Welcome everyone to the third quarter 2026 Regenexx BIO earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1 1 again. Please be advised that today's conference is being recorded. I will now turn the conference over to Patrick Christmas, Chief Legal Officer of RegenXBIO. Please go ahead.

Good morning, and thank you for joining us today. Earlier this morning, RegenXBIO released financial and operating results for the third quarter ended September 30, 2026. 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 Risks Factors, and the Management's Discussion and Analysis section of GENXBIO's annual report on Form 10-K for the full year ended December 31, 2025, and comparable risk factor sections of GENXBIO's quarterly report on Form 10-Q, which will be 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, November 5, 2026. 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 in 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'll now turn the call to Curran Simpson, President and CEO of Regenexx Bio.

Thank you, Patrick, and good morning, everyone. Thank you for joining us today. This is an exciting time for RegenXBIO. We are on the cusp of multiple potential regulatory approvals and are preparing for commercialization. Joining me today are Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer. I'll provide an update on our lead programs before turning it over to them for additional details. I'll begin with SuraVec, our potential first-in-class gene therapy for wet AMD and diabetic retinopathy, developed in collaboration with AbbVie. Ahead of our top-line pivotal readout expected later this year, last month we hosted an investor event featuring five leading retina specialists. They discussed the opportunity for SuraVec to transform the wet AMD treatment paradigm. I want to highlight Three key takeaways from the event. First, the unmet need in wet AMD remains significant. While anti-VEGF therapies are highly effective, the burden of frequent injections can be difficult for patients to sustain over time. As a result, patients who are unable to maintain consistent treatment schedules may not realize the full potential benefit of today's standard of care and subsequently lose vision. Second, Cerevec has the potential to benefit a broad spectrum of patients, including those with high disease activity and those with bilateral disease. During our event, the physicians reinforced the advantages of a one-time treatment across the range of wet AMD patients they treat and pointed to patients with high disease burden as ideal candidates for gene therapy. We and AbbVie incorporated these patients in our clinical studies, giving us a potential advantage in obtaining a broad label and treating these patients commercially. Cirovec pivotal studies include a broad population of patients, including newly diagnosed and treatment-experienced patients. Additionally, Cirovec is the only late-stage gene therapy program with reported clinical data supporting treatment in both eyes. Third, Cirovec is well positioned for commercial adoption. The commercial infrastructure for subretinal delivery already exists due to Luxturna and other approved therapies. Further, we know the majority of retina specialists performing these procedures are at high volume centers, many of whom are included in our pivotal studies. We at AbbVie have already laid a significant foundation having trained over 500 retina specialists to date. Together, these factors, combined with AbbVie's global reach and leading eye care capabilities, give us confidence as we proceed towards potential commercial launch. We expect top line results from both Atmosphere and Ascent this quarter, followed by global regulatory submissions in 2027. Looking ahead, we see wet AMD as the tip of the spear in terms of transforming the treatment paradigm for chronic retinal diseases. We are currently enrolling in the Phase 2b-3 Navigate Study in Diabetic Retinopathy. Current anti-VEGF therapy is vastly underused in DR due to the treatment burden for patients who are predominantly middle-aged working adults. We believe a one-time gene therapy has the potential to address this large unmet need and represents another potential blockbuster opportunity for Serovac. Turning to RGX202, we continue to make meaningful progress towards bringing our potential best-in-class gene therapy for Duchenne to patients in the U.S. and beyond. Preparations are well underway to submit the BLA under the accelerated approval pathway in the first quarter of 2027, supporting potential approval in the U.S. in the second half of 2027. As a reminder, the BLA is expected to include data from both our pivotal and confirmatory studies. Outside of the U.S., momentum for RGX202 continues and we remain on track to initiate our XUS randomized placebo-controlled study named AffinityRISE in the first half of 2027. We continue to be highly encouraged by the impressive microdystrophin functional and safety data to date, which demonstrate the potential of RGX202 to meaningfully improve outcomes for patients with Duchenne. We have strong support from treating physicians families, and advocacy organizations. At our FDA-inspected in-house manufacturing innovation center, we continue to manufacture intended commercial supply in anticipation of a potential 2027 commercial launch. We are sharply focused on advancing our late-stage pipeline, executing against upcoming milestones and making disciplined investment decisions that support these priorities. With that, I'll turn it over to Steve.

