Earnings Call Transcript - RGNX Q2 2024

Operator, Operator

Welcome everyone to the Q2 2024 REGENXBIO's Earnings Conference Call. All lines have been muted to avoid background noise. After the speakers speak, we will have a question-and-answer session. At this point, I would like to hand the conference over to Patrick Christmas, Chief Legal Officer of REGENXBIO. Please proceed.

Patrick Christmas, Chief Legal Officer

Good afternoon and thank you for joining us today. Earlier this afternoon, REGENXBIO released financial and operating results for the second quarter ended June 30th, 2024. The press release 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 and product development 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 31st, 2023, and comparable risk factors sections of REGENXBIO's quarterly reports on Form 10-Q, 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 1st, 2024, 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 like to now turn the call over to Curran Simpson, President and CEO of REGENXBIO.

Curran Simpson, President and CEO

Thank you, Patrick. Good afternoon everyone and thank you for joining us. I'm pleased to be leading today's call. My first one is REGENXBIO's Chief Executive Officer. Today, we'll be sharing a number of exciting positive updates and discuss the momentum happening across our pipeline of differentiated AAV therapeutics. I'll begin with a recap of our business highlights as well as an update of our corporate goals and key milestones that we have achieved. Dr. Steve Pakola, our Chief Medical Officer, will provide an update on our clinical programs; and then Vit Vasista, our Chief Financial Officer, will provide an overview of financial results for the second quarter ended June 30th, 2024. At the end of the call, we'll open up the line for questions. It's been a productive first half of the year for REGENXBIO as we make significant progress advancing each of our programs toward pivotal stage clinical trials and future commercialization. Our priority programs are RGX-202 for the treatment of Duchenne; AbbVie RGX-314 program for the treatment of wet AMD and diabetic retinopathy or DR, being developed in collaboration with AbbVie; and RGX-121 for the treatment of MPS II or Hunter syndrome. Our lead programs, specifically 202 and 314 represent large commercial opportunities where our product candidates are differentiated from current standard-of-care, can be expedited via accelerated approval due to significant unmet need and support meaningful value generation soon and for the long term. Let me begin with RGX-202 which represents the next generation of microdystrophin gene therapies and is poised to potentially be the second AAV-based product to reach the market. There are a number of exciting developments for RGX-202. Steve will share more details about the positive data reported today, demonstrating consistent, robust microdystrophin expression across treated patients reflecting a broad range of ages, but I first want to highlight the differentiating factors that we believe will make RGX-202 a best-in-class product and the excellent progress we are making to both expedite its development and maximize its commercial potential. RGX-202 is a differentiated product candidate utilizing an advanced microdystrophin construct with potential for improved functional benefit as shown in our preclinical data. It is the only microdystrophin product that includes the C-terminal domain, a key region of the naturally occurring dystrophin gene, which has been shown in preclinical studies to protect the muscle from contraction-induced stress and improve the ability of the muscle to repair itself. As