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Sarepta Therapeutics (SRPT -0.66%)
Q3 2020 Earnings Call
Nov 05, 2020, 4:30 p.m. ET


  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:


Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics third-quarter 2020 earnings call. [Operator instructions] As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, senior manager, investor relations. Please go ahead.

Mary Jenkins -- Senior Manager, Investor Relations

Thank you, Catherine, and thank you all for joining today's call. Earlier today, we released our financial results for the third-quarter 2020. The press release is available on our website at sarepta.com, and our 10-Q was filed with Securities and Exchange Commission earlier this afternoon. Joining us on the call today are Doug Ingram, 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'll be making a number of forward-looking statements.

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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 and annual report on Form 10-K and most recent quarterly report on Form 10-Q filed with the SEC 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 CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Doug Ingram -- Chief Executive Officer and President

Thank you, Mary. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics third-quarter 2020 investor conference call. Tonight, I'm very proud to share with you the progress we have made this quarter including our performance in serving the Duchenne community with EXONDYS 51 and VYONDYS 53, our progress with respect to our RNA platform and the strides we have made in building out our enduring gene therapy engine, and -- but I'm going to take things a bit out of order this evening. I'm going to commence this call by discussing an important milestone for Sarepta and for the Duchenne patient community.

As you will have read in our press release today, as we worked with the FDA, we now anticipate dosing the trial with commercial process material this year. Let me provide some background and context. As you well know, SRP-9001 is our gene therapy and the goal of it is to treat Duchenne muscular dystrophy by safely delivering the skeletal diaphragm and cardiac muscle, a gene that robustly codes for the truncated but functional form of the structural protein dystrophin that we call microdystrophin. So that there are now -- DMD make essentially no dystrophin, which results in rapid degeneration and inevitable death, and the goal is to arrest this degeneration by giving them a properly localized functional form of that structural protein.

After many years of design and preclinical testing by Doctors Jerry Mendell and Louise Rodino-Klapac, we commenced and completed a small four-patient proof-of-concept studies, Study 101, and that was positive and very encouraging. The therapy was well-tolerated. The gene was indeed robustly delivered to the target muscles, about 3.3 genome copies per nucleus. So, the expression of the protein approached nearly normal levels and on all functional measures, the children continued to improve in ways that cannot be explained by our natural industry.

Based on this proof-of-concept study, we commenced a randomized, blinded placebo-controlled trial. And you'll recall that it's Study 102. Study 102 is intended to show, in a well-controlled trial, that SRP-9001 is safe and effective in children with DMD. The last one-year visit will occur in December, and we plan to release results from our Study 102 in the first quarter of 2021.

Now the material for Study 101 and 102 was manufactured by our partner, Nationwide Children's Hospital, pursuant to a process that will not scale for large commercial purposes. Over the last two years or so, we have developed a commercial process, and we've built significant manufacturing capacity. Assuming SRP-9001 proves effective, these efforts are expected to provide us capability to produce and launch a commercial product to fully serve the community. By early 2020, we completed our process development and analytical development, and we commenced our GMP runs for SRP-9001 commercial process material to use in future clinical studies.

In the summer of this year, with the GMP material in hand to conduct additional studies, we requested a meeting with OTAT. OTAT is the division of the FDA responsible for cell and gene therapy, and we did that to obtain their blessing to commence a commercial material validation study. We originally contemplated a larger, 70-patient, blinded, placebo-controlled trial called Study 301. However, in light of the likelihood of a second wave of COVID-19, which now may be coming to fruition and the risk it poses to clinical trial participation and execution in anticipation on our September Type C meeting, we proposed a leaner open-label commercial material validation study, Study 103, in the same patient population as Study 102, our ongoing placebo-controlled trial.

The goal of Study 103 is to gain insight and validate the performance of our commercial process material on both microdystrophin expression and safety in up to 10 patients with the primary analysis of Part 1 of the study conducted at 12 weeks post-infusion in all treated patients. Our Type C meeting with the division, which was conducted entirely in writing, occurred in September of this year. In its written response, the division have raised concerns with the potency release assay approach that we had proposed. As I stated at the time that this occurred, if we were to follow a formal process to resolve our issues with the division, it would would've taken a minimum of a matter of months and could could've extended into next year.

However, I am pleased to report that the division was willing to work with us expeditiously and informally to discuss the issues, to really provide guidance to us and to provide us with a pathway to commence our commercial validation study this year. Indeed, the division moved quickly to meet with us in late September, and we have spent the time since then gathering the data necessary for our updated potency assay approach for Study 103 and finalizing the meeting minutes. Based on those discussions, the status and the next steps for our program are these: first, after discussion and obtaining division guidance, we proposed and the division accepted, a potency assay approach for Study 103. We have already completed the work and generated the data from the new potency assay approach for Study 103.

Second, the division will permit us to commence Study 103, which we intend to do before the end of this year. And third, to remind you, Study 102 will have the last 12-month visit in the December of this year, and we will report the results of Study 102 in the first quarter of 2021. Depending on the external environment and the pandemic, we anticipate speaking with the division and starting our larger Study 301 in 2021 after we have data available from Study 103. We also intend to design and propose to the division for their review and input additional studies, including, importantly, in older and non-ambulant patients.

I would like to thank OTAT for their responsiveness and their willingness to informally and rapidly meet with us on this extremely important program, even in the midst of this pandemic that has placed additional burden on an already overworked group of professionals at the FDA, so that we may embark on Study 103. Now staying with the build of our gene therapy engine. Over the course of this quarter, we've had a number of very positive updates. As you will recall, back in June, the one-year results for Study 101 were published in JAMA Neurology, reporting robust expression, good tolerability and safety and functional improvements across all measures in the cohort of Duchenne boys in that study.

At the World Muscle Society Conference in the third quarter, we provided an update on Study 101, reporting not only continued safety and tolerability, but also durability of the effect with all boys continuing to show functional benefits of the gene therapy at two years. At World Muscle as well, we also provided updates on our two -- three-patient cohorts for SRP-9001 for the treatment of limb-girdle muscular dystrophy type 2E or LGMD2E. Cohort 1 was dosed at 5E to the 13th and Cohort 2 was dosed at 2E to the 14th, same dose used in our studies for SRP-9001. As you will recall, both cohorts were generally well-tolerated with a significant dose-dependent increase in expression in Cohort 2.

At World Muscle, we reported the 18-month results for Cohort 1, showing not merely stabilization, but a really substantial improvement in function above baseline and above natural history. We also reported the early six-month data for Cohort 2, where the children are already showing a near stabilization of function, but improvement in function, again, against both baseline and against natural history. We are particularly pleased with these LGMD2E results, and in particular, the safety, expression, biomarker and functional results of Cohort 2 as it has potential read through to our remaining LGMD portfolio, and supportive confirmatory read through to our SRP-9001 program in DMD as the program shared its investor, the same promoter, the same design approach. And in the case of Cohort 2, the same dose as well.

With respect to LGMD2E, we are completing manufacturing and material for our next trial, and we'll engage with the agency on the design for what we hope will be the pivotal study. We will provide an update on both of these matters and our perspective on our development and the regulatory pathway and timing for our entire LGMD portfolio in 2021. We continue to build our gene therapy platform and gather technologies to improve and enhance gene therapy. Over the course of 2020, we have completed some 22 gene therapy related transactions, 20 of which were completed since this pandemic sent us all to a largely work-from-home environment.

We also have what is likely the largest pipeline of high-potential gene therapy candidates in biopharma today. We are considering an R&D Day in 2021, at which we can update on our entire pipeline of gene therapy and RNA, and we will provide more details on it early next year. Moving on now to our RNA platform. Let me now discuss our commercial performance in the quarter.

