Q3 2021 Regenxbio Inc Earnings Call
Good afternoon, and welcome to the <unk> 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 <unk> you may begin.
Good afternoon, and thank you for joining us today with US today are Ken Mills, <unk>, President and Chief Executive Officer Dr.
Dr. Steve Nicola, our Chief Medical Officer, and beat the system, our Chief Financial Officer.
Earlier this afternoon, <unk> released financial and operating results for the third quarter ended September 32021.
The press release reporting our financial results is available on our website at Www Dot <unk> Dot 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 analysis sections of <unk>.
<unk> annual report on Form 10-K for the full year ended December 31, 2020, and comparable risk factors sections of <unk> 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 <unk> 2021, and we undertake no obligation to update any forward looking statements. We may make on this call account, 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.
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 Eyecare collaboration with Abbvie for the development and commercialization of Rdx three months.
Steve will then provide an update on our clinical programs and it will provide an overview of financial results for the third quarter of 2021.
At the end of the call will open up the line for questions.
Sorry.
I am very excited about our recent news of our partnership with Abbvie to develop and commercialize <unk> hundred one four or one time gene therapy for the treatment of wet age related macular degeneration, diabetic retinopathy and other chronic eye disorders.
We believe Abbvie has a strong complementary partner for <unk> bio and we plan to leverage their commercial infrastructure and leadership in high care with our expertise in AAV gene therapy clinical development and are keeping in house knowledge of manufacturing and production.
We believe this is.
Important partnership that could expand the impact of <unk> 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 <unk> 314 for the treatment of wet AMD and diabetic retinopathy using in office supercritical delivery.
We're pleased with the early clinical profile around Super Choroidal delivery looking ahead, we plan to report additional interim data from our phase II trial in wet AMD using Super Choroidal 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 <unk>, one and <unk> 111 for the treatment of Hunter syndrome, and Hurler syndrome, respectively, as well as Rdx 202 for the treatment of Duchenne muscular.
<unk> dystrophy.
I wanted to take this moment to thank our <unk> 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 are 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 and the.
The first of two pivotal plan trial to evaluate efficacy and safety of the sub retinal delivery of <unk> 314 for the treatment of wet AMD we.
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 <unk> 314 from the phase two 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 Super Choroidal space of the eye and <unk>.
Michael trial.
At six months after a onetime administration of <unk> 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 mean annualized injection rate during the 12 months prior.
Here to <unk> 314 administration.
Looking at the safety profile, our JAK $3, 14, which are reported to be well tolerated across 50 patients dosing cohorts one through three as of September 13th 21 2021.
Among patients in cohort one common treatment emergent adverse events in the study were generally mild and none were severe mild <unk> location was observed in four out of 15 patients in all cases were resolved within days to weeks on topical corticosteroids, which have been discontinued these.
Cases of inflammation were asymptomatic and observed a slit lamp examination.
And it's important to remember that these patients were not receiving prophylactic steroids before after administration of <unk>.
We also announced the expansion of the AAV trial to enroll patients in two additional cohorts to evaluate a third dose level of one <unk> genome copies per eye.
Cohort four will enroll <unk> patients, who will receive <unk> 314 at this dose level and cohort five will evaluate our gen. III 14 at the same dose level cohorts four 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 two will be presented at the upcoming <unk> conference in mid November.
We also presented positive initial data from our altitude trial, which is our phase II trial to evaluate <unk> $3 14 for the treatment of diabetic retinopathy or Dr. Using Super choroidal delivery at.
The ASR.
Srs conference in mid October.
Dr can start in young adulthood, and often progresses quickly leading to vision, threatening complications, including diabetic macular edema, or <unk> and Neovascular ization that can lead to vision loss.
Estimated that one third of Dr patients in the United States have moderate to severe and PDR without equal.
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.
Most patients with NPR 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 neovascular ization that can lead to blindness.
We believe that onetime treatment with <unk> 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.
And the altitude trial as of September 29, our Gen III teen.
<unk> was reported to be well tolerated and 15 patients dosed in cohort, one and no intra ocular inflammation was observed on slit lamp examination.
33% of patients dosed with <unk> hundred 14 in cohort one demonstrated a two step or greater improvement from baseline on the diabetic retinopathy severity scale or DRA SaaS at three months compared to zero of the five patients of the observational control arm.
Of note <unk> III <unk> treated patients had a four step improvement.
