Q3 2020 Solid Biosciences Inc Earnings Call

[music].

Ladies and gentlemen, please standby your conference call is scheduled to begin momentarily. Thank you for your patience and please continue to standby.

Operator: Ladies and gentlemen, please stand by; your conference call is scheduled to begin momentarily. Ladies and gentlemen, thank you for standing by, and welcome to the Solid Biosciences update call. At this time, all participants are in a listen-only mode.

Operator: Please be advised that today's conference may be recorded. Later, we will conduct a question and answer session, and instructions will be given at that time. If you require any further assistance, please press star and then zero.

[music].

Jennifer Zolkowski: I would now like to hand the conference over to your speaker today, Ms. Jennifer Zolkowski, Chief Financial Officer. Ms. Zolkowski, you may begin. Thank you, Crystal. Good morning and welcome to the Solid Biosciences 3rd Quarter 2020 Financial Results and Business Update conference call. This call is being recorded.

Jennifer Zolkowski: Before we get started, I would like to remind everyone that during this conference call, we may make forward-looking statements, including statements about the company's financial results, financial guidance, future business strategies and operations, and product development and regulatory process, including statements about the ongoing IGNITE-DMD clinical trial. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory process, the extent and duration of the impact of the COVID-19 pandemic, and other risks described in the risk factors section of our most recently filed quarterly report on Form 10-Q and other periodic reports filed with the SEC. We undertake no obligation to update any forward-looking statements after the date of this call. For introductions and opening remarks, I'd like to turn the call over to Ilan Ganot, co-founder, president, and chief executive officer of Solid Biosciences. Ilan?

Yeah.

Uh-huh.

Yeah.

Ladies and gentlemen, thank you for calling bye and welcome to the solid Biosciences update call. At this time all participants are in a listen only mode. Please.

Please be advised that today's conference maybe record it later.

Later, we will conduct a question and answer session and instructions will be given at that time. If you require any further assistance. Please stuffed dog and then zero.

Oh, not like the hand, the conference over here for me for today.

Jennifer <unk> Chief Financial Officer, Ma'am, you may begin.

Thank you Crystal.

Good morning, and welcome to the solid Biosciences third quarter, 2020 financial resolved and business update conference call.

They just call is being recorded.

Before we get started I would like to remind everyone that during this conference call. We may make forward looking statements, including statements about the company's financial results.

Cancel guidance.

Future business strategies, an operation and product development and regulatory process, including statements about the ongoing ignite D. M V clinical trial.

Actual results could differ materially from those discuss it needs forward looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory process, the extent and duration of the impact of the COVID-19 pandemic and other risks described in the risk factors that.

Ilan Ganot: Thanks, Jen. Good morning, and thank you all for dialing in. Also joining us today are Joel Schneider, our Chief Technology Officer, and Catherine Clary, our Acting Chief Medical Officer. The focus of today's call is to review recent important advances for our company and patients living with Duchenne muscular dystrophy. The first is the FDA's lifting of the clinical hold on the Phase 1-2 IGNITE-DMD trial of SGP001, which puts us on track to resume dosing in the trial in the first quarter of 2021. The second is the additional capital that we brought into the company from a strategic collaboration with Ultragenyx and then at the market or ATM, equity finance. While each of these events is important individually, combined they help us further our goal of developing gene therapies that have the potential to improve outcomes for patients living with Duchenne and creating value for our shepherds. They also give us a great deal of forward momentum as we close out 2020 and move into 2020.

<unk> Harper most recently filed quarterly report on form 10-Q, and other periodic reports filed with the S. E C.

We undertake no obligation to update any forward looking statements. After the date of this call.

For introductions, an opening remark I'd like to turn the call over too long cannot cofounder, President and Chief Executive Officer, a solid biosciences.

Good morning, and thank you for dining room.

Also joining us today are jokes Schneider, our cheapest mojo prefer dropping prairie or acting Chief Medical officer.

The focus of two days cool is to review recent important advances for our company inpatient switching we do spend muscular dystrophy.

The first is it.

B, a lifting will be critical hold on the phase 129 P. M. Detroit, Okay. Two two 001.

Which puts us on truck to read you'd go see in Detroit in the first quarter of 2021.

