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

Welcome everyone to the 39th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at JPMorgan. I'm joined by Tessa Romero and Matt Bannon from the team. The next presenting company is Sarepta. And speaking on behalf of the company, we have CEO, Doug Ingram. Before I turn it over to Doug, just wanted to highlight for those listeners on the webcast to please use the submit-a-question, ask-a-question button in the portal, and I'm happy to ask the question on your behalf. Doug, take it away.

Thank you very much, Anupam. Let's pull the slide deck up now. Great. And good morning to everyone online, and thank you for joining us today. As a mission-driven leader in precision genetic medicine for rare disease with a pipeline of over 40 candidates across RNA and gene therapy and gene editing, there is much that we can discuss today. However, in light of the recent release of our interim results for SRP-9001, our Duchenne muscular dystrophy gene therapy, I will break from my historical approach, and will spend the bulk of my time today discussing those 1 and 2 results. And I will have an important update on Study 102 Part 2 expression in this presentation as well. Next slide. Before I begin, I will remind you to please look to Sarepta's public filings for the risks and uncertainties attendant with making predictions about the future. Next slide. On Thursday, January 7, we announced the interim results from Part 1 of Study 102, evaluating our gene therapy, SRP-9001, for the treatment of Duchenne muscular dystrophy. Next slide. To remind you, Study 102 is a double-blinded, placebo-controlled trial studying SRP-9001 in patients 4 to 7 years old. There are 41 patients in this study. In Part 1, it is randomized 1:1 between treatment and placebo and it was the 48-week timepoint data from Part 1 that was released last week. In Part 2, we maintain the blind and we cross patients over, where those who received active therapy in the first part will get a placebo to maintain the blind and those who have taken placebo in Part 1 will get the active therapy. All patients will have crossed over in the next few days, so this means we will have an important opportunity by the end of this year to review the results for all 41 patients on therapy, including prespecified trajectory analyses, evaluation against a natural history cohort, and additional evaluations as well. It also means that we must be very careful to avoid anything that could impact the ongoing integrity of the study, which also means that we will assiduously avoid providing patient-level data before Part 2 reads out. Now with respect to Part 1 of the study, the primary functional measure was changed in the North Star Ambulatory Assessment, or NSAA, at week 48. We also identified that 4- to 5-year olds will traject differently than 6- to 7-year olds. So we stratified by those age cohorts, and prespecified as important secondary functional endpoints, a separate NSAA assessment in the 4 to 5s and the 6 to 7s. Next slide. We robustly achieved statistical significance on all expression-related markers, achieving vector genome copies of 1.56 and expression on Western blot of 28.1% of normal. And likewise, we strongly hit statistical significance on the immunofluorescence measures as well. One will note that while robust and statistically significant, the expression numbers in Part 1 of Study 102 appear lower than those we achieved in our earlier Study 101. For Study 101 when one removes patient 4, who was a super responder, our Western blot quantification was 53.7% while our expression in Part 1 in Study 102 here was 28.1%. Now Dr. Louise Rodino-Klapac explained last week that our material supplier, Nationwide Children's Hospital, originally used a less precise titering method that they called supercoiled qPCR that resulted in lot-to-lot variability and reduced overall mean expression. However, Sarepta has validated a new and much more precise linear qPCR titering process, and we have already applied this more precise titering process to the crossover patients in Study 102 and in our Study 103, where we have already dosed 11 patients with our commercial process supply. Last week, we predicted that when we are able to review the biopsies of patients' dosed using the more precise titering process, we believed we would see expression levels that are closer to what we have seen in Study 101. Now the very important news today is that we no longer need to speculate or predict this outcome. Our translational group has worked rapidly to analyze the first 11 patients dosed in the 102 crossover. Immunofluorescence is going to take longer, but I can now provide you with the Western blot quantification of dystrophin for SRP-9001 on crossover using lots made with our updated linear qPCR titering method. Next slide. And here it is. As you look to the far right of this slide, you will see that micro-dystrophin expression on the first 11 patients on crossover in study 102 is 51.7% of normal. This is nearly identical to the 53.7% we saw in Study 101, and we achieved 2.62 genome copies per nucleus, even greater transduction than we saw in the 3 patients in Study 1. The overall age of these 11 patients at biopsy was 7.14 years; and at 6 and 5 patients, respectively, the participants were evenly balanced between the 4 to 5 and 6 to 7 cohorts, and mean expression levels between the age groups were very similar. As we predicted, with the use of our updated linear titering method, in the first 11 patients of crossover, we obtained transduction and expression levels equivalent to what was predicted by Study 101. This confirms our confidence in the differentiated expression of our therapy, and it does get us excited about what we could see at the readout of Part 2 by the end of this year. Now let us move to the functional results from Part 1 of Study 102. Next slide. This graph shows the NSAA