Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics Second Quarter 2020 Earnings Call. At this time, As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, Manager, Investor Relations. Please go ahead.
Thank you, operator, and thank you all for joining today's call. Earlier today, we released our financial results for the second quarter 2020. The press release is available on our website at sarepta.com and our 10 Q was filed with the SEC earlier this afternoon. Joining us on the call today are Doug Ingram, Bo Kumbo, Ian Estepan, Doctor. Gilmore O'Neil and Doctor.
Louise Rodino Klapac. After our formal remarks, we'll open the call for Q And A. I'd like to note that during this call, we will be making a number of forward looking statements. Please take a moment to review our slide on the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties, many of which are beyond Sarepta's control.
Actual results could materially differ from these forward looking statements and any such risks can materially and adversely affect the business, the results of operations and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10 K and most recent quarterly report on Form 10 Q filed with the Securities And Exchange Commission as well as the company's other SEC filings. The company does not undertake any obligation to publicly state is forward looking statements, including any financial projections provided today based on subsequent events or circumstances. With that, let me turn the call over to our CEO, Doug Ingram who will provide an overview of our recent progress. Doug?
Thank you, Mary. Good afternoon, and thank you all for joining us for Sarepta Therapeutics second quarter 2020 investor conference call. As I have mentioned in the past, on March 13th this year, all but a small contingent of our workforce transitioned it to a virtual work environment. Notwithstanding this unusual approach, we are not merely remaining productive, but have arguably become even more efficient and effective as we advance our multi program, multi platform genetic medicine ambitions. As you will hear this afternoon, We have continued to serve the patient community with our approved therapies.
Have already achieved or remained on track for our many planned 2020 mile stems, expanded our 3 pronged approach to building our enduring gene therapy engine, And as we have generated additional evidence in 2020, continued to build confidence in our unique approach to gene therapy, is proving successful and highly differentiated. With that, let us review our performance. As we continue to serve keep them safe in this pandemic. I am pleased to report that our 2nd quarter net sales stand at 111 point $3,000,000, an 18% increase over the same quarter last year. As I mentioned in our last quarterly conference call, We withdrew our 2020 guidance in light of the potential impact of COVID-nineteen on clinic visits.
As you can see, that impact has thus far been modest. Given the uncertainties of the external environment, we are not ready yet to set full year guidance. However, consistent with what we have seen thus far in 2020, we anticipate that any continuing impact from COVID-nineteen will remain modest. Moving to our development programs. Let me begin by commenting on our RNA franchise.
In the second quarter, we completed our NDA submission for casimersen. Our RNA therapy built on our PMO platform and designed to treat the 8% of the Duchenne community, who are Exxon 45 amenable. The PDUFA date for casimersen should be in the first quarter of 2021. If we are successful in obtaining this approval, Tazimersen will be our 3rd FDA approved therapy, bringing the percentage of Duchenne community with available PMO therapies to nearly 30%. With a successful casimersen approval, we will have more than doubled the size of the treatable patient population since the Tuplarsen was first approved.
As successful as our PMO platform has been, we are not satisfied with the status quo. We have been working on our next generation of the PMO, which if successful, may profoundly improve the efficacy and convenience of our RNA technology. We are in our multi ascending dose study, called the MOMENTUM study for our peptide conjugated PMO or PPMO, and that is candidate 5051. Here, we are using a proprietary positively charged peptide to increase the first quarter of 2019. The dystrophin production.
Treating Duchenne is all about increasing the production of the structural shock absorptive. So if the PPMO candidates are able to increase exon skipping, and dystrophin production, we could see a profoundly more efficacious RNA platform and that monthly dosing versus the PMO weekly dosing, we would have a far more convenient therapy as well. In the second half of this year, we will have completed and will release our safety tissue exposure, PKPD and comparative exon skipping for our PPMO-five thousand and fifty one candidate, at 20 mgs per kg. This would be an important proof of concept for the therapy, not only within DMD, but more broadly as a potential RNA platform to treat diseases where our steric blocking RNA technology could provide therapeutic benefit. Let's move now to our gene therapy engine.
There are 3 pillars upon which we are building our gene therapy engine. 1st, our pipeline 2nd, manufacturing capacity and manufacturing expertise and third, advancing and improving the science and effectiveness of gene therapy itself. Over the course of 2020, we have not only stayed on track, by having successfully dosed a significant number of DMD and lgMD patients. And with the additional data we have generated already this year, We continue to build confidence consider the additional confirmatory data on our gene therapy platform that has been generated already this year. One of the most significant challenges the full body neuromuscular gene therapy is safely delivering the proper number of genome copies to cell nuclei.
In this regard, Sarepta's programs have shown good tolerability and exceptional expression at reasonable doses. Now you will recall that in 2018, we announced the results of Study 101, our first 4 DMD patients on SRP-nine thousand and one. Reporting a mean of 3.3 genome copies per nucleus, protein expression approaching normal as measured by Western blot, IF positive fibers and IF intensity, and that all patients improved significantly on each and every functional endpoint measured. On that basis, we commenced a placebo controlled trial for SRP 9001 in fourth quarter of 2018, using clinical material. Then in 2019, we announced the results from our 3 patient low dose cohort for SRP 9003 to treat LGMD2E.
This is important for our platform because SRP-nine thousand and SRP-nine thousand and three share much in common, including the design approach under the guidance of Doctor. Luis Rodino Klapac, the same viral vector, our 874 and the same promoter, the heavy chain MHCK7. We were pleased to report that even at a quarter of our SRP 900 dose, we had exceptional expression. As an example, over 50% of beta sarcoglycan positive fibers were achieved. Every child improved significantly on every functional measure at 9 months.
In 2020, we have generated additional consistent data and gained more experience with our constructs. In the second quarter, the 1 year results for Study 101 in Duchenne republished in JAMA Neurology showing that every boy in the study improved significantly on each and every function line point. At 1 year. In the same month, we announced the results of our second 3 patient cohort treated with SRP-nine thousand and three for LTMD using the same dose that we have used for SRP 9001 for Duchenne. We were very pleased to announce that with the increased dose, we yet again confirmed the ability of our construct to safely deliver gene therapy robustly.
The children in the study had 4.2 genome copies per nucleus on average. There was a significant dose dependent increase in expression with children achieving 62% of normal on Western blot, 72% of protein positive fibers, and 73% on intensity. Additionally, in our 41 patient placebo controlled study 102, using SRP-nine thousand and one for DMD, we had dosed all children for the main analysis in our dosing children on crossover as well. By now between our 2 studies for SRP-nine thousand and one and our study for SRP-nine thousand and three, We have dosed over 35 patients with active therapy. Far more than any similar clinical programs in these studies are proceeding at pace.