The second at the additional tropical but we broke into the company from the Philippines, you could've duration mhm for genetics.

And then add the market or a T M equity financing.

<unk> have you been to the both of them individually combine to help us further our goals hopefully that'll be <unk>.

<unk> outcomes so patient.

And creating value for our shareholders.

They also give us a great deal of forward momentum busy close up 2020 and moving from 2020.

During today's call will discuss these recent events into context of our overall corporate strategy. We will review our third quarter of 2020 financial results and provides an overview of our near term goals and priorities will then take your questions before reviewing a recent progress I'd like to take a step back and remind ever.

Ilan Ganot: During today's call, we will discuss these recent events in the context of our overall corporate strategy. We will review our third quarter 2020 financial results and provide an overview of our near-term goals and priorities. We will then take your questions. Before reviewing our recent progress, I'd like to take a step back and remind everyone that Solid was founded specifically to focus on transformative therapies for Duchenne. As many of you know, I'm the father of a child with a... This experience and the deep relationship that we have established with other members of the community are daily reminders of the urgent need that these patients have for therapies that can improve outcomes and quality of life. They are also stark reminders of the need to balance rapid development with ensuring that patient safety is always a priority. As an emerging therapeutic modality, there is still much to be learned about gene therapy.

He won the.

Solid was founded specifically to focus on transformative therapies for sure.

As many of you know I'm, a father of the child due to ship.

This experience and the deep relationships that we have established with other members of the community Ah daily reminders of the urgent need that these patients have therapies that can improve outcomes and quality of life.

They are also stark reminders of the needs to buttons rapid development with ensuring that patient safety is always a priority.

As an emerging therapeutic modality, there's still much to be learned a budget therapy.

Ilan Ganot: We believe that advancing the field of Duchenne gene therapy will create additional opportunities for Solid to achieve our mission of improving outcomes for patients with Duchenne. The potential for these opportunities was a key driver for entering into the collaboration with Ultragenyx, and patient safety has driven and will continue to drive every decision we make with respect to clinical development and manufacturing of our current and potential future therapeutic candidates. With this overarching mission in mind, let me now review our recent progress. As most of you are likely aware...

Ilan Ganot: In November 2019, the FDA placed a clinical hold on the IGNITE-BNB trial after the third patient in the 2E14 VG per kilogram dose cohort experienced a serious adverse event, or SAE. Following this essay, we conducted a thorough internal review of the potential factors that could have contributed to it, looking at all aspects of the program, and consulted with leading experts to inform our decision-making and communications with the FDA. Based on a review of the information we submitted regarding manufacturing improvements, clinical protocol amendments, and all the safety, efficacy, and functional data generated to date in IGNITE-DMD, the FDA lifted the clinical hold on IGNITE trials in early October of this year. We are well on the way toward completing the activities necessary to resume dosing, including submitting the amended protocol to the Institutional Review Board at our clinical site and initiating patient screening following Resuming screening and dosing in India DMV as quickly as possible is our top priority. I'll now turn the call over to Joel, who will review the improvements that we made to our manufacturing process. Thank you, along.

Physical and we are well under way towards competing activities necessary to resume dosing, including submitting damage to the protocol to the institutional review board at our clinical sites and initiating patient screening following appropriate approvals.

Resuming screening and dosing and then you'll have bnb as quickly as possible is our top priority.

I will now turn the call over to Joel who will review the improvements that we've made so a manufacturing process.

Thank you along.

Joel Schneider: As part of our commitment to continuously improve our manufacturing processes over the years, we have now implemented process changes that remove the majority of empty viral capsids from the SGT001 drug product, effectively allowing target dosing of 2 times 10 to the 14th vector genomes per kilogram to be achieved with fewer viral parts, thereby reducing the total viral load delivered to patients. As with all AAV production methods, pre-purification, our process produces full cathodes, which contain our microdystrophin, as well as empty cathodes. Both of which contribute to the total viral load in humans. The improvements to our manufacturing process reduced the number of empty caps in the, and we are now averaging 90% full.

As part of our commitment to continuously improve our manufacturing processes over the years, we have now implemented process changes that remove the majority of empty file cabinets from the S. G T 001 drug product.