I mentioned, RGX-202 is demonstrating consistently high levels of microdystrophin expression across patients of all ages. But I want to note that it is in older ambulatory boys where we're seeing the highest levels of microdystrophin expression reported in older ambulatory patients, especially compared to other published data. RGX-202 has been well-tolerated and no serious adverse events have been reported, which is a significant element of the overall risk-benefit analysis for patients, caregivers, and regulatory agencies and a meaningful differentiator versus other Duchenne gene therapy trials. As I mentioned, our goal is to be the next approved gene therapy in Duchenne and we are taking all of the necessary steps towards this goal. We recently completed a successful end-of-Phase 2 meeting with the U.S. FDA and walked away from this meeting confident in our plans to file a Biologics License Application using microdystrophin as the primary endpoint for accelerated approval. The meeting also involved a discussion of our industry-leading NAVXpress suspension-based commercial-ready manufacturing process used in this trial. At our in-house manufacturing facility, we have the capacity and yields to produce 2,500 doses of RGX-202 per year. Given the differentiating characteristics of RGX-202 and the significant ongoing unmet need in the Duchenne community, plus our manufacturing expertise, we are well-positioned to advance this program towards commercialization. Turning to AbbVie RGX-314, our gene therapy being developed in chronic retinal diseases with our partner AbbVie, we have made several advancements across the suprachoroidal trials in diabetic retinopathy and wet AMD. First, with regard to the ALTITUDE trial of 314 for the treatment of DR using suprachoroidal delivery, we are accelerating plans for our end-of-Phase 2 meeting with the FDA. This meeting is now expected to take place in the fourth quarter of this year versus our initial guidance of the first quarter 2025. The new timeline supports the rapid acceleration towards pivotal trials with initiation expected in the first half of 2025. Importantly, REGENXBIO will be entitled to a $200 million milestone payment upon successful dosing of the first patient with AbbVie RGX-314 in DR, which again is anticipated in 2025. We are also excited to announce that working with our partners at AbbVie, we will be expanding the broad multi-indication global potential of 314 by initiating a new cohort in the ALTITUDE trial for patients with diabetic macular edema, DME; 314 is well-positioned to become the standard-of-care to treat and limit the progression of diabetic retinopathy. Broadening the ALTITUDE trial to include patients with DME further expands the global potential of 314. We have also made important progress on our 314 programs for wet AMD as well as in our RGX-121 program for MPS II as we approach potential approval and becoming the first gene therapy for Hunter syndrome. We remain on schedule to initiate a rolling BLA filing in the third quarter of 2024. Approval of the planned BLA could result in receipt of a priority review voucher in 2025. Overall, we are making excellent progress and have provided positive updates across all programs with a number of additional catalysts on track to be shared later this year. We remain excited about our progress as we continue on the strategic plan. We are accelerating the development of our pipeline and expanding their value for shareholders, while bringing potentially life-changing therapies to patients facing great unmet need. With that update, I'd like to now turn the call over to Steve for an update on our clinical programs. Steve?