The second pillar of our gene therapy engine is our manufacturing expertise. And there, we have built impressively over the last 2 years. We have among the greatest capacity available for gene therapy in biotech in just 2 years between our dedicated suites of Catalent and our dedicated site in Lexington, with Thermo Fisher. At the same time, we have built out our centralized gene therapy manufacturing expertise within Sarepta. It is this group that drives the analytical and process development approaches across our programs and with our partners.
Across Burlington Andover, Cambridge, and our Gene Therapy Center of Excellence in Columbus, we now have some 270 professionals dedicated to technical operations, manufacturing and quality. For SRP 9001, we completed process development and analytical development and have completed GMP runs for the material we plan to use both for our next clinical trial and commercially. For SRP 9003, we're in GMP runs now. The 3rd pillar of our gene therapy engine is bringing together the technology and tools necessary to improve and advance the science and of gene therapy. We are a science driven company, and I am pleased to note that COVID-nineteen has done nothing to slow our progress in this endeavor.
Having built out 1 of the deepest and most valuable gene therapy pipelines commencing in 2017, and then spending an enormous effort in the last 2 plus years on building out our manufacturing prowess, commencing in 20 team, we began to aggressively look for opportunities to advance the science of gene therapy to complement our internal program development. In this quarter alone, we have added 4 new approaches to our armamentarium. In May, we announced our partnership with Dyno Therapeutics for the use of their AI and machine learning approach to develop improved capsids. From there, we entered into a partnership with Selective Biosciences to use their ImmTOR technology in an effort to empower Redosing. And then we entered into a partnership with Harnes at BioPharma to access Inlymphidase with the goal of using it to ablate preexisting neutralizing antibodies and to open gene therapy to patients that would otherwise be left behind.
And then from there, we entered into a relationship with Kodiak Biosciences to explore the use of exosomes across gene therapy, gene editing and RNA. Now looking forward, we have important gene therapy engine milestones across the remainder of 2020 first. With respect to SRP-nine thousand and one, we have 2 main year efforts ongoing. We must complete Study 102, our blinded placebo controlled trial using clinical Material. That trial is on track to have last patient last visit by the end of this year and to read out in the first quarter of 2021.
Secondly, with GMP, the commercial material now in hand, our goal is to start our next trial in the second half of this year. Our remaining gating items there are the following: 1st, engaging sites and obtaining IRB approvals. The pandemic has certainly created some challenges here, but the team is working through them and things are going quite well. And second, gaining alignment with the FDA on the initiation. Using the GMP material, which will start our pivotal trial in 2021.
Our 2 gating items here are these: first, completing assay development and obtaining the release of our GMP for SRP-nine thousand and three. Now this is an important dialogue as it will not only inform the development for SRP-nine thousand and three, but we believe We'll provide insight into the development pathway for our other 5 LGMD candidates. We will provide an update on the LGMD discussions and our GMP materials for SRP 9003 in the in early 2021. That, if you will indulge me on a personal note, the second quarter marked my 3 year anniversary at Sarepta. In three years, we have made an enormous amount of progress toward our goal of bringing a better, longer life to those living with and too often dying from rare disease.
And we have done so by building and articulating an ambitious vision to become the leader in rare disease genetic medicine with a robust and productive RNA platform and an enduring gene therapy engine. By gathering the resources and talent to make that a reality. And by then ruthlessly executing with a rapidity that to the extent possible, matches the urgency of the very patients that we serve patients who have their movement and their lives stolen from them bit by bit. Daily and hourly. In the last three years, we have built a pipeline with over 40 programs.
Are now in 10 clinical trials advancing multiple therapies, have made enormous progress in building from ideation to reality our 3 pillar gene therapy engine and are seeing consistent and confirming results thus far. As I reflect on our progress, the credit should go to 3 groups. The sophisticated, dedicated and hardworking professionals that make up our over 1000 strong workforce at Sarepta. Our external partners, in my opinion, the best and brightest in genetic medicine around the world. And of course, our patient community, a community that not only relies upon us, but also informs us and guides us and often pushes us.
So we are clear. Now is not yet the time for a victory lap or premature celebration. We are encouraged by our success so far, but we have much to do over the course of the next 15 months and still much more to prove. But you can count on this team, stay on mission, to address and remove obstacles that appear in front of us and to execute for patients. Waiting for our therapies.
And with that, I'll now turn the call over to Bo for a commercial update. Bo?
Thank you, Doug. Good afternoon, everyone. All our experienced teams at Sarepta are continuing to work through the headwinds of the COVID-nineteen pandemic. I'm pleased to report We've overcome some unique obstacles created by the pandemic and are extremely proud of the teams and their accomplishments in the face of these challenges. While we're still navigating and responding to the strain the pandemic is placed on the healthcare systems, the modifications we've made to our commercial execution strategy have allowed eligible patients with external partners have played a key role in the ability to start new patients to keep others on therapy.
This is a testament to our teams expertise and commitment to our mission to our patients and mitigate major treatment disruptions through a unified effort with our manufacturers, distributors, specialty pharmacies, healthcare providers and payers. Many of our patients are choosing not to delay or stop therapy, and we believe this is partly due to the fact that the majority of patients on EXONDYS 51 and BiONDYS 53 are receiving weekly infusions in their homes. We are currently working closely with healthcare providers and specialty pharmacy to transition additional patients to weekly home infusions since many clinics and hospitals still have restrictions in place. We have thoughtfully deployed measures to minimize the risk of transmitting COVID-nineteen which includes making personal protective equipment available to all our patients who have requested supplies This will help ensure that patients have access to protective equipment when nurses administer their weekly infusions at their homes. Patient safety remains our top priority and we will continue assessing any and all efforts that will patients to our commercial strategy, especially in places seeing surges and infections.
As noted last quarter, the dynamics of initiating treatment with EXONDYS 51 and BIONDYS 53 remain affected. Since many clinics are not seeing patients for normal in person appointments, This has resulted in fewer patients initiating treatment. Physicians typically want to monitor patients in the clinic for the 1st couple Fusions, and we've worked with key opinion leaders to find options for patients to safely initiate treatment with BioNTus 53 or EXONDYS 51 in their home or an alternative site rather than the clinic. This has provided physicians and patients a path to initiate care. Hospitals in each to state's ease restrictions and clinics resume normal operations.