Effectively allowing target dosing two times 10 of the 14th vector gino's per kilogram to be achieved with fewer viral particles.

Thereby reducing the total viral load delivered to patients.

As with all Avi production methods pre purification, our process produces full catfights, which contained or micro dystrophin as well as empty houses both of which contribute to the total viral load administered.

Joel Schneider: This means that we can continue administering 2x714 viral particles that contain our differentiated microdystrophin construct and are able to do so while delivering fewer empty cabinets. This reduces the total number of viral particles without reducing our dose. This reduction, combined with a reduction in the way that patients are dosed and the protocol changes Catherine will discuss, is intended to support safe dosing of SGT001 for the duration of the IGNITE-GMD trial. One key criterion that was important to the FDA in terms of lifting the clinical hold was to understand the comparability of our previous SGT001 manufacturing process and our improved process with regard to microdiptyphin expression. This required the development of an assay that would produce a quantitative assessment of protein expression. Over the last few years, we've developed an in-vitro assay that measures the expression of our microadmystrophin constructs following addition of SGT001 to cells grown in culture.

Made with our new process and we were able to show that expression levels were similar between the process.

We are proud of our improved HSV based manufacturing process and we believe that it offers significant advantages in developing gene therapies in therapeutic areas that require large systemic doses.

I'll now turn the call over to Kathryn who will review the amendments to reignite DMD clinical protocol.

Joel Schneider: This assay enables us to rapidly and quantitatively measure the amount of microdystrophin expressed. We used this assay to quantify microdystrophin expression from SGT001 made with our previous process and SGT001 made with our new process. And we were able to show that expression levels were similar between the processes. We are proud of our improved HSV-based manufacturing process, and we believe that it offers significant advantages in developing gene therapies in therapeutic areas that require large systemic... I'll now turn the call over to Catherine, who will review the amendments to the IGNITE-DMD clinical protocol. Thank you, Joel.

Catherine Clary: After carefully reviewing our patient's safety data and consulting with clinical and scientific experts, we believe that there may be a correlation between viral load and complement activation. To mitigate the risk of serious drug-related adverse events associated with complement activation, we've amended the IGNITE-DMD protocol to include the prophylactic use of two complement inhibitors, ecolizumab and C1 esterase inhibitor, and also an increase in the prednisone dose utilized in the first month post-dose. Based on the advice of the experts we consulted about the time course of the role that complement plays in the immune system response, We believe that ecolizumab and C1S3 inhibitor, each of which acts at a different point in the complement activation cascade, may be more effective when administered together prior to the potential onset of the complement activation process.

Catherine Clary: We also believe that prophylactic administration of both of these agents will more effectively prevent or reduce the magnitude of a potential complement response. We are also increasing the prednisone dose in the first month of post-dosing from one milligram per kilogram to two milligrams per kilogram. We're also reducing the maximum weight of the next two patients' doses to 18 kilograms per patient, which in combination with the reduction in empty capsids that Joel just described will reduce the total viral load delivered to patients. While we won't need additional approval from the FDA to increase the weight limit after dosing our next two patients, patients 7 and 8, in the trial, we'll review the totality of the 30-day clinical and laboratory data for each After that, her protocol allows for an increase in the weight of subsequent patients.

No in ignite CMV and we look forward to sharing these results with you at an appropriate time.

I'll now turn the call back to along for a review of the Ultragenyx collaboration.

Thanks Catherine.

Now, let me turn to the strategic collaborations with the projects that we announced two weeks ago. The goal of which is to expand the pipeline of potential gene therapies for Duchenne and provides patients.

Catherine Clary: We intend to move as rapidly as possible with dosing additional patients while maintaining a strong focus on patient safety. As mentioned earlier, our submission to the FDA also included updated functional efficacy data, which included a six-minute walk test and the NorthStar ambulatory assessment for all patients dosed to date in IGNITE-DMD.

Increased royalty payments.

Importantly, as we move forward with this exciting collaboration we retain full rights to actually two 001 is what is the opportunity to establish additional partnerships around the city of yours, there were one or our micro dystrophin construct outside of Aviate Varian.

I will now turn the call over to Jan for a review of our third quarter financial results.

