Sarepta Therapeutics, Inc. (SRPT) Earnings Call Transcript & Summary

Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics 2020 Virtual Annual Meeting of Stockholders. As a reminder, today's program is being recorded. And now I'd like to introduce your host for today's program, Dr. Kathy Behrens, Chairwoman of Sarepta Therapeutics. Please go ahead.

Good morning. Welcome to the 2020 Annual Meeting of Stockholders for Sarepta Therapeutics. It's now 9:00 a.m., and the meeting will please come to order. My name is Dr. M. Kathleen Behrens, and as Chairwoman of the Board of Directors of Sarepta, I will be presiding over this meeting. Doug Ingram, President and Chief Executive Officer of Sarepta, will also preside over portions of the meeting. Ty Howton, Executive Vice President, General Counsel and Corporate Secretary, will record the proceedings. As all of you are aware, we are living in an extraordinary time. COVID-19 risk mitigation measures are keeping most of us in our homes and away from our typical daily routines. I hope that all of you are staying safe and healthy. Hosting our first-ever virtual shareholder meeting is just one example of the many adjustments we have made to help combat the virus while continuing to fulfill our responsibilities. The order of business at today's meeting will follow the agenda, which is attached in the Document section of the webcast. [Operator Instructions] We will conduct the business portion of our meeting first and answer questions at the end of the meeting. Though we may not be able to answer every question, we will do our best to provide a response to as many as possible. Joining me today are the following current members of the Sarepta Board of Directors: Doug Ingram, who will be speaking later; as well as Richard Barry, Dr. Mary Ann Gray; Dr. Claude Nicaise; Dr. Hans Wigzell; and Dr. John Martin. I would like now to call your attention to the individuals who have agreed to stand for election as Class I directors to the Board of Directors at this meeting. Doug Ingram, Dr. Hans Wigzell; and Dr. Mary Ann Gray are standing for election to the Board of Directors. Also present today are the company's executive officers. Doug Ingram, President and Chief Executive Officer; Sandy Mahatme, Executive Vice President, Chief Financial Officer and Chief Business Officer; Diane Berry, Senior Vice President, Global Health Policy, Government and Patient Affairs; Bill Ciambrone, Executive Vice President, Technical Operations; Bo Cumbo, Executive Vice President and Chief Commercial Officer; Ian Estepan, Senior Vice President, Chief of Staff and Corporate Affairs; Ty Howton, Executive Vice President, General Counsel and Corporate Secretary; Joan Nickerson, Senior Vice President, Human Resources; Gilmore O'Neill, Executive Vice President, R&D and Chief Medical Officer; and Louise Rodino-Klapac, Senior Vice President, Gene Therapy. In addition, Bryan McCorry and Stacey Farese of KPMG, the company's independent registered public accountants, have joined us today. This morning, our program will proceed as follows. First, I will conduct the official business of the 2020 Annual Meeting as presented in the company's proxy statement filed with the Securities and Exchange Commission. This year, the following are the 5 proposals for stockholders to vote on. First, the election of Class I directors; second, the advisory vote on named executive officer compensation; third, the approval of an amendment to the company's amended and restated certificate of incorporation, as amended, to increase the number of authorized shares of common stock from 99 million to 198 million shares; fourth, contingent upon the approval of the third proposal, the approval of an amendment to the company's 2018 Equity Incentive Plan to increase the maximum aggregate number of shares of common stock that may be issued under the 2018 Equity Incentive Plan by 3.8 million shares to 8,187,596 shares; and fifth, the ratification of KPMG LLP as the company's independent registered public accounting firm for fiscal 2020. The company has not received notice from any of its stockholders as required under its bylaws of any other matters to be considered at today's meeting, and therefore, no other proposals may be properly introduced by stockholders. After we complete the official business of the meeting, Mr. Ingram will address those attending today and open up the meeting to general question-and-answer session with stockholders on the company's business. We have a quorum present, which allows us to proceed to the official business of this meeting. We also have an affidavit from Computershare, the company's inspector of elections, certifying that the notice of Internet availability of proxy materials was mailed and deposited with the United States Post Office commencing on April 21, 2020, to each stockholder of record as of April 13, 2020. Additional proxy materials, which included a proxy statement, proxy card, annual report and other material necessary to vote at this meeting, were mailed to shareholders who requested to receive printed copies of the proxy materials. The Board of Directors has appointed Computershare to serve as inspector of election. And Emily Curcio of Computershare will determine, first, the number of shares outstanding; second, the shares represented at the meeting; and third, validity of the proxies and ballots. Ms. Curcio will also tabulate the votes for this annual meeting. I will now turn to the official business of the meeting. The first matter to be voted upon is proposal 1, the election of Class I directors. Nominations are now in order for Class I directors on the Board of Sarepta for the 2-year term expiring at the 2022 Annual General Meeting of Stockholders and until their successors are duly elected and qualified. The current Board of Directors favors the election of the following individuals: Douglas S. Ingram, Hans Wigzell, Mary Ann Gray. Do we have a nomination?