From a reimbursement standpoint, our health care providers have become regarding the authorization and reauthorization process for our products. And due to the ongoing education to managed care organizations, reimbursement efforts have been productive. We are confident that over time, eligible patients will ultimately receive access to reimbursements for FiONDYS 53 and EXONDYS 51 and start therapy in the timely manner. We have successfully adapted on identifying patients amenable to EXOND 5153 skipping and driving prescriptions. We also continue to educate payer about the importance of patients starting and staying on therapy regardless of ambulation status age or gender.
We're encouraged by these efforts which have resulted in new start forms and new patients initiating treatment starts during the quarter. Moving on to Axon 45. In June, we announced the completion of our NDA submission for casimersen or SRP-four thousand and forty five. As we await FDA's response, our commercial and medical affairs teams preparing for another successful launch. The foundation of our plan will be tailored to reaching patients who are amenable to Exxon 45 Skippy, who represent another 8% of the Duchenne community.
We will leverage our knowledge and experience from the EXONDYS 51 3 launches to facilitate patient access to casimersen as quickly as possible. While we're very proud of the accomplishments of the team has made to date, we still have an immense amount of work these treatments. Expansions in these unprecedented times. As the global leader in precision genetic medicine. And with that, I will now turn the call over to Ian for an update on our financials.
Ian?
Thanks, Beau. Good afternoon, everyone. This afternoon's press release provided details for the second quarter of 2020 on a non GAAP basis as well as a GAAP basis. The press release is available on Sarepta's website. Please refer to our press release for a full reconciliation
of
GAAP to non GAAP financial results. Net product revenue for the second quarter of 2020 from our products EXONDYS 51 and VYONDYS 53 was $111,300,000 compared to $94,700,000 for EXONDYS 51 alone for the same period of 2019. The increase primarily reflects higher demand for our products. Collaboration revenue, which primarily relates to our collaboration arrangement with Roche. The co development costs under the Roche agreement totaled $8,900,000 for the second quarter and are included as a reduction to our R and D expenses.
On a GAAP basis, we reported a net loss of $150,800,000
$276,400,000
or $193,000,003.74 per basic and diluted share for the second quarter of 2020 2019, respectively. We reported a non GAAP net loss of $117,900,000 or $1.51 per basic and diluted share in the second quarter of 2020 compared to a non GAAP net loss of $61,200,000 or $0.83 per basic and diluted share in the second quarter of 2019. In the second quarter of 2020, we recorded approximately $13,300,000 in cost of sales compared $15.9 certain batches of EXONDYS 51 not meeting our quality specifications for the 3 months ended June 30, 2019. With no similar activity for we recorded $188,500,000 $113,300,000 in R and D expenses for the second quarter of 2020 and 2019, respectively, which is a year over year increase of $75,200,000. This increase is primarily related on a non GAAP basis, R and D expenses were $160,400,000 for the second quarter of 2020 compared to $87,500,000 for the same period of 2019, an increase of $72,900,000.
The year over year growth in non GAAP R and D expenses was driven primarily due to a continuing ramp up of our micro dystrophin program. Now turning to SG of expenses for the second quarter of 2020 2019, respectively, a year over year increase of $6,300,000. On a non GAAP basis, SG and A expenses were $55,100,000 for the second quarter of 2020, compared to $52,300,000 The year over year increase was driven by significant organizational growth and expansion that supported our commercial launch as well as our over 40 therapies in various stages of development across several therapeutic modalities. On a packed basis, We've recorded $12,400,000 in other expenses net for the second quarter of 2020 compared to 900,000 dollars in other expenses net for the same period of 2019. The unfavorable change primarily reflects interest expense on our debt facilities entered in December 2019.
We had approximately $2,100,000,000 in cash, cash equivalents and investment as of June 30, 2020. And with that, I'll turn the call over to Gilmore for an update on our research and development activities. Gilmar?
Thank you, Ian, and good afternoon, everyone. Doug has already shared the highlights from our most advanced gene therapy programs. I will thus focus my remarks on the progress of our RNA portfolio, which comprises 1 of the 3 platforms Sarepta uses to drive its precision genetic medicine therapeutic strategy I will begin If accepted and subsequently approved, we will have more than doubled the size of the treatment patient population since the TAPerson was first approved. Importantly, our post marketing requirement or PMR studies are progressing despite the challenges arising from the COVID-nineteen pandemic. In summary, the mission, 402 PMR study for a teperson is dosing, and we have enrolled the 1st cohort of patients.
The essence study of Golodirsen as casimersen is progressing. Thanks to the immense efforts of investigators, patients and their families, and our clinical operations teams, we have managed to minimize the disruptive impact of COVID-nineteen on the trial. Prior to the onset of the pandemic, we had already been moving many patient visits including for dosing to their homes. In recent months, we have significantly increased the number of patients using home visits. As we were accelerating plans for at home visits, we did initially see some patients missed doses and visits.
Nevertheless, now, I am pleased to say that the new mitigation has significantly improved the situation, and most patients are on track to death. All of our mitigation strategies comply with guidances from regulatory agencies around the world on the conduct of trials during the COVID-nineteen pandemic. Indeed, the pandemic usurps not only as a disruptor, but a catalyst to permanently change our approach to clinical trial execution. Thus, by not being complacent, we are confident that we can deal with and minimize the impacts on all of our future trials in the face 51 program for DRD specifically. Let me give you an update on the U.
S. Ambrid collaboration. In late April, we announced an early research collaboration with U. S. Amyrid that would exploit our PPMO technology as a potential therapeutic for COVID-nineteen.
The work is ongoing with U. S. Emirates and we have expanded the collaboration to work with the leading research group in Sweden. Early results support performing additional confirmatory experiments. Now turning to Sarepta's R and D strategy is to enhance tissue or muscle penetration of our PMO chemistry and thus enhance efficacy by increasing dystrophin expression.
We have a number of research programs that support this strategy. To remind you, our PPMO platform fuses a cell pantry peptide 2 PMO to enhance cellular and nuclear penetration. Our most advanced PPMO program is SOP-five thousand and fifty one. Our ongoing SRP-five thousand and fifty one, two zero one multi ascending dose trial named momentum is advancing. And all patients in the 20 mgkg cohort have been dosed.
COVID-nineteen related shutdowns caused one patient to miss a dose, But since then, with new mitigations in place, we are executing well. Just to remind you, we started the MOMENTUM study at 4 mgs per kg and have an escalation already to 20 mgkg. This represents dosing beyond that, which we originally anticipated. We plan to continue to dose escalate based on reviews of safety. And so far, we have not seen any safety signals.