Catherine Clary: We're continuing to collect 12-month data and assessments from all six patients dosed in IGNITE-CMD, and we look forward to sharing these results with you at an appropriate time. I'll now turn the call back to Alon for a review of the Ultrajetix collaboration. Thanks, Catherine.

Thank you ally.

Earlier today, we filed our form 10-Q for the quarter ended September 32020, which contains detailed financial results and is available on the solid website.

Rather than focusing on that detailed information is provided in the 10-Q I'd like to provide an update regarding how our recent activity, which resulted in additional capital of approximately 64 million has extended our cash runway into the second half of 2021.

As of September 32020, we had cash and cash equivalents of $24.8 million.

As John noted in October we had two additional events that strengthened our balance sheet.

Ilan Ganot: Now let me turn to the strategic collaboration with Ultragenyx that we announced two weeks ago, the goal of which is to expand the pipeline of potential gene therapies for Duchenne and provide patients with additional therapeutic options. We believe this collaboration has a great chance of achieving these goals for two reasons. First, it combines our differentiated microdystrophin containing the neuronal nitric oxide synthase or NOS domain with Ultragenyx's HeLa producer cell line manufacturing platform for use with AAVH variants. We are excited that this collaboration creates opportunities for our unique microdystrophin to be used in multiple vectors and with multiple manufacturing processes. And to be working with a leader in the field of rare disease therapy to develop additional treatments for patients with Duchenne.

The first was the execution of the strategic collaboration agreement with Ultragenyx, which included a $40 million upfront investment in solid.

In addition, we also completed an ATM equity financing that resulted in net proceeds of $23.2 million.

These additional cash resources enable us to make further progress in support of our clinical and manufacturing objective in 2021, including re initiating dosing and ignite DMD in the first quarter.

I'll now turn the call back to Alon.

After what has been a rather challenging year. This is an exciting time for everyone at for that.

I would like to take this opportunity to thank the patients and physicians who are participating in the ignite DMD trials for their support of our efforts to advance testing two 001 and for their patients over the last several months as we resolve the clinical hold and as we take there were necessary steps to resume dosing safely.

Ilan Ganot: Second, the investment by Ultragenyx provides us with capital that enables us to move forward with the development of SGT001 and provides potential opportunities for longer-term value creation through milestones and royalties generated from programs developed under the collaboration. As part of this collaboration, Ultrogenics made a $40 million investment in SOLID at $5.11 per share and has agreed to pay up to $255 million upon the achievement of certain milestones and tiered royalties on worldwide net sales.

Can I have your disclosure timeline and and politically how many patients do you plan to does dramatic night studying all goes well with the purse too and and I realized that there are like that how soon do you think you could provide investors with clinical data from the newly dose patients. Thank you.

Hey, Thanks for the questions I've enjoyed is getting a good good manufacturing one and maybe a Catholic will talk about the conduct investor.

Ilan Ganot: Upon achievement of proof of concept, Solid has the right to opt-in to co-fund programs in the collaboration in return for participation in a profit share or increased royalty payment. Importantly, as we move forward with this exciting collaboration, we retain full rights to SET001, as well as the opportunity to establish additional partnerships around SET001 or our microdistribution construct outside of AAV8 variants. I will now turn the call over to Jen for a review of our third quarter financial results. Thank you, Alon. Earlier today, we filed our Form 10-Q for the quarter ended September 30, 2020, which contains detailed financial results and is available on the SOLID website. Instead of focusing on that detailed information that's provided in the 10-Q, I'd like to provide an update regarding how our recent activity, which resulted in additional capital of approximately $64 million, has extended our cash runway into the second half of 2021. As of September 30, 2020, we had cash and cash equivalents of $24.8 million.

Sure. Thanks for the question you know it would be highlighted previously our strategy around manufacturing them for them and it's really were aimed to be synergistic with our clinical mitigation strategy. That's supposed to be yeah. It's a specifically by removing empty passes from the.

Jennifer Zolkowski: As Alon noted, in October, we had two additional events that strengthened our balance sheet. The first was the execution of the Strategic Collaboration Agreement with Ultragenyx, which included a $40 million upfront investment in solids. In addition, we also completed an ATM equity financing that resulted in net proceeds of $23.2 million.

We're done we're in fact, we're going to continue to improve the products I off until the day, we lock down the manufacturing methods that.