I hereby nominate for election as directors of the company to serve a term expiring on the date of the 2022 Annual General Meeting of Stockholders and until his or her successor is duly elected and qualified, Mr. Ingram, Dr. Wigzell and Dr. Gray, who are named as Director nominees in the company's proxy statement to this annual meeting. May I have a second?

I second the motion.

A motion to nominate the individuals listed in the proxy statement and announced at this meeting as directors of Sarepta for the 2-year term expiring at the 2022 Annual General Meeting of Stockholders has been made and seconded. Is there any discussion requested for this proposal? The second matter to be voted upon is proposal 2, the advisory vote on 2019 named executive officer compensation. May I have a motion?

I hereby move to approve the advisory vote on 2019 named executive officer compensation. May I have a second?

I second the motion.

A motion to approve the advisory vote on the 2019 named executive officer compensation has been made and seconded. Is there any discussion requested for this proposal? The third matter to be voted upon is proposal 3, the approval of an amendment to the company's amended and restated certificate of incorporation, as amended, to increase the number of authorized shares of common stock from 99 million to 198 million shares. May I have a motion?

I move to approve the amendment to the company's amended and restated certificate of incorporation. May I have a second?

I second the motion.

A motion to approve the amendment to the company's amended and restated certificate of incorporation, as amended, has been made and seconded. Is there any discussion requested for this proposal? The fourth matter to be voted upon at this meeting is proposal 4, which is contingent upon the approval of the third proposal, to approve an amendment to the company's 2018 Equity Incentive Plan to increase the maximum aggregate number of shares of common stock that may be issued pursuant to the 2018 Equity Incentive Plan by 3.8 million shares to 8,187,596 shares. May I have a motion?

I move to approve the amendment to the company's 2018 Equity Incentive Plan. May I have a second?

I second the motion.

A motion to approve the amendment to the company's 2018 Equity Incentive Plan has been made and seconded. Is there any discussion requested for this proposal? The fifth matter to be voted upon at this meeting is proposal 5, the ratification of the selection of KPMG LLP as the independent registered public accounting firm for the company for 2020. May I have a motion?

I move that the selection of KPMG as the independent registered public accounting firm for the company for 2020 be ratified and approved. May I have a second?

I second the motion.

A motion has been made and seconded to ratify and approve the selection of KPMG LLP as the independent registered public accounting firm for the company for the fiscal year ending December 31, 2020. Is there any discussion requested for this proposal? We will now proceed to vote on the motions for proposals 1 through 5 in the company's proxy statement, which are the proposals relating to the election of Class I directors, the advisory vote on named executive officer compensation, the approval of an amendment to the company's amended and restated certificate of incorporation, the approval of an amendment to the company's 2018 Equity Incentive Plan and the ratification of the company's auditors for fiscal 2020. Stockholders who sent in proxies or voted via telephone or internet and do not wish to change their vote do not need to take any further action. If you previously returned a proxy card and do not wish to change your vote, you do not need to vote again at this meeting. If you wish to vote at this meeting and have not yet done so, you may do so now by clicking on the Cast Your Vote button on the web portal and following the instructions at that location. [Voting]

We will now close the voting polls with respect to the 5 proposals in the proxy statement. Since the voting polls are now closed, I now move that the official business portion of this meeting be concluded. May I have a second?