As you will recall, in light of the dynamic and unpredictable nature of the COVID-19 pandemic, we withdrew our guidance for 2020. Nevertheless, due to the collaborative work of our multidisciplinary team, I am pleased to once again report that we've been able to serve the Duchenne community with our approved therapies with only modest impact from the current pandemic. For our PMO franchise, currently EXONDYS and VYONDYS, our net product revenue for the quarter is $121.4 million, and that represents a nearly 23% increase over the same quarter last year. As we have never taken a price increase on our therapies, our performance relates directly to our ability to serve the patient community.

We continue to monitor performance closely and as we are, indeed, in uncertain times. It is important to our patient community that they have an uninterrupted supply of therapy. While challenging, we have been able to largely ensure that this is the case, in part, due to great execution from the team and also due, in part, to the fact that the vast majority of our patients receive in-home infusions rather than having to go into a clinic or hospital and also due to the fact that, thus far, the majority of payers have responded positively in this pandemic and have shown flexibility in our authorizations and reauthorizations for this vulnerable DMD patient community. So, now moving to our PMO pipeline.

The FDA has accepted our filing for our third Duchenne therapy, casimersen, a PMO therapy intended to treat those 8% of Duchenne patients who are amenable to skipping Exon 45. The brand name for casimersen will be a AMONDYS 45. Our PDUFA date is February 25, 2021, and the review is going well. If we obtain approval for casimersen, we will have three approved therapies, capable together of treating nearly 30% of the DMD community.

And as we have mentioned before, our RMA technology has the potential to bring therapies to as many as about 85% of patients living with DMD, so we do have much left to do. As you well know, we are also making progress on our next-generation in the PMO, which, if successful, may be -- 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 key PMO, and that's candidate, SRP-5051. To remind you, 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, entering greatly increasing exon skipping and the [Inaudible] dystrophin production. So, before the end of this year, we will provide an update on the safety systemic exposure and exon skipping for our PPMO SRP-5051 candidate at 20 mg per kg. And for us, the most important measure of all of these, and I've said this many times in the past, will be safety. Consider the following: our PMOs are safe and they're precise.

Their ultimate limitation, however, is that they are also neutrally charged. They penetrate muscle cells passively, and they clear the body in about four hours. This will limit the amount of therapy that can get to the right place and simply increasing the dose will not reverse this limitation. Our PPMO technology, on the other hand, potentially addresses this limitation in a dose-dependent way.

Indeed, our preclinical models have now consistently shown that if one can safely dose the PPMOs to a sufficiently high level, we should hit a point where we get a significant increase in cell exposure and a great increase in exon skipping and dystrophin production, and that should translate into a great increase in benefit to patients living with Duchenne muscular dystrophy. Our most significant question in the MOMENTUM Study has always been safety upon repeat dosing and maximum dose. We will present the 20-mg-per-kg result this year, but we have already moved to 30 mg per kg, and intend, if safety signals will permit, to continue to dose even up to 40 mg per kg early next year and ultimately, potentially even higher than that if we are able to reduce so. While we can make some educated guesses from animal models, until we observe it in human clinical trials, we can't know with any certainty precisely where the inflection point on exposure and dystrophin will occur in patients, purely based on animal studies, but what we are confident about is the PPO's mechanism of action, which means we are very confident that if we can safely achieve high enough doses, and we should see a very substantial increase in dystrophin production.

So not to belabor this point, but it is all about safety, safety signals and maximum tolerated dose at this point. In conclusion, notwithstanding the unusual external environment, the team continues to execute with an unrelenting purpose, and the third quarter of 2020 perfectly exemplified our culture of performance, consistent with our message, our mission policies. We kept the patient front and center in everything we did, which means we focused on the science. We continued to execute with a sense of urgency that our mission requires and when faced with the inevitable obstacles and roadblocks, we did not simply accept them or regress to the mean, but we moved with rapidity and creativity to address them to remove them and to keep progressing toward our ultimate goal of intervening and changing the lives of as many children living with and dying from Duchenne limb-girdle muscular dystrophy, and other rare diseases as science will make possible.

And with that, let me turn the call to Ian Estepan, who will provide us an update on the financials. Ian?

Ian Estepan -- Senior Vice President, Corporate Affairs, Chief of Staff, Investor Relations

Thanks, Doug. Good afternoon, everyone. This afternoon's press release provided details for the third 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. Now starting with our net product revenue for the third quarter of 2020 from our products EXONDYS 51 and VYONDYS 53 was $121.4 million, compared to $99 million for the EXONDYS 51 alone for the same period of 2019. The increase primarily reflects higher demand for our products. In the quarter ended September 30, 2020, we recognized $22.5 million of collaboration revenue, which relates to our collaboration arrangement with Roche.

The co-development cost under the Roche agreement totaled $16.9 million for the third quarter and are included as a reduction to our operating expenses. On a GAAP basis, we reported a net loss of $196.5 million and $126.3 million or $2.50 and $1.70 per basic and diluted share for the third quarter of 2020 and 2019, respectively. We reported a non-GAAP net loss of $111.5 million or the $1.42 per basic and the diluted share in the third quarter of 2020, compared to a non-GAAP net loss of $84.4 million or $1.14 per basic and diluted share in the third quarter of 2019. In the third quarter of 2020, we recorded approximately $15 million in cost of sales, compared to $13 million in the same period of 2019.

The increase is primarily due to increasing demand for our products. On a GAAP basis, we recorded $190.4 million and $133.9 million in R&D expenses for the third quarter of 2020 and 2019, respectively, which is our year-over-year increase of $56.5 million. This increase is primarily related to a $42.5 million increase in manufacturing expenses, primarily due to the continuing ramp-up of our gene therapy programs. On a non-GAAP basis, R&D expenses were $159.9 million for the third quarter of 2020, compared to $110.5 million for the same period of 2019, an increase of $49.4 million.

The year-over-year growth in non-GAAP R&D expenses was driven primarily due to continuing ramp-up of our gene therapy. This -- we recorded approximately $75.4 million of expenses for both of the third quarters of 2020 and 2019. On a non-GAAP basis, the SG&A expenses were $57.2 million for the third quarter of 2020, compared to $59.6 million for the same period of 2019, a decrease of $2.4 million. The year-over-year decrease was driven by a decrease in professional services, primarily due to a decrease in reliance on third-party contractors as a result of an increase in hiring and headcount.

On a GAAP basis, we recorded $14.3 million in other expenses net for the third-quarter 2020, compared to $2.5 million in other expenses net for the same period of 2019. The unfavorable change primarily reflects the interest expense on our debt facility we entered into in December of 2019. And finally, we had approximately $1.8 billion in cash equivalents and investments as of September 30, 2020. And with that, I'll turn the call over to Gilmore for an update on our research and development activities.


Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

Thank you, Ian, and good afternoon, everyone. Doug has already shared the highlights from our most advanced gene therapy programs. I will, therefore, focus my remarks on Sarepta's presentation that this year's World Muscle Society Congress and the progress we've made in advancing our RNA portfolio. Let me start with the World Muscle highlights.

This year's meeting used a virtual format because of the ongoing COVID-19 pandemic. Nevertheless, we were able to present 16 posters that described data across Sarepta's RNA and the gene therapy platforms. From our gene therapy platform, the nonclinical and clinical data that we presented support the hypothesis that the rh74-MHCK7 construct we use generates durable gene product expression and durable physiological benefits in skeletal muscle. Dr.

