REGENXBIO inc (RGNX) Q3 2021 Earnings Call Transcript

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REGENXBIO inc (RGNX -5.40%)
Q3 2021 Earnings Call
Nov 2, 2021, 4:30 p.m. ET

Contents:

• Prepared Remarks
• Questions and Answers
• Call Participants

Prepared Remarks:

Operator

Good afternoon and welcome to the REGENXBIO Third Quarter 2021 Earnings Conference Call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be given at that time. As a reminder, this conference call is being recorded.

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

Patrick Christmas -- Chief Legal Officer

Good afternoon and thank you for joining us today. With us today 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 third quarter ended September 30, 2021. 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, 2020 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, November 2, 2021. 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 report 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?

Ken Mills -- President & Chief Executive Officer

Thank you, Patrick. Good afternoon, everyone and thanks for joining us. I'm pleased to begin today's call with the recap of our recent business highlights, including the strategic eye care collaboration with AbbVie for the development and commercialization of RGX-314. Steve will then provide an update on our clinical programs and Vit will provide an overview of finance results for the third quarter of 2021. At the end of the call, we will open up the line for questions.

To start, I'm very excited about our recent news of our partnership with AbbVie to develop and commercialize RGX-314, our one-time gene therapy for the treatment of wet age-related macular degeneration, diabetic retinopathy and other chronic eye disorders. We believe AbbVie is a strong and complementary partner for REGENXBIO and we plan to leverage their commercial infrastructure and leadership in eye care with our expertise in AAV gene therapy clinical development and our deep in-house knowledge of manufacturing and production. We believe this is an important partnership that could expand the impact of RGX-314 for millions of patients around the world. The transaction is expected to close by the end of 2021 subject to the satisfaction of customary closing conditions, including applicable regulatory approvals. Now Steve will shortly review our recently announced positive initial data from our ongoing Phase II trials of RGX-314 for the treatment of wet AMD and diabetic retinopathy using in-office suprachoroidal delivery. We're pleased with the early clinical profile around suprachoroidal delivery.

Looking ahead, we plan to report additional interim data from our Phase II trial in wet AMD using suprachoroidal delivery at the American Academy of Ophthalmology 2021 Annual Meeting later this month. We also continue our development of innovative gene therapy treatments beyond ophthalmology, including our CNS platform programs RGX-121 and RGX-111 for the treatment of Hunter syndrome and Hurler syndrome respectively, as well as RGX-202 for the treatment of Duchenne muscular dystrophy. I want to take this moment to thank our REGENXBIO team, our clinical investigators and the patient communities for their steadfast commitment to the development of innovative gene therapies. As we look forward to 2022, we believe we're well positioned to drive the development of potentially curative one-time gene therapies for patients.

And with that, I'm going to turn the call over to Steve for a bit more detail on the clinical and regulatory status of our program.

Steve Pakola -- Chief Medical Officer

Thanks, Ken. We continue to enroll patients in the atmosphere, the first of two pivotal planned trials to evaluate efficacy and safety of the sub-retinal delivery of RGX-314 for the treatment of wet AMD. We look forward to continued execution across this pivotal program, including the initiation of our second pivotal trial which we expect to take place later this year. We're encouraged by the emerging clinical profile of RGX-314 from the Phase 2 AAVIATE trial for the treatment of wet AMD which was announced at the Retina Society meeting at the beginning of October. This was the first ever data reported from a gene therapy delivered to the suprachoroidal space of the eye in a clinical trial. At six months after one time administration of RGX-314, patients at the first dose level were observed to have stable visual acuity and retinal thickness along with a 75.9% reduction in anti-VEGF treatment burden compared to the mean annualized injection rate during the 12 months prior to RGF-314 administration.

