Q1 2020 Earnings Call

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Ladies and gentlemen, and welcome to the Sarepta Therapeutics first quarter 2020 earnings call. At this time, all participants are not listen only mode. After the speaker presentation. There will be a question and answer session ask a question James especially when you want me to press Star one on your telephone if you acquire any for their system.

He's supposed started zero as around minded studies program is being recorded and now I'd like to introduce your host for todays program in S. Upon senior Vice President Chief of staff and Corporate Affairs. Please go ahead.

Operator: Good morning, ladies and gentlemen, and welcome to the Sarepta Therapeutics first quarter 2020 earnings call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star one on your telephone.

Thank you all for joining today's call earlier today, we released our financial results the first quarter 2020.

A press release is available on our website at Www Dot Sarepta Dotcom and our 10-Q was filed with the FCC earlier this afternoon.

Joining up on the call today, or Doug Ingram, Sandy Mahatme me full combo, Dr. Gilmore O'neil and Dr. movies routine Oakley back after our formal remarks, well open up the call for QNX I'd like to note that during this call will be making a number of forward looking statement. Please take a moment to review our slides on the webcast which contain.

Operator: If you require any further assistance, please press star zero. As a reminder, today's program is being recorded. And now, I'd like to introduce your host for today's program, Ian Estepan, Senior Vice President, Chief of Staff, and Corporate Affairs. Please go ahead.

Ian M. Estepan: Thank you all for joining today's call. Earlier today, we released our financial results for the first quarter of 2020. The press release is available on our website at www.sarepta.com, and our 10-Q was filed with the SEC earlier this afternoon. Joining us on the call today are Doug Ingram, Sandy Mahatme, Bo Cumbo, Dr. Gilmour O'Neill, and Dr. Louise Rodino-Kleypak. After our formal remarks, we'll open up the call for Q&A. I'd like to note that during this call, we'll be making a number of forward-looking statements. Please take a moment to review our slides on the webcast, which contain our forward-looking statements. These forward-looking statements involve risks and uncertainty, any of which are beyond Sarepta's control. Actual results can materially differ from these forward-looking statements, and any such risk can materially and adversely affect the business, the results of operations, and the trading prices for Sarepta's common stock.

In our forward looking statement.

These forward looking statements involve risks and uncertainty any of which are beyond sarepta 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 the trading prices for surface common stock for a detailed description of the clickable risks and uncertainty we incur.

I need you to review our daughter companies. Most recent annual report on form 10-Q filed with the Securities and Exchange Commission as well 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 as Dan.

With that let me turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress Doug.

Ian M. Estepan: For a detailed description of applicable risks and uncertainty, we encourage you to review the company's most recent annual report on Form 10-Q filed with the Securities and Exchange Commission, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances. With that, let me turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress.

Thanks, Julie and.

Good afternoon, and thank you all for joining us for Sarepta Therapeutics first quarter 2020 financial results Conference call.

Let us begin with a topic that is on all of our collective mines, which opened 19.

I'm very proud that the Sarepta team. So rapidly responded to this crisis focusing on the safety and welfare of our patients in our workers, while ensuring that our operations run smoothly and that our programs precede.

We are able to do this not only due to our execution oriented ability to adapt and remain focused but also because a good advanced the planet.

Douglas S. Ingram: Thank you, Ian. Good afternoon, and thank you all for joining us for Sarepta Therapeutics' first quarter 2020 financial results conference call. Let us begin with a topic that is on all of our collective minds, COVID-19. I am very proud that the Sarepta team so rapidly responded to this crisis.

Numerous up our functions, including in particular information technology group, our clinical operations function in our commercial organization began contingency planning. It's early in January of 2020.

Douglas S. Ingram: Focusing on the safety and welfare of our patients and our workers while ensuring that our operations run smoothly and that our programs proceed. We are able to do this not only due to our execution-oriented ability to adapt and remain focused but also because of good advanced planning. Numerous of our functions, including, in particular, our information technology group, our clinical operations function, and our commercial organization, began contingency planning as early as January of 2020 in the event that what came to be called COVID-19 became a crisis. For that reason, we were able to rapidly respond to the situation on COVID. By Friday, March 13, I had ordered that all but a small but dedicated facility-dependent staff would work from home. And because of our advanced preparations, we had the systems and the infrastructure in place to seamlessly transition, execute, and remain connected as we commenced work that Monday morning.

In the event that what came to be called killed the 19 became a crisis.

For that reason, we were able to rapidly response and situation a little bit by Friday March 13th I had ordered that all but a small but dedicated facility dependent staff would work from home and because of our advanced preparations have the systems and infrastructure in place to seamlessly transition.

Execute and remain connected as we commenced work that Monday morning.

About 10% of our workforce is designated as facility to pad and we have worked at the cheap safe and comfortable as they come into the facility to run labs and experiments continue our manufacturing activities and alike.

We also immediately reached out to our partners suppliers and other important third parties to assess new working arrangements with them and to assure that they and we would continue to deliver and we reached out to our patients and patient advocates to ensure that we were fulfilling their needs to the fullest extent possible.

Douglas S. Ingram: About 10% of our workforce is designated as facility-dependent, and we have worked to keep them safe and comfortable as they come into the facility to run labs and experiments, continue our manufacturing activities, and the like. We also immediately reached out to our partners, suppliers, and other important third parties to assess new working arrangements with them and to assure that they, and we, would continue to deliver. And we reached out to our patients and patient advocates to ensure that we were fulfilling their needs to the fullest extent possible. It is for these reasons, and others, that while COVID-19 has created some challenges for Sarepta and for our plans, Sarepta is in a privileged position even in these difficult times. Yes, as with all biotech companies, COVID-19 has created some challenges and uncertainty and will continue to do so for the next few quarters. All in all, we are uniquely positioned to drive our strategic plans and to stay on mission during these difficult times. In that regard, consider the following. First,

It is for these reasons and others that well Cobot 19 has created some challenges for SREP there for our plans sarepta is any privileged position even in these difficult times, yes, as with all biotech companies cope at night team has created some challenges and uncertainties.

And we'll continue to do so for the next few quarters.

Well in the whole, we're uniquely positioned to drive our strategic plans and to stay on mission during these difficult times.

In that regard to consider the following.

First anticipating that we would need greater certainty around cash and capital in 2020 M. beyond 2020.

In late 2019, we entered into the Roes transaction, which added another $1.15 billion to our balance sheet.

Do stubbornly through cross sharing and entitles us to milestones along the development.

And regulatory pathway.

Along with our Roche infusion at the end of the first quarter, we had nearly $2.2 billion of cash on hand.

Douglas S. Ingram: Anticipating that we would need greater certainty around cash and capital in 2020 and beyond 2020, in late 2019, we entered into the ROSE transaction, which added another $1.15 billion to our balance. Do start early through cost-sharing and entitles us to milestones along the development and Regulatory Pathways. Along with our Roche infusion, at the end of the first quarter, we had nearly $2.2 billion of cash on hand, not including another $250 million in a debt facility that we have not drawn down.

Including another $250 million in a debt facility that we have not drawn down.

This level of cash on hand places us in a rarified position among biotech companies and of course that is before considering our revenue from EXONDYS 51 by on this 53.

If and when approved CASM your son.

We are in a strong position to whether any cobrand related uncertainties.

Stayed focused on executing our plans.

And emerged from this pandemic on track.

Second.

The greatest development challenge reported in the biopharmaceutical industry right now is the commencement of new trials. Fortunately, we are already in Tenda human clinical trials, all of which are intact and progressing and our talented development and clinical operations team worked diligently to minimize the into.

Douglas S. Ingram: This level of cash on hand places us in a rarefied position among biotech companies, and of course, that is before considering our revenue from Exondys 51, Biondys 53, and, if and when approved, Casimir's. We are in a strong position to weather any COVID-related uncertainties, stay focused on executing our plans, and emerge from this pandemic on track.

Cobot 19.

Moreover, with respect to study what our to our gene therapy trial for micro dystrophin.

This is a onetime therapy at all patients for the primary 48 week analysis have been dose that trial progressive and there is no foreseeable risk of a delay in trying to leader.

Douglas S. Ingram: The greatest development challenge reported in the biopharmaceutical industry right now is the commencement of new trials. Fortunately, we are already in 10 human clinical trials, all of which are intact and progressing, and our talented development and clinical operations team work diligently to minimize the impact of COVID-19. Moreover, with respect to study 102, our gene therapy trial for microdistrophy,

Next with respect to our currently available therapies excited this and beyond this for the treatment of Duchenne muscular dystrophy.

Our supply chain is fully intact, and we are able to manufacture and supply therapy without any interruption and we do not anticipate is changing.

And finally, one of our most significant strategic activities is building out commercial gene therapy manufacturing capacity.

Douglas S. Ingram: This is a one-time therapy, and all patients for the primary 48-week analysis have been dosed. The trial progresses, and there is no foreseeable risk of a delay in trial reading. Next, with respect to our currently available therapies, Exondus and Biondus for the treatment of Duchenne Muscular Dystrophy, our supply chain is fully intact, and we are able to manufacture and supply these products without any interruption, and we do not anticipate this change. And finally, one of our most significant strategic activities is building out commercial gene therapy manufacturing capacity, but we have made less progress in the last 12 months.

We made less progress in the last 12 months Cobot 19 could have caused significant disruption.

Fortunately our progress has kept us on track during this crisis the necessary facilities and suites are largely build out.

All of our ask phase our bills.

And all but two of the 24 necessary ask days are either already validated or qualified as required.

With the remaining two near completion, even as we speak our process development is complete and our engineering runs and commercial GNP runs are on track, hence notwithstanding this pandemic we remain on track to have SRP nine 001 GMP material. This July as originally.

Douglas S. Ingram: COVID-19 could have caused significant disruption. Fortunately, our progress has kept us on track during this crisis. The necessary facilities and suites are largely billed out.

The anticipated.

Douglas S. Ingram: All of our assays... and all but two of the 24 necessary assays are either already validated or qualified as required, with the remaining two near completion even as we speak. Our process development is complete, and our engineering runs and commercial GMP runs are on track. Hence, notwithstanding this pandemic, we remain on track to have SRP 9001 GMP material this July, as originally anticipated. With that, I will comment on current period performance, and then I will touch on the status of some of our most significant development programs. I am pleased to report that in the first quarter, our net sales were $100.4 million.

With that I will comment on current period performance and then I will touch on the status of some of our most significant development programs.

I'm pleased to report that in the first quarter, our net sales were $100.4 million.

How does a 15% increase over the same period last year.

There was a modest impact on revenue in the quarter as a result of the cobot crisis, but as you can see from our reported performance. It was not significant looking forward. While we have a number of elements that are encouraging and protected such as the high percentage of infusions that occur in home.

Douglas S. Ingram: That is a 15% increase over the same period last year. There was a modest impact on revenue in the quarter as a result of the COVID crisis. But as you can see from our reported performance, it was not significant. Looking forward, while we have a number of elements that are encouraging and protective, such as the high percentage of infusions that occur in homes, we do anticipate that COVID-19 will have a negative short-term impact on revenue. For instance, some patients may have difficulties getting or keeping infusion appointments in hospitals. This impact will be more significant for biondus, which is just launching, than for exondus, as most existing patients are already on home infusions for that treatment. Additionally, while we anticipate that this will not occur often, some patients could forego an infusion to avoid a third party in their homes during the peak of this crisis.

We do anticipate the Tobin 19 will have a negative short term impact.

To revenue.

For instance, some patients may have difficulty getting are keeping infusion appointments in hospitals.

This impact will be more significant BYOD. This which is just launching the next saunders as most existing patients already on home infusion for that treatment.

Additionally, while we anticipate that this will not occur often some patients could forego infusion to of weight of third party and their rooms.

During the peak of this crisis.

Finally, and he payer delays in processing Reauthorizations could impact revenue, although we assume that payers both state and try that will understand the duty in these difficult times to make reauthorization efficient I will not take advantage of this crisis to profit by slowing the reauthorization process.

And we're working with payers and patients to remove any tobin related roadblocks to we authorizations.

We currently anticipate the kobin related impact on sales can be both modest and shortly.

Douglas S. Ingram: Finally, any payer delays in processing reauthorizations could impact revenue, although we assume that payers, both state and private, will understand the duty in these difficult times to make reauthorization efficient and will not take advantage of this crisis to profit by slowing the reauthorization process. And we are working with payers and patients to remove any COVID-related roadblocks to reauthorization. We currently anticipate the COVID-related impact on sales to be both modest and short-lived.

Given the dynamic and Unprecedent nature of this pandemic. However, we do not have sufficient clarity yet to accurately forecast and provide updated revenue guidance that reflects the impact of the virus, we will monitor and on our second quarter earnings call will provide an updated view Lou.

Turning to our clinical programs as noted above our plans remain intact with only a modest impact on the timing of some programs anticipated.

With respect to SRP nine 001, micro dystrophin gene therapy program, our study one or two evaluating the safety and efficacy of SRP nine 001 in patients would be Shen muscular dystrophy.

Douglas S. Ingram: Given the dynamic and unprecedented nature of this pandemic, however, we do not yet have sufficient clarity yet to accurately forecast and provide updated revenue guidance that reflects the impact of the virus. We will monitor it, and at our second quarter earnings call, we'll provide an updated view. Moving to our clinical program. As noted above, our plans remain intact, with only a modest impact on the timing of some programs anticipated. With respect to SRP9001, our microdystrophin gene therapy program, our study 102, evaluating the safety and efficacy of SRP9001 in patients with Duchenne muscular dystrophy, is proceeding, and it is in good shape.

Is proceeding and it didnt good shape.

All patients for the 48 week analysis have been dosed and while there were some delayed functional visits we work to minimize any disruption and documented the few delays in accordance with the FDA guidance on this topic and our statistical analysis indicates there was little risk to the powering award the integrity of the study.

We anticipate the going forward there should be view substantial delays.

We have two sites for study one or two.

Our site at nationwide children's hospital with Dr., Jerry Mendell, it's priced principal investigator impose restrictions on some in hospital visits however nationwide children's hospital has loosened those restrictions already while maintaining the safety of our trial participants our site. It you CLA with Dr. Perry said.

Douglas S. Ingram: All patients for the 48-week analysis have been dosed, and while there were some delayed functional visits, we worked to minimize any disruption and documented the few delays in accordance with the FDA's guidance on this topic, and our statistical analysis indicates there is little risk to the powering or the integrity of this study. We anticipate that going forward, there should be few substantial delays. We have two sites for Study 102. Our site at Nationwide Children's Hospital, with Dr. Jerry Mendel as principal investigator, imposed restrictions on some in-hospital visits.

Hey, as investigator continued throughout two permit visits uninterrupted. So in short study one or two is on track. It is progressing well and it is set to read out in the first quarter of 2021 as anticipated.

As it relates to the commencement of study three one we continue to progress to remind you studies real one is our planned multicenter multi country study for SRP nine 001, using current commercial process material.

Douglas S. Ingram: However, Nationwide Children's Hospital has loosened those restrictions already while maintaining the safety of our trial participants. Our site at UCLA, with Dr. Perry Shea as investigator, continued throughout to permit visits uninterrupted. So, in short, Study 102 is on track, it is progressing well, and it is set to read out in the first quarter of 2021 as anticipated. As relates to the commencement of Study 301, we continue to progress. To remind you, Study 301 is our planned multi-center, multi-country study for SRP 9001 using commercial process materials.

We are continuing to make progress towards the initiation of trial sites. However, cobot 19 does create some challenges here.

While the team is making progress Coburn 19 creates uncertainties around the status of some clinical sites.

And we'll likely delay some necessary site initiation visits.

