

[Operator Closing Remarks]

Duration: 43 minutes

Call participants:

Patrick J. Christmas II, J.D. -- Chief Legal Officer

Ken Mills -- President and Chief Executive Officer

Steve Pakola, M.D -- Chief Medical Officer

Vit Vasista -- Chief Financial Officer

Gena Wang -- Barclays -- Analyst

Alec Stranahan -- Bank of America -- Analyst

Mani Foroohar -- SVB -- Analyst

Sam Lee -- Chardan -- Analyst

Connor McGuinness Meehan -- Morgan Stanley -- Analyst

Luca Issi -- RBC Capital -- Analyst

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