Time all participants are in a listen only mode. As a reminder, today's program is being recorded. And now I'd like to introduce your host for today's program Ian Esteban, Senior Vice President, Chief of Staff And Corporate Affairs. Please go ahead.
Thank you all for joining today's call. Earlier today, we released our financial results for the first quarter 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 Mahhotme, Bokumbo, Doctor. Gilmore O'Neal and Doctor.
Luis Rodino Klapac. After our formal remarks, we'll open up the call for Q And 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 could materially differ from these forward looking statements and any such risks to materially and adversely affect the business the results of operations and the trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainty, we encourage you to review Arcadia'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.
To our CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?
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-nineteen. I am very proud that the Sarepta team so rapidly responded to prices, 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, information technology group, our clinical operations function and our commercial organization.
He began contingency planning as early as January of 2020 In the event that on the situation unfolded. 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, had the systems and the infrastructure in place, to seamlessly transition, execute and remain connected as we commenced work on that Monday morning. 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 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-nineteen has created some challenges and uncertainties and will continue to do so for the next few quarters. On hole, we are uniquely positioned to drive our strategic plans and to stay on mission during these difficult times. In that regard, consider the following.
1st, 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 $1,150,000,000 to our balance sheet. We do start burn rate through cost sharing and entitles as to milestones along the development and regulatory pathway. Along with our Roche infusion, at the end of the first quarter, we had nearly 2 point $2,000,000,000 of cash on hand not including another $250,000,000 in a debt facility that we have not drawn down. This level of cash on That is before considering our revenue from EXONDYS 51, BYONDYS 53, and if and when approved, casimersen.
We are in a strong on track. 2nd, 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 worked diligently to minimize the impact of COVID-nineteen. Moreover, with respect to Study 102, our gene therapy trial for micro dystrophin. This is a one time therapy in all patients for the primary 48 week analysis have been dosed.
That trial progresses, and there is no foreseeable risk of a delay in trial data. Next, with respect to our currently available therapies, EXONDYS and Viandis for the treatment of Duchenne muscular dystrophy, Our supply chain is fully intact and we are able to manufacture its supply therapy without any interruption, and we do not anticipate this changing. And finally, one of our most significant strategic activities is building out commercial gene therapy manufacturing And we made less progress in the last 12 months. COVID-nineteen 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 assays are built. And all but 2 of the 24 necessary assays are either already validated qualified as required with the remainder to 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 $100,400,000. 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 home, We do anticipate For instance, some patients This impact will be more significant for Viondis, which is just launching than EXONDYS as most existing patients 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. 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 reauthorizations. We currently anticipate the COVID related impact on sales to be both modest and short lived.
Given the dynamic and unprecedented nature of this endemic, 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 at our second quarter earnings call, will provide an updated view. Moving 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 9001, our micro dystrophin gene therapy program, Our Study 102 evaluating the safety and efficacy of SRP 9001 in patients with Duchenne muscular dystrophy is proceeding and it is in good shape.
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 delay. We have 2 sites for Study 102. Our site at Nationwide Children's Hospital with Doctor.
Jerry Mendell as principal investigator imposed 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 at UCLA with Doctor. 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 material. We are continuing to make progress toward the initiation of trial sites. However, COVID-nineteen does create some challenges here.
While the team is making progress, COVID-nineteen creates uncertainties around the status of some clinical sites and will likely delay some necessary site initiation visits. 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 participants. We had anticipated commencing Study 301 around the middle of 2020. We are on track to modestly delay initiation, but we'll still anticipate commencing Study 301 in the second half of this year. Moving to lgMD2E.
As you will recall, our goal was to have expression safety data from our 3 patient cohort in our high dose arm for SRP 9003 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 are also on course.
Manufacturing is progressing. We've commenced our first commercial GMP run for LGMD2E, And we intend to commence what we hope to be the pivotal trial in 2021 as previously anticipated. With respect to MPS IIIA, our gene therapy trial with Lysogene, that trial has enrolled in dose 19 of the 20 patients in that trial, and is on track to dose As you know, we are in our rolling submission for our 3rd RNA therapy, casimersen intended to treat DMP patients revenue mutation amenable to exon 45 skipping. That submission is proceeding and it has not been impacted by COVID-nineteen. We should have that submission complete this quarter as anticipated.
Our 2 RNA confirmatory trials, and that's mission for EXONDYS, and essence for Viandis and its approved casimersen, our chronic therapy trials and largely ex CRS. And thus, COVID-nineteen 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 impact, ensure trial integrity is preserved and that the trials are proceeding. Next, we are in our multi ascending dose trial for SRP-five thousand and fifty one, 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 advancement over our current RNA technology, the PMO.
It was our intention to provide a data While we still intend to announce those results in 2020, COVID-nineteen did interrupt some dosing, which caused a very modest delay, but importantly, the team quickly addressed the obstacles, permitting 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 6 mgs per kg to as high as about 12 mgs per kg. However, We have escalated through those doses, and we are already dosing a 20 mgs per kg, 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. Again, we still anticipate a readout for our PPMO in the second half of this year, 2020. Speaking of our PPMO 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 UsamRID, the lead Medical Biologics Labs for the Department of Defense have entered into a cooperative research and development agreement to evaluate our PPMO to treat COVID-nineteen. 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 coronaviruses.