Looking at the safety profile, RGF-314 was reported to be well tolerated across 50 patients, dosing cohorts, one through three as of September 13, 2021. Among patients in Cohort 1 common treatment emergent adverse events in the study eye were generally mild and none were severe. Mild intraocular inflammation was observed in 4 out of 15 patients and all cases were resolved within days to weeks on topical corticosteroids which have been discontinued. These cases of inflammation were asymptomatic and observed on slit-lamp examination. And it's important to remember that these patients were not receiving prophylactic steroids before or after administration of RGF-314. We also announced the expansion of the AAVIATE trial to enroll patients two additional cohorts to evaluate a third dose of 1E12 genome copies per eye. Cohort 4 will enroll 15 patients who will receive RGF-314 at this dose level. And Cohort 5 will evaluate RGF-314 at the same dose level as Cohort 4 in 20 patients who are neutralizing antibody positive. As Ken mentioned, our next update from this trial which will include six-month data from Cohort 2, will be presented at the upcoming AAO conference in mid-November.

We also presented positive initial data from our ALTITUDE trial which is our Phase 2 trial to evaluate RGX-314 for the treatment of diabetic retinopathy or DR, using super choroidal delivery at the ASRS conference in mid-October. DR can start in young adulthood and often progresses quickly leading to vision threatening complications, including diabetic macular edema or DME and neovascularization that can lead to vision loss. It's estimated that one third of DR patients in the United States have moderate to severe NPDR without DME equal to about three million patients. There are treatment options for patients with DR, including chronic, repeated, anti-VEGF injections, retinal laser treatment and surgery. However, most patients with NPDR go untreated as the current standard of care is watchful waiting until vision becomes threatened. Unfortunately, without treatment, a large proportion of these patients will eventually develop vision threatening complications, including diabetic macular edema and neovascularization that can lead to blindness.

We believe that one time treatment with RGX-314 has the potential to provide sustainable, long-term, anti-VEGF protein production in the eye which could reduce the severity of DR and prevent vision threatening complications. In the ALTITUDE trial as of September 29, RGX-314 was reported to be well tolerated in the 15 patients dosed in Cohort 1 and no intraocular inflammation was observed on slit-lamp examination. 33% of patients dose with RGX-314 in Cohort 1, demonstrated a two-step or greater improvement from baseline on the diabetic retinopathy severity scale, or DRSS at three months compared to zero of the five patients of the observational control arm. Off note one RGX-314 treated patient had a four-step improvement. We're encouraged to see these results at this early time point of three months after the one-time administration. In the ALTITUDE trial dosing of patients at the second dose level and Cohorts 2 and 3 is ongoing. And we look forward to providing additional updates from this trial next year We believe the preliminary safety profile from our AAVIATE and ALTITUDE trials support continued development of the suprachoroidal route of delivery of RGX-314.

Shifting focus to our rare genetic disease programs. Our team is on track to submit an investigational new drug application or IND to the FDA for RGX-202, a potential one-time gene therapy for the treatment of Duchenne by year end 2021.

From our ongoing Phase 1/2 trial of RGX-121 in MPS II patients up to five years old, we continue to enroll patients. And today announced that we have expanded cohort 3 to include up to six additional patients. We expect to report additional data from this trial in the first half of 2022. Enrollment is ongoing in Cohort 2 of the Phase 1/2 trial of RGX-111 for the treatment of MPS I. And we expect to share initial data from this trial in the first half of 2022. We continue to evaluate the path forward for the RGX-181 program for the treatment of CLN2 disease. And we plan to provide an update in 2022. We are also conducting additional preclinical studies of RGX-381 for the treatment of ocular manifestations of CLN2 disease. And we are in discussions with regulatory agencies.

We plan to provide a program update in 2022. We have made meaningful progress across our entire portfolio of gene therapy candidates throughout 2021. And we look forward to building on this momentum for the remainder of this year and into 2022.

With that, I turn the call back over to Ken.

Ken Mills -- President & Chief Executive Officer

Thanks, Steve, for the good updates and thanks to the team for the good progress this past quarter.

As I mentioned on our last quarterly call, we've moved in and begun utilizing our new headquarters in Rockville, Maryland. Our internal GMP facility is located within this headquarters and it's still on track to be fully operational in the first half of 2022 and is expected to allow for production of NAV vectors at scaled up to 2,000 liters using our platform suspension cell culture process. We believe our integrated approach of having manufacturing suites onsite will allow for more efficient development and manufacturing of our product candidates.

