Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics Second Quarter 2021 Earnings Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer 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.
Thank you, operator, and thank you all for joining today's call. Earlier today, we released our financial results for the Q2 2021. The press release is available on our website at serenda.com and our 10 Q was filed with the Securities and Exchange Commission earlier this afternoon. Joining us on the call today are Doug Ingram, Ian Estipan, Balan Murray, Doctor. Gilmore O'Neill and Doctor.
Louise Rosino Klapac. After our formal remarks, we'll open the call for Q and A. I'd like to note that during this call, we will be making a number of forward looking statements. Please take a moment to review our slide on the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties, many of which are beyond Sarepta's control.
Actual results could materially differ from these forward looking statements and any such risks can materially and adversely affect the business, the results of operations and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, We encourage you to review the company's most recent 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. I'll now turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?
Thank you, Mary. Good afternoon, everyone, and thank you all for joining Sarepta Therapeutics' Q2 2021 investor conference call. With a large multi platform genetic medicine pipeline, which spans RNA, gene therapy and gene editing, And with 3 approved therapies, we have a significant number of important initiatives underway in 2021. And I and my team I'm very pleased to share with you this evening our progress in the achievement of our milestones. I am particularly proud of this team's focus on execution and the consistent achievement of our goals this year, which you will see reflected in our results.
Now, as pleased as I am with our quarterly performance, I am going to dispense with my usual order and start by updating you on the important and very positive developments for SRP-nine thousand and one this quarter. I am delighted to share with you the progress we've made with respect to SRP-nine thousand and one and the positive outcome of our recently completed meeting with FDA's Office of Tissue and Advanced Therapies. I'll be referring to that division going forward Now, as a reminder, earlier in the Q2, we announced the 12 week results of our first 11 patient cohort for Study 103 also known as Endeavor. To remind you, Study 103 is our trial evaluating the expression and safety of our commercially representative material. This is an extremely important study as it has confirmed the performance of our therapy Using the material process with which we intend to launch SRP-nine thousand and one, we were pleased to report that on every biomarker, Our commercially representative material performs as well as or better than the clinical supply material we used in our prior study.
Armed with the positive Study 103 results, we scheduled a meeting with OTAT to review our CMC and our plans For Study 301 that is our proposed pivotal trial for SRP-nine thousand and one. I would like to thank OTAC for what was a very productive informative meeting. And based on that meeting, we are on track to commence Study 301 as proposed to the division, both in the United States and then globally, and we believe we should be able to initiate Study 301 in September of this year. The initiation of Study 301 is an important moment, not merely But for families living with Duchenne, following receipt of the final minutes, I will provide detailed information regarding our study design And some of the reasons we are so confident that our program will be successful. Now, we've taken a deep dive into Part 1 of Study 102.
And we are not only very confident in the performance and transformative potential of SRP-nine thousand and one, But we are also very confident that 301 is well designed with a high probability of success of showing that performance. Again, Once we have the final minutes and are ready to initiate the trial, I will share with you the details of the trial and the data driven basis for our confidence in this program. Looking forward, Study 102 is proceeding and remains blinded. And we will have a readout of the second phase of that study, including 1 year and 2 year As you know, we have seen very robust results from our first two cohorts in our proof of concept I can now report that we have already solicited and received written feedback from both the FDA and EMA regarding our plans for SRP-nine thousand and three, both confirming the possibility of using protein expression as an endpoint for accelerated approval in the U. S.
And for conditional approval in Europe. From here, we need to gain alignment with the FDA and EMA on Precise clinical and regulatory approach, appropriate for LGMD2E and then appropriate for the rest of the LGMD pipeline. And based on the written feedback we have received, we are investing time now considering how we might move our entire LGMD sarcoglycan platform Forward together. With the successes that we've seen with our gene therapy approach to LGMD, We are expanding our portfolio. Doctor.
O'Neill will provide additional color on our license to another rh74 mediated gene Moving now to our RNA franchise, you will recall that earlier this year, we announced positive results from Part A of our MOMENTUM trial, Studying our next generation version of our Morpolino platform, the peptide conjugated PMO or PPMO SRP-five thousand and fifty one, which is designed to enhanced version of our PMO technology for Duchenne patients who have a mutation amenable to exon 51 skipping. Doctor. O'Neill will review the positive results of that study at 30 mgs per kg and will provide our plans to commence our pivotal phase of our trial for SRP-five thousand and fifty one later this year. The advancement of our PPMO technology solidifies our singular leadership in evidence driven RNA technology to treat rare diseases where steric blocking can provide benefit. Our PMO is predictable And a durable platform that has already produced 3 FDA approved therapies that can in turn improve the lives of nearly 30% of Duchenne patients In the United States and at least for now to a lesser extent outside the United States.
Our next generation PPMO, if confirmed in upcoming trials, We'll greatly extend the reach and impact of our RNA platform. As Doctor. O'Neill will share with you, our PBMO platform has the potential of being a leap forward in the treatment of Duchenne. With the PBMO platform, we have the technical ability to construct therapies We're well over 80% of Duchenne patients and with profoundly greater dystrophin production, the opportunity exists to And our reach far beyond the United States with approvals globally. And even as we develop our PPMOs for Duchenne, we are exploring additional genetic diseases Where steric blocking may provide benefit.
We will work with the neurology division at Cedar and that's the division with an FDA responsible For our RNA platform to gain alignment on Part B of SRT-five thousand and fifty one trial and once confirmed with We will commence our Part B pivotal trial before the end of this year as we also advance additional constructs for other mutations. Now, let me comment on our quarterly performance serving the Duchenne community with our 3 approved RNA therapies. In addition to continuing to adapt to this pandemic, the team is focused on serving our patients with EXONDYS and VYONDYS And launching Amondis, which as you know was approved back in February of this year. And their success is reflected in our quarterly numbers. I am pleased to report that in the 2nd quarter, we achieved net part revenue of approximately $142,000,000 That represents a nearly 27% growth over the same quarter last year.
Since our first approved therapy nearly 5 years We have enjoyed long term compounded annual growth of about 20%. And as a testament Fusions amid a troubling pandemic environment, the adherence rate for our therapies remains well over 90%, An extremely impressive and telling metric. Based on this success, we have today increased our full year net product revenue guidance. At the commencement of this year, our guidance was in the range of $537,000,000 to $547,000,000 Today, we are raising that guidance to between $565,000,000 $575,000,000 representing at the midpoint a growth of more than 25% over last year. Our Chief Commercial Officer, Dalen Murray, We'll provide more color on our quarterly performance in a moment.
Now, the progress we've made in 2021 demonstrates While not currently reflected in our stock price, I am proud that the Sarepta team has executed this year and achieved nearly every one of To summarize, let's consider the accomplishments so far in 2021, and I'll do this chronologically. First, additional Evidence driven confidence in the probability of success of SRP-nine thousand and one. Even though Part 1 of Study 102 did not achieve statistical significance on the analysis of the 4 to 5 year olds where the baselines were in line, we saw not nearly strong statistically significant benefit, but perhaps the best results So far in a trial for Duchenne. Moreover, an analysis of the complete data set from Study 1 And more than just that, the study has informed the design of our next trial, greatly enhancing its probability of success. Next, we received FDA approval for and launched our 3rd RNA therapy to treat Duchenne.
