Q2 2023 Sarepta Therapeutics Inc Earnings Call
Okay.
Good afternoon.
And welcome to this wrapped up therapeutics second quarter 2023 earnings call. At this time all participants are in a listen only mode. After the speaker's presentation. There will be a question and answer session to ask a question. During this session you'll need to press star one on your telephone.
Question has been answered and you'd like to remove yourself from the queue simply press Star One again as a reminder, today's program is being recorded at this time I'll turn the call over to Francesca Nolan Executive Director Investor Relations and corporate Communications. Please go ahead.
Thank you Jonathan and thank you all for joining today's call.
Earlier. This afternoon, we released our financial results for the second quarter 2023. The press releases are available on our website at <unk> Dot Com and our 10-Q was filed with the Securities and Exchange Commission. This afternoon.
Joining us on the call today are Doug Ingram, E&S, Japan, Devlin, Murray and Dr. Louise Rodino <unk>.
After our formal remarks, we'll open the call for Q&A I'd like to note that during this call we will be making a number of forward looking statements. Please take a moment to review our slide on the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties many of which are beyond <unk> control.
Actual results could materially differ from these forward looking statements and any such risks can materially and adversely affect the business the results of operations and trading prices for <unk> common stock for.
For a detailed description of applicable risks and uncertainties. We encourage you to review the company's most recent quarterly report on Form 10-Q filed with the SEC as long as the company's other SEC filings. The company does not undertake any obligation to publicly update it forward looking statements, including any financial projections provided today based on something.
When events or circumstances.
And now I will turn the call over to our President and CEO , Doug Ingram, who will.
Find an overview of our recent progress.
Thank you Fred and good afternoon, and thank you for joining our second quarter 2023 financial results Conference call.
The second quarter of this year was perhaps the most consequential period and the long consequential history of Suraj.
For families living with Duchenne it was far more than that still.
Buddy Cassidy living with Duchenne and then elaborate as Advisory Committee member shared the collective view of the Duchenne community on the June approval in words far more eloquent than I could possibly muster.
He said and I quote there is more to do but now I'll, let us pause and bask in the glow of our achievement, let us basket and gratitude.
Let us pause and celebration, let US watch it's gone rises and brings in the day close quote in.
In may of this year. The FDA held an advisory committee meeting on our gene therapy SRP 9001, now called the Lumpiness with a majority vote. The Advisory Committee recommended the approval of <unk> for the treatment of ambulatory patients with Duchenne muscular dystrophy.
June 22, the FDA granted accelerated approval for <unk> to treat Duchenne muscular dystrophy currently labeled for four and five year old patients at the time of the approval FDA leadership informed throughout the and the patient community.
Our confirmatory trial embark meets its objectives. The F D. A will in the words of Dr. Peter marks move with maximum speed following submission of the data to the agency minimizing any impediments to review results rapidly.
Broadening the label by removing any age restrictions.
So we have our path.
Our confirmatory trial is well designed and powered to show a statistically significant benefit in the study population for the avoidance of doubt we have powered this study to show a benefit in the studied population that's four to seven years old and with that we will have confirmed the mechanism of action in duchenne patients applicable.
Two all age ranges.
We will have the top line for embark in the fourth quarter of this year, we will announce top line results as we submit them to the division followed shortly by a label supplement if successful we should be able to expand our label in the first half of 'twenty 'twenty four to include the majority of Duchenne patients. Additionally, we.
We have already commenced our non ambulatory trial envision or study 303, and we intend to commence two trials with alternative approaches to cleaving or clearing preexisting antibodies. Our goal is to expand our label for elaborate as to cover as much as 95% of the Duchenne patients.
In a moment, our chief customer officer, Alan Murray will provide details on the early days of the 11th. This watch 11. This is our fourth approved Duchenne therapy, and we have been very successful with all of our prior launches.
Consistent with our track record the 11th this launch is going well a significant number of sites are now initiated and ready to infuse payer discussions and negotiations have been more positive than our prior successful therapies and there is a significant amount of enthusiasm from physicians and families as we have a sub.
Stansell number of start forms and more coming weekly in fact, I am pleased to announce that the first reimbursed elaborate its infusion occurred earlier today that is faster than we projected and speaks to the team's ability to execute.
Now it's positive actually this is please bear in mind that the significant ramping infusions will begin later this year.
Primarily due to payer logistics answered the release protocol for <unk>, which requires that both we and the FDA released each a lot.
Shortly our head of research and development and Chief Scientific Officer, Dr. Louise Rodino playback will provide an overview of our development activities, including progress with our next generation P. P. M. O S. R. P 50, 51 and on the gene therapy side with our L. G. M D portfolio to remind you in.
In addition to <unk>, we have a deep pipeline and are advancing a number of therapies in 2023. In fact, we have 24 ongoing human clinical trials by the end of this year and we continue to advance our next generation viral capsid mile AAV, which in early animal models.
It looks to be as much as 10 fold more tropic than current AAV is.
From a manufacturing perspective, our technical operations professionals more than 400, and all our focus with our partner capital and on producing and releasing lots to support our watch.
Looking to the mid term we will also continue to work to establish comparability at our Thermo Fisher plant for 11 years, and then looking to the future. We are advancing our approach to manufacturing to support our entire pipeline. In fact, we have made great progress in next generation suspension technology, which we will use across our gene.
Therapy portfolio, including the L. G M D programs and even potentially for <unk>.