I want to transition from and take a moment to talk about our commitment to improve lives through the curative potential of gene therapy, where we've recently supported the founding of two important consortiums. As a leader in the development of gene therapies, we take immense pride in being affiliated with both groups and look forward to working alongside or new collaborators to advance gene therapies on behalf of patients and families in need. Today, in collaboration with our Solid Biosciences, we announced the formal launch of the pathway development consortium, a multi-stakeholder initiative which seeks to bring together a broad and diverse group from the rare disease and AAV gene therapy communities for meaningful scientific and policy discussion. The pathway development consortium has the unique potential to bring together these diverse perspectives in the rare disease community with the interest of the patient at the forefront.

And last week, we announced that REGENXBIO joined the FDA, NIH, industry and not-for-profit partners in the formation of the Bespoke Gene Therapy Consortium with the aim of advancing the field of AAV gene therapy for rare diseases through additional collaboration. This consortium will support a series of research projects; aim to create new tools and resources for AAV clinical development and regulatory evaluation of future AAV therapies. And this consortium will also support clinical trials of new gene therapies for rare diseases to be conducted at the NIH. Additionally, this corner we're pleased to see meaningful progress from our licensees across AAV-based gene therapy for rare diseases using our NAV technology platform, including updates about recent studies from Ultragenyx and the Novartis team.

With those updates, I'll now turn the call over to Vit for a review of our last quarter financials. Vit?

Vit Vasista -- Chief Financial Officer

Thank you, Ken. REGENXBIO ended the quarter on September 30, 2021 with cash, cash equivalents and marketable securities totaling $533.5 million compared to $522.5 million as of December 31, 2020. The increase was primarily due to the $216.1 million of aggregate net proceeds received from our follow-on public offering of common stock completed in January 2021, including the full exercise of underwriters' option to purchase additional shares and connection with the offering. The increase was partially offset by net cash used in operating activities of $107.4 million, cash used to purchase property and equipment of $69.6 million and Zolgensma royalties paid to Healthcare Royalty Management of $33.3 million during the nine months ended September 30, 2021.

Based on our current operating plan, we expect the balance in cash, cash equivalents and marketable securities of $533.5 million as of September 30, 2021 to fund our operations, including the completion of our internal manufacturing capabilities and clinical advancement of our product candidates into the second half of 2023, excluding the effect of any potential payments that may be received under our collaboration with AbbVie which is expected to close by the end of 2021, subject to the satisfaction of customary closing conditions, including applicable regulatory approvals.

With that, I will turn the call back to Ken to provide final thoughts.

Ken Mills -- President & Chief Executive Officer

Thanks, Vit, for that good summary.

I was reflecting this past quarter on the impact of REGENXBIO's mission to improve lives through the curative potential of gene therapy. And once that became evident among the data that we've been seeing in the market including the fact that Novartis has treated over 1,600 patients with Zolgensma and that the totality of our ongoing clinical trials using NAV technology is abundant. It's reasonable to assume that each day patients and families are receiving gene therapy treatments, or product candidates based on the REGENXBIO technology. This past year we've made substantial progress advancing multiple development programs inside the company, including for our lead product candidate, RGX-314.

We continue to drive a number of internal development programs using NAV Vectors for rare genetic diseases. Lead those over 100 subjects in our clinical trials across our pipeline. I look forward to the continued progress across all of our programs in the coming months. I'm excited about our upcoming milestones, including our update at AAO, the Duchenne IND filing and next year's data updates from our Hunter and Horner Syndrome trials.

With that, we turn the call over for questions. Operator?

Questions and Answers:

Operator

Thank you. [Operator Instructions] Your first question is from Gena Wang of Barclays. Your line is now open.