Rodino-Klapac's lab demonstrates durable sarcoglycan expression, histological benefit and significantly increased resistance to contraction induced injury in the Tibialis anterior muscle in aged alpha- and beta-sarcoglycan knockout mice that have been treated, respectively without a sarcoglycan or beta-sarcoglycan transgene containing rh74 constructs. So, moving to our clinical data, we announced positive two-year functional results in support of our lead gene therapy candidate, SRP-9001 in Duchenne patients. The results demonstrated that two years after a onetime infusion of SRP-9001, trial participants exhibited a mean 7.0-point improvement in the North Star Ambulatory Assessment or NSAA, as compared to baseline. Please note and remember that at one-year post treatment, the mean increase was 5.5 points from baseline.

Thus, this functional data reflect a mean 1.5 point NSAA improvement between years one and two post SRP-9001 infusion. These data have been generated from four ambulatory participants aged four to seven in Sarepta's open-label trial, Study 101, and showed continued safety and tolerability of a onetime infusion of SRP-9001 at a dose of 2 to 14 biogenome per kilogram. We also announced positive clinical data for SRP-9003, our gene therapy candidate for limb-girdle muscular dystrophy type 2E. These results included 18-month functional data from three clinical trial participants in Cohort 1, the low-dose cohort and six-month functional data from three participants in Cohort 2, the high dose cohort, which was dosed at the same dose level we are using for SRP-9001 that is two to the 14th lined genomes per kilogram.

We demonstrated Cohort 1 that the three participants continued to show improvements from baseline across all functional measures, including the North Star Assessment for dystrophinopathies, otherwise, the NSAD in addition to time to rise, four-stair climb, 100-meter walk test and 10-meter walk-run test at 18 months. The mean NSAD improvement from baseline in Cohort 1 was 5.7 points at 18 months. In Cohort 2 at six months post infusion, all three participants demonstrated improvement from baseline across all functional measures. The mean NSAD improvement from baseline in Cohort 2 was 3.7 points.

This compares to a 3.0 point change in Cohort 1 at six months. Based on the safety, expression and clinical results we are moving the high dose four for future clinical development of SRP-9003. To sum up these presentations, we are very pleased that both therapies seem to be well tolerated. The clinical data presented at World Muscle from the micro-dystrophin, SRP-9001, 101 Study and the limb-girdle muscular dystrophy 2E, SRP-9003 101 Study, support durable functional outcome at the two-year and 18-month time points, respectively.

This further supports our hypothesis that because muscle, which is entirely differentiated tissue, it does enable a durable benefit following a single administration of the gene therapy. As you know, we are developing six additional therapies or six different therapies for the treatment of six subtypes of limb-girdle muscular dystrophy. We also presented nonclinical data at WMS, supporting the transition of our next limb-girdle construct limb-girdle MD2C into the clinic. Sarepta also presented data generated from its RNA platform.

These included long-term safety data for golodirsen from the 4053-101 clinical study supporting a safety profile. PK/PD data for casimersen from the 4045-101 clinical trial, demonstrating dose proportion exposure and an interim analysis of casimersen-treated patients in the ESSENCE pivotal study showing a statistically significant increase in skeletal muscle dystrophin expressions from baseline to 48 weeks. All in all, we were happy to be able to share so much data across our comprehensive portfolio with the academic, prescribing and patient communities. Beyond the data we shared at WMS, there have been numerous developments across our RNA platform.

The FDA review of our NDA for our lead PMO candidate, AMONDYS 45, is ongoing and from all accounts going well. Our PDUFA date stands at February 25, 2021, and the FDA, as indicated, does not currently plan to hold an advisory committee meeting to discuss the application. Moving to our post-marketing commitment study for exon 51, also known as the MIS51ON Study, we are pleased that we have enrolled part one of the dose escalation arm, and we are in discussions with the agency about the timeline for the duration of the study. Before I turn to our PPMO platform and the PPMO SRP-5051 program with 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. More recently, and based on in vitro results, we are planning to initiate a proof-of-concept in vivo study in collaboration with USAMRIID. Now turning to our PPMO development programs for Duchenne muscular dystrophy, the 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, or CPP, to a 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 [Inaudible]. We are pleased to say that we continue to escalate doses to levels higher than was originally planned at the initiation of this program.

This is because to date, we have not seen clinical results -- sorry, we have not seen clinical natural toxicity in our PPMO clinical trials and no new safety signals have been observed across all studies, thus enabling us to keep escalating doses in the MOMENTUM trial. We have now commenced dosing SRP-5051 in the 30-milligram per kilogram cohort. This year, we will be reviewing 12-week data from our 20 mg per kg cohort in Duchenne patients treated with our PPMO candidate, SRP-5051. We will be examining systemic PK, tissue penetration, safety and exon skipping efficiency data.

I remind you that our preclinical in vitro and in vivo models have demonstrated a robust correlation between the amount of exon skipping efficiency and the amount of dystrophin production. We presented SRP-5051 preclinical data at the OTS meeting in late September, in which the PK/PD of SRP-5051 was evaluated in non-dystrophic nonhuman primates. A single dose of SRP-5051 resulted in sustained exon 51 skipping that was maintained for at least 28 days. This sustained exon skipping supports the Q4 week dosing regime currently being studied in the clinic.

We also observed repeat at Q4 week dosing of SRP-5051 for 12 weeks, demonstrating a cumulative exon skipping effect that increased after each infusion. For SRP-5051, we will measure exon skipping efficiency by digital drop, PCR or ddPCR, allowing us to directly compare the efficacy of our PMO and PPMO candidates. Now 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 and point to demonstrate proof-of-concept that the CPP or cell penetrant peptide enhances muscle tissue exposures and thus enhances downstream exon skipping efficiency. So although the data we generate will not be representative of steady stage efficacy for SRP-5051, it will determine if we should move this technology forward with the urgency necessary to really meet the needs of patients with Duchenne.

Now whereas we are examining 12-week time points in the dose escalation part of the mentioned study, we will be using legacy 24-week data from PMO for some comparison. I will remind you that dystrophin accumulates over time, and so based on observations in prior PMO studies, we will expect to see higher levels of dystrophin at a later time point if the therapy is successful. Indeed, muscle biopsies at later time points are planned in the latter part of the MOMENTUM Study once we have selected our final dose for SRP-5051. Finally, I want to thank all of the patients, their families, study sites and coordinators, my R&D colleagues and our partners who have done so much work under difficult circumstances to maintain our urgent mission to deliver new, highly effective therapies to people with rare diseases within the context of the ongoing COVID-19 pandemic.

Now I will hand back to Doug for our Q&A. Doug?

Doug Ingram -- Chief Executive Officer and President

Thank you very much, Dr. O'Neill. Catherine, let's open the lines for Q&A.

Questions & Answers:


Thank you. [Operator instructions] Our first question comes from Brian Skorney with Baird. Your line is open.

Unknown speaker

Hi. Thank you. This is Jack calling in for Brian. So just one question really from us.

What do you expect to see from the 10-person clinical trial you're expecting to initiate? And will the data be strong enough to show consistent effect compared to the data that you have in Study 102. We know there's significant variability within the first four patients of data looking at the Phase 1 trials for the DMD product, and we're just wondering what the lower and upper bound of confidence are with respect to the dystrophin measurements that you're looking at for this new planned clinical trial. Thank you.

Doug Ingram -- Chief Executive Officer and President

Yes. Thanks a lot for that, Jack. First, let me answer the latter part of the question first. It is -- 10 patients is very sufficiently powered to obtain the data that we're interested in obtaining.