Thank you, Curran, and good morning, everyone. I'll begin by speaking to our retina franchise, Cerevec, which is being developed in both wet AMD and diabetic retinopathy with our partner, AbbVie. As we approach top-line data later this quarter, Let me review the design of our two pivotal trials, Atmosphere and Ascent. Together, these trials have enrolled over 1,300 patients across 14 countries and represent the largest gene therapy trials ever conducted. Both trials randomized patients one-to-one-to-one to low-dose Cirovec, high-dose Cirovec, or an on-label anti-VEGF control. The control in atmosphere is monthly ranivizumab. In ascent, the control is a plibrocept every eight weeks. The primary endpoint in both studies is non-inferiority on change in best corrected visual acuity, or BCBA, at 54 weeks. Both studies are powered at 90% with more than 600 patients in each. In the phase 1-2 study, at doses similar to those in the pivotal studies, Suravec treated patients demonstrated stable to improve visual acuity and had meaningful reductions in anti-VEGF treatment burden through five years. Additionally, Suravec is the only gene therapy in development to demonstrate the potential to treat both eyes. We believe the ability to target the immune privilege subretinal space is a significant advantage for patients and physicians and could meaningfully differentiate Cerevec from other investigational gene therapies. We look forward to reporting top line results by year end and expect this joint announcement with AbbVie to highlight injection burden and anatomy in addition to the primary endpoint. Turning to diabetic retinopathy where we continue to advance our pivotal development program. Earlier this year, we dosed the first patient in the Phase 2b3 Navigate study, which triggered a $100 million milestone payment from Appy. More recently, at the Retina Society annual meeting, we presented encouraging three-year results from the Phase 2 altitude trial. The new data highlighted long-term results from dose level 3, the same dose being evaluated in Navigate. At three years, 60% of patients improved by at least two steps on the diabetic retinopathy severity scale without additional treatment. And three of four patients who improved one step at one year without additional treatment went on to improve by at least two steps by year three. These findings reinforce the potential of SuraVac to change the treatment landscape for the millions of patients with non-proliferative diabetic retinopathy. Turning to RGX121, we continue to collect and evaluate additional patient imaging and long-term follow-up data with the goal of getting this important treatment to patients. We are working closely with the FDA and with the support of our partner, NS Pharma, and we'll update you on the path forward as we progress. Within the rare disease franchise, our primary focus remains on our potential best-in-class therapy for Duchenne, RGX202. RGX202 was designed to maximize potential benefit through the combination of three key elements. One, our novel microdystrophins. The inclusion of the CT domain makes RGX202 the closest construct to naturally occurring dystrophin, two, a proactive immune suppression regimen, and three, a commercial-ready manufacturing process that delivers industry-leading product purity. As reported in May, our top-line pivotal results demonstrated robust microdystrophin expression, encouraging functional improvement and a favorable safety profile. These positive results together with the statistically significant strong correlation observed between micro dystrophin expression and NSA improvement at one year will be key components of our upcoming BLA submission. Our BLA is expected to include safety data from more than 60 patients across the pivotal and confirmatory studies. It's important to remember that our analysis plans for the pivotal study were pre-specified and have not changed. The confirmatory study has already completed dosing and the vast majority of patients in the pivotal and confirmatory studies received RGX202 using the intended commercial manufacturing process. These are all factors that we believe contribute to the strength of our plan BLA. Outside the U.S., we continue to make progress towards initiating the AffinityRISE global double-masked placebo-controlled randomized trial that is designed to enroll approximately 100 patients with a two-to-one active-to-placebo randomization. We expect this study to initiate in the first half of next year. I want to thank our investigators and the patients and families who make our progress possible. With that, I'll turn the call over to Mitch to review our financial results.