I second the motion.

Thank you very much. I'll now turn the meeting over to Mr. Ingram.

Thank you very much, Dr. Behrens. Hopefully, everyone can hear me now. On behalf of Sarepta's Board of Directors and Sarepta's Executive Committee, I want to thank you all for joining us, albeit virtually for Sarepta's 2020 Annual Meeting of Shareholders. I will be making forward-looking statements today. So please refer to Sarepta's public filings with the SEC for a list of the various risks and uncertainties that are intended when making statements and predictions about the future. The next slide. This month will be my third anniversary as the CEO of Sarepta. I made the decision to join Sarepta in 2017 because I believed that we had all the elements, the science, the talent and the mission to improve and extend the lives of those with rare disease and so doing, to build one of the most profoundly important and sustainable genetic medicine companies in biotech. Let's quickly review where we have come since 2017, a short 36 months ago. In 2017, we had one recently approved therapy, and that was EXONDYS, generic name eteplirsen. As Sarepta had been single-mindedly focused on the approval of EXONDYS for a number of years, there was good science and some dedicated professionals at Sarepta, but there was little in R&D moving forward behind it, and there were no concrete plans for the future. By the end of 2017, we had built a strong operational plan for the future and a vision to become a leading genetic medicine company focused on rare disease. By 2018, we articulated what we saw at the time and what we still see as a revolution taking place in the science of genetic medicine, and we find our vision to become the leader of this revolution for the benefit of rare disease patients founded on 2 platforms: our RNA platform, focusing on our current PMO technology, but also advancing our next-generation PPMO program; and second, on our intention to build a gene therapy engine, pursuant to which we would build a first-in-class gene therapy team of scientists and manufacturing prowess and bring forward and rapidly develop high probability of success gene therapy candidates to treat well-characterized rare serious genetic disease. Over the course of 2018 and 2019, we executed diligently and passionately to bring the vision into sharp focus and to make our [indiscernible] strategy from well-articulated idea, to actionable reality. Next slide. We did this by focusing on the following elements that you will see on this slide. We strengthened our balance sheet to meet our strategic objectives, both through equity raises and non-dilutive finances. We deepened our pipeline, both through internal research and development and through in-licensing and collaborations. In fact, from late 2017 through today, we have executed 36 business development transactions. We advanced our clinical development. As we sit here today, we have 10 ongoing human clinical trials. We strengthened our infrastructure. We added talent across the globe, building gene therapy prowess second to none and building next-generation gene-editing capabilities as well. We focused on becoming world leaders in gene therapy manufacture -- manufacturing, going from an idea in early 2018 to today, where we have among the world's greatest gene therapy capacity and world-leading expertise in gene therapy process development and analytical development. We expanded our alliances, realizing that we can multiply our impact by aligning with the world's best and brightest. Today, we have 23 commercial alliances and close alliances with the world's top academics and thought leaders in genetic medicine and gene therapy to drive our thinking and to progress. And chief among our collaborations, of course, is our ex U.S. relationship with Roche to bring our lead gene therapy candidate, which is SRP-9001, to children around the world who are living with and degenerating from Duchenne muscular dystrophy. And we have broadened our reach, from a company in 2017 that was solely focused on Duchenne muscular dystrophy, to a company with programs and research efforts in over 23 serious life-limiting and far too often life-ending rare diseases. Next slide. Here is Sarepta in a snap shell -- in a snapshot today. In 2017, we were less than 200 employees. Today, we have around 800, slightly under 800 employees. And if one counts our contingent workers, we are