Gena Wang -- Barclays -- Analyst

Thank you for taking my questions. I have four very quick questions. So, the first one is how transparent and a real time are you when sharing data with AbbVie? And then my second question is, we saw the first cohort suprachoroidal data in both diabetic retinopathy and wet AMD and we saw slightly different inflammation rate. What could be the reason calling this difference? My third question is regarding your Cohort 2 AAO update. We see western blot protein data and also longer follow up safety data from Cohort 1? My last question, sorry, is for, you adding Cohort 4 and 5 for suprachoroidal wet AMD trial, is that because you don't expect to see optimal efficacy from Cohort 3 and 4, or is it because safety from Cohort 3 and 4 make you confident to further dose escalate?

Ken Mills -- President & Chief Executive Officer

Hi. Gena, this is Ken. Thank you for the question. We are in a process of regulatory review of the transaction and collaboration with AbbVie. We stated at the time of the announcement that AbbVie had completed full diligence with respect to the process that led us to the announcement of the transaction and collaboration. And we can't comment any further on, anything else at this time but look forward to between now and the end of the year, the closing of that transaction. With respect to some of the points that related mostly I think to 314, Steve, do you have the ability to compile some quick responses there?

Steve Pakola -- Chief Medical Officer

Sure. Hi, Gena, Steve here. Yes. As you mentioned we are excited that we were able to have initial data come out from initial cohort for both the wet AMD and the DR study. And there has been theoretical discussion people have had about whether different populations may or may not be more prone to inflammatory response to different treatments. Our belief just based empirically based on a lot of data with different biologics is, there really isn't any solid evidence that you should expect to see a greater immune response to a treatment in one disease or the other, given all the data that's out there in DME and DR as well as wet AMD with various repeated injection biologics. We're just happy to be at a point where we can actually have data for our supercoiling office approach and have completed dosing. The first cohort in both studies advancing to a higher dose level in both Nab negative and now NAb positive patients. And in AAVIATE having completed dose Level 2 enrollment for both NAb negative, NAb positive being able to go up to a third dose level in still NAb negative and NAb positive patients and all the while continuing as we have been from the beginning without prophylactic steroids. So, one of your other questions was why go up to a third dose level? The tail end of your question was really a key aspect that yes, we are comfortable to do that and comfortable to do that without prophylactic steroids, when developing drugs at this early stage, this is the time to really evaluate potential dose response. So, for us, it's really a great opportunity given the safety and tolerability that we've seen to take advantage of that and continue to explore dose response potentially in the AAVIATE study.

Gena Wang -- Barclays -- Analyst

Good. Thanks, Steve.

Operator

Your next question is from Mani Foroohar with SVB Leerink. Your line is now open.

Mani Foroohar -- SVB Leerink -- Analyst

Thanks, taking the question. I guess I have a little more of a broad-based question which is a follow up on something that Gena brought up. So, shoot outside of the indication is defined thus far, is there an avenue to expand the collaboration ophthalmology into other indications or is this specific degenerative vision loss focus of the partner? What you see is the primary focus of the AbbVie partnership? Or is there interest to potentially expand into rare ophthalmological diseases, etcetera?

Ken Mills -- President & Chief Executive Officer

Hey. Mani thanks for the question and what we've announced is that the collaboration with AbbVie is focused on the development and commercialization of RGX-314 for wet AMD, diabetic retinopathy and other chronic retinal diseases. And I think that you can infer from that, that we're talking about things that fall in the category of a mechanism of action associated with VEGF inhibition. So, the relationship between us and AbbVie through this collaboration is focused in eye care and anti-VEGF indications where obviously we've already presented data and demonstrated evidence of pharmacology and safety effects with 314 and wet AMD and DR. We're looking forward to broadening the scope of that program as we continue to advance. But it would not transition to be direct about your question. We wouldn't expect that the scope of this collaboration as it exists would transition into non-anti-VEGF indications at this time.

Mani Foroohar -- SVB Leerink -- Analyst

Clear. Thanks, guys. That's helpful.

Operator

Your next question is from Vikram Purohit of Morgan Stanley. Your line is now open.