Steve Pakola, Chief Medical Officer

Thank you, Curran. I will begin with RGX-202, a potential one-time gene therapy for treating Duchenne. We have reported new microdystrophin expression data from two new patients, aged 5.8 and 8.5 years, who received RGX-202 at dose level 2, which we are moving forward to the pivotal phase. The microdystrophin expression was measured at 77.2% and 46.5%, respectively, compared to the control after three months. As of July 8, 2024, RGX-202 remains well tolerated with no serious adverse events reported. All patients who reached the three-month trial assessments showed significant increases in RGX-202 microdystrophin expression and a decrease in serum creatinine kinase levels from baseline, indicating clinical improvement. We are very excited as today’s data enhances the overall evidence showing consistently high microdystrophin expression across all treated patients. Furthermore, early signs of strength improvement in motor function were noted through trial clinic assessments and home videos shared by caregivers. Based on the strength of our data, we announced in June the expansion of the AFFINITY DUCHENNE trial to include a new group of patients aged one to three years. This new cohort involves boys without any approved gene therapy products available and represents a significant segment of the untreated population of Duchenne boys. As we aim for a broad label for RGX-202, we will keep generating data in areas where limited or no data exists to further establish a distinguished product profile that can enhance RGX-202's market potential. Moving on to 314, being developed in partnership with AbbVie for the treatment of wet AMD, DR, and DME through both subretinal and suprachoroidal administration. I will start with 314 for DR, currently being evaluated in the Phase 2 ALTITUDE trial using in-office suprachoroidal delivery. As mentioned by Curran, we have expedited our end-of-Phase 2 meeting with the FDA alongside our partner, AbbVie, which we believe positions us well to start our first pivotal trial in DR. Today, we announced that we are enrolling a new cohort in the ALTITUDE trial to assess 314 in patients with center-involved diabetic macular edema or CIDME. DME is a serious complication of diabetic eye disease affecting over 30 million patients worldwide. We are also investigating 314 for wet AMD treatment through subretinal delivery in two ongoing pivotal trials, ATMOSPHERE and ASCENT, across the U.S., Europe, and Japan. These trials are progressing well, and our long-term follow-up data from the Phase 1/2 subretinal trial extending to four years have established a benchmark in clinical development for wet AMD gene therapy. We have fully enrolled the open-label Fellow Eye study assessing 314 in patients previously treated with the therapy in the other eye. This study aims to support a label that includes bilateral use, offering a valuable option for the considerable number of patients with wet AMD in both eyes. Additionally, regarding wet AMD, we announced today that 314 was well tolerated at dose level 3 in the AVA trial for wet AMD via in-office suprachoroidal delivery. In patients who received short-course prophylactic steroid eye drops, there were no drug-related serious adverse events or instances of intraocular inflammation, ophthalmitis, vasculitis, retinal artery occlusion, choroidal infusion, or any other significant issues. We are encouraged by the positive safety profile observed to date, and we plan to enroll a new cohort at dose level 4 as we evaluate different dose levels moving towards pivotal stages. We remain optimistic about the advancement of our 314 programs, particularly highlighting the safety profile seen in our suprachoroidal approaches. We are confident in our strategy to progress into new disease areas and dose levels due to this safety profile, especially with the short course of seven-week prophylactic steroid eye drops. Among over 130 patients treated in office, we are observing a unique safety profile for ocular gene therapies, indicating a meaningful potential treatment alternative for patients and physicians globally. Lastly, regarding RGX-121, developed for the treatment of MPS II or Hunter syndrome. In February at the WORLDSymposium, we shared that the CAMPSIITE pivotal trial achieved its primary endpoint with strong statistical significance. Patients treated with RGX-121 exhibited a decrease in cerebrospinal fluid levels of heparan sulfate, a crucial biomarker of brain disease activity, falling below the maximum attenuated disease levels. We are set to share new data from the CAMPSIITE trial in the latter half of this year. We believe RGX-121 is positioned well to be the first gene therapy and one-time treatment for Hunter syndrome. We successfully completed a pre-BLA meeting with the FDA that finalized details regarding our planned rolling BLA submission. The key points from the meeting included alignment on using CSF D26 as a critical biomarker and surrogate endpoint likely to predict clinical benefit for accelerated approval. We also agreed on CMC manufacturing requirements and the design of the confirmatory trial. In conclusion, we are making significant advancements with data updates and trial progression across all our pipeline programs. I want to express gratitude to the patients, families, clinicians, and patient advocacy representatives involved and supportive of all these trials. Now, I will turn the call over to Vit for an overview of our financial guidance. Vit?

Vit Vasista, Chief Financial Officer

Thank you, Steve. REGENXBIO ended the quarter on June 30th, 2024, with cash, cash equivalents, and marketable securities of $327 million compared to $314 million as of December 31st, 2023. The increase was primarily attributable to $131 million in net proceeds received from an upsized public offering of common stock and prefunded warrants completed in March 2024, partially offset by cash used to fund operating activities in the first half of 2024. R&D expenses were $49 million for the second quarter of 2024 compared to $60 million for the second quarter of 2023. The decrease was largely driven by reduced manufacturing and clinical supply costs for AbbVie RGX-314 and RGX-202 and personnel-related costs as a result of reduced headcount. The decrease was partially offset by increases in clinical trial expenses for AbbVie RGX-314 and RGX-202. REGENXBIO expects its balance in cash, cash equivalents, and marketable securities of $327 million as of June 30th, 2024, to fund its operations into 2026. This cash runway guidance is based on the company's current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under AbbVie RGX-314 collaboration, including a potential $200 million milestone for achievement of the first patient dose in the pivotal trial for suprachoroidal delivery for treatment of DR. Additionally, our runway guidance excludes the potential monetization of our priority review voucher that may be received for RGX-121. With that, I will turn the call back to Curran to provide his first set of final thoughts.

