Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics second quarter 2020 earnings call. At this time, all participants are in a listen-only mode. After the speaker's presentation, we will conduct a question and answer session. To ask a question during the session, you will need to press star and then one on your telephone. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Klem, 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 Cumbo, Ian Estepan, Dr. Gilmore O'Neill, and Dr. Louise Rodino-Klapac. After our formal remarks, we'll open the call for Q&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 update its 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 Chief Executive Officer, Doug Ingram, who will provide an overview of our recent progress. Doug?
Thank you, Mary. Good afternoon, 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 of this year, all but a small contingent of our workforce transitioned 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 remain on track for our many planned 2020 milestones, expanded our three-pronged approach to building our enduring gene therapy engine, and as we have generated additional evidence in 2020, continued to build confidence that our unique approach to gene therapy is proving successful and highly differentiated.
With that, let us review our performance. As we continue to serve our patient community while endeavoring to keep them safe in this pandemic, I am pleased to report that our second quarter net sales stand at $111.3 million, 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-19 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. Consistent with what we have seen thus far in 2020, we anticipate that any continuing impact from COVID-19 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 exon 45 amenable. The PDUFA date for casimersen should be in the first quarter of 2021. If we are successful in obtaining this approval, casimersen will be our third 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 eteplirsen 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 50-51. Here, we are using a proprietary positively charged peptide to increase penetration. In animal models, we have shown that if we can safely achieve appropriate dose levels, the PPMO greatly increases tissue exposure, in turn greatly increasing exon skipping and thus dystrophin production. Treating Duchenne is all about increasing the production of the structural shock absorber dystrophin. If the PPMO candidates are able to increase exon skipping and dystrophin production, we could see a profoundly more efficacious RNA platform, and at 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, PK/PD, and comparative exon skipping for our PPMO 50-51 candidate at 20 mg per kg. This will 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 three pillars upon which we are building our gene therapy engine. First, our pipeline. Second, manufacturing capacity and manufacturing expertise. Third, advancing and improving the science and effectiveness of gene therapy itself.
Over the course of 2020, we have not only stayed on track, but 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 that our unique approach to gene therapy research and development is first in class and replicable. Consider the additional confirmatory data on our gene therapy platform that has been generated already this year. One of the most significant challenges with 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.
You will recall that in 2018, we announced the results of Study 101, our first four DMD patients on SRP-9001, 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 the fourth quarter of 2018 using clinical material. In 2019, we announced the results from our three-patient low-dose cohort for SRP-9003 to treat LGMD2E. This is important for our platform because SRP-9001 and SRP-9003 share much in common, including the design approach under the guidance of Dr. Louise Rodino-Klapac, the same viral vector, rh74, and the same promoter, the heavy chain MHCK7.
We were pleased to report that even at a quarter of our SRP-9001 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 nine months. In 2020, we have generated additional consistent data and gained more experience with our constructs. In the second quarter, the one-year results for Study 101 in Duchenne were published in JAMA Neurology, showing that every boy in the study improved significantly on each and every functional endpoint at one year. In the same month, we announced the results of our second three-patient cohort treated with SRP-9003 for LGMD2E 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-9001 for DMD, we have dosed all children for the main analysis and are dosing children on crossover as well. By now, between our two studies for SRP-9001 and our study for SRP-9003, we have dosed over 35 patients with active therapy, far more than any similar clinical programs, and the 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 two years.
We have among the greatest capacity available for gene therapy in biotech in just two years between our dedicated suites at 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 have 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 third pillar of our gene therapy engine is bringing together the technology and tools necessary to improve and advance the science and effectiveness of gene therapy. We are a science-driven company, and I am pleased to note that COVID-19 has done nothing to slow our progress in this endeavor. Having built out one of the deepest and most valuable gene therapy pipelines commencing in 2017, and then spending an enormous effort in the last two plus years on building out our manufacturing prowess. Commencing in 2019, 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 four 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.