Ahead of DecemberID has a detailed knowledge of our PMO technology. And on that basis, reached out to us, to propose working on 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 insufficient supply to be evaluated. We have already transferred them to Samarid which will be responsible for testing and evaluating them in their proprietary in vitro models to determine their potential in reducing viral replication. If 1 or more of our candidates shows promise, usamrit 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 in the leg. It has been fullest extent that we can avoid that. 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 had technology that may benefit society in this fight. So when crisis came, we answered the call, and through this cooperative agreement with Usamarin, have been able to employ our technology rapidly and to do so without distracting us or taking substantial resources away from our main mission. Things are going quite well. Our employees are removing obstacles and staying on mission, 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, Indura, North Carolina, where we are already in our facility and hiring scientists all under the leadership of Doctor.
Charlie Gersbach of Duke University. I would like now to give a big thanks to our dedicated facility dependent workers who've 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 with references to COVID 19. 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-nineteen, 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 metal, our resourcefulness, our creativity, our optimism and our commitment. We often learn far more about ourselves in time of crisis than in times of ease. 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 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.
And I am proud that Sarepta, with our proprietary RNA technology is playing a role in fighting this disease, even as we remain laser focused on advancing our rare disease mission and serving our patients. This COVID-nineteen may seem fierce to some, but it is by no means invincible and it is no match for biotechnology innovation. Armed with 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, one driven by a commitment to develop therapies with a pace that allows us to intervene in time to save lives.
The diseases we fight are unrelenting. They do not take time off for this crisis and so our Sarepta family is unrelenting. We have not taken time off for this crisis. When this crisis came, this team answered the call, adapted and kept executing. And like so many people today, they did all of this while dealing with new challenges.
New working environments, having to juggle work and childcare and loved one concerns and concern for themselves. 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 bothered. And with that, I'll turn the call over to Bo. Bob?
Thank you, Doug. Good afternoon, everyone. Despite the headwinds facing our healthcare system due to the COVID-nineteen pandemic, I am pleased to report that our product revenue for the first quarter of 2020 totaled $100,400,000 Our experienced teams at Sarepta are actively working to navigate through the challenges of the COVID-nineteen pandemic. And allow us to mitigate major We will continue to navigate 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 ByONDYS 53 to patients.
Consistency of supply is key. Therefore, we will continue our efforts to ensure that EXONDYS 51 and Biones 53 are supplied to the strain the COVID-nineteen pandemic is placed on healthcare workers, hospitals and distribution channels. Due to recent shutdowns and restrictions at hospitals and clinics, our team is working closely with healthcare 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. 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 appointment with healthcare providers. We will continue exploring options for patients to safely initiate treatments with IL-fifty 3.
We are still engaging with key opinion leaders and other healthcare 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 ambulish 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 engagements. To help minimize access and reimbursement barriers, We continue to work with commercial and state Medicaid plans on reauthorizations so that patients are able to stay on therapy. We are encouraged by the efforts payers have made to not disrupt patients treatment plans during this difficult time.
Transitioning to our performance for the first quarter. 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 patients on EXONDYS 51 without significant disruptions. In the current environment, the dynamics of initiating treatment with EXONDYS 51 or Biologous 53 are affected. While many clinics are closed or not seeing patients normal in person appointments. The impact has resulted in fewer patients initiating treatment.
However, we do anticipate this will change as restrictions ease and claims resume normal operations. As a reminder, BioNT 53 year voters had received accelerated approval from the U. S. FDA on December 12, 2019. Dialen's 53 treats patients with Duchenne muscular dystrophy, who are amenable to Epsilon 53 skipping.
We anticipate that patient demographics for Vionicus 53, we will be similar to EXONDYS 51 with regards to the average age of patients on therapy and 6 commercial versus Medicaid patients. Over the past 3.5 years, we've continuously reviewed and refined our approach for EXONDYS 51. While we're revising a deep knowledge and expertise from that launch, we will continue to monitor the impact that COVID-nineteen pandemic has on and beyond this 53 launch, trajectory. We are confident over time, patients will ultimately receive access and reimbursement for MYONIS 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 that Dean has made to date, particularly in light of the extraordinary circumstances.
And knowledge we gather strengthens our plans for future launches, including casimersen in 2021. The depth of experience on our 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 of precision genetic medicine. And with that, I'll turn the call over to Sandy. Sam?
Thanks, Bo. Good afternoon, everyone. In the first quarter, total revenues 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 IND's 53 We are in a strong financial position with significant capital to fund that pipeline and ramp up manufacturing, while maintaining our overall timelines. In addition, we now have the ability to access Roche's significant expertise and greatly enhance our global opportunity for SRP-nine thousand and one. Moving to the financials.
This afternoon's press release provided details for the first quarter of 2020 on a non GAAP basis as well as a GAAP basis. The press release is available on Sarepta's website. Please refer to our press release for full reconciliation of GAAP to non GAAP. Net product revenues for the first quarter of 2020 from our products EXONDYS 51 and YONIS 53 with 100,400,000 compared to $87,000,000 for the same period of 2019. The increase primarily reflects higher demand for our products.
In the quarter ended March 31, 2020, we recognized $13,200,000 of collaboration revenue, which relates to our collaboration agreements as well. In February 2020, we received an aggregate of approximately $1,200,000,000 in cash consideration from Roche consisting of an upfront payment and an equity investment in Sarepta. From an accounting perspective, $242,700,000 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 for Sarepta's policy. For the quarter, core development costs under the Roche agreement totaled $16,400,000 and are included as a reduction to R and D expenses.
