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. After the speakers' presentation, there will be a question and answer Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Mr. Tim Palmer, Corporate Communications Manager at Solid Biosciences.
Sir, you may begin.
Good morning. Thank you, operator. Before we get started, I would like to remind everyone that during this 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 progress, including statements about the ongoing IgniteDMD clinical trial. Actual results could differ materially from those discussed 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-nineteen pandemic and other risks described in the Risk Factors section of our most recently filed annual report on Form 10 ks and other periodic reports filed with the SEC. We undertake no obligation to update any forward looking statements after the date of this call.
Me on today's call are Elon Ganot, Co Founder, President and Chief Executive Officer of Solid Biosciences Doctor. Joel Schneider, our Chief Operating Officer Doctor. Catherine Clary, our acting Chief Medical Officer and Doctor. Carl Morris, our Chief Scientific Officer. For opening remarks, I'd like to turn the call over to Ilan Gonoe.
Yolong?
Thank you, Tim. Good morning and thank you all for joining us today. The focus on today's call is to provide an update on our current corporate activities as we continue to progress along our 2021 corporate goals and what our progress means for patients with Duchenne muscular dystrophy. First, we'll provide an update on dosing in IGNITE DMD. 2 patients were dosed this quarter in IGNITE DMD using SGT-one produced with our improved manufacturing process and under an amended clinical protocol.
As previously reported, patient 7 has been dosed safely and continues to do well. Today, we are reporting on an additional patient dosed. Patient 8 experienced a serious adverse event or SAE, but has since been discharged and as of the patient's 30 day follow-up visit, lab values have either returned to normal or continue to trend towards normal. In a moment, Doctor. Catherine Clary will review this SAE and the steps we are taking to evaluate its course.
Following Catherine's review of the patient dosing, Doctor. Carl Morris will present on encouraging long term biopsy data collected from the first 3 patients dosed at the 2x1014 vector genomes per kilogram dose. Carl will also be presenting this data later today at the American Society of Gene and Cell Therapy Annual Meeting. These data provide evidence of sustained microdystrophin expression for 12 months to 24 months post dosing and are potentially supportive of the recently reported positive trends in clinical biomarker and functional data from the IGNITE DMD study. To our knowledge, IGNITE BMD is the 1st Duchenne gene therapy trial to show durable microdystrophin expression out to 24 months.
In conjunction with our growing clinical experience, we believe that the totality of the clinical data will establish a risk benefit profile for SGT-one that will be meaningful to patients with Duchenne. Today, as Doctor. Joel Schneider, SOLID's Chief Operating Officer will share, we are also providing an update on our preclinical pipeline, which includes the nomination of SGT-three program as our next development candidate and an update on our ongoing collaboration with Ultragenyx. SGD-three will combine a novel capsid and our proprietary microdystrophin construct to enable a next generation gene therapy for Duchenne with enhanced delivery to muscle cells. Data from our novel capsid program have also been presented at ASG CT.
I'm pleased with our progress on all these fronts as we continue to generate additional evidence to support the long term potential of ST-two thousand and 1 while expanding our pipeline of differentiated gene therapies. I'll now turn the call over to Catherine, who will briefly review our clinical update and the steps we are taking as we progress the SGT-one program.
Thank you, Alain. As we reported in March, we resumed enrollment in IgniteDMD and have subsequently dosed 2 patients under our amended clinical protocol. As a reminder, we're working closely with our Data Safety Monitoring Board or DSMB to carefully review all the data generated with a built in waiting period of 45 days minimum between each dosing. We also previously reported that patient 7 dosed at the 2e14 vector genome per kilogram was dosed uneventfully and continues to do well. As we do with all patients, we are continuing to monitor this patient and will be collecting additional data throughout the year.
Today, we're sharing that patient 8 experienced an inflammatory response, which was classified as a serious adverse event and considered by the investigator to be drug related. This event is described in our investigator's brochure and is not considered unexpected. As of the patient's 30 day follow-up visit, laboratory values had either returned to normal or continue to trend towards normal. We've shared the data related to this FAE with the FDA and also the IGNITE DSMB and are working closely both internally as well as with external experts to further our understanding of the outcome of this dosing and how it may impact our clinical strategy moving forward. While we already had a minimum built in waiting period of 45 days between dosing patients 89, the complexity of this event requires us to carefully analyze all the data before continuing to dose additional patients in IGNITE DMD.