We entered into a partnership with Selecta Biosciences to use their ImmTOR technology in an effort to empower redosing. We entered into a partnership with Hansa Biopharma to access imlifidase with the goal of using it to ablate preexisting neutralizing antibodies and to open gene therapy to patients that would otherwise be left behind. From there, we entered into a relationship with Codiak BioSciences to explore the use of exosomes across gene therapy, gene editing, and RNA. Looking forward, we have important gene therapy engine milestones across the remainder of 2020. With respect to SRP-9001, we have two major 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 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. First, 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. Second, gaining alignment with the FDA on the initiation using the GMP material, which will occur this quarter. With respect to SRP-9003, our goal is to start our pivotal trial in 2021. Our two gating items here are these. First, completing assay development and obtaining the release of our GMP runs, and second, completing a dialogue with the agency on the appropriate regulatory and development pathway for SRP-9003.
This is an important dialogue as it will not only inform the development for SRP-9003, but we believe will provide insight into the development pathway for our other five LGMD candidates. We will provide an update on the LGMD discussions and our GMP material for SRP-9003 in early 2021. With that, if you will indulge me on a personal note, the second quarter marked my three-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.
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 three-pillar gene therapy engine, and are seeing consistent and confirming results thus far.
As I reflect on our progress, the credit should go to three groups, the sophisticated, dedicated, and hardworking professionals that make up our over 1,000-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. 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. You can count on this team to stay on mission, to address and remove obstacles that appear in front of us, and to execute for patients waiting for our therapies.
With that, I'll now turn the call over to Bo for a commercial update. Bo?
Thank you, Doug. Good afternoon, everyone. While our experienced teams at Sarepta are continuing to work through the headwinds of the COVID-19 pandemic, I'm pleased to report that our product revenue for the second quarter of 2020 totaled $111.3 million. 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 has placed on the healthcare systems, the modifications we've made to our commercial execution strategy have allowed eligible patients to start and stay on therapy in this unprecedented time. Our strong relationships with external partners have played a key role in the ability to start new patients and keep others on therapy. This is a testament to our team's expertise and commitment to our mission to serve the Duchenne community.
We continue to provide an uninterrupted supply of therapies 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 VYONDYS 53 are receiving weekly infusions in their homes. We are currently working closely with healthcare providers and specialty pharmacies 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-19, 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 help patients feel safe during this unusual time. We continue to monitor the pandemic and are ready to react quickly with additional modifications to our commercial strategy, especially in places seeing surges in infections. As noted last quarter, the dynamics of initiating treatment with EXONDYS 51 and VYONDYS 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 first couple of infusions, and we've worked with key opinion leaders to find options for patients to safely initiate treatment with VYONDYS 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 state are operating under different rules and regulations. We anticipate the intake of start forms will slowly increase as states ease restrictions and clinics resume normal operations. From a reimbursement standpoint, our healthcare providers have become experts regarding the authorization and reauthorization process for our products. 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 VYONDYS 53 and EXONDYS 51 and start therapy in a timely manner. We have successfully adapted our weekly engagements with key opinion leaders and payers to virtual interactions. Our discussions with key opinion leaders and other healthcare providers are focused on identifying patients amenable to exon 51 and 53 skipping and driving prescriptions.
We also continue to educate payers 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 exon 45. In June, we announced the completion of our NDA submission for casimersen or SRP-4045. As we await FDA's response, our commercial and medical affairs teams are preparing for another successful launch. The foundation of our plan will be tailored to reaching patients who are amenable to exon 45 skipping, who represent another 8% of the Duchenne community. We will leverage our knowledge and experience from the EXONDYS 51 and VYONDYS 53 launches to facilitate patient access to casimersen as quickly as possible.
While we're very proud of the accomplishments the team has made to date, we still have an immense amount of work ahead of us. With each new therapy that is developed and advanced in our pipeline, we have the responsibility to pave the way for patients to access these treatments. This is an incredible responsibility and one that we do not take lightly. The depth of our experience on our teams has helped us navigate through complex launches in these unprecedented times. With each new launch and lessons learned, we become a stronger company, one that's better able to serve our patients and deliver on our mission as the global leader in precision genetic medicine. With that, I will now turn the call over to Ian for an update on our financials. Ian?
Thanks, Bo. 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 $111.3 million compared to $94.7 million for EXONDYS 51 alone for the same period of 2019. The increase primarily reflects higher demand for our products. In the quarter ended June 30, 2020, we recognized $26 million of collaboration revenue, which primarily relates to our collaboration arrangement with Roche. The co-development costs under the Roche agreement totaled $8.9 million for the second quarter and are included as a reduction to our R&D expenses.
