Good day and thank you for standing by. Welcome to the Q1 2022 REGENXBIO Inc. Earnings Conference Call. At this time, all participants are in listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions] I would now like to hand the conference over to our speaker today Mr. Patrick Christmas, Chief Legal Officer. Please go ahead.

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 first quarter ended March 31, 2022. 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 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 31, 2021, 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, May 4, 2022, 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. Ken?

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I’m pleased to begin today’s call with a recap of our recent business highlights as well as an update on our corporate goals. Steve will then provide an update on our clinical programs and Vit will provide an overview of financial results for the first quarter ended March 31, 2022. At the end of the call, we will open up the line for questions. Before Steve gets into the pipeline progress in detail, I would like to provide some context on the gene therapy industry. Despite the recent market performance of the sector, I cannot help but be optimistic for the future of gene therapy. Over the years we’ve seen a high level of investment in people, quality and safety monitoring, and have developed a deeper understanding of the science across the industry. The recent challenges experienced across the industry are not if history tells us as the guide uncommon for novel modalities like gene therapy. Challenges created opportunities in a relatively new field like gene therapy the recent challenges have led to greater collaboration and involvement with key leaders, including regulatory agencies that I believe will better to find the path towards getting gene therapies to patients with unmet needs as efficiently as possible. Well, this past year has led to a difficult decision for a number of gene therapy companies, I’m encouraged that REGENXBIO and the field of gene therapy had deeper in the 2022 and beyond. To this end, I’m incredibly proud of how our company has been advancing our pipeline during these times and I believe our fundamentals have never been stronger. This is why we felt this past quarter was the right time to announce our 5 by 25 strategy to progress 5 AAV…

Thanks, Ken. I’ll begin with an update on RGX-314, which is being developed in collaboration with AbbVie to treat multiple ocular indications including wet AMD and diabetic retinopathy. RGX-314 uses the NAV AAV8 Vector to deliver a gene and coding a therapeutic antibody fragment to inhibit vascular endothelial growth factor or VEGF. Wet AMD is the leading cause of vision loss in people over 60, affecting more than 2 million patients in the U.S., Europe and Japan. The current standard of care for wet AMD patients are anti-VEGF treatments, which require patients to receive injections into the eye every 4 to 12 weeks. Real world evidence shows that patients with wet AMD are severely undertreated due to the unsustainable treatment burden of these frequent injections. As a result, the majority of wet AMD patients experienced significant vision loss over time. RGX-314 is being developed as a onetime treatment for wet AMD that has the potential to reduce the frequency of anti-VEGF treatments and preserve vision loss for patients with wet AMD. Data presented to-date for our Phase 1/2 clinical trial RGX-314 for the treatment of wet AMD using the subretinal delivery approach has demonstrated a durable treatment effect over three years now including mean improvement in vision and stable retinal thickness and reduction in anti-VEGF treatment burden. We believe RGX-314 represents a significant potential advancement for the treatment of wet AMD. We continue to enroll patients in ATMOSPHERE and ASCENT our two pivotal clinical trials evaluating the efficacy and safety of our RGX-314 in patients with wet AMD using subretinal delivery approach. ASCENT is the first trial to be initiated under our eye care collaboration agreement with AbbVie. These two trials are expected to support a PLA submission for RGX-314 in 2024. We are also advancing to additional programs that…