Curran Simpson, President and CEO

Thanks, Vit. Today was another exciting day for our 202 program with additional positive data, and as we look ahead to the rest of the year, we are encouraged by our discussions with the FDA and are finalizing pivotal plans that will enable us to utilize the accelerated approval pathway to rapidly advance 202 as a potential best-in-class treatment for Duchenne. Additionally, we are pleased to be accelerating the end-of-Phase 2 meeting for DR in partnership with AbbVie to enable a pivotal start next year. As we close today and reflect on the first half of the year, I'll emphasize that our plans are on track, and we continue to expand value as we make excellent progress in advancing therapies that address significant unmet needs. Thanks, everyone, for your time today. I'll turn the call over for questions.

Operator, Operator

Thank you. We will now begin the question-and-answer session. Your first question comes from the line of Gena Wang of Barclays. Please go ahead.

Gena Wang, Analyst

Thank you. Congrats on the new data regarding the 202 in DMD. So I have two questions, one is about 202. I know you will meet with the FDA, finalized the pivotal study. But do you have a goal for the protein level you wanted to achieve? And do you expect FDA will set some threshold in order for accelerated approval? And another question is regarding the 314, suprachoroidal indication in DR and wet AMD, what's the reason for dose level 4? And will you also account for existing neutralizing antibodies?

Curran Simpson, President and CEO

Thank you for the question, Gena. Can I clarify the first question about the goal for the protein level you wanted to achieve?

Gena Wang, Analyst

Protein level.

Curran Simpson, President and CEO

We've proposed a threshold for microdystrophin level with the FDA, and we will be waiting for further discussions and a final agreement, along with the pivotal plan submission. From our investigations, it seems that a level above 10% is likely to provide functional benefits. This will be an ongoing discussion. Based on our current data, every patient we have published results for has exceeded that threshold. I'll hand over the next question to Steve, if that's alright.

Steve Pakola, Chief Medical Officer

Hi Gena, thanks for the questions. So, 314 and going to dose level 4 in our Phase 2 studies, why do that? We've certainly met our target product profile in diabetic retinopathy. And that's why we're so excited with the advancement towards pivotal that Curran and I have summarized. We've also seen, as I have highlighted, excellent safety. So, I think the key consideration is we're going higher in part because we can. We really have the flexibility given the excellent safety that we believe is due to the compartmentalized route into the suprachoroidal space as opposed to less compartmentalized spaces like the intravitreal space, particularly with our product, RGX-314. So, in collaboration with AbbVie, we're doing this. And I think it also fits just general basic drug development, where if you have good safety to really fully characterize a product, it makes sense to continue looking at higher doses if you can.

Gena Wang, Analyst

Thank you.

Vikram Purohit, Analyst

Great. Thanks for taking our questions. We had two, first, on 202. Could you just speak a bit more about the functional assessment data we're going to be getting later this year? What your guidance is to best interpret that to get a sense of differentiation, real-world differentiation and how that data set might play into discussions with regulators on next steps? And then secondly, on 314, for suprachoroidal and wet AMD. Could you talk a bit about how you see that program moving towards a pivotal program? And how that pivotal program potentially for 314 and wet AMD could overlap with efforts with the subretinal approach? Thanks.

Curran Simpson, President and CEO

Okay. I can take the first question or Steve, do you want to start with the second one or?

Steve Pakola, Chief Medical Officer

I missed the second question. Could you repeat it?

Vikram Purohit, Analyst

Sure. Yes. It was about 314 suprachoroidal and wet AMD. Just wondering what the path is to a pivotal program there? And how you see a potentially pivotal effort 314 and wet AMD overlapping with your efforts with the subretinal program you currently have underway?