Moreover, we do not want to promote dosing at sites until we are confident not merely that they can initiate but that will be able to remain operational can dose.

Can consistently and timely assess.

Douglas S. Ingram: We are continuing to make progress toward the initiation of trial sites. However, COVID-19 does pose some challenges. While the team is making progress, COVID-19 creates uncertainties around the status of some clinical sites and will likely delay some necessary site initiation. Moreover, we do not want to commence dosing at sites until we are confident, not merely that they can initiate, but that they will be able to remain operational, can dose, and can consistently and timely assess Parkinson's. We had anticipated commencing study 301 around the middle of 2020. We are on track to have GMP material for Study 301 by July of this year. However, in light of COVID-19, we may modestly delay initiation, but we'll still anticipate commencing Study 301 in the second half of this year. Moving to LGMDQE.

Participants.

We added anticipated commencing studies real one around the middle of 2020.

We are on track to have GMP material for study real one but by July of this year. However in light of Cobot 19, we may modestly delay initiation, but we'll still anticipate commencing study 301.

In the second happened this year.

Moving to LG M.D. two week.

As you will recall our goal was to have expression and safety data from our three patient cohort in our high dose arm for SRP nine 003 to treat LG M.D. two week.

In the second quarter.

The team is addressed and overcome Tobin related obstacles and we are indeed on track to evaluate and release that data this quarter given that lad conferences have been canceled we will reflect on the best approach and we will update but again our goal is to release this quarter.

As anticipated.

Douglas S. Ingram: As you recall, our goal was to have expression and safety data from our three-patient cohort in our high-dose arm for SRP9003 to treat LGMD2E in the second quarter. The team has addressed and overcome COVID-related obstacles, and we are indeed on track to evaluate and release that data this quarter. Given that live conferences have been canceled, we will reflect on the best approach, and we will update. But again, our goal is to release this quarter as anticipated. Beyond that, the remainder of our plans is also on course, and manufacturing is progressing.

Beyond that the remainder of our plans are also encores manufacturing is progressing and we've commenced our first commercial GMP run for LG M.D. too we.

And we intend to commence where we hope to be the pivotal trial in 2021 as previously anticipated.

With respect to MPS three a our gene therapy trial with licensing.

That trial has enrolled and dosed 19 of the 20 patients in that trial and is on track to dose all patients by mid year.

As previously indicated.

Now moving onto the or are they platform as you know we are in our rolling submission for our third already therapy cats Amir some intended to treat DMD patients globally mutation amenable to exon 45 skipping.

Douglas S. Ingram: We've commenced our first commercial GMP run for LGMD2E, and we intend to commence what we hope will be the pivotal trial in 2021, as previously anticipated. With respect to MPS 3A, our gene therapy trial with lysogenes, that trial has enrolled and dosed 19 of the 20 patients in that trial and is on track to dose all patients by mid-year, as previously indicated. Now, moving on to the RNA platform, as you know, we are in our rolling submission for our third RNA therapy, Casimirsin, intended to treat DMT patients who have a mutation amenable to exon 45. That submission is proceeding, and it has not been impacted by COVID-19.

That's a mission is proceeding and it has not been impacted by Cobot 19, we should have that submission complete this quarter.

As anticipated.

Our too far in a confirmatory trials and that's mission for excited us.

An essence for by on this and if approved CASM Pearson.

Or chronic therapy trials and largely ex us invest cobot 19 has created more disruption no gene therapy trials, both in terms of Miss visits and some missed doses. However, the team is working diligently to reduce impact.

Sure trial integrity is preserved.

Douglas S. Ingram: We should have that submission complete this quarter as anticipated. There are two RNA confirmatory trials, Mission for Exondus and Essence for Biondis and its approved Cazimiercin, are chronic therapy trials and largely ex-US. And thus COVID-19 has created more disruption than our gene therapy trials, both in terms of missed visits and some missed doses. However, the team is working diligently to reduce the impact, ensure trial integrity is preserved, and that the trials are proceeding.

And that the trials are proceeding.

Next we are in our multi ascending dose trial for SRP 50, 51, our next generation ORADUR technology founded on our peptide conjugated PMO.

Or p. PMO platform for short.

This is a significant program and the goal of our multi ascending dose study is to evaluate whether we are able to safely reach high doses of the p. PMO if were able to safely achieve therapeutic doses with this technology, our preclinical models predicts that the p. PMO could be a potentially pro van.

Douglas S. Ingram: Next, we are in our multi-ascending dose trial for SRP5051, our next generation RNA technology founded on our peptide conjugated PMO, or PPMO Platform for short. This is a significant program, and the goal of our multi-ascending dose study is to evaluate whether we are able to safely reach high doses of the PPMO. If we are able to safely achieve therapeutic doses with this technology, our preclinical models predict that the PPMO could be a potentially profound advance over our current RNA technology, the PMR. It was our intention to provide data release on SRP 5051 by mid-2020. While we still intend to announce those results in 2020, it will likely come in the second half of 2020 for two independent reasons. COVID-19 did interrupt some dosing, which caused a very modest delay.

And advancement over our current army technology the PMO.

It was our intention to provide data release on SRP 50, 51 by mid 2020.

Douglas S. Ingram: But importantly, the team quickly addressed the obstacles, allowing infusions to continue. But more significantly still, this is a dose-escalating study. And the timing of readout depends in large measure on the doses achieved. At the inception of this program, we had anticipated that we could achieve robust expression between 6 mg per kg and as high as about 12 mg per kg. However, we have escalated through those doses, and we are already dosing at 20 mg per kick, nearly 100% higher than the top end of original expectations. Reaching higher doses than we anticipated when the study commenced has necessarily resulted in some delay in reading out that study. Although, as you can imagine, it is not the sort of delay with which we are particularly upset.

While we still intend to announce those results in 2020, it will likely come in the second half of 2020 for two independent reasons.

Coven 19 did interrupt some dosing, which caused me very modest delay, but importantly, the team quickly address the obstacles permitting infusions that continue.

But more significantly still this is a dose escalating study.

And the timing of read out depends in large measure on the doses achieved.

At the inception of this program, we had anticipated that we could achieve robust expression between six makes per keurig two as high as about 12 makes per kid.

However, we have escalated through those doses and we're already dosing at 20 makes for kick nearly 100% higher than the top end of original expectations.

Reaching higher doses than we anticipated when the study command has necessarily resulted in some delay in reading out that steady although as you can imagine it is not the sort of delay with with which we are particularly upset again, we still anticipate a readout for our PMO in the second half of this year 2020.

Speaking of our PBM out program you will have seen on April 28, a press release in which we announced that Sarepta and the United States Army Medical Research Institute of infectious disease or you Sam read the lead medical Biologics labs for the Department of Defense have entered into a cooperative research and the.

Well Im an agreement to evaluate our PMO to treat cobot 19.

Douglas S. Ingram: Again, we still anticipate a readout for our PPMO in the second half of this year. Speaking of our PPMO program, you may have seen on April 28th a press release in which we announced that Sarepta and the United States Army Medical Research Institute of Infectious Disease, or USAMRID, the lead medical biologics lab for the Department of the Army, have entered into a cooperative research and development agreement to evaluate our PPMO to treat COVID-19. While we are not currently focused on antivirals, it was a focus of Sarepta in the past, and our RNA platform has shown promise in treating viruses, including coronavirus.

Well, we're not currently focused on anti viral it was a focus of sarepta in the past and there aren't a platform has shown promise in treating viruses, including Corona viruses.

Head of December it has a detailed knowledge of our PMO technology and on that basis reached out to us to propose working until that therapies.

Informed by each damage knowledge of the Sars tobe to virus and potential hot spots that might be targeted even before the room. It was executed we built a number of therapeutic candidates based on our ppm O platform and had been manufactured insufficient supply.

To be evaluated.

We have already transfer them to do stammer, it which will be responsible for testing in evaluating them and their proprietary in vitro models to determine their potential.

Douglas S. Ingram: The head of Zustamari has detailed knowledge of our PMO technology and, on that basis, reached out to us to propose working on COVID therapy, informed by USAMRIID's knowledge of the SARS-CoV-2 virus and potential hotspots that might be targeted. Even before the agreement was executed, we built a number of therapeutic candidates based on our PPMO platform and had them manufactured in sufficient supply to be evaluated. We have already transferred them to Samarit, which will be responsible for testing and evaluating them in their proprietary in vitro models to determine their potential for reducing viral replication. If one or more of our candidates shows promise, Nusamred and Sarepta will discuss a plan to move forward. Sarepta is a mission-driven organization dedicated to using our science to bring a longer, richer, more liberated life to those living with, and far too often dying from, rare genetic diseases. Diseases like Duchenne muscular dystrophy and LGMD and the like.

In reducing viral replication.

If one or more of our candidates shows promise you Sam or did Sarepta will discuss the plan.

Move forward.

So when Theres, a mission driven organization dedicated to using our science to bring a longer richer more liberated life to those living with and far too often dying from rare genetic diseases. These are like duchenne muscular dystrophy, an L.D. and the like.

It has been critically important to us that we do not find ourselves thrown up mission by this current prices.

And that our patients do not suffer delay in our programs through the fullest extent that we can avoid that and I'm proud to say that we had been able to largely fulfill that goal.

Nevertheless, we are in a crisis and like others. We had technology that may benefit society in this spike.

So when crisis came we answered the call and through this cooperative agreement wouldn't standard and I've been able to employ our technology rapidly and to do so without distracting us or taking substantial resources away from our main mission.

Douglas S. Ingram: It has been critically important to us that we do not find ourselves thrown off mission by this current crisis and that our patients do not suffer delay in our programs to the fullest extent that we can avoid it. And I am proud to say that we have been able to largely fulfill that goal. Nevertheless, we are in a crisis, and like others, we have technology that may benefit society in this fight. So when the call came, we answered the call, and through this cooperative agreement with USAMRIID, have been able to employ our technology rapidly and do so without distracting us or taking substantial resources away from our main mission. Moving to infrastructure and talent, things are going quite well. Our employees are removing obstacles and staying on mission.

Moving to infrastructure and talent things are going quite well our employees, a removing obstacles and staying on mission with our strong cash position and revenue stream, we're able to focus on executing our plans and hitting our milestones with only limited delay.

All of our facilities are operational including our technical operations and CMC related facilities are gene therapy Center of excellence in Ohio, and our gene editing Innovation Center in Durham, North Carolina, where we are already in our facility and hiring.

As chip of Dr., Charlie Gerspach of Duke University I.

Douglas S. Ingram: With our strong cash position and revenue stream, we are able to focus on executing our plans and hitting our milestones with only limited delay. All of our facilities are operational, including our technical operations and CMC-related facilities, our Gene Therapy Center of Excellence in Ohio, and our Gene Editing Innovation Center in Durham, North Carolina, where we are already in our facility and hiring scientists all under the leadership of Dr. Charlie Gerspach of Duke University. I would like now to give a big thanks to our dedicated facility-dependent workers who have been coming into the facilities and laboratories during this difficult time to ensure that experiments and other facility-dependent activities proceed without delay. In summary, I do apologize that this discussion has been dominated by references to COVID-19.

I would like not to give a big thanks to our dedicated facility dependent workers, who have been coming into the facilities that laboratories. During this difficult time.

To ensure that experiments and other facility independent activities proceed without delay.

In summary.

I do apologize that this discussion has been dominated with references the cobot 19.

But it is indeed, a crisis and it is a crisis there must be taken seriously.

No rational person desire the crisis and certainly not a crisis like cobot 19, which has caused so much here suffering a loss of life.

But the one thing that clarify and product prices, but it does the test our metal our resourcefulness, our creativity, our optimism and our commitment.

We often learned far more about ourselves in time of crisis been in times of the.

And in that regard, we all have much about which to be proud first we should all be proud of this biotechnology industry in times of crisis. This industry is answered the call with energy and passion and investment building diagnostics quickly developing therapies working on vaccines consider the great work of the.

Douglas S. Ingram: But it is indeed a crisis, and it is a crisis that must be taken seriously. No rational person desires a crisis, and certainly not a crisis like COVID-19, which has caused so much fear, suffering, and loss of life. But the one thing that is clarifying about a crisis is that it does indeed test our mettle, our resourcefulness, our creativity, our optimism, and our commitment. We often learn far more about ourselves in times of crisis than in times of peace.

Douglas S. Ingram: And in that regard, we all have much about which to be proud. First, we should all be proud of this biotechnology in the... In times of crisis, this industry has answered the call with energy and passion and investment, building diagnostics, quickly developing therapies, working on vaccines. Consider the great work of the many companies that have joined this fight.

Douglas S. Ingram: And I am proud that Sarepta, with our proprietary RNA technology, is playing a role in fighting this disease, even as we remain razor focused on advancing our rare disease mission and serving our patients. This COVID-19 may seem fierce to some, but it is by no means invincible, and it is no match for biotech innovation. Armed with science and commitment, our industry will defeat this pernicious disease. And second, I am particularly proud of my Sarepta team.

Many companies that have joined the this fight.

I am proud that sarepta without proprietary aren't any technology is playing a role and play this disease.

Even as we remain razor focused on advancing our rate rare disease mission and serving our patients.

The scope of 19 may seem fear system, but it is by no means invincible and it is no match for biotechnology innovation armed with the science and commitment our industry will defeat this pernicious disease.

And second I am, particularly proud of my Sarepta team.

We have spoken about the importance of our mission often wasn't driven by a commitment to develop therapies with a pace that allows us to intervene in time to save lives. The diseases, we fight or unrelenting. They do not take time off for this crisis and so our Sarepta family is unrelenting, we have not taking time off for the.

Crisis. When this crisis came this team answered the call adopted and kept executed and like so many people today. They did all of this while dealing with new challenges, new working environment, having to juggle work and childcare and loved one concerns and concerns for themselves and because of their commitments Raptor.

Douglas S. Ingram: We have spoken about the importance of our mission often, one driven by an commitment to develop therapies at a pace that allows us to intervene in time to save lives. The diseases we fight are unrelenting. They do not take time off during this crisis, and so our Sarepta family is unrelenting.

He is on mission on strategy and our programs have been largely unaffected by this crisis. So to all of the dedicated Sarepta workers, who spend their days focused on moving our goals forward, while protecting the patients that we serve I want to say thank you.

Douglas S. Ingram: We have not taken time off for this crisis. When this crisis came, this team answered the call, adapted, and kept excelling. And like so many people today, they did all of this while dealing with new challenges, new working environments, having to juggle work and child care, loved one concerns, and concerns for themselves.

Douglas S. Ingram: And because of their commitments, Sarepta remains on mission, on strategy, and our programs have been largely unaffected by this crisis. So to all of the dedicated Sarepta workers who spend their days focused on moving our goals forward while protecting the patients that we serve, I want to say thank you, and I couldn't be prouder. And with that, I'll turn the call over to Bo.

And I couldn't be prouder.

And with that I'll turn the call over tomorrow.

Well.

Thank you Doug good afternoon, everyone.

Despite the headwinds facing our health care system due to the cobot 19 pandemic.

Im pleased to report that our product revenue for the first quarter of 2020 totaled 100.4 million.

Our experienced team since Sarepta are actively work you to navigate through the challenges of the cobot 19 pending.

And allow us to mitigate major treatment disruptions for patients taking EXONDYS 51, whereby on just 53.

We will continue to navigate the environment and our learning more each day on how to better serve our patients in this unprecedented time.

