Regenxbio Inc (RGNX) 2021 Q3 法說會逐字稿

Good afternoon, and welcome to the REGENXBIO Third Quarter 2021 Earnings Conference Call. (Operator Instructions) 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.

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 in 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 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, 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 financial results for the third quarter of 2021. At the end of the call, we will open up the line for question.

To start, I'm very excited about our recent news of our partnership with AbbVie to develop and commercialize RGX-314, our onetime 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 onetime 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.

Thanks, Ken. We continue to enroll patients in the atmosphere, the first of 2 pivotal plan trials to evaluate efficacy and safety of the subretinal 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 II 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 6 months after onetime 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 RGX-314 administration.

Looking at the safety profile, RGX-314 was reported to be well tolerated across 50 patients dosed in Cohorts 1 through 3 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 RGX-314.

We also announced the expansion of the AAVIATE trial to enroll patients in 2 additional cohorts to evaluate a third dose level of 1E12 genome copies per eye. Cohort 4 will enroll 15 patients who will receive RGX-314 at this dose level and Cohort 5 will evaluate RGX-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 6-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 II trial to evaluate RGX-314 for the treatment of diabetic retinopathy or DR using suprachoroidal delivery at the ASRS -- excuse me -- 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 1/3 of DR patients in the United States have moderate to severe NPDR without DME, equal to about 3 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 onetime 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 dosed with RGX-314 and Cohort 1 demonstrated a 2-step or greater improvement from baseline on the diabetic retinopathy severity scale, or DRSS, at 3 months compared to 0 of the 5 patients of the observational control arm. Of note, one RGX-314 treated patient had a 4-step improvement.

We're encouraged to see these results at this early time point of 3 months after the onetime administration. In the ALTITUDE trial, dosing of patients at the second dose level in 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 onetime gene therapy for the treatment of Duchenne by year-end 2021. From our ongoing Phase I/II trial of RGX-121 in MPS II patients up to 5 years old, we continue to enroll patients and today announced that we have expanded Cohort 3 to include up to 6 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 I/II 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 our 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.

Thanks, Steve, for the good update, 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 is still on track to be fully operational in the first half of 2022 and is expected to allow for production of NAV vectors at scales up to 2,000 liters using our platform suspension cell culture process. We believe our integrated approach of having manufacturing suites on-site will allow for more efficient development and manufacturing of our product candidates.

I want to transition for 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 2 important consortium. 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 our new collaborators to advance gene therapies on behalf of patients and families in need.

Today, in collaboration with our friends at 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 aimed 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 quarter, we're pleased to see meaningful progress from our licensees across AAV-based gene therapies 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?

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 in 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 9 months ended September 20, 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, including the effect or excluding -- pardon me -- 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.

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 one fact 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. We dose 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 Hurler syndrome trials.

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

(Operator Instructions) Your first question is from Gena Wang of Barclays.

I have 4 very quick questions. So the first one is how transparent and the 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 a slightly different inflammation rate. What could be the reason causing these differences? My third question is regarding your Cohort 2 AAO update. Will we see western blot protein data and also longer follow-up safety data from Cohort 1? My last question, sorry, is for -- you're adding Cohort 4 and 5 for suprachoroidal 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?

Gena, this is Ken. Thank you for the question. We are in a process of regulatory review of the transaction in 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?

Sure. Gena, Steve here. Yes. As you mentioned, we're 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 suprachoroidal in 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 and 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.

Your next question is from Mani Foroohar from SVB Leerink.

I guess I have a little more of a broad-based question, which is a follow-up on some of what Gena brought up. So it's true outside of the indications defined thus far, is there avenue to expand the collaboration ophthalmology into other indications or is this specific degenerative vision loss focus of the partnership what you see is the primary focus of the AbbVie partnership? Or is there interest to potentially expand into rare ophthalmological diseases, et cetera?

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 in 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 it 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.

Your next question is from Vikram Purohit of Morgan Stanley.

So 2 from my side on 2 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?

Absolutely. Thanks Vikram. 2 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 subretinal 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 what age-related macular degeneration. I think we envision 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 onetime 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 real wide opportunity that I think you really can only approach with a onetime 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 the 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 onetime 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.

Yes. I think to expand on that in the actual ophthalmologist's office, these patients with moderate NPDR to severe NPDR and even mild PDR are showing up in droves really around the country and around the world really. And we know that repeated anti-VEGFs can actually stay 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 onetime treatment to provide the sustained anti-VEGF and assess that for potential benefit in these patients.

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 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.

Your next question is from Esther Rajavelu of UBS.

Congrats on all the progress. I guess a couple of follow-up questions with some of the prior questions that were asked. First, given the collaboration with AbbVie and some of the early data you've shared, can you please give us a sense for how you're now prioritizing the subretinal 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 landscape of available medication?

Esther, thanks for the questions. We continue to expand and press forward with respect to the RGX-314 program on both the subretinal and suprachoroidal fronts. We have meaningful data from years of study of RGX-314 delivered subretinally that feeds into our pivotal plan where we're executing on the plan of 2 pivotal trials to support a potential BLA filing in 2024 of the subretinal procedure. Still believe that a onetime gene therapy using the subretinal 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 2 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 it's 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 onetime 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.

And your next question is from Dane Leone of Raymond James.

Just I guess 2 quick ones from me. At the upcoming AAO meeting for RGX-314 where you'll present Cohort 2 and wet AMD, can you just clarify if we're going to get time course on slit-lamp observation for any patients that do have sell grade observation of inflammation to maybe just avoid some of the discourse around the last update? And then secondly, maybe just a fine 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 in how your approach might be different.

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 will 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 filed 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 enrolled in our study on the basis of pre-existing 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 to the potential of making this a therapy that could be available for patients. Thanks for bringing that up.

(Operator Instructions) Next question is from Luca Issi of RBC Capital.

Congrats on all the progress. I have 2. One on diabetic retinopathy, the other one on DMD. So diabetic retinopathy, I think you showed impressive changing in DRSS actually on par with PANORAMA and RIDE. However, I think PANORAMA and RIDE also show 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. 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 in exon, 13, 29 and 3?

So 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 so 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 BAA 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.

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 a 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 weave 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 we've been able to obviously take inventory of things that are going on out in front of us and 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 announce 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.

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.

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

And this concludes today's conference call. Thank you all for participating. You may now disconnect.