On a GAAP basis, we reported a net loss of $17,500,000 $76,600,000 or $0.23 per share and a dollar $7 per share for the first quarter of 2020 2019, respectively. We reported a non GAAP net loss of 79,800,000 or $1.04 per share in the first quarter of 2020 compared to non GAAP net loss of $53,800,000 or $0.75 per share in the first quarter of 2019. In the first quarter of 2020, we recorded approximately $12,600,000 in cost of sales, compared to $12,100,000 in the same period of 2019. The increase was due to royalties paid to BioMarin Pharmaceuticals, and University of Western Australia, as well as higher product costs as a result of increasing demand for our products. This was partially offset by right off of certain batches of EXONDYS 51 that did not meet our quality specifications for the 1st 3 months of last year.
There's no similar activity for the 1st 3 months that ended March 31, 2020, I. E. This year. On a GAAP basis, we recorded $136,100,000 and $90,600,000 in R and D expenses for the first quarter of 2020 2019, respectively, which is a year over year increase of $45,500,000. This increase is primarily related to $43,300,000 increase in clinical and manufacturing expenses.
A non GAAP basis, R and D expenses were $114,200,000 for the first quarter of 2020 compared to $81,400,000 for the same period of 2019, an increase of $32,800,000. The year over year growth in non GAAP R and D expenses has been primarily due to a continuing wrap up of our micro dystrophin program as well as our SSENSE program. Turning to SG And A, on a GAAP basis, we recorded $82,800,000 $60,600,000 for expenses for the first quarter of 2020 2019, respectively. Year over year increase of $22,200,000. On an non GAAP basis, the SG and A expenses were $54,500,000 for the first quarter of 2020.
Compared to $47,800,000 for the same period of 2019, an increase of $6,700,000. The year over year increase was driven by significant organizational growth and expansion which 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 $7,400,000 in expenses, net for the first quarter of 2020 compared to $200,000 in other expenses, net for the same period of 2019. The unfavorable change primarily reflects interest expense on our debt facility entered into in December of 2019. In February of 2020, we entered into an agreement to sell the rare pediatric disease, priority of the voucher that we received from the FDA in contention with the approval by August 53.
In March of 2020, we completed our sale of the PRV and received proceeds of $108,100,000 net of commissions, which was recorded as a gain from the sale of the PRB, as it did not have any carrying value at the time of the sale. There was no similar activity during the 3 months ended March 31, 2019. We had approximately $2,200,000,000 in cash, cash equivalents, and investments as of March 31, 2020. With that, I'd like to turn the call over to Gilmore for an update on our research and development activities. Gilmore?
Thank you, Sandy, and good afternoon. I would dive a little deeper into the top R and D related activities that Doug has highlighted With particular emphasis on the actions the team is taking to minimize and where possible eliminate the distractions and obstacles caused by COVID-nineteen. First, our FRP-nine thousand and one microdystrophy program. We look forward to the imminent publication of the 1 year safety and functional data from the 4 clinical trial participants who received microdystrophin 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 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 of patient visits to the site we have been able to continue monitoring patient safety and data quality. While some clinical evaluations would take place outside of the protocol defined window 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 are also pleased to see that Nationwide Children's Hospital is loosening COVID-nineteen restrictions and starting to resume clinical trial activities.
And I should note, that our second study 102 site, UCLA, with Perry Shea's investigator, has not imposed restrictions. So study 102 is proceeding and we anticipate no delay in the readout of that study. As far as our study 301 plans go, I am pleased to report that we have adapted rapidly to the COVID-nineteen 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 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. Further, the bandwidth of ethics review boards and regulatory agencies to engage in reviews of new protocols made effective as they deal with a large volume of requests from multiple sponsors in multiple therapeutic areas to amend current studies impacted by the COVID-nineteen pandemic. Fortunately, 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 SRP-nine thousand and three 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 5 to 13 VG per kilogram to dose arm of four times that dose. 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. 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 sarcoglycate programs. We also believe that this data would have some read through to our microdystrophin study because the progress share the same vector and promoter.
And if you recall, are both dosed at high dose equivalents of 2 to 14th failure genomes per kilogram. We will also gain experience with this dosing in older and larger patient population. As the 3 patients in the high dose arm of 9003 are older and larger than the 4 to 7 year age range of boys included in the micro dystrophin study. Now moving on to our PPO platform and more specifically, our SRP-five thousand and fifty one program. I am very excited about this program because of its potential to improve upon the ability of PMO to increase disclose an expression by fusing a cell pen from peptide to the PMO to enhance intracellular and intranuclear dosing.
We have made a lot of progress over the past year and through modifications of the original development plan, achieved single dosing of 28 per kg in healthy human volunteers and are already achieving multiple dosing of 20 milligrams per kilogram in boys with Duchenne. If the 20 milligram per kilogram or 20 mgkg dose continues to be safe and well tolerated, we plan to continue to dose escalate to 30 mgkg and then potentially even to 40 mgkg. 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 the PPMO compared to PMO. In the coming months, we plan on analyzing the 12 week biopsies from the SRP-five thousand and fifty one hundred and twenty meter care cohort.
We will measure exon skipping by digital drop PCR, allowing us to directly compare the efficacy of our PMO and PPMO And we will find an appropriate forum this year to provide the safety biomarker and other data from the program to date. If we are able to reach a therapeutic window for SRP-five thousand and fifty one, the data will read through to our other DFP programs. Where we have a total of 6 PPMO 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 rare Exxon populations, which together make up about 35% of the address population. And finally, the results of SRP-five thousand and fifty one will also inform the viability of the PPO platform for new therapeutic areas.