over 1,000 dedicated professionals. That is an enormous expertise in genetic medicine. That's scientists, it's geneticists, biologists, chemists, et cetera. It's regulatory experts, CMC, technical operations, manufacturing, process development, analytical development, clinical operations, pharmacovigilance, biostatistics and the list goes on and on, all to fuel our growth and enhance our expertise. We now have 13 global locations. Our corporate center is in Kendall Square Cambridge, the so-called Most Innovative One Square Mile in the world. We have manufacturing and process development and analytical development expertise in Burlington and Andover, Massachusetts. Importantly, we have built our world-leading Gene Therapy Center of Excellence in Columbus, Ohio. And we are in our facility in building our Gene Editing Innovation Center right now in Durham, North Carolina. We now have 43 pipeline programs, and we have 3 platforms now. We have RNA, we have gene therapy. And as I've just mentioned, we have gene editing research as well and that's being headed up by Dr. Charlie Gersbach in Durham, North Carolina in our Gene Editing Innovation Center. We have 2 approved therapies. And if all goes well by the first quarter of next year, we will be among the very few biotechs that have 3 internally developed and approved therapies in the United States. And to fuel our ambitions and to ensure that we are not taken off mission or distracted by things like this global pandemic, we have a very strong balance sheet right now with $2.2 billion in cash as of the end of Q1 of this year. Next slide. This is a breakdown of our 43 programs. Time obviously does not permit me to go into them all in any depth, but you will see that our pipeline is today among the most significant in all of biotech. Now as profound as the last 3 years have been, and I will say, I am extremely proud of what this team at Sarepta has accomplished in a mere 36 months, the next 12 to 18 months will be monumental for us at Sarepta and for the patients that we serve. We knew that would be the case as we tracked into 2020. And I am very pleased that even in the face of this global COVID pandemic, we have been able to stay on mission and largely on track, due in part to go-forward planning and a strong balance sheet, but also due to fabulous work by the passionate, mission-driven Sarepta workforce. And I really thank them for their hard work during this difficult and distracting period of time. Our most advanced and singularly important program is SRP-9001, our gene therapy to treat Duchenne muscular dystrophy. Our placebo trial, Study 102, is on track and will be complete for its main analysis by the end of this year, and we will report that data out early next year. We plan to have GMP material for our next trial and for commercial purposes as well by July next month. We plan to gain insight from the FDA and to commence our next trial in the second half of this year. We plan to have safety and dosing insight readout for our PPMO program, SRP-5051, that's our next-generation PMO platform in the second half of this year. This will be a proof-of-concept for PPMO certainly, but not only for Duchenne, also for its potential in other therapeutic areas. We will complete our rolling submission for casimersen this month, and we will get the readout for the high-dose cohort in our first LGMD program, LGMD2E, this month. And this will provide dosing insight for 2E and across our LGMD platform. We plan to commence our pivotal trial for LGMD2E in 2021, and we are already in GMP manufacturing runs for that trial today. So a lot is going on this year, and there will be a lot of milestones in 2020. In summary, we have come a long way in a short 3 years. We crafted what may have seemed a few years ago an audacious vision to become the leader in genetic medicine, and then we fiercely executed to make that a reality. If we are successful in the coming few years, we will not merely realize the success that we have been building toward in the last 3 years, but we will profoundly extend and improve the lives of thousands, perhaps tens of thousands of young people who would otherwise be confined to certain degeneration. We never drift far from the understanding that what we do is life or death for the patients that we serve. And so we intend to keep executing. Thank you. And with that, I will turn the meeting back over to Dr. Behrens.

