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: Luca Issi - RBC Capital Markets, LLC - Analyst
: That's fantastic. Glad that RBC has that privilege. So we have a long list of questions, but maybe before we talk about individual programs, can you
just talk about what progress the organization has made over the last few months and maybe most importantly, what's ahead here for Wave?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Terrific. Terrific. So helpful. Maybe pivoting to C9, you already mentioned you already have pretty impressive evidence of target engagement
actually in humans, right, with these reduction in poly(GP). Can you just talk about the importance of poly(GP) as a biomarker? What's the likelihood
that those impressive reduction in poly(GP) will actually translate into benefit in patients?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: That's terrific. And then maybe you already talked about dosing, but if I recall it correctly, in the 30-milligram and 60-milligram dose, we also saw
some NfL elevations. Can you characterize those NfL elevations? Were they sustained over time, were they transient? How you're thinking about
the safety profile more broadly of the molecule so far?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Super helpful. And also, I think the 60-milligram dose is no longer being pursued, if I recall it correctly. Can you just maybe articulate on the reasons
behind that?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Got it, got it. That's helpful. And I think one of the other biomarkers that you were tracking was also p75 in the urine. I don't think we've seen that
data quite yet. Well, maybe if you could talk more broadly what's the -- how are you [putting] that biomarker more broadly for these patients; and
two, when are we going to see that data?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Got it. Got it. That's helpful. Maybe let's pivot to Huntington for a minute. Actually, maybe before we talk about some of the nuances around the
dosing and whatnot, can you just talk about why you believe that allele specificity matter and why you think that some of the failures that we've
seen in the space, including the Ionis's and Roche program recently -- even if they're not giving up on a molecule, they are running a new phase
there at a different -- prioritizing patients with younger and lower disease burden. But maybe high level, why do you think that allele specificity
matter for Huntington?
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MAY 17, 2022 / 6:05PM, WVE.OQ - WAVE Life Sciences Ltd at RBC Capital Markets Global Healthcare Conference
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Super helpful. I think you guided data in 2022.
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: How should we think about timing, number one? And then maybe number two, I believe that this data set is going to be for SNP3. Is there any
plan to go back and potentially revisit SNP1 and 2? So I've got two questions: one on timing, but obviously the data; two, any plan for SNP1 and
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Got it. Got it. It's helpful. What's the bogey? What's the bogey for success at this point? I mean, we've seen Ionis's and Roche showing a reduction
mutant huntingtin in the CSF up to 40%, I think, 40%, 45%. Is that a realistic bar?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Got it. Got it. That's helpful. You already touched upon DMD earlier. You have better pharmacology on this novel molecule with higher Cmax,
higher area under curve. Again, what's the bogey here in term of what you need to see in the muscle biopsy? Again, the data is coming in 2022.
Where do you see the bar for success for that program?
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MAY 17, 2022 / 6:05PM, WVE.OQ - WAVE Life Sciences Ltd at RBC Capital Markets Global Healthcare Conference
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Got it. Got. That's helpful. Maybe on A1AT, you talked about it earlier, and again, you have something -- impressive proof of concept in animals. I
think you got to like 18.5 micromolar, which is obviously above what is considered therapeutically relevant. Two questions. One, how are you
thinking about that program? Two, when is the earliest you can possibly have an IND over the finish line?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Terrific. I know we only have a couple of more minutes. Maybe two more questions, if I may. One, why not editing the DNA for A1AT? I mean, we
see a lot of companies that are trying to edit the DNA. And they will be kind of one and done versus -- obviously RNA editing would still require
some chronicity or some chronic dosing at some point. So what will be your pushback to what I just said?
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Terrific. Last question. I know we're out of time, but $110 million in cash. I think you have a runway into Q2 2023. How are you thinking about ways
of extending the runway? And maybe related to it, we have seen a few deals in the space in the RNA editing, including like Shape and Roche and
some of the other -- ProQR and Eli Lilly. How are you thinking about [BD]? Would the deals that they have signed be compelling to you? How are
you thinking about that far?
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MAY 17, 2022 / 6:05PM, WVE.OQ - WAVE Life Sciences Ltd at RBC Capital Markets Global Healthcare Conference
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: Fantastic. Appreciate your time, Paul. (multiple speakers)
Question: Luca Issi - RBC Capital Markets, LLC - Analyst
: And yeah, we will talk soon. Thanks again.
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MAY 17, 2022 / 6:05PM, WVE.OQ - WAVE Life Sciences Ltd at RBC Capital Markets Global Healthcare Conference
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