1 of our research goals is to identify conditions suitable for treatment with PPMO. If we have positive data from SRP-five thousand and fifty one, we would be able to rapidly accelerate the development of the PPMO platform both within DMD and beyond DMD to other therapeutic areas. Further highlights the potential versatility of the PPO platform. As you recall, we just announced collaboration with U. S.
Amarin to explore therapeutic agents to come back COVID-nineteen. This collaboration builds on the antiviral therapeutic potential of PMO's identified and published by the company in the early 2000s. That original work found that Pivos had demonstrated antiviral activity in in vitro model 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. He built this by Duplexing to specific candidate sequences in the coronavirus RNA.
For example, the transcription regulatory sequence, and thus, theoretically inhibiting translation initiation and downstream suppressing viral replication In this new collaboration, U. S. Hamlet would use in vitro assays to evaluate the affinity of the PP mode to suppress viral proliferation and threat. This collaboration will enable Sarepta to contribute to the efforts to treat COVID-nineteen, while adding to our of the PMO platform. We did consider utilizing the PMO, PMO X, PMO Plus Platforms in the research collaboration.
However, based on our recent experience with our PPO platform, we are confident that it is the right approach for this effort. We are quite pleased to be able to collaborate with U. S. Ambridge in a way that allows us to contribute to the fight against COVID-nineteen but does not distract us from our R and D priorities. Our mission to deliver precision genetic medicines occasions with rare and serious genetic disorders
has served
as our North Star to guide us in how we are maintaining critical work on 1, RNA and gene therapy discovery portfolios 2, nonclinical 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 healthy There will be multiple data readouts over the next two quarters that will guide our next steps. And with that, I will hand back to Doug.
Thank you, Doctor. O'Neill.
Questions. Our first question comes from Salveen Richter with Goldman Sachs. Good afternoon. Thanks for taking my question. So with regard to
limb girdle, when you present this high dose data set in the second quarter, Could you just comment on poster selection what the options are going forward with regard to trial designs for the registrational study?
Sure. Thank you for that question, Savi. 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-nineteen limit issues, all of the kids that they does, all of the kids have bad news. By ops, we will provide an update, both on, biomarker data expression data and safety data that's quarter and then shortly thereafter we'll make a dose selection that would form than Google.
Can we inform the rest of the sarcoglycan dosing as well? And then over the course of the rest of this year, we're doing 2 things. The one thing we're doing is building manufacturing supply. You may have heard during my, opening remarks that we actually are already in a GMP run for Lim Girl 2E, which I would just linger on for a second and give kudos to our technical operations group for being able to get to 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 towards the end of year, but 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 with in 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 an approval. But I can't 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 2 things complete. We should have our process development complete as we do GMP material ready and, a good understanding Ceeper and FDA on the development pathway for limb girdle 2E and then the development pathway for the rest of the limb girdles. 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 t.
Our next question comes from Brian Abrahams with RBC Capital Markets.
Hey, thanks very much for taking my question. On micro dystrophin regulatory path, I'm curious your latest thoughts as to what you aim to bring to regulators for Study 3 to potentially support approval. I guess I'm wondering if that may change If there might be any changes given what you might be able to collect or what FDA 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 102? And how if at all, you think that might impact your ability to use it as registration enabling without full functional data from the commercial scale material and study
Yes, thanks for the questions. Let me start with the second part of the question first and go to the first. So on the second part of the question, we have documented 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-nineteen. In ways that ensure that we don't find ourselves with studies that are significantly delayed as a result of COVID 19.
So there's already guidance on that. And we're documenting any delayed visits in accordance with that. The second thing I should note is that there have not been a significant number of delays. That 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 reason to believe there's an effect on the powering of the study or on the readout or on the viability of the study or on the timing of the study.
So I just want to be very clear, in an abundance of the transparency, there may be a few instances of patients with out of window functional visits in light of the fact that 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. 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 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 Study 100 and 2 right now. And I'm giving my own team a lot of credit. I should give credit to 2 groups.
I'm certainly very proud of our clinical operations team and the like for the work they've done to minimize kind of disruption in study 1 or 2, but I should equally give an enormous amount of credit to Nationwide Children's Hospital and their operations team frankly, massive kudos to Doctor. Jerry Mendell, who's just been a hero in this process. Going to Study 301 or the broader group of studies Study 3, short answer on that is that we are proceeding right now, with the same approach that we had before COVID-nineteen. And we're executing along this pathway. So Study 301 will be four to seven year olds.
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 hold 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. With notwithstanding that, I do want to point out that we have to be realistic that they're that 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 GMQ material released on time. And as I 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 we had anticipated before COVID-nineteen.
The next issue is a choice. We want to make sure as we're tracking forward, that with respect to sites, they're not only ready to start taking patients. And if we get through all of the various processes and the IRB process in their lives, But then we're very confident that those will continue on into the fall and into the winter and into 2021 and beyond. And that may modestly delay us, but as I said 4, I really am talking about modest delays. It's still our goal to start the Study 301 and its Progyny in the second half of twenty twenty and But now, we feel confident that it's not going to be an issue.
Our next question comes
Thanks for taking my questions. I'm sorry if this question was already asked, but can you clarify for me for CD102 How often are the patients actually being monitored for the functional business? And specifically, I just wanted to get a sense for the biopsies that were scheduled to be taken for the patients in study 2. Do they have to be taken per protocol at a certain point of time after they were dosed or is there any flexibility in when the biopsies can be taken?