It's our responsibility and I think our recent accomplishments both on the.

See improvements is called the analytical improvements related to our mutual expression that that's really highlight how forward looking we're trying to be.

On CMC product development and so.

Fundamentally I think we've done a lot of hard work and there's certainly more to do but we're excited and looking forward to going back to the clinic with a dramatically improved product.

Let me hand, it over to Catherine to answer your clinical questions.

Jennifer Zolkowski: These additional cash resources enable us to make further progress in support of our clinical and manufacturing objectives in 2021, including re-initiating dosing and IGNITE-DMV in the first quarter. I'll now turn the call back to Alon. After what has been a rather challenging year, this is an exciting time for everyone at Toilet. I would like to take this opportunity to thank the patients and physicians who are participating in the IGNITE-DMD trial for their support of our efforts to advance STD-001 and for their patience over the last several months as we resolve the clinical hold and as we take the necessary steps to resume dosing safely. We remain committed to the Duchenne community and thank its members and our employees for their continued support and dedication to our shared mission. Ladies and gentlemen, if you have a question at this time, please press the star and the number one key on your telephone. Once again, that's star number one to ask a question. And our first question comes from the line of Joseph Schwartz with SBB Learning. Your line is open. Mr. Schwartz, if your line is on mute, please unmute it.

Looking at all of that data for the first six patients three at the logos three the high dose and compile it and decide when the most appropriate time is to reveal that data, including some of the functional data such as the North star on the six minute walk test.

Okay got it and then it's.

Would you at least as your dosing 151 would there be.

Potential disclosure of just you're moving onto the next patient with like we got some sort of signal like that even.

In terms of whether I'm moving on.

In terms of when we're moving onto the next patient.

Maybe I shouldn't hey, yeah, that'd be all she jumped yeah.

We're not gonna guide to the level of disclosure that we will be you know providing begin.

I would just say that you probably would have come to figures from solids. We don't we're not shy about disclosures, we we update on everything's looking forward to it that you know.

Ilan Ganot: Our next question comes from Martin Auser with Craigslist. Your line is open. Hi, everyone. This is Morkan from ARTI.

Market needs to know and we are excited to be dosing vision forget into the first corner is kathie mentioned and knocked more data is getting gathered behind it you also asked when will we provide clinical data from the new patients that will be built.

Unidentified Speaker: Thanks for taking my question. I'm curious about more details on how the manufacturing change improves product quality, and specifically, what was the fraction of full or total caps before your manufacturing change? And then in terms of your manufacturing process itself, how confident are you that you'll be able to efficiently scale up this new process as you advance further? And then as far as the IGNITE-DMD trial is concerned, as you mentioned, you plan to resume dosing in Q1. I'm just curious to get a little better understanding of your disclosure timeline and, specifically, how many patients do you plan to dose in that IGNITE study if all goes well with the first two? And I realize it's early, but how soon... could you provide investors with clinical data from the newly-dosed patients? Thank you. Thanks for the questions. I think Joel is going to take the manufacturing one, and maybe Catherine will talk about Ignite D&D after that.

Again, not something we were gonna guys for today, but you know having already those six patient with collective data for over two years.

Some of those patients I think we have a very good understanding on on what we're looking for what we are comparing to what influence in what time initial biopsies and and all of that good hopefully be showed the appropriate for the next year.

Okay, great. Thanks for all the the clarity.

And once again, ladies and gentlemen that star one to ask the question. Our next question comes from Joseph for it but it'd be Mary.

Great. Thanks, very much and congrats on the progress Uhm I was just wondering if you could describe for some more how your quantity that's fine.

Joel Schneider: Sure. Thanks for the question. You know, as we highlighted previously, our strategy around manufacturing improvements really was aimed to be synergistic with our clinical mitigation strategy as opposed to the FDA. And so, specifically, by removing empty caskets from the SGT001 drug products, we're effectively reducing the total viral load given to patients. And so what we highlighted today, now having a number of GMP manufacturing runs under our belts, is that we're averaging about 90% full CAFs in the mix, so 90% full and about 10% empty. Previously, we were at about 50% full and empty, and as a reminder, all methods produce a mix of full and empty CAFs, and sponsors, based on their experiences, choose to either eliminate those or not. And, you know, in general, this information is fairly proprietary. It isn't really often communicated or shared.