In fact, we have largely completed our.
Our suspension based process development for <unk> and we have made three runs at 250 leaders with a fourth run to occur later this year with the goal of scaling to 1000 leaders in the near term we have seen yields that are multiples greater than the current standard as the leader in gene therapy for rare diseases.
We'll continue to focus on improving the science and the delivery of our therapies.
Moving finally to quarterly performance, we've had another strong quarter with our three approved PMO exon, there's a modest and by Anders I am pleased to announce that second quarter total revenue came in at $261.2 million in net product revenue from our PMO was came in at $239 million.
Exceeding internal estimates and analysts' consensus.
The second quarter, we saw no negative impact on the approved P. M OS for many warehousing related to the 11th its approval and do not anticipate substantial impact over the course of the year, we remain comfortable with our full year guidance of $925 million for total PMO net product revenue with our current bias.
To modestly exceeding that guidance. It is too early in the launch to provide accurate guidance for <unk>, but we will continue to evaluate that.
At <unk>, we have a distinct culture. It is easy to say one is patient focused but our words are backed by action, we put the welfare of our patients above all other considerations and when necessary.
We fight for them and the tools we use for that fight include are glued our commitment to scientific excellence.
And the courage to represent the patients who depend upon us.
It may not always be easy, but it is paying off we have four approved therapies and a wealth of potentially life enhancing programs in our pipeline.
We will achieve $925 million on the first three of our four approved therapies this year and far more next year. When our newest approval is considered we have a near term high probability of success plan to broaden the label of the first and only gene therapy for Duchenne.
And if our plans are successful we can be profitable in the next few quarters, we have done a lot already but with our plans our pipeline and our commitment.
It's a dogged execution, we have only just begun and with that I will turn the call over to Dr. Rodino playback, who will provide an update on our research and development progress.
Yeah.
Yeah.
Thanks, Doug.
The accomplishments within R&D over the last quarter are many and highlighted by the approval of a lot of it is.
On June 22nd 2023.
A prolonged all of it has to be the accelerated approval pathway represents a win for the Duchenne community and an important step towards the broad approval for individuals living with duchenne, regardless of age or ambulatory status.
As we look forward to the weeks and months ahead, we remain firmly committed to our value to follow the science and present objective evidence that supports a lot of it is just the ability to change the trajectory of Duchenne muscular dystrophy.
Since 2018 and across multiple studies with us the largest number of dish and patients more than any other gene therapy in development for this debate.
Mind, you we've shown consistent results across three clinical studies with respect to both biomarker expression and functional result.
In clinical trials.
Evidence demonstrated positive results at multiple time points, including one two and four years. After treatment. In addition to a consistent safety profile.
The BLA for <unk> concluded efficacy and safety data from studies 1011, or two in 103 or in Denver as well as an integrated analysis across these three clinical study comparing functional results two propensity score matched external control.
Importantly, the functional data reinforced the consistency of NSA improvement across these three independent trials.
And xiaomi improvements across key secondary functional endpoints, such as time to rise and 10 meter walk run.
The data from studies 101102, and 103 cohort, one which is aged four to seven have now than either published or accepted for publication in peer reviewed journals.
When compared to the appropriate control populations. All evidence has consistently shown a treatment effect as measured by change in NSA a score at one year, we have applied the learnings from studies 1012, and three and the design execution and statistical analysis plan for a phase III embark study.
To remind you embark is a double blind randomized placebo controlled trial of <unk> in duchenne patients aged four to seven we.
We completed enrollment in the fall of 2022.
The trial will be considered successful if the entire treated population shows a statistically significant positive difference, let's say eight quarters at one year compared to placebo.
Our conviction around embark is founded in data that was designed based on our experience with studies 1012, and three along with our propensity weighted external control and natural history data sources.
Took extensive measures to ensure success which include the following.
The embark study design improves studied 102 with measures to increase how much of your navy, including a floor and a ceiling on baseline NFA and requirements for rice in Florida to be less than five seconds at screening.
We also insurance insert a balanced distribution between treatment arms by including a stratification factor for baseline score.
We also increased the target sample size to 120 to increase power and enrolled 125 patients.
All of these measures resulted in a study powered well over 90%.
As discussed at the Advisory Committee in May what I feel.
Better deviation of three five points and 120 patients and park with power greater than 90% to see a 2.2 point difference.
And then a fad.
We've always been confident in the powering of the study with.
It was also done extensive analysis and modeling that provides additional confidence in the successful in park.
First when the integrated summary of efficacy population included in the BLA, which is an N of 52 is narrowed to those who meet 301 entry criteria for an N of 33, the standard deviation goes down.
Firming increased TV 80.
We also performed simulation models of four scenarios drawn from existing program data that if all yielded greater than 90% power.
Given the entire protocol, we're able to detect an even lower treatment effect.
For example, with an assumed the standard deviation of three five points our analysis what protects statistics.
<unk> observed treatment effects in the overall intent to treat population as low as one three points.
Taken together, we have strong confidence in the part one readout of embark.
With regards to additional studies, we've commenced dosing in envision our placebo controlled phase III study in non ambulant in older patients with Duchenne.
We're also on track to convince in a freezer study as well as an <unk> study and AZ are aged 74 antibody positive patients this year.
This is all with the goal of serving the entire edition population.
Now moving to limb girdle, muscular dystrophy, where L. G M D.
We remain committed to advancing our LG and b portfolio across a variety of subtypes.