change over time in Part 1 of the study. The blue line represents placebo patients and the red line, treated patients. For this graph, we have normalized the starting point at baseline for all patients to 0. This may create the false impression that all patients had similar functional baselines. Unfortunately, such was not the case. And as I will discuss later, this had a major impact on the Part 1 readout. You will note that in each and every timepoint, therapy performed better than placebo, and the on-therapy cohort had a statistically significant improvement from baseline at 48 weeks, while the placebo group did not. However, importantly, on the primary analysis of change from baseline at 48 weeks compared to placebo, we did not achieve statistical significance. Now like many of you, last week, when I was told that we had missed statistical significance on our primary functional endpoint, I was not merely disappointed. I was, to be frank, utterly gobsmacked. Everything we have seen from this therapy, all of the preclinical work and animal models, and the remarkable results from our proof-of-concept study, stood in opposition to this result and predicted we should have seen statistical significance. But immediately after my astonishment, we turned to our prespecified functional analyses and the apparent reason for this discordant result jumped off the page. Next slide. As I have noted, predicting a difference in the 4 to 5 age group versus 6 to 7 group, we stratified in prespecified analysis by age. Now I want to emphasize this again. I want to reemphasize it. When I show you the age-specific results, these are not after the fact, post-hoc analyses. These are our prespecified functional assessments. And this slide here shows the baseline functional characteristics of the treated and placebo cohorts in the 4 to 5 age range. And as you can see, our rigor around entrance criteria worked well. And the baseline characteristics are strikingly similar to one another on each and every functional measure. At baseline, these are the same patient population. Next slide. And when we get baselines correct, here's what you see. In our double-blinded trial with the placebo -- or where the baseline populations were the same, SRP-9001 robustly hits statistical significance. This is a p-value of 0.017. And it revealed a substantial and clinically meaningful benefit at 48 weeks. These children improved 4.3 points in 48 weeks, and the effect was so profound that SRP-9001 hit statistical significance with 16 patients. Let's linger on this for just a moment. This is the first time in all of history that in a double-blinded, placebo-controlled trial of DMD patients, a therapy has shown this sort of statistically significant and clinically meaningful benefit on NSAA in DMD patients. This is everything we could have asked for and perfectly predicted by all of the evidence that precedes the study. But next slide. But now let's move to the 6- to 7-year old cohorts. These are the baseline functional characteristics of the active and placebo arms in the stratified 6- to 7-year-old cohorts. And as you can immediately see, these are strikingly different populations. On every single functional measure, there is a substantial and very marked difference in the baseline characteristics of the treated group and the placebo group. Notice NSAA. There is an enormous 4.4-point difference between these groups at baseline. That has a p-value of 0.0046. The chance of this occurring was about 4 in 1,000. And what it means is that the group in the treated arm is much more severe than the placebo population. And this occurs for each and every functional measure. And these are not modest differences. Just look, for instance, at the 12.35-second difference in the 100-meter or the 71% slower time to rise in the active group at baseline. Simply said, these children may be the same age, but they are very different and noncomparable populations with different disease progressions at baseline. And sure enough, when you take a more aggressively degenerating population of DMD patients at baseline and you treat them, and then you try to compare them to a significantly milder population, we were unable to reveal a statistically significant difference. Any drug effect would be masked by the fact that we compared the wrong populations. This is an undeniable fact. The miss in statistical significance in the primary functional analysis is entirely explained by the miss between these 2 noncomparable populations. Next slide. Obviously, logic and good science tells us that comparing a severe treated group to a milder placebo group could confound the trial. But the DMD literature supports this. From a recent publication, here you see a graph of a large natural history study showing different progression curves based on NSAA. There are 4 identified populations of patients with different progression curves. What you see here is that class 1 in red is the most severe and the steepest-declining population in the relevant time frame, and that black square there is the NSAA of our treated group. And as you can see, our treated group is at or worse than the most severe group in this study. This natural history study would have predicted that our treated patients would be in steep decline, perhaps 4 to 5 points of decline in 1 year. In our study, these severe patients did not decline, but in fact remained stable. But as the placebo population was much milder, the placebo has actually improved over the course of that year, masking any potential benefits of SRP-9001 in the analysis. Next slide. Turning now to safety. Study 102 continues to show that we have a very differentiated therapy on safety. We had no new safety signals, and importantly, no clinical complement activation was observed. We have now treated about 55 patients with SRP-9001 and our confidence in the safety profile of our therapy only strengthens. Next slide. So