Editas Medicine, Inc. (NASDAQ:EDIT) Q3 2023 Earnings Call Transcript

Steve Seedhouse: Gilmore, just to clarify, I was asking about lymphocyte and neutrophil counts like longitudinally post-transplant, just because in the exa-cel data they don’t recover to baseline. So I’m just curious if that’s something you plan to share and it’d be interesting to know whether it’s different or the same given the different genomic target and different editor.

Gilmore O’Neill: So well, we have not seen, we’ve been actually very happy with our counts. We’re actually very happy with our counts to-date. But what I can follow-up on that.

Operator: Our next question is from the line of Luca Issi with RBC. Pleased proceed with your questions.

Luca Issi: Great. Thanks so much for taking my question. Two quick one here, maybe based on sounds like you’re obviously on track with those 20 patients by January 2024. Are you enrolling any adolescents? Just trying to understand if there is a scenario where your initial label will just include adults versus some of your peers would also get a broad label that also includes adolescents. So got any color there much appreciated. And the second one, quick one for Linda here. I think during the AdCom earlier this week, one of the potential suggestions to further characterize off-target editing risk was to actually do whole genome sequencing in 20 patients before and after the genetic manipulation. So I just wonder if that’s something that you’re contemplating to do. Thanks so much.

BaisongMei: Yes. Thank you for your question. I take your first question and I pass that. I think we have a plan to dosing adolescent patients and also for the general clinical program, we are intended to go to patients of all ages. So that’s kind of what because this disease is essentially starting with genetic disease start from very young age. So we intend to actually be able to have this molecule benefit a patient from all ages. So that’s kind of our intention I mean, all those things of course, the label you mentioned will have further discussion and alignment with FDA.

Linda Burkly: Yes. Thank you for your question. Yes, that was a very interesting conversation at the AdCom. One of our orthogonal methods, and this also came up at the AdCom was the method of using a biochemical method applied to naked DNA. And this is a method, one of the methods that we use, which is unbiased method in which the naked DNA is taken from cells and you subject it to cutting with your RMP and then you do whole genome sequencing basically to look for off-target editing. And so this is called digenome. And so this is one of our methods that we use and look at with our drug product before putting that drug product into patients. So I think this is one of the robust criteria that we — one of the robust methods that we use in our approach.

So I think that’s basically one of the reasons that we are confident in our approach to, in addition to the other methods that we use in silico and guide seek that were described. I guess that’s basically what I’m prepared to share at this moment. But there are many other aspects to our approach that will also make us very confident to — about our approach to the off-target editing package that we’re preparing.

Operator: Our next question is from the line of Jay Olson with Oppenheimer. Pleased proceed with your question.

Jay Olson: Hey, congrats on the progress and thank you for taking the question. Maybe just another question on read across from the exa-cel AdCom since there was some discussion about long-term monitoring and surveillance of these patients. Can you just share your thoughts and plans to follow EDIT-301 patients long-term in a post approval setting? Thank you.

Gilmore O’Neill: Yes. Thanks for your question, Jay. So we certainly will have a long-term follow-up for patients. And so that study is designed to actually follow the patient up to 15 years for anybody who actually dose with EDIT-301. And that’s also consistent with the regulatory requirements. So that’s absolutely our plan.

Operator: Next question is from the line of Liisa Bayko with Evercore ISI. Please proceed with your question.

Jingming Chen: Hey, this is Jingming on for Liisa. Thanks for taking our questions and congrats on the progress. So we have three questions. First, we know that CDC has filed an appeal to the Court of Appeals. When do we expect to hear the decision for that? And then, second, as I know you mentioned that more substantial data set for sickle cell is coming in the middle of 2024. Just wondering when should we expect more substantial data set for the TDT patients. And then the third is, you mentioned that in the second half you plan to engage with the FDA to seek alignment or regulatory path for EDIT-301. We are wondering, will you inform the Street on the result of the discussion? And if so, what’s the timing for that? Thank you.

Erick Lucera: Hi, this is Erick. Just with respect to any cases in front of — court cases in front of the CDC or anything like that, we don’t really want to comment on those until the final decision is actually rendered. So we’ll just be anxiously awaiting for all of that just like you.

Baisong Mei: Yes. So this is Baisong for your second question about the middle of next year for the program, and that two part of that. One is, as we just shared that we will have 20 patients dose by January timeframe next year, then by middle of next year, we will have substantial follow-up for those 20 patients. And we’ll also have continued dosing patients over the course. So we’ll have a lot of data over the middle of next year for sickle cell disease. Yes. Regarding the —

Jingming Chen: I want to ask for the beta thalassemia?

Baisong Mei: Yes, yes, absolutely, absolutely. Yes, in there — yes, because I saw your question probably covered both, but I just make sure that we cover on that. And we actually have very strong momentum for EdiTHAL study. Also, as we just shared, we actually already have eight patients enrolled and we continue to enroll and dosing patients, but we have not shared what is the goal for next year, how many patients, EdiTHAL patients were dosed yet? We will share that in appropriate time. And your third part of your question is about FDA engagement and outcome. And so we are — we were well engaged with FDA and we continue to have the engagement with FDA, as we just mentioned that we actually have this RMAT designation allow us a lot more frequent interaction with the agency and including senior management over the agency. But we have not share — have a timeline to share the outcome yet. We’ll share that in appropriate time.