On a GAAP basis, we reported a net loss of $150.8 million and $276.4 million, or $1.93 and $3.74 per basic and diluted share for the second quarter of 2020 and 2019 respectively. We reported a non-GAAP net loss of $117.9 million or $1.51 per basic and diluted share in the second quarter of 2020 compared to a non-GAAP net loss of $61.2 million or $0.83 per basic and diluted share in the second quarter of 2019. In the second quarter of 2020, we recorded approximately $13.3 million in cost of sales compared to $15.9 million in the same period of 2019. The decrease is primarily due to write-offs of certain batches of EXONDYS 51 not meeting our quality specifications for the three months ended June 30, 2019 with no similar activity for the three months ended June 30, 2020.
On a GAAP basis, we recorded $188.5 million and $113.3 million in R&D expenses for the second quarter of 2020 and 2019 respectively, which is a YoY increase of $75.2 million. This increase is primarily related to $81.6 million increase in manufacturing expenses, primarily due to the continuing ramp-up of our micro-dystrophin program. On a non-GAAP basis, R&D expenses were $160.4 million for the second quarter of 2020 compared to $87.5 million for the same period of 2019, an increase of $72.9 million. The YoY growth in non-GAAP R&D expenses was driven primarily due to a continuing ramp-up of our micro-dystrophin program. Now turning to SG&A. On a GAAP basis, we recorded $73.7 million and $67.4 million of expenses for the second quarter of 2020 and 2019 respectively, a YoY increase of $6.3 million.
On a non-GAAP basis, the SG&A expenses were $55.1 million for the second quarter of 2020 compared to $52.3 million for the same period of 2019, an increase of $2.8 million. The YoY 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 GAAP basis, we've recorded $12.4 million in other expenses net for the second quarter of 2020 compared to $0.9 million 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 $2.1 billion in cash equivalents, and investments as of June 30th, 2020. With that, I'll turn the call over to Gilmore for an update on our research and development activities. Gilmore?
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 one of the three platforms Sarepta uses to drive its precision genetic medicine therapeutic strategy. I will begin with the PMO portfolio. As Doug mentioned in June, as planned, we completed the NDA rolling submission to the FDA for casimersen. If accepted and subsequently approved, we will have more than doubled the size of the treatment patient population since eteplirsen was first approved. Importantly, our post-marketing requirement or PMR studies are progressing despite the challenges arising from the COVID-19 pandemic. In summary, the MIS51ON PMR study for eteplirsen is dosing, and we have enrolled the first cohort of patients. The ESSENCE study of golodirsen and 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-19 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 miss 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 again. All of our mitigation strategies comply with guidances from regulatory agencies around the world on the conduct of trials during the COVID-19 pandemic. Indeed, the pandemic has served not only as a disruptor, but a catalyst to permanently change our approach to clinical trial execution.
While not being complacent, we are confident that we can deal with and minimize the impacts on all of our future trials in the face of surges in COVID-19 infections. Before I turn to the PPMO platform and the PPMO-5051 program for DMD specifically, let me give you an update on the USAMRIID collaboration. In late April, we announced an early research collaboration with USAMRIID that would exploit our PPMO technology as a potential therapeutic for COVID-19. The work is ongoing with USAMRIID. We have expanded the collaboration to work with a leading research group in Sweden. Early results support performing additional confirmatory experiments. Turning to PPMO development. A key element of Sarepta's R&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-penetrating peptide to PMO to enhance cellular and nuclear penetration. Our most advanced PPMO program is SRP-5051. Our ongoing SRP-5051 201 multi-ascending dose trial, named MOMENTUM, is advancing, and all patients in the 20 mg per kg cohort have been dosed. COVID-19-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 mg per kg and have escalated already to 20 mg per kg. 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 mg per kg cohort of Duchenne patients treated with PPMO-5051.
We will be examining systemic PK, tissue penetration, safety, and exon skipping data. We will measure exon skipping by digital drop PCR or ddPCR, allowing us to directly compare the efficacy of our PMO and PPMO candidates. Our pre-clinical in vitro and in vivo models have demonstrated a robust correlation between the amount of exon skipping and amount of dystrophin production. We will be publishing 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-penetrating 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. So we expect to see higher levels of dystrophin at later time points if the therapy is successful. Muscle biopsies at later time points 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, additionally, will potentially extend to other PPMO Duchenne programs. We have already designed and selected five additional PPMO candidates, in fact, that could treat 50% of Duchenne patients who carry skip-amenable mutations in their dystrophin gene.