Well, I'm going to turn this over actually to Louise Rodino Klapac to respond to both of those questions.
There is a certain visit window for the biopsies. But as Doug mentioned, in terms of, flexibility of any out of window just has these been documented as per the FDA guidance. So, as we look at it, there's no particular delays or misses within the protocols that will be meaningful to the outcome of the study. Okay. And did you know what percent of patients were a little bit delayed in getting the biopsies?
No, I'm sorry. We are distant. For those who may wonder why there was an enormous delay. We are distant from one another and I actually insisted that I correct request. Apologies to that, Louise.
So Luis, I'm sorry. Did you answer the question?
No, there was just I don't know the exact number. Of visits that were delayed, but it's few.
Okay. Very small number.
Okay. And then as we think about Limb Girdle to follow-up for the pivotal study that you're going to start next year for the 2 EEG subgroup. Can you just remind us of how big of a population that is relative to the rest of LGMG?
It's a very rare it's a this is an ultra rare population. I don't think we've gone out and given the exact numbers as one of the things we've said, given the higher epidemiology and population of all of the patients that we're looking at. We're talking about a group of patients that are about percent of the size of Duchenne muscular dystrophy. But the Limb girdle 2E is a very rare form of Limb girdle and the very severe form of limb girdle. So we haven't nailed that, haven't nailed the size of the trial bound.
But obviously, with that in mind, when you consider the rarity of the disease, as you consider that this is really this is kind of in the perfect sweet spot of opportunity for a gene therapy This is a monogenic disease, well characterized, well understood. It is the lack of a structural protein. It is a single structural productivity that is causing all of the damage into generation and loss of life that comes with 2E. And the gene therapy that we, that we have created and that Luis has created, will will introduce a gene that codes for the actual native protein, the lack of which is targeted to buy. So 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 has the speed and leanness that one would imagine with the disease of this rarity well characterized monogenic and with the we can get very high expressions, then we'll all be there with our current dose, that returns to these patients the very protein, the lack of which is causing their, their disease.
Okay.
Just a reminder, everyone. One question
one of really questions.
Yes, 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 early period of time being looking for dystrophin. So I wouldn't commit in advance that we will even be looking for dystrophin at the 12 weeks. What we will be looking at is Exxon Skipping. And what you'll find with and of course, through Reminds, I'm not sure everyone knows this, but the Exxon Skipping is the very activity that results in the truncated form of of the, RNA that in turn creates the protein that is the slightly truncated, but otherwise fully functional district that we're trying to achieve.
And what we know, at least the animal, we know 2 things. Number 1, there is an obvious direct correlation between amount of exon skipping, as you wouldn't well imagine, an amount of dystrophin production. So looking at exon skipping is a perfect a brilliant marker for what kind of dystrophin we might find over time. I'll give you an example with respect to Golodirsen we had made some fairly bold statements about what we probably would see with Golodirsen versus the Teferson. And that was all because of what we had seen early days in the relative exon skipping that came from Golodirsen versus the Kepler.
And 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 skipping you might see from a PMO 12 weeks versus the amount of exon skipping that we will see with the PPMO at the doses that we're talking about right now, we don't have it yet. We'll see it in the second half and we'll all come together. We'll have data on tissue exposure and exon skipping and safety and the like and dosing levels. 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 we're now dosing through.
And I think at the inception of the single ascending dose and then the multi ascending dose study, when we started this, our animal models are mouse models and then our non human primate models got us to point that where we would be very happy with 6 rigs, but we would get increasingly happy up to about 12 mgs per kg, which is what we envisioned would be the ceiling and how high we could get. And certainly with respect to this peptide conjugated TMO, more is better with respect to dosing if we can get the higher doses safely. And the fact that we're at 20 mgs per kg right now, makes us very and 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 in any rational person that's going to complain about.
Thank you. Our next question comes from Alethia Young with Cantor Fitzgerald.
Hi. This is Emma on for OITIA. Can you give us any color on what the actual process requirements look like for a patient to transition to home infusion just what the percentage of patients are currently on home infusion for VIAONDYS and what those trends might look like next quarter?
Sure. I'm going to turn this over to Bo before I turn it over. Just to remind everyone, with respect to EXONDYS, as it stands today, the vast majority of patients are on home infusion. And while with respect to a launching product line, my on this, you will see a greater percentage of patients that start in hospital. It will over time be the same case that the vast majority of Bionys patients will become home infusion as well.
And of course, Beau can talk to you about the things that we're doing today to accelerate that process. May beau with that?
Yes. Thanks, Doug. And to Doug's point, the majority of our patients are already at home infusion. But for the patients that are being dosed in the hospital, it's really just a authorization like a reauthorization process with payers So it's just more of a paperwork process of transferring the authorization from in hospital infusion to home infusion. 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.
One other thing I will say on this topic is this there's a lot of process involved, both in the launch of Beyond and EXONDYS 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 themselves essentially advantage of the disruption that's occurring with COVID-nineteen, in ways that might profit them to the detriment of children. 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 the, the some of the obstacles that might be imposed as a result of COVID-nineteen to ensure that kids could stay on therapy. I'll give you just one example. With respect to state Medicaid, I am informed every single state, all 50 state Medicaid have been requested and have been granted by CMS, waivers 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 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-nineteen doesn't get in the way kids access to therapy. I really do think big savings are too much for that.