Thank you, Steve, and good morning, everyone. RegenXBIO ended the first quarter of 2026 with cash, cash equivalent, and marketable securities of $251 million compared to $241 million as of December 31, 2025. The increase was due to a $100 million milestone payment received from AbbVie in July 2026 for the dosing of the first patients in the Phase 2b-3 Navigate study and the net proceeds of $108 million from an underwritten public offering of common stocks and pre-funded warrants completed in July 2026. This total excludes a $49 million payment received in October 2026 pursuant to a settlement agreement with Surrupta Therapeutics. Our cash runway is late into fourth quarter of 2027, beyond multiple milestones, including top-line data in WET-AMD, the BLA submissions for RGX-202, and CERAVEC in WET-AMD, and the expected PDUFA date for RGX-202. This cash runway guidance does not include the receipt of any potential proceeds associated with other potential non-dilutive sources of funding. We continue to receive Zolgensma royalties in select OUS territories and IFSMA royalties in the US and certain OUS territories. We find ourselves well positioned as we advance towards multiple potential product launches. With that, I will turn the call back to Curran to provide final thoughts.

Thank you, Mitch. Before we turn to Q&A, I want to reiterate our excitement as we approach key milestones in our lead programs through the remainder of 2026. The CERAVEC pivotal topline data expected this quarter could reshape the treatment paradigm for wet AMD. With RGX202 quickly advancing towards a potential BLA submission early next year, we're making important progress in bringing this These key near-term milestones are paving the way for our transition to a commercial company via programs with blockbuster potential. With that, I'll turn the call over for questions. Operator?

Thank you. At this time, we will conduct the question and answer session. As a reminder, to ask a question, you will need to press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. Please stand by while we compile the Q&A roster. Our first call comes from Annabelle Samimi with Stifle. Go ahead, your line is open.

Hi, good morning, guys. Thanks for taking my questions. I've had a few here. So for 314, I want to know how important the fellow I data is going to be and any of the regulatory reviews that you're going to do that the FDA has for gene therapy and wet AMD. And then secondly, also for 314, clearly you're approaching the tail end of this program. You have 500 plus physicians that have been training and they seem to want to use 314 and they seem to know which patients they'll want to use it in. But they've all said that it's going to depend on the payer. So can you talk about The payer work that you've done here, the reception from the payers, and if there is actually reception for gene therapy from the payers. And then I guess lastly, one quick one on 202. I thought the BLA, is the BLA filing still a rolling filing or is it just a straight filing? And what impact does that have on any kind of timing of review? Thanks.

Thanks, Annabelle. I'll take the last question first just to clarify. The intention is in Q1 we will file the full BLA for RGX 202, so we chose to change the filing strategy from a rolling BLA to just filing the entire BLA because we wanted to have an opportunity this year to discuss in a pre-BLA setting the data that is intended to be submitted. So the entire BLA will go in. That has, in a great way, no impact on the eventual timeline for the PDUFA date because it'll be a single submission. I think on the payer side, I would characterize a few things around discussions we've had. And this is certainly an area where AbbVie will take a lead role in discussions as we get closer with payers. But I think in general, some things to note are the codes for reimbursement associated with subretinal administration are well established. And being the key element of delivery of the product, there shouldn't be a lot. The only thing we really need ultimately is J-code for the product itself. So the mechanics of payment I think are well established, albeit for programs that are more Rare disease like Luxturna as an example. But we feel really optimistic. And one element that is critical to discussions we've had with payers to date is the existence of long-term data, durability data that supports the gene therapy and the benefit from that. We're out with the subretinal administration for what, A&D five years now with some patients. And so that will be a really important element as we discuss reimbursement with payers down the road, but we don't see any particular obstacles. I'll let Steve speak to the fellow I study. We do think it is absolutely a critical element of our value proposal, and Steve can describe what we've discussed, how that's incorporated in the study and what we've discussed over time with FDA on this topic.