Of course, that is Amandis, Now serving a record percentage of patients. As Dowan will detail in a moment, our performance across all three of our RNA based therapies, EXONDYS, VIONYS And Imonidis, even in these challenging times, has been exceptional. We also reported Over the course of this year, several positive clinical data readouts that bolster not only our approach but the intrinsic value of both Our gene therapy and RNA platforms consider exceptional functional improvement and durability results For SRP-nine thousand and three, our LGMD2E gene therapy. We also reported impressive clinical results For SRP-five thousand and fifty one, the first of our candidates from our next generation PPMO platform, we also reported exceptional clinical results From the first cohort of Study 103 confirming the performance of our commercially representative material for SRP-nine thousand and one And frankly, the culmination of an enormous work and investment to advance our gene therapy manufacturing process over the last few years. And as you have heard today, we've completed a productive meeting with OTAT regarding our pivotal trial for SRP-nine thousand and one that is Of course, Study 301, and we are on track to initiate that trial in September of this year in the United States and then around the world as well.
Looking forward to the rest of the year, in addition to initiating Study 301 and continuing Study 102 for SRP-nine thousand and one, we will engage the FDA and ministries of health to align on our clinical and regulatory pathway for our LGMD portfolio. And separately, we will align with the FDA and other ministries of health around the world with the goal of advancing our PPMOS RP-five thousand and fifty one to a pivotal trial to start this year. I look forward to updating you as we progress and to continue to execute on our milestones over the remainder of this year. And with that, Let me turn the call over to Ian Estapan, who will provide an update on the financials. Ian?
Thanks, Doug. Good afternoon, everyone. This afternoon's financial results press release provided details for the Q2 of 2021 on a non GAAP basis as well as a GAAP basis. Please refer to the press release available on Sarepta's website for a full reconciliation of GAAP to non GAAP financial Total net product revenue for the Q2 of 2021 from our PMO exon skipping franchise was $141,800,000 compared to $111,300,000 for the same period of 2020. For the Q2 of 2021, individual net product sales were $112,500,000 for EXONDYS $51,000,000 $22,400,000 for VYONDYS 53 $6,900,000 for the newly launched AMONDYS 45.
The increase primarily reflects higher demand for our products in the launch of AMONDYS 45. As Doug said, due to strong performance, we have increased our 2021 revenue guidance range for our RNA franchise. In the quarter ended June 30, 2021, we recognized $22,300,000 of collaboration revenue compared to $26,000,000 recognized in the same period of 2020, which primarily relates to our collaboration arrangement with Roche. The reimbursable co development costs under the Roche agreement totaled $17,700,000 for the Q2 of 2021 compared to $8,900,000 for the same period of 2020. On a GAAP basis, We reported a net loss of $81,400,000 $150,800,000 or 1 $0.02 and $1.93 per basic and diluted share for the Q2 of 2021 2020, respectively.
We reported a non GAAP net loss of $121,200,000 or $1.52 per basic and diluted share in the Q2 of 2021 compared to a non GAAP net loss of $111,900,000 or 1 point We recorded approximately $19,500,000 in cost of sales compared to $13,300,000 in the same period of 2020. The increase in cost of sales is primarily due to increasing demand for the company's products. On a GAAP basis, we recorded $239,600,000 $188,500,000 in R and D expenses for the Q2 of 2021 2020, respectively, a year over year increase of $51,100,000 This increase is primarily due to an increase in milestone and manufacturing expenses. On a non GAAP basis, R and D expenses were $189,000,000 for the Q2 of 2021 compared to $160,400,000 for the same period of 2020, an increase of 28.6 Now turning to SG and A. On a GAAP basis, we recorded approximately $72,300,000 and $73,700,000 for expenses for the 2nd quarters of 2021 and 2020, respectively, a decrease of $1,400,000 The year over year decrease was driven primarily by a decrease in compensation, personnel and professional service expenses.
On a non GAAP basis, the SG and A expenses were $54,000,000 For the Q2 of 2021 compared to $55,100,000 for the same period of 2020, a decrease of $1,100,000 On a GAAP basis, we recorded $16,200,000 in other expenses net for the Q2 of 2021 compared to 12 point $4,000,000 in other expenses net for the same period of 2020. The increase primarily reflects an increase in Interest expense incurred on the company's term loan debt facility due to an increase in the outstanding balance, partially offset by a reduction of interest expense incurred on the company's convertible debt related to the adoption of ASU 2020-six. In February 20 We entered into an agreement to sell the rare pediatric disease priority review voucher or PRV we received from the FDA in connection with the approval of OMANDIS-forty five. In April of 2021, we completed our sale of the PRV And received proceeds of $102,000,000 with no commission costs, which were recorded as a gain from sale of the PRV Did not carry any did not have any carrying value at the time of the sale. There was no similar activity during the Q2 of 2020.
We had approximately $1,740,000,000 of cash, cash equivalents and investments as of June 30, 2021. As Doug just outlined and Gilmore will go over in more detail, we progressed several pipeline programs this year. For this reason, we anticipate running multiple pivotal study in 2022. We will continue to invest in our manufacturing scale up in anticipation of delivering these therapies to patients. And with that, I'll turn the call over to Dalen for an update on our commercial activities.
Dalen?
Thank you, Ian, and good afternoon, everyone. The team has yet again exceeded expectations across all three of our approved products in the Q2 of 2021. Due to the strong performance, as Doug mentioned, we have increased our guidance range by almost $30,000,000 with the new guidance being $565,000,000 to $575,000,000 up from the $537,000,000 to $547,000,000 As mentioned, total revenue reached approximately $142,000,000 in Q2, representing double digit growth over the previous quarter, and we approached Nearly 27% growth versus Q2 of 2020. The 2nd quarter represents Our strongest rate of revenue growth since the EXONDYS fifty one launch phase. It's our 19th consecutive quarter of revenue growth since launch in 2016.
To put that into perspective, that's 1 quarter shy of 5 years of consistent quarter over quarter revenue growth. Keep in mind that this consistent growth has been achieved without taking a single price increase at any point, making this accomplishment that much more distinct and impressive. Now, transitioning to the details of our performance in the second quarter. Our revenue in Q2 was driven by strong performance across all three of our approved PMO based exon skipping medicines. I'll review each In chronological order, beginning with EXONDYS 51, the team has continued to execute, driving revenue to over 112,000,000 Q2 2021, which represents roughly 8% growth versus Q2 2020.
We've worked hard throughout the COVID-nineteen pandemic to mitigate the risks to EXONDYS fifty one and have emerged in a strong position. Our impressive performance from Q1 to Q2 2021 for EXONDYS 51 was a result of the team driving a robust rate of During the insurance changes we typically see at the beginning of each year. As such, we don't expect See the rate of growth we saw in the Q2 with EXONDYS fifty one to continue at the same rate. It's important to be reminded that we now see The exon 51 amenable population as mature and well penetrated in the ambulatory setting. Having had an approved therapy on the market for nearly 5 years, As a result, any growth that we see for EXONDYS fifty one will be primarily driven by newly diagnosed or incident patients.