I do want to highlight that rarer deletions susceptible to exon skipping impact an additional 35% of patients with Duchenne, and we are creating a development strategy that would attempt to accelerate the approval of PPMO compounds if successful for these patients using a novel platform or an extrapolation strategy. Proof of concept for PPMO in DMD, or Duchenne, would also read through to the selection of that chemistry to target new non-muscle therapeutic areas. Non-DMD and non-muscle therapeutic area targeting is a key pillar of Sarepta's R&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&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 will hand back to Doug for Q&A. Doug?
Thank you very much, Dr. O'Neill. Crystal, with that, let's open the call for questions.
Thank you. Ladies and gentlemen, if you have a question at this time, please press the star followed by the number one key on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. In the interest of time, we do ask that you please limit yourself to one question per caller. 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? Also, do you plan on asking them about the plan to test the non-ambulatory or older patients as well? Thanks.
Thanks a lot for that, Tazeen. 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 do. We've got two things to do. As many of you know, we've got to get our next study using commercial process material up and running and getting patients dosed, and we're going to do that in the second half of this year. The team, notwithstanding COVID-19 and the distractions that occur and some of the challenges occur with COVID-19, have done really remarkable jobs keeping on pace and moving to get that study started. Before we can do that, we have to have our meeting with the agency.
As I said in my prepared remarks, we will be meeting with the agency this quarter, there really are just two things that, they're significant things, but there are two things that we need to get alignment and concurrence on with the agency. The first, of course, is our CMC approach itself for the commercial material, to use the commercial material in the next study. We're very pleased at how these GMP runs have come out. 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 in a manner that would be predicted to affect the therapy in any way functionally or efficacy-wise or safety-wise. That's one significant concurrence we need. Of course, the second one is to gain concurrence on the next study.
Certainly, in connection with that, 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 therapy in the United States.
Thank you. 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?
Yeah, that's a great question. I'll answer that briefly, and then I'll turn this over to Dr. O'Neill to maybe talk about some of the areas we could take this RNA technology. First and foremost, the excitement of the PPMO, if it is successful, is for an even more profound impact from an RNA perspective with respect to Duchenne muscular dystrophy. Of course, the question will immediately arise thereafter of how will 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 not available or not yet available.
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've been able to address that issue scientifically, that is larger than the eteplirsen population. Of course, with a significantly more impactful RNA platform, it may very well be the case that there is a role for a combination both of gene therapy and of RNA. 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 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.
Beyond that, of course, 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, et cetera. To move beyond DMD, we need a more penetrative RNA technology like the PPMO, if it works. If it does, the theory is that we can take this far beyond DMD, and the research group is already working on the kinds of areas we would take that. Perhaps with that, I can turn this over to Dr. O'Neill, who can touch on that issue.
Thanks very much, Doug. I think you actually articulated very nicely. The beauty of the RNA 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 upregulation, or rather skipping and a dystrophin isoform expression by returning it to in frame. With the PPMO experimentation with SRP-5051 and the study readouts, we hope to test the hypothesis that increased exposure driven by the cell-penetrating peptide fusion to the PMO will enhance tissue exposure in muscle. 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, et cetera.
We are actually doing a comprehensive genome-driven review of those tissues and the associated diseases to identify those that are amenable to exon skipping. That is work that is ongoing, and I'm very excited about it.
One final thing I will say, moving back to DMD, I think people know this, but I will remind you. We're currently using SRP-5051 exon 51 PPMO as our proof of concept. Of course, later this year, we'll have some of the PKPD tissue dosing safety and exon skipping, importantly, exon skipping for SRP-5051 as our proof of concept. We have already built beyond SRP-5051 constructs with the peptide conjugated to them for exons that could treat already about 50% of the Duchenne community with a path, as Dr. O'Neill mentioned in his opening remarks, with a path to get to a significant percentage beyond that, perhaps another 35% beyond that, with a slightly more exotic platform approach.