We're encouraged to see these results at this early time point of three months after the onetime administration.
And the altitude trial dosing of patients in the second dose level cohorts, two and three is ongoing and we look forward to providing additional updates from this trial next year.
We believe the preliminary safety profile from our aviate, an altitude trials support continued development of the Super Choroidal route of delivery of <unk> three 2014.
Shifting focus to our rare genetic disease programs. Our team is on track to submit an investigational new drug application or <unk> to the FDA for <unk>, a potential onetime gene therapy for the treatment of Duchenne by year end 2021.
From our ongoing phase one two trial of <unk> hundred 21 in NPS to patients up to five years old we continue to enroll patients and today announced that we have expanded cohort three 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 two of the phase one two trial of <unk> hundred 11 for the treatment of NPS, one 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 <unk> 181 program for the treatment of <unk> disease, and we plan to provide an update in 2022.
We are also conducting additional preclinical studies of our Gen. Three one for the treatment of ocular manifestations of <unk> 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'll turn the call back over to Ken.
Thanks.
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.
It scales up to 2000 leaders 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 wanted 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 consortium as.
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.
Our multi stakeholder initiative, which seeks to bring together a broad and diverse group from the rare disease in AAV gene therapy communities for meaningful scientific and policy discussion paths.
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 <unk> bio 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 collaborations.
This consortium will support a series of research product projects aimed to create new tools and resources for <unk> clinical development and regulatory evaluation of future <unk> 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 are pleased to see meaningful progress from our licensees across AEG AAV based gene therapies for rare diseases, using our NAV technology platform, including updates about recent studies from ultra <unk> and the Novartis team.
With those updates I will now turn the call over to Bill for a review of our last quarter financials.
Thank you Ken <unk> in the quarter on September 30 of 2021 with cash cash equivalents and marketable securities totaling $533 5 million compared to $522 $5 million.
December 31, 2020, the increase was primarily due to the $216 1 million dollar of aggregate net proceeds received from our follow on public offering of common stock completed in January 2021, including the full exercise of the underwriters option to purchase.
As 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 both reservoir royalties paid.
Healthcare royalty management of 33.
$3 million.
During the nine months ended September 20th 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 32021 to fund our operations, including the completion.
Our internal manufacturing capabilities and clinical advancement of our product candidates into the second half of 2023.
Including the effect.
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.
Faction of customary closing conditions, including applicable regulatory approvals with that I will turn the call back to Ken for all final bonds.
Thanks for that good summary.
I was reflecting this past quarter on the impact of <unk> 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 600 patients with <unk>.
And that the totality of our ongoing clinical trials using NAV technology is abundant.
Reasonable to assume that each day patients and families are receiving gene therapy treatments or product candidates based on the <unk> bio technology.
This past year, we've made substantial progress advancing multiple development programs inside the company, including for our lead product candidate <unk> hundred one four.
We continue to drive a number of internal development programs using NAV vectors for rare genetic diseases.
We've dosed 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 and I'm excited about our upcoming milestones, including our update at <unk>.
The Duchenne IND filing and next year's data updates from our Hunter and Hurler syndrome trials.
With that.
Ill turn the call over for questions operator.
Thank you Sir.
Our audio attendance if you would like to register a question. Please press star followed by the number one on your telephone keypad.
If your question has been answered and you would like to withdraw your registration. Please press in the past.
One moment for your first question.
Yes first question is from Gena Wang of Barclays. Your line is now open.
Thank you for taking my questions I have for very quick questions.
The first one is how chance power and then the real time.
You win sharing data with Abbvie.
Then my second question is that we.
So the first cohort typically though data in booths that that.
Well I cannot see in wet AMD and we saw.
Slightly different inflammation rate, what could be the reason, causing desk differences Mike.
My third question is regarding your cohorts.
We see western blot protein data.
Also longer follow up safety data from cohort one.
My last question sorry is for you Adam.
Adding <unk> five possible credo.
The trial is that because you don't expect to see optimal efficacy from cohorts three and four or is it because safety from cohorts three and four make you confident to further dose escalate.
Hi, Dana this.
This is Ken Thank you for the question.
We.
Our in process of regulatory review of the transaction in collaboration with Abbvie.
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 in 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 the three one for Steve do you have.
The ability to compile some quick responses there sure hygiene Steve here.
Yeah as you mentioned.
We're excited that we were able to have initial data come out from initial cohorts for both the wet AMD and Dr study.