Moving now to VYONDYS 53. Although, we're still very much in the launch phase, I'm happy to report We are seeing minimal competitive impact on the demand from both patient and physician community. Revenue totaled over $22,000,000 in the 2nd quarter, representing nearly 30% growth versus the Q1 of 2021, Reinforcing our leading position, our expertise in Duchenne and the flawless execution of our team. The team has done a great job in the Q2 getting patients on therapy. And as we work through the start forms from the launch phase, we expect More modest growth in subsequent quarters due to a smaller base of start forms to work from.
Overall, from what we're seeing to date, The vast majority of exon 53 treated patients are on VYONDYS 53 and the competitive launch has had Limited impact on our overall launch trajectory to date. And finally, OMANDIS 45. Perhaps Our most exciting news coming out of the Q2 is our stronger than anticipated launch. While it's early days, we're seeing revenue from IMONDYS 45 cracking ahead of the EXONDYS fifty one trend, which is even more impressive given the relative size differences of the 2 patient populations. Adjusting for the relative population sizes, the rate of new patient start forms for MODIS 45 are in line with what we saw for the EXONDYS 51 launch.
However, based on our deep experience in Duchenne And constant improvements in terms of the execution of the team, the time to getting patients access to and on therapy has been faster for AMONDYS 45 than what we saw for EXONDYS 51. As a result, the team has delivered nearly $7,000,000 in revenue in our first Full quarter with ELONGIS 45. The successful launch of ELONGIS 45 is our 3rd since 2016 and represents the dedication of our team who work every day on behalf of patients. Our deep experience with Duchenne has enabled us to serve more patients, expedite access to drug and offer best in class support through SelectAssist. We are We're proud of the team and will continue to apply learnings toward our number one priority, which is serving the nearly 30% of Duchenne patients who may benefit from our And now, I'll turn the call over to Gilmore for an update on our research and development activities.
Gilmore?
Thank you, Dalen, and good afternoon, everyone. In the second quarter, a great deal of progress was made in advancing both our RNA and gene therapy programs. Before beginning with our RNA based PPMO program SRP-five thousand and fifty one, I want to echo Doug's sentiment That we are very pleased with our recent meeting with OTAT regarding the SRP-nine thousand and one program. We remain on track to initiate our Study 301 this September in the United States and globally. As you recall, in early May 2021, we announced Positive clinical data from the 30 mg per kg arm of the MOMENTUM study for SRP-five thousand and fifty one evaluating safety and change from baseline at week 12 for exon skipping and dystrophin expression in both ambulance and non ambulant patients.
3 of the patients In their late teens and one patient was 7 years old at the time of treatment. The results were impressive. The 30 mg per kg cohort showed a significant Dose dependent increase in exon skipping. SRP-five thousand and fifty one when dosed once per month at 30 mgs per kg achieved approximately 11% mean exon skipping at week 12. Compared to the PPMO 20 mg per kg dose, At the 30 mgkg dose, we observed a greater than fourfold dose dependent increase in exon skipping at only a 50% increase in dose.
Further, when compared to the current standard of care at tepeersen, We observed an 18 fold increase in exon skipping. Now in terms of expression, the 30 mg per kg dose SRP-five thousand and fifty one demonstrated more than 6.5% mean to stroke and protein expression as measured by Western blot, representing a greater than 100% increase in expression versus the 20 mg per kg cohort at only week 12. Here are some other notable aspects of the data. 1st, the results were not driven by a single All the patients respond well to therapy. 2nd, based on our predictive modeling, we should comfortably achieve greater than 10% dystrophin with once Per month dosing over time.
3rd, baseline dystrophin levels are not a predictor of post treatment expression. In fact, we observed that 2 patients with the lowest baseline had the highest level of post treatment expression. And 4th, We continue to believe that the hypomagnesemia observed in the study remains monitorable and manageable with magnesium supplementation And is not correlated with changes in renal function. Our next step is to meet with FDA regarding Part B of momentum And based on the outcome of that meeting, our intention is to dose Part B by the end of 2021. We are thrilled with the SRP-five thousand and fifty one And the potential that SRP-five thousand and fifty one hold to offer individuals with Duchenne a more convenient once per month treatment option with a manageable safety profile and superior dystrophin expression.
Now shifting to our gene therapy program, I will begin with our safety and biopsy results reported in mid May from the first 11 patients in Study 103 using our commercially representative material for SRP-nine thousand and one. These results are tremendously valuable because they confirm the Characteristics of the commercially represented material for SRP-nine thousand and one, which achieved robust transduction for a mean of 3.87 vector genome copies per nucleus. In addition, we reported robust expression of microdystrophin Correctly localized to the sarcolemma membrane. And we have measured this 3 different ways with a mean of 55 point And correctly localized to the membrane. Furthermore, we observed a consistent safety profile with our clinical manufacturing process With no clinical complement manifestation, it cannot be understated that the Study 103 results provide Confirmation of our manufacturing process and analytics positioning us to aptly serve the Duchenne Population.
I'd also like to remind you about some critical findings from Part One of our ongoing SRP-nine thousand and one-ten thousand and two study. I want to emphasize that because of the study's stratified randomization design and the statistic analysis plan, we can state with confidence That the pre specified subgroup analysis of 4 to 5 year old Duchenne boys' stratum demonstrated that SRP-nine thousand and one treated boys achieved NSAA That were both clinically meaningful and superior to placebo treated boys with statistical falciparum. This means That the SRP-nine thousand and one micro dystrophin construct is functional in humans and confers physiological and clinical benefit, thus substantially increasing the probability of success for this program. Now, moving to our Limb girdle muscular dystrophy portfolio. Our Six development stage programs have the potential to address approximately 70% of all limb girdle patients.
These programs are progressing well And we continue to hold a leading position in limb girdle muscular dystrophy, grounded in differentiated science and a deep understanding of the disease. Currently, Sarepta has several problems in development to treat various subtypes of limb girdle muscular dystrophy. And this morning, We announced the execution of a licensing agreement with Nationwide Children's Hospital for CALPANE3, a gene therapy candidate to treat limb girdle muscular dystrophy Type 2a or calpainopathy. Limb girdle muscular dystrophy type 2a is caused by mutations in the calpain 3 gene And it's the most common form of Limb girdle, accounting for 1 third of Limb girdle muscular dystrophy diagnosis. We are pleased to report The preclinical research and safety studies led by Doctor.
Sarif Sahenk at Nationwide Children's Hospital have provided early proof concept for Kalp injury in limb girdle type 2a and support further advancement. We will apply the learnings from our SRP-nine thousand and one and SRP-nine thousand and three development program to the Calpain 3 program and our 5 other limb girdle programs, all of which use Same, AVrh74 vector designed to robustly deliver treatment to skeletal muscle, making it an ideal candidate to treat muscular disease. Now turning to SRP-nine thousand and three, our lead limb girdle gene therapy candidate in development to treat limb girdle type 2e, which demonstrated positive data earlier this year at the 2021 Muscular Dystrophy Association's Annual Clinical and Scientific Conference. We presented the 1st expression data from biopsies of participants in cohort 1, the low dose cohort, taken 2 years after a single administration of SRP-nine thousand and three. The results showed sustained protein expression in muscle tissue.