This will allow us to determine what if any changes we might make to the clinical protocol to further enhance patient safety, which is always our top priority. I'll now turn the call over to Carl, who will review the long term biopsy data from patients 4 through 6, which we believe support the potential of SZT-one to provide benefit to patients with Duchenne. Carl?
Thank you, Catherine. Today, I'm excited to share our analysis of the long term biopsy data from patients 4 to 6, which provide compelling evidence that a single dose of SGT001 at the 2e14 vector genomes per kilogram dose leads to sustained expression of our proprietary micro dystrophin construct containing the neuronal nitric oxide synthase or NMOS binding domain for up to 24 months post dosing. The muscle biopsies were collected from patients 4 to 6 and taken at 24, 18 and 12 months respectively post dosing of SGT-one. For each patient, the baseline and the last time point biopsies were taken from the right quadriceps, while the DAN-ninety sample was acquired from the left quadricep muscle. Over the next few slides, I will share with you immunofluorescence and western blood data from the long term biopsies that demonstrate micro dystrophin expression remains comparable to the levels observed in the day 90 biopsies for all 3 patients at this high dose.
I will then walk you through results highlighting microdystrophin protein function through the co localization of the dystrophin associated protein beta sarcoglycan as well as NMOS. Finally, we'll look at some morphological analyses of the muscle biopsies that demonstrate overall only minimal muscle deterioration since the day 90 time points with mild active dystrophic pathology observed in the long term biopsies. Collectively, these data are potentially supportive of the positive trends in the clinical biomarker and functional data, which we shared in March. Slide 7 shows immunofluorescence results from patients 4 through 6 at baseline day 90 and at their last time point. As we previously shared, the 90 day biopsies shared micro dystrophin positive fibers in all 3 patients that weren't seen in the baseline samples.
The longer term data that we are reporting today shows that proportion of microdystrophin positive fibers is maintained for up to 24 months. Specifically, patient 4 has 10% to 30% positive fibers at 24 months. Patient 5 is seen to have 85% positive fibers at 18 months, while patient 6 shows 50% to 60% positive muscle fibers at 12 months post dosing. On Slide 8, we show western blot data collected for patients 45 in the top panel and then patient 6 in the bottom panel. As shown in the table on the right hand side, patient 4's microdystrophin level remains below the level of quantitation of 5% of normal dystrophin, but is still detectable after 24 months.
Patient size micro dystrophin level is seen to be 69.8% of normal at 18 months compared to 17.5% at day 19. For patient 6, an average level of 20.3% of normal was found at 12 months post dosing compared to 8% at day 19. Slide 9 summarizes the micro dystrophin expression as assessed by Western blot and IF of the 3 2e14 vector genome per kilogram patients for up to 2 years post dose. The figure on the upper left shows the western blot I mentioned on the last slide. Interestingly, you can see there is an apparent increase in microdystrophin expression for both patients 56 that both had quantifiable levels at 3 months, while patient 4's micro dystrophin expression remains clearly detectable, but below the level of quantitation of 5%.
On the right hand side, we've shown the immunofluorescence results from our validated automated analysis. The results show measurement of stable, persistent microsrophin positive muscle fibers out to the 24 month time point that remain comparable to the level seen in the day 90 samples. Switching to additional functional analysis of the biopsies, we observed restoration of dystrophin associated proteins to the muscle cell membrane and show co localization in microdystrophin positive muscle fibers. The column on the left shows micro dystrophin in red, middle column shows beta sarcoglycan in green, while the right column shows these 2 images merged with the co localized proteins appearing yellow. As you can see, these 2 proteins co localized to the membrane, cell membrane, which demonstrates the capacity of our microdystrophin to recruit dystrophin associated proteins to the muscle sarcolemma.
On Slide 11, we show a similar analysis, but looking at NMOS activity and localization to the muscle membrane. Again, microsoraphan is shown in red on the left, while the right panel shows NMOS activity using an enzymatic stain as indicated by dark purple staining at the muscle cell membrane. These results demonstrate both N loss activity and its localization to the muscle membrane aligning with the expression of our microdystrophin protein. The continued N loss activity observed in these long term biopsies provides additional evidence of the durable functionality of our micro dystrophin construct. The next slide shows histological staining of the muscle biopsies from each patient at baseline 90 days and at 12 to 24 months.