Bo Cumbo: Thank you, Doug. Good afternoon, everyone. Despite the headwinds facing our health care system due to the COVID-19 pandemic, I am pleased to report that our product revenue for the first quarter of 2020 totaled $100.4 million. Our experienced teams at Sarepta are actively working to navigate through the challenges of the COVID-19 pandemic and allow us to mitigate major treatment disruptions for patients taking Exondys 51 or Biondys 53. We will continue to adapt to the environment and are learning more each day about how to better serve our patients in this unprecedented time. We continue to work closely with our manufacturers, distributors, and specialty pharmacies to provide an uninterrupted supply of our therapies. As a result, we've had no disruptions in supplying Exondys 51 or Biondys 53 to patients.

We continue to work closely with our manufacturers distributors and specialty pharmacies to provide an uninterrupted supply of our therapy.

As a result, we've had no disruptions in supplying EXONDYS 51 or buy on this 53 to patients.

Consistency of supply is key.

Therefore, we will continue our efforts to ensure that EXONDYS 51, and bound is 53 are supplied to patients throughout the cobot 19 pandemic.

We have modified our commercial execution strategy in response to the strain to covert 19 pandemic has placed on healthcare workers hospitals and distribution channels.

Due to recent shutdowns of restrictions at hospitals and clinics. Our team is working closely with health care providers and specialty pharmacies to transition patients to weekly home infusions.

Bo Cumbo: Consistency of supply is key. Therefore, we will continue our efforts to ensure that Exondys 51 and Bionys 53 are supplied to patients throughout the COVID-19 pandemic. We have modified our commercial execution strategy in response to the strain the COVID-19 pandemic has placed on health care workers, hospitals, and distribution channels. Due to recent shutdowns and restrictions at hospitals and clinics, our team is working closely with health care providers and specialty pharmacies to transition patients to weekly home infusions. Fortunately, the vast majority of patients on Exondys 51 are already receiving home infusions.

Fortunately the vast majority of patients on EXONDYS 51 already receiving home infusions.

Patient safety remains our top priority and since many of our patients are choosing not to delay or stop therapy. We have thoughtfully deployed measures to minimize the risk of Coca 19 for all our patients and we'll continue to assess these efforts.

We're working towards initiation patients on volumes 53. However, this environment is challenging because physicians typically want to monitor patients in the clinic for the first couple of infusions and many patients are having difficulty maintaining gregory scheduled appointments with health care providers.

Bo Cumbo: Patient safety remains our top priority, and since many of our patients are choosing not to delay or stop therapy, we have thoughtfully deployed measures to minimize the risk of COVID-19 for all our patients and will continue to assess these efforts. We are working towards initiating patients on Vionis 53. However, this environment is challenging because physicians typically want to monitor patients in the clinic for the first couple of infusions, and many patients are having difficulty maintaining regularly scheduled appointments with their healthcare providers.

We will continue exploring options for patients to safely initiate treatment with bio is 53.

We are still engaging with key opinion leaders and other health care providers on a weekly basis. Additionally, we were having ongoing conversations with payers about the need for patients to start and stay on therapy, regardless of the abuse ambulation status HR gender.

While many of our face to face meetings have been placed on hold the strong relationships. We've established with our partners over the years have helped us transition from in person interactions to virtual engagements.

Bo Cumbo: We will continue exploring options for patients to safely initiate treatment with Biohazard 53. We are still engaging with key opinion leaders and other health care providers on a weekly basis. Additionally, we are having ongoing conversations with payers about the need for patients to start and stay on therapy regardless of ambulation status, age, or gender. While many of our face-to-face meetings have been placed on hold, the strong relationships we've established with our partners over the years have helped us transition from in-person interactions to virtual engagement, to help minimize access and reimbursement barriers. We continue to work with commercial and state Medicaid plans on reauthorization.

To help minimize access and reimbursement barriers, we continue to work with commercial estate Medicaid plans on Reauthorizations. So that patients are able to stay on therapy.

We are encouraged by the efforts payers of me.

To not disrupt patients treatment plans during this difficult time.

Transitioning to our performance for the first quarter.

Many bound biotechnology companies, often face headwinds related to typical health plan enrollment cycles that impact revenue.

The team has been able to successfully navigate these challenges and maintain patients on EXONDYS 51 without significant disruptions.

Bo Cumbo: So that patients are able to stay on therapy. We are encouraged by the efforts payers have made to do not disrupt patients' treatment plans during this difficult time. Transitioning to our performance for the first quarter.

In the current environment, the dynamics of initiating treatment with EXONDYS 51, or by almost 53 are affected.

While mini clinics are closed or Nazi patients for normal in person appointments impact has resulted in fewer patients initiating treatment.

Bo Cumbo: Many biotechnology companies often face headwinds related to typical health plan enrollment cycles that impact revenue. The team has been able to successfully navigate these challenges and maintain patience on Exondus 51 without significant disruption. In the current environment, the dynamics of initiating treatment with Exondys 51 or Vyondys 53 are effective. However, while many clinics are closed or not seeing patients for normal in-person appointments, the impact has resulted in fewer patients initiating treatment.

However, we do anticipate this will change as restriction fees and clinics resumed normal operations.

As a reminder, bound is 53 years old nurse and receipt accelerated approval from U.S. After <unk> on December 12 2019.

Sounds 53 treats patients with Duchenne muscular dystrophy, who are amenable to exon 53 skipping.

We anticipate that patient demographics for Validus 53, we will be similar to EXONDYS 51, with regards to the average age of patients on therapy, and the mix of commercial versus Medicaid patients.

Bo Cumbo: However, we do anticipate this will change as restrictions ease and clinics resume normal operations. As a reminder, BioNTech's 53-year-old Gola Dursun received accelerated approval from the U.S. FDA on December 12, 2019. Zions 53 treats patients with Duchenne Muscular Dystrophy who are amenable to Exxon 53 Skippy.

Over the past three and half years, we've continuously reviewed and refined our approach for EXONDYS 51.

While we're leveraging our deep knowledge and expertise from that launch we will continue to monitor the impact that Kobin Nike pandemic has on the Don is 53 launch trajectory.

We feel confident that overtime patients will ultimately receive access and reimbursement for by almost 53 instart therapy in a timely manner.

Bo Cumbo: We anticipate that patient demographics for Valiantis 53 will be similar to Exondis 51 with regard to the average age of patients on therapy and the mix of commercial versus Medicaid patients. Over the past three and a half years, we've continuously reviewed and refined our approach for Exondus 51. While we're leveraging our deep knowledge and expertise from that launch, we will continue to monitor the impact that the COVID-19 pandemic has on the Beyond This 53 launch trajectory. We feel confident that over time, patients will ultimately receive access to and reimbursement for Biontis 53 and start therapy in a timely manner. Launching a new rare disease drug is already a complex undertaking, and we're very proud of the accomplishments the team has made to date, particularly in light of the extraordinary circumstances.

Launching a new rare disease drug is already a complex undertaking and we're very proud of the accomplishments. The team has made to date, particularly in light of the extraordinary circumstances.

The knowledge, we gather strengthens our plans for future launches.

Including CASM Berson in 2021.

The depth of experience on her teams has helped us navigate through this unprecedented time and we feel confident that the lessons learned will make us a stronger company better able to serve our patients and deliver on our mission as a global leader precision genetic medicine.

And with that I'll turn the call over to Sandy Sandy.

Thanks Bill.

Good afternoon, everyone.

In the first quarter Coker revenues inline with expectations and following the close of the agreements with Roche and the sale of the project review voucher that we received in conjunction with the approval of bond is 53, wearing a strong financial position with significant capital to fund our pipeline and ramp up manufacturing, while maintaining our overall timelines.

Bo Cumbo: The knowledge we gather strengthens our plans for future launches, including Kazim Bersin in 2021. The depth of experience on their teams has helped us navigate through this unprecedented time. And we feel confident that the lessons learned will make us a stronger company, better able to serve our patients and deliver on our mission as a global leader in precision genetic medicine.

In addition, we now have the ability to access roche's significant expertise and greatly enhance our global opportunity for SRP nine 001.

Sandy Mahatme: Thanks, Paul. Good afternoon, everyone. In the first quarter, total revenue is in line with expectations, and following the close of the agreement with Roche and the sale of the Priority Review Voucher that we received in conjunction with the approval of BionBase 53, we are in a strong financial position with significant capital to fund our pipeline and ramp up manufacturing while maintaining our overall timeline. In addition, we now have the ability to access Roche's significant expertise and greatly enhance our global opportunity for SRP 9001. Moving to the financials, this afternoon's press release provided details for the first quarter of 2020 on a non-gap basis as well as on a gap basis. The press release is available on Sarepta's website. Please refer to our press release for full reconciliation of GAAP to non-GAAP.

Sandy Mahatme: Net product revenue for the first quarter of 2020 from our products Exondys 51 and Vyondys 53 was $100.4 million, compared to $87 million for the same period of 2019. The increase primarily reflects higher demand for our products. In the quarter ended March 31, 2020, we recognized $13.2 million in collaboration revenue, which relates to our collaboration agreement with Rove. In February 2020, we received an aggregate of approximately $1.2 billion in cash consideration from Roche, consisting of an upfront payment and an equity investment in Sarepta. From an accounting perspective, $342.7 million is being recognized in revenue on a straight-line basis over the performance period, which we estimate to be through the fourth quarter of 2023. However, this revenue has been excluded on a non-gap basis per Sarepta's policy.

Moving to the financials. This afternoon's press release provided details for the first quarter 2020 on a non-GAAP based basis as well as I've got basis. The press releases are available Sareptas website.

Please refer to our press release for for reconciliation of GAAP to non-GAAP.

Net product revenue for the first quarter of 2020 from our products EXONDYS 51, and wireless 53 was 100.4 million compared to $87 million for the same period of 2019.

The increase primarily reflects higher demand for our products.

Sandy Mahatme: For the quarter, co-development costs under the Roche Agreement total $16.4 million and are included as a reduction to R&D expenses. On a gap basis, we reported a net loss of $17.5 million and $76.6 million, or $0.23 per share and $1.07 per share, for the first quarter of 2020 and 2019, respectively. We reported a non-GAAP net loss of $79.8 million, or $1.04 per share, in the first quarter of 2020, compared to a non-GAAP net loss of $53.8 million, or $0.75 per share, in the first quarter of 2019. In the first quarter of 2020, we recorded approximately $12.6 million in cost of sales, compared to $12.1 million in the same period of 2019.

In the quarter ended March 31, 2020, we recognized 13.2 million of collaboration revenue.

Which relates to our collaboration agreement with Roche.

In February 2020 received an aggregate of approximately $1.2 billion in cash consideration from Roche consisting of an upfront payment and an equity investment in sarepta.

From an accounting perspective, 342.7 million is being recognized in revenue on a straight line basis over the performance period, which we estimate to be through the fourth quarter of 2023.

This revenue has been excluded on a non-GAAP basis course RFS policy.

For the quarter co development costs under the Roche agreement totaled 16.4 million and are included as a reduction R&D expenses.

On a GAAP basis, we reported a net loss of 17.5 million and 76.6 million or 23 cents per share and $1.70 cents per share for the first quarter of 2020 and 29 team respectively.

We reported a non-GAAP net loss of 79.8 million or dollar and four cents per share in the first quarter of 2020 compared to non-GAAP net loss of 53.8 million or 75 cents per share in the fourth quarter of 2019.

In the first quarter Twentytwenty be recorded approximately 12.6 million in cost of sales compared to 12.1 million in the same peered screening 19.

Increase was due to royalties.

Page two Biomarin pharmaceuticals, and you know sort of western Australia, as well as higher product costs as result of increasing demand for our products.

This was partially offset by write offs of certain batches of EXONDYS 51, but did not meet our quality specifications for the first three months last year. There's no similar activity for the first three months that ended March 31, 2020 are you this year.

On a GAAP basis, we recorded a 136.1 million and 90.6 million in R&D expenses for the first quarter of 2020, and 2019, respectively, which is a year over year increased 45.5 million.

Sandy Mahatme: The increase was due to royalties paid to Biomarin Pharmaceuticals and the University of Western Australia, as well as higher product costs as a result of increasing demand for our product. This was partially offset by write-offs of certain batches of Exandus 51 that did not meet our quality specifications for the first three months of last year. There will be no similar activity for the first three months that ended March 31, 2020, i.e., this year.

This increase is primarily related to $43.3 million increase in clinical and manufacturing expenses.

On a non-GAAP basis, R&D expenses were 114.2 million for the first quarter of 2020 compared to 81.4 million for the same period screening 19, an increase of 32.8 million.

Sandy Mahatme: On a gap basis, we recorded $136.1 million and $90.6 million in R&D expenses for the first quarter of 2020 and 2019, respectively, which is a year-over-year increase of $45.5 million. This increase is primarily related to a $43.3 million increase in clinical and manufacturing expenses. On a non-GAAP basis, R&D expenses were $114.2 million for the first quarter of 2020, compared to $81.4 million for the same period of 2019, an increase of $32.8 million. The year-over-year growth in non-GAAP R&D expenses is driven primarily due to a continuing ramp-up of our microdistributing program, as well as our SNS program. Turning to SG&A, on a gap basis, we recorded $82.8 million and $60.6 million of expenses for the first quarter of 2020 and 2019, respectively, a year-over-year increase of $22.2 million. On a non-gap basis, IGN expenses were $54.5 million for the first quarter of 2020 compared to $47.8 million for the same period of 2019, an increase of $6.7 million.

The year over year growth in non-GAAP R&D expenses.

When parameter you due to a continuing ramp up off our micro dystrophin program as well as our essence program.

Turning to as Jimmy on a GAAP basis, we recorded 82.8 million and 60.6 million if expenses for the first quarter of 20, 2020, and 2019, respectively, a year over year increase of 22.2 million.

On a non-GAAP basis via human expenses were 54, and a half million for the first quarter 2020.

Compared to 47.8 million for the same period of 2019, an increase of 6.7 million.

The year over year increase was driven by significant organizational growth and expansion.

Supported our commercial launch plans as well as 40 therapies in various stages of development across several therapeutic modalities.

On a GAAP basis, we recorded $7.4 million expenses net for the first quarter of 2020 compared to 0.2 million other expenses net for the same period of 2019.

The unfavorable change primarily reflects the interest expense on our debt facility entered into in December of 2019.

Sandy Mahatme: The year-over-year increase was driven by significant organizational growth and expansion that supported our commercial launch plans, as well as 4D therapies in various stages of development across several therapeutic modalities. On a gap basis, we recorded $7.4 million in expenses, net for the first quarter of 2020, compared to $0.2 million in other expenses, net for the same period of 2019. The unfavorable change primarily reflects the interest expense on our debt facility entered into in December of 2019. In February of 2020, we entered into an agreement to fill the rare pediatric disease priority review voucher that we received from the FDA in conjunction with the approval of Bondage 53. In March of 2020, we completed our sale of the PRV and received proceeds of $108.1 million, net of commissions, which was recorded as a gain from the sale of the PRV as it did not have any carrying value at the time of the sale. There was no similar activity during the three months ended March 31, 2019. We had approximately $2.2 billion in cash, cash equivalents, and investments as of March 31, 2020.

In February of Twentytwenty, we entered into an agreement to sell the rare pediatric disease. Prior due to reroute sure, but you see from the FDA in conjunction with the approval of bond is 53.

In March of Twentytwenty, you completed our sale of the PRB and receive cross proceeds from 108.1 million net of commissions, which was recorded as a gain from the saves the PRB as it did not have any carrying value of the time of the sale.

There was no similar activity during the three months ended March 31 2019.

We had approximately $2.2 billion in cash cash equivalents and investments as of March 31 2020.

With that I'd like to turn the call over the Gilmore for an update on a research and development activities getting more.

Thank you Sandy and good afternoon.

I would guide that is deeper into the top R&D related activities that Doug as highlighted with particular emphasis on the actions. The team is taking to minimize and where possible eliminates the distractions and obstacles caused by covert 19.