We are thrilled with these results for the SRP-nine thousand and two program as they also provide read through to our 9,001 program And any program that utilizes rh74 and the MHCK7 promoter. Now, Turning to our functional results for SOP-nine thousand and twenty three. Assessments were taken 2 years following treatment in Cohort 1 and 1 year after treatment in cohort through the high dose cohort. We were pleased to observe that patients continue to demonstrate stability in the NorthStar assessment For different endophies or MSAT total score and improvement on time function tests. The results from both cohorts continue Support a differentiated safety profile of the rh74 vector compared to other AV serotypes.
In fact, Between our SRP-nine thousand and one and SRP-nine thousand and three programs, we have dosed nearly 80 patients and have maintained a consistent safety profile. We also believe that the high level expression observed with our constructs led to durable outcomes that are critically important for patients receiving a one time therapy. All these therapies are not a coincidence as SRP-nine thousand and one was rationally designed And then the learnings from this candidate have been applied and continue to be applied to SRP-nine thousand and three and our 5 other limb girdler candidates. The SRP-nine thirty three results represent a solid foundation, a virtual engine to build and advance a steady stream of additional sarcoglycan derived indications in Limb girdle muscular dystrophy. Now, many of you are likely aware that in early September, the Cellular Tissue and Gene Therapies Advisory Committee is meeting to discuss the toxicity risks of adeno associated virus or AAV vector based gene therapy.
With the results we have thus far from our SRP-nine thousand and one and SRP-nine thousand and three programs, we expect the discussion with Center Round's vector specific We look forward to the meeting and expect that the shared learnings will be helpful and continue to drive the field of gene therapy forward. Additionally, we look forward to sharing data from our gene therapy and RNA pipeline program at the 2021 Annual Congress of the World Muscle being held virtually from September 20th to 24th. Finally, and most importantly, I want to thank all the patients, The families, study sites and coordinators, my R and D colleagues and our partners who have done so much work under incredibly difficult circumstances caused by the pandemic to maintain our urgent mission to deliver new highly effective therapies to people who are diseases. I will now turn the call back over to Doug to open the question and answer session.
Doug? Thank you very much, Doctor. O'Neill, and thank you for the rest of my colleagues. May, let's open up the lines for the Q and A now.
As a reminder, callers will be limited to 1 question only. Your first question comes from the line of Gena Wang of Barclays. Your line is open.
Thank you for taking my questions. I have one question regarding study 301. Doug, I know you will share detail about The trial design, just wondering if you can share the high level of a trial design now? And also regarding Study 102 crossover data in Q1 next year. Just wondering, have you defined pre specified natural history control?
Yes. Thank you for that question. Gina, first of all, thank you for the first question, because I'm going to get this question a lot this evening and this gives me an opportunity to frustrate everybody just once. So, I am not going to provide details on Study 301, other than of course to let you know that it is a Placebo controlled double blinded trial that will be our pivotal trial. There are a lot of nuance behind that, The powering of it, the end, the age groups, etcetera.
And we have really put a lot of thought into Study 301, Informed enormously by Study 102. And I'm going to be I'm very excited to share that with you. And I'm very excited that we were able very recently To have met with the division and on that basis, gained confidence that we're going to initiate that trial in September of this year. But I'm going to frustrate you and not provide a ton of detail other than to know that it is obviously a robust, very well powered, double blind, Placebo controlled trial. Our Study 102, of course, before the team is working on natural history sets and all of the statistics associated with that, It is a blinded trial.
As you know, we'll have to fully lock all of that down and lock The natural history sets down before we unblind. I think that chem has still has additional statistical work to do before the stat plan is Fully locked. And thank you for both of those questions, Gina.
Your next question comes from the line of Tazeen Ahmad of BOA. Your line is open. Hi, guys. Good afternoon. Thanks for taking my questions and thanks for all the positive updates.
So my one question, Doug, is about timing of your study. You're expecting to start in September. On the Pfizer call that happened recently, they didn't seem to make specific mention of what is happening with their DMD study, At least with U. S. Enrollment, so I'm curious if you end up on a timeline where you're starting the study every single U.
S. Please just around the same time, what's your view without overlapping clinical trial sites and whether or not it might impact the ability to enroll? Thanks.
Yes. Thank you for that. A couple of thoughts. One, I want to be clear. I'm not I'm going to avoid directly Comparing to other programs, as you can well imagine, I have probably a well earned reputation for leaning towards the competitive.
So I'm going to Fight my natural instinctive, but I will say the following. Let me say the following. First, that there is no one else that has the amount of clinical Data to inform their program and the confidence of their therapy like Sarepta does. As Doctor. O'Neill, I believe mentioned in his Opening remarks.
We have by now dosed at these levels SRP-nine thousand and one And ambulatory and non ambulatory children and nearly 80 children, if not more than 80 by now. So we have an enormous amount of information that informs the confidence of our therapy and informs the next program and gives us a lot of confidence that we're going to do quite well. And we've done Study 101, we've done Study 102 To part 1, we will have Study 102 Part 2 that we'll read out very early next year. We have Study 103 in the first cohort of that of course looks Brilliant in the performance of that therapy from an expression and safety perspective, and we'll start 301 and initiate that trial The fall goes well in September and we feel very confident about that. So we're in very good shape to drive SRP-nine thousand and one forward.
And of course, the reason that we want to do that with enormous amounts of urgency is, hopefully everybody that's near this rare disease knows, There are thousands and thousands and thousands of children, literally hundreds of thousands of children around the world who are having their muscles stolen from them Today, literally unrelenting and unfortunately invariably fatal. So, we need to move fast and we will do that. On the specific question you asked, the second part of your question that you asked, again, there is an enormous Need for therapies like this, for this gene therapy, for PPMO and others, and therefore, Understandably an enormous amount of interest and desire that we move fast, both from families who are living with And unfortunately and invariably dying from Duchenne muscular dystrophy and from physicians and investigators. So, I stand by the proposition that I've had for some time that this program, once we get going, Should enroll with significant rapidity.
Your next question comes from the line of Brian Abrahams of RBC Capital Markets. Your line is open.
Hey, guys. Congrats on all the progress and thanks for taking my question. I'm just wondering with respect to Study 301, are there any additional gating factors to getting that study up and running both in the U. S. And internationally?
Any additional back and forth required to the agency? And can you confirm that, I guess, at this point you're aligned and all the necessary potency assays are expected to be aligned shortly? Thanks.
A couple of thoughts. One, just to initiate a study and roll a study out around the world, there are lots of steps. So I want to say, this is The team has been frankly, in my view, fantastic in their execution, but we have a lot to do to get this study fully enrolled around the world. So there's I don't want to create the impression that there's not a lot of additional work to do there is. It seems very, very competent and confident in getting all of that done.
