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: Kripa Devarakonda - Truist Securities - Analyst
: Hey. Good morning, guys, and thank you so much for taking my question. The first question I have is the changes that you saw in the lean body
mass and thigh muscle volume, especially the thigh muscle volume, did you -- given that you plan to go forward in DMD, could you put that in
context for a DMD patient? Any metrics to better understand what sort of functional benefit that might translate into for patients.
And on the hemoglobin changes, was this something that was expected as per your preclinical data? Is there a sense of what the hemoglobin
levels -- baseline hemoglobin levels in DMD patients are? And would this be considered something that you would need to monitor in the Phase
2 trial? Thank you.
Question: Kripa Devarakonda - Truist Securities - Analyst
: Okay, thank you so much. I'll get back.
Question: Tyler Buren - TD Cowen - Analyst
: Hey, guys. Thanks very much for the data and the questions. So I realized the standard of care bar is low for DMD and that these patients are
ultimately losing ambulation. So as we think about the 1% to 2% increase in lean mass that we're seeing in 85 days here, is there any reason to
believe that the impact is plateauing, or are you seeing something that suggests that it could continue to improve over the longer term?
And then the second question is just, do you expect the Phase 2 study to have relatively traditional design, or would you propose one with unique
endpoints given 065's mechanism of action?
Question: Greg Harrison - Scotiabank - Analyst
: Good morning. Thanks for the update and thanks for taking our question. I'm curious how you're interpreting the lack of dose dependence when
it comes to change in bone mineral density and fat mass. Is this a function of the low end or are there any implications here you're thinking about
for future development and DMD or other indications in the future?
Question: Greg Harrison - Scotiabank - Analyst
: Thanks, that's helpful. And just given the broad range of activity you're seeing here, how do you decide how to prioritize your development going
forward beyond DMD? It sounds like other rare diseases could be likely, but there's also activity that could benefit patients with much more prevalent
diseases. So how do you decide how to focus your efforts?
Question: Greg Harrison - Scotiabank - Analyst
: Great. Thanks, again.
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Question: Thomas Smith - Leerink Partners - Analyst
: Hey, guys. Good morning. Thanks for taking the questions and congrats on the data. Just wondering if we could follow up on the hemoglobin
increases and if you could just talk about the effect you're seeing there, is this an effect you think would be desirable in DMD patients and how
does that impact your thoughts on dosing?
And then, secondly just operationally, if you could talk a little bit about the gating factors for starting the Phase 2, what kind of feedback you're
looking for from the FDA with the meeting in Q3 and how are you think about those selection in the Phase 2 study. Thanks.
Question: Kelly Shi - Jefferies - Analyst
: Congrats on the progress and thank you for taking my questions. I have two, firstly, quickly just curious on the adiponectin biomarker for fat mass.
Do we usually see a good inverse correlation between adiponectin and body weight?
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Question: Kelly Shi - Jefferies - Analyst
: Thanks very much.
Question: Jason Zemansky - Bank of America - Analyst
: Good morning. Thanks for squeezing us in and congrats on the update. I wanted to follow up on your previous comments in terms of development.
Are you prioritizing any specific stage of DMD? I mean, it sounds like you could expect it to be effective regardless of progression, but is there a
level where you think the benefit is more likely to be meaningful or I guess more sustainable?
Secondarily, can you speak to your confidence regarding the bleeding risks that have been a challenge for previous members of the class won't
manifest going forward, especially given the elevated hemoglobin levels. And then finally, I know you've downplayed pursuing this in the past,
but just based on the safety data, do you still believe 065 could be effective, at least in theory, for obesity patients? Thanks.
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Question: Jason Zemansky - Bank of America - Analyst
: Great and the potential for obesity patients.
Question: Jason Zemansky - Bank of America - Analyst
: Got it. Thank you for the color.
Question: Andreas Argyrides - Oppenheimer & Co. Inc. - Analyst
: Hey. Good morning and thanks for taking our question and for the progress here on the program. Jas, you previously alluded in the earlier comments
and just recently on ACE-031. So I was curious how these results compare to previous results seen from 031 and healthy volunteers with the
understanding that there was a different patient population. And then yeah, just, any other color on kind of differences given some similar mechanism
but just differences in the molecules. That'd be helpful. thanks.
Question: Andreas Argyrides - Oppenheimer & Co. Inc. - Analyst
: Thanks for the color, and congrats again. Thanks.
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