Although these muscle biopsies show variable degrees of dystrophic pathology, it is encouraging to see limited disease progression between baseline after the long term time point. And out to 2 years, only mild active dystrophic changes in muscle pathology have observed, supporting the role of microdystrophin in slowing progression of muscle loss. So overall, these new results from the long term biopsies are encouraging as they demonstrate persistent and potentially increased microstripin expression between 90 days and out to 12 to 24 months. The minimal progression of muscle deterioration since the baseline provides potential support for the recently reported positive trends in the clinical biomarker and functional data from the IGNITE DMD as summarized on the previously reported efficacy heat map. As Alain noted at the start of the call, I'll be presenting these data at 1:45 p.
M. Today at the ASGCT conference. My full presentation will be posted to the SOLID BioSciences website once my talk is complete. Now, I'll turn it over to Joel for an update on the preclinical pipeline.
Thanks, Carl.
The growing body of evidence supporting the potential for SGT-one to provide benefit to patients with Duchenne is very encouraging and we look forward to generating more data for this program as we dose additional patients in IGNITE DMD. While advancing SGT-one remains our priority, we continue to explore new and innovative ways to improve outcomes for patients with Duchenne and to potentially address the needs of patients with other musculoskeletal disorders. Toward that end, we have been actively evaluating a library of novel, rationally designed AAV9 based castings. Today, we are announcing our next generation Duchenne microdystrophin gene transfer program called SGT003. This program is an internally developed preclinical asset that leverages our broad expertise in gene therapy and muscle biology.
Data presented at the ASGCT meeting by Doctor. Jennifer Green demonstrate that we have successfully developed a library of novel capsids with increased muscle tropism that corresponds with decreases in liver by distribution and drive improved efficiency compared with AAV9 in various in vitro and in vivo models. SGT-three is a preclinical candidate that combines a novel capsid designed to enhance delivery to muscles with our proprietary end nodes containing microdystrophin. We are currently conducting lead optimization for SGT003 and we look forward to sharing additional data with you as this program advances with a potential timeline to the clinic in approximately 18 months. This slide summarizes data from a dose response study exploring AAV9 alongside capsid candidate SLB-one hundred and 1.
As you can see, at all doses, our novel caps had led to increased by distribution and ultimately microdystrophin expression. We will aim to provide additional program and pipeline updates as we progress SGT003 as well as other candidates which leverage our strong internal research capabilities. In addition to our internal research and development efforts, we also have a collaboration with Ultragenyx to explore other next generation opportunities to develop additional Duchenne gene therapies. The companies have been collaborating to optimize candidate vectors that leverage our NMOS containing microdystrophin construct with an AAV8 like acid within the ultragenyx HeLa producer cell line manufacturing approach. I am pleased to share that this is a very productive collaboration that has leveraged each company's expertise and resources.
Ultragenyx is leading efforts around vector construction, optimization and creation of the HeLa producer cell line and in vitro and in vivo screening of the novel vectors has been expedited by routing expression analytics through SOLID's research team and leveraging our established assets. We expect to provide an update on this program by the end of 2021. As a company committed to improving outcomes for patients with Duchenne, we believe that having multiple ways to deliver our proprietary micro dystrophin constructs enhances our ability to make meaningful differences in these patients' lives and we are excited to expand our pipeline with additional opportunities. I will now turn to our Q1, 2021 financials. Earlier today, we filed our Form 10 Q for the quarter ended March 31, 2021, which contains detailed financial results
and is available on the
SOLID website.
Although I'm not going
to review our detailed results during today's call, I do want to highlight that during the Q1 of 2021, we closed the public offering, including the full exercise of the overallotment option, resulting in gross proceeds of approximately $143, 800, 000 before deducting underwriting discounts, commissions and offering expenses. This financing further strengthened our balance sheet and we ended the quarter with $268, 500, 000 in cash and cash equivalents. We expect that our cash and cash equivalents will enable us to fund our operating expenses into the Q4 of 2022. And I'll now turn the call back to Eilon for closing remarks.
Thanks, Joel, Karl and Catherine. Before we take your questions, I want to take a moment to review our 2021 priorities and anticipated milestones. As previously announced, we successfully achieved our Q1 2021 milestones and as Joe just discussed, today we are expanding our pipeline with SGT-three. We remain on track to present additional 90 day biopsy data in the second half of this year from patients 78 who were recently dosed. The long term biopsy results we presented today are encouraging and further increase our confidence in our technologies, in our team and strategies for making gene therapy a reality for patients with Duchenne.