I'd say the biggest the most significant issue for us to ensure that we could move rapidly and initiate Try out quickly, particularly in the United States was to have that meeting with OTAT. And of course, we had that meeting. I will say, although I'm not going to give you the exact date, it was very recent. And as I said, a very positive meeting, Very informative meeting, very well prepared meeting on both sides, by the way. And as a result of that, we have an enormous amount of confidence that we're going to be able to initiate this trial Very soon and our goal is to have that done in September and we feel confident about that.
As it relates to the potency assay, we've done a lot of work on the potency assay. As you may recall, back last year in September, we actually had been stalled For a short period of time, it turns out, based on potency assay, that was September of 2020, that actually ended up being for us a blessing. It gave us an opportunity to have live dialogue with the division and to really understand at a fairly Granular level, what the division was looking for, not only then but in the future on potency assays. And so, we've done a bunch of work since then. We've shared our perspective on the potentiassays and our approach to potentiassay with the division.
They have agreed with our approach And endorsed our approach and we've gathered the data and we as I said before, there's a lot still left to do as one could imagine to get a trial up and running around But we feel very confident that we'll be initiating that trial this year. And frankly, we feel confident we'll be initiating in September.
Your next question comes from the line of Alethia Young of Cantor. Your line is open.
Hey, guys. Thanks for taking my question and congrats on the progress. I'll ask one, but I will put a contingency in because you may not answer it. I guess, I wanted to get your perspective on like PPMO potential endpoints. Is there a potential for similar endpoints to PMO?
Or is it more functional? And then if you don't answer that, can you just talk about maybe some other indications where steric blocking is associated with BPMO and
Yes. I will start with so on the PBMO, there'll be Essentially, there will be a functional endpoint, of course, as we will eventually need to confirm the benefits Functionally to continue to have the therapy approved around the world. But our goal in the first instance with the PPMO Is to seek an accelerated approval in the United States and potentially even a conditional approval in Europe on the basis of the robust Expression that we're seeing, we have a well understood pathway in the United States with the accelerated approval pathway. So, it will be It will be essentially 2 broad things. There will be functional endpoints for the PPMO and then there will be I don't think that we've landed on or We don't yet disclose probably what particular endpoint that we'll use for it.
It'll obviously be there'll be some complication because with functional endpoints, we'll have to think about The functional endpoint for the ambulatory population and then functional endpoint potentially for the non ambulatory population. But we'll also have expression and that will be dystrophin production and We have a well understood pathway in the United States, and we'll have a dialogue with EMA on that basis as well. And then on additional indications, I will turn this over either to Doctor. O'Neill or Doctor. Medina Klapac for that to maybe chat about some of the other possibilities.
Your next question comes
Before we go on, I think there's Maybe you had another question I want to answer. I just created confusion because I'm supposed to be the master of ceremony and called on 2 people. So I will start with Doctor. O'Neill Either answer it or turn it over to Doctor. Omidyho Klapac.
Yes. So we're actually very interested in leveraging the acute Beyond muscle and muscular dystrophy and are looking at a number of tissues are targets of interest, Both in with muscle indication as well as potentially renal indication. So that's the kind of work that we're still performing in our discovery phase, and we will be leveraging our learnings From the ongoing 5,051 and then applying those and accelerating those forward once we have absolute clarity On our 5,050 1 progress and the PPMOs across the exon skipping amenable population for Duquesne, Louise, I don't know if you want to add anything to that?
No, I think you covered it well. Thanks.
We do have preclinical work ongoing exploring a number of different Disease areas where steric blocking might be interesting, but that isn't Duchenne. And then of course, I should say, just to remind everybody, We are working on PPMO-five thousand and fifty one, but we've got a number of other constructs for additional mutations. And at least theoretically, We can build constructs with a high probability of success that could treat patients, From a technical perspective, it could be well over 80% of patients that we could build therapies. There is a small percentage of very rare exons, very rare mutations where This stair blocking the technology and exon skipping isn't available, but the good news is a very small percentage of Duchenne patients.
Your next question comes from the line of Anupam Rama of JPMorgan. Your line is open.
Hi, guys. Thanks so much for taking the question and congrats on the progress. Just on Study 301 And the initiation here in September, how do you think about getting sort of global sites up and running, particularly with the pandemic Very variable in different regions and how you think about the enrollment curve here? Thanks so much.
I think, I'll save the broad strokes. Again, I think enrollment, You raise an interesting point about, we live in uncertain times. Every time we think we have clarity on this pandemic, new information comes out that It forces us to continue to be humble in our prognostication about the future. I would still broadly say that I am very confident, the team is very confident that this is going to be Robustly very robustly enrolled therapy. And even in the height of the pandemic, We have experienced that sites ex U.
S. And U. S, but ex U. S. Is the kind of thing I think a lot of us are worried about, Have been able to stay up and running and continue to execute without much of a problem.
In the absolute raging Most difficult part of the pandemic, we were running a very significant, as you know, in other trials, we were running a very significant global trial, which is the ESSENCE trial to confirm the benefits of Viandis and Immodis. And even in the most difficult times with very few exceptions, we were able to continue to execute and patients were able to get in and Got their infusions. And this is going to be that's even more complicated because of course those infusions are weekly and then got their functional results. There were a few missed visits, but not very many. So, I think we'll be able to navigate through things.
Of course, I will say again what I've said to the team about 1,000,000,000 times. We have to remain humble in our prognostication about the pandemic. But, I don't think that's going to be the limiter on things. I think the most significant limiter is just our ability to get up, execute, get Sites up and running, get them qualified and going, and then I think recruitment will go very, very well.
Your next question comes from the line of Salveen Richter of Goldman Sachs. Your line is open.
Thanks for taking my questions. In your meeting with OTAD, was there any discussion about the potential for 9,000 1 approval on in the 4 to 5 year olds with DMD just based on data to date? And then secondly, can you help us understand how these 2 limb girdle muscular dystrophy type 2a programs will coexist within your portfolio?
Yes. So, I'll answer the first one and I'll probably leave it to Doctor. Rodino Klapac to touch on the calpainopathy in Girdle. So, let's be since we're very straightforward about this, we were very clear about our goals. When we ended the meeting with OTAT, we asked Very specific questions.
And in broad strokes, the specific question was, here's our CMC, review our CMC, here's our protocol For Study 301, do you object to us commencing Study 301? It's that discussion. We had absolutely No discussions. We didn't broach the issue of something else like an accelerated approval the 4 to 5 year olds or the 4 to 7 year olds, based on the data we've seen already or coming up the data that we might early next year in Part 2 of Study 102. Just to remind everybody, we're going to get some really exciting data well, some interesting data at a minimum and hopefully exciting data In the Q1 of next year, because we'll have 2 cohorts, 1 cohort with 1 year functional results in part 2 and another with 2 years functional results.
That is not an issue that we've grossed with the agency. There were no discussions around that. This was really very, very much Around Study 301 and the convention of Study 301. And I'd say, I'm very excited that we are we had a very, very productive, very Positive meeting with the agency. And on that basis, we're going to initiate that trial very soon.