Thank you, Doug. At this time, we will take questions or comments from our stockholders -- excuse me, from your stockholders, apologies. [Operator Instructions] Please note, we will attempt to answer as many questions as time allows, but only questions that are germane to the meeting will be addressed.

This is Ian. Just due to the virtual nature of the meeting, we have received questions from shareholders. So I will read them, and Doug can respond accordingly. So to start with the first question. Doug, on the 9001 program, what is the status of our assay development?

Those who will remember at my last earnings call, we mentioned that there were 24 assays necessary to be built, qualified or validated. And that as of the earnings call, we were very proud of the fact that of those 24, all had been built, and 22 of 24 had either been qualified or validated as required. I'm pleased to say that as we sit here today, all 24 assays have been built, of course, and all of them have been validated and/or are qualified depending on the requirement for each. So our assay development work is complete.

Thank you. Regarding our publication of the 1-year data, when is that supposed to come out?

So at the last earnings call, you will remember that I said it was coming out imminently as it has been accepted, was in the publication process. And I was informed it would be out literally in a matter of days. Obviously, I do not control the timing of publication. And given the difficult circumstances of late, there may have been some delays from a publication perspective that are beyond our control. But with that said, I am told with some confidence that it will be -- it will issue this month. So it is coming, it is coming soon, and it should be a June event.

Thank you. Is Sarepta waiting for a commercial manufacturing facility to be up and running and validated before proceeding with the limb-girdle programs?

That's not the -- so we'll not -- we have capacity earmarked for LGMD, indeed, as part of our agreement with Paragon at the time, now Catalent-Paragon. One of the elements of that agreement was to provide us capacity for limb-girdle 2E and beyond. But this is what is going on right now, and we're making great progress with the limb-girdle franchise. LGMD2E will inform our work across the LGMD franchise. Remember, we have 5 programs from Myonexus in a fixed program out of Nationwide Children's Hospital for a total of 6 LGMD programs. It will take the greater part of 2020, but our goal is to do the following 3 things: one, dose selection. So we'll have a readout on 2E. On the high dose of 2E, we said it would be in the second quarter. That, of course, means it's going to happen in June. If we meet our timelines, and we certainly do intend to meet our timelines, and then we'll make a dose selection on 2E. That dose selection will inform 2E and beyond 2E. We then need to complete the ADPD and GMP runs for 2E for trial. There's a lot of work to be done there. But as I have said earlier, we are making great progress there. In fact, we're already in GMP runs for 2E. But that will take the better part of this year. We will then define, at the same time, in parallel, we will define the clinical and regulatory pathway for limb-girdle 2E. This work will inform the path for the rest of the sarcoglycans and then the remainder of the pipeline. So this is very important that we get this right. And there are, as I said, 6 candidates there. We will be able to provide clarity on the flight path for LGMD pipeline in early 2021 the process right now, that again is dose selection, which we can do in relatively short order once we have the high-dose data available and released. And then we needed to do 2 other things that will take some time in parallel. And that is, of course, manufacturing and the other is dialogue with the agency and defining the regulatory and development pathway. We've already commenced those discussions. But that is going to be initial discussions needed as we're framing that out with the agency, educating one another and come into alignment, and we have the time to do that because we're working on manufacturing at the same time.

Shifting to a question on our RNA platform. When will the PPMOs enter the clinic to treat numerous different DMD population?

Well it is going to be premature for us to give that answer now. We need to get the dosing and safety insight from our first program in the second half of this year. And that is, of course, our goal. And we are very pleased that we are already dosing in our multi-ascending dosing trial at 20 mg per kg, so things are certainly proceeding there. Armed with that, we'll be able to build out plans for not just SRP-5051, but also the remainder of the programs, and then we'll communicate our perspective. And I would assume that we can get all that done, and we'll be able to do that early next year, after we have reviewed the data and after we've had conversations with the agency and really considered the most efficient way to provide this therapy as fast as possible to as many patients as is possible that are waiting for a therapy like this. We have constructs already built, so we know how to treat over 50% of the Duchenne muscular dystrophy community. And we can build constructs to treat about 85% of the Duchenne muscular dystrophy completely. There are some exons and mutations that are not amenable to exon skipping, but there appear to be at least 85% or so of patients that could benefit from exon skipping, and we could build constructs. To get to that last 35%, we will need a creative approach, a different sort of platform approach, and we have already had early discussions before this year with the agency about that. And we'll need -- we would need to come to an understanding with the agency on the ways we could efficiently build that out to get to that ultra-rare exon. But if we did, we could go even beyond that 50% all the way up to about 85%. That is all work that we're going to do over the course of this year. Once we see the data, then we'll come back next year and talk about it. But be assured, our goal if we see positive signals in the PPMO, is to be as thoughtful and creative as possible in concert and collaboration with our regulatory agencies to move these therapies along as fast as possible because we never forget that patients are waiting for us.