to summarize Study 102 Part 1 results, first, we have seen no new safety signals. Second, we robustly hit statistical significance on all expression and related biomarkers, but while the previous titering method created variability that brought the expression mean down in Part 1 versus our prior prop of concept study, the crossover biopsies that were announced today indicate that as we had predicted with a more precise linear titering method, we are achieving transduction and expression equivalent to what was predicted in Study 101. And third, while we robustly achieved statistical significance and bring meaningful benefit on the functional endpoint in the properly matched 4 to 5 age cohort, the baseline populations in the 6 to 7 age group were entirely mismatched. And because of the results of the 6 to 7 age group, we missed overall statistical significance on the primary functional endpoint of this study. We will address this imbalance. We will assure it does not occur in our next study, and we will work to address it as well in the Part 2 analysis of Study 102. Next slide. We have a lot to do, and more readouts in the coming 12 months. We will continue to advance Study 102, and we will have our functional readout on all 41 patients at the end of this year as we unblind. Second, we have enrolled and dosed 11 patients in our Study 103 evaluating our commercial process material, and we will have biomarker and safety readouts there next quarter. And third, we will take our unique insights from Part 1 of Study 102. We will use them to finalize Study 301, and we will get that study started by midyear. Next slide. We will continue to move with urgency and purpose. Because while we are, of course, very disappointed by the imbalance in the treatment arms and very disappointed by this miss on stat sig, the data in this study strengthens our belief in SRP-9001, and it hardens our resolve to get this therapy to patients. Next slide. And so we're clear, this is important because around the world there are literally hundreds of thousands of patients degenerating every day, that desperately need a transformative therapy. Next slide. I'll now move to the RNA franchise. Next slide. As you know, we have 2 RNA therapies approved today, EXONDYS 51 and VYONDYS 53. The PDUFA date on our third potential therapy, AMONDYS 45, is next month, and we are prepared to immediately launch that therapy if approved. In the fourth quarter of 2020, our revenue stood at $122.6 million, a 22.6% growth over the same quarter in the prior year; and our full year revenue was $456 million, a 19.7% growth over 2019 -- impressive performance in the midst of a very destabilizing pandemic. Looking to this year, our guidance range, including AMONDYS, is $537 million to $547 million or about a 19% growth over last year. And in addition to the revenue that we will have this year, as of December 31, 2020, we had approximately $1.9 billion of cash on our balance sheet. Next slide. We are also working on our next-generation version of the PMO, the peptide-conjugated PMO, or PPMO. Now the goal here is to enhance the functional benefits of PMO by using a peptide to increase cell penetration, exon skipping and dystrophin production. In the fourth quarter of last year, we showed early results from our first PMO -- or PPMO construct, SRP-5051 at the 20 mg per kg test, and the results were very encouraging. Even as we have not yet entered the predicted [ C ] part of the dose response curve, at 1/10 the total drug exposure versus our current PMO EXONDYS, we saw an increase in tissue exposure, an increase in exon skipping and a fivefold increase in dystrophin production. We will continue to dose escalate to 30 mg per kg and hopefully 40 mg per kg, and we will report out those results, after which we will decide our pivotal trial for SRP-5051. Next slide. I'm now going to move back to gene therapy. Next slide. Looking here, you'll see that we have one of the most robust and impressive pipelines in gene therapy for rare disease. Probably all that exists today in [ trial ]. Next slide. One of the most advanced programs after SRP-9001 is our 5 program LGMD portfolio. And the most advanced therapy there is SRP-9003 intended to treat LGMD2E [ life ending ] neuromuscular disease. In 2020, we reported safety, expression and function from our low and high dose cohort studies for LGMD2E, and the results were impressive. On this slide, you will see the results from our low-dose cohort. And therapy, as you can see here, was well tolerated. We obtained very good expression, and at 18 months, we saw an impressive improvement in function versus baseline. Next slide. This slide represents our high-dose cohort results for LGMD2E, and these results are again very impressive and very encouraging. At a dose similar to SRP-9001 the therapy was again, safe and well tolerated, expression was very impressive. And even at 6 months, we saw a significant increase in function against baseline. Next slide. We will meet with the division this year to gain alignment on the development and regulatory pathway for LGMD2E, and we will then apply that insight across all of our LGMD programs. Next slide. Understandably, I ask that the bulk of this presentation discussing our micro-dystrophin program, but we should not lose sight of the fact that we are building for the future with one of the most extensive and impressive multi-platform genetic medicine pipelines now in existence. Next slide. And as is always the case at Sarepta, we have an enormous amount to do to advance our plans this year, and we have significant milestones to achieve across 2021. And you can see some of them on this slide. And we will do all of this as we keep ourselves razor focused on our top priorities, and we move with a sense of urgency and the creativity that our patients need and deserve. And with that, thank you. We should bring the slides down and shortly commence Q&A.

