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: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Alex, maybe I'll start with you. You recently had a data update for your allo CAR T program. Walk us through -- and it was extensive, but walk us
through here what you presented.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Maybe in the context of those statistics that you just provided, what is the target profile or target product profile for azer-cel?
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JUNE 15, 2023 / 3:40PM, DTIL.OQ - Precision BioSciences Inc at Goldman Sachs Healthcare Conference
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And of the -- maybe the results that you just highlighted for us, that's across all the patients that have been treated in the relapsed population, but
maybe dig more specifically into the latest cohort because that is ultimately the dose, the lymphodepletion regimen that you're planning on taking
forward.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Remind us what was the median follow-up for these patients?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And there was some discussion around MRD status and you are following that in some of these evaluable patients. Help us or maybe frame for us
the use of MRD status as a predictive measure of durability.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And do you believe that, that's comparable as you think about the different patient populations that were studied in ZUMA-1 versus the patients
that you have here?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And there's been a lot of discussion around 3 months CR rate and that being maybe the metric that you should look for to understand durability.
We'll wait and see on that. But maybe when can we expect the next data disclosure?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Got it. And then at that point, we should have a better sense as to the longer follow-up and the potential for this 3 months.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: You touched on the FDA meeting so we can go there. Maybe provide some background on this meeting, what you're looking to get out of the
meeting and what next steps could potentially be?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Do you have a sense or maybe early thoughts on what a trial design could look like?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And do you have an understanding -- and this will come from the FDA meeting, but the number of patients or the extent of follow-up that might
be necessary to really support moving forward to a registrational trial?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Are there precedents out there that the FDA has looked at the totality of the data. And as you mentioned, you have many more than just these 7
patients that were in the latest cohort. But are there precedents where the FDA has looked at that versus saying, no, we only want to look at this
very specific set, this latest cohort. And in that cohort, you need x number of patients?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: How do you think about -- when you think about that totality of data there, just given that there are the majority of patients that are at different
doses from lymphodepletion regimens, which clearly we've seen then impacts safety and tolerability. How do you think about maybe the
interpretation of that data set and being able to really read through from those patients to the profile that you'll ultimately see with respect to
durability.
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JUNE 15, 2023 / 3:40PM, DTIL.OQ - Precision BioSciences Inc at Goldman Sachs Healthcare Conference
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
:
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Got it. And -- but that's not exclusive just to the, say, like the fourth line or the last line setting, that's across as auto CAR Ts are moving to the...
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And how do you think about maybe the landscape that has been evolving in a post-auto CAR T market? I mean, bispecifics, where does an allo
CAR T really fit in relative to these other agents?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And do we have a sense as to the number of patients, I guess, maybe when they're progressing post-auto CAR T? How many are you still retaining
that CD19?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And then as you think about auto CAR T moving to the earlier lines, as you mentioned, what does that imply for your strategy as a company in
terms of the trials you would need to run and your capacity interest even keep following them to earlier lines.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Maybe another way to ask that, as auto CAR T goes, it's in second line now, if it goes to the [front line], would you be interested in running studies
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Maybe let's jump to 19B then, which you've been referencing. Remind us how it's different than azer-cel?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: How does this compare? Maybe just the early profile you've been seeing. How does that compare to maybe other immune-cloaking approaches?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And then in terms of the data that you did present for 19B very early, a few patients, but walk us through what you saw there?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Same question for as I had for azer-cel maybe when can we expect the next update here?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Do you have a sense maybe in that context, if 6-month CR is not sufficient? Maybe what is the length of time that would make you feel comfortable
that each really do have a durable construct?
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JUNE 15, 2023 / 3:40PM, DTIL.OQ - Precision BioSciences Inc at Goldman Sachs Healthcare Conference
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And as you think about bringing forth azer-cel as well as PBCAR19B, there's been some talk around exploring a partnership or someone to take on
19B, maybe provide a little bit more color around that and what you would specifically be looking for in terms of a partner or a collaborator?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Perfect. Well, maybe we can jump over to the in vivo gene editing, which a lot of focus, obviously, is on the allo programs, but you guys also do
have this in vivo portfolio. Cassie, let me bring you in here. Maybe speak to the efforts across this portfolio. Notably, you have both partnered
programs, you have your own in-house programs, maybe describe for us first the overview and the setup of that portfolio.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Maybe let's start with the DMD data since you did just present it at a medical meeting. Walk us through the data that you showed and how
meaningful is it in the context of maybe other assets that are being developed in this space?
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JUNE 15, 2023 / 3:40PM, DTIL.OQ - Precision BioSciences Inc at Goldman Sachs Healthcare Conference
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And when you think about excising this hotspot that you've spoken about, how crucial is it that you have the ARCUS nucleus that's doing that
cutting versus maybe another editing tool?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And maybe one last question on this program. Just where do you stand in terms of bringing a candidate forward towards the clinic?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Maybe switching over to your partnership with Novartis. The decision to explore sickle cell, beta thal in the context of a field that is rapidly expanding,
it's evolving, there's products that could be approved quite soon. How did that decision come about in terms of how Precision and Novartis decide
to go forth in this program?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And maybe continuing with ARCUS and maybe the benefits or the advantages to this particular nuclease, how do you think about the risk of
off-target editing? And how is maybe -- maybe speak to that?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And then as you think about maybe where the field is going, there's -- now there's base editing, there's prime editing, there's gene writing, there's
epigenomic editing, how does ARCUS fit into that entire toolbox.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: And then you've got a number of other programs that we haven't fully touched on, maybe highlights from those where you stand with them and
a preview, if you're able to, into the R&D Day that's coming next month.
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: You've got a long...
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Any highlights from the OTC, HBV, PH1 programs that you'd like to share?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Alex, maybe a question for you here. Obviously, a lot going on across both parts of the house. How do you think about resource allocation? And
are there opportunities, say, with PH1 where it's been deprioritized here? Is that a prime opportunity to then look for a partner or someone to take
that?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Can you remind us on your cash position as of the end of 1Q and maybe what that runway is expected to support?
Question: Andrea Tan - Goldman Sachs Group, Inc., Research Division - Analyst
: Perfect. Well, looking forward to it. Thank you both for joining us. Thank you, everyone.
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