Look forward to providing updates on these important programs in the months ahead.
We're pleased to report that we have fully enrolled journey our L. P. M. D. Natural history study 102006 patients with buckle black and apathy have been enrolled and will be followed for 36 months.
We've also made excellent progress enrolling voyaging a phase one study evaluating SRP nine zero growth rate for the treatment of limb girdle muscular dystrophy type QE in ambulant.
Adult patients and non ambulant patients using clinical process SRP 9003 material.
Combined with positive expression and functional data shared from our initial study SRP 9003101, we believe the data from voyaging will give us insights into a broader patient population.
Our next milestones for voyaging include completing enrollment in Q3 of this year and initiating our phase III study in you think commercially representative process material later in the year.
Finally, we commenced dosing of a systemic pilot study now of a gene.
A S. R. P 6004 dual vector our age 74 mediated gene therapy to treat L. G. M D to be characterized by the absence of the protein just feeling the innovative dual vector strategy allows us to deliver the full let's just throw them gene the sole cause of L. G. M. D T V.
We also continue to make progress in manufacturing for all LTE M. D candidates in our pipeline and look forward to initiating clinical studies as rapidly as possible.
Turning now to the progress we've made with our RNA platform.
We were pleased to complete enrollment in the first quarter of 2023 for a momentum study for SRP 50 51.
And on track to announce data from this study in the back half of 2023.
In regards to our post marketing studies for the P&L as mentioned last quarter, we completed enrollment in the essence trial, our post marketing requirement for gold Harrison and CASM are set and continue to make good progress with our mission study, which is on track to be fully enrolled this year.
The accomplishments of 2023 and the opportunities before us speak to the promise of science to fundamentally impact and change the lives of patients around the world.
On the research side, we continue to make excellent progress on the bio E platform, along with an exciting pipeline of genetic medicine candidates.
Deepest gratitude to our R&D colleagues across RNA gene therapy, and gene editing for their extraordinary work to get us where we are today.
Finally to the patient community, we understand the urgency and we're working tirelessly to bring forth transformative genetic medicines as rapidly as science will allow we are doing everything we can to expedite the embark study readout that we can expand the 11th playbook. We expect the data will be available in the next three or four months.
I will now turn the call over to Don for an update on our commercial activities gallon.
Thank you Luis and good afternoon.
We were thrilled to receive approval for <unk> on June 22nd and we owe a tremendous debt of gratitude to the patients who participated in the trials the kols and our R&D colleagues for getting us to this point as Doug and Louise have said, we are deeply committed to broadening the label at the earliest time point that is feasible.
During this time the team is diligently working day and night to expedite access to this potentially transformative therapy for all eligible four to five year old Duchenne patients.
Their dedication is focused on ensuring swift and seamless access for those who can benefit from <unk>.
In these early stages much of our focus is on supporting those sites, who are boys turning six years old.
At the same time, we are also rapidly preparing the sites to treat all of the patients who are eligible today and laying the groundwork to be ready for a broader patient population when the time comes.
The team's remarkable progress and unwavering sense of urgency in executing our now for Duchenne launches are truly commendable and reflect our commitment to providing timely access to potentially transformative patients' therapies for patients the.
The commercial and medical teams began educating the centers within 24 hours of approval as we've done in our previous three launches right from day, one our case managers were fully prepared to assist patients in navigating the interested aspects of the gene therapy treatment journeys.
And finally to touch on antibody testing over 700 kits, where in the hands of our key sites within a day or two of approval testing is currently underway in the process is working smoothly.
We've seen very strong demand for <unk> and are encouraged by the discussions with Kols payors and the broader community.
We began receiving enrollment forms within hours of approval and we continued to see them come in on a daily basis.
Since June 22nd the team has had over 500 interactions with both treating and referring sites.
In these early discussions our focus has been first and foremost on patient safety.
Eligibility procurement dosing in preparation and reimbursement.
Launching the first gene therapy for Duchenne patients requires a multifaceted approach with a high level of communication not only with Hcp's insight, but also patients' families and payers to ensure patients have timely access to this groundbreaking therapy.
As a result of our preparation and diligent efforts. We're now at the point, where patients can begin receiving <unk> with confidence.
As I previously highlighted our engagement with Payors has been constructive the team is optimistic and making progress towards our goal of ensuring timely access.
Of note, we are seeing states reach out to their local kols and experts to a foreign policies. We expect to have pair policies in place within three to six months, depending on the plan.
In the interim we are working with Payors on a case by case basis.
To obtain access for patients to that end, we have received multiple authorizations and as Doug mentioned, we were thrilled to announce that the first patient was dose today.
Over the course of the next week and a half we have a handful of additional infusions across the country scheduled with patients representing a combination of commercial.
And Medicaid insurance coverage.
We are gratified by the tremendous work being done across the country to support patients in particular, there has been a concerted effort since approval to rally around those patients who are approaching their sixth birthday.
The incredible efforts to support those patients from all involved has it been truly humbling.
While we are encouraged by our progress and accomplishments to date is important to reiterate that we are not providing revenue guidance in the early stages of this one of a kind launch.
As Doug me clear on the approval call, we expect us to take some time in the early stages before dosing can begin in earnest to generate the launch ramp we are confident will come.
I want to take a moment prior to reflecting on our PMO business to think everyone within <unk> and our key partners, who have worked so hard to make this launch a reality and allowed us to be ready to serve these patients right from day one.