Suppression produced in culture in cells.

Especially during manufacturing improvements in manufacturing comparability, which is how we most recently utilized in terms of having the whole lifted.

Similar to our our.

Our next question methods in the clinic, we also have a variety of different ways that we assess micro dystrophin expression.

Joel Schneider: We're trying to be a little forward-looking and a bit educational as we think about the path forward. So really importantly, and especially for ourselves, this removal of empty CAFs really has a meaningful, significant reduction in the total viral load. So same effective dose, so we're going back into the clinic with continuing our dose level of 2 times 10 to the 14th vector genomes per kilogram, but doing that with a smaller total load than any patient would be given. And so, alongside the clinical mitigation strategy, we think this is a very compelling and exciting way to try and ensure safety as we go back into Ignite DMV. I think the second question was related to our confidence in scaling.

In the in vitro setting as well.

They excitedly and this is something that the team is working on for a few years, we envision that this would be a way to demonstrate protein expression in the in vitro standing out as part of releasing characterization strategy of our chassis product and we're really excited to see what the FDA view this as a viable path to demonstrate protein expression and specifically.

Comparability of our improved manufacturing methods.

Okay. That's helpful. Thanks, and then.

I know that you.

Retained all rights to Caesars or one following ultragenyx deal and.

You're still able to explore additional partnerships around that product or even.

Potentially avail yourselves or other.

Other vectors to deliver it.

How are you thinking about.

The desire if at all to explore.

Explore other vectors and.

And and bring them in house and do that yourself.

Versus.

Joel Schneider: I think, you know, importantly, we transitioned our program from an adherent small-scale process to a suspension manufacturing method well before going into the fund. And so, all patients that have been dosed with SGT001 have been done so with a suspension-based manufacturing method. But that doesn't mean that we're done.

HOPPS securing additional partnerships like you just did.

Thanks, Good question Joe.

I mean, you you know the background here and.

Catherine Clary: In fact, we're going to continue to improve the product up until the day we lock down the manufacturing method. That's our responsibility, and I think our recent accomplishments both in the CMC improvements as well as the analytical improvements related to our in vitro expression methods really highlight how forward-looking we're trying to be in CMC product development. And so, fundamentally, I think we've done a lot of hard work, and there's certainly more to do, but we're excited and looking forward to going back to the clinic with a dramatically improved product. Now, I'll hand it over to Catherine to answer your clinical questions. Thanks, Joel, and thanks for the questions about IGNITE-DMD. We are truly excited about getting back into the clinic and dosing our next patient. And as Alon mentioned, there's a couple of activities that are taking place right now to ensure that that happens in the first quarter.

Catherine Clary: We are working with our investigators closely to identify patients who are at the appropriate weight as we send our protocol and regulatory packages to the IRBs and the IBCs at the different sites. Our first two patients will be, and we're going to proceed cautiously with our new risk mitigation strategies and new manufacturing process on board, which gives us confidence that we'll be able to treat patients safely. But for each of the first two patients, we will review 30 days of clinical and laboratory data with our DSMB before we move on to the next patient. So we'll dose the first patient in the first quarter, we'll evaluate the information from that patient with the DSMB, and then move on to the second patient. So some of that will depend on how many patients we do decide to dose next year. We haven't, we've got some plans, but we haven't revealed that number yet, but we do plan to actively dose throughout the year.

[music].

Catherine Clary: In terms of data disclosure, we're currently, this week, our sixth patient dosed in the trial who had the SAE, which led to the clinical hold, is actually in clinic getting his one year efficacy evaluation and safety evaluation. And we will be looking at all that data for the first six patients, three at the low dose and three at the high dose, combining it and deciding when the most appropriate time is to reveal that data, including some of the functional data, such as the North Star and the six minute walk test. Okay, I got it.

Unidentified Speaker: And then, so it sounds, say, would you at least, as you're dosing one by one, would there be potential disclosure of just you're moving on to the next patient? Would we get some sort of signal like that even? In terms of whether I'm moving on..., in terms of when we're moving on to the next patient. We're not going to guide you to the level of disclosure that we will be providing then. I would just say that you probably would have come to see this from Solid.