This year, we will be reviewing 12 week data from our 20 week per key cohort of Duchenne patients treated with Pfemof-five thousand and fifty one. We will be examining systemic PK, tissue penetration, safety and exon skipping data. We will measure exon skipping by digital drop PCR or DEPCR, allowing us to directly compare the see our PMO and PPMO candidates. Our preclinical in vitro and in vivo models have demonstrated a robust correlation the amount of exon skipping and amount of dystrophin production. And we will cover shape these data soon.
Thus, exon skipping is a good marker for the kinds of dystrophin production we expect to see over time. It is important to note that while 12 weeks is an early time point to assess exon skipping, we are confident that this is an appropriate time point to demonstrate proof of concept that the cell penetration peptide or CPP will enhance muscle tissue exposures and thus enhance downstream exon skipping. This allows us the potential to move this technology forward with the urgency necessary to meet the needs of patients with Duchenne. It is also important to remember that dystrophin accumulates over time as we have observed in our PMO study. And so we expect to see higher levels of dystrophin at later time points if therapy is successful.
Muscle biopsies at later time point are planned in the latter part of the MOMENTUM study once we have selected our final dose. The readout for MOMENTUM will be important for the 5051 program dose selection, but additionally, we'll potentially extend to other PPMO Duchenne programs. We have already designed and selected 5 additional PPMO candidates. In fact, that could treat 50% of Duchenne patients who carry skip amenable mutations in their distrophin gene. I do want to highlight that rare deletions acceptable to exon skipping impacts an additional 35% of patients with Duchenne, and we are creating a development strategy that will attempt to accelerate the approval of PPMO compounds if successful for these patients using a novel platform or extrapolation strategy.
Proofa concept for PPMO in DMD or Duchenne will also read through to the selection of that chemistry to target new non muscle therapeutic areas. Non DXD and non muscle therapeutic area targeting is a key pillar of Sarepta's R and D strategy. In summary, I am very pleased with our response to the incredible challenges created by the COVID 19 pandemic and the progress we've made during this time. Finally, I want to thank all the patients, their families, study sites and coordinators, my R and D colleagues and our partners who have done so much work under incredibly difficult circumstances to maintain our urgent mission to deliver new, highly effective therapies to people with desperate diseases. Now I would hand back to Doug for Q And A.
Doug?
Thank you very much, Doctor. O'Neill. And Krystal, with that, let's open the call for questions. Thank
and
answer session.
And our first question comes from Tazeen Ahmad from Bank of America. Your line is open.
Good afternoon. Thanks so much for taking my question. Doug, I just wanted to get a little bit more color as it relates to the FDA discussion this quarter. Can you talk about some of the more important items that you'll need to get alignment on from the agency? And also, do you plan on asking them about plans attached to non ambulatory or older patients as well?
Thanks.
Thanks a lot for that, Tazeen. So let's review one of the things we had said earlier this year that we would have GMP material on hand by July of this year. And the good news is that we did So we've got 2 things to do. As you know, we've got to get our next study using commercial process material up and running. Getting patients dosed.
And we're going to do that, in the second half of this year. And the team notwithstanding COVID-nineteen 18 and the distractions that occur and some of the challenges occur with COVID-nineteen have done really remarkable jobs keeping on pace and moving to get that study started. But before we can do that, we have to have our meeting with the agency. And I said, in my prepared remarks, we will be meeting with the agency this quarter. And there really are just two things that there are significant things, but there are two things we need to get alignment and concurrence on with the agency.
The first of course is our CMC approach itself for the commercial material. If you use the material in the next study. We're very pleased at how these GMP runs have come out. Very, very confident in the approach that we're taking. From our perspective, there are no significant quality attributes that are different from the clinical material a manner that would be predicted to affect the therapy in any way functionally or efficacy wise or safety wise.
So that's one significant we need. And of course, the second one is to gain concurrence on the next study. Certainly, in connection with that, we'll we will have conversations with the agency about our non ambulatory study as well. It is one of our goals to commence the non ambulatory study as soon thereafter as is possible, perhaps even close to being concurrently with the next study, the main study that we'll be using if we're successful for the approval of the therapeutic in the United States.
Our next question comes from Salveen Richter from Goldman Sachs. Your line is open.
Good afternoon. Thanks for taking my question. If successful, how
do you see the PPMO platform fitting into the treatment paradigm versus PMO and gene therapy in DMD? And what are your plans for non DMD approaches on the forward?
Yes, that's a great question. I'm going to I'll answer that briefly and then I'll turn this over to Doctor. O'Neil, can you maybe talk about some of the areas we could take this RNA technology. So 1st and foremost, the excitement of the PPMO, if it is successful, is for a an even more profound impact from an RNA perspective with respect to Duchenne muscular dystrophy. Now of course, the question will immediately arise thereafter.
Of what is the how well gene therapy and RNA coexist? And the short answer to that is we don't yet know there will certainly be even with a transformative gene therapy, there will be, a place for a profound RNA for Duchenne muscular dystrophy in children who, for instance, are screened out for gene therapy in places where gene therapy is available or not yet available. So there will already be a significant place for it. And if one considers just the preexisting neutralizing antibodies alone, that is with respect to our capsid right now about 15% or so. And that population that would be screened out until we we've been able to address that issue scientifically.
That is larger than the eteplirsen population. But of course, with a significantly, more in taxable RNA platform, it may very well be the case that there is a role for a combination both of gene therapy and RNA. So first, we need to see what the PPMO will look like. And then we are doing work even as we speak right now from a non clinical perspective to test the high hypothesis about whether a profound gene therapy followed either in the near term or in the longer term with a profound RNA would be synergistically beneficial to patients. But beyond that, of course, if one of the things about our PMO is that the PMOs are neutrally charged.
It is creating phenotypic change in these children giving them longer time out of a wheelchair, etcetera. But to move beyond VMD, we need a more penetrative, RNA technology like the PPMO if it works. But if it does, the theory is that we could take this far beyond DMD and the research group is already working on the kinds of areas we would take that. But perhaps with that, I can turn this over to Doctor. O'Neal can touch on that issue.
Thanks very much, Doug. I think you actually articulated very nicely. The beauty of the RNA 10 technology and the PMO chemistry specifically is that we have proof of biology. We have demonstrated that we can deliver the PMO to, muscle and actually see downstream skipping and dystrophin of regulation or rather skipping and trophin ison form expression by returning it to in frame. With the PPMO experimentation with fifty-fifty 1, and the study readouts.
We hope to test the hypothesis that increased exposure driven by the cell pension type fusion, to the PMO when it has tissue exposure and bustle. What we actually also know is that will, extrapolate beyond just that muscle tissue to other tissues. And, as you're aware, many tissues are amenable and certainly, the PPMO and PMO have demonstrated tropism for other tissues, including liver, kidney, heart, etcetera. And we are actually doing a comprehensive genome driven review of those tissues and the associated diseases, to identify those that are amenable excellent skipping. So that is work that is ongoing and, I'm very excited about it.