Now regarding our PMO business, it's critical to highlight the fact that we have not lost a step supporting our PMO patient community, while we've risen to the challenge of the first two Shen gene therapy launch a.
Doug mentioned, we delivered $239 million in net product revenue in the second quarter, representing $13, 1% growth over the second quarter of 2022.
<unk> 51 totaled $134.7 million, representing $6, 6% growth over Q2 of 22 four by on just 53 sales were $32.6 million growing roughly 8.2% over the second quarter of 2022.
And for a modest 45 sales totaled $71.7 million.
Representing more than 31% growth versus Q2 of 2022.
Total X U S net product revenue in the second quarter was roughly $35 million. This represented an increase over the prior quarter, which was expected and fully reflected in our annual guidance and forecast.
As discussed on last quarter's call, we expect to see even more quarter to quarter fluctuations in the overall net product revenue is the X U S becomes a bigger percentage of the overall PMO mix.
Importantly, these quarter to quarter fluctuations along with the shifting mix of the TMO revenue base have all been anticipated.
And fully reflected in our annual guidance overall, the fundamentals of the PMO business coming out of Q2 are completely in line with what we expected at this point in the year and we reiterate our full year guidance of $925 million in net product revenue for our Pmo's therapies.
In closing our teams are fully engaged in executing today to support patients with all four of our approved therapies I'm, particularly pleased.
To see the continued success of our PMO business in the second quarter.
In fact looking back on the execution of all three of our PMO launches should provide a high level of confidence in the future of <unk>.
And our potential to transform the duchenne space with a <unk> and paved the way for precision genetic medicines for other rare patient populations, such as limb girdle muscular dystrophy and with that I'll turn the call over to E&S dependent for an update on our financials Ian.
Good afternoon, everyone.
Afternoons financial results press release provided detailed for the second quarter of 2023 on a non-GAAP basis as well we got Nathan Please affront in a press release available on his website for full reconciliation a gap to non-GAAP financial results.
So the three months ended June 30th 2023, the company recorded a total revenues of $261.2 million, which convinced them that product revenues in collaboration revenues compared to revenues of $233.5 million for the same period coin 22, an increase of $27.7 million.
Neck Potok revenue from the second corner of 20th 23 from our <unk> with $239 million compared to $211.2 million for the same period of 2022, the increasing that product revenue primarily reflect increasing demand for our T M O products and.
Each of Accordant ended June 30th <unk>, we recognise $22.3 million, a collaboration revenue, which relates to our collaboration arrangement with Roche the reimbursement I'll call development caught under the Roshi agreement totaled $28.2 million for the second quarter client 23, compared to 26.4 million.
For the same period of 2022.
On a gap basis, we reported a net loss of $23.9 million or 27 cents per basic and thyroid, sir and $231.5 million or $2.65 per basic and diluted Sir but a second quarter of 2023 and 20.2, respectively. We.
We reported it non-GAAP net loss of $75.2 million or 85 cents per day, and diluted chair and a second quarter of 2023 compared to a non-GAAP net loss of $103 million or one dollar and 18th cents per bathing in February chair in the second quarter of 2022.
And the second corner clients nine three we recorded approximately $34.1 million <unk> compared to $37.8 million in the same period of 2022.
A decrease in cause micelle, primarily reflect a decrease in a royalty payments during the three months ended June 30th 2023 due to changes in the bomber and royalty times and a decrease in right office or in batches of our product not meeting are are the specifications for the three months ended June 30th 2023 as compared to the same period.
2022, partially offset by an increasing demand from our per our camo products.
On a gap basis, we recorded $241.9 million and $252.3 million and R&D expenses for the second quarter of 2023, and 2022, respectively, a year over year decrease of $10.4 million.
Decreases primarily due to a decrease in manufacturing expenses, partially offset by increasing clinical trial expensive compensation and other personnel expenses.
And on that basis, R&D expenses were $212.2 million for the second quarter of 2023 compared to $230.4 million for the same period of coin 22, a decrease in $18.2 million.
During the SG&A on a gap basis, we recorded approximately 118.6 million dollar and $154.3 million of expenses, but a second quarters of 2023, and 2022, respectively, a decrease of $35.7 million.
The decrease was driven primarily by a decrease in fact based compensation expense.
On a non-GAAP basis, SG&A expenses were $90.3 million for the second quarter of 2023 compared to $63.7 million for the same period of 2022, an increase of $26.6 million.
On a gap atheism, you're required to $16.9 million and other income net for the second quarter of 2023 compared to $17 million. Another expense that for the same period 2022, the changes primarily due to an increase in interest income and accretion of investment discount give your investment mix of our investment portfolio as.
Well the reduction of interest expense incurred as a result of the repayment of December 20th 19 term loan during 2022.
In the second quarter, we entered into an agreement to sell the rare pediatric disease. Prior to review voucher received from the F. D. A in connection with the approval of a <unk> or a consideration of $102 million with no Commission cough. The net proceeds are recorded at the game from the sale of the P. R V. As 11th did not have a carrying valley.
<unk> at the time of the sale.
And finally, we add approximately $1.9 billion in cash cash equivalent investment and longterm restricted cash as of June 30th 2023. So in closing I'd just like to reiterate how exciting at time at his first rap then notations <unk> being the first patient dose today <unk> joy to all of our Hearts and.
And then from a financial perspective, we're looking forward to being one of the rare biotech company to actually make the transition to profitability. In fact, we anticipate becoming non-GAAP E. T F positive in the upcoming quarters.