Vikram Purohit -- Morgan Stanley -- Analyst

Great. Thanks for taking my questions. So, two from my side on two different topics. So, first, you touched on this a little bit in your prepared remarks but I was wondering if you could expand on your current thoughts now that you've had some data in this space on the commercial opportunity for 314 in diabetic retinopathy. And which specific patient profiles you think would be best suited for suprachoroidal delivery in this disease setting? And then secondly and separately for your efforts in HAE, could you just mention to us where you are in your current work internally and what you would need to see in order to be able to nominate and then progress of product candidate here?

Ken Mills -- President & Chief Executive Officer

Absolutely. Thanks, Vikram, two questions. I'll start the first, Steve, maybe you want to jump in. I guess standing on top of the response to Mani here, what we are focused on with respect to RGX-314 and the commercial opportunity is where sub-retinal can provide a solution for patients in wet AMD. We've broadened the spectrum of opportunity here by studying the suprachoroidal approach in the diabetic population and starting with a population of patients that have diabetic retinopathy diagnosis but without evidence of macular edema. When I talked about being able to step into a broader spectrum of anti-VEGF indication potential, I think we would view that we could include patients with underlying conditions of diabetic retinopathy as well and also with diabetic macular edema, similar to what we're studying in wet age-related macular degeneration. I think we envisioned that other sort of labeled known anti-VEGF indications would be part of the commercial potential here. There is though I think a very unique profile in thinking about diabetic retinopathy patients that are early in their progression of disease and one that I think is uniquely suited for a one-time approach of gene therapy that we have brought forward and even presented evidence of data on early here with respect to the first study that we've designed in having an opportunity to access patients in clinics that have not begun to receive anti-VEGF therapy is really the top of the funnel in my view when it comes to the potential of a commercial opportunity in diabetic retinopathy. It's a realwide opportunity that I think-you really can only approach with a one-time therapy because being able to convert patients in that sort of status with therapy that would require repeat or chronic treatment, especially with intraocular injection, we know from evaluation of the market and discussion of opportunities in the clinic is really a challenge. But when you present physicians and patients and stakeholders with an opportunity to address, what ends up being a potential opportunity with a one-time treatment, it really can change the paradigm in terms of the standard of care for these patients. And I think Steve may have some perspective even into the details of what goes on clinically there.

Steve Pakola -- Chief Medical Officer

Yes, I think to expand on that or in the actual ophthalmologist office these patients with moderate NPDR to severe NPDR and even mild PDR are showing up and droves really around the country and around the world really. And we know that repeated anti-VEGFs can actually stave off progression to the vision threatening complications but these patients at that stage do not want to sign up for repeated injections indefinitely. And the treating physicians don't want to sign the patients up for that either at that stage before they've developed the site threatening complications. So really the-we know what works. The missing link is a sustainable treatment option. And that's really why, as Ken mentioned, this is a unique opportunity to have a nonsurgical one-time treatment to provide the sustained anti-VEGF and assess that for potential benefit in these patients.

Ken Mills -- President & Chief Executive Officer

And with respect to your second question about HAE, we continue to conduct research and preclinical studies to evaluate the advancement of a gene therapy candidate for HAE. We're-Vikram, we're using all of our sort of normal variables and algorithms for evaluation of how to progress a potential clinical candidate forward there, very typical for us at this stage when we're still doing research and preclinical development. Thanks.

Operator

Your next question is from Esther Rajavelu of UBS. Your line is now open.

Esther Rajavelu -- UBS -- Analyst

Hi, thank you for taking my question and congrats on all the progress. I guess a couple of follow-up questions to submit the prior questions that were asked. First, on given the collaboration with AbbVie and some of the early data you've shared, can you please give us a sense for how you are now prioritizing the sub-retinal versus the suprachoroidal for wet AMD patients? And then more broadly as a follow-up to the HAE question, can you just help us understand the rationale and some of the considerations that went into selecting HAE as an area of R&D interest given the current landscapes of available medications?