First our SRP nines user one micro disposal program.

Sandy Mahatme: With that, I'd like to turn the call over to Gilmour for an update on our research and development activities. Thank you, Sandy, and good afternoon. I will dive a little deeper into the top R&D activities that Doug highlighted, with particular emphasis on the actions the team is taking to minimize and, where possible, eliminate the distractions and obstacles caused by COVID-19. First, our SRP 9001 micro dystrophy program. We look forward to the imminent publication of the one-year safety and functional data from the four clinical trial participants who received mycotestosterone in Study 101. As with any one-time therapy, we know you are interested in the durability of the functional response in these patients. As Doug mentioned, Study 102 is in very good shape.

We look forward to the imminent publication of the one year safety and functional data from the four clinical trial participants who received Michael just strokes and its study one zero loan.

Gilmour O'Neill: We have dosed all patients in Part A, which is the randomized, double-blind, placebo-controlled portion of the 102 study. Notwithstanding some of the constraints arising from restrictions on patient visits to the sites, we have been able to continue monitoring patient safety and data quality. While some clinical evaluations will take place, quote, outside of the protocol-defined window, unquote, these are not critical outcomes, and we are already evaluating how to mitigate the impact of out-of-window assessments in our analysis and regulatory plan. We are also pleased to see that Nationwide Children's Hospital is loosening COVID-19 restrictions and starting to resume clinical trial activities. And I should note that our second Study 102 site, UCLA, with Harry Shales, Investigator, has not imposed restrictions. So, Study 102 is proceeding, and we anticipate no delay in the readout of that study.

As with any onetime therapy. We know you are interested into Jordan, finishing up the function of response in these patients.

As Doug mentioned study, one or two isn't very good shape.

We have dosed all patients and party, which is the randomized double blind placebo controlled portion of the one or two study.

Notwithstanding some of the constraints arising from restrictions of patient visits to the sites, we have been able to continue monitoring patient safety and stage quality.

While some clinical evaluations will take place close outside of the protocol defined into unquote. These are not critical outcomes and we are already evaluating how to mitigate the impact of out of window assessments in our analysis and regulatory plans.

We're also pleased to see that nationwide children's hospital is loosely covert 19 restrictions and starting to assume clinical trial activity as I should note that our second study window to site you Sealy with heavy chaise investigator has nofs impose restrictions.

So study one or two is proceeding and we anticipate no delay in the read out of that study.

As far as our study three 1% scope I am pleased to report that we have adopted rapidly to cope with 19 related uncertainties of the next few months.

We are maintaining close contact with all of our sites around the world.

Investigators remained very excited about program and want to start as soon as possible.

Nevertheless, we must acknowledge that uncertainty hoppers over the readiness of individual countries as hospitals.

To remove travel and visit restrictions.

Gilmour O'Neill: As far as our Study 301 plan... I am pleased to report that we have adapted rapidly to the COVID-19 related uncertainties of the next few months. We are maintaining close contact with all of our sites around the world. Investigators remain very excited about the program and want to start it as soon as possible. Nevertheless, we must acknowledge that uncertainty hovers over the readiness of individual countries and hospitals to remove travel and visit restrictions. Bandwidth of Ethics Review Boards and Regulatory Agencies to engage in reviews of new protocols may be affected as they deal with a large volume of requests from multiple sponsors in multiple therapeutic areas to amend current studies impacted by the COVID-19 pandemic.

Further.

The bank with Ethics review boards and regulatory agencies to engaging reviews of new protocols may be effective as they deal with a large volume of request from multiple sponsors in most of therapeutic areas to a man's current studies impacted by the covert 19 pandemic.

Fortunately global footprint of our clinical sites gives us significant flexibility to enable dosing this year.

Doug has told you about the status of our ongoing study of SRP nine 003 engine in garden muscular dystrophy tight to eat through.

To remind you of the study design, we are comparing the safety as expression data of a low dose arm of five because the 13 BG per kilogram to dose arm, a four times that dose.

We plan on releasing 48 weeks functional data from the low dose cohorts and especially from the high dose cohorts in this second quarter.

Gilmour O'Neill: The global footprint of our clinical sites gives us significant flexibility to enable dosing this year. Doug has told you about the status of our ongoing study of SRP 9003 in limb-girdle muscular dystrophy type 2E. To remind you of the study design, we are comparing the safety and expression data of a low dose arm of five to the 13 VG per kilogram dose arm of four times that. We plan on releasing 48-week functional data from the low-dose cohort and expression data from the high-dose cohort in this second quarter. After a final safety review, we will make a formal dose selection decision in the third quarter.

Gilmour O'Neill: These results will not only inform the development path for the 2E program but should also inform the dose selection and accelerate the development pathway for our other SARS-CoV-2 programs. We also believe that this data will have some read-through to our microdystrophin study because the programs share the same vector and promoter and, if you recall, are both dosed at high dose equivalents of 2 to the 14th genomes per kilogram. We will also gain experience with this dose in an older and larger patient population, as the three patients in the high dose arm of 9003 are older and larger than the four to seven-year age range of boys included in the micro dystrophin study. Now moving on to our PPMOD platform and more specifically our SRP 5051 program.

After a final safety review, we will make a form of dose selection decision indeed third quarter.

These results were not only informed to develop paths to the to eat program, but should also informed dose selection and accelerate the development pathway for our other sectors like and programs.

We also believes that this data would have some read through to our micro dystrophin study because the programs share the same factor at promoter and if you recall are both dosed at high dose equivalents of two to the 14th favorite Gtms per kilogram.

We will also gain experience with this dose in an older and larger patient population.

As to treat patients at the high dose arm up nine years, you at three our older and larger than before to set your age range of Boise suitors in the micro to strokes and study.

Now moving onto our people a password and more specifically our SRP 50 61 program.

I am very excited about this program because it was potential to approve upon the ability of PMO to increase dystrophin expression by fusing a sell penetrant peptides to the PMO to enhance interest Senator and introduce your dealers.

We have made a lot of progress over the past year and true modifications that the original development plan, but cheap single dosing of trends you make for key in healthy human volunteers and are already achieving multiple dosing of 20 milligrams per kilogram in boys with Duchenne.

Gilmour O'Neill: I am very excited about this program because of its potential to improve upon the ability of PMO to increase dystrophin expression by fusing a cell penetrant peptide to the PMO to enhance intracellular and intranuclear DNA. We have made a lot of progress over the past... and through modifications of the original development plan, achieved single doses of 20 mg per kg in healthy human volunteers and are already achieving multiple dosings of 20 If the 20 mg per kg or 20 mg per kg dose continues to be safe and well tolerated, we plan to continue to dose escalate to 30 mg per kg and then potentially even to 40 mg. To remind you, these doses are significantly higher than expectations at the commencement of this program. Preliminary biomarker data from our Healthy Human Volunteer Study support our hypothesis for enhanced potency of PPMO compared to PMO.

If the 20 milligram per kilogram or trends unique for keep dose continues to be safe and well toleration. We plan to continue to dose escalate to 30. It makes for cake and then potentially even just 14 expertise.

Remind you these doses are significantly higher than expectations at the commencement of this program.

Preliminary biomarker data from our healthy human volunteer study support our hypothesis for enhanced potency PMO compared to people.

In becoming bugs, we plan on analyzing the 12 week biopsies.

From the extra P 50, 51% unique for key cohort.

We will measure exon skipping by digital drop PCR, allowing us to directly compare the efficacy of our PMO and people candidates.

And we will find an appropriate for him this year to provide the safety biomarker at other data from the program to date.

If we are able to reach a therapeutic window for SRP 50, 51 days it will read through to our other DMP programs, where we have a total of six people candidates already built that could treat over 50% institution population.

Gilmour O'Neill: In the coming months, we plan on analyzing the 12-week biome from the SRP 5051 20 mg per kg cohort. We will measure exon skipping by digital drop PCR, allowing us to directly compare the efficacy of our PMO and PPMO candidates. And we will find an appropriate forum this year to present the safety, biomarker, and other data from the program to date. If we are able to reach a therapeutic window for SRP 5051, the data will read through to our other DMD programs, where we have a total of 6 PPO candidates already built that could treat over 50% of the Duchenne population. We are also formulating development strategies that could bring the PPMO platform to the rarer Exxon population, which together make up about 35% of the addressable population, and finally, the results of SRP 5051 will also inform the viability of the PPMO platform for new therapeutics. One of our research goals is to identify conditions suitable for treatment with PPMO.

We're also formulation development strategies that could bring to PMO platform to the rare Exxon populations, which together make up about 35% of the addressable population.

And finally.

The results of SRP 50, 51 will also informed the viability of that people can form for new therapeutic areas.

One of our research goals is to identify conditions suitable for treatment with Pizzino. If we have positive data from SRP 50, 51, we would be able to rapidly accelerate the development popular platform, both within DMD and beyond DMD other therapeutic areas.

Further highlighting the potential first Tennessee at that people are platform as you recall, we just announced collaboration with us ambridge to explore therapeutic agents to combat who've at 19.

This collaboration builds on the anti viral therapeutic potential appear most identified and published by the company in the early two thousands.

Gilmour O'Neill: If we have positive data from SRP 5051, we will be able to rapidly accelerate the development of the PPMO platform both within DMD and beyond DMD to other therapeutic areas. Further highlighting the potential versatility of the PPMO platform, as you recall, we just announced collaboration with U.S. Emirates to explore therapeutic agents to combat COVID-19. This collaboration builds on the antiviral therapeutic potential of PMOs identified and published by the company in the early 2000s. That original work found that PMOs demonstrated antiviral activity in in vitro models against coronaviruses like SARS-CoV, which is the cause of SARS. The antiviral effect of PMO derives from its ability to inhibit the viral replication process.

That original work found that Timos had demonstration antiviral activity in India for models against Croda viruses like Sars code, which is the cause of Sars.

Gilmour O'Neill: We do this by duplexing the Transcription Regulatory Sequence. In this new collaboration, U.S. AMRIT will use in vitro assays to evaluate the ability of the PPMO to suppress viral proliferation and spread. This collaboration will enable Sarepta to contribute to the efforts to treat COVID-19 while adding to our understanding of the PPMO platform. We did consider utilizing the PMO, PMOX, and PMO Plus platforms in the research collaboration. However, based on our recent experience with our PPMO platform, we are confident that it was the right approach for this project. We are quite pleased to be able to collaborate with U.S. Emirates in a way that allows us to contribute to the fight against COVID-19 but does not distract us from our R&D priorities. Our mission is to deliver precision genetic medicine. Patients with rare and serious genetic disorders have served as our North Star to guide us in how we are maintaining critical work on 1. RNA and Gene Therapy Discovery Portfolio, 2.

The anti viral effect of PMO derives from its ability to inhibit the fire ratification process.

It does this by Duplexing two specific candidate sequences in the Corona buyers are an eight for example, the transcription regulatory sequels, and thus guarantee inhibiting translation association and downstream suppressing viral replication.

In this new collaboration us on rates would use in vitro assets to evaluate get busy up to TP mode to suppress viral proliferation and spread.

This collaboration will enable sarepta to contribute to the efforts to treat cobot 19, quiet, adding to our understanding of that PMO platform.

We did consider utilizing to PMO PMO ex PMO touched platforms in the research collaboration however, based on our recent experience with our popular platform. We are confident that it was the right approach for this effort.

We're quite pleased to be able to collaboration with US average in a way that allows us to contribute to the fight against a little bit 19, but does not distract us from our R&D priorities.

Our ambition to deliver precision genetic medicines to patients with rare S series genetic disorders has served as our north star.

Guide us in how we are maintaining critical work on one.

R&D and gene therapy discovery portfolios.

Nonclinical toxicology studies needed to support our portfolio and treat the translational biomarker develop and validation and execution that is so vital to our clinical trials.

I too am very crowded by colleagues in R&D and the work Phantom should enable us to deliver on the promise of our therapeutic portfolio that is so critical to the people who are desperately waiting for helped.

Gilmour O'Neill: Non-clinical toxicology studies needed to support our portfolio and 3. The translational biomarker development, validation, and execution that is so vital to our clinical trials. I, too, am very proud of my colleagues in R&D and the work they have done to enable us to deliver on the promise of our therapeutic portfolio that is so critical to the people who are desperately waiting for help. There will be multiple data readouts over the next two quarters that will guide our next step. And with that, I would hand it back to Doug.

It will be multiple data read outs over the next few quarters that with guide our next steps.

And with that I would hand back to Doug Doug.

Thank you Dr. O'neil, let's open the lines now for questions.

Thank you as a reminder to ask a question you any of the press star one on your telephone to it you know your question test the county.

And the interest a timely asset you. Please limit yourself to one question. Please stand by we can have a 10 day roster.

My first question comes from SAPIEN favorite Goldman Sachs. Your line is now open.

Good afternoon. Thanks for taking my question Simplistically Paralytic hurdle and when you pretend that Titus dataset in the second quarter could you just comment on the selection what the options there going forward with regard to trial designs for the Registrational funny.

Douglas S. Ingram: Thank you, Dr. O'Neill. Let's open the lines now for questions. Thank you. As a reminder, to ask a question, you will need to press star one on your telephone. To withdraw your question, press the pound key.

Operator: In the interest of time, we ask that you please limit yourself to one question. Please stand by while we compile the Q&A roster. Our first question comes from Salveen Richter with Goldman Sachs. Your line is now open. Good afternoon.

Sure. Thanks that questions coming so broadly speaking, we're working with the agency on those questions.

And the development pathway, even as we speak so as we heard earlier the good news is that notwithstanding the till the 19 related issues all of the kids, but dosed all of the kids a bad biopsy will look we will provide an update both gone.

Salveen Jaswal Richter: Thanks for taking my question. So with regard to LimbGirdle, when you present this high-dose data set in the second quarter, could you just comment, post-dose selection, on what the options are going forward with regard to trial design for the registrational study? Sure, thanks for that question. So broadly speaking, we're working with the agency on those questions and the development pathway, even as we speak. So, as we heard earlier, the good news is that, notwithstanding the COVID-19-related issues, all of the kids have been dosed, and all of the kids have been biopsied. We will provide an update both on biomarker data, expression data, and safety data this quarter, and then shortly thereafter, we'll make a dose selection that will inform when Google will form the rest of the sarcoglycan doses And then over the course of the rest of this year, we're doing two things. The one thing we're doing is building a manufacturing supply.

Biomarker data expression data and safety data this quarter in them. Shortly thereafter, we'll make a dose selection that will inform lingered on to it will form the rest of the cycle like can dosing as well and then over the course of the rest of this year. We're doing two things. One thing were doing is building manufacturing supply you may occur.

Heard during my opening remarks that we actually are already in a GMP run for limb girdle to a which I am I would just linger on for a second and give kudos to our technical operations group for being able to get to that point, even during these very disruptive times and then with respect to the development.

The way we're in an ongoing dialogue with the agency on that we'll update toward the end of this year, but our broad view is that we need to find.

Development and regulatory pathway that takes into account the rarity of this the.

Douglas S. Ingram: You may have heard during my opening remarks that we are actually in a GMP run for limb girdle 2E, which I would just linger on for a second and give kudos to our technical operations group for being able to get to that point even during these very disruptive times. And then, with respect to the development pathway, we're in an ongoing dialogue with the agency on that. We'll update you toward the end of this year. But you know, our broad view is that we need to find a development and regulatory pathway that takes into account the rarity of this. This disease so that it is executable, a pathway that is fast and efficient, and one that considers the fact that, with respect to this particular gene therapy, the gene that is being inserted into these children and the protein that's being expressed is the native protein, the absence of which is causing the degeneration and ultimately the demise of patients who have limb girdle 2E, which one might argue should create a very efficient pathway to approval.