And we believe it will be in September. Now, on Lynn Girdle, I'll turn it over to Doctor. Rodino Klapac to touch on calpainopathy.
Thank you for that question. I think that there was Some confusion. We only have 1 LGMD2A program. We did have an option to Doctor. Program for quite some time, so we've been talking about it in the context of our portfolio and then we recently executed the license on that program.
So, it's just 1 LGMD2A program, which now rounds out our portfolio, and we're really excited about it. Doctor. Tang has been working Quite tirelessly to complete the preclinical data, which shows efficacy both biologically and functionally And a calpain knockout mice
at
both in both young mice and old mice and really gives us a lot of confidence moving forward in our development program for this program? Thanks for the question.
We're particularly we're very excited about the fact that this is another 74 mediated gene therapy because as you can imagine with the amount of data that we've developed over the last few years, We are only more confident in the differentiated aspects of rh74 as the vector delivery mechanism for gene therapy.
Your next question comes from the line of Brian Skorney of Baird. Your line is open.
Congrats on getting clearance to go into Phase 3.
I guess, I don't know if the question
is most appropriate for Doctor. Dana Claypak or Doctor. O'Neill.
But I know you guys
have been exploring certain AAV redosing strategies. I think you have partnership And we've also discussed some other potential strategies in terms of tolerizing patients for re dosing. Any thoughts on the progress there? I know based on the LGMD data, it doesn't look like there's really waning of protein expression here. But sort of in the long term, Where are you in terms of looking at the potential for redosing AAVs?
Let me say a couple Preface remarks and then I'll turn this over to Doctor. Rodino Klapac, if she wants to make any broad statements. I suspect we're not in a stage where we're going to probably provide Any detail other than this is important. There's a lot of reasons why this is important. It's important for redosing, it's important for knocking down neutralizing antibodies To bring into frame the number of children that can benefit from our therapies, it's important for the ability.
If there was ever A topping up was needed. It would really be a fascinating opportunity. And we have a number of different programs That we've been advancing and we're pretty excited about it from at least from a preclinical perspective. I doubt we're going to provide a
I'll just reiterate that We've been very thoughtful about having a comprehensive strategy to be able to treat patients with pre existing antibodies And if needed, for the ability to redose and our we have both internal programs and then partnerships with Hans and Selekta that Really, will address the aspects of that. So, knocking down antibodies that have them and that's essentially preventing antibodies in patients that are treated for the first time. So, it's something that we're working very aggressively on.
I think this is important for the entire field of gene therapy eventually. Knocking down pre existing neutralizing It's extraordinarily important. Now, we are fortunate. We're in a fortunate position. But I think compared to many others, Our screen out rate for neutralizing antibodies is relatively low.
It's 15% to 17%, maybe percent But that's still 15% to 17% of kids that until we find a solution for them would be screened out. So, It gives us a real opportunity to bring those kids back in frame. And if all works well, give them a much better life. So that's why this is extraordinarily important, including redosing as well.
Your next question comes from the line of Gil Blom of Needham and Company. Your line is open.
Hello, everyone, and congratulations Great quarter. Just a quick one from us. So, the FDA is going to have An advisory meeting on the safety of AAVs. Is there any potential for new guidelines coming out of this meeting that might Require amendments to protocols. Thank you.
Well, I will let Doctor. O'Neill touch on that if he has additional comments. The one thing I would say is This is not an issue that we just had a discussion with the division. We just had a meeting on The all parameters of CMC and protocol and the like regarding 9,001 in our next study, and certainly none of these issues were And we're even part of that discussion. And frankly, we don't have any data preclinical or clinical that would Give us any concern around at least the issues as we understand them that are being raised in this upcoming advisory committee meeting.
We're very interested in it. We'd love to provide whatever information we have, but we're not particularly concerned that this would have an impact on therapies that we're working on right now. Doctor. O'Neill, do you have any other comments or contract comments?
No. I think you said it nicely. I would Yes, that is our belief and I think the data that we have and others have support the view that different serotypes in the AAV Group of capsids are different and behave differently and are differentiated. And I think we have a robust data set That show our differentiation and how we anticipate designing our protocols and how we design them is based On the empiric data sets that we have generated and as Doug said, we have dosed around 80 or in excess of 80 patients by now. So Those are the key things that we will use to drive our study designs.
And I will also say that we are looking forward to learning more from this advisory group. It is also probably worth The FDA guidance are actually relatively recently published. And I think you can speak in broad swades, but as I said, Different serotypes, different data sets. And so we believe and continue to believe that we will be designing our protocols around the empiric data that we are generating with from our clinical experience with our rh74 capsid. Thanks, Doug.
Your next question comes from the line of Ritu Baral of Cowen. Your line is open. Hi, guys. Thanks for taking the question. Doug, did I hear you right that your conversations with FDA around the LGMD sarcoglycanopathies.
Will Sacaglycan levels be sufficient to drive accelerated approval? And does that apply do you think that applies to like All of the second glycopathies, could it apply to A and calpain as well?
Yes. So, yes, I want to say dialogue, because this is a written response. So, we haven't had direct Live dialogue with the division yet. But the written dialogue that we've had, the written feedback that we've had from The division is that it is possible to use protein expression levels For QEE, presumably it's beta sarcoglycan levels as the basis for an approval. And that's obviously consistent both with It's because of the number of guidances that exist at the FDA, Vocibar and the neuro side of things.
So, we're very excited about That guidance and we're excited about the written fee that we've got. And there really is no reason on the face of it why that wouldn't apply to the SARCs, All of the stocks. They're very similar in a number of regards. And I think they're and similar in a number of regards and in some ways less Complicated than some other gene therapies. One of the issues with AAV mediated gene therapy is that of course the packaging Ability of AAV is limited and a lot of times the gene is larger than the packaging and you have to do interesting things like we have with micro dystrophin So a lot of great work.
The similar thing with respect to each of these sarco glycans Is that the gene is actually comfortably able to fit inside the AAV. So, we are making The a gene that codes with the native protein in all of the sarcoglycans, that's right now that's alpha, beta And Gamma, we're making the actual native protein unaltered that is the sole cause of the disease that And so I think it's not unreasonable for the agency to have suggested to us that We can use protein as a surrogate endpoint reasonably likely to lead to clinical benefit for purposes of That is still work we have to do. We have more conversations to have. We have to have a broader conversation both with MS by the way, I should say. And we've had similar feedback from EMA.
So, there's opportunity for accelerated approval in U. S, opportunity for conditional approval In Europe, as an example. And now from there, we've got to get more concrete about the plans and think about what this means To me, what the clinical program should look like, what the regulatory pathway will look like for that, both for The approval and then also obviously for the post approval confirmatory data that we're going to need, and we need to do that not only for 2E but for Gamma and for alpha as well. So that's one of the things we're working on right now.
Your next question comes from the line of Difei Yang of Mizuho Securities. Your line is open.
Hi, good afternoon and thanks for taking our questions. So just a clarifying question. At the ODAP meeting, did the FDA provide any guidance with regards to How to construct the natural history cohort for Study 102?