Sure. Hi, Annabel. Thanks for the questions. Yeah, the fellow I aspect actually has always been a critical component for our program, and it's an example of how comprehensive our global program has been. And I think a key aspect is we're really differentiated here in the ability to treat the fellow I. This is one of the benefits of the immune-privileged subretinal delivery, that you can have more comfort from a safety and efficacy standpoint to treat both eyes. And that's very relevant, not just for the FDA, but of course all of the treatment community, the retina specialists, because roughly half of patients, not surprisingly, do have or will develop fellow eye disease. A treating clinician wants to know they can not only treat the first eye, but also have the opportunity to have one-time treatment for their patients in the fellow eye. Fortunately, we always plan for this, and for a lot of the same reasons, the FDA has also cared about this. It has always been a part of this, and we did align at the end of Phase II meeting for Having the package of patients who have been treated in the fellow eye. So we've addressed that in two separate studies. One, a bilateral treatment study. And the other is actual patients from our various clinical studies who can roll over and have the fellow eye treated. Back on your question of the I think it's important to highlight AbbVie's work on this and their commitment to the global potential for one-time gene therapy, where the actual evidence of their commitment, one is clearly the ACHIEVE Phase 3b study that they have taken on proactively. This is another step that we think is just going to bolster the value proposition.

Great. Thanks for the caller. Congratulations.

Thank you. Our next call comes from the line of Maxwell Score with Morgan Stanley. Your line is now open.

Great. Thank you very much for taking my question. So after the Q1 BLA submission, when and where do you expect to present updated 201 data? What can we expect in this update?

Just trying to lay expectations for next year. Thank you.

Thanks, Max. We haven't given a specific meeting or event around disclosure of the data, but we do intend to publish the data post-filing of the BLA. I think we've pointed to, in terms of the submission, which will resemble very closely the future publication is, we have a safety database that's accumulated between the pivotal study and confirmatory study that is north of 60 patients. We have biomarker data for the full pivotal data set and then about half of those, which was a 30-patient study, will be through their 12-month functional assessments. So I think that would give the parameters of the data that would be supplied. One thing I would point out that I think is super critical to our discussions with FDA and the power of the data set that we have is we're the only company that's been able to correlate microdystrophin levels to functional benefit. and that was an element that was missing in the Elevitus Adcom if you go back to the discussions there. They weren't able to do that. That removes a significant element of risk associated with a single arm study because the children don't know how much microdystrophin they have so correlating the level of microdystrophin to functional outcomes is a super powerful element of our value proposal and I think what we hope will Please the reviewers who didn't necessarily see that in the past. Very helpful. Thank you.

Thank you. One moment. Our next call comes from the line of Lily Songo with Lyric Partners. Your line is now open. Hi. Good morning.

Thank you for the update. Just a little bit regarding the pre-BLA meeting for Duchenne. Going into the meeting, what are some of the points or elements for which you go in with confidence and those points that you would particularly try to gain some clarity on? And also, how long after the meeting do you expect to get and share feedback?

I can start with the last part of that. Typically, we'll receive minutes from the meeting about 30 days post the meeting itself. I think the objectives of the meeting are basically point by point to discuss with FDA how we feel our program meets the key criteria for accelerated approval. So the key elements of that are we feel that we're in a great position on safety. We've got a large safety database created now between the pivotal and the confirmatory study. and a significant reduction in liver toxicity demonstrated in our pivotal data set and hopefully we can show that as well that carries over into our confirmatory study which hasn't been published yet. The second part as I mentioned earlier is the ability to correlate the micro dystrophin levels we've measured in the biopsy samples for the primary endpoint to NSAA in particular, which is a 12-month assessment on function, that correlation we feel underpins the fact that the primary endpoint, microdystrophin, is reasonably likely to predict a clinical benefit. And that's, I think, the main message we want to discuss with FDA, again, because that's the main tenet of an accelerated approval pathway. And so we're really excited about our data and excited to engage with FDA. And all of the elements related to the BLA submission are on track for a Q1 submission.

Thank you for the added color. Thank you. One moment. Our next call comes from the line of Greg Sivanova with Mizuho. Go ahead. Your line is open.

Hey, good morning. Thank you for taking my questions and congratulations on the progress that you're making across your two lead programs. I was wondering if you could just provide an update on maybe the competitive landscape in DMD. You do have another gene therapy competitor I think that just reported yesterday, no conference call, but can you maybe compare and contrast kind of the Thank you, Greg. Happy to comment on that. I think it starts out with the initial design of the construct, which includes the C-terminus. So from the very beginning, we wanted to bring a differentiated product into clinical development.