Disease.

So that it is executable.

Pathway that is a fast.

And efficient and one that considers the fact that.

With respect this particular gene therapy, the gene that that is being inserted was in these children in the protein that's being expressed isn't the native protein the absence of which is causing the degeneration and ultimately the demise of patients who have limb girdle too, we which one might argue should create a very.

Efficient pathway to approval, but I can give you the specifics of that yet we're in the midst of discussions with the agency over the course of this year by early next year, we should have a few things complete we should have our process development complete as we do GMP material ready and a good understanding with a cheaper and that FDA on the development.

Pathway for limb girdle Julian ended at all a pathway for the rest of 11 rentals and we'll come back and we'll talk about all of that next year. Our goal certainly is to start a pivotal trial in 2021 with respect to limb girdle Julie.

Helpful. Thank God.

Thank you.

Next question comes from.

Fine Abrahams at RBC capital markets.

Hey, guys Heinrich Moller, taking my question.

Hey, Thanks, very much for taking my question on on micro dystrophin.

Regulatory path.

I'm curious your latest thoughts.

As to what you aim to bring to regulators for study three hot potentially support approval I guess I'm wondering if that may change.

Brian Corey Abrahams: Thanks, Doug. Thank you. Our next question comes from Brian Abrahams with RBC Capital Markets. Hey guys, thanks very much for taking my questions.

If it make any changes given what you might be able to collect or what ft might require given the pandemic in the later start and then I guess along those lines have you had any specific feedback from FDA on this out of window data collection from study one or two and how if at all you think that might impact your ability to use it is registered.

Douglas S. Ingram: Thanks very much for taking my question. On microdystrophin regulatory paths, I'm curious about your latest thoughts. I wonder if there might be any changes given what you might be able to collect or what FDA might require given the pandemic and the later start. And then, I guess along those lines, have you had any specific feedback from FDA on this out-of-window data collection from Study 102 and, if at all, you think that might impact your ability to use it as registration enabling without funding? I mean, it's all functional data from the commercial scale material in Study 03, thanks. Yeah, thanks for the questions.

Mission, enabling without full functional data from the commercial scale material and study three thanks.

Yeah. Thanks for the questions. Let me start with the second part of the quite at birth and go to the first so on the second part of the question. We have documented you out of window.

Functional visits as the FDA guidance has suggested so the good news for all advice dealing with this pandemic is that the FDA being very forward thinking and thoughtful about addressing the the disruption associated with drove a 19 in ways that ensure that that we don't.

Shelves with studies that are significantly.

Douglas S. Ingram: Let me start with the second part of the question first. So on the second part of the question, we have documented a few out-of-window functional visits as the FDA's guidance has suggested. So the good news for all of us dealing with this pandemic is that the FDA is being very forward-thinking and thoughtful about addressing the disruption associated with COVID-19 in ways that ensure that we don't find ourselves with studies that are significantly delayed as a result of COVID-19. So I just want to be very clear, you know, in an abundance of transparency, there have been a few instances of patients with out-of-window functional visits in light of the fact that there was a period of time, which is coming to an end now, at Nationwide, where Nationwide Children's Hospital was limiting some of those in-clinic visits.

Delayed as a result and cover 90, so theres already guidance on that would documenting any delays visits in accordance with that the second thing I should note is that there have not been a significant number of the way that you know even independent of that documentation process to ensure that we don't have any technical problems as.

We look carefully at the study there is no reasonably there's an effect on the powering of the study or on the read out around the viability of this study where on the timing of the studies I just want to be very clear you don't have done an abundance of the transparency there and then a few instances the patients without a window functional visits in light of it back.

That there was a period of time, which is coming to an end now at nationwide where nationwide children's hospital was limiting.

Some of those in clinic visit that disappearing, but that will not from our analysis have any impact on the viability of the study of the integrity of the study or the read out or the timing or the probability of success of the study. So I think we're in really good shape with respect to study went out to you right now and honestly I say this and I am giving my own seem a lot of credit I see.

To give credit to two groups I'm, certainly very proud of our clinical operations team in the light for the work they've done to minimize any kind of disruption in study what to do but I should equally give an enormous amount of credit to nationwide children's hospital in their operations team and frankly massive through those the doctor Jerry than Dallas has just been a hero through this process going to study.

Douglas S. Ingram: That's disappearing, but that will not, from our analysis, have any impact on the viability of the study or the integrity of the study or the readout or the timing or the probability of success of the study. So I think we're in really good shape with respect to that. Study 102 right now. And honestly, I say this and I'm giving my own team a lot of credit.

Three or one or the broader group of study study three short answer on that is that we are proceeding right now with the same approach that we had before cobot 19, and we're executing well on this pathway. So study three one would be four to seven year olds it'll be a placebo controlled trial, we're reaching out to studies.

Douglas S. Ingram: I should give credit to two groups. I'm certainly very proud of our clinical operations team and the like for the work they've done to minimize any kind of disruption in Study 102, but I should equally give an enormous amount of credit to Nationwide Children's Hospital and their operations team and, frankly, massive kudos to Dr. Jerry Bendell, who's just been a hero. Going to Study 301, or the broader group of studies, Study 3, the short answer on that is that we are proceeding right now with the same approach that we had before COVID-19, and we're executing along those pathways. So Study 301 will be 4- to 7-year-olds.

Sites, even as we speak I will tell you actually.

The study sites themselves on the whole are not only very enthusiastic about study three one the commercial supply trial study, but actually quite optimistic about their ability to start that study and the started on time.

With notwithstanding that I do want to point out that we have to be realistic that there that that we got it and so some of the destroyed the biggest rate limit or.

Douglas S. Ingram: It'll be a placebo-controlled trial. We're reaching out to study sites even as we speak. I will tell you, actually, the study sites themselves, on the whole, are not only very enthusiastic about Study 301, the commercial supply trial study, but actually quite optimistic about their ability to start that study and to start it on time. Notwithstanding that, I do want to point out that we have to be realistic about where we've got to, and so some of this is choice; the biggest rate limiter up till now has been ensuring that we can get GMP material released on time, and as I've said before in my opening remarks, and I'll repeat again, through the great work of our technical operations group, we are on track right now to have GMP material by July, just as The next issue is a choice.

Uptil now has been ensuring that we can get.

GMP material released on time and as I've said before in my opening remarks, I'll repeat again through the great work of our technical operations group. We are on track right now to have GMP material by July just as we had anticipated work opened 19.

The next issues, it's a choice we want to make sure as were tracking forward that with respect to site there not only ready to start taking patients and then we get through all of those processes in the IB process in real life, but there were very confident that those sites will continue on into the fall and into the winter into 2021 and beyond that that may modest.

We delay us, but as I've said before I really I'm talking about modest delays that still our goal to start the study three one and it's progeny and a second half of 2020 and right now we feel confident that is not going to be an issue.

Douglas S. Ingram: We want to make sure, as we're tracking forward, that with respect to sites, they're not only ready to start taking patients, but we get through all of the various processes and the IRB processes and the like, but then we're very confident that those sites will continue on into the fall and into the winter and into 2021 and beyond. And that may modestly delay us, but as I've said before, I really am talking about modest delays. It's still our goal to start study 301 and its progeny in the second half of 2020. And right now, we feel confident that it's not going to be an issue. Thanks very much, Doug.

Thanks, very much Doug.

Thank you.

Thank you.

Our next question consequences in the month with Bank of America. Your line is Hamilton.

Hi, Good afternoon, guys. Thanks for taking my question I'm sorry. That's question. It's already aspect that can you clarify penny per study went out to.

How often are the patients actually being monitored her at the functional visits and specifically I just wanted to get a sense biopsies that were scheduled to be taken for the patients in study to do they have to take in per protocol that at a certain point of time. After they were dose or is there any flexibility and when the biopsies.

Douglas S. Ingram: Thank you. Thank you. Our next question comes from Tazeen Ahmad with Bank of America. Your line is now open.

Tazeen Ahmad: Hi. Good afternoon, guys. Thanks for taking my questions. I'm sorry if this question was already asked, but Doug, can you clarify for me, for Study 102, how often are the patients actually being monitored for the functional visits? And specifically, I just wanted to get a sense of whether the biopsies that were scheduled to be taken for the patients in Study 2, did they have to be taken per protocol at a certain point in time after they were dosed, or is there any flexibility in when the biopsies can be taken? So I'm going to turn this over to Louise Rodino Clayback to respond to both of those questions. Thanks, Doug. There is a certain visit window for the biopsies, but as Doug mentioned, in terms of flexibility, any out of window needs to be documented as per FDA guidance.

Can be taken.

So I'm going to turn this over actually the two Luis with you know clay back to respond to both of those questions.

Sure. Thanks Stan.

There's a certain visit wind down for the biopsies that as Ted mentioned he turns on a flexibility any out of window that needs to be documented as per the FDA guidance now as they look at it there's no particular.

The delays or Mrs. Within the protocol that will be meaningful activity the outcome of the study.

Okay, and and did you know what percent of patients, where you have a little bit delayed and getting the biopsy.

Louise R. Rodino: So as we look at it, there's no delays or misses within the protocol that will be meaningful to the outcome of the study. Okay, and did you know what percent of patients were, you know, a little bit delayed in getting the biopsy? I'm sorry. For those who may wonder why there was an enormous delay, we are distant from one another, and I actually insisted that I direct the questions. So apologies for that, Louise. So Louise, I'm sorry. Did you answer the question?

Oh I'm sorry.

[laughter], we're discussing this but those are they wonder why there was an enormous delay we're just in for one another and it and I actually insisted that I direct your question apologies to that varies so Luis I'm sorry.

Answer the question.

Yeah, No. There was just I I don't know the exact number.

Yes, it's that were delayed and it's Tim.

Okay, very very small number.

Okay, and then as we as we think about limit or another follow up for the pivotal study that you're going to start next year for that for the Q E kept Graham can you just remind us how big of a population that is relative to the rest about GMP.

Cost of doing around it's this is an ultra rare population I don't think we've we've gone out in a given the exact numbers as one of the Big We've said if you look at the entire.

Douglas S. Ingram: Yeah, no, there was just I don't know the exact number of visits that were delayed, but it's a few. Okay, a very, very small number. Okay, and then as we think about Lim Girdle to follow up for the pivotal study that you're going to start next year for the 2E subgroup, can you just remind us of how big of a population that is relative to the rest of LGMG? Quotes are very rare.

After the neologism population of all of the patients that were looking at we're talking about a group of patients that are about 70% of the size of duchenne muscular dystrophy, but the limb girdle to me is a very rare form of limb girdle and the very severe form limb girdle, so that we haven't Dale.

But nailed the size of the trial down, but obviously with that in mind when you consider the rarity of the disease.

Douglas S. Ingram: This is an ultra-rare population, so I don't think we've gone out and given the exact numbers. As one of the things we've said, if you look at the entire... Epidemiology and Population of all of the patients that we're looking at, we're talking about a group of patients that is about 70% of the size of Duchenne muscular dystrophy. But the limb girdle 2E is a very rare form of limb girdle and a very severe form of limb girdle.

And you consider that this is really it's just kind of it is a perfect sweet spot.

Opportunity for for a gene therapy. This is monogenic disease, well characterized well understood. It is the lack of a structural protein and as a single structural protein that is causing all of the damage in the generation then loss of life that comes with TV and the.

Douglas S. Ingram: So we haven't nailed down the size of the trial yet. But obviously, with that in mind, when you consider the rarity of the disease, and you consider that this is really, this is kind of the perfect sweet spot opportunity for a gene therapy. This is a monogenic disease, well characterized, well understood. It is the lack of a structural protein.

Gene therapy that we that we have created in that loses created.

Well will introduce a gene that codes for the actual native protein that about the lack of which has gone into buying so.

Douglas S. Ingram: It is a single structural protein that is causing all of the damage and degeneration and loss of life that comes with 2E. And the gene therapy that we have created and that Louise has created, we'll introduce a gene that codes for the actual native protein, the lack of which has gone through demise. So again, I don't have an answer yet on the exact development pathway, but we're working on that actively with the agency over the course of 2020. But it is certainly our goal to have a very, have a development pathway, and regulatory pathway that has the speed and leanness that one would imagine with the disease of this rarity, well-characterized monogenic, and with the, if we can get to very high expressions, and we're already there with our current dose, that, you know, returns to these patients the very protein, the lack of which is causing their, their, their. Just Our next question comes from Debjit Chattopadhyay with H.C. Winkler. Your line is now open.

Again, I don't have an answer yet on the exact development pathway. We're working on that actively with the agency over the course of 2020, but it is certainly our goal to have a very have a development pathway regulatory pathway that that has the speed and lean is that one would imagine with the disease of this.

Rarely well characterized monogenic and with that if we can get very high expression and we're already there with our current does well that that that you to return to these patients the very protein the lack of which is causing there there their disease.

Okay. Thank you remind everyone. As a reminder, everyone. One question you never really fall to the lines going to be silenced. After you ask one question. Thanks.

Thank you I'm next question kind of stuff that they try to today, where they see everything in line is now open.

Hey, good afternoon, guys and thanks for taking the questions. So given that you're at the 20 mix looking to us with a healthy 50 51 now what should be the 12, we can dystrophin expression.

That's what excites you to advance to 45 and 53 programs rapidly.

Yeah. Thank you very much of that Debjit look what what I can say broadly about this is bad but first of all 12 weeks is a very early period of time being mean looking for district, and so I wouldn't commit in advance that we will even be looking for district and the 12 weeks, what we will be looking at its exon skipping, but you will find with and of course through remind those I'm sure everyone.

Debjit D. Chattopadhyay: Hey, good afternoon, guys, and thank you for taking the questions. So given that you're at the 20 mg per kick dose with SRP 50-51, what should be the 12-week dystrophin expression that would excite you to advance the 45 and 53 programs rapidly? Yeah, thank you very much for that, Debjit. Look, what I can say broadly about this is that, first of all, 12 weeks is a very short period of time to even be looking for dystrophin.

No this but yes exon skipping is the very activity that results in the truncated format.

Or M&A that in turn creates the the protein that is the slightly truncated, but otherwise fully functional district and that we're trying to achieve and what we know at least in animal. We know two things number one there is an obvious direct correlation between amount of exon skipping as you wouldn't well a man.

Douglas S. Ingram: So I wouldn't commit in advance that we will even be looking for dystrophin at 12 weeks. What we will be looking at is exon skipping. And what you will find within, and of course, to remind those of you, I'm sure everyone knows this, but exon skipping is the very activity that results in the truncated form of the RNA that in turn creates the protein.

Engine and amount of dystrophin production. So looking at exon skipping is a perfect you know a brilliant marker for what kind of dystrophin, we might find over time I'll give you. An example, with respect to go nurse and we had made some fairly bold statements about where we probably would see would go orders versus a tougher center.

Douglas S. Ingram: That is the slightly truncated but otherwise fully functional dystrophin that we're trying to achieve. And what we know, at least in animals, we know two things. We know number one, there is an obvious direct correlation between the amount of exon skipping, as you would well imagine, and the amount of dystrophin production. So looking at exon skipping is a perfect, you know, a brilliant marker for what kind of dystrophin we might find over time. I'll give you an example with respect to golandersin.

That was all because of what we had seen early days in the relative exon skipping. The came from go orders versus the temperature. So I think exon skipping is a great marker and what we're going to be looking for of course that would get US excited is the relative amount of excellent keeping you might see from the PMO.

12 weeks versus the amount of exon skipping that we'll see where the PMO at the doses that were talking about right now.