Our next question comes from Gena Wang with Barclays. Your line is now open. So there was an abstract 503 on cohort 1 data at the ASGCT PDF abstract. But somehow the final website doesn't have this app here anymore. Just want to double check the deal below.
They are at the ASGCT for limb girdle. And also given good safety so far, we're seeing from micro dystrophin program. Synchrony virtual program has the same factor and the promoter. Is it fair to say higher dose safety should be largely in line with micro dystrophin program?
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 Lynn Girdle, 2E, both expressions, safety, But your point is well taken, just so we're clear that factually 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, a high dose on limb girdle is, the same dosing level that the kids with Duchenne muscular dystrophy have received both the 101102, and together that is a significant number of patients that have been dosed. With that, I'm going to turn it over to Luis who might comment on the ASGCT and the verbal, but maybe give an overview of what, what kind of abstracts or posters we might
have at ASGCT, if you know?
Sure. Yes. In terms
of the clinical data, it will be we will release it later this quarter. So we will at ASGCT, there are 2 preclinical abstracts that will be presented as poster for limb girdle and that will be focused on, long term expression data and then also regression in in later stages of disease. This is all preclinical. And then of course, we'll have our symposium, where we'll outline the the background on the development of our constructs, and also talk about the micro dystrophin data, 9 months data. Thank
you.
Our next question comes from Matthew Baral with Cowen. Your line is now open.
Hey, guys. This is Tashant on for Ritu. A question on study 1 of 2. I'm wondering what some
of the assessments being outside of Brookdale Have
you had to revisit the statistical analysis plan? I just want
to just emphasize on that. Thanks.
We've look at it carefully and there's no adjustment necessary. We don't have a concern on the stats, particular analysis plan and broad strokes, on powering, we don't think there's any impacts 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. Doctor. O'Neal, if there's anything I missed in that, just for you to add to that or correct me if I
no corrections necessary at all, Doug. We have looked and, these out window assessments are not absolutely critical and have no impact on our statistical analysis plan. So we remain outside.
Thank you. Our next question comes from Joseph Schwartz with SVB Leerink. Your line is now open.
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 powered to detect the difference between the two arms? Copies literature suggesting that North Star can increase during the Sage range. So I'm just trying to think about what kind of an increase 9001 will have to produce in order to succeed?
Let me give you the broad stroke I'll give you a broad stroke answer on that. Some of the nuance I'm probably going to avoid simply for confidentiality on and competitive reasons. I'll give you the broadest stroke. The broadest stroke is the following. I suspect that no one has better patient level data on Duchenne muscular dystrophy, it's epidemiology, it's course then Sarepta.
Obviously, their leaders in Duchenne muscular dystrophy would have been close to the patient community and the data for EON 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 the study that we built here point in a nuanced way, what you see across four seven year olds in broad strokes is that, that there is the possibility for a couple of point potential linear. Not linearized. I apologize.
Nonlinearized. A couple of points increase potentially between the 4 5 year range, but you'll see them coming over the top and actually declining starting to significantly decline in the six and seven year old range. As you know, these kids are 4 to 7. All of that nuance understanding of the the course of this disease that and then used to perform the protocol that we beyond that, of
course, have a benefit of the first four patients
that we've had. So
we feel confident about the call. About borrowing.
It's really not the progress
that I wanted to.
Thank you. Our next question comes from Chris Sunrise from Nomura. Your line is now open.
Hey, good afternoon. Just about the path forward for limb girdle muscular dystrophy regulators and and the potential for using expression data. I was wondering just how much expression data, you might need to see there. And if FDA has any concerns or if you have any concerns about also, I suppose, just the sarcoglycan complex being sort of reformed by re expressing the protein and making sure that's intact. And if you are prepared to provide and equalization data in that regard if it's required.
And then just real quick with the PPMO, I mean, how much data will will really satisfy you guys with respect to safety at any given dose, call it 20 mgs per kg. What kind of timeframe are we looking at before, we can get confident that you can move forward, let's say, 20 or 50. Thank you.
Okay. So on as it relates to limb girdle, we're still working on the development and regulatory pathway itself. As you've anticipated, We, we certainly think that this 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 proved on a biomarker of expression. You raised a really interesting point about what about the reconstitution of the dystrophin associated protein complex?
And the fact that we think that is a very important, element to looking at this. And that's another important biomarker is there when you upper regulate as an example, beta sarcoglycan, do you see that the dystrophin associated protein complex itself also begins to up regulate so that, other proteins in that complex would start to come together and be expressed what 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, and we do think that's something important that we should show and be able to approve. And the good news is, by the way, we have seen that repeatedly that one of the things that should get someone very excited about This is that it's that is the native gene, the native protein already at our previous dose, very strong expression.
Properly localized in the right place. And to your good point, associated with the up regulations of the other proteins that make up dystrophin associated approach to companies. This is one example. You see a very strong correlate between upregulation of beta sarcoglycan and alpha alpha sarcoglycan, which these kids have a gene that's improperly 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. So all of I think all of the facts and circumstances around that are going to come into play with think about the development plan, what we think we should be able to avoid would be a lengthy trial that would require care, 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 see the land on the right answer. There are a lot of there are a lot of places to land between an accelerated approval on a biomarker, which would of course be not The answer we think is great. And on the other hand, a full blown placebo controlled trial, then we'll come back early next year
and provide an update to everyone. Thank
you. Our next question.
Before we go on, because I did promise it even though there were two questions. I will turn this over to Doctor. O'Neill to answer the question on the PPMO.