And one final thing I will say moving back to DMD, just I think people know this, but I will remind you, we're currently using fifty-fifty 1 exon 51 PPMO as our proof of concept. And of course, later this year, we'll have some of the PKPD tissue dosing safety and exon skipping, importantly exon skipping for fifty-fifty 1 is our proof of concept. We have already built beyond fifty-fifty 1 constructs with the peptide conjugated to them for exons that could treat already about 50% of the Duchenne community, which path as Doctor. O'Neil mentioned in his opening remarks, when they passed to get to a significant percentage beyond that, perhaps another 35% beyond that with a a slightly more exotic, platform approach. And so, and we will start moving as rapidly as we can, assuming, of course, that the results are, support that when we look at them later this year.
Our next question comes from Anupam Rama from JP Morgan. Your line is open.
Hey guys, thanks so much for taking the question. We got a quick one here on we've gotten a lot of inbounds on 9001 safety recently. And I think some of it may revolve around some comments from Doctor. Mendell, maybe at ASPCT, that 9001 may be associated with some sort of mild complement activation. Any context you can provide here?
And just maybe remind us, of how frequently did DSMB meets for Study 2? Thanks so much.
Sure. I'm going to turn this question over to Luis who can answer this, but I will say upfront just so we're absolutely clear that that was a misunderstanding. There is absolutely no signal that we've seen of complement associated with 9001 at all. But I think Doctor. Irudino Klapac can provide the context around that misunderstanding.
Sure.
Yes. Doctor. Mendell, was speaking, that is broad experience in gene therapy and that includes other vectors like AV9. As Doug mentioned, we presented our data in both different medical meetings as well as investor calls on lgMD2E as well as our Gamma Neurology paper and just reiterating. We've not seen any evidence of complement activation in our studies, and Study 1 continues to, continues on as planned, no interruptions.
I think people were reading through comments he was making about other programs. This has been seen in a number of other programs primarily associated with the 89 wasn't speaking to R874. We've seen nothing relating to compliment there. And just to remind you, and of course, I know Doctor. O'Neill as a proper scientist we have to measure ourselves by the evidence on hand.
But the good news for us is that, with respect to Rx7 4 across both Duchenne Muscular District and Lynn Girdle, we have now dosed, as you heard in my opening remarks, over 35 children and the study 102 continues to move apace. So, I think the evidence on our broad safety profile, I think, is getting bolstered over the course of the last 24 months and really significantly this year.
Thank you. Our next question comes from Brian Skorney from Baird. Your line is open. Pardon me, sir. We're not able to hear you.
And we'll move on to our next caller, Matthew Harrison with Morgan Stanley.
Hi, this is Max Gore on for Matthew Harrison. Just a quick question. Based on the recent agreements you signed, how are you thinking about integrating these new So just to remind everybody, I think you heard in my opening remarks, we've done a number of really interesting transactions over the last quarter. To, to evolve the science and gene therapy as there's some you've got Dina, which is artificial intelligence and machine learning for making better capsids. We've got Kodiak which is an entirely different approach using exosomes for delivery device that you can use in RNA, gene therapy and gene editing.
And then I think But in more near term, perhaps, they're really impactful as both Hazza, within, with Inlymphidase and Selecta with ImmTOR, Hans' technology would ablate, preexisting neutralizing antibodies so that you could essentially empower at a minimum empower the people that would otherwise have preexisting neutralizing antibodies that they would have got environmentally, have them in frame for the ability to get gene therapy and of course select this is this concept of co administrating their ImmTOR technology with a gene therapy, so that you could essentially trick the body into believing that the gene therapy that it's receiving and the cat that it's receiving itself and not florence if you don't build up build up neutralizing antibodies, which could empower re dosing even multiple doses over time. The short answer on your question, however, is we've just entered into these agreements, these tools have gone into the arboretarium that Doctor. Rodino Klapac and her team have to advance this and we're looking carefully at the timelines and how we can move these as fast as possible forward. As you can imagine, as a mission driven organization, it is our goal to move these technologies as fast as possible and, improve gene therapy as much as possible more importantly bring the largest number patients into frame for gene therapy.
So we don't have timelines right now, but we're moving as fast as we can on that. And I suspect we'll have updates early next year on that.
Thank you. Our next question comes from Martin Auster from Credit Suisse.
Hi, everyone. This is Mark on for Marty. Thanks for taking my question. So my question relates to the upcoming readout for SRP 5051. I know that you framed the program relative to EXONDIS in terms of dystrophin expression.
I'm curious what is the level of Exxon skipping that EXONDIS achieves in humans And could you speak to the magnitude of improvement in exon skipping you're hoping to see with SRP-five thousand five hundred and fifty one as part of this initial clinical trial update? Thank you.
Sure. Doctor. O'Neill, you might want to take this and talk a little bit about what we'll be looking for in the second half and perhaps the way we would look at to compare it to what we might be seeing. With Eteplirsen and Golodirsen and Calendousin?
Yes. So I think the key thing is that we will be looking first of all, it's very important to understand that we are using, digital drop PCR, and we will actually be looking at that for PMO and PPO. It's important to actually distinguish that from, the older quantitative RT PCR that's been used in the past. And we will actually be looking at data from both PMO historic samples and then looking at our DDP CR in our fifty-fifty 1 program. I think the other point I want to make is that or restate is that we have actually with our non clinical data, both in vitro and cell lines, as well as in animal models at seen a robust correlation between DD, digital drop PCR and dystrophin levels.
And What we've actually seen there is a ratio of between 2 to 3. Obviously, we have to validate that in human, but what we've seen in our experimentation to date has been, how should I say, gives us confidence with regards to both the robustness of the correlation, the ratios and what we hope to see in our human studies.
Thank you. Our next question comes from Alethia Young from Cantor Fitzgerald. Your line is open.
Hey guys, thanks for taking my question and congrats on the quarter. I just wanted to
talk a little bit about kind
of hitting the GMP material and the good visibility seems like that's a big deal for you guys and want to talk about what that means going forward for this and other platforms of yours. And then just on Selecta and that Amtor I was just interested in Doctor. Klapac's perspective on like how to think about redosing and how this technology in hypothetically might be important in that. Thank you.
Okay. Before I turn it over to Luis to talk about select at all. So thank you, Alethia. I agree with you. It's a big deal.