Steve Pakola, Chief Medical Officer

Sure. With all the experience we have with wet AMD, we have a pretty good sense of assessing both safety and efficacy, both from our subretinal experience and also the prior cohorts we've done with suprachoroidal. So, I think it's the usual endpoints that we think of. We get to take advantage of the totality of evidence on top of, of course, having good safety. So biomarkers like retinal thickness, where we get a very good objective measure, BCVA, and of course, a reduction in treatment burden. So, we'll really look at all of those to see what type of response we get at six months and longer and really compare that to what's been seen, for example, even in our subretinal program, where we want to see good durability and really maintain anatomic control as measured by retinal thickness and BCVA control, while dramatically reducing injection burden. And we've often talked about at least 50% of patients not needing any injections and over 50% reduction in injection burden.

Curran Simpson, President and CEO

And this is Curran. To the first question, in terms of functional data in the fall, just to baseline, we measure microdystrophin at a three-month time point. And then on the functional assays, we measure generally every three months, a number of functional assessments that you've seen historically with other programs. And in the fall, I would expect to see a significant amount of functional data out to 12 months for the dose level 1 patients. And for some of the early dose level 2 patients, you'll see six and probably nine-month data for them, things like time to rise and SAA as an example. We haven't finalized exactly what data will come out because it's still emerging, but that's the plan for the fall.

Steve Pakola, Chief Medical Officer

Vikram, you also had the question of how do we see this in the context of our ongoing pivotal subretinal program for wet AMD when thinking about suprachoroidal. I think it's fair to say that both we and AbbVie really see opportunities in both, and that's why we both are together advancing further evaluation of SCS at DL4 for this while we continue with the pivotal program with subretinal. Certainly, the opportunity that we see with subretinal, given the gold standard in safety and efficacy that we've shown an excellent durability has us very excited about that. There's, of course, the opportunity to expand the optionality by having an in-office procedure. And we look forward to gathering more data in the additional cohort to keep evaluating that option.

Paul Choi, Analyst

Hi, good afternoon and thanks for taking our questions. My first is, I was wondering if you can maybe just elaborate on interest levels in the patient community and clinical community for 202, a potential pivotal trial later this year or early next year in the wake of the recent elevates label expansion and approval there? And then my second question on 314 is with regard to dose cohort 4 here. I know you specified that there would be prophylactic steroid use here, but could you maybe comment on a view whether there would be potentially reduced need for rescue VEGF use? A competitor recently provided some updated data for their program. And then I think this came as a bit of a surprise. So, just any thoughts there as to whether dose cohort 4 could potentially either increase the injection-free frequency or reduce the rescue usage as you prosecute that cohort? Thank you.

Curran Simpson, President and CEO

I can take the first one, and then I'll have Steve address the second question. I think on patient recruitment for 202, we've been able to check in with sites and investigators at meetings like PPMD, and we've seen nothing but really strong interest in the program. Many of these centers have up to 100 patients. And just having discussions with the investigators around are people still interested in our study, given that there's a product on the market. The resounding answer is yes. There are many patients that are looking at our program and seeing the differentiation that it offers, seeing the safety that's been demonstrated to date. And I would say that there's strong interest. And therefore, on our end, strong confidence in our ability to recruit the study. So, early days, I feel very confident in our ability to recruit the pivotal program. And I'll turn it over to Steve to address the 314 question.

Steve Pakola, Chief Medical Officer

Sure. Regarding DL4 assessment in these indications, we certainly think there's the opportunity that we could decrease rescue use further than what we've seen while maintaining visual acuity benefit stability and also anatomic control. As I mentioned to one of the earlier questions, we have the flexibility to do that. You referred to some other data that's come out earlier this year. And I think one always has to look at that issue of if you can go up higher on dose and if there's either a concern about additional safety issues, including immune response and inflammation. And also, if you start to run into the issue of how long a duration of prophylactic steroids can really be tolerated. So, that's why we're very excited about our safety profile where we don't need extended prophylactic steroids. So, I think the totality of that does give us the chance to go up higher and see if we can reduce injection burden further.