Let me explain what this is about. So let's go back. Remember, for some time, we had been thinking about a larger placebo-controlled trial using commercial material, and we'll still do that at some point as well. There was a number of reasons for that study, which is called the Study 301, of course.

One is global purposes. The other is additional confirmatory data. Remember, we're going to have Study 102 that's going to read out at the beginning of next year. So we're already going to have results from Study 102, both on safety and function of this construct, but the other thing that we would get out of Study 301.

This larger study, and acutely, the thing that we are most interested in to get as quickly as possible is validation of that commercial material. This is the same construct, but generated using a different process and a different manufacturing process, as you know, in iCELLis units and so what we had planned with respect to 301 is to get what we really are most interested in right now, we would do an interim analysis of a subset of those type of patients around the same subset is what we're actually going to do with 103 early next year. So we would start that study, but we would actually get the real benefit -- we're most interested in right now, which is the validation of our commercial material, both on expression and on safety early next year. And then, of course, in the summer, as we thought this through, and we watched the external environment.

We thought about the risks associated with a larger study, the potential risk to patients for another placebo-controlled study as one example in the midst of a pandemic and exposure from the pandemic as well as just the risk of the program and then the execution of the program, it dawned on us that there is a far more efficient way in the mix of this pandemic to get exactly the same information, and that's where we devised the concept of Study 103. So there is no reason to do a placebo-controlled trial for the expression that we're looking for in for the safety that we're looking for, and we've always known we only needed about 10 patients or so, eight to 10 patients to get that information. And so rather than getting that information out of a larger study at a large multi-center, multi-country study, we should also propose a much leaner approach, which is Study 103, and that's exactly what we did. And as you know, we went through the agency in writing in September with this proposal.

We -- and the big issue that occurred, of course, in the September meeting, which is, I think, we all know, was all on the written record was that the agency had questions and ultimately, had issues with the approach that we were taking to the clinic to release [Inaudible] for our Study 103. That could've taken us a long time to resolve, frankly. It could could've taken us somewhere in between two, three and also maybe four, six months, just to get an answer from it. I'm really pleased to say that the Division was willing to meet with us informally.

They met with us in September, so we're very clear about that. We came to an alignment in September. They agreed to our updated approach on the potency assay. We've spent the time since late September, doing two things at the same time, of course: finalizing the meeting minutes of that discussion with the agency, but also we're gathering the data for our alternative approach to the potency assay.

And as we sit here today, that, that is all done. The minutes are done and we should be able to start 103 by the end of this year, which means sometime in 2021, really on the earlier side of 2021 hopefully, fairly close to one another, we should have both a read-out of Study 102, which will show us both the efficacy, the functional benefits in a well-controlled placebo-controlled trial, blinded trial from 102 with respect to SRP-9001. And we'll have safety read-out from that study as well, and we'll have the commercial validation results from Study 103, which will have micro-dystrophin expression results. We'll have safety results out of that study at the 12-week time point as well, which of course, is a crucial time point from a safety perspective.

We also will have a wealth of additional CMC-related information, much of this -- which we've already gathered. And at that point, of course, we will sit down with the agency and talk about the steps forward. The one thing we all know, of course, all of us are well aware, that in the United States and then around the world, every single day, and the thousands and thousands of children wake up in the morning with Duchenne muscular dystrophy. And throughout that day, they degenerate.

And unfortunately, every week and every month, some percentage of those kids die. So with all of that information available to us, we'll sit down with the agency and figure out what is the fastest approach to getting this therapy to the kids that are now waiting. And hopefully, at the time we have that and of course, the studies have to -- the scientists have to cooperate and studies have to come out the right way. We should be able to sit down with them armed with information that shows, if we're successful, that the SRP-9001 is now safe and effective and the commercial material has been validated along with the clinical performance that we'll see out of Study 102.

So thank you for your question.


Thank you. Our next question comes from Tazeen Ahmad with Bank of America. Your line is open.

Tazeen Ahmad -- Bank of America Merrill Lynch -- Analyst

Hi. Good afternoon, guys. Thanks for taking my question. Doug, just to stay on topic with Study 103, so I just want to make sure I understand this.

You were not doing a specific potency assay, but you are using Study 103 in lieu of a specific potency assay? Just want to confirm that. And then secondly, do you still have to do Study 301? Is it still part of your assumption that you will need to submit Study 2 plus now Study 103 and some data from 301 or is it your plan to go talk to FDA to see if you can apply, assuming that the Study 102 and 103 show what you want those to show and not necessarily have to do 301?

Doug Ingram -- Chief Executive Officer and President

Yes, thank you for the question, Tazeen. Let me clarify something that I might have created to confuse you about. Now study -- we have come to an understanding with the agency on the assay approach to Study 103. As you may recall, what we said at the time that we had the Type C meeting, we had an approach that we thought was appropriate with respect to the potency release assay for Study 103.

The division had questions and disagreed with this approach that we were taking. We didn't have clarity on precisely what that -- those issues were, and if we had gone through a formal process, it could literally have taken two to six months to ultimately have enough clarity to know where to go. By the end of September, we had an informal meeting with the agency. We understood much better what their issues were, and we were able to quickly propose, essentially, a new potency approach that they found acceptable.

And we then gathered the data for that. That's already done and completed. And so we have the potency approach now for Study 103, and that allows us, of course, to start Study 103. With respect to Study 301, so -- and Study 103 will get us exactly what we wanted out of an interim analysis from Study 301.

So really, it is a very thoughtful approach to a pandemic, to gather the information through Study 103 and get the most salient information in the near-term when we get 102 read out, which is that in patients and in biopsies, we could show that as we would predict from the -- all of our CMC work, the material in Study 103, from a commercial material perspective, will perform in a similar way, both from micro-dystrophin expression and also from a safety perspective. With respect to Study 301, there's still lots of reasons to conduct Study 301. First and foremost, that is a global study. We have global ambitions.

It is our goal to get approvals around the world. And of course, Study 301 will provide us with that opportunity. The second thing to know, of course, is that it'll be really additional confirmatory evidence that, regardless of the pathway forward in the United States or anywhere else around the world, will be additional information that would be valuable as we were bringing this therapy out to patients. And so the third, of course, is we need to -- I don't want to -- I want to be very clear about something that I think I've said many, many times before, which is that we are going to sit down with the agency and talk about the pathway from a regulatory and development perspective, once we have the read-out from Study 102 and now the read-out from this commercial material validation of Study 103, and we've been very clear about this.

I remain resolute that this is the right approach. We explicitly said we weren't going to discuss this with -- in advance of the Type C meeting and after the Type C meeting. So we'll get into next year. We'll get the results of 102.

If it's successful, we'll have shown in a well-controlled trial that our therapy is -- it creates functional benefits, and it's doing good for these kids and that it's safe. We'll have the commercial validation material out of Study 103, hopefully not far after that. And then with all of that, armed with that, we will sit down with the U.S. FDA and the Division and talk about what -- in light of the quality of the data and the data that we now have, what is the most appropriate and the fastest way to get this therapy if it's safe and efficacious to kids that are waiting for it in the U.S.

That's a conversation, obviously, we'll have after we get the data in hand.


Thank you. And our next question comes from Alethia Young with Cantor Fitzgerald. Your line is open.

Unknown speaker

Hi. This is Emma on for Alethia. So given the agreement here with the FDA on Study 103 is kind of a leaner approach for getting the most important information for 9001 in the midst of COVID, is there -- do you expect kind of similar flexibility for 003 and LGMD to report a streamlined pivotal program?