Okay. Thanks for that, Doug. And Doug, maybe you want to introduce the broader team that you have on the line here.

Yes. Thank you very much for that, Anupam. I'm very proud to say with me today is Dr. Gilmore O'Neill, our Head of R&D; Dr. Louise Rodino-Klapac, our Chief Scientific Officer. We have Dallan Murray with us, who is our Chief Commercial Officer; and our Chief Financial Officer, Ian Estepan, is with us today as well.

I just want to remind all the listeners on the webcast to ask a question via the portal, and I'm happy to ask the question on your behalf. And our first question actually comes from the portal, which is the statistics around the study. Why wasn't -- not the ANCOVA analysis used, which adjusts for sort of baseline factors like NSSA (sic) [ NSAA ]? Why was -- why did you not use this statistical methodology?

I'm turning this over to Dr. O'Neill. He wants to comment on this.

So we actually decided to -- the analysis that we actually determined were based on a number of factors. We had to make some choices. We actually decide to use the methodologies, the MMRM to account for variation in data and actually also the potential for missing data. And with regard to ANCOVA, there are additional analyses that we are running that we will be looking at over the coming weeks and months to help us better understand our data set. But we had to make some important choices when we were designing the study and between the stratification, this prespecified subgroup analyses and our MMRM methodologies. These were the choices that we had to make.

Got it. And then maybe a question, obviously, post the results there's a bit of a debate on the street. But maybe first, focusing in on the 4- to 5-year-olds. There are some that push back on the 2.5 point NSAA delta that you observed in these 4- to 5-year-olds, saying that these patients are still in a gain of function, right? And so maybe you could help us put that 2.5 point change into context in these 4- and 5-year-olds specifically?

Yes. A couple of things. First of all, let's be clear. The treated group actually had significantly over a 4-point change from baseline. And so yes, as we had predicted, we had talked about with any investor that spent any time with us over the last year to 1.5 years has heard us talk about the fact that having a significant understanding of Duchenne, we had predicted that the 4- to 5-year-olds and the 6- to 7-year-olds could traject differently and that the 4- to 5-year-olds could gain as much as 1 to 2 points over the course of the year. That's exactly what we saw. So our predictions were very consistent with what we actually saw. Now the active group had 4.3 or so points over their baseline, significantly improving much better than natural history, but more importantly than that, much better than placebo, with, I will note, again, a p-value of 0.017. And I think if we talk to thought leaders in Duchenne muscular dystrophy, I think they would generally agree with us: That is not only an unprecedented result in a trial, I mean there is not a trial to date that has ever shown this kind of significance in a prespecified placebo-controlled way, but it is very clinically meaningful for these patients.

We've got another question from the portal, which is how many prespecified analyses are there in Study 102?

Do you have a number of those on function itself? Dr. Rodino-Klapac, are you aware of the number of prespecified functional assessments that were done? I don't think they were very many.

Primary prespecified was the randomization, the stratification based on age of 4 to 5 and 6 to 7. Dr. O'Neill, do you want to add anything to that?

No. I think the key prespecified analyses were around, again, we had the stratification a priori and then we prespecified the subgroup analyses around the 4- to 5- and the 6- to 7-year-olds. There are a few others, but these are a relatively small number of prespecified analyses. We do actually have a number of prespecified analyses in the exploratory outcomes, but I can't give you an exact number. But they are detailed in our statistical analysis plan.

We've got another question from the portal, which is how are you thinking about better age match controls in the limb-girdle studies given your learnings from SRP-9001 results?

Well, we -- the short answer is we've only looked at the top line very recently and we've got a lot of thinking to do about not only SRP-9001 or Study 301, but also our limb-girdle programs as well. So I can't give you an answer in advance, nor can Dr. O'Neill or Dr. Klapac about precisely what we'll do. I can promise you this. We have a unique insight as a result of this study. We have very significant amounts of information that we can apply to future development to ensure that we increase probability of success. We can certainly address this imbalance issue in the next study for SRP-9001, and we will also take all of this learning and apply it to the limb-girdles, but we've got work to do there.