Cause it has truly been quite an accomplishment on all fronts fries and were particularly proud of what we've been we've been able to do.
And with that I'll turn the call over back to Doug I'm, sorry for <unk>.
Thank you very much in adhere to those last comments, Jonathan let's open the call for questions.
Certainly and ladies and gentlemen, we ask that you. Please limit yourselves to one question. Each you may get back in the queue. As time allows one moment for our first question. Our first question comes from the line of calling <unk> from UBS. Your question. Please.
Hey, good afternoon, and congrats on another mall today with the the first patient taste.
I guess, that's what a two part question or around the the covenant tomato or onion bock hitting with as low as a 1.3 point Delta I'm curious what is the <unk>. The the 1.3 Delta is.
And as if he didn't discussion with F. D. A whether this would be considered clinically meaningful and then just as I said a follow up to that I think the most common question. We're getting is that it's in bulk misses the primary and the iPhone population, but shows a directional benefit in four to five year old given this name multiplicity strategy is F D. A given any indicate.
Whether this would be sufficient to maintain the approval status. Thank you.
So I'm Gonna turn the question over to Luis on the towering uhm issue, but real quickly just.
On the ladder questions that the so we haven't had discussions with the agency directly about clinically meaningful Miss of a particular number but I want to remind you that vehicle that one sees at 52 weeks isn't the effect, it's a signal of the longterm.
<unk> you know as a using Ah.
Inartful metaphor, if we took off in from Los Angeles, and we were one degree off trying to get to Tokyo by the time, we get Catalina Island, we wouldn't be that far off but if we continue with that one degree we'd be five 600 700 miles away from Tokyo, That's what we're dealing with here. This is a disease that.
Is degenerate over degenerative over time, if we can see is statistically significant benefit and a meter 52 weeks, we have clearly changed the trajectory of this disease in a very positive way. So with that said, let me turn the the technical question on the power and calculations over to Doctor.
Video playback.
Thanks <unk>.
So based on the modeling that we did that says that a traditional power calculation. So this was a simulation model in order to determine the lowest that's exercise that we can see in order to <unk>.
Can we put the following assumptions, that's our motto, which wasn't 125 patients such as the number of patients we enrolled no dropouts, because we've not seen any dropouts and embark.
We assumed a standard deviation of 3.5 across the entire four to seven year old population and then we varied the effect size in order to see how long we can <unk> <unk> statistical significance and that's how we arrived at the equal to or greater than 1.3 as the lowest number this isn't.
<unk>. This is as low as the modeling will shell S <unk> statistical significance.
And one one final thing.
Call and you have to ask about the way the agency would look at the data again were we our power for success and embark in our goal is deceased statistically significant meaningful benefit from the therapy and 52 weeks at the end of the the day the agency's standard as to look at the totality of evidence across to pry.
Mary and the secondary so.
Sort of statistical significance, we'd look into that presumably they look at the totality of evidence to justify the mechanism of action and expand the label to the broader populations.
Thank you one moment for our next question.
And our next question comes from the line O'brien Abrahams from RBC capital markets. Your question. Please.
Hey, guys. Good afternoon, and thanks for taking my question. So in the back half of this year I'm going to see data from several late stage D. N D gene therapies Uhm I guess I'm curious based on what you're seeing on the ground. So far if you have a sense as to what physicians and patients are going to be looking for is a potentially choose.
<unk>, a gene therapy to to take assuming there's multiple therapy available and what are your views on the potential approval pads based on interim interim functional analysis is that something that you guys ever explored with the contemplate your explore with the F D. A.
We are not we are we if there's one therapy that's approved for Duchenne muscular dystrophy, that's a gene therapy.
And to be honest, we think very little about other.
<unk> right now we have.
A pass in front of US we have to serve the patients that are available to benefit from our therapy today, and we need to broaden that label and that's what we're focused on right now and of the things that we worry about right now those are the the you know all of the top priorities and worry about other organizations and.
Their plans is is to assure you are less than tertiary does.
Thank you one moment for our next question.
And our next question comes from the line that <unk> from Bank of America. Your question. Please.
Hi, guys. Good afternoon. Thanks for taking my question Uhm, I was hoping to get a little bit more detail on that one patient that was justice today Uhm, maybe if you can share at the age of the patient and also how long it took from the time the doctor identified the patient it's as appropriate for the draft here today and.
Expect that timeline to be representative on a go forward basis at least for the next few months for uptake. Thanks.
So I'm the patient and the patient I believe this was I would normally not answered. This question because of patient privacy, but I think that's already add on social media. The patient was one day away from their sixth birthday. So <unk> as you can appreciate there was an enormous amount of work not merely on our teams sake, but <unk>.
Glee on the physician and their staffs part to ensure that this patient didn't age out and frankly kudos to the payer for prioritizing. This to ensure this patient received access which I think speaks it speaks to the executed the team it speaks to the passion and ability of the.
The physicians that we're working with and and.
Frankly, it speaks to the willingness of payers to provide access and to be thoughtful about patients, which I think is a wonderful answer for all of US looking forward you know as I've said before and I think Darwin reiterated in his remarks, you should assume that the significant ramp in this therapy is going to occur.
<unk> later this year there are obvious administrative reasons for that payer's policies and the like up to get completed and then administratively we have to release product and that's a dual release process right now we release it and then the F D. A release it it releases it as well, but as Dallas noted.