So To date, we are actually happy with the safety profile that we're seeing, and with the numbers in our, cohorts are ascending cohorts in about the ascending dose, we believe that we have sufficient numbers. I think we are able to judge safety by looking both at clinical outcomes but actually are obviously able to monitor the target talks, that was identified as non clinical talks. So we believe with those numbers, we can't comfortably make decisions. Obviously, way. Looking forward, safety is always a matter of numbers, but these numbers are, we believe, adequate for making that initial dose selection.
Thanks.
Our next question comes from Joel Beatty with Citi. Your line is now open.
Hi, this is Sean Egan calling in for Joel. Thank you for taking my questions. On the PPEMO, congratulations on the 20 mgkg dosing. Maybe can you comment or talk a little bit about any preclinical data or work you've done on micro dystrophin gene therapy plus a PTMO combo and what you have 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 both on 2 different One concept is the value of pretreatment with the PMO or PPMO, in advance of a gene therapy to enhance the comment at the value of those therapies and to enhance, expression. And as it relates to that, I can lean over and talk about literature that already exists. Doctor.
Voytes, as an example, has some literature where he in the mouse model pretreated with a, I believe, with a peptide conjugated PMO. And then, dose of gene therapy found enhanced session. That's one area we're looking at that area. The second one, of course, is a very significant one, which is to explore the benefit of a cognitive value of a PPMO on the one hand and gene therapy on the other. And you can envision a world in which you would get.
A synergistic value between a really significant expression from the gene therapy construct like our SRP-nine thousand and one coupled with robust express of a truncated dystrophin that you would get from a PPMO. We don't have a bunch 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 and clinical development as well as in advance of the success of our PPMO program as well. And I think a lot of people, their models our working assumption that gene therapy will entirely cannibalize the RNA plan form. And while I understand why some people look at the exciting aspects of our gene therapy and imagine that, I think it's probably early to believe that's the case.
And we have some cannibalizing research, but might say there is a value to these 2 therapies together.
Thank you. Our next question comes from Matthew Harrison with Morgan Stanley.
Thank you. This is Max Gore on for Matthew Harrison.
Sorry if
I missed this, but can you confirm that all micro dystrophin release assays have been built but 2 still need to be validated?
And are these the in vitro potency assays? Thank you.
Yes, great question. So yes, I can confirm that all of the assays are built. I can confirm it of 24 assays. All of them are either validated or qualified. Asset.
So all of the really significant ones have already been validated.
Thank you. Our next question comes from Vincent Chen with Bernstein. Your line is now open.
Just wanted to follow-up quickly
on your comments earlier on looking for exon skipping at 12 weeks, but not necessarily dystrophin. After you start getting soundscape dystrophin or in the case of gene therapy after you start expressing micro dystrophin 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?
Yes. So I'll give you, I'll give you the broad stroke and Doctor. Anil, if you have additional color on it. I mean, look, first of all, we've got to we definitely have to take RNA and micro dystrophin gene therapy and put them in 2 different buckets. They're very different.
With respect to micro dystrophin, we know the answer is 3 months. So what we've seen, as you know, with respect to SRP-nine thousand and one in our first four kids. We took biopsies after 3 months and those kids had 90% and higher dispute expression. So clearly, we get a really fast, onset for the gene therapy. For RNA, it is a slower process and it occurs over time, probably for a host of reasons.
But it occurs over time. In fact, we saw a significant difference over a long course period of time in both EXONDYS and with respect to VAYONDYS. I mean, we saw, I think, the one between 1 year 4 years, there was a significant difference the amount of dystrophin. And that may be in part because, when you with this chronic therapy, the chronic therapy keeps increasing the benefit over the long One of the reasons, frankly, during this difficult COVID crisis that we had so focused on ensuring that 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 lumpy of time.
But With that said, I'll turn this over to Doctor. O'Neill if you can provide some more color.
So Vincent, thanks for your question. The half life of wild type, your full length of stroke, is in the order of weeks. This, we know, And as Doug has said, the, not surprisingly, the time to transcribe as high fidelity and translates the high fidelity to scrofa, which is such a large gene, actually is a measurable in many hours. I think so those are important biological questions. Then from the point of view of looking at 12 week data, the key decision will be driven by the relative equivalencies of PPMO to PLO.
And, what we really what the robust data set that we have at that timeframe really, forces us or drives us to compare Exxon Skipping, which I think is actually a very valuable way of making the decisions about going forward and selecting doses. And as Doug has said, And as you can expect from what I just said about the half life of dystrophin and the, the synthetic timelines for the disclosure. One would expect to see ongoing accumulation over many weeks months. So that's the reason that we're actually focusing on the Exxon skipping at 12 weeks. And remember also, we are measuring tissue levels of the PPMO and would be able to compare that with PMO as well.
So that really enables us to really test multiple elements of the hypothesis.
I understand this about let's talk a moment about what we know about our RNA technology to PMO generally. So the things about our PMO that make it that make it so clever is that number 1, it's precise that we know if we get the BMO to the right place. It induces ex hunt skipping, it transforms messenger RNA. It puts it back in frame and it makes dystrophin. We know that.
We know it's safe, right? The PMO we can dose a very high level that had a very significant safety window and when it's therapeutic window. The one limitation and it is a significant limitation of our PMOs is that as a neutrally charged molecule, it doesn't get into the cell well. And as a result of that, a lot of the PMO that is infused, it doesn't get into the cells and it can utilize. It's urinated now.