Curran Simpson, President and CEO

And just one follow-on, Paul, to the question on recruitment. I mean one of the purposes of the data release we did today was really to show people the differentiated level of microdystrophin in some of the older patients that have been treated. So, that's just another piece of information that a patient can use when they're deciding what therapy to have their child treated with. So, that was one of the purposes of that release is to help people understand our product a bit more.

C.J. Yeh, Analyst

Hi, good afternoon. This is C.J. Yeh on for Mani. Thanks for taking our question. Could you please comment on your strategy in DMD? Are you targeting younger patients than the current label since you've announced you've expanded the age range to one to three, while the youngest age is four? Thanks.

Curran Simpson, President and CEO

This is Curran. I think it's safe to say we want to build an adequate safety database at a minimum for the patients aged one to three, but we'll be enrolling across a wide variety of ages in the studies.

Steve Pakola, Chief Medical Officer

And we had made the decision to expand and look at this broad age range, including one to three before any label expansion. So, we've seen this opportunity. And now with the results safety that we've seen and the microdystrophin expression we've seen across a wide age range on the upper end. It's a great opportunity to look at one to three-year-old patients.

Operator, Operator

The question comes from Eric calling in for Eliana. Thank you for your question. My first one is about 202. Do you have any internal data on patients who have already been dosed with 202 beyond three months to help us better understand durability and the potential effects over time? What do you expect to observe over time? Do you anticipate that levels will remain stable for three months or more, or will they increase over time?

Curran Simpson, President and CEO

I'm sorry, are you referring to microdystrophin levels beyond three months or other functional data, for example?

Unidentified Analyst, Analyst

Microdystrophin?

Curran Simpson, President and CEO

Okay. No, we're taking a biopsy prior to treatment and then at three months. We don't have biopsies beyond that time point based on direction from the FDA. However, we'll obviously be measuring durability in terms of functional outcomes in the out periods. Yes, we don't have data beyond three months. But I think our preclinical data would suggest really excellent durability. And the construct that includes the C terminus has a longer half-life than microdystrophin that is devoid of that. So, I would expect both higher levels of microdystrophin and perhaps sustained levels relative to what you've seen in other literature.

Unidentified Analyst, Analyst

Got it. And just one quick follow-up. Is there anything that you've been able to identify in terms of patient characteristics that's correlated so far with a higher microdystrophin expression or higher functional assessment?

Steve Pakola, Chief Medical Officer

So, the usual caveat that there aren't enough patients to really have confidence in terms of predictors. I'd say the biggest learning so far is actually that age is not predicting a lower microdystrophin as you go up on age, which has been a concern that the community has had, whether that might be the case in some data from other programs suggest that that may be an issue, but we've not seen that at all. So, I think the striking thing from our data is even in patients aged eight and older, we're seeing very robust microdystrophin levels. Beyond that, we're seeing microdystrophin levels across the patients. So no clear differentiator or predictor in this sample to date.

Luca Issi, Analyst

Thank you for taking my question and congratulations on your progress. Curran, could you provide more details about your recent end-of-Phase 2 meeting with the FDA? I understand you're still awaiting the minutes, but can you confirm that your interpretation is that you can obtain accelerated approval and expression for all patients with DMD, not just those who are currently excluded from the Sarepta label due to age or existing immunity? This is an important distinction, so any insight would be appreciated. Also, regarding DMD, what are your thoughts on a confirmatory trial? Will we continue to use North Star as the primary endpoint, or do you believe there are other endpoints that may be more sensitive? Any guidance would be greatly appreciated. Thank you.

Curran Simpson, President and CEO

I can work a bit in reverse. We'll use North Star, but we're obviously measuring several additional functional indicators, such as time to rise and some of the timed walks associated with it. I would characterize it as we're assessing everything because this is still a field where functional assays are evolving, and each product is different. We want to capture as much functional data as possible across as many indices as we can. Regarding accelerated approval, our strategy is to seek broad approval, not just for patients ineligible for current therapy. The foundation of this approach is the differentiation of the product. One of the key principles of accelerated approval is addressing unmet need, and we believe we have a strong, evolving case for that, with the ambition of obtaining as broad a label as possible.