Doug Ingram -- Chief Executive Officer and President

We won't know that until we have conversations with them, obviously. So I don't want to make any presumptions. One of the things that we said is that we need to do a couple of things with respect to -- this is a [Inaudible] girdle in this -- in 9003. We have to do it to get the GMP material completed and released.

We're still working on that. We've actually got the process development done, and we're running a process development run, but we have a lot of analytical work to do as well. Obviously, the work that we've done recently with the agency is going to be very helpful to 9003. And then with that in hand, we want to sit down with the agency and talk about now next trial and then hopefully, what we would like to believe will be our pivotal trial for 9003, but we'll give an update on that early next year once we have that framed out.

Obviously, as we've seen, given the pandemic and how busy the agency is, the opportunity is to have discussions with the agency are formal, and we've got to be very thoughtful about that. So I want to make sure we have the right kind of information in hand when we have those discussions, and then we can give a better view on what the pathway forward is for 9003.


Thank you. Our next question comes from Anupam Rama with JP Morgan. Your line is open.

Anupam Rama -- J.P. Morgan -- Analyst

Hey, guys. Thanks so much for taking the question. I'm sorry, I'm just a little bit confused. So for Study 103, looking at safety and some of the assay comparability work, right? And I think that's at 12 weeks, and you called that part one.

So are there additional parts to the study where you'll be looking at more functional type of measures? And as the FDA indicated, any sort of interest in seeing that before you have some of the regulatory discussions, right? Thanks.

Doug Ingram -- Chief Executive Officer and President

Yes, it's a great question. No. Look, this is an open-label study. The goal of SRP -- goal of Study 103 is to get essentially exactly what we would have gotten out of an interim analysis for 301.

It is essentially precisely the same thing. The reason I mentioned that it's part one is, of course, in these studies, you'll have longer-term safety follow-up. And so in that sense, the study maintained, even after you get the primary readout on it. It's the same with all of our studies.

It's the same with, frankly, Study 102. We'll have Study 102. We'll have -- Study 102, we'll have the readout from a primary analysis, one-year analysis that will be when the kids crossed over, etc., we'll look at the results, and we'll see if as we have hypothesized, we'll see a statistically significant and meaningful improvement in NSA versus placebo. And then here, of course, that study will continue.

Those kids will continue to be monitored from a safety perspective. So that's what we mean when we say part 1, part 2. But the goal of Study 103 is essentially to get, in a sort of a more straightforward leaner way, the information that we were hoping to get out of an interim analysis of Study 301, which was the previously proposed placebo-controlled trial.


Thank you. Our next question comes from Gena Wang with Barclays. Your line is open.

Gena Wang -- Barclays -- Analyst

Thank you for taking my questions. So maybe, Doug, just wondering, what is the criteria for approval? Will Study 102 plus Study 103, would that be sufficient? And also, did the FDA already sign off the potency assay? And do you still need to do the release assay in order to get approval? And in the case, you have to run Study 301, do you need to run another potency assay for Study 301?

Doug Ingram -- Chief Executive Officer and President

So the FDA signed off on our approach for the release assay, for Study 103. I can't tell you what the requirements will be precisely for an approval because I think we've said a number of times in the past, we've all talked about it, and we're not going to broach that issue and have those discussions with the agency until we have the data in hand. So let's get through Study 102. Let's see how compelling the matter looks from 102.

Let's get through our commercial validation study. Let's see how compelling the micro-dystrophin expression and safety looks there and then we'll have that dialogue with the agency and then figure out what the appropriate pathway forward is, as you can imagine, we feel a great sense of duty and obligation to move as fast as possible. So you should envision that we are going to certainly propose the leanest approach forward. But that discussion will happen once we have the data available to us.

On the -- as it relates to the release assay, and this is the release assay. I want to be very clear, if I'm not making it clear. We haven't approached the potency release assay. We've had the blessing of the agency on that approach.

We have the data for that available and that's -- so that's why we have to get to start the study, and that's why we get to move forward, and we've been able to essentially unpause what was a pause part of this program. That approach could certainly, when we get around doing additional studies, 301's only going to be the other ones, we want to do a non-ambulant study as well. We could obviously very likely use this same potency release approach or we could use an updated one. As we've said many times in the past, one of the reasons we were so confident that -- we weren't confident on the timing at the time of when we would be able to get this issue resolved with the agency.

We were always very confident that we would get it resolved and one of the reasons for that is that we have a number of assays that can be potency assay. So we'll use the most optimized one at the time that we start additional studies, but certainly, one of the possibilities is to use this potency assay that we're using for Study 103 because we've obtained the concurrence from the agency on that one right now.


Thank you. Our next question comes from Joseph Schwartz with SVB Leerink. Your line is open.

Joseph Schwartz -- SVB Leerink -- Analyst

Thanks very much. I was wondering, have the sites that you were going to use for 301 led to the -- did they lead you to believe that it was going to be challenging to execute the trial? And how do you feel about their ability to collect and evaluate data from 102 in this arm?

Doug Ingram -- Chief Executive Officer and President

So the interestingly enough, the short answer to that question would be, no. Thankfully, they're not coming back to us and saying that this would be challenging. But to be honest, there is an extraordinarily high motivation from sites to participate in our studies for the SRP-9001. It is certainly -- and I think people are very excited about the potential for what this could mean for children with Duchenne muscular dystrophy.

And I can think of situations where even in the raging portions of the pandemic sites where -- even when sites were currently shut down, and we're suggesting they could really start immediately. So the truth is we had to make an independent assessment of this issue, and it really was this discussion really comes down to this. We want to start 301, we certainly do. We have a global ambitions.

It will be very important globally. And it will be good in the U.S. as well. I don't think it's just global.

But the thing that we really acutely need is that right after we have Study 102 results, we need to have results from a commercial material validation study that shows that in patients that we're getting the same kinds of micro-dystrophin expression, and then we have the same safety. We have a lot of CMC work done that tells us that should be the case, and we feel very good about it, but we need to confirm that. And as we thought about that, what about this in the summer time, frankly, we realized that we could do this as an interim look in a larger study or we would simply adapt ourselves to the environment we're in and really create a lean study that gets us information to validate the commercial and material from an expression and safety perspective directly. So, let's just do it directly and start a study with the right number of patients and execute rapidly.

And that's how we kind of landed on where we were. With respect to Study 102, we are very confident about where we are. The team has done a brilliant job. I wouldn't give so much credit to a lot of people.

Our clinical team, our clinical operations team have just done a bang-up job of dealing with this -- with what is certainly, at the beginning, were enormous challenges associated with this pandemic and how to adapt to it. I want to give also an enormous amount of kudos and congratulations and thanks to our clinical investigators, both at UCLA and then as mentioned, right, Children's Hospital. And I want to give just -- I can't begin to describe how much I'm thankful to Dr. Jerry Mendell, our principal investigator, who tacked this issue with an enormous passion to make sure that this study is staying on track in these kids were able to still come in and get their functional readouts and the like and that the study is tracking forward.

So I stand -- we stand here today, notwithstanding the pandemic and notwithstanding even the potential for the second wave. We're really confident as it relates to Study 102 that we'll have last patient, last visit in December, and we will have a readout in the first quarter of next year. I'm very confident about that. And it also relates to the fact that it is -- while it's a larger study than our study 103, it is only two sites.

And so, it certainly comes down significantly to the passion, hard work, and commitment of Dr. Mendell and his entire team.


Thank you. And our next question comes from Colin Bristow with UBS. Your line is open.