And maybe, Doug, on this point that you're talking about, addressing some controls with the next study in terms of -- like when we think about DMD trials, it just seems like the heterogeneity of the population has been a problem for many trials. So how exactly should we think about controlling this and different factors to kind of ensure a more homogeneous population being enrolled?

Well, let me say a couple of things. First, I think I'm going to be very defensive on behalf of the team for just a moment. And I would note that the baseline differences in the 6- to 7-year-olds had a p-value of 0.0046. That should not have occurred, of course. What that tells us is that, that should have only occurred about 4 times in 1,000. So unless there was something that systemically had happened, and we have seen nothing like that in all of our audits of enrollment, this was just an extraordinarily bad luck. With that said, it doesn't mean we're not going to address this issue going forward. We had done a lot already, of course. We had done what I think others may have missed before us, which is even in this fairly tight age population of 4 to 7, we had prespecified 4 to 5 separate from 6 to 7. We had stratified on behalf of those agents, which I think everyone knows. I don't think I've had an investor call over the last 1.5 years where we haven't discussed that. So we had -- in that regard, we thought we were very careful about that. We put some [ caps ] in place and a floor. We'll have to do even more. I mean I think your point is well taken. The degenerative diseases can be difficult. There are a number of things we can do to ensure that this doesn't occur again, and one possibility and certainly the top of mind possibility is that you, in fact, stratify on the basis of the baseline functional characteristics. But we're going to look at all of that and ensure that we apply it to our next Study 301 so that this will not occur again in the next study. And I think, without getting too far over our skis, I think in advance of unblinding part 2 of this study, there are things that we can do and look to, to ensure that we can moderate significantly the impact of these differences in baseline characteristics before we unblind, and we'll certainly look to that over the course of the rest of this year.

We've got another e-mail question here, which is through the portal. Based on these data from 901 (sic) [ 9001 ], how do you consider the correlation between micro-dystrophin expression and function?

Well, I don't think -- we haven't done an analysis at that depth right now to have that conversation. I mean, broadly speaking, obviously, what we've -- first of all, a couple of things before I start talking about hitting the stat sig in the 4- to 5-year olds. I do want to linger on the fact, I mean we're deeply disappointed that we missed statistical significance in the primary analysis. I don't want to pretend like we're trying to gloss over that. This was very surprising to us and very disappointing. But when we got the age -- the baseline populations right in the 4- to 5-year-olds, we strongly hit statistical significance. And we didn't just sort of just make it. This was 0.017 p-value, and we've got like 16 patients. So I think that there is clearly the case from our perspective that we're big believers in this therapy and that if we address the baseline characteristics and the kinds of expression that we can get with our therapy, we will see a significant benefit in the active versus placebo group in future studies and perhaps even in Part 2 of this study.

We've got another portal question here. How would you describe the differences in doses of steroids between the 2 arms?

They're the same. There's no difference in steroids.

Okay. Okay. Another question through the portal, which is what are the time lines for the LGMD2B study? Any timing granularity would be helpful.

Yes. We're not prepared to give time lines yet. We need to sit with the agency, talk about the regulatory and development pathway, first for LGMD2E, and then we can apply that thinking across all of our LGMD portfolios, and then we will come back and provide an update, including time lines and the like for everyone.

For -- another question through the portal, actually. For Part 2 for 9001, what would be considered a positive result? And is there any analyses that are specified?

So we can continue to ponder the specification because it's still blinded now. We do have prespecified analyses. I don't want to go into a lot of detail yet about that. I will say, broadly speaking they include, for instance, the review of all of the patients against the natural history cohort. Remember, we can't look at active versus placebo in Part 2 because every child will, by the time we unblind Part 2, have been on -- have been on active treated therapy, but we can look against the natural history cohort in relation to that in advance of that unblinding. We can consider how to properly match the natural history cohort to what the patients look like in the study itself. We also have -- and I can't give a lot of detail on this -- we have prespecified trajectory analysis as well, which could be very interesting for us. Remember, what we have in addition to the data that you've seen in Part 1, is we will have had patients for a significant period of time with functional results that are not on therapy, that we can see their natural trajectories. And then, of course, we can intervene with therapy and do an analysis thereafter and change the trajectory, which is something we're certainly going to look carefully to and make sure it's all thought through and prespecified in advance of locking the stat plan and unblinding the therapy. And the last patient/last visit will happen in the fourth quarter, so that should all happen right around the end of this year.