No, that wouldn't be an issue that The meeting with the division OTAT was all about the commencement of our pivotal trial study 301. And that of course isn't going to have a natural history cohort, it's a placebo controlled trial. So that wasn't an issue that was we didn't broach the question and that Didn't come up in
the meeting. Okay. And then do you Rob, just to follow-up on that. Do you think Eventually, you'll need to have FDA's buying on the natural history, the comparator's
Well, no, I think that's on us to make sure that it's done with that's done and done robustly enough That is meaningful and insightful. But generally speaking, I think we are the masters of our stat plan For purpose of 102, we just have to make sure that it's robust and it meets the statistical muster. And that's of course what we're doing. I have to say, we have a great team on that. We have a strong staff team.
And obviously, I think a very expert development team that's Considering all of those issues.
Your next question comes from the line of Tim Lugo of William Blair. Your line is open.
Hi, guys. This is John on for Tim. Congrats on the quarter and thanks for the question. I was just wondering if you could provide any updates on your views on longer term competition or maybe even opportunities from CRISPR based therapies in neuropathy diseases. Thanks.
Yes.
So, we're very excited about CRISPR technology. Let me be very Clear about that. We're making significant investments in CRISPRCas9. Hopefully, everyone knows we have Essentially, three platforms. We've got our RNA therapy, we've got gene therapy.
I think by now we are, If not the leaders in portfolio of gene therapy, we are certainly one of the top couple Leaders in Gene Therapy. And then, we've got this gene editing innovation center in Durham, North Carolina, where we're Looking at CRISPRCas9 and the ability to directly edit the genome as another long term opportunity to bring a better life to people With genetic disease, including neuromuscular genetic disease. And I'm excited about that. I'm very excited about, for instance, The fact that we have as our leader of our gene editing innovation center, Doctor. Charlie Gersbach, who is Brilliant.
And I think one of the significant world leaders in CRISPRCas9 generally and neuromuscular specifically. I will You'll note if you did a search of Google search of the articles, you'll see that I say this all the time, it probably would embarrass Doctor. Gerstach. But, I know that Doctor. Doudna, who I think we all know, won the Nobel Prize for CRISPRCas9 was asked Not too long ago, I think earlier this year, who she was excited about in the CRISPRCas9 space for the future.
And our own Doctor. Charlie Kirschbach was one of the names she gave and I think it was the only name she gave in relation to biotech. So we are very focused and very excited about it. Now, CRISPRCas9, certainly for full body infusion neuromuscular diseases is challenging today. So, from our perspective, this is a research project right now.
We are not yet ready to translate the Fascinating work on CRISPRCas9 into a clinical program right now in patients, because there are a lot of things that have to be unlocked Before you consider full body infusion, this is not trying to do CRISPRCas9 in let's say ocular Well, in the liver where it's sort of where these AAV mediated therapies significantly go, this is asking a lot more Of editing. So we've got a lot more work to do. So I don't I think CRISPRCas9 is excited. I think it could be very exciting for neuromuscular diseases as well, potentially even including Duchenne muscular dystrophy. But I don't see this as a near term competition to, for instance, gene therapy, which is right in front of us today.
I think this is A research project that we've got some work to do before we can translate this into clinical programs. But that is not to say we're not excited about it. We definitely are. We think there is going to be an enormous the time is now for RNA and gene therapy. And we think gene editing is something that could be very
Your next Question comes from the line of Matthew Harrison of Morgan Stanley. Your line is open.
Hi. This is Max Skor on for Matthew Harrison. Thank you for taking our question. So how should we think about the crossover arm in terms of the findings from Study 102? That is, would you expect it to replicate some of the age differences and other
So, no, the good news is that the one so There were 2 flaws in Part 1 of Study 102. 1 of the 2, of course, is the titering. As a result of the kind of PCR titling that was done in the Nationwide Children's Hospital material, Which is a super coiled PCR. We some of the kids were had less than the target dose, a significant number, 60% of the kids had less than the target dose. That's correct, let's just be clear.
That's correct. And for the crossover patients, Fully corrected. We were using our current titering method. We do not have that issue. The second and probably really significant The problem with part 1 was this enormous baseline imbalance, because even with those titering issues, When we had the baselines right and we did in the 4 to 5 year olds, you saw a clinically meaningful and very strong statistically significant improvement In the kids that were on treatment, it's 16 kids.
So clearly getting the baseline life is important. And the problem was in the 6 to 7 year old, there is enormous delta between active and the treated where the active kids were all very severe and The placebo kits were far more mild. I mean, significant, like the P value on that was 0.0 0.4. So, 4 times out of 1,000, you would have even had this problem. The good news of The crossover is that there's no way to look at a placebo arm anymore, of course.
All the kids will be on therapy. Some will be on therapy for 2 years, Some will be on therapy for 1 year. So the good question that was asked previously, we need to make sure we build a strong natural history before we ever unblind, Build a natural history model that is appropriate and matches and then we'll look at this therapy versus natural history. So that's essentially self correcting, that second issue, is self correcting on the Part 2. And then, on all the crossovers, the titering issue is corrected with our more precise titering that was used for all of the Doses and the crossover.
Your next question comes from the line of Joseph Schwartz of SVP Leerink, your line is open.
Hi, thanks very much. I was wondering beyond those two flaws that you were just Talking about Doug, how much have you been able to learn from your analysis of Study 102 that you can use to implement in Study 301 in order to improve the Probability of success. Do you have any examples that you can give us to help us appreciate how the POS might Rising going forward?
Well, I'm going to apologies, I'm going to beg off the detail and we'll talk about that A little bit later when I have the minutes and we're closer to initiating the trial because we will come back and talk about the trial and how we become even more Confident in the program and POS. But I'll give you a summary answer, Joseph, and I know this is frustrating, but I'll give you a summary at least. And that is There is an enormous amount of insight that we got from Part 1 of 102. I mean, we did a step sig, Some of us, myself, certainly included, we watched for a while and then immediately realized that we had an enormous amount of opportunity In front of us. And I'd say 2 things that came out of that part 1.
In summary fashion, and we'll talk about some of this in more detail when we get the minutes and we talk about The 301 trial. One is that we're just very confident as a people In our program and the probability of success of the program and the transformative potential of SRP-nine thousand and one, we feel Very confident. For instance, if we didn't have the baseline issues that we had with those kids, Unfortunately, we would have had a very different outcome on Part 1 of Study 102. And the second thing I would say is that it has informed 301 in ways that should significantly increase the probability of success.
Your next question comes from the line of Colleen Bristow of UBS. Is open.
Hi, this is Qing on for calling. Thanks for taking our question. Congrats on the quarter. So we have a follow-up question related to the potential accelerated filing for 901. We understand it was not discussed over the meeting you had with Odad.
Just want to get an idea of where do you stand now on the possibility of filing Micro dystrophin expression like biomarker, especially now with aducanumab approval where FDA did Take a much more flexible approach for this neurology diseases with high on malignancies. And any KAUSTRITA filing with preliminary expression data from 301 and of course with all the data you have with We also have a follow-up question, a separate question on the competitive landscape where We did notice Pfizer announced plans to start a Phase III trial in non ambulatory patients With no age restrictions in Phase II for early symptomatic patients aged 2 to 3 years old, any plans from your side to initiate studies in this population in near term? Thank you.