So the goal of all of this, the unlocking of our RNA technology and a potential profound improvement of the RNA technology comes from something that is It is simple to discuss, frustratingly simple and difficult to execute, which is, can you find a delivery via that can safely get the PMO in the cells in much greater exposure. That's the question. In animal models, the PMO does very mechanically. It's a positively charged peptide. It interacts with the negatively charged elements on the outside of the cell.
It creates wobbling. It uses vesicles to get them to sell in great abundance, an order of magnitude greater than you would see with the PMO. And so that's the biggest issue for us right now. Can we get to strong therapeutic doses, what we envision to be therapeutic doses of the PPMO without inducing a safety signal. And if we can, I think mechanically, we're going to be very excited about the potential in the PPMO?
And in that regard, we need to see this data and we'll have a data readout in the second second half of twenty twenty. The exciting thing about where we are right now is that we're a clean mix per kid when we envisioned at least some time ago. That our ceiling was going to be about 12 minutes per day.
Thank you.
Our next question comes from Anupam Mama with JP Morgan. Your line is now open.
Hi, all. This is Tessa on the call today Anupam. Hope you're all well, and thanks for taking our question. I think in the prepared remarks, Doug, you mentioned that you do not yet have clarity to provide updated guidance for EXONDYS. But maybe from a high level, what were the levers for growth from $31,000,000 in 20.19 18?
And maybe how are you thinking about the push and pull leverage for the guidance to consider with the ongoing, COVID-nineteen pandemic? Thanks so much.
Yes, I'll give you the broad strokes. So our growth with respect to saw on this. Doesn't relate to price. Let's make sure we're clear about that. So there's only one lever and it's patients on therapy, staying on therapy, and we're finding more patients and more patients coming on therapy.
That is it because, as I hopefully people know, and as I think I've said in other forums many times, we launched, EXONDYS in 2016. We have taken no price increases over the period of time, and we don't envision taking a price increase. Our goal is to grow through serving this community and having more kids benefit from our therapies. And as an example, with respect to Vionic we priced it at parity with EXONDYS. We didn't obviously take a price increase there.
So when we look forward, that's the lever. And the the, the risk to the revenue is modest for the second half of the year, certainly with respect to EXONDYS, because with respect to your Saunders, the great majority of kids are already at 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 that the peak of this crisis to get into a hospital to get fusion if you're in hospital and the good news is most of the kids are not. So when I talk about EXONDYS, in particular, we're talking about it.
Based on everything we see today, as I said, I am not giving up any guidance 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 short term impact. We'll see a little more significant delay and impact on, by August, for the simple reason, that we're in launch phase, if not, that a more significant number of patients will start in the hospital and 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, land issue. So broadly speaking, I think we're good shape. We're serving the basic community.
We're trying our very best. And I think we're so far succeeding in keeping us safe. Any impact on revenue ought to be really, quite modestly, quite shortly. But with that, though, am I missed anything?
No, Doug, you covered everything. Thank you.
Thank you. Our next question comes from Tim Lugo with William Blair. Your line is now open.
Thanks for taking the question. For the commercial supply trial, have the ongoing issues on COVID impact 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?
Thanks for the question. Now the study remains on pace and on track. As originally envisioned both the size of the study, the speculative multi country, multi multi institute, the fact that it's placebo controlled, etcetera. So the design hasn't changed on that study. 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.
You've got to get lip. In a perfect world, you have in person site initiation visits from our clinical operations in the light, we want to make sure everyone's properly, trained 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 we're being thoughtful and cautious about that the COVID-nineteen as we're starting, that means there'll 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 of months about measured in significant amounts of time. But beyond that, things are proceeding and we haven't made any significant changes to the approach that we're taking.
Thank you. Our next question comes from Lisa Baker with JMP Securities. Your line is now open. Lisa, if your line is muted, please unmute.
Thanks for taking the questions and congratulations on the strong quarter. Just wanted to know if you could give us a little bit more details on the and color from quarter itself in terms of how many patients actually started on VYONDIS, just so we can better understand the dynamics there? And then also Any changes in gross to nets or any of those kind of details that we should be thinking about, 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? Thank you.
Sure.
I will turn this question over to Beau, although I think some of the new ones probably not at liberty to disclose.
Yes, Lisa, obviously, we're not going to get we never got into patient numbers for EXONDYS. We want with Biondas. And, but I will tell you, even though Biondas is obviously smaller than EXONDis from a population standpoint, We were very pleased with the progress that we were making with payers and coverage. We were, we were much farther ahead than we were with EXONDYS. We have 148,000,000 lives that are covered on feeder, restricted policy or two labels.
So we were very pleased with that. And the launch was going to our expectations. So It's just the small hiccup of the pandemic that put a little things, put some punches in but we were overall very, very happy.
Thank you. I'm not showing any further questions at this time. I would now like to turn the call back over to Doug Ingram for closing remarks.
All right. Thank you. I'll be brief. Thank you all very much for joining us for our earnings call. Hopefully, you'll have seen here that like so many other companies, I mean, frankly, individuals are not just in the United States but around the world, this pandemic has, caused enormous numbers, challenges and obstacles for the Sarepta team.
But as I've 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 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 that 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 working from home. So there is a lot of learning in this, probably not just disruptive 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 on 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 loved ones and about themselves, they have not lost sight of the fact that Sarepta is on mission. Can I continue to execute across 2020? I'm going to be excited to give you additional updates across the year. We can give you better clarity on 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 enormous tonsequential 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. Let's get this crisis behind us.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.