We do have you know a.
A handful of patients over the next literally coming few days.
That are currently scheduled for infusion, but but I really wanted to reiterate you should look out to really see that ramp at infusions later this year.
[noise]. Thank you one moment for our next question.
And our next question comes from the line of <unk> from Barclays. Your question. Please.
Thank you I would also ask questions.
Questions. So based on your initial interactions are the hospitals, how may I have the.
Patients approaching 60, or so and what's strategy you have tried cheetos patient before past policy. Please.
Matthew.
For the pain of policy. Please.
I I apologize I didn't I didn't I missed the last part of your question I'm Gonna turn this over to balance to answer, but you'll see that the strategy, which is not our own strategy alone, but it is a shared strategy with these very passionate physicians I'm working in.
<unk> with Payors is to prioritize children and ensure they can get access if at all possible before they age out of the label and you saw that poignantly today with a boy who received access today, one day before six birthday, but.
Down and do you have any other thoughts on that yeah <unk>. Thank you for the question in the short term we've been focused on on those boys that are that are turning six in the in the next while we as Doug said and the earnings call. We there's an incident population into shed or a 400 page.
<unk> born each year no not all of those patients are diagnosed by the age of their their sixth birthday.
There's actually a pretty.
Good percentage that don't get diagnosed by their six birthday, but there is a you know a good number of patients sitting in in the sites that are that are turning six at a big focus in terms of our execution as as Doug also said and as we said before we are we are dieting around patient numbers, we're gonna.
We're going to focus on net revenue as we've done with the other launches.
Thank you one moment for our next question.
And our next question comes from the line is helping Richter from Goldman Sachs. Your question. Please.
Okay.
Thanks for taking my question and congrats as well and I'm treating the first patient here Uhm with regard to the 3.5 standard deviation assumption you know recognize that that's what you saw I believe in and study 103 now I guess is there anything else are you using to support that assumption and and you know in the context.
Heterogeneity and and even with trying to vacation.
<unk>, yeah, how you're thinking you might be impact here, if it's higher thank you.
Well actually one would actually presume potentially the opposite so the 3.5 standard deviation was the original powering for embark.
That consider the historical trials, but didn't take into consideration the.
Various tightening of the protocol that occurred with respect to <unk> pier, one or embark to reduce had originated and make create more homogeneity. You'll recall, we have a very strong ceiling and floor in that trial, you'll recall that we have <unk>.
<unk> not only on the basis of N S. A basslines, but also an age 45 or six to seven year old range, you'll recall that we have restricted all of the patients to rise time under five seconds all of that should should theoretically at least.
Reduced heterogeneity create more homogeneity across the population and reduced standard deviation, but the 3.5 points that Luis mentioned was considered before those it didn't take those indicators impatient and indeed when.
<unk> get their modeling.
Change the standard deviation, so they didn't actually I'm gonna need that for for for their modeling purposes. So we feel very good about where we are right now and then remind you. We the study was powered off of 120 patients with a presumption of some <unk>.
Drop outs, we sit here today. The study was over enrolled it's now 125 patients and we've had absolutely zero dropouts and frankly don't anticipate any drop outs before the last patient last visit which will occur in mid September .
So we feel very good about where we are right now.
Thank you one moment for our next question.
And.
Our next question comes from the line of guilt Bloom from need at My company. Your question. Please.
Good afternoon, everyone and thanks for taking a question maybe a bit of a rephrasing I'm. An earlier question here is there a situation.
And what you think the data from embark will be.
Broken up based on H.
Given.
Potential different feedback from the agency. Thank you.
No no we don't the study has been powered.
To see a treatment effect in this study in population, which is four to seven it's very very.
Barry well power to see that and.
We've had conversations with the F D a and F D a leadership.
Which is is confirm that if we are successful in our trial. They will move rapidly once we've submitted data to them to review that data and to remove the age limitations.
Thank you one moment for our next question.
And our next question comes from the line up we ear from <unk>. Your question. Please.
And can I see how congrats on the dosing the first patients. So my question is referring to what does uhm sat empty prepared remarks, you said you're going to expand the label in the first half cause the majority of patients can you kind of elaborate by what you mean by that <unk>.
<unk>.
Older patients in the Nonambulatory patient would those be included with that come much later, where I. Thank you for calling if you've reached 95 per cent of the patience. Thanks.
Yeah. Thank you very much for your questions. So let's go there's sort of two steps to this so as we stand here today, we have a label for four and five year old patients.
When embark is successful and you know we have a lot of conviction around embark in the successor to embark we will immediately submit that data to the agency before we've even submitted a b L. A supplement with the goal of them beginning the review as soon as they receive that data <unk>.
Assuming that embarks successful, we would assume that all age limitations will be removed from the label and that gets us to the majority of patients.
Both ambulatory non ambulatory in all age ranges as we've said we've had conversations with the agency about removing all of the age ranges there would be no logical basis to assume that.
Ambulation status would be a reason to limit access it's not as if this.
Protein is aware that a patient in a wheelchair or not so we feel very confident about that there is one additional thing we'll do witches will take a cut from our current non ambulatory study to have additional.
Safety data that supplements the safety data, we already have on the older and non ambulatory patients as well. So that's our goal first early next year to have a broad label that covers all patients. There are two there will still remain to limitations.
And the ability to receive that therapy, one of them is this 5% or so of mutations that her coverage.
