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: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Yeah, great. And I guess to start things off, maybe we could talk a little bit about the next-gen backbone chemistry that's now in the clinic, the
hypothesis behind having this neutral charge to your molecules and kind of what you've seen preclinically as you moved into the clinic now.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Yeah, I mean, it makes sense even with a single enantiomer, you'll still have a ton of sequence variation that you can explore there that really won't
matter. I guess for 2022, the biggest question for Wave is probably going to be translatability of all this preclinical data into the clinic. And on your
recent earnings call, you talked a little bit about some initial exposure data from the DMD program. And I wonder if you could comment a little
more on that and on a relative basis, where that exposure sits compared to what you've seen preclinically and how that affirms your confidence
in the translatability so far.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: And I guess where do you see yourself now on dosing in terms of therapeutic index, I guess? How much you're 2.5-fold higher on Cmax and 4.5
with very end of the curve? How much more room do you have to dose up if you need to?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: And I guess at this point, you have also a reason to believe that you should be seeing similar increase in exposure for the SNP3 program in HD as
well?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Yeah. Fair enough. Okay. And now let's shift over to C9, the C9 program, and I think we talked about this a little bit before the call. But yeah, we
had some disclosure from the Ionis/Biogen program this morning that they're discontinuing their C9 program. I guess there's limited details. We
don't know that much about knockdown or safety there. I guess maybe if you could just lay the groundwork about how your program and your
trial is different. Maybe a little bit about the preclinical safety profile you've seen and your confidence or not in the program ongoing right now.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: For sure. And I guess maybe one other key difference too is that you're including ALS and FTD patients. And I wonder if you could comment a little
bit on the level of support for targeting FTD versus ALS, and how we should think about that in the context of these early readouts for the Wave
program.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: And in which population do we know who's going to progress faster clinically, and if we could see early changes in one of those versus the other?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Okay. So then just going back to 2022 as a whole, you've guided for data for all three of these programs for this year. Understanding that these are
adaptive design trials, at least for C9 and HD, I wonder if you could go over exactly what you've been saying recently about when you -- what's the
trigger for disclosure and how you expect to disclose early data here.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: And I guess -- I don't know if you're going to be able to answer this, but do you expect to provide any more granularity moving forward on timings
perhaps this year or is that still TBD?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: No, fair enough.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Yeah. And I guess on ADAR2, I'm just curious if you could outline what are the final steps before you submit your IND in 3Q for that program in
AAT?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
MARCH 28, 2022 / 1:15PM, WVE.OQ - WAVE Life Sciences Ltd at Stifel CNS Days (Virtual)
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: And I guess would it be reasonable to assume this can be in the clinic next year?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Yeah. Makes sense. And then finally, I mean, you've been talking a little bit more about partnerships in manufacturing to help extend near-term
cash runway. I wonder if you could sort of talk about how that might impact your own manufacturing processes and maybe how quickly you could
reasonably generate manufacturing revenue if you did go down that route.
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Great. Well, in the last minute or two, any other final thoughts before I let you go?
Question: Alex Thompson - Stifel, Nicolaus & Company, Incorporated - Analyst
: Great, Paul. Thanks so much. It's always fun to talk to you.
|