Perfect. Switching gears back to our gene therapy platform and specifically 9001. I think this is in response to Pfizer putting out data using mass spec. So the question is, will you collect mass spec quantification data?

We have a [ mere ] expert translational group that looks at numerous new and experimental measures, and they considered a lot of things like mass spec and other approaches to looking at biomarkers and the like. But we are focused on the one thing that has been the gold standard. And the only quantification method that to date has ever been accepted by the agency and something that we are very expert at accomplishing, and that is, of course, Western blot. So our primary approach for our therapies is the quantification of dystrophin or truncated dystrophin or micro-dystrophin using Western blot. And then secondarily, we're obviously focused on immunohistochemistry or fluorescent -- immunofluorescence. And in that regard, that's of course, dystrophin-positive fibers, but as important, intensity so we look at those things. But from a pure quantification perspective, we rely on the gold standard and the one thing that's been reliably approved by the agency, and that's Western blot.

Okay. A question on both our RNA and gene therapy platform. When will the PMO, PPMOs enter the clinic with the goal to enhance microdystrophin expression?

Well look, we are -- this is a very important issue, and we are very keenly interested in and potentially excited about the combination therapy of a chronic PPMO and gene therapy together and the potential that, that could enhance the value of these therapies should these children extend their lives and better their lives. But it is too soon to know the answer to this. We must first do the preclinical work to support the hypothesis, that the combination of PPMO with microdystrophin will provide a synergistic benefit to DMD patients. And we're doing that right now. The [ leasing ] group are doing a lot of work on that. Once we have that in hand, we'll review that data. And if it's encouraging, we'll make decisions regarding clinical approaches. But before we get to a clinical pathway, we have to confirm the hypothesis that there is a combination value between these 2. And there certainly have -- there's certainly a good reason to believe that, that is a reasonable hypothesis. And there is already some literature about the combination value of RNA and gene therapy together in certain circumstances, but we have to do more work on that before we could come up with a clinical pathway.

Okay. Here's a question on our research agreement with USAMRIID in regards to COVID-19. What data do we have for COVID PPMO? And when will clinical work commence?

Yes. We are, of course, proud to have done our part in this COVID crisis when called upon to do so. As you may recall, just for background, everyone, we were approached by the Department of Defense and its laboratory, USAMRIID, about whether we would be willing to collaborate with them and build constructs that they could test as potential therapies for COVID-19 on their belief. And I believe that we understand this and is supported by science that our PPMO technology might provide a benefit, might be a valid approach to treating COVID-19. We built numerous PPMO candidates in collaboration with the Department of Defense-USAMRIID, and that was our role. Now USAMRIID's role is to do the in vivo work now to test them. We don't have those results back yet at all. Once we do get them back, then, of course, we'll sit down, we'll look at them with USAMRIID. And then the Department of Defense and Sarepta, armed with that data, will evaluate the utility, and then we'll discuss with the Department of Defense what the next steps would be. But at this point, they're still getting their work done on that -- on those constructs.

Okay. Another question on our PPMO platform. Will you -- for SRP-5051, will you include an exon 51 skip arm in your Phase III study?

So we have not completed our strategy yet. We have the -- and we don't have a stated view yet on PPMO development. Once -- as I've said earlier, once we have dosing and safety insight, we are going to build out our entire strategy for the PPMO, both for -- not merely 5051, but the best -- the potential with Duchenne muscular dystrophy. And then, frankly, we're going to consider what that dosing and safety insight means for the ability to bring the PPMO into other areas. And then we'll communicate all of that data. But we first have to get the results of our dosing and safety insight before we can really do that work and provide a meaningful flight path and potential protocols. One thing I will say is, and I would remind folks that our first 2 therapies in RNA, the PMOs, were based on an accelerated approval pathway on the basis of the ability to quantify meaningful amounts of truncated dystrophin. And so we have not had conversations with the agency about this topic, and we don't even have the proof-of-concept in hand. But certainly, when we have those conversations, front of mind for us will be the fastest approach that brings if it proves to be a meaningful improvement on our RNA technology to kids that are waiting. And in that regard, of course, we would be having a very robust discussion with the agency about the use of the accelerated pathway approach and using dystrophin as a biomarker reasonably likely to lead to clinical benefit as part of that clinical trial. But that will require us to get the data from the proof-of-concept to build the strategy and, of course, to have conversations with our regulator, the FDA and so as their views on that.