Yes. I'll answer the second question first. Certainly, One of the things that's happened historically with for good understandable reasons, but not for good results is that The non ambulatory patient population has been underserved in the clinical trial setting. We certainly will be looking to do trials in the non ambulatory population. And I should tell you right now, in Study 103, we've already dosed a lot of kids in 103.
We are dosing non ambulatory patients and larger kids as well In Study 103, so that is a big part of our plans. On the accelerated approval pathway, I'm going to say 2 things I'm going to say 3 things. The first, of course, is We understand that kids are getting damaged every day by this disease and that we need to think about How we can urgently move our therapies forward as fast as possible. And so, I wouldn't no one should assume that we're not willing to be thoughtful and creative about things. And the second thing I would say in broad strokes unrelated to 9,001 is that accelerated approval as a pathway has been An enormously valuable and innovative approach to bringing better lives to patients over the course of many years because accelerated approval has existed long before It was in the 21st Century Cures Act.
It has brought a better life to countless patients. And We could literally just reel off the cancer patients and AIDS patients and others whose lives have been Better or saved as a result of this wonderful approach when it's appropriate. But I will say this, finally, when I get to 9,001, I want to say 2 things about it. The first is that, as we're very clear, we've had no conversations with the division about it and there's nothing that would have come out of our meeting with OTAC that would have given us Any reason to be more or less confident in that as a concept? But the second thing I would say is that Study 301 is our pivotal trial.
And we have built it as our pivotal trial and it's robust and it's placebo controlled and it's from our perspective very well powered. And if I was an investor, I would presume that 301 is the pathway to success From a clinical perspective and from an approval perspective, both in the United States and around the world, and I wouldn't want to overpromise on other things, because that's our focus right now. Our focus right now is getting 301 initiated, up and running, fast enrolled and confirming the results that we've And what we presume we're going to see in early next year. And so, I just want to make sure we're not creating the wrong impression. I think 301 is our pivotal trial.
That's what I think people ought to focus on as the pathway for approval right now for SRP-nine thousand and one.
And maybe just maybe just one quick point of clarification. I don't believe Pfizer started a pivotal study in the non ambulatory patient population. The last update I think they gave was that They had an SAE in that population and they were doing a protocol change and adding sirolimus to their protocol before dosing more patients. So I don't believe they're in a pivotal study or not population?
No, I think they're not even I think their Phase 1 study that was going to explore non ambulatory patients. I apologize, I didn't realize you had suggested they had started. I think that they're actually not Moving forward right now, because they're going to do a protocol amendment to add another prophylaxis. I think they already have SOLIRIS, if I'm not mistaken.
Your next question comes from the line of Daniel Bril of Raymond James. Your line is open.
Hi. This is Alex on for Danielle. Thanks for taking the question. I just want to expand on your points on 103. When can we expect to see the expanded data set?
And if you can comment, how many patients have you dosed in those Non ambulatory and the older cohorts? Thanks.
So, we will find an appropriate Medical meeting down the road to provide additional information and insight in Study 103. So, we haven't selected A medical meeting yet that will be very interested to show that information. And I don't have the details, the number, Complete number, but we've dosed the number of non ambulatory patients and a number of larger kids with
Your next question comes from the line of Yun Zhong of BTIG. Your line is open.
Yes. Thanks very much for taking the question. And On Limb girdle 2E, it's very encouraging to see to hear the surrogate endpoint discussion, but You showed very positive functional data even after 1 year of treatment then. My question is why don't you go for full approval with the functional data? Does the pathway relying on surrogate endpoint, would that allow you to maybe avoid running a placebo controlled study?
Or Would that allow you to talk to the FDA on potential approval maybe even before 1 year after treatment?
Well, we're going to have to at a let's go at a bare minimum. We one has to dose Sheldon, using the commercially representative material, so let's start there. Understand that the data that we have right now, say the first two cohorts, I agree with you completely. It's brilliant data. It's brilliant on expression.
It's great on safety and it's great on functional signals as well. But that's in the That is in the clinical supply material. We've got to get all of the work done process development and clinical development and dose kids on the commercially representative material as a predicate It's A approval, that's as a first part. And then once we do that, why do why would we be interested in Looking at expression as opposed to just looking at function, the short answer is, this is a very rare disease and it is More heterogeneous than Duchenne muscular dystrophy. And so, it would just be We would make people wait far too long in our view if we had to wait For a statistically significant functional readout on 2 eKid.
This is to be direct, This is a perfect place for the use of a surrogate endpoint and accelerated approval. You've got a well characterized disease, at least the mechanism of action of the disease is well characterized, which is it is a single gene mutation, a Lack of a structural protein and that lack of structural protein is causing the demise and eventual fatalities for these Patients, these kids and adults. And we are able to deliver that exact protein, The literal native protein in robust amounts to the muscles. So, this is the kind of place where accelerated approval Was intended where you can bring this therapy because it's going to have such a high probability of being clinically meaningful to patients, you can bring it to patients And then, have an ongoing study that confirms those clinical results over time, so that you don't have to wait To bring the therapy to patients. And so, that's going to be our approach, and we've got more work to do with the FDA and EMA on that.
But I am happy to say that at least at a philosophical policy level, the division agrees that that is a potential, both that EMA agrees and FDA, hotel agrees as well.
There are no question at this time. I will now turn the call over to Doug Ingram for closing remarks.
Thank you very much. I will just extemporaneously say a few things, but not many as I know people it's getting late for everyone. Thank you very much for joining us this evening. I am very proud of the work this team has done, both to serve the patients That we have with the therapies available to us today, and I think the team has just done a brilliant job of that. 1 of the people haven't asked questions about our revenue, but That revenue is impressive because it reflects commercial expertise and a field based expertise That I got to tell you, I'm extraordinarily proud of and it's going to benefit us enormously when we launch some of these gene therapies.
And as we don't take price increases as a company, we've priced all of our therapies at parity, It is a reflection of our ability to serve patients and bring a better life to patients. So, I'm really thrilled with that. I'm obviously thrilled with the team For the great work we've done in advancing our portfolio and pipeline over the course of this year, We've made a lot of great progress and we have multi platforms to do that on. We've got our RNA. We've got our PPMO next generation version of the PMO.
We've got our gene therapy And of course, the list would go on with the sarcoglycans and the remainder of the LGMD. And then, we've got a bunch of therapies behind that that we don't have I'm even to speak about today. So, I'm excited about that. I'm looking forward to updating people across the course of this year. And I want to echo the words of Doctor.
Gilmore. I want to also thank the patients and their families, particularly those who have been willing to participate in the clinical trials That have created so much insight and I think in the end will provide so much hope to patients living with Duchenne muscular dystrophy limb girdle and the other diseases that we're fighting to try to reduce the impact of hopefully save lives of people. So, thank you for that. We look forward to additional updates over the course of this year.
This concludes today's conference call.