Thanks, Steve, for the good updates and to the team for the progress we’ve made over this past quarter and in the first part of this year. Before turning the call over to Vit I would like to highlight our progress that we’ve mentioned about manufacturing. As you know, we’ve begun utilizing our new headquarters in Rockville, Maryland that houses our new cGMP manufacturing facility. I’m excited to share that this facility is now operational, allowing for high yield production of NAV vectors and scales up to 2000 meters using our platform suspension cell culture process. With the new headquarters in GMP manufacturing facility, we are continuing to expand our capabilities from basic research and development to commercial scale infrastructure. We have over 21,000 square feet of our corporate headquarters dedicated to our in-house GLP Pilot Plant, advanced analytics lab, multiple 2,000 liter bioreactor suites for GMP manufacturing of both drugs and flexible state of the art fill finished suite to support multiple programs. This capability remains a key differentiator for REGENXBIO and a key element of our strategy. Our facility of cutting edge allows us to move quickly from candidate selection to the production of clinical grade material which supports accelerating the early development of our AAV therapeutics. Additionally, we believe our approach focuses on early product quality and process control which lessens the need for changes during clinical development to enable efficient transition from clinical trials to commercial readiness. Furthermore, our manufacturing team has formulated AAV therapeutics to be used with a number of innovative delivery device solution and techniques including pioneering the clinical use of the super choroidal technique and the intracisternal interventions that Steve alluded to. We believe being a leader in innovative device delivery of AAV therapeutics is a distinguishing competency for REGENXBIO. So at this stage, I’m going to turn the call over to Vit for review of our financial guidance.

Thank you Ken. REGENXBIO ended the quarter on March 31, 2022 with cash, cash equivalents and marketable securities totaling $764.8 million compared to $849.3 million as of December 31, 2021. The decrease was primarily the result of cash used to fund operating activities and capital expenditures as well as temporary unrealized losses on marketable debt securities during the quarter ended March 31, 2022. R&D expenses were $55.6 million for the quarter ended March 31, 2022, compared to $39.7 million for the quarter ended March 31, 2021. The increase was primarily attributable to personal costs and costs associated with clinical trials and manufacturing related activities for our lead product candidates. It was partially offset by REGENXBIO through and for development cost reimbursable by AbbVie under eye care collaboration. In accordance with a collaboration agreement REGENXBIO will continue to fund certain ongoing clinical trials for RGX-314 through the end of 2022 while other 314 development costs are shared with AbbVie. Beginning in 2023, AbbVie will be responsible for funding the majority of all RGX-314 development expenses. Based on our current operating plan, we expect the balance in cash, cash equivalents and marketable securities of $764.8 million as of March 31, 2022 to fund our operations into 2025. With that, I will turn the call back to Ken to provide final thoughts.

Thank you, Vit. Overall, REGENXBIO continues to perform at a very high level and I would like to take this time to thank our REGENXBIO team, our investigators and patient communities for their commitment to the development of our innovative AAV therapeutics. Taking just one moment to circle back on RGX-202 I want to reiterate our strong commitment to the Duchene community. This unexpected delay reinforces the importance of our in-house GMP manufacturing facility. We plan for the first runs in this facility to support our clinical supply for RGX-202, and RGX-314 program. We’re also able to begin to install new process improvements that we plan to use in our facility that will be expected to produce, for example, in the case of RGX-202 approximately five times improvements in vector yield per batch than previously used processes. s So to summarize what you’ve heard from us today, in my views on the overall status of our progress so far this year, we continue to be a leader in the field of AAV therapeutics. There are over 2,000 patients dosed with AAV therapeutics derived from our NAV technology platform. Our global eye care collaboration with AbbVie continues to advance and is on track for the first PLA filing in 2024. Emerging clinical trial data and expanded trial enrollment mentioned by Steve supports excellent progress in our super choroidal delivery programs to unlock additional value. We have strong science and clinical data behind our rare disease pipeline of AAV therapeutics, where we look to take advantage of accelerated pathways to address high unmet needs. Our internal GMP capability is operational, supporting the high yield manufacturer of quality AAV therapeutics across a diverse range of programs and at the scales necessary to support clinical development and commercialization. We also have an amazing team…

Certainly. [Operator Instructions] Our first question comes from Alec Stranahan of Bank of America. Your line is open.