Ken Mills -- President & Chief Executive Officer

Hi Esther, thanks for the questions. We can continue to expand and press forward with respect to the RGX-314 program on both the sub-retinal and suprachoroidal fronts. We have meaningful data from years of study of RGX-314 delivered sub-retinal that feeds into our pivotal plan where we're executing on the plan of two pivotal trials to support a potential BLA filing in 2024 of the sub-retinal procedure. Still believe that a one-time gene therapy using the sub-retinal approach with the clinical evidence that we've presented over several years now supports a very strong profile, a safe profile for a potential drug candidate for the treatment of wet AMD. As I mentioned, the suprachoroidal in-office delivery approach allows for two things, it changes the potential site of care. And I think has the potential in wet AMD to expand the opportunity for patients to receive RGX-314 with an alternative site of delivery of the different route of administration, also is continuing to allow us to take RGX-314 into new and different anti-VEGF indications, including diabetic retinopathy and other chronic retinal diseases that are responsive to anti-VEGF therapy. So, we feel really good about the background of the pharmacology of the drug. And I think we've been really thoughtful about the fact that multiple routes of administration can help us achieve optimal outcomes from a clinical and potential commercial perspective here with RGX-314.

When it comes to HAE, we talked about this when we announced the program, the origination of our interest in HAE here was about the use of and drawing on experience that we had in the expression of antibodies, for instance, like in RGX-314, where we've shown that we can deliver a gene therapy to express a therapeutic antibody and in essence replace the need for chronic administration of treatment. In that case, of course, we're talking about supplanting repeat intraocular inflammations, converting patients over to a one-time treatment where the gene therapy then takes over and is continuously expressing that therapeutic. And that was our approach with respect to the announcement of our research work in HAE. We saw an opportunity where therapies were coming to market and have established themselves in the market for the treatment of HAE that involve antibody-based therapies that are delivered on a chronic basis. And we think that gene therapy has an opportunity through our research, now this would be obviously a different route of administration than intraocular. But one that we continue to study and have research focused on is the expression of therapeutic antibodies for the treatment of a variety of diseases.

Operator

And your next question is from Dane Leone of Raymond James. Your line is now open.

Dane Leone -- Raymond James -- Analyst

Hi, thanks for taking the questions and all the commentary on the updates. Just, I guess, two quick ones for me. At the upcoming AAO meeting for RGX-314, where you'll present Cohort 2 in wet AMD, can you just clarify if we're going to get time course on split life observation for any patients that do have cell grade observation of inflammation to maybe just avoid some of the discourse around the last update? And then secondly, maybe just a point on the DMD program and how the clinical strategy might differentiate versus the Sarepta program, Pfizer program or Solid programs which are obviously in the clinic and taking up some patients. So, any color there would probably be helpful to get more of an understanding and how your approach might be different. Thank you.

Ken Mills -- President & Chief Executive Officer

Hi Dane, thanks a lot. We've announced today, obviously that we'll be having an update at AAO but we're not providing any more details at this point about what the composition of that presentation will be. It'll be a podium presentation by one of our investigators planned and it's just a couple of weeks away. Keeping in mind that we only had an update, I think, what about a month ago, 30 days ago from today, this is a pretty short interval between our last updates and the next podium presentation with our investigators that we're excited to bring forward. When it comes to Duchenne, I guess, thanks for that question. We're excited to get the IND file by the end of this year, we've talked about the differentiating properties of the product candidate including the C terminal domain which we think provides some scientific differentiation in terms of the biology of a functional dystrophin gene delivering, the ability to recruit different types of proteins to the dystrophin associated complex, as well as provide some additional strength to the sort of cell membrane overall. I think that other aspects that are unique to our program are the status of our capabilities in terms of our manufacturing process, we're looking to bring a-of course, a scaled GMP process into the clinic from the very beginning of our initiation here. And we've been working to optimize that process in support of the BLA filing at the end of this year. So, we think that our clinical and regulatory strategy is strengthened by the opportunity to start with a process that we believe is in a position to carry us all the way through the clinical development. The other piece that I think is important to keep in mind is we're using a different capsid of course, in our product profile. And so, the opportunity to have patients enroll in our study on the basis of preexisting immunity or other inclusion, exclusion criteria that may orbit around the biology of the AAV itself could provide us an opportunity for sort of unique enrollment here. So, I think that, we're really committed to the approach that we're taking into the Duchenne community. We think we have differentiation coming into the clinic and we think that differentiation carries us through our clinical and regulatory strategy and hopefully all the way into the potential of making this a therapy that could be available for patients. Thanks for bringing that up.

