WAVE Life Sciences Ltd Q4 2024 Earnings Call Transcript

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

Question: Salim Syed - Mizuho - Analyst : Great. Good morning, guys. Thanks for the questions, Paul, maybe just a few from us on DMD, since we're getting that read up pretty soon. Here the case confirmed for us. Are the discussions being had with the 48 week data, the discussions with regulators being had with the 48 week data? Or is it just the 24 week and Could you perhaps, just as we get closer here, bookend the scenarios that we should be considering coming out on this readout. And then just two quick clarifications here, just remind us where, how much PMO switchers, if that's even a consideration in this current data set. And then also, just as you're kind of considering, you know, with the current data that you have in hand just your pharmacoeconomic work, how you're preliminary thinking about pricing here. Thank you.

Question: Joon Lee - Truist Securities - Analyst : Congrats on the progress, and thanks for taking our questions. Correct me if I'm wrong, but our understanding is that pending outcome of the meeting PTC is having with the FDA in second quarter, there is a chance that Huntington lowering may be deemed an adequate surrogate endpoint reasonably likely to predict clinical outcome. If that is the case, would you still proceed with the MRI as a primary endpoint? And I have a quick follow up?

Question: Joon Lee - Truist Securities - Analyst : Great and then you mentioned Huntington, prevalence of more than 200,000 in the US and Europe alone, which is higher than the 50 to 60,000 historically cited by other companies. What changed? And you know, what kind of data are you looking at that tells you that the opportunity here might be much bigger than previously thought.

Question: Joe Schwartz - Leerink Partners - Analyst : Great. Thanks very much. I was wondering, how should we think about how the effect of WBE 06 could look after multiple doses of 200-milligrams? Can you share any general insights that you have from your pre-clinical, healthy volunteer or modeling work so that we can appreciate how the kinetics might evolve from the strong single dose data you reported last year.

Question: Joe Schwartz - Leerink Partners - Analyst : Okay, thanks. And then for WVE-007, how long will patients be treated? By the time you share data this year? Do you hope to match the total weight loss effect of GLP one therapy at a similar time point, recognizing that these would be cross trial comparisons, of course, or should we focus on the amount of non-lean body mass changes, given this is a different mechanism,

Question: Roger Song - Jefferies - Analyst : Great. Thanks for the question. So a couple questions on us. So the first one is for the ATD. I think Paul, you mentioned you. Are you expecting the. Higher dose on the multi dose and higher dose may be able to increase the M-protein production, correction and then total protein production. 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 04, 2025 / 1:30PM, WVE.OQ - Q4 2024 WAVE Life Sciences Ltd Earnings Call So my question is, do we know the coloration between the M-protein level and the total protein level versus the liver and the lung function given so far. We only have the replacement therapy, but we have some other genetic medicine try to increase the level a little bit higher than what you have seen being the set 200 meter web dose. Thank you.

Question: Roger Song - Jefferies - Analyst : Thank you. Maybe just a quick one for DMD, understanding you're talking with the FDA, and then for the potential regulatory path. So understanding you try also maybe be able to do some umbrella kind of a registrational trial. Would that be the topic we will get the update quarter, or there will be a coming a bit later? Thank you.

Question: Catherine Novack - Jones Trading - Analyst : I just wonder, you know, if you can give any more details about the multiple dose cohorts in ATD, you know, how many patients are you anticipating? Granularity as to when, in 2025, we should expect this. And then, you know, in conjunction with that, thoughts on the Maat threshold, you know, understanding that Z-protein is not functional, so we're not really looking at total AAT, do you think you know, consider the 20 micromolar M-AAT to be the threshold for success. Thanks.

Question: Catherine Novack - Jones Trading - Analyst : Got it. Thanks. And then I had just one more on your thoughts on siRNA obesity targets outside of inhibitory such as GPR75 for example. Is this something you could explore in conjunction with the inhibitory program? Thanks.

Question: Ryan Deschner - Raymond James - Analyst : For the 007 program what a late stage clinical study assessing the therapeutic as more of a maintenance therapy actually look like? And what would you anticipate an initial label to look like for this program, regarding positioning as a mono add on, or maintenance therapy, if successful.

Question: Madison El-Saadi - B. Riley - Analyst : Hi. Thanks for taking our question what are your plans for the MBA conference? Will you host an event around the conference to provide that regulatory update, and then second on 003 I know you had an updated analysis of CHDI, as you mentioned in your prepared remarks, just wondering if you could kind of remind us or characterize. The previously reported NFL data in terms of change from baseline. And then secondly, how do you see 003, profile relative to the exile, one targeted candidates. 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. MARCH 04, 2025 / 1:30PM, WVE.OQ - Q4 2024 WAVE Life Sciences Ltd Earnings Call