I mean, and it's not a 1 quarter effort. The fact that we have GMP material on hand right now is the result of an enormous amount of work by a significant number of professionals over a long period of time. We started this really at ideation stage a couple of years ago that we were going to build out this gene therapy engine And we knew at that time that we needed to build out manufacturing, not simply for capacity, but also expertise process development, analytical development, etcetera. And we you've sort of lived with us as we've worked through that process optimized or in the process of optimizing process development, getting all of our analytical development done 24 assays involved here, all are complete either qualified or validated. And if the case is required for each of those, And then of course, we've done numerous runs in having GMP material on hand right now is enormously important.
And it will not I will also say, of course, we also have to meet with the agency. We're going to do that this quarter. We have to get them to concur in our view the material, etcetera, but we feel very good about where we are in the results that we've seen. And to your very good question, it's going it's going to pay significant dividends on our platform more broadly. As I said, and I think in my prepared remarks, this content of the gene therapy engine isn't simply a narrative.
It is a platform that is built on 3 significant pillars. All of which are required to build an enduring gene therapy platform. The first, of course, is that pipeline and we have a significant pipeline between research and things in the development stage and even in the later development stage, we have approaching 30 programs right now, at least, depending on how you count them. Then of course, that's the latter part I talked about is that Luis will talk to you about is the advancing the science, making gene therapy even more effective and bringing more patients in so they can get treated. And of course, that middle concept of manufacturing is crucial.
And I'm very proud of this team for having decided early on at the very ideation of this concept of building a gene therapy platform to begin to invest significantly both in the talent and beyond the talent in building manufacturing. And so the fact that we have GM material on hand right now speaks to Reims to 9001, but also 9003, also the rest of our limb girdles. And I believe beyond that into the other gene therapy programs that are sitting behind this program. So we're I don't want to I said in my opening remarks, it is so easy to become to get humorous when you have a little bit of success. And we don't want to do that.
We have a lot yet to do. I think meeting with the agency is a significant next step for us. But we're very excited about the progress to date. And we're very excited, frankly, about the results that we've received to date. Still have more results to come in.
We have to see 102 at the beginning of next year. But certainly, as you triangulate the results we're getting, both 101 than the 9 months on 101, then the start of 102 and the fact that we've been able to dose all of the kids for the main study. And then below those, on 9003 and the 9 months there. And then the low dose of 9003 and the successful 1 year there, then the 1 year on 101. And now of course, the high dose on 9003 and exceptional expression we're getting there and the good safety profile that we're seeing across all of the pro terms gives us some confidence that we're on the right track to be able to do what we're trying to do.
And what we're trying to do is simply something that will be commercially successful, but far more profoundly than that, we want to bring a much better life to these patients when verbal and to send patients. And then beyond that, who are living with and degenerating from. And as I said before, far too often, so coming to these rare genetic diseases. So thank you for that question. And with that, I apologize, I've been rambled for a second.
So I should turn this over Doctor. Rodino Klapac, he's going to talk to Select Amol.
Yes. Thank you for that question. So we're excited about both of both Hamza and Celexa. Hamza gives us an opportunity to, ablate antibodies in patients that are positive for AB antibodies. So that helps potentially get to those, 15% or so patients that have preexisting antibodies and also gives us the opportunity potentially for re dosing down the road.
Flexa, we're particularly excited about because it has the potential to prevent those antibodies from forming in the first place. So in with the first dose of gene therapy. Patients could be code administered this drug potentially and then prevent those antibodies from forming with the potential to redose down the road. So we just entered into these agreements and there's additional non clinical studies to be able to prove that these will be effective and safe. But we're definitely excited about the potential there.
We want to make sure that we have every every tool, to reach all patients and that's our goal. So we're just we may never need it, but it's better to be looking at it proactively and that's what we're doing. So thank you.
Thank you. Our next question comes from Tyler Van Buren from Piper Sandler. Your line is open.
Hey, good afternoon. I had just a quick follow-up question on the momentum trial. So You mentioned that you started at 4 mgs per kg and are up now to 20 mgs per kg. But if I'm not mistaken, you mentioned you could also potentially go higher than that. So based upon the animal model data, can you just define potentially a ceiling or a dose ceiling that you observed and what that was based on?
I'm assuming it was some sort of safety or toxicity.
Yes. Thank you for that question. So the short answer is that we are going to continue to so first understand the following. We are already above what we had, we would have considered success, at least from our preclinical models. Now please don't don't over read that.
We've got to get the data and we've got to see the biopsies and we've got to do the work. And so I don't want to oversell this, but I will say If you look from a preclinical prediction perspective, we're already beyond that point where we would have said we, we're very excited and this is great. We are going to continue to push that dose. We know what we're looking for and what it is is a real signal. That in preclinical models, that will be the dose limiting signal that will help us choose the dose.
Our goal, of course, is to achieve reasonably achieve the highest dose that is safe for these kids. And so we're going to continue to dose up until we get to a place where we think we've seen a signal that tells us that we're at the right margin between safety and efficacy and certainly, we're going to go to 30 mgs per kg this year. So you won't have a down on 30 mgs per kg. It won't come in time, but we'll have the 20 mgs per kg, which will be both certainly insightful for us. Is there anything there that I missed, Doctor.
O'Neill?
No, you nasal the target organ is the kitty, and that's what we're monitoring closely. And, it's been good so far.
Thank you. Our next question comes from Brian Abrahams from RBC Capital Markets.
Hey guys, thanks for taking my question and congrats on all the progress. With the full 1 year data for 9001 now published, I'm curious how this longer term data guides you with respect to conduct, patient selection endpoint selection for your next studies. Just when we look across some of the the positive results, but maybe a little bit of variability measured to measure in CK and function across the NSAA components as well as the learnings maybe there from the cardiac MRIs? Thanks.
Yes, I
would say broadly, one of the values of of Study 101 is that it allows us real time insight as we build out as we build out study 102. And then as we look forward to our commercial process trial as well. So we already had a significant amount of experience with 101 by the time we commenced of the primary, functional end pointing and SAA, we've had an opportunity to continue to monitor these children as you saw. And then we have the 1 year data, we have that reported out in JAMA Neurology. And I will tell you that it is confirming of the approach that we took with respect to Study 102, the way we've, chosen NSA and frankly the way we have powered that study.
So we feel very good about where we are and I think 101 has been very helpful, in helping us power that study and give confidence that we're on the right track.
Thank you. Our next question comes from Gena Wang from Barclays. Your line is open.
I have one question regarding TPMO has 3 parts So the first is just follow-up with the earlier question. Could you remind us the percentage of exoskipping for Xtandi51 in early animal data. And the second question is for the non muscle lung DMD program, what peptide you have in mind for the PPMO program? And lastly, do you see antibody oligo based therapy a threat to your key TMO franchise.