Operator

[Operator Instructions] The next question is from Luca Issi of RBC Capital. Your line is now open.

Luca Issi -- RBC Capital -- Analyst

Great. Thanks so much for taking my question. Congrats on all the progress. I have two. One on diabetic retinopathy, the other one on DMD. So, in diabetic retinopathy, I think you showed impressive changing in DRSS actually on par with PANORAMA RIDE. However, I think PANORAMA RIDE also showed solid improvement in visual acuity by BCVA even at an earlier time point. And I don't think you have shown that data in ALTITUDE. So, wondering if you can elaborate a little bit more on that? And then, maybe on DMD we've seen this new safety signal from Pfizer. I'm wondering what was your reaction to that news? How you're thinking about implications for your program and maybe more specifically, whether you're planning to exclude patients in mutation [indecipherable]. Thanks so much.

Steve Pakola -- Chief Medical Officer

So, hi Luca. It's Steve, here. I can take the DR question. So, I think it's always important to take into account the context of the patient population. So, RISE and RIDE, were patients with diabetic macular edema. So, there you expect to see visual acuity improvement. PANORAMA was not moderate to severe NPDR and there, because you don't have background center involved DME, you don't see a sizable change. So especially at an early time point. So, for us early time points, you really think of BA more from a safety standpoint in a population like a high-risk NPDR and mild PDR population. So, these patients see fine. So, they're not symptomatic from a visual acuity standpoint and you want to improve their diabetic retinopathy severity to prevent their risk of developing the site threatening complications.

Ken Mills -- President & Chief Executive Officer

And Luca with respect to your question about Duchenne and some of the recent updates with respect to what's happening in the field, including in some ongoing clinical studies, I want to steer you and others back again to comment I made earlier about our formal launch and announcement today about the pathway development consortium in collaboration with Solid Biosciences. It's a multi-stakeholder initiative. It is also an initiative that is also rather already started some pre-competitive work specifically in the Duchenne community space. We've had workshops where we've included other industry sponsors, patient groups, the food and drug administration, academics, providers of services to the Duchenne community. And it's been important for us to leave ourselves into that as we've begun to plan the designs for our own clinical work and talk to people about what we think our product candidate can contribute. Talk about issues that are important to all of us as sponsors and stakeholders and share information across multiple different stakeholder fronts. I think for us, that means we've been able to obviously take inventory of things that are going on out in front of us in areas where we may be ahead with some of our own capabilities. We're able to share that with some of these stakeholders and perspectives as well. So, we'll be able to provide more updates on the details of things like our inclusion and exclusion criteria once we announced that we've filed the IND and can talk further detail about an approved clinical trial design which we expect again, the IND to be filed by the end of this year. Thanks for that question on Duchenne also.

Operator

And there are no further questions at this time. This concludes the Q&A session. I will now turn the call back to Ken Mills for closing remarks.

Ken Mills -- President & Chief Executive Officer

Thank you, operator. Thanks everyone for joining us today. Have a great rest of your evening.

Operator

[Operator Closing Remarks]

Duration: 44 minutes

Call participants:

Patrick Christmas -- Chief Legal Officer

Ken Mills -- President & Chief Executive Officer

Steve Pakola -- Chief Medical Officer

Vit Vasista -- Chief Financial Officer

Gena Wang -- Barclays -- Analyst

Mani Foroohar -- SVB Leerink -- Analyst

Vikram Purohit -- Morgan Stanley -- Analyst

Esther Rajavelu -- UBS -- Analyst

Dane Leone -- Raymond James -- Analyst

Luca Issi -- RBC Capital -- Analyst

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