Okay. Another question on our LGMD2E drug candidate. Last fall, the company was scheduled to meet with the FDA on a pathway forward for LGMD constructs. Did that meeting happen? And what is the FDA's guidance?

So we are very much on track for what we have said historically about our pathway to developing a development and regulatory approach that is efficient and as fast as possible, not only for 2E, but for all of the limb-girdles that we're working on. What we have said is that we need to work with the agency over time to devise an appropriate development and regulatory pathway for 2E and the other programs. And what we also said is that we have the luxury of some time to do that because we have another rate limiter at the same time, which, of course, is our manufacturing process development, analytical development, GMP runs and the like. We have been in dialogue with the agency, and we expect those discussions to take place over the course of 2020 in parallel with our manufacturing work, which will also take the bulk of 2020 as well. Our goal is to have those discussions complete and then we'll provide insight on 2E and limb-girdle generally in early 2020. As you can imagine, our goal is to take the time and the education and the discussions with the agency to come to what would be the most efficient and appropriate pathway for the limb-girdles. And limb-girdles are -- have some unique aspects to them. These are gene therapies that deal not only with well-characterized monogenic diseases, they -- and they not only address structural proteins and missing structural proteins that are being replaced but in the -- for most of these limb girdles that we're working on, we are literally restoring the native protein, the loss of which is causing and is the sole cause of the degeneration and death in these patients. And these are also on top of that, at least with respect to 2E, extremely rare therapeutic areas. And on that basis, we think we have a very -- the ability to have very good dialogue about a very efficient, very fast approach to bringing these therapies to waiting patients. We're going to continue that dialogue over the course of this year just as we continue our manufacturing, and then we'll have a readout on all of that early next year.

Okay. A question on our PMO platform and casimersen, specifically. What's the status of the NDA? Is there anything left for the company to respond to? And when can it be expected to be accepted by the agency?

Well, actually, we're in the midst of the submissions. We're in a rolling submission for the NDA. We have said that the submission would be completed in the second quarter. And given that this is June, it means that we have plans to complete that submission this month. Thereafter, the agency reviews and accepts it, and then we'll have our PDUFA date. So we are very much on track with casimersen.

Okay. Another combined question for our gene therapy platform and COVID. The question is, Novartis announced that they will develop a vaccine using AAV, which may be reactive to AAV gene therapy. What is the risk of this vaccine being reactive or becoming a lead vaccine?

So first, let me just be clear. We have no position on whether this approach, this viral approach is it could be a lead vaccine or is independent of the other issues, is a meaningful approach, whether you could actually manufacture it, you could manufacture it at scale. There are lots of issues there. But to your very good question, we do worry from the sidelines for all gene therapy and for patients whose lives could be saved by gene therapy if an AAV-mediated vaccine was actually developed and generally available. To remind us all, AAV is a delivery device for hundreds and hundreds of therapies that have the potential to improve and extend the lives of literally theoretically, hundreds of millions of patients around the world living with and dying from genetic disease. It's been an amazing potential delivery tool that health care is working on. If one used AAV as a broadly available part of a vaccine, and if that resulted in widespread seroconversion, it would be a travesty. And it would be a travesty at a magnitude that would swamp the harm that is being done right now by, believe it or not, the COVID pandemic. And so of course, this is something that everyone has to take very seriously. And I'm sure that the companies like Novartis, who are certainly interested in AAV and the use of AAV to bring a better life to patients and, frankly, that their [ therapies ] are bringing a better life to patients with AAV even as we speak. I'm sure this is a concern for them. We are still investigating this issue. There are some -- we've heard some viral approaches that are not AAV. I think Johnson & Johnson announced that as adenovirus. And we've heard from others that they believe that they have approaches that did not create AAV -- other AAV seroconversion. But those are the initial responses we're getting. And while the initial responses are encouraging on their face, we're still investigating this. And we strongly believe that no virus-mediated vaccine should even be considered until it is certain that AAV seroconversion is not possible so that we don't, in the rush to do something that is enormously important for all of us in society, and that is to develop a vaccine for COVID-19. We don't do something that inadvertently takes away from patients waiting for life-saving therapies the ability to use AAV in gene therapy, something that people have been working on for 30 years to get to where we are today. So it is an important issue. We don't have enough information to know if there is a real issue with it yet. We're still investigating that, but it's something that should be on people's minds as we consider the various approaches to vaccines to treat this horrible COVID-19 pandemic that we're dealing with now.