Colin Bristow -- UBS -- Analyst

Thanks for taking the questions. So first one, I understand the agency hasn't fully blessed the faster market, but in terms of the willingness to accept this eight to 10 patients is sufficient evidence of comparability between the commercial and clinical product. Have they signed off on that? And then just quickly on the timing of Phase 3 initiation of 301. It seems like 103 is going to ring sensitive commercial to clinical products question.

And just, I guess, why not initiate the 301 even in parallel or as soon as you can get the results of the potency assay issue ironed out? Thanks.

Doug Ingram -- Chief Executive Officer and President

Yes, a couple of things. One, I want to be very clear about this. We have not had discussions with the agency about the sufficiency of evidence to support a BLA. We've been absolutely on purpose.

And I think I said long in advance of the Type C meeting that we explicitly don't want to have those discussions until we have data available. I don't want to -- with a very busy agency, I don't want to have those kinds of discussions at theoretical level. I want to have it with data in the hand. With respect to 103, 103 was designed originally because of the pandemic, to get us the information we really need in the leanest way without risk to the program, and more importantly, without placing risk on children, including the placebo kits, who would be having to go into doctors' offices with the potential of being on a placebo.

So there's a number of reasons why we talked to the agency and formally, we talked about the commencement of Study 103. The reason we are -- we have proposed to the agency, actually, that we would consider a commencement of Study 301 once we had 103 in hand, and they concurred in that concept. There's a lot of reasons for that: one, it's the leanest way to get to the information that we're most interested in now, and it allows us to focus on the information that we want most acutely, which is the validation of the commercial material, which will marry up with the results of Study 102 and then have discussions with the agency; two, it reduces the risk to the program and to the patients along the way; and then three, of course, we might learn step from 103 that could really inform the way we designed 301. And so it made sense to us to really focus on study 103.

That's what we focused on in our informal discussion with the agency, and so that's why we got the blessing to commence.


Thank you. Our next question comes from Brian Abrahams with RBC Capital Markets. Your line is open.

Brian Abrahams -- RBC Capital Markets -- Analyst

Hey, guys. Thanks for taking my questions. So can you clarify, is FDA sign off on the actual potency data that you've generated using the agreed-upon approach dating for the start of study 103 or for ultimate approval? And how do you view, I guess, the difference in potential risk-based on your discussions and any regulatory precedent, to seeking approval using open-label rather than placebo-controlled safety and expression data from commercial scale material? Thanks.

Doug Ingram -- Chief Executive Officer and President

A couple of things. We have the data in hand. So we feel very confident that we're going to commence the study. We don't -- we don't believe that we should -- that there are gating items other than just getting it executed to commence study 103 from our viewpoint, given what we're looking at for the commercial validation study, the fact that it's open-label versus placebo really isn't significantly relevant.

We're looking at expression. Obviously, you can't placebo -- you can't get us a placebo effect on expression and we're looking at safety as well. So we don't think that's going to be a significant delta but we haven't had the conversations about the pathway to approve and we'll have those discussions at the appropriate time of the agency with data in hand. You we'll know it when we know it.


Our next question comes from Ritu Baral with Cowen. Your line is open.

Ritu Baral -- Cowen and Company -- Analyst

Thanks for taking the question. Doug, help me understand this. What assurance for, I guess, general messaging you have from FDA that the 10 patients that you're thinking about the 103 is going to be large enough to capture the potential variance of the commercial product and the safety of the commercial product? Just given what we've seen with the size compound and complement in some patients and not others, what do you have in writing from the Type C meeting on the end of 10? And also, now that you have, it sounds like you have the potency assay settled, what you're going to use, having it validated, do you have all the clinical product you need to potentially run 301? I just -- I want to make sure that you're not going from 70 to 10 because of a lack of validated product.

Doug Ingram -- Chief Executive Officer and President

Yes, that's a really good question. Thank you for asking that. We do not have a material shortage problems. So that -- thank you, I hadn't even considered that someone might have imagined that would be the case.

We have done many GMP runs. We have sufficient material to start Study 103 and 301, etc., an non-ambulatory studies as well. So none of this relates to the unavailability of materials. So thank you, actually, for giving an opportunity to say that ahead and considered that someone might have worried about that.

The issues -- the decision about the size of the study is ours. It really isn't the FDA's. The division doesn't essentially bless it or confirm that they agree with the analysis that we're now doing and they haven't, and we wouldn't have asked them to do that because really, it's -- that is our decision. I mean the risk around the size of the study is ours.

I will note, however, that the size of this study is essentially exactly the size of what we would have envisioned in an interim analysis for Study 301. So this is essentially the same thing we were going to do with Study 301 in the form of an interim analysis on a subset of patients. We were kind of looking at the 8% to 12% range, and that's where we are now right now, at eight to 10 patients in the study and that relies on our own evaluation, and we are confident about the size of the study, and we're confident about the fact that we'll get insight and validation around commercial material, both expression and safety out of it. You did touch on something.

And I just -- you are well aware of this, but I'll say it yet again. Just so we're absolutely clear, we have -- I know that other programs have seen very troubling issues, for instance, the combo and media A, whose the -- like, we've never seen those issues, and so there's really no reason to believe that we would see anything like that in -- out of our commercial process materially either. And so we're very confident about that, but that's never been an AE that's appeared with any of our rh74-driven program. So I just wanted to make that very clear.

Thank you for your question.


Our next question comes from Vincent Chen with Bernstein. Your line is open.

Vincent Chen -- Bernstein Research -- Analyst

Hey. Thank you very much for taking the questions. A couple from me largely following up on just clarifying some things. Sorry if they were asked before.

First is really just to clarify, is there specific data you're waiting for from Study 103 that gates the start of Study 301 or in a non-COVID world, could you go ahead and start study 301 if you wanted to since the FDA sort of aligned on the potency assay? And then I have a follow-up after.

Doug Ingram -- Chief Executive Officer and President

We wouldn't -- Yes. Thank you. I mean we proposed study 103 because of the COVID issues. And frankly, to be honest.

It's only -- we proposed this in the summer, it's only become more compelling recently. It does appear, unfortunately, certainly in the United States, and probably around the world right now that the second wave is upon us. It only becomes more compelling to us. When we met with the agency in our informal discussions at the end of September, we proposed and they concurred in the concept of a Study 103 and we actually proposed that we'd start 301 after we had data from 103, and they concurred in that.

So that's the decision that we made in light of where we are then, and we think it makes tons sense. And there might be something that we learn out of 103 that could actually inform 301, but we're very focused on getting the information that we're most interested in right now, which is validating our commercial material, both in expression and safety in children with Duchenne muscular dystrophy and having that information as soon after the readout of the Study 102 and hopefully as close in time as possible so that we have together, at the same time, essentially three things. We have this new -- from a placebo-controlled trial, we have the compelling evidence that the therapy is providing significant benefits to these kids. And I believe if we're right, statistically significant and clinically and uniquely and that we have safety data.

And then, of course, with respect to the commercial material, we'd have commercial validation that we're getting the kind of expression we would expect out of the commercial material. We're seeing the same safety signs out of the commercial material we will see out of the Study 102, at least, what we've seen certainly so far. So we're really focused on that. And then we'll have a discussion with the agency, and we'll talk about the pathway to getting this therapy to these kids who are, as we all know, also waiting and time is not on their side.

So we'll certainly meet with the agency as soon as we have that information available to us.


[Operator instructions] Our next question comes from Tyler Van Buren with Piper Sandler. Your line is open.

Tyler Van Buren -- Piper Sandler -- Analyst

Hey, there. Thank you. Good evening. I had a question on the Momentum trial read out by year-end.