Yes, fine. Okay. So first of all, So the last question I'll answer now, and we're excited that other folks are looking at researching ways to bring better lives to Duchenne Muscular Dystrophy, but we certainly feel that in that our programs are the most advanced, and the most hopeful. So we're really focused on our own programs. Back to your other two questions, I will turn this over to Doctor.
O'Neil who can give you some perspective. But broadly speaking, and I'll let Doctor. O'Neil touch on this with more, expertise than me. First of all, know that the percentage of exon skipping you might look to historically with respect to the Tuplirsen, it won't be meaningful here because the approach to looking at excellent skipping is different. We're using digital drop PCR now in the past.
We used, a different form. I think it was over the RPC arm, but I thought Doctor. Emile will remind us of that. And so it's a bit apples to oranges, but good news, just so we know, is that when we when we announce the results of the 20 megs on the PPMO, we will have data using the same exon skipping, technology for a teplorsen. So there will be an ability to see what we might have seen with a teplorsen versus what we will see fifty-fifty one.
And then I believe I'll let Doctor. O'Neill confirm it that we would be using the same proprietary peptide that we're currently using that we put a lot of work into. But Doctor. O'Neill, you can correct me if I've said anything incorrect, yes?
No, that's correct. And I think, Gina, you asked, just make the restates. You're asking questions about peptide and the PMO skipping. I think I could do one better with regards to be stored than the animal in that we did actually, look at it at 24 weeks, actually over several week periods, but 24 weeks in the Permovi in humans in patients, we saw about 0 point 6% skipping that exosome, but it's very important to emphasize that's digital drop PCR. It's not the quantitative PCR, that was used in prior descriptions.
And that, I think, is disclosed in our MDA poster. So the beauty of the GD PCR is it's very tight and actually allows you then to compare, across patients and very importantly and more importantly to protein expression downstream. And then you asked other question about peptides. We've not disclosed the nature of the cell membrane peptide, but I think Doug I have already highlighted in this earnings call and in others that we continue to we're never complacent. We're never satisfied with the status quo.
And we continue to invest in exploring and optimizing, possibly better peptides and obviously our Kodiak collaboration that we announced is also part of an overall approach to both improve tissue penetration and, frankly, specific or target or tissue specific targeted.
Our next question comes from Joel Beatty from Citi. Your line is open.
This is Shawn Egan calling in for Joel. Thanks for the updates today and for taking my questions. Maybe could you talk a little bit about the package you plan to go to the FDA with in regards to the path forward for the Limon Girdle Muscular disease in general? I guess aside from the fact that these are mostly monogenic conditions, what other factors help make the case for an accelerated path to approval and what else should we be considering?
Yes. Thank you for that. Great question. So I think as Dan said in my opening remarks, We have 2 significant things to do over the course of this year. 1 is to complete the GMP runs, which is a combination of 2 things.
Is so we're clear. It's not simply the runs themselves, but it's of course all the assay work. We can leverage a lot of what we've done. I mean, an enormous amount of what we've done with 1, but there are bespoke assays for each particular program. So we've got to complete that work as well.
And the second thing to your good question is we've got we need to engage in a dialogue with the agency appropriate regulatory and development pathway forward. And one of the things we said is that we'll we will we've already started the commencing a dialogue there and we intend to have a before the end of this year on that issue as well. And to your point, why would we we are? And then I think we're unabashed about our view at least and we'll see where we end up. That, that it would be in the best interest of patients and also appropriate from a development and regulatory perspective to have a rapid an accelerated approach to the development of not only Limb girdle 2E, but also at a minimum, the other cervical eye cancer perhaps beyond that.
There really are a number of characteristics of this disease and this therapy that we at least believe, justify that to your point, these are well characterized genetic diseases. We know what is causing these diseases, and it's the lack of a structural protein that is the sole and exclusive reason that these children are degenerating and are living shortened lives. 2nd of all, these are ultra rare diseases. So these are as rare as Duchenne muscular dystrophy these are, in aggregate, limb girdle is significant. Individually, these are small disease states.
So that creates, a compelling reason to find a creative approach. This, the third reason, of course, is that this is the therapy, at hand because of the particular genes and proteins that issue here. We are able to comfortably package in our rh74 capsid, the gene that codes for the native protein. So we can replace the precise native protein that these children are lacking and the absence of which is causing their demise. And so the only remaining question then is can we produce a significant amount of it?
And the short answer is that we've seen in the both the low dose and the high dose that indeed we can. In the low dose, we were already producing a significant percentage. I might be offline percentage or 2, but on Western blot, I believe we were in the 30% and in on in positive fibers, we were over 50%. And then of course, as you've seen in the higher dose, it was well tolerated. And even as it's well tolerated, we're able to, we get 4.2 genome copies per nucleus.
The expression is over 70% on both protein positive fibers. And on intensity and nearly that on Western blot. And so from our perspective, this is you look at the guidance and you look at the statements that the agency has made publicly about, opportunities in gene therapy that seems on its face at least to be the kind of gene therapy that would justify, an accelerated approach to bringing these therapies to these kids? With that said, we have work to do over the course of this year, both in GMP and in dialogue with the agency to align on that. I won't make promises in advance about where we'll end up with but we certainly will come back at the beginning of next year and give you an update on those discussions with the agency.
And at that time, we hopefully, we can plan out exactly what the path is, not just for 2E, but for all of the other limb hurdles as well.
Thank you. Our next question comes from Vincent Chen from Bernstein. Your line is open.
Thanks for
taking the question and congrats on progress. Over the last few months, in addition to, I guess, your updated 1 year data for your DMD gene therapy program, I guess, we've also seen some updates from your competitor or peer Pfizer's micro dystrophin gene therapy program. And clearly there are different programs, but I was wondering whether you have a do you think that their findings could provide some insights into the likely effect size with microdisciplined gene therapy? And If so, how has this affected? How you think about what is the likely effect size with a microdyst in gene therapy?
And you're thinking around the powering of, of Study 2?
Well, let me say this. I'll say I'm going to do my best to talk about our programs and not about others. I will say that to the extent that that others see some functional signals even with very modest expression that, of course, only gives us additional confidence in what we have seen both pre clinically and given the robust expression that we're able to achieve with our current constructs, the functional results that we ought to see. And that frankly, although they are small numbers, what we appear to be seeing already is, as we've talked about, with study. 1 of one of the first four kids, every child, they're not stabilized.
