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: Mani Foroohar - Leerink - Analyst
: Thanks for taking my question. One quick one about the one patient that, I don't know if I heard this correctly, received a supplemental injection
when they didn't necessarily meet criteria. Can you give us a little more detail on sort of how that happens? And actions that may or may not need
to be taken in terms of tightening up sort of trial conduct to make sure that there is a little closer following of the criteria? And I have a follow-up.
Question: Mani Foroohar - Leerink - Analyst
: Okay. And separately, I think as everyone would expect, the percentage of patients that are injection free at 52 weeks or lower than 24 weeks. I
don't think that's surprising to anybody, given the underlying biology. But I guess this might be for the physicians on the call. Given that there's
going to be a meaningful number of patients in real world use post the pivotal trial, who will require supplemental injections, how frequently
would you be bringing patients back, either in the severe or broad population in real world practice?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 18, 2024 / 8:15PM, FDMT.OQ - 4D Molecular Therapeutics Inc To Host 150 Wet AMD Development
Day
So i.e., how much of the visit burden is going to be taken off the self off of the patient in your office, given that you're still going to have to track
them on some kind of (inaudible) ongoing basis?
Question: Salveen Richter - Goldman Sachs - Analyst
: Good afternoon. Thanks for taking my questions. Two questions for me. One is just given the non-inferiority trial as against 2 milligram aflibercept
and not the high dose and given that aflibercept arm isn't allowed supplemental injections, how do you think about the relevance of the study
design to clinical practice?
And then secondly, what is now your level of confidence on the safety profile, including the risk of developing cataracts at this point with the
sample size? And a follow-up. Thank you.
Question: Salveen Richter - Goldman Sachs - Analyst
: Excellent. Thank you.
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 18, 2024 / 8:15PM, FDMT.OQ - 4D Molecular Therapeutics Inc To Host 150 Wet AMD Development
Day
Question: John Miller - Evercore - Analyst
: Hi, guys. Thanks so much for giving us the update and taking my question. I'd like to start with a follow-up to Mani's question. Looking closely at
those image plots, it looks like there were actually a fair number of patients that got supplemental injections solely based on investigator discretion
that did not appear to meet the central criteria.
Honestly gave a little color about why those patients got those injections, but do you guys think that those weren't fair injections? It would seem
to materially change the rate of rescue needed if you, for instance, didn't include those patients.
So for a Phase 3 where central reads are going to be standard for determining eligibility for supplemental injection, do you anticipate to see different
rates there versus what you saw in the Phase 2b? And I have got a follow-up as well.
Question: Tazeen Ahmad - Bank of America - Analyst
: Hey, guys, can you guys hear me?
Question: Tazeen Ahmad - Bank of America - Analyst
: Okay. So thanks for the presentation today. A couple of my conversations recently with investors seem to revolve around how they think efficacy
should be defined for gene therapy versus how you as a company and also the doctors on the call define efficacy.
So specifically, how do you talk about the importance of injection freedom, meaning not needing any rescue injection versus having a reduction
in injection rates? And I know that the doctors have talked about this earlier in the presentation, but it seems to be a sticky point so I thought I
would ask again.
Question: Tazeen Ahmad - Bank of America - Analyst
: So I just want to, maybe on the thought of injection freedom, how long would injection freedom need to last? Is it forever? Or is there a minimal
amount if you could say the importance of injection freedom in this is?
Question: Gena Wang - Barclays - Analyst
: Thank you for taking my question. I have several parts of questions, but mainly for Phase 3 study design. So maybe first question is how do you
impute the BCVA data when patients, the drug arm that requires rescue injection?
And the second is how do you define response to a aflibercept?
And then third question is, I just want to double check with the baseline BCVA, was that defined at the week minus five? And is that common
practice?
Question: Gena Wang - Barclays - Analyst
: Yes. No. So more the screening stage, like you have (multiple speakers) one loading dose, and you define the patient will respond to aflibercept.
How do you define that?
Question: Gena Wang - Barclays - Analyst
: Thank you.
Question: Kostas Billoris - BMO - Analyst
: Hello. Thanks for taking our question and congrats on the progress. Two questions from us. Can you talk a little bit about the patient, the patient
who experienced subretinal macular hemorrhage in Phase 1/2? Any color you can provide around that would be helpful. And I have a follow-up.
Question: Kostas Billoris - BMO - Analyst
: The next question is on the Phase 3 design. The reference point here on Phase 3 appears to be after three loading doses, whereas in Phase 1 and
Phase 2, the reference point was after one or two loading doses. Given that we have two different reference points here and potentially difference
is also in the rest of criteria, how should we be thinking about comparing your result in Phase 1/2 versus Phase 3? Thank you.
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 18, 2024 / 8:15PM, FDMT.OQ - 4D Molecular Therapeutics Inc To Host 150 Wet AMD Development
Day
Question: Kelly Shi - Jefferies - Analyst
: Thank you for taking my questions. First question to doctors. So if you present the current profile of 4D-150 to patients, what would be your best
guess on the proportion of patients would be willing to switch to a gene therapy who are currently high dose EYLEA or VABYSMO? And I also have
a follow-up. Thank you.
Question: Kelly Shi - Jefferies - Analyst
: Thank you so much. I also have a follow-up, if I may. Okay. So 4D-150 show the sustainable control of CST in patients with different severity. But
when you take a closer look, you can see some like up and down over time. So it's kind of like a challenging to draw a trend line from any time
point, which actually sometimes investors question at a previous like a data cut.
Curious what is the biology behind this type of shape of curve, given that gene therapy is supposed to give a stable inhibition of VEGF, now like a
repeated injection regimen? EYLEA used to like a fluctuation on CST? Thank you.
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 18, 2024 / 8:15PM, FDMT.OQ - 4D Molecular Therapeutics Inc To Host 150 Wet AMD Development
Day
|