Douglas S. Ingram: We had made some fairly bold statements about what we thought we would see with golandersin versus teplersin. And that was all because of what we had seen in the early days with the relative exon skipping that came from golandersin versus teplersin. So I think exon skipping is a great marker.

We don't have it yet we'll see it will see at the second half. It will all come together will have data on tissue exposure at exon skipping and safety and alike and dosing levels, but I can at least tell you that in animal models.

We were we would have been getting excited at doses significantly below the doses that were now dosing through and I think you know at the inception of the single ascending dose and then the multi ascending dose study.

When we started this our animal models are you know our mouse model than in our non human primate models out as to point, there, where we would be very happy with six bags, but we would get increasingly happy up to about 12 rigs for King, which is what we envision would be the the feeling and how high we could get and certainly with her.

Douglas S. Ingram: And what we're going to be looking for, of course, that would get us excited is the relative amount of exon skipping you might see from the PMO. [inaudible] We would have been getting excited at doses. Significantly below the doses that we're now dosing through. And I think, you know, at the inception of the single ascending dose and then the multi-ascending dose study, when we started this, our animal models, our, you know, our mouse models and then our non-human primate models got us to a point where we would be very happy with six bigs, but we would get increasingly happy up to about 12 bigs per king, which is what we envisioned would be the ceiling and how high we could get.

Back to this peptide conjugated PMO more is better with respect to dosing, but you get the higher doses safely and the fact that were at 20 makes Burger King right now.

Is makes us very very pleased I will say.

And while it has that affected us or a delay in the program as we have to continue dosing I don't think it's a delay that any rational person when to complain about.

Thank you I'm next question comes from Maria Young with Cantor Fitzgerald Your line is handling.

Douglas S. Ingram: And certainly, with respect to this peptide conjugated PMO, more is better with respect to dosing if we can get to higher doses safely. And the fact that we're at 20 bigs per king right now makes us very, very pleased, I will say, and while it has affected us, a delay in the program as we have to continue dosing, I don't think it's a delay that any rational person would Our next question comes from Alethea Young with Cancer Fitzgerald. Your line is now open. Hi, this is Emma from Aletheia. Can you give us any color on what the actual process and requirements look like for a patient to transition to home infusion and just what the percentage of patients are currently on home infusion for Biondis and Exondis and what those trends might look like next quarter?

Hi, this is on that on infer anything I can you give us any color on what the actual processing requirements look like for patient to transition to home in teaching just what the penetrations are currently ongoing fishing for my honest next I'm asking you know what those trends might look like next quarter.

Sure I'm going to turn this over to but before I turn it over just to remind everyone with respect to axon. This as it stands today. The vast majority of patients are on home infusion and while with respect to launching product line by on this you will see a greater percentage of patients that start in hospital. It will overtime the same.

In case, the vast majority of buyout biologist stations will become home infusion as well and of course, but can talk you about the things that we're doing today to accelerate that process.

But would that yet.

Unknown Executive: Sure, I'm going to turn this over to Bob before I turn it over, just to remind everyone, with respect to Exandus, as it stands today, the vast majority of patients are on home infusion, and while with respect to a new product like Biondus, you will see a greater percentage of patients that start in the hospital, it will, over time, be the same case that the vast majority of Biondus patients will become home infusion as And, of course, Bo can talk to you about the things that we're doing today to accelerate that process. So there is a couple of weeks delay for those patients that do need to transition, but we do expect this to ease over the next couple of months.

Yeah, Thanks, Doug Yeah into Doug's point, though.

The majority or patients already at home infusion, but for the patients there are being dosed in the hospital, it's really just a authorization like a reauthorization process with payors.

So it's just more of a paperwork process of transferring.

The authorization from in hospital infusion to home infusion. So yeah. There is a couple of weeks delay for those patient that do need to transition, but oh, we do expect this to you know ease over the next couple months.

One other thing I won't say on this topic because this is yes, there's a lot of.

Our process involved both in the launch of by on this and softness in one of the things you might have heard in my opening remarks was about the fact that we're watching very carefully how payers are going to react in the middle of this crisis to ensure that private payers don't themselves.

Take advantage of the disruption that's occurring with over 19 in ways that might profit them to the detriment of children in fairness I should note. The payers are based on everything we're seeing right now going exactly the opposite but in fact, I think that very laudably payers have that we're looking for ways to reduce the.

Unknown Executive: One other thing I will say on this topic is that, you know, there's a lot of process involved both in the launch of IONDUS and EXONDUS, and one of the things you might have heard in my opening remarks was about the fact that we're watching very carefully how payers are going to react in the middle of this crisis to ensure that payers don't themselves take advantage of the disruption that's occurring with COVID-19 in ways In fairness, I should note that payers are, based on everything we're seeing right now, doing exactly the opposite. But, in fact, I think that, very laudably, payers have been looking for ways to reduce some of the obstacles that might be imposed as a result of COVID-19 to ensure that kids can stay on therapy. I'll give you just one example.

That some of the obstacles that might be impose as a result that cobot 19 to ensure that kids can stay on therapy I'll give you. Just one example, with respect to state Medicaid I Uninformed every single state all 50 state Medicaid have had requested and have been granted by CMS a waiver.

Version that allow them in turn to waive prior authorization requirements and to extend kids on therapy without the need to go through the prior authorization process. I don't have suggested that means that they're not going to impose prior authorizations, they they likely still well, but certainly that is a significant step.

Douglas S. Ingram: With respect to state Medicades, I am informed that every single state, all 50 state Medicades have requested and have been granted waivers by CMS waivers that allow them, in turn, to waive prior authorization requirements and to extend therapy to kids without the need to go through the prior authorization process. But I don't want to suggest that that means that they're not going to impose prior authorizations. They likely still will, but certainly that is a significant step in the direction of ensuring that COVID-19 doesn't get in the way of kids' access to therapy, and I really do think they deserve kudos for that. Thank you. Our next question comes from Gena Wang with Barclays. Your line is now open.

And the direction of ensuring that killed the 19 doesn't get in the way of kids access to therapy, and I really do think they'd stages or kudos for that.

Thank you.

Next question comes then generally with Barclays. Your line is now open.

Thank you for taking my questions I have to one they got even glendon Bina damn blue and abstract fiber to the uncool one data at the F. G. P. T P S abstract.

Somehow the final website doesn't have leased abstract just want to double check who deal would be new data and the TCP equaling Qiagen and also giving good safety still probably see from micro tissue from program.

Seeking grew proven has the same factor and the promoting.

Gena Wang: Thank you for taking my questions. I have one regarding Lingerto data. There was an abstract 503 on COHO-1 data in the ASGCT PDF abstract, but somehow, the final website doesn't have this abstract anymore.

Fair to say hi is going to lose safety should be largely in line with my condition from program.

Well I'm, the latter part I'm going to say.

That we're going to comment on that when we released the data and we'll have that data released this quarter. So you won't have to wait very long to get an update on limb girdle to anybody expression in safety, but your point well taken so were cleared it's actually you're exactly right, which is this is the same promoter. This is the same vector and the weak what we're currently calling rose.

Douglas S. Ingram: Just want to double check that there will be no data at the ASGCT for Lingerto. And also, given good safety so far we've seen from microdistribution programs, since Lingerto program has the same factor and a promoter, is it fair to say higher dose safety should be largely in line with microdistribution programs? On the latter part, I'm going to say that we're going to comment on that when we release the data, and we'll have that data released this quarter, so you won't have to wait very long to get an update on limb girdle QE, both expression and safety, but your point's well taken, so we're clear that actually you're exactly right, which is this is the same promoter, this is the same vector, and what we're currently calling the prior dose, the high dose on limb girdle, is the same dosing level that the kids with Duchenne Muscular Dystrophy have received, both in 101 and 102, and together, that is a significant number of patients that have been dosed.

The prior to us a high dose or limb girdle is the same dosing level that the kids with Duchenne muscular dystrophy ever see both in what I want to add one or two and together that is a significant number of patients that have been dose would that I'm going to turn it over to lose who might comment on the I guess TCP and like girdle, but maybe.

Maybe give an overview of what what kind of abstracts are posters, we might have it is to see too small.

Sorry, I meant in terms of the clinical data and it will be we won't really that later, the cornerstone well and at CCT there aren't too preclinical abstracts that will be presented a poster at four lingered on that we'll be focused on on long term expression Dan outside of express.

Douglas S. Ingram: With that, I'm going to turn it over to Louise, who might comment on the ASGCT and the limb girdle but maybe give an overview of what kind of abstracts or posters we might have at ASGCT. In terms of the clinical data, we will release it later this quarter. At AHGCT, there are two preclinical abstracts that will be presented as posters for limb-girdle, and they will be focused on long-term expression data and then also expression in later stages of the disease.

And then in later stages and see if not preclinical and then of course, we have our symposium.

I will outline the background on the development of our contracts and I'll talk about same accurate as Jonathan.

Nine month data.

Thank you I'm next question comes from the Tubarao with Cowen. Your line is now open.

Hey, guys to shot on for two question on study one or two I'm wondering with some of the assessments being outside the window have you had to revisit the statistical analysis plan and want to just think about doing that.

Louise R. Rodino: This is all preclinical. And then, of course, we'll have our symposium where we'll outline the background to the development of our constructs and also talk about the microdystrophin nine-month data. Thank you. Our next question comes from Ritu Baral with Cowen. Your line is now open. Hey guys, it's Peshawn Thon on behalf of Ritu.

Weve rig yeah. Thanks walking the question, we've we've looked at it carefully and there's no adjustments necessary. We don't have a we don't have a concern on the stat Statistical analysis plan in broad strokes. You don't have is concerned on powering we don't think theres any impact from the modest number of out of window functional visits to called any concern regarding integrity.

Ritu Baral: A question on study 102. I'm wondering if you have had to revisit the statistical analysis plan, and what adjustments have had to be made? Thanks. Thanks a lot for the question. We've looked at it carefully, and there are no adjustments necessary. We don't have a concern about the statistical analysis plan in broad strokes. We don't have a concern about powering.

And certainly as we've said before it won't ever any effect on timing.

Dr. O'neill, if there's anything I've missed in that feel free to add to that are correct me if I misstated than me.

Douglas S. Ingram: We don't think there's any impact from the modest number of out-of-window functional visits to cause any concern regarding integrity. And certainly, as we've said before, it won't have any effect on timing. Dr. O'Neill, if there's anything I've missed in that, feel free to add to that or correct me if I'm wrong. No corrections necessary at all, Doug. We have looked, and these out-of-window assessments are not absolutely critical and have no impact on our statistical analysis.

No corrections at necessary at told Doug we have looked and these when do assessments are not absolute critical and has no impact on our statistical analysis plan.

So we remain on site.

Thank you I next question kind of San Jacinto Sweats. It SVB, Inc. Your line you're kind of open.

Thanks, very much so based on the age of the patients that you've been Roland study window to how do you expect there north star scores to change over the duration of part and how is the study powered to detect a difference between the two arms there's.

Cookies literature, suggesting that Northstar can increase during this age range. So im just trying to think about what kind of an increase 9001, we'll have to produce in order to succeed.

Douglas S. Ingram: So we remain on time. Thank you. Our next question comes from Joseph Schwartz with SVB Lyrinc. Your line is now open.

Joseph Patrick Schwartz: Thanks very much. So based on the age of the patients that you've enrolled in study 102, how do you expect their North Star scores to change over the duration of Part A? And how is the study power to detect a difference between the two arms?

But let me give you the broad stroke are going to broad stroke answer on that from the nuance I'm, probably going to avoid simply for you know.

Competent Gianni competitor, you're talking to the broadest or the broadest stroke is the power I suspect that no one has better patient level data on duchenne muscular dystrophy. Its epidemiology. Its course, then sarepta obviously the leaders in Duchenne muscular dystrophy, we've been close to the.

Douglas S. Ingram: There Kopi's literature is suggesting that North Star can increase during this age range, so I'm just trying to think about what kind of an increase 9001 will have to produce in order to succeed. But let me give you the broad stroke, I'll give you a broad stroke answer on that from the nuance I'm probably going to avoid simply for, you know, Confidentiality and Competitiveness. But I'm going to do the broadest stroke. The broadest stroke is the following. I suspect that no one has better patient-level data on Duchenne muscular dystrophy, its epidemiology, and its course than Sarepta.

Patient community and the data for E arms, we have that we have not only all of the literature, we have our own data patient level data, we have access to synergy data we have access to all of the data from Biomarin that they had back in the day that we negotiated and so we're well informed to build as the study that we built to.

Point in a nuanced way what you see across four to seven year old. The broad strokes is that that there is the possibility for a couple point potential linear I'm not linear rise apology nonlinear rise the couple of points increase potentially between the four and five year range, but you will see them coming over the top and actually declining.

Douglas S. Ingram: Obviously, the leaders in Duchenne muscular dystrophy have been close to the patient community and the data for eons. We have not only all of the literature; we have our own data, patient-level data. We have access to Synergy data. We have access to all of the data from Biomarin. And we're well informed to build this study that we built. To your point, in a nuanced way, what you see across 4 to 7-year-olds is that there is the possibility for a couple point potential, linear potential. Not linearized, I apologize, non-linearized, a couple of points of increase, potentially between the four and five year range, but you will see them coming over the top and actually declining, starting to significantly decline in the six and seven year old range.

Turning to significantly declined in the five six and seven year old range. As you know these kids are four to seven all of that new wants to understanding of the the the course of this disease that is been used to perform the protocol that we views and beyond just that we of course had the benefit.

First for patients that we've had we've hair cut about some of the some of that data as well, but we've used center. The data that we have gleaned from the first four pieces that help us consider the powering of this study I will note by the way I don't know.

Douglas S. Ingram: As you know, these kids are four to seven; all that nuanced understanding of the course of this disease that has been used to inform the protocol that we've used. We've used some of the data that we have gleaned from the first four patients to help us consider the powering of this study. I will note, by the way, I don't know if we've talked about this, but the one-year publication on the first four kids in that cohort will be published very soon, imminently. Anyways, all that went into this, and just to make a broad stroke point, the powering of this study was over 90% when we got to 40 patients. So we feel confident about the protocol, about the powering, and certainly about the progress of Study 100. Thank you. Our next question comes from Christopher Murai with Nomura. Your line is now open.

I'm talking about this but the the one year publication on that first for kids.

That cohort will be will be published very soon eminently any with all that went into this and just to make the blocks. Your point. The powering of this study was over 90% when we got to 40 patients.

So we feel we feel confident about the protocol about the powering and certainly not the progress study one or two.

Thank you I next question comes from Christopher Am I right written a ma'am your line is now open.

Hey, good afternoon, I'm, just thinking about the path forward for lingered on muscular dystrophy.

Regulators and and the potential for using expression data I was wondering you know.

Just how how much especially the European might be to see there and is FDA has any concerns or if you have any concerns about also I suppose.

Just as hard look like and complex being sort of reformed by re expressing the protein and making sure that intact and if you are prepared and provide any localization data in that regard. It's required and then just real quick with P. CMO I mean, how much data will only satisfy you guys with respect to safety at.

Christopher Murai: Good afternoon. I'm just thinking about the path forward for limb-griddle muscular dystrophy, you know, with regulators, using expression data. I was wondering, you know, how much expression data you might need to see there and if FDA has any concerns or if you have any concerns about The Sarco-glycan complex being sort of reformed by re-expressing the protein and making sure that's intact, and if you are prepared to provide any localization data in that regard if it's required. And then, really quick with PPMO, I mean, how much data will You know, what kind of time frame are we looking at before we can get confident that you can move forward at, say, 20? Thank you. Okay, so on as relates to the limb girdle, you know, we're still working on the development and regulatory pathway itself, as you've anticipated. We certainly think that this should be a very lean approach.