We will start moving as rapidly as we can, assuming, of course, that the results support that when we look at them later this year.
Thank you. Our next question comes from Anupam Rama from JPMorgan. Your line is open.
Hey, guys. Thanks so much for taking the question. We've gotten a lot of inbounds on SRP-9001 safety recently. I think some of it may revolve around some comments from Dr. Mendell, maybe at ASGCT, that SRP-9001 may be associated with some sort of mild complement activation. Any context you can provide here? Just maybe remind us of how frequently the DSMB meets for Study 2. Thanks so much.
Sure. I'm going to turn this question over to Louise, who can answer this. I will say up front, just so we're absolutely clear, that was a misunderstanding. There is absolutely no signal that we've seen of complement associated with SRP-9001 at all. I think Dr. Rodino-Klapac can provide the context around that misunderstanding.
Sure, yeah. Dr. Mendell was speaking about his broad experience in gene therapy, and that includes other vectors like AAV9. As Doug mentioned, we presented our data in both different medical meetings as well as investor calls on LGMD2E, as well as our JAMA Neurology paper, and just to reiterate, we've not seen any evidence of complement activation in our studies. Study 1 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 AAV9. He wasn't speaking to rh74. We've seen nothing relating to complement there. Just to remind you, and of course, I know Dr. O'Neill is a proper scientist. We have to measure ourselves by the evidence on hand. The good news for us is that, with respect to rh74 across both Duchenne muscular dystrophy and limb-girdle, we have now dosed, as you heard in my opening remarks, over 35 children. The Study 102 continues to move apace. I think the evidence on our broad safety profile, I think is only 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. We'll move on to our next caller, Matthew Harrison with Morgan Stanley.
Hi, this is Maxwell Skor on for Matthew Harrison. Just a quick question. Based on the recent agreements you signed, how are you thinking about integrating these new technologies into your pipeline? Thank you.
Thanks a lot for that question, Max. 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 evolve the science of gene therapy. We've got Dyno, which is artificial intelligence and machine learning for making better capsids. We've got Codiak, which is an entirely different approach using exosomes for a delivery device that you could use in RNA gene therapy and gene editing. Then I think for the more near term, perhaps, but really impactful is both Hansa with imlifidase and Selecta with ImmTOR. Hansa's technology would ablate preexisting neutralizing antibodies so that you could essentially, 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.
Of course, Selecta's is this concept of co-administering 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 capsule it's receiving is self and not foreign, so you don't build up neutralizing antibodies, which could empower redosing and 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 armamentarium that Dr. Rodino-Klapac and her team have to advance this, 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 of patients into frame for gene therapy.
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. Your line is open.
Hi, everyone. This is Mark from Marty. Thanks for taking my question. My question relates to the upcoming readout for SRP-5051. I know that you framed the program relative to EXONDYS in terms of dystrophin expression. I'm curious, what is the level of exon skipping that EXONDYS achieves in humans? Could you speak to the magnitude of improvement in exon skipping you're hoping to see with SRP-5051 as part of this initial clinical trial update? Thank you.
Sure. Dr. 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, golodirsen, and casimersen.
Yeah. 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 PPMO. It's important to actually distinguish that from the older quantitative RT-PCR that's been used in the past. We will actually be looking at data from both PMO historical samples and then looking at our ddPCR in our 5051 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, in cell lines, as well as in animal models, seen a robust correlation between dd, digital drop PCR, and dystrophin levels. What we've actually seen there is a ratio of between 2:3 .
Obviously, we have to validate that in human, but what we've seen in our experimentation to date.
Has been, I should say, gives us confidence with regards to both the robustness of the correlation and 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. Congrats on the quarter. I just wanted to talk a little bit about hitting the GMP material and the comparability. It 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. Then just on Selecta and that ImmTOR, I was just interested in Dr. Rodino-Klapac's perspective on how to think about redosing and how the technology hypothetically might be important in that. Thank you.
Okay. Before I turn it over to Louise to talk about Selecta et al. Thank you, Alethia. I agree with you. It's a big deal. It's not a one-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, et cetera. You've sort of lived with us as we've worked through that process, optimized or are in the process of optimizing process development, getting all of our analytical development done. There's 24 assays involved here.