Thank you. All right. So for our last question, and it happens to be a funny one. It's probably directed to Bo, but I'll ask you, Doug. What percentage of executive pelotons have turned into expensive clothes hangers during this pandemic?

Okay. Well, look, mine hasn't. I use mine every day. And it's -- I use it more -- it looks more like a shower when I'm done with it than a clothes hanger. I will say this, I'm going to answer this seriously. I am proud that our Sarepta workers are not only getting their work done and the missions served, but that they have taken the time to have balance in their lives as well. And it's hard. This is really hard for a lot of people to have balance in their lives right now. This work-from-home environment that we live in right now has surprising efficiencies associated with it. But in those efficiencies, it can sometimes be grueling for people, and then they have a lot of other things to worry about: family issues, child care issues and the like. So I actually am happy that our employees and workers are taking time to attend to their families and to their loved ones, and that they're taking time for themselves and that they're taking time to move and keep healthy. And in fact, that does include pelotons and bikes and running and walking and dancing, hiking and yoga and the like. And for those outside the company, you will not know that I do a weekly video. And in almost every video, I encourage, cajole, pressure people to get out and move and make sure they have balance. And I think it is important that for all of us that we not only get through these difficult times, but that we get through these times and at the same time, do our very best to remain sane. There are some people like Bo, it's too late for him. But for the rest of us, remain sane and also healthy during this difficult period of time. Are there any other questions?

No. That concludes the Q&A portion. So I would like to turn the meeting back over to Dr. Behrens.

So first, thank you very much for all these questions. I personally enjoyed the last one, so thank you. I'd now like to ask Ms. Curcio to summarize the tabulation of stockholder votes on the proposals raised at this annual meeting.

Thank you, Dr. Behrens. The preliminary tabulations of results are as follows: proposal 1, election of directors. Our preliminary tabulation of votes received, immediately prior to and at this meeting, indicate that the director nominees named in the proxy statement: Mr. Ingram; Dr. Wigzell; and Dr. Gray, have been elected to serve for the 2-year term expiring at the 2022 Annual Meeting of Stockholders and until his or her successor is duly elected and qualified. Proposal 2, advisory votes on named executive officer compensation. Our preliminary tabulation of votes received, immediately prior to and at this meeting, indicate that the advisory vote on 2019 named executive officer compensation has been approved. Proposal 3, the approval of an amendment to the company's amended and restated certificate of incorporation, as amended. Our preliminary tabulation of votes received, immediately prior to and at this meeting, indicate that the amendment to the company's amended and restated certificate of incorporation, as amended, has been approved. Proposal 4, the approval of an amendment to the company's 2018 Equity Incentive Plan. Our preliminary tabulation of votes received, immediately prior to and at this meeting, indicate that the amendment to the company's 2018 Equity Incentive Plan has been approved. Proposal 5, ratification of the selection of KPMG as our independent registered public accounting firm for 2020. Our preliminary tabulation of votes received, immediately prior to and at this meeting, indicate that the selection of KPMG LLP as the independent registered public accounting firm for the company for 2020 has been ratified and approved.

Thank you, Ms. Curcio. And thank you all for attending Sarepta's Annual Meeting of Stockholders. The meeting is now adjourned with respect to all matters. I hope you enjoy the rest of your day and continue to stay safe. Thank you again.

Thank you for participating. You may now disconnect.