I'll close by reiterating our commitment to the Duchenne community and to working every day to advance therapies that improve their lives and address the challenges of this horrible disease. This commitment for obvious reasons is a deeply personal 1 for me. What makes Solids such a special company is that every 1 of our employees is as committed as I am and we see these boys with Duchenne for who they are today, kids who just want to go out and have fun with their friends. We also know what the future holds for them without an effective therapy. And what inspires all of us at SOLID every day is the prospect of giving them a different future.
This commitment is what guides us through the challenges and drives us to build on our successes. We thank the Duchenne community, our employees and our investors for their continued support and dedication to our shared mission. I look forward to updating you as we continue to make progress in our clinical and preclinical programs. We'll now take your questions.
Your first question comes from the line of Joseph Swartz with SVB Leerink.
Hi, everyone. Congrats on all the progress. My first question is on the longer it seems like they correspond or correlate most closely to 6 minute walk in FEC clinical benefits as opposed to an SAA. So, I was just wondering, how are you thinking about being able to take advantage of this observation, if you agree with that and try to establish whether these clinical endpoints or a composite of these with or without an SAA might improve the chances to get micro dystrophin gene therapy such as SGT-one or 3 across the goal line with the FDA?
Great question, Joe. Good to hear from you. I think Carl will start and Catherine will finish.
Yes. Pretty good that you picked that up so quickly. Yes. There seems to be an apparent correlation, but it's a down of 3. And as you know, you can sort of make any associations you like.
I think we're encouraged by the data overall. There is variability expected in these biological assays. We took from different muscles from different at different time points. So but we are sort of quite sort of happy about it.
It's not unexpected
that we would see an increase in expression over time, but we need to get more data from more patients to really sort of start trying to look at specific relationships. I'll turn it over to Catherine to talk about the clinical trial and how we might be thinking based on that.
Sure. Yes. Thanks a lot for the question. We were certainly very encouraged by these long term results. And I think you asked a question which really gets to the heart of what we're thinking about as a company in terms of designing our registration trial.
How do we which outcome measures do we use? How can we find the robust way to measure functional benefit in patients in such a heterogeneous disease. And as Carl said, this is a small data set so far, but we're encouraged by it. We it's a bit early to start doing correlation analyses, although we're certainly thinking about that and we look forward to getting the data on our 2 additional patients and others so that we can make those decisions.
Okay, great. I appreciate that. And then as far as dosing additional patients in the future, I was just wondering what does that path look like for you from here? And could you characterize the SAE, which I heard you say was not unexpected, but had some complexity. Was this triggered by lab and or clinical findings?
What can you tell us about the last patient to be treated with SVT001?
Catharine, you can yes, keep going. Sure.
I'll just keep going, yes. So as I said, the patient had an inflammatory response with elements similar to some of our other patients, which is why it was not unexpected. However, the severity of the event was less severe than patient 6. We still are really looking at all the components of it from a causality perspective, the patient course, we're working both internally and with external experts to fully understand it so that we can move forward in a way that will ensure patient safety. You asked about the path to dose the next patient.
We are working of course with our DSMB. They reviewed the case and we'll going back to them with the results of our investigation and they will need to approve dosing the next patient. We'd already built in a minimum of 45 days between patients 89. So while we can't speculate on exactly when we'll dose patient 9, it already there was going to be a bit of a delay. And then we're working with FDA as well.
Thank you very much.
And your next question comes from the line of Gena Wang with Barclays.
I have 3 questions. The first 1 also regarding the patient 8 inflammatory response. Just wondering any additional color you can give regarding this patient? You did present at the ASGCT showing the seropositive AAV9 is related to complement activation. And I think Pfizer also shares some additional color on their safety, understanding of the safety.
So if you can give a little bit more color on this patient and what exactly that inflammatory response was and what kind of baseline characteristic from this patient, was it seropositive, also the platelet count, any other information you can give? That's the first question. My second question is regarding the SLB-one hundred and 1. Did you test in non human primates? What is the safety look like in non human primates?
And also which backbone was for was derived for SLB-one hundred and 1? My last question is more the future direction. You do have your own internal SGT003, but you also have a rare collaboration. How do you prioritize? Do you see this as internal competition?
Awesome questions, Gina. I think we're going to start with Catherine to talk a little bit about the SAE and then she can hand it over to Carl to finish that off and then talk about SOB-one hundred and 1 and I'll talk about the priorities at the end.