All are complete, either qualified or validated as the case is required for each of those. Of course, we've done numerous runs, and having GMP material on hand right now is enormously important. Now, 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 of the material, et cetera, but we feel very good about where we are and the results that we've seen. To your very good question, it's going to pay significant dividends on our platform more broadly. As I said, and I think in my prepared remarks, this concept of a gene therapy engine isn't simply a narrative. It is a platform that is built on three significant pillars, all of which are required to build an enduring gene therapy platform.
The first, of course, is the pipeline. 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. Of course, the latter part I talked about is that Louise 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. Of course, that middle concept of manufacturing is crucial. 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.
The fact that we have GMP material on hand right now speaks reams 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 those programs. I said in the end of my opening remarks, it is so easy to get hubris 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. We still have more results to come in. We have to see 102 at the beginning of next year.
Certainly, as you triangulate the results we're getting, both 101, then the nine 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 a low dose on 9003 and the nine months there, and then the low dose on 9003 and the successful one year there. Then the one year on 101, and now, of course, the high dose on 9003 and the exceptional expression we're getting there and the good safety profile that we're seeing across all the programs, gives us some confidence that we're on the right track to be able to do what we're trying to do.
What we're trying to do isn't simply something that will be commercially successful, but far more profoundly than that, we want to bring a much better life to these patients with girdle and Duchenne patients, and then beyond that, who are living with and degenerating from, and as I said before, far too often, succumbing to these rare genetic diseases. Thank you for that question. With that, I apologize. I rambled for a second, I should turn this over to Dr. Rodino-Klapac, who can then talk to Selecta et al.
Yeah. Thank you for that question. We're excited about both Hansa and Selecta. Hansa gives us an opportunity to ablate antibodies in patients that are positive for AAV antibodies. That helps us potentially get to those 15% or so patients that have preexisting antibodies and also gives us the opportunity potentially for redosing down the road. Selecta, we're particularly excited about because it has the potential to prevent those antibodies from forming in the first place. With the first dose of gene therapy, patients could be co-administered this drug potentially, and then prevent those antibodies from forming with the potential to redose down the road. 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. We're definitely excited about the potential there.
We want to make sure that we have every tool to reach all patients, and that's our goal. We may never need it, but it's better to be looking at it proactively, and that's what we're doing. 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. You mentioned that you started at 4 mgs per kg and are up now to 20 mgs per kg. If I'm not mistaken, you mentioned you could also potentially go higher than that. 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.
Yeah. Thank you for that question. First understand the following. We are already above what we would have considered success, at least from our pre-clinical models. Now, please don't overread that. We've got to get the data, and we've got to see the biopsies, and we've got to do the work. I don't want to oversell this, but I will say, if you look from a pre-clinical prediction perspective, we're already beyond that point where we would've said, 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 a real signal. In pre-clinical models, that will be the dose-limiting signal that will help us choose the dose.
Our goal, of course, is to reasonably achieve the highest dose that is safe for these kids. 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. Certainly, we're going to go to 30 mg per kg this year. You won't have the data on 30 mg per kg. It won't come in time, but we'll have the 20 mg per kg, which will be certainly insightful for us. Is there anything there that I missed, Dr. O'Neill?
No, you nailed it. The target organ is the kidney, and that's what we're monitoring closely. It's been good so far.
Thank you. Our next question comes from Brian Abrahams from RBC Capital Markets. Your line is open.
Hey, guys. Thanks for taking my question. Congrats on all the progress. With the full one-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 positive results, but maybe a little bit of variability measure to measure in CK and function across the NSAA components, as well as the learnings maybe there from the cardiac MRIs. Thanks.
I'd say broadly, one of the values of Study 101 is that it allows us real-time insight as we build out Study 102, as we look forward to our commercial process trial as well. We already had a significant amount of experience with 101 by the time we commenced Study 102. It already informed our thinking, and it informed the powering of that study, as well as the selection of the primary functional endpoint being NSAA. We've had an opportunity to continue to monitor these children, as you saw, of course, we have the one-year data, we have that reported out in JAMA Neurology. I will tell you that it is confirming of the approach that we took with respect to Study 102, the way we've chosen NSAA, and frankly, the way we have powered that study.
We feel very good about where we are, and I think Study 101 has been very helpful in helping us power that study and give us confidence that we're on the right track.