Coverage Exxon aid and and were nine that are contra indicated for those mutations that will remain for some time, we're gonna continue to do work on that and hopefully narrow that but for the time being that will be in the label and then the second one is.
Preexisting antibodies about 13.9% based off based on are so prevalent study represent patients that have some environmental exposure to something that looks like an antibody for our 874 of those patients cannot currently be dose.
But as Dr. Video playback mentioned in her prepared remarks were starting to different alternative approaches to clear those antibodies one in Melissa days to cleave them in a <unk> as a second alternative to clear antibodies and if one or both of those are successful that in the near term we could start presumably.
Empowering physicians to safely and effectively dose even patients that have preexisting antibodies that would get us to more than just the majority of patients to potentially as much as 95 per cent of patients. So that's our goal.
Thank you one moment for our next question.
And our next question comes from the line of Michael's from Morgan Stanley . Your question. Please.
Hey, guys. Thanks for taking the question maybe just another one on the <unk> launch can you prepared remarks, you mentioned greater than 50 sites are trained and activated currently.
Just curious if you can give us the total number of sites expected just trying to get a sense of whether that represents a majority currently or where you're at in that process. Thanks.
50 sites is a tremendous number of sites where I am.
Completely <unk>.
Thrilled with the team's ability 50, so about 80% of all Duchenne patients are covered by about 50 centres of care in the United States.
So 50 sites is an enormous number of sites too.
Two interviews 11 is our goal is to get to as many as 70 sites over time.
And were significantly ahead of schedule in our <unk>.
<unk> goal to do that so we're in really.
Really great shape from our site perspective in Darwin and his team deserve an enormous amount of kudos for where we've got right now.
Thank you one moment for our next question.
And our next question comes from the line of Danielle Brill from Raymond James Your question. Please.
Good afternoon, and thanks for the question I have a quick follow up to.
The question on the standard deviation I guess I'm I'm curious are you able to see minded S D data or any other applying the day definitely embark that allows you to just ask your party and just kind of helping you can elaborate further on on your confidence.
And the assumption thing.
We do not have access to so I'm looking at the blinded data wouldn't be very insightful and we don't have access to the unblinded data so.
We feel very what we feel <unk>.
Very comfortable with is that our initial study was powered with the standard deviation that we're seeing in studies that did not have the homogenized thing restrictions that embarked has in it and yet when we did the simulations when Luis and her team and our staffs group did the simulation.
<unk> to test the powering assumptions and the like they did not actually take into account the homogenized and aspects of the change in protocol things like as I mentioned before Stratifying on age and and.
Making sure that all kids had a rise time under five seconds and having strong.
Floors and ceilings and the like so we feel very good about the powering of the study and we feel good as we sit here today on the standard deviation, but we haven't had access to unblinded data to test those assumptions.
Thank you one moment for our next question.
And our next question comes from the line of writ too far off from T. D. Cowan Your question. Please.
Good afternoon, guys. Thanks for taking the question just <unk>.
To the centers for a second <unk> can you say, it's the handful of patience that you were referencing in the next.
And in the next couple of weeks will they be at five different centers and of the 50, and then can you talk a little bit about.
Are there centers that you expect to move faster than others, maybe given their history with silken nine how comfortable they are and then if I can squeak. Another one in do you know how many.
Antibody tests had been run and can you really fast.
So I don't know the last answer for you I can answer the first two all of them. So all of the handful or a different sites.
So they're broadly across different sites.
And then if a any sites are faster.
The good news on the site first of all.
The amount of passion from the physicians in.
Is really wonderful and and everyone's trying to move as fast as possible to get kids.
Infused and benefit these kids, particularly kids that might otherwise age out we have really benefited and the team has really benefited from the fact that these most of these sites. The majority of these sites have had experience with souled gentleman know how to infuse gene therapy. So they were well prepared but.
Four we went in and got the process of initiated them and getting them site ready so.
I would say broadly speaking there is a lot of enthusiasm to move as fast as reasonably possible to get patients benefiting from 11 is.
Thank you one moment.
In terms of the the the antibody testing, it's going smoothly and and functioning well and we are disclosing the numbers right now, but it is working well and and going smoothly.
And we're happy with the demand.
Thank you. Our next question comes from the line of a new <unk> from J P. Morgan Your question. Please.
Hey, guys. Thanks, so much for taking my question.
Just a clarification point sorry, if I missed that what are the gating factors to starting that F. M. P. 9003 pivotal study later this year. Thanks, so much.
I'll turn this over to Luis if I'm getting any of it wrong, but I think it's all just C. M C. In manufacturing if I'm not mistaken, but Luis you tell me if I.
If I'm missing a nuance.
Yeah, that's accurate.
<unk> <unk> <unk> <unk> <unk>.
But we as I mentioned, we fully involved caring and have a great deal of a patient <unk> and most of them having.
What's your date as well.
Thank you one moment for our next question.
And our next question comes from the line of Joseph Schwartz from Leerink Partners. Your question. Please [noise].
Hi, all this is will on furniture today, thanks for taking our questions and I'll add my congrats on that progress this quarter. So one for US wondering what the opportunity is for SRP 50, 51 relative to the PM. Those that you're currently market is this a potential franchise expander and are there any reasons why a patient is not currently on a P.
<unk>, what would be a candidate for a P. P. M L. Thank you.
So not on it so that you know I think.