Brian Skorney, Analyst

Thank you for your question. Regarding the Phase 3 plans for diabetic retinopathy, I would like to understand how confident AbbVie is in initiating this in the first half of next year. What key questions does the company aim to have answered by the FDA, and what do you believe the FDA will require at the end-of-Phase 2 meeting to ensure the Phase 3 study can begin? Additionally, could you provide some insights on the DME cohort? Is there any intention to include more advanced patients, or is the plan mainly to gather additional data from patients who have transitioned to DME?

Curran Simpson, President and CEO

I'll give a brief comment and then turn it over to Steve. I certainly think we and AbbVie have a really high level of confidence in DR progressing into pivotal next year. And I think that's shown in the acceleration of the end-of-Phase 2 meeting into this year. We're eager to get this study up and going. And I think I'll let Steve comment on the details of the pivotal approach, but we feel like there's a very standard approach to developing in diabetic retinopathy, established endpoints and precedent. But I'll let Steve comment on the details of what we're thinking and how that study should go.

Steve Pakola, Chief Medical Officer

Yes. Fortunately, as Curran mentioned, there's a road map for diabetic retinopathy that's been laid by repeat injection anti-VEGF that has really allowed us to know what to expect from a negative control arm if you're not receiving an active agent. And that, of course, really helps us to power studies in combination with the actual proof-of-concept data that we have. We also know a lot about the endpoints that are accepted. So, we feel very confident that this has really derisked quite a lot from a regulatory standpoint. So, it's really assessing some things around the edges and some definitions and the like, frankly, when it comes to the pivotal design. So, I think that's one of the aspects that, given the positive results that we've seen, has allowed us to accelerate and bring this end-of-Phase 2 meeting into Q4 instead of Q1 next year. And then your question on the DME cohort? Yes. So, we are looking at center-involved DME. Of course, that's the traditional indication. So, we don't see a need to go outside the box in terms of the type of patient population that we'd be looking at so that we can really have the context of what others have looked at and what others have seen. So, yes, it's just a great opportunity. We do know diseases like DME or the complication of DME and wet AMD that there's a little higher VEGF drive than exists with, say, non-proliferative diabetic retinopathy without DME. So, we think we're right around the dose response range where this is going to be very interesting to look at dose level 4 in this patient population.

Daniil Gataulin, Analyst

Hey guys. Thank you for taking the question and congrats on your role. I have a quick question on the wet AMD program, this retina delivery. When do you anticipate sharing data from the Fellow Eye study? And as a follow-up to that, how it translates to suprachoroidal injections given those are more likely to interact with the immune system? What is your long-term strategy for treating the Fellow Eye using suprachoroidal to a degree?

Curran Simpson, President and CEO

I'll turn that one to Steve.

Steve Pakola, Chief Medical Officer

Hi Daniil. So, as far as when we would have Fellow Eye results, we have the update today that we completed enrollment of the Fellow Eye study. So, if you add on up to six months or so from that would give you kind of a rough range of when we traditionally have been comfortable releasing data in this type of indication to really show a stable effect at least. We were excited to have this study not just from a regulatory standpoint to have that label expansion to allow bilateral disease. But as you referred, the immune response, whether you would have a greater immune response that could affect safety and/or efficacy when you dose the second eye. Subretinal, I think, is the most straightforward to feel confident that you won't have an issue dosing the fellow, because of the immune privileged status of the subretinal space. Suprachoroidal we've seen much less inflammation and no safety issues in terms of inflammation compared to, say, intravitreal administration. So, that gives us confidence that suprachoroidal would have that opportunity as well. So, we decided to take it one step at a time, first look at subretinal Fellow Eye, but over time, certainly, it would make sense to assess Fellow Eye with suprachoroidal as well.

Operator, Operator

Ladies and gentlemen, there are no questions. That concludes today's call. Thank you all for joining. You may now disconnect.