I was just hoping you could refine expectations there. I think you mentioned specifically you're going to compare digital drop PCR, the 12-week PPMO data to 24-week PMO data. So is the ultimate goal at this initial readout to see -- skipping that is higher with the PPMO product at the early time point or are you hoping to see several magnitudes higher of skipping? Could you put a finer point on that? And are you dose-escalating because maybe the skipping is not where you would like to see it yet, and you think by getting to 30 or 40, you'll be able to achieve that ultimate goal, even though it appears that you're seeing great safety?

Doug Ingram -- Chief Executive Officer and President

Yes, there -- so I would say, first of all, don't read much into -- don't read anything into my comments that -- about what we've seen because we haven't. The reason we haven't released the 20 mg per kg data is we don't have the data in hand right now. So I'm going to turn this over to Dr. O'Neill to provide some color on what we're going to look at between the two.

I'd say a couple of things. It would be nice to see some additional exon skipping even though it's in a different time point between the PMO and the PPMO at 20 mg per kg, but the reason that we're dose-escalating is because it has always been our goal. The goal is to get to the highest possible dose we can get to here before we see a dose-limiting toxicity, so that we know that we are creating the most excellent skipping in district when possible. And we've seen, in the animal models, that, that's -- that if you push the dose high enough and you can do so safely, that at least, if the animal models bear out, you will see a very substantial, eventually, very substantial increase.

Where that exactly is, it's -- correlating between humans and animals is always difficult as the diametric dosing, and it's a late based dosing. You get some hybrid between the 2. So we don't know yet, but we're very confident in the mechanism of action. And so I will say, one more thing that I'm going to turn it over to Dr.

O'Neill to give more data-driven color on this. This is all about safety for us. We're really -- what we're curious of seeing is one of the safety signals, the 20 mg per gig led to the chance we can go to 30 mg per kg. And then from 30 mg per kg, what's the chance that we can go to 40 mg per kg to make sure that we get to the optimal dose for these kids.

With that said, Dr. O'Neill, you have additional color on this?

Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

Yes, thank you very much. So I think the key thing to say is that the Momentum study has two objectives: The first is to test our hypothesis that the cell-penetrating peptide fused with the PMO significantly enhances tissue, cell and ultimately, nuclear penetration to enable higher levels of skipping and dystrophin expression, as we have seen in the nonclinical data. And the second objective, this is what's really driving your question, is that we want to maximize the benefit risk. And so if we can continue, notwithstanding a proof of principle in the human of higher exposure, notwithstanding seeing that, when we should see it we would still want to push the dose as high as we can considering the severity of this disease so that we can get a maximum tolerated dose and so maximize the benefit.

So I -- so that is the key reason for dose escalation. I will -- and yes, I will reemphasize that what we will be examining is systemic PK, very importantly, to the proof of principal tissue penetration in addition to ongoing safety as well as exon skipping data. Thank you.


Thank you. Our next question comes from Danielle Brill with Raymond James. Your line is open.

Unknown speaker

Hi. This is Danil on for Danielle Brill. Thank you for taking our question. Staying on the topic of PPMO.

Good question in terms of dystrophin expression. As your dose-escalating, are you collecting the dystrophin expression data? And do you have any idea of when you might be able to share with that? Thank you.

Doug Ingram -- Chief Executive Officer and President

We're going to have -- we'll have an update on the 20 mg per kg at 12 weeks, Dr. O'Neill, you're going to correct me if I misdating it. So 12 weeks with the PPMO. We're actually going to compare it, ironically, to the PMO at 24 weeks, but we're going to have an update on exon skipping and tissue exposure at 12 weeks.

We're not looking for dystrophin discipline now for the simple reason that it is very early. You can get some lead of exon skipping in an early day, but one of the things we know with certainty is that dystrophin barrels over time and 12 weeks is very early to start looking for dystrophin. We've seen and with our PMOs and EXONDYS as an example, we've seen a significant difference in the amount of dystrophin that was produced literally at four years versus one year, so I'm not saying it will be that long, but 12 weeks is a little bit early to look for dystrophin. With that, Dr.

O'Neill, so do you want to add any additional color on that?

Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

I can confirm first that what Doug has said is entirely accurate. And the only thing I will remind you of is that we have demonstrated non-clinically that there is a proportionality between exon skipping and dystrophin expression downstream. But as Doug has said, 12 weeks data is very early for dystrophin expression. And as we have learned in the past with PMO, dystrophin does accumulate over time.

And so that is the reason that from a proof of concept and a proof-of-concept point of view, we are focusing on, first and foremost, tissue distribution or tissue concentration as well as exon skipping at 12 weeks.


Thank you. Our next question comes from Salveen Richter with Goldman Sachs. Your line is open.

Unknown speaker

Hi. This is Sonya on for Salveen. Where do you stand with longer-term strategy on DMD with next-generation approaches unique -- using genomic medicine?

Doug Ingram -- Chief Executive Officer and President

Dr. O'Neill -- first of all, I'll say this, we are, of course, in addition to our gene therapies in addition to are both our PMOs right now and our next-generation PPMO, which we've just touched upon on. And in addition to gene editing, and that I should tell you, we have a Gene Editing Innovation Center in Durham, North Carolina, under Dr. Charlie Gersbach, that's looking at a number of different things, including this potential for gene editing and Duchenne, although I think it's a bit out -- a bit off in the distance.

There are a number of other ideas that we're looking at as well, and I don't really think it'd come to fruition yet, but if you have any additional thoughts on that, Dr. O'Neill.

Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

No, I think you said it nicely. The key things are that we actually have a nice portfolio of platform approaches with gene delivery, gene editing and our antisense and I think that choice -- certainly, when I think about gene delivery pending gene editing for revolution and our antisense enables patients to make optimal choices, we hope, in the future. And obviously, they complement one another. And so I think, as we've said before, you remember, we are even looking at the possibility of combinations of these therapies in the context of really optimizing therapies for patients with Duchenne.

And I will actually also highlight what Doug said earlier, is that, obviously, the other thing we want to do with our portfolio for Duchenne patient is ensure that we develop the data for patients at various stages of this disease just to make sure we can maximize benefits for as many people as possible. Thank you.


Thank you. Our next question comes from Joel Beatty with Citi. Your line is open.

Shawn Egan -- Citi -- Analyst

Good everning. This is Sean Egan calling in for Joel. Circling back to 9001, it seems like not much has changed today in regards to what you guys hope to expect to eventually file with that being a positive 102 study and positive bridging results. But maybe can you confirm that there's nothing new from the FDA discussions, which suggest that a positive 102 and positive bridging study alone would not support approval? And also, has blinded 102 data helped inform the design and scale of Study 103 in any way?

Doug Ingram -- Chief Executive Officer and President

I'd say Study 101 really has informed both 102 and 103, and again, just lingering for a moment, I want to make sure I'm very clear about the fact that we haven't had no discussions with the agency to solicit their views on what the data would be necessary for this, for instance, the BLA side, I don't want to mislead. I mean I do agree with you that we are in the same position with our approach today as we would have been -- as we were thinking about study 301, which was an interim analysis. Probably the one difference -- and there's two differences: one, if you roll the clock back, of course, we were under the impression that the potency assay approach that we had as we tracked to the September meeting was appropriate, and that has caused us a couple of months delay as we sorted that out and got new data. That's a difference, but I must say, in fairness, and I really want to give tons of kudos both to the teams and the willingness of OTAT to talk to us, we were able to resolve those issues in advance of 103 as fast as I think it is possible to do.