Thank you. Our next question comes from Gena Wang from Barclays. Your line is open.
Thank you for taking my questions. I have one question regarding PPMO, has three parts. The first is just follow up with the earlier question. Could you remind us the percentage of exon skipping for EXONDYS 51 in early animal data? The second question is for the non-muscle, non-DMD program, what peptide you have in mind for the PPMO program? Lastly, do you see antibody oligo-based therapy a threat to your PPMO franchise?
Yeah. Fine. Okay. First of all, to the last question, I'll answer no. We're excited that other folks are looking at researching ways to bring better lives to Duchenne muscular dystrophy. We certainly feel that our programs are the most advanced and the most hopeful. We're really focused on our own programs. Going back to your other two questions, I will turn this over to Dr. O'Neill, who can give you some perspective. Broadly speaking, I'll let Dr. O'Neill 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 eteplirsen, it won't be meaningful here because the approach to looking at exon skipping is different. We're using digital drop PCR now. In the past, we used a different form.
I think it was RT-PCR. Dr. O'Neill will remind us of that. It's a bit apples to oranges. The good news, just so we know, is that when we announce the results of the 20 mgs on the PPMO, we will have data using the same exon skipping technology for eteplirsen. There will be an ability to see what we might have seen with the eteplirsen versus what we will see with 5051. I believe, I'll let Dr. 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. Dr. O'Neill, you can correct me if I've said anything incorrect there.
No, that's correct. I think, Gena, you asked, just restate, you asked two questions about peptide and the PMO skipping. I think I could do one better with regard to the ddPCR than the animal, in that we did actually look at it at 24 weeks, actually over several week periods, but at 24 weeks in the PROMOVI in humans, in patients, we saw about 0.6% skipping that exon skip. It's very important to emphasize that's digital drop PCR. It's not the quantitative PCR that was used in prior descriptions. That, I think is disclosed in our MDA poster. The beauty of the ddPCR is it's very tight and actually allows you then to compare across patients and very importantly, and more importantly, to protein expression downstream. Then you asked another question about peptides. We've not disclosed the nature of the cell-penetrating peptide.
I think Doug and 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. Obviously our Codiak collaboration that we announced is also part of an overall approach to both improve tissue penetration and frankly, specific or target or tissue specific targeting.
Thank you. Our next question comes from Joel Beatty from Citi. Your line is open.
Hi, this is Yigal 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 limb-girdle muscular dystrophies 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?
Yeah. Thank you for that. Great question. I think, as I said in my opening remarks, we have two significant things to do over the course of this year. One is to complete the GMP runs, which is a combination of two things, just 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 SRP-9001, but there are bespoke assays for each particular program, so we've got to complete that work as well.
The second thing to your good question is, we need to engage in a dialogue with the agency about the appropriate regulatory and development pathway forward. One of the things we said is that we've already started commencing a dialogue there. We intend to have a meeting before the end of this year on that issue as well. To your point, why would we? We are, I think we're unabashed about our view at least, and we'll see where we end up, that it would be in the best interest of patients and also appropriate from a development and regulatory perspective to have a rapid and accelerated approach to the development of not only limb-girdle 2E, but also at a minimum, the other sarcoglycans, and 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 monogenic 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. Second of all, these are ultra-rare diseases. These are, as rare as Duchenne muscular dystrophy is, 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 to this. The third reason, of course, is that this is the therapy at hand. Because of the particular genes and proteins at issue here, we are able to comfortably package in our rh74 capsid, a gene that codes for the native protein.
We can replace the precise native protein that these children are lacking, and the absence of which is causing their demise. The only remaining question then is, can we produce a significant amount of it? The short answer is that we've seen in 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 off by a percentage or two, but on Western blot, I believe we were in the 30%, and on protein positive fibers, we were over 50%. Then, of course, as you're seeing in the higher dose, it was well-tolerated, and even as it's well-tolerated, 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.
From our perspective, you look at the guidance and you look at the statements that the agency's made publicly about opportunities in gene therapy, this 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 those, but we certainly will come back at the beginning of next year and give you an update on those discussions with the agency. At that time, hopefully, we can plan out exactly what the path is, not just for 2E, but for all of the other limb-girdles as well.
Thank you. Our next question comes from Vincent Chen from Bernstein. Your line is open.
