Solid Biosciences Inc To discuss SGT-003 INSPIRE DUCHENNE Data Update Conference Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Maury Raycroft - Jefferies - Analyst : Hi, good morning. Much congrats on the robust data update. I, I'm wondering for your meeting with FDA coming up in the middle of 2025, can you provide more specifics on the plan there? How many total patients of data do you need to have for the meeting? And do you need to have functional data such as time to rise velocity or SV 95C? And then will you disclose the new data cut to the public before or after the FDA meeting?

Question: Joseph Schwartz - Leerink Partners - Analyst : Hey Jenny on for Joe. I was just wondering if you can talk a bit more about how you're selecting patients for the inspired you Shin trial. Why do you think this is going to be key for differentiation. Also, given the high level of expression and biomarker movement, we're already seeing at 90 days, is there a potential to see clinical changes before 12 to 18 months? And is that something you've incorporated into your study design? And will we be able to see any of that data, or will it stay blinded for registration? Thank you. Yeah, thank.

Question: Biren Amin - Piper Sandler - Analyst : Yeah, hi, guys. Thanks for taking my questions and congrats on the data. Maybe it's a three-part question. First, I guess on the patient that experienced grade 3 thrombocytopenia, anything you can glean from patient baseline as it relates to this patient versus, the other 5 patients. Second question, when can we expect maybe the next data update? I think PPMD is coming up in June. Should we expect, additional follow-up there on ALL6 patients, including biopsy data on the most recent 3? And then I guess the third question is for Doctor McDonald with these data, how would you compare these data and SCT 003's profile to Alvius or to Reinic Bioo's DMD gene therapy program. Thanks. Great.

Question: Anupam Rama - J.P. Morgan - Analyst : Hey guys, thanks so much for taking the question and congrats on the data. I had a quick clinical trial question. You, in your kind of clinical trial slide, you talked about some of the changes that you're making relative to what others have studied, but I was still struck by the age range that you're studying for to less than 12 because I think some of the competitors have had some issues in terms of in that lower cohort 4 to 6 group. showing a benefit because those patients are still in the gain of function phase. How do we think about that? And along those lines for Dr. McDonnell, what would be your advice to the company in terms of controlling heterogeneity in the clinical trial overall, but in particular for that younger cohort of patients where it's been more difficult to show functional benefit.

Question: Charles C. Duncan - Cantor Fitzgerald & Co. - Analyst : Hey, good morning, Bo and Gabe. Congrats on, great data and thanks for taking the question. I had a question along the lines of the cardiac benefit that is emerging which seems pretty interesting to me. I guess I'm wondering if, you mentioned that you actively enrolled some, patients with cardiac signals. Was that based on the troponin levels or ejection fraction? Do you anticipate being able to use those biomarkers going forward and within the 20 patients by year end, are you enriching, would you like to enrich for a certain number of patients that have those biomarkers? And then if I could ask the KOL Doctor McDonald what he thinks about the left ventricular ejection fraction changes at 180 days. If that 8% is meaningful to him, thanks. Thanks, Charles, and thanks for.

Question: Sami Corwin - William Blair - Analyst : Hi there. Good morning and thank you for taking my questions. I have another one on the cardiac benefit. How are you thinking about capturing the potential functional cardiac benefits in a in a pivotal or confirmatory trial, and how might that support a stronger and more differentiated label? And then we've seen with other products a decrease in the positive fibers in older patients compared to younger patients. So I guess what are your expectations for dystrophin expression in 8 to 11 year olds given the robust expression you've seen in the first three patients? Thank you.

Question: Arthur He - H.C. Wainwright & Co. - Analyst : Hey, good morning, Boeing team. Congrats. So, for me, I just want to follow up on the cardiac function discussion. So, could you tell us what kind of bi marker, potentially could be recognized by the FDA, for an approval in terms of the cardiac function benefit. And, also, for Doctor McDonald, besides the Microdistricting expression level. What other data, including biomark or functional data, you'd like to see could be, solidified the XL approval pathway for SGTO3. Thank you.

Question: Gena Wang - Barclays - Analyst : Thank you for taking my questions. Also, thank you for such a comprehensive and thorough analysis of the data, a very good data. So maybe I would just follow up on the biomarker data, just discussed, in the past few questions. So, we know that we saw the data from 5 to 7 years old, those are relatively younger patients. I know you do want to enroll patient up to 12 years old. What are your thoughts like based on the other data, the older patient population, what could the biomarker look like? Would that be look a little bit worse than what we see now, in the younger patient population? And also, regarding the biomarker for muscle integrity, I think you have about 7 or 8 different measurements. Just wondering, I know these are the main numbers and how consistent across the three patients? I know it's only 3 patients, but I think that these are the data did not have a standard deviation if you can share that. And then quickly on the exclusion criteria for this study, I know in the past, you did mention you are, very cautious on the patient enrollment to make sure, the safety that will meet the standard. You can show pretty good safety. So you, maybe if you can, share a little bit more the thoughts going forward, are you willing to lose a little bit the inclusion criteria to include certain patient population with certain mutations? Yeah, thanks, Gina. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.

Question: Joon Lee - Truist Securities - Analyst : Congrats on the impressive data and thanks for taking our questions. Your biomarkers look very impressive, so just curious about your preference for month 18 assessment versus month 12 by some of your peers. Thank you. I apologize, June. I had a hard time hearing you. What was. That question?

Question: Joon Lee - Truist Securities - Analyst : To assess for efficacy at month 18 versus month 12 by some of the. Okay, that's an easy question. Look, I, we fundamentally believe in muscle integrity. Look. That once you understand that you're shoring up the muscle. It leads to a clinical benefit but you can also look at any of the companies that have published data in the past.

Question: Laura Chico - Wedbush Securities - Analyst : Hi, thank you very much for taking the question. This is Dylan on for Laura Chico. We're just wondering, so what are your expectations on the timing for regulatory feedback and beyond that, will you also engage with ex US regulators this summer? Yeah, the, and I'll take this and then, because we're running.

Question: Silvan Tuerkcan - Citizens JMP - Analyst : Hi Bo, congratulations on the data and thanks for taking my question. This is Josh on for Sylvan. Is there any context that we need to analyze these data in relation to the non-dystrophin related biomarkers for this age group? So like similarly to the functional endpoints in the 4 to 7 age group where you know they may increase, is there any potential for the non-distrophin biomarkers to improve? And is there any maybe natural history data for some of these biomarkers that you've looked at to sort of confirm? Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.