Hey, everyone, this is John on for Alec. Thank you for taking our questions. Just a couple from us. I think the first question could you just elaborate a little bit more on the decision to delay the 202 program. What exactly was the issue that you ran into? And how do you think this delay will affect kind of the overall competitiveness of the 202 in the field of DMD? So that’s the first question. Second question was also on DMD. So how do you with recent news on the obviously safety issues run into by other companies how do you wish to kind of learn from all of that and kind of approach and eliminate the concerns of those issues in your study? That’s the second question. Third question, just a short one on manufacturing, obviously, in-house manufacturing is very promising. But what do you think are the challenges going forward with regards to that? Thank you.

Okay, John, thanks for the questions. I’ll start. I will let Steve comments on clinical trial comparisons and also circle back on in-house manufacturing. So as I reported, we had this unexpected isolated observation in the very final stages of a manufacturing process for RGX-202 clinical supply. And the unexpected nature of this in the late stage nature of this caused us to have to report this delay and re-guide first patient dose to the first half of 2023 which is I think telegraphing that we have a 6 to 12 month delay. This was something that was reported at a third party contract manufacturer of ours and that didn’t meet our quality criteria. So that relates to your third question, John, which is look we’ve been looking at the landscape for a while, we’ve been communicating with all of you about how important we think it is to bring the quality and availability of clinical supply and commercial supply for our pipeline in house. We started that investment, pre-COVID, with the build out of our headquarter facility in the intent to bring online the GMP facility. And we announced today that the GMP facility is operational. So for us, a key elements of the 5 by 25 strategy to be able to be a company with five programs in pivotal phase or commercialized by 2025 like imperative for us to bring those in house capabilities for GMP manufacturing here to Rockville and enable our team that level of support. With respect to the 202 comparisons to other trials in our approach, I’ll turn it over to Steve.

Hey, John, thanks. Thanks for the good questions. On the historical data sets that we can look at in the AAV field we do have the benefit of all of our licensees and all of our collective understanding in the field of both in Duchene but also other programs. And I think we got to take advantage of all of this. And I think it’s been very collaborative in the field of later, appropriately. So where we get to take advantage of what’s been learned? I think each program is specific though, and you can’t really carry too much forward from one program to another because of all the different aspects. So for example, AAV8 versus AAV9, so some of the programs have seen complement activation, for example, with AAV9 at higher doses. And the other finding of potential transgene immunogenicity, but we’ve taken advantage of the learnings from that in our AAV8 program where clinically complement activation hasn’t been as a real issue in the same way that has been seen with high dose AAV9 nevertheless out of an abundance of caution for our first in human study we’re being very robust and how we look at this initially where for example, including complement inhibition, with Eculizumab just a short course as well as other typical immune suppression regimens to really cover for that aspect. So we feel very excited both based on that as well as our preclinical package where we’ve seen very good safety at doses including the very first dose that we’re looking at clinically by the way we see very good clinical and pathophysiologic proof of concept in our model. So all of this ties together make us feel very good about our innovative transgene promoter within AAV8 as really an exciting program to advance on.

And our next question comes from Vikram Purohit of Morgan Stanley. Your line is open.

Good day everyone. This is Gospel on for Vikram. So I’m going to we have two questions sort of the first one is, once dosing has begun for the RGX-202 program, how many patients were updated? And how much follow up do you think you would need prior to sharing our initial outlook or the data? And then for the initial data set that is initially generated what do you aim to see to establish that 202 have a competitive safety and efficacy profile?