Thank you, Gina for the question. So, as I mentioned, patient 8 did have an inflammatory response with elements that were similar to what we had seen in some of the other patients. You asked about complement activation. It's interesting complement activation is part of the innate immune response and we've actually seen laboratory evidence of complement activation in all 8 of our treated patients. Patient 7 had, as we reported before, had some complement activation in lab, but did not it was lower than what we had seen in some other patients.
We're still really evaluating the elements of Patient 8. Every patient appears to be somewhat different and we haven't really identified a common factor with our SAEs, but we're still examining certain elements of it and we will once the results of that investigation are done, we can provide some additional information.
Hi, this is Karl. So yes, the ASGCT poster is highlighting that in the presence of antibodies, AV, it doesn't matter if it's AV9 or AV8, we showed both, can activate the complement system. So we think this is an effect that will be seen through within different programs. So I think we're getting a better handle on it and other companies are seeing that as well. Regarding SLB-one hundred and 1 and SUT-three, the capsid is derived from rational design that we did internally.
So rather than going through a computational analysis like a number of companies, we sort of went from the other way and looked at using our muscle expertise to think about how best to target muscles specifically. And we identified a number of capsids that look promising. It's very early on in the plan, and we're still in sort of lead optimization right now. So we haven't sort of finalized the candidate, but we plan to be moving into IND enabling studies as soon as possible once we've identified the specific capsid and specific transgene as well that we're using. I'll pass it back to Alain.
Just to say that we're actually making good progress with Ultragenyx. There's I enjoy seeing the 2 teams collaborate. There's some real complementary skill sets here when you think about doing DMD, but with another manufacturing system and it happens to be that their gene therapy people are here in Cambridge too. So I think there's a lot of good chemistry and very good people involved from both companies and I think they'll probably be leading the communication on the next steps there, but we are committed to really helping in any way we can advance that program. Ginny, you've heard me say this before, I don't think there could ever be enough programs there for DMD gene therapy.
There's just so many patients in such a massive unmet need. And I would just say that there is a potential to consider other musculoskeletal disorders as well as additional next generation gene therapies like SOB-three and Carl keeps confusing me with those numbers. And I look forward to continuing to update on the existing and more programs in the future.
Thank you very much.
Thank you, Gina.
And your next question comes from the line of Salveen Richter with Goldman Sachs.
Hi, thanks so much for taking our question. This is Sonia on for Salveen. So I know when you're currently evaluating what caused the inflammation in patient age, do you happen to have any initial hypotheses on why that might have happened? And then our second question was just when are we going see any additional functional data from patient 7 and 8?
Thank you.
Hey, thank you, Sonia. Catherine?
So thanks very much for the question. So we really I don't want to speculate right now on causality because we are looking at several things. As we reported, our principal investigator did deem the event to be drug related. And it does have some elements that are similar to our other patients, but less severe than we saw in patient 6. And patient 7, of course, dosed under our new protocol had a very safe dosing.
So we're evaluating a number of different factors. And as I said before, we'll definitely get back to you when we have a better handle on exactly what happened and what some of the causes were. In terms of the functional data, patient 7 is approaching a 90 day visit. It will be a while. We don't really look at functional data at 90 days to report it because of the steroids still lingering in the system.
So it will be a while before there will be additional functional data and we haven't actually guided to that.
Your next question comes from the line of Bola Amossa with Chardan.
Hi. Thanks for taking my call. Just wanted to a couple of questions about the long term data on patients 4 to 6. It's pretty noteworthy stuff. And I know it's n equals 3, there's variability in assays, etcetera, etcetera.
So we can't make easy conclusions. But could you update us on any hypotheses you might have on what factors could be a play that create a difference for what you see in patient 4 versus 56? And then is there anything you can do going forward to encourage results that are more like those seen in patients 56? And again, I know it's early days here.
Hey, Bola, it's good to have you. This is a calm question I know because I asked that to him just 2 days ago.
Yes. We just got the we just sort of pulled these data in and started analyzing very recently. So it is very early, but we do have confidence in our assays and given that we're seeing good results from all these different sort of orthogonal type assays, it does look as very encouraging that the way this dose is sort of a good dose and where we're generating improved responses over time. It's not unexpected, I guess, if you think about they just sort of continued production over time and then finding sort of more spaces to fill in on the membrane and stabilize the overall muscle. So it's very encouraging.