Any given dose call it 20 extricate.

Kind of timeframe are we looking at the floor.

We we can get confident that you can move forward at day 20, either 50. Thank you.

Okay. So on as it relates to limb girdle, you know, we're still working on the development and regulatory pathway itself as Youve anticipated. We you know, we certainly think that there should be a very lean approach and frankly in a perfect world. We think that this is that this would be a perfect.

Opportunity to be approved on a biomarker of expression you raised it really interesting point about you know what about the reconstitution of the dystrophin associated protein complex in the back because we think that has a very important element to the looking at this and that's another important biomarker <unk> is there when.

Douglas S. Ingram: And frankly, in a perfect world, we think that this would be a perfect opportunity to be approved on a biomarker of expression. You raise a really interesting point about, you know, what about the reconstitution of the district and associated protein complex. And the fact is, we think that is a very important element to look at this. And that's another important biomarker. When you upregulate, as an example, beta-sarcoma glycan, do you see that the district and associated protein complex itself also begins to upregulate so that other proteins in that complex would start to come together and be expressed where they would be missing before because the DAPC is not coming together? That is important, and regardless of whether the FDA, on its face, would demand it, we do think that's something important that we should show and be able to prove.

Do you up regulate as an example beta circuit why can do you see that they put the dystrophin associate protein complex itself also begins to up regulate so that other proteins in that complex would start to come together and be express where they would be missing before because the the D.A.P. She is not kinda together.

That is important and that that regardless of whether the FDA on its face with demanded we do think that's something important that we should show and be able to prove and the good news is by the way we have seen that repeatedly that one of the one of the things that should get someone very excited about this is that it's that this is that the native gene the native protein yeah.

You know already at our at our previous Ddos very strong expression properly localized to the right place and feel good point associated with the up regulation does the other proteins that make up the dystrophin associated broke the company is one example, you see a very strong.

Douglas S. Ingram: And the good news is, by the way, we have seen that repeatedly, one of the things that should get someone very excited about this is that this is the native gene, the native protein, you know, already at our previous dose, very strong expression, properly localized to the right place, and, to your good point, associated with the up-regulation of the other proteins that make up the dystrophin-associated protein coupling. This is one example. You see a very strong correlation between up-regulation of beta-sarcoglycan and alpha-sarcoglycan, which these kids have a gene that can properly code for alpha-sarcoglycan, but you don't see it in a significant amount because, in the absence of beta-sarcoglycan, there is no DAPC.

Correlate between Upregulation of beta started to go I can't and Alphatec are episodic a blank in which these kids have a gene that properly code throughout the second weekend, but you don't see it in a significant amount because in the absence of beta sort of like and there is no D.A.P.C. So all I think though all of the facts and circumstances.

Around that they're going to come into play when we think about the development plan. What we think we should be able to avoid would be a lengthy trial that would require you know.

For instance, a placebo controlled trial and the like and we're still working with the agency, we still a lot of discussions to go with the agency the land on the right answer there and what if there are a lot of places to land between an accelerated approval on a biomarker, which would of course be our the answer we think is great and on the other hand, you know a full blown.

Douglas S. Ingram: So, I think all of the facts and circumstances around that are going to come into play when we think about the development plan. What we think we should be able to avoid would be a lengthy trial that would require, for instance, a placebo-controlled trial and the like, and we're still working with the agency. We still have a lot of discussions to go with the agency to land on the right answer. There are a lot of places to land between an accelerated approval for a biomarker, which would, of course, be the answer we think is great, and, on the other hand, a full-blown placebo-controlled trial, but we'll come back early next year and provide an update to everyone.

Placebo controlled trial, but we'll come back early next year and provide an update everyone.

[noise] thinking I'm, sorry quest.

Before we go on because I've been promise that even though there were there with regard to I will turn this over to Dr. O'neill to answer the question on the PMO.

[noise] at so to date, we are actually happy with the safety profile that we're seeing and with the numbers in our cohorts are ascendiant cohorts and about the ascending dose. We believe that we have sufficient numbers I think we're it suggests safety at like looking both at clinical outcomes, but actually are obviously.

Douglas S. Ingram: Thank you. But before we go on, because I didn't promise it, even though there were two questions, I will turn this over to Dr. O'Neill to answer the question about the people. So, to date, we are actually happy with the safety profile that we're seeing, and with the numbers in our cohorts, our ascending cohorts in the multi-ascending dose, we believe that we have sufficient numbers. I think we are able to judge safety by looking both at clinical outcomes, but we are obviously able to monitor the target tox that were identified in non-clinical toxicology.

But to monitor the target talks backwards hdinsight not take on top so we believe with those numbers. We can compete make decisions. Obviously looking forward safety is always a matter of numbers, but these numbers are we'd be adequate for making that initial at dose selection.

Thanks.

Thank you I next question comes down Jodi Regts Citi. Your line is now open.

Hi, This is Sean Egan, calling in for dual thank you for taking my question.

Oh, the CP ammo congratulations on the 20 matrix pick dosing.

Gilmour O'Neill: So, we believe with those numbers, we can confidently make decisions. Obviously, looking forward, safety is always a matter of numbers, but these numbers are, I believe, adequate for making that initial dose selection. Thank you. Our next question comes from Joel Beattie with Citi. Your line is now open.

Maybe you can you comment or talk a little bit about a preclinical data or work you've done Microdisplay pinching therapy, plus if you know combo and what you're seeing there.

So weird.

I think at this point all all will say is that it's good it's an area of keen interest to us and we are doing a preclinical work on both on two different concepts. One concept is the value of pre treatment with the PMO where PMO.

Joel Beattie: Hi, this is Sean Egan calling in for Joel. Thank you for taking my question. On the PPMO, congratulations on the 20 mg per kg dose. Maybe can you comment or talk a little bit about any preclinical data or work you've done on microdistribution gene therapy plus a PPMO combo and what you've seen there? So we're, I think at this point, all we'll say is that it's an area of keen interest to us, and we are doing preclinical work on two different concepts. One concept is the value of pre-treatment with a PMO or PPMO in advance of a gene therapy to enhance the combinative value of those therapies and to enhance expression. And as relates to that, I can lean over and talk about literature that already exists. Dr. Voigt, for example, has some literature where he in a mouse model pre-treated with a, I believe it was a peptide conjugated PMO and then a dose of gene therapy, and he found enhanced expression.

In advance of a gene therapy to enhance the accommodate the value of those therapies and to enhance expression and as relates to that I can I can lead over and talk about literature that already exists Dr. voice as an example.

Has and it has some literature, where he in the a mouse model pretreated with a I believe as the peptide conjugated PMO.

And then dosing gene therapy. He found enhanced expression, that's one area and we're looking at that area. The second one of course is a very significant one which is to explore the benefit of a common at the value of a ppm, though on the one end and GM gene therapy any other and you can envision a world in which you would get.

A synergistic value between it really significant expression from the gene therapy, Karnes drug like our SRP Nigeria's Irwin coupled with robust expression of a of a truncated dystrophin that you would get from P. PMO, we're doing a bunch of work on that we're not ready to discuss it in any detail, but it is certainly something.

Douglas S. Ingram: That's one area, and we're looking at that area. The second one, of course, is a very significant one, which is to explore the benefit of the combined value of PPMO on the one hand and gene therapy on the other. And you can imagine a world in which you would get synergistic value between a really significant expression from a gene therapy construct like our SRP 9001, coupled with robust expression of a truncated distrofen that you would get from a PPMO. We're doing a lot of work on that. We're not ready to discuss it in any detail, but it is certainly something that we need to continue to work on in advance of both the launch of our gene therapy program, assuming that we're successful in clinical development, as well as in advance of the success of our PPMO program as well.

That we need to continue to work on in advance of both the launch of our gene therapy program, assuming that were successful in clinical development as well as in advance of the success of our PBM O program as well and I think you know what people their models now working assumption that gene therapy, well entirely cannon.

<unk> was the or in a platform and like I understand why some people look at the exciting aspects of our gene therapy in imagine that I think it's probably early tour to believe that's the case and we have some tantalizing research that might say there is a valued at these two therapies together.

Thank you. Our next question kind of stand Matthew Harrison written like in Stanley. Your line is now open.

Douglas S. Ingram: I think, you know, a lot of people in their models have a working assumption that gene therapy will entirely cannibalize the RNA platform, and while I can understand why some people look at the exciting aspects of our gene therapy and think that, I think it's probably early to believe that's the case, and we have some tantalizing research that might say there is value in these two therapies.

Thank you Oh this is Matt score on for Matthew Harrison, Sorry, if I missed this but can you confirm that all micro dystrophin released that they have been built but to still need to be validated and are these the in vitro potency assets. Thank you.

Yeah, Great question. So, yes, I can confirm that all of the assays are belts.

I can confirm it up 24 hours days all of them are either are either validated requalified. Some assay they'd be qualified traffic maybe the validated and there are two that are in the later stages of that validation I can also tell you that it's not that those yet but all of that all of the really crucial bespoke assays.

Lisa Baker: Thank you. The next question comes from Matthew Harrison with Morgan Stanley. Your line is now open. Thank you. This is Max Scoron for Matthew Harrison.

Douglas S. Ingram: Sorry if I missed this, but can you confirm that all the microdistrophin release assays have been built, but two still need to be validated? And are these the in vitro potency assays? Thank you. Yeah, a great question. So, yes, I can confirm that all of the assays are built. I can confirm that of 24 assays, all of them are either validated or qualified; some assays need to be qualified, a lot of assays need to be validated, and that there are two that are in the later stages of that validation. I can also tell you that it's not the potency assay, and all of the really crucial bespoke assays are either validated or qualified; the last two that are in the last stages are really very routine; these are, sort of, nearly off-the-shelf routine assays, so all of the really significant ones have already been validated.

Our either validated or qualify the last two that are in the last stages are really that a very routine either buried sort of nearly off the shelf routine assets. So all of the really significant ones have already been validated.

Thank you I'm next question comes condensing 10, Rethinking your line is now up in.

Thanks for taking my question, though just wanted to follow quickly on your comment earlier on looking for exon skipping a 12 week, but not necessarily dystrophin I'm asking you start getting exxon's give dystrophin or in the case of gene therapy. After you started expressing micro dystrophin M&A. How quickly would you expect to start seeing meaningful dystrophin levels and and then how does that continue to trend.

It overtime, how long does it take for example to to reach that he said.

Yeah. So I'll give you I'll give you the broad stroke, and then Dr. Neil if you have additional color on it I mean look first of all we've got to we definitely have to take arent, a and micro dystrophin gene.

Bo Cumbo: Thank you. Our next question comes from Vincent Chen on behalf of Bernstein. Your line is now open.

Vincent Chen: Thanks for taking the question. I just wanted to follow up quickly on your comment earlier on looking for exon skipping at 12 weeks, but not necessarily dystrophin. After you start getting exon skip dystrophin, or in the case of gene therapy, after you start expressing microdystrophin mRNA, how quickly would you expect to start seeing meaningful dystrophin levels? And then how does this continue to trend over time? How long does it take, for example, to reach steady state?

Gene therapy and put it in two different buckets, there very different with respect to micro district, and we know the answer is three months. So what we've seen as you know with respect to SRP nine 001 in our first for kids. We took biopsies. After three months in those kids had you know, 90% and higher dystrophin expression. So clearly we get.

Douglas S. Ingram: Yeah, so I'll give you the broad strokes, and then Dr. O'Neill, if you have additional color on it. I mean, look, first of all, we definitely have to take RNA and micro dystrophin gene therapy and put them in two different pockets. They're very different with respect to micro dystrophin.

It really fast onset or that gene therapy for or a day. It is a slower processing. It occurs over time, probably for a host of reasons, but it. It occurs over time in fact, we saw a significant difference over a long course period of time with.

I don't think scientists and with respect to buy on this and we saw I think the wind between one year at four years. There was a significant difference in there not a dystrophin and that may be in part because when you would this chronic therapy the chronic therapy keeps increasing the benefit over the long term one of the reasons Franco.

During this difficult Kobin crisis that reason so focused on ensuring the kids don't skip dosing because skipping a dosing isn't simply missing a dose it may be missing the cumulative benefit that you're getting from it being on therapy over a long period of time, but with that said I'll turn this every doctor or any other can maybe provide some more color.

<unk>.

So as Vincent Thanks for your question.

The at half life of wives tight you pull them to scope. It isn't the order of weeks. This we know and as Doug said, the or not surprisingly the time to transcribe at high Fidelity and translates high fidelity to Scotiabank, we just such an art large G actually is it may.

Sure Bill in many hours I think so those are important biological question then for the points you are looking at 12 week data the key decisions will be driven by the rent as opposed to seize up people to PMO and what we read the robust data set that we have.

At that time frame really add.

Forces us for prices to compare exon skipping, which I think is actually a very valuable way of making the decisions going forward and selecting doses as Doug said and as you can expect front, but I just said about the half life up just frozen and the.

Syntactic timelines at 14 or dispose and one would expect to see ongoing accumulation over many weeks and months. So thats. The reason that actually focusing exon skipping at 12 and remember also we're measuring tissue leveled off the TPL, though it would be to compare that with people as well.

So that really enables us to refi test mostly out of the hypothesis.

Yeah I understand this about let's talk a moment about what we know.

About our R&D technology. The PMO generally so you know with the things about our PMO that make it.

It gets so clever is that number one is for subs that we know we get the PMO to the right place it induces exon skipping a transforms messenger ordinary it puts it back and frame. It. It makes district, we love It we know check right. The PMO, we can dose at very high level that has a very significant safety window there with its therapy.

Douglas S. Ingram: We know the answer is three months. So what we've seen, as you know, with respect to SRP 9001 in our first four kids, we took biopsies after three months, and those kids had, you know, 90% and higher dystrophin expression. So clearly, we get a really fast onset for the gene therapy. But for RNA, it is a slower process, and it occurs over time, probably for a host of reasons.

Douglas S. Ingram: But it occurs over time. In fact, we saw a significant difference over a long course period of time with both extondus and with respect to biondus. And we saw, I think the one between one year and four years, there was a significant difference in the amount of dystrophin. And that may be in part because when you give this chronic therapy, the chronic therapy keeps increasing the benefit over the long term. One of the reasons, frankly, during this difficult COVID crisis, that we have been so focused on ensuring that kids don't skip doses because skipping a dose isn't simply missing a dose; it may be missing the cumulative benefit that you're getting from being on therapy over a long period of time.

That window, the the one limitation and it is a significant limitation of our Vmoso is that at the neutrally charge molecule it doesn't get into the sell well and as a result of that.

Douglas S. Ingram: But with that said, I'll turn this over to Dr. O'Neill to maybe provide some more color. So Vincent, thanks for your question. The half-life of wild-type full-length dystrophin is in the order of weeks.

Gilmour O'Neill: This we know. And as Doug has said, not surprisingly, the time to transcribe at high fidelity and translate the high-fidelity dystrophin, which is such a large gene, actually is measurable over many hours. I think those are important biological questions.

Douglas S. Ingram: But then from the point of view of looking at 12-week data, the key decision will be driven by the relative potencies of PPMO to PMO. And what we really have, the robust data set that we have at that timeframe really... For more information, visit www. ISGlobal.org, I understand this about, let's talk for a moment about what we know about our RNA technology, the PMO generally. So, you know, the things about our PMO that make it so clever are that, number one, it's precise. That we know if we get the PMO to the right place, it induces exon skipping, it transforms messenger RNA, it puts it back in frame, and it causes dystrophy.

A lot of the PMO that is infused it doesn't get into the sell to utilize urinated out. So the goal of all of this you know the unlocking of already technology and a potential profound improvement of the R&D technology comes from something that is that is it just simple to discuss front.