Thanks for taking the question. Congrats on the progress. Over the last few months, in addition to, I guess, your updated one-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. Clearly they're different programs, but I was wondering, do you think that their findings could provide some insight into the likely effect size with micro-dystrophin gene therapy? If so, how has this affected how you think about what is the likely effect size with a micro-dystrophin gene therapy and your thinking around the powering 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 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, and as we've talked about with Study 101 in the first four kids, every child, they're not stabilized. They're improving on every functional endpoint with respect to those kids. You go over to limb-girdle, very similar. There's an enormous amount of read-through. Of course, you see the same thing. You get great expression.
It appears to be tolerable and safe, and we're getting, even at a low dose in one year with the low dose of LGMD, the 2E construct, SRP-9003, you've seen significant benefit on every functional endpoint there. Then we've got great expression with respect to the higher dose of 2E to the 14th using supercoiled PCR. We're excited there. What I'm going to try to do is avoid talking about other programs, because as I was discussing with someone earlier today, for those of you on the call that may play chess, 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. Frankly, we're excited about the way the board's 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 first, frankly, and I say this with the potential of embarrassing Dr. Louise Rodino-Klapac and our team, but it does start with that team. Under Louise's guidance, the design, empirical testing, and optimization of these constructs is unique to our approach, and I think it's yielding benefits. Our capsid is different than others. We alone use rh74. Our promoter is unique to us. It's MHCK7. 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.
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 one year, as we've seen in JAMA Neurology this last quarter. Of course, we had the start of 102, 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. 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 Sarepta.
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. Just, we really want to focus on our programs, but we're very excited about the results we've received so far. 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.
Thank you. Our next question comes from Joseph Schwartz from SVB Leerink. Your line is open.
Thanks very much. Given the NSAA is comprised of 17 domains and they're scored from zero, one, and two, 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 III or different time course observations. Any sorts of characterizations you can share that make you particularly optimistic?
Yeah, I can turn this over to Louise. I'll make an introductory statement about it. First of all, of course, there is always risk in overanalyzing small data sets. This is four children with respect to 9001, and so far three children with respect to 9003 that we've seen functional results regarding. With that said, you ask a very good question, which is, you're seeing these significant improvements across this composite score of NSAA, which is built on lots of individual paths. 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, you can worry about what that might mean.
The thing that excites us and gives us confidence, both in DMD with 101, as well as currently with the low dose of limb-girdle, because that's where we've seen the functional results so far, is that every kid is benefiting on every functional endpoint. If you look at the composite, they're benefiting across all of the functions in that composite. They are generally improving functionally, both on time tests, on NSAA, and on the individual components of NSAA, which is certainly confirmatory. With that, Dr. Louise Rodino-Klapac , 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 NSAA, as well as the other supportive measures like time to rise, as well as all of the time tests, we see supportive data coming out of our Study 101 that led to the powering of our Study 102. We feel confident based on those results across those measures as well as our other studies like our limb-girdle study that we will be successful.
Thank you. Our next question comes from Dae Gon Ha from Mizuho Securities. Your line is open.
Hi. Good afternoon. Thanks for taking my question. I have a quick one with regards to the SRP-9001 program registrational pathway. To what extent do you expect the interim analysis of Study 301 will be part of that package?
It is certainly our goal that if they're successful with Study 102 and with an interim analysis of Study 301, that the interim analysis of 301 would play a significant role in the registration of the trial. One of the things I said is 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 for SRP-9001 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 working, that it's benefiting them, that we would have a very significant data set on safety and tolerability across the two studies.
Then, of course, 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 biopsies. Here we have the opportunity to get biopsies. That means in the first quarter of next year, we could have it via an 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. Also we would have actual empirical in vivo biopsy data showing the expression levels as well as safety and tolerability. On that basis, we would certainly intend to approach and discuss with the agency the opportunity to submit for a BLA.
That will be a discussion that we'll have with data in hand in the first quarter of 2021.
Thank you. I am showing no further questions from our phone lines at this time. I would now like to turn the conference back over to Doug Ingram 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 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 two approved therapies, and hopefully, if approved, three therapies by the first quarter of next year. 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, it would be a mistake for us 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, 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. You may all disconnect. Everyone, have a wonderful day.