Thanks. Gospel, this is Ken. I’ll start and let Steve provide some more color, if necessary. So we’ve reported that the trial design at this stage includes two dose levels with a basic three plus three design 1E14, moving up to 2E14. And each one of those dose cohorts has the potential for expansion as well. When it comes to the profile of RGX-202 and the execution of the design, we’d be looking to complete the initial cohorts of three patients, look at some of the primary safety evidence as well as the secondary endpoints that we’ll be measuring such as micro dystrophin expression at about 90 days as well as, intermediate measures of things like functional outcomes, and imaging on the order of three months, six months and beyond. We’ll be looking for there to be similarity parody, if you will, to existing treatments that are microdystrophin, AAV gene therapy based candidates being studied clinically. And look for a safety profile that is similar to improve microdystrophin levels that are similar to improve, to continue to support evidence that we see a place for RGX-202 in the treatment paradigm. I think the points to emphasize about differentiating aspects that we think are key in addition are we’re using a different capsid. And to the extent that we see the same types of evidence that I think others have demonstrated, or can continue to demonstrate, with microdystrophin based AAV gene therapy, we believe that there could be a place in the market for patients who couldn’t access because of pre-existing immune status, other types of products using AAV9 or other capsid, that would be able to access RGX-202 for instance, because of its differentiating immunological profile with AAV8. In addition, we think the C terminal domain is likely to provide opportunities for us to look for longer term functional outcome improvements. Things that maybe Gospel we wouldn’t pick up in the first 90 days or six months, but at a year, at 18 months or two years, would start to show a longer term differentiating profile. And that would be meaningful, not only for our continued long term follow up and clinical development, but also in the marketplace. And then finally, on the manufacturing side, the emphasis today sort of lesson learned on the importance of having control over the quality as well as the control of the process itself here at our manufacturing facilities are of paramount importance and having control over that supply, being able to advance with our people, our processes, and with improvements to those processes that we plan to bring to our new facility as well we think that being a major contributor to supplying the market, and with higher yields processes, potentially having an advantage on cost to goods will be things that in addition to those early looks are meaningful, long term contribution to helping patients with Duchene and having a differentiated profile.

Thank you. And our next question comes from Gena Wang of Barclays. Your line is open.

Thank you. We have three, this is Tom on for Gena. We have three questions. The first one is about the 314. So as you completing the re-moment of two Super choroidal trials I am thinking, when can we expect to see the protein level data and second is regarding to the cGMP facility. Just to clarify beyond the analytical wrongs that you mentioned are there any other or what are the other steps to go through to get a folio ready for clinical supply? And for the Duchene program the last question is you I saw the ASGCT abstract you should hire AAV mediate transgene expression in the skeletal muscle in the disease model versus normal mice, driven by down regulation of new factors. Just wondering how relevant is defining to human? And how does this help you to refine the dose escalating strategy and also dose fraction strategy in a clinical study.

Thanks for the question. Do you want to grab 314?

Sure. So on 314, as you mentioned, we have the two super choroidal in office delivery studies, one in wet AMD the other in diabetic retinopathy where we have the clinical proof of concept in both studies, and we continue to evaluate dose response. In both indications, we have very good non-invasive ways to assess clinical response. Your question on aqueous humor protein, we did choose to include measurement of a aqueous humor protein with this new route of administration as these were the first studies ever done with super chorodial delivery just to include that for assessment. And we haven’t given any guidance on when we’d have data for that and these ongoing studies are focused particularly now that we actually have clinical signal and even clinically meaningful signal and even a signal in the case of diabetic retinopathy on the endpoint variable that’s used for actual approval. Our focus is heavily there. I think your second question related to bioreactor clinical supply where fortunately, that’s not on the critical path, as far as how we advance there. So as Ken mentioned, we continue to be on track with our plan to have a PLA submission in 2024. And that’s really driven on completing the two pivotal studies.

Right, and with respect to the question relating to the preclinical work done and around candidates for Duchene including RGX-202. There is a totality of evidence that obviously went into support our IND for the purposes of selecting doses and for FDA granting and safe to proceed. We certainly looked at disease models as well as different types of background models with respect to evaluating things like transduction efficiency, and safety and bio distribution. I think it’s people in our research and development organization, as well as people in this space have learned that there are differences across animal models with respect to a disease model, and what you may see in terms of transaction and what you might find in black six mics or other types of models. So we reconciled all of those things, build the appropriate package to be able to inform the doses that Steve alluded to today, starting at 2014. Planning for dose escalation 14. This we’re continuing to roll out over time different parts of the package that went into the IND, but the totality of evidence has been there, since we filed the IND at the end of last year. Thanks for the question.