And the C-terminus in our preclinical data showed a clear benefit from a microdystrophin construct that's devoid of the C-terminus. And we think that's what's carrying over into the clinical study. The other elements of differentiation I think that are critical is, I'd say second, the manufacturing process, which delivers a capsid at 80% purity, also at a great cost of goods. It's hard to get both, but we've been able to achieve that. We've invested in CMC for over seven years, and now we're bearing the fruits of that for this program. I think that's a second element, which we believe not only helps with the high microdystrophin levels we're seeing in patients' biopsies, but also on safety. And then that leads to the last leg of the stool is the Immune Suppression Regimen that's been developed for this program, as I mentioned earlier, has allowed us to drastically reduce the liver toxicity events seen with patients. But more importantly, and I think the long-term value of the program is it allows us to deliver the maximum dose of product to give the Duchenne boys the maximum potential benefit of long-term functional benefit. and I think that was the aim is to match our preclinical data where we saw tremendous restoration of function in the MDX mouse models. We were able to get to that dose because of that IS regimen and now we're seeing it in our functional data which does differentiate it from our competitor. We still to this day haven't seen anything to compare to from our competitor whereas I think we've been very are all transparent and proud of our functional data and the fact that it correlates to the microdistricts and levels.

Okay, great, thanks. And if I could just ask a quick follow-up. I think as related to your reported financials, the royalty revenue I think was just maybe slightly below what, or actually it was actually better than we had modeled. Could you just walk through and the complexion of the royalty for this particular quarter.

Yeah, I'll let Mitch cover that one.

Yeah, that's a great question. As a reminder, SOGENSMA, the IP for the U.S. have since expired. There are certain jurisdictions that we continue to receive royalties for SOGENSMA, but equally so, IFISMA has since launched and is launching in a pretty good fashion. I think the differential that you're seeing right there is the pickup from Avisma as we get the royalties from certain jurisdictions, including the U.S.

Thank you.

Thank you. One moment. Our next call comes from the line of Brian Scorny with Baird. Go ahead. Your line is open.

Hey, good morning, guys. Thanks for taking my question. Maybe just to follow up a little bit on the last question, just wondering on FDA discussions, obviously the three companies are all dialoguing with the agency right now on prophylactic immunosuppression regimens. Repta is trying to provide data, effectively switch to more or less your regimen, given the history of some deaths on treatment due to uncontrolled liver tox. You're talking about a submission with the pre-specified regimen and solids going to the agency with sort of a lighter regimen with just corticosteroids. So I guess it'd be great to get some granularity on how the agency is actually discussing sort of these different regimens and their concerns over some of the liver talks that we've seen for systemic gene therapy and the burden of evidence needed with and without serolimus and eculizumab in the clinical practice right now. You know, maybe this is more a Sarepta question, but how prevalent is it becoming among physicians Thanks.

Yeah, I think that safety, in particular for an accelerated approval approach, is absolutely critical. And I think what we've shown in our pivotal data set on safety is highly encouraging. We don't really see liver enzyme elevation through the course of treatment. And again, keeping in mind that we're treating at 2E14 per kilo, which is the maximum dose we wanted to attain based on our preclinical data. So that in return gives patients the best possible chance, I believe, for functional benefit. I think FDA will have this as top of mind. I think our data set will be in line with that. We do know that other companies are adjusting their IS regimen in the field, in clinical studies, etc. We haven't had to do that in any of our studies. We've been consistent throughout our pivotal and confirmatory study patients using the same exact immune suppression regimen. So I feel like that's a strength for our program is the consistency of product Consistency of Safety Outcomes, and the fact that the patients are all doing well. I think it's important to maybe look two years out, though, and say, what if all these companies, including ourselves, equalize on safety because we learn over time how to deliver AAV more safely? That's where I think we're still going to win because we have the C-terminus, we have the highest purity, I believe, in the field. And what the market will react to then is what will deliver the best efficacy, if safety is sort of the same across programs. And I believe we're in a position to win based on the data we've published and the reaction we're getting, not so much in just the functional assessments, but what we hear from investigators. I can let Steve maybe comment a little bit on what we're hearing in the field as well on this topic.