Broadly speaking if it was successful so we'll see the data later this year and then we'll if we're successful with the data later this year and the risk benefit justifies. It we're going to seek an NDA 450 51 next year. The goal then would be there's a couple of things. The first goal is that it would be.
Ah more convenient and and potentially much more powerful version of our Pmo's. So for patients already on the therapy It would.
Would be a great transition to this therapy for them and benefit them.
You could envision in the United States that we might have you been <unk>, we've got a great success record with access we might have even greater success with access if we were making you know, 345%, which would be multiple four holds more uhm dystrophin then the Pmo's and then it does always offer the.
Theoretical opportunity to move outside of the United States, which is something we're very excited to do which is bringing R. RNA technology more broadly around the world to benefit patients. So there's a lot of potential opportunity with the P. P. M. O's if successful so we're looking forward to looking at the party.
<unk> of momentum later this year in the making some decisions across the portfolio and aggressively bringing things forward. If we see success with 50 51.
Thank you one moment for our next question.
And our next question comes from the line of Christian <unk> from Cantor Fitzgerald. Your question. Please.
Hi, everyone. Congrats on today's milestone given that you're taking all these steps now across sides pairs an antibody testing is it your expectation that if you ended up getting a broader label in the first half of next year that the three to four months timeline, you've <unk>, you've communicated to us in terms of.
Patients getting on therapy could be shortened thank you.
Yeah. The answer is generally speaking yes.
This really is an initiation issue with the launch of the therapy policies have to be in place in some places code have to be in place and the like and then of course, we have this current release process. So we ought to be in a position to move faster over time, but as as I said before.
We've had a lot of very fantastic early success, but we should assume that the the real ramp begins later this year.
[noise]. Thank you one moment for our next question.
And our next question comes from the line of <unk> from William Blair. Your question. Please.
Hi, This is John <unk>. Thanks, so much for taking our questions. So I was just wondering beyond the accurate that the team is making to get access to patient approaching six years of age just wondering if you could talk a little about any higher demand your thing from the patients the protein that age cut off and if you're seeing more patient transferring notification.
Uhm.
Turn this over to a Dallas.
Yeah. Thanks for the question, we're seeing demand broadly in both the four and five year old age group.
In terms of access early on it is skewing heavily to those patients that are that are that have the birthdays just be more so maybe because of the urgency and the dialogue that's happening between kols payers and and the teams right now so early on yes, we are seeing it skewed towards towards six you the the people.
Birthdays, turning sick, but we expect that to normalize very quickly as we as we start to work towards all of the patients that are eligible that four to five age group. So we're seeing we're seeing demand and start forms across the ages will likely get access skewing to the <unk>.
Older Kids in that age range for the obvious reason that we want to get them dose before there's an issue with the label Yep.
Thank you one moment for our next question.
And our next question comes from the line of Brian's Corny from Bird your question. Please.
Hey, this is Luke on for Brian . Thanks, just taking a question regarding piece of progress, giving you envision trial up and running have you seen any signals that the availability of commercial product might be a headwinds your enrollment in the ambulatory population.
The answer that is going to be no. So we were very thoughtful about the protocol for envision in fact, we've significantly limited the the number of patients that are gonna come out of the United States to ensure that we don't have an issue with the the progress of that therapy as we broaden the label now that.
Does that mean that the enrollment for envision will move slower than for instance, embark which rapidly enrolled as everyone may recall.
But ultimately we're not seeing an impact from this approval on that basis and part of it's because we're we're gonna to enroll the majority of.
Subjects outside of the United States.
Thank you one moment for our final question for today and.
And our final question for today comes from the lined up digit Chattopadhyaya from Guggenheim. Your question. Please.
Hey, good afternoon. Thanks for taking me getting my question and.
What is the current gross margin for <unk>, and where do you think it will migrate either on peak capacity or if you could successfully migrate to the 1000 litre scale.
Sure I'll turn this over to EM.
So we said that we would expect the margins to be like a high quality uhm.
Talking to agents, so <unk> in that 80 per cent range.
Similar to what we're seeing with our PMO now obviously, it's gonna be higher when the the earlier patients are being dose and then F heavier patients and we spend a label turned down but basically into you know kind of that that 80% range and then obviously to your good.
Point, we could see a big change or not in a position to quantify that from where we are from a manufacturing perspective on our suspension process, but obviously you had.
<unk>, a big impact on our on our yield and so we can see that driving that number higher but obviously, we're not willing to commit to that just yet.
Thank you.
Does conclude the question and answer session of today's program I'd like to hand, the program back to management for any further remarks.
Mmm. Thank you Jonathan and thank you everyone for joining us this evening and for your your questions I. Appreciate it. We've obviously made from my perspective, great progress so far this year both.
With the approval of a <unk> and then serving patients both with <unk> and with our existing Tmo's I will remind us that the team has done a brilliant job of continuing to serve the community with RPM OS and where.
We feel very confident about our current guidance on the <unk>, we have a lot to do for the rest of the year. Both serving these patients continuing to serve the patience with the Pmo's.
Getting the Embarq read out about which we have an enormous amount of conviction and then we will share that with you at essentially the same time that we share with our colleagues at the F. D. A and our goal of course is to broaden this label as soon as reasonably possible and bring 11 as to the.
Vast majority of patients living with two Shannon their families would that I look forward to updating everyone over the course of the quarter and I would ask everyone to have a lovely evening.
Thank you, ladies and gentlemen for your participation in today's conference. This does conclude the program you may now disconnect good day.
Mmm.
[music].