And.
There has been theoretical discussion people have had about weather different populations may or it may not be more prone to and planetary response too.
Different treatments.
Our 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 and wanted to seeds or the other given all of the data that's out there in <unk> 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.
Supercoil in office approach and have completed dosing in the first cohort.
In both studies advancing to a higher dose level in both NAV negative and now NAV positive patients.
And Eva having completed dose level two enrollment.
For both NAV.
Negative not positive being able to go up to a third dose level in still NAV negative in that 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.
Sure.
At the tail end of your question was really a key aspect that yes, we are comfortable to do that and can and comfortable to do that with out prophylactic steroids.
When developing.
The drugs at this early stage visit 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 too.
Take advantage of that and continue to explore.
Dose response potentially.
88 study.
Thanks, Steve.
Your next question is from Manny flaw.
SBB Leerink your line is now open.
Hi, Thanks for taking my question I guess I have a little more of a broad based question was a follow up on some of the genome brought up.
Outside of our.
Outside of the indications defined as far as their avenue to expand the collaboration ophthalmology into other indications.
Or are there specific degenerative.
Vision loss.
Focus of the partnership.
What you see as the primary focus of the Abbvie partnership.
Or is there interest to potentially expand into rare layered ophthalmologists.
Tara.
Yeah.
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 Rdx 3144.
Sure.
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 the mechanism of action associated with that Jeff and ambitions. So the relationship between us and Abbvie through this collaboration is focused in eyecare, an anti VEGF indications, where obviously we have already.
Presented data and demonstrated.
Evidence of pharmacology and safety effects with 304 in wet AMD and Dr. We're looking forward to broadening the scope of that program as we continue to advance.
It would not transition to be direct about your question, we wouldn't expect that the scope of this collaboration as it exist would transition into non anti VEGF indications at this time.
Alright, Thanks, guys Thats helpful.
Yeah.
Your next question is from Vikram for Rohit.
Of Morgan Stanley. Your line is now open.
Great. Thanks for taking my questions.
So two from my side on <unk>.
Two different topics so first.
You touched on this a little bit in your prepared remarks, but I was wondering if you could.
<unk> on your current thoughts now that you've had some data in this space.
The commercial opportunity for 301, four in diabetic retinopathy in.
Which specific patient profiles, you think would be best suited for Super credit delivery in this disease setting and then secondly, and separately for your efforts in <unk> 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 their progress.
<unk> product candidate.
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 Mani here. What we are focused on with respect to <unk> hundred one four and the commercial opportunity is where sub retinal can provide a solution for patients in wet AMD, we broadened the spectrum of opportunity here by studying the <unk>.
<unk> approach.
The diabetic population and starting with the population of patients that have <unk>.
Diabetic retinopathy diagnosis, but without <unk>.
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 envision that other sort of label known anti VEGF indications would be part of the commercial potential here.
There is no 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 or data on early here with respect to the first study that we've designed.
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 our commercial opportunity in diabetic retinopathy is 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 intra ocular 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.
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 office these patients with moderate and PDR to severe and PDR, even mild PDR.
Are showing up in <unk> and.
And drove really around.
The country and around the world really and we know that repeated anti bad jobs.
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 assign the patients up for that either at that stage, where they've developed this 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.
Non surgical onetime treatment.
To provide the sustained anti that Jeff and assess that for potential.
<unk> in these patients.
And with respect to your second question about ETE, we continue to conduct research and preclinical studies to evaluate the advancement of gene therapy candidate for ETE.
I think from where we're using all of our sort of normal variables and algorithms for.
Valuation of how to progress a potential clinical candidate forward there.
Very typical for us at this stage when we are still doing research and preclinical development.
Thanks.
Your next question is from <unk>.
Taylor of UBS. Your line is now open.
Hi, Thank you for taking my question and congrats on all the progress.
I guess a follow up question.
Question.
Given the collaboration with Abbvie.
Chad can you please give us a sense.
We are now prioritizing this tablet.
<unk>.
Olson.
And then more broadly.
Hey, Nathan.
Question.
Can you just help us understand the rationale and some of the considerations that went into selecting ETE is an area of R&D into Kevin.
Landscape.
<unk> medication.
Hi, Sir thanks for the question.
We continue to.
Expand.
The press forward with respect to the RG <unk> program on both the sub retinal and Super crew idle fronts, we have.