Yeah, I think one of the nice things is, of course, we have actual experience doing this. And as Vern has highlighted, right from the beginning, we've used this regimen. And I think the aspect that comes here is, if you look at the overall benefit-risk equation, certainly patients and patient families want safety, and they also want to have their best shot on goal because they literally only have one shot. As soon as you take a gene therapy, there's no chance to get a second one. So you really want to have an optimal chance at success, and that's where all the differentiators that Curran mentioned come in. And I think on your question of the regulatory view on this, it really is the highest sensitivity in the field delivered. And I think the differentiation there is so clear that It really adds up to a really strong case for us as far as benefit over and above existing therapy.

Thank you. One moment for our next question. The next question comes from the line of Luca Izzi with RBC. Your line is now open.

Oh, great. Good morning, team. Congrats on all the progress. This is Cassie for Luca. Curran, as you have elaborated on and highlighted already, the pivotal data showed a highly statistically significant correlation between week 12 microdistrict expression and NSA improvement at one year in nine patients. So now as you move towards your pre-BLA meeting, how do you expect the FDA, especially now under new leadership, to weigh this correlation given you were the first to show such data and evidence is only now in single-digit number of patients Will there be a threshold or a similar degree of correlation they expect? And lastly, the correlation was demonstrated on the NSAA as a functional measure. Can you speak to the timed function test if those are also showing a similar picture and if such data will be included in the DLE package? Thanks so much.

Thanks. Great question. We do think that the review team will be excited about the correlation between the two because I think it ultimately speaks to the drug effect. And I think in a single arm study, the things that we hear in particular, I think Kareem McHale talking about at FDA, where they're looking at updating regulatory frameworks for rare disease, What we're bringing to the table in terms of our data set is directly in line with what we're hearing from FDA leadership, and that would be a few things. One is magnitude of effect. I believe our NSAA data to date shows a dramatic magnitude of effect advantage, particularly in the older patients where they would normally be in a decline phase. And then I think, again, the second is in a single arm study, the ideal outcome is that you can correlate your biomarker to a clinical benefit that underscores that your biomarker is reasonably likely to predict clinical benefit. And again, I think that's directly in line with what we're hearing from FDA. So I think as FDA focuses on rare disease, which I believe they are, and as we bring forward the data that we've shown and the additional data that'll be part of the BLA submission, those two are directly in line with each other. Let me let Steve talk just a little bit about what we're seeing in some of the other time function tests.

Yeah, so one of the strengths of the functional data that we've released at the top line standpoint is not only the magnitude of effect but the consistency of effect. Where we've seen functional benefit in not just the broad NSAA that traditionally people have thought is not so sensitive, but yet we've seen that effect. We've seen that effect across different ways of different methods of controlling. And then we've also seen consistent effect across the time function tests with a particular focus on time to stand as the traditional One that is of most interest. We'll have more patients, 14 patients, for the BLA. So we'll look for continued nice magnitude of effect and totality of evidence. And since we've seen this strong correlation with NSAA, the broad measure, yes, we'll also be assessing correlation with the time function test. We feel good about this overall analysis because of how consistent the magnitude of the effect was.

I think one thing just to add to Steve's note is in some of the communications FDA has sent our way, They have acknowledged the difference in microdystrophin that our product is unique and contains the C-terminus. So they're looking at it, I believe, as a unique construct that deserves a unique review.

Thank you. One moment. Our next question comes from the line of Sean McCutcheon with Raymond James. Your line is now open.

Hi guys, thanks for the question. Can you speak to the randomization criteria, patient disposition, and rescue criteria for atmosphere in a sense, how this differs to competitor pivotal studies for extended treatment interval therapies, and how that informs expectations for BCVA stability and interpretation of treatment burden reduction? Thanks.

Hi Sean, happy to do that. I'll let Steve give the specifics.

