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: Joon Lee - Truist Securities Inc - Analyst
: For the INHBE program, what's the trigger for data disclosure? Would you need to have completed dosing in all 5 SAT cohorts for the disclosure or
once you have reached some other internal threshold? And I have a quick follow-up question.
Question: Joon Lee - Truist Securities Inc - Analyst
: Got it. And then you have a few drugs slated for accelerated approval, including for DMD and Huntington's. Can you confirm that they are all under
CDER and not CBER? And any risks to the accelerated approval process for you guys?
Question: Joon Lee - Truist Securities Inc - Analyst
: If I could squeeze one more in just on the Huntington's program, the recent data, very fresh data from Votoplam, the splice modulator. It seems to
imply that the MRI of the caudate may not be as consistent per their data. Any thoughts there? And also, they include a Stage III, which I don't think
you'll be including in your proposed Phase II/III, any thoughts based on that -- read-through from that data?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MAY 08, 2025 / 12:30PM, WVE.OQ - Q1 2025 WAVE Life Sciences Ltd Earnings Call
Question: Yun Zhong - Wedbush Securities Inc - Analyst
: A follow-up question on the AATD program reporting. Why do you divide the data set into two separate announcement, third quarter versus fall?
And why don't you just combine data from both cohorts into a more comprehensive data set? Do you think it's important to provide maybe some
piece of data as soon as it becomes available?
Question: Yun Zhong - Wedbush Securities Inc - Analyst
: Okay. Then on the DMD program, and would you expect to include monthly dosing data into the NDA submission package?
Question: Salim Syed - Mizuho Securities USA - Analyst
: I guess a couple from us. One on INHBE. Paul, from your comments around like the one, three and six months, is it that you haven't decided yet
which time point you want to cut at? Or are you looking at some sort of like -- and can you remind, do you have access to the blinded data here
that can help inform your decision? Or you just haven't disclosed? It just wasn't clear there. And then one on -- go ahead, sorry. And then a follow-up
on...
Question: Salim Syed - Mizuho Securities USA - Analyst
: Okay. Just to be clear, you're not looking at blinded data and you already have a predefined time cutoff, you just haven't disclosed it.
Question: Salim Syed - Mizuho Securities USA - Analyst
: Okay. And then just on DMD, your comments around the initial plans for using dystrophin expression for accelerated approval, are those plans
locked in? Like do you have it in writing from the agency that, that would be acceptable for an accelerated approval? Or is there any risk here that
if we do get a new Cedar head that would like to change things or has a different view on accelerated approval for diseases like DMD, is there any
risk there to that endpoint moving?
Question: Salim Syed - Mizuho Securities USA - Analyst
: Okay. So just to be clear, their view is that's the endpoint right now, but it's not locked in, correct? I mean, if we do get a new CDER head who has
a different view, there is potential for that to move, correct?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MAY 08, 2025 / 12:30PM, WVE.OQ - Q1 2025 WAVE Life Sciences Ltd Earnings Call
Question: Catherine Novack - Jones Research - Analyst
: Just thinking about the enrollment for the monthly DMD cohorts, how many additional DMD patients do you expect that you'll need to ensure
monthly dosing regimen at launch? And then following up on that, as you're enrolling in the study and working with these investigators, are you
getting any indication that providers are maybe reassessing the risk-benefit profile of gene therapy in DMD?
Question: Catherine Novack - Jones Research - Analyst
: Okay. That's helpful. And then thinking about the AATD program, how are you interpreting your increase in serum Z protein versus being significant
decrease in Z protein? What do you hypothesize is happening to the Z protein aggregates in the liver with DNA base editing versus RNA base
editing?
Question: Ananda Ghosh - H.C. Wainwright & Co - Analyst
: Two questions from me. The first one is a lot of GLP-1 discussion revolves around the pleiotropic effects. I was wondering what does the fundamental
biology talks about inhibin and the pleiotropic effects of weight loss in terms of the signaling pathway? The second question is, I wanted to
understand your thoughts on the Fazirsiran and how does it differentiate with your program?
Question: Ananda Ghosh - H.C. Wainwright & Co - Analyst
: Yeah. So the second one is with respect to Fazirsiran and your AATD program, differentiation in terms of what you have learned from the Fazirsiran
data?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MAY 08, 2025 / 12:30PM, WVE.OQ - Q1 2025 WAVE Life Sciences Ltd Earnings Call
Question: Ananda Ghosh - H.C. Wainwright & Co - Analyst
: That's right, yes, for the AATD program.
| 