And our next question comes from Mani Foroohar of SVB Securities. Your lines is open.

Hey, good afternoon. This is Rick on Mani. Thanks for taking our questions. So given there’s roughly a 64 month delay in the dosing of RGX-202, could you just walk us through some of the steps that need to take place before you can initiate dosing? Is it simply that you have to produce a new batch of clinical product from your in-house facility? Or would you potentially have to revise manufacturing protocols seek a new third party manufacturer or any other potential steps?

So I think the essence of the 6 to 12 month delay is that we have to re establish our clinical supply and the primary plan for doing so is using the new GMP capabilities here in Rockville. I think we will have strategies like we always do to sort of mitigate different potential outcomes from that but I think the essence is of us hitting that re-guided point is going to be relying on the great capabilities we have here to make that both drug supply get it fill wouldn’t get it prepared for patients that will be done under and sort of as part of the IND that was filed at the end of last year. And then we announced it was safe to proceed early this year. As we continue to make progress on that and get closer to that next milestone we’ll update something in addition to that first half of the year guidance, but this is a recent and unexpected event for us at this stage. So, this is where we are guiding right now.

All right. That’s it from us. Thanks for taking the question.

Certainly. [Operator Instructions] Our next question comes from Andreas Argyrides of Wedbush Securities, your line is open.

Yes. Good afternoon, and thanks for taking your question. We’ve also got one on 202. You guys have an abstract that ASGCT that talks about the recruitment of [Indiscernible] so just a quick question as to how 202 may differentiate from other competitors on targeting and mass binding domain. Thanks.

Yes, thanks, Andreas. I’m glad you raised that. We should have brought the R&D team to this call, apparently. But I’ll try to fill these. We’ve talked about the fact that we think that the C-terminal domain and other elements of the RGX-202 construct which again was selected from sort of a multiyear effort on the part of our scientists to select a candidate that was differentiated based on science, based on manufacturability, based on safety. But as we got closer and closer to selecting the final candidate that became RGX-202, we were looking at the entirety of things that contributed to improvements in the structural integrity of the truncated dystrophin in its function, as well as the ability of the biology to recruit things, including enough but not limited to, I think, in that abstract and another circumstances we have published data that shows that there are other aspects of the dystrophin associated protein complex that we view are being recruited or essential to have the C-terminal domain as part of the expressed protein in order to recruit and stabilize that complex. And so we think that [Indiscernible] among them, which we know has been something that has been talked about as part of, for instance, the construct that we know solid biosciences is brought into the clinic. Our view is that is a good direction to go and always has been making improvements to the science, the biology of the C-terminal domain components of dystrophin has been a key component for us of evaluation and candidate selection.

Great, and congrats on going live at Rockville. It’s very cool. Thanks.

And our next question comes from Danielle [Indiscernible] your line is open.

Hi and thank you all for taking the question. I have one on 202 really related as well. The manufacturing parts are for 202. Is that the news for any of the other programs? And if so, is there a chance that any of those other programs could be affected?

And the third party manufacturer for this program is not a group that we use for any of the other programs that REGENXBIO was part of our pipeline.

Okay, thank you.

I’m showing no further questions at this time. I would now like to turn the conference back to Mr. Ken Mills for closing remark.

Thanks, operator again just want to reiterate. Thanks, everyone. For the time today continue to be excited for the field of gene therapy for the progress we’re making here at REGENXBIO and the capabilities we have to help as many patients as possible. So thanks for everyone’s questions and support and we’ll look forward to seeing you all soon. Have a good day.

This concludes today’s conference. Thank you for participating. You may now disconnect.