I think in light of how busy that agency is and in light of pandemic, I think the ability to actually get an informal meeting with the division so soon after our Type C meeting and to come to a resolution, literally by the end of this September, I think, has said a lot about the team and its willingness to execute and its execution ability and it also said something about the division's willingness to have conversations with us, and I really appreciate that. I think the one additional thing that I'd say between where we are and where we might have been before is that, I think 103 is -- has 103 is a much higher -- will get us data much quicker, I think, than even with an interim analysis of 301. So I think in many regards, we will get the information that will give us insight on our commercial material with a higher TOS in the middle of a pandemic and faster than we would have done with some larger studies. Other than that, I think the approach is the same.

If all goes well, it may be a 60-day delta between what we would have anticipated in the summer and where we are today, we should have both the results from our Study 102 that show the functional benefits of this therapy and safety signals and tolerability, and we should also have the result of this commercial validation study, and the same information we would have hoped to get out of the interim analysis for Study 301 on both micro-dystrophin expression as well as safety. So I think the one caveat that we're probably 60 days delayed versus where we were, where we thought we would have been. So let's say, in July of this year, we're basically in the same position with the same kind of data by early next year.


Thank you. Our next question comes from Hartaj Singh with Oppenheimer. Your line is open.

Jackie Yan -- Oppenheimer and Company Inc. -- Analyst

Hi. This is Jackie Yan for Hartaj. Thanks for [Inaudible]. Maybe just a clarification question.

Since you noted in your earlier prepared remarks that you're going to pursue additional studies in older and non-ambulant boys, could you just clarify whether those will be separate from Study 301 or cohorts added to that study. Thank you.

Doug Ingram -- Chief Executive Officer and President

There would be a separate study on non-ambulatory patients, at least that's the way we envision it today.


Thank you. Our next question comes from Difei Yang with Mizuho Securities. Your line is open.

Difei Yang -- Mizuho Securities -- Analyst

Good afternoon. Thanks for taking my question. So just asking a clarifying historical question. So back in the days before all these discussion on potency test started, when the original plan was to go to the FDA with study -- with results from 102 plus interim readout on 301, did the agency -- did the FDA ever confirm that will be enough to form the basis for BLA submission or was it at the point just to enough to start the conversation about potential submission?

Doug Ingram -- Chief Executive Officer and President

It's always been our strategy. I think we've communicated this often, that we will have those discussions with the division once we have data in hand. And that until we have data in hand, we won't have discussions about the level of evidence necessary for a BLA for the simple reason that I think it's asking a lot of the agency in the middle of all -- how busy they are the pandemic and the like to have theoretical discussions. We'll get 102 executed, we get the results of 102.

We'll get the material from our commercial validation study and then we'll talk to the agency about the path forward with the initial studies or a BLA or the like. I think everyone knows what our goals are in that regard, but we've been very consistent in that perspective.


Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your line is open.

Max Skor -- Morgan Stanley -- Analyst

Hi. Thank you. This is Max Skor on for Matthew Harrison. Just a quick question.

Is Study 103 gating at all for initiating discussions with the agency regarding the limb-girdle 2E trial with commercial material or specifically, is there any read-through from one potency assay to the packs?

Doug Ingram -- Chief Executive Officer and President

I don't think there's any gating -- there's no gating item between Study 103 and then discussions with the agency about limb-girdle 2E, no. I think that there is no doubt that the discussions we've had with the agency about the potency assay approach will inform the approach we take with limb-girdle 2E. So we're in much better shape as a result of those in discussions. We can apply those -- that learning to the limb-girdle 2E without a doubt.

The real gating item to have discussions with the agency on limb-girdle 2E limit is also getting the GMP material released and that requires us to continue to do analytical work and asking work that is well on the way, but not completed yet.


Thank you. Our next question comes from Martin Auster with Credit Suisse. Your line is open.

Matt Terwelp -- Credit Suisse -- Analyst

Hi. This is Matt Terwelp on for Marty. Thanks for taking the question. I just wanted to confirm the Study 103 is a single center study? Thanks a lot.

Doug Ingram -- Chief Executive Officer and President

I believe it is, but I'm going to let Dr. O'Neill confirm that for us.

Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

The final number of sites will actually be determined as we execute the study. We have actually built in redundancy because as Doug had said, one of the things we did, and the reason that we actually conceived the 103 study was to actually handle the -- or to deal with the consequences of the ongoing pandemic. And so we have actually built in a flexibility so that we are able to enroll and execute the study as quickly as possible.


Thank you. There are no further questions in the queue. I'd like to turn the call back to management for any closing remarks.

Doug Ingram -- Chief Executive Officer and President

Thank you all very much for joining us this evening. I will stand very pleased with the team for continuing to execute during this pandemic, both in the performance of our proof of therapies as well as our development programs. As it relates specifically to SRP-9001 is, of course, the top of mind for Sarepta because it is such an important program for Duchenne muscular dystrophy boys that are waiting. We did have a setback in September as one could expect to have when they're ambitious.

We had hoped that in September that we would have a blessing to commence Study 103 in September, but I'm very pleased to say that we executed consistent with our culture. We moved rapidly. We were able to get an informal meeting with the agency at the end of September. We were able to come to a meeting of the minds on the commencement of Study 103 and the potency release assay associated with Study 103.

We could not have, I think, envisioned a faster resolution of this issue so that we can commence with little delay the next commercial validation study in an extraordinarily important program. And so I'll continue to update people across the course of the year as we execute but I want to give a lot of thanks to OTAT certainly for their willingness to engage with us and help us find the pathway forward. I want to thank the Sarepta team for their willingness to continue to execute fiercely across the organization and serve these patients. And I, of course, want to thank the patients, both those who participate in our trials as well as those patients across all of our disease states, both Duchenne muscular dystrophy and currently limb-girdle muscular dystrophy for their commitment to the programs, but also for staying with us as we continue to execute these programs.

We have a single-minded goal as an organization. Our success will come from our ability to intervene in the lives of patients who are suffering from these rare diseases in far, far too often with respect to Duchenne muscular dystrophy and these limb-girdles being stolen from their families by a shorter life because of these diseases, and we will work like mad to move as fast as possible. And I do believe that Study 103, that which we proposed and was accepted by the agency is going to be an enormously important step forward in our ability to accelerate the therapy to patients that are waiting as soon as the science cooperates. With that, have a lovely evening, and we will continue to update across the course of the year and into next year.

Thank you.


[Operator signoff]

Duration: 89 minutes

Call participants:

Mary Jenkins -- Senior Manager, Investor Relations

Doug Ingram -- Chief Executive Officer and President

Ian Estepan -- Senior Vice President, Corporate Affairs, Chief of Staff, Investor Relations

Gilmore O'Neill -- Executive Vice President, R&D, Chief Medical Officer

Unknown speaker

Tazeen Ahmad -- Bank of America Merrill Lynch -- Analyst

Anupam Rama -- J.P. Morgan -- Analyst

Gena Wang -- Barclays -- Analyst

Joseph Schwartz -- SVB Leerink -- Analyst

Colin Bristow -- UBS -- Analyst

Brian Abrahams -- RBC Capital Markets -- Analyst

Ritu Baral -- Cowen and Company -- Analyst

Vincent Chen -- Bernstein Research -- Analyst

Tyler Van Buren -- Piper Sandler -- Analyst

Gilmore ONeill -- Executive Vice President, R&D, Chief Medical Officer

Shawn Egan -- Citi -- Analyst

Jackie Yan -- Oppenheimer and Company Inc. -- Analyst

Difei Yang -- Mizuho Securities -- Analyst

Max Skor -- Morgan Stanley -- Analyst

Matt Terwelp -- Credit Suisse -- Analyst

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