The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Anupam Rama - J.P. Morgan Securities LLC - Analyst
: Congrats on the update. Wasn't expecting this. So it's great to see. No, the initial study here starting in the second half 2025, any other gating factors
to starting the study beyond sort of getting IRB approvals which you mentioned in your opening comments?
And then are there any sort of KOL education considerations here we should be thinking about given the sort of novel dual-dosing administration
strategy? Thanks so much guys.
Question: Anupam Rama - J.P. Morgan Securities LLC - Analyst
: Great. Thanks so much for taking our question.
Question: Maury Raycroft - Jefferies LLC - Analyst
: Hi, good morning. Congrats on this update, and thanks for taking my question. Just wondering if you can contextualize the mouse cerebellum
expression data that you're showing -- that you showed in the slides versus what's seen with normal frataxin expression? And is there anything
additional you can say about the promoter or construct that's being used to optimize expression?
Question: Maury Raycroft - Jefferies LLC - Analyst
: Got it. Thanks for taking my question.
Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst
: Hi. Thanks for taking my question. Congrats on the update. Could you discuss the doses you plan on using both for the IV administration and the
ID administration? And what levels of frataxin expression you expect those to confer in both the heart and cerebellar dentate based on your
preclinical studies? Thank you.
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Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst
: What levels of frataxin expression you expect those doses to confer in the respective areas?
Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst
: All right. Thank you very much.
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Question: Joseph Schwartz - Leerink Partners LLC - Analyst
: Great. Thanks so much. And congrats on this important program. I wish you all the best with it. Can you -- I have a couple of questions. First, could
you talk about how quickly the study can progress with patient enrollment, and evaluation of subjects to confirm that the dosing and exposure is
as you'd expect it to be based on your pre-clinical data?
Do you need to confirm that you're seeing what you hope to see in a group of sentinel patients before you can enroll additional patients? And at
what point do you expect to be able to report the first data from patients treated with SGT-212? And what quantum of data might that encompass?
Question: Joseph Schwartz - Leerink Partners LLC - Analyst
: That's very helpful. Thank you. And then to what extent do you think that you can rescue the phenotype that's already present in FA adults who
have the disease by restoring frataxin after the disease has been underway for a while, based on what's known about frataxin and its function.
You've done a lot of great work in animals. Are any of these models informative about the ability to produce a benefit on clinical endpoints such
as LVMI and mFARS in the types of patients that will be enrolled in the Phase 1b study?
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Question: Joseph Schwartz - Leerink Partners LLC - Analyst
: That makes sense. Keep up the good work.
Question: Arthur He - H.C. Wainwright & Co., LLC - Analyst
: Hey, good morning, Bo and team, and congrats on achieving the milestone. I had two quick questions. So, Gabe regarding the mouse study, do
you guys have any data regarding the -- in those mouse model, how the treatment impact lifespan of the mice?
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Question: Arthur He - H.C. Wainwright & Co., LLC - Analyst
: Awesome. Thanks for that, Gabe. And my second question is actually regarding the manufacturing side. So could you tell us more about the
readiness regarding the manufacturing of the 212 for the upcoming clinical trial?
Question: Arthur He - H.C. Wainwright & Co., LLC - Analyst
: Oh, thanks, Bo. And congrats again.
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