Ilan Ganot: We're not shy on our disclosures. We update on everything that's important, the market needs to know, and we are excited to be dosing patients again in the first quarter. As Catherine mentioned, a lot more data is getting gathered behind it. You also asked when we will provide clinical data from the new patients that will be dosed. Again, not something we're going to guide for today, but having already had those six patients and collected data for over two years on certain of those patients, I think we have a very good understanding of what we're looking for, what we're comparing it to, what endpoints and what timing for biopsies, and all of that will hopefully be shared appropriately next year. Okay, great. Thanks for all the clarity. And once again, ladies and gentlemen, I've starved myself one to ask a question. Our next question comes from Joseph Swartz with SBB Lyrics. Your line is open.

Operator: Great, thanks very much, and congrats on all the progress. I was just wondering if you could describe for us some more how you're quantifying the amount of microdystrophin, specifically what tools you use. Is it something like mass spec or something different? Is it semi-quantitative like Western blot? Is it a combination of the two?

Joseph Schwartz: Any insight here would be very interesting as everyone, including the agency, continues to learn about the best ways to characterize the potency of drugs like SGT-001. Thank you, Joe. It's good to hear from you. Glad we got the phone to work. This is Dead in Joe's Corner.

Unnamed Speaker: Sure. Thanks, Joe. We quantify microdystrophin expression in the clinic using multiple methods, immunofluorescence, which gives us a very good idea of how the protein is localizing to the muscle membrane. We also have previously disclosed data from both the use of Western blot and Mass MEK. So, we're looking at all three methods collectively and orthogonally to really fully capture the amount of protein expression that we're detecting. You're absolutely right.

Unnamed Speaker: The agency's expectations are clearly evolving, not just on the clinical side, but also on the CMC side. And so, you know, the in vitro expression assay that we developed is a very quantitative way and actually a very quick way to get an assessment of microdystrophin expression produced in culture in cells, especially during manufacturing improvements and manufacturing comparability, which is how we most recently utilized it in terms of having the hold lifted. You know, similar to our expression methods in the clinic, we also have a variety of different ways that we assess microdystrophin expression in the in vitro setting as well.

Unnamed Speaker: But I think, excitingly, you know, this is something that the team has been working on for a few years. We envision that this would be a way to demonstrate protein expression in the in vitro setting and as part of a release and characterization strategy for our GMT product. And we're really excited to see if the FDA views this as a viable path to demonstrate protein expression and, specifically, the comparability of our improved manufacturing methods. Okay, great. That's helpful. Thanks. And then I will know that you...

[music].

Operator: You retained full rights to SGT-001 following the ultragenics deal, so you're still able to explore additional partnerships around that product or even other vectors to deliver it. How are you thinking about [inaudible] Thanks. Great question, Joe.

Ilan Ganot: I mean, you know, the background here, and despite a lot of progress that has been made by us and many others in the field, I am still very concerned about the availability of a drug product, distribution, manufacturability, access, global access, and every time there's an opportunity to create more options for patients, I think we're going to take a very serious look at it. We also recognize that the gene therapies that are in the clinic today are showing us how to do things. They keep showing us how to do things; it's a very iterative process. The same thing goes for next-generation vector and delivery systems. We've been on. The Research Front on Next Generation Vectors for 5 or 6 years now, including a number of collaborations that we were public about and many that we weren't, and we are constantly looking to identify things that have better tropism for muscle, things that would allow easier manufacturability of drug products and improvements in delivery, but also in potential transgene and vector, and promoter biology. So, always open, always interested in a lot of in-house work in our labs but also open to collaborate with other like-minded groups. It's a great example of great chemistry between the teams and somebody that brings a lot of regulatory capabilities, regulatory affairs, manufacturing, delivery, and people that we are very impressed with and are excited to work with.

Operator: Great, thank you. Thank you, and I'm not asking any further questions at this time. Ladies and gentlemen, this concludes today's conference call. Thank you for your participation. You may now disconnect. Everyone have a good day. Thank you. [inaudible] Bo Cumbo, Bo Cumbo, Huidong Wang, Anupam Rama, Kevin Tan, Max Riso, Solid Biosciences Inc.

[music].