We're going to obviously spend a lot of time thinking about mechanisms to look at this. Patient 4 started unfortunately, like with all drug trials, you have responders, non responders. And unfortunately, patient 4 was not as had lower levels and below the level of quantitation, still very detectable levels. So there may be some threshold effect here that we are just not aware of. But importantly, patient 4 did see trend with functional improvements that we presented back in March.
So I think even with less than 5%, there's a sort of really promising results coming out. The overall sort of increased apparent increase is something that we're definitely going to be looking into.
I would just add, well, I would just add that this notion of long term durability, we got a little lucky here because this was supposed to all be 1 year biopsies that ended up being delayed because of COVID and because clinics were closed, patients couldn't show up, the 1 year visit ended up being a year later and now we have 2 year data. I think when you think about gene therapies as you obviously do all the time, the notion of duration, the notion of continued or even improved durability is clearly going to feature. And in a disease like Duchenne that is so hard to measure the functional outcomes, I'd like to hope that such biomarkers are going to provide us with a lot of confidence that this is not something that just disappears after, I don't know, 3 or 6 months.
Got it. And just another 1 really quickly. I think earlier in the call, someone referred to delayed kinetics, and I know we have seen that elsewhere in the AV space. But the magnitude of change seems greater here for solid and I know it's apples to oranges and n equals 3, etcetera, etcetera. But what's the state of art on biological reasons why there might be delayed kinetics?
Or is there a reason, an explanation that we can sort of watch on to the feel like maybe this is a real phenomenon going forward?
I'd love to talk to you for a while about this. If the muscle becomes more stable and so it's healthy, healthier, there may be sort of a more consistent production of protein production that's occurring. More my nuclei being fused in as the muscle can grow and build and therefore every time we get a positive STT001 positive nuclei in there, it could produce more protein. Again, it's all speculation. There's kinetically, we generally see stabilization after about 28 days in our preclinical models with sort of a continued but very slow increases.
So we do see a doubling in patients an apparent doubling in patient 6 and a threefold in patient 5, again apparent. So we don't know that there could be other things happening. And actually, importantly, someone asked about age and could age be a factor. Patient 4 was older, we really hope not, but patient 4 was about 11 years of age. So that's something that we really need to be thinking about as we move forward.
And your next question comes from the line of Monica Merchandini with Evercore.
Great. Thanks for taking the question. Based on what you're seeing for patient 8, do you think there are any additional potential changes in the protocol that might be able to further decrease the risk of these inflammatory events like changes in the dose of the immunosuppressive drugs or timing or other factors? And what might that look like? Thanks.
I mean, first, obviously, Catherine will take it, but I'll just say Monica that again, we learn so much from every patient and the idea here is to identify a long term solution to a pretty serious problem. And I feel that if things need to be tweaked, that's a great thing and hopefully arrive at a very happy place at the end. And other companies are hopefully doing the same. And I'll have Catherine add if she's got some.
Sure. Thanks, Monica. I mean, it's such an important question. And obviously, safety is really our first concern with these patients. And I mean, the really probably 1 of the biggest questions we are grappling with right now is to understand the events so that we can potentially modify the risk mitigation profile in a way that we think will optimize safety.
And we're still really in the middle of that investigation, including consulting with some external consultants who are experts in this area. So we don't really have I can't really tell you what our hypothesis is at this point, because we're still really exploring different options. But we will certainly share that with you, if and when we do decide to make protocol changes. And of course, those will need to be approved by our DSMB and shared with FDA as well. I think you also asked the question about the doses and that is 1 of the things we're looking at is dosing, but potentially other options.
Got it. Thank you.
And your next question comes from the line of Anupam Rama with JPMorgan.
Hi, guys. Thanks so much for taking the question. Just a clarification question here. Has the patient SAE and sort of clinical profile of this patient been shared with FDA and the DSMB? And what are the timeline for feedback from both of those groups?
Yes. Hi Anupam. Absolutely, shared with FDA and with the DSMB and we have ongoing dialogues. We're not really able to project timelines, but have been shared and discussed.
And at this time, there are no further audio questions. Are there any closing remarks?
I mean, look, thanks everybody for dialing in and have a great weekend and we look forward to talking again soon. Thank you.
And thank you. This concludes today's conference call. Thank you for your participation. You may now disconnect.