Douglas S. Ingram: We know that. We know it's safe, right? The PMO we can dose at very high levels. It has a very significant safety window and with its therapeutic window. The one limitation, and this is a significant limitation of our PMOs, is that, as a neutrally charged molecule, it doesn't get into the cell well.

It really simple and difficult to execute which is can you find a delivery vehicle that can safely get the PMO into cells in much greater exposure.

Douglas S. Ingram: And as a result of that, a lot of the PMO that is infused doesn't get into the cells and isn't utilized; it's urinated out. So, the goal of all of this, you know, the unlocking of our RNA technology and a potential profound improvement of the RNA technology comes from something that is, that is, it is... Simple to discuss. Frustratingly simple and difficult to execute, which is, can you find a delivery vehicle that can safely get the PMO into the cells at a much greater exposure? That's the question. In animal models, the PBMO does that very mechanically. It's a positively charged peptide. It interacts with the negatively charged elements on the outside of the cell, creates warbling, induces vesicles, and it gets into the cell in great abundance, you know, an order of magnitude greater than you would see with a PMO.

That's the question in animal models, the PBM out does that very mechanically positively charged up time it interacts with the negatively charged on elements on the outside of the celebrates warbling. It Didnt. This is vessels it gets into the sell in greater bundles, you know in order of magnitude greater than you would see where the PMO.

And so that's the biggest issue for US right now can we get too.

Strong therapeutic doses, we envision any therapeutic doses of the P. PMO without and do you do see inducing a safety signal that if we can I think mechanically we're going to be very.

Douglas S. Ingram: And so that's the biggest issue for us right now. Can we get to, www.larryweaver.com, The second half of 2020? The exciting thing about where we are right now is that we're at 20 minutes per gig when we envisioned at least some time ago that our ceiling was going to be about 12. Thank you. Our next question comes from Anupam Rama with JP Morgan. Your line is now open.

We're excited about the potential in the PBM Alan and in that regard we need to see this data it will have a data read out in the second.

Second half of 2020, the exciting thing about where we already know that work when it makes for keurig. When we envisioned at least sometime ago that are feeling was going to be about 12 experts.

Thank you.

Our next question comes from Anupam Rama with JP Morgan Your line is Hamilton.

Hi, all this is happening across many fronts anytime hook, Alabama and thanks for taking your question.

I think the prepared in the prepared remarks down he mentioned that you do not have clarity to provide updated guidance I understand that.

But maybe from a high level now we're going to lever in turn gross and three 1 million in 2019, and maybe how are you thinking about that push and pull that variants in the guidance to consider way he aren't going to come at 19 pandemic. Thanks, so much.

Anupam Rama: Hi all, this is Tess on the call tonight for Anupam. Hope you're all well and thanks for taking our questions. I think in the prepared remarks, Doug, you mentioned that you do not yet have clarity to provide updated guidance for exondus. But maybe, from a high level, what were the levers for growth from 381 million in 2019?

Yeah, I'll give you the broad strokes, though so the our growth with respect to axon deals.

Doesn't relate to price, let's make sure we're clear about that to the there was only one lever and its patients on therapy staying on therapy, and we're finding more patients will more patients coming on therapy that is it because as I hopefully people now and I think I've said in other forms many times.

Douglas S. Ingram: And maybe how are you thinking about the push and pull levers for guidance to consider with the ongoing COVID-19 pandemic? Thanks so much. Yeah, I'll give you the broad strokes that our growth, with respect to exon, doesn't relate to price.

We launched I'm excited to 2016, we have taken no price increase over that time, and we don't envision taking a price increase our goal is to grow through serving as community and having more kids benefit from our therapies and as an example, with respect to buy on this we priced at apps.

Douglas S. Ingram: Let's make sure we're clear about that. So there is only one lever, and that is patients on therapy, staying on therapy, and finding more patients, and more patients coming on therapy. That is it. Because, as hopefully people know, and I think I've said in other forums many times, we launched Exondus in 2016. We have taken no price increases over that period of time, and we don't anticipate taking a price increase.

Parity with Exxon is we Didnt, obviously, good price increase there. So when we look forward, that's the lever and the and the.

The the risk to revenue is modest for the second half of the year certainly with respect to act on this because with respect to act on this the great majority of kids are already in home infusion and while there are a number of different.

Douglas S. Ingram: Our goal is to grow by serving this community and having more kids benefit from our therapies. And as an example, with respect to Vyondus, we priced it at parity with Exondus. We obviously didn't take a price increase there. So, when we look forward, that's the lever, and the... The risk to revenue is modest for the second half of the year, certainly with respect to exondus because, with respect to exondus, the great majority of kids are already on home infusions. And while there are a number of different elements that could be disruptive over the course of 2020, the significant disruption is kids that don't have the ability at the peak of this crisis to get into a hospital to get an infusion if they're in a hospital. The good news is that most of these kids don't.

Elements that could be disruptive over the course of 2020 that significant disruption that kids that don't have the ability at the peak of this crisis to get into a hospital to get it infusion if you're in hospital in the good news about the kids or not so when I talk about axon. This in particular, we're talking about it they said everything we see today and as I said I'm not giving up there.

Got it because we're in the middle of something and we don't have sufficient clarity to give guidance that we feel confident about other than to say, what we're seeing right now would be a modest in shortly short churn in that well see a little more significant delaying impact on BYOD. This for the simple reason that were in large phase with violence that anymore signal.

We get number of patients well start in the hospital or that we're working to try to reduce that number I'm, but even with respect to that we think that's going to be a modest and we think that's going to be a short lived issue. So so broadly speaking I think we're in good shape, we're serving the patient community. We're trying our very best and I think were so far.

Douglas S. Ingram: So when I talk about Exondys in particular, we're talking about it based on everything we see today. And as I said, I am not giving updated guidance because we're in the middle of something, and we don't have the sufficient clarity to give guidance that we feel confident about other than to say that what we're seeing right now would have a modest and short-term impact. We'll see a little more significant delay and impact on Biondys for the simple reason that we're in the launch phase with Biondys, and a more significant number of patients will start in the hospital, although we're working to try to reduce that number. But even with respect to that, we think that's going to be modest, and we think that's going to be a short-lived issue.

Succeeding and keeping them safe and any impact on revenue ought to be really quite modest and quite shortly but with that though I missed anything.

No Doug you covered everything thank you.

Thank you I next question comes from Tim Lugo with William Blair. Your line is now open.

Oh, Thanks for taking my question for the commercial supply trial have the ongoing issues on Covidien talking about how you're thinking about that study size, maybe adding additional sites than you originally anticipate or listening geography easily. Meanwhile, patients.

Douglas S. Ingram: So, broadly speaking, I think we're in good shape. We're serving the patient community. We're trying our very best, and I think we're so far succeeding in keeping them safe. And any impact on revenue ought to be really quite modest and quite short-lived. But with that, though, have I missed anything?

Well, even just feel that powering of that study.

Douglas S. Ingram: No, Doug, you covered everything. Thank you. Thank you. Our next question comes from Tim Lugo with William Blair. Your line is now open.

The they sort of question, though the.

The.

Study remains on pace and on track as originally envisioned both the size of the study says that it's multi country multi multi institute. The fact that would keep a controlled et cetera. So the design hasn't changed on that study I have.

Tim Lugo: Thanks for taking the question. For the commercial supply trial, have the ongoing issues around COVID impacted at all how you think about that study size, maybe adding additional sites than you originally anticipated or additional geographies where you may enroll patients, or even just the overall powering of that study?

Said that the the one logistical risk in the started that study is that you know you've got to get studies up and running you've got to get lip.

Douglas S. Ingram: Thanks for the question. The study remains on pace and on track as originally envisioned, both the size of the study, the fact that it's multi-country, multi-institutive, the fact that it's placebo-controlled, etc. So the design hasn't changed for that study.

Perfect World you have in person site initiation visits from our clinical operations team and the like we want to make sure everyone's properly.

Trains so that we have consistency across all of the sites all around the world. So there's a lot to do and so we envision that there will be a modest delay in the initiation of that trial just to make sure that were being thoughtful and cautious about that the overnight units were starting that means they'll be a modest delay I.

Douglas S. Ingram: I have said that the one logistical risk in the start of that study is that you've got to get studies up and running. In a perfect world, you would have in-person site initiation visits from our clinical operations team and the like. We want to make sure everyone's properly trained so that we have consistency across all of the sites all around the world. We anticipate that there will be a modest delay in the initiation of that trial just to make sure that we're being thoughtful and cautious about COVID-19 as we start. That means there will be a modest delay. I say modest because we still have every intention of starting study 301 in the second half of this year. So we're talking delays measured in a couple months, not measured in significant amounts of time.

Say modest because we're still we still have every intention of starting study three one in the second half of this year. So we're talking delays you know measured in a couple of months not measured in significant amounts of time, but beyond that I think things are proceeding and we haven't made any significant changes to the approach that we're taking.

Thank you and next question kind of some recent Baker with JMP Securities. Your line is how open.

We set your line is needed season yet.

Oh, sorry, [laughter], thanks for taking my questions and congratulations on a strong quarter I'm just wanted to know if you could give us a little bit more details on a and color from the quarter at south in terms that you know how many patients that hits Pentagon by on that somebody can better understand that the dynamics there and then.

Douglas S. Ingram: But beyond that, I think things are proceeding, and we haven't made any significant changes to the approach. Thank you. Our next question comes from Lisa Baker with J&P Securities. Your line is now open.

So any changes in gross to nets or any of those kind of detailed that we should be thinking about you know.

Given you know patients insurance and maybe kind of levels are not implement that there might be what's the right way to think about some of those dynamic. Thank you Sarah I will turn this question ever double although I think some of the new wants would probably not at liberty to disclose.

Douglas S. Ingram: Lisa, if your line is muted, please unmute. Thanks for taking the questions and congratulations on a strong quarter. I just wanted to know a little bit more details and color from the quarter itself in terms of, you know, how many patients actually started on Biondos just so we can better understand the dynamics there and then also any changes in growth to NETs or any of those kind of details that we should be thinking about, you know, given patients' insurance and maybe kind of levels of unemployment there might be. What's the right way to think about some of those dynamics?

Yeah Lisa.

Obviously, we're not going to get we never got into patient numbers for Exone is so we won't with by honest and but I will tell you. Even though you know biologists is obviously a smaller than Exxon is from a population standpoint, we were very pleased with the progress that we're making with payors and coverage we were.

Well, we were much farther ahead to it than we were with Exxon does so we have a 148 million lives that are covered don't Peter you know restricted policy or to label. So we're very pleased with that.

Bo Cumbo: Thank you. Sure. I will turn this question over to Bo, although I think some of the new ones are probably not at liberty to disclose.

Lisa Baker: Yeah, Lisa, obviously, we're not going to get We never got into patient numbers for exondus, and we won't with biondus. And I will tell you, even though, you know, biondus is obviously smaller than exondus from a population standpoint, we were very pleased with the progress that we were making with payers and coverage; we were, we were much further ahead than we were with exondus. So we have 148 million lives that are covered up either by restrictive policies or two labels.

And though the launch was going to our expectations. So.

It's just the small hick up up the pandemic that put a little things put some clutches and to but we were overall very very I'm happy.

Thank you I'm not showing any further questions at this time I would now like to tend to contact of attacking them for closing remarks.

Alright. Thank you I'll be brief thank you all very much for joining us for our earnings call I'm hopeful you'll have seen here that like so many other companies and frankly individuals are not just in the United States, but around the world. This pandemic has.

Bo Cumbo: So we were very pleased with that, and the launch was going according to our expectations. So it's just the small hiccup of the pandemic that put some little things, put some glitches in it, but we were overall very, very happy. Thank you. I am not showing any further questions at this time. I would now like to turn the call back over to Doug Ingram for closing remarks. Alright, thank you.

Cause enormous numbers challenges and obstacles or the sarepta team, but as I said before I'll say it again, because I I. It bears repeating I am unbelievably proud of the Sarepta team for the ability to stay on mission, 90% of this team transition.

Douglas S. Ingram: I'll be brief. Thank you all very much for joining us for our earnings call. Hopefully, you'll see and hear that, like so many others, frankly, individuals, not just in the United States but around the world.

Two working from home and yet we did that on a Friday, we got up on Monday I have taken a careful look at every metric you can see and not only externally when you see the way we're tracking against our milestones, but through all of the metrics you'll see that this organization remains as productive as it was on a Friday before we all want to.

Douglas S. Ingram: This pandemic has caused enormous numbers of challenges and obstacles for the Sarepta team. But as I've said before, and I'll say it again, because it bears repeating, I am unbelievably proud of the Sarepta team for the ability to stay on mission. 90% of this team transitioned to working from home. And yet, we did that on a Friday; we got up on Monday. I have taken a careful look at every metric you can see. And not only externally, when you see the way we're tracking against our milestones, but through all of the metrics, you'll see that this organization remains as productive as it was on the Friday before we all went to work from home. So there is a lot of learning in this, probably not just for Sarepta but for all of us about the ability to be efficient, even in a virtual manner.

Working from home. So there's a lot of learning and that's probably not just for sarepta, but for all of us about the ability to be efficient even in a virtual manner. We are mission oriented as an organization, we have never taken our eye off the the need to bring a better life to these kids and even through this difference.

Open distracting period of time, even in a period of time, when our workers themselves have to worry about their own loved ones and about themselves. They have not lost sight of the fact that strep is on mission or can that continue to actually the cute across 2020, I'm gonna be excited to give you additional updates across the year, we can give you better clarity.

Douglas S. Ingram: We are mission-oriented as an organization. We have never taken our eye off the need to bring a better life to these kids. And even through this difficult and distracting period of time, even in a period of time when our workers themselves have to worry about their own, and about themselves, they have not lost sight of the fact that Sarepta is on a mission.

Sales in our second quarter earnings call, we have a number of significant milestones.

Across the rest of this year I think the rest of this year into 2021 is going to be an enormously consequential a period of time not only for sarepta, but for the patients that we serve so thank you all very much everyone. Please stay safe wash your hands and let's get this crisis behind us.

Douglas S. Ingram: We're going to continue to execute across 2020. I'm going to be excited to give you additional updates throughout the year. We can give you better clarity on sales in our second quarter earnings call.

Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.

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Douglas S. Ingram: We have a number of significant milestones across the rest of this year. I think the rest of this year and into 2021 is going to be an enormously significant period of time, not only for Sarepta but for the patients that we serve. So thank you all very much. Everyone, please stay safe, wash your hands, and let's get this crisis behind us. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. Thanks for watching!

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Operator: .. .. .. .. .. .. .. .. .. .. ... [inaudible] Tazeen Ahmad, Tim Lugo, Debjit Chattopadhyay, Ritu Baral, Uy Ear, Brian Abrahams, Gil Blum, Gena Wang, Anupam Rama, Judah Frommer, Ian Estepan, Douglas Ingram, Dallan Murray, Jacob Elkins, Mike Ulz, John Boyle, Anvita Gupta, Zhiqiang Shu, Mary Jenkins, John Boyle, Ian Estefan, Francesca Nolan, Sarepta Therapeutics Inc Tazeen Ahmad, Tim Lugo, Debjit Chattopadhyay, Ritu Baral, Uy Ear, Brian Abrahams, John Boyle, Anvita Gupta, Zhiqiang Shu, Mary Jenkins, John Boyle, Anvita Gupta, Zhiqiang Shu, Annabel Witt, Anvita Gupta, Anvita Gupta Music ?? ?? ?? ?? ?? ?? Copyright © 2020 Mooji Media Ltd. All Rights Reserved. No part of this recording may be reproduced without Mooji Media Ltd.'s express consent.

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