Meaningful data from years of study of <unk> hundred one for delivered sub readily that feeds into our pivotal plan, where we're executing on.
The plan of two pivotal trials to support 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 of safe profile for a potential drug candidate for the treatment of wet AMD as I mentioned the Super Choroidal in office delivery approach allows for two things changes the potential site of care and I think has the potential.
Pencil in wet AMD to expand the opportunity for patients to receive rdx three one floor with an alternative site of delivery of the different route of administration also is continuing to allow us to take our gx 314 into new and different anti VEGF indications, including diabetic retinopathy in.
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 our <unk> hundred one four.
When it comes to ETE.
<unk> talked about this when we announced the program the new origination of our interest in ETE here was about the use of and drawing on experience that we had in the expression of antibodies for instance, like in Rdx <unk> four where we've shown we can deliver a gene therapy to.
Express a therapeutic antibody.
Essence replace the need for chronic administration of treatment in that case of course, we are talking about supplanting repeat intra ocular inflammations converting patients over to a onetime treatment, where the gene therapy, then takes over and continuously expressing that therapeutic and that.
Our approach with respect to the announcement of our research work in <unk>.
We saw an opportunity where.
Therapies, we're coming to market and have established themselves in the market for the treatment of ETE that involve antibody based therapies that are delivered on a chronic basis and we think that gene therapy has an opportunity through our research. This would be obviously a different route of administration than intra ocular, but one that we can.
<unk> 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 Your line is now open.
Hi, Thanks for taking the questions.
Commentary on the updates.
Just I guess.
Two quick ones for me.
At the upcoming <unk>.
Meeting for our guests through four where you'll present cohort two in wet AMD.
Can you just clarify if we're going to get time course on split life observation for any patient that you have.
Celebrate observation of inflammation.
So maybe just to avoid some of the discourse around the last update.
Then.
Secondly.
Maybe just opine on the DMD program.
How the clinical strategy might differentiate versus disruptive program visor program, our solid programs.
We are obviously.
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.
Hi, Dan Thanks, a lot, where we've announced today, obviously that will be having an update at AAN, but we're not providing any more details at this point about what.
Composition of that presentation will be it'll be a podium presentation by one of our investigators plan.
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 from today.
This is a pretty short interval between our last update in the next podium presentation with our investigators that were 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 provide 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.
Think that other aspects that are unique to our program or 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.
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 cap set of course in our product profile and so the opportunity to have patients enrolled in our study on the basis of preexisting immunity or other inclusion exclusion criteria that may orbit.
Round, the biology of the AEP itself could provide us an opportunity for sort of unique enrollment here, so I think that.
We are 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 in <unk>.
Hopefully all the way to the potential of making this a therapy that could be available for patients.
Thanks for bringing that up.
A reminder, again if you would like to register a question. Please press star followed by the number one on your telephone keypad.
The next question is from Luca.
RBC capital your line is now open.
Great. Thanks, so much for taking my question Congrats on all the progress I have two one on diabetic retinopathy get at one on DMD. So in diabetic retinopathy I think you showed impressive changing in Drs actually on par with Panorama and right. However, I think Panorama ride also showed solid improvement in visual acuity by CVA even at.
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 youre thinking about implications for your program and maybe more specifically whether you are planning to exclude patients with mutation in exon 913, 2019 third thanks, so much.
So hi.
Hi, Luca Steve here I can take the Dr question. So.
So I think it's always important to take into account the context of that patient population. So.
Ryzen, right, where patients with diabetic macular edema. So there you expect to see visual acuity improvement.
Panorama was not.
Moderate to severe and PDR and there because you don't have background center involved.
You don't see a sizable change so.
Especially at an early time point so for US early time points, you really think of it more from a safety standpoint.
In a population like.
A higher risk and PDR and <unk> population.
So these patients see.
And so they're not symptomatic from a visual acuity standpoint, and you want to improve their diabetic retinopathy severity that prevent their risk of developing the sight threatening complications.
Okay.
And Luka 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 an announcement today about the pathway development consortium in collaboration with solid Biosciences.
It is a multi stakeholder initiatives.
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 academic.
Providers of services to the Duchenne community.
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.
<unk>.
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 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 <unk>.
Details of things like our inclusion and exclusion criteria. Once we announced that we have filed the IMD and can talk further detail about an improved 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 for the questions at this time. This concludes the Q&A session I would 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.
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