Sure. Hey, Sean. So you've hit on a lot of the key aspects that certainly were part of our prospective planning. And I think the key aspect is we wanted to have clinically relevant data. So for us, as far as the patients we enrolled and randomized, our goal was to address those patients that the clinicians told us they were eager to to be able to treat with gene therapy, a one-time opportunity. And we hear over and over again that the big unmet need is the patients who have high disease activity and therefore have still frequent injections, even with the newer agents. And that's roughly in the 40%. So if you want to address that unmet need, you have to actually evaluate it in your phase two and particularly your pivotal study. So we include treatment-experienced patients, including those patients with high disease activity. And we also use screening criteria where, yes, you have to show that there's a response to ranivizumab injection but we also look one week after dosing where that's going to allow you to enroll not just patients who are responsive but also these patients who yes they respond at one week but they may have disease recurrence very quickly and therefore those are exactly the kind of patients you'd want to enroll. So the other benefit is we use the same inclusion-exclusion as are bioreactor pharmacodynamic study and also the same rescue criteria. And that gives us confidence because in that study we saw stable to improve vision, stable disease control, and a very high reduction in treatment burden, including patients not needing any injections at a year. You mentioned the rescue criteria. Here again, same as the bioreactor study, it's all pre-specified objective based on change in best corrected visual acuity and or reduction or worsening of fluid or disease activity measured by OCT. So all these factors give us confidence here, and we powered the studies based on consideration of of this patient population. So the goal, again, was having positive studies, but also clear evidence in the patients that we care most about. So it really gives an example of how comprehensive our programs are in wet AMD, where they're not just large, they're not just global, but they're also looking at the patients that the clinicians care about.

Thank you. One moment. Our next call comes from the line of Ellie Merle with Barclays. Your line is now open.

Hey, guys. Thanks so much for taking the question. Just ahead of the Phase III wet AMD data, I guess, expected imminently, a common investor question is around sort of the effect size on treatment, burden reduction, and the proportions that will be injection-free. and how to think about that in the context of the broader gene therapy landscape given we'll be getting other phase three trials from multiple other companies next year as well in the gene therapy space. I guess given that you have a more severe patient population enrolled, I guess how are you thinking about what would be a competitive proportion of patients that become treatment free and then the proportion of like percent treatment reduction and how to think about how this could compare to some of the other trials that might be enrolling more treatment naive patients. Thanks.

Thanks, Allie. I'll start with just I think we're in really good shape in terms of alignment with AbbVie that at the outset of the program and really based off our Phase 1-2 data, we were able to develop a target product profile that is in full alignment between us on those So I'll let Steve comment a little bit on what expectations we have in terms of sort of what good looks like in the studies.

Yeah. Hi, Ellie. That's a great question because context matters here. You can't really go across studies and compare nominally treatment reductions without taking into account how tough to treat The goal is really to have at least a 50% reduction in treatment burden with a meaningful proportion of those patients not needing any injection. And the key is that If we hit that mark, we're hitting it in a very tough to treat population. So that's the evidence that the clinicians need and the patients need in order to make their decision on treatment.

I think one other aspect that is important to that, that we've gotten information from polling a number of retinal specialists is that if the program shows beyond 12-month durability, there's a high level of interest in gene therapy and just As a reminder, we're out five years on some patients. So I think the element of durability that's associated with sort of value and trust in our program, we're going to have excellent data at the time of review and eventual disclosure to support all of that.

Great. Thanks. Thank you. One moment. Our next question comes from the line of Yi-Yen with HC Rainwhite. Go ahead. Your line is open.

Thank you for taking my questions.

Could you please clarify what decisions the FDA has to make post the pre-ELA meeting to enable the accelerated approval of 202?

Thank you.

Hi, Yi. Thanks for the question. At the main decision and one of the main questions that's the topic of the pre-BLA meeting is, does the data set that we have in hand qualify for review under the accelerated approval pathway? And obviously, we'll have, I think, a reasonable understanding of their position on that coming out of the pre-BLA meeting. But the true stamp on that is two months post-filing that they accept the BLA for review. And then it really becomes a review process through the next six months to go in depth through the data and support that review process. But I think the key decision point coming up, and we'll get some color on that through the pre-BLA process, is acceptance of the BLA under the AA pathway. And we expect that about two months after submission.

Thank you.

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