They're improving on every functional endpoint. On with respect to those kids and then you go over to limb girdle, very similar there's enormous amount of read through. And of course, you see the same thing You get great expression. It appears to be tolerable and safe. And we're getting even at the low dose in 1 year with the low dose of LGMD2E construct SRP 9003, you've seen significant benefit on every functional endpoint there.
And then we've got great expression with respect to the higher dose of 2E to 14th. Using suprachoroidal PCR. So we're excited there. What I would say, this is what I'm going to try to do is avoid talking about other programs because as I was, especially with someone earlier today, for those of you on the call that may play best. There's a famous strategy in chess that says you're supposed to play the board, don't play the opponent, and that's what we're going to do.
And frankly, we're excited about the way the board's laid out, laying out right now. Our gene therapy engine and the approach that we take with these constructs is unique to us and it is differentiated from others. It starts your first frankly. And I say this, with the potential of embarrassing Doctor. Luis Rodino Klapac and her team, but it does start with that team.
Under Luis's guidance, the design empirical testing and optimization of these constructs is unique to our approach. And it's, I think it's yielding benefits. Our capsid is different than others. We alone use R874. Our promoter is unique to us.
It's MH CK7. We alone use MHCK7. And the transgene that we use is unique to ours. And I think that's why frankly, just focusing on our programs and not on others, the evidence continues to build that our approach hopefully is not only differentiated, but is paying off to the benefit of patients. And I've talked about this already, but I'll remind you, study 101.
We're very excited about the results that we've seen both from expression, tolerability and safety, but also function. We've seen that out to 1 year, as we've seen in JAMA Neurology this last quarter. And then of course, we had the start of 1 to. And we've now dosed all of the kids for the main, study and we're dosing kids on crossover and that study continues, apace. And that's I think that says a lot about the construct, but I think it also says a lot about the team that was able to overcome obstacles in the middle of this pandemic, both at nationwide and at reptum.
And of course, we have 9003, both low dose and high dose, and what we're seeing both from an expression perspective, but also at least currently with respect to the low dose from a functional perspective, and what we've seen from a safety and tolerability perspective. So just we really want to focus on our programs, but we're very excited about the results that we've received so far. And from our perspective, our goal is to move as fast as possible and this only creates for us an obligation to get going for these kids.
Our next question comes from Joseph Short from SVB Leerink. Your line is open.
Thanks very much. So given the NSAA is comprised of 17 domains and they're scored from 1 and 2. I was wondering if there are certain patterns in the patients that have been treated with 9001 that make you confident that the results will be replicated in the phase 3 or different time course observations, any sorts of characterizations you can share that, make you particularly optimistic?
Yes. I can turn this over to Luis. I'll make an introductory statement about it. I mean, what excites, look, first of all, of course, there is always risk in over analyzing small data sets. This is 4 children with respect to 9001 and so far 3 children with respect to 90 that we've seen functional results regarding.
But with that said, you asked a very good question, which is So you're seeing these significant improvements across this composite score of NSAA, which is built on lots of individual tasks. It could be the case that what we're seeing in aggregate is one function that is significantly improving versus all of the other functions. And then you can worry about what that might mean. The thing that gives us a lot of the excites it gives us confidence, both in DMD with 101 as well as with currently with the low dose of limb girdle that's where we've seen the functional results so far is that every kid is benefiting on every functional endpoint. And if you look at the composite, they're benefiting across all of the functions in that composite.
It is really they are generally improving functionally both on prime tests on NSAA and on the individual components of NSAA, which which is certainly confirmatory. But with that, Doctor. Luiz, you might, Doctor. Rudi Narek, you might have some more nuanced views than I've just given.
I think you captured it well. I think what's important is that, across the components of an SAA as well as the supportive measures like, time to rise, as well as all of the time tests we see supportive data coming out of our one on one study that led to the powering of our study 102. And so we feel confident based on those results across those measures as well as our other studies like our Limbrel study that we will, be successful.
Thank you. Our next question comes from Difei Yang from Mizuho Securities.
Hi, good afternoon and thanks for taking my question. I have a quick one with regards to the 9001 program registrational pathway. So to what extent do you expect the interim analysis of that the 301 will be part of that package?
It is certainly our goal that if they're successful in the with 102 and with an interim analysis of, study 301 that the interim analysis of 301 would play significant role in the registration of the trial. And one of the things I said is in the first if all goes well and we start this study in the second half of this year, then we should have in the first quarter a significant data set on the functional aspects as well as the safety and tolerability aspects, 49001 to treat Duchenne muscular dystrophy. Across both Study 102 and an interim analysis of 301, we would have functional results showing in a placebo controlled manner that the therapy provides functional benefits to these children that it's that is working, that it's benefiting them that we would have a very significant data set on safety and tolerability across the 2 studies. And then, of course, with, with biopsies, and that's one of the things that we get here that in some programs you don't get, like for instance, Zolgensma and that program, there are no buy opsies. But here, we have the opportunity to get biopsies.
So that means in the first quarter of next year, we could have it via interim analysis not only all of the CMC work that we've already done showing that in all of the material ways it ought to operate in the same way as the clinical material But also we would have actual empirical, in vivo biopsy data showing the expression levels as well as safety and tolerability. And on that basis, we would certainly intend to approach and discuss with the agents the opportunity to submit. For a BLA. But that will be a discussion that we'll have with data in hand in the first quarter of 2020. Thank
you. And I am showing no further questions from those phone lines at this time. I would now like to turn the conference back over to Doug Ingram from for any closing remarks.
Thank you very much. Thank you all for participating this evening and thanks for the questions. We really appreciated the opportunity to answer them. I'll repeat apologies. I'll repeat what I said in my opening remarks, which is simply We have a lot to do as an organization.
We've had a lot of success. We're very excited about the progress we've made to date on our gene therapy engine. We're very excited about the opportunity to continue to serve our Duchenne Muscular Dystrophy community with our 2 approved therapies. And hopefully, if approved, 3 therapies by the first quarter of next year. And we're very excited about the potential for our next generation RNA technology, PPMO, to deliver an even more profound impact both in Duchenne muscular dystrophy and beyond Duchenne muscular dystrophy to other areas where steric blocking technology could bring therapeutic benefit to patients.
We have a lot to do. So as much as we are excited about the progress we've made to date, would it be a mistake for us to have to develop hubris or complacency? And I hope you would agree with me that the team at Sarepta does not intend to do that. This is a team that's very focused. I'm really proud of our team.
I'm very proud of our partners. I'm very proud of our clinical investigators. Who have continued to serve these patients during what has been for everyone, a trying time over the course of 2020. We'll stay on mission. And I look forward to providing additional updates as we progress across 2020 with that and have a wonderful evening and rest of the week.
Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone, have a wonderful day.