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: Gena Wang - Barclays - Analyst
: Okay, so maybe, I think usually we ask about the wet AMD program, but this time I would change because you will have a CF data coming in near
term, and that will be pretty important update for the companies. So maybe share with us what we saw in the past that we know that you did have
a high protein expression, but if we want, we did not see too much improvement.
Now you will have less severe patients, so maybe walk us through why we think, in this next update, should we expect to see the functional FEVI
or the other lung clearance index? Should we see the improvement in there and why we should see that?
Question: Gena Wang - Barclays - Analyst
: So maybe how many patients should we expect to see and then in terms of the baseline FEVI, I don't know if you can share that yet?
Question: Gena Wang - Barclays - Analyst
: Okay. And FEVI, you did mention you saw anecdotally 5%, 6% improvement. Would that be the bar we should be looking for when you have the
data update?
Question: Gena Wang - Barclays - Analyst
: What about the lung clearance index? What could be the benchmark there?
Question: Gena Wang - Barclays - Analyst
: Okay. And I think for the non-sense mutation, I think Vertex and Moderna are also doing the RNA therapy. They likely will also share data later this
year.
So what do you think, like, of course, it's all moving targets, right? So like how do you think from the gene therapy perspective, the efficacy bar, do
you think it will still be similar? Do you need to be competitive with the RNA therapy?
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 12, 2025 / 2:00PM, FDMT.OQ - 4D Molecular Therapeutics Inc at Barclays Global Healthcare Conference
Question: Gena Wang - Barclays - Analyst
: Okay. How would you share the data? Will it be a press release and the conference call and we'll walk through the data? What would be a medical
conference?
Question: Gena Wang - Barclays - Analyst
: Okay, good. Now switching gear to your 150, and I do have two different indications, wet AMD and DME. You shared the data on both indications.
So maybe some pushback from the investors, I would say like in the data overall looks pretty good, but you do have an investor pushback, the
durability. And I think that was the key question that may be hurting the stock when certain data update, the stock reacts differently when maybe
when we look at it.
So maybe help us understand, see it from the durability question and also what endpoint would be the most important based on your interaction
with the doctors?
Question: Gena Wang - Barclays - Analyst
: Good. And then maybe -- I know from the investor community we're focusing on -- they were focusing on injection freely and in addition to the
burden reduction, right? So maybe based on your doctor feedback, which one day care or the patient perspective, which one they care more and
also there is a caveat is what the -- in the practice versus clinical trial setting, the criteria of a retreatment is very different, right?
So usually in the clinical trial setting, it's much loose or in the reality, real world, maybe every doctor has their own threshold when they wanted to
do the injection. So you have a large variability there. So how do you -- more thinking fast forward and given the Phase 3 trial and I think you have
a very good chance to show positive study. But fast forward, when you go to the doctors and the patient, how do you send in the message? What
do they care most when you're presenting the data profile?
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 12, 2025 / 2:00PM, FDMT.OQ - 4D Molecular Therapeutics Inc at Barclays Global Healthcare Conference
Question: Gena Wang - Barclays - Analyst
: You also mentioned that you already start (inaudible) Phase 3 trial and what is your -- I know it's a little bit difficult question like a (technical difficulty),
what is, say your assumption building for the enrollment completion? The time that it would take.
Question: Gena Wang - Barclays - Analyst
: And then regarding the physician or patients, the threshold, the treatment burden reduction, what is the number there? So I talked to also lots of
doctors, there's some like your investigators. So they're like wide range of numbers, but let's see for the treatment, the injection free rate, usually
the threshold is about 50%. So the burden reduction will be a little bit higher. So we'd love to hear your thoughts based on your interaction.
Question: Gena Wang - Barclays - Analyst
: Okay. And we are out of time, but do you want to quickly -- one question each for you two. For Chris is, our conversation with the [peer] is for gene
therapy, actually very simple formula, standard care times 5 or times 3 to 5, that's the price they're willing to pay for gene therapy. Is that aligned
with your thinking and quickly for Uneek, cash and the runway assumption?
|