I think Tessa from the team wanted to ask a question on the PPMO franchise. Tess?

Well, yes, actually, first one on 102, actually. So Doug, I believe you made some comments on the differences in the Western blot technique that you used between Part 1 and Part 2. Can you provide just a little more color on that? And then is the Part 2 technique what you'll use in 103? And then I have a PPMO question as well.

Yes. So just to understand, this is the broadest of strokes. And then, of course, if you get more technical than this, I can always turn to Louise to describe it in more depth than you would like. The short answer is this. The Nationwide Children's Hospital which is the provider of our clinical material, had historically what's called supercoiled qPCR. And it turns out that that approach creates some variability lot-to-lot. We had anticipated when we saw the data that in fact that that variability would have reduced the overall mean of expression. And if we were right, if we used our more precise linear qPCR -- because Sarepta has developed a far more precise titering method called linear qPCR -- that we would get far less variability, and that would actually increase the [indiscernible]. Now just so we're very clear about this, we developed that, and we've applied it already. We applied it for all of the crossover patients in Study 102. And we've used it for all of the patients so far dosed in Study 103. So every patient in crossover 102 and all of the 11 patients so far on 103. And what you saw today -- what I talked about today was that we have the Western blots for the first 11 patients at crossover, or the first 11 patients that have been done. This is not -- just so we're clear, it's not 11 of 20 or cherry-picking. These are all the patients that have been done. And as we had anticipated last week, with our more precise linear qPCR method we get, obviously, [ tied to us ], and we do get a higher expression on mean, and indeed, we get, at least in these 11 patients, we saw exactly what we would have predicted we would see from Study 101. As you may recall, Study 101 was around 53% for the first 3 patients, excluding the super responder. And what we saw here was about 51.5 or so percent. So they're essentially identical. And the genome copies per nucleus were very robust in this -- these 11 patients. And in fact, the genome copies per nucleus were higher even than you saw in Study 101. So I think what we had suggested last week and we didn't have the data yet, we suggested last week has borne out in these 11 patients. And we -- I think this is further confirmation that we have a very differentiated therapy with rh74 and the approach that we take and our promoter on expression. In addition, of course, to safety.

If I may quickly add. The Western blot method itself was exactly the same between Part 1 and Part 2. Because that was part of the question, I believe.

Apologies. I missed that.

Yes.

Another question from the e-mail portal here. Will you be able to stratify according to both age and baseline NSAA in Study 301? Or will you switch to stratifying by baseline NSAA only?

Well, we're still looking at that. We're going to make a thoughtful and careful decision about that. Obviously, the superficial answer from someone like me is we'll stratify for both and ensure that we never have this issue. But the actual developers of the protocol will take a careful look and make sure that we've built a protocol that has the maximum probability of success, and we'll update everyone on that. I'll tell you one irony. As you know, back in September of last year, we found ourselves in a position where we hadn't and weren't able yet to start Study 301, and I was disappointed at the time. I have to say we're -- ironically, we're in a good place because we haven't yet started 301. We have the ability to take all of this wealth of information out of Study 102. And there's a lot more information out of this than the top line. And use all of that to maximize the probability of success as we finalize Study 301. We can do all of that without in any way, reducing the -- slowing the time lines down for the start of 301. So we will do all of that and maximize our probability of success.

Maybe final question here, which is another one from the e-mail portal, which is on expression. What was the range of expression that you saw using your methodologies?

We have yet to describe the ranges on expression. And I will say one thing going forward, semi-related probably -- just semi-related to this. I will say as we go forward, I think one of the things that people would love to see even more information on a patient-level perspective on the study, but we can't provide that. And I'll be very direct about that. There's an enormous amount of value in the Part 2 of this study. We need to make sure that we don't -- that we fulfill our obligation to provide the top line on this study and people deserve to know about the top line of the study, of course. But we need to maintain the integrity and conduct of part 2 because we're very interested in seeing the results at the end of this year on Part 2. So we've got to avoid anything that might look like we're providing to the outside world patient-level data in advance of the unblinding of Part 2.

Great. Okay. That looks like all the questions in the queue. Doug and team, I want to thank you guys so much for this super informative session. And thanks to all the questions from the audience. That was super cool. And I hope everyone has a good rest of the conference, and go Buckeyes.

Thank you